A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy


To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation in an open-label proof-of-concept study.

Study Start Date

March 2015

Estimated Completion Date

June 2017


  • Drug: Ganaxolone


  • Pediatrics: Pediatric Neurology
  • Neurology: Seizure
  • Pharmacy: Other Drugs

MeSH Terms

  • Epilepsy
  • Ganaxolone

Study ID

Marinus Pharmaceuticals -- 1042-900



Trial ID


Study Type


Trial Phase

Phase 2

Enrollment Quota



Marinus Pharmaceuticals


Inclusion Criteria

    1. Have parent or legal guardian available and willing to give written informed consent. 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 and/or non-clustered focal dyscognitive or focal convulsive seizures. 5. Subjects should be on a stable regimen of anti-epileptic medication, and generally in good health. 6. Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar. 7. Able and willing to take study medication with food, two or three times daily. Key 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 < 90 days prior to screening, or plans to participate in another drug or device trial at any time during the study. 5. Concurrent use of vigabatrin, tiagabine, or ezogabine is not permitted. 6. 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. 7. 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. 8. 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. 9. Are currently following or planning to follow a ketogenic diet.




2 Years to 10 Years

Accepts Healthy Volunteers


Study Locations and Contact Information (6)

Study Location Distance Name Phone Email
Boston Childrens Hospital - Boston, Massachusetts 2.6 miles None None None
JWM Neurology - Indianapolis, Indiana 798.4 miles None None None
Nicklaus Childrens Hospital - Miami, Florida 1,265.3 miles Claudia Rodriguez None Claudia.RodriguezPaez@mch.com
Phoenix Childrens Hospital - Phoenix, Arizona 2,294.8 miles Penny Overgaard None povergaard@phoenixchildrens.com
Sutter Institute for Medical Research - Sacramento, California 2,628.2 miles A Linh DangChu 916-733-4482 DangAL1@sutterhealth.org
University of California San Francisco - San Francisco, California 2,699.8 miles None None None

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