Ireland
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Active, not recruiting Phase 3

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

NCT05206773 · tracked via the Priya Life Science USA tracker
Sponsor
Phase
Phase 3
Started
2022-03-11
Last updated
2026-10-13

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

Venglustat (GZ402671) →Placebo

Venglustat (GZ402671): Pharmaceutical form: Tablet Route of administration: Oral

Placebo: Pharmaceutical form: Tablet Route of administration: Oral

Study 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 an additional 12 months or until the Common Study End of Treatment Day (CSEOTD).

Eligibility

Sex
ALL
Min age
16 Years
Max age
—
Healthy volunteers
No
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. * 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.

Primary outcome measure(s)

  • Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) — From baseline to 6 months
  • Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome (FD-PRO) items (neuropathic pain in upper extremities, neuropathic pain in lower extremities, or abdominal pain) — From baseline to 12 months

Trial sites (50)

FacilityCityRegionStatus
University of Alabama -The Kirklin Clinic- Site Number : 8400011 Birmingham Alabama
David Geffen School of Medicine at UCLA- Site Number : 8400006 Los Angeles California
University of California Irvine Medical Center- Site Number : 8400019 Orange California
AdventHealth Orlando- Site Number : 8400008 Orlando Florida
Emory University School of Medicine - Atlanta- Site Number : 8400010 Atlanta Georgia
WMCHealth Advanced Physician Services- Site Number : 8400001 Hawthorne New York
Cincinnati Children's Hospital Medical Center- Site Number : 8400013 Cincinnati Ohio
UPMC Montefiore- Site Number : 8400009 Pittsburgh Pennsylvania
Renal Disease Research Institute - Dallas - Live Oak Street- Site Number : 8400012 Dallas Texas
University of Utah Health Hospital- Site Number : 8400005 Salt Lake City Utah
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004 Fairfax Virginia
Investigational Site Number : 0320001 Pergamino Buenos Aires
Investigational Site Number : 0320003 Quilmes Buenos Aires
Investigational Site Number : 0320002 La Rioja Argentina
Investigational Site Number : 0360001 Parkville Victoria
Investigational Site Number : 0400001 Vienna Austria
Hospital de Clinicas de Porto Alegre- Site Number : 0760001 Porto Alegre Rio Grande do Sul
Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 0760002 São Paulo Brazil
Investigational Site Number : 1240003 Calgary Alberta
Investigational Site Number : 1240001 Halifax Nova Scotia
Investigational Site Number : 1240005 Toronto Ontario
Investigational Site Number : 1560001 Beijing China
Investigational Site Number : 1560003 Shanghai China
Investigational Site Number : 1560004 Taiyuan China
Investigational Site Number : 1560006 Zhengzhou China
Investigational Site Number : 2080001 Copenhagen Denmark
Investigational Site Number : 2460001 Turku Finland
Investigational Site Number : 2500001 Garches France
Investigational Site Number : 2760003 Munich Germany
Investigational Site Number : 2760001 Würzburg Germany
Investigational Site Number : 3000003 Athens Greece
Investigational Site Number : 3000002 Ioannina Greece
Investigational Site Number : 3800001 Naples Napoli
Investigational Site Number : 3800004 Rome Roma
Investigational Site Number : 3800005 Bologna Italy
Investigational Site Number : 3920004 Fukuoka Japan
Investigational Site Number : 3920003 Tokyo Japan
Investigational Site Number : 4840001 Monterrey Nuevo León
Investigational Site Number : 4840002 Mexico City Mexico
Investigational Site Number : 5780001 Bergen Norway

+ 10 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05206773 on ClinicalTrials.gov ↗ ← All trials in the USA