Venglustat for Fabry Disease
(CARAT Trial)
Trial Summary
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, if you are on strong or moderate inducers or inhibitors of cytochrome P450 CYP3A4, you may need to stop them at least 14 days before starting the trial.
How does the drug Venglustat differ from other treatments for Fabry disease?
Venglustat is unique because it is an oral medication that works by inhibiting glucosylceramide synthase, which reduces the production of glycosphingolipids that accumulate in Fabry disease. This approach is different from enzyme replacement therapies that are typically administered intravenously and focus on replacing the deficient enzyme in patients.12345
What is the purpose of this trial?
This is an 18-month, multicenter, randomized, active-control, parallel-group Phase 3 study, in which participants will be randomized to venglustat versus standard of care therapy (agalsidase alfa, agalsidase beta, or migalastat) to evaluate the effect of venglustat on left ventricular mass index (LVMI) in adult participants with Fabry disease and left ventricular hypertrophy.* Study visits will take place approximately every 3 to 6 months* Participants who complete the randomized period may continue to the long-term extension (LTE) to receive venglustat for up to additional 45 months with the total study duration up to 5.3 years maximum.
Research Team
Clinical Sciences & Operations Clinical Sciences and Operations
Principal Investigator
Sanofi
Eligibility Criteria
Adults aged 18-65 with Fabry disease and heart enlargement (left ventricular hypertrophy) can join this trial. They may be new or existing patients treated with specific enzyme replacement therapies or migalastat. Participants must not be pregnant, breastfeeding, or donating sperm, and should have no severe liver issues, certain infections like HIV/Hepatitis B/C, recent use of strong drugs affecting the liver's drug processing enzymes, major cardiovascular events/surgeries/kidney transplant history, seizures requiring treatment, extreme cardiac fibrosis on MRI scans unrelated to Fabry disease.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants are randomized to receive either venglustat or standard of care therapy for 18 months
Follow-up
Participants are monitored for safety and effectiveness after treatment
Long-term extension
Participants may continue to receive venglustat for up to an additional 45 months
Treatment Details
Interventions
- Agalsidase alfa
- Agalsidase beta (GZ419828)
- Migalastat
- Venglustat (GZ402671)
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris