PCDH19 Female Pediatric Epilepsy (FPE) MedDRA version: 18.0 Level: LLT Classification code 10032062 Term: Other forms of epilepsy, with intractable epilepsy System Organ Class: 100000004852
Conditions
Interventions
Product Name: ganaxolone
Product Code: CCD 1042, MD 9150000, Mepalon 1042, SPT316
Pharmaceutical Form: Oral suspension
INN or Proposed INN: GANAXOLONE
CAS Number: 38398-32-2
Current Sponsor code: CCD
Sponsors
Marinus Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Have parent or legal guardian available and willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures. 2. Female outpatients > 2 years of age and younger than 11 years of age at time of consent. 3. Have confirmed PCDH19 genetic mutation. 4. Have uncontrolled cluster seizures (approximately 10 or more seconds between seizures), lasting for one hour or more per episode, with inter-cluster episodes of not more than 6 weeks, AND/OR, non-clustered focal dyscognitive or focal convulsive seizures with a frequency =4 seizures per 28-day period during baseline. 5. Subjects should be on a stable regimen of AED medication, and generally in good health. 6. Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar for the duration of the study. 7. Able and willing to take study medication with food, two or three times daily. Ganaxolone must be administered with food. 8. Subject must be approved to participate by Sponsor and Principal Investigator after review of medical history and baseline seizure calendars. Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Have had previous exposure to ganaxolone. 2. Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds. 3. Have SCN1A genetic mutation. 4. Exposure to any investigational drug or device 3 times upper limits of normal (ULN), or total bilirubin >1.5 time ULN at the baseline visit. 10. Are currently following or planning to follow a ketogenic diet. 11. Is sexually active or pregnant. 12. Unwilling to forgo grapefruit and grapefruit juice from diet during the entire clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation.;Secondary Objective: To evaluate the safety and tolerability of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation.;Primary end point(s): Percentage change in seizure (focal dyscognitive or focal convulsive) frequency per 28 days relative to baseline.;Timepoint(s) of evaluation of this end point: 28 days from calculated baseline. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy: Evaluation of inter-cluster interval calculated using daily seizure diary. Seizure-free interval calculated using daily seizure diary. Proportion of responders by cohort calculated using daily seizure diary. Proportion of subjects whose inter-cluster interval increases by cohort calculated using daily seizure diary. Time to reach baseline number of seizures (per 28 days) calculated using daily seizure diary. Clinician Global Impression of Change score as assessed by questionnaire. Patient Global Impression of Change score as assessed by questionnaire. Safety: Evaluation of safety and tolerability of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation, based on adverse event log and other clinical safety assessments. ;Timepoint(s) of evaluation of this end point: Efficacy: 26 weeks Safety: 30 weeks | — |
Countries
Italy, United States
Contacts
Public ContactMedical Representative
Marinus Pharmaceuticals, Inc.
Outcome results
None listed