Trofinetide Phase 3 Trial in 180 girls with Rett syndrome

Feb. 23, 2020

Neuren Pharmaceuticals is developing what will hopefully be the first real drug for Rett Syndrome. They partnered with Acadia Pharmaceuticals to conduct a phase 3 trial in Rett syndrome.

This Phase 3 randomized, double-blind placebo-controlled study will evaluate Trofinetide and placebo in about 180 girls with Rett syndrome. It will measure the Rett Syndrome Behavior Questionnaire (RSBQ), a caregiver assessment, and the Clinical Global Impression of Improvement (CGI-I), a physician assessment, as co-primary efficacy endpoints.

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