Thrombocytopenia Clinical Trial
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.
Status | Completed |
Enrollment | 0 |
Est. completion date | |
Est. primary completion date | |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 17 Years |
Eligibility |
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 |
Endpoint Classification: Efficacy Study, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | University of California Los Angeles Medical Center | Los Angeles | California |
Lead Sponsor | Collaborator |
---|---|
National Center for Research Resources (NCRR) | University of California, Los Angeles |
United States,
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