Natural History Study of Serious Medical Events in PWS

Prader-Willi Syndrome Clinical Trial

— PATH for PWS  

Official title:

Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03718416
• Other study ID #: PATH-PWS-001
• Status: Completed
• Start date: September 28, 2018
• Est. completion date: January 31, 2024

Study information

• Verified date: February 2024
• Source: Foundation for Prader-Willi Research
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• 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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Locations

Country	Name	City	State
United States	Foundation for Prader-Willi Research (FPWR)	Walnut	California

Sponsors (6)

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

Country where clinical trial is conducted

United States, 

Outcome

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

External Links

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