< Back University of Minnesota Evaluation of an Oral Microbiota-based Therapeutic as a Treatment Option for PSC Early Phase 1 Study Previous Next

PSC Registry Overview for Researchers At a Glance 2,776 Participants from 55 countries 6,500 Completed surveys 23 Publications made possible through Registry data and recruitment as of Jan 2026 Research Partnerships DATA SHARING Aggregate or de-identified individual-level Registry data can be provided for approved research. Additional questionnaires can be sent to participants if needed. STUDY RECRUITMENT The Registry emails info on clinical trials and other studies to patients matching the criteria. For confidentiality, it is up to the patient to contact the investigators. For more details, read the Registry's Data Access Policy . CONTACT US Patient Voices in Action Source: Stephen Rossi et al. "Defining Acute Cholangitis as a Clinical Outcomes Endpoint in Adults with Primary Sclerosing Cholangitis" Source: Michael Li et al. "The Externally-Led Patient-Focused Drug Development Meeting for Primary Sclerosing Cholangitis: Insights on Patient Involvement in Clinical Trials" Current Registry Surveys To date, de-identified survey data has been used in 19 publications. Links and more information can be found on the Registry Impact page. Clinical Survey: This annual survey collects core medical information, family history, current symptoms, medications, and more. With 11 years of data, and this provides a rich data set for analysis, and is used to pre-screen for clinical trial recruitment messages. Health Equity in PSC: This survey collects additional demographic data and information about access to medical care. It also measures patients' confidence in and satisfaction with their care. Acute Cholangitis: Patient history, symptom course, and care received during most recent flare/cholangitis episode are collected in this survey. PSC & Cholangiocarcinoma (CCA): This survey elicits patient experience surrounding monitoring for CCA, awareness of and education about CCA, and discussion with their clinicians about CCA. Symptom Assessment: This survey is part of the Symptom Assessment Project, a PSC Partners initiative to develop fatigue, cognitive impairment, and liver pain PRO measures for potential use as clinical trial endpoints. The goal of this survey is to support validation of the PRO instruments in a real-world cohort. Past surveys include the Our Voices Survey, the results of which were presented to the FDA at the Patient-Focused Drug Development Forum for PSC; the PSC-specific COVID-19 Survey; the Oral Vancomycin and Insurance Survey, which helped inform a legislative advocacy effort; and the external RAND Health SF-36 quality-of-life measure. Clinicians help the Registry grow! Patients often learn of the Registry through their doctor. It is an easy and secure way for all with PSC to help advance PSC research, whether pre- or post-transplant. Caregivers can join on behalf of children or adults with PSC (adults must consent). We encourage you to discuss the Registry with your patients. Informational materials including brochures, flyers, and posters are available upon request.

< Back 2021. FINDINGS FROM THE PATIENT-FOCUSED DRUG DEVELOPMENT FORUM ON PRIMARY SCLEROSING CHOLANGITIS AASLD The Liver Meeting Poster #630 Conclusions: Findings from the Our Voices survey informed the development of the externally-led PFDD Forum for PSC and highlight the need for a more robust, validated Patient Reported Outcome Measure (PROM) for PSC. Overall, our results demonstrate the willingness of the PSC patient community to share experiences and guide the development of PROMs, which requires further perspective and expertise from the PSC research community. Previous Next

The VIADUCT Newsletter Join our Email List! The Viaduct - Issue 10, Winter 2026 Dear Registry Community, 2025 has been a banner year for the Patient Registry. Between important new surveys, Registry-led publications, external research support, and the launch of Spanish and French translations, the Registry is reaching ever greater heights. At the heart of the Registry’s success, making everything we do possible, is your active participation in the Registry. With 223 new participants in 2025, the Registry now represents over 2,770 people living with PSC f Feb 10 The Viaduct - Issue 9, Fall 2025 Living with PSC, Leading with Hope You might notice this issue of The Viaduct has a new look, in more ways than one! As your new Registry director, it is my honor to serve you in fulfilling the Registry’s mission to facilitate, expedite, and advance patient-centered research towards treatments and a cure for PSC. When I was diagnosed with PSC, I felt the same as many of you did: hopeless, uncertain, fearful. As I pored over medical websites, papers, and personal stories, two Sep 23, 2025 The VIADUCT - Issue 8 Issue 8 - Spring 2025 In this issue: From the Registry Director • Cholangiocarcinoma (CCA) Survey • WIND-PSC Enrolling Patients • Clinical Trial News • External Studies and Surveys • Symptom Assessment Project • Registry Superstars • Learning from Registry Data From the Registry Director Rachel Gomel (left) and Mary P Vyas (right) Dear Registry Community, Sixteen years have gone by since the PSC Partners Patient Registry was just a lofty idea, a dream I believed could turn in Apr 9, 2025 The VIADUCT Newsletter Issue 7 Issue 7 - Winter 2025 In this issue: From the Registry Director • A New Era • Your Data in Publication - 2024 • Clinical Trial News • Registry Data Presented at TLM • Your Powerful Impact • PSC Partners Registry and Other Registries From the Registry Director Dear Registry Participants, This has been an incredibly fruitful year for the Registry. Between a move to the new and improved Registry platform, several publications making use of Registry data, and the launch of two ne Dec 28, 2024 The VIADUCT Newsletter Issue 6 Issue 6 - June 2023 In this issue: From the Registry Director • New CZI Grant Awarded to PSC Partners • Patient Focus Groups Provide Feedback • Oral Vancomycin & Insurance Coverage • European Clinicians Survey Results • Call to Action: What is the Registry Not Asking? • Highlights: Clinical Trials & Studies From the Registry Director Dear Registry Participants, PSC Partners and the PSC Partners Patient Registry are moving at rapid speed through involvement in large internatio Jun 29, 2023 The VIADUCT Newsletter Issue 5 Issue 5 - October 2022 Welcome! In this issue: • From the Registry Director • The Clinical Trial Landscape in PSC • Current International PSC Research • The ChiLDRen Study • Gilead's PRIMUS Trial Terminated • Patient-friendly Clinical Trial Design • Highlights of the 2022 International Liver Congress • Meet PSCP's Ruth-Anne Pai, PhD • Clinical Trials Are Recruiting From the Registry Director Dear Registry Participants, Whether you are a person living with PSC, or the relat Oct 29, 2022 The VIADUCT Newsletter Issue 4 In this issue: • From the Registry Director • Mini-PFDDs • Patient Registry Video Tour • Help Us Grow & Diversify the Registry! • PSC Partners WIND Natural History Project • ROADMAP Wrap-up • PSC Research: Collaborative Efforts From the Registry Director Dear Registry Participants, Excitement is mounting as we approach the 18th PSC Partners Annual Conference, which is being planned in collaboration with Brigham and Women's and Massachusetts General hospitals (Harvard Univers May 24, 2022 The VIADUCT Newsletter Issue 3 Welcome! In this issue: • From the Registry Director • Profile: Dr. Frank Anania of the FDA • Your Registry Data at Work • PSC Partners Poster Presentation: AASLD Liver Meeting, Nov. 2021 • Three Clinical Trials Currently Recruiting • Three Minutes with Frank Sasinowski • AASLD Patient Track Recap • Update on the ROADMAP Initiative From the Registry Director Dear Registry Participant, The holidays are here and, once again, COVID-19 is affecting our lives in many different Dec 1, 2021 The VIADUCT Newsletter Issue 2 Dear Registry Participant, Thank you for your participation in the PSC Partners Patient Registry. We are happy to bring the second issue of The VIADUCT to you! Email us at any time with your feedback, input or questions at registrycoordinator@pscpartners.org . In this issue: From the Registry Director: Moving Research Forward Clinical Trials: Stay informed with Registry Resources | Fred Sabernick Shares His First-Hand Experience Boston: An Emerging Hub for PSC Research Home Aug 23, 2021

< Back 2025. AASLD The Liver Meeting Abstract and Poster: 4540: "IT'S MORE LIKE A DULL, ACHY PAIN THAT'S JUST CONSTANT": LIVER PAIN EXPERIENCES IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS (PSC) TO INFORM MEASURE DEVELOPMENT Authors: Donna Evon, Lauren Wright, Laura Mkumba, Nicole Lucas, Kaya Merkler, Ricky Safer, Joanne Hatchett, Rachel Gomel, Stephen Rossi, Mark Swain, Sasha Deutsch-Link, Bryce Reeve Abstract: Background: An in-depth understanding of experiences with liver pain associated with chronic liver disease is virtually non-existent, especially for individuals living with PSC. The development of a PSC-specific liver pain measure suitable for clinical trials relies upon understanding key characteristics of liver pain in this population. Methods: Adults with PSC were recruited through the PSC Partners Patient Registry and hepatology experts in the U.S. Participants were phone screened for the presence of multiple PSC-related symptoms. Purposeful sampling was used to recruit a representative sample of 17 adults for in-depth qualitative interviews about key PSC symptoms experienced in the last month, including liver pain. Interviews were conducted virtually, audio-recorded, transcribed, and analyzed using NVivo software. Results: Ten out of 17 participants who reported liver pain as a prominent PSC symptom described their experiences in detail, while another three spontaneously mentioned liver pain during their interviews. Participants were, on average 43 years old (range: 27-65), 50% male, 60% white, 30% black, 10% Asian and 30% had inflammatory bowel disease (IBD). Participants’ experiences of their liver pain are found in the Table below. Participants described the location of PSC-associated liver pain generally in the right upper quadrant area, but said it can extend around the side and back. Eleven of 13 described liver pain as “stabbing” or “sharp.” Eight described their pain as “achy” or “dull.” The sharp/stabbing pain is typically brief, while the achy/dull pain is more persistent. All but one participant used the word “pain” to describe their sensations. Liver pain can “come and go” intermittently, happens at random, and seems unassociated with specific triggers. Some reported pain daily, others weekly, and most reported pain occurring outside of cholangitis attacks. Participants were able to differentiate PSC-related liver pain versus abdominal pain from other causes (including IBD) based on location and type of pain, timing, response to medications, and laboratory/clinical markers. Liver pain interfered with sleep and caused anxiety, but relative to fatigue and brain fog, it appears to interfere less with daily functioning. Participants tended to “ push through it.” Conclusion: Dull/achy and sharp/stabbing liver pain may be more frequent and severe in symptomatic adults with PSC than previously recognized. These in-depth qualitative findings will inform the development of a de novo PSC-specific liver pain measure, inclusive of heterogeneity of experiences, and suitable for future PSC research studies. Previous Next

Learn more about the Registry's policies and procedures for researchers to obtain the approved types of de-identified registry data and/or confidentially send recruitment trial requests to eligible Registry participants. Data Access Policy Overview Introduction PSC Partners Seeking a Cure (PSC Partners) is committed to providing investigators and industry an opportunity to access the PSC Partners Patient Registry (Registry) data, and to assist in study recruitment while maintaining Registry participant privacy. Qualified non-profit investigators will be accorded Registry data without charge, while industry will be required to pay PSC Partners a license fee for the data to assist in covering some of the costs of maintaining the registry. In making data available to investigators, PSC Partners is bound by its responsibility to guard the confidentiality of registry participants. This policy provides information and guidelines for investigators and industry sponsors wishing to request access to de-identified patient data residing in the Registry. This policy requires that Registry data use remain ethical, purposeful and consistent with each original study proposal and request to access data. Levels of Data Four (4) levels of data can be requested by investigators and approved by the Registry Committee: Graphs of de-identified aggregate data. This data can be requested from the Registry Committee by emailing registryresearch@pscpartners.org . Case-by-case de-identified data . This level of data requires a project proposal, IRB approval or exemption and the approval of the Registry Director and the Registry Committee Chair. To be delivered on a password-protected spreadsheet or .csv file. Request for additional patient data through emailed questionnaire. This level of data requires a project proposal, IRB approval or exemption and the approval of the Registry Committee. [This is when the shared Registry data is insufficient and the investigator requests the implementation of their own survey.] Recruitment of registrants for clinical trials. Request to access individual subjects requires a project proposal, IRB approval and the approval of the Registry Committee. The Registry curator can perform queries based on the inclusion/exclusion criteria of a study and send potential subjects information about a study for which they may qualify. The Registry may provide the subject/caregiver a recruitment flier, informed consent form, and/or contact information for the site(s) conducting the study. Personal identifiers or subject contact information will not be provided to investigators. It is the responsibility of the consenting registry participants to contact the investigators. Requestor's Responsibilities Regarding Data Use The specific purpose for the data use must be agreed to in writing by the investigator and his/her employer institution (“Institution”) or the industry sponsor requesting the material and the selected representatives of the Registry Committee including medical and patient representatives (Registry Committee) for the approval of the request. The investigator and Investigator’s Institution or industry sponsor (if any) must sign this Data Use Agreement, agreeing to abide by the Researcher Data Access Policy. The investigator must provide an electronic copy of an IRB approval, or for surveys, IRB exemption (see Levels of Data below), a study proposal including the specific aims of the study/clinical trial, and the name and qualifications of the research center. The rationale for selection of the population inclusion and exclusion criteria must be described in the project proposal. Other information may be required as requested by the Registry Director. The investigator and investigator’s Institution or industry sponsor (if any) must agree in writing that the data will be used only for the stated purpose and only by the requesting party and may not be passed on or shared with a third party. (a) This Data Use Agreement is not transferable. (b) Data will not be used to establish the individual identities of any of the subjects from whom data is obtained. (c) Investigator, team, Institution and industry sponsors, if applicable, employees, and other research staff members must agree not to transfer data to any third party entity or any individual. The investigator, Institution and industry sponsor (if any) must agree that they will not sell or distribute the data and will only share the data with research staff members. The full team of individuals involved in the study are subject to the terms of the Data Use Agreement. The data may be used only for the time period specified in the approved request. Approval for access to the Registry data will be for an interval of no more than one (1) year from the date of approval, and Investigators must re-apply and submit a progress report to be approved by the Registry Committee in order to continue to use Registry data to recruit, or to continue to use the data for an approved study. The impact of research on people living with PSC is to be a key consideration for each project. Best ethical practices for ensuring the interest of the patient are to be applied. Any publication, oral and written presentations, or disclosures resulting from any and all analyses of the Registry data or based on the Registry data shall be subject to prior review by the Registry Director, must specify PSC Partners as the source of data, and are restricted to the scope of the approved study proposal. A copy of the final publication(s) must be sent to the Registry Director within 30 days of publication. Investigators, their Institution and industry sponsors accessing and using patient data must agree in writing to assume full responsibility for any and all uses of such data. Recipients of Registry data agree not to use it in advertising, publicity, or for purposes other than the purposes for which access to Registry data was granted, and not to use the logo, symbol, or other image of PSC Partners without its prior written consent. Steps to Request Access to Data An investigator and industry sponsor will be permitted access to Registry data only after meeting the following requirements: Contact the Registry via email at registryresearch@pscpartners.org . The Registry Team will provide a Data Access Form and Data Use Agreement. Complete the Data Access Form confirming that the investigator and investigator’s Institution or industry sponsor and research staff will abide by the Data Access Policy. Provide a project proposal, including: (a) Study proposal including the specific aims of the study (b) Name and qualifications of the investigator(s); include the full research and team of all individuals involved in this study with their qualifications. (c) Research center (d) In the case of a clinical trial: (i) Description of the procedures that will protect the confidentiality and security of Registry participants (ii) Sample study recruitment letter (iii) Consent form (iv) Rationale for selection of the population inclusion and exclusion criteria should be described in the project proposal (e) Disclosure of financial conflict of interest (f) Description of the data being requested Provide IRB approval with the project proposal if the requested data involves de-identified case-by-case results, a request for additional de-identified data through an e-mailed questionnaire, and/or a request for participants for a clinical trial. Receive written approval of the Registry Committee if the proposal requests research access to the Registry data. Submit a declaration of interest (included in the Data Access Form and Data Use Agreement). This may be submitted following a positive review of the project proposal if so desired. In the case of surveys to all or selected Registry participants, coordinate with the Registry staff on both the timing and content of the email to be sent to Registry participants informing them of the opportunity to participate in the Registry Committee-approved survey. If you have any questions r egarding these policies or regarding t he procedure to request access to Registry data, please contact us at registryresearch@pscpartners.org .

< Back Brigham & Women’s Hospital and Harvard University Diet in PSC (DOCK Survey) Pilot Study Previous Next

< Back Mayo Clinic Vancomycin for Primary Sclerosing Cholangitis - The purpose is to assess the effects of different oral vancomycin doses on the clinical and biochemical course in adult patients with PSC. Phase 2 Study Previous Next

< Back Mirum Pharmaceuticals: VISTAS - A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by PSC 2022 Kowdley, K et al. Impact of pruritus in primary sclerosing cholangitis (PSC): a multinational survey. Journal of Hepatology, Vol 77 EASL ILC Abstracts. Please find Abstract # THU442 (on page 337 via online pagination; p. S312 of journal; or use cntrl-f THU442 to search the document for the abstract). Text available without subscription at 10.1016/S0168-8278(22)00991-6 Notes: A multinational survey (39 questions) of PSC patients (n=482) focused on the impact of itch, how itch severity affected quality of life (QoL), and therapeutic interventions used to address itch. Responses collected June 2020 - Feb 2021.

See the impact of the collective voice of our 2500+ Registry participants, including research and other projects that have been published using de-identified Registry data. Registry Impact 2700+ Registered Participants 328,000+ Survey Question Responses 700+ Healthcare Provider Listings Your Data in Publication The following publications resulted from access to your de-identified PSC Partners Registry data. 2025. Digestive Disease Week Abstract and Poster: DIVERSITY AND SOCIOECONOMIC STATUS OF PATIENTS WITH PRIMARY SCLEROSING CHOLANGITIS (PSC) AND ITS IMPACT ON HEALTHCARE RECEIVED, A SURVEY OF PSC PARTNERS PATIENT REGISTRY ► Read More 2025. AASLD The Liver Meeting: SYMPTOM PROFILES AMONG ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS IDENTIFIES LOW, MODERATE AND HIGH BURDENED GROUPS ► Read More 2025. AASLD The Liver Meeting Abstract and Poster: 4540: "IT'S MORE LIKE A DULL, ACHY PAIN THAT'S JUST CONSTANT": LIVER PAIN EXPERIENCES IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS (PSC) TO INFORM MEASURE DEVELOPMENT ► Read More 2025. AASLD The Liver Meeting Abstract and Poster: 4479: "THIS IS NOT NORMAL”; UNDERSTANDING COGNITIVE IMPAIRMENT THROUGH QUALITATIVE INTERVIEWS WITH ADULTS WITH PSC TO INFORM MEASURE DEVELOPMENT ► Read More 2025. AASLD The Liver Meeting Abstract and Poster: 4476: IDENTIFYING GAPS IN PSC PATIENT EDUCATION AND UNDERSTANDING OF CHOLANGIOCARCINOMA SCREENING AND SURVEILLANCE PRACTICES: RESULTS OF A MULTINATIONAL PATIENT SURVEY ► Read More 2024. The PSC Forum: Characterizing Acute Cholangitis Experiences: Further Results from the PSC Flare or Cholangitis Attack Patient Survey ► Read More 2024. Patient-focused drug development in primary sclerosing cholangitis: Insights on patient priorities and involvement in clinical trials ► Read More 2024. Caregiver-reported symptom burden and preferences for therapeutic goals in pediatric primary sclerosing cholangitis ► Read More 2024. AASLD Liver Meeting Abstract and Poster: 4346: DEFINING ACUTE CHOLANGITIS AS A CLINICAL OUTCOMES ENDPOINT IN ADULTS WITH PRIMARY SCLEROSING CHOLANGITIS: RESULTS OF A MULTINATIONAL PATIENT SURVEY TO DEVELOP A PATIENT-REPORTED OUTCOMES MEASURE. ► Read More 2024. AASLD Liver Meeting Abstract and Poster: 4333: UNDERSTANDING FATIGUE EXPERIENCES THROUGH QUALITATIVE INTERVIEWS WITH ADULTS LIVING WITH PRIMARY SCLEROSING CHOLANGITIS (PSC): AN ESSENTIAL FIRST STEP TOWARDS CUSTOMIZING A PSC-SPECIFIC PROMIS® MEASURE ► Read More 2023. AASLD Liver Meeting Abstract and Poster: 4640-C: CAREGIVER-REPORTED SYMPTOM BURDEN AND PREFERENCES FOR THERAPEUTIC GOALS IN PEDIATRIC PRIMARY SCLEROSING CHOLANGITIS ► Read More 2023. AASLD Liver Meeting Abstract and Poster: 4579-C: THE EXTERNALLY-LED PATIENT-FOCUSED DRUG DEVELOPMENT MEETING FOR PRIMARY SCLEROSING CHOLANGITIS: INSIGHTS ON PATIENT INVOLVEMENT IN CLINICAL TRIALS ► Read More 2023. AASLD Liver Meeting Abstract and Poster: 4577-C: SYMPTOMS OF PRIMARY SCLEROSING CHOLANGITIS (PSC) IN THE LAST MONTH: PRELIMINARY DATA FROM THE PSC-PARTNERS PATIENT REPORTED OUTCOME MEASURE PROJECT ► Read More 2023. AASLD Liver Meeting Abstract and Poster: 4558-C: NETWORK ANALYSIS OF SYMPTOMS EXPERIENCED BY PATIENTS WITH PRIMARY SCLEROSING CHOLANGITIS ► Read More 2023. AASLD Liver Meeting Abstract and Poster: 3784-C: CENTERING THE PATIENT VOICE IN MULTI-STAKEHOLDER ENGAGEMENT TO DEVELOP A STRATEGIC RESEARCH PLAN FOR THE PSC COMMUNITY ► Read More 2021. FINDINGS FROM THE PATIENT-FOCUSED DRUG DEVELOPMENT FORUM ON PRIMARY SCLEROSING CHOLANGITIS ► Read More 2019. Pregnancy Experiences in Women with Primary Sclerosing Cholangitis ► Read More 2019. Potential Association of Doxycycline With the Onset of Primary Sclerosing Cholangitis: A Case Series ► Read More 2019. Clinical Characteristics and Outcomes Reported by Patients with Primary Sclerosing Cholangitis Through an Online Registry ► Read More 2017. Development and validation of a primary sclerosing cholangitis–specific patient‐reported outcomes instrument: The PSC PRO ► Read More 2016. Medical Treatments in Patients with Primary Sclerosing Cholangitis Differ Between Europe and North America ► Read More 2016. Frequency of Familial Primary Sclerosing Cholangitis (PSC) and Inflammatory Bowel Disease: Results From the PSC Partners Patient Registry ► Read More 2015. Characterization Of A Patient Driven International Registry For Primary Sclerosing Cholangitis ► Read More Advocacy The Patient Registry enables advocacy on behalf of PSC patients. Clinical Trial and Stu dy Protocol: PSC Partners is collaborating with pharmaceutical companies planning and conducting clinical trials by adding your patient perspective to the trial protocol. We also collaborate with researchers on their academic study protocols. Communication of Trial and Study Opportunities with Patients: Through the Patient Registry, we are able to share clinical-trial and study information with the PSC community. Those participants who have elected to receive notification from the Registry will be contacted by the Registry if they may be eligible for a trial or study. Special Projects: Through the Patient Registry, PSC Partners is able to conduct special projects of importance to the patient community. 2022 Participation in multinational survey on pruritus in PSC. Results presented at International Liver Congress. ► Read More 2020 PFDD: Externally-led Patient Focused Drug Development Meeting on PSC with FDA ► Read More 2020 Diet in PSC Survey (DOCK) - Active Now ► Read More 2020 Diet in PSC Study (DINER) - Active Now ► Read More 2020 Covid & PSC Survey - Active and Ongoing ► Read More 2018 Presentation on PSC Databases at the PSC Forum Meeting 2 ► Read More 2018 Emerging Trends Conference: The Patient Perspective: Insights on Clinical Trial Participation and Retention from the PSC Partners Community ► Read More 2017 Registry Information Informs PSC & PBC "Call to Action" Education Campaign ► Read More 2015 Trial Design and Endpoints for Clinical Trials in Adults and Children with Primary Sclerosing Cholangitis: GREAT Workshop, AASLD-FDA Collaborative. Presentation of Registry Participants Survey Responses ► Read More History of Collaboration: The PSC Partners Patient Registry has worked, or is working with, to provide the patient voice and support recruitment support. Ipsen Pharmaceuticals Chemomab Therapeutics Mirum Pharmaceuticals Pliant Therapeutics Escient Pharmaceuticals HighTide Therapeutics Gilead Sciences CymaBay Therapeutics NGM Biopharmaceuticals Takeda Pharmaceuticals Intercept Pharmaceuticals Lumena Pharmaceuticals Shire Pharmaceuticals Durect Pharmaceuticals Tobira Therapeutics Conatus Pharmaceuticals GlaxoSmithKline (GSK) Patara Pharma Albireo Pharma Clinical Trials and Studies

How does WIND-PSC differ from the Registry? While both are developed, led, and sponsored by PSC Partners, the PSC Partners Patient Registry and the WIND-PSC study differ in the function they serve, the type of data and the way these data are captured. The Patient Registry is an ongoing, prospective patient-reported registry; patients complete core annual clinical surveys that cover their clinical history and PSC symptoms as well as periodic surveys that cover other areas of interest, such as health equity or topics related to current PSC Partners projects. De-identified data is shared with PSC Partners-vetted research teams. Registry participants receive newsletters and information about drug trials and studies they have the option to join. Patients can join the Registry from anywhere in the world; patients from 54 countries currently participate. The only requirements to participate in the Registry are having a PSC diagnosis and being able to log onto the secure online platform to complete the surveys. Data collected in the Registry facilitates, expedites, and advances PSC research. WIND-PSC is a patient- and clinician-reported observational study and includes systematic and regular collection of data from medical records, imaging data, hospitalization records, lab test results, and annual blood samples. Participants in WIND-PSC must be enrolled at a participating site and must meet eligibility criteria such as not having had a liver transplant or liver cancer. Study coordinators obtain data from annual patient visits and ongoing tests over a period of five years. At the same time, participants will be asked to complete patient-reported surveys online every three months. The systematic capture of these health data over time will create a database of PSC progression. WIND-PSC is a more structured study, data is monitored by a third party, and reported liver-related events (such as progression of fibrosis or acute cholangitis) are confirmed. The data gathered is intended to fulfill the requirements of the FDA to be used to support the design of PSC clinical trials and regulatory filings for new treatments; provide key biomarker data; and provide insight into patient-reported experiences. WIND-PSC will be recruiting patients from up to 20 medical centers in Europe and across North America. We hope you will consider joining the PSC Partners Patient Registry now and, if you are being treated close to one of the WIND-PSC study sites, you will consider joining that initiative as well. You can do both, as each serves different purposes. Both are helping accelerate PSC research in different ways! Read more about the WIND-PSC initiative on the PSC Partners website and on the National Institutes of Health Clinical Trials online database .

Help researchers worldwide unlock the mysteries of primary sclerosing cholangitis (PSC). Complete your profile and join PSC Partners Seeking a Cure in advancing PSC research towards a cure. Your participation is important. About Us The PSC Partners Patient Registry: The Registry was created with the guidance of the NIH Office of Rare Disease Research (ORDR) . In 2012, PSC Partners was one of the rare disease organizations selected by the ORDR to participate in their Global Rare Disease (Patient) Registry and Data Repository pilot program. This registry is the exclusive property of PSC Partners. Mission: The Registry's mission is to facilitate, expedite, and advance patient-centered research towards treatments and a cure for PSC, by educating, supporting, and engaging all PSC patients to participate in patient-reported and clinical research. The Registry aims to represent the global community, across all stages of disease, including all ages, post-transplant, and deceased; and to reach underserved communities. The PSC Partners Patient Registry Committee: The PSC Partners Patient Registry Committee ensures the efficient and responsible governance of the registry. Brian Thorsen, BA Registry Director Video Bio Chris Bowlus, MD Registry Scientific Advisor Chief of Gastroenterology Univ. of California, Davis About Dr. Bowlus Fernanda Quevedo, BSA Registry Assistant Director Rachel Gomel, MA Registry Founder and Advisor Sharon Nanz, BA Registry Coordinator Video Bio PSC Partners Seeking a Cure: PSC Partners Seeking a Cure is a US-based 501(c)3 nonprofit foundation created in 2005. The mission of PSC Partners is to drive research to identify treatments and a cure for primary sclerosing cholangitis (PSC) while providing education and support for those impacted by this rare disease. Please visit the PSC Partners website at www.pscpartners.org for more information about the organization, including research grants, patient conferences, and the PSC Partners online resources (education, newsletters, social media locations).