RecruitingPhase 1Phase 2NCT05127226

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

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

Sponsor

Ionis Pharmaceuticals, Inc.

Enrollment

70 participants

Start Date

Dec 22, 2021

Study Type

INTERVENTIONAL

Conditions

Angelman Syndrome

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.

Eligibility

Min Age: 0 YearsMax Age: 50 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called ION582 for people with angelman syndrome. The study is currently recruiting participants at 11 locations. People eligible for this study include aged 0 Years to 50 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGION582

ION582 will be administered by IT injection.

Locations(11)

Rady Children's Hospital

San Diego, California, United States

Colorado Children's Hospital Research Institute

Aurora, Colorado, United States

Rush University Medical Center

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

University of North Carolina at Chapel Hill School of Medicine

Carrboro, North Carolina, United States

Texas Children's Hospital

Houston, Texas, United States

Sydney Children's Hospital, Kids Cancer Centre

Randwick, Australia

Necker-Enfants Malades Hospital

Paris, France

Sheba Medical Center

Ramat Gan, Israel

Azienda Ospedaliera Universitaria Pisana

Pisa, Italy

STRONG Group University of Oxford

Oxford, Oxfordshire, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT05127226

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