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Active, not recruiting Phase 1Phase 2 Interventional

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

ClinicalTrials.gov ID: NCT05127226

Public ClinicalTrials.gov record NCT05127226. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 11, 2026, 4:37 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

The purpose of this study is to evaluate the safety and tolerability of ascending doses of ION582 administered intrathecally in participants with Angelman syndrome.

Study identification

NCT ID
NCT05127226
Recruitment status
Active, not recruiting
Study type
Interventional
Phase
Phase 1, Phase 2
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Enrollment
71 participants

Conditions and interventions

Interventions

Drug

Eligibility (public fields only)

Age range
0 Years to 50 Years
Sex
All
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Dec 21, 2021
Primary completion
Feb 28, 2029
Completion
Feb 28, 2029
Last update posted
Aug 19, 2026

2021 – 2029

United States locations

U.S. sites
6
U.S. states
6
U.S. cities
6
Facility City State ZIP Site status
Rady Children's Hospital San Diego California 92123
Colorado Children's Hospital Research Institute Aurora Colorado 80045
Rush University Medical Center Chicago Illinois 60612
Boston Children's Hospital Boston Massachusetts 02215
University of North Carolina at Chapel Hill School of Medicine Carrboro North Carolina 27510
Texas Children's Hospital Houston Texas 77030

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT05127226, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Aug 19, 2026 · Synced Sep 11, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT05127226 live on ClinicalTrials.gov.

View official ClinicalTrials.gov record →