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
Estimated Completion Date
- Drug: Ganaxolone
- Pediatrics: Pediatric Neurology
- Neurology: Seizure
- Pharmacy: Other Drugs
Marinus Pharmaceuticals -- 1042-900
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
3. Have confirmed PCDH19 genetic mutation.
4. Have uncontrolled cluster seizures and/or non-clustered focal dyscognitive or focal
5. Subjects should be on a stable regimen of anti-epileptic medication, and generally in
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)
|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||Noneemail@example.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|