NCT05222906

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

A Phase 3, Multicenter, Multinational, Randomized, Double-blind, Double-dummy, Active-comparator Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (GD3) Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ERT)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 43 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Screening period: 45 days Double blind, double-dummy, primary analysis treatment period: 52 weeks Open label extended treatment period: minimum of 52 weeks due to a common study end of treatment date Follow up phone call: 30-37 days after end of treatment

Conditions

• Gaucher's Disease Type III

Interventions

Type	Name	Description
DRUG	Venglustat	tablet; oral
DRUG	imiglucerase	sterile lyophilized product; intravenous

Timeline

• Start date: 2022-04-18
• Primary completion: 2025-10-02
• Completion: 2026-10-30
• First posted: 2022-02-03
• Last updated: 2025-10-20

Locations

22 sites across 11 countries: United States, Argentina, Canada, China, France, Germany, Hungary, Italy, Japan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study