Angelman Syndrome Study Aims to Understand Disease Symptoms, Support Clinical Trials
A new natural history study on Angelman syndrome, led by the Angelman Syndrome Foundation, Baylor College of Medicine, and UCLA, is underway. This study, set to conclude in August 2022, aims to build on previous research and understand disease symptoms most important to patients. It involves over 450 patients and is supported by FAST and other institutions.
The ongoing study, registered as NCT04507997, is enrolling up to 150 patients of all ages across seven U.S. sites. It is funded by the U.S. Food and Drug Administration and is expected to identify relevant clinical trial goals. This research is part of a larger effort by the Angelman Syndrome Biomarker and Outcome Measure (ABOM) Consortium, RTI International, and Boston Children's Hospital to better understand AS features over time and support the development of patient-centric clinical trial endpoints.
Natural history studies, like this one, collect crucial information about a disease's onset and progression without testing any therapies on participants. This study follows a previous NIH-funded study from 2006 to 2014, which had over 300 participants. FAST is co-funding the current research to expedite data analysis and add unique information and endpoints.
The ongoing natural history study on Angelman syndrome, involving over 450 patients, is expected to conclude in August 2022. It aims to understand disease symptoms most important to patients and support the development of more sensitive clinical trial measures. The findings from this study, along with the previous NIH-funded study, will provide valuable insights into AS and potentially improve future clinical trials.
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