A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies



Status:Completed
Conditions:Neurology, Epilepsy
Therapuetic Areas:Neurology, Other
Healthy:No
Age Range:2 - 18
Updated:3/30/2019
Start Date:March 2015
End Date:January 4, 2019

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A Multicenter, Open-Label Proof-of-Concept Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies Followed by 52 Week Open-Label Treatment

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.

The purpose of this proof-of-concept study is to evaluate ganaxolone as adjunctive therapy
for uncontrolled seizures in female children with PCDH19 mutations and other rare genetic
epilepsies. After establishing baseline seizure frequency, qualifying subjects will enter the
study and be treated with open-label ganaxolone for up to six months.

Key Inclusion Criteria:

1. Have parent or legal guardian available and willing to give written informed consent.

2. Male and female outpatients between 2 and 18 years of age years of age at time of
consent.

3. Have any of the following epilepsy syndromes: PCDH19; CDKL5; Dravet Syndrome; Lennox
Gastaut Syndrome (LGS); Continuous Spikes and Waves during Sleep (CSWS)

4. Have uncontrolled cluster seizures and/or non-clustered 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. 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.

4. Concurrent use of vigabatrin, tiagabine, or ezogabine is not permitted.

5. 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.

6. 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.

7. 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.
We found this trial at
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sites
1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Randa Jarra, MD
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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700 Childrens Drive
Columbus, Ohio 43205
(616) 722-2000
Nationwide Children's Hospital At Nationwide Children’s, we are creating the future of pediatric health care....
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Columbus, OH
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Annapurna Poduri, MD
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Hackensack, New Jersey 07601
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Indianapolis, Indiana 46237
Principal Investigator: Keith Ridel, MD
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Indianapolis, IN
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Livingston, NJ
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5600 Oakbrook Parkway
Norcross, Georgia 30093
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Norcross, GA
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Rome,
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Sacramento, California 95816
Principal Investigator: Michael Chez, MD
Phone: 916-733-4482
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San Francisco, California 94143
Principal Investigator: Joseph Sullivan, MD
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San Francisco, CA
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