PTSD
Conditions
Keywords
PTSD, Posttraumatic Stress Disorder, pharmacotherapy, ganaxolone
Brief 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.
Interventions
DRUGGanaxolone
200-600 mg bid, capsules, up to 12 weeks
DRUGPlacebo
capsules, bid, up to 12 weeks
Sponsors
INTRuST, Post-Traumatic Stress Disorder - Traumatic Brain Injury Clinical Consortium
U.S. Army Medical Research and Development Command
Marinus Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 55 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Baseline (Week 0) and at Week 2, Week 4 and Week 6 | The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The CAPS total score is the sum of frequency and intensity ratings for each item and the score range was 0: no symptoms to 136: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in PTSD Re-Experience Score | Baseline (Week 0) and at Week 2, Week 4 and Week 6 | The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Re-experience score is the sum of frequency and intensity ratings for items from cluster B and the score range was 0: no symptoms to 40: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Change From Baseline in PTSD Avoidance Score | Baseline (Week 0) and at Week 2, Week 4 and Week 6 | The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Avoidance score is the sum of frequency and intensity ratings for items from cluster C.and the score range was 0: no symptoms to 56: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Change From Baseline in PTSD Hyperarousal Score | Baseline (Week 0) and at Week 2, Week 4 and Week 6 | The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Hyperarousal score is the sum of frequency and intensity ratings for items from cluster D and the score range was 0: no symptoms to 40: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 2, Week 4 and Week 6 | The CGI-II scale is a 7-point scale where the investigator was required to assess how much the participant's illness has improved or worsened relative to a Baseline state at the beginning of the intervention, and rated as 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. Higher score indicated worse symptoms. The score was grouped into two categories for analysis: Responders=1 or 2, Non-responders=3-7. |
| Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score | Baseline (Week 0) and Week 6 | The CD-RISC comprises 25 items, each rated on a 5-point scale (0-4), with higher scores reflecting greater resilience that may be a target for treatment in stress disorders. The total score is the sum of 25 item scores and ranged from 0: no symptoms to 100: worse symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value |
| Change From Baseline in PTSD Checklist (PCL) Scores | Baseline (Week 0) and at Week 2, Week 4 and Week 6 | The PCL is a 17-item self-report scale representing all DSM-IV criteria for PTSD, including re-experiencing, avoidance and numbing, and hyperarousal criteria. Each item is scored 1-5 (1=not at all; 2=a little bit; 3=moderately; 4=quite a bit; 5=extremely). The total score is the sum of 17 item scores and ranged from 17: no symptoms to 85: severe symptoms. Higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Change From Baseline in Profile of Mood States (POMS) Total Score | Baseline (Week 0) and Week 6 | The POMS is a factor-analytically derived 65 item self-report inventory with 5-point measures of five dimensions of negative mood: anger/irritability, anxiety/tension, depression/dejection, confusion/bewilderment, and fatigue/inertia, as well as vigor/activity. Total score was calculated as Fatigue + Mood (sum \[Confusion, anger, Anxiety/Tension, Depression\])- Vigor. Score ranged from 0: no symptoms to 200: severe symptoms; higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Change From Baseline in Patient Health Questionnaire (PHQ-9) Score | Baseline (Week 0) and Week 6 | The PHQ-9 is the nine-item depression scale of the Patient Health Questionnaire. The nine items of the PHQ-9 are based directly on the nine diagnostic criteria for major depressive disorder in the DSM-IV. Each item is scored 0-3 (0=not at all; 1=several days; 2=more than half the days; 3= nearly every day). The total score is the sum of 9 item scores and ranged 0: no depression to 27: severe depression. The higher the total score, the worse the depression. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Change From Baseline in Insomnia Severity Index (ISI) Score | Baseline (Week 0) and Week 6 | The ISI is a self-rated questionnaire assessing the difficulty over the past two weeks with falling to sleep, awakening in the middle of the night, and early morning awakening, as well as the degree to which insomnia impairs daily function. It included 5 items (rst item has 3 sub-questions), so a total of 7 questions. Each question is scored 0-4 (0=none, 1=mild, 2=moderate, 3=severe, 4=very severe). The total score is the sum of 7 item scores and ranged from 0: no symptoms to 28: severe symptoms. Higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value. |
| Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 2, Week 4 and Week 6 | The CGI-IS scale is a 7-point scale where the participant assesses how much the illness has improved or worsened relative to a Baseline state at the beginning of the intervention, and rated as 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. Higher score indicated worse symptoms. The score was grouped into two categories for analysis: Responders=1 or 2, Non-responders=3-7. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Ganaxolone active
Ganaxolone: 200-600 mg bid, capsules, up to 12 weeks | 59 |
| Placebo non-active
Placebo: capsules, bid, up to 12 weeks | 53 |
| Total | 112 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 1 | 2 |
| Overall Study | Lost to Follow-up | 9 | 3 |
| Overall Study | multiple reasons | 1 | 1 |
| Overall Study | Noncompliance | 1 | 0 |
| Overall Study | Other | 2 | 2 |
| Overall Study | Relocated out of area | 0 | 1 |
| Overall Study | Withdrawal by Subject | 3 | 0 |
Baseline characteristics
| Characteristic | Placebo | Total | Ganaxolone |
|---|---|---|---|
| Age, Continuous | 37.74 years STANDARD_DEVIATION 10.86 | 38.29 years STANDARD_DEVIATION 10.66 | 38.78 years STANDARD_DEVIATION 10.54 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 4 Participants | 10 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 48 Participants | 98 Participants | 50 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants | 4 Participants | 3 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 2 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 16 Participants | 35 Participants | 19 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 3 Participants | 3 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 6 Participants | 5 Participants |
| Race (NIH/OMB) White | 35 Participants | 65 Participants | 30 Participants |
| Sex: Female, Male Female | 9 Participants | 24 Participants | 15 Participants |
| Sex: Female, Male Male | 44 Participants | 88 Participants | 44 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 59 | 0 / 53 |
| other Total, other adverse events | 39 / 59 | 32 / 53 |
| serious Total, serious adverse events | 3 / 59 | 0 / 53 |
Outcome results
Primary
Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6
The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The CAPS total score is the sum of frequency and intensity ratings for each item and the score range was 0: no symptoms to 136: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 2 | 65.78 Scores on a scale | Standard Deviation 24.4 |
| Placebo | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 4 | 63.2 Scores on a scale | Standard Deviation 23.06 |
| Placebo | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 6 | 60.64 Scores on a scale | Standard Deviation 27.54 |
| Ganaxolone | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 2 | 62.67 Scores on a scale | Standard Deviation 21.39 |
| Ganaxolone | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 4 | 61.30 Scores on a scale | Standard Deviation 23.42 |
| Ganaxolone | Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6 | Week 6 | 54.05 Scores on a scale | Standard Deviation 25.86 |
Secondary
Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score
The CD-RISC comprises 25 items, each rated on a 5-point scale (0-4), with higher scores reflecting greater resilience that may be a target for treatment in stress disorders. The total score is the sum of 25 item scores and ranged from 0: no symptoms to 100: worse symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value
Time frame: Baseline (Week 0) and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score | 60.84 Scores on a scale | Standard Deviation 20.67 |
| Ganaxolone | Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score | 61.01 Scores on a scale | Standard Deviation 19.56 |
Secondary
Change From Baseline in Insomnia Severity Index (ISI) Score
The ISI is a self-rated questionnaire assessing the difficulty over the past two weeks with falling to sleep, awakening in the middle of the night, and early morning awakening, as well as the degree to which insomnia impairs daily function. It included 5 items (rst item has 3 sub-questions), so a total of 7 questions. Each question is scored 0-4 (0=none, 1=mild, 2=moderate, 3=severe, 4=very severe). The total score is the sum of 7 item scores and ranged from 0: no symptoms to 28: severe symptoms. Higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Insomnia Severity Index (ISI) Score | 17.04 Scores on a scale | Standard Deviation 7.69 |
| Ganaxolone | Change From Baseline in Insomnia Severity Index (ISI) Score | 14.36 Scores on a scale | Standard Deviation 7.94 |
Secondary
Change From Baseline in Patient Health Questionnaire (PHQ-9) Score
The PHQ-9 is the nine-item depression scale of the Patient Health Questionnaire. The nine items of the PHQ-9 are based directly on the nine diagnostic criteria for major depressive disorder in the DSM-IV. Each item is scored 0-3 (0=not at all; 1=several days; 2=more than half the days; 3= nearly every day). The total score is the sum of 9 item scores and ranged 0: no depression to 27: severe depression. The higher the total score, the worse the depression. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Patient Health Questionnaire (PHQ-9) Score | 12.13 Scores on a scale | Standard Deviation 7.47 |
| Ganaxolone | Change From Baseline in Patient Health Questionnaire (PHQ-9) Score | 10.71 Scores on a scale | Standard Deviation 6.99 |
Secondary
Change From Baseline in Profile of Mood States (POMS) Total Score
The POMS is a factor-analytically derived 65 item self-report inventory with 5-point measures of five dimensions of negative mood: anger/irritability, anxiety/tension, depression/dejection, confusion/bewilderment, and fatigue/inertia, as well as vigor/activity. Total score was calculated as Fatigue + Mood (sum \[Confusion, anger, Anxiety/Tension, Depression\])- Vigor. Score ranged from 0: no symptoms to 200: severe symptoms; higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Placebo | Change From Baseline in Profile of Mood States (POMS) Total Score | 80.07 Scores on a scale | Standard Deviation 50.21 |
| Ganaxolone | Change From Baseline in Profile of Mood States (POMS) Total Score | 66.98 Scores on a scale | Standard Deviation 45.29 |
Secondary
Change From Baseline in PTSD Avoidance Score
The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Avoidance score is the sum of frequency and intensity ratings for items from cluster C.and the score range was 0: no symptoms to 56: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in PTSD Avoidance Score | Week 2 | 27.06 Scores on a scale | Standard Deviation 11.54 |
| Placebo | Change From Baseline in PTSD Avoidance Score | Week 4 | 25.87 Scores on a scale | Standard Deviation 11.69 |
| Placebo | Change From Baseline in PTSD Avoidance Score | Week 6 | 24.82 Scores on a scale | Standard Deviation 11.45 |
| Ganaxolone | Change From Baseline in PTSD Avoidance Score | Week 6 | 23.52 Scores on a scale | Standard Deviation 13.1 |
| Ganaxolone | Change From Baseline in PTSD Avoidance Score | Week 2 | 26.29 Scores on a scale | Standard Deviation 10.76 |
| Ganaxolone | Change From Baseline in PTSD Avoidance Score | Week 4 | 26.09 Scores on a scale | Standard Deviation 11.87 |
Secondary
Change From Baseline in PTSD Checklist (PCL) Scores
The PCL is a 17-item self-report scale representing all DSM-IV criteria for PTSD, including re-experiencing, avoidance and numbing, and hyperarousal criteria. Each item is scored 1-5 (1=not at all; 2=a little bit; 3=moderately; 4=quite a bit; 5=extremely). The total score is the sum of 17 item scores and ranged from 17: no symptoms to 85: severe symptoms. Higher score indicated worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in PTSD Checklist (PCL) Scores | Week 2 | 54.4 Scores on a scale | Standard Deviation 14.24 |
| Placebo | Change From Baseline in PTSD Checklist (PCL) Scores | Week 4 | 52.93 Scores on a scale | Standard Deviation 15.31 |
| Placebo | Change From Baseline in PTSD Checklist (PCL) Scores | Week 6 | 51.75 Scores on a scale | Standard Deviation 15.73 |
| Ganaxolone | Change From Baseline in PTSD Checklist (PCL) Scores | Week 6 | 46.33 Scores on a scale | Standard Deviation 17.29 |
| Ganaxolone | Change From Baseline in PTSD Checklist (PCL) Scores | Week 2 | 50.79 Scores on a scale | Standard Deviation 15.05 |
| Ganaxolone | Change From Baseline in PTSD Checklist (PCL) Scores | Week 4 | 48.74 Scores on a scale | Standard Deviation 16.2 |
Secondary
Change From Baseline in PTSD Hyperarousal Score
The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Hyperarousal score is the sum of frequency and intensity ratings for items from cluster D and the score range was 0: no symptoms to 40: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in PTSD Hyperarousal Score | Week 2 | 22.16 Scores on a scale | Standard Deviation 7.73 |
| Placebo | Change From Baseline in PTSD Hyperarousal Score | Week 4 | 20.78 Scores on a scale | Standard Deviation 7.8 |
| Placebo | Change From Baseline in PTSD Hyperarousal Score | Week 6 | 20.32 Scores on a scale | Standard Deviation 9.07 |
| Ganaxolone | Change From Baseline in PTSD Hyperarousal Score | Week 2 | 21.94 Scores on a scale | Standard Deviation 7.76 |
| Ganaxolone | Change From Baseline in PTSD Hyperarousal Score | Week 4 | 21.05 Scores on a scale | Standard Deviation 8.48 |
| Ganaxolone | Change From Baseline in PTSD Hyperarousal Score | Week 6 | 18.21 Scores on a scale | Standard Deviation 8.55 |
Secondary
Change From Baseline in PTSD Re-Experience Score
The CAPS was a structured interview that queries participants about each of the 17 Diagnostic and Statistical manual of Mental Disorders IV (DSM-IV) criteria B, C, and D symptoms of PTSD. Each item has a frequency score (0-4) and intensity score (0-4). The Re-experience score is the sum of frequency and intensity ratings for items from cluster B and the score range was 0: no symptoms to 40: severe symptoms. Higher score indicates worse symptoms. Baseline was defined as the Day 0 assessment before study drug infusion. Change from Baseline was calculated by subtracting Baseline value from the post-dose visit value.
Time frame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Placebo | Change From Baseline in PTSD Re-Experience Score | Week 2 | 16.56 Scores on a scale | Standard Deviation 10.61 |
| Placebo | Change From Baseline in PTSD Re-Experience Score | Week 4 | 16.54 Scores on a scale | Standard Deviation 8.03 |
| Placebo | Change From Baseline in PTSD Re-Experience Score | Week 6 | 15.5 Scores on a scale | Standard Deviation 11.11 |
| Ganaxolone | Change From Baseline in PTSD Re-Experience Score | Week 2 | 14.45 Scores on a scale | Standard Deviation 8.98 |
| Ganaxolone | Change From Baseline in PTSD Re-Experience Score | Week 4 | 14.16 Scores on a scale | Standard Deviation 8.83 |
| Ganaxolone | Change From Baseline in PTSD Re-Experience Score | Week 6 | 12.31 Scores on a scale | Standard Deviation 9.28 |
Secondary
Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale
The CGI-IS scale is a 7-point scale where the participant assesses how much the illness has improved or worsened relative to a Baseline state at the beginning of the intervention, and rated as 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. Higher score indicated worse symptoms. The score was grouped into two categories for analysis: Responders=1 or 2, Non-responders=3-7.
Time frame: Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 2 - Non-Responder | 45 Participants |
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 2 - Responder | 5 Participants |
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 4 - Non-Responder | 39 Participants |
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 4 - Responder | 7 Participants |
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 6 - Non-Responder | 37 Participants |
| Placebo | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 6 - Responder | 7 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 6 - Non-Responder | 37 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 2 - Non-Responder | 46 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 4 - Responder | 6 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 2 - Responder | 2 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 6 - Responder | 5 Participants |
| Ganaxolone | Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale | Week 4 - Non-Responder | 37 Participants |
Secondary
Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale
The CGI-II scale is a 7-point scale where the investigator was required to assess how much the participant's illness has improved or worsened relative to a Baseline state at the beginning of the intervention, and rated as 1, very much improved; 2, much improved; 3, minimally improved; 4, no change; 5, minimally worse; 6, much worse; or 7, very much worse. Higher score indicated worse symptoms. The score was grouped into two categories for analysis: Responders=1 or 2, Non-responders=3-7.
Time frame: Week 2, Week 4 and Week 6
Population: mITT Population. Only those participants with data available at the specified data points were analyzed
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 2 - Non-Responder | 42 Participants |
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 2 - Responder | 7 Participants |
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 4 - Non-Responder | 37 Participants |
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 4 - Responder | 9 Participants |
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 6 - Non-Responder | 32 Participants |
| Placebo | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 6 - Responder | 12 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 6 - Non-Responder | 27 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 2 - Non-Responder | 45 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 4 - Responder | 5 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 2 - Responder | 3 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 6 - Responder | 14 Participants |
| Ganaxolone | Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale | Week 4 - Non-Responder | 38 Participants |