Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia
Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation. In addition, depletion CD3±CD19 cells may contribute to prevent developing severe acute graft versus host disease (GVHD) in haploidentical transplantation.
Purpose: This phase I/II trial is to evaluate the safety and efficacy of fludarabine, cyclophosphamide and antithymocyte globulin with CD3±CD19 depleted graft from haploidentical donors in treating patients with aplastic anemia.
|Aplastic Anemia||Biological: anti-thymocyte globulin Biological: filgrastim Drug: Fludarabine Drug: Cyclophosphamide Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation||Phase 1 Phase 2|
|Study Design:||Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||HLA-haploidentical Allogeneic Hematopoietic Cell Transplantation Using CD3±CD19 Depletion for Patients With Aplastic Anemia After Conditioning of Fludarabine, Cyclophosphamide and Antithymocyte Globulin|
- To assess the engraftment rate and survival of CD3±CD19 depleted haploidentical peripheral blood stem cell transplantation after conditioning with fludarabine, cyclophosphamide and anti-thymocyte globulin. [ Time Frame: 2 years post-transplant ]
- To assess engraftment and graft failure [ Time Frame: 28 days post-transplant ]Number of patients who failed to engraft by 28 days.
- To estimate the risk of acute GVHD [ Time Frame: 100 days post-transplant ]Number of patients with acute GVHD.
- To assess treatment related mortality [ Time Frame: 100 days post-transplant ]Number of death after transplantation
- To estimate overall survival [ Time Frame: 1 year after transplantation ]
|Study Start Date:||July 2009|
|Study Completion Date:||July 2012|
|Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Biological: anti-thymocyte globulin
On days -3 to -1Biological: filgrastim
Beginning on day 4 and continuing until blood counts recoverDrug: Fludarabine
30mg/M2 once daily IV on days -6 to -2Drug: Cyclophosphamide
60 mg/kg IV on day-3 and -2Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation
Immunogenetic depletion on CliniMACS
Please refer to this study by its ClinicalTrials.gov identifier: NCT01105273
|Korea, Republic of|
|Asan Medical Center|
|Seoul, Korea, Republic of, 138-736|
|Principal Investigator:||Ho Joon Im, MD & PhD||Asan Medical Center|