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RAndomized Therapy In Status Epilepticus (RAISE)

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ClinicalTrials.gov Identifier: NCT04391569
Recruitment Status : Recruiting
First Posted : May 18, 2020
Last Update Posted : October 21, 2022
Information provided by (Responsible Party):
Marinus Pharmaceuticals

Brief Summary:
This study will evaluate the effectiveness and safety of an investigational product, IV ganaxolone, to treat subjects with status epilepticus.

Condition or disease Intervention/treatment Phase
Status Epilepticus Convulsive Status EPILEPTICUS Non-Convulsive Status Epilepticus Epilepsy Drug: Ganaxolone Drug: Placebo Phase 3

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 before IV anesthetic during the treatment of SE. Subjects will be screened for inclusion/exclusion criteria prior to receiving investigational product by continuous IV infusion. Subjects will be followed for approximately 4 weeks.

Subjects who are known to be at risk for SE may be consented or assented prior to an SE event.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 124 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus
Actual Study Start Date : October 10, 2020
Estimated Primary Completion Date : October 31, 2023
Estimated Study Completion Date : October 31, 2023

Arm Intervention/treatment
Placebo Comparator: IV Placebo
Placebo bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
Drug: Placebo
Double-blind study that will randomize subjects to ganaxolone or placebo in 1:1 ratio.

Experimental: IV ganaxolone active
Ganaxolone bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
Drug: Ganaxolone
Double-blind study that will randomize subjects to ganaxolone or placebo in 1:1 ratio.

Primary Outcome Measures :
  1. SE Cessation [ Time Frame: 30 minutes ]
    Proportion of participants with status epilepticus cessation within 30 minutes of IP initiation without medications for the acute treatment of status epilepticus. SE cessation is determined by clinical and EEG findings.

  2. Progression to IV anesthesia [ Time Frame: 36 hours ]
    Proportion of participants with no progression to IV anesthesia for 36 hours following IP initiation

Secondary Outcome Measures :
  1. Progression to IV anesthesia [ Time Frame: 72 hours ]
    No progression to IV anesthesia for 72 hours following IP initiation

  2. SE Cessation [ Time Frame: 48 hours ]
    Time to SE cessation following IP initiation

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Participant, participant's parent, guardian, or LAR must provide signature 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
  2. Male or females 12 years of age and older at the time of the first dose of IP
  3. Convulsive or nonconvulsive SE with seizure burden warranting imminent progression to IV anesthesia for seizure control. Ictal patterns and burden defined below.

    • Convulsive SE: Clinical and EEG seizure activity or
    • Nonconvulsive SE: Must have an ictal EEG pattern consistent with modified Salzburg criteria (details provided in Appendix 2)
    • Ictal burden of approximately 6 minutes or more within 30 minutes immediately prior to IP initiation is targeted
  4. Participants must have received a benzodiazepine and two or more of the following second-line IV AEDs for treatment of the current episode of SE, administered at an adequate dose and duration to demonstrate efficacy, in the opinion of the investigator (guidelines for adequate doses are provided in Appendix 1)

    • Fosphenytoin/phenytoin,
    • Valproic acid,
    • Levetiracetam,
    • Lacosamide
    • Brivaracetam, or
    • Phenobarbital
  5. 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 a rapidly reversable metabolic condition as the primary cause of SE (e.g., hypoglycemia < 50 mg/dL or hyperglycemia > 400 mg/dL)
  3. Participants on IV anesthesia (e.g., midazolam, propofol, thiopental, or pentobarbital) with the primary intent specifically to treat seizures or achieve burst suppression
  4. Seizure burden or clinical state would NOT warrant IV anesthesia for seizure control over the next 24 hours
  5. Participants with an advanced directive that would not allow the institution to administer their SOC for the treatment of SE (e.g., directive Do Not Intubate)
  6. Participants known or suspected to be pregnant
  7. Participants with known allergy or sensitivity to progesterone or allopregnanolone medications/supplements
  8. Receiving a concomitant IV product containing Captisol® (US-marketed products listed in Appendix 3)
  9. Known or suspected hepatic insufficiency or hepatic failure
  10. Known or suspected stage 3B (moderate to severe; eGFR 44-30 mL/min/1.73m2), stage 4 (severe; eGFR 29-15 mL/min/1.73m2), or stage 5 (kidney failure; eGFR < 15 mL/min/1.73m2 or dialysis) kidney disease
  11. 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.
  12. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04391569

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Contact: Maciej Gasior, MD, PhD 484-639-4875 mgasior@marinuspharma.com
Contact: Heather Van Heusen 484-802-2470 hvanheusen@marinuspharma.com

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Sponsors and Collaborators
Marinus Pharmaceuticals
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Study Director: Maciej Gasior, MD, PhD Marinus Pharmaceuticals, Inc.
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Responsible Party: Marinus Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04391569    
Other Study ID Numbers: 1042-SE-3003
First Posted: May 18, 2020    Key Record Dates
Last Update Posted: October 21, 2022
Last Verified: October 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Marinus Pharmaceuticals:
Additional relevant MeSH terms:
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Status Epilepticus
Nervous System Diseases
Neurologic Manifestations
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
GABA Modulators
GABA Agents