REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome

To join this study, participants need to have Angelman syndrome caused by a specific gene fault (either a UBE3A deletion or mutation). They should be between 2 and 50 years old, depending on which group they fit into. Participants need to be generally well enough to undergo some medical procedures mentioned below.

It's important that any other medicines they are taking for Angelman syndrome, like those for seizures or sleep, have been on a steady dose for at least 8 weeks before the study starts. This helps doctors see the effect of the new medicine more clearly. Also, their caregiver or legal guardian must agree to all study procedures and not share personal medical details about the study online.

Some things might mean a person can't join. For example, if they have certain serious medical issues that might make the new medicine unsafe, or if they have brain or spine problems that would make a procedure called a 'lumbar puncture' difficult or risky. The study doctors will carefully check everyone to make sure it's safe for them to take part.

If you join the study, it will involve several parts over a long period. First, there's a screening period of up to 28 days to check if you're suitable. Then, for about 60 weeks, you'll be in the main treatment phase where you'll either receive the study medicine (ION582) or a dummy medicine (placebo). Neither you, your family, nor the study doctors will know which one you're getting during this time.

After this, everyone who completes this first phase can enter a longer period, lasting about 25 months, where all participants will receive the active study medicine. Finally, there's a follow-up period of about 8 months after you stop taking the medicine to see how you're doing. The study involves regular visits to the clinic for assessments and to receive the medication. The total study duration, including all phases, is over three years.