New FDA-Funded Study Aims to Improve Angelman Syndrome Clinical Trials
A new study, backed by the U.S. Food and Drug Administration, is in progress to enhance understanding of Angelman syndrome. Building on a previous NIH-funded study, this research aims to improve clinical trial outcomes and better assist patients.
The ongoing study, registered as NCT04507997, is currently recruiting up to 150 patients of all ages at seven U.S. sites. It seeks to build on the knowledge gained from the previous natural history study, which ran from 2006 to 2014 and involved over 300 participants. The current research focuses on understanding the most significant disease symptoms and developing more sensitive and specific measures for clinical trials.
This collaborative effort is supported by the Foundation for Angelman Syndrome Therapeutics (FAST), the Angelman Syndrome Biomarker and Outcome Measure (ABOM) Consortium, the Research Triangle Institute International (RTI), and Boston Children's Hospital. Additionally, a new partnership involving the Forschungsinstitut für Nutztierbiologie (FBN) and Hochschule Stralsund is accelerating data analysis, correlation, and publication of datasets from the ongoing and previous studies.
The study is expected to conclude in August 2022, with data from over 450 Angelman patients analyzed. This will help identify relevant goals for clinical trials and improve outcomes for those living with Angelman syndrome.
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