NCT00004485

Bone Marrow Transplantation in Treating Children With Sickle Cell Disease

Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 50 (planned)
• Sponsor: Fred Hutchinson Cancer Center · Academic / Other
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Sickle Cell Anemia

Interventions

Type	Name	Description
DRUG	cyclosporine
DRUG	fludarabine
DRUG	mycophenolate mofetil
PROCEDURE	Bone Marrow Transplantation

Timeline

• Start date: 1999-12-01
• Primary completion: 2007-08-01
• First posted: 1999-10-19
• Last updated: 2008-09-10

Locations

2 sites across 1 country: United States