Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Ra… (NCT02358538) | Clinical Trial Compass
CompletedPhase 2
Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
United States30 participantsStarted 2015-11-06
Plain-language 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.
Who can participate
Age range2 Years – 18 Years
SexALL
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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.
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 \< 90 days prior to screening, or plans to participate in another drug or device trial at any time during the study.
✕. Concurrent use of vigabatrin, tiagabine, or ezogabine is not permitted.
✕. Have any medical condition that, in the investigator's judgment, is considered to be clinically significant and could potentially affect subject safety or study outcome, including but not limited to: clinically significant cardiac, renal, pulmonary, gastrointestinal, hematologic or hepatic conditions; or a condition that affects the absorption, distribution, metabolism or excretion of drugs.
What they're measuring
1
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
Timeframe: Baseline through 52 week open label period
2
Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
Timeframe: Baseline through 52-week open- label period
✕. Have active suicidal plan/intent, or have had active suicidal thoughts in the past 6 months or a suicide attempt in the past 3 years.
✕. Have Alanine transferase (ALT; SGPT) or Aspartate transferase (AST; SGOT) levels \> 3 times upper limits of normal (ULN), or total bilirubin \>1.5 time ULN at the screening and baseline visits.