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A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Adult Participants With Fabry Disease

About

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 adult participants 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.

Primary Purpose
Treatment
Study Type
Interventional
Phase
Phase III

Eligibility

Gender
All
Healthy Volunteers
No
Minimum Age
18 Years
Maximum Age
N/A

Inclusion Criteria:

  • Male and female adult patients 18 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.
  • Elevated plasma globotriaosylsphingosine (lysoGL3) at screening, or a previous biopsy of any organ that shows deposition of GL3.
  • 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.
  • 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, major cardiovascular surgery, or kidney transplantation.
  • History of ongoing clinically significant cardiac arrhythmia or prior or ongoing treatment for the above.
  • 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.
  • 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.
  • 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 CYP3A per FDA classification within 14 days or 5 halflives, 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.

Join this Trial

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Study Stats
Protocol No.
21-001919
Category
Nephrology
Contact
Anita Mkrttchyan
Location
  • UCLA Westwood
For Providers
NCT No.
NCT05206773
For detailed technical eligibility, visit ClinicalTrials.gov.