A Phase 3 interventional study of Ganaxolone and Placebo in Refractory Status Epilepticus, sponsored by Marinus Pharmaceuticals. Withdrawn at 52 sites in 16 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-05-14.
Sponsored by Marinus Pharmaceuticals (part of Immedica Pharma) · Phase 3, Interventional, and Treatment
This is a multicenter, double-blind, randomized, placebo-controlled study that will evaluate the efficacy, safety, and tolerability of intravenous (IV) ganaxolone versus placebo co-administered with IV antiepileptic drug (AED) according to standard of care for the treatment of RSE. Approximately 70 participants will be randomized in a 1:1 ratio to receive ganaxolone IV solution or placebo IV solution along with standard of care (SOC) IV AED.
124 studies on the registry are indexed under Status Epilepticus; 37 are open to participants now.
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 warranting imminent progression of treatment meeting the following criteria:
a) A diagnosis of SE, warranting imminent progression of treatment for seizure control, with or without prominent motor features based on clinical and EEG findings:
i. Diagnosis is established by:
ii. For any type of SE:
Participants must have received a benzodiazepine and at least 1 of the following IV AEDs for treatment of the current episode of SE administered at an adequate dose and for a sufficient duration, in the judgement of the investigator, to demonstrate efficacy. The benzodiazepine and at least 1 of the IV AEDs must have been administered at a dose that would be expected to be effective for the termination of the current episode of SE.
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.
Drug: Ganaxolone · Drug: Standard of care
Drug: Placebo · Drug: Standard of care
Ganaxolone will be administered as IV solution.
Placebo will be administered as IV solution.
A non-anesthetic medication not previously used for treatment of SE within the current episode and will be administered at a dose sufficient for the termination of SE according to investigator judgment.
Percentage of participants who will report cessation of SE within 30 minutes of investigational product (IP) initiation of at least 30 minutes duration
Status epilepticus cessation will be determined by the investigator based on clinical and electroencephalography (EEG) features
Time frame: Up to 30 minutes
Percentage of participants who will report no escalation of treatment for persistent or recurrent SE within 36 hours of IP initiation
Time frame: Up to 36 hours
Percentage of participants who will report cessation of SE within 30 minutes of IP initiation of at least 30 minutes duration
Time frame: Up to 30 minutes
Percentage of participants who will report no escalation of treatment for persistent or recurrent SE within 72 hours of IP initiation
Time frame: Up to 72 hours
Time to SE cessation
Time frame: Up to 72 hours
Percentage of participants having cessation of SE within 30 minutes of IP initiation of at least 30 minutes duration without escalation of treatment
Time frame: Up to 30 minutes
Percentage of participants reporting no escalation of treatment for persistent or recurrent SE within 36 hours of IP initiation
Time frame: Up to 36 hours
Percentage of participants reporting no escalation of treatment for persistent or recurrent SE within 72 hours of IP initiation
Time frame: Up to 72 hours
Change from Baseline in Modified Rankin Scale (mRS) at the time of hospital discharge
The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered a stroke or other causes of neurological disability. The scale runs from 0-6, running from perfect health without symptoms to death where 0 - No symptoms; 1=No significant disability. Able to carry out all usual activities, despite some symptoms; 2=Slight disability. Able to look after own affairs without assistance, but unable to carry out all previous activities; 3=Moderate disability. Requires some help, but able to walk unassisted; 4=Moderately severe disability. Unable to attend to own bodily needs without assistance, and unable to walk unassisted; 5=Severe disability. Requires constant nursing care and attention, bedridden, incontinent; 6=Dead. Higher scores will indicate high degree of disability.
Time frame: Baseline and Up to Day 31
Change from Baseline in level of responsiveness as assessed by the Full Outline of UnResponsiveness (FOUR) Score scale
The FOUR Score is a 17-point scale (with potential scores ranging from 0 - 16). Decreasing FOUR Score is associated with worsening level of consciousness. The FOUR coma scale includes 4 parameters with a minimum score of 0 and a maximum score of "4" for each of them: eye reactions (eye opening and tracking), motor responses (pain response and simple commands), stem reflexes (pupillary, corneal and cough) and respiratory patterns (respiratory rhythm and respiratory attempts in patients on a ventilator). The points are summed up, their sum is estimated. The interpretation of results will be as 15 to 16 score: clear consciousness; Less than 15: Impairment of consciousness; from 4 to 8: Coma and 0-4: Death. Lower the score, the greater the coma gravity.
Time frame: Baseline and at 24, 36 and 72 hours
Change from Baseline in level of sedation/ agitation as assessed by Richmond Agitation and Sedation Scale (RASS)
The RASS is a medical scale used to measure the agitation or sedation level. It is a 10-point scale that ranges from -5 to +4 with -5=unarousable and +4=combative. Zero means the patient is alert and calm. higher scores indicate more agitation.
Time frame: Baseline and at 24, 36 and 72 hours
Percentage of participants with mRS > 3 at the time of hospital discharge
The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered a stroke or other causes of neurological disability. The scale runs from 0-6, running from perfect health without symptoms to death where 0 - No symptoms; 1=No significant disability. Able to carry out all usual activities, despite some symptoms. 2=Slight disability. Able to look after own affairs without assistance, but unable to carry out all previous activities. 3=Moderate disability. Requires some help, but able to walk unassisted. 4=Moderately severe disability. Unable to attend to own bodily needs without assistance, and unable to walk unassisted. 5=Severe disability. Requires constant nursing care and attention, bedridden, incontinent. 6=Dead. Higher scores will indicate high degree of disability.
Time frame: Up to 122 hours
Change from Baseline in Clinical Global Impression-Improvement (CGI-I) following IP initiation and at hospital discharge
The CGI-I is a 7-point Likert scale that the parent(s)/caregiver(s)/legally authorized representative (LAR)(s) and clinician uses to rate the change in overall seizure control, behavior, safety, and tolerability after initiation of the IP relative to Baseline (prior to treatment with the IP). It was rated as: 1- "very much improved", 2- "much improved', 3- "minimally improved", 4- "no change", 5- "minimally worse", 6- "much worse", and 7- "very much worse". Higher scores indicated worse condition.
Time frame: Baseline and at 24, 36, and 72 hours
Number of hours on positive pressure ventilation attributable to the episode of SE or its treatment
Healthcare Utilization Questionnaires include the Hospitalization Questionnaire and the Positive Pressure Ventilation (PPV) and Intubation Questionnaire. The Hospitalization Questionnaire should be collected at hospital discharge or at final study visit/contact. The need for non-invasive or invasive ventilatory support within 24 hours prior to IP initiation and following IP initiation and within 48 hours following IP discontinuation should be collected as close as possible to the event.
Time frame: Up to 4 Weeks
Number of hours on positive pressure ventilation
Time frame: Up to 4 Weeks
Length of stay (days) in intensive care unit (ICU)
Time frame: Up to 4 Weeks
Length of stay (days) in hospital
Time frame: Up to 4 Weeks
Percentage of participants requiring artificial ventilation after initiation of IP
Time frame: Up to 122 hours
Percentage of participants not requiring IV anesthesia for SE treatment within 36 hours of IP initiation
Time frame: Up to 36 hours
Percentage of participants not requiring IV anesthesia for SE treatment within 72 hours of IP initiation
Time frame: Up to 72 hours
Percentage of participants not requiring IV anesthesia for SE treatment through the final study follow-up visit/contact
Time frame: Up to 4 Weeks
Percentage of participants who do not develop super refractory status epilepticus (SRSE) through the final study follow-up visit/contact
Time frame: Up to 4 Weeks
Change from Baseline in Euro Quality of Life (five-level EuroQoL five-dimensional [EQ-5D-5L]) score
The EQ-5D-5L is the EuroQoL 5D-5L, a descriptive system that comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The participant is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions. This decision results in a 1-digit number that expresses the level selected for that dimension. The digits for the five dimensions can be combined into a 5-digit number that describes the participant's health state. A positive score means quality of life improvement, a negative score, a worsening of quality of life. Higher scores indicate more problems.
Time frame: Baseline and Up to 4 Weeks
Number of AEDs at discharge
Time frame: Up to 122 hours
Percentage of participants requiring supplemental oxygen after initiation of IP
Time frame: Up to 4 Weeks
Plan to share: No
No publications or documents are linked to this record.
Start is estimated for Mar 2024; primary completion for Aug 2024, 5 months later.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in May 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ganaxolone