Trial | Phase | Enrollment | Study Type | Start Date | Status |
[NCT01042457] | Phase 3 | 24 participants (Actual) | Interventional | 2009-05-31 | Completed |
A Study to Determine the Safety and Efficacy in Lipoprotein Lipase-Deficient Subjects After Intramuscular Administration of AMT-011, an Adeno-Associated Viral Vector Expressing Human Lipoprotein LipaseS447X[NCT01109498] | Phase 2/Phase 3 | 14 participants (Actual) | Interventional | 2007-08-31 | Active, not recruiting |
Measurement and Analysis of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Patients With Autoimmune Diseases Treated With Mycophenolate (MPA)[NCT00351377] | Phase 3 | 111 participants (Actual) | Interventional | 2006-06-30 | Completed |
A 6-month, Prospective, Open-label, Randomized, Controlled, Pilot Study Evaluating the Efficacy, Safety and Toxicity of an Optimized Immunosuppressive Regimen of CellCept (Mycophenolate Mofetil, MMF) and Reduced Doses of Both Calcineurin-inhibitors and Pr[NCT01213394] | Phase 3 | 2 participants (Actual) | Interventional | 2010-10-31 | Terminated(stopped due to Poor recruitment.) |
Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy[NCT00303719] | Phase 2 | 342 participants (Actual) | Interventional | 2002-03-26 | Terminated(stopped due to IRB Study Closure) |
A Phase I/II Dose Escalation Trial of Clofarabine, in Addition to Melphalan and Thiotepa as Myeloablative Regimen Followed by an Allogeneic Unmodified Hematopoietic Stem Cell Transplant From HLA-Compatible Related or Unrelated Donors for the Treatment of [NCT00423514] | Phase 1/Phase 2 | 38 participants (Actual) | Interventional | 2006-11-20 | Completed |
A Phase III, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Rituximab in Subjects With ISN/RPS Class III or IV Lupus Nephritis[NCT00282347] | Phase 3 | 144 participants (Actual) | Interventional | 2006-01-31 | Completed |
Impact of Lymphocyte Anti-metabolite Immunosuppressions on Donor-Specific Anti-HLA Antibody and Kidney Graft Outcome: Open-label, Multi-center, Single Arm, Phase 4 Trial (DoSAKOM)[NCT03794492] | Phase 4 | 169 participants (Actual) | Interventional | 2018-03-31 | Active, not recruiting |
Immunosuppressive Medications for Previous Participants in Clinical Trial NIS01 (ITN005CT, NCT00014911)[NCT01309022] | | 0 participants | Expanded Access | | No longer available |
A Multi-Center, Phase II Trial of HLA-Mismatched Unrelated Donor Hematopoietic Cell Transplantation With Post-Transplantation Cyclophosphamide for Patients With Hematologic Malignancies[NCT04904588] | Phase 2 | 300 participants (Anticipated) | Interventional | 2021-09-30 | Recruiting |
Registry of IgA Nephropathy in Chinese Children[NCT03015974] | | 1,200 participants (Anticipated) | Observational [Patient Registry] | 2016-01-31 | Recruiting |
Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) With Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases[NCT05805605] | Phase 2 | 56 participants (Anticipated) | Interventional | 2023-05-01 | Recruiting |
A Randomized, Open-label,Controlled Phase II b Study to Demonstrate Efficacy and Safety of Sirolimus Chronic Rejection After Lung Transplant[NCT04415476] | Phase 2 | 0 participants (Actual) | Interventional | 2020-06-30 | Withdrawn(stopped due to Study did not proceed to IRB approval.) |
A Pharmacoeconomic Study Comparing the Use of Mycophenolate Mofetil or Cyclophosphamide as Induction Therapy in Lupus Nephritis Patients in Egypt[NCT05195086] | | 122 participants (Actual) | Observational | 2018-07-01 | Completed |
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 1 | 120 participants (Anticipated) | Interventional | 2020-12-03 | Recruiting |
Organ Function Preservation by the Combination Treatment of the optImuM Dose of calcineUrin Inhibitor and Mycophenolate Sodium in Kidney Recipients: OPTIMUM Study[NCT01159080] | Phase 4 | 350 participants (Actual) | Interventional | 2010-04-01 | Completed |
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 1 | 30 participants (Anticipated) | Interventional | 2014-12-05 | Recruiting |
Optimizing Haploidentical Aplastic Anemia Transplantation (CHAMP) (BMT CTN 1502)[NCT02918292] | Phase 2 | 32 participants (Actual) | Interventional | 2017-07-03 | Completed |
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen[NCT02722668] | Phase 2 | 16 participants (Actual) | Interventional | 2017-05-15 | Active, not recruiting |
A Phase III, Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF, Modified Release (MR) Tacrolimus/MMF and Neoral (Cyclosporine)/MMF in de Novo Kidney Transplant Recipients[NCT00064701] | Phase 3 | 668 participants (Actual) | Interventional | 2003-06-30 | Completed |
A Double-Blind, Placebo -Controlled, Randomized Study to Assess the Efficacy and Safety of Zenapax in Combination With CellCept, Cyclosporine, and Corticosteroids in Patients Undergoing Cardiac Transplantation.[NCT00048165] | Phase 4 | 434 participants (Actual) | Interventional | 1999-08-31 | Completed |
A Study of Two Different Treatment Strategies in IgG4-related Disease Patients With Re-elevation of Serum IgG4 Level During Maintenance Remission Period: a Randomized Double Blind Placebo Controlled Multicenter Study[NCT05974683] | | 108 participants (Anticipated) | Interventional | 2023-08-01 | Not yet recruiting |
An Open-label, Single-sequence Study to Investigate the Effects of BMS-986165 at Steady State on the Single Dose Pharmacokinetics of Mycophenolate Mofetil (MMF) in Healthy Male Subjects[NCT03660436] | Phase 1 | 131 participants (Actual) | Interventional | 2018-08-14 | Completed |
A Trial to Evaluate the Long Term Prognosis in Rosai-Dorfman Disease[NCT05284942] | Phase 4 | 20 participants (Anticipated) | Interventional | 2011-10-01 | Recruiting |
A Multi-Center, Randomized, Double Blind, Phase III Trial Evaluating Corticosteroids With Mycophenolate Mofetil vs. Corticosteroids With Placebo as Initial Systemic Treatment of Acute Graft-Vs-Host-Disease (BMT CTN #0802)[NCT01002742] | Phase 3 | 236 participants (Actual) | Interventional | 2010-01-31 | Completed |
Attenuating Ischemia Reperfusion Injury After Living Donor Renal Transplantation[NCT01149993] | Phase 4 | 0 participants (Actual) | Interventional | 2010-06-30 | Withdrawn(stopped due to FDA clinical hold, IND withdrawn.) |
Research Institute of Nephrology, Jinling Hospital, Nanjing University School of Medicine, Nanjing, China.[NCT01269021] | | 176 participants (Actual) | Interventional | 2010-11-30 | Completed |
An Open-label, Balanced, Randomized, Two-treatment, Four-period, Two Sequence, Single Dose, Replicate, Crossover, Bioequivalence Study of Mycophenolate Mofetil 500 mg Tablet of Dr. Reddy's Laboratories Limited Comparing With That of Cellcept 500 mg Tablet[NCT01283841] | Phase 1 | 48 participants (Actual) | Interventional | 2007-11-30 | Completed |
Randomized Controlled Trial Study of Anti-r-HuEpo Associated PRCA Treated by Cyclosporine and Mycophenolate Mofetil (MMF) Compared With Cyclophosphamide and Prednisolone[NCT01288131] | Phase 3 | 8 participants (Actual) | Interventional | 2009-01-31 | Terminated(stopped due to We observed >90 % efficacy in cyclophosphamide and Prednisolone group for treatment of anti-i-HuEpo associated PRCA) |
A Three-Part Phase 1 Study to Determine the Potential Drug Interaction Between ACH-0144471 and Midazolam, Fexofenadine and Mycophenolate Mofetil in Healthy Subjects[NCT03108274] | Phase 1 | 35 participants (Actual) | Interventional | 2017-04-18 | Completed |
A Multicenter, Two Arm, Randomized, Open Label Clinical Study Investigating Renal Function in an Advagraf® Based Immunosuppressive Regimen With or Without Sirolimus in Kidney Transplant Patients[NCT01363752] | Phase 4 | 853 participants (Actual) | Interventional | 2011-03-08 | Completed |
Allogeneic Islet Cells Transplanted Into the Omentum[NCT02821026] | Phase 1/Phase 2 | 4 participants (Actual) | Interventional | 2016-05-31 | Completed |
Effect of Cyclophosphamide Versus Mycophenolate Mofetil in Induction Therapy of Lupus Nephritis in Nepalese Population[NCT03200002] | Phase 2 | 49 participants (Actual) | Interventional | 2014-01-01 | Completed |
The Mechanisms and Significances of Synergistic Effects of Mycophenolic Acid and Lipopolysaccharide on Interleukin-1β Secretion by Mononuclear[NCT02435368] | | 10 participants (Anticipated) | Observational | 2015-04-30 | Recruiting |
Vorinostat With Gemcitabine/Clofarabine/Busulfan for Allogeneic Transplantation for Aggressive Lymphomas[NCT04220008] | Phase 2 | 30 participants (Anticipated) | Interventional | 2023-06-01 | Not yet recruiting |
Mycophenolate Mofetil Therapy for Reduction of the HIV Reservoir[NCT03262441] | Phase 2 | 5 participants (Actual) | Interventional | 2018-02-12 | Completed |
Safety and Efficacy of Thymus Transplantation in Complete DiGeorge Anomaly, IND#9836[NCT01220531] | | 0 participants | Expanded Access | | Approved for marketing |
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes[NCT02224872] | Phase 2 | 18 participants (Actual) | Interventional | 2014-08-31 | Completed |
Effect of Sirolimus or Mycophenolate With Tacrolimus on Survival of Pancreas and Kidney Grafts in Type 1 Diabetic Recipients After Simultaneous Pancreas and Kidney Transplantation[NCT03582878] | Phase 4 | 238 participants (Actual) | Interventional | 2004-01-01 | Completed |
To Evaluate Prevention Effect Of Everolimus and Low-dose Tacrolimus in Comparison With Standard-dose Tacrolimus Therapy With Mycophenolic Acid on the New Onset Diabetes Mellitus After Transplantation in the Renal Allograft Recipients[NCT02036554] | Phase 4 | 234 participants (Anticipated) | Interventional | 2013-03-31 | Recruiting |
A 12-Month, Randomized, Open-Label, Phase IIA Study Evaluating the Safety and Efficacy of Siplizumab in Combination With Belatacept and MPA Compared to Standard of Care Immunosuppression in de Novo Renal Transplant Recipients (ASCEND)[NCT05669001] | Phase 2 | 90 participants (Anticipated) | Interventional | 2023-11-30 | Not yet recruiting |
Everolimus in Association With Very Low-dose Tacrolimus Versus Enteric-coated Mycophenolate Sodium With Low-dose Tacrolimus in de Novo Renal Transplant Recipients - A Prospective Single Center Study[NCT02084446] | Phase 4 | 120 participants (Actual) | Interventional | 2012-12-31 | Completed |
Open Label, Multi-center, Randomized Study to Compare of Tacrolimus and Steroids in Combination With Mycophenolate Mofetil or Without Mycophenolate Mofetil in Liver Transplantation With Hepatitis B Virus(HBsAg) Positive[NCT02075242] | Phase 4 | 170 participants (Anticipated) | Interventional | 2014-01-31 | Recruiting |
A Pilot Randomized Study to Assess the Effect and Safety Profile of Thymoglobulin® in Primary Cardiac Transplant Recipients: A 12-month, Single Center, Randomized, Open-label Study of Efficacy Comparing Immediate Treatment With and Without Thymoglobulin® [NCT03292861] | Phase 2 | 60 participants (Anticipated) | Interventional | 2018-09-13 | Enrolling by invitation |
Clinical Evaluation of the Long-term Benefit of Enteric-coated Mycophenolate (MPAs) After Liver Transplantation[NCT05707520] | | 500 participants (Anticipated) | Observational | 2022-12-04 | Recruiting |
Shortened-duration Tacrolimus Following Nonmyeloablative, Related Donor BMT With High-dose Posttransplantation Cyclophosphamide[NCT01342289] | Phase 1 | 127 participants (Actual) | Interventional | 2011-08-31 | Completed |
The Effect of Everolimus on the Pharmacokinetics of Tacrolimus in Renal Transplant Patients, and the Effect of ABCB1、CYP3A4、CYP3A5、PORGenetic Polymorphism on the Two Drugs[NCT02077556] | Phase 4 | 14 participants (Actual) | Interventional | 2014-04-30 | Completed |
Open Label, Multicenter Randomized Control Study to Investigate the Incidence of NODAT (New-Onset Diabetes After Transplantation), Safety and Efficacy of Corticosteroids Early Withdrawal in Liver Transplanted Recipients[NCT02095418] | | 152 participants (Anticipated) | Interventional | 2014-02-28 | Recruiting |
Allogeneic Stem Cell Transplantation for Patients With Hematological Malignancies Using Multiple Unrelated Cord Blood Units[NCT00547196] | | 10 participants (Actual) | Interventional | 2005-08-16 | Active, not recruiting |
Efficacy and Safety of Induction Strategies Combined With Low Tacrolimus Exposure in Kidney Transplant Recipients Receiving Everolimus or Sodium Mycophenolate[NCT01354301] | Phase 4 | 300 participants (Actual) | Interventional | 2011-05-31 | Completed |
A Phase 1, Open Label, Single Sequence, Drug Interaction Study of the Pharmacokinetics of ASP015K and Mycophenolate Mofetil (MMF) After Separate and Concomitant Administration to Healthy Adult Volunteers[NCT01364987] | Phase 1 | 24 participants (Actual) | Interventional | 2009-05-31 | Completed |
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 2 | 90 participants (Anticipated) | Interventional | 2014-04-22 | Completed |
A Phase 2a, Randomized, Open-Label, Active Control, Multi-Center Study to Assess the Efficacy and Safety of Bleselumab in Preventing the Recurrence of Focal Segmental Glomerulosclerosis in de Novo Kidney Transplant Recipients[NCT02921789] | Phase 2 | 67 participants (Actual) | Interventional | 2017-05-22 | Completed |
Initial Systemic Treatment of Acute GVHD: A Phase II Randomized Trial Evaluating Etanercept, Mycophenolate Mofetil (MMF), Denileukin Diftitox (ONTAK), and Pentostatin in Combination With Corticosteroids (BMT CTN #0302)[NCT00224874] | Phase 2 | 180 participants (Actual) | Interventional | 2005-09-30 | Completed |
Allogeneic Hematopoietic Cell Transplantation Using a Non-Myeloablative Preparative Regimen of Total Lymphoid Irradiation and Anti-Thymocyte Globulin for Older Patients With Hematologic Malignancies[NCT00185640] | Phase 2 | 303 participants (Actual) | Interventional | 2003-03-31 | Completed |
Low Steroid Dose Combined With Mycophenolic Acid (Myfortic) Compared With High Dose Steroid for Minimal Change Nephrotic Syndrome[NCT01197040] | Phase 3 | 117 participants (Actual) | Interventional | 2009-10-31 | Completed |
Phase III, Open, Randomized, Parallel-group Clinical Trial, to Evaluate the Efficacy and Safety of Treatment With Prednisone, Cyclosporine, Mycophenolic Acid Versus Prednisone and Mycophenolic Acid in Lupus Nephritis Type III-IV-V.[NCT01299922] | Phase 3 | 0 participants (Actual) | Interventional | 2011-02-28 | Withdrawn(stopped due to IT was impossible to find patients) |
Investigating New Onset Diabetes Mellitus in Kidney Transplant Recipients Receiving an Advagraf-Based Immunosuppressive Regimen With or Without Corticosteroids - A Multicenter, Two Arm, Randomized, Open Label Clinical Study[NCT01304836] | Phase 4 | 1,166 participants (Actual) | Interventional | 2011-01-22 | Completed |
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 2 | 46 participants (Actual) | Interventional | 2010-05-19 | Terminated(stopped due to The study experienced lower accrual rates after the onset of COVID. Upon review of the collected data it was deemed that an adequate amount of subjects has been enrolled to date to assess study aims.) |
Use of Zevalin to Enhance the Efficacy of Non-Myeloablative Allogeneic Transplantation in Patients With Relapsed or Refractory CD20+ Non-Hodgkin's Lymphoma[NCT01811368] | Phase 2 | 20 participants (Anticipated) | Interventional | 2013-03-12 | Active, not recruiting |
New Onset of Type 1 Diabetes Mycophenolate Mofetil-Daclizumab Clinical Trial (Preservation of Pancreatic Production of Insulin Through Immunosuppression-POPPII #1)[NCT00100178] | Phase 2 | 126 participants (Actual) | Interventional | 2004-05-31 | Completed |
Open Label Balanced Randomized Two-treatment Two-period Two-sequence Single Dose Two-way Crossover Oral Bioequivalence Study of Mycophenolate Mofetil Capsules 250 mg in Normal Healthy Adult Human Subjects Under Fed Conditions[NCT01080417] | | 84 participants (Actual) | Interventional | 2008-06-30 | Completed |
A 2 Step Approach to Matched Related Hematopoietic Stem Cell Transplantation for Patients With Hematological Malignancies-5+5 Dosing[NCT03712878] | Phase 2 | 10 participants (Actual) | Interventional | 2018-09-19 | Active, 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 2 | 135 participants (Actual) | Interventional | 2005-11-18 | Active, not recruiting |
Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial[NCT01092962] | Phase 3 | 70 participants (Actual) | Interventional | 2010-09-30 | Completed |
Reduced Intensity Myeloablative Total Body Irradiation and Thymoglobulin Followed by Allogeneic Peripheral Blood Stem Cell Transplantation[NCT00709592] | Phase 2 | 42 participants (Actual) | Interventional | 2008-07-21 | Completed |
Population Pharmacokinetic and Pharmacodynamic Model-based Dosing Strategy of Mycophenolate Mofetil in Pediatric Hematopoietic Stem Cell Transplantation(HSCT) Patients[NCT04868786] | Phase 1 | 20 participants (Anticipated) | Interventional | 2020-10-26 | Recruiting |
A Phase 2 Pilot, Multicenter, Single Arm Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of GSK1070806 Plus Standard of Care for the Prevention of Delayed Graft Function in Adult Subjects After Renal Transplantation[NCT02723786] | Phase 2 | 7 participants (Actual) | Interventional | 2016-08-27 | Terminated(stopped due to Lack of efficacy) |
Phase I/II Study Using Prophylactic Donor Lymphocyte Infusion Early Post-Transplant After Allogeneic Hematopoietic Cell Transplantation Using Post-Transplantation Cyclophosphamide for High-Risk Hematologic Malignancies[NCT05327023] | Phase 1/Phase 2 | 430 participants (Anticipated) | Interventional | 2022-05-23 | Recruiting |
Evaluating the Safety and Efficacy of Chemotherapy in Patients With Relapsed Small Cell Lung Cancer in Combination With Allopurinol and MycoPhenolate (CLAMP Trial)[NCT05049863] | Phase 1/Phase 2 | 36 participants (Anticipated) | Interventional | 2023-02-27 | Recruiting |
A Randomized Trial of Treatment in Patients With IgG4-Related Disease[NCT02458196] | Phase 2 | 60 participants (Anticipated) | Interventional | 2015-04-30 | Recruiting |
Phase II/III Randomized Study to Improve Overall Survival in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus Mycop[NCT02416388] | Phase 2/Phase 3 | 3,100 participants (Anticipated) | Interventional | 2015-01-31 | Recruiting |
Prospective Multicenter Observational Cohort Study of Comparative Effectiveness of Disease-modifying Treatments for Myasthenia Gravis[NCT03490539] | | 167 participants (Actual) | Observational [Patient Registry] | 2018-05-07 | Completed |
A Comparison of PTCy-ATG and ATG Strategy in Haploidentical HSCT for Acute Graft-versus-host Disease Prophylaxis[NCT03689465] | Phase 4 | 260 participants (Anticipated) | Interventional | 2018-10-29 | Recruiting |
CYCLONES - CYClophosphamide LOw Dose and No Extra Steroid[NCT03492255] | | 49 participants (Actual) | Interventional | 2018-04-12 | Terminated(stopped due to Significative difference between percentage of renal response (primary outcome) between the two study arms.) |
A Phase 1/1b Adaptive Dose Escalation Study of Mycophenolate Mofetil (MMF) in Combination With Standard of Care for Patients With Glioblastoma[NCT05236036] | Phase 1 | 60 participants (Anticipated) | Interventional | 2022-08-08 | Recruiting |
Single-Arm, Open Label, Interventional Phase II Clinical Trial Evaluating MGTA-456 in Patients With High-Risk Malignancy[NCT03674411] | Phase 2 | 22 participants (Actual) | Interventional | 2019-01-02 | Active, not recruiting |
Belatacept in De Novo Heart Transplantation - Pilot Study[NCT04477629] | Phase 2 | 12 participants (Anticipated) | Interventional | 2020-08-06 | Recruiting |
REduCing Immunogenicity to PegloticasE (RECIPE) Study[NCT03303989] | Phase 2 | 35 participants (Actual) | Interventional | 2018-06-14 | Completed |
A Partially Blinded, Prospective, Randomized Multicenter Study Evaluating Efficacy, Safety and Tolerability of Oral Sotrastaurin Plus Standard or Reduced Exposure Tacrolimus vs. Mycophenolic Acid Plus Tacrolimus in de Novo Renal Transplant Recipients[NCT01064791] | Phase 2 | 298 participants (Actual) | Interventional | 2009-12-31 | Completed |
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis[NCT03426969] | Early Phase 1 | 3 participants (Actual) | Interventional | 2018-01-31 | Completed |
Prospective Multicenter Randomized Openlabel Study to Evaluate the Benefit on Renal Function at 12months Post-transplantation of Immunosuppressive Treatment With Withdrawal of Calcineurin Inhibitor at 3months and Combining Mycophenolate Sodium-Everolimus [NCT02334488] | Phase 3 | 329 participants (Actual) | Interventional | 2014-12-11 | Completed |
Prospective, Randomized 2 x 2 Factorial Trial of Rabbit Anti-thymocyte Globulin Induction (Single vs. Alternate Day Administration) at Renal Transplantation, With Delayed Calcineurin-inhibitor Withdrawal vs. Minimization[NCT00556933] | Phase 4 | 180 participants (Actual) | Interventional | 2004-04-01 | Completed |
Rituximab Versus Mycophenolate Mofetil in Children With Steriod-dependent Nephrotic Syndrome: A Single-center, Randomized Controlled Trial[NCT05843968] | Phase 2 | 46 participants (Anticipated) | Interventional | 2023-07-01 | Recruiting |
Mycophenolate Mofetil in Systemic Sclerosis: A Phase 1 Pharmacokinetic Study of Orally Ingested Mycophenolate Mofetil Tablets in Patients Suffering From Systemic Sclerosis[NCT03678987] | | 35 participants (Actual) | Observational | 2018-09-13 | Completed |
A Phase 2/3, Prospective, Randomized, Multi-center, Open-Label, Controlled Trial to Assess the Efficacy & Safety of Cellular Immunotherapy With MDR-102 for Induction of Immune Quiescence™in Recipients of HLA-mismatched, LD Kidney Transplants[NCT03605654] | Phase 2/Phase 3 | 172 participants (Anticipated) | Interventional | 2024-12-31 | Not yet recruiting |
Hematopoietic Cell Transplantation in the Treatment of Infant Leukemia and Myelodysplastic Syndrome[NCT00357565] | Phase 2 | 20 participants (Anticipated) | Interventional | 2005-11-30 | Recruiting |
A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) For the Treatment of Acute GVHD in Patients Who Receive Allogeneic Hematopoietic Stem Cell Transplantation[NCT00136903] | Phase 2 | 32 participants (Actual) | Interventional | 2005-04-27 | Completed |
Immunosuppression With Antithymocyte Globulin, Rituximab, Tacrolimus, Mycophenolate Mofetil and Sirolimus, Followed by Withdrawal of Immunosuppression, in Living-donor Renal Transplant Recipients[NCT01318915] | Early Phase 1 | 10 participants (Actual) | Interventional | 2011-07-25 | Terminated(stopped due to The stopping rule for incidence of acute rejection was met.) |
Multi-center, Open-label, Randomized Controlled Phase 4 Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients Undergoing Maintenance Therapy With CNI Plus MPA.[CORAL Study][NCT05193565] | Phase 4 | 206 participants (Anticipated) | Interventional | 2021-11-19 | Recruiting |
Clinical Study of MMF in Treatment of IIM-ILD and Its Effect on Peripheral Blood Treg Cells[NCT05129410] | Phase 4 | 20 participants (Anticipated) | Interventional | 2020-12-01 | Recruiting |
Calcineurin-Sparing in a Steroid-free Maintenance Immunosuppression Protocol After Kidney Transplantation[NCT01062555] | Phase 1/Phase 2 | 527 participants (Actual) | Interventional | 2006-10-01 | Completed |
TAILOR Study: Tacrolimus Versus Mycophenolate for Autolmmune Hepatitis Patients With incompLete Response On First Line Therapy: a Randomized Trial[NCT05221411] | Phase 4 | 86 participants (Anticipated) | Interventional | 2022-01-19 | Recruiting |
A Crossover, Randomized and Multi-center Study to Evaluate the Efficacy and Safety of Abatacept Versus Mycophenolate Mofetil (MMF) in Treatment of PV[NCT05303272] | Phase 4 | 60 participants (Anticipated) | Interventional | 2021-02-01 | Recruiting |
High-dose Post-transplantation Cyclophosphamide as Graft Versus-host Disease Prophylaxis After Allogeneic Hematopoietic Stem Cell Transplantation[NCT02294552] | Phase 2 | 200 participants (Actual) | Interventional | 2014-10-31 | Completed |
MyOra® (Mycophenolate Mofetil) Post-Authorization Safety Study for Prophylaxis in de Novo Renal Transplant Patients[NCT03517982] | | 12 participants (Actual) | Observational | 2014-10-31 | Completed |
Intracoronary Analysis of Cardiac Allograft Vasculopathy in Comparison to Coronary Artery Disease by Means of Optical Coherence Tomography[NCT02254668] | Phase 4 | 278 participants (Anticipated) | Interventional | 2013-12-31 | Recruiting |
An Observational Study of Mycophenolate Mofetil Combined With Glucocorticoid in the Treatment of Relapse Vogt-Koyanagi-Harada Disease[NCT05627739] | | 15 participants (Anticipated) | Observational | 2021-10-01 | Recruiting |
A Phase II Study of IL-6 Receptor Blockade to Ameliorate Acute Graft Versus Host Disease and Early Toxicity After Double Unit Cord Blood Transplantation in Adults With Hematologic Malignancies.[NCT03434730] | Phase 2 | 46 participants (Actual) | Interventional | 2018-02-07 | Active, not recruiting |
A Phase 2, Randomized, Double-blind, Placebo-controlled Evaluation of the Safety and Efficacy of BMS-986165 With Background Treatment in Subjects With Lupus Nephritis[NCT03943147] | Phase 2 | 16 participants (Actual) | Interventional | 2019-07-15 | Terminated(stopped due to Insufficient enrollment) |
Phase 3 Study of Treatment of IgAN With Multi-glycoside of Tripterygium Wilfordii HOOK. f[NCT02187900] | Phase 3 | 300 participants (Anticipated) | Interventional | 2014-06-30 | Recruiting |
Radiation- and Alkylator-free Hematopoietic Cell Transplantation for Bone Marrow Failure Due to Dyskeratosis Congenita / Telomere Disease[NCT01659606] | Phase 2 | 40 participants (Anticipated) | Interventional | 2012-07-31 | Active, not recruiting |
Ruxolitinib With Calcineurin and Methotrexate vs. Calcineurin Plus Methotrexate and Mycophenolate Mofetil as Graft Versus Host Disease Prophylaxis for HLA-haploidentical Hematopoietic Stem Cell Transplantation[NCT04838704] | Phase 4 | 206 participants (Anticipated) | Interventional | 2021-04-08 | Recruiting |
Reduce Intensity Conditioning (RIC) Allogenic Hematopoietic Stem Cell Transplantation (Allo HSCT) for Patients With Relapsed Multiple Myeloma: A Pilot Study[NCT04205240] | Phase 2 | 1 participants (Actual) | Interventional | 2020-12-22 | Terminated(stopped due to Poor accrual) |
A Single-Dose, 2-Period, 2-Treatment, 2-Way Crossover Bioequivalence Study of Mycophenolate Mofetil 250 mg Capsules Under Fasting Conditions[NCT00911274] | Phase 1 | 53 participants (Actual) | Interventional | 2006-10-31 | Completed |
A Pilot Randomized Controlled Trial of De Novo Belatacept-Based Immunosuppression in Lung Transplantation[NCT03388008] | Phase 2 | 27 participants (Actual) | Interventional | 2019-12-17 | Completed |
12-month Open Label, Randomized, Multicenter Study Evaluating Efficacy, Safety and Tolerability of Oral AEB071 Plus Tacrolimus (Converted to Mycophenolic Acid After 3 Months), vs. Mycophenolic Acid Plus Tacrolimus in de Novo Renal Transplant Recipients[NCT00403416] | Phase 1/Phase 2 | 215 participants (Actual) | Interventional | 2006-10-31 | Completed |
Evaluation of Acute Rejection Rates in de Novo Renal Transplant Recipients Following Thymoglobulin Induction, CNI-free, Nulojix (Belatacept)-Based Immunosuppression[NCT02137239] | Phase 2 | 58 participants (Actual) | Interventional | 2015-12-31 | Completed |
Clinical and Laboratory Evaluation of Acute Rejection, Myocyte Growth, Repair, and Oxidative Stress Following de Novo Cardiac Transplant: A Comparison Between Tacrolimus- and Cyclosporine- Based Immunoprophylactic Regimens With MPA TDM[NCT00157014] | Phase 3 | 111 participants (Actual) | Interventional | 2004-05-10 | Completed |
A Phase IV, Single Center Pilot Study Using Alemtuzumab (Campath-1H) Induction Combined With Prednisone-Free, Calcineurin-Inhibitor-Free Immunosuppression in Kidney Transplantation[NCT00166712] | Phase 4 | 40 participants (Actual) | Interventional | 2005-04-30 | Terminated(stopped due to Study stopped due to lack of efficacy & funding.) |
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a CNI-free Regimen With Enteric-Coated Mycophenolate Sodium (EC-MPS) and Everolimus in Comparison to Standard Therapy With Enteric-Coated Mycophenolate Sodium (EC-MPS) a[NCT00154310] | Phase 4 | 300 participants (Actual) | Interventional | 2005-06-30 | Completed |
Intra-osseous Co-transplant of Cord Blood and Mesenchymal Stromal Cells: A Feasibility Study[NCT02181478] | Early Phase 1 | 6 participants (Actual) | Interventional | 2015-07-22 | Completed |
Myfortic (Enteric-coated Mycophenolate Sodium) for the Treatment of Non-infectious Intermediate Uveitis - a Prospective, Controlled, Randomized Multicenter Trial[NCT01092533] | Phase 3 | 44 participants (Actual) | Interventional | 2010-03-31 | Completed |
Phase II/III, Open-Label, Randomized, Controlled, Multiple-Dose Study of Efficacy and Safety of BMS-224818 (Belatacept) as Part of a Quadruple Drug Regimen in First Renal Transplant Recipients[NCT00035555] | Phase 2 | 230 participants (Actual) | Interventional | 2001-03-31 | Completed |
A Single-Dose, Replicate, Comparative Bioavailability Study of Two Formulations of Mycophenolate Mofetil 500 mg Tablets Under Fed Conditions[NCT00908128] | Phase 1 | 40 participants (Actual) | Interventional | 2006-08-31 | Completed |
Randomized Controlled Trial to Evaluate the Efficacy of Enteric-coated Mycophenolate Sodium Versus Azathioprine for the Induction and Maintenance of Remission of the Extra-renal Lupus Manifestations[NCT01112215] | Phase 4 | 240 participants (Actual) | Interventional | 2009-12-31 | Completed |
A 12-Month, Randomized, Controlled, Open-Label, Dose Escalation Study Evaluating Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of an Anti-CD2 Monoclonal Antibody, TCD601(Siplizumab) Compared to Anti-thymocyte Globulin (rATG), as In[NCT04311632] | Phase 2 | 24 participants (Anticipated) | Interventional | 2021-05-26 | Recruiting |
Matched Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplantation With a Conditioning Regimen of Targeted Busulfan, Cyclophosphamide, and Thymoglobulin[NCT00611351] | Phase 2 | 5 participants (Actual) | Interventional | 2005-06-07 | Completed |
Allogeneic Peripheral Blood Stem Cell Transplantation With Minimally Myelosuppressive Regimen of Pentostatin and Low-dose Total-body Irradiation[NCT00571662] | Phase 2 | 76 participants (Actual) | Interventional | 2000-12-08 | Completed |
Booster Effects With Autoimmune Treatments in Patients With Poor Response to Initial COVID-19 Vaccine (ACV01)[NCT05000216] | Phase 2 | 257 participants (Actual) | Interventional | 2021-08-13 | Active, not recruiting |
Efficacy of Mycophenolate Mofetil Versus Leflunomide as Maintenance Treatment for IgG4-RD Patients With Internal Organ Involvement[NCT05789017] | | 60 participants (Anticipated) | Interventional | 2022-07-01 | Recruiting |
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 3 | 435 participants (Anticipated) | Interventional | 2023-03-15 | Recruiting |
Partially HLA-Mismatched Related Donor Hematopoietic Stem Cell Transplantation Using Killer Immunoglobulin Receptor and Human Leukocyte Antigen Based Donor Selection[NCT02880293] | | 44 participants (Actual) | Interventional | 2016-08-23 | Completed |
A Randomized Open-label Study of Fixed-dose Versus Concentration-controlled Mycophenolate Mofetil for the Treatment of Active Lupus Nephritis[NCT03920059] | Phase 4 | 2 participants (Actual) | Interventional | 2019-08-20 | Terminated(stopped due to Slow recruitment rate) |
Sirolimus-based Immunosuppression Treatment Regimen for Liver Transplantation: A Multicenter, Open-label, Randomized, Controlled Clinical Trial in Liver Transplant Recipients With Hepatocellular Carcinoma[NCT03500848] | Phase 2/Phase 3 | 130 participants (Anticipated) | Interventional | 2018-05-01 | Not yet recruiting |
Extended Follow-Up After Islet Transplantation in Type 1 Diabetes (CIT-08)[NCT01369082] | | 75 participants (Actual) | Observational | 2011-05-31 | Completed |
Mycophenolate Mofetil and Prednisolone Versus Prednisolone Alone in Fibrotic Hypersensitivity Pneumonitis: a Randomized Controlled Trial[NCT05626387] | Phase 4 | 144 participants (Anticipated) | Interventional | 2022-11-23 | Recruiting |
Low-dose Combination of Mycophenolate Mofetil and Tacrolimus for Refractory Lupus Nephritis: a 12-month Prospective Study[NCT01203709] | Phase 4 | 20 participants (Actual) | Interventional | 2010-08-31 | Completed |
Multicenter Registry of Pediatric Lupus Nephritis in China[NCT03791827] | | 1,200 participants (Anticipated) | Observational | 2018-12-01 | Recruiting |
JAK Inhibitor Prior to Allogeneic Stem Cell Transplant for Patients With Primary and Secondary Myelofibrosis: A Prospective Study[NCT02251821] | Phase 2 | 99 participants (Actual) | Interventional | 2014-10-20 | Active, not recruiting |
A Single-center,Randomized,Open-label,12 Months Study,2 Parallel Group to Compare the Efficacy of Everolimus Combination + Tacrolimus in Regression of Left Ventricular Hypertrophy vs Tacrolimus + MMF in Renal Transplant Patients[NCT03415750] | Phase 4 | 20 participants (Actual) | Interventional | 2016-11-30 | Completed |
Adoptive Immunotherapy in Patients With Relapsed Hematological Malignancy: Effect of Duration and Intensity of Early GVHD Prophylaxis on Long-Term Clinical Outcomes[NCT02593123] | Phase 2 | 31 participants (Actual) | Interventional | 2015-11-04 | Completed |
Steroid Free Immunosuppression or Calcineurin Inhibitor Minimization After Basiliximab Induction Therapy in Kidney Transplantation: Comparison With a Standard Quadruple Immunosuppressive Regimen[NCT01560572] | Phase 4 | 305 participants (Actual) | Interventional | 2011-04-30 | Completed |
A Two Part, Phase 1/2, Safety, PK and PD Study of TOL101, an Anti-TCR Monoclonal Antibody for Prophylaxis of Acute Organ Rejection in Patients Receiving Renal Transplantation[NCT01154387] | Phase 1/Phase 2 | 85 participants (Anticipated) | Interventional | 2010-07-31 | Active, not recruiting |
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study of Mycophenolate Mofetil 500 mg Tablet of Dr. Reddy's Laboratories Limited Comparing With That of Cellcept 500 mg Tablet of Roche La[NCT01283867] | Phase 1 | 92 participants (Actual) | Interventional | 2007-11-30 | Completed |
A Randomized Controlled Multi-center Trial of Mycophenolate Mofetil for the Patient With High Risk Membranous Nephropathy[NCT01282073] | Phase 3 | 62 participants (Anticipated) | Interventional | 2011-03-31 | Recruiting |
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) | Interventional | 2016-03-10 | Completed |
Weaning of Immunosuppression in Nephritis of Lupus[NCT01284725] | Phase 3 | 100 participants (Actual) | Interventional | 2011-01-31 | Active, not recruiting |
Myfortic® Combined With Low-dose Steroid in Minimal Change Nephrotic Syndrome[NCT01185197] | Phase 4 | 20 participants (Actual) | Interventional | 2010-09-30 | Completed |
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 2 | 64 participants (Anticipated) | Interventional | 2022-08-31 | Not yet recruiting |
Budesonide in Liver Transplantation[NCT03315052] | Phase 4 | 0 participants (Actual) | Interventional | 2019-01-31 | Withdrawn(stopped due to Delay in IRB approval) |
A Study Evaluating Escalating Doses of 90Y-DOTA-BC8 (Anti-CD45) Antibody Followed by Allogeneic Stem Cell Transplantation for High-Risk Acute Myeloid Leukemia (AML) Acute Lymphoblastic Leukemia (ALL), or Myelodysplastic Syndrome (MDS)[NCT01300572] | Phase 1 | 16 participants (Actual) | Interventional | 2012-01-31 | Completed |
Relationships Between Pharmacokinetic and Pharmacodynamic Strategies for Assessment of the Risks for Acute Rejection and Side Effects of Mycophenolate Mofetil[NCT01292226] | Phase 2 | 45 participants (Actual) | Interventional | 2006-12-31 | Completed |
Pharmacokinetics of Mycophenolate Mofetil in de Novo Lung Allograft Recipients[NCT01014442] | Phase 3 | 68 participants (Actual) | Interventional | 2010-01-31 | Completed |
Alloantibodies in Pediatric Heart Transplantation[NCT01005316] | | 290 participants (Actual) | Observational | 2010-01-31 | Terminated(stopped due to Inability to meet accrual goals within the funding period.) |
Early vs. Delayed EVERolimus in de Novo HEART Transplant Recipients: Optimization of the Safety/Efficacy Profile (EVERHEART Study)[NCT01017029] | Phase 4 | 182 participants (Actual) | Interventional | 2009-09-30 | Completed |
Open-Label, Randomized, Multicenter, Parallel-Group Efficacy and Safety Study of Tacrolimus Immunosuppressive Therapy After Kidney Transplantation[NCT00717379] | Phase 4 | 50 participants (Actual) | Interventional | 2007-05-31 | Completed |
A Randomized, Open Label Study Comparing the Effect of CellCept Combined With Low Dose Versus Standard Dose Tacrolimus, and Corticosteroids, on Kidney Function in Renal Transplantation Patients[NCT00758602] | Phase 4 | 210 participants (Actual) | Interventional | 2008-09-30 | Completed |
Phase IV, Open-Label, Multicenter, Randomized Study Comparing Mycophenolate Mofetil (MMF) Dose Adjustment Based on Blood MPA Concentration to Standard Care Treatment With MMF in Renal Transplant Recipients Receiving Tacrolimus[NCT00737659] | Phase 4 | 138 participants (Anticipated) | Interventional | 2008-08-31 | Recruiting |
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 2 | 50 participants (Anticipated) | Interventional | 2008-01-31 | Recruiting |
A Two Step Approach to Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies[NCT02566304] | Phase 2 | 35 participants (Anticipated) | Interventional | 2015-11-13 | Active, not recruiting |
The Efficacy and Safety of Bone Marrow-derived Mesenchymal Stem Cells in Kidney Transplantation From Chinese Donation After Citizen Death (DCD): A Multi-center Randomized Controlled Trial[NCT02561767] | Phase 1/Phase 2 | 120 participants (Anticipated) | Interventional | 2015-10-31 | Not yet recruiting |
Phase II Pilot Cohort Study to Investigate the Safety and Efficacy of Infliximab as Additional Therapy in the Treatment if Anti-Neutrophil Cytoplasm Antibody Associated Vasculitis[NCT00753103] | Phase 2 | 37 participants (Actual) | Interventional | 2003-01-31 | Completed |
Patient Reported Outcomes in Renal Transplant Patients Tolerating GI Symptoms Converted to Myfortic (EC-MPS).[NCT00676221] | Phase 4 | 110 participants (Anticipated) | Interventional | 2006-07-31 | Active, not recruiting |
A Multicenter, Randomized, Double Blind, Double Dummy Controlled Study to Assess the Tolerability of an Increased Dose of Enteric Coated MPA After Conversion From MMF in Renal Transplant Recipients Who Required MMF Dose Reductions Due to Gastrointestinal [NCT00658333] | Phase 4 | 30 participants (Actual) | Interventional | 2008-03-31 | Terminated |
A Randomised, Multicenter, Open-label Study Evaluating the Impact on the Fibrosis at Week 52 of an Early Biopsy at (D10) Versus Standard Management in Patients Who Have Undergone de Novo Renal Transplantation and Received a Marginal Organ Transplant[NCT00817687] | Phase 4 | 66 participants (Actual) | Interventional | 2009-01-31 | Completed |
A Randomised, Double-Blind, Placebo Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of Two Doses of Ocrelizumab in Patients With WHO or ISN Class III or IV Nephritis Due to Systemic Lupus Erythematosus[NCT00626197] | Phase 3 | 381 participants (Actual) | Interventional | 2008-02-15 | Terminated(stopped due to Study was terminated due to an imbalance of serious and opportunistic infections in the ocrelizumab treated patients versus the placebo arm.) |
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies-Increasing GVT Effects Without Increasing Toxicity[NCT03032783] | Phase 2 | 63 participants (Anticipated) | Interventional | 2017-01-31 | Recruiting |
Reduced Intensity Stem Cell Transplantation (RIST) for Patients With Hematological Malignancies Conditioned With Fludarabine and Busulfan[NCT00619645] | Phase 2 | 8 participants (Actual) | Interventional | 2007-06-30 | Completed |
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 2 | 0 participants (Actual) | Interventional | 2008-09-30 | Withdrawn(stopped due to lack of accrual) |
Post-Transplant Bendamustine (PT-BEN) for GVHD Prophylaxis[NCT04022239] | Phase 1/Phase 2 | 40 participants (Anticipated) | Interventional | 2020-03-13 | Recruiting |
Very Low-dose Total Body Irradiation in Combination With Total Lymphoid Irradiation and Anti-Thymocyte Globulin to Improve Donor Engraftment in Patients Undergoing Non-Myeloablative Hematopoietic Cell Transplantation[NCT03734601] | Phase 2 | 22 participants (Actual) | Interventional | 2018-11-05 | Completed |
A Phase 1/2a, Open-Label, Multicenter, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravenous Administration of RGI-2001 in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (AHSCT)[NCT01379209] | Phase 1/Phase 2 | 68 participants (Actual) | Interventional | 2011-09-30 | Completed |
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 2 | 15 participants (Actual) | Interventional | 2010-08-31 | Completed |
Islet Transplantation in Type I Diabetes With LEA29Y (Belatacept) Maintenance Therapy (CIT-04)[NCT00468403] | Phase 2 | 10 participants (Actual) | Interventional | 2008-10-31 | Completed |
Tacrolimus and Mycophenolate Mofetil as Post-Grafting Immunosuppression After Conditioning With Fludarabine and Low-Dose Total Body Irradiation for Recipients of HLA-Matched or Mis-Matched Family or Unrelated Donor HCT[NCT02178683] | Phase 3 | 50 participants (Anticipated) | Interventional | 2010-11-30 | Recruiting |
Multi-center, Open-label, Randomized Controlled Phase 4 Study to Evaluate the Efficacy and Safety of CertiroBell® Compared With Mycophenolate Mofetil in Primary Living Donor Liver Transplant Recipients.[NCT04471441] | Phase 4 | 150 participants (Anticipated) | Interventional | 2020-06-30 | Recruiting |
A Multicenter, Randomized, Double-blind, Placebo-controlled TRial Evaluating Immunosuppressive Treatment in Patients With Chronic Virus-Negative Inflammatory cardiomyopaThY (TRINITY Trial)[NCT05570409] | Phase 2/Phase 3 | 130 participants (Anticipated) | Interventional | 2023-03-28 | Recruiting |
Allogeneic Stem Cell Transplantation With Alternative Donor in Treatment of Hematologic Malignancy[NCT02487069] | | 876 participants (Actual) | Interventional | 2015-06-30 | Completed |
A Phase II Study of Cytokine Induced Memory-like NK Cell Adoptive Therapy After Haploidentical Donor Hematopoietic Cell Transplantation[NCT02782546] | Phase 2 | 60 participants (Anticipated) | Interventional | 2017-01-30 | Recruiting |
A Prospective, Multi-center, Single-arm, Interventional Study of Thymoglobuline® Induction Therapy in Adult Recipients of Donated After Cardiac Death Kidney Transplant in China[NCT03099122] | Phase 4 | 115 participants (Actual) | Interventional | 2017-08-16 | Completed |
Belatacept for the Management of Moderately Sensitized Patients at Risk for Delayed Graft Function (DGF)[NCT02130817] | Phase 4 | 0 participants (Actual) | Interventional | 2014-09-24 | Withdrawn(stopped due to Organ transplant criteria made recruitment difficult. Closed with IRB 10/09/2015.) |
A Prospective, Randomized, Controlled Pilot Study of Early-Use Long Acting Tacrolimus (Envarsus XR) in Lung Transplant Recipients[NCT04469842] | Early Phase 1 | 48 participants (Anticipated) | Interventional | 2023-08-31 | Not yet recruiting |
Comparisons Of Inflammatory Biomarkers And Cardiovascular Risk Scores Before And After Conversion To Full Dose Myfortic® Using Two Hour Neoral® Monitoring.[NCT02058875] | Phase 4 | 0 participants (Actual) | Interventional | 2014-02-28 | Withdrawn(stopped due to The study funder retracted their grant funding offer before contract signed.) |
A Randomised Clinical Trial Assessing the Efficacy and Safety of Mycophenolate Mofetil Versus Azathioprine for Induction of Remission in Treatment Primary Biliary Cholangitis-Autoimmune Hepatitis Overlap Syndrome[NCT04933292] | Phase 4 | 78 participants (Anticipated) | Interventional | 2021-06-16 | Recruiting |
Allogeneic Stem Cell Transplantation for Patients With Multiple Myeloma: a Pilot Feasibility Study Using a Novel Protocol[NCT02447055] | Early Phase 1 | 0 participants (Actual) | Interventional | 2015-12-31 | Withdrawn |
Comparison of Safety and Efficacy of de Novo Everolimus Plus Low Dose of Cyclosporine With Standard Dose of Cyclosporine Plus Cellcept on CMV and BK Virus Infections Prevention in Renal Transplant Patients[NCT04906304] | | 35 participants (Actual) | Interventional | 2020-01-01 | Completed |
Prospective, Multicenter, Randomized, Open-label, Phase 2, Lasting 12 Weeks, Evaluating the Pharmacodynamics, Efficacy and Safety of Basiliximab in de Novo Adult Renal Transplant Patients at Low Risk Receiving Either a Cumulative Dose of Basiliximab of 40[NCT01596062] | Phase 2 | 16 participants (Actual) | Interventional | 2012-03-31 | Completed |
Mycophenolate Sodium (Myfortic®) in the Treatment of Corticosteroid-refractory Autoimmune Uveitis:Pilot Study[NCT01261169] | | 40 participants (Anticipated) | Observational | 2009-01-31 | Recruiting |
Treg Adoptive Therapy in Subclinical Inflammation in Kidney Transplantation (CTOT-21)[NCT02711826] | Phase 1/Phase 2 | 14 participants (Anticipated) | Interventional | 2016-09-20 | Completed |
Scleroderma Lung Study III (SLS III): Combining the Anti-fibrotic Effects of Pirfenidone (PFD) With Mycophenolate (MMF) for Treating Scleroderma-related Interstitial Lung Disease[NCT03221257] | Phase 2 | 51 participants (Actual) | Interventional | 2017-11-28 | Completed |
Maintaining or Stopping Immunosuppressive Therapy in Patients With ANCA Vasculitis and End-stage Renal Disease: a Prospective, Multicenter, Randomized, Open-label, Clinical Trial[NCT03323476] | Phase 3 | 136 participants (Anticipated) | Interventional | 2018-02-02 | Recruiting |
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major[NCT03171831] | Phase 4 | 30 participants (Anticipated) | Interventional | 2017-04-01 | Recruiting |
Haploidentical Allogeneic Peripheral Blood Transplantation: Clinical Trial and Laboratory Correlates Examining Checkpoint Immune Regulators' Expression[NCT03480360] | Phase 3 | 20 participants (Anticipated) | Interventional | 2018-03-28 | Active, not recruiting |
Multicenter Uveitis Steroid Treatment (MUST) Trial[NCT00132691] | Phase 4 | 255 participants (Actual) | Interventional | 2005-09-30 | Completed |
Impact of Cyclosporine or Steroid Withdrawal at 3 Months Post Transplantation on Graft Function, Patient Survival and Cardiovascular Surrogate Markers the First 5 Years After Renal Transplantation.[NCT00903188] | Phase 4 | 152 participants (Anticipated) | Interventional | 2008-10-31 | Recruiting |
Phase 4, Randomized, Open-label, Comparative, Multicenter Study to Assess the Safety and Efficacy of Induction Agents, Alemtuzumab, Basiliximab or Rabbit Anti-thymocyte Globulin in Combination With Tacrolimus, MMF, and a Rapid Steroid Withdrawal in Renal [NCT00113269] | Phase 4 | 501 participants (Actual) | Interventional | 2005-05-31 | Completed |
Phase-I/II Trial to Assess the Safety and Efficacy of Venetoclax in Addition to Sequential Conditioning With Fludarabine / Amsacrine / Ara-C (FLAMSA) + Treosulfan for Allogeneic Blood Stem Cell Transplantation in Patients With MDS, CMML or sAML (FLAMSACla[NCT05807932] | Phase 1/Phase 2 | 38 participants (Anticipated) | Interventional | 2023-06-26 | Recruiting |
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 2 | 320 participants (Anticipated) | Interventional | 2021-09-23 | Recruiting |
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 2 | 73 participants (Actual) | Interventional | 2007-12-14 | Completed |
Controlled, Randomized, Parallel Group Study to Assess Efficacy and Tolerability of Full Dose Enteric-coated Mycophenolate Sodium, in Addition to Cyclosporine for Microemulsion Reduced Dose, in Maintenance Renal Transplant Recipients[NCT00434590] | Phase 4 | 10 participants (Actual) | Interventional | 2007-03-31 | Terminated(stopped due to The study has been stopped because of the lack of enrollment) |
A Phase II Multicenter Trial to Assess the Safety and Efficacy of Campath-1H and Tacrolimus Followed By Immunosuppression Withdrawal in Liver Transplantation (ITN024ST)[NCT00105235] | Phase 2 | 27 participants (Actual) | Interventional | 2005-06-30 | Completed |
A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Ex-Vivo Cultured Allogenic Mesenchymal Stem Cells For the Treatment of Extensive Chronic Graft Versus Host Disease[NCT00972660] | Phase 2 | 52 participants (Anticipated) | Interventional | 2009-09-30 | Enrolling by invitation |
A Prospective Multicenter Open-label Randomized Study to Assess Efficacy and Safety of Everolimus in Combination With Cyclosporine Microemulsion Versus Everolimus Without Calcineurine Inhibitor in Combination With Enteric-coated Mycophenolate Sodium (EC-M[NCT00425308] | Phase 3 | 30 participants (Actual) | Interventional | 2006-10-31 | Completed |
A Multicenter Randomized Open Label Study to Assess Efficacy and Safety of a Steroid Avoidance Regimen in Comparison to a Treatment With Steroids, in Combination With Enteric-coated Mycophenolate Sodium (EC-MPS) 2.16 g/d for 6 Weeks and Cyclosporine Micro[NCT00413920] | Phase 3 | 222 participants (Actual) | Interventional | 2007-04-30 | Completed |
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Liver Transplant Recipients (MyLiver)[NCT00405652] | Phase 3 | 34 participants (Actual) | Interventional | 2007-01-31 | Completed |
A Randomized, Multicenter, Parallel-group, Open-label Study to Evaluate the Therapeutic Benefit of an Initially Intensified Dosing Regimen of Mycophenolate Sodium Versus a Standard Dosing Regimen, in Combination With Cyclosporine and Corticosteroids in de[NCT00419926] | Phase 4 | 313 participants (Actual) | Interventional | 2006-12-31 | Completed |
A Randomised, Double-Blind, Placebo Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of Two Doses of Ocrelizumab in Patients With Active Systemic Lupus Erythematosus[NCT00539838] | Phase 3 | 33 participants (Actual) | Interventional | 2007-12-19 | Terminated(stopped due to The study was terminated prematurely when the decision was made that ocrelizumab was not likely to benefit this patient population.) |
A Randomized, Open Label Study Comparing the Effect of CellCept With Therapeutic Drug Monitoring, Tacrolimus and a Corticosteroid-sparing Regimen Versus Fixed Dose CellCept, Tacrolimus and Corticosteroids Maintained up to 6 Months, on Acute Rejection and [NCT00545402] | Phase 4 | 180 participants (Actual) | Interventional | 2007-11-30 | Completed |
Efficacy and Safety of Anti-cytomegalovirus Prophylaxis Versus Pre-emptive Approaches With Valganciclovir in Heart Transplant Recipients Treated With Everolimus or Mycophenolate. A Randomized Open-label Study for Prevention of Cardiaca Allograft Vasculopa[NCT00966836] | Phase 3 | 100 participants (Anticipated) | Interventional | 2009-04-30 | Recruiting |
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 3 | 224 participants (Actual) | Interventional | 2006-12-31 | Completed |
Phase I Feasibility Study of Clofarabine and Low Dose Total Body Irradiation (TBI) as a Non-myeloablative Preparative Regimen for Stem Cell Transplantation (SCT) for Hematologic Malignancies[NCT01041508] | Phase 1 | 36 participants (Anticipated) | Interventional | 2010-02-28 | Completed |
Open Randomized Multi-Center Study to Evaluate Safety and Efficacy of Low Molecular Weight Sulfated Dextran (LMW-SD) in Islet Transplantation After Kidney Transplantation (CIT-01B)[NCT00790439] | Phase 2 | 0 participants (Actual) | Interventional | 2008-07-31 | Withdrawn(stopped due to Due to funding limitations) |
Randomized, Double-blind, Double-dummy Trial of Mycophenolic Acid Versus Azathioprine in the Treatment of Corticosteroid-refractory Myasthenia Gravis[NCT00997412] | | 40 participants (Anticipated) | Interventional | 2009-05-31 | Active, not recruiting |
Phase I Study of CellCept for Advanced Pancreatic Cancer[NCT00997958] | Phase 1 | 12 participants (Actual) | Interventional | 2004-06-30 | Completed |
Pilot, Single-arm, Non-comparative, Open-label Study of Daclizumab in Combination With Mycophenolate Mofetil and Sirolimus in the Prevention of Acute Rejection in Cardiac Allografts Recipients in Risk of Deteriorated Renal Function[NCT02554955] | Phase 4 | 36 participants (Actual) | Interventional | 2004-02-29 | Completed |
A Randomized, Open-label Study of the Achievement of a Mycophenolic Acid Therapeutic Window During Treatment With 2 Dosing Regimens of Oral CellCept Administered as a Component of Standard Immunosuppressive Therapy in Patients With Kidney Transplants[NCT00788567] | Phase 3 | 136 participants (Actual) | Interventional | 2005-06-30 | Completed |
Open, Single Center, Randomised, Parallel Group Pilot Study to Investigate a Calcineurin Free Immunosuppressive Treatment for de Novo Renal Transplant Recipients: A Comparison of a Rapamycin/MMF/Steroids Regime to a Cyclosporine A Neoral/MMF/Steroids Regi[NCT00812123] | Phase 4 | 127 participants (Actual) | Interventional | 2001-01-31 | Completed |
A Randomized, Open-Label, Multicenter, Parallel-Group Study of Belatacept (BMS-224818)-Based Corticosteroid-Free Regimens in Renal Transplant[NCT00455013] | Phase 2 | 93 participants (Actual) | Interventional | 2007-07-31 | Completed |
Nordic Everolimus (Certican) Trial in Heart and Lung Transplantation: Results at 24 Months[NCT00377962] | Phase 4 | 282 participants (Actual) | Interventional | 2005-12-31 | Completed |
A Prospective, Randomised, Double-blind, Placebo-controlled Trial Evaluating the Effects of Mycophenolate Mofetil on 'Surrogate Markers' for Atherosclerosis in Female Patients With SLE.[NCT01101802] | Phase 4 | 71 participants (Actual) | Interventional | 2006-03-31 | Completed |
Comparative Clinical Study of Reduced Dose of Tacrolimus and Standard Dose of Mycophenolate Mofetil vs. Conventional Dose of Tacrolimus and Reduced Dose of Mycophenolate Mofetil in De Novo Renal Transplant Recipients[NCT03968588] | Phase 4 | 108 participants (Actual) | Interventional | 2014-07-29 | Completed |
Pilot Study Using Donor Stem Cells and Campath-1H to Induce Renal Transplant Tolerance (ITN022ST)[NCT00183248] | Phase 1/Phase 2 | 9 participants (Actual) | Interventional | 2004-09-30 | Completed |
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 3 | 87 participants (Actual) | Interventional | 2003-10-31 | Completed |
A Multicenter Pilot Study to Determine the Pharmacokinetics of Astagraf XL, Prograf and Mycophenolate Mofetil in Renal Transplant Candidates Who Have Undergone Laparoscopic Sleeve Gastrectomy[NCT02221583] | Phase 4 | 26 participants (Actual) | Interventional | 2014-05-31 | Completed |
Open-label Prospective Randomized Study to Determine the Efficacy and Safety of Two Dosing Regimens of ACTHar in the Treatment of Proliferative Lupus Nephritis.[NCT02226341] | Phase 4 | 20 participants (Anticipated) | Interventional | 2014-10-31 | Recruiting |
A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Atacicept in Subjects With Lupus Nephritis in Combination With Mycophenolate Mofetil Therapy.[NCT00573157] | Phase 2/Phase 3 | 6 participants (Actual) | Interventional | 2007-12-31 | Terminated(stopped due to The study was terminated due to unanticipated safety issues) |
Prospective Donor-specific Cellular Alloresponse Assessment for Immunosuppression Minimization in de Novo Renal Transplantation[NCT02540395] | | 184 participants (Actual) | Interventional | 2015-03-31 | Completed |
Targeting Inflammation and Alloimmunity in Heart Transplant Recipients With Tocilizumab (RTB-004)[NCT03644667] | Phase 2 | 200 participants (Anticipated) | Interventional | 2018-12-20 | Recruiting |
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen[NCT02776202] | Phase 2 | 15 participants (Anticipated) | Interventional | 2016-05-31 | Recruiting |
Minimizing Glucocorticoid Administration in Patients With Proliferative Lupus Nephritis During the Induction of Remission Period-EUROLUPUS vs. RITUXILUP Regimen: A Randomized Study[NCT05207358] | Phase 4 | 30 participants (Anticipated) | Interventional | 2022-03-02 | Recruiting |
Phase IV Study of Enteric-coated Mycophenolate Sodium in Combination With Tacrolimus in Renal Transplant Patient[NCT00646737] | Phase 4 | 18 participants (Actual) | Interventional | 2008-05-31 | Completed |
Pilot Safety and Feasibility Trial of Mycophenolate and Sirolimus for Prevention of GVHD in Mismatched Unrelated and Related Donor Hematopoietic Stem Cell Transplantation for Hematologic Malignancies[NCT02728700] | Phase 1 | 1 participants (Actual) | Interventional | 2016-02-29 | Terminated(stopped due to Accrual factor) |
Sequential, Related Donor Partial Liver Transplantation Followed by Bone Marrow Transplantation for Fibrolamellar or Non-fibrolamellar Hepatocellular Carcinoma (HCC) Including Fibrolamellar HCC[NCT02702960] | Phase 2 | 0 participants (Actual) | Interventional | 2016-03-31 | Withdrawn(stopped due to This study was withdrawn due to lack of necessary resources from the liver transplant surgical group.) |
The Study of Immunotherapy With Rituximab and Pulse Dexamethasone Followed by With Mycophenolate Mofetil or Placebo in Adult Patients With Persistent and Chronic Immune Thrombocytopenia[NCT02649504] | Phase 3 | 0 participants (Actual) | Interventional | 2016-12-01 | Withdrawn |
Mycophenolate Mofetil Treatment With Neuromyelitis Optica Spectrum Disorders in Chinese Patients[NCT02809079] | Phase 4 | 100 participants (Anticipated) | Interventional | 2016-01-31 | Enrolling by invitation |
Validation of Population Pharmacokinetic Model Derived From Healthy Volunteer in Kidney Transplant Recipients[NCT02808065] | | 40 participants (Anticipated) | Observational | 2016-05-31 | Recruiting |
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study[NCT04009525] | Phase 4 | 800 participants (Anticipated) | Interventional | 2019-06-01 | Recruiting |
An Open, Multicentre, Randomised, Parallel Group Pilot-Study to Compare Safety and Efficacy of Discontinuation of Mycophenolate Mofetil From a Tacrolimus/MMF/Steroid Triple Regimen Following Kidney Transplantation[NCT00693381] | Phase 3 | 152 participants (Actual) | Interventional | 2003-02-28 | Completed |
A Prospective, Randomized, Multi-Center Double-Blind Study of Early Corticosteroid Cessation vs. Long Term Corticosteroid Therapy With Prograf and CellCept in Primary Renal Transplant Patients[NCT00650468] | Phase 4 | 397 participants (Actual) | Interventional | 1999-11-30 | Completed |
A Randomized Trial for the Treatment of Relapsing Aplastic Anemia With Mycophenolate Mofetil (MMF) and Cyclosporine (CSA)[NCT00005935] | Phase 2 | 130 participants | Interventional | 2000-06-30 | Completed |
A Multi-Institutional Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Malignant Neuro-Epithelial and Other Solid Tumors[NCT02653196] | Early Phase 1 | 1 participants (Actual) | Interventional | 2015-09-30 | Terminated(stopped due to The Principal Investigator left the institution.) |
Identification of Drug-drug Interaction Between Tacrolimus and Mycophenolate Mofetil in Healthy Adults[NCT02743247] | Phase 1 | 18 participants (Actual) | Interventional | 2015-10-31 | Completed |
Haploidentical Hematopoietic Stem Cell Transplantation Using A Novel Clofarabine Containing Conditioning Regimen For Patients With Refractory Hematologic Malignancies[NCT00824135] | Phase 1 | 34 participants (Actual) | Interventional | 2009-01-31 | Completed |
Everolimus and Mycophenolate Sodium as GvHD Prophylaxis in Allogeneic Stem Cell Transplantation[NCT00856505] | Phase 1/Phase 2 | 38 participants (Anticipated) | Interventional | 2008-03-31 | Recruiting |
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Treosulfan Based Preparative Regimen[NCT00796068] | Phase 2 | 130 participants (Actual) | Interventional | 2009-02-24 | Completed |
An Open-label, Single-sequence Study to Investigate the Effects of BMS-986256 at Steady State on the Single Dose Pharmacokinetics of Mycophenolate Mofetil in Healthy Male Participants[NCT04039373] | Phase 1 | 15 participants (Actual) | Interventional | 2019-07-22 | Completed |
Optimized Cord Blood Transplantation for the Treatment of High-Risk Hematologic Malignancies in Adults and Pediatrics[NCT06013423] | Phase 2 | 54 participants (Anticipated) | Interventional | 2024-02-06 | Not yet recruiting |
A Phase II/III Multi-Center, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Abatacept Versus Placebo on a Background of Mycophenolate Mofetil and Glucocorticosteroids in Subjects With Active Proliferative Glomeru[NCT00430677] | Phase 2/Phase 3 | 423 participants (Actual) | Interventional | 2007-06-30 | Terminated(stopped due to Terminated due to failure to meet the primary efficacy endpoint in the Short-term Period) |
Study of the Pharmacokinetics of Mycophenolate Mofetil in Patients Who Have Undergone Orthotopic Liver Transplantation[NCT00007059] | | 20 participants | Interventional | 1998-06-30 | Completed |
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 2 | 16 participants (Actual) | Interventional | 2006-02-28 | Terminated(stopped due to Competing study was started.) |
Multi-center, Open-label, Prospective, Randomized, Parallel Group, Long-term Study Investigating a Standard Regimen in de Novo Kidney Transplant Patients Versus a CNI-free Regimen and a CNI-low Dose Regimen[NCT00514514] | Phase 3 | 802 participants (Actual) | Interventional | 2007-07-31 | Completed |
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf® (Tacrolimus)/ Myfortic® and Advagraf® (Extended Release Tacrolimus) / Myfortic® in de Novo Liver Transplant Recipients[NCT01018914] | Phase 4 | 44 participants (Actual) | Interventional | 2009-04-30 | Completed |
Gastrointestinal Adverse Effect Outcomes of De Novo African American Kidney Transplant Recipients Treated With Tacrolimus, Corticosteroids and Mycophenolate Mofetil or Enteric Coated Mycophenolate Sodium[NCT00522548] | Phase 4 | 37 participants (Actual) | Interventional | 2007-03-31 | Terminated(stopped due to enrollment halted in order to have all patients complete follow-up by Jan 2011) |
A 24-month, Multi-center, Randomized, Open-label, Non-inferiority Study of Efficacy and Safety Comparing Two Exposures of Concentration-controlled Everolimus With Reduced Cyclosporine Versus 3.0 g Mycophenolate Mofetil With Standard Dose Cyclosporine in d[NCT00300274] | Phase 3 | 721 participants (Actual) | Interventional | 2006-01-31 | Completed |
Mycophenolate Mofetil (MMF) ,Carnitine and Phosphodiesterase Type 5 Inhibitor, Three Potential Treatments for Resistant Proteinuria and for Slowing the Deterioration of Diabetic Nephropathy in Patients With Type II Diabetes Mellitus[NCT01566006] | | 80 participants (Anticipated) | Interventional | 2012-04-30 | Not yet recruiting |
Sirolimus, Mycophenolate Mofetil and Bortezomib as Graft-Versus-Host Disease Prophylaxis After Non-Myeloablative Allogeneic Peripheral Blood Stem Cell Transplantation[NCT00548717] | Phase 2 | 15 participants (Actual) | Interventional | 2007-10-31 | Terminated(stopped due to First two patients enrolled after trial reopened, developed grade III-IV acute GVHD and subsequently passed away.) |
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Haematological Diseases Using a Reduced Intensity Conditioning Regimen[NCT00959231] | Phase 2 | 60 participants (Anticipated) | Interventional | 2009-01-31 | Recruiting |
Infusion of Expanded Cord Blood T Cells Following Cord Blood Transplantation[NCT00972101] | Phase 1 | 0 participants (Actual) | Interventional | 2009-09-30 | Withdrawn(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 1 | 17 participants (Actual) | Interventional | 2010-04-21 | Completed |
Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation[NCT00506948] | Phase 2 | 13 participants (Actual) | Interventional | 2006-09-30 | Terminated(stopped due to Halted due to high incidence of veno-oclusive disease of the liver.) |
A Prospective, Randomized, Active Controlled, Parallel Group, Multi-center Trial to Assess the Efficacy and Safety of Mycophenolate Mofetil (MMF) in Inducing Response and Maintaining Remission in Subjects With Lupus Nephritis.[NCT00377637] | Phase 3 | 370 participants (Actual) | Interventional | 2005-07-31 | Completed |
Rheumatoid Arthritis: Tolerance Induction by Mixed Chimerism[NCT00282412] | Phase 1 | 4 participants (Actual) | Interventional | 2002-09-30 | Terminated(stopped due to No participant enrolled for three years. No plan to continue study.) |
A Phase 2, Randomized, Open-Label, Parallel Group, Multi-Center Study to Assess the Safety and Efficacy of Alefacept in de Novo Kidney Transplant Recipients[NCT00543569] | Phase 2 | 323 participants (Actual) | Interventional | 2008-02-29 | Completed |
Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma[NCT00481832] | Phase 2 | 50 participants (Actual) | Interventional | 2007-01-31 | Terminated(stopped due to Accrual Factor) |
Research Institute of Nephrology, Jinling Hospital,[NCT01056237] | | 206 participants (Actual) | Interventional | 2010-02-28 | Completed |
Open Label Balanced Randomized Two-treatment Two-period Two-sequence Single Dose Two-way Crossover Oral Bioequivalence Study of Mycophenolate Mofetil Capsules 250 mg in Normal Healthy Adult Human Subjects Under Fasting Conditions[NCT01080443] | | 56 participants (Actual) | Interventional | 2008-06-30 | Completed |
SIMPLE Study: A Prospective and Randomized Trial of a Simplified Immunosuppressive Protocol Utilizing Low Dose EnvarsusXR[NCT04773392] | Phase 4 | 80 participants (Anticipated) | Interventional | 2021-11-23 | Recruiting |
Combination Therapy Using Mycophenolate Mofetil (CellCept) and Human Interferon beta1a (Rebif) in Early Treatment of Multiple Sclerosis[NCT00618527] | Early Phase 1 | 31 participants (Actual) | Interventional | 2006-08-31 | Completed |
A Two-Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Advanced Cutaneous T Cell Lymphoma[NCT02548468] | Phase 1 | 0 participants (Actual) | Interventional | 2015-11-20 | Withdrawn(stopped due to Slow accrual) |
An Open, Randomized, Multicentre Clinical Study to Compare the Safety and Efficacy of a Combination of Sequential Therapy of Tacrolimus (FK506) With Monoclonal Anti-IL2R Antibodies and Mycophenolate Mofetil Versus Tacrolimus (FK506) With Steroids in Liver[NCT00693524] | Phase 2 | 94 participants (Actual) | Interventional | 2002-11-30 | Completed |
A Multicenter, Three Arm, Randomized, Open Label Clinical Study to Compare Renal Function in Liver Transplant Recipients Receiving an Immunosuppressive Regimen of Advagraf (Immediately or Delayed Post-transplant) and MMF With or Without a Monoclonal Anti-[NCT01011205] | Phase 3 | 893 participants (Actual) | Interventional | 2009-09-30 | Completed |
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 2 | 32 participants (Actual) | Interventional | 2015-07-31 | Terminated(stopped due to Poor recruitment) |
Induction of Mixed Hemopoietic Chimerism in Patients Using Fludarabine, Low Dose TBI, PBSC Infusion and Post Transplant in Immunosuppression w/CSA & Mycophenolate Mofetil[NCT00531635] | Phase 1 | 350 participants (Anticipated) | Interventional | 2002-07-31 | Recruiting |
A Prospective, Multicenter, Randomized Controlled Trial of Mycophenolate Mofetil (MMF) in Patients With IgA Nephropathy (IgAN)[NCT00657059] | Phase 3 | 151 participants (Actual) | Interventional | 2007-09-30 | Completed |
A Prospective, Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multicenter, 36-Week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Maintain or Improve Symptom Control With Reduced Corticosteroid in Subjects[NCT00683969] | Phase 3 | 136 participants (Anticipated) | Interventional | 2004-08-31 | Completed |
The Use of Thymoglobulin in a Calcineurin Inhibitor and Steroid Minimization Protocol[NCT00706680] | Phase 4 | 30 participants (Anticipated) | Interventional | 2008-02-29 | Recruiting |
Prospective Observational Trial to Evaluate Clinical Prognosis and the Risk Factors for Progression for Myasthenia Gravis Patients[NCT04101578] | | 2,000 participants (Anticipated) | Observational | 2017-02-08 | Recruiting |
Pharmacokinetic Profile of Myfortic (Enteric Coated Mycophenolate Sodium) in a Rapid Steroid Withdrawal Protocol in Combination With Tacrolimus in Stable Renal Transplant Recipients in the Fed and Fasting State[NCT00585468] | Phase 4 | 21 participants (Actual) | Interventional | 2007-12-31 | Completed |
Gastrointestinal Mucosal Findings in Patients Receiving Mycophenolic Acid (MPA) as Demonstrated by Small Bowel Capsule Endoscopy (SBCE)[NCT00652834] | Phase 4 | 23 participants (Actual) | Interventional | 2009-04-30 | Completed |
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Hematologic Malignancies[NCT01760655] | Phase 2 | 62 participants (Actual) | Interventional | 2012-12-24 | Completed |
A Multi-center, Randomized, Open-label, Parallel Group Study Investigating the Renal Tolerability, Efficacy and Safety of a CNI-free Regimen (Everolimus and MPA) Versus a CNI-regimen With Everolimus in Heart Transplant Recipients[NCT00862979] | Phase 4 | 162 participants (Actual) | Interventional | 2009-02-24 | Completed |
The Highly Sensitized Patients: Effects of Rituximab and Mycophenolate Mofetil (MMF) On Anti-Human Leukocyte Antigen (HLA) Antibody Levels In Patients Awaiting Cadaveric Renal Transplant.[NCT00446251] | Phase 2 | 14 participants (Actual) | Interventional | 2006-12-31 | Completed |
Reduced Intensity Conditioning Hematopoietic Cell Transplantation for Pediatric Patients With Hematologic Malignancies at High Risk for Transplant Related Mortality With Standard Transplantation[NCT00795132] | Phase 2 | 47 participants (Actual) | Interventional | 2004-04-30 | Completed |
A Randomized, Open Label Study Comparing the Effect of CellCept Combined With 2 Regimens of Reduced Calcineurin Inhibitors on Kidney Function in Liver Transplant Patients[NCT00717314] | Phase 4 | 87 participants (Actual) | Interventional | 2008-05-31 | Completed |
A Phase 2 Study in Poor Risk Diffuse Large B-cell Lymphoma of Total Lymphoid Irradiation & Antithymocyte Globulin Followed by Matched Allogeneic Hematopoietic Transplantation as Consolidation to Autologous Hematopoietic Cell Transplantation[NCT00482053] | Phase 2 | 3 participants (Actual) | Interventional | 2006-10-31 | Terminated(stopped due to Low accrual) |
Sirolimus and Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation[NCT01220297] | Phase 2 | 3 participants (Actual) | Interventional | 2006-08-31 | Terminated(stopped due to Low accrual) |
Three-arm Clinical Trial for Patients With Hematologic Malignancies and Mismatched Donors - Haploidentical, 1 Antigen Mismatch Related or Unrelated, and Matched Unrelated Donor (MUD)- Using a T-cell Replete Allograft and High-dose Post-transplant Cyclopho[NCT01010217] | Phase 2 | 176 participants (Actual) | Interventional | 2009-11-05 | Completed |
Post Transplant Cyclophosphamide for Unrelated and Related Allogeneic Hematopoietic Stem Cell Transplantation for Hematological Malignancies[NCT01349101] | Phase 2 | 80 participants (Actual) | Interventional | 2011-02-10 | Completed |
Optimum Immunosuppression in Renal Transplant Recipients at High Risk of Developing New Onset Diabetes After Transplantation: A Multicenter, Prospective, Controlled and Randomized Trial.[NCT01002339] | Phase 4 | 134 participants (Actual) | Interventional | 2010-02-28 | Terminated(stopped due to Terminated: higher rate of acute rejection in the Cyclosporin A group) |
Low-Dose TBI and Fludarabine Followed by Nonmyeloablative Unrelated Donor Peripheral Blood Stem Cell Transplantation Using Enhanced Postgrafting Immunosuppression for Patients With Hematologic Malignancies and Renal Cell Carcinoma - A Multi-center Trial[NCT00027820] | Phase 1/Phase 2 | 106 participants (Actual) | Interventional | 2001-08-31 | Completed |
A Multicenter, Four Arm, Randomized, Open Label Clinical Study Investigating Optimized Dosing in a Prograf®-/Advagraf®-Based Immunosuppressive Regimen in Kidney Transplant Subjects (OSAKA Study)[NCT00717470] | Phase 4 | 1,252 participants (Actual) | Interventional | 2008-05-14 | Completed |
Mycophenolate Mofetil Maintenance Therapy for Liver Transplantation Following Campath-1H Induction[NCT00849238] | | 0 participants (Actual) | Interventional | | Withdrawn(stopped due to feasibility issues) |
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF and Extended Release (XL) Tacrolimus /MMF and Steroid Withdraw in de Novo Liver Transplant Recipients.[NCT00720408] | Phase 3 | 48 participants (Actual) | Interventional | 2007-12-31 | Completed |
Triple Arm, Prospectively Randomized Multi Centre Study Phase IV to Evaluate Calcineurin Inhibitor Reduced, Steroid Free Immunosuppression After Renal Transplantation in Non-risk Patients[NCT00724022] | Phase 4 | 600 participants (Anticipated) | Interventional | 2008-06-30 | Completed |
Mycophenolate vs. Oral Cyclophosphamide in Scleroderma Interstitial Lung Disease (Scleroderma Lung Study II)[NCT00883129] | Phase 2 | 142 participants (Actual) | Interventional | 2009-09-30 | Completed |
A Phase II/III, Randomized, Open-Label, Active Controlled, Multicenter Trial to Evaluate the Safety and Efficacy of Efalizumab Compared With Cyclosporine, Both in Combination With Mycophenolate Mofetil and Corticosteroids, As an Immunosuppressant Regimen [NCT00729768] | Phase 2/Phase 3 | 0 participants (Actual) | Interventional | | Withdrawn |
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 2 | 12 participants (Anticipated) | Interventional | 2023-11-30 | Recruiting |
A Phase II Multi-center Study of High-Dose Cyclophosphamide and Antithymocyte Globulin Followed by Autologous Hematopoietic Cell Transplantation With Post Transplant Maintenance for the Treatment of Systemic Sclerosis[NCT01413100] | Phase 2 | 21 participants (Actual) | Interventional | 2011-09-15 | Active, not recruiting |
Randomized, Prospective Single-center Study Comparing a Rapid Discontinuation of Corticosteroids (Steroid Withdrawal) With Corticosteroid Therapy in Kidney Transplantation Using Mycophenolate Mofetil and Tacrolimus Maintenance Therapy[NCT00596947] | Phase 4 | 18 participants (Actual) | Interventional | 2005-10-31 | Terminated(stopped due to due to low study enrollment) |
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 250 mg Capsules Under Fasting Conditions[NCT00893542] | | 37 participants (Actual) | Interventional | 2005-11-30 | Completed |
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 500 mg Tablets Under Fasting Conditions[NCT00894088] | | 38 participants (Actual) | Interventional | 2006-01-31 | Completed |
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 2 | 80 participants (Actual) | Interventional | 2016-12-31 | Completed |
Patient Reported Outcomes in Renal Transplant Patients With and Without Gastrointestinal Symptoms[NCT00529269] | Phase 4 | 200 participants (Anticipated) | Interventional | 2006-12-31 | Completed |
A Single Centre, Prospective, Open-Label, Parallel Group, Randomized Study to Compare the Gastrointestinal Tolerability of Mycophenolate Mofetil (MMF, CellCept) and Enteric-Coated Mycophenolate Sodium (EC-MPS, Myfortic) in Maintenance Transplant Patients [NCT00611494] | Phase 4 | 400 participants (Anticipated) | Interventional | 2008-01-31 | Recruiting |
Ph 2, Double-Masked, Randomized, Parallel, Sham Surgery/Placebo Control, Multi-Center Study to Evaluate Systemic IMT Regimens as Graft Rejection Prophylaxis Following Transplantation of hESC Derived RPE Cells in Patients With AMD[NCT02563782] | Phase 2 | 0 participants (Actual) | Interventional | 2015-08-24 | Withdrawn(stopped due to Changes to the study design and the cell line) |
Predictors of Rejection in Pediatric Kidney Transplantation[NCT04292418] | | 70 participants (Anticipated) | Observational | 2020-05-01 | Not yet recruiting |
Intensified Dosing of Cellcept in Kidney Transplantation Trial[NCT00943228] | Phase 4 | 40 participants (Actual) | Interventional | 2010-02-28 | Completed |
A Randomized Single-center Trial of Mycophenolate Mofetil for the Prophylaxis of Graft-versus-host Disease in High Risk Allogeneic Stem Cell Transplantation[NCT00563589] | | 30 participants (Anticipated) | Interventional | 2003-08-31 | Recruiting |
A Phase 2a, Randomized, Open-label, Active Control, Multi-Center Study to Assess the Efficacy and Safety of ASKP1240 in de Novo Kidney Transplant Recipients[NCT01780844] | Phase 2 | 149 participants (Actual) | Interventional | 2013-03-05 | Completed |
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity[NCT04339777] | Phase 2 | 66 participants (Anticipated) | Interventional | 2020-09-22 | Recruiting |
A Therapeutic Exploratory Study to Determine the Efficacy and Safety of Calcineurin-Inhibitor-Free De-novo Immunosuppression After Liver Transplantation[NCT00890253] | Phase 2 | 29 participants (Anticipated) | Interventional | 2010-01-31 | Recruiting |
A Prospective, Randomized, Open Label, Case-Controlled Study on the Efficacy of Mycophenolate Mofetil for IgA Nephropathy Patients With Heavy Proteinuria Despite Angiotensin Blockade[NCT00863252] | Phase 4 | 40 participants (Actual) | Interventional | 2002-03-31 | Completed |
The Clinical Efficacy and Economic Evaluation of EC-MPS (Myfortic) in the Treatment of Relapse or Resistant Proliferative Lupus Nephritis[NCT01015456] | Phase 3 | 59 participants (Actual) | Interventional | 2010-01-31 | Terminated(stopped due to Data Safety Monitoring Board concerning of the participants' safety) |
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 250 mg Capsules Under Fed Conditions[NCT00893919] | | 37 participants (Actual) | Interventional | 2005-11-30 | Completed |
Conversion of CellCept to Myfortic: A Prospective Study on the Tolerability and Safety of Myfortic in Liver Transplant Recipients[NCT00336895] | | 29 participants (Actual) | Interventional | 2006-11-30 | Completed |
Randomized, Multi-Center Comparative Trial of Tacrolimus w/Steroids and Standard Daclizumab Induction vs a Novel Steroid-Free Tacrolimus Based Immunosuppression Protocol w/ Extended Daclizumab Induction in Pediatric Renal Transplantation[NCT00141037] | Phase 1/Phase 2 | 130 participants (Actual) | Interventional | 2004-03-31 | Completed |
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a CNI-free Regimen With Enteric-coated Mycophenolate Sodium and Everolimus in Comparison to Standard Therapy With Enteric-coated Mycophenolate Sodium and Ciclosporin Mic[NCT00332839] | Phase 4 | 93 participants (Actual) | Interventional | 2005-11-30 | Terminated(stopped due to The trial was terminated early due to slow enrollment. It was determined that the planned sample size of 300 could not be achieved.) |
Umbilical Cord Blood (UCB) Allogeneic Stem Cell Transplant for Hematologic Malignancies[NCT01093586] | Phase 2 | 14 participants (Actual) | Interventional | 2007-09-30 | Completed |
Prospective and Randomized Study to Evaluate the Effect of Everolimus in the Clinical and Intra-Cardiac Ecography Progression of Heart Graft Vascular Illness.[NCT00695344] | Phase 4 | 52 participants (Actual) | Interventional | 2006-01-31 | Active, not recruiting |
A Phase 2 Study of Autologous Followed by Nonmyeloablative Allogeneic Transplantation Using Total Lymphoid Irradiation (TLI) and Antithymocyte Globulin (ATG) in Multiple Myeloma Patients[NCT00899847] | Phase 2 | 9 participants (Actual) | Interventional | 2009-05-31 | Completed |
An Open-label Study to Evaluate the Effect of Iguratimod Concomitant With Conventional Immunosuppressive Drugs on Preventing Antibody-induced Rejection in Human Leukocyte Antigen(HLA) Highly Mismatched Kidney Transplant Recipients[NCT02839941] | | 60 participants (Anticipated) | Interventional | 2016-08-31 | Recruiting |
A Comparison of Effects of Standard Dose vs. Low Dose Advagraf® With IL-2 Receptor Antibody Induction, MMF and Steroids, With or Without ACEi/ARB - Based Antihypertensive Therapy on Renal Allograft Histology, Function, and Immune Response[NCT00933231] | Phase 3 | 281 participants (Actual) | Interventional | 2009-08-17 | Completed |
Pilot Study of Unrelated Cord Blood Transplantation in Patients With Poor Risk Haematological Malignancies[NCT00916045] | Phase 2 | 40 participants (Anticipated) | Interventional | 2009-09-30 | Terminated(stopped due to Recruitment issues) |
Mycophenolate Mofetil and Tacrolimus vs Tacrolimus Alone for the Treatment of Idiopathic Membranous Glomerulonephritis (IMG)[NCT00843856] | Phase 4 | 40 participants (Actual) | Interventional | 2009-03-03 | Completed |
A 12-month, Multicenter, Randomized, Open-label Study to Investigate Efficacy and Safety of Concentration Controlled Everolimus With Reduced Dose Cyclosporine A Versus Mycophenolate Mofetil With Standard Dose Cyclosporine A in de Novo Renal Transplant Adu[NCT00658320] | Phase 3 | 122 participants (Actual) | Interventional | 2008-02-29 | Completed |
A Randomized Multicenter Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Mycophenolate Mofetil (CellCept) for the Treatment of Refractory Interstitial Cystitis (IC)[NCT00451867] | Phase 3 | 210 participants (Actual) | Interventional | 2007-03-31 | Terminated(stopped due to The major and primary reason for the study termination is the observed reduced efficacy of CellCept compared to placebo.) |
Comparison of 3g Versus 2g Mycophenolate Mofetil in Combination With Tacrolimus on Progression of Chronic Histology Changes in Kidney Transplant Recipients[NCT01860183] | Phase 4 | 76 participants (Actual) | Interventional | 2013-05-31 | Completed |
A Prospective, Randomized, Open-Label, Pilot Study To Compare The Effect On Carotid Atherosclerosis Of A Tacrolimus-Based Regimen With Conversion From A Tacrolimus- To A Sirolimus-Based Regimen At 3-4 Months Post-Transplant In De Novo Renal Transplant Rec[NCT00311311] | Phase 3 | 72 participants (Actual) | Interventional | 2006-04-30 | Terminated(stopped due to See termination reason in detailed description.) |
Non-Myeloablative Allogeneic Bone Marrow Transplantation for Hematologic Malignancies Using Haploidentical Donors: A Phase I Trial of Pre-Transplant Cyclophosphamide[NCT00006042] | Phase 1 | 0 participants | Interventional | 1999-12-31 | Completed |
A Scandinavian Controlled, Randomized, Open-label, and Multi-centre Study Evaluating if Once-daily Tacrolimus or Twice-daily Cyclosporin, Reduces the 3-year Incidence of Chronic Lung Allograft Dysfunction After Lung Transplantation[NCT02936505] | | 249 participants (Actual) | Interventional | 2016-10-12 | Active, not recruiting |
Gemtuzumab Ozogamicin (GO), Fludarabine, And Low-Dose TBI Followed By Donor Stem Cell Transplantation For Patients With Advanced Acute Myeloid Leukemia Or Myelodysplastic Syndrome[NCT00008151] | Phase 2 | 0 participants | Interventional | 2000-10-31 | Completed |
Non-Ablative Chemotherapeutic Conditioning Before Allogeneic Stem Cell Transplantation[NCT00008307] | Phase 2 | 52 participants (Anticipated) | Interventional | 1998-04-30 | Active, not recruiting |
Efficacy and Safety of Alefacept in Combination With Tacrolimus, Mycophenolate Mofetil and Steroids in de Novo Kidney Transplantation - a Multicenter, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study[NCT00617604] | Phase 2 | 218 participants (Actual) | Interventional | 2007-12-31 | Completed |
Mycophenolate Mofetil in the Treatment of Wegener's Granulomatosis and Related Vasculitides[NCT00001764] | Phase 1 | 50 participants | Interventional | 1998-04-30 | Completed |
Evaluation of the Benefit/Risk Ratio of a Reduction of Tacrolimus Dose in Association With Mycophenolate Mofetil on the Prevention of Complications in Adult Liver Transplantation[NCT00151632] | Phase 3 | 195 participants (Actual) | Interventional | 2003-05-31 | Terminated(stopped due to insufficient enrollment) |
Mycophenolate Mofetil Versus Azathioprine for Maintenance Therapy of Lupus Nephritis[NCT02949349] | Phase 2 | 32 participants (Actual) | Interventional | 2015-07-31 | Completed |
Nature of Anifrolumab Impact on Vaccine-Emergent Immunity in Patients With Moderately to Severely Active Systemic Lupus Erythematosus: A Multi-Centre Open Label Parallel Group Trial:[NCT04726553] | Early Phase 1 | 20 participants (Anticipated) | Interventional | 2021-01-20 | Recruiting |
A Multicentre Randomised Trial of First Line Treatment Pathways for Newly Diagnosed Immune Thrombocytopenia: Standard Steroid Treatment Versus Combined Steroid and Mycophenolate[NCT03156452] | Phase 3 | 123 participants (Actual) | Interventional | 2017-10-25 | Completed |
A Study of the Effect of Conversion to Enteric-Coated Mycophenolate Sodium (EC-MPS) on Quality of Life in Patients With Gastrointestinal (GI) Symptoms Related to Mycophenolate Mofetil Therapy After Kidney Transplantation (MYQOL)[NCT00239005] | Phase 4 | 134 participants (Actual) | Interventional | 2005-09-30 | Completed |
Conversion Trial From Mycophenolate Mofetil (MMF) to Enteric-Coated Mycophenolate Sodium (EC-MPS) in Stable Transplanted Patients Suffering From GI Adverse Events While on Mycophenolate Mofetil Therapy[NCT00149942] | Phase 4 | 23 participants (Actual) | Interventional | 2004-10-31 | Completed |
A Pilot Study of Mycophenolate Mofetil (MMF) in Patients With p-ANCA Microscopic Polyangiitis and Mild to Moderate Renal Dysfunction.[NCT00405860] | Phase 1 | 18 participants (Actual) | Interventional | 2002-12-31 | Completed |
Safety and Tolerability of Enteric-Coated Mycophenolate Sodium in Renal Transplant Patients With GI Intolerance[NCT00150020] | Phase 4 | 728 participants (Anticipated) | Interventional | 2004-10-31 | Completed |
An Open-label Study to Assess the Efficacy and Safety of Alipogene Tiparvovec (AMT-011), Human LPL [S447X], Expressed by an Adeno-Associated Viral Vector After Intramuscular Administration in LPL-deficient Adult Subjects[NCT00891306] | Phase 2/Phase 3 | 5 participants (Actual) | Interventional | 2009-02-28 | Completed |
An Open-Label, Randomized, Prospective Multicenter Study To Compare The Efficacy And Safety Among 3 Immunosuppressant Treatment Regimens In Patients Receiving A Liver Transplant For ESLD Caused By Chronic Hepatitis C[NCT00163657] | Phase 4 | 312 participants (Actual) | Interventional | 2002-07-31 | Completed |
A 3 Month, Multicenter, Open-label Study to Evaluate the Impact of the Immunosuppressive Combination of Enteric-Coated Mycophenolate Sodium (EC- MPS), Basiliximab and Cyclosporine for Microemulsion With C2 Monitoring, on Efficacy and Safety Outcomes in de[NCT00154232] | Phase 4 | 46 participants | Interventional | 2004-06-30 | Completed |
Pilot Trial for Implementation of a Medroxyprogesterone(MPA)Pharmacokinetic(PK) Monitoring Strategy in Patients on Mycophenolate Mofetil(MMF)/FK Based Immunosuppression.[NCT00187941] | Early Phase 1 | 22 participants (Actual) | Interventional | 2005-08-31 | Completed |
A Single Dose, 2-Period, 2-Treatment 2-Way Crossover Bioequivalency Study of Mycophenolate Mofetil 500 mg Tablets Under Fed Conditions[NCT00893958] | | 39 participants (Actual) | Interventional | 2006-01-31 | Completed |
A 36 Month Multi-center, Open Label, Randomized, Comparator Study to Evaluate the Efficacy and Safety of Everolimus Immunosuppression Treatment in Liver Transplantation for Hepatocellular Carcinoma Exceeding Milan Criteria[NCT02081755] | Phase 4 | 336 participants (Anticipated) | Interventional | 2014-03-31 | Active, not recruiting |
Prospective Cohort Study of Clinical and Imaging Patterns of Neuroinflammation Diseases (CLUE)[NCT04106830] | | 1,000 participants (Anticipated) | Observational [Patient Registry] | 2019-01-01 | Recruiting |
Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Non-Myeloablative Preparative Regimen[NCT00719849] | Phase 2 | 13 participants (Actual) | Interventional | 2005-11-30 | Terminated(stopped due to A new protocol was developed to replace this protocol in 2008, with removal of ATG and extension of MMF duration.) |
A Phase II Exploratory Study to Determine the Safety and Study the Immunomodulatory Functions of Induction Therapy With Campath, Combined With Chronic Immunosuppression With Mycophenolate Mofetil and Sirolimus[NCT00240994] | Phase 2 | 35 participants (Actual) | Interventional | 2005-01-31 | Completed |
A Randomized, Open-label Study to Compare the Effect of CellCept Plus Corticosteroids, and Cyclophosphamide Plus Corticosteroids Followed by Azathioprine, on Remission Rate in Patients With Lupus Nephritis[NCT00425438] | Phase 3 | 52 participants (Actual) | Interventional | 2007-03-31 | Terminated(stopped due to Study was terminated early for administrative reasons.) |
The Research of Standard Diagnosis and Treatment for Henoch-Schonlein Purpura Nephritis in Children[NCT02532777] | Phase 2 | 100 participants (Anticipated) | Interventional | 2015-08-31 | Recruiting |
A Randomized, Multicenter, Open-label, 6-month Study to Explore the Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Combination With Two Corticosteroid Regimens for the Treatment of Lupus Nephritis Flare[NCT00423098] | Phase 2 | 81 participants (Actual) | Interventional | 2007-02-28 | Completed |
Phase II Clinical Trial of Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis[NCT01047072] | Phase 2 | 0 participants (Actual) | Interventional | | Withdrawn |
A Prospective, Open-label, Controlled Multicenter Trial to Assess the Efficacy and Safety of an Induction Regimen of Cyclosporine Micro Emulsion, Enteric-coated Mycophenolate Sodium (EC-MPS) and Corticosteroids, Followed by Administration of Everolimus an[NCT00371826] | Phase 4 | 126 participants (Actual) | Interventional | 2006-03-31 | Completed |
A 4-week, Multicenter, Double-blind, Double-dummy, Randomized, Parallel Group Study to Compare the Gastrointestinal Safety and Tolerability of EC-MPS & MMF When Administered in Combination With Calcineurin Inhibitors in Renal Transplant Recipients Experie[NCT00400400] | Phase 4 | 400 participants (Actual) | Interventional | 2006-10-31 | Completed |
Non-myeloablative Allogeneic Transplantation for the Treatment of Multiple Myeloma[NCT00185614] | Phase 2 | 63 participants (Actual) | Interventional | 2000-08-31 | Completed |
Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating an Intensified Enteric-coated Mycophenolate Sodium (EC-MPS) Dosing Regimen in Comparison to a Standard Dosing Regimen of EC-MPS in Combination With Cyclosporin Microemul[NCT00369278] | Phase 3 | 128 participants (Actual) | Interventional | 2006-06-30 | Completed |
Timed Sequential Busulfan and Post Transplant Cyclophosphamide for Allogeneic Transplantation[NCT02861417] | Phase 2 | 204 participants (Actual) | Interventional | 2016-08-05 | Active, not recruiting |
A Randomized Controlled Trial of Mycophenolate Mofetil in Patients With IgA Nephropathy[NCT00318474] | Phase 3 | 184 participants (Actual) | Interventional | 2002-01-31 | Terminated(stopped due to DSMB recommended stopping the trial because of lack of effect.) |
Transplantation of Unrelated Umbilical Cord Blood for Patients With Hematological Diseases With Cyclophosphamide/Fludarabine/Total Body Irradiation Myeloablative Preparative Regimen[NCT00309842] | Phase 2 | 213 participants (Actual) | Interventional | 2005-07-28 | Completed |
Haploidentical Stem Cell Transplant Using Post Transplant Cyclophosphamide for GvHD Prophylaxis: A Pilot Study[NCT02248597] | Phase 2 | 27 participants (Actual) | Interventional | 2015-02-25 | Completed |
A Multicenter, National, Open-label, Prospective, Randomized Study to Evaluate Efficacy and Tolerability of Enteric-coated Mycophenolate Sodium 1440 mg/Day With Tacrolimus Reduced Dose Versus Enteric-coated Mycophenolate Sodium 720 mg/Day With Tacrolimus [NCT00284934] | Phase 3 | 94 participants (Actual) | Interventional | 2005-12-31 | Completed |
Steroid Free Immunosuppression in Liver Transplantation[NCT00296244] | Phase 4 | 40 participants (Actual) | Interventional | 2006-02-28 | Completed |
Single Center, Open-label Randomized Prospective Trial: Effect of Sirolimus on Polycystic Liver Disease[NCT00934791] | | 2 participants (Actual) | Interventional | 2009-02-28 | Terminated(stopped due to Terminated due to inadequate enrollment) |
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Simultaneous Pancreas-Kidney Transplant Recipients[NCT00267150] | Phase 3 | 31 participants (Actual) | Interventional | 2005-11-30 | Completed |
Efficacy and Safety Study Comparing Concentration-controlled Everolimus in Two Doses (1.5 and 3.0 mg/Day Starting Doses) With Reduced Cyclosporine Versus 1.44 g Mycophenolic Acid (as Sodium Salt) With Standard Dose Cyclosporine in de Novo Renal Transplant[NCT00251004] | Phase 3 | 833 participants (Actual) | Interventional | 2005-10-31 | Completed |
The Highly Sensitized Patients: Effects of Mycophenolate Mofetil (MMF) On Anti-Human Leukocyte Antigen (HLA) Antibody Levels In Patients Awaiting Cadaveric Renal Transplant[NCT00446459] | Phase 2 | 45 participants (Actual) | Interventional | 2006-04-30 | Completed |
CellCept (Mycophenolate Mofetil, MMF) Maintenance Immunosuppression in Liver Transplant Recipients With Long-term Follow-up Post-transplantation for Non-Autoimmune Liver Disease - A Prospective, Randomized, Multicenter Trial.[NCT00206076] | Phase 4 | 19 participants (Actual) | Interventional | 2006-08-31 | Completed |
An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies[NCT04547049] | Phase 3 | 160 participants (Anticipated) | Interventional | 2020-09-01 | Recruiting |
Phase IV Study of Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion (CsA-ME) in Kidney Transplant Patients[NCT00239083] | Phase 4 | 40 participants (Anticipated) | Interventional | 2005-01-31 | Completed |
[NCT01895049] | Phase 4 | 171 participants (Actual) | Interventional | 2013-08-31 | Completed |
A Multicenter, Single Arm, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Satralizumab in Patients With Neuromyelitis Optica Spectrum Disorder (NMOSD)[NCT04660539] | Phase 3 | 119 participants (Actual) | Interventional | 2021-03-02 | Active, not recruiting |
Regulatory T Cell Modulation in Kidney Transplantation With Biologic Blockade of Dual Effector Pathways, CD28 and IL-6 (CTOT-24)[NCT04066114] | Phase 1/Phase 2 | 10 participants (Actual) | Interventional | 2019-12-11 | Completed |
Randomized Controlled Study: Effect of Mycophenolatmofetil in Patients With Histologically Proven Chronic Allograft Nephropathy[NCT00204230] | | 86 participants | Interventional | 1999-10-31 | Terminated |
[NCT00411515] | Phase 4 | 0 participants | Interventional | | Completed |
A Prospective, Open Label Protocol to Assess the Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients[NCT00238953] | Phase 4 | 30 participants (Actual) | Interventional | 2005-02-28 | Completed |
A Prospective, Randomized, Open Label, Multicenter Trial of EC-MPS With Steroid Withdrawal vs EC-MPS With Standard Steroid Regimen for the Prevention of Acute Rejection Episodes in de Novo Renal Transplant Recipients.[NCT00238992] | Phase 3 | 144 participants | Interventional | 2002-12-31 | Completed |
Multicentre, Controlled, Prospective, Randomized, Open-label Clinical Trial to Compare Enteric-Coated Mycophenolate Sodium (EC-MPS) Plus Reduced Dose Cyclosporine Microemulsion (CsA-ME) Vs EC-MPS Plus Standard Dose CsA-ME in Elderly de Novo Renal Transpla[NCT00239031] | Phase 3 | 117 participants (Actual) | Interventional | 2002-03-31 | Completed |
A Prospective, Open-label, Multicenter, International Follow-up Study on the Safety and Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) in Kidney Transplant Recipients[NCT00239070] | Phase 3 | 69 participants (Actual) | Interventional | 2003-04-30 | Completed |
A Prospective, Open-label, Multicenter, International Follow-up Study on the Safety and Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) in Kidney Transplant Recipients[NCT00241059] | Phase 4 | 183 participants (Actual) | Interventional | 2002-08-31 | Completed |
Non-Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation for the Treatment of Metastatic Renal Cell Carcinoma[NCT00243009] | Phase 2 | 1 participants (Actual) | Interventional | 2005-06-30 | Terminated(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 1 | 8 participants (Actual) | Interventional | 2013-10-07 | Terminated(stopped due to Funding unavailable) |
A Prospective, Open, Randomized Controlled Stage II Trial Investigating the Efficacy and Safety of Thymosin Alpha-1 in Treating Moderate to Severe Immune-related Adverse Events[NCT06178146] | Phase 4 | 40 participants (Anticipated) | Interventional | 2023-09-01 | Recruiting |
ICON1: Physician Treatment Decisions and Patient-Reported Outcomes in Pediatric Refractory Immune Thrombocytopenia[NCT01971684] | | 120 participants (Actual) | Observational | 2013-08-31 | Completed |
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 2 | 25 participants (Anticipated) | Interventional | 2013-11-30 | Active, not recruiting |
A One-Year Prospective, Randomized, Placebo-Controlled, Double-Blind, Phase II/III Safety Trial of Combination Therapy With IFN Beta-1a (Avonex) and Mycophenolate Mofetil (Cellcept) in Early Multiple Sclerosis[NCT00223301] | Phase 2/Phase 3 | 24 participants | Interventional | 2004-07-31 | Completed |
MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions[NCT02582775] | Phase 2 | 17 participants (Actual) | Interventional | 2016-03-31 | Completed |
6-month, Open-label, Randomized, Multicenter, Prospective, Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Everolimus in de Novo Renal Transplant Recipients Participating in the Eurotransplant Senior Program[NCT00956293] | Phase 4 | 207 participants (Actual) | Interventional | 2009-07-31 | Terminated(stopped due to The study was terminated because the required sample size of 240-260 de novo senior renal transplant patients was not achieved within a reasonable time.) |
Phase I/II Study of HLA-Matched Non-Myeloablative Peripheral Blood Mobilized Hematopoietic Progenitor Cell Transplantation as Treatment for Patients With Metastatic Renal Cell Carcinoma. A Multi-Center Trial.[NCT00005851] | Phase 1/Phase 2 | 11 participants (Actual) | Interventional | 2000-02-29 | Completed |
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 2 | 0 participants | Interventional | 1999-11-30 | Completed |
Efficacy and Safety of Certican® (Everolimus) in Combination With Myfortic® (EC-MPS, Enteric-coated Mycophenolate Sodium) After Early CNI Elimination Versus Myfortic® in Combination With Prograf® in Renal Transplant Recipients[NCT00965094] | Phase 4 | 36 participants (Actual) | Interventional | 2009-12-31 | Completed |
A 12-month, Single-blind, Randomized, Parallel Group, Multicenter Study to Investigate the Efficacy and Safety of ERL080A Compared With MMF in de Novo Heart Recipients[NCT00574743] | Phase 4 | 162 participants (Actual) | Interventional | 2002-01-31 | Completed |
A Randomized Open-Label Study Comparing the Efficacy and Safety of Sirolimus Combined With Daclizumab, Mycophenolate and Corticosteroids vs Cyclosporine, Mycophenolate and Corticosteroids in Renal Allograft Recipients Receiving Kidneys From Older Donors[NCT00195273] | Phase 3 | 61 participants (Actual) | Interventional | 2004-11-30 | Completed |
Comparison Between Tacrolimus and Mycophenolate Mofetil for Induction of Remission in Lupus Nephritis[NCT01580865] | | 84 participants (Actual) | Interventional | 2012-05-31 | Completed |
A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Safety, Tolerability and Clinical Effect of Laquinimod in Active Lupus Nephritis Patients, in Combination With Standard of Care (Mycophenolate Mofetil and Steroid[NCT01085097] | Phase 2 | 46 participants (Actual) | Interventional | 2010-09-01 | Completed |
Efficacy and Safety of Mycophenolate Mofetil as Maintenance Therapy After Rituximab Treatment in Childhood-onset, Frequently-relapsing or Steroid-dependent Nephrotic Syndrome: a Multicenter Double-blind, Randomized, Placebo-controlled Trial[NCT04531865] | Phase 3 | 0 participants (Actual) | Interventional | 2021-01-01 | Withdrawn(stopped due to lack of funding) |
Pharmacologic Pretransplant Immunosuppression (PTIS) + Reduced Toxicity Conditioning (RTC) Allogeneic Stem Cell Transplantation in Inherited Hematologic Disorders[NCT05293509] | Phase 2 | 0 participants (Actual) | Interventional | 2022-03-02 | Withdrawn(stopped due to 0 accrual) |
Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) for the Treatment of Hematological Diseases [MT2015-32][NCT02661035] | Phase 2 | 156 participants (Actual) | Interventional | 2017-03-09 | Completed |
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 1 | 24 participants (Anticipated) | Interventional | 2015-06-25 | Active, not recruiting |
Tacrolimus, Mini-dose Methotrexate and Mycophenolate Mofetil Versus Tacrolimus and Methotrexate for the Prevention of Acute Graft-versus-Host-Disease[NCT01951885] | Phase 3 | 101 participants (Actual) | Interventional | 2014-07-07 | Completed |
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 2 | 273 participants (Actual) | Interventional | 2012-10-16 | Active, not recruiting |
Multicenter, Open-label, Randomized, 24 Months Follow-up, Two Arm Study to Compare the Efficacy of Everolimus in Improving the Cardiovascular Profile in a Regimen With Mycophenolic Acid vs. a Regimen of CNI+MPA in Maintenance Renal Transplant Recipients.[NCT01169701] | Phase 4 | 71 participants (Actual) | Interventional | 2010-08-31 | Completed |
Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant[NCT03249831] | Phase 1 | 3 participants (Actual) | Interventional | 2019-01-04 | Active, not recruiting |
A Randomized, Open-Label, Comparative, Multi-Center Study to Assess the Safety and Efficacy of Prograf (Tacrolimus)/MMF and Extended Release (XL) Tacrolimus /MMF in de Novo Kidney Transplant Recipients[NCT00717678] | Phase 3 | 73 participants (Actual) | Interventional | 2007-12-31 | Completed |
Hematopoietic Stem Cell Transplantation From Haploidentical Donors in Patients With Hematological Malignancies Using a Treosulfan-Based Preparative Regimen[NCT04195633] | Phase 2 | 60 participants (Anticipated) | Interventional | 2021-01-25 | Recruiting |
An Multi-site, Open, Prospective Study to Assess the Efficacy and Safety of Multi-target Therapy in the Treatment of Class Ⅲ,Ⅳ,Ⅴ,Ⅲ+Ⅴand Ⅳ+Ⅴ Lupus Nephritis[NCT00876616] | | 362 participants (Actual) | Interventional | 2009-04-30 | Completed |
Head to Head Comparison of Tacrolimus and Myfortic vs Tacrolimus and Sirolimus Used in Combination in Non-HLA Identical Living Donor Kidney Transplants[NCT01038505] | Phase 4 | 0 participants (Actual) | Interventional | 2010-01-31 | Withdrawn(stopped due to Lost funding source) |
Pharmacokinetic Evaluation of an Intensified and Decreasing Dosing Regimen of Mycophenolate Sodium in Combination With Tacrolimus During the First 3 Months Post Kidney Transplant (the myFORTic Study)[NCT00941824] | Phase 4 | 15 participants (Anticipated) | Interventional | 2009-02-28 | Completed |
Open Randomized Mult-Center Study to Evaluate Safety and Efficacy of Low Molecular Weight Sulfated Dextran in Islet Transplantation (CIT-01)[NCT00789308] | Phase 2 | 24 participants (Actual) | Interventional | 2008-07-11 | Completed |
Multicentre, Open Study for the Setting up of Population Pharmacokinetic Models and Bayesian Estimators for Individual Dose Adjustment of Immunosuppressive Drugs (Cyclosporine, Tacrolimus, Mycophenolate Mofetil, Everolimus) During the First Year Post-graf[NCT00812786] | Phase 4 | 42 participants (Actual) | Interventional | 2007-07-31 | Completed |
Open Label Randomized Study of Thymoglobulin Versus Daclizumab Induction Therapies for the Reduction of Acute Rejection in Live Donor Kidney Transplant Recipients With a Positive Crossmatch[NCT00275509] | Phase 3 | 56 participants (Actual) | Interventional | 2007-01-31 | Completed |
Total Marrow Irradiation and Myeloablative Chemotherapy Followed By Double Umbilical Cord BloodTransplantation In Patients With Refractory Acute Leukemia[NCT00686556] | Phase 1 | 12 participants (Actual) | Interventional | 2012-08-31 | Completed |
"Prospective Randomized Controlled Trial to Compare a Calcineurin Inhibitor Free Immunosuppression With a Low Dose Tacrolimus Based Immunosuppression in Old for Old Kidney Transplantation."[NCT00912678] | Phase 4 | 90 participants (Actual) | Interventional | 2002-03-31 | Completed |
Gemtuzumab Ozogamicin in Combination With Busulfan and Cyclophosphamid and Allogenic Stem Cell Transplantation in Patients With High Risk CD33+ Acute Myelogenous Leukemia/Myelodysplastic Syndrome/Juvenile Myelomonocytic Leukemia[NCT00669890] | Phase 1 | 12 participants (Actual) | Interventional | 2004-05-31 | Terminated(stopped due to PI left institution) |
Biomarker-Linked Outcomes of Cellcept in Lupus Arthritis[NCT00594932] | Phase 1/Phase 2 | 27 participants (Actual) | Interventional | 2006-11-30 | Completed |
Comparative Bioavailability of Myfenax® (Teva) and CellCept® (Roche) in Stable Patients After Renal Transplantation[NCT00991510] | Phase 4 | 43 participants (Actual) | Interventional | 2009-08-31 | Terminated(stopped due to Slow recruitment and lack of time to product launch) |
A Phase II Study of Optimally Dosed Clofarabine in Combination With Low-Dose TBI to Decrease Relapse Rates After Related or Unrelated Donor Hematopoietic Cell Transplantation in Patients With AML[NCT01252667] | Phase 2 | 44 participants (Actual) | Interventional | 2011-01-25 | Completed |
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 1 | 24 participants (Actual) | Interventional | 2016-07-31 | Active, not recruiting |
Allogeneic Stem Cell Transplantation for Children and Adolescents With CML: Conditioning Regimen, Donor Selection, Supportive Care and Diagnostic Procedures[NCT02707393] | Phase 2/Phase 3 | 13 participants (Actual) | Interventional | 2009-04-30 | Completed |
A 12 Month, Multi-center, Randomized, Open-label Non-inferiority Study Comparing Safety and Efficacy of Concentration-controlled Everolimus With Low Dose Tacrolimus to Mycophenolate Mofetil With Standard Dose Tacrolimus in de Novo Renal Transplant Recipie[NCT01025817] | Phase 3 | 613 participants (Actual) | Interventional | 2010-01-31 | Completed |
Efficacy and Safety of Mycophenolate Mofetil in subjectswithSjogren's Syndrome[NCT02691949] | Phase 2 | 54 participants (Anticipated) | Interventional | 2016-02-29 | Enrolling by invitation |
Sirolimus Associated With Tacrolimus at Low Doses in Elderly Kidney Transplant Patients: A Prospective Randomized Controlled Trial[NCT02683291] | Phase 4 | 48 participants (Actual) | Interventional | 2014-01-31 | Completed |
A Phase III Multicenter, Randomized Trial Comparing Tacrolimus/Sirolimus/Methotrexate Versus Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil for GVHD Prophylaxis After Reduced Intensity Allogeneic Stem Cell Transplantation for Patients With [NCT00928018] | Phase 3 | 139 participants (Actual) | Interventional | 2009-06-30 | Completed |
A Three-Month, Open-Label, Two Cohort Study to Investigate the Safety and Tolerability of Myfortic in Combination With Neoral (Cyclosporin) or Prograf (Tacrolimus) in Liver Transplant Recipients With GI Intolerance[NCT00619216] | | 31 participants (Actual) | Observational | 2008-03-31 | Completed |
A Prospective Randomized Trial of Prednisone and Tacrolimus Versus Prednisone, Tacrolimus and Mycophenolate Mofetil in Pediatric Liver Transplantation[NCT00656266] | Phase 4 | 13 participants (Actual) | Interventional | 2004-11-30 | Terminated(stopped due to insufficient funding) |
A Single-Dose, 2-Period, 2-Treatment, 2-Way Crossover Bioequivalence Study of Mycophenolate Mofetil Capsules Under Fed Conditions[NCT00910663] | Phase 1 | 60 participants (Actual) | Interventional | 2006-10-31 | Completed |
Selective CD28 Blockade With Lulizumab Compared to CNI Inhibition With Tacrolimus in Renal Transplant Recipients[NCT04903054] | Phase 2 | 0 participants (Actual) | Interventional | 2022-01-10 | Withdrawn(stopped due to Limited period of availability of a supply of the study drug and difficulties in enrollment) |
Cyclophosphamide Versus Anti-thymocyte Globulin for GVHD Prophylaxis After RIC Allo-SCT[NCT02876679] | Phase 2 | 94 participants (Actual) | Interventional | 2017-04-06 | Completed |
Evaluation of the Benefit of Antithymocyte Induction Therapy on Hepatic Fibrosis in de Novo Hepatitis C Virus Liver Transplant Patients.[NCT00538265] | Phase 4 | 100 participants (Anticipated) | Interventional | 2005-05-31 | Completed |
IIT2021-11-PAQUETTE-OmitMMF: Fludarabine and Total Body Irradiation 800 cGy or 1125 cGy For Allogeneic Stem Cell Transplant Using Graft Versus Host Disease Prophylaxis With Post-Transplant Cyclophosphamide and Tacrolimus, Without Mycophenolate Mofetil[NCT05256537] | Phase 2 | 60 participants (Anticipated) | Interventional | 2022-04-26 | Recruiting |
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 1 | 25 participants (Actual) | Interventional | 2020-08-14 | Active, not recruiting |
Pharmacogenetics of Mycophenolic Acid in Kidney Transplant Patients[NCT00433953] | Phase 1 | 44 participants (Actual) | Interventional | 2007-02-28 | Terminated(stopped due to No longer following patient and no plans to publish.) |
Phase II Study of Shortened-duration Tacrolimus Following Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Malignancies That Are Challenging to Engraft[NCT02556931] | Phase 2 | 117 participants (Actual) | Interventional | 2015-12-31 | Completed |
Pilot Study of Mycophenolate Mofetil in Congenital Uropathies[NCT00193635] | Phase 1 | 12 participants (Actual) | Interventional | 2002-03-31 | Completed |
Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Cyclosporine Microemulsion Based Regimen in de Novo Living Donor Kidney Transplant Recipients ; A Prospective, Open Label, Multi-center Study[NCT00537862] | Phase 4 | 200 participants (Actual) | Interventional | 2006-05-31 | Completed |
An Open, Prospective, Randomised, Controlled, Multi-Center Study Comparing Fixed Dose vs Concentration Controlled Mycophenolate Mofetil Regimens for de Novo Patients Following Transplantation[NCT00166244] | Phase 4 | 901 participants (Actual) | Interventional | 2003-05-31 | Completed |
Non-Myeloablative Allogeneic Hematopoietic Cell Transplantation For Patients With Disease Relapse Or Myelodysplasia After Prior Autologous Transplantation[NCT00053196] | Phase 2 | 82 participants (Actual) | Interventional | 2002-12-31 | Completed |
The Effect of Sirolimus on the Pharmacokinetics of Tacrolimus[NCT00166829] | Phase 4 | 40 participants | Interventional | 2004-05-31 | Recruiting |
Enteric Coated Mycophenolic Acid (Myfortic) in Liver Transplant Recipients- Effect on Compliance and Calcineurin Inhibitor and Corticosteroid Sparing[NCT00167492] | Phase 4 | 0 participants (Actual) | Interventional | 2005-09-30 | Withdrawn |
An Open Label, Multi-centre Trial of Alipogene Tiparvovec for the Treatment of LPLD Patients[NCT02904772] | Phase 2 | 0 participants (Actual) | Interventional | 2016-10-31 | Withdrawn(stopped due to uniQure, has decided not to renew the Marketing Authorization of Glybera in the EU. This decision is not related to any safety, efficacy or quality issue) |
A Pilot Study of Double Cord Blood Stem Cell Transplantation in Patients With Hematologic Malignancies[NCT00423826] | | 0 participants | Expanded Access | 2007-01-31 | No longer available |
Phase II Trial of Non-Myeloablative Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation Using Fludarabine, Low-Dose TBI, and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil (MMF) Followed by Donor Lymphocyte Infusion[NCT00006233] | Phase 2 | 0 participants | Interventional | 2000-01-31 | Completed |
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 2 | 21 participants (Actual) | Interventional | 2000-05-31 | Completed |
Open-label Study to Evaluate the Tolerability, Safety and Efficacy of the Equimolar Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium in Patients With Stable Renal Transplant Receiving Tacrolimus[NCT00171392] | Phase 3 | 132 participants (Actual) | Interventional | 2004-03-31 | Completed |
Open Label, Randomized, Single-dose, Crossover Study to Evaluate the Pharmacokinetic Characteristics of CKD-4101 Tablet, in Healthy Volunteers[NCT01016626] | Phase 1 | 28 participants (Actual) | Interventional | 2009-07-31 | Completed |
A Pilot Study of Campath-1H Induction Therapy Combined With Rituximab®, Myfortic™ and a Short Course of Calcineurin Inhibitor Therapy to Allow for a Long Term Calcineurin Inhibitor Free Regimen After Renal Transplantation[NCT00579592] | | 11 participants (Actual) | Interventional | 2006-04-30 | Terminated(stopped due to Higher than expected rate of acute rejection) |
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in Renal Transplant Patients.[NCT00149916] | Phase 3 | 139 participants (Actual) | Interventional | 2000-04-30 | Completed |
An Open Label, Prospective, Randomized, Controlled, Multicenter Study Assessing Fixed Dose vs. Concentration Controlled CellCept Regimens for Patients Following a Single Organ Renal Transplantation in Combination With Full Dose and Reduced Dose Calcineuri[NCT00217152] | Phase 4 | 12 participants (Actual) | Interventional | 2005-03-31 | Terminated(stopped due to Roche decided to prematurely terminate study.) |
Role of Th1, Th2 and Monokine Responses as Risk Factors of Acute and Chronic Renal Transplant Rejection - Impact of Different Immunosuppressive Protocols[NCT00150891] | | 84 participants (Actual) | Observational | 1998-01-31 | Completed |
Evaluating the Safety of Myfortic (Mycophenolate Sodium) in Patients With Lupus Nephritis: a 12 Month, Single-arm, Observational Study in Taiwan Population[NCT04645589] | | 64 participants (Anticipated) | Observational | 2021-03-16 | Recruiting |
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 2 | 27 participants (Anticipated) | Interventional | 2020-02-12 | Recruiting |
A Pilot Study of Campath-1H Induction Therapy Combined With CellCept® Therapy to Allow for a Calcineurin Inhibitor Free Regimen After Renal Transplantation[NCT00214266] | Phase 2 | 31 participants (Actual) | Interventional | 2005-01-31 | Completed |
A Phase III, Randomised, Single-site Trial on the Minimisation of Immunosuppression In Elderly Renal Transplant Recipients.[NCT05073822] | Phase 1/Phase 2 | 0 participants (Actual) | Interventional | 2023-02-20 | Withdrawn(stopped due to Funding not secured) |
Induction in Sensitized Kidney Transplant Recipients Without Preexisting Donor-specific antiboDies: a Randomized Multicentre Trial Between a Lymphocyte Depleting and Basiliximab.[NCT05385432] | Phase 3 | 244 participants (Anticipated) | Interventional | 2023-09-12 | Not yet recruiting |
Rituximab For Prevention Of Acute Graft-Versus-Host Disease (GVHD) After Unrelated Donor Allogeneic Hematopoietic Cell Transplantation (HCT)[NCT01044745] | Phase 2 | 20 participants (Actual) | Interventional | 2009-12-10 | Terminated(stopped due to Study was closed to accrual for safety related to the frequency of BK infections.) |
A Randomized Controlled Trial to Compare the Efficacy of Oral Mycophenolate Mofetil With Placebo in Patients With Systemic Sclerosis Related Early Interstitial Lung Disease[NCT02896205] | Phase 3 | 41 participants (Actual) | Interventional | 2016-10-31 | Completed |
Phase III Multicenter Open-label Randomized Clinical Trial Comparing Everolimus and Low Dose Tacrolimus to Tacrolimus and Mycophenolate Mofetil at 6 mo Post-Transplant to Prevent Long-term Complications After Pediatric Heart Transplantation[NCT03386539] | Phase 3 | 211 participants (Actual) | Interventional | 2018-01-29 | Active, not recruiting |
A Phase II Trial of Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched Peripheral Blood Stem Cells for Patients With Hematologic Malignancies[NCT00782379] | Phase 2 | 20 participants (Actual) | Interventional | 2008-10-31 | Completed |
Standard Therapy or Individualized Immunosuppression For Lowering Adverse Event Risk[NCT04473924] | Phase 2 | 0 participants (Actual) | Interventional | 2022-07-31 | Withdrawn(stopped due to Study has never started as sponsor did not approve for funding.) |
A Pilot Study Comparing the Safety and Efficacy of Zortress (Everolimus) With Low Dose Tacrolimus to Early Conversion to Calcineulin Inhibitor-Free Regimen and Mycophenolic Acid With Standard Dose Tacrolimus in Recipients of ECD/DCD Kidneys[NCT01878786] | Phase 2/Phase 3 | 25 participants (Actual) | Interventional | 2013-06-30 | Terminated(stopped due to Interim results suggested a concern for patient outcomes and safety) |
Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies[NCT01471067] | Phase 1 | 33 participants (Actual) | Interventional | 2012-07-13 | Completed |
A Study to Investigate the Impact of Pharmacogenetics on CellCept Use, in Patients Participating in a Study in Renal Transplantation[NCT00337493] | Phase 4 | 155 participants (Actual) | Interventional | 2005-12-31 | Completed |
Open-Label, Randomized Study Comparing the Patient Reported Severity of GI Side Effects of MMF Versus EC-MPS in Maintenance Heart Transplant Patients.[NCT00468936] | Phase 3 | 100 participants (Anticipated) | Interventional | 2007-05-31 | Recruiting |
Randomized Comparative Study on Effects of Immunosuppression on HCV Recurrence After Living Donor Liver Transplantation - Comparison Between Tacrolimus + MMF and Tacrolimus + Steroid[NCT00469131] | Phase 3 | 79 participants (Actual) | Interventional | 2003-09-30 | Completed |
Mycophenolic Acid Monotherapy in Recipients of HLA-identical Living-Related Transplantation[NCT01053221] | Phase 2 | 16 participants (Actual) | Interventional | 2006-03-31 | Terminated(stopped due to Funding loss) |
Evaluate the Efficacy and Safety of RaparoBell® Tablet Plus Calcineurin Inhibitors Compared With Mycophenolate Mofetil Plus Calcineurin Inhibitors in ABO Incompatible De Novo Living Kidney Transplant Recipients. [ART Study][NCT04700709] | Phase 4 | 158 participants (Anticipated) | Interventional | 2021-01-31 | Not yet recruiting |
Pharmacokinetics of Everolimus and Enteric-Coated Mycophenolatesodium Before and After Withdrawal of Cyclosporine in Stable Renal Transplant Patients[NCT00443937] | Phase 4 | 15 participants (Anticipated) | Interventional | 2004-01-31 | Completed |
Natural Killer Cells in Allogeneic Cord Blood Transplantation[NCT01619761] | Phase 1 | 13 participants (Actual) | Interventional | 2013-05-03 | Active, not recruiting |
A Randomized, Prospective, Multicenter Trial to Compare the Effect on Chronic Allograft Nephropathy Prevention of Mycophenolate Mofetil Versus Azathioprine as the Sole Immunosuppressive Therapy for Kidney Transplant Recipients[NCT00494741] | Phase 4 | 233 participants (Actual) | Interventional | 2007-05-31 | Completed |
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplantation With JSP191-Based Conditioning in Participants With GATA2 Deficiency[NCT05907746] | Phase 2 | 32 participants (Anticipated) | Interventional | 2023-11-29 | Recruiting |
Targeted Astatine-211-Labeled BC8-B10 Monoclonal Antibody as Reduced Intensity Conditioning for Nonmalignant Diseases[NCT04083183] | Phase 1/Phase 2 | 40 participants (Anticipated) | Interventional | 2020-06-16 | Recruiting |
Mesenchymal Stem Cells Under Basiliximab/Low Dose RATG to Induce Renal Transplant Tolerance[NCT00752479] | Phase 1/Phase 2 | 4 participants (Actual) | Interventional | 2008-05-31 | Terminated(stopped due to Necessity of major revision of the protocol) |
A Randomized Study To Compare The Safety And Efficacy Of Two Immunosuppressive Regimens In De Novo Renal Allograft Recipients:Sirolimus Plus Mycophenolate Mofetil Plus Corticosteroids Following A Rabbit Anti-Human Thymocyte Globulin Induction (RATG) Vs Ta[NCT00261820] | Phase 4 | 160 participants | Interventional | | Completed |
Benadamustine, Fludarabine and Busulfan Conditioning in Recipients of Haploidentical Stem Cell Transplantation (FluBuBe)[NCT04942730] | Phase 2 | 40 participants (Anticipated) | Interventional | 2021-01-21 | Recruiting |
SCHEDULE - Scandinavian Heart Transplant Everolimus de Novo Study With Early CNI Avoidance[NCT01266148] | Phase 4 | 115 participants (Actual) | Interventional | 2009-11-30 | Completed |
An Open-Label, Prospective, Randomized, Controlled, Multi-Center Study Assessing Fixed Dose Versus Concentration Controlled Cellcept® Regimens for Patients Following a Single Organ Renal Transplantation in Combination With Full Dose and Reduced Dose Calci[NCT00087581] | Phase 4 | 720 participants (Actual) | Interventional | 2004-06-30 | Completed |
Study of Tacrolimus vs Mycophenolate Mofetil in Pediatric Patients With Frequently Relapsing or Steroid Dependent Nephrotic Syndrome: a Randomized, Multicenter, Open-label, Parallel-arm Study[NCT04048161] | Phase 4 | 270 participants (Actual) | Interventional | 2019-11-12 | Completed |
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 2 | 400 participants (Anticipated) | Interventional | 2019-07-09 | Recruiting |
Mycophenolate Sodium Treatment in Patients With Primary Sjogren's Syndrome - An Open Label Pilot Trial[NCT00542763] | Phase 1 | 12 participants (Actual) | Interventional | 2005-04-30 | Completed |
Cord Blood Ex-Vivo MSC Expansion Plus Fucosylation to Enhance Homing and Engraftment[NCT03096782] | Phase 2 | 6 participants (Actual) | Interventional | 2017-10-13 | Completed |
Multitarget Therapy for Idiopathic Membranous Nephropathy[NCT04424862] | Phase 4 | 82 participants (Actual) | Interventional | 2020-06-09 | Completed |
Autologous Followed by Non-myeloablative Allogeneic Transplantation for Non-Hodgkin's Lymphoma[NCT00882895] | Phase 2 | 18 participants (Actual) | Interventional | 2009-05-05 | Active, not recruiting |
An Exploratory, Open-Label, Single Center Study to Assess the Efficacy and Dose Titration of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Reducing Donor Specific Antibody (DSA) Strength in Maintenance Kidney Transplant Recipients[NCT01044303] | Phase 4 | 32 participants (Actual) | Interventional | 2010-01-31 | Completed |
Allogeneic Hematopoietic Cell Transplantation for Patients With Nonmalignant Inherited Disorders Using a Treosulfan Based Preparative Regimen[NCT00919503] | Phase 2 | 98 participants (Actual) | Interventional | 2009-07-31 | Completed |
Nonmyeloablative BMT With Post-transplant Cyclophosphamide, Rituximab and Optimized Donor Selection for B-cell Lymphomas[NCT00946023] | Phase 2 | 135 participants (Actual) | Interventional | 2009-07-31 | Terminated(stopped due to Funding was unavailable to complete the study as originally planned.) |
5, 6 or 7 Year Follow-up Control After the SCHEDULE Study (SCANDINAVIAN HEART TRANSPLANT EVEROLIMUS DE NOVO STUDY WITH EARLY CNI AVOIDANCE)[NCT02864706] | Phase 4 | 95 participants (Actual) | Interventional | 2016-01-18 | Completed |
Conversion From MPA to Zortress (Everolimus) for GI Toxicity Post-renal Transplantation[NCT02974686] | Phase 4 | 1 participants (Actual) | Interventional | 2016-11-30 | Terminated(stopped due to low recruitment) |
Allogeneic Stem Cell Transplantation For Multiple Myeloma: A Two Step Approach To Reduce Toxicity Involving High Dose Melphalan and Autologous Stem Cell Transplant Followed By PBSC Allografting After Low Dose TBI[NCT00003954] | Phase 1/Phase 2 | 40 participants (Anticipated) | Interventional | 1999-03-31 | Completed |
Evaluation of Efficacy and Safety of Rituximab in Association With Mycophenolate Mofetil Versus Mycophenolate Mofetil Alone in Patients With Interstitial Lung Diseases (ILD) Non-responders to a First-line Immunosuppressive Treatment[NCT02990286] | Phase 3 | 122 participants (Actual) | Interventional | 2017-01-20 | Completed |
Phase II Study of IV Busulfan Combined With 12 cGy of Fractionated Total Body Irradiation (FTBI) and Etoposide (VP-16) as a Preparative Regimen for Allogeneic Bone Marrow Transplantation for Patients With Advanced Hematological Malignancies[NCT00534430] | Phase 2 | 30 participants (Actual) | Interventional | 2000-02-29 | Active, not recruiting |
A Randomized, Open Label, Phase II Multicenter Study of Non-Myeloablative Autologous Transplantation With Auto-CD34+HPC Versus Currently Available Immunosuppressive/Immunomodulatory Therapy for Treatment of Systemic Lupus Erythematosus[NCT00230035] | Phase 2 | 0 participants (Actual) | Interventional | 2005-09-30 | Withdrawn(stopped due to Recommended by DSMB due to lack of accrual) |
A One Arm, Open-label Study to Investigate the Tolerability and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Renal Transplant Recipients Who Received Mycophenolate Mofetil (MMF)[NCT00238966] | Phase 4 | 187 participants (Actual) | Interventional | 2002-11-30 | Completed |
An Open Label Study to Evaluate the Tolerability and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion (CsA-ME) in Maintenance Renal Transplant Recipients[NCT00239044] | Phase 3 | 40 participants | Interventional | 2002-12-31 | Completed |
Efficacy and Safety of Two Immunosuppressive Regimens Mycophenolate Sodium (EC-MPS) With Short-term Steroid Use or Free of Steroids Compared With a Regimen of EC-MPS With Standard Steroids in de Novo Kidney Recipients[NCT00240955] | Phase 4 | 79 participants (Actual) | Interventional | 2004-03-31 | Completed |
A 12-week Multicenter, Randomized, Open Study to Evaluate the Effects of Enteric-coated Mycophenolate Sodium (EC-MPS) in Terms of Quality of Life in Patients With Gastrointestinal (GI) Symptoms Treated With MMF (Mycophenolate Mofetil) After Kidney Transpl[NCT00400647] | Phase 4 | 136 participants (Actual) | Interventional | 2006-07-31 | Completed |
Mycophenolate for Pulmonary Sarcoidosis[NCT00262132] | Phase 3 | 20 participants | Interventional | 2003-09-30 | Terminated |
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 2 | 40 participants (Anticipated) | Interventional | 2005-02-28 | Completed |
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 2 | 100 participants (Actual) | Interventional | 2000-06-30 | Completed |
A Cyclophosphamide/Fludarabine/Total Body Irradiation Preparative Regimen for Patients With Hematological Malignancy Receiving Unrelated Donor Umbilical Cord Blood Transplantation[NCT00290641] | | 68 participants (Actual) | Interventional | 2001-04-30 | Completed |
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 2 | 75 participants (Anticipated) | Interventional | 2005-04-30 | Completed |
A Randomized Double Blinded Placebo-Controlled Phase III Trial Comparing Cyclosporine Plus Steroids With or Without Myfortic as Primary Treatment for Extensive Chronic Graft Versus Host Disease[NCT00298324] | Phase 3 | 34 participants (Actual) | Interventional | 2006-09-30 | Terminated(stopped due to Due to slow accrual) |
Mycophenolate Mofetil Versus Intravenous Cyclophosphamide Pulses in the Treatment of Crescentic IgA Nephropathy[NCT00301600] | | 40 participants (Actual) | Interventional | 2003-01-31 | Completed |
Pilot Study of Reduced-Intensity Umbilical Cord Blood Transplantation in Adult Patients With Advanced Hematopoietic Malignancies[NCT00301951] | Phase 1 | 7 participants (Actual) | Interventional | 2004-09-30 | Completed |
Mycophenolate Mofetil Versus Azathioprine for Maintenance Therapy in ANCA Associated Systemic Vasculitis[NCT00307645] | Phase 3 | 160 participants | Interventional | 2003-05-31 | Terminated |
Effect of Enteric-Coated Mycophenolate Sodium (EC-MPS) Plus Valsartan as Part of Intensified Multi-factorial Intervention Compared to EC-MPS Plus Standard Practice of Care on Development of Transplant Nephropathy in Cadaver Donor Kidney Recipients Given B[NCT00308425] | Phase 3 | 119 participants (Actual) | Interventional | 2002-10-31 | Completed |
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 2 | 170 participants (Anticipated) | Interventional | 2023-11-01 | Not yet recruiting |
A Pilot Study of Reduced Intensity HLA-Haploidentical Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide in Patients With Advanced Myelofibrosis[NCT03118492] | Phase 1 | 16 participants (Anticipated) | Interventional | 2017-05-24 | Recruiting |
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Mutations in GATA2 or the MonoMAC Syndrome[NCT01861106] | Phase 2 | 144 participants (Anticipated) | Interventional | 2013-07-24 | Recruiting |
A Phase II Trial of Total Body Irradiation Plus Metabolism-Based Cyclophosphamide Dosing as Preparative Therapy for Allogeneic Hematopoietic Cell Transplant for Patients With Hematological Malignancy[NCT00317785] | Phase 2 | 50 participants (Anticipated) | Interventional | 2005-05-31 | Completed |
An Open-Label Study to Evaluate the Effect on Quality of Life of Switching Kidney Transplant Patients From Reduced Dose EC-MPS to a Higher Than the Equimolar Dose of CellCept[NCT00420472] | Phase 4 | 0 participants | Interventional | 2007-03-31 | Terminated |
Hemophagocytic Lymphohistiocytosis[NCT00334672] | Phase 3 | 288 participants (Anticipated) | Interventional | 2006-03-31 | Active, not recruiting |
A Pilot Study to Evaluate the Co-infusion of Ex Vivo Expanded Umbilical Cord Blood Progenitors With an Unmanipulated Cord Blood Graft in Patients Undergoing Umbilical Cord Blood Transplantation for Hematologic Malignancies[NCT00343798] | Phase 1 | 23 participants (Actual) | Interventional | 2006-04-30 | Completed |
Low-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial[NCT00093743] | Phase 1 | 2 participants (Actual) | Interventional | 2000-01-31 | Completed |
A Comparison of Oral Methylprednisolone Plus Azathioprine or Mycophenolate Mofetil for the Treatment of Bullous Pemphigoid[NCT00431119] | Phase 2 | 70 participants | Interventional | 1997-10-31 | Completed |
Phase I, Open-label Study of Mycophenolate Mofetil In Systemic Sclerosis[NCT00433186] | Phase 1 | 30 participants (Anticipated) | Interventional | 2006-03-31 | Completed |
Study to Investigate the Clinical Outcomes of Different Regimens of Myfortic® in De Novo Kidney Tx Pts Using Simulect® and Neoral® With or Without Steroids[NCT00101738] | Phase 3 | 342 participants (Actual) | Interventional | 2003-03-31 | Completed |
Interruption of the Calcineurine Inhibitors (ICN) and Introduction of Mycophenolate Mofetil (MMF) in Liver Transplant Patients With Side Effects Due to ICN: Study of the Reduction of the Risks of Rejection by Mycophenolate Mofetil Therapeutic Drug Monitor[NCT00456235] | Phase 4 | 92 participants (Actual) | Interventional | 2006-09-30 | Completed |
A Randomized, Controlled, Multi-Center Study of Thymoglobulin Induction Therapy With a Calcineurin Inhibitor Sparing Regimen in Liver Transplant Patients[NCT00117689] | Phase 2 | 75 participants (Actual) | Interventional | 2005-04-30 | Completed |
A Multicenter, Randomized, Open Label, Parallel Study to Evaluate and Compare the Efficacy and Safety of FK506MR vs Prograf® in Combination With MMF and Steroids in Patients Undergoing Kidney Transplantation and a Pharmacokinetics Study.[NCT00481819] | Phase 3 | 240 participants (Actual) | Interventional | 2007-07-31 | Completed |
[NCT00120419] | Phase 4 | 90 participants (Anticipated) | Interventional | 2005-04-30 | Recruiting |
A Randomized, Open-label Study of the Effect of Replacing CNI With Sirolimus in a Standard Care Regimen of CNI, CellCept, and Steroids on Renal Function in Heart Transplant Patients[NCT00121784] | Phase 4 | 12 participants (Actual) | Interventional | 2005-10-31 | Terminated(stopped due to Poor recruitment) |
Cord Blood Expansion on Mesenchymal Stem Cells[NCT00498316] | Phase 1 | 98 participants (Actual) | Interventional | 2007-07-03 | Completed |
A Randomized, Multicenter Study to Assess the Efficacy on Diseases Activity of Enteric-coated Mycophenolate Sodium Versus Continuation of Azathioprine in Patients With Systemic Lupus Erythematosus on Azathioprine Maintenance Therapy.[NCT00504244] | Phase 3 | 12 participants (Actual) | Interventional | 2007-07-31 | Terminated(stopped due to Insufficient recruitment) |
Evaluation of the Long-term Safety and Efficacy of a Tacrolimus-based 5-day Steroid Rapid Withdrawal Immunoprophylactic Regimen in de Novo Renal Transplantation[NCT00133172] | Phase 4 | 85 participants (Actual) | Interventional | 2005-07-31 | Terminated(stopped due to Varience of supply chain from that required by protocol) |
A Multicenter, Open, Single Arm, Pilot Study to Evaluate Efficacy, Tolerability and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion and Steroids in Pediatric de Novo Renal Transplant Patients[NCT00154206] | Phase 4 | 15 participants | Interventional | 2004-09-30 | Completed |
[NCT00154245] | Phase 4 | 20 participants | Interventional | 2004-01-31 | Completed |
A European Multicenter Open-Label Randomised Trial to Evaluate the Efficacy and Safety of Sirolimus and Tacrolimus Compared to MMF and Tacrolimus With Short-Course Induction Therapy, Short-Term Steroids Application in de Novo SPK Transplanted Diabetic Pat[NCT00140543] | Phase 3 | 228 participants | Interventional | 2002-02-28 | Completed |
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 2 | 61 participants (Actual) | Interventional | 2006-11-28 | Completed |
Tacrolimus and Mycophenolate Mofetil vs Tacrolimus and Sirolimus in SPK, Pancreas After Kidney or Pancreas Transplant Alone[NCT00533442] | Phase 2 | 170 participants (Actual) | Interventional | 2000-09-30 | Completed |
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 2 | 29 participants (Actual) | Interventional | 2006-06-30 | Completed |
Reduced-intensity, Related-donor Allogeneic BMT With Fludarabine, Busulfan, and High-dose Posttransplantation Cyclophosphamide for Hematologic Malignancies[NCT01135329] | Phase 2 | 15 participants (Actual) | Interventional | 2010-08-31 | Terminated(stopped due to The stopping rule was met and hence the study was closed) |
Tandem Autologous HCT/Nonmyeloablative Allogeneic HCT From HLA-Matched Related and Unrelated Donors Followed by Bortezomib Maintenance Therapy for Patients With High-Risk Multiple Myeloma[NCT00793572] | Phase 2 | 32 participants (Actual) | Interventional | 2008-10-31 | Completed |
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 2 | 16 participants (Actual) | Interventional | 2010-03-18 | Completed |
Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders[NCT01043640] | Phase 2 | 46 participants (Actual) | Interventional | 2009-12-31 | Completed |
he Efficacy and Safety of Combining Mycophenolate Mofetil With Methimazole on Remission of Newly Diagnosis Graves' Disease (3M-RGD Trial): an Open-label, Randomized Trial[NCT06068179] | Phase 2/Phase 3 | 205 participants (Anticipated) | Interventional | 2023-10-08 | Not yet recruiting |
A 12-month, Prospective, Randomized, Dual Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of Myfortic® (Mycophenolic Acid) Loading Regimens in Combination With Thymoglobulin® [Anti-thymocyte Globulin (Rabbit)] or Simulect® (Basiliximab)[NCT01336296] | Phase 4 | 61 participants (Actual) | Interventional | 2010-09-30 | Completed |
Comparative Efficacy of Mizoribine With Mycophenolate Mofetil for Living Related Kidney Transplantation Recipients[NCT06114953] | Phase 4 | 152 participants (Anticipated) | Interventional | 2023-01-01 | Recruiting |
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in Maintenance Renal Transplant Patients[NCT00149864] | Phase 3 | 264 participants (Actual) | Interventional | 2000-02-29 | Completed |
Multicenter, Double-blind, Randomized, Parallel Group Study on Efficacy and Safety of Enteric-coated Mycophenolate Sodium vs. Mycophenolate Mofetil in de Novo Chinese Renal Transplant Recipients[NCT00149903] | Phase 3 | 300 participants | Interventional | 2005-01-31 | Completed |
Pharmacokinetics of Mycophenolate Mofetil Alone and in Combination With Valganciclovir in Renal and Heart Transplant Recipients[NCT00189150] | Phase 4 | 16 participants (Actual) | Interventional | 2005-04-30 | Completed |
A Randomized, Double-Blind Study to Evaluate the Safety of Continued Treatment With CellCept in Patients With Well-Controlled Myasthenia Gravis Receiving a Stable Dose of Prednisone[NCT00408213] | Phase 3 | 136 participants (Anticipated) | Interventional | 2004-06-30 | Completed |
Effects of Mycophenolate Mofetil (MMF) on Surrogate Markers for Cardiovascular Disease in HIV-1 Infected Patients[NCT00247494] | Phase 4 | 90 participants (Anticipated) | Interventional | 2005-04-30 | Recruiting |
A Dose Finding Study of Total Body Irradiation for Conditioning Patients With Severe Aplastic Anemia Transplanted With Umbilical Cord Blood[NCT00354419] | Phase 1 | 30 participants (Anticipated) | Interventional | 2006-02-28 | Terminated(stopped due to Low accrual) |
Efficacy and Safety of Maintenance Neoral Compared to Bitherapy Neoral-Imurel or Neoral-CellCept in Renal Transplantation[NCT00461825] | Phase 3 | 207 participants | Interventional | 1998-07-31 | Completed |
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study in Healthy, Adult,Human, Male Subjects Under Fasting Conditions.[NCT01513044] | Phase 1 | 68 participants (Actual) | Interventional | 2009-01-31 | Completed |
An Open-label, Balanced, Randomized, Two-treatment, Two-period, Two Sequence, Single Dose, Crossover, Bioequivalence Study in Healthy, Adult,Human, Male Subjects Under Fed Conditions.[NCT01513057] | Phase 1 | 61 participants (Actual) | Interventional | 2009-01-31 | Completed |
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 2 | 70 participants (Anticipated) | Interventional | 2020-02-07 | Recruiting |
Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders[NCT00053989] | Phase 2 | 41 participants (Actual) | Interventional | 2002-01-29 | Completed |
Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies After a Non-Myeloablative Conditioning Regimen From HLA-Matched Sibling Donors[NCT00006350] | Phase 2 | 0 participants | Interventional | 2000-01-31 | Completed |
Comparative Study of the Efficacy of Induction Therapy With Cyclophosphamide or Mycophenolate Mofetil for Non-Life-Threatening Relapses of PR3- or MPO-ANCA Associated Vasculitis[NCT00103792] | Phase 3 | 90 participants (Anticipated) | Interventional | 2004-12-31 | Recruiting |
An Open Label Study to Evaluate the Effect of CellCept in Combination With Cyclosporine A and Steroids on Renal Function and the Prevention of Acute Rejection in Heart Transplant Patients.[NCT02091414] | Phase 3 | 36 participants (Actual) | Interventional | 2006-08-31 | Completed |
A Dose-Blinded, 2-Dose Level, Parallel-Group, Multicenter, Long-Term Extension Study to Evaluate the Long-Term Safety, Efficacy, and Immunogenicity of BIIB023 in Subjects With Lupus Nephritis[NCT01930890] | Phase 2 | 87 participants (Actual) | Interventional | 2013-11-30 | Terminated(stopped due to Results from pre-specified criteria in study NCT01499355 (211LE201) did not demonstrate sufficient efficacy to warrant continuation of the study) |
A Phase I Study Combining Escalating Doses of Radiolabeled BC8 (Anti-CD45) Antibody With Fludarabine, Low Dose TBI, PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to Establish Mixed or Full Donor Chimerism [NCT00008177] | Phase 1 | 79 participants (Actual) | Interventional | 1999-07-27 | Completed |
Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies[NCT00281879] | Phase 2 | 200 participants (Actual) | Interventional | 2006-02-28 | Terminated |
Induction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil[NCT00008450] | Phase 1 | 6 participants (Actual) | Interventional | 1997-08-11 | Completed |
Pharmacokinetics of Mycophenolic Acid and Mycophenolate 7-O-Phenolic Glucuronide in Healthy Subjects With or Without Two Common Genetic Polymorphisms in the Promoter Region of Uridine Diphosphate Glucuronosyltransferase 1A9[NCT00128947] | Phase 1 | 130 participants | Interventional | 2005-07-31 | Completed |
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 2 | 0 participants (Actual) | Interventional | 2001-06-30 | Withdrawn |
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 2 | 0 participants | Interventional | 2001-07-31 | Completed |
Sibling Donor Cord Blood Banking and Transplantation[NCT00029380] | Phase 2 | 30 participants (Anticipated) | Interventional | 1999-01-31 | Completed |
Nonmyeloablative PBSC Allografting From HLA Matched Related Donors Using Fludarabine and/or Low Dose TBI With Disease-Risk Based Immunosuppression[NCT00014235] | | 160 participants (Anticipated) | Interventional | 2000-12-31 | Completed |
A Phase I/II Randomized, Double-Blind, Placebo-Controlled Pilot Study of Beta-D-2,6-diaminopurine Dioxolane (DAPD) Versus DAPD Plus Mycophenolate Mofetil (MMF) in Treatment-Experienced Subjects[NCT00038272] | Phase 2 | 56 participants | Interventional | | Completed |
Treatment of Patients With Membranous Nephropathy and Renal Insufficiency With Mycophenolate Mofetil and Prednisone: a Pilot Study[NCT00135967] | Phase 2/Phase 3 | 30 participants | Interventional | 2002-05-31 | Completed |
Randomised, Double-Arm, Controlled, Open-Label Study Comparing Calcineurin Inhibitor-Free Immunosuppression (Zenapax®, CellCept® and Prednisolone) and Cyclosporine A Based Immunosuppression (Sandimmun Neoral®, CellCept® and Prednisolone) on the Outcome of[NCT00138970] | Phase 4 | 70 participants | Interventional | 2002-01-31 | Completed |
A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and Corticosteroid With Tacrolimus and Corticosteroid as Immunosuppressive Treatment for Lupus Membra[NCT00404794] | Phase 3 | 20 participants (Anticipated) | Interventional | 2005-11-30 | Completed |
A Prospective Randomized Open-label Study to Compare Mycophenolate Mofetil and Corticosteroid With Conventional Immunosuppressive Treatment on Proteinuria in Idiopathic Membranous Nephropathy (MN) and Focal Segmental Glomerulosclerosis (FSGS)[NCT00404833] | Phase 3 | 16 participants (Anticipated) | Interventional | 2003-01-31 | Completed |
[NCT00010361] | | 20 participants | Interventional | 2000-11-30 | Completed |
A Prospective, Randomized Trial of Calcineurin-Inhibitor Withdrawal in Renal Allograft Recipients[NCT00275535] | Phase 4 | 165 participants (Actual) | Interventional | 2001-04-30 | Completed |
A Phase I/II Study of the Safety, Tolerability, and Antiretroviral Activity of Mycophenolate Mofetil As an Adjunct to Abacavir Therapy in HIV-Infected Subjects With Treatment Failure and Extensive Prior Antiretroviral Exposure[NCT00021489] | Phase 2 | 0 participants (Actual) | Interventional | | Withdrawn |
Allogeneic Hematopoietic Cell Transplantation From HLA-matched Donors After Reduced-intensity Conditioning: a Phase II Randomized Study Comparing 2 GVHD Prophylaxis Regimens[NCT01428973] | Phase 2 | 200 participants (Actual) | Interventional | 2011-09-30 | Active, not recruiting |
Induction Therapy for Lupus Nephritis With no Added Oral Steroids: An Open Label Randomised Multicentre Controlled Trial Comparing Oral Corticosteroids Plus Mycophenolate Mofetil (MMF) Versus Obinutuzumab and MMF[NCT04702256] | Phase 3 | 196 participants (Anticipated) | Interventional | 2021-12-09 | Recruiting |
Calcineurin Inhibitor Sparing Protocol in Living Donor Pediatric Kidney Transplantation[NCT00023231] | | 35 participants (Actual) | Interventional | 2001-02-28 | Completed |
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 2 | 30 participants (Anticipated) | Interventional | 2001-09-30 | Completed |
Pharmacokinetic Evaluation of Plasmapheresis in Cross Match Positive or ABO Incompatible Kidney Allograft Recipients[NCT00203281] | | 0 participants (Actual) | Observational | 2003-02-28 | Withdrawn(stopped due to funding withdrawn) |
A Randomized Multicenter Trial Comparing Mycophenolate Mofetil and Azathioprine as Remission-maintaining Treatment for Proliferative Lupus Glomerulonephritis. The MAINTAIN Nephritis Trial.[NCT00204022] | Phase 3 | 105 participants (Actual) | Interventional | 2001-02-28 | Completed |
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 2 | 10 participants (Actual) | Interventional | 2001-10-31 | Terminated(stopped due to Poor accrual and difficulty with multicenter logistics) |
Prolonged Mycophenolate Mofetil and Truncated Cyclosporine Postgrafting Immunosuppression to Reduce Life-Threatening GVHD After Unrelated Donor Peripheral Blood Cell Transplantation Using Nonmyeloablative Conditioning for Patients With Hematologic Maligna[NCT00078858] | Phase 1/Phase 2 | 37 participants (Actual) | Interventional | 2003-09-30 | Completed |
Low Dose Total-Body Irradiation And Fludarabine Followed By HLA Matched Allogeneic Stem Cell Transplantation For Hematologic Malgnancies - A Multi-Center Study[NCT00044954] | Phase 2 | 0 participants | Interventional | 1999-11-30 | Completed |
A Randomized, Open-label Study Comparing the Effects of Low-dose Cyclosporine vs Cyclosporine Withdrawal on Renal Function in Kidney Transplant Patients Treated With CellCept and Daclizumab[NCT00048152] | Phase 3 | 539 participants (Actual) | Interventional | 2000-12-31 | Completed |
Lowering Total Immunosuppressive Load in Renal Transplant Recipients More Than 12 Months Posttransplant - Randomised Withdrawal of Mycophenolate Mofetil (CellCept®) or Cyclosporine A (Sandimmun Neoral®)[NCT00148252] | Phase 4 | 298 participants | Interventional | 2003-02-28 | Terminated(stopped due to To high rejection rate in CsA withdrawal arm) |
Multicenter, Open-label Follow-up Study on the Safety of Enteric-coated Mycophenolate Sodium in de Novo Renal Transplant Patients[NCT00149929] | Phase 3 | 246 participants (Actual) | Interventional | 1999-12-31 | Completed |
Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Renal Transplant Recipients (MyLife)[NCT00149968] | Phase 4 | 196 participants (Actual) | Interventional | 2005-04-30 | Completed |
Measurement of Patient Reported Outcomes in Renal Transplant Patients With and Without Gastrointestinal (GI) Symptoms (PROGIS)[NCT00150007] | Phase 4 | 335 participants | Interventional | 2004-06-30 | Completed |
Prospective Randomized Study to Characterize Risk Factors of Polyomavirus-related Transplant Nephropathy and the Impact of Three Immunosuppressive Regimens on Nephropathy Incidence[NCT00160966] | Phase 4 | 108 participants (Actual) | Interventional | 2004-09-30 | Completed |
The Relationships Between Mycophenolic Acid Levels, T-Cell Subsets and Outcomes in Pediatric Heat Transplant Recipients Receiving Mycophenolate Mofetil (Cellcept)[NCT00166153] | | 30 participants | Interventional | 2003-01-31 | Terminated |
Open-label Study to Evaluate the Tolerability, Safety and Efficacy of the Equimolar Conversion From Mycophenolate Mofetil (MMF) to Enteric-Coated Mycophenolate Sodium (EC-MPS) in Patients With Renal Transplant[NCT00171379] | Phase 3 | 162 participants (Actual) | Interventional | 2004-03-31 | Completed |
Pilot Trial for Implementation of a MPA PK Monitoring Strategy[NCT00187915] | | 24 participants (Actual) | Interventional | 2003-07-31 | Completed |
Comparison Of Efficacy Of Two Immunosuppressive Protocols Including Tacrolimus With Or Without Mycophenolate Mofetil In Pediatric Liver Transplantation Aimed In Early Termination Of Steroid Therapy[NCT00195988] | Phase 4 | 40 participants | Interventional | 2002-09-30 | Completed |
A Prospective, Randomized, Open, Multicentric Study Intended to Evaluate the Efficacy and Tolerability of Sequential Treatment Based on Rabbit Anti-T-lymphocyte Serum, of Mycophenolate Mofetil and of Cyclosporin, Without Concomitant Corticosteroids, After[NCT00200551] | Phase 4 | 200 participants | Interventional | 2001-01-31 | Completed |
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 2 | 0 participants (Actual) | Interventional | 2006-05-31 | Withdrawn(stopped due to Funding cut, no patients enrolled) |
Post Transplant Infusion of Allogeneic Cytokine Induced Killer Cells as Consolidative Therapy After Non-Myeloablative Allogeneic Transplantation in Patients With Myelodysplasia or Myeloproliferative Disorders[NCT01392989] | Phase 2 | 44 participants (Actual) | Interventional | 2011-03-31 | Completed |
A Phase II Study of the Combination of Tacrolimus and Mycophenolate Mofetil for the Prevention of Acute Graft-Versus-Host Disease[NCT00096096] | Phase 2 | 0 participants | Interventional | 2004-08-31 | Completed |
A Prospective Study on the Tolerability and Efficacy of the de Novo Use of Myfortic in Liver Transplant Recipients[NCT00336817] | | 30 participants (Actual) | Interventional | 2006-11-30 | Completed |
Pulmonary Involvement in Scleroderma: Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients[NCT00333437] | | 7 participants (Actual) | Interventional | 2006-05-31 | Completed |
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 2 | 77 participants (Actual) | Interventional | 2006-01-31 | Completed |
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 2 | 14 participants (Actual) | Interventional | 2006-05-24 | Terminated(stopped due to Low accrual) |
Safety of Tacrolimus And Methotrexate (MTX) Versus Tacrolimus And Mycophenolate Mofetil (MMF) As Graft Versus Host Disease Prophylaxis In Allogeneic Hematopoietic Cell Transplants (HCT)[NCT00360685] | | 89 participants (Actual) | Interventional | 2005-09-30 | Completed |
A 12-month, Prospective, Randomized, Single Center, Open Label Pilot Study to Evaluate the Safety and Efficacy of Myfortic in Combination With Tacrolimus and Thymoglobulin in Early Corticosteroid Withdrawal[NCT00374803] | Phase 4 | 45 participants (Actual) | Interventional | 2006-04-30 | Completed |
A Single Center, Pilot Study of Corticosteroid Discontinuation in Liver Transplant Recipients Utilizing a Tacrolimus/Mycophenolate Mofetil Based Maintenance Immunosuppression Protocol[NCT00374231] | Phase 4 | 40 participants (Actual) | Interventional | 2002-10-31 | Completed |
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 2 | 17 participants (Actual) | Interventional | 2006-07-31 | Completed |
A Two Step Approach To Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies From HLA Partially-Matched Related Donors[NCT00429143] | Phase 1/Phase 2 | 27 participants (Actual) | Interventional | 2006-01-31 | Completed |
A California Cooperative Clinical Study Comparing Allogeneic Hematopoietic Cell Transplantation Using Nonmyeloablative Host Conditioning With Total Lymphoid Irradiation and Anti-thymocyte Globulin Versus Best Standard of Care in Acute Myeloid Leukemia (AM[NCT00568633] | Phase 3 | 58 participants (Actual) | Interventional | 2007-08-31 | Terminated(stopped due to Poor accrual) |
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Anemia and Other Hemoglobinopathies[NCT00489281] | Phase 2 | 43 participants (Actual) | Interventional | 2008-06-23 | Terminated(stopped due to Initiation of CMS BMT study for sickle-cell disease operating under NCT01166009 made further accrual to this study impossible.) |
Evaluation of Belatacept as First Line Immunosuppression in De Novo Liver Transplant Recipients[NCT00555321] | Phase 2 | 260 participants (Actual) | Interventional | 2008-01-31 | Terminated |
Clinical Outcome of Delayed or Standard Prograf Together With Induction Therapy Followed by Conversion to Advagraf in Donation After Cardiac (or Circulatory) Death (DCD) Kidney Transplant Recipients: A Randomized, Open-label, Multicenter Clinical Trial[NCT03644485] | Phase 4 | 284 participants (Actual) | Interventional | 2018-10-21 | Completed |
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 2 | 31 participants (Actual) | Interventional | 2000-02-17 | Completed |
Phase 0/I Dose Escalation Study of Mycophenolate Mofetil Combined With Radiation Therapy in Glioblastoma[NCT04477200] | Phase 1 | 68 participants (Anticipated) | Interventional | 2020-08-05 | Recruiting |
Efficacy of Chimeric Monoclonal Anti-CD20 Antibodies (Rituximab Biosimilar) in the Treatment of Childhood Steroid-dependent Nephrotic Syndrome and Development of Cell Biomarkers Predicting Outcome. The RTX 4 Trial.[NCT04402580] | Phase 2 | 30 participants (Actual) | Interventional | 2019-07-01 | Terminated(stopped due to due to unexpectedly high rate of relapse in the active comparator arm.) |
Allogeneic Hematopoietic Cell Transplantation After Nonmyeloablative Conditioning for Patients With Severe Systemic Sclerosis[NCT00622895] | Phase 1/Phase 2 | 3 participants (Actual) | Interventional | 2006-09-01 | Completed |
Multicenter, Open-Label, Randomized Study on Steroid-Free Immunosuppression, in Comparison With Daily Steroid Therapy, in Pediatric Renal Transplant : Impact on Growth, Bone Metabolism and Acute Rejection[NCT00707759] | Phase 3 | 28 participants (Actual) | Interventional | 2008-06-30 | Completed |
Optimization of NULOJIX® (Belatacept) Usage as a Means of Avoiding CNI and Steroids in Renal Transplantation (CTOT-10)[NCT01436305] | Phase 2 | 19 participants (Actual) | Interventional | 2011-09-30 | Terminated(stopped due to Secondary to safety concerns plus change in Campath® (alemtuzumab) availability.) |
Comparing and Combining Bortezomib and Mycophenolate in SSc Pulmonary Fibrosis Grant Number: R34HL122558[NCT02370693] | Phase 2 | 9 participants (Actual) | Interventional | 2016-03-31 | Completed |
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 2 | 5 participants (Actual) | Interventional | 2006-06-30 | Terminated(stopped due to Unacceptable morbidity & mortality) |
A Randomized Phase II Study to Compare the Net Clinical Benefit of Cyclosporine and Sirolimus Combined With MMF or Post-Transplant Cyclophosphamide as GVHD Prophylaxis After HLA-Matched or HLA-Mismatched Unrelated G-CSF Mobilized Blood Cell Transplantatio[NCT03246906] | Phase 2 | 160 participants (Anticipated) | Interventional | 2017-09-11 | Recruiting |
The Effects of Mycophenolate Mofetil on Renal Outcomes in Patients With Advanced IgA Nephropathy: a Randomized Open-label Study[NCT01854814] | | 238 participants (Actual) | Interventional | 2013-07-31 | Completed |
"A Feasibility Study of Early Allogeneic Hematopoietic Cell Transplantation for Relapsed or Refractory High-Grade Myeloid Neoplasms"[NCT02756572] | Phase 2 | 30 participants (Actual) | Interventional | 2016-09-22 | Completed |
Randomized, Open Label, Multicenter Study of Belatacept-based Early Steroid Withdrawal Regimen With Alemtuzumab or rATG Induction Compared to Tacrolimus-based Early Steroid Withdrawal Regimen With rATG Induction in Renal Transplantation[NCT01729494] | Phase 4 | 316 participants (Actual) | Interventional | 2012-09-30 | Completed |
A 60 Month, Single-arm, Proof-of-concept Study to Induce Allogeneic Tolerance in Deceased Donor Liver Transplant Recipients Using Siplizumab, an Anti-CD2 Antibody in Combination With Cyclophosphamide and Splenectomy[NCT06019507] | Phase 2 | 12 participants (Anticipated) | Interventional | 2022-06-29 | Recruiting |
A Phase II Trial of De-escalated PTCy and Ruxolitinib for GVHD Prophylaxis in Patients Undergoing Reduced Intensity Conditioning Allogeneic HCT[NCT05622318] | Phase 2 | 56 participants (Anticipated) | Interventional | 2023-08-29 | Recruiting |
A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Subjects With VEXAS (Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic) Syndrome[NCT05027945] | Phase 2 | 37 participants (Anticipated) | Interventional | 2023-02-23 | Recruiting |
Peritransplant Ruxolitinib for Patients With Primary and Secondary Myelofibrosis[NCT04384692] | Phase 2 | 45 participants (Anticipated) | Interventional | 2020-12-18 | Recruiting |
Randomized Double-Blind Placebo-Controlled Clinical Trial to Assess the Efficacy of Mycophenolate Mofetil in Subclinical Interstitial Lung Disease Associated With Systemic Sclerosis: a Feasibility Study[NCT05785065] | Phase 2 | 35 participants (Anticipated) | Interventional | 2023-12-31 | Not yet recruiting |
A Randomized, Placebo-controlled, Double-blind, Multicenter Study Investigating Basiliximab in Combination With MMF, Cyclosporine Microemulsion and Prednisone in the Prevention of Acute Rejection in Pediatric Renal Allograft Recipients[NCT00228020] | Phase 3 | 212 participants (Actual) | Interventional | 2001-05-31 | Completed |
APOMYGRE : Multicenter, Randomized, Open-Label Study of MMF Therapeutic Follow-up's Interest in the the 12 First Months in Kidney Transplantation[NCT00199667] | Phase 4 | 137 participants | Interventional | 2002-10-31 | Active, not recruiting |
A Randomised Clinical Trial of Mycophenolate Mofetil Versus Cyclophosphamide for Remission Induction in ANCA Associated Vasculitis.[NCT00414128] | Phase 2/Phase 3 | 140 participants (Actual) | Interventional | 2007-03-31 | Completed |
A One-year, Randomized, Open Label, Parallel Group Study to Investigate the Safety and the Effect of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Either Full Dose or Reduced Dose Cyclosporine Microemulsion in de Novo Kidney Transplant [NCT00238940] | Phase 3 | 55 participants (Actual) | Interventional | 2003-02-28 | Completed |
A 6-month, 1-arm, Open-label Study to Investigate the Tolerability and Safety of Converting Stable Renal Transplant Recipients Who Receive Tacrolimus With or Without Corticosteroids From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium ([NCT00238979] | Phase 4 | 50 participants | Interventional | 2003-01-31 | Completed |
A Prospective, Open-label, Multicenter, Follow-up Study on the Efficacy and Safety of Enteric-coated Mycophenolate Sodium Administered in de Novo Kidney Transplant Patients[NCT00239018] | Phase 4 | 144 participants (Actual) | Interventional | 2003-04-30 | Completed |
Enteric-Coated Mycophenolate Sodium (EC-MPS) Administration in Maintenance Renal Transplant Patients Receiving Cyclosporine Microemulsion (CsA-ME) and Steroids, for the Withdrawal of Concomitant Steroid Therapy: a Prospective, Open-label, Exploratory Stud[NCT00239057] | Phase 3 | 23 participants (Actual) | Interventional | 2002-05-31 | Completed |
[NCT01680952] | Phase 4 | 158 participants (Actual) | Interventional | 2012-09-30 | Completed |
Development of Population Pharmacokinetic-Pharmacodynamic (PK-PD) Models of Mycophenolic Acid for Bayesian Dose Individualization in Pediatric Kidney Transplant Patients[NCT00281619] | | 29 participants (Actual) | Observational | 2006-01-31 | Completed |
Nonmyeloablative Bone Marrow Transplants in Hematologic Malignancies: Dose Finding Study for Post-Transplant Immunosuppression[NCT00255710] | Phase 1 | 60 participants (Anticipated) | Interventional | 2002-07-31 | Completed |
Transplantation of Umbilical Cord Blood From Related and Unrelated Donors[NCT00290628] | | 43 participants (Actual) | Interventional | 1999-10-31 | Terminated(stopped due to Replaced with another study) |
An Open, Prospective Study to Assess the Efficacy and Safety of FK506 Combined MMF in the Treatment of Class III,IV,V + IV or V + III Lupus Nephritis[NCT00298506] | | 120 participants (Actual) | Interventional | 2005-09-30 | Completed |
Non-Myeloablative HLA-Matched Sibling Allogeneic Peripheral Blood Stem Cell Transplantation for Metastatic Renal Cell Carcinoma[NCT00262886] | Phase 2 | 35 participants (Anticipated) | Interventional | 2001-08-31 | Completed |
Open, Multicenter, Randomized Clinical Trial in Patients With Moderate-Severe Psoriasis (PASI > 10) to Compare the Efficacy of Mycophenolate Mofetil Versus Cyclosporine A.[NCT00295425] | Phase 2 | 50 participants | Interventional | 2000-05-31 | Active, not recruiting |
Treatment of Necrotizing Vasculitides for Patients Older Than 65 Years Comparison of Two Strategies Combining Steroids With or Without Immunosuppressants[NCT00307671] | Phase 4 | 108 participants (Actual) | Interventional | 2005-07-31 | Completed |
A Twelve-month, Randomized, Multicenter, Open-label, Exploratory Study to Investigate the Clinical Outcomes of an Immunosuppressive Regimen of Basiliximab, Cyclosporine Microemulsion (CsA-ME) and Enteric-coated Mycophenolate Sodium (EC-MPS) Free of Steroi[NCT00284921] | Phase 3 | 60 participants | Interventional | 2004-04-30 | Terminated |
A Trial of Mycophenolate Mofetil in Myasthenia Gravis[NCT00285350] | Phase 3 | 80 participants | Interventional | 2002-09-30 | Completed |
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) | Interventional | 2005-05-31 | Active, not recruiting |
An Open, Randomised, Multicentre Clinical Study to Investigate the Safety and Efficacy of Steroid Withdrawal With Tacrolimus, Mycophenolate Mofetil and Daclizumab Against Tacrolimus, Mycophenolate Mofetil and Steroids in Children After Kidney Transplantat[NCT00296348] | Phase 3 | 198 participants (Actual) | Interventional | 2005-11-30 | Completed |
Desensitization of Renal Transplant Candidates[NCT00298883] | Phase 1 | 9 participants (Actual) | Interventional | 2006-02-28 | Completed |
A Prospective, Randomized, Open-label Study Evaluating the Efficacy of Mycophenolate Mofetil in the Prevention of Relapse of Steroid Dependent Nephrotic Syndrome in Children[NCT01895894] | Phase 4 | 34 participants (Actual) | Interventional | 2013-09-30 | Completed |
MMF Versus Intravenous CTX Pulses in the Treatment of Adult Severe Henoch-Schonlein Purpura Nephritis[NCT00301613] | | 60 participants (Actual) | Interventional | 2003-01-31 | Completed |
Mycophenolate Mofetil Versus Cyclophosphamide in the Induction Treatment of ANCA Associated Vasculitis[NCT00301652] | | 60 participants (Actual) | Interventional | 2003-06-30 | Completed |
Safety and Efficacy of Low-dose Cyclosporine in Association With Everolimus to Minimize Renal Dysfunction in Heart Transplant Recipients[NCT00420537] | Phase 4 | 34 participants (Actual) | Interventional | 2006-09-30 | Terminated(stopped due to A cluster of adverse events in everolimus arm was noted.) |
A Prospective, Open Label, Multicenter Study to Assess the Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients[NCT00312143] | Phase 4 | 35 participants (Actual) | Interventional | 2004-02-29 | Completed |
Myfortic® Monotherapy to Prevention of de Novo Allosensitization in Islet Transplant Recipients Following Complete Graft Loss[NCT01999361] | | 18 participants (Anticipated) | Interventional | 2009-01-31 | Recruiting |
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 1 | 15 participants (Actual) | Interventional | 2012-01-09 | Completed |
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders[NCT04644016] | Phase 2 | 31 participants (Anticipated) | Interventional | 2020-11-20 | 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 2 | 10 participants (Anticipated) | Interventional | 2012-01-31 | Recruiting |
Hematopoietic Stem Cell Transplantation Using Alternate Donor Umbilical Cord Blood Options[NCT01652014] | Phase 2 | 0 participants (Actual) | Interventional | 2014-01-31 | Withdrawn(stopped due to Funding unavailable) |
Immune Tolerance and Alloreactivity in Liver Transplant Recipients on Different Monotherapy Immunosuppressive Agents[NCT01678937] | | 31 participants (Actual) | Observational | 2007-09-30 | Completed |
A Prospective, Randomised, Double-blind, Placebo-controlled, Parallel Group, Mult-center, 52-week Trial to Assess the Efficacy and Safety of Adjunct Mycophenolate Mofetil (MMF) to Achieve Remission With Reduced Corticosteroid in Subjects With Pemphigus Vu[NCT00683930] | Phase 3 | 96 participants (Actual) | Interventional | 2004-05-31 | Completed |
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 2 | 41 participants (Actual) | Interventional | 2008-10-13 | Completed |
Phase I/II Trial of Thymus Transplantation With Immunosuppression, #950[NCT00579527] | Phase 1/Phase 2 | 14 participants (Actual) | Interventional | 2005-12-19 | Completed |
[NCT01706471] | Phase 4 | 60 participants (Actual) | Interventional | 2009-06-30 | Completed |
A Phase 1, Open-Label, Sequential Study of the Effect of Multiple Doses of Isavuconazole on the Pharmacokinetics of a Single Dose of Mycophenolate Mofetil in Healthy Adult Subjects[NCT01711489] | Phase 1 | 24 participants (Actual) | Interventional | 2012-03-31 | Completed |
A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of BMS-188667 (Abatacept) or Placebo on a Background of Mycophenolate Mofetil and Corticosteroids in the Treatment of Subjects With Active Class III or IV Lup[NCT01714817] | Phase 3 | 695 participants (Actual) | Interventional | 2013-01-22 | Terminated(stopped due to Inability to meet protocol objectives.) |
Multicenter, Open-label, Parallel Clinical Investigation of the Safety and Efficacy of Advagraf® (Extended Release Tacrolimus) vs. Prograf® (Tacrolimus) in de Novo Kidney Recipients 1 Month After Kidney Transplantation[NCT01742624] | Phase 4 | 60 participants (Actual) | Interventional | 2012-04-30 | Completed |
A Multi-center, Open, Randomized-control Study to Compare the Effects and Safety of Idarubicin-strengthened Pretreatment Program and Conventional Busulfan Cyclophosphamide Pretreatment Program on High-risk Acute Myeloid Leukemia Patient[NCT01766375] | Phase 3 | 200 participants (Anticipated) | Interventional | 2012-08-31 | Recruiting |
Open Label, Multicenter, Non-comparative Study to Evaluate the Efficacy and Safety of MY-REPT Capsule in Primary, Liver Transplantation Recipients.[NCT01766518] | Phase 4 | 120 participants (Anticipated) | Interventional | 2009-11-30 | Recruiting |
A Single-Dose, Replicate, Comparative Bioavailability Study of Two Formulations of Mycophenolate Mofetil 500 mg Tablets Under Fasting Conditions[NCT00907907] | Phase 1 | 40 participants (Actual) | Interventional | 2006-07-31 | Completed |
Phase 3 Open Label Randomised Multicentre Controlled Trial of Rituxmab and Mycophenolate Mofetil Without Oral Steroids for the Treatment of Lupus Nephritis[NCT01773616] | Phase 3 | 24 participants (Actual) | Interventional | 2015-04-30 | Terminated(stopped due to Study assessments for patients recruited continuing per protocol so patients receive a minimum of 6 months follow up. No safety concerns have been raised.) |
A Phase II Study Evaluating Selective Depletion of CD45RA+ T Cells From Allogeneic Peripheral Blood Stem Cell Grafts From HLA-Matched Related and Unrelated Donors for Prevention of GVHD[NCT02220985] | Phase 2 | 84 participants (Actual) | Interventional | 2015-02-03 | Active, not recruiting |
Prospective Evaluation of the Efficacy and Safety of Zortress (Everolimus)/Myfortic (Enteric Coated Mycophenolate Sodium) Conversion in High MELD Liver Transplantation[NCT01807767] | | 0 participants (Actual) | Interventional | 2013-03-31 | Withdrawn(stopped due to funding was withdrawn) |
Kidney Graft Function Under the Immunosuppression Strategies With Low Dose of Neoral®(Cyclosporine) and Standard Dose of Myfortic®(Enteric-Coated Mycophenolate Sodium) vs. With Conventional Dose of Neoral®(Cyclosporine) and Reduced Dose of Myfortic®(Enter[NCT01817322] | Phase 4 | 140 participants (Actual) | Interventional | 2011-06-30 | Completed |
A Prospective Study to Investigate Mycophenolic Acid (MPA) Exposure Through Area Under the Curve (AUC) in Renal Transplants Recipients Treated With Mycophenolate Mofetil (MMF) and After Conversion to Mycophenolate Sodium (EC-MPS)[NCT01822483] | Phase 4 | 100 participants (Actual) | Interventional | 2013-04-30 | Completed |
Open Label, Randomized, Multi-center, Phase 4 Trial to Evaluate the Efficacy and Safety of My-Rept® Tablet(Mycophenolate Mofetil 500mg/Tab.) Versus My-Rept® Capsule(Mycophenolate Mofetil 250mg/Cap.) in Combination With Tacrolimus for 26 Weeks in Kidney Tr[NCT01842269] | Phase 4 | 156 participants (Actual) | Interventional | 2013-01-31 | Completed |
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 3 | 174 participants (Actual) | Interventional | 2010-11-30 | Completed |
A Two Step Approach to Haploidentical Hematopoietic Stem Cell Transplantation for Patients in Remission From HLA Partially-Matched Related Donors-Effect of Maternal Donors on Outcomes[NCT01871441] | Phase 2 | 4 participants (Actual) | Interventional | 2013-05-17 | Terminated(stopped due to Trial was closed due to poor accrual.) |
To Compare the Efficacy of Tacrolimus and Mycophenolate Mofetil for the Initial Therapy of Active Lupus Nephritis[NCT00371319] | Phase 4 | 150 participants (Actual) | Interventional | 2005-09-30 | Completed |
An Open Label Study of the Effects of a Combination of NeoRecormon, CellCept and Prednisone on Hematological Parameters and Cytogenesis in Patients With Low or Intermediate Risk Myelodysplastic Syndromes.¿[NCT00551291] | Phase 2 | 10 participants (Actual) | Interventional | 2007-08-31 | Completed |
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) | Interventional | 2013-05-31 | Active, not recruiting |
Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)[NCT01626092] | | 3 participants (Actual) | Interventional | 2012-07-11 | Completed |
Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation as Second Transplantation for Patients With Disease Relapse or Myelodysplasia After Prior Autologous Transplantation[NCT01118013] | Phase 2 | 6 participants (Actual) | Interventional | 2010-12-31 | Terminated |
A Phase II, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Obinutuzumab in Adolescent Patients With Active Class III or IV Lupus Nephritis, Including an Evaluation of Open Label Sa[NCT05039619] | Phase 2 | 40 participants (Anticipated) | Interventional | 2022-05-12 | Recruiting |
Haploidentical Donor Hematopoietic Progenitor Cell and Natural Killer Cell Transplantation With a TLI Based Conditioning Regimen in Patients With Hematologic Malignancies[NCT01807611] | Phase 2 | 82 participants (Actual) | Interventional | 2013-05-16 | Completed |
A Randomized, Multicenter, Open-label Phase II Trial to Compare Prophylaxis of Graft Versus Host Disease With Tacrolimus and Mycophenolate Mofetil Versus Ruxolitinib After Post-transplant Cyclophosphamide[NCT04669210] | Phase 2 | 128 participants (Actual) | Interventional | 2020-11-03 | Active, not recruiting |
A Phase Ib/2 Trial of Fludarabine/Melphalan/Total Body Irradiation With Post Transplant Cyclophosphamide as Graft Versus Host Disease Prophylaxis in Matched-Related and Matched-Unrelated Allogeneic Hematopoietic Cell Transplantation[NCT03192397] | Phase 1/Phase 2 | 35 participants (Actual) | Interventional | 2017-08-09 | Active, not recruiting |
T-Cell Replete Haploidentical Donor Hematopoietic Stem Cell Plus Natural Killer (NK) Cell Transplantation in Patients With Hematologic Malignancies Relapsed or Refractory Despite Previous Allogeneic Transplant[NCT01621477] | Phase 2 | 34 participants (Actual) | Interventional | 2012-08-31 | Terminated(stopped due to Study was terminated in August 2016 due to replacement by a new study.) |
Once Daily Dosing to Improve Medication Adherence and Patient Satisfaction in Kidney Transplant Recipients: A Pilot Study[NCT02426502] | | 76 participants (Actual) | Interventional | 2016-04-30 | Active, not recruiting |
Phase 1 Pilot Study Using Autologous CD4+CD25+FoxP3+ T Regulatory Cells and Campath-1H to Induce Renal Transplant Tolerance[NCT01446484] | Phase 1/Phase 2 | 30 participants (Anticipated) | Interventional | 2011-10-31 | Recruiting |
A Randomised Prospective Open Label Pilot Trial Comparing Mycophenolate Mofetil (MMF) With no Immunosuppression in Adults With Limited Cutaneous Systemic Sclerosis[NCT04927390] | Phase 2 | 120 participants (Anticipated) | Interventional | 2021-12-08 | Active, not recruiting |
A Phase II Trial of Allogeneic Peripheral Blood Stem Cell Transplantation From Matched Unrelated Donors in Patients With Advanced Hematologic Malignancies and Hematological Disorders[NCT00544115] | Phase 2 | 260 participants (Actual) | Interventional | 2001-10-16 | Active, not recruiting |
An Open-label Study of the Pharmacokinetics of Mycophenolic Acid as Myfortic (Enteric-coated Mycophenolate Sodium) When Used in Combination With Prograf (Tacrolimus) and Corticosteroids in Patients Undergoing de Novo Liver Transplantation[NCT01467011] | | 25 participants (Actual) | Observational | 2010-12-31 | Completed |
Investigating the Links Between Microbiota Composition and Variability Observed in the Pharmacological Response to Immunosuppressive Therapies in Kidney Transplant Patients.[NCT04360031] | | 100 participants (Anticipated) | Observational | 2020-02-10 | Recruiting |
A Multicenter Clinical Trial: Risk Factors of Chinese Kidney Transplant Recipients DSA Based on MPA Immunosuppressive Regimen[NCT04444843] | Phase 4 | 300 participants (Anticipated) | Interventional | 2021-01-22 | Recruiting |
Prospective, Multicenter, Randomized Open Study to Evaluate the Progression of Renal Graft Fibrosis According to the Epithelial-mesenchymal Transition (EMT) in de Novo Renal Transplant Recipients Treated Either by a CNI Free Immunosuppressive Regimen With[NCT01079143] | Phase 3 | 194 participants (Actual) | Interventional | 2009-09-30 | Completed |
Clinical Evaluation of New Treatment Strategy of Mucous Membrane Pemphigoid Using Large Dose of Prednisolone Plus Intra-lesional of Triamcinolone Acetonide Followed by Combination of Mycophenolate Mofetil, Dapsone and Low Dose Prednisolone[NCT04744623] | Phase 2/Phase 3 | 10 participants (Actual) | Interventional | 2020-09-30 | Active, not recruiting |
A Randomized Controlled Clinical Trial of Low Dose Thymoglobulin and Extended Delay of Calcineurin Inhibitor Therapy for Renal Protection After Liver Transplantation[NCT00970073] | | 30 participants (Actual) | Interventional | 2009-08-31 | Completed |
Induction of Mixed Hematopoietic Chimerism in Older Patients With B-Cell Malignancies and in Selected Other Diseases, Using Low Dose TBI , PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to be Followed by Do[NCT00003196] | | 63 participants (Actual) | Interventional | 1997-09-30 | Completed |
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Human Leukocyte Antigen Partially-Matched Related Donor[NCT01341301] | Phase 2 | 25 participants (Actual) | Interventional | 2010-05-31 | Completed |
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 2 | 66 participants (Actual) | Interventional | 2004-12-31 | Completed |
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 2 | 12 participants (Actual) | Interventional | 2005-03-31 | Completed |
A Randomized, Open-label Study of the Effect of a Long-term Calcineurin Inhibitor-free Maintenance Regimen With CellCept and Sirolimus on Preservation of Renal Function and Prevention of Acute Rejection in Recipients of an Orthotropic Liver Transplant[NCT00118742] | Phase 4 | 293 participants (Actual) | Interventional | 2005-08-31 | Completed |
Open Label Multicenter Randomized Trial Comparing Standard Immunosuppression With Tacrolimus and Mycophenolate Mofetil With a Low Exposure Tacrolimus Regimen in Combination With Everolimus in de Novo Renal Transplantation in Elderly Patient[NCT03797196] | Phase 4 | 374 participants (Anticipated) | Interventional | 2019-07-29 | Active, not recruiting |
A Study to Evaluate the Efficacy of CellCept, Administered in a Sequential Treatment Scheme, in Delaying Progressive Renal Damage in Patients With Lupus Nephritis[NCT02081183] | Phase 3 | 16 participants (Actual) | Interventional | 2006-03-31 | Terminated |
Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients:A Large, Multiple-Center Prospective Study[NCT05872568] | Phase 4 | 270 participants (Anticipated) | Interventional | 2023-06-01 | Not yet recruiting |
Donor Statin Treatment for Prevention of Severe Acute GVHD After Nonmyeloablative Hematopoietic Cell Transplantation[NCT01527045] | Phase 2 | 47 participants (Actual) | Interventional | 2012-09-25 | Completed |
A Randomized Study to Evaluate The Efficacy of Mycophenolate Mofetil Added to The Systemic Immunosuppressive Regimen First Used For Treatment of Chronic Graft-Versus-Host Disease[NCT00089141] | Phase 3 | 151 participants (Actual) | Interventional | 2004-05-31 | Terminated(stopped due to Low probability of positive outcome) |
A Multi-Center Study of Nonmyeloablative Conditioning With TBI or Fludarabine/TBI for HLA-matched Related Hematopoietic Cell Transplantation for Treatment of Hematologic Malignancies With Post Grafting Immunosuppression With Tacrolimus and Mycophenolate M[NCT00089011] | Phase 2 | 150 participants (Actual) | Interventional | 2004-04-30 | Completed |
Open-label, Multicenter Phase II Study of Combination Therapy of Imatinib Mesylate and Mycophenolate Mofetil in Children With Steroid-Refractory Sclerotic/Fibrotic Type Chronic Graft-versus-host Disease[NCT01898377] | Phase 2 | 9 participants (Actual) | Interventional | 2013-08-31 | Terminated(stopped due to New treatment option introduced for patients with the study indication) |
A Randomized Phase II Study to Determine the Most Promising Postgrafting Immunosuppression for Prevention of Acute GVHD After Unrelated Donor G-CSF Mobilized Peripheral Blood Mononuclear Cell (G-PBMC) Transplantation Using Nonmyeloablative Conditioning fo[NCT00105001] | Phase 2 | 210 participants (Actual) | Interventional | 2004-11-30 | Completed |
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 2 | 16 participants (Actual) | Interventional | 2002-10-31 | Completed |
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 2 | 17 participants (Actual) | Interventional | 2002-04-30 | Completed |
Safety, Efficacy and Feasibility of Haploidentical Stem Cell Transplantation (Haplo-SCT) Using Post-Transplant Cyclophosphamide (PTCy) as an Alternative Donor Source for Patients Who Lack a Matched Sibling/Unrelated Donor Options[NCT03088709] | Phase 2 | 40 participants (Anticipated) | Interventional | 2017-01-18 | Recruiting |
Pentostatin and Donor Lymphocyte Infusion for Low Donor T-cell Chimerism After Hematopoietic Cell Transplantation - A Multi-center Trial[NCT00096161] | Phase 2 | 36 participants (Actual) | Interventional | 2003-05-31 | Completed |
Randomized Clinical Trial to Compare Efficacy and Safety of Repeated Courses of Rituximab to That of Maintenance Mycophenolate Mofetil Following Single Course of Rituximab in Maintaining Remission Over 24 Months Among Children With Steroid Dependent Nephr[NCT03899103] | Phase 3 | 100 participants (Anticipated) | Interventional | 2019-05-15 | Active, not recruiting |
Tacrolimus In Combination, Tacrolimus Alone Compared (TICTAC Trial): A Prospective Randomized Trial Of Minimized Immunosuppression In Adult Heart Transplant Recipients[NCT00299221] | Phase 4 | 150 participants (Actual) | Interventional | 2004-04-30 | Completed |
A Phase III Randomized Open-label Multi-center Study of Ruxolitinib vs. Best Available Therapy in Patients With Corticosteroid-refractory Chronic Graft vs Host Disease After Allogeneic Stem Cell Transplantation (REACH3)[NCT03112603] | Phase 3 | 330 participants (Actual) | Interventional | 2017-06-29 | Completed |
A Study of the Safety, Tolerability and Pharmacokinetics of Oral CellCept® (Mycophenolate Mofetil, MMF) in Pediatric Liver Transplant Recipients on Concomitant Treatment With Cyclosporine and Corticosteroids[NCT02630563] | Phase 2 | 9 participants (Actual) | Interventional | 2003-05-31 | Terminated(stopped due to Due to extremely slow recruitment, infrequent use of combination triple therapy (MMF, cyclosporine, steroids), study was discontinued; Part 2 was not conducted.) |
A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies[NCT01384513] | Phase 2 | 40 participants (Actual) | Interventional | 2011-08-04 | Completed |
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 2 | 38 participants (Actual) | Interventional | 2017-05-30 | Active, not recruiting |
Reduced Intensity, Partially HLA Mismatched Allogeneic BMT for Hematologic Malignancies Using Donors Other Than First-degree Relatives[NCT01203722] | Phase 1/Phase 2 | 100 participants (Anticipated) | Interventional | 2010-09-30 | Recruiting |
Randomized, Multicenter, Open-label, Comparative Study of Efficacy and Safety of Treatment With a Calcineurin Inhibitor (CNI), Associating Myfortic ® and Neoral ® Compared to a CNI-free Treatment, Combining Myfortic ® and Certican ® , in Adult Patients Wi[NCT01595984] | Phase 3 | 90 participants (Actual) | Interventional | 2012-05-03 | Active, not recruiting |
Observational Study of Visual Outcomes in Retinal Disease[NCT01613963] | | 2,000 participants (Anticipated) | Observational | 2012-05-31 | Enrolling by invitation |
A Controlled Randomized Open-label Multicenter Study Evaluating if Early Conversion to Everolimus (Certican) From Cyclosporine (Neoral) in de Novo Renal Transplant Recipients Can Improve Long-term Renal Function and Slow Down the Progression of Chronic Al[NCT00634920] | Phase 4 | 204 participants (Actual) | Interventional | 2008-03-31 | Completed |
Belatacept Therapy for the Failing Renal Allograft[NCT01921218] | Phase 3 | 13 participants (Actual) | Interventional | 2013-08-31 | Completed |
A Prospective Cross-over Study Comparing the Effect of Sirolimus Versus Mycophenolate on Viral Load in Liver Transplant Recipients With Recurrent Chronic HCV Infection[NCT01134952] | Phase 4 | 11 participants (Actual) | Interventional | 2010-06-30 | Completed |
Allogeneic Nonmyeloablative Hematopoietic Stem Cell Transplant for Patients With BCR-ABL Tyrosine Kinase Inhibitor Responsive Ph+ Acute Leukemia ? A Multi-center Trial[NCT00036738] | Phase 2 | 28 participants (Actual) | Interventional | 2001-07-13 | Completed |
Non-Myeloablative Conditioning and Unrelated Umbilical Cord Blood Transplantation for Children and Adults With Serious Oncohematologic Diseases[NCT00255684] | | 16 participants (Actual) | Interventional | 2003-12-31 | Terminated(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 2 | 210 participants (Actual) | Interventional | 2004-10-31 | Completed |
Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematological Malignancies Using a Non-Myeloablative Preparative Regimen[NCT00305682] | Phase 2 | 295 participants (Actual) | Interventional | 2005-06-30 | Completed |
Donor Specific Regulation (DSR) Guided Tacrolimus Withdrawal to Myfortic Monotherapy in Liver Transplantation[NCT01230502] | | 11 participants (Actual) | Interventional | 2011-11-30 | Terminated(stopped due to insufficient study population to meet study objective) |
First-line Antimetabolites as Steroid-sparing Treatment Uveitis Pilot Trial[NCT01232920] | Phase 3 | 80 participants (Actual) | Interventional | 2010-10-31 | Completed |
A Randomized, Open Label, Four-way Crossover Study to Compare the Pharmacokinetics of Mycophenolate Mofetil Metabolites From Four Tablet Formulations in Healthy Subjects[NCT02981290] | Phase 1 | 32 participants (Actual) | Interventional | 2008-07-31 | Completed |
Sirolimus and Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor Hematopoietic Cell Transplantation[NCT00350181] | Phase 2 | 11 participants (Actual) | Interventional | 2006-08-31 | Completed |
An Open, Multicentre, Randomised, Parallel Group Study to Compare in Marginal Old-for-old Renal Transplant Patients the Safety and Efficacy of Two Treatments: Sequential Mycophenolate Mofetil/Delayed Tacrolimus Plus Steroids vs Tacrolimus Plus Mycophenola[NCT00321113] | Phase 3 | 142 participants (Actual) | Interventional | 2004-09-30 | Completed |
A Pilot Study of Corticosteroid-Free, Calcineurin-Sparing Immunosuppression Protocol for HLA-Identical Living Donor Renal Transplant Recipient[NCT00352092] | Phase 4 | 20 participants (Actual) | Interventional | 2002-06-30 | Completed |
A Randomized, Open-label, Parallel-Group Multicenter Study to Determine the Safety/Efficacy of Mycophenolate Mofetil in Mono & Combination Therapy With Interferon Beta 1a in Patients With Relapsing Remitting Multiple Sclerosis[NCT00324506] | Phase 2 | 43 participants (Actual) | Interventional | 2006-05-31 | Completed |
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 2 | 20 participants (Actual) | Interventional | 2011-11-16 | Terminated(stopped due to Terminated due to insufficient funding) |
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using Two Related Donors[NCT01532635] | Phase 2 | 4 participants (Actual) | Interventional | 2012-03-31 | Terminated(stopped due to Slow accrual) |
Randomized Controlled Trial of Infliximab (Remicade®) Induction Therapy for Deceased Donor Kidney Transplant Recipients (CTOT-19)[NCT02495077] | Phase 2 | 290 participants (Actual) | Interventional | 2015-11-02 | Completed |
Impact of Two Prednisone-free Maintenance Immunosuppressive Regimens With Reduced Dose FK506+Everolimus vs. Standard Dose Tacrolimus (FK506)+ Mycophenolate Mofetil (MMF) on Subpopulation of T and B Cells, Renal Allograft Function and Gene Expression Profi[NCT01653847] | | 88 participants (Actual) | Interventional | 2013-02-28 | Completed |
A 2 Step Approach to Haploidentical Transplant Using Radiation-Based Reduced Intensity Conditioning[NCT05031897] | Phase 2 | 67 participants (Anticipated) | Interventional | 2021-10-25 | Recruiting |
Non-Myeloablative Allogeneic Bone Marrow Transplant for Hematologic Malignancies[NCT00003572] | Phase 2 | 20 participants (Anticipated) | Interventional | 1998-08-31 | Completed |
Donor-Alloantigen-Reactive Regulatory T Cell (darTreg) Therapy in Liver Transplantation (RTB-002)[NCT02188719] | Phase 1 | 15 participants (Actual) | Interventional | 2014-12-17 | Terminated(stopped due to The trial could not be completed within the grant timeline.) |
Induction of Mixed Hematopoietic Chimerism Using Fludarabine, Low Dose TBI , PBSC Infusion and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate Mofetil to be Followed by Donor Lymphocyte Infusion In Patients With Chronic Myeloid Leuke[NCT00003145] | Phase 2 | 18 participants (Actual) | Interventional | 1997-08-31 | Completed |
A Randomized Trial Comparing Methotrexate Versus Mycophenolate Mofetil for Remission Maintenance in Wegener's Granulomatosis and Related Vasculitides[NCT00004567] | Phase 2 | 75 participants | Interventional | 2000-02-29 | Completed |
TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study[NCT04182984] | | 200 participants (Anticipated) | Observational [Patient Registry] | 2019-11-04 | Recruiting |
Bone Marrow Transplantation and High Dose Post-Transplant Cyclophosphamide for Chimerism Induction and Renal Allograft Tolerance (ITN054ST)[NCT02029638] | Phase 2 | 4 participants (Actual) | Interventional | 2014-01-07 | Terminated(stopped due to Slow accrual) |
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Haploidentical Donor[NCT01982682] | Phase 2 | 41 participants (Actual) | Interventional | 2013-11-04 | Completed |
A Randomized, Double-Blind, Double-Dummy, Active-Comparator, Multicenter Study to Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients With Pemphigus Vulgaris[NCT02383589] | Phase 3 | 135 participants (Actual) | Interventional | 2015-05-26 | Completed |
De Novo Everolimus Versus Tacrolimus in Combination With Mofetil Mycophenolate and Low Dose Corticosteroids to Reduce Tacrolimus Induced Nephrotoxicity in Liver Transplantation: a Prospective, Multicentric, Randomised Study[NCT02909335] | Phase 3 | 0 participants (Actual) | Interventional | 2016-11-30 | Withdrawn |
Multi-center, Randomized, Open Label, Comparative, Phase IV Study to Evaluate the Efficacy and Safety of a Combination of Mycophenolate Mofetil and Corticosteroid for 48 Weeks in Advanced IgA Nephropathy[NCT02981212] | Phase 4 | 100 participants (Anticipated) | Interventional | 2016-06-30 | Recruiting |
A Multicenter, Randomized, Double-Masked Study To Evaluate The Safety, Tolerability, And Efficacy Of SURF-100 Ophthalmic Solution (A Mycophenolic Acid/Betamethasone Sodium Phosphate Combination) In Subjects With Dry Eye Disease[NCT04734197] | Phase 2 | 351 participants (Actual) | Interventional | 2021-01-11 | Completed |
A Randomized Prospective Trial of Conversion to Everolimus Therapy From Calcineurin Inhibitor Based Maintenance Immunosuppression in Association With Mycophenolic Acid in Liver Transplantation: Examination of Impact on Long Term Renal Function.[NCT01936519] | | 24 participants (Actual) | Interventional | 2013-12-16 | Completed |
A 12-month Randomized, Multiple Dose, Open-label, Study Evaluating Safety, Tolerability, Pharmacokinetics/Pharmacodynamics (PK/PD) and Efficacy of an Anti-CD40 Monoclonal Antibody, CFZ533, in Combination With Mycophenolate Mofetil (MMF) and Corticosteroid[NCT02217410] | Phase 1/Phase 2 | 59 participants (Actual) | Interventional | 2015-02-05 | Completed |
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 2 | 21 participants (Actual) | Interventional | 2016-09-30 | Completed |
Impact of the Microbiota on the Likelihood of Renal Graft Rejection[NCT04736381] | | 70 participants (Anticipated) | Observational | 2021-04-14 | Not yet recruiting |
Gemcitabine/Clofarabine/Busulfan and Allogeneic Transplantation for Aggressive Lymphomas[NCT01701986] | Phase 1/Phase 2 | 80 participants (Anticipated) | Interventional | 2012-10-25 | Active, not recruiting |
A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplantation for the Treatment of Relapsed Non-Hodgkin and Hodgkin Lymphoma[NCT00057954] | Phase 2 | 6 participants (Actual) | Interventional | 2005-11-09 | Terminated(stopped due to Slow accrual) |
A Phase II Study of Reduced Intensity Allogeneic Bone Marrow Transplant for the Treatment of Myelodysplastic Syndromes[NCT00045305] | Phase 2 | 17 participants (Actual) | Interventional | 2006-10-24 | Completed |
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 1 | 26 participants (Actual) | Interventional | 2007-10-05 | Terminated(stopped due to Terminated due to loss of funding) |
Donor-derived Mesenchymal Stromal Cells, Alemtuzumab, Co-stimulation Blockade and Sirolimus for Tolerance Induction in Adult Kidney Allograft Recipients (ITN062ST)[NCT03504241] | Phase 1 | 8 participants (Actual) | Interventional | 2018-07-30 | Active, not recruiting |
Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease[NCT00004485] | Phase 1/Phase 2 | 50 participants | Interventional | 1999-12-31 | Completed |
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) | Interventional | 1999-11-30 | Completed |
Post Transplant Infusion of Allogeneic CD8 Memory T-Cells as Consolidative Therapy After Non-myeloablative Allogeneic Hematopoietic Cell Transplantation in Patients With Leukemia and Lymphoma[NCT02424968] | Phase 2 | 18 participants (Actual) | Interventional | 2015-06-30 | Completed |
Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Hematological Diseases Using a Non-Myeloablative Preparative Regimen[NCT00365287] | Phase 1/Phase 2 | 148 participants (Actual) | Interventional | 2000-06-30 | Completed |
A Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haploidentical Peripheral Blood Stem Cell Transplantation[NCT03018223] | Phase 1 | 32 participants (Actual) | Interventional | 2017-01-31 | Completed |
Once-daily Regimen With Envarsus® to Optimize Immunosuppression Management and Outcomes in Kidney Transplant Recipients[NCT02954198] | | 40 participants (Actual) | Interventional | 2016-12-01 | Completed |
Open Label Randomized Single Study to Evaluate the Safety & Efficacy of Early CNI Withdrawal in Recipients of Primary Renal Allografts Maintained Long-Term on Mycophenolate Mofetil; MMF (CellCept) and Sirolimus (Rapamune)[NCT00374647] | Phase 4 | 17 participants (Actual) | Interventional | 2005-03-31 | Completed |
Dosing Requirements of Astagraf XL® in African American Kidney Transplant Recipients Converted From Twice-daily Tacrolimus[NCT02953873] | Phase 4 | 25 participants (Actual) | Interventional | 2017-05-05 | Completed |
NEw Clinical Endpoints in Patients With Primary Sjögren's Syndrome (pSS): an Interventional Trial Based on stratifYing Patients[NCT05113004] | Phase 2 | 300 participants (Anticipated) | Interventional | 2022-01-20 | Recruiting |
Umbilical Cord Blood Transplant With Co-Infusion of T Regulatory Cells[NCT00376519] | Phase 1 | 3 participants (Actual) | Interventional | 2007-05-31 | Terminated(stopped due to Slow accrual) |
A Multi-center Phase III Study Comparing Myeloablative to Nonmyeloablative Transplant Conditioning in Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia[NCT00322101] | Phase 3 | 25 participants (Actual) | Interventional | 2006-01-31 | Completed |
A Multicenter, Randomization, Open-label Phase 4 Clinical Trial to Evaluate the Efficacy and Safety of MYREPTIC-N® or MY-REPT® in Stable Patients After Kidney Transplant Recipients[NCT06044493] | Phase 4 | 158 participants (Anticipated) | Interventional | 2023-11-01 | Not yet recruiting |
A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Obinutuzumab in Patients With ISN/RPS 2003 Class III or IV Lupus Nephritis[NCT02550652] | Phase 2 | 126 participants (Actual) | Interventional | 2015-11-13 | Completed |
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 2 | 163 participants (Actual) | Interventional | 2012-12-11 | Completed |
Compare the Safety and Effective of HLA-mismatched Microtransplantation With HLA-matched Nonmyeloablative Transplantation for Acute Myeloid Leukemia in Intermediate-risk[NCT02461121] | Phase 3 | 156 participants (Actual) | Interventional | 2004-05-31 | Completed |
Effect of the Introduction of mTor Inhibitors in the Activation of the Cytomegalovirus (CMV) -Specific Cellular Immune Response to Control Viral Replication in Kidney Transplant Patients[NCT04936971] | Phase 4 | 46 participants (Anticipated) | Interventional | 2021-09-30 | Not yet recruiting |
A Randomized, Multicenter Study of Belatacept, Everolimus, rATG and Early Steroid Withdrawal Versus Belatacept, Mycophenolate, rATG and Chronic Steroids in Renal Transplantation[NCT04849533] | Phase 4 | 120 participants (Anticipated) | Interventional | 2021-04-09 | Recruiting |
Mycophenolate Mofetil Versus Cyclophosphamide in the Induction Therapy of Pediatric Patients With Active Proliferative Lupus Nephritis: A Prospective, Randomized, Multicenter, Open-label, Parallel-arm Study[NCT05495893] | Phase 4 | 224 participants (Anticipated) | Interventional | 2022-07-25 | Recruiting |
Comparing the Effects of an Immunosuppressant (Mycophenolate Mofetil or MMF) on the Urinary Sodium Excretion Response to Mental Stress in a Crossover Design (MMF)[NCT02432339] | Phase 1/Phase 2 | 50 participants (Anticipated) | Interventional | 2014-04-22 | Recruiting |
Pilot Study of Infusion of Fucosylated Regulatory T Cells to Prevent Graft Versus Host Disease[NCT02423915] | Phase 1 | 5 participants (Actual) | Interventional | 2015-07-30 | Completed |
A Randomized, Multicenter, Phase III Trial of Tacrolimus/Methotrexate Versus Post-Transplant Cyclophosphamide/Tacrolimus/Mycophenolate Mofetil in Non-Myeloablative/Reduced Intensity Conditioning Allogeneic Peripheral Blood Stem Cell Transplantation (BMT C[NCT03959241] | Phase 3 | 428 participants (Anticipated) | Interventional | 2019-06-25 | Active, not recruiting |
Prospective, Multicenter, Randomized, Evaluating Two Induction Therapies With Simulect® Versus ATG® Fresenius Associated With Tacrolimus and Myfortic® in the Prevention of Treatment Failure, in Sensitized Renal Transplant[NCT02377193] | Phase 4 | 60 participants (Actual) | Interventional | 2013-09-30 | Completed |
A Single-arm, Single-center Trial of Prophylactic Tocilizumab for Acute GVHD Prevention After Haploidentical HSCT.[NCT04688021] | Phase 2 | 46 participants (Anticipated) | Interventional | 2020-12-03 | Recruiting |
Treatment Outcome in Early Diffuse Cutaneous Systemic Sclerosis[NCT02339441] | | 320 participants (Actual) | Observational | 2010-06-30 | Completed |
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 2 | 11 participants (Actual) | Interventional | 2015-09-14 | Active, not recruiting |
Prospective Study of Combined ATG Regimen for Prophylaxis of aGVHD in Matched Sibling Donor PBSCT[NCT02677181] | Phase 4 | 100 participants (Actual) | Interventional | 2016-01-31 | Completed |
A Prospective Randomized, Controlled Trial of Mycophenolate Mofetil Plus Steroid in the Treatment Of Patients With Progressive Idiopathic Membranous Nephropathy[NCT03170323] | Phase 4 | 128 participants (Anticipated) | Interventional | 2018-07-01 | Recruiting |
The Efficacy and Safety of Corticosteroid Combining Noncorticosteroid Systemic Immunomodulatory Therapy in VKH[NCT05120687] | | 200 participants (Anticipated) | Observational | 2021-10-15 | Recruiting |
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 2 | 30 participants (Anticipated) | Interventional | 2019-07-10 | Recruiting |
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 2 | 75 participants (Anticipated) | Interventional | 2017-10-24 | Suspended(stopped due to Administrative - FDA Comments) |
T Cell-Reduced Unrelated Donor Allogeneic Peripheral Blood Stem Cell Transplantation With Pentostatin and Low-Dose Total Body Irradiation[NCT00816413] | Phase 1/Phase 2 | 0 participants (Actual) | Interventional | 2008-09-30 | Withdrawn(stopped due to Screenings yielded inadequate eligible subjects to enroll.) |
Effectiveness of Mycophenolate Mofetil Combined With Tacrolimus for Steroid Tapering in Systemic Lupus Erythematosus: A Prospective, Random Control, Open-label, Single Center Clinical Trial[NCT05916781] | Phase 4 | 220 participants (Anticipated) | Interventional | 2023-07-01 | Recruiting |
Assessment of Gastrointestinal Tolerability and Efficacy of Enteric-coated Mycophenolate Sodium (Myfortic®) in Heart Transplant Recipients[NCT00574197] | Phase 4 | 11 participants (Actual) | Interventional | 2006-06-30 | Completed |
Immune Monitoring and Calcineurin Inhibitor (CNI) Withdrawal in Low Risk Recipients of Kidney Transplantation[NCT01517984] | Phase 2 | 52 participants (Actual) | Interventional | 2010-11-30 | Terminated(stopped due to Absence of equipoise on the basis of predetermined stopping rules.) |
Pharmacokinetics Study of Mycophenolic Acid in Patients With an Autoimmune Bullous Dermatose, Pemphigus or Cicatricial Pemphigoid.[NCT02993133] | Phase 3 | 53 participants (Actual) | Interventional | 2016-12-31 | Completed |
Total Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation[NCT00352976] | Phase 2/Phase 3 | 83 participants (Actual) | Interventional | 2006-05-18 | Completed |
Mycophenolate Mofetil Versus Cyclosporin A in the Treatment of Primary Biliary Cholangitis-autoimmune Hepatitis Overlap Syndrome Duo to Nonresponse to Standard Therapy[NCT04376528] | Phase 4 | 89 participants (Anticipated) | Interventional | 2021-06-16 | Recruiting |
Using mTOR Inhibitors in the Prevention of BK Nephropathy[NCT01649609] | | 40 participants (Actual) | Interventional | 2012-03-31 | Completed |
Busulfan and Cyclophosphamide Followed By Allogeneic Hematopoietic Cell Transplantation In Patients With Hematological Malignancies[NCT01685411] | | 5 participants (Actual) | Interventional | 2013-01-31 | Terminated |
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 2 | 29 participants (Actual) | Interventional | 2008-12-31 | Completed |
Topical 0.03% Tacrolimus Versus Systemic Mycophenolate Mofetil for Preventing Graft Rejection After Repeat Keratoplasty: One-year Results of a Randomized Clinical Trial[NCT04147390] | Phase 2/Phase 3 | 58 participants (Anticipated) | Interventional | 2019-11-30 | Recruiting |
Pharmacokinetic Cross-over Study to Evaluate the Influence of Pantoprazole on MPA Bioavailability Administered as Mycophenolate Mofetil and Enteric Coated Mycophenolate Sodium in Maintenance Renal Transplant Patients[NCT01801280] | Phase 4 | 20 participants (Actual) | Interventional | 2012-01-31 | Completed |
A Novel Immunosuppression Intervention for the Treatment of Amyotrophic Lateral Sclerosis (ALS)[NCT01884571] | Phase 2 | 31 participants (Actual) | Interventional | 2013-10-31 | Completed |
A 12-Month, Open Label Study of Extended Release Tacrolimus (Envarsus XR®, LCPT) With Mycophenolate, Rabbit Antithymocyte Globulin (rATG) and Early Steroid Withdrawal in de Novo Kidney Transplant Recipients[NCT03828682] | Phase 4 | 60 participants (Anticipated) | Interventional | 2019-06-21 | Recruiting |
A Pilot Study of Pharmacokinetics-based Mycophenolate Mofetil Dosing for Graft-Versus-Host-Disease Prophylaxis in Pediatric Blood and Marrow Transplantation[NCT01487577] | Phase 2 | 19 participants (Actual) | Interventional | 2010-06-30 | Completed |
Allogeneic Hematopoietic Stem Cell Transplantation With Nonmyeloablative Conditioning for Patients With Chronic Lymphocytic Leukemia - A Multi-center Trial[NCT00060424] | Phase 2 | 21 participants (Actual) | Interventional | 2003-03-31 | Completed |
A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of BIIB023 in Subjects With Lupus Nephritis[NCT01499355] | Phase 2 | 276 participants (Actual) | Interventional | 2012-07-31 | Terminated(stopped due to Results from pre-specified criteria did not demonstrate sufficient efficacy to warrant continuation of the study.) |
Allogeneic Hematopoietic Cell Transplantation of Positively Selected CD34+ Cells and Defined Inoculum of T Cells From Related Haploidentical Donors for Older Patients With Indolent Hematologic Malignancies[NCT00185692] | Phase 2 | 16 participants (Actual) | Interventional | 2000-08-31 | Completed |
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 2 | 60 participants (Actual) | Interventional | 2001-11-30 | Completed |
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 2 | 53 participants (Actual) | Interventional | 2002-01-31 | Completed |
Proteomics Combined With Metabolomics Studies on the Efficacy of Telitacicept in Chinese Patients of Systemic Lupus Erythematosus[NCT05666336] | Phase 4 | 30 participants (Anticipated) | Interventional | 2022-12-31 | Recruiting |
A Pilot Study of Allogeneic Hematopoietic Stem Cell Transplantation for Pediatric and Adolescent-Young Adults Patients With High Risk Solid Tumors[NCT04530487] | Phase 2 | 40 participants (Anticipated) | Interventional | 2020-08-19 | Recruiting |
Efficacy & Safety of Pharmacokinetically-Driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis- A Double-Blind Placebo Controlled Clinical Trial[NCT05538208] | Phase 2 | 105 participants (Anticipated) | Interventional | 2023-08-31 | Not yet recruiting |
A Phase II Trial Evaluating the Safety and Efficacy of Non-myeloablative 90Y-Ibritumomab Tiuxetan (Anti-CD20) Antibody With Fludarabine, Low-Dose Total Body Irradiation (TBI) and HLA Matched Allogeneic Transplantation for Relapsed B-cell Lymphoma[NCT00119392] | Phase 2 | 42 participants (Actual) | Interventional | 2004-06-30 | Completed |
An Open Label, Randomized, Multicenter Study to Evaluate the Efficacy and Safety of Early Calcineurin Inhibitor Withdrawal in Recipients of Primary Renal Allografts Maintained Long-term on Mycophenolate Mofetil (MMF) (CellCept®) and Sirolimus (Rapamune®)[NCT00121810] | Phase 4 | 305 participants (Actual) | Interventional | 2003-08-31 | Completed |
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 2 | 5 participants (Actual) | Interventional | 2003-04-30 | Terminated(stopped due to Poor accrual) |
Optimization of NULOJIX® (Belatacept) Usage as a Means of Minimizing CNI Exposure in Simultaneous Pancreas and Kidney Transplantation (CTOT-15)[NCT01790594] | Phase 2 | 46 participants (Actual) | Interventional | 2013-02-28 | Terminated(stopped due to Slow accrual within enrollment time period: projected accrual goal not achieved.) |
Nonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding Study[NCT00453388] | Phase 2 | 6 participants (Actual) | Interventional | 2007-02-28 | Completed |
Phase II, Randomized, Placebo Controlled Trial of the Safety and Tolerability of Mycophenolate in Children With Juvenile Neuronal Ceroid Lipofuscinosis[NCT01399047] | Phase 2 | 19 participants (Actual) | Interventional | 2011-07-31 | Completed |
Allogeneic Hematopoietic Cell Transplantation for Patients With Treatment-Refractory Crohn's Disease: A Phase 2 Study[NCT01570348] | Phase 2 | 2 participants (Actual) | Interventional | 2012-07-17 | Terminated(stopped due to Annual accrual goal not met) |
Steroid and Tacrolimus Avoidance Using NULOJIX® (Belatacept) in Renal Transplantation (CTOT-16)[NCT01856257] | Phase 2 | 71 participants (Actual) | Interventional | 2013-07-31 | Terminated(stopped due to Safety: Stopping rule not met.) |
Advancing Transplantation Outcomes in Children (CTOT-41)[NCT06055608] | Phase 2 | 200 participants (Anticipated) | Interventional | 2023-11-01 | Not yet recruiting |
Effect of Azole/Echinocandin Use on Tacrolimus Pharmacokinetics in Kidney Transplant Recipients[NCT06044558] | | 507 participants (Actual) | Observational | 2022-09-01 | Completed |
A Multi-centre Phase II Trial of GVHD Prophylaxis Following Unrelated Donor Stem Cell Transplantation Comparing Thymoglobulin vs. Calcineurin Inhibitor or Sirolimus-based Post-transplant Cyclophosphamide[NCT04888741] | Phase 2 | 400 participants (Anticipated) | Interventional | 2021-02-22 | Recruiting |
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 2 | 25 participants (Actual) | Interventional | 2007-11-13 | Completed |
Head to Head Comparison of Myfortic vs. Cellcept in the Treatment of Kidney Transplant Recipients Using Our Current Center Standardized Concomitant Immunosuppressive Protocol[NCT00533624] | Phase 2/Phase 3 | 150 participants (Actual) | Interventional | 2004-12-31 | Completed |
"MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and Off-the-shelf Mesenchymal Stem Cells"[NCT01033552] | Phase 1/Phase 2 | 32 participants (Actual) | Interventional | 2010-01-31 | Completed |
Belimumab for the Treatment of Diffuse Cutaneous Systemic Sclerosis: A Phase 2a, Single-centered, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Pilot Study.[NCT01670565] | Phase 2 | 20 participants (Actual) | Interventional | 2012-08-31 | Completed |
A Pilot Study Comparing Two Different Sirolimus-based Transition Regimens in African-American Renal Transplant Recipients[NCT01005706] | | 40 participants (Actual) | Interventional | 2009-08-31 | Completed |
Comparison of Pharmocokinetics of Mycophenolate Mofetil and Enteric Coated Mycophenolate Sodium in Calcineurininhibitor-free Treated Patients After Renal Transplantation[NCT01033864] | Phase 4 | 23 participants (Actual) | Interventional | 2009-11-30 | Completed |
A Phase 1/2 Multi-Center Trial of Vorinostat for Graft vs Host Disease Prevention in Children, Adolescents and Young Adults Undergoing Allogeneic Blood and Marrow Transplantation[NCT03842696] | Phase 1/Phase 2 | 49 participants (Anticipated) | Interventional | 2020-02-04 | Recruiting |
Pharmacokinetically-driven Dosing of Mycophenolate Mofetil for the Treatment of Pediatric Proliferative Lupus Nephritis[NCT05101447] | Phase 2 | 0 participants (Actual) | Interventional | 2023-07-31 | Withdrawn(stopped due to funding not secured) |
Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies[NCT04776850] | Early Phase 1 | 0 participants (Actual) | Interventional | 2020-12-29 | Withdrawn(stopped due to Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.) |
Evaluation of the Effectiveness and Safety of Immunosuppressive and Biological Therapy of Atopic Dermatitis in Childhood[NCT04895423] | Phase 4 | 160 participants (Anticipated) | Interventional | 2021-11-25 | Not yet recruiting |
Non-interventional Clinical Study With Target of Kidney Function Follow-up in Routine Clinical Practice on De Novo Kidney Transplant Recipients Who Are on CellCept Immunosuppressive Combination Therapy in Routine Clinical Practice[NCT01672957] | | 128 participants (Actual) | Observational | 2011-09-30 | Completed |
Pilot Study of JAK Inhibitor Therapy Followed by Reduced Intensity Haploidentical Transplantation for Patients With Myelofibrosis[NCT04370301] | Phase 2 | 10 participants (Anticipated) | Interventional | 2021-02-09 | Recruiting |
An Investigator-Initiated, Phase II, Randomized, Withdrawal Study of Mycophenolate Mofetil (MMF) in Patients With Stable, Quiescent Systemic Lupus Erythematosus (SLE)[NCT01946880] | Phase 2 | 102 participants (Actual) | Interventional | 2013-11-20 | Terminated(stopped due to Slow enrollment.) |
Mechanisms and Treatment of Chronic Allograft Injury (CAI) Due to Calcineurin Inhibitor (CNI) Toxicity[NCT01473732] | | 2 participants (Actual) | Interventional | 2012-03-31 | Terminated(stopped due to terminated after 2 patients due to difficulty in enrollment) |
A Multi-center Phase II Trial Randomizing Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN #1203; Progress I)[NCT02208037] | Phase 2 | 279 participants (Actual) | Interventional | 2014-08-31 | Completed |
A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation[NCT01529827] | Phase 2 | 94 participants (Actual) | Interventional | 2012-02-28 | Completed |
In-Vivo Activated T-Cell Depletion to Prevent GVHD[NCT00594308] | | 10 participants (Actual) | Interventional | 2007-10-31 | Terminated(stopped due to Treatment ineffective) |
A Pilot Study of an Immunosuppressive and Myeloablative Preparative Regimen for Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation (HSCT) for Severe Sickle Cell Disease[NCT01279616] | Phase 2 | 8 participants (Actual) | Interventional | 2010-09-30 | Terminated(stopped due to PI moving to a different institution.) |
Trial Of Double Umbilical Cord Blood Transplantation[NCT00763490] | Phase 2 | 20 participants (Actual) | Interventional | 2008-12-31 | Completed |
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 2 | 77 participants (Actual) | Interventional | 2010-12-01 | Completed |
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 2 | 24 participants (Actual) | Interventional | 2014-07-11 | Terminated(stopped due to The study was halted early due to slow accrual.) |
A Two Step Approach to Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies in Remission From HLA Partially-Matched Related Donors[NCT01350245] | Phase 2 | 28 participants (Actual) | Interventional | 2010-07-31 | Completed |
Dose-Intense Yttrium-90 Ibritumomab Tiuxetan (Zevalin)-Containing Non-Myeloablative Conditioning for Allogeneic Stem Cell Transplantation in B-cell Malignancies[NCT01490723] | Phase 2 | 20 participants (Actual) | Interventional | 2013-01-31 | Completed |
A 3-month, Multicenter, Randomized, Open Label Study to Evaluate the Impact of Early Versus Delayed Introduction of Everolimus on Wound Healing in de Novo Kidney Transplant Recipients With a Follow-up Evaluation at 12 Month After Transplant (NEVERWOUND St[NCT01410448] | Phase 3 | 383 participants (Actual) | Interventional | 2011-11-30 | Completed |
Safety and Efficacy of Mycophenolic Acid Withdrawal With Conversion to Zortress (Everolimus) in Renal Transplant Recipients With BK Virus Infection[NCT01624948] | Phase 4 | 40 participants (Actual) | Interventional | 2012-09-30 | Completed |
Remission Induction Therapy for Refractory Systemic Lupus Erythematosus With Autologous Hematopoietic Stem Cell Transplantation (AHSCT) Versus Rituximab (antiCD20) Followed by Maintenance Therapy With Mycophenolate Mofetil (MMF)[NCT05063513] | Phase 2/Phase 3 | 0 participants (Actual) | Interventional | 2009-07-31 | Withdrawn |
Clofarabine Pre-conditioning Followed by Hematopoietic Stem Cell Transplant With Post-Transplant Cyclophosphamide for Non-remission Acute Myeloid Leukemia[NCT04002115] | Phase 2 | 2 participants (Actual) | Interventional | 2020-06-03 | Terminated(stopped due to terminated due to low accrual) |
A Randomized, Double-Blind, Placebo-Controlled Study of Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis[NCT03844061] | Phase 2 | 30 participants (Anticipated) | Interventional | 2019-07-29 | Recruiting |
A Phase II, Multicenter, Randomized, Open-label Study to Investigate the Safety and Efficacy of Mycophenolate Mofetil and Rilonacept (Anti-interleukin-1) in Patients With Alcoholic Hepatitis[NCT01903798] | Phase 2 | 4 participants (Actual) | Interventional | 2014-12-31 | Completed |
Comparison of the Efficacy and Safety of Sirolimus, Everolimus or Mycophenolate in Renal Transplant Recipients Receiving Induction With Anti-thymocyte Globulin, Tacrolimus and Prednisone[NCT03468478] | Phase 4 | 1,209 participants (Actual) | Interventional | 2017-06-18 | Completed |
A Phase 2, Double-Blind, Placebo-Controlled Trial of Mycophenolate Mofetil Alone or With Voclosporin for Systemic Lupus: Examining Distinct Immunophenotypes to Validate and Enhance Rational Treatment (DIVERT) (ALE10)[NCT05306873] | Phase 2 | 120 participants (Anticipated) | Interventional | 2022-10-28 | Recruiting |
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 1 | 0 participants (Actual) | Interventional | 2022-09-30 | Withdrawn(stopped due to Change in Study Design) |
Immunosuppressive Drugs and Gut Microbiome: Pharmacokinetic- and Microbiome Diversity Effects[NCT04207177] | Phase 4 | 100 participants (Anticipated) | Interventional | 2019-10-30 | Active, not recruiting |
A Randomized, Double-Blind, Parallel-Controlled, Multi-Center Study to Evaluate the Safety and Efficacy of Human Umbilical Cord Mesenchymal Stem Cells in Patients With Lupus Nephritis[NCT03580291] | Phase 2 | 230 participants (Anticipated) | Interventional | 2018-08-01 | Not yet recruiting |
A Randomized Open-label Study to Evaluate the Efficacy and Safety of Tacrolimus and Corticosteroids in Comparison With Mycophenolate Mofetil and Corticosteroids in Subjects With Class III/IV±V Lupus Nephritis[NCT02630628] | Phase 4 | 130 participants (Actual) | Interventional | 2015-12-05 | Active, not recruiting |
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major Treatment: a Multi-center, Open, Randomized, Controlled Clinical Study[NCT02342145] | Phase 4 | 40 participants (Anticipated) | Interventional | 2014-06-30 | Recruiting |
A Multicenter, Two Arms, Randomized, Open Label Clinical Phase IV Study Investigating the Proportion of CMV Seropositive Kidney Transplant Recipients Who Will Develop a CMV Infection Within the First 6 Months Post-transplantation When Treated With an Immu[NCT02328963] | Phase 4 | 186 participants (Actual) | Interventional | 2014-05-02 | Completed |
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 2 | 24 participants (Anticipated) | Interventional | 2023-02-09 | Recruiting |
A National Prospective Study of Patients With Hepatitis Induced by Immune Checkpoint Inhibitors; Characterization of Liver Injury, Outcome of Therapy and Randomization to Either Prednisolone or Mycophenolate Mofetil Treatment in Case of Relapse[NCT04810156] | Phase 2 | 60 participants (Anticipated) | Interventional | 2021-04-07 | Recruiting |
Randomized, Open-Label Trial of Tacrolimus/Everolimus vs. Tacrolimus/Enteric-Coated Mycophenolate Sodium to Prevent Biopsy-Proven Acute Rejection and Chronic Allograft Injury in Adult, Primary Kidney Transplantation[NCT01680861] | Phase 3 | 32 participants (Actual) | Interventional | 2012-11-30 | Completed |
Reappraisal of Second-line Therapies of Refractory Autoimmune Hemolytic Anemia in Systemic Lupus Erythematosus[NCT05057481] | Phase 3 | 30 participants (Anticipated) | Interventional | 2021-09-15 | Active, not recruiting |
A Randomised, Open-label Clinical Trial Assessing the Efficacy and Safety of Mycophenolate Mofetil Versus Azathioprine for Induction of Remission in Treatment Naive Autoimmune Hepatitis[NCT02900443] | Phase 4 | 70 participants (Anticipated) | Interventional | 2017-01-31 | Active, not recruiting |
A Pilot Study of Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease[NCT02678143] | Phase 1 | 1 participants (Actual) | Interventional | 2016-04-26 | Terminated(stopped due to Closed early due to competing studies) |
The Efficacy and Safety of Mycophenolate Mofetil in the Treatment of Lymphocytic Myocarditis in Comparison With Azathioprine[NCT05237323] | Phase 3 | 50 participants (Anticipated) | Interventional | 2020-10-01 | Recruiting |
"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 2 | 24 participants (Anticipated) | Interventional | 2022-04-08 | Recruiting |
A Prospective, Open-label, Multicenter, Randomized Study to Evaluate the Benefits and Risks of Conversion of Existing Adolescent Renal Allograft Recipients Aged 12 to Less Than 18 Years of Age to a Belatacept-based Immunosuppressive Regimen as Compared to[NCT04877288] | Phase 3 | 102 participants (Anticipated) | Interventional | 2021-07-21 | Recruiting |
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 2 | 74 participants (Anticipated) | Interventional | 2017-07-30 | Recruiting |
A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease[NCT03121001] | Phase 2 | 50 participants (Anticipated) | Interventional | 2017-03-20 | Recruiting |
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 2 | 1 participants (Actual) | Interventional | 2016-09-13 | Terminated(stopped due to Study was unable to accrue subjects) |
Transplantation of Umbilical Cord Blood in Patients With Hematological Malignancies Using a Reduced-Intensity Preparative Regimen (A Multi-Center Trial Coordinated by the FHCRC)[NCT00723099] | Phase 2 | 73 participants (Actual) | Interventional | 2008-06-25 | Completed |
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 2 | 76 participants (Actual) | Interventional | 1999-09-30 | Completed |
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 2 | 147 participants (Actual) | Interventional | 2005-06-30 | Completed |
Safety and Efficacy of Basiliximab, Delayed Dose Tacrolimus Plus ECMPA, Versus Standard Dose Tacrolimus, ECMPA Plus Corticosteroids in Patients Undergoing Liver Transplant[NCT02123108] | Phase 4 | 59 participants (Actual) | Interventional | 2011-01-31 | Completed |
A National, Multi-center, Randomized, Open Label Study to Evaluate the Efficacy and Safety of Everolimus Combined With Enteric-coated Mycophenolate Sodium Compared to the Standard Treatment Combining Tacrolimus and Enteric-coated Mycophenolate Sodium in d[NCT01625377] | Phase 3 | 188 participants (Actual) | Interventional | 2012-12-31 | Completed |
First-line Antimetabolites as Steroid-sparing Treatment (FAST) Uveitis Trial[NCT01829295] | Phase 3 | 216 participants (Actual) | Interventional | 2013-08-31 | Completed |
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 2 | 30 participants (Actual) | Interventional | 2013-06-24 | Completed |
A Randomized Study Comparing the Incidence of Acute Clinical or Subclinical Rejection With Two Dosing Regimens of CellCept in de Novo Renal Transplant Recipients Receiving Induction, Cyclosporine and Brief Steroid Therapy[NCT02005562] | Phase 3 | 252 participants (Actual) | Interventional | 2006-05-31 | Completed |
Prospective Randomised Marker-based Trial to Assess the Clinical Utility and Safety of Biomarker-guided Immunosuppression Withdrawal in Liver Transplantation[NCT02498977] | Phase 4 | 116 participants (Actual) | Interventional | 2015-10-31 | Active, not recruiting |
Does the Mycophenolate Improve the Ability of Weaning Patients Off the Treatment in Chronic Inflammatory Demyelinating Polyradiculopathy (CIDP)[NCT02494505] | Phase 3 | 40 participants (Actual) | Interventional | 2013-11-18 | Completed |
A Randomized Study to Evaluate Antibody Response to an Additional Dose of SARS-CoV-2 Vaccination With and Without Immunosuppression Reduction in Kidney and Liver Transplant Recipients[NCT05077254] | Phase 2 | 400 participants (Anticipated) | Interventional | 2021-12-06 | Recruiting |
A Pilot Study of Allogeneic Hematopoietic Cell Transplantation for Patients With High Grade Central Nervous System Malignancies[NCT04521946] | Phase 1 | 0 participants (Actual) | Interventional | 2021-01-14 | Withdrawn(stopped due to No participants enrolled.) |
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism[NCT00176865] | Phase 2 | 19 participants (Actual) | Interventional | 2002-08-31 | Completed |
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) | Interventional | 1999-11-30 | Completed |
Randomized Conversion Of Epstein-Barr Virus (EBV)+ Kidney Transplant Recipients Of Living Or Standard Criteria Donors At Three Months Post Transplantation To Belatacept With MPA Or Belatacept With Low-Dose Tacrolimus (50% Of Dose) Compared To Patients Rem[NCT02213068] | Phase 4 | 28 participants (Actual) | Interventional | 2014-07-31 | Completed |
Pediatric Blood & Marrow Transplant Consortium (PBMTC) Phase II Myeloablative Haploidentical BMT With Post-transplantation Cyclophosphamide for Pediatric Patients With Hematologic Malignancies[NCT02120157] | Phase 2 | 35 participants (Actual) | Interventional | 2015-07-02 | Completed |
12 Month, Multi-center, Open-label, Prospective, Randomized, Parallel Group Study Investigating a Standard Regimen in de Novo Kidney Transplant Patients Versus a Certican® Based Regimen Either in Combination With Cyclosporin A or Tacrolimus[NCT01843348] | Phase 3 | 612 participants (Actual) | Interventional | 2012-12-27 | Completed |
Decitabine Followed by Bone Marrow Transplant and High-Dose Cyclophosphamide for the Treatment of Relapsed and Refractory Acute Myeloid Neoplasms[NCT01707004] | Phase 2 | 20 participants (Actual) | Interventional | 2013-05-16 | Completed |
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 2 | 18 participants (Actual) | Interventional | 2003-05-31 | Terminated(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] |
Changes in GI Symptom Severity After Conversion From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS)
Changes in GI symptom severity was measured by changes in the Gastrointestinal Symptom Rating Scale (GSRS) total score from baseline visit to the visit at 6-8 weeks. This total score was calculated as the average of the 15 single items (each ranging from 1-7 score points) and thus also had a range from 1-7 score points. Higher values indicate more unfavorable conditions. (NCT00351377)
Timeframe: Baseline and 6 - 8 weeks
Intervention | Scores on a scale (Mean) |
---|
| Baseline (N= 111) | 6-8 Weeks (N= 102) | Change From Baseline (N = 102) |
---|
Enteric-coated Mycophenolate Sodium | 2.28 | 2.02 | -0.28 |
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Changes in Psychological General Well-Being Index (PGWB) After Conversion to Enteric-coated Mycophenolate Sodium
The PGWB consists of 22 single items (each ranging from 0-5) with 7 dimensions (including the total score) to be calculated. Lower scores indicate more unfavorable conditions. The total raw score is calculated by summing up all of the single items and thus has a hypothetical range from 0-110 score points. This raw score is further transformed using the formula: (raw score / 110) x 100 to fit a range from 0-100. (NCT00351377)
Timeframe: Baseline and 6-8 weeks
Intervention | Scores on a scale (Mean) |
---|
| Baseline (N= 110) | 6-8 Weeks (N= 103) | Change From Baseline (N= 103) |
---|
Enteric-coated Mycophenolate Sodium | 65.3 | 66.8 | 2.0 |
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Changes in Psychological General Well-Being Index (PGWB) Subscales After Conversion to Enteric-coated Mycophenolate Sodium
The change from baseline to the 6-8 week visit for each of the six subscores (each ranging from 0-5) of the PGWB were analyzed individually. Each of the subscores was transformed to fit a range from 0-100. Lower scores indicate more unfavorable conditions, so an increase in score indicates an improvement in symptoms. (NCT00351377)
Timeframe: Baseline and 6-8 weeks
Intervention | Scores on a scale (Mean) |
---|
| Anxiety [n=103] | Depressed mood [n=104] | Positive well-being [n=104] | Self control [n=104] | General health [n=104] | Vitality [n=104] |
---|
Enteric-coated Mycophenolate Sodium | 1.3 | 1.5 | 0.7 | 1.5 | 3.4 | 2.7 |
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Changes in the GI Symptom Severity Subscales After Conversion to Enteric-coated Mycophenolate Sodium
Changes in GI symptom severity was measured by changes in the total scores of 5 subscales (reflux, diarrhea, constipation, abdominal pain and indigestion) of the Gastrointestinal Symptom Rating Scale (GSRS) from baseline visit to the visit at 6-8 weeks. The GSRS is a 15-item instrument with a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). (NCT00351377)
Timeframe: Baseline and 6-8 weeks
Intervention | Scores on a scale (Mean) |
---|
| Reflux [n =102] | Diarrhea [n =104] | Constipation [n = 104] | Abdominal pain [n =102] | Indigestion [n = 104] |
---|
Enteric-coated Mycophenolate Sodium | -0.24 | -0.27 | 0.02 | -0.51 | -0.42 |
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Overall Treatment Effects for GI Symptoms Assessed by the Patient
"Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms. The possible answers were: Improved, about the same, or worse. The questionnaire was completed by the patient." (NCT00351377)
Timeframe: 6-8 week
Intervention | Participants (Number) |
---|
| Improved | About the same | Worsened | Missing data |
---|
Enteric-coated Mycophenolate Sodium | 38 | 53 | 9 | 11 |
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Overall Treatment Effects for GI Symptoms Assessed by the Physician
"Assessed using the Overall Treatment Effects for GI symptoms questionnaire. The question was: Has there been any change in the participant's GI symptoms since his/her last study visit? Please indicate if there has been any change in his/her symptoms. The possible answers were: Improved, about the same, or worse. The questionnaire was completed by the physician." (NCT00351377)
Timeframe: 6-8 week
Intervention | Participants (Number) |
---|
| Improved | About the same | Worsened | Missing data |
---|
Enteric-coated Mycophenolate Sodium | 49 | 43 | 13 | 6 |
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Acute Graft-Versus-Host Disease
Grade III-IV graft versus host disease (NCT00303719)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
High Risk Patients | 2 |
Standard Risk Patients | 79 |
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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
Intervention | Participants (Count of Participants) |
---|
High Risk Patients | 12 |
Standard Risk Patients | 289 |
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Overall Survival
(NCT00303719)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
High Risk Patients | 8 |
Standard Risk Patients | 181 |
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Serious Adverse Events
Safety by development of severe adverse events within 100 days of transplant (NCT00303719)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
High Risk Patients | 0 |
Standard Risk Patients | 47 |
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Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0
(NCT00423514)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Dose Level 1: Clofarabine at 20 mg/m^2/Dose x 5 + THIO-MEL | 31 |
Dose Level 2: Clofarabine at 30 mg/m^2/Dose x 5 + THIO-MEL | 7 |
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British Isles Lupus Assessment Group (BILAG) Index Score Over 52 Weeks
The BILAG Index assesses 86 clinical signs and symptoms and laboratory measures of systemic lupus erythematosus in 8 organ system domains: General, mucocutaneous, neurological, musculoskeletal, cardiorespiratory, vasculitis, renal, and hematologic. Most of the 86 items are rated on the following scale: 0=Not present, 1=Improving, 2=Same, 3=Worse, 4=New. Some items are rated as either Yes or No. A single alphabetic score of A (very active) through E (not or never active) for each of the 8 domains is determined from the rating of the individual items in each domain. The total BILAG score is the sum of the scores of the 8 domains where A=9, B=3, C=1, D=0, and E=0. The total score ranges from 0 to 72 with a higher score indicating greater lupus activity. To calculate a BILAG score over the 52 week treatment period of the study, the area under the response-time curve of BILAG scores assessed every 4 weeks was divided by the number of days in the time curve minus the Baseline BILAG score. (NCT00282347)
Timeframe: Baseline to Week 52
Intervention | Units on a scale (Mean) |
---|
Rituximab | -8.49 |
Placebo | -8.58 |
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Change From Baseline in Anti-double-stranded DNA at Week 52
(NCT00282347)
Timeframe: Baseline to Week 52
Intervention | IU/mL (Mean) |
---|
Rituximab | 0.45 |
Placebo | 1.06 |
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Change From Baseline in the Systemic Lupus Erythematosus Expanded Health Survey Physical Function Score at Week 52
The systemic lupus erythematosus Expanded Health Survey is based on the Short Form 36 Health survey with additional questions specific to lupus. The physical function component score of the survey can range from 0-100. A higher score indicates better health. A positive change score indicates improvement. (NCT00282347)
Timeframe: Baseline to Week 52
Intervention | Units on a scale (Mean) |
---|
Rituximab | 4.8 |
Placebo | 5.7 |
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Percentage of Participants Who Achieved a Complete Renal Response at Week 24 and Maintained it to Week 52
A participant had a complete renal response if they met the following 3 criteria: (1) Normalization of serum creatinine as evidenced by a serum creatinine level ≤ the upper limit of the normal range of central laboratory values or a serum creatinine level ≤ 15% greater than Baseline, if Baseline serum creatinine was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field and absence of red cell casts; (3) Urinary protein to creatinine ratio < 0.5. (NCT00282347)
Timeframe: Week 24 to Week 52
Intervention | Percentage of participants (Number) |
---|
Rituximab | 1.4 |
Placebo | 6.9 |
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Percentage of Participants Who Achieved a Complete Renal Response at Week 52
A participant had a complete renal response if they met the following 3 criteria: (1) Normalization of serum creatinine as evidenced by a serum creatinine level ≤ the upper limit of the normal range of central laboratory values or a serum creatinine level ≤ 15% greater than Baseline, if Baseline serum creatinine was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field and absence of red cell casts; (3) Urinary protein to creatinine ratio < 0.5. (NCT00282347)
Timeframe: Week 52
Intervention | Percentage of participants (Number) |
---|
Rituximab | 26.4 |
Placebo | 30.6 |
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Percentage of Participants With a Baseline Urine Protein to Creatinine Ratio of > 3.0 Who Achieved a Urine Protein to Creatinine Ratio of < 1.0 at Week 52
(NCT00282347)
Timeframe: Baseline to Week 52
Intervention | Percentage of participants (Number) |
---|
Rituximab | 47.4 |
Placebo | 53.7 |
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Time to Achieve a Complete Renal Response
(NCT00282347)
Timeframe: Baseline to Week 52
Intervention | Weeks (Median) |
---|
Rituximab | 11.99 |
Placebo | 12.12 |
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Change From Baseline in C3 and C4 Complement Levels at Week 52
(NCT00282347)
Timeframe: Baseline to Week 52
Intervention | mg/dL (Mean) |
---|
| C3 Complement | C4 Complement |
---|
Placebo | 25.9 | 6.6 |
,Rituximab | 37.5 | 9.9 |
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Percentage of Participants Who Achieved a Complete Renal Response (CRR), a Partial Renal Response (PRR), or no Renal Response (NRR) at Week 52
A participant had a CRR if they met the following 3 criteria: (1) Normalization of serum creatinine (SC) as evidenced by a SC level ≤ the upper limit of the normal range of central laboratory values or a SC level ≤ 15% greater than Baseline, if Baseline SC was within the normal range of the central laboratory values; (2) Inactive urinary sediment (as evidenced by < 5 red blood cells/high-power field (RBCs/HPF) and absence of red cell casts; (3) Urinary protein (UP) to creatinine ratio (CR) < 0.5. A participant had a PRR if they met the following 3 criteria: (1) A SC level ≤ 15% above Baseline; (2) RBCs/HPF ≤ 50% above Baseline and no RBC casts; (3) 50% improvement in the UP to CR, with 1 of the following conditions met: If the Baseline UP to CR was ≤ 3.0, then a UP to CR of < 1.0 or if the Baseline UP to CR was > 3.0, then a UP to CR of ≤ 3.0. A participant had a NRR if they did not achieve either a CRR or PRR. (NCT00282347)
Timeframe: Week 52
Intervention | Percentage of participants (Number) |
---|
| CRR | PRR | NRR |
---|
Placebo | 30.6 | 15.3 | 54.2 |
,Rituximab | 26.4 | 30.6 | 43.1 |
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Percentage of Participants With Acute Graft-vs-host-disease (GVHD)
Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). Acute GVHD is graded by consensus grading (Przepiorka 1995) per BMTCTN manual of procedures (MOP). The time of onset of grades II-IV and grades III-IV acute GVHD were recorded. This endpoint is evaluated through 100 days post-transplant. Cumulative percentage of acute GVHD post-transplant are estimated using the cumulative incidence function, treating death prior to acute GVHD as the competing risk. (NCT02918292)
Timeframe: Day 100
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 16.1 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 25.8 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 77.4 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 80.6 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 77.4 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 12.9 |
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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
Intervention | percentage of participants (Number) |
---|
Haplo Bone Marrow HSCT | 3.2 |
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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
Intervention | infections (Number) |
---|
| Bacterial infection | Viral infection | Fungal infection | Protozoal infection | Other infection |
---|
Haplo Bone Marrow HSCT | 26 | 32 | 3 | 0 | 3 |
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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
Intervention | cells/uL (Mean) |
---|
| CD3 at Baseline | CD3 at Day 100 | CD3 at 6 Months | CD3 at 1 Year | CD4 at Baseline | CD4 at Day 100 | CD4 at 6 Months | CD4 at 1 Year | CD8 at Baseline | CD8 at Day 100 | CD8 at 6 Months | CD8 at 1 Year | CD19 at Baseline | CD19 at Day 100 | CD19 at 6 Months | CD19 at 1 Year | CD56 at Baseline | CD56 at Day 100 | CD56 at 6 Months | CD56 at 1 Year |
---|
Haplo Bone Marrow HSCT | 862 | 550.8 | 640.5 | 1121 | 434.1 | 122.3 | 172.6 | 472.7 | 326.9 | 272.9 | 333.3 | 569.7 | 106.2 | 221.1 | 204.8 | 264.6 | 124.6 | 237.6 | 260.3 | 293.2 |
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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
Intervention | mg/dL (Mean) |
---|
| IgA at Baseline | IgA at 1 Year | IgG at Baseline | IgG at 1 Year | IgM at Baseline | IgM at 1 Year |
---|
Haplo Bone Marrow HSCT | 172.3 | 111.6 | 987.5 | 1004 | 102.8 | 96 |
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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
Intervention | Participants (Count of Participants) |
---|
| Abnormal Liver Symptoms | Blood and Lymphatic Disorders | Cardiovascular Disorders | Chemistry/Investigations | GI Disorders | General Disorders | Hemorrhagic Disorders | Hepatic Disorders | Immune System Disorders | Metabolism and Nutrition Disorders | Musculoskeletal and Connective Tissue Disorders | Nervous System Disorders | Renal Disorders | Respiratory, Thoracic and Mediastinal Disorders | Total (any of above SOC) |
---|
Haplo Bone Marrow HSCT | 7 | 1 | 15 | 2 | 10 | 5 | 3 | 6 | 1 | 7 | 1 | 4 | 5 | 8 | 23 |
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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
Intervention | percentage of participants (Number) |
---|
| Cumulative Percentage of Participants with EBV | Cumulative Percentage of Participants with CMV | Cumulative Percentage of Participants with PTLD |
---|
Haplo Bone Marrow HSCT | 9.7 | 22.6 | 6.5 |
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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
Intervention | percentage of participants (Number) |
---|
| Day 28 | Day 56 |
---|
Haplo Bone Marrow HSCT | 93.5 | 93.5 |
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Graft Survival at One Year
"Graft survival defined as any participant who did not meet the criteria for graft loss, where graft loss is defined as any re-transplant, permanent return to dialysis (> 30 days), patient death, or participant whose outcome at one year was unknown.~Participants were only counted once regardless of how many criteria were met." (NCT00064701)
Timeframe: One year
Intervention | percentage of participants (Number) |
---|
Tacrolimus | 91.5 |
Tacrolimus Modified Release | 95.3 |
Cyclosporine | 95.3 |
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Kaplan-Meier Estimate of Graft Survival at the End of the Study
"Graft survival was defined as any participant who did not meet the definition of graft loss, where graft loss was any retransplant or the permanent return to dialysis (more than 30 days) or patient death.~Graft survival was censored at the time of last follow-up contact." (NCT00064701)
Timeframe: End of study (maximum time on study was 1,941 days).
Intervention | percentage of participants (Number) |
---|
Tacrolimus | 82.7 |
Tacrolimus Modified Release | 84.7 |
Cyclosporine | 83.9 |
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Kaplan-Meier Estimate of Patient Survival at the End of the Study
Patient survival was defined as any participant who was alive at the end of the study. Patient survival was censored at the time of last follow-up contact. (NCT00064701)
Timeframe: End of study (maximum time on study was 1,941 days).
Intervention | percentage of participants (Number) |
---|
Tacrolimus | 91.2 |
Tacrolimus Modified Release | 93.2 |
Cyclosporine | 91.7 |
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Number of Participants Experiencing Multiple Rejection Episodes
This analysis includes rejection episodes that were either confirmed by biopsy by the clinical site pathologist or were clinically treated. (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
Tacrolimus | 2 |
Tacrolimus Modified Release | 4 |
Cyclosporine | 8 |
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Number of Participants Requiring Anti-lymphocyte Antibody Therapy for Treatment of Rejection
"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Participants with histologically-proven Banff Grade II or III rejection or participants with steroid-resistant rejection were treated with anti-lymphocyte antibody treatment according to institutional practice.~Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
Tacrolimus | 6 |
Tacrolimus Modified Release | 8 |
Cyclosporine | 18 |
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Number of Participants Who Crossed Over Due to Treatment Failure
Participants were allowed to cross over to an alternative primary immunosuppressive regimen (either to the tacrolimus or cyclosporine treatment arms) to address an adverse event which led to randomized study drug discontinuation or in the case of severe or refractory rejection. Crossover to the modified release tacrolimus treatment arm was not permitted. (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
Tacrolimus | 6 |
Tacrolimus Modified Release | 10 |
Cyclosporine | 39 |
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Number of Participants With Clinically Treated Acute Rejection Episodes
A clinically treated acute rejection episode was any biopsy-confirmed or suspected rejection episode that was treated with immunosuppressive therapy. (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
Tacrolimus | 25 |
Tacrolimus Modified Release | 39 |
Cyclosporine | 45 |
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Number of Participants With Treatment Failure
Treatment failure was defined as the discontinuation of randomized study drug for any reason. Participants who met the definition of treatment failure were to be followed throughout the 12-month treatment period. (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
Tacrolimus | 33 |
Tacrolimus Modified Release | 31 |
Cyclosporine | 61 |
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Patient Survival at One Year
Patient survival is defined as any participant who is known to be alive one year after the skin closure date. Participants who died or whose outcome was unknown at one year were considered to be non-survivors. (NCT00064701)
Timeframe: One year
Intervention | percentage of participants (Number) |
---|
Tacrolimus | 93.9 |
Tacrolimus Modified Release | 97.2 |
Cyclosporine | 97.2 |
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Percentage of Participants With Efficacy Failure
"Efficacy failure is defined as any participant who died, experienced a graft failure (permanent return to dialysis [> 30 days] or retransplant), had a biopsy-confirmed (Banff Grade ≥ I) acute rejection (BCAR), or was lost to follow-up.~Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year
Intervention | percentage of participants (Number) |
---|
Tacrolimus | 15.1 |
Tacrolimus Modified Release | 14.0 |
Cyclosporine | 17.0 |
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Time to First Biopsy-confirmed Acute Rejection Episode
"Time to first biopsy-confirmed acute rejection episode defined as the number of days from skin closure (Day 0) to the date of biopsy. Rejection episodes were confirmed by biopsy by the clinical site pathologist and graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel.~Acute rejection is defined as a grade ≥ I." (NCT00064701)
Timeframe: one year
Intervention | days (Median) |
---|
Tacrolimus | 156.00 |
Tacrolimus Modified Release | 11.00 |
Cyclosporine | 52.00 |
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Change From Month 1 in Creatinine Clearance at Month 6 and Month 12
Renal function was assessed by creatinine clearance, calculated using the Cockcroft-Gault formula. (NCT00064701)
Timeframe: Month 1, Month 6, and Month 12
Intervention | mL/min (Mean) |
---|
| At 6 months [N=184, 184, 167] | At 12 months [N=173, 182, 145] |
---|
Cyclosporine | -1.79 | -0.25 |
,Tacrolimus | 0.83 | 1.50 |
,Tacrolimus Modified Release | 0.47 | 2.62 |
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Change From Month 1 in Serum Creatinine at Month 6 and Month 12
Renal function was assessed by the change from Month 1 in serum creatinine six months and 12 months after transplant. (NCT00064701)
Timeframe: Month 1, Month 6, and Month 12
Intervention | mg/dL (Mean) |
---|
| At 6 months [N=184, 184, 169] | At 12 months [N=173, 182, 147] |
---|
Cyclosporine | -0.01 | -0.04 |
,Tacrolimus | -0.09 | -0.08 |
,Tacrolimus Modified Release | -0.08 | -0.14 |
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Percentage of Participants With Biopsy Confirmed Acute Rejection at 6 and 12 Months
"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade I: Significant interstitial infiltration and foci of moderate to severe tubulitis; Grade II: Mild to severe intimal arteritis Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel.~Acute rejection is defined as a grade ≥ I." (NCT00064701)
Timeframe: Six months and 12 months
Intervention | percentage of participants (Number) |
---|
| At 6 Months | At 12 Months |
---|
Cyclosporine | 11.8 | 13.7 |
,Tacrolimus | 3.8 | 7.5 |
,Tacrolimus Modified Release | 7.9 | 10.3 |
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Severity of Acute Rejection
"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 1997 Banff criteria:~Borderline: No intimal arteritis present but foci of mild tubulitis; Grade IA: Significant interstitial infiltration and foci of moderate tubulitis; Grade IB: Significant interstitial infiltration and foci of severe tubulitis; Grade IIA: Mild to moderate intimal arteritis in at least 1 arterial cross section Grade IIB: Severe intimal arteritis comprising >25% of the luminal area lost in at least 1 arterial cross section; Grade III: Transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic infiltrate in vessel." (NCT00064701)
Timeframe: one year
Intervention | participants (Number) |
---|
| Grade I-A | Grade I-B | Grade II-A | Grade II-B | Grade III |
---|
Cyclosporine | 14 | 6 | 6 | 1 | 2 |
,Tacrolimus | 8 | 4 | 3 | 1 | 0 |
,Tacrolimus Modified Release | 11 | 3 | 6 | 1 | 1 |
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Number of Participants Who Developed Acute Rejection Episode Within 6 Months Post-Transplant
The acute rejection episode was a composite end-point of acute rejection and treatment failure within 6 months post-transplant (PT). Participants with acute rejection included participants with a biopsy histology of International Society of Heart and Lung Transplant (ISHLT) Grade IIIA, IIIB, or IV and participants with hemodynamic compromise (HDC) who were treated for acute rejection (whether or not a biopsy was done and regardless of the grade of the biopsy). Participants who had treatment failure included participants who died within 6 months of transplantation before experiencing acute rejection or who were re-transplanted within 6 months of the primary transplantation and who did not experience an acute rejection or who were lost to follow-up. (NCT00048165)
Timeframe: Up to 6 months PT
Intervention | participants (Number) |
---|
Daclizumab | 77 |
Placebo | 104 |
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Number of Participants Who Developed Acute Rejection Episode Within the 12 Months PT
The acute rejection episode was a composite end-point of acute rejection and treatment failure within 6 months. Participants with acute rejection included participants with a biopsy histology of ISHLT Grade IIIA, IIIB, or IV and participants with hemodynamic compromise (HDC) who were treated for acute rejection (whether or not a biopsy was done and regardless of the grade of the biopsy). Participants who had treatment failure included participants who died within 6 months of transplantation before experiencing acute rejection or who were re-transplanted within 6 months of the primary transplantation and who did not experience an acute rejection or who were lost to follow-up (NCT00048165)
Timeframe: Up to 12 months PT
Intervention | participants (Number) |
---|
Daclizumab | 97 |
Placebo | 116 |
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Mean Maintenance Doses of Mycophenolate Mofetil, Cyclosporine, and Cumulative Dose of Corticosteroids at 6 and 12 Months PT
The maintenance doses of mycophenolate mofetil (1.5g twice a day daily), cyclosporine (1-4 mg/kg IV or 2-6 mg/kg oral [PO]/nasogastric [NG] within 72 hours post-operative]), and cumulative dose of corticosteroids (500-1000 mg IV methylprednisolone pre-operative switched to oral at 0.5-1 mg/kg/day. Tapered to 0.2 mg/kg/d by Day 28, 0.1-0.15 mg/kg/day from Days 36 to 90, and 0.1-0.15 mg/kg/day from Days 120 to 180)at 6 and 12 months PT were reported. Maintenance dose was calculated as total dose per day summed over all days that a participant was administered drug within the specified time interval, divided by number of days that a participant took drug within that time interval. (NCT00048165)
Timeframe: Within 6 months and 12 months PT
Intervention | mg (Mean) |
---|
| MMF dose, 6 months PT (n=193, 201) | MMF dose,12 months PT (n=188, 194) | IV Cyclosporine dose, 6 months PT (n=2, 1) | IV Cyclosporine dose, 12 months PT (n=4, 2) | PO/NG Cyclosporine dose, 6 months PT (n=184, 182) | PO/NG Cyclosporine dose, 12 months PT (n=170, 170) | Cumulative corticosteroids,6 months PT(n=203, 206) | Cumulative corticosteroids,12 months PT(n=195,200) |
---|
Daclizumab | 2522.2 | 2394.1 | 86.11 | 93.5 | 321.9 | 294.7 | 848.1 | 1199.6 |
,Placebo | 2450 | 2380.1 | 38.1 | 46.7 | 331.1 | 305.7 | 955.4 | 1288.9 |
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Number of Acute Rejection Episodes Per Participant Within the First 6 Months and 12 Months PT
The number of participants with 0,1, 2, 3 or 4 episodes at 6 and 12 months PT were reported. An episode of acute rejection was defined according to the date of positive biopsy of Grade IIIA or worse or the date of start of treatment for HDC, whichever came first. (NCT00048165)
Timeframe: Within 6 months and 12 months PT
Intervention | participants (Number) |
---|
| Within 6 months, 0 episode | Within 6 months, 1 episode | Within 6 months, 2 episodes | Within 6 months, 3 episodes | Within 6 months, 4 episodes | Within 12 months, 0 episode | Within 12 months, 1 episode | Within 12 months, 2 episodes | Within 12 months, 3 episodes | Within 12 months, 4 episodes |
---|
Daclizumab | 139 | 63 | 12 | 2 | 0 | 119 | 68 | 23 | 3 | 3 |
,Placebo | 114 | 82 | 19 | 2 | 1 | 102 | 90 | 19 | 5 | 2 |
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Number of Participant Who Died Within 6 Months 12 Months and 3 Years PT
The survival of the graft and participants at 6,12 months and 3 years PT was reported (NCT00048165)
Timeframe: At 6 months, 12 months , 3 years PT
Intervention | participants (Number) |
---|
| Within 6 months | Within 12 months | Within 3 years |
---|
Daclizumab | 16 | 21 | NA |
,Placebo | 10 | 12 | NA |
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Number of Participants Using Monomurab Cluster of Differentiation 3, Orthoclone Polyclonal Antithymocyte Globulin or Antilymphocyte Globulin in the First 6 Months and 12 Months PT
The number of participants who received monomurab Cluster of differentiation 3, orthoclone, polyclonal antithymocyte globulin or antilymphocyte globulin for treatment of biopsy proven rejection or HDC within 6 and 12 months PT were reported. (NCT00048165)
Timeframe: Within 6 months and 12 months PT
Intervention | participants (Number) |
---|
| Within 6 months | Within 12 months |
---|
Daclizumab | 17 | 23 |
,Placebo | 19 | 21 |
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Number of Participants With Any Adverse Events and Any Serious Adverse Event, and Adverse Events Leading to Premature Withdrawal
An adverse event (AE) is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Pre-existing conditions which worsened during this study were reported as AEs. A serious adverse event (SAE) is any untoward medical occurrence that at any dose results in death, are life threatening, requires hospitalization or prolongation of hospitalization or results in disability/incapacity, and congenital anomaly/birth defect. (NCT00048165)
Timeframe: Up to 12 months
Intervention | participants (Number) |
---|
| Any AEs | Any SAE's | Any AEs leading to premature discontinuation |
---|
Daclizumab | 214 | 108 | 14 |
,Placebo | 207 | 102 | 11 |
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Number of Participants With Malignancies and Opportunistic Infections
The opportunistic infections included infections with Cytomegalovirus, Aspergillus, Candida, Pneumocystis, Cryptococcus, Listeria, Herpes simplex, Herpes zoster. For malignancy, participants with any type of malignancy whose date of onset or diagnosis was after randomization was reported. (NCT00048165)
Timeframe: Up to 12 months
Intervention | participants (Number) |
---|
| Participants with malignancies | Participants with opportunistic infections |
---|
Daclizumab | 11 | 71 |
,Placebo | 11 | 80 |
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Number of Participants With Marked Laboratory Abnormalities: Biochemistry Parameters
A marked reference range was predefined by Roche. The marked reference range is broader than the standard reference range. Values falling outside the marked reference range (low or high) that also represent a defined change from Baseline were considered marked laboratory abnormalities (i.e. potentially clinically relevant). Biochemistry included blood urea nitrogen, creatinine, serum glutamic oxalacetic transaminase (SGOT), serum glutamic pyruvic transaminase (SGPT), gamma-glutamyl transferase (GGT), phosphorous, total bilirubin, direct bilirubin, total protein, albumin, glucose, alkaline phosphatase, low density lipoprotein (LDH), uric acid, carbon dioxide, magnesium, sodium, potassium, chloride, calcium, LDL, HDL, total cholesterol, and triglycerides. (NCT00048165)
Timeframe: Up to 12 months
Intervention | participants (Number) |
---|
| SGPT-high (n=200, 200) | ALP-high (n=201, 200) | SGOT-high (n=201, 200) | GGT-high (n=180, 175) | LDH-high (n=194, 191) | Total bilirubin-high (n=201, 200) | BUN-high (n= 210, 200) | Creatinine-high (n=211, 203) | Albumin-low (n=198,197) | Total protein-high (n=195,196) | Total protein-low(n=195,196) | Cholesterol-high (n=174, 174) | Triglycerides-high (n=164, 164) | Carbondioxide-high (n=206, 197) | Carbondioxide-low (n=206, 197) | Chloride-high (n=211, 203) | Chloride-low (n=211, 203) | Potassium-high (n=211, 204) | Potassium-low (n=211, 204) | Sodium-high (n=211, 203) | Sodium-low (n=211, 203) | Calcium-low (n=207, 201) | Glucose fasting-high (n=210, 203) | Glucose fasting-low (n=210, 203) | Phosphate-high (n=199, 194) | Phosphate-low (n=199,194) | Uric acid high (n=191, 188) |
---|
Daclizumab | 48 | 15 | 22 | 90 | 53 | 28 | 93 | 66 | 47 | 5 | 85 | 3 | 35 | 27 | 12 | 1 | 42 | 7 | 4 | 2 | 8 | 39 | 28 | 3 | 54 | 32 | 23 |
,Placebo | 45 | 7 | 25 | 74 | 54 | 20 | 95 | 64 | 50 | 0 | 79 | 4 | 30 | 21 | 5 | 3 | 36 | 7 | 2 | 1 | 11 | 44 | 27 | 3 | 48 | 26 | 20 |
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Number of Participants With Marked Laboratory Abnormalities: Hematology Parameters
A marked reference range was predefined by Roche. The marked reference range is broader than the standard reference range. Values falling outside the marked reference range (low or high) that also represent a defined change from Baseline were considered marked laboratory abnormalities (i.e. potentially clinically relevant). Hematology included hemoglobin, hematocrit, white blood cell count (WBC) with differential (including granulocytes or neutrophils, basophils, eosinophils, monocytes, lymphocytes), platelets, and erythrocyte count (NCT00048165)
Timeframe: Up to 12 months
Intervention | participants (Number) |
---|
| Hematocrit-high (n=210, 203) | Hematocrit-low (n=210, 203) | Hemoglobin-high (n=210, 204) | Hemoglobin-low (n=210, 204) | Platelets-high (n=210, 203) | Platelets-low (n=210, 203) | RBC-high (n=209, 203) | RBC-low (n=209, 203) | Basophils-high (n=199, 195) | Eosinophils-high (n=199, 195) | Lymphocytes-high (n=199, 198) | Lymphocytes-low (n=199, 198) | Monocytes-high (n=199, 198) | Monocytes-low (n=199, 198) | Neutrophils-low (n= 199, 198) | WBC-high (n=207, 201) | WBC-low (n=207, 201) |
---|
Daclizumab | 0 | 116 | 0 | 107 | 2 | 30 | 1 | 116 | 4 | 0 | 2 | 157 | 5 | 14 | 33 | 61 | 43 |
,Placebo | 0 | 117 | 0 | 112 | 1 | 22 | 2 | 106 | 4 | 0 | 2 | 157 | 6 | 20 | 33 | 57 | 29 |
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Number of Participants With Worst ISHLT Biopsy Grade Within First 6 Months and 12 Months PT
The number of participants with Worst International Society of Heart and Lung Transplant (ISHLT) grade within first 6 months and 12 months PT were reported. ISHLT is a standardized grading method to determine the acute cellular rejection on endomyocardial biopsy ; where 0= no rejection, IA= focal (perivascular or interstitial) infiltrate without necrosis, IB= diffuse but sparse infiltrate without necrosis, II=one focus only with aggressive infiltration and/or focal myocyte damage, IIIA=multifocal aggressive infiltrates and/or myocyte damage, IIIB= diffuse inflammatory process with necrosis, IV= diffuse aggressive polymorphous and/or infiltrate and/or edema and/or hemorrhage and/or vasculitis, with necrosis (NCT00048165)
Timeframe: Within 6 months and 12 months PT
Intervention | participants (Number) |
---|
| Within 6 months, Grade 0 | Within 6 months, Grade IA | Within 6 months, Grade IB | Within 6 months, Grade II | Within 6 months, Grade IIIA | Within 6 months, Grade IIIB | Within 6 months, Grade IV | Within 12 months, Grade 0 | Within 12 months, Grade IA | Within 12 months, Grade IB | Within 12 months, Grade II | Within 12 months, Grade IIIA | Within 12 months, Grade IIIB | Within 12 months, Grade IV |
---|
Daclizumab | 9 | 64 | 26 | 55 | 48 | 8 | 1 | 7 | 56 | 22 | 51 | 63 | 11 | 1 |
,Placebo | 5 | 51 | 28 | 38 | 74 | 15 | 1 | 4 | 39 | 21 | 47 | 85 | 15 | 1 |
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Cumulative Incidence of a Severe/Life-threatening/Fatal Infections
(NCT01002742)
Timeframe: Year 1
Intervention | percentage of participants (Number) |
---|
Placebo | 42.9 |
Mycophenolate Mofetil | 44.5 |
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Disease-Free Survival (DFS) Post-Randomization
DFS includes death or progression/relapse of malignancy (NCT01002742)
Timeframe: Year 1
Intervention | percentage of participants (Number) |
---|
Placebo | 63 |
Mycophenolate Mofetil | 53.9 |
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Incidence of Chronic GVHD
(NCT01002742)
Timeframe: 12 months post-randomization
Intervention | percentage of participants (Number) |
---|
Placebo | 43.3 |
Mycophenolate Mofetil | 41.5 |
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Incidence of Cytomegalovirus (CMV) Reactivation
(NCT01002742)
Timeframe: Year 1
Intervention | percentage of participants (Number) |
---|
Placebo | 39 |
Mycophenolate Mofetil | 44 |
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Incidence of Epstein-Barr Virus (EBV)-Associated Lymphoma
(NCT01002742)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Placebo | 4 |
Mycophenolate Mofetil | 6 |
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Incidence of GVHD Flares Requiring Increased Therapy
Flares are defined as any progression of acute GVHD after an initial response (i.e., earlier CR or PR) that requires re-escalation of steroid dosing, or initiation of additional topical or systemic therapy. (NCT01002742)
Timeframe: Day 90
Intervention | participants (Number) |
---|
Placebo | 16 |
Mycophenolate Mofetil | 8 |
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Incidence of Systemic Infections
Number of participants that experienced at least one infection. (NCT01002742)
Timeframe: 6 Months
Intervention | participants (Number) |
---|
Placebo | 77 |
Mycophenolate Mofetil | 81 |
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Incidence of Topical/Non-absorbable Therapy
(NCT01002742)
Timeframe: Day 56
Intervention | participants (Number) |
---|
Placebo | 81 |
Mycophenolate Mofetil | 77 |
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Cumulative Steroid Dose
The cumulative steroid dose for each patient will be calculated by adding the doses (end of each week's dose) for each of the first four weeks of treatment, divided by the number of days of survival during this interval. The cumulative steroid dose was calculated for all patients per treatment arm and compared. (NCT01002742)
Timeframe: Days 28 and 56
Intervention | mg/kg (Number) |
---|
| Day 28 | Day 56 |
---|
Mycophenolate Mofetil | 0.60 | 0.17 |
,Placebo | 0.63 | 0.20 |
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GVHD-free Survival
Success is defined as alive and free of GVHD at day 56 after randomization, all others are considered to be a study failure. (NCT01002742)
Timeframe: Day 56
Intervention | participants (Number) |
---|
| GVHD free | Study Failure |
---|
Mycophenolate Mofetil | 69 | 47 |
,Placebo | 60 | 59 |
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Overall GVHD-free Survival Post-randomization
(NCT01002742)
Timeframe: Months 6 and 12
Intervention | percentage of participants (Number) |
---|
| 6 Months | 12 Months |
---|
Mycophenolate Mofetil | 72.0 | 57.8 |
,Placebo | 73.4 | 64.7 |
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Percentage of Surviving Participants With Complete Response (CR)
CR is defined as a score of 0 for the GVHD grading in all evaluable organs. (NCT01002742)
Timeframe: Days 14, 28, and 56
Intervention | percentage of participants (Number) |
---|
| Day 14 | Day 28 | Day 56 |
---|
Mycophenolate Mofetil | 44 | 46.6 | 60.3 |
,Placebo | 49.6 | 44.5 | 53.8 |
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Blood CD4+ T Cells Per mm^3 Blood
Frequency of participants with any time point with <200 CD4+ T cells per mm^3 from 4 sampled time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 0 |
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Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 Effector Memory CD4+ T Cells Over 12 Months
Regression slope of change in cell-associated HIV DNA (ca-DNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 3 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months
Intervention | log10 caDNA copies per 10^6 T-cells/week (Mean) |
---|
Mycophenolate Mofetil | 0.001 |
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Change in Cell-associated HIV DNA (Ca-DNA) Levels Per 10^6 T Cells Over 12 Months
Regression slope of change in cell-associated HIV DNA (ca-DNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 4 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months
Intervention | log10 caDNA copies per 10^6 T-cells/week (Mean) |
---|
Mycophenolate Mofetil | -0.00033 |
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Change in Cell-associated Intact HIV DNA (Ca-iDNA) Levels Per 10^6 T Cells Over 12 Months
Regression slope of change in cell-associated intact HIV DNA (ca-iDNA) as measured by multiplexed digital droplet PCR in study participants on MMF calculated from 4 time points between 0 & 12 months (NCT03262441)
Timeframe: 12 months
Intervention | log10 caDNA copies per 10^6 T-cells/week (Mean) |
---|
Mycophenolate Mofetil | .0024 |
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Incidence of Opportunistic Infection
Number of participants experiencing opportunistic infection (NCT03262441)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 18 |
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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
Intervention | days (Median) |
---|
Bone Marrow Transplant | 18.85 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 17 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 7 |
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Number of Patients That Have Survived at One Year
(NCT02224872)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 18 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 17 |
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Participants That Were GVHD Free, Relapse Free Survival (GRFS)
(NCT02224872)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 14 |
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Participants With Chronic GVHD at One Year
(NCT02224872)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 3 |
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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
Intervention | percentage 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
Intervention | percentage of surviving patients (Number) |
---|
Regimen I (FTBI, Cyclophosphamide, Fludarabine) | 60 |
Regimen III (TBI, Cyclophosphamide, Fludarabine) | 100 |
Regimen IV (Fludarabine, Melphalan) | 50 |
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Percentage of Participants With Efficacy Failure Through 12 Months Post Transplant
Efficacy failure was defined as BPAR, death, graft loss or lost to follow-up through 12 months post transplant. (NCT02921789)
Timeframe: 12 Months post transplant
Intervention | Percentage of participants (Number) |
---|
SOC Regimen | 32.3 |
Bleselumab Regimen | 32.0 |
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Percentage of Participants With Recurrence of Focal Segmental Glomerulosclerosis (rFSGS) or Death or Graft Loss or Lost to Follow-up Through 3 Months Post Transplant
rFSGS was defined as nephrotic range proteinuria with a protein/creatinine ratio (≥ 3.0 g/g). Death, graft loss or lost to follow-up was imputed as rFSGS. (NCT02921789)
Timeframe: At 3 Months post transplant
Intervention | Percentage of participants (Number) |
---|
SOC Regimen | 31.3 |
Bleselumab Regimen | 18.5 |
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Percentage of Participants With Biopsy Proven rFSGS Through 3, 6 and 12 Months Post-Transplant
Percentage of participants with biopsy-proven rFSGS determined by a blinded central review of images from electron microscopy (EM) and slides for light microscopy (LM) by an independent pathologist. (NCT02921789)
Timeframe: At 3, 6 and 12 Months post transplant
Intervention | Percentage of participants (Number) |
---|
| Month 3 | Month 6 | Month 12 |
---|
Bleselumab Regimen | 31.8 | 30.4 | 29.2 |
,SOC Regimen | 35.7 | 36.7 | 36.7 |
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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Through 3, 6, and 12 Months Post Transplant
All episodes of kidney dysfunction based on clinical signs and symptoms were evaluated for possible BPAR. BPAR was confirmed if participants Banff criteria >=1. (NCT02921789)
Timeframe: At 3, 6 and 12 Months post transplant
Intervention | Percentage of participants (Number) |
---|
| Month 3 | Month 6 | Month 12 |
---|
Bleselumab Regimen | 26.9 | 26.9 | 29.2 |
,SOC Regimen | 20.0 | 20.0 | 24.1 |
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Percentage of Participants With rFSGS or Death or Graft Loss or Lost to Follow-up Through 6 and 12 Months Post Transplant
rFSGS was defined as nephrotic range proteinuria with a protein/creatinine ratio (≥ 3.0 g/g). Death, graft loss or lost to follow-up was imputed as rFSGS. (NCT02921789)
Timeframe: At 6 and 12 Months post transplant
Intervention | Percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Bleselumab Regimen | 18.5 | 23.1 |
,SOC Regimen | 31.3 | 35.5 |
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Cumulative Incidence of Systemic Infections
(NCT00224874)
Timeframe: Measured at Day 270
Intervention | percentage of participants (Number) |
---|
Etanercept | 47 |
Mycophenolate Mofetil | 44 |
Denileukin Diftitox | 62 |
Pentostatin | 57 |
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Number of Complete Response (CR) at Day 28 of Therapy
Complete response at day 28 after randomization. CR was defined as resolution of all signs and symptoms of Graft-Versus-Host Disease (GVHD) in all evaluable organs in comparison to Day 1 scoring. (NCT00224874)
Timeframe: Measured at Day 28
Intervention | participants (Number) |
---|
Etanercept | 12 |
Mycophenolate Mofetil | 27 |
Denileukin Diftitox | 25 |
Pentostatin | 16 |
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Number of Patients With Acute Graft-versus-host Disease (GVHD) Flares at Day 90
Flares were defined as any increase in symptoms of or therapy for acute GVHD after an initial response (i.e., progression from an earlier CR or PR). (NCT00224874)
Timeframe: Measured at Day 90
Intervention | participants (Number) |
---|
Etanercept | 16 |
Mycophenolate Mofetil | 12 |
Denileukin Diftitox | 15 |
Pentostatin | 15 |
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Number of Patients With Chronic Graft-versus-host Disease (GVHD)
Number of patients with limited and extensive chronic GVHD at 9 months (NCT00224874)
Timeframe: Measured at 9 months
Intervention | participants (Number) |
---|
Etanercept | 11 |
Mycophenolate Mofetil | 19 |
Denileukin Diftitox | 15 |
Pentostatin | 12 |
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Proportion of Treatment Failure
(NCT00224874)
Timeframe: Measured at Day 56
Intervention | percentage of participants (Number) |
---|
Etanercept | 24 |
Mycophenolate Mofetil | 9 |
Denileukin Diftitox | 26 |
Pentostatin | 29 |
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Number of Partial Response (PR), Mixed Response (MR), and Progression
Partial response, mixed response, and progression at Day 28 after randomization. Partial response was defined as improvement in one or more organs involved with Graft-Versus-Host Disease (GVHD) symptoms without progression in others. Mixed response was defined as improvement in one or more organs with deterioration in another organ manifesting symptoms of GVHD or development of symptoms of GVHD in a new organ. Progression was defined as deterioration in at least one organ without any improvement in others. (NCT00224874)
Timeframe: Measured at Day 28
Intervention | participants (Number) |
---|
| Partial Response | Mixed Response | Progression |
---|
Denileukin Diftitox | 3 | 0 | 3 |
,Etanercept | 10 | 3 | 7 |
,Mycophenolate Mofetil | 8 | 4 | 1 |
,Pentostatin | 10 | 2 | 4 |
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Number of Patients Discontinuing Immune Suppression Without Flare
Immunosuppression discontinuation was defined as the discontinuation of corticosteroids and all additional immunosuppressives, except cyclosporine or tacrolimus, for treatment of acute GVHD without subsequent flare by Day 90 post-initiation of therapy and later by discontinuation of all immunosuppressive medications, including cyclosporine or tacrolimus. (NCT00224874)
Timeframe: Measured at Days 90, 180, and 270 post-treatment
Intervention | participants (Number) |
---|
| Day 90 | Day 180 | Day 270 |
---|
Denileukin Diftitox | 5 | 8 | 10 |
,Etanercept | 7 | 12 | 16 |
,Mycophenolate Mofetil | 4 | 13 | 17 |
,Pentostatin | 2 | 8 | 10 |
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Number of Patients Surviving at 6 and 9 Months Post Randomization
(NCT00224874)
Timeframe: Measured at 6 and 9 months
Intervention | participants (Number) |
---|
| Month 6 | Month 9 |
---|
Denileukin Diftitox | 28 | 24 |
,Etanercept | 26 | 22 |
,Mycophenolate Mofetil | 32 | 29 |
,Pentostatin | 24 | 21 |
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Acute Graft vs Host Disease (GvHD)
"The incidence of acute GvHD after transplantation was assessed per Glucksberg GvHD grade, a compound scale based on the following combinations of disease stages.~Skin Stages~0: No rash~1: Maculopapular (MP) rash <25% of body surface area~2: MP rash on 25-50% of body surface area~3: Generalized erythroderma (ED)~4: Generalized ED with bullous formation and desquamation~Liver Stages (Bilirubin in mg/dL)~0: <2~1: 2-3~2: 3.01-6~3: 6.01-15.0~4: >15~Gastrointestinal (GI) Stages (diarrhea)~0: None or < 500 mL/day~1: 500-999 mL/day~2: 1000-1499 mL/day~3: >1500 mL/day~4: Severe abdominal pain, with or without ileus~Glucksberg Overall grade~Grade 1: Skin 1/2; GI 0; Liver 0; Karnofsky performance scale (KPS) 90-100%~Grade 2: Skin 1-3; GI 1; Liver 1; KPS 70-80~Grade 2: Skin 2/3; GI 2/3; Liver 2-4; KPS 50-60~Grade 4: Skin 2-4; GI 2-4; Liver 2-4; KPS 30-40" (NCT00185640)
Timeframe: 100 days post-transplant
Intervention | percentage of participants (Number) |
---|
Non-myeloablative Transplantation | 2.7 |
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Acute Graft vs Host Disease (GvHD), All Evaluable
"The incidence of acute GvHD after transplantation was assessed per Glucksberg GvHD grade, a compound scale based on the following combinations of disease stages.~Skin Stages~0: No rash~1: Maculopapular (MP) rash <25% of body surface area~2: MP rash on 25-50% of body surface area~3: Generalized erythroderma (ED)~4: Generalized ED with bullous formation and desquamation~Liver Stages (Bilirubin in mg/dL)~0: <2~1: 2-3~2: 3.01-6~3: 6.01-15.0~4: >15~Gastrointestinal (GI) Stages (diarrhea)~0: None or < 500 mL/day~1: 500-999 mL/day~2: 1000-1499 mL/day~3: >1500 mL/day~4: Severe abdominal pain, with or without ileus~Glucksberg Overall grade~Grade 1: Skin 1/2; GI 0; Liver 0; Karnofsky performance scale (KPS) 90-100%~Grade 2: Skin 1-3; GI 1; Liver 1; KPS 70-80~Grade 2: Skin 2/3; GI 2/3; Liver 2-4; KPS 50-60~Grade 4: Skin 2-4; GI 2-4; Liver 2-4; KPS 30-40" (NCT00185640)
Timeframe: 100 days post-transplant
Intervention | percentage of participants (Number) |
---|
Non-myeloablative Transplantation | 11 |
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Incidence of Relapse
Reports the overall rate of disease relapse, occurring any time within 3 years after transplant (NCT00185640)
Timeframe: 3 years
Intervention | percentage of participants (Number) |
---|
Non-myeloablative Transplantation | 53 |
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Event-free Survival (EFS)
Reports the number and proportion of participants who neither died due to any cause nor experienced relapse. (NCT00185640)
Timeframe: 3 and 5 years
Intervention | Participants (Count of Participants) |
---|
| 3 years | 5 years |
---|
Non-myeloablative Transplantation | 44 | 38 |
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Overall Survival (OS)
(NCT00185640)
Timeframe: 3 and 5 years
Intervention | percentage of participants (Number) |
---|
| 3 years | 5 years |
---|
Non-myeloablative Transplantation | 70 | 64 |
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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
Intervention | percent of participants (Number) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 40 |
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Incidence of Grades III/IV Acute Graft Versus Host Disease
Grading determined by organ system stages. Grade III/IV acute graft versus host disease is defined as skin: stage IV, liver: stages II-IV, and/or gastrointestinal tract: stages II-IV. (NCT01028716)
Timeframe: At day 84
Intervention | percent (Number) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 82 |
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Incidence of Primary Graft Failure
Defined as < 5% donor CD3 chimerism. Chimerism will be measured by short tandem repeat-polymerase chain reaction on peripheral blood sorted into CD3 and CD33 cell fractions. (NCT01028716)
Timeframe: At day 84
Intervention | Participants (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
Intervention | percent 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
Intervention | transfusions (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
Intervention | transfusions (Median) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 8 |
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Percentage of Participants With Chronic Graft Versus Host Disease
Scored according to the National Cancer Institute criteria. Mild-to-severe chronic GVHD at 2 years. (NCT01028716)
Timeframe: Up to 2 years post-transplant
Intervention | percentage of participants (Number) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 26 |
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Point Estimate of Overall Survival at 3 Years
Kaplan Meier estimate of the percentage of participants with overall survival at 3 years (NCT01028716)
Timeframe: 3 years
Intervention | percent (Number) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 45.3 |
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Relapse of Malignancy After Transplantation
Defined by either morphological or cytogenetic evidence of acute leukemia consistent with pre-transplant features, or radiologic evidence of lymphoma progression. When in doubt, the diagnosis of recurrent or progressive lymphoma should be documented by tissue biopsy. (NCT01028716)
Timeframe: Up to 7 years
Intervention | percent (Number) |
---|
Treatment (Nonmyeloablative HCT, TBI) | 29 |
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Time to Neutrophil Recovery
Achievement of an absolute neutrophil count greater or equal to 500/mm^3 for three consecutive measurements on different days. The first of the three days will be designated the day of neutrophil recovery. (NCT01028716)
Timeframe: Up to day 84 post-transplant
Intervention | days (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
Intervention | days (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
Intervention | events (Number) |
---|
| Cardiac | Fever | Rash | Gastrointestinal | Infections | CMV Reactivation | Febrile Neutropenia | Metabolic/laboratory | Musculoskeletal | Neurologic | Pain | Pulmonary | Renal/Genitourinary |
---|
Treatment (Nonmyeloablative HCT, TBI) | 13 | 9 | 4 | 17 | 55 | 26 | 25 | 24 | 3 | 5 | 6 | 10 | 10 |
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Mean Stimulated C-peptide Area Under the Curve
The primary outcome is the area under the stimulated C-peptide curve (AUC) based on data collected at time 0 to 2 hours of a 4-hour mixed meal glucose tolerance test (MMTT) conducted at the primary endpoint visit. The timed measurements are done at: 0, 15, 30 60, 90, and 120 minutes. (NCT00100178)
Timeframe: 2 years
Intervention | pmol/ml (Geometric Mean) |
---|
MMF and DZB | 0.28 |
Placebo Control | 0.27 |
MMF Alone | 0.25 |
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Acute Graft-Versus-Host Disease (GVHD)
(NCT00709592)
Timeframe: 2 year rate (%)
Intervention | percentage of participant (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 27.2 |
B:Thymoglobulin: 2.5 mg/kg/Day | 4.5 |
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Chronic Graft-Versus-Host Disease (GVHD)
(NCT00709592)
Timeframe: 2 year GVHD rate
Intervention | percentage of participants (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 23.8 |
B:Thymoglobulin: 2.5 mg/kg/Day | 31.8 |
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Donor Lymphocyte Infusion
(NCT00709592)
Timeframe: 2 year rate of DLI
Intervention | percentage of participants (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 8.9 |
B:Thymoglobulin: 2.5 mg/kg/Day | 45.5 |
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Engraftment of Donor Hematopoietic Stem Cells, as Measured by Time in Days to Neutrophil and Platelet Count Recovery Following Allogeneic PBSCT.
(NCT00709592)
Timeframe: Up to 52 weeks post transplant.
Intervention | Days (Median) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 12 |
B:Thymoglobulin: 2.5 mg/kg/Day | 12 |
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Event-free Survival
(NCT00709592)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 62.2 |
B:Thymoglobulin: 2.5 mg/kg/Day | 44.5 |
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Relapse
Patients with different disease relapses was determined according to current clinical standards based on the disease. For example, AML or MDS relapse is determined by a bone marrow biopsy. Multiple myeloma relapse requires a number of labs and/or biopsy to diagnose such as SPEP, UPEP, immunofixation, serum and urine light chains. In lymphoma disease is followed using CT and/or PET scans. (NCT00709592)
Timeframe: 2 year relapse rate (%)
Intervention | Percent patients relapsing (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 28 |
B:Thymoglobulin: 2.5 mg/kg/Day | 50 |
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Survival
(NCT00709592)
Timeframe: 2-year survival rate (%)
Intervention | percentage of patient surviving (Number) |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 71.3 |
B:Thymoglobulin: 2.5 mg/kg/Day | 62.4 |
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The Comparison of Functional Immune Reconstitution at 6-9 Months Following Transplant as Measured by Antibody Response to Vaccination With Inactivated Hepatitis A or B Vaccine.
A positive test result will indicate immune reconstitution, while a negative test results will indicate lack of immune reconstitution. Participants not done (ND) will be counted with the negative (Neg). (NCT00709592)
Timeframe: Up to 9 months following transplant
Intervention | participants (Number) |
---|
| Positive | Negative/Not Done |
---|
A:Thymoglobulin: 1.7 mg/kg/Day | 8 | 11 |
,B:Thymoglobulin: 2.5 mg/kg/Day | 3 | 19 |
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Maximum Plasma Concentration (Cmax) of GSK1070806
Serial blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. Log-transformed geometric mean and 95% confidence interval have been presented. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion
Intervention | Log (nanograms per milliliter) (Geometric Mean) |
---|
GSK1070806 3 mg/kg IV | 36315.1 |
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Number of Participants Having Infections
Number of participants having infections were summarized. (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
GSK1070806 3 mg/kg IV | 5 |
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Number of Participants Requiring Dialysis During the First 7 Days Post Transplant
The requirement of dialysis (except as needed for hyperkalaemia during the first 24 hours [hrs]) were used to assess the frequency of delayed graft function (DGF) in donation after circulatory death (DCD) renal transplant recipients treated with GSK1070806. The 'Analysis Population' (AP) is defined as participants in the 'All Subjects' Population who have been declared to have DGF or have reached 7 days. (NCT02723786)
Timeframe: Up to Day 7
Intervention | Participants (Count of Participants) |
---|
GSK1070806 3 mg/kg IV | 4 |
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Number of Participants Who Are Dialysis Independent at Visits up to 12 Months Post-transplant
Number of participants who are dialysis independent at visits up to 12 months post transplant was evaluated to assess the effect of GSK1070806 on dialysis dependency and graft survival. (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
GSK1070806 3 mg/kg IV | 2 |
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Number of Participants With Dialysis Events in the First 30 Days Post-transplant
Number of participants with dialysis events in the first 30 days post transplant was evaluated to assess the effect of GSK1070806 on dialysis dependency and graft survival. The AP Population is defined as participants having Baseline and at least one post-Baseline assessment. (NCT02723786)
Timeframe: Up to 30 days
Intervention | Participants (Count of Participants) |
---|
GSK1070806 3 mg/kg IV | 5 |
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Number of Participants With Episodes of Biopsy-proven Acute Rejection
Number of participants with episodes of biopsy-proven acute rejection were evaluated to assess the effect of GSK1070806 on acute rejection risk, and rejection/Pharmacodynamic (PD) biomarkers. (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
GSK1070806 3 mg/kg IV | 1 |
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Area Under the Plasma Concentration Time Curve (AUC) From Time 0 to the Last Measurable Concentration (AUC[0-t]) and AUC From Time 0 to Infinite Time (AUC[0-inf]) of GSK1070806
Blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. Log-transformed geometric mean and 95% confidence interval have been presented. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion
Intervention | Log (Hour*nanograms per milliliter) (Geometric Mean) |
---|
| AUC (0-t), n=7 | AUC (0-inf), n=6 |
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GSK1070806 3 mg/kg IV | 26131338.2 | 41032450.7 |
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Baseline and Change From Baseline in Serum Levels of Free, Total, and GSK1070806 Bound Interleukin 18 (IL-18) Over Time Post-transplant
IL-18 is itself rapidly secreted from intracellular stores following inflammasome mediated-activation. The appearance of IL-18 marks the initiation of the inflammatory response leading to further injury. Blood samples were collected at indicated time points to assess serum levels of free, total, and GSK1070806 bound IL-18. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. (NCT02723786)
Timeframe: Baseline and at 0.75 hours, 4-8 hours, Day 1, Day 2, Day 30, Day 90, 6 months and 12 months post reperfusion
Intervention | Picograms per milliliter (Mean) |
---|
| Serum Free IL-18, Baseline (pre-operative), n=5 | Serum Free IL-18, Day 0, 0.75 hour, n=5 | Serum Free IL-18, Day 0, 4-8 hour, n=5 | Serum Free IL-18, Day 1, n=5 | Serum Free IL-18, Day 2, n=4 | Serum Free IL-18, Day 30, n=4 | Serum Free IL-18, Day 90, n=2 | Serum Bound IL-18, Baseline (pre-operative), n=5 | Serum Bound IL-18, Day 0, 0.75 hour, n=5 | Serum Bound IL-18, Day 0, 4-8 hour, n=5 | Serum Bound IL-18, Day 1, n=5 | Serum Bound IL-18, Day 2, n=4 | Serum Bound IL-18, Day 30, n=4 | Serum Bound IL-18, Day 90, n=2 | Serum Total IL-18, Baseline (pre-operative), n=7 | Serum Total IL-18, Day 0, 0.75 hour, n=6 | Serum Total IL-18, Day 0, 4-8 hour, n=6 | Serum Total IL-18, Day 1, n=6 | Serum Total IL-18, Day 2, n=5 | Serum Total IL-18, Day 30, n=6 | Serum Total IL-18, Day 90, n=7 | Serum Total IL-18, 6 months, n=7 | Serum Total IL-18, 12 months, n=6 |
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GSK1070806 3 mg/kg IV | 26.840 | -22.620 | -22.540 | -23.890 | -5.263 | -27.925 | -2.250 | 21.156 | 362.084 | 314.864 | 472.204 | 485.740 | 617.543 | 946.020 | 130.6857 | 572.3333 | 576.3667 | 636.5667 | 660.6600 | 1175.5000 | 1423.5429 | 1303.7143 | 1091.0833 |
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Number of Participants Having Any Abnormal Clinical Chemistry Results of Potential Clinical Importance
Blood samples were collected to evaluate clinical chemistry parameters. Number of participants with abnormal chemistry results of potential clinical importance (high or low) in any of these parameters at any time post Baseline visit have been presented. PCI (high or low) was considered if albumin (low<30), calcium (low<2, high>2.75), creatinine (high: CHB>44.2 increase), glucose (low<3, high>9), magnesium (low<0.5, high>1.23), phosphorus (low<0.8, high>1.6), potassium (low<3, high>5.5), sodium (low: 130, high>150), Total carbon dioxide (CO2) (low:18, high>32), Alanine aminotransferase (ALT) (high>=2*upper limit of normal [ULN]), Aspartate aminotransferase (AST) (high: >=2*ULN), Alkaline phosphatase (ALP) (high:>=2*ULN), Total bilirubin (high: >2*ULN), Total bilirubin+ALT (high: 1.5*ULN total bilirubin with >=2*ULN ALT). (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
| Albumin, Low | Calcium, Low | Glucose, High | Potassium, Low | Potassium, High | Total Bilirubin, High | Sodium, Low | ALT, High | ALP, High | AST, High |
---|
GSK1070806 3 mg/kg IV | 6 | 7 | 5 | 1 | 3 | 1 | 2 | 1 | 1 | 1 |
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Number of Participants Having Any Abnormality in Hematology Results of Potential Clinical Importance
Blood samples were collected to evaluate hematology parameters. Number of participants with abnormality in any hematology parameter results of potential clinical importance (high or low) observed at any time post Baseline are presented. PCI (high or low) was considered if hematocrit (high:>0.54;low:change from baseline [CFB] 0.075 decrease), hemoglobin (high:180; low: CFB 25 decrease), lymphocytes (low: 0.8), neutrophil count (low: 1.5), platelet count (low: 100; high: 550), White blood cells (low: 3; high:20). (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
| Lymphocytes, Low | Hematocrit, High | White Blood Cells, High | White Blood Cells, Low | Platelet Count, Low | Total neutrophils, Low |
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GSK1070806 3 mg/kg IV | 7 | 1 | 1 | 1 | 1 | 1 |
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Number of Participants Having Any Abnormality of Potential Clinical Importance of Vital Signs Results
Vital signs parameters included analysis of systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR) and body temperature. Number of participants with any abnormality of potential clinical importance (high or low) in any of these vitals signs at any time post Baseline visit have been presented. PCI (high or low) was considered if SBP (low: <85, high:>160), DBP (low: <45, high>100), HR (low: <40, high: >110) and temperature (low: <35.5, high: >37.5). (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
| SBP, High | SBP, Low | DBP, High | DBP, Low | HR, High | Temperature, High | Temperature, Low |
---|
GSK1070806 3 mg/kg IV | 5 | 1 | 2 | 1 | 1 | 1 | 2 |
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Number of Participants With Adverse Event (AE) and Serious Adverse Event (SAE)
AE is any untoward medical occurrence in a participant, temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Any untoward event resulting in death, life threatening, requires hospitalization or prolongation of existing hospitalization, results in disability/incapacity, congenital anomaly/birth defect or any other situation according to medical or scientific judgment were categorized as SAE. (NCT02723786)
Timeframe: Up to 12 months
Intervention | Participants (Count of Participants) |
---|
| Any AE | Any SAE |
---|
GSK1070806 3 mg/kg IV | 7 | 6 |
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Serum Concentrations of GSK1070806
Serial blood samples were collected to evaluate PK of GSK1070806 at Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion. PK Population included participants in the 'All Subjects' Population for whom a serum PK sample is obtained and analyzed for GSK1070806. (NCT02723786)
Timeframe: Pre-operative, 0.75 hours, 4-8 hours, 24 hours, 168 hours, Day 30, Day 90, 6 months and 12 months after kidney reperfusion
Intervention | Nanograms per milliliter (Mean) |
---|
| Pre-operative, n=7 | 0.75 hours, n=6 | 4-8 hours, n=6 | 24 hours, n=6 | 168 hours, n=5 | Day 30, n=6 | Day 90, n=7 | 6 months, n=7 | 12 months, n=6 |
---|
GSK1070806 3 mg/kg IV | 0.0 | 58783.3 | 60033.3 | 50933.3 | 28260.0 | 17366.7 | 5047.0 | 1083.4 | 19.2 |
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Serum Creatinine at Baseline and Change From Baseline Over Time Post Transplant
Blood samples were collected to measure serum creatinine at the indicated timepoints to assess graft function in DCD renal transplant recipients treated with GSK1070806. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. NA indicates data is not available as standard deviation could not be calculated due to n=1. The AP Population is defined as participants having Baseline and at least one post-Baseline assessment. (NCT02723786)
Timeframe: Baseline and up to 12 months
Intervention | Micromoles per liter (Mean) |
---|
| Screening, n=7 | Day 0, n=7 | Day 1, n=7 | Day 2, n=7 | Day 3, n=7 | Day 4, n=7 | Day 5, n=6 | Day 6, n=6 | Day 7, n=6 | Day 8, n=5 | Day 9, n=3 | Day 10, n=3 | Day 11, n=3 | Day 12, n=2 | Day 13, n=2 | Day 14, n=2 | Day 15, n=2 | Day 16, n=2 | Day 17, n=2 | Day 18, n=2 | Day 19, n=2 | Day 20, n=2 | Day 21, n=1 | Day 22, n=1 | Day 23, n=1 | Day 24, n=1 | Day 25, n=1 | Day 26, n=1 | Day 27, n=1 | Day 28, n=1 | Day 30, n=7 | Day 90, n=7 | 6 months, n=7 | 12 months, n=6 |
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GSK1070806 3 mg/kg IV | 679.0 | -39.3 | -44.7 | -99.0 | -36.7 | -104.6 | -57.2 | -75.7 | -43.5 | -150.8 | -24.0 | -53.0 | -97.0 | 110.0 | 38.0 | 4.0 | -51.0 | -61.5 | -107.5 | -145.0 | -173.5 | -183.5 | -117.0 | -91.0 | -95.0 | -115.0 | -152.0 | -155.0 | -175.0 | -191.0 | -478.3 | -489.9 | -467.4 | -490.5 |
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Serum Interferon Gamma-induced Protein 10 (IP-10) and Serum Monokine Induced Gamma Interferon (Mig) Levels at Baseline and Change From Baseline Over Time Post Transplant
The interferon-gamma -inducible chemokine IP10 and the interferon-gamma -inducible chemokine Mig have been identified as an early predictive marker of antibody-mediated kidney graft rejection. Baseline value was the latest pre-dose assessment value. Change from Baseline was calculated as post Baseline value minus Baseline value. (NCT02723786)
Timeframe: Baseline and at 0.75 hours, 4-8 hours, Day 1, Day 2, Day 30, Day 90, 6 months and 12 months post reperfusion
Intervention | Picograms per milliliter (Mean) |
---|
| IP-10, Baseline, n=7 | IP-10, Day 0, 0.75 hour, n=6 | IP-10, Day 0, 4-8 hour, n=6 | IP-10, Day 1, n=6 | IP-10, Day 2, n=5 | IP-10, Day 30, n=6 | IP-10, Day 90, n=7 | IP-10, 6 months, n=7 | IP-10, 12 months, n=5 | Mig, Baseline, n=7 | Mig, Day 0, 0.75 hour, n=6 | Mig, Day 0, 4-8 hour, n=6 | Mig, Day 1, n=6 | Mig, Day 2, n=5 | Mig, Day 30, n=6 | Mig, Day 90, n=7 | Mig, 6 months, n=7 | Mig, 12 months, n=5 |
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GSK1070806 3 mg/kg IV | 518.83817 | -48.36607 | -262.30099 | -214.27224 | -91.07498 | -215.96831 | 221.97286 | 145.05039 | 241.29317 | 175.76865 | -14.02145 | -49.28436 | -67.61716 | -133.99600 | -159.17646 | -78.69081 | -43.68148 | -30.52711 |
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Urine Volume at Baseline and Change From Baseline Over Time Post Transplant
Urine volume at Baseline and over time post transplant was measured to assess graft function in DCD renal transplant recipients treated with GSK1070806. Baseline value was the latest pre-dose assessment value. Change from Baseline was post Baseline value minus Baseline value. All Subjects Population comprised of participants who received the dose of study medication. (NCT02723786)
Timeframe: Baseline (Pre-operative) and up to Day 28
Intervention | Liter (Mean) |
---|
| Baseline (Pre-operative), n=5 | Day 0, n=4 | Day 1, n=5 | Day 2, n=5 | Day 3, n=5 | Day 4, n=5 | Day 5, n=4 | Day 6, n=4 | Day 7, n=4 | Day 8, n=3 | Day 9, n=2 | Day 10, n=2 | Day 11, n=2 | Day 12, n=1 | Day 13, n=1 | Day 14, n=1 | Day 15, n=1 | Day 16, n=1 | Day 17, n=1 | Day 18, n=1 | Day 19, n=1 | Day 20, n=1 | Day 21, n=1 | Day 22, n=1 | Day 23, n=1 | Day 24, n=1 | Day 25, n=1 | Day 26, n=1 | Day 27, n=1 | Day 28, n=1 |
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GSK1070806 3 mg/kg IV | 0.6700 | -0.5150 | 0.5862 | 1.2820 | 0.8840 | 1.1480 | 0.8270 | 1.1168 | 1.2985 | 1.2090 | 1.2135 | 0.8585 | 0.4985 | 1.4000 | 1.6900 | 1.1800 | 1.5500 | 1.8500 | 1.5000 | 1.9000 | 1.6000 | 1.2500 | 0.7500 | 1.0500 | 1.1500 | 1.4500 | 2.0500 | 2.0000 | 1.5500 | 1.4600 |
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Acute Rejection Per Kidney Biopsy (Banff Grading Criteria)
(NCT00556933)
Timeframe: Two years
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus | 7 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 4 |
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 8 |
Divided-dose rATG(1.5mg/kgx4), Sirolimus/Mycophenolate Mofetil | 9 |
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Acute Tubular Necrosis (ATN) Rate, Defined as the Requirement for Dialysis Within 7 Days Post-transplantation.
(NCT00556933)
Timeframe: Seven days
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus | 3 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 1 |
Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 6 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 2 |
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Graft Survival
Graft failure = permanent return of patient to dialysis. (NCT00556933)
Timeframe: Two years
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 0 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 1 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 2 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 0 |
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Lymphoid Cell Sub-type CD3 Absolute Numbers
(NCT00556933)
Timeframe: One year
Intervention | CD3 Cell Numbers/mm^3 (Mean) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 446 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 375 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 392 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 266 |
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New-onset Diabetes and Hyperglycemia After Transplantation (NODAT)
(NCT00556933)
Timeframe: Six months
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 10 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 7 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 5 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 12 |
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New-onset Polyomavirus (BK Virus) Disease Per Kidney Biopsy
(NCT00556933)
Timeframe: Two years
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 1 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 2 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 0 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 2 |
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Patient Survival
(NCT00556933)
Timeframe: Two years
Intervention | participants (Number) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 0 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 1 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 0 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 1 |
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Ratio of CD4/CD8 Lymphoid Cells
(NCT00556933)
Timeframe: One year
Intervention | Ratio of cell counts (Mean) |
---|
Single Dose rATG (6 mg/kg) and Tacrolimus/Sirolimus | 0.84 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 0.84 |
Single-dose rATG (6mg/kg) and Sirolimus/Mycophenolate Mofetil | 0.98 |
Divided-dose rATG(1.5mg/kgx4); Sirolimus/Mycophenolate Mofetil | 1.01 |
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Safety Profile
Number of events: cytomegalovirus (CMV) disease, opportunistic infections (bacteremia, abscess, pneumonia, fungal), Post-transplantation Lymphoproliferative Disorder (PTLD), wound healing problems within 30 days, and lymphoceles. (NCT00556933)
Timeframe: Two years
Intervention | Events (Number) |
---|
Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus | 11 |
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 14 |
Ingle-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 10 |
Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 17 |
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Average of Renal Function
Calculated Glomerular Filtration Rate (GFR) by using the abbreviated MDRD (aMDRD) formula and patient serum creatinine and demographic data; averaged values from months four through 24. (NCT00556933)
Timeframe: Two years
Intervention | ml/min/1.73m2 (Mean) |
---|
| Average GFR, months 1-3 | Average GFR, months 4-6 | Average GFR, months 7-9 | Average GFR, months 10-12 | Average GFR, months 13-15 | Average GFR, months 16-18 | Average GFR, months 19-21 | Average GFR, months 22-24 |
---|
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 50.1 | 57.3 | 55.0 | 55.6 | 57.3 | 56.3 | 54.8 | 57.0 |
,Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 50.4 | 54.8 | 55.8 | 56.6 | 58.5 | 56.6 | 56.8 | 57.6 |
,Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus | 50.4 | 57.3 | 54.8 | 55.8 | 56.8 | 57.6 | 56.6 | 56.9 |
,Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 53.6 | 60.2 | 61.0 | 57.4 | 61.8 | 62.3 | 60.2 | 62.9 |
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Chronic Allograft Nephropathy (Cumulative Calcineurin-inhibitor Nephrotoxicity/Transplant Nephropathy) Per Protocol Surveillance Kidney Biopsies (Banff Grading Criteria).
Protocol kidney biopsies collected at approximately 12 and 24 months were scored by a transplant renal pathologist blinded to treatment group assignment for evidence of rejection, BK virus nephropathy, antibody-mediated rejection, recurrent disease, inflammation, and Banff 2005 categories of chronic renal injury. Chronic injury categories were arteriolar hyaline thickening (ah), allograft glomerulopathy (cg), interstitial fibrosis (ci), tubular atrophy (ct), and vascular fibrous intimal thickening (cv). Severity scores within each category could be 0 (<5%; none or minimal), 1 (>5% - <25%; mild), 2 (>25% - <50%, moderate), or 3 (>50%, severe). The proportions of patients in each severity grade (0, 1, 2, and 3) for both the individual categories and a composite were compared using Fisher's exact test. (NCT00556933)
Timeframe: Two years
Intervention | percentage of participants (Number) |
---|
| Banff histopathology cumulative grade = 0 | Banff histopathology cumulative grade = 1 | Banff histopathology cumulative grade = 2 | Banff histopathology cumulative grade = 3 |
---|
Divided-dose rATG (1.5mg/kg x 4) and Tacrolimus/Sirolimus | 40 | 46 | 9 | 6 |
,Divided-dose rATG(6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 51 | 41 | 7 | 1 |
,Single Dose rATG (6 mg/kg x 1) and Tacrolimus/Sirolimus | 43 | 37 | 16 | 4 |
,Single-dose rATG (6mg/kg ) and Sirolimus/Mycophenolate Mofetil | 53 | 45 | 2 | 0 |
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Immunosuppression-free Duration in Days, Defined as Time From Completion of Immunosuppression Withdrawal to End of Trial Participation or to Time of Restarting Immunosuppression
Time (in days) from when the participant is off all immunosuppression to the end of trial participation or re-initiation of immunosuppression, whichever is earliest. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Days (Median) |
---|
Transplanted | 374 |
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Percent of Participants Requiring Anti-lymphocyte Therapy (OKT3, ATG) for an Acute Rejection Event
Anti-lymphocyte therapy is a drug that targets specific cells in the immune system called lymphocytes (white blood cells). This therapy helps stop the participant's immune system from attacking the donor kidney. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 20 |
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Percent of Participants Successfully Withdrawn From Immunosuppression and Remained Off Immunosuppression for at Least 52 Weeks
Participants are considered successfully withdrawn from immunosuppression if they remained off immunosuppression for at least 52 weeks without evidence of rejection, as determined by a biopsy performed 52 weeks after completion of immunosuppression withdrawal. All participants who failed to complete immunosuppression withdrawal, regardless of reason, or failed to have a biopsy 52 weeks after completion of immunosuppression withdrawal, were considered to have failed. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks after discontinuation of all immunosuppression
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 20 |
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Percent of Participants With Chronic T Cell-mediated or Antibody-mediated Rejection
This assessment included participants who experienced chronic T cell-mediated rejection or chronic antibody mediated rejection as well as progressive interstitial fibrosis/tubular atrophy (IF/TA), transplant glomerulopathy or chronic obliterative arteriopathy without an alternative, non-rejection-related cause. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 0 |
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Percent of Transplant Participants Who Died
Death after receiving a kidney transplant. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 0 |
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Percent of Transplanted Participants Who Achieve Either Sirolimus Monotherapy or Monotherapy on a Mycophenolic Compound Within 52 Weeks Post-transplant
Participants that were treated with only sirolimus or treated with only mycophenolate mofetil (MMF) or mycophenolic acid within 52 weeks after transplantation. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 70 |
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Percent of Transplanted Participants Who Achieve MMF or Mycophenolic Acid Monotherapy Within 52 Weeks Post-transplant in Those Participants Intolerant of Sirolimus
Participants that were treated with only mycophenolate mofetil (MMF) or mycophenolic acid within 52 weeks after transplantation in those who could not tolerate sirolimus. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 33 |
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Percent of Transplanted Participants Who Achieve Sirolimus Monotherapy Within 52 Weeks Post-transplant
Participants that were treated with only sirolimus within 52 weeks after transplantation in those who could tolerant sirolimus. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks post-transplantation
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 86 |
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Percent of Transplanted Participants Who Remain Off Immunosuppression for at Least 52 Weeks Including Those in Whom the 52 Week Biopsy Was Not Performed
Participants are considered successfully withdrawn from immunosuppression if they remained off immunosuppression for at least 52 weeks without evidence of rejection. A biopsy performed 52 weeks after completion of immunosuppression withdrawal confirmed that there was no sub-clinical evidence of rejection. This result considers a participant off all immunosuppression for at least 52 weeks with or without the confirmatory week 52 biopsy as a success. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through 52 weeks after discontinuation of all immunosuppression
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 20 |
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Percent of Transplanted Participants Who Remain Off Immunosuppression for the Duration of the Study as Defined as Completion of All Schedules of Events/Followed Through August 25, 2017
Participants that remained off all immunosuppression through the completion of study participation. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through study completion (up to 4.4 years post-transplant)
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 10 |
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Percent of Transplanted Participants With Acute Rejection or Presumed Acute Rejection
Participants with either biopsy proven acute rejection per Banff guidelines or participants that were treated for acute rejection in the absence of a biopsy. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Percent of participants (Geometric Least Squares Mean) |
---|
Induction (Rituximab and ATG) | 90 |
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Percent of Transplanted Participants With Graft Loss
A participant is considered to have graft loss when the donated kidney needs to be removed, the participant is retransplanted with another donor kidney, or chronic dialysis is instituted. The endpoint is summarized with a two-sided, 95% exact binomial confidence interval. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Percent of participants (Number) |
---|
Induction (Rituximab and ATG) | 0 |
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Time From Completion of Immunosuppression Withdrawal to First Episode of Acute Rejection or Presumed Acute Rejection
Time (in days) from when the participant is off all immunosuppression to the first episode of biopsy proven or presumed acute rejection. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Days (Median) |
---|
Induction (Rituximab and ATG) | 380 |
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Time From Transplant to the First Episode of Acute Rejection Requiring Treatment
Time (in days) from transplant to the start date of the first dose of treatment for acute rejection. This includes acute rejection episodes requiring treatment that are not biopsy proven. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Days (Median) |
---|
Induction (Rituximab and ATG) | 1008.5 |
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Histological Severity of Biopsies Demonstrating Acute Rejection as Measured by Banff 2007 Grade
"Biopsy-confirmed 1.) acute cellular rejection and 2.) acute antibody-mediated rejection was classified according to Banff 2007 criteria of renal allograft pathology for renal allograft rejection. A Banff result of indeterminate was not classified as rejection.~Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection is defined as a grade ≥ IA. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe.~Acute antibody-mediated rejection-or humoral rejection-is defined as a grade ≥1. Severity is graded as I, II, or III, with I being the mildest form of antibody-mediated rejection and III being the most severe." (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Biopsies (Number) |
---|
| Acute Cellular Rejection (Type IA) | Acute Cellular Rejection (Type IB) | Acute Cellular Rejection (Type IIA) | Acute Cellular Rejection (Type IIB) | Acute Cellular Rejection (Type III) | Acute Antibody-Mediated Rejection (Type I) | Acute Antibody-Mediated Rejection (Type II) | Acute Antibody-Mediated Rejection (Type III) |
---|
Induction (Rituximab and ATG) | 7 | 3 | 0 | 0 | 0 | 0 | 0 | 0 |
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Number of Adverse Events, Including Number of Post-transplant Infections, Wound Complications, Lymphocoele, Post-transplant Diabetes Mellitus, and Malignancies
Adverse events that are reported as being a post-transplant infection, wound complication, lymphocoele (a collection of fluid in the lymphatic system), post-transplant diabetes mellitus or malignancy. (NCT01318915)
Timeframe: Transplantation through end of trial participation (up to 4.4 years post-transplant)
Intervention | Events (Number) |
---|
| Post-Transplant Infection | Wound Complications | Lymphocoele | Post-Transplant Diabetes Mellitus | Malignancy |
---|
Induction (Rituximab and ATG) | 4 | 1 | 0 | 0 | 0 |
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Participant Diastolic Blood Pressure Over Time
Diastolic blood pressure measures the pressure in the arteries when the heart is at rest and is thus filled with blood. A normal diastolic blood pressure is lower than 80 mmHg. High blood pressure, as known as hypertension, is a risk factor for coronary artery disease, stroke, heart failure, and other complications if left unmanaged. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant
Intervention | mmHg (Median) |
---|
| 26 Weeks Post-Transplant | 52 Weeks Post-Transplant | 104 Weeks Post-Transplant | 156 Weeks Post-Transplant | 208 Weeks Post-Transplant |
---|
Induction (Rituximab and ATG) | 77.5 | 77 | 79 | 76 | 73 |
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Participant Glucose Level Over Time
This is a measure of glucose found in the blood. Glucose, a sugar, is an energy source that the body relies on to properly function. If levels are too high for a long period of time, diabetes can develop. Diabetes can result in many long-term complications such as eye, kidney, and nerve damage, stroke, and cardiovascular complications. Fasting levels for glucose should be around 70-99 mg/dL and less than 140 mg/dL within 2 hours after a meal. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant
Intervention | mg/dL (Median) |
---|
| 26 Weeks Post-Transplant | 52 Weeks Post-Transplant | 104 Weeks Post-Transplant | 156 Weeks Post-Transplant | 208 Weeks Post-Transplant |
---|
Induction (Rituximab and ATG) | 103.5 | 108 | 96.5 | 104.5 | 83.5 |
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Participant Renal Function as Measured by GFR Using CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. A value less than 15 indicates kidney failure, 15 to 29 indicates severe loss of kidney function, 30 to 44 indicates moderate to severe loss of kidney function, 45 to 59 mild to moderate loss of kidney function, 60 to 89 indicates mild loss of kidney function, and 90 or higher indicates normal kidney function. The equation developed by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) is used to estimate GFR from serum creatinine. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant
Intervention | mL/min/1.73m^2 (Median) |
---|
| 26 Weeks Post-Transplant | 52 Weeks Post-Transplant | 104 Weeks Post-Transplant | 156 Weeks Post-Transplant | 208 Weeks Post-Transplant |
---|
Induction (Rituximab and ATG) | 60.3 | 70.9 | 63.7 | 56.7 | 56.0 |
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Participant Systolic Blood Pressure Over Time
Systolic blood pressure measures the pressure on the blood vessels when the heart is beats and thus is pushing blood to the rest of the body. A normal systolic blood pressure is lower than 120 mmHg. High blood pressure, as known as hypertension, is a risk factor for coronary artery disease, stroke, heart failure, and other complications if left unmanaged. The value closest to and within 6 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant
Intervention | mmHg (Median) |
---|
| 26 Weeks Post-Transplant | 52 Weeks Post-Transplant | 104 Weeks Post-Transplant | 156 Weeks Post-Transplant | 208 Weeks Post-Transplant |
---|
Induction (Rituximab and ATG) | 131.5 | 135 | 137 | 140 | 132 |
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Participant Total Cholesterol Over Time
Total cholesterol measures the amount of cholesterol found in the blood. Cholesterol is a waxy substance your body needs to build cells, but too much can be a problem since it can build-up in arteries. Narrowed arteries can result in heart attack or stroke. A value less than 200 mg/dL is considered good. The value closest to and within 12 weeks of the day expected was selected. (NCT01318915)
Timeframe: 26, 52, 104, 156, and 208 Weeks Post-Transplant
Intervention | mg/dL (Median) |
---|
| 26 Weeks Post-Transplant | 52 Weeks Post-Transplant | 104 Weeks Post-Transplant | 156 Weeks Post-Transplant | 208 Weeks Post-Transplant |
---|
Induction (Rituximab and ATG) | 188 | 141.5 | 165 | 175 | 173 |
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Phase I: Acute Rejection-Free Survival
Percent of participants at 6 months without acute rejection. (NCT01062555)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Phase I Arm 1 | 80 |
Phase I Arm 2 | 85 |
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Phase I: The Minimization of Negative Side Effects - Graft Survival
Percent of participants at 6 months with a functioning graft (without graft failure). (NCT01062555)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Phase I Arm 1 | 99 |
Phase I Arm 2 | 99 |
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Phase I: The Minimization of Negative Side Effects - Patient Survival
The percentage of patients alive at 6 month post transplant. (NCT01062555)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Phase I Arm 1 | 99 |
Phase I Arm 2 | 98 |
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Phase II: Acute Rejection-Free Survival
The percentage of patients without acute rejection at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years
Intervention | percentage of participants (Number) |
---|
Phase II Arm 1 | 76 |
Phase II Arm 2 | 81 |
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Phase II: The Minimization of Negative Side Effects - Graft Survival
The percentage of patients with a functioning graft (without graft failure) at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years
Intervention | percentage of participants (Number) |
---|
Phase II Arm 1 | 80 |
Phase II Arm 2 | 78 |
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Phase II: The Minimization of Negative Side Effects - Patient Survival
The percentage of patients alive at 7 years post transplant or at the end of study activities. Not all participants completed 7-year follow-up. (NCT01062555)
Timeframe: up to 7 years
Intervention | percentage of participants (Number) |
---|
Phase II Arm 1 | 89 |
Phase II Arm 2 | 85 |
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Percent Change From Baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR) at Week 24 in the Blinded Treatment Period (Part B)
The percent change from baseline in UPCR based on 24-hour urine collections. 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 24. (NCT03943147)
Timeframe: Week 24
Intervention | Percent change from baseline (Number) |
---|
Open Label MMF + BMS-986165 | -34.86 |
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The Number of Participants Experiencing Averse Events in the Blinded Treatment Period (Part B)
An adverse event (AE) is defined as any new untoward medical occurrence or worsening of a preexisting medical condition in a clinical investigation participant administered study drug and that does not necessarily have a causal relationship with this treatment. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 1 |
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The Number of Participants With Abnormal Laboratory Parameters of Clinical Significance in the Blinded Treatment Period (Part B)
The number of participants with abnormal laboratory parameters (Chemistry, hematology, coagulation, immunohematology) that have been considered clinically significant. Clinically relevant laboratory results are determined by the investigator. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Clinically Significant ECG Abnormalities in the Blinded Treatment Period (Part B)
The number of participants with clinically significant abnormalities in electrocardiograms (ECGs) parameters. The following ECG parameters will be measured: HR, PR-interval, QRS-duration, QT-interval, QTc-interval. A single 12-lead ECG will be recorded after the participant has been supine for at least 5 minutes. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Complete Renal Response (CRR) at Week 24 in the Blinded Treatment Period (Part B)
The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline. (NCT03943147)
Timeframe: Week 24
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Complete Renal Response (CRR) at Week 52 in the Blinded Treatment Period (Part B)
The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline. (NCT03943147)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 24 in the Blinded Treatment Period (Part B)
The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline who was also able to successfully taper corticosteroid use to ≤ 7.5 mg/day. (NCT03943147)
Timeframe: Week 24
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Complete Renal Response (CRR) Plus Successful Corticosteroid Taper to ≤ 7.5 mg/Day at Week 52 in the Blinded Treatment Period (Part B)
The number of participants with complete renal response (CRR) defined as a 24-hour Urine Protein:Creatinine Ratio (UPCR) ≤ 0.5 mg/mg and an estimated glomerular filtration rate (eGFR) (using the MDRD equation) ≥ 60 mL/min or ≤ 20% decrease from baseline who was also able to successfully taper corticosteroid use to ≤ 7.5 mg/day. (NCT03943147)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Partial Renal Response (PRR) at Week 24 in the Blinded Treatment Period (Part B)
The number of participants with partial renal response (PRR) defined as ≥ 50% reduction from baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR). 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 24. (NCT03943147)
Timeframe: Week 24
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Number of Participants With Partial Renal Response (PRR) at Week 52 in the Blinded Treatment Period (Part B)
The number of participants with partial renal response (PRR) defined as ≥ 50% reduction from baseline in 24-hour Urine Protein:Creatinine Ratio (UPCR). 24-hour urine specimens measure the levels of proteins and creatinine in urine and will be used for the UPCR at baseline (week 12) and week 52. (NCT03943147)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Open Label MMF + BMS-986165 | 0 |
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The Percent Change in Vital Sign Measurements in the Blinded Treatment Period (Part B)
The percent change from baseline in Vital sign measurements including: blood pressure, heart rate, respiratory rate, and temperature. Blood pressure and heart rate are measured after the participant has been resting quietly for at least 5 minutes. Data collected from the week 12 visit in Part A will be used for baseline values in Part B. (NCT03943147)
Timeframe: From baseline up to 52 weeks after first dose in Part B
Intervention | Percent change from baseline (Number) |
---|
| Diastolic Blood Pressure(mmHg) | Systolic Blood Pressure(mmHg | Heart Rate(beats/min) | Respiratory Rate(breaths/min) | Temperature(C) |
---|
Open Label MMF + BMS-986165 | -3.45 | 5.22 | 16.87 | 6.25 | -1.35 |
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Number of Patients With a Partial Response
The proportion of each type of response with a 95% CI will be reported for all evaluable patients, assuming a binomial distribution. (NCT04205240)
Timeframe: Approximately 11 months
Intervention | patients (Number) |
---|
Treatment (Conditioning Regimen, Stem Cell Transplant) | 1 |
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Number of Patients With Grade II-IV Acute Graft-versus Host Disease (GvHD (aGVHD)
The event will be onset of grade II-IV aGvHD and time to aGvHD will be defined as the period of time from transplantation to the event of aGvHD. Death and early relapse without aGvHD will be competing risks. Cumulative incidence rate of aGVHD with 95% confidence intervals will be estimated from the cumulative incidence curves. To evaluate the association between patient characteristics and aGVHD, the Gray's test accounting for competing risks will be used to compare the cumulative incidence curves and a proportional hazards model for the sub distribution of competing risks will be used to estimate the hazard ratio. The cumulative incidence of chronic GVHD (cGVHD) will be similarly analyzed. (NCT04205240)
Timeframe: Up to 6 weeks
Intervention | participants (Number) |
---|
Treatment (Conditioning Regimen, Stem Cell Transplant) | 0 |
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AUC0-inf - Area Under Concentration-time Curve From Time Zero to Infinity (Extrapolated)
Bioequivalence based on AUC0-inf (NCT00911274)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 15.7365 |
CellCept® (Reference) | 15.7289 |
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AUC0-t - Area Under Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration
Bioequivalence based on AUC0-t (NCT00911274)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 14.8297 |
CellCept® (Reference) | 14.6761 |
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Cmax - Maximum Observed Concentration
Bioequivalence based on Cmax (NCT00911274)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 8.5858 |
CellCept® (Reference) | 9.2170 |
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Donor-specific HLA Antibodies, Re-transplantation, or Death
The Outcome Measure is a composite primary endpoint of the development of donor-specific HLA antibodies, re-transplantation, or death. Testing for donor-specifc HLA antibodies was performed at study-specified time points using the single antigen bead assay at the study core lab. Donor-specific HLA antibodies were defined as reactivity with a mean fluorescence intensity (MFI) ≥ 2,000. (NCT03388008)
Timeframe: 365 days
Intervention | participants (Number) |
---|
| Death | Donor-specific HLA antibodies | Re-transplantation |
---|
Belatacept-based Immunosuppression | 5 | 3 | 0 |
,Standard of Care | 0 | 3 | 0 |
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Percentage of Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6 Months
Number of Participants with Clinically-suspected biopsy-proven acute rejection (CSBPAR) at 6 Months (NCT02137239)
Timeframe: 6 Months
Intervention | Percentage of participants (Number) |
---|
Treatment A | 7.7 |
Treatment B | 9.4 |
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Percentage of Participants With Adverse Events (AEs)
Percentage of participants with AEs up to 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 months Post-Transplant
Intervention | Percentage of participants with AEs (Number) |
---|
Treatment A | 100.0 |
Treatment B | 97.0 |
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Percentage of Participants With Serious Adverse Events (SAEs)
Percentage of participants with SAEs up to 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 months Post-Transplant
Intervention | Percentage of participants with SAEs (Number) |
---|
Treatment A | 52.0 |
Treatment B | 60.6 |
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Time to Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR).
Time to Clinically suspected biopsy proven acute rejection (NCT02137239)
Timeframe: Up to 24 Months
Intervention | Months (Mean) |
---|
Treatment A | NA |
Treatment B | NA |
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Absolute Calculated Glomerular Filtration Rate (cGFR): Mean
Absolute (mean and median) cGFR values at 3, 6, 12 and 24 months post-transplant, as determined from the 4-variable Modification of Diet in Renal Disease (MDRD) formula (NCT02137239)
Timeframe: Up 24 Months post-transplant
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| At 3 Months | At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 69.2 | 66.0 | 66.2 | 71.8 |
,Treatment B | 62.2 | 63.9 | 62.0 | 68.7 |
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Absolute Values of Blood Pressure: Mean
Absolute (mean and median) values for SBP and DBP at 3, 6, 12 and 24 months posttransplant; (NCT02137239)
Timeframe: Up to 24 Months
Intervention | mmHg (Mean) |
---|
| Diastolic Month 3 | Systolic Month 3 | Diastolic Month 6 | Systolic Month 6 | Diastolic Month 12 | Systolic Month 12 | Diastolic Month 24 | Systolic Month 24 |
---|
Treatment A | 78.7 | 134.2 | 77.4 | 128.1 | 78.7 | 131.0 | 78.1 | 130.9 |
,Treatment B | 77.7 | 131.0 | 79.4 | 133.0 | 80.1 | 131.0 | 78.5 | 131.7 |
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Absolute Values of Fasting Lipid Values: Mean
"Absolute (mean and median) values at 3, 6, 12 and 24 months post-transplant for the following:~Serum total cholesterol (TC) Serum high density lipoprotein (HDL) cholesterol Serum low density lipoprotein (LDL) cholesterol Serum triglycerides (TG)" (NCT02137239)
Timeframe: Up to 24 Months
Intervention | mg/dL (Mean) |
---|
| TC Month 3 | TC Month 6 | TC Month 12 | TC Month 24 | HDL Month 3 | HDL Month 6 | HDL Month 12 | HDL Month 24 | LDL Month 3 | LDL Month 6 | LDL Month 12 | LDL Month 24 | TG Month 3 | TG Month 6 | TG Month 12 | TG Month 24 |
---|
Treatment A | 181.2 | 197.7 | 189.0 | 193.2 | 50.6 | 46.4 | 49.4 | 50.1 | 96.9 | 115.2 | 107.5 | 97.9 | 171.6 | 180.0 | 162.4 | 263.4 |
,Treatment B | 174.4 | 175 | 169.9 | 168.2 | 50.4 | 53.9 | 49.6 | 51.3 | 96.5 | 93.7 | 88.0 | 91.5 | 137.8 | 138.3 | 161.3 | 145.0 |
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Clinically-suspected Biopsy-proven Acute Rejection (CSBPAR) at 6, 12 and 24 Months
"Clinically-suspected biopsy-proven acute rejection (CSBPAR) at 6, 12 and 24 Months~Change in the incidence of CSBPAR at 6, 12 and 24 months post transplant, in the belatacept + EVL(Treatment A) as compared to TAC + MMF (Treatment B)." (NCT02137239)
Timeframe: Up to 24 Months
Intervention | Percentage of CSBPAR (Number) |
---|
| CSBPAR at 6 Months | CSBPAR at 12 months | CSBPAR at 24 Months |
---|
Treatment A | 7.7 | 11.5 | 15.4 |
,Treatment B | 9.4 | 12.5 | 12.5 |
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Mean and Mean Change From Baseline in Blood Glucose
Mean fasting blood glucose levels, and mean changes from baseline values at Months 6, 12 and 24 months post- transplant (NCT02137239)
Timeframe: Up to 24 months
Intervention | mg/dL (Mean) |
---|
| Mean Value at 6 months | Change from baseline at 6 months | Mean Value at 12 months | Change from baseline at 12 months | Mean Value at 24 months | Change from baseline at 24 months |
---|
Treatment A | 107.2 | 4.9 | 101.1 | -1.3 | 127.5 | 22.3 |
,Treatment B | 107.2 | 4.8 | 127.5 | 20.6 | 111.8 | 15.0 |
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Mean and Mean Change From Baseline in Whole Blood HbA1c
Mean whole blood HbA1C concentrations, and mean changes from baseline values at Months 6, 12 and 24 months post-transplant. (NCT02137239)
Timeframe: Up to 24 months
Intervention | mg/dL (Mean) |
---|
| Mean Value at 6 months | Change from baseline at 6 months | Mean Value at 12 months | Change from baseline at 12 months | Mean Value at 24 months | Change from baseline at 24 months |
---|
Treatment A | 6.11 | 0.34 | 6.18 | 0.47 | 6.24 | 0.66 |
,Treatment B | 6.13 | 0.48 | 6.21 | 0.32 | 6.29 | 0.41 |
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Mean Change From Month 3 in cGFR
The mean change from Month 3 cGFR at 3, 6, 12 and 24 months post-transplant (NCT02137239)
Timeframe: Up 24 Months post-transplant
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| At 3 Months | At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 0 | -3.2 | -3.1 | 3.1 |
,Treatment B | 0 | 2.8 | 1.4 | 6.3 |
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Mean Changes From Baseline Values for Blood Pressure
Mean changes from baseline values for SBP and DBP at 6, 12 and 24 months post-transplant (NCT02137239)
Timeframe: Up to 24 Months
Intervention | mmHg (Mean) |
---|
| Diastolic Month 6 | Systolic Month 6 | Diastolic Month 12 | Systolic Month 12 | Diastolic Month 24 | Systolic Month 24 |
---|
Treatment A | 1.0 | -4.0 | 2.3 | -1.1 | 0.9 | -2.3 |
,Treatment B | 4.8 | -0.7 | 5.4 | -3.2 | 2.1 | -4.2 |
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Mean Changes From Baseline Values of Lipid Values
"Mean changes from baseline values in the following:~Serum total cholesterol (TC) Serum high density lipoprotein (HDL) cholesterol Serum low density lipoprotein (LDL) cholesterol Serum triglycerides (TG)" (NCT02137239)
Timeframe: at months 12 and 24
Intervention | mg/dL (Mean) |
---|
| TC Month 12 | TC Month 24 | HDL Month 12 | HDL Month 24 | LDL Month 12 | LDL Month 24 | TG Month 12 | TG Month 24 |
---|
Treatment A | 25.7 | 26.6 | 5.4 | 6.2 | 25.7 | 17.4 | 3.3 | 106.8 |
,Treatment B | -2.8 | 10.0 | 1.9 | 4.8 | 10.8 | 15.7 | -86.1 | -13.6 |
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Number of Participants Deaths Post Transplant
Number of participant deaths at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: up to 24 months
Intervention | Participants (Count of Participants) |
---|
| At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 0 | 0 | 0 |
,Treatment B | 0 | 0 | 0 |
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Number of Participants Who Experience Graft Loss Post Transplant
Number of all participants who experience graft loss at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: At 6, 12 and 24 months
Intervention | Participants (Count of Participants) |
---|
| At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 1 | 1 | 1 |
,Treatment B | 1 | 1 | 1 |
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Number of Participants Who Survive With a Functioning Graft
Number of all participants who survive with a functioning graft at 6, 12 and 24 months post transplant (NCT02137239)
Timeframe: At 6, 12 and 24 months
Intervention | Participants (Count of Participants) |
---|
| At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 25 | 25 | 25 |
,Treatment B | 31 | 31 | 31 |
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Percentage of Participants With BANFF Grade by Severity Grades. BANFF Type (Grade) for Acute/Active Rejection
"Treatment differences in the severity grades to treat all episodes of CSBPAR at 6, 12, and 24 months post-transplant.~Type 1A - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/Tubular cross section or group of 10 Tubular cell). Type 1B - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/Tubular cross section or group of 10 Tubular cell).Type 2A - Cases with mild to moderate intimal arteritis.Type 2B - Cases with severe intimal arteritis comprising >25% of the luminal area. Type 3 - Cases with transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (v3 with accompanying lymphocytic inflammation)" (NCT02137239)
Timeframe: At 6, 12 and 24 Months
Intervention | Percentage of Participants (Number) |
---|
| 6 Months: Mild Acute (1A) | 6 Months: Mild Acute (1B) | 6 Months: Moderate Acute (2A) | 6 Months: Moderate Acute (2B) | 6 Months: Severe Acute | 12 Months: Mild Acute (1A) | 12 Months: Mild Acute (1B) | 12 Months: Moderate Acute (2A) | 12 Months: Moderate Acute (2B) | 12 Months: Severe Acute | 24 Months: Mild Acute (1A) | 24 Months: Mild Acute (1B) | 24 Months: Moderate Acute (2A) | 24 Months: Moderate Acute (2B) | 24 Months: Severe Acute |
---|
Treatment A | 3.8 | 0 | 7.7 | 0 | 0 | 7.7 | 0 | 7.7 | 0 | 0 | 11.5 | 0 | 7.7 | 0 | 0 |
,Treatment B | 0 | 3.1 | 6.3 | 0 | 0 | 3.1 | 3.1 | 6.3 | 0 | 0 | 3.1 | 3.1 | 6.3 | 0 | 0 |
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Percentage of Participants With De Novo Donor Specific Anti-HLA Antibodies (DSA)
Characterization of any de novo DSA detected by IgM and IgG subclasses, and by the presence or absence of complement fixing properties. (NCT02137239)
Timeframe: Up to 24 Months
Intervention | Percentage (Number) |
---|
| Baseline Class 1 DSA | Baseline Class 2 DSA | Baseline Both Class 1 and 2 DSA | De Novo 12 Month Class 1 DSA | De Novo 12 Month Class 2 DSA | De Novo 12 Month Both Class 1 and 2 DSA | De Novo 24 Month Class 1 DSA | De Novo 24 Month Class 2 DSA | De Novo 24 Month Both Class 1 and 2 DSA |
---|
Treatment A | 10 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
,Treatment B | 0 | 0 | 0 | 0 | 0 | 0 | 3.45 | 0 | 0 |
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Percentage of Participants With Donor Specific Anti-HLA Antibodies (DSA)
Percentage of participants with, and titers of pre-existing (pre-transplant) DSA on Day 1 (pre-transplant, pre-dose), and at Months 12 and 24 posttransplant (NCT02137239)
Timeframe: Up to 24 Months
Intervention | Percentage of Participants (Number) |
---|
| Baseline Class 1 DSA | Baseline Class 2 DSA | Baseline Both Class 1 and 2 DSA | 12 Month Class 1 DSA | 12 Month Class 2 DSA | 12 Month Both Class 1 and 2 DSA | 24 Month Class 1 DSA | 24 Month Class 2 DSA | 24 Month Both Class 1 and 2 DSA |
---|
Treatment A | 10 | 0 | 0 | 8.00 | 0 | 0 | 8.00 | 0 | 0 |
,Treatment B | 0 | 0 | 0 | 0 | 3.03 | 0 | 3.03 | 3.03 | 0 |
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Percentage of Participants With Events of Special Interest (ESIs)
"Percentage of participants which have one of the following events of special interest:~Serious Infections Post-Transplant Lymphoproliferative Disorder (PTLD) Progressive multifocal leukoencephalopathy (PML) Malignancies (Other than PTLD) including non-melanoma skin carcinomas (Malignancies) Tuberculosis Infections Central Nervous System (CNS) Infections Viral Infections Infusion Related reactions within 24 hours since belatacept infusion" (NCT02137239)
Timeframe: Up to 24 Months
Intervention | Percentage of participants with ESIs (Number) |
---|
| Serious Infections | PTLD | PML | Malignancies | TB | CNS Infections | Viral Infections | Infusion Related Reactions |
---|
Treatment A | 16 | 4.0 | 0 | 4.0 | 0 | 0 | 0 | 1 |
,Treatment B | 15.2 | 3.0 | 0 | 3.0 | 0 | 0 | 0 | 0 |
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Percentage of Participants With New Onset Diabetes After Transplant
Percentage of participants with New Onset Diabetes After Transplantation (NODAT) at 6, 12, and 24 months post-transplant. (NCT02137239)
Timeframe: up to 24 months
Intervention | Percentage of participants (Number) |
---|
| Up to 6 Months | Up to 12 Months | Up to 24 Months |
---|
Treatment A | 11.5 | 11.5 | 15.4 |
,Treatment B | 6.3 | 6.3 | 12.5 |
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Percentage of Particpants With Laboratory Test Abnormalities (LTAs)
Percentage of participants with laboratory tests with marked laboratory abnormalities (NCT02137239)
Timeframe: At 24 Months
Intervention | Percentage of participants (Number) |
---|
| Hemoglobin (Low) | Leukocytes (low) | Lymphocyte (Absolute) (low) | Neutrophils (Absolute) (low) | Aspartate Aminotransferase (High) | Creatinine (High) | Inorganic Phosphorus (low) | Potassium (high) | Sodium (low) | Albumin (low) | Glucose (high) | Triglycerides (high) | Uric Acid (high) |
---|
Treatment A | 12.0 | 0 | 84.0 | 0 | 4.0 | 16.0 | 24.0 | 4.0 | 4.0 | 0 | 8.0 | 12.0 | 8.0 |
,Treatment B | 6.1 | 3.0 | 69.7 | 3.0 | 0 | 3.0 | 12.1 | 0 | 0 | 3.0 | 12.1 | 0 | 0 |
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Time to Event: Graft Loss and Death
The Number of days to participant Graft Loss and death for any reason (NCT02137239)
Timeframe: Up to 728 Days
Intervention | Days (Number) |
---|
| Graft Loss |
---|
Treatment A | 107 |
,Treatment B | 2 |
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Treatment Differences in Therapeutic Modalities
Treatment Received for Biopsy Proven Acute Rejection (Banff Grade IA or Higher), or Humoral (Antibody Mediated) Rejection Treatment regimen: Categorical analysis of CSBPAR episodes by treatment received. (NCT02137239)
Timeframe: at 6, 12 and 24 Months
Intervention | Percentage of participants with CSBPARs (Number) |
---|
| Corticosteroids (6 months) | Lymphocyte depleting agent (6 months) | Plasmapheresis (6 months) | IVIG (6 months) | Rituximab (6 months) | Corticosteroids (12 months) | Lymphocyte depleting agent (12 months) | Plasmapheresis (12 months) | IVIG (12 months) | Rituximab (12 months) | Corticosteroids (24 months) | Lymphocyte depleting agent (24 months) | Plasmapheresis (24 months) | IVIG (24 months) | Rituximab (24 months) |
---|
Treatment A | 7.7 | 0 | 0 | 0 | 0 | 15.4 | 3.8 | 0 | 0 | 0 | 19.2 | 3.8 | 0 | 0 | 0 |
,Treatment B | 9.4 | 6.3 | 0 | 3.1 | 0 | 12.5 | 6.3 | 0 | 3.1 | 0 | 12.5 | 6.3 | 0 | 3.1 | 0 |
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Urine Protein Creatinine Ratio (UPr/Cr)
Urine protein to creatinine ratio (UPr/Cr) at 3, 6, 12 and 24 months post-transplant. (NCT02137239)
Timeframe: Up 24 Months post-transplant
Intervention | mg Protein/mg Creatinine (Mean) |
---|
| At 3 Months | At 6 Months | At 12 Months | At 24 Months |
---|
Treatment A | 0.3146 | 0.3896 | 0.2835 | 0.3940 |
,Treatment B | 0.1412 | 0.1461 | 0.1849 | 0.1685 |
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Mean Cases of Acute Rejection (MCAR) Per Patient
"MCAR represents the average number of acute rejections among all patients in each treatment group. Results were based on rejection episodes with endomyocardial biopsies.~Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection." (NCT00157014)
Timeframe: 52 Weeks
Intervention | MCAR per patient (Mean) |
---|
Tacrolimus - Adult | 0.15 |
Cyclosporine - Adult | 0.17 |
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Mean Cases of Acute Rejection (MCAR) Per Patient (Pediatric Population)
"MCAR represents the average number of acute rejections among all patients in each treatment group. Results were based on rejection episodes with endomyocardial biopsies.~Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection." (NCT00157014)
Timeframe: 52 Weeks
Intervention | MCAR per patient (Mean) |
---|
Tacrolimus - Pediatric | 0.60 |
Cyclosporine - Pediatric | 0.50 |
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Number of Cardiac Rejection Episodes Requiring Treatment
The number of rejection episodes requiring treatment (medications started/ stopped, non-medication treatment, or both) regardless of biopsy grade or presence of hemodynamic compromise. (NCT00157014)
Timeframe: 52 Weeks
Intervention | Rejection Episodes (Number) |
---|
Tacrolimus - Adult | 12 |
Cyclosporine - Adult | 11 |
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Number of Cardiac Rejection Episodes Requiring Treatment (Pediatric Population)
A summary of rejection episodes requiring treatment regardless of biopsy grade or presence of hemodynamic compromise. (NCT00157014)
Timeframe: 52 Weeks
Intervention | Rejection Episodes (Number) |
---|
Tacrolimus - Pediatric | 3 |
Cyclosporine - Pediatric | 3 |
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Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection
Severe Acute Rejection is defined as rejection with ISHLT Grade 4. (NCT00157014)
Timeframe: 52 Weeks
Intervention | Patients (Number) |
---|
Tacrolimus - Adult | 0 |
Cyclosporine - Adult | 0 |
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Number of Patients Requiring Antilymphocyte Antibodies or Steroids for Treatment of Severe Acute Rejection (Pediatric Population)
Severe Acute Rejection was defined as rejection with ISHLT Grade 4. (NCT00157014)
Timeframe: 52 Weeks
Intervention | Patients (Number) |
---|
Tacrolimus - Pediatric | 0 |
Cyclosporine - Pediatric | 0 |
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Time to First Acute Rejection Episode Following de Novo Cardiac Transplant
"Acute Rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Time to first acute rejection is defined as: date of onset - date of transplant." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Days (Mean) |
---|
Tacrolimus - Adult | 55.0 |
Cyclosporine - Adult | 35.60 |
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Time to First Acute Rejection Episode Following de Novo Cardiac Transplant (Pediatric Population)
"Acute Rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Time to first acute rejection is defined as: date of onset - date of transplant." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Days (Mean) |
---|
Tacrolimus - Pediatric | 56.3 |
Cyclosporine - Pediatric | 49.0 |
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Changes in Circulating Markers of Inflammation and Oxidation: Cystatin-C (Pediatric Population)
Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | mg/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Pediatric | 0.77 | 0.84 | -0.01 |
,Tacrolimus - Pediatric | 0.86 | 0.87 | -0.11 |
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Changes in Circulating Markers of Inflammation and Oxidation: F2 Isoprostanes (Pediatric Population)
Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | pg/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Pediatric | 104.68 | 66.48 | -30.07 |
,Tacrolimus - Pediatric | 106.06 | 69.71 | -38.31 |
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Changes in Circulating Markers of Inflammation and Oxidation: hsCRP (Pediatric Population)
Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | mg/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Pediatric | 12.08 | 2.43 | -13.94 |
,Tacrolimus - Pediatric | 30.46 | 26.31 | -7.85 |
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Changes in Circulating Markers of Inflammation and Oxidation: Nitrotyrosine (Pediatric Population)
Change is defined as Week 52 assessment - Pre-Transplant assessment (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | nM (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Pediatric | 12701.21 | 41147.62 | 21514.62 |
,Tacrolimus - Pediatric | 233.08 | 5462.99 | 5148.42 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: BNP
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~BNP= Brain Natriuretic Peptide" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | ng/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 4240.8 | 1856.8 | -1446.7 |
,Tacrolimus - Adult | 4314.8 | 670.1 | -4018.4 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Cystatin-C
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | mg/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 1.29 | 1.48 | 0.27 |
,Tacrolimus - Adult | 1.21 | 1.29 | 0.06 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: E-selectin
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | ng/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 98.96 | 80.93 | -19.16 |
,Tacrolimus - Adult | 90.40 | 68.60 | -18.58 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: F2 Isoprostanes
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | pg/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 53.94 | 50.44 | -3.43 |
,Tacrolimus - Adult | 52.03 | 30.08 | -13.29 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Fibrinogen
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | g/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 4.4 | 3.8 | -0.5 |
,Tacrolimus - Adult | 4.4 | 3.4 | -1.1 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: GSH/GSSG
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~GSH/GSSG= ratio of reduced to oxidised glutathione" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | Ratio (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 58.83 | 53.72 | -5.55 |
,Tacrolimus - Adult | 55.07 | 51.69 | -2.07 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Homocysteine
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | μmol/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 15.9 | 15.8 | 0.7 |
,Tacrolimus - Adult | 14.2 | 13.5 | 0.3 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: hsCRP
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~hsCRP= high-sensitivity C Reactive Protein" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | mg/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 21.83 | 3.95 | -18.69 |
,Tacrolimus - Adult | 32.85 | 3.01 | -34.32 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-18
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | pg/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 496.2 | 427.2 | -71.0 |
,Tacrolimus - Adult | 574.0 | 534.6 | 5.2 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: IL-6
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~IL= Interleukin" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | pg/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 2.54 | 0.90 | -1.56 |
,Tacrolimus - Adult | 3.36 | 0.98 | -2.84 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: MCP-1
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~MCP-1= monocyte chemoattractant protein-1" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | pg/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 193.63 | 180.90 | -16.49 |
,Tacrolimus - Adult | 233.05 | 229.96 | 42.92 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Nitrotyrosine
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | nM (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 482.43 | 368.95 | -99.79 |
,Tacrolimus - Adult | 422.63 | 451.88 | 71.44 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Osteopontin
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | ng/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 11.14 | 10.49 | 0.20 |
,Tacrolimus - Adult | 11.88 | 8.77 | -2.22 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: s-ICAM
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~s-ICAM= soluble-intracellular adhesion molecule" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | ng/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 674.46 | 503.71 | -183.96 |
,Tacrolimus - Adult | 766.58 | 590.30 | -227.58 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: T-bars
"Change is defined as Week 52 assessment - Pre-Transplant assessment.~T-bars = thiobarbituric acid reactive substances" (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | nmol/mL (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 3.91 | 3.14 | -0.77 |
,Tacrolimus - Adult | 3.78 | 3.25 | -0.64 |
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Changes in Circulating Markers of Inflammation, Oxidation, Growth, Apoptosis, Differentiation and Survival: Troponin T
Change is defined as Week 52 assessment - Pre-Transplant assessment. (NCT00157014)
Timeframe: Pre-Transplant and 52 Weeks
Intervention | ug/L (Mean) |
---|
| Pre-Transplant | Week 52 | Change from Pre-Transplant at Week 52 |
---|
Cyclosporine - Adult | 0.28 | 0.04 | -0.27 |
,Tacrolimus - Adult | 0.30 | 0.03 | -0.32 |
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Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria
"Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Patients may report more than one acute rejection." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Rejection Episodes (Number) |
---|
| Total Acute Rejection Episodes | Acute Rejection Episodes with ISHLT Grade ≥3A | Acute Rejection Episodes w/ Hemodynamic Compromise |
---|
Cyclosporine - Adult | 8 | 7 | 2 |
,Tacrolimus - Adult | 8 | 3 | 6 |
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Number of Acute Rejection Episodes by International Society of Heart and Lung Transplantation (ISHLT) Criteria (Pediatric Population)
"Acute rejection was defined as a rejection with ISHLT Grade ≥3A or by the presence of hemodynamic compromise.~ISHLT Grades ≥3A include: Multifocal Moderate Rejection; Diffuse, Borderline Severe Acute Rejection; and Severe Acute Rejection.~Patients may report more than one rejection episode." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Rejection Episodes (Number) |
---|
| Total Acute Rejection Episodes | Acute Rejection Episodes with ISHLT Grade ≥3A | Acute Rejection Episodes w/ Hemodynamic Compromise |
---|
Cyclosporine - Pediatric | 3 | 3 | 0 |
,Tacrolimus - Pediatric | 3 | 3 | 0 |
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Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52
A successful steroid taper or withdrawal was defined as steroids (prednisone) being discontinued or tapered to the suggested dose level after week 26. (NCT00157014)
Timeframe: 26 Weeks and 52 Weeks
Intervention | Patients (Number) |
---|
| Week 26 | Week 52 |
---|
Cyclosporine - Adult | 16 | 29 |
,Tacrolimus - Adult | 22 | 33 |
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Number of Patients With Successful Steroid Taper or Withdrawal at Weeks 26 and 52 (Pediatric Population)
A successful steroid taper or withdrawal was defined as steroids (prednisone) being discontinued or tapered to the suggested dose level after week 26. (NCT00157014)
Timeframe: 26 Weeks and 52 Weeks
Intervention | Patients (Number) |
---|
| Week 26 | Week 52 |
---|
Cyclosporine - Pediatric | 3 | 1 |
,Tacrolimus - Pediatric | 2 | 1 |
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Number of Patients With Treatment Failure and Crossover for Treatment Failure
"Treatment failure was defined as death, re-transplantation, withdrawal due to an Adverse Event, or a switch of main immunosuppressant medication, whichever came first.~Crossover was defined as a switch from originally administered primary immunosuppressant (tacrolimus or cyclosporine) to the alternate primary immunosuppressant." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Patients (Number) |
---|
| Treatment Failures | Crossover for Treatment Failures |
---|
Cyclosporine - Adult | 11 | 8 |
,Tacrolimus - Adult | 6 | 2 |
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Number of Patients With Treatment Failure and Crossover for Treatment Failure (Pediatric Population)
"Treatment failure was defined as death, re-transplantation, withdrawal due to an Adverse Event, or a switch of main immunosuppressant medication, whichever came first.~Crossover was defined as a switch from originally administered primary immunosuppressant (tacrolimus or cyclosporine) to the alternate primary immunosuppressant." (NCT00157014)
Timeframe: 52 Weeks
Intervention | Patients (Number) |
---|
| Treatment Failures | Crossover for Treatment Failures |
---|
Cyclosporine - Pediatric | 3 | 3 |
,Tacrolimus - Pediatric | 1 | 0 |
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The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies
"The markers assessed were p-ERK ½ (phosphorylated extracellular signal-regulated kinase), p-JNK (phosphorylated jun N-terminal kinase) and p-p38 MAPK (phosphorylated mitogen-activated protein kinase).~The data for each biopsy marker were expressed as a ratio of its densitometry / densitometry of glyceraldehyde-3-phosphate dehydrogenase (GAPDH).~Change is defined as Week 52 assessment- Week 2 assessment." (NCT00157014)
Timeframe: 2 Weeks and 52 Weeks
Intervention | Densitometry / Densitometry of GAPDH (Mean) |
---|
| p-ERK ½ - Week 2 | p-ERK ½ - Week 52 | p-ERK ½ - Change from Week 2 | p-JNK - Week 2 | p-JNK - Week 52 | p-JNK - Change from Week 2 | p-p38 MAPK - Week 2 | p-p38 MAPK - Week 52 | p-p38 MAPK - Change from Week 2 |
---|
Cyclosporine - Adult | 0.90 | 0.79 | -0.05 | 1.23 | 1.46 | 0.22 | 0.54 | 0.77 | 0.23 |
,Tacrolimus - Adult | 0.70 | 0.87 | 0.05 | 1.10 | 1.33 | 0.03 | 0.48 | 0.63 | 0.14 |
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The Change in the Markers of Growth, Apoptosis, Inflammation and Oxidation Measured in Endomyocardial Biopsies (Pediatric Population)
"The markers assessed were p-ERK ½, p-JNK and p-p38 MAPK.~The data for each biopsy marker were expressed as a ratio of its densitometry / densitometry of glyceraldehyde-3-phosphate dehydrogenase (GAPDH).~Change is defined as Week 52 assessment- Week 2 assessment." (NCT00157014)
Timeframe: 2 Weeks and 52 Weeks
Intervention | Densitometry / Densitometry of GAPDH (Mean) |
---|
| p-ERK ½ - Week 2 | p-ERK ½ - Week 52 | p-ERK ½ - Change from Week 2 | p-JNK - Week 2 | p-JNK - Week 52 | p-JNK - Change from Week 2 | p-p38 MAPK - Week 2 | p-p38 MAPK - Week 52 | p-p38 MAPK - Change from Week 2 |
---|
Cyclosporine - Pediatric | 1.67 | 1.22 | -0.27 | 0.91 | 0.82 | 0.04 | 0.43 | 0.58 | 0.34 |
,Tacrolimus - Pediatric | 1.74 | 0.93 | -0.09 | 1.17 | 0.57 | -0.21 | 0.83 | 0.24 | -0.04 |
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The Incidence of Biopsy-proven Acute Allograft Rejection During the First 12 Months of Transplant.
The incidence of rejection is determined by the proportion of patients experiencing biopsy proven acute allograft rejection during the first 12 months post-transplant. (NCT00166712)
Timeframe: Within 12 months post kidney transplant
Intervention | Participants (Number) |
---|
Groups | 4 |
Group 2 | 6 |
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Renal Function (Nankivell Formula) at Month 12 Post Transplantation.
Renal function at the end of the trial assessed as mean absolute values of the glomerular filtration rate (GFR) calculated by Nankivell formula 12 months after renal transplantation. The Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^2 + C ; where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. Estimated GFR is expressed in mL/min per 1.73m^2. (NCT00154310)
Timeframe: at Month 12 post transplantation
Intervention | mL/min /1.73m^2 (Mean) |
---|
Everolimus + Mycophenolate Sodium | 71.84 |
Cyclosporine + Mycophenolate Sodium | 61.24 |
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Changes in Cardiovascular Risk From Month 4.5 to Final Assessment at Month 12
An updated 1991 Framingham coronary prediction algorithm was used to estimate the total risk of developing coronary heart diseases (CHD) over the course of 10 years. Risk was calculated separately for male and females. To calculate risk, points were assigned for each of the following risk factors: age, levels of LDL cholesterol, HDL cholesterol, blood pressure, cigarette smoking, and diabetes mellitus. The sum of the individual risk factor points gives a total point score, which ranges from -5 to 18 for men and -16 to 24 for women. Higher points indicate a higher risk for CHD. (NCT00154310)
Timeframe: Month 4.5 and Month 12
Intervention | Points (Mean) |
---|
| Male (n= 55, 37) | Female (n= 22, 35) | Total Population (n= 77, 72) |
---|
Cyclosporine + Mycophenolate Sodium | 0.1 | 0.8 | 0.4 |
,Everolimus + Mycophenolate Sodium | 0.5 | 0.0 | 0.4 |
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Number of Participants Who Experienced an Adverse Event or Serious Adverse Event
Additional information about the number of participants who experienced Adverse Events (greater than 5%) or Serious Adverse Events can be found in the Adverse Event section. (NCT00154310)
Timeframe: Aes from end of core study period (month 12) to end of follow-up period (month 60)
Intervention | Participants (Number) |
---|
| Adverse Events | Serious Adverse Events |
---|
Cyclosporine + Mycophenolate Sodium | 145 | 86 |
,Everolimus + Mycophenolate Sodium | 155 | 95 |
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Number of Participants With Occurrence of Biopsy Proven Acute Rejection (BPAR), Graft Loss or Death
The number of participants with occurrence of biopsy proven acute rejection (BPAR), graft loss, or death up to Month 12 during the randomized treatment period. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III according to Banff 97 classification. A graft core biopsy was performed prior to 24 hours following initiation of graft rejection therapy. The allograft is presumed to be lost on the day the patient starts dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00154310)
Timeframe: Up to Month 12
Intervention | Participants (Number) |
---|
| BPAR: Yes | BPAR: No | Graft Loss: Yes | Graft Loss: No | Death: Yes | Death: No |
---|
Cyclosporine + Mycophenolate Sodium | 5 | 141 | 0 | 146 | 1 | 145 |
,Everolimus + Mycophenolate Sodium | 15 | 139 | 0 | 154 | 0 | 154 |
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Number of Participants With Occurrence of Treatment Failures
Treatment failures defined as a composite endpoint of biopsy proven acute rejection, graft loss, death, loss to follow up and discontinuations due to lack of efficacy or toxicity, or conversion to another regimen (at least one condition must be present). (NCT00154310)
Timeframe: up to or at Month 12
Intervention | Participants (Number) |
---|
| Treatment failure: Yes | Treatment failure: No |
---|
Cyclosporine + Mycophenolate Sodium | 23 | 123 |
,Everolimus + Mycophenolate Sodium | 29 | 125 |
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Number of Participants With an Episode of Clinically-suspected and Biopsy-proven Acute Rejection (CSPAR)
No participant was to receive treatment for acute rejection without a biopsy to confirm the diagnosis. CSPAR=Clinically-suspected rejection, defined as an increase in serum creatinine ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function, and biopsy-proven rejection, which includes all cases in which a biopsy was read by the central pathologist as demonstrating acute rejection regardless of the reason why the biopsy was performed. (NCT00035555)
Timeframe: By Month 6 posttransplant (From Day 1 to Month 6)
Intervention | Participants (Number) |
---|
Belatacept: More Intensive (MI) Regimen | 5 |
Belatacept: Less Intensive (LI) Regimen | 4 |
Cyclosporine Regimen | 6 |
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Mean Iohexol Clearance
Iohexol, a true glomerular filtration marker, is used to measure glomerular filtration rate. (NCT00035555)
Timeframe: By Months 1, 6, and 12 posttransplant (Day 1 to Months 1, 6, and 12)
Intervention | mL/min per 1.73 m^2 (Mean) |
---|
| Month 1 (n=53, 51, 48) | Month 6 (n=41, 41, 31) | Month 12 (n=32, 37, 27) |
---|
Belatacept: Less Intensive (LI) Regimen | 60.2 | 64.5 | 62.1 |
,Belatacept: More Intensive (MI) Regimen | 59.7 | 62.2 | 66.3 |
,Cyclosporine Regimen | 54.0 | 56.0 | 53.5 |
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Mean LDL Cholesterol, HDL Cholesterol, Total Cholesterol, Triglyceride, and Non-HDL Levels
LDL=low-density lipoprotein; HDL=high-density lipoprotein. Total cholesterol=LDL + HDL + very low-density (VLDL) cholesterol. VLDL=triglycerides divided by 5. Non-HDL cholesterol=Total cholesterol minus HDL cholesterol. (NCT00035555)
Timeframe: By Months 1, 6, and 12 posttransplant (Day 1 to Months 1, 6, and 12)
Intervention | mg/dL (Mean) |
---|
| LDL cholesterol: Month 1 (n=69, 69, 66) | LDL cholesterol: Month 6 (n=63, 66, 55) | LDL cholesterol: Month 12 (n=61, 60, 52) | HDL cholesterol: Month 1 (n=68, 68, 64) | HDL cholesterol: Month 6 (n=62, 65, 62) | HDL cholesterol: Month 12 (n=60, 57, 48) | Total cholesterol: Month 1 (n=69, 69, 65) | Total cholesterol: Month 6 (n=63, 65, 54) | Total cholesterol: Month 12 (n=60, 58, 50) | Triglycerides: Month 1 (n=69, 69, 65) | Triglycerides: Month 6 (n=63, 65, 54) | Triglycerides: Month 12 (n=60, 58, 50) | Non-HDL: Month 1 (n=68, 68, 64) | Non-HDL cholesterol: Month 6 (n=62, 64, 51) | Non-HDL cholesterol: Month 12 (n=59, 56, 48) |
---|
Belatacept: Less Intensive (LI) Regimen | 120 | 121 | 125 | 68 | 56 | 56 | 210 | 202 | 201 | 147 | 168 | 152 | 142 | 143 | 144 |
,Belatacept: More Intensive (MI) Regimen | 129 | 125 | 120 | 64 | 54 | 53 | 222 | 204 | 198 | 168 | 177 | 176 | 159 | 150 | 145 |
,Cyclosporine Regimen | 137 | 131 | 125 | 70 | 62 | 59 | 239 | 224 | 212 | 185 | 198 | 186 | 169 | 165 | 151 |
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Number of Participants Meeting Marked Abnormality Criteria for Select Hemolytic, Blood Chemistry, and Urinalysis Laboratory Test Results
Normal laboratory values: Hemoglobin (g/dL): Males (18-64 years) 13.8-17, (65 years and older) 11.8-16.8; Females (18-64 years) 12.0-15.6, F (65 years and older) 11.1-15.5. Platelets (per mm^3) 130,000-400,000. Leukocytes (18 years and older) 3.8-10.8 1000/uL. ALT (u/L)(13 years and older) 0-48. (NCT00035555)
Timeframe: Days 8 and Months 1, 3, 6, 9, and 12 posttransplant (from Day 1)
Intervention | Participants (Number) |
---|
| Hemoglobin, low | Hemoglobin, high | Platelet count, low | Platelet count, high | Leukocytes, low | Leukocytes, high | Alanine aminotransferase (ALT), low | ALT, high |
---|
Belatacept: More Intensive (MI) Regimen | 13 | NA | 1 | NA | 1 | NA | NA | 9 |
,Belatacept:Less Intensive (LI) Regimen | 7 | NA | 0 | NA | 4 | NA | NA | 3 |
,Cyclosporine Regimen | 9 | NA | 3 | NA | 5 | NA | NA | 7 |
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Number of Participants With Hypertension
Hypertension is defined as diastolic blood pressure ≥90 mm Hg and/or systolic blood pressure ≥140 mm Hg or, the use of any antihypertensive medication. (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)
Intervention | Participants (Number) |
---|
| At Month 6 | At Month 12 |
---|
Belatacept: Less Intensive (LI) Regimen | 15 | 12 |
,Belatacept: More Intensive (MI) Regimen | 16 | 14 |
,Cyclosporine Regimen | 18 | 11 |
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Number of Participants With Posttransplant Diabetes Mellitus
Posttransplant diabetes mellitus is defined as the need for treatment of hyperglycemia with either an oral agent or insulin for a total of >4 weeks or hemoglobin A1c (HbA1c) >7% in a participant not known to be diabetic prior to transplantation (NCT00035555)
Timeframe: By Months 1, 3, 6, 9, and 12 posttransplant (Day 1 to Months 1, 3, 6, 9, and 12 )
Intervention | Participants (Number) |
---|
| Up to Month 1: All events | Month 1: Hyperglycemia medication | Month 1: HbA1c >7% | Up to Month 3: All events | Up to Month 3: Hypoglycemic medication | Up to Month 3: HbA1c >7% | Up to Month 6: All events | Up to Month 6: Hyperglycemia medication | Up to Month 6: HbA1c >7% | Up to Month 9: All events | Up to Month 9: Hyperglycemia medication | Up to Month 9: HbA1c >7% | Up to Month 12: All events | Up to Month 12: Hyperglycemia medication | Up to Month 12: HbA1c >7% |
---|
Belatacept: Less Intensive (LI) Regimen | 0 | 0 | 0 | 1 | 0 | 1 | 3 | 0 | 3 | 3 | 0 | 3 | 4 | 0 | 4 |
,Belatacept: More Intensive (MI) Regimen | 5 | 0 | 5 | 5 | 0 | 5 | 6 | 0 | 6 | 8 | 2 | 7 | 8 | 2 | 7 |
,Cyclosporine Regimen | 1 | 1 | 0 | 3 | 2 | 1 | 4 | 2 | 2 | 5 | 2 | 3 | 7 | 3 | 4 |
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Percentage of Participants Who Had Chronic Allograft Nephropathy
Based on postbaseline biopsies (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)
Intervention | Percentage of participants (Number) |
---|
| Month 6 (n= 32, 33, 27) | Month 12 (n=52, 54, 45) |
---|
Belatacept: Less Intensive (LI) Regimen | 9.1 | 20.4 |
,Belatacept: More Intensive (MI) Regimen | 18.8 | 28.8 |
,Cyclosporine Regimen | 33.3 | 44.4 |
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Percentage of Participants Who Used Antihypertensive Medication
Hypertension is defined as diastolic blood pressure ≥90 mm Hg and/or systolic blood pressure ≥140 mm Hg (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)
Intervention | Percentage of participants (Number) |
---|
| Month 6: Total requiring at least 1 medication | Month 6: Requiring 1 medication | Month 6: Requiring 2 medications | Month 6: Requiring 3 medications | Month 6: Requiring 4 medications | Month 6: Requiring 5 medications | Month 6: Requiring 6 medications | Month 6: Requiring >6 medications | Month 12: Total requiring at least 1 medication | Month 12: Requiring 1 medication | Month 12: Requiring 2 medications | Month 12: Requiring 3 medications | Month 12: Requiring 4 medications | Month 12: Requiring 5 medications | Month 12: Requiring 6 medications | Month 12: Requiring >6 medications |
---|
Belatacept: Less Intensive (LI) Regimen | 78.6 | 27.1 | 28.6 | 14.3 | 7.1 | 1.4 | 0.0 | 0.0 | 71.6 | 26.9 | 9.0 | 9.0 | 7.5 | 0.0 | 0.0 | 0.0 |
,Belatacept: More Intensive (MI) Regimen | 87.7 | 24.7 | 23.3 | 23.3 | 11.0 | 2.7 | 2.7 | 0.0 | 79.7 | 21.7 | 23.2 | 23.2 | 8.7 | 5.8 | 1.4 | 0.0 |
,Cyclosporine Regimen | 88.4 | 21.7 | 31.9 | 21.7 | 11.6 | 1.4 | 0.0 | 0.0 | 86.4 | 20.3 | 22.0 | 22.0 | 15.3 | 3.4 | 0.0 | 0.0 |
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Percentage of Participants With Acute Rejection or Presumed Acute Rejection (PAR)
Throughout this study, acute rejection=clinically-suspected and biopsy-proven acute rejection (BPAR). Clinically-suspected rejection is defined as an increase in serum creatinine ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function. BPAR includes all cases in which a biopsy was read by the central pathologist as demonstrating acute rejection regardless of the reason why the biopsy was performed. PAR is defined as an elevation in SCr ≥0.5 mg/dL compared with the baseline value in the absence of other factors known to adversely affect renal function that led the investigator to suspect that the participant had experienced acute rejection, and in whom either the biopsy did not confirm acute rejection and the participant received treatment for acute rejection or the participant received treatment for acute rejection without a biopsy to confirm the diagnosis. (NCT00035555)
Timeframe: By Months 6 and 12 posttransplant (Day 1 to Months 6 and 12)
Intervention | Percentage of participants (Number) |
---|
| Month 6: Acute rejection or PAR | Month 12: Acute rejection or PAR | Month 6: PAR | Month 12: PAR |
---|
Belatacept: Less Intensive (LI) Regimen | 9 | 10 | 3 | 4 |
,Belatacept: More Intensive (MI) Regimen | 11 | 11 | 5 | 5 |
,Cyclosporine Regimen | 10 | 11 | 1 | 3 |
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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) or Who Received Treatment for Acute Rejection
BPAR includes all cases in which a biopsy read by the central pathologist demonstrates acute rejection, regardless of the reason that the biopsy was performed. A participant was reported as having had an episode of treated acute rejection if he or she received antirejection therapy during an episode of rejection (clinically-suspected or biopsy-proven rejection). (NCT00035555)
Timeframe: By Months 3, 6, and 12 posttransplant (Day 1 to Months 3, 6, and 12)
Intervention | Percentage of participants (Number) |
---|
| By Month 3 | By Month 6 | By Month 12 |
---|
Belatacept: Less Intensive (LI) Regimen | 29.6 | 32.4 | 38.0 |
,Belatacept: More Intensive (MI) Regimen | 21.6 | 23.0 | 28.4 |
,Cyclosporine Regimen | 17.8 | 24.7 | 27.4 |
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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR) Through Months 6 and 12
BPAR includes all cases in which a biopsy read by the central pathologist demonstrates acute rejection, regardless of the reason that the biopsy was performed. (NCT00035555)
Timeframe: Through Months 6 and 12 posttransplant (From Day 1 to Months 6 and 12)
Intervention | Percentage of participants (Number) |
---|
| Up to 6 months | Up to 12 months |
---|
Belatacept: Less Intensive (LI) Regimen | 23.9 | 29.6 |
,Belatacept: More Intensive (MI) Regimen | 14.9 | 18.9 |
,Cyclosporine Regimen | 17.8 | 17.8 |
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AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)
Bioequivalence based on AUC0-inf (NCT00908128)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil | 24.5141 |
CellCept® | 24.9890 |
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AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of the Last Non-zero Concentration
Bioequivalence based on AUC0-t (NCT00908128)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil | 23.3091 |
CellCept® | 23.5366 |
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Cmax - Maximum Observed Concentration
Bioequivalence based on Cmax (NCT00908128)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg/mL (Mean) |
---|
Mycophenolate Mofetil | 7.4578 |
CellCept® | 8.2195 |
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Event-free Survival
(NCT00611351)
Timeframe: 2 years post transplant
Intervention | Participants (Count of Participants) |
---|
Unrelated Donor Allogeneic | 2 |
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Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
(NCT00611351)
Timeframe: at day 100 post transplantation
Intervention | Participants (Count of Participants) |
---|
Unrelated Donor Allogeneic | 4 |
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Overall Survival
(NCT00611351)
Timeframe: 2 years post transplant
Intervention | Participants (Count of Participants) |
---|
Unrelated Donor Allogeneic | 2 |
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Transplantation-related Mortality at 100 Days Post-transplantation
(NCT00611351)
Timeframe: at the 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
Unrelated Donor Allogeneic | 2 |
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Incidence of Acute and Chronic Graft-versus-host Disease
Incidence of acute and chronic graft-versus-host disease. Acute GVHD usually occurs during the first three months following transplant. Chronic GVHD usually develops after the third month post-transplant. (NCT00571662)
Timeframe: twice weekly until day 100 up to 1 year post transplant
Intervention | Percent of Particpants (Number) |
---|
| Acute GVHD | Chronic GVHD |
---|
Cohort I | 31 | 33 |
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Kinetics of Immunologic Reconstitution
Rate of return of immune cells after allogeneic transplantation (NCT00571662)
Timeframe: at day 100 post transplantation
Intervention | percentage of cells in peripheral blood (Median) |
---|
| CD3 cells | CD4 cells | CD8 cells |
---|
Day + 28 Post Transplant | 7 | 3.5 | 1.7 |
,Study Baseline | 13 | 5 | 5 |
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Percent of Participants With Chimerism: Full Donor Chimerism Defined as >95% Donor CD3+ Cell in Blood as Assessed by DNA Fingerprinting
the efficacy of the regimen as determined by engraftment rate and establishment of donor hematopoietic chimerism at day +28 and day +70. (NCT00571662)
Timeframe: days +28 and +70
Intervention | percent of participants (Median) |
---|
| Day 28 | Day 70 |
---|
Cohort I | 85 | 90 |
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Responses to Therapy
event-free and overall survival at 12 months (NCT00571662)
Timeframe: every 6 mo. up to 2 years
Intervention | Percent of Participants (Number) |
---|
| Event free survival | Overall survival |
---|
Cohort I | 52 | 59 |
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Toxicity for the Combination of Pentostatin and Low Dose Total Body Irradiation (TBI)
(NCT00571662)
Timeframe: Conditioning regimen to count recovery (D + 28 post transplant)
Intervention | Participants (Count of Participants) |
---|
| Absolute neutrophil count < 500/mm^3 | platelet count < 20,000/mm^3 | Grade 3 or 4 Fever | Grade 3 or 4 hypokalemia | Grade 3 or 4 bacteremia | Grade 3 or 4 infection | Grade 3 or 4 renal toxicity | Grade 3 or 4 thromboembolism |
---|
Cohort I | 40 | 29 | 2 | 1 | 2 | 6 | 1 | 1 |
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Determine the Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Diagnosis of Engraftment Loss.
Number of patients with engraftment loss. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 0 |
Arm II (MMF-30, Filgrastim) | 1 |
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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Chronic Graft vs Host Disease (GVHD)
The differences in the rates of chronic GVHD between patients randomized to MMF-30 (control cohort) and MMF-15 (investigational cohort) (NCT02593123)
Timeframe: 60 Days following stem cell transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 9 |
Arm II (MMF-30, Filgrastim) | 7 |
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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Diagnosed With Opportunistic Infections
The number of patients diagnosed with an opportunistic infections. (NCT02593123)
Timeframe: 60 Days following stem cell transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 7 |
Arm II (MMF-30, Filgrastim) | 9 |
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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) With Acute Graft Vs Host Disease (GVHD)
The number of patients diagnosed with acute acute GVHD between patients randomized to MMF-30 (control cohort) and MMF-15 (investigational cohort) (NCT02593123)
Timeframe: 60 Days following stem cell transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 4 |
Arm II (MMF-30, Filgrastim) | 8 |
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Differences Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)Diagnosed With Differences in the Rates of Engraftment Syndrome.
Number of patients diagnosed with engraftment syndrome. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 0 |
Arm II (MMF-30, Filgrastim) | 0 |
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Differences in the Rates of Achieving Donor Chimerisms (the Percentage of DNA in the Sample Which Comes From the Donor) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).
Number of patients that achieved donor chimerisms by day 100. (NCT02593123)
Timeframe: 100 Days following Stem Cell Transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF-15, Sargramostim) | 15 |
Arm II (MMF-30, Filgrastim) | 11 |
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Differences in the Rates of T-cell Recovery Kinetics Following Stem Cell Transplantation (SCT) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort).
Number of T Cells 10E3 per microL per arm indicating rates of T-cell recovery by Day 100 following SCT. (NCT02593123)
Timeframe: 100 Days Following Stem Cell Transplantation
Intervention | 10^3 *cells* per microliter (Mean) |
---|
Arm I (MMF-15, Sargramostim) | 313.6 |
Arm II (MMF-30, Filgrastim) | 222.2 |
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The Difference Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort) Day 60 Donor-derived (dd) Cluster of Differentiation (CD)3 10E3per microL Counts.
Day 60 donor-derived (dd) cluster of differentiation (CD)3 counts measured by 10E3per microL. (NCT02593123)
Timeframe: 60 Days Following Stem Cell Transplant
Intervention | 10^3 *cells* per microliter (Mean) |
---|
Arm I (MMF-15, Sargramostim) | 383.0 |
Arm II (MMF-30, Filgrastim) | 254.1 |
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The Probability of Overall Survival (OS) Between Patients Randomized to MMF-30 (Control Cohort) and MMF-15 (Investigational Cohort)
Overall survival (days to event or survival: time-to-event; survival: categorical) (NCT02593123)
Timeframe: Randomization up to 2 years
Intervention | Probablility of 2 year survival (Number) |
---|
Arm I (MMF-15, Sargramostim) | 0.78 |
Arm II (MMF-30, Filgrastim) | 0.5 |
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Number of Participants With Absolute Neutrophil Count >= 500/Mcl for 3 Consecutive Measurements on Different Days and Platelet Count > 20,000/mm^3 With no Platelet Transfusions in the Preceding 7 Days
To determine engraftment of neutrophils and platelets at 28 days following alpha/beta T-cell depletion using Human Leukocyte Antigen (HLA) haploidentical donors for stem cell transplant in relapsed lymphoma. (NCT02652468)
Timeframe: At day 28 after transplantation
Intervention | Participants (Count of Participants) |
---|
Peripheral Blood Stem Cell Transplant | 11 |
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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
Intervention | Participants (Count of Participants) |
---|
Peripheral Blood Stem Cell Transplant | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Peripheral Blood Stem Cell Transplant | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Peripheral Blood Stem Cell Transplant | 0 |
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Overall Survival (OS)
(NCT02652468)
Timeframe: Median follow up of 1689 days
Intervention | days (Median) |
---|
Peripheral Blood Stem Cell Transplant | 352 |
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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
Intervention | days (Median) |
---|
Peripheral Blood Stem Cell Transplant | 352 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 13 |
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Disease-free Survival
Number of study participants who are alive and remains in complete remission after transplant. (NCT01300572)
Timeframe: 100 days after transplant
Intervention | participants (Number) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 7 |
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Duration of Remission
"Median time to relapse after achieving complete remission (CR). CR is defined as complete resolution of all signs of myelodysplasia or leukemia for at least 4 weeks with all of the following:~Normal bone marrow with blasts <5% with normal cellularity, normal megakaryopoiesis, > 15% erythropoiesis and > 25% granulocytopoiesis~Normalization of blood counts (no blasts, platelets > 100000/mm3, granulocytes >1500/mm3)~No extramedullary disease.~Relapse Criteria:~After CR: >5% blasts in the bone marrow and/or peripheral blood~After partial remission (PR): increase of blasts cells in the marrow to >50% of those during PR~Extramedullary disease confirmed cytologically or histologically." (NCT01300572)
Timeframe: 1 year
Intervention | days (Median) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 213 |
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Overall Survival
Number of participants who are still alive after transplant with or without disease. (NCT01300572)
Timeframe: Up to 5 years
Intervention | Participants (Count of Participants) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 7 |
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Rates of Donor Chimerism
Number of participants who has 100% donor chimerism within 100 days after transplant (NCT01300572)
Timeframe: Up to 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 14 |
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Rates of Engraftment
Average number of days to ANC >= 500 after transplant (NCT01300572)
Timeframe: Up to 84 days post-transplant
Intervention | days (Mean) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 16 |
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The MTD of Radiation Delivered Via 90Y-DOTA-BC8 When Combined With FLU and 2 Gy TBI as a Preparative Regimen for Patients Aged ≥ 18 With Advanced AML, ALL, and High-risk MDS.
The MTD will be defined as the dose that is associated with a true DLT rate of 25%. The highest dose achieved was 28 Gy but none of the patients experienced a DLT. Thus, the MTD was not reached. (NCT01300572)
Timeframe: Within the first 30 days following transplant
Intervention | Gy (Number) |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 28 |
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Estimation of Absorbed Radiation Doses to Normal Organs, Marrow and Tumor
The amount of energy absorbed per unit weight of the organ or tissue is called absorbed dose and is expressed in units of gray (Gy). One gray dose is equivalent to one joule radiation energy absorbed per kilogram of organ or tissue weight. (NCT01300572)
Timeframe: Approximately day -20 to day -12 prior to transplant
Intervention | Gy (Mean) |
---|
| Average absorbed dose to the marrow | Average absorbed dose to the liver | Average abosorbed dose total body | Average absorbed dose to the spleen |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 11.4 | 17.2 | 3.1 | 70 |
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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
Intervention | Participants (Count of Participants) |
---|
| Severe refractory GVHD | Bacterial infection |
---|
Treatment (90Y-BC8, Allogeneic PBSC or Bone Marrow Transplant) | 1 | 1 |
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Percentage of Participants Surviving
(NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28
Intervention | percentage of participants (Number) |
---|
MMF Monotherapy | 97.7 |
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Percentage of Participants With Acute Rejection
Diagnosis of acute rejection was suspected in any participant with an increase in serum creatinine greater than or equal to (≥) 25 percent (%). All suspected acute rejections were confirmed by biopsy. The start date of acute rejection was identified as the date of biopsy. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28
Intervention | percentage of participants (Number) |
---|
MMF Monotherapy | 9.1 |
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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR)
BPAR was defined according to 1997 Banff Criteria as a biopsy Banff grade of IA, IB, IIA, IIB, or III. Grade IA was defined as significant interstitial infiltration with greater than (>)25% of parenchyma affected, and foci of moderate tubulitis with >4 mononuclear cells per tubular cross section or group of 10 tubular cells. Grade IB was defined as significant interstitial infiltration with >25% parenchyma affected, and foci of severe tubulitis with >10% mononuclear cells per tubular cross section or group of 10 tubular cells. Grade IIA was defined as mild to moderate intimal arteritis. Grade IIB was defined as severe intimal arteritis comprising >25% of the luminal area. Grade III was defined as transmural arteritis and/or arterial fibrinoid changes and necrosis of medial smooth muscle cells. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28
Intervention | percentage of participants (Number) |
---|
MMF Monotherapy | 4.5 |
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Percentage of Participants With Graft Loss
An allograft was presumed to be lost if a participant started dialysis and was not able to subsequently be removed from dialysis. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28
Intervention | percentage of participants (Number) |
---|
MMF Monotherapy | 2.3 |
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Time to Rejection
The mean time, in days, from the date of enrollment to date of biopsy confirming acute rejection. (NCT01292226)
Timeframe: Day 1, Weeks 2, 4, 12, 24, and 28
Intervention | days (Mean) |
---|
MMF Monotherapy | 23.67 |
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Free MPA (mcg/mL) by Visit
Drug quantification of free MPA in the plasma was measured at T = 0, 40, and 120 mins. (NCT01292226)
Timeframe: Weeks 2, 4, 12, 24, safety follow-up (Week 28), and any unscheduled visits
Intervention | mcg/mL (Mean) |
---|
| T=0, Week 2 (n=40) | T=0, Week 4 (n=39) | T=0, Week 12 (n=33) | T=0, Week 24 (n=32) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, overall mean value (n=154) | T=40, Week 2 (n=41) | T=40, Week 4 (n=42) | T=40, Week 12 (n=35) | T=40, Week 24 (n=35) | T=40, safety follow-up (n=3) | T=40, unscheduled visit (n=7) | T=40, overall mean value (n=163) | T=120, Week 2 (n=43) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=35) | T=120, safety follow-up (n=3) | T=120, unscheduled visit (n=7) | T=120, overall mean value (n=165) |
---|
MMF Monotherapy | 0.03 | 0.04 | 0.03 | 0.03 | 0.01 | 0.04 | 0.03 | 0.11 | 0.11 | 0.11 | 0.38 | 0.06 | 0.09 | 0.17 | 0.11 | 0.11 | 0.16 | 0.10 | 0.10 | 0.07 | 0.12 |
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IMPDH Expression I by Visit and Timepoint
IMPDH I gene expression was measured by real time polymerase chain reaction (QRT-PCR) based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of messenger ribonucleic acid (mRNA) copies per cell (copies/cell). (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits
Intervention | number of mRNA copies/cell (Mean) |
---|
| T=0, BL (n=44) | T=0, Week 2 (n=41) | T=0, Week 4 (n=42) | T=0, Week 12 (n=34) | T=0, Week 24 (n=34) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, mean value (n=205) | T=120, BL (n=0) | T=120, Week 2 (n=43) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=34) | T=120, safety follow-up (n=3) | T=120, unscheduled visit (n=7) | T=120, mean value (n=164) |
---|
MMF Monotherapy | 2.32 | 32.29 | 3.16 | 4.10 | 1.95 | 11803.48 | 4.00 | 181.48 | 0 | 4146.17 | 1692.11 | 1556.06 | 3.02 | 107.30 | 1038.52 | 1899.45 |
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IMPDH Expression II by Visit and Timepoint
IMPDH II gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits
Intervention | number of mRNA copies/cell (Mean) |
---|
| T=0, BL (n=44) | T=0, Week 2 (n=41) | T=0, Week 4 (n=42) | T=0, Week 12 (n=34) | T=0, Week 24 (n=34) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, mean value (n=205) | T=120, BL (n=0) | T=120, Week 2 (n=43) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=34) | T=120, safety follow-up (n=3) | T=120, unscheduled visit (n=7) | T=120, mean value (n=164) |
---|
MMF Monotherapy | 115.33 | 113.43 | 117.16 | 112.43 | 114.21 | 123.86 | 116.57 | 114.82 | 0 | 304.64 | 148.32 | 143.11 | 109.00 | 140.51 | 167.19 | 180.71 |
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Inosine MonoPhosphate DeHydrogenase (IMPDH) Activity by Visit and Timepoint
"IMPDH activity in peripheral blood mononuclear cells (PBMCs) was measured at 2 timepoints per visit, 0 and 120 minutes and presented in enzyme units. The unit of measure of enzyme activity is U. One U is defined as the amount of the enzyme that produces a certain amount of enzymatic activity that is, the amount that catalyzes the conversion of 1 micro mole of substrate per minute under pre-specified conditions (temperature, pH)." (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits
Intervention | enzyme units (Mean) |
---|
| T=0, BL (n=44) | T=0, Week 2 (n=42) | T=0, Week 4 (n=42) | T=0, Week 12 (n=35) | T=0, Week 24 (n=34) | T=0, safety follow-up (n=0) | T=0, unscheduled visit (n=7) | T=0, mean value (n=204) | T=120, BL (n=0) | T=120, Week 2 (n=42) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=34) | T=120, safety follow-up (n=0) | T=120, unscheduled visit (n=7) | T=120, mean value (n=160) |
---|
MMF Monotherapy | 5.01 | 3.96 | 3.89 | 6.74 | 9.58 | 0 | 6.00 | 5.65 | 0 | 3.06 | 3.10 | 3.75 | 6.39 | 0 | 3.89 | 3.97 |
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Interleukin 8 (IL-8) Expression by Visit and Timepoint
IL-8 gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits
Intervention | number of mRNA copies/cell (Mean) |
---|
| T=0, BL (n=44) | T=0, Week 2 (n=41) | T=0, Week 4 (n=42) | T=0, Week 12 (n=34) | T=0, Week 24 (n=34) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, mean value (n=205) | T=120, BL (n=0) | T=120, Week 2 (n=43) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=34) | T=120, safety follow-up (n=3) | T=120, unscheduled visit (n=7) | T=120, mean value (n=164) |
---|
MMF Monotherapy | 4532.72 | 29960.41 | 54101.30 | 1829.87 | 10391.60 | 1254.29 | 19148.94 | 20748.32 | 0.0 | 1028.93 | 21227.11 | 12022.83 | 9418.65 | 397887.30 | 220.14 | 17512.31 |
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MPA Area Under the Concentration - Time Curve From Time 0 to 12 Hours (AUC0-12) (mcg/mL) by Visit
The AUC0-12 of MPA was estimated on the validated limited sampling strategy, AUC (milligrams multiplied by height over liter [mg.h/L]) = 7.182 + 4.607 multiplied by (*) concentration at 0 minutes (C0)+ 0.998 * the concentration at 40 minutes (C0.67) + 2.149 * the concentration at 120 minutes (C2). (NCT01292226)
Timeframe: Predose and 40 minutes and 2 hours postdose at Weeks 2, 4, 12, and 24, and at the Safety follow-up (Week 28)
Intervention | mcg*hr/mL (Mean) |
---|
| Week 2 (n=41) | Week 4 (n=42) | Week 12 (n=35) | Week 24 (n=35) | Follow-up (n=3) |
---|
MMF Monotherapy | 38.3 | 39.7 | 39.7 | 39.8 | 29.7 |
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Percentage of Participants With Gastrointestinal Toxicities
Gastrointestinal adverse events (AEs) according to WHO worst grade observed. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-up Visit)
Intervention | percentage of participants (Number) |
---|
| Abdominal pain (moderate) | Abdominal pain (severe) | Anal fissure (mild) | Diarrhoea (mild) | Diarrhoea (moderate) | Diarrhoea (severe) | Dyspepsia (mild) | Gastritis (moderate) | Gastritis erosive (moderate) | Gingival hyperplasia (mild) | Haemorrhoids (mild) | Intra-abdominal haematoma (mild) | Nausea (moderate) | Stomatitis (mild) | Vomiting (mild) | Vomiting (moderate) | Vomiting (severe) |
---|
MMF Monotherapy | 2.22 | 2.22 | 2.22 | 4.44 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 4.44 | 4.44 |
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Percentage of Participants With Hematologic Toxicity
Hematological toxicities graded according to WHO worst grade observed (Grade 1=mild, Grade 2=moderate). (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up)
Intervention | percentage of participants (Number) |
---|
| Grade 1 hemoglobin decreased | Grade 1 leukocytes decreased | Grade 2 leukocytes decreased | Grade 1 granulocytes decreased | Grade 2 granulocytes decreased | Grade 1 platelets decreased | Grade 1 bilirubin increased | Grade 2 bilirubin increased | Grade 1 hypoglycemia | Grade 1 alkaline phosphatase increased | Grade 2 alkaline phosphatase increased | Grade 1 aspartate aminotransferase increased | Grade 1 alanine aminotransferase increased | Grade 2 alanine aminotransferase increased | Grade 1 cholesterol increased | Grade 2 cholesterol increased | Grade 1 triglycerides increased | Grade 2 triglycerides increased | Grade 1 blood urea nitrogen increased | Grade 2 blood urea nitrogen increased |
---|
MMF Monotherapy | 13.33 | 11.11 | 8.89 | 6.67 | 6.67 | 6.67 | 4.44 | 2.22 | 4.44 | 13.33 | 2.22 | 6.67 | 8.89 | 2.22 | 26.67 | 8.89 | 24.44 | 2.22 | 2.22 | 2.22 |
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Percentage of Participants With Infection
Infections were graded according to the World Health Organization (WHO) worst grade observed. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up)
Intervention | percentage of participants (Number) |
---|
| Acarodermatitis (mild) | Bronchitis (moderate) | Cytomegalovirus (CMV) infection (mild) | CMV infection (moderate) | CMV infection (severe) | CMV viraemia (mild) | Gastroenteritis proteus (severe) | Gastrointestinal infection (severe) | Legionella infection (life-threatening) | Oral herpes (mild) | Sepsis (life-threatening) | Tracheitis (mild) | Urethritis (mild) | Urinary tract infection (mild) | Urinary tract infection (moderate) |
---|
MMF Monotherapy | 2.22 | 2.22 | 13.33 | 8.89 | 4.44 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 2.22 | 28.89 | 2.22 |
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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Gastrointestinal Toxicity
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| IMPDH I expression (n=334) | IMPDH II expression (n=351) |
---|
MMF Monotherapy | 0.082 | 0.030 |
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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Hematologic Toxicity
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| IMPDH I expression (n=334) | IMPDH II expression (n=351) |
---|
MMF Monotherapy | -0.116 | -0.004 |
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Spearman's Rank Correlation Coefficient Between IMPDH Expression and Risk of Infection
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| IMPDH I expression (n=334) | IMPDH II expression (n=351) |
---|
MMF Monotherapy | 0.045 | 0.047 |
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Spearman's Rank Correlation Coefficient Between IMPDH I Expression and Free Fraction
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| p Free fraction, T=0 (n=140) | p Free fraction, T=120 (n=143) |
---|
MMF Monotherapy | 0.047 | 0.080 |
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Spearman's Rank Correlation Coefficient Between IMPDH I Expression and MPA Levels
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA, T=0 (n=141) | Free MPA, T=120 (n=143) | Total MPA, T=0 (n=147) | Total MPA, T=120 (n=143) |
---|
MMF Monotherapy | 0.073 | -0.024 | 0.037 | -0.140 |
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Spearman's Rank Correlation Coefficient Between IMPDH II Expression and Free Fraction
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| p Free fraction, T=0 (n=144) | p Free fraction, T=120 (n=153) |
---|
MMF Monotherapy | -0.027 | 0.015 |
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Spearman's Rank Correlation Coefficient Between IMPDH II Expression and MPA Levels
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA, T=0 (n=145) | Free MPA, T=120 (n=153) | Total MPA, T=0 (n=152) | Total MPA, T=120 (n=153) |
---|
MMF Monotherapy | 0.007 | 0.073 | -0.001 | 0.084 |
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Spearman's Rank Correlation Coefficient Between IMPDH Inhibition and Risk of Acute Rejection
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| IMPDH I expression, T=0 (n=189) | IMPDH I expression T=120 (n=145) | IMPDH II expression, T=0 (n=196) | IMPDH II expression (T=120, n=155) | MPDH Activity, T=0 (n=198) | IMPDH Activity, T=120 (n=155) |
---|
MMF Monotherapy | 0.030 | 0.083 | -0.062 | -0.010 | -0.121 | -0.004 |
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Spearman's Rank Correlation Coefficient Between MPA Levels and IMPDH Activity
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA, T=0 (n=144) | Free MPA, T=120 (n=153) | Total MPA, T=0 (n=152) | Total MPA, T=120 (n=153) |
---|
MMF Monotherapy | 0.063 | 0.028 | 0.034 | -0.050 |
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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Gastrointestinal Toxicity
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA (n=300) | Total MPA (n=308) | AUC MPA (n=152) |
---|
MMF Monotherapy | 0.106 | 0.142 | 0.187 |
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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Hematologic Toxicity
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA (n=300) | Total MPA (n=308) | AUC MPA (n=152) |
---|
MMF Monotherapy | -0.004 | -0.037 | -0.038 |
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Spearman's Rank Correlation Coefficient Between MPA Levels and Risk of Infection
The Spearman's rank correlation coefficient was computed by ranking the data from 2 time points, 0 minutes and 120 minutes, and using the ranks in the Pearson product-moment correlation formula. In case of ties, the averaged ranks were used. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24
Intervention | correlation coefficient (Number) |
---|
| Free MPA (n=300) | Total MPA (n=308) | AUC MPA (n=152) |
---|
MMF Monotherapy | -0.020 | 0.063 | 0.030 |
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Total Mycophenolate Acid (MPA) by Visit and Timepoint
Drug quantification of total MPA (micrograms per milliliter [mcg/mL]) in the plasma was measured at time (T) = 0 minutes (min), 40 mins, and 120 mins. (NCT01292226)
Timeframe: Weeks 2, 4, 12, 24, and 28 (Safety Follow-Up Visit), and any unscheduled visits
Intervention | mcg/mL (Mean) |
---|
| T=0, Week 2 (n=41) | T=0, Week 4 (n=42) | T=0, Week 12 (n=35) | T=0, Week 24 (n=35) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, overall mean value (n=163) | T=40, Week 2 (n=43) | T=40, Week 4 (n=42) | T=40, Week 12 (n=35) | T=40, Week 24 (n=35) | T=40, safety follow-up (n=3) | T=40, unscheduled visit (n=7) | T=40, overall mean value (n=165) | T=120, Week 2 (n=41) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, safety follow-up (n=3) | T=120, Week 24 (n=35) | T=120, unscheduled visit (n=7) | T=120, overall mean value (n=163) |
---|
MMF Monotherapy | 1.75 | 1.87 | 1.79 | 1.90 | 1.16 | 1.32 | 1.79 | 6.44 | 6.96 | 7.23 | 6.47 | 5.71 | 4.06 | 6.63 | 8.86 | 8.95 | 8.57 | 4.91 | 10.04 | 7.03 | 8.92 |
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Tumor Necrosis Factor (TNF) Expression by Visit and Timepoint
TNF gene expression was measured by QRT-PCR based cytokine measurement of PBMCs at 2 timepoints per visit, 0 and 120 minutes and expressed as number of mRNA copies/cell. (NCT01292226)
Timeframe: BL and Weeks 2, 4, 12, and 24, and safety follow-up (Week 28) and any unscheduled visits
Intervention | number of mRNA copies/cell (Mean) |
---|
| T=0, BL (n=44) | T=0, Week 2 (n=41) | T=0, Week 4 (n=42) | T=0, Week 12 (n=34) | T=0, Week 24 (n=34) | T=0, safety follow-up (n=3) | T=0, unscheduled visit (n=7) | T=0, mean value (n=205) | T=120, BL (n=0) | T=120, Week 2 (n=43) | T=120, Week 4 (n=42) | T=120, Week 12 (n=35) | T=120, Week 24 (n=34) | T=120, safety follow-up (n=3) | T=120, unscheduled visit (n=7) | T=120, mean value (n=164) |
---|
MMF Monotherapy | 4532.72 | 29960.41 | 54101.30 | 1829.87 | 10391.60 | 1254.29 | 19148.94 | 20748.32 | 0 | 1028.93 | 21227.11 | 12022.83 | 9418.65 | 397887.30 | 220.14 | 17512.31 |
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AUC0-12 of Free MPA at Day 20
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | hours*mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 48.530 |
MMF - COPD, Emphysema, IPF, or A1AD | 89.315 |
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AUC0-12 of Free MPA at Day 4
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours times micrograms per liter (hours*[mcg/L]). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | hours*mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 135.703 |
MMF - COPD, Emphysema, IPF, or A1AD | 80.272 |
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AUC0-12 of Free MPA at Day 8
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | hours*mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 61.155 |
MMF - COPD, Emphysema, IPF, or A1AD | 83.683 |
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AUC0-12 of Free MPA at Day 90
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | hours*mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 68.874 |
MMF - COPD, Emphysema, IPF, or A1AD | 99.212 |
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Cmax of Free MPA at Day 20
Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 51.822 |
MMF - COPD, Emphysema, IPF, or A1AD | 100.094 |
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Cmax of Free MPA at Day 4
Cmax was expressed in micrograms per liter (mcg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 135.275 |
MMF - COPD, Emphysema, IPF, or A1AD | 84.014 |
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Cmax of Free MPA at Day 8
Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 59.571 |
MMF - COPD, Emphysema, IPF, or A1AD | 95.553 |
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Cmax of Free MPA at Day 90
Cmax was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 87.293 |
MMF - COPD, Emphysema, IPF, or A1AD | 101.472 |
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Cmin of Free MPA at Day 20
Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 20 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 12.193 |
MMF - COPD, Emphysema, IPF, or A1AD | 20.237 |
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Cmin of Free MPA at Day 4
Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 4 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 40.188 |
MMF - COPD, Emphysema, IPF, or A1AD | 23.711 |
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Cmin of Free MPA at Day 8
Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 8 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 18.161 |
MMF - COPD, Emphysema, IPF, or A1AD | 15.669 |
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Cmin of Free MPA at Day 90
Cmin was expressed in mcg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 90 post-transplantation
Intervention | mcg/L (Mean) |
---|
MMF - Cystic Fibrosis | 8.102 |
MMF - COPD, Emphysema, IPF, or A1AD | 18.831 |
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Forced Expiratory Volume in 1 Second (FEV1) at Day 90 Post-Transplantation
FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. (NCT01014442)
Timeframe: Day 90 post-transplantation
Intervention | L (Mean) |
---|
MMF - Cystic Fibrosis | 3.087 |
MMF - COPD, Emphysema, IPF, or A1AD | 2.834 |
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Forced Vital Capacity (FVC) at Day 90 Post-Transplantation
FVC at Day 90 post-transplantation is reported. (NCT01014442)
Timeframe: Day 90 post-transplantation
Intervention | L (Mean) |
---|
MMF - Cystic Fibrosis | 3.445 |
MMF - COPD, Emphysema, IPF, or A1AD | 3.619 |
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Free Fraction of Free MPA at Day 20
MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | percentage of free fraction (Mean) |
---|
MMF - Cystic Fibrosis | 0.2922 |
MMF - COPD, Emphysema, IPF, or A1AD | 0.3052 |
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Free Fraction of Free MPA at Day 4
MPA Free fraction (in percent [%]) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | percentage of free fraction (Mean) |
---|
MMF - Cystic Fibrosis | 0.5789 |
MMF - COPD, Emphysema, IPF, or A1AD | 0.3208 |
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Free Fraction of Free MPA at Day 8
MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | percentage of free fraction (Mean) |
---|
MMF - Cystic Fibrosis | 0.3595 |
MMF - COPD, Emphysema, IPF, or A1AD | 0.3594 |
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Free Fraction of Free MPA at Day 90
MPA Free fraction (in %) was calculated by dividing free MPA AUC0-12 by total MPA AUC0-12 times 100%. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | percentage of free fraction (Mean) |
---|
MMF - Cystic Fibrosis | 0.2270 |
MMF - COPD, Emphysema, IPF, or A1AD | 0.3020 |
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Percent of Predicted FEV1 at Day 90 Post-Transplantation
FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Percent predicted FEV1 [%] = (FEV1 [L] / Predicted normal value FEV1 [L]) * 100% (NCT01014442)
Timeframe: Day 90 post-transplantation
Intervention | percentage of predicted FEV1 (Mean) |
---|
MMF - Cystic Fibrosis | 81.75 |
MMF - COPD, Emphysema, IPF, or A1AD | 90.17 |
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Percentage of Participants With Opportunistic Infections
Opportunistic infections included all infections which occurred due to aspergillus, candida, pneumocystis, cryptococcus, listeria, herpes zoster, herpes simplex, cytomegalovirus pathogens. (NCT01014442)
Timeframe: Up to Day 90
Intervention | percentage of participants (Number) |
---|
MMF - Cystic Fibrosis | 18.2 |
MMF - COPD, Emphysema, IPF, or A1AD | 20.0 |
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Area Under the Curve From Time 0 to 12 Hours (AUC0-12) of MPA, MPAG and AcMPAG at Day 4
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours time milligrams per liter (hours*[mg/L]). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | hours*(mg/L) (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 27.174 | 829.14 | 13.672 |
,MMF - Cystic Fibrosis | 23.460 | 1095.98 | 10.414 |
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AUC0-12 of MPA, MPAG and AcMPAG at Day 20
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | hours*(mg/L) (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 28.290 | 1095.37 | 9.482 |
,MMF - Cystic Fibrosis | 18.925 | 812.65 | 3.252 |
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AUC0-12 of MPA, MPAG and AcMPAG at Day 8
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | hours*(mg/L) (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 23.352 | 888.96 | 9.081 |
,MMF - Cystic Fibrosis | 17.841 | 881.50 | 6.064 |
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AUC0-12 of MPA, MPAG and AcMPAG at Day 90
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours and expressed in hours*(mg/L). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | hours*(mg/L) (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 31.864 | 972.69 | 7.219 |
,MMF - Cystic Fibrosis | 32.365 | 776.28 | 7.776 |
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Change From Baseline in Intracellular Adenosine-Tri-Phosphate (iATP) Levels
iATP was expressed in ng/mL. (NCT01014442)
Timeframe: Baseline, Days 4, 8, 20 and 90 post-transplantation
Intervention | ng/mL (Mean) |
---|
| Day 4 | Day 8 | Day 20 | Day 90 |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 85.4 | 144.9 | -45.9 | -114.7 |
,MMF - Cystic Fibrosis | 24.0 | 162.9 | -42.4 | -164.6 |
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Change From Baseline in T-Cell Phenotype
Reported values are change in the T-cell phenotype status from baseline to Day 20 and 90 for cluster of differentiation (CD) 3, CD19, CD4, CD4CD25, CD28, CD45RA, CD45RO, CD69, CD127, and CD152. (NCT01014442)
Timeframe: Baseline, Days 20 and 90 post-transplantation
Intervention | percentage of lymphocytes (Mean) |
---|
| CD3: Change at Day 20 | CD3: Change at Day 90 | CD19: Change at Day 20 | CD19: Change at Day 90 | CD4: Change at Day 20 | CD4: Change at Day 90 | CD4CD25: Change at Day 20 | CD4CD25: Change at Day 90 | CD28: Change at Day 20 | CD28: Change at Day 90 | CD45RA: Change at Day 20 | CD45RA: Change at Day 90 | CD45RO: Change at Day 20 | CD45RO: Change at Day 90 | CD69: Change at Day 20 | CD69: Change at Day 90 | CD127: Change at Day 20 | CD127: Change at Day 90 | CD152: Change at Day 20 | CD152: Change at Day 90 |
---|
MMF - COPD, Emphysema, IPF, or A1AD | -3.01 | -2.81 | 1.88 | 0.61 | -14.71 | -1.93 | -0.53 | -0.10 | -4.44 | -1.04 | -5.11 | -4.88 | -2.83 | -0.43 | 0.01 | -0.04 | -3.90 | -2.03 | -10.97 | -15.59 |
,MMF - Cystic Fibrosis | -1.34 | 1.74 | 3.29 | 0.59 | 13.29 | 9.00 | 2.72 | 0.89 | 4.54 | 10.15 | 5.35 | -1.57 | -0.08 | -1.92 | 5.68 | -0.46 | -1.36 | -0.19 | -3.78 | -0.28 |
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CL of MPA, MPAG and AcMPAG at Day 20
CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | L/hour (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 58.295 | 1.4788 | 232.34 |
,MMF - Cystic Fibrosis | 83.068 | 1.9338 | 680.68 |
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CL of MPA, MPAG and AcMPAG at Day 8
CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | L/hour (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 81.578 | 1.7847 | 253.81 |
,MMF - Cystic Fibrosis | 90.921 | 2.3235 | 410.86 |
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CL of MPA, MPAG and AcMPAG at Day 90
CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in L/hour. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | L/hour (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 36.922 | 1.2433 | 223.56 |
,MMF - Cystic Fibrosis | 38.091 | 1.5701 | 231.28 |
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Clearance (CL) of MPA, MPAG and AcMPAG at Day 4
CL is a quantitative measure of the rate at which a drug substance is removed from the body and expressed in liters per hour (L/hour). (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | L/hours (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 56.709 | 1.7615 | 140.96 |
,MMF - Cystic Fibrosis | 68.471 | 1.6754 | 206.60 |
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Cmax of MPA, MPAG and AcMPAG at Day 20
Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 6.762 | 124.450 | 1.434 |
,MMF - Cystic Fibrosis | 5.284 | 93.882 | 0.624 |
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Cmax of MPA, MPAG and AcMPAG at Day 8
Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 6.106 | 100.128 | 1.361 |
,MMF - Cystic Fibrosis | 5.124 | 100.127 | 1.007 |
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Cmax of MPA, MPAG and AcMPAG at Day 90
Cmax was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 7.294 | 113.453 | 1.163 |
,MMF - Cystic Fibrosis | 12.915 | 104.938 | 1.375 |
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Cmin of MPA, MPAG and AcMPAG at Day 20
Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 20 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 1.234 | 69.023 | 0.528 |
,MMF - Cystic Fibrosis | 0.782 | 58.484 | 0.261 |
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Cmin of MPA, MPAG and AcMPAG at Day 8
Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 8 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.772 | 57.189 | 0.517 |
,MMF - Cystic Fibrosis | 0.750 | 54.566 | 0.386 |
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Cmin of MPA, MPAG and AcMPAG at Day 90
Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 90 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 1.570 | 80.488 | 0.466 |
,MMF - Cystic Fibrosis | 1.103 | 57.957 | 0.449 |
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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 20
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | hours/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.01923 | 0.7440 | 0.00642 | 0.0000597 |
,MMF - Cystic Fibrosis | 0.01402 | 0.6059 | 0.00239 | 0.0000399 |
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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 4
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | hours/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.02016 | 0.6165 | 0.01056 | 0.0000594 |
,MMF - Cystic Fibrosis | 0.01652 | 0.7720 | 0.00739 | 0.0000943 |
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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 8
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | hours/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.01624 | 0.6270 | 0.00644 | 0.0000580 |
,MMF - Cystic Fibrosis | 0.01256 | 0.6157 | 0.00426 | 0.0000428 |
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Dose-Normalized AUC0-12 of MPA, MPAG, AcMPAG and Free MPA at Day 90
AUC0-12 is a measure of the serum concentration of the drug from time 0 to 12 hours. Dose-normalized AUC0-12 was determined (in hours/L) by dividing the AUC0-12 by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | hours/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.03172 | 0.9190 | 0.00700 | 0.00001023 |
,MMF - Cystic Fibrosis | 0.03126 | 0.7887 | 0.00748 | 0.0000752 |
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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 20
Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | 1/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MP |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.00456 | 0.08430 | 0.000969 | 0.0000666 |
,MMF - Cystic Fibrosis | 0.00395 | 0.07044 | 0.000482 | 0.0000439 |
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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 4
Dose-normalized Cmax was determined (in 1 per liter [1/L]) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | 1/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.00422 | 0.06749 | 0.001388 | 0.0000625 |
,MMF - Cystic Fibrosis | 0.00557 | 0.08286 | 0.001122 | 0.0000929 |
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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 8
Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | 1/L (Mean) |
---|
| MPA | MPAG | AcMPA | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.00435 | 0.07070 | 0.000967 | 0.0000663 |
,MMF - Cystic Fibrosis | 0.00356 | 0.06980 | 0.000702 | 0.0000409 |
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Dose-Normalized Cmax of MPA, MPAG, AcMPAG and Free MPA at Day 90
Dose-normalized Cmax was determined (in 1/L) by dividing the Cmax by the actual dose taken. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | 1/L (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 0.00742 | 0.10948 | 0.001134 | 0.0001043 |
,MMF - Cystic Fibrosis | 0.01261 | 0.10671 | 0.001377 | 0.0000945 |
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Minimum Concentration (Cmin) of MPA, MPAG and AcMPAG at Day 4
Cmin was expressed in mg/L. (NCT01014442)
Timeframe: Predose (0 hour) on Day 4 post-transplantation
Intervention | mg/L (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 1.094 | 47.493 | 0.733 |
,MMF - Cystic Fibrosis | 1.267 | 66.917 | 0.602 |
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Time to Maximum Concentration (Tmax) of MPA, MPAG, AcMPAG and Free MPA at Day 4
(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | hour (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 2.892 | 4.891 | 3.405 | 1.405 |
,MMF - Cystic Fibrosis | 1.823 | 3.713 | 2.379 | 1.472 |
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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 20
(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | hour (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 2.463 | 4.907 | 3.411 | 1.366 |
,MMF - Cystic Fibrosis | 2.293 | 3.762 | 2.842 | 1.236 |
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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 8
(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | hour (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 1.659 | 3.647 | 2.546 | 1.446 |
,MMF - Cystic Fibrosis | 2.138 | 3.669 | 2.802 | 1.272 |
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Tmax of MPA, MPAG, AcMPAG and Free MPA at Day 90
(NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | hour (Mean) |
---|
| MPA | MPAG | AcMPAG | Free MPA |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 2.143 | 4.420 | 3.564 | 1.485 |
,MMF - Cystic Fibrosis | 1.450 | 3.095 | 2.234 | 1.490 |
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Volume of Distribution (Vz) of MPA, MPAG and AcMPAG at Day 4
Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 4 post-transplantation
Intervention | Liter (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 287.74 | 12.062 | 705.38 |
,MMF - Cystic Fibrosis | 345.46 | 11.681 | 1065.02 |
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Vz of MPA, MPAG and AcMPAG at Day 20
Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 20 post-transplantation
Intervention | Liter (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 257.57 | 9.533 | 1571.93 |
,MMF - Cystic Fibrosis | 476.06 | 12.798 | 3006.18 |
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Vz of MPA, MPAG and AcMPAG at Day 8
Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 8 post-transplantation
Intervention | Liter (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 331.51 | 12.740 | 1173.38 |
,MMF - Cystic Fibrosis | 465.45 | 12.168 | 1432.88 |
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Vz of MPA, MPAG and AcMPAG at Day 90
Vz is defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration of a drug. (NCT01014442)
Timeframe: Predose (0 hour), 0.5, 1, 1.5, 2, 4, 8, 10, and 12 hours post-dose on Day 90 post-transplantation
Intervention | Liter (Mean) |
---|
| MPA | MPAG | AcMPAG |
---|
MMF - COPD, Emphysema, IPF, or A1AD | 192.53 | 9.201 | 980.49 |
,MMF - Cystic Fibrosis | 139.48 | 9.254 | 811.15 |
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Percentage of Participants -Overall Participant and Graft Survival
This measure looks at the participants who did not die and/or did not receive a subsequent heart transplant. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 93.8 |
Cohort B: Sensitized, Crossmatch Positive | 93.8 |
Cohort B: Sensitized, Crossmatch Negative | 87.4 |
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Percentage of Participants Experiencing Acute Rejection
Acute rejection is defined as any one of the following types of rejection: acute cellular rejection (International Society for Heart and Lung Transplant (ISHLT)) system for grading rejection grade 2R or greater), acute refractory cellular rejection (acute cellular rejection unresponsive to two sequential courses of corticosteroids), acute antibody mediated rejection (histological evidence of unequivocal acute capillary injury, with complement deposition and margination of macrophages with or without neutrophils), acute mixed rejection (evidence of acute antibody mediated rejection with ISHLT grade 1R or greater), or acute clinical rejection (clinically-based acute rejection, no matter the ISHLT grade, leading to an acute augmentation of immunosuppression). (NCT01005316)
Timeframe: Transplantation to the end of study.
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 27.8 |
Cohort B: Sensitized, Crossmatch Positive | 75.0 |
Cohort B: Sensitized, Crossmatch Negative | 49.6 |
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Percentage of Participants Positive for de Novo Donor-Specific Alloantibody Production in the First Year Post-Transplantation
A de novo donor-specific alloantibody (DSA) is a newly developed alloantibody that is against the donor organ. This measure includes all de novo DSA (≥1000 MFI) regardless of is persistence or timing within the first year post-transplant. Alloantibodies are important mediators of acute and chronic rejection. (NCT01005316)
Timeframe: Transplantation to first year post transplant (up to 12 months post transplant).
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 22.7 |
Cohort B: Sensitized, Crossmatch Positive | 50.0 |
Cohort B: Sensitized, Crossmatch Negative | 37.8 |
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Percentage of Participants Positive for Event of Death, Graft Loss or Rejection With Hemodynamic Compromise at 12 Months Post-Transplantation
"This is a composite outcome of death, graft loss or rejection with hemodynamic compromise.~Rejection was considered to be with hemodynamic compromise if the rejection event had new onset echocardiographically measured from fractional shortening <26% with ≥5% fall from last echocardiogram or the rejection event had new onset of heart failure." (NCT01005316)
Timeframe: 12 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 5.2 |
Cohort B: Sensitized, Crossmatch Positive | 12.5 |
Cohort B: Sensitized, Crossmatch Negative | 11.8 |
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Percentage of Participants Positive for Severe Infection(s)
Severe infections are defined as a clinical illness considered likely infectious in origin that leads to hospitalization. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 34.0 |
Cohort B: Sensitized, Crossmatch Positive | 43.8 |
Cohort B: Sensitized, Crossmatch Negative | 30.7 |
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Percentage of Participants With Occurrence of Re-Hospitalization(s)
Hospitalization is defined as any hospitalization lasting greater than 24 hours. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 66.0 |
Cohort B: Sensitized, Crossmatch Positive | 75.0 |
Cohort B: Sensitized, Crossmatch Negative | 62.2 |
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Percentage of Participants With the Presence of Anti-HLA IgG Antibodies by Luminex SA Testing
Luminex SA testing was used to detect the presence of anti-HLA IgG Antibodies for all samples at a central laboratory. (NCT01005316)
Timeframe: Pre-transplantation
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 22.7 |
Cohort B: Sensitized, Crossmatch Positive | 81.3 |
Cohort B: Sensitized, Crossmatch Negative | 68.5 |
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Percentage of Participants With the Presence of Anti-MICA Antibodies by Luminex TM Assay
Major histocompatibility complex class I chain-related gene A (MICA) is an antigen that is a potential marker of rejection. Luminex TM assay was used to detect its presence. (NCT01005316)
Timeframe: Pre-Transplantation
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 8.2 |
Cohort B: Sensitized, Crossmatch Positive | 18.8 |
Cohort B: Sensitized, Crossmatch Negative | 11.0 |
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Percentage of Participants- Mortality While on Transplantation Wait-List
Death that occurred while on the transplantation wait-list, and thus before receiving a heart transplant. (NCT01005316)
Timeframe: Pre-transplantation
Intervention | percentage of participants (Number) |
---|
Enrolled, Not Transplanted | 39.2 |
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Presence of C4d on Endomyocardial Biopsy (EMB)
The biopsy of the heart stained positive for the presence of C4d, a potential marker of rejection. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | percentage of participants (Number) |
---|
Cohort A: Non-Sensitized | 18.6 |
Cohort B: Sensitized, Crossmatch Positive | 62.5 |
Cohort B: Sensitized, Crossmatch Negative | 34.6 |
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Time From Participant Listing on Organ Wait-List to Receiving Organ Transplant, Death or De-Listing
Time (in days) from listing on the organ wait-list to receiving an organ transplant, death or de-listing. This measure is calculated as time from listing on the organ wait-list until the earliest time among transplantation, death and de-listing. (NCT01005316)
Timeframe: Study enrollment to transplantation
Intervention | Days (Mean) |
---|
Enrolled | 128.3 |
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Time to Acute Rejection
Time (in days) to acute rejection. Acute rejection is defined as any one of the following types of rejection: acute cellular rejection (International Society for Heart and Lung Transplant (ISHLT) system for grading rejection grade 2R or greater), acute refractory cellular rejection (acute cellular rejection unresponsive to two sequential courses of corticosteroids), acute antibody mediated rejection (histological evidence of unequivocal acute capillary injury, with complement deposition and margination of macrophages with or without neutrophils), acute mixed rejection (evidence of acute antibody mediated rejection with ISHLT grade 1R or greater), or acute clinical rejection (clinically-based acute rejection, no matter the ISHLT grade, leading to an acute augmentation of immunosuppression). Time to acute rejection is time from transplantation to first acute rejection date. (NCT01005316)
Timeframe: Transplantation to the end of study.
Intervention | Days (Mean) |
---|
Cohort A: Non-Sensitized | 151.0 |
Cohort B: Sensitized, Crossmatch Positive | 74.5 |
Cohort B: Sensitized, Crossmatch Negative | 124.7 |
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Time to Diagnosis of Chronic Rejection
Time (in days) to the diagnosis of chronic rejection. Chronic rejection is defined as stenosis, irregularity, or ectasia of the epicardial vessels, or severe peripheral pruning of the distal coronary artery tree. Time to diagnosis is time from transplantation until the first diagnosis of chronic rejection. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | Days (Mean) |
---|
Cohort A: Non-Sensitized | 606.8 |
Cohort B: Sensitized, Crossmatch Positive | NA |
Cohort B: Sensitized, Crossmatch Negative | 398.9 |
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Time to New-Onset Diabetes Mellitus
Time (in days) to new-onset diabetes mellitus. New-onset diabetes mellitus is defined as the new onset of insulin dependency or the need for oral hypoglycemic agents lasting more than 30 days post-transplant. Time to new-onset diabetes is time from transplantation until the diagnosis of new-onset diabetes. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | Days (Mean) |
---|
Cohort A: Non-Sensitized | 73 |
Cohort B: Sensitized, Crossmatch Positive | 48 |
Cohort B: Sensitized, Crossmatch Negative | 283.4 |
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Time to Post-Transplantation Lymphoproliferative Disorder
Time (in days) post-transplant lymphoproliferative disorder (PTLD). PTLD is defined as histopathological evidence of lymphoid proliferation (nodal or extranodal) fulfilling the criteria of the revised classification of the WHO 2008 (Swerdlow 2008). Time to PTLD is time from transplantation until the diagnosis of PTLD. (NCT01005316)
Timeframe: Transplantation to the end of study (up to 4 years post transplant).
Intervention | Days (Mean) |
---|
Cohort A: Non-Sensitized | 910 |
Cohort B: Sensitized, Crossmatch Positive | NA |
Cohort B: Sensitized, Crossmatch Negative | 118.7 |
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Time to Production of Post-Transplant de Novo Donor-specific Alloantibodies
Time (in days) from transplant to development of de novo donor-specific alloantibodies (DSA). This measure is calculated as time from transplant until the earliest time of development of any de novo DSA. The DSA is a newly developed alloantibody that is against the donor organ. Alloantibodies are important mediators of acute and chronic rejection. (NCT01005316)
Timeframe: Transplantation to first year post transplant (up to 12 months post transplant).
Intervention | Days (Mean) |
---|
Cohort A: Non-Sensitized | 28.1 |
Cohort B: Sensitized, Crossmatch Positive | 15.4 |
Cohort B: Sensitized, Crossmatch Negative | 55.1 |
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Percentage of Participants -Quantification of Anti-HLA IgG Antibodies by Luminex SA Testing
Quantification of anti-HLA IgG antibodies is measured in mean fluorescence intensity (MFI). The maximum MFI for the given subject is provided. (NCT01005316)
Timeframe: Pre-transplantation
Intervention | percentage of participants (Number) |
---|
| Missing | None | MFI 1000-3999 | MFI 4000-7999 | MFI ≥8000 |
---|
Cohort A: Non-Sensitized | 0 | 77.3 | 20.6 | 1.0 | 1.0 |
,Cohort B: Sensitized, Crossmatch Negative | 2.4 | 29.1 | 29.9 | 14.2 | 24.4 |
,Cohort B: Sensitized, Crossmatch Positive | 0 | 18.8 | 6.3 | 12.5 | 62.5 |
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Hazard Cox's Model Analysis of Pericardial/Pleural Effusions
Pericardial effusions: any pericardial effusion defined as at least moderate (i.e. measuring at least 2.0 cm in diastole, in the point of largest distance between the pericardial leaflets), with or without signs of hemodynamic compromise, or leading to drainage or to prolonged hospitalization. Pleural effusions: need for surgical drainage tubes for longer than 7 days after surgery and subsequent pleural effusions leading to drainage. CI = confidence interval, HR = hazard ratio, MDRD = Modification of Diet in Renal Disease (NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Immediate Introduction of Everolimus | 30 |
Delayed Introduction of Everolimus | 18 |
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Participants With at Least One Occurrence of Composite Treatment Failure Events
Comparison of 6-months cumulative incidence of composite treatment failure events (BPAR ≥ 2R, rejection with hemodynamic compromise, graft loss, or death) between delayed everolimus arm and immediate everolimus arm (NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Immediate Introduction of Everolimus | 33 |
Delayed Introduction of Everolimus | 26 |
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Participants With at Least One Occurrence of Safety Composite Endpoint After 6 Months by Treatment Group
Comparison of 6-month cumulative incidence of safety composite endpoint (wound healing delay) related to initial transplant surgery, pleural/pericardial effusions and occurrence of acute renal insufficiency, defined as estimated glomerular filtration rate (eGFR) ≤ 30 mL/min/1.73 m2, between delayed everolimus arm and immediate everolimus arm (NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Immediate Introduction of Everolimus | 40 |
Delayed Introduction of Everolimus | 30 |
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Absolute and Percent Frequencies of Patients With LDL ≥ 100 mg/mL at 1, 3 and 6 Months, by Treatment Group
LDL = low density lipoprotein (NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
| Month 1 | Month 3 | Month 6 |
---|
Delayed Introduction of Everolimus | 38 | 37 | 36 |
,Immediate Introduction of Everolimus | 41 | 37 | 34 |
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Partcipants With at Least One Occurrence of Each Safety Composite Endpoint Event After 6 Months by Treatment Group
(NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
| Wound healing complication | Pleural effusion | Pericardial effusion | eGFR ≤ 30 mL/min/1.73 m2 |
---|
Delayed Introduction of Everolimus | 8 | 1 | 18 | 8 |
,Immediate Introduction of Everolimus | 10 | 1 | 30 | 7 |
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Participants With CMV Infection and CMV Syndrome/Disease After 6 Months by Treatment Group
CMV infection is defined as pp65 antigenemia or DNAemia (NCT01017029)
Timeframe: 6 months
Intervention | participants (Number) |
---|
| CMV infections | CMV syndrome/disease |
---|
Delayed Introduction of Everolimus | 63 | 6 |
,Immediate Introduction of Everolimus | 46 | 3 |
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Chronic Allograft Damage Index (CADI) Score at Month 12 After Transplantation
CADI scoring was defined for 6 histological categories: interstitial inflammatory cell infiltration (0 equals (=) no or mild inflammation, 1=approximately (~)25 percent (%) cell infiltration, 2=26-50% cell infiltration, and 3=greater than (>)50% cell infiltration); interstitial fibrosis (0=none, 1=~25% interstitial affected, 2=26-50% interstitial affected, and 3=>50% interstitial affected); tubular atrophy (0=none, 1=~15% proximal tubular atrophy [PTA], 2=16-30% PTA, and 3=>30% PTA); mesangial matrix proliferation (MMP; 0=none, 1=25% non-glomerulosclerosis [NGS] combined with moderate MMP, 2=25-50% NGS combined with MMP, and 3=>50% NGS combined with MMP); glomerular sclerosis (0=none, 1=~15% glomerulus affected, 2=16-50% glomerulus affected, and 3=>50% glomerulus affected); endothelial proliferation (EP; 0=none, 1=EP to less than (<)25% remaining artery/small artery membrane [RA/SAM], 2=EP to 26-50% [RA/SAM], and 3=>50% [RA/SAM]). CADI score was the sum of the 6 histological findings. (NCT00758602)
Timeframe: Month 12
Intervention | score on a scale (Mean) |
---|
MMF, Standard Dose Tacrolimus | 1.82 |
MMF, Low Dose Tacrolimus | 2.13 |
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Glomerular Filtration Rate (GFR) at Month 12 After Transplantation
GFR was determined using the Cockcroft-Gault formula to calculate the creatinine clearance, at Month 12 after renal transplantation. For males, creatinine clearance [milliliters per minute (mL/min)] = [(140 minus age) multiplied by (*) (body weight in kg) divided by [72 * serum creatinine mg per deciliter (mg/dL)]. For females, creatinine clearance (mL/min) = 0.85 * [(140 minus age) * (body weight in kg)] divided by [72 * serum creatinine (mg/dL)]. (NCT00758602)
Timeframe: Month 12
Intervention | mL/min (Mean) |
---|
MMF, Standard Dose Tacrolimus | 77.08 |
MMF, Low Dose Tacrolimus | 80.12 |
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Percentage of Participants With Treatment Failure at 12 Months Post-Transplant
Treatment failure was defined by the occurrence of any of the following: use of additional maintenance immunosuppressive medication not specified in the assigned treatment group; discontinuation of any of the assigned immunosuppressants for more than 14 consecutive days or 30 cumulative days; graft loss or return to chronic dialysis; or death. (NCT00758602)
Timeframe: Month 12
Intervention | percentage of participants (Number) |
---|
MMF, Standard Dose Tacrolimus | 9.6 |
MMF, Low Dose Tacrolimus | 6.6 |
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Time to First Acute Rejection Post-Transplant
The median time, in days, between randomization and acute rejection. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52
Intervention | days (Median) |
---|
MMF, Standard Dose Tacrolimus | 40 |
MMF, Low Dose Tacrolimus | 18 |
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Time to First Acute Rejection Post-Transplant - Number of Participants With an Event
(NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52
Intervention | participants (Number) |
---|
MMF, Standard Dose Tacrolimus | 2 |
MMF, Low Dose Tacrolimus | 4 |
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Glomerular Filtration Rate (GFR) (mL/Min)
The mean GFR values in mL/min at BL, Weeks 2, 4, 13, 26, 39, and 52. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52
Intervention | mL/min (Mean) |
---|
| Baseline (n=95,96) | Week 2 (n=101,102) | Week 4 (n=99,98) | Week 13 (n=88,91) | Week 26 (n=84,83) | Week 39 (n=83,77) | Week 52 (n=86,82) |
---|
MMF, Low Dose Tacrolimus | 15.41 | 71.02 | 72.87 | 75.71 | 76.77 | 78.00 | 80.12 |
,MMF, Standard Dose Tacrolimus | 14.37 | 67.95 | 71.77 | 78.13 | 75.92 | 77.34 | 77.08 |
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Participant and Graft Survival
The percentage of participants surviving with grafts intact at 6 and 12 months after renal transplant. (NCT00758602)
Timeframe: Months 6 and 12
Intervention | percentage of participants (Number) |
---|
| 6 months post-transplant | 12 months post-transplant |
---|
MMF, Low Dose Tacrolimus | 100.0 | 99.1 |
,MMF, Standard Dose Tacrolimus | 100.0 | 100.0 |
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Percentage of Participants Experiencing Acute Rejection, Graft Loss, or Death at 6 and 12 Months Post-Transplant
(NCT00758602)
Timeframe: Months 6 and 12
Intervention | percentage of participants (Number) |
---|
| Acute rejection, 6 months post-transplant | Acute rejection, 12 months post-transplant | Graft loss, 6 months post-transplant | Graft loss, 12 months post-transplant | Death, 6 months post-transplant | Death, 12 months post-transplant |
---|
MMF, Low Dose Tacrolimus | 5.2 | 5.2 | 0.0 | 0.0 | 0.0 | 0.9 |
,MMF, Standard Dose Tacrolimus | 2.6 | 2.6 | 0.0 | 0.0 | 0.0 | 0.0 |
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Serum Creatinine (Micromoles Per Liter [µmol/L])
The mean serum creatinine values in µmol/L at Baseline (BL), Weeks 2, 4, 13, 26, 39, and 52. (NCT00758602)
Timeframe: BL, Weeks 2, 4, 13, 26, 39, and 52
Intervention | µmol/L (Mean) |
---|
| Baseline (n=95,96) | Week 2 (n=101,102) | Week 4 (n=99,98) | Week 13 (n=88,91) | Week 26 (n=84,83) | Week 39 (n=83,77) | Week 52 (n=86,82) |
---|
MMF, Standard Dose Tacrolimus | 662.93 | 154.57 | 118.47 | 97.52 | 105.70 | 102.22 | 103.10 |
,MMFl, Low Dose Tacrolimus | 639.70 | 115.71 | 107.08 | 100.10 | 97.51 | 95.16 | 94.19 |
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Average Daily Doses (mg) of Enteric-coated Mycophenolate Acid (MPA) and Mycophenolate Mofetil (MMF) by Treatment Duration Intervals
The average daily doses of enteric-coated mycophenolate acid (MPA) and mycophenolate mofetil (MMF) at baseline and during the last 2 weeks of treatment. 1000 mg MMF = 720 mg enteric-coated MPA (MPA equivalent dose). (NCT00658333)
Timeframe: Baseline and week 4 to week 6
Intervention | mg (Mean) |
---|
| MMF Baseline Dose (n=15, 15) | Week 4 to Week 6 (n=13, 14) |
---|
Enteric-coated Mycophenolate Acid | 1033.30 | 914.5 |
,Mycophenolate Mofetil | 1216.7 | 1567.6 |
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Number of Participants With Response (Yes/no)
The primary variable was the response (yes/no) with positive response being defined as an increase of 360 mg/day enteric-coated mycophenolate acid (MPA)from the baseline daily dose, tolerated and maintained for a 4 week duration until the end of the study (Week 6). Tolerability was defined as the overall assessment of improvement or no change in the intensity of physician assessed gastrointestinal (GI) symptoms at end of study as reported on the physician administered evaluation of GI symptomatology. (NCT00658333)
Timeframe: 6 weeks
Intervention | Participants (Number) |
---|
| Yes | No |
---|
Enteric-coated Mycophenolate Acid | 10 | 5 |
,Mycophenolate Mofetil | 9 | 6 |
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Percentage of Participants Who Achieved Overall Response
Overall response rate (ORR) equals (=) CRR + PRR. CRR was defined as: 1. Normal serum creatinine (and with no more than a 25% increase from baseline) 2. Improvement in urinary protein:urinary creatinine ratio to ≤0.5. PRR was defined as at least 50 % reduction in proteinuria from Baseline, without more than 25% increase of serum creatinine at Week 48, compared with Baseline. If Baseline urine protein:urine creatinine ratio is >3, a urine protein:urine creatinine ratio of <3 needs to be achieved. (NCT00626197)
Timeframe: Week 48
Intervention | Percentage of Participants (Number) |
---|
OCR 400 mg + SOC | 66.7 |
OCR 1000 mg + SOC | 67.1 |
Placebo + SOC | 54.7 |
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Mean Absolute Counts of Cluster of Differentiation (CD) 19 Positive (+) Cells Per Visit
CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. CD19+ cells were measured as cells per microliter (cells/uL). (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32
Intervention | Cells/uL (Mean) |
---|
| Baseline | Day 15 | Week 4 | Week 16 | Week 32 | Week 48 | Day 1 Pre-infusion | Day 1 Post-infusion | Day 15 Pre-infusion | Day 15 Post-infusion | Week 16 Pre-infusion | Week 16 Post-infusion | Week 32 Pre-infusion | Week 32 Post-infusion |
---|
OCR + SOC | 240.8 | 2 | 1.4 | 2.3 | 5.1 | 7.3 | 240 | 8.4 | 2 | 0.9 | 2.3 | 1 | 3.6 | 0.9 |
,OCR 1000 mg + SOC | 224.1 | 2.2 | 1.3 | 2 | 2.1 | 2.6 | 222.8 | 5.7 | 2.1 | 0.9 | 2.1 | 0.8 | 2 | 0.7 |
,OCR 400 mg + SOC | 256.3 | 1.9 | 1.4 | 2.6 | 7.8 | 11.7 | 256.3 | 11.1 | 1.9 | 1 | 2.5 | 1.2 | 5.1 | 1 |
,Placebo + SOC | 203.5 | 262.7 | 209.7 | 125.9 | 116.6 | 110 | 203.5 | 103.1 | 264.4 | 91.2 | 127.8 | 52.7 | 125 | 46.2 |
,Placebo-Euro Lupus (EL) | 186.3 | 163.3 | 143.3 | 74.7 | 68.5 | 61.3 | 186.3 | 104.3 | 164.5 | 65.8 | 76.3 | 30.9 | 72.2 | 27.6 |
,Placebo-Mycophenolate Mofetil (MMF) | 213.7 | 327.4 | 253.4 | 154.3 | 141.2 | 134.9 | 213.7 | 102.4 | 322.5 | 106.3 | 156.5 | 64.2 | 153.4 | 56.9 |
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Number of Participants Who Achieved Complete Renal Response (CRR)
CRR was defined as: 1. Normal serum creatinine (and with no more than a 25 percent [%] increase from Baseline); 2. Improvement in urinary protein:urinary creatinine ratio to less than or equal to (≤) 0.5. PRR was defined as at least 50 percent (%) reduction in proteinuria from Baseline, without more than 25% increase of serum creatinine at Week 48, compared with Baseline. If Baseline urine protein:urine creatinine ratio was greater than (>) 3, a urine protein:urine creatinine ratio of less than (<) 3 needed to be achieved. (NCT00626197)
Timeframe: Week 48
Intervention | Percentage of Participants (Number) |
---|
| CRR | PRR |
---|
OCR 1000 mg + SOC | 31.5 | 35.6 |
,OCR 400 mg + SOC | 42.7 | 24 |
,Placebo + SOC | 34.7 | 20 |
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Percentage of Participants With CD19+ Absolute B Cell Counts <10 Cells Per Microliter (Cells/uL)
CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. 0 represents 0% of participants. (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32
Intervention | Percentage of Participants (Number) |
---|
| Baseline | Day 15 | Week 4 | Week 16 | Week 32 | Week 48 | Day 1 Pre-infusion | Day 1 Post-infusion | Day 15 Pre-infusion | Day 15 Post-infusion | Week 16 Pre-infusion | Week 16 Post-infusion | Week 32 Pre-infusion | Week 32 Post-infusion |
---|
OCR + SOC | 2.9 | 98.6 | 99.3 | 96.5 | 93.6 | 93.4 | 2.9 | 82.5 | 98.6 | 100 | 97 | 100 | 94.4 | 100 |
,OCR 1000 mg + SOC | 1.5 | 97.1 | 100 | 97.1 | 95.6 | 98.5 | 1.5 | 83.8 | 97.1 | 100 | 97 | 100 | 95.2 | 100 |
,OCR 400 mg + SOC | 4.2 | 100 | 98.5 | 95.8 | 91.8 | 88.7 | 4.2 | 81.2 | 100 | 100 | 97 | 100 | 93.7 | 100 |
,Placebo + SOC | 4.3 | 4.5 | 4.4 | 4.3 | 7.4 | 5.9 | 4.3 | 10 | 4.4 | 7.5 | 4.5 | 11.5 | 3.3 | 15 |
,Placebo-Euro Lupus (EL) | 0 | 0 | 0 | 0 | 4.3 | 8.7 | 0 | 3.7 | 0 | 0 | 0 | 9.5 | 0 | 22.7 |
,Placebo-Mycophenolate Mofetil (MMF) | 6.8 | 7.5 | 7.3 | 6.7 | 8.9 | 4.4 | 6.8 | 14 | 7 | 11.9 | 7 | 12.5 | 5.1 | 10.5 |
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Percentage of Participants With CD19+ Absolute B Cell Counts <20 Cells/uL by Visit
CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. n = number of participants analyzed at the specified visit. 0 represents 0% of participants. (NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32
Intervention | Percentage of Participants (Number) |
---|
| Baseline | Day 15 | Week 4 | Week 16 | Week 32 | Week 48 | Day 1 Pre-infusion | Day 1 Post-infusion | Day 15 Pre-infusion | Day 15 Post-infusion | Week 16 Pre-infusion | Week 16 Post-infusion | Week 32 Pre-infusion | Week 32 Post-infusion |
---|
OCR + SOC | 7.3 | 99.3 | 100 | 98.6 | 96.5 | 94.9 | 7.2 | 89.1 | 99.3 | 100 | 98.5 | 100 | 97.6 | 100 |
,OCR 1000 mg + SOC | 3 | 98.5 | 100 | 98.6 | 100 | 98.5 | 3 | 91.2 | 98.6 | 100 | 98.5 | 100 | 100 | 100 |
,OCR 400 mg + SOC | 11.3 | 100 | 100 | 98.6 | 93.2 | 91.5 | 11.3 | 87 | 100 | 100 | 98.5 | 100 | 95.2 | 100 |
,Placebo + SOC | 7.1 | 7.6 | 10.3 | 11.4 | 13.2 | 17.6 | 7.1 | 22.9 | 7.4 | 17.9 | 10.4 | 41 | 8.3 | 35 |
,Placebo-Euro Lupus (EL) | 0 | 3.8 | 7.4 | 8 | 8.7 | 26.1 | 0 | 25.9 | 4 | 12 | 8.3 | 57.1 | 4.8 | 50 |
,Placebo-Mycophenolate Mofetil (MMF) | 11.4 | 10 | 12.2 | 13.3 | 15.6 | 13.3 | 11.4 | 20.9 | 9.3 | 21.4 | 11.6 | 32.5 | 10.3 | 26.3 |
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Percentage of Participants With CD19+ Absolute B Cell Counts Less Than the Lower Limit of Normal (LLN) by Visit
CD19 is a cell surface molecule which assembles with the antigen receptor of B lymphocytes. It is a critical signal transduction molecule that regulates B lymphocyte development, activation, and differentiation. NCT00626197)
Timeframe: Baseline, Day 15, Week 4, 16, 32, 48, and by infusion (pre and post infusion) on Day 1, 15, Week 16, 32
Intervention | Percentage of Participants (Number) |
---|
| Baseline | Day 15 | Week 4 | Week 16 | Week 32 | Week 48 | Day 1 Pre-infusion | Day 1 Post-infusion | Day 15 Pre-infusion | Day 15 Post-infusion | Week 16 Pre-infusion | Week 16 Post-infusion | Week 32 Pre-infusion | Week 32 Post-infusion |
---|
OCR + SOC | 35 | 100 | 100 | 100 | 98.6 | 98.5 | 34.8 | 98.5 | 100 | 100 | 100 | 100 | 99.2 | 100 |
,OCR 1000 mg + SOC | 33.3 | 100 | 100 | 100 | 100 | 100 | 32.8 | 100 | 100 | 100 | 100 | 100 | 100 | 100 |
,OCR 400 mg + SOC | 36.6 | 100 | 100 | 100 | 97.3 | 97.2 | 36.6 | 97.1 | 100 | 100 | 100 | 100 | 98.4 | 100 |
,Placebo + SOC | 27.1 | 28.8 | 39.7 | 57.1 | 52.9 | 51.5 | 27.1 | 55.7 | 30.0 | 58.2 | 56.7 | 80.3 | 50 | 88.3 |
,Placebo-Euro Lupus (EL) | 23.1 | 26.9 | 40.7 | 72 | 87 | 78.3 | 23.1 | 59.3 | 28 | 68 | 70.8 | 95.2 | 85.7 | 95.5 |
,Placebo-Mycophenolate Mofetil (MMF) | 29.5 | 30 | 39 | 48.9 | 35.6 | 37.8 | 29.5 | .5 | 32.6 | 52.4 | 48.8 | 72.5 | 30.8 | 84.2 |
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Number of Patients Alive 24 Months Post Day 100 Transplant
Patients will be followed for survival for 24 months post day 100 transplant. (NCT00619645)
Timeframe: 24 months post day 100 transplant
Intervention | Participants (Count of Participants) |
---|
RIST for Heme Malignancies | 3 |
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Disease Progression
Transplant recipients will be assessed for disease progression at 1 year after hematopoietic cell transplantation (HCT). The outcome is reported as the number of transplant recipients who experienced disease progression. (NCT03734601)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
TBI+TLI | 7 |
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Event-free Survival (EFS) at 1 Year
Event-free survival (EFS) is defined as the number of transplant recipients remaining alive at 12 months after transplant and who did not experience disease relapse defined as blasts < 5%. The outcome is expressed as the number of transplant recipients remaining alive at 12 months after transplant without disease relapse, a number without dispersion. (NCT03734601)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
TBI+TLI | 16 |
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Full-dose Donor Chimerism (FDC) at Day 28 Following TLI/ATG/TBI Conditioning.
Subsequent to TLI/ATG/TBI conditioning, the proportion of participants with full dose donor chimerism (FDC) will be determined by Day 28. FDC is defined as achieving ≥ 95% donor type in the CD3+ lineage within 28 days of donor cell infusion, as assessed by short tandem repeat (STR) testing. The outcome will be expressed as the number of participants that achieve FDC by Day 28, a number without dispersion. (NCT03734601)
Timeframe: Day 28
Intervention | Participants (Count of Participants) |
---|
TBI+TLI | 13 |
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Non-relapse Mortality (NRM)
Non-relapse mortality (NRM) is defined as death without known disease relapse or recurrence. The outcome is expressed as the number of transplant recipients whose cause of death was not disease relapse or recurrence, a number without dispersion. (NCT03734601)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
TBI+TLI | 2 |
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Overall Survival (OS)
Overall survival (OS) is defined as the number of transplant recipients remaining alive at 12 months after transplant. The outcome is expressed as the number of transplant recipients who remained alive at 12 months after treatment, a number without dispersion. (NCT03734601)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
TBI+TLI | 16 |
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Graft vs Host Disease (GvHD)
"Recipients will be monitored for Grade 2 to 4 graft vs host disease (GvHD). The outcome is reported as the number of transplant recipients who experienced acute GvHD grades 2 to 4, the number of transplant recipients who experienced chronic and extensive GvHD. In addition, the number of transplant recipients with chronic and extensive GvHD that was refractory to treatment (persistent) is reported. Per protocol, the result for chronic extensive and persistent GvHD is based on the subset of participants that had chronic and extensive GvHD." (NCT03734601)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
| Acute GvHD | Chronic extensive GvHD | Chronic extensive and persistent GvHD |
---|
TBI+TLI | 5 | 8 | 3 |
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Area Under the Curve (AUC) of CD25 Saturation by Basiliximab From Day 0 to Day 84
CD25 saturation is the percentage of T cells expressing CD25. Mean AUC of CD25 was calculated only for patients who received two Simulect® injections. (NCT01596062)
Timeframe: Day 84 (Week 12) after transplantation
Intervention | Weeks * Percentage of saturated CD25 (Mean) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 8.4 |
Simulect 80mg + Neoral + Myfortic + Steroids | 11.1 |
Simulect 80mg + Certican + Myfortic + Steroids | 9.7 |
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AUC of Basiliximab Binding to CD25 Receptors From Day 0 to Day 84
Mean AUC was calculated only for patients who received two Simulect injections. (NCT01596062)
Timeframe: Day 84 (Week 12) post-transplantation
Intervention | Weeks * Percentage of T cells (Mean) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 7.0 |
Simulect 80mg + Neoral + Myfortic + Steroids | 9.9 |
Simulect 80mg + Certican + Myfortic + Steroids | 8.4 |
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Estimated Glomerular Filtration Rate (eGFR) at Day 8 and Week 24
(MDRDa formula) with imputation by last observation carried forward (LOCF) (NCT01596062)
Timeframe: Day 8, Week 24
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Day 8 | Week 24 (n= 3, 6, 6) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 35.0 | 31.9 |
,Simulect 80mg + Certican + Myfortic + Steroids | 49.9 | 54.4 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 53.8 | 55.7 |
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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) According to Type and Severity
Antibody mediated acute rejection: C4d deposition, presence of circulating antidonor antibody, morphologic evidence of acute tissue injury such as acute tubular necrosis-like minimal inflammation or capillary and/or glomerular inflammation and/or thromboses or arterial inflammation. Cellular acute rejection: acute T-cell mediated rejection Type IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells) Type IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells) Type IIA: Mild to moderate intimal arteritis. Type IIB: Severe intimal arteritis comprising > 25% of the lumenal area. Type III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation). (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24 post-transplantation
Intervention | Percentage of participants (Number) |
---|
| Day 84 (Week 12): Antibody mediated AR - No | Day 84 (Week 12): Antibody mediated AR - Yes | Day 84 (Week 12): Cellular AR - No | Day 84 (Week 12): Cellular AR - Yes | Day 84 (Week 12): Banff type lA | Day 84 (Week 12): Banff type lB | Day 84 (Week 12): Banff type llA | Day 84 (Week 12): Banff type llB | Day 84 (Week 12): Banff type lll (C AR) | Week 24: Antibody mediated AR - No | Week 24: Antibody mediated AR - Yes | Week 24: Cellular AR - No | Week 24: Cellular AR - Yes | Week 24: Banff type lA | Week 24: Banff type lB | Week 24: Banff type llA | Week 24: Banff type llB | Week 24: Banff type lll |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 66.7 | 33.3 | 100.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 66.7 | 33.3 | 100.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 |
,Simulect 80mg + Certican + Myfortic + Steroids | 85.7 | 14.3 | 57.1 | 42.9 | 14.3 | 14.3 | 0.0 | 14.3 | 0.0 | 85.7 | 14.3 | 57.1 | 42.9 | 0.0 | 28.6 | 0.0 | 14.3 | 0.0 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 100.0 | 0.0 | 83.3 | 16.7 | 0.0 | 16.7 | 0.0 | 0.0 | 0.0 | 100.0 | 0.0 | 83.3 | 16.7 | 0.0 | 16.7 | 0.0 | 0.0 | 0.0 |
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Percentage of Participants With of Biopsy Proven Acute Rejection (BPAR)
BPAR is one of the components of treatment failure. One assessment of efficacy was BPAR. Renal graft biopsies were performed and the renal tissue was examined to determine if there was acute rejection of the renal transplant. (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24 post-transplantation
Intervention | Percentage of participants (Number) |
---|
| Day 84 (Week 12): No | Day 84 (Week 12):Yes | Week 24: No | Week 24:Yes |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 66.7 | 33.3 | 66.7 | 33.3 |
,Simulect 80mg + Certican + Myfortic + Steroids | 42.9 | 57.1 | 42.9 | 57.1 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 83.3 | 16.7 | 83.3 | 16.7 |
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Percentage of Participants With of Treatment Failures
Treatment failure was defined either as a BPAR, a graft loss, a death or a loss to follow-up. An extended treatment failure was also defined including treated borderline lesions, BPAR, graft loss, death or loss to follow-up. Treated borderline lesions were considered as acute rejection by investigators and DMC experts. (NCT01596062)
Timeframe: Day 84 (Week 12), Week 24
Intervention | Percentage of participants (Number) |
---|
| Day84 (Week 12): BPAR and/or borderline lesions-No | Day84 (Week 12):BPAR and/or borderline lesions-Yes | Day 84 (Week 12): Graft loss - No | Day 84 (Week 12): Graft loss - Yes | Day 84 (Week 12): Death - No | Day 84 (Week 12): Death - Yes | Day 84 (Week 12): Loss to follow-up - No | Day 84 (Week 12): Loss to follow-up - Yes | Week 24: BPAR or borderline lesions - No | Week 24: Graft loss - No | Week 24: Death - No | Week 24: Loss to follow-up - No | Week 24: Loss to follow-up - Yes | Week 24: BPAR or borderline lesions - Yes | Week 24: Graft loss - Yes | Week 24: Death - Yes |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 66.7 | 33.3 | 66.7 | 33.3 | 100.0 | 0.0 | 100.0 | 0.0 | 66.7 | 66.7 | 100.0 | 100.0 | 0.0 | 33.3 | 33.3 | 0.0 |
,Simulect 80mg + Certican + Myfortic + Steroids | 14.3 | 85.7 | 100.0 | 0.0 | 100.0 | 0.0 | 100.0 | 0.0 | 14.3 | 100.0 | 100.0 | 100.0 | 0.0 | 85.7 | 0.0 | 0.0 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 66.7 | 33.3 | 100.0 | 0.0 | 100.0 | 0.0 | 100.0 | 0.0 | 66.7 | 100.0 | 100.0 | 100.0 | 0.0 | 33.3 | 0.0 | 0.0 |
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Percentage of T-cells That Bind Basiliximab to CD25 Receptors
This is the percentage of T cells binding basiliximab at all timepoints. (NCT01596062)
Timeframe: Day 0, Day 1, Day 4, Day 6, Day 14, Day 21, Day 28, Day 42, Day 56 and Day 84 (Week 12) post-transplantation
Intervention | Percentage of T cells (Mean) |
---|
| Day 0 (before injection) | Day 0 (2 hours after injection) | Day 1 (n= 3, 5, 7) | Day 4 (before injection): (n= 3, 6, 6) | Day 4 (2 hours after injection): (n= 3, 5, 5) | Day 6: (n= 3, 6, 6) | Day 14: (n= 3, 6, 6) | Day 21: (n= 3, 6, 6) | Day 28: (n= 3, 6, 6) | Day 42: (n= 3, 6, 5) | Day 56: (n= 3, 6, 5) | Day 84 (Week 12): (n= 3, 6, 5) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 0.0 | 95.3 | 70.0 | 100.0 | 100.0 | 91.7 | 100.0 | 89.0 | 76.3 | 47.3 | 53.0 | 6.7 |
,Simulect 80mg + Certican + Myfortic + Steroids | 4.7 | 81.6 | 91.4 | 53.0 | 81.2 | 98.3 | 93.8 | 85.8 | 82.7 | 78.8 | 75.2 | 15.8 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 0.0 | 96.2 | 64.8 | 59.0 | 83.4 | 99.0 | 89.0 | 87.7 | 91.0 | 84.0 | 88.8 | 57.7 |
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Proportion of CD3+, CD4+, CD8+, CD19+ and CD56+ T Cells
Cell counts of various subpopulations of T, B and NK lymphocytes (CD3, CD4, CD8, CD19 and CD56) (flow cytometry). (NCT01596062)
Timeframe: Day 0, Day 6, Day 42, Day 84 (Week 12)
Intervention | 10^9 cells/L (Mean) |
---|
| CD3 cells count at Day 0 (before injection) | CD3 cells count at Day 6: (n= 3, 6, 6) | CD3 cells count at Day 42: (n= 3, 6, 5) | CD3 cells count at Day 84 (Week 12): (n= 3, 6, 6) | CD4 cells count at Day 0 (before injection) | CD4 cells count at Day 6: (n= 3, 6, 6) | CD4 cells count at Day 42: (n= 3, 6, 5) | CD4 cells count at Day 84 (Week 12): (n= 3, 6, 6) | CD8 cells count at Day 0 (before injection) | CD8 cells count at Day 6: (n= 3, 6, 6) | CD8 cells count at Day 42: (n= 3, 6, 5) | CD8 cells count at Day 84 (Week 12): (n= 3, 6, 6) | CD19 cells count at Day 0 (before injection) | CD19 cells count at Day 6: (n=3, 6, 6) | CD19 cells count at Day 42: (n= 3, 6, 5) | CD19 cells count at Day 84 (Week 12): (n= 3, 6, 6) | CD56 cells count at Day 0 (before injection) | CD56 cells count at Day 6: (n= 3, 6, 6) | CD56 cells count at Day 42: (n= 3, 6, 5) | CD56 cells count at Day 84 (Week 12): (n= 3, 6, 6) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 0.5 | 0.6 | 0.5 | 0.8 | 0.4 | 0.5 | 0.3 | 0.6 | 0.1 | 0.2 | 0.1 | 0.2 | 0.1 | 0.1 | 0.1 | 0.2 | 0.1 | 0.1 | 0.1 | 0.1 |
,Simulect 80mg + Certican + Myfortic + Steroids | 0.7 | 0.9 | 1.1 | 1.1 | 0.5 | 0.6 | 0.8 | 0.7 | 0.2 | 0.2 | 0.4 | 0.3 | 0.1 | 0.2 | 0.2 | 0.1 | 0.2 | 0.2 | 0.2 | 0.1 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 0.9 | 1.1 | 1.2 | 1.2 | 0.6 | 0.7 | 0.8 | 0.8 | 0.3 | 0.3 | 0.3 | 0.3 | 0.1 | 0.2 | 0.2 | 0.1 | 0.2 | 0.1 | 0.2 | 0.1 |
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Saturation Rate of CD25 Antigen Saturation by Basiliximab
CD25 saturation is the percentage of T cells expressing CD25 (NCT01596062)
Timeframe: Day 0, Day 1, Day 4, Day 6, Day 14, Day 21, Day 28, Day 42, Day 56 and Day 84 (Week 12) post-transplantation
Intervention | percentage of T cells (Mean) |
---|
| Day 0 (before injection) | Day 0 (2 hours after injection) | Day 1 | Day 4 (before injection); (n= 3, 6, 6) | Day 4 (2 hours after injection): (n= 3, 6, 5) | Day 6: (n= 3, 6, 6) | Day 14: (n= 3, 6, 6) | Day 21: (n= 3, 6, 6) | Day 28: (n= 3, 6, 6) | Day 42: (n= 3, 6, 5) | Day 56: (n= 3, 6, 5) | Day 84 (Week 12): (n= 3, 6, 5) |
---|
Simulect 40mg + Neoral + Myfortic + Steroids | 0.0 | 93.7 | 95.7 | 100.0 | 96.7 | 98.7 | 98.3 | 100.0 | 95.3 | 78.3 | 65.0 | 0.0 |
,Simulect 80mg + Certican + Myfortic + Steroids | 0.0 | 94.7 | 99.0 | 93.7 | 84.8 | 94.0 | 97.3 | 92.5 | 92.7 | 99.2 | 94.2 | 14.2 |
,Simulect 80mg + Neoral + Myfortic + Steroids | 0.0 | 96.7 | 97.7 | 96.2 | 96.3 | 100.0 | 96.7 | 94.5 | 100.0 | 100.0 | 93.3 | 67.5 |
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3.0% or Greater Improvement From Baseline in FVC-%.
The time (in months) required for each treatment arm to achieve a 3.0% or greater improvement from baseline in the FVC-% over the 18-month treatment period. (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | months (Median) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 17.8 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 12.3 |
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Forced Vital Capacity Volume (FVC, in ml)
Change from baseline to month 18 in the Forced Vital Capacity volume (FVC, in ml) (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | ml (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 121.53 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 112.33 |
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Greater Than 5% Improvement in FVC-%
The percentage of subjects in each treatment arm achieving greater than a 5% improvement in FVC-% over the 18-month treatment period. (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | Participants (Count of Participants) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 6 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 9 |
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Health Assessment Questionnaire Modified for Scleroderma (HAQ-DI)
"Change from baseline to month 18 as a subjective measure of dyspnea and quality of life.~HAQ-DI ranges from 0 (no disability) to 3 (severe disability)." (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | score on a scale (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | -0.03 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -0.17 |
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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Lobe of Maximal Involvement (QILD-LM)
"Change from screening to month 18 in computer-generated scoring of HRCT data for the percentage of imaging pixels exhibiting features characteristic for any of three patterns of ILD (including QGG, QLF and QHC) within the lobe of maximal involvement at baseline.~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative interstitial lung disease." (NCT03221257)
Timeframe: Screening to 18 months
Intervention | percent (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 2.99 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -0.99 |
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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Interstitial Lung Disease Score in the Whole Lung (QILD-WL)
"Change from screening to month 18 in computer-generated scoring of HRCT data from the whole lung for the percentage of imaging pixels that exhibit features of any of the three patterns of interstitial lung disease (ILD) including quantitative ground-glass opacity (QGG), lung fibrosis (QLF) and quantitative honeycombing (QHC).~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative interstitial lung disease." (NCT03221257)
Timeframe: Screening to 18 months
Intervention | percent (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 2.36 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -1.15 |
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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Lobe of Maximal Involvement (QLF-LM)
"Change from screening to month 18 in computer-generated scoring of HRCT data for the percentage of imaging pixels that exhibit features characteristic for lung fibrosis within the lobe of maximal involvement at baseline.~Individual image score range 0 to 100%, with higher percentages representing greater extent of quantitative lung fibrosis." (NCT03221257)
Timeframe: Screening to 18 months
Intervention | percent (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 2.57 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 0.13 |
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High Resolution Computerized Tomography (HRCT) Measures of Quantitative Lung Fibrosis Score in the Whole Lung (QLF-WL)
"Change from screening to month 18 in computer-generated scoring of HRCT data from the whole lung for the percentage of imaging pixels that exhibit features characteristic for lung fibrosis.~Individual image scores range 0 to 100%, with higher percentages representing greater extent of quantitative lung fibrosis." (NCT03221257)
Timeframe: Screening to 18 months
Intervention | percent (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 1.46 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -0.11 |
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High Resolution Computerized Tomography (HRCT) Measures of Total Lung Capacity (TLC)
"Change from screening to 18 months in quantitative HRCT measurement of TLC at maximal inspiration (HRCT-TLC).~Higher scores indicates a better outcome." (NCT03221257)
Timeframe: Screening to 18 months
Intervention | ml (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 70.69 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 191.99 |
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Mahler Modified Transitional Dyspnea Index (TDI)
The change from baseline to 18 months in dyspnea. The TDI score for each of three domains ranges from -3 (major deterioration) to +3 (major improvement). The sum of all domains yields the TDI total score (-9 to +9). (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | score on a scale (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 1.13 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 1.99 |
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Modified Rodnan Skin Score (mRSS)
Change from baseline to month 18 in the mRSS. mRSS scores have a range from 0 to 51, with higher score indicating greater skin involvement. (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | score on a scale (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | -5.42 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -4.96 |
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Percent Predicted Forced Vital Capacity (FVC-%)
Change from baseline to month 18 in the mean forced vital capacity (represented as the percentage of the age-; height-; gender-; and race-adjusted predicted value, i.e. FVC-%). (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | percent predicted (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 2.24 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 2.09 |
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Percent Predicted Single-breath Diffusing Capacity for Carbon Monoxide (DLCOHb-%)
Change from baseline to month 18 in DLCO, calculated as a percent of the age-; height-; gender-; race-; and hemoglobin-adjusted predicted value (DLCOHb-%). The raw DLCO value and adjusting it for all of these factors and presenting it as a percent of predicted (expected) is the outcome measure (DLCOHb-%). (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | percent predicted (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | 1.25 |
Pirfenidone (PFD) + Mycophenolate (MMF) | 1.24 |
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St. George's Respiratory Questionnaire (SGRQ)
Change from baseline to month 18 as a subjective measure of dyspnea and quality of life. SGRQ ranges from 0 (no impairment) to 100 (maximum impairment). (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | score on a scale (Least Squares Mean) |
---|
Placebo (Plac) + Mycophenolate (MMF) | -4.77 |
Pirfenidone (PFD) + Mycophenolate (MMF) | -6.11 |
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Time to Withdrawal From the Study Drug or Treatment Failure
The time from start of treatment to withdrawal or removal from active drug therapy (MMF or Plac/PFD separately) for any reason will be plotted over the course of the 18-month treatment as a measure of tolerability and toxicity. Median times to withdrawal are not available for reporting as less than half of the participants discontinued the study drugs. (NCT03221257)
Timeframe: Baseline to 18 months
Intervention | days (Median) |
---|
Placebo (Plac) + Mycophenolate (MMF) | NA |
Pirfenidone (PFD) + Mycophenolate (MMF) | NA |
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Cataract - Incident Cataract
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Fluocinolone Acetonide Implant | 90.7 |
Systemic Therapy | 44.9 |
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Change in Best-corrected Visual Acuity (Change in the Numbers of Letters Read From a Standard ETDRS Eye Chart) From Baseline to 24 Months in Eyes With Uveitis
Best-corrected visual acuity was measured as the number of letters read from standard logarithmic visual acuity charts by study-certified examiners who were masked to treatment. Visual acuity was measured at all study visits. The primary outcome was eye-specific change in visual acuity from baseline to 2-year follow-up. Positive change values indicate improved vision while negative change values indicate vision has gotten worse. A change of 7.5 letters is considered clinically meaningful. (NCT00132691)
Timeframe: 24 months
Intervention | letters (Mean) |
---|
Flucinolone Acetonide Implant | 6.0 |
Systemic Therapy | 3.2 |
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Change in SF-36 Mental Component Score From Baseline to 24 Months
Self-reported health related QoL was measured with the SF 36 survey. The mental component score for the SF 36 is a summary measure of mental health primarily based on the social functioning, role emotional, mental health and vitality domains. The score is scaled to a population norm with a mean of 50 and standard deviation of 10. Higher scores represent better outcomes. The mean change in scores between baseline and 24 months was calculated for each treatment group. (NCT00132691)
Timeframe: 24 months
Intervention | units on a scale (Mean) |
---|
Flucinolone Acetonide Implant | 2.55 |
Systemic Therapy | -1.1 |
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Change in SF-36 Physical Component Score From Baseline to 24 Months
Self-reported health related QoL was measured with the SF 36 survey. The physical component score for the SF 36 is a summary measure of physical health primarily based on the physical functioning, role physical, bodily pain and general health domains of the survey. The score is scaled to a population norm with a mean of 50 and standard deviation of 10. Higher scores represent better outcomes. The mean change in scores between baseline and 24 months was calculated for each treatment group. A 3 to 5 point difference is considered to be clinically meaningful. (NCT00132691)
Timeframe: 24 months
Intervention | units on a scale (Mean) |
---|
Flucinolone Acetonide Implant | 1.15 |
Systemic Therapy | -1.8 |
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Diabetes Mellitus
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of participants (Number) |
---|
Flucinolone Acetonide Implant | 1.0 |
Systemic Therapy | 3.6 |
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Glaucoma - Incident
Glaucoma was diagnosed by a glaucoma specialist through review of visual fields, clinical data, and fundus images. (NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Fluocinolone Acetonide Implant | 16.5 |
Systemic Therapy | 4.0 |
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Hyperlipidemia - Incident
LDL greater than or equal to 160 mg/mL (NCT00132691)
Timeframe: 24 months
Intervention | percentage of participants at risk (Number) |
---|
Flucinolone Acetonide Implant | 9.8 |
Systemic Therapy | 11.0 |
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Hypertension Diagnosis Requiring Treatment
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of participants (Number) |
---|
Flucinolone Acetonide Implant | 4.6 |
Systemic Therapy | 10.5 |
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Intraocular Pressure - Incident IOP Elevation >= 10 mmHg Above Baseline
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Fluocinolone Acetonide Implant | 51.8 |
Systemic Therapy | 15.5 |
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Intraocular Pressure - Incident IOP Greater Than or Equal to 24 mm Hg
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Flucinolone Acetonide Implant | 53.1 |
Systemic Therapy | 18.7 |
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Intraocular Pressure - Incident IOP Greater Than or Equal to 30 mm Hg
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Flucinolone Acetonide Implant | 32.8 |
Systemic Therapy | 6.3 |
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Intraocular Pressure - IOP-lowering Surgery
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Flucinolone Acetonide Implant | 26.2 |
Systemic Therapy | 3.7 |
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Intraocular Pressure (IOP) - Incident Use of IOP-lowering Medical Therapy (Percentage of Eyes With Uveitis That Were Not Being Treated With IOP-lowering Medical Therapy at Baseline and Underwent IOP Lowering Therapy During the 24 Month Follow-up.
The percentage of subjects who used topical or systemic treatment for elevated IOP at any time during the 2 year follow-up and were not on IOP-lowering therapy at baseline is reported. (NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis at risk (Number) |
---|
Flucinolone Acetonide Implant | 61.1 |
Systemic Therapy | 20.1 |
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Macular Edema
center point macular thickness >= 240 micrometers assessed on OCT (Stratus OCT-3 [Carl Zeiss Meditec, Dublin, CA]) as graded by Central Reading Center (NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis (Number) |
---|
Flucinolone Acetonide Implant | 22 |
Systemic Therapy | 30 |
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Mortality
(NCT00132691)
Timeframe: 24 months
Intervention | percentage of participants (Number) |
---|
Flucinolone Acetonide Implant | 1.6 |
Systemic Therapy | 0 |
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Uveitis Activity
Uveitis activity was determined by clinician assessment at each study visit. The study ophthalmologist evaluated each eye as active, inactive/never had uveitis or cannot assess. (NCT00132691)
Timeframe: 24 months
Intervention | percentage of eyes with uveitis (Number) |
---|
Fluocinolone Acetonide Implant | 12 |
Systemic Therapy | 29 |
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Clinically Treated Acute Rejection
"Clinically treated acute rejection is defined as patient incidence of any rejection (suspected or otherwise) for which treatment was provided. Data is reported as the percentage of patients with Clinically Treated Acute Rejection.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 22.9 |
Conventional High-Risk Patients | 21.7 |
Alemtuzumab Low- Risk Patients | 12.8 |
Conventional Low-Risk Patients | 26.9 |
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Efficacy Failure
"Efficacy Failure is a composite measure of biopsy confirmed acute rejection, graft loss and death. Data is reported as the percentage of patients with Efficacy Failure.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 22.9 |
Conventional High-Risk Patients | 29.0 |
Alemtuzumab Low- Risk Patients | 15.9 |
Conventional Low-Risk Patients | 24.6 |
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Graft Survival at 12 Months
"Graft survival is defined as no graft loss (re-transplant, return to dialysis for more than 30 days or death) with 12 months of skin closure.~Kaplan Meier analysis was used to estimate percentage of patients with event. Patients with no event by the time of the scheduled visit or whose first event was after premature discontinuation of randomized study drug or tacrolimus were censored on the scheduled day of assessment, on the day of premature treatment discontinuation or last evaluation day, whichever came first." (NCT00113269)
Timeframe: 12 months
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 95.6 |
Conventional High-Risk Patients | 92.1 |
Alemtuzumab Low- Risk Patients | 97.5 |
Conventional Low-Risk Patients | 95.1 |
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Overall Graft Survival
"Overall graft survival is defined as not having graft loss (re-transplant, return to dialysis for more than 30 consecutive days, or death) at any time following skin closure. Data is reported as the percentage of patients with Overall Graft Survival.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 88.6 |
Conventional High-Risk Patients | 82.6 |
Alemtuzumab Low- Risk Patients | 90.9 |
Conventional Low-Risk Patients | 91.2 |
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Overall Patient Incidence of BCAR
"Overall patient incidence of BCAR is defined as a suspected new rejection at any time following skin closure confirmed by a Banff Grade ≥ 1A as assigned by a local pathologist. Incidence is reported as the percentage of patients with BCAR. The Banff 97 scale is a classification system for interpreting histology of allograft biopsies. The grades range from Mild (1A & 1B) to Moderate (2A & 2B) to Severe (3).~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 15.7 |
Conventional High-Risk Patients | 13.0 |
Alemtuzumab Low- Risk Patients | 9.8 |
Conventional Low-Risk Patients | 21.6 |
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Overall Patient Survival
"Overall patient survival is defined as not dead at any time following skin closure. Data is reported as the percentage of patients with Overall Patient Survival.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 95.7 |
Conventional High-Risk Patients | 89.9 |
Alemtuzumab Low- Risk Patients | 93.9 |
Conventional Low-Risk Patients | 95.3 |
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Patient Incidence of Biopsy-confirmed Acute Rejection (BCAR) at 6 Months
"A BCAR is a suspected new rejection w/in 6 mos. of skin closure, confirmed by Banff Grade ≥1A assigned by a pathologist. The Banff 97 classification system is used for interpreting histology of allograft biopsies, including Mild (1A/1B), Moderate (2A/2B) & Severe (3).~Kaplan Meier analysis was used to estimate % of pts. w/event. Patients w/no event at time of scheduled visit or whose 1st event was after premature discontinuation of study drug/tacrolimus were censored on the scheduled day of a) assessment, b) of premature treatment discontinuation or c) last evaluation, whichever came 1st." (NCT00113269)
Timeframe: 6 months
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 6.2 |
Conventional High-Risk Patients | 9.4 |
Alemtuzumab Low- Risk Patients | 1.9 |
Conventional Low-Risk Patients | 17.5 |
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Patient Survival at 12 Months
"Patient survival is defined as not dead within 12 months after skin closure.~Kaplan Meier analysis was used to estimate percentage of patients with event. Patients with no event by the time of the scheduled visit or whose first event was after premature discontinuation of randomized study drug or tacrolimus were censored on the scheduled day of assessment, on the day of premature treatment discontinuation or last evaluation day, whichever came first." (NCT00113269)
Timeframe: 12 months
Intervention | Percentage of Patients (Number) |
---|
Alemtuzumab High-Risk Patients | 98.6 |
Conventional High-Risk Patients | 96.9 |
Alemtuzumab Low- Risk Patients | 98.1 |
Conventional Low-Risk Patients | 98.7 |
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Time to First BCAR
"Time to first BCAR is defined as the number of days from skin closure to the first episode of BCAR.~End of Study was defined as the last day of evaluation and could have included bivariate assessments after 36 months." (NCT00113269)
Timeframe: End of Study (36 months)
Intervention | Days (Median) |
---|
Alemtuzumab High-Risk Patients | 226 |
Conventional High-Risk Patients | 49 |
Alemtuzumab Low- Risk Patients | 469 |
Conventional Low-Risk Patients | 13 |
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Renal Function Abnormalities Based on Creatinine Clearance
"Increases in creatinine clearance usually indicates an improvement.~Change in creatinine clearance from month 1 was calculated.~Change from 1 month is calculated by month 36 - month 1." (NCT00113269)
Timeframe: 1 month and End of Study (36 months)
Intervention | mL/s (Mean) |
---|
| Month 1 (N= 65; 65; 162; 161) | Month 36 (N= 48; 47; 125; 131) | Change from Month 1 (N= 48; 46; 125; 129) |
---|
Alemtuzumab High-Risk Patients | 0.882 | 0.976 | 0.031 |
,Alemtuzumab Low- Risk Patients | 0.906 | 1.011 | 0.080 |
,Conventional High-Risk Patients | 0.833 | 0.862 | 0.039 |
,Conventional Low-Risk Patients | 0.891 | 1.039 | 0.140 |
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Renal Function Abnormalities Based on Serum Creatinine
"Decrease in serum creatinine usually indicates an improvement.~Change in creatinine clearance from month 1 was calculated.~Change from 1 month is calculated by month 36 - month 1." (NCT00113269)
Timeframe: 1 month and End of Study (36 months)
Intervention | mcmol/L (Mean) |
---|
| Month 1 (N= 67; 65; 163; 162) | Month 36 (N= 49; 47; 126; 131) | Change from Month 1 (N= 49; 46; 126; 129) |
---|
Alemtuzumab High-Risk Patients | 151.7 | 136.6 | 1.8 |
,Alemtuzumab Low- Risk Patients | 139.0 | 121.7 | -8.0 |
,Conventional High-Risk Patients | 155.9 | 152.0 | 3.4 |
,Conventional Low-Risk Patients | 138.2 | 117.1 | -18.7 |
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Disease-Free Survival (DFS)
Estimate the one-year disease-free survival (DFS) for research participants who receive this study treatment. DFS is defined as time from transplantation to the occurrence of relapse or death due to relapse. Patients who are alive at the time of analysis or die due to other causes will be censored at the time of their events. The estimated percentage of participants with DFS at one-year post-transplantation is reported using Kaplan-Meier analysis. (NCT00566696)
Timeframe: One year post-transplant
Intervention | Percentage of participants (Number) |
---|
High-Risk Hematologic Malignancies | 70.1 |
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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
Intervention | Percentage of participants (Number) |
---|
High-Risk Hematologic Malignancies | 54.8 |
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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
Intervention | Percentage of participants (Number) |
---|
High-Risk Hematologic Malignancies | 71.0 |
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To Estimate the Cumulative Incidence of Relapse for Research Participants Who Receive This Study Treatment.
The Cumulative Incidence of Relapse will be reported as the proportion of number of relapses since transplant to the total number of patients at risk at five years post-transplant. (NCT00566696)
Timeframe: five years post-transplant
Intervention | Percentage of participants (Number) |
---|
| The Cumulative Incidence of Relapse at five year p | Estimate±SE |
---|
High-Risk Hematologic Malignancies | 30.0 | 8.6 |
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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
Intervention | Percentage of participants (Number) |
---|
| Rate of Overall Grade III-IV Acute AVHD | Rate of limited grade Chronic GVHD |
---|
High-Risk Hematologic Malignancies | 22.58 | 9.68 |
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Proportion of Participants Successfully Withdrawn and Remain Off Immunosuppressants
This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee, were successfully withdrawn from immunosuppressants, and remained off immunosuppressants at the time the trial ended. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks and do not restart immunosuppressant drugs after successful withdrawal. (NCT00105235)
Timeframe: From 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)
Intervention | Proportion of Participants (Number) |
---|
Alemtuzumab | 0.1 |
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Proportion of Participants Successfully Withdrawn From Immunosuppressants
This measure of tolerance induction includes the proportion of participants who qualify for immunosuppression withdrawal as determined by a review of individual clinical results by a protocol withdrawal committee. Successful withdrawal definition: participants who remain off immunosuppression for at least 8 weeks. (NCT00105235)
Timeframe: From 1 year post- transplantation until study completion or participant termination (participants followed up to 48 months post-transplant)
Intervention | Proportion of Participants (Number) |
---|
Alemtuzumab | 0.2 |
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Proportion of Participants Who Had Graft Loss or Death
Proportion of participants who had liver graft loss or who died or terminated from the study within 2 years of initiating immunosuppression withdrawal (NCT00105235)
Timeframe: Within 2 years after initiation of immunosuppression withdrawal
Intervention | Proportion of Participants (Number) |
---|
Alemtuzumab | 0 |
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Proportion of Participants Who Have Graft Loss or Death
Proportion of participants who had liver graft loss or who died within 1 year of undergoing transplantation. Note: Participants who discontinued treatment or terminated the study prior to 1 year post transplantation are considered treatment failures and are included in this measure. (NCT00105235)
Timeframe: Within 1 year of post-transplantation
Intervention | Proportion of Participants (Number) |
---|
Alemtuzumab | 0.22 |
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Change in Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients.
Primary efficacy endpoint: between treatment analysis of change in iohexol plasmatic clearance (mL/min) from baseline to Month 12 (M12) (NCT00425308)
Timeframe: From Baseline to Month 12
Intervention | (mL/min) (Least Squares Mean) |
---|
Cyclosporine | -4.07 |
Enteric-coated Mycophenolate Sodium (EC-MPS) | 10.30 |
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Change in the Glomerular Filtration Rate Estimated by Iohexol Plasma Clearance 12 Months After Randomization Between the 2 Groups of Patients Who Completed Trial
Primary efficacy endpoint: between treatment analysis of change in iohexol plasmatic clearance (mL/min) from baseline to Month 12 (M12) (NCT00425308)
Timeframe: From Baseline to Month 12
Intervention | (mL/min) (Least Squares Mean) |
---|
Cyclosporine | 1.46 |
Enteric-coated Mycophenolate Sodium (EC-MPS) | 12.00 |
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Assessing Cardiovascular Risk Factors Based on Fasting C-reactive Protein (CRP).
Blood chemistry - C-reactive Protein (CRP) (mg/L) (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12
Intervention | mg/L (Mean) |
---|
| Baseline [N = 13; N = 14] | Month 3 [N = 11; N = 11] | Month 6 [N = 11; N = 9] | Month 12 [N = 14; N = 12] |
---|
Cyclosporine | 11.4 | 6.3 | 8.0 | 34.2 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 3.0 | 10.3 | 5.6 | 12.6 |
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Assessing Cardiovascular Risk Factors Based on Fasting Glucose.
Blood chemistry - fasting glycemia (mmol/L) (NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12
Intervention | mmol/L (Mean) |
---|
| Baseline [N = 15; N = 15] | Month 1 [N = 15; N = 15] | Month 3 [N = 14; N = 15] | Month 6 [N = 14; N = 14] | Month 9 [N = 12; N = 13] | Month 12 [N = 14; N = 13] |
---|
Cyclosporine | 5.5 | 5.2 | 5.6 | 5.5 | 5.3 | 4.8 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 5.2 | 5.3 | 5.2 | 5.1 | 5.6 | 5.4 |
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Assessing Cardiovascular Risk Factors Based on Fasting High-density Lipoprotein (HDL) Cholesterol, Low-density Lipoprotein (LDL) Cholesterol.
(NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12
Intervention | mmol/L (Mean) |
---|
| Baseline LDL [N = 10; N = 14] | Baseline HDL [N = 10; N = 14] | Month 3 LDL [N = 12; N = 14] | Month 3 HDL [N = 12; N = 13] | Month 6 LDL [N = 12; N = 12] | Month 6 HDL [N = 12; N = 12] | Month 12 LDL [N = 10; N = 11] | Month 12 HDL [N = 10; N = 11] |
---|
Cyclosporine | 3.4 | 1.5 | 3.2 | 1.7 | 3.2 | 1.6 | 3.0 | 1.7 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 3.1 | 1.5 | 3.4 | 1.5 | 3.3 | 1.4 | 3.2 | 1.5 |
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Assessing Cardiovascular Risk Factors Based on Fasting Total Cholesterol.
Blood chemistry - total cholesterol (mmol/L) (NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12
Intervention | mmol/L (Mean) |
---|
| Baseline [N = 13; N = 14] | Month 1 [N = 11; N = 14] | Month 3 [N = 13; N = 14] | Month 6 [N = 12; N = 12] | Month 9 [N = 9; N = 12] | Month 12 [N = 12; N = 11] |
---|
Cyclosporine | 5.7 | 5.0 | 5.3 | 5.5 | 5.4 | 5.6 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 6.0 | 5.7 | 5.6 | 5.7 | 5.4 | 5.8 |
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Assessing Cardiovascular Risk Factors Based on Fasting Triglycerides.
(NCT00425308)
Timeframe: From Baseline to Month 1, 3, 6, 9, and 12
Intervention | mmol/L (Mean) |
---|
| Baseline [N = 11; N = 14] | Month 1 [N = 10; N = 14] | Month 3 [N = 13; N = 14] | Month 6 [N = 12; N = 12] | Month 9 [N = 10; N = 12] | Month 12 [N = 12; N = 11] |
---|
Cyclosporine | 2.4 | 2.1 | 2.0 | 1.9 | 1.9 | 2.3 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 1.8 | 2.0 | 2.2 | 2.1 | 2.2 | 2.3 |
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Change in Renal Function Assessed by Creatinine Clearance at Month 3, Month 6 and Month 12
Change in creatinine clearance, Nankivell formula (mL/min/1.73m²) from baseline to M12 (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12
Intervention | mL/min/1.73m² (Mean) |
---|
| Baseline [N = 12; N = 13] | Month 3 [N = 10; N = 14] | Month 6 [N = 10; N = 12] | Month 12 [N = 7; N = 11] |
---|
Cyclosporine | 56.3 | 57.3 | 50.3 | 48.7 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 60.3 | 67.1 | 68.1 | 70.1 |
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Change in Renal Function Assessed by Proteinuria at Month 3, Month 6 and Month 12
Change in proteinuria (g/24h) from baseline to M12 (NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12
Intervention | g/24h (Mean) |
---|
| Baseline [N = 9; N = 10] | Month 3 [N = 9; N = 8] | Month 6 [N = 6; N = 5] | Month 12 [N = 7; N = 10] |
---|
Cyclosporine | 0.44 | 0.53 | 0.44 | 0.48 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 0.47 | 0.24 | 0.33 | 0.75 |
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Change in Renal Function Assessed by Serum Creatinine at Month 3, Month 6 and Month 12
(NCT00425308)
Timeframe: From Baseline to Month 3, 6, and 12
Intervention | µmol/L (Mean) |
---|
| Baseline [N = 13; N = 14] | Month 3 [N = 12; N = 14] | Month 6 [N = 12; N = 13] | Month 12 [N = 13; N = 14] |
---|
Cyclosporine | 161.7 | 156.6 | 170.8 | 227.7 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 153.2 | 138.9 | 139.8 | 130.4 |
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Number of Participants With Biopsy-proven Acute Rejection (BPAR) at Month 6 and Month 12.
(NCT00425308)
Timeframe: Month 6 and 12
Intervention | participants (Number) |
---|
| Yes: treatment failure Month 6 | No: treatment failure Month 6 | Yes: treatment failure Month 12 | No: treatment failure Month 12 |
---|
Cyclosporine | 0 | 0 | 1 | 12 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 0 | 0 | 0 | 14 |
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Number of Participants With Treatment Failures Assessed by Biopsy-proven Acute Rejection (BPAR), Graft Loss/Re-transplantation, Death or Lost to Follow-up at Month 12.
(NCT00425308)
Timeframe: Month 12
Intervention | participants (Number) |
---|
| BPAR 12 months | Graft Loss 12 months | Death 12 months | Lost to Follow-up 12 months |
---|
Cyclosporine | 1 | 0 | 1 | 0 |
,Enteric-coated Mycophenolate Sodium (EC-MPS) | 0 | 0 | 0 | 0 |
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Number of Participants With the Occurrence of Treatment Failures at 6 Months Post-transplantation
Treatment failures defined as Biopsy Proven Acute Rejection (BPAR), graft loss, death or loss to follow-up. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. Graft loss: allograft will be presumed to be lost on the day the patient starts dialysis and is not able to subsequently be removed from dialysis. (NCT00413920)
Timeframe: 6 months post transplantation
Intervention | Number of participants (Number) |
---|
Without Steroids | 20 |
With Steroids | 16 |
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Number of Participants Requiring Steroids in Non-steroid Treatment Group
(NCT00413920)
Timeframe: Months 3 and 6
Intervention | Number of participants (Number) |
---|
| 3 Months | 6 Months |
---|
Without Steroids | 25 | 20 |
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Number of Participants With Subclinical Histological Rejections
The number of participants with subclinical histological rejections was determined by renal biopsy screening at 3 months in 125 patients, providing adequate samples for 112 biopsies. (NCT00413920)
Timeframe: Month 3
Intervention | Number of participants (Number) |
---|
| Sample quality inadequate | Subclinical rejection | Borderline lesions | BPAR |
---|
With Steroids | 6 | 17 | 5 | 12 |
,Without Steroids | 7 | 12 | 2 | 10 |
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Number of Participants With Treatment Failure at 3 Months by Graft Recovery Status
"The number of participants with treatment failure defined as a Biopsy Proven Acute Rejection (BPAR), a graft loss, a death, or a loss to follow-up at 3 months by graft recovery status.~Delayed graft function is defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day.~Slow graft function is defined as a serum creatinine value > 250 µmol/L at day 5." (NCT00413920)
Timeframe: Month 3
Intervention | Number of participants (Number) |
---|
| Delayed Graft Function [N= 25,24] | Slow Graft Function [N= 36,23] | Immediate Graft Function [N= 51,63] |
---|
With Steroids | 4 | 1 | 3 |
,Without Steroids | 8 | 6 | 3 |
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Number of Participants With Treatment Failure, BPAR, Clinical Acute Rejection (AR) and Treated AR at 3 Months
A treatment failure is a Biopsy Proven Acute Rejection (BPAR), a graft loss, a death, or a loss to follow-up. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. Graft loss: The allograft will be presumed lost on the day the patient starts dialysis and is not able to subsequently be removed from dialysis. (NCT00413920)
Timeframe: Month 3
Intervention | Number of participants (Number) |
---|
| Treatment Failure | Biopsy Proven Acute Rejection | Graft Loss | Death | Loss to Follow-up | Acute Rejection | Treated Acute Rejection |
---|
With Steroids | 8 | 5 | 1 | 2 | 0 | 19 | 16 |
,Without Steroids | 17 | 10 | 5 | 2 | 0 | 32 | 31 |
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The Number of Participants With BPAR, Clinical Acute Rejection (AR) and Treated AR at 6 Months
If a participant experienced several BPAR, only the rejection with highest grade is taken into account. Only events that occurred before study treatment discontinuation are taken into account. Only BPAR from other biopsies than the protocol defined biopsy at Month 3 are described. Acute rejection: an episode of acute renal dysfunction diagnosed as rejection on the basis of biopsy or clinical observations, treated with anti-rejection medication. BPAR: renal transplant biopsy finding of acute cellular or antibody mediated rejection. (NCT00413920)
Timeframe: Month 6
Intervention | Number of participants (Number) |
---|
| Biopsy Proven Acute Rejection | Graft Loss | Death | Loss to Follow-up | Acute Rejection | Treated Acute Rejection |
---|
With Steroids | 8 | 3 | 5 | 0 | 21 | 19 |
,Without Steroids | 13 | 5 | 2 | 0 | 37 | 36 |
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The Number of Participants With Subclinical Rejection as Evaluated by a Change in Liver Enzymes
The number of participants with subclinical rejection episodes as defined by a steroid-sensitive, clinically relevant increase of AST, ALT, gamma-GT, AP or bilirubin (i.e., elevation of one or more of these enzymes that was considered clinically relevant and showed resolution upon treatment with a slight increase of steroid dosage). (NCT00405652)
Timeframe: 12-20 weeks
Intervention | Participants (Number) |
---|
Enteric-coated Mycophenolate Sodium | 6 |
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Number of Patients With Treatment Failure 6-months Post Transplant Measured by the Combined Incidence of Biopsy Proven Acute Rejection, Graft Loss, and Death
To evaluate therapeutic benefit by comparing the efficacy defined as the number of participants with treatment failure (biopsy-proven acute rejection [BPAR], graft loss [GFL] or death) at 6 months post-transplant. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III using Banff 2000 classification. A graft core biopsy was performed within 24 hours of initiation of anti-rejection therapy. GFL was defined as the day the allograft was presumed lost (the day the patient started dialysis, the day of nephrectomy or the day of irreversible graft loss demonstrated by imaging techniques.) (NCT00419926)
Timeframe: 6 months
Intervention | number of participants (Number) |
---|
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen | 33 |
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen | 36 |
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen | 26 |
Standard Mycophenolate Sodium (Myfortic) Dosing Regimen | 35 |
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Comparison of Overall Treatment Failure at Days 21 and 84 Post-transplantation Assessed by Biopsy Proven Acute Rejection (BPAR), GFL, and Death
The overall treatment differences of the number of participants with at least one occurrence of the composite event BPAR, GFL or death at study days 21 and 84 post-transplantation. BPAR was defined as a biopsy graded IA, IB, IIA, IIB or III using Banff 2000 classification. A graft core biopsy was performed within 24 hours of initiation of anti-rejection therapy. GFL was defined as the day the allograft was presumed lost (the day the patient started dialysis, the day of nephrectomy or the day of irreversible graft loss demonstrated by imaging techniques.) (NCT00419926)
Timeframe: 21 and 84 days
Intervention | number of participants (Number) |
---|
| Day 84 | Day 21 |
---|
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen | 20 | 33 | 14 | 26 |
,Standard Mycophenolate Sodium (Myfortic) Dosing Regimen | 21 | 34 | 20 | 33 |
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Renal Function Assessed by Glomerular Filtration Rate (GFR)at Each Visit
The Modification of Diet in Renal Disease (MDRD) formula was used to calculate the GFR. Serum creatinine levels, age, sex and race were used to estimate the GFR levels in mL/min/1.73m^2. (NCT00419926)
Timeframe: at 21 days, 84 days and 180 days
Intervention | (mL/min/1.73m^2) (Mean) |
---|
| At 21 days | At 84 days | At 180 days |
---|
Intensified Mycophenolate Sodium (Myfortic) Dosing Regimen | 47.3 | 52.1 | 53.5 |
,Standard Mycophenolate Sodium (Myfortic) Dosing Regimen | 46.8 | 51.8 | 51.3 |
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Graft Survival
The median time, in months, between randomization and graft loss event. Participants were censored at the date of last follow up, the date of last contact or premature withdrawal, and date of death. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment.
Intervention | months (Median) |
---|
Adjusted MMF + Tacrolimus + CS | 12.9 |
Fixed-Dose MMF + Tacrolimus + CS | 12.9 |
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Overall Survival (OS) at Month 12 - Percentage of Participants With an Event
OS was defined as the time between the date of randomization and death up to Month 12. Participants were censored at the date of last follow up and the date of last contact or premature withdrawal. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12
Intervention | percentage of participants (Number) |
---|
Adjusted MMF + Tacrolimus + CS | 8.9 |
Fixed-Dose MMF + Tacrolimus + CS | 11.1 |
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Overall Survival at Month 12
The median time, in months, between randomization and OS event. Participants were censored at the date of last follow up and the date of last contact or premature withdrawal. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12
Intervention | months (Median) |
---|
Adjusted MMF + Tacrolimus + CS | 12.9 |
Fixed-Dose MMF + Tacrolimus + CS | 12.9 |
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Percentage of Participants With Graft Loss
Graft survival was defined as the time between the randomization date and the graft loss date. Participants were censored at the date of last follow up, the date of last contact or premature withdrawal, and date of death. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment.
Intervention | percentage of participants (Number) |
---|
Adjusted MMF + Tacrolimus + CS | 2.2 |
Fixed-Dose MMF + Tacrolimus + CS | 5.6 |
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Percentage of Participants With Treated Biopsy Proven Acute Rejection (BPAR) According to Banff Criteria up to 12 Months Post-Transplant
Banff criteria required at least 2 of the 3 following features for a histopathological diagnosis of acute rejection: portal inflammation, bile duct inflammation, and venous endothelial inflammation. Each item was graded from 0 to 3 where 0 equals (=) mild, 2 = moderate, and 3 = severe. The sum of the 3 individual scores, from 0 to 9, corresponded to the Rejection Activity Index (RAI). If RAI = 0, 1, or 2, there was no evidence of rejection. If RAI = 3, there was borderline acute rejection. If RAI = 4 or 5, there was mild acute rejection. If RAI = 6 or 7, there was moderate acute rejection. If RAI = 8 or 9, there was severe acute rejection. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12, 28 days after Month 12 or last dose of study treatment, and 6 and 12 months after the last dose of study treatment
Intervention | percentage of participants (Number) |
---|
Adjusted MMF + Tacrolimus + CS | 8.0 |
Fixed-Dose MMF + Tacrolimus + CS | 8.2 |
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Percentage of Participants by Graft Histology at 12 Months Post-Transplant - Central Review
The percentage of participants with biopsies of grafts evaluated by central review and scored according to Banff criteria at Month 12 post-transplant. (NCT00545402)
Timeframe: Days 0, 5, and 14, Month 1, 2, 3, 6, 9, and 12
Intervention | percentage of participants (Number) |
---|
| Normal liver | Minor lesions | Acute rejection | Chronic rejection | Chronic hepatitis | Vascular lesions | Pathology of biliary obstruction | Lobular hepatitis | Recurrence of initial autoimmune disease | Other |
---|
Adjusted MMF + Tacrolimus + CS | 4.8 | 4.8 | 0.0 | 7.1 | 26.2 | 35.7 | 4.8 | 4.8 | 0.0 | 35.7 |
,Fixed-Dose MMF + Tacrolimus + CS | 11.4 | 17.1 | 0.0 | 2.9 | 22.9 | 11.4 | 5.7 | 2.9 | 0.0 | 45.7 |
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Number of Participants With Engraftment Syndrome
(NCT00412360)
Timeframe: Day 100 post-transplant
Intervention | Participants (Count of Participants) |
---|
Single UCB Transplant | 11 |
Double UCB Transplant | 7 |
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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
Intervention | percentage of participants (Number) |
---|
Single UCB Transplant | 70 |
Double UCB Transplant | 64 |
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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
Intervention | percentage of participants (Number) |
---|
Single UCB Transplant | 73 |
Double UCB Transplant | 65 |
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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
Intervention | percentage of participants (Number) |
---|
Single UCB Transplant | 12 |
Double UCB Transplant | 14 |
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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
Intervention | percentage of participants (Number) |
---|
| Acute GVHD Grade II-IV | Acute GVHD Grade III-IV |
---|
Double UCB Transplant | 56 | 23 |
,Single UCB Transplant | 57 | 13 |
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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
Intervention | percentage of participants (Number) |
---|
| Chronic GVHD | Extensive Chronic GVHD |
---|
Double UCB Transplant | 32 | 15 |
,Single UCB Transplant | 30 | 9 |
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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
Intervention | percentage of participants (Number) |
---|
| Neutrophil Engraftment at Day 42 | Platelet Engraftment at Day 100 |
---|
Double UCB Transplant | 88 | 65 |
,Single UCB Transplant | 89 | 76 |
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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
Intervention | days (Median) |
---|
| Neutrophil Engraftment | Platelet Engraftment |
---|
Double UCB Transplant | 23 | 84 |
,Single UCB Transplant | 21 | 58 |
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Number of Participants Using Antihyperlipidemic Medications at Month 12 - Intent to Treat Population
Participants using > = 1 antihyperlipidemic medication at Month 12. (NCT00455013)
Timeframe: Month 12
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 11 |
Belatacept, Sirolimus | 10 |
Tacrolimus, MMF | 9 |
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Number of Participants With Acute Rejection of Transplant up to End of Month 48 Post Transplantation - Intent to Treat Population in Long Term Extension
AR defined as a clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) >= 25 % from baseline plus one or more of the following: unexplained decreased urine output; fever and graft tenderness; a SCr that remained elevated within 14 days after transplantation and clinical suspicion of AR; reason other than those listed and participant was treated for this episode. Day 1 was day of transplantation. Banff grade used Banff 97 working classification of kidney transplant pathology. ITT population was all randomized and transplanted participants. (NCT00455013)
Timeframe: End of Month 12 to end of Month 48 Post Transplantation
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 0 |
Belatacept, Sirolimus | 0 |
Tacrolimus, MMF | 0 |
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Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 12 - Intent to Treat Population
Subjects with graft loss or death prior to Month 12 were considered having an event of AR, therefore, the incidence of AR was reported as a composite of AR, death, and graft loss. (NCT00455013)
Timeframe: Day 1 up to Month 12
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 7 |
Belatacept, Sirolimus | 3 |
Tacrolimus, MMF | 1 |
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Number of Participants With Composite of Death, Graft Loss and Acute Rejection up to Month 6 - Intent to Treat Population
Participants with graft loss or death prior to Month 6 were considered having an event of AR, therefore, the incidence of AR was reported as a composite of AR, death, and graft loss. (NCT00455013)
Timeframe: Day 1 up to Month 6
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 6 |
Belatacept, Sirolimus | 2 |
Tacrolimus, MMF | 1 |
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Number of Participants With Delayed Graft Function - Intent to Treat Population
Delayed graft function (DGF) is defined as participant requiring dialysis within the first week (Day 1-8) post transplantation. Participants losing their graft less than 48 hours post transplant and receiving chronic dialysis were not considered as having DGF. Day 1 was day of transplantation. Intent to treat population defined as all participants randomized and transplanted (NCT00455013)
Timeframe: From Day 1 up to and including Day 8 post transplantation
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 6 |
Belatacept, Sirolimus | 4 |
Tacrolimus, MMF | 2 |
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Number of Participants With New Onset Diabetes Mellitus From Baseline to Month 12 Post Transplantation - Intent to Treat Population
"Baseline defined as day before transplantation. A participant who did not have diabetes prior to randomization and received an antidiabetic medication for a duration of at least 30 days or a participant who meets the following criteria and did not have diabetes prior to randomization: Symptoms of diabetes plus casual plasma glucose (PG) concentration ≥ 200 mg/dL (11.1 mmol/L); or fasting plasma glucose ≥ 126 mg/dL (7.0 mmol/L); or 2-hour PG ≥ 200 mg/dL (11.1 mmol/L) during an oral glucose tolerance test and a confirmatory laboratory test based on measurements of venous PG must have been done on another day in the absence of unequivocal hyperglycemia accompanied by acute metabolic decompensation.~Intent to treat population included all participants randomized and transplanted." (NCT00455013)
Timeframe: Baseline to Month 12
Intervention | participants (Number) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 0 |
Belatacept, Sirolimus | 2 |
Tacrolimus, MMF | 1 |
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Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Month 3, Month 6 and Month 12 Post Transplantation - Intent to Treat Population
Blood urea nitrogen (BUN) in mg/dL; Albumin (Alb) in g/dL;Serum creatinine (SCr) in mg/dL; Age in years. Glomerular filtration rate (GFR) was calculated based upon serum creatinine (SCr) using the Modification of Diet in Renal Disease (MDRD) formula as suggested by Levey et al: MDRD GFR = 170 x [SCr/0.95]^(-0.999) x [Age]^(-0.176) x [0.762 if participant was female] x [1.180 if participant was black] x [BUN]^(-0.170) x [Alb]^(+0.318). Intent to Treat (ITT) population is defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Months 3, 6 and 12 post transplantation
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Month 3 post transplantation(n=29,24,27) | Month 6 post transplantation(n=29,24,25) | Month 12 post transplantation(n=27, 23, 29) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 57.8 | 57.5 | 63.6 |
,Belatacept, Sirolimus | 60.5 | 58.7 | 61.8 |
,Tacrolimus, MMF | 51.2 | 51.7 | 54.0 |
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Mean (Standard Deviation) in Calculated Glomerular Filtration Rate (GFR) mL/Min/1.73m^2 at Months 24, 36 and 48 Post Transplantation - Intent to Treat Population in Long Term Extension
GFR was calculated based upon serum creatinine (SCr) using the Modification of Diet in Renal Disease (MDRD) formula as suggested by Levey et al: MDRD GFR = 170 x [SCr/0.95]^(-0.999) x [Age]^(-0.176) x [0.762 if participant was female] x [1.180 if participant was black] x [BUN]^(-0.170) x [Alb]^(+0.318). Age in years, Alb = Albumin in g/dL; SCr = in mg/dL; BUN =Blood urea nitrogen in mg/dL. Intent to Treat population is defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Months 24, 36 and 48 post transplantation
Intervention | mL/Min/1.73m^2 (Mean) |
---|
| Month 24 (N=27, 18, 27) | Month 36 (N=25, 16, 22) | Month 48 (N=18, 14, 15) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 60.6 | 62.8 | 59.6 |
,Belatacept, Sirolimus | 66.0 | 69.9 | 72.2 |
,Tacrolimus, MMF | 52.2 | 55.5 | 55.7 |
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Mean Change From Baseline (BL) to Month 12 Post Transplantation in Lipid Values - Intent to Treat Population
Baseline (BL) was value obtained day prior to transplantation. Lipid values measured in milligrams/deciliter (mg/dL) included: high density lipoprotein cholesterol (HDL-C), low density lipoprotein cholesterol (LDL-C), non-HDL cholesterol (non-HDL-C), total cholesterol (TC), triglycerides. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline to Month 12
Intervention | mg/dL (Mean) |
---|
| HDL-C change from BL to Month 12 | Non-HDL-C change from BL to Month 12 | LDL-C change from BL to Month 12 | Total cholesterol change from BL to Month 12 | Triglycerides change from BL to Month 12 |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 1.5 | 16.0 | 23.9 | 17.5 | -11.4 |
,Belatacept, Sirolimus | -3.7 | 16.1 | 25.0 | 12.5 | -1.1 |
,Tacrolimus, MMF | -0.5 | 20.5 | 34.0 | 20.0 | -14.2 |
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Mean Systolic, Diastolic and Arterial Blood Pressure at Baseline and Month 12 - Intent to Treat Population
Systolic, diastolic and mean arterial blood pressures were measured in millimeters of mercury (mm Hg). Baseline was defined as value obtained before transplantation. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline and 12 months post transplantation
Intervention | mm Hg (Mean) |
---|
| Systolic blood pressure at baseline | Systolic blood pressure at Month 12 | Diastolic blood pressure at baseline | Diastolic blood pressure at Month 12 | Mean Arterial pressure at baseline | Mean Arterial pressure at Month 12 |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 133.1 | 129.3 | 78.6 | 73.3 | 96.8 | 91.9 |
,Belatacept, Sirolimus | 126.9 | 131.0 | 72.3 | 75.1 | 90.5 | 93.7 |
,Tacrolimus, MMF | 141.8 | 138.2 | 75.3 | 77.6 | 97.5 | 97.8 |
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Number of Corticosteroid-free Participants at 6 and 12 Months Post Transplantation - Intent to Treat Population
Participants were said to be corticosteroid-free at Month 6 if they were not receiving corticosteroids for greater than (>) 7 consecutive days during Days 141 through Days 196, and at Month 12 if not receiving corticosteroids for > 7 days during Days 337 through 392. Intent to treat population included all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 through Month 12
Intervention | participants (Number) |
---|
| At 6 Months post transplantation | At 12 Months post transplantation |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 27 | 24 |
,Belatacept, Sirolimus | 23 | 20 |
,Tacrolimus, MMF | 28 | 28 |
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Number of Corticosteroid-free Participants at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
In the LTE, a participant was considered corticosteroid-free if they were not receiving corticosteroids for >7 consecutive days during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. (NCT00455013)
Timeframe: End of Month 12 to end of Long Term Extension (Year 4)
Intervention | participants (Number) |
---|
| Month 24 (N=27, 18, 27) | Month 36 (N=26, 16, 23) | Month 48 (N=19, 14, 16) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 22 | 22 | 16 |
,Belatacept, Sirolimus | 15 | 14 | 12 |
,Tacrolimus, MMF | 25 | 20 | 16 |
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Number of Participants Who Switched Between MMF and Sirolimus During Long Term Extension up to Study Completion
Long Term extension was the period from the end of Month 12 to the end of Month 48 post transplantation and the completion of the study 31 July 2012. At any time in the study, participants who were unable to tolerate MMF in the Bela-MMF and Tac-MMF groups could discontinue (DC) MMF and switch to sirolimus and remain in the study and those in the Bela-Siro group who were unable to tolerate sirolimus could DC sirolimus and switch to MMF and remain in the study. Study completion=data base (DB) lock. (NCT00455013)
Timeframe: End of Month 12 to end of Study (Month 48)
Intervention | participants (Number) |
---|
| By Month 24 Switched between MMF and Sirolimus | By Month 36 Switched between MMF and Sirolimus | By Month 48 Switched between MMF and Sirolimus | Up to DB Lock Switched between MMF and Sirolimus |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 0 | 0 | 0 | 0 |
,Belatacept, Sirolimus | 1 | 2 | 3 | 3 |
,Tacrolimus, MMF | 0 | 0 | 0 | 0 |
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Number of Participants Who Used Anti-hypertension Medications at Baseline and at 12 Months Post Transplantation - Intent to Treat Population
Baseline was defined as day prior to transplantation. Number of anti-hypertension medications taken were categorized from 1 to 6 and greater than (>)6. Intent to treat population included all participants randomized and transplanted. (NCT00455013)
Timeframe: Baseline and Month 12
Intervention | participants (Number) |
---|
| Total using at least 1 medication at baseline | Total using at least 1 medication at Month 12 | Participants using 1 medication at baseline | Participants using 1 medication at Month 12 | Participants using 2 medications at baseline | Participants using 2 medications at Month 12 | Participants using 3 medications at baseline | Participants using 3 medications at Month 12 | Participants using 4 medications at baseline | Participants using 4 medications at Month 12 | Participants using 5 medications at baseline | Participants using 5 medications at Month 12 | Participants using 6 medications at baseline | Participants using 6 medications at Month 12 | Participants using >6 medications at baseline | Participants using >6 medications at Month 12 |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 31 | 25 | 10 | 11 | 5 | 6 | 8 | 6 | 3 | 1 | 3 | 0 | 2 | 1 | 0 | 0 |
,Belatacept, Sirolimus | 24 | 20 | 9 | 5 | 5 | 8 | 5 | 5 | 2 | 2 | 1 | 0 | 2 | 0 | 0 | 0 |
,Tacrolimus, MMF | 28 | 20 | 5 | 4 | 8 | 13 | 8 | 0 | 3 | 3 | 2 | 0 | 1 | 0 | 1 | 0 |
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Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Participants were considered corticosteroid-free at Months 24, 36, and 48 if they were not receiving corticosteroids for >7 consecutive days during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. Participants were considered CNI-free at Months 24, 36, and 48 if they were not receiving CNI during Day 701 and Day 756, Day 1065 and Day 1120, as well as Day 1429 and Day 1484, respectively. Participants in the tacrolimus arm were not relevant to this analysis because tacrolimus is a calcinurin inhibitor. (NCT00455013)
Timeframe: Months 24, 36, 48
Intervention | participants (Number) |
---|
| Month 24 (N=27, 18, 27) | Month 36 (N=26, 16, 23) | Month 48 (N=19, 14, 16) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 22 | 22 | 16 |
,Belatacept, Sirolimus | 15 | 14 | 12 |
,Tacrolimus, MMF | 0 | 0 | 0 |
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Number of Participants Who Were Corticosteroid-free at Months 6 and 12 and Number of Participants Who Were Both Calcineurin Inhibitor-free (CNI-free)and Corticosteroid-free at Months 6 and 12 Post Transplantation - Intent to Treat Population
Participants were said to be CNI-free at Month 6 or 12 if they were not receiving a CNI during Day 141 to Day 196, or Day 337 to Day 392. Participants in the tacrolimus arm were not relevant to this analysis because tacrolimus is a calcinurin inhibitor. Participants were corticosteroid-free (CS-free) at Month 6 if they were not receiving corticosteroids for > 7 consecutive days during Days 141 through Days 196, and at Month 12 if not receiving corticosteroids for > 7 days during Days 337 through 392. Day 1 was day of transplantation. Intent to treat population included all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 to Month 12 post transplantation
Intervention | participants (Number) |
---|
| CS-free Month 6 (N=33, 26, 30) | CS-free Month 12 (N=32, 26, 30) | CNI-free + CS-free Month 6 (N=32, 26, 30) | CNI -free + CS-free Month 12 (N=32,26,30) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 27 | 24 | 25 | 24 |
,Belatacept, Sirolimus | 23 | 20 | 19 | 18 |
,Tacrolimus, MMF | 28 | 28 | 1 | 1 |
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Number of Participants With Acute Rejection (AR) of Transplant up to 6 Months Post Transplantation - Intent to Treat (ITT) Population
AR is clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) greater than or equal to 25% from baseline plus one or more of the following: unexplained decreased urine output; fever, graft tenderness; SCr that remained elevated 14 days post-transplantation and clinical suspicion of AR; other reason and participant treated for episode. Day 1=transplantation. Banff 97 working classification of kidney transplant pathology: Type I=tubulointerstitial AR without arteritis (IA: interstitial infiltration with >25% of parenchyma affected and moderate tubulitis with >4 mononuclear cells/tubular cross section; IB: >10 mononuclear cells; Type II vascular AR with (IA) intimal arteritis (IIA=mild - moderate; IIB=severe; Type III=severe rejection with transmural arterial changes, necrosis of smooth muscle cells. (NCT00455013)
Timeframe: Day 1 to Month 6 post-transplantation
Intervention | participants (Number) |
---|
| Total Number of Participants with AR | Mild Acute IA | Mild Acute IB | Moderate Acute IIA | Moderate Acute IIB |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 4 | 0 | 0 | 2 | 1 |
,Belatacept, Sirolimus | 1 | 0 | 0 | 0 | 1 |
,Tacrolimus, MMF | 1 | 0 | 0 | 1 | 0 |
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Number of Participants With Acute Rejection of Transplant up to Month 12 Post Transplantation - Intent to Treat Population
AR defined as a clinicopathological event requiring clinical evidence and biopsy confirmation by central pathologist. One or more conditions were met and a renal biopsy revealed histologic evidence of rejection: unexplained rise of serum creatine (SCr) >= 25 % from baseline plus one or more of the following: unexplained decreased urine output; fever and graft tenderness; a SCr that remained elevated within 14 days after transplantation and clinical suspicion of AR; reason other than those listed and participant was treated for this episode. Day 1 was day of transplantation. Banff grade used Banff 97 working classification of kidney transplant pathology. ITT population was all randomized and transplanted participants. (NCT00455013)
Timeframe: Day 1 to Month 12 post transplantation
Intervention | participants (Number) |
---|
| Total number of participants with AR | Mild Acute IA | Mild Acute IB | Moderate Acute IIA | Moderate Acute IIB |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 5 | 0 | 0 | 3 | 2 |
,Belatacept, Sirolimus | 1 | 0 | 0 | 0 | 1 |
,Tacrolimus, MMF | 1 | 0 | 0 | 1 | 0 |
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Number of Participants With Graft Loss or Death at Months 24, 36, 48 Post Transplantation - Intent to Treat Population in Long Term Extension
Graft loss was defined as either functional loss or physical loss. Functional loss was defined as either: sustained level of SCr greater than or equal to (>=) 6.0 mg/dL (530 micromoles/Liter; micromol/L) for >= 4 weeks as determined by the local laboratory; regularly scheduled dialysis treatments over a period of 56 days; impairment of renal function to such a degree that the participant undergoes re-transplant. Day 1 was day of transplantation. ITT population defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: End of Month 12 to end of Long Term Extension (Year 4)
Intervention | participants (Number) |
---|
| Graft Loss or Death at Month 24 (N=27,19,27) | Graft Loss or Death at Month 36 (N=27,19,27) | Graft Loss or Death at Month 48 (N=27,19,27) |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 0 | 1 | 1 |
,Belatacept, Sirolimus | 1 | 2 | 2 |
,Tacrolimus, MMF | 0 | 0 | 0 |
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Number of Participants With Graft Loss or Death up to Month 6 and Month 12 Post Transplantation - Intent to Treat Population
Graft loss was defined as either functional loss or physical loss. Functional loss was defined as either: sustained level of SCr greater than or equal to (>=) 6.0 mg/dL (530 micromoles/Liter; micromol/L) for >= 4 weeks as determined by the local laboratory; regularly scheduled dialysis treatments over a period of 56 days; impairment of renal function to such a degree that the participant undergoes re-transplant. Day 1 was day of transplantation. ITT population defined as all participants randomized and transplanted. (NCT00455013)
Timeframe: Day 1 to Month 6 and Month 12 post transplantation
Intervention | participants (Number) |
---|
| Graft loss up to Month 6 post transplantation | Graft loss up to Month 12 post transplantation | Death up to Month 6 post transplantation | Death up to Month 12 post transplantation |
---|
Belatacept, Mycophenolate Mofetil (MMF) | 1 | 2 | 1 | 1 |
,Belatacept, Sirolimus | 1 | 2 | 0 | 0 |
,Tacrolimus, MMF | 0 | 0 | 0 | 0 |
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Change in Forced Expiratory Volume in 1 Second (FEV1) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup
Forced expiratory volume in 1 second (FEV1) was measured by spirometry conducted according to internationally accepted standards. FEV1 is the volume delivered in the first second of a forced vital capacity (FVC) maneuver. A positive change score indicates improved lung function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | Liters (Mean) |
---|
Everolimus + CNI Reduction | -0.2 |
Control | -0.1 |
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Change in Forced Vital Capacity (FVC) From Baseline to End of Study (Month 24) in the Lung Transplant Subgroup
Forced vital capacity (FVC) was measured by spirometry conducted according to internationally accepted standards. FVC is the volume delivered during an expiration made as forcefully and completely as possible starting from full inspiration. A positive change score indicates improved lung function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | Liters (Mean) |
---|
Everolimus + CNI Reduction | -0.2 |
Control | -0.1 |
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Mean Days of Hospitalization From Baseline to End of Study (Month 24)
(NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | Days (Mean) |
---|
Everolimus + CNI Reduction | 8.5 |
Control | 16.2 |
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Number of Patients in Need of Dialysis From Month 12 to End of Study (Month 24)
(NCT00377962)
Timeframe: Month 12 to end of study (Month 24)
Intervention | Participants (Number) |
---|
Everolimus + CNI Reduction | 0 |
Control | 2 |
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Number of Patients With Biopsy-proven Acute Rejection From Month 12 to End of Study (Month 24)
Biopsy-proved acute rejection was defined as a treated acute rejection confirmed by biopsy, graded locally according to the International Society for Heart & Lung Transplantation (ISHLT) criteria. A treated acute rejection was defined as an acute rejection clinically suspected, whether biopsy-proven or not, which had been treated and confirmed by the investigator according to the response to therapy. (NCT00377962)
Timeframe: Month 12 to end of study (Month 24)
Intervention | Participants (Number) |
---|
Everolimus + CNI Reduction | 6 |
Control | 5 |
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Change in Left Ventricular Function (Diameter and Thickness Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup
Left ventricular function was assessed by echocardiography which was performed according to local routine practice. Echocardiography parameters were left ventricular end diastolic diameter (LVEDD), left ventricular end systolic diameter (LVESD), interventricular septal wall thickness (IVSTd), and posterior wall thickness (PWTd). A positive change score indicates improved left ventricular function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | cm (Mean) |
---|
| LVEDD | LVESD | IVSTd | PWTd |
---|
Control | -0.0 | 0.1 | -0.1 | -0.1 |
,Everolimus + CNI Reduction | -0.1 | 0.1 | -0.4 | -0.5 |
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Change in Left Ventricular Function (Filling and Ejection Fraction Parameters) From Baseline to End of Study (Month 24) in the Heart Transplant Subgroup
Left ventricular function was assessed by echocardiography which was performed according to local routine practice. Echocardiography parameters were filling fraction (FF) and ejection fraction (EF). A positive change score indicates improved left ventricular function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | percentage (Mean) |
---|
| EF | FF |
---|
Control | 0.1 | 0 |
,Everolimus + CNI Reduction | -0.6 | 0 |
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Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to End of Study (Month 24)
Renal function was assessed by determining the measured glomerular filtration rate (mGFR) using creatinine ethylenediamine tetraacetic acid (Cr-EDTA) clearance or an equivalent method. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | mL/min (Mean) |
---|
| Month 0 | Month 24 | Change |
---|
Control | 49.1 | 46.8 | -2.4 |
,Everolimus + CNI Reduction | 49.3 | 52.5 | 3.2 |
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Change in Measured Glomerular Filtration Rate (mGFR) From Baseline to Month 12
Renal function was assessed by determining the measured glomerular filtration rate (mGFR) using creatinine ethylenediamine tetraacetic acid (Cr-EDTA) clearance or an equivalent method. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to Month 12
Intervention | mL/min (Mean) |
---|
| Baseline | Month 12 | Change from Baseline |
---|
Control | 48.0 | 47.5 | -0.5 |
,Everolimus + CNI Reduction | 48.6 | 53.2 | 4.6 |
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Change in Serum Creatinine From Baseline to End of Study (Month 24)
Renal function was assessed by determining serum creatinine using standard laboratory methods. A positive change score indicates improved renal function. (NCT00377962)
Timeframe: Baseline to end of study (Month 24)
Intervention | μmol/L (Mean) |
---|
| Month 0 | Month 24 | Change |
---|
Control | 129 | 132 | 3 |
,Everolimus + CNI Reduction | 126 | 126 | 0 |
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Number of Patients Discontinued From the Study Due to Adverse Events From Month 12 to End of Study (Month 24)
(NCT00377962)
Timeframe: Month 12 to end of study (Month 24)
Intervention | Participants (Number) |
---|
| Total discontinued due to AE(s) | Pulmonary embolism | Skin problems | Hypercholesterolemia | Stroke | Muscular pain | Diarrhea | Edema |
---|
Control | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
,Everolimus + CNI Reduction | 8 | 2 | 1 | 1 | 1 | 1 | 1 | 1 |
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Number of Patients Who Died and Number of Patients With Graft Loss From Month 12 to End of Study (Month 24)
Number of patients not alive and number of patients with loss of their graft. (NCT00377962)
Timeframe: Month 12 to end of study (Month 24)
Intervention | Participants (Number) |
---|
| Death | Graft Loss |
---|
Control | 0 | 0 |
,Everolimus + CNI Reduction | 3 | 0 |
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Graft Survival at Three Years Post-Transplant
"Number of participants that did not experience kidney graft failure[1] at three years post-transplant~[1]Graft failure is defined as the institution of chronic dialysis (at least 6 consecutive weeks, excluding participants with delayed graft function), transplant nephrectomy, or retransplantation." (NCT00183248)
Timeframe: Three years post kidney transplant
Intervention | participants (Number) |
---|
DBMCs | 4 |
Control Group | 5 |
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Number of Chronic Allograft Nephropathies
"Number of chronic allograft nephropathies[1,2,3] at 3 years post kidney transplant.~Chronic allograft nephropathy is defined as renal biopsies with Banff 97 Grade I or greater[2] with higher numeric scores indicating more severe nephropathy~The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification[3]~Reference: Racusen LC, Solez K, Colvin RB et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00183248)
Timeframe: Three years post kidney transplant
Intervention | Nephropathy Events (Number) |
---|
DBMCs | 6 |
Control Group | 2 |
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Number of Graft-versus-host Disease (GVHD) Events
A disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Symptoms include jaundice, skin rash or blisters, a dry mouth, or dry eyes. Also called graft-versus-host disease. (NCT00183248)
Timeframe: Three years post kidney transplant
Intervention | GVHD Events (Number) |
---|
DBMCs | 0 |
Control Group | 0 |
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Number of Kidney Biopsy-proven Acute Rejection
"Biopsy-proven acute renal (kidney) rejection[1,2].~Diagnosis of acute rejection was made by renal biopsy using the Banff 97 criteria. The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification. Acute rejection is defined by a renal biopsy demonstrating a Banff 97 classification of Grade IA or greater, with higher scores indicating more severe rejection[2]~Ref: Racusen LC et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00183248)
Timeframe: Three years post kidney transplant
Intervention | Rejection Events (Number) |
---|
DBMCs | 1 |
Control Group | 1 |
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Overall Kidney Graft Survival at One Year Post-Transplant
"Number of participants that did not experience kidney graft failure[1] at one year post-transplant~[1]Graft failure is defined as the institution of chronic dialysis (at least 6 consecutive weeks, excluding participants with delayed graft function), transplant nephrectomy, or retransplantation." (NCT00183248)
Timeframe: One year post kidney transplant
Intervention | participants (Number) |
---|
DBMCs | 4 |
Control Group | 5 |
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Overall Participant Survival at One Year Post Kidney Transplant
(NCT00183248)
Timeframe: One year post kidney transplant
Intervention | participants (Number) |
---|
DBMCs | 4 |
Control Group | 5 |
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Participant Survival at Three Years Post Kidney Transplant
(NCT00183248)
Timeframe: Three years post kidney transplant
Intervention | participants (Number) |
---|
DBMCs | 4 |
Control Group | 5 |
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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
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 72 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 48 |
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Incidence of Grades II-IV Acute GVHD
Percentage patients with grades II-IV GHVD, estimated by cumulative incidence methods (NCT00075478)
Timeframe: 120 days after transplant
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 46 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 32 |
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Incidence of Graft Rejection
Donor CD3 chimerism less than 5% (NCT00075478)
Timeframe: 1 year after transplant
Intervention | participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 0 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 2 |
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Incidence of Non-relapse Mortality
Percentage of NRM as estimated by cumulative incidence methods with competing risks (NCT00075478)
Timeframe: 3 years after transplant
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 7 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 9 |
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Incidence of Relapse/Progression
Percentage of relapse estimated by cumulative incidence methods (NCT00075478)
Timeframe: 3 years after transplant
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 40 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 55 |
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Overall Survival
Percentage of patients surviving as estimated by Kaplan-Meier. (NCT00075478)
Timeframe: 3 years after transplant
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 65 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 54 |
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Progression-free Survival
Percentage of patients with progression-free survival, estimated by cumulative incidence methods (NCT00075478)
Timeframe: 3 years after transplant
Intervention | percentage of participants (Number) |
---|
Arm I (Chemotherapy, TBI, Transplant, GVHD Prophylaxis) | 53 |
Arm II (TBI, Transplant, GVHD Prophylaxis) | 36 |
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Duration (Days) Until Participants Obtained Platelet Engraftment
Summarized using a range and median of measure in days until participants reached platelet engraftment. Platelet engraftment was defined as the first of 7 consecutive days when the platelet count exceeded 20 x 109/L (untransfused). (NCT00796068)
Timeframe: At 6 months
Intervention | days (Median) |
---|
Arm I (Low Risk for Graft Failure) | 31 |
Arm II (High Risk for Graft Failure) | 31 |
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Incidence of Acute or Chronic Graft-versus-host Disease (GVHD)
Overall GVHD is determined based on the organ stage, response to treatment and whether GVHD was a major cause of death. Chronic GVHD will be defined according to the National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease. Described as mild, moderate, or severe as graded according to the attached Organ Scoring Sheet. Symptoms consistent with both chronic and acute GVHD occurring after day 100 will be considered overlap chronic GVHD syndrome. (NCT00796068)
Timeframe: At 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 7 |
Arm II (High Risk for Graft Failure) | 6 |
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Incidence of Clinically Significant Infections
Collected and graded according to the modified National Cancer Institute Common Toxicity Criteria. (NCT00796068)
Timeframe: At 6 months
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 53 |
Arm II (High Risk for Graft Failure) | 61 |
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Incidence of Relapse or Disease Progression
Cumulative incidence estimates using non-relapse mortality as competitive event. (NCT00796068)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 17 |
Arm II (High Risk for Graft Failure) | 11 |
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Non-relapse Mortality
Death of any cause other than relapse or disease progression was considered. (NCT00796068)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 8 |
Arm II (High Risk for Graft Failure) | 10 |
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Number of Participants Surviving by 1 Year
Overall survival was measured from the first day of CBT infusion until death from any cause. (NCT00796068)
Timeframe: At 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 51 |
Arm II (High Risk for Graft Failure) | 47 |
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Number of Participants Surviving up to 2 Years Without Disease Progression
Progression-free survival is the time interval from start of treatment to documented evidence of disease progression. Disease progression was defined by European LeukemiaNet 2017 criteria and International Working Group (IWG) within 3 years of transplant. (NCT00796068)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 37 |
Arm II (High Risk for Graft Failure) | 41 |
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Number of Participants With Acute Graft-versus-host Disease (GVHD) Grade II-IV by Day 100
Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. Diagnosing and grading acute GVHD is based on clinical findings (the organ stage, response to treatment and whether GVHD was a major cause of death) and frequently varies between transplant centers and independent reviewers. (NCT00796068)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 36 |
Arm II (High Risk for Graft Failure) | 37 |
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Number of Participants With Graft Failure/Rejection
"Patients will be considered primary graft failure provided they meet any criteria listed below, in the absence of documented disease persistence/recurrence or active bone marrow infection (ex. Cytomegalovirus (CMV)):~i. Absence of 3 consecutive days with neutrophils >500/u1 combined with host CD3+ peripheral blood chimerism ≥50% at day 42 ii. Absence of 3 consecutive days with neutrophils >500/u1 under any circumstances at day 55 iii. Death after day 28 with neutrophil count <100/u1 without any evidence of engraftment (< 5% donor CD3+) iv. Primary autologous count recovery with < 5% donor CD3+ peripheral blood chimerism at count recovery and without relapse" (NCT00796068)
Timeframe: Up to 100 days
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 2 |
Arm II (High Risk for Graft Failure) | 5 |
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Number of Participants With Secondary Graft Failure
Secondary graft failure is defined as decline of neutrophil count to <500/ul with loss of donor chimerism after day 55 (NCT00796068)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 0 |
Arm II (High Risk for Graft Failure) | 0 |
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Number of Patients With Non-relapse Mortality (NRM)
Defined as death from any cause without sign of disease progression or relapse. Loss to follow up are censored, whereas disease relapse or progression are considered competing risks. (NCT00796068)
Timeframe: At day -200
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 7 |
Arm II (High Risk for Graft Failure) | 6 |
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The Number of Participants Alive at Two-years Follow up.
Overall survival of participants after two-years of follow up. (NCT00796068)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Low Risk for Graft Failure) | 50 |
Arm II (High Risk for Graft Failure) | 44 |
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Change in SLICC/ACR Damage Index From Baseline During Short-term Period
SLICC/ACR score or damage index is a measure of cumulative damage due to Systemic Lupus Erythematosus (SLE). Damage is defined as non-reversible change (not related to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months. A score of 0=no damage, early damage is defined as ≥1. The total maximum score is 48, and increasing score indicates increasing disease severity. Change from baseline=Postbaseline - baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Postbaseline (Month 12 or 28 days after last dose)
Intervention | Units on a scale (Mean) |
---|
Abatacept 30/10 mg/kg | 0.17 |
Abatacept 10/10 mg/kg | 0.11 |
Placebo | 0.13 |
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Number of Months CRR Was Maintained During Short-term Period
Durability of CRR, defined as the number of months (number of consecutive planned visits beyond Day 15) a participant met the definition of CRR during the double-blind treatment period. Refer to outcome 1 for description of CRR. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 Months
Intervention | Months (Median) |
---|
Abatacept 30/10 mg/kg | 0 |
Abatacept 10/10 mg/kg | 0 |
Placebo | 0 |
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Number of Participants Achieving Renal Response (RR) at Month 12 During Short-term Period
RR is defined as meeting BOTH of the following criteria:RENAL FUNCTION: Less than or equal to 25% increase from baseline;PROTEINURIA: Greater than or equal to 50% improvement in the urine protein/creatinine ratio with one of the following - urine protein/creatinine ratio (UPCR) <113 mg/mmol,, if the baseline ratio was <=339 mg/mmol OR UPCR <339 mg/mmol,if the baseline ratio > 339 mg/mmol. A participant was considered as achieving RR if response criteria at both months 11 and 12 (Days 337 and 365, respectively) were met. For 95% CI within each group, normal approximation is used if n>=5. (NCT00430677)
Timeframe: Month 12
Intervention | Participants (Number) |
---|
Abatacept 30/10 mg/kg | 45 |
Abatacept 10/10 mg/kg | 39 |
Placebo | 33 |
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Number of Participants With a Treatment-emergent Seropositive Result During the Long-term Extension Period
Collected in at least 1 sample. Assessment includes immunogenicity (detection of serum antibodies which bind to CTLA4-Ig in the in vitro assays) and exposure to corticosteroids (NCT00430677)
Timeframe: Day 365 to end of long-term extension period
Intervention | Participants (Number) |
---|
Abatacept 10 mg/kg | 17 |
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Number of Participants With Confirmed Complete Renal Response (CRR) During Short-term Period
Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: Day 1 to 12 months
Intervention | Participants (Number) |
---|
Abatacept 30/10 mg/kg | 22 |
Abatacept 10/10 mg/kg | 27 |
Placebo | 20 |
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Participants Achieving a Confirmed Complete Renal Response (CRR) at Month 12 During Short-term Period
Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: At Month 12 from Day 1
Intervention | Participants (Number) |
---|
Abatacept 30/10 mg/kg | 9 |
Abatacept 10/10 mg/kg | 11 |
Placebo | 8 |
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Participants Achieving Renal Improvement (RI) or CRR at Month 12 During Short-term Period
CRR defined as meeting all of 5 criteria. RF: (Glomerular filtration rate [GFR] calculated using MDRD equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: At Month 12 from Day 1
Intervention | Participants (Number) |
---|
Abatacept 30/10 mg/kg | 38 |
Abatacept 10/10 mg/kg | 37 |
Placebo | 31 |
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Time to Achieve First Confirmed Renal Improvement (RI) During Short-term Period (as Determined by Kaplan-Meier Methodology)
RI is defined as meeting all of the following criteria. Renal function: If MDRD is abnormal at screening, within 10% of the MDRD at screening; if MDRD is 60-89 at screening, greater than or equal to 50% improvement based on the screening value or 90% or greater of MDRD at screening; if MDRD is 15-59 at screening, if MDRD is normal at screening-within 10% of the MDRD at screening. Proteinuria: improvement greater than or equal to 50% from screening. Hematuria: red blood cell (RBC)count within normal limit of central laboratory. Pyuria: white blood cell (WBC) count within normal limit of central laboratory. Cylindruria: No RBC or WBC casts. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 months.
Intervention | Days (Median) |
---|
Abatacept 30/10 mg/kg | 141 |
Abatacept 10/10 mg/kg | 136 |
Placebo | 144 |
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Time to First Confirmed Complete Renal Response (CRR) During the Short-term (Double-blind) Period
Confirmed at 2 consecutive visits. CRR defined as meeting all of 5 criteria. Renal function (RF): (Glomerular filtration rate [GFR] calculated using Modification of Diet in Renal Diseases equation equation) Calculated function abnormal at screening visit - return of renal function to greater than or equal to 90% of function at approximately 6 months prior to onset of the current episode of lupus nephritis. Calculated function normal at screening visit - estimated renal function 90% or greater of level at screening visit. Proteinuria: urinary protein/creatinine ratio <30 mg/mmol. Hematuria: red blood cell (RBC) count within normal limits of Central Laboratory. Pyuria: White blood cell count (WBC) within normal limits of Central Laboratory. Cylindruria: No RBC or WBC casts reported. (NCT00430677)
Timeframe: Day 1 (randomization) to 12 months.
Intervention | days (Number) |
---|
Abatacept 30/10 mg/kg | NA |
Abatacept 10/10 mg/kg | NA |
Placebo | NA |
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Baseline and Post Baseline Systemic Lupus International Collaborating Clinics (SLICC)/American College of Rheumatology (ACR) Damage Index During Short-term Period
SLICC/ACR score or damage index is a measure of cumulative damage due to Systemic Lupus Erythematosus (SLE). Damage is defined as nonreversible change (not related to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months. A score of 0=no damage, early damage is defined as ≥1. The total maximum score is 48, and increasing score indicates increasing disease severity. (NCT00430677)
Timeframe: Baseline (Day 1), Post baseline (Month 12 or 28 days after last dose)
Intervention | Units on a scale (Mean) |
---|
| Baseline (n=68, 67, 70) | Post Baseline Mean (n=68, 67, 70) |
---|
Abatacept 10/10 mg/kg | 0.27 | 0.40 |
,Abatacept 30/10 mg/kg | 0.34 | 0.53 |
,Placebo | 0.29 | 0.44 |
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Baseline Fatigue as Measured by Fatigue Severity Scale-Krupp During Short-term Period
The reduction of fatigue assessed by Fatigue Severity Scale (FSS). The FSS questionnaire is comprised of 9 statements inquiring about the examinee's sleep habits over the preceding week. Participants are asked to rate their level of agreement (toward seven) or disagreement (toward zero) with the nine statements. A score of 36 and above (out of a maximum of 63) indicates the presence of significant fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Baseline (Day 1) for Day 85 (n=90, 94, 95) | Baseline (Day 1) for Day 169 (n=92, 94, 97) | Baseline (Day 1) for Day 253 ( n=92, 94, 97) | Baseline (Day 1) for Day 365 ( n=92, 94, 97) |
---|
Abatacept 10/10 mg/kg | 39.14 | 39.14 | 39.14 | 39.14 |
,Abatacept 30/10 mg/kg | 40.60 | 40.41 | 40.41 | 40.41 |
,Placebo | 39.64 | 39.59 | 39.59 | 39.59 |
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Baseline Fatigue as Measured by the Fatigue Visual Analog Scale During Short-term Period
A visual analogue scale (VAS) is a psychometric response scale for measurement of subjective characteristics or attitudes that cannot be directly measured. The VAS for Fatigue (VAS-F) consists of a 100 mm line, with 0 (No Fatigue) on 1 end and 100 (Extreme Fatigue) on the other end, which a participant marks to indicate how much fatigue he or she feels. The marked point in mm is converted into a numeric value from 0 to 100, where 0=no fatigue and 100=maximum fatigue. Increasing numbers=increasing fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Baseline (Day 1) for Day 85 (n=90, 94, 95) | Baseline (Day 1) for Day 169 (n=92, 94, 97) | Baseline (Day 1) for Day 253 (n=92, 94, 97) | Baseline (Day 1) for Day 365 (n=92, 94, 97) |
---|
Abatacept 10/10 mg/kg | 41.95 | 41.95 | 41.95 | 41.95 |
,Abatacept 30/10 mg/kg | 48.86 | 48.80 | 48.80 | 48.80 |
,Placebo | 48.93 | 48.25 | 48.25 | 48.25 |
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Baseline Mental Component Summary of the Short SF-36 During Short-term Period
The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Baseline (Day 1) for Day 85 ( n=89, 91, 93) | Baseline (Day 1) for Day 169 (n=92,94,96) | Baseline (Day 1) for Day 253 ( n=92,94,96) | Baseline (Day 1) for Day 365 ( n=92,94,96) |
---|
Abatacept 10/10 mg/kg | 43.80 | 41.84 | 41.84 | 41.84 |
,Abatacept 30/10 mg/kg | 42.18 | 44.08 | 44.08 | 44.08 |
,Placebo | 42.68 | 42.59 | 42.59 | 42.59 |
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Baseline Quantitative Immunoglobulins During the Short-term Period
A quantitative immunoglobulins (Igs) test is used to detect abnormal levels of the three major classes of Igs (IgG, IgA, and IgM). Abnormal test results typically indicate that there is something affecting the immune system which requires further testing. (NCT00430677)
Timeframe: Baseline (Day 1)
Intervention | mg/dL (Mean) |
---|
| Immunoglobulin IgA | Immunoglobulin IgG | Immunoglobulin IgM |
---|
Abatacept 10/10 mg/kg | 218.04 | 864.12 | 97.10 |
,Abatacept 30/10 mg/kg | 246.28 | 939.80 | 100.96 |
,Placebo | 230.23 | 1013.17 | 98.49 |
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Baseline Renal Function Over Time During Short-term Period
Baseline (BL) renal function, as estimated by calculation of the MDRD (Modification of Diet in Renal Disease) equation, over time. Renal MDRD is an equation (calculation) used to estimate Glomerular Filtration Rate (GFR) in participants with impaired renal function based on serum creatinine, age, race, and gender. GFR (mL/min/1.73 m^2) = 175 * (Scr)^-1.154 * (Age)^-0.203 * (0.742 if female) * (1.212 if African American) (conventional units). mL, milliliters; min, minute; m^2, meters squared; Scr, serum creatinine. A negative value indicates worsening. (NCT00430677)
Timeframe: Baseline (Day 1), Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365
Intervention | milliliters per minute (mL/min)/1.73 m^2 (Mean) |
---|
| Baseline (Day 1) for Day 15 (n=98,94,98) | Baseline (Day 1) for Day 29 (n=98,95,98) | Baseline (Day 1) for Day 57 (n=94, 89, 96) | Baseline (Day 1) for Day 85 (n=90, 91, 93) | Baseline (Day 1) for Day 113 (n=91, 83, 91) | Baseline (Day 1) for Day 141 (n=89, 83, 90) | Baseline (Day 1) for Day 169 (n=83, 82, 85) | Baseline (Day 1) for Day 197 (n=84, 81, 87) | Baseline (Day 1) for Day 225 (n=84, 81, 84) | Baseline (Day 1) for Day 253 (n=81, 76, 81) | Baseline (Day 1) for Day 281 (n=78, 77, 80) | Baseline (Day 1) for Day 309 (n=77, 76, 78) | Baseline (Day 1) for Day 337 (n=74, 75, 79) | Baseline (Day 1) for Day 365 (n=75, 73, 78) |
---|
Abatacept 10/10 mg/kg | 99.09 | 98.56 | 98.76 | 100.22 | 101.23 | 101.05 | 101.95 | 101.85 | 101.64 | 101.25 | 102.64 | 100.64 | 101.00 | 101.30 |
,Abatacept 30/10 mg/kg | 92.57 | 93.08 | 91.54 | 94.53 | 94.43 | 93.62 | 94.83 | 94.55 | 94.87 | 94.89 | 94.17 | 94.74 | 95.51 | 95.04 |
,Placebo | 91.01 | 91.23 | 91.58 | 92.76 | 92.41 | 92.83 | 92.56 | 92.69 | 93.25 | 93.38 | 93.13 | 92.71 | 92.90 | 93.03 |
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Change From Baseline in Mental Component Summary of the SF-36 During Short-term Period
The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Day 85 (n=89, 91, 93) | Day 169 (n=92, 94, 96) | Day 253 (n=92, 94, 96) | Day 365 (n=92, 94, 96) |
---|
Abatacept 10/10 mg/kg | 3.10 | 5.08 | 4.83 | 4.23 |
,Abatacept 30/10 mg/kg | 1.07 | 2.90 | 2.45 | 2.62 |
,Placebo | 1.87 | 3.69 | 2.99 | 2.84 |
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Change From Baseline in Physical Component Summary of the SF-36 During Short-term Period
The SF-36 is a validated instrument measuring health-related quality of life across multiple disease states. It has 36 questions with 8 subscale scores and 2 summary scores (1) physical component summary=physical functioning, role-physical, bodily pain, and general health; (2) mental component summary=vitality, social functioning, role-emotional, and mental health. There is no total overall score; scoring is done for both subscores and summary scores. For subscores and summary scores, 0 =worst score (or quality of life) and 100=best score. Change from Baseline= post-Baseline - Baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Day 85 (n=89, 91, 93) | Day 169 (n=92, 94, 96) | Day 253 (n=92, 94, 96) | Day 365 (n=92, 94, 96) |
---|
Abatacept 10/10 mg/kg | 2.61 | 4.07 | 4.80 | 5.00 |
,Abatacept 30/10 mg/kg | 4.17 | 4.18 | 4.23 | 4.24 |
,Placebo | 2.86 | 3.39 | 3.45 | 3.77 |
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Change in Fatigue From Baseline as Measured by Fatigue Severity Scale-Krupp During Short-term Period
The reduction of fatigue assessed by Fatigue Severity Scale (FSS). The FSS questionnaire is comprised of 9 statements inquiring about the examinee's sleep habits over the preceding week. Participants are asked to rate their level of agreement (toward seven) or disagreement (toward zero) with the nine statements. A score of 36 and above (out of a maximum of 63) indicates the presence of significant fatigue. (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Least Squares Mean) |
---|
| Day 85 (n=90, 94, 95) | Day 169 (n=92, 94, 97) | Day 253 ( n=92, 94, 97) | Day 365 ( n=92, 94, 97) |
---|
Abatacept 10/10 mg/kg | -1.40 | -1.69 | -2.95 | -3.21 |
,Abatacept 30/10 mg/kg | -1.54 | -2.68 | -3.54 | -4.20 |
,Placebo | -0.67 | -1.08 | -3.06 | -4.79 |
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Change in Fatigue From Baseline as Measured by the Fatigue Visual Analog Scale During Short-term Period
"A visual analogue scale is a psychometric response scale for measurement of subjective characteristics or attitudes that cannot be directly measured.~The VAS for Fatigue (VAS-F) consists of a 100 mm line, with 0 (No Fatigue) on 1 end and 100 (Extreme Fatigue) on the other end, which a participant marks to indicate how much fatigue he or she feels. The marked point in mm is converted into a numeric value from 0 to 100, where 0=no fatigue and 100=maximum fatigue. Increasing numbers=increasing fatigue." (NCT00430677)
Timeframe: Baseline (Day 1), Days 85, 169, 253, and 365
Intervention | Units on a scale (Mean) |
---|
| Day 85 (n=90, 94, 95) | Day 169 (n=92, 94, 97) | Day 253 (n=92, 94, 97) | Day 365 (n=92, 94, 97) |
---|
Abatacept 10/10 mg/kg | -4.94 | -9.52 | -11.90 | -12.32 |
,Abatacept 30/10 mg/kg | -9.18 | -7.18 | -8.78 | -12.21 |
,Placebo | -6.20 | -4.71 | -7.35 | -11.07 |
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Change in Quantitative Immunoglobulin From Baseline During Short-term Period
"A quantitative immunoglobulin (Ig) test is used to detect abnormal levels of the 3 major classes of Ig (IgG, IgA, and IgM). Abnormal test results typically indicate that something is affecting the immune system and further testing is required.~Please refer to Outcome 31 for the respective baseline values" (NCT00430677)
Timeframe: Day 365
Intervention | mg/dL (Mean) |
---|
| Ig A (n=76, 73, 78) | IgG (n=76, 73, 78) | IgM (n=76, 73, 78) |
---|
Abatacept 10/10 mg/kg | -34.48 | 27.21 | -19.38 |
,Abatacept 30/10 mg/kg | -32.83 | 41.41 | -17.76 |
,Placebo | -26.51 | 23.42 | -20.62 |
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Change in Renal Function From Baseline Over Time During Short-term Period
Mean change from baseline in renal function, as estimated by calculation of the MDRD equation, over time. Renal MDRD is an equation (calculation) used to estimate Glomerular Filtration Rate (GFR) in participants with impaired renal function based on serum creatinine, age, race, and gender. GFR (mL/min/1.73 m^2) = 175 * (Scr)^-1.154 * (Age)^-0.203 * (0.742 if female) * (1.212 if African American) (conventional units). mL, milliliters; min, minute; m^2, meters squared; Scr, serum creatinine. A positive value indicates improvement. Change from baseline=Post-baseline-baseline value. (NCT00430677)
Timeframe: Baseline (Day 1), Day 15, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365
Intervention | milliliters per minute (mL/min)/1.73 m^2 (Mean) |
---|
| Day 15; (n=98,94,98) | Day 29 (n=98, 95, 98) | Day 57 (n=94 89 96) | Day 85 (n=90, 91, 93) | Day 113 (n=91, 83, 91) | Day 141 (n=89, 83, 90) | Day 169 (n=83, 82, 85) | Day 197 (n=84, 81, 87) | Day 225 (n=84, 81, 84) | Day 253 (n=81, 76, 81) | Day 281 (n=78, 77, 80) | Day 309 (n=77, 76, 78) | Day 337 (n=74, 75, 79) | Day 365 (n=75, 73, 78) |
---|
Abatacept 10/10 mg/kg | 1.15 | 4.51 | 7.28 | 6.20 | 10.23 | 7.90 | 10.55 | 10.67 | 7.72 | 7.22 | 6.81 | 9.53 | 10.15 | 11.03 |
,Abatacept 30/10 mg/kg | -0.24 | 2.26 | 5.96 | 8.56 | 5.31 | 8.33 | 8.12 | 7.71 | 7.32 | 4.70 | 5.68 | 6.34 | 5.34 | 5.17 |
,Placebo | 0.62 | 1.70 | 2.38 | 2.89 | 4.12 | 3.62 | 7.34 | 6.79 | 7.19 | 5.86 | 5.38 | 6.00 | 4.39 | 5.68 |
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Mean Change From Baseline in SLICC/ACR Damage Index
SLICC=Systemic Lupus International Collaborating Clinics; ACR=American College of Rheumatology. The SLICC/ACR Damage Index measures organ damage (nonreversible change, unrelated to active inflammation) occurring since onset of lupus, ascertained by clinical assessment and present for at least 6 months unless otherwise stated. The index assesses 47 items in 12 systems: Ocular, Neuropsychiatric, Renal, Pulmonary, Cardiovascular, Gastrointestinal, Peripheral Vascular, Musculoskeletal, Skin, Premature Gonadal Failure, Diabetes, Malignancy. Scores range from 0 to 2, and the same lesion cannot be scored twice. If damage is noted for a particular item, it is scored 1. No damage is scored 0. Some items may score 2 points if they occur more than once, so that the maximum possible score is 47. Scores can only increase with time, but scores rarely reach over 12. It is usually completed (or updated) yearly. (NCT00430677)
Timeframe: Day 365 to termination of the long-term extension phase
Intervention | Units on a scale (Mean) |
---|
| Day 365 (n=69, 65, 74) | Day 729 (n=66, 65, 69) | Day 1093 (n=41, 38, 44) |
---|
Abatacept 10/10 mg/kg | -0.17 | -0.28 | -0.16 |
,Abatacept 30/10 mg/kg | -0.12 | -0.21 | -.27 |
,Placebo | -0.08 | -0.10 | 0.02 |
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Number of Participants Achieving Complete Response by ACCESS Definition
The Abatacept and Cyclophosphamide Combination Efficacy and Safety Study (ACCESS) defines complete response as a response meeting all of the following criteria: serum creatinine ≤upper limit of normal as defined by the central laboratory or ≤125% of the higher value at either screening or baseline; urine protein/creatinine ratio <50 mg/mmoL; and prednisone or prednisone-equivalent dose tapered to 10 mg per day. (NCT00430677)
Timeframe: End of short-term period (Day 365) to termination of the long-term extension period
Intervention | participants (Number) |
---|
| Day 365 | Day 645 (n=55, 56, 61) |
---|
Abatacept 10/10 mg/kg | 30 | 28 |
,Abatacept 30/10 mg/kg | 29 | 27 |
,Placebo | 25 | 25 |
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Number of Participants Achieving Patient Response (PR) at Month 12 During the Short-term Period
"PR is either CRR, Partial Renal Response(PRR),or no Response(NR).~CRR= Serum creatinine(SC)is normal, Inactive urinary sediment, No cellular casts, Urinary protein/creatinine (UPCR) ratio <56.5 mg/mmoL; PRR= SC is normal OR SC not >25% above BL, RBCs at reference range, UPCR <56.5 mg/mmoL OR ≥50% improvement in UPCR with one of the following: UPCR <113 or <339 mg/mmoL, based on the BL ratio; NR= Not achieving either a CRR or a PRR. Participants achieved response if criteria at both months 11 and 12 (Days 337 and 365) were met. Participants who Early discontinuations were categorized as NR." (NCT00430677)
Timeframe: Month 12
Intervention | Participants (Number) |
---|
| No Renal Response | Partial Renal Response | Complete Renal Response |
---|
Abatacept 10/10 mg/kg | 69 | 9 | 21 |
,Abatacept 30/10 mg/kg | 61 | 14 | 24 |
,Placebo | 66 | 14 | 20 |
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Number of Participants Achieving Patient Response of Complete or Partial Response, Based on the June 2010 Food and Drug Administration Guidance Document for Lupus Nephritis
Patient response=complete, partial, or no response. Complete response=serum creatinine (SCr) normal, inactive urinary sediment, no cellular casts, urinary protein/creatinine (UPCR) ratio<56.5 mg/mmol. Partial response=SCr normal or ≤25% above baseline value, RBCs at reference range, UPCR <56.5 mg/mmoL OR ≥50% improvement in UPCR with one of the following: UPCR <113 or <339 mg/mmoL, based on the baseline ratio. No response=Not achieving complete or partial response criteria. (NCT00430677)
Timeframe: At Day 365 (end of Short-term Period) and Day 645
Intervention | Participants (Number) |
---|
| Day 365 | Day 645 (n=59, 59, and 62) |
---|
Abatacept 10/10 mg/kg | 39 | 29 |
,Abatacept 30/10 mg/kg | 43 | 45 |
,Placebo | 42 | 36 |
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Number of Participants Achieving Renal Response
Renal response=serum creatinine level ≤25% above baseline value and greater than or equal to 50% improvement in the urine protein/creatinine ratio with 1 of the following: urine protein/creatinine ratio (UPCR) <113 mg/mmol, if the baseline ratio was <= 339 mg/mmol OR UPCR <339 mg/mmol,if the baseline ratio >339 mg/mmol. (NCT00430677)
Timeframe: At Day 365 (end of short-term period) and Day 645
Intervention | participants (Number) |
---|
| Day 365 | Day 645 (n=59, 59, 62) |
---|
Abatacept 10/10 mg/kg | 66 | 59 |
,Abatacept 30/10 mg/kg | 46 | 47 |
,Placebo | 74 | 62 |
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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period
preRX=pretreatment; LLN=lower limit of normal; ULN=upper limit of normal. Hemoglobin (g/dL): >3g/dL decrease from preRX value. Hematocrit(%): <0.75*preRX. Erythrocytes (*10^6 c/uL): <0.75*preRX. Platelet count (*10^9 c/L): <0.67*LLN, or >1.5*ULN, or if preRX 1.25*ULN, or if preRX ULN; if preRX>ULN, use >1.2*preRX or 7.50*10^3 c/uL. Monocytes (absolute) (*10^3 c/uL): If value >2000/mm^3. Basophils (absolute)(*10^3 c/uL): If value >.750*10^3 c/uL. Eosinophils (absolute) (*10^3 c/uL): If value >.750*10^3 c/uL. ALP (U/L): >2*ULN, or if preRX>ULN, use >3* preRX. AST (U/L): >3*ULN, or if preRX>ULN, use >4*preRX. ALT (U/L): >3*ULN, or if preRX>ULN, use >4*preRX. GGT (U/L):>2*ULN, or if preRX >ULN, use >3*preRX. Bilirubin, total (mg/dL): >2*ULN, or if preRX>ULN, use >4*preRX. BUN (mg/dL): >1.5*preRX. (NCT00430677)
Timeframe: From start of study drug on Day 365 up to 56 days after last dose in the long-term extension period
Intervention | Participants (Number) |
---|
| Hemoglobin (low) | Hemoglobin (high) | Hematocrit (n=210) (low) | Hematocrit (n=210) (high) | Erythrocytes (low) | Erythrocytes (high) | Platelet count (n=210) (low) | Platelet count (n=210) (high) | Leukocytes (low) | Leukocytes (high) | Neutrophils + Bands (absolute) (low) | Neutrophils + Bands (absolute) (high) | Lymphocytes (absolute) (low) | Lymphocytes (absolute) (high) | Monocytes (absolute) (low) | Monocytes (absolute) (high) | Basophils (absolute) (low) | Basophils (absolute) (high) | Eosinophils (absolute) (low) | Eosinophils (absolute) (high) | Alkaline phosphatase (ALP) (low) | ALP (high) | Aspartate aminotransferase (AST) (low) | AST (high) | Alanine aminotransferase (ALT) (low) | ALT (high) | G-glutamyl transferase (GGT) (low) | GGT (high) | Bilirubin, total (low) | Bilirubin, total (high) | Blood urea nitrogen (BUN) (low) | BUN (high) | Creatinine (low) | Creatinine (high) |
---|
Abatacept 10 mg/kg | 13 | NA | 11 | NA | 10 | NA | 1 | 0 | 40 | 9 | 4 | NA | 59 | 0 | NA | 0 | NA | 0 | NA | 8 | NA | 3 | NA | 3 | NA | 5 | NA | 14 | NA | 0 | NA | 9 | NA | 16 |
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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)
LLN=lower limit of normal; ULN=upper limit of normal; preRX=pretreatment. Sodium, serum (mEq/L): <0.95*LLN or >1.05*ULN, or if preRXULN if preRX>ULN, use >1.05*preRX or 1.1*ULN, or if preRX ULN if preRX>ULN, use >1.1*preRX or 1.1*ULN, or if preRXULN. Calcium, total (mg/dL): <0.8*LLN or >1.2*ULN, or if preRXULN if preRX>ULN, use >1.25*preRX or 220 mg/dL. Glucose, fasting serum (mg/dL): <0.8*LLN or >1.5*ULN, or if preRX ULN if preRX>ULN, use >2.0*preRX or 2*preRX. Triglycerides (mg/dL): >=2.5*ULN, or if preRX>ULN, use >=2.5*preRX. Triglycerides, fasting (mg/dL): >=2*ULN, or if preRX>ULN, use >2.0*preRX. (NCT00430677)
Timeframe: From start of study drug on Day 365 up to 56 days after last dose in the long-term extension period
Intervention | Participants (Number) |
---|
| Sodium, serum (low) | Sodium, serum (high) | Potassium, serum (n=210) (low) | Potassium, serum (n=210) (high) | Chloride, serum (low) | Chloride, serum (high) | Calcium, total (n=210) (low) | Calcium, total (n=210) (high) | Glucose, serum (low) | Glucose, serum (high) | Glucose, fasting serum (low) (n=143) | Glucose, fasting serum (high) (n=143) | Protein, total (low) | Protein, total (high) | Albumin (low) | Albumin (high) | Cholesterol, total (low) (n=32) | Cholesterol, total (high) (n=32) | Triglycerides (low) (n=20) | Triglycerides (high) (n=20) | Triglycerides, fasting (low) (n=18) | Triglycerides, fasting (high) (n=18) | Protein, urine (low) | Protein, urine (high) | Glucose, urine (low) | Glucose, urine (high) | Blood, urine (low) | Blood, urine (high) | Leukocyte esterase, urine (low) (n=185) | Leukocyte esterase, urine (high) (n=185) |
---|
Abatacept 10 mg/kg | 1 | 2 | 11 | 3 | 0 | 0 | 1 | 2 | 23 | 2 | 2 | 2 | 19 | 0 | 12 | NA | 32 | NA | 20 | NA | 18 | NA | NA | 9 | NA | 1 | NA | 35 | NA | 27 |
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Number of Participants With Marked Laboratory Abnormalities During the Long-term Extension Period (Continued)
preRX=pretreatment. Protein, urine: If missing preRX, use >=2, or if value >=4, or if preRX =0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 OR 3 then use >=4. Glucose, urine: If missing preRX, use >=2, or if value >=4, or if preRX=0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. Blood, urine: If missing preRX, use >=2, or if value >=4, or if preRX =0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. Leukocyte esterase, urine: If missing preRX, use >=2, or if value >=4, or if preRX=0 or 0.5, use >=2, or if preRX=1, use >=3, or if preRX=2 or 3, use >=4. (NCT00430677)
Timeframe: From start of study drug on Day 365 to up to 56 days after last dose in the long-term extension period
Intervention | Participants (Number) |
---|
| Protein, urine (low) | Protein, urine (high) | Glucose, urine (low) | Glucose, urine (high) | Blood, urine (low) | Blood, urine (high) | Leukocyte esterase, urine (low) (n=185) | Leukocyte esterase, urine (high) (n=185) |
---|
Abatacept 10 mg/kg | NA | 9 | NA | 1 | NA | 35 | NA | 27 |
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Number of Participants With Positive Abatacept-induced Responses (ECL Method) Over Time During the Short-term Period
A validated, sensitive electrochemiluminescence (ECL) immunoassay based on Meso-Scale Discovery instrumentation was used to evaluate immunogenicity. The ECL assay differentiated between two antibody specificities: (1) the 'Ig and/or Junction (Jn) Region' and (2) 'CLTA4 and possibly Ig'. A sample was considered positive if it had a titer of 10 or greater and if immunodepletion was observed with abatacept with or without CTLA4-T. (NCT00430677)
Timeframe: Day 169, Day 365
Intervention | Participants (Number) |
---|
| CTLA4 and possibly Ig; Day 169 (n=90) | CTLA4 and possibly Ig; Day 365 (n=74) | CTLA4 and possibly Ig;Overall on TRT visits (n=90) | CTLA4 and possibly Ig; Overall Post visits (n=20) | CTLA4 and possibly Ig; Overall (n=96) | Ig/Jn region; Day 169 (n=90) | Ig/Jn region; Day 365 (n=78) | Ig/Jn region; Overall on TRT visits (n=90) | Ig/Jn region; Overall on Post visits (n=20) | Ig/Jn region; Overall (n=95) |
---|
Abatacept 10/10 mg/kg | 1 | 0 | 1 | 5 | 6 | 0 | 0 | 0 | 1 | 1 |
,Abatacept 30/10 mg/kg | 2 | 1 | 3 | 7 | 9 | 0 | 1 | 1 | 0 | 1 |
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Participants With Adverse Events (AEs), Serious AEs (SAEs), Deaths, and Discontinuations Due to AEs Reported During the Short-term Period
AE=any new unfavorable symptom, sign, or disease or worsening of a preexisting condition that may not have a causal relationship with treatment. SAE=a medical event that at any dose results in death, persistent or significant disability/incapacity, or drug dependency/abuse; is life-threatening, an important medical event, or a congenital anomaly/birth defect; or requires or prolongs hospitalization. Related=possibly, probably, or certainly related to and of unknown relationship to study drug. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| Deaths | SAEs | Related SAEs | AEs | Related AEs | Discontinued due to AEs |
---|
Abatacept 10/10 mg/kg | 2 | 28 | 19 | 89 | 53 | 13 |
,Abatacept 30/10 mg/kg | 5 | 33 | 20 | 93 | 61 | 14 |
,Placebo | 7 | 31 | 15 | 94 | 55 | 9 |
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Participants With AEs of Special Interest During the Short-term Period
AEs of special interest were prospectively identified to be those that may be associated with the use of immunomodulatory agents. They are a subset of all AEs and may be either serious or non-serious. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| Infections and Infestations | Malignancies | Autoimmune Disorders | Acute Infusional AEs | Peri-infusional AEs |
---|
Abatacept 10/10 mg/kg | 70 | 1 | 5 | 18 | 18 |
,Abatacept 30/10 mg/kg | 75 | 0 | 4 | 23 | 23 |
,Placebo | 75 | 1 | 3 | 17 | 17 |
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Participants With Marked Abnormalities Urinalysis During the Short-term Period
PTV=pretreatment value. Criteria for marked abnormality: Protein, glucose, blood, leukocyte esterase , if missing PTV then use >=2+ (or, if value >=4, or if PTV=0 or 0.5, >=2 or if PTV=1, >=3, or if PTV=2 or 3, >=4). (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| Urine Protein (n=99,98,99) | Urine Glucose (n=99,98,99) | Urine Blood (n=99,98,99) | Urine Leukocyte esterase (n=85,91,90) |
---|
Abatacept 10/10 mg/kg | 3 | 3 | 19 | 8 |
,Abatacept 30/10 mg/kg | 6 | 1 | 16 | 10 |
,Placebo | 8 | 0 | 17 | 8 |
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Participants With Marked Hematology Abnormalities During the Short-term Period
LLN=lower limit of normal; ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age. Low(↓)Hemoglobin:>3g/dL decrease from PTV; ↓Hematocrit:<0.75xPTV;↓Erythrocyte count:<0.75xPTV; high(↑)Platelet count:>1.5xULN;↓Platelet count:<0.67xLLN;↓Leukocyte count:<0.75X LLN;↑Leukocyte count:>1.25xULN;↓Absolute(AB)Neutrophils+Bands:<1.00x10^3c/uL;↑AB Lymphocyte count:>7.50x10^3 c/uL; ↓AB lymphocyte count:<0.750x10^3 c/uL;↑AB monocyte count:>2000/mm^3;↑AB basophil count:>400/mm^3;↑AB eosinophil count:>0.750x10^3 c/uL. (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| Low Hemoglobin (n=98, 98, 99) | Low Hematocrit (n=97, 98, 99) | Low Erythrocyte Count (n=98, 98, 99) | High Platelet Count (n=98, 98, 98) | Low Platelet Count (n=98, 98, 98) | High Leukocyte Count (n=98, 98, 99) | Low Leukocyte Count (n=98,98,99) | Low Absolute Neutrophils + Bands (n=98,98,99) | High Absolute Lymphocyte Count (n=98,98,99) | Low Absolute Lymphocyte Count (n=98, 98, 99) | High Absolute Monocyte Count (n=98, 98, 99) | High Absolute Basophil Count (n=98, 98, 99) | High Absolute Eosinophil Count (n=98, 98, 99) |
---|
Abatacept 10/10 mg/kg | 3 | 1 | 1 | 0 | 3 | 10 | 18 | 2 | 2 | 32 | 1 | 0 | 3 |
,Abatacept 30/10 mg/kg | 6 | 5 | 4 | 1 | 1 | 15 | 13 | 1 | 0 | 47 | 1 | 0 | 0 |
,Placebo | 9 | 7 | 7 | 1 | 1 | 11 | 16 | 7 | 0 | 53 | 1 | 0 | 0 |
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Participants With Marked Laboratory Abnormalities During the Short-term Period
LLN=lower limit of normal; ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age. ↑Serum Sodium:>1.05x ULN;↓Serum Potassium:<0.9x LLN;↑Serum Potassium:>1.1x ULN;↓Total Calcium:<0.8X LLN;↑Total Calcium:>1.2x ULN; ↓Serum Glucose(SG):<65 mg/dL;↑SG:>220 mg/dL;↓Fasting SG:<0.8x LLN;↑Fasting SG:>1.5x ULN;↓Total Protein:<0.9x LLN;↓Albumin:<0.9x LLN;↑Total Cholesterol:>2x PTV;↑Triglycerides:>=2.5x ULN;↑Fasting Triglycerides:>=2x ULN (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| High Serum Sodium (n=98, 98, 99) | Low Serum Potassium (n=98, 98, 99) | High Serum Potassium (n=98, 98, 99) | Low Total Calcium (n=98, 98, 99) | High Total Calcium (n=98, 98, 99) | Low Serum Glucose (n=98, 98, 99) | High Serum Glucose (n=98, 98, 99) | Low Fasting Serum Glucose (n=72, 73, 68) | High Fasting Serum Glucose (n=72, 73, 68) | Low Total Protein (n=98, 98, 99) | Low Albumin (n=98, 98, 99) | High Total Cholesterol (n=93,93,96) | High Triglycerides (n=71, 79, 75) | High Fasting Triglycerides (n=65, 64, 63) |
---|
Abatacept 10/10 mg/kg | 2 | 14 | 0 | 1 | 2 | 16 | 2 | 2 | 1 | 18 | 6 | 0 | 0 | 0 |
,Abatacept 30/10 mg/kg | 1 | 12 | 4 | 0 | 1 | 12 | 2 | 3 | 1 | 18 | 10 | 2 | 2 | 3 |
,Placebo | 0 | 15 | 2 | 1 | 1 | 18 | 2 | 0 | 0 | 25 | 4 | 1 | 0 | 0 |
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Participants With Marked Liver and Kidney Function Abnormalities During the Short-term Period
"ULN=upper limit of normal; PTV=pretreatment value. Normal ranges are provided by the Central Laboratory and may vary according to sex and age.~Alkaline Phosphatase:>2x ULN; ↑Aspartate Aminotransferase: >3x ULN; ↑Alanine Aminotransferase : >3x ULN; G-Glutamyl Transferase : >2x ULN; ↑Total Bilirubin : >2x ULN or if PTV > ULN then > 4x PTV; ↑Blood Urea Nitrogen >2x PTV; ↑Creatinine >1.5x PTV." (NCT00430677)
Timeframe: From Baseline (Day 1) up to 56 days post last dose in the double-blind period or the first dose in the open-label long-term extension, whichever occurred first.
Intervention | Participants (Number) |
---|
| High Alkaline Phosphatase | High Aspartate Aminotransferase | High Alanine Aminotransferase | High G-Glutamyl Transferase | High Total Bilirubin | High Blood Urea Nitrogen | High Creatinine |
---|
Abatacept 10/10 mg/kg | 1 | 0 | 4 | 7 | 0 | 1 | 6 |
,Abatacept 30/10 mg/kg | 1 | 2 | 7 | 7 | 0 | 5 | 16 |
,Placebo | 1 | 0 | 1 | 5 | 0 | 6 | 12 |
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Vital Signs Summary During the Short-term Period: Diastolic Blood Pressure (DBP)
(NCT00430677)
Timeframe: 0 - 12 Months
Intervention | mm Hg (Mean) |
---|
| Day 1: DBP-before infusion; n=76,81,73 | Day 1: DBP-1hour after infusion; n=69,78,70 | Day 1: DBP-2.5 hours after infusion; n=79,81,76 | Day 15: DBP-before infusion; n=78,78,74 | Day 15: DBP- 1hour after infusion; n=72,75,71 | Day 15: DBP-2.5 hours after infusion; n=80,78,78 | Day 29: DBP-before infusion; n=77,79,75 | Day 29: DBP-1hour after infusion; n=72,75,74 | Day 29: DBP-2.5 hours after infusion; n=80,79,82 | Day 57: DBP-before infusion; n=74,76,70 | Day 57: DBP-1hour after infusion; n=74,69,68 | Day 57: DBP-2.5 hours after infusion; n=81,73,73 | Day 85: DBP- before infusion; n=73,71,67 | Day 85: DBP-1hour after infusion; n=73,69,72 | Day 113: DBP-before infusion; n=72,70,70 | Day 113: DBP-1hour after infusion; n=75,70,75 | Day 141: DBP-before infusion; n=66,70,66 | Day 141: DBP-1hour after infusion; n=72,71,70 | Day 169: DBP-before infusion; n=70,70,66 | Day 169: DBP-1hour after infusion; n=75,71,71 | Day 197: DBP-before infusion; n=68,70,64 | Day 197: DBP-1hour after infusion; n=72,73,70 | Day 225: DBP-before infusion; n=70,64,63 | Day 225: DBP-1hour after infusion; n=72,65,68 | Day 253: DBP-before infusion; n=63,66,63 | Day 253: DBP-1hour after infusion; n=69,67,67 | Day 281: DBP-before infusion; n=63,66,58 | Day 281: DBP-1hour after infusion; n=66,65,64 | Day 309: DBP-before infusion; n=63,63,59 | Day 309: DBP-1hour after infusion; n=67,66,64 | Day 337: DBP-before infusion; n=63,64,61 | Day 337: DBP-1hour after infusion; n=68,65,67 |
---|
Abatacept 10/10 mg/kg | 80.6 | 79.6 | 79.0 | 80.0 | 78.7 | 78.0 | 78.8 | 78.0 | 78.2 | 78.7 | 75.3 | 76.1 | 75.2 | 75.6 | 77.0 | 76.3 | 74.0 | 74.7 | 74.1 | 74.4 | 75.8 | 74.5 | 73.6 | 74.5 | 75.9 | 74.9 | 73.3 | 74.5 | 72.9 | 72.9 | 73.4 | 73.3 |
,Abatacept 30/10 mg/kg | 80.8 | 79.6 | 79.3 | 81.1 | 79.6 | 81.4 | 79.6 | 79.9 | 78.5 | 80.0 | 78.9 | 79.5 | 79.7 | 78.1 | 78.5 | 76.1 | 78.8 | 77.8 | 75.5 | 76.5 | 76.8 | 76.1 | 74.5 | 75.0 | 76.1 | 74.5 | 77.6 | 75.9 | 78.1 | 76.4 | 78.0 | 78.1 |
,Placebo | 80.4 | 80.1 | 80.9 | 82.6 | 80.4 | 82.8 | 81.3 | 80.8 | 79.5 | 78.7 | 78.7 | 80.2 | 78.7 | 78.1 | 76.7 | 76.9 | 76.5 | 76.1 | 77.0 | 76.4 | 75.8 | 75.4 | 75.7 | 76.0 | 77.1 | 75.7 | 76.6 | 76.6 | 77.4 | 76.6 | 76.2 | 76.1 |
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Vital Signs Summary During the Short-term Period: Heart Rate
(NCT00430677)
Timeframe: 0 - 12 Months
Intervention | beats per minute (Mean) |
---|
| Day 1: before infusion; n=99,98,100 | Day 1: 1hour after infusion; n=98,98,98 | Day 1: 2.5 hours after infusion; n=97,96,96 | Day 15: before infusion; n=97,93,99 | Day 15: 1hour after infusion; n=96,94,98 | Day 15: 2.5 hours after infusion; n=93,92,95 | Day 29: before infusion; n=94,93,99 | Day 29: 1hour after infusion; n=93,92,99 | Day 29: 2.5 hours after infusion; n=92,90,97 | Day 57: before infusion; n=93,88,84 | Day 57: 1hour after infusion; n=93,84,94 | Day 57: 2.5 hours after infusion; n=91,84,91 | Day 85: before infusion; n=89,86,91 | Day 85: 1hour after infusion; n=88,84,89 | Day 113: before infusion; n=88,82,92 | Day 113: 1hour after infusion; n=87,80,91 | Day 141: before infusion; n=84,82,89 | Day 141: 1hour after infusion; n=84,81,86 | Day 169: before infusion; n=85,82,86 | Day 169: 1hour after infusion; n=84,82,85 | Day 197: before infusion; n=83,82,84 | Day 197: 1hour after infusion; n=82,82,85 | Day 225: before infusion; n=83,76,81 | Day 225: 1hour after infusion; n=83,76,81 | Day 253: before infusion; n=78,77,81 | Day 253: 1hour after infusion; n=78,77,81 | Day 281: before infusion; n=75,76,77 | Day 281: 1hour after infusion; n=74,76,76 | Day 309: before infusion; n=76,75,77 | Day 309: 1hour after infusion; n=76,74,77 | Day 337: before infusion; n=73,75,80 | Day 337: 1hour after infusion; n=73,73,79 |
---|
Abatacept 10/10 mg/kg | 79.2 | 79.6 | 82.2 | 81.0 | 80.1 | 81.7 | 80.8 | 79.9 | 82.1 | 82.2 | 81.6 | 83.4 | 82.3 | 80.4 | 80.4 | 80.2 | 79.3 | 79.1 | 78.0 | 78.8 | 78.1 | 78.7 | 77.6 | 78.0 | 79.1 | 79.3 | 78.3 | 77.6 | 76.5 | 77.4 | 76.4 | 77.4 |
,Abatacept 30/10 mg/kg | 82.4 | 81.3 | 81.8 | 83.1 | 81.3 | 81.5 | 83.7 | 81.7 | 81.3 | 83.4 | 81.0 | 82.5 | 84.8 | 83.3 | 82.1 | 81.8 | 82.8 | 81.4 | 80.1 | 79.5 | 81.7 | 79.8 | 80.1 | 79.1 | 81.4 | 78.8 | 80.1 | 78.0 | 79.8 | 77.9 | 78.5 | 77.6 |
,Placebo | 82.6 | 81.9 | 83.8 | 83.7 | 82.6 | 84.7 | 83.0 | 81.7 | 84.7 | 82.8 | 81.8 | 83.8 | 82.5 | 83.5 | 83.0 | 82.6 | 82.6 | 81.3 | 79.1 | 81.4 | 79.3 | 79.6 | 78.9 | 80.2 | 81.5 | 79.5 | 79.3 | 78.7 | 78.2 | 78.4 | 78.9 | 78.3 |
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Vital Signs Summary During the Short-term Period: Systolic Blood Pressure (SBP)
(NCT00430677)
Timeframe: 0 - 12 Months
Intervention | mmHg (Mean) |
---|
| Day 1: SBP-before infusion; n=76,81,73 | Day 1: SBP-1hour after infusion; n=69,78,70 | Day 1: SBP-2.5 hours after infusion; n=79,81,76 | Day 15: SBP-before infusion; n=78,78,74 | Day 15: SBP- 1hour after infusion; n=72,75,71 | Day 15: SBP-2.5 hours after infusion; n=80,78,78 | Day 29: SBP-before infusion; n=77,79,75 | Day 29: SBP-1hour after infusion; n=72,75,74 | Day 29: SBP-2.5 hours after infusion; n=80,79,82 | Day 57: SBP-before infusion; n=74,76,70 | Day 57: SBP-1hour after infusion; n=74,69,68 | Day 57: SBP-2.5 hours after infusion; n=81,73,73 | Day 85: SBP- before infusion; n=73,71,67 | Day 85: SBP-1hour after infusion; n=73,69,72 | Day 113: SBP-before infusion; n=72,70,70 | Day 113: SBP-1hour after infusion; n=75,70,75 | Day 141: SBP-before infusion; n=66,70,66 | Day 141: SBP-1hour after infusion; n=72,71,70 | Day 169: SBP-before infusion; n=70,70,66 | Day 169: SBP-1hour after infusion; n=75,71,71 | Day 197: SBP-before infusion; n=68,70,64 | Day 197: SBP-1hour after infusion; n=72,73,70 | Day 225: SBP-before infusion; n=70,64,63 | Day 225: SBP-1hour after infusion; n=72,65,68 | Day 253: SBP-before infusion; n=63,66,63 | Day 253: SBP-1hour after infusion; n=69,67,67 | Day 281: SBP-before infusion; n=63,66,58 | Day 281: SBP-1hour after infusion; n=66,65,64 | Day 309: SBP-before infusion; n=63,63,59 | Day 309: SBP-1hour after infusion; n=67,66,64 | Day 337: SBP-before infusion; n=63,64,61 | Day 337: SBP-1hour after infusion; n=68,65,67 |
---|
Abatacept 10/10 mg/kg | 127.8 | 126.7 | 127.8 | 125.5 | 125.9 | 124.9 | 126.2 | 125.9 | 126.4 | 124.8 | 122.1 | 123.6 | 118.7 | 119.6 | 121.7 | 121.0 | 119.2 | 117.9 | 117.6 | 118.2 | 118.9 | 117.4 | 118.8 | 119.5 | 120.2 | 117.1 | 116.5 | 119.4 | 116.3 | 114.1 | 117.3 | 117.7 |
,Abatacept 30/10 mg/kg | 128.2 | 127.7 | 127.1 | 127.6 | 126.9 | 130.1 | 125.6 | 126.2 | 126.3 | 126.4 | 124.6 | 127.0 | 122.0 | 121.8 | 122.6 | 121.5 | 121.4 | 120.9 | 118.5 | 120.3 | 119.0 | 117.7 | 118.2 | 116.6 | 120.6 | 118.1 | 120.7 | 119.5 | 119.4 | 119.6 | 119.9 | 120.4 |
,Placebo | 126.2 | 126.0 | 126.2 | 129.2 | 128.2 | 131.2 | 126.9 | 128.6 | 127.6 | 123.0 | 124.4 | 126.1 | 122.2 | 122.0 | 119.9 | 120.1 | 118.8 | 120.4 | 118.6 | 118.9 | 118.7 | 120.4 | 119.5 | 118.8 | 120.9 | 119.7 | 120.3 | 121.0 | 120.5 | 119.2 | 119.3 | 119.3 |
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Vital Signs Summary During the Short-term Period: Temperature
(NCT00430677)
Timeframe: 0 - 12 Months
Intervention | degree celcius (Mean) |
---|
| Day 1: before infusion; n=99,98,99 | Day 1: 1hour after infusion; n=98,96,97 | Day 1: 2.5 hours after infusion; n=97,93,97 | Day 15: before infusion; n=97,94,99 | Day 15: 1hour after infusion; n=96,91,98 | Day 15: 2.5 hours after infusion; n=93,91,95 | Day 29: before infusion; n=94,93,99 | Day 29: 1hour after infusion; n=93,91,98 | Day 29: 2.5 hours after infusion; n=92,90,97 | Day 57: before infusion; n=93,88,93 | Day 57: 1hour after infusion; n=93,83,94 | Day 57: 2.5 hours after infusion; n=90,84,90 | Day 85: before infusion; n=89,86,91 | Day 85: 1hour after infusion; n=87,84,89 | Day 113: before infusion; n=88,82,92 | Day 113: 1hour after infusion; n=87,79,91 | Day 141: before infusion; n=84,81,89 | Day 141: 1hour after infusion; n=84,80,86 | Day 169: before infusion; n=85,81,86 | Day 169: 1hour after infusion; n=84,80,84 | Day 197: before infusion; n=83,82,85 | Day 197: 1hour after infusion; n=82,81,84 | Day 225: before infusion; n=83,76,81 | Day 225: 1hour after infusion; n=83,75,81 | Day 253: before infusion; n=78,77,80 | Day 253: 1hour after infusion; n=78,77,81 | Day 281: before infusion; n=75,76,77 | Day 281: 1hour after infusion; n=74,76,76 | Day 309: before infusion; n=76,75,77 | Day 309: 1hour after infusion; n=75,73,77 | Day 337: before infusion; n=73,75,79 | Day 337: 1hour after infusion; n=73,72,79 |
---|
Abatacept 10/10 mg/kg | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.4 | 36.5 | 36.5 | 36.4 | 36.4 | 36.5 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.3 | 36.3 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.4 | 36.3 | 36.4 | 36.4 | 36.4 |
,Abatacept 30/10 mg/kg | 36.6 | 36.6 | 36.6 | 36.5 | 36.6 | 36.6 | 36.5 | 36.5 | 36.5 | 36.5 | 36.1 | 36.5 | 36.4 | 36.4 | 36.5 | 36.4 | 36.5 | 36.5 | 36.5 | 36.4 | 36.5 | 36.4 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 |
,Placebo | 36.5 | 36.6 | 36.6 | 36.5 | 36.5 | 36.6 | 36.5 | 36.5 | 36.5 | 36.5 | 36.6 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.4 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 | 36.5 |
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Number of Participants (Patients) Who Attained Neutrophil Engraftment
"Defined as absolute neutrophils (ANC) > 5 x 10^8/Liter for 3 consecutive days.~ANC is the real number of white blood cells (WBCs) that are neutrophils. The absolute neutrophil count is commonly called the ANC. The ANC is not measured directly. It is derived by multiplying the WBC count times the percent of neutrophils in the differential WBC count. The percent of neutrophils consists of the segmented (fully mature) neutrophils) + the bands (almost mature neutrophils). The normal range for the ANC = 1.5 to 8.0 (1,500 to 8,000/mm3)." (NCT00354172)
Timeframe: Day 42 Post Transplant
Intervention | Participants (Number) |
---|
Evaluable Patients | 13 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 5 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 14 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 15 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 4 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 10 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 11 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 1 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 2 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 6 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 1 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 1 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 3 |
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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
Intervention | Participants (Number) |
---|
Evaluable Patients | 0 |
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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
Intervention | Percentage of Engrafted Cells (Median) |
---|
| Day 21 | Day 100 | 6 Months |
---|
Evaluable Patients | 92 | 100 | 96.5 |
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GFR at Month 12 Utilizing Modification of Diet in Renal Disease (MDRD) Method
"Change in GFR (Modification of Diet in Renal Disease calculated using the -MDRD formulat:~For men: GFR = 170 × (serum creatinine -0,999)×(age-0,176) x (urea nitrogen -0,17) × (albumin0,318) For women: GFR = 170 × (serum creatinine -0,999) × (age-0,176) × (urea nitrogen -0,17) x (albumin0,318) × 0.762 with urea nitrogen = urea / 2.144. ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate." (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant
Intervention | ml/min/1.73m² (Least Squares Mean) |
---|
Standard Regimen | 50.23 |
CNI Free Regimen | 56.36 |
CNI Low Regimen | 50.24 |
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GFR at Month 60 Utilizing Modification of Diet in Renal Disease (MDRD) Method
"Change in GFR (Modification of Diet in Renal Disease calculated using the -MDRD formulat:~For men: GFR = 170 × (serum creatinine -0,999)×(age-0,176) x (urea nitrogen -0,17) × (albumin0,318) For women: GFR = 170 × (serum creatinine -0,999) × (age-0,176) × (urea nitrogen -0,17) x (albumin0,318) × 0.762 with urea nitrogen = urea / 2.144. ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate." (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60 post-transplant
Intervention | ml/min/1.73m² (Least Squares Mean) |
---|
Standard Regimen | 47.56 |
CNI Free Regimen | 53.41 |
CNI Low Regimen | 44.79 |
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GFR Via Nankivell Method at Month 12 - CNI-Free vs Standard Regimen
Demonstrate superiority of CNI-Free vs Standard Regimen in GFR using the Nankivell formula (GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C where where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kilograms, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The calculated GFR is expressed in mL/min per 1.73m², last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model, with treatment, center, donor type (deceased vs. living) as factors and BL2-value at V4/M3/BL2 as covariate. P-values are not adjusted (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant
Intervention | ml/min/1.73m² (Least Squares Mean) |
---|
Standard Regimen | 63.03 |
CNI Free Regimen | 68.59 |
CNI Low Regimen | 63.08 |
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Mean Change in Serum Creatinine From Month 3 to Month 12
Change in venous blood serum creatinine. Last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 12 post-transplant
Intervention | mg/dl (Least Squares Mean) |
---|
Standard Regimen | 1.66 |
CNI Free Regimen | 1.58 |
CNI Low Regimen | 1.76 |
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Mean Change in Serum Creatinine From Month 3 to Month 60
Change in venous blood serum creatinine. Last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT00514514)
Timeframe: From randomization at BL2 (Month 3) to Month 60 post-transplant
Intervention | mg/dl (Least Squares Mean) |
---|
Standard Regimen | 1.94 |
CNI Free Regimen | 1.69 |
CNI Low Regimen | 2.01 |
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Change From BL2 (Month 3) to Month 12 in Cardiovascular Risk (Framingham Score; 10-year Cardiovascular Risk)
The Framingham Score (based on LDL cholesterol level) estimates the coronary heart disease risk (%) of developing one of the following coronary heart diseases: angina pectoris, myocardial infarction, or coronary disease death, over the course of 10 years. (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 12
Intervention | Percent risk (Mean) |
---|
| Baseline 1/Visit 1 (n=165,171,161) | Baseline 2/Month 3 (n=165,171,161) | Month 12 (n=158,166,156) | Change from Baseline 2 to Month 12 (n=158,166,156) |
---|
CNI Free Regimen | 10.2 | 8.8 | 9.1 | 0.4 |
,CNI Low Regimen | 9.5 | 9.3 | 8.7 | -0.7 |
,Standard Regimen | 10.9 | 10.3 | 9.4 | -0.7 |
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Efficacy Event Data After Month 12 to Month 60
Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: Events starting after Month 12
Intervention | Participants (Number) |
---|
| BPAR | Graft loss | Death | Lost to follow-up | Discontinuation due to adverse event | Therapy failure (composite endpoint) |
---|
CNI Free Regimen | 13 | 7 | 4 | 15 | 8 | 35 |
,CNI Low Regimen | 12 | 3 | 9 | 13 | 4 | 36 |
,Standard Regimen | 13 | 7 | 7 | 17 | 10 | 38 |
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Efficacy Event Data Baseline 2 (Month 3) to Month 12
Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 12
Intervention | Participants (Number) |
---|
| BPAR | Graft loss | Death | Lost to follow-up | Discontinuation due to lack of efficacy | Discontinuation due to adverse event | Therapy failure composite |
---|
CNI Free Regimen | 20 | 2 | 2 | 0 | 3 | 44 | 58 |
,CNI Low Regimen | 13 | 1 | 2 | 0 | 1 | 27 | 35 |
,Standard Regimen | 13 | 1 | 3 | 0 | 2 | 25 | 34 |
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Efficacy Event Data From Baseline 2 (Month 3) to Month 6
Efficacy events were: Biopsy-proven acute rejection (BPAR), graft loss, death, and treatment failure (defined as composite endpoint of BPAR, graft loss, death, loss to follow-up, discontinuation due to lack of efficacy or due to toxicity). (NCT00514514)
Timeframe: From Baseline 2 (Month 3) to Month 6
Intervention | Participants (Number) |
---|
| BPAR | Graft loss | Death | Lost to follow-up | Discontinuation due to lack of efficacy | Discontinuation due to adverse event | Therapy failure composite |
---|
CNI Free Regimen | 15 | 1 | 1 | 0 | 2 | 26 | 37 |
,CNI Low Regimen | 10 | 1 | 0 | 0 | 1 | 13 | 19 |
,Standard Regimen | 6 | 0 | 1 | 0 | 1 | 8 | 14 |
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Modification of Diet and Renal Disease (MDRD) Measured Glomerular Filtration Rates in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
Modification of Diet and Renal Disease (MDRD) Measured Glomerular Filtration Rates were used as part of a measure of renal function and measured at 24 weeks in patients who were still participating in the study at that point. (NCT00522548)
Timeframe: 6 months
Intervention | MDRD (mL/min/1.73m2) (Mean) |
---|
Myfortic Comparator Group | 59.6 |
CellCept Comparator Group | 54.8 |
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Serum Creatinine in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
Serum creatinine lab values were used as part of a measure of renal function at 24 weeks for any patient that was still participating in the study at this time-point. (NCT00522548)
Timeframe: 6 months
Intervention | serum creatinine mg/dL (Mean) |
---|
Myfortic Comparator Group | 1.9 |
CellCept Comparator Group | 1.9 |
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The Incidence of Intolerance (Defined as Transient Dose Reduction or Transient Discontinuation of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The incidence of intolerance was defined as transient dose reduction or transient discontinuation of enteric coated mycophenolate sodium or mycophenolate mofetil or its generic equivalent manufactured by Sandoz meaning doses could subsequently be resumed or increased back to original starting dose, once intolerance resolved. (NCT00522548)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Myfortic Comparator Group | 1 |
CellCept Comparator Group | 0 |
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The Incidence of Rejection in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz.
All rejection episodes of the kidney transplant were proven by kidney transplant biopsy and were measured at 24 weeks for all patients still participating in the study at that timepoint. (NCT00522548)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Myfortic Comparator Group | 2 |
CellCept Comparator Group | 1 |
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The Incidence of the Requirement of Full Dose Proton Pump Inhibitors in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
If patients were not on any medications from a medication class known as H-2 antagonists, they were started on ranitidine 150 mg orally twice daily after transplant (with dose adjusted for renal function). Patients were excluded if they were on proton pump inhibitor at time of study screening, however some patients required addition of proton pump inhibitor post-study enrollment. If a patient had upper gastrointestinal side effects such as acid reflux unreleived on ranitidine therapy or nausea, they were switched to a proton pump inhibitor. (NCT00522548)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Myfortic Comparator Group | 3 |
CellCept Comparator Group | 1 |
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Gastrointestinal Symptom Rating Scale (GSRS) Score Changes From Baseline to 24 Weeks After Transplant in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item self-administered questionnaire to assess symptoms associated with common gastrointestinal (GI) disorders, and has been validated in renal transplant recipients. It uses a seven-graded Likert scale, where 1 represents the most positive option and 7 the most negative one and the patient grades symptoms based on the past 7 days. A score of ≥ 2 indicates the presence of GI symptoms. Higher values indicate more unfavorable conditions. The questionairre is divided into 5 sub-scales: diarrhea, indigestion, constipation, abdominal pain, and reflux and a mean score is calculated for each dimension. The lowest mean score possible for each dimension is 1 and the highest is 7. Patients completed a GSRS survey at baseline (pre-transplant to two days after transplant), and at weeks 1,4,12 and 24 after transplant. (NCT00522548)
Timeframe: baseline (pre-transplant to two days after transplant) and at 6 months after transplant.
Intervention | Participants (Count of Participants) |
---|
| Worsening of GSRS Score pre-transplant-wk 24 | No Worsening of GSRS Score pre-transplant-wk 24 |
---|
CellCept Comparator Group | 4 | 10 |
,Myfortic Comparator Group | 4 | 13 |
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Pharmacokinetics (Mycophenolic Acid Area Under the Curve) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of Enteric-Coated Mycophenolate Sodium or Mycophenolate Mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose). A mycophenolic acid area under the curve value, also known as AUC represents drug exposure. (NCT00522548)
Timeframe: 1 and 6 months
Intervention | mg*hr/L (Mean) |
---|
| Mycophenolic Acid AUC (mg*hr/L) at week 4 | Mycophenolic Acid AUC (mg*hr/L) at week 24 |
---|
CellCept Comparator Group | 33.8 | 42.4 |
,Myfortic Comparator Group | 23.9 | 21.1 |
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Pharmacokinetics (Mycophenolic Acid Maximum Concentration) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of Enteric-Coated Mycophenolate Sodium or Mycophenolate Mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose).A mycophenolic acid drawn at the peak level is called C Max or maximum concentration. (NCT00522548)
Timeframe: 1 and 6 months
Intervention | mg/L (Mean) |
---|
| Mycophenolic Acid C max (mg/L) at week 4 | Mycophenolic Acid C max (mg/L) at week 24 |
---|
CellCept Comparator Group | 10.5 | 13.9 |
,Myfortic Comparator Group | 4.3 | 6.4 |
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Pharmacokinetics (Mycophenolic Acid Trough Levels) of Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz in a Sub-set of Patients
Pharmacokinetics is the study of how a drug is absorbed and broken down in the body. Pharmacokinetics of enteric coated mycophenolate sodium and mycophenolate mofetil are measured by a level called mycophenolic acid level. Mycophenolic acid levels in this pharmacokinetic study were drawn starting in the morning at time zero (immediately prior to morning dose, also known as trough), then at 0.5, 1,1.5, 2, 2.5, 3,3.5,4,6, 8 and 12 hours post dose. Data was also dose-normalized (concentration was divided by dose). (NCT00522548)
Timeframe: 1 and 6 months
Intervention | mg/L (Mean) |
---|
| Mycophenolic Acid Trough (mg/L) at week 4 | Mycophenolic Acid Trough(mg/L) at week 24 |
---|
CellCept Comparator Group | 1.9 | 1.5 |
,Myfortic Comparator Group | 2.4 | 1.9 |
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The Incidence of the Occurrence of Lower Gastrointestinal Symptoms Per GSRS Scale Ratings in Patients on Enteric Coated Mycophenolate Sodium or Mycophenolate Mofetil or Its Generic Equivalent Manufactured by Sandoz
The Gastrointestinal Symptom Rating Scale (GSRS) is a 15-item self-administered questionnaire to assess symptoms associated with common gastrointestinal (GI) disorders, and has been validated in renal transplant recipients. It uses a seven-graded Likert scale, where 1 represents the most positive option and 7 the most negative one and the patient grades symptoms based on the past 7 days. A score of ≥ 2 indicates the presence of GI symptoms. Higher values indicate more unfavorable conditions. The questionairre is divided into 5 sub-scales: diarrhea, constipation, abdominal pain, indigestion and reflux and a mean score is calculated for each dimension. The lowest mean score possible for each dimension is 1 and the highest is 7. Patients completed a GSRS survey at baseline (pre-transplant to two days after transplant), and at weeks 1,4,12 and 24 post-transplant. Patients who withdrew from the study only had data included up to the point of withdraw. No values were carried forward. (NCT00522548)
Timeframe: 6 months
Intervention | Participants (Count of Participants) |
---|
| Number of Participants with Lower GI symptoms | Number of Participants without Lower GI symptoms |
---|
CellCept Comparator Group | 9 | 5 |
,Myfortic Comparator Group | 8 | 9 |
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Change From Baseline in the Average Maximum Intimal Thickness at Month 12
Maximum intimal thickness was assessed using Intravascular Ultrasound (IVUS). IVUS is a technique for taking ultrasound pictures of the wall of an artery from inside the artery itself. It shows the thickness of the artery wall and any narrowing of the artery. (NCT00300274)
Timeframe: Baseline, Month 12
Intervention | mm (Mean) |
---|
Everolimus 1.5 mg | 0.03 |
Everolimus 3.0 mg | 0.04 |
Mycophenolate Mofetil | 0.07 |
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Percentage of Participants With Cardiac Allograft Vasculopathy (CAV) at Month 12
Cardiac allograft vasculopathy is defined as a 0.5 mm increase in maximum intimal thickness as measured by Intravascular Ultrasound (IVUS) in at least one matched slice between baseline and Month 12. (NCT00300274)
Timeframe: 12 Months
Intervention | Percentage of participants (Number) |
---|
Everolimus 1.5 mg | 12.5 |
Everolimus 3.0 mg | 21.6 |
Mycophenolate Mofetil | 26.7 |
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Percentage of Participants With Composite Efficacy Failure at 12 Months
"Composite efficacy failure was defined as Biopsy Proven Acute Rejection(BPAR) of International Society for Heart and Lung Transplantation(ISHLT) grade ≥3A, Acute Rejection associated with Hemodynamic Compromise, Graft loss/Retransplant, Death or Loss to follow-up.~Identification of acute rejection was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤30% or 25% lower than Baseline or Fractional shortening ≤20% or 25% lower than Baseline and/or use of inotropic treatment." (NCT00300274)
Timeframe: 12 Months
Intervention | Percentage of participants (Number) |
---|
Everolimus 1.5 mg | 35.1 |
Everolimus 3.0 mg | 35.1 |
Mycophenolate Mofetil | 33.6 |
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Percentage of Participants With Composite Efficacy Failure at 24 Months
"Composite efficacy failure was defined as Biopsy Proven Acute Rejection (BPAR) of International Society for Heart and Lung Transplantation grade ≥ 3A, Acute Rejection associated with Hemodynamic Compromise, Graft loss/Retransplant, Death or Loss to follow-up.~Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline and/or use of inotropic treatment." (NCT00300274)
Timeframe: 24 Months
Intervention | Percentage of participants (Number) |
---|
Everolimus 1.5 mg | 39.4 |
Everolimus 3.0 mg | 41.1 |
Mycophenolate Mofetil | 41.3 |
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Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 12 Months
Loss to follow-up for this composite endpoint included participants who did not experience graft loss/re-transplant or death and whose last day of contact was prior to Day 316 (start day of the Month 12 visit window). (NCT00300274)
Timeframe: 12 Months
Intervention | Percentage of participants (Number) |
---|
Everolimus 1.5 mg | 11.7 |
Everolimus 3.0 mg | 11.9 |
Mycophenolate Mofetil | 8.9 |
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Percentage of Participants With Graft Loss/Re-transplant, Death or Loss to Follow-up at 24 Months
Loss to follow-up for this composite endpoint included participants who did not experience graft loss/re-transplant or death and whose last day of contact was prior to Day 631 (start day of 24 Month visit window). (NCT00300274)
Timeframe: 24 Months
Intervention | Percentage of participants (Number) |
---|
Everolimus 1.5 mg | 15.2 |
Everolimus 3.0 mg | 16.1 |
Mycophenolate Mofetil | 15.1 |
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Renal Function Calculated by Glomerular Filtration Rate (GFR) at 24 Months
"GFR was calculated using the Modification of Diet and Renal Disease (MDRD) formula:~GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R~C is the serum concentration of creatinine [mg/dL] A is age [years] G=0.742 when gender is female, otherwise G=1 R=1.21 when race is black, otherwise R=1" (NCT00300274)
Timeframe: 24 Months
Intervention | mL/min/1.73^2 (Mean) |
---|
Everolimus 1.5 mg | 59.50 |
Everolimus 3.0 mg | 61.84 |
Mycophenolate Mofetil | 64.52 |
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Renal Function Measured by Glomerular Filtration Rate (GFR) at 12 Months
"GFR was calculated using the Modification of Diet and Renal Disease (MDRD) formula:~GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where C is the serum concentration of creatinine [mg/dL] A is age [years] G=0.742 when gender is female, otherwise G=1 R=1.21 when race is black, otherwise R=1" (NCT00300274)
Timeframe: 12 Months
Intervention | mL/min/1.73^2 (Mean) |
---|
Everolimus 1.5 mg | 59.21 |
Everolimus 3.0 mg | 59.78 |
Mycophenolate Mofetil | 64.37 |
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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection (AR) Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 24
"Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline, and/ or use of inotropic treatment." (NCT00300274)
Timeframe: 24 Months
Intervention | Percentage of participants (Number) |
---|
| AR associated with HDC | BPAR of ISHLT grade ≥ 3A | Death | Graft loss/re-transplant |
---|
Everolimus 1.5 mg | 4.3 | 24.1 | 10.6 | 2.5 |
,Everolimus 3.0 mg | 3.6 | 28.6 | 11.9 | 3.0 |
,Mycophenolate Mofetil | 5.2 | 27.3 | 9.2 | 3.7 |
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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR of ISHLT Grade ≥ 3A), Acute Rejection Associated With Hemodynamic Compromise (HDC), Graft Loss/Re-transplant and Death at Month 12
"Identification of acute rejections was based on the local pathologist's evaluation of endomyocardial biopsy slides.~Hemodynamic compromise was present if 1 or more of the following were met: Ejection fraction ≤ 30% or 25% lower than Baseline or Fractional shortening ≤ 20% or 25% lower than Baseline, and/or use of inotropic treatment." (NCT00300274)
Timeframe: 12 Months
Intervention | Percentage of participants (Number) |
---|
| AR associated with HDC | BPAR of ISHLT ≥ 3A | Death | Graft loss/re-transplant |
---|
Everolimus 1.5 mg | 3.9 | 22.3 | 7.8 | 1.4 |
,Everolimus 3.0 mg | 3.0 | 25.6 | 10.1 | 3.0 |
,Mycophenolate Mofetil | 2.6 | 24.7 | 4.8 | 1.8 |
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Donor Stem Cell Engraftment, Including Donor-host Hematopoietic Chimerism Studies Post Transplant
Engraftment of donor cells by week 4 after transplantation. Assessment of donor stem cell engraftment will include donor-host hematopoietic chimerism analyses at 4 weeks and every 3 months after transplantation. Chimerism measured from peripheral blood or from bone marrow. (NCT00548717)
Timeframe: 30 days
Intervention | participants (Number) |
---|
Siro/MMF | 9 |
Siro/MMF/Bort | 2 |
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Incidence of 100 Day Mortality
(NCT00548717)
Timeframe: 100 days
Intervention | percentage of participants (Number) |
---|
Siro/MMF | 31 |
Siro/MMF/Bort | 0 |
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Incidence of Chronic GVHD
"Chronic GVHD will be graded according to the modified Seattle criteria. Chronic GVHD divided into limited and extensive and evaluated across the following systems: skin, cutaneous structures, liver, mouth, eyes, esophagus, intestines, lung, musculoskeletal, serous, nervous, urologic, vagina, hematopoietic, and immune.~Localized skin involvement with or without hepatic dysfunction is classified as limited disease.~Generalized skin involvement or limited disease plus eye involvement, oral involvement, hepatic dysfunction with abnormal liver histology, or involvement of any other target organ was classified as extensive disease.~Lee SJ, Vogelsang G, Flowers ME. Chronic graft-versus-host disease. Biol Blood Marrow Transplant.~2003;9:215-33." (NCT00548717)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Siro /MMF | 15 |
Siro/MMF/Bort | 50 |
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Overall Survival
(NCT00548717)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Siro/MMF | 39 |
Siro/MMF/Bort | 0 |
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The Rate of Renal Insufficiency
"Renal function will be measured weekly after transplant for 4 weeks, at 8 weeks and then at 3 month intervals from transplantation. Sentinel renal events such as the occurrence of thrombotic microangiopathy will be noted.~The rationale for the substitution of mycophenolate mofetil for tacrolimus is to continue to prevent acute GVHD, but minimize renal toxicity after transplantation. We will thus monitor renal toxicity closely in this study. In our previous experience, the rate of grade III-V renal toxicity by day 100 after transplantation was about 10%. Here, grade III is defined as a creatinine level between 3.1 to 6 times the normal level, grade IV is defined as a creatinine level 6 times or more the normal level, and grade V is defined as a fatality due to renal toxicity." (NCT00548717)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Siro/MMF | 1 |
Siro/MMF/Bort | 0 |
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To Determine the Rate of Grade II-IV Acute GVHD When Sirolimus and Mycophenolate Mofetil or Sirolimus, Mycophenolate Mofetil and Bortezomib is Used for GVHD Prophylaxis After Allogeneic Stem Cell Transplantation in Patients With Hematologic Malignancies
(NCT00548717)
Timeframe: 150 days
Intervention | percentage of participants (Number) |
---|
Siro/MMF | 77 |
Siro/MMF/Bort | 100 |
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Number of Participants With Acute Graft-versus-host Disease (aGVHD)
Participants who had acute graft-versus-host disease (aGVHD) within 100 days post transplant. Physical exam and bloodwork every week (for the first 90-100 days after the transplant). (NCT00506948)
Timeframe: Baseline to 100 days post transplant
Intervention | participants (Number) |
---|
Thymoglobulin + Sirolimus + MMF | 6 |
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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Deaths
Treatment Failure was adjudicated by a clinical endpoints committee (CEC) and was defined as the time to the earliest occurrence of any one of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or a requirement for rescue therapy for exacerbation or deterioration of Lupus nephritis (LN). (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | Deaths (Number) |
---|
Mycophenolate Mofetil (MMF) | 0 |
Azathioprine (AZA) | 1 |
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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With End-stage Renal Disease (ESRD)
Time to treatment failure, adjudicated by the Clinical Endpoints Committee (CEC), was defined as any 1 the following: death, ESRD, sustained doubling of serum creatinine, renal flare (proteinuric or nephritic), or requirement for rescue therapy to treat deterioration or exacerbation of Lupus nephritis. ESRD is defined as progression to chronic hemodialysis or renal transplant. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | participants (Number) |
---|
Mycophenolate Mofetil (MMF) | 0 |
Azathioprine (AZA) | 3 |
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Maintenance Phase: Events Contributing to the Primary Endpoint: Number of Participants With Sustained Doubling of Serum Creatinine
Sustained doubling of serum creatinine concentration is defined as the first serum creatinine value that is twice the mean of the lowest 2 values from screening to end of induction, as confirmed by a second serum creatinine value obtained at least 4 weeks after the initial doubling. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 1 |
Azathioprine | 5 |
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Maintenance Phase: Participants With Major Extra-renal Flare
A major extra-renal flare is defined as a British Isles Lupus Assessment Group (BILAG) Score category A in one extrarenal organ or three organs with concurrent category B scores. BILAG indices provide a scoring system for the assessment of lupus disease activity in terms of the need for steroid treatment in 8 organs/systems. Eighty-six items were scored resulting in a classification of A (severe activity), B (moderate activity), C (mild activity), D (no current activity) and E (no activity ever observed) for each organ system. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 7 |
Azathioprine | 6 |
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Induction Phase: Change From Baseline in Short-Form Health Survey (SF-36) Domain and Component Scores
The SF-36 is a 36 item quality of life questionnaire. The short-form version has eleven questions that permit the participant to rate how they feel that particular day. The SF-36 consists of eight scaled scores and two component scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 score with the higher scores indicating better quality of life. (NCT00377637)
Timeframe: Baseline and 24 weeks
Intervention | Scores on a scale (Mean) |
---|
| Physical Component Summary [n=139, 137] | Mental Component Summary [n=139, 137] | Bodily Pain Score [n=141, 137] | General Health Score [n=139, 137] | Mental Health Score [n=141, 137] | Physical functioning Score [n=141, 137] | Role-Emotional Score [n=141, 137] | Role-Physical Score [n=141, 137] | Social Function Score [n=141, 137] | Vitality Score [n=141, 137] |
---|
Intravenous Cyclophosphamide | 6.4 | 5.7 | 16.8 | 11.5 | 9.8 | 9.3 | 18.4 | 34.0 | 18.2 | 11.6 |
,Mycophenolate Mofetil | 5.2 | 6.7 | 13.4 | 9.1 | 9.3 | 11.6 | 23.4 | 28.6 | 17.7 | 14.2 |
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Induction Phase: Change From Baseline to Week 24 in 24-hour Urine Protein
24-hour urine protein was measured at Baseline and Week 24. (NCT00377637)
Timeframe: Baseline, Week 24
Intervention | mg/day (Mean) |
---|
| Baseline [n=180, 180] | Week 24 [n= 150, 144] | Change from Baseline to Week 24 [n= 146, 142] |
---|
Intravenous Cyclophosphamide | 4451.4 | 1831.6 | -2513.7 |
,Mycophenolate Mofetil | 4208.9 | 1599.0 | -2510.6 |
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Induction Phase: Change From Baseline to Week 24 in Serum Albumin
(NCT00377637)
Timeframe: Baseline, Week 24
Intervention | g/L (Mean) |
---|
| Baseline [n=184, 185] | Week 24 [n=155, 151] | Change from Baseline to Week 24 [n=154, 151] |
---|
Intravenous Cyclophosphamide | 28.6 | 38.3 | 9.0 |
,Mycophenolate Mofetil | 30.5 | 38.4 | 7.5 |
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Induction Phase: Change From Baseline to Week 24 in Serum Creatinine
(NCT00377637)
Timeframe: Baseline, Week 24
Intervention | µmol/L (Mean) |
---|
| Baseline [n= 184, 185] | Week 24 [n= 155, 151] | Change from Baseline to Week 24 [n= 154, 151] |
---|
Intravenous Cyclophosphamide | 92.7 | 83.5 | -5.1 |
,Mycophenolate Mofetil | 108.6 | 77.6 | -18.9 |
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Induction Phase: Change in Renal British Isles Lupus Assessment Group (BILAG) Score
"BILAG indices provide a scoring system for the assessment of lupus disease activity in terms of the need for steroid treatment in 8 organs/systems. Eighty-six items were scored resulting in a classification of A (severe activity), B (moderate activity), C (mild activity), D (no current activity) and E (no activity ever observed) for each organ system. The BILAG individual system summaries were calculated by a program supplied by ADS-Limathon (Sheffield, UK).~The score at baseline was compared to the score at the 24 week endpoint for each treatment group, reported here for the renal system." (NCT00377637)
Timeframe: Baseline, 24 weeks
Intervention | Percentage of participants (Number) |
---|
| Shift from Baseline=A to 24 Week Endpoint=A | Shift from Baseline=A to 24 Week Endpoint=B | Shift from Baseline=A to 24 Week Endpoint=C | Shift from Baseline=A to 24 Week Endpoint=D | Shift from Baseline=B to 24 Week Endpoint=A | Shift from Baseline=B to 24 Week Endpoint=B | Shift from Baseline=B to 24 Week Endpoint=C | Shift from Baseline=B to 24 Week Endpoint=D | Shift from Baseline=C to 24 Week Endpoint=A | Shift from Baseline=C to 24 Week Endpoint=B | Shift from Baseline=C to 24 Week Endpoint=C | Shift from Baseline=C to 24 Week Endpoint=D | Shift from Baseline=D to 24 Week Endpoint=A | Shift from Baseline=D to 24 Week Endpoint=B | Shift from Baseline=D to 24 Week Endpoint=C | Shift from Baseline=D to 24 Week Endpoint=D |
---|
Intravenous Cyclophosphamide | 27.1 | 34.8 | 24.9 | 9.4 | 0.0 | 1.1 | 1.7 | 0.0 | 0.0 | 0.6 | 0.0 | 0.0 | 0.0 | 0.0 | 0.6 | 0.0 |
,Mycophenolate Mofetil | 17.1 | 39.2 | 33.1 | 5.5 | 0.0 | 1.7 | 2.2 | 1.1 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 |
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Induction Phase: Number of Participants Achieving Complete Remission
Number of participants achieving complete remission as defined by return to normal serum creatinine, proteinuria ≤500 mg/24 hours and an inactive urinary sediment (absence of red blood cells, white blood cells or cellular or granular casts) after 24 weeks. (NCT00377637)
Timeframe: 24 weeks
Intervention | participants (Number) |
---|
| Complete Remission - Yes | Complete Remission - No |
---|
Intravenous Cyclophosphamide | 15 | 170 |
,Mycophenolate Mofetil | 16 | 169 |
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Induction Phase: Number of Patients Showing Treatment Response
Treatment response was adjudicated by a blinded clinical endpoints committee (CEC) and defined as: a) Decrease in proteinuria, defined as a decrease in the urine protein to creatinine ratio (UPCr) to <3 in subjects with baseline proteinuria ≥3 UPCr or a decrease in the UPCr by ≥50% in subjects with proteinuria <3 UPCr at Baseline, and b) Stabilization of serum creatinine or improvement. UPCr were derived from the 24 hour urine collection. Patients who did not show a treatment response at Week 24 or who withdrew earlier than Week 24 were considered non-responders. (NCT00377637)
Timeframe: 24 weeks
Intervention | participants (Number) |
---|
| Responder | Non-responder |
---|
Intravenous Cyclophosphamide | 98 | 87 |
,Mycophenolate Mofetil | 104 | 81 |
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Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Not Receiving Rescue Therapy
The primary efficacy parameter was the time to treatment failure, adjudicated by the Clinical Endpoints Committee (CEC), defined as any of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or requirement for rescue therapy to treat deterioration or exacerbation of Lupus nephritis. Kaplan-Meier survival curves were estimated from the observed time to rescue treatment for each patient. The data presented are the percentage of participants who were rescue treatment free at each time interval as estimated by Kaplan-Meier. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | Percentage of participants (Number) |
---|
| Start of Maintenance Phase to Month 3 | Month 3 to Month 6 | Month 6 to Month 9 | Month 9 to Month 12 | Month 12 to Month 15 | Month 15 to Month 18 | Month 18 to Month 21 | Month 21 to Month 24 | Month 24 to Month 27 | Month 27 to Month 30 | Month 30 to Month 33 | Month 33 to Month 36 |
---|
Azathioprine | 99.1 | 95.1 | 93.0 | 91.9 | 88.4 | 87.1 | 83.1 | 83.1 | 81.7 | 80.3 | 78.8 | 75.9 |
,Mycophenolate Mofetil | 100 | 98.2 | 97.2 | 94.2 | 94.2 | 94.2 | 93.1 | 91.9 | 90.8 | 90.8 | 90.8 | 90.8 |
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Maintenance Phase: Events Contributing to the Primary Endpoint: Kaplan-Meier Estimates of Percentage of Participants Renal Flare Free, by Time Interval
A proteinuric flare is defined as a doubling of the urine protein:creatinine ratio, and proteinuria ≥1 g/24 h in patients with urine protein ≤0.5 g/24 h at the end of the induction phase, or proteinuria ≥2 g/24 h if urine protein was >0.5 g/24 h at the end of the induction phase. A nephritic flare is defined as a 25% increase in serum creatinine accompanied by 1 or more of the following: (a) simultaneous doubling of the proteinuria reaching a minimum of 2 g/24 h (b) new/increased hematuria or (c) the appearance of cellular casts. All flares were adjudicated by a clinical endpoints committee. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | Percentage of participants (Number) |
---|
| Start of Maintenance Phase to Month 3 | Month 3 to Month 6 | Month 6 to Month 9 | Month 9 to Month 12 | Month 12 to Month 15 | Month 15 to Month 18 | Month 18 to Month 21 | Month 21 to Month 24 | Month 24 to Month 27 | Month 27 to Month 30 | Month 30 to Month 33 | Month 33 to Month 36 |
---|
Azathioprine | 97.2 | 90.3 | 87.2 | 85.0 | 82.8 | 79.2 | 78.0 | 75.5 | 74.2 | 74.2 | 72.9 | 70.1 |
,Mycophenolate Mofetil | 98.2 | 94.6 | 90.8 | 87.8 | 87.8 | 86.8 | 86.8 | 86.8 | 86.8 | 86.8 | 85.6 | 85.6 |
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Maintenance Phase: Kaplan-Meier Estimates of Percentage of Participants Treatment Failure Free, by Time Interval
Treatment Failure was adjudicated by a clinical endpoints committee and was defined as the time to the earliest occurrence of any one of the following: death, end stage renal disease, sustained doubling of serum creatinine, renal flare, or a requirement for rescue therapy for exacerbation or deterioration of Lupus nephritis. Kaplan-Meier survival curves were estimated from the observed time to treatment failure for each patient. The data presented are the percentage of participants who were treatment-failure free at each time interval as estimated by Kaplan-Meier. (NCT00377637)
Timeframe: From the start of the Maintenance Phase to Month 36
Intervention | Percentage of participants (Number) |
---|
| Start of Maintenance Phase to Month 3 | Month 3 to Month 6 | Month 6 to Month 9 | Month 9 to Month 12 | Month 12 to Month 15 | Month 15 to Month 18 | Month 18 to Month 21 | Month 21 to Month 24 | Month 24 to Month 27 | Month 27 to Month 30 | Month 30 to Month 33 | Month 33 to Month 36 |
---|
Azathioprine | 97.2 | 89.3 | 86.2 | 83.0 | 77.5 | 74.1 | 70.7 | 68.3 | 67.1 | 65.9 | 63.4 | 58.6 |
,Mycophenolate Mofetil | 98.2 | 93.7 | 89.9 | 86.0 | 86.0 | 84.9 | 84.9 | 83.9 | 82.8 | 82.8 | 81.7 | 81.7 |
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Survival
The number of participants who survived treatment (NCT00282412)
Timeframe: up to 5 years
Intervention | Participants (Count of Participants) |
---|
Hematopoietic Stem Cell Transplantation | 2 |
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Percentage of Participants With BCAR at Month 12 Assessed by Local Review
"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Day 365 was used for the analyses at 12 months. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 12 months
Intervention | percentage of participants (Number) |
---|
Tacrolimus/MMF/Basiliximab | 15.4 |
Alefacept QW/Tacrolimus/MMF | 29.0 |
Alefacept QW/Tacrolimus | 20.2 |
Alefacept QOW/Tacrolimus/MMF | 18.1 |
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Percentage of Participants With Biopsy-confirmed Acute Rejection (BCAR) at Month 6 Assessed by Local Review
"Rejection episodes were confirmed by biopsy by the clinical site pathologist. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Day 182 was used for the analyses at 6 months. Lost to follow-up or patients with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Tacrolimus/MMF/Basiliximab | 12.7 |
Alefacept QW/Tacrolimus/MMF | 26.3 |
Alefacept QW/Tacrolimus | 18.8 |
Alefacept QOW/Tacrolimus/MMF | 16.7 |
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Time to First BCAR Assessed by Central Review
The time to first BCAR (central review) was calculated as the first biopsy date in which the central reviewer confirmed an acute rejection minus the date of skin closure +1. Only participants with a BCAR are included in the analysis. (NCT00543569)
Timeframe: 12 months
Intervention | days (Median) |
---|
Tacrolimus/MMF/Basiliximab | 19 |
Alefacept QW/Tacrolimus/MMF | 10 |
Alefacept QW/Tacrolimus | 12 |
Alefacept QOW/Tacrolimus/MMF | 11.5 |
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Time to First BCAR Assessed by Local Review
The time to first BCAR (local review) was calculated as the first biopsy date in which the local reviewer confirmed an acute rejection minus the date of skin closure +1. Only participants with a BCAR are included in the analysis. (NCT00543569)
Timeframe: 12 months
Intervention | days (Median) |
---|
Tacrolimus/MMF/Basiliximab | 9 |
Alefacept QW/Tacrolimus/MMF | 12 |
Alefacept QW/Tacrolimus | 19 |
Alefacept QOW/Tacrolimus/MMF | 12.5 |
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Change From Week 4 in GFR by Iothalamate Clearance at Month 6
The glomerular filtration rate was measured directly using iothalamate clearance. (NCT00543569)
Timeframe: Week 4 and Month 6
Intervention | mL/min per 1.73 m^2 (Mean) |
---|
| Week 4 | Change at Month 6 (N=48, 45, 45, 47) |
---|
Alefacept QOW/Tacrolimus/MMF | 52.09 | 6.60 |
,Alefacept QW/Tacrolimus | 44.36 | 3.56 |
,Alefacept QW/Tacrolimus/MMF | 56.51 | 3.47 |
,Tacrolimus/MMF/Basiliximab | 48.00 | 5.81 |
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Change From Week 4 in Glomerular Filtration Rate Estimated by the MDRD Method at Month 6 and Month 12
The glomerular filtration rate (GFR) was calculated using the Modification of Diet in Renal Disease (MDRD) method. (NCT00543569)
Timeframe: Week 4, Month 6 and Month 12
Intervention | mL/min per 1.73 m^2 (Mean) |
---|
| Week 4 | Change at Month 6 (N=65, 66, 67, 66) | Change at Month 12 (n=66, 64, 64, 66) |
---|
Alefacept QOW/Tacrolimus/MMF | 58.0 | 2.5 | 2.7 |
,Alefacept QW/Tacrolimus | 51.6 | 8.3 | 9.1 |
,Alefacept QW/Tacrolimus/MMF | 59.3 | 3.2 | 3.3 |
,Tacrolimus/MMF/Basiliximab | 54.7 | 5.7 | 8.9 |
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Change From Week 4 in Serum Creatinine at Month 6 and 12
(NCT00543569)
Timeframe: Week 4 and Month 6 and 12
Intervention | mg/dL (Mean) |
---|
| Week 4 | Change at Month 6 (N=69, 69, 68, 70) | Change at Month 12 (N=67, 67, 65, 68) |
---|
Alefacept QOW/Tacrolimus/MMF | 1.5 | -0.0 | 0.1 |
,Alefacept QW/Tacrolimus | 1.6 | -0.3 | -0.2 |
,Alefacept QW/Tacrolimus/MMF | 1.6 | -0.2 | -0.2 |
,Tacrolimus/MMF/Basiliximab | 1.5 | -0.0 | -0.1 |
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Gastrointestinal Quality of Life Index Score Over Time
The impact of gastrointestinal (GI) symptoms on health-related quality of life was assessed using the Gastrointestinal Quality of Life Index (GIQLI) symptom severity score. The GIQLI is a 36-item self-administered questionnaire that assesses the impact of gastrointestinal symptoms during the past 2 weeks on a scale from 0 (all of the time) to 4 (never). Possible overall scores ranged from 0 to 4, with higher scores indicating a better quality of life according to the different symptomatic criteria. (NCT00543569)
Timeframe: Months 1, 3, 6, and 12
Intervention | units on a scale (Mean) |
---|
| Month 1 (N=63, 60, 59, 58) | Month 3 (N=53, 52, 53, 54) | Month 6 (N=58, 61, 57, 59) | Month 12 (N=62, 60, 52, 58) |
---|
Alefacept QOW/Tacrolimus/MMF | 2.98 | 3.16 | 3.17 | 3.19 |
,Alefacept QW/Tacrolimus | 3.00 | 3.11 | 3.24 | 3.26 |
,Alefacept QW/Tacrolimus/MMF | 2.97 | 3.29 | 3.26 | 3.18 |
,Tacrolimus/MMF/Basiliximab | 2.85 | 2.96 | 3.05 | 3.13 |
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Gastrointestinal Symptom Rating Scale Scores Over Time
The impact of gastrointestinal (GI) symptoms on health-related quality of life was assessed using the Gastrointestinal Symptom Rating Scale Scores (GSRS). The GSRS a 15-item self-administered questionnaire that assesses the impact of gastrointestinal symptoms during the past week on a scale from 1 (no discomfort at all) to 7 (very severe discomfort). Possible overall scores range from 1 to 7, with lower scores indicating a better quality of life with respect to gastrointestinal symptoms. (NCT00543569)
Timeframe: Months 1, 3, 6, and 12
Intervention | units on a scale (Mean) |
---|
| Month 1 (N=70, 61, 61, 64) | Month 3 (N=58, 55, 53, 56) | Month 6 (N=63, 67, 62, 63) | Month 12 (N=63, 65, 53, 65) |
---|
Alefacept QOW/Tacrolimus/MMF | 1.48 | 1.34 | 1.42 | 1.57 |
,Alefacept QW/Tacrolimus | 1.59 | 1.47 | 1.49 | 1.52 |
,Alefacept QW/Tacrolimus/MMF | 1.43 | 1.42 | 1.41 | 1.42 |
,Tacrolimus/MMF/Basiliximab | 1.72 | 1.52 | 1.63 | 1.63 |
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Graft Survival at Month 6 and Month 12
"Graft survival was defined as any participant who was known to have a functioning graft (i.e., not graft loss) at 6 months and 12 months after the skin closure date. Graft loss was defined as patient death, retransplant, permanent return to dialysis (dialysis greater than 30 days) or transplant nephrectomy.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 92.3 | 85.8 |
,Alefacept QW/Tacrolimus | 96.0 | 86.5 |
,Alefacept QW/Tacrolimus/MMF | 93.5 | 90.9 |
,Tacrolimus/MMF/Basiliximab | 96.2 | 87.3 |
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Patient Survival at Month 6 and Month 12
"Patient survival is any participant who is known to be alive 6 months and 12 months after the skin closure date.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 93.6 | 89.7 |
,Alefacept QW/Tacrolimus | 97.3 | 87.8 |
,Alefacept QW/Tacrolimus/MMF | 94.8 | 92.2 |
,Tacrolimus/MMF/Basiliximab | 96.2 | 87.3 |
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Percentage of Participants With Anti-lymphocyte-treated Rejection at Months 6 and 12
"Participants with histologically proved Banff Grade II or III rejection could receive anti-rejection therapy with anti-lymphocyte antibodies per institutional protocol.~The use of anti-lymphocyte antibody therapy at any time during a suspected or proven rejection episode for the treatment of acute rejection was considered an event." (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 7.7 | 7.7 |
,Alefacept QW/Tacrolimus | 12.0 | 12.0 |
,Alefacept QW/Tacrolimus/MMF | 20.8 | 20.8 |
,Tacrolimus/MMF/Basiliximab | 6.3 | 6.3 |
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Percentage of Participants With BCAR at Month 6 and 12 Assessed by Central Review
"Rejection episodes were confirmed by biopsy by a central reviewer. Biopsies were graded according to the 2005 Banff criteria. All biopsies (T-cell and/or antibody mediated) of grade 1 or higher were considered a BCAR.~The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit." (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 14.2 | 15.5 |
,Alefacept QW/Tacrolimus | 12.1 | 12.1 |
,Alefacept QW/Tacrolimus/MMF | 18.3 | 19.7 |
,Tacrolimus/MMF/Basiliximab | 7.7 | 7.7 |
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Percentage of Participants With Clinically Treated Acute Rejection at Month 6 and Month 12
Patients who received immunosuppressive medications for the treatment of suspected or BCAR were considered to have a clinically-treated acute rejection. (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 23.1 | 23.1 |
,Alefacept QW/Tacrolimus | 29.3 | 30.7 |
,Alefacept QW/Tacrolimus/MMF | 33.8 | 35.1 |
,Tacrolimus/MMF/Basiliximab | 19.0 | 20.3 |
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Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Central Review
Efficacy failure is defined as death, graft failure (permanent return to dialysis [>30 days] or retransplant), BCAR according to central review, or lost to follow-up. (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 20.5 | 25.6 |
,Alefacept QW/Tacrolimus | 16.0 | 21.3 |
,Alefacept QW/Tacrolimus/MMF | 22.1 | 26.0 |
,Tacrolimus/MMF/Basiliximab | 11.4 | 19.0 |
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Percentage of Participants With Efficacy Failure at 6 and 12 Months Assessed by Local Review
Efficacy failure is defined as death, graft failure (permanent return to dialysis [>30 days] or retransplant), BCAR according to local review, or lost to follow-up. (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 23.1 | 29.5 |
,Alefacept QW/Tacrolimus | 22.7 | 29.3 |
,Alefacept QW/Tacrolimus/MMF | 29.9 | 33.8 |
,Tacrolimus/MMF/Basiliximab | 15.2 | 25.3 |
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Percentage of Participants With Multiple Rejection Episodes at Months 6 and 12
All participants were evaluated for the incidence of multiple rejection episodes (clinically treated and/or BCAR as assessed by the local reviewer) through 6 months and 12 months. (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 2.6 | 3.8 |
,Alefacept QW/Tacrolimus | 4.0 | 4.0 |
,Alefacept QW/Tacrolimus/MMF | 3.9 | 7.8 |
,Tacrolimus/MMF/Basiliximab | 1.3 | 1.3 |
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Percentage of Participants With Treatment Failure at Month 6 and 12
Treatment failure was defined as death, graft loss, BCAR (local review), lost to follow-up or early discontinuation of treatment regimen. The Kaplan-Meier estimates at Days 182 and 365 were used for the analyses at 6 months and 12 months respectively. Lost to follow-up or participants with missing outcomes were censored at their last follow up visit. (NCT00543569)
Timeframe: 6 months and 12 months
Intervention | percentage of participants (Number) |
---|
| Month 6 | Month 12 |
---|
Alefacept QOW/Tacrolimus/MMF | 29.5 | 34.7 |
,Alefacept QW/Tacrolimus | 38.4 | 45.2 |
,Alefacept QW/Tacrolimus/MMF | 37.7 | 45.5 |
,Tacrolimus/MMF/Basiliximab | 26.6 | 35.5 |
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Achieving Full Donor Chimerism
Achieving full donor chimerism (donor T cells >95%): Blood was sent for donor cell percentage measured by short tandem repeat (STR) at post-transplant Day 30; Day 60; Day 90; Day 120; Day 180; Day 270; and Day 360. Full donor chimerism is defined as donor CD3+ cells > 95%. (NCT00481832)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
T & B Cell Mobilization Auto & Allo HCT | 10 |
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Event-free Survival (EFS)
"Event-free survival (EFS) as determined for participants who receive both planned transplants, for a minimum of 3 years.~Events are defined as disease progression/relapse and death of all causes." (NCT00481832)
Timeframe: 3 years
Intervention | percentage of participants (Number) |
---|
T & B Cell Mobilization Auto & Allo HCT | 35 |
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Incidence of Acute Graft Versus Host Disease (GvHD)
The development of GvHD in vaccinated patients of any grade and at 6 months. (NCT00481832)
Timeframe: 6 Months
Intervention | Participants (Count of Participants) |
---|
T & B Cell Mobilization Auto & Allo HCT | 3 |
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Incidence of Chemotherapy-associated Pneumonitis
Interstitial pneumonitis (IP) is a risk associated with high-dose carmustine (BCNU) or other chemotherapy drugs used for transplantation. IP is diagnosed by 1) a decrease of >25% in DLCO compared with pre-transplant PFT DLCO values or 2) a drop of 7% or more in oxygen saturation after exertion. (NCT00481832)
Timeframe: 3 years
Intervention | Participants (Count of Participants) |
---|
T & B Cell Mobilization Auto & Allo HCT | 16 |
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Incidence of Chronic Graft Versus Host Disease (GvHD)
The development of GvHD in vaccinated patients of any grade at 6 months. (NCT00481832)
Timeframe: 3 years
Intervention | Participants (Count of Participants) |
---|
T & B Cell Mobilization Auto & Allo HCT | 3 |
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Overall Mortality Rate
Overall mortality is determined by Kaplan-Meier estimation. The overall morality rate is expressed as the percentage of patients who died for any reason, including disease-related death. (NCT00481832)
Timeframe: 3 years
Intervention | percentage of participants (Number) |
---|
T & B Cell Mobilization Auto & Allo HCT | 56 |
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Overall Survival (OS)
Overall Survival (OS) 3 years after transplant, for participants who received both transplants, as determined by Kaplan-Meier estimation. (NCT00481832)
Timeframe: 3 years
Intervention | percentage of participants (Number) |
---|
T & B Cell Mobilization Auto & Allo HCT | 57 |
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Relapse Rate
Relapse rate (disease recurrence) 3 years after transplant, for participants who received both transplants, as determined by Kaplan-Meier estimation. (NCT00481832)
Timeframe: 3 years
Intervention | percentage of participants (Number) |
---|
T & B Cell Mobilization Auto & Allo HCT | 27 |
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Area Under the Curve (AUC) From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid (MPA)
AUC (0-12) = Area under the plasma concentration versus time curve from time zero (pre-dose) to 12 hours post-dose, measured in microgram-hours per milliliter (mcg*h/mL) (NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | mcg*h/mL (Mean) |
---|
Myfortic - Fed State | 56.5 |
Myfortic - Fasting State | 63.9 |
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Area Under the Curve From Time Zero to 12 Hours Post-Dose [AUC (0-12)] of Mycophenolic Acid Glucuronide (MPAG)
AUC (0-12) = Area under the plasma concentration versus time curve from time zero (pre-dose) to 12 hours post-dose, measured in microgram-hours per milliliter (mcg*h/mL) (NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | mcg*h/mL (Mean) |
---|
Myfortic - Fed State | 967.5 |
Myfortic - Fasting State | 962.2 |
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Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid (MPA)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | micrograms per milliliter (Mean) |
---|
Myfortic - Fed State | 12.8 |
Myfortic - Fasting State | 18.7 |
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Maximum Observed Plasma Concentration (Cmax) of Mycophenolic Acid Glucuronide (MPAG)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | micrograms per milliliter (Mean) |
---|
Myfortic - Fed State | 104.9 |
Myfortic - Fasting State | 113.0 |
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Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid (MPA)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | micrograms per milliliter (Mean) |
---|
Myfortic - Fed State | 2.2 |
Myfortic - Fasting State | 1.7 |
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Minimum Observed Plasma Concentration (Cmin) of Mycophenolic Acid Glucuronide (MPAG)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | micrograms per milliliter (Mean) |
---|
Myfortic - Fed State | 59.4 |
Myfortic - Fasting State | 57.0 |
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Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid (MPA)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | hours (Median) |
---|
Myfortic - Fed State | 5 |
Myfortic - Fasting State | 3 |
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Time to Reach Maximum Observed Plasma Concentration (Tmax) of Mycophenolic Acid Glucuronide (MPAG)
(NCT00585468)
Timeframe: 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12 hours post-dose
Intervention | hours (Median) |
---|
Myfortic - Fed State | 3 |
Myfortic - Fasting State | 3 |
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GI Mucosal Lesions Change and Clinical Symptoms Using The Gastrointestinal Symptom Rating Scale (GSRS) Score
"The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~A higher GSRS indicate worse symptoms and a difference between D30 and last SBCE scores greater or equal to 0.3 can be considered as a clinically significant improvement in the symptoms." (NCT00652834)
Timeframe: one month
Intervention | GSRS score (Mean) |
---|
| baseline | day 30 |
---|
Kidney Transplant Recipients With GI Symptoms | 2.99 | 2.19 |
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Reciprocal Creatinine Slope Between Month 6 and Month 18
Reciprocal Creatinine Slope is an indication of renal function over time with a higher slope value indicating an improvement in renal function. (NCT00862979)
Timeframe: Between Month 6 and Month 18
Intervention | 1/(μmol/L)/(hour) (Mean) |
---|
CNI-regimen | 0.045 |
CNI-free-regimen | 0.403 |
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Calculated Glomerular Filtration Rate (cGFR) According to Cockcroft-Gault at Month 12 and 18
Calculated Glomerular Filtration Rate (cGFR) according to Cockcroft-Gault at Month 12 and 18. For men: GFR=(140-Age) x Body weight (kg) / 72 x Serum Creatinine (mg/dl) For women: GFR=0.85 (140 -Age) x Body weight(kg)/ 72 x Serum Creatinine (mg/dl) (NCT00862979)
Timeframe: Month 12 and 18
Intervention | mL/min (Mean) |
---|
| Month 12 | Month 18 |
---|
CNI-free-regimen | 69.8 | 66.9 |
,CNI-regimen | 54.2 | 54.2 |
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Occurrence of Major Cardiac Events (MACE) From Month 6 to 18
Major cardiac events (MACE) was defined as one of the following: any death, myocardial infarction, coronary artery bypass grafting (NCT00862979)
Timeframe: Month 6 to Month 18
Intervention | Occurences (Number) |
---|
| Myocardial infarction | Death |
---|
CNI-free-regimen | 0 | 1 |
,CNI-regimen | 1 | 0 |
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Occurrence of Treatment Failures From Month 6 to 9 and Month 9 to 18
Treatment failure was defined as composite endpoint of biopsy proven acute rejection of ISHLT 1990 grade ≥ 3A resp. ISHLT 2004 grade ≥ 2R, acute rejection episodes associated with hemodynamic compromise, graft loss / re-transplant, death, loss to follow up (at least one condition must be present). If participant had an occurrence in each period it was counted for each period. (NCT00862979)
Timeframe: Month 6 to Month 9; Month 9 to Month 18
Intervention | Occurences (Number) |
---|
| Month 6 to Month 9 Treatment failure - all reasons | Month 9 to Month 18 Treatment failure-all reasons |
---|
CNI-free-regimen | 4 | 15 |
,CNI-regimen | 1 | 3 |
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Serum Creatinine at Month 6, 8, 9, 10 12 and 18
Serum Creatinine is an indicator of renal function measured in the blood (NCT00862979)
Timeframe: Month 6, 8, 9, 10 12 and 18
Intervention | μmol/L (Mean) |
---|
| Month 6 | Month 8 | Month 9 | Month 10 | Month 12 | Month 18 |
---|
CNI-free-regimen | 1.49 | 1.27 | 1.24 | 1.20 | 1.22 | 1.27 |
,CNI-regimen | 1.53 | 1.44 | 1.58 | 1.53 | 1.53 | 1.50 |
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The Number of Subjects Who Experience a Change From Baseline in Their Panel of Reactive Antibody (PRA) Titers at 12 Months Post Rituximab Infusion.
(NCT00446251)
Timeframe: Month 12 from start of study
Intervention | Participants (Number) |
---|
Rituximab Infusion and Mycophenolate Mofetil Group | 6 |
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The Number of Subjects Who Experience a Decrease in Their Panel of Reactive Antibodies (PRA) at 6 Months Post Rituximab Infusion.
the number of subjects who experience a decrease in their Panel of Reactive Antibodies (PRA) at 6 months and 12 months post Rituximab infusion (NCT00446251)
Timeframe: Month 6 from start of study
Intervention | Participants (Number) |
---|
Rituximab Infusion and Mycophenolate Mofetil Group | 6 |
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The Number of Subjects With a Negative Crossmatch at the Time of Transplant.
(NCT00446251)
Timeframe: Month 12 from start of study
Intervention | Participants (Number) |
---|
Rituximab Infusion and Mycophenolate Mofetil Group | 0 |
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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
Intervention | participants (Number) |
---|
Related BM PBSC | 16 |
Unrelated BM PBSC | 18 |
Unrelated Cord Blood | 10 |
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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
Intervention | probability (Mean) |
---|
Related BM PBSC | 0.501 |
Unrelated BM PBSC | 0.395 |
Unrelated Cord Blood | 0.438 |
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Change From Baseline in Corrected Creatinine Clearance (mL/Min) at Week 52
Corrected creatinine clearance was calculated using the Cockcroft and Gault formula: For adult males, creatinine clearance in mL/min = [(140 - age in years) * (weight in kg] divided by [72 * serum creatinine in mg/dL]. For adult females, creatinine clearance in mL/min = 0.85 * [(140 - age in years) * (weight in kg)] divided by (72 * serum creatinine in mg/dL). (NCT00717314)
Timeframe: Week 52
Intervention | mL/min (Mean) |
---|
MMF, 50% CNI | 6.551 |
MMF, 75% CNI | 6.442 |
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Percentage of Participants Experiencing Acute Rejection, Graft Loss, Death, or a Decrease From BL in Creatinine Clearance of ≥20% at Week 52
The percentage of participants who experienced at least 1 of the following: a ≥20% decrease from BL in creatinine clearance, acute rejection, graft loss, or death 1 year after randomization. (NCT00717314)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
MMF, 50% CNI | 8.1 |
MMF, 75% CNI | 8.8 |
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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) at Week 52
BPAR was graded according to Banff criteria. (NCT00717314)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
MMF, 50% CNI | 0.0 |
MMF, 75% CNI | 5.9 |
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Percentage of Participants With Decrease in Glomerular Filtration Rate (GFR) of Greater Than 20%
The percentage of participants with a greater than 20% decrease of GFR during the 1-year period following regimen adjustment. Cockcroft and Gault formula was used for calculated creatinine clearance. (NCT00717314)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
MMF, 50% CNI | 5.4 |
MMF, 75% CNI | 0.0 |
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Changes From Baseline in Creatinine Clearance (Milliliters Per Minute [mL/Min])
Creatinine clearance calculated using the Cockcroft and Gault formula: For adult males, creatinine clearance in mL/min equaled (=) [(140 minus (-) age in years) multiplied by (*) (weight in kilograms (kg)] divided by [72 * serum creatinine in milligrams per deciliter (mg/dL)]. For adult females, creatinine clearance in mL/min = 0.85 * [(140 - age in years) * (weight in kg)] divided by (72 * serum creatinine in mg/dL). (NCT00717314)
Timeframe: Baseline and Weeks 16, 28, and 40
Intervention | mL/min (Mean) |
---|
| Baseline (n=36,31) | Change at Week 16 (n=37,33) | Change at Week 28 (n=34,33) | Change at Week 40 (n=35,27) |
---|
MMF, 50% CNI | 1.385 | 4.884 | 5.333 | 6.131 |
,MMF, 75% CNI | 2.250 | 5.629 | 4.560 | 4.125 |
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Percentage Change in Creatinine Clearance From Baseline
Creatinine clearance was calculated using the Cockcroft and Gault formula. (NCT00717314)
Timeframe: Weeks 16, 28, 40, and 52
Intervention | percentage change from baseline (Mean) |
---|
| Week 16 (n=37,33) | Week 28 (n=34,33) | Week 40 (n=35,27) | Week 52 (n=37,34) |
---|
MMF, 50% CNI | 10.38 | 11.45 | 12.37 | 10.17 |
,MMF, 75% CNI | 8.72 | 7.51 | 7.03 | 11.51 |
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Percentage of Participants With Graft Loss or Death at Week 52
Graft loss was defined for this protocol as re-transplantion or death. (NCT00717314)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
| Graft loss | Death |
---|
MMF, 50% CNI | 0.0 | 0.0 |
,MMF, 75% CNI | 0.0 | 0.0 |
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Event-free Survival (EFS) Per Protocol
Event-free survival (EFS) through 4 years, as assessed in participants with poor-risk recurrent or primary refractory DLBCL treated with TLI and ATG followed by matched allogeneic hematopoietic cell transplantation as a consolidation to HCT. Event is defined as tumor progression or death. (NCT00482053)
Timeframe: 48 months
Intervention | months (Median) |
---|
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL | 48 |
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Incidence of Chronic Graft vs Host Disease (GvHD)
The incidence of chronic graft vs host disease (GvHD) is reported as any events within 3 years. Note that GvHD was assessed per investigator judgement. There was no protocol-specified criteria of GvHD. (NCT00482053)
Timeframe: 3 years
Intervention | Participants (Count of Participants) |
---|
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL | 0 |
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Overall Survival (OS)
To evaluate the overall and transplant related mortality rate, reported as the number of subjects remaining alive 3 years after transplant. (NCT00482053)
Timeframe: 3 years
Intervention | Participants (Count of Participants) |
---|
Auto-HCT Followed by Allo-HCT for Poor-risk DLBCL | 2 |
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Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)
"Assessed as the incidence of grade 2 to 4 acute graft-vs-host disease (GvHD) at Day 100 post-transplant.~Stage of Acute GvHD was assessed as follows.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea > 500 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea >1000 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus~Grade of Acute GvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage" (NCT01220297)
Timeframe: 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo | 0 |
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo | 1 |
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Acute GvHD (Grade 3 to 4)
"Assessed as the incidence of grade 3 to 4 acute GvHD at Day 100 post-transplant.~Stage of Acute GvHD was assessed as follows.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea > 500 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea >1000 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus~Grade of Acute GvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2 to 3 Liver + No Gut stage" (NCT01220297)
Timeframe: 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo | 0 |
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo | 1 |
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Disease-free Survival (DFS)
Assessed as survival without recurrence of disease (NCT01220297)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo | 0 |
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo | 0 |
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Overall Survival
Overall survival is defined as time from enrollment to time of death or last follow-up, within 2 years. (NCT01220297)
Timeframe: 2 years
Intervention | Days (Median) |
---|
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo | 405 |
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Veno-occlusive Disease (VoD)
Assessed as the incidence of veno-occlusive disease (VoD) at 100 days post-transplant. (NCT01220297)
Timeframe: 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
GvHD Prophylaxis of Sirolimus & MMF After BCNU+VP16+Cyclo | 0 |
GvHD Prophylaxis of Sirolimus & MMF After FTBI + Cyclo | 1 |
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cGVHD
Chronic graft vs host disease (NCT01010217)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Haplo Arm | 11 |
1AgMM Related/Unrelated Donors | 7 |
MUD Donor | 0 |
Second Transplant | 0 |
Myelofibrosis | 0 |
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Disease Free Survival
Participants who have survived without their original disease. (NCT01010217)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Haplo Arm | 50 |
1AgMM Related/Unrelated Donors | 24 |
MUD Donor | 12 |
Second Transplant | 0 |
Myelofibrosis | 0 |
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Engraftments
(NCT01010217)
Timeframe: Day 28
Intervention | Participants (Count of Participants) |
---|
Haplo Arm | 80 |
1AgMM Related/Unrelated Donors | 50 |
MUD Donor | 21 |
Second Transplant | 0 |
Myelofibrosis | 0 |
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Grade III-IV aGVHD
Acute Graft vs host disease (NCT01010217)
Timeframe: 100 days
Intervention | Participants (Count of Participants) |
---|
Haplo Arm | 11 |
1AgMM Related/Unrelated Donors | 7 |
MUD Donor | 1 |
Second Transplant | 0 |
Myelofibrosis | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Haplo Arm | 8 |
1AgMM Related/Unrelated Donors | 7 |
MUD Donor | 2 |
Second Transplant | 0 |
Myelofibrosis | 0 |
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Blood Pressure
Diastolic pressure (mmHg) (NCT01002339)
Timeframe: 1 year
Intervention | mmHg (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 76.67 |
Tacrolimus With Steroids Minimization | 74.59 |
CsA With Steroid Minimization | 76.64 |
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Blood Pressure
Systolic pressure (mmHg) (NCT01002339)
Timeframe: 1 year
Intervention | mmHg (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 135.36 |
Tacrolimus With Steroids Minimization | 133.97 |
CsA With Steroid Minimization | 136.28 |
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Lipidic Profile (Cholesterol)
Lipidic Profile (total cholesterol) (NCT01002339)
Timeframe: 1 year
Intervention | mg/dl (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 169.05 |
Tacrolimus With Steroids Minimization | 178.24 |
CsA With Steroid Minimization | 168.89 |
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Lipidic Profile (HDL-c)
(NCT01002339)
Timeframe: 1 year
Intervention | mg/dl (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 44.84 |
Tacrolimus With Steroids Minimization | 49.29 |
CsA With Steroid Minimization | 48.35 |
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Lipidic Profile (LDL-c)
(NCT01002339)
Timeframe: 1 year
Intervention | mg/dl (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 94.00 |
Tacrolimus With Steroids Minimization | 95.43 |
CsA With Steroid Minimization | 88.65 |
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Lipidic Profile (Triglycerides)
(NCT01002339)
Timeframe: 1 year
Intervention | mg/dl (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 159.44 |
Tacrolimus With Steroids Minimization | 145.59 |
CsA With Steroid Minimization | 160.78 |
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Number of Antihypertensive Drugs Patients Reported Taking.
(NCT01002339)
Timeframe: 1 year
Intervention | number of antihypertensive drugs (Median) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 2 |
Tacrolimus With Steroids Minimization | 2 |
CsA With Steroid Minimization | 2 |
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Patients Treated With Insulin or Oral Antidiabetic Drugs
(NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 20 |
Tacrolimus With Steroids Minimization | 15.4 |
CsA With Steroid Minimization | 2.6 |
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Percentage of Patients Using Acetylsalicylic Acid (ASA)
(NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 53.9 |
Tacrolimus With Steroids Minimization | 48.7 |
CsA With Steroid Minimization | 52.8 |
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Percentage of Patients Using Statins
(NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 56. |
Tacrolimus With Steroids Minimization | 61.5 |
CsA With Steroid Minimization | 73.7 |
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Primary Outcome Measure (Glucose Intolerance)
Glycemia >=140 and <200 mg/dl, 2 hours after a standard oral glucose tolerance test. Measured values: glucose intolerance at 1 year defined by ADA criteria. (NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 26.9 |
Tacrolimus With Steroids Minimization | 31.0 |
CsA With Steroid Minimization | 33.3 |
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Proteinuria
(NCT01002339)
Timeframe: 1 year
Intervention | mg/day (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 208 |
Tacrolimus With Steroids Minimization | 241 |
CsA With Steroid Minimization | 343.2 |
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Rejection
Biopsy proven acute rejection. Measured variable: Rate of Biopsy proven acute rejection. (NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 11.4 |
Tacrolimus With Steroids Minimization | 4.8 |
CsA With Steroid Minimization | 21.4 |
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Renal Function
Estimated Glomerular Filtration Rate (ml/min/1.73 m^2) (NCT01002339)
Timeframe: 1 year
Intervention | ml/min/1.73 m^2 (Mean) |
---|
Tacrolimus With Rapid Steroid Withdrawal | 51.9 |
Tacrolimus With Steroids Minimization | 47.4 |
CsA With Steroid Minimization | 44.6 |
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"Primary Outcome Measure New Onset Diabetes After Renal Transplantation (NODAT)"
American Diabetes Association criteria (ADA) including an oral glucose tolerance test. (NCT01002339)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
| % of patients with NODAT | % of patients without NODAT |
---|
CsA With Steroid Minimization | 7.9 | 92.1 |
,Tacrolimus With Rapid Steroid Withdrawal | 34.1 | 65.9 |
,Tacrolimus With Steroids Minimization | 23.1 | 76.9 |
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Fibrosis Score, as Measured by Thoracic High Resolution Computerized Tomography (HRCT)
Imaging of the whole lung (WL) is performed using a volumetric high resolution computerized tomography (HRCT) scan, which is then analyzed using a computer algorithm to determine the percentage of overall pixels exhibiting features characteristic for quantitative lung fibrosis (QLF). Higher percentages for QLF-WL therefore represent greater involvement by lung fibrosis. (NCT00883129)
Timeframe: Measured at baseline and Month 24
Intervention | % of lung exhibiting QLF (Mean) |
---|
| Baseline | Month 24 |
---|
Cyclophosphamide Arm | 8.91 | 8.48 |
,Mycophenolate Arm | 8.25 | 7.99 |
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Forced Vital Capacity (FVC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
The primary outcome is the course over time from baseline to 24 months for the FVC %-predicted. The FVC %-predicted represents the adjusted volume of air (adjusted as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity) that can be forcibly exhaled from the lungs after taking the deepest breath possible. The FVC %-predicted is reduced in patients with interstitial lung disease and is used as a measure of lung involvement and disease severity. (NCT00883129)
Timeframe: Measured at study Baseline and Months 3, 6, 12, 15, 18, 21, and 24
Intervention | FVC %-pred (Mean) |
---|
| Baseline | Month 3 | Month 6 | Month 9 | Month 12 | Month 15 | Month 18 | Month 21 | Month 24 |
---|
Cyclophosphamide Arm | 66.52 | 67.03 | 67.86 | 69.42 | 69.86 | 71.94 | 72.57 | 72.55 | 70.15 |
,Mycophenolate Arm | 66.52 | 66.22 | 68.02 | 68.11 | 68.43 | 69.84 | 70.57 | 70.87 | 69.65 |
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Single-breath Diffusing Capacity for Carbon Monoxide (DLCO), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
The DLCO is a pulmonary function test that measures the capacity for the lung to carry out gas exchange between the inhaled breath and the pulmonary capillary blood vessels and the DLCO %-predicted represents the DLCO expressed as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity. The DLCO %-predicted is reduced in patients with interstitial lung disease and is used as a measure of disease severity. (NCT00883129)
Timeframe: Measured at study entry and Months 3, 6, 12, 15, 18, 21, and 24
Intervention | DLCO %-pred (Mean) |
---|
| Baseline | Month 3 | Month 6 | Month 9 | Month 12 | Month 15 | Month 18 | Month 21 | Month 24 |
---|
Cyclophosphamide Arm | 54.05 | 51.92 | 50.87 | 51.55 | 53.12 | 53.62 | 55.9 | 54.26 | 52.90 |
,Mycophenolate Arm | 53.99 | 53.38 | 54.86 | 54.13 | 55.32 | 57.77 | 56.62 | 55.47 | 55.31 |
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Skin Involvement, as Measured by the Modified Rodnam Skin Thickness Scores (mRSS)
Skin thickness is quantified using the modified Rodnan measurement method (mRSS), with a scale that ranges from 0 (no skin involvement) to a maximum of 51. The reported skin score is determined by a clinical assessment of skin thickness, which is performed by a trained reader, and represents the sum of individual assessments that are made in each of 17 body areas. Each area is given a score in the range of 0-3 (0 = normal; 1= mild thickness; 2 = moderate; 3 = severe thickness). A higher score represents more severe skin involvement. (NCT00883129)
Timeframe: Measured at baseline and Months 3, 6, 9, 12, 15, 18, 21, and 24
Intervention | mRSS score (Mean) |
---|
| Baseline | Month 3 | Month 6 | Month 9 | Month 12 | Month 15 | Month 18 | Month 21 | Month 24 |
---|
Cyclophosphamide Arm | 14.04 | 12.85 | 11.95 | 10.61 | 9.47 | 9.80 | 9.87 | 8.50 | 7.87 |
,Mycophenolate Arm | 15.32 | 16.03 | 14.37 | 14.33 | 12.45 | 12.43 | 11.98 | 11.22 | 11.40 |
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Tolerability, as Assessed by the Time to Withdrawal From the Study Drug or Meeting Protocol-defined Criteria for Treatment Failure.
The number of participants who remained in the study at the listed time points are reported (NCT00883129)
Timeframe: Continuous assessment from randomization to 24 months
Intervention | Participants (Count of Participants) |
---|
| Baseline | Month 3 | Month 6 | Month 9 | Month 12 | Month 15 | Month 18 | Month 21 | Month 24 |
---|
Cyclophosphamide Arm | 73 | 64 | 56 | 51 | 46 | 44 | 42 | 39 | 38 |
,Mycophenolate Arm | 69 | 66 | 58 | 55 | 52 | 52 | 49 | 49 | 49 |
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Total Lung Capacity (TLC), as a Percent of the Age, Height, Gender, and Ethnicity Adjusted Predicted Value
The TLC represents the total volume of air within the lung after taking the deepest breath possible and the TLC %-predicted represents the TLC expressed as a percentage of the expected normal valued based on the participant's age, height, gender and ethnicity. The TLC %-predicted is reduced in patients with interstitial lung disease and is used as a measure of disease severity. (NCT00883129)
Timeframe: Measured at study entry and Months 6, 12, 18, and 24
Intervention | TLC %-pred (Mean) |
---|
| Baseline | Month 6 | Month 12 | Month 18 | Month 24 |
---|
Cyclophosphamide Arm | 65.49 | 67.39 | 68.25 | 69.63 | 66.97 |
,Mycophenolate Arm | 66.16 | 67.84 | 67.31 | 68.50 | 68.24 |
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Toxicity, as Measured by Adverse Events, Serious Adverse Events, and Death
(NCT00883129)
Timeframe: Measured throughout the 2-year study
Intervention | Participants (Count of Participants) |
---|
| Leukopenia (<2.5x10^3 WBC/microliter) | Neutropenia (<1.0x10^3 neutrophils/microliter) | Anemia (Hgb <10 g/dl) | Thrombocytopenia (<100x10^3 platelets/microliter) | Hematuria (>10 RBC/high power field) | Pneumonia | SAE-Total | SAE-related to treatment | Deaths |
---|
Cyclophosphamide Arm | 30 | 7 | 13 | 4 | 2 | 4 | 22 | 7 | 11 |
,Mycophenolate Arm | 4 | 3 | 8 | 0 | 3 | 5 | 27 | 3 | 5 |
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Transitional Dyspnea Index Score
Change in breathlessness was assessed using the Transitional Dyspnea Index, which compares current symptoms to those at baseline. Total score ranges from - 9 to + 9. The lower the score, the more deterioration in severity of dyspnea. (NCT00883129)
Timeframe: Measured at Months 6, 12, 18, and 24
Intervention | Transitional Dyspnea Index Score (Mean) |
---|
| Month 6 | Month 12 | Month 18 | Month 24 |
---|
Cyclophosphamide Arm | 0.31 | 1.23 | 1.78 | 2.09 |
,Mycophenolate Arm | 0.74 | 1.17 | 0.91 | 1.86 |
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Number of Participants With Cytomegalovirus Infection or Disease
(NCT00336895)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Liver Transplant Subjects | 0 |
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Gastrointestinal Side Effects and Quality of Life (-Subscales of GSRS)
"The GSRS contains 15 items, each rated on a seven- point likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items breakdown into the following five scales: abdominal ( Abdominal pain, hunger pains and nausea): reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome ( borborygmus, abdominal distention, eructation and increased flatus) and constipation syndrome (constipation, hard stools and feeling of incomplete evacuation) The range of the scale for abdominal pain was 3 to 21, reflux 2 to 14, diarrhea 3 to 21, indigestion 4 to 28 and constipation 3 to 21.~Higher values represent more severe discomfort." (NCT00336895)
Timeframe: 12 weeks
Intervention | units on a scale (Mean) |
---|
| Abdominal Pain | Reflux | Indigestion | Diarrhea | Constipation |
---|
Liver Transplant Subjects | 10.78 | 2.64 | 7.08 | 5.44 | 4.56 |
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Gastrointestinal Side Effects and Quality of Life (Total Score of GSRS)
"The gastrointestinal Symptom Rating Scale (GSRS) is a validated scale, the items range from 1= No discomfort at all to 7= Very severe discomfort.~The scale ranges from a minimal value of 15 ( No discomfort at all) to a maximum of 105 ( Very severe discomfort)~The GSRS contains 15 items, each rated on a seven- point likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items breakdown into the following five scales: abdominal ( Abdominal pain, hunger pains and nausea): reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome ( borborygmus, abdominal distention, eructation and increased flatus) and constipation syndrome (constipation, hard stools and feeling of incomplete evacuation)" (NCT00336895)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
Liver Transplant Subjects | 46.000 | 29.0400 | 26.2800 | 25.3600 |
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Comparison by Treatment Assignment in the Number of Biopsy-Proven Acute Rejections Within 12 Months Post Kidney Transplantation
"Biopsy-proven acute renal (kidney) rejection [1, 2].~Diagnosis of acute rejection was made by renal biopsy using the Banff 97 criteria. The Banff 97 diagnostic category for renal allograft biopsies is an international standardized histopathological classification. Acute rejection is defined by a renal biopsy demonstrating a Banff 97 classification of Grade IA or greater, with higher scores indicating more severe rejection[2]~Ref: Racusen LC et al. The Banff 97 working classification of renal allograft pathology. Kidney Int, 55: 713-723, 1999" (NCT00141037)
Timeframe: Up to one year post kidney transplantation procedure
Intervention | Rejection Events (Number) |
---|
Steroid-Free Immunosuppression | 18 |
Steroid-Based Immunosuppression | 19 |
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The Difference in Linear Growth by Treatment Assignment at 1 Year Post Kidney Transplantation
Standardized Z-scores were computed following a formula using an age- and gender-specific calculation provided by the NHANES III 2000 Growth Data set. The Z-score system expresses anthropometric values of height as several standard deviations (SDs) below (e.g., a negative value) or above (a positive value) the reference mean or median value. In this study the measure was used to test whether there is a difference in the change in height between the treatment groups: Steroid-Based versus Steroid-Free (NCT00141037)
Timeframe: One year post kidney transplantation procedure
Intervention | Standard Deviation Score (SDS) (Mean) |
---|
Steroid-Free Immunosuppression | 0.37 |
Steroid-Based Immunosuppression | 0.35 |
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Number of Participants Who Experienced Adverse Events and Death
Participants were monitored for adverse events, serious adverse events and deaths thorughout the prospective and follow-up phases of the study. (NCT00332839)
Timeframe: 12 months
Intervention | Participants (Number) |
---|
| Adverse events (serious and non-serious) | Serious adverse events | Deaths |
---|
Calcineurin Inhibitor (CNI) Group | 44 | 11 | 1 |
,Certican Group | 44 | 12 | 1 |
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Disease Free
Number of participants that were disease free (NCT01093586)
Timeframe: At 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I | 4 |
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Disease Free
Number of participants that were disease free (NCT01093586)
Timeframe: At 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I | 4 |
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Hematologic Engraftment
Number of participants that were able to complete engraftment by day 42. (NCT01093586)
Timeframe: On day +42
Intervention | Participants (Count of Participants) |
---|
Arm I | 10 |
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Incidence of Acute Graft-versus-host Disease (GVHD)
Number of participants that had acute GVHD (NCT01093586)
Timeframe: At 100 days
Intervention | Participants (Count of Participants) |
---|
Arm I | 11 |
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Incidence of Chronic GVHD
Number of participants that have chronic GVHD. Chronic GVHD will be diagnosed and graded on clinical and histological criteria from the Center for International Blood and Marrow Transplant Research (CIBMTR) (NCT01093586)
Timeframe: At 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I | 1 |
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Occurrence of Serious Infections
Number of participants that had infections (NCT01093586)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I | 13 |
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Overall Survival
Number of participants alive at 180 days post engraftment. (NCT01093586)
Timeframe: On day +180
Intervention | Participants (Count of Participants) |
---|
Arm I | 8 |
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Overall Survival
Number of participants that were alive. (NCT01093586)
Timeframe: At 1 year
Intervention | Participants (Count of Participants) |
---|
Arm I | 6 |
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Overall Survival
Number of participants that were alive. (NCT01093586)
Timeframe: At 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I | 4 |
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Recurrence or Relapse
Number of subjects that had disease recurrence (NCT01093586)
Timeframe: one year in patients post UCBT
Intervention | Participants (Count of Participants) |
---|
Arm I | 2 |
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Recurrence or Relapse
Number of subjects that had disease recurrence (NCT01093586)
Timeframe: two years post transplant
Intervention | Participants (Count of Participants) |
---|
Arm I | 2 |
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Complete Response Rate (CRR)
"Complete response rate (CRR) was assessed as all of:~Negative immunoflixation on the serum and urine~Disappearance of any soft tissue plasmacytomas~< 5% plasma cells in bone marrow" (NCT00899847)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Autologous-Allogeneic Hematopoietic Stem Cell Transplant | 3 |
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Event-free Survival (EFS)
To evaluate the graft versus myeloma effect by monitoring rate of event-free survival (EFS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled
Intervention | percentage of participants (Number) |
---|
Autologous-Allogeneic Hematopoietic Stem Cell Transplant | 44 |
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Incidence of Graft Versus Host Disease (GvHD)
To evaluate the incidence acute GvHD of this tandem autologous/allogeneic transplant setting (NCT00899847)
Timeframe: 2 years after the last participant is enrolled.
Intervention | Participants (Count of Participants) |
---|
Autologous-Allogeneic Hematopoietic Stem Cell Transplant | 1 |
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Overall Response Rate (ORR)
Overall response rate (ORR) = Complete Response Rate (CRR) + Partial Response Rate (PRR) (NCT00899847)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant | 7 |
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Overall Survival (OS)
To evaluate the graft versus myeloma effect by monitoring rate of overall survival (OS) (NCT00899847)
Timeframe: 2 years after the last participant is enrolled
Intervention | percentage of participants (Number) |
---|
Autologous-Allogeneic Hematopoietic Stem Cell Transplant | 67 |
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Partial Response Rate (PRR)
"Partial response rate (PRR) was assessed as~> 50% reduction in serum M-protein plus urine M-protein reduction by 90% or < 200 mg/24 hr~If serum M-protein is not measurable, then > 50% reduction in the involved serum free light chain~If involved serum free light chain is not measurable, then > 50% reduction in the bone marrow plasma cell percentage + > 50% reduction in the size of any soft tissue plasmacytoma." (NCT00899847)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Autologous-Allogeneic Peripheral Blood Stem Cell Transplant | 4 |
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Number of Patient Who Gained Remission From the Nephrotic Syndrome
Efficacy of mycophenolate in preventing relapse of nephrotic syndrome secondary to membranous glomerulonephritis on withdrawal of tacrolimus therapy. (NCT00843856)
Timeframe: 10-109 weeks
Intervention | Participants (Count of Participants) |
---|
Tacrolimus | 8 |
Tacrolimus and Mycophenolate Mofetil | 8 |
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Number of Patients Achieved Remission
The degree of remission of proteinuria obtained (complete or partial) The rate of decline of renal function measured by the Modification of Diet in Renal Disease equation for glomerular filtration rate. (NCT00843856)
Timeframe: 6-12 months
Intervention | Participants (Count of Participants) |
---|
Tacrolimus | 16 |
Tacrolimus and Mycophenolate Mofetil | 19 |
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Core Study: Number Participants With Combined Graft Loss, Death or Loss to Follow-up
"The allograft was presumed to be lost on the day the patient starts dialysis and was not able to stop dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss.~A loss to follow-up in graft loss, death or loss to follow-up is a patient who did not experience graft loss or death and whose last day of contact was prior to Day 316, i.e. prior to the Month 12 visit window." (NCT00658320)
Timeframe: 12 months
Intervention | Participants (Number) |
---|
Everolimus + Reduced Dose of Cyclosporine | 5 |
Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine | 3 |
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Core Study: Number of Patients With Composite Efficacy Endpoint
"The composite efficacy endpoint consisted of treated biopsy proven acute rejection (BPAR) episodes, graft loss, death or loss to follow-up. A treated BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III and which was treated with anti-rejection therapy. The allograft was presumed to be lost on the day the patient starts dialysis and was not able to stop dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss.~For the individual components (including loss of follow-up)of the composite endpoint, patients are counted for the first event to occur." (NCT00658320)
Timeframe: 12 months
Intervention | Participants (Number) |
---|
| Composite Efficacy Endpoint | Treated BPAR | Graft Loss | Death | Loss to follow up (see caveats) |
---|
Everolimus + Reduced Dose of Cyclosporine | 7 | 3 | 0 | 0 | 4 |
,Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine | 7 | 5 | 0 | 0 | 2 |
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Extension Study: Cyclosporine Trough Levels
Blood was collected at all visits after Day 3 for trough (collected 5 minutes before study drug dose) cyclosporine levels and was analyzed at a central laboratory using immunoassay. (NCT00658320)
Timeframe: Month 24, Month 48
Intervention | ng/mL (Mean) |
---|
| Month 24 | Month 48 (n=7,0) |
---|
Everolimus + Reduced Dose of Cyclosporine | 54.1 | 34.2 |
,Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine | 105.5 | NA |
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Extension Study: Everolimus Trough Levels
Blood was collected at all visits after Day 3 for trough (collected 5 minutes before study drug dose) everolimus levels and was analyzed at a central laboratory using liquid chromatography mass spectrometry. (NCT00658320)
Timeframe: Month 24, Month 48
Intervention | ng/mL (Mean) |
---|
| Month 24 | Month 48 (n=8) |
---|
Everolimus + Reduced Dose of Cyclosporine | 5.258 | 4.408 |
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Extension Study: Number of Participants With Adverse Events and Serious Adverse Events
Additional information about Adverse Events can be found in the Adverse Event Section. (NCT00658320)
Timeframe: 24 Months
Intervention | Participants (Number) |
---|
| Adverse Events | Serious Adverse Events |
---|
Everolimus + Reduced Dose of Cyclosporine | 50 | 30 |
,Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine | 50 | 30 |
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Extension Study: Number of Participants With Combined Efficacy Endpoint: Graft Loss, Death, Loss to Follow-up and/or Treated Biopsy Proven Acute Rejection (BPAR)
"Graft loss was defined as the day the patient started dialysis and was not able to subsequently be removed from dialysis or re-transplant.~Loss-to-follow was a patient who did not experience a treated BPAR, graft loss or death and whose last day of contact was prior to Month 24.~A Graft Biopsy was done within 48 hours of suspect rejection. Biopsies were read by the local pathologist according to the updated Banff '97 criteria.~Treated BPAR was based on local laboratory biopsy results and was defined as a biopsy Banff criteria graded IA to III that was treated with anti-rejection therapy." (NCT00658320)
Timeframe: 24 Months
Intervention | Participants (Number) |
---|
| Combined Efficacy Endpoint | Treated BPAR | Graft Loss | Death | Loss to follow-up |
---|
Everolimus + Reduced Dose of Cyclosporine | 4 | 3 | 0 | 0 | 1 |
,Mycophenolate Mofetil (MMF) + Standard Dose of Cyclosporine | 5 | 5 | 0 | 0 | 0 |
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Annual Change Rate in Total Plaque Volume (TPV) From Pre-conversion Baseline to 12 Months Post-transplant
Within-subject annual change rate in TPV in the left and right distal common carotid arteries from the pre-conversion baseline to 12 months post kidney transplant as determined by ultrasound. Annual change rate equals (=) (TPV at month 12 post-transplant minus [-] TPV at pre-conversion baseline) divided (/) by imaging interval in years. TPV is the sum of assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline and 12 months post-transplant
Intervention | millimeter cube/year (mmˆ3/year) (Mean) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | -49.31 |
Tacrolimus With Mycophenolate/Prednisone | 0.66 |
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CIMT at Pre-conversion Baseline
Mean CIMT=average of left CIMT and right CIMT. (NCT00311311)
Timeframe: Pre-conversion baseline
Intervention | mm (Mean) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.735 |
Tacrolimus With Mycophenolate/Prednisone | 0.773 |
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TPV at Pre-conversion Baseline
TPV is the sum of the assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline
Intervention | mmˆ3 (Mean) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 68.68 |
Tacrolimus With Mycophenolate/Prednisone | 48.57 |
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Annual Change Rate in Carotid Intima Media Thickness (CIMT) From Pre-conversion Baseline at 12, 18, 24 and 36 Months Post-transplant
Within-subject annual change rate in CIMT as determined by ultrasound. Mean CIMT=average of left CIMT and right CIMT. Annual CIMT Change Rate (mm/year) = (CIMT at Month x Post-transplant Visit - CIMT at Conversion Baseline) / Imaging interval in years. (NCT00311311)
Timeframe: Pre-conversion baseline, and 12, 18, 24 and 36 months post-transplant
Intervention | millimeter/year (mm/year) (Mean) |
---|
| Annual Change Rate at 12 months (n=16,28) | Annual Change Rate at 18 months (n=15,26) | Annual Change Rate at 24 months (n=15,23) | Annual Change Rate at 36 months (n=8,13) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.048 | 0.041 | 0.023 | 0.028 |
,Tacrolimus With Mycophenolate/Prednisone | 0.012 | -0.0002 | 0.015 | -0.007 |
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Annual Rate of Change in TPV From Pre-conversion Baseline to 18, 24 and 36 Months Post Transplant
Within-subject annual change rate in TPV in the left and right distal common carotid arteries from the pre-conversion baseline to 18, 24 and 36 months post kidney transplant as determined by ultrasound. Annual change rate equals (=) (TPV at month 18, 24 and 36 post-transplant minus [-] TPV at pre-conversion baseline) divided (/) by imaging interval in years. TPV is the sum of assessment in left and right distal common carotid arteries. (NCT00311311)
Timeframe: Pre-conversion baseline, and 18, 24 and 36 months post-transplant
Intervention | mmˆ3/year (Mean) |
---|
| Month 18 post-transplant (n=12,12) | Month 24 post-transplant (n=11,14) | Month 36 post-transplant (n=7,11) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 17.15 | 25.28 | 6.62 |
,Tacrolimus With Mycophenolate/Prednisone | 19.71 | 9.10 | 8.98 |
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Change From Pre-conversion Baseline in Adiponectin at Months 12, 24 and 36 Post-transplant
Adiponectin is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates less risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | microgram per milliliter (µg/mL) (Mean) |
---|
| Change at 12 Months post-transplant (n=23,33) | Change at 24 Months post-transplant (n=18,31) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 5.352 | 2.244 | 1.487 |
,Tacrolimus With Mycophenolate/Prednisone | -2.205 | -0.267 | -1.413 |
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Change From Pre-conversion Baseline in Endothelin-1 at Months 12, 24 and 36 Post-transplant
Endothelin-1 is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | pg/mL (Mean) |
---|
| Change at 12 Months post-transplant (n=22,30) | Change at 24 Months post-transplant (n=18,30) | Change at 36 Months post-transplant (n=14,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.294 | 0.426 | 0.411 |
,Tacrolimus With Mycophenolate/Prednisone | 0.028 | 0.138 | -0.104 |
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Change From Pre-conversion Baseline in Fasting Lipid Parameters at 12, 18, 24 and 36 Months Post-transplant
Total Cholesterol (TC), Low Density Lipoprotein (LDL), High Density Lipoprotein (HDL) and Triglyceride (Tg) blood concentrations. Higher levels of TC, LDL and Tg are less desirable. Lower levels of HDL are less desirable. Change for each parameter = value at 12, 18, 24 and 36 months post-transplant - value at pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, and 12, 18, 24 and 36 months post-transplant
Intervention | millimole/liter (mmol/L) (Mean) |
---|
| Change in TC 12 Months post-transplant (n=23,30) | Change in LDL 12 Months post-transplant (n=21,30) | Change in HDL 12 Months post-transplant (n=23,30) | Change in Tg 12 Months post-transplant (n=23,30) | Change in TC 18 Months post-transplant (n=21,29) | Change in LDL 18 Months post-transplant (n=20,29) | Change in HDL 18 Months post-transplant (n=21,29) | Change in Tg 18 Months post-transplant (n=21,29) | Change in TC 24 Months post-transplant (n=19,27) | Change in LDL 24 Months post-transplant (n=16,27) | Change in HDL 24 Months post-transplant (n=19,27) | Change in Tg 24 Months post-transplant (n=19,27) | Change in TC 36 Months post-transplant (n=15,21) | Change in LDL 36 Months post-transplant (n=13,21) | Change in HDL 36 Months post-transplant (n=15,21) | Change in Tg 36 Months post-transplant (n=15,21) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.510 | 0.277 | 0.059 | 0.617 | 0.361 | 0.222 | 0.106 | 0.359 | 0.240 | 0.067 | 0.067 | 0.547 | 0.386 | 0.027 | 0.098 | 0.527 |
,Tacrolimus With Mycophenolate/Prednisone | -0.164 | -0.056 | -0.053 | -0.117 | 0.082 | 0.138 | -0.030 | -0.057 | -0.008 | 0.054 | -0.019 | -0.088 | -0.323 | -0.257 | 0.111 | -0.385 |
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Change From Pre-conversion Baseline in Fibrinogen at Months 12, 24 and 36 Post-transplant
Fibrinogen is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | gram per liter (g/L) (Mean) |
---|
| Change at 12 Months post-transplant (n=22,31) | Change at 24 Months post-transplant (n=19,29) | Change at 36 Months post-transplant (n=14,20) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.62 | 0.45 | 0.40 |
,Tacrolimus With Mycophenolate/Prednisone | 0.20 | 0.33 | 0.21 |
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Change From Pre-conversion Baseline in Glucose at Months 12, 24 and 36 Post-transplant
Fasting plasma glucose. Change = value at month x post-transplant - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | mmol/L (Mean) |
---|
| Change at 12 Months post-transplant (n=23,33) | Change at 24 Months post-transplant (n=19,31) | Change at 36 Months post-transplant (n=14,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.606 | 0.832 | 0.686 |
,Tacrolimus With Mycophenolate/Prednisone | -0.083 | -0.076 | -0.214 |
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Change From Pre-conversion Baseline in Glycosylated Hemoglobin(HbA1C) at Months 12, 24, and 36 Post-transplant
HbA1C, change = value at month x post-transplant - pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | percentage of glucose (Mean) |
---|
| Change at 12 Months post-transplant (n=23,26) | Change at 24 Months post-transplant (n=17,25) | Change at 36 Months post-transplant (n=14,16) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.008 | 0.009 | 0.012 |
,Tacrolimus With Mycophenolate/Prednisone | 0.001 | 0.006 | -0.001 |
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Change From Pre-conversion Baseline in High Sensitivity C-Reactive Protein (hsCRP) at Months 12, 24 and 36 Post-transplant.
hsCRP is a biomarker of cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | mg/L (Mean) |
---|
| Change at 12 Months post-transplant (n=23,33) | Change at 24 Months post-transplant (n=18,31) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 1.611 | -0.563 | 0.336 |
,Tacrolimus With Mycophenolate/Prednisone | 1.379 | 1.723 | 3.164 |
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Change From Pre-conversion Baseline in Homocysteine at Months 12, 24 and 36 Post-transplant
Homocysteine is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | micromole/liter (µmol/L) (Mean) |
---|
| Change at 12 Months post-transplant (n=23,33) | Change at 24 Months post-transplant (n=19,31) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 1.278 | 1.316 | 1.087 |
,Tacrolimus With Mycophenolate/Prednisone | 0.530 | 2.158 | 2.459 |
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Change From Pre-conversion Baseline in Insulin at Months 12, 24, and 36 Post-transplant
Fasting insulin. Change = value at month x post-transplant - pre-conversion baseline. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | picomole/liter (pmol/L) (Mean) |
---|
| Change at 12 Months post-transplant (n=16,23) | Change at 24 Months post-transplant (n=13,23) | Change at 36 Months post-transplant (n=10,14) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | -4.549 | -6.120 | -22.759 |
,Tacrolimus With Mycophenolate/Prednisone | 17.953 | 32.180 | -2.731 |
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Change From Pre-conversion Baseline in Interleukin-6 (IL-6) at Months 12, 24 and 36 Post-transplant
IL-6 is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | pg/mL (Mean) |
---|
| Change at 12 Months post-transplant (n=23,32) | Change at 24 Months post-transplant (n=18,29) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | -0.239 | 0.128 | -0.800 |
,Tacrolimus With Mycophenolate/Prednisone | 0.463 | 0.783 | 0.164 |
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Change From Pre-conversion Baseline in Lipoprotein(a) at Months 12, 24 and 36 Post-transplant
Lipoprotein(a) is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | milligram per deciliter (mg/dL) (Mean) |
---|
| Change at 12 Months post-transplant (n=23,30) | Change at 24 Months post-transplant (n=18,26) | Change at 36 Months post-transplant (n=13,17) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 13.496 | 12.517 | 11.885 |
,Tacrolimus With Mycophenolate/Prednisone | -11.680 | 6.073 | 6.547 |
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Change From Pre-conversion Baseline in Tumor Necrosis Factor Alpha (TNF-alpha) at Months 12, 24 and 36 Post-transplant
TNF-alpha is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | pg/mL (Mean) |
---|
| Change at 12 Months post-transplant (n=23,32) | Change at 24 Months post-transplant (n=18,30) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 0.991 | -0.367 | -0.441 |
,Tacrolimus With Mycophenolate/Prednisone | 0.000 | 1.894 | 0.000 |
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Change From Pre-conversion Baseline in Uric Acid at Months 12, 24 and 36 Post-transplant
Uric Acid is a biomarker for cardiovascular disease and atherosclerosis risk. A higher level indicates a greater risk. Change = month x post-transplant values - pre-conversion baseline values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | µmol/L (Mean) |
---|
| Change at 12 Months post-transplant (n=23,33) | Change at 24 Months post-transplant (n=19,31) | Change at 36 Months post-transplant (n=15,22) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | -51.53 | -29.02 | -19.03 |
,Tacrolimus With Mycophenolate/Prednisone | 11.10 | 3.89 | -0.58 |
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Change From Pre-conversion Baseline in Vitamin B12 at Months 12, 24 and 36 Post-transplant
Vitamin B12 is a biomarker for cardiovascular disease and atherosclerosis risk. A lower level indicates a greater risk. Change = month x post-transplant values - pre-conversion values. (NCT00311311)
Timeframe: Pre-conversion baseline, 12, 24 and 36 months post-transplant
Intervention | pmol/L (Mean) |
---|
| Change at 12 Months post-transplant (n=21,33) | Change at 24 Months post-transplant (n=19,29) | Change at 36 Months post-transplant (n=14,21) |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | -10.21 | -36.23 | -40.00 |
,Tacrolimus With Mycophenolate/Prednisone | -11.38 | -27.46 | -46.39 |
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Number of Participants Who Used Anti-hypertensive Medications
"Participants who reported yes for taking anti-hypertensive medications as concomitant medication." (NCT00311311)
Timeframe: From consent to conversion, from conversion to Month 12, from Months 12 to 24, and from Months 24 to 36 post-transplant
Intervention | participants (Number) |
---|
| From consent to conversion | From conversion to Month 12 | From Months 12 to Month 24 | From Months 24 to Month 36 |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 19 | 21 | 21 | 18 |
,Tacrolimus With Mycophenolate/Prednisone | 30 | 30 | 30 | 28 |
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Number of Participants Who Used Lipid Lowering Therapies
"Participants who reported yes for taking lipid lowering therapies as concomitant medication." (NCT00311311)
Timeframe: From consent to conversion, from conversion to Month 12, from Months 12 to 24, and from Months 24 to 36 post-transplant
Intervention | participants (Number) |
---|
| From consent to conversion | From conversion to Month 12 | From Months 12 to Month 24 | From Months 24 to Month 36 |
---|
Tacrolimus Then Sirolimus With Mycophenolate/Prednisone | 13 | 22 | 23 | 18 |
,Tacrolimus With Mycophenolate/Prednisone | 24 | 29 | 28 | 27 |
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GFR Measured by Iothalamate Clearance at Month 6
GFR measured using the iothalamate clearance method and determined by a central laboratory. (NCT00617604)
Timeframe: Month 6
Intervention | mL/minute (Mean) |
---|
Placebo | 59.6029 |
Alefacept | 55.1613 |
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Graft Survival
"Graft survival was defined as any participant who was known to have a functioning graft (i.e., not graft loss) at 6 months. Graft loss is defined as re-transplantation, nephrectomy, death or as dialysis ongoing at end of study or at discontinuation of the participant unless superseded by follow-up information.~The Kaplan-Meier estimate of graft survival within the first 6 months following transplantation is reported. Participants lost to follow-up were censored at the time of last assessment." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 90.6 |
Alefacept | 95.2 |
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Patient Survival
Patient survival is any participant known to be alive at Month 6. The Kaplan-Meier estimate of patient survival within the first 6 months following transplantation is reported. Participants lost to follow-up were censored at the time of last assessment. (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 97.1 |
Alefacept | 99.0 |
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Percentage of Participants With Acute Rejection Diagnosed by Signs and Symptoms at Month 6
Acute rejection diagnosed by signs and symptoms, including biopsy-confirmed or suspected (not confirmed by biopsy - i.e. no biopsy was performed or biopsy did not confirm an acute T-cell mediated rejection). The Kaplan-Meier estimate of acute rejection diagnosed by signs and symptoms within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit. (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 27.4 |
Alefacept | 22.3 |
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Percentage of Participants With Anti-Lymphocyte Antibody Therapy for Treatment of Rejection at Month 6
The Kaplan-Meier estimate of anti-lymphocyte antibody therapy for acute rejection (clinically-treated or biopsy-confirmed) within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 4.7 |
Alefacept | 6.7 |
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Percentage of Participants With Biopsy Confirmed Acute Mixed T-Cell Mediated and Antibody-Mediated Rejection at Month 6
"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification. A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute mixed T-cell mediated and antibody-mediated rejections within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 0.0 |
Alefacept | 1.0 |
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Percentage of Participants With Biopsy Confirmed Acute Rejection (T-Cell Mediated or Antibody Mediated) at Month 6
"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification. A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed acute T-cell mediated or antibody-mediated rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 9.3 |
Alefacept | 12.4 |
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Percentage of Participants With Biopsy Confirmed Antibody-Mediated Acute Rejection at Month 6
"Biopsies were graded by the clinical site pathologist.according to the Banff 97/05 updated histological classification:~Acute antibody-mediated rejection - documented anti-donor antibody ('suspicious for' if antibody not demonstrated):~Grade I: acute tubular necrosis-like - complement split product positive (C4d+), minimal inflammation;~Grade II: capillary-margination and/or thromboses, C4d+~Grade III: arterial - v3, C4d+.~A biopsy confirmed acute rejection was an event of suspected acute rejection confirmed by a graft biopsy result of Banff grade ≥ 1.~The Kaplan-Meier estimate of biopsy-confirmed antibody-mediated acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 2.9 |
Alefacept | 3.8 |
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Percentage of Participants With Clinically Treated Acute Rejection at Month 6
Patients who received immunosuppressive medications for the treatment of suspected or biopsy-confirmed acute rejections were considered to have a clinically-treated acute rejection. The Kaplan-Meier estimate of clinically treated acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 24.8 |
Alefacept | 15.4 |
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Percentage of Participants With Delayed Graft Function
Delayed graft function was defined as the requirement for dialysis within the first week post-transplant. (NCT00617604)
Timeframe: 1 week
Intervention | percentage of participants (Number) |
---|
Placebo | 12.1 |
Alefacept | 7.6 |
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Percentage of Participants With Efficacy Failure at Month 6
"Efficacy failure is defined as death, graft loss, biopsy-confirmed acute T-cell mediated rejection assessed by local reading or lost to follow-up.~The Kaplan-Meier estimate of efficacy failure within the first 6 months following transplantation is reported." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 15.0 |
Alefacept | 21.0 |
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Percentage of Participants With Steroid-resistant Acute Rejection at Month 6
"A steroid-resistant acute rejection is defined as a rejection episode which did not resolve following treatment with corticosteroids. In the case that a rejection episode was not treated with corticosteroids first but only with antibodies, it was included in this category.~The Kaplan-Meier estimate of steroid-resistant acute rejection within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow up visit." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 7.6 |
Alefacept | 5.7 |
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Percentage of Participants With Treatment Failure at Month 6
Treatment failure is defined as efficacy failure (death, graft loss, biopsy-confirmed acute T-cell mediated rejection assessed by local reading, lost to follow-up) or early discontinuation of alefacept/placebo at any time (during the 12-week administration period) for any reason. The Kaplan-Meier estimate of treatment failure within the first 6 months following transplantation is reported. Participants lost to follow-up or with missing outcomes were censored at their last follow-up visit. (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
Placebo | 20.6 |
Alefacept | 25.7 |
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Change From Month 1 in Creatinine Clearance
The creatinine clearance was calculated according to the Cockcroft-Gault formula. (NCT00617604)
Timeframe: Month 1, 3, and 6
Intervention | mL/minute (Mean) |
---|
| Change From Month 1 to Month 3 (n=81, 79) | Change From Month 1 to Month 6 (n=77, 73) |
---|
Alefacept | -0.5849 | 3.0227 |
,Placebo | 3.0336 | 4.5388 |
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Change From Month 1 in Glomerular Filtration Rate (GFR)
The GFR was calculated using the Modification of Diet in Renal Disease (MDRD) formula. (NCT00617604)
Timeframe: Month 1, 3, and 6
Intervention | mL/min/1.73 m² (Mean) |
---|
| Change From Month 1 to Month 3 (n=93, 87) | Change From Month 1 to Month 6 (n=83, 78) |
---|
Alefacept | 0.2889 | 2.5444 |
,Placebo | 3.1548 | 2.4275 |
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Change From Month 1 in Serum Creatinine
(NCT00617604)
Timeframe: Month 1, 3, and 6
Intervention | µmol/L (Mean) |
---|
| Change From Month 1 to Month 3 (n=94, 88) | Change From Month 1 to Month 6 (n=86, 81) |
---|
Alefacept | -5.0830 | -11.7212 |
,Placebo | -5.0125 | -6.1777 |
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Maximum Histological Grade of All Biopsies After Local Review
"The grade of acute rejection was classified according to Banff 97/05 updated version. If a patient had more than 1 rejection episode, the episode with the most severe grade was used.~Acute T-cell mediated rejection:~Grade IA: significant interstitial infiltration (>25% parenchyma affected) and foci of moderate tubulitis;~Grade IB: significant interstitial infiltration (>25% parenchyma affected) and foci of severe tubulitis;~Grade IIA: mild to moderate intimal arteritis;~Grade IIB: severe intimal arteritis comprising >25% of the luminal area;~Grade III: transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocyte inflammation.~Acute antibody-mediated rejection:~Grade I: acute tubular necrosis-like - complement split product positive (C4d+), minimal inflammation;~Grade II: capillary-margination and/or thromboses, C4d+~Grade III: arterial - v3, C4d+." (NCT00617604)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
| T-Cell Mediated Rejection - No Event | T-Cell Mediated Rejection - Grade IA | T-Cell Mediated Rejection - Grade IB | T-Cell Mediated Rejection - Grade IIA | T-Cell Mediated Rejection - Grade IIB | T-Cell Mediated Rejection - Grade III | Antibody Mediated Rejection - No Event | Antibody Mediated Rejection - Grade I | Antibody Mediated Rejection - Grade II | Antibody Mediated Rejection - Grade III |
---|
Alefacept | 89.5 | 1.9 | 1.0 | 4.8 | 2.9 | 0.0 | 96.2 | 1.9 | 1.9 | 0.0 |
,Placebo | 93.5 | 2.8 | 0.0 | 1.9 | 1.9 | 0.0 | 97.2 | 2.8 | 0.0 | 0.0 |
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Number of Participants With Adverse Events
"Causally related was defined as adverse events (AEs) assessed by the Investigator as possibly or probably related to study drug or records where the relationship was missing.~A serious adverse event (SAE) was any untoward medical occurrence that, at any dose:~Resulted in death.~Was life-threatening.~Resulted in persistent or significant disability/incapacity.~Resulted in congenital anomaly or birth defect.~Required patient hospitalization or led to prolongation of hospitalization~Was considered a medically important event.~All rejections and any BK virus, Epstein Barr virus and/or cytomegalovirus infection had to be reported as an SAE" (NCT00617604)
Timeframe: 6 Months
Intervention | participants (Number) |
---|
| Any adverse event | AE causally related to alefacept/placebo | AE causally related to MMF | AE causally related to tacrolimus | AE causally related to steroids | Serious adverse events | SAE causally related to alefacept/placebo | SAE causally related to MMF | SAE causally related to tacrolimus | SAE causally related to steroids | AE leading to discontinuation of alefacept/placebo | AE leading to discontinuation of MMF | AE leading to discontinuation of tacrolimus | AE leading to discontinuation of steroids | Deaths |
---|
Alefacept | 101 | 41 | 56 | 49 | 46 | 57 | 16 | 21 | 20 | 15 | 10 | 6 | 3 | 2 | 1 |
,Placebo | 102 | 36 | 56 | 49 | 46 | 62 | 19 | 19 | 14 | 11 | 7 | 8 | 8 | 1 | 3 |
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Mycophenolic Acid (MPA) Maintenance Treatment
The primary assessment was based on the percentage of patients who were maintained at week 13 on a dose at least one dose equivalent greater than at baseline (visit 2/week 1). A dose equivalent was defined as EC-MPS 180 mg/day or MMF 250 mg/day. (NCT00239005)
Timeframe: at week 13 (last visit)
Intervention | Percentage of Patients (Number) |
---|
Enteric-Coated Mycophenolate Sodium (EC-MPS) | 47.06 |
Mycophenolate Mofetil (MMF) | 16.39 |
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Changes in Gastrointestinal (GI) Symptoms as Measured by the Gastrointestinal Symptom Rating Scale (GSRS).
The GSRS is a 15-item instrument designed to assess the impact of upper and lower GI symptoms. There are five subscales: reflux, diarrhea, constipation, abdominal pain, and indigestion-each of which produces a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). A higher score represents greater impairment of quality of life due to GI symptoms (range from 1 to 7). (NCT00239005)
Timeframe: At week 3 and week 13 (last visit)
Intervention | Units on a scale (Least Squares Mean) |
---|
| Week 3: change in GSRS Total Score (N= 61, 59) | Week 13: change in GSRS Total Score (N= 60, 56) |
---|
Enteric-Coated Mycophenolate Sodium (EC-MPS) | -0.63 | -0.44 |
,Mycophenolate Mofetil (MMF) | -0.32 | -0.25 |
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Changes in Gastrointestinal Symptoms as Measured by the Gastrointestinal Quality of Life Index (GIQLI).
Health-related quality of life (HRQoL)was assessed by the Gastrointestinal Quality of Life Index (GIQLI). The GIQLI is a 36-item questionnaire to assess the impact of GI disease on daily life. The GIQLI also has five different subscales (GI symptoms, emotional status, physical and social functions, and stress of medical treatment) producing a total score of the 36 items. Lower scores represent more dysfunction. A higher score represents a better quality of life (range from 0 to 144). (NCT00239005)
Timeframe: At week 3 and week 13 (last visit)
Intervention | Units on a scale (Least Squares Mean) |
---|
| Week 3: change in GIQLI Total Score (N= 61, 58) | Week 13: change in GIQLI Total Score (N= 60, 56) |
---|
Enteric-Coated Mycophenolate Sodium (EC-MPS) | 11.65 | 4.84 |
,Mycophenolate Mofetil (MMF) | 6.08 | 1.77 |
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Freedom From Acute Rejection or HCV Recurrence or Treatment Failure
"Freedom from acute rejection (Banff>grade 2 with RAI score>4) or freedom from HCV recurrence (Batts/Ludwig>Stage 2, or >Grade 3) that requires HCV antiviral therapy or treatment failure (patient death, graft loss, premature withdrawal from study regimen or treatment with more than 1 dose of corticosteroids for presumptive rejection without a biopsy to confirm the rejection; reported values represent the Number of participants with Freedom From Acute Rejection or HCV Recurrence or Treatment Failure" (NCT00163657)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Treatment Arm 1 | 69 |
Treatment Arm 2 | 70 |
Treatment Arm 3 | 133 |
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Freedom From HCV Recurrence Within First Year That Requires HCV Antiviral Therapy and Freedom From Treatment Failure
Participants would have their blood drawn and tested for the HCV virus to determine if they had recurrence (NCT00163657)
Timeframe: 12 month post transplant
Intervention | participants (Number) |
---|
Treatment Arm 1 | 39 |
Treatment Arm 2 | 39 |
Treatment Arm 3 | 72 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 0 |
Cyclophosphamide/Fludarabine/TBI/ATG | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 3 |
Cyclophosphamide/Fludarabine/TBI/ATG | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 3 |
Cyclophosphamide/Fludarabine/TBI/ATG | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 3 |
Cyclophosphamide/Fludarabine/TBI/ATG | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 3 |
Cyclophosphamide/Fludarabine/TBI/ATG | 6 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 2 |
Cyclophosphamide/Fludarabine/TBI/ATG | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 3 |
Cyclophosphamide/Fludarabine/TBI/ATG | 7 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 2 |
Cyclophosphamide/Fludarabine/TBI/ATG | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 2 |
Cyclophosphamide/Fludarabine/TBI/ATG | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 1 |
Cyclophosphamide/Fludarabine/TBI/ATG | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
Cyclophosphamide/Fludarabine/TBI | 1 |
Cyclophosphamide/Fludarabine/TBI/ATG | 5 |
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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
Intervention | progression free survival probability (Number) |
---|
Cyclophosphamide/Fludarabine/TBI | 0.25 |
Cyclophosphamide/Fludarabine/TBI/ATG | 0.38 |
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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
Intervention | progression free survival probability (Number) |
---|
Cyclophosphamide/Fludarabine/TBI | 0.25 |
Cyclophosphamide/Fludarabine/TBI/ATG | 0.25 |
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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
Intervention | survival probability (Number) |
---|
Cyclophosphamide/Fludarabine/TBI | 0.25 |
Cyclophosphamide/Fludarabine/TBI/ATG | 0.50 |
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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
Intervention | survival probability (Number) |
---|
Cyclophosphamide/Fludarabine/TBI | 0.25 |
Cyclophosphamide/Fludarabine/TBI/ATG | 0.38 |
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Chimerism
Count of participants who experienced dominance of one cord blood unit (defined by >or= 95% contribution of one cord blood unit to BM and all PB fractions -- CD3+, CD33+, CD56+, and CD19+) at 7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant. (NCT00719849)
Timeframe: 7 days, 14 days, 21 days, 28 days, 56 days, and 80 days, 6 months, 1 and 2 years post transplant
Intervention | Participants (Count of Participants) |
---|
| Day 7 | Day 14 | Day 21 | Day 28 | Day 56 | Day 80 | 6 months | 1 year | 2 years |
---|
Cyclophosphamide/Fludarabine/TBI | 0 | 0 | 1 | 1 | 1 | 1 | 2 | 2 | 2 |
,Cyclophosphamide/Fludarabine/TBI/ATG | 0 | 0 | 1 | 5 | 5 | 6 | 7 | 7 | 7 |
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The Proportion of Participants With Graft Loss or Death Within 12 Months Post Kidney Transplantation
Graft loss is defined as the need for dialysis for more than 30 days duration, allograft nephrectomy, or the decision to withdraw immunosuppression due to graft failure. (NCT00240994)
Timeframe: Up to one year post kidney transplantation procedure
Intervention | Proportion of participants (Number) |
---|
Alemtuzumab (Campath) | 0.057 |
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Duration of Exposure to Study Medication
The duration of exposure was calculated as the date of the last Mycophenolate sodium dose minus the date of the last Mycophenolate sodium dose +1. (NCT00423098)
Timeframe: 24 weeks
Intervention | days (Mean) |
---|
Standard Dose | 164.5 |
Low Dose | 157.7 |
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Change From Baseline in Overall Disease Activity With British Isles Lupus Assessment Group (BILAG)
BILAG (British Isles Lupus Assessment Group) index divides lupus activity into 8 organs/systems and was based on the principle of the physician's intention to treat, assessing activity in the previous one month. Each organ or system was given a score of A to E, where A = disease that is sufficiently active to require disease modifying treatment; a B = problems requiring symptomatic treatment; C = stable mild disease; D = previously affected but currently inactive system; and E = the system or organ has never been involved. [A=9, B=3, C=1, D/E=0 the score range for each patient will be 0-72]. (NCT00423098)
Timeframe: From Baseline to week 4, week 12 and week 24
Intervention | Units on a scale (Mean) |
---|
| Change from baseline Week 4: (N= 40, 37) | Change from baseline Week 12: (N= 41, 35) | Change from baseline Week 24: (N= 40, 34) |
---|
Low Dose | -5.5 | -8.7 | -9.4 |
,Standard Dose | -4.8 | -8.6 | -8.6 |
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Change From Baseline in Overall Disease Activity With Systematic Lupus Erythematosus Disease Activity Index (SLEDAI)
SLEDAI stands for Systemic Lupus Erythematosus Disease Activity Index and was a well established global score index based on assessment of 24 items measuring a disease activity in the 10-day period prior to the assessment. SLEDAI item weights range from 1 for fever to 8 for seizures. A maximum theoretical score is 105. Total score range from 1 to 105. A flare has been defined as a SLEDAI score increase of 3 or more to a level of 8 or higher. During flares SLEDAI scores of 25 to 30 are common. (NCT00423098)
Timeframe: From Baseline to week 4, week 12 and week 24
Intervention | Units on a scale (Mean) |
---|
| Change from Baseline to Week 4: (N= 39, 37) | Change from Baseline to Week 12: (N= 41, 35) | Change from Baseline to Week 24: (N= 39, 34) |
---|
Low Dose | -7.7 | -10.3 | -9.8 |
,Standard Dose | -7.4 | -9.7 | -10.3 |
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Cumulative Dose of Prednisone Equivalent Corticosteroids (CS)
Corticosteroid use was measured as cumulative dose until 12 and 24 weeks of treatment as well as daily doses at baseline, 12 and 24 weeks. (NCT00423098)
Timeframe: 12 Weeks and 24 Weeks
Intervention | mg/kg (Mean) |
---|
| Week 12 | Week 24 |
---|
Low Dose | 68.2 | 73.0 |
,Standard Dose | 106.1 | 114.2 |
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Number of Patients With Adverse Events and Infections
Safety assessments included collecting all adverse events (AEs), serious adverse events (SAEs), with their severity and relationship to study drug. According to FDA 21CFR 314.80, a serious adverse event (SAE) is described as any adverse event that leads to death, is life threatening ( NIH criteria Grade 4), causes or prolongs hospitalization, results in a congenital anomaly, or any other important medical event not described above. (NCT00423098)
Timeframe: 24 weeks
Intervention | participants (Number) |
---|
| At least one adverse event | Any severe adverse event | Any drug related adverse event | Any serious adverse event | Any infection | Any severe infection | Any drug related infection | Any serious infection |
---|
Low Dose | 30 | 3 | 16 | 4 | 17 | 1 | 6 | 1 |
,Standard Dose | 35 | 7 | 18 | 8 | 25 | 3 | 10 | 4 |
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Number of Patients With Complete Remission
Complete remission was defined as urine protein/urine creatinine ratio < 0.5 gram urine protein per gram urine creatinine, urine sediment normalized (no cellular casts, < 5 red cells per high power field), and serum creatinine within 10% of normal range according to local lab. (NCT00423098)
Timeframe: 24 Weeks
Intervention | Participants (Number) |
---|
| Yes | No |
---|
Low Dose | 8 | 31 |
,Standard Dose | 8 | 34 |
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Number of Patients With Complete Remission
Complete remission was defined as urine protein/urine creatinine ratio < 0.5 gram urine protein per gram urine creatinine, urine sediment normalized (no cellular casts, < 5 red cells per high power field), and serum creatinine within 10% of normal value. (NCT00423098)
Timeframe: 12 Weeks
Intervention | participants (Number) |
---|
| Yes | No |
---|
Low Dose | 5 | 34 |
,Standard Dose | 9 | 33 |
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Number of Patients With Moderate to Severe Flares
A moderate to severe flare was defined as the occurrence of increased lupus activity after partial or complete remission, based on the presence of 1 BILAG A score or >=3 BILAG B scores. British Isles Lupus Assessment Group (BILAG) index divides lupus activity in 8 organs/systems which are each given a score of A to E. A=disease sufficiently active to need disease modifying treatment; B=problems requiring symptomatic treatment; C=mild stable disease; D=previously affected but currently inactive system; E=the system or organ has never been involved. BILAG score: A=9, B=3, C=1, D/E=0; range(0-72) (NCT00423098)
Timeframe: 12 and 24 weeks
Intervention | participants (Number) |
---|
| At week 12 - Yes | At week 12 - No | At week 24 - Yes | At week 24 - No |
---|
Low Dose | 0 | 39 | 0 | 39 |
,Standard Dose | 0 | 42 | 1 | 41 |
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Number of Patients With Partial Remission
Partial remission was defined as urine protein/creatinine ratio reduced by at least 50% from baseline and stable serum creatinine within 10% of baseline value) or improved. (NCT00423098)
Timeframe: Baseline to 12 and 24 weeks
Intervention | Participants (Number) |
---|
| At 12 weeks - Yes | At 12 weeks - No | At 24 weeks - Yes | At 24 weeks - No |
---|
Low Dose | 11 | 28 | 14 | 25 |
,Standard Dose | 16 | 26 | 20 | 22 |
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Number of Patients With Treatment Failure
Treatment failure was defined as no therapeutic response (without complete or partial remission) or premature discontinuation during the first 24 weeks from study medication or the study for any reason except complete or partial remission. (NCT00423098)
Timeframe: 12 Weeks and 24 Weeks
Intervention | participants (Number) |
---|
| At 12 weeks - Yes | At 12 weeks - No | At 24 weeks - Yes | At 24 weeks - No |
---|
Low Dose | 25 | 14 | 22 | 17 |
,Standard Dose | 23 | 19 | 21 | 21 |
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Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (12 Months Analysis)
The glomerular filtration rate (GFR) was calculated by the Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^ 2 + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. (NCT00371826)
Timeframe: At Month 12
Intervention | mL/min per 1.73 m^2 (Mean) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 65.2 |
CNI+MPA+ Steroid | 69.3 |
Steroid Withdrawal | 66.9 |
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Mean Serum Creatinine (12 Months Analysis)
(NCT00371826)
Timeframe: At Month 12
Intervention | umol/L (Mean) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 118.9 |
CNI+MPA+ Steroid | 161.0 |
Steroid Withdrawal | 148.6 |
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Mean Urine Albumin/Creatinine Ratio (ACR) as Measurement of Proteinuria (12 Months Analysis)
Proteinuria is measured by spot morning urine Albumin/Creatinine Ratio [ACR]. When the ACR is more than or equal to 30 mg/mmol then it is known as proteinuria. (NCT00371826)
Timeframe: At Month 12
Intervention | mg/mg (Mean) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 0.2 |
CNI+MPA+ Steroid | 0.1 |
Steroid Withdrawal | 0.1 |
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Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (12 Months Analysis)
This analysis is reporting number of participants hospitalized for reasons (such as acute rejection, infection, gastrointestinal (GI) events, cardiovascular event, metabolic disorder and Other) other than primary transplantation. (NCT00371826)
Timeframe: Month 12
Intervention | Participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 34 |
CNI+MPA+ Steroid | 31 |
Steroid Withdrawal | 13 |
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Number of Participants Hospitalized for Reasons Other Than Primary Transplantation (36 Months Analysis)
This analysis is reporting number of participants hospitalized for reasons (such as acute rejection, infection, gastrointestinal (GI) events, cardiovascular event, metabolic disorder and Other) other than primary transplantation. (NCT00371826)
Timeframe: Month 36
Intervention | Participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 16 |
CNI+MPA+ Steroid | 30 |
Steroid Withdrawal | 1 |
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Number of Participants With Antibody-mediated Rejection Per Treatment Group (12 Months Analysis)
(NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 5 |
CNI+MPA+ Steroid | 2 |
Steroid Withdrawal | 2 |
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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (12 Months Analysis)
A biopsy-proven acute rejection is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification. (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 15 |
CNI+MPA+ Steroid | 6 |
Steroid Withdrawal | 5 |
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Number of Participants With Composite Endpoint of Treatment Failure (12 Months Analysis)
"Composite endpoint of treatment failure includes biopsy-proven acute rejection (BPAR), graft loss, death and loss-to-follow-up. A BPAR is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification.~The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss." (NCT00371826)
Timeframe: Month 12
Intervention | Participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 16 |
CNI+MPA+ Steroid | 8 |
Steroid Withdrawal | 11 |
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Number of Participants With Erythropoietin Usage (12 Months Analysis)
(NCT00371826)
Timeframe: Month 12
Intervention | participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 20 |
CNI+MPA+ Steroid | 10 |
Steroid Withdrawal | 8 |
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Number of Participants With Erythropoietin Usage (36 Months Analysis)
(NCT00371826)
Timeframe: Month 36
Intervention | participants (Number) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 5 |
CNI+MPA+ Steroid | 7 |
Steroid Withdrawal | 2 |
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Calculated Glomerular Filtration Rate (cGFR) After Kidney Transplant to Evaluate Kidney Function (36 Months Analysis)
The glomerular filtration rate (GFR) was calculated by the Nankivell formula: GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)^ 2 + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. (NCT00371826)
Timeframe: At Month 24 and 36
Intervention | mL/min per 1.73 m^2 (Mean) |
---|
| Month 24 (n= 23, 36, 4) | Month 36 (n= 19, 35, 3) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 69.5 | 71.6 |
,CNI+MPA+ Steroid | 71.8 | 69.1 |
,Steroid Withdrawal | 67.0 | 61.0 |
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Change in Bone Mineral Density Between Week 2 and Month 24 (36 Months Analysis)
Measurements of bone mineral density (BMD) by Dual Energy X-ray Absorptiometry (DEXA) were done at Week 2 and Month 24. Change in BMD between week 2 and Month 24 were done for neck of femur and lumbar spine. (NCT00371826)
Timeframe: Week 2, Month 24
Intervention | g/cm^2 (Mean) |
---|
| Neck of Femur (Month 24- Week 2) | Lumbar Spine (Month 24 - Week 2) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 0.2 | -0.0 |
,CNI+MPA+ Steroid | -0.1 | -0.1 |
,Steroid Withdrawal | -0.1 | -0.0 |
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Mean Serum Creatinine (36 Months Analysis)
(NCT00371826)
Timeframe: At Month 12, 18, 24 and 36
Intervention | umol/L (Mean) |
---|
| At Month 12 (n= 23, 39, 4) | At Month 18 (n= 22, 36, 4) | At Month 24 (n = 23, 36, 4) | At Month 36 (n= 19, 35, 3) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 112.7 | 113.6 | 119.9 | 119.4 |
,CNI+MPA+ Steroid | 123.0 | 121.4 | 123.7 | 131.8 |
,Steroid Withdrawal | 146.8 | 146.3 | 146.5 | 176.0 |
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Mean Urine Albumin/Creatinine Ratio [ACR] as Measurement of Proteinuria (36 Months Analysis)
Proteinuria is measured by spot morning urine Albumin/Creatinine Ratio [ACR]. When the ACR is more than or equal to 30 mg/mmol then it is known as proteinuria. (NCT00371826)
Timeframe: At Month 12, 18, 24 and 36
Intervention | mg/mmol (Mean) |
---|
| At 12 Month (n = 18, 32, 2) | At 18 Month (n= 17, 31, 2) | At 24 Month ( n= 16, 29, 1) | At 36 Month ( n= 15, 25, 0) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 23.5 | 28.2 | 30.0 | 30.6 |
,CNI+MPA+ Steroid | 8.1 | 8.1 | 6.6 | 23.4 |
,Steroid Withdrawal | 4.8 | 5.3 | 7.0 | NA |
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Number of Participants With Antibody-mediated Rejection Per Treatment Group (36 Months Analysis)
(NCT00371826)
Timeframe: At Month 12, 24 and 36
Intervention | Participants (Number) |
---|
| At Month 12 | At Month 24 | At Month 36 |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 1 | 1 | 1 |
,CNI+MPA+ Steroid | 0 | 0 | 2 |
,Steroid Withdrawal | 0 | 0 | 0 |
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Number of Participants With Any Wound Problems (36 Months Analysis)
Patients with any wound healing problem such as infection related to kidney surgery, dehiscence, lymphocele, hernia, seroma, hematoma, ureteral anastomotic complication and other were reported in this analysis. (NCT00371826)
Timeframe: At Month 12, 24 and 36
Intervention | Participants (Number) |
---|
| At Month 12 | At Month 24 | At Month 36 |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 6 | 9 | 10 |
,CNI+MPA+ Steroid | 11 | 13 | 13 |
,Steroid Withdrawal | 2 | 2 | 2 |
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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (12 Months Analysis)
The influence of demographic characteristics and comorbidities on incidence of BPAR were analyzed in the following way: Demographic characteristics were age (<55 years, ≥55 years), Expanded criteria Donor (ECD) organ (donor age >60 years or donor non heart-beating and donor age >50), gender, living vs. deceased donor, Body Mass Index (BMI) classes (underweight <18.5, normal 18.5 - <25.0, overweight 25.0 - <30.0, obesity 30.0 and above), years on dialysis before transplantation (<1, 1-5, >5 years). Comorbidities were diabetes, hypertension, cardiovascular diseases/events, nephrosclerosis, glomerulonephritis/glomerular disease, polycystic disease, and Cytomegalovirus status. (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| Age < 55 years | Age > = 55 years | ECD Organ | NO ECD Organ | Male | Female | Living donor | Deceased donor | BMI < 18.5 | BMI 18.5 - <25 | BMI 25 - <30 | BMI >= 30 | Years on dialysis before transplantation: None | < 1 Year on dialysis before transplantation | 1 - 5 Years on dialysis before transplantation | > 5 Years on dialysis before transplantation | Diabetes mellitus: No | Diabetes mellitus: Yes | Hypertension: No | Hypertension: Yes | Cardiovascular disease : No | Cardiovascular disease: Yes | Nephrosclerosis: No | Nephrosclerosis: Yes | Glomerulonephritis/glomerular disease : No | Glomerulonephritis/glomerular disease: Yes | Cytomegalovirus : Negative | Cytomegalovirus : Positive |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 11 | 4 | 3 | 12 | 11 | 4 | 9 | 6 | 0 | 5 | 8 | 2 | 1 | 3 | 8 | 3 | 10 | 5 | 2 | 13 | 4 | 11 | 15 | 0 | 8 | 7 | 2 | 13 |
,CNI+MPA+ Steroid | 5 | 1 | 0 | 6 | 6 | 0 | 6 | 0 | 1 | 2 | 3 | 0 | 0 | 3 | 1 | 2 | 4 | 2 | 1 | 5 | 3 | 3 | 6 | 0 | 5 | 1 | 2 | 4 |
,Steroid Withdrawal | 4 | 1 | 2 | 3 | 4 | 1 | 2 | 3 | 0 | 3 | 1 | 1 | 0 | 1 | 2 | 2 | 4 | 1 | 0 | 5 | 2 | 3 | 4 | 1 | 3 | 2 | 0 | 5 |
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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Influenced by Demographic Characteristics and Morbidities (36 Months Analysis)
The influence of demographic characteristics and comorbidities on incidence of BPAR were analyzed in the following way: Demographic characteristics were age (<55 years, ≥55 years), Expanded Criteria Donor [ECD] organ (donor age >60 years or donor non heart-beating and donor age >50), gender, living vs. deceased donor, Body Mass Index (BMI) classes (underweight <18.5, normal 18.5 - <25.0, overweight 25.0 - <30.0, obesity 30.0 and above), years on dialysis before transplantation (<1, 1-5, >5 years). Comorbidities were diabetes, hypertension, cardiovascular diseases/events, nephrosclerosis, glomerulonephritis/glomerular disease, polycystic disease, and Cytomegalovirus status. (NCT00371826)
Timeframe: At Month 36
Intervention | Participants (Number) |
---|
| Age < 55 years | Age > = 55 years | ECD Organ | NO ECD Organ | Male | Female | Living donor | Deceased donor | BMI < 18.5 | BMI 18.5 - <25 | BMI 25 - <30 | BMI >= 30 | Years on dialysis before transplantation: None | < 1 Year on dialysis before transplantation | 1 - 5 Years on dialysis before transplantation | > 5 Years on dialysis before transplantation | Diabetes mellitus: No | Diabetes mellitus: Yes | Hypertension: No | Hypertension: Yes | Cardiovascular disease : No | Cardiovascular disease: Yes | Nephrosclerosis: No | Nephrosclerosis: Yes | Glomerulonephritis/glomerular disease : No | Glomerulonephritis/glomerular disease: Yes | Cytomegalovirus : Negative | Cytomegalovirus : Positive |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 6 | 0 | 0 | 6 | 4 | 2 | 5 | 1 | 1 | 1 | 4 | 0 | 0 | 3 | 2 | 1 | 3 | 3 | 1 | 5 | 3 | 3 | 6 | 0 | 4 | 2 | 1 | 5 |
,CNI+MPA+ Steroid | 7 | 1 | 0 | 8 | 7 | 1 | 6 | 2 | 0 | 3 | 4 | 1 | 1 | 4 | 2 | 1 | 6 | 2 | 1 | 7 | 5 | 3 | 7 | 1 | 7 | 1 | 4 | 4 |
,Steroid Withdrawal | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
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Number of Participants With Biopsy Proven Acute Rejection (BPAR) Per Treatment Group (36 Months Analysis)
A biopsy-proven acute rejection is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification. (NCT00371826)
Timeframe: At Month 12, 24 and 36
Intervention | Participants (Number) |
---|
| At Month 12 | At Month 24 | At Month 36 |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 4 | 5 | 6 |
,CNI+MPA+ Steroid | 4 | 5 | 8 |
,Steroid Withdrawal | 0 | 0 | 0 |
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Number of Participants With Composite Endpoint of Treatment Failure (36 Months Analysis)
"Composite endpoint of treatment failure includes biopsy-proven acute rejection (BPAR), graft loss, death and loss-to-follow-up. A BPAR is defined as a biopsy graded IA, IB, IIA, IIB, or III as per Banff 97 classification.~The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss." (NCT00371826)
Timeframe: At Month 12, 24 and 36
Intervention | Participants (Number) |
---|
| At Month 12 | At Month 24 | At Month 36 |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 4 | 6 | 7 |
,CNI+MPA+ Steroid | 4 | 6 | 10 |
,Steroid Withdrawal | 0 | 0 | 0 |
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Number of Participants With Employment Status (12 Months Analysis)
"The various employment status reported are:~Employed/self employed full time~Employed part time~Unemployed~Homemaker~Volunteer~Permanently disabled~Non-permanently disable~Retired~Other" (NCT00371826)
Timeframe: At screening (at day 0 +/- 7 days ), At Month 12
Intervention | Participants (Number) |
---|
| Screening visit: Employed/self employed full time | Month 12: Employed/self employed full time | Screening visit: Employed part time | Month 12: Employed part time | Screening visit: Unemployed | Month 12: Unemployed | Screening visit: Homemaker | Month 12 : Homemaker | Screening visit: Volunteer | Month 12: Volunteer | Screening visit: Permanently disabled | Month 12: Permanently disabled | Screening visit: Non-permanently disabled | Month 12: Non-permanently disabled | Screening visit: Retired | Month 12: Retired | Screening visit: Other | Month 12: Other |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 18 | 6 | 9 | 3 | 10 | 6 | 3 | 2 | 1 | 1 | 2 | 1 | 1 | 1 | 4 | 1 | 1 | 1 |
,CNI+MPA+ Steroid | 21 | 14 | 7 | 5 | 8 | 1 | 5 | 5 | 0 | 0 | 2 | 2 | 2 | 0 | 2 | 2 | 0 | 0 |
,Steroid Withdrawal | 9 | 2 | 4 | 0 | 10 | 6 | 5 | 2 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 1 | 0 |
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Number of Participants With Employment Status (36 Months Analysis)
"The various employment status reported are:~Employed/self employed full time~Employed part time~Unemployed~Homemaker~Volunteer~Permanently disabled~Non-permanently disable~Retired~Other" (NCT00371826)
Timeframe: At screening (at day 0 +/- 7 days ), At Month 36
Intervention | Participants (Number) |
---|
| Screening visit: Employed/self employed full time | Month 36: Employed/self employed full time | Screening visit: Employed part time | Month 36: Employed part time | Screening visit: Unemployed | Month 36: Unemployed | Screening visit: Homemaker | Month 36 : Homemaker | Screening visit: Permanently disabled | Month 36: Permanently disabled | Screening visit: Non-permanently disabled | Month 36: Non-permanently disabled | Screening visit: Retired | Month 36: Retired | Screening visit: Other | Month 36: Other |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 5 | 4 | 4 | 3 | 5 | 3 | 3 | 3 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 1 |
,CNI+MPA+ Steroid | 10 | 10 | 7 | 4 | 7 | 6 | 3 | 3 | 1 | 1 | 1 | 1 | 2 | 2 | 0 | 0 |
,Steroid Withdrawal | 1 | 1 | 0 | 0 | 1 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
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Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (12 Months Analysis)
"A per-protocol biopsy was performed at Baseline and Month 12 and read by an independent blinded pathologist in order to assess chronic allograft nephropathy. Chronic rejection is characterized by a slow progressive decline in renal function and is typically preceded by the histological picture of chronic allograft nephropathy. The presence of biopsy confirmed Grade I, II or III chronic allograft nephropathy by Banff 97 criteria was assessed on all optional biopsies obtained for clinical suspicion of chronic rejection.~Data summarized by 3 categories. Yes - Patients with histological evidence of CAN ; No - Patients with histological evidence of CAN and Not Done - Central protocol defined kidney allograft biopsies were not done." (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| YES | NO | Not Done |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 8 | 15 | 7 |
,CNI+MPA+ Steroid | 6 | 26 | 8 |
,Steroid Withdrawal | 2 | 5 | 3 |
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Number of Participants With Histological Evidence Chronic Allograft Nephropathy (CAN) (36 Months Analysis)
"Chronic rejection is characterized by a slow progressive decline in renal function and is typically preceded by the histological picture of chronic allograft nephropathy. The presence of biopsy confirmed Grade I, II or III chronic allograft nephropathy by Banff 97 criteria was assessed on all optional biopsies obtained for clinical suspicion of chronic rejection.~Data summarized by 3 categories. Yes - Patients with histological evidence of CAN ; No - Patients with histological evidence of CAN and Not Done - Central protocol defined kidney allograft biopsies were not done." (NCT00371826)
Timeframe: At Month 36
Intervention | Participants (Number) |
---|
| YES | NO | Not Done |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 2 | 8 | 8 |
,CNI+MPA+ Steroid | 1 | 16 | 14 |
,Steroid Withdrawal | 1 | 1 | 1 |
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Number of Participants With New Onset Diabetes Mellitus After Transplantation (NODAT) and Impaired Fasting Glucose (36 Months Analysis)
"The symptoms of new onset diabetes mellitus after transplantation (NODAT) and impaired fasting glucose are defined as any of the following conditions:~Patients receiving glucose lowering treatment~2 fasting plasma glucose (FPG) values >= 126 mg/dL or 2 random plasma glucose (RPG) values >= 200 mg/dL or FPG value >= 126 mg/dL and 1 RPG value >= 200 mg/dL~Diabetes reported as treatment emergent AE with end date > Day 15" (NCT00371826)
Timeframe: At Month 36
Intervention | Participants (Number) |
---|
| Glucose lowering treatment | FPG or RPG | Diabetes as treatment emergent AE | Any of the above symptoms |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 5 | 3 | 5 | 6 |
,CNI+MPA+ Steroid | 10 | 2 | 4 | 11 |
,Steroid Withdrawal | 2 | 1 | 2 | 2 |
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Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)
"Notable abnormal systolic blood pressure is defined as :~Either an increase of >=30 that results in >=180 or >200 (mm/Hg)~OR a decrease of >=30 that results in <=90 or <75 (mm/Hg)from baseline~Notable abnormal diastolic blood pressure is defined as :~Either an increase of >=20 that results in >=105 or >115 (mm/Hg)~OR a decrease of >=20 that results in <=50 or <40 (mm/Hg) from baseline" (NCT00371826)
Timeframe: Baseline, Overall post-baseline up to 12 month
Intervention | Participants (Number) |
---|
| SBP: >=180 mm/Hg or 200 mm/Hg | SBP: < = 90 mm/Hg or < 200 mm/Hg | DBP : >=105 mm/Hg or >115 mm/Hg | DBP: <=50 mm/Hg or <40 mm/Hg |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 5 | 1 | 3 | 1 |
,CNI+MPA+ Steroid | 5 | 0 | 6 | 4 |
,Steroid Withdrawal | 7 | 0 | 5 | 0 |
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Number of Participants With Notable Abnormal Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP) as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)
"Notable abnormal systolic blood pressure is defined as :~Either an increase of >=30 that results in >=180 or >200 (mm/Hg)~OR a decrease of >=30 that results in <=90 or <75 (mm/Hg) from baseline~Notable abnormal diastolic blood pressure is defined as :~Either an increase of >=20 that results in >=105 or >115 (mm/Hg)~OR a decrease of >=20 that results in <=50 or <40 (mm/Hg) from baseline" (NCT00371826)
Timeframe: Baseline, Overall post baseline up to Month 36
Intervention | Participants (Number) |
---|
| SBP: >=180 mm/Hg or 200 mm/Hg | SBP: < = 90 mm/Hg or < 200 mm/Hg | DBP : >=105 mm/Hg or >115 mm/Hg | DBP: <=50 mm/Hg or <40 mm/Hg |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 3 | 1 | 2 | 1 |
,CNI+MPA+ Steroid | 4 | 0 | 5 | 4 |
,Steroid Withdrawal | 1 | 0 | 1 | 0 |
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Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (12 Months Analysis)
"Notable abnormal total cholesterol is defined as : High: >= 9.1 mmol/L , normal range is 0.00 - 5.17 mmol/L~Notable abnormal triglycerides is defined as : High: >= 8.5 mmol/L, normal range is 0.30 - 2.00 mmol/L" (NCT00371826)
Timeframe: Overall post baseline up to month 12
Intervention | Participants (Number) |
---|
| Total Cholesterol: High (n = 48, 47,30) | Triglycerides : High (n= 48, 46, 30) |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 6 | 1 |
,CNI+MPA+ Steroid | 0 | 0 |
,Steroid Withdrawal | 3 | 0 |
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Number of Participants With Notable Abnormalities in Total Cholesterol and Triglycerides as Measurement of Effect of Treatment on Cardiovascular Health (36 Months Analysis)
"Notable abnormal total cholesterol is defined as : High: >= 9.1 mmol/L , normal range is 0.00 - 5.17 mmol/L~Notable abnormal triglycerides is defined as : High: >= 8.5 mmol/L, normal range is 0.30 - 2.00 mmol/L" (NCT00371826)
Timeframe: Overall Post Baseline up to month 36
Intervention | Participants (Number) |
---|
| Total Cholesterol: High | Triglycerides : High |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 6 | 0 |
,CNI+MPA+ Steroid | 0 | 1 |
,Steroid Withdrawal | 1 | 0 |
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Number of Participants With Post Transplant Diabetes Mellitus (PTDM) and Impaired Fasting Glucose (12 Months Analysis)
"The symptoms of post transplant diabetes mellitus (PTDM) and impaired fasting glucose are defined as any of the following conditions:~Patients receiving glucose lowering treatment~Fasting plasma glucose (FPG) >= 126 mg/dL on 2 separate occasions~Hemoglobin subtype A1c (HbA1c) > 6.5%~Diabetes reported as treatment emergent AE with end date > Day 15" (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| Glucose lowering treatment | FPG >= 126 mg/dL on 2 separate occasions | HbA1c > 6.5% | Diabetes as treatment emergent AE | Any of the above symptoms |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 10 | 6 | 12 | 8 | 19 |
,CNI+MPA+ Steroid | 9 | 0 | 3 | 2 | 10 |
,Steroid Withdrawal | 5 | 5 | 5 | 3 | 8 |
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Number of Participants With Sub Clinical Acute Rejection (12 Months Analysis)
"Based on Banff 97 criteria, sub clinical acute rejection can be:~GRADE IA - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IB - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IIA - Cases with significant interstitial infiltration and mild to moderate intimal arteritis (v1).~GRADE IIB - Cases with moderate to severe intimal arteritis comprising >25% of the luminal area (v2).~GRADE III - Cases with transmural arteritis or fibrinoid change and necrosis of medial smooth muscle cells (v3).~Borderline category is used when no intimal arteritis is present, but there are foci of mild tubulitis (1 to 4 mononuclear cells/tubular cross section)." (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| NO | Borderline | Grade IA | Grade IB | Grade IIA | Grade IIB | Grade III | Not Done |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 20 | 2 | 1 | 0 | 0 | 0 | 0 | 7 |
,CNI+MPA+ Steroid | 29 | 2 | 0 | 1 | 0 | 0 | 0 | 8 |
,Steroid Withdrawal | 5 | 2 | 0 | 0 | 0 | 0 | 0 | 3 |
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Number of Participants With Sub Clinical Acute Rejection (36 Months Analysis)
"Based on Banff 97 criteria, sub clinical acute rejection can be:~GRADE IA - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>4 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IB - Cases with significant interstitial infiltration (>25% of parenchyma affected) and foci of moderate tubulitis (>10 mononuclear cells/tubular cross section or group of 10 tubular cells).~GRADE IIA - Cases with significant interstitial infiltration and mild to moderate intimal arteritis (v1).~GRADE IIB - Cases with moderate to severe intimal arteritis comprising >25% of the luminal area (v2).~GRADE III - Cases with transmural arteritis or fibrinoid change and necrosis of medial smooth muscle cells (v3).~Borderline' category is used when no intimal arteritis is present, but there are foci of mild tubulitis (1 to 4 mononuclear cells/tubular cross section)." (NCT00371826)
Timeframe: At Month 36
Intervention | Participants (Number) |
---|
| Month 36: NO | Month 36: Borderline | Month 36: Grade IA | Month 36: Grade IB | Month 36: Grade IIA | Month 36: Grade IIB | Month 36: Grade III | Month 36: Not Done |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 9 | 1 | 0 | 0 | 0 | 0 | 0 | 8 |
,CNI+MPA+ Steroid | 15 | 1 | 1 | 0 | 0 | 0 | 0 | 14 |
,Steroid Withdrawal | 2 | 0 | 0 | 0 | 0 | 0 | 0 | 1 |
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Number of Participants With Wound Problems(12 Months Analysis)
Patients with any wound healing problem such as infection related to kidney surgery, dehiscence, lymphocele, hernia, seroma, hematoma, ureteral anastomotic complication and other were reported in this analysis. (NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| Any wound healing problem | Infection related to kidney surgery | Dehiscence | Lymphocele | Hernia | Seroma | Hematoma | Ureteral anastomotic complication | Other |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 16 | 2 | 3 | 3 | 3 | 4 | 4 | 2 | 3 |
,CNI+MPA+ Steroid | 15 | 4 | 2 | 4 | 2 | 9 | 0 | 1 | 1 |
,Steroid Withdrawal | 9 | 3 | 2 | 3 | 1 | 3 | 2 | 1 | 0 |
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Number of Patient Survival and Graft Survival (12 Months Analysis)
(NCT00371826)
Timeframe: At Month 12
Intervention | Participants (Number) |
---|
| Patient Survival | Graft Survival |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 49 | 49 |
,CNI+MPA+ Steroid | 46 | 45 |
,Steroid Withdrawal | 30 | 30 |
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Number of Patient Survival and Graft Survival (36 Months Analysis)
(NCT00371826)
Timeframe: At Month 12, 24 and 36
Intervention | Participants (Number) |
---|
| Month 12: Patient Survival | Month 12: Graft Survival | Month 24: Patient Survival | Month 24: Graft Survival | Month 36: Patient Survival | Month 36: Graft Survival |
---|
Calcineurin Inhibitor (CNI) Withdrawal | 23 | 23 | 23 | 23 | 23 | 23 |
,CNI+MPA+ Steroid | 39 | 39 | 39 | 39 | 39 | 39 |
,Steroid Withdrawal | 4 | 4 | 4 | 4 | 4 | 4 |
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Change From Baseline in Lower and Upper GI Symptom Burden Measured by GI Symptom Rating Scale Score
This is reflected by the total score. The total score incorporates lower and upper GI elements. GSRS overall score is the mean of 15 individual GI symptom scores, each rated on a 7- point scale: 1 = no discomfort, 2= minor discomfort, 3 = mild discomfort, 4 = moderate discomfort, 5 = moderately sever discomfort, 6 = severe discomfort and 7 = very severe discomfort. Change from Baseline was calculated using ANCOVA, model includes GSRS, center and treatment group. (NCT00400400)
Timeframe: Baseline, Day 30
Intervention | change in score on a scale (Mean) |
---|
Enteric-coated Mycophenolate Sodium | -0.6 |
Mycophenolate Mofetil | -0.5 |
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Change From Baseline (BL) to Day 30 in the Gastrointestinal Quality of Life Index (GIQLI) Total Score and Subscale Scores
The GIQLI is a 36-item questionnaire to assess the impact of GI disease on daily life. The GIQLI has 5 different subscales (GI symptoms, emotional status, physical and social functions, and stress of medical treatment) that are rated on a 5-point scale from 0 to 4. The individual scores are summed to produce a total score of the 36 items for a total possible score of 0 to 144. Lower scores represent greater dysfunction. (NCT00400400)
Timeframe: Baseline, Day 30
Intervention | Score on a scale (Mean) |
---|
| Overall Total Score: BL ( n= 197,197) | Overall Total Score : Day 30 (n= 195,192) | Overall Total Score : Change from BL (n=193,192) | GI Symptom: BL (n= 197, 197) | GI Symptom: Day 30 (n= 195, 192) | GI Symptom: Change from BL (n= 193, 192) | Emotional Status: BL (n= 196, 197) | Emotional Status: Day 30 (n= 195, 192) | Emotional Status: Change from BL (n= 192, 192) | Physical Function: BL (n= 196, 197) | Physical Function: Day 30 (n= 195, 192) | Physical Function: Change from BL (n= 192, 192) | Social Function: BL (n= 197, 197) | Social Function: Day 30 (n= 195, 192) | Social Function: Change from BL (n= 193, 192) | Medical treatment: BL (n= 197, 197) | Medical treatment: Day 30 (n= 194, 192) | Medical treatment: Change from BL (n= 192, 192) |
---|
Enteric-coated Mycophenolate Sodium | 94.3 | 105.0 | 10.7 | 46.7 | 52.6 | 5.9 | 11.9 | 13.2 | 1.3 | 21.7 | 23.7 | 2.1 | 10.7 | 11.9 | 1.2 | 3.3 | 3.6 | 0.2 |
,Mycophenolate Mofetil | 93.4 | 103.5 | 10.2 | 46.6 | 51.9 | 5.4 | 11.6 | 13.1 | 1.5 | 21.5 | 23.3 | 1.9 | 10.4 | 11.6 | 1.2 | 3.4 | 3.6 | 0.3 |
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Change From Baseline to Day 30 in the Severity of Gastrointestinal Symptoms Overall Total Score
The Severity Score for each GI symptom for each participant was calculated based on the physician's evaluation of current GI symptoms recorded at Baseline and Day 30. For each of the 16 individual GI symptoms the severity score ranged from 0 (absent) to 3 (severe). The Overall Total Score is the Mean of severity ratings of the 16 individual symptoms. (NCT00400400)
Timeframe: Baseline, Day 30
Intervention | Score on a scale (Mean) |
---|
| Baseline (n= 198,195) | Day 30 (n=193, 191) | Change from Baseline to Day 30 (n=193,189) |
---|
Enteric-coated Mycophenolate Sodium | 0.7 | 0.4 | -0.3 |
,Mycophenolate Mofetil | 0.6 | 0.4 | -0.2 |
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Change in Gastrointestinal Symptom Rating Scale Subscale Scores After 30 Days of Treatment
The GSRS has five subscales (reflux, diarrhea, constipation, abdominal pain, indigestion) producing a mean subscale score ranging from 1 (=no discomfort at all) to 7 (very severe discomfort). The mean score at baseline (BL), the mean score at Day 30 and the mean Change from BL to Day 30 is presented for each of the five subscales. (NCT00400400)
Timeframe: Baseline to Day 30
Intervention | Score on a scale (Mean) |
---|
| Diarrhea: Baseline (BL) | Diarrhea: Day 30 | Diarrhea: Change from BL to Day 30 | Indigestion : Baseline | Indigestion: Day 30 | Indigestion: Change from BL to Day 30 | Constipation: Baseline | Constipation: Day 30 | Constipation: Change from BL to Day 30 | Abdominal pain: Baseline | Abdominal pain: Day 30 | Abdominal pain: Change from BL to Day 30 | Reflux : Baseline | Reflux : Day 30 | Reflux : Change from BL o Day 30 |
---|
Enteric-coated Mycophenolate Sodium | 3.3 | 2.4 | -0.9 | 2.8 | 2.1 | -0.7 | 2.2 | 1.7 | -0.4 | 2.3 | 1.8 | -0.5 | 2.2 | 1.7 | -0.5 |
,Mycophenolate Mofetil | 3.3 | 2.5 | -0.8 | 2.8 | 2.4 | -0.5 | 2.1 | 1.8 | -0.3 | 2.5 | 2.0 | -0.5 | 2.2 | 1.7 | -0.5 |
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Number of Participants With Biopsy-proven Acute Rejection (BPAR) and Treated Acute Rejection (TAR)
"TAR was defined as an episode of acute rejection that was suspected on clinical grounds and was treated and confirmed by the investigator according to the patient's response to therapy.~BPAR was defined a treated acute rejection that was confirmed by biopsy. A graft core biopsy was performed before or within 24 hours of initiation of anti-rejection therapy and was assessed by the pathologist at the center according to the BANFF 1997 criteria." (NCT00400400)
Timeframe: 30 days
Intervention | Participants (Number) |
---|
| BPAR | TAR |
---|
Enteric-coated Mycophenolate Sodium | 0 | 0 |
,Mycophenolate Mofetil | 1 | 1 |
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Number of Participants With Reported Dose Changes or Interruption of Study Medication During the 30 Days of Treatment
The number of participants with reported dose changes or interruption of study medication during the 30 days of treatment.The most common dose adjustments were dose increases back to baseline levels following a decrease or interruption and decreases due to abnormal laboratory value Adverse Events (leucopenia, thrombocytopenia, neutropenia, or anemia). (NCT00400400)
Timeframe: 30 days
Intervention | Participants (Number) |
---|
| Study drug change or interruption: YES | Study drug change or interruption: NO |
---|
Enteric-coated Mycophenolate Sodium | 11 | 188 |
,Mycophenolate Mofetil | 12 | 185 |
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The Number of Participants Who Responded to the Conversion to Mycophenolate Sodium (EC-MPS) Therapy
Response assessed using the Gastrointestinal Symptom Rating Scale (GSRS), designed to assess common symptoms with gastrointestinal (GI) disorders. The GSRS has 5 subscales (reflux, diarrhea, constipation, abdominal pain and indigestion) producing a mean subscale score ranging from 1 (no discomfort) to 7 (very severe discomfort). The total score is an average of scores across all 15 items; a higher score indicates more GI symptoms. Response was defined as Day 30 improvement in the GSRS Total Score (change from baseline) of greater than or equal to 0.3. Minimum score is 1; maximum score is 7. (NCT00400400)
Timeframe: Baseline, Day 30
Intervention | Participants (Number) |
---|
| Response | No Response |
---|
Enteric-coated Mycophenolate Sodium | 123 | 74 |
,Mycophenolate Mofetil | 109 | 88 |
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Acute Graft-vs-Host-Disease (aGvHD)
Development of acute graft-vs-host-disease (aGvHD) within 6 months, for participants receiving Allo-HCT. (NCT00185614)
Timeframe: 6 months
Intervention | Participants (Count of Participants) |
---|
Auto- Then Allo-HCT | 7 |
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Event-free Survival (EFS)
"Event-free survival (EFS) as determined for all participants who received the initial Auto-HCT treatment. Event was defined as any of the following within 3 years of the participant's last infusion of Auto-HCT or Allo-HCT: relapse; death; or last follow-up if there is no data to document the participant remained alive at 3 years." (NCT00185614)
Timeframe: 3 years
Intervention | Participants (Count of Participants) |
---|
| Auto-HCT only | Auto-HCT then Allo-HCT | All Participants |
---|
Auto- Then Allo-HCT | 0 | 24 | 24 |
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Overall Survival (OS)
Overall Survival (OS) as determined for all participants who received the initial Auto-HCT treatment, as assessed from the date of the last transplant. (NCT00185614)
Timeframe: 3 years
Intervention | participants (Number) |
---|
| Auto-HCT only | Auto-HCT then Allo-HCT | All Participants |
---|
Auto- Then Allo-HCT | 1 | 41 | 42 |
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Relapse Rate
Relapse rate as determined for all participants who received the initial Auto-HCT treatment. Relapse was protocol-specified as progressive disease, indicated by an increase as compared to pre-Auto-HCT baseline, of serum or urine monoclonal protein >25%; bone marrow plasmacytosis >25%; or bone lesions on skeletal survey (any increase). (NCT00185614)
Timeframe: 3 years
Intervention | participants (Number) |
---|
| Auto-HCT only | Auto-HCT then Allo-HCT | All Participants |
---|
Auto- Then Allo-HCT | 2 | 29 | 31 |
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Number of Participants With Any Treatment Failure
Treatment failures were defined as a composite endpoint of biopsy proven acute rejection (BPAR), graft loss, and death, loss to follow up and discontinuations from study drug treatment due to lack of efficacy or toxicity (at least one condition must be present) during the first 6 months or until final assessment. Any participants who were suspected of having acute rejection episodes had biopsies performed to prove whether a rejection had occurred. Graft loss was considered as the day the patient started dialysis and was not able to subsequently be removed or the day of graft nephrectomy. (NCT00369278)
Timeframe: 6 months
Intervention | Participants (Number) |
---|
Intensified Mycophenolate Sodium | 19 |
Standard Mycophenolate Sodium | 24 |
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Time to First Occurrence of a Mycophenolic Acid (MPA) Plasma Concentration of ≥ 40 mg*h/L
Non-compartmental MPA pharmacokinetic parameters were derived from individual plasma concentration-time profiles using WinNonLin 5.2 software. The areas under the curve were calculated by means of the linear trapezoidal rule. (NCT00369278)
Timeframe: Assessed on day 3, 10, 21, 42, 56 and 84
Intervention | Days (Median) |
---|
Intensified Mycophenolate Sodium | 7.00 |
Standard Mycophenolate Sodium | 43.00 |
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Number of Participants With Single Treatment Failures
"Rates for all individual components of the primary endpoint 'treatment failure' until day 180:~Acute rejection diagnosed by biopsy (BPAR)~graft loss~death~loss to follow up~discontinuation from study drug due to lack of efficacy or toxicity (adverse events, every adverse event had to be interpreted as toxicity)~conversion to another dosing regimen (conversion to tacrolimus, prograf, etc.)" (NCT00369278)
Timeframe: 6 months
Intervention | Participants (Number) |
---|
| Biopsy-proven acute rejection | Graft loss | Death | Loss to follow-up | Discontinuation due to lack of efficacy / toxicity | Conversion of doses | Treated rejections |
---|
Intensified Mycophenolate Sodium | 2 | 1 | 0 | 1 | 17 | 8 | 13 |
,Standard Mycophenolate Sodium | 11 | 2 | 0 | 0 | 15 | 10 | 24 |
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Renal Function as Measured by Glomerular Filtration Rate (GFR)
"The Glomerular Filtration Rate (GFR) was calculated using the following formulas:~Cockcroft-Gault formula: calculation using the participant's age, gender, weight, and serum creatinine levels.~MDRD formula: calculation using the participant's age, gender, serum creatinine, urea nitrogen, and albumin levels." (NCT00369278)
Timeframe: 6 months
Intervention | ml/min (Mean) |
---|
| MDRD formula: Baseline [n=44, 56] | MDRD formula: End of study [n=61, 64] | Cockcroft-Gault formula: Baseline [n=61, 65] | Cockcroft-Gault formula: End of study [n=63, 65] |
---|
Intensified Mycophenolate Sodium | 9.0 | 41.1 | 12.1 | 52.4 |
,Standard Mycophenolate Sodium | 9.4 | 40.6 | 12.0 | 47.9 |
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Renal Function as Measured by Serum Creatinine
(NCT00369278)
Timeframe: 6 months
Intervention | mg/dL (Mean) |
---|
| Baseline | End of study |
---|
Intensified Mycophenolate Sodium | 8.0 | 2.2 |
,Standard Mycophenolate Sodium | 7.8 | 2.6 |
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Change in Proteinuria - Uprotein/Creatinine Ratio
Urine protein/creatinine ratio after 6 months treatment with MMF or placebo. (NCT00318474)
Timeframe: Plan was to measure uprotein/creatinine ratio for 12 months on MMF or placebo, and then 12 months post-treatment. Data given after 6 months MMF/placebo.
Intervention | ratio (Mean) |
---|
Mycophenolate Mofetil (MMF) | 1.40 |
Placebo | 1.58 |
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Number of Participants Who Died Due to Transplant
Determine the incidence of transplant-related mortality at 6 months after UCBT (NCT00309842)
Timeframe: At Month 6
Intervention | Participants (Count of Participants) |
---|
Unrelated UCBT for Blood Cancers | 58 |
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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
Intervention | Participants (Count of Participants) |
---|
Unrelated UCBT for Blood Cancers | 130 |
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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
Intervention | Participants (Count of Participants) |
---|
Unrelated UCBT for Blood Cancers | 39 |
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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
Intervention | Participants (Count of Participants) |
---|
Unrelated UCBT for Blood Cancers | 21 |
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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
Intervention | Participants (Count of Participants) |
---|
Unrelated UCBT for Blood Cancers | 159 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Unrelated UCBT for Blood Cancers | 99.1 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Unrelated UCBT for Blood Cancers | 100 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Unrelated UCBT for Blood Cancers | 98.1 |
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Percentage Chimerism on Day 100
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: Day 100
Intervention | percentage of donor cells (Mean) |
---|
Unrelated UCBT for Blood Cancers | 98.1 |
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Percentage Chimerism on Day 21
Chimerism studies will be performed on the bone marrow on days 21 and 100 and at 6 months, 1 year and 2 years. In cases of slow engraftment a bone marrow biopsy may be repeated on day +28. (NCT00309842)
Timeframe: Day 21
Intervention | percentage of donor cells (Mean) |
---|
Unrelated UCBT for Blood Cancers | 92.6 |
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Number of Participants With Acute Graft-Versus-Host Disease
"Determine the incidence of grade II-IV and grade III-IV acute graft-versus-host disease (GVHD) at day 100 after UCBT. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria following Przepiorka et al, 1995, Consensus Clinical Stage and Grade of Acute GVHD.~Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level." (NCT00309842)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
| Grade II-IV | Grade III-IV |
---|
Unrelated UCBT for Blood Cancers | 106 | 49 |
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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
Intervention | percentage 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
Intervention | percentage of participants (Number) |
---|
Treatment (Stem Cell Transplant With GVHD Prophylaxis) | 66.7 |
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Progression Free Survival
Kaplan-Meier estimation of the percentage of patients who are alive without progressive disease at one year. (NCT02248597)
Timeframe: At 12 months
Intervention | percentage 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
Intervention | percentage of participants (Number) |
---|
Treatment (Stem Cell Transplant With GVHD Prophylaxis) | 51.8 |
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Relapse-free Mortality
Non-relapse mortality will be defined as time from registration to death due to anything other than relapse of hematological malignancy. Patients who relapse will be treated as a competing risk. (NCT02248597)
Timeframe: At 12 months
Intervention | days (Mean) |
---|
Treatment (Stem Cell Transplant With GVHD Prophylaxis) | 86.8 |
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Number of Participants With Graft and Patient Survivals at 6 Months
Graft survival was defined as the number of patients with no graft loss. The allograft was presumed lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient went through a graft nephrectomy, then the day of nephrectomy was the day of graft loss. Patient survival was defined as the number of patients alive with or without a functioning graft. (NCT00284934)
Timeframe: 6 months
Intervention | Participants (Number) |
---|
| Graft survival | Patient survival |
---|
High EC-MPS | 45 | 45 |
,Standard Dose EC-MPS | 47 | 47 |
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Number of Participants With Treatment Failure Parameters (Biopsy-Proven Acute Rejection (BPAR), Graft Loss, Death, or Loss to Follow-up) at 6 Months
A biopsy-proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB, or III based on the Banff 1997 classification.The allograft was presumed lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient went through a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00284934)
Timeframe: 6 months
Intervention | Participants (Number) |
---|
| Biopsy proven acute rejection | Treated acute rejection | Graft loss | Death |
---|
High EC-MPS | 0 | 0 | 0 | 0 |
,Standard Dose EC-MPS | 0 | 0 | 0 | 0 |
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Renal Function Assessed by Change in Estimated Glomerular Filtration Rate(eGFR)
Change in estimated glomerular filtration rate from baseline to Month 6 calculated by using abbreviated Modification of Diet in Renal Disease (MDRD) formula. Modification of Diet in Renal Disease (MDRD) formula is: GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where -C is the serum concentration of creatinine [mg/dL], -A is patient age at sample collection date [years], -G=0.742 when gender is female, otherwise G=1, -R=1.21 when race is black, otherwise R=1. (NCT00284934)
Timeframe: Baseline and Month 6
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Baseline (n= 47, 45) | Month 6 (n= 45, 43) | Change from Baseline - Month 6 (n= 45, 43) |
---|
High EC-MPS | 56.4 | 49.1 | 2.4 |
,Standard Dose EC-MPS | 45.3 | 44.7 | -0.4 |
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Renal Function at 3 Months Assessed by Change in Estimated Glomerular Filtration Rate (eGFR)
Change in estimated glomerular filtration rate from baseline to Month 3 calculated by using abbreviated MDRD formula. Modification of Diet in Renal Disease (MDRD) formula is: GFR [mL/min/1.73m^2] = 186.3*(C^-1.154)*(A^-0.203)*G*R where -C is the serum concentration of creatinine [mg/dL], -A is patient age at sample collection date [years], -G=0.742 when gender is female, otherwise G=1, -R=1.21 when race is black, otherwise R=1. (NCT00284934)
Timeframe: Baseline and 3 months
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Baseline (n= 47, 45) | Month 3 (n= 44, 43) | Change from Baseline to Month 3 (n= 44, 43) |
---|
High EC-MPS | 46.4 | 48.6 | 2.1 |
,Standard Dose EC-MPS | 45.3 | 44.6 | -0.4 |
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Acute Rejection Rate
Biopsy proven acute rejection defined by biochemical and histological changes as well as the need for temporary steroid use occurred in 1 patient in each group both of which were steroid responsive (NCT00296244)
Timeframe: 6 months post-transplant
Intervention | Percentage of participants (Number) |
---|
Control Group | 5 |
Study Group | 5 |
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Incidence and Severity of HCV Recurrence Post-OLT
The incidence and severity of HCV recurrence based on Hepatitis C PCR levels and protocol liver biopsy findings were found to be similar between the 2 groups. (NCT00296244)
Timeframe: 6 months post-transplant
Intervention | Percentage of participants (Number) |
---|
Control Group | 27 |
Study Group | 29 |
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Infection as an Adverse Effect of Steroids
Incidence of bacterial infection was similar in the control group as well as study group, 4 patients in both groups had infection (NCT00296244)
Timeframe: 3 months post-transplant
Intervention | Percentage of participants (Number) |
---|
Control Group | 20 |
Study Group | 21 |
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New-onset Diabetes Mellitus (NODM) as Secondary Outcome
The incidence of new-onset Diabetes mellitus (NODM, based on percentage of previously non-diabetic patients who developed DM post-transplantation, was similar between the 2 groups. (NCT00296244)
Timeframe: 6 months
Intervention | Percentage of participants (Number) |
---|
Control Group | 40 |
Study Group | 42 |
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Graft Survival Rate
Percentage of recipients whose liver grafts are still working at the end of 1 and 2 years. (NCT00296244)
Timeframe: 1 and 2 years
Intervention | percentage of participants (Number) |
---|
| 1-year graft survival rate | 2-year graft survival rate |
---|
Control Group | 100 | 90 |
,Study Group | 94.7 | 84 |
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Patient Survival Rate
Percentage of recipients who are still alive at the end of 1 and 2 years. (NCT00296244)
Timeframe: 1 and 2 years
Intervention | Percentage of participants (Number) |
---|
| 1-year patient survival rate | 2-year patient survival rate |
---|
Control Group | 100 | 90 |
,Study Group | 94.7 | 84 |
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Gastrointestinal Symptoms Under MMF-based Immunosuppressive Therapy
Assessed by GI complications at baseline. (NCT00267150)
Timeframe: week 0
Intervention | Participants (Number) |
---|
| Any complication | Diarrhea | Dyspepsia | Nausea | Abdominal pain/bloating/fullness | Other |
---|
Enteric Coated Mycophenolate-sodium | 19 | 12 | 4 | 3 | 11 | 1 |
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Non-inferiority Analysis on Percentage of Participants With Composite Efficacy Endpoints
The primary efficacy endpoint was the 12 month analysis of primary efficacy failure defined as a composite endpoint including treated biopsy proven acute rejection (BPAR), graft loss, death or loss to follow-up. In the definition of composite efficacy failure, loss to follow-up includes patients who did not experience treated BPAR, graft loss or death on or after day 1 and whose last day of contact was prior to day 316, the start day of the 12 month visit window. (NCT00251004)
Timeframe: 12 months
Intervention | Percentage of Participants (Number) |
---|
Low-dose Everolimus Group | 27.1 |
High-dose Everolimus Group | 21.5 |
Control Group | 25.3 |
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Percentage of Participants With the Composite Incidence of Graft Loss, Death or Loss to Follow up at 12 Months Post-transplantation
"Graft loss was defined as graft loss (the allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis) and re-transplant.~A loss to follow-up patient in the composite endpoint of graft loss, death or loss to follow-up (the main secondary efficacy endpoint) was a patient who did not experience graft loss or death from day 1 and whose last day of contact was prior to study day 316." (NCT00251004)
Timeframe: 12 months
Intervention | Percentage of participants (Number) |
---|
Low-dose Everolimus Group | 11.6 |
High-dose Everolimus Group | 9.7 |
Control Group | 9.4 |
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Number of Participants With Composite Efficacy Endpoints - 12 Month Analysis
The primary efficacy endpoint was the 12 month analysis of primary efficacy failure defined as a composite endpoint including treated biopsy proven acute rejection (BPAR), graft loss, death or loss to follow-up. A treated BPAR episode was defined as a biopsy graded IA, IB, IIA, IIB, or III that was treated with anti-rejection therapy. Graft loss was defined as the allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis as well as re-transplant. (NCT00251004)
Timeframe: 12 months
Intervention | Participants (Number) |
---|
| Composite Endpoint (n) | --Death | --Graft Loss | --Treated BPAR | --Lost to follow up |
---|
Control Group | 70 | 6 | 9 | 50 | 9 |
,High-dose Everolimus Group | 60 | 9 | 13 | 38 | 6 |
,Low-dose Everolimus Group | 75 | 8 | 13 | 48 | 12 |
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The Number of Kidney Transplant up to 12 Months.
The number of kidney transplants up to month 12. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for infection over 12 months or until they seperated from the study. (NCT00446459)
Timeframe: Enrollment to month 8 or month 12 post enrollment.
Intervention | Participants (Number) |
---|
Mycophenolate Mofetil (MMF) Single Arm Study | 3 |
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The Number of Pariticpants With a White Blood Cell Count Below 2.0 Thousand (Low) or Total IgG/IgM Titers Below Range (620-1490 mg/dL).
The number of subjects with adverse hematologic effects with MMF while on-study. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for hematologic effects up to 12 months. (NCT00446459)
Timeframe: Enrollment to month 12.
Intervention | Participant (Number) |
---|
Mycophenolate Mofetil (MMF) Single Arm Study | 0 |
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The Number of Subjects With a 10% Decrease in PRA Level at Month 8.
(NCT00446459)
Timeframe: Enrollment to month 8
Intervention | Participant (Number) |
---|
Mycophenolate Mofetil (MMF) Single Arm Study | 9 |
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The Number of Subjects With Significant Infections up to Month 12.
The number of infections while on-study up to month 12. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for infection over 12 months or until they seperated from the study. (NCT00446459)
Timeframe: From enrollment to month 12.
Intervention | Participants (Number) |
---|
Mycophenolate Mofetil (MMF) Single Arm Study | 4 |
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The Number of Transplants With a Negative Crossmatch at Transplant.
The number negative crossmatch transplants up to month 12. Positivie crossmatch transplant carries a higher risk for rejection. Subjects who's PRA decreased by 10% at month 8 and who went on to the Mycophenolate mofetil + Rituximab study were followed to month 8. Those subjects who stayed on the Mycophenolate mon-therapy study were observed for negative crossmatch transplants to 12 months. (NCT00446459)
Timeframe: Number of Transplants with a Negative Crossmatch.
Intervention | Participant (Number) |
---|
Mycophenolate Mofetil (MMF) Single Arm Study | 0 |
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Number of Biopsy Proven Rejections at 12 Months
assessed by liver biopsy using Banff International Consensus Schema (NCT00206076)
Timeframe: 12 months
Intervention | participants (Number) |
---|
MMF, CNI Discontinued | 0 |
MMF; CNI Decreased | 0 |
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Number of Participants With Adverse Events Including Infections at 12 Months
(NCT00206076)
Timeframe: 12 months
Intervention | participants (Number) |
---|
MMF, CNI Discontinued | 2 |
MMF; CNI Decreased | 1 |
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Patient and Graft Survival at 12 Months
(NCT00206076)
Timeframe: 12 months
Intervention | participants (Number) |
---|
CNI Discontinued | 6 |
CNI Reduction | 9 |
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Number of Participants Who Experienced Adverse Events, Serious Adverse Events and Death
Participants with adverse events (serious plus non-serious), serious adverse events and death were reported. (NCT00956293)
Timeframe: Months 6, 12, 24, 36, 48 and 60
Intervention | Participants (Number) |
---|
| Adverse events (serious and non-serious) | Serious adverse events | Deaths |
---|
Control Group | 21 | 11 | 0 |
,Everolimus Group | 50 | 28 | 0 |
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Assessment of GFR by the Cockcroft-Gault Method (LOCF)
the GFR was also calculated using the Cockcroft-Gault method (Cockcroft and Gault 1976) and the Modification of Diet in Renal Disease (MDRD) method (Levey et al., 1999, Rodrigo et al., 2003; Pierrat et al., 2003).Cockcroft-Gault formula For men: GFR= (140-Age)X Body Weight[kg]/72X Serum Creatinine[mg/dl] For women: GFR= 0,85x(140-Age) x Body Weight[kg]/72x Serum Creatinine [mg/dl] The Last Observation Carried Forward (LOCF) imputation technique was used for this analysis. (NCT00965094)
Timeframe: 9 months
Intervention | mL/min (Mean) |
---|
Everolimus | 72.6 |
Reference Therapy | 72.7 |
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Renal Function Assessed as Glomerula Filtration Rate (GFR) - Nankivell Method - 9 Months After Renal Transplantation (LOCF)
The glomerular filtration rate (GFR) is the best clinical estimate of renal function in health and disease, and correlates well with the clinical severity of renal function disturbances. The GFR, calculated according to the Nankivell formula, was used as the primary outcome measure in this study. This equation has been validated in renal transplant patients against the true GFR measured by a radionuclide method and has been confirmed as a very accurate method to calculate the GFR in this specific population (Gaspari et al., 2004)GFR = 6.7 / Scr + BW / 4 - Surea / 2-100 / (height)² + C Scr = serum creatinine concentration expressed in mmol/L. BW = body weight in kg, Surea = serum urea in mmol/L and Height is expressed in meters.The Last Observation Carried Forward (LOCF) imputation technique was used for this analysis. (NCT00965094)
Timeframe: 9 months
Intervention | mL/min/1.73m^2 (Mean) |
---|
Everolimus | 15.0 |
Reference Therapy | 16.6 |
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Change in Renal Function (Creatinine Slope)
X(slope)=(1/value of creatinine). (NCT00965094)
Timeframe: 3 months, 5 months, 7 months, 9 months
Intervention | mg/dl per month (Mean) |
---|
| (ITT) Baseline 2: Month 3 N=15, 15 | (ITT) Month 3 (1st week) N=15, 4 | (ITT) Month 3 (2nd week) N=15, 4 | (ITT) Month 3 (3rd week) N=13, 2 | (ITT) Month 3 (4th week) N=12, 0* | (ITT) Month 5 N=14, 15 | (ITT) Month 7 N=14, 14 | (ITT) Month 9 N=11, 15 | (PP) Baseline 2: Month 3 N=11, 15 | (PP) Month 3 (first week) N=11, 4 | (PP) Month 3 (second week) N=11, 4 | (PP) Month 3 (third week) N=10, 2 | (PP) Month 3 (fourth week) N=9, 0* | (PP) Month 5 N=11, 15 | (PP) Month 7 N=11, 14 | (PP) Month 9 N=11, 15 |
---|
Everolimus | 0.7 | 0.7 | 0.8 | 0.7 | 0.8 | 0.8 | 0.7 | 0.8 | 0.7 | 0.7 | 0.8 | 0.7 | 0.8 | 0.8 | 0.8 | 0.8 |
,Reference Therapy | 0.7 | 0.8 | 0.9 | 0.9 | NA | 0.7 | 0.7 | 0.8 | 0.7 | 0.8 | 0.9 | 0.9 | NA | 0.7 | 0.7 | 0.8 |
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Participants Who Had Occurrence of Biopsy Proven Acute Rejection, Graft Loss or Death.
Biopsy-proven acute rejection was defined as a biopsy gradeed IA, IB, IIA, IIB, or III. The allograft was presumed to be lost if the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, then the day of nephrectomy was the day of graft loss. (NCT00965094)
Timeframe: 9 months
Intervention | Participants (Number) |
---|
| No | Yes |
---|
Everolimus | 14 | 1 |
,Reference Therapy | 14 | 1 |
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Participants Who Had Occurrence of Treatment Failure.
Treatment failure was defined as a composite endpoint of biopsy-proven acute rejection, graft loss, death, loss to follow up, discontinuation due to lack of efficacy or toxicity or conversion to another regimen. (NCT00965094)
Timeframe: 9 months
Intervention | Participants (Number) |
---|
| No | Yes |
---|
Everolimus | 10 | 5 |
,Reference Therapy | 14 | 1 |
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Mean Creatinine Clearance Rate
Creatinine clearance is a measure of kidney function. Creatinine clearance rate is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Creatinine clearance can be measured directly or estimated using established formulas. For this study, the creatinine clearance rate was calculated using the Nankivell formula. Normal values for healthy, young males are in the range of 100-135 ml/min and for females 90-125 ml/min. A low creatinine clearance indicates poor kidney function. (NCT00195273)
Timeframe: 12 months
Intervention | ml/min (Mean) |
---|
Sirolimus | 56.800 |
Cyclosporine (CsA) | 53.449 |
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Mean Creatinine Clearance Rate - 3 Months
Creatinine clearance is a measure of kidney function. Creatinine clearance rate is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Creatinine clearance can be measured directly or estimated using established formulas. For this study, the creatinine clearance rate was calculated using the Nankivell formula. Normal values for healthy, young males are in the range of 100-135 ml/min and for females 90-125 ml/min. A low creatinine clearance indicates poor kidney function. (NCT00195273)
Timeframe: 3 months
Intervention | ml/min (Mean) |
---|
Sirolimus | 55.200 |
Cyclosporine (CsA) | 51.878 |
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Number of Patients With Acute Rejection
The diagnosis of acute rejection was made via kidney biopsy (Banff criteria). The Banff criteria are standardized diagnostic categories based on histological assessments (e.g., cell types and distributions). Biopsy was performed before initiation of anti-rejection therapy, or at least within 24 hours of the start of therapy. (NCT00195273)
Timeframe: 3 and 12 months
Intervention | patients (Number) |
---|
| 3 months | 12 months |
---|
Cyclosporine (CsA) | 9 | 10 |
,Sirolimus | 3 | 5 |
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Patient and Graft Survival
Graft survival is measured by graft loss which is defined as removal of the transplant. (NCT00195273)
Timeframe: 12 months
Intervention | patients (Number) |
---|
| Patient survival | Graft loss |
---|
Cyclosporine (CsA) | 29 | 3 |
,Sirolimus | 31 | 0 |
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Number of Participants With Adverse Events (AEs)
An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. (NCT01085097)
Timeframe: Baseline up to Week 28
Intervention | participants (Number) |
---|
Placebo | 14 |
Laquinimod 0.5 mg | 15 |
Laquinimod 1 mg | 15 |
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Percent Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Week 24
Estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease (MDRD) formula. (NCT01085097)
Timeframe: Baseline, Week 24
Intervention | percent change (Mean) |
---|
Placebo | 12.1 |
Laquinimod 0.5 mg | 18.0 |
Laquinimod 1 mg | 24.3 |
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Incidence of Hepatotoxicity as Measured by Bilirubin
100-day incidence of hepatotoxicity will be compared using a Gray test. Median and range of largest total bilirubin (mg/dL), (NCT01951885)
Timeframe: Up to day +100
Intervention | mg/dL (Median) |
---|
Group A (Tacrolimus, Methotrexate) | 1.5 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 1.1 |
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Incidence of Hepatotoxicity as Measured by Veno-occlusive Disease (VOD)
100-day incidence of hepatotoxicity will be compared using the Gray test. Percentage of patients with VOD and 95% confidence interval (NCT01951885)
Timeframe: Up to day +100
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 8 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 2 |
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Incidence of Infection
100-day incidence of infection will be compared using a the Gray test. (NCT01951885)
Timeframe: Up to day +100
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 63 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 53 |
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Incidence of Pulmonary Toxicity Measured by Pulmonary Edema
180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with pulmonary edema and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 14 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 4 |
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Incidence of Pulmonary Toxicity Measured by Pulmonary Hemorrhage
180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with pulmonary hemorrhage and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 2 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 2 |
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Incidence of Pulmonary Toxicity Measured by Respiratory Failure
180-day incidence of pulmonary toxicity will be compared using the Gray test. Percentage of patients with respiratory failure and 95% confidence interval (NCT01951885)
Timeframe: Up to day +180
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 9 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 6 |
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Length of Hospitalization
Hospital stay will be compared between patients in groups A and B using the Wilcoxon rank sum test. (NCT01951885)
Timeframe: Date of transplant to date of discharge, assessed up to 1 year
Intervention | days (Median) |
---|
Group A (Tacrolimus, Methotrexate) | 31 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 27 |
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Length of Time on Continuous Infusion Narcotics
Start and stop dates for the need of continuous IV infusion of narcotics for severe mucositis pain will be recorded. Time on IV infusion will be compared between patients in groups A and B using the Wilcoxon rank sum test. (NCT01951885)
Timeframe: up to +28 day
Intervention | days (Median) |
---|
Group A (Tacrolimus, Methotrexate) | 10 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 9 |
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Overall Survival
Estimated using the Kaplan-Meier method and compared between the 2 groups using the log-rank test. (NCT01951885)
Timeframe: Up to 1 year
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 71 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 72 |
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Percentage of Participants Using Total Parenteral Nutrition (TPN) Within 100 Days
TPN use will be compared using the Chi-square test. (NCT01951885)
Timeframe: Up to day 100
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 41 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 38 |
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Percentage of Severe (Grade 3-4) Mucositis Graded According to the World Health Organization (WHO) Grading Scale
"Participants (percentage of) will be graded three times per week through day 28 of the study. Incidence will be compared through Wilcoxon rank sum tests.~The WHO grading scale for mucositis is scaled depending on the severity of mucositis symptoms, with a lower stage associated with a better outcome and greater stages associated with worse outcomes. The staging is as follows:~Stage 0: None Stage 1 (mild): Oral soreness, erythema Stage 2 (moderate): Oral erythema, ulcers, solid diet tolerated Stage 3 (severe): Oral ulcers, liquid diet only Stage 4 (life-threatening): Oral alimentation impossible" (NCT01951885)
Timeframe: Up to day 28
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 81.6 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 57.4 |
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Progression-free Survival
Estimated using the Kaplan-Meier method and compared between the 2 groups using the log-rank test. (NCT01951885)
Timeframe: Up to 1 year
Intervention | percentage of participants (Number) |
---|
Group A (Tacrolimus, Methotrexate) | 59 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 68 |
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Time to Neutrophil Engraftment
The number of days to reach a neutrophil count of greater than or equal to 500/ul for three consecutive laboratory values obtained on different days. The day of engraftment will be the first day of the three consecutive laboratory values. (NCT01951885)
Timeframe: Up to 28 days
Intervention | days (Median) |
---|
Group A (Tacrolimus, Methotrexate) | 17 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 15 |
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Time to Platelet Engraftment
The number of days to reach a platelet count of greater than or equal to 20,000/ul for three consecutive laboratory values obtained on different days, independent of platelet transfusions the prior 7 days. The day of engraftment will be the first day of the three consecutive laboratory values. For cases of delayed engraftment beyond 28 days, results will be recorded once the participant engrafts. (NCT01951885)
Timeframe: The date the participant engrafts, up to 28 days
Intervention | days (Median) |
---|
Group A (Tacrolimus, Methotrexate) | 27 |
Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 23 |
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Cumulative Incidence of Participants With Acute GVHD
"Acute GVHD by grade will be estimated using cumulative incidence methods and compared using the Gray test.~A lower clinical grade and/or stage of GVHD corresponds to a better participant outcome and a higher grade corresponds to a worse participant outcome. Grading is as follows:~Grade 0: No stage 1-4 of any organ Grade 1: Stage 1-2 skin without liver, upper GI, or lower GI involvement. Grade 2: Stage 3 rash and/or stage 1 liver and/or stage 1 upper GI and/or stage 1 lower GI.~Grade 3: Stage 2-3 liver and/or stage 2-3 lower GI, with stage 0-3 skin and/or stage 0-1 upper GI.~Grade 4: Stage 4 skin, liver, or lower GI involvement, with stage 0-1 upper GI.~A greater stage of GVHD involves worsening end-organ involvement and worse participant outcomes based on the skin, liver, lower GI, and upper GI." (NCT01951885)
Timeframe: Day 7- Day 100
Intervention | percentage of participants (Number) |
---|
| Grade 1-4 | Grade 2-4 | Grade 3-4 |
---|
Group A (Tacrolimus, Methotrexate) | 37 | 27 | 4 |
,Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 47 | 28 | 13 |
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Incidence of Chronic GVHD
"Chronic GVHD will be graded as mild, moderate, or severe based on the National Institutes of Health Consensus Development Project. The type and duration of immunosuppressive treatment given for cGVHD will be recorded. Mild grades are associated with a better participant outcome and severe corresponds to a worse participant outcome. Grading is as follows:~Mild: 1 or 2 organs involved with no more than score 1 plus Lung score 0~Moderate: 3 or more organs involved with no more than score 1 OR At least 1 organ (not lung) with a score of 2 OR Lung score 1~Severe: At least 1 organ with a score of 3 OR Lung score of 2 or 3~Organ scores can range from 0 to 3, with 0 being no symptoms on the target organ and a better participant outcome while a score of 3 correlates to severe symptoms on the target organ and worse participant outcomes.~Potential target organs include: skin, mouth, eyes, GI tract, liver, lungs, joint /fascia, and genital tract" (NCT01951885)
Timeframe: at 12 months
Intervention | percentage of participants (Number) |
---|
| any chronic GVHD | moderate-severe chronic GVHD |
---|
Group A (Tacrolimus, Methotrexate) | 25 | 20 |
,Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 36 | 23 |
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Incidence of Chronic GVHD
"Chronic GVHD will be graded as mild, moderate, or severe based on the National Institutes of Health Consensus Development Project. The type and duration of immunosuppressive treatment given for cGVHD will be recorded. Mild grades are associated with a better participant outcome and severe corresponds to a worse participant outcome. Grading is as follows:~Mild: 1 or 2 organs involved with no more than score 1 plus Lung score 0~Moderate: 3 or more organs involved with no more than score 1 OR At least 1 organ (not lung) with a score of 2 OR Lung score 1~Severe: At least 1 organ with a score of 3 OR Lung score of 2 or 3~Organ scores can range from 0 to 3, with 0 being no symptoms on the target organ and a better participant outcome while a score of 3 correlates to severe symptoms on the target organ and worse participant outcomes.~Potential target organs include: skin, mouth, eyes, GI tract, liver, lungs, joint /fascia, and genital tract" (NCT01951885)
Timeframe: at 6 months
Intervention | percentage of participants (Number) |
---|
| any chronic GVHD | moderate-severe chronic GVHD |
---|
Group A (Tacrolimus, Methotrexate) | 16 | 12 |
,Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 15 | 11 |
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Incidence of Hepatotoxicity as Measured by Elevated Liver Enzymes
100-day incidence of hepatotoxicity will be compared using a Gray test. Percentage of patients with elevated Aspartate Aminotransferase (AST), Alanine Aminotransferase (ALT), and alkaline phosphatase and a 95% confidence interval (NCT01951885)
Timeframe: Up to day +100
Intervention | percentage of participants (Number) |
---|
| AST | ALT | Alkaline Phosphatase |
---|
Group A (Tacrolimus, Methotrexate) | 29 | 33 | 2 |
,Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 15 | 28 | 2 |
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Incidence of Nephrotoxicity
100-day incidence of nephrotoxicity will be compared using a Chi-squared test. Percentage of patients requiring dialysis and those with elevated creatinine using a 95% confidence interval (NCT01951885)
Timeframe: Up to day +100
Intervention | percentage of participants (Number) |
---|
| Participants requiring dialysis | Participants with elevated creatinine |
---|
Group A (Tacrolimus, Methotrexate) | 12 | 27 |
,Group B (Tacrolimus, Methotrexate, Mycophenolate Mofetil) | 4 | 2 |
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Change From Baseline in Left Ventricular Mass Index (LVMI)
Left ventricular hypertrophy grade was assessed by echocardiogram where the left ventricular mass index was calculated. The presence of LVM was defined as > 49.2 g/m^2.7 in men and >46.7 g/m^2.7 in women. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, Month 24
Intervention | g/m^2.7 (Mean) |
---|
Tacrolimus | -6.071 |
Everolimus | -4.008 |
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Percentage of Participants With Major Cardiovascular Events (MACE)
The percentage of participants who experienced MACE were reported. MACE included acute myocardial infarction, insertion or replacement of implantable defibrillator, peripheral vascular disorders, congestive heart failure, coronary artery bypass, other events, percutaneous coronary intervention and stroke. (NCT01169701)
Timeframe: Month 24
Intervention | Percentage of participants (Number) |
---|
Tacrolimus | 0.00 |
Everolimus | 0.00 |
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Change From Baseline in Cardiovascular Biomarkers, C-reactive Protein (CRP)
Blood samples were collected to analyze CRP. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | mg/dl (Mean) |
---|
| Month 6 (n=27,30) | Month 24 (n=24,24) |
---|
Everolimus | 0.326 | -0.040 |
,Tacrolimus | 0.512 | 0.100 |
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Change From Baseline in Cardiovascular Biomarkers: Troponin I and Collagen Type 1 C-telopeptide (ICTP)
Blood samples were collected to analyze Troponin I and collagen type 1 C-telopeptide (ICTP). A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | ng/ml (Mean) |
---|
| Troponin 1, Month 6 (n=27,30) | Troponin 1, Month 24 (n=24,24) | ICTP, Month 6 (n=27,30) | ICTP, Month 24 (n=24,24) |
---|
Everolimus | -0.006 | -0.007 | -0.195 | -0.125 |
,Tacrolimus | 0.000 | 0.003 | 0.049 | -0.035 |
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Change From Baseline in Mean 24 Hour Systolic and Diastolic Blood Pressure
Blood pressure was measured using ambulatory blood pressure monitoring (ABPM). A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, Month 6, month 12, month 24
Intervention | mmHg (Mean) |
---|
| Month 6 (n=31,29) | Month 12 (n=28,24) | Month 24 (n=29,24) |
---|
Everolimus | 3.2 | 2.7 | 2.0 |
,Tacrolimus | -0.6 | 2.1 | 2.2 |
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Change From Baseline in the Cardiovascular Biomarker, Glycosylated Hemoglobin (HbA1c)
Blood samples were collected to analyze HbA1c. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | Percentage of HbA1c (Mean) |
---|
| Month 6 (n=20,22) | Month12 (n=22,20) |
---|
Everolimus | 0.159 | 0.185 |
,Tacrolimus | 0.015 | 0.045 |
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Change From Baseline in the Cardiovascular Biomarker, Myeloperoxidase (MPO)
Blood samples were collected to analyze MPO. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | U/mL (Mean) |
---|
| Month 6 (n=27,30) | Month 24 (n=24,24) |
---|
Everolimus | -0.093 | -0.642 |
,Tacrolimus | 0.433 | -0.329 |
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Change From Baseline in the Cardiovascular Biomarker, N-terminal Pro-brain Natriuretic Peptide Fraction (NT-proBNP)
Blood samples were collected to analyze NT-proBNP. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | pg/mL (Mean) |
---|
| Month 6 (n=27,30) | Month 24 (n=24,24) |
---|
Everolimus | -79.10 | -193.3 |
,Tacrolimus | 21.604 | -80.20 |
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Change From Baseline in the Cardiovascular Biomarker, Type 1 Procollagen Amino-terminal-propeptide (PINP)
Blood samples were collected to analyze PCR. A negative change from baseline indicates improvement. (NCT01169701)
Timeframe: Baseline, month 6, month 24
Intervention | ug/l (Mean) |
---|
| Month 6 (n=27,30) | Month 24 (n=24,24) |
---|
Everolimus | -33.36 | -28.17 |
,Tacrolimus | -13.82 | -13.65 |
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Percentage of Participants With Biopsy-proven Acute Rejection (BPAR), Graft Loss, Death and Lost to Follow up
The incidence of BPAR, graft loss, death and lost to follow-up events was calculated using relative frequency. (NCT01169701)
Timeframe: Month 24
Intervention | Percentage of participants (Number) |
---|
| BPAR | Graft loss | Deaths | Lost to follow-up |
---|
Everolimus | 0.00 | 0.00 | 0.00 | 0.00 |
,Tacrolimus | 0.00 | 0.00 | 0.00 | 3.13 |
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Pulse Wave Velocity (PWV)
Utilizing the SphygmoCor Device, ECG leads placed at the carotid and femoral arteries provided the measure of the pulse wave at that particular arterial location. The distance between the two vascular beds divided by the pulse wave time shift provided a measure of the pulse wave velocity. (NCT01169701)
Timeframe: Month 6, month 24
Intervention | m/sec (Mean) |
---|
| Month 6 (n=31,30) | Month 24 (n=28,25) |
---|
Everolimus | 7.40 | 7.06 |
,Tacrolimus | 7.01 | 7.58 |
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Renal Function as Measured by Creatinine Clearance
Creatinine clearance was calculated using the Cockroft-Gault formula. (NCT01169701)
Timeframe: Month 6, month 12, month 24
Intervention | mg/min (Mean) |
---|
| Month 6 (n=29,25) | Month 12 (n=28,25) | Month 24 (n=28,24) |
---|
Everolimus | 76.618 | 73.363 | 72.910 |
,Tacrolimus | 64.841 | 65.037 | 66.933 |
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Renal Function as Measured by Estimated Glomerular Filtration Rate (eGFR)
Estimated GFR was caluclated using the modification of diet in renal disease (MDRD) formula. (NCT01169701)
Timeframe: Month 6, month 12, month 24
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Month 6 (n=33,29) | Month 12 (n=32,28) | Month 24 (n=31,26) |
---|
Everolimus | 63.781 | 61.225 | 60.779 |
,Tacrolimus | 55.648 | 57.757 | 57.727 |
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Renal Function Measured by Serum Creatinine
Serum samples were collected to analyze serum creatinine. (NCT01169701)
Timeframe: Month 6, month 12, month 24
Intervention | mg/dl (Mean) |
---|
| Month 6 (n=33,29) | Month 12 (n=32,28) | Month 24 (n= 31,26) |
---|
Everolimus | 1.234 | 1.256 | 1.260 |
,Tacrolimus | 1.232 | 1.231 | 1.217 |
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6-month Acute Antibody-mediated Rejection Rate (AMR)
A diagnosis of AMR was based on the 2013 international Banff Classification Criteria and is defined as the presence of circulating donor-specific antibody (DSA) and either: 1) peritubular capillary staining of C4d and at least one of the following: peritubular capillaritis (ptc) score>0, glomerulitis (g) score>0, acute thrombotic microangiopathy (TMA) in the absence of any other cause, or other features consistent with AMR (endothelial injury, fibrin thrombi, microinfarctions, interstitial hemorrhage), or 2) absence of capillary staining of C4d and the presence of ptc>0 and g>0 or ptc>0 or g>0 and acute TMA, in the absence of any other cause of TMA. (NCT00275509)
Timeframe: Up to 6 months
Intervention | Participants (Count of Participants) |
---|
Tymoglobulin | 17 |
Daclizumab | 16 |
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6-month Cumulative Rejection Incidence (Either CMR, AMR or Both)
Biopsy shows evidence of either AMR or CMR or evidence both. (NCT00275509)
Timeframe: Up to 6 months
Intervention | Participants (Count of Participants) |
---|
Tymoglobulin | 21 |
Daclizumab | 23 |
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Arthritis Complete Response
Complete response at three months (This is defined as = 0.25 of the total tender plus swollen joints observed at baseline and British Isles Lupus Assessment Group (BILAG) index C (mild) or D (no longer present) score in the Musculoskeletal system), comparing treatment to placebo group as complete responder or not complete responder. This was an intent to treat analysis so dropouts were counted as non-responders. (NCT00594932)
Timeframe: 3 months
Intervention | participants (Number) |
---|
Patients Who Received MMF for the First 3 Months | 4 |
Patients Who Received Placebo for the First 3 Months | 0 |
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Major and Partial Clinical Response
Those meeting the Criteria for Major or Complete Clinical Response as listed in the preceding endpoints or who meet criteria for partial response defined as at least a 25% decrease in tender and swollen joint count and improvement of at least one severity rating for arthritis as at least one level on the British Isles Lupus Assessment Group Index. (NCT00594932)
Timeframe: 3 Months
Intervention | participants (Number) |
---|
Patients Who Received MMF for the First 3 Months | 9 |
Patients Who Received Placebo for the First 3 Months | 5 |
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Major Arthritis Response
This is defined as at least a 50% reduction in tender + swollen joint counts and severity rated as mild as defined by the British Isles Lupus Assessment Group Index (NCT00594932)
Timeframe: 3 months
Intervention | participants (Number) |
---|
Patients Who Received MMF for the First 3 Months | 5 |
Patients Who Received Placebo for the First 3 Months | 0 |
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Area Under the Plasma Concentration-time Curve (AUC(0-6h)) of Mycophenolate Mofetil
Area under the plasma concentration-time curve during a dosage interval at steady state (calculated using the trapezoidal rule, from t = 0 to t = 6 hours). (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration
Intervention | hour* µg /ml (Mean) |
---|
CellCept | 33.523 |
Myfenax | 31.100 |
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Area Under the Plasma Concentration-time Curve (AUC(0-tau)) of Mycophenolate Mofetil
For participants with a 0-12h profile: Area under the plasma concentration-time curve during a dosage interval at steady state (calculated using the trapezoidal rule, from t = 0 to t = 12 hours). For participants with a 0-6h profile: AUC(0-tau) was calculated based on AUC(0-6h) using the extrapolation formula according to Fleming. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration
Intervention | hour* µg /ml (Mean) |
---|
CellCept | 49.846 |
Myfenax | 48.255 |
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Degree of Fluctuation of the Concentration Levels of Mycophenolate Mofetil Over One Dosing Interval (PTF)
PTF was calculated as: (Cmax-Cmin)/(AUCt/t)*100 (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration
Intervention | percentage of AUC for a dosing interval (Mean) |
---|
CellCept | 351.05 |
Myfenax | 323.67 |
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Maximum Observed Plasma Concentration (Cmax) of Mycophenolate Mofetil
Cmax was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration
Intervention | µg /ml (Mean) |
---|
CellCept | 16.189 |
Myfenax | 14.308 |
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Minimum Observed Plasma Concentration (Cmin) of Mycophenolate Mofetil
Cmin was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration and at 30 min, 1 hour, 1.5, 2, 3, 4, 5, 6, 8, 10, and 12 hours after drug administration
Intervention | µg /ml (Mean) |
---|
CellCept | 1.584 |
Myfenax | 1.567 |
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Plasma Concentrations of Mycophenolate Mofetil in Pre-Administration Samples (Cpd)
Cpd was directly obtained from measured values of plasma concentrations. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration
Intervention | µg /ml (Mean) |
---|
CellCept | 2.693 |
Myfenax | 3.001 |
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Time Corresponding to Occurrence of Cmax (Tmax) of Mycophenolate Mofetil
Tmax was directly obtained from measured values. (NCT00991510)
Timeframe: Day 14 and Day 28 (end of first two cross-over periods) before drug administration
Intervention | hours (Mean) |
---|
CellCept | 1.119 |
Myfenax | 1.344 |
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Summary of Participants With Adverse Events
"Summary of adverse events across three study time periods. The on-treatment time frame spanned the time during which study drug was administered. Relation to study drug was assessed by the investigator.~The Adverse Event count includes serious and non-serious AEs. A serious AE (SAE) was any event that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity or congenital anomaly/birth defect or was an important medical event could have jeopardized the patient's safety or required medical or surgical intervention to prevent one of the outcomes listed above.~Severity was measured on a three-point scale: mild, moderate, severe." (NCT00991510)
Timeframe: Day 1 up to Day 112
Intervention | participants (Number) |
---|
| Adverse Events | Related adverse events | Severe adverse events | Adverse events leading to discontinuation | Serious adverse events | Adverse events leading to death |
---|
CellCept | 15 | 3 | 0 | 2 | 1 | 0 |
,Myfenax | 17 | 7 | 0 | 1 | 1 | 0 |
,Overall | 26 | 9 | 0 | 3 | 1 | 0 |
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Number of Non-Relapse Mortalities (NRM)
Number of patients who expired without disease progression/relapse. (NCT01252667)
Timeframe: 100 days post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 1 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 1 |
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Number of Participants Surviving Overall
Number of patients surviving overall post-transplant. (NCT01252667)
Timeframe: 1 Year post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 2 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 1 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 22 |
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Number of Participants Surviving Progression-free.
Number of patients surviving without progressive disease post-transplant. Progression is defined as the presence of >5% aberrant blasts by morphology on a marrow aspirate or the presence of circulating blasts in the peripheral blood. (NCT01252667)
Timeframe: 1 Year post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 2 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 1 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 21 |
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Number of Participants Who Graft Rejected.
Number of patients who graft rejected post-transplant. Graft rejection is defined as <5% donor peripheral blood T cells (CD3+). (NCT01252667)
Timeframe: 1 Year post-transplant.
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
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Number of Participants With Minimal Residual/Recurring Disease (MRD) Post-transplant
Number of patients with MRD post-transplant, detected in the bone marrow as cytogenetic abnormalities or <5% monoclonal blasts by flow cytometry. (NCT01252667)
Timeframe: 1 Year post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 2 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 6 |
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Part 1: Number of Participants With Dose Limiting Toxicities (DLT)
"The primary objective of part 1 is to determined the highest dose of clofarabine that can be tolerated safely in conjunction with nonmyeloablative transplant. If three patients successfully transplant without DLT, dose escalation occurs. If one of those three patients experiences DLT an additional three patients will be treated using the same dose. If a second DLT is observed in the first 3-6 patients, or if three patients are successfully transplanted without DLT at the highest dose the study will proceed to Part 2 using the maximum tolerated dose.~A Clofarabine related dose-limiting toxicity is defined as a grade 4 toxicity involving the lungs, heart, liver (not resolving in 48 hours), kidneys (not resolving in 48 hours), gastrointestinal tract, and/or central nervous system." (NCT01252667)
Timeframe: 14 days post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
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Part 2: Number of Participants With Relapsed Disease
Number of high risk patients with relapsed disease after receiving the maximum dose of clofarabine. Relapse is defined as the presence of >5% aberrant blasts by morphology on a marrow aspirate or the presence of circulating blasts in the peripheral blood. (NCT01252667)
Timeframe: 6 months post-transplant
Intervention | Participants (Count of Participants) |
---|
Part 1 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 1 (30 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 2 (40 mg/m^2 Clofarabine) | 0 |
Part 2 - Dose 3 (50 mg/m^2 Clofarabine) | 3 |
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Pharmacokinetics (PK) of Clofarabine
Pharmacokinetic measurement of the volume of plasma from which clofarabine is completely removed per unit time. Clearance normalized to actual body weight. Pharmacokinetic analyses were performed on only a subset of patients and no aggregate calculations were made using the data. (NCT01252667)
Timeframe: Blood was drawn on day -6 before the first clofarabine dosing, at the end of the infusion, and at 3, 4, 5, 6 and 24 hours after the start of infusion. Only pre-dose samples were drawn on days -5, -4, and -3.
Intervention | L/h/kg (Number) |
---|
| ID 1 | ID 2 |
---|
Part 1 - Dose 2 (40 mg/m^2 Clofarabine) | 0.53 | 0.37 |
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Pharmacokinetics (PK) of Clofarabine
Pharmacokinetic measurement of the volume of plasma from which clofarabine is completely removed per unit time. Clearance normalized to actual body weight. Pharmacokinetic analyses were performed on only a subset of patients and no aggregate calculations were made using the data. (NCT01252667)
Timeframe: Blood was drawn on day -6 before the first clofarabine dosing, at the end of the infusion, and at 3, 4, 5, 6 and 24 hours after the start of infusion. Only pre-dose samples were drawn on days -5, -4, and -3.
Intervention | L/h/kg (Number) |
---|
| ID 4 | ID 5 | ID 6 |
---|
Part 1 - Dose 3 (50 mg/m^2 Clofarabine) | 0.29 | 0.40 | 0.50 |
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Estimated Glomerular Filtration Rate (eGFR)
Renal function was assessed by estimated Glomerular Filtration Rate (eGFR) using the Modification of Diet in Renal Disease (MDRD) formula. MDRD formula: GFR [mL/min/1.73m˄2] = 186.3*(C˄-1.154)*(A˄-0.203)*G*R. DEFINITIONS: C = serum concentration of creatinine [mg/dL]; A = age [years]; G = 0.742 when gender is female, otherwise G = 1; R = 1.21 when race is black, otherwise R = 1 (NCT01025817)
Timeframe: 12 Months
Intervention | mL/min/1.73m˄2 (Mean) |
---|
Everolimus and Low Dose Tacrolimus | 63.14 |
Mycophenolate Mofetil and Standard Dose Tacrolimus | 63.06 |
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Number of Participants With Incidence of Adverse Events, Serious Adverse Events, and Tacrolimus-associated Adverse Events
Incidence of adverse events, serious adverse events, and tacrolimus-associated adverse events by System Organ Class (NCT01025817)
Timeframe: 12 Months
Intervention | Participants (Number) |
---|
| Any system organ class | Metabolism and nutrition disorders | Gastrointestinal disorders | Injury, poisoning and procedural complications | General disorders &administration site conditions | Infections and infestations | Investigations | Renal and urinary disorders | Vascular disorders | Blood and lymphatic system disorders | Nervous system disorders | Respiratory, thoracic and mediastinal disorders | Musculoskeletal and connective tissue disorders | Skin and subcutaneous tissue disorders | Psychiatric disorders | Reproductive system and breast disorders | Cardiac disorders | Eye disorders | Immune system disorders | Endocrine disorders | Neoplasms benign,malignant,other incl cysts/polyps | Ear and labyrinth disorders | Hepatobiliary disorders | Surgical and medical procedures | Congenital, familial and genetic disorders | Social circumstances |
---|
Everolimus and Low Dose Tacrolimus | 303 | 266 | 233 | 223 | 199 | 184 | 150 | 141 | 131 | 130 | 125 | 122 | 110 | 109 | 96 | 56 | 51 | 26 | 13 | 12 | 10 | 7 | 6 | 2 | 0 | 0 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 302 | 263 | 247 | 202 | 177 | 196 | 143 | 160 | 121 | 163 | 150 | 134 | 114 | 108 | 106 | 40 | 47 | 36 | 11 | 8 | 15 | 11 | 3 | 0 | 6 | 1 |
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Number of Participants With Incidence of CMV (Viremia, Syndrome and Disease)
Participants with incidence of CMV (viremia, syndrome and disease). CMV is cytomegalovirus. (NCT01025817)
Timeframe: 12 Months
Intervention | Participants (Number) |
---|
| CMV syndrome event | Lab evidence of CMV Viremia | CMV Disease |
---|
Everolimus and Low Dose Tacrolimus | 9 | 7 | 2 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 13 | 10 | 8 |
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Number of Participants With Incidence of Composite Efficacy Failure
Efficacy failure rate used the composite endpoint of: (1) treated biopsy-proven acute rejection (BPAR)*, (2) graft loss**, (3) participant death or(4) loss to follow-up. *A treated BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III and which was treated with anti-rejection therapy. **Graft loss is defined as when the allograft was presumed lost on the day the participant started dialysis and was not able to subsequently be removed from dialysis. (NCT01025817)
Timeframe: 12 Months
Intervention | Participants (Number) |
---|
| Composite Endpoint | Treated Biopsy-proven Acute rejection (BPAR) | Graft Loss | Death | Loss to follow up |
---|
Everolimus and Low Dose Tacrolimus | 76 | 59 | 4 | 6 | 9 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 62 | 34 | 12 | 5 | 17 |
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Number of Participants With Incidence of New Onset of Diabetes Mellitus
Incidence of new onset diabetes mellitus defined as non-diabetic patients before transplantation, who are receiving glucose lowering treatment for more than 30 days post-transplant, or with a random plasma glucose ≥200 mg dL (11.1 mmol/L) with 2 fasting plasma glucose values ≥126 mg/dL (7 mmol/L) (NCT01025817)
Timeframe: 12 Months
Intervention | Participants (Number) |
---|
| Any New Onset Diabetes | randomGlucose≥200mg/dL w/2 fastingGlucose≥126mg/dL | Concomitant Diabetes medicine for 30 days or more |
---|
Everolimus and Low Dose Tacrolimus | 25 | 15 | 13 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 22 | 12 | 14 |
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Number of Participants With Incidence of Proteinuria Events
Number of participants with Incidence of proteinuria events indicating chronic kidney disease (NCT01025817)
Timeframe: Baseline and 12 Months
Intervention | Participants (Number) |
---|
| Baseline: Proteinuria (>=300 mg/g) | Month 12, Day 316-450: Proteinuria (>=300 mg/g) |
---|
Everolimus and Low Dose Tacrolimus | 243 | 36 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 250 | 35 |
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Number of Participants With Incidence Rates of BKV Viremia, BKV Viruria, or BKV Nephropathy
Participants with Incidence of BKV (viremia, viruria, or nephropathy). BKV is Polyomavirus type BK. (NCT01025817)
Timeframe: 12 Months
Intervention | Participants (Number) |
---|
| Lab evidence of BKV Viremia | Lab evidence of BKV Viruria | BKV Disease (Nephropathy) |
---|
Everolimus and Low Dose Tacrolimus | 19 | 19 | 5 |
,Mycophenolate Mofetil and Standard Dose Tacrolimus | 27 | 15 | 5 |
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To Compare 2-year Overall Survival of Patients With Lymphoma Undergoing RIC SCT Between Those Receiving Tacrolimus/Sirolimus/Methotrexate and Those Receiving Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil
(NCT00928018)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Sirolimus-Containing Regimen | 70 |
Sirolimus-Free Regimen | 68 |
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To Compare 2-year Progression-free Survival Between the Two Treatment Arms
(NCT00928018)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Sirolimus-Containing Regimen | 61 |
Sirolimus-Free Regimen | 58 |
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To Compare the 2-year Cumulative Incidence of Chronic GVHD Between the Two Treatment Arms.
(NCT00928018)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Sirolimus-Containing Regimen | 59 |
Sirolimus-Free Regimen | 63 |
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To Compare the 180-day Cumulative Incidence of Grades II-IV and Grades III-IV Acute GVHD Between the Two Treatment Arms
(NCT00928018)
Timeframe: 6 months
Intervention | percentage of participants (Number) |
---|
| Grade II-IV aGVHD | Grade III-IV aGVHD |
---|
Sirolimus-Containing Regimen | 9 | 3 |
,Sirolimus-Free Regimen | 25 | 4 |
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To Compare the 2-year Cumulative Incidences of Disease Progression and of Non-relapse Mortality Between the Two Treatment Arms
(NCT00928018)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
| Cumulative incidence of relapse/progression | Non-relapse mortality |
---|
Sirolimus-Containing Regimen | 26 | 14 |
,Sirolimus-Free Regimen | 30 | 12 |
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To Compare the 2-year of Overall Survival, Progression-free Survival, Cumulative Incidences of Progression and Non-relapse Mortality Between the Treatment Arms for Each Histology Studied.
(NCT00928018)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
| Overall Survival | Progression Free Survival | Cumulative Incidence of Progression | Non-relapse mortality |
---|
Aggressive Group: Sirolimus-Containing Regimen | 54 | 46 | 32 | 21 |
,Aggressive Group: Sirolimus-Free Regimen | 76 | 64 | 27 | 9 |
,Indolent Group: Sirolimus-Containing Regimen | 82 | 71 | 21 | 8 |
,Indolent Group: Sirolimus-Free Regimen | 63 | 53 | 33 | 15 |
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AUC0-inf
Bioequivalence based on AUC0-inf - Area under concentration-time curve from time zero to infinity (extrapolated) (NCT00910663)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*h/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 14.6569 |
CellCept® (Reference) | 14.2048 |
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AUC0-t
Bioequivalence based on AUC0-t - Area under concentration-time curve from time zero to time of last non-zero concentration (NCT00910663)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*h/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 13.8388 |
CellCept® (Reference) | 13.4016 |
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Cmax
Bioequivalence based on Cmax - Maximum Drug Concentration (NCT00910663)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg/mL (Mean) |
---|
Mycophenolate Mofetil (Test) | 2.8358 |
CellCept® (Reference) | 2.9768 |
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Number of Number of Participants Who Experience Grades III-IV GVHD, Day 360 (D60)
Number of participants who experience grade III or IV GVHD by Day 360. All participants are evaluable. (NCT02556931)
Timeframe: Day 360
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 18 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 5 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 7 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 10 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 14 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 16 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 10 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 14 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D60 | 42 |
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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
Intervention | Participants (Count of Participants) |
---|
PBSCT D90 | 33 |
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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
Intervention | months (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Rituximab and Allogeneic HCT Transplant) | 16 |
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Overall Survival
Estimated using Kaplan-Meier estimator. (NCT01044745)
Timeframe: From the date of transplant with death from any cause as a censored event, up to 2 years
Intervention | months (Median) |
---|
Treatment (Rituximab and Allogeneic HCT Transplant) | 12 |
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Achievement of >90% (Full) Donor Chimerism in the T-Cell Lineage as Measured by PCR at Day 30 Post-transplantation
Chimerism analysis of peripheral blood mononuclear cells using PCR (polymerase chain reaction) for STR/VNTR (short tandme repeat/variable number tandem repeat) will be performed post transplant. On each occasion, the peripheral blood will be separated into the T-cell component (using e.g. CD3 selection) and the myeloid component (using e.g.CD14/15 selection) before assessment of chimerism. (NCT00782379)
Timeframe: Day 30
Intervention | participants (Number) |
---|
Haploidentical Transplant | 18 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 18 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 13 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 7 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 16 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 0 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 2 |
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Non-relapse Mortality at Day 100 After Peripheral Blood Stem Cell Transplantation (PBSCT)
(NCT00782379)
Timeframe: Day 100
Intervention | participants (Number) |
---|
Haploidentical Transplant | 2 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 2 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 14 |
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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
Intervention | participants (Number) |
---|
Haploidentical Transplant | 17 |
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Average Level of Protenuria at Week 52
Proteinuria is measured as the ratio of albumin/creatinine mg/mmol. Measurements were taken from participants urine samples. (NCT01266148)
Timeframe: 52 weeks
Intervention | mg/mmol (Mean) |
---|
Everolimus | 7.2 |
Control | 1.2 |
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Measured Glomerular Filtration Rate (mGFR), 12 Months After Heart Transplantation
Measured Glomerular Filtration Rate (mGFR) describes the flow rate of filtered fluid through the kidney. GFR is equal to the clearance rate when any solute is freely filtered and is neither reabsorbed nor secreted by the kidneys. The rate therefore measured is the quantity of the substance in the urine that originated from a calculable volume of blood. Participants' urine was used for this assessment at week 52 after heart transplant. (NCT01266148)
Timeframe: Week 52
Intervention | mGFR ml/min (Mean) |
---|
Everolimus | 79.8 |
Control | 61.5 |
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Calculated Glomerular Filtration Rate From Pre-Transplantation to Week 52
Calculated Glomerular Filtration Rate from pre-transplantation to week 52 was calculated according to the Modification of Diet in Renal Disease (MDRD) method. Measurements were taken prior to transplant (day 1), between weeks 7 to 11 and end of week 52. (NCT01266148)
Timeframe: Day 1, weeks 7 to 11 and of week 52
Intervention | mGFR mL/min (Mean) |
---|
| Day 1 (n= 46, 48) | Weeks 7 to 11 (n=50, 51) | Week 52 (n= 45, 55) |
---|
Control | 66.1 | 69.6 | 69.3 |
,Everolimus | 65.4 | 84.9 | 104.5 |
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Change in Calculated Glomerular Filtration Rate From Pre-transplantation to Week 52
Change in calculated glomerular filtration rate from pre-transplantation to week 52 was calculated according to the Modification of Diet in Renal Disease (MDRD) method. Measurements were taken prior to transplant (day 1), between weeks 7 to 11 and end week 52. (NCT01266148)
Timeframe: Day 1, weeks 7 to 11(baseline) and of week 52
Intervention | mGFR mL/min (Mean) |
---|
| Day 1 (n= 43, 47) | Weeks 7 to 11 (n= 46, 49) | Week 52 (n= 42, 53) |
---|
Control | -12.6 | -6.8 | -8.0 |
,Everolimus | -13.0 | 7.4 | 27.8 |
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Change in Quality of Life - Euro Quality of Life 5D (EQ-5D)
Change in Quality of Life was assessed via the EQ-5D questionnaire which consists of: EQ-5D-5L descriptive system and EQ Visual Analogue scale (EQ VAS). The EQ-5D-5L comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression). Each dimension has 5 levels (no problems, slight problems, moderate problems, severe problems, and extreme problems). The patient indicates his/her health state by checking the most appropriate statement. This decision results in a 1-digit number expressing the level selected for that dimension. The digits for 5 dimensions are combined in a 5-digit number describing the respondent's health state. The possible score is 1 to 5 where a lower number indicates improvement. The EQ VAS records the patient's self-rated health on a 20 cm vertical, visual analogue scale with endpoints labelled 'the best health you can imagine' and 'the worst health you can imagine'. The score is 0 to 100 where a higher score represents improvement. (NCT01266148)
Timeframe: Pre transplant and 52 weeks
Intervention | Units on a scale (Mean) |
---|
| EQ-5D-5L pre- transplant (n=45,49) | EQ-5D-5L at week 52 (n=41,44) | EQ VAS pre- transplant (n=41,48) | EQ VAS at week 52 (n=41,42) |
---|
Control | 0.5069 | 0.8367 | 38.9 | 79.4 |
,Everolimus | 0.5750 | 0.8329 | 46.0 | 80.0 |
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Change in Quality of Life Assessed by SF-36 (Minnesota Living With Heart Failure Questionnaire ([MLHF)]) From Pre-transplant to Week 52 of Treatment
Change in Quality of Life was assessed via the SF-36 (Minnesota Living with Heart Failure questionnaire ([MLHF)]) before transplant surgery and at week 52 of treatment. The SF-36 is a validated, self-administered questionnaire. The questionnaire, which includes 36 questions measures 8 dimensions of health: physical function, role-physical, bodily pain, general health, vitality, social function, role-emotional, and mental health. Scores can be summarized in 2 summary components assessing physical and mental health. Items in each dimension are coded, aggregated, summed, and transformed into a scale ranging from 0 (worse health) to 100 (best health). (NCT01266148)
Timeframe: Pre transplant and 52 weeks
Intervention | Units on a scale (Mean) |
---|
| Physical Health pre- transplant | Physical Health at week 52 | Mental Health pre- transplant | Mental Health at week 52 |
---|
Control | 32.9 | 48.8 | 38.7 | 53.9 |
,Everolimus | 30.8 | 48.8 | 46.2 | 51.5 |
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Lipid Profile at 12 Months
Total Cholesterol, LDL-Chol, HDL-Chol and TG at week 52. Measurements were taken via participants blood samples. (NCT01266148)
Timeframe: 52 weeks
Intervention | mmol/L (Mean) |
---|
| Total cholesterol | LDL-C | HDL-C |
---|
Control | 5.1 | 2.8 | 1.6 |
,Everolimus | 5.3 | 2.9 | 1.6 |
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Number of Rejections Leading to Hemodynamic Compromise
Number of all rejections were recorded through the duration of the study with the intent to identify rejections leading to hemodynamic compromise. (NCT01266148)
Timeframe: 52 weeks
Intervention | rejections (Number) |
---|
| Total rejection | Treated rejection | Rejections with hemodynamic compromise |
---|
Control | 128 | 17 | 0 |
,Everolimus | 185 | 43 | 0 |
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Occurrence of Treatment Failures up to 12 Months After Transplant
Treatment failure was defined as the number of participants who died or lost their graft at any timepoint througout the duration of the study. (NCT01266148)
Timeframe: 52 weeks
Intervention | participants (Number) |
---|
| Treatment failure (death) | Graff loss | Total treatment failures (graft loss + death |
---|
Control | 3 | 0 | 3 |
,Everolimus | 2 | 0 | 2 |
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Progression of Chronic Allograft Vasculopathy (CAV) Based on Incidence of Chronic Allograft Vasculopathy (CAV) From Baseline to Week 52
the progression of chronic allograft vasculopathy (CAV) assessed by intravascular ultrasound (IVUS) examinations, measured the incidence of CAV (in percent of patients) at baseline and at week 52. Incidence of CAV represents percent of patients having a MIT (maximal intima thickness) > 0.5 mm. (NCT01266148)
Timeframe: Baseline and week 52
Intervention | Percentage of patients (Number) |
---|
| No CAV at baseline (week 7) | CAV at baseline (week 7) | No CAV at week 52 | CAV at week 52 |
---|
Control | 47.9 | 52.1 | 35.4 | 64.6 |
,Everolimus | 43.5 | 56.5 | 50 | 50 |
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Progression of Chronic Allograft Vasculopathy (CAV) Based on Maximal Intimal Thickness (MIT) From Baseline to Week 52
The progression of chronic allograft vasculopathy (CAV) was assessed by intravascular ultrasound (IVUS) examinations and measured Maximal Intimal Thickness (MIT)(in mm). A major coronary epicardial artery (preferentially the left-anterior descending coronary artery) was imaged, and the MIT parameters were recorded at baseline and at week 52. (NCT01266148)
Timeframe: Baseline and week 52
Intervention | mm (Mean) |
---|
| Baseline (Week 7) | Week 52 |
---|
Control | 0.56 | 0.65 |
,Everolimus | 0.52 | 0.55 |
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Overall Survival
Number of participants alive 1 year post transplant. (NCT03096782)
Timeframe: Up to 12 months after transplant
Intervention | Participants (Count of Participants) |
---|
Chemotherapy Plus Cord Blood Transplant | 4 |
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Disease-free Survival
Number of participants that were in remission post transplant. (NCT03096782)
Timeframe: Up to12 months
Intervention | Participants (Count of Participants) |
---|
| Complete Remission at 30 days | Complete Remission at 12 months |
---|
Chemotherapy Plus Cord Blood Transplant | 6 | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
| Twenty Two Days | Twenty Nine Days | Thirty Days | Nine Days | Thirty Two Days |
---|
Chemotherapy Plus Cord Blood Transplant | 2 | 1 | 1 | 1 | 1 |
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Percent Change in Mean Fluorescence Index (MFI) of Donor Specific Antibodies (DSA) With Increasing Doses of Enteric-Coated Mycophenolate Sodium (EC-MPS)
(NCT01044303)
Timeframe: 24 months
Intervention | percent of MFI change (Mean) |
---|
Mycophenolic Acid (MPA) Escalation | 43.1 |
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To Assess the Rate of Rejection, Infection and Renal Function as Mycophenolic Acid Dose is Increased.
(NCT01044303)
Timeframe: 24 months
Intervention | participants (Number) |
---|
Rate of Infection | 2 |
Rate of Rejection | 2 |
Renal Function | 29 |
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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
Intervention | Participants (Count of Participants) |
---|
Regimen A (PBSCT and BMT) | 78 |
Regimen B (UBCT) | 8 |
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Immune Reconstitution Following Hematopoietic Cell Transplantation
Number of patients with immune reconstitution (defined by a normal range CD3) at 1 year post transplant (NCT00919503)
Timeframe: 1 year following transplant
Intervention | Participants (Count of Participants) |
---|
Regimen A (PBSCT and BMT) | 39 |
Regimen B (UBCT) | 4 |
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Non-relapse Mortality
Number of patients who experienced non-relapse mortality by 1 year following transplant (NCT00919503)
Timeframe: 1 year following transplant
Intervention | Participants (Count of Participants) |
---|
Regimen A (PBSCT and BMT) | 2 |
Regimen B (UBCT) | 5 |
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Number of Participants With Infections
Number of participants with clinically significant infections (bacterial, fungal, viral) requiring treatment within 100 days following transplant (NCT00919503)
Timeframe: 100 days post transplant
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
| Greater than equal to 95% | 50 - 94% | 5-49% | Less than 5% |
---|
Regimen A (PBSCT and BMT) | 49 | 29 | 6 | 0 |
,Regimen B (UBCT) | 7 | 2 | 1 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
| Greater than equal to 95% | 50-94% | 5-49% | Less than 5% |
---|
Regimen A (PBSCT and BMT) | 72 | 8 | 4 | 0 |
,Regimen B (UBCT) | 7 | 2 | 2 | 1 |
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Graft Failure
Percentage of participants who failed to engraft. (NCT00946023)
Timeframe: Day 60
Intervention | Participants (Count of Participants) |
---|
Transplant | 2 |
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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
Intervention | percentage of participants (Number) |
---|
Transplant | 11 |
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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
Intervention | percentage of participants (Number) |
---|
Transplant | 41 |
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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
Intervention | percentage of participants (Number) |
---|
Transplant | 5 |
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Non-relapse Mortality
Percentage of participants who died due to BMT-related reasons. (NCT00946023)
Timeframe: 1 year post intervention
Intervention | percentage of participants (Number) |
---|
Transplant | 8 |
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Engraftment
Percentage of patients who engrafted neutrophils and platelets. (NCT00946023)
Timeframe: Day 60
Intervention | percentage of participants (Number) |
---|
| Neutrophil engraftment | Platelet engraftment |
---|
Transplant | 98 | 98 |
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Overall Survival
Percentage of participants alive. (NCT00946023)
Timeframe: 1 year post intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 86 | 83 | 94 | 86 | 78 |
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Overall Survival
Percentage of participants alive. (NCT00946023)
Timeframe: 2 years post intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 76 | 73 | 87 | 76 | 69 |
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Progression-free Survival
Percentage of participants alive and without relapse or disease progression. (NCT00946023)
Timeframe: 1 year post-intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 71 | 70 | 82 | 70 | 65 |
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Progression-free Survival
Percentage of participants alive with and without relapse. (NCT00946023)
Timeframe: 2 years post-intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 60 | 63 | 68 | 59 | 56 |
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Relapse
Percentage of participants alive with relapse or disease progression. (NCT00946023)
Timeframe: 1 year post intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 20 | 20 | 18 | 23 | 17 |
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Relapse
Percentage of participants alive with relapse or disease progression. (NCT00946023)
Timeframe: 2 years post intervention
Intervention | percentage of participants (Number) |
---|
| All participants | Haploidentical recipients | VV donor | VF donor | FF donor |
---|
Transplant | 27 | 23 | 25 | 31 | 22 |
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Change From Baseline in the Euro Quality of Life 5D
"Change from baseline in Euro Quality of Life-5D from 3 Year Follow-Up to 5 to 7 Year Follow-Up Baseline Visit 1 (ITT Set)~Euro Quality of Life 5D (EQ-5D): is a descriptive system of healthrelated quality of life states consisting of five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression) each of which can be assessed as one of three levels of severity (no problems/some or moderate problems/extreme problems). A Visual Analogue Scale (VAS)-scale is also included in the EQ-5D questionnaire.~The EQ-5D index is calculated based on the United Kingdom Time Trade-Off (TTO) N3 value set which converts the five dimensions scores into a single measure with a possible range from -0.163 (worst possible health state) to +1 (perfect health). A positive change from baseline indicates an improvement in Quality of Life." (NCT02864706)
Timeframe: Baseline, 5-7 year visit
Intervention | scores on the scale (Mean) |
---|
Everolimus | 0.2323 |
Control | 0.2982 |
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Change From Baseline in Visual Analog Scale (VAS)
"Change in visual analog scale (VAS) from baseline to the 5 to 7 Year follow up visit.~0 is no pain; and 10 is the worst possible pain" (NCT02864706)
Timeframe: baseline, at the 5-7 year visit
Intervention | mm (Mean) |
---|
Everolimus | 35.6 |
Control | 34.0 |
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Measured Glomerular Filtration Rate (mGFR)
Renal function as assessed by measured Glomerular Filtration Rate (mGFR) (Cr-EDTA or iohexol clearance). Baseline Visit 1 and Patient 4252 excluded from the intent treat analysis set. (NCT02864706)
Timeframe: at the 5-7 year follow-up visit
Intervention | mL/min/1.73m2 (Least Squares Mean) |
---|
Everolimus | 74.7 |
Control | 62.4 |
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Percent of Participants With Incidence of Coronary Allograft Vasculopathy (CAV)
"Cardiac Allograft Vasculopathy (CAV) was defined as mean maximal intimal thickness (MIT)~≥0.5 mm, measured for the entire matched pullback recording by intravascular ultrasound (IVUS). The incidence of CAV at 5-7 years was compared between groups using the Cochran-Mantel-Haenszel test with stratification according to baseline distribution of CAV incidence." (NCT02864706)
Timeframe: at the 5-7 year follow-up
Intervention | percent of participants (Number) |
---|
Everolimus | 53 |
Control | 74 |
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Progression of Cardiac Allograft Vasculopathy (CAV) Recorded by Intravascular Ultrasound (IVUS)
"Cardiac Allograft Vasculopathy (CAV) was defined as mean maximal intimal thickness (MIT)~≥0.5 mm, measured for the entire matched pullback recording by intravascular ultrasound (IVUS). The incidence of CAV at 5-7 years was compared between groups using the Cochran-Mantel-Haenszel test with stratification according to baseline distribution of CAV incidence." (NCT02864706)
Timeframe: within 5-7 years
Intervention | mm (Mean) |
---|
Everolimus | 0.13 |
Control | 0.23 |
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Myocardial Structure and Function
Myocardial structure and function by echocardiography assessment measured by ventricular end systolic diameter. (NCT02864706)
Timeframe: within 5-7 years
Intervention | cm (Mean) |
---|
| LVESD (left ventricular end systolic diameter) | LVEDD (left ventricular end diastolic diameter) |
---|
Control | 3.1 | 4.9 |
,Everolimus | 3.1 | 4.7 |
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Number of Participants With Beck Depression Inventory (BDI)
Beck Depression Inventory (BDI) Score has the following categories of depression. Normal, Mild, Moderate Severe and Missing. (NCT02864706)
Timeframe: at the 5-7 year visit
Intervention | Participants (Count of Participants) |
---|
| Normal | Mild | Moderate | Severe | Missing |
---|
Control | 13 | 4 | 5 | 0 | 5 |
,Everolimus | 15 | 6 | 2 | 1 | 3 |
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Quality of Life by SF-36 Change From Pre-transplantation to 5-7 Year Follow-up
This Quality of life Short Form Survey with 36 items (Minnesota Living with Heart Failure Questionnaire)was administered to patients pre-transplantation and after transplantation at the 5-7 year visit. This data represents the change. The survey consist of scores on a scale. Each form is scaled from 0 t 100. 0 = maximum disability and 100 equals no disability. (NCT02864706)
Timeframe: at the 5-7 year visit
Intervention | scores on a scale (Mean) |
---|
| Physical Health Summary | Mental Health Summary | Physical Functioning | Role Physical | Bodily Pain | General Health | Vitality | Social Functioning | Role Emotional | Mental Health |
---|
Control | 13.2 | 15.3 | 40.8 | 55.1 | 7.2 | 23.4 | 28.9 | 43.9 | 42.8 | 14.4 |
,Everolimus | 16.8 | 10.4 | 36.7 | 50.2 | 10.3 | 25.7 | 30.0 | 39.9 | 23.8 | 8.6 |
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Disease-free Survival at Five Years Post-transplant
Kaplan-Meier estimate of an event of relapse or death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of transplant to five years post-transplant
Intervention | percentage of survival probability (Number) |
---|
Busulfan, FTBI and VP16 | 40 |
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Overall Survival at 5 Years Post-Transplant.
Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant
Intervention | percentage of survival probability (Number) |
---|
Busulfan, FTBI and VP16 | 40 |
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Overall Survival Comparing Diagnosis Groups
Kaplan-Meier estimate of an event of death estimated at five years post-transplant. 95% confidence intervals were calculated from the logit transform of the Greenwood variance. The transformation was necessary to keep the estimate within the probability space of 0 to 100%. (NCT00534430)
Timeframe: Date of Transplant to Five Years post-Transplant
Intervention | percentage of survival probability (Number) |
---|
AML/1CR | 67 |
AML/R1 | 50 |
AML/IF | 30 |
Active ALL | 33 |
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Immune Reconstruction/CD4+ Count at 1 Year
(NCT00425802)
Timeframe: 1 year
Intervention | cells/microliter (Median) |
---|
Treatment | 333 |
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Immune Reconstruction/CD4+ Count at 3 Months
(NCT00425802)
Timeframe: 3 months
Intervention | cells/microliters (Median) |
---|
Treatment | 253 |
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Immune Reconstruction/CD4+ Count at 6 Months
(NCT00425802)
Timeframe: 6 months
Intervention | cells/microliter (Median) |
---|
Treatment | 312 |
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Incidence of Chronic GVHD at 1 Year
(NCT00425802)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Treatment | 14 |
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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
Intervention | percentage of patients (Number) |
---|
Treatment | 18 |
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Overall Survival at 1 Year
(NCT00425802)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Treatment | 90 |
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Time to Neutrophil Engraftment
(NCT00425802)
Timeframe: 2 years
Intervention | days (Median) |
---|
Treatment | 15 |
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Time to Platelet Engraftment
(NCT00425802)
Timeframe: 1 year
Intervention | days (Median) |
---|
Treatment | 12 |
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Event-Specific Survival Comparisons
Freedom from biopsy-proven acute rejection of the kidney allograft; Freedom from biopsy-proven acute rejection of the pancreas allograft; Death-censored kidney graft survival; Death-censored pancreas graft survival; Death-uncensored graft (kidney and pancreas) survival; and Patient survival. (NCT00533442)
Timeframe: over 1-10 years post-transplant
Intervention | Participants (Count of Participants) |
---|
| Biopsy-Proven Acute Rejection of the Kidney | Biopsy-Proven Acute Rejection of the Pancreas | Death-Censored Kidney Graft Failure | Death-Censored Pancreas Graft Failure | Death-Uncensored Graft (Kidney&Pancreas) Survival | Patient Death |
---|
Tacrolimus Plus MMF Plus Steroids | 22 | 9 | 18 | 7 | 26 | 16 |
,Tacrolimus Plus Rapamycin Plus Steroids | 8 | 1 | 10 | 2 | 25 | 15 |
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Overall Kidney Transplant Function at 12, 36, and 60 Months Post-transplant.
Comparisons of renal function (eGFR, measured in mL/min/1.73 m^2) at 12, 36, and 60 months post-transplant. (NCT00533442)
Timeframe: at 1-5 years post-transplant
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Mean eGFR at 12 months | Mean eGFR at 36 months | Mean eGFR at 60 months |
---|
Tacrolimus Plus MMF Plus Steroids | 67.2 | 61.5 | 61.9 |
,Tacrolimus Plus Rapamycin Plus Steroids | 71.2 | 63.0 | 56.5 |
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Overall Pancreas Transplant Function at 12, 36, and 60 Months Post-transplant.
Comparisons of pancreas function (C-peptide in ng/mL) at 12, 36, and 60 months post-transplant. (NCT00533442)
Timeframe: at 1-5 years post-transplant
Intervention | ng/mL (Mean) |
---|
| Mean Log {C-peptide at 12 months} | Mean Log {C-peptide at 36 months} | Mean Log {C-peptide at 60 months} |
---|
Tacrolimus Plus MMF Plus Steroids | 1.15 | 0.990 | 1.17 |
,Tacrolimus Plus Rapamycin Plus Steroids | 1.08 | 0.986 | 1.22 |
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Clinical Significant Infection, Requiring Treatment, Within 100 Days Post Transplant
Number of patients who experienced a clinical significant infection, requiring treatment, within 100 days post transplant. (NCT00553098)
Timeframe: 100 days
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 21 |
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Disease Response by 1 Year Post Transplant
Number of patients at 1 year with disease response (defined as no clinical evidence of active disease and/or sufficient level of donor chimerisms to prevent disease recurrence) (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 15 |
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Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant
Number of Patients Who Achieve Greater Than 50% CD19+ Donor Chimerisms at 1 Year Post Transplant (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 16 |
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Greater Than 50% CD33+ Donor Chimerisms at 1 Year Post Transplant
Number of patients who achieve greater than 50% CD33+ donor chimerisms at 1 year post transplant. (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 8 |
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Immune Reconstitution by 1 Year Post Transplant
Number of patients with normal range CD3 at 1 year post transplant (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 7 |
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Number of Patients Diagnosed With Acute GVHD
Number of patients diagnosed with acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 18 |
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Number of Patients Diagnosed With Chronic GVHD
Number of patients diagnosed with chronic GVHD within 1 year post transplant (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 8 |
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Number of Patients Diagnosed With Overall Grade 1 or Grade 2 Acute GVHD
Number of patients diagnosed with overall grade I or grade II acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 12 |
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Number of Patients Diagnosed With Overall Grade III or Grade IV Acute GVHD
Number of patients diagnosed with overall Grade III or Grade IV Acute GVHD by Day 100 post transplant (NCT00553098)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 6 |
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Number of Patients Who Achieve Greater Than 50% Donor T-cell Chimerism
The study will be considered a success and the protocol worthy of further study if there is sufficient evidence that this rate is greater than the 50% rate observed in the most recently transplanted patients with nonmalignant disorders. Analyses will be carried out separately for the alemtuzumab recipients and the TBI recipients. We will be 80% confidence of success if a one-sided 80% confidence interval for the proportion of patients with successful chimerism exceeds 50%. Cumulative incidence will be used to evaluate the probability of chimerism. (NCT00553098)
Timeframe: At 1 year post transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 13 |
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Overall Survival
Number of patients alive at 1 year (NCT00553098)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy, Low Dose Radiation) | 19 |
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Graft Failure
Percentage of participants who failed to engraft. (NCT01135329)
Timeframe: Day 60
Intervention | Participants (Count of Participants) |
---|
Transplant | 8 |
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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
Intervention | Participants (Count of Participants) |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 21 |
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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
Intervention | Participants (Count of Participants) |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 14 |
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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
Intervention | Participants (Count of Participants) |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 10 |
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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
Intervention | Participants (Count of Participants) |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 14 |
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Number of Patients With Toxicities Related to Bortezomib Maintenance Therapy
Number of subjects with toxicities related to bortezomib maintenance therapy post-transplant. (NCT00793572)
Timeframe: Up to 100 days after the autograft or allograft
Intervention | Participants (Count of Participants) |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
| 200 days post allo | 1 Year post allo |
---|
Tandem Auto-/Nonmyeloablative Allo-HCT and Maintenance Therapy | 1 | 1 |
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Event-Free Survival (EFS)
Number of patients surviving without relapsed/progressive disease (NCT01008462)
Timeframe: 1 Year post-autograft
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
| 200 days | 1 Year |
---|
Treatment (Autologous HCT, Donor HCT) | 0 | 1 |
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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,
Intervention | Participants (Count of Participants) |
---|
| Acute Graft-versus-Host-Disease | Chronic extensive Graft-versus-Host-Disease |
---|
Treatment (Autologous HCT, Donor HCT) | 8 | 1 |
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Donor Cell Chimerism Following Transplant
Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: 6 months
Intervention | percentage of donor cells (Mean) |
---|
Transplant Patients | 94 |
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Donor Cell Chimerism Following Transplant
Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 100
Intervention | percentage of donor cells (Mean) |
---|
Transplant Patients | 90 |
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Donor Cell Chimerism Following Transplant
Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 28
Intervention | percentage of donor cells (Mean) |
---|
Transplant Patients | 95 |
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Donor Cell Chimerism Following Transplant
Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: Day 42
Intervention | percentage of donor cells (Mean) |
---|
Transplant Patients | 93 |
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Donor Cell Chimerism Following Transplant
Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin. (NCT01043640)
Timeframe: One year
Intervention | percentage of donor cells (Mean) |
---|
Transplant Patients | 99 |
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Number of Patients Who Died Peri-Transplant
Peri-transplant is defined as within 100 days of transplant. (NCT01043640)
Timeframe: By Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 2 |
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Number of Patients With Donor Derived Engraftment
Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells. (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 42 |
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Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 32 |
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Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 2 |
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Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)
"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 5 |
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Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)
"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 2 |
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Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)
"GVHD grading is performed using modified Glucksberg criteria and is as follows:~grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4" (NCT01043640)
Timeframe: Day 100 Post Transplant
Intervention | Participants (Count of Participants) |
---|
Transplant Patients | 5 |
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Difference in Renal Function
"Difference in renal function between groups at listed time points assessed by mean serum creatinine. Increased serum creatinine could indicate worsening renal function. A normal serum creatinine range for the transplant population varies by patient, but a typical range for Scr would be 1-2 mg/dL." (NCT01336296)
Timeframe: Difference at 1 month, 3 months, 6 months, 1 year
Intervention | mg/L (Mean) |
---|
| 1 month | 3 months | 6 months | 1 year |
---|
Myfortic Preload | 1.41 | 1.45 | 1.43 | 1.36 |
,Myfortic Standard | 1.43 | 1.39 | 1.40 | 1.5 |
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Incidence of Biopsy-confirmed Acute Rejection by Banff '97 Criteria (Updated 2007) 3, 6 and 12 Months Post Transplant
"Incidence of biopsy-confirmed acute rejection by Banff '97 Criteria (updated 2007) post transplant. The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~As with humoral rejection, there are both acute & chronic forms:~The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~Class IA: there is at least 25% of parenchymal showing interstitial infiltration and foci of moderate tubulitis (defined as a certain number of immune cells present in tubular cross-sections).~Class IB: just like Class IA except there is more severe tubulitis.~Class IIA: there is mild-to-moderate intimal arteritis.~Class IIB: there is severe intimal arteritis comprising at least 25% of the lumenal area.~Class III: there is transmural (e.g. the full vessel wall thickness) arteritis." (NCT01336296)
Timeframe: 3, 6 and 12 months post transplant
Intervention | participants (Number) |
---|
| 3 months | 6 months | 12 months |
---|
Myfortic Preload | 7 | 7 | 8 |
,Myfortic Standard | 3 | 3 | 4 |
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Incidence of Chronic Alloantibody Rejection or Chronic Allograft Arteriopathy by Banff '97
The Banff features suggestive of chronic rejection were: a) chronic transplant glomerulopathy: Glomerular basement membrane duplication and mesangial cell proliferation, and b) vasculopathy: Fibrous intimal thickening often with fragmentation of internal elastic lamina. Chronic changes in the interstitium (ci), tubules (ct), vessels (cv), and glomerulus (cg) were likewise graded into 0, 1, 2, and 3. The severity of interstitial fibrosis and tubular atrophy, as also chronic transplant glomerulopathy and vasculopathy were used to grade chronic allograft changes. (NCT01336296)
Timeframe: 1 year
Intervention | participants (Number) |
---|
| mild Chronic allograft nephropathy (CAN) | Moderate Chronic allograft nephropathy (CAN) |
---|
Myfortic Preload | 9 | 0 |
,Myfortic Standard | 3 | 1 |
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Number of Patients Requiring Anti-lymphocyte Therapy for Acute Rejection
(NCT01336296)
Timeframe: 1 year
Intervention | participants (Number) |
---|
| Pulse methylprednisolone | Thymoglobulin | Plasmapheresis | Intravenous immunoglobulin (IVIg) | Rituximab |
---|
Myfortic Preload | 5 | 6 | 0 | 0 | 0 |
,Myfortic Standard | 3 | 0 | 2 | 0 | 1 |
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Severity of Acute Rejection by Banff '97 Criteria
"Severity of biopsy-confirmed acute rejection by Banff '97 Criteria (updated 2007) at 1 year. The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~As with humoral rejection, there are both acute & chronic forms:~The acute form of T-cell mediated rejection is furthermore subclassified as follows. Since this is the most common form of rejection, it is useful to know:~Class IA: there is at least 25% of parenchymal showing interstitial infiltration and foci of moderate tubulitis (defined as a certain number of immune cells present in tubular cross-sections).~Class IB: just like Class IA except there is more severe tubulitis.~Class IIA: there is mild-to-moderate intimal arteritis.~Class IIB: there is severe intimal arteritis comprising at least 25% of the lumenal area.~Class III: there is transmural (e.g. the full vessel wall thickness) arteritis." (NCT01336296)
Timeframe: Severity 1 year post transplant
Intervention | participants (Number) |
---|
| Grade IA | Grade IB | Grade IIA | Grade IIB | Antibody Mediated Rejection | Mixed Acute Rejection |
---|
Myfortic Preload | 3 | 4 | 1 | 0 | 0 | 0 |
,Myfortic Standard | 1 | 1 | 0 | 0 | 1 | 1 |
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Acute GvHD
overall grade II-IV acute GvHD (NCT00053989)
Timeframe: Day +100
Intervention | Participants (Count of Participants) |
---|
All Patients | 16 |
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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
Intervention | Participants (Count of Participants) |
---|
All Patients | 4 |
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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
Intervention | percentage of participants (Number) |
---|
All Patients | 44 |
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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
Intervention | percentage of participants (Number) |
---|
All Patients | 27 |
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Percentage of Participants Discontinuing Immunosuppressants (MMF) for More Than 14 Consecutive Days or 30 Cumulative Days Within 24 Weeks of Transplantation
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
MMF + CsA | 13.89 |
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Percentage of Participants Lost To Follow Up Within 24 Weeks of Transplantation
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
MMF + CsA | 0.0 |
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Percentage of Participants Requiring Use of Additional Immunosuppressants Not Specified in the Protocol Within 24 Weeks of Transplantation
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
MMF + CsA | 0.0 |
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Percentage of Participants With Graft Loss Within 24 Weeks of Transplantation
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
MMF + CsA | 0.0 |
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Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and During Treatment
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
| Serum creatinine | BUN |
---|
MMF + CsA | 13.89 | 72.22 |
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Percentage of Participants With Abnormal Serum Creatinine and BUN Values at Baseline and Normal Values During Treatment
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
| Serum creatinine | BUN |
---|
MMF + CsA | 16.67 | 2.78 |
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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR) by Week
Percentage of participants with BPAR of greater than or equal to (≥) International Society of Heart and Lung Transplant (ISHLT) Grade III. The ISHLT graded symptoms on a scale of Grade 0 through VI. Grade 0 equals (=) no rejection. Grade IA = regional (perivascular or interstitial) infiltration and no necrosis, and grade IB = dissemination but little infiltration and no necrosis. Grade II = 1 focus of invasive infiltration with or without (+/-) associated cardiomyocyte necrosis. Grade IIIA = 2 or more foci of invasive infiltration +/- associated cardiomyocyte necrosis, and grade IIIB = diffuse inflammatory pathological changes associated with cardiomyocyte necrosis. Grade IV = diffuse, infiltrative multi-foci +/- edema; +/- hemorrhage; and +/-vasculitis. (NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
| Day 1 | Week 1 | Week 2 | Week 4 | Week 8 | Week 12 | Week 16 | Week 20 | Week 24 |
---|
MMF + CsA | 0.0 | 0.0 | 2.78 | 2.78 | 2.78 | 2.78 | 2.78 | 2.78 | 2.78 |
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Percentage of Participants With Normal Serum Creatinine and Blood Urea Nitrogen (BUN) Values At Baseline (BL) And During Treatment
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
| Serum creatinine | BUN |
---|
MMF + CsA | 47.22 | 8.33 |
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Percentage of Participants With Normal Serum Creatinine and BUN Values at Baseline and Abnormal Values During Treatment
(NCT02091414)
Timeframe: Day 1, Weeks 1, 2, 4, 8, 12, 16, 20 and 24
Intervention | percentage of participants (Number) |
---|
| Serum creatinine | BUN |
---|
MMF + CsA | 22.22 | 16.67 |
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Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
AEs with a start date on or after the first dose date in study 211LE202. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the subject at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the subject or may require intervention to prevent one of the other outcomes listed above. (NCT01930890)
Timeframe: Up to Week 108
Intervention | participants (Number) |
---|
| Any event | Moderate or severe event | Severe event | Event related to dose-blinded treatment | Event related to MMF | Serious event | Serious event related to dose-blinded treatment | Serious event related to MMF | Fatal event |
---|
BIIB023 20 mg/kg (211LE201) to BIIB023 20 mg/kg (211LE202) | 19 | 6 | 3 | 4 | 9 | 3 | 1 | 2 | 1 |
,BIIB023 3 mg/kg (211LE201) to BIIB023 3 mg/kg (211LE202) | 23 | 12 | 4 | 5 | 8 | 7 | 2 | 3 | 0 |
,Placebo (211LE201) to BIIB023 20 mg/kg (211LE202) | 7 | 3 | 1 | 1 | 2 | 4 | 1 | 1 | 0 |
,Placebo (211LE201) to BIIB023 3 mg/kg (211LE202) | 4 | 2 | 0 | 1 | 2 | 1 | 0 | 0 | 0 |
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Number of Participants Who Discontinued Study Treatment or Withdrew From Study Due to an AE
AEs with a start date on or after the first dose date in study 211LE202. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the subject at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the subject or may require intervention to prevent one of the other outcomes listed above. (NCT01930890)
Timeframe: Up to Week 108
Intervention | participants (Number) |
---|
| Discontinued treatment due to an AE | Withdrew from study due to an AE |
---|
BIIB023 20 mg/kg (211LE201) to BIIB023 20 mg/kg (211LE202) | 0 | 1 |
,BIIB023 3 mg/kg (211LE201) to BIIB023 3 mg/kg (211LE202) | 0 | 2 |
,Placebo (211LE201) to BIIB023 20 mg/kg (211LE202) | 0 | 0 |
,Placebo (211LE201) to BIIB023 3 mg/kg (211LE202) | 0 | 0 |
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Full Donor Chimerism (FDC)
Proportion of patients achieving full donor T-cell chimerism (FDC) by on or before Day 90 post non-myeloablative allogeneic transplant with allogeneic cytokine-induced killer (CIK) cells will be determined. FDC is defined as the attainment of >95% donor type CD3+ cells. The outcome will be reported as number of participants who achieved full donor chimerism, a number without dispersion. (NCT01392989)
Timeframe: 90 days
Intervention | Participants (Count of Participants) |
---|
Allogeneic Cytokine Induced Killer Cells (CIK) | 6 |
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Overall Survival (OS)
Overall survival (OS) is an expression of the number of participants that remain alive 2 years after cytokine-induced killer (CIK) infusion. The outcome will be reported as the number of participants alive 2 years after CIK infusion, a number without dispersion. (NCT01392989)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Cytokine Induced Killer Cells (CIK) | 16 |
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Event-free Survival (EFS) Rate
Event-free Survival (EFS) rate will be assessed on all enrolled participants and is defined as the duration of time after cytokine-induced killer (CIK) cell infusion that the participants remain alive with experiencing relapse, Grade 3 to 4 acute graft vs host disease (aGVHD), or death. The outcome will be reported as the number of participants, stratified by receipt of CIK cells, that did not experience a specified event, a number without dispersion. (NCT01392989)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
| CIK-infused participants | Non-CIK-infused participants |
---|
Allogeneic Cytokine Induced Killer Cells (CIK) | 14 | 4 |
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Number of Participants That Experience Grade 2 to 4 aGvHD Within 100 Days and 1 Year
"Acute graft vs host disease (aGvHD) Grade 2 to 4 was staged & graded using modified Keystone criteria, as below. The outcome is reported as the number of participants that experience Grade 2 to 4 aGvHD within 100 days and 1 year.~Stage 1: Skin: rash < 25% of skin. Liver: bilirubin 2 to 3 mg/dL. Gut: diarrhea 500 to 1000 mL/day or persistent nausea with positive biopsy for GvHD~Stage 2: Skin: rash 25 to 50% of skin. Liver: bilirubin 3 to 6 mg/dL. Gut: diarrhea 1000 to 1500 mL/day.~Stage 3: Skin: rash > 50% of skin. Liver: bilirubin 6 to 15 mg/dL. Gut: diarrhea > 1500 mL/day.~Stage 4: Skin: generalized erythroderma with bulla formation. Liver: bilirubin > 15 mg/dL. Gut: severe abdominal pain with or without ileus Grade of aGvHD was determined as follows.~Grade 1: Stage 1-2 Skin + No Liver stage + No Gut stage~Grade 2: Stage 3 Skin OR Stage 1 Liver or Stage 1 Gut~Grade 3: No Skin stage + Stage 2 to 3 Liver Stage 2 to 4 Gut~Grade 4: Stage 4 Skin + or Stage 2" (NCT01392989)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
| All participants within 100 days | All participants within 1 year | CIK-infused participants within 100 days | CIK-infused participants within 1 year | Non-CIK-infused participants within 100 days | Non-CIK-infused participants within 1 year |
---|
Allogeneic Cytokine Induced Killer Cells (CIK) | 9 | 11 | 3 | 5 | 6 | 6 |
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Drug Discontinuation Due to Side Effects
Drug discontinuation due to drug side effects regarding Cellcept and Myfortic. (NCT00336817)
Timeframe: 12 weeks
Intervention | Participants (Count of Participants) |
---|
Myfortic Group | 1 |
CellCept Group | 2 |
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Incidence of Biopsy-proven Acute Cellular Rejection During the Study Period
number of patients with ACR (NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 2 |
CellCept Group | 1 |
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Incidence of Cytomegalovirus Infection or Disease During the Study Period
number of participants (NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 0 |
CellCept Group | 0 |
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Incidence of Graft Loss or Death During the Study Period
number of patients (NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 0 |
CellCept Group | 0 |
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Number of Participants With Bone Marrow Suppression
Number of participants with: Thrombocytopenia (<50,000 mm3), Leukopenia (< 2000 mm3), absolute neutrophils count ( <1000 mm3) or hemoglobin ( < 7.0 g/dL) (NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 0 |
CellCept Group | 0 |
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Number of Participants With Clinically Significant Decrease in Serum Creatinine From Baseline Through Week 12
Creatinine levels (NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 0 |
CellCept Group | 0 |
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Number of Participants With Neurotoxicity
(NCT00336817)
Timeframe: 12 weeks
Intervention | participants (Number) |
---|
Myfortic Group | 0 |
CellCept Group | 0 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms. The total GSRS range of scores is 15 to 105 with higher scores meaning the worst of symptoms." (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 23.07 | 22.14 | 26.07 | 25.21 |
,Myfortic Group | 21.80 | 24.90 | 28.90 | 21.80 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Constipation Subscale)
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~Constipation subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 4.07 | 4.21 | 4.79 | 4.54 |
,Myfortic Group | 4.50 | 4.60 | 6.50 | 4.50 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Diarrhea Subscale)
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The Diarrhea subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 4.79 | 4.36 | 4.50 | 5.29 |
,Myfortic Group | 4.00 | 5.80 | 6.60 | 3.70 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil ( Indigestion Subscale)
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The Indigestion subscale range is 4 to 28 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 5.86 | 6.43 | 7.50 | 6.86 |
,Myfortic Group | 5.40 | 5.80 | 6.50 | 5.60 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Abdominal Pain Subscale)
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~The abdominal Pain subscale range is 3 to 21 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 5.29 | 4.79 | 5.93 | 5.36 |
,Myfortic Group | 5.20 | 5.80 | 6.40 | 5.30 |
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GI Side Effects as Assessed by Gastro Intestinal Symptoms Rating Scale (GSRS) of Enteric-coated Mycophenolate Sodium vs Mycophenolate Mofetil (Reflux Subscale)
"The GSRS contains 15 items, each rated on a seven-point Likert scale from no discomfort to very severe discomfort. Based on a factor analysis, the 15 GSRS items break down into the following five scale: abdominal pain syndrome ( abdominal pain, hunger pains, and nausea); reflux syndrome (heartburn and acid regurgitation), diarrhea syndrome (diarrhea, loose stools and urgent need for defecation), indigestion syndrome (borborygmus, abdominal distension, eructation and increased flatus) and constipation syndrome (constipation, hard stools, and feeling of incomplete evacuation).~The GSRS is a disease-specific instrument of 15 items combined into five symptom clusters depicting Reflux, Abdominal pain, Indigestion, Diarrhea, and Constipation. The GSRS has a seven-point graded Likert-type scale where 1 represents absence of troublesome symptoms and 7 represents very troublesome symptoms.~Reflux subscale range is 2 to 14 with higher scores means worst symptoms" (NCT00336817)
Timeframe: screening, 2, 6 and 12 weeks
Intervention | units on a scale (Mean) |
---|
| screening | 2 weeks | 6 weeks | 12 weeks |
---|
CellCept Group | 3.07 | 2.36 | 3.36 | 3.07 |
,Myfortic Group | 2.70 | 2.90 | 2.90 | 2.70 |
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Mean Change From Baseline in Forced Vital Capacity (FVC)
compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration. (NCT00333437)
Timeframe: Baseline, 12 months
Intervention | Liters (Mean) |
---|
Treatment | 0.1786 |
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Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)
DLCO was measured before beginning and after completion of study therapy (NCT00333437)
Timeframe: 12 months
Intervention | Liters (Mean) |
---|
Treatment | 1.86 |
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Mean Change in Six Minute Walk Distance
Comparison of 6-minute walk distance before beginning and after completing study therapy (NCT00333437)
Timeframe: 12 months
Intervention | Feet (Mean) |
---|
Treatment | 264.3 |
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Change in Shortness of Breath (Self-reported)
Participants reported frequency of shortness of breath experienced with exertion (NCT00333437)
Timeframe: Baseline, 12 months
Intervention | participants (Number) |
---|
| Patient-reported less shortness of breath | Patient-reported no change in shortness of breath | Patient-reported increased shortness of breath |
---|
Treatment | 6 | 1 | 0 |
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Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils)
BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy. (NCT00333437)
Timeframe: Baseline, 12 months
Intervention | Cells/uL (Mean) |
---|
| Mean change in neutrophil count | Mean change in eosinophil count |
---|
Treatment | -3 | -6.3 |
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Number of Patients Who Engrafted
Continued engraftment will be defined as the detection of donor T-cells (CD3+) as a proportion of the total T-cell of greater than 5%. (NCT00397813)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Arm A - Dose Level 1 | 35 |
Arm A - Dose Level 2 | 0 |
Arm A - Dose Level 3 | 0 |
Arm B - Dose Level 1 | 12 |
Arm B - Dose Level 2 | 4 |
Arm B - Dose Level 3 | 24 |
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Number of Patients Who Had Infections
Number of patients who experienced bacterial, fungal, or viral infections. (NCT00397813)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Arm A - Dose Level 1 | 24 |
Arm A - Dose Level 2 | 0 |
Arm A - Dose Level 3 | 0 |
Arm B - Dose Level 1 | 12 |
Arm B - Dose Level 2 | 4 |
Arm B - Dose Level 3 | 24 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm A - Dose Level 1 | 4 |
Arm A - Dose Level 2 | 0 |
Arm A - Dose Level 3 | 0 |
Arm B - Dose Level 1 | 5 |
Arm B - Dose Level 2 | 3 |
Arm B - Dose Level 3 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm A - Dose Level 1 | 24 |
Arm A - Dose Level 2 | 0 |
Arm A - Dose Level 3 | 0 |
Arm B - Dose Level 1 | 1 |
Arm B - Dose Level 2 | 2 |
Arm B - Dose Level 3 | 11 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm A - Dose Level 1 | 6 |
Arm A - Dose Level 2 | 0 |
Arm A - Dose Level 3 | 0 |
Arm B - Dose Level 1 | 6 |
Arm B - Dose Level 2 | 3 |
Arm B - Dose Level 3 | 6 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 0 |
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Graft Rejection
Number of patients with graft rejection (CD3 donor chimerisms <5%). (NCT00358657)
Timeframe: Day 84
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 4 |
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Incidence of Chronic GVHD
Number of patients diagnosed with chronic GVHD by 1 year post transplant (NCT00358657)
Timeframe: 1 year post transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 11 |
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Incidence of Grade I/II Acute Graft Versus Host Disease (GVHD)
Number of patients diagnosed with overall GI/G2 acute GVHD by Day 100 (NCT00358657)
Timeframe: Day 100 post transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 7 |
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Incidence of Grade III/IV Acute Graft Versus Host Disease (GVHD)
Number of patients diagnosed with overall GIII/IV acute GVHD by Day 100 (NCT00358657)
Timeframe: Day 100 post transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 5 |
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Number of Patients With Infections
Number of patients with clinically significant infections requiring treatment within 200 days after HCT (NCT00358657)
Timeframe: Through day 200 after HCT
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemo, Total-body Irradiation, Transplant) | 13 |
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Incidence of Acute Graft-vs-host Disease (aGVHD)
incidence of aGVHD (grades 2 - 4) 100 days post allogeneic hematopoietic cell transplantation (NCT00360685)
Timeframe: 100 days post transplant
Intervention | participants (Number) |
---|
Tacrolimus And Methotrexate | 45 |
Tacrolimus And Mycophenolate Mofetil | 33 |
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Incidence of Severe Mucositis
Mucositis was assessed prospectively daily while the patient was hospitalized and graded retrospectively based on nurse and clinician assessments according to the clinical criteria set forth in the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE; version 3.0). Severe mucositis as defined as grade 3 or grade 4. (NCT00360685)
Timeframe: 2 year
Intervention | participants (Number) |
---|
Tacrolimus And Methotrexate | 25 |
Tacrolimus And Mycophenolate Mofetil | 14 |
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Overall Survival
number of participants alive at one year (NCT00360685)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Tacrolimus And Methotrexate | 28 |
Tacrolimus And Mycophenolate Mofetil | 27 |
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GI Toxicities
Hospitalizations due to Gastrointestinal (GI) toxicities of mycophenolic acid enteric coated (Myfortic) (NCT00374803)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Mycophenolic Acid Loading Group | 5 |
Mycophenolic Acid No Load Group | 4 |
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Incidence of All Biopsy Proven Acute Rejection.
Treatment efficacy, defined as the incidence of all biopsy proven acute rejection. Biopsy was proven with tissue samples collected on patients with elevated serum creatinine (NCT00374803)
Timeframe: 12 months
Intervention | Participants (Number) |
---|
Mycophenolic Acid Loading Group | 1 |
Mycophenolic Acid No Load Group | 4 |
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Incidence of Post Transplant Infections
Incidence of post transplant infections that resulted in hospitalization (NCT00374803)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Mycophenolic Acid Loading Group | 4 |
Mycophenolic Acid No Load Group | 4 |
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Renal Function at 12 Months
Renal function measured by serum creatinine (SCr) at 12 months post-transplant (NCT00374803)
Timeframe: 12 months
Intervention | mg/dL (Mean) |
---|
Mycophenolic Acid Loading Group | 1.36 |
Mycophenolic Acid No Load Group | 1.5 |
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Patient and Allograft Survival 12 Months
Patient and allograft survival at 12 months post-transplant. Allograft survival is different from rejection. An allograft can have rejection, but the allograft can still have survival. If an allograft fails and is no longer functioning this would be considered allograft failure and non-survival. (NCT00374803)
Timeframe: 12 months
Intervention | participants (Number) |
---|
| Patient Survival | Allograft Survival |
---|
Mycophenolic Acid (Myfortic) Preload | 22 | 22 |
,Mycophenolic Acid (Myfortic) Standard | 23 | 22 |
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Incidence of Biopsy Confirmed Acute Rejection at 12 Months.
(NCT00374231)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Corticosteroid Withdrawa. | 5 |
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Patient Survival.
(NCT00374231)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Corticosteroid Withdrawal | 39 |
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Time Post Transplant Corticosteroid Withdrawal
The mean days from post transplant corticosteroid withdrawal. (NCT00374231)
Timeframe: 12 months
Intervention | days (Mean) |
---|
Corticosteroid Withdrawal | 257 |
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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
Intervention | participants (Number) |
---|
| Alive at 1 Year Post Transplant | Died Prior to 1 Year Post Transplant |
---|
Unrelated Donor Umbilical Cord Transplant | 10 | 6 |
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Engraftment Rates
To assess hematopoietic engraftment rates. (NCT00429143)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Haploidentical Allogeneic Transplantation | 23 |
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Incidence of Grades III-IV GVHD
"To determine the incidence and severity of GVHD in these patients using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.'~Severity was graded using CTCAE 3.0 (1=mild, 2=moderate, 3=severe, 4=life threatening/disabling, 5=death)" (NCT00429143)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Haploidentical Allogeneic Transplantation | 2 |
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Lymphoid Recovery
To assess the pace of lymphoid recovery in this patient population. (NCT00429143)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Haploidentical Allogeneic Transplantation | 23 |
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Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD
"To determine the optimal dose of CD3+ donor lymphocytes required for consistent engraftment without the development of grade III/IV GVHD.~Measured as CD3+ donor lymphocytes given as n x 10^8/kg.~n was found to be 2 and was found to be the optimal dose and was the only dose given." (NCT00429143)
Timeframe: 6 months
Intervention | lymphocytes x 10^8/kg (Number) |
---|
Haploidentical Allogeneic Transplantation | 2 |
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Overall Survival of Participants
To determine overall survival at 6 months post-transplant. (NCT00429143)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Haploidentical Allogeneic Transplantation | 13 |
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Complete Donor Hematopoietic Cell Chimerism
Complete donor hematopoietic cell chimerism was evaluated in transplant recipients. Complete donor chimerism will be assessed as the presence of > 95% donor T-cells (CD3+) in the blood. The outcome is reported as the percentage of participants that achieve complete donor chimerism, a number without dispersion. (NCT00568633)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Allo-HSCT + TLI + ATG | 56 |
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Disease-free Survival (DFS)
Disease-free survival is defined as the time interval between the date of attaining a first complete remission (CR) and the date of relapse. Disease free survival (DFS) will compared to conventional therapy vs Non-myeloablative Host Conditioning (NMA HCT). The outcome is reported as the number of participants which never experienced disease relapse (without dispersion). (NCT00568633)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Allo-HSCT + TLI + ATG | 5 |
Best Standard Care | 9 |
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Early Graft Loss
Early graft loss means a failure to achieve donor T-cell chimerism of > 5% at any time after transplant. The outcome is reported as the percentage of participants that experience early graft loss, a number without dispersion. (NCT00568633)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Allo-HSCT + TLI + ATG | 4 |
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Non-relapse Mortality
Non-relapse mortality is defined as death that occurs after therapy, from any cause except a cause associated with relapse. This will be reported as the number of participants experiencing non-relapse mortality (a number without dispersion). (NCT00568633)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Allo-HSCT + TLI + ATG | 1 |
Best Standard Care | 2 |
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Overall Survival (OS)
Overall survival defined as the time interval between the date of attaining a first complete remission (CR) and the date of death from any cause. The outcome is reported as the number of participants alive (without dispersion). (NCT00568633)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Allo-HSCT + TLI + ATG | 9 |
Best Standard Care | 13 |
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Patients Completing the Intended Therapy in Both Arms
The assessment for completion of the intended therapy (in both arms) will be reported as the percentage of participants, a number without dispersion (NCT00568633)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Allo-HSCT + TLI + ATG | 100 |
Best Standard Care | 81.8 |
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Relapse Rate
Relapse will be determined as ≥ 5% blast cells in the bone marrow, not secondary to regeneration after myelosuppressive therapy; OR emergence of extramedullary leukemia; OR the re-emergence of blasts in the peripheral blood. The outcome will be reported as the number and percentage of participants that meet these criteria (a number without dispersion). (NCT00568633)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Allo-HSCT + TLI + ATG | 20 |
Best Standard Care | 24 |
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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
Intervention | Participants (Count of Participants) |
---|
| Whole blood72092237 | Whole blood72092236 | T cells72092237 | T cells72092236 |
---|
| 95-100% | Unknown or not measured | 5-94% | 0-4% |
---|
Transplant - 200 cGy | 6 |
Transplant - 400 cGy | 9 |
Transplant - 200 cGy | 9 |
Transplant - 400 cGy | 3 |
Transplant - 200 cGy | 1 |
Transplant - 400 cGy | 1 |
Transplant - 200 cGy | 7 |
Transplant - 400 cGy | 10 |
Transplant - 400 cGy | 2 |
Transplant - 200 cGy | 0 |
Transplant - 400 cGy | 0 |
Transplant - 200 cGy | 13 |
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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
Intervention | Participants (Count of Participants) |
---|
| Whole blood72092236 | Whole blood72092237 | T cells72092236 | T cells72092237 |
---|
| 95-100% | 5-94% | 0-4% | Unknown or not measured |
---|
Transplant - 200 cGy | 12 |
Transplant - 400 cGy | 8 |
Transplant - 200 cGy | 15 |
Transplant - 400 cGy | 3 |
Transplant - 200 cGy | 1 |
Transplant - 400 cGy | 1 |
Transplant - 200 cGy | 4 |
Transplant - 400 cGy | 4 |
Transplant - 200 cGy | 18 |
Transplant - 200 cGy | 5 |
Transplant - 400 cGy | 0 |
Transplant - 200 cGy | 2 |
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Belatacept Pharmacokinetic (PK) Parameter: Maximum Serum Concentration
Maximum Plasma Concentration (Cmax) is the maximum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | µg/mL (Geometric Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 221.3 |
Group 2: Belatacept (MI) + MMF | 227.6 |
Group 3: Belatacept (LI) + MMF | 205.4 |
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Belatacept Pharmacokinetic (PK) Parameter: Time to Achieve the Maximum Plasma Concentration
Maximum Plasma Concentration (Tmax) is the time taken to reach the maximum observed plasma concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | hour (Median) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1.00 |
Group 2: Belatacept (MI) + MMF | 1.08 |
Group 3: Belatacept (LI) + MMF | 1.00 |
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Belatacept PK Parameter: Amount Excreted in Ascites Fluid Over Days 1 to 14
Amount Excreted in Ascites (Ae,asc) was estimated from the ascites drug concentrations and volumes within a dosing interval. (NCT00555321)
Timeframe: Days 1 to 14
Intervention | µg (Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 46654 |
Group 2: Belatacept (MI) + MMF | 31425 |
Group 3: Belatacept (LI) + MMF | 81451 |
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Belatacept PK Parameter: Area Under the Serum Concentration-time Curve to the End of the Dosing Period (AUCtau)
Area under the plasma concentration-time curve for each dosing interval is determined using the linear trapezoidal rule. The AUC(TAU) of belatacept from the MI regimens and LI regimens were calculated over 2 and 4 weeks respectively. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | µg*h/mL (Geometric Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 19865 |
Group 2: Belatacept (MI) + MMF | 21526 |
Group 3: Belatacept (LI) + MMF | 19730 |
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Belatacept PK Parameter: Minimum Plasma Concentration
Minimum Plasma Concentration (Cmin) is the minimum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | µg/mL (Geometric Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 23.63 |
Group 2: Belatacept (MI) + MMF | 27.20 |
Group 3: Belatacept (LI) + MMF | 5.91 |
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Belatacept PK Parameter: Terminal Half-life
Terminal Half-life (T 1/2) is the time a drug takes for the concentration levels to fall to 50% of their value. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | hour (Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 240.80 |
Group 2: Belatacept (MI) + MMF | 227.74 |
Group 3: Belatacept (LI) + MMF | 207.88 |
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Belatacept PK Parameter: Total Body Clearance
Total body clearance is the rate and extent at which the drug is eliminated from the body. The clearance of a drug is used to understand the processes involved in drug elimination, distribution and metabolism. CLT was estimated from AUC (TAU) between Weeks 12 and 16, assuming steady state. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | mL/h/kg (Geometric Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0.45 |
Group 2: Belatacept (MI) + MMF | 0.41 |
Group 3: Belatacept (LI) + MMF | 0.45 |
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Belatacept PK Parameter: Volume of Distribution
Volume of distribution (Vss) is the volume in which the total amount of drug would need to be uniformly distributed to produce the desired blood concentration. . Vss was estimated from AUC (TAU) between Weeks 12 and 16, assuming steady state. (NCT00555321)
Timeframe: Samples were collected at Pre dose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105.
Intervention | L/kg (Mean) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0.09 |
Group 2: Belatacept (MI) + MMF | 0.08 |
Group 3: Belatacept (LI) + MMF | 0.11 |
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Percentage of Participants Surviving With Functional Graft by End of Study (Includes LTE Data)
For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)
Intervention | percentage of participants (Number) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 93.3 |
Group 2: Belatacept (MI) + MMF | 85.2 |
Group 3: Belatacept (LI) + MMF | 95.8 |
Group 4: Tacrolimus + MMF | 92.1 |
Group 5: Tacrolimus | 96.2 |
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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection (AR), Graft Loss, or Death by 6 Months Post-transplant
Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a blinded central histopathologist using Banff grading schema. Graft loss was defined as impairment of liver function to such a degree that the participant died or underwent re-transplantation. For 95% (confidence interval) CI within each group, normal approximation is used if N>=5, otherwise exact method is used. (NCT00555321)
Timeframe: At 6 months posttransplant
Intervention | percentage of participants (Number) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 48.0 |
Group 2: Belatacept (MI) + MMF | 41.7 |
Group 3: Belatacept (LI) + MMF | 46.9 |
Group 4: Tacrolimus + MMF | 15.1 |
Group 5: Tacrolimus | 38.0 |
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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by 12 Months
Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a blinded central histopathologist using Banff grading. Graft loss was defined as impairment of liver function to such a degree that the participant died or underwent re-transplantation. For 95% CI within each group, normal approximation is used if N>=5. Otherwise exact method is used. For 95% CI of difference, adjustment is made for randomization strata if N >= 5 in each treatment arm. (NCT00555321)
Timeframe: At 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 52.0 |
Group 2: Belatacept (MI) + MMF | 47.9 |
Group 3: Belatacept (LI) + MMF | 53.1 |
Group 4: Tacrolimus + MMF | 18.9 |
Group 5: Tacrolimus | 40.0 |
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Percentage of Participants Who Experienced at Least One Episode of Acute Rejection, Graft Loss, or Death by End of Study (Includes LTE Data)
Any AR that was clinically suspected and biopsy proven (by central pathologist) was included in this triple composite end point. All biopsies for suspected AR were assessed by a central histopathologist using Banff criteria. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)
Intervention | percentage of participants (Number) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 36.7 |
Group 2: Belatacept (MI) + MMF | 37.0 |
Group 3: Belatacept (LI) + MMF | 25.0 |
Group 4: Tacrolimus + MMF | 21.1 |
Group 5: Tacrolimus | 23.1 |
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Belatacept Trough Concentration Before Each Infusion During the LTE
Minimum Plasma Concentration (Cmin) is the minimum observed serum drug concentration. (NCT00555321)
Timeframe: Samples were collected predose on Days 5, 14, 28, 56, 84, 112, 168, 252, 336, 364; after end of infusion on Days 1, 5, 84, 112, 196, 336; and on Days 9, 85, 91, 98, 105, 532, 728.
Intervention | µg/mL (Geometric Mean) |
---|
| Day 5: (n=40, 43, 41) | Day 14: (n=41, 43, 38) | Day 28: (n=42, 38, 37) | Day 56: (n=12, 13, 12) | Day 84: (n=34, 31, 34 ) | Day 112: (n=29, 26, 29) | Day 168: (n=30, 29, 29) | Day 252: (n=28, 28, 27) | Day 336: (n=26, 23, 21) | Day 364: (n=28, 28, 22 ) | Day 532: (n=22, 24, 14) | Day 728: (n=10, 12, 14) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 79.15 | 32.39 | 23.51 | 18.91 | 27.11 | 10.12 | 7.30 | 3.65 | 3.22 | 2.52 | 2.71 | 4.75 |
,Group 2: Belatacept (MI) + MMF | 79.40 | 31.16 | 22.23 | 18.84 | 27.32 | 9.64 | 8.01 | 3.74 | 3.38 | 4.01 | 4.68 | 4.20 |
,Group 3: Belatacept (LI) + MMF | 70.88 | 29.21 | 21.72 | 6.57 | 6.60 | 6.75 | 3.57 | 3.59 | 4.37 | 3.62 | 4.91 | 4.22 |
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Glycosylated Hemoglobin (HbA1C) Values: 12-month Treatment Phase
The HbA1c test is important in diabetes as a long-term measure of control over blood glucose, where the glucose bound to hemoglobin during the past 3-4 months is measured. A baseline diabetes participant was one who had a medical history of diabetes or being under anti-diabetic medication at the time of the transplantation. BL = baseline, DM = Diabetes mellitus. (NCT00555321)
Timeframe: 6, 12 months (mth) posttransplant
Intervention | Percentage of glycosylated hemoglobin (Mean) |
---|
| 6 mo: All participants (n=39, 34, 38, 47, 35) | 6 mo: DM at BL (n=15, 12, 10, 12, 10) | 6 mo: DM at BL/DM at 6m (n=23, 14, 14, 19, 18) | 12 mo: All participants (n=40, 35, 35, 44, 37) | 12 mo: DM at BL (n=15, 11, 7, 11, 12) | 12 mo: DM at BL/DM at 12 mth (n=23,13 ,11,18,20) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 5.8 | 6.5 | 6.1 | 6.0 | 6.9 | 6.3 |
,Group 2: Belatacept (MI) + MMF | 5.8 | 6.6 | 6.4 | 5.8 | 6.7 | 6.6 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 5.6 | 6.1 | 5.9 | 5.7 | 6.7 | 6.2 |
,Group 4: Tacrolimus + MMF | 5.4 | 5.8 | 5.5 | 5.5 | 6.2 | 6.0 |
,Group 5: Tacrolimus | 5.4 | 5.4 | 5.4 | 5.8 | 6.5 | 6.3 |
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Mean Change From Baseline in Calculated GFR During the LTE
GFR is a measure of the rate at which blood is filtered by the kidney. MDRD is an equation (calculation) used to estimate GFR in participants with impaired renal function based on serum creatinine (ScR), age, race, gender, blood urea nitrogen (BUN), and albumin (Alb). GFR (mL/min/1.73 m^2) = 170*(Scr)^-0.999*(Age)^-0.176*(0.762 if female)*(1.180 if African American)*(BUN)^-0.170*(Alb)^+0.318. ). BL= baseline, mL= milliliters; min= minute; m^2= meters squared. (NCT00555321)
Timeframe: Baseline (pretransplant time point), 1, 2, 3, 6,12, 18, 24, 30, 36 months posttransplant
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| Baseline (BL); (n=27,24,20,33,23) | 1 month; (n=28,27,23,36,25) | Change from BL to 1 month; (n=26,24,20,31,23) | 2 months; (n=21,21,15,19,19) | Change from BL to 2 months; (n=20,19,14,17,18) | 3 months; (n=27, 26, 24, 35, 24) | Change from BL to 3 months; (n=24, 24, 20, 30, 21) | 6 months; (n=29, 26, 24, 24, 38, 23) | Change from BL to 6 months; (n=27, 23, 20, 33, 20) | 12 months; (n=29, 25, 24, 36, 24) | Change from BL to 12 months; (n=26, 23, 20, 31,21) | 18 months; (n=24, 25, 21, 33, 21) | Change from BL to 18 months; (n=21, 23, 17, 28,18) | 24 months; (n=13, 15, 15, 22, 15) | Change from BL to 24 months; (n=10, 13, 11, 19,13) | At 30 months; (n=6, 6, 6, 12, 7) | Change from BL to 30 months; (n=5, 5, 3, 8, 6) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 64.4 | 88.2 | 26.3 | 86.2 | 26.0 | 87.0 | 23.3 | 87.6 | 24.0 | 88.1 | 22.6 | 84.8 | 22.5 | 84.8 | 27.9 | 102.9 | 41.4 |
,Group 2: Belatacept (MI) + MMF | 85.6 | 103.1 | 20.2 | 99.4 | 12.6 | 97.0 | 13.7 | 93.1 | 8.6 | 101.3 | 19.4 | 95.0 | 11.2 | 87.6 | 9.9 | 94.4 | 40.4 |
,Group 3: Belatacept (LI) + MMF | 78.0 | 95.1 | 16.0 | 102.3 | 30.7 | 97.0 | 17.0 | 92.3 | 12.2 | 94.8 | 16.5 | 91.3 | 7.4 | 96.3 | 15.2 | 90.4 | 17.1 |
,Group 4: Tacrolimus + MMF | 77.9 | 68.6 | -7.6 | 71.2 | -11.6 | 66.5 | -8.1 | 64.6 | -10.9 | 67.8 | -9.3 | 69.0 | -6.8 | 73.3 | -2.2 | 64.1 | -15.0 |
,Group 5: Tacrolimus | 83.0 | 59.1 | -22.6 | 64.4 | -17.4 | 57.0 | -23.5 | 56.7 | -23.3 | 61.7 | -20.2 | 63.2 | -6.1 | 66.3 | 2.3 | 59.5 | -10.7 |
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Mean Change From Baseline in Calculated GFR, by Modification of Diet in Renal Disease (MDRD) Equation: 12-month Treatment Phase
GFR is a measure of the rate at which blood is filtered by the kidney. MDRD is an equation (calculation) used to estimate GFR in participants with impaired renal function based on serum creatinine (ScR), age, race, gender, blood urea nitrogen (BUN), and albumin (Alb). GFR (mL/min/1.73 m^2) = 170*(Scr)^-0.999*(Age)^-0.176*(0.762 if female)*(1.180 if African American)*(BUN)^-0.170*(Alb)^+0.318. ). BL= baseline, mL= milliliters; min= minute; m^2= meters squared. (NCT00555321)
Timeframe: Baseline [BL] (pretransplant time point), 1, 2, 3, 6 and 12 months posttransplant
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| BL; (n=47, 42, 42, 45, 45) | 1 month; (n=45, 44, 42, 49, 49) | Change from BL to 1 month; (n=43, 38, 37, 41, 45) | 2 months; (n=29, 30, 23, 25, 27) | Change from BL to 2 months; (n=28, 26, 20, 22, 25) | 3 months; (n=36, 36, 35, 46, 37) | Change from BL to 3 months; (n=33, 34, 30, 40, 33) | 6 months; (n=39, 34, 38, 49, 36) | Change from BL to 6 months; (n=37, 30, 33, 42, 31) | 12 months; (n=40, 33, 35, 46, 38) | Change from BL to 12 month; (n=37, 29, 30, 41, 33) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 66.3 | 85.6 | 21.0 | 86.1 | 25.3 | 86.5 | 22.0 | 82.2 | 18.6 | 83.8 | 18.2 |
,Group 2: Belatacept (MI) + MMF | 77.4 | 93.1 | 15.3 | 96.6 | 18.3 | 91.7 | 13.3 | 90.3 | 7.6 | 97.7 | 19.2 |
,Group 3: Belatacept (LI) + MMF | 76.6 | 89.6 | 11.4 | 105.8 | 31.5 | 96.6 | 18.3 | 86.0 | 5.6 | 85.6 | 4.2 |
,Group 4: Tacrolimus + MMF | 73.7 | 64.9 | -8.7 | 76.3 | -1.5 | 65.0 | -8.4 | 61.9 | -11.7 | 68.4 | -6.3 |
,Group 5: Tacrolimus | 80.2 | 62.2 | -17.7 | 66.9 | -14.0 | 60.4 | -20.2 | 59.8 | -22.7 | 63.8 | -17.1 |
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Mean Change From Baseline in Measured Glomerular Filtration Rate (GFR): 12-month Treatment Phase
"GFR was assessed using a true measure of glomerular filtration via iothalamate clearance test. The month 2 time point was selected as the baseline time point with respect to measured GFR due to logistical difficulty in obtaining measured GFR at the time of liver transplant and post-transplant renal function largely stabilizing by 2 months. All Measured GFR > 200 were truncated at 200." (NCT00555321)
Timeframe: Baseline (2 month), 12 months posttransplant
Intervention | mL/min/1.73m^2 (Mean) |
---|
| 2 months (n=37, 36, 35, 41, 37) | 12 months (n=39, 35, 29, 40, 32) | Change from 2 to 12 months (n=31, 29, 26, 36, 29) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 72.4 | 88.9 | 14.2 |
,Group 2: Belatacept (MI) + MMF | 86.6 | 93.1 | 5.8 |
,Group 3: Belatacept (LI) + MMF | 98.6 | 73.1 | -19.6 |
,Group 4: Tacrolimus + MMF | 65.9 | 75.2 | 5.3 |
,Group 5: Tacrolimus | 58.5 | 70.5 | 7.3 |
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Mean Change From Baseline Serum Creatinine at Months 1, 2, 3, 6 and 12
Measurement of SCr is commonly used as an indicator of renal function. High creatinine blood level is an indicator of deficient filtering by the kidney. SCr was determined at baseline and various post-baseline time points. (NCT00555321)
Timeframe: Baseline (pretransplant time point), 1, 2, 3, 6 and 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| Baseline (BL); (n=47, 42, 42, 45, 45) | At 1 months; (n=45, 44, 42, 49, 49) | Change from BL to 1 months; (n=43, 38, 37, 41, 45) | At 2 months; (n=29, 30, 23, 25, 27) | Change from BL to 2 months; (n=28, 26, 20, 22, 25) | At 3 months; (n=36, 36, 35, 46, 37) | Change from BL to 3 months; (n=33, 34, 30, 40, 33) | At 6 months; (n=39, 34, 38, 49, 36) | Change from BL to 6 months; (n=37, 30, 33, 42, 31) | At 12 months; (n=40, 33, 35, 46, 38) | Change from BL to 12 months; (n=37, 29, 30, 41,33) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1.4 | 0.9 | -0.5 | 0.9 | -0.7 | 0.9 | -0.6 | 1.0 | -0.5 | 1.0 | -0.5 |
,Group 2: Belatacept (MI) + MMF | 1.0 | 0.8 | -0.1 | 0.8 | -0.2 | 0.9 | -0.1 | 0.9 | 0.0 | 0.8 | -0.2 |
,Group 3: Belatacept (LI) + MMF | 0.9 | 0.9 | -0.1 | 0.8 | -0.2 | 0.8 | -0.1 | 0.9 | 0.1 | 1.0 | 0.1 |
,Group 4: Tacrolimus + MMF | 1.1 | 1.2 | 0.1 | 1.0 | 0.0 | 1.2 | 0.1 | 1.3 | 0.2 | 1.2 | 0.1 |
,Group 5: Tacrolimus | 1.0 | 1.3 | 0.3 | 1.2 | 0.2 | 1.3 | 0.3 | 1.3 | 0.4 | 1.2 | 0.3 |
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Mean Change in Baseline Values of Cystatin C at 2 and 12 Months
Cystatin C is a protein encoded by the CST3 gene, which is mainly used as a biomarker of kidney function. If kidney function and glomerular filtration rate decline, the blood levels of cystatin C rise. (NCT00555321)
Timeframe: Baseline (pretransplant), 2, and 12 months posttransplant
Intervention | mg/L (Mean) |
---|
| Baseline (BL); (n=44, 47, 48, 49, 43) | 2 months; (n=40, 39, 39, 48, 43) | Change from BL to 2 months; (n=35, 38, 38, 44,37) | 12 months; (n=40, 36, 36, 49, 40) | Change from BL to 12 months; (n=35, 35, 35,45,34) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1.2 | 1.1 | -0.2 | 1.1 | -0.2 |
,Group 2: Belatacept (MI) + MMF | 1.2 | 1.1 | -0.1 | 0.9 | -0.2 |
,Group 3: Belatacept (LI) + MMF | 1.1 | 1.0 | -0.1 | 1.2 | 0.1 |
,Group 4: Tacrolimus + MMF | 1.4 | 1.4 | 0.1 | 1.3 | -0.1 |
,Group 5: Tacrolimus | 1.2 | 1.5 | 0.3 | 1.3 | 0.1 |
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Number of Participants (Who Were HCV Positive at Baseline) With HCV Ribonucleic Acid (RNA) Levels >2.4*10^6 U/mL and >4.7*10^6 U/mL: 12-month Treatment Phase
Recurrent hepatitis C infection of the allograft following liver transplantation can be detected by monitoring HCV RNA levels. In HCV positive participants, quantitative HCV RNA levels > 2.4 * 10^6 U/mL and > 4.7 * 10^6 U/mL were descriptively summarized by treatment group. BL=baseline (NCT00555321)
Timeframe: Baseline (pretransplant), 6 and 12 months (mo) posttransplant
Intervention | participants (Number) |
---|
| BL; HCV RNA > 2.4*10^6 U/mL (n=21,22,19,22,23) | BL; HCV RNA > 4.7 *10^6 U/mL (n=21,22,19,22,23) | 6 mo; HCV RNA > 2.4*10^6 U/mL (n=16,15,15,21,14) | 6 mo; HCV RNA > 4.7*10^6 U/mL (n=16,15,15,21,14) | 12 mo; HCV RNA > 2.4*10^6 U/mL (n=15,13,14,20,13) | 12 mo; HCV RNA > 4.7*10^6 U/mL (n=15,13,14,20,13) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 2 | 0 | 9 | 7 | 7 | 6 |
,Group 2: Belatacept (MI) + MMF | 4 | 1 | 7 | 7 | 7 | 6 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 2 | 1 | 8 | 7 | 7 | 5 |
,Group 4: Tacrolimus + MMF | 3 | 0 | 14 | 11 | 13 | 11 |
,Group 5: Tacrolimus | 1 | 0 | 11 | 10 | 7 | 7 |
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Number of Participants (Who Were HCV Positive at Baseline) With HCV RNA Levels >2.4 * 10^6 U/mL and >4.7 * 10^6 U/mL During the LTE
Recurrent hepatitis C infection of the allograft following liver transplantation can be detected by monitoring HCV RNA levels. In HCV positive participants, quantitative HCV RNA levels > 2.4 x 10^6 U/mL and > 4.7 x 10^6 U/mL were descriptively summarized by treatment group. BL = baseline (NCT00555321)
Timeframe: BL (pretransplant), 12, 18, 24, 30 months (mo) posttransplant
Intervention | participants (Number) |
---|
| BL; HCV RNA > 2.4*10^6 U/mL (n=10,11,6,15,10) | 12 mo; HCV RNA > 2.4*10^6 U/mL (n=11,10,8,16,10) | 12 mo; HCV RNA > 4.7*10^6 U/mL (n=11,10,8,16,10) | 18 mo; HCV RNA > 2.4*10^6 U/mL (n=7,10,3,12,9) | 18 mo; HCV RNA > 4.7*10^6 U/mL (n=7,10,3,12,9) | 24 mo; HCV RNA > 2.4*10^6 U/mL (n=4,3,4,8,4) | 24 mo; HCV RNA > 4.7*10^6 U/mL (n=4,3,4,8,4) | 30 mo; HCV RNA > 2.4*10^6 U/mL (n=1,1,1,2,2) | 30 mo; HCV RNA > 4.7*10^6 U/mL (n=1,1,1,2,2) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0 | 6 | 6 | 3 | 2 | 2 | 2 | 1 | 1 |
,Group 2: Belatacept (MI) + MMF | 1 | 5 | 4 | 2 | 2 | 1 | 0 | 0 | 0 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 0 | 4 | 3 | 2 | 2 | 1 | 0 | 0 | 0 |
,Group 4: Tacrolimus + MMF | 2 | 10 | 8 | 7 | 6 | 6 | 5 | 1 | 1 |
,Group 5: Tacrolimus | 1 | 4 | 4 | 3 | 2 | 1 | 1 | 1 | 1 |
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Number of Participants at Risk of First Acute Rejection as Determined by Kaplan-Meier Method by 12 Months
The time from transplantation to the first AR episode in each treatment arm was summarized using Kaplan-Meier curves. Acute Rejections were clinically suspected and biopsy proven by central pathologist. (NCT00555321)
Timeframe: 3, 6, 9 and 12 months posttransplant
Intervention | participants (Number) |
---|
| By 3 months | By 6 months | By 9 months | By 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 30 | 27 | 25 | 23 |
,Group 2: Belatacept (MI) + MMF | 31 | 29 | 29 | 25 |
,Group 3: Belatacept (LI) + MMF | 30 | 28 | 27 | 23 |
,Group 4: Tacrolimus + MMF | 48 | 47 | 47 | 42 |
,Group 5: Tacrolimus | 34 | 32 | 32 | 28 |
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Number of Participants Having Acute Rejection by Rejection Activity Index During the LTE
Acute Rejections were clinically suspected and biopsy proven by central pathologist. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI). An overall score of 0-2 is considered indeterminate, score of 3-4 is mild, score of 5-6 is moderate, and score of 7-9 is severe. Only the episode with the highest total RAI score for each participant was counted. (NCT00555321)
Timeframe: Day 1 (randomization) through End of study (database lock of 20-June-2011)
Intervention | participants (Number) |
---|
| By 12 months; Indeterminate score | By 12 months; Mild Score | By 12 months; Moderate Score | By 12 months; Severe Score | At end of study; Indeterminate score | At end of study; Mild Score | At end of study; Moderate Score | At end of study; Severe Score |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0 | 8 | 1 | 0 | 0 | 8 | 1 | 0 |
,Group 2: Belatacept (MI) + MMF | 0 | 5 | 1 | 0 | 0 | 5 | 1 | 0 |
,Group 3: Belatacept (LI) + MMF | 0 | 2 | 4 | 0 | 0 | 2 | 4 | 0 |
,Group 4: Tacrolimus + MMF | 0 | 5 | 1 | 0 | 0 | 5 | 1 | 0 |
,Group 5: Tacrolimus | 0 | 4 | 0 | 2 | 0 | 4 | 0 | 2 |
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Number of Participants Having Acute Rejections During the LTE
Acute rejections were clinically suspected and biopsy proven by central pathologist. The number of episodes of AR was counted. (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)
Intervention | participants (Number) |
---|
| By 12 months (m); Overall | By 12 m ; 1 episode | By 12 m ; 2 episodes | By 12 m; >2 episodes | By Database lock; Overall | By Database lock ; 1 episode | By Database lock ; 2 episodes | By Database lock ; >2 episodes |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 9 | 8 | 1 | 0 | 9 | 8 | 1 | 0 |
,Group 2: Belatacept (MI) + MMF | 6 | 6 | 0 | 0 | 6 | 6 | 0 | 0 |
,Group 3: Belatacept (LI) + MMF | 6 | 5 | 1 | 0 | 6 | 5 | 1 | 0 |
,Group 4: Tacrolimus + MMF | 6 | 5 | 1 | 0 | 6 | 5 | 1 | 0 |
,Group 5: Tacrolimus | 6 | 5 | 1 | 0 | 6 | 5 | 0 | 1 |
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Number of Participants Having Acute Rejections: 12-month Treatment Phase
Acute rejections were clinically suspected and biopsy proven by central pathologist. The number of episodes of AR was counted. (NCT00555321)
Timeframe: 3 , 6, and 12 months
Intervention | participants (Number) |
---|
| By 3 months ; Overall | By 3 months ; 1 episode | By 3 months ; 2 episodes | By 3 months ; >2 episodes | By 6 months ; Overall | By 6 months ; 1 episode | By 6 months ; 2 episodes | By 6 months ; >2 episodes | By 12 months ; Overall | By 12 months ; 1 episode | By 12 months ; 2 episodes | By 12 months ; >2 episodes |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 17 | 15 | 1 | 1 | 20 | 18 | 1 | 1 | 22 | 18 | 3 | 1 |
,Group 2: Belatacept (MI) + MMF | 15 | 14 | 1 | 0 | 15 | 12 | 3 | 0 | 16 | 13 | 3 | 0 |
,Group 3: Belatacept (LI) + MMF | 14 | 14 | 0 | 0 | 15 | 14 | 1 | 0 | 16 | 14 | 2 | 0 |
,Group 4: Tacrolimus + MMF | 4 | 4 | 0 | 0 | 5 | 4 | 1 | 0 | 7 | 6 | 1 | 0 |
,Group 5: Tacrolimus | 13 | 12 | 1 | 0 | 15 | 13 | 2 | 0 | 15 | 13 | 2 | 0 |
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Number of Participants Who Had Acute Rejection by Banff Grade by 12 Months
Acute Rejections (AR) were clinically suspected and biopsy proven by central pathologist. The Banff grading is a classification of renal allograft pathology and AR. Grade I: AR requiring moderate (>25%) to severe mononuclear cell interstitial infiltrate and moderate tubulitis; Grade II: AR requiring severe tubulitis and/or intimal arteritis; Grade III: AR requiring transmural arteritis. Only the episode with highest Banff grade for each participant was counted. (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant
Intervention | participants (Number) |
---|
| By 3 months: Grade 1 | By 3 months: Grade 2 | By 3 months: Grade 3 | By 6 months: Grade 1 | By 6 months: Grade 2 | By 6 months: Grade 3 | By 12 months: Grade 1 | By 12 months: Grade 2 | By 12 months: Grade 3 |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 14 | 3 | 0 | 16 | 4 | 0 | 15 | 7 | 0 |
,Group 2: Belatacept (MI) + MMF | 8 | 6 | 1 | 6 | 8 | 1 | 7 | 8 | 1 |
,Group 3: Belatacept (LI) + MMF | 7 | 6 | 1 | 7 | 7 | 1 | 7 | 8 | 1 |
,Group 4: Tacrolimus + MMF | 3 | 1 | 0 | 4 | 1 | 0 | 6 | 1 | 0 |
,Group 5: Tacrolimus | 6 | 5 | 2 | 7 | 6 | 2 | 7 | 6 | 2 |
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Number of Participants Who Had Adverse Events (AEs), Death, Serious AEs (SAEs) or Were Discontinued Due to AEs (Includes Long Term Extension [LTE] Data)
AE=any new untoward medical occurrence or worsening of a pre-existing medical condition in a subject administered an investigational product and that does not necessarily have a causal relationship with this treatment. SAE=any untoward medical occurrence that results in death, is life-threatening, requires or prolongs inpatient hospitalization (including elective surgery), results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. (NCT00555321)
Timeframe: Day 1 (randomization) to Week 104 + within 56 Days after the last infusion/dose, Deaths were monitored up to database lock (20-June-2011)
Intervention | participants (Number) |
---|
| SAEs | Related SAEs | Discontinued due to SAEs | AEs | Related AEs | Discontinued due to AEs | Deaths (due to AE) | Deaths (not due to AE) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 18 | 5 | 2 | 30 | 27 | 2 | 2 | 0 |
,Group 2: Belatacept (MI) + MMF | 22 | 9 | 4 | 27 | 21 | 5 | 3 | 1 |
,Group 3: Belatacept (LI) + MMF | 17 | 9 | 1 | 23 | 20 | 1 | 1 | 0 |
,Group 4: Tacrolimus + MMF | 29 | 12 | 1 | 38 | 33 | 1 | 3 | 0 |
,Group 5: Tacrolimus | 21 | 9 | 0 | 26 | 23 | 0 | 0 | 0 |
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Number of Participants Who Had Adverse Events of Special Interest During 12-month Treatment Phase
AE of of special interest included malignancies (including skin carcinomas), infections (viral, cytomegalovirus, herpes, fungal, and bacterial), serious infections (NCT00555321)
Timeframe: Day 1 (randomization) to 12 months or ≤ 56 days after discontinuation of study medication
Intervention | participants (Number) |
---|
| Malignancies | Post-transplant lymphoproliferative disorder | All Infections | Bacterial infections | Fungal infections | Viral infections | Cytomegalovirus infections | Polyoma virus infections | Herpes infections | Hepatitis C virus recurrence | Serious infections | Autoimmune events | Acute peri-infusional events | Thrombotic and embolic events |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1 | 0 | 32 | 5 | 6 | 10 | 5 | 0 | 3 | 14 | 11 | 0 | 5 | 3 |
,Group 2: Belatacept (MI) + MMF | 0 | 0 | 39 | 11 | 9 | 11 | 4 | 1 | 3 | 7 | 12 | 0 | 1 | 3 |
,Group 3: Belatacept (LI) + MMF | 2 | 1 | 30 | 11 | 14 | 14 | 10 | 0 | 4 | 6 | 13 | 1 | 0 | 7 |
,Group 4: Tacrolimus + MMF | 2 | 0 | 31 | 6 | 6 | 9 | 4 | 0 | 3 | 13 | 12 | 0 | NA | 10 |
,Group 5: Tacrolimus | 2 | 0 | 29 | 13 | 5 | 7 | 1 | 0 | 2 | 9 | 12 | 2 | NA | 5 |
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Number of Participants Who Had AEs of Special Interest During the LTE
AE of special interest included malignancies (including skin carcinomas), infections (viral, cytomegalovirus, herpes, fungal, and bacterial). (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)
Intervention | participants (Number) |
---|
| Malignancies | Infections and Infestations |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1 | 8 |
,Group 2: Belatacept (MI) + MMF | 4 | 6 |
,Group 3: Belatacept (LI) + MMF | 3 | 9 |
,Group 4: Tacrolimus + MMF | 8 | 11 |
,Group 5: Tacrolimus | 3 | 8 |
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Number of Participants Who Had AEs, Death, SAEs or Were Discontinued Due to AEs: 12-month Treatment Phase
AE=any new untoward medical occurrence or worsening of a pre-existing medical condition in a participant administered an investigational product and that does not necessarily have a causal relationship with this treatment. SAE=any untoward medical occurrence that results in death, is life-threatening, requires or prolongs inpatient hospitalization (including elective surgery), results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event (NCT00555321)
Timeframe: Day 1 (randomization) to 12 m + 8 week follow-up or ≤ 56 days after discontinuation of study medication
Intervention | participants (Number) |
---|
| Death | SAEs | Related SAEs | Discontinued due to SAEs | AEs | Related AEs | Discontinued due to AEs |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 4 | 28 | 12 | 7 | 50 | 45 | 7 |
,Group 2: Belatacept (MI) + MMF | 4 | 29 | 11 | 6 | 48 | 34 | 7 |
,Group 3: Belatacept (LI) + MMF | 9 | 37 | 14 | 11 | 48 | 33 | 12 |
,Group 4: Tacrolimus + MMF | 1 | 40 | 16 | 4 | 53 | 42 | 7 |
,Group 5: Tacrolimus | 4 | 35 | 19 | 13 | 50 | 42 | 18 |
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Number of Participants Who Received Anti-hypertensive Therapy at Month 12
(NCT00555321)
Timeframe: 12 months posttransplant
Intervention | participants (Number) |
---|
| Received at least 1 medication | Received 1 medication | Received 2 medications | Received 3 medications | Received 4 medications |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 20 | 13 | 6 | 1 | 1 |
,Group 2: Belatacept (MI) + MMF | 19 | 16 | 2 | 1 | 0 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 14 | 8 | 2 | 4 | 0 |
,Group 4: Tacrolimus + MMF | 26 | 17 | 5 | 3 | 1 |
,Group 5: Tacrolimus | 21 | 12 | 5 | 3 | 1 |
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Number of Participants With Acute Rejections by Rejection Activity Index (RAI) by 12 Months
Acute Rejections were clinically suspected and biopsy proven by central pathologist. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI). An overall score of 0-2 is considered indeterminate, score of 3-4 is mild, score of 5-6 is moderate, and score of 7-9 is severe. Only the episode with the highest total RAI score for each participant was counted. (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant
Intervention | participants (Number) |
---|
| By 3 months; Indeterminate score | By 3 months; Mild Score | By 3 months; Moderate Score | By 3 months; Severe Score | By 6 months; Indeterminate score | By 6 months; Mild Score | By 6 months; Moderate Score | By 6 months; Severe Score | By 12 months; Indeterminate score | By 12 months; Mild Score | By 12 months; Moderate Score | By 12 months; Severe Score |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0 | 14 | 3 | 0 | 0 | 17 | 3 | 0 | 0 | 17 | 4 | 1 |
,Group 2: Belatacept (MI) + MMF | 0 | 8 | 6 | 1 | 0 | 6 | 8 | 1 | 0 | 7 | 8 | 1 |
,Group 3: Belatacept (LI) + MMF | 0 | 7 | 7 | 0 | 0 | 7 | 8 | 0 | 0 | 7 | 9 | 0 |
,Group 4: Tacrolimus + MMF | 0 | 3 | 1 | 0 | 0 | 4 | 1 | 0 | 0 | 6 | 1 | 0 |
,Group 5: Tacrolimus | 0 | 7 | 2 | 4 | 0 | 8 | 3 | 4 | 0 | 8 | 3 | 4 |
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Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type by 12 Months
Acute rejections were clinically suspected and biopsy proven by central pathologist. Corticosteroid-resistant rejection = Continued rejection, as documented by liver biopsy, after the completion of 2 days of corticosteroids and requiring use of T-cell depleting agent. Refractory rejection=Continued rejection, as documented by liver biopsy, after use of corticosteroids and T cell depletion therapy. Increase in the dose of TAC was monitored in participants who were assigned to one of the TAC-based regimens. TRT= treatment (NCT00555321)
Timeframe: 3, 6 and 12 months posttransplant
Intervention | participants (Number) |
---|
| By 3 months: Acute rejections | By 3 months: Treated participants | By 3 months: Corticosteroid treatment Only | By 3 months: Corticosteroid resistant | By 3 months: Refractory | By3 months: Initial lymphocyte depleting TRT | By 3 months: Increase in dose of Tacrolimus | By 3 months: Other / not available | By 6 months: Acute rejections | By 6 months: Treated participants | By 6 months: Corticosteroid treatment Only | By 6 months: Corticosteroid resistant | By 6 months: Refractory | By 6 months: Initial lymphocyte depleting TRT | By 6 months: Increase in dose of Tacrolimus | By 6 months: Other / not available | By 12 months: Acute rejections | By 12 months: Treated participants | By 12 months: Corticosteroid treatment Only | By 12 months: Corticosteroid resistant | By 12 months: Refractory | B12 months: Initial lymphocyte depleting TRT | 12 months: Other / not available |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 7 | 10 | 10 | 0 | 0 | 0 | 0 | 0 | 20 | 12 | 12 | 0 | 0 | 0 | 0 | 0 | 22 | 12 | 12 | 0 | 0 | 0 | 0 |
,Group 2: Belatacept (MI) + MMF | 15 | 11 | 7 | 1 | 1 | 2 | 0 | 0 | 15 | 11 | 6 | 1 | 1 | 3 | 0 | 0 | 16 | 12 | 7 | 1 | 1 | 3 | 0 |
,Group 3: Belatacept (LI) + MMF | 14 | 7 | 7 | 0 | 0 | 0 | 0 | 0 | 15 | 8 | 8 | 0 | 0 | 0 | 0 | 0 | 16 | 8 | 8 | 0 | 0 | 0 | 0 |
,Group 4: Tacrolimus + MMF | 4 | 3 | 2 | 0 | 0 | 0 | 1 | 0 | 5 | 4 | 3 | 0 | 0 | 0 | 1 | 0 | 7 | 5 | 4 | 0 | 0 | 0 | 0 |
,Group 5: Tacrolimus | 13 | 10 | 9 | 0 | 0 | 0 | 1 | 0 | 15 | 12 | 10 | 0 | 0 | 0 | 1 | 1 | 15 | 2 | 10 | 0 | 0 | 0 | 1 |
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Number of Participants With Central Biopsy Proven Acute Rejection by Treatment Type During the LTE
Acute rejections were clinically suspected and biopsy proven by central pathologist. Corticosteroid-resistant rejection = Continued rejection, as documented by liver biopsy, after the completion of 2 days of corticosteroids and requiring use of T-cell depleting agent. Refractory rejection=Continued rejection, as documented by liver biopsy, after use of corticosteroids and T cell depletion therapy. Increase in the dose of TAC was monitored in participants who were assigned to one of the TAC-based regimens. DBL=database lock, TRT=treatment (NCT00555321)
Timeframe: Day 1 (randomization) through database lock (20-June-2011)
Intervention | participants (Number) |
---|
| By 12 months: Acute rejections | By 12 months: Treated participants | By 12 months: Corticosteroid treatment Only | By 12 months: Corticosteroid resistant | By 12 months: Refractory | By 12 months: Initial lymphocyte depleting TRT | By 12 months: Other / not available | By 12 months: Increase in dose of TAC | By DBL: Acute rejections | By DBL: Treated participants | By DBL: Corticosteroid treatment Only | By DBL: Corticosteroid resistant | By DBL: Refractory | By DBL: Initial lymphocyte depleting treatment | By DBL: Other/ Not available | By DBL: Increase in dose of TAC |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 9 | 4 | 4 | 0 | 0 | 0 | 0 | 0 | 9 | 4 | 4 | 0 | 0 | 0 | 0 | 0 |
,Group 2: Belatacept (MI) + MMF | 6 | 5 | 4 | 0 | 0 | 1 | 0 | 0 | 6 | 5 | 4 | 0 | 0 | 1 | 0 | 0 |
,Group 3: Belatacept (LI) + MMF | 6 | 4 | 4 | 0 | 0 | 0 | 0 | 0 | 6 | 4 | 4 | 0 | 0 | 0 | 0 | 0 |
,Group 4: Tacrolimus + MMF | 6 | 5 | 4 | 0 | 0 | 0 | 0 | 1 | 6 | 5 | 4 | 0 | 0 | 0 | 0 | 1 |
,Group 5: Tacrolimus | 6 | 6 | 5 | 0 | 0 | 0 | 0 | 0 | 6 | 6 | 5 | 0 | 0 | 0 | 0 | 1 |
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Number of Participants With Marked Hematology Abnormalities During the LTE
Low platelet count: <50*10^9 c/µl; Low leukocytes: <2.0*10^3 c/µl; Low lymphocytes (absolute): <0.5*10^3 c/µl; Low neutrophils (absolute): <1.0*10^3 c/µl. (NCT00555321)
Timeframe: Every 4 weeks from Week 53 to Week 104.
Intervention | participants (Number) |
---|
| Low Platelets: (n=29, 26, 22, 37, 25) | Low Leukocytes : (n=29, 27, 22, 37, 25) | Low Absolute Lymphocyte: (n=29, 27, 22, 36, 25) | Low Absolute Neutrophils: (n=29, 27, 22, 36, 25) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 1 | 1 | 8 | 0 |
,Group 2: Belatacept (MI) + MMF | 2 | 2 | 3 | 2 |
,Group 3: Belatacept (LI) + MMF | 0 | 0 | 3 | 1 |
,Group 4: Tacrolimus + MMF | 2 | 3 | 5 | 2 |
,Group 5: Tacrolimus | 0 | 0 | 3 | 0 |
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Number of Participants With Marked Hematology Abnormalities: 12-month Treatment Phase
Low hemoglobin: <8 g/dL; Low platelet count: <50*10^9 C/L; Low leukocytes: <2.0 *10^3 c/µL; Low lymphocytes (absolute): <0.5*10^3 c/µL; Low neutrophils (absolute): <1.0*10^3 Cc/µL. (NCT00555321)
Timeframe: Baseline (pretransplant), 2, 4, 8, 12 weeks, and every 4 weeks for week 16 to 52
Intervention | participants (Number) |
---|
| Low Hemoglobin: (n=48, 46, 44, 52, 50) | Low Platelets: (n=48, 46, 43, 52, 50) | Low Leukocytes : (n=48, 46, 44, 52, 50) | Low Absolute Lymphocyte: (n=48, 46, 44, 52, 50) | Low Absolute Neutrophils : (n=48, 46, 44, 52, 50) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 2 | 0 | 6 | 29 | 6 |
,Group 2: Belatacept (MI) + MMF | 6 | 1 | 6 | 18 | 4 |
,Group 3: Belatacept (LI) + MMF | 2 | 4 | 5 | 26 | 4 |
,Group 4: Tacrolimus + MMF | 0 | 2 | 4 | 17 | 8 |
,Group 5: Tacrolimus | 6 | 2 | 3 | 16 | 1 |
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Number of Participants With Marked Liver and Kidney Function Abnormalities During the LTE
ULN= upper limit of normal; Normal ranges are provided by the Central Laboratory and may vary according to sex and age. High alanine aminotransferase (ALT): >5.0*ULN U/L; High aspartate aminotransferase (AST): >5.0*ULN U/L; High direct bilirubin: >3.0*ULN mg/dL; High g-glutamyl transferase (GGT): >5.0*ULN U/L; High total bilirubin: >3.0*ULN mg/dL; High creatinine: > 3.0*ULN mg/dL (NCT00555321)
Timeframe: Every 4 weeks from Week 53 to Week 104.
Intervention | participants (Number) |
---|
| High ALT | High AST | High Direct Bilirubin | High GGT | High Total Bilirubin | High Creatinine |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 0 | 0 | 1 | 2 | 1 | 0 |
,Group 2: Belatacept (MI) + MMF | 2 | 3 | 4 | 6 | 3 | 0 |
,Group 3: Belatacept (LI) + MMF | 1 | 2 | 1 | 6 | 1 | 0 |
,Group 4: Tacrolimus + MMF | 4 | 4 | 3 | 13 | 2 | 1 |
,Group 5: Tacrolimus | 3 | 3 | 2 | 8 | 0 | 0 |
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Number of Participants With Marked Liver and Kidney Function Abnormalities: 12-month Treatment Phase
ULN= upper limit of normal; Normal ranges are provided by the central laboratory and may vary according to sex and age. High alkaline phosphatase (ALP): >5.0*ULN U/L; High alanine aminotransferase (ALT): >5.0*ULN U/L; High aspartate aminotransferase (AST): >5.0*ULN U/L; High direct bilirubin: >3.0 * ULN mg/dL; High g-glutamyl transferase (GGT): >5.0*ULN U/L; High total bilirubin: >3.0*ULN mg/dL; High creatinine: > 3.0*ULN mg/dL (NCT00555321)
Timeframe: Baseline (pretransplant), 4, 12, 24, 52 weeks
Intervention | participants (Number) |
---|
| High ALP: (n=48, 47, 44, 53, 50) | High ALT: (n=48, 47, 44, 53, 50) | High AST: (n=48, 47, 44, 53, 50) | High Direct Bilirubin: (n=48, 47, 44, 53, 50) | High GGT: (n=48, 47, 44, 53, 50) | High Total Bilirubin: (n=48, 47, 44, 53, 50) | High Creatinine: (n=47, 44, 44, 53, 50) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 5 | 16 | 9 | 15 | 25 | 9 | 0 |
,Group 2: Belatacept (MI) + MMF | 6 | 18 | 14 | 18 | 24 | 13 | 0 |
,Group 3: Belatacept (LI) + MMF | 6 | 19 | 16 | 19 | 24 | 16 | 0 |
,Group 4: Tacrolimus + MMF | 4 | 16 | 8 | 20 | 27 | 12 | 0 |
,Group 5: Tacrolimus | 6 | 9 | 2 | 16 | 22 | 10 | 0 |
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Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) by 12 Months
HCV Recurrence is defined as Histological confirmation on liver biopsy by the Ishak (modified Knodell) system and required both a score >= 5 out of 18 on modified Histological Activity Index grading and a fibrosis Score >= 2 out of 6 on modified staging. All biopsies, including Week 52 biopsies, were considered. Only the first HCV recurrence episode for each participant was counted. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. For 95% CI of difference, normal approximation was used if N>=5 in both arms, otherwise exact method was used. (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| 6 months | 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 39.1 | 60.9 |
,Group 2: Belatacept (MI) + MMF | 21.7 | 30.4 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 23.8 | 28.6 |
,Group 4: Tacrolimus + MMF | 20.0 | 52.0 |
,Group 5: Tacrolimus | 33.3 | 37.5 |
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Percentage of Participants (Who Were Hepatitis C Virus [HCV] Positive at Baseline) With HCV Recurrence (Assessed by Central Pathologist) During the LTE
HCV Recurrence is defined as Histological confirmation on liver biopsy by the Ishak (modified Knodell) system and required both a score >= 5 out of 18 on modified Histological Activity Index grading and a fibrosis Score >= 2 out of 6 on modified staging. All biopsies, including Week 52 biopsies, were considered. Only the first HCV recurrence episode for each participant was counted. For 95% CI within each group, normal approximation was used if N>=5, otherwise exact method was used. For 95% CI of difference, normal approximation was used if N>=5 in both arms, otherwise exact method was used. (NCT00555321)
Timeframe: 12 months posttransplant, end of study (database lock, 20-June-2011)
Intervention | percentage of participants (Number) |
---|
| At 12 months | At end of study |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 50.0 | 66.7 |
,Group 2: Belatacept (MI) + MMF | 41.7 | 50.0 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 0 | 22.2 |
,Group 4: Tacrolimus + MMF | 58.8 | 64.7 |
,Group 5: Tacrolimus | 72.7 | 72.7 |
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Percentage of Participants Meeting the Definition of Dyslipidemia, Hypertriglyceridemia or Hypercholesterolemia at Any Given Time: 12-month Treatment Phase
Percentage of participants at any given time (at Month 6 and Month 12) who met the definition of dyslipidemia.Dyslipidemia is defined as hypertriglyceridemia (TGs ≥ 500 mg/dL [5.65 mmol/L]), hypercholesterolemia (LDL ≥ 100 mg/dL [2.59 mmol/L]), or elevated non-HDL (non-HDL ≥ 130 mg/dL [3.36 mmol/L]) in the presence of high TGs (TGs ≥ 200 mg/dL [2.26 mmol/L]). (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| 6 months: Dyslipidemia | 6 months: Hypertriglyceridemia | 6 months: Hypercholesterolemia | 12 months: Dyslipidemia | 12 months: Hypertriglyceridemia | 12 months: Hypercholesterolemia |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 36.0 | 6.0 | 30.0 | 40.0 | 4.0 | 30.0 |
,Group 2: Belatacept (MI) + MMF | 31.3 | 0 | 29.2 | 33.3 | 0 | 29.2 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 30.6 | 0 | 24.5 | 44.9 | 2.0 | 40.8 |
,Group 4: Tacrolimus + MMF | 32.1 | 0 | 30.2 | 34.0 | 1.9 | 30.2 |
,Group 5: Tacrolimus | 26.0 | 0 | 22.0 | 42.0 | 2.0 | 36.0 |
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Percentage of Participants Surviving With Functional Graft: 12-month Treatment Phase
For 95% CI within each group, normal approximation was used if N>=5. Otherwise exact method was used. (NCT00555321)
Timeframe: At 6 and 12 months
Intervention | percentage of participants (Number) |
---|
| At 6 months | At 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 90.0 | 90.0 |
,Group 2: Belatacept (MI) + MMF | 89.6 | 83.3 |
,Group 3: Belatacept (LI) + MMF | 77.6 | 67.3 |
,Group 4: Tacrolimus + MMF | 92.5 | 92.5 |
,Group 5: Tacrolimus | 90.0 | 88.0 |
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Percentage of Participants Who Develop Dyslipidemia, Hypertriglyceridemia and Hypercholesterolemia After Randomization and Transplantation: 12-month Treatment Phase
Percentage of participants who develop dyslipidemia, defined as hypertriglyceridemia (triglycerides [TGs] ≥ 500 mg/dL [5.65 mmol/L]), hypercholesterolemia (Low density lipoprotein [LDL] ≥ 100 mg/dL [2.59 mmol/L]), or elevated non-high density lipoprotein (non- high density lipoprotein [HDL] ≥ 130 mg/dL [3.36 mmol/L]) in the presence of high TGs (TGs ≥ 200 mg/dL [2.26 mmol/L]). (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| By 6 month: Dyslipidemia | By 6 month: Hypertriglyceridemia | By 6 month: Hypercholesterolemia | By 12 month: Dyslipidemia | By 12 month: Hypertriglyceridemia | By 12 month: Hypercholesterolemia |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 50.0 | 2.4 | 42.9 | 59.5 | 4.8 | 54.8 |
,Group 2: Belatacept (MI) + MMF | 54.3 | 0 | 48.6 | 57.1 | 0 | 54.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 45.7 | 0 | 39.1 | 58.7 | 2.2 | 52.2 |
,Group 4: Tacrolimus + MMF | 33.3 | 0 | 28.6 | 50.0 | 2.4 | 42.9 |
,Group 5: Tacrolimus | 59.5 | 0 | 51.4 | 70.3 | 0 | 62.2 |
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Percentage of Participants Who Developed Hypertension in 12-month Treatment Phase
Percentage of participants who develop hypertension after randomization and transplantation. Transient post-operative increases in BP were not to be counted as new onset hypertension. Hypertension was to be assessed only at or after the Week 4 visit. Participants were considered to have hypertension when either of the following criteria were met: (1) SBP ≥ 130 mm Hg or DBP ≥ 80 mm Hg or (2) participant received an antihypertensive medication(s) for the indication of hypertension or due to medical history of hypertension. (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| By 6 months | By 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 94.1 | 100 |
,Group 2: Belatacept (MI) + MMF | 93.3 | 93.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 93.8 | 93.8 |
,Group 4: Tacrolimus + MMF | 100.0 | 100.0 |
,Group 5: Tacrolimus | 100 | 100.0 |
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Percentage of Participants Who Have Hypertension at Any Given Time During the 12-month Treatment Phase
Percentage of participants at any given time who meet the definition of hypertension. Participants were considered to have hypertension when either of the following criteria were met: (1) SBP ≥ 130 mm Hg or DBP ≥ 80 mm Hg or (2) participant received an antihypertensive medication(s) for the indication of hypertension or due to medical history of hypertension. (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| By 6 months | By 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 64.0 | 72.0 |
,Group 2: Belatacept (MI) + MMF | 77.1 | 68.8 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 69.4 | 59.2 |
,Group 4: Tacrolimus + MMF | 71.7 | 79.2 |
,Group 5: Tacrolimus | 70.0 | 70.0 |
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Percentage of Participants With New Onset Diabetes Mellitus (NODM): 12-month Treatment Phase
A participant who did not have diabetes prior to randomization was determined to have NODM if(i) the participant received an antidiabetic medication for a duration of at least 30 days or(ii) at least two fasting plasma glucose (FPG) tests indicate that FPG is>=126 mg/dL (7.0 mmol/L). For 95% CI within each group, normal approximation is used if N>=5. For 95% CI of difference, adjustment is made for randomization strata (HCV-Infection status at baseline) if N >= 5 in each treatment arm. (NCT00555321)
Timeframe: 6 and 12 months posttransplant
Intervention | percentage of participants (Number) |
---|
| 6 months | 12 months |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 35.5 | 35.5 |
,Group 2: Belatacept (MI) + MMF | 15.6 | 15.6 |
,Group 3: Belatacept (LI) + MMF | 13.9 | 13.9 |
,Group 4: Tacrolimus + MMF | 21.1 | 23.7 |
,Group 5: Tacrolimus | 35.1 | 37.8 |
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Summary Statistics for Diastolic Blood Pressure: 12-month Treatment Phase
Participants were considered to have hypertension if they had Diastolic Blood Pressure (SBP) ≥ 80 mmHg. (NCT00555321)
Timeframe: BL (pretransplant), 1, 3, 6, 9, 12 months posttransplant
Intervention | mm Hg (Mean) |
---|
| BL (n=50, 48, 49, 53, 49) | 1 month (n=50, 48, 49, 52, 48) | Change from BL to 1 month (n=50, 48, 49, 52, 47) | 3 months (n=44, 46, 41, 48, 42) | Change from BL to 3 months (n=44, 46, 41, 48, 41) | 6 months (n=43, 40, 36, 47, 37) | Change from BL to 6 months (n=43, 40, 36, 47, 36) | 9 months (n=34, 30, 29, 41, 31) | Change from BL to 9 months (n=34, 30, 29, 41, 31) | 12 months (n=42, 37, 36, 46, 38) | Change from BL to 12 months (n=42, 37, 36, 46, 37) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 64.5 | 74.4 | 10.0 | 77.4 | 11.5 | 74.4 | 8.8 | 75.4 | 10.0 | 76.5 | 10.26 |
,Group 2: Belatacept (MI) + MMF | 63.1 | 74.7 | 11.7 | 78.3 | 15.2 | 79.4 | 17.0 | 78.5 | 15.2 | 78.5 | 16.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 62.9 | 72.6 | 9.7 | 76.1 | 12.0 | 76.3 | 12.5 | 77.6 | 13.2 | 74.5 | 10.6 |
,Group 4: Tacrolimus + MMF | 67.5 | 77.8 | 10.7 | 81.7 | 14.4 | 77.8 | 10.7 | 79.5 | 11.2 | 80.3 | 10.21 |
,Group 5: Tacrolimus | 68.6 | 74.6 | 5.8 | 78.2 | 9.9 | 76.6 | 8.6 | 79.5 | 11.2 | 79.5 | 10.8 |
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Summary Statistics for Lipid Parameters - Serum Triglyceride: 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| BL (n=29, 30, 25, 34, 32) | 1 month (n=29, 33, 29, 41, 43) | Change from BL to 1 month (n=16, 20, 17, 27, 30) | 6 months (n=26, 29, 25, 41, 30) | Change from BL to 6 months (n=15, 19, 18, 28, 22) | 12 months (n=38, 29, 32, 42, 34) | Change from BL to 12 months (n=22, 19, 20, 25, 21) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 94.4 | 153.9 | 71.2 | 234.3 | 199.9 | 255.1 | 237.2 |
,Group 2: Belatacept (MI) + MMF | 110.1 | 162.9 | 45.7 | 157.1 | 18.3 | 159.0 | 20.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 80.2 | 149.0 | 85.8 | 162.7 | 88.7 | 158.2 | 91.6 |
,Group 4: Tacrolimus + MMF | 84.4 | 149.1 | 68.2 | 148.1 | 60.8 | 156.4 | 72.3 |
,Group 5: Tacrolimus | 89.2 | 163.5 | 71.8 | 167.2 | 84.3 | 190.5 | 92.6 |
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Summary Statistics for Lipid Parameters- Serum Cholesterol: 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| BL (n=45, 42, 44, 49, 47) | 1 month (n=46, 47, 42, 49, 49) | Change from BL to 1 month (n=42, 41, 39, 45, 46) | 6 months (n=39, 34, 38, 48, 36) | Change from BL to 6 months (n=35, 30, 35, 44, 33) | 12 months (n=40, 33, 35, 45, 37) | Change from BL to 12 months (n=35, 29,32, 42, 34) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 112.9 | 166.3 | 57.0 | 180.8 | 68.6 | 186.8 | 79.4 |
,Group 2: Belatacept (MI) + MMF | 119.7 | 184.9 | 67.7 | 177.3 | 51.3 | 178.9 | 57.5 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 121.3 | 190.6 | 61.3 | 182.2 | 56.6 | 186.4 | 60.7 |
,Group 4: Tacrolimus + MMF | 111.0 | 162.9 | 54.0 | 170.6 | 58.9 | 163.8 | 58.2 |
,Group 5: Tacrolimus | 106.4 | 164.1 | 53.4 | 177.9 | 60.8 | 175.2 | 57.3 |
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Summary Statistics for Lipid Parameters- Serum Low Density Lipoprotein Cholesterol (LDL): 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| BL (n=29, 30, 25, 34, 32) | 1 month (n=30, 34, 29, 41, 42) | Change from BL to 1 month (n=17, 21, 17, 27, 30) | 6 months (n=27, 29, 27, 41, 30) | Change from BL to 6 months (n=16, 19, 18, 28, 22) | 12 months (n=38, 29, 32, 42, 35) | Change from BL to 12 months (n=22, 19, 20, 25, 22) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 52.3 | 95.2 | 42.7 | 99.2 | 41.2 | 93.0 | 37.8 |
,Group 2: Belatacept (MI) + MMF | 58.6 | 93.2 | 34.3 | 107.5 | 45.9 | 107.2 | 45.4 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 58.9 | 115.7 | 57.1 | 98.2 | 48.7 | 103.2 | 37.2 |
,Group 4: Tacrolimus + MMF | 63.6 | 89.8 | 26.1 | 96.7 | 28.9 | 89.0 | 23.7 |
,Group 5: Tacrolimus | 59.9 | 98.4 | 41.5 | 92.8 | 26.1 | 93.8 | 13.7 |
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Summary Statistics for Lipid Parameters-Serum Total Non-High Density Lipoprotein (Non-HDL) Cholesterol: 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| BL (n=45, 42, 44, 49, 46) | 1 months (n=46, 47, 42, 49, 49) | Change from BL to 1 months (n=42, 41, 39, 45, 45) | 6 months (n=39, 34, 38, 48, 36) | Change from BL to 6 months (n=35, 30, 35, 44, 32) | 12 months (n=40, 33, 35, 45, 37) | Change from BL to 12 months (n=35, 29, 32, 42, 33) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 80.4 | 125.1 | 47.2 | 139.3 | 59.5 | 143.2 | 66.7 |
,Group 2: Belatacept (MI) + MMF | 83.5 | 147.9 | 66.0 | 134.4 | 44.0 | 137.4 | 49.2 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 84.8 | 148.0 | 54.1 | 138.9 | 48.2 | 142.2 | 53.4 |
,Group 4: Tacrolimus + MMF | 77.7 | 121.7 | 47.0 | 125.1 | 48.1 | 118.2 | 47.4 |
,Group 5: Tacrolimus | 76.8 | 130.0 | 47.5 | 135.4 | 48.6 | 131.9 | 44.1 |
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Summary Statistics for Lipid Parameters; Serum HDL Cholesterol: 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 6, 12 months posttransplant
Intervention | mg/dL (Mean) |
---|
| BL (n=46, 42, 44, 49, 46) | 1 month (n=46, 47, 42, 49, 49) | Change from BL to 1month (n=43, 41, 39, 45, 45) | 6 months (n=39, 34, 38, 48, 36) | Change from BL to 6 months (n=36, 30, 35, 44, 32) | 12 months (n=40, 33, 35, 45, 37) | Change from BL to12 months (n=36, 29, 32, 42, 33) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 32.1 | 41.3 | 9.9 | 41.5 | 9.2 | 43.6 | 12.7 |
,Group 2: Belatacept (MI) + MMF | 36.2 | 36.9 | 1.7 | 42.8 | 7.3 | 41.5 | 8.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 36.5 | 42.5 | 7.2 | 44.2 | 9.4 | 44.2 | 7.3 |
,Group 4: Tacrolimus + MMF | 33.3 | 41.2 | 7.1 | 45.5 | 10.8 | 45.6 | 10.9 |
,Group 5: Tacrolimus | 31.1 | 34.1 | 3.4 | 42.6 | 9.5 | 43.3 | 10.0 |
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Summary Statistics for Mean Arterial Pressure: 12-month Treatment Phase
(NCT00555321)
Timeframe: BL (pretransplant), 1, 3, 6, 9, 12 months posttransplant
Intervention | mm Hg (Mean) |
---|
| BL (n=50, 48, 49, 53, 49) | 1 month (n=50, 48, 49, 52, 48) | Change from BL to 1 month (n=50, 48, 49, 52, 47) | 3 months (n=44, 46, 41, 48, 41) | Change from BL to 3 months (n=44, 46, 41, 48, 41) | 6 months (n=43, 40, 36, 47, 37) | Change from BL to 6 months (n=43, 40, 36, 47, 36) | 9 months (n=34, 30, 29, 41, 31) | Change from BL to 9 months (n=34, 30, 29, 41, 31) | 12 months (n=42, 37, 36, 46, 38) | Change from BL to 12 months (n=42, 37, 36, 46, 37) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 81.4 | 90.7 | 9.3 | 93.9 | 11.3 | 91.0 | 8.7 | 92.1 | 10.1 | 93.0 | 10.1 |
,Group 2: Belatacept (MI) + MMF | 80.5 | 91.9 | 11.4 | 94.5 | 14.0 | 95.1 | 14.4 | 94.3 | 13.1 | 94.6 | 14.3 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 79.1 | 88.7 | 9.6 | 92.2 | 12.2 | 92.4 | 12.6 | 93.0 | 13.2 | 90.1 | 10.6 |
,Group 4: Tacrolimus + MMF | 85.4 | 95.0 | 10.0 | 100.1 | 15.1 | 95.4 | 10.5 | 98.2 | 12.3 | 99.2 | 14.3 |
,Group 5: Tacrolimus | 87.5 | 91.3 | 4.1 | 96.5 | 9.6 | 95.0 | 8.5 | 97.7 | 11.3 | 99.0 | 12.1 |
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Summary Statistics for Systolic Blood Pressure: 12-month Treatment Phase
(NCT00555321)
Timeframe: Baseline (pretransplant), 1, 3, 6, 9, 12 months posttransplant
Intervention | mm Hg (Mean) |
---|
| BL (n=50, 48, 49, 53, 49) | 1 month (n=50, 48, 49, 52, 48) | Change from BL to 1 month (n=50, 48, 49, 52, 47) | 3 months (n=44, 46, 41, 48, 42) | Change from BL to 3 months (n=44, 46, 41, 48, 41) | 6 months (n=43, 40, 36, 47, 37) | Change from BL to 6 months (n=43, 40, 36, 47, 36) | 9 months (n=34, 30, 29, 41, 31) | Change from BL to 9 months (n=34, 30, 29, 41, 31) | 12 months (n=42, 37, 36, 46, 38) | Change from BL to 12 months (n=42, 37, 36, 46, 37) |
---|
Group 1: Basiliximab+Belatacept (MI) + MMF | 115.3 | 123.3 | 8.0 | 127.0 | 10.9 | 124.0 | 8.4 | 125.5 | 10.4 | 125.8 | 9.2 |
,Group 2: Belatacept (MI) + MMF | 115.4 | 126.3 | 10.9 | 126.9 | 11.7 | 126.5 | 9.3 | 125.8 | 8.8 | 127.0 | 10.2 |
,Group 3: Belatacept Less Intensive (LI) + MMF | 111.4 | 120.9 | 9.4 | 124.4 | 12.7 | 124.6 | 12.8 | 123.9 | 13.3 | 121.2 | 10.6 |
,Group 4: Tacrolimus + MMF | 121.2 | 129.2 | 8.5 | 136.9 | 16.3 | 130.6 | 10.0 | 135.6 | 14.4 | 137.0 | 15.9 |
,Group 5: Tacrolimus | 125.3 | 124.6 | 0.6 | 133.0 | 9.1 | 132.0 | 8.3 | 133.9 | 11.6 | 138.0 | 14.6 |
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Number of Participants Experiencing Graft Failure
graft failure = absolute neutrophil count (ANC) <5 x 10^8/L and an acellular bone marrow aspirate/biopsy (NCT00630253)
Timeframe: From Day 1 to event, assessed up to100 days
Intervention | Participants (Count of Participants) |
---|
Marrow Isolex | 0 |
UCB Arm | 0 |
Marrow Clinimax | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Marrow Isolex | 15 |
UCB Arm | 8 |
Marrow Clinimax | 5 |
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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
Intervention | Participants (Count of Participants) |
---|
Marrow Isolex | 0 |
UCB Arm | 0 |
Marrow Clinimax | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Marrow Isolex | 2 |
UCB Arm | 0 |
Marrow Clinimax | 0 |
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EFS
event-free survival after umbilical cord blood transplant (NCT00622895)
Timeframe: 5 years
Intervention | Participants (Count of Participants) |
---|
Treatment: Allogeneic UCB After Reduced Intensity Conditioning | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 0 |
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Overall Survival
Event is defined as death due to any cause. (NCT00622895)
Timeframe: Up to 5 years
Intervention | Participants (Count of Participants) |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 2 |
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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
Intervention | units on a scale (Number) |
---|
| acute GVHD severity maximum grade | chronic GVHD maximum grade |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 2 | 2 |
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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
Intervention | units on a scale (Number) |
---|
| pre-transplant SHAQ | 5 year post transplant | pre-transplant Raynaud symptoms | 5 year post transplant Raynaud symptoms | pre-transplant Finger ulcer symptoms | 5 year post-transplant Finger ulcer symptoms | pre-transplant Overall health symptoms | 5 year post-transplant overall health symptoms |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 1.125 | 0.125 | 3 | 0.5 | 3 | 0 | 2.5 | 0.2 |
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Skin Score
The skin score measure is a scale: the name of the scale is the modified Rodnan skin score (mRSS). Total score of mRSS is from 0 to 51. Higher values represents worse skin score. Highest value is 51, represents very hidebound tight thick skin. Lowest value is 0, represent normal skin, no tightness. (NCT00622895)
Timeframe: Up to 5 years post-transplant
Intervention | units on a scale (mRSS) (Number) |
---|
| Pre-transplant (baseline) skin score | 5 year post-transplant skin score |
---|
Treatment: Allogeneic HCT After Reduced Intensity Conditioning | 17 | 4 |
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Stimulation of Growth After 12 Months (Delta Z-score)
(NCT00707759)
Timeframe: 12 months
Intervention | units on a scale (Mean) |
---|
A: Withdrawal Steroids | 1.2 |
B: Control Steroids | 0.6 |
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Count of Participants With Acute Cellular Rejection Grade Equal to or Greater Than IA, by the Banff 2007 Criteria
Acute cellular rejection is when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade ≥ IA by Banff 2007 criteria. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 2 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 4 |
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Count of Participants With Antibody Mediated Rejection
Antibody mediated rejection (AMR) is defined as diffusely positive staining for C4d, presence of circulating anti-donor antibodies and morphologic evidence of acute tissue injury. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 1 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 |
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Count of Participants With Biopsy Proven Acute Rejection at Any Time Post-Transplant
Biopsy proven acute rejection was defined as histologic evidence of borderline or higher cellular rejection per local pathologist. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 3 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 2 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 5 |
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Count of Participants With CAN/IFTA Grade I, II or III at Any Time Post-transplant
CAN/IFTA grades were determined per local pathology interpretations of biopsy tissue. These grades reflect the severity of interstitial fibrosis and tubular atrophy present in the tissue obtained during a kidney biopsy. Higher grades indicate greater severity in interstitial fibrosis and tubular atrophy present the kidney biopsy tissue. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 1 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 3 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 5 |
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Count of Participants With de Novo Anti-donor HLA Antibodies at Wk 52
The presence of antibodies reactive to Histocompatibility Antigen (HLA) molecules expressed on the renal allograft have been associated with both acute and chronic injury to the transplanted kidney. The development of de novo anti- donor HLA antibodies may mean a person is more likely to reject the graft. (NCT01436305)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 |
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Count of Participants With Delayed Graft Function Post-Transplant
Delayed graft function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function (NCT01436305)
Timeframe: Any time within the first week post-transplant
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 2 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 |
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Count of Participants With Infections Requiring Hospitalization or Systemic Therapy Reported as Serious Adverse Events
Infections of certain types (i.e., excluding those identified in the protocol as occurring commonly in this study population) were required to be reported as a serious adverse event if they required either inpatient hospitalization of prolongation of a current hospitalization. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 2 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 2 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 1 |
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Count of Participants With Rejection
The number of participants who were treated by their local physician for any type of rejection including, but not limited to cellular rejection and antibody- mediated rejection of the transplanted kidney regardless of the presence of a biopsy. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 1 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 3 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 5 |
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Count of Participants With Treated Diabetes Between Day 14 and Wk 52
Treated diabetes is defined as the receipt of oral medication or insulin for >14 days between 14 days and 52 weeks post-transplant (NCT01436305)
Timeframe: Day 14 to Week 52
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 1 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 1 |
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Count of Participants With Use of Anti-hypertensive Medications at Wk 52
Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stoke and myocardial infarction. (NCT01436305)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 3 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 3 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 7 |
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Mean Glomerular Filtration Rate (GFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52
GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥ 90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. (NCT01436305)
Timeframe: Week 52
Intervention | mL/min/1.73m^2 (Mean) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 55.9 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 51.6 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 58.3 |
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Number of Events of Death or Graft Loss
This measure counts deaths and graft loss occurring at any point post transplantation. Graft loss is defined as need for dialysis for greater than 30 days duration, allograft nephrectomy, or retransplantation. (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Events (Number) |
---|
Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 2 |
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 3 |
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 |
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Count of Participants by Chronic Kidney Disease (CKD) Stage Post-Transplant
"The stages of Chronic Kidney Disease are defined using the participant's GFR value as indicated below:~Stage 1 if GFR value is ≥90; Stage 2 if GFR value is ≥60 and < 90; Stage 3A if 45 ≤GFR < 60; Stage 3B if 30 ≤ GFR < 45; Stage 4 if 15 ≤GFR < 30;l Stage 5 if GFR < 15.~Stage 1 means kidney function is normal. Stage 2 indicates mildly reduced kidney function, pointing to kidney disease. Stages 3A and 3B indicate moderately reduced kidney function. Stage 4 indicates severely reduced kidney function. Stage 5 indicates very severe or end stage kidney failure." (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156
Intervention | Participants (Count of Participants) |
---|
| Week 52 - Stage 1 | Week 52 - Stage 2 | Week 52 - Stage 3A | Week 52 - Stage 3B | Week 52 - Stage 4 | Week 104 - Stage 1 | Week 104 - Stage 2 | Week 104 - Stage 3A | Week 104 - Stage 3B | Week 104 - Stage 4 | Week 156 - Stage 1 | Week 156 - Stage 2 | Week 156 - Stage 3A | Week 156 - Stage 3B | Week 156 - Stage 4 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 | 1 | 1 | 0 | 1 | 1 | 0 | 1 | 0 | 0 | 0 | 1 | 1 | 0 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 1 | 3 | 0 | 0 | 0 | 1 | 1 | 1 | 0 | 0 | 1 | 1 | 1 | 0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 | 3 | 3 | 1 | 0 | 1 | 3 | 2 | 1 | 0 | 0 | 5 | 1 | 1 | 0 |
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Count of Participants by Severity of First Acute Cellular Rejection by Wk 52
"Acute cellular rejection is when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade ≥ IA by Banff 2007 criteria. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Originally, this endpoint was worded as The severity of first and highest acute cellular rejection within the first 52 weeks. But since the highest grade for each subject coincided with the first ACR episode for each subject, only a summary of severity of the first episode is presented here." (NCT01436305)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
| Grade IA | Grade IB | Grade IIA | Grade IIB | Grade III |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 1 | 0 | 0 | 1 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 0 | 0 | 0 | 0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 1 | 0 | 2 | 1 | 0 |
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Count of Participants With BKV and CMV Viremia (Local Center Monitoring) Reported as Adverse Events
"Viral infections following renal transplantation is significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during this study using participant blood samples.~Acronyms: BK Polyoma Virus (BKV); Cytomegalovirus (CMV)." (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Participants (Count of Participants) |
---|
| BKV | CMV |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 1 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 2 | 0 |
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Count of Participants With CKD Stage 4 or 5
"The stages of Chronic Kidney Disease are defined using the participant's GFR value as indicated below.~Stage 1 if GFR value is ≥90; Stage 2 if 60 ≤ GFR < 90; Stage 3A if 45 ≤ GFR < 60; Stage 3B if 30 ≤ GFR < 45; Stage 4 if 15 ≤ GFR < 30; Stage 5 if GFR < 15.~Stage 1 means kidney function is normal. Stage 2 indicates mildly reduced kidney function, pointing to kidney disease. Stages 3A abd 3B indicate moderately reduced kidney function. Stage 4 indicates severely reduced kidney function. Stage 5 indicates very severe or end stage kidney failure." (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156
Intervention | Participants (Count of Participants) |
---|
| Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 1 | 0 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 0 | 0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 | 0 | 0 |
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Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Wk 52 Based on Criteria Specified by the ADA and WHO
"New onset diabetes is the development of diabetes post-kidney transplant. It was identified by the clinical sites caring for each participant and reported directly in the clinical database. Impaired fasting glucose (IFG) is a determination made by referencing glucose measurements obtained from a standard chemistry panel. Any fasting glucose measure that is between 110 and 125 mg/dL is classified as IFG.~Acronyms: American Diabetes Association (ADA); World Health Organization (WHO)." (NCT01436305)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
| New onset diabetes during first 52 weeks | Impaired fasting glucose at week 52 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 1 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 | 0 |
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Count of Participants With Estimated Glomerular Filtration Rate (GFR) < 60 mL/Min/1.73 m^2 by CKD EPI
GFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. This measure specifically looked at participants with scores less than 60. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156
Intervention | Participants (Count of Participants) |
---|
| Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 2 | 1 | 1 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 3 | 2 | 2 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 4 | 3 | 2 |
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Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90mm Hg Within 24 Hours of Onset of Transplant Procedure
Temperature of >39 degrees Celsius would be an indication of fever most often in response to an infection or illness. Systolic blood pressure <90mm Hg would be an indication of low blood pressure. (NCT01436305)
Timeframe: 24 hours after transplantation
Intervention | Participants (Count of Participants) |
---|
| Fever >39 degrees | Systolic BP <90 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 | 1 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 0 | 0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 | 0 |
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Count of Participants With Use of Lipid Lowering Medications at Baseline and Wks 24, 52, 104 and 156
Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood (NCT01436305)
Timeframe: Baseline, Week 24, Week 52, Week 104, Week 156
Intervention | Participants (Count of Participants) |
---|
| Baseline | Week 24 | Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 1 | 1 | 1 | 1 | 1 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 5 | 4 | 3 | 3 | 3 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 2 | 2 | 2 | 3 | 3 |
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Fasting Lipid Profile (Total Cholesterol, Non-HDL Cholesterol, LDL, HDL, and Triglyceride) at Baseline and Wks 24, 52, 104 and 156
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are detailed below.~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease" (NCT01436305)
Timeframe: Baseline, Week 24, Week 52, Week 104, Week 156
Intervention | mg/dL (Mean) |
---|
| Tot. Chol. Baseline | Tot. Chol. W24 | Tot. Chol. W52 | Tot. Chol. W104 | Tot. Chol. W156 | Non-HDL Baseline | Non-HDL W24 | Non-HDL W52 | Non-HDL W104 | Non-HDL W156 | LDL Baseline | LDL W24 | LDL W52 | LDL W104 | LDL W156 | HDL Baseline | HDL W24 | HDL W52 | HDL W104 | HDL W156 | Triglyc. Baseline | Triglyc. W24 | Triglyc. W52 | Triglyc. W104 | Triglyc. W156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 160.2 | 159.0 | 187.0 | 142.5 | 133.5 | 118.8 | 129.3 | 151.0 | 110.5 | 102.5 | 86.6 | 86.6 | 114.0 | 58.0 | 55.5 | 41.3 | 29.7 | 36.0 | 32.0 | 31.0 | 307.8 | 249.7 | 187.0 | 220.0 | 228.0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 141.2 | 171.6 | 156.0 | 170.5 | 183.5 | 108.2 | 128.0 | 117.5 | 129.5 | 138.5 | 76.7 | 76.7 | 69.5 | 100.5 | 116.0 | 33.0 | 43.6 | 38.5 | 41.0 | 45.0 | 158.7 | 161.0 | 319.3 | 146.0 | 115.0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 165.6 | 185.6 | 157.5 | 189.0 | 181.7 | 122.3 | 129.0 | 61.0 | 135.6 | 136.0 | 83.4 | 83.4 | 49.0 | 101.4 | 106.0 | 43.3 | 56.6 | 59.0 | 53.4 | 45.7 | 206.9 | 115.9 | 58.0 | 172.8 | 156.5 |
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HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156
Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control. A value below 6.0% reflects normal levels, 6.0% to 6.4% reflects prediabetes, and a value of ≥ 6.5% reflects diabetes. (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156
Intervention | percent (Mean) |
---|
| Day 28 | Day 84 | Week 24 | Week 36 | Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 5.1 | 5.0 | 5.1 | 5.3 | 4.8 | 5.2 | 5.2 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 5.3 | 5.7 | 6.9 | 6.7 | 7.0 | 5.7 | 5.6 |
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HbA1c Measured at Days 28 & 84, and Weeks 24, 36, 52, 72, 104 and 156
Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control. A value below 6.0% reflects normal levels, 6.0% to 6.4% reflects prediabetes, and a value of ≥ 6.5% reflects diabetes. (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156
Intervention | percent (Mean) |
---|
| Day 28 | Day 84 | Week 24 | Week 36 | Week 52 | Week 72 | Week 104 | Week 156 |
---|
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 5.8 | 5.9 | 6.5 | 7.2 | 7.6 | 8.1 | 6.6 | 7.8 |
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Mean Calculated eGFR Using MDRD 4 Variable Model
The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 47.8 | 69.3 | 65.5 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 52.4 | 54.2 | 49.0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 55.7 | 60.4 | 61.5 |
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Number of All Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse events were collected systematically from enrollment through last study visit. Displayed below are counts of all adverse events per treatment group (including both serious and non-serious adverse events). Separately counts of all adverse events determined to be serious are displayed per treatment group. More detail about adverse events for this trial is displayed in the 'Adverse Event' section. (NCT01436305)
Timeframe: Enrollment through last study visit (up to week 156)
Intervention | Events (Number) |
---|
| All Adverse Events | Serious Adverse Events |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 25 | 11 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 21 | 6 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 40 | 7 |
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Standardized Blood Pressure Measurement at Wk 52
A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart. Systolic measures of <120 and diastolic measures of <80 are considered normal. Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension). Systolic measures of ≥140 and diastolic measures of ≥90 are considered high. (NCT01436305)
Timeframe: Week 52
Intervention | mmHg (Mean) |
---|
| Systolic Blood Pressure at Week 52 | Diastolic Blood Pressure at Week 52 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 146.7 | 92.7 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 147.5 | 80.8 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 139.9 | 79.3 |
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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine
The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI). A score of ≥90 means kidney function is normal. A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Scores between 30 and 59 indicates moderately reduced kidney function. Scores between 15 and 29 indicate severely reduced kidney function. Scores below 15 indicate very severe or endstage kidney failure. An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it can be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function. Larger numbers indicate greater change in kidney function. (NCT01436305)
Timeframe: Week 52, Week 104, and Week 156
Intervention | Change in eGFR (mL/min/1.73m^2) by month (Mean) |
---|
| Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0.62 | 0.62 | 0.69 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 1.29 | 1.27 | 1.33 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 1.07 | 0.68 | 0.48 |
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Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156
This is a measure of the total number of pills a participant was prescribed on a given day (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156
Intervention | Number of pills (Mean) |
---|
| Day 28 | Day 84 | Week 24 | Week 36 | Week 52 | Week 104 | Week 156 |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 15.8 | 13.5 | 8.3 | 14.0 | 14.3 | 15.5 | 15.0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 28.8 | 22.2 | 14.8 | 13.0 | 14.6 | 15.3 | 14.0 |
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Total Daily Prescribed Pill Number at Days 28 and 84, and Wks 24, 36, 52, 72, 104 and 156
This is a measure of the total number of pills a participant was prescribed on a given day (NCT01436305)
Timeframe: Day 28, Day 84, Week 24, Week 36, Week 52, Week 72, Week 104, Week 156
Intervention | Number of pills (Mean) |
---|
| Day 28 | Day 84 | Week 24 | Week 36 | Week 52 | Week 72 | Week 104 | Week 156 |
---|
Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 27.3 | 21.3 | 16.6 | 17.0 | 17.8 | 16.0 | 14.8 | 12.7 |
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Type of Treatment of Rejection
"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of biopsy findings and corresponding treatment are presented here for each instance of treatment for rejection. Acronyms and abbreviations are defined below.~ACR=Acute Cellular Rejection ATG=Anti-thymocyte globulin therapy Chr. AMR=Chronic Antibody Mediated Rejection Gd.=Grade IFTA=Interstitial Fibrosis and Tubular Atrophy IVIG=Intravenous Immunoglobulin therapy.~Only 'for cause' biopsies were performed post-transplant; thus, it is possible for a participant to be included in the analysis population and not have a biopsy for this outcome measure." (NCT01436305)
Timeframe: Transplantation through last study visit (up to week 156)
Intervention | Biopsy (Number) |
---|
| Borderline rejection; IVIG and plasmapheresis | ACR Gd. IA + Chr. AMR + IFTA Gd. I; Pulse Steroids | ACR Gd. IA + IFTA Gd. II; Pulse Steroids | ACR Gd. IIB; ATG and Pulse Steroids | Borderline + IFTA Gd. I; with Pulse Steroids | ACR Gd. IA + IFTA Gd. I; Pulse Steroids | ACR Gd. IIA; Pulse Steroids | ACR Gd. IIA + IFTA Gd. I; ATG and Pulse Steroids | ACR Gd. IIB + IFTA Gd. I; ATG and Pulse Steroids |
---|
Induction: Alemtuzumab, Maintenance: MMF + Belatacept | 0 | 1 | 1 | 1 | 0 | 0 | 0 | 0 | 0 |
,Induction: Alemtuzumab, Maintenance: MMF + Tacrolimus | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
,Induction: Basiliximab, Maintenance: MMF + Belatacept + Tac | 0 | 0 | 0 | 0 | 1 | 1 | 1 | 2 | 1 |
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Change of Lung Function Measured by Forced Vital Capacity (FVC) Between Baseline and 24 Weeks
Forced vital capacity, or FVC, is the amount in liters of air that can be forcibly exhaled from the lungs after taking the deepest breath possible. It measures the effect that lung disease has on a person's ability to inhale and exhale. Higher values are better, and decreases over time can indicate disease progression. (NCT02370693)
Timeframe: 24 weeks
Intervention | Percentage of change in FVC %predicted (Mean) |
---|
Bortezomib Plus Mycophenolate Mofetil | 0.51 |
Placebo Plus Mycophenolate Mofetil | -0.69 |
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Change of Skin Fibrosis Measured by the Rodnan Skin Score Between Baseline and 24 Weeks
The Modified Rodnan Skin Score (mRSS) is a measure of skin thickness that sums individual scores of skin thickness (0 - No Thickening, 1 - Mild Thickening, 2 - Moderate Thickening, 3 - Severe Thickening) measured at 17 body sites to reach a total score (Range: 0 to 51, higher value representing thicker skin). The Score is used as a surrogate for disease activity and severity, and a worsening score over time is associated with worse outcomes. (NCT02370693)
Timeframe: 24 weeks
Intervention | Score on a Scale (Mean) |
---|
Bortezomib Plus Mycophenolate Mofetil | -3.00 |
Placebo Plus Mycophenolate Mofetil | -0.33 |
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Number of Participants With Serious Adverse Events
To assess the safety and tolerability of bortezomib with mycophenolate mofetil assessed by the incidence of serious adverse events. (NCT02370693)
Timeframe: First dosing day to last study visit day: Mean duration 8 months.
Intervention | Number of Participants with Serious Adve (Mean) |
---|
Bortezomib Plus Mycophenolate Mofetil | 0.33 |
Placebo Plus Mycophenolate Mofetil | 0.25 |
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Number of Patients That Engrafted Blood Counts by 30 Days After Transplant
Number of patients whose Absolute Neutrophil Count (ANC) recovered to >500 x10^3/uL for at least 3 consecutive days after transplant (NCT00827099)
Timeframe: Day 30
Intervention | participants (Number) |
---|
Umbilical Cord Blood Transplant | 5 |
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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
Intervention | Participants (Count of Participants) |
---|
Received Allogeneic HCT on Study | 0 |
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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
Intervention | days (Median) |
---|
Treatment (Chemotherapy, HCT) | 49 |
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Event-free Survival (EFS) Among Patients Who Received Early Transplant
Event-free survival among patients who received early allogeneic HCT on study as estimated with the Kaplan-Meier method. Events included death, relapse, and grade 3-4 acute graft vs host disease. (NCT02756572)
Timeframe: Up to 100 days post-transplant
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 91 |
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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
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 82 |
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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)
Intervention | years (Median) |
---|
Received Allogeneic HCT on Study | 55 |
Did Not Receive Allogeneic HCT on Study | 57 |
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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
Intervention | Percentage of participants (Number) |
---|
Did Not Receive Allogeneic HCT on Study | 75 |
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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
Intervention | Percentage of participants (Number) |
---|
Did Not Receive Allogeneic HCT on Study | 62 |
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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
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 91 |
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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
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 82 |
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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
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 91 |
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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
Intervention | Percentage of participants (Number) |
---|
Received Allogeneic HCT on Study | 82 |
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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
Intervention | Participants (Count of Participants) |
---|
| Enrollment PROs returned | Post G-CLAM PROs returned | Pre-HCT PROs returned | 6 months post-HCT PROs returned | 12 months post-HCT PROs returned |
---|
Treatment (Chemotherapy, HCT) | 27 | 23 | 8 | 4 | 3 |
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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)
Intervention | Participants (Count of Participants) |
---|
| Female | Male |
---|
Did Not Receive Allogeneic HCT on Study | 10 | 11 |
,Received Allogeneic HCT on Study | 8 | 1 |
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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
Intervention | Participants (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) | 9 | 21 |
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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
Intervention | Participants (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 Study | 6 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
| CR with MRD | CR without MRD | CRi with MRD | CRi without MRD | MLFS with MRD | MLFS without MRD | Relapse |
---|
Received Allogeneic HCT on Study | 0 | 7 | 0 | 1 | 0 | 0 | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
| CR with MRD | CR without MRD | CRi with MRD | CRi without MRD | MLFS with MRD | MLFS without MRD | Relapse |
---|
Received Allogeneic HCT on Study | 0 | 4 | 0 | 2 | 0 | 0 | 2 |
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# of Patients Developing Denovo Donor Specific Antibody (DSA) Post-transplant
Number of patients (%) with development of denovo DSA after transplant (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 5 |
Group B | 1 |
Group C | 5 |
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# Patients Experiencing a First Biopsy-proven ACR With Severity Banff > or = 2a Grade
Number of patients with their First BPACR with a Banff grade >= Banff 2a using the Banff 2007 classification system for biopsy grading. Banff grade 2a and above is considered severe cellular rejection and includes grades of Banff 2a, Banff 2b, and Banff 3. (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 5 |
Group B | 12 |
Group C | 0 |
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# Patients Experiencing a Graft Loss But Not Including Patients Who Died With Functioning Graft (Death-censored Graft Loss)
Number of patient who experienced Graft loss, not including (censored) patients who lost graft due to death (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 0 |
Group B | 1 |
Group C | 1 |
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# Patients With Composite Endpoint of Either Experiencing Death, Graft Loss, or eGFR < 45ml/Min at 24 Months
Composite Endpoint of number of patients who experienced either patient death, allograft loss, or had an eGFR < 45 ml/min/1.73m2 (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 11 |
Group B | 13 |
Group C | 21 |
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# Patients With Composite Endpoint of Experiencing Either Death, Graft Loss, or eGFR < 45ml/Min
Number of Patients that experienced patient Death or Graft Loss or had an estimated GFR (eGFR) (MDRD) < 45 mL/min (NCT01729494)
Timeframe: 12 months
Intervention | Participants (Count of Participants) |
---|
Group A | 9 |
Group B | 15 |
Group C | 14 |
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Biopsy Proven Acute Antibody Mediated Rejection
Incidence of patients experiencing a Biopsy proven acute antibody mediated rejection (BPAMR) (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 2 |
Group B | 2 |
Group C | 3 |
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Biopsy Proven Acute Cellular Rejection
Biopsy proven acute cellular rejection (BPACR) (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 14 |
Group B | 22 |
Group C | 2 |
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Biopsy Proven Acute Rejection
Incidence of all biopsy proven acute rejection whether clinically relevant or clinically silent. (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 20 |
Group B | 26 |
Group C | 7 |
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Biopsy Proven Mixed Acute Rejection
Incidence of patients experiencing a Biopsy proven acute cellular rejection with either DSA positive or C4d staining positive indicating antibody rejection (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 4 |
Group B | 2 |
Group C | 2 |
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Delayed Graft Function
Number of patients experiencing Delayed graft function (DGF) within first week after transplant. DGF is defined as need for dialysis within the first week after transplant. (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 3 |
Group B | 1 |
Group C | 5 |
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Discontinuation of Mycophenolate
Number of patients who were discontinued from mycophenolate treatment at 2 years (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 11 |
Group B | 9 |
Group C | 13 |
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Discontinuation of Study Treatment (Belatacept or Tacrolimus)
Number of patients who had to Discontinue study treatment (belatacept or tacrolimus) at 2 years (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 11 |
Group B | 9 |
Group C | 5 |
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eGFR (MRDRD) < 45 ml/Min/1.73m2
Patients with reduced Renal function measured by estimated GFR MDRD < 45 ml/min at 24 months (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 9 |
Group B | 8 |
Group C | 20 |
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Leukopenia (WBC < 2000/mm3)
Number of patients developing leukopenia defined as WBC < 2000/mm3 (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 22 |
Group B | 14 |
Group C | 15 |
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Mean eGFR (MDRD) (ml/Min/1.73m2)
Mean eGFR (MDRD) (ml/min/1.73m2) measured for all patients reaching 2 year endpoint (NCT01729494)
Timeframe: 24 months
Intervention | ml/min/1.73m2 (Mean) |
---|
Group A | 65.5 |
Group B | 65.3 |
Group C | 63.4 |
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New Onset Diabetes After Transplantation (NODAT)
Number of patients developing New Onset Diabetes Mellitus after Transplant by one of 5 definitions; only patients without preexisting diabetes were included (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 11 |
Group B | 5 |
Group C | 12 |
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Patient Death
Number of Patients who experienced death, all causes (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 2 |
Group B | 4 |
Group C | 1 |
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Proteinuria UPC Ratio > 0.8
Number of Patients with a Urine protein/creatinine (UPC) ratio > 0.8 (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 11 |
Group B | 5 |
Group C | 21 |
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Requirement of T-cell Depleting Therapy for Biopsy Proven Acute Rejection (BPAR)
Number of patients requiring anti-lymphocyte therapy for the treatment of BPAR rejection (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 8 |
Group B | 15 |
Group C | 0 |
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Steroid Therapy
Number of patients on treatment with corticosteroids at 2 years (NCT01729494)
Timeframe: 24 months
Intervention | Participants (Count of Participants) |
---|
Group A | 16 |
Group B | 14 |
Group C | 9 |
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Time to First BPAR
Mean Time to first episode of BPAR (days) (NCT01729494)
Timeframe: 24 months
Intervention | days (Mean) |
---|
Group A | 229 |
Group B | 131.6 |
Group C | 159.6 |
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Duration of Prednisone Maintenance Dosing
The duration of prednisone maintenance dosing was defined as the number of days that subjects maintained a prednisone dose of not more than 10 mg/day in the absence of new persistent lesions. (NCT00683930)
Timeframe: 52 weeks
Intervention | Days (Median) |
---|
Placebo | 136.5 |
MMF 2 g/Day and MMF 3 g/Day Groups Combined | 186.0 |
Mycophenolate Mofetil 2 g/Day | 185.0 |
Mycophenolate Mofetil 3 g/Day | 187.0 |
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Percentage of Patients Achieving Responder Status at Week 52
The proportion of subjects achieving responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52) in the two active treatment groups combined (2 g/day and 3 g/day mycophenolate mofetil) compared with the placebo group (NCT00683930)
Timeframe: 52 weeks
Intervention | Percentage of Participants (Number) |
---|
Placebo | 63.9 |
MMF 2 g/Day and MMF 3 g/Day Groups Combined | 69.0 |
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Time to Initial Response
Time to initial response defined as the time that the subject first demonstrated responder status (defined as no new persistent lesions and prednisone dose of not more than 10 mg/day from Week 48 until study termination at Week 52) (NCT00683930)
Timeframe: up to 52 weeks
Intervention | Weeks (Median) |
---|
Placebo | 31.3 |
MMF 2 g/Day and MMF 3 g/Day Groups Combined | 24.1 |
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Time to Sustained Response
Time to sustained response is defined as the week the subject first demonstrates both of the conditions of responder status provided the conditions are maintained through to study termination at Week 52. If a subject does not have a sustained response, time to sustained response is censored on the last day of the study. (NCT00683930)
Timeframe: up to 52 weeks
Intervention | Weeks (Median) |
---|
Placebo | 46.0 |
MMF 2 g/Day and MMF 3 g/Day Groups Combined | 32.1 |
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Number of Non-relapse Participant Mortalities
Defined as death in any patient for whom there has not been a diagnosis of relapse or disease progression. (NCT00789776)
Timeframe: Day 200
Intervention | Participants (Count of Participants) |
---|
Dose 1 (2.5 x 10^6/kg NK Cells) | 0 |
Dose 2 (5.0 x 10^6/kg NK Cells) | 0 |
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Number of Participants Who Experienced Chronic Extensive GVHD
Number of patients who developed chronic extensive GVHD post-transplant. The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD. (NCT00789776)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
Dose 1 (2.5 x 10^6/kg NK Cells) | 2 |
Dose 2 (5.0 x 10^6/kg NK Cells) | 3 |
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Number of Participants Who Experienced Graft Failure
Graft failure is defined as grade IV thrombocytopenia and neutropenia after Day +21 that lasts >2 weeks and is refractory to growth factor support. (NCT00789776)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Dose 1 (2.5 x 10^6/kg NK Cells) | 0 |
Dose 2 (5.0 x 10^6/kg NK Cells) | 4 |
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Number of Participants With Dose Limiting Toxicities
Defined as having at least one of the following adverse events, independent of the attribution to the Natural Killer cell infusion: grade IV infusional toxicity (based on the Adapted Common Toxicity Criteria); grade IV regimen-related toxicity (based on Adapted Common Toxicity Criteria); grade IV acute Graft-Versus-Host Disease; non-relapse mortality. (NCT00789776)
Timeframe: Day 35 (28 days after NK cell infusion)
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Dose 1 (2.5 x 10^6/kg NK Cells) | 5 |
Dose 2 (5.0 x 10^6/kg NK Cells) | 25 |
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Immune Reconstitution Efficacy - Naive CD4 T Cells
The development of total naive CD4 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | cells/mm3 (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 156 |
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Immune Reconstitution Efficacy - Naive CD8 T Cells
The development of total naive CD8 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | cells/mm3 (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 37 |
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Immune Reconstitution Efficacy - Response to Mitogens
Measurement of the T cell proliferative response to the mitogen phytohemagglutin (PHA). (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | counts per minute (cpm) (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 139189 |
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Immune Reconstitution Efficacy - Total CD3 T Cells
The development of total CD3 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | cells/mm3 (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 726 |
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Immune Reconstitution Efficacy - Total CD4 T Cells
The development of total CD4 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | cells/mm3 (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 593 |
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Immune Reconstitution Efficacy - Total CD8 T Cells
The development of total CD8 T cells at one year as measured using flow cytometry (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | cells/mm3 (Median) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 145 |
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Survival at 1 Year Post-CTTI
Survival at 1 year post cultured thymus tissue implantation was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time. (NCT00579527)
Timeframe: 1 year post-CTTI
Intervention | % of participants who survive to 1 year (Number) |
---|
Cultured Thymus Tissue With Immunosuppression | 71 |
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Survival at 2 Years Post-CTTI
Survival at 2 years post cultured thymus tissue implantation was assessed using the Kaplan Meier Estimated Survival. This mathematical function estimates the survival for a certain length of time. (NCT00579527)
Timeframe: 2 years post-CTTI
Intervention | % of participants who survive to 2 years (Number) |
---|
Cultured Thymus Tissue Implantation With Immunosuppression | 71 |
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Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During Year 1 of the Double-blind Period
"Adjusted mean change from baseline in British Isles Lupus Assessment Group (BILAG) score over time during Year 1 of the double-blind period based on a repeated measure mixed model and presented at each visit in the first 12-month of the double-blind period. BILAG index measures disease activity in different organs/systems separately. BILAG score is calculated for each of 9 systems depending on the clinical features present and whether they are new (4 points), worse (3 points), the same (2 points), improving (1 point) or not present (0 points) in the last 4 weeks compared with previously. BILAG A represents the presence of serious features of lupus. BILAG B represents more moderate features of the disease. BILAG C includes only mild symptomatic features. BILAG D represents prior activity with no current symptoms due to active lupus. BILAG E represents an organ that has never been involved. Overall BILAG score ranges from 0-108, with higher scores reflecting a worse outcome." (NCT01714817)
Timeframe: Day 1 to Day 365
Intervention | Scores on a Scale (Mean) |
---|
Abatacept IV | -8.22 |
Placebo IV | -7.60 |
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Adjusted Mean Change From Baseline in UPCR at Day 365 of the Double-blind Period in Overall Population
Adjusted Mean Change from Baseline in Urine protein/creatinine ratio (UPCR) at Day 365 of the double-blind period in the overall population (NCT01714817)
Timeframe: Day 1 and Day 365
Intervention | UPCR (mg/mg) (Mean) |
---|
Abatacept IV | -2.99 |
Placebo IV | -2.90 |
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Adjusted Mean Change From Baseline in Urine Protein/Creatinine Ratio (UPCR) at Day 365 of the Double-blind Period in Nephrotic Participants
Adjusted Mean Change from Baseline in UPCR at Day 365 of the double-blind period in nephrotic participants (NCT01714817)
Timeframe: Baseline and Day 365
Intervention | UPCR (mg/mg) (Mean) |
---|
Abatacept IV | -5.01 |
Placebo IV | -4.84 |
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AUC (TAU): Area Under the Serum Concentration Time Curve Over a Dosing Interval
AUC (TAU): Area under the serum concentration time curve over a dosing interval between Days 337 to 365. (NCT01714817)
Timeframe: Days 337 to 365
Intervention | ug*h/mL (Geometric Mean) |
---|
Abatacept IV | 36480.24 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Percentage of Blood)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | Percentage (Mean) |
---|
Abatacept IV | 0.0328 |
Placebo IV | 0.0325 |
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Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 365 of the Double-blind Period
Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 365
Intervention | Percentage (Number) |
---|
Abatacept IV | 27 |
Placebo IV | 29.5 |
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Percentage of Nephrotic Participants in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period
Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 729
Intervention | Percentage (Number) |
---|
Abatacept IV | 50.0 |
Placebo IV | 49.0 |
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Percentage of Participants in Complete Renal Response (CR) of Lupus Glomerulonephritis at Day 365 of the Double-blind Period
Number of participants achieving CR was divided by the total number of participants in that arm and expressed as a percentage. CR defined as: eGFR is normal or no <85% of the baseline; eGFR based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equiv. for at least 28 days prior to assessment. Participants with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as having achieved CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use (Yes/No), race (Asian/ Black/Caucasian/Other) and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 365
Intervention | Percentage (Number) |
---|
Abatacept IV | 35.1 |
Placebo IV | 33.5 |
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Percentage of Participants in Overall Population in Complete Renal Response of Lupus Glomerulonephritis at Day 729 of the Double-blind Period
Number of participants achieving CR was divided by total participants in that arm, expressed as a percentage. CR is defined the following criteria: eGFR is normal or no <85% of the baseline value; eGFR is based on mean creatinine value from day 358 and 365. Proteinuria: UPCR<0.5 mg/mg. Urine sediment: No cellular casts. Corticosteroid dose: Daily dose must be no >10 mg prednisone or equivalent for at least 28 days prior. Subjects with >10mg/day prednisone or equivalent for non-renal disease within 28 days prior to day 365 will be imputed as CR if the following are true: Met all criteria for CR at day 337 and all criteria for CR except corticosteroid dose at day 365; Investigator confirms increase in steroid dose is not related to renal disease. Adjusted odds ratio is estimated from logistic regression model which includes treatment group, baseline ACEi/ARBs use, race and baseline UPCR as a continuous variable. (NCT01714817)
Timeframe: Day 729
Intervention | Percentage (Number) |
---|
Abatacept IV | 61.9 |
Placebo IV | 52.7 |
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Adjusted Mean Change From Baseline in Disease Activity as Measured by BILAG 2004 Over Time During the Double-blind Period
"BILAG index measures and reports disease activity in different organs/systems separately. The BILAG score is calculated for each of nine systems depending on the clinical features present and whether they are new (4 points), worse (3 points), the same (2 points), improving (1 point) or not present (0 points) in the last 4 weeks compared with previously. A BILAG A represents the presence of one or more serious features of lupus. A BILAG B represents more moderate features of the disease. A BILAG C includes only mild symptomatic features. A BILAG D represents only prior activity with no current symptoms due to active lupus. A BILAG E represents an organ that has never been involved. Overall BILAG score ranges from 0-108, with higher scores reflecting a worse outcome." (NCT01714817)
Timeframe: Day 1 to Day 729; Day 365 to Day 729
Intervention | Scores on a Scale (Mean) |
---|
| Day 1 to Day 729 | Day 365 to Day 729 |
---|
Abatacept IV | -9.31 | -0.95 |
,Placebo IV | -8.53 | -0.40 |
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Adjusted Mean Change From Baseline in eGFR Over Time
Estimated glomerular filtration rate(eGFR), will be calculated by the CKD-EPI formula shown below.50 eGFR is expressed as mL/min per 1.73m2. For the purpose of this study lower limit of normal eGFR is defined as 90mL/min per 1.73m2 eGFR = 141 X min (Scr/k, 1)α X max (Scr/k, 1)-1.209 X 0.993Age X (1.018 [if female]) X (1.159 [if black]) Where Scr is serum creatinine (mg/dL), k is 0.7 for females and 0.9 for males, α is -0.329 for females and -0.411 for males, min indicates the minimum of Scr/k or 1, and max indicates the maximum of Scr/k or 1, age in years. (NCT01714817)
Timeframe: Day 365, Day 729
Intervention | mL/min per 1.73m2 (Mean) |
---|
| Day 365 | Day 729 |
---|
Abatacept IV | 6.85 | 7.20 |
,Placebo IV | 5.85 | 7.91 |
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Adjusted Mean Change From Baseline in UPCR Over Time
A repeated measure mixed model that included the baseline UPCR value, randomization stratification factors, time, and time by treatment interaction as fixed effects and subject as a random effect was used. (NCT01714817)
Timeframe: Day 365; Day 729, includes data up to July 1st 2017 when double-blind therapy ended
Intervention | UPCR (mg/mg) (Mean) |
---|
| Day 365 | Day 729 |
---|
Abatacept IV | -2.95 | -3.13 |
,Placebo IV | -2.68 | -2.72 |
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Cmax: Maximum Observed Serum Concentration Following Participants Receiving Active Abatacept IV
Cmax: Maximum observed serum concentration following participants receiving active abatacept IV (NCT01714817)
Timeframe: at 1 hour post Day 1 dose and 30 minutes post Day 337 dose
Intervention | ug/mL (Geometric Mean) |
---|
| Day 1 | Day 337 |
---|
Abatacept IV | 527.43 | 203.51 |
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Cmin (ug/mL): Trough Level Serum Concentration of Abatacept Prior to the Administration of the IV Infusion
Trough level serum concentration of abatacept prior to the administration of the IV infusion on Days 1 to 365 (NCT01714817)
Timeframe: Days 1 to 365
Intervention | ug/mL (Geometric Mean) |
---|
| Day 15 | Day 29 | Day 57 | Day 85 | Day 113 | Day 169 | Day 281 | Day 337 | Day 365 |
---|
Abatacept IV | 69.97 | 90.46 | 36.43 | 34.46 | 16.42 | 13.98 | 14.44 | 14.99 | 13.62 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (g/L)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | g/L (Mean) |
---|
| ALBUMIN (g/L) | HEMOGLOBIN (g/L) | PROTEIN, TOTAL (g/L) |
---|
Abatacept IV | 9.2 | 8.8 | 9.9 |
,Placebo IV | 8.1 | 9.0 | 10.1 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (mmol/L)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | mmol/L (Mean) |
---|
| BLOOD UREA NITROGEN (mmol/L) | CALCIUM, TOTAL (mmol/L) | CHLORIDE, SERUM (mmol/L) | GLUCOSE, SERUM (mmol/L) | PHOSPHORUS, INORGANIC (mmol/L) | POTASSIUM, SERUM (mmol/L) | SODIUM, SERUM (mmol/L) |
---|
Abatacept IV | -2.31 | 0.097 | -1.1 | -0.23 | -0.077 | -0.02 | -0.2 |
,Placebo IV | -2.25 | 0.108 | -0.5 | -0.58 | -0.037 | -0.10 | -0.5 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (U/L)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | U/L (Mean) |
---|
| ALANINE AMINOTRANSFERASE (ALT) (U/L) | ALKALINE PHOSPHATASE (ALP) (U/L) | ASPARTATE AMINOTRANSFERASE (AST) (U/L) | G-GLUTAMYL TRANSFERASE (GGT) (U/L) |
---|
Abatacept IV | -2.2 | 8.2 | 0.3 | -5.3 |
,Placebo IV | -3.4 | 11.7 | 0.3 | -4.1 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (Umol/L)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | umol/L (Mean) |
---|
| BILIRUBIN, TOTAL (umol/L) | CREATININE (umol/L) |
---|
Abatacept IV | 1.77 | -5.6 |
,Placebo IV | 1.00 | -6.2 |
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Mean Change From Baseline in Laboratory Analytes During the Double-blind Period (x10^9 Cells/L)
"Laboratory assessments were analyzed centrally, with the exception of pregnancy testing. Blood draws and urine collections were performed at visits specified in the protocol.~Change from Baseline = Post-baseline - Baseline value." (NCT01714817)
Timeframe: Day 729
Intervention | x10^9 cells/L (Mean) |
---|
| EOSINOPHILS (ABSOLUTE) (x10^9 cells/L) | LYMPHOCYTES (ABSOLUTE) (x10^9 cells/L) | MONOCYTES (ABSOLUTE) (x10^9 cells/L) | NEUTROPHILS (ABSOLUTE) (x10^9 cells/L) | PLATELET COUNT (x10^9 cells/L) |
---|
Abatacept IV | 0.034 | 0.141 | -0.018 | -2.259 | -4.9 |
,Placebo IV | 0.010 | -0.149 | -0.050 | -2.289 | -9.1 |
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Number of Participants With Abatacept Induced Antibody Response Over Time in the Double-blind Period
Participants who experienced a positive antibody response relative to baseline (ECL Assay) (NCT01714817)
Timeframe: Day 365, Day 729
Intervention | Participants (Number) |
---|
| Day 365, overall |
---|
Abatacept IV | 7 |
,Placebo IV | 9 |
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Number of Participants With Any Adverse Events (AEs) During Year 1 of the Double-blind Period
All AEs were coded and grouped into preferred terms (PT) by system organ class (SOC), using the Medical Dictionary for Regulatory Activities (MedDRA, version 21.0). Investigators determined the intensity of each AE as mild, moderate, severe, or very severe and assessed the relationship to study drug. (NCT01714817)
Timeframe: From Day 1 up to 56 days post last dose in Year 1 of the double-blind period
Intervention | Participants (Number) |
---|
| Participants with Adverse Events | Participants with Serious Adverse Events | Participants with infection Adverse Events | Participants with malignancies | Participants with autoimmune events | Participants with peri-infusional Adverse Events | Participants with acute infusional Adverse Events |
---|
Abatacept IV | 188 | 49 | 150 | 2 | 10 | 7 | 2 |
,Placebo IV | 194 | 39 | 147 | 1 | 9 | 9 | 4 |
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Number of Participants With Any Adverse Events (AEs) During Year 2 of the Double-blind Period and Long-term Extension
All AEs were coded and grouped into preferred terms (PT) by system organ class (SOC), using the Medical Dictionary for Regulatory Activities (MedDRA, version 21.0). Investigators determined the intensity of each AE as mild, moderate, severe, or very severe and assessed the relationship to study drug. (NCT01714817)
Timeframe: From the first dose in Year 2 of the double-blind period up to 56 days post last dose
Intervention | Participants (Number) |
---|
| Participants with Adverse Events | Participants with Serious Adverse Events | Participants with infection Adverse Events | Participants with malignancies | Participants with autoimmune events |
---|
Abatacept IV | 127 | 15 | 100 | 0 | 7 |
,Placebo IV | 137 | 25 | 107 | 1 | 11 |
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Number of Participants With Marked Electrolyte Laboratory Abnormalities During Year 1 of the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value SODIUM, SERUM mmol/L 4.0 NA <0.95X LLN OR >1.05X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.05X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier
Intervention | participants (Number) |
---|
| SODIUM, SERUM, low | SODIUM, SERUM, high | POTASSIUM, SERUM, low | POTASSIUM, SERUM, high | CHLORIDE, SERUM, low | CHLORIDE, SERUM, high | CALCIUM, TOTAL, low | CALCIUM, TOTAL, high | PHOSPHORUS, INORGANIC, low | PHOSPHORUS, INORGANIC, high |
---|
Abatacept IV | 1 | 2 | 3 | 7 | 0 | 0 | 1 | 2 | 9 | 13 |
,Placebo IV | 1 | 2 | 5 | 7 | 1 | 0 | 1 | 0 | 7 | 13 |
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Number of Participants With Marked Electrolyte Laboratory Abnormalities in the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value SODIUM, SERUM mmol/L 4.0 NA <0.95X LLN OR >1.05X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.05X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR 1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.1X PRE RX OR NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | participants (Number) |
---|
| SODIUM, SERUM, low | SODIUM, SERUM, high | POTASSIUM, SERUM, low | POTASSIUM, SERUM, high | CHLORIDE, SERUM, low | CHLORIDE, SERUM, high | CALCIUM, TOTAL, low | CALCIUM, TOTAL, high | PHOSPHORUS, INORGANIC, low | PHOSPHORUS, INORGANIC, high |
---|
Abatacept IV | 0 | 0 | 2 | 3 | 0 | 0 | 0 | 1 | 3 | 6 |
,Placebo IV | 0 | 1 | 2 | 2 | 1 | 0 | 0 | 0 | 4 | 3 |
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Number of Participants With Marked Hematology Laboratory Abnormalities During Year 1 of the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value HEMOGLOBIN g/L 4.0 HB >3 G/DL DECREASE FROM PRE RX HEMATOCRIT vol 6.3 HCT <0.75X PRE RX ERYTHROCYTES x10*12 c/L 5.2 RBC <0.75X PRE RX PLATELET COUNT x10*9 c/L 5.0 PLAT <0.67X LLN OR >1.5X ULN, OR IF PRE RX1.25X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.2X PRE RX OR .750 X10*3 c/uL BASOPHILS (ABSOLUTE) x10*9 c/L 8.3 BASOA IF VALUE > 400/MM3 MONOCYTES (ABSOLUTE) x10*9 c/L 8.3 MONOA IF VALUE > 2000/MM3 LYMPHOCYTES (ABSOLUTE) x10*9 c/L 8.3 LYMPA IF VALUE < .750 X10*3 c/uL OR IF VALUE > 7.50 X10*3 c/uL~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier
Intervention | participants (Number) |
---|
| HEMOGLOBIN, low | HEMOGLOBIN, high | HEMATOCRIT, low | HEMATOCRIT, high | ERYTHROCYTES, low | ERYTHROCYTES, high | PLATELET COUNT, low | PLATELET COUNT, high | LEUKOCYTES, low | LEUKOCYTES, high | EOSINOPHILS (ABSOLUTE), low | EOSINOPHILS (ABSOLUTE), high | BASOPHILS (ABSOLUTE), low | BASOPHILS (ABSOLUTE), high | MONOCYTES (ABSOLUTE), low | MONOCYTES (ABSOLUTE), high | LYMPHOCYTES (ABSOLUTE), low | LYMPHOCYTES (ABSOLUTE), high |
---|
Abatacept IV | 6 | NA | 12 | NA | 7 | NA | 4 | 0 | 35 | 29 | NA | 2 | NA | 1 | NA | 0 | 81 | 1 |
,Placebo IV | 10 | NA | 12 | NA | 10 | NA | 3 | 0 | 21 | 25 | NA | 6 | NA | 1 | NA | 1 | 104 | 2 |
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Number of Participants With Marked Hematology Laboratory Abnormalities in the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value HEMOGLOBIN g/L 4.0 HB >3 G/DL DECREASE FROM PRE RX HEMATOCRIT vol 6.3 HCT <0.75X PRE RX ERYTHROCYTES x10*12 c/L 5.2 RBC <0.75X PRE RX PLATELET COUNT x10*9 c/L 5.0 PLAT <0.67X LLN OR >1.5X ULN, OR IF PRE RX1.25X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.2X PRE RX OR .750 X10*3 c/uL BASOPHILS (ABSOLUTE) x10*9 c/L 8.3 BASOA IF VALUE > 400/MM3 MONOCYTES (ABSOLUTE) x10*9 c/L 8.3 MONOA IF VALUE > 2000/MM3 LYMPHOCYTES (ABSOLUTE) x10*9 c/L 8.3 LYMPA IF VALUE < .750 X10*3 c/uL OR IF VALUE > 7.50 X10*3 c/uL~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | participants (Number) |
---|
| HEMOGLOBIN, low | HEMOGLOBIN, high | HEMATOCRIT, low | HEMATOCRIT, high | ERYTHROCYTES, low | ERYTHROCYTES, high | PLATELET COUNT, low | PLATELET COUNT, high | LEUKOCYTES, low | LEUKOCYTES, high | EOSINOPHILS (ABSOLUTE), low | EOSINOPHILS (ABSOLUTE), high | BASOPHILS (ABSOLUTE), low | BASOPHILS (ABSOLUTE), high | MONOCYTES (ABSOLUTE), low | MONOCYTES (ABSOLUTE), high | LYMPHOCYTES (ABSOLUTE), low | LYMPHOCYTES (ABSOLUTE), high |
---|
Abatacept IV | 8 | NA | 6 | NA | 4 | NA | 2 | 1 | 19 | 3 | NA | 11 | NA | 0 | NA | 0 | 43 | 0 |
,Placebo IV | 9 | NA | 2 | NA | 2 | NA | 4 | 0 | 24 | 5 | NA | 6 | NA | 0 | NA | 0 | 62 | 0 |
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Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities During Year 1 of the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value ALKALINE PHOSPHATASE (ALP) U/L 5.0 ALP >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX ASPARTATE AMINOTRANSFERASE (AST) U/L 5.0 AST >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX ALANINE AMINOTRANSFERASE (ALT) U/L 5.0 ALT >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX G-GLUTAMYL TRANSFERASE (GGT) U/L 5.0 GGT >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX BILIRUBIN, TOTAL umol/L 5.1 TBILI >2X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX BILIRUBIN, DIRECT umol/L 5.1 DBILI >1.5X ULN, OR IF PRE RX>ULN THEN USE >2X PRE RX BLOOD UREA NITROGEN mmol/L 5.1 BUN >2X PRE RX CREATININE umol/L 5.0 CREAT >1.5X PRE RX~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier
Intervention | participants (Number) |
---|
| ALKALINE PHOSPHATASE (ALP), low | ALKALINE PHOSPHATASE (ALP), high | ASPARTATE AMINOTRANSFERASE (AST), low | ASPARTATE AMINOTRANSFERASE (AST), high | ALANINE AMINOTRANSFERASE (ALT), low | ALANINE AMINOTRANSFERASE (ALT), high | G-GLUTAMYL TRANSFERASE (GGT), low | G-GLUTAMYL TRANSFERASE (GGT), high | BILIRUBIN, TOTAL, low | BILIRUBIN, TOTAL, high | BILIRUBIN, DIRECT, low | BILIRUBIN, DIRECT, high | BLOOD UREA NITROGEN, low | BLOOD UREA NITROGEN, high | CREATININE, low | CREATININE, high |
---|
Abatacept IV | NA | 1 | NA | 5 | NA | 8 | NA | 17 | NA | 0 | NA | 0 | NA | 20 | NA | 24 |
,Placebo IV | NA | 1 | NA | 0 | NA | 2 | NA | 15 | NA | 0 | NA | 0 | NA | 12 | NA | 20 |
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Number of Participants With Marked Liver and Kidney Function Laboratory Abnormalities in the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value ALKALINE PHOSPHATASE (ALP) U/L 5.0 ALP >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX ASPARTATE AMINOTRANSFERASE (AST) U/L 5.0 AST >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX ALANINE AMINOTRANSFERASE (ALT) U/L 5.0 ALT >3X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX G-GLUTAMYL TRANSFERASE (GGT) U/L 5.0 GGT >2X ULN, OR IF PRE RX>ULN THEN USE >3X PRE RX BILIRUBIN, TOTAL umol/L 5.1 TBILI >2X ULN, OR IF PRE RX>ULN THEN USE >4X PRE RX BILIRUBIN, DIRECT umol/L 5.1 DBILI >1.5X ULN, OR IF PRE RX>ULN THEN USE >2X PRE RX BLOOD UREA NITROGEN mmol/L 5.1 BUN >2X PRE RX CREATININE umol/L 5.0 CREAT >1.5X PRE RX~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | participants (Number) |
---|
| ALKALINE PHOSPHATASE (ALP), low | ALKALINE PHOSPHATASE (ALP), high | ASPARTATE AMINOTRANSFERASE (AST), low | ASPARTATE AMINOTRANSFERASE (AST), high | ALANINE AMINOTRANSFERASE (ALT), low | ALANINE AMINOTRANSFERASE (ALT), high | G-GLUTAMYL TRANSFERASE (GGT), low | G-GLUTAMYL TRANSFERASE (GGT), high | BILIRUBIN, TOTAL, low | BILIRUBIN, TOTAL, high | BILIRUBIN, DIRECT, low | BILIRUBIN, DIRECT, high | BLOOD UREA NITROGEN, low | BLOOD UREA NITROGEN, high | CREATININE, low | CREATININE, high |
---|
Abatacept IV | NA | 4 | NA | 2 | NA | 4 | NA | 17 | NA | 0 | NA | 1 | NA | 10 | NA | 17 |
,Placebo IV | NA | 0 | NA | 3 | NA | 3 | NA | 11 | NA | 0 | NA | 0 | NA | 9 | NA | 16 |
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Number of Participants With Marked Urinalysis Laboratory Abnormalities During Year 1 of the Double Blind Period
LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value PROTEIN, URINE Unknown UPRO IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 GLUCOSE, URINE N/A UGLU IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 BLOOD, URINE N/A UBLD IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 RBC, URINE hpf 5.0 URBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 WBC, URINE hpf 5.0 UWBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier
Intervention | participants (Number) |
---|
| PROTEIN, URINE, low | PROTEIN, URINE, high | GLUCOSE, URINE, low | GLUCOSE, URINE, high | BLOOD, URINE, low | BLOOD, URINE, high | Red blood cells (RBC), URINE, low | Red blood cells (RBC), URINE, high | White blood cells (WBC), URINE, low | White blood cells (WBC), URINE, high |
---|
Abatacept IV | NA | 0 | NA | 0 | NA | 0 | NA | 93 | NA | 91 |
,Placebo IV | NA | 0 | NA | 0 | NA | 0 | NA | 103 | NA | 98 |
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Number of Participants With Marked Urinalysis Laboratory Abnormalities in the Double Blind Period
LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value PROTEIN, URINE Unknown UPRO IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 GLUCOSE, URINE N/A UGLU IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 BLOOD, URINE N/A UBLD IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 RBC, URINE hpf 5.0 URBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 WBC, URINE hpf 5.0 UWBC IF MISSING PRE THEN USE >=2, OR IF VALUE >=4, OR IF PRE RX =0 OR 0.5 THEN USE >=2, OR IF PRE RX =1 THEN USE >=3, OR IF PRE RX =2 OR 3 THEN USE >=4 (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | participants (Number) |
---|
| PROTEIN, URINE, low | PROTEIN, URINE, high | GLUCOSE, URINE, low | GLUCOSE, URINE, high | BLOOD, URINE, low | BLOOD, URINE, high | Red blood cells (RBC), URINE, low | Red blood cells (RBC), URINE, high | White blood cells (WBC), URINE, low | White blood cells (WBC), URINE, high |
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Abatacept IV | NA | 0 | NA | 0 | NA | 0 | NA | 58 | NA | 46 |
,Placebo IV | NA | 0 | NA | 0 | NA | 0 | NA | 55 | NA | 59 |
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Number of Participants With Other Marked Chemistry Laboratory Abnormalities During Year 1 of the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value CALCIUM, TOTAL mmol/L 5.2 CA <0.8X LLN OR >1.2X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.25X PRE RX OR 1.25X ULN, OR IF PRE RXULN GLUCOSE, SERUM mmol/L 4.1 GLUC <65 mg/dL, OR >220 mg/dL PROTEIN, TOTAL g/L 5.0 TPRO <0.9X LLN OR >1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE 1.1X PRE RX OR 2X PRE R~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 365; includes data up to 56 days post last dose in year 1 of double-blind period or first dose date in the year 2 of double-blind period, whichever is earlier
Intervention | participants (Number) |
---|
| GLUCOSE, SERUM, low | GLUCOSE, SERUM, high | PROTEIN, TOTAL, low | PROTEIN, TOTAL, high | ALBUMIN, low | ALBUMIN, high |
---|
Abatacept IV | 33 | 10 | 44 | 0 | 10 | NA |
,Placebo IV | 29 | 5 | 26 | 1 | 11 | NA |
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Number of Participants With Other Marked Chemistry Laboratory Abnormalities in the Double Blind Period
"LLN= Lower limit of normals ULN= Upper limit of normals Pre RX = Baseline value CALCIUM, TOTAL mmol/L 5.2 CA <0.8X LLN OR >1.2X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE >1.25X PRE RX OR 1.25X ULN, OR IF PRE RXULN GLUCOSE, SERUM mmol/L 4.1 GLUC <65 mg/dL, OR >220 mg/dL PROTEIN, TOTAL g/L 5.0 TPRO <0.9X LLN OR >1.1X ULN, OR IF PRE RXULN, OR IF PRE RX>ULN THEN USE 1.1X PRE RX OR 2X PRE R~N = the number of participants with at least 1 on treatment lab result for each analyte" (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | participants (Number) |
---|
| GLUCOSE, SERUM, low | GLUCOSE, SERUM, high | GLUCOSE, FASTING SERUM, low | GLUCOSE, FASTING SERUM, high | PROTEIN, TOTAL, low | PROTEIN, TOTAL, high | ALBUMIN, low | ALBUMIN, high |
---|
Abatacept IV | 15 | 3 | 3 | 3 | 10 | 2 | 4 | NA |
,Placebo IV | 24 | 2 | 1 | 1 | 7 | 3 | 5 | NA |
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Number of Participants With Sustained Change From Higher Level of Response to no Response During the Double-blind Period
Sustained change to no response is defined as going from CR (or PR) to NR and remaining in NR for at least 2 consecutive visits; visits should be approximately 4 weeks apart. This analysis will be based on time from response CR (or PR) to the first visit in which the no response (NR) was achieved and sustained to the next visit. (NCT01714817)
Timeframe: Day 365, Day 729
Intervention | Participants (Number) |
---|
| Day 365 | Day 729 |
---|
Abatacept IV | 5 | 52 |
,Placebo IV | 3 | 56 |
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Percentage of Participants in Treatment Failure Over Time During the Double-blind Period
"Lupus treatment failure is defined as any of the following: Death, unless due to physical trauma or violence; Renal Flare; sustained doubling of creatinine from baseline (greater of Screening or Study Day 1 value); initiation of rescue therapy for treatment of active lupus nephritis after Study Week 20.~Overall treatment failure is defined as lupus treatment failure plus discontinuation of study drug for any reason except death due to physical trauma or violence, pregnancy or administrative decision by Sponsor." (NCT01714817)
Timeframe: Day 365, Day 729
Intervention | Percentage (Number) |
---|
| Lupus treatment failure (LTF) - Day 365 | Overall treatment failure (OTF) - Day 365 | LTF - Day 729 | OTF - Day 729 |
---|
Abatacept IV | 3.5 | 4.5 | 4.5 | 5.2 |
,Placebo IV | 4.4 | 4.9 | 5.3 | 8.4 |
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Percentage of Participants With Ranked Outcome of Complete Renal Response, Partial Renal Response (PR), and No Renal Response (NR) During the Double-blind Period
Complete Renal Response or Complete Response (CR): defined as meeting ALL of the following criteria: eGFR normal OR no less than 85% of the baseline value; UPCR < 0.5; Urine sediment: No cellular casts; Daily corticosteroid dose must be no greater than 10 mg prednisone or equivalent for at least 28 days prior to assessment. Partial Renal Response or Partial Response (PR): defined as meeting ALL of the following criteria: Participant does not meet criteria for CR; eGFR no less than 85% of the lesser of the values at screening or randomization (Day 1); UPCR < 0.5 OR 50% reduced from baseline and < 1 if baseline value was < 3, OR 50% reduced from baseline and < 3 if baseline value was greater than or equal to 3; Urine sediment: no cellular casts; daily corticosteroid dose no greater than 10 mg/day prednisone or prednisone equivalent for at least 28 days prior to assessment. No Renal Response or No Response (NR): defined as not meeting criteria for CR or PR or withdrawn (NCT01714817)
Timeframe: Day 365, Day 729
Intervention | Percentage (Number) |
---|
| CR - Day 365 | PR - Day 365 | NR - Day 365 | CR - Day 729 | PR - Day 729 | NR - Day 729 |
---|
Abatacept IV | 35.1 | 20.8 | 44.1 | 60.7 | 25.9 | 13.4 |
,Placebo IV | 33.5 | 21.7 | 44.8 | 53.6 | 22.7 | 23.6 |
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Summary Statistics for Diastolic Blood Pressure
Summary statistics for diastolic blood pressure (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | mmHg (Mean) |
---|
| Day 1, end of observation | Day 365, end of observation | Day 729, end of observation |
---|
Abatacept IV | 76.5 | 73.5 | 67.5 |
,Placebo IV | 77.0 | 77.5 | 72.7 |
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Summary Statistics for Heart Rate
Summary statistics for Heart Rate (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | beats per minute (Mean) |
---|
| Day 1, end of observation | Day 365, end of observation | Day 729, end of observation |
---|
Abatacept IV | 80.6 | 78.5 | 76.2 |
,Placebo IV | 81.4 | 70.0 | 76.7 |
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Summary Statistics for Systolic Blood Pressure
Summary statistics for systolic blood pressure (NCT01714817)
Timeframe: Day 1 to Day 729
Intervention | mmHg (Mean) |
---|
| Day 1, end of observation | Day 365, end of observation | Day 729, end of observation |
---|
Abatacept IV | 122.0 | 112.3 | 108.6 |
,Placebo IV | 122.6 | 115.0 | 114.2 |
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AUC0-inf - Area Under the Concentration-time Curve From Time Zero to Infinity (Extrapolated)
Bioequivalence based on AUC0-inf (NCT00907907)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil | 26.6752 |
Cellcept® | 26.7354 |
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AUC0-t - Area Under the Concentration-time Curve From Time Zero to Time of Last Non-zero Concentration (Per Participant)
Bioequivalence based on AUC0-t (NCT00907907)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg*hr/mL (Mean) |
---|
Mycophenolate Mofetil | 25.2539 |
Cellcept® | 25.3253 |
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Cmax - Maximum Observed Concentration
Bioequivalence based on Cmax (NCT00907907)
Timeframe: Blood samples collected over 72 hour period
Intervention | µg/mL (Mean) |
---|
Mycophenolate Mofetil | 11.0939 |
Cellcept® | 11.1715 |
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Number of Non-Relapse Mortalities
Number of subjects expired without disease progression/relapse. (NCT01231412)
Timeframe: Up to 1 year
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF and CSP) | 8 |
Arm II (MMF, CSP, and Sirolimus) | 2 |
Arm 0 (CSP and Sirolimus) | 0 |
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Number of Participants With Disease-free Survival (DFS)
Disease free survival (DFS), defined as the time to death, relapse or disease progression. (NCT01871441)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Haploidentical Allogeneic HSCT) | 1 |
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Number of Participants With Relapse of Disease
Relapse of Disease is defined as the return of a disease or the signs and symptoms of a disease after a period of improvement. Relapse is almost always associated with the immunological failure of the donor immune system to recognize and/or respond to reemergence of a tumor. The number of participants with relapse of disease will be collected. (NCT01871441)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Haploidentical Allogeneic HSCT) | 2 |
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Percentage of Participants With at Least One Adverse Event (AE)
An AE was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. (NCT00551291)
Timeframe: Up to approximately 2 years
Intervention | percentage of participants (Number) |
---|
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | 90.00 |
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Mean Number of Blood Transfusions Per Visit
(NCT00551291)
Timeframe: Up to approximately 2 years
Intervention | transfusions/visit (Mean) |
---|
| Baseline (n=8) | Week 12 (n=6) | Week 18 (n=5) | End of Study (n=3) |
---|
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | 4.13 | 5.83 | 2.80 | 2.33 |
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Percentage of Participants With Clinical Response as Measured by the International Working Group (IWG) Criteria for Hematological Improvement
International Working Group (IWG) criteria for hematological improvement was defined as having hemoglobin (Hgb) <11 g/dL (pretreatment) and an increase in Hgb ≥1.5 g/dL after ≥8 weeks of treatment. (NCT00551291)
Timeframe: Up to approximately 2 years
Intervention | percentage of participants (Number) |
---|
| Week 12 (n=4) | Week 18 (n=7) | End of study (n=3) |
---|
Mycophenolate Mofetil + Prednisone + Erythropoietin Beta | 50.00 | 71.43 | 100.00 |
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Donor (Allogeneic) Hematopoietic Engraftment
Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT. (NCT01626092)
Timeframe: Day 100 Following Hematopoietic Cell Transplant (HCT)
Intervention | participants (Number) |
---|
Intent-To-Treat Patients | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Experimental: Transplant Recipients | 49 |
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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 .
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Experimental: Transplant Recipients | 18 |
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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
Intervention | Participants (Count of Participants) |
---|
Experimental: Transplant Recipients | 70 |
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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
Intervention | Participants (Count of Participants) |
---|
Experimental: Transplant Recipients | 59 |
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Disease-Free Survival (DFS)
Estimate the DFS at one-year post-transplantation. The event is defined as relapse or death due to relapse. The number of participants who did not relapse up to one year post transplant is reported. (NCT01621477)
Timeframe: one year post transplant
Intervention | participants (Number) |
---|
Treatment | 7 |
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Event-Free Survival (EFS)
Estimate the EFS at one-year post-transplantation. The event is defined as relapse or death due to any cause. The number of participants who were alive without relapse at one year post-transplant is reported. (NCT01621477)
Timeframe: one year post transplant
Intervention | participants (Number) |
---|
Treatment | 4 |
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Incidence of Malignant Relapse
Estimate the incidence of malignant relapse at one year post-transplant. The number of participants with malignant relapse or progressive disease is given. Relapse was evaluated using standard WHO criteria for each disease. (NCT01621477)
Timeframe: One year post transplantation.
Intervention | participants (Number) |
---|
Treatment | 10 |
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One-year Survival (OS)
Evaluate the number of participants alive at 1 year. The number of participants surviving to one-year post-transplantation is given. (NCT01621477)
Timeframe: One year post transplant
Intervention | participants (Number) |
---|
Treatment | 7 |
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Incidence and Severity of Acute Graft Versus Host Disease (GVHD)
The number of participants with acute GVHD is given, organized by grade. Participants are graded on a scale from 1 to 4, with 1 being mild and 4 being severe. (NCT01621477)
Timeframe: 100 days post transplant
Intervention | participants (Number) |
---|
| No Acute GVHD | Grade I | Grade II | Grade III | Grade IV |
---|
Treatment | 9 | 3 | 1 | 3 | 1 |
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Incidence and Severity of Chronic Graft Versus Host Disease (GVHD)
The severity of chronic GVHD will be described. Chronic GVHD was evaluated using NIH Consensus Global Severity Scoring. The number of participants with chronic GVHD is given, organized by severity. (NCT01621477)
Timeframe: 100 days post transplant
Intervention | participants (Number) |
---|
| No Chronic GVHD | Mild | Moderate | Severe |
---|
Treatment | 15 | 0 | 2 | 0 |
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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
Intervention | days (Median) |
---|
Regimen I | 17 |
Regimen II | 16 |
Regimen III | 15 |
Regimen IV | 14 |
Regimen V | 18 |
Regimen VI | 16 |
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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
Intervention | percentage of survival probability (Number) |
---|
Regimen I | 58 |
Regimen II | 50 |
Regimen III | 54 |
Regimen IV | 50 |
Regimen V | 38 |
Regimen VI | 50 |
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Change in EMT Score
"Change (M12 - M3) in EMT Score. Progression in EMT score is defined as an increase by >=1 of EMT score from M3 to M12.~EMT score 0 (the best): <1 EMT score 1 (the better) : 1-10% of tubular atrophy EMT score 2 : 10-25% of tubular atrophy EMT score 3 : 25-50% of tubular atrophy EMT score 4 (the worst): >50% of tubular atrophy" (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | scores on a scale (Mean) |
---|
Certican EMT+ | -0.3 |
Certican EMT- | 0.9 |
Neoral EMT+ | -0.3 |
Neoral EMT- | 0.6 |
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Change in Urine Protein/Creatinine Ratio (Without Imputation)
One of the factors associated with EMT progression between M3 and M12 according to univariate analysis (ITT biopsies M3 & M12). (NCT01079143)
Timeframe: Month 3 (baseline), Month 12
Intervention | mg/mmol (Mean) |
---|
Certican EMT+ | 44.4 |
Certican EMT- | 3.5 |
Neoral EMT+ | 16.0 |
Neoral EMT- | 29.8 |
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Change From Baseline (M3) in Estimated Glomerular Filtration Rate (eGFR)
"eGFR was calculated according to the abbreviated Modification of Diet in Renal Disease (MDRD) Formula and creatinine clearance according to the Cockcroft-Gault formula (mL/min/1.73m²).~LOCF = Last observation carried forward" (NCT01079143)
Timeframe: M3 (baseline) to M12 post transplantation
Intervention | mL/min/1.73m^2 (Least Squares Mean) |
---|
| without imputation | imputation by LOCF(96, 97) |
---|
Certican | 6.99 | 5.96 |
,Neoral | 2.54 | 2.15 |
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Change in Estimated Glomerular Filtration Rate (eGFR) at M12 From Baseline (M3) - ANCOVA Model
The average patient renal function was evaluated on the basis of eGFR was calculated according to the abbreviated MDRD formula and creatinine clearance according to the Cockcroft-Gault formula (mL/min/1.73m²). (NCT01079143)
Timeframe: Baseline (M3), M12
Intervention | mL/min/1.73m² (Mean) |
---|
| without imputation | imputation by LOCF (36, 60, 39, 58) |
---|
Certican EMT- | 5.6 | 27.3 |
,Certican EMT+ | 8.6 | -11.9 |
,Neoral EMT- | 3.8 | -4.9 |
,Neoral EMT+ | 1.5 | 5.1 |
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Change in Interstitial Fibrosis/Tabular Atrophy (IF/TA) Grade
Difference (M12 - M3) in IF/TA grade. IF/TA grade was assessed during centralized reading according to the Banff 2005/2007 classification comprising grade I (<25%), grade II (25-50%) and grade III (>50% of lesions). (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | Number of Participants (Number) |
---|
| Difference in IF/TA grade -1 | Difference in IF/TA grade 0 | Difference in IF/TA grade 1 | Difference in IF/TA grade 2 | Difference in IF/TA grade 3 |
---|
Certican EMT- | 1 | 21 | 18 | 3 | NA |
,Certican EMT+ | 5 | 9 | 7 | 3 | 2 |
,Neoral EMT- | 2 | 33 | 13 | 5 | NA |
,Neoral EMT+ | 7 | 9 | 11 | 5 | 0 |
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Change in Percentage of Interstitial Fibrosis (IF) by Numerical Quantification
Percentage of IF measured by numerical quantification. The IF percentage was transposed in grade according to the following rule: grade 0 for an IF % ≤5%, grade I for an IF % ranging from >5% to < 25%, grade II for an IF % ranging from 25 to 50%, grade III for an IF % >50%. Interstitial graft fibrosis has been identified as the primary cause of graft loss following death with a functional graft [3-5]. (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | Percentage of IF (Mean) |
---|
| Percentage of IF at M3 (n=26,43,32,53) | Percentage of IF at M12 (n=24,42,32,53) | Change in Percentage of IF (n=24,42,32,53) |
---|
Certican EMT- | 15.9 | 20.9 | 4.9 |
,Certican EMT+ | 22.8 | 27.6 | 5.1 |
,Neoral EMT- | 17.8 | 20.4 | 2.7 |
,Neoral EMT+ | 23.4 | 27.4 | 3.9 |
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Incidence (Number) of BPAR
A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system. (NCT01079143)
Timeframe: M6 and M12 post transplantation
Intervention | Number of participants (Number) |
---|
| M6 - No | M6 - Yes | M12 - No | M12 - Yes |
---|
Certican EMT- | 52 | 8 | 43 | 17 |
,Certican EMT+ | 33 | 3 | 29 | 7 |
,Neoral EMT- | 59 | 0 | 56 | 3 |
,Neoral EMT+ | 39 | 0 | 37 | 2 |
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Incidence (Number) of Participants With Graft Losses
If a participant underwent a graft nephrectomy, then the day of nephrectomy was considered as the day of graft loss. (NCT01079143)
Timeframe: M6 and M12 post transplantation
Intervention | Participants (Number) |
---|
| M6 - No | M6 - Yes | M12 - No | M12 - Yes |
---|
Certican EMT- | 60 | 0 | 56 | 4 |
,Certican EMT+ | 36 | 0 | 35 | 1 |
,Neoral EMT- | 59 | 0 | 59 | 0 |
,Neoral EMT+ | 39 | 0 | 38 | 1 |
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Incidence (Number) of Subclinical Rejections and Borderline Lesions
"Incidence of subclinical rejections and borderline lesions with regards to histological evidence of rejection with clinical findings.~Subclinical rejections: rejection without clinical symptoms which is diagnosed by chance when a graft biopsy is performed.~Clinically suspected BPAR: rejection suspected because of the presence of clinical symptoms (fever, pain, increase of creatinine) and then confirmed by the graft biopsy.~Borderline lesions: suspicious for acute T-cell mediated rejection. This category is used when no intimal arteritis is present, but there are foci of tubulitis with minor interstitial infiltration or interstitial infiltration with mild tubulitis." (NCT01079143)
Timeframe: M3
Intervention | Participants (Number) |
---|
| Subclinical rejections-No (n=68, 84) | Subclinical rejections- Yes (n=68, 84) | Subclinical rejections- missing (n=68, 84) | Clinically suspected BPAR - No (N=68, 84) | Clinically suspected BPAR - Yes (N=68, 84) | Clinically suspected BPAR - Missing (N=68, 84) | Borderline lesions - No (n=68,84) | Borderline lesions - Yes (n=68,84) | Borderline lesions - Missing (n=68,84) |
---|
Certican | 67 | 1 | 1 | 68 | 0 | 1 | 62 | 6 | 1 |
,Neoral | 84 | 0 | 1 | 84 | 0 | 1 | 71 | 13 | 1 |
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Interstitial Fibrosis/Tabular Atrophy (IF/TA)
Incidence and severity of IF/TA according to 2005/2007 Banff classification system grades (grades l to lll). IF/TA grade was assessed during centralized reading according to the Banff 2005/2007 classification comprising grade I (<25%), grade II (25-50%) and grade III (>50% of lesions). (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | Number of participants (Number) |
---|
| Interstitial Fibrosis/Tubular Atrophy (IF/TA) | IF/TA grade at M3 at grade 1 | IFTA grade at M3 at grade II | IF/TA grade at M3 at grade III | IF/TA grade at M12 at grade 0 | IT/TA grade at M12 at grade I | IF/TA grade at M12 at grade II | IF/TA grade at M12 at grade III |
---|
Certican EMT- | 38 | 5 | 0 | 0 | 20 | 18 | 5 | 0 |
,Certican EMT+ | 13 | 11 | 2 | 0 | 9 | 8 | 6 | 3 |
,Neoral EMT- | 44 | 9 | 0 | 0 | 31 | 15 | 6 | 1 |
,Neoral EMT+ | 13 | 17 | 1 | 1 | 7 | 15 | 9 | 1 |
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Number of Participants With Epithelial-mesenchymal Transition (EMT) Score
"Incidence and severity of EMT score. The intensity of EMT markers expression present and detectable in the renal graft at an early stage following transplantation is known as EMT score.~EMT score 0 (the best): <1 EMT score 1 (the better): 1-10% of tubular atrophy EMT score 2: 10-25% of tubular atrophy EMT score 3: 25-50% of tubular atrophy EMT score 4 (the worst): >50% of tubular atrophy" (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | participants (Number) |
---|
| EMT Score 0 at M3 (n= 26,43,32, 53) | EMT Score 1 at M3 (n= 26,43,32, 53) | EMT Score 2 at M3 (n= 26,43,32, 53) | EMT Score 3 at M3 (n= 26,43,32, 53) | EMT Score 4 at M3 (n= 26,43,32, 53) | EMT Score 0 at M12 (n= 25,41,32, 53) | EMT Score 1 at M12 (n= 25,41,32, 53) | EMT Score 2 at M12 (n= 25,41,32, 53) | EMT Score 3 at M12 (n= 25,41,32, 53) | EMT Score 4 at M12 (n= 25,41,32, 53) | EMT Missing Score at M12 (n= 25,41,32, 53) |
---|
Certican EMT- | 26 | 17 | 0 | 0 | 0 | 13 | 11 | 11 | 4 | 2 | 2 |
,Certican EMT+ | 0 | 0 | 17 | 9 | 0 | 1 | 7 | 10 | 4 | 3 | 1 |
,Neoral EMT- | 28 | 25 | 0 | 0 | 0 | 19 | 17 | 12 | 4 | 1 | 0 |
,Neoral EMT+ | 0 | 0 | 20 | 8 | 4 | 0 | 9 | 10 | 9 | 4 | 0 |
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Number of Participants With Epithelial-mesenchymal Transition (EMT) Status
Incidence and severity of EMT status. EMT status was determined centrally using the graft biopsy taken at 3 months. The result was quickly obtained (within 7 to 15 days) and sent to the company in charge of randomization in order to allocate each patient to a treatment group and to provide the investigator with this information without confirming EMT status. (NCT01079143)
Timeframe: M3 and M12 post transplantation
Intervention | Participants (Number) |
---|
| EMT Status at M3-Negative (n=26,43,32,53) | EMT Status at M3- Positive (n=26,43,32,53) | EMT Status at M3 - Not done (n=26,43,32,53) | EMT Status at M12- Negative (n=25,41,32,53) | EMT Status at M12- Positive (n=25,41,32,53) | EMT Status at M12- Not done (n=25,41,32,53) |
---|
Certican EMT- | 43 | 0 | 0 | 24 | 17 | 0 |
,Certican EMT+ | 0 | 26 | 0 | 8 | 17 | 0 |
,Neoral EMT- | 53 | 0 | 0 | 36 | 17 | 0 |
,Neoral EMT+ | 0 | 32 | 0 | 9 | 23 | 0 |
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Number of Participants With Progression of Renal Fibrosis Using Numerical Quantification
Comparisons according to epithelial-mesenchymal transition (EMT) profile and immunosuppressive treatment (NCT01079143)
Timeframe: M3 to M12 post transplantation
Intervention | Participants (Number) |
---|
| Fibrosis Progression - No (n=24, 42, 31, 53) | Fibrosis progression - Yes (n=24, 42,31,43) | Fibrosis progression - Missing (n=24, 42, 31, 53) |
---|
Certican EMT- | 30 | 12 | 1 |
,Certican EMT+ | 18 | 6 | 2 |
,Neoral EMT- | 42 | 11 | 0 |
,Neoral EMT+ | 19 | 12 | 0 |
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Number of Participants With Progression of Renal Graft Fibrosis (Primary Comparison - ITT Population
"Progression of Interstitial Fibrosis/Tabular Atrophy (IF/TA) is the percentage (%) of participants with an increase >= 1 in IF/TA grade according to Banff (2005 - 2007)according to Epithelial-mesenchymal transition (EMT) profile and by treatment groups.~Grade I (the better): mild interstitial fibrosis and tubular atrophy (<25% of cortical area) Grade II : moderate interstitial fibrosis and tubular atrophy (26-50% of cortical area) Grade III (the worse) : severe interstitial fibrosis and tubular atrophy (>50% of cortical area)" (NCT01079143)
Timeframe: Month 3 (M3) and Month 12 (M12) post transplantation
Intervention | Participants (Number) |
---|
| Participants with an IF/TA grade <= II at M3 | Participants with Fibrosis progression, M3 to M12 |
---|
Certican EMT+ | 26 | 12 |
,Neoral EMT+ | 31 | 16 |
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Risk Factors of IF/TA Progression
"Composite factors regarding fibrosis progression at 12 months using a logistic regression model; the parameters initially considered concerned the demographic characteristics of both recipient and donor, transplantation characteristics, hypertension, diabetes, study treatments, immunosuppression, EMT, IF/TA, function at M3, the onset of acute rejection, the presence of anti-donor antibodies at M12, infections and BK virus viremia.~Arteriolar hyaline thickening is thickening of the walls of arterioles by the deposition of homogeneous pink hyaline material.~BPAR is biopsy proven acute rejection. TEM progression is the increase ≥ 1 of TEM score between Month 3 and Month 12" (NCT01079143)
Timeframe: M12 post transplantation
Intervention | Participants (Number) |
---|
| Donor Sex - Male | Donor Sex - Female | Donor Age - <=50 years | Donor Age - >50 years | Expanded Criteria donor: NO | Expanded Criteria Donor: Yes | Delayed graft function: No | Delayed graft function: Yes | CC at M3 <50 mL/min/1.73m^2 | CC at M3 >=50 mL/min/1.73m^2 (n=67, 85) | Mesangial matrix increase at M3 - 0 (n=64, 85) | Mesangial matrix (mm) increase at M3: 1 (n=64, 85) | Mesangial matrix increase at M3: 2 (n=64, 85) | Interstitial fibrosis (ci) at M3: 0 (n=66, 85) | Interstitial fibrosis (ci) at M3: 1 (n=66, 85) | Interstitial fibrosis (ci) at M3: 2 (n=66, 85) | Arteriolar hyaline thickening (ah) at M3: 0 | Arteriolar hyaline thickening (ah) at M3: 1 | Arteriolar hyaline thickening (ah) at M3: 2 | Arteriolar hyaline thickening (ah) at M3: 3 | BPAR - No | BPAR - Yes | TEM Progression fron M3-M12: No (n=67, 83) | TEM Progression fron M3-M12: Yes (n=67, 83) | TEM Progression fron M3-M12: Missing (n=67, 83) |
---|
No- No Fibrosis Progression | 48 | 38 | 48 | 38 | 63 | 23 | 76 | 10 | 35 | 50 | 84 | 1 | 0 | 55 | 27 | 3 | 56 | 21 | 8 | 1 | 79 | 7 | 57 | 26 | 3 |
,Yes- Fibrosis Progression | 45 | 22 | 28 | 39 | 40 | 27 | 52 | 15 | 40 | 27 | 59 | 2 | 3 | 52 | 14 | 0 | 36 | 15 | 10 | 6 | 55 | 12 | 26 | 41 | 0 |
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Severity of BPAR
"A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system.~Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area.~Biopsy graded III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation)." (NCT01079143)
Timeframe: M6 and M12 post transplantation
Intervention | Participants (Number) |
---|
| M6: Banff type Grade IA | M6: Banff type Grade IB | M6: Banff type Grade IIA | M6: Banff type Grade IIB | M6: Banff type Grade III | M12: Banff type Grade IA | M12: Banff type Grade IB | M12: Banff type Grade IIA | M12: Banff type Grade IIB | M12: Banff type Grade III |
---|
Certican EMT- | 2 | 4 | 0 | 1 | 0 | 7 | 5 | 1 | 1 | 0 |
,Certican EMT+ | 0 | 0 | 1 | 0 | 0 | 2 | 2 | 1 | 0 | 0 |
,Neoral EMT- | 0 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 |
,Neoral EMT+ | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 0 | 0 | 0 |
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Treatment Failures
A treatment failure is defined as biopsy proven acute rejection (BPAR), a graft loss, a death or a loss to follow up. It was assessed between randomization and 6 and 12 months post-transplantation. (NCT01079143)
Timeframe: M6 and M12 post transplantation
Intervention | Number of Participants (Number) |
---|
| M6: Treatment Failure- No | M6: Treatment Failure- Yes | M12: Treatment Failure- No | M12: Treatment Failure- Yes | M6: BPAR - No | M6: BPAR -Yes | M12: BPAR - No | M12: BPAR - Yes | M6: Graft Loss - No | M6: Graft Loss - Yes | M12: Graft Loss - No | M12: Graft Loss - Yes | M6: Death - No | M6: Death - Yes | M12: Death - No | M12: Death - Yes | M6: Loss to follow-up - No | M6: Loss to follow-up - Yes | M12: Loss to follow-up - No | M12: Loss to follow-up - Yes |
---|
Certican EMT- | 52 | 8 | 42 | 18 | 52 | 8 | 43 | 17 | 60 | 0 | 56 | 4 | 60 | 0 | 60 | 0 | 60 | 0 | 59 | 1 |
,Certican EMT+ | 33 | 3 | 29 | 7 | 33 | 3 | 29 | 7 | 36 | 0 | 35 | 1 | 36 | 0 | 36 | 0 | 36 | 0 | 36 | 0 |
,Neoral EMT- | 59 | 0 | 56 | 3 | 59 | 0 | 56 | 3 | 59 | 0 | 59 | 0 | 59 | 0 | 59 | 0 | 59 | 0 | 59 | 0 |
,Neoral EMT+ | 39 | 0 | 36 | 3 | 39 | 0 | 37 | 2 | 39 | 0 | 38 | 1 | 39 | 0 | 39 | 0 | 39 | 0 | 39 | 0 |
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Type of Biopsy Proven Acute Rejection (BPAR)
"A biopsy proven acute rejection (BPAR) is defined as a biopsy graded IA, IB, IIA, IIB or III as per 2005/2007 Banff histological classification system.~Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells).~Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area.~Biopsy graded III: Transmural (full vessel wall thickness) arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells (with accompanying lymphocytic inflammation)." (NCT01079143)
Timeframe: M6 and M12 post transplantation
Intervention | Participants (Number) |
---|
| Cellular AR - No | Cellular AR - Yes |
---|
Certican EMT- | 46 | 14 |
,Certican EMT+ | 30 | 6 |
,Neoral EMT- | 58 | 1 |
,Neoral EMT+ | 38 | 1 |
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Allograft Rejection Rates at 30 Days
Acute Allograft Rejection (NCT00970073)
Timeframe: 30 days
Intervention | Participants (Count of Participants) |
---|
Delayed CNI Group 1 | 3 |
Delayed CNI Group 2 | 1 |
Early CNI / Control Arm | 0 |
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Estimated Glomerular Filtration Rate (eGFR) at 12 Months Post-surgery
Postoperative acute kidney injury is measured as reduced (eGFR) within 12 months post-surgery. (NCT00970073)
Timeframe: 12 Months
Intervention | mL/min (Median) |
---|
Delayed CNI Group 1 | 72 |
Delayed CNI Group 2 | 87.8 |
Early CNI / Control Arm | 51.0 |
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Graft Survival
90% graft survival, related to the deaths of 3 patients during the study period. (NCT00970073)
Timeframe: 12 months post transplant
Intervention | Participants (Count of Participants) |
---|
Delayed CNI Group 1 | 10 |
Delayed CNI Group 2 | 9 |
Early CNI / Control Arm | 8 |
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Patient Survival
(NCT00970073)
Timeframe: 12 months post-transplant
Intervention | Participants (Count of Participants) |
---|
Delayed CNI Group 1 | 10 |
Delayed CNI Group 2 | 9 |
Early CNI / Control Arm | 8 |
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Number of Participants That Experience One Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplant (HSCT)
To assess relapse free survival in participants undergoing Hematopoietic Stem Cell Transplant (HSCT) using the Thomas Jefferson University 2 step approach with an extra day inserted between the donor lymphocyte infusion (DLI) and administration of cyclophosphamide. (NCT01341301)
Timeframe: 1 year after undergoing hematopoietic stem cell transplant
Intervention | Participants (Count of Participants) |
---|
Allogeneic HSCT | 5 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 46 |
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Number of Participants With Relapse/Progression
"Relapse/Progression criteria for CLL~Progressive disease:~≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~Relapsed disease:~Criteria of progression occurring 6 months after achievement of complete or partial remission." (NCT00104858)
Timeframe: Day 84
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 1 |
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Number of Patients Achieving Complete Response and Partial Response (Overall Response Rate)
"Complete Remission (CR):~Imaging studies (Xray, CT, MRI) (nodes, liver, and spleen): Normal Peripheral blood by flow cytometry: No clonal lymphocytes Bone marrow by morphology: No nodules; or if present, nodules are free from CLL cells by immunohistochemistry Duration: ≥2 months~CR with minimal residual disease Peripheral blood or bone marrow by flow cytometry: >0 - <1 CLL cells/1000 leukocytes (0.1%)~Partial Remission (PR):~Both criteria:~Absolute lymphocyte count in peripheral blood: ≥50% decrease Physical exam/Imaging studies (nodes, liver, and/or spleen): ≥50% decrease Duration: ≥2 months" (NCT00104858)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 35 |
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Overall Survival
Number of participants surviving post-transplant (NCT00104858)
Timeframe: At 18 months
Intervention | Participants (Count of Participants) |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 34 |
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Donor and Host Polymorphisms of the FCgammaRIIIa Receptor and CD32 and Their Impact on Disease Response and Relapse
Number of participants without progressive disease and surviving at one year. Participants with FCgammaRIIIa receptor vs participants without FCgammaRIIIa receptor. (NCT00104858)
Timeframe: 1 Year
Intervention | Participants (Count of Participants) |
---|
| Surviving participants w/ FCgammaRIIIa receptor | Surviving participants w/o FCgammaRIIIa receptor | w/ FCgammaRIIIa receptor w/o progressive disease | w/o FCgammaRIIIa receptor w/o progressive disease |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 2 | 21 | 2 | 19 |
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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
Intervention | ug/ml (Median) |
---|
| Day 60 | Day 84 | Day 180 | 1 year |
---|
Treatment (Chemotherapy and Rituximab Followed by HCT) | 109 | 51 | 1.3 | .03 |
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Disease Progression/Relapse
"CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever greater than 38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~AML, ALL >5% marrow blasts by morphologic or flow cytometric, or appearance of extramedullary disease.~CLL ≥1 of: Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT00118352)
Timeframe: Up to 5 years
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 25 |
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Incidence of Grade III-IV Acute GVHD
"Severity of Individual Organ Involvement~Liver:~Stage 2 - bilirubin (3-5.9mg/100ml) Stage 3 - bilirubin (6-14.9mg/100ml) Stage 4 - bilirubin > 15mg/100ml~Gut:~Diarrhea is graded stage 1 to stage 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as stage 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall~Severity of GVHD~Grade III - Stage 2 to 4 gastrointestinal involvement and/or Stage 2 to 4 liver involvement with or without a rash Grade IV - Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00118352)
Timeframe: 100 days after transplant
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 25 |
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Incidence of Graft Rejection
Percentage patients that experienced graft rejection. (NCT00118352)
Timeframe: 84 days after transplant
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 0 |
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Incidence of High-dose Corticosteroid Utilization.
Percentage patients requiring steroids greater than 1 mg/kg. (NCT00118352)
Timeframe: 100 days after transplant
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 83.3 |
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Incidence of Infection
Percentage patients that experienced infection(s). (NCT00118352)
Timeframe: Up to 5 years post-transplant
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 100 |
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Incidence of Non-relapse Mortality
Percentage patient deaths due to non-relapse mortality (NCT00118352)
Timeframe: 100 days after transplant
Intervention | percentage of participants (Number) |
---|
Dose Level 1 (No Campath) | 8.3 |
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Change From Baseline in Glomerular Filtration Rate (GFR) at 12 Months Posttransplant
Mean percent change from baseline in estimated glomerular filtration rate (GFR) calculated by modification of diet in renal disease (MDRD)-6 variable equation at 12 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 12 months posttransplant
Intervention | Percent change in GFR (mL/min) (Mean) |
---|
CellCept + CNI (Tacrolimus or Cyclosporine) | 1.2 |
CellCept + Sirolimus | 19.7 |
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Change From Baseline in Glomerular Filtration Rate (GFR) at 24 Months Posttransplant
Mean percent change from baseline in estimated GFR calculated by modification of diet in renal disease (MDRD)-6 variable equation at 6 and 24 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 24 months posttransplant
Intervention | Mean percent change in GFR (mL/min) (Mean) |
---|
CellCept + CNI (Tacrolimus or Cyclosporine) | -8.6 |
CellCept + Sirolimus | 13.5 |
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Change From Baseline in Glomerular Filtration Rate (GFR) at 6 Months Posttransplant
Mean percent change from baseline in estimated GFR calculated by modification of diet in renal disease (MDRD)-6 variable equation at 6 and 24 months posttransplantation. MDRD-6 variables: serum creatinine, albumin and urea nitrogen, gender, age and ethnicity. (NCT00118742)
Timeframe: 6 months posttransplant
Intervention | Percent change in GFR (mL/min) (Mean) |
---|
CellCept + CNI (Tacrolimus or Cyclosporine) | 1.1 |
CellCept + Sirolimus | 25.5 |
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Change From Baseline in Creatinine Clearance
Mean percent change from baseline in calculated creatinine clearance (CL) at 6, 12, and 24 months posttransplantation (NCT00118742)
Timeframe: 6, 12, and 24 months posttransplantation
Intervention | Percent change in creatinine CL (mL/min) (Mean) |
---|
| 6 months | 12 months | 24 months |
---|
CellCept + CNI (Tacrolimus or Cyclosporine) | -1.3 | -3.0 | -12.8 |
,CellCept + Sirolimus | 18.5 | 14.0 | 7.9 |
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Number of Donors Discontinuing Atorvastatin Due to Toxicity
"The number of donors who prematurely discontinue atorvastatin therapy due to toxicity. Donors will be assessed for the following events:~Musculoskeletal and connective tissue disorders: grade 2-5~Hepatobiliary disorders: grade 2-5~Other unexpected events thought related to the use of atorvastatin; grade 2-5~In cases where the NCI criteria do not apply, intensity will be defined as:~Mild: awareness of symptom or sign, but easily tolerated~Moderate: discomfort is enough to cause interference with normal activities~Severe: inability to perform normal daily activities~Life threatening: immediate risk of death from the reaction as it occurred" (NCT01527045)
Timeframe: Prior to stem cell collection
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 2 |
Primary - Reg B (TBI Alone) | 0 |
Adjunct | 1 |
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Number of Non-relapse Mortalities
Number of patients who died without relapsed/progressive disease. (NCT01527045)
Timeframe: 1 Year post-transplant
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 4 |
Primary - Reg B (TBI Alone) | 1 |
Adjunct | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 23 |
Primary - Reg B (TBI Alone) | 3 |
Adjunct | 4 |
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Number of Patients Surviving Overall
Number of patients surviving overall post-transplant (NCT01527045)
Timeframe: 1 Year post-transplant
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 21 |
Primary - Reg B (TBI Alone) | 3 |
Adjunct | 9 |
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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
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 8 |
Primary - Reg B (TBI Alone) | 1 |
Adjunct | 5 |
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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
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 2 |
Primary - Reg B (TBI Alone) | 0 |
Adjunct | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 15 |
Primary - Reg B (TBI Alone) | 2 |
Adjunct | 6 |
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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
Intervention | Participants (Count of Participants) |
---|
Primary - Reg A (Flu/TBI) | 7 |
Primary - Reg B (TBI Alone) | 4 |
Adjunct | 3 |
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Bronchiolitis Obliterans
Development of bronchiolitis obliterans during treatment (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 5 |
Placebo | 4 |
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Cure of Chronic GVHD Without Resorting to Secondary Systemic Therapy
Withdrawal of all systemic immunosuppressive treatment after resolution of chronic GVHD, before death or onset of recurrent malignancy (NCT00089141)
Timeframe: 2 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 11 |
Placebo | 10 |
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Death
Death from any cause after enrollment in the study (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 19 |
Placebo | 10 |
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Death or Recurrent Malignancy
Death due to any cause or development of recurrent malignancy at any time after enrollment (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 25 |
Placebo | 15 |
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Definitive Absence of Efficacy Success
Administration of secondary systemic therapy for chronic GVHD, death during primary therapy, or onset of recurrent malignancy or bronchiolitis obliterans during primary therapy (NCT00089141)
Timeframe: 2 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 45 |
Placebo | 40 |
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End of Systemic Treatment
Withdrawal of all immunosuppressive treatment without recurrent malignancy (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 15 |
Placebo | 15 |
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Non-relapse Mortality
Death without prior development of recurrent malignancy (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 8 |
Placebo | 5 |
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Open Label Systemic Treatment Because of Inadequate Response to Primary Therapy
Administration of any systemic therapy other than the immunosuppressive agents used for initial treatment, because of persistent or progressive chronic graft-versus-host disease (NCT00089141)
Timeframe: 2 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 24 |
Placebo | 25 |
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Recurrent Malignancy
Development of recurrent malignancy after enrollment in the study (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 17 |
Placebo | 10 |
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Withdrawal of Prednisone
Withdrawal of treatment with prednisone after improvement or resolution of chronic GVHD (NCT00089141)
Timeframe: within 4 years
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 30 |
Placebo | 33 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 15 |
Arm II (Nonmyeloablative Conditioning With TBI) | 6 |
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Incidence of Grade III/IV GVHD
"Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00089011)
Timeframe: Day 180 post-transplantation
Intervention | Participants (Count of Participants) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 2 |
Arm II (Nonmyeloablative Conditioning With TBI) | 4 |
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Incidences of Grades II-IV Acute GVHD
"Number of patients who developed acute/chronic GVHD post-transplant. aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA a maculopapular eruption involving 25 - 50% BSA generalized erythroderma generalized erythroderma with bullous formation and often with desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL bilirubin 3 - 5.9 mg/100 mL bilirubin 6 - 14.9 mg/100 mL bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 3 skin and/or stage 1 gut involvement and/or stage 1 liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00089011)
Timeframe: Day 180 post-transplantation
Intervention | Participants (Count of Participants) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 26 |
Arm II (Nonmyeloablative Conditioning With TBI) | 14 |
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Incidences of Graft Rejection
Number of patients who rejected their graft. Rejection is defined as the inability to detect or loss of detection of greater than 5% donor T cells (CD3+) as a proportion of the total T cell population, respectively, after nonmyeloablative HCT. (NCT00089011)
Timeframe: Day 180 post-transplantation
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 85 |
Arm II (Nonmyeloablative Conditioning With TBI) | 39 |
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Rate and Duration of Steroid Use for the Treatment of Chronic GVHD
Number of patients that received prednisone treatment of chronic GVHD, and number of days for which they received prednisone. (NCT00089011)
Timeframe: Up to 5 years
Intervention | days (Median) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 78 |
Arm II (Nonmyeloablative Conditioning With TBI) | 89 |
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Rates of Disease Progression
"Relapse/Progression criteria:~CML New cytogenetic abnormality and/or development of accelerated phase or blast crisis. The criteria for accelerated phase will be defined as unexplained fever >38.3°C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, marrow blasts and promyelocytes >20%.~CMML, AML, ALL >30% BM blasts w/ deteriorating performance status, or worsening of anemia, neutropenia, or thrombocytopenia.~CLL ≥1 of: Physical exam/imaging studies ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ treatment; or definite increase in the size or number of plasmacytomas or lytic bone lesions." (NCT00089011)
Timeframe: Up to 5 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Nonmyeloablative Conditioning With Fludarabine and TBI) | 41 |
Arm II (Nonmyeloablative Conditioning With TBI) | 33 |
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Number of Non-Relapse Mortalities
"Percentage of NRM as estimated by cumulative incidence methods with competing risks.~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 200 days after transplant
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF and Tacrolimus) | 48 |
Arm II (MMF and Tacrolimus Alternate Schedule) | 47 |
Arm III (MMF, Tacrolimus, and Sirolimus) | 40 |
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Number of Participants Surviving Without Progression
"Number of patients with progression-free survival, estimated by cumulative incidence methods~Cumulative incidence methods are the standard way to estimate incidence of an endpoint in the presence of competing risks and censoring (ref) Here is the reference. Gooley TA, Leisenring W, Crowley J, Storer BE: Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine 18:695-706, 1999. PMID 10204198" (NCT00105001)
Timeframe: 2 Years post-transplant
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF and Tacrolimus) | 38 |
Arm II (MMF and Tacrolimus Alternate Schedule) | 35 |
Arm III (MMF, Tacrolimus, and Sirolimus) | 22 |
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Number of Participants With Grades II-IV Acute GVHD
"Number of patients with grades II-IV acute GVHD~aGVHD Stages~Skin:~a maculopapular eruption involving < 25% BSA~a maculopapular eruption involving 25 - 50% BSA~generalized erythroderma~generalized erythroderma w/ bullous formation and often w/ desquamation~Liver:~bilirubin 2.0 - 3.0 mg/100 mL~bilirubin 3 - 5.9 mg/100 mL~bilirubin 6 - 14.9 mg/100 mL~bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients w/ visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade II: Stage 1 - 2 skin w/ no gut/liver involvement Grade III: Stage 2 - 4 gut involvement and/or stage 2 - 4 liver involvement Grade IV: Pattern and severity of GVHD similar to grade 3 w/ extreme constitutional symptoms or death" (NCT00105001)
Timeframe: 150 days after transplant
Intervention | Participants (Count of Participants) |
---|
Arm I (MMF and Tacrolimus) | 44 |
Arm II (MMF and Tacrolimus Alternate Schedule) | 34 |
Arm III (MMF, Tacrolimus, and Sirolimus) | 32 |
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Engraftment
Number of subjects who engrafted post-transplant. Engraftment will be monitored in a sequential fashion. (NCT00054353)
Timeframe: Up to 5 years
Intervention | Participants (Count of Participants) |
---|
Related Donor | 12 |
Unrelated Donor | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
Related Donor | 2 |
Unrelated Donor | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Related Donor | 4 |
Unrelated Donor | 1 |
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Non-relapse Mortality
Early NRM will be monitored in a sequential fashion. (NCT00054353)
Timeframe: At day 100
Intervention | Participants (Count of Participants) |
---|
Related Donor | 1 |
Unrelated Donor | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Related Donor | 4 |
Unrelated Donor | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Related Donor | 1 |
Unrelated Donor | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Related Donor | 2 |
Unrelated Donor | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
| 6 Months | 1 Year | 2 Years | 3 Years | 4 Years | 5 Years |
---|
Related Donor | 8 | 5 | 4 | 3 | 3 | 3 |
,Unrelated Donor | 3 | 2 | 0 | 0 | 0 | 0 |
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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
Intervention | percentage 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
Intervention | percentage of participants (Number) |
---|
Treatment (Nonmyeloablative Donor PBSC Transplant) | 0 |
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Incidence of Relapse
Percent patients with relapsed disease post-transplant. (NCT00045435)
Timeframe: By 1 year after transplant
Intervention | percentage of participants (Number) |
---|
Treatment (Nonmyeloablative Donor PBSC Transplant) | 41.2 |
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Nonrelapse Mortality (NRM)-Incidence of Nonrelapse Death
Defined as death without morphologic evidence of disease. Sufficient evidence will be taken to be an observed rate of NRM within 200 days of transplant that corresponds to a one-sided 80% confidence interval with a lower limit greater than 15%. (NCT00045435)
Timeframe: 200 days after transplant
Intervention | percentage of participants (Number) |
---|
Treatment (Nonmyeloablative Donor PBSC Transplant) | 6 |
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Overall Survival
Percent patients surviving. (NCT00045435)
Timeframe: By 1 year after transplant
Intervention | percentage of participants (Number) |
---|
Treatment (Nonmyeloablative Donor PBSC Transplant) | 70.6 |
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Incidence of Acute and Chronic GVHD
Percent patients with acute/chronic GVHD (NCT00045435)
Timeframe: aGVHD: 100 days after transplant; cGVHD: 1 Year after transplant.
Intervention | percentage of participants (Number) |
---|
| Grade II-IV aGVHD | cGVHD |
---|
Treatment (Nonmyeloablative Donor PBSC Transplant) | 35.3 | 35.3 |
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Incidence of Grade IV Acute GVHD
"Clinical Stage of acute GVHD according to Organ System~Skin:~- Maculopapular rash <25% of body surface~- Maculopapular rash 25-50% of body surface~- Maculopapular rash >50% body surface area or generalized erythroderma~- Generalized erythroderma with bullous formation and desquamation~Liver:~- Bilirubin 2-3 mg/dl~- Bilirubin 3.1-6 mg/dl~- Bilirubin 6.1-15 mg/dl~- Bilirubin >15 mg/dl~Gut:~- >500-1000 mL diarrhea per day or (nausea, anorexia or vomiting with biopsy (EGD) confirmation of upper GI GVHD~- >1000 -1500 mL diarrhea per day~- >1500 mL diarrhea per day~- >1500 mL diarrhea per day plus severe abdominal pain with or without ileus~Overall Clinical Grading of Severity of acute GVHD Grade IV: 0-4 Skin, 2-4 Liver, and/or 2-4 GI" (NCT00096161)
Timeframe: Within 100 days after the last DLI
Intervention | percentage of participants (Number) |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 5 |
Group 1B (Pentostatin, DLI Dose Level 2) | 0 |
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 0 |
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Incidence of Infections
(NCT00096161)
Timeframe: 100 days after DLI
Intervention | percentage of participants (Number) |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 80 |
Group 1B (Pentostatin, DLI Dose Level 2) | 70 |
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 33.3 |
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Incidence of Relapse/Progression
"CML Acquisition of a new cytogenetic abnormality and/or development of accelerated phase or blast crisis. Criteria for accelerated phase: unexplained fever >38.3° C, new clonal cytogenetic abnormalities in addition to a single Ph-positive chromosome, BM blasts and promyelocytes >20%.~AML, ALL, CMML >30% BM blasts w/ deteriorating performance status, or worsening of anemia, neutropenia, or thrombocytopenia.~CLL Progressive disease: ≥1 of: physical exam/imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, and lymph node biopsy w/ Richter's transformation.~NHL >25% increase in the sum of the products of the perpendicular diameters of marker lesions, or the appearance of new lesions.~MM~≥100% increase of the serum myeloma protein from its lowest level, or reappearance of myeloma peaks that had disappeared w/ tx; or definite increase in the size/number of plasmacytomas or lytic bone lesions." (NCT00096161)
Timeframe: 1 year after DLI
Intervention | percentage of participants (Number) |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 45 |
Group 1B (Pentostatin, DLI Dose Level 2) | 20 |
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 33.3 |
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Percentage Patients With an Increase of at Least 10 Percentage Points in Donor T-cell Chimerism
"A regimen will be considered successful if 20 patients are enrolled, at least 13 demonstrate improved chimerism. If fewer than 5 patients have shown improvement in chimerism then it can be at least 75% confident that the true rate of improvement is less than 0.53. Enrollment to the regimen will stop and the next regimen will be opened. Enrollment to a regimen may also be stopped at any time it becomes impossible to achieve 5 of 10 or 13 of 20 successful improvements.~Chimerism in hematopoietic cell transplant derives from this idea of a mixed entity, referring to someone who has received a transplant of genetically different tissue. A test for chimerism after a hematopoietic cell transplant involves identifying the genetic profiles of the recipient and of the donor and then evaluating the extent of mixture in the recipient's blood cells or marrow cells." (NCT00096161)
Timeframe: From the time of enrollment maintained to day 56 after the last DLI, up to Day 112
Intervention | percentage of participants (Number) |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 60 |
Group 1B (Pentostatin, DLI Dose Level 2) | 30 |
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 33.3 |
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Survival
Percentage patients surviving. (NCT00096161)
Timeframe: 1 year after DLI
Intervention | percentage of participants (Number) |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 60 |
Group 1B (Pentostatin, DLI Dose Level 2) | 90 |
Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 66.7 |
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Incidence of GVHD
"Percentage patients with acute or chronic GVHD.~The diagnosis of chronic GVHD requires at least one manifestation that is distinctive for chronic GVHD as opposed to acute GVHD. In all cases, infection and others causes must be ruled out in the differential diagnosis of chronic GVHD." (NCT00096161)
Timeframe: 1 year after DLI
Intervention | percentage of participants (Number) |
---|
| aGVHD | cGVHD |
---|
Group 1A (Pentostatin, DLI Dose Level 1) | 20 | 50 |
,Group 1B (Pentostatin, DLI Dose Level 2) | 0 | 20 |
,Group 2C (Pentostatin, DLI Dose Level 1, Add'l IS) | 0 | 16.7 |
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Mean International Society for Heart and Lung Transplantation Biopsy Score Over the First 6 Months Post-transplantation
Mean ISHLT biopsy score Biopsies of the heart may be various grades and each is assigned a numerical score. Grade 0 is 0 points, 1A = 1, 1B = 2, grade 2 = 3, grade 3A = 4, Grade 3B = 5, and grade 4 = 6 units. The mean biopsy score is the numeric average of the biopsy scores for the first 6 post-transplant months. Best value is 0, worst score is 6. (NCT00299221)
Timeframe: 6 months
Intervention | units on a scale (Mean) |
---|
Monotherapy | 0.7 |
Combination Therapy | 0.65 |
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Mean ISHLT Biopsy Score Over First Year Post-transplant
Mean ISHLT biopsy score Biopsies of the heart may be various grades and each is assigned a numerical score. Grade 0 is 0 points, 1A = 1, 1B = 2, grade 2 = 3, grade 3A = 4, Grade 3B = 5, and grade 4 = 6 units. The mean biopsy score is the numeric average of the biopsy scores for the first 6 post-transplant months. Best value is 0, worst score is 6. (NCT00299221)
Timeframe: 1 year
Intervention | units on a scale (Mean) |
---|
Monotherapy | 0.67 |
Combination Therapy | 0.62 |
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Number of Patients With Allograft Vasculopathy (CAD) at One Year Post Transplant
Number of patients diagnosed with allograft vasculopathy / coronary artery disease (CAD) at one year post transplant (NCT00299221)
Timeframe: 1 year
Intervention | patients (Number) |
---|
Monotherapy | 0 |
Combination Therapy | 0 |
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Number of Patients With Cytomegalovirus (CMV) at One Year Post-transplant
Number of patients developing cytomegalovirus disease by 1 year post-transplant (NCT00299221)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Monotherapy | 2 |
Combination Therapy | 2 |
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Percent of Patients Alive at One Year Post-transplant
Percent of patients alive at one year post-transplant. In other words, all cause mortality over time (NCT00299221)
Timeframe: 1 year
Intervention | percent of participants (Number) |
---|
Monotherapy | 98 |
Combination Therapy | 98 |
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Best Overall Response (BOR) at Cycle 7 Day 1
BOR was defined as the percentage of participants who achieved an overall response (CR+PR) based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response at any time point (up to Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD). Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. This analysis was based on the primary cutoff date (May 2020). (NCT03112603)
Timeframe: up to Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib | 76.4 |
Best Available Therapy | 60.4 |
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BOR During Cross-over Treatment With Ruxolitinib
BOR was defined as the percentage of participants who achieved an overall response (CR+PR) based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response at any time point (up to Cycle 7 Day 1 or the start of additional systemic therapy for cGvHD). Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: from Crossover Cycle 1 Day 1 to any time point up to and including Crossover Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib Cross-Over Period | 81.4 |
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Duration of Response Through Study Completion
DOR was defined as the time from first response until cGvHD progression, death, or the date of change/addition of systemic therapies for cGvHD and as assessed for responders only. Response was based on cGvHD disease assessments (National Institutes of Health consensus criteria). Duration of response was evaluated in participants who achieved a CR or PR at or before Cycle 7 Day 1. The analysis included a larger number of participants than the number of participants who achieved CR or PR at Cycle 7 Day 1 (82 ruxolitinib and 42 BAT) because the analysis took into account not only those participants who achieved CR or PR at Cycle 7 Day 1, but also participants who achieved CR or PR before Cycle 7 Day 1 but who may have lost their response at Cycle 7 Day 1. For this reason, the number of participants in this analysis does not align with the best overall response (BOR) at Cycle 7 Day 1. This analysis was based on the cutoff date upon study completion (December 2022). (NCT03112603)
Timeframe: from first response to LPLV (approximately 5 years)
Intervention | Months (Median) |
---|
Ruxolitinib | NA |
Best Available Therapy | 6.4 |
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Efficacy of Ruxolitinib Versus Investigator's Choice Best Available Therapy (BAT) in Participants With Moderate or Severe Steroid Refractory Chronic Graft Versus Host Disease (SR-cGvHD) Assessed by Overall Response Rate (ORR) at the Cycle 7 Day 1 Visit
ORR was defined as the percentage of participants in each arm demonstrating a complete response (CR) or partial response (PR) based on chronic GvHD (cGvHD) disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response. Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib | 49.7 |
Best Available Therapy | 25.6 |
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Number of Participants With Any Treatment-emergent Adverse Event (TEAE)
Adverse events were defined as the appearance of (or worsening of any pre-existing) undesirable signs, symptoms, or medical conditions that occurred after the participant's signed informed consent was obtained. Abnormal laboratory values or test results occurring after informed consent constituted adverse events only if they induced clinical signs or symptoms, were considered clinically significant, required therapy (e.g., hematologic abnormality that required transfusion or hematological stem cell support), or required changes in study medication(s). TEAEs were defined as those AEs that started or worsened during the on-treatment period (either randomized or cross-over period). (NCT03112603)
Timeframe: from Baseline to LPLV (approximately 5 years)
Intervention | Participants (Count of Participants) |
---|
Ruxolitinib | 165 |
Best Available Therapy | 148 |
Ruxolitinib Cross-Over Period | 70 |
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ORR at the End of Cycle 3
ORR was defined as the percentage of participants in each arm demonstrating a CR or PR based on cGvHD disease assessments (National Institutes of Health Consensus Criteria) without the requirement of additional systemic therapies for an earlier progression, mixed response, or non-response. Scoring of response was relative to the organ score at the time of randomization. CR: complete resolution of all signs and symptoms of cGVHD in all evaluable organs without the initiation or addition of new systemic therapy. PR: improvement in at least one organ (e.g., improvement of 1 or more points on a 4- to 7-point scale, or an improvement of 2 or more points on a 10- to 12-point scale) without progression in other organs or sites, initiation, or addition of new systemic therapies. (NCT03112603)
Timeframe: Cycle 4 Day 1 (each cycle was comprised of 4 weeks)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib | 54.5 |
Best Available Therapy | 31.1 |
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Overall Survival (OS)
Overall survival was defined as the time from the date of randomization to the date of death due to any cause. (NCT03112603)
Timeframe: from the date of randomization to the date of death due to any cause up to LPLV (approximately 5 years)
Intervention | months (Median) |
---|
Ruxolitinib | NA |
Best Available Therapy | NA |
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Rate of Failure-free Survival (FFS)
Composite time to event endpoint incorporating the following FFS events: (i) relapse or recurrence of underlying disease or death due to underlying disease, (ii) nonrelapse mortality, or (iii) addition or initiation of another systemic therapy for cGvHD. (NCT03112603)
Timeframe: Baseline to when the last participant reached Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Intervention | months (Median) |
---|
Ruxolitinib | NA |
Best Available Therapy | 5.7 |
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Rate of FFS at Study Completion
Composite time to event endpoint incorporating the following FFS events: (i) relapse or recurrence of underlying disease or death due to underlying disease, (ii) nonrelapse mortality, or (iii) addition or initiation of another systemic therapy for cGvHD. (NCT03112603)
Timeframe: From Baseline to Last Participant Last Visit (LPLV) (approximately 5 years)
Intervention | months (Median) |
---|
Ruxolitinib | 38.4 |
Best Available Therapy | 5.7 |
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Rate of Participants With Clinically Relevant Improvement of the Modified Lee cGvHD Symptom Scale Score
"To assess improvement of symptoms based on the total symptom score (TSS); a responder was defined as having achieved a clinically relevant reduction from Baseline of the TSS. The scale consists of 30 items in 7 subscales (skin, eye, mouth, lung, nutrition, energy, and psychological). Participants reported their level of symptom bother over the previous month on a 5-point likert scale: not at all, slightly, moderately, quite a bit, or extremely. Subscale scores and the summary score range from 0 to 100, with a higher score indicating worse symptoms." (NCT03112603)
Timeframe: Baseline; Cycle 7 Day 1 (each cycle was comprised of 4 weeks)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib | 24.2 |
Best Available Therapy | 11.0 |
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Utilization of Medical Resources
The percentage of participants with at least one submission to healthcare encounter was assessed. (NCT03112603)
Timeframe: from Baseline to LPLV (approximately 5 years)
Intervention | percentage of participants (Number) |
---|
Ruxolitinib | 57.0 |
Best Available Therapy | 65.8 |
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AUCinf of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"AUCinf was defined as the area under the concentration-time curve from time 0 to infinity. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | ng*hour/mL (Geometric Mean) |
---|
| Day 1 |
---|
Ruxolitinib | 642 |
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AUClast of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"AUClast was defined as the area under the concentration-time curve up to the last measurable concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | ng*hour/mL (Geometric Mean) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 636 | 945 |
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Change From Baseline in EQ-5D-5L
The EQ-5D-5L is a descriptive classification consisting of five dimensions of health: mobility, self-care, usual activities, anxiety/depression, and pain/discomfort. The five-level version (no problems, slight problems, moderate problems, severe problems, and extreme problems) uses a 5-point Likert scale, with 1 being no problems and 5 being extreme problems. (NCT03112603)
Timeframe: Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)
Intervention | scores on a scale (Mean) |
---|
| Cycle 2 Day 1 | Cycle 3 Day 1 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 9 Day 1 | Cycle 12 Day 1 | Cycle 15 Day 1 | Cycle 18 Day 1 | Cycle 21 Day 1 | Cycle 24 Day 1 | Cycle 27 Day 1 | Cycle 30 Day 1 | Cycle 33 Day 1 | Cycle 36 Day 1 | Cycle 39 Day 1 |
---|
Best Available Therapy | -0.01 | -0.04 | -0.01 | -0.03 | 0.01 | -0.00 | -0.02 | 0.02 | 0.03 | 0.02 | -0.00 | 0.01 | 0.03 | 0.01 | 0.05 | 0.04 | 0.01 |
,Ruxolitinib | 0.03 | 0.03 | 0.04 | 0.02 | 0.05 | 0.07 | 0.07 | 0.07 | 0.07 | 0.07 | 0.08 | 0.07 | 0.06 | 0.05 | 0.00 | 0.06 | 0.06 |
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Change From Baseline in Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT)
"Change from Baseline was calculated as the post-Baseline value minus the Baseline value. The FACT-BMT is a 50-item self-report questionnaire that measures the effect of a therapy on domains including physical, functional, social/family, and emotional well-being, together with additional concerns relevant for bone marrow transplantation participants. The questions were based on a 5-point Likert scale, where 0 corresponds to not at all and 4 corresponds to very much. The higher the final score, the better the quality of life. The FACT-BMT total score ranges from 0 to 148." (NCT03112603)
Timeframe: Baseline; up to Cycle 39 Day 1 (each cycle was comprised of 4 weeks)
Intervention | scores on a scale (Mean) |
---|
| Cycle 2 Day 1 | Cycle 3 Day 1 | Cycle 4 Day 1 | Cycle 5 Day 1 | Cycle 6 Day 1 | Cycle 7 Day 1 | Cycle 9 Day 1 | Cycle 12 Day 1 | Cycle 15 Day 1 | Cycle 18 Day 1 | Cycle 21 Day 1 | Cycle 24 Day 1 | Cycle 27 Day 1 | Cycle 30 Day 1 | Cycle 33 Day 1 | Cycle 36 Day 1 | Cycle 39 Day 1 |
---|
Best Available Therapy | -0.22 | -1.82 | -1.05 | -0.23 | 2.41 | 0.85 | 1.20 | 3.74 | 7.58 | 8.19 | 3.68 | 7.84 | 5.10 | 5.75 | 5.67 | 4.77 | 6.64 |
,Ruxolitinib | 2.32 | 0.62 | 1.98 | 1.25 | 2.91 | 4.14 | 5.32 | 7.26 | 5.07 | 4.10 | 5.24 | 5.90 | 6.23 | 7.53 | 5.17 | 8.72 | 8.65 |
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CL/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"CL/F was defined as the oral dose clearance of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | Liters/hour (Geometric Mean) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 15.6 | 15.2 |
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Cmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"Cmax was defined as the maximum observed plasma concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | nanograms per milliliter (ng/mL) (Geometric Mean) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 167 | 215 |
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Cumulative Incidence of Malignancy Relapse/Recurrence (MR)
Defined as the cumulative incidence rate from competing risk analysis of MR from the date of randomization to hematologic malignancy relapse/recurrence. (NCT03112603)
Timeframe: Months 3, 6, 12, 18, 24, 30, and 36
Intervention | percentage of participants (Number) |
---|
| 0 to < 3 months | 3 to < 6 months | 6 to < 12 months | 12 to < 18 months | 18 to < 24 months | 24 to <30 months | 30 to <36 months |
---|
Best Available Therapy | 1.31 | 2.65 | 6.08 | 6.08 | 6.08 | 6.78 | 7.50 |
,Ruxolitinib | 1.92 | 3.22 | 5.18 | 7.82 | 8.48 | 8.48 | 8.48 |
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Cumulative Incidence of Non-relapse Mortality (NRM)
Defined as the cumulative incidence rate from competing risk analysis for NRM from the date of randomization to the date of death not preceded by underlying disease relapse/recurrence. (NCT03112603)
Timeframe: Months 3, 6, 12, 18, 24, 30, and 36
Intervention | percentage of participants (Number) |
---|
| Month 3 | Month 6 | Month 12 | Month 18 | Month 24 | Month 30 | Month 36 |
---|
Best Available Therapy | 4.44 | 6.43 | 15.12 | 16.48 | 19.22 | 19.22 | 22.0 |
,Ruxolitinib | 5.45 | 9.13 | 15.30 | 15.93 | 17.83 | 17.83 | 17.83 |
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Percentage of Participants Successfully Tapered Off of All Corticosteroids
All corticosteroid dosages prescribed to the participant and all dose changes during the study were to be recorded for assessment of participants who successfully tapered off of all corticosteroids. Participants who completely tapered off corticosteroids refer to those who permanently discontinued steroids as per the dose administration panel and who did not restart steroids in the same interval. Participants who were tapered off and continued follow-up were also counted as being tapered off with 0 dose in subsequent intervals until they discontinued from the main treatment period or restarted steroid treatment. (NCT03112603)
Timeframe: up to Day 179
Intervention | percentage of participants (Number) |
---|
| Day 1 to ≤ Day 28 | Day 29 to ≤ Day 56 | Day 57 to ≤ Day 84 | Day 85 to ≤ Day 112 | Day 113 to ≤ Day 140 | Day 141 to ≤ Day 168 | Day 169 to ≤ Day 179 |
---|
Best Available Therapy | 2.6 | 5.4 | 8.5 | 10.3 | 12.4 | 16.8 | 15.9 |
,Ruxolitinib | 2.5 | 9.6 | 14.0 | 16.3 | 19.7 | 24.2 | 24.1 |
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Percentage of Participants With a ≥ 50% Reduction in Daily Corticosteroid Dose
All corticosteroid dosages prescribed to the participant and all dose changes during the study were to be recorded for assessment of participants with a ≥ 50% reduction in daily corticosteroid dose. (NCT03112603)
Timeframe: from Day 15 up to Day 182
Intervention | percentage of participants (Number) |
---|
| Day 15 to ≤ Day 28 | Day 29 to ≤ Day 42 | Day 43 to ≤ Day 56 | Day 57 to ≤ Day 70 | Day 71 to ≤ Day 84 | Day 85 to ≤ Day 98 | Day 99 to ≤ Day 112 | Day 113 to ≤ Day 126 | Day 127 to ≤ Day 140 | Day 141 to ≤ Day 154 | Day 155 to ≤ Day 168 | Day 169 to ≤ Day 182 |
---|
Best Available Therapy | 13.2 | 33.1 | 41.1 | 47.9 | 51.4 | 54.0 | 60.4 | 66.2 | 68.3 | 68.3 | 71.6 | 88.8 |
,Ruxolitinib | 12.7 | 35.0 | 48.4 | 58.7 | 62.3 | 69.5 | 71.2 | 73.2 | 72.8 | 70.0 | 74.6 | 81.9 |
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t1/2 of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"t1/2 was defined as the apparent terminal phase disposition half-life of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | hours (Geometric Mean) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 2.40 | 2.32 |
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Tmax of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"tmax was defined as the time to reach the maximum plasma concentration of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | hours (Median) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 0.833 | 1.00 |
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Vz/F of Ruxolitinib After Single (Cycle 1 Day 1) and Multiple (Cycle 1 Day 15) Doses
"Vz/F was defined as the apparent oral dose volume of distribution of ruxolitinib. Early enrolling participants (approximately the first 8 adult and first 4 adolescent participants) randomized to ruxolitinib arm followed an extensive PK sampling schedule. Subsequent participants randomized to ruxolitinib, any randomized participants receiving ruxolitinib after Cycle 6, and any randomized participants receiving BAT that cross over to ruxolitinib followed the sparse PK sampling schedule." (NCT03112603)
Timeframe: Extensive Sampling Schedule: Cycle 1 Days 1 and 15: predose; 0.5, 1, 1.5, 4, 6, and 9 hours post-dose. Sparse Sampling Schedule: Cycle 1 Days 1 and 15: predose; 1.5 hours post-dose
Intervention | Liters (Geometric Mean) |
---|
| Day 1 | Day 15 |
---|
Ruxolitinib | 54.0 | 50.9 |
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Area Under the Plasma Concentration Time Curve From 0-12 Hours for Mycophenolic Acid and Mycophenolic Acid Glucuronide Phenolic Glucuronide of Mycophenolic Acid
The area under the plasma concentration-time curve from time zero to twelve hours (AUC [0-12h]) is area under the plasma concentration-time curve from time zero through 12 hours. AUC (0-12) hours was computed using the linear trapezoidal rule. For the calculations of AUC (0-12h), concentrations below the limit of quantification were assigned a value of zero if they occurred at the beginning of a profile. When such values appeared at the end of a profile they were assigned as missing data. AUC was reported in microgram hour per milliliter (mcg*h/mL). (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants greater than (>) 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants less than (<) 24 months
Intervention | mcg*h/mL (Mean) |
---|
| MPA Raw | MPAG Raw | MPAG (MPA Equivalents) |
---|
Drug MMF | 29 | 487 | 289 |
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Area Under the Plasma Concentration-Time Curve From 0 to 12 Hours of Mycophenolic Acid Normalized for Dose And for Body Surface Area
The area under the plasma concentration-time curve from time zero to twelve hours (AUC [0-12h]) is area under the plasma concentration-time curve from time zero through 12 hours. AUC (0-12) hours was computed using the linear trapezoidal rule. For the calculations of AUC (0-12h), concentrations below the limit of quantification were assigned a value of zero if they occurred at the beginning of a profile. When such values appeared at the end of a profile they were assigned as missing data. AUC0-12h was normalized to 600 milligram per square meter (mg/m^2) and 1.5 gram. AUC was reported in microgram hour per milliliter (mcg*h/mL). (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants greater than (>) 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants less than (<) 24 months
Intervention | mcg*h/mL (Mean) |
---|
| Normalized to 600mg/m^2 | Normalized to 1.5g |
---|
Drug MMF | 65.6 | 363 |
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Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide
The Plasma Concentration (Cmax) is defined as maximum observed analyte concentration. Cmax was obtained directly from the measured plasma concentration-time curves. (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants > 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants < 24 months
Intervention | mcg/mL (Mean) |
---|
| MPA Raw | MPAG Raw | MPAG (MPA Equivalents) |
---|
Drug MMF | 7.98 | 54.6 | 32.4 |
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Number of Participants With Adverse Events and Serious Adverse Events
An AE is defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. An SAE is defined as any untoward medical occurrence that, at any dose, results in death, is life threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is a significant medical event. (NCT02630563)
Timeframe: Up to Day 32
Intervention | participants (Number) |
---|
| Participants with AE | Participants with SAE |
---|
Drug MMF | 1 | 0 |
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Time to Maximum Plasma Concentration for Mycophenolic Acid and Mycophenolic Acid Glucuronide
Tmax is the amount of time after dosing to when the maximum concentration of MPA and MPAG was achieved. (NCT02630563)
Timeframe: Pre-dose, 0.5, 0.75, 1.0, 1.5, 2.0, 4.0, 8.0, and 12.0 hours post oral dosing for participants > 24 months, and at pre-dose, 0.75, 2.0, 4.0 and 12.0 hours post oral dosing for participants < 24 months
Intervention | hour (Median) |
---|
| MPA Raw | MPAG Raw |
---|
Drug MMF | 1.35 | 1.99 |
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Acute Graft Versus Host Disease (aGVHD) of Grades 2-4 According to the Consensus Grading
Acute graft versus host disease is graded according to the 1994 Keystone Consensus Grading. aGVHD grade was evaluated from day 0 through 100 days post-transplant. The first day of acute GVHD onset at grades 2-4 was used to calculate the cumulative incidence. Relapse/death prior to onset was considered competing events. (NCT03128359)
Timeframe: Up to 100 days post-stem cell infusion
Intervention | percentage of probability (Number) |
---|
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis) | 47 |
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis) | 53 |
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Graft-Versus-Host Disease-Free, Relapse-Free Survival (GRFS) at 1 Year
Estimates will be calculated using the Kaplan-Meier method, Greenwood formula will be used to calculate standard error (SE), and log-log transformation method will be used to construct 95% confidence intervals. Graft versus host disease (GVHD, acute and chronic), disease status and vital status will be monitored per clinical standard operating procedure. For this endpoint failure is defined as the first occurrence of grade 3 or 4 acute GVHD, or moderate/severe chronic GVHD, or disease relapse (for patients in complete remission at the start of conditioning) or disease progression (for patients with active disease at the start of conditioning) or death (from any cause). Patients not experiencing any of these will be censored at his/her date of last contact. (NCT03128359)
Timeframe: From stem cell infusion to grade 3-4 acute graft versus host disease (GVHD), moderate-severe chronic GVHD, relapse, progression or death (from any cause), whichever occurs first, assessed for up to 1 year.
Intervention | percentage of survival probability (Number) |
---|
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis) | 53 |
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis) | 84 |
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Overall Survival (OS) at 1 Year
Estimates was calculated using the Kaplan-Meier method, Greenwood formula was used to calculate SE, and log-log transformation method was used to construct 95% confidence intervals. Each patient's vital status was monitored per clinical standard operating procedure. For this endpoint failure is defined as death (from any cause). Patients not experiencing a death event was censored at his/her date of last contact. (NCT03128359)
Timeframe: From start of transplant to death, or last follow up, whichever occurs first, assessed for up to 1 year.
Intervention | percentage of probability (Number) |
---|
Regimen A (Fludarabine, Melphalan, PBSC HCT, GVHD Prophylaxis) | 68 |
Regimen C (Fludarabine, TBI, PBSC HCT, GVHD Prophylaxis) | 100 |
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Measured Glomerular Filtration Rate
Progression of renal function measured by mean mGFR at 36 months after renal TX. The mGFR was measured using Iohexol or Cr-EDTA clearance according to local practice. (NCT00634920)
Timeframe: Month 36
Intervention | mL/min/1.73m^2 (Mean) |
---|
Everolimus (CNI-free) | 48.2 |
Control (CsA) | 46.1 |
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Measured Glomerular Filtration Rate
To compare the efficacy between treatment regimens by assessing the difference in renal function evaluated by mean measured glomerular filtration rate (mGFR) 12 months after renal transplantation (TX). The mGFR was measured using Iohexol or Cr-EDTA clearance according to local practice. (NCT00634920)
Timeframe: Month 12
Intervention | mL/min/1.73m^2 (Mean) |
---|
Everolimus (CNI-free) | 51.5 |
Control (CsA) | 47.8 |
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Time to First Malignancy
This is the time to first diagnosed malignancy. Malignancies (skin- or solid cancer) were listed whether they reoccurred in situ, were metastatic or de novo. This is shown as mean time. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Months (Mean) |
---|
Everolimus (CNI-free) | 35.5 |
Control (CsA) | 35.1 |
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Time to Treatment Failure
Treatment failure was defined as graft loss or death.Time to treatment failure is shown as mean time to treatment failure. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Days (Mean) |
---|
Everolimus (CNI-free) | 972.7 |
Control (CsA) | 959.5 |
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Calculated Glomerular Filtration Rate
The GFR was calculated according to the Modification of Diet in Renal Disease Study Group (MDRD) method, the Cockcroft-Gault method, and the Nankivell formula. cGFR was calculated from blood samples collected at predefined time points. (NCT00634920)
Timeframe: Months 12, 36
Intervention | mL/min/1.73m^2 (Mean) |
---|
| MDRD M12 | MDRD M36 | Cockcroft-Gault M12 | Cockcroft-Gault M36 | Nankivel M12 | Nankivel M36 |
---|
Control (CsA) | 60.1 | 57.4 | 45.6 | 42.1 | 61.8 | 58.9 |
,Everolimus (CNI-free) | 65.0 | 59.4 | 45.4 | 43.1 | 66.3 | 61.8 |
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Lipid Profile for Apolipoprotein
Blood lipid levels of patients in both groups for Apolipoprotein (Apo) A1 and B. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | g/L (Mean) |
---|
| Month 12: Apolipoprotein A1 | Month 24: Apolipoprotein A1 | Month 36: Apolipoprotein A1 | Month 12: Apolipoprotein B | Month 24: Apolipoprotein B ( | Month 36: Apolipoprotein B |
---|
Control (CsA) | 1.46 | 1.36 | 1.56 | 0.923 | 1.058 | 0.934 |
,Everolimus (CNI-free) | 1.59 | 1.55 | 1.70 | 0.935 | 1.178 | 0.984 |
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Lipid Profile for HDL-C, LDL-C,Total Cholesterol, and Triglycerides
Blood lipid levels of patients in both groups: HDL-C, LDL-C,Total cholesterol, and triglycerides. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | mmol/L (Mean) |
---|
| Month 12: HDL Cholesterol | Month 24: HDL Cholesterol | Month 36: HDL Cholesterol | Month 12: LDL Cholesterol | Month 24: LDL Cholesterol | Month 36: LDL Cholesterol | Month 12: Total Cholesterol | Month 24: Total Cholesterol | Month 36: Total Cholesterol | Month 12: Triglycerides | Month 24: Triglycerides | Month 36: Triglycerides |
---|
Control (CsA) | 1.419 | 1.409 | 1.529 | 3.130 | 2.925 | 2.822 | 5.318 | 5.112 | 4.830 | 1.868 | 1.757 | 1.580 |
,Everolimus (CNI-free) | 1.486 | 1.477 | 1.495 | 3.569 | 3.381 | 3.206 | 6.091 | 5.823 | 5.595 | 2.461 | 2.288 | 2.164 |
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Number of Antihypertensive Drugs Taken
(NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Number of antihypertensive dugs (Mean) |
---|
| Month 12 | Month 24 | Month 36 |
---|
Control (CsA) | 2.5 | 2.4 | 2.2 |
,Everolimus (CNI-free) | 2.5 | 2.5 | 2.0 |
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Number of Lipid-lowering Drugs Taken
(NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Number of lipid-lowering drugs (Mean) |
---|
| Month 12 | Month 24 | Month 36 |
---|
Control (CsA) | 0.8 | 0.9 | 0.8 |
,Everolimus (CNI-free) | 0.9 | 1.0 | 0.9 |
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Percentage of Participants on Antihypertensive Drugs
(NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Percentage of participants (Number) |
---|
| Month 12: No antihypertensive drugs | Month 12: Has antihypertensive drugs | Month 24: No antihypertensive drugs | Month 24: Has antihypertensive drugs | Month 36: No antihypertensive drugs | Month 36: Has antihypertensive drugs |
---|
Control (CsA) | 3.3 | 96.7 | 5.3 | 94.7 | 12.8 | 87.2 |
,Everolimus (CNI-free) | 9.2 | 90.8 | 4.2 | 95.8 | 15.6 | 84.4 |
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Percentage of Participants on Lipid-lowering Drugs
(NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Percentage of participants (Number) |
---|
| Month 12 | Month 24 | Month 36 |
---|
Control (CsA) | 60.0 | 65.0 | 63.0 |
,Everolimus (CNI-free) | 75.0 | 78.0 | 73.0 |
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Percentage of Participants Who Developed CAN (Chronic Allograft Nephropathy)
Assessed by protocol biopsies findings (Banff 1997 lesion scores and morphometry of the interstitial space) (NCT00634920)
Timeframe: Month 12, Month 36
Intervention | Percentage of participants (Number) |
---|
| Month 12 | Month 36 |
---|
Control (CsA) | 1.0 | 64.0 |
,Everolimus (CNI-free) | 1.0 | 59.0 |
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Percentage of Participants Who Had Donor Specific Antibodies (DSA)
Venous blood was drawn for donor specific (DSA) measurements prior to transplantation and at the final visit (36 months). The blood sample was first screened for the presence of PRA i.e. donor specific Immunoglobulin-G antibodies against specific HLA antigens. If PRA antibodies were detected, the blood sample was tested for specific DSAs on single antigen Luminex beads (coated with single HLA class I or II molecules). In this way, the specificity of these antibodies could be determined. (NCT00634920)
Timeframe: Month 36
Intervention | Percentage of participants (Number) |
---|
| ND (not done) | Negative | Positive |
---|
Control (CsA) | 9.0 | 70.0 | 21.0 |
,Everolimus (CNI-free) | 7.0 | 78.0 | 15.0 |
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Percentage of Participants With Biopsy Proven Acute Rejection (BPAR)
A BPAR was defined as a biopsy graded IA, IB, IIA, IIB, or III (Banff 97 classification). Biopsy graded IA: Significant interstitial infiltration (> 25% of parenchyma) and foci of moderate tubulitis (> 4 mononuclear cells/tubular cross section or group of 10 tubular cells). Biopsy grade IB: Significant interstitial infiltration (> 25% of parenchyma) and foci of severe tubulitis (> 10 mononuclear cells/tubular cross section or group of 10 tubular cells). Biopsy grade IIA: Mild to moderate intimal arteritis. Biopsy graded IIB: Severe intimal arteritis comprising > 25% of the lumenal area. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Percentage of participants (Number) |
---|
| Month 12: lA | Month 12: lB | Month 12: llA | Month 12: llB | Month 24: lA | Month 24: lB | Month 36: lA | Month 36: lB |
---|
Control (CsA) | 4.4 | 0.0 | 2.2 | 1.1 | 4.4 | 3.3 | 1.1 | 0.0 |
,Everolimus (CNI-free) | 19.6 | 10.9 | 2.2 | 2.2 | 5.4 | 1.1 | 2.2 | 1.1 |
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Percentage of Participants With Graft Loss or Death
The allograft was presumed to be lost on the day the patient started dialysis and was not able to subsequently be removed from dialysis. If the patient underwent a graft nephrectomy, the day of nephrectomy was the day of graft loss. Graft loss was considered an SAE (serious adverse event). (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Percentage of participants (Number) |
---|
| Month 12: Event First Year | Month 12: No Event First Year | Month 24: Event Second Year | Month 24: No Event Second Year | Month 36: Event Third Year | Month 36: No Event Third Year |
---|
Control (CsA) | 0.0 | 100.0 | 1.1 | 98.9 | 2.2 | 97.8 |
,Everolimus (CNI-free) | 0.0 | 100.0 | 1.1 | 98.9 | 0.0 | 100.0 |
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Percentage of Participants With Treatment Failures
Treatment failure was defined as graft loss or death. (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | Percentage of participants (Number) |
---|
| Month 12: No Failure | Month 12: Failure | Month 24: No Failure | Month 24: Failure | Month 36: No Failure | Month 36: Failure |
---|
Control (CsA) | 100.0 | 0.0 | 98.8 | 1.2 | 96.7 | 3.3 |
,Everolimus (CNI-free) | 100.0 | 0.0 | 98.8 | 1.2 | 98.8 | 1.2 |
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Progression of Measured Glomerular Filtration Rate
Change in renal progression measured by mean mGFR from week 7 to Month 36 (NCT00634920)
Timeframe: Week 7, Week 52, Month 36
Intervention | mL/min/1.73m^2 (Mean) |
---|
| Week 7 | Week 52 | Change from week 7 to Week 52 | Month 36 | Change from week 7 to Month 36 |
---|
Control (CsA) | 47.8 | 47.8 | 0.0 | 46.1 | -1.7 |
,Everolimus (CNI-free) | 46.3 | 51.5 | 5.6 | 48.2 | 1.3 |
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Proteinuria (Measured as Urine Albumin/Creatinine Ratio (mg/mmol))
"Proteinuria is when a large amount of protein, that should remain circulating in a person's blood, is spilled into their urine and eliminated from the body." (NCT00634920)
Timeframe: Months 12, 24, 36
Intervention | mg/mmol (Mean) |
---|
| Month 12 | Month 24 | Month 36 |
---|
Control (CsA) | 11.27 | 24.55 | 80.73 |
,Everolimus (CNI-free) | 17.31 | 62.83 | 78.78 |
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Number of Infectious Complications
The number of infections complications occurring among study participants is presented here. (NCT01921218)
Timeframe: Baseline up to Month 36
Intervention | complications (Number) |
---|
Belatacept Treatment Group | 12 |
Control Group | 18 |
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Number of Participants With Donor-specific Antibody Formation
The number of participants in each group with donor-specific antibody formation at 36 months following randomization. (NCT01921218)
Timeframe: Month 36
Intervention | Participants (Count of Participants) |
---|
Belatacept Treatment Group | 3 |
Control Group | 4 |
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Time to Initiation of Dialysis
Time to dialysis is measured as the time of randomization to initiation of dialysis. Participants already requiring dialysis at the time of enrollment were excluded from this endpoint analysis. (NCT01921218)
Timeframe: Up to Year 2
Intervention | months (Mean) |
---|
Belatacept Treatment Group | 11.75 |
Control Group | 10.5 |
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Glomerular Filtration Rate (GFR)
The glomerular filtration rate (GFR) assesses kidney function. GFR uses values for serum creatinine (SCr) measured in mg/dL, age in years, blood urea nitrogen (BUN) measures in mg/dL, and serum albumin (Alb) measured in g/dL. GFR is calculated as 170 x (SCr/0.95)^(-0.999) x (Age)^(-0.176) x (0.762 if the patient is female) x (1.180 if the patient is black) x (BUN)^(-0.170) x (Alb)^(0.318). A value of 90 or above is considered normal while values between 15 and 29 indicate severely decreased kidney function and values below 15 indicate kidney failure. The GFR in participants who do not require dialysis will be followed for two years. (NCT01921218)
Timeframe: Baseline up to Month 24
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| Baseline | Month 1 | Month 2 | Month 3 | Month 6 | Month 9 | Month 12 |
---|
Control Group | 14.5 | 11 | 10.5 | 16.5 | 14.5 | 13.5 | 20 |
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Glomerular Filtration Rate (GFR)
The glomerular filtration rate (GFR) assesses kidney function. GFR uses values for serum creatinine (SCr) measured in mg/dL, age in years, blood urea nitrogen (BUN) measures in mg/dL, and serum albumin (Alb) measured in g/dL. GFR is calculated as 170 x (SCr/0.95)^(-0.999) x (Age)^(-0.176) x (0.762 if the patient is female) x (1.180 if the patient is black) x (BUN)^(-0.170) x (Alb)^(0.318). A value of 90 or above is considered normal while values between 15 and 29 indicate severely decreased kidney function and values below 15 indicate kidney failure. The GFR in participants who do not require dialysis will be followed for two years. (NCT01921218)
Timeframe: Baseline up to Month 24
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| Baseline | Month 1 | Month 2 | Month 3 | Month 6 | Month 9 | Month 12 | Month 18 |
---|
Belatacept Treatment Group | 14.25 | 14.25 | 19.33 | 16.33 | 14.67 | 14.67 | 13.67 | 7 |
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Number of Participants With Anti-human Leukocyte Antigen (HLA) Alloantibodies
The presence of anti-HLA Class I and Class II alloantibodies is categorized as being negative (absent for both classes of alloantibodies), positive for Class I, positive for Class II, and positive for both Class I and Class II alloantibodies. (NCT01921218)
Timeframe: Baseline up to Month 36
Intervention | Participants (Count of Participants) |
---|
| Baseline - Negative | Baseline - Positive Class I | Baseline - Positive Class II | Baseline - Positive Class I and II | Month 12 - Negative | Month 12 - Positive Class I | Month 12 - Positive Class II | Month 12 - Positive Class I and II | Month 24 - Negative | Month 24 - Positive Class I | Month 24 - Positive Class II | Month 24 - Positive Class I and II | Month 36 - Negative | Month 36 - Positive Class I | Month 36 - Positive Class II | Month 36 - Positive Class I and II |
---|
Belatacept Treatment Group | 6 | 0 | 0 | 0 | 4 | 1 | 0 | 0 | 1 | 0 | 2 | 0 | 1 | 0 | 2 | 0 |
,Control Group | 5 | 2 | 0 | 0 | 2 | 4 | 3 | 3 | 1 | 2 | 1 | 1 | 1 | 2 | 1 | 1 |
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Delta Alanine Aminotransferase
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 9 |
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Delta Alkaline Phosphatase
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 13.8 |
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Delta Bilirubin
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | -6.0 |
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Delta Cholesterol Fasting Level
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 1.2 |
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Delta Hemoglobin
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | -2.7 |
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Delta Hepatitis C Viral Load
Percent change in HCV load determined 3 months after switch from MMF to SRL. (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 15 |
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Delta Platelet Count
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | -8.5 |
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Delta Tacrolimus Trough Level
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 23 |
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Delta Triglyceride Fasting Level
Percent change determined 3 months after switch from MMF to SRL (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | 23 |
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Final Hepatitis C Viral Load
Percent change in HCV load determined 3 months after switch from SRL to MMF (NCT01134952)
Timeframe: 3 month
Intervention | percent change (Mean) |
---|
MMF SRL Switch | -47 |
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Sirolimus Trough Level
(NCT01134952)
Timeframe: 3 month
Intervention | ng/ml (Mean) |
---|
MMF SRL Switch | 7.2 |
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Relapse Free Survival
Number of patients with relapsed disease within 1 Year post-transplant. Relapse is defined as the detection of > 5% blasts after a documented complete remission. (NCT00036738)
Timeframe: Assessed up to 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic Nonmyeloablative HSCT) | 3 |
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Leukemia-free Survival
Number of patients surviving in CR up to five years post-transplant. (NCT00036738)
Timeframe: Assessed up to 5 years
Intervention | Participants (Count of Participants) |
---|
| 6 Months | 1 Year | 2 Years | 3 Years | 4 Years | 5 Years |
---|
Treatment (Allogeneic Nonmyeloablative HSCT) | 19 | 17 | 13 | 11 | 11 | 10 |
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Overall Survival
Number of patients surviving up to five years post-transplant. (NCT00036738)
Timeframe: Assessed up to 5 years
Intervention | Participants (Count of Participants) |
---|
| 6 Months | 1 Year | 2 Years | 3 Years | 4 Years | 5 Years |
---|
Treatment (Allogeneic Nonmyeloablative HSCT) | 26 | 24 | 19 | 17 | 16 | 13 |
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Number of Participants Who Developed Acute Graft Versus Host Disease
(NCT00255684)
Timeframe: 3 months
Intervention | participants (Number) |
---|
Conditioning Therapy Followed by TBI | 0 |
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Number of Participants Who Survived 100 Days or Longer
(NCT00255684)
Timeframe: 100 days
Intervention | participants (Number) |
---|
Conditioning Therapy Followed by TBI | 13 |
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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
Intervention | percentage of participants (Number) |
---|
Mini-haplo BMT | 13 |
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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
Intervention | percentage of participants (Number) |
---|
Mini-haplo BMT | 9.5 |
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Progression-free Survival
Percentage of participants who do not experience disease relapse, disease progression, or death. (NCT00134004)
Timeframe: 2 years
Intervention | percentage of participants (Number) |
---|
Mini-haplo BMT | 34 |
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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
Intervention | percentage of participants (Number) |
---|
Mini-haplo BMT | 55 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 43 |
Arm 2 - No Prior Autologous Transplant | 32 |
Arm 3 - Refractory Leukemia/Lymphoma | 1 |
Arm 4: MT2006-01 Coenrolling Patients | 21 |
Arm 5 - Previous Autologous Transplant | 16 |
Arm 6 - No Prior Autologous Transplant | 24 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 36 |
Arm 2 - No Prior Autologous Transplant | 25 |
Arm 3 - Refractory Leukemia/Lymphoma | 1 |
Arm 4: MT2006-01 Coenrolling Patients | 17 |
Arm 5 - Previous Autologous Transplant | 16 |
Arm 6 - No Prior Autologous Transplant | 20 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 43 |
Arm 2 - No Prior Autologous Transplant | 14 |
Arm 3 - Refractory Leukemia/Lymphoma | 4 |
Arm 4: MT2006-01 Coenrolling Patients | 7 |
Arm 5 - Previous Autologous Transplant | 20 |
Arm 6 - No Prior Autologous Transplant | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 49 |
Arm 2 - No Prior Autologous Transplant | 19 |
Arm 3 - Refractory Leukemia/Lymphoma | 4 |
Arm 4: MT2006-01 Coenrolling Patients | 11 |
Arm 5 - Previous Autologous Transplant | 20 |
Arm 6 - No Prior Autologous Transplant | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 59 |
Arm 2 - No Prior Autologous Transplant | 40 |
Arm 3 - Refractory Leukemia/Lymphoma | 1 |
Arm 4: MT2006-01 Coenrolling Patients | 26 |
Arm 5 - Previous Autologous Transplant | 26 |
Arm 6 - No Prior Autologous Transplant | 25 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 50 |
Arm 2 - No Prior Autologous Transplant | 31 |
Arm 3 - Refractory Leukemia/Lymphoma | 1 |
Arm 4: MT2006-01 Coenrolling Patients | 20 |
Arm 5 - Previous Autologous Transplant | 21 |
Arm 6 - No Prior Autologous Transplant | 23 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 10 |
Arm 2 - No Prior Autologous Transplant | 20 |
Arm 3 - Refractory Leukemia/Lymphoma | 2 |
Arm 4: MT2006-01 Coenrolling Patients | 5 |
Arm 5 - Previous Autologous Transplant | 3 |
Arm 6 - No Prior Autologous Transplant | 6 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 45 |
Arm 2 - No Prior Autologous Transplant | 24 |
Arm 3 - Refractory Leukemia/Lymphoma | 1 |
Arm 4: MT2006-01 Coenrolling Patients | 12 |
Arm 5 - Previous Autologous Transplant | 13 |
Arm 6 - No Prior Autologous Transplant | 13 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 18 |
Arm 2 - No Prior Autologous Transplant | 20 |
Arm 3 - Refractory Leukemia/Lymphoma | 0 |
Arm 4: MT2006-01 Coenrolling Patients | 3 |
Arm 5 - Previous Autologous Transplant | 3 |
Arm 6 - No Prior Autologous Transplant | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 93 |
Arm 2 - No Prior Autologous Transplant | 65 |
Arm 3 - Refractory Leukemia/Lymphoma | 6 |
Arm 4: MT2006-01 Coenrolling Patients | 32 |
Arm 5 - Previous Autologous Transplant | 32 |
Arm 6 - No Prior Autologous Transplant | 29 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm 1-Previous Autologous Transplant | 75 |
Arm 2 - No Prior Autologous Transplant | 47 |
Arm 3 - Refractory Leukemia/Lymphoma | 3 |
Arm 4: MT2006-01 Coenrolling Patients | 28 |
Arm 5 - Previous Autologous Transplant | 34 |
Arm 6 - No Prior Autologous Transplant | 25 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1-Previous Autologous Transplant | 94 |
Arm 2 - No Prior Autologous Transplant | 94 |
Arm 3 - Refractory Leukemia/Lymphoma | 100 |
Arm 4: MT2006-01 Coenrolling Patients | 93 |
Arm 5 - Previous Autologous Transplant | 85 |
Arm 6 - No Prior Autologous Transplant | 86 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1-Previous Autologous Transplant | 96 |
Arm 2 - No Prior Autologous Transplant | 98 |
Arm 3 - Refractory Leukemia/Lymphoma | 88 |
Arm 4: MT2006-01 Coenrolling Patients | 94 |
Arm 5 - Previous Autologous Transplant | 91 |
Arm 6 - No Prior Autologous Transplant | 98 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1-Previous Autologous Transplant | 77 |
Arm 2 - No Prior Autologous Transplant | 73 |
Arm 3 - Refractory Leukemia/Lymphoma | 57 |
Arm 4: MT2006-01 Coenrolling Patients | 77 |
Arm 5 - Previous Autologous Transplant | 69 |
Arm 6 - No Prior Autologous Transplant | 68 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1-Previous Autologous Transplant | 99 |
Arm 2 - No Prior Autologous Transplant | 98 |
Arm 4: MT2006-01 Coenrolling Patients | 99 |
Arm 5 - Previous Autologous Transplant | 87 |
Arm 6 - No Prior Autologous Transplant | 100 |
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Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)
"This outcome measure is used to determine if the reduction of calcineurin inhibitor immunosuppression leads to improved native kidney function. Native kidney function is assessed using the Modification of Diet in Renal Disease (MDRD) estimation of glomerular filtration rate (GFR) from serum or plasma creatinine samples at the reported time points.~Reference intervals include:~Healthy 18 years and up: 60-120 mL/min/1.73 sqm Chronic kidney disease: GFR < 60 mL/min/1.73 sqm Kidney failure: GFR < 15 mL/min/1.73 sqm" (NCT01230502)
Timeframe: 12 months post enrollment/randomization
Intervention | mL/min/1.73 sqm (Mean) |
---|
Group 3: Donor Specific Regulation (DSR) -, Standard of Care | 82.4 |
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Modification of Diet in Renal Disease (MDRD) Estimation of Glomerular Filtration Rate (GFR)
"This outcome measure is used to determine if the reduction of calcineurin inhibitor immunosuppression leads to improved native kidney function. Native kidney function is assessed using the Modification of Diet in Renal Disease (MDRD) estimation of glomerular filtration rate (GFR) from serum or plasma creatinine samples at the reported time points.~Reference intervals include:~Healthy 18 years and up: 60-120 mL/min/1.73 sqm Chronic kidney disease: GFR < 60 mL/min/1.73 sqm Kidney failure: GFR < 15 mL/min/1.73 sqm" (NCT01230502)
Timeframe: 6 months post enrollment/randomization
Intervention | mL/min/1.73 sqm (Mean) |
---|
Group 3: DSR (-), Standard of Care | 60.14 |
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Change in Best Spectacle-corrected Visual Acuity (BSCVA)
Change in best spectacle-corrected visual acuity (BSCVA) from baseline. Analysis on eye level (NCT01232920)
Timeframe: 6 months
Intervention | LogMAR (Mean) |
---|
Methotrexate | -0.26 |
Mycophenolate Mofetil | -0.19 |
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Number of Participants Achieving Treatment Success
"TREATMENT SUCCESS is defined as controlled ocular inflammation in both eyes with less than or equal to 10 mg/day of prednisone and/or 2 topical steroid drops/day sustained for 2 visits separated by at least 28 days (control of inflammation and prednisone dose must be achieved by 5-month visit and sustained until 6-month visit).~Discontinuation of study medication at any time due to efficacy, tolerability, or safety may result in a declaration of TREATMENT FAILURE. Note that all patients will be classified as either a treatment success or failure." (NCT01232920)
Timeframe: 6 months
Intervention | participants (Number) |
---|
Methotrexate | 24 |
Mycophenolate Mofetil | 15 |
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Time to Control of Inflammation
(NCT01232920)
Timeframe: 6 months
Intervention | days (Median) |
---|
Methotrexate | 139 |
Mycophenolate Mofetil | 124 |
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Number of Eyes With Resolution of Macular Edema
(NCT01232920)
Timeframe: 6 months
Intervention | Eyes (Number) |
---|
| Eyes with Macular Edema at Baseline | Eyes with Resolved Macular Edema |
---|
Methotrexate | 22 | 17 |
,Mycophenolate Mofetil | 13 | 7 |
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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
Intervention | Days (Median) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 16 |
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Hematopoietic Toxicity
Clinical sequelae due to hematopoietic toxicity as defined by incidences of neutropenic fever and bleeding. (NCT01434472)
Timeframe: Up to day 100
Intervention | Incidences (Number) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 5 |
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Overall Survival
(NCT01434472)
Timeframe: Up to 2 years post transplant
Intervention | Participants (Count of Participants) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 14 |
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Platelet Engraftment
The median time in days to achieve platelet recovery as defined as platelet >20,000uL. (NCT01434472)
Timeframe: Up to day 100
Intervention | Days (Median) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 9 |
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Progression-free Survival
Based on a one-sample chi-square test with one-sided significance level of five percent. This study will be deemed successful if the 1 year progression-free survival of this highest-risk group of patients is 54% or greater. (NCT01434472)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 11 |
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Response Rates
Response is defined as having achieved a Partial Response or better. (NCT01434472)
Timeframe: Day 100
Intervention | Participants (Count of Participants) |
---|
Treatment (Radiolabeled Antibody, TBI, Allogeneic PBSCT) | 16 |
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BANFF Grades of First AMR.
Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 6 months post-transplantation
Intervention | Participants (Count of Participants) |
---|
Experimental | 0 |
Control | 0 |
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Change in eGFR Between 3 Months and 24 Months as Measured by CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 3 and 24 was calculated as the month 24 eGFR minus the month 3 eGFR for each participant. A window of +/- 14 days was used for month 3 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 3 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 2.7 |
Control | 4.4 |
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Change in eGFR Between 3 Months and 24 Months as Measured by MDRD
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 3 and 24 was calculated as the month 24 eGFR minus the month 3 eGFR for each participant. A window of +/- 14 days was used for month 3 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 3 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 2.8 |
Control | 4.8 |
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Change in eGFR Between 6 Months and 24 Months as Measured by CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 6 and 24 was calculated as the month 24 eGFR minus the month 6 eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 0.8 |
Control | 4.8 |
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Change in eGFR Between 6 Months and 24 Months as Measured by MDRD
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. The change in eGFR between months 6 and 24 was calculated as the month 24 eGFR minus the month 6 eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 1.0 |
Control | 5.2 |
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Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. Post-transplant nadir was defined as the lowest value of eGFR from the first 6 months post-transplant. The change in eGFR between nadir and month 24 was calculated as the month 24 eGFR minus the nadir eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 8.5 |
Control | 12.0 |
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Change in eGFR Between Post-transplant Nadir and 24 Months as Measured by MDRD
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. Post-transplant nadir was defined as the lowest value of eGFR from the first 6 months post-transplant. The change in eGFR between nadir and month 24 was calculated as the month 24 eGFR minus the nadir eGFR for each participant. A window of +/- 21 days was used for month 6 and +/- 1 month was used for month 24. (NCT02495077)
Timeframe: 6 months and 24 months post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 8.1 |
Control | 11.6 |
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Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 2 Divided by he First Creatinine After Surgery
Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 2 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. (NCT02495077)
Timeframe: Day 2 post-transplantation
Intervention | Percentage (Mean) |
---|
Experimental | 24.28 |
Control | 20.97 |
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Creatinine Reduction Ratio (CRR), Defined as the First Creatinine on Day 5 Divided by the First Creatinine After Surgery.
Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 5 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. (NCT02495077)
Timeframe: Day 5 post-transplantation
Intervention | Percentage (Mean) |
---|
Experimental | 47.06 |
Control | 43.37 |
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Days From Transplantation Until Event (ACR, AMR, or Hospitalization for Infection and/or Malignancy)
Participants are considered to have met this endpoint if they experienced biopsy-proven T-cell mediated rejection (ACR) or antibody mediated rejection (AMR) based on central pathology reading or were hospitalized for infection and/or malignancy. For participants who met one or more of these three components, the earliest event date of the three components was used as the time of meeting the endpoint. Participants who did not meet any of the three components were censored at their last date of follow-up. Event (or censor) day was calculated as event (or censor) date minus transplant date. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | Days to event (Median) |
---|
Experimental | 642 |
Control | 613 |
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Duration of Delayed Graft Function (DGF), Defined as Time From Transplantation to the Last Required Dialysis Treatment.
Participants are considered to have had DGF if they had at least one dialysis treatment in the first week post-transplant. For this endpoint, duration was calculated as the date of last post-transplant dialysis treatment minus the date of the first post-transplant dialysis treatment. (NCT02495077)
Timeframe: First post-transplant dialysis treatment to last post-transplant dialysis treatment
Intervention | Days (Mean) |
---|
Experimental | 13.27 |
Control | 15.74 |
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eGFR Values as Measured by CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Day 7 post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 41.01 |
Control | 41.85 |
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eGFR Values as Measured by MDRD
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Day 7 post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 38.93 |
Control | 39.96 |
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Number of Dialysis Sessions.
The number of dialysis sessions a person had during their first 8 weeks post-transplant was used for this endpoint. (NCT02495077)
Timeframe: 8 weeks post-transplantation
Intervention | Dialysis sessions (Mean) |
---|
Experimental | 0.14 |
Control | 0.26 |
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Percent of Participants That Required at Least One Dialysis Treatment.
Dialysis within the first week post-transplant is used in the setting of delayed graft function (DGF). Participants are considered to have had DGF if they had at least one dialysis treatment in the first week post-transplant. (NCT02495077)
Timeframe: 1 week post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 31.0 |
Control | 35.7 |
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Percent of Participants With Any Infection Requiring Hospitalization or Resulting in Death.
Participants were considered to have met this endpoint if they had an infection that required hospitalization or resulted in death. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 43.0 |
Control | 39.3 |
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Percent of Participants With BANFF Chronicity Scores > or Equal 2 on the 24 Month Biopsy.
The Banff 2013 classification involves scoring numerous characteristics of renal biopsy specimens. The ci (interstitial fibrosis) and ct (tubular atrophy) scores are two such characteristics. The scores can take values of 0, 1, 2, or 3 for each characteristic (ci and ct), indicating increasing severity of disease as the scores increase. Participants are considered to have met this endpoint if their ci + ct score on the 24 month biopsy summed to be > or equal to 2. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 73.1 |
Control | 36.4 |
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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR)
Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive.Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 6 months post-transplantation
Intervention | Participants (Count of Participants) |
---|
Experimental | 0.0 |
Control | 0.0 |
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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection (AMR).
Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR within 24 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, or C4d staining positive. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participant (Number) |
---|
Experimental | 1.3 |
Control | 0.0 |
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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR
Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR or suspicious for AMR within 6 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, C4d staining positive, or suspicious. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes; suspicious-when 2 of 3 factors for acute/active are present. (NCT02495077)
Timeframe: 6 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 2.8 |
Control | 0.0 |
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Percent of Participants With Biopsy Proven Acute Antibody Mediated Rejection AMR or Suspicious for AMR.
Antibody mediated rejection (AMR) was defined based on central lab pathology interpretation using the Banff 2013 criteria. Participants with a Banff finding of AMR or suspicious for AMR within 24 months of transplant were determined to have met the endpoint. AMR is classified as acute/active, chronic/active, C4d staining positive, or suspicious. Criteria include: acute/active-histologic evidence of acute tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of donor-specific antibodies (DSAs); chronic/active-morphologic evidence of chronic tissue injury, evidence of current/recent antibody interaction with vascular endothelium, and serologic evidence of DSAs; C4d staining positive-linear C4d staining in peritubular capillaries, glomerulitis=0, peritubular capillary=0, chronic glomerulopathy=0, no acute cell-mediated rejection or borderline changes; suspicious-when 2 of 3 factors for acute/active are present (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 3.8 |
Control | 1.4 |
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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR)
Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of greater than or equal to IA with or without clinical symptoms within 6 months of transplant were determined to have met the endpoint. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection.Criteria include: IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 6 month post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 4.2 |
Control | 3.0 |
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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection
Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of borderline or greater than or equal to IA with or without clinical symptoms within 24 months of transplant were determined to have met the endpoint. Severity is graded as Borderline, IA, IB, IIA, IIB, or III, with borderline representing possible cellular rejection, IA being the mildest form of cellular rejection, and III being the most severe form of cellular rejection. Criteria include: Borderline-no intimal arteritis is present but foci of mild tubulitis; IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 12.8 |
Control | 7 |
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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR) or Borderline Rejection.
Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of borderline or greater than or equal to IA with or without clinical symptoms within 6 months of transplant were determined to have met the endpoint. Severity is graded as Borderline, IA, IB, IIA, IIB, or III, with borderline representing possible cellular rejection, IA being the mildest form of cellular rejection, and III being the most severe form of cellular rejection.Criteria include: Borderline-no intimal arteritis is present but foci of mild tubulitis; IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 6 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 8.5 |
Control | 6.1 |
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Percent of Participants With Biopsy Proven Acute Cellular Rejection (BPAR).
Acute cellular rejection was defined based on central lab pathology interpretation using the Banff 2007 criteria. Participants with a Banff grade of greater than or equal to IA with or without clinical symptoms within 24 months of transplant were determined to have met the endpoint. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Criteria include: IA-significant interstitial infiltration and foci of moderate tubulitis; IB-significant interstitial infiltration and foci of severe tubulitis; IIA-mild to moderate intimal arteritis; IIB-severe intimal arteritis; III-transmural arteritis and/or arterial fibrinoid change and necrosis of medial smooth muscle cells with accompanying lymphocytic inflammation. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 5.1 |
Control | 4.2 |
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Percent of Participants With BK Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site.
Participants were considered to have met this endpoint if they had a reported case of BK viremia that required a change in their existing immunosuppression or the use of anti-viral therapy. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | Percent of participants (Number) |
---|
Experimental | 28.9 |
Control | 13.4 |
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Percent of Participants With CMV Viremia That Require a Change in Immunosuppression or Anti-viral Treatment as Per Standard of Care at the Site
Participants were considered to have met this endpoint if they had a reported case of CMV viremia that required a change in their existing immunosuppression or the use of anti-viral therapy. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 18.4 |
Control | 11.6 |
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Percent of Participants With de Novo DSA.
Donor specific antibody (DSA) can be formed post-transplant as part of the recipient's alloimmune response to the transplanted organ. DSA was determined by a central laboratory. Participants with newly developed DSA (i.e., de novo) following transplant were considered to have met this endpoint. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 8.0 |
Control | 3.6 |
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Percent of Participants With Death or Graft Failure.
Participants who died or experienced graft failure were considered to have met this endpoint. Graft failure was defined as the need for post-transplant dialysis for more than 56 days. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 5.3 |
Control | 7.1 |
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Percent of Participants With Impaired Wound Healing Manifested by Wound Dehiscence, Wound Infection, or Hernia at the Site of the Transplant Incision
Participants were considered to have met this endpoint if they had a reported case of impaired wound healing at the site of the transplant incision manifested by one wound dehiscence, wound infection, or hernia. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | Percent of participants (Number) |
---|
Experimental | 7.9 |
Control | 11.6 |
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Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.
Biopsies were read by the local pathologist at the hospital where the participant was a patient. These local reads informed clinical care for the participant, which may or may not include prescribing/administering medication to the participant to help with clinical concerns or findings noted on a biopsy. Participants were considered to have met this endpoint if they have a report of receiving treatment for clinical or biopsy-proven rejection during the 24 month post-transplant follow-up. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 16.2 |
Control | 27 |
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Percent of Participants With Locally Treated Rejection, Defined as Treatment Administered for Rejection Based on Clinical Signs or Biopsy Findings.
Biopsies were read by the local pathologist at the hospital where the participant was a patient. These local reads informed clinical care for the participant, which may or may not include prescribing/administering medication to the participant to help with clinical concerns or findings noted on a biopsy. Participants were considered to have met this endpoint if they have a report of receiving treatment for clinical or biopsy-proven rejection during the first 6 months post-transplant. (NCT02495077)
Timeframe: 6 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 12.6 |
Control | 20.5 |
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Percent of Participants With Malignancy.
Participants were considered to have met this endpoint if they had a reported case of malignancy. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | Percent of Participants (Number) |
---|
Experimental | 1.8 |
Control | 0.9 |
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Percent of Participants With Mycobacterial or Fungal Infections
Participants were considered to have met this endpoint if they had at least one mycobacterial of fungal infection. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 6.1 |
Control | 6.3 |
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Percent of Participants With Only Graft Failure.
Participants who experienced graft failure were considered to have met this endpoint. Graft failure was defined as the need for post-transplant dialysis for more than 56 days. (NCT02495077)
Timeframe: 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 2.7 |
Control | 2.7 |
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Percent of Participants With Primary Non-Function (PNF), Defined as Dialysis-dependency for More Than 3 Months.
Post-transplant dialysis is sometimes required in the setting of kidney transplant. If such dialysis continues for more than 3 months, the participant is considered to have PNF and, as such, meets this endpoint definition. (NCT02495077)
Timeframe: Transplantation through at least month 3 up to month 24
Intervention | Percent of Participants (Number) |
---|
Experimental | 2.8 |
Control | 0.9 |
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The Difference Between the Mean eGFR (Modified MDRD) in the Experimental vs. Control Groups.
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the month 24 eGFR for each treatment group. (NCT02495077)
Timeframe: 24-Month post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
Experimental | 52.45 |
Control | 57.35 |
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The Percent of Participants Who Need Dialysis After Week 1.
Participants who needed dialysis after the first week post-transplant were considered to have met this endpoint. (NCT02495077)
Timeframe: 1 week to 24 months post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 9.0 |
Control | 2.8 |
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The Percent of Participants Whose Day 2 Serum CRR Was Less Than 30%.
Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 2 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. A participant was considered to have met this endpoint if their day 2 serum CRR was less than 30%. (NCT02495077)
Timeframe: Day 2 post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 57.1 |
Control | 68.6 |
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The Percent of Participants Whose Day 5 Serum CRR Was Less Than 70%.
Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. CRR was calculated as the day 1 post-transplant creatinine value minus the day 5 creatinine value divided by the day 1 creatinine value and multiplied by 100, resulting in a percentage. Higher numbers indicate a greater reduction in serum creatinine and, thus, potentially better kidney function. A participant was considered to have met this endpoint if their day 5 serum CRR was less than 70%. (NCT02495077)
Timeframe: Day 5 post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 74.4 |
Control | 88.4 |
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The Percent of Participants With a Serum Creatinine of More Than 3 mg/dL.
Serum creatinine (mg/dL) is used to measure kidney function. A normal result is 0.7 to 1.3 mg/dL for men and 0.6 to 1.1 mg/dL for women. Higher results indicate poorer kidney function, as creatinine is removed from the body by the kidneys. This endpoint is ascertaining slow graft function in the immediate days post-transplant. A participant was considered to have met this endpoint if their day 5 serum creatinine was greater than 3 mg/dL. (NCT02495077)
Timeframe: Day 5 post-transplantation
Intervention | percentage of participants (Number) |
---|
Experimental | 47.4 |
Control | 42.9 |
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eGFR Values as Measured by CKD-EPI
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Days 30, 90, and 180 post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
| Day 30 | Day 90 | Day 180 |
---|
Control | 50.63 | 51.44 | 52.65 |
,Experimental | 49.29 | 49.80 | 50.56 |
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eGFR Values as Measured by MDRD
Glomerular filtration rate (GFR) is a measure of kidney function and helps determine the stage of kidney disease. eGFR was estimated using the Modification of Diet in Renal Disease (MDRD) equation. A value of 90+ means kidney function is normal. A value between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease. Values between 30 and 59 indicates moderately reduced kidney function. Values between 15 and 29 indicate severely reduced kidney function. Values below 15 indicate very severe or endstage kidney failure. eGFR values from day 7 and months 1, 3, 6, 12, 18, and 24 were used to generate an estimate of the eGFR at each time point of interest for each treatment group. (NCT02495077)
Timeframe: Days 30, 60, and 180 post-transplantation
Intervention | mL/min/1.73m2 (Mean) |
---|
| Day 30 | Day 90 | Day 180 |
---|
Control | 48.20 | 48.99 | 50.16 |
,Experimental | 46.64 | 47.14 | 47.89 |
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Acute Rejection
Number of subjects who experience acute rejection of the renal allograft. (NCT01653847)
Timeframe: 12 months post transplant
Intervention | Participants (Count of Participants) |
---|
Group 1: Tacrolimus With MMF. | 4 |
Group 2: Tacrolimus With Everolimus | 0 |
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Patient Survival
The number of patients who were alive at 2 years post transplant (NCT01653847)
Timeframe: baseline - 24 months post transplant
Intervention | Participants (Count of Participants) |
---|
Group 1: Tacrolimus With MMF. | 20 |
Group 2: Tacrolimus With Everolimus | 20 |
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Renal Allograft Survival
The number of subjects with renal allograft survival. (NCT01653847)
Timeframe: 12 months post-transplant
Intervention | Participants (Count of Participants) |
---|
Group 1: Tacrolimus With MMF. | 20 |
Group 2: Tacrolimus With Everolimus | 20 |
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Change in Glomerular Filtration Rate (GFR)
Evaluate the change in graft function (as measured by GFR) at 12 months post-transplant from baseline. (NCT01653847)
Timeframe: 3 months, 6 months, and 12 months post-transplant
Intervention | ml/minutes per 1.73 meters^2 (Mean) |
---|
| 3 months | 6 months | 12 months |
---|
Group 1: Tacrolimus With MMF. | 63 | 64 | 65 |
,Group 2: Tacrolimus With Everolimus | 66 | 64 | 72 |
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Change in T Cell & B Cell Generation
Evaluate the change in regulatory T cell generation and review the relationship of the newly generated T cells with their function in the two maintenance immunosuppressive regimens at baseline, 3 and 12 months post-transplant. (NCT01653847)
Timeframe: Baseline, 3 months, and 12 months post-transplant
Intervention | Mean % of Treg cells in peripheral blood (Mean) |
---|
| Baseline | 3 Months | 12 Months |
---|
Group 1: Tacrolimus With MMF. | 1.05 | 0.8 | 0.81 |
,Group 2: Tacrolimus With Everolimus | 0.93 | 1.12 | 1.18 |
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Percent of Participants With Adverse Events (AEs) Attributable to the Donor Alloantigen Reactive Tregs (darTregs) Infusion
"AEs classified by the site investigator/clinician as possibly or definitely related to the study treatment, the Donor Alloantigen Reactive Tregs (darTregs) infusion. These AEs include:~infusion reaction~Grade 3 or higher cytokine release syndrome (Reference: National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009) grading criteria~malignant cellular transformation." (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation
Intervention | Percent of Participants (Number) |
---|
Cohort 2 | 0 |
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Percent of Participants With Grade 3 or Higher Infectious Adverse Event(s)
"The severity of infectious adverse events (AEs) was classified into grades as follows:~Grade 1 = asymptomatic; clinical or diagnostic observation only; intervention with oral antibiotic, antifungal, or antiviral agent only; no invasive intervention required~Grade 2 = symptomatic; intervention with intravenous antibiotic, antifungal, or antiviral agent; invasive intervention may be required~Grade 3 = any infection associated with hemodynamic compromise requiring pressors; any infection necessitating intensive care unit level of care; any infection necessitating operative intervention; any infection involving the central nervous system; any infection with a positive fungal blood culture; any proven or probable aspergillus infection; any tissue invasive fungal infection; any pneumocystis jiroveci infection~Grade 4 = life-threatening infection~Grade 5 = death resulting from infection" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation
Intervention | Percent of Participants (Number) |
---|
Cohort 1 | 0 |
Cohort 2 | 11.1 |
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Percent of Participants With Grade 3 or Higher Wound Complication(s) Adverse Event(s)
"The severity of adverse events (AEs) was classified into grades using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009):~Grade 3 wound complications are defined as Hernia without evidence of strangulation; fascial disruption/dehiscence; primary wound closure or revision by operative intervention indicated~Grade 4 complications are defined as Hernia with evidence of strangulation; major reconstruction flap, grafting, resection, or amputation indicated" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation
Intervention | Percent of Participants (Number) |
---|
Cohort 1 | 0 |
Cohort 2 | 0 |
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Percent of Participants With Biopsy-Proven Acute and/or Chronic Rejection
"Biopsy-proven acute rejection graded as Mild, Moderate or Severe, per 1997 Banff classification. Chronic Rejection graded using Banff 2000 classification.~References: 1.) Banff Schema for Grading Liver Allograft Rejection: An International Consensus Document developed by an international panel of experts in liver transplantation pathology, hepatology, and surgery (Hepatology 1997; 25(3): 658-663). 2.) Update of the International Banff Schema for Liver Allograft Rejection: Working Recommendations for the Histopathologic Staging and Reporting of Chronic Rejection (Hepatology 2000; 31(3): 792-799)." (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation
Intervention | Percent of Participants (Number) |
---|
| Mild Acute Rejection | Moderate Acute Rejection | Severe Acute Rejection | Chronic Rejection |
---|
Cohort 1 | 0 | 0 | 0 | 0 |
,Cohort 2 | 11.1 | 0 | 0 | 0 |
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Percent of Participants With Grade 2 or Higher Hematologic Adverse Events (AEs) of Anemia, Neutropenia, and/or Thrombocytopenia
"The severity of adverse events (AEs) was classified into grades using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.0 (4/28/2009):~Grade 1 = mild AE~Grade 2 = moderate AE~Grade 3 = severe and undesirable AE~Grade 4 = life-threatening or disabling AE~Grade 5 = death" (NCT02188719)
Timeframe: Transplantation to 40 Weeks Post Transplantation
Intervention | Percent of Participants (Number) |
---|
| Anemia | Neutropenia | Thrombocytopenia |
---|
Cohort 1 | 66.7 | 16.7 | 16.7 |
,Cohort 2 | 22.2 | 0 | 0 |
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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)
Intervention | Days (Mean) |
---|
Enrolled, Transplanted | 9.5 |
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Duration in Days of Graft-versus-Host Disease in Transplanted Participants
Graft-versus-host disease (GVHD) is a medical complication that can occur after a person receives transplanted tissue, most commonly occurring after a bone marrow transplant. The white blood cells from the donated tissue recognize the tissue recipient's cells as foreign. These donor cells then attack the recipient's cells. Duration (in days) is measured as the time from the start of the GVHD event to the end of the GVHD event. (NCT02029638)
Timeframe: Transplant to Two Years Post-Transplant
Intervention | Days (Mean) |
---|
Enrolled, Transplanted | 7 |
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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
Intervention | Days (Mean) |
---|
Enrolled, Transplanted | 15 |
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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
Intervention | Days (Mean) |
---|
Enrolled, Transplanted | 36 |
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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)
Intervention | Days (Mean) |
---|
Enrolled, Transplanted | 23 |
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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
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 1 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 0 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 0 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 0 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 0 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 1 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 1 |
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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)
Intervention | Participants (Count of Participants) |
---|
Enrolled, Transplanted | 0 |
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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
Intervention | Percent of participants (Number) |
---|
Enrolled, Transplanted | 0 |
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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)
Intervention | participants (Number) |
---|
| Acute Cellular Rejection Banff Grade IA | Acute Cellular Rejection Banff Grade IB | Acute Cellular Rejection Banff Grade IIA | Acute Cellular Rejection Banff Grade IIB | Acute Cellular Rejection Banff Grade III | Acute Antibody Mediated Rejection |
---|
Enrolled, Transplanted | 0 | 0 | 0 | 0 | 0 | 0 |
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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)
Intervention | Events (Number) |
---|
| Grade 1 | Grade 2 | Grade 3 | Grade 4 | Grade 5 |
---|
Enrolled, Not Transplanted | 0 | 0 | 0 | 0 | 0 |
,Enrolled, Transplanted | 0 | 0 | 2 | 0 | 0 |
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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)
Intervention | Events (Number) |
---|
| Infection | Wound complications | Post-transplant diabetes | Hemorrhagic cystitis | Malignancy |
---|
Enrolled, Not Transplanted | 0 | 0 | 0 | 0 | 0 |
,Enrolled, Transplanted | 2 | 0 | 0 | 0 | 0 |
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Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach
(NCT01982682)
Timeframe: Up to 1 year after HSCT
Intervention | Participants (Count of Participants) |
---|
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT) | 27 |
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Count of Participants That Experienced Death as a Result of Graft-versus-host Disease (GVHD)
"Graft versus host disease was clinically characterized based on 4 stages of progression for three major body areas, skin, liver, gut. Subjects who experienced life threatening reactions in their skin, liver and gut ultimately experienced functional impairment and expired.~Life threatening reactions in the Skin were characterized by desquamation (the shedding of the outer layers of skin) and bullae (a bubblelike cavity filled with air or fluid, in particular).~Life threatening reactions in the Liver were characterized by Bilirubin, > 15 mg/dl Life threatening reactions in the Gut were characterized by Pain +/- ileus (a painful obstruction of the ileum or other part of the intestine.)" (NCT01982682)
Timeframe: Up to 1 year after HSCT
Intervention | Participants (Count of Participants) |
---|
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT) | 2 |
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Number of Participants With Successful Engraftment
Will be reported descriptively. Successful engraftment is defined as ANC (absolute neutrophil count, the number of white blood cells (WBCs) that are neutrophils) ≥ 0.5x109/L for at least 30 days and Platelet engraftment > 20,000 with no transfusion x 7 days. (NCT01982682)
Timeframe: Up to 1 year after HSCT
Intervention | Participants (Count of Participants) |
---|
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT) | 37 |
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Median Pace of T Cell Immune Recovery at 28 Days Post Hematopoietic Stem Cell Transplantation (HSCT)
An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 28 days will be monitored by collecting cluster of differentiation (CD24) and (CD38). (NCT01982682)
Timeframe: At 28 days post HSCT
Intervention | cells/ul (Median) |
---|
| Median CD3/4 count at d+28 | Median cluster of diff. 38 (CD3/8) count at d+28 |
---|
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT) | 41.3 | 48 |
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Median Pace of T Cell Immune Recovery at 90 Days Post Hematopoietic Stem Cell Transplantation (HSCT)
An excess amount of residual T-lymphocytes in the Peripheral Blood Stem Cell Collection (PBSC) product may increase the risk of GVHD. The ideal amount of T-cells left in the PBSC product is no greater than 5x104/kg. Every effort will be made to keep T-cell amounts to below this threshold. The median pace of T-cell immune recovery at 90 days will be monitored by collecting cluster of differentiation (CD24) and (CD38). (NCT01982682)
Timeframe: 90 days post HSCT
Intervention | cells/ul (Median) |
---|
| Median CD3/4 count at d+90 | Median CD3/8 count at d+90 |
---|
Treatment (TBI, DLI, Cyclophosphamide, CD34+ Donor HSCT) | 141 | 384 |
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Cumulative Oral Corticosteroid Dose
(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)
Intervention | milligram (mg) (Median) |
---|
Rituximab (RTX) | 2775.00 |
Mycophenolate Mofetil (MMF) | 4005.00 |
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Percentage of Participants (Excluding Telemedicine [TM] Participants) Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score
(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to clinical cut-off date (CCOD) of 28 November 2018)
Intervention | Percentage of Participants (Number) |
---|
Rituximab (RTX) | 40.3 |
Mycophenolate Mofetil (MMF) | 9.5 |
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Percentage of Participants With Anti-Drug Antibodies (ADA)
Participants with treatment-induced and treatment-enhanced anti-drug antibodies. The clinical relevance of anti-rituximab antibody formation in RITUXAN treated pemphigus vulgaris (PV) participants is unclear. (NCT02383589)
Timeframe: Baseline up to 52 Weeks (up to CCOD of 28 November 2018)
Intervention | Percentage of Participants (Number) |
---|
Rituximab (RTX) | 31.7 |
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Time to Initial Sustained Complete Remission
(NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)
Intervention | Weeks (Median) |
---|
Rituximab (RTX) | NA |
Mycophenolate Mofetil (MMF) | NA |
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Time to Protocol Defined Disease Flare
Disease flare is defined as the appearance of three or more new lesions a month that do not heal spontaneously within 1 week or by the extension of established lesions in a participant who has achieved disease control. (NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)
Intervention | Weeks (Median) |
---|
Rituximab (RTX) | NA |
Mycophenolate Mofetil (MMF) | NA |
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Total Number of Protocol Defined Disease Flares
Disease flare is defined as appearance of three or more new lesions a month that do not heal spontaneously within 1 week, or by the extension of established lesions, in a participant who has achieved disease control. (NCT02383589)
Timeframe: From Baseline up to 52 Weeks (up to CCOD of 28 November 2018)
Intervention | Number of Flares (Number) |
---|
Rituximab (RTX) | 6 |
Mycophenolate Mofetil (MMF) | 44 |
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Percentage of Participants With Immunoglobulin (Ig) Levels Below Lower Limit of Normal (LLN)
(NCT02383589)
Timeframe: Baseline; Weeks 16, 24, 40 and 52; (end of treatment: up to Week 52) (up to CCOD of 28 November 2018)
Intervention | Percentage of Participants (Number) |
---|
| Baseline (IgA) | Week 16 (IgA) | Week 24 (IgA) | Week 40 (IgA) | Week 52 (IgA) | Baseline (IgG) | Week 16 (IgG) | Week 24 (IgG) | Week 40 (IgG) | Week 52 (IgG) | Baseline (IgM) | Week 16 (IgM) | Week 24 (IgM) | Week 40 (IgM) | Week 52 (IgM) |
---|
Mycophenolate Mofetil (MMF) | 0 | 1.8 | 2.2 | 2.7 | 3.6 | 6.0 | 1.8 | 2.2 | 0 | 0 | 11.9 | 23.2 | 28.3 | 24.3 | 28.6 |
,Rituximab (RTX) | 0 | 0 | 1.7 | 0 | 0 | 6.1 | 9.8 | 3.4 | 3.5 | 4.3 | 7.6 | 24.6 | 27.1 | 29.8 | 29.8 |
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Recipient and Donor Genotyping for Selected Variants of CYP3A5, ABCB1 (MDR1), and CYP4A Genes
A blood sample was obtained from recipients and donors to measure gene polymorphism effects on metabolism of calcineurin inhibitor and everolimus. The polymorphisms are represented as the number of SNP occurrences for the CYP3A5, ABCB1 (MDR1) gene, and CYP4A4*22 genes. (NCT01936519)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
| Recipient Genotypes : rs776746 (CYP3A5 gene) | Recipient Genotypes : rs1045642 (ABCB1 gene) | Recipient Genotypes : rs1128503 (ABCB1 gene) | Recipient Genotypes : rs2032582 (ABCB1 gene) | Recipient Genotypes : rs35599367 (CYP4A4*22 gene) | Donor Genotypes : rs776746 (CYP3A5 gene) | Donor Genotypes : rs1045642 (ABCB1 gene) | Donor Genotypes : rs1128503 (ABCB1 gene) | Donor Genotypes : rs2032582 (ABCB1 gene) | Donor Genotypes : rs35599367 (Cyp4A4*22) |
---|
Calcineurin Inhibitor and Mycophenolic Acid | 11 | 6 | 7 | 1 | 1 | 3 | 1 | 0 | 0 | 0 |
,Everolimus and Mycophenolic Acid | 11 | 6 | 8 | 0 | 1 | 0 | 5 | 0 | 0 | 1 |
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Renal Function as Measured by 24 Hour Urine Creatinine Clearance
Renal Function was assessed by 24 hr urine collection creatinine clearance measured (mL/min). 24 Hr urine collection was assessed at baseline, 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years
Intervention | mL/min (Mean) |
---|
| 24hr Urine Creatinine Clearance at 6 months | 24hr Urine Creatinine Clearance at 1 year | 24hr Urine Creatinine Clearance at 2 years |
---|
Calcineurin Inhibitor With Mycophenolic Acid | 68.09 | 68.09 | 61.54 |
,Everolimus With Mycophenolic Acid | 70.75 | 86.8 | 90.63 |
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Renal Function as Measured by Cockcroft Gault Creatinine Clearance
The Cockcroft-Gault formula for estimating creatinine clearance was determined at 6 months, 1 year, and 2 years post transplant (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years
Intervention | mL/min/1.73m2 (Mean) |
---|
| Cockcroft Gault Creatinine Clearance at 6 months | Cockcroft Gault Creatinine Clearance at 1 year | Cockcroft Gault Creatinine Clearance at 2 years |
---|
Calcineurin Inhibitor and Mycophenolic Acid | 79.84 | 86.84 | 80.85 |
,Everolimus and Mycophenolic Acid | 100.17 | 113.47 | 108.16 |
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Renal Function as Measured by Iothalamate Clearance
Iothalamate Clearance was assessed at 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years
Intervention | mL/min/1.73m2 (Mean) |
---|
| Iothalamate Clearance at 6 months | Iothalamate Clearance at 1 year | Iothalamate Clearance at 2 years |
---|
Calcineurin Inhibitor and Mycophenolic Acid | 67.99 | 66.65 | 57.19 |
,Everolimus and Mycophenolic Acid | 74.23 | 104.01 | 79.41 |
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Renal Function as Measured by Modification of Diet in Renal Disease (MDRD) Estimated Glomerular Filtration Rate (eGFR)
Modification of Diet in Renal Disease (MDRD) estimated Glomerular Filtration Rate (eGFR) was assessed at 6 months, 1 year, and 2 years post transplant. (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years
Intervention | mL/min/1.73 m2 (Mean) |
---|
| MDRD eGFR at 6 months | MDRD eGFR at 1 year | MDRD eGFR at 2 years |
---|
Calcineurin Inhibitor and Mycophenolic Acid | 62.18 | 60.63 | 53.29 |
,Everolimus and Mycophenolic Acid | 81.27 | 88.01 | 87.37 |
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Renal Function as Measured by Serum Creatinine Level
Serum creatinine levels were assessed at 6 months, 1 year, and 2 years post transplant (NCT01936519)
Timeframe: 6 months, 1 year, and 2 years
Intervention | mg/dL (Mean) |
---|
| Serum Creatinine at 6 months | Serum Creatinine at 1 year | Serum Creatinine at 2 years |
---|
Calcineurin Inhibitor and Mycophenolic Acid | 1.29 | 1.29 | 1.51 |
,Everolimus and Mycophenolic Acid | 1.02 | 0.95 | 0.95 |
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Anti-CFZ533 Antibodies - Part I
To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study
Intervention | anti-CFZ533 antibodies (Number) |
---|
CFZ533 + TAC + MMF (Part 1) | 0 |
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Mean AUClast Pharmacokinetic Parameter - Part I
Quantify pharmacokinetics of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. (NCT02217410)
Timeframe: Day 1
Intervention | day*ug/mL (Mean) |
---|
CFZ533 + TAC + MMF (Part 1) | 367 |
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Mean Cmax Pharmacokinetic Parameter- Part I
Pharmacokinetics as defined by the systemic concentrations and Cmax of certain immunosuppressant medications used in Part I (NCT02217410)
Timeframe: Day 1
Intervention | ug/mL (Mean) |
---|
CFZ533 + TAC + MMF (Part 1) | 66.3 |
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Mean Tmax Pharmacokinetic Parameter - Part I
Quantify pharmacokinetics of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. (NCT02217410)
Timeframe: Day 1
Intervention | day (Median) |
---|
CFZ533 + TAC + MMF (Part 1) | 0.237 |
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Anti-CFZ533 Antibodies - Part II
To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study (screening, baseline, Day 141, Day 225, Day 309, Study Completion)
Intervention | anti-CFZ533 antibodies (Number) |
---|
| Screening | Day 141 | Day 225 | Day 309 | Study Completion |
---|
Tac + MMF (Part 2) | 0 | 0 | 0 | 0 | 0 |
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Anti-CFZ533 Antibodies - Part II
To evaluate the immunogenicity of CFZ533 via the quantitative analysis of anti-CFZ533 antibodies (NCT02217410)
Timeframe: Baseline to end of study (screening, baseline, Day 141, Day 225, Day 309, Study Completion)
Intervention | anti-CFZ533 antibodies (Number) |
---|
| Screening | Baseline | Day 141 | Day 225 | Day 309 | Study Completion |
---|
CFZ533 + MMF (Part 2) | 0 | 0 | 0 | 0 | 0 | 0 |
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CFZ533 Plasma PK Concentrations - Part II
Quantify the systemic concentrations of CFZ533 in combination with MMF, CS, and tacrolimus in de novo renal transplant patients during the treatment and follow-up periods. A full pharmacokinetic analysis can be performed on the concentration-time data to evaluate the impact of renal transplantation on the various medications used in the treatment regimen. (NCT02217410)
Timeframe: throughout study period (day 84 to day 336)
Intervention | ug/mL (Mean) |
---|
| Day 84 | Day 112 | Day 140 | Day 168 | Day 196 | Day 224 | Day 252 | Day 280 | Day 308 | Day 336 | End of study |
---|
CFZ533 + MMF (Part 2) | 247 | 211 | 178 | 157 | 148 | 147 | 151 | 160 | 132 | 156 | 133 |
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Efficacy as Defined by the Frequency and Severity (Banff Classification) of Treated Biopsy Proven Acute Rejection (tBPAR) Adjudicated Data - Part II
"To assess the activity of the investigational arm as compared to the standard of care control arm in de novo renal transplant patients as measured by the frequency and severity of tBPAR as measured on the Banff classification scale.~An adjudication was performed on all on cause renal biopsies by an independent expert committee blinded to therapy." (NCT02217410)
Timeframe: 3, 6, 9, and 12 months
Intervention | events (Number) |
---|
| Month 3 | Month 6 | Month 9 | Month 12 |
---|
CFZ533 + MMF (Part 2) | 6 | 7 | 7 | 7 |
,Tac + MMF (Part 2) | 2 | 3 | 3 | 3 |
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eGFR - Part II
"Renal function as assessed by MDRD (Modification of Diet in Renal Disease) formula.~eGFR: Estimated glomerular filtration rate" (NCT02217410)
Timeframe: Day 1, Day 29, Day 337,
Intervention | ml/min (Mean) |
---|
| Day 1 | Day 29 | Day 337 |
---|
CFZ533 + MMF (Part 2) | 9.8 | 55.6 | 58.2 |
,Tac + MMF (Part 2) | 9.7 | 44.3 | 44.2 |
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Free CD40 and Total CD40 on B Cells - Part II
The magnitude and duration of peripheral blood CD40 occupancy. MESF: molecules of equivalent soluble fluorochrome (NCT02217410)
Timeframe: Baseline to end of study (Day 1/predose)
Intervention | MESF (Mean) |
---|
| CFZ553 + MMF (Baseline) | CFZ553 + MMF (D1 - 6h post dose) | CFZ553 + MMF (D15) | CFZ553 + MMF (D 29) | CFZ553 + MMF (D 57) | CFZ553 + MMF (D 85) | CFZ553 + MMF (D 197) | CFZ553 + MMF (253) | CFZ553 + MMF (EoS) | Tac + MMF (Baseline) | Tac + MMF (D1 - 6h post dose) | Tac + MMF (D15) | Tac + MMF (D29) | Tac + MMF (D57) | Tac + MMF (D85) | Tac + MMF (D197) | Tac + MMF (D253) | Tac + MMF (EoS) |
---|
Free CD40 on Whole Blood B Ceels | 30836.00 | 1623.81 | 799.62 | 817.63 | 597.13 | 635.47 | 3699.0 | 2667.38 | 176.17 | 31508.33 | 26437.65 | 24441.67 | 27840.00 | 27994.29 | 25044.00 | 21360.00 | 15752.75 | 21200.00 |
,Total CD40 on Whole Blood B Cells | 12778.80 | 13806.90 | 15160.38 | 13299.60 | 12234.38 | 9330.86 | 2820.72 | 1427.14 | 1069.67 | 14581.43 | 13715.00 | 13707.14 | 12698.75 | 12583.85 | 8701.54 | 2067.60 | 1750.50 | 6276.17 |
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Total sCD40 Plasma Concentrations - Part II
To quantify the change from baseline and recovery of peripheral blood total soluble CD40 (NCT02217410)
Timeframe: 12 months
Intervention | ng/mL (Mean) |
---|
| Baseline | Day 1 | Day 4 | Day 15 | Day 29 | Day 57 | Day 85 | Day 113 | Day 141 | Day 169 | Day 197 | Day 225 | Day 253 | Day 281 | Day 309 | Day 337 | End of Study |
---|
CFZ533 + MMF (Part 2) | 3.02 | 6.95 | 24.6 | 69.6 | 101 | 140 | 189 | 215 | 237 | 238 | 253 | 258 | 236 | 273 | 286 | 298 | 303 |
,Tac + MMF (Part 2) | 3.67 | 1.16 | 1.16 | 0.869 | 0.362 | 0.438 | 0.429 | 0.391 | 0.453 | 0.537 | 0.423 | 0.452 | 0.457 | 0.455 | 0.454 | 0.411 | 0.959 |
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Total Soluble CD40 and Total Soluble CD154 Concentrations in Plasma - Part 1
To quantify the change from baseline and recovery of peripheral blood total soluble CD40 and total soluble CD154 (NCT02217410)
Timeframe: Baseline to end of study (Day 1, Day 29, Day 337)
Intervention | ng/ml (Mean) |
---|
| Baseline | Day 1 | Day 2 | Day 3 | Day 4 | Day 8 | Day 15 | Day 22 | Day 29 | Day 36 | Day 43 | Day 50 | Day 57 | Day 64 | Day 71 | Day 85 | Day 99 | Day 113 | Day 127 | EoS |
---|
sCD154 | 0.125 | 0.0585 | 0.139 | 0.157 | 0.241 | 0.399 | 0.0879 | 0.0500 | 0.225 | 0.116 | 0.0193 | 0.0478 | 0 | 0.0316 | 0.0148 | 0.0139 | 0 | 0.0668 | 0.0488 | 0.0184 |
,sCD40 (Part I) | 4.03 | 8.86 | 16.7 | 24.8 | 31.3 | 54.0 | 84.1 | 102 | 120 | 128 | 145 | 156 | 161 | 163 | 156 | 168 | 155 | 85.7 | 12.2 | 0.918 |
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GVHD-free Relapse-free Survival (GRFS)
Number of participants alive, without relapse, and without GVHD at 1 year. (NCT02833805)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 19 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 21 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 19 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 21 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 19 |
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Overall Survival at One Year
Number of participants alive at one year after BMT. (NCT02833805)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 19 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 21 |
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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
Intervention | Participants (Count of Participants) |
---|
Bone Marrow Transplant | 20 |
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100-day Overall Survival
Proportion of patients who survived 100 days or more after enrolled on the study (NCT00057954)
Timeframe: Assessed at least twice a week for the first 60 days and weekly until day 100.
Intervention | Proportion of participants (Number) |
---|
Transplant | 0.83 |
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Progression-free Survival
Progression-free survival was defined as time from enrollment to disease progression or death from any cause, whichever occurred first. Patients who did not have progression-free survival events were censored at last date of disease assessment. (NCT00057954)
Timeframe: Assessed day 100 post transplant and every 3 months during year 1, every 6 months during years 2-3, then every 12 months during years 4-5 or through diagnosis of disease progression
Intervention | days (Median) |
---|
Transplant | 104 |
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Proportion of Participants With Successful Engraftment
(NCT00057954)
Timeframe: Assessed daily during inpatient stay
Intervention | Proportion of participants (Number) |
---|
Transplant | 1 |
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Complete Response Rate
"Completed response is defined as:~Bone marrow evaluation: Repeat bone marrow showing < 5% myeloblasts with normal maturation of all cell lines, with no evidence for dysplasia (see dysplasia qualifier under peripheral blood evaluation).~Peripheral blood evaluation [absolute values must last at least 2 months] Hemoglobin >11 g/dl (untransfused, not on erythropoietin) Neutrophils (1500/mm3 (not on a myeloid growth factor)) Platelets (100,000/mm3 (not on a thrombopoetic agent)) Blasts - 0% No dysplasia. No detectable cytogenetic abnormality, if preexisting abnormality was present" (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.
Intervention | percentage of participants (Number) |
---|
Arm I | 35.3 |
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Number of Patients Who Developed Disease Progression After Achieving Complete Response
Disease free survival (DFS) was listed as a secondary endpoint in the study protocol, which would be assessed in patients who achieved complete response (CR). It was defined to be time from CR to documented progression or to death without progression. Patients without documented progression or death reported were censored at the time of last disease evaluation. However, due to the small number of patients with CR, the number of patients who developed disease progression was reported here. (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.
Intervention | participants (Number) |
---|
Arm I | 1 |
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Overall Survival
Overall survival (OS) is defined to be the time from registration to death from any cause, with follow-up censored at the date of last contact. Kaplan-Meier method was used to estimate the distribution of OS. (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.
Intervention | years (Median) |
---|
Arm I | 1.2 |
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Proportion of Graft Versus Host Disease
Proportion of Graft versus Host Disease is calculated as number of patients with Graft versus Host Disease divided by all eligible and treated patients (NCT00045305)
Timeframe: Monthly for the first 3 months from study entry, every 3 months for the first two years from study entry thereafter and then every 6 months for years 3-5.
Intervention | proportion of participants (Number) |
---|
Arm I | 0.412 |
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Time to Engraftment for Neutrophil
Time to neutrophil engraftment is defined from date of infusion to date of neutrophil engraftment. Neutrophil engraftment is defined as ANC > 500/mm3 on two consecutive measurements. The date of engraftment is the date of the first ANC > 500/mm3. (NCT00045305)
Timeframe: Daily while hospitalized and then at least 1x/week for the first 50 days and then at least every other week until day 100.
Intervention | days (Median) |
---|
Arm I | 18 |
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Time to Engraftment for Platelet
Time to platelet engraftment is defined from date of infusion to date of platelet engraftment. The platelet engraftment is defined as platelets > 20,000 on two consecutive measurements, at least seven days apart, without platelet transfusions in between and for at least three days before the first measurement that is over 20,000. The date of engraftment is the date of the first measurement that is over 20,000. (NCT00045305)
Timeframe: Daily while hospitalized and then at least 1x/week for the first 50 days and then at least every other week until day 100.
Intervention | days (Median) |
---|
Arm I | 18 |
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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
Intervention | Participants (Count of Participants) |
---|
Dose Level 1: 12 Gy Iodine-131 + BC8 Monoclonal Antibody | 0 |
Dose Level 2: 14 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 |
Dose Level 3: 16 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 |
Dose Level 4: 18 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 |
Dose Level 5: 20 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 |
Dose Level 6: 22 Gy Iodine-131 + BC8 Monoclonal Antibody | 0 |
Dose Level 7: 24 Gy Iodine-131 + BC8 Monoclonal Antibody | 0 |
Dose Level 8: 26 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 |
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Chronic Graft vs Host Disease (GvHD)
The incidence of chronic graft vs host disease (cGvHD) following the infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells will be assessed. The outcome is reported as the number of allogeneic CD8+ memory T-cell recipients who experience cGvHD more 30 days but within 1 year of the cellular infusion, a number without dispersion. (NCT02424968)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 2 |
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Disease Progression (TDP)
Whether or not the treated disease returns, known as disease progression or relapse, is a measure of treatment efficacy. Recipients of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells were monitored for disease progression through 1 year after the cellular infusion. The outcome is reported as the number of allogeneic CD8+ memory T-cells recipients that experienced disease progression within 12 months (1 year). (NCT02424968)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 4 |
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Event-free Survival (EFS
Event-free survival (EFS) is defined as the number of transplant recipients of allogeneic cluster of differentiation 8 (CD8+) memory T-cells that remain alive at 12 months after transplant without disease relapse. Relapse is defined as bone marrow blasts > 5% . The outcome is expressed as the number of allogeneic CD8+ memory T-cell recipients remaining alive at 1 year after transplant without disease relapse, a number without dispersion. (NCT02424968)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 15 |
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Full-dose Donor Chimerism (FDC)
A measure of success for the therapeutic infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells is full-dose donor chimerism (FDC). This means to achieve ≥ 95% donor cells in either the CD3+ blood cell lineage or whole blood, within 90 days of the allogeneic CD8+ memory T-cell infusion. The outcome is reported as the number of participants that achieve FDC within 90 days, a number without dispersion. (NCT02424968)
Timeframe: 3 months
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 12 |
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Incidence of Acute Graft vs Host Disease (GvHD)
Occurrence of acute graft vs host disease (aGvHD) following the infusion of allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells will be assessed. The outcome is reported as the number of allogeneic CD8+ memory T-cell recipients who experience aGvHD within 30 days of the cellular infusion, a number without dispersion. (NCT02424968)
Timeframe: Up to 30 days post-infusion
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 1 |
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LOWSKY Grade 3 or Higher Toxicities
Related adverse events, ie, toxicities, ≥ Grade 3 are significant considerations in the treatment of study participants receiving allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells. The outcome is reported as the number of allogeneic CD8+ memory T-cells transplant recipients who experienced ≥ Grade 3 toxicity within 60 days of infusion of the allogeneic transplantation of cluster of differentiation 8 (CD8+) memory T-cells, a number without dispersion. (NCT02424968)
Timeframe: Up to 60 days post-infusion
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 0 |
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Non-relapse Mortality (NRM)
Non-relapse mortality (NRM) is defined as death without known disease relapse or recurrence. The outcome is expressed as the number of allogeneic CD8+ memory T-cells tr. ansplant recipients whose cause of death was not disease relapse or recurrence, a number without dispersion (NCT02424968)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 1 |
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Overall Survival (OS)
Overall survival (OS) is defined as remaining alive 12 months after the infusion of allogeneic cluster of differentiation 8 (CD8+) memory T-cells. The outcome is reported as the number of allogeneic CD8+ memory T-cell transplant recipients remaining alive at 12 months after the cellular infusion, a number without dispersion (NCT02424968)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
Infusion of Allogeneic CD8+ Memory T-cells | 15 |
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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
Intervention | percentage of participants (Number) |
---|
Conditioning/HCT/GVHD Prophylaxis | 20.0 |
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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)
Intervention | percentage of participants (Number) |
---|
Conditioning/HCT/GVHD Prophylaxis | 18.8 |
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Overall Survival (OS)
Overall survival is defined as time from transplant to death or last follow-up, and is reported as percentage of surviving participants. (NCT03018223)
Timeframe: Up to 1 year post HCT
Intervention | percentage of participants (Number) |
---|
Conditioning/HCT/GVHD Prophylaxis | 70.2 |
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Progression Free Survival (PFS)
Progression-free survival defined by the time interval from transplant to relapse/recurrence, to death or to last follow-up. Reported as percentage of participants who are disease free one year post HCT. (NCT03018223)
Timeframe: Up to 1 year post HCT
Intervention | percentage of participants (Number) |
---|
Conditioning/HCT/GVHD Prophylaxis | 56.6 |
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Percent of Participants Experiencing Acute Allograft Rejection
Estimate the composite of treatment failure rate, defined as acute allograft rejection with a Banff grade 1A or higher, graft loss, or death at six months post-conversion, in patients converted to a once-daily immunosuppressant regimen of Envarsus®, everolimus, and prednisone versus patients converted to a twice-daily regimen of Envarsus®, MMF, and prednisone. (NCT02954198)
Timeframe: Baseline to 6 months post conversion
Intervention | Participants (Count of Participants) |
---|
Control: Envarsus + MMF | 0 |
Intervention: Envarsus + Everoliumus | 0 |
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Percent of Participants Who Experienced Kidney Transplant Graft Loss
Measure and compare time-to-event analysis between the two arms graft loss (time to event analysis) (NCT02954198)
Timeframe: Baseline to 6 months post conversion
Intervention | Participants (Count of Participants) |
---|
Control: Envarsus + MMF | 0 |
Intervention: Envarsus + Everoliumus | 0 |
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Self-reported Medication Adherence From Baseline to 6 Months.
Percent of subjects reporting high medication adherence at baseline compared to 6 months post-conversion, using the Morisky Medication Adherence scale (MMAS). The MMAS rates medication adherence on a scale of 0 to 8. 0 is high adherence, 1-2 is medium adherence, and greater than or equal to 3 is low adherence. (NCT02954198)
Timeframe: 6 months post conversion
Intervention | % of participants (Number) |
---|
| Baseline | 6 months post-conversion |
---|
Control: Envarsus + MMF | 80 | 59 |
,Intervention: Envarsus + Everoliumus | 45 | 47 |
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Subject Specific Change on Medication Side Effect Scale
Examine subject specific change on a validated Medication Side Effect Scale at the time of the conversion versus six months post-conversion, compared between the two arms. Side effect burden scale is from 0 to 180. A lower score is less side effect burden, a higher score is more side effect burden. (NCT02954198)
Timeframe: Baseline to 6 months post conversion
Intervention | units on a scale (Mean) |
---|
| Baseline | 6 month post-conversion |
---|
Control: Envarsus + MMF | 71 | 93 |
,Intervention: Envarsus + Everoliumus | 37 | 38 |
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Dose Modifications
Number of dose modifications from baseline to 3 months post-conversion. (NCT02953873)
Timeframe: Baseline to 3 months post conversion
Intervention | number of dose modifications (Median) |
---|
| Homozygous CYP3A5 1 expressors | Heterozygous CYP3A5 1 expressors | CYP3A5 1 non expressers |
---|
Conversion Arm | 1 | 2 | 4 |
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Dose-normalized Trough
Difference in dose-normalized trough at steady state before and after conversion from tacrolimus IR to Astagraf XL® (NCT02953873)
Timeframe: Baseline to 3 months post-conversion
Intervention | ng/dL (Median) |
---|
| Pre-Conversion Dose Trough | Post-Conversion Dose Trough |
---|
Conversion Arm | 0.59 | 0.44 |
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Number of Days to Reach Therapeutic Trough Goal
Days to reach therapeutic goal after conversion (NCT02953873)
Timeframe: Baseline to 3 months post conversion
Intervention | number of days (Median) |
---|
| Homozygous CYP3A5 1 expressors | Heterozygous CYP3A5 1 expressors | CYP3A5 1 non-expressors |
---|
Conversion Arm | 15 | 10 | 15 |
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Total Daily Dose
Difference in Total Daily Dose necessary for steady state therapeutic goal (NCT02953873)
Timeframe: Baseline to 3 months post conversion
Intervention | mg (Median) |
---|
| Pre-Conversion Tacrolimus Dose | Post-Conversion Tacrolimus Dose |
---|
Conversion Arm | 10 | 15 |
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Weight-Based Dose Requirement
Weight-based dose requirements to reach therapeutic goal pre- and post-conversion (NCT02953873)
Timeframe: Baseline to 3 months post conversion
Intervention | mg/kg (Median) |
---|
| Pre-Conversion | Post-Conversion |
---|
Conversion Arm | 0.11 | 0.16 |
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Donor Cell Engraftment
Chimerism analysis was performed in patients who recieved nonmyeloablative tranplsnat. In this group the definition of engraftment was a CD3 count greater than 50%. In the myeloablative group, engraftment was defined as an absolute neutrophil count greater than 50%. (NCT00322101)
Timeframe: After stem cell infusion to day 28
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 11 |
Arm II (Myeloablative Regimen) | 11 |
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Incidence and Severity of Acute and Chronic Graft-vs-host Disease
(NCT00322101)
Timeframe: After transplantation
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 1 |
Arm II (Myeloablative Regimen) | 4 |
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Incidence of Disease Progression/Relapse
Disease progression/relapse was defined by IWG criteria (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 7 |
Arm II (Myeloablative Regimen) | 2 |
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Non-relapse Mortality
(NCT00322101)
Timeframe: At 100 days
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 0 |
Arm II (Myeloablative Regimen) | 0 |
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Overall Survival
(NCT00322101)
Timeframe: At 2 years
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 6 |
Arm II (Myeloablative Regimen) | 6 |
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Progression-free Survival
IWG criteria was used to determine disease progression (NCT00322101)
Timeframe: After stem cell infusion to date of last follow up.
Intervention | participants (Number) |
---|
Arm I (Nonmyeloablative Regimen) | 7 |
Arm II (Myeloablative Regimen) | 5 |
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Change From Baseline in Anti-Double Stranded Deoxyribonucleic Acid (Anti-dsDNA) Antibody Levels at Week 52
Anti-dsDNA antibodies are a group of anti-nuclear antibodies targeting double stranded DNA. (NCT02550652)
Timeframe: Baseline and Week 52
Intervention | log IU/mL (Mean) |
---|
OBINUTUZUMAB 1000MG and MMF | -0.810 |
PLACEBO and MMF | -0.080 |
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Change From Baseline in C4 Levels at Week 52
Complement C4 is a blood test that reflects activation of complement pathway associated with immune deposition in certain autoimmune diseases. (NCT02550652)
Timeframe: Baseline, Week 52
Intervention | g/L (Mean) |
---|
OBINUTUZUMAB 1000MG and MMF | 0.101 |
PLACEBO and MMF | 0.003 |
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Change From Baseline in Complement Component 3 (C3) Levels at Week 52
Complement C3 is a blood test that reflects activation of complement pathway associated with immune deposition in certain autoimmune diseases. (NCT02550652)
Timeframe: Baseline and Week 52
Intervention | g/L (Mean) |
---|
OBINUTUZUMAB 1000MG and MMF | 0.311 |
PLACEBO and MMF | 0.108 |
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Percentage of Participants Who Achieve Protocol Defined Complete Renal Response (CRR) at Week 52
Percentage of participants with normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 red blood cells (RBCs)/high-power field (HPF) and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the upper limit of normal (ULN) range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: From baseline to Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 34.9 |
PLACEBO and MMF | 22.6 |
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Percentage of Participants Who Achieve Protocol Defined CRR at Week 24
CRR defined as normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 red blood cells (RBCs)/high-power field (HPF) and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the upper limit of normal (ULN) range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: Week 24
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 23.8 |
PLACEBO and MMF | 24.2 |
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Percentage of Participants Who Achieve Protocol Defined Modified CRR (mCRR1) at Week 52
mCRR1 has got two components only, i.e. serum Creatinine and urinary protein to creatinine ratio. mCRR1 is defined by attainment of normalization of serum creatinine as evidenced by 1.) serum creatinine ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is above the ULN or serum creatinine ≤15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine ≤ the ULN range of central laboratory values and 2.) Urinary protein to creatinine ratio <0.5. (NCT02550652)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 39.7 |
PLACEBO and MMF | 25.8 |
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Percentage of Participants Who Achieve Protocol Defined Overall Response (OR) at Week 52
OR includes both CRR and partial renal response (PRR). CRR as defined in the primary outcome measure above. PRR defined as 50% improvement in urine protein:creatinine ratio, with one of following conditions met: 1. If baseline urine protein:creatinine ratio is ≤ 3.0, then urine protein:creatinine ratio of <1.0. 2. If baseline protein:creatinine ratio is > 3.0, then urine protein:creatinine ratio of <3.0, serum creatinine ≤15% above baseline value, and no urinary red cell casts and either RBCs/HPF ≤50% above baseline or <10 RBCs/HPF. (NCT02550652)
Timeframe: From baseline to Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 55.6 |
PLACEBO and MMF | 35.5 |
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Percentage of Participants Who Achieve Protocol Defined Partial Renal Response (PRR) at Week 52
PRR defined as serum creatinine ≤15% above baseline value, no urinary red cell casts and either RBCs/HPF ≤ 50% above baseline or < 10 RBCs/HPF, 50% improvement in urine protein:creatinine ratio, with one of following conditions met: 1. If baseline urine protein:creatinine ratio is ≤ 3.0, then a urine protein:creatinine ratio of < 1.0. 2. If baseline protein:creatinine ratio is > 3.0, then a urine protein:creatinine ratio of < 3.0. (NCT02550652)
Timeframe: From baseline to Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 55.6 |
PLACEBO and MMF | 33.9 |
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Percentage of Participants Who Achieve Protocol Defined Second mCRR (mCRR2) at Week 52
mCRR2 is defined by normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 RBCs/HPF and the absence of red cell casts), and urinary protein to creatinine ratio <0.5. Normalization of serum creatinine as evidenced by the following: Serum creatinine ≤15% above baseline if baseline (Day 1) serum creatinine is above the normal range of the central laboratory values or ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. (NCT02550652)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 44.4 |
PLACEBO and MMF | 33.9 |
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Percentage of Participants Who Achieve Protocol Defined Third mCRR (mCRR3) at Week 52
mCRR3 is defined by normalization of serum creatine as evidenced by serum creatinine ≤ the ULN range of central laboratory values and urinary protein to creatinine ratio < 0.5. (NCT02550652)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 46.0 |
PLACEBO and MMF | 38.7 |
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Percentage of Participants With Anti-Drug Antibody (ADA) to Obinutuzumab
Antibodies are a blood protein produced in response to and counteracting a specific antigen. (NCT02550652)
Timeframe: From baseline up to Week 104
Intervention | percentage of participants (Number) |
---|
OBINUTUZUMAB 1000MG and MMF | 9.38 |
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Area Under the Plasma Concentration Versus Time Curve (AUC) of Obinutuzumab
(NCT02550652)
Timeframe: Week 0, Week 24, Week 52
Intervention | ug/mL*day (Mean) |
---|
| Week 0-24 | Week 24-52 | Week 0-52 |
---|
OBINUTUZUMAB 1000MG and MMF | 10595 | 15811 | 26406 |
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Change From Baseline of Participant's Global Assessment of Disease Activity Visual Analog Scale (VAS) Score
Each VAS had a range from 0-100 with higher scores indicating greater symptom impact on global health status. (NCT02550652)
Timeframe: Baseline (Day 1), Weeks 4, 12, 24, 36, 52
Intervention | score on scale (Mean) |
---|
| Baseline | Week 4 | Week 12 | Week 24 | Week 36 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 41.3 | -14.4 | -19.9 | -25.0 | -24.8 | -25.4 |
,PLACEBO and MMF | 39.4 | -8.7 | -11.6 | -20.8 | -19.6 | -23.3 |
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Maximum Observed Plasma Concentration (Cmax) of Obinutuzumab
(NCT02550652)
Timeframe: Week 0, Week 24, Week 52
Intervention | ug/mL (Mean) |
---|
| Week 0-24 | Week 24-52 | Week 0-52 |
---|
OBINUTUZUMAB 1000MG and MMF | 559 | 605 | 605 |
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Percent Change From Baseline in Circulating CD19-Positive B-Cell Levels
CD19+ B cell is a B-lymphocyte with a transmembrane protein that is encoded by the gene CD19. (NCT02550652)
Timeframe: Baseline, Week 2, Week 4, Week 12, Week 24, Week 52
Intervention | Percent change of cells/uL (Mean) |
---|
| Week 2 | Week 4 | Week 12 | Week 24 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | -97.469 | -98.777 | -97.045 | -96.628 | -98.620 |
,PLACEBO and MMF | 39.293 | -5.186 | 0.661 | -11.446 | 37.695 |
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Percentage of Participants With Adverse Events (AEs)
An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. AEs, including AEs of Special Interest were reported based on the national cancer institute common terminology criteria for AEs, Version 4.0 (NCI-CTCAE, v4.0). Reported are the number of subjects with AEs, Grade 3-5 AEs, and Serious Adverse Events (SAEs), Infections and Serious infections. (NCT02550652)
Timeframe: From Baseline up to Week 104
Intervention | percentage of participants (Number) |
---|
| Adverse Events | Grade 3 AEs | Grade 4 AEs | Grade 5 AEs | Serious Adverse Events | Infections | Serious infections |
---|
OBINUTUZUMAB 1000MG and MMF | 90.6 | 29.7 | 10.9 | 1.6 | 25.0 | 75.0 | 7.8 |
,PLACEBO and MMF | 88.5 | 24.6 | 13.1 | 6.6 | 29.5 | 62.3 | 18.0 |
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Percentage of Participants With First Protocol Defined CRR Over the Course of 52 Weeks
CRR included normalization of serum creatinine, inactive urinary sediment (as evidenced by < 10 RBCs/HPF and the absence of red cell casts) and urinary protein to creatinine ratio < 0.5. Normalization of serum creatinine is defined as serum creatinine ≤ the ULN range of central laboratory values if baseline serum creatinine is above the ULN or serum creatinine ≤ 15% above baseline and ≤ the ULN range of central laboratory values if baseline (Day 1) serum creatinine is ≤ the ULN range of central laboratory values. Percentage of participants with response at various time points were measured using Kaplan Meier method. (NCT02550652)
Timeframe: From Baseline to Week 52
Intervention | percentage of participants (Number) |
---|
| Week 12 | Week 24 | Week 36 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 10 | 26 | 36 | 50 |
,PLACEBO and MMF | 10 | 28 | 35 | 40 |
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Percentage of Participants With First Protocol Defined Overall Response Over the Course of 52 Weeks
OR includes both CRR and partial renal response(PRR). CRR as defined in the primary outcome measure above. PRR defined as 50% improvement in upcr, with one of these conditions met: 1. If baseline upcr is ≤3.0, then upcr of <1.0. 2. If baseline pcr is > 3.0, then upcr of <3.0, serum creatinine ≤15% above baseline value, and no urinary red cell casts and either RBCs/HPF ≤50% above baseline or <10 RBCs/HPF. Percentage of participants with response at various time points were measured using Kaplan Meier method. (NCT02550652)
Timeframe: From baseline to Week 52
Intervention | percentage of participants (Number) |
---|
| Week 12 | Week 24 | Week 36 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 26 | 57 | 63 | 75 |
,PLACEBO and MMF | 19 | 41 | 51 | 58 |
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Systemic Clearance of Obinutuzumab
(NCT02550652)
Timeframe: Day 0, Week 24, Week 52
Intervention | L/day (Mean) |
---|
| Day 0 | Week 24 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 0.255 | 0.147 | 0.137 |
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Terminal Plasma Half-Life (t1/2) of Obinutuzumab
(NCT02550652)
Timeframe: Day 0, Week 24, Week 52
Intervention | day (Mean) |
---|
| Day 0 | Week 24 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 13.1 | 20.5 | 22.1 |
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Volume of Distribution Under Steady State (Vss) of Obinutuzumab
(NCT02550652)
Timeframe: Day 0, Week 24, Week 52
Intervention | L (Mean) |
---|
| Day 0 | Week 24 | Week 52 |
---|
OBINUTUZUMAB 1000MG and MMF | 3.67 | 3.67 | 3.67 |
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Non-relapse Mortality
(NCT01690520)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Standard of Care) | 16 |
Arm II (Experimental) | 16 |
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Overall Survival
(NCT01690520)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Standard of Care) | 52 |
Arm II (Experimental) | 57 |
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Proportion of Participants With Chronic Graft Versus Host Disease
(NCT01690520)
Timeframe: Up to 2 years
Intervention | Participants (Count of Participants) |
---|
Arm I (Standard of Care) | 27 |
Arm II (Experimental) | 23 |
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Proportion of Patients With Severe Acute Graft Versus Host Disease
(NCT01690520)
Timeframe: Up to 100 days post-transplant
Intervention | proportion of participants (Number) |
---|
Arm I (Standard of Care) | 0.14 |
Arm II (Experimental) | 0.16 |
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Time to Neutrophil Engraftment
First of two consecutive days with absolute neutrophil count (ANC) equal to or greater than 500 cell/microliter measured from day of transplant. (NCT01690520)
Timeframe: Up to 55 days post-transplant
Intervention | days (Median) |
---|
Arm I (Standard of Care) | 20.0 |
Arm II (Experimental) | 22.0 |
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Time to Platelet Engraftment (20k)
First day of seven consecutive days with platelet count equal to or greater than 20,000 cells/microliter without platelet transfusions measured from day of transplant. (NCT01690520)
Timeframe: Up to 100 days post-transplant
Intervention | days (Median) |
---|
Arm I (Standard of Care) | 40.0 |
Arm II (Experimental) | 38.0 |
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GI Tolerability as Measured by GSRS
"Side-effects using the GSRS (Gastrointestinal Symptoms Rating Scale)~GSRS has 15 items, each rated on a 7-point scale from 1 (no discomfort) to 7 (very severe discomfort). GSRS total minimum value is 15; maximum value is 105. Higher scores represent greater discomfort." (NCT00574197)
Timeframe: Baseline and 6 months
Intervention | score on a scale (Mean) |
---|
| GSRS at baseline | GSRS at 6 months |
---|
Enteric-coated Mycophenolate Sodium (Myfortic) | 55.67 | 34.17 |
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Allograft Survival Rate
Allograft survival is defined as participants who did not need to be re-transplanted or placed on dialysis due to the failure of their allograft transplantation during the course of this study. (NCT01517984)
Timeframe: 6 to 18 months post-randomization
Intervention | participants (Number) |
---|
Randomized to Tacrolimus Withdrawal | 14 |
Randomized to Control Group | 7 |
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Incidence of Acute Rejection
Acute renal allograft rejection is defined as histological reading of borderline or greater determined by the local pathology laboratory. Participants suspected of having a rejection episode on the basis of clinical signs, symptoms, or on the basis of laboratory tests, had a renal ultrasound and underwent a renal transplant biopsy. Any detection of acute cellular rejection or acute humoral rejection resulted in participants in the 'Randomized to Tacrolimus Withdrawal' group to be restarted on tacrolimus and followed per the reduced follow-up schedule of events. (NCT01517984)
Timeframe: 6 to 18 months post-randomization
Intervention | participants (Number) |
---|
Randomized to Tacrolimus Withdrawal | 6 |
Randomized to Control Group | 0 |
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Participant Survival Rate
Number of participants who did not die within the course of this study. (NCT01517984)
Timeframe: 6 to 18 months post-transplantation
Intervention | participants (Number) |
---|
Randomized to Tacrolimus Withdrawal | 14 |
Randomized to Control Group | 7 |
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Percentage of Participants in the Experimental Arm Off Tacrolimus
Participants in the 'Randomized to Tacrolimus Withdrawal' group were considered fully withdrawn once they no longer received any doses of tacrolimus. Participants met this endpoint if they did not resume taking tacrolimus as of 18 months post randomization with stable allograft function and without rejection of donor-specific antibodies. (NCT01517984)
Timeframe: 18 months post-randomization
Intervention | percentage of participants (Number) |
---|
Randomized to Tacrolimus Withdrawal | 43 |
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Percentage of Participants With New Donor Specific Antibodies (DSAs)
Donor specific antibodies are antibodies that are directed against antigens expressed on donor organs. These antibodies can result in an immune attack on the transplanted organ, increasing risk of graft loss and/or rejection. (NCT01517984)
Timeframe: 6 to 18 months post-randomization
Intervention | percentage of participants (Number) |
---|
Randomized to Tacrolimus Withdrawal | 36 |
Randomized to Control Group | 14 |
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Estimated GFR Using the Chronic Kidney Disease Epidemiology (CKD-EPI) Equation
Estimated glomerular filtration rate (eGFR) is a test to measure the level of kidney function. In this measure, the effects of tacrolimus withdrawal on long-term kidney function was assessed by comparing absolute 24 month eGFR (18 months post-randomization) and change in eGFR from 6 to 24 months (randomization to 18 months randomization). Lower numbers indicate poorer kidney function (NCT01517984)
Timeframe: 6 months post-transplantation, 24 months post-transplantation
Intervention | mL/min (Mean) |
---|
| 6 Month eGFR | 24 Month eGFR | Change in eGFR from 6 to 24 months |
---|
Randomized to Control Group | 62.3 | 68.6 | 6.3 |
,Randomized to Tacrolimus Withdrawal | 56.2 | 61.7 | 5.5 |
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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
(NCT00352976)
Timeframe: by 100 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 88.0 |
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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
(NCT00352976)
Timeframe: by 180 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 98.7 |
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Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
(NCT00352976)
Timeframe: by 365 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 107.2 |
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Average IgG Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
(NCT00352976)
Timeframe: by 365 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 724.0 |
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Average IgG Levels as a Measure of Immune Reconstitution After Transplant, by 180 Days
(NCT00352976)
Timeframe: by 180 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 652.9 |
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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 100 Days
(NCT00352976)
Timeframe: by 100 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 71.1 |
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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 180 Days
(NCT00352976)
Timeframe: by 180 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 77.0 |
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Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 365 Days
(NCT00352976)
Timeframe: by 365 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 82.8 |
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Average Immunoglobulin G (IgG) Levels as a Measure of Immune Reconstitution After Transplant, by 100 Days
(NCT00352976)
Timeframe: by 100 days
Intervention | mg/dL (Mean) |
---|
Treatment With TBI | 550.6 |
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Number of Participant With Neutrophil Recovery
Number of participant with neutrophil recovery. Neutrophil recovery is defined as absolute neutrophil count ≥500/µL for three consecutive days (NCT00352976)
Timeframe: by day 42
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 78 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 15 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 6 |
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Number of Participants Experiencing Infections by Day 100
(NCT00352976)
Timeframe: by day 100
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 53 |
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Number of Participants Experiencing Infections by Day 180
(NCT00352976)
Timeframe: by day 180
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 55 |
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Number of Participants Experiencing Infections by Day 365
(NCT00352976)
Timeframe: by day 365
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 56 |
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Number of Participants Experiencing Overall Survival
Number of participants experiencing overall survival by 1 year (NCT00352976)
Timeframe: at one year
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 70 |
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Number of Participants With Secondary Graft Failure at 100 Days
Secondary Graft Rejection by day 100 (NCT00352976)
Timeframe: 100 days
Intervention | Participants (Count of Participants) |
---|
Treatment With TBI | 4 |
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Number of Participants With BK Viral Load <600 Copies/mL
A Viral load of <600 copies/mL for at least 3 months indicates sustained clearance of BK viremia, confirmed by blood test (NCT01649609)
Timeframe: Up to 12 months from enrollment
Intervention | Participants (Count of Participants) |
---|
Standard Immunosuppression Reduction Arm | 13 |
mTOR Substitution Arm | 17 |
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Number of Participants With Incidence of BK Nephropathy
The number of people with incidence of BK Nephropathy in each of the two Arms (NCT01649609)
Timeframe: Up to 24 months from randomization
Intervention | Participants (Count of Participants) |
---|
Standard Immunosuppression Reduction Arm | 3 |
mTOR Substitution Arm | 1 |
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Count of Participants Who Achieved Neutrophil Engraftment
Neutrophil engraftment is defined as the first day of three consecutive days where the neutrophil count (absolute neutrophil count) is 500 cells/mm^3 (0.5 x 10^9/L) or greater. (NCT01685411)
Timeframe: By Day 42
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 5 |
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Count of Participants With Disease Free Survival
The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 5 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 1 |
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Count of Participants With Disease Free Survival
The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this done by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 7 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 1 |
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Counts of Participants With Disease Free Survival
The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. In a clinical trial, measuring the disease-free survival is one way to see how well a new treatment works. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 3 |
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Number of Participant Who Were Alive at 2 Years Post Transplant
Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 2 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 4 |
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Number of Participant Who Were Alive at 5 Years Post Transplant
Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 5 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 3 |
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Number of Participant Who Were Alive at 7 Years Post Transplant
Overall survival will be defined as time from date of enrollment to date of death or censored at the date of last documented contact for patients still alive. (NCT01685411)
Timeframe: 7 Years
Intervention | Participants (Count of Participants) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 1 |
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Percentage of Participants With Chronic Graft-Versus-Host Disease
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 6 Months
Intervention | percentage of participants (Number) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 0 |
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Percentage of Participants With Chronic Graft-Versus-Host Disease
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: 1 Year
Intervention | percentage of participants (Number) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 0 |
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Percentage of Participants With Engraftment Failure
Graft failure is defined as not accepting donated cells. The donated cells do not make the new white blood cells, red blood cells and platelets. (NCT01685411)
Timeframe: Day 42
Intervention | percentage of participants (Number) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 0 |
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Percentage of Participants With Relapse
The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 1 Year
Intervention | percentage of participants (Number) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 40 |
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Percentage of Participants With Relapse
The return of disease after its apparent recovery/cessation. Patients with leukemia involving the BM and myelodysplastic syndrome will have this assessed by BM biopsy and additional special studies such as cytogenetics or flow cytometry as appropriate. Patients will also have radiology studies such as plain X-rays or CT scans and/or other studies such as blood tumor markers to document presence or absence of disease as clinically indicated. (NCT01685411)
Timeframe: 2 Years
Intervention | percentage of participants (Number) |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 40 |
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Percentage of Participants With Acute Graft-Versus-Host Disease by Grade
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host. Patients will be staged weekly between days 0 and 100 after transplantation using standard criteria used for staging. Patients will be assigned an overall GVHD score based on extent of skin rash, volume of diarrhea and maximum bilirubin level. The stages of individual organ involvement are combined to produce an overall grade. Grade I GVHD is characterized as mild disease, grade II GVHD as moderate, grade III as severe, and grade IV life-threatening. (NCT01685411)
Timeframe: Day 100
Intervention | percentage of participants (Number) |
---|
| Grade 2-4 | Grade 3-4 |
---|
Allogeneic Hematopoietic Stem Cell Transplant | 40 | 20 |
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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
Intervention | Participants (Count of Participants) |
---|
Reduced Intensity Regimen | 29 |
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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
Intervention | Participants (Count of Participants) |
---|
| Expired Secondary to Sepsis | Expired Secondary to GVHD | Survived Participants |
---|
Reduced Intensity Regimen | 1 | 2 | 26 |
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Dose-normalized AUC of Mycophenolic Acid
"Bioavailability (12h AUC) of mycophenolic acid in renal transplant patients after administration of MMF+/-PAN and EC-MPS+/-PAN~For evaluation of pharmacokinetic and pharmacodynamic parameters blood will be collected before, 0.5h, 1h, 1.5h, 2h, 3h, 4h, 5h, 6h, 8h, 10h, 12h after drug intake." (NCT01801280)
Timeframe: Study duration for each patient: 2 months. After 10-14 days of drug intake blood samples for PK/PD analysis will be collected. On the next day new treatment starts. There are 4 study visits at the study center. Duration will be approximately 12hours
Intervention | mg*h/L (Mean) |
---|
Mycophenolate Mofetil (MMF) | 41 |
MMF+PAN | 38 |
EC-MPS | 43 |
EC-MPS + PAN | 46 |
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Collection of Blood for Future Analysis of Peripheral Blood Mononuclear Cells (PBMCs)
Blood was drawn and banked for future use in order to characterize immune system markers and further the understanding of the immune factors that contribute to disease progression in ALS. (NCT01884571)
Timeframe: Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6), Post-Treatment Period (Months 8 and 12)
Intervention | Participants (Count of Participants) |
---|
Immunosuppression Regimen | 31 |
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Collection of Cerebrospinal Fluid for Future Analysis of Cytokine Levels
Lumbar punctures (LPs) were performed to collect cerebrospinal fluid (CSF). CSF is banked for future use to characterize immune system markers and to further the understanding of the immune factors that contribute to disease progression in ALS. Cytokines are markers of neuroinflammation and can be categorized as neurotoxic or neuroprotective. The role that cytokines play in in ALS progression is still not yet fully understood. (NCT01884571)
Timeframe: Pre-Treatment Period (two months prior to the start of treatment), Treatment Period (Months 2 and 6), Post-Treatment Period (Month 12)
Intervention | Participants (Count of Participants) |
---|
Immunosuppression Regimen | 29 |
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Mean Rate of Change in Grip Strength Treatment Compared to Pre-Treatment
Hand grip was measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. The grip strength of the left and right hands were analyzed together. Grip strength was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. Grip strength is a measurement of muscle strength and declines as ALS progresses. A positive value means that scores during treatment were higher than pre-treatment scores, indicating an increase in grip strength over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)
Intervention | pounds change per month (Mean) |
---|
Immunosuppression Regimen | 1.38 |
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Mean Rate of Change of ALSFRS-R Scores During Treatment Compared to Pre-Treatment
The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). ALSFRS-R was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in ability over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)
Intervention | units on a scale change per month (Mean) |
---|
Immunosuppression Regimen | -0.24 |
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Mean Rate of Change of Hand-Held Dynamometry (HHD) During Treatment Compared to Pre-Treatment
Hand held dynamometry (HHD) is a measure of muscle strength and scores decrease as ALS progresses. Six proximal muscle groups were examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group are established from the initial values for each participant. Strength determinations were converted to Z scores and averaged to provide an HHD megascore. The Z-score indicates the number of standard deviations away from the mean of 0. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. HHD was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in strength over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)
Intervention | z-score change per month (Mean) |
---|
Immunosuppression Regimen | -0.05 |
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Mean Rate of Change of Slow Vital Capacity (SVC) During Treatment Compared to Pre-Treatment
Vital capacity (VC), percent of predicted normal, was determined using the slow VC method. SVC measures the amount of air exhaled following a deep breath. For this test, participants hold a mouthpiece in their mouth, breathe in deeply, and breathe out as much air as they can. The test was done seated in a chair and then repeated while lying on an exam table at the Screening Visit. For all other visits, this test was done while seated in a chair. This test takes 15-20 minutes. SVC was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. SVC is a way to measure respiratory insufficiency in persons with ALS and SVC decreases as ALS progresses. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline over time. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)
Intervention | percent predicted change per month (Mean) |
---|
Immunosuppression Regimen | -0.18 |
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Mean Rate of Change of T-cell Subsets in Blood Treatment Compared to Pre-Treatment
Blood was collected for ribonucleic acid (RNA) and the mean rate of decline of T-cells during the 6 month treatment period compared to the pre-treatment period was assessed (blood was collected twice during the 3-month long lead in period). The precise role that T-cells have in ALS is unknown and this study aims to further the understanding of how T-cells operate in persons with ALS. T-cell measurement is a ratio where the relative expression levels of FOXP3 messenger ribonucleic acid (mRNA) was calculated using the Comparative CT Method (ΔΔCT Method), normalizing to β-actin. Samples were obtained during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in T-cells over time. (NCT01884571)
Timeframe: Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6)
Intervention | fold change per month (Mean) |
---|
Immunosuppression Regimen | -0.04 |
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Number of Participants With an Average Increase in ALSFRS-R Score of One Point Per Month
The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). A clinical response is defined as a rate of change of ALSFRS-R of +6 points over 6 months (mean of +1 point per month), where typically patients with ALS have a decline in ALSFRS-R by an average of -1/month. (NCT01884571)
Timeframe: Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6)
Intervention | Participants (Count of Participants) |
---|
Immunosuppression Regimen | 0 |
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Number of Participants in Overall Survival.
(NCT01487577)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 15 |
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Number of Participants Who Experienced Nonrelapse Mortality.
(NCT01487577)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 0 |
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Number of Participants Who Experienced Relapse.
(NCT01487577)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 4 |
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Number of Participants With Grade ≥3 Toxicities Scored According to the CTCAE Version 4.0.
(NCT01487577)
Timeframe: 100 days
Intervention | participants (Number) |
---|
Mycophenolate Mofetil | 0 |
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Number of Participants With Acute Grade II-IV GVHD, Acute Grade III-IV GVHD, and Chronic GVHD.
Acute GVHD will be graded according to the Modified Glucksberg Staging Criteria. Chronic GVHD will be graded according to NIH Chronic GVHD Consensus Guidelines. (NCT01487577)
Timeframe: 1 year
Intervention | participants (Number) |
---|
| Acute grade II-IV GVHD | Acute grade III-IV GVHD | Chronic GVHD |
---|
Mycophenolate Mofetil | 6 | 4 | 2 |
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Number of Participants With Neutrophil and Platelet Engraftment.
Neutrophil and platelet engraftment definitions as defined by the CIBMTR Data Management Manual. (NCT01487577)
Timeframe: 100 days
Intervention | participants (Number) |
---|
| Incidence of neutrophil engraftment | Incidence of platelet engraftment |
---|
Mycophenolate Mofetil | 18 | 15 |
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Pharmacokinetic Analysis of MMF AUC to Evaluate MMF Dose Relationships to Drug Exposure.
Pharmacokinetic analysis includes, but is not limited to, area under the plasma concentration versus time curve (AUC). (NCT01487577)
Timeframe: 100 days
Intervention | mcg*hr/mL (Median) |
---|
| AUC, Intermittent IV dosing | AUC, Continuous infusion | AUC, Intermittent PO dosing |
---|
Mycophenolate Mofetil | 46.5 | 40.1 | 36.1 |
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Pharmacokinetic Analysis of MMF Clearance to Evaluate MMF Dose Relationships to Drug Exposure.
Pharmacokinetic analysis includes, but is not limited to, clearance. (NCT01487577)
Timeframe: 100 days
Intervention | mL/min/kg (Median) |
---|
| Clearance, Intermittent IV dosing | Clearance, Continuous infusion | Clearance, Intermittent PO dosing |
---|
Mycophenolate Mofetil | 10.2 | 15.1 | 10.9 |
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Pharmacokinetic Analysis of MMF Steady State Concentration to Evaluate MMF Dose Relationships to Drug Exposure.
Pharmacokinetic analysis includes, but is not limited to, steady-state concentrations. (NCT01487577)
Timeframe: 100 days
Intervention | mcg/mL (Median) |
---|
| Steady-state concentration, Intermittent IV dosing | Steady-state concentration, Continuous infusion | Steady-state concentration, Intermittent PO dosing |
---|
Mycophenolate Mofetil | 1.9 | 1.7 | 1.5 |
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Overall Survival
Number of patients surviving 18 months post-transplant. (NCT00060424)
Timeframe: At 18 months
Intervention | Participants (Count of Participants) |
---|
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis) | 15 |
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Rate of Relapse
Number of patients with relapsed disease post-transplant. Relapse/progression is defined as 1) Physical exam/Imaging studies (nodes, liver, and/or spleen) ≥50% increase or new, 2) circulating lymphocytes by morphology and/or flow cytometry ≥50% increase, or 3) lymph node Biopsy Richter's transformation. (NCT00060424)
Timeframe: 18 months
Intervention | Participants (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.
Intervention | Participants (Count of Participants) |
---|
| Acute GVHD | Chronic extensive GVHD |
---|
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis) | 10 | 10 |
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Rate and Types of Infections
Number of infections patients experienced, by infection type. (NCT00060424)
Timeframe: 18 months
Intervention | infections (Number) |
---|
| Viral | Fungal | Fever of unknown origin | Bacterial | Other |
---|
Treatment (Enzyme Inhibitor, Transplant, GVHD Prophylaxis) | 27 | 13 | 6 | 53 | 5 |
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Duration of Renal Response in Participants Who Achieve Partial or Complete Renal Response at Any Time During the Study
Number of days between first visit with response to last consecutive visit with partial or complete response. Complete renal response is defined as: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). Estimated from the Kaplan-Meier Curve. (NCT01499355)
Timeframe: up to Week 52
Intervention | days (Mean) |
---|
Placebo | 48.3 |
BIIB023 3 mg/kg | 45.6 |
BIIB023 20 mg/kg | 52.1 |
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Percentage of Participants Who Achieve a Complete or Partial Renal Response at Week 52
Complete renal response is defined as: (1) urinary protein:creatinine ratio (uPCR) < 0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline; from a 24 hour urine collection); and (2) estimated glomerular filtration rate (eGFR) within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). (NCT01499355)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
Placebo | 25 |
BIIB023 3 mg/kg | 16 |
BIIB023 20 mg/kg | 31 |
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Percentage of Participants Who Achieve Complete Renal Response at Week 52
Complete renal response is defined as uPCR < 0.5 mg/mg with ≥ 50% reduction of uPCR from Baseline (from a 24-hour urine collection) and eGFR within normal range. (NCT01499355)
Timeframe: Week 52
Intervention | percentage of participants (Number) |
---|
Placebo | 6 |
BIIB023 3 mg/kg | 8 |
BIIB023 20 mg/kg | 8 |
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Percentage of Participants With Active Urinary Sediment at Baseline Who Have Inactive Urinary Sediment at Week 52
Active urinary sediment is defined by 1 of the following (in the absence of a urinary tract infection or menses): > 5 red blood cell/high power field (RBC/HPF) or above the reference range for the laboratory, and > 5 white blood cell/high power field (WBC/HPF) or above the reference range for the laboratory, and presence of cellular casts (RBC or WBC). Inactive urinary sediment is defined as: < 5 RBC/HPF and < 5 WBC/HPF, or within the laboratory reference range, and no cellular casts (no RBC or WBC casts). (NCT01499355)
Timeframe: Baseline, Week 52
Intervention | percentage of participants (Number) |
---|
Placebo | 38 |
BIIB023 3 mg/kg | 5 |
BIIB023 20 mg/kg | 21 |
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Percentage of Participants With uPCR > 3.0 mg/mg at Baseline Who Achieve uPCR <1.0 mg/mg at Week 52
(NCT01499355)
Timeframe: Baseline (Day 1), Week 52
Intervention | percentage of participants (Number) |
---|
Placebo | 0 |
BIIB023 3 mg/kg | 22 |
BIIB023 20 mg/kg | 13 |
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Time to Renal Response (Partial or Complete) in Participants Who Achieve Renal Response at Week 52
Onset of renal response was calculated as weeks elapsed from baseline date to first visit where renal response was achieved. Complete renal response is defined as: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. Partial renal response is defined as: (1) ≥ 50% reduction in uPCR from Day 1 (Baseline; from a 24-hour urine collection) and, (2) with one of the following: (a) uPCR of < 1.0 mg/mg if the Day 1 (Baseline) was ≤ 3.0 mg/mg, or, (b) uPCR < 3.0 mg/mg if the Day 1 (Baseline) ratio was > 3.0 mg/mg; and stabilization of renal function (eGFR + or - 25% of Day 1 [Baseline] or serum creatinine within normal range). Estimated from the Kaplan-Meier Curve. (NCT01499355)
Timeframe: Baseline to Week 52
Intervention | weeks (Median) |
---|
Placebo | 10.6 |
BIIB023 3 mg/kg | 5.2 |
BIIB023 20 mg/kg | 4.1 |
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Duration of Renal Response in Participants Who Achieve Complete Renal Response at Week 52
Duration of response was calculated as the days in between the date of Week 52 visit and the date when the participant last became complete renal responder on or before Week 52 visit. Complete renal response: (1) uPCR <0.5 mg/mg with ≥ 50% reduction of uPCR from Day 1 (Baseline) (from a 24 hour urine collection); and (2) eGFR within normal range. (NCT01499355)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
| 1-day duration | 27-day duration | 78-day duration | 141-day duration | 169-day duration |
---|
BIIB023 20 mg/kg | 2 | 1 | 0 | 0 | 1 |
,BIIB023 3 mg/kg | 3 | 0 | 1 | 0 | 0 |
,Placebo | 2 | 0 | 0 | 1 | 0 |
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Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs Leading to Study Discontinuation During the Run-In Period
AEs that had an onset on or after dosing of MMF on run-in Day 1 up to the first double-blind dose, or any pre-existing condition that worsened. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed above. (NCT01499355)
Timeframe: Day 1 to Week 12
Intervention | participants (Number) |
---|
| Any Event | Moderate or Severe Event | Severe Event | Related Event to MMF | Serious Event | Related Serious Event to MMF | Fatal Event | Discontinued Treatment Due to Event | Withdrew From Study Due to Event |
---|
Run-In: All Enrolled Participants | 209 | 94 | 18 | 90 | 28 | 12 | 2 | 0 | 10 |
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Number of Participants With AEs, SAEs and AEs Leading to Study Discontinuation During the Double-Blind Period
AEs that had an onset on or after dosing of BIIB023 or placebo, or any pre-existing condition that worsened. AE: any untoward medical occurrence that does not necessarily have a causal relationship with this treatment. SAE: any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; or results in a congenital anomaly/birth defect. An SAE may also be any other medically important event that, in the opinion of the Investigator, may jeopardize the participant or may require intervention to prevent one of the other outcomes listed above. (NCT01499355)
Timeframe: Week 12 to Week 56
Intervention | participants (Number) |
---|
| Any event | Moderate or severe event | Severe event | Event related to double-blind treatment | Event related to MMF | Serious event | Serious event related to double-blind treatment | Serious event related to MMF | Fatal event |
---|
BIIB023 20 mg/kg | 53 | 22 | 5 | 11 | 22 | 10 | 2 | 3 | 0 |
,BIIB023 3 mg/kg | 60 | 32 | 6 | 15 | 24 | 11 | 3 | 4 | 0 |
,Placebo | 48 | 26 | 4 | 5 | 21 | 7 | 3 | 6 | 1 |
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Acute Graft-versus-Host Disease (GVHD) Grade 2-4 Risk From Time of Transplant Until Day 90 Post-transplant
GVHD grading system goes from 0-4 where grade 4 is the most severe. Grade 0 and 1 do not require systemic treatment, Grade 2-4 require treatment. This trial evaluated the risk of developing acute GVHD grades 2-4 within 90 days of transplant. (NCT00185692)
Timeframe: 90 days
Intervention | Participants (Count of Participants) |
---|
Transplantation of CD34+ Cells | 1 |
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Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning
number achieving donor cell engraftment (>95%) by day 90 after transplant. (NCT00185692)
Timeframe: 100 days
Intervention | Participants (Count of Participants) |
---|
Transplantation of CD34+ Cells | 4 |
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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
Intervention | percentage 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
Intervention | percentage 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
Intervention | percentage of participants (Number) |
---|
| CD3 - Graft rejection | CD3 - Mixed chimerism | CD3 - Full donor chimerism | CD3 - Unknown | CD33 - Graft Rejection | CD33 - Mixed chimerism | CD33 - Full donor chimerism | CD33 - Unknown |
---|
Dose Level 1 (No Campath) | 3.3 | 18.3 | 70 | 8.33 | 1.7 | 3.3 | 80 | 15 |
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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
Intervention | percentage of participants (Number) |
---|
| Grade III-IV aGVHD | cGVHD |
---|
Dose Level 1 (No Campath) | 23.3 | 41.7 |
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Donor Engraftment (Chimerism)
Defined by the detection of at least 50% donor derived T-cells (CD3+), as a proportion of the total T-cell population (NCT00049504)
Timeframe: At day +84 after transplantation
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Nonmyeloablative HSCT) | 4 |
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Response Rates
(NCT00119392)
Timeframe: Up to 8 years
Intervention | Participants (Count of Participants) |
---|
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant) | 25 |
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Engraftment and Hematopoietic Toxicity
Median number of days after transplantation to a neutrophil count less than 500 neutrophils per microliter and a platelet count less than 50,000 platelets per microliter. (NCT00119392)
Timeframe: At day +100
Intervention | days (Median) |
---|
| Neutrophils | Platelets |
---|
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant) | 17 | 11 |
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Incidence and Severity of Acute Graft-versus-host Disease (GVHD) and Chronic GVHD.
(NCT00119392)
Timeframe: At day +84
Intervention | Participants (Count of Participants) |
---|
| Acute GVHD: Grade 1-2 | Acute GVHD: Grade 3 | Chronic extensive GVHD |
---|
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant) | 27 | 4 | 5 |
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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
Intervention | percent (Number) |
---|
| Overall survival | Progression free survival |
---|
Treatment (90Y Ibritumomab Tiuxetan, Hematopoietic Transplant) | 54 | 31 |
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Mean Percent Change in Calculated Creatinine Clearance From Baseline to Months 6, 12, and 24
"Renal allograft function determined by mean percent change from baseline in calculated creatinine clearance (Cockroft and Gault method) by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(calculated creatinine clearance at Month t - calculated creatinine clearance at baseline)/calculated creatinine clearance at baseline]*100 percent, where t=6, 12, and 24 months postrandomization" (NCT00121810)
Timeframe: baseline 6, 12, and 24 months
Intervention | percent change (Mean) |
---|
| Baseline | Percent Change from Baseline at Month 6 | Percent Change from Baseline at Month 12 | Percent Change from Baseline at Month 24 |
---|
Mycophenolate Mofetil + Cyclosporine or Tacrolimus | 60.5 | -1.7 | -2.3 | -4.2 |
,Mycophenolate Mofetil + Sirolimus | 59.7 | 7.0 | 4.4 | 4.7 |
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Mean Percent Change in Calculated Glomerular Filtration Rate From Baseline to Months 6, 12, and 24 (Nankivell Equation)
"Renal allograft function determined by mean percent change from baseline in calculated Glomerular Filtration Rate (Nankivell equation) by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(calculated Glomerular Filtration Rate at Month t - calculated Glomerular Filtration Rate at baseline)/calculated Glomerular Filtration Rate at baseline]*100 percent, where t=6, 12, and 24 months postrandomization." (NCT00121810)
Timeframe: baseline, 6, 12, and 24 months
Intervention | percent change (Mean) |
---|
| Baseline | Percent Change from Baseline at Month 6 | Percent Change from Baseline at Month 12 | Percent Change from Baseline at Month 24 |
---|
Mycophenolate Mofetil + Cyclosporine or Tacrolimus | 72.7 | -0.5 | -0.9 | -1.8 |
,Mycophenolate Mofetil + Sirolimus | 71.3 | 8.3 | 5.2 | 6.5 |
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Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 12
"The primary efficacy endpoint was mean percent change in renal function from baseline to 12 months postrandomization, as measured by Glomerular Filtration Rate utilizing renal clearance of cold iothalamate.~percent change= [(Glomerular Filtration Rate at Month 12-Glomerular Filtration Rate at baseline)/Glomerular Filtration Rate at baseline]*100 percent." (NCT00121810)
Timeframe: baseline to 12 months
Intervention | percent change (Mean) |
---|
| Baseline | Mean Percent Change from Baseline at Month 12 |
---|
Mycophenolate Mofetil + Cyclosporine or Tacrolimus | 58.8 | 5.2 |
,Mycophenolate Mofetil + Sirolimus | 59.5 | 24.4 |
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Mean Percent Change in Glomerular Filtration Rate From Baseline to Month 24
"A secondary efficacy endpoint was mean percent change in renal function from baseline to 24 months postrandomization, as measured by Glomerular Filtration Rate utilizing renal clearance of cold iothalamate.~percent change= [(Glomerular Filtration Rate at Month 24-Glomerular Filtration Rate at baseline)/Glomerular Filtration Rate at baseline]*100 percent." (NCT00121810)
Timeframe: Baseline to 24 months
Intervention | percent change (Mean) |
---|
| Baseline | Percent Change from Baseline at Month 24 |
---|
Mycophenolate Mofetil + Cyclosporine or Tacrolimus | 58.8 | 3.4 |
,Mycophenolate Mofetil + Sirolimus | 59.5 | 8.6 |
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Mean Percent Change in Serum Creatinine From Baseline to Months 6, 12, and 24
"Renal allograft function determined by mean percent change from baseline in serum creatinine by treatment group at 6, 12, and 24 months postrandomization.~percent change= [(serum creatinine at Month t-serum creatinine at baseline)/serum creatinine at baseline]*100 percent, where t=6, 12, and 24 months postrandomization." (NCT00121810)
Timeframe: baseline, 6, 12, and 24 months
Intervention | percent change (Mean) |
---|
| Baseline | Percent Change from Baseline at Month 6 | Percent Change from Baseline at Month 12 | Percent Change from Baseline at Month 24 |
---|
Mycophenolate Mofetil + Cyclosporine or Tacrolimus | 124.4 | 6.6 | 20.4 | 30.8 |
,Mycophenolate Mofetil + Sirolimus | 121.1 | -3.7 | 6.0 | 6.1 |
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Overall Survival
measured from date of registration to study until death from any cause with patients still alive censored at date of last contact (NCT00053014)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Treatment | 1 |
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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
Intervention | participants (Number) |
---|
Treatment | 0 |
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Count of Participants Diagnosed With Epstein-Barr Virus (EBV) Infection as an Adverse Event
Viral infections following renal transplantation is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during this study using participant blood samples. Displayed are counts of all participants diagnosed with EBV infection as an adverse event by EBV test(s), diagnosed by test results from the local clinical pathology laboratory. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 0 |
Control | 0 |
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Count of Participants Diagnosed With Malignancy as an Adverse Event
An increased risk/incidence of malignancy is a recognized complication of immunosuppression in recipients of organ transplants. In Phase 3 clinical trials, overall malignancy rates were similar across all treatment groups, with the exception of posttransplant lymphoproliferative disease (PTLD).Displayed are counts of all participants who experienced malignancy reported as an adverse event. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 0 |
Control | 0 |
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Count of Participants With an Infectious Disease Serious Adverse Event(s) Requiring Hospitalization or Systemic Therapy
Infections were required to be reported as a serious adverse event if they required either inpatient hospitalization or prolongation of a current hospitalization. Displayed are counts of all participants who experienced infection(s) as an adverse event, by treatment group. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 11 |
Control | 11 |
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Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant
"The stages of Chronic Kidney Disease (CKD) are defined using the participant's GFR value:~Stage 1 if GFR value is ≥ 90 (kidney function is normal)~Stage 2 if 60 ≤ GFR < 90 (mildly reduced kidney function, pointing to kidney disease)~Stage 3A if 45 <= GFR < 60*~Stage 3B if 30 <= GFR < 45*~Stage 4 if 15 ≤ GFR < 30 (severely reduced kidney function)~Stage 5 if GFR < 15 (severe or end stage kidney failure).~Stages 3A abd 3B indicate moderately reduced kidney function.*" (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 0 |
Control | 0 |
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Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant
Delayed grafted function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function. (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 1 |
Control | 1 |
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Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant
"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 5 |
Control | 5 |
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Count of Participants With Evidence of Pancreatic Loss at Week 52 Post-Transplant
C-peptide is a measure of pancreatic function. The definition of pancreatic loss: a C-peptide value of <0.3 ng/mL. (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 1 |
Control | 0 |
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Count of Participants With Evidence of Partial Pancreatic Graft Function at Week 52 Post-Transplant
C-peptide is a measure of pancreatic function. The definition of partial pancreatic graft function: a fasting C-peptide levels >0.3ng.mL (0.1nmol.L) plus the participant's continued requirement for exogenous insulin or oral hypoglycemic agent(s). (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 1 |
Control | 0 |
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Count of Participants With Full Pancreatic Graft Function (Insulin Independent) at Wk 52 Post-Transplant
Participants with full pancreatic graft functions are defined as those that no longer require exogenous insulin therapy. (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 19 |
Control | 21 |
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Count of Participants With Successful Discontinuation of Tacrolimus in Recipients Randomized to the Investigational Arm
Participants achieved successful discontinuation if they were able to discontinue (e.g., off tacrolimus therapy completely) over a 4-8 weeks after tacrolimus withdrawal was initiated at week 40. (NCT01790594)
Timeframe: Week 40 through week 48 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
Investigational | 5 |
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Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant
"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate severe or endstage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | mL/min/1.73m^2 (Mean) |
---|
Investigational | 68.7 |
Control | 67.0 |
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Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant
"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | mL/min/1.73m^2 (Mean) |
---|
Investigational | 77.0 |
Control | 74.6 |
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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant
"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or endstage kidney failure.~An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it could be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function.~Larger numbers indicate greater change in kidney function." (NCT01790594)
Timeframe: Day 28 through Week 52 Post-Transplant
Intervention | eGFR change over time (by month) (Mean) |
---|
Investigational | 0.1 |
Control | -0.1 |
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Count of Participant Diagnosed With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia As Adverse Events
"Viral infections following renal transplantation is a significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss.~Specific viruses were monitored during this study using participant blood samples. Displayed are counts of participants who experienced BKV and CMV viremia as adverse events, diagnosed by test results from the local clinical pathology laboratory." (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Count of Participants (Number) |
---|
| BK Viremia | CMV Viremia |
---|
Control | 3 | 3 |
,Investigational | 8 | 5 |
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Count of Participants With Acute Rejection (AR) of Kidney or Pancreatic Transplant During the First 52 Wks Post-Transplant
"Biopsy-proven acute rejection (AR) of the kidney (renal) or pancreas during the first 52 weeks post-transplant. AR grading using standard Banff* criteria. For both kidney and pancreas, AR is defined as a grade ≥1.~AR for the kidney: Banff 2007 criteria. Severity of AR is graded by as IA, IB, IIA, IIB, or III, with IA defined as the mildest form of AR and III being the most severe.~AR for the pancreas: Banff 2011 Criteria. Severity of AR is graded as I, II, or III, with I defined as the mildest form of AR and III being the most severe." (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Count of Participants (Number) |
---|
| Kidney | Pancreas |
---|
Control | 2 | 1 |
,Investigational | 2 | 5 |
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Count of Participants With Biopsy-Proven Humoral Rejection During the First 52 Weeks Post-Transplant
"Humoral rejection (i.e., antibody mediated rejection) of:~the kidney as defined by diffusely positive staining for C4d, presence of circulating anti-donor antibodies, and morphologic evidence of acute tissue injury determined by local pathology and,~the pancreas as defined by the presence of circulating anti-donor antibodies, and histopathological data including morphologic evidence of microvascular tissue injury and C4d staining in interacinar capillaries determined by local pathology." (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Count of Participants (Number) |
---|
| Kidney | Pancreas |
---|
Control | 0 | 0 |
,Investigational | 0 | 0 |
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Count of Participants With De Novo Anti-Donor Antibodies or Anti-Human Leukocyte Antigen (HLA) Antibodies During the First 52 Weeks Post-Transplant
"The de novo development of donor-specific antibody (DSA) is associated with an increased risk of graft rejection.~The presence of anti-Histocompatibility Antigen (HLA) antibodies (alloantibodies) is associated with increased risk of acute and chronic injury to the transplant allograft." (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Count of Participants (Number) |
---|
| De novo DSA | Anti-HLA |
---|
Control | 0 | 1 |
,Investigational | 0 | 2 |
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Count of Participants With Event of Death, Graft Loss, or Undetectable C-peptide
"This measure counts death, graft loss, or undetectable C-peptide value (e.g., C-peptide <0.3 ng/mL) occurring at any point post-transplant and independent of each other.~Kidney Graft Loss was defined as 90 consecutive days of dialysis dependency.~Pancreas graft loss was defined as returning to exogenous insulin therapy or initiation of oral hypoglycemic agents for greater than 30 days.~Factitious hypoglycemia due to surreptitious insulin administration results in elevated serum insulin levels and low or undetectable C-peptide levels." (NCT01790594)
Timeframe: Transplant through Week 52 Post-Transplant
Intervention | Participants (Count of Participants) |
---|
| Death | Kidney Graft Loss | Pancreas Graft Loss | Undetectable C-peptide |
---|
Control | 0 | 0 | 0 | 0 |
,Investigational | 1 | 0 | 1 | 0 |
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Count of Participants With the Occurrence of Adverse Events (AEs) and Serious Adverse Events (SAEs)
"Adverse events were collected systematically. Counts of all participants who experienced at least one adverse event (AEs, SAEs) by assigned treatment group.~Refer to the Serious Adverse Events and Other Adverse Events tables for more detail." (NCT01790594)
Timeframe: From Enrollment (Pre-Transplant) to Week 52 Post-Transplant
Intervention | Count of Participants (Number) |
---|
| All Adverse Events | Serious Adverse Events |
---|
Control | 21 | 19 |
,Investigational | 22 | 20 |
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Count of Participants With Use of Anti-hypertensive Medication From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stoke and myocardial infarction. (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52
Intervention | Count of Participants (Number) |
---|
| Baseline | Day 28 | Day 84 | Week 28 | Week 36 | Week 52 |
---|
Control | 19 | 10 | 7 | 11 | 11 | 11 |
,Investigational | 18 | 14 | 11 | 13 | 13 | 13 |
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Count of Participants With Use of Lipid Lowering Medications at Baseline, Wk 28 and Wk 52 Post-Transplant
Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood (NCT01790594)
Timeframe: Baseline (Pre-Transplant), Week 28, and Week 52
Intervention | participants (Number) |
---|
| Baseline | Week 28 | Week 52 |
---|
Control | 18 | 16 | 16 |
,Investigational | 18 | 19 | 19 |
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Fasting Blood Sugar (FBS) From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
"Fasting blood sugar (e.g., glucose) test is used to help diagnose diabetes, prediabetes, and gestational diabetes.~Reference fasting blood sugar (glucose) values:~70 to 99 mg/dL is normal~100 to 125 mg/dL is considered prediabetes~126 mg/dL or higher on two separate tests is considered diabetes." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52
Intervention | mg/dL (Mean) |
---|
| Baseline | Day 28 | Day 84 | Week 28 | Week 36 | Week 52 |
---|
Control | 217.3 | 100.9 | 89.7 | 96.2 | 87.2 | 91.5 |
,Investigational | 183.3 | 100.0 | 96.0 | 106.5 | 96.0 | 98.6 |
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Fasting Lipid Profile at Baseline (Pre-Transplant)
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Baseline (Pre-Transplant)
Intervention | mg/dL (Mean) |
---|
| Tot. Chol. Baseline | Non-HDL Baseline | LDL Baseline | HDL Baseline | Triglyc. Baseline |
---|
Control | 140.5 | 82.4 | 60.7 | 58.1 | 107.5 |
,Investigational | 142.8 | 91.0 | 66.5 | 51.8 | 120.9 |
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Fasting Lipid Profile at Wk 28 Post-Transplant
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Week 28 Post-Transplant
Intervention | mg/dL (Mean) |
---|
| Tot. Chol. Week 28 | Non-HDL Week 28 | LDL Week 28 | HDL Week 28 | Triglyc. Week 28 |
---|
Control | 149.2 | 97.4 | 81.2 | 51.8 | 87.8 |
,Investigational | 159.9 | 112.6 | 93.1 | 47.3 | 93.2 |
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HbA1c at Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
"Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control:~A value below 6.0% reflects normal levels,~6.0% to 6.4% reflects prediabetes, and~a value of ≥ 6.5% reflects diabetes." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52
Intervention | percent (Mean) |
---|
| Baseline | Day 28 | Day 84 | Week 28 | Week 36 | Week 52 |
---|
Control | 8.5 | 6.1 | 4.9 | 5.2 | 5.1 | 5.3 |
,Investigational | 8.6 | 6.0 | 4.8 | 5.3 | 5.3 | 5.5 |
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Lipid Profile at Wk 52 Post-Transplant
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01790594)
Timeframe: Week 52 Post-Transplant
Intervention | mg/dL (Mean) |
---|
| Tot. Chol. Week 52 | Non-HDL Week 52 | LDL Week 52 | HDL Week 52 | Triglyc. Week 52 |
---|
Control | 162.8 | 112.3 | 92.7 | 47.3 | 95.4 |
,Investigational | 164.2 | 115.7 | 96.9 | 48.5 | 88.6 |
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Severity Grade of First Biopsy-Proven Acute Rejection (AR) During the First 52 Weeks Post-Transplant
"AR grading using standard Banff* criteria. For both kidney and pancreas, AR is defined as a grade ≥1.~AR for the kidney: Banff 2007 criteria. Severity of AR is graded by as IA, IB, IIA, IIB, or III, with IA defined as the mildest form of AR and III being the most severe.~AR for the pancreas: Banff 2011 Criteria. Severity of AR is graded is I, II, or III, with I defined as the mildest form of AR and III being the most severe." (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Participants (Number) |
---|
| Kidney First Grade IA | Kidney First Grade IB | Kidney First Grade IIA | Kidney First Grade IIB | Pancreas First Grade I | Pancreas First Grade II |
---|
Control | 1 | 0 | 1 | 0 | 0 | 1 |
,Investigational | 0 | 1 | 0 | 1 | 4 | 1 |
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Standardized Blood Pressure Measurement From Baseline (Pre-Transplant) Through Wk 52 Post-Transplant
"A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart.~Systolic measures of <120 and diastolic measures of <80 are considered normal.~Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension).~Systolic measures of ≥140 and diastolic measures of ≥90 are considered high." (NCT01790594)
Timeframe: Baseline (Pre-Transplant) and Days 28, 84, and Weeks 28, 36, and 52
Intervention | mmHg (Mean) |
---|
| Systolic BP at Baseline | Systolic BP at Day 28 | Systolic BP at Day 84 | Systolic BP at Week 28 | Systolic BP at Week 36 | Systolic BP at Week 52 | Diastolic BP at Baseline | Diastolic BP at Day 28 | Diastolic BP at Day 84 | Diastolic BP at Week 28 | Diastolic BP at Week 36 | Diastolic BP at Week 52 |
---|
Control | 158.3 | 112.0 | 123.8 | 126.2 | 126.7 | 127.0 | 85.2 | 65.4 | 73.4 | 73.3 | 76.8 | 77.0 |
,Investigational | 161.3 | 116.9 | 126.3 | 136.1 | 133.5 | 132.0 | 82.7 | 67.0 | 72.8 | 76.6 | 76.5 | 74.2 |
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Type of Treatment(s) Participants Received for Biopsy-Proven Pancreatic Allograft Rejection During the First 52 Weeks Post-Transplant
"Participants are stratified by kidney biopsy results/treatment received.~Upon having a for-cause biopsy performed, persons often receive treatment for rejection based on the biopsy results, which may or may not reveal signs of rejection. Details of biopsy findings and corresponding treatment are provided for each instance of treatment for rejection.~Results summary format: biopsy results; treatment.~Acronyms and abbreviations:~ACR=Acute Cellular Rejection~IFTA=Interstitial Fibrosis and Tubular Atrophy~ATG=Anti-thymocyte globulin therapy~IVIG=Intravenous Immunoglobulin therapy~Gd =Grade~PO=Orally~QD=Daily" (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Participants (Count of Participants) |
---|
| ACR-Gd. I/ATG | ACR-Gd. I/ATG, Pulse Steroids | ACR-Gd. I/ATG, Pulse Steroids, IVIG | ACR-Gd. I, IFTA-Gd. I/Pulse Steroids, Solumedrol, | ACR-Gd. II/ATG, Pulse Steroids | No grade reported/None |
---|
Control | 0 | 0 | 0 | 0 | 1 | 0 |
,Investigational | 1 | 1 | 1 | 1 | 1 | 1 |
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Type of Treatment(s) Participants Received for Biopsy-Proven Renal Allograft Rejection During the First 52 Weeks Post-Transplant
"Participants are stratified by kidney biopsy results/treatment received.~In the event of a for cause renal (kidney) biopsy:~-The diagnosis of acute cellular rejection (ACR) using the Banff 2007 renal allograft pathology criteria. These criteria for renal allograft biopsies is an international histopathological classification standard. ACR is defined by a renal biopsy demonstrating a Banff 2007 classification of Grade IA or greater, with higher scores indicating more severe rejection. (Ref: Solez K, Colvin RB et al. Banff 07 classification of renal allograft pathology: updates and future directions. Am J Transplant 2008 8(4): 753-60).~Acronyms and abbreviations:~ACR=Acute Cellular Rejection*~Normal*~Borderline* (criteria for ACR not fulfilled)~Gd.=Grade*~IFTA=Interstitial Fibrosis and Tubular Atrophy*~ATG=Anti-thymocyte globulin therapy~IVIG=Intravenous Immunoglobulin therapy~PO=Orally~QD=Daily *Banff 2007 renal allograft pathology criteria" (NCT01790594)
Timeframe: Transplant through Week 52
Intervention | Participants (Count of Participants) |
---|
| Borderline/None | Borderline/Pulse Steroids | Borderline, IFTA-Gd. I/Pulse Steroids, IVIG | ACR-Gd. IA/ATG, Pulse Steroids | ACR-Gd. IB/ATG, Pulse Steroids | ACR-Gd. IIA/ATG, Pulse Steroids | ACR-Gd. IIB/ATG, Pulse Steroids | IFTA-Gd. I/None | IFTA-Gd. I/Potassium citrate | Normal/Steroids QD | No grade reported/IVIG | No grade reported/None | Normal/None |
---|
Control | 0 | 1 | 1 | 1 | 0 | 1 | 0 | 0 | 0 | 1 | 0 | 4 | 2 |
,Investigational | 1 | 0 | 0 | 0 | 1 | 0 | 1 | 1 | 2 | 0 | 1 | 1 | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation) | 3 |
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation) | 0 |
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Incidence of Grades III-IV Acute GVHD
"Number of subjects who developed maximum grade acute graft-vs-host disease~aGVHD Stages~Skin:~- a maculopapular eruption involving < 25% BSA~- a maculopapular eruption involving 25 - 50% BSA~- generalized erythroderma~- generalized erythroderma with bullous formation and often with desquamation~Liver:~- bilirubin 2.0 - 3.0 mg/100 mL~- bilirubin 3 - 5.9 mg/100 mL~- bilirubin 6 - 14.9 mg/100 mL~- bilirubin > 15 mg/100 mL~Gut:~Diarrhea is graded 1 - 4 in severity. Nausea and vomiting and/or anorexia caused by GVHD is assigned as 1 in severity. The severity of gut involvement is assigned to the most severe involvement noted. Patients with visible bloody diarrhea are at least stage 2 gut and grade 3 overall.~aGVHD Grades Grade III: Stage 2 - 4 gastrointestinal involvement and/or +2 to +4 liver involvement, with or without a rash Grade IV: Pattern and severity of GVHD similar to grade 3 with extreme constitutional symptoms or death" (NCT00453388)
Timeframe: Up to Day 100
Intervention | Participants (Count of Participants) |
---|
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation) | 1 |
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation) | 0 |
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Number of Patients Who Engraft at Each Dose of TBI Used
Number of subjects who engrafted. Engraftment defined as greater than 95% donor chimerism. (NCT00453388)
Timeframe: Up to Day 200
Intervention | Participants (Count of Participants) |
---|
Arm II (2 vs 2.5 vs 3 vs 1 vs 0 Gy TBI De-escalation) | 5 |
Arm III (2 vs 2.5 vs 3 Gy TBI Dose-escalation) | 1 |
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Tolerability - Number of Participants Who Completed Each Arm on Assigned Study Drug Dose
The primary outcome measure is tolerability, defined as the completion of 8 weeks on the assigned dosage of study drug. (NCT01399047)
Timeframe: Baseline to 8 weeks
Intervention | Participants (Count of Participants) |
---|
Mycophenolate | 17 |
Placebo | 19 |
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Unified Batten Disease Rating Scale Behavior Subscale Change
"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Mood and behavior severity was measured by the behavior subscale of the UBDRS with a range of 0-55 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in behavior subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks
Intervention | units on a scale (Mean) |
---|
Mycophenolate | -1.28 |
Placebo | -0.37 |
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Unified Batten Disease Rating Scale Capability Subscale Change
"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Capability severity was measured by the capability subscale of the UBDRS with a range of 0-14 with higher scores indicating better outcome.~The overall treatment effect was determined - mean difference in capability subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks
Intervention | units on a scale (Mean) |
---|
Mycophenolate | 0.33 |
Placebo | 0.47 |
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Unified Batten Disease Rating Scale Physical Subscale Change
"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Motor impairment was measured by the physical subscale of the UBDRS with a range of 0-112 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in physical subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks
Intervention | units on a scale (Mean) |
---|
Mycophenolate | -1.18 |
Placebo | 2.17 |
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Unified Batten Disease Rating Scale Seizure Subscale Change
"The Unified Batten Disease Rating Scale (UBDRS) is a disease-specific clinical assessment. Seizure severity was measured by the seizure subscale of the UBDRS with a range of 0-54 with zero indicating better outcome.~The overall treatment effect was determined - mean difference in seizure subscale change (Mycophenolate minus placebo periods) in outcome, adjusted for baseline value and period effects." (NCT01399047)
Timeframe: Baseline to 8 weeks
Intervention | units on a scale (Mean) |
---|
Mycophenolate | -0.24 |
Placebo | -1.44 |
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Development of Infectious Complications
The incidence of definite and probable viral, fungal and bacterial infections will be tabulated for each patient. (NCT01570348)
Timeframe: Up to 5 years
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic BMT) | 0 |
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EFS
Described graphically using a Kaplan-Meier estimate. Generated with confidence intervals using Greenwood's formula to calculate the standard error. Estimated with exact 90% confidence intervals. (NCT01570348)
Timeframe: Up to 5 years post-transplant
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic BMT) | 0 |
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Count of Participant Deaths or Graft Loss by Wk 52 Post-Transplant
This measure counts deaths and graft loss occurring at any point post transplantation. Graft loss is defined as 90 days of dialysis dependency. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 2 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 0 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 |
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Count of Participants With Acute Cellular Rejection Grade ≥ IA Defined by Banff 2007 Criteria By Wk 52 Post-Transplant
Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade equal to or greater than IA by Banff 2007 criteria as determined by local pathology. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 1 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 10 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 4 |
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Count of Participants With Antibody Mediated Rejection by Wk 52 Post-Transplant
Antibody mediated rejection is defined by diffusely positive staining for C4d, presence of circulating anti-donor antibodies, and morphologic evidence of acute tissue injury and was determined by local pathology. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 1 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 1 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 |
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Count of Participants With Biopsy Proven Acute Rejection By Wk 52 Post-Transplant
Biopsy proven acute rejection definition: histologic evidence of a Banff grade of ≥1A per local pathologist. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 1 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 10 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 4 |
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Count of Participants With Defined CKD Stage 4 or 5 at Wk 52 Post-Transplant
"The stages of Chronic Kidney Disease (CKD) are defined using the participant's GFR value:~Stage 1 if GFR value is ≥ 90 (kidney function is normal)~Stage 2 if 60 ≤ GFR < 90 (mildly reduced kidney function, pointing to kidney disease)~Stage 3A if 45 <= GFR < 60*~Stage 3B if 30 <= GFR < 45*~Stage 4 if 15 ≤ GFR < 30 (severely reduced kidney function)~Stage 5 if GFR < 15 (severe or end stage kidney failure).~Stages 3A abd 3B indicate moderately reduced kidney function.*" (NCT01856257)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 4 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 4 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 |
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Count of Participants With Delayed Graft Function at Wk 52 Post-Transplant
Delayed grafted function is defined as dialysis in the first week on one or more occasions for any indication other than the treatment of acute hyperkalemia in the setting of otherwise acceptable renal function. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 6 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 9 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 |
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Count of Participants With eGFR < 60 mL/Min/1.73 m^2 Measured by CKD-EPI at Wk 52 Post-Transplant
"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01856257)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 21 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 15 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 6 |
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Count of Participants With Graft Rejection by Wk 52 Post-Transplant
The number of participants who were treated by their local physician for any type of rejection including, but not limited to cellular rejection and antibody- mediated rejection of the transplanted kidney regardless of the presence of a biopsy. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 7 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 14 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 4 |
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Count of Participants With Infections Requiring Hospitalization or Systemic Therapy by Wk 52 Post-Transplant
Infections of certain types (i.e., excluding those identified in the protocol as occurring commonly in this study population) were required to be reported as a serious adverse event if they required either inpatient hospitalization or prolongation of a current hospitalization. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 1 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 8 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 2 |
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Count of Participants With Treated Diabetes Between Day 14 and Wk 52 Post-Transplant
Treated diabetes is defined as receipt of any oral medication or insulin for the treatment of diabetes for >14 days. (NCT01856257)
Timeframe: Day 14 through week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 3 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 2 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 |
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Count of Participants With Use of Anti-hypertensive Medication at Wk 52 Post-Transplant
Anti-hypertensive medications are a class of drugs that are used to treat hypertension. The medications seek to prevent the complications of high blood pressure, such as stroke and myocardial infarction. (NCT01856257)
Timeframe: Week 52
Intervention | Participants (Count of Participants) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 18 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 23 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 8 |
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Mean Calculated eGFR Using MDRD 4 Variable Model at Wk 52 Post-Transplant
"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Modification of Diet in Renal Disease equation (MDRD):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate severe or endstage kidney failure." (NCT01856257)
Timeframe: Week 52
Intervention | mL/min/1.73m^2 (Mean) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 56.1 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 57.0 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 58.2 |
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Mean Estimated Glomerular Filtration Rate (eGFR) Calculated for Each Treatment Group Using the CKD-EPI Equation at Wk 52 Post-Transplant
"eGFR was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or end stage kidney failure." (NCT01856257)
Timeframe: Week 52
Intervention | mL/min/1.73m^2 (Mean) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 59.2 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 61.5 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 63.0 |
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The Slope of eGFR by CKD-EPI Over Time Based on Serum Creatinine Post-Transplant
"The estimated Glomerular Filtration Rate (eGFR) was calculated using the Chronic Kidney Disease Epidemiology Collaboration equation (CKD-EPI):~A score of ≥ 90 means kidney function is normal.~A score between 60 and 89 indicates mildly reduced kidney function, pointing to kidney disease.~Scores between 30 and 59 indicates moderately reduced kidney function.~Scores between 15 and 29 indicate severely reduced kidney function.~Scores below 15 indicate very severe or endstage kidney failure.~An estimate of the slope, or change over time, in eGFR was produced using standard statistical linear modeling procedures. The estimate was then re-scaled so that it could be interpreted as a change in eGFR per month. Positive numbers indicate increasing kidney function.~Larger numbers indicate greater change in kidney function." (NCT01856257)
Timeframe: Day 28 through Week 52 Post-Transplant
Intervention | eGFR change over time (by month) (Mean) |
---|
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 0.3 |
Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 1.3 |
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0.8 |
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Count of Participants by Severity of First Acute Cellular Rejection by Wk 52 Post-Transplant
Acute cellular rejection occurs when lesions at the site of the graft characteristically are infiltrated with large numbers of lymphocytes and macrophages that cause tissue damage. Acute cellular rejection for this endpoint is defined as a grade equal to or greater than IA by Banff 2007 criteria as determined by local pathology. Severity is graded as IA, IB, IIA, IIB, or III, with IA being the mildest form of cellular rejection and III being the most severe form of cellular rejection. Originally it was 2 endpoints but all participants' highest grade was also their first grade so only reporting their first grade. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
| IA | IB | IIA | IIB | III |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 2 | 2 | 0 | 0 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 3 | 1 | 4 | 0 | 2 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 0 | 1 | 0 | 0 | 0 |
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Count of Participants Experiencing ≥ 1 Adverse Event (AEs) or Serious Adverse Events (SAEs) by Wk 52
Adverse events were collected systematically from enrollment through Wk 52, the last study visit. Provided are numbers of participants with ≥ 1 adverse event (serious or non-serious adverse events) by treatment arm. (NCT01856257)
Timeframe: Enrollment through Week 52
Intervention | Participants (Count of Participants) |
---|
| Adverse Events | Serious Adverse Events |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 9 | 6 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 28 | 21 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 21 | 19 |
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Count of Participants With BK Polyoma Virus (BKV) and Cytomegalovirus (CMV) Viremia (Local Center Monitoring) as Adverse Events by Wk 52 Post-Transplant
Viral infections following renal transplantation is significant source of recipient morbidity and mortality, and a significant cause of allograft dysfunction and loss. Specific viruses were monitored during the study, using participant blood samples. Displayed are counts of participants who experienced BKV and CMV viremia as adverse events by treatment arm. (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
| BKV | CMV |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 1 | 1 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 4 | 6 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 0 | 1 |
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Count of Participants With Either New Onset Diabetes After Transplant (NODAT) or Impaired Fasting Glucose (IFG) at Week 52 Post-Transplant -Based on Criteria Specified by the ADA and WHO
"New onset diabetes is the development of diabetes post-kidney transplant. It was identified by the clinical sites caring for each participant and reported directly in the clinical database. Impaired fasting glucose (IFG) is a determination made by referencing glucose measurements obtained from a standard chemistry panel. Any fasting glucose measure that is between 110 and 125 mg/dL is classified as IFG.~Acronyms: American Diabetes Association (ADA); World Health Organization (WHO)." (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Participants (Count of Participants) |
---|
| New onset diabetes during first 52 weeks | Impaired fasting glucose at week 52 |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 1 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 1 | 2 |
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Count of Participants With Fever > 39 Degrees Celsius and Blood Pressure < 90 mmHg Within 24 Hours of Onset of Transplant Procedure
Temperature of >39 degrees Celsius (e.g., 102.2 degrees Fahrenheit) would be an indication of fever most often in response to an infection or illness. Systolic blood pressure <90mm Hg would be an indication of low blood pressure. (NCT01856257)
Timeframe: Within 24 Hours of transplant procedure
Intervention | Participants (Count of Participants) |
---|
| Fever >39 Celsius | Systolic BP < 90 mmHg |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 0 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 0 | 0 |
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Count of Participants With Use of Lipid Lowering Medications at Baseline and Wk 28 and Wk 52 Post-Transplant
Lipid lowering medications are used in the treatment of high levels of fats (lipids), such as cholesterol in blood. (NCT01856257)
Timeframe: Baseline (Pre-Transplant), Week 28, and Week 52
Intervention | Participants (Count of Participants) |
---|
| Baseline | Week 28 | Week 52 |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 3 | 4 | 5 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 7 | 9 | 9 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 13 | 8 | 9 |
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Fasting Lipid Profile at Baseline (Pre-Transplant)
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Baseline
Intervention | mg/dL (Mean) |
---|
| Total cholesterol | Non-HDL | LDL | HDL | Triglyceride |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 167.6 | 123.4 | 65.2 | 44.1 | 227.1 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 159.4 | 116.1 | 83.1 | 43.3 | 194.4 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 167.1 | 122.0 | 91.1 | 45.1 | 156.8 |
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Fasting Lipid Profile at Wk 28 Post-Transplant
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Week 28
Intervention | mg/dL (Mean) |
---|
| Total cholesterol | Non-HDL | LDL | HDL | Triglyceride |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 167.3 | 115.0 | 82.7 | 52.3 | 166.6 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 179.5 | 133.5 | 109.8 | 46.0 | 153.3 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 169.6 | 119.6 | 95.6 | 51.0 | 126.4 |
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Fasting Lipid Profile at Wk 52 Post-Transplant
"A fasting lipid profiles measures total cholesterol, LDL cholesterol, HDL cholesterol, and triglyceride levels. These measurements are used in assessing one's risk of cardiovascular disease. Target ranges for each of these measures are provided:~Total cholesterol: 75-169 mg/dL if age ≤ 20; 100-199 mg/dL if age ≥ 21; high values indicate risk of cardiovascular disease~LDL cholesterol: <70 mg/dL for people with documented cardiovascular disease or metabolic syndrome; <100 mg/dL for people considered high risk for cardiovascular disease; <130 mg/dL for people considered low risk for cardiovascular disease; high values indicate risk of cardiovascular disease~HDL cholesterol: 40mg/dL and higher; high values indicate reduced risk of cardiovascular disease~Non-HDL cholesterol: 30 mg/dL above the target value for LDL cholesterol; high values indicate risk of cardiovascular disease and~Triglycerides: <150 mg/dL; high values indicate risk of cardiovascular disease." (NCT01856257)
Timeframe: Week 52
Intervention | mg/dL (Mean) |
---|
| Total cholesterol | Non-HDL | LDL | HDL | Triglyceride |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 174.8 | 130.8 | 94.6 | 44.0 | 182.8 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 163.7 | 121.2 | 86.3 | 42.4 | 170.0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 177.1 | 128.6 | 102.9 | 48.5 | 125.8 |
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Hemoglobin A1c (HbA1c) Measurements Over Time
"Hemoglobin A1c (HbA1c) measures the average blood glucose levels over 8-12 weeks, thus acting as a useful long-term gauge of blood glucose control:~A value below 6.0% reflects normal levels,~6.0% to 6.4% reflects prediabetes, and~a value of ≥ 6.5% reflects diabetes." (NCT01856257)
Timeframe: Baseline (Pre-Transplant) and Days 28 and -84, and Weeks 28, -36, and -52 Post-Transplant
Intervention | percentage (Mean) |
---|
| Baseline | Day 28 | Day 84 | Week 28 | Week 36 | Week 52 |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 5.9 | 5.4 | 5.4 | 5.6 | 5.5 | 5.8 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 5.7 | 5.6 | 5.5 | 6.0 | 5.9 | 6.7 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 5.7 | 5.4 | 5.6 | 6.2 | 6.7 | 7.8 |
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Standardized Blood Pressure Measurement at Wk 52 Post-Transplant
"A blood pressure measurement consists of two numbers: the systolic and diastolic pressures. Systolic pressure measures the pressure in blood vessels when the heart beats. Diastolic pressure measures the pressure in blood vessels between beats of the heart.~Systolic measures of <120 and diastolic measures of <80 are considered normal.~Systolic measures of 120-139 and diastolic measures of 80-89 are considered at risk (or pre-hypertension).~Systolic measures of ≥140 and diastolic measures of ≥90 are considered high." (NCT01856257)
Timeframe: Week 52
Intervention | mmHg (Mean) |
---|
| Systolic BP at W52 | Diastolic BP at W52 |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 132.0 | 75.7 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 133.7 | 79.1 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 135.0 | 77.7 |
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Total Daily Prescribed Pill Count
This is a measure of the total number of pills a participant was prescribed on a given day (NCT01856257)
Timeframe: Day 28, Day 84, Week 28, Week 36, and Week 52
Intervention | pills per day (Mean) |
---|
| Day 28 | Day 84 | Week 28 | Week 36 | Week 52 |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 23.5 | 21.0 | 16.6 | 13.9 | 13.7 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 18.9 | 17.2 | 15.5 | 15.4 | 15.7 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 25.8 | 22.2 | 19.7 | 19.1 | 24.4 |
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Type of Rejection Classified by Pathologist - For Cause Kidney Biopsies
"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of local biopsy findings are presented here for rejection. Acronyms and abbreviations are defined as follows:~ACR= Acute T-Cell Mediated rejection~AMR= Acute Antibody-mediated rejection~Chr. AMR=Chronic Antibody Mediated Rejection~Gd.=Grade~IFTA=Interstitial Fibrosis and Tubular Atrophy" (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Biopsy (Number) |
---|
| Borderline | Borderline, AMR-Gd. I (ATN-Like) | Borderline, AMR-Gd.I (ATN-Like), Chr.AMR, IFTA-Gd1 | Borderline, AMR-Gd. I (ATN-Like), IFTA-Gd. I | Borderline, Chr.AMR, IFTA-Gd. I | Borderline, IFTA-Gd. I | Borderline, IFTA- Gd. II | ACR-Gd. IA | ACR-Gd. IA, IFTA-Gd. I | ACR-Gd. IA, IFTA-Gd. II | ACR-Gd. IB | ACR-Gd. IB, IFTA-Gd. I | ACR-Gd. IB, IFTA-Gd. II | ACR-Gd. IB, IFTA-Gd. III | ACR-Gd. IIA | ACR-Gd. IIA, IFTA-Gd. I | ACR-Gd. III | AMR-Gd. II (Capillary/Glomerular) | IFTA-Gd. I | IFTA-Gd. II |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 | 0 | 0 | 0 | 0 | 1 | 0 | 2 | 0 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 1 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 5 | 0 | 0 | 1 | 0 | 2 | 1 | 4 | 1 | 0 | 0 | 1 | 0 | 1 | 2 | 2 | 2 | 0 | 5 | 2 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 3 | 1 | 1 | 0 | 1 | 1 | 2 | 0 | 0 | 1 | 1 | 0 | 0 | 0 | 0 | 0 | 0 | 1 | 4 | 1 |
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Type of Treatment for Detected Graft Rejection
"Upon having a biopsy performed, persons often receive treatment for rejection based on the results of the biopsy, which may or may not have shown signs of rejection. Details of treatment are presented here for rejection. Acronyms and abbreviations are defined below.~ATG=Thymoglobulin" (NCT01856257)
Timeframe: Transplantation through Week 52
Intervention | Biopsy (Number) |
---|
| ATG | ATG, Pulse Steroids | ATG, Pulse Steroids, Prograf | Antibiotic | Plasmapheresis | Plasmapheresis, Oral Steroids | Pulse Steroids | Pulse Steroids, Leflunomide | Pulse Steroids, Plasmapheresis, Eculizumab | Prednisone |
---|
Induction:MEDROL+Simulect+Tac, Maintenance:MMF+Belatacept | 0 | 2 | 0 | 0 | 0 | 0 | 3 | 0 | 0 | 0 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Belatacept | 1 | 5 | 1 | 0 | 0 | 0 | 9 | 1 | 0 | 2 |
,Induction:MEDROL+Thymoglobulin, Maintenance:MMF+Tac | 0 | 0 | 0 | 1 | 1 | 1 | 6 | 0 | 1 | 0 |
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Disease Relapse or Progression as Measured by CT Scan or PET
(NCT00574496)
Timeframe: 3 years
Intervention | months (Median) |
---|
High-Risk or Relapsed Hodgkin Lymphoma | 34.3 |
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Overall Survival
(NCT00574496)
Timeframe: up to 8 years
Intervention | months (Median) |
---|
High-Risk or Relapsed Hodgkin Lymphoma | 70.3 |
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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
Intervention | proportion of progression-free pts (Number) |
---|
High-Risk or Relapsed Hodgkin Lymphoma | 33.3 |
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Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
Diffusing capacity of the lungs for carbon monoxide (DLCO) measures how much oxygen travels from the alveoli of the lungs to the blood stream. It is used to determine the severity of lung disease. DLCO for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. DLCO % predicted compares the patients DLCO values with the reference values. An individuals DLCO result that is at least 80% of the predicted value is considered normal. Change in DLCO % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median. (NCT01670565)
Timeframe: Baseline and Week 52
Intervention | % predicted (Median) |
---|
Mycophenolate Mofetil + Belimumab | 2.00 |
Mycophenolate Mofetil + Saline (Placebo) | 0.00 |
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Change in Forced Vital Capacity (FVC)
Forced vital capacity (FVC) is the amount of air that can be forcibly exhaled from the lungs after taking a deep breath. It is used to determine the severity of lung disease. FVC for a given individual is compared to reference or predicted values. The reference values are based on healthy individuals with normal lung function and indicates values that would be expected for someone of the same sex, age and height. FVC % predicted compares the patients FVC values with the reference values. Results are considered normal if FVC is 80 percent or more of the predicted value. Change in FVC % predicted is measured by median change (and interquartile range) from Baseline median to week 52 median. (NCT01670565)
Timeframe: Baseline and Week 52
Intervention | % predicted (Median) |
---|
Mycophenolate Mofetil + Belimumab | 5.00 |
Mycophenolate Mofetil + Saline (Placebo) | -2.00 |
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Change in Modified Rodnan Skin Score (MRSS)
Change in MRSS is measured by median change (and interquartile range) from Baseline median to week 52 median. The efficacy of the drug will be measured as the change in the Modified Rodnan Skin Score (MRSS) at 52 weeks. The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. The skin score is 0 for uninvolved skin through 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas. The minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease (NCT01670565)
Timeframe: Baseline and at 52 weeks
Intervention | units on a scale (Median) |
---|
Mycophenolate Mofetil + Belimumab | -10 |
Mycophenolate Mofetil + Saline (Placebo) | -3.0 |
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Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ DI)
"The Scleroderma Health Assessment Questionnaire (SHAQ) consist of the Health Assessment Questionnaire (HAQ) and 8 other domains addressing scleroderma related manifestations that contribute to disability. It is a quality of life measure. Each question is scored from 0 (without difficulty) to 3 (unable to do). Some domains in the SHAQ are visual analog scales that are measured first and then changed to a 0-3 scale.~The maximum from each category is added together and divided by the number of categories completed. The total scale range is 0-3. A higher score indicates worse functionality.~Change in Scleroderma Health Assessment Questionnaire Disability Index (SHAQ-DI) is measured as median change (and interquartile range) from Baseline median to week 52 median. The reported median change can range from -3 to 3. A negative median change indicates a better outcome." (NCT01670565)
Timeframe: Baseline and Week 52
Intervention | score on a scale (Median) |
---|
Mycophenolate Mofetil + Belimumab | -0.25 |
Mycophenolate Mofetil + Saline (Placebo) | 0.00 |
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Number of Adverse Events and Serious Adverse Events
The safety and tolerability of belimumab in patients with systemic sclerosis will be as assessed by comparing the rates of adverse events (AEs) and serious adverse events (SAEs) between treatment and placebo groups. (NCT01670565)
Timeframe: At 52 weeks
Intervention | number of AEs (Number) |
---|
| Total number of AEs | Total number of infectious AEs | Serious AEs |
---|
Mycophenolate Mofetil + Belimumab | 53 | 18 | 0 |
,Mycophenolate Mofetil + Saline (Placebo) | 56 | 16 | 3 |
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Effectiveness and Safety of a Particular Drug Regimen to Prevent Kidney Rejection
Number of Participants with Kidney Rejections (NCT01005706)
Timeframe: 12 months
Intervention | participants (Number) |
---|
Tacrolimus Withdrawal Arm | 4 |
Tacrolimus Minimization Arm | 1 |
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Dose-Normalized C0
"Dose normalized C0 was determined (in mg/L) from blood samples collected predose. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized C0 equals (=) C0 divided by (/) (actual dose taken/1000) For the EC-MPS group: Dose normalized C0 = C0 / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 2.962 |
EC-MPS/Prednisone | 4.658 |
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Dose-Normalized Cmax (mg/L)
"Dose-normalized Cmax in plasma was determined (in mg/L) from blood samples collected predose and postdose on Day 1. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized Cmax = Cmax / (actual dose taken/1000) For the EC-MPS group: Dose normalized Cmax = Cmax / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 18.402 |
EC-MPS/Prednisone | 29.996 |
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Dose-Normalized Cmin
"Dose-normalized Cmin was determined (in mg/L) from blood samples collected predose and postdose. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized Cmin = Cmin/ (actual dose taken/1000) For the EC-MPS group: Dose normalized Cmin = Cmin / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 1.702 |
EC-MPS/Prednisone | 2.613 |
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Dose-Normalized MPA AUC0-12
"Dose-normalized MPA AUC0-12 in plasma was determined (mg*h/L) from blood samples collected predose and postdose on Day 1. Both MMF and EC-MPS doses were normalized to a standard dose of 1 g MMF or 720 mg EC-MPS, respectively. The dose normalization was calculated as follows:~For the MMF group: Dose normalized MPA AUC = MPA AUC / (actual dose taken/1000) For the EC-MPS group: Dose normalized MPA AUC = MPA AUC / (actual dose taken/720)" (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg*h/L (Mean) |
---|
MMF/Prednisone | 61.53862 |
EC-MPS/Prednisone | 94.65765 |
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Maximum Plasma Concentration (Cmax)
The mean maximum MPA concentration in plasma was determined (in mg/L) in blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 15.385 |
EC-MPS/Prednisone | 17.827 |
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Minimum Plasma Concentration (Cmin)
The mean minimum MPA concentration in plasma was determined (in mg/L) from blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 1.385 |
EC-MPS/Prednisone | 1.620 |
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MPA Area Under the Curve From 0 to 12 Hours (AUC0-12)
The mean MPA AUC0-12 in plasma was determined (in mg multiplied by hours, per Liter [mg*h/L]) from blood samples collected predose and postdose on Day 1. (NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | mg*h/L (Mean) |
---|
MMF/Prednisone | 50.36348 |
EC-MPS/Prednisone | 57.06682 |
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Pre-dose Trough Concentration (C0)
The mean mycophenolic acid (MPA) concentration in plasma was determined (in milligrams per liter [mg/L]) from blood samples collected predose (immediately before receiving study treatment). (NCT01033864)
Timeframe: Day 1 predose
Intervention | mg/L (Mean) |
---|
MMF/Prednisone | 2.387 |
EC-MPS/Prednisone | 2.944 |
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Percentage of Participants By Time to Maximum Plasma Concentration (Tmax)
(NCT01033864)
Timeframe: Day 1 predose and postdose at 30 and 60 minutes and 2, 3, 4, 5, 6, 8 10 and 12 hours
Intervention | percentage of participants (Number) |
---|
| Tmax equals (=) 0.5333 hours (hrs) | Tmax=0.6000 hrs | Tmax=0.7333 hrs | Tmax=0.7667 hrs | Tmax=1.0667 hrs | Tmax=1.0833 hrs | Tmax=1.1167 hrs | Tmax=1.2167 hrs | Tmax=2.0167 hrs | Tmax=2.0833 hrs | Tmax=2.1000 hrs | Tmax=2.1167 hrs | Tmax=2.1667 hrs | Tmax=3.1167 hrs | Tmax=3.1833 hrs |
---|
EC-MPS/Prednisone | 0.0 | 0.0 | 0.0 | 0.0 | 9.1 | 0.0 | 0.0 | 0.0 | 9.1 | 18.2 | 9.1 | 27.3 | 9.1 | 9.1 | 9.1 |
,MMF/Prednisone | 16.7 | 16.7 | 16.7 | 8.3 | 8.3 | 16.7 | 8.3 | 8.3 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 | 0.0 |
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Regression Coefficients For Participants Receiving MMF
The estimated regression coefficients for participants who received MMF presented in milligrams per liter (mg/L). (NCT01033864)
Timeframe: Day 1 at 30 minutes and 1 and 2 hours postdose
Intervention | mg/L (Number) |
---|
| Intercept | Concentration at 30 minutes (C0.5) | Concentration at 1 hour (C1) | Concentration at 2 hours (C2) |
---|
MMF/Prednisone | 2.17192 | 0.74031 | 1.89323 | 2.85923 |
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Creatinine Clearance at 1 Month After Transplantation
Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 milliliters per minute (mL/min) and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 1
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 65.82 |
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Creatinine Clearance at Month 12 After Transplantation
Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 mL/min and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 12
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 69.69 |
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Creatinine Clearance at Month 6 After Transplantation
Creatinine clearance is an indicator of renal function. Creatinine clearance is the volume of blood plasma that is cleared of creatinine by the kidneys per unit time. Normal values for healthy, young males are in the range of 100-135 mL/min and for females, 90-125 mL/min. Creatinine clearance decreases with age. A low creatinine clearance rate indicates poor kidney function. (NCT01672957)
Timeframe: Month 6
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 73.38 |
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GFR at Month 12 After Transplantation
GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is > 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR < 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 12
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 58.03 |
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GFR at Month 6 After Transplantation
GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is > 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR < 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 6
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 56.78 |
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Glomerular Filtration Rate (GFR) at Month 1 After Transplantation
GFR is an index of kidney function. GFR describes the flow rate of filtered fluid through the kidney. A normal GFR is greater than (>) 90 mL/min, although children and older people usually have a lower GFR. Lower values indicates poor kidney function. A GFR less than (<) 15 mL/min indicates kidney failure. (NCT01672957)
Timeframe: Month 1
Intervention | mL/min (Mean) |
---|
Renal Transplant Participants | 53.54 |
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Mean Dose of Mycophenolate Mofetil
(NCT01672957)
Timeframe: Baseline, Months 1, 6, and 12
Intervention | milligrams (mg) (Mean) |
---|
| Baseline (n=121) | Month 1 (n=119) | Month 6 (n=88) | Month 12 (n=69) |
---|
Renal Transplant Participants | 2033.06 | 1718.49 | 1457.39 | 1539.86 |
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Percentage of Participants Who Received Other Immunosuppressive Agents in Combination With Mycophenolate Mofetil
Participants could have received more than one other immunosuppressive agents, at the discretion of treating physician. Percentage of participants who received 1 other immunosuppressive agent, 2 other immunosuppressive agents, and 3 other immunosuppressive agents are reported. (NCT01672957)
Timeframe: Baseline, Months 1, 6, and 12
Intervention | percentage of participants (Number) |
---|
| 1 Other agent: at baseline (n=121) | 1 Other agent: at Month 1 (n=119) | 1 Other agent: at Month 6 (n=86) | 1 Other agent: at Month 12 (n=69) | 2 Other agents: at baseline (n=121) | 2 Other agents: at Month 1 (n=119) | 2 Other agents: at Month 6 (n=86) | 2 Other agents: at Month 12 (n=69) | 3 Other agents: at baseline (n=121) | 3 Other agents: at Month 1 (n=119) | 3 Other agents: at Month 6 (n=86) | 3 Other agents: at Month 12 (n=69) |
---|
Renal Transplant Participants | 0.8 | 0.8 | 2.3 | 7.2 | 90.9 | 99.2 | 97.7 | 92.8 | 8.3 | 0 | 0 | 0 |
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Percentage of Participants With Acute Rejection
Percentage of participants who experienced acute rejection within 1 month of transplantation, Month 2 to Month 6 after transplantation, Month 7 to Month 12 after transplantation are reported. (NCT01672957)
Timeframe: Baseline to Month 1, Months 2 to 6, Months 7 to 12
Intervention | percentage of participants (Number) |
---|
| Baseline to Month 1 (n=127) | Months 2 to 6 (n=124) | Months 7 to 12 (n=111) |
---|
Renal Transplant Participants | 16.5 | 0.8 | 0.9 |
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Percentage of Participants With Graft Survival
Graft survival was defined as those participants who did not experience graft loss. Graft loss defined as physical loss (nephrectomy), functional loss (necessitating maintenance dialysis for >8 weeks), re-transplant or death during the first 12 months after transplantation. (NCT01672957)
Timeframe: Months 1, 6, and 12
Intervention | percentage of participants (Number) |
---|
| Month 1 (n=127) | Month 6 (n=123) | Month 12 (n=111) |
---|
Renal Transplant Participants | 100 | 99.2 | 98.4 |
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All-Cause Mortality
All-cause mortality is defined as death from any cause occurring after randomization. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 0 |
MMF Withdrawal | 0 |
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Cumulative Excess Systemic Steroid Dose From Time of Clinically Significant Disease Reactivation to Return to Pre-Flare Dose or End of Trial Participation
"For each participant who experienced disease reactivation, excess systemic steroid dose was summed from the time of clinically significant disease reactivation until the dose returns to pre-flare levels or the end of study participation, whichever occurred first. Excess systemic steroid dose was defined as the total dose given for the flare minus (-) a participant's pre-flare steroid dose. Participants who do not have an increase in their steroid use due to the flare had their excess dose set to zero. Steroids include medications that code to a medication class which includes the terms glucocorticoid or corticosteroid. Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM)." (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Steroid dose (mg) (Mean) |
---|
MMF Maintenance | 812.8 |
MMF Withdrawal | 1750.7 |
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Cumulative Systemic Steroid Dose by Week 60
"Steroids include medications that code to a medication class which includes the terms glucocorticoid or corticosteroid. Systemic steroids will include any of these steroids that are taken by mouth (PO), intravenous (IV), or intramuscular (IM). Total cumulative systemic steroid dose, in milligrams, was summarized over the 60 week study period, or until early study termination, for each participant." (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | steroid dose (mg) (Mean) |
---|
MMF Maintenance | 851 |
MMF Withdrawal | 912 |
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Number of Malignancies Reported as Adverse Events (AEs).
The number of malignancies reported as AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Adverse Events (Number) |
---|
MMF Maintenance | 2 |
MMF Withdrawal | 3 |
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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 20 |
MMF Withdrawal | 25 |
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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 9 |
MMF Withdrawal | 12 |
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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF<2000 mg/Day Subgroup
"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 11 |
MMF Withdrawal | 13 |
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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 15 |
MMF Withdrawal | 19 |
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Number of Participants Experiencing a Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 5 |
MMF Withdrawal | 6 |
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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 4 |
MMF Withdrawal | 8 |
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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Lupus Nephritis Subgroup
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 3 |
MMF Withdrawal | 7 |
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Number of Participants Experiencing a Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 Within the Non-Lupus Nephritis Subgroup
The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 1 |
MMF Withdrawal | 1 |
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Number of Participants Experiencing Any British Isles Lupus Assessment Group (BILAG) A Flare by Week 60
The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 1 |
MMF Withdrawal | 4 |
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Number of Participants Experiencing Clinically Significant Disease Reactivation by Week 60
Disease reactivation requires:1) SELENA-SLEDAI mild/moderate or severe flare,and 2) Increased immunosuppressive therapy on a sustained basis,defined by one of the following criteria:a) Sustained activity:Significant prolonged SLE flare requiring steroid increase/burst to ≥15 mg/day prednisone (or equivalent) for >4 weeks.b) Frequent relapsing/remitting:Participant flares requiring an increase/burst of steroids and is successfully tapered to <15 mg/day within 4 weeks, but this occurs on >2 occasions, or IA, IM or IV steroids on more than1 occasion.c)Clinical activity of sufficient severity to warrant resumption of/increased dose of MMF or addition of other major immunosuppressive including AZA or MTX.Regardless of steroid use, if the investigator observes disease activity of sufficient severity to warrant resumption, addition or increase in dosage of major immunosuppressant in the setting of a SELENA-SLEDAI flare, participant has met the primary endpoint.Risk difference also included (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 5 |
MMF Withdrawal | 9 |
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Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF <2000 mg/Day Subgroup
"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 3 |
MMF Withdrawal | 6 |
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Number of Participants Experiencing Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare by Week 60 in the Baseline MMF ≥ 2000 mg/Day Subgroup
"The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included.~MMF: mycophenolate mofetil" (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 1 |
MMF Withdrawal | 2 |
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Number of Participants in the Lupus Nephritis Subgroup Experiencing a British Isles Lupus Assessment Group (BILAG) A Renal Flare by Week 60
The BILAG assesses participants on a variety of disease activity criteria in nine body system categories (constitutional, mucocutaneous, neuropsychiatric, musculoskeletal, cardiorespiratory, gastrointestinal, ophthalmic, renal, and hematological). Each category is scored as an A, B, C, or D/E, where A indicates most severe disease activity and D/E indicates inactive/no disease activity. An estimate of the risk difference ((i.e. risk(MMF Withdrawal) - risk(MMF Maintenance) and its corresponding 95% confidence interval (CI) were also included. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 0 |
MMF Withdrawal | 2 |
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Number of Serious Adverse Events (SAEs).
The number of SAEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Serious Adverse Events (Number) |
---|
MMF Maintenance | 12 |
MMF Withdrawal | 6 |
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The Addition of Aggressive Adjunctive Therapy to Mycophenolate Mofetil (MMF) or Change in MMF Therapy to Cytotoxic Drug Due to Flare by Week 60
The addition of aggressive adjunctive therapy could include intravenous (IV) immunoglobulin or rituximab at any point during the participant's study participation. A change in therapy to cytotoxic drug due to flare could include drugs such as cyclophosphamide, etc. A blinded list of study medications was reviewed to identify the addition of aggressive adjunctive therapy or cytotoxic drugs. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Participants (Count of Participants) |
---|
MMF Maintenance | 0 |
MMF Withdrawal | 1 |
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Time From Clinically Significant Disease Reactivation to Improvement in British Isles Lupus Assessment Group (BILAG) From Maximum Level During Flare
For each participant who experienced disease reactivation, time from clinically significant disease reactivation to improvement in BILAG from maximum level (at least an A or B) during the flare was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG improvement. If multiple body systems had a BILAG flare at the visit, then the body system with the most severe score was tracked for improvement; if multiple body systems had the same score (at least an A or B), then just one needed to show improvement. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Days (Mean) |
---|
MMF Maintenance | 114.5 |
MMF Withdrawal | 40.5 |
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Time From Clinically Significant Disease Reactivation to Recovery to Baseline British Isles Lupus Assessment Group (BILAG) Score or BILAG C
For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to baseline BILAG scores or BILAG C, whichever is worse, was calculated in study days as: date of clinically significant disease reactivation minus (-) date of BILAG recovery. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Days (Mean) |
---|
MMF Maintenance | 114.5 |
MMF Withdrawal | 77.7 |
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Time From Clinically Significant Disease Reactivation to Return to Pre-Flare Steroid Dose
For each participant who experienced disease reactivation, time from clinically significant disease reactivation to recovery to pre-flare steroid dose was calculated in study days as: date of clinically significant disease reactivation minus (-) date of return to pre-flare steroid dose. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Days (Mean) |
---|
MMF Maintenance | NA |
MMF Withdrawal | 37 |
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Time to Clinically Significant Disease Reactivation
The time to clinically significant disease reactivation was defined as the time from Baseline/Day 0 to the date of the first Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) assessment that met (or went on to meet) the criteria for clinically significant disease reactivation. Time to clinically significant disease reactivation was defined in study weeks as: date of SELENA-SLEDAI assessment that met reactivation criteria minus (-) baseline date. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Weeks (Mean) |
---|
MMF Maintenance | 38.0 |
MMF Withdrawal | 38.5 |
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Time to First Mild/Moderate or Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
The time to first mild/moderate or severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first mild/moderate or severe SELENA-SLEDAI flare. Time to SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Weeks (Mean) |
---|
MMF Maintenance | 20.5 |
MMF Withdrawal | 27.5 |
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Time to First Severe Safety of Estrogens in Lupus Erythematosus National Assessment-Systemic Lupus Erythematosus-Disease Activity Index (SELENA-SLEDAI) Flare
The time to first severe SELENA-SLEDAI flare was defined as the time from Baseline/Day 0 to the date of the first severe SELENA-SLEDAI flare. Time to severe SELENA-SLEDAI flare was defined in study weeks as: date of SELENA-SLEDAI flare minus (-) baseline date. The SELENA-SLEDAI assesses systemic lupus erythematosus (SLE) disease activity and categorizes mild/moderate or severe flares based on changes in the SLEDAI score, the Physician's Global Assessment (PGA), medication use (prednisone, Nonsteroidal anti-inflammatory drugs, Plaquenil, major immunosuppressives), other disease activity criteria, and hospitalization due to SLE. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Weeks (Mean) |
---|
MMF Maintenance | 43.5 |
MMF Withdrawal | 41.5 |
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Change From Baseline in the Functional Assessment of Chronic Illness Therapy (FACIT-F) Fatigue Scale (FS): Total Score
FACIT-Fatigue scale (FS) is a 13-item questionnaire completed by the patient (participant), that provides a measure of fatigue/quality of life, with a 7-day recall period. The participant scored each item on a 5-point scale: 0 (Not at all) to 4 (Very much). The larger the participant's response to the questions (with the exception of 2 negatively stated), the greater the fatigue. For all questions, except for the 2 negatively stated ones, the code was reversed and a new score was calculated as 4 minus the participant's response. The sum of all responses resulted in the FACIT-FS score for a total possible score of 0 (worse score) to 52 (better score). A higher score reflected an improvement in the participant's health status. A decrease in the FACIT-FS score reflects worse fatigue/quality of life. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Intervention | units on a scale (Mean) |
---|
| Week 24 | Week 48 | Week 60 |
---|
MMF Maintenance | -2.41 | -3.39 | -3.13 |
,MMF Withdrawal | -0.81 | -0.85 | 0.42 |
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Change From Baseline in the Lupus Quality of Life (QoL)Score
The Lupus QoL assessment is a 34 item questionnaire across 8 domains that is designed to find out how systemic lupus erythematosus (SLE) affects a participant's life over the preceding 4 weeks. Scores range from 0 (worst QoL) to 100 (best QoL). Domains include physical health, pain, planning, intimate relationships, burden to others, emotional health, body image, and fatigue. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Intervention | Scores on a Scale (Mean) |
---|
| Change in Physical Health at Week 24 | Change in Physical Health at Week 48 | Change in Physical Health at Week 60 | Change in Pain at Week 24 | Change in Pain at Week 48 | Change in Pain at Week 60 | Change in Planning at Week 24 | Change in Planning at Week 48 | Change in Planning at Week 60 | Change in Intimate Relationships at Week 24 | Change in Intimate Relationships at Week 48 | Change in Intimate Relationships at Week 60 | Change in Burden to Others at Week 24 | Change in Burden to Others at Week 48 | Change in Burden to Others at Week 60 | Change in Emotional Health at Week 24 | Change in Emotional Health at Week 48 | Change in Emotional Health at Week 60 | Change in Body Image at Week 24 | Change in Body Image at Week 48 | Change in Body Image at Week 60 | Change in Fatigue at Week 24 | Change in Fatigue at Week 48 | Change in Fatigue at Week 60 |
---|
MMF Maintenance | -1.11 | -2.83 | 0.00 | -0.17 | -3.10 | -0.19 | 0.35 | -5.43 | -3.03 | -1.47 | -0.81 | -2.08 | 1.56 | -0.58 | -1.33 | -0.87 | -2.03 | -1.52 | 2.94 | -0.17 | 3.22 | 3.78 | -2.18 | -0.57 |
,MMF Withdrawal | 1.04 | 0.46 | 1.56 | 1.77 | 1.60 | 1.42 | 3.90 | -0.18 | 0.18 | 4.29 | -1.10 | 2.86 | 0.18 | -4.43 | -3.55 | 0.09 | -1.22 | -0.87 | -1.09 | -0.56 | -4.45 | -1.17 | -1.56 | -1.95 |
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Change From Baseline in the Systemic Lupus International Collaborating Clinics/American College of Rheumatology Disease Damage Index for Systemic Lupus Erythematosus (SLICC/DI): Total Score
The SLICC/DI measures accumulated damage that has occurred since the onset of systemic lupus erythematosus (SLE), regardless of cause, in 12 organ systems. SLE damage is defined as an irreversible change in an organ or system that has been present for at least 6 months. The SLICC/DI includes 39 areas of damage in 12 domains, where each item is rated as present or absent; if evidence of damage is present for a particular item, it is given a score of 1. Some items are scored with 2 or 3 points in the case of recurring events or end stage renal disease. The SLICC/DI total score will be computed as the sum of all scores for items indicated as present; scores can range from 0 to 45. Higher scores indicate more damage. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 24, Week 48, and Week 60
Intervention | Scores on a Scale (Mean) |
---|
| Week 24 | Week 48 | Week 60 |
---|
MMF Maintenance | 0.0 | 0.0 | 0.0 |
,MMF Withdrawal | 0.0 | 0.0 | 0.0 |
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Number of Grade 3, 4, or 5 Adverse Events (AEs)
The number of Grade 3, 4, or 5 AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Adverse Events (Number) |
---|
| Grade 3 | Grade 4 | Grade 5 |
---|
MMF Maintenance | 18 | 2 | 0 |
,MMF Withdrawal | 15 | 0 | 0 |
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Number of Grade 3, 4, or 5 Hematological Adverse Events (AEs).
The number of Grade 3, 4, or 5 hematological AEs. The severity of AEs was classified using the National Cancer Institute's Common Toxicity Criteria for Adverse Events (NCI-CTCAE), Version 4.0. (NCT01946880)
Timeframe: Baseline (Treatment Randomization) to Week 60
Intervention | Adverse Events (Number) |
---|
| Grade 3 | Grade 4 | Grade 5 |
---|
MMF Maintenance | 3 | 0 | 0 |
,MMF Withdrawal | 0 | 0 | 0 |
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Percentage of Participants With Chronic GVHD
Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 39 |
Tacrolimus/Methotrexate/Maraviroc | 43 |
Tacrolimus/MMF/Cyclophosphamide | 28 |
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Percentage of Participants With Chronic GVHD Requiring Immunosupressive Therapy
Chronic GVHD is classified per 2005 NIH Consensus Criteria (Filipovich et al. 2005) into categories of severity: none, mild, moderate, and severe. Occurrence of chronic GVHD is defined as the occurrence of mild, moderate, or severe chronic GVHD per this classification. This endpoint considers the occurrence of chronic GVHD that necessitated initiation of immunosuppressive therapy for treatment. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 29 |
Tacrolimus/Methotrexate/Maraviroc | 33 |
Tacrolimus/MMF/Cyclophosphamide | 22 |
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Percentage of Participants With Disease Relapse or Progression
Relapse is defined by either morphological or cytogenetic evidence of acute leukemia or MDS consistent with pretransplant features, or radiologic evidence of lymphoma. Progression of disease applies to patients with lymphoproliferative diseases (lymphoma or chronic lymphocytic leukemia) not in remission prior to transplantation and is defined as increase in size of prior sites of disease or evidence of new sites of disease. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 24 |
Tacrolimus/Methotrexate/Maraviroc | 31 |
Tacrolimus/MMF/Cyclophosphamide | 28 |
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Percentage of Participants With Disease-free Survival
Disease-free survival is defined as being alive and free of disease relapse or progression. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 58 |
Tacrolimus/Methotrexate/Maraviroc | 56 |
Tacrolimus/MMF/Cyclophosphamide | 60 |
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Percentage of Participants With Grade II-IV Acute GVHD
"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL~2-3 mg/dL~3.01-6 mg/dL~6.01-15.0 mg/dL~>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT02208037)
Timeframe: Day 180 Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 26 |
Tacrolimus/Methotrexate/Maraviroc | 32 |
Tacrolimus/MMF/Cyclophosphamide | 27 |
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Percentage of Participants With Grade III-IV Acute GVHD
"Acute GVHD is graded according to the scoring system proposed by Przepiorka et al.1995:~Skin stage:~0: No rash~Rash <25% of body surface area~Rash on 25-50% of body surface area~Rash on > 50% of body surface area~Generalized erythroderma with bullous formation~Liver stage (based on bilirubin level)*:~0: <2 mg/dL 1.2-3 mg/dL 2.3.01-6 mg/dL 3.6.01-15.0 mg/dL 4.>15 mg/dL~GI stage*:~0: No diarrhea or diarrhea <500 mL/day~Diarrhea 500-999 mL/day or persistent nausea with histologic evidence of GVHD~Diarrhea 1000-1499 mL/day~Diarrhea >1500 mL/day~Severe abdominal pain with or without ileus * If multiple etiologies are listed for liver or GI, the organ system is downstaged by 1.~GVHD grade:~0: All organ stages 0 or GVHD not listed as an etiology I: Skin stage 1-2 and liver and GI stage 0 II: Skin stage 3 or liver or GI stage 1 III: Liver stage 2-3 or GI stage 2-4 IV: Skin or liver stage 4" (NCT02208037)
Timeframe: Day 180 Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 8 |
Tacrolimus/Methotrexate/Maraviroc | 9 |
Tacrolimus/MMF/Cyclophosphamide | 2 |
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Percentage of Participants With GVHD-free Survival
GVHD-free survival is defined as being alive without previous onset of Grade III-IV acute GVHD or chronic GVHD requiring immunosuppressive therapy. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 43 |
Tacrolimus/Methotrexate/Maraviroc | 34 |
Tacrolimus/MMF/Cyclophosphamide | 53 |
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Percentage of Participants With GVHD/Relapse or Progression-free Survival (GRFS)
GRFS is defined as being free of grade III-IV acute GVHD onset, chronic GVHD onset requiring systemic immunosuppressive therapy, disease relapse or progression, and death from any cause. (NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 35.5 |
Tacrolimus/Methotrexate/Maraviroc | 27.2 |
Tacrolimus/MMF/Cyclophosphamide | 44.1 |
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Percentage of Participants With Overall Survival
(NCT02208037)
Timeframe: 1 Year Post-transplant
Intervention | percentage of participants (Number) |
---|
Tacrolimus/Methotrexate/Bortezomib | 68 |
Tacrolimus/Methotrexate/Maraviroc | 66 |
Tacrolimus/MMF/Cyclophosphamide | 71 |
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Percentage of Participants With Neutrophil Recovery
Neutrophil recovery is defined as achieving an absolute neutrophil count (ANC) ≥ 500/mm^3 for three consecutive measurements on three different days. (NCT02208037)
Timeframe: Days 28 and 100 Post-transplant
Intervention | percentage of participants (Number) |
---|
| Day 28 | Day 100 |
---|
Tacrolimus/Methotrexate/Bortezomib | 94 | 96 |
,Tacrolimus/Methotrexate/Maraviroc | 93 | 95 |
,Tacrolimus/MMF/Cyclophosphamide | 95 | 98 |
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Percentage of Participants With Platelet Recovery
Platelet recovery is defined as the first day of a sustained platelet count >20,000/mm^3 with no platelet transfusion in the preceding seven days. (NCT02208037)
Timeframe: Days 60 and 100 Post-transplant
Intervention | percentage of participants (Number) |
---|
| Day 60 | Day 100 |
---|
Tacrolimus/Methotrexate/Bortezomib | 91 | 91 |
,Tacrolimus/Methotrexate/Maraviroc | 92 | 92 |
,Tacrolimus/MMF/Cyclophosphamide | 90 | 96 |
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Donor Cell Engraftment
Donor cell engraftment will be assessed with donor/recipient chimerism. Chimerism may be evaluated in bone marrow, whole blood, or CD3 fractions. Full donor chimerism is defined as the presence of ≥ 95% of donor cells as a proportion of total cells. Mixed chimerism is defined as the presence of donor cells, as a proportion of total cells, of < 95% but > 5% in the bone marrow or peripheral blood. Full and mixed chimerism will be evidence of donor cell engraftment. Donor cells of ≤ 5% will be considered as graft rejection. (NCT02208037)
Timeframe: Days 28 and 100 Post-transplant
Intervention | Participants (Count of Participants) |
---|
| Day 2872130179 | Day 2872130181 | Day 2872130180 | Day 10072130180 | Day 10072130181 | Day 10072130179 |
---|
| Full Chimerism | Mixed Chimerism | Graft Rejection | Dead | No Assay Performed |
---|
Tacrolimus/Methotrexate/Bortezomib | 58 |
Tacrolimus/MMF/Cyclophosphamide | 64 |
Tacrolimus/Methotrexate/Bortezomib | 11 |
Tacrolimus/MMF/Cyclophosphamide | 8 |
Tacrolimus/Methotrexate/Bortezomib | 1 |
Tacrolimus/Methotrexate/Maraviroc | 1 |
Tacrolimus/MMF/Cyclophosphamide | 2 |
Tacrolimus/Methotrexate/Bortezomib | 2 |
Tacrolimus/Methotrexate/Maraviroc | 4 |
Tacrolimus/MMF/Cyclophosphamide | 0 |
Tacrolimus/Methotrexate/Bortezomib | 17 |
Tacrolimus/Methotrexate/Maraviroc | 21 |
Tacrolimus/MMF/Cyclophosphamide | 18 |
Tacrolimus/Methotrexate/Bortezomib | 63 |
Tacrolimus/Methotrexate/Maraviroc | 56 |
Tacrolimus/MMF/Cyclophosphamide | 62 |
Tacrolimus/Methotrexate/Bortezomib | 14 |
Tacrolimus/Methotrexate/Maraviroc | 17 |
Tacrolimus/MMF/Cyclophosphamide | 13 |
Tacrolimus/Methotrexate/Bortezomib | 5 |
Tacrolimus/Methotrexate/Maraviroc | 3 |
Tacrolimus/MMF/Cyclophosphamide | 3 |
Tacrolimus/Methotrexate/Bortezomib | 4 |
Tacrolimus/Methotrexate/Maraviroc | 10 |
Tacrolimus/MMF/Cyclophosphamide | 5 |
Tacrolimus/Methotrexate/Bortezomib | 3 |
Tacrolimus/Methotrexate/Maraviroc | 6 |
Tacrolimus/MMF/Cyclophosphamide | 9 |
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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
Intervention | percentage of participants (Number) |
---|
Treatment (Reduced Intensity Allogeneic PBSCT) | 45 |
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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
Intervention | percentage of participants (Number) |
---|
Treatment (Reduced Intensity Allogeneic PBSCT) | 85 |
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Number of Days for Absolute Neutrophil Count to Recover
Average number of day per patient for absolute neutrophil count to recover(> 500/mm3 for 3 consecutive days). (NCT00594308)
Timeframe: From Day -1 (day before stem cell infusion) to Day+20 (20 days after stem cell infusion)
Intervention | days per patient (Mean) |
---|
Basiliximab 20 mg | 14.00 |
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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
Intervention | participants (Number) |
---|
Basiliximab 20 mg | 10 |
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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
Intervention | participants (Number) |
---|
Basiliximab 20 mg | 10 |
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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)
Intervention | days per patient (Mean) |
---|
Basiliximab 20 mg | 15.33 |
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Percentage of Participants Alive at 1 Year After Transplant
One-year survival rate after transplant (NCT00763490)
Timeframe: 1 year
Intervention | percentage of participants (Number) |
---|
Double Cord Blood Tranplant | 40 |
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Percentage of Patients Alive at the End of the Trial
Event Free Survival (EFS) was determined. Patients were followed up to 5 years (median time of 2.35 years). (NCT00763490)
Timeframe: 5 Years
Intervention | percentage of patients (Number) |
---|
Double Cord Blood Tranplant | 35 |
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Cumulative Incidence of Neutrophil and Platelet Engraftment
The failure to achieve a neutrophil count > 500/uL or a platelet count >30.0 x 10e9 /L within 35 days of the stem cell infusion will be defined as primary engraftment failure. (NCT00763490)
Timeframe: Day 35
Intervention | percentage of participants (Number) |
---|
| Cumulative incidence of platlet engraftment | Cumulative incidence of neutrophil engraftment |
---|
Double Cord Blood Tranplant | 73 | 89 |
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Incidence of Acute (Grade II-IV) and Chronic Graft-vs-host Disease(GVHD)
"The percentage of patients with acute GVHD (Grade II-IV) was determined at 100 days. Patients were followed up to 5 years and the percentage of patients that developed chronic GVHD at the end of the study was tabulated.~Acute GVHD is staged and graded (grade 0-IV, where grade 0 is no involvement and involvement increases by grade) by the number and extent of organ involvement. Patients can have involvement of three organs: skin (rash/dermatitis), liver (hepatitis/jaundice), and gastrointestinal tract (abdominal pain/diarrhea)." (NCT00763490)
Timeframe: Up to 5 years
Intervention | percentage of patients (Number) |
---|
| Acute GVHD (Grades II-IV) | Chronic GVHD |
---|
Double Cord Blood Tranplant | 40 | 35 |
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Number of Non-Relapse Mortalities
Number of patients expired without disease progression/relapse. (NCT01251575)
Timeframe: 100 days post-transplant
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Class I Mismatch | 15 |
Class II Mismatch | 12 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment (Fludarabine, Transplant, Immunosuppression) | 2 |
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Number of Participants With Event-free Survival (EFS)
"The Kaplan-Meier estimate of EFS with relapse, death due to any cause and graft failure as events along with their standard errors will be calculated using the SAS macro (bmacro251-Excel2007kme) available in the Department of Biostatistics at St. Jude, where EFS = min (date of last follow-up, date of relapse, date of graft failure, date of death due to any cause) - date of transplant, and all participants surviving at the time of analysis without events will be censored. The number of participants who did not experience any of these events through one year post-transplant is given.~Due to the early close of the study, a small number of patients were enrolled. Subsequently, the number of patients who did not experience any events defined above is provided." (NCT02199041)
Timeframe: One year after transplantation
Intervention | Participants (Count of Participants) |
---|
Treatment | 4 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment | 7 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment | 11 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment | 6 |
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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
Intervention | Participants (Count of Participants) |
---|
Treatment | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
| No Acute GVHD | Grade I | Grade II | Grade III | Grade IV |
---|
Treatment | 8 | 0 | 1 | 2 | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
| No Chronic GVHD | Mild | Moderate | Severe |
---|
Treatment | 11 | 0 | 0 | 1 |
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Disease-Free Survival (DFS)
1-year post-transplant disease free survival (DFS), defined as success if a patient is alive and disease free at 1-year post-transplant. (NCT01350245)
Timeframe: 1 year post-transplant
Intervention | percentage of patients (Number) |
---|
TJU 2 Step Regimen | 78.6 |
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Probability of Overall Survival at 15 Months Post-treatment
Probability of overall survival at 15 months post-treatment, defined as success if a patient is alive 1-year post-transplant. (NCT01350245)
Timeframe: 15 months
Intervention | percentage of probability (Number) |
---|
TJU 2 Step Regimen | 85 |
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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
Intervention | Participants (Count of Participants) |
---|
Yttrium-90 Ibritumomab + Chemo | 14 |
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Change From Baseline in Serum Creatinine - ITT
Blood samples were collected to assess serum creatinine measurements. A negative change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 3 months
Intervention | mg/dL (Mean) |
---|
Immediate Everolimus (IE) | -4.79 |
Delayed Everolimus (DE) | -5.13 |
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Change From Baseline in Serum Creatinine - Modified ITT
Blood samples were collected to assess serum creatinine measurements. A negative change from baseline indicates improvement. (NCT01410448)
Timeframe: baseline, 12 months
Intervention | mg/dL (Mean) |
---|
Immediate Everolimus (IE) | -4.96 |
Delayed Everolimus (DE) | -5.22 |
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Duration of DGF
The duration of DGF was defined as the elapsed time from first to last day of post-transplant dialysis. (NCT01410448)
Timeframe: 3 months
Intervention | Days (Median) |
---|
Immediate Everolimus (IE) | 8.50 |
Delayed Everolimus (DE) | 5.50 |
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Participant/Graft Survival Rate: Percentage of Participants With Failure Events of Death or Graft Loss - Worst-case Scenario
The percentage of participants who experienced death or graft loss was assessed. In the worst-case scenario, failure, i.e. participants death or graft loss, was identified in one of the following cases: occurrence of at least one failure event or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 6.74 |
Delayed Everolimus (DE) | 18.42 |
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Patient Survival Rate: Percentage of Deaths - Worst-case Scenario
The percentage of deaths was assessed. In the worst-case scenario, failure, i.e. death, was identified in one of the following cases: participant's death or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 Months, 12 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 6.22 |
Delayed Everolimus (DE) | 18.42 |
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Percentage of Participants Who Experienced Treatment Failure - Worst-case Scenario
The percentage of participants who experienced treatment failure was assessed. Treatment failure was defined as the occurrence of at least one failure event among death, graft loss or biopsy-proven acute rejection (BPAR). In the worst-case scenario, treatment failure was identified in one of the following cases: occurrence of at least one treatment failure event or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 11.40 |
Delayed Everolimus (DE) | 21.05 |
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Percentage of Participants With a New Onset of Diabetes
The percentage of participants with a new onset of diabetes was assessed. (NCT01410448)
Timeframe: 12 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 3.14 |
Delayed Everolimus (DE) | 4.05 |
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Percentage of Participants With a New Onset of Malignancy
The percentage of participants with a new onset of malignancy was assessed. (NCT01410448)
Timeframe: 12 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 0 |
Delayed Everolimus (DE) | 0.68 |
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Percentage of Participants With Acute Rejection (AR)
AR was defined as an episode of increased serum creatinine >30% that was clinically diagnosed as an acute rejection but was not biopsy proven. (NCT01410448)
Timeframe: 12 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 12.44 |
Delayed Everolimus (DE) | 10.53 |
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Percentage of Participants With Delayed Graft Function (DGF) -
DGF was defined as the need for dialysis in the first week after transplant, excluding Renal Replacement Therapy within the first 24 hours after transplantation. (NCT01410448)
Timeframe: 3 Months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 23.83 |
Delayed Everolimus (DE) | 31.58 |
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Percentage of Participants Without Wound Healing Complications - Worst-case Scenario
The percentage of participants without wound healing complications was assessed. Wound healing complications consisted of lymphorrhea, fluid collections, wound dehiscence, wound infections and incisional hernia. In the worst-case scenario, failure, i.e. at least one healing complication occurrence, was identified in one of the following cases: wound complication occurrence, missing information about wound complication occurrence or study discontinuation due to any reason for participants who did not complete the 12 month follow-up visit. (NCT01410448)
Timeframe: 12 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 65.80 |
Delayed Everolimus (DE) | 59.47 |
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Percentage of Participants Without Wound Healing Complications - Worst-case Scenario
The percentage of participants without wound healing complications was assessed. Wound healing complications consisted of lymphorrhea, fluid collections, wound dehiscence, wound infections and incisional hernia. In the worst-case scenario, failure, i.e. at least one healing complication occurrence, was identified in one of the following cases: wound complication occurrence, missing information about wound complication occurrence, or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months
Intervention | Percentage of participants (Number) |
---|
Immediate Everolimus (IE) | 68.39 |
Delayed Everolimus (DE) | 61.58 |
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Graft Survival Rate: Percentage of Participants With Graft Loss - Worst-case Scenario
The percentage of participants who experienced graft loss was assessed. In the worst-case scenario, failure, i.e. graft loss, was identified in one of the following cases: occurrence of graft loss or discontinuation due to any reason. (NCT01410448)
Timeframe: 3 months, 12 months
Intervention | Percentage of participants (Number) |
---|
| 3 months | 12 months |
---|
Delayed Everolimus (DE) | 18.42 | 19.47 |
,Immediate Everolimus (IE) | 6.74 | 7.25 |
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Percentage of Participants With BPAR - Worst-case Scenario
A biopsy-proven acute rejection was defined as a biopsy graded IA, IB, IIA, IIB or III. In the worst-case scenario, failure, i.e. BPAR, was identified in one of the following cases: occurrence of BPAR or study discontinuation due to any reason. (NCT01410448)
Timeframe: 3 Months, 12 months
Intervention | Percentage of participants (Number) |
---|
| 3 monts | 12 months |
---|
Delayed Everolimus (DE) | 21.05 | 24.74 |
,Immediate Everolimus (IE) | 11.40 | 15.54 |
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Percentage of Participants With Proteinuria
Incidence of proteinuria (>1,000 mg/day in urine collected in 24 hours or > 1.0 if measured on the urine protein/creatinine concentration ratio in a spot urine sample) was assessed. (NCT01410448)
Timeframe: 3 months
Intervention | Percentage of participants (Number) |
---|
| Yes | No | Missing |
---|
Delayed Everolimus (DE) | 4.21 | 68.42 | 27.37 |
,Immediate Everolimus (IE) | 4.15 | 68.91 | 26.94 |
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Cholesterol
(NCT01624948)
Timeframe: 3 months post-randomization
Intervention | mg/dL (Mean) |
---|
Everolimus+Tacrolimus/Prednisone | 212 |
Standard of Care: 50% Reduction of MPA | 170 |
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Evaluation for the Development of BK Virus Nephropathy or Doubling of BK Viremia Levels
A doubling of BK viremia levels or the development of BKV nephropathy in subjects enrolled in the experimental study arm will prompt a conversion to standard care therapy. We will closely monitor BKV levels in the urine and blood and assess renal function monthly, as per our usual standard of care. Based on BKV results as well as renal function, as assessed by serum Cr, biopsies may be done for cause. Patients will have a final visit at month 4 to monitor for adverse events. (NCT01624948)
Timeframe: 3 months post-randomization
Intervention | participants (Number) |
---|
Everolimus+Tacrolimus/Prednisone | 2 |
Standard of Care: 50% Reduction of MPA | 2 |
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Evidence of Reduction of BK Viruria and/or Clearance of BK Viremia
composite outcome of a 50% or greater reduction in BKV urine levels and/or complete clearance of BKV viremia by 3 months after randomization (NCT01624948)
Timeframe: 3 months post-randomization
Intervention | participants (Number) |
---|
Everolimus+Tacrolimus/Prednisone | 11 |
Standard of Care: 50% Reduction of MPA | 8 |
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p70S6 Kinase Phosphorylation
(NCT01624948)
Timeframe: 3 months post-randomization
Intervention | Mean Fluorescence Intensity (Median) |
---|
Reached Primary Endpoint | 5002 |
Failed to Reach Primary Endpoint | 4353 |
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Proteinuria
(NCT01624948)
Timeframe: 3 months post-randomization
Intervention | g/g creatinine (Mean) |
---|
Everolimus+Tacrolimus/Prednisone | 0.16 |
Standard of Care: 50% Reduction of MPA | 0.23 |
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Complete Remission (CR) Rate at Day 30 Post HSCT
The CR rate at 30 days (Day +30) post stem cell transplant infusion (NCT04002115)
Timeframe: 30 days
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 100 |
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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
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 0 |
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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
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 0 |
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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
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 0 |
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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
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 0 |
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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
Intervention | percentage of Participants (Number) |
---|
Clofarabine 30 mg/m^2 | 0 |
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Number of Patients Reported Ascites
(NCT01903798)
Timeframe: Week 24
Intervention | Participants (Count of Participants) |
---|
Continue Prednisolone (Lille <0.45) | 0 |
Rilonacept + Prednisolone (Lille <0.45) | 0 |
Standard of Care (Lille ≥ 0.45) | 1 |
Mycophenolate + Prednisolone (Lille ≥ 0.45) | 0 |
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Survival at Day 29 of the Assigned Treatment
"To determine whether treatment with prednisolone + mycophenolate mofetil is better than standard of care treatment among patients with alcoholic hepatitis who fail to respond to 1 week of prednisolone (i.e., Lille score of ≥0.45). Primary outcome is survival at Day 29.~All study participants received the Standard of care (prednisolone) with or without experimental drug at Day 1 (based on randomization). Response to the treatment was determined at Day 8. Data was collected for both responders and non-responders." (NCT01903798)
Timeframe: Day 8 to Day 29
Intervention | Participants (Count of Participants) |
---|
Continue Prednisolone (Lille <0.45) | 2 |
Rilonacept + Prednisolone (Lille <0.45) | 0 |
Standard of Care (Lille ≥ 0.45) | 1 |
Mycophenolate + Prednisolone (Lille ≥ 0.45) | 1 |
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Incidence of Cytomegalovirus Infection or Disease
Incidence of CMV infection/disease in three study groups (SRL, EVR anda MPS). (NCT03468478)
Timeframe: 12 months follow up
Intervention | Participants (Count of Participants) |
---|
Sirolimus +Tacrolimus | 9 |
Everolimus +Tacrolimus | 7 |
Mycophenolate +Tacrolimus | 39 |
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BPAR (Biopsy-proven Acute Rejection) Incidence During the First 12 Months Post-transplant
BPAR (biopsy-proven acute rejection) incidence during the first 12 months post-transplant. Grading is determined using standard Banff criteria. (NCT01680861)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Tacrolimus/Everolimus | 1 |
Tacrolimus/EC-MPS | 3 |
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Discontinuance of Any Study Medication (Tacrolimus, Everolimus, or EC-MPS)
(NCT01680861)
Timeframe: during the first 12 months post-transplant
Intervention | participants (Number) |
---|
Tacrolimus/Everolimus | 0 |
Tacrolimus/EC-MPS | 1 |
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eGFR (Calculated Glomerular Filtration Rate), i.e., Renal Function, at 1 Month Post-transplant.
using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 1 month post-transplant
Intervention | ml/min per 1.73 m2 (Mean) |
---|
Tacrolimus/Everolimus | 82.1 |
Tacrolimus/EC-MPS | 62.1 |
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eGFR (Renal Function) at 6 Months Post-transplant
using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 6 months post-transplant
Intervention | ml/min per 1.73 m2 (Mean) |
---|
Tacrolimus/Everolimus | 66.7 |
Tacrolimus/EC-MPS | 63.7 |
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eGFR (Renal Function) at Month 3 Post-transplant
Renal function as determined by the estimated glomerular filtration rate (eGFR) at 3 months post-transplant, using the abbreviated MDRD formula. (NCT01680861)
Timeframe: at 3 months post-transplant
Intervention | ml/min per 1.73 m^2 (Mean) |
---|
Tacrolimus/Everolimus | 75.7 |
Tacrolimus/EC-MPS | 65.6 |
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Graft Loss (Return to Permanent Dialysis or Death)
(NCT01680861)
Timeframe: during the first 12 months post-transplant
Intervention | participants (Number) |
---|
Tacrolimus/Everolimus | 0 |
Tacrolimus/EC-MPS | 0 |
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Incidence of Chronic Allograft Nephropathy (CAI) at 12 Months Post-transplant
Incidence of (biopsy-proven) chronic allograft nephropathy (CAI) [interstitial fibrosis and tubular atrophy, using standard Banff criteria] at 12 months post-transplant. (NCT01680861)
Timeframe: 1 year
Intervention | participants (Number) |
---|
Tacrolimus/Everolimus | 3 |
Tacrolimus/EC-MPS | 3 |
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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
Intervention | Participants (Count of Participants) |
---|
Nonmyeloablative Conditioning and BMT | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
Nonmyeloablative Conditioning and BMT | 1 |
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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
Intervention | Participants (Count of Participants) |
---|
| Grades II-IV acute GVHD | Grades III-IV acute GVHD |
---|
Nonmyeloablative Conditioning and BMT | 0 | 0 |
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Graft Failure
Number of participants with primary and/or secondary graft failure. (NCT02080195)
Timeframe: 60 days
Intervention | Participants (Count of Participants) |
---|
| Primary graft failure | Secondary graft failure |
---|
Nonmyeloablative Conditioning and BMT | 0 | 0 |
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Survival
Number of patients alive and alive without relapse, respectively. (NCT02080195)
Timeframe: 1 year
Intervention | Participants (Count of Participants) |
---|
| Overall Survival | Event Free Survival |
---|
Nonmyeloablative Conditioning and BMT | 1 | 1 |
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Number of Participants With Graft Failure/Rejection
descriptive (NCT00723099)
Timeframe: By day 55
Intervention | participants (Number) |
---|
Treatment (Chemotherapy, Transplant) | 3 |
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Overall Survival
Kaplan-Meier and cumulative incidence estimates will be used. (NCT00723099)
Timeframe: At 1 year
Intervention | percent of patients (Number) |
---|
Treatment (Chemotherapy, Transplant) | 35 |
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Percent of Patients With Acute GVHD Grades III-IV
Fischer's exact test was used to determined percent of patients with acute grade III-IV GVHD by Glucksberg criteria (NCT00723099)
Timeframe: 100 days
Intervention | percent of patients (Number) |
---|
Treatment (Chemotherapy, Transplant) | 12 |
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Percent of Patients With Chronic GVHD
Kaplan-Meier and cumulative incidence estimates will be used to measure percent of patients with chronic GVHD by NIH consensus criteria. (NCT00723099)
Timeframe: At 2 years
Intervention | percent of patients (Number) |
---|
Treatment (Chemotherapy, Transplant) | 19 |
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Percent of Patients With Grade II-IV Acute Graft Versus Host Disease
Chi-square test was used to determine percent of grade II-IV GVHD using Glucksberg criteria (NCT00723099)
Timeframe: By day 100
Intervention | percent of patients (Number) |
---|
Treatment (Chemotherapy, Transplant) | 67 |
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Percent of Patients With Non-relapse Mortality
Kaplan-Meier and cumulative incidence estimates (NCT00723099)
Timeframe: 1 year
Intervention | percent 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
Intervention | percent of patients (Number) |
---|
Treatment (Chemotherapy, Transplant) | 21 |
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Time to Platelet Engraftment of > 20,000 Cells Per mm3
median and range (NCT00723099)
Timeframe: By 6 months
Intervention | days (Median) |
---|
Treatment (Chemotherapy, Transplant) | 46 |
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Engraftment of HLA Identical PBSC Allografts
"Number of patients who engrafted by Day 56 post allogeneic transplant. Failure to engraft is defined as the absence of detectable donor cells in the marrow. The rates and accompanying confidence intervals associated with failure of engraftment at day +56 will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 56
Intervention | Participants (Count of Participants) |
---|
Treatment (Tandem Transplantation) | 53 |
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Non-Relapse Mortality
"The rates and accompanying confidence intervals associated with transplant-related mortality will be calculated after every 5th patient is enrolled on the study. If the lower limit to the appropriate one-sided 80% confidence interval exceeds 25%, this will be considered sufficient evidence of an excess failure rate and the study will be stopped. For these purposes, all patients will be evaluated together (patients with chemosensitive and chemoresistant disease)." (NCT00005803)
Timeframe: Day 100 post-non-myeloablative allografting following mobilization and high-dose chemotherapy with autografting
Intervention | Participants (Count of Participants) |
---|
Treatment (Tandem Transplantation) | 3 |
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Overall Survival (OS)
Number of patients surviving by interval. Chemosensitive and chemoresistant subjects will be analyzed separately. (NCT00005803)
Timeframe: From the date of autologous transplant until the time of death, assessed up to 3 years
Intervention | Participants (Count of Participants) |
---|
| 6 Months | 1 Year | 1.5 Years | 2 Years | 3 Years |
---|
Chemoresistant Group | 14 | 10 | 9 | 8 | 8 |
,Chemosensitive Group | 39 | 31 | 27 | 26 | 23 |
,Unknown Chemosensitivity Group | 1 | 0 | 0 | 0 | 0 |
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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
Intervention | Participants (Count of Participants) |
---|
| 6 Months | 1 Year | 1.5 Years | 2 Years | 3 Years |
---|
Chemoresistant Group | 12 | 8 | 8 | 8 | 8 |
,Chemosensitive Group | 36 | 27 | 25 | 25 | 22 |
,Unknown Chemosensitivity Group | 1 | 0 | 0 | 0 | 0 |
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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
Intervention | percentage of analyzed participants (Number) |
---|
| Day 100 | Month 6 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 55 | 60 |
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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
Intervention | percentage of analyzed participants (Number) |
---|
| Year 1 | Year 2 | Year 3 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 64.6 | 66 | 67.3 |
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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
Intervention | Participants (Count of Participants) |
---|
| Year 1 | Year 2 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 27 | 33 |
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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
Intervention | percentage of analyzed participants (Number) |
---|
| Year 1 | Year 2 | Year 3 | Year 5 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 60 | 48 | 42 | 29 |
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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
Intervention | percentage of analyzed participants (Number) |
---|
| Year 1 | Year 2 | Year 3 | Year 5 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 48 | 39 | 35 | 29 |
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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
Intervention | percentage of analyzed participants (Number) |
---|
| Year 1 | Year 2 |
---|
Busulfan (Bu), Fludarabine (Flu), Total Body Iradiation (TBI) | 13 | 20 |
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Participants Experiencing Acute Rejection Episode
(NCT02123108)
Timeframe: 12 months post liver transplant
Intervention | Participants (Count of Participants) |
---|
Basiliximab | 3 |
Tacrolimus Group | 4 |
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Participants Experiencing Adverse Event Attributable to Study Drug
(NCT02123108)
Timeframe: 12 months post liver transplantation
Intervention | Participants (Count of Participants) |
---|
Basiliximab | 0 |
Tacrolimus Group | 0 |
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Participants Experiencing Graft Failure
(NCT02123108)
Timeframe: 12 months post transplant
Intervention | Participants (Count of Participants) |
---|
Basiliximab | 2 |
Tacrolimus Group | 5 |
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Renal Recovery/ Function
Number of participants who experienced dialysis independence or improvement based upon kidney function labs as a measure of renal recovery/ function following OLT in patients after undergoing orthotopic liver transplant (NCT02123108)
Timeframe: 12 months post-transplant
Intervention | Participants (Count of Participants) |
---|
Basiliximab | 28 |
Tacrolimus Group | 26 |
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Survival
Participants at risk minus incidence of death within the first year post-transplant (NCT02123108)
Timeframe: 12 months post liver transplant
Intervention | Participants (Count of Participants) |
---|
Basiliximab | 28 |
Tacrolimus Group | 26 |
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Change From Baseline (Randomization) in Renal Function
"Change in renal function was measured by change in glomerular filtration rate (GFR). GFR calculated using the abbreviated modification of diet in renal disease (aMDRD) formula.~GFR in mL/min/1.73m^2 for men of non-black ethnicity: 186 * [C/88]^-1.154 * [A]^-0.023*G*R ; C = serum creatinine (in μmol/L); A = Age (in years). G = 0.742 when the patient is a women; Otherwise G=1 R= 1.21 when the patient was of black ethnicity; Otherwise R = 1 Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24
Intervention | mL/min/1.73m^2 (Least Squares Mean) |
---|
Tacrolimus | -13.29 |
Everolimus (RAD001) | 1.05 |
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Change From Baseline (Randomization) in Serum Creatinine
"Change in serum creatinine concentrations from baseline (randomization) to week 24 post-randomization was one of the efficacy assessments of renal function.~Baseline was Day 28 visit." (NCT01625377)
Timeframe: Baseline, Week 24
Intervention | µmol/L (Mean) |
---|
Tacrolimus | 7.2 |
Everolimus (RAD001) | -1.3 |
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Change From Baseline (Randomization) in Urine Protein/Creatinine Ratio
Change in urine protein/creatinine ratio from baseline (randomization) to week 24 post-randomization was one of the efficacy assessments of renal function. Baseline was Day 28 visit. (NCT01625377)
Timeframe: Baseline, week 24
Intervention | mg/mmol (Mean) |
---|
Tacrolimus | -2.3 |
Everolimus (RAD001) | 21.9 |
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Number of Patients in Different Stages of Chronic Kidney Diseases According to the K/DOQI Classification System
"Kidney disease outcomes quality initiative (K/DOQI) classification is based on glomerular filtration rate (GFR), abbreviated MDRD formula (mL/min/1.73m^2) :~Stage 1 : GFR >= 90; Stage 2 = GFR was between 60-89; Stage 3 = GFR was between 30-59 ; Stage 4 = GFR was between 15-29; Stage 5 = GFR was < 15 (or dialysis)" (NCT01625377)
Timeframe: At Week 24
Intervention | Patients (Number) |
---|
| Stage 1 | Stage 2 | Stage 3 | Stage 4 | Stage 5 |
---|
Everolimus (RAD001) | 41 | 29 | 4 | 0 | 0 |
,Tacrolimus | 24 | 34 | 28 | 0 | 0 |
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Number of Patients Reported With Different Categories of Severity of BPAR According to Banff Classification
"Biopsy proven acute rejection was defined as a clinically suspected acute rejection confirmed by biopsy.~The severity of BPAR was categorized as :~Mild (Banff grade I, RAI = 4 and 5) Moderate (Banff grade II, RAI = 6 and 7) Severe (Banff grade III, RAI = 8 and 9) Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted." (NCT01625377)
Timeframe: at 12 week and 24 week
Intervention | Patients (Number) |
---|
| Week 12: Mild | Week 12: Moderate | Week 12: Severe | Week 24: Mild | Week 24: Moderate | Week 24: Sever |
---|
Everolimus (RAD001) | 1 | 1 | 0 | 5 | 3 | 0 |
,Tacrolimus | 1 | 1 | 0 | 1 | 1 | 0 |
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Number of Patients With Any Adverse Events, Serious Adverse Events, Death and Premature Discontinuation
Baseline was Day 28 visit. This endpoint reports patients with total adverse events (any), serious adverse events, death and premature discontinuation. (NCT01625377)
Timeframe: Baseline to 24 weeks
Intervention | Patients (Number) |
---|
| Any Adverse events | Serious Adverse events | Death | At least one AE led to premature discontinuation |
---|
Everolimus (RAD001) | 81 | 42 | 2 | 18 |
,Tacrolimus | 85 | 28 | 1 | 4 |
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Number of Patients With Death or Graft Loss
The graft was presumed to be lost on the day the patient was registered again on the waiting list, or the day he/she received a new graft. (NCT01625377)
Timeframe: at week 24
Intervention | Patients (Number) |
---|
| Graft Loss | Death |
---|
Everolimus (RAD001) | 0 | 1 |
,Tacrolimus | 1 | 1 |
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Number of Patients With Treated or Not Treated Biopsy Proven Acute Rejection (BPAR)
Biopsy proven acute rejection was defined as a clinically suspected acute rejection confirmed by biopsy. (NCT01625377)
Timeframe: at 12 week and 24 week
Intervention | Patients (Number) |
---|
| Week 12, Treated BPAR | Week 12, Not treated BPAR | Week 24, Treated BPAR | Week 24, Not Treated BPAR |
---|
Everolimus (RAD001) | 2 | 0 | 8 | 1 |
,Tacrolimus | 2 | 0 | 2 | 0 |
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Number of Patients With Treated or Untreated BPAR With RAI Score Greater Than 3
"Biopsy proven acute rejection (BPAR) was defined as a clinically suspected acute rejection confirmed by biopsy. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted.~The patients with treated or untreated BPAR having RAI score > 3 were reported in this end point." (NCT01625377)
Timeframe: At 24 weeks
Intervention | Patients (Number) |
---|
| Not Treated BPAR: RAI score >3 | Not Treated BPAR: Missing RAI score | Treated BPAR: RAI score >3 |
---|
Everolimus (RAD001) | 0 | 1 | 8 |
,Tacrolimus | 0 | 0 | 2 |
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Number of Patients With Treatment Failures
"Incidence of treatment failures, assessed with composite criterion including treated biopsy proven acute rejection (tBPAR) with a rejection activity index (RAI) according to Banff classification >3, graft loss or death at 6 months.~Biopsy proven acute rejection (BPAR) was defined as a clinically suspected acute rejection confirmed by biopsy. The Banff Rejection Activity Index (RAI) comprises 3 components scored from 0 to 3: venous endothelial inflammation; bile duct inflammation damage; and portal inflammation; the scores are combined to an overall score (the RAI) ranging from 0 to 9. An overall score of 0-3 is considered indeterminate, score of 4-5 is mild acute, score of 6-7 is moderate acute , and score of 8-9 is severe acute. Only the episode with the highest total RAI score for each participant was counted.~The graft was presumed to be lost on the day the patient was registered again on the waiting list, or the day he/she received a new graft." (NCT01625377)
Timeframe: At week 12 and week 24
Intervention | Patients (Number) |
---|
| week 12, Treatment failures - NO | week 12, Treatment failures - YES | week 24, Treatment failures - NO | week 24, Treatment failures - YES |
---|
Everolimus (RAD001) | 88 | 2 | 81 | 9 |
,Tacrolimus | 91 | 2 | 89 | 4 |
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Number of Participants Achieving Treatment Success After Switching to Other Medication (Phase II, 0-6 Months)
Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate for patients who crossed over to other medication following treatment failure at 6 months (or earlier). (NCT01829295)
Timeframe: 6 Months
Intervention | Participants (Count of Participants) |
---|
Switched Over to Methotrexate | 20 |
Switched Over to Mycophenolate Mofetil | 7 |
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Number of Participants Achieving Treatment Success at 12 Months on Same Medication (Phase I, 6-12 Months)
Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate in patients who were a treatment success at the primary outcome of 6 months. (NCT01829295)
Timeframe: 12 Months
Intervention | Participants (Count of Participants) |
---|
Methotrexate | 48 |
Mycophenolate Mofetil | 40 |
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Number of Participants Achieving Treatment Success at 6 Months (Phase I, 0-6 Months)
Controlled ocular inflammation (≤ 0.5+ anterior chamber cells, ≤ 0.5+ vitreous haze, no active retinal/choroidal lesions in both eyes) with 7.5 mg/day of oral prednisone and ≤ 2 drops/day of topical 1% prednisolone acetate. (NCT01829295)
Timeframe: 6 Months
Intervention | Participants (Count of Participants) |
---|
Methotrexate | 64 |
Mycophenolate Mofetil | 56 |
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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
Intervention | percent probability (Number) |
---|
Arm I (Busulfan, Cyclophosphamide, Melphalan) | 17 |
Arm II (Busulfan, Fludarabine Phosphate) | 55 |
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Percent Probability of Event-free Survival (EFS)
Probability of Event-free Survival (EFS) for Patients after 18 months. An event is either treatment related mortality (TRM), primary or secondary graft failure, or relapse/non-response (as defined in protocol section 10). Time to event is time from transplant with patients who die between the start of the conditioning regimen and transplant given a time to event of zero. (NCT01824693)
Timeframe: From transplant up to 18 months
Intervention | percent probability (Number) |
---|
Arm I (Busulfan, Cyclophosphamide, Melphalan) | 83 |
Arm II (Busulfan, Fludarabine Phosphate) | 22 |
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Percentage of Participants Who Experience Primary Graft Failure Event Between Arms
Primary Graft failure is defined as the failure to achieve an ANC >= 500/uL after 42 days, determined by 3 consecutive measurements on different days; OR < 5% donor cells in blood or bone marrow by day +42 (as demonstrated by a chimerism assay), without evidence of Juvenile Myelomonocytic Leukemia (JMML). (NCT01824693)
Timeframe: Day 0 - day 540 (18 months) following completion of stem cell transplant
Intervention | percentage of patients (Number) |
---|
Arm I (Busulfan, Cyclophosphamide, Melphalan) | 0 |
Arm II (Busulfan, Fludarabine Phosphate) | 0 |
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Graft Loss - Percentage of Participants With an Event
Graft loss was defined as physical loss (nephrectomy), functional loss [necessitating maintenance dialysis for greater than (>)8 weeks], retransplant or death. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | percentage of participants (Number) |
---|
Mycophenolate Mofetil, Adapted Dose | 5.6 |
Mycophenolate Mofetil, Fixed Dose | 5.0 |
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Participant Survival
Participants survival was defined as the percentage of participants living with or without a functioning graft between Weeks 0 and 52. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | percentage of participants (Number) |
---|
Mycophenolate Mofetil, Adapted Dose | 98.4 |
Mycophenolate Mofetil, Fixed Dose | 98.3 |
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Percentage of Participants With Biopsy-Proven Acute Rejection (BPAR) Before Week 12 or Acute Subclinical Rejection on Protocol Biopsy at Week 12
BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10 percent (%) compared to baseline (BL) values and BPAR of Grade greater than or equal to (≥) 1 according to Banff 1997 classification at Week 12. (NCT02005562)
Timeframe: Week 12
Intervention | percentage of participants (Number) |
---|
Mycophenolate Mofetil, Adapted Dose | 24.2 |
Mycophenolate Mofetil, Fixed Dose | 19.8 |
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Time to Graft Loss
The median time, in days, from randomization to graft loss event. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | days (Median) |
---|
Mycophenolate Mofetil, Adapted Dose | NA |
Mycophenolate Mofetil, Fixed Dose | NA |
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Time to Occurrence of First BPAR Between Day 0 and Week 52
BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection at Week 12 was included in the analysis. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10% compared to BL values and BPAR of Grade ≥1 according to Banff 1997 classification at Week 12. The occurrence of the first BPAR was defined as the time from randomization to the first recorded BPAR between Day 0 and Week 52. The results of protocol biopsies at Week 12 were taken into account. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | days (Median) |
---|
Mycophenolate Mofetil, Adapted Dose | NA |
Mycophenolate Mofetil, Fixed Dose | NA |
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Time to Occurrence of First BPAR Between Day 0 and Week 52 - Percentage of Participants With an Event
BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Subclinical acute rejection at Week 12 was included in the analysis. Subclinical acute rejection was defined as an increase of serum creatinine at Week 12 strictly less than 10% compared to BL values and BPAR of Grade ≥1 according to Banff 1997 classification at Week 12. The occurrence of the first BPAR was defined as the time from randomization to the first recorded BPAR between Day 0 and Week 52. The results of protocol biopsies at Week 12 were taken into account. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Day 0, Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | percentage of participants (Number) |
---|
Mycophenolate Mofetil, Adapted Dose | 24.6 |
Mycophenolate Mofetil, Fixed Dose | 14.9 |
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Creatinine Clearance Values Estimated With the Cockcroft-Gault Equation (Milliliters Per Minute [mL/Min])
The mean creatinine clearance values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52 estimated using the Cockcroft-Gault equation. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | mL/min (Mean) |
---|
| Week 2 (n=120,114) | Week 4 (n=119,113) | Week 6 (n=119,113) | Week 12 (n=117,112) | Week 16 (n=116,112) | Week 26 (n=115,112) | Week 40 (n=114,108) | Week 52 (n=115,110) |
---|
Mycophenolate Mofetil, Adapted Dose | 46.68 | 51.03 | 53.66 | 56.55 | 56.55 | 60.21 | 59.17 | 58.29 |
,Mycophenolate Mofetil, Fixed Dose | 45.26 | 46.73 | 49.77 | 53.01 | 53.49 | 55.18 | 56.81 | 56.45 |
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Creatinine Clearance Values Estimated With the Modification of Diet in Renal Disease (MDRD) Simplified Equation
The mean creatinine clearance values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52 estimated using the MDRD simplified equation. For males, the MDRD simplified equation was defined as MDRD (mL/min/1.73 square meters [m^2]) =186 multiplied by (*) serum creatinine in mg/L raised to the power of (^) -1.154 * age ^ -0.203. For females, the MDRD simplified equation was defined as MDRD (mL/min/1.73 m^2) = males formula * 0.742. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | mL/min/1.73 m^2 (Mean) |
---|
| Week 2 (n=120,114) | Week 4 (n=119,113) | Week 6 (n=119,113) | Week 12 (n=117,112) | Week 16 (n=116,112) | Week 26 (n=115,112) | Week 40 (n=114,108) | Week 52 (n=115,110) |
---|
Mycophenolate Mofetil, Adapted Dose | 40.61 | 45.02 | 48.12 | 50.93 | 50.93 | 54.22 | 52.23 | 50.82 |
,Mycophenolate Mofetil, Fixed Dose | 39.60 | 41.31 | 44.69 | 47.68 | 48.52 | 49.46 | 50.01 | 48.88 |
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Graft Histology - Percentage of Participants With at Least One Borderline Lesion at Week 12 and Week 52
Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52
Intervention | percentage of participants (Number) |
---|
| Week 12 | Week 52 |
---|
Mycophenolate Mofetil, Adapted Dose | 7.9 | 6.4 |
,Mycophenolate Mofetil, Fixed Dose | 12.4 | 5.0 |
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Graft Histology - Percentage of Participants With at Least One Chronic Graft Nephropathy at Week 12 and Week 52
Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52
Intervention | percentage of participants (Number) |
---|
| Week 12 | Week 52 |
---|
Mycophenolate Mofetil, Adapted Dose | 20.6 | 32.5 |
,Mycophenolate Mofetil, Fixed Dose | 17.4 | 34.7 |
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Percentage of Participants With at Least One BPAR at Week 12 and Week 52
BPAR was defined as the presence of clinical signs and kidney biopsy that confirmed the rejection before Week 12. Participants were censored at the date of last treatment, date of last contact or withdrawal, and date of death. (NCT02005562)
Timeframe: Weeks 12 and 52
Intervention | percentage of participants (Number) |
---|
| Week 12 | Week 52 |
---|
Mycophenolate Mofetil, Adapted Dose | 14.1 | 9.1 |
,Mycophenolate Mofetil, Fixed Dose | 10.0 | 10.0 |
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Serum Creatinine Values [Micromoles Per Liter (µmol/L)]
The mean serum creatinine values at Weeks 2, 4, 6, 12, 16, 26, 39, and 52. (NCT02005562)
Timeframe: Weeks 2, 4, 6, 12, 16, 26, 39, and 52
Intervention | µmol/L (Mean) |
---|
| Week 2 (n=120,114) | Week 4 (n=119,113) | Week 6 (n=119,113) | Week 12 (n=117,112) | Week 16 (n=116,112) | Week 26 (n=115,112) | Week 40 (n=114,108) | Week 52 (n=115,110) |
---|
Mycophenolate Mofetil, Adapted Dose | 202.41 | 168.92 | 155.74 | 140.80 | 145.48 | 131.73 | 136.24 | 148.62 |
,Mycophenolate Mofetil, Fixed Dose | 201.31 | 174.86 | 158.96 | 147.70 | 148.48 | 143.73 | 141.89 | 144.72 |
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Incidence of Chronic Graft Versus Host Disease (cGVHD)
Chronic graft versus host disease (cGVHD) is a reaction which typically develops 3 to 6 months after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. (NCT00176865)
Timeframe: 6 months and 1 year
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 1 |
Arm 2 - Matched Unrelated Donor | 0 |
Arm 3 - Mismatched Double Cord Donors | 0 |
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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
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 1 |
Arm 2 - Matched Unrelated Donor | 3 |
Arm 3 - Mismatched Double Cord Donors | 0 |
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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
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 1 |
Arm 2 - Matched Unrelated Donor | 1 |
Arm 3 - Mismatched Double Cord Donors | 0 |
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Number of Subjects Alive at 100 Days
(NCT00176865)
Timeframe: Day 100
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 3 |
Arm 2 - Matched Unrelated Donor | 8 |
Arm 3 - Mismatched Double Cord Donors | 4 |
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Number of Subjects Alive at One Year
(NCT00176865)
Timeframe: Day 365
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 3 |
Arm 2 - Matched Unrelated Donor | 7 |
Arm 3 - Mismatched Double Cord Donors | 3 |
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Number of Subjects With Mixed Chimerism
>10% Donor Cells at Day 100 (NCT00176865)
Timeframe: Day 100
Intervention | participants (Number) |
---|
Arm 1 - Matched Sibling Donor | 3 |
Arm 2 - Matched Unrelated Donor | 8 |
Arm 3 - Mismatched Double Cord Donors | 4 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1 - Matched Sibling Donor | 96.5 |
Arm 2 - Matched Unrelated Donor | 75.5 |
Arm 3 - Mismatched Double Cord Donors | 100 |
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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
Intervention | percentage of donor cells (Mean) |
---|
Arm 1 - Matched Sibling Donor | 88.9 |
Arm 2 - Matched Unrelated Donor | 73.3 |
Arm 3 - Mismatched Double Cord Donors | 90.5 |
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Percentage of Donor Chimerism at 365 Days
The percent of recipient bone marrow and blood cells that are of donor origin. (NCT00176865)
Timeframe: Day 365
Intervention | percentage of donor cells (Mean) |
---|
Arm 1 - Matched Sibling Donor | 81.9 |
Arm 2 - Matched Unrelated Donor | 78.6 |
Arm 3 - Mismatched Double Cord Donors | 91.7 |
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Death From GVHD
(NCT00112593)
Timeframe: Within the first 360 days
Intervention | Participants (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
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation) | 0 |
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Overall Survival
Kaplan-Meier estimate assessed at 1 year. (NCT00112593)
Timeframe: Up to 1 year
Intervention | survival probability (Number) |
---|
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation) | 0.40 |
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Progression of HIV
Count of participants with HIV progression. (NCT00112593)
Timeframe: Within 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation) | 0 |
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Reconstitution of HIV-specific Immunity
(NCT00112593)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation) | 2 |
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Successful Induction of Mixed Hematopoietic Chimerism as Assessed by the Percentage of Peripheral Blood T Cells That Are of Donor Origin
Determined by a DNA-based assay that compares the profile of amplified fragment length polymorphisms (ampFLP) of the patient and donor. (NCT00112593)
Timeframe: Up to day 80
Intervention | Participants (Count of Participants) |
---|
Treatment (Allogeneic Hematopoietic Stem Cell Transplantation) | 5 |
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Acute Rejection
Number of Participants with Acute Rejection (AR). AR is defined as allograft dysfunctions in the setting of recipient immune system engaging an allo-response against the kidney transplant. (NCT02213068)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Belatacept + MPA | 4 |
Belatacept + Low-Dose Tac | 0 |
Tacrolimus + MPA Standard Treatment Regimen | 2 |
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Change in eGFR (MDRD) at 2 Years Post-transplant Compared to Baseline at Month 3 (Conversion)
To assess change in renal function by calculated (MDRD) GFR of adult EBV seropositive renal transplant recipients of living or standard criteria donors converted from Tacrolimus to Belatacept or low dose Tacrolimus with Belatacept at three months post-operatively compared to renal transplant recipients randomized to remain on standard dose Tacrolimus and MPA for maintenance therapy at 2 years post-transplantation (NCT02213068)
Timeframe: 2 years
Intervention | mL/min/1.73m2 (Mean) |
---|
Belatacept + MPA | -5.44 |
Belatacept + Low-Dose Tac | 8.08 |
Tacrolimus + MPA Standard Treatment Regimen | -0.38 |
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Graft Survival
Number of Subjects with a functioning Graft (NCT02213068)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Belatacept + MPA | 8 |
Belatacept + Low-Dose Tac | 8 |
Tacrolimus + MPA Standard Treatment Regimen | 10 |
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Patient Survival
Number of Subjects alive at the end of 24 months (NCT02213068)
Timeframe: 2 years
Intervention | Participants (Count of Participants) |
---|
Belatacept + MPA | 9 |
Belatacept + Low-Dose Tac | 7 |
Tacrolimus + MPA Standard Treatment Regimen | 9 |
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Cumulative Incidence of Chronic GVHD
Cumulative incidence (measured as a percentage) of chronic graft versus host disease (GVHD). (NCT02120157)
Timeframe: 2 years
Intervention | percent (Number) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 11 |
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Cumulative Incidence of Non-relapse Mortality
Cumulative incidence (measured as a percentage) of non-relapse mortality at 180 days following myeloablative, Human Leukocyte Antigen (HLA)-mismatched bone marrow transplant (BMT) for patients with high risk hematologic malignancies. (NCT02120157)
Timeframe: Day 180
Intervention | percent (Number) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 0 |
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Incidence of Donor Cell Engraftment
Incidence of donor cell engraftment measured as the percentage of donor cell engraftment. (NCT02120157)
Timeframe: 60 days
Intervention | percentage of donor cell engraftment (Number) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 84 |
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Number of Participants With Donor Cell Engraftment
Number of Participants with Donor Cell Engraftment at Day 60 following myeloablative, HLA-mismatched BMT. (NCT02120157)
Timeframe: Day 60
Intervention | Participants (Count of Participants) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 27 |
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Cumulative Incidence of Acute Graft Versus Host Disease (GVHD) Grades 2-4 and Grades 3-4
Cumulative incidence (measured as a percentage) of acute GVHD grades 2-4 (overall) and grades 3-4 (severe). (NCT02120157)
Timeframe: 100 days
Intervention | percent (Number) |
---|
| Grades 2-4 | Grades 3-4 |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 10 | 0 |
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Primary and Secondary Graft Failure
Incidence (measured as a percentage) of primary and secondary graft failure. (NCT02120157)
Timeframe: 2 years
Intervention | percentage of graft failure (Number) |
---|
| Primary | Secondary |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 16 | 0 |
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Steroid and Non-steroid Immunosuppressants
Number of participants who used steroid and non-steroid immunosuppressants to treat GVHD. (NCT02120157)
Timeframe: Two Years
Intervention | Participants (Count of Participants) |
---|
| Steroid immunosuppressants | Non-steroid immunosuppressants |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 4 | 2 |
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Steroid and Non-steroid Immunosuppressants Use Duration
Duration of use of steroid and non-steroid immunosuppressants (in months) to treat GVHD. (NCT02120157)
Timeframe: Two Years
Intervention | months (Number) |
---|
| Steroid immunosuppressants | Non-steroid immunosuppressants |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 18 | 19 |
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Survival
Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 1 year. Incidence as a percentage. (NCT02120157)
Timeframe: up to 1 years
Intervention | percent (Number) |
---|
| overall survival (OS) | progression-free survival (PFS) | disease-free survival (DFS) | event-free survival | Relapse-free GVHD-free survival (GRFS) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 77 | 68 | 68 | 68 | 65 |
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Survival
Estimate incidence of overall survival (OS), progression-free survival (PFS), disease-free survival (DFS), event-free survival, and relapse-free GVHD-free survival (GRFS) in patients receiving myeloablative, HLA-mismatched BMT for patients with high risk hematologic malignancies at 2 years. Incidence as a percentage. (NCT02120157)
Timeframe: up to 2 years
Intervention | percent (Number) |
---|
| overall survival (OS) | progression-free survival (PFS) | disease-free survival (DFS) | event-free survival | Relapse-free GVHD-free survival (GRFS) |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 73 | 64 | 64 | 64 | 52 |
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Time to Neutrophil and Platelet Recovery
Time to neutrophil and platelet recovery in median days (NCT02120157)
Timeframe: 100 days
Intervention | days (Median) |
---|
| neutrophil | platelet |
---|
Haploidentical BMT With PTCy for Acute Leukemias and MDS | 22 | 21 |
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Duration of Wound Healing
A wound will be considered healed if all the suture material and staples are removed and the wound is intact. Number of participants is based on all patients of the respective treatment group in the safety set, excluding patients with no answer (unknown). (NCT01843348)
Timeframe: Post transplant until individual reporting
Intervention | days (Mean) |
---|
TAC+MPA | 42.4 |
TAC+Certican | 54.1 |
CycA+Certican | 85.3 |
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Glomular Filtration Rate (GFR) mL/Min Via Cockcroft- Gault Method at Month 12 Post Transplant
Cockcroft-Gault formula: For men: GFR= ((140-age) × body weight in kg)∕(72 x serum creatinine in mg∕dl)For women: GFR= (0.85×(140-age) × body weight in kg)∕(72 x serum creatinine in mg/dl), ), last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant
Intervention | mL/min per 1.73m² (Least Squares Mean) |
---|
TAC+MPA | 60.26 |
TAC+Certican | 52.25 |
CycA+Certican | 51.30 |
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Glomular Filtration Rate (GFR) Via Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) Method at Month 12 Post Transplant
Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) method = GFR=141 x min(Scr/κ, 1)α x max(Scr/κ, 1)1.209 x 0.993Age x 1.018 [if female] x 1.159 [if black] where Scr is serum creatinine, κ is 0.7 for females and 0.9 for males, α is 0.329 for females and 0.411 for males, min indicates the minimum of Scr/κ or 1, and max indicates the maximum of Scr/κ or 1. last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant
Intervention | mL/min per 1.73m² (Least Squares Mean) |
---|
TAC+MPA | 51.62 |
TAC+Certican | 44.42 |
CycA+Certican | 42.44 |
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Glomular Filtration Rate (GFR) Via Modification of Diet in Renal Disease (MDRD) Method at Month 12 Post Transplant
Modification of Diet in Renal Disease (MDRD) = For men: GFR = 170 x (serum creatinine -0,999) x (age-0,176) x (urea nitrogen -0,17) x (albumin0,318) For women: GFR = 170 x (serum creatinine -0,999) x (age-0,176) x (urea nitrogen -0,17) x albumin0,318) x 0.762 with urea nitrogen = urea / 2.144. last observation carried forward (LOCF) was used for imputation of missing values, ANCOVA model (NCT01843348)
Timeframe: Month 12 post transplant
Intervention | mL/min per 1.73m² (Least Squares Mean) |
---|
TAC+MPA | 53.24 |
TAC+Certican | 45.72 |
CycA+Certican | 43.47 |
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Glomular Filtration Rate (GFR) mL/Min Via Nankivell Method at Month 12 - Standard Regimen vs Certican Regimens
"To demonstrate non-inferiority in renal function assessed by glomerular filtration rate (Nankivell formula) in at least one of the Certican® treatment regimens compared to the standard regimen group at month 12 post-transplantation in renal transplant patients. Nankivell formula:~GFR = 6.7/Scr + BW/4 - Surea/2 - 100/(height)² + C where Scr is the serum creatinine concentration expressed in mmol/L, BW the body weight in kg, Surea the serum urea in mmol/L, height in m, and the constant C is 35 for male and 25 for female patients. The eGFR is expressed in mL/min per 1.73m². If a patient was on dialysis at the time of urea or creatinine assessment, the eGFR was set to 0. Analysis set = per protocol set" (NCT01843348)
Timeframe: One year post transplant
Intervention | mL/min per 1.73m² (Mean) |
---|
| Month 1 - Day 1 to 60 (146,111,78) | Month 3 - Day 61 to 136 (143,108,79) | Month 6 - Day 137 to 228 (142,108,76) | Month 9 - Day 229 to 319 (140,106,77) | Month12 - Day 320 to 450 (147,111, 80) |
---|
CycA+Certican | 59.47 | 62.22 | 63.17 | 62.89 | 61.51 |
,TAC+Certican | 60.54 | 61.21 | 62.76 | 64.68 | 63.34 |
,TAC+MPA | 62.62 | 66.36 | 68.05 | 69.47 | 70.41 |
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Percent of Participants With Delayed Graft Function and Slow Graft Function
Delayed graft function (DGF) was defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day. Slow graft function (SGF) was defined as a serum creatinine >3.0 mg/dL at Day 5 post-transplantation. Full analysis set (NCT01843348)
Timeframe: Post transplant to month 12
Intervention | Percent of participants (Number) |
---|
| Delayed graft function (197,187,172) | Slow graft function (195,187,171) |
---|
CycA+Certican | 22.1 | 49.7 |
,TAC+Certican | 20.3 | 48.7 |
,TAC+MPA | 17.8 | 46.2 |
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Percent of Participants With Delayed Graft Function by Day
Delayed graft function (DGF) was defined as the need for dialysis within the first 7 days post-transplantation, excluding the first post-transplantation day. (NCT01843348)
Timeframe: Post transplant up to day 7
Intervention | Percent of participants (Number) |
---|
| day 1 (8,7,4) | day 2 (2,2,2) | day 3 (5,1,2) | day 4 (4,2,4) | day 5 (4,5,3) | day 6 (1,1,2) | day 7 (2,4,0) | >7 days (9,16,21) |
---|
CycA+Certican | 10.5 | 5.3 | 5.3 | 10.5 | 7.9 | 5.3 | 0.0 | 55.3 |
,TAC+Certican | 18.4 | 5.3 | 2.6 | 5.3 | 13.2 | 2.6 | 10.5 | 42.1 |
,TAC+MPA | 22.9 | 5.7 | 14.3 | 11.4 | 11.4 | 2.9 | 5.7 | 25.7 |
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Percent of Participants With Viral Infections
Viral infections for BKV Virus Humane Polyomavirus 1 and Cytomegalovirus (NCT01843348)
Timeframe: Post transplant to month 12
Intervention | Percent of participants (Number) |
---|
| Viral infections - CMVMissing | Viral infections - CMVAsymptomatic | Viral infections - CMVMild | Viral infections - CMVModerate | Viral infections - CMVSevere | Viral infections - BKVAsymptomatic | Viral infections - BKVMild | Viral infections - BKVModerate | Viral infections - BKVSevere |
---|
CycA+Certican | 1.0 | 1.0 | 1.0 | 1.0 | 0.0 | 5.0 | 3.0 | 1.0 | 0 |
,TAC+Certican | 0.0 | 1.0 | 2.0 | 1.0 | 0.0 | 8.0 | 7.0 | 2.0 | 0 |
,TAC+MPA | 1.0 | 7.0 | 5.0 | 6.0 | 1.0 | 10.0 | 5.0 | 7.0 | 0 |
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Percent of Participants With Wound Healing Complications During Study
Information collected to report wound healing process which included percentage of participants with complications, fluid collections detected and occurrence of lymphoceles (NCT01843348)
Timeframe: Post transplant until individual reporting
Intervention | Percent of participants (Number) |
---|
| Wound healing complication | Fluids detected | Occurrence of lymphoceles |
---|
CycA+Certican | 22.2 | 27.8 | 21.8 |
,TAC+Certican | 19.1 | 26.8 | 18.2 |
,TAC+MPA | 14.3 | 18.7 | 11.8 |
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Percentage of Participants With Composite Treatment Failure Endpoints - Difference Between Groups at Month 12
Combined endpoint included: biopsy proven acute rejection (BPAR) defined as a rejection which was acute and proven by biopsy, graft loss (GL) defined as: allograft was presumed to be lost on the day the patient starts dialysis and not able to be removed from dialysis or death. Patients who prematurely discontinued the study: if the patient did not suffer from an event before discontinuation and reason was not related to efficacy, the patient was assessed as having had no event, otherwise the patient was assessed as having had an event. Full analysis set (FAS) (NCT01843348)
Timeframe: Month 12 post transplant
Intervention | Percentage of participants (Number) |
---|
| BPAR or graft loss or death | BPAR, graft loss, death, or loss of follow-up |
---|
CycA+Certican | 24.6 | 32.7 |
,CycA+Certican -Tac+MPA - Difference Between Groups | 14.9 | 17.1 |
,TAC+Certican | 13.0 | 22.6 |
,Tac+Certican - Tac+MPA - Difference Between Groups | 3.2 | 7.0 |
,TAC+MPA | 9.8 | 15.6 |
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Percentage of Participants With Treatment Failure Endpoints at Month 12
Treatment failure endpoints: biopsy proven acute rejection (BPAR) defined as a rejection which was acute and proven by biopsy, graft loss (GL) defined as: allograft was presumed to be lost on the day the patient starts dialysis and not able to be removed from dialysis or death. Patients who prematurely discontinued the study: if the patient did not suffer from an event before discontinuation and reason was not related to efficacy, the patient was assessed as having had no event, otherwise the patient was assessed as having had an event. Full analysis set (FAS) (NCT01843348)
Timeframe: Month 12 post transplant
Intervention | Percentage of participants (Number) |
---|
| Biopsy proven acute rejection (BPAR) | Treated BPAR (tBPAR) | Graft loss | Death |
---|
CycA+Certican | 24.6 | 23.6 | 9.0 | 6.5 |
,TAC+Certican | 12.0 | 11.5 | 6.3 | 6.3 |
,TAC+MPA | 9.3 | 8.8 | 5.4 | 4.9 |
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Cumulative Incidence of Chronic GVHD According to BMTCTN
Will be summarized with a proportion and a 95% confidence interval. (NCT01707004)
Timeframe: Up to 1 year
Intervention | percentage (Number) |
---|
Decitabine + Bone Marrow Transplant | 40.7 |
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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
Intervention | percentage of participants (Number) |
---|
Decitabine + Bone Marrow Transplant | 27.8 |
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Number of Participants With Complete Remission After Transplantation
(NCT01707004)
Timeframe: Up to 1 year
Intervention | Participants (Count of Participants) |
---|
Decitabine + Bone Marrow Transplant | 14 |
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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
Intervention | Participants (Count of Participants) |
---|
Decitabine + Bone Marrow Transplant | 0 |
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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
Intervention | percentage of participants (Number) |
---|
Decitabine + Bone Marrow Transplant | 64.7 |
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Percentage of Participants With Platelet Recovery by Day 30
Platelet recovery is defined as the first day of a platelet count greater than 20,000/mm^3 with no platelet transfusions. Will be summarized with mean and standard deviation or median and interquartile range, and the change will be tested using a one-sample paired t-test at a two-tailed significance level of 0.05. (NCT01707004)
Timeframe: Up to day 30
Intervention | percentage with plt engraftment, day 30 (Number) |
---|
Decitabine + Bone Marrow Transplant | 58 |
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Progression Free Survival
(NCT01707004)
Timeframe: Up to 1 year
Intervention | days (Median) |
---|
Decitabine + Bone Marrow Transplant | 141 |
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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
Intervention | days (Mean) |
---|
Decitabine + Bone Marrow Transplant | 16 |
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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
Intervention | Participants (Count of Participants) |
---|
Dose Level 1: 12 Gy Iodine-131+ BC8 Monoclonal Antibody | 0 |
Dose Level 7: 22 Gy Iodine-131+ BC8 Monoclonal Antibody | 2 |
Dose Level 8: 24 Gy Iodine-131+ BC8 Monoclonal Antibody | 1 |
Dose Level 9: 26 Gy Iodine-131+ BC8 Monoclonal Antibody | 0 |
Dose Level 10: 28 Gy Iodine-131+ BC8 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
| Day 28 Donor Chimerism | Day 84 Donor Chimerism |
---|
Dose Level 1: 12 Gy Iodine-131+ BC8 Monoclonal Antibody | 0 | 0 |
,Dose Level 10: 28 Gy Iodine-131+ BC8 | 7 | 4 |
,Dose Level 7: 22 Gy Iodine-131+ BC8 Monoclonal Antibody | 1 | 1 |
,Dose Level 8: 24 Gy Iodine-131+ BC8 Monoclonal Antibody | 3 | 3 |
,Dose Level 9: 26 Gy Iodine-131+ BC8 Monoclonal Antibody | 2 | 2 |
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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
Intervention | Participants (Count of Participants) |
---|
| Number of participants that are in CR 4 weeks after transplant | Number of participants that relapsed 4 weeks after transplant |
---|
Dose Level 1: 12 Gy Iodine-131 + BC8 Monoclonal Antibody | 0 | 1 |
,Dose Level 10: 28 Gy Iodine-131 + BC8 Monoclonal Antibody | 6 | 2 |
,Dose Level 7: 22 Gy Iodine-131 + BC8 Monoclonal Antibody | 2 | 0 |
,Dose Level 8: 24 Gy Iodine-131 + BC8 Monoclonal Antibody | 3 | 0 |
,Dose Level 9: 26 Gy Iodine-131 + BC8 Monoclonal Antibody | 1 | 1 |
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