Current Research Studies
Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults with Angelman Syndrome
REVEAL
What is the goal of the study?
This is a Phase 3, randomized, double-blind (DB), placebo-controlled, global, multi-center registrational study consisting of a Screening Period of up to 28 days, a 60-week (approximately 15-month) Double Blind Treatment Evaluation Period, followed by a 25-month Long-Term Extension Treatment Period, wherein all trial participants will receive ION582, and an 8-month Post-LTE Treatment Follow-up Period. The primary assessment for efficacy will occur at a clinic visit on Week 52 of the Treatment Evaluation Period. ION582 will be evaluated at two dose levels to assess efficacy and safety relative to Placebo as outlined below.
Who can participate in the study?
Please contact the study team listed below to learn more.