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

NCT05280548 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 104

Last updated 2026-03-10

No results posted yet for this study

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.

Conditions

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Clinical Sciences & Operations Clinical Sciences and Operations · Sanofi

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-05-03
Primary Completion
2026-05-15
Completion
2027-12-06
FDA Drug
Yes

Countries

  • United States
  • Austria
  • Canada
  • China
  • Czechia
  • Denmark
  • France
  • Germany
  • Greece
  • Italy
  • Japan
  • Netherlands
  • Norway
  • Poland
  • South Korea
  • Spain
  • Taiwan
  • Turkey (Türkiye)
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05280548 on ClinicalTrials.gov