Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-24 @ 10:07 PM
Ignite Modification Date:
2025-12-24 @ 10:07 PM
NCT ID:
NCT00578435
Brief Summary:
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.
Detailed Description:
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:
NCT00578435
Study Brief:
Protocol Section:
NCT00578435