Abstract Details

Title Phase I/II Trial of SAGE-547 in Super-Refractory Status Epilepticus

Topic Epilepsy/Clinical Neurophysiology (EEG)

Presentation(s) (-)

Poster/Presentation
Number 004

Objective

Background SAGE-547 is a solution of allopregnanolone in (Captisol®). There is ample evidence indicating a role for allopregnanolone in the treatment of seizures and status epilepticus (SE). For example, In the rat kainate-induced model of SE, acute administration of allopregnanolone eliminates SE at both 10 and 70 minutes supporting the hypothesis that allopregnanolone may provide anticonvulsant efficacy at a time when prolonged seizure activity has become resistant to benzodiazepine treatment.

Design/Methods This is an open-label trial of patients (n=20) 2 years and older diagnosed with super-refractory status epilepticus (SRSE). The study consists of a 4-day treatment period followed by a 1-day taper and a 3-week follow-up. Outcome Measures: 1) Successful wean off of cIV-AED after hour 48, 2) Successful taper off SAGE-547 after hour 96. Additional measures were Clinical Global Impression of Severity/Improvement, GCS and cEEG. SAGE-547 is administered adjunctive to standard of care as a 1 hour loading dose followed by a continuous infusion.

Results 71% (12/17) of evaluable patients were successfully weaned off their anesthetic agents while SAGE-547 was being administered. 71% were also successfully weaned off SAGE-547. Generally, patients who responded to SAGE-547 demonstrated rapid improvement over the first five days following treatment. Responders continued to improve over the 30-day follow-up period. 13 patients (65%) reported SAE’s; none were considered drug-related. Mean exposure was approximately 200 nM.

Conclusions This trial is the first to demonstrate activity of SAGE-547 in SRSE. A favorable safety profile and predictable exposures supports further development of SAGE-547 in this indication.

Authors/Disclosures
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file
	No disclosure on file

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