Ganaxolone Treatment in Children With Fragile X Syndrome

Phase 2 Completed 59 enrolled Marinus Pharmaceuticals U.S. Army Medical Research and Development Command · collab University of California, Davis · collab

RandomizedCrossoverQuadruple-blindTreatment

Summary

This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, crossover study to investigate ganaxolone treatment in children with fragile x syndrome (FXS). The objective of the study is to assess the safety, tolerability and efficacy of ganaxolone in the treatment of anxiety and attention in subjects with FXS.

Timeline

Start: 2012-11
Primary completion: 2015-10-16
Completion: 2016-11-01

Outcome

Missed primary endpoint

paper There were no statistically significant improvements observed on the primary outcome measure (Clinical Global Impression-Improvement) PMID 28764646 ↗

registry analysis (superiority test); Ganaxolone vs Placebo; p = 0.4475; Linear mixed-effect NCT01725152 ↗

Publications

Ligsay A, Van Dijck A, Nguyen DV, Lozano R, Chen Y, Bickel ES, Hessl D, Schneider A, Angkustsiri K, Tassone F, Ceulemans B, Kooy RF, Hagerman RJ. A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome. J Neurodev Disord. 2017 Aug 2;9(1):26. doi: 10.1186/s11689-017-9207-8.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ganaxolone	Small molecule	12 mg/kg	Oral