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NCT04507997 Clinical Trial

Angelman Syndrome Natural History Study

Angelman Syndrome Clinical Trial

Official title:
Angelman Syndrome Natural History Study

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT04507997
Other study ID # IRB-P00025249
Secondary ID
Status Recruiting
Phase
First received
Last updated
Start date August 1, 2018
Est. completion date August 2025

Study information
Verified date May 2024
Source Boston Children's Hospital
Contact Wen-Hann Tan, BMBS
Phone 617-355 6394
Email wen-hann.tan@childrens.harvard.edu
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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.

Description:

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.

Recruitment information / eligibility
Status Recruiting
Enrollment 300
Est. completion date August 2025
Est. primary completion date August 2025
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility Inclusion Criteria: - Molecular diagnosis of Angelman syndrome Exclusion Criteria: - Presence of another condition, unrelated to Angelman syndrome, that affects neurodevelopment

Study Design

Related Conditions & MeSH terms

Locations
Country Name City State
Canada Alberta Children's Hospital Calgary Alberta
Canada Children's Hospital of Eastern Ontario Ottawa Ontario
Canada BC Children's Hospital Vancouver British Columbia
United States Emory University School of Medicine Atlanta Georgia
United States Boston Children's Hospital Boston Massachusetts
United States The Carolina Institute for Developmental Disabilities Carrboro North Carolina
United States Rush University Children's Hospital Chicago Illinois
United States Monroe Carell Jr. Children's Hospital at Vanderbilt Nashville Tennessee
United States Rady Children's Hospital, San Diego San Diego California

Sponsors (8)
Lead Sponsor Collaborator
Boston Children's Hospital Angelman Syndrome Biomarker & Outcome Measure Consortium (A-BOM), Angelman Syndrome Foundation Canada, Angelman Syndrome Foundation, Inc., Food and Drug Administration (FDA), Foundation for Angelman Syndrome Therapeutics, Foundation for Angelman Syndrome Therapeutics Canada, University of California, San Diego

Countries where clinical trial is conducted

United States,  Canada, 

Outcome
Type Measure Description Time frame Safety issue
Primary Medical History Includes various medical complications, including seizure history Through study completion, an average of 1 year
Primary Vineland Adaptive Behavior Scales, 3rd edition Developmental and Adaptive skills Through study completion, an average of 1 year
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