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

Sponsor

Marinus Pharmaceuticals (Industry)

Overall Status

Terminated

CT.gov ID

NCT02519439

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

5.2

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

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.

Study Design

Study Type:

Interventional

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 :

Feb 1, 2015

Actual Primary Completion Date :

Dec 1, 2016

Actual Study Completion Date :

Dec 1, 2016

Arms and Interventions

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 Names: CCD 1042

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 Names:

CCD 1042

Outcome Measures

Primary Outcome Measures

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 [104 Weeks]

Secondary Outcome Measures

To assess efficacy of adjunctive ganaxolone during a two-year open-label treatment in adult subjects with drug-resistant partial-onset seizures [104 Weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Neurological Research Institute	Santa Monica	California	United States	90404
2	Bluegrass Epilepsy Research, LLC	Lexington	Kentucky	United States	40504
3	Minneapolis Clinic of Neurology	Golden Valley	Minnesota	United States	55422
4	Northeast Regional Epilepsy Group	Hackensack	New Jersey	United States	07601
5	Texas Epilepsy Group	Dallas	Texas	United States	75251