The proposed EMPEROR Phase 3 Study is a global, double-blind, sham-controlled study. The study objectives will be to assess the efficacy, safety, and tolerability of zorevunersen in patients with Dravet syndrome.

Study site information is not yet available. More information will be provided on this page as we approach study initiation.

The MONARCH study was a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a genetic mutation in the SCN1A gene. The primary objectives for the study were to assess the safety and tolerability of zorevunersen (STK-001), as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective was to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency.

Following completion of MONARCH, patients who met study entry criteria were eligible to continue treatment in SWALLOWTAIL, an open-label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of zorevunersen. The study also evaluated the long-term effects of zorevunersen on convulsive seizure frequency and on behavior, cognition and overall quality of life.

The ADMIRAL study was a Phase 1/2a open-label study of children and adolescents ages 2 to <18 who have an established diagnosis of Dravet syndrome and have evidence of a genetic mutation in the SCN1A gene. The primary objectives for the study were to assess the safety and tolerability of multiple doses of zorevunersen (STK-001), as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective was to assess the effect of multiple doses of zorevunersen as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency. Overall clinical status and quality of life were secondary endpoints of ADMIRAL.

Following completion of ADMIRAL, patients who met study entry criteria were eligible to continue treatment in LONGWING, an open-label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of zorevunersen. The study also evaluated the long-term effects of zorevunersen on convulsive seizure frequency and on behavior, cognition and overall quality of life.

BUTTERFLY was an observational study of Dravet syndrome patients initiated by Stoke. This non-interventional two-year study, conducted at approximately 20 sites in the United States, observed children and adolescents ages 2 to 18 years with mutations in the SCN1A gene.

FALCON is a multicenter, prospective natural history study of people ages 8 to 60 who have an established clinical diagnosis of ADOA that is caused by a heterozygous OPA1 gene variant. No investigational medications or other treatments will be provided. The study enrolled patients across 10 sites in the U.S., U.K., Italy and Denmark.