Expired Study
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Sacramento, California 95817


Purpose:

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.


Study 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.


Criteria:

Inclusion Criteria: - molecular documentation of FMR1 full mutation - ages 6-17 yrs, inclusive - sexually active subjects are required to use a medically acceptable form of birth control Exclusion Criteria: - non-English or Spanish speaking subjects - concomitant systemic steroid, vigabatrin, felbamate and ketoconazole - changes in medications within last 2 months - clinically unstable medical disease, progressive CNS disease/disorder - history of recurrent status epilepticus - unwilling to withhold grapefruit or grapefruit juice for the duration of the study - actively suicidal


NCT ID:

NCT01725152


Primary Contact:

Principal Investigator
Randi J Hagerman, MD
M.I.N.D. Institute at University of California at Davis Medical Center


Backup Contact:

N/A


Location Contact:

Sacramento, California 95817
United States



There is no listed contact information for this specific location.

Site Status: N/A


Data Source: ClinicalTrials.gov

Date Processed: November 20, 2017

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