Phase 3 study
Investigational medicine, ION582 is administered by IT injection
Not Recruiting
ION582 is an investigational antisense oligonucleotides (ASO) designed to increase production of UBE3A protein.
This is a Phase 3, randomized, double-blind, placebo-controlled study.
There are 4 phases to the study:
- Screening period of up to 28 days
- Treatment period: 60-week double blind, placebo-controlled
- Long-Term Extension (LTE) treatment period: approximately 25-months
- Post-LTE follow-up: approximately 8-months
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 8 weeks prior to first visit.
- Parent must be willing to not share personal or study information on social media or websites until notified that the study is completed.