Stem Cell Transplantation (SCT) for Genetic Diseases

Sponsor
National Center for Research Resources (NCRR) (NIH)
Overall Status
Completed
CT.gov ID
NCT00004378
Collaborator
University of California, Los Angeles (Other)
1

Study Details

Study Description

Brief Summary

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.

  1. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
Condition or Disease Intervention/Treatment Phase
  • Procedure: Stem Cell Transplantation
N/A

Detailed Description

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 Design

Study Type:
Interventional
Primary Purpose:
Treatment
Study Start Date :
Jan 1, 1995

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Years to 17 Years
    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

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 University of California Los Angeles Medical Center Los Angeles California United States 90024

    Sponsors and Collaborators

    • National Center for Research Resources (NCRR)
    • University of California, Los Angeles

    Investigators

    • Study Chair: Stephen A. Feig, University of California, Los Angeles

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    , ,
    ClinicalTrials.gov Identifier:
    NCT00004378
    Other Study ID Numbers:
    • 199/11981
    • UCLA-92010034
    First Posted:
    Oct 19, 1999
    Last Update Posted:
    Jun 24, 2005
    Last Verified:
    Apr 1, 2002

    Study Results

    No Results Posted as of Jun 24, 2005