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. 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. 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. 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. Researchers studying PSC need accurate information from many people to understand how PSC affects people, information only you and your doctor can provide. 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 2,500 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. Since March 2020, COVID data are being collected. 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.

You have the right to know about the procedures, risks, and benefits of participating in the PSC Partners Patient Registry. To participate in this registry, you will need to give your consent after reading the document, “Understanding your Participation.” You will find this statement of consent at the end of the document. When you click on the statement affirming your participation and use of the information you are providing, you will have “consented.” If you decide to participate, you can change your mind later and leave the registry. Please take your time to make your decision and discuss it with your family, friends, and caregivers. You, the participant, may be invited to participate in a research study or clinical trial. Participation in a research study or clinical trial, if you qualify, will be based solely upon the voluntary consent of the patient to participate in the study. You may be contacted by a PSC Partners research staff member to schedule an interview with a trained clinician to assess the impact of PSC on your quality of life. Your participation is voluntary.

The participant may be invited to participate in a research study or clinical trial. Participation in a research study or clinical trial, if the participant qualifies, will be based solely upon the voluntary consent of the patient. If the participant agrees, they may be contacted by a research staff member to schedule an interview with a trained clinician to assess the impact of PSC on quality of life. The participant may be asked to provide the registry staff access to the participant’s electronic records - these records will be integrated into the survey data. These medical records may include electronic health records, photos, videos, audio recordings, data from wearables, self-reported health information/data, or survey data. At any time, the participant can opt out of sharing their medical records.

There is minimal risk in taking part in the registry. The registry includes questions that can be sensitive, and you may feel uncomfortable answering. You do not have to share any information you do not want to share. There may be risks to your privacy. The PSC Partners Patient Registry will store study records and other information about you in a secure location and will grant access only to registry personnel authorized by PSC Partners. Compliance to HIPAA and FISMA rules and standards and an annual review of the registry by an Institutional Review Board (IRB) serve to minimize risks to the patient. However, just like other personal information kept by your health care providers, your banks, and others, even these safeguards cannot guarantee absolute protection of the data.

Providing your information to the registry and participating in this registry are voluntary. Participation may not benefit you personally, medically, or financially. However, your participation may help all those with PSC by increasing the understanding of the disease. Collected data may help speed up research. Researchers may learn whether and how treatments work. Medical professionals may be able to improve how they treat the disease. Participants have the option to receive information about opportunities to participate in research and clinical trials.

Participating in this registry is your choice, is completely voluntary, and will not affect your healthcare. If you decide to participate in the registry, you can decide to stop at any time. Simply contact the registry coordinator at registrycoordinator@pscpartners.org, and all of your data will be removed from the PSC Partners Patient Registry. However, pre-authorized de-identified data cannot be retrieved from researchers that have already accessed it prior to your request for removal.

The goal of the PSC Partners Patient Registry is to share de-identified detailed medical and other information with scientists and other researchers while protecting your privacy. All the information you provide will be maintained in a safe (“secure”) HIPAA and Federal Information Security Management Act (FISMA) compliant environment. Any information that could identify you and your family members will not be shared without your approval. Your privacy will be protected by hiding the name, address and other “identifying” information from researchers. We call this information “de-identified” because all personal identifiers have been removed. Your personal information such as your name, address, or other information that identifies you or your family will be labeled with a code number, encrypted (coded), stored and protected with a password. Only authorized people who work on the registry will know the code and be able to identify you if needed. The PSC Partners Patient Registry is the guardian of the information contained within the registry. Your identifiable information will not be shared with anyone outside the registry. Approved scientists, researchers, and clinicians will be allowed to see only the de- identified data for their studies. Your identifiable information will not be shared with anyone outside the PSC Partners Patient Registry. Only authorized members of the PSC Partners Patient Registry team will have access to your Identifying information. At any time, you may request the PSC Partners Patient Registry coordinator to remove the de-identified data that has been collected in the registry.

PSC Partners will make every reasonable effort to assure that your personal information, in our possession, will be kept confidential. If information from the registry is published or presented at scientific meetings, your name and other personal information will NOT be used. However, we cannot guarantee total privacy. Your personal information may be released if ever required by law. The Food and Drug Administration (FDA) and North Star Review Board may also inspect the registry records. PSC Partners owns the registry data. This registry will be managed by Matrix (Across Healthcare), a company that manages other rare disease registries like the PSC Partners Patient Registry. Matrix strictly follows the rules and standards provided by the United States Health Insurance Portability and Accountability Act (HIPAA) and Federal Information Security Management Act (FISMA) and uses established industry standards for security. Matrix is hosted in a HIPAA and FISMA compliant infrastructure with dedicated firewalls and advanced intrusion detection to secure the participant's data. All registry network transmissions are encrypted for an added level of protection. In the unlikely event PSC Partners Seeking a Cure ceases to exist, all attempts will be made to find another suitable entity to take ownership of the registry. You will be asked to give your consent to the transfer of your data (identifiable and de-identified information). The data of those not consenting for such a transfer will be destroyed. If all attempts to find a suitable entity fail, then all data collected by the PSC Partners Patient Registry will be destroyed, thus protecting your privacy. You will be contacted and informed of this action. If you would like more detailed information regarding the security of the PSC Partners Patient Registry, please contact the registry coordinator at registrycoordinator@pscpartners.org.

We may assign a PALIND (Privacy-aware Linkable Identifier) to help link your data both within and outside of this study without sharing your identifying information. PALIND allows us to potentially gain more valuable insights from your data by linking with other datasets while maintaining privacy. Please note that linking of data will occur only with the approval of the PSC Partners Registry when it is scientifically important to the research. The use of PALIND gives enhanced privacy protection for you and gives the PSC Partners Registry visibility and control over when your data gets linked.

There is no cost for you to participate in this registry. Neither you nor your insurance carrier will be charged for participating in the registry.

You will not be paid for taking part in this registry. PSC Partners Seeking a Cure maintains ownership of the de-identified data collected and compiled by the registry and is the guardian of the information contained within the registry. You will have no property ownership or interest in such data derived from the registry and no right or entitlement in any research or research product using or derived from the data.

You, or your authorized representative, should finish reading the document, "Understanding Your Participation," and then decide if you want to participate in the registry. If you choose to participate, you, or your authorized representative, will need to complete and sign the “Informed Consent,” affirming that you understand the risks and benefits of participation, and that you agree to participate in the registry. The next step is to provide basic demographic information in order to create the account. Once you create an account, you will be able to access the registry surveys. If you require assistance to complete the surveys, please email registrycoordinator@pscpartners.org. In the future, the registry may be linked to a biobank which is a place that stores tissue, blood or other samples from patients. If a biobank is created for people with PSC, you may be asked to donate samples to the biobank. If you decide to donate your samples, you will be asked to provide separate consent for the biobank.

You can participate in this registry because you, the PSC patient, or the PSC patient you represent, has been diagnosed with PSC. Patients over the age of 18 who understand the consent form (and who do not have a legal guardian) are eligible to join the registry on their own. Otherwise, the legally authorized representative of an adult patient, legal guardian of a minor, or parent of the patient must give consent for the patient to join. When the patient becomes a legal adult, consent for continued participation will be obtained directly from the patient if they do not have a legally authorized representative. Those minors who choose not to re-enroll as adults will have their records deleted from the registry.

Yes, it is okay and important to join other registries. Your information will be used differently, for different purposes and by different researchers looking for different aspects of PSC. Your choice to participate or not to participate will not affect the care you are given by your physicians.

Although one of the main goals of the registry is to make it easier for PSC patients to participate in research, there is no guarantee that you will be eligible for a trial. Even if you are eligible for a trial based on the data you store in the registry, it is possible that you do not meet all the trial requirements. To participate in any trial, you will need to discuss the trial with the research staff and fill out a separate informed consent form specific to the trial. Informing you about the existence of a trial does not imply that PSC Partners endorses it. Before you consent to be in a clinical trial, you should discuss your participation with your physician.

Even if you do not want to take part in a clinical trial, your de-identified information will still be useful to researchers who are trying to learn more about patients with PSC. By joining the registry, you will be helping to better understand the geographical spread of the PSC population, the progression of disease, evaluation of treatments and tests, monitoring the safety of medications, and more.

PSC Partners Seeking a Cure makes no claims that information in the PSC Partners Patient Registry is appropriate or may be downloaded outside of the United States. Access to the registry may not be legal in certain countries or for certain persons. If you access the registry from outside of the United States, you do so at your own risk and are responsible for compliance with the laws of your jurisdiction regarding online conduct and acceptable content. User personal information ("Information") that is submitted to this registry will be collected, processed, stored, disclosed and disposed of in accordance with applicable U.S. law and the PSC Partners Patient Registry Privacy Policy. The PSC Partners Patient Registry is operated by Across Healthcare, a US corporation that manages other registries. PSC Partners Patient Registry is the sole owner of the data. Across Healthcare makes no representation that materials in the registry are appropriate or available for use in all locations worldwide. You are solely responsible for compliance with local laws, if and to the extent local laws are applicable. Access to the registry from jurisdictions where the contents of our registry are illegal or penalized is prohibited. Across Healthcare’s privacy policy (December 2023) states Australia, California, Canada, European Union, India, New Zealand, Singapore, and many other locations around the world have additional or different notifications, disclosures, and other applicable privacy rights and/or policies not covered specifically within this privacy policy. Matrix is committed to global participation in its Services. Please contact Matrix at privacy@acrossmatrix.com for specific questions, notices, compliance information, or other information related to your rights within your specific geographic region. Please visit https://acrossmatrix.com/ for more information on Across Healthcare’s policies. If you are a non-U.S. user, you acknowledge and agree that the PSC Partners Patient Registry may collect and use your de-identified information and disclose it to other entities outside your resident jurisdiction. In addition, such information may be stored on servers located outside your resident jurisdiction. US law may not provide the degree of protection for information that is available in other countries. By providing us with your de-identified Information, you acknowledge that you consent to the transfer of such information outside your resident jurisdiction to the US or other jurisdictions that may require different levels of protection. If you do not consent to such transfer, you may not use the registry.

If you have any questions about the registration process, about participation in the registry, or about reporting problems that result from your participation in the registry, please contact the registry coordinator at registrycoordinator@pscpartners.org. This research is being overseen by an Institutional Review Board (“IRB”). An IRB is a group of people who perform independent review of research studies. You may contact them at info@northstarreviewboard.org if: You have questions, concerns, or complaints that are not being answered by the research team. You are not getting answers from the research team. You cannot reach the research team. You want to talk to someone else about the research. You have questions about your rights as a research subject. As you complete the registry survey, if you have questions that you do not understand, first go to the Glossary, and if you have further questions, please contact the Registry Coordinator at registrycoordinator@pscpartners.org . For additional information regarding the terms and conditions of the registry website or the privacy policy, please go to Terms and Conditions and Privacy Policy. You will find the statement of consent at the end of the Informed Consent Form. By clicking to agree to participate on the “Informed Consent,” you do not give away any legal rights or benefits to which you are otherwise entitled.