NCT00578435

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 25 (estimated)
• Sponsor: Memorial Sloan Kettering Cancer Center · Academic / Other
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

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.

Conditions

• Genetic Disorders
• Sickle Cell Anemia

Interventions

Type	Name	Description
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

Timeline

• Start date: 1994-01-01
• Primary completion: 2008-08-01
• Completion: 2008-08-01
• First posted: 2007-12-21
• Last updated: 2008-09-12

Locations

1 site across 1 country: United States