Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
2024-518266-27-00Phase 3Authorised
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: Primary EU/RoW interim efficacy endpoint, Primary EU/RoW final efficacy endpoint and 2 more. Planned start Nov 2024; status: authorised.
Beyond the preview
Study design, results and competitive context are available in the Gosset platform where reported.
Available in the Gosset platform where reported.
Available in the Gosset platform where reported.
See how Gosset can support your research on 2024-518266-27-00.