Status EpilepticusPhase 2NCT03350035
Intravenous Ganaxolone as Adjunctive Therapy to Treat Subjects With Status Epilepticus
A Phase 2 study testing IV Ganaxolone active in people with Status Epilepticus and Convulsive Status EPILEPTICUS, run by Marinus Pharmaceuticals at 8 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Status Epilepticus, Convulsive Status EPILEPTICUS, Non Convulsive Status Epilepticus
- What is being tested?
- IV Ganaxolone active
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 8 sitesUnited States
- Study dates
- 19 Feb 2018to 18 Sep 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects 12 years of age and older
- Clinical and/or electrographic seizures
Who cannot join
- 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
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Grady Hospital
Atlanta, Georgia, 30303
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Henry Ford Hospital
Detroit, Michigan, 48202
Duke Medical Center
Durham, North Carolina, 27710
Nicklaus Children's Hospital
Miami, Florida, 33155
Oschner Clinic Foundation
New Orleans, Louisiana, 70121
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
Nemours/AI duPont Hospital for Children
Wilmington, Delaware, 19803
About this study
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
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.
Read the 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.
- Conditions
- Status Epilepticus, Convulsive Status EPILEPTICUS, Non Convulsive Status Epilepticus, Epilepsy
- Being tested
- IV Ganaxolone active, IV Placebo, non-active
- Medical subject headings
- Status Epilepticus, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IV Ganaxolone activeDrug
IV
IV Placebo, non-activeDrug
IV
| Study group | Role | What they receive |
|---|---|---|
| IV Ganaxolone active | Experimental | Ganaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper. Receives: Drug: IV Ganaxolone active |
| IV Placebo, non-active | Placebo comparator | Placebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper. Receives: Drug: IV Placebo, non-active |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 24 hours post study drug initiation
Secondary outcomes (4)
Time to Cessation of SE
Summary of Time to SE Cessation
Measured: Time to SE Cessation, assessed up to 24 hours
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
Measured: Drug initiation through follow-up period, up to approximately 4 weeks
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.
Measured: Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
Seizure Burden
Seizure Burden (%) Baseline and Percentage Change from Baseline by Time Point
Measured: Baseline (Pre-dose) to <-24hrs (Post Dose)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Nov 2017
- Study start19 Feb 2018
- Primary completion18 Sep 2019
- Study completion18 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Status EpilepticusStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Maciej Gasior, MD, PhD, Study director, Marinus Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03350035
- Sponsor's protocol ID
- 1042-SE-2001
- Status last verified
- Mar 2023
- First submitted
- 17 Nov 2017
- Last updated
- 9 Mar 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 35748707
Study at a glance
From the registry record, last updated by the sponsor on 9 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03350035
- Acronym / sponsor ID
- 1042-SE-2001
- Conditions
- Status Epilepticus, Convulsive Status EPILEPTICUS, Non Convulsive Status Epilepticus, Epilepsy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Feb 2018 (actual)
- Primary completion
- 18 Sep 2019 (actual)
- Study completion
- 18 Sep 2019 (actual)
- First posted
- 22 Nov 2017 (actual)
- Results
- Posted 15 Dec 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| IV Ganaxolone active | Experimental | Drug: IV Ganaxolone activeGanaxolone IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper. |
| IV Placebo, non-active | Placebo comparator | Drug: IV Placebo, non-activePlacebo IV loading dose with continuous infusion (maintenance dose) for 2-4 days followed by an 18-hour taper. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Subjects 12 years of age and older
- Clinical and/or electrographic seizures
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IV Ganaxolone activeIV | Drug | IV Ganaxolone active |
| IV Placebo, non-activeIV | Drug | IV Placebo, non-active |
Primary outcome
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment
Time frame: 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.
4 secondary outcomes
Time to Cessation of SE
Time frame: Time to SE Cessation, assessed up to 24 hours
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
Number of Participants With No SE Recurrence Per Principal Investigator
Time frame: Baseline (within 24hrs of treatment) through follow up period, up to approximately 4 weeks.
Seizure Burden
Time frame: Baseline (Pre-dose) to <-24hrs (Post Dose)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Mar 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35748707Vaitkevicius 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Maciej Gasior, MD, PhD study director
Marinus Pharmaceuticals
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IV Ganaxolone active in people with Status Epilepticus and Convulsive Status EPILEPTICUS, run by Marinus Pharmaceuticals at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Dec 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Did Not Require an IV Anesthetic Drug for SE Treatment (time frame: 24 hours post study drug initiation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03350035, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Mar 2023.