Fabry Disease
Sanofi – PERIDOT
Drug name: Venglustat
- Phase 3, double-blinded, Placebo-controlled
- Mechanism: Glucosylceramide synthase (GCS) inhibitor
Key inclusion criteria:
- Age ≥ 18
- Confirmed diagnosis of Fabry disease
- Participants who are treatment-naïve or without prior treatment with an approved or experimental therapy for Fabry disease within at least 6 months prior to screening
Key exclusion criteria:
- Estimated glomerular filtration rate <60 mL/min/1.73 m2
- A history of drug and/or alcohol abuse within the past year prior to the screening visit. A history of alcohol dependence within the 5 years prior to the screening visit.
Study Coordinator: Josh Ooka
[email protected]