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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: | NCT00176917 |
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 Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell disease.
Condition | Intervention | Phase |
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Mucopolysaccharidosis I Mucopolysaccharidosis VI Mannosidosis Mucolipidosis Type II (I-Cell 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: | Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis) |
Enrollment: | 38 |
Study Start Date: | May 1999 |
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 | Masonic Cancer Center, University of Minnesota |
Responsible Party: | Masonic Cancer Center at the University of Minnesota ( Orchard, Paul J., MD ) |
Study ID Numbers: | 0104M93821, MT1999-07 |
Study First Received: | September 12, 2005 |
Last Updated: | September 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00176917 |
Health Authority: | United States: Institutional Review Board |
stem cell transplant storage disease errors of metabolism |
Methylprednisolone Cyclophosphamide Brain Diseases Bone Diseases Alpha-L-iduronidase deficiency Metabolism, Inborn Errors Mucopolysaccharidoses Musculoskeletal Diseases I cell disease Connective Tissue Diseases Brain Diseases, Metabolic, Inborn Deficiency Diseases Alpha-Mannosidosis Metabolic Diseases Mucolipidoses |
Lysosomal Storage Diseases Mannosidase Deficiency Diseases Central Nervous System Diseases Bone Diseases, Metabolic Mucopolysaccharidosis I Antilymphocyte Serum Genetic Diseases, Inborn Busulfan Mucopolysaccharidosis Mucopolysaccharidosis type 6 Metabolic disorder Hurler syndrome Mucopolysaccharidosis VI Brain Diseases, Metabolic |
Immunologic Factors Molecular Mechanisms of Pharmacological Action Lysosomal Storage Diseases, Nervous System Antineoplastic Agents Nervous System Diseases Physiological Effects of Drugs Mucinoses Immunosuppressive Agents |
Pharmacologic Actions Therapeutic Uses Myeloablative Agonists Antineoplastic Agents, Alkylating Antirheumatic Agents Carbohydrate Metabolism, Inborn Errors Alkylating Agents |