🏛️ Verified NIH Record • Updated 2025-03-30
Angelman Syndrome Natural History Study (NCT04507997)
🟢 Recruiting Interventional Protocol 📊 Observational Clinical Research Sponsor: Boston Children's Hospital
🩺 Plain-English Summary (8th-Grade Level)
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be useful for future clinical trials.
Who can join: Review the eligibility criteria below.
Location: Cedars-Sinai Guerin Children's — Los Angeles, California
📋 "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 (11 Sites)
- Cedars-Sinai Guerin Children's — Los Angeles, California
- Rady Children's Hospital, San Diego — San Diego, California
- Children's Hospital Colorado, Anschutz Medical Campus — Aurora, Colorado
- Emory University School of Medicine — Atlanta, Georgia
- Rush University Children's Hospital — Chicago, Illinois
- Boston Children's Hospital — Boston, Massachusetts
- The Carolina Institute for Developmental Disabilities — Carrboro, North Carolina
- Monroe Carell Jr. Children's Hospital at Vanderbilt — Nashville, Tennessee
- Alberta Children's Hospital — Calgary, Alberta
- BC Children's Hospital — Vancouver, British Columbia
- Children's Hospital of Eastern Ontario — Ottawa, Ontario
📞 Contact the Study Coordinator
Phone: (617) 355-6394
Email: wen-hann.tan@childrens.harvard.edu
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
- A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome — Los Angeles, California
- BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome. — San Diego, 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.