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
Study of the Effects of an Investigational Medication on Pain in Fabry Disease
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 an additional 12 months or until the Common Study End of Treatment Day (CSEOTD).
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
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Age: 16 years or above
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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.
The purpose of this study is to investigate the effects of an investigational medication on neuropathic and abdominal pain in individuals with Fabry disease. This study involves participants who are 16 years of age or older and have either never been treated for Fabry disease or have not received treatment for at least six months.
Participants in this study will be randomly assigned to receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine. The study is double-blind, meaning neither the participants nor the researchers know who is receiving the investigational medication or the placebo. After the initial 12-month treatment period, participants may continue to receive the investigational medication in an open-label extension phase.
- Who can participate: Participants must be 16 years of age or older and have a confirmed diagnosis of Fabry disease with clinical symptoms. They should be treatment-naïve or not have received treatment for Fabry disease for at least 6 months. Key criteria include a weight of at least 30 kg and an average symptom score of 3 or higher on a specific symptom scale.
- Study details: Participants will be randomly assigned to receive either the investigational medication or a placebo. The study is double-blind, meaning neither participants nor researchers know who receives which treatment. After the double-blind period, participants may enter an open-label phase where they receive the investigational medication.
- Study timelines: The study will last 14 months.
