A Phase III, multicenter, randomized, double-blind, sham-controlled, parallel-group clinical trial to evaluate the efficacy, safety, and tolerability of zorevunersen (STK-001) in patients with Dravet syndrome.

THE MAIN OBJECTIVES OF THIS TRIAL ARE: 1) TO EVALUATE THE EFFECT OF THE STUDY DRUG ON THE FREQUENCY OF SEIZURES IN CHILDREN WITH DRAVET SYNDROME; 2) TO DETERMINE WHETHER THE STUDY DRUG AFFECTS THE IMPAIRMENTS CAUSED BY DRAVET SYNDROME AND HOW IT DOES SO, INCLUDING BEHAVIORAL AND DEVELOPMENTAL DELAYS AND LANGUAGE AND SPEECH DIFFICULTIES; 3) TO ASSESS HOW LONG THE STUDY DRUG CAN REDUCE THE FREQUENCY OF SEIZURES IN CHILDREN AND ADOLESCENTS WITH DRAVET SYNDROME (KNOWN AS “DURABILITY”); 4) TO STUDY THE SAFETY OF THE STUDY DRUG IN CHILDREN AND ADOLESCENTS AND HOW WELL A CHILD TOLERATES IT (KNOWN AS “TOLERABILITY”): THIS INVOLVES MONITORING SIDE EFFECTS, VITAL SIGNS, OVERALL HEALTH, MOVEMENT AND BALANCE, AND THE PARTICIPANT’S HEART RATE, AMONG OTHER SAFETY CHECKS, AS WELL AS HOW THE PARTICIPANT’S IMMUNE SYSTEM (THE BODY’S DEFENSE SYSTEM) RESPONDS TO THE STUDY DRUG; 5) STUDYING HOW THE BODY ABSORBS AND METABOLIZES THE INVESTIGATIONAL DRUG (KNOWN AS “PHARMACOKINETICS” OR “PK”).