Phase 2
Completed N=112
Ganaxolone in Posttraumatic Stress Disorder (PTSD)
Source: ClinicalTrials.gov NCT01339689 ↗Enrolled (actual)
112
Serious AEs
2.7%
Results posted
Dec 2022
Primary outcomePrimary: Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 — 65.78; 62.67; 63.2; 61.30 Scores on a scale
Summary
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 |
65.78; 62.67; 63.2; 61.30; 60.64; 54.05 | — |
| SECONDARY Change From Baseline in PTSD Re-Experience Score |
16.56; 14.45; 16.54; 14.16; 15.5; 12.31 | — |
| SECONDARY Change From Baseline in PTSD Avoidance Score |
27.06; 26.29; 25.87; 26.09; 24.82; 23.52 | — |
| SECONDARY Change From Baseline in PTSD Hyperarousal Score |
22.16; 21.94; 20.78; 21.05; 20.32; 18.21 | — |
| SECONDARY Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale |
42; 45; 7; 3; 37; 38 | — |
| SECONDARY Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale |
45; 46; 5; 2; 39; 37 | — |
| SECONDARY Change From Baseline in PTSD Checklist (PCL) Scores |
54.4; 50.79; 52.93; 48.74; 51.75; 46.33 | — |
| SECONDARY Change From Baseline in Profile of Mood States (POMS) Total Score |
80.07; 66.98 | — |
| SECONDARY Change From Baseline in Patient Health Questionnaire (PHQ-9) Score |
12.13; 10.71 | — |
| SECONDARY Change From Baseline in Insomnia Severity Index (ISI) Score |
17.04; 14.36 | — |
| SECONDARY Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score |
60.84; 61.01 | — |
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01339689). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.