An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

Phase 1/2 Completed 62 enrolled Stoke Therapeutics, Inc

Non-randomizedSequentialOpen-labelTreatment

Summary

Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency, overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

Timeline

Start: 2020-06-29
Primary completion: 2023-11-14
Completion: 2023-11-14

Outcome

Met primary endpoint

release “Zorevunersen has been generally well tolerated across the Phase 1/2a and OLE studies.” stoketherapeutics.com ↗

Publications

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.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	zorevunersen	Antisense oligonucleotide	10 mg	Intrathecal
Subject	zorevunersen	Antisense oligonucleotide	20 mg	Intrathecal
Subject	zorevunersen	Antisense oligonucleotide	30 mg	Intrathecal
Subject	zorevunersen	Antisense oligonucleotide	45 mg	Intrathecal
Subject	zorevunersen	Antisense oligonucleotide	70 mg	Intrathecal