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

Conditions: Fabry Disease

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 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.

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:
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.