Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
Primary Purpose
Status Epilepticus, Convulsive Status EPILEPTICUS, Non Convulsive Status Epilepticus
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
IV Ganaxolone active
IV Placebo, non-active
Sponsored by
About this trial
This is an interventional treatment trial for Status Epilepticus
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
Sites / Locations
- Nemours/AI duPont Hospital for Children
- Nicklaus Children's Hospital
- Grady Hospital
- Oschner Clinic Foundation
- Brigham and Women's Hospital
- Henry Ford Hospital
- Duke Medical Center
- Allegheny General Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
IV Ganaxolone active
IV Placebo, non-active
Arm Description
Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Outcomes
Primary Outcome Measures
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
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
Time to Cessation of SE
Summary of Time to SE Cessation
Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE
Number of participants who Required No Escalation of Treatment for Ongoing or Recurrent SE
Number of Participants With No SE Recurrence Per Principal Investigator
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
Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point
Full Information
NCT ID
NCT03350035
First Posted
November 17, 2017
Last Updated
March 8, 2023
Sponsor
Marinus Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT03350035
Brief Title
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
2. Study Status
Record Verification Date
March 2023
Overall Recruitment Status
Completed
Study Start Date
February 19, 2018 (Actual)
Primary Completion Date
September 18, 2019 (Actual)
Study Completion Date
September 18, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Marinus Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Status Epilepticus, Convulsive Status EPILEPTICUS, Non Convulsive Status Epilepticus, Epilepsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
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.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
17 (Actual)
8. Arms, Groups, and Interventions
Arm Title
IV Ganaxolone active
Arm Type
Experimental
Arm Description
Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Arm Title
IV Placebo, non-active
Arm Type
Placebo Comparator
Arm Description
Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper.
Intervention Type
Drug
Intervention Name(s)
IV Ganaxolone active
Intervention Description
IV
Intervention Type
Drug
Intervention Name(s)
IV Placebo, non-active
Intervention Description
IV
Primary Outcome Measure Information:
Title
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
Description
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.
Time Frame
24 hours post study drug initiation
Secondary Outcome Measure Information:
Title
Time to Cessation of SE
Description
Summary of Time to SE Cessation
Time Frame
Time to SE Cessation, assessed up to 24 hours
Title
Number of Participants Who Required No Escalation of Treatment for Ongoing or Recurrent SE
Description
Number of participants who Required No Escalation of Treatment for Ongoing or Recurrent SE
Time Frame
Drug initiation through follow-up period, up to approximately 4 weeks
Title
Number of Participants With No SE Recurrence Per Principal Investigator
Description
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.
Time Frame
Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
Title
Seizure Burden
Description
Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point
Time Frame
Baseline (Pre-dose) to <-24hrs (Post Dose)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Maciej Gasior, MD, PhD
Organizational Affiliation
Marinus Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Nemours/AI duPont Hospital for Children
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Facility Name
Nicklaus Children's Hospital
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Grady Hospital
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30303
Country
United States
Facility Name
Oschner Clinic Foundation
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Henry Ford Hospital
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48202
Country
United States
Facility Name
Duke Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Allegheny General Hospital
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15212
Country
United States
12. IPD Sharing Statement
Citations:
PubMed Identifier
35748707
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.
Results Reference
derived
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Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
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