Angelman Syndrome Natural History Study

Brief Title

Angelman Syndrome Natural History Study

Official Title

Angelman Syndrome Natural History Study

Brief 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.
    

Detailed 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.
    


Study Type

Observational


Primary Outcome

Medical History


Condition

Angelman Syndrome



Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

150

Start Date

August 1, 2018

Completion Date

August 2022

Primary Completion Date

August 2022

Eligibility Criteria

        Inclusion Criteria:

          -  Molecular diagnosis of Angelman syndrome

        Exclusion Criteria:

          -  Presence of another condition, unrelated to Angelman syndrome, that affects
             neurodevelopment
      

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Wen-Hann Tan, BMBS, 617-355 6394, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT04507997

Organization ID

IRB-P00025249


Responsible Party

Principal Investigator

Study Sponsor

Boston Children's Hospital

Collaborators

 Food and Drug Administration (FDA)

Study Sponsor

Wen-Hann Tan, BMBS, Study Chair, Boston Children's Hospital


Verification Date

August 2021