Status Epilepticus – RAISE

Name: RAISE (A double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of intravenous ganaxolone in status epilepticus)

Contact: Ralisa Pop (Lead CRC, 352-273-5550 x45693) and Jessica Spana (Back-up CRC, 352-273-5554), Dr. Billy Roth (PI)

Enrolling hours: Everyday but no enrollments after 5pm, still need to work out on call schedule.

Diagnosis: SE

Inclusion:

  1. Participant, participant’s parent, guardian, or LAR must provide signature of informed consent/assent, and once capable (per institution guidelines), there must be documentation of consent/assent by the participant demonstrating they are willing and aware of the investigational nature of the study and related procedures
  2.    Male or females 18 years of age and older at the time of the first dose of IP
  3.    Convulsive or nonconvulsive SE with seizure burden warranting imminent progression to IV anesthesia for seizure control. Ictal patterns and burden defined below.
  • Convulsive SE: Clinical and EEG seizure activity or
  • Nonconvulsive SE: Must have an ictal EEG pattern consistent with modified Salzburg criteria
  • Ictal burden of approximately 6 minutes or more within 30 minutes immediately prior to IP initiation is targeted
  1.   Participants must have received a benzodiazepine and two or more of the following second-line IV AEDs for treatment of the current episode of SE, administered at an adequate dose and duration to demonstrate efficacy, in the opinion of the investigator
  • Fosphenytoin/phenytoin,
  • Valproic acid,
  • Levetiracetam,
  • Lacosamide
  • Brivaracetam, or
  • Phenobarbital
  1.   BMI < 40 or, if BMI is not able to be calculated at screening, participant is assessed by investigator as not morbidly obese

Exclusion:

  1. Life expectancy of less than 24 hours
  2. Anoxic brain injury or a rapidly reversable metabolic condition as the primary cause of SE (e.g., hypoglycemia < 50 mg/dL or hyperglycemia > 400 mg/dL)
  3. Participants on IV anesthesia (e.g., midazolam, propofol, thiopental, or pentobarbital) with the primary intent specifically to treat seizures or achieve burst suppression
  4. Seizure burden or clinical state would NOT warrant IV anesthesia for seizure control over the next 24 hours
  5. Participants with an advanced directive that would not allow the institution to administer their SOC for the treatment of SE (e.g., directive Do Not Intubate)
  6. Participants known or suspected to be pregnant
  7. Participants with known allergy or sensitivity to progesterone or allopregnanolone medications/supplements
  8. Receiving a concomitant IV product containing Captisol®
  9. Known or suspected hepatic insufficiency or hepatic failure
  10. Known or suspected stage 3B (moderate to severe; eGFR 44-30 mL/min/1.73m2), stage 4 (severe; eGFR 29-15 mL/min/1.73m2), or stage 5 (kidney failure; eGFR < 15mL/min/1.73m2 or dialysis) kidney disease
  11. 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.
  12. 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.