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

Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome

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.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome

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 Type: Observational
• Enrollment (Actual): 700

Contacts and Locations

Study Locations

• United States
  • California
    • Walnut, California, United States, 91789
      • Foundation for Prader-Willi Research (FPWR)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with PWS who are at least 5 years old and live in the United States, Canada, Australia, or New Zealand

Description

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 Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	Up to 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluate the incidence of non-serious thrombotic events	Thrombotic events include blood clots in a blood vessel, such as an artery or vein	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	Up to 4 years
Evaluate prescription medication use associated with serious medical events and thrombotic events		Up to 4 years
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	Hyperphagia means the intense, constant hunger that often occurs in individuals with PWS	Up to 4 years
Evaluate hyperphagia management using an online survey		Up to 4 years
Evaluate food-related behaviors using an online survey		Up to 4 years
Analyze medical information to evaluate the natural history of PWS	Medical information will be from birth to before enrolling in the study	Up to 4 years

Collaborators and Investigators

• Sponsor: Foundation for Prader-Willi Research
• Collaborators: Prader-Willi Syndrome Association USA; Foundation for Prader-Willi Research Canada; Prader-Willi Research Foundation Australia; Prader-Willi Syndrome Association of Australia; Prader-Willi Syndrome Association NZ

Publications and helpful links

Helpful Links

• Global Prader-Willi Syndrome Registry
• Foundation for Prader-Willi Research Website
• PATH for PWS / PATH-PWS-001 Study Website

Study record dates

Study Major Dates

• Study Start (Actual): September 28, 2018
• Primary Completion (Actual): January 31, 2024
• Study Completion (Actual): January 31, 2024

Study Registration Dates

• First Submitted: October 12, 2018
• First Submitted That Met QC Criteria: October 22, 2018
• First Posted (Actual): October 24, 2018

Study Record Updates

• Last Update Posted (Estimated): February 29, 2024
• Last Update Submitted That Met QC Criteria: February 28, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Prader-Willi syndrome; observational; PWS; non-interventional
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Overweight; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Syndrome; Prader-Willi Syndrome
• Other Study ID Numbers: PATH-PWS-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: FPWR allows for registry use by request and releases de-identified data to the public.
• IPD Sharing Access Criteria: Data access will be governed by the Global PWS Registry advisory board.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No