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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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27 March 2023 |
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Main ID: |
NCT02358538 |
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Date of registration:
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30/01/2015 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies
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Scientific title:
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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 |
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Date of first enrolment:
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November 6, 2015 |
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Target sample size:
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30 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT02358538 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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Italy
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United States
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Key inclusion & exclusion criteria
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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.
Age minimum:
2 Years
Age maximum:
18 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Epilepsy
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Intervention(s)
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Drug: Ganaxolone
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Primary Outcome(s)
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Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)
[Time Frame: Baseline through 52 week open label period]
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Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)
[Time Frame: Baseline through 52-week open- label period]
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Secondary Outcome(s)
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Summary of CGII-C
[Time Frame: End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78]
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Summary of CGII-P
[Time Frame: Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ]]
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Number of Participants With Responder Rate of Seizure Frequency
[Time Frame: Month 3 and Week 26]
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Mean Percentage Change of Individual Seizure-free Days
[Time Frame: Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE Period]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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