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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: | NCT00176904 |
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic storage disease.
Condition | Intervention | Phase |
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Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Cell Leukodystrophy Gaucher's Disease Fucosidosis Wolman Disease Niemann-Pick Disease Batten Disease GM1 Gangliosidosis Tay Sachs Disease Sandhoff Disease |
Procedure: Stem Cell Transplant Drug: Busulfan, Cyclophosphamide, ATG |
Phase II Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study |
Official Title: | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation |
Enrollment: | 135 |
Study Start Date: | January 1995 |
Study Completion Date: | March 2008 |
Primary Completion Date: | March 2008 (Final data collection date for primary outcome measure) |
Prior to transplantation, subjects will receive Busulfan intravenously (IV) via the Hickman line four times daily for 4 days, Cyclophosphamide intravenously via the Hickman line once a day for 4 days, and Anti-Thymocyte Globulin IV via the Hickman line twice daily for three days before the transplant. These three drugs are being given to subjects to help the new marrow "take" and grow.
On the day of transplantation, the donor's hematopoietic cells will be transfused via central venous catheter.
After hematopoietic cell transplant, subjects will then receive two drugs, cyclosporin and either methylprednisolone or Mycophenolate Mofetil (MMF). Cyclosporin and methylprednisolone or MMF are given to help prevent the complication of graft-versus-host disease and to decrease the chance that the new donor cells will be rejected.
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Minnesota | |
University of Minnesota Medical Center | |
Minneapolis, Minnesota, United States, 55455 |
Principal Investigator: | Paul Orchard, MD | University of Minnesota Medical Center |
Responsible Party: | University of Minnesota ( Orchard, Paul J., MD ) |
Study ID Numbers: | 9412M09107, MT1995-01 |
Study First Received: | September 12, 2005 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00176904 |
Health Authority: | United States: Institutional Review Board |
Inborn errors Storage disease errors of metabolism stem cell transplant |
Pick Disease of the Brain Sphingolipidoses Frontotemporal dementia Methylprednisolone Adrenal Gland Diseases Demyelinating diseases Tay-Sachs disease Hypoadrenalism Brain Diseases Neurodegenerative Diseases Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Adrenoleukodystrophy Addison Disease Infant, Newborn, Diseases |
Spielmeyer-Vogt disease Metachromatic leukodystrophy Brain Diseases, Metabolic, Inborn Tay-Sachs Disease Sandhoff disease Niemann-Pick Disease Delirium Adrenal Insufficiency Speech Disorders Cholesterol Ester Storage Disease Metabolic Diseases Demyelinating Diseases Lysosomal Storage Diseases Aphasia Language Disorders |
Reticuloendotheliosis Molecular Mechanisms of Pharmacological Action Immune System Diseases Immunologic Factors Antineoplastic Agents Lysosomal Storage Diseases, Nervous System Nervous System Diseases Physiological Effects of Drugs Sulfatidosis Hereditary Central Nervous System Demyelinating Diseases |
Immunosuppressive Agents Pharmacologic Actions Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Histiocytosis, Non-Langerhans-Cell Mental Retardation, X-Linked Alkylating Agents Carbohydrate Metabolism, Inborn Errors |