Stem Cell Transplantation (SCT) for Genetic Diseases

Completed · Not applicable

Conditions studied: Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major, Pure Red-Cell Aplasia, Inborn Errors of Metabolism

In brief

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.

Key facts

Study ID

NCT00004378

Run by

National Center for Research Resources (NCRR)

Starts

1995-01-01

Last updated by the study team

2005-06-24

Who can join

Age: any, up to 17. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of California Los Angeles Medical Center — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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