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Clinical Trials in the UK / NCT05280548
Active, not recruiting Phase 3

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

NCT05280548 · tracked via the Priya Life Science UK tracker
Sponsor
Sanofi
Phase
Phase 3
Started
2022-05-03
Last updated
2026-06-24

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

Venglustat (GZ402671)Agalsidase alfaAgalsidase beta (GZ419828)Migalastat

Venglustat (GZ402671): Tablet; Oral

Agalsidase alfa: Concentrate for solution for infusion; IV infusion

Agalsidase beta (GZ419828): Powder for concentrate for solution for infusion; IV infusion

Migalastat: Hard capsules; Oral

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria: * Male and female participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease. * Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated. * Left ventricular hypertrophy. * Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant. * A signed informed consent must be provided prior to any study-related procedures. Exclusion Criteria: * History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery or kidney transplantation. * History of seizures currently requiring treatment. * Underlying medical condition that may cause or contribute to left ventricular hypertrophy. * Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease. * Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving \>50% of myocardial thickness on screening cardiac MRI. * History of clinically significant cardiac arrhythmia. Atrial fibrillation that is well controlled on a stable medical regimen for at least 12 months is not an exclusion if the CHA2DS2-VASc score is 0 for males or 1 for females. * Estimated glomerular filtration rate \<45 mL/min/1.73m2. * Presence of severe depression as measured by Beck's Depression Inventory (BDI)-II \>28 and/or a history of an untreated, unstable major affective disorder within 1 year of the screening visit. * Patients with hepatitis C, HIV, or hepatitis B infection. * Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment. * History of drug and/or alcohol abuse. * Moderate to severe hepatic impairment. * History of or active hepatobiliary disease. * Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin \>2 times the upper limit of normal. * Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization. * Known contraindication to undergoing MRI or known hypersensitivity to gadolinium-based contrast agents. The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Primary outcome measure(s)

Trial sites (54)

FacilityCityRegionStatus
University of Alabama -The Kirklin Clinic- Site Number : 8400010 Birmingham Alabama
University of California Los Angeles Medical Center- Site Number : 8400008 Los Angeles California
Emory University School of Medicine - Atlanta- Site Number : 8400009 Atlanta Georgia
Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005 Chicago Illinois
Maryam Banikazemi, MD- Site Number : 8400001 Hawthorne New York
Renal Disease Research Institute- Site Number : 8400012 Dallas Texas
University of Utah Health Hospital- Site Number : 8400006 Salt Lake City Utah
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004 Fairfax Virginia
Investigational Site Number : 0400001 Graz Austria
Investigational Site Number : 1240003 Calgary Alberta
Investigational Site Number : 1240006 Edmonton Alberta
Investigational Site Number : 1240002 Vancouver British Columbia
Investigational Site Number : 1240005 Toronto Ontario
Investigational Site Number : 1560002 Beijing China
Investigational Site Number : 1560005 Beijing China
Investigational Site Number : 1560001 Chengdu China
Investigational Site Number : 1560007 Guangzhou China
Investigational Site Number : 1560003 Shanghai China
Investigational Site Number : 2030001 Prague Czechia
Investigational Site Number : 2080001 Copenhagen Denmark
Investigational Site Number : 2500001 Garches France
Investigational Site Number : 2760003 Berlin Germany
Investigational Site Number : 2760004 Hochheim am Main Germany
Investigational Site Number : 2760005 Mainz Germany
Investigational Site Number : 2760001 Würzburg Germany
Investigational Site Number : 3000002 Athens Greece
Investigational Site Number : 3000003 Athens Greece
Investigational Site Number : 3000001 Heraklion Greece
Investigational Site Number : 3800001 Milan Lombardy
Investigational Site Number : 3800002 Naples Napoli
Investigational Site Number : 3800003 Naples Napoli
Investigational Site Number : 3800004 Bologna Italy
Investigational Site Number : 3920006 Sapporo Hokkaido
Investigational Site Number : 3920007 Kagoshima Kagoshima-ken
Investigational Site Number : 3920003 Kagoshima Kagoshima-ken
Investigational Site Number : 3920005 Kawasaki Kanagawa
Investigational Site Number : 3920002 Sendai Miyagi
Investigational Site Number : 3920004 Fukuoka Japan
Investigational Site Number : 3920001 Tokyo Japan
Investigational Site Number : 5280001 Amsterdam Netherlands

+ 14 more sites — see the full list on the official registry below.

On this site

📄 Galafold (migalastat) drug profile →

More Sanofi trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05280548 on ClinicalTrials.gov ↗ ← All trials in the UK