A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures
Condition: Drug Resistant Partial Onset Seizure
MeSH terms: Seizures
Intervention: ganaxolone
Intervention description: 225 mg capsules 450 mg to 900 mg 2x/day
Intervention MeSH terms: Ganaxolone
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 26.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Partial Onset Seizures; complex partial seizures; simple partial seizures; anticonvulsant; ganaxolone; neurosteroid; Marinus; epilepsy

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
    

Eligibility 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.