Conditions: Gaucher Disease
Keywords: Gaucher disease Genz-112638 beta-glucosidase acid β-glucosidase glucocerebrosidase glucosylceramide D-glucosyl-N-acylsphingosine glucohydrolase substrate reduction therapy
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 170
Healthy volunteers: no

Brief summary:
The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).

Detailed description:
NOTE: Other Phase 3 studies being conducted with eliglustat tartrate (Genz-112638) are GZGD02507 (ENGAGE): NCT00891202 and GZGD02607 (ENCORE): NCT00943111

Arms:
arm 1: Patients will receive either 50 mg BID or 100 mg BID arm 2: Patients will receive either 100 mg QD or 200 mg QD

Interventions: intervention 1: Eliglustat tartrate
Intervention descriptions: intervention 1: Oral Capsule in 50 mg or 100 mg dosages

Protocol document: