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Sponsored by: |
University of Minnesota |
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Information provided by: | University of Minnesota |
ClinicalTrials.gov Identifier: | NCT00352976 |
This is a single arm, TBI trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
Condition | Intervention | Phase |
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Fanconi Anemia |
Drug: Cyclophosphamide Drug: Fludarabine Drug: ATG Procedure: Total Body Irradiation Procedure: Bone Marrow Transplantation |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Crossover Assignment, Safety/Efficacy Study |
Official Title: | Total Body Irradiation Dose De-Escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation |
Estimated Enrollment: | 45 |
Study Start Date: | May 2006 |
Estimated Study Completion Date: | May 2016 |
Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
Study Treatment: 1. If the subject is to receive total body irradiation with thymic shielding, it will be given six days before the stem cells are given (day -6). 2. Day -5 through Day -2, subjects will receive a chemotherapy regimen of Fludarabine, Cyclophosphamide, ATG, and Methylprednisone (a steroid used to help make sure the transplant "takes") via central line (i.e. Hickman or Broviac). On days -5 to -1, subjects will receive ATG and Methylprednisone. The methylprednisone will continue until about three weeks after transplant.3. Starting Day -3, subjects will be given cyclosporin A (CSA) therapy to help prevent graft-versus-host-disease. We will continue to give CSA until about six months after the transplant. 4. If the subject is receiving bone marrow or "peripheral" stem cells (cells collected from the donor's arm via a cell separator), on the day of transplantation, the stem cells taken from the donor will be put into a machine which will separate the lymphocytes (the cells that cause graft-versus-host disease [GVHD]) from the stem cells. If the subject is receiving an umbilical cord blood, the lymphocytes will not be removed because the risk of GVHD is not as high. Otherwise all patients will receive the same treatment. The stem cells are given as an infusion into the subject's existing catheter over 1-2 hours on day 0.5. On the day after transplant (day +1) subjects will be given G-CSF to stimulate the growth of the transplanted cells. 6. While receiving treatment and until the subject's blood counts recover he/she will have daily blood tests, and several bone marrow biopsies and aspirates. After recovery, subjects will be seen once a month for a health assessment and blood tests until at least 3 months after the cells have been infused. Additional blood tests or assessments may be done as medically indicated.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Margaret L MacMillan, M.D. | 612-626-2778 | macmi002@umn.edu |
United States, Minnesota | |
University of Minnesota Medical Center | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Margaret L MacMillan, M.D. 612-626-2778 macmi002@umn.edu |
Principal Investigator: | Margaret L MacMillan, M.D. | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota Medical Center ( MacMillan, Margaret L., MD ) |
Study ID Numbers: | 0605M85788 |
Study First Received: | July 14, 2006 |
Last Updated: | June 16, 2008 |
ClinicalTrials.gov Identifier: | NCT00352976 |
Health Authority: | United States: Institutional Review Board |
Bone Marrow transplant stem cell transplant cord blood transplant total body irradiation thymic shielding |
Metabolic Diseases Hematologic Diseases Fanconi Anemia Anemia Cyclophosphamide Fludarabine monophosphate Genetic Diseases, Inborn |
Fanconi's anemia Anemia, Aplastic Fludarabine Bone Marrow Diseases Aplastic anemia Metabolic disorder |
Antimetabolites Antimetabolites, Antineoplastic Immunologic Factors Molecular Mechanisms of Pharmacological Action Antineoplastic Agents DNA Repair-Deficiency Disorders Physiological Effects of Drugs Immunosuppressive Agents |
Pharmacologic Actions Anemia, Hypoplastic, Congenital Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Alkylating Agents |