drugset / Trial / NCT01703533
A Safety Study of NNZ-2566 in Patients With Rett Syndrome
Phase 2
Completed
67 enrolled
Neuren Pharmaceuticals Limited
Baylor College of Medicine · collabInternational Rett Syndrome Foundation · collab
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 adolescent and adult females.
Timeline
- Start
- 2013-03
- Primary completion
- 2014-09
- Completion
- 2014-09
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 | 35 mg/kg | Oral |
| Subject | Trofinetide | Peptide | 70 mg/kg | Oral |