Overview

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.