Clinical Trial: Ganaxolone Treatment in Children With Fragile X Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Controlled, Double-blind, Crossover Trial of Ganaxolone in Children With Fragile X Syndrome

Brief 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). Up to 60 subjects (ages 6-17 yrs) will be randomized to receive either ganaxolone or placebo for 6 weeks and then cross over to the opposite treatment for another 6 weeks. The aim of the study is assess the safety, tolerability and efficacy of ganaxolone for treatment of anxiety and attention in subjects with FXS. The hypothesis is that ganaxolone treatment compared to placebo will improve anxiety and attention as measured by the several neuropsychological and psychometric tests.

Detailed Summary: This is a single center study at UC Davis MIND Institute. Children with fragile x syndrome between the ages of 6-17yrs, inclusive will be randomized at a 1:1 ratio to receive ganaxolone or placebo treatment for 6 weeks, discontinue treatment and washout for 2 weeks, and then cross over to the opposite treatment for another 6 weeks. The primary aim of the study is to assess efficacy of ganaxolone treatment compared to placebo on clinical behaviors such as anxiety and attention as measured by Clinician's Global Impression-Improvement (CGI-I). The key secondary efficacy measure is the Pediatric Anxiety Scale (PARS). Other secondary efficacy measures include the visual analog scale (VAS), Anxiety, Depression, Attention, and Mood Scale (ADAMS), Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV), and Aberrant Behavior Checklist- Community Edition (ABC-C). Tolerability and safety will be monitored by routine vital signs, physical/neurological exams, ECGs, clinical laboratory and adverse event assessments.
Sponsor: Marinus Pharmaceuticals

Current Primary Outcome: Clinician's Global Impression-Improvement (CGI-I) [ Time Frame: Weeks 3, 6, 8, 11, 14 ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pediatric Anxiety Rating Scale (PARS) [ Time Frame: Baseline, Weeks 3, 6, 8, 11, 14 ]
  • Visual Analog Scale [ Time Frame: Baseline, Weeks 6, 8, 14 ]
  • Anxiety, Depression, and Mood Scale (ADAMS) [ Time Frame: Baseline, Weeks 6, 8, 14 ]
  • Aberrant Behavior Checklist [ Time Frame: Baseline, Weeks 6, 8, 14 ]
  • Swanson, Nolan, and Pelham-IV Questionnaire (SNAP-IV) [ Time Frame: Baseline, Weeks 6, 8, 14 ]


Original Secondary Outcome: Same as current

Information By: Marinus Pharmaceuticals

Dates:
Date Received: November 7, 2012
Date Started: November 2012
Date Completion: June 2016
Last Updated: March 14, 2016
Last Verified: March 2016