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Clinical Trials in the USA / NCT04740476
Active, not recruiting Phase 2

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

NCT04740476 · tracked via the Priya Life Science USA tracker
Sponsor
Stoke Therapeutics, Inc
Phase
Phase 2
Started
2021-01-20
Last updated
2026-02-04

Condition(s) studied

Dravet Syndrome

Investigational drug(s) / intervention(s)

zorevunersen (STK-001)

zorevunersen (STK-001): zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.

Study 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.

Eligibility

Sex
ALL
Min age
30 Months
Max age
Healthy volunteers
No
Inclusion Criteria: * 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. Exclusion Criteria: * 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.

Primary outcome measure(s)

Trial sites (17)

FacilityCityRegionStatus
University of California San Francisco Medical Center San Francisco California
Children's Hospital Colorado Aurora Colorado
Children's National Medical Center Washington D.C. District of Columbia
Nicklaus Children's Hospital Miami Florida
Florida Hospital for Children Orlando Florida
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois
University of Iowa Children's Hospital Iowa City Iowa
Massachusetts General Hospital Boston Massachusetts
Michigan Medicine Ann Arbor Michigan
Mayo Clinic Rochester Minnesota
NYU Comprehensive Epilepsy Center New York New York
Oregon Health & Science University Portland Oregon
Children's Hospital of Philadelphia Philadelphia Pennsylvania
UT LeBonheur Pediatric Specialists, Inc. Memphis Tennessee
Cook Children's Medical Center Fort Worth Texas
Seattle Children's Hospital Seattle Washington
MultiCare Health System Institute for Research and Innovation Tacoma Washington

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 NCT04740476 on ClinicalTrials.gov ↗ ← All trials in the USA