Marinus Pharmaceuticals announced it is initiating a single global pivotal Phase 3 clinical study (Violet Study) evaluating oral ganaxolone in children with PCDH19-related epilepsy (PCDH19-RE), a rare genetic epilepsy. If successful, the Violet Study is intended to support the regulatory filings for approval of ganaxolone in this underserved and refractory patient population.
The Violet Study is a global, double-blind, randomized, placebo-controlled pivotal Phase 3 clinical study evaluating ganaxolone in children with PCDH19-RE. The study will enroll up to 70 patients between the age of 1 and 17 with a confirmed PCDH19 mutation. All patients that meet eligibility will be stratified into one of two biomarker groups and randomized (ganaxolone or placebo) within each stratum. The trial will consist of an 8-week prospective baseline period to collect seizure data, followed by a 17-week double-blind treatment phase.
Patients randomized to ganaxolone will titrate over four weeks to a dose of up to 600 mg of ganaxolone oral liquid suspension three times a day and maintain that dose for the following 13-weeks. After the double-blind period, all patients who meet certain eligibility requirements will have the opportunity to receive ganaxolone in an open label phase of the study. The company expects to begin screening patients for enrollment into the study in the second quarter of 2019 and data from the study are estimated to be available in 2021.