Randomized Therapy In Status Epilepticus

NCT04391569 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: 1042-SE-3003
• Study Type: interventional
• Target: 100
• Locations: 3 countries
• Sites: 74

Brief Summary

This study evaluated the effectiveness and safety of an investigational product (IP), intravenous (IV) ganaxolone, to treat participants with status epilepticus (SE).

Conditions

Status Epilepticus

Keywords

Seizure; Status Epilepticus; Convulsive Status Epilepticus; Non-Convulsive Status Epilepticus; Epilepsy

Outcome Measures

Primary Outcomes (3)

• Percentage of Participants With SE Cessation Within 30 Minutes of Investigational Product (IP) Initiation Without Medications for the Acute Treatment of SE

  SE cessation was determined by the investigator based on clinical and electroencephalography (EEG). Medications for the acute treatment of SE were defined as antiepileptic drugs (AEDs) administered to abort ongoing SE or prevent imminent recurrence of SE based on clinical or EEG evidence.

  Up to 30 minutes

• Percentage of Participants With no Progression to Intravenous (IV) Anesthesia for 36 Hours Following Investigational Product (IP) Initiation

  Percentage of participants with no progression to IV anesthesia for 36 hours following IP initiation SE cessation was based on investigator report with confirmation by assessment of concomitant medication data.

  Up to 36 hours after IP initiation

• Number of Participants With Treatment Emergent Adverse Events

  An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant who has been administered a pharmaceutical product; it does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse event (TEAE) is defined as an AE that occurred or worsened at the time of or following IP initiation.

  Up to 4 weeks after IP initiation

Secondary Outcomes (7)

• Percentage of Participants With no Progression to IV Anesthesia for 72 Hours Following IP Initiation

  Up to 72 hours after IP initiation

• Time to SE Cessation Following IP Initiation

  Up to 72 hours after IP initiation

• Percentage of Participants With Any Escalation of Treatment in the First 24 Hours Following IP Initiation

  Up to 24 hours after IP initiation

• Time to Treatment Escalation in the First 24 Hours Following IP Initiation

  Up to 24 hours after IP initiation

• Time to Initiation of Anesthesia for SE Treatment Through the Final Study Follow-up Visit/Contact

  Up to 4 Weeks following IP initiation

Study Arms (2)

IV Placebo

PLACEBO COMPARATOR

Placebo bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper

Drug: Placebo

IV ganaxolone active

EXPERIMENTAL

Ganaxolone bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper

Drug: Ganaxolone

Interventions

Ganaxolone DRUG

Ganaxolone will be administered.

IV ganaxolone active

Placebo DRUG

Placebo will be administered.

IV Placebo

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant, participant's parent, guardian, or legal authorized representative (LAR) must provide signed of informed consent/assent, and once capable (per institution guidelines), there must be documentation of consent/assent by the participant demonstrating they are willing and aware of the investigational nature of the study and related procedures. Where allowed by law, where the participant lacks the capacity to make informed decisions regarding his/her medical treatment options, the treating clinician may follow their deferred consenting practices. The clinician will make the final decision based on the best interests of the particiapant.
• Male or females 12 years of age and older at the time of the first dose of IP
• SE meeting the following criteria:
• a. A diagnosis of SE with or without prominent motor features based on clinical and EEG findings:
• i. Diagnosis is established by:
• For SE with prominent motor features: Clinical and EEG seizure activity indicative of convulsive, myoclonic or focal motor SE.
• For SE without prominent motor features (nonconvulsive SE): Appropriate clinical features and an EEG indicative of non-convulsive status epilepticus (NCSE)
• ii. For any type of SE:
• At least 6 minutes of cumulative seizure activity over a 30-minute period within the hour before IP initiation, AND
• Seizure activity during the 30 minutes immediately prior to IP initiation
• b. The treating clinician(s) anticipate that IV anesthesia is likely to be the next treatment for SE that persists following initiation of IP
• Participants must have received any two or more of the following agents for treatment of the current episode of SE administered at an adequate dose and for a sufficient duration, in the judgment of the investigator, to demonstrate efficacy
• Benzodiazepines,
• IV Fosphenytoin/phenytoin,
• IV Valproic acid,

You may not qualify if:

• Life expectancy of less than 24 hours
• Anoxic brain injury or an uncorrected rapidly reversable metabolic condition as the primary cause of SE (e.g., hypoglycemia \< 50 milligram per deciliter \[mg/dL\] or hyperglycemia \> 400 mg/dL)
• Participants who have received high-dose IV anesthetics (e.g., midazolam, propofol, thiopental, or pentobarbital) during the current episode of SE for more than 18 hours, or who continue to have clinical or electrographic evidence of persistent seizures while receiving high-dose IV anesthetics.
• Clinical condition or advance directive that would NOT permit use of IV anesthesia
• Participants known or suspected to be pregnant
• Participants with known allergy or sensitivity to progesterone or allopregnanolone medications/supplements
• Receiving a concomitant IV product containing Captisol®
• Known or suspected hepatic insufficiency or hepatic failure leading to impaired synthetic liver function.
• Known or suspected stage 3B (moderate to severe; estimated glomerular filtration rate \[eGFR\] 44-30 milliliter/minutes/1.73-meter square \[mL/min/1.73m\^2\]), stage 4 (severe; eGFR 29-15 mL/min/1.73m\^2), or stage 5 (kidney failure; eGFR \< 15 mL/min/1.73m\^2 or dialysis) kidney disease
• 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.

Sponsors & Collaborators

• Marinus Pharmaceuticals: lead

Study Sites (74)

Marinus Research Site

Birmingham, Alabama, 35233, United States

Location

Marinus Research Site

Phoenix, Arizona, 85013, United States

Location

Marinus Research Site

Little Rock, Arkansas, 72205, United States

Location

Marinus Research Site

Downey, California, 90242, United States

Location

Marinus Research Site

La Jolla, California, 92037, United States

Location

Marinus Research Site

Orange, California, 92868, United States

Location

Marinus Research Site

Sacramento, California, 95817, United States

Location

Marinus Research Site

Aurora, Colorado, 80045, United States

Location

Marinus Research Site

Denver, Colorado, 80204, United States

Location

Marinus Research Site

New Haven, Connecticut, 06510, United States

Location

Marinus Research Site

Gainesville, Florida, 32608, United States

Location

Marinus Research Site #1

Miami, Florida, 33136, United States

Location

Marinus Research Site

Miami, Florida, 33136, United States

Location

Marinus Research Site

Miami, Florida, 33155, United States

Location

Marinus Research Site

Port Saint Lucie, Florida, 34987, United States

Location

Marinus Research Site

Tampa, Florida, 33606, United States

Location

Marinus Research Site

Chicago, Illinois, 60611, United States

Location

Marinus Research Site

Chicago, Illinois, 60612, United States

Location

Marinus Research Site

Urbana, Illinois, 61801, United States

Location

Marinus Research Site

Louisville, Kentucky, 40202, United States

Location

Marinus Research Site

New Orleans, Louisiana, 70121, United States

Location

Marinus Research Site

Shreveport, Louisiana, 71103, United States

Location

Marinus Research Site #1

Baltimore, Maryland, 21201, United States

Location

Marinus Research Site

Baltimore, Maryland, 21201, United States

Location

Marinus Research Site

Baltimore, Maryland, 21215, United States

Location

Marinus Research Site

Boston, Massachusetts, 02111, United States

Location

Marinus Research Site

Boston, Massachusetts, 02114, United States

Location

Marinus Research Site

Boston, Massachusetts, 02115, United States

Location

Marinus Research Site

Boston, Massachusetts, 02118, United States

Location

Marinus Research Site

Boston, Massachusetts, 02215, United States

Location

Marinus Research Site

Worcester, Massachusetts, 01655, United States

Location

Marinus Research Site

Ann Arbor, Michigan, 48109, United States

Location

Marinus Research Site

Grand Rapids, Michigan, 49503, United States

Location

Marinus Research Site

Columbia, Missouri, 65212, United States

Location

Marinus Research Site

St Louis, Missouri, 63110, United States

Location

Marinus Research Site

New Brunswick, New Jersey, 08901, United States

Location

Marinus Research Site

Albuquerque, New Mexico, 87106, United States

Location

Marinus Research Site

Albany, New York, 12208, United States

Location

Marinus Research Site

Brooklyn, New York, 11203, United States

Location

Marinus Research Site

New York, New York, 10029, United States

Location

Marinus Research Site

New York, New York, 10032, United States

Location

Marinus Research Site

Rochester, New York, 14642, United States

Location

Marinus Research Site

Chapel Hill, North Carolina, 27514, United States

Location

Marinus Research Site

Charlotte, North Carolina, 28203, United States

Location

Marinus Research Site

Durham, North Carolina, 27710, United States

Location

Marinus Research Site

Cincinnati, Ohio, 45219, United States

Location

Marinus Research Site

Cleveland, Ohio, 44195, United States

Location

Marinus Research Site

Columbus, Ohio, 43210, United States

Location

Marinus Research Site

Portland, Oregon, 97225, United States

Location

Marinus Research Site

Portland, Oregon, 97239, United States

Location

Marinus Research Site #1

Philadelphia, Pennsylvania, 19104, United States

Location

Marinus Research Site #2

Philadelphia, Pennsylvania, 19104, United States

Location

Marinus Research Site

Philadelphia, Pennsylvania, 19104, United States

Location

Marinus Research Site

Philadelphia, Pennsylvania, 19107, United States

Location

Marinus Research Site

Philadelphia, Pennsylvania, 19140, United States

Location

Marinus Research Site

Pittsburgh, Pennsylvania, 15212, United States

Location

Marinus Research Site

Knoxville, Tennessee, 37920, United States

Location

Marinus Research Site

Memphis, Tennessee, 38103, United States

Location

Marinus Research Site

Dallas, Texas, 75235, United States

Location

Marinus Research Site

Fort Worth, Texas, 76104, United States

Location

Marinus Research Site

San Antonio, Texas, 78229, United States

Location

Marinus Research Site

Murray, Utah, 84107, United States

Location

Marinus Research Site

Richmond, Virginia, 23298, United States

Location

Marinus Research Site

Madison, Wisconsin, 53792, United States

Location

Marinus Research Site

Randwick, New South Wales, 2031, Australia

Location

Marinus Research Site

South Brisbane, Queensland, 4101, Australia

Location

Marinus Research Site

Box Hill, Victoria, 3128, Australia

Location

Marinus Research Site

Melbourne, Victoria, 3004, Australia

Location

Marinus Research Site

Melbourne, Victoria, 3050, Australia

Location

Marinus Research Site

Calgary, Alberta, T2N 4Z6, Canada

Location

Marinus Research Site

Calgary, Alberta, T3B 6A8, Canada

Location

Marinus Research Site

Kingston, Ontario, K7L 2V7, Canada

Location

Marinus Research Site

Québec, Quebec, G1V 4G2, Canada

Location

Marinus Research Site

Saskatoon, Saskatchewan, S7N 0W8, Canada

Location

MeSH Terms

Conditions

Status Epilepticus; Seizures; Epilepsy

Interventions

ganaxolone

Condition Hierarchy (Ancestors)

Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Brain Diseases; Central Nervous System Diseases

Results Point of Contact

• Title: Marinus Clinical Trials Submission Manager
• Organization: Marinus Pharmaceuticals, Inc.

clinicaltrials@marinuspharma.com

484-801-4670

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 30, 2020
• First Posted: May 18, 2020
• Study Start: December 9, 2020
• Primary Completion: March 30, 2024
• Study Completion: April 28, 2024
• Last Updated: May 29, 2025
• Results First Posted: May 29, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CA (5); AU (5); US (64)