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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 : March 29, 2022
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

Tracking Information
First Submitted Date October 12, 2018
First Posted Date October 24, 2018
Last Update Posted Date March 29, 2022
Actual Study Start Date September 28, 2018
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 22, 2018)		 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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: October 22, 2018)
  • 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
  • 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
  • Evaluate prescription medication use associated with serious medical events and thrombotic events [ Time Frame: Up to 4 years ]
  • Evaluate the change in weight [ Time Frame: Up to 4 years ]
  • Evaluate the change in height [ Time Frame: Up to 4 years ]
  • Evaluate the change in height-adjusted weight [ Time Frame: Up to 4 years ]
  • Evaluate PWS complexity using an online survey [ Time Frame: Up to 4 years ]
  • 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
  • Evaluate hyperphagia management using an online survey [ Time Frame: Up to 4 years ]
  • Evaluate food-related behaviors using an online survey [ Time Frame: Up to 4 years ]
  • 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
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title 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
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.
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration 4 Years
Biospecimen Not Provided
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
Condition Prader-Willi Syndrome
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Active, not recruiting
Actual Enrollment
 (submitted: September 12, 2019)		 700
Original Estimated Enrollment
 (submitted: October 22, 2018)		 500
Estimated Study Completion Date October 2022
Estimated Primary Completion Date October 2022   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: All
Ages 5 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03718416
Other Study ID Numbers PATH-PWS-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
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.
Current Responsible Party Foundation for Prader-Willi Research
Original Responsible Party Zafgen, Inc.
Current Study Sponsor Foundation for Prader-Willi Research
Original Study Sponsor Zafgen, Inc.
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
Investigators Not Provided
PRS Account Foundation for Prader-Willi Research
Verification Date March 2022