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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
EU CTIS ID: 2024-511990-31-00
What this study is testing
To assess the effect of venglustat on neuropathic or abdominal pain in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months prior to screening
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Male and female adult patients 16 year of age or older with a confirmed diagnosis 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.
You likely can't join if
- Any manifestations of Fabry disease that preclude placebo administration.
- 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.
See the full eligibility criteria
- Male and female adult patients 16 year of age or older with a confirmed diagnosis 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.
- Any manifestations of Fabry disease that preclude placebo administration.
- 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.
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Argentina
- Switzerland
- Australia
- Turkey
- India
- China
- Japan
- United Kingdom
- Canada
- Brazil
- Mexico
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Argentina; Switzerland; Australia; Turkey; India; China and 6 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.