Trial record 1 of 1 for:    A Multicenter, Double Blind, Randomized, Placebo Controlled Trial to Determine the Efficacy and Safety of Ganaxolone as Adjunctive Therapy for Adults with Drug-Resistant Partial-Onset Seizures
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Phase 2 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures, With Long-term Open-label Extension

This study is currently recruiting participants. (see Contacts and Locations)
Verified July 2014 by Marinus Pharmaceuticals
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01963208
First received: October 11, 2013
Last updated: July 18, 2014
Last verified: July 2014
  Purpose

The study will evaluate the effectiveness and safety of an investigational drug-ganaxolone - on partial seizure frequency in adults with epilepsy taking a maximum of 3 antiepileptic medications (AEDs).


Condition Intervention Phase
Drug Resistant Partial Onset Seizure
Drug: ganaxolone
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Multicenter, Double Blind, Randomized, Placebo-Controlled Trial to Determine the Efficacy and Safety of Ganaxolone as Adjunctive Therapy for Adults With Drug-Resistant Partial-Onset Seizures Followed by Long-term Open-Label Treatment

Resource links provided by NLM:


Further study details as provided by Marinus Pharmaceuticals:

Primary Outcome Measures:
  • Percentage change in seizure frequency per 28 days in the double blind period relative to baseline [ Time Frame: 14 wks (cohort 2) ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in seizure frequency per 28 days [ Time Frame: 9 wks (Cohort 1), 14 wks (Cohort 2) and 52 weeks (both) ] [ Designated as safety issue: No ]
  • Proportion of responders experiencing a ≥50% reduction from baseline to the end of the period, in total partial seizure frequency per 28 days [ Time Frame: 9 wks (cohort 1), 14 wks (cohort 2) and 52 weeks (both) ] [ Designated as safety issue: No ]
  • Proportion of seizure free subjects [ Time Frame: 9 wks (cohort 1), 14 wks (cohort 2) and 52 weeks (both ] [ Designated as safety issue: No ]
  • Clinical Global Impression of change [ Time Frame: 9 wks (cohort 1), 14 wks (cohort 2) and 52 weeks (both ] [ Designated as safety issue: Yes ]
  • Patient Global Impression of change [ Time Frame: 9 wks (cohort 1), 14 wks (cohort 2) and 52 weeks (both ] [ Designated as safety issue: Yes ]
  • Change in the number of seizure-free days [ Time Frame: 9 wks (cohort 1), 14 wks (cohort 2) and 52 weeks (both ] [ Designated as safety issue: No ]

Estimated Enrollment: 150
Study Start Date: October 2013
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: June 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ganaxolone
active
Drug: ganaxolone
200 mg and 225 mg capsules 2x/day
Other Name: gnx
Placebo Comparator: Placebo
placebo, non-active
Drug: Placebo
placebo
Other Name: pbo

Detailed Description:

This is a 2-cohort study comprised of 2 phases in each cohort. Phase 1 is a double-blind phase followed by Phase 2, an open-label phase. Cohort 1 will provide tolerability, safety, and PK information for ganaxolone 1200mg/d, 1800mg/d and placebo. Cohort 2 will investigate the efficacy, tolerability and safety of ganaxolone 1800mg/d compared to placebo. Cohort 1 (N= approximately 50) will enroll into a 67-week study comprised of a 4-week prospective baseline period plus 4 week retrospective baseline followed by two treatment phases: a 9-week randomized DB placebo-controlled treatment phase followed by a 52-week open label (OL) treatment phase. Cohort 2 (N=150) will enroll into a 72-week study comprised of a 4-week prospective baseline period plus 4 week retrospective baseline followed by two treatment phases: a 14-week randomized DB placebo-controlled treatment phase followed by a 52-week open label (OL) treatment phase.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Able to give informed consent in writing, or have a legally authorized representative able to do so
  • Willing to enter and participate for the full term of the double blind phase and willing to enter into the open-label phase
  • Male or female outpatients > 18 years of age
  • Have a confident diagnosis of drug-resistant epilepsy with partial-onset seizures (POS), with or without secondary generalization, for ≥2 years. Have residual POS despite having been treated in the past with at least 2 approved anti-epilepsy drugs (AEDs) either alone or in combination
  • Based on history, subjects would be anticipated to have at least 3 POS during each 4-week Baseline period and unlikely to have 21 or more consecutive POS-free days
  • Currently being treated and maintained with a stable regimen of 1, 2, or 3 AEDs
  • Able and willing to maintain daily seizure calendar
  • Able and willing to take drug with food twice daily
  • Sexually active women of childbearing potential must use acceptable birth control and have a negative pregnancy test at all visits

Exclusion Criteria:

  • Have had previous exposure to ganaxolone
  • Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds
  • Exposure to any investigational drug or device < 30 days prior to screening, or plans to take another investigational drug at any time during the study
  • Time of onset of epilepsy treatment < 2 years prior to enrollment
  • Have generalized epilepsy, such as Lennox-Gastaut syndrome, juvenile myoclonic epilepsy, absence epilepsy, or non-epileptic seizures within the last 12- month period prior to study entry
  • Have less than 3 POS seizures in a 28-day period or more than 21 consecutive seizure-free days during the Baseline period
  • Have only simple partial seizures without any observable motor component
  • Have innumerable seizures or status epilepticus within the last 12-months prior to screening
  • Have more than 100 POS per 4-week Baseline period
  • Have seizures secondary to illicit drug or alcohol use, infection, neoplasm, demyelinating disease, degenerative neurological disease, or central nervous system (CNS) disease deemed progressive, metabolic illness, or progressive degenerative disease
  • Current use of vigabatrin is not permitted. If prior use of vigabatrin, must have documented stable visual fields
  • Current use of ezogabine is not permitted. If prior use, must have been off the medication for at least 3 months prior to screening and have had documented normal fundoscopic exam by ophthalmologist
  • Are planning surgery, or to be evaluated for surgery, during the double blind phase to control seizures including VNS implantation
  • Are suffering from acute or progressive neurological disease, moderate or severe psychiatric disease, or severe mental abnormalities that are likely to require changes in drug therapy during the double blind portion of the study or interfere with the objectives of the study or the ability to adhere to the protocol requirements
  • Have a history of an actual suicide attempt in the last 5 years or more than 1 lifetime suicide attempt
  • Have a positive urine drug screen at Screening or meet criteria for current or historical Substance Use Disorder (DSM-V criteria) within the past 5 years.
  • Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome, including but not limited to: clinically significant cardiac, renal, pulmonary, gastrointestinal, hematologic or hepatic conditions; or a condition that affects the absorption, distribution, metabolism or excretion of drugs
  • Have elevated ALT (SGPT) or AST (SGOT) greater than 3 times upper limits of normal, or total bilirubin greater than 1.5 time ULN
  • Have a history of malignancy within the past 2 years, with the exception of basal cell carcinoma
  • Are currently following or planning to follow a ketogenic diet
  • Use of dietary supplements or herbal preparations are not permitted if subject has been using them consistently for less than 6 months prior to screening, or does not plan on remaining on stable doses for the duration of the double blind phase. Use of St. John's Wort is not permitted
  • Females who are pregnant, currently breastfeeding or planning to become pregnant during the duration of the study
  • A history of chronic noncompliance with drug regimens
  • Inability to withhold grapefruit and grapefruit juice from diet during the entire clinical trial
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01963208

Contacts
Contact: Julia Tsai, Ph.D. 203-315-5820 jtsai@marinuspharma.com

Locations
United States, Alabama
University of Alabama Epilepsy Center Recruiting
Birmingham, Alabama, United States, 35294-3280
Contact: Cheryl R Hall, CCRC    205-934-1654    Chall@uab.edu   
Contact: Jennifer DeWolfe, MD    205-934-1654      
Principal Investigator: Jennifer DeWolfe, MD         
United States, California
Neuro-Pain Medical Center, Inc Recruiting
Fresno, California, United States, 93710
Contact: Phillip Escamilla    559-437-9700    neurophillip@yahoo.com   
Contact: Perminder Bhatia, MD    559-437-9700      
Principal Investigator: Perminder Bhatia, MD         
Neurological Research Institute Recruiting
Santa Monica, California, United States, 90404
Contact: Erica Espinosa    310-315-1456    eespinosa@drkudrow.com   
Contact: Jaime Latorre    310-315-1456    jlatorre@drkudrow.com   
Principal Investigator: David Kudrow, MD         
United States, Colorado
University of Colorado- Anschutz Outpatient Pavilion Recruiting
Aurora, Colorado, United States, 80045
Contact: Kristen Lervik    303-724-7309    kristen.lervik@ucdenver.edu   
Contact: Laura Strom, MD    303.724.7309      
Principal Investigator: Laura Strom, MD         
United States, Florida
Medsol Clinical Research Center Recruiting
Port Charlotte, Florida, United States, 33952
Contact: Maria Vasconcelos    941-623-9744    mvasconcelos@medsolcrc.com   
Contact: Susan Coldiron    941-623-9744    scoldiron@medsolcrc.com   
Principal Investigator: George Li, MD         
United States, Kentucky
Bluegrass Epilepsy Research, LLC Recruiting
Lexington, Kentucky, United States, 40504
Contact: Amber Chumley    859-313-4273    bgepilepsy@outlook.com   
Contact: Toufic Fakhoury, MD    859.313.4273    bgepilepsy@outlook.com   
Principal Investigator: Toufic Fakhoury, MD         
United States, Maryland
Mid-Atlantic Epilepsy Center Recruiting
Bethesda, Maryland, United States, 20817
Contact: Arkady Barber    301-530-9744    barbera@epilepsydc.com   
Contact: Lenka Goldman    301-530-9744      
Principal Investigator: Pavel Klein, MD         
United States, Minnesota
Minneapolis Clinic of Neurology Recruiting
Golden Valley, Minnesota, United States, 55422
Contact: Kathy Sowell, MA    763-302-4135    kathy.sowell@mpls-clinic.com   
Contact: Katrina Miller    763-302-4135      
Principal Investigator: Joanne Rogin, MD         
United States, Missouri
The Comprehensive Epilepsy Care Center for Children and Adults Recruiting
Chesterfield, Missouri, United States, 63017
Contact: Patty Schaefer    314-453-9300    werosenfeld@msn.com   
Principal Investigator: William Rosenfeld, MD         
United States, New Jersey
Cooper Medical Center of Rowan University Recruiting
Camden, New Jersey, United States, 08103
Contact: Tamara Lee    856-968-7222    Lee-tamara@cooperhealth.edu   
Contact: Cory Hackmyer    856-342-2460    Hackmyer-Cory@CooperHealth.edu   
Principal Investigator: Evren Burakgazi-Dalkilic, MD         
Northeast Regional Epilepsy Group Recruiting
Hackensack, New Jersey, United States, 07601
Contact: Munazza Malik, MD    201-343-6676 ext 122    mmalik@epilepsygroup.com   
Contact: Evan Fertig, M.D.    201-343-6676      
Principal Investigator: Evan Fertig, M.D.         
United States, New York
Winthrop University Hospital Recruiting
Mineola, New York, United States, 11501
Contact: Kim Brynes    516-663-2815    kbyrnes@winthrop.org   
Principal Investigator: Shicong Ye, MD         
New York University Comprehensive Epilepsy Center Recruiting
New York, New York, United States, 10016
Contact: Stephanie Tenzigolski    646-558-0842    Stephanie.Tenzigolski@nyumc.org   
Contact: Maria Hopkins       maria.hopkins@nyumc.org   
Principal Investigator: Blanca Vazquez, MD         
United States, Ohio
Ohio Clinical Research Partners, LLC Recruiting
Canton, Ohio, United States, 44718
Contact: Jennifer Simpkins-Krec    330-494-2097 ext 138    JKrec@SSandG.com   
Contact: Wendy Shaffer    330-494-2097 ext 138    WShaffer@SSandG.com   
Principal Investigator: Jennifer Drake, DO         
Ohio State University Recruiting
Columbus, Ohio, United States, 43221
Contact: Stephanie Scarberry, RN    614-688-4678    Stephanie.Scarberry@osumc.edu   
Principal Investigator: Bassel Shneker, MD         
United States, Oklahoma
Lynn Health Institute Recruiting
Oklahoma City, Oklahoma, United States, 73112
Contact: Aimee Freyman    405-602-3928    afreyman@lhsi.net   
Contact: Phyllis Fahrenbruch    405-602-3939    pmorris@lhsi.net   
Principal Investigator: Mark Fisher, MD         
United States, Pennsylvania
Temple University School of Medicine Recruiting
Philadelphia, Pennsylvania, United States, 19140
Contact: Brian Augelli    215-707-7310    brian.augelli@tuhs.temple.edu   
Principal Investigator: Mercedes Jacobson, MD         
Jefferson Comprehensive Epilepsy Center Recruiting
Philadelphia, Pennsylvania, United States, 19107
Contact: Gillian Hewitt    215-503-3166    Gillian.Hewitt@jefferson.edu   
Contact: Pranoti Pradhan    215-955-2245    Pranoti.Pradhan@Jefferson.edu   
Principal Investigator: Michael Sperling, MD         
United States, Texas
Texas Epilepsy Group Recruiting
Dallas, Texas, United States, 75251
Contact: Joyce Brown    972-707-8378    jbrown@texasepilepsy.org   
Contact: Tawaika Sneed    972-707-8378    tsneed@texasepilepsy.org   
Principal Investigator: Jay Harvey, MD         
United States, Washington
Rainier Clinical Research Center, Inc. Recruiting
Renton, Washington, United States, 98057
Contact: Carole Burton, RN    425-251-1720    cdburton@rainier-research.com   
Principal Investigator: David Vossler, MD         
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
Study Director: Gail Farfel, PhD Marinus Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01963208     History of Changes
Other Study ID Numbers: 1042-0603
Study First Received: October 11, 2013
Last Updated: July 18, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Marinus Pharmaceuticals:
partial onset seizures
complex partial seizures
simple partial seizures
anticonvulsant
ganaxolone
neurosteroid
Marinus
epilepsy

Additional relevant MeSH terms:
Seizures
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms

ClinicalTrials.gov processed this record on September 14, 2014