Prader-Willi SyndromePATH for PWSNCT03718416
Natural History Study of Serious Medical Events in PWS
An observational study of Prader-Willi Syndrome, run by Foundation for Prader-Willi Research at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Prader-Willi Syndrome
- How many people
- 700 participants
- Where
- 1 siteUnited States
- Study dates
- 28 Sep 2018to 31 Jan 2024
- How long will I take part?
- 4 Years
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with PWS who are at least 5 years old and live in the United States, Canada, Australia, or New Zealand
Who may be able to join
- 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:
Show all 9Show fewer
- 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
Who cannot join
- Participants or their caregiver meeting any of the following criteria will be excluded:
- Is not able to read and understand English
Trial locations and contacts
1 site in 1 country.
United States1 site
Foundation for Prader-Willi Research (FPWR)
Walnut, California, 91789
About this study
Official title: Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
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.
Read the 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.
- Conditions
- Prader-Willi Syndrome
- Keywords
- observational, non-interventional, Prader-Willi syndrome, PWS
- Medical subject headings
- Prader-Willi Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Up to 4 years
Secondary outcomes (11)
Evaluate the incidence of non-serious thrombotic events
Thrombotic events include blood clots in a blood vessel, such as an artery or vein
Measured: Up to 4 years
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
Measured: Up to 4 years
Evaluate prescription medication use associated with serious medical events and thrombotic events
Measured: Up to 4 years
Evaluate the change in weight
Measured: Up to 4 years
Evaluate the change in height
Measured: Up to 4 years
Evaluate the change in height-adjusted weight
Measured: Up to 4 years
Evaluate PWS complexity using an online survey
Measured: Up to 4 years
Evaluate the pattern of hyperphagia behaviors using an online survey
Hyperphagia means the intense, constant hunger that often occurs in individuals with PWS
Measured: Up to 4 years
Evaluate hyperphagia management using an online survey
Measured: Up to 4 years
Evaluate food-related behaviors using an online survey
Measured: Up to 4 years
Analyze medical information to evaluate the natural history of PWS
Medical information will be from birth to before enrolling in the study
Measured: Up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Oct 2018
- Study start28 Sep 2018
- Primary completion31 Jan 2024
- Study completion31 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 700 participants (actual)
- Target duration
- 4 Years
Sponsor and investigators
- Lead sponsor
- Foundation for Prader-Willi Research
- Sponsor type
- University, hospital or other
- Collaborators
- Prader-Willi Syndrome Association USA, Foundation for Prader-Willi Research Canada, Prader-Willi Research Foundation Australia, Prader-Willi Syndrome Association
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03718416
- Sponsor's protocol ID
- PATH-PWS-001
- Acronym
- PATH for PWS
- Status last verified
- Feb 2024
- First submitted
- 12 Oct 2018
- Last updated
- 29 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: FPWR allows for registry use by request and releases de-identified data to the public.
Publications
Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.
PubMed 38671361
Study at a glance
From the registry record, last updated by the sponsor on 29 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-Interventional, Observational, Natural History Study of Serious Medical Events in Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT03718416
- Acronym / sponsor ID
- PATH for PWS / PATH-PWS-001
- Conditions
- Prader-Willi Syndrome
- Keywords
- observational, non-interventional, Prader-Willi syndrome, PWS
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 700 participants (actual)
- Lead sponsor
- Foundation for Prader-Willi ResearchUniversity, hospital or other
- Collaborators
- Prader-Willi Syndrome Association USA, Foundation for Prader-Willi Research Canada, Prader-Willi Research Foundation Australia, Prader-Willi Syndrome Association
- Study start
- 28 Sep 2018 (actual)
- Primary completion
- 31 Jan 2024 (actual)
- Study completion
- 31 Jan 2024 (actual)
- First posted
- 24 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Other
- Target follow-up
- 4 Years
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with PWS who are at least 5 years old and live in the United States, Canada, Australia, or New Zealand
Key inclusion criteria (9 listed)
- 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)
- Have access to the internet to complete online surveys at least every 6 months
Key exclusion criteria (2 listed)
- Is not able to read and understand English
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
11 secondary outcomes
Evaluate the incidence of non-serious thrombotic events
Time frame: Up to 4 years
Analyze D-dimer concentrations in a subset of participants who agree to provide a blood sample
Time frame: Up to 4 years
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Feb 2024.
- Derived from this study: PubMed 38671361Strong TV, Miller JL, McCandless SE, Gevers E, Yanovski JA, Matesevac L, Bohonowych J, Ballal S, Yen K, Hirano P, Cowen NM, Bhatnagar A. Behavioral changes in patients with Prader-Willi syndrome receiving diazoxide choline extended-release tablets compared to the PATH for PWS natural history study. J Neurodev Disord. 2024 Apr 26;16(1):22. doi: 10.1186/s11689-024-09536-x.
- Global Prader-Willi Syndrome RegistryLink provided by the sponsor.
- Foundation for Prader-Willi Research WebsiteLink provided by the sponsor.
- PATH for PWS / PATH-PWS-001 Study WebsiteLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. FPWR allows for registry use by request and releases de-identified data to the public.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Prader-Willi Syndrome, run by Foundation for Prader-Willi Research at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the incidence of serious medical events (time frame: Up to 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03718416, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Feb 2024.