Ganaxolone in Posttraumatic Stress Disorder (PTSD)

A Proof-of-Concept, Double-blind, Randomized, Placebo-controlled Study of Ganaxolone in Posttraumatic Stress Disorder

This Phase 2 proof-of-concept study is a double-blind, randomized, placebo-controlled, 15-week investigation of ganaxolone versus placebo for the treatment of Posttraumatic Stress Disorder (PTSD). Up to 120 participants will be enrolled and randomized to receive either ganaxolone or placebo for 6 weeks. After 6 weeks of randomized treatment all participants will continue for 6 weeks on ganaxolone. The aim of the study is to assess the efficacy of ganaxolone compared to placebo for the treatment of PTSD symptoms after 6 weeks of treatment using the Clinician-Administered PTSD Rating Scale (CAPS). The second aim of the study is to evaluate the safety and tolerability of ganaxolone in the PTSD population.

Key Inclusion Criteria:

• Veteran or civilian adult outpatients 18-55 years of age, with primary PTSD as defined by Diagnostic and Statistical manual of Mental Disorders-IV (DSM-IV) for at least 6 months
• Must be in general good health-confirmed by medical history, physical examination, and screening laboratory results
• Negative urine drug screen for drugs of abuse
• Negative urine pregnancy test for females of childbearing potential
• Sexually active participants are required to use a medically acceptable form of birth control

Key Exclusion Criteria

• Clinically unstable medical disease; progressive CNS disorder/disease; history of seizures (except childhood febrile seizure); moderate or severe traumatic brain injury (TBI)
• Females who are pregnant or currently breast feeding
• Current or past psychotic disorder, bipolar Type I disorder, or dementia
• Participants with recent drug abuse or dependency (excluding nicotine and caffeine)
• Participants unwilling to comply with the required alcohol prohibition during the trial
• Current suicidal or homicidal ideation necessitating intervention, and those with a history of suicide attempt in the past 10 years
• Participants with pending litigation related to the traumatic event
• Participants who are unwilling to withhold grapefruit or grapefruit juice for the duration of the study
• Participants receiving psychotherapy without a stable paradigm for at least 3 months
• Non-English speaking participants.