Phase III Study of Intrathecal Rugonersen Compared to Sham in Children and Adults with Angelman Syndrome

What this Trial Is About

Researchers are evaluating the safety and effectiveness of intrathecal rugonersen in children and adults with Angelman syndrome. This Phase III study compares rugonersen to a sham procedure in up to 165 participants to assess improvements in cognition and communication. Following this, there is an open-label extension lasting about 116 weeks to monitor long-term effects and safety of the treatment. Participants will be randomly assigned to receive either the study drug rugonersen or a sham procedure administered intrathecally. The initial treatment phase lasts 56 weeks, after which participants may join the open-label extension for up to 2 years to continue receiving and being assessed for the investigational treatment. Both parts include multiple clinical visits and monitoring procedures. During the study, participants will undergo assessments including cognitive and communication testing, clinician evaluations of Angelman syndrome symptoms, EEG recordings, and safety monitoring for adverse events. Blood draws, lumbar punctures, and intrathecal injections will be performed under sedation or anesthesia as needed. Caregivers play an important role in providing information and completing assessments. The total participation duration can extend over several years including long-term follow-up.

BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.