NCT05280548

A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

A Randomized, Open-label, Parallel-group, 18-month Phase 3 Study to Evaluate the Effect of Venglustat Compared With Usual Standard of Care on Left Ventricular Mass Index in Participants With Fabry Disease and Left Ventricular Hypertrophy

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 104 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy. * Study visits will take place approximately every 3 to 6 months * Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.

Detailed description

Randomized period: the total duration will be up to approximately of 20 months (1 month screening 18 months of treatment and a possible follow-up period of 1 month if no participation in the long-term extension period) Long-term extension period: the total duration will be from minimum 19 months (18 months of treatment and 1 month of follow-up period) to maximum 46 months (45 months of treatment and 1 month of follow-up period). The maximum total study duration is approximately 5.3 years

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	Venglustat (GZ402671)	Tablet; Oral
DRUG	Agalsidase alfa	Concentrate for solution for infusion; IV infusion
DRUG	Agalsidase beta (GZ419828)	Powder for concentrate for solution for infusion; IV infusion
DRUG	Migalastat	Hard capsules; Oral

Timeline

• Start date: 2022-05-03
• Primary completion: 2026-05-15
• Completion: 2027-12-06
• First posted: 2022-03-15
• Last updated: 2026-03-10

Locations

54 sites across 19 countries: United States, Austria, Canada, China, Czechia, Denmark, France, Germany, Greece, Italy, Japan, Netherlands, Norway, Poland, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study