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Clinical Trials in the USA / NCT06872125
Recruiting Phase 3

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

NCT06872125 · tracked via the Priya Life Science USA tracker
Sponsor
Stoke Therapeutics, Inc
Phase
Phase 3
Started
2025-06-04
Last updated
2026-08-24

Condition(s) studied

Dravet Syndrome

Investigational drug(s) / intervention(s)

zorevunersenSham Comparator

zorevunersen: Treatment Period 1: 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. Treatment Period 2: Group assigned to zorevunersen in Treatment Period 1 will receive 45 mg of zorevunersen on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84).

Sham Comparator: Treatment Period 1: Sham group will not have drug administered. Sham group will have a procedure intended to mimic the drug administration. Treatment Period 2: Group assigned to sham in Treatment Period 1 will receive 70 mg of zorevunersen on Day 393 (Week 56) and on Day 477 (Week 68), and 45 mg of zorevunersen Day 589 (Week 84).

Study summary

The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.

Eligibility

Sex
ALL
Min age
2 Years
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (61)

FacilityCityRegionStatus
Phoenix Children's Hospital Phoenix Arizona Active Not Recruiting
Arkansas Children's Hospital Little Rock Arkansas Active Not Recruiting
Cedars Sinai Medical Center Los Angeles California Active Not Recruiting
Children's Hospital of Orange County Orange California Active Not Recruiting
USCF Medical Center San Francisco California Active Not Recruiting
Children's Hospital Colorado Aurora Colorado Active Not Recruiting
Children's National Medical Center Washington D.C. District of Columbia Active Not Recruiting
Nemours Children's Health Jacksonville Florida Active Not Recruiting
Nicklaus Children's Hospital Miami Florida Active Not Recruiting
Advent Health Neuroscience Research Institute Orlando Florida Active Not Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Active Not Recruiting
Massachusetts General Hospital Boston Massachusetts Active Not Recruiting
Boston Children's Hospital Boston Massachusetts Active Not Recruiting
CS Mott Children's Hospital Ann Arbor Michigan Active Not Recruiting
Mayo Clinic Rochester Minnesota Active Not Recruiting
NYU Langone Health New York New York Active Not Recruiting
Weill Cornell Medicine New York New York Active Not Recruiting
University of Rochester Medical Center Rochester New York Active Not Recruiting
University of North Carolina at Chapel Hill Chapel Hill North Carolina Active Not Recruiting
Duke University Health System Durham North Carolina Active Not Recruiting
Cleveland Clinic Cleveland Ohio Active Not Recruiting
Nationwide Children's Hospital Columbus Ohio Active Not Recruiting
Oregon Health & Science University (OHSU) Portland Oregon Active Not Recruiting
LeBonheur Children's Hospital Memphis Tennessee Active Not Recruiting
Cook Children's Medical Center Fort Worth Texas Active Not Recruiting
Texas Children's Hospital Houston Texas Active Not Recruiting
University of Utah Primary Children's Hospital Salt Lake City Utah Active Not Recruiting
UVA Health Charlottesville Virginia Active Not Recruiting
Peking University First Hospital Beijing China Recruiting
The Second Affiliated Hospital of Guangzhou Medical university Guangzhou China Recruiting
Wuhan Children's Hospital Wuhan China Recruiting
Hôpital de la Timone Marseille France Active Not Recruiting
Hôpital Necker - Enfants Malades Paris France Active Not Recruiting
Hôpital Robert Debré - Paris Paris France Active Not Recruiting
Charité - Campus Virchow-Klinikum Berlin Germany Active Not Recruiting
Universitaetsklinikum Bonn AoeR Bonn Germany Active Not Recruiting
Universitaetsklinikum Frankfurt Goethe-Universitaet Frankfurt Germany Active Not Recruiting
Universitaetsklinikum Freiburg Friedberg Germany Active Not Recruiting
Universitaetsklinikum Heidelberg Heidelberg Germany Active Not Recruiting
Integriertes Sozialpaediatrisches Zentrum München Germany Active Not Recruiting

+ 21 more sites — see the full list on the official registry below.

More Stoke Therapeutics, Inc trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06872125 on ClinicalTrials.gov ↗ ← All trials in the USA