Current Research Studies
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 [Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome] ION337-CS1
IONIS 337
What is the goal of the study?
This is an Open Label multi-center Phase 1-2 study that consists of two parts. Part 1 is single ascending dose and part 2 is multiple ascending dose. It is expected up to 15 participants will be enrolled over 2 cohorts 2 to 12 years of age. The primary objective is to evaluate the safety and tolerability of ION337. Secondary objectives are to evaluate the effects of ION337 on seizure frequency and to characterize the pharmacokinetics of ION337 in the CSF and Plasma. Part 1 is expected to be 26 weeks [+5-7 weeks Screening Period]. Part 2 is expected to 104 weeks [+ 30 weeks Safety Follow-Up].
Who can participate in the study?
Please contact the study team listed below to learn more.