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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00186810
Recruitment Status : Completed
First Posted : September 16, 2005
Last Update Posted : May 29, 2009
Information provided by:
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.

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Busulfan, Cyclophosphamide, Horse ATG Procedure: Allogeneic stem cell transplant Phase 2

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation From HLA/MLC Genotype Identical Donors for Patients With High Risk Sickle Cell Disease
Study Start Date : December 1992
Actual Primary Completion Date : February 2006
Actual Study Completion Date : October 2007

Resource links provided by the National Library of Medicine

Drug Information available for: Busulfan
U.S. FDA Resources

Arm Intervention/treatment
1 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

Primary Outcome Measures :
  1. 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. [ Time Frame: March 2007 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00186810

United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Sponsors and Collaborators
St. Jude Children's Research Hospital
Principal Investigator: Gregory Hale, MD St. Jude Children's Research Hospital

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: Gregory Hale, MD / Principal Investigator, St. Jude Children's Research Hospital Identifier: NCT00186810     History of Changes
Other Study ID Numbers: SCALLO
First Posted: September 16, 2005    Key Record Dates
Last Update Posted: May 29, 2009
Last Verified: May 2009

Keywords provided by St. Jude Children's Research Hospital:
Sickle Cell

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Hematologic Diseases
Genetic Diseases, Inborn
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists