- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004485
Bone Marrow Transplantation in Treating Children With Sickle Cell Disease
Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease
RATIONALE: Sickle cell disease is an inherited disorder in which abnormal, crescent-shaped red blood cells interfere with the ability of the blood to carry oxygen through the body and can cause severe pain, stroke, and organ damage. Bone marrow transplantation, is a procedure in which the soft, sponge-like tissue in the center of bones producing white blood cells, red blood cells, and platelets is replaced by bone marrow from a another person. Bone marrow transplantation may be an effective treatment in relieving the symptoms of sickle cell disease.
PURPOSE: Phase I/II trial to study the effectiveness of bone marrow transplantation in treating children who have sickle cell disease.
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.
Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.
Study Type
Enrollment
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
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California
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Oakland, California, United States, 94609
- Children's Hospital of Oakland
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Washington
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Seattle, Washington, United States, 98109
- Fred Hutchinson Cancer Research Center
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by: Recurrent painful events (at least 2 painful events in past year) which cannot be explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2 years that required hospitalization, oxygen, and RBC transfusion Any combination of painful events and ACS episodes that total 2 events within the past year Abnormal cerebral MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing performance
No stage III or IV sickle cell lung disease
Genotypically HLA identical sibling donor available
--Prior/Concurrent Therapy--
No prior transfusions with greater than 5 units RBC
--Patient Characteristics--
Performance status: Karnofsky 70-100%
Hepatic:
- No active hepatitis
- No moderate/severe portal fibrosis
Renal: Glomerular filtration rate at least 30% predicted for age
Neurologic:
- No severe residual functional neurologic impairment
- Hemiplegia alone allowed
Other:
- HIV negative
- Not pregnant or nursing
- Fertile patients must use effective contraception
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Mark Walters, Children's Hospital of Oakland
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Hemoglobinopathies
- Anemia, Sickle Cell
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Anti-Infective Agents
- Enzyme Inhibitors
- Antirheumatic Agents
- Antineoplastic Agents
- Immunosuppressive Agents
- Immunologic Factors
- Dermatologic Agents
- Anti-Bacterial Agents
- Antibiotics, Antineoplastic
- Antifungal Agents
- Antitubercular Agents
- Antibiotics, Antitubercular
- Calcineurin Inhibitors
- Fludarabine
- Mycophenolic Acid
- Cyclosporine
- Cyclosporins
Other Study ID Numbers
- 199/14243
- FHCRC-1373.00
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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