Actively Recruiting
Angelman Syndrome Natural History Study
Led by Boston Children's Hospital · Updated on 2025-04-03
300
Participants Needed
11
Research Sites
N/A
Total Duration
On this page
Sponsors
B
Boston Children's Hospital
Lead Sponsor
U
University of California, San Diego
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a prospective, longitudinal natural history study to better understand Angelman syndrome in children and adults. The study aims to collect detailed data through both investigator observations and reports from parents. This information will help establish Angelman syndrome-specific norms for outcome measures, supporting the design of future clinical trials and improving care for those affected by the condition. This observational study does not involve any treatments or interventions. Instead, participants will be followed over time to gather information about their medical history and development. The study collects data through various assessments including the Vineland Adaptive Behavior Scales, Bayley Scales of Infant and Toddler Development, and the Observer-Reported Communication Ability Measure, conducted over an average period of one year. Participants will be involved in regular evaluations that include medical history reviews and developmental assessments. These assessments provide valuable information on communication abilities and adaptive behaviors. The study is sponsored by Boston Children's Hospital and will continue through April 2031. The collected data aims to support future research and enhance understanding of Angelman syndrome's progression and care needs.
CONDITIONS
Brief Title
Angelman Syndrome Natural History Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Molecular diagnosis of Angelman syndrome
You will not qualify if you...
- Presence of another condition, unrelated to Angelman syndrome, that affects neurodevelopment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Approximately 1 year
Participants are observed over time to collect data on the natural history of Angelman syndrome and to establish outcome measure norms.
Multiple visits over the course of 1 year
Trial Site Locations
Total: 11 locations
1
Cedars-Sinai Guerin Children's
Los Angeles, California, United States, 90048
Not Yet Recruiting
2
Rady Children's Hospital, San Diego
San Diego, California, United States, 92123
Actively Recruiting
3
Children's Hospital Colorado, Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Actively Recruiting
5
Rush University Children's Hospital
Chicago, Illinois, United States, 60612
Actively Recruiting
6
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
7
The Carolina Institute for Developmental Disabilities
Carrboro, North Carolina, United States, 27510
Actively Recruiting
8
Monroe Carell Jr. Children's Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
Actively Recruiting
9
Alberta Children's Hospital
Calgary, Alberta, Canada
Actively Recruiting
10
BC Children's Hospital
Vancouver, British Columbia, Canada
Actively Recruiting
11
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Actively Recruiting
Research Team
W
Wen-Hann Tan, BMBS
B
Batsheva Friedman, MA
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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