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Sponsors and Collaborators: |
University of Minnesota National Marrow Donor Program |
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Information provided by: | University of Minnesota |
ClinicalTrials.gov Identifier: | NCT00176852 |
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease.
Condition | Intervention | Phase |
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Sickle Cell Disease Thalassemia Severe Congenital Neutropenia Diamond-Blackfan Anemia Shwachman-Diamond Syndrome |
Drug: Cyclophosphamide, Fludarabine, Campath Drug: Busulfan, Cyclophosphamide, ATG |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Parallel Assignment, Efficacy Study |
Official Title: | Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism |
Estimated Enrollment: | 30 |
Study Start Date: | June 2002 |
Estimated Study Completion Date: | June 2014 |
Estimated Primary Completion Date: | June 2012 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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1
Full Preparative Regimen for subjects with matched donors
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Drug: Cyclophosphamide, Fludarabine, Campath
Cyclophosphamide 50 mg/kg FLU 175 mg/m2 Campath 10 mg/kg TBI 300cGy
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2
Nonmyeloablative Preparative Regimen for subjects with mismatched related or unrelated donors
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Drug: Busulfan, Cyclophosphamide, ATG
BU 16 mg/kg CY 200 mg/kg ATG 90 mg/kg
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Prior to transplantation, subjects will receive either:
Cyclophosphamide Fludarabine Campath TBI
Or
Busulfan Cyclophosphamide ATG
These three drugs (and the radiation) are being given to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via IV catheter.
After stem cell transplantation, subjects will be given CSA and MMF/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.
Ages Eligible for Study: | up to 35 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients with SCD 0-35 years of age with an acceptable stem cell donor and disease characteristic defined by the following:
Second Transplants
Exclusion Criteria:
Contact: Tim Krepski, RN | 612-273-2800 | tkrepsk1@fairview.org |
United States, Minnesota | |
University of Minnesota Medical Center | Recruiting |
Minneapolis, Minnesota, United States, 55455 | |
Contact: Tim Krepski, RN 612-273-2800 tkrepsk1@fairiview.org | |
Principal Investigator: Angela Smith, MD |
Principal Investigator: | Angela Smith, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Smith, Angela R ) |
Study ID Numbers: | 0206M26241, MT2002-07 |
Study First Received: | September 12, 2005 |
Last Updated: | December 31, 2008 |
ClinicalTrials.gov Identifier: | NCT00176852 |
Health Authority: | United States: Institutional Review Board |
high risk hemoglobinopathy stem cell transplant donor lymphocyte infusion transfusion dependent |
stem cell donor cord blood marrow transfusion dependent non-malignant hematologic disorders |
Severe congenital neutropenia Shwachman-Diamond syndrome Leukocyte Disorders Cyclophosphamide Granulocytopenia Sickle cell anemia Red-Cell Aplasia, Pure Alemtuzumab Anemia, Diamond-Blackfan Anemia, Aplastic Hemoglobinopathy Anemia, Sickle Cell Hematologic Diseases Agranulocytosis Anemia |
Aase syndrome Anemia, Hemolytic Fludarabine monophosphate Thalassemia Shwachman syndrome Anemia, Hemolytic, Congenital Neutropenia Genetic Diseases, Inborn Busulfan Hemoglobinopathies Diamond Blackfan anemia Fludarabine Leukopenia Bone Marrow Diseases Aplastic anemia |
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 Antirheumatic Agents Alkylating Agents |