Treatment of Fragile X Syndrome with a Neuroactive Steroid

Abstract

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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Document Details

Document Type
Technical Report
Publication Date
Aug 01, 2015
Accession Number
AD1000374

Entities

People

  • Randi Hagerman

Organizations

  • University of California

Tags

DTIC Thesaurus Topics

  • Abstracts
  • Biomedical Research
  • Data Analysis
  • Data Sets
  • Department Of Defense
  • Diptera
  • Disease Attributes
  • Diseases And Disorders
  • Drosophila
  • Electronic Mail
  • Fragile-X Syndrome
  • Health Services
  • Information Operations
  • Standards
  • Statistical Analysis

Fields of Study

  • Biology

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