Angelman Syndrome Natural History Study

NCT04507997 · tracked via the Priya Life Science Canada tracker

Sponsor

Boston Children's Hospital

Phase

Observational

Started

2018-08-01

Last updated

2025-04-03

Condition(s) studied

Angelman Syndrome

Study summary

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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

Inclusion Criteria: * Molecular diagnosis of Angelman syndrome Exclusion Criteria: * Presence of another condition, unrelated to Angelman syndrome, that affects neurodevelopment

Primary outcome measure(s)

Medical History — Through study completion, an average of 1 year
Includes various medical complications, including seizure history
Vineland Adaptive Behavior Scales, 3rd edition — Through study completion, an average of 1 year
Developmental and Adaptive skills
Bayley Scales of Infant and Toddler Development, 4th edition — Through study completion, an average of 1 year
Developmental skills
Observer-Reported Communication Ability (ORCA) Measure — Through study completion, an average of 1 year
Communication skills

Trial sites (11)

Facility	City	Region	Status
Cedars-Sinai Guerin Children's	Los Angeles	California	Not Yet Recruiting
Rady Children's Hospital, San Diego	San Diego	California	Recruiting
Children's Hospital Colorado, Anschutz Medical Campus	Aurora	Colorado	Recruiting
Emory University School of Medicine	Atlanta	Georgia	Recruiting
Rush University Children's Hospital	Chicago	Illinois	Recruiting
Boston Children's Hospital	Boston	Massachusetts	Recruiting
The Carolina Institute for Developmental Disabilities	Carrboro	North Carolina	Recruiting
Monroe Carell Jr. Children's Hospital at Vanderbilt	Nashville	Tennessee	Recruiting
Alberta Children's Hospital	Calgary	Alberta	Recruiting
BC Children's Hospital	Vancouver	British Columbia	Recruiting
Children's Hospital of Eastern Ontario	Ottawa	Ontario	Recruiting

More Boston Children's Hospital trials in Canada

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Outcomes and Health Care Resource Utilization in Pediatric Congenital Heart Disease Patients Undergoing Non-Cardiac Procedures Recruiting

Improving Infant Hydrocephalus Outcomes in Uganda Active, not recruiting

Other trials for the same condition

Long-term Extension of GTX-102 in Angelman Syndrome Active, not recruiting

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) Active, not recruiting

REVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman Syndrome Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04507997 on ClinicalTrials.gov ↗ ← All trials in Canada