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Study Details

Testing a New Drug called Venglustat Compared to the Standard of Care on the Heart in Adults with Fabry Disease (The CARAT (EFC16158) Study)

(IRB#: IRB_00167895)

This study will test a new drug called Venglustat compared to the usual standard of care treatment in adults with Fabry Disease. This study is for people who have heart problems (enlargement of the heart's main pump (left ventricle)). Participants in this study will be randomized to be treated with Venglustat or one of the standard of care treatments. Later in the study, participants on the standard of care treatment can be switched to Venglustat. Participants will be in the study for 2 years and 2 months. There will be several clinic visits for the study and there may be the option for some home health visits. There will also be phone call visits.


  • All genders
  • Over 18 years old
  • Volunteers with special conditions
  •   In Person
  • Paid

Who can participate?

 Gender: All genders

  Age: Over 18 years old

  Volunteers: Volunteers with special conditions

   Location: In Person

Inclusion Criteria

  • Ages 18 to 65 years old
  • Confirmed diagnosis of Fabry disease
  • Diagnosed left ventricular hypertrophy (enlargement and thickening of the heart's main pump (left ventricle))
  • Willing to use specific birth control during study participation
  • Attend in person at the study clinic with some options for home health visits

Exclusion Criteria

  • History of stroke, heart failure, heart surgery
  • History of seizures requiring treatment
  • Medical condition that may cause left ventricular hypertrophy
  • Current severe depression measured by Becks Depression Inventory (BDI)
  • History of drug or alcohol abuse

Will I be paid for my time?


For more information contact:

Carrie Bailey


IRB#: IRB_00167895

PI: Nicola Longo


Approval Date: 2023-09-27 06:00:00

Specialties: Pediatric Genetics

Last Updated: 6/8/23