Clinical trial NCT01339689: Ganaxolone in Posttraumatic Stress Disorder (PTSD)
Official title: A Proof-of-Concept, Double-blind, Randomized, Placebo-controlled Study of Ganaxolone in Posttraumatic Stress Disorder
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: PTSD
Keywords: PTSD
Posttraumatic Stress Disorder
pharmacotherapy
ganaxolone
Lead sponsor: Marinus Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: INTRuST, Post-Traumatic Stress Disorder - Traumatic Brain Injury Clinical Consortium
name: U.S. Army Medical Research and Development Command
First posted: 2011-04-21
Enrollment: 112
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Change From Baseline in Clinician-Administered Posttrautamtic Stress Disorder (PTSD) Scale (CAPS) to Week 6
timeFrame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
Secondary outcomes:
measure: Change From Baseline in PTSD Re-Experience Score
timeFrame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
measure: Change From Baseline in PTSD Avoidance Score
timeFrame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
measure: Change From Baseline in PTSD Hyperarousal Score
timeFrame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
measure: Number of Participants With Response to Clinical Global Impression - Improvement (Investigator) (CGI-II) Scale
timeFrame: Week 2, Week 4 and Week 6
measure: Number of Participants With Response to CGI-I Subject Scale Clinical Global Impression - Improvement (Subject) (CGI-IS) Scale
timeFrame: Week 2, Week 4 and Week 6
measure: Change From Baseline in PTSD Checklist (PCL) Scores
timeFrame: Baseline (Week 0) and at Week 2, Week 4 and Week 6
measure: Change From Baseline in Profile of Mood States (POMS) Total Score
timeFrame: Baseline (Week 0) and Week 6
measure: Change From Baseline in Patient Health Questionnaire (PHQ-9) Score
timeFrame: Baseline (Week 0) and Week 6
measure: Change From Baseline in Insomnia Severity Index (ISI) Score
timeFrame: Baseline (Week 0) and Week 6
measure: Change From Baseline in Connor-Davidson Resilience Scale (CD-RISC) Score
timeFrame: Baseline (Week 0) and Week 6
Other outcomes:
Locations:
city: San Diego
country: United States
facility: VA San Diego Healthcare System/ University of California, San Diego
state: California
city: Washington
country: United States
facility: Washington DC VA Medical Center/ Uniformed Services University of the Health Services
state: District of Columbia
city: Boston
country: United States
facility: VA Boston Healthcare Services/ Spaulding Rehabilitation Hospital
state: Massachusetts
city: Manchester
country: United States
facility: Manchester VA Medical Center/ Dartmouth College
state: New Hampshire
city: Durham
country: United States
facility: Durham VA Medical Center /Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati VA Medical Center/ University of Cincinnati
state: Ohio
city: Charleston
country: United States
facility: Charleston VA Medica Center/ Medical University of South Carolina
state: South Carolina
city: White River Junction
country: United States
facility: White River Junction VA Medical Center/ Dartmouth College
state: Vermont
Study officials:
affiliation: Duke University Medical Center and Durham VA Medical Center
name: Christine E Marx, MD, MA
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston University School of Medicine Research Affiliate, National Center for PTSD
name: Ann Rasmusson, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Rasmusson AM, Marx CE, Jain S, Farfel GM, Tsai J, Sun X, Geracioti TD, Hamner MB, Lohr J, Rosse R, Summerall L, Naylor JC, Cusin C, Lang AJ, Raman R, Stein MB. A randomized controlled trial of ganaxolone in posttraumatic stress disorder. Psychopharmacology (Berl). 2017 Aug;234(15):2245-2257. doi: 10.1007/s00213-017-4649-y. Epub 2017 Jul 1.