A Phase 3 interventional study of zorevunersen and Sham Comparator in Dravet Syndrome, sponsored by Stoke Therapeutics, Inc. Active, not recruiting at 61 sites in 8 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-20.
Sponsored by Stoke Therapeutics, Inc · Phase 3, Interventional, and Treatment
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Among 61 studies in Epilepsies, Myoclonic with a site in United States — 11 open to participants now, 17 under way, 4 of them phase 3. Epilepsies, Myoclonic · United States.
Among the 61 studies in Epilepsies, Myoclonic with a site in United States, this one is the 7th largest by planned enrollment of the 43 interventional ones that give a count, is one of 3 that started in 2025, is one of 20 in phase 3, reaches primary completion later than 7 of the 16 other live ones, and was the 56th registered.
92 studies on the registry are indexed under Epilepsies, Myoclonic; 21 are open to participants now.
Browse Epilepsies, Myoclonic studies →11 of the 61 studies in Epilepsies, Myoclonic with a site in United States are open now. Recruiting first, the newest first:
Scripps Translational Science Institute · Sites in United States
Ages 2–60
Ionis Pharmaceuticals, Inc. · Sites in United States, Canada
Ages 2–12
The University of Texas Health Science Center, Houston · Sites in United States
Ages 16+
University of Colorado, Denver · Sites in United States
Ages 12 mo–24 mo
Encoded Therapeutics · Sites in United States, Australia, United Kingdom
Ages 6 mo–17 yrs
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 14 Epilepsies, Myoclonic studies recruiting now, 3 (21%) set no upper age limit and 10 (71%) stop at 75 or younger; 11 take children under 18; 3 take healthy volunteers.
This study: 2 Years to 17 Years · all sexes · no healthy volunteers.
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61 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 9 · Phase 2 15 · Phase 3 20 · Phase 4 3
First in 2000; this study’s year is marked. 3 started in 2025.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 170 is above the median of 31 across 56 interventional studies indexed under Epilepsies, Myoclonic.
Median enrollment 36 across the 43 interventional studies, 58 across the 11 observational. Of the 33 that completed or were terminated, 21 (64%) have results posted.
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41 of 61 have an industry lead sponsor. The 10 most frequent:
Stoke Therapeutics, Inc is the lead sponsor of 3 studies on the registry; none are open to participants now.
Stoke Therapeutics, Inc has 3 studies filed under Epilepsies, Myoclonic — fewer than ten, so there is no comparison to draw.
10 of the studies in Epilepsies, Myoclonic with a site in United States that are under way reach primary completion within a year, 4 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06660394Phase 3Longboard PharmaceuticalsA Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS) | 2026-12-31 |
| NCT06872125Phase 3Stoke Therapeutics, IncA Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome | 2027-03 |
| NCT04462770Phase 3EpygenixA Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome | 2027-04 |
| NCT06908226Phase 3Longboard PharmaceuticalsA Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE) | 2027-11-11 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
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Among the 61 studies in Epilepsies, Myoclonic with a site in United States, from their registry updates:
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The purpose of the study is to evaluate the efficacy, safety, and tolerability of zorevunersen in Patients with Dravet syndrome.
Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome. It is an antisense oligonucleotide (ASO) that is intended to increase the level of productive SCN1A messenger RNA (mRNA) and consequently increase the expression of the sodium channel Nav1.1 protein. This RNA-based approach is not gene therapy, but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA).
Zorevunersen is designed to upregulate Nav1.1 protein expression from the nonmutant (wild-type) copy of the SCN1A gene to restore physiological Nav1.1 levels. Nav1.1 levels are reduced in people with Dravet syndrome.
This is a global, multicenter, randomized, double-blind, sham-controlled, parallel group Phase 3 study to assess the efficacy, safety, and tolerability of zorevunersen in patients with Dravet syndrome. The study duration and endpoints are designed to evaluate the potential of zorevunersen for disease modification. The study consists of two parts, Treatment Period 1 and Treatment Period 2. The primary and secondary endpoints will be assessed at the conclusion of Treatment Period 1. These endpoints will be assessed again at the end of Treatment Period 2. The primary endpoint is the change from baseline in major motor seizure frequency. Secondary endpoints include the change in behavior and cognition, clinical status, and health-related quality of life in patients with Dravet syndrome.
Patients will have the opportunity to enroll in an open label extension study and receive zorevunersen if they meet eligibility criteria at the end of the study.
Key Inclusion Criteria:
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..
Key Exclusion Criteria:
Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham in Treatment Period 1 (approximately 52 weeks). Upon the completion of Treatment Period 1 all eligible patients, will enter Treatment Period 2 and receive zorevunersen, regardless of initial treatment assignment.
Drug: zorevunersen
Eligible patients will be randomly assigned in a 1:1 ratio to zorevunersen:sham
Other: Sham Comparator
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).
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).
Measurement of Seizure Change
Measured by daily seizure diary
Time frame: Week 28
Measurement of Seizure Change
Measured by daily seizure diary
Time frame: Week 52
Multi-component Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Outcome Score
Measurement of change from baseline for multi-component score. Scoring is non-parametric and based on rank between treatment and sham groups. Individual items are scored on a scale of 0, 1, or 2, indicating never, sometimes, or usually or often, or on a scale of 0 or 2, indicating no or yes, with higher scores indicating greater performance
Time frame: Week 52
Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Subdomain Score
Measurement of change from baseline for individual subdomains. Scoring is point-based on a scale that varies, depending on subdomain, Individual items are scored on a scale of 0, 1, or 2, indicating never, sometimes, or usually or often, or on a scale of 0 or 2, indicating no or yes, with higher scores indicating greater performance.
Time frame: Week 52
Started Jun 2025 (actual). Primary completion is estimated for Mar 2027, 21 months after the start.
Of 15 phase 3 interventional studies under Epilepsies, Myoclonic, half reached primary completion within 35 months of starting (the median), counting those still running; a quarter within 27 and three quarters within 72. This study's planned 21 months is below that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Aug 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Zorevunersen
Stoke Therapeutics, Inc