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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

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients with Dravet Syndrome

Ann M Dandurand

Stoke Therapeutics, Inc

45 Wiggins Avenue, Bedford, MA 01730

clinicaltrials@stoketherapeutics.com

jRCT Inquiry Receipt Center

IQVIA Services Japan G.K.

Keikyu Dai-1 Building 4-10-18 Takanawa, Minato-ku, Tokyo

+81-3-6859-9500

mariko.nomura@iqvia.com

Recruiting

July. 01, 2025

Interventional

randomized controlled trial

double blind

no treatment control/standard of care control

parallel assignment

treatment purpose

-Patients must be >=2 and <18 years of age.
-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 of age, of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures.No other known etiology causing clinical DS manifestations.
-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.
-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.
-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.
-Patient must be taking at least one ASM. Benzodiazepines or ASMs used on a standing basis for any indication will be considered an ASM.
-Patients' maintenance ASMs and interventions for seizures (i.e., ketogenic diet or VNS) must have been stable (unless adjusted for weight) during the Baseline Period.

-Patient has documented variant in the SCN1A gene associated with gain-of-function
-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.
-Patient is currently treated with neuromodulation techniques (e.g., responsive neurostimulation, deep brain stimulation, or transcranial magnetic stimulation), with the exception of VNS.
-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.

Both

Dravet Syndrome

Zorevunersen group will receive study drug by intrathecal (IT) administration on Day 1 (after the 8-week Baseline Period), Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40) at a dose level of 70 mg on Day 1 and Day 57, and 45 mg on Day 169 and Day 281.
Sham group will not have drug administered. Sham group will have a lumbar puncture with CSF withdrawal.

Measurement of Seizure Reduction at Week 28

Measurement of Seizure Reduction at Week 52
Multi-component Vineland-3 Outcome Score at Week 52 (Measurement of change from baseline)
Vineland-3 Subdomain Score at Week 52 (Measurement of change from baseline)

+81-92-682-7000

June. 03, 2025

No

United States of America/United Kingdom