CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 29 enrolled
Drug / intervention
Ganaxolone +1 moredrug
Likely dose
Not stated in record
Key inclusion· 4
  • ✓Confirmed pathogenic or likely pathogenic PCDH19 variant by molecular testing
  • ✓Inadequate seizure control despite 2 or more anti-seizure medications
  • ✓At least 12 seizures of the primary seizure type over the 12 weeks prior to screening
  • ✓On stable concomitant anti-seizure medication regimen (unchanged for 3 months prior to screening)
Key exclusion· 5
  • ✕Prior exposure to ganaxolone
  • ✕More than 8 consecutive weeks of seizure freedom during the 12 weeks before screening
  • ✕Concurrent use of strong CYP3A4/5/7 inducers or inhibitors
  • ✕Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) during the double-blind phase

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03865732
NCT03865732Phase 2Completed
CompareCT.gov

A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment.

Marinus Pharmaceuticals·interventional·Posted Mar 7, 2019·Updated Jun 26, 2023

In Brief

A Phase 2 clinical trial evaluating Ganaxolone and Placebo for PCDH19-Related Epilepsy. Completed, enrolled 29 participants across 12 sites in 5 countries.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPCDH19-Related Epilepsy
CountriesHungary, Italy, Netherlands, Poland, United States
Collaborators--

Timeline

Phase 2CompletedFinished
20192020202120222023202420252026
First PostedMar 7, 2019
Enrollment StartMay 17, 2019
Primary CompletionJan 19, 2021
Study CompletionJun 20, 2022
TodayJul 2, 2026
Enrollment to primary: 1.7 yearsPosted 7.3 years ago

Interventions

Ganaxolonedrug

active drug

Placebodrug

inactive