Arbaclofen in fragile X syndrome: results of phase 3 trials.

ABSTRACT:

Background

Arbaclofen improved multiple abnormal phenotypes in animal models of fragile X syndrome (FXS) and showed promising results in a phase 2 clinical study. The objective of the study is to determine safety and efficacy of arbaclofen for social avoidance in FXS.

Methods

Two phase 3 placebo-controlled trials were conducted, a flexible dose trial in subjects age 12-50 (209FX301, adolescent/adult study) and a fixed dose trial in subjects age 5-11 (209FX302, child study). The primary endpoint for both trials was the Social Avoidance subscale of the Aberrant Behavior Checklist-Community Edition, FXS-specific (ABC-C_FX). Secondary outcomes included other ABC-C_FX subscale scores, Clinical Global Impression-Improvement (CGI-I), Clinical Global Impression-S

SUBMITTER: Berry-Kravis E 

PROVIDER: S-EPMC5467054 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature