NCT02536755

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Open Label Interventional Multicenter Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Successfully Completed the Phase 2 or Phase 3 Studies

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 31 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Primary Objective: Evaluate long term skeletal response to eliglustat in adult participants who successfully completed one of the Phase 2 or Phase 3 eliglustat studies. Secondary Objective: Evaluate the safety of eliglustat (by serious adverse event continuous monitoring), the quality of life (Short Form-36 Health Survey \[SF-36\]) and biomarkers of Gaucher disease type 1 (GD1) (chitotriosidase, plasma glucosylceramide \[GL-1\] and lyso glucosylceramide \[lyso-GL-1\]) in adult participants who successfully completed one of the Phase 2 or Phase 3 studies.

Detailed description

Study duration for individual participants was to be of minimum 2 years and up to 4 years, or until commercial eliglustat was available to participants through reimbursement. If after 4 years since the beginning of the study eliglustat was not approved and available to participants in participating country, participants in this country might continue to receive study treatment until eliglustat was available to participants through the compassionate use (expanded access) program or was approved and available through reimbursement, whichever came first.

Conditions

• Gaucher Disease

Interventions

Type	Name	Description
DRUG	Eliglustat, GZ385660	Pharmaceutical form: capsule Route of administration: oral

Timeline

• Start date: 2015-10-27
• Primary completion: 2021-06-24
• Completion: 2021-06-24
• First posted: 2015-09-01
• Last updated: 2022-07-15
• Results posted: 2022-07-15

Locations

4 sites across 3 countries: Canada, Russia, Tunisia