Study of Skeletal Response to Eliglustat in Patients With Gaucher Disease
Status:
Completed
Trial end date:
2021-06-24
Target enrollment:
Participant gender:
Summary
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.