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

This study has been completed.
Information provided by:
St. Jude Children's Research Hospital Identifier:
First received: September 9, 2005
Last updated: May 28, 2009
Last verified: May 2009

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 Intervention Phase
Sickle Cell Disease Drug: Busulfan, Cyclophosphamide, Horse ATG Procedure: Allogeneic stem cell transplant Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:

Further study details as provided by St. Jude Children's Research Hospital:

Primary Outcome Measures:
  • 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 ]

Enrollment: 15
Study Start Date: December 1992
Study Completion Date: October 2007
Primary Completion Date: February 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

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
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Please refer to this study by its identifier: 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
  More Information

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
Study First Received: September 9, 2005
Last Updated: May 28, 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 processed this record on September 21, 2017