Natural History Study for Angelman Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to better understand Angelman Syndrome by observing children and adults diagnosed with it. Researchers will gather information from medical professionals and parents to track how the condition changes over time. This knowledge will help improve future treatments. Individuals with a confirmed diagnosis of Angelman Syndrome and no other neurodevelopmental issues may find this trial suitable. As an unphased trial, participation offers a unique opportunity to contribute to foundational research that could lead to better treatments.
Do I need to stop my current medications for the trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Why are researchers excited about this trial?
Researchers are excited about the Angelman Syndrome Natural History Study because it aims to deepen our understanding of Angelman Syndrome by observing the condition's natural progression over time. Unlike treatment-focused studies, this observational study doesn't involve administering new drugs or therapies. Instead, it collects valuable data on how the syndrome affects individuals as they grow, which could lead to better treatment strategies in the future. By providing a clearer picture of the condition's development, this study may pave the way for innovative treatments tailored to the unique needs of those with Angelman Syndrome.
Who Is on the Research Team?
Wen-Hann Tan, BMBS
Principal Investigator
Boston Children's Hospital
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observation
Participants are observed for developmental, communication, and adaptive skills using various scales
Follow-up
Participants are monitored for safety and effectiveness after the observation period
What Are the Treatments Tested in This Trial?
Interventions
- Observational Study
Find a Clinic Near You
Who Is Running the Clinical Trial?
Boston Children's Hospital
Lead Sponsor
Angelman Syndrome Foundation Canada
Collaborator
Angelman Syndrome Foundation, Inc.
Collaborator
Food and Drug Administration (FDA)
Collaborator
Foundation for Angelman Syndrome Therapeutics Canada
Collaborator
Canadian Angelman Syndrome Society
Collaborator
Foundation for Angelman Syndrome Therapeutics
Collaborator
University of California, San Diego
Collaborator
Angelman Syndrome Biomarker & Outcome Measure Consortium (A-BOM)
Collaborator