An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome

NCT04988867 ↗

Phase 2/3 Terminated 15 enrolled ACADIA Pharmaceuticals Inc.

NaSingle-groupOpen-labelTreatment

Summary

To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls with Rett syndrome

Timeline

Start: 2021-09-22
Primary completion: 2023-05-31
Completion: 2023-05-31

Outcome

Outcome not reported

Stopped: “As a result of marketing approval of trofinetide on 10 March 2023, the study was terminated by the Sponsor with the intent of switching patients to commercially available product.”

Publications

Percy AK, Ryther R, Marsh ED, Neul JL, Benke TA, Berry-Kravis EM, Feyma T, Lieberman DN, Ananth AL, Fu C, Buhrfiend C, Barrett A, Doshi D, Darwish M, An D, Bishop KM, Youakim JM. Results from the phase 2/3 DAFFODIL study of trofinetide in girls aged 2-4 years with Rett syndrome. Med. 2025 Jun 13;6(6):100608. doi: 10.1016/j.medj.2025.100608. Epub 2025 Mar 4.
Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Trofinetide	Peptide	10 ml	Oral
Subject	Trofinetide	Peptide	30 ml	Oral