CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 62 enrolled
Drug / intervention
STK-001 - Single Ascending Doses +1 moredrug
Likely dose
STK-001 - Single Ascending Doses 10mgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04442295
NCT04442295Phase 2Completed
CompareCT.gov

An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single and Multiple Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents With Dravet Syndrome

Stoke Therapeutics, Inc·interventional·Posted Jun 22, 2020·Updated May 18, 2025

In Brief

A Phase 2 clinical trial evaluating STK-001 - Single Ascending Doses and STK-001 - Multiple Ascending Doses for Dravet Syndrome. Completed, enrolled 62 participants across 18 sites.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDravet Syndrome
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
202120222023202420252026
First PostedJun 22, 2020
Enrollment StartJun 29, 2020
Primary CompletionNov 14, 2023
TodayJul 2, 2026
Enrollment to primary: 3.4 yearsPosted 6.0 years ago

Interventions

STK-001 - Single Ascending Dosesdrug

Experimental : Single Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Four dose levels will be evaluated ( 10mg, 20mg,30mg, 45mg and 70mg ).

STK-001 - Multiple Ascending Dosesdrug

Experimental : Multiple Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Three dose levels will be evaluated ( 20mg,30mg and 45mg ).