Stem Cell Transplantation With Identical Donors for Patients With Sickle Cell Disease
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Purpose
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 Endpoint Classification: Efficacy Study Intervention Model: Single Group Assignment Masking: 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 |
- 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 ] [ Designated as safety issue: Yes ]
| 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.
Eligibility| Ages Eligible for Study: | up to 21 Years |
| Genders Eligible for Study: | Both |
| 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
Contacts and Locations| United States, Tennessee | |
| St. Jude Children's Research Hospital | |
| Memphis, Tennessee, United States, 38105 | |
| Principal Investigator: | Gregory Hale, MD | St. Jude Children's Research Hospital |
More Information
Additional Information:
No publications provided
| Responsible Party: | Gregory Hale, MD / Principal Investigator, St. Jude Children's Research Hospital |
| ClinicalTrials.gov Identifier: | NCT00186810 History of Changes |
| Other Study ID Numbers: | SCALLO |
| Study First Received: | September 9, 2005 |
| Last Updated: | May 28, 2009 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by St. Jude Children's Research Hospital:
|
Anemia Sickle Cell |
Additional relevant MeSH terms:
|
Anemia, Sickle Cell Anemia, Hemolytic, Congenital Anemia, Hemolytic Anemia Hematologic Diseases Hemoglobinopathies Genetic Diseases, Inborn Busulfan Cyclophosphamide Immunosuppressive Agents |
Immunologic Factors Physiological Effects of Drugs Pharmacologic Actions Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Therapeutic Uses Myeloablative Agonists Antirheumatic Agents |
ClinicalTrials.gov processed this record on May 19, 2013