Status:
ACTIVE_NOT_RECRUITING
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
Lead Sponsor:
Sanofi
Conditions:
Fabry Disease
Eligibility:
All Genders
16+ years
Phase:
PHASE3
Brief 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 ...
Detailed Description
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 t...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 11 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 4 2027
Estimated Enrollment :
122 Patients enrolled
Trial Details
Trial ID
NCT05206773
Start Date
March 11 2022
End Date
January 4 2027
Last Update
July 20 2025
Active Locations (58)
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1
Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
Birmingham, Alabama, United States, 35233
2
UCLA Medical Center_Investigational Site Number: 8400006
Los Angeles, California, United States, 90095
3
University of California Irvine Medical Center- Site Number : 8400019
Orange, California, United States, 92868
4
Advent Health Orlando_Investigational Site Number: 8400008
Orlando, Florida, United States, 32804