Clinical Trial

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

Recruiting Phase 1/2
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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.
Protocol Amendment History 21 amendments
This ClinicalTrials.gov record has been amended 21 times since 2021-11-09; most recent amendment 2025-11-12.
Status change: Active, Not Recruiting → Recruiting 2025-09-04
Status change: Recruiting → Active, Not Recruiting 2023-12-07
Status change: Not Yet Recruiting → Recruiting 2021-12-22
Trial Details
NCT Number NCT05127226
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Angelman Syndrome
Enrollment 70 participants
Start Date 2021-12-22
Primary Completion 2029-03 (estimated)
Study Completion 2029-03 (estimated)
Updated on ClinicalTrials.gov 2025-11-14