Summary:
Study Summary:
Two clinical research studies for individuals 18 years of age and older who have been diagnosed with Fabry disease.
Study Purpose:
The purpose of the Sanofi CARAT and PERIDOT Studies is to evaluate the safety and efficacy of an oral study drug, venglustat, in patients with Fabry disease. The CARAT Study focuses on managing progressed symptoms of left ventricular hypertrophy in patients with Fabry disease, while the PERIDOT Study focuses on reducing nerve pain and abdominal pain in patients with Fabry disease.
In the CARAT Study, venglustat is being compared to standard-of-care treatment (approved Fabry therapies such as agalsidase beta or migalastat). Venglustat works by inhibiting glucosylceramide synthase and reducing the production of glycosphingolipids, and it has the potential to penetrate tissues affected by Fabry disease. Participants will be randomized to receive either venglustat or standard Fabry disease treatment. Participants receiving venglustat will take their treatment once daily. Eventually, all participants in the CARAT Study will receive venglustat in the long-term extension study. Participation in the CARAT Study can last up to approximately three years.
In the PERIDOT Study, venglustat is being compared to a placebo, which is a substance that looks like the study drug but contains no active ingredients. Participants will be randomized to receive either venglustat or the placebo, which will both be taken once daily. Eventually, all participants in the PERIDOT Study will receive venglustat in the open-label extension period. Participation in the PERIDOT Study can last up to approximately two years.
Eligibility Criteria:
Eligible participants must:
- Be 18 years old and older
- Have a confirmed diagnosis of Fabry disease
- Not have a prior history of heart attack, stroke, or heart failure
- (PERIDOT Study only) Not have had prior treatment for Fabry disease within the past six months
- (CARAT Study only) Have Fabry-related heart involvement
- (PERIDOT Study only) Have had nerve pain or abdominal pain related to Fabry disease
There are additional eligibility requirements, which the study doctor can explain to you.