An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome
NCT ID: NCT04740476
Last Updated: 2026-02-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
60 participants
INTERVENTIONAL
2021-01-20
2029-03-31
Brief Summary
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Detailed Description
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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. Stoke has generated preclinical data demonstrating proof-of-mechanism for zorevunersen.
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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zorevunersen (STK-001) multiple dose levels
Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. Patients will receive IT administration of study drug zorevunersen at the dose level they received while participating in Study STK-001-DS-101 or STK-001-DS-102, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an zorevunersen Phase 1/2 study, and doses above 45 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56.
zorevunersen (STK-001)
zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.
Interventions
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zorevunersen (STK-001)
zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.
Eligibility Criteria
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Inclusion Criteria
* Had satisfactory compliance with study visits and procedures in Study STK-001-DS-101 or Study STK-001-DS-102 per Investigator and Sponsor judgment.
* Completed Study STK-001-DS-101 or STK-001-DS-102 within 4 weeks of the start of their participation in Study STK-001-DS-501 unless approved by sponsor.
Exclusion Criteria
* Currently treated with an antiepileptic drug (AED) acting primarily as a sodium channel blocker, as maintenance therapy, including phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide or rufinamide (with the exception of cenobamate, which is permitted).
* Clinically significant unstable medical conditions other than epilepsy.
* Clinically relevant symptoms or a clinically significant illness (in the judgment of the Investigator) at Screening or prior to dosing on Day 1, other than epilepsy.
* Spinal deformity or other condition that may alter the free flow of CSF or has an implanted CSF drainage shunt.
* Treated (or is being treated) with an investigational product (other than zorevunersen) since participating in Study STK-001-DS-101 or STK-001-DS-102.
* Participating in an observational study, they are excluded unless approved by the Sponsor.
30 Months
ALL
No
Sponsors
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Stoke Therapeutics, Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Ann Dandurand, MD
Role: STUDY_DIRECTOR
Medical Director
Locations
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University of California San Francisco Medical Center
San Francisco, California, United States
Children's Hospital Colorado
Aurora, Colorado, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Nicklaus Children's Hospital
Miami, Florida, United States
Florida Hospital for Children
Orlando, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Iowa Children's Hospital
Iowa City, Iowa, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Michigan Medicine
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
NYU Comprehensive Epilepsy Center
New York, New York, United States
Oregon Health & Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
UT LeBonheur Pediatric Specialists, Inc.
Memphis, Tennessee, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
MultiCare Health System Institute for Research and Innovation
Tacoma, Washington, United States
Countries
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Other Identifiers
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STK-001-DS-501
Identifier Type: -
Identifier Source: org_study_id
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