Venglustat versus placebo for pain in Fabry disease

Phase 3, randomised, double-blind trial in Fabry disease (FD) comparing Venglustat with Placebo (2 arms). Primary endpoints: Percent change from baseline at 6 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome items and Percent change from baseline at 12 months in the most bothersome symptom of 3 Fabry Disease Patient-Reported Outcome items. Started Nov 2022; status: active, not recruiting.