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

Launched by STOKE THERAPEUTICS, INC · Mar 6, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called zorevunersen for children and teenagers with Dravet syndrome, a severe form of epilepsy. The goal is to see how effective, safe, and well-tolerated this medication is for managing seizures related to this condition. The trial is currently not recruiting participants, but it aims to include patients aged 2 to 17 who have a confirmed diagnosis of Dravet syndrome and have experienced certain types of seizures. To qualify, participants must also have had a genetic test showing a specific change in the SCN1A gene and have tried at least two other treatments for their seizures without enough success.

If your child is eligible and chooses to participate, they will be closely monitored throughout the study. This means their health and any changes in their seizures will be regularly checked. The study will help researchers understand if zorevunersen can be a better option for managing Dravet syndrome. It’s important to note that some children may not be able to participate if they are currently on certain medications or have had new seizure types recently. This trial is an important step in finding new treatments for this challenging condition.

Gender

ALL

Eligibility 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 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.