Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)

Marinus Pharmaceuticals29 enrolled

Overview

A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in female children and young adults with genetically confirmed PCDH19 gene mutation.

The Violet Study is a global, double-blind, placebo-controlled, Phase 2 clinical trial that plans to enroll approximately 25 female patients between the ages of 1 and 17 with a confirmed disease-related PCDH19 gene variant. Patients will undergo a baseline period before being randomized to receive, in addition to their existing anti-seizure treatment, either ganaxolone or placebo for 17 weeks. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study's primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures will include non-seizure-related endpoints to capture certain behavioral and sleep disturbances that have been seen in previous clinical studies with ganaxolone.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

PCDH19-Related Epilepsy

Interventions

GanaxoloneDRUG

active drug

PlaceboDRUG

inactive

Eligibility

Sex: FEMALEMin age: 1 YearMax age: 17 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant * Failure to control seizures despite 2 or more anti-seizure medications * 12 seizures over a 12-week period of primary seizure types prior to screening * On a stable regimen of concomitant AEDs, Ketogenic diets, and modified Atkins diet should be unchanged for 3 months prior to screening) Exclusion Criteria: * Previous exposure to ganaxolone * \> 8 consecutive weeks of seizure freedom during the 12 weeks prior to screening * Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted * Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) is prohibited during the double-blind phase * Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening

Locations (12)

Marinus Research Site

Little Rock, Arkansas, United States

Marinus Research Site

Los Angeles, California, United States

Marinus Research Site

San Francisco, California, United States

Outcomes

Primary Outcomes

Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)

Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)

Time frame: End of the double-blind 17 week treatment period

Secondary Outcomes

Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)

Summary of 28-day Seizure Frequency for Seizure Types for Subjects in the Biomarker-positive Stratum through 17 weeks (Median Percent Change)

Time frame: [Time Frame: End of the double-blind 17 week treatment period]

50% Primary Seizure Reduction

Percent of subjects experiencing a greater than or equal to 50% reduction in 28-day primary seizure frequency relative to the 12-week baseline

Time frame: End of the double-blind 17 week treatment period

Data from ClinicalTrials.gov

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