Natural History Study of Serious Medical Events in PWS

Completed

• Conditions: Prader-Willi Syndrome

• Registration Number: NCT03718416
• Lead Sponsor: Foundation for Prader-Willi Research

Brief Summary

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.

Detailed 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.

Study Timeline

• Study Start: 2018-09-28
• Study First Submitted: 2018-10-12
• Study First Submitted QC: 2018-10-22
• Study First Posted: 2018-10-24
• Primary Completion: 2024-01-31
• Study Completion: 2024-01-31
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-28
• Last Update Posted: 2024-02-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 700

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluate the incidence of serious medical events	Up to 4 years	Serious medical events are those that result in death, are life-threatening, require hospitalization or an emergency room visit, or are medically significant

Secondary Outcome Measures

Name	Time	Method
Evaluate food-related behaviors using an online survey	Up to 4 years
Evaluate hyperphagia management using an online survey	Up to 4 years
Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample	Up to 4 years	D-dimer is a protein in the blood that is present when a blood clot is forming or has formed
Evaluate prescription medication use associated with serious medical events and thrombotic events	Up to 4 years
Analyze medical information to evaluate the natural history of PWS	Up to 4 years	Medical information will be from birth to before enrolling in the study
Evaluate the change in weight	Up to 4 years
Evaluate the change in height	Up to 4 years
Evaluate the change in height-adjusted weight	Up to 4 years
Evaluate PWS complexity using an online survey	Up to 4 years
Evaluate the pattern of hyperphagia behaviors using an online survey	Up to 4 years	Hyperphagia means the intense, constant hunger that often occurs in individuals with PWS
Evaluate the incidence of non-serious thrombotic events	Up to 4 years	Thrombotic events include blood clots in a blood vessel, such as an artery or vein

Trial Locations

Locations (1)

Foundation for Prader-Willi Research (FPWR); 🇺🇸 Walnut, California, United States