Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
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|ClinicalTrials.gov Identifier: NCT00578435|
Recruitment Status : Completed
First Posted : December 21, 2007
Last Update Posted : September 12, 2008
|Condition or disease||Intervention/treatment||Phase|
|Genetic Disorders Sickle Cell Anemia||Procedure: Busulfan, Cyclophosphamide, BMD||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||25 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis|
|Study Start Date :||January 1994|
|Actual Primary Completion Date :||August 2008|
|Actual Study Completion Date :||August 2008|
- Procedure: Busulfan, Cyclophosphamide, BMD
Busulfan 0.8 or 1 mg/Kg/day Days 8-6 Cyclophosphamide 50 mg/Kg/day Days 2-5 BMT Day 0
- 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 ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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|