Study Information

Natural history studies track the course of disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes. Thorough understanding of disease natural history is the foundation upon which a clinical development program for drugs, biologics, medical foods or medical devices is built.¹

¹U.S. FDA. “Information on the Orphan Products Natural History Grants Program.” FDA.gov. https://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/OrphanProductsNaturalHistoryGrantsProgram/ucm487336.htm (accessed July 18, 2018).

For questions or more information, please contact: [email protected]

The PATH for PWS study builds on the power of the Global PWS Registry to advance clinical trials. It also:

• Will help us understand the medical complications individuals with PWS experience, and what factors increase or decrease risk
• Paves the way for greater understanding of potential new treatments for PWS and informs new drug development
• Will help us understand and track the changes of PWS over time

At enrollment and every 6 months, caregivers of those enrolled in PATH for PWS will be asked to provide an update on any medical issues, serious medical events, and related medical procedures or prescriptions, as well as provide information about conditions often associated with PWS such as hyperphagia. Clinic visits will not be required, as the information will be provided through internet-based surveys. The data will be analyzed to evaluate the incidence of serious medical events, prescription medication use associated with medical events of interest, patterns of hyperphagia-related behaviors, as well as additional areas of importance to the PWS community such as changes in body weight, management strategies for hyperphagia and characterization of traits and actions common to individuals with PWS. The analyses will also help FPWR and others in the Prader-Willi research community identify potential areas for future study.

More information is available in the consent form on our Resources page.

• Required hospitalization
• Required an emergency room (ER) visit
• Were considered life-threatening
• Were considered medically significant, such as blood clots, pneumonia, major infections, psychosis, significant self-injury, extreme aggression, suicidal thoughts, seizures, extreme food consumption in a short amount of time, severe edema

For those enrolled in the PATH for PWS study, information to be captured as soon as possible after each serious medical event includes:

• Date of the event
• Description of the event including clinical symptoms, contributing factors or events
• Medication changes
• Medical records such as lab reports, imaging reports, clinician’s notes, and discharge summary

Participants will be asked to enter information through internet-based surveys in an online database at study entry and every 6 months. Clinic visits are not required.

The initial surveys required are expected to take approximately 1 to 2 hours overall to complete. These surveys should be completed as soon as possible after entering the study, but they do not need to be completed all at once. You can save partially completed surveys online and come back later to complete and submit the surveys. The follow-up surveys given every 6 months are expected to take approximately 30 minutes overall to complete. The time it takes to complete the surveys may depend on the amount of information, medical history, or serious medical events that a study participant has.

For the time and effort related to participation in the PATH for PWS study, participants will receive a $100 e-gift card for completing the initial surveys at study entry and a $50 e-gift card for completing the follow-up surveys every 6 months.

Participants can keep all their medical information in one place for easy future reference and see how their answers, behaviors and experiences compare to other respondents in the PWS community. An example of what these comparisons look like is available on our Resources page.

Another benefit? Participate from the comfort of home – no doctor visits are required.

Additional information and FAQs about the Global PWS Registry can be found here.