A Phase 3 interventional study of Ganaxolone and Placebo in Status Epilepticus, sponsored by Marinus Pharmaceuticals. Completed at 74 sites in 3 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2025-05-29.
Sponsored by Marinus Pharmaceuticals (part of Immedica Pharma) · Phase 3, Interventional, and Treatment
This study evaluated the effectiveness and safety of an investigational product (IP), intravenous (IV) ganaxolone, to treat participants with status epilepticus (SE).
This is a double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of IV ganaxolone in status epilepticus. Investigational product was added to standard of care following failure of any two or more antiseizure medications (benzodiazepine and one IV antiepileptic drug (AED) or two IV AEDs. Participants were screened for inclusion/exclusion criteria prior to receiving investigational product by continuous IV infusion. Participants were followed for approximately 4 weeks. Participants who are known to be at risk for SE were consented or assented prior to an SE event.
124 studies on the registry are indexed under Status Epilepticus; 37 are open to participants now.
This study's enrollment of 100 is above the median of 80 across 75 interventional studies indexed under Status Epilepticus.
Browse Status Epilepticus studies →Marinus Pharmaceuticals is the lead sponsor of 22 studies on the registry; none are open to participants now.
Of its 9 completed or terminated interventional studies of FDA-regulated products, 9 (100%) have results posted.
First submitted before Marinus Pharmaceuticals became part of Immedica Pharma.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
SE meeting the following criteria:
a. A diagnosis of SE with or without prominent motor features based on clinical and EEG findings:
i. Diagnosis is established by:
ii. For any type of SE:
Seizure activity during the 30 minutes immediately prior to IP initiation
b. The treating clinician(s) anticipate that IV anesthesia is likely to be the next treatment for SE that persists following initiation of IP
Participants must have received any two or more of the following agents for treatment of the current episode of SE administered at an adequate dose and for a sufficient duration, in the judgment of the investigator, to demonstrate efficacy
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Placebo bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
Drug: Placebo
Ganaxolone bolus dose followed by continuous infusion for 36 hours, followed by 12 hour taper
Drug: Ganaxolone
Ganaxolone will be administered.
Placebo will be administered.
Percentage of Participants With SE Cessation Within 30 Minutes of Investigational Product (IP) Initiation Without Medications for the Acute Treatment of SE
SE cessation was determined by the investigator based on clinical and electroencephalography (EEG). Medications for the acute treatment of SE were defined as antiepileptic drugs (AEDs) administered to abort ongoing SE or prevent imminent recurrence of SE based on clinical or EEG evidence.
Time frame: Up to 30 minutes
Percentage of Participants With no Progression to Intravenous (IV) Anesthesia for 36 Hours Following Investigational Product (IP) Initiation
Percentage of participants with no progression to IV anesthesia for 36 hours following IP initiation SE cessation was based on investigator report with confirmation by assessment of concomitant medication data.
Time frame: Up to 36 hours after IP initiation
Number of Participants With Treatment Emergent Adverse Events
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant who has been administered a pharmaceutical product; it does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse event (TEAE) is defined as an AE that occurred or worsened at the time of or following IP initiation.
Time frame: Up to 4 weeks after IP initiation
Percentage of Participants With no Progression to IV Anesthesia for 72 Hours Following IP Initiation
Percentage of participants with no progression to IV anesthesia for 72 hours following IP initiation SE cessation was assessed by the investigator based on clinical and EEG features.
Time frame: Up to 72 hours after IP initiation
Time to SE Cessation Following IP Initiation
Time to SE cessation was assessed for the first 72 hours following IP using the Kaplan-Meier method.
Time frame: Up to 72 hours after IP initiation
Percentage of Participants With Any Escalation of Treatment in the First 24 Hours Following IP Initiation
SE cessation will be determined by the investigator based on clinical and EEG. Percentage of participants with any escalation of treatment in the first 24 hours following IP initiation, i.e. any medication other than IP administered for the acute treatment of SE in the first 24 hours following IP initiation was summarized
Time frame: Up to 24 hours after IP initiation
Time to Treatment Escalation in the First 24 Hours Following IP Initiation
For time to treatment escalation (any mediation used for acute treatment of SE), the estimates are based descriptive statistics on participants with treatment escalation in the first 24 hours following IP initiation. For participants without treatment escalation, the time to treatment escalation was censored at IP completion, or discontinuation from the study or death, whichever occurs earlier. The median time to treatment escalation has been presented.
Time frame: Up to 24 hours after IP initiation
Time to Initiation of Anesthesia for SE Treatment Through the Final Study Follow-up Visit/Contact
Time to initiation of anesthesia for SE treatment through the final study follow-up visit/contact, were calculated based on descriptive statistics. The estimate was censored at study discontinuation, death, or last follow-up of the participant, whichever occurs first. The median time to initiation of anesthesia has been presented.
Time frame: Up to 4 Weeks following IP initiation
Percentage of Participants Who Develop Super Refractory Status Epilepticus (SRSE) Through the Final Study Follow-up Visit/Contact
Percentage of participants who developed SRSE through the final study follow-up visit/contact was provided for each treatment group.
Time frame: Up to 4 Weeks following IP initiation
Percent Change From Baseline in Seizure Burden Through 72 Hours Following IP Initiation
The seizure burden through 72 hours following IP initiation is described as the percent of time during which there is electrographic seizure activity from IP initiation to 72 hours. The change from baseline of seizure burden was summarized using descriptive statistics by treatment group. The baseline seizure burden is defined for 30 minutes prior to IP initiation.
Time frame: Up to 72 hours after IP initiation
This was a double-blind, randomized, placebo-controlled study that evaluated the efficacy and safety of ganaxolone intravenous (IV) solution in status epilepticus (SE).
| Milestone | Ganaxolone | Placebo |
|---|---|---|
| Started | 51 | 49 |
| Completed | 30 | 35 |
| Not completed | 21 | 14 |
| Withdrew: Death | 15 | 10 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Lost to follow-up | 3 | 2 |
| Withdrew: Lack of efficacy | 0 | 1 |
| Withdrew: Participant discharged to hospice facility | 0 | 1 |
| Withdrew: Participant family decision | 1 | 0 |
| Withdrew: Participant discharged prematurely | 1 | 0 |
SE cessation was determined by the investigator based on clinical and electroencephalography (EEG). Medications for the acute treatment of SE were defined as antiepileptic drugs (AEDs) administered to abort ongoing SE or prevent imminent recurrence of SE based on clinical or EEG evidence.
| Percentage of participants | Ganaxolone | Placebo |
|---|---|---|
| Percentage of Participants With SE Cessation Within 30 Minutes of Investigational Product (IP) Initiation Without Medications for the Acute Treatment of SE | 79.59 (65.657 to 89.755) | 12.77 (4.832 to 25.741) |
Percentage of participants with no progression to IV anesthesia for 36 hours following IP initiation SE cessation was based on investigator report with confirmation by assessment of concomitant medication data.
| Percentage of participants | Ganaxolone | Placebo |
|---|---|---|
| Percentage of Participants With no Progression to Intravenous (IV) Anesthesia for 36 Hours Following Investigational Product (IP) Initiation | 63.27 (48.288 to 76.578) | 51.06 (36.064 to 65.924) |
An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant who has been administered a pharmaceutical product; it does not necessarily have a causal relationship with this treatment. Treatment-emergent adverse event (TEAE) is defined as an AE that occurred or worsened at the time of or following IP initiation.
| Participants | Ganaxolone | Placebo |
|---|---|---|
| Any TEAE | 48 | 43 |
| Serious TEAEs | 19 | 18 |
| TEAE Leading to study drug discontinuation | 1 | 2 |
| TEAE leading to withdrawal | 2 | 0 |
| TEAE resulting in death | 11 | 11 |
Percentage of participants with no progression to IV anesthesia for 72 hours following IP initiation SE cessation was assessed by the investigator based on clinical and EEG features.
| Percentage of participants | Ganaxolone | Placebo |
|---|---|---|
| Percentage of Participants With no Progression to IV Anesthesia for 72 Hours Following IP Initiation | 51.02 (36.338 to 65.575) | 46.81 (32.112 to 61.922) |
Time to SE cessation was assessed for the first 72 hours following IP using the Kaplan-Meier method.
| hours | Ganaxolone | Placebo |
|---|---|---|
| Time to SE Cessation Following IP Initiation | 0.07 (0.0 to 5.4) | 2.85 (0.0 to 40.8) |
SE cessation will be determined by the investigator based on clinical and EEG. Percentage of participants with any escalation of treatment in the first 24 hours following IP initiation, i.e. any medication other than IP administered for the acute treatment of SE in the first 24 hours following IP initiation was summarized
| Percentage of participants | Ganaxolone | Placebo |
|---|---|---|
| Percentage of Participants With Any Escalation of Treatment in the First 24 Hours Following IP Initiation | 55.10 (40.231 to 69.335) | 80.85 (66.740 to 90.851) |
For time to treatment escalation (any mediation used for acute treatment of SE), the estimates are based descriptive statistics on participants with treatment escalation in the first 24 hours following IP initiation. For participants without treatment escalation, the time to treatment escalation was censored at IP completion, or discontinuation from the study or death, whichever occurs earlier. The median time to treatment escalation has been presented.
| hours | Ganaxolone | Placebo |
|---|---|---|
| Time to Treatment Escalation in the First 24 Hours Following IP Initiation | 5.42 (0.5 to 21.6) | 2.32 (0.3 to 20.1) |
Time to initiation of anesthesia for SE treatment through the final study follow-up visit/contact, were calculated based on descriptive statistics. The estimate was censored at study discontinuation, death, or last follow-up of the participant, whichever occurs first. The median time to initiation of anesthesia has been presented.
| hours | Ganaxolone | Placebo |
|---|---|---|
| Time to Initiation of Anesthesia for SE Treatment Through the Final Study Follow-up Visit/Contact | 15.00 (1.5 to 417.2) | 3.17 (0.6 to 202.7) |
Percentage of participants who developed SRSE through the final study follow-up visit/contact was provided for each treatment group.
| Percentage of participants | Ganaxolone | Placebo |
|---|---|---|
| Percentage of Participants Who Develop Super Refractory Status Epilepticus (SRSE) Through the Final Study Follow-up Visit/Contact | 22.45 (11.774 to 36.624) | 25.53 (13.945 to 40.350) |
The seizure burden through 72 hours following IP initiation is described as the percent of time during which there is electrographic seizure activity from IP initiation to 72 hours. The change from baseline of seizure burden was summarized using descriptive statistics by treatment group. The baseline seizure burden is defined for 30 minutes prior to IP initiation.
| Percent change | Ganaxolone | Placebo |
|---|---|---|
| Percent Change From Baseline in Seizure Burden Through 72 Hours Following IP Initiation | -59.1 ± 88.98 | -93.9 ± 19.79 |
Collected over Up to 4 Weeks. Non-serious events are listed at a 3% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Placebo | 11/49 (22.4%) | 18/49 (36.7%) | 39/49 (79.6%) |
| Ganaxolone | 15/51 (29.4%) | 19/51 (37.3%) | 43/51 (84.3%) |
| Event | Placebo | Ganaxolone |
|---|---|---|
| Respiratory failureRespiratory, thoracic and mediastinal disorders | 3/49 | 5/51 |
| HypotensionVascular disorders | 1/49 | 5/51 |
| Subdural haematomaInjury, poisoning and procedural complications | 3/49 | 3/51 |
| PneumoniaInfections and infestations | 2/49 | 1/51 |
| Septic shockInfections and infestations | 2/49 | 1/51 |
| Cardio-respiratory arrestCardiac disorders | 0/49 | 2/51 |
| Status epilepticusNervous system disorders | 1/49 | 2/51 |
| Heparin-induced thrombocytopeniaBlood and lymphatic system disorders | 1/49 | 0/51 |
| Cardiac arrestCardiac disorders | 1/49 | 1/51 |
| Sinus tachycardiaCardiac disorders | 1/49 | 0/51 |
| Event | Placebo | Ganaxolone |
|---|---|---|
| HypotensionVascular disorders | 8/49 | 12/51 |
| Urinary tract infectionInfections and infestations | 4/49 | 10/51 |
| PyrexiaGeneral disorders | 8/49 | 4/51 |
| HypokalaemiaMetabolism and nutrition disorders | 5/49 | 1/51 |
| PneumoniaInfections and infestations | 0/49 | 5/51 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 0/49 | 5/51 |
| HypothermiaGeneral disorders | 2/49 | 5/51 |
| HyperglycaemiaMetabolism and nutrition disorders | 4/49 | 2/51 |
| HypernatraemiaMetabolism and nutrition disorders | 4/49 | 1/51 |
| ThrombocytopeniaBlood and lymphatic system disorders | 4/49 | 0/51 |
Safety Population comprises all participants who received the investigational product.
| Age, Continuous(years) | Ganaxolone | Placebo | Total |
|---|---|---|---|
| Mean | 57.9 ± 19.40 | 57.0 ± 19.03 | 57.5 ± 19.13 |
| Sex: Female, Male(Participants) | Ganaxolone | Placebo | Total |
|---|---|---|---|
| Female | 21 | 19 | 40 |
| Male | 30 | 30 | 60 |
| Ethnicity (NIH/OMB)(Participants) | Ganaxolone | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 7 | 6 | 13 |
| Not Hispanic or Latino | 43 | 43 | 86 |
| Unknown or Not Reported | 1 | 0 | 1 |
| Race (NIH/OMB)(Participants) | Ganaxolone | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 13 | 14 | 27 |
| White | 34 | 32 | 66 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 4 | 3 | 7 |
PDFs hosted by the registry, ClinicalTrials.gov.
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Studies of the drug tested here: Ganaxolone