drugset / Trial / NCT02715115
A Safety Study of NNZ-2566 in Pediatric Rett Syndrome
RandomizedParallel-groupQuadruple-blindTreatment
Summary
The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett syndrome in children and adolescents.
Timeline
- Start
- 2016-03
- Primary completion
- 2017-01-05
- Completion
- 2017-01-05
Outcome
Met primary endpoint
release “Neuren’s Phase 2 trial of trofinetide demonstrates significant clinical benefit in pediatric Rett syndrome” neurenpharma.com ↗
Publications
- Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
- 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 | 50 mg/kg | Oral |
| Subject | Trofinetide | Peptide | 100 mg/kg | Oral |
| Subject | Trofinetide | Peptide | 200 mg/kg | Oral |