Additional Relevant Details
The Phase 2 study enrolled 59 children between 6 and 17 years of age with a diverse range and severity of FXS-related symptoms. The trial utilized multiple clinician- and parent-administered rating scales designed to identify a signal among a variety of symptoms common to FXS children. Twenty-nine of the treated patients had a high level of anxiety at baseline (defined as baseline Pediatric Anxiety Rating Scale (PARS) ≥13) and showed improvements in anxiety, attention and hyperactivity as measured by scales designed to detect these symptomatic changes (Visual Analog Scale (VAS), Anxiety, Depression & Mood Scale (ADAMS), and Aberrant Behavior Checklist (ABC)).


Ganaxolone was generally safe and well tolerated with no serious adverse events (AEs) reported. Consistent with other pediatric studies conducted with ganaxolone, the most common AEs (>5%) considered related to ganaxolone treatment and reported at greater rates than placebo were fatigue, somnolence, diarrhea, decreased appetite and rash. Most AEs were mild to moderate in severity and resolved. Five patients discontinued the study due to AEs: four during treatment with ganaxolone and one during placebo.