Natural History Study of Serious Medical Events in PWS (PATH for PWS)

Foundation for Prader-Willi Research

Status: Completed

Conditions: Prader-Willi Syndrome

Study type: Observational

Funder types: Other

Identifiers

NCT03718416

PATH-PWS-001

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 700 patients

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

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