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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: Completed Phase 2 study of Gaucher Disease, Type 1 and Cerebroside Lipidosis Syndrome, sponsored by Genzyme, a Sanofi Company.

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. According to ClinicalTrials.gov, the official US trial registry.

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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.

Primary Outcome

A meaningful clinical response was defined as an improvement in at least 2 of the 3 main efficacy parameters: a) an increase in hemoglobin of greater than or equal to (\>=) 0.5 gram/deciliter from baseline, b) an increase in platelets of \>=15 percent (%) from baseline, c) reduction in total spleen volume of \>= 15% from baseline. As hemoglobin, platelets, total spleen volume were abnormal at baseline, within each participant, only those parameters were used in the evaluation of meaningful clini

Conditions Studied

Gaucher Disease, Type 1 Cerebroside Lipidosis Syndrome Glucocerebrosidase Deficiency Disease Glucosylceramide Beta-Glucosidase Deficiency Disease Gaucher Disease, Non-Neuronopathic Form

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

FieldValue
Enrollment Target 26 participants
Start Date 2006-06
Est. Completion 2015-12
Phase Phase 2
Genzyme, a Sanofi Company

196 total trials

What the finished NCT00358150 record still lists

NCT00358150 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 26 participants, a relatively small participant target, below the 56-participant average among 8 other Gaucher Disease, Type 1 trials with a reported enrollment target (54% lower).

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 lists 17 locations in 2 states (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.

Learn More About Clinical Trials

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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT00358150's enrollment target sits among peer trials

26 6th of 8 higher than 3 of 8 other Gaucher Disease, Type 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Gaucher Disease, Type 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Related registry views

Source: ClinicalTrials.gov NCT00358150, the US trial registry maintained by the National Library of Medicine. NCT00358150 (small enrollment · multi site footprint · completed) retrieved and formatted by PlainTrial, see methodology.