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

June 20, 2023 updated by: Marinus Pharmaceuticals

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.

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

29

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Hungary
      • Budapest, Hungary, 1083
        • Marinus Research Site
  • Italy
      • Firenze, Italy, 50139
        • Marinus Research Site
      • Rome, Italy, 00165
        • Marinus Research Site
  • Netherlands
      • Heeze, Netherlands, 5591
        • Marinus Research Site
      • Zwolle, Netherlands, 8025
        • Marinus Research Site
  • Poland
      • Krakow, Poland, 30-363
        • Marinus Research Site
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Marinus Research Site
    • California
      • Los Angeles, California, United States, 90095
        • Marinus Research Site
      • San Francisco, California, United States, 94158
        • Marinus Research Site
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Marinus Research Site
    • Pennsylvania
      • York, Pennsylvania, United States, 17403
        • Marinus Research Site
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Marinus Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 17 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
placebo suspension 3x's /day for 17 weeks
Drug: Placebo
inactive
Other Names:
  • Placebo (for ganaxolone)
Experimental: Ganaxolone
ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks
Drug: Ganaxolone
active drug

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
End of the double-blind 17 week treatment period

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
[Time Frame: End of the double-blind 17 week treatment period]
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
End of the double-blind 17 week treatment period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Marinus Pharmaceuticals

Investigators

  • Study Director: Maciej Gasior, M.D., Ph.D, Marinus Pharmaceuticals, Inc.
  • Study Director: Paula Bokesk, M.D., FAAP, Marinus Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 17, 2019

Primary Completion (Actual)

January 19, 2021

Study Completion (Actual)

June 20, 2022

Study Registration Dates

First Submitted

March 5, 2019

First Submitted That Met QC Criteria

March 5, 2019

First Posted (Actual)

March 7, 2019

Study Record Updates

Last Update Posted (Actual)

June 26, 2023

Last Update Submitted That Met QC Criteria

June 20, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1042-PCDH19-3002
  • 2018-004496-12 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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