NCT03865732

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
29

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2019

Typical duration for phase_2

Geographic Reach
5 countries

12 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 5, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 7, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

May 17, 2019

Completed
1.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 19, 2021

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 20, 2022

Completed
3 months until next milestone

Results Posted

Study results publicly available

September 8, 2022

Completed
Last Updated

June 26, 2023

Status Verified

June 1, 2023

Enrollment Period

1.7 years

First QC Date

March 5, 2019

Results QC Date

May 24, 2022

Last Update Submit

June 20, 2023

Conditions

PCDH19-Related Epilepsy

Keywords

refractory seizuresepilepsy in childrenseizure disorder

Outcome Measures

Primary Outcomes (1)

  • Summary of 28-day Seizure Frequency Through 17 Week Post-Baseline Phase (Median Percent Change)

    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 Outcomes (2)

  • Summary of 28-day Seizure Frequency for Subjects in the Biomarker-positive Stratum (Median Percent Change)

    [Time Frame: End of the double-blind 17 week treatment period]

  • 50% Primary Seizure Reduction

    End of the double-blind 17 week treatment period

Study Arms (2)

Placebo

PLACEBO COMPARATOR

placebo suspension 3x's /day for 17 weeks

Drug: Placebo

Ganaxolone

EXPERIMENTAL

ganaxolone suspension (50 mg/ml) 3x's /day for 17 weeks

Drug: Ganaxolone

Interventions

GanaxoloneDRUG

active drug

Ganaxolone
PlaceboDRUG

inactive

Also known as: Placebo (for ganaxolone)
Placebo

Eligibility Criteria

Age1 Year - 17 Years
Sexfemale(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Molecular confirmation of a pathogenic or likely pathogenic PCDH19 variant
  • Failure to control seizures despite 2 or more anti-seizure medications
  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Marinus Research Site

Little Rock, Arkansas, 72202, United States

Location

Marinus Research Site

Los Angeles, California, 90095, United States

Location

Marinus Research Site

San Francisco, California, 94158, United States

Location

Marinus Research Site

Durham, North Carolina, 27710, United States

Location

Marinus Research Site

York, Pennsylvania, 17403, United States

Location

Marinus Research Site

Salt Lake City, Utah, 84113, United States

Location

Marinus Research Site

Budapest, 1083, Hungary

Location

Marinus Research Site

Florence, 50139, Italy

Location

Marinus Research Site

Rome, 00165, Italy

Location

Marinus Research Site

Heeze, 5591, Netherlands

Location

Marinus Research Site

Zwolle, 8025, Netherlands

Location

Marinus Research Site

Krakow, 30-363, Poland

Location

Related Publications (1)

  • Sullivan J, Gunning B, Zafar M, Guerrini R, Gecz J, Kolc KL, Zhao Y, Gasior M, Aimetti AA, Samanta D. Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy. Epilepsy Res. 2023 Mar;191:107112. doi: 10.1016/j.eplepsyres.2023.107112. Epub 2023 Feb 22.

    PMID: 36870093DERIVED

MeSH Terms

Conditions

SeizuresEpilepsy

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Neurologic ManifestationsNervous System DiseasesSigns and SymptomsPathological Conditions, Signs and SymptomsBrain DiseasesCentral Nervous System Diseases

Results Point of Contact

Title
Marinus Clinical Trials Submission Manager
Organization
Marinus Pharmaceuticals, Inc.
clinicaltrials@marinuspharma.com
484-801-4670

Study Officials

  • Maciej Gasior, M.D., Ph.D

    Marinus Pharmaceuticals, Inc.

    STUDY DIRECTOR

  • Paula Bokesk, M.D., FAAP

    Marinus Pharmaceuticals, Inc.

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: The double-blind phase will randomize subjects to adjunctive ganaxolone or placebo at a 1:1 ratio to standard of care.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 5, 2019

First Posted

March 7, 2019

Study Start

May 17, 2019

Primary Completion

January 19, 2021

Study Completion

June 20, 2022

Last Updated

June 26, 2023

Results First Posted

September 8, 2022

Record last verified: 2023-06

Data Sharing

IPD Sharing
Will not share

Locations

US(6)PL(1)IT(2)NL(2)HU(1)