Prader-Willi Syndrome
Conditions
Keywords
observational, non-interventional, Prader-Willi syndrome, PWS
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.
Interventions
None listed
Sponsors
Prader-Willi Syndrome Association USA
Foundation for Prader-Willi Research Canada
Prader-Willi Research Foundation Australia
Prader-Willi Syndrome Association
Foundation for Prader-Willi Research
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
5 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| 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 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 |
| 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 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 | Up to 4 years | Medical information will be from birth to before enrolling in the study |
| Evaluate PWS complexity using an online survey | Up to 4 years | — |
Countries
United States
Outcome results
None listed