Clinical trialNCT00004378CompletedInterventional

Stem Cell Transplantation (SCT) for Genetic Diseases

SponsorNational Center for Research Resources (NCRR)Fanconi's AnemiaInborn Errors of MetabolismMetachromatic LeukodystrophyPure Red-Cell AplasiaThalassemia MajorThrombocytopenia

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

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Investigators

The overall officials registered on this study. Names that resolve to a FindMyKOL profile link to it; the rest are shown as registered.

Stephen A. Feig
University of California, Los Angeles
Study Chair
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A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

1 registered facility, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

United States

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

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

No drug or biological interventions are in our data for this study. It may test a procedure, device or behavioral intervention, or the intervention list isn't in our current snapshot. See the registry record ↗. Why? →

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 31 Jan 1995
Primary completion: —
Completion: —
Last update posted: 24 Jun 2005
Registered enrollment: —
Registry id: NCT00004378

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Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 4 Nov 2026. Registry facts are shown as registered by the study sponsor; FindMyKOL does not interpret them.