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
NCT ID: NCT05206773
Last Updated: 2025-07-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
122 participants
INTERVENTIONAL
2022-03-11
2027-01-04
Brief Summary
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* 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).
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Detailed Description
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Open-label extension period: the total duration will be approximately of 46 months (12 month of OLE treatment, additional OLE treatment until a common study end of treatment date (CSEOTD, approximately 33 months), and 1 month of follow-up period)
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Venglustat
Participant will receive venglustat dose once daily for 12 months
Venglustat (GZ402671)
Pharmaceutical form: Tablet Route of administration: Oral
Placebo
Participants will receive placebo once daily for 12 months
Placebo
Pharmaceutical form: Tablet Route of administration: Oral
Interventions
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Venglustat (GZ402671)
Pharmaceutical form: Tablet Route of administration: Oral
Placebo
Pharmaceutical form: Tablet Route of administration: Oral
Eligibility Criteria
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Inclusion Criteria
* 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
* 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.
16 Years
ALL
No
Sponsors
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Sanofi
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Sciences & Operations
Role: STUDY_DIRECTOR
Sanofi
Locations
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Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011
Birmingham, Alabama, United States
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
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
Napoli, , Italy
Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003
Palermo, , Italy
Fondazione Policlinico Universitario_Investigational Site Number: 3800004
Roma, , Italy
Investigational Site Number : 3920005
Kawasaki, Kanagawa, Japan
Fukuoka University Hospital_Investigational Site Number: 3920004
Fukuoka-shi, Fukuoka, , Japan
The Jikei University Hospital_Investigational Site Number: 3920003
Minato-ku, Tokyo, , Japan
Tohoku University Hospital_Investigational Site Number: 3920001
Sendai-shi, Miyagi, , Japan
Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001
Monterrey, Nuevo León, Mexico
Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_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 : 6160004
Rzeszów, , Poland
Investigational Site Number: 6160003
Wroclaw, , Poland
Institutul Clinic Fundeni_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 : 7920004
Malatya, , Turkey (Türkiye)
Investigational Site Number: 8260001
Cambridge, Cambridgeshire, United Kingdom
Investigational Site Number: 8260002
London, London, City of, United Kingdom
Investigational Site Number : 8260003
Salford, Manchester, United Kingdom
Countries
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Other Identifiers
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U1111-1256-9310
Identifier Type: REGISTRY
Identifier Source: secondary_id
2024-511990-31
Identifier Type: REGISTRY
Identifier Source: secondary_id
2021-002350-90
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
EFC17045
Identifier Type: -
Identifier Source: org_study_id
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