Angelman Syndrome Natural History Study
ConditionAngelman Syndrome
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorBoston Children's Hospital
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
Molecular diagnosis of Angelman syndrome
Disqualifiers
Presence of another condition, unrelated to Angelman syndrome, that affects neurodevelopment
Trial population
All individuals with a molecular diagnosis of Angelman syndrome
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
Medical History
Includes various medical complications, including seizure history
Time frame
Through study completion, an average of 1 year2
Vineland Adaptive Behavior Scales, 3rd edition
Developmental and Adaptive skills
Time frame
Through study completion, an average of 1 year3
Bayley Scales of Infant and Toddler Development, 4th edition
Developmental skills
Time frame
Through study completion, an average of 1 year4
Observer-Reported Communication Ability (ORCA) Measure
Communication skills
Time frame
Through study completion, an average of 1 yearSponsors and contacts
Click on the lead sponsor to view all of their trials.
Boston Children's Hospital
Lead sponsor
University of California, San Diego
Collaborator
Angelman Syndrome Biomarker & Outcome Measure Consortium (A-BOM)
Collaborator
Food and Drug Administration (FDA)
Collaborator
Angelman Syndrome Foundation, Inc.
Collaborator
Source ClinicalTrials.gov