A Safety Study of NNZ-2566 in Patients With Fragile X Syndrome

Phase 2 Completed 72 enrolled Neuren Pharmaceuticals Limited

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The purpose of this study is to determine whether NNZ-2566 is safe and well tolerated in the treatment of Fragile X Syndrome in adolescent and adult males.

Timeline

Start: 2014-01
Primary completion: 2015-09
Completion: 2015-10

Outcome

Met primary endpoint

release “Primary endpoint achieved - both dose levels were well tolerated and no safety concerns were” neurenpharma.com ↗

Publications

Berry-Kravis E, Horrigan JP, Tartaglia N, Hagerman R, Kolevzon A, Erickson CA, Hatti S, Snape M, Yaroshinsky A, Stoms G; FXS-001 Investigators; Glass L, Jones NE. A Double-Blind, Randomized, Placebo-Controlled Clinical Study of Trofinetide in the Treatment of Fragile X Syndrome. Pediatr Neurol. 2020 Sep;110:30-41. doi: 10.1016/j.pediatrneurol.2020.04.019. Epub 2020 May 23.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Trofinetide	Peptide	35 mg/kg	Oral
Subject	Trofinetide	Peptide	70 mg/kg	Oral