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Sponsors and Collaborators: |
Orchard, Paul J., MD Masonic Cancer Center, University of Minnesota |
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Information provided by: | University of Minnesota |
ClinicalTrials.gov Identifier: | NCT00176878 |
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for non-malignant congenital bone marrow failure disorders.
Condition | Intervention | Phase |
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Diamond-Blackfan Anemia Kostmann's Neutropenia Shwachman-Diamond Syndrome |
Procedure: Stem cell transplant Drug: Busulfan, fludarabine and anti-thymocyte globulin (ATG) Procedure: Total lymphoid irradiation |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders |
Estimated Enrollment: | 16 |
Study Start Date: | June 2000 |
Estimated Study Completion Date: | June 2010 |
Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
Prior to transplantation, subjects will receive the drugs busulfan (orally or through the catheter), as well as fludarabine and anti-thymocyte globulin (ATG) via the catheter. Busulfan, fludarabine and ATG will be given with Total Lymphoid Irradiation (TLI) to help the new donor bone marrow take and grow after transplantation.
Those patients receiving donor marrow will have the T cells (a type of white blood cell in the donor marrow) removed to lower the risk that the new marrow will react to their body, a condition called Graft-Versus-Host-Disease (GVHD). After bone marrow transplantation, subjects will receive drugs to help prevent GVHD, including cyclosporin and mycophenolate mofetil (MMF).
Blood samples are taken at day 28, day 60, day 100, 1 year and as required by medical status yearly for five years after transplant to evaluate how well the new marrow is growing. A bone marrow biopsy is required at day 21, at day 100 and 1 year.
Ages Eligible for Study: | up to 35 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients eligible for transplantation under this protocol will be <35 years of age, and will be diagnosed with:
Diamond Blackfan Anemia:
Kostmann's Neutropenia, Shwachman-Diamond syndrome:
Exclusion Criteria:
Contact: Paul Orchard, M.D. | 612-626-2961 | orcha001@umn.edu |
United States, Minnesota | |
University of Minnesota Medical Center | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu |
Principal Investigator: | Paul Orchard, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Paul Orchard, M.D. ) |
Study ID Numbers: | 9504M09637, MT2000-18 |
Study First Received: | September 12, 2005 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00176878 |
Health Authority: | United States: Institutional Review Board |
Stem cell transplant T-cell depletion TLI bone marrow failure disorders |
Hematologic Diseases Aase syndrome Anemia Agranulocytosis Shwachman-Diamond syndrome Pancytopenia Leukocyte Disorders Fludarabine monophosphate Granulocytopenia Shwachman syndrome Antilymphocyte Serum |
Red-Cell Aplasia, Pure Neutropenia Genetic Diseases, Inborn Busulfan Diamond Blackfan anemia Anemia, Diamond-Blackfan Anemia, Aplastic Fludarabine Aplastic anemia Leukopenia Bone Marrow Diseases |
Disease Immunologic Factors Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Physiological Effects of Drugs Immunosuppressive Agents Pharmacologic Actions |
Pathologic Processes Anemia, Hypoplastic, Congenital Syndrome Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Alkylating Agents |