Now that protocol-specific questions from the FDA have been answered, a phase 3 study (NCT03350035) of ganaxolone (Marinus, Radnor, PA) for refractory status epilepticus (RSE) will be initiated. Enrollment in the RAISE trial is scheduled to begin in October 2020.
The phase 3 Raise trial is a randomized double-blind placebo-controlled trial in participants who have status epilepticus that does not respond to benzodiazepines and 2 or more second-line intravenous antiseizure medications. Approximately 125 participants will be enrolled and randomly assigned to receive ganaxolone or placebo adjunctive to the standard of the participant’s care. The study is powered to detect a 30% efficacy difference between ganaxolone and placebo.
“In our recent phase 2 trial in RSE, status epilepticus was controlled at a median time of 5 minutes after starting ganaxolone, with no patients progressing to intravenous anesthesia for control of seizures within 24 hours, the primary study endpoint. There is a pressing need for better treatments for RSE, and we are eager to begin our phase 3 clinical trial for this indication,” said Joe Hulihan, MD, chief medical officer.
Participants on ganaxolone will receive an IV bolus, then a 36-hour infusion, followed by a 12-hour taper for a total 48-hour treatment period. This treatment targets a plasma concentration of 500 ng/mL or more for 12 hours. The RAISE trial aimS to have results for a proportion of participants with SE cessation within 30 minutes of treatment initiation without other medications for the treatment of seizures.
Carlos E.V. Sollero, MD; Hsin-Pin Lin, MD; Miguel Chuquilin, MD; James Wymer, MD; and Aaron M. Carlson, MD
Ajay Sood, MD, PhD, and David Bennett, MD
Adam M. Staffaroni, PhD; Elena Tsoy, PhD; Jack Taylor, BS; Adam L. Boxer, MD, PhD; and Katherine L. Possin, PhD