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Trials & Studies in AS

FAST is committed to keeping you up to date with the latest in research for Angelman syndrome and news on upcoming and ongoing clinical trials. On this page we have included a few resources to assist you in understanding the basics of clinical trials and observational studies along with what is currently active on clinicaltrials.gov for Angelman syndrome. We are here to keep you informed and up to date on the various different options available for individuals living with Angelman syndrome.

Participate in a Clinical Trial

ASO / Ultragenyx / FAST Pillar 2 / Status: Active, Not Recruiting

ASO / Ionis / FAST Pillar 2 / Status: Active, Not Recruiting

ASO / Roche / FAST Pillar 2 / Status: Active, Not Recruiting

Downstream Target / Roche / FAST Pillar 3 / Status: Recruiting

Downstream Target / Neuren / FAST Pillar 3 / Status: Recruiting

Observational Studies

In some studies the investigator (sometimes referred to as a researcher, or a primary investigator [PI]), does not test new drugs on an individual with a disorder they are evaluating. Instead, the goal of the research is to observe the person or patient to learn more about a condition and how it changes (or doesn’t change) over time. This observation or assessment helps the investigator understand the disorder, better describe the condition, and understand the most impacted areas of that condition (for example: communication abilities, sleep, motor function, fine motor function, seizures, etc). A common example of an observational study is a natural history study where the investigator, or doctor, is trying to understand what Angelman syndrome looks like in individuals over time without a therapy. This helps to identify what the important symptoms of the condition are that might be evaluated in clinical trials of new drugs intended to treat the condition.  Sometimes treatment studies compare outcomes in patients taking a new drug to those in untreated patients from observational studies to help prove that the drug is improving symptoms of the condition beyond what would be expected as part of the natural history of the disorder.

Participate in an Observational Study

Angelman Syndrome Natural History Study

Angelman Syndrome Natural History Study - UK

Angelman Syndrome Video Assessment (ASVA) Source Material Study (ASVA SMS)

The Global Angelman Syndrome Registry

Development of a Newborn Screening Assay for Angelman Syndrome and Prader-Willi Syndrome

Angelman Syndrome News