Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
NA
INTERVENTIONAL
1995-01-31
Brief Summary
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II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
Detailed Description
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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.
Conditions
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Study Design
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TREATMENT
Interventions
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Stem Cell Transplantation
Eligibility Criteria
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Inclusion 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
0 Years
17 Years
ALL
No
Sponsors
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University of California, Los Angeles
OTHER
National Center for Research Resources (NCRR)
NIH
Principal Investigators
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Stephen A. Feig
Role: STUDY_CHAIR
University of California, Los Angeles
Locations
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University of California Los Angeles Medical Center
Los Angeles, California, United States
Countries
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Other Identifiers
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UCLA-92010034
Identifier Type: -
Identifier Source: secondary_id
199/11981
Identifier Type: -
Identifier Source: org_study_id