Actively Recruiting
Phase 3 Study of Obudanersen to Treat Angelman Syndrome in Children and Adults Ages 2 to 50
Led by Ionis Pharmaceuticals, Inc. · Updated on 2026-08-12
158
Participants Needed
42
Research Sites
139 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are studying the effects of obudanersen in children and adults with Angelman syndrome caused by specific genetic changes in the UBE3A gene. This Phase 3 clinical trial aims to evaluate the safety and effectiveness of obudanersen compared to a placebo. The study involves two groups based on age pediatric participants aged 2 to less than 18 years, and adults aged 18 to 50 years. Participants will receive 80 mg of obudanersen or a matching placebo through injections into the spinal fluid every 12 weeks during a double-blind treatment period lasting about 60 weeks. After this, participants can enter a long-term extension period of about 25 months where all will receive obudanersen, with the dose kept blinded. Following the extension, there is an 8-month follow-up to monitor participants. Throughout the study, participants and their caregivers will attend scheduled visits for treatment and assessments. These include tests measuring communication, cognition, behavior, daily living skills, sleep problems, and motor skills using various clinical scales. Safety is monitored by tracking side effects, vital signs, and lab tests. The total study participation spans several years including screening, treatment, extension, and follow-up periods.
CONDITIONS
Brief Title
REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome
Research Team
I
Ionis Pharmaceuticals, Inc.
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