A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease

• Status: Not yet recruiting
• Trial end date: 2025-12-26
• Target enrollment: 0
• Participant gender: All

Summary

This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in adult participants with Fabry disease who are treatment-naïve or untreated for at least 6 months.

• Study visits will take place approximately every 3 months.

• The double-blind period will be followed by an open-label extension (OLE) during which participants who have completed the double-blind period will be treated with venglustat for up to an additional 12 months.

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

Details

• Lead Sponsor: Genzyme, a Sanofi Company

Criteria

Inclusion Criteria:

• Male and female adult patients 18 year of age or older, who have had a previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease

• Patients who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening.

• Elevated plasma globotriaosylsphingosine (lysoGL3) at screening, or a previous biopsy of any organ that shows deposition of GL3.

• Average score of ≥3 (0=no symptom, 10=symptom as bad as you can imagine) on the participant-defined most-bothersome symptom (among neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain), as measured by the Fabry Disease Patient-Reported Outcome (FD-PRO) at screening.

• Contraception (with double contraception methods) for male and female participants; not pregnant or breastfeeding for female participants; no sperm donation for male participants.

• A signed informed consent must be provided prior to any study-related procedures.

Exclusion Criteria:

• Any manifestations of Fabry disease that preclude placebo administration.

• History of transient ischemic attack, stroke, myocardial infarction, heart failure, major cardiovascular surgery, or kidney transplantation.

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

• Patients with hepatitis C, HIV, or hepatitis B infection.

• Neuropathic pain in upper or lower extremities, or abdominal pain not related to Fabry disease.

• History of seizures currently requiring treatment.

• Uncontrolled hypertension over the past 12 months prior to screening.

• 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.

• Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID 19 requiring hospitalization within 6 months of enrollment.

• Moderate to severe hepatic impairment.

• History of drug and/or alcohol abuse.

• History of or active hepatobiliary disease.

• Liver enzymes (alanine aminotransferase (ALT)/aspartate aminotransferase (AST)) or total bilirubin >2 times the upper limit of normal (ULN).

• Cortical cataract ≥one-quarter of the lens circumference (Grade cortical cataract [COR-2]) or a posterior subcapsular cataract ≥2 mm (Grade posterior subcapsular cataract [PSC-2]) at the time of screening.

• Chronic regimen of potentially cataractogenic medications.

• Initiation of chronic treatment for pain, or change in pain medication regimen, within 3 months prior to randomization.

• Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A per FDA classification within 14 days or 5 halflives, whichever is longer, prior to randomization.

The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.