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)
Safety of multiple doses of zorevunersen — Screening (Day -1) until 6 months after multiple drug dosing 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.
Trial sites (17)
Facility
City
Region
Status
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
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.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time on our Cookie Policy page. See also our Privacy Policy.