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

Completed · Phase 2 · Has a placebo group

Conditions studied: PCDH19-Related Epilepsy

In brief

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.

Key facts

Study ID

NCT03865732

Run by

Marinus Pharmaceuticals

People needed

29

Starts

2019-05-17

Expected to finish

2022-06-20

Last updated by the study team

2023-06-26

Who can join

Age: 1 and older, up to 17. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Marinus Research Site — Little Rock, Arkansas, United States
• Marinus Research Site — Los Angeles, California, United States
• Marinus Research Site — San Francisco, California, United States
• Marinus Research Site — Durham, North Carolina, United States
• Marinus Research Site — York, Pennsylvania, United States
• Marinus Research Site — Salt Lake City, Utah, United States
• Marinus Research Site — Budapest, Hungary
• Marinus Research Site — Florence, Italy
• Marinus Research Site — Rome, Italy
• Marinus Research Site — Heeze, Netherlands
• Marinus Research Site — Zwolle, Netherlands
• Marinus Research Site — Krakow, Poland

Full record on ClinicalTrials.gov

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