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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
100
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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Marinus Research Site
Birmingham, Alabama, United States
Marinus Research Site
Phoenix, Arizona, United States
Marinus Research Site
Little Rock, Arkansas, United States
Marinus Research Site
Downey, California, United States
Marinus Research Site
La Jolla, California, United States
Marinus Research Site
Orange, California, United States
Marinus Research Site
Sacramento, California, United States
Marinus Research Site
Aurora, Colorado, United States
Marinus Research Site
Denver, Colorado, United States
Marinus Research Site
New Haven, Connecticut, United States
...and 64 more locations
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