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A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02519439
Recruitment Status : Terminated (Ganaxolone missed its primary endpoint in the double-blind portion of the 1042-0603 study. Due to this outcome Marinus discontinued this extension study.)
First Posted : August 11, 2015
Last Update Posted : August 26, 2020
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

Brief Summary:
A follow-on, two-year open-label extension study of ganaxolone as add-on therapy in adult patients with drug-resistant partial-onset seizures

Condition or disease Intervention/treatment Phase
Drug Resistant Partial Onset Seizure Drug: ganaxolone Phase 3

Detailed Description:
This study is a 2-year, open-label continuation for those patients benefiting from ganaxolone treatment after completing Protocol 1042-0603.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A follow-on, Two-year Open-label Extension Study of Ganaxolone as add-on Therapy in Adult Patients With Drug-resistant Partial-onset Seizures
Study Start Date : February 2015
Actual Primary Completion Date : December 2016
Actual Study Completion Date : December 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Seizures

Arm Intervention/treatment
Experimental: ganaxolone
Up to a maximum of 1800 mg/day
Drug: ganaxolone
225 mg capsules 450 mg to 900 mg 2x/day
Other Name: CCD 1042




Primary Outcome Measures :
  1. To assess the safety and tolerability of adjunctive ganaxolone during a two-year open-label treatment in adult subjects with drug-resistant partial-onset seizures [ Time Frame: 104 Weeks ]

Secondary Outcome Measures :
  1. To assess efficacy of adjunctive ganaxolone during a two-year open-label treatment in adult subjects with drug-resistant partial-onset seizures [ Time Frame: 104 Weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Subjects who have completed all scheduled clinical study visits in the previous protocol 1042-0603 and have shown a minimum 35% improvement in mean 28-day seizure frequency over the last three 28-day periods in study 1042-603 as compared to the baseline of study 1042-603.
  • Subjects whose daily study drug compliance in Study 1042-0603 was 90% or greater, and for whom the investigator feels that the subject was compliant with the full dose as prescribed.
  • Able to give informed consent in writing, or have a legally authorized representative able to do so, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
  • Currently being treated and maintained with a stable regimen of 1, 2, or 3 anti-epileptic drugs (AED) at a consistent dose for one month prior to study entry.
  • Implanted Vagus Nerve Stimulator (VNS) is permitted and will not count towards the number of concomitant AEDs.
  • Able and willing to maintain an accurate and complete daily written seizure calendar or has a caregiver who is able and willing to maintain an accurate and complete daily written seizure calendar.
  • Able and willing to take drug with food twice daily. Ganaxolone must be administered with food.
  • Sexually active women of childbearing potential (WCBP) must be using a medically acceptable method of birth control and have a negative pregnancy test at Visit 1 and at subsequent visits.

Exclusion Criteria:

  • 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
  • Experienced a Serious Adverse Event or a moderate or severe medically important adverse event judged probably or definitely related to open-label ganaxolone in the previous study, 1042-0603
  • Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels > 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN during Study 1042-0603.
  • Have a history of malignancy within the past 2 years, with the exception of basal cell carcinoma.
  • 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.
  • Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months. Have a history of an actual suicide attempt in the last 5 years or more than 1 lifetime actual suicide attempt as classified by the Columbia-Suicide Severity Rating Scale (C-SSRS).
  • Have a history of drug or alcohol abuse within the past 5 years. As with other AEDs, the use of alcohol is not advised.
  • Are currently following or planning to follow a ketogenic diet.
  • Current use of vigabatrin or ezogabine (retigabine; Potiga; Trobalt) is not permitted.
  • Females who are pregnant, currently breastfeeding or planning to become pregnant during the study.
  • Inability/unwillingness to withhold grapefruit and grapefruit juice from diet during the entire clinical trial.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02519439


Locations
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United States, California
Neurological Research Institute
Santa Monica, California, United States, 90404
United States, Kentucky
Bluegrass Epilepsy Research, LLC
Lexington, Kentucky, United States, 40504
United States, Minnesota
Minneapolis Clinic of Neurology
Golden Valley, Minnesota, United States, 55422
United States, New Jersey
Northeast Regional Epilepsy Group
Hackensack, New Jersey, United States, 07601
United States, Texas
Texas Epilepsy Group
Dallas, Texas, United States, 75251
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
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Study Director: Joseph Hulihan, M.D. Marinus Pharmaceuticals
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02519439    
Other Study ID Numbers: 1042-0604
First Posted: August 11, 2015    Key Record Dates
Last Update Posted: August 26, 2020
Last Verified: August 2020
Keywords provided by Marinus Pharmaceuticals:
Partial Onset Seizures
complex partial seizures
simple partial seizures
anticonvulsant
ganaxolone
neurosteroid
Marinus
epilepsy
Additional relevant MeSH terms:
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Seizures
Neurologic Manifestations
Nervous System Diseases