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Stem Cell Transplantation (SCT) for Genetic Diseases
NCT00004378 · View on ClinicalTrials.gov ↗
Study 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. II. Determine whether clinical manifestations of the specific disease may be arrested or reversed by this treatment.
Conditions Studied
Interventions
- PROCEDURE Stem Cell Transplantation
Study Locations (1)
California
- University of California Los Angeles Medical Center — Los Angeles
Trial Details
| Field | Value |
|---|---|
| Start Date | 1995-01 |
| Phase | NA |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00004378
The ClinicalTrials.gov registry entry for NCT00004378 describes a study currently listed as completed. It is categorized as NA, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. An enrollment target was not published in the registry record, which is common for early-stage or observational entries. The listed sponsor is National Center for Research Resources (NCRR), which has 20 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 6 conditions, with Thrombocytopenia appearing as the primary indexed condition, and to 1 intervention — of which Stem Cell Transplantation is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00004378 reports 1 study location spanning 1 distinct geographic area — top geographies include California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT00004378 about?
NCT00004378 is a clinical study titled "Stem Cell Transplantation (SCT) for Genetic Diseases". 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 b...
What is the current status of trial NCT00004378?
This trial is currently completed. It is a NA study. The study started on 1995-01.
What conditions does trial NCT00004378 study?
This clinical trial studies the following conditions: Thrombocytopenia, Inborn Errors of Metabolism, Thalassemia Major, Metachromatic Leukodystrophy, Fanconi's Anemia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00004378?
The interventions under investigation include: Stem Cell Transplantation (PROCEDURE). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00004378?
This trial is sponsored by National Center for Research Resources (NCRR), which has 20 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00004378 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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