LAVENDER: reading results alongside tolerability.
A 12-week comparative trial, published in 2023.
Results presented
LAVENDER randomized 187 participants: trofinetide n=93, placebo n=94. RSBQ and CGI-I favored trofinetide at 12 weeks, with diarrhea 80.6 % versus 19.1 % on placebo.
Randomized, double-blind, placebo-controlled phase 3 study, 12 weeks.
187 participants aged 5 to 20: trofinetide n=93, placebo n=94; 155 completed.
RSBQ LSM: −4.9 vs −1.7, p=0.0175. CGI-I: 3.5 vs 3.8, p=0.0030. CSBS-DP-IT: −0.1 vs −1.1, p=0.0064.
At least 1 TEAE: 92.5 % vs 54.3 %. Diarrhea: 80.6 % vs 19.1 %. Serious AEs: 3.2 % in each group.
The Nature Medicine paper lists funding, contributions and interests in the publisher information.
12-week follow-up, more discontinuations on trofinetide and frequent digestive effects.
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
The study is comparative and positive on its primary endpoints, but the average effect remains modest and digestive tolerability led to discontinuations.
Original title (English)
Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study
Neul JL et al. · Nature Medicine
Online publication · Jun 8, 2023
PMID 37291210
DOI 10.1038/s41591-023-02398-1
Related study
Trofinetide · LAVENDER
Consulted · Sep 8, 2026
Sources consulted on 8 September 2026 · AI-assisted summaries and translations, not medically validated.