Natural History Study of Serious Medical Events in PWS

Foundation for Prader-Willi Research700 enrolled

Overview

PATH for PWS is a study to help researchers better understand serious medical events in PWS over a 4-year period, as well as evaluate how PWS-related behaviors change over time. The data from this study is intended to inform the development and clinical trial design of potential new treatments.

Every 6 months, participants or their caregivers will be asked to update online surveys about medical problems and serious medical events, as well as provide information about conditions and behaviors often associated with PWS such as hyperphagia. Because this is an observational study, no study drug will be provided and no visits to a doctor or clinic are required.

Study Type

OBSERVATIONAL

Enrollment

700

Conditions

Prader-Willi Syndrome

Eligibility

Sex: ALLMin age: 5 Years

Medical Language ↔ Plain English

Inclusion Criteria: Participants must meet all of the following criteria: * Confirmed diagnosis of PWS * At least 5 years of age * Live in the United States, Canada, Australia, or New Zealand * Must be enrolled or willing to enroll in the Global PWS Registry (https://pwsregistry.org) Participants or their caregiver must also meet all of the following criteria: * Have access to the internet to complete online surveys at least every 6 months * Agree to receive reminders to complete online surveys * Allow the staff to ask questions about survey responses if more information is needed and to enter data into the surveys Exclusion Criteria: Participants or their caregiver meeting any of the following criteria will be excluded: * Is not able to read and understand English

Locations (1)

Foundation for Prader-Willi Research (FPWR)

Walnut, California, United States

Outcomes

Primary Outcomes

Evaluate the incidence of serious medical events

Serious medical events are those that result in death, are life-threatening, require hospitalization or an emergency room visit, or are medically significant

Time frame: Up to 4 years

Secondary Outcomes

Evaluate the incidence of non-serious thrombotic events

Thrombotic events include blood clots in a blood vessel, such as an artery or vein

Time frame: Up to 4 years

Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample

D-dimer is a protein in the blood that is present when a blood clot is forming or has formed

Time frame: Up to 4 years

Evaluate prescription medication use associated with serious medical events and thrombotic events

Time frame: Up to 4 years

Evaluate the change in weight

Time frame: Up to 4 years

Evaluate the change in height

Time frame: Up to 4 years

Evaluate the change in height-adjusted weight

Time frame: Up to 4 years

Evaluate PWS complexity using an online survey

Time frame: Up to 4 years

Evaluate the pattern of hyperphagia behaviors using an online survey

Hyperphagia means the intense, constant hunger that often occurs in individuals with PWS

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.