Ganaxolone Treatment in Children With Fragile X Syndrome

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.

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