Status:
COMPLETED
Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
Lead Sponsor:
Marinus Pharmaceuticals
Conditions:
PCDH19-Related Epilepsy
Eligibility:
FEMALE
1-17 years
Phase:
PHASE2
Brief 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 genetica...
Detailed Description
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-relat...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
May 17 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 20 2022
Estimated Enrollment :
29 Patients enrolled
Trial Details
Trial ID
NCT03865732
Start Date
May 17 2019
End Date
June 20 2022
Last Update
June 26 2023
Active Locations (12)
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1
Marinus Research Site
Little Rock, Arkansas, United States, 72202
2
Marinus Research Site
Los Angeles, California, United States, 90095
3
Marinus Research Site
San Francisco, California, United States, 94158
4
Marinus Research Site
Durham, North Carolina, United States, 27710