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Stem Cell Transplantation (SCT) for Genetic Diseases Clinical Trials References presented on Clinical Trials Search isn't meant to be a substitute for proven healthcare advice, trips or professional assistance using a genuine physician. We are not docs. Always confer with your physician about Stem Cell Transplantation (SCT) for Genetic Diseases conditions. Clinical Trials Search.org is a site devoted to listing clinical research studies in human subjects. Stem Cell Transplantation (SCT) for Genetic Diseases Clinical research trials and Stem Cell Transplantation (SCT) for Genetic Diseases healthcare trials happen in hundreds of localities throughout the United States of America. A clinical trial or clinical study is a research project with human volunteer subjects. Clinical drug trials and pharmaceutical clinical trials usually evaluate the potency of new drugs. The propose of the studies / projects is to answer particular human health questions. Clinical trials are a popular way for mDs, government agencies, and private sector companies to detect cures for all sorts of conditions, such as Stem Cell Transplantation (SCT) for Genetic Diseases. Stem Cell Transplantation (SCT) for Genetic Diseases Clinical Trials and other clinical trials allow volunteers to acquire healthcare treatment choices before they are available to the general public. Some times the subjects recieve professional assistance for free, and every now and again they are compensated for their time. Sometimes there is a cost for a Stem Cell Transplantation (SCT) for Genetic Diseases clinical trial. Subjects frequently obtain the most expert healthcare possible for their Stem Cell Transplantation (SCT) for Genetic Diseases condition. Risks are a reality, nevertheless, and can include more or frequent doctor trips, medical risks (possibly life-threatening), and/or the treatment being uneffective. Trials are federally governed with stern guidelines to protect clinical trials patients.
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Home > "S" Clinical Trials Conditions > Stem Cell Transplantation (SCT) for Genetic Diseases  Stem Cell Transplantation (SCT) for Genetic Diseases
Stem Cell Transplantation (SCT) for Genetic Diseases
For Condition: Pure Red-Cell Aplasia,Fanconi's Anemia,Inborn Errors of Metabolism,Thalassemia Major,Metachromatic Leukodystrophy,Thrombocytopenia
Status: Completed
Sponsor(s): National Center for Research Resources (NCRR) , University of California, Los Angeles
Synopsis: 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.
Details: 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:
Study Type: Interventional, Treatment, Efficacy Study
Minimum Age/Maximum Age: /17 Years
Genders: Both
Protocol Entry Criteria: 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
Total Enrollment:
Location and Contact Information:
Overall Study Official:
StephenFeig, Study Chair, University of California, Los Angeles
University of California Los Angeles Medical Center
Los Angeles, California, 90024
United States
Additional Information:
Study ID Numbers: 199/11981; UCLA-92010034
Study Start Date: January 1995
Record last reviewed: April 2002
Additional information available at: clinicaltrials.gov
Clinicaltrials.gov Reference link: NCT00004378
Other Metachromatic Leukodystrophy Studies:
1. Stem Cell Transplantation (SCT) for Genetic Diseases
Related Studies:
Other Metachromatic Leukodystrophy Clinical Trials
Other California Clinical Trials
Other Los Angeles Clinical Trials
Stem Cell Transplantation (SCT) for Genetic Diseases
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