Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE)

Sponsor

Marinus Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05757544

Collaborator

(none)

120

Enrollment

Locations

Arms

Anticipated Duration (Months)

Patients Per Site

39.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study is designed to optimize the dosing regimen and evaluate the preliminary safety and efficacy of Intravenous (IV) ganaxolone as adjuvant therapy for ESE receiving initial IV antiepileptic drug (AED) according to currently practiced standard of care (SOC). The study will be composed of 2 phases: an initial, open-label, dose optimization phase followed by IV ganaxolone versus placebo double-blind phase.

Condition or Disease	Intervention/Treatment	Phase
Status Epilepticus	Drug: IV Ganaxolone Drug: IV Placebo	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

120 participants

Allocation:

Randomized

Intervention Model:

Sequential Assignment

Masking:

Triple (Participant, Care Provider, Investigator)

Masking Description:

All participating staff involved in the evaluation and execution of the study will remain blinded to the participant's treatment assignment during the double-blind phase.

Primary Purpose:

Treatment

Official Title:

A Study to Optimize the Dosing Regimen and Assess Safety and Efficacy of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus

Anticipated Study Start Date :

Jan 1, 2026

Anticipated Primary Completion Date :

Feb 1, 2026

Anticipated Study Completion Date :

Feb 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Dose optimization phase (Open Label): IV Ganaxolone followed by bolus	Drug: IV Ganaxolone IV Ganaxolone will be administered.
Experimental: Double-blind phase: IV Ganaxolone + SOC	Drug: IV Ganaxolone IV Ganaxolone will be administered.
Placebo Comparator: Double-blind phase: IV Placebo + SOC	Drug: IV Placebo IV Placebo will be administered

Arm

Intervention/Treatment

Experimental: Dose optimization phase (Open Label): IV Ganaxolone followed by bolus

Drug: IV Ganaxolone

IV Ganaxolone will be administered.

Experimental: Double-blind phase: IV Ganaxolone + SOC

Drug: IV Ganaxolone

IV Ganaxolone will be administered.

Placebo Comparator: Double-blind phase: IV Placebo + SOC

Drug: IV Placebo

IV Placebo will be administered

Outcome Measures

Primary Outcome Measures

Number of participants with absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus [Up to Day 7]

Secondary Outcome Measures

Time to clinical seizure and/or electrographic Status epilepticus (SE) cessation [Up to Day 7]
Percentage of participants with no escalation of care (including re-administration of Investigational product [IP]) at any point within 24 hours from the initial IP bolus administration [Up to 24 hours]
Time to escalation of care [Up to Day 7]
Percentage of participants requiring endotracheal intubation [Up to Day 7]
Percentage of participants requiring admission to the intensive care unit (ICU) [Up to Day 7]
Percentage of participants with no escalation to IV anesthesia for treatment of seizures during IP infusion [Up to Day 7]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Males or females at least 18 years of age at the time of the first IP bolus dose.
Has received benzodiazepines before or after arrival to the ED for generalized convulsive seizures lasting more than 5 minutes.
Has received the last dose of benzodiazepine more than 5 minutes prior to IP bolus initiation.
Ongoing or recurrent convulsions, or evidence of electrographic SE on rapid EEG immediately prior to the first IP bolus initiation.
The participant has not yet received a second-line IV AED for the treatment of the current episode of SE or the first IP bolus can be initiated within 15 minutes before or 10 minutes of administration of such IV AED(s).

Exclusion Criteria:

The participant is intubated or the decision to proceed with intubation has been made.
The cause of SE is acute anoxic brain injury, cardiac arrest, acute trauma, hyper- or hypo-glycemia, or eclampsia.
The participant is known or suspected to be pregnant.
The participant is incarcerated at the time of SE occurrence.
Participants who pre-emptively opted out of the study.
A known allergy or sensitivity to progesterone or allopregnanolone medications/ supplements.
Receiving a concomitant IV product containing Captisol®.
Known estimated glomerular filtration rate (eGFR) <30 milliliters per minute (mL/min) and not receiving dialysis (may initiate first IP bolus prior to acute labs).
Individual weighing or suspected to weigh <40 kilograms (kg).
Hypotension requiring 2 or more vasopressors.
An individual who, in the opinion of the investigator has the life expectancy of less than 24 hours.
Use of an investigational product for which less than 30 days or 5 half-lives have elapsed from the final product administration. Participation in a non-interventional clinical study does not exclude eligibility.
Known or suspected history or evidence of a medical condition that, in the investigator's judgment, would expose participant to an undue risk of a significant adverse event or interfere with assessments of safety or efficacy during the study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Marinus Research Site	Albuquerque	New Mexico	United States	87106
2	Marinus Research Site	Cincinnati	Ohio	United States	45267
3	Marinus Research Site	Columbus	Ohio	United States	43210