Trial Profile

HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome

Status: Recruiting

Phase of Trial: Phase I/II

Latest Information Update: 07 Oct 2025

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At a glance

Drugs ION 582 (Primary)
Indications Angelman syndrome
Focus Adverse reactions; First in man
Acronyms HALOS
Sponsors Ionis Pharmaceuticals

Most Recent Events

11 Sep 2025 Number of treatment arms increased from 9 to 10 by the addition of Experimental: Part 1 MAD: Cohort F arm. Planned enrollment increased. Minimum inclusion age reduced from 2 years to 0 years, since a cohort of patients with Angelman Syndrome under the age of 2 years old will also be included.
11 Sep 2025 Planned number of patients changed from 55 to 70.
11 Sep 2025 Status changed to recruiting.

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