Status EpilepticusPhase 2NCT05757544
Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE)
A Phase 2 study testing IV Ganaxolone in people with Status Epilepticus, run by Marinus Pharmaceuticals.
Trial summary
This study is designed to optimize the dosing regimen and evaluate the preliminary safety and efficacy of Intravenous (IV) ganaxolone as adjuvant therapy for participants with ESE receiving initial IV antiepileptic drug (AED) according to currently practiced standard of care (SOC). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Status Epilepticus
- What is being tested?
- IV Ganaxolone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2023to Feb 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females at least 18 years of age at the time of the first IP bolus dose.
- Has received benzodiazepines before or after arrival to the ED for generalized convulsive seizures lasting more than 5 minutes.
- Has received the last dose of benzodiazepine more than 5 minutes prior to the first IP bolus initiation.
- Ongoing or recurrent convulsions, or evidence of electrographic SE on rapid EEG immediately prior to the first IP bolus initiation.
- The participant has not yet received a second-line IV AED for the treatment of the current episode of SE or the first IP bolus can be initiated within 15 minutes before or 10 minutes after the administration of such IV AED(s).
Who cannot join
- The participant is intubated or the decision to proceed with intubation has been made.
- The cause of SE is acute anoxic brain injury, cardiac arrest, acute trauma, hyper- or hypo-glycemia, or eclampsia.
- The participant is known or suspected to be pregnant.
- The participant is incarcerated at the time of SE occurrence.
- Participants who pre-emptively opted out of the study.
- A known allergy or sensitivity to progesterone or allopregnanolone medications/ supplements.
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- Receiving a concomitant IV product containing Captisol®.
- Known estimated glomerular filtration rate (eGFR) <30 milliliters per minute (mL/min) and not receiving dialysis (may initiate first IP bolus prior to acute labs).
- Individual weighing or suspected to weigh <40 kilograms (kg).
- Hypotension requiring 2 or more vasopressors.
- An individual who, in the opinion of the investigator has a life expectancy of less than 24 hours.
- Use of an investigational product for which less than 30 days or 5 half-lives have elapsed from the final product administration. Participation in a non-interventional clinical study does not exclude eligibility.
- Known or suspected history or evidence of a medical condition that, in the investigator's judgment, would expose participant to an undue risk of a significant adverse event or interfere with assessments of safety or efficacy during the study.
About this study
Official title: A Study to Optimize the Dosing Regimen and Assess Safety and Efficacy of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus
This study is designed to optimize the dosing regimen and evaluate the preliminary safety and efficacy of Intravenous (IV) ganaxolone as adjuvant therapy for participants with ESE receiving initial IV antiepileptic drug (AED) according to currently practiced standard of care (SOC). The study will be composed of 2 phases: an initial, open-label, dose optimization phase followed by IV ganaxolone versus placebo double-blind phase.
- Conditions
- Status Epilepticus
- Being tested
- IV Ganaxolone, IV Placebo
- Keywords
- Established status epilepticus, Ganaxolone, Antiepileptic drug, Adjuvant therapy
- Medical subject headings
- Status Epilepticus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IV GanaxoloneDrug
IV Ganaxolone will be administered.
IV PlaceboDrug
IV Placebo will be administered
| Study group | Role | What they receive |
|---|---|---|
| Dose optimization phase (open label): IV Ganaxolone bolus (variable) followed by infusion (variable) | Experimental | Not described Receives: Drug: IV Ganaxolone |
| Double-blind phase: IV Ganaxolone + SOC | Experimental | Not described Receives: Drug: IV Ganaxolone |
| Double-blind phase: IV Placebo + SOC | Placebo comparator | Not described Receives: Drug: IV Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus or recurrence of convulsions at 1 hour after the first IP bolus administration without the use of any additional medications with anti-seizure properties
Measured: At 1 hour
Secondary outcomes (6)
Percentage of participants with no escalation of care for Status epilepticus (SE) during investigational product (IP) infusion
Measured: Up to Day 7
Time to clinical seizure and electrographic SE cessation
Measured: Up to Day 7
Percentage of participants with no escalation of care (including re-administration of IP) at any point within 24 hours from the initial IP bolus administration
Measured: Up to 24 hours
Percentage of participants with no escalation to IV anesthesia for treatment of seizures during IP infusion
Measured: Up to Day 7
Percentage of participants with no escalation to IV anesthesia for treatment of seizures within 24 hours of the initial IP bolus
Measured: Up to 24 hours
Time to clinical or electrographic seizure recurrence within 24 hours from the initiation of the first IP bolus
Measured: Up to 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Mar 2023
- Study start1 Apr 2023
- Primary completionFeb 2026 (estimated)
- Study completionFeb 2026 (estimated)
Why it stopped: for business reasons
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Marinus Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05757544
- Sponsor's protocol ID
- 1042-SE-2002
- Status last verified
- May 2024
- First submitted
- 24 Feb 2023
- Last updated
- 7 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Optimize the Dosing Regimen and Assess Safety and Efficacy of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus
- ClinicalTrials.gov ID
- NCT05757544
- Acronym / sponsor ID
- 1042-SE-2002
- Conditions
- Status Epilepticus
- Keywords
- Established status epilepticus, Ganaxolone, Antiepileptic drug, Adjuvant therapy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: for business reasons
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Marinus PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2023 (actual)
- Primary completion
- Feb 2026 (estimated)
- Study completion
- Feb 2026 (estimated)
- First posted
- 7 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to optimize the dosing regimen and evaluate the preliminary safety and efficacy of Intravenous (IV) ganaxolone as adjuvant therapy for participants with ESE receiving initial IV antiepileptic drug (AED) according to currently practiced standard of care (SOC). The study will be composed of 2 phases: an initial, open-label, dose optimization phase followed by IV ganaxolone versus placebo double-blind phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dose optimization phase (open label): IV Ganaxolone bolus (variable) followed by infusion (variable) | Experimental | Drug: IV Ganaxolone |
| Double-blind phase: IV Ganaxolone + SOC | Experimental | Drug: IV Ganaxolone |
| Double-blind phase: IV Placebo + SOC | Placebo comparator | Drug: IV Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Males or females at least 18 years of age at the time of the first IP bolus dose.
- Has received benzodiazepines before or after arrival to the ED for generalized convulsive seizures lasting more than 5 minutes.
- Has received the last dose of benzodiazepine more than 5 minutes prior to the first IP bolus initiation.
- Ongoing or recurrent convulsions, or evidence of electrographic SE on rapid EEG immediately prior to the first IP bolus initiation.
- The participant has not yet received a second-line IV AED for the treatment of the current episode of SE or the first IP bolus can be initiated within 15 minutes before or 10 minutes after the administration of such IV AED(s).
Key exclusion criteria (13 listed)
- The participant is intubated or the decision to proceed with intubation has been made.
- The cause of SE is acute anoxic brain injury, cardiac arrest, acute trauma, hyper- or hypo-glycemia, or eclampsia.
- The participant is known or suspected to be pregnant.
- The participant is incarcerated at the time of SE occurrence.
- Participants who pre-emptively opted out of the study.
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 GanaxoloneIV Ganaxolone will be administered. | Drug | Dose optimization phase (open label): IV Ganaxolone bolus (variable) followed by infusion (variable), Double-blind phase: IV Ganaxolone + SOC |
| IV PlaceboIV Placebo will be administered | Drug | Double-blind phase: IV Placebo + SOC |
Primary outcome
Absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus or recurrence of convulsions at 1 hour after the first IP bolus administration without the use of any additional medications with anti-seizure properties
Time frame: At 1 hour
6 secondary outcomes
Percentage of participants with no escalation of care for Status epilepticus (SE) during investigational product (IP) infusion
Time frame: Up to Day 7
Time to clinical seizure and electrographic SE cessation
Time frame: Up to Day 7
Percentage of participants with no escalation of care (including re-administration of IP) at any point within 24 hours from the initial IP bolus administration
Time frame: Up to 24 hours
Percentage of participants with no escalation to IV anesthesia for treatment of seizures during IP infusion
Time frame: Up to Day 7
Percentage of participants with no escalation to IV anesthesia for treatment of seizures within 24 hours of the initial IP bolus
Time frame: Up to 24 hours
Time to clinical or electrographic seizure recurrence within 24 hours from the initiation of the first IP bolus
Time frame: Up to 24 hours
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2024.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IV Ganaxolone in people with Status Epilepticus, run by Marinus Pharmaceuticals.
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 5 inclusion and 13 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: withdrawn, as of the sponsor's last update on 7 May 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Absence of electrographic (rapid electroencephalography [EEG]) evidence of status epilepticus or recurrence of convulsions at 1 hour after the first IP bolus administration without the use of any additional medications with anti-seizure properties (time frame: At 1 hour). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 7 May 2024.