Phase 2
N=30
Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
Epilepsy
Bottom Line
View on ClinicalTrials.gov: NCT02358538 ↗Enrolled (actual)
30
Serious AEs
23.3%
Results posted
Sep 2022
Primary outcome: Primary: Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation) — -31.23; NA; 122.10; 52.83 percentage of change of frequency
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Ganaxolone (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Marinus Pharmaceuticals
- Primary completion
- Jan 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation) |
-31.23; NA; 122.10; 52.83; -20.55; NA | — |
| PRIMARY Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change) |
-47.34; -10.22; -25.98; NA; -37.70; -9.19 | — |
| SECONDARY Summary of CGII-C |
0; 2; 1; NA; 3; 4 | — |
| SECONDARY Summary of CGII-P |
0; 3; 1; NA; 1; 2 | — |
| SECONDARY Number of Participants With Responder Rate of Seizure Frequency |
4; 4; 6; NA; 3; 2 | — |
| SECONDARY Mean Percentage Change of Individual Seizure-free Days |
11.84; -1.12; 7.87; NA; 11.80; -2.13 | — |
Summary
To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation and other rare genetic epilepsies in an open-label proof-of-concept study.
Eligibility Criteria
Key Inclusion Criteria
- Have parent or legal guardian available and willing to give written informed consent.
- Male and female outpatients between 2 and 18 years of age years of age at time of consent.
- Have any of the following epilepsy syndromes: PCDH19; CDKL5; Dravet Syndrome; Lennox Gastaut Syndrome (LGS); Continuous Spikes and Waves during Sleep (CSWS)
- Have uncontrolled cluster seizures and/or non-clustered seizures.
- Subjects should be on a stable regimen of anti-epileptic medication, and generally in good health.
- Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar.
- Able and willing to take study medication with food, two or three times daily.
Key Exclusion Criteria
- Have had previous exposure to ganaxolone.
- Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds.
- Exposure to any investigational drug or device 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN at the screening and baseline visits.
Data sourced from ClinicalTrials.gov (NCT02358538). 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.