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 Elafibranor Advances to Phase 3 Clinical Trial for Treatment of PSC The Viaduct - Issue 10, Winter 2026 Patient Registry Now Accessible in Spanish & French Now Available: Clinical Survey 2026 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

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. Registry Overview WHAT IS THE REGISTRY? PRIVACY AND SECURITY CURRENT SURVEYS OTHER REGISTRY FEATURES The PSC Partners Patient Registry is a digital platform where basic data on people living with PSC is collected and stored. Data is primarily collected through online surveys completed by the participant living with PSC, or a qualified caregiver. Confidentiality and privacy measures are built into the Registry to ensure participant privacy. This data is then used to accelerate PSC research and advocate for the unmet needs of PSC patients. Researchers request de-identified patient data which is analyzed to gain new insights on PSC. Click here to view the current list of publications made possible through the Registry. Researchers can also request the Registry's assistance in recruiting for a clinical trial or study. The Registry team identifies participants who best meet the study's inclusion and exclusion criteria and privately send these participants information about the study. The participants can then choose to contact the study team if they want to learn more and/or are interested in joining the study. This practice ensures that the participant's identity is withheld from researchers and that it is solely the participant’s choice to join a study. The Registry also provides current news on clinical trials and studies in a few additional ways: the Ongoing Clinical Trials page, the Publications Related to Clinical Trials page, the VIADUCT Newsletter , and News posts . This information is publicly available under the "NEWS AND RESOURCES" and the “CLINICAL TRIALS” sections on the navigation menu. No identifying data ever leaves the Registry. The Registry’s operating software, Matrix, was designed and is operated in accordance with applicable US privacy protection provisions of HIPAA,¹ the European GDPR,² and FDA regulations.³ These regulations require specific documented operational controls and security procedures. Information that you provide to the Registry via the Matrix website is encrypted* and stored on secure servers with restricted physical access. To further protect the best interests of Registry participants, the Registry is annually reviewed by an Institutional Review Board (IRB) and all Registry team members have current HIPAA training. Any personal information that could be used to identify you or your family is labeled as Personally Identifiable Information (PII). Your PII is securely stored and encrypted. Only authorized Registry staff can access your PII and contact you if needed. Information that has had all PII changed to a code is called "de-identified." Only de-identified data is ever shared with researchers. ¹HIPAA: Health Insurance Portability and Accountability Act of 1996 ²GDPR: General Data Protection Regulation (GDPR) ³FDA regulations: Food and Drug Administration Title 21 of the Code of Federal Regulations Part 11 (21 CFR part 11) *Encryption means that even if a third party were to obtain access to the data, they must have access to a long “passkey” to decrypt and view the original information. Without having the passkey, it takes decades for even the most powerful supercomputers to figure out the original passkey. Clinical Survey The Clinical Survey is the heart of the Registry. This survey collects information on a wide range of topics, such as PSC diagnosis, symptoms and complications, other diseases, family history, and more. A new Clinical Survey is available each year so that the Registry can track changes over time. Health Equity Survey The Center for Disease Control defines health equity as the state in which everyone has a fair and just opportunity to attain their highest level of health. This survey seeks to build an understanding of the impact of PSC and barriers to receiving high quality care. PSC Flare or Cholangitis Attack Patient Survey Many people living with PSC are intimately familiar with what a cholangitis attack feels like. However, there is not yet a widely agreed-upon medical definition for this major complication associated with PSC. This survey aims to capture participants' history of cholangitis and symptoms during their most recent attack. Healthcare Provider Submission Form The PSC Partners Registry keeps a map of healthcare providers submitted by Registry participants. By submitting a provider via this short form, Registry participants can help other people living with PSC to find a doctor who is familiar with PSC or who specializes in PSC care. For the Bereaved If you have a loved one who had PSC and has since passed away, whether due to PSC or not, you can create a "Person who has lost a loved one" profile to access this survey. Everyone with PSC is an important piece of the PSC puzzle, and their history is important to capture. We acknowledge and appreciate the generosity of those who have lost someone with PSC. The Registry has a wealth of other tools for you. To access these, you must create or log in to your Registry account. Detailed tutorials are available to get you started. View Summarized Survey Responses Registry participants can view a summary of other participants' de-identified responses to many survey questions. For instance, you can see the breakdown of age of PSC diagnosis across the whole Registry, or the number of Registry participants who have a family member living with PSC. This is available through the "Survey Analysis" tab in the navigation menu of the Registry dashboard. Health Info Tracking Basic Health Info: Log your current and past medications and supplements, treatments and medical procedures, labs/tests, and more. Symptoms and Activities: Log common PSC symptoms (or add your own) and activities such as exercise. Journal: Keep track of notes from doctor visits, prepare your questions to ask your doctor, or save notes on whatever else helps you keep up with your PSC care. Multiple Language Support The Registry Dashboard supports English, Spanish, French, Italian, and German languages. Surveys are currently only available in English; Spanish and French versions will be available later in 2025. Electronic Health Record (EHR) Import If your healthcare providers use an EHR system, the Registry platform supports importing data from many of the common EHR systems. You may select which records you wish to import into the Registry. To learn more about the Registry, please visit the Registry's Frequently Asked Questions (FAQ) . You can also contact the Registry team for any questions you have!

Publications Related to Clinical Trials in PSC Participants may receive emails from the Registry alerting them to clinical trial opportunities for which they may be eligible. Since the Registry will never release identifying information about a participant, it is up to each participant to contact the trial or study coordinators to learn more about the opportunity. PSC Partners encourages all patients to learn as much as possible when making decisions about joining a study or trial. Publications related to ongoing clinical trials can be found on this page. Please expand the section of interest by clicking on the title. PSC Partners Seeking a Cure recognizes the urgent need for treatments to stop or slow the progression of PSC. We support the development of clinical trials testing the safety and efficacy of new drugs for PSC, including the investigation of drugs currently given off-label. These publication resources are being provided to support patients and caregivers in making decisions about possible clinical trial participation. Please feel free to email us at registryresearch@pscpartners.org to suggest additional publications for consideration. Notes are added solely to assist in navigating the list. Readers are encouraged to read the full text to consider the full context of the evidence in the publication. Please see the PSC Partners Statement about the Use of Medications Not Approved by the FDA for PSC . PSC Partners is in the process of developing layperson educational materials on how to evaluate medical research, and a link will be added here soon. In the meantime, some aspects to consider: In what journal has this study been published? Is this a prominent journal with a peer-review process? Is this a less-prominent journal? Is this print or online? Is this an abstract from professional meeting proceedings (such as AASLD, EASL, NASPGHAN)? Typically this means a less rigorous review process than a journal article. Who is being studied? Adults? Pediatric? Both? With IBD or not? Stage of PSC? Pre-transplant or post-transplant? How large is the population being studied? The larger the population, typically the more generalizable the result. The more diverse the patient population being studied, the larger the study population needs to be in order to draw conclusions. Is there a comparator population? Is there a group against which the studied patients are compared? This could be a group receiving a placebo. Or this could be a group receiving an alternative treatment. Possibly the comparison is based on current understanding of the 'natural history' of disease progression. How long is the study? Is it prospective (defines patient cohort and collects data moving forward in time) or retrospective (uses data previously collected in the past)? Prospective cohort studies in general have higher accuracy (can decide exactly what should be collected and can pre-determine cohort) and retrospective cohort studies have higher efficiency (data already exists and cohort is limited by those on whom data exists). Chemomab: SPRING Study - CM-101 in PSC Patients Read More Escient Pharmaceuticals: PACIFIC Study - EP547 in Subjects With Cholestatic Pruritus Read More Mayo Clinic: Oral Vancomycin for PSC Read More Mirum Pharmaceuticals: VISTAS - A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by PSC Read More Pliant Therapeutics: Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With PSC Read More

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

News and Announcements Join our Email List! Elafibranor Advances to Phase 3 Clinical Trial for Treatment of PSC On February 4, 2026, the initial study details for Ipsen’s Phase 3 ELASCOPE clinical trial for elafibranor for treatment of PSC were listed on ClinicalTrials.gov . Study enrollment is anticipated to start around April 2026 and key details of the study design are now available. “PSC Partners is thrilled to see Ipsen move forward with this global Phase III study,” said Ricky Safer, Founder and CEO of PSC Partners. “This is a key step forward in fulfilling our mission of findi 8 hours ago 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 Patient Registry Now Accessible in Spanish & French The Registry team is thrilled to announce that the Registry is now available in Spanish and French languages. Surveys, informed consent forms, and all other features are available in Spanish and French . This includes much of the general information on the Registry available at pscpartnersregistry.org . ACCESSING THE TRANSLATIONS From pscpartnersregistry.org , you can access translations by selecting the flag icon at the top of the webpage or within the page menu (on mobile) Jan 28 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? What’s new? In the Clinical Survey 2026, you will find changes and additions to the Symptoms section. These updates include questions developed by the PSC Partners’ Symptom Assessment Project (SAP) team and asked in the recent Symptom Assessment Survey (now closed). Th Jan 26 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 Phase 2b VISTAS Study to Treat PSC-Related Pruritus Completes Enrollment On September 8, 2025, Mirum Pharmaceuticals Inc. issued a press release announcing enrollment completion for the Phase 2b VISTAS study. This clinical trial is studying volixibat versus placebo to treat pruritus (itch) associated with PSC. Volixibat works by reducing bile acids in the blood, which may decrease the frequency and severity of pruritus. The VISTAS study targeted an enrollment of 200 adult and pediatric (12 years and older) participants living with PSC. Earlier bli Sep 8, 2025 A New Chapter for the Registry Dear PSC Registry Community, It is time for me to leave my position of Director of the PSC Partners Patient Registry. As founder of the... Sep 4, 2025 Now Available: Symptom Assessment Survey The Registry team is excited to announce that a new Symptom Assessment Survey is now available in the Registry. Developed by the PSC... Aug 20, 2025 Positive News from EASL Congress on Two Potential Treatments for PSC In May 2025, the European Association for Study of the Liver (EASL) hosted its annual congress in Amsterdam, Netherlands. Late-breaking oral presentations on potential treatments for PSC featured results from two clinical trials: Dr. Falk Pharma: Norucholic acid for the treatment of primary sclerosing cholangitis: 96-week analysis of a pivotal phase 3 trial , presented by Michael Trauner Ipsen Pharmaceuticals: Elafibranor for primary sclerosing cholangitis: the ELMWOOD phase May 7, 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 1 2 3 4 5

< Back 2019. Potential Association of Doxycycline With the Onset of Primary Sclerosing Cholangitis: A Case Series Authors: Buness JG, Ali AH, Tabibian JH, Buness CW, Cox KL, Lindor KD Journal: [published online ahead of print, 2019 Aug 30]. Am J Ther. 2019;10.1097/MJT.0000000000001065. doi:10.1097/MJT.0000000000001065 Excerpts: "Primary sclerosing cholangitis (PSC) is linked to inflammatory bowel diseases (IBD). Evidence suggests an association between the gut microbiome and PSC. However, the putative relationship between exposure to antibiotics and onset of PSC has never been reported. We observed 3 cases in which patients without antecedent liver or bowel issues developed symptoms leading to diagnosis of IBD and subsequently PSC after being exposed to doxycycline. We aimed to identify, through the PSC Partners national patient registry, additional cases of PSC in which there is a temporal relationship between exposure to doxycycline and onset of PSC or PSC-IBD." Pubmed.gov listing and abstract Previous Next

< Back 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 Authors: Donna Evon, Kaya Merkler, Laura Mkumba, Lauren Wright, Nicole Lucas, Ricky Safer, Joanne Hatchett, Rachel Gomel, Stephen Rossi, Patrick Smith, Mark Swain, Sasha Deutsch-Link, Bryce Reeve Abstract: Background: Understanding cognitive impairment experiences for adults living with PSC is essential for the development of PSC-specific, patient-reported outcome (PRO) measures that accurately reflect their real-world impact. Cognitive impairment (“brain fog”) in PSC is under-recognized yet can be frequent and debilitating, and is differentiated from hepatic encephalopathy. Methods: Adults were recruited through the PSC Partners Patient Registry and PSC hepatologists in the U.S and screened for a range of PSC-related symptoms. Purposeful sampling was used to select 17 participants for in-depth, individual interviews focused on key symptoms, including cognitive impairment. Interviews were conducted virtually, audio-recorded, transcribed, and analyzed using NVivo software. Results: Eleven out of 17 participants were interviewed regarding their experience with cognitive impairment. Participants were on average 43 years old (range: 27- 65), 55% male, 27% Black, 64% White, and 55% had inflammatory bowel disease. The Table below provides participants’ illustrative quotes aligned with cognitive domains and life impacts. Severity ranged from mild to debilitating, with a few participants reporting inability to drive, cook independently, work full-time, or engage socially. Participants primarily characterized their cognitive impairments as occurring in the areas of working memory, verbal fluency, sustained attention, and processing speed. Others described a mental “wall” preventing them from absorbing and processing information. Half of participants endorsed experiencing some type of cognitive impairment on a daily basis, while others experienced it “a few days” a week or month. A “domino effect” can occur that begins with cognitive impairment reducing work productivity, which cuts into social time, and in turn, worsens mental health. Cognitive impairment. often accompanied fatigue, but reportedly can also occur alone. Most participants were able to differentiate PSC-related cognitive impairment from other causes based on association with severe fatigue, improvements with ERCPs and lactulose, and worsening during cholangitis attacks. Conclusion: PSC cognitive impairment is multi-faceted and impacts daily, work-related, and social activities among symptomatic adults with PSC. These findings will inform the customization of a PSC-specific PROMIS® Cognitive Function measure with strong content validity, enhancing its relevance for future clinical research and treatment evaluation. Previous Next

< Back 2024. Patient-focused drug development in primary sclerosing cholangitis: Insights on patient priorities and involvement in clinical trials Authors: Michael Li, Ruth-Anne Pai, Rachel Gomel, Mary Vyas, Sarah Curup Callif, Joanne Hatchett, Christopher L Bowlus, Jennifer C Lai Journal: Hepatology Communications 2024 May 10;8(6):e0433. Abstract: Background: According to the new AASLD Practice Guidance, all patients with primary sclerosing cholangitis (PSC) should be considered for participation in clinical trials. However, PSC’s rarity has posed challenges to characterizing patient interest in trial participation and identifying predictors of patient willingness to participate in drug trials. Methods: PSC Partners Seeking a Cure developed the “Our Voices” survey to inform the development of the Externally-Led Patient-Focused Drug Development Forum, an FDA initiative to capture patient experiences and perspectives on drug development. Results: Of 797 survey respondents from over 30 countries, 536 (67%) identified slowing disease progression as the most important outcome. Eighty-nine percent identified their hepatologist/gastroenterologist as someone they would approach for advice about trials. Although 61% reported being willing to participate in drug trials, only 26% had ever been asked to participate. Notable barriers to trial involvement included unknown long-term risks (71%), long travel times to the study center (32%), and a liver biopsy requirement (27%). On multivariable logistic regression, pruritus (OR 1.62, 95% CI: 1.09–2.40, p = 0.017) was positively associated with willingness to participate in disease-modifying therapy trials, while jaundice (OR 0.34, 95% CI: 0.19–0.61, p < 0.001) and inflammatory bowel disease (OR 0.64, 95% CI: 0.42–0.98, p = 0.038) were negatively associated. Pruritus (OR 2.25, 95% CI: 1.50–3.39, p < 0.001) was also independently associated with willingness to participate in symptom treatment trials. Conclusions: Most patients with PSC report interest in participating in clinical trials, but few have been asked to participate. Referral of patients with PSC by their hepatologist/gastroenterologist to clinical trials and patient education on trial participation are vital to closing the gap between trial interest and participation. Pruritus may serve as a key indicator of patient interest in trial participation. Previous Next

< Back 2017 Registry Information Informs PSC & PBC "Call to Action" Education Campaign PSC Partners Seeking a Cure Surpasses 1,000 Patients in International Registry and Discovers Unexpected Misdiagnoses and PSC Partners Seeking a Cure and PSC Support Announce a Call to Action for Medical Stakeholders to Better Understand and Diagnose Rare Liver Diseases An unexpected result of enrolling more than 1,000 patients in the registry is that some of those who tried to enroll learned -- after answering a series of questions about their diagnosis and symptoms -- that they do not actually have PSC. Instead, they have primary biliary cholangitis (PBC), a distinctly different disease that also affects the bile ducts. This misdiagnosis is troubling, given that effective treatment exists for PBC and patients who thought they had PSC may have been missing out on treatment that would slow the progression of PBC. PSC Partners Seeking a Cure and PSC Support, a nonprofit PSC patient organization based in the UK, have been working on an educational campaign to promote a better understanding and more accurate diagnosis of these two rare liver diseases. Given the similarity of their names, PSC and PBC have been confused in medical publications, among regulatory bodies, and in medical practice -- as well as in individual patient diagnoses, as seen in the PSC patient registry. With the recent renaming of another liver disease, primary biliary cholangitis (PBC), formerly called primary biliary cirrhosis, an opportunity has arisen to alert clinicians of the potential for confusion and stress the importance of correctly identifying these two distinctly different bile duct diseases, PBC and PSC. Patients with PSC also have a devastating and distinctly increased risk of colon and bile duct cancers, while patients with PBC fortunately do not have increased risk for either of these cancers. Consequently, patients with PSC need special cancer surveillance, as well as advice on how to live with cancer risk in a way that is supportive and not life-limiting. Excerpts above are from press releases by PSC Partners Seeking a Cure. See full text of related press releases: 2017-04 Press Release and 2016-11 Press Release .

< Back 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 Authors: Donna Evon, Kaya Merkler, Jing Yuan, Laura Mkumba, Lauren Wright, Nicole Lucas, Joanne Hatchett, Ricky Safer, Stephen Rossi, Bryce Reeve Abstract: Background: A comprehensive understanding of fatigue experienced by adults living with PSC is necessary for the development of a PSC-specific patient-reported outcome measure that captures essential fatigue concepts and is suitable for clinical trials to assess treatment benefits. Methods: Adults with PSC were recruited through the PSC Partners Patient Registry or their hepatologists. Participants consented and were screened for the presence, frequency, severity, and distress associated with multiple PSC-related symptoms. Purposeful sampling was used to capture a representative sample of 17 adults to engage in in-depth, individual qualitative interviews to describe key symptoms, including fatigue. Interviews were conducted virtually, audio-recorded, and transcribed. Transcripts were coded and analyzed using Nvivo. A codebook was developed to identify themes related to fatigue severity, frequency, interference, and symptom clustering. Results: Twelve out of 17 participants who reported fatigue as a prominent symptom were asked to describe in-depth their experiences living with fatigue. Participants were, on average, 39 years old (range: 22 to 58), and diagnosed with PSC, on average, at 31 years old. Participants were 42% female, 8% Asian, 17% Black, and 75% White, and 67% had inflammatory bowel disease (IBD). Table 1 provides illustrative quotes of participants’ terminology used to describe the experience of fatigue. Half of the participants described experiencing fatigue “all the time”, while some described variation in fatigue severity throughout their day and week and several required daytime naps. Some participants felt best in the morning and described fatigue getting progressively worse throughout the day. Fatigue interfered with their daily domestic tasks, job responsibilities, and family and social activities. Fatigue impacted mental health with participants expressing feelings of frustration, sadness, and guilt. Rationing energy, breaking up daily tasks, and naps were used to cope with fatigue. Participants can describe the difference between fatigue and a poor night’s sleep or simple tiredness. All participants reported experiencing concomitant “brain fog” with fatigue, although they can occur separately. Some participants report that fatigue can be accompanied by body pain or nausea. Conclusion: Fatigue emerged as a prominent and distressing symptom in adults with PSC. These qualitative findings will directly inform the customization of a PSC-specific PROMIS® fatigue measure with strong evidence for its content validity. The measure will be suitable for future PSC clinical trials and research to find effective treatments for people living with PSC-fatigue. Previous Next

< Back Ipsen Pharmaceuticals A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis. (ELMWOOD) Phase 2 Study Previous Next