Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

Summary

This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

Timeline

Start

2010-11

Primary completion

2013-08

Completion

2013-08

Outcome

Missed primary endpoint

paper Neither of the studies achieved the primary efficacy end point PMID 26764156 ↗

Publications

• Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.

Drugs

Indications