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New pill could protect hearts in rare genetic disease

NCT ID NCT05280548

Disease control 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study tests whether venglustat, an experimental oral drug, can slow heart thickening better than current standard treatments in 104 adults with Fabry disease. Participants are randomly assigned to venglustat or usual care (enzyme replacement or migalastat) for 18 months. The main goal is to measure changes in heart muscle mass using MRI.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
venglustat
What this could lead to
If successful, venglustat could offer a new oral treatment option to slow heart damage in Fabry disease, potentially improving long-term heart health.
What could go wrong
This is a mid-stage trial with only 104 participants, so results may not apply to all patients. Venglustat is still experimental and may not prove better than current therapies.

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

104 people

The number who actually took part.

Started

May 2022

Expected to finish

Dec 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

18 to 65 years

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 participants aged 18 to 65 with previously confirmed diagnosis of Fabry disease and a history of clinical symptoms of Fabry disease. * Participants may be receiving treatment with agalsidase alfa, agalsidase beta, or migalastat, or may be untreated. * Left ventricular hypertrophy. * Contraception for male or female participants: not pregnant or breastfeeding; no sperm donating for male participant. * 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, major cardiovascular surgery or kidney transplantation. * History of seizures currently requiring treatment. * Underlying medical condition that may cause or contribute to left ventricular hypertrophy. * Asymmetric hypertrophy by cardiac MRI at screening if considered by central reader to be not related to Fabry disease. * Advanced cardiac fibrosis, defined as significant late gadolinium enhancement affecting 3 or more segments involving \>50% of myocardial thickness on screening cardiac MRI. * 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. * Estimated glomerular filtration rate \<45 mL/min/1.73m2. * 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. * Patients with hepatitis C, HIV, or hepatitis B infection. * Positive SARS-CoV-2 virus test within 2 weeks of enrollment, or COVID-19 requiring hospitalization within 6 months of enrollment. * History of drug and/or alcohol abuse. * Moderate to severe hepatic impairment. * History of or active hepatobiliary disease. * Liver enzymes (alanine aminotransferase/aspartate aminotransferase) or total bilirubin \>2 times the upper limit of normal. * Strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4 within 14 days or 5 half-lives, whichever is longer, prior to randomization. * Known contraindication to undergoing MRI or known hypersensitivity to gadolinium-based contrast agents. 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

  • Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 8400005

    Chicago, Illinois, 60611, United States

  • Emory University School of Medicine - Atlanta- Site Number : 8400009

    Atlanta, Georgia, 30322, United States

  • Investigational Site Number : 0400001

    Graz, 8036, Austria

  • Investigational Site Number : 1240002

    Vancouver, British Columbia, V5Z 1M9, Canada

  • Investigational Site Number : 1240003

    Calgary, Alberta, T2M 0L6, Canada

  • Investigational Site Number : 1240005

    Toronto, Ontario, M5T 3H7, Canada

  • Investigational Site Number : 1240006

    Edmonton, Alberta, T6G 2B7, Canada

  • Investigational Site Number : 1560001

    Chengdu, 610041, China

  • Investigational Site Number : 1560002

    Beijing, 100034, China

  • Investigational Site Number : 1560003

    Shanghai, 200025, China

  • Investigational Site Number : 1560005

    Beijing, 100034, China

  • Investigational Site Number : 1560007

    Guangzhou, 510080, China

  • Investigational Site Number : 1580001

    Taipei, 104, Taiwan

  • Investigational Site Number : 1580003

    Taichung, 407, Taiwan

  • Investigational Site Number : 2030001

    Prague, 128 08, Czechia

  • Investigational Site Number : 2080001

    Copenhagen, 2100, Denmark

  • Investigational Site Number : 2500001

    Garches, 92380, France

  • Investigational Site Number : 2760001

    Würzburg, 97080, Germany

  • Investigational Site Number : 2760003

    Berlin, 10117, Germany

  • Investigational Site Number : 2760004

    Hochheim am Main, 65239, Germany

  • Investigational Site Number : 2760005

    Mainz, 55131, Germany

  • Investigational Site Number : 3000001

    Heraklion, 711 10, Greece

  • Investigational Site Number : 3000002

    Athens, 115 27, Greece

  • Investigational Site Number : 3000003

    Athens, 124 62, Greece

  • Investigational Site Number : 3800001

    Milan, Lombardy, 20122, Italy

  • Investigational Site Number : 3800002

    Naples, Napoli, 80131, Italy

  • Investigational Site Number : 3800003

    Naples, Napoli, 80131, Italy

  • Investigational Site Number : 3800004

    Bologna, 40138, Italy

  • Investigational Site Number : 3920001

    Tokyo, 105-8461, Japan

  • Investigational Site Number : 3920002

    Sendai, Miyagi, 980-8574, Japan

  • Investigational Site Number : 3920003

    Kagoshima, Kagoshima-ken, 890-8520, Japan

  • Investigational Site Number : 3920004

    Fukuoka, 814-0180, Japan

  • Investigational Site Number : 3920005

    Kawasaki, Kanagawa, 215-0026, Japan

  • Investigational Site Number : 3920006

    Sapporo, Hokkaido, 060-8648, Japan

  • Investigational Site Number : 3920007

    Kagoshima, Kagoshima-ken, 890-0064, Japan

  • Investigational Site Number : 4100001

    Seoul, Seoul-teukbyeolsi, 03722, South Korea

  • Investigational Site Number : 4100002

    Yangsan, Gyeongsangnam-do, 50612, South Korea

  • Investigational Site Number : 5280001

    Amsterdam, 1105 AZ, Netherlands

  • Investigational Site Number : 5780001

    Bergen, 5021, Norway

  • Investigational Site Number : 6160001

    Krakow, 31-202, Poland

  • Investigational Site Number : 6160003

    Lodz, Lódzkie, 02-213, Poland

  • Investigational Site Number : 7240001

    Pontevedra, 36312, Spain

  • Investigational Site Number : 7240002

    Madrid, Madrid, Comunidad de, 28007, Spain

  • Investigational Site Number : 7240003

    Alicante, 03010, Spain

  • Investigational Site Number : 7920001

    Ankara, 06560, Turkey (Türkiye)

  • Investigational Site Number : 7920002

    Istanbul, 34098, Turkey (Türkiye)

  • Investigational Site Number : 7920003

    İzmit, 41000, Turkey (Türkiye)

  • Investigational Site Number : 8260001

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

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

    Fairfax, Virginia, 22030, United States

  • Maryam Banikazemi, MD- Site Number : 8400001

    Hawthorne, New York, 10532, United States

  • Renal Disease Research Institute- Site Number : 8400012

    Dallas, Texas, 75204, United States

  • University of Alabama -The Kirklin Clinic- Site Number : 8400010

    Birmingham, Alabama, 35233, United States

  • University of California Los Angeles Medical Center- Site Number : 8400008

    Los Angeles, California, 90095, United States

  • University of Utah Health Hospital- Site Number : 8400006

    Salt Lake City, Utah, 84132, United States

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