Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

Sponsor

Marinus Pharmaceuticals (Industry)

Overall Status

Completed

CT.gov ID

NCT03350035

Collaborator

(none)

Enrollment

Locations

Arms

18.9

Actual Duration (Months)

2.1

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Status Epilepticus Convulsive Status EPILEPTICUS Non Convulsive Status Epilepticus Epilepsy	Drug: IV Ganaxolone active Drug: IV Placebo, non-active	Phase 2

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 Design

Study Type:

Interventional

Actual Enrollment :

17 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

Double-blind study that will randomize subjects to ganaxolone or placebo in a 1:1 ratio as adjunctive therapy to their standard of care.Double-blind study that will randomize subjects to ganaxolone or placebo in a 1:1 ratio as adjunctive therapy to their standard of care.

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Feb 19, 2018

Actual Primary Completion Date :

Sep 18, 2019

Actual Study Completion Date :

Sep 18, 2019

Arms and Interventions

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

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

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

Outcome Measures

Primary Outcome Measures

Primary Outcome [24 hours post study drug initiation]
The number of subjects who didn't require IV anesthetic for SE treatment within the first 24 hours after study drug initiation

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Nemours/AI duPont Hospital for Children	Wilmington	Delaware	United States	19803
2	Nicklaus Children's Hospital	Miami	Florida	United States	33155
3	Grady Hospital	Atlanta	Georgia	United States	30303
4	Oschner Clinic Foundation	New Orleans	Louisiana	United States	70121
5	Brigham and Women's Hospital	Boston	Massachusetts	United States	02115
6	Henry Ford Hospital	Detroit	Michigan	United States	48202
7	Duke Medical Center	Durham	North Carolina	United States	27710
8	Allegheny General Hospital	Pittsburgh	Pennsylvania	United States	15212

Sponsors and Collaborators

Marinus Pharmaceuticals

Investigators

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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Marinus Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT03350035

Other Study ID Numbers:

1042-SE-2001

First Posted:

Nov 22, 2017

Last Update Posted:

Oct 22, 2020

Last Verified:

Oct 1, 2020

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Status Epilepticus

Ganaxolone

Study Results

No Results Posted as of Oct 22, 2020