Open-Label Extension Study of Trofinetide for Rett Syndrome

Phase 3 Terminated 77 enrolled ACADIA Pharmaceuticals Inc.

NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start: 2020-11-08
Primary completion: 2023-06-30
Completion: 2023-06-30

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

Background Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Barrett AM, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Long-term safety and efficacy results of the 32-month, open-label LILAC-2 study. Med. 2024 Oct 11;5(10):1275-1281.e2. doi: 10.1016/j.medj.2024.06.007. Epub 2024 Jul 17.
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	—	Oral