Registry FAQ

A registry is a place to store detailed information about individuals and family members who are affected by a specific disease, syndrome or group of diseases. In this case, the PSC Partners Patient Registry is for individuals affected by PSC. When you join the registry, you will create your own personal health record. Your personal record, combined with those of many other people with PSC, creates a registry that meets several critical needs.

1. The Registry is a place where you can collect and store information about your PSC, including medical history and information about other affected family members. You can track information that helps you work with your doctor and other healthcare providers to facilitate management of your personal health.

2. It is a place where you can include information about your health, including PSC diagnosis, test results (possibly genetic testing in the future), and information on the progress of your disease, so you have access to the most accurate and up-to-date information.

3. It will enable PSC Partners Seeking a Cure to provide you with the most up-to-date information about research that may be of specific interest to you and other people with PSC, including clinical trials to evaluate possible new treatments.

4. Researchers studying PSC need accurate information from many people to understand how PSC affects people, information only you and your doctor can provide.

5. Researchers who are ready to start research studies or clinical trials can use de-identified information in the registry to help the Registry staff identify people who may be eligible to participate in studies. The researcher gives the Registry staff their criteria, and the staff identifies those who may be eligible to participate. A member of the PSC Partners’ Registry team will then contact you and other individuals who may be a match and will give you the investigator’s contact information. You will have the choice to contact the investigator or not. Scientists cannot contact you directly, as they only have access to de-identified data.

The online patient-driven PSC Partners Patient Registry was launched in 2014, as part of an NIH ORDR rare disease registry pilot project. Currently the Registry houses data from over 2,600 people with PSC. Its objectives are to identify PSC patients, collect longitudinal prospective data, facilitate clinical trial recruitment, expedite research, assess treatments (when we have treatments), and identify unmet patient needs.

PSC patients from all over the world join the Registry.

Primary Sclerosing Cholangitis (PSC) is a poorly understood disease for which there are currently no approved therapies proven to be effective in slowing or stopping progression of the disease. PSC has no known cure. The disease is rare, which makes it difficult to gather information and develop treatments. The Registry will collect information from PSC patients all over the world to help researchers have a better understanding of PSC. The rarity of the disease makes it difficult to conduct large clinical trials. That is why PSC Partners has developed a patient registry to collect detailed medical information on PSC patients: to help discover possible factors contributing to disease causes and progression; and to help find and evaluate new therapies.

The purpose of the PSC Partners Patient Registry is to collect and store medical and other information from individuals with PSC. Information from the Registry will be used to better understand PSC and to develop new treatments. This information will be helpful to understand how PSC affects those living with the disease. The Registry may lead to studies that help to better understand the natural history (progression) of PSC and/or environmental factors that may contribute to the disease. The Registry may also be used by the PSC Partners’ Registry team to contact patients about research studies or clinical trials for which they may be eligible, including those investigating new treatments.

The Registry also collects data using existing and widely used Patient-Reported Outcome (PRO) tools (surveys) that measure how you feel and function with PSC. You may also be asked to evaluate such surveys to understand whether PRO surveys accurately describe your PSC experience. The results of such surveys may help researchers design research projects that include how you feel and function with PSC.

The Registry’s main survey, the Clinical Survey, collects demographic data, diagnosis, family history, symptoms, quality of life, overlapping diseases, surgeries, and medications. The questions were created with NIH ORDR while the PSC Partners Registry was involved in their pilot program.

From March 2020 to December 2024, the Registry was collecting data on COVID.

SF-36, a non-disease-specific quality-of-life validated tool was recently added. Research-specific surveys, such as COVID-response in Registry participants, have been added as a section of the Clinical Survey. A survey for the Patient-Focused Drug Development meeting with the FDA was implemented to identify the challenges and obstacles faced by PSC patients in joining clinical trials. This survey is not being served to Registry participants at this time.

A Health Equity survey has been added to understand the PSC community’s access to care.

The data is collected from individuals who have a diagnosis of PSC, including data on those who have passed away.

Each is approved by the North Star Review Board before being served to registry participants.

The Registry contains patient-reported outcomes and requires a proof of diagnosis the participant uploads to their file. Diagnosis must be confirmed by a doctor’s report or an MRI report.

The data is collected globally from individuals who have a diagnosis of PSC and those who have passed away. In the case of those affected by PSC, the diagnosis must be confirmed by an MRCP, doctor’s report, or other PSC diagnostic test results.

Learn more about the differences between WIND-PSC and the Registry by visiting this page.