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Natural History Study of Serious Medical Events in PWS (PATH for PWS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03718416
Recruitment Status : Active, not recruiting
First Posted : October 24, 2018
Last Update Posted : February 3, 2021
Sponsor:
Collaborators:
Prader-Willi Syndrome Association USA
Foundation for Prader-Willi Research Canada
Prader-Willi Research Foundation Australia
Prader-Willi Syndrome Association of Australia
Prader-Willi Syndrome Association NZ
Information provided by (Responsible Party):
Foundation for Prader-Willi Research

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.

Condition or disease
Prader-Willi Syndrome

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.

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Study Type : Observational [Patient Registry]
Actual Enrollment : 700 participants
Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration: 4 Years
Official Title: Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
Actual Study Start Date : September 28, 2018
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : October 2022

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  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 Outcome Measures :
  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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03718416


Locations
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United States, California
Foundation for Prader-Willi Research (FPWR)
Walnut, California, United States, 91789
Sponsors and Collaborators
Foundation for Prader-Willi Research
Prader-Willi Syndrome Association USA
Foundation for Prader-Willi Research Canada
Prader-Willi Research Foundation Australia
Prader-Willi Syndrome Association of Australia
Prader-Willi Syndrome Association NZ
Additional Information:
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Responsible Party: Foundation for Prader-Willi Research
ClinicalTrials.gov Identifier: NCT03718416    
Other Study ID Numbers: PATH-PWS-001
First Posted: October 24, 2018    Key Record Dates
Last Update Posted: February 3, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: FPWR allows for registry use by request and releases de-identified data to the public.
Access Criteria: Data access will be governed by the Global PWS Registry advisory board.

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Foundation for Prader-Willi Research:
observational
non-interventional
Prader-Willi syndrome
PWS
Additional relevant MeSH terms:
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Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders