Natural History Study of Serious Medical Events in PWS

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.

Full Title of Study: “Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome”

Study Type

  • Study Type: Observational [Patient Registry]
  • Study Design
    • Time Perspective: Other
  • Study Primary Completion Date: January 31, 2024

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.

Clinical Trial Outcome Measures

Primary Measures

  • Evaluate the incidence of serious medical events
    • Time Frame: 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 Measures

  • Evaluate the incidence of non-serious thrombotic events
    • Time Frame: Up to 4 years
    • Thrombotic events include blood clots in a blood vessel, such as an artery or vein
  • Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample
    • Time Frame: 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
    • 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
    • Time Frame: Up to 4 years
    • Hyperphagia means the intense, constant hunger that often occurs in individuals with PWS
  • Evaluate hyperphagia management using an online survey
    • Time Frame: Up to 4 years
  • Evaluate food-related behaviors using an online survey
    • Time Frame: Up to 4 years
  • Analyze medical information to evaluate the natural history of PWS
    • Time Frame: Up to 4 years
    • Medical information will be from birth to before enrolling in the study

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 5 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Foundation for Prader-Willi Research
  • Collaborator
    • Prader-Willi Syndrome Association USA
  • Provider of Information About this Clinical Study
    • Sponsor

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