NCT02358538

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.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Nov 2015

Typical duration for phase_2

Geographic Reach
2 countries

10 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

January 30, 2015

Completed
10 days until next milestone

First Posted

Study publicly available on registry

February 9, 2015

Completed
9 months until next milestone

Study Start

First participant enrolled

November 6, 2015

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 16, 2018

Completed
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 4, 2019

Completed
3.7 years until next milestone

Results Posted

Study results publicly available

September 7, 2022

Completed
Last Updated

March 21, 2023

Status Verified

March 1, 2023

Enrollment Period

2.2 years

First QC Date

January 30, 2015

Results QC Date

May 11, 2021

Last Update Submit

March 17, 2023

Conditions

Epilepsy

Keywords

PCDH19ganaxoloneneurosteroidCDKL5LGSCSWS

Outcome Measures

Primary Outcomes (2)

  • Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation)

    Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change \& Standard Deviation)

    Baseline through 52 week open label period

  • Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change)

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

    Baseline through 52-week open- label period

Secondary Outcomes (4)

  • Summary of CGII-C

    End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78

  • Summary of CGII-P

    Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ]

  • Number of Participants With Responder Rate of Seizure Frequency

    Month 3 and Week 26

  • Mean Percentage Change of Individual Seizure-free Days

    Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE Period

Study Arms (1)

Ganaxolone

EXPERIMENTAL

Maximum of 1800 mg/day or 63 mg/kg/day

Drug: Ganaxolone

Interventions

GanaxoloneDRUG

oral suspension or capsules

Also known as: CCD 1042
Ganaxolone

Eligibility Criteria

Age2 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Have parent or legal guardian available and willing to give written informed consent.
  • Male and female outpatients between 2 and 18 years of age years of age at time of consent.
  • Have any of the following epilepsy syndromes: PCDH19; CDKL5; Dravet Syndrome; Lennox Gastaut Syndrome (LGS); Continuous Spikes and Waves during Sleep (CSWS)
  • Have uncontrolled cluster seizures and/or non-clustered seizures.
  • Subjects should be on a stable regimen of anti-epileptic medication, and generally in good health.
  • Parent or guardian is able and willing to maintain an accurate and complete daily written seizure calendar.
  • Able and willing to take study medication with food, two or three times daily.

You may not qualify if:

  • Have had previous exposure to ganaxolone.
  • Known sensitivity or allergy to any component in the study drug, progesterone, or other related steroid compounds.
  • 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.
  • Concurrent use of vigabatrin, tiagabine, or ezogabine is not permitted.
  • 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.
  • 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.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Sutter Institute for Medical Research

Sacramento, California, 95816, United States

Location

University of California San Francisco

San Francisco, California, 94143, United States

Location

Center for Rare Neurological Diseases

Norcross, Georgia, 30093, United States

Location

JWM Neurology

Indianapolis, Indiana, 46239, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Northeast Regional Epilepsy Group

Hackensack, New Jersey, 07601, United States

Location

Institute of Neurology and Neurosurgery at St. Barnabas

Livingston, New Jersey, 07039, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

Bambino Gesu Children's Hospital, IRCCS

Rome, 00165, Italy

Location

MeSH Terms

Conditions

Epilepsy

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Brain DiseasesCentral Nervous System DiseasesNervous System Diseases

Limitations and Caveats

CSWS cohort was not included in efficacy summaries as only 2 subjects were enrolled in cohort; however, cohort was included in the subject data listings. Overall number of participants affected is associated with all TEAEs and not \>-5%. Individual AEs are associated with \>-5%. One patient in the CDKL5 cohort had duplication of data on 6 days, which appeared to PI and sponsor to be erroneous. This datapoint is represented accordingly in outcome measure

Results Point of Contact

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

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 30, 2015

First Posted

February 9, 2015

Study Start

November 6, 2015

Primary Completion

January 16, 2018

Study Completion

January 4, 2019

Last Updated

March 21, 2023

Results First Posted

September 7, 2022

Record last verified: 2023-03

Data Sharing

IPD Sharing
Will not share

Locations

IT(1)US(9)