The PWS-Clinical Investigation Collaborative (PWS-CLIC) is a network of PWS clinical experts, formally established in 2021. The work of the PWS-CLIC is supported by FPWR. This network has expanded over the past two years and now includes 25 clinical sites in the United States and 3 clinical sites in Canada (see the list of participating clinical sites here.) The mission of the PWS-CLIC is to improve the quality of clinical research and medical care for people with PWS across the lifespan through collaborative investigation and research to support evidence-based care.
One of the ways that the PWS-CLIC is working toward their mission of improving the quality of clinical research is to learn from every patient so that care can be improved for all. By better understanding the “natural history” of PWS, or the normal variation in symptoms and clinical challenges of PWS across the lifespan, health care professionals can anticipate possible problems and work to reduce the incidence and severity of clinical concerns.
To do this effectively, the clinicians need to be able to ‘compare notes’, look for data trends, and understand the true incidence of certain complications associated with PWS across a large, representative PWS population. To accomplish this goal, the PWS-CLIC, along with FPWR, has created and launched the PWS-CLIC Studytrax Database. This database is a place for clinicians to enter de-identified (i.e., with all identifying information like names and birthdates removed) clinical data to track participants with PWS over time. This database will complement the caregiver-entered data in the Global PWS Registry. The Studytrax system is a cloud-based electronic data capture system chosen for its ease of use and robust security features.
A working group of PWS-CLIC members worked together to define a set of standardized data elements that represent the most common clinical features and clinical concerns associated with PWS. There are currently 16 different forms of Studytrax data that measure data from many topic areas, including developmental milestones, behavior, diet and exercise, endocrine, orthopedics, sleep, medications, clinical labs and procedures, and more. By pooling this clinical data and tracking outcomes over time, clinicians can develop a better idea of how to effectively treat the many medical concerns associated with PWS. By complementing this information with the caregiver-reported data in the Global PWS Registry, our community has a powerful way to capture a complete picture of issues that impact both medical management and quality of life for families. We hope that families will participate in the Studytrax database and will opt to link that information with their Global PWS Registry record.
Another benefit to the PWS-CLIC Studytrax database is the ability to recruit a more representative population of people with PWS and increase the diversity of participants active in PWS research, clinical care, and clinical trials. The members of the PWS-CLIC can recruit their patients directly for this study, and since they will enter the data, participation does not rely on having access to online recruitment or having English as a first language. The consent forms have started being translated into different languages (including Spanish, Chinese, and Tigrinya) to decrease barriers to research participation.
Data on the first participants with PWS were entered into the PWS-CLIC Studytrax database in October 2023, with additional participants and sites continuing to come online and contribute their data over the coming year. Please ask your clinician if they are participating in this project or reach out to FPWR to learn more.
Over the next several months, we’ll be sharing more information about this powerful new tool to help document clinical aspects of PWS, answer important clinical questions, and inform the development of new assessments to support PWS research.
