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

• Status: Not yet recruiting
• Trial end date: 2026-09-01
• Target enrollment: 0
• Participant gender: All

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 open-label extension (OLE) to receive venglustat for up to an additional 18 months.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Genzyme, a Sanofi Company

Criteria

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.

• Advanced cardiac fibrosis, defined as significant late gadolinium enhancement beyond the basal inferolateral left ventricular wall on cardiac MRI.

• History of ongoing clinically significant cardiac arrythmia, prior or ongoing treatment for the above.

• Estimated glomerular filtration rate <60 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 per Food and Drug Administration (FDA) classification within 14 days or 5 halflives, 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.