Venglustat in late-onset and juvenile/adolescent GM2 gangliosidosis and related ultra-rare sphingolipid disorders

Randomised, double-blind trial in GM2 gangliosidosis, GM1 gangliosidosis (GM1), Saposin C Deficiency (SCD) and 2 more comparing Venglustat with Placebo (2 arms). Primary endpoints: Percent change in CSF GM2 biomarker from baseline to Week 104 in primary population, Annualized rate of change in 9-hole pegboard test (9-HPT) in primary population and 10 more. Started Oct 2020; status: completed.