Recruiting Studies
Advanced SearchData: ClinicalTrials.gov · Source of record for eligibility and locations
Top Cities for Angelman Syndrome Clinical Trials
Angelman Syndrome clinical trials are recruiting across 11 cities. Here are the cities with the most active studies:
Angelman Syndrome Trial Phases
5 research organizations are sponsoring angelman syndrome trials.
About Angelman Syndrome
Angelman syndrome is a rare genetic disorder primarily affecting the nervous system, characterized by severe developmental delays, speech impairment, seizures, and a happy demeanor. It is caused by loss of function of the UBE3A gene. Treatment focuses on managing symptoms and maximizing developmental potential.
Clinical trials are advancing new treatments for angelman syndrome. Currently, 5 studies are recruiting a combined 547 participants across the United States. Research is being conducted by 5 organizations including Boston Children's Hospital, OHB Pediatrics Ltd., Massachusetts General Hospital and 2 others.
2026 Angelman Syndrome Research Landscape
As of October 2026, the angelman syndrome clinical trial landscape includes 5 actively recruiting studies across 11 cities in the United States. These studies are collectively seeking 547 participants, with an average enrollment target of 109 per study.
The research spans multiple phases of development: 2 studies are in PHASE3, 1 study is in PHASE1, 1 study is in PHASE2. The presence of Phase 3 trials indicates that some angelman syndrome treatments are in late-stage testing and may be approaching regulatory review.
Research is being led by 5 different organizations, including Boston Children's Hospital, OHB Pediatrics Ltd., Massachusetts General Hospital, Ionis Pharmaceuticals, Inc., MavriX Bio, LLC.
Geographically, angelman syndrome trials are most concentrated in Chicago, Illinois (3 trials); Los Angeles, California (2 trials); San Diego, California (2 trials); Boston, Massachusetts (2 trials); Carrboro, North Carolina (2 trials) and 6 other cities.
Featured Angelman Syndrome Studies
Highlighted recruiting studies for angelman syndrome, selected by enrollment size and research scope.
Angelman Syndrome Natural History Study
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.
BEACON - Phase III Clinical Study of Rugonersen in Angelman Syndrome.
Purpose of the study is to evaluate the efficacy and safety of intrathecally administered rugonersen in pediatric and adult participants with Angelman syndrome.
A Natural History Study of Angelman Syndrome
The goal of this observational study is to learn about the natural progression of Angelman syndrome (AS) in children and adults with a confirmed genetic diagnosis of AS. The main questions it aims to answer are: * How do developmental skills, such as communication, motor abilities, and adaptive behaviors, change over a 1-year period in people with AS? * Are there specific patterns in brain activi...
Frequently Asked Questions About Angelman Syndrome Clinical Trials
Are there angelman syndrome clinical trials near me?
Yes, there are 5 angelman syndrome clinical trials currently recruiting across 11+ cities in the United States, including Chicago, Illinois; Los Angeles, California; San Diego, California. Browse the studies above to find one at a location convenient for you.
How do I join a angelman syndrome clinical trial?
To join a angelman syndrome clinical trial: 1) Browse the available studies on this page, 2) Click on a study that interests you, 3) Check the study locations to find a site near you, 4) Review the eligibility criteria, and 5) Contact the study site or complete the eligibility form. The process is free and you can withdraw at any time.
Are angelman syndrome clinical trials free?
Yes, participation in angelman syndrome clinical trials is free. Study-related treatments, medical tests, and doctor visits are provided at no cost to participants. Many studies also offer compensation for your time and travel expenses.
What types of angelman syndrome treatments are being studied?
Current angelman syndrome clinical trials are testing a range of approaches across PHASE3 (2 studies), PHASE1 (1 study), PHASE2 (1 study). These include new drugs, combination therapies, medical devices, and other interventions sponsored by 5 research organizations.
Is it safe to participate in angelman syndrome clinical trials?
Clinical trials are carefully regulated by the FDA and institutional review boards (IRBs). All trials must follow strict safety protocols, and participants receive close medical monitoring throughout the study. You can withdraw from a trial at any time without penalty.
Data updated October 14, 2026 from ClinicalTrials.gov
About This Data
Data: ClinicalTrials.gov · Source of record for eligibility and locations
Clinical trial information on this page is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health (NIH) and National Library of Medicine (NLM). Study data is refreshed every hour to ensure accuracy.
Medical Disclaimer: The information provided on this page is for informational purposes only and does not constitute medical advice. Always consult with a qualified healthcare provider before making decisions about clinical trial participation or changes to your treatment plan.
Page reviewed by the HelloStudys Research Team · Last updated October 14, 2026 · Data from ClinicalTrials.gov