Study of Skeletal Response to Eliglustat in Patients With Gaucher Disease
Completed
Genzyme, a Sanofi Company
Phase 3
2015-10-28
Primary Objective:
Evaluate long term skeletal response to eliglustat in adult patients who successfully
completed one of the Phase 2 or Phase 3 eliglustat studies.
Secondary Objective:
Evaluate the safety of eliglustat (by [serious] adverse event [AE] 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 patients who successfully completed one of the Phase 2 or Phase 3
studies.
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Recruiting
Sanofi
Phase 3
2018-04-11
Primary Objective:
Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (≥2 to <18 years
old).
Secondary Objective:
Evaluate the efficacy of eliglustat and quality of life in pediatric patients (≥2 to <18
years old).
Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III
Gaucher disease patients and the efficacy on soft tissue diseases.
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