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

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: Fabry Disease

In brief

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).

Key facts

Study ID

NCT05206773

Run by

Sanofi

People needed

122

Starts

2022-03-11

Expected to finish

2027-01-04

Last updated by the study team

2026-02-04

Who can join

Age: 16 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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 may not qualify 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.

Where it is running

• UCLA Medical Center_Investigational Site Number: 8400006 — Los Angeles, California, United States
• University of California Irvine Medical Center- Site Number : 8400019 — Orange, California, United States
• Advent Health Orlando_Investigational Site Number: 8400008 — Orlando, Florida, United States
• Emory Genetics- Site Number : 8400010 — Atlanta, Georgia, United States
• Westchester Medical Center Healthcare Corporation- Site Number : 8400001 — Hawthorne, New York, United States
• Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013 — Cincinnati, Ohio, United States
• Cleveland Clinic Site Number : 8400016 — Cleveland, Ohio, United States
• Children's Hospital Of Pittsburgh- Site Number : 8400009 — Pittsburgh, Pennsylvania, United States
• Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012 — Dallas, Texas, United States
• University Of Utah Health Sciences Center- Site Number : 8400005 — Salt Lake City, Utah, United States
• Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004 — Fairfax, Virginia, United States
• Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002 — La Rioja, Argentina
• Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003 — Quilmes, Argentina
• Investigational Site Number : 0360001 — Parkville, Victoria, Australia
• Investigational Site Number: 0400001 — Vienna, Austria
• Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001 — Porto Alegre, Rio Grande do Sul, Brazil
• Instituto de Genética e Erros Inatos do Metabolismo - IGEIM- Site Number : 0760002 — São Paulo, Brazil
• M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003 — Calgary, Alberta, Canada
• Medicine Dalhousie University_Investigational Site Number : 1240001 — Halifax, Nova Scotia, Canada
• University Health Network_Investigational Site Number : 1240005 — Toronto, Ontario, Canada
• No.8, Xishiku Street, Xicheng District_Site Number: 1560001 — Beijing, China
• No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003 — Shanghai, China
• No.85 South Jiefang road, Yingze District_Site Number: 1560004 — Taiyuan, China
• Investigational Site Number : 1560006 — Zhengzhou, China
• Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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