Topline results from the pivotal Phase 3 LAVENDER clinical trial reveal that trofinetide met its co-primary efficacy endpoints, providing significant therapeutic benefits in the core symptoms of RettSyndrome. Neurodevelopment RareDiseases
Two additional efficacy analyses were conducted post hoc: CGI-I scale responders at week 12 and coprimary endpoints assessed in the presence or absence of the most commonly reported TEAE of diarrhea.A sample size of 184 participants was planned to provide 90% power for both coprimary endpoints combined with a two-sided significance level of 0.05. Efficacy was assessed in the full analysis set ; the safety analysis set consisted of participants who received at least one dose.
Coprimary and key secondary efficacy endpoints were analyzed using the MMRM method assuming data missing at random. The MMRM included randomization strata of age group and baseline RSBQ severity score, baseline RSBQ , baseline CGI-S and baseline CSBS-DP-IT Social Composite score , treatment, visit, treatment-by-visit interaction and baseline-by-visit interaction as fixed effects and participant as a random effect; an unstructured covariance matrix modeled within-participant errors.
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