Angelman Syndrome clinical trials at UCLA

2 in progress, 1 open to eligible people

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
open to eligible people ages 2-50
The purpose of this study is to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome caused by a deletion or mutation of the UBE3A gene.
Los Angeles, California and other locations
Long-term Extension of GTX-102 in Angelman Syndrome
Sorry, accepting new patients by invitation only
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Los Angeles, California and other locations

Last updated: July 28, 2026

REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome