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Clinical Trials in the UK / NCT05206773
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 UK tracker
Sponsor
Sanofi
Phase
Phase 3
Started
2022-03-11
Last updated
2026-02-04

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)

Trial sites (58)

FacilityCityRegionStatus
Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham Alabama
UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles California
University of California Irvine Medical Center- Site Number : 8400019 Orange California
Advent Health Orlando_Investigational Site Number: 8400008 Orlando Florida
Emory Genetics- Site Number : 8400010 Atlanta Georgia
Westchester Medical Center Healthcare Corporation- Site Number : 8400001 Hawthorne New York
Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400013 Cincinnati Ohio
Cleveland Clinic Site Number : 8400016 Cleveland Ohio
Children's Hospital Of Pittsburgh- Site Number : 8400009 Pittsburgh Pennsylvania
Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012 Dallas Texas
University Of Utah Health Sciences Center- Site Number : 8400005 Salt Lake City Utah
Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004 Fairfax Virginia
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
Investigational Site Number: 0400001 Vienna Austria
Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001 Porto Alegre Rio Grande do Sul
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
Medicine Dalhousie University_Investigational Site Number : 1240001 Halifax Nova Scotia
University Health Network_Investigational Site Number : 1240005 Toronto Ontario
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
Investigational Site Number: 2080001 Copenhagen Denmark
Investigational Site Number: 2460001 Turku Finland
Investigational Site Number : 2500001 Garches France
ISphinCS GmbH_Investigational Site Number: 2760004 Hochheim am Main Germany
Investigational Site Number : 2760005 Mainz Germany
Investigational Site Number: 2760003 München Germany
Investigational Site Number: 2760001 Würzburg Germany
Investigational Site Number : 3000003 Athens Greece
University Hospital of Heraklion_Investigational Site Number: 3000001 Heraklion Greece
University Hospital of Ioannina_Investigational Site Number: 3000002 Ioannina Greece
IRCCS Policlinico di Sant'Orsola_Investigational Site Number : 3800005 Bologna Italy
Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002 Monza Italy
Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001 Naples Italy
Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003 Palermo Italy
Fondazione Policlinico Universitario_Investigational Site Number: 3800004 Roma Italy

+ 18 more sites — see the full list on the official registry below.

More Sanofi trials in the UK

Other trials for the same condition

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 UK