Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
|Genetic Disorders Sickle Cell Anemia||Procedure: Busulfan, Cyclophosphamide, BMD||Phase 2|
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis|
- Define the role of bone marrow transplantation for the treatment of sickle cell disease and the reversibility of sickle cell vasculopathy and organ damage, good risk thalassemia major and Diamond-Blackfan Anemia. [ Time Frame: 2 years ]
|Study Start Date:||January 1994|
|Study Completion Date:||August 2008|
|Primary Completion Date:||August 2008 (Final data collection date for primary outcome measure)|
Procedure: Busulfan, Cyclophosphamide, BMD
The trial proposed is a single armed phase II treatment protocol designed to examine the engraftment,toxicity and graft-versus-host disease following a novel cytoreductive regimen including cyclophosphamide and Busulfan for the treatment of patients with Severe Sickle Cell Anemia,Thalassemia, and Diamond Blackfan Anemia using stem cell transplants derived from HLA-genotypically identical siblings.
Patients will be conditioned for transplantation with cyclophosphamide (50 mg/kg/day x 4 days), and busulfan [(if < 4 years of age 1 mg/kg 4 times per day x 4 days), (if > 4 years of age 0.8 mg/kg 4 times per day x 4 days)]. Patients will receive Methotrexate & Cyclosporin-A for prophylaxis against GvHD and GCSF to promote engraftment.
The preferred source of stem cells from related HLA-matched related donors will be unmodified bone marrow stem cells.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00578435
|United States, New York|
|Memorial Sloan-Kettering Cancer Center|
|New York, New York, United States, 10065|
|Principal Investigator:||Farid Boulad, MD||Memorial Sloan Kettering Cancer Center|