Phase 2
N=21
Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)
PCDH19-Related Epilepsy
Bottom Line
View on ClinicalTrials.gov: NCT03865732 ↗Enrolled (actual)
21
Serious AEs
28.6%
Results posted
Sep 2022
Primary outcome: Primary: Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change) — -23.97; -61.52 Median % Change in Number of Seizures
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ganaxolone (Drug); Placebo (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- Female
- Sponsor
- Marinus Pharmaceuticals
- Primary completion
- Jan 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change) |
-23.97; -61.52 | — |
| SECONDARY Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change) |
-18.71; -35.90 | — |
| SECONDARY 50% Primary Seizure Reduction |
4; 5 | — |
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.
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT03865732). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.