An observational study in Angelman Syndrome, sponsored by Boston Children's Hospital. Recruiting at 11 sites in 2 countries. Per ClinicalTrials.gov, last updated 2025-04-03.
Sponsored by Boston Children's Hospital · Observational
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.
The overall goal is to increase our understanding of the long-term natural history of Angelman syndrome and obtain Angelman-specific norms for outcome measures that can be used in clinical trials, ultimately improving the care of individuals with Angelman syndrome.
46 studies on the registry are indexed under Angelman Syndrome; 16 are open to participants now.
This study's planned enrollment of 300 is above the median of 101 across 20 observational studies indexed under Angelman Syndrome.
Browse Angelman Syndrome studies →Boston Children's Hospital is the lead sponsor of 598 studies on the registry; 151 are open to participants now.
Of its 31 completed or terminated interventional studies of FDA-regulated products, 18 (58%) have results posted.
Counted across the registry records on this site, refreshed daily.
All individuals with a molecular diagnosis of Angelman syndrome
Exclusion Criteria:
Medical History
Includes various medical complications, including seizure history
Time frame: Through study completion, an average of 1 year
Vineland Adaptive Behavior Scales, 3rd edition
Developmental and Adaptive skills
Time frame: Through study completion, an average of 1 year
Bayley Scales of Infant and Toddler Development, 4th edition
Developmental skills
Time frame: Through study completion, an average of 1 year
Observer-Reported Communication Ability (ORCA) Measure
Communication skills
Time frame: Through study completion, an average of 1 year
Plan to share: No
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Boston Children's Hospital