Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
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.
Interventional
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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.
2 years
Yes
Farid Boulad, MD
Principal Investigator
Memorial Sloan-Kettering Cancer Center
United States: Institutional Review Board
94-005
NCT00578435
January 1994
August 2008
Name | Location |
---|---|
Memorial Sloan-Kettering Cancer Center | New York, New York 10021 |