Actively Recruiting
EMPEROR A Multicenter, Randomized, Double-blind, Sham-controlled, Parallel Group, Phase 3 Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen STK-001 in Patients With Dravet Syndrome
Led by Stoke Therapeutics, Inc · Updated on 2026-06-02
170
Participants Needed
61
Research Sites
82 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of zorevunersen, an investigational antisense oligonucleotide medicine, in children with Dravet syndrome, a rare epilepsy disorder caused by reduced Nav1.1 protein due to SCN1A gene mutations. This Phase 3, global, randomized, double-blind, sham-controlled study aims to assess the safety, tolerability, and potential to modify the disease by increasing Nav1.1 protein levels from the nonmutant gene copy. The study also measures changes in seizure frequency, behavior, cognition, clinical status, and quality of life. Participants are randomly assigned to one of two groups for Treatment Period 1, lasting about 52 weeks either receive zorevunersen or undergo a sham procedure without drug administration. Zorevunersen is given by intrathecal injection at specific days and doses during this period. After Treatment Period 1, all eligible patients enter Treatment Period 2, where everyone receives zorevunersen at defined intervals and doses. Patients may also join an open-label extension study if they meet criteria after the main study. Throughout the study, patients will be monitored regularly to assess seizure changes at weeks 28 and 52, as well as adaptive behavior using the Vineland-3 scale. Safety and tolerability are closely followed with standardized evaluations. The study lasts at least 84 weeks, including both treatment periods. Researchers aim to understand how zorevunersen may impact major motor seizures and overall functioning in children with Dravet syndrome.
CONDITIONS
Brief Title
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be 2 years of age or older and younger than 18 years.
- Clinical diagnosis of Dravet syndrome confirmed by the Epilepsy Study Consortium.
- Onset of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures before 13 months of age with no other known cause.
- Documented pathogenic, likely pathogenic, or uncertain significance variant in the SCN1A gene.
- Experience the required number of major motor seizures during the 6-week observation period.
- Have used at least two prior seizure interventions including anti-seizure medications, ketogenic diet, or vagus nerve stimulation.
- Currently taking at least one anti-seizure medication, including benzodiazepines used regularly.
- Maintenance therapies and interventions must have been stable during the baseline period unless adjusted for weight.
You will not qualify if you...
- Documented gain-of-function variant in the SCN1A gene.
- Currently treated with maintenance anti-seizure medications that primarily block sodium channels such as phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate.
- Currently treated with neuromodulation techniques except for vagus nerve stimulation.
- Emergence of new or reemergence of past seizure types during baseline period.
- More than one hospitalization for seizures during the baseline period.
Research Team
E
Emperor Information Center
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