The Global Prader-Willi Syndrome Registry [www.pwsregistry.org] was developed in 2015 by FPWR in collaboration with international experts in PWS and stakeholders in the PWS community. The goals of the Registry are to build a comprehensive database to better understand the natural history and full spectrum of PWS characteristics, facilitate clinical trial recruitment and enrollment, and determine areas of needed research and treatment for PWS.
The Registry is hosted on the National Organization for Rare Disorders IAMRARE Registry platform, and currently includes approximately 2,000 PWS participants from 28 countries. The majority of participants (~75%) are from the US, which likely represents ~10% of the US PWS population, followed by Canada, the UK and Australia.
The Registry consists of a series of surveys to gather information from parent/caregivers on the natural history of PWS, including pregnancy, birth history, diagnosis, developmental history, medical history, behavior and mental health, medications, supplements, nonmedical interventions, and quality of life. Surveys are updatable, and participants are asked to update medical history at annual intervals. In addition, longitudinal surveys capture behavioral assessments over time.
The Registry has also supported several substudies. A recently initiated longitudinal study of serious medical events in PWS (“PATH for PWS”), supported by Zafgen in collaboration with FPWR, will provide a reference dataset to better understand the scope and frequency of these events in the PWS population over a four-year period. The Registry is also being used to support specific activities of the PWS-Clinical Trial Consortium, such as caregiver burden assessment and development of new clinical trial outcome measures. The ability to link these measures with other aspects of the PWS participants’ record, such as information on medical and social history, enhances the richness of the data available for analysis.
De-identified, aggregated data from the Registry is being shared with academic and industry collaborators conducting retrospective studies or conducting research and/or clinical trials on new therapies. Third parties are granted access to de-identified Registry data according to the guidelines of the Global PWS Registry Advisory Board, and an independent IRB provides ethical oversight for Registry activities. Third parties seeking to recruit participants for research studies through the Registry must demonstrate evidence of IRB approval of the research study that will be recruited. For additional information or inquiries, please contact us at: firstname.lastname@example.org.
For access to downloads of IRB approved Global PWS Registry Protocol and Informed Consent documents, click here.
To receive monthly reports of data coming out of the Registry, please send an e-mail to email@example.com
For more information contact Theresa Strong, Ph.D., Director of Research programs (Theresa.strong at FPWR.org) and Jessica Bohonowych, Ph.D., Associate Director of Research programs (Jessica.bohonowych at FPWR.org)