for people ages 16 years and up (full criteria)
at Orange, California and other locations
study started
completion around



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.

Official Title

A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 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 31 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 18 months), and 1 month of follow-up period)


Fabry Disease, Abdominal Pain, Venglustat, Venglustat (GZ402671)


You can join if…

Open to people ages 16 years and up

  • 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.

You CAN'T join if...

  • 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.


  • University of California Irvine Medical Center Site Number : 8400019 accepting new patients
    Orange California 92868 United States
  • UCLA Medical Center_Investigational Site Number: 8400006 accepting new patients
    Los Angeles California 90095 United States


accepting new patients
Start Date
Completion Date
Phase 3 Fabry Disease Research Study
Study Type
Expecting 114 study participants
Last Updated