A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Gaucher Disease, Type 1
Drug: Eliglustat tartrate
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||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)|
- Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared to Placebo [ Time Frame: Baseline, Week 39 ] [ Designated as safety issue: No ]Percent change in spleen volume = ([spleen volume at Week 39 minus spleen volume at baseline] divided by [spleen volume at baseline]) multiplied by 100, where all volumes are in MN.
- Hemoglobin Level [ Time Frame: Baseline ] [ Designated as safety issue: No ]
- Absolute Change From Baseline in Hemoglobin Level at Week 39 [ Time Frame: Baseline, Week 39 ] [ Designated as safety issue: No ]Absolute change = hemoglobin level at Week 39 minus hemoglobin level at baseline.
- Percent Change From Baseline in Liver Volume (in MN) at Week 39 [ Time Frame: Baseline, Week 39 ] [ Designated as safety issue: No ]Percent change in liver volume = ([liver volume at Week 39 minus liver volume at baseline] divided by [liver volume at baseline]) multiplied by 100, where all volumes are in MN.
- Percent Change From Baseline in Platelet Counts at Week 39 [ Time Frame: Baseline, Week 39 ] [ Designated as safety issue: No ]Percent change in platelet count = ([platelet count at Week 39 minus platelet count at baseline] divided by [platelet count at baseline]) multiplied by 100.
|Study Start Date:||November 2009|
|Estimated Study Completion Date:||November 2015|
|Primary Completion Date:||July 2012 (Final data collection date for primary outcome measure)|
Drug: Eliglustat tartrate
Eliglustat tartrate capsule as a single 50 milligram (mg) dose on Day 1 followed by eliglustat tartrate 50 mg capsule twice daily (BID) from Day 2 to Week 4, and then either eliglustat tartrate 50 mg capsule BID (in participants who had a Genz-99067 [active moiety of eliglustat tartrate in plasma] trough plasma concentration greater than or equal to [>=] 5 nanogram per milliliter [ng/mL]) or eliglustat tartrate 100 mg capsule BID (in participants who had a Genz-99067 trough plasma concentration less than [<] 5 ng/mL), up to Week 39. The pharmacokinetic (PK) assessment at Week 2 was used for dose adjustment after Week 4.
Other Name: Genz-112638
Placebo Comparator: Placebo
Matching placebo capsule once daily on Day 1 followed by matching placebo capsule BID from Day 2 through Week 39.
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 is designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate in adult participants (>16 years) with Gaucher disease Type 1. The study consists of 2 periods: The Double-Blind Primary Analysis Period (Day 1 to Week 39) and the Open-Label Period (post-Week 39 [Day 1 of the Open-Label Period] through study completion).
Please refer to this study by its ClinicalTrials.gov identifier: NCT00891202
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|Study Director:||Medical Monitor||Genzyme, a Sanofi Company|