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Study identifier: NCT05127226 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

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

Condition: Angelman Syndrome  ·  Sponsor: Ionis Pharmaceuticals, Inc.

PhasePhase 1/Phase 2
Planned participants70
Who can joinAll sexes, 0 Years to 50 Years
Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Ionis Pharmaceuticals  ·  (844) 200-6263  ·  IonisAngelmanStudy@clinicaltrialmedia.com

Always discuss trial participation with your own doctor first.

Locations (11)

Rady Children's HospitalSan Diego, California, United StatesRecruiting
Colorado Children's Hospital Research InstituteAurora, Colorado, United StatesRecruiting
Rush University Medical CenterChicago, Illinois, United StatesRecruiting
Boston Children's HospitalBoston, Massachusetts, United StatesRecruiting
University of North Carolina at Chapel Hill School of MedicineCarrboro, North Carolina, United StatesRecruiting
Texas Children's HospitalHouston, Texas, United StatesActive Not Recruiting
Sydney Children's Hospital, Kids Cancer CentreRandwick, AustraliaActive Not Recruiting
Necker-Enfants Malades HospitalParis, FranceRecruiting
Sheba Medical CenterRamat Gan, IsraelRecruiting
Azienda Ospedaliera Universitaria PisanaPisa, ItalyRecruiting
STRONG Group University of OxfordOxford, Oxfordshire, United KingdomActive Not Recruiting

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Source record: clinicaltrials.gov/study/NCT05127226