drugset / Trial / NCT04279314
Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
NaSingle-groupOpen-labelTreatment
Summary
To investigate the safety and tolerability of long-term treatment with oral trofinetide in girls and women with Rett syndrome
Timeline
- Start
- 2020-01-29
- Primary completion
- 2022-08-19
- Completion
- 2022-08-19
Publications
- Percy AK, Neul JL, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, An D, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: Results from the open-label extension LILAC study. Med. 2024 Sep 13;5(9):1178-1189.e3. doi: 10.1016/j.medj.2024.05.018. Epub 2024 Jun 24.
- 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 | 30 ml | Oral |
| Subject | Trofinetide | Peptide | 60 ml | Oral |