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

  • STATUS
    Recruiting
  • End date
    Oct 6, 2025
  • participants needed
    114
  • sponsor
    Sanofi
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Updated on 3 December 2024
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abdominal pain
neuropathic pain

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.

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 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)

Details
Condition Fabry Disease
Age 16years or above
Treatment Placebo, venglustat (GZ402671)
Clinical Study IdentifierNCT05206773
SponsorSanofi
Last Modified on3 December 2024

Eligibility

 Yes No Not Sure

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
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
Urine protein to creatinine ratio >= 1 g/g at screening
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
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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