LAVENDER protocol: how the phase 3 trial was designed
The protocol and outcome measures for a 12-week study in 187 participants aged 5 to 20.
Results presented
This paper describes the LAVENDER design before results: randomized, double-blind, placebo-controlled phase 3, over 12 weeks, in 187 girls and women aged 5 to 20.
LAVENDER protocol: randomized, double-blind, placebo-controlled phase 3 study, 12 weeks.
Planned population: 187 girls and women with Rett syndrome, aged 5 to 20.
Planned endpoints: RSBQ, CGI-I and CSBS-DP-IT; this publication presents the plan, not observed results.
The protocol plans safety monitoring but does not yet report observed adverse events.
The publication lists Acadia Pharmaceuticals support; interests and contributions should be read in the publisher article.
A protocol describes objectives and methods before results; clinical interpretation depends on the separate results paper.
RE2T record: documentary writing by RE2T, primary source checked, AI-assisted translation. Editorial review still to complete; no medical validation claimed.
RE2T Recherche · source-controlled-ai-assisted-editorial-pending-no-medical-validation
Limits to keep in mind
A protocol paper helps explain endpoints and limits before reading results; it does not demonstrate treatment efficacy or tolerability.
Original title (English)
Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome
Percy AK et al. · Contemporary Clinical Trials
Online publication · Mar 1, 2022
PMID 35149233
DOI 10.1016/j.cct.2022.106704
Related study
Trofinetide · LAVENDER
Consulted · Sep 8, 2026
Sources consulted on 8 September 2026 · AI-assisted summaries and translations, not medically validated.