Prader-Willi Syndrome Clinical Trial
— PATH for PWSOfficial title:
Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
Verified date | February 2024 |
Source | Foundation for Prader-Willi Research |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational [Patient Registry] |
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.
Status | Completed |
Enrollment | 700 |
Est. completion date | January 31, 2024 |
Est. primary completion date | January 31, 2024 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 5 Years and older |
Eligibility | 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 |
Country | Name | City | State |
---|---|---|---|
United States | Foundation for Prader-Willi Research (FPWR) | Walnut | California |
Lead Sponsor | Collaborator |
---|---|
Foundation for Prader-Willi Research | Foundation for Prader-Willi Research Canada, Prader-Willi Research Foundation Australia, Prader-Willi Syndrome Association NZ, Prader-Willi Syndrome Association of Australia, Prader-Willi Syndrome Association USA |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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 | 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 | |
Secondary | 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 | |
Secondary | Evaluate prescription medication use associated with serious medical events and thrombotic events | Up to 4 years | ||
Secondary | Evaluate the change in weight | Up to 4 years | ||
Secondary | Evaluate the change in height | Up to 4 years | ||
Secondary | Evaluate the change in height-adjusted weight | Up to 4 years | ||
Secondary | Evaluate PWS complexity using an online survey | Up to 4 years | ||
Secondary | 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 | |
Secondary | Evaluate hyperphagia management using an online survey | Up to 4 years | ||
Secondary | Evaluate food-related behaviors using an online survey | Up to 4 years | ||
Secondary | 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 |
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