NCT05206773

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 122 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 16 Years
• Healthy volunteers: Not accepted

Summary

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo-controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months. * Study visits will take place approximately every 3 months. * The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for an additional 12 months or until the Common Study End of Treatment Day (CSEOTD).

Detailed description

Double blind period: the total duration will be up to approximately of 14 months (1 month of screening 12 month of treatment period, and a possible follow-up period of 1 month if no participation in the open label extension period) Open-label extension period: the total duration will be approximately of 46 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 33 months), and 1 month of follow-up period)

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Venglustat (GZ402671)	Pharmaceutical form: Tablet Route of administration: Oral
DRUG	Placebo	Pharmaceutical form: Tablet Route of administration: Oral

Timeline

• Start date: 2022-03-11
• Primary completion: 2025-12-17
• Completion: 2027-01-04
• First posted: 2022-01-25
• Last updated: 2026-02-04

Locations

58 sites across 21 countries: United States, Argentina, Australia, Austria, Brazil, Canada, China, Denmark, Finland, France, Germany, Greece, Italy, Japan, Mexico, Norway, Poland, Romania, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study