a two-year open-label extension study of ganaxolone in patients with drug-resistant partial-onset seizures

Conditions: Drug Resistant Partial Onset Seizure

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

this study is a 2-year, open-label continuation for those patients benefiting from ganaxolone treatment after completing protocol 1042-0603.

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.

Keywords: partial onset seizures complex partial seizures simple partial seizures anticonvulsant ganaxolone neurosteroid marinus epilepsy