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A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
NCT00358150 · View on ClinicalTrials.gov ↗
Study Summary
Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system. Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. The primary objective of this study is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate, administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to men and women with Gaucher disease Type 1 for 52 weeks.
Conditions Studied
Interventions
- DRUG Eliglustat tartrate
Study Locations (17)
Other
- Aprillus Asistencia e Investigación — Buenos Aires
- Hospital de Oncologia Maria Curie — Buenos Aires
- Hospital Ramos Mejia — Buenos Aires
- IMAI — Buenos Aires
- Instituto Argentino de Diagnostico y Tratamiento (IADT) — Buenos Aires
- — Buenos Aires
- Rambam Medical Center — Haifa
- — Haifa
- Sha'are Zedek Medical Centre — Jerusalem
- — Jerusalem
- Universita degli Studi di Milano — Milan
- Instituto Mexicano del Seguro Social — D.f.
- — Mexico City
- Hematology Research Center of Ministry of Healthcare of the Russian Federation — Moscow
- — Moscow
New York
- New York University — New York
- — New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 26 participants |
| Start Date | 2006-06 |
| Est. Completion | 2015-12 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00358150
The ClinicalTrials.gov registry entry for NCT00358150 describes a study currently listed as completed. It is categorized as Phase 2, 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. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Genzyme, a Sanofi Company, which has 52 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 5 conditions, with Gaucher Disease, Type 1 appearing as the primary indexed condition, and to 1 intervention — of which Eliglustat tartrate 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: NCT00358150 reports 17 study locations spanning 2 distinct geographic areas — top geographies include Other, New York. 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 NCT00358150 about?
NCT00358150 is a clinical study titled "A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients". Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In participants with Gaucher disease, the ...
What is the current status of trial NCT00358150?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 26 participants. The study started on 2006-06. Estimated completion is 2015-12.
What conditions does trial NCT00358150 study?
This clinical trial studies the following conditions: Gaucher Disease, Type 1, Cerebroside Lipidosis Syndrome, Glucocerebrosidase Deficiency Disease, Glucosylceramide Beta-Glucosidase Deficiency Disease, Gaucher Disease, Non-Neuronopathic Form. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00358150?
The interventions under investigation include: Eliglustat tartrate (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00358150?
This trial is sponsored by Genzyme, a Sanofi Company, which has 52 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00358150 being conducted?
This trial has 17 study locations across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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