A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Investigating the Effect of an Investigational Medication on Pain in Fabry Disease

Not Recruiting
16 years or above
All
Phase 3
122 participants needed

Study Overview

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 participants ≥16 years of age 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.

Study Details

Double blind period: the total duration will be up to approximately of 14 months (1 month of screening 12 month of treatment period, and a possible follow-up period of 1 month if no participation in the open label extension period)

Open-label extension period: the total duration will be approximately of 46 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 33 months), and 1 month of follow-up period)

Eligibility Criteria

You may be eligible for this study if you meet the following criteria:

  • Conditions: Fabry Disease
  • Age: 16 years or above
  • Gender: All

Inclusion Criteria:

  • Male and female adult patients 16 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.
  • 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.
  • Weight ≥30 Kg
  • 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, evidence of left ventricular hypertrophy and/or cardiac fibrosis, major cardiovascular surgery, or kidney transplantation.
  • 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.
  • 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, or systolic BP >=150 or diastolic BP >=100 at screening.
  • Estimated glomerular filtration rate <60 mL/min/1.73m².
  • Urine protein to creatinine ratio >= 1 g/g at screening.
  • 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).
  • 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 3A within 14 days or 5 half-lives, 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.

Updated on 04 Apr 2025. Study ID: NCT05206773

This study investigates the effects of an investigational medication on neuropathic and abdominal pain in people with Fabry disease. Fabry disease is a genetic disorder that affects the body's ability to break down a specific type of fat, leading to pain and other symptoms. The study is for participants aged 16 and older who have Fabry disease and have not received treatment for at least 6 months.

Participants will be randomly assigned to receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine. The study includes a double-blind period where neither the participants nor the researchers know who is receiving the investigational medication. After this period, there is an open-label extension where all participants may receive the investigational medication.

  • Who can participate: Adults aged 16 and older with Fabry disease who have not been treated for at least 6 months may participate. Key eligibility includes a history of Fabry disease symptoms and an average pain score of 3 or higher. Participants must not have certain medical conditions such as significant heart issues or infections.
  • Study details: Participants will be assigned to either the investigational medication or a placebo group in a double-blind manner. After completing this phase, they may enter an open-label extension where they receive the investigational medication. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine.
  • Study Timelines and Visits: The study will last 14 months. The study requires 5 visits.