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This study plans to use ganaxolone, an investigational medicine, in addition to current first- and second-line treatment medicines used in an emergency department setting. The standard first-line treatment medicine is a benzodiazepine and the second-line treatment medicine is a US Food and Drug Administration (FDA) approved antiepileptic drug normally used to stop prolonged seizure activity. Investigational means ganaxolone has not been approved by the FDA for use outside of clinical studies like this one. Ganaxolone has been studied in many clinical trials across a variety of seizure disorders.

For more information please follow this link.

There are currently no approved treatment options specifically for SE or ESE. There are several FDA approved antiepileptic drugs that can be used as a second-line treatment to try and stop prolonged seizures, however, 50% of patients may continue to have seizures after being given them. Since current medicines are not always effective at treating ESE, it is important for clinical studies to see if an investigational medicine may help.

Once a patient has enrolled in the study, they may be a participant for up to 4 weeks. Because there are multiple study locations and each will have patients enrolling at different times, the clinical study will be open for 2 years and the goal is to enroll up to 120 patients across the US.

Any patient who is 18 years or older arriving to the emergency department that has received a dose of first-line treatment such as a benzodiazepine and is still experiencing persistent seizures may be considered for the study.

Click for a list of hospitals participating in the study.
Status epilepticus is a neurological emergency and patients experiencing prolonged seizures must be treated quickly, because the chances of permanent brain damage and death increase with extended seizure duration.1 As a result, treatment cannot ethically be delayed while the study doctor attempts to obtain consent from a legally authorized representative (LAR) or objection from a family member. In research studies such as these, the FDA allows the research study to proceed with Exception from Informed Consent (EFIC).

Click to learn more about Exception from Informed Consent (EFIC)

In the event that a LAR can be identified and is able to provide consent for the patient without delaying treatment, the research doctor will seek to obtain consent from the LAR. Efforts made to contact the LAR and obtain consent will be documented. In the event that a LAR cannot be identified but a family member can be identified without delaying treatment, the research doctor will provide an opportunity for the family member to object to enrolling the patient in the study. 1. Madžar, D., Geyer, A., Knappe, R. U., Gollwitzer, S., Kuramatsu, J. B., Gerner, S. T., Hamer, H. M., & Huttner, H. B. (2016). Association of seizure duration and outcome in refractory status epilepticus. Journal of neurology, 263(3), 485–491. https://doi.org/10.1007/s00415-015-7992-0

Safeguards are put in place to help ensure patients are protected from unethical research practices:

  1. The research doctor must inform the community about the study and obtain feedback about the study before it begins.
  2. The research doctor must inform the community of plans for the study, its risks and expected benefits before it starts and provide an opportunity for community members to decline or refuse participation in the study.
  3. The research doctor must inform the community about the results of the study when it’s completed.
  4. The research doctor must plan for contact of legally authorized representatives (LAR) or family members to seek informed consent for the study participants' continued participation in the study without delaying treatment if possible or as soon as possible after enrollment.
  5. The research doctor must ensure there is a separate group of medical experts formed to monitor the safety of patients enrolled in the study.

Ganaxolone has been given orally to over 1,900 people and intravenously to over 130 people. The longest infusion given to date has been 114 hours. Ganaxolone is still an investigational medicine and is being studied in a number of clinical studies to evaluate its safety and efficacy as it relates to specific epilepsies and seizure disorders. As with other investigational medicines with limited number of patients the risks associated with receiving ganaxolone by infusion are not fully understood.

Increased sleepiness and dizziness have been the most common (≥10%) side effects of ganaxolone. Less reported (≥1% – < 10%) side effects include:

  • Poor coordination
  • Difficulty maintaining balance
  • Sedation (unusual state of calmness, inactivity)
  • Abnormal speech
  • Lethargy (lack of energy and enthusiasm)
  • Disturbance in attention (inability to focus)
  • Fatigue
  • Asthenia (weakness)
  • Gait disturbance (abnormal walking)
  • Fever
  • Euphoric mood (unusually happy or giddy)
  • Confusion
  • Irritability
  • Cough
  • Eye problems (blurred or double vision)
  • Decreased appetite

Participating Sites

Henry Wang, MD
Ohio State Wexner Medical Center
376 W. 10th Ave, 725 Prior Hall
Columbus, OH 43210
614-293-8305

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