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A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

Testing Venglustat's effect on heart health in Fabry disease patients.

Recruiting
18-65 years
All
Phase 3

Fabry disease is a rare genetic disorder that affects the heart and other parts of the body. This study is looking at how a medicine called venglustat affects the heart in people with Fabry disease. Specifically, it examines the left ventricular mass index (LVMI), which measures the size of the heart's left pumping chamber. Participants will be randomly assigned to either take venglustat or continue with their usual treatment, like agalsidase alfa, agalsidase beta, or migalastat, for 18 months. Visits to the study site are needed every 3 to 6 months.

  • The study runs up to 4.4 years if participants join the long-term extension.
  • To join, participants must be 18 to 65 years old and have Fabry disease with heart enlargement.
  • Participants can't have certain health issues, like recent heart attacks or untreated mental health disorders.

Those in the study will help researchers learn more about how to treat heart issues in Fabry disease. Before joining, a signed consent form is required, and individuals must not have certain other health conditions.

Study details
    Fabry Disease

NCT05280548

Sanofi

23 November 2024

Step 1 Get in touch with the nearest study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer  to help accelerate the development of new treatments and to get notified about similar trials.

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