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HALOS: ION582 for Angelman Syndrome

This study, called HALOS, is testing a drug called ION582 for people with Angelman syndrome. Researchers want to see how safe ION582 is and how well people tolerate different doses when it's given by IT injection (into the fluid around the spinal cord). You may be able to join if you are between 0 and 50 years old and have a confirmed diagnosis of Angelman syndrome (due to a UBE3A deletion or mutation). The main goal is to track any side effects and changes in lab tests. This study plans to enroll about 70 participants. The current status of the study is unclear, but some sites are recruiting individuals under 2 years old.

Study design
This is an open-label study, meaning you and your doctors will know which treatment you are receiving. It involves about 70 participants and has three parts, with different doses of ION582 tested.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Safety and tolerability will be measured for up to 45 weeks in Part 1, and up to 81 weeks in Part 2. There is also a follow-up period after each part of the study.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05127226

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

Active, Not Recruiting
PHASE1Ages 0–50InterventionalTreatment
Ionis Pharmaceuticals, Inc.
~71 participants
Updated 2026-08-20 on ClinicalTrials.gov
What's tested:ION582

At a glance

Recruiting sites
0 of 11 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters).
Measured over Part 1: Up to Week 45; Part 2: Up to Week 81

Conditions

Angelman Syndrome

Explore related trials

Angelman SyndromeUbiquitin-Protein Ligase E3A

Where it's being run

11 sites across 11 states
California1
Colorado1
Illinois1
Massachusetts1
North Carolina1
Texas1
Australia1
France1

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

What this trial measures

  • To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters).Part 1: Up to Week 45; Part 2: Up to Week 81

    The safety and tolerability of ION582 will be assessed by determining the incidence, severity, and dose relationship of adverse effects and changes in the laboratory parameters by dose.