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NCT00358150 · ClinicalTrials.gov registry record · Phase 2
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
A Phase 2 study of Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome, sponsored by Genzyme, a Sanofi Company.
- Completed
- Registry status
- Phase 2
- Development phase
- 26
- Enrollment target
- 17
- Study locations
NCT00358150 is a Phase 2 study of Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome that has completed, run by Genzyme, a Sanofi Company. The registered enrollment target is 26 participants, below the 56-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (54% lower). The trial reports 17 study locations across 2 states.
The verdict
NCT00358150, a Phase 2 study of Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome, has completed, sponsored by Genzyme, a Sanofi Company.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 26 participants
- Enrollment target
- 17
- Study locations
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 |
Interested in This Trial?
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, categorized as Phase 2. The registered enrollment target is 26 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 56-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (54% lower). The listed sponsor is Genzyme, a Sanofi Company, which has 196 total studies on file at ClinicalTrials.gov.
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.
NCT00358150 reports 17 study locations spanning 2 distinct geographic areas - top geographies include Other, New York.
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.
What interventions are being tested in trial NCT00358150?
The interventions under investigation include: Eliglustat tartrate (DRUG).
Who is sponsoring clinical trial NCT00358150?
This trial is sponsored by Genzyme, a Sanofi Company, which has 196 total clinical trials registered on ClinicalTrials.gov.
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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