Trofinetide in young girls: what does DAFFODIL show?
An open-label study in 15 girls aged 2–4.
Results presented
DAFFODIL enrolled 15 girls. Diarrhea 80.0 % and vomiting 53.3 % were the most frequent AEs; 2 participants stopped because of AEs.
Open-label phase 2/3 DAFFODIL, two periods: 12 weeks then about 21 months, without placebo.
15 participants received trofinetide; 12 completed, 2 stopped because of AEs and 1 because of noncompliance.
Mean CGI-I: 3.3 week 4, 3.1 week 12, 2.2 week 78. ICND-QoL: 3.9 baseline, 4.2 week 12, 4.6 week 78.
TEAE in 14/15 participants. Diarrhea 80.0 %, vomiting 53.3 %, COVID-19 46.7 %; 4/15 had a serious TEAE.
PubMed funding: Acadia Pharmaceuticals. Interest declarations should be read in the Med article.
Small sample, open-label design, no comparator; efficacy assessments are exploratory.
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 open-label, small and has no comparator. Exploratory improvements alone do not prove a causal effect.
Original title (English)
Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2–4 years with Rett syndrome
Percy AK et al. · Med
Online publication · Mar 4, 2025
PMID 40043705
DOI 10.1016/j.medj.2025.100608
Related study
Trofinetide · DAFFODIL
Consulted · Sep 8, 2026
Sources consulted on 8 September 2026 · AI-assisted summaries and translations, not medically validated.