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

Running, not enrolling · Phase 2

Conditions studied: Dravet Syndrome

In brief

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.

Key facts

Study ID

NCT04740476

Run by

Stoke Therapeutics, Inc

People needed

60

Starts

2021-01-20

Expected to finish

2029-03-31

Last updated by the study team

2026-02-04

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

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.

Where it is running

• 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

Full record on ClinicalTrials.gov

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