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Treatment of Fragile X Syndrome with a Neuroactive Steroid

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Technical Report,15 Jul 2014,14 Jul 2015

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University of California, Davis Sacramento

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This study is a Phase II trial to assess the safety, tolerability, and efficacy of ganaxolone, a GABAA agonist, for the treatment of behavioral problems, including anxiety and inattention in children with FXS. It has been demonstrated in the fragile X mouse model and the Drosophila fruit fly models of FXS that the GABAA system, including multiple receptors, is dramatically down-regulated. Ganaxolone is a drug that enhances GABAA activity. We hypothesized that ganaxolone will significantly improve behavioral problems such as anxiety, inattention, and impulsivity problems in children with fragile X syndrome. We planned to enroll 60 children, ages 6-17 years, with fragile X syndrome over a 4-year period and they would be randomized to receive either ganaxolone or a placebo initially and then crossed over after 6 weeks. We have used innovative outcome measures in addition to standard outcome measures that have been successful in previous treatment trials in fragile X syndrome at baseline and follow-up visits.

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