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Sponsors and Collaborators: |
Baylor College of Medicine The Methodist Hospital System Texas Children's Hospital Center for Cell and Gene Therapy |
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Information provided by: | Baylor College of Medicine |
ClinicalTrials.gov Identifier: | NCT00040469 |
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
Condition | Intervention | Phase |
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Sickle Cell Anemia Hemoglobinopathy Thalassemia |
Drug: Campath -1H Drug: Dilantin Drug: Busulfan Drug: Cyclophosphamide |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Allogeneic Bone Marrow Transplant From HLA Identical Related Donors for Patients With High Risk Hemoglobinopathies: Hemoglobin SS, Hemoglobin SC, Hemoglobin SB0/+ Thalassemia, or Homozygous B0/+ Thalassemia or Severe Variants of B0/+ Thalassemia |
Estimated Enrollment: | 15 |
Study Start Date: | August 2000 |
To do the bone marrow transplant, we must first kill the cells in the bone marrow that make the abnormal red blood cells that are found in patients with severe thalassemia or sickle cell disease.
We will do this by using three drugs: busulfan, cyclophosphamide, and CAMPATH-1H. CAMPATH-IH is an investigational drug. CAMPATH-1H is used to prevent participants from rejecting or refusing to let the donor blood cells grow in the body. After the drug treatment, participants will be given bone marrow from a brother or sister who has healthy bone marrow that matches.
Ages Eligible for Study: | up to 64 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion:
Previous central nervous system vaso-occlusive episode with or without residual neurologic findings; Frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; Recurrent SCD chest syndrome events which necessitate chronic transfusion therapy.
Exclusion:
United States, Texas | |
Texas Children's Hospital | |
Houston, Texas, United States, 77030 | |
The Methodist Hospital | |
Houston, Texas, United States, 77030 |
Study Chair: | Malcolm K. Brenner, MD | Baylor College of Medicine |
Study ID Numbers: | H6847, Scallo2 |
Study First Received: | June 26, 2002 |
Last Updated: | April 9, 2007 |
ClinicalTrials.gov Identifier: | NCT00040469 |
Health Authority: | United States: Food and Drug Administration |
Hemoglobin SC disease Hematologic Diseases Hemoglobin SC Disease Anemia Anemia, Hemolytic Cyclophosphamide Thalassemia Sickle cell anemia |
Phenytoin Anemia, Hemolytic, Congenital Genetic Diseases, Inborn Busulfan Alemtuzumab Hemoglobinopathies Hemoglobinopathy Anemia, Sickle Cell |
Molecular Mechanisms of Pharmacological Action Immunologic Factors Antineoplastic Agents Therapeutic Uses Physiological Effects of Drugs Myeloablative Agonists |
Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents Immunosuppressive Agents Pharmacologic Actions |