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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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ClinicalTrials.gov Identifier: NCT02358538
Recruitment Status : Completed
First Posted : February 9, 2015
Results First Posted : September 7, 2022
Last Update Posted : September 19, 2022
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

Brief 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.

Condition or disease Intervention/treatment Phase
Epilepsy Drug: Ganaxolone Phase 2

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: 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
Actual Study Start Date : November 6, 2015
Actual Primary Completion Date : January 16, 2018
Actual Study Completion Date : January 4, 2019


Arm Intervention/treatment
Experimental: Ganaxolone
Maximum of 1800 mg/day or 63 mg/kg/day
Drug: Ganaxolone
oral suspension or capsules
Other Name: CCD 1042




Primary Outcome Measures :
  1. 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 ]
    Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change & Standard Deviation)

  2. 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 ]
    Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Median Percent Change)


Secondary Outcome Measures :
  1. 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 ]
    Clinician Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ] CGII-C scale is qualitative values and not quantitative.

  2. Summary of CGII-P [ Time Frame: Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ] ]
    Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ] CGII-P scale is qualitative values and not quantitative.

  3. Number of Participants With Responder Rate of Seizure Frequency [ Time Frame: Month 3 and Week 26 ]
    Responder Rate in Terms of 28-day Seizure Frequency Based on the Sum of Individual Seizures and Clusters

  4. 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 ]
    Mean Percentage Change of Individual Seizure-free days per 28-day period (through 52-week OLE) period relative to baseline



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02358538


Locations
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United States, Arizona
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
United States, California
Sutter Institute for Medical Research
Sacramento, California, United States, 95816
University of California San Francisco
San Francisco, California, United States, 94143
United States, Georgia
Center for Rare Neurological Diseases
Norcross, Georgia, United States, 30093
United States, Indiana
JWM Neurology
Indianapolis, Indiana, United States, 46239
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, New Jersey
Northeast Regional Epilepsy Group
Hackensack, New Jersey, United States, 07601
Institute of Neurology and Neurosurgery at St. Barnabas
Livingston, New Jersey, United States, 07039
United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Italy
Bambino Gesu Children's Hospital, IRCCS
Rome, Italy, 00165
Sponsors and Collaborators
Marinus Pharmaceuticals
  Study Documents (Full-Text)

Documents provided by Marinus Pharmaceuticals:
Study Protocol  [PDF] April 8, 2016
Statistical Analysis Plan  [PDF] July 17, 2018

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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02358538    
Other Study ID Numbers: 1042-900
First Posted: February 9, 2015    Key Record Dates
Results First Posted: September 7, 2022
Last Update Posted: September 19, 2022
Last Verified: September 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Marinus Pharmaceuticals:
PCDH19
ganaxolone
neurosteroid
CDKL5
LGS
CSWS
Additional relevant MeSH terms:
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Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Ganaxolone
Neurosteroids
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
GABA Modulators
GABA Agents