An Open-Label Study to Investigate the Safety and Pharmacokinetics of Single Ascending Doses of Antisense Oligonucleotide STK-001 in Children and Adolescents with Dravet Syndrome
What is the goal of the study?
This is an open-label, Phase I/IIA 2-part single ascending dose study to evaluate the safety and tolerability of STK-001 in patients with Dravet Syndrome. Secondary endpoints include accessing effectiveness as an adjunct antiepileptic treatment and evaluation of overall clinical status and quality of life of Dravet patients. The study will be two parts. Each part (A and B) consists of a Screening Visit followed by a 4-week Observation Period, a Baseline Visit, single dose study drug administration at Day 1, and a 6-month Follow-Up Period, which consists of 5 study site visits and 3 telephone calls. Part A of the study will have 2 cohorts with ascending doses. After dosing of Cohort 2 the FDA will review preclinical data and provide approval for the study to continue to Part B of the study. Part 2 of the study will also include 2 cohorts (Cohort 3 and 4) with ascending doses of 40 and 80 mg (or adjusted dose based on Safety Monitoring Committee). Additional cohorts may be added to Part B. The study drug, STK-001 is administered by intrathecal administration. The study will last about 7 months and may enroll up to 48 patients.
Who can participate in the study?
Please contact the study team listed below to learn more.