Ignite Creation Date:
2025-12-24 @ 10:07 PM
Ignite Modification Date:
2026-01-13 @ 3:36 AM
Study NCT ID:
NCT00578435
Status:
COMPLETED
Last Update Posted:
2008-09-12
First Post:
2007-12-18
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Sponsor:
Memorial Sloan Kettering Cancer Center
Organization:
Study Overview
Official Title:
Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis
Status:
COMPLETED
Status Verified Date:
2008-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
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 Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: