Groundbreaking FDA-Backed Study Aims to Improve Angelman Syndrome Clinical Trials
A groundbreaking study, backed by the U.S. Food and Drug Administration, is underway to understand disease symptoms that matter most to patients with Angelman syndrome (AS). This research, led by Anne Wheeler of RTI International, aims to develop more sensitive and specific measures for clinical trials. The study, set to conclude in August 2022, is expected to analyze data from over 450 AS patients.
The ongoing study, NCT04507997, is currently recruiting up to 150 patients of all ages at seven U.S. sites. This follows a previous natural history study, funded by the National Institutes of Health (NIH), which ran from 2006 to 2014 and involved over 300 participants.
The research, supported by the Foundation for Angelman Syndrome Therapeutics (FAST), is a collaboration between the ABOM Consortium, RTI, and Boston Children's Hospital. It seeks to better understand AS over time, including key clinical characteristics, medical complications, and quality of life. This understanding will help improve clinical trial outcomes and patient care.
The study is expected to conclude in August 2022, with data from over 450 AS patients analyzed to identify relevant clinical trial goals. A new collaboration aims to speed up data analysis and publication from this ongoing study and a previous study on AS, further advancing our understanding and treatment of this condition.
