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

ClinicalTrials.gov ID: NCT05280548

Public ClinicalTrials.gov record NCT05280548. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

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ClinicalTrials.gov public records Last synced Oct 21, 2026, 5:13 AM EDT

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Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID: NCT05280548
Recruitment status: Active, not recruiting
Study type: Interventional
Phase: Phase 3
Lead sponsor: Sanofi; Industry
Enrollment: 104 participants

Conditions and interventions

Conditions

Fabry Disease

Interventions

Drug

Eligibility (public fields only)

Age range: 18 Years to 65 Years
Sex: All
Healthy volunteers: Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date: May 3, 2022 (Actual)
Primary completion: May 22, 2026 (Actual)
Completion: Dec 6, 2027 (Estimated)
Last update posted: Jun 24, 2026

2022 – 2027

United States locations

U.S. sites: 8
U.S. states: 8
U.S. cities: 8

Facility	City	State	ZIP	Site status
University of Alabama -The Kirklin Clinic- Site Number : 8400010	Birmingham	Alabama	35233	—
University of California Los Angeles Medical Center- Site Number : 8400008	Los Angeles	California	90095	—
Emory University School of Medicine - Atlanta- Site Number : 8400009	Atlanta	Georgia	30322	—
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005	Chicago	Illinois	60611	—
Maryam Banikazemi, MD- Site Number : 8400001	Hawthorne	New York	10532	—
Renal Disease Research Institute- Site Number : 8400012	Dallas	Texas	75204	—
University of Utah Health Hospital- Site Number : 8400006	Salt Lake City	Utah	84132	—
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004	Fairfax	Virginia	22030	—

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 46 non-U.S. sites.

About this trial record page

What this page shows: Public field values for ClinicalTrials.gov record NCT05280548, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do: No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from: Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh: Last update posted Jun 24, 2026 · Synced Oct 21, 2026

Open the official record

The complete public registry record, listed eligibility criteria, and available contact information for NCT05280548 live on ClinicalTrials.gov.

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