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Completed

NCT Number: NCT03718416

Natural History Study of Serious Medical Events in PWS

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.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

5 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Foundation for Prader-Willi Research (FPWR)

Walnut, California, 91789, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. 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 outcomes

  1. 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

  2. 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

  3. Evaluate prescription medication use associated with serious medical events and thrombotic events

    Time frame: Up to 4 years

  4. Evaluate the change in weight

    Time frame: Up to 4 years

  5. Evaluate the change in height

    Time frame: Up to 4 years

  6. Evaluate the change in height-adjusted weight

    Time frame: Up to 4 years

  7. Evaluate PWS complexity using an online survey

    Time frame: Up to 4 years

  8. 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

  9. Evaluate hyperphagia management using an online survey

    Time frame: Up to 4 years

  10. Evaluate food-related behaviors using an online survey

    Time frame: Up to 4 years

  11. 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

Sponsors and collaborators

Lead sponsor

Foundation for Prader-Willi Research

Other

Collaborators

  • Foundation for Prader-Willi Research Canada
  • Prader-Willi Research Foundation Australia
  • Prader-Willi Syndrome Association
  • Prader-Willi Syndrome Association USA

Registry information

Official study title

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

Acronym: PATH for PWS

Important dates

Study start
2018
Primary completion
2024
Study completion
2024
First posted
Oct 24, 2018
Registry last updated
Feb 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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