- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01339689
Ganaxolone in Posttraumatic Stress Disorder (PTSD)
November 30, 2022 updated by: Marinus Pharmaceuticals
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
112
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
California
-
San Diego, California, United States, 92093
- VA San Diego Healthcare System/ University of California, San Diego
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District of Columbia
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Washington, District of Columbia, United States, 20422
- Washington DC VA Medical Center/ Uniformed Services University of the Health Services
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Massachusetts
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Boston, Massachusetts, United States, 02130
- VA Boston Healthcare Services/ Spaulding Rehabilitation Hospital
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New Hampshire
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Manchester, New Hampshire, United States, 03104
- Manchester VA Medical Center/ Dartmouth College
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North Carolina
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Durham, North Carolina, United States, 27705
- Durham VA Medical Center /Duke University Medical Center
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Ohio
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Cincinnati, Ohio, United States, 45220
- Cincinnati VA Medical Center/ University of Cincinnati
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South Carolina
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Charleston, South Carolina, United States, 29401
- Charleston VA Medica Center/ Medical University of South Carolina
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Vermont
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White River Junction, Vermont, United States, 05009
- White River Junction VA Medical Center/ Dartmouth College
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 55 years (Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Ganaxolone
active
|
200-600 mg bid, capsules, up to 12 weeks
Other Names:
|
Placebo Comparator: Placebo
non-active
|
capsules, bid, up to 12 weeks
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6
Time Frame: 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.
|
Baseline (Week 0) and at Week 2, Week 4 and Week 6
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in PTSD Re-Experience Score
Time Frame: 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.
|
Baseline (Week 0) and at Week 2, Week 4 and Week 6
|
Change From Baseline in PTSD Avoidance Score
Time Frame: 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.
|
Baseline (Week 0) and at Week 2, Week 4 and Week 6
|
Change From Baseline in PTSD Hyperarousal Score
Time Frame: 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.
|
Baseline (Week 0) and at Week 2, Week 4 and Week 6
|
Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale
Time Frame: 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.
|
Week 2, Week 4 and Week 6
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Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale
Time Frame: 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.
|
Week 2, Week 4 and Week 6
|
Change From Baseline in PTSD Checklist (PCL) Scores
Time Frame: 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.
|
Baseline (Week 0) and at Week 2, Week 4 and Week 6
|
Change From Baseline in Profile of Mood States (POMS) Total Score
Time Frame: 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.
|
Baseline (Week 0) and Week 6
|
Change From Baseline in Patient Health Questionnaire (PHQ-9) Score
Time Frame: 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.
|
Baseline (Week 0) and Week 6
|
Change From Baseline in Insomnia Severity Index (ISI) Score
Time Frame: 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.
|
Baseline (Week 0) and Week 6
|
Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score
Time Frame: 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
|
Baseline (Week 0) and Week 6
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Christine E Marx, MD, MA, Duke University Medical Center and Durham VA Medical Center
- Principal Investigator: Ann Rasmusson, MD, Boston University School of Medicine Research Affiliate, National Center for PTSD
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
April 1, 2011
Primary Completion (Actual)
January 1, 2014
Study Completion (Actual)
March 1, 2014
Study Registration Dates
First Submitted
April 19, 2011
First Submitted That Met QC Criteria
April 20, 2011
First Posted (Estimate)
April 21, 2011
Study Record Updates
Last Update Posted (Estimate)
December 29, 2022
Last Update Submitted That Met QC Criteria
November 30, 2022
Last Verified
November 1, 2022
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 1042-0700
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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