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Study 5 of 211 for search of: | "Hemoglobinopathies" |
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Sponsored by: |
National Institute of Allergy and Infectious Diseases (NIAID) |
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Information provided by: | National Institute of Allergy and Infectious Diseases (NIAID) |
ClinicalTrials.gov Identifier: | NCT00034528 |
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high dose chemotherapy with transplantation.
Condition | Intervention | Phase |
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Hemoglobinopathies Anemia, Sickle Cell Hemoglobin SC Disease Thalassemia Thalassemia Major |
Drug: Busulfex Drug: Fludarabine Drug: FK506 Drug: Prednisone |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Allogeneic Stem Cell Transplantation Following Non-Myeloablative Chemotherapy in Patients With Hemoglobinopathies |
Enrollment: | 20 |
Study Start Date: | January 2005 |
Primary Completion Date: | December 2005 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1: Experimental
Participants will receive a non-myeloablative conditioning regimen of fludarabine and Busulfex prior to allogeneic peripheral blood stem cell infusions. FK506 and prednisone will be administered for graft versus host disease prophylaxis.
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Drug: Busulfex
0.8 mg/kg/d administered as a single intravenous infusion over 3 hours for 4 days. All infusions are anticipated to be given in the outpatient clinic.
Drug: Fludarabine
30 mg/m^2/d administered as a bolus infusion over 30 minutes for 4 days. All infusions are anticipated to be given in the outpatient clinic.
Drug: FK506
0.15 mg/kg taken orally daily for 12 to 14 weeks
Drug: Prednisone
0.5 mg/kg taken orally four times daily on Day 7 and increase to 1 mg/kg taken orally four times daily on Day 14. Participants will continue regimen until Day 30 before a 20-25% taper per week.
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Hemoglobinopathies, such as sickle cell disease and thalassemia major, are genetic diseases associated with significant morbidity and premature death. Allogeneic bone marrow transplantation (BMT) is the only potential cure for severe hemoglobinopathies. Typical regimens have used high doses of chemotherapy or chemo-radiotherapy to ablate recipient hematopoiesis and to prevent graft rejection. The widespread use of this treatment has been limited by toxicity, risk of end-organ damage, and donor availability. This study will use a non-myeloablative regimen of fludarabine and Busulfex to attempt to generate consistent engraftment with donor hematopoietic stem cells in patients with severe hemoglobinopathy.
G-CSF mobilization of the donor's peripheral blood white blood cells will precede donor apheresis. A non-myeloablative conditioning regimen of fludarabine and Busulfex will be administered to patients prior to allogeneic peripheral blood stem cell infusions. FK506 and prednisone will be administered for graft versus host disease prophylaxis. Patients will be evaluated for engraftment, donor:host hematopoietic chimerism, toxicity, and hemoglobinopathy.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion criteria:
All patients must:
Patients with sickle cell disease should have 1 or more of the following:
Patients with thalassemia should have 1 or more of the following:
Exclusion criteria:
United States, Massachusetts | |
Dana-Farber Cancer Institute/Harvard Cancer Center, Brigham and Women's Hospital and Massachusetts General Hospital | |
Boston, Massachusetts, United States, 02115 |
Principal Investigator: | Catherine J. Wu, MD | Dana-Farber Cancer Institute, Harvard Medical School |
Responsible Party: | DAIT/NIAID ( Associate Director, Clinical Research Program ) |
Study ID Numbers: | DAIT DF/HCC 01-098, P01 A 129530 |
Study First Received: | April 30, 2002 |
Last Updated: | September 26, 2008 |
ClinicalTrials.gov Identifier: | NCT00034528 |
Health Authority: | United States: Federal Government |
Prednisone Hemoglobin SC disease Hematologic Diseases Hemoglobin SC Disease Beta-thalassemia Anemia Anemia, Hemolytic Tacrolimus Fludarabine monophosphate Thalassemia |
Sickle cell anemia Anemia, Hemolytic, Congenital Thalassemia minor Genetic Diseases, Inborn Busulfan Beta-Thalassemia Hemoglobinopathies Fludarabine Hemoglobinopathy Anemia, Sickle Cell |
Anti-Inflammatory Agents Antineoplastic Agents, Hormonal Antineoplastic Agents Therapeutic Uses Physiological Effects of Drugs |
Hormones, Hormone Substitutes, and Hormone Antagonists Hormones Glucocorticoids Pharmacologic Actions |