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Study on Ganaxolone for Patients with Refractory Status Epilepticus

Fast replyInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition known as Refractory Status Epilepticus (RSE). RSE is a severe form of seizure that does not respond to standard treatments. The study is testing a medication called Ganaxolone, which is given through an intravenous (IV) method, meaning it is administered directly into the bloodstream. Ganaxolone is being tested to see if it can help stop seizures when added to the usual care that patients with RSE receive.

The purpose of the study is to evaluate how effective, safe, and tolerable Ganaxolone is when used alongside standard treatments for RSE. Participants in the study will receive either Ganaxolone or a placebo, which looks like the medication but does not contain the active ingredient. The study is designed to be double-blind, meaning neither the participants nor the researchers know who is receiving Ganaxolone and who is receiving the placebo, to ensure unbiased results.

Throughout the study, participants will be closely monitored to see if their seizures stop within a short time after starting the treatment and to check if there is a need for additional treatments. The study will also look at the safety of Ganaxolone by observing any side effects that may occur. The trial aims to provide valuable information on whether Ganaxolone can be a beneficial addition to the treatment options for people with RSE.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, informed consent must be obtained. This means that either you or your legally authorized representative (LAR) will sign a document indicating understanding and willingness to participate in the study. If you are unable to make decisions, the clinician will decide based on your best interests.

  2. Step 2

    Initial assessment

    An initial assessment will be conducted to confirm eligibility. This includes verifying that you are 18 years or older and have a diagnosis of refractory status epilepticus (RSE). You must have received certain medications for your current condition.

  3. Step 3

    Treatment administration

    You will receive an intravenous (IV) treatment. This involves administering the study medication, ganaxolone, or a placebo. The treatment is given in addition to your standard care for RSE.

  4. Step 4

    Monitoring and evaluation

    Your response to the treatment will be monitored. The primary goal is to see if the status epilepticus stops within 30 minutes and does not return within 36 hours. Safety and any side effects will also be closely observed.

  5. Step 5

    Follow-up assessments

    Further assessments will be conducted at 24, 36, and 72 hours after starting the treatment. These assessments will evaluate your level of responsiveness, sedation, and overall improvement.

  6. Step 6

    Final evaluation

    At the time of hospital discharge or your last assessment, a final evaluation will be performed. This will include measuring your recovery and any remaining symptoms.

Who can join the trial?

5 criteria

  • The participant or their legally authorized representative must sign an informed consent form. This means they agree to join the study and understand what it involves. If the participant cannot make medical decisions, a doctor may decide based on what is best for them.
  • The participant must be a male or female who is 18 years of age or older at the time of the first dose of the study medication.
  • The participant must have a condition called Status Epilepticus (SE), which is a type of seizure. This can be with or without noticeable movements. The diagnosis is based on clinical signs and an EEG (a test that measures brain activity). The participant must have had about 6 minutes of seizure activity over a 30-minute period within the hour before starting the study medication, and seizure activity during the 30 minutes immediately before starting the study medication.
  • The participant must have been treated with a medication called a benzodiazepine and at least one of the following intravenous (IV) anti-seizure drugs for the current episode of SE: IV Fosphenytoin/phenytoin, IV Valproic acid, IV Levetiracetam, IV Lacosamide, IV Brivaracetam, or IV Phenobarbital. These medications must have been given in doses expected to be effective for stopping the seizures.
  • The participant's Body Mass Index (BMI) must be less than 40. If BMI cannot be calculated, the participant must be assessed by the investigator as not being morbidly obese. BMI is a measure of body fat based on height and weight.

Who cannot join the trial?

10 criteria

  • Patients who have a history of severe allergic reactions to the study medication cannot participate.
  • Patients with certain heart conditions that are not well controlled are not eligible.
  • Patients who are pregnant or breastfeeding cannot take part in the study.
  • Patients who have participated in another clinical trial within the last 30 days are excluded.
  • Patients with severe liver or kidney disease are not eligible.
  • Patients who are unable to follow the study procedures or attend the required visits cannot participate.
  • Patients with a history of drug or alcohol abuse within the past year are excluded.
  • Patients who are currently taking medications that might interfere with the study treatment are not eligible.
  • Patients with certain mental health conditions that are not well controlled cannot participate.
  • Patients who have had a major surgery within the last 30 days are excluded.
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Investigated drugs

Ganaxolone is a medication being tested in this clinical trial. It is given through an intravenous (IV) line, which means it is delivered directly into the bloodstream. Ganaxolone is being studied to see if it can help people who are experiencing a severe type of seizure called refractory status epilepticus. This condition is when seizures do not stop even after trying other treatments. The goal of using ganaxolone is to see if it can work alongside standard seizure medications to help control these difficult seizures, while also checking if it is safe and well-tolerated by patients.

What is already known about the treatment

Ganaxolone – Ganaxolone is administered intravenously, meaning it is given directly into a vein through an injection. It is currently being studied in clinical trials to assess its effectiveness, safety, and tolerability when used alongside standard treatments for a severe form of epilepsy known as refractory status epilepticus (RSE). This medication is primarily indicated for patients who do not respond to typical anti-epileptic drugs. At the molecular level, ganaxolone works by enhancing the activity of a neurotransmitter called GABA, which helps calm overactive brain signals that can lead to seizures. It is classified as a neuroactive steroid and acts as a positive allosteric modulator of the GABA-A receptor.

Investigated diseases

Refractory Status Epilepticus – Refractory Status Epilepticus is a severe neurological condition where a person experiences continuous or repeated seizures that do not respond to initial treatment. It is characterized by prolonged seizure activity lasting more than 30 minutes or recurrent seizures without regaining consciousness between them. The condition progresses as the brain remains in a state of persistent electrical activity, which can lead to further complications. Over time, the ongoing seizures can cause damage to brain cells and disrupt normal brain function. The condition requires immediate medical attention to prevent further neurological damage. It is often seen in individuals who have not responded to standard anti-seizure medications.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2022-502540-12-00Protocol code1042-SE-3004Estimated enrolment76 patientsSponsorMarinus Pharmaceuticals Inc.

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On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).