New pill could protect hearts in rare genetic disease

NCT ID NCT05280548

Ongoing Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

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.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

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

More trials for these conditions

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