FAST Supports Work Into Data Gained in 2 Natural History Studies
A new collaboration will help to speed the analysis, correlation, and publication of data sets from an ongoing natural history study, and a previous similar study in people with Angelman syndrome (AS).
The ongoing study (NCT04507997), funded by the U.S. Food and Drug Administration, is looking to enroll up to 150 patients of all ages — children through adults — and is still recruiting at seven U.S. sites.
The natural history study that ran from 2006 to 2014 had more than 300 participants, and was funded by the National Institutes of Health (NIH).
Natural history studies collect crucial information about a disease’s onset and progression in the absence of treatment; in these observational studies, no therapies are tested on participants.
The Foundation for Angelman Syndrome Therapeutics (FAST), though its U.S. and Canada offices, is supporting this research partnership involving the Angelman Syndrome Biomarker and Outcome Measure (ABOM) Consortium, the Research Triangle Institute International (RTI), and Boston Children’s Hospital.
Its goal is to have a clearer idea of the common features of Angelman syndrome over an extended period of time — including key clinical characteristics, medical complications, and quality of life. Such knowledge can support the development of clinical trials outcomes, known as endpoints, that best reflect patient concerns and needs, and ultimately help to improve their care and treatment.
“Identifying outcome measures that accurately reflect the experiences of individuals with Angelman syndrome and their families and are also sensitive to potential treatment effects is critical for the success of clinical trials,” Anne Wheeler, PhD, neurodevelopmental physiologist and leader of RTI’s research team, said in a FAST press release.
“Fortunately, the Angelman community has provided a wealth of information on promising outcome measures through their participation in the Natural History Study. This funding [from FAST] will allow us to look closely at how well these measures work to best inform the tools used to assess how well new therapeutics are working.”
The ongoing natural history study into Angelman syndrome aims to build on the knowledge gained in the earlier effort. Particularly, it is looking to better understand disease symptoms of greatest importance to patients, while also developing more sensitive and specific measures of use in clinical trials of potential treatments.
This study is expected to conclude in August 2022.
Data on the potentially more than 450 Angelman patients in these two studies will be analyzed to identify relevant goals for clinical trials that might best capture their symptoms, concerns, and treatment responses.
“This FAST US and FAST Canada co-funded grant will enable RTI International to expedite the analysis of this robust data in unique ways as additional information and endpoints are added,” FAST stated in its release.