Phase 1/2 study
Investigational medicine, ION582 is administered by IT injection

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.
Locations & Contacts
Boston Children’s Hospital
Boston, MA
Liana Cole – liana.cole@childrens.harvard.edu
Liana Cole – liana.cole@childrens.harvard.edu
University of North Carolina at Chapel Hill
Carrborro, NC
Carrborro, NC
Hannah Riehl – hannah.riehl@cidd.unc.edu
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: IonisAngelmanStudy@clinicaltrialmedia.com
Reference Study ID Number: NCT05127226