search
Back to results

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

Primary Purpose

Status Epilepticus

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
IV Ganaxolone
IV Placebo
Sponsored by
Marinus Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Status Epilepticus focused on measuring Established status epilepticus, Ganaxolone, Antiepileptic drug, Adjuvant therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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 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. An individual who, in the opinion of the investigator has a 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.

Sites / Locations

  • Marinus Research Site
  • Marinus Research SiteRecruiting
  • Marinus Research Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

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

Double-blind phase: IV Ganaxolone + SOC

Double-blind phase: IV Placebo + SOC

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
February 24, 2023
Last Updated
May 19, 2023
Sponsor
Marinus Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT05757544
Brief Title
Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE)
Official Title
A Study to Optimize the Dosing Regimen and Assess Safety and Efficacy of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 1, 2023 (Actual)
Primary Completion Date
February 2026 (Anticipated)
Study Completion Date
February 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Marinus Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Status Epilepticus
Keywords
Established status epilepticus, Ganaxolone, Antiepileptic drug, Adjuvant therapy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigator
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.
Allocation
Randomized
Enrollment
120 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dose optimization phase (open label): IV Ganaxolone bolus (variable) followed by infusion (variable)
Arm Type
Experimental
Arm Title
Double-blind phase: IV Ganaxolone + SOC
Arm Type
Experimental
Arm Title
Double-blind phase: IV Placebo + SOC
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
IV Ganaxolone
Intervention Description
IV Ganaxolone will be administered.
Intervention Type
Drug
Intervention Name(s)
IV Placebo
Intervention Description
IV Placebo will be administered
Primary Outcome Measure Information:
Title
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 Outcome Measure Information:
Title
Percentage of participants with no escalation of care for Status epilepticus (SE) during investigational product (IP) infusion
Time Frame
Up to Day 7
Title
Time to clinical seizure and electrographic SE cessation
Time Frame
Up to Day 7
Title
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
Time Frame
Up to 24 hours
Title
Percentage of participants with no escalation to IV anesthesia for treatment of seizures during IP infusion
Time Frame
Up to Day 7
Title
Percentage of participants with no escalation to IV anesthesia for treatment of seizures within 24 hours of the initial IP bolus
Time Frame
Up to 24 hours
Title
Time to clinical or electrographic seizure recurrence within 24 hours from the initiation of the first IP bolus
Time Frame
Up to 24 hours

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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. An individual who, in the opinion of the investigator has a 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Becky Williams
Phone
484-801-4669
Email
reset@marinuspharma.com
Facility Information:
Facility Name
Marinus Research Site
City
Albuquerque
State/Province
New Mexico
ZIP/Postal Code
87106
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Marinus Research Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45267
Country
United States
Individual Site Status
Recruiting
Facility Name
Marinus Research Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Individual Site Status
Not yet recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs