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Hope for fabry pain: new drug trial targets nerve and belly discomfort

NCT ID NCT05206773

Symptom relief Sponsor: Sanofi Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether venglustat can reduce nerve pain in the arms and legs, and belly pain, in people with Fabry disease. Participants are 16 or older and have not had Fabry treatment for at least 6 months. The trial lasts 12 months, with visits every 3 months, and compares venglustat to a placebo.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

122 people

The number who actually took part.

Started

Mar 2022

Expected to finish

Jan 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

16 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Fabry disease

Contacts and locations

Locations

  • Advent Health Orlando_Investigational Site Number: 8400008

    Orlando, Florida, 32804, United States

  • Azienda Ospedaliera Universitaria "Federico II", U.O. di Nefrologia- Diparimento di Sanità Pubblica_Investigational Site Number: 3800001

    Naples, 80138, Italy

  • Azienda Ospedaliera Universitaria_Investigational Site Number: 3800003

    Palermo, 90127, Italy

  • Children's Hospital Of Pittsburgh- Site Number : 8400009

    Pittsburgh, Pennsylvania, 15213, United States

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

    Cincinnati, Ohio, 45229-3026, United States

  • Cleveland Clinic Site Number : 8400016

    Cleveland, Ohio, 44195, United States

  • Emory Genetics- Site Number : 8400010

    Atlanta, Georgia, 30322, United States

  • Fondazione IRCCS San Gerardo dei Tintori, S.C. Nefrologia - Clinica Nefrologica_Investigational Site Number: 3800002

    Monza, 20052, Italy

  • Fondazione Policlinico Universitario_Investigational Site Number: 3800004

    Roma, 00168, Italy

  • Fukuoka University Hospital_Investigational Site Number: 3920004

    Fukuoka-shi, Fukuoka, 814-0180, Japan

  • Fundacion Cori para la Investigación y Prevención del Cancer_Investigational Site Number: 0320002

    La Rioja, F5300, Argentina

  • Hospital Universitario "Dr. José Eleuterio González" Departamento de Genética Centro Universitario contra el cáncer_Investigational Site Number: 4840001

    Monterrey, Nuevo León, 64460, Mexico

  • Hospital de Clínicas de Porto Alegre_Investigational Site Number: 0760001

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • IRCCS Policlinico di Sant'Orsola_Investigational Site Number : 3800005

    Bologna, 40138, Italy

  • ISphinCS GmbH_Investigational Site Number: 2760004

    Hochheim am Main, 65239, Germany

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

    São Paulo, 04020-041, Brazil

  • Instituto de Investigaciones Clínicas Quilmes (IICQ) SRL_Investigational Site Number: 0320003

    Quilmes, B1878GEG, Argentina

  • Institutul Clinic Fundeni_Investigational Site Number: 6420001

    Bucharest, 22328, Romania

  • Investigational Site Number : 0360001

    Parkville, Victoria, 3050, Australia

  • Investigational Site Number : 1560006

    Zhengzhou, 450052, China

  • Investigational Site Number : 2500001

    Garches, 92380, France

  • Investigational Site Number : 2760005

    Mainz, 55131, Germany

  • Investigational Site Number : 3000003

    Athens, 115 28, Greece

  • Investigational Site Number : 3920005

    Kawasaki, Kanagawa, 215-0026, Japan

  • Investigational Site Number : 6160002

    Poznan, Greater Poland Voivodeship, 60-780, Poland

  • Investigational Site Number : 6160004

    Rzeszów, 35-301, Poland

  • Investigational Site Number : 7560001

    Zurich, 8091, Switzerland

  • Investigational Site Number : 7920001

    Ankara, 06560, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    İzmit, 41000, Turkey (Türkiye)

  • Investigational Site Number : 7920004

    Malatya, 44280, Turkey (Türkiye)

  • Investigational Site Number : 8260003

    Salford, Manchester, M6 8HD, United Kingdom

  • Investigational Site Number: 0400001

    Vienna, 1090, Austria

  • Investigational Site Number: 2080001

    Copenhagen, 2100, Denmark

  • Investigational Site Number: 2460001

    Turku, FI-20520, Finland

  • Investigational Site Number: 2760001

    Würzburg, 97080, Germany

  • Investigational Site Number: 2760003

    München, 80336, Germany

  • Investigational Site Number: 5780001

    Bergen, 5021, Norway

  • Investigational Site Number: 6160003

    Wroclaw, 50-556, Poland

  • Investigational Site Number: 8260001

    Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom

  • Investigational Site Number: 8260002

    London, London, City of, NW3 2QG, United Kingdom

  • Lysosomal and Rare Disorders Research and Treatment Center_Investigational Site Number: 8400004

    Fairfax, Virginia, 22030, United States

  • M.A.G.I.C Calgary LTD_Investigational Site Number: 1240003

    Calgary, Alberta, T2E7Z4, Canada

  • Medicine Dalhousie University_Investigational Site Number : 1240001

    Halifax, Nova Scotia, B3H 2Y9, Canada

  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011

    Birmingham, Alabama, 35233, United States

  • No.197,2nd Ruijin road, Huangpu district_Site Number: 1560003

    Shanghai, 200010, China

  • No.8, Xishiku Street, Xicheng District_Site Number: 1560001

    Beijing, 100034, China

  • No.85 South Jiefang road, Yingze District_Site Number: 1560004

    Taiyuan, 030001, China

  • Odette del Carmen DIAZ-AVENDAÑO Clinstile, S.A. de C.V. Durango_Investigational Site Number: 4840002

    Mexico City, 06700, Mexico

  • Renal Disease Research Institute, An affiliate of: Dallas Nephrology Associates_Investigational Site Number: 8400012

    Dallas, Texas, 75235, United States

  • The Jikei University Hospital_Investigational Site Number: 3920003

    Minato-ku, Tokyo, 105-8471, Japan

  • Tohoku University Hospital_Investigational Site Number: 3920001

    Sendai-shi, Miyagi, 980-8574, Japan

  • UCLA Medical Center_Investigational Site Number: 8400006

    Los Angeles, California, 90095, United States

  • University Health Network_Investigational Site Number : 1240005

    Toronto, Ontario, M5T 3L9, Canada

  • University Hospital of Heraklion_Investigational Site Number: 3000001

    Heraklion, 711 10, Greece

  • University Hospital of Ioannina_Investigational Site Number: 3000002

    Ioannina, 455 00, Greece

  • University Of Utah Health Sciences Center- Site Number : 8400005

    Salt Lake City, Utah, 84132, United States

  • University of California Irvine Medical Center- Site Number : 8400019

    Orange, California, 92868, United States

  • Westchester Medical Center Healthcare Corporation- Site Number : 8400001

    Hawthorne, New York, 10532, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.