Actively Recruiting
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
Led by Stoke Therapeutics, Inc · Updated on 2026-06-02
170
Participants Needed
61
Research Sites
82 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects of zorevunersen, an investigational antisense oligonucleotide medicine, in children with Dravet syndrome, a rare epilepsy disorder caused by reduced Nav1.1 protein due to SCN1A gene mutations. This Phase 3, global, randomized, double-blind, sham-controlled study aims to assess the safety, tolerability, and potential to modify the disease by increasing Nav1.1 protein levels from the nonmutant gene copy. The study also measures changes in seizure frequency, behavior, cognition, clinical status, and quality of life. Participants are randomly assigned to one of two groups for Treatment Period 1, lasting about 52 weeks: either receive zorevunersen or undergo a sham procedure without drug administration. Zorevunersen is given by intrathecal injection at specific days and doses during this period. After Treatment Period 1, all eligible patients enter Treatment Period 2, where everyone receives zorevunersen at defined intervals and doses. Patients may also join an open-label extension study if they meet criteria after the main study. Throughout the study, patients will be monitored regularly to assess seizure changes at weeks 28 and 52, as well as adaptive behavior using the Vineland-3 scale. Safety and tolerability are closely followed with standardized evaluations. The study lasts at least 84 weeks, including both treatment periods. Researchers aim to understand how zorevunersen may impact major motor seizures and overall functioning in children with Dravet syndrome.
CONDITIONS
Brief Title
A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be 2 years of age or older and younger than 18 years.
- Clinical diagnosis of Dravet syndrome confirmed by the Epilepsy Study Consortium.
- Onset of recurrent focal with motor signs, hemiclonic, or generalized tonic-clonic seizures before 13 months of age with no other known cause.
- Documented pathogenic, likely pathogenic, or uncertain significance variant in the SCN1A gene.
- Experience the required number of major motor seizures during the 6-week observation period.
- Have used at least two prior seizure interventions including anti-seizure medications, ketogenic diet, or vagus nerve stimulation.
- Currently taking at least one anti-seizure medication, including benzodiazepines used regularly.
- Maintenance therapies and interventions must have been stable during the baseline period unless adjusted for weight.
You will not qualify if you...
- Documented gain-of-function variant in the SCN1A gene.
- Currently treated with maintenance anti-seizure medications that primarily block sodium channels such as phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, rufinamide, or cenobamate.
- Currently treated with neuromodulation techniques except for vagus nerve stimulation.
- Emergence of new or reemergence of past seizure types during baseline period.
- More than one hospitalization for seizures during the baseline period.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 52 weeks
Participants receive zorevunersen or sham procedures during this initial treatment period to evaluate efficacy and safety.
4 treatment visits on Day 1, Day 57 (Week 8), Day 169 (Week 24), and Day 281 (Week 40)
Duration - Approximately 36 weeks
All participants receive zorevunersen during this open-label extension period following the initial blinded phase.
3 treatment visits on Day 393 (Week 56), Day 477 (Week 68), and Day 589 (Week 84)
Trial Site Locations
Total: 61 locations
1
Phoenix Children's Hospital
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
Actively Recruiting
3
Cedars Sinai Medical Center
Los Angeles, California, United States, 90048
Actively Recruiting
4
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
5
USCF Medical Center
San Francisco, California, United States, 94158
Actively Recruiting
6
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
7
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
8
Nemours Children's Health
Jacksonville, Florida, United States, 32207
Actively Recruiting
9
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
10
Advent Health Neuroscience Research Institute
Orlando, Florida, United States, 32804
Actively Recruiting
11
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
12
University of Iowa Hospital and Clinics
Iowa City, Iowa, United States, 52242
Not Yet Recruiting
13
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
14
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
15
CS Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
16
Mayo Clinic
Rochester, Minnesota, United States, 55905
Actively Recruiting
17
Washington University in St. Louis School of Medicine
St Louis, Missouri, United States, 63110
Not Yet Recruiting
18
NYU Langone Health
New York, New York, United States, 10016
Actively Recruiting
19
Weill Cornell Medicine
New York, New York, United States, 10021
Actively Recruiting
20
University of Rochester Medical Center
Rochester, New York, United States, 14642
Actively Recruiting
21
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27514
Actively Recruiting
22
Duke University Health System
Durham, North Carolina, United States, 27705
Actively Recruiting
23
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Not Yet Recruiting
24
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
25
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
26
Oregon Health & Science University (OHSU)
Portland, Oregon, United States, 97239
Actively Recruiting
27
LeBonheur Children's Hospital
Memphis, Tennessee, United States, 38103
Actively Recruiting
28
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
29
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
30
University of Utah Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
31
UVA Health
Charlottesville, Virginia, United States, 22903
Actively Recruiting
32
Hôpital de la Timone
Marseille, France
Actively Recruiting
33
Hôpital Necker - Enfants Malades
Paris, France
Actively Recruiting
34
Hôpital Robert Debré - Paris
Paris, France
Actively Recruiting
35
Charité - Campus Virchow-Klinikum
Berlin, Germany
Actively Recruiting
36
Universitaetsklinikum Bonn AoeR
Bonn, Germany
Actively Recruiting
37
Universitaetsklinikum Frankfurt Goethe-Universitaet
Frankfurt, Germany
Actively Recruiting
38
Universitaetsklinikum Freiburg
Friedberg, Germany
Actively Recruiting
39
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Actively Recruiting
40
Integriertes Sozialpaediatrisches Zentrum
München, Germany
Actively Recruiting
41
Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer
Florence, Italy
Actively Recruiting
42
Istituto Giannina Gaslini-Ospedale Pediatrico IRCCS
Genova, Italy
Actively Recruiting
43
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, Italy
Actively Recruiting
44
Ospedale Pediatrico Bambino Gesù
Roma, Italy
Actively Recruiting
45
Fukuoka Children's Hospital
Fukuoka, Japan
Actively Recruiting
46
Hokkaido University Hospital
Hokkaido, Japan
Actively Recruiting
47
Kyoto University Hospital
Kyoto, Japan
Actively Recruiting
48
Nagoya University Hospital
Nagoya, Japan
Actively Recruiting
49
National Hospital Organization Nishi Niigata Central Hospital
Niigata, Japan
Actively Recruiting
50
Okayama University Hospital
Okayama, Japan
Actively Recruiting
51
Osaka City General Hospital
Osaka, Japan
Actively Recruiting
52
Jichi Medical University Hospital
Shimotsuke, Japan
Actively Recruiting
53
NHO Shizuoka
Shizuoka, Japan
Actively Recruiting
54
National Center of Neurology and Psychiatry
Tokyo, Japan
Actively Recruiting
55
Yokohama City University Medical Center
Yokohama, Japan
Actively Recruiting
56
Hospital Blua Sanitas Valdebebas
Madrid, Spain
Actively Recruiting
57
Hospital Ruber Internacional
Madrid, Spain
Actively Recruiting
58
Clinica Universidad de Navarra
Pamplona, Spain
Actively Recruiting
59
Royal Hospital for Children
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
60
Great Ormond Street Hospital for Children
London, United Kingdom, WC1N 3JH
Actively Recruiting
61
Sheffield Children's Hospital
Sheffield, United Kingdom, S10 2TH
Actively Recruiting
Research Team
E
Emperor Information Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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