Overview

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome

Status:
Not yet recruiting
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Ionis Pharmaceuticals, Inc.