At a glance

ClinicalIndex Comparison Record

Phase 2Completed· 26 enrolled

Drug / intervention

Eliglustat tartratedrug

Likely dose

Eliglustat tartrate 50 mgfrom record

Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00358150

NCT00358150Phase 2Completed

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A Phase 2, Open-Label, Multi-Center Study Evaluating the Efficacy, Safety and Pharmacokinetics of Genz-112638 in Gaucher Type 1 Patients

Genzyme, a Sanofi Company·interventional·Posted Jul 31, 2006·Updated Feb 15, 2017

In Brief

A Phase 2 clinical trial evaluating Eliglustat tartrate for Gaucher Disease, Type 1 and 4 related conditions. Completed, enrolled 26 participants across 17 sites in 6 countries.

Detailed 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.

Study Details

Study Typeinterventional

Allocation--

Masking--

Primary Purpose--

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

CountriesArgentina, Israel, Italy, Mexico, Russia, United States

Collaborators--

Timeline

Phase 2CompletedFinished

2006200720082009201020112012201320142015201620172018201920202021202220232024202520262027

Enrollment StartJun 2006

First PostedJul 2006

Primary CompletionAug 2009

Study CompletionDec 2015

TodayJul 2026

First PostedJul 31, 2006

Enrollment StartJun 1, 2006

Primary CompletionAug 1, 2009

Study CompletionDec 1, 2015

TodayJul 2, 2026

Enrollment to primary: 3.2 yearsPosted 19.9 years ago

Interventions

Eliglustat tartratedrug

Eliglustat (Genz-112638) capsule as single 50 mg dose on Day 1 then eliglustat 50 mg twice daily (BID) from Day 2 to Day 19, then either eliglustat 50 mg BID (if Genz-99067\[active moiety of eliglustat in plasma\] trough plasma concentration was greater than or equal to \[\>=\]5 nanogram per milliliter \[ng/mL\] on Day 10) or eliglustat 100 mg BID(if Genz-99067 trough plasma concentration was less than \[\<\] 5 ng/mL), from day 20 to Year 4. After primary completion date (Week 52) participants underwent treatment interruption period of approximately 2 weeks before continuing same treatment through study completion (Year 9). Participant receiving 100 mg BID could be considered for further dose increase to 150 mg BID at Week 24 if they met certain criteria (for example, had been on treatment for at least 24 months, had not reached therapeutic goals established for participants receiving Cerezyme, and if all other causes for lack of treatment effect had been evaluated and ruled out).