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Sponsored by: |
Memorial Sloan-Kettering Cancer Center |
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Information provided by: | Memorial Sloan-Kettering Cancer Center |
ClinicalTrials.gov Identifier: | NCT00578435 |
The purpose of this study is to determine and confirm the role of bone marrow transplantation in the treatment of disorders of the red cell and hemoglobin including sickle cell anemia, thalassemia and diamond blackfan anemia.
Condition | Intervention | Phase |
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Genetic Disorders Sickle Cell Anemia |
Procedure: Busulfan, Cyclophosphamide, BMD |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Open Label, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis |
Estimated Enrollment: | 25 |
Study Start Date: | January 1994 |
Study Completion Date: | August 2008 |
Primary Completion Date: | August 2008 (Final data collection date for primary outcome measure) |
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.
Ages Eligible for Study: | 1 Year and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
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 |
Responsible Party: | Memorial Sloan-Kettering Cancer Center ( Fourid Boulad ) |
Study ID Numbers: | 94-005 |
Study First Received: | December 18, 2007 |
Last Updated: | September 10, 2008 |
ClinicalTrials.gov Identifier: | NCT00578435 History of Changes |
Health Authority: | United States: Institutional Review Board |
ERYTHROPOIESIS Genetic Disorders Sickle Cell Anemia Thalassemia Diamond Blackfan Anemia |
Diamond-Blackfan Anemia Immunologic Factors Hematologic Diseases Anemia Anemia, Hemolytic Cyclophosphamide Immunosuppressive Agents Thalassemia Anemia, Hemolytic, Congenital Aase Syndrome |
Genetic Diseases, Inborn Busulfan Hemoglobinopathies Anemia, Diamond-Blackfan Sickle Cell Anemia Antineoplastic Agents, Alkylating Antirheumatic Agents Hemoglobinopathy Alkylating Agents Anemia, Sickle Cell |
Disease Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Hematologic Diseases Physiological Effects of Drugs Anemia Anemia, Hemolytic Cyclophosphamide Immunosuppressive Agents Pharmacologic Actions |
Anemia, Hemolytic, Congenital Pathologic Processes Genetic Diseases, Inborn Therapeutic Uses Hemoglobinopathies Busulfan Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents Anemia, Sickle Cell |