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Clinical Trial 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.


Clinical Trial Description

This is a Phase 1-2a, open-label dose-escalation study of ION582 enrolling up to approximately 44 participants. Following a screening period of up to 4 weeks, eligible participants will receive intrathecal (IT) injections of ION582. Participants will be followed for up to 32 weeks after dosing. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05127226
Study type Interventional
Source Ionis Pharmaceuticals, Inc.
Contact Ionis Pharmaceuticals
Phone 800-679-4747
Email [email protected]
Status Not yet recruiting
Phase Phase 1/Phase 2
Start date November 2021
Completion date December 2023

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