NCT00186810

Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease

Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: St. Jude Children's Research Hospital · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

This protocol studied the effect of administration of a myeloablative pretransplant preparative regimen followed by an infusion of donor stem cells in children with severe sickle cell disease. The donor graft consisted of bone marrow or cord blood derived from a genetically matched sibling. The primary aim of the study was to evaluate how well the donated cells migrated to the bone marrow and begin producing healthy red blood cells, white blood cells and platelets (engrafted), how well the recipients immune system recovered, and assess any regimen related toxicities including a potentially life-threatening transplant related complication called graft-versus-host-disease or GVHD.

Detailed description

The secondary objectives of this protocol evaluated the effect of this transplant procedure on the subsequent clinical course of patients with severe SCD. Specifically, to determine whether pre-transplant organ dysfunction (brain, heart, lung, kidney, liver, spleen, etc) resultant from sickle hemoglobinopathy can be reversed following this particular transplant procedure.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Busulfan, Cyclophosphamide, Horse ATG	Transplant recipients received a myeloablative conditioning regimen of cyclophosphamide, Anti-Thymocyte Globulin (horse), and Busulfan. Cyclosporine and methotrexate were administered for GVHD prophylaxis.
PROCEDURE	Allogeneic stem cell transplant	Allogeneic stem cell transplant Matched sibling donor transplant Cord blood transplant

Timeline

• Start date: 1992-12-01
• Primary completion: 2006-02-01
• Completion: 2007-10-01
• First posted: 2005-09-16
• Last updated: 2009-05-29

Locations

1 site across 1 country: United States