Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Summary

This is a parallel arm, Phase 3, double-blind, double-dummy, active-comparator, 2 arm study to evaluate the efficacy and safety of daily oral venglustat versus intravenous Cerezyme infusions every two weeks for improvement or stabilization of the neurological manifestations and maintenance of systemic disease stability in participants aged ≥12 and \<18 years and adult patients with Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

Timeline

Start

2022-04-18

Primary completion

2025-10-02

Completion

2026-10-30

Outcome

Met primary endpoint

release “Positive results from the LEAP2MONO phase 3 study (clinical study identifier: NCT05222906 ) demonstrated that venglustat met the primary” sanofi.com ↗

Drugs

Indications