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

Marinus Pharmaceuticals0 sitesApril 1, 2023

ConditionsStatus Epilepticus

InterventionsIV Ganaxolone IV Placebo

DrugsIV Ganaxolone IV Placebo

Overview

Phase: Phase 2
Intervention: IV Ganaxolone
Conditions: Status Epilepticus
Sponsor: Marinus Pharmaceuticals
Primary Endpoint: Absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus or recurrence of convulsions at 1 hour after the first IP bolus administration without the use of any additional medications with anti-seizure properties
Status: Withdrawn
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

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 participants with 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.

Registry

clinicaltrials.gov

Start Date

April 1, 2023

End Date

February 2026

Last Updated

last year

Study Type

Interventional

Study Design

Sequential

Sex

All

Investigators

Sponsor

Marinus Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

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 the first 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 after the 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.

Arms & Interventions

Dose optimization phase (open label): IV Ganaxolone bolus (variable) followed by infusion (variable)

Intervention: IV Ganaxolone

Double-blind phase: IV Ganaxolone + SOC

Intervention: IV Ganaxolone

Double-blind phase: IV Placebo + SOC

Intervention: IV Placebo

Outcomes

Primary Outcomes

Absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus or recurrence of convulsions at 1 hour after the first IP bolus administration without the use of any additional medications with anti-seizure properties

Time Frame: At 1 hour

Secondary Outcomes

Time to clinical seizure and electrographic SE cessation(Up to Day 7)
Percentage of participants with no escalation of care (including re-administration of IP) at any point within 24 hours from the initial IP bolus administration(Up to 24 hours)
Percentage of participants with no escalation to IV anesthesia for treatment of seizures during IP infusion(Up to Day 7)
Percentage of participants with no escalation to IV anesthesia for treatment of seizures within 24 hours of the initial IP bolus(Up to 24 hours)
Time to clinical or electrographic seizure recurrence within 24 hours from the initiation of the first IP bolus(Up to 24 hours)
Percentage of participants with no escalation of care for Status epilepticus (SE) during investigational product (IP) infusion(Up to Day 7)