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

Phase 2

Completed

• Conditions: Sickle Cell Disease
• Interventions: Drug: Busulfan, Cyclophosphamide, Horse ATG; Procedure: Allogeneic stem cell transplant

• Registration Number: NCT00186810
• Lead Sponsor: St. Jude Children's Research Hospital

Brief 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.

Study Timeline

• Study Start: 1992-12
• Study First Submitted: 2005-09-09
• Study First Submitted QC: 2005-09-09
• Study First Posted: 2005-09-16
• Primary Completion: 2006-02
• Study Completion: 2007-10
• Status Verified: 2009-05
• Last Update Submitted: 2009-05-28
• Last Update Posted: 2009-05-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 15

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	Busulfan, Cyclophosphamide, Horse ATG	-
1	Allogeneic stem cell transplant	-

Primary Outcome Measures

Name	Time	Method
To evaluate engraftment, GVHD, hematopoietic and immune reconstitution, and regimen-related mortality and morbidity in patients with severe sickle cell disease undergoing transplant using either HLA matched sibling bone marrow or cord blood grafts.	March 2007

Trial Locations

Locations (1)

St. Jude Children's Research Hospital; 🇺🇸 Memphis, Tennessee, United States