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 from 55 countries. And with 2,083 surveys completed last year alone (more than double the previous year!), your voice is heard loud and clear. Your contributions to research are invaluable, and we will ensure that all the information you have shared will tangibly drive research forward. Looking ahead to 2026, we aim to further expand the Registry’s global reach and to bring the insights made possible by your data into new publications, which will be shared back with the community. You can also look forward to a new survey launching later this year to capture transplant and post-transplant experiences. On behalf of the Registry team, we wish you all good health and good care in 2026. May this year continue to bring us ever closer to a cure for PSC. - Brian Thorsen, Director, PSC Partners Patient Registry Meet our New Assistant Director Assistant Registry Director Fernanda Quevedo  joined the Patient Registry Team in early January, but she is not new to PSC research. Her work experience includes several years as the research program/project manager at the Resnek Family Center for PSC Research at Brigham and Women’s Hospital in Boston, Massachusetts, where she worked with Dr. Joshua Korzenik and his team. Now Available: Clinical Survey 2026 The new Clinical Survey 2026 was just released and is available in your Registry account. Have you ever wondered why the Registry updates this survey every year and why we request that you update it annually? New Language Support: Spanish and French We are excited to announce that key parts of the Registry’s internal data platform, including most current surveys, are now available in Spanish and French . In addition, the public website also includes newly translated sections. With these translations, the Registry improves its global access and representation for those with PSC. Cholangiocarcinoma (CCA) Survey Results In Spring 2025, the Registry conducted the PSC and Cholangiocarcinoma (CCA) Patient Survey. This survey collected information on current practices of monitoring for CCA (also known as bile duct cancer), educational resources used by patients, and communication between patients and providers regarding CCA. Results from the survey were presented at AASLD’s The Liver Meeting 2025. Stay Connected with PSC Partners Sign up to receive the information that matters most to you— from research and volunteer openings to our three e-newsletters:  The Beacon - new, scientific research newsletter providing a comprehensive look at how patient-driven research and donor contributions make an impact. The Duct - the latest PSC news, updates and events The Viaduct - Patient Registry news For your privacy, any connection to the Registry will not be recorded in this signup form.

In Spring 2025, the Registry, in collaboration with PSC Support , conducted the PSC and Cholangiocarcinoma (CCA) Patient Survey. This multinational survey collected information on current practices of monitoring for CCA (also known as bile duct cancer), educational resources used by patients, and communication between patients and providers regarding CCA. Results from the survey  were presented at AASLD’s The Liver Meeting 2025 in Washington, DC. With 623 responses in just 7 weeks  between the Registry and PSC Support, it is clear this topic is of high importance to the community. Thank you to everyone who participated in this survey! Background: CCA Risk and Surveillance Guidelines People living with PSC are at a 400-fold higher risk for developing CCA than the general population: between 9% and 20% of people with PSC will develop CCA at some point in their lifetime. Even after liver transplant, CCA risk remains elevated, in part due to the chance of developing recurrent PSC. Unlike other liver cancers, CCA is not associated with the severity of liver fibrosis and disease and can happen at any time point after diagnosis. Therefore, ongoing CCA surveillance testing and early detection is an important part of patient care and may improve clinical outcomes. Early detection of CCA can lead to more treatment options, including potentially transplant, and a quicker connection to important support for cancer treatment. AASLD and EASL guidelines* recommend annual surveillance for CCA through MRCP (MRI of the liver, bile ducts, and pancreas) or ultrasound imaging. AASLD further specifies MRCP over ultrasound when possible. Doctors may also order blood tests for CA 19-9, a protein that can be elevated in the presence of CCA. The guidance does not make a recommendation for or against CA 19-9 testing, as the test has somewhat limited diagnostic power. More frequent follow-up imaging is recommended in cases of suspected (potential, but not confirmed), CCA. Through the International Collaborative Research Network, PSC Partners is supporting leading researchers who are working to develop and validate a new blood test for CCA. This test may allow for earlier, less invasive, and more accurate detection of CCA in PSC patients. Read more about the BIOMAP-PSC Project here . Patient-Provider Discussion of CCA Survey results showed that 50.8% of providers proactively discussed CCA risk with their patients. Proactive discussion from providers was strongly associated with patient knowledge on CCA. Overall, only 36% of respondents reported they had enough information on CCA. For patients in the United States, proactive discussion was also associated with adherence to the recommended use of annual MRCP. When CCA is proactively discussed without patient prompting, 56% of patients report adequate CCA knowledge . When healthcare providers have not proactively discussed CCA risk, 15% of PSC patients report adequate CCA knowledge . Healthcare providers in North America were more likely than providers in Europe to proactively discuss CCA. Hepatologists were more likely than gastroenterologists to proactively discuss CCA. Patient Perspectives As CCA is a serious topic but does not affect everyone, preferences on CCA discussion and management can vary greatly. Among survey respondents, 24% brought up significant fear or anxiety regarding CCA, and 5% of patients mentioned not wanting to discuss CCA until it becomes necessary. Here are a few standout quotes from de-identified survey responses: “It should not be underestimated how scary this sword hanging over the head of PSCers is.” “If I haven’t been diagnosed with it I don’t really need to discuss it with anyone. All it causes is anxiety.” “Getting CCA is a constant worry. I used to think it was an instant death sentence, but being a part of [PSC groups], I now realize that that is not necessarily true.” “It's a lingering risk, but it's helpful that my doctors have shown both a proactive approach to detection, and openness to discussing the real risk, but tempered by a calm approach to manage my expectations, and not excessively worry about something that hasn't happened yet.” It’s important to note you can shape the conversation around CCA . While only 68% of US patients reported annual MRCP (a key part of recommended management of CCA risk), you can discuss with your doctor whether it is appropriate for you to receive this imaging. PSC Partners is committed to building additional educational resources for CCA, along with other common issues associated with PSC. For more information about CCA, from education to support for patients and caregivers, please visit the Cholangiocarcinoma Foundation at cholangiocarcinoma.org . * Sources: AASLD practice guidance on primary sclerosing cholangitis and cholangiocarcinoma  (2022); EASL Clinical Practice Guidelines on sclerosing cholangitis  (2022)

In January, PSC Partners welcomed Assistant Registry Director Fernanda Quevedo as the newest member of the Registry team. She has extensive experience in research and research coordination and will be involved broadly in the work of the Registry. Fernanda holds a bachelor’s degree in neuroscience, with a minor in psychology, from the University of Texas at Austin. During her time there, she conducted research in pre-clinical studies of post-traumatic stress disorder (PTSD). More recently, she supported PTSD research as an adherence and supervision coordinator for research at a subsidiary of the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS). Prior to her time at MAPS, Fernanda spent five years working with Dr. Joshua Korzenik in the Crohn’s and Colitis Center and the Resnek Center for PSC at Brigham and Women’s Hospital in Boston, Massachusetts. Dr. Korzenik, the centers’ director, is a longtime friend and supporter of PSC Partners and frequently presents at the organization’s events. His involvement introduced Fernanda to the PSC Partners community.  “It was working with PSC Partners where … I was able to see what the community was doing and just how passionate people were about getting answers,” said Fernanda. “When I would meet with Ricky Safer [PSC Partners founder and CEO] and Rachel Gomel [Registry founder and advisor], I could really see how dedicated they were to not only finding answers for themselves, but also just finding better ways to support the PSC community.” As she looks forward, Fernanda hopes her efforts will help strengthen the work that’s already being done by PSC Partners, and she affirms that “we are relying on the community to express what the needs are.” She is focused on advancing high-quality research and expanding collaborative partnerships. Fernanda grew up in El Paso, Texas, and has returned to the general area, where she works remotely. Newly married, she and her husband Mat enjoy hiking with their pup. She has “many a hobby,” including baking, ceramics, and making wildlife ponds and aquariums.

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

Help researchers worldwide unlock the mysteries of primary sclerosing cholangitis (PSC). Complete your profile and join the PSC Partners Patient Registry to contribute your voice and experiences to our collective knowledge of PSC! You are an important piece of the PSC puzzle! Join the PSC Partners Registry to take action and drive PSC research forward. JOIN NOW LOG IN The Registry is a platform to amplify your voice in PSC research. The PSC Partners Patient Registry is a digital platform where basic data on people living with primary sclerosing cholangitis (PSC) is collected and stored. Data is primarily collected through online surveys completed by the participant living with PSC, or a qualified caregiver. Join the Registry to... Complete surveys that confidentially capture your experiences and address key questions in PSC research; Document and track your medical history with PSC, including treatments, symptoms, and healthcare providers; Elect to receive information about participating in research and clinical trials . You will be notified if you qualify for a study and can choose to contact the study team. The PSC Partners Patient Registry is open to everyone with PSC , wherever you are in the world. Parents and caregivers can join on behalf of their children. Any information you share is de-identified before sharing with researchers. No patient-identifying data ever leaves the Registry. LEARN MORE LATEST NEWS Patient Registry Now Accessible in Spanish & French Now Available: Clinical Survey 2026 The Viaduct - Issue 9, Fall 2025 Phase 2b VISTAS Study to Treat PSC-Related Pruritus Completes Enrollment What is the PSC Partners Patient Registry? What can I expect after I join? Learn More Join the Registry to add your information to our collective voice. Join Now Interactive map of healthcare providers provided by PSC Partners Patient Registry participants. Find PSC Specialists Data is power Thanks to people like you, the experience of living with PSC can be understood like never before. The more health information we collect through this patient registry, the closer we can get to helping our research community find treatments that work. Check out this video to learn more. "Where well-implemented registries and active patient organizations exist, the likelihood for developing a treatment for the disease in question is increased." (Eurordis-NORD-CORD) For questions, email registrycoordinator@pscpartners.org Visit PSC Partners website: pscpartners.org Join the Registry today! We're together in the fight, whatever it takes. With appreciation to Espen Bunæs for design support

Clinical Trials Clinical trials and research studies offer an opportunity for patients to participate in scientific and drug development research. In a clinical trial, participants receive specific interventions according to the research plan or protocol created by the investigators. These interventions may be medical products, such as drugs or devices; procedures; or changes to participants’ behavior, such as diet. Researchers will need your input through various surveys the Registry team will ask you to complete. You are the PSC patient's voice. One important function of the Registry is to assist in connecting PSC patients with clinical trials or studies for which they may be eligible. Based upon a Registry participant’s answers to the patient surveys, a participant may receive an email with information about a clinical trial that is recruiting PSC patients. You can also visit this page anytime to see the list of clinical trials conducted by drug developers working with the PSC Partners Patient Registry team. PSC Partners is collaborating with pharmaceutical companies planning and conducting clinical trials by adding your patient perspective to the trial protocol; by representing the patient voice throughout the drug development process; and by sharing clinical-trial information with the PSC community. Check this site often, as new clinical trials will be added as soon as they launch. The following clinical trials and studies are recruiting PSC patients. Contact the study for up-to-date information. An overview of the phases of clinical trials can be found here (source FDA). PSC Clinical Trials ♦ Indicates Studies Conducted with PSC Partners Collaboration or Input Filter by Country All Filter by Status All Sponsor or Research Institution Study Title & Phase Details (last updated September 2025) Links Arbor Research, NIH (NIDDK) and ChiLDReN, the Childhood Liver Disease Research Network ♦ Primary Sclerosing Cholangitis in Children: a 10-year Prospective Natural History Study in PSC Recruiting: Seeking 700 pediatric (ages 2-25) patients at multiple sites in North America for observational study. Study sites can be found at clinicaltrials.gov listing. ClinicalTrials.gov Identifier: NCT04181138 Learn More Brigham & Women's Hospital and Harvard University Detoxification of the Liver In PSC (DoLPHin) Phase 2 Study Recruiting: For adult PSC patients who live in the US. All study visits will take place remotely, and a nurse will visit your home to draw labs. ClinicalTrials.gov Identifier: NCT05835505 Learn More Brigham & Women’s Hospital and Harvard University ♦ Diet in PSC (DOCK Survey) Pilot Study Open Survey: US residents 18 and over: If you haven't already, please complete this short PSC nutrition survey. Survey Link Brigham & Women’s Hospital and Harvard University ♦ Sulfasalazine for the Treatment of Primary Sclerosing Cholangitis (SHIP) Phase 2 Study Active, Not Recruiting: For PSC patients aged 15 - 80. 12 Study locations in the US and patients can be enrolled anywhere in the US with nurse coming to your home to draw labs, provide medication, etc. ClinicalTrials.gov Identifier: NCT03561584 European Joint Program on Rare Diseases and national funding agencies in Germany, Canada, Belgium, Poland and Hungary ♦ Improving health-related quality of life in patients with rare autoimmune liver diseases by structured peer-delivered support: a transnational effectiveness-implementation hybrid trial Active, Not Recruiting: Belgium (Ghent), Canada (Toronto), Germany (Hamburg-Eppendorf), Hungary (Debrecen), and Poland (Warsaw) Trial Registration: ISRCTN15030282 Study Participant Info Ipsen Pharmaceuticals ♦ A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis. (ELMWOOD) Phase 2 Study Active, Not Recruiting: For adult PSC patients. US, Canada and Spain study locations. ClinicalTrials.gov Identifier: NCT05627362 Learn More Mirum Pharmaceuticals ♦ A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (VISTAS) Phase 2 Study Active, Not Recruiting: For PSC patients including AIH-PSC, both large and small-duct PSC, ages 12+, with any level of pruritus. 26+ US and Canada study sites and virtual participation option. ClinicalTrials.gov Identifier: NCT04663308 Learn More about the Decentralized (Virtual) Option Study Website National Institutes of Health: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research Approach Recruiting: Observational study for adult PSC patients and controls living in same household. ClinicalTrials.gov Identifier: NCT04685200 Learn More PSC Partners Seeking a Cure ♦ WIND-PSC: A Global Multi-Center Prospective Observational Cohort to Support Drug Development in Adult Patients With Primary Sclerosing Cholangitis Observational Study Recruiting: Global PSC patients aged 18-75, without transplant, small-duct PSC, IgG4-related cholangitis, or cirrhosis. New sites are opening soon; check regularly for more details! ClinicalTrials.gov Identifier: NCT06297993 Learn More about the PSC Partners WIND-PSC Initiative University of Minnesota Evaluation of an Oral Microbiota-based Therapeutic as a Treatment Option for PSC Early Phase 1 Study Recruiting: Seeking 28 adults aged 18-76. Visits take place at University of Minnesota Medical Center. ClinicalTrials.gov Identifier: NCT06197308 Learn More ClinicalTrials.gov Website ClinicalTrials.gov is a website that records clinical trials being conducted around the world. This website is maintained by the National Library of Medicine (NLM) at the US National Institutes of Health (NIH). Search for PSC Trials Above link will list all interventional trials on ClinicalTrials.gov for PSC that are recruiting, not-yet recruiting, active but not recruiting, and enrolling by invitation. Interactive Map of PSC Trials Above link will open an interactive map showing where the above listed trials are taking place. Sample Map (as of December 2023) Information about planned, ongoing, paused, and completed trials for PSC and a wide range of other diseases and conditions can also be found on this site. Visit ClinicalTrials.gov to explore the status of all clinical trials in PSC. More information about the ClinicalTrials.gov site can be found here . European Union Residents: Visit the EU Clinical Trials Register here to see active clinical trials in the EU. United Kingdom Residents: Visit the PSC Support UK Clinical Trials page for an interactive list and map of active clinical trials in the UK. History of Collaboration The following is a list of trial sponsors with whom PSC Partners Patient Registry has worked or is working to provide the patient voice and recruitment support. Visit Registry Impact to see a listing of publications and studies that have resulted from collaboration with the PSC Partners Patient Registry. 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 All rights reserved © PSC Partners Seeking a Cure