Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
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Phase
Phase 3
Status
Active, not recruiting
Study type
Interventional
Design
Randomized · Quadruple · Treatment
Enrollment
43actual
Sites
22
Countries
Argentina, Canada, China +8
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2022-04-18 | actual | When the study began enrolling |
| Primary completion | 2025-10-02 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-10-30 | estimated | The whole study's end, after follow-up |
| First posted | 2022-02-03 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-13 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Gaucher's Disease Type III
Interventions
- Drug: Venglustat
- Drug: imiglucerase — also filed as Cerezyme®
Primary outcomes
what the primary-completion date above is the date OFChange in Scale for Assessment and Rating of Ataxia (SARA) modified total score
measured From baseline to Week 52
Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score
measured From baseline to Week 52
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