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Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)

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ClinicalTrials.gov Identifier: NCT03865732
Recruitment Status : Completed
First Posted : March 7, 2019
Results First Posted : September 8, 2022
Last Update Posted : September 8, 2022
Sponsor:
Information provided by (Responsible Party):
Marinus Pharmaceuticals

Study Description
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Brief Summary:
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in female children and young adults with genetically confirmed PCDH19 gene mutation.

Condition or disease Intervention/treatment Phase
PCDH19-Related Epilepsy Drug: Ganaxolone Drug: Placebo Phase 2

Detailed Description:
The Violet Study is a global, double-blind, placebo-controlled, Phase 2 clinical trial that plans to enroll approximately 25 female patients between the ages of 1 and 17 with a confirmed disease-related PCDH19 gene variant. Patients will undergo a baseline period before being randomized to receive, in addition to their existing anti-seizure treatment, either ganaxolone or placebo for 17 weeks. Following the treatment period, all patients that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study's primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures will include non-seizure-related endpoints to capture certain behavioral and sleep disturbances that have been seen in previous clinical studies with ganaxolone.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 29 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: The double-blind phase will randomize subjects to adjunctive ganaxolone or placebo at a 1:1 ratio to standard of care.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment.
Actual Study Start Date : May 17, 2019
Actual Primary Completion Date : January 19, 2021
Actual Study Completion Date : June 20, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy Seizures

Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo
placebo suspension 3x's /day for 17 weeks
 Drug: Placebo
inactive
Other Name: Placebo (for ganaxolone)
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
 Drug: Ganaxolone
active drug


Outcome Measures
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Primary Outcome Measures :
  1. Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change) [ Time Frame: End of the double-blind 17 week treatment period ]
    Summary of 28-Day Seizure Frequency for Seizure Types through 17 week Post-Baseline Phase (Median Percent Change)


Secondary Outcome Measures :
  1. Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change) [ Time Frame: [Time Frame: End of the double-blind 17 week treatment period] ]
    Summary of 28-day Seizure Frequency for Seizure Types for Subjects in the Biomarker-positive Stratum through 17 weeks (Median Percent Change)

  2. 50% Primary Seizure Reduction [ Time Frame: End of the double-blind 17 week treatment period ]
    Percent of subjects experiencing a greater than or equal to 50% reduction in 28-day primary seizure frequency relative to the 12-week baseline


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant
  • Failure to control seizures despite 2 or more anti-seizure medications
  • 12 seizures over a 12-week period of primary seizure types prior to screening
  • On a stable regimen of concomitant AEDs, Ketogenic diets, and modified Atkins diet should be unchanged for 3 months prior to screening)

Exclusion Criteria:

  • Previous exposure to ganaxolone
  • > 8 consecutive weeks of seizure freedom during the 12 weeks prior to screening
  • Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted
  • Use of tetrahydrocannabinol (THC) or non-approved cannabidiol (CBD) is prohibited during the double-blind phase
  • Exposure to any other investigational drug within 30 days or fewer than 5 half-lives prior to screening
Contacts and Locations
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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): NCT03865732


Locations
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United States, Arkansas
Marinus Research Site
Little Rock, Arkansas, United States, 72202
United States, California
Marinus Research Site
Los Angeles, California, United States, 90095
Marinus Research Site
San Francisco, California, United States, 94158
United States, North Carolina
Marinus Research Site
Durham, North Carolina, United States, 27710
United States, Pennsylvania
Marinus Research Site
York, Pennsylvania, United States, 17403
United States, Utah
Marinus Research Site
Salt Lake City, Utah, United States, 84113
Hungary
Marinus Research Site
Budapest, Hungary, 1083
Italy
Marinus Research Site
Firenze, Italy, 50139
Marinus Research Site
Rome, Italy, 00165
Netherlands
Marinus Research Site
Heeze, Netherlands, 5591
Marinus Research Site
Zwolle, Netherlands, 8025
Poland
Marinus Research Site
Krakow, Poland, 30-363
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
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Study Director: Maciej Gasior, M.D., Ph.D Marinus Pharmaceuticals, Inc.
Study Director: Paula Bokesk, M.D., FAAP Marinus Pharmaceuticals, Inc.
  Study Documents (Full-Text)

Documents provided by Marinus Pharmaceuticals:
Study Protocol  [PDF] January 20, 2021
Statistical Analysis Plan  [PDF] March 1, 2021

More Information
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03865732    
Other Study ID Numbers: 1042-PCDH19-3002
2018-004496-12 ( EudraCT Number )
First Posted: March 7, 2019    Key Record Dates
Results First Posted: September 8, 2022
Last Update Posted: September 8, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Marinus Pharmaceuticals:
refractory seizures
epilepsy in children
seizure disorder
Additional relevant MeSH terms:
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Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Pregnanolone
Ganaxolone
Neurosteroids
 Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
GABA Modulators
GABA Agents
Anesthetics
Central Nervous System Depressants