Purpose

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

Condition

Eligibility

Eligible Ages
Over 12 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  1. 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. 2. Male or females 12 years of age and older at the time of the first dose of IP 3. 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 4. 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, - IV Levetiracetam, - IV Lacosamide, - IV Brivaracetam, or - IV Phenobarbital 5. Body mass index (BMI) < 40 or, if BMI is not able to be calculated at screening, participant is assessed by investigator as not morbidly obese

Exclusion Criteria

  1. Life expectancy of less than 24 hours 2. 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) 3. 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. 4. Clinical condition or advance directive that would NOT permit use of IV anesthesia 5. Participants known or suspected to be pregnant 6. Participants with known allergy or sensitivity to progesterone or allopregnanolone medications/supplements 7. Receiving a concomitant IV product containing Captisol® 8. Known or suspected hepatic insufficiency or hepatic failure leading to impaired synthetic liver function. 9. 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 10. 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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Placebo Comparator
IV Placebo
Placebo bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
  • Drug: Placebo
    Placebo will be administered.
Experimental
IV ganaxolone active
Ganaxolone bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
  • Drug: Ganaxolone
    Ganaxolone will be administered.

Recruiting Locations

Marinus Research Site
Birmingham, Alabama 35233

Marinus Research Site
Downey, California 90242

Marinus Research Site
Orange, California 92868

Marinus Research Site
Sacramento, California 95816

Marinus Research Site
Sacramento, California 95817

Marinus Research Site
Aurora, Colorado 80045

Marinus Research Site
Orlando, Florida 32803

Marinus Research Site
Chicago, Illinois 60611

Marinus Research Site
Urbana, Illinois 61801

Marinus Research Site
Louisville, Kentucky 40202

Marinus Research Site
New Orleans, Louisiana 70121

Marinus Research Site
Shreveport, Louisiana 71103

Marinus Research Site #1
Baltimore, Maryland 21201

Marinus Research Site
Baltimore, Maryland 21215

Marinus Research Site
Bethesda, Maryland 20817

Marinus Research Site
Boston, Massachusetts 02111

Marinus Research Site
Boston, Massachusetts 02114

Marinus Research Site
Boston, Massachusetts 02115

Marinus Research Site
Saint Louis, Missouri 63110

Marinus Research Site
New Brunswick, New Jersey 08901

Marinus Research Site
Durham, North Carolina 27710

Marinus Research Site
Cincinnati, Ohio 45219

Marinus Research Site
Cleveland, Ohio 44195

Marinus Research Site
Portland, Oregon 97225

Marinus Research Site
Portland, Oregon 97239

Marinus Research Site
Memphis, Tennessee 38103

Marinus Research Site
Fort Worth, Texas 76104

More Details

NCT ID
NCT04391569
Status
Recruiting
Sponsor
Marinus Pharmaceuticals

Study Contact

Marinus
484-801-4670
clinicaltrials@marinuspharma.com

Detailed Description

This is a double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of IV ganaxolone in status epilepticus. Investigational product will be added to standard of care following failure of any two or more antiseizure medications (benzodiazepine and one IV antiepileptic drug (AED) or two IV AEDs. Participants will be screened for inclusion/exclusion criteria prior to receiving investigational product by continuous IV infusion. Participants will be followed for approximately 4 weeks. Participants who are known to be at risk for SE may be consented or assented prior to an SE event.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.