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Zorevunersen treatment for children with Dravet syndrome

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

Phase 3 Interventional Stoke Therapeutics, Inc · NCT06872125

This trial will test whether zorevunersen can reduce major motor seizures and improve behavior and quality of life in children aged 2–17 with Dravet syndrome.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment170 (estimated)
Ages2 Years to 17 Years
SexAll
SponsorStoke Therapeutics, Inc Industry-sponsored
Locations61 sites (Phoenix, Arizona and 60 other locations)
Trial IDNCT06872125 on ClinicalTrials.gov

What this trial studies

Zorevunersen is an antisense oligonucleotide designed to increase productive SCN1A mRNA and restore Nav1.1 protein levels from the nonmutant SCN1A gene copy. The Phase 3 trial is randomized, double-blind, sham-controlled and conducted at multiple centers with two treatment periods; primary and secondary endpoints are assessed after each period. The primary outcome is change from baseline in major motor seizure frequency, and secondary outcomes include behavior, cognition, clinical status, and health-related quality of life. The goal is to determine whether RNA modulation by zorevunersen can lead to disease-modifying benefits in pediatric Dravet patients.

Who should consider this trial

Good fit: Ideal candidates are children aged 2 to under 18 with a clinical diagnosis of Dravet syndrome, a pathogenic/likely pathogenic SCN1A variant or variant of uncertain significance, and the required baseline count of major motor seizures.

Not a fit: Patients without an identified SCN1A variant, adults, or those whose seizures are caused by other known etiologies are unlikely to be eligible or to benefit from this targeted ASO approach.

Why it matters

Potential benefit: If successful, zorevunersen could meaningfully reduce disabling seizures and improve daily functioning and quality of life for children with Dravet syndrome.

How similar studies have performed: Antisense oligonucleotide approaches for neurological genetic disorders have shown promise in early-phase trials, and earlier-stage data for zorevunersen/STK-001 suggested signals, but definitive phase 3 efficacy has not been established.

Eligibility criteria

Show full inclusion / exclusion criteria 
Key Inclusion Criteria:

1. Patients must be ≥2 and \<18 years of age.
2. Patients must have a clinical diagnosis of DS confirmed by the Epilepsy Study Consortium, Inc. (ESCI) and as defined by:

   Onset, prior to 12 months (inclusive, \<13 months), of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures. No other known etiology causing clinical DS manifestations..
3. Patient must have a documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the sodium voltage-gated channel type 1 alpha subunit (SCN1A) gene. Patients who have SCN1A testing results of Negative (no variants identified) cannot be randomized.
4. Patient must experience the required number of major motor seizures during the 6-week Observation Period. Major motor seizure types included are Seizure types included in counts are Hemiclonic, Focal with Motor Signs, Focal to Bilateral Tonic-Clonic, Generalized Tonic-Clonic, Tonic, Tonic/Atonic (Drop Attacks with fall or risk of fall), and Bilateral Clonic.
5. Patient must have used at least 2 prior interventions for seizures. These can include anti-seizure medications (ASMs), ketogenic diet and/or vagus nerve stimulation (VNS) with either lack of adequate seizure control or discontinued due to an AE(s). These interventions can be ongoing therapies.
6. Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis (i.e., not as needed \[PRN\]) for any indication will be considered an ASM.
7. Patients' maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS), as well as any marijuana- or cannabinoid-based products, must have been stable (unless adjusted for weight) during the Baseline Period.

Key Exclusion Criteria:

1. Patient has documented variant in the SCN1A gene associated with gain-of-function
2. Patient is currently treated with a maintenance ASM acting primarily as a sodium channel blocker, including but not limited to phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate, given the mechanism of action of zorevunersen.
3. Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS.
4. Patient has emergence of a new seizure type or reemergence of a past seizure type (seizure types that last occurred more than 12 months before Screening Visit A) during the Baseline Period, or has more than 1 hospitalization for seizures during the Baseline Period.

Where this trial is running

Phoenix, Arizona and 60 other locations

+11 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Dravet SyndromePediatric epilepsyEpileptic EncephalopathiesRefractory Myoclonic EpilepsySevere Myoclonic Epilepsy in InfancySTK-001
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.