Stem Cell Transplantation (SCT) for Genetic Diseases
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| ClinicalTrials.gov Identifier: NCT00004378 |
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Recruitment Status :
Completed
First Posted : October 19, 1999
Last Update Posted : June 24, 2005
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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 or disease | Intervention/treatment | Phase |
|---|---|---|
| Thrombocytopenia Metachromatic Leukodystrophy Fanconi's Anemia Thalassemia Major Pure Red-Cell Aplasia Inborn Errors of Metabolism | Procedure: Stem Cell Transplantation | Not Applicable |
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.
| Study Type : | Interventional (Clinical Trial) |
| Primary Purpose: | Treatment |
| Study Start Date : | January 1995 |
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| Ages Eligible for Study: | 0 Years to 17 Years (Child) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
- Hereditary enzymopathies, such as: Metachromatic leukodystrophy
- Congenital Immunodeficiencies
- Heritable hematologic disorders, such as: Thalassemia major Refractory Diamond-Blackfan anemia Fanconi anemia Amegakaryocytic thrombocytopenia
--Patient Characteristics--
- Age: Under 18
- Other: SCT is performed using a histocompatible related donor, an unrelated donor, or an unrelated cord blood donor Haploidentical donors are accepted for patients with severe congenital immunodeficiency
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00004378
| 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 |
| ClinicalTrials.gov Identifier: | NCT00004378 |
| Other Study ID Numbers: |
199/11981 UCLA-92010034 |
| First Posted: | October 19, 1999 Key Record Dates |
| Last Update Posted: | June 24, 2005 |
| Last Verified: | April 2002 |
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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 |
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Leukodystrophy, Metachromatic Fanconi Syndrome Anemia Thrombocytopenia Thalassemia beta-Thalassemia Fanconi Anemia Red-Cell Aplasia, Pure Metabolism, Inborn Errors Hematologic Diseases Blood Platelet Disorders Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn |
Anemia, Hypoplastic, Congenital Anemia, Aplastic Congenital Bone Marrow Failure Syndromes Bone Marrow Failure Disorders Bone Marrow Diseases DNA Repair-Deficiency Disorders Metabolic Diseases Renal Tubular Transport, Inborn Errors Kidney Diseases Urologic Diseases Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases |