Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

March 8, 2023 updated by: Marinus Pharmaceuticals

A Double-Blind Randomized, Placebo Controlled Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

This study will evaluate the effectiveness and safety of an investigational drug, IV ganaxolone, as adjunctive therapy to standard of care to treat subjects with status epilepticus.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

This is a double-blind, randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of adjunctive IV ganaxolone in subjects with SE.

Study drug will be added to standard of care before IV anesthetic during the treatment of SE.

Subjects will be screened for inclusion/exclusion criteria prior to receiving study drug as adjunctive therapy by continuous IV. Subject's will be followed up for 3 weeks post-treatment.

Subjects who are known to be at risk for SE may be consented and/or assented prior to an SE event.

Study Type

Interventional

Enrollment (Actual)

17

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

  • United States
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Nemours/AI duPont Hospital for Children
    • Florida
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30303
        • Grady Hospital
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Oschner Clinic Foundation
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Brigham and Women's Hospital
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Hospital
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke Medical Center
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15212
        • Allegheny General Hospital

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

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Subjects 12 years of age and older
  • Clinical and/or electrographic seizures

Exclusion Criteria:

  • Life expectancy of less than 24 hours
  • Anoxic brain injury as primary cause of SE
  • Recent (<24 hour) traumatic brain injury as the primary cause of SE
  • Administered anesthesia for the treatment of SE

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
Experimental: IV Ganaxolone active
Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Drug: IV Ganaxolone active
IV
Placebo Comparator: IV Placebo, non-active
Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Drug: IV Placebo, non-active
IV

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
Time Frame: 24 hours post study drug initiation
Number of participants who did not require an intravenous (IV) Anesthetic Drug (a third-line Treatment) for Status Epilepticus (SE) within the first 24 hours after Study Drug Initiation.
24 hours post study drug initiation

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to Cessation of SE
Time Frame: Time to SE Cessation, assessed up to 24 hours
Summary of Time to SE Cessation
Time to SE Cessation, assessed up to 24 hours
Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE
Time Frame: Drug initiation through follow-up period, up to approximately 4 weeks
Number of participants who Required No Escalation of Treatment for Ongoing or Recurrent SE
Drug initiation through follow-up period, up to approximately 4 weeks
Number of Participants With No SE Recurrence Per Principal Investigator
Time Frame: Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
Number of participants with No SE Recurrence per Principal Investigator within 24hrs of starting treatment, during treatment period (excluding taper), during taper, during 24-hr follow-up period, and during follow-up period.
Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
Seizure Burden
Time Frame: Baseline (Pre-dose) to <-24hrs (Post Dose)
Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point
Baseline (Pre-dose) to <-24hrs (Post Dose)

Collaborators and Investigators

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

Sponsor

Marinus Pharmaceuticals

Investigators

  • Study Director: Maciej Gasior, MD, PhD, Marinus Pharmaceuticals

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

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)

February 19, 2018

Primary Completion (Actual)

September 18, 2019

Study Completion (Actual)

September 18, 2019

Study Registration Dates

First Submitted

November 17, 2017

First Submitted That Met QC Criteria

November 17, 2017

First Posted (Actual)

November 22, 2017

Study Record Updates

Last Update Posted (Estimate)

March 9, 2023

Last Update Submitted That Met QC Criteria

March 8, 2023

Last Verified

March 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1042-SE-2001

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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