clinical trial · NCT07531745
ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
Ionis Pharmaceuticals, Inc.·phase2·recruiting·n = 32
Dravet SyndromeION337
brief summary
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
started
Apr 1, 2026
primary completion
Dec 1, 2030
completion
Dec 1, 2030
last updated
Apr 21, 2026
detailed description
This is an open-label study of ION337 in people with DS between the ages of 2 and 12 years old (inclusive). The study consists of 2 parts: Part 1) 6-month single ascending dose (SAD) and Part 2) 24-month multiple ascending dose (MAD), followed by a 7-month safety follow up.
official title
Phase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol