Clinical trial NCT03350035: Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
Official title: A Double-Blind Randomized, Placebo Controlled Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Status Epilepticus
Convulsive Status EPILEPTICUS
Non Convulsive Status Epilepticus
Epilepsy
Lead sponsor: Marinus Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2017-11-22
Enrollment: 17
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Double-blind study that will randomize subjects to ganaxolone or placebo in a 1:1 ratio as adjunctive therapy to their standard of care.
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

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

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

Primary outcomes:
measure: Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
timeFrame: 24 hours post study drug initiation
Secondary outcomes:
measure: Time to Cessation of SE
timeFrame: Time to SE Cessation, assessed up to 24 hours
measure: Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE
timeFrame: Drug initiation through follow-up period, up to approximately 4 weeks
measure: Number of Participants With No SE Recurrence Per Principal Investigator
timeFrame: Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
measure: Seizure Burden
timeFrame: Baseline (Pre-dose) to <-24hrs (Post Dose)
Other outcomes:
Locations:
city: Wilmington
country: United States
facility: Nemours/AI duPont Hospital for Children
state: Delaware
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Grady Hospital
state: Georgia
city: New Orleans
country: United States
facility: Oschner Clinic Foundation
state: Louisiana
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Durham
country: United States
facility: Duke Medical Center
state: North Carolina
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
Study officials:
affiliation: Marinus Pharmaceuticals
name: Maciej Gasior, MD, PhD
role: STUDY_DIRECTOR
References:
citation: Vaitkevicius H, Ramsay RE, Swisher CB, Husain AM, Aimetti A, Gasior M. Intravenous ganaxolone for the treatment of refractory status epilepticus: Results from an open-label, dose-finding, phase 2 trial. Epilepsia. 2022 Sep;63(9):2381-2391. doi: 10.1111/epi.17343. Epub 2022 Jul 10.