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

• Status: Active, not recruiting
• Trial end date: 2022-05-31
• Target enrollment: 0
• Participant gender: Female

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Marinus Pharmaceuticals
• Treatments: Ganaxolone; Pregnanolone

Criteria

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