Study of Safety and Tolerability of ION337 Injection in Children Ages 2 to 12 With Dravet Syndrome

What this Trial Is About

Researchers are evaluating the safety and tolerability of ION337 in children aged 2 to 12 years with Dravet syndrome, a severe form of epilepsy. This open-label study is designed to assess how well participants tolerate this investigational drug and to monitor its effects over time. The study is led by Ionis Pharmaceuticals, Inc. and aims to gather detailed safety data in this pediatric population. Participants receive ION337 through an intrathecal bolus injection directly into the spinal fluid. The study has two parts Part 1 involves a 6-month period with a single ascending dose of ION337, where dosing levels increase sequentially. Participants who complete this phase may enter Part 2, which lasts 24 months and involves multiple ascending doses at the same dose level assigned in Part 1. After these treatment phases, there is a 7-month safety follow-up period to monitor participants. During the study, participants will be closely monitored for any treatment-emergent adverse events and changes in laboratory tests, vital signs, ECGs, physical and neurological exams, and suicidality assessments. Researchers will also measure ION337 concentrations in plasma and cerebrospinal fluid and track changes in seizure frequency. The total participation time can be up to 37 months, including treatment and follow-up, with regular study visits for assessments throughout the trial.

ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome