Clinical Trial


Phase 1/2 study

Investigational medicine, ION582 is administered by IT injection

IONIS Pharaceuticals
ION582 is an investigational antisense oligonucleotides (ASO) designed to increase production of UBE3A protein.
Following a screening period of up to 4 weeks, eligible participants will receive ION582. Participants will be followed for up to 32 weeks after dosing.

Participant Requirements

Inclusion Criteria

  • Diagnosis of Angelman syndrome (mutation or deletion)
  • Between 2 and 50 years old
  • Currently receiving stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose.
  • Parent must be willing to not share personal or study information on social media or websites until notified that the study is completed.

See full details on the clinical trials website.

Locations & Contacts

Baylor School of Medicine/Texas Children’s Hospital
Houston, TX
Beverly Feldman-
Boston Children’s Hospital
Boston, MA
Monica Ciaffi –
Colorado Children’s Hospital
Aurora, CO
Jessica Duis, MD –
Rady Children’s Hospital
San Diego, CA
Lynne Bird, MD –
Rush University Medical Center
Chicago, IL
Kathryn Kudlacz  –
University of North Carolina at Chapel Hill
Carrborro, NC
Hannah Riehl –

Sydney Children’s Hospital, Kids Cancer Centre
Randwick, Australia

Sheba Medical Center
Ramat Gan, Israel

Azienda Ospedaliera Universitaria Pisana
Pisa, Italy

STRONG Group University of Oxford
Oxford, Oxfordshire, United Kingdom

More Information

Questions about this study can be directed to:

Reference Study ID Number: NCT05127226