Natural History Study of Serious Medical Events in PWS

NCT03718416 | Completed

• 1 other identifier: PATH-PWS-001
• Study Type: observational
• Target: 700
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Keywords

observational; non-interventional; Prader-Willi syndrome; PWS

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (11)

• Evaluate the incidence of non-serious thrombotic events

  Up to 4 years

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

  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

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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

You may not qualify if:

• Participants or their caregiver meeting any of the following criteria will be excluded:
• Is not able to read and understand English

Sponsors & Collaborators

• Foundation for Prader-Willi Research: lead
• Prader-Willi Syndrome Association USA: collaborator
• Foundation for Prader-Willi Research Canada: collaborator
• Prader-Willi Research Foundation Australia: collaborator
• Prader-Willi Syndrome Association: collaborator

Study Sites (1)

Foundation for Prader-Willi Research (FPWR)

Walnut, California, 91789, United States

Location

Related Publications (1)

• Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.

  PMID: 38671361 DERIVED

Related Links

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

MeSH Terms

Conditions

Prader-Willi Syndrome

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Target Duration: 4 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 12, 2018
• First Posted: October 24, 2018
• Study Start: September 28, 2018
• Primary Completion: January 31, 2024
• Study Completion: January 31, 2024
• Last Updated: February 29, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will share
• Access Criteria: Data access will be governed by the Global PWS Registry advisory board.

Locations

US (1)