Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

Phase 2

Completed

• Conditions: Epilepsy; Status Epilepticus; Convulsive Status EPILEPTICUS; Non Convulsive Status Epilepticus
• Interventions: Drug: IV Placebo, non-active; Drug: IV Ganaxolone active

• Registration Number: NCT03350035
• Lead Sponsor: Marinus Pharmaceuticals

Brief Summary

This study will evaluate the effectiveness and safety of an investigational drug, IV ganaxolone, as adjunctive therapy to standard of care to treat subjects with status epilepticus.

Detailed Description

This is a double-blind, randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of adjunctive IV ganaxolone in subjects with SE.

Study drug 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 study drug as adjunctive therapy by continuous IV. Subject's will be followed up for 3 weeks post-treatment.

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

Study Timeline

• Study First Submitted: 2017-11-17
• Study First Submitted QC: 2017-11-17
• Study First Posted: 2017-11-22
• Study Start: 2018-02-19
• Primary Completion: 2019-09-18
• Study Completion: 2019-09-18
• Disposition First Submitted: 2020-09-25
• Results First Submitted: 2022-09-28
• Results First Submitted QC: 2022-11-18
• Disposition First Submitted QC: 2022-11-18
• Results First Posted: 2022-12-15
• Disposition First Posted: 2022-12-15
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-08
• Last Update Posted: 2023-03-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 17

Inclusion Criteria

• Subjects 12 years of age and older
• Clinical and/or electrographic seizures

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Exclusion Criteria

• Life expectancy of less than 24 hours
• Anoxic brain injury as primary cause of SE
• Recent (<24 hour) traumatic brain injury as the primary cause of SE
• Administered anesthesia for the treatment of SE

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
IV Placebo, non-active	IV Placebo, non-active	Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
IV Ganaxolone active	IV Ganaxolone active	Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.

Primary Outcome Measures

Name	Time	Method
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment	24 hours post study drug initiation	Number of participants who did not require an intravenous (IV) Anesthetic Drug (a third-line Treatment) for Status Epilepticus (SE) within the first 24 hours after Study Drug Initiation.

Secondary Outcome Measures

Name	Time	Method
Time to Cessation of SE	Time to SE Cessation, assessed up to 24 hours	Summary of Time to SE Cessation
Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE	Drug initiation through follow-up period, up to approximately 4 weeks	Number of participants who Required No Escalation of Treatment for Ongoing or Recurrent SE
Number of Participants With No SE Recurrence Per Principal Investigator	Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.	Number of participants with No SE Recurrence per Principal Investigator within 24hrs of starting treatment, during treatment period (excluding taper), during taper, during 24-hr follow-up period, and during follow-up period.
Seizure Burden	Baseline (Pre-dose) to <-24hrs (Post Dose)	Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point

Trial Locations

Locations (8)

Duke Medical Center; 🇺🇸 Durham, North Carolina, United States

Brigham and Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States

Henry Ford Hospital; 🇺🇸 Detroit, Michigan, United States

Grady Hospital; 🇺🇸 Atlanta, Georgia, United States

Allegheny General Hospital; 🇺🇸 Pittsburgh, Pennsylvania, United States

Oschner Clinic Foundation; 🇺🇸 New Orleans, Louisiana, United States

Nemours/AI duPont Hospital for Children; 🇺🇸 Wilmington, Delaware, United States