Clinical trial NCT03718416: Natural History Study of Serious Medical Events in PWS
Official title: Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Prader-Willi Syndrome
Keywords: observational
non-interventional
Prader-Willi syndrome
PWS
Lead sponsor: Foundation for Prader-Willi Research
Sponsor class: OTHER
Collaborators: name: Prader-Willi Syndrome Association USA
name: Foundation for Prader-Willi Research Canada
name: Prader-Willi Research Foundation Australia
name: Prader-Willi Syndrome Association
First posted: 2018-10-24
Enrollment: 700
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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

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.

Eligibility criteria: 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

Primary outcomes:
measure: Evaluate the incidence of serious medical events
timeFrame: Up to 4 years
Secondary outcomes:
measure: Evaluate the incidence of non-serious thrombotic events
timeFrame: Up to 4 years
measure: Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample
timeFrame: Up to 4 years
measure: Evaluate prescription medication use associated with serious medical events and thrombotic events
timeFrame: Up to 4 years
measure: Evaluate the change in weight
timeFrame: Up to 4 years
measure: Evaluate the change in height
timeFrame: Up to 4 years
measure: Evaluate the change in height-adjusted weight
timeFrame: Up to 4 years
measure: Evaluate PWS complexity using an online survey
timeFrame: Up to 4 years
measure: Evaluate the pattern of hyperphagia behaviors using an online survey
timeFrame: Up to 4 years
measure: Evaluate hyperphagia management using an online survey
timeFrame: Up to 4 years
measure: Evaluate food-related behaviors using an online survey
timeFrame: Up to 4 years
measure: Analyze medical information to evaluate the natural history of PWS
timeFrame: Up to 4 years
Other outcomes:
Locations:
city: Walnut
country: United States
facility: Foundation for Prader-Willi Research (FPWR)
state: California
Study officials:
References: