A randomized double-blind, placebo-controlled trial of ganaxolone in children and adolescents with fragile X syndrome.

ABSTRACT:

Background

Gamma-aminobutyric acid (GABA) system deficits are integral to the pathophysiologic development of fragile X syndrome (FXS). Ganaxolone, a GABA_A receptor positive allosteric modulator, is hypothesized to improve symptoms such as anxiety, hyperactivity, and attention deficits in children with FXS.

Methods

This study was a randomized, double-blind, placebo-controlled, crossover trial of ganaxolone in children with FXS, aged 6-17 years.

Results

Sixty-one participants were assessed for eligibility, and 59 were randomized to the study. Fifty-five participants completed at least the first arm and were included in the intention-to-treat analysis; 51 participants completed both treatment arms. There were no statistically significant improvements observed

SUBMITTER: Ligsay A 

PROVIDER: S-EPMC5540519 | biostudies-literature | 2017 Aug

REPOSITORIES: biostudies-literature