NCT00891202

A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study Confirming the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 (ENGAGE)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.

Detailed description

Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Type 1 Gaucher disease, the most common form accounts for greater than (\>) 90% of cases and does not involve the central nervous system (CNS). Typical manifestations of Type 1 Gaucher disease include splenomegaly, hepatomegaly, thrombocytopenia, anemia, skeletal pathology and decreased quality of life. The disease manifestations are caused by the accumulations of glucosylceramide (storage material) in Gaucher cells which have infiltrated the spleen and liver as well as other tissue. Eliglustat tartrate is a small molecule developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells. This study was designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (\>16 years) with Gaucher disease Type 1. The study consisted of 2 periods: The Double-Blind Primary Analysis Period (PAP \[Day 1 to Week 39\]) and the Long Term Treatment Period (LTTP/Open-Label Period (post-Week 39 \[Day 1 of the Open-Label Period\] through study completion).

Conditions

• Gaucher Disease, Type 1

Interventions

Type	Name	Description
DRUG	Eliglustat tartrate	PAP: Eliglustat tartrate (ET) capsule 50 mg orally on Day 1 followed by ET 50 mg capsule twice daily (BID) from Day 2 to Week 4, then either ET 50 mg capsule BID (participants with Genz-99067 \[active moiety of ET in plasma\] trough plasma concentration \>=5 ng/mL) or ET 100 mg capsule BID (participants with Genz-99067 trough plasma concentration \<5 ng/mL), up to Week 39. PK assessment at Week 2 used for dose adjustment after Week 4. LTTP: Participants of the eliglustat arm in PAP who completed PAP were included in LTTP and received ET capsule 50 mg BID orally from Day 1 (post Week 39) until Week 43 followed by ET 50 mg or 100 mg capsule BID up to Week 47, then ET 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.
DRUG	Placebo	PAP: Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39. LTTP: Participants of the placebo arm in PAP who completed PAP were included in LTTP and received eliglustat tartrate from Day 1 (post Week 39) up to Week 312. Day 1 (post Week 39) was considered as baseline for LTTP. On Day 1, participants received eliglustat tartrate capsule 50 mg BID orally until Week 43 followed by eliglustat tartrate 50 mg or 100 mg capsule BID up to Week 47, then eliglustat tartrate 50 mg or 100 mg or 150 mg capsule BID up to Week 312. Dose adjustments at Week 43 and Week 47 were based on Genz-99067 trough plasma concentrations (if trough plasma concentration \<5 ng/mL: next higher dose administered; if \>=5 ng/mL: same dose continued) at Week 41 \& Week 45, respectively.

Timeline

• Start date: 2009-11-01
• Primary completion: 2012-07-01
• Completion: 2016-01-01
• First posted: 2009-05-01
• Last updated: 2017-03-03
• Results posted: 2014-09-03

Locations

18 sites across 12 countries: United States, Bulgaria, Canada, Colombia, India, Israel, Lebanon, Mexico, Russia, Serbia, Tunisia, United Kingdom