Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus

NCT ID: NCT03350035

Last Updated: 2023-03-09

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

17 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-19

Study Completion Date

2019-09-18

Brief Summary

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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

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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.

Conditions

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Status Epilepticus Convulsive Status EPILEPTICUS Non Convulsive Status Epilepticus Epilepsy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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IV Ganaxolone active

Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.

Group Type EXPERIMENTAL

IV Ganaxolone active

Intervention Type DRUG

IV

IV Placebo, non-active

Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.

Group Type PLACEBO_COMPARATOR

IV Placebo, non-active

Intervention Type DRUG

IV

Interventions

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IV Ganaxolone active

IV

Intervention Type DRUG

IV Placebo, non-active

IV

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Marinus Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Maciej Gasior, MD, PhD

Role: STUDY_DIRECTOR

Marinus Pharmaceuticals

Locations

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Nemours/AI duPont Hospital for Children

Wilmington, Delaware, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Grady Hospital

Atlanta, Georgia, United States

Site Status

Oschner Clinic Foundation

New Orleans, Louisiana, United States

Site Status

Brigham and Women's Hospital

Boston, Massachusetts, United States

Site Status

Henry Ford Hospital

Detroit, Michigan, United States

Site Status

Duke Medical Center

Durham, North Carolina, United States

Site Status

Allegheny General Hospital

Pittsburgh, Pennsylvania, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 35748707 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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1042-SE-2001

Identifier Type: -

Identifier Source: org_study_id

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