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| Sponsors and Collaborators: |
National Center for Research Resources (NCRR) University of California, Los Angeles |
|---|---|
| Information provided by: | National Center for Research Resources (NCRR) |
| ClinicalTrials.gov Identifier: | NCT00004378 |
Purpose
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
| Condition | Intervention |
|---|---|
|
Thrombocytopenia Metachromatic Leukodystrophy Fanconi's Anemia Thalassemia Major Pure Red-Cell Aplasia Inborn Errors of Metabolism |
Procedure: Stem Cell Transplantation |
| Study Type: | Interventional |
| Study Design: | Treatment, Efficacy Study |
| Study Start Date: | January 1995 |
PROTOCOL OUTLINE: Patients receive either cyclophosphamide and high dose total body irradiation (TBI) or busulfan and cyclophosphamide.
Cyclophosphamide IV is given on days -5 and -4 and TBI on days -2, -1, and 0. Busulfan is given orally every 6 hours on days -9 through -6 and cyclophosphamide IV on days -5 through -2. Patients rest on day -1.
Patients receive bone marrow infusion on day 0. For GVHD prophylaxis, patients receive methotrexate on day 1, then on days 3, 6, and 11. Cyclosporine IV begins on day -2 over 12 hours, followed by continuous infusion for 21 days. Then, oral doses of cyclosporine are given every 12 hours to patients who tolerate oral feeding.
Cyclosporine is continued 6 months posttransplant, then tapered 10% per week and stopped.
Patients who receive genotypically HLA nonidentical stem cells undergo additional GVHD prophylaxis with methylprednisolone (IV or PO) or its equivalent every 12 hours on days 3 to day 100. Dose is then tapered as tolerated over 1 month.
Patients who receive cord blood stem cells receive methylprednisolone instead of methotrexate for GHVD prophylaxis. Methylprednisolone is given 3 times daily beginning on day 5 and continuing until day 17. Then, methylprednisolone is tapered 10% per week as clinically tolerated.
To accelerate engraftment, patients receive filgrastim IM daily beginning on day +1 and continuing until ANC equals 5000.
Eligibility| Ages Eligible for Study: | up to 17 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
--Patient Characteristics--
Contacts and Locations| United States, California | |
| University of California Los Angeles Medical Center | |
| Los Angeles, California, United States, 90024 | |
| Study Chair: | Stephen A. Feig | University of California, Los Angeles |
More Information
| Study ID Numbers: | 199/11981, UCLA-92010034 |
| Study First Received: | October 18, 1999 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004378 History of Changes |
| Health Authority: | United States: Federal Government |
|
Fanconi's anemia amegakaryocytic thrombocytopenia aplastic anemia congenital pure red cell aplasia genetic diseases and dysmorphic syndromes hematologic disorders |
inborn errors of metabolism metachromatic leukodystrophy pure red cell aplasia rare disease sphingolipidoses thalassemia major |
|
Lipid Metabolism, Inborn Errors Metachromatic Leukodystrophy Sphingolipidoses Leukodystrophy, Metachromatic Aplastic Anemia Brain Diseases Leukodystrophy Fanconi's Anemia Red-Cell Aplasia, Pure Metabolism, Inborn Errors Pure Red Cell Aplasia Thrombocytopenia Anemia, Aplastic Brain Diseases, Metabolic, Inborn Hemoglobinopathy |
Metabolic Disorder Metabolic Diseases Demyelinating Diseases Hematologic Diseases Blood Platelet Disorders Fanconi Anemia Lysosomal Storage Diseases Beta-thalassemia Rare Diseases Anemia Sphingolipidosis Anemia, Hemolytic Central Nervous System Diseases Thalassemia Thrombocytopathy |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Leukodystrophy, Metachromatic DNA Repair-Deficiency Disorders Brain Diseases Hereditary Central Nervous System Demyelinating Diseases Red-Cell Aplasia, Pure Metabolism, Inborn Errors Thrombocytopenia Anemia, Aplastic Brain Diseases, Metabolic, Inborn Metabolic Diseases Demyelinating Diseases Hematologic Diseases Lysosomal Storage Diseases, Nervous System |
Blood Platelet Disorders Lysosomal Storage Diseases Fanconi Anemia Nervous System Diseases Anemia Sulfatidosis Anemia, Hemolytic Central Nervous System Diseases Thalassemia Anemia, Hemolytic, Congenital Anemia, Hypoplastic, Congenital Genetic Diseases, Inborn Hemoglobinopathies Beta-Thalassemia Lipidoses |
