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ACTIVE NOT RECRUITING

NCT05206773

PHASE3

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

Sponsor: Sanofi

View on ClinicalTrials.gov

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).

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

Key Details

Gender

All

Age Range

16 Years - Any

Study Type

INTERVENTIONAL

Enrollment

122

Start Date

2022-03-11

Completion Date

2027-01-04

Last Updated

2026-10-13

Healthy Volunteers

Conditions

Fabry Disease

Interventions

DRUG

Venglustat (GZ402671)

Pharmaceutical form: Tablet Route of administration: Oral

DRUG

Placebo

Pharmaceutical form: Tablet Route of administration: Oral

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.

Locations (50)

University of Alabama -The Kirklin Clinic- Site Number : 8400011

Birmingham, Alabama, United States

David Geffen School of Medicine at UCLA- Site Number : 8400006

Los Angeles, California, United States

University of California Irvine Medical Center- Site Number : 8400019

Orange, California, United States

AdventHealth Orlando- Site Number : 8400008

Orlando, Florida, United States

Emory University School of Medicine - Atlanta- Site Number : 8400010

Atlanta, Georgia, United States

WMCHealth Advanced Physician Services- Site Number : 8400001

Hawthorne, New York, United States

Cincinnati Children's Hospital Medical Center- Site Number : 8400013

Cincinnati, Ohio, United States

UPMC Montefiore- Site Number : 8400009

Pittsburgh, Pennsylvania, United States

Renal Disease Research Institute - Dallas - Live Oak Street- Site Number : 8400012

Dallas, Texas, United States

University of Utah Health Hospital- Site Number : 8400005

Salt Lake City, Utah, United States

Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)- Site Number : 8400004

Fairfax, Virginia, United States

Investigational Site Number : 0320001

Pergamino, Buenos Aires, Argentina

Investigational Site Number : 0320003

Quilmes, Buenos Aires, Argentina

Investigational Site Number : 0320002

La Rioja, Argentina

Investigational Site Number : 0360001

Parkville, Victoria, Australia

Investigational Site Number : 0400001

Vienna, Austria

Hospital de Clinicas de Porto Alegre- Site Number : 0760001

Porto Alegre, Rio Grande do Sul, Brazil

Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 0760002

São Paulo, Brazil

Investigational Site Number : 1240003

Calgary, Alberta, Canada

Investigational Site Number : 1240001

Halifax, Nova Scotia, Canada

Investigational Site Number : 1240005

Toronto, Ontario, Canada

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, Italy

Investigational Site Number : 3800004

Rome, Roma, Italy

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, Mexico

Investigational Site Number : 4840002

Mexico City, Mexico

Investigational Site Number : 5780001

Bergen, Norway

Investigational Site Number : 6160002

Poznan, Greater Poland Voivodeship, Poland

Investigational Site Number : 6160003

Wroclaw, Lower Silesian Voivodeship, Poland

Investigational Site Number : 6160004

Rzeszów, Podkarpackie Voivodeship, Poland

Investigational Site Number : 6420001

Bucharest, Romania

Investigational Site Number : 7560001

Zurich, Switzerland

Investigational Site Number : 7920001

Ankara, Turkey (Türkiye)

Investigational Site Number : 7920002

İzmit, Turkey (Türkiye)

Investigational Site Number : 8260001

Cambridge, Cambridgeshire, United Kingdom

Investigational Site Number : 8260002

London, England, United Kingdom

Investigational Site Number : 8260003

Salford, Manchester, United Kingdom