Ganaxolone in Posttraumatic Stress Disorder (PTSD)

NCT01339689 | Completed Phase 2 Results DMC

• 1 other identifier: 1042-0700
• Study Type: interventional
• Target: 112
• Locations: 1 country
• Sites: 8

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.

Conditions

PTSD

Keywords

PTSD; Posttraumatic Stress Disorder; pharmacotherapy; ganaxolone

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline (Week 0) and at Week 2, Week 4 and Week 6

Secondary Outcomes (10)

• Change From Baseline in PTSD Re-Experience Score

  Baseline (Week 0) and at Week 2, Week 4 and Week 6

• Change From Baseline in PTSD Avoidance Score

  Baseline (Week 0) and at Week 2, Week 4 and Week 6

• Change From Baseline in PTSD Hyperarousal Score

  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

  Week 2, Week 4 and Week 6

• 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

Study Arms (2)

Ganaxolone

EXPERIMENTAL

active

Drug: Ganaxolone

Placebo

PLACEBO COMPARATOR

non-active

Drug: Placebo

Interventions

Ganaxolone DRUG

200-600 mg bid, capsules, up to 12 weeks

Also known as: GNX

Ganaxolone

Placebo DRUG

capsules, bid, up to 12 weeks

Also known as: PBO

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead
• INTRuST, Post-Traumatic Stress Disorder - Traumatic Brain Injury Clinical Consortium: collaborator
• U.S. Army Medical Research and Development Command: collaborator

Study Sites (8)

VA San Diego Healthcare System/ University of California, San Diego

San Diego, California, 92093, United States

Location

Washington DC VA Medical Center/ Uniformed Services University of the Health Services

Washington D.C., District of Columbia, 20422, United States

Location

VA Boston Healthcare Services/ Spaulding Rehabilitation Hospital

Boston, Massachusetts, 02130, United States

Location

Manchester VA Medical Center/ Dartmouth College

Manchester, New Hampshire, 03104, United States

Location

Durham VA Medical Center /Duke University Medical Center

Durham, North Carolina, 27705, United States

Location

Cincinnati VA Medical Center/ University of Cincinnati

Cincinnati, Ohio, 45220, United States

Location

Charleston VA Medica Center/ Medical University of South Carolina

Charleston, South Carolina, 29401, United States

Location

White River Junction VA Medical Center/ Dartmouth College

White River Junction, Vermont, 05009, United States

Location

Related Publications (1)

• 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.

  PMID: 28667510 DERIVED

MeSH Terms

Conditions

Stress Disorders, Post-Traumatic

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Stress Disorders, Traumatic; Trauma and Stressor Related Disorders; Mental Disorders

Results Point of Contact

• Title: Marinus Clinical Trials Submission Manager
• Organization: Marinus Pharmaceuticals, Inc.

clinicaltrials@marinuspharma.com

484-801-4670

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 19, 2011
• First Posted: April 21, 2011
• Study Start: April 1, 2011
• Primary Completion: January 1, 2014
• Study Completion: March 1, 2014
• Last Updated: December 29, 2022
• Results First Posted: December 29, 2022

Record last verified: 2022-11

Locations

US (8)