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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 : Recruiting
First Posted : March 7, 2019
Last Update Posted : July 26, 2019
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 3

Detailed Description:
The Violet Study is a global, double-blind, placebo-controlled, Phase 3 clinical trial that will enroll approximately 50-70 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)
Estimated Enrollment : 50 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
Estimated Primary Completion Date : April 30, 2021
Estimated Study Completion Date : March 31, 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
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
 Drug: Ganaxolone
active drug
Placebo Comparator: Placebo
placebo suspension 3x's /day for 17 weeks
 Drug: Placebo
inactive
Other Name: Placebo (for ganaxolone)


Outcome Measures
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Primary Outcome Measures :
  1. Percent change in 28-day seizure frequency [ Time Frame: End of the double-blind 17 week treatment period ]
    Percent change in 28-day seizure frequency during the double-blind treatment period relative to the 8-week prospective baseline period


Secondary Outcome Measures :
  1. Arithmetic change in percent of seizure-free days of primary seizure types [ Time Frame: End of the double-blind 17 week treatment period ]
    Arithmetic change in percent of seizure-free days of primary seizure types during the double-blind treatment period of ganaxolone compared to placebo

  2. Behavior Rating Inventory of Executive Function [ Time Frame: End of the double-blind 17 week treatment period ]
    Behavior rating inventory of executive function during the double-blind treatment period of ganaxolone compared to placebo

  3. Aberrant Behavior Checklist - Community [ Time Frame: End of the double-blind 17 week treatment period ]
    Aberrant behavior checklist - community during the double-blind treatment period of ganaxolone compared to placebo

  4. Children's Sleep Habit Questionnaire [ Time Frame: End of the double-blind 17 week treatment period ]
    Children's sleep habit questionnaire during the double-blind treatment period of ganaxolone compared to placebo


Eligibility Criteria
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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
  • 4 seizures per 28 days over an 8-week period of primary seizure types
  • On a stable regimen of anti-seizure medications (Vagus nerve stimulator, ketogenic diet, and modified Atkins diet do not count towards this limit)

Exclusion Criteria:

  • Previous exposure to ganaxolone
  • ≥ 8 continuous months of seizure freedom over the previous 12 consecutive months
  • Concurrent use of strong inducers or inhibitors of CYP3A4/5/7 is not permitted
  • Use of tetrahydrocannabinol (THC) or non-regulated 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


Contacts
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Contact: Alex Aimetti, Ph.D 484-801-4681 ext 681 aaimetti@marinuspharma.com
Contact: Jason Balderson 484-801-5638 ext 698 jbalderson@marinuspharma.com

Locations
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United States, Arkansas
Marinus Research Site Recruiting
Little Rock, Arkansas, United States, 72202
United States, California
Marinus Research Site Recruiting
San Francisco, California, United States, 94158
Sponsors and Collaborators
Marinus Pharmaceuticals
Investigators
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Study Director: Maciej Gasior, M.D., Ph.D Marinus Pharmaceuticals, Inc.
More Information
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03865732     History of Changes
Other Study ID Numbers: 1042-PCDH19-3002
2018-004496-12 ( EudraCT Number )
First Posted: March 7, 2019    Key Record Dates
Last Update Posted: July 26, 2019
Last Verified: July 2019
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
Anesthetics
Central Nervous System Depressants
Physiological Effects of Drugs