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

Sickle Cell Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00186810
• Other study ID #: SCALLO
• Status: Completed
• Phase: Phase 2
• First received: September 9, 2005
• Last updated: May 28, 2009
• Start date: December 1992
• Est. completion date: October 2007

Study information

• Verified date: May 2009
• Source: St. Jude Children's Research Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: October 2007
• Est. primary completion date: February 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 21 Years

Eligibility

Inclusion criteria:

Diagnosis of severe' disease is denoted by one of the following:

• Previous central nervous system vaso-occlusive episode with or without residual neurologic findings or

• Frequent painful vaso-occlusive episodes with significant interference with normal life activities and which necessitates chronic transfusion therapy or

• Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.

Exclusion criteria:

• Patient with SCD chronic lung disease greater than or equal to stage 3

• Patient with severe renal dysfunction defined as creatinine clearance < 40 ml/min/1.73m2.

• Patient with severe cardiac dysfunction defined as echocardiogram shortening fraction < 25%.

• Patient with HIV infection.

• Pregnant or lactating.

• Patient with unspecified chronic toxicity that in the opinion of the Principal Investigator is serious enough to detrimentally affect the patient's capacity to tolerate SCT.

• Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process

Study Design

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• Sickle Cell Disease

Intervention

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

Locations

Country	Name	City	State
United States	St. Jude Children's Research Hospital	Memphis	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
St. Jude Children's Research Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Yes

External Links

•   St. Jude Children's Research Hospital