Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.
PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.
|Sickle Cell Anemia||Drug: cyclosporine Drug: fludarabine Drug: mycophenolate mofetil Procedure: Bone Marrow Transplantation||Phase 1 Phase 2|
|Study Design:||Primary Purpose: Treatment|
|Official Title:||Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease|
|Study Start Date:||December 1999|
|Primary Completion Date:||August 2007 (Final data collection date for primary outcome measure)|
PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.
Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00004485
|United States, California|
|Children's Hospital of Oakland|
|Oakland, California, United States, 94609|
|United States, Washington|
|Fred Hutchinson Cancer Research Center|
|Seattle, Washington, United States, 98109|
|Study Chair:||Mark Walters||Children's Hospital of Oakland|