BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. (NCT07605429)
A Randomized, Multi-center, Double-blind, Sham-controlled, Phase III Clinical Study to Evaluate the Efficacy and Safety of Intrathecally Administered Rugonersen in Pediatric and Adult Participants With Angelman Syndrome
🩺 Plain-English Summary (8th-Grade Level)
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
Who can join: Review the eligibility criteria below.
Location: Rady Children's Hospital — San Diego, California
Age range: 1 Year to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (2 Sites)
- Rady Children's Hospital — San Diego, California
- University of North Carolina at Chapel Hill School of Medicine — Chapel Hill, North Carolina
📞 Contact the Study Coordinator
Phone: (857) 302-2294
Email: patientadvocacy@oakhillbio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Angelman Syndrome
- HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome — San Diego, California
- Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) — Los Angeles, California
- Angelman Syndrome Natural History Study — Los Angeles, California
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome — Los Angeles, California
- A Natural History Study of Angelman Syndrome — Lexington, Massachusetts
Explore by condition: All Angelman Syndrome clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.