An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

NCT04740476 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: STK-001-DS-501
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 17

Brief Summary

Stoke Therapeutics is evaluating the long-term safety \& tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

Conditions

Dravet Syndrome

Keywords

Pediatric epilepsy; Epileptic Encephalopathies; Refractory Myoclonic Epilepsy; Severe Myoclonic Epilepsy in Infancy

Outcome Measures

Primary Outcomes (1)

• Safety of multiple doses of zorevunersen

  Safety variables for analysis include the incidence, type, severity, and seriousness of AEs, and changes in vital signs, ECG, laboratory, immunogenicity, physical examination, and outcomes on the cerebellar function clinical screening battery.

  Screening (Day -1) until 6 months after multiple drug dosing

Secondary Outcomes (5)

• Pharmacokinetic (PK) Parameters

  Dosing (Day 1) until 6 months after multiple drug dosing

• Exposure of zorevunersen in Cerebrospinal Fluid (CSF)

  Dosing (Day 1) and every 4 months until last study drug dosing day

• Measurement of Seizure Frequency

  Screening (Day -1) until 6 months after multiple drug dosing

• Change in overall clinical status

  Screening (Day -1) until 6 months after multiple drug dosing

• Change in Quality of Life

  Screening (Day -1) until 6 months after multiple drug dosing

Study Arms (1)

zorevunersen (STK-001) multiple dose levels

EXPERIMENTAL

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.

Drug: zorevunersen (STK-001)

Interventions

zorevunersen (STK-001) DRUG

zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.

zorevunersen (STK-001) multiple dose levels

Eligibility Criteria

• Age: 30 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Completed dosing with zorevunersen and the End of Study Visit in Study STK-001-DS-101 or Study STK-001-DS-102, with an acceptable safety profile per Investigator judgment.
• 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.

You may not qualify if:

• Met any withdrawal criteria from Study STK-001-DS-101 or STK-001-DS-102.
• 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.

Sponsors & Collaborators

• Stoke Therapeutics, Inc: lead

Study Sites (17)

University of California San Francisco Medical Center

San Francisco, California, 94158, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Location

Florida Hospital for Children

Orlando, Florida, 32803, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

University of Iowa Children's Hospital

Iowa City, Iowa, 52242, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Michigan Medicine

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

NYU Comprehensive Epilepsy Center

New York, New York, 10016, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

UT LeBonheur Pediatric Specialists, Inc.

Memphis, Tennessee, 38103, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

MultiCare Health System Institute for Research and Innovation

Tacoma, Washington, 98405, United States

Location

Related Publications (1)

• Laux L, Sullivan J, Perry MS, Brunklaus A, Desurkar A, Schreiber JM, Roberts CM, Knupp KG, Wheless JW, Wirrell EC, Ventola P, Wang F, Meena, Lynch J, Parkerson KA, Ticho B, Cross JH; MONARCH, ADMIRAL, SWALLOWTAIL, and LONGWING Study Groups. Zorevunersen in Children and Adolescents with Dravet Syndrome. N Engl J Med. 2026 Mar 5;394(10):969-982. doi: 10.1056/NEJMoa2506295.

  PMID: 41780062 DERIVED

MeSH Terms

Conditions

Epilepsies, Myoclonic

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Study Officials

• Ann Dandurand, MD

  Medical Director

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2021
• First Posted: February 5, 2021
• Study Start: January 20, 2021
• Primary Completion (Estimated): March 31, 2029
• Study Completion (Estimated): March 31, 2029
• Last Updated: February 4, 2026

Record last verified: 2026-02

Locations

US (17)