Phase 3 Trappsol Cyclo in Niemann-Pick disease type C1

Phase 3, randomised, double-blind trial in Niemann-Pick disease type C1 (NPC1) comparing Trappsol Cyclo + standard of care with Placebo + standard of care (2 arms). Primary endpoints: Improvement in 4-domain Niemann-Pick disease Type C Severity Scale composite score and Improvement in 5-domain Niemann-Pick disease Type C Severity Scale composite score. Started Jun 2021; status: active.