Search Results

A new survey outside the Registry that focuses on understanding and improving the care of people living with PSC is now available. Every survey fills a different function, and this one, developed by two researchers at University of Miami, serves to build a comprehensive understan ding of PSC care, including quality of care and access to care. Please visit the survey website and complete the survey today!   Your input is incredibly valuable. Below is a message from the study investigators: Dear Registry Participants, Welcome to our survey! We're working to make healthcare better for people living with Primary Sclerosing Cholangitis (PSC), and your input is super important. We want to know about your experiences and thoughts to find ways to improve patient care. Your contribution means a lot to us. The survey includes simple and clear questions, and it should only take around 20-25 minutes. Your dedication to this research can really make a difference for people dealing with PSC, and we're excited about the positive impact we can make together. Best, Cynthia Levy, MD Adrielly Martins, FMD Shiff Center for Liver Diseases University of Miami If you have any questions or concerns regarding this survey, please contact Cynthia Levy or Adrielly Martins: (phone: 305-302-2474 or email: axm8717@med.miami.edu )

On February 19, 2025, Chemomab Therapeutics issued a press release announcing plans for a Phase 3 clinical trial for the treatment of PSC. The full press release is available on the Chemomab website.  The investigational drug being tested has been named nebokitug, previously studied in PSC in the SPRING Study as CM-101. PSC Partners is encouraged by the news that a potential drug for PSC is moving forward into the final stages of the drug approval process (Phase 3 clinical trial).  Some highlighted excerpts from the press release: The novel trial design does not require a liver biopsy. The trial is a randomized placebo-controlled (2:1 active to placebo ratio). Patients in the drug treatment arm will receive 20 mg/kg of nebokitug administered intravenously every three weeks. The primary endpoint* is the time-to-first clinical event[s] … associated with PSC disease progression, including more common clinical events such as acute cholangitis, strictures requiring intervention or portal hypertension as well as cholangiocarcinoma, transplant, or death. See the press release for the full list of specific events. Enrolled patients will remain in the trial until they experience one of the primary endpoint clinical events. On average, participants are expected to remain in the trial for about two years. Approximately 350 PSC patients will be enrolled in the trial, and the study population will be enriched** for patients with moderate and advanced disease. The FDA has agreed that a single Phase 3 trial, if successful, would be sufficient to proceed to regulatory review and potential drug approval. *Primary endpoint: The single most important outcome that researchers measure to determine whether a new treatment is effective. **Enriched: The investigators intend to enroll a higher number of participants matching these criteria than may be representative of the overall population of people with PSC. A Phase 3 trial is the final phase of clinical trials before the Food and Drug Administration (FDA) conducts a review to determine whether to approve the drug for market. An overview of the phases of clinical trials can be found here  (source FDA). PSC Partners will continue to update the community as more information becomes available regarding this study. Please check back for updates, including on the Registry’s Ongoing Clinical Trials page. For more information regarding CM-101: Previous news article: Chemomab Announces CM-101 Phase 2 SPRING Trial Results More results from the open-label extension of the SPRING trial are expected in the first quarter of 2025. Two scientific publications related to CM-101 are available in the Related Publications page  of the Registry’s Clinical Trials section.

A new survey on PSC and diet is available through the European Reference Network (ERN) and open to people living with PSC around the world. Diet and nutrition in PSC is a frequently discussed topic, and adding your voice will help to progress research in this important area! This survey was developed by Catarina Lindqvist (Karolinska Institutet, Sweden) and the ERN’s PSC Working Group. Because this anonymous survey is not connected to the Registry, it will ask some basic questions about your health. You can access the survey here. Below is a message from the study investigators: Dear individuals with PSC, We invite you to participate in an anonymous questionnaire that aims to further understand the dietary habits of individuals living with Primary Sclerosing Cholangitis (PSC). This research is designed to explore how individuals adapt their diets due to PSC, and if applicable, due to underlying Inflammatory Bowel Disease (IBD). Additionally, we seek to identify the primary sources of information individuals with PSC rely on for dietary advice. The questionnaire is divided into three sections: Background information Questions regarding PSC Questions regarding IBD (only applicable to individuals with coexisting IBD) Your participation in this questionnaire is crucial for advancing our understanding of how PSC and IBD influence dietary habits. The insights gained from this study will contribute to developing better support and resources for individuals managing these conditions. The link to the questionnaire:  https://ec.europa.eu/eusurvey/runner/a4aa42c5-0fe8-3d66-fc9c-e290ec541d4a The questionnaire is available in English, French, Spanish, Italian, German, Croatian, Hungarian, Czech, Polish and Swedish. Thank you for taking the time to share your experiences and contribute to this important research. Sincerely, Catarina Lindqvist, Karolinska Institutet, Sweden and the PSC Working Group We hope you will participate in this important survey. If you have any questions regarding this study, please contact the team using the Contact Form available in the right menu at the survey link.

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 new surveys, the Registry is more active than ever before! With so much coming out from the Registry, the VIADUCT will be published more frequently next year. I look forward to the Registry continuing this growth in 2025. Be on the lookout for a new 2025 Clinical Survey launching on January 2. If you haven't yet logged into the new platform, or completed the 2024 survey, you've got a few days to get to it before the next survey launches! To best capture changes over time, it’s recommended to wait at least 6 months since completing your last annual survey. You will receive a reminder when it’s the ideal time to take the 2025 Clinical Survey. Thank you for your continued support and engagement with the Registry. By logging in, completing any available surveys, and updating your information when needed, you directly support PSC research and center the patient voice in the path towards a cure for PSC. Kind regards, Rachel Gomel and the Registry Team A New Era for the PSC Partners Registry Community 2024 has been a big year for the PSC Partners Patient Registry, which celebrated the 10th anniversary of its initial opening and the March launch of an upgraded data platform. Click the "READ MORE" button below to learn about the upgraded Registry site. Driving Research: Your Data in Publication - 2024 Thanks to your active participation in the Registry, significant progress has been made in PSC research. Four publications using Registry data were released in 2024. Topics include improving patient-centered clinical trial development, special considerations of pediatric PSC patients, and better understanding PSC-related fatigue and cholangitis. Clinical Trial News Announced at PSC Partners Annual Conference The 20th Anniversary PSC Partners Annual Conference took place in Phoenix, Arizona, on October 18-20, 2024. The "Industry Updates: Ongoing PSC Clinical Trials" general session featured reports by pharmaceutical representatives about several trials. A few weeks later, more late-breaking news came from The Liver Meeting , held in San Diego, California by the American Association for the Study of Liver Diseases (AASLD). Registry Data Presented at PSC Forum and AASLD's The Liver Meeting In November, a PSC Partners contingent, representing both the U.S. and Canada, attended the 8th PSC Forum and the AASLD's The Liver Meeting  (TLM). Two current PSC Partners research projects were discussed at the PSC Forum, and two posters that included your (de-identified) patient data were presented at TLM. Your Powerful Impact PSC Partners opened the Patient Registry for enrollment 10 years ago. However, the process of building a Registry began several years before its 2014 launch. From a 2012 pilot program with the NIH to a “gold mine” for PSC research and accelerating drug development, the Registry has grown far beyond what was once thought possible for patient-led research initiatives. PSC Partners Registry and Other Registries: An Example of Different Types of Registries This past July, the American Liver Foundation (ALF) announced the opening of a new patient registry. While similar in function and structure, the ALF has different goals and eligibility requirements compared to the PSC Partners Patient Registry. PSC patients are encouraged to join all registries for which they qualify! Learn more about the new ALF registry and how the two registries differ. Snapshot of Registry Data This word cloud was generated from responses to the "PSC Flare or Cholangitis Attack Patient Survey", currently active in the Registry. If you haven't yet, add your response today! Established in collaboration with the Office of Rare Diseases Research ( ORDR ), the National Center for Advancing Translational Sciences (NCATS), and the National Institutes of Health (NIH) © 2024 PSC Partners Seeking a Cure

This past July, the American Liver Foundation (ALF) announced the opening of a new patient registry. You may be wondering how it compares to the PSC Partners Registry and whether you should join the ALF registry. Similarities of structure and function between the two include: a reliance on patient-reported data through registry surveys; a sharing of de-identified data with approved researchers; the ability for patients to add to or update their information through secure patient accounts; and communication with patients about research opportunities and research news. However, the purposes of the two registries and the patient groups included in each differ in important ways. The PSC Partners Patient Registry  is for those diagnosed with PSC, and the data collected supports research specifically focusing on PSC and the challenges facing those who live with PSC. The PSC Partners Registry includes patients from more than 50 countries around the world and of any age, including children. The only requirements to participate in the PSC Partners Registry are having a PSC diagnosis and having the ability to log onto the secure internet site to complete the surveys. Patients complete a core clinical survey that asks questions about their clinical history and PSC symptoms. Annual survey updates provide researchers with a record of disease progression or stability. Surveys that cover other areas of PSC research interest, such as health equity or topics related to current projects of PSC Partners, are available periodically. De-identified survey data is shared with PSC Partners-vetted research teams.  The PSC Partners Registry is a powerful tool for understanding the PSC experience in a detailed and focused way, and it supports PSC research by providing the patient perspective to researchers. The American Liver Foundation   Patient Registry  is for those diagnosed with any of more than 100 liver diseases. Adults 18 and over living in the U.S.  may join and participate. The ALF Registry’s stated purpose is “to provide researchers with a better understanding of liver diseases, the impact of treatments and resources for … liver disease, and how … liver disease affects [patients].” ALF may also provide educational resources to patients. A potential outcome and benefit of the ALF Registry’s broader study of liver diseases may be the discovery of commonalities among those diseases. This could lead to research into the cross-purposing of treatments between differing liver diseases. PSC patients are encouraged to join all registries for which they qualify. Each registry has its own purpose, and the more information that patients provide to researchers, the more quickly the researchers and drug developers may discover effective treatments for patients.

In November, a PSC Partners contingent, representing both the U.S. and Canada, attended the 8th PSC Forum and the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting  (TLM) in San Diego. The PSC Forum, held just prior to TLM, is a multi-stakeholder gathering of researchers, regulators, drug developers, and patients organized by UC Berkeley’s The Forum for Collaborative Research. The agenda included discussion of two current PSC Partners research projects: the WIND-PSC Study and the Acute Cholangitis Survey. The survey was developed by a PSC Forum working group co-chaired by PSC Partners and PSC Support U.K. and conducted, in part, through the PSC Partners Patient Registry. Registry Analyst Brian Thorsen presented an early analysis of the survey data (see slides here ). Following the PSC Forum, PSC Partners exhibited during TLM to extend awareness of its programs and research funding to the many research clinicians attending the meeting from around the world. Throughout the four-day event, meetings were held between PSC Partners and collaborators, potential collaborators, and industry partners. Two abstracts/posters that included your (de-identified) patient data were presented at TLM and have been added to the Registry website . To ensure your patient experience is represented in these and future analyses, please be sure to complete the surveys offered to you through the Registry!

PSC Partners opened the Patient Registry for enrollment 10 years ago. However, the process of building a Registry began several years before its 2014 launch. Looking back to 2010, the concept of a patient-driven registry was in the realm of the impossible. The medical world questioned the value of any patient initiative, and the rare disease field remained unexplored. Hungry for treatments for our incurable disease, Registry Director and Founder, Rachel Gomel attended a crowded National Institutes of Health (NIH) meeting in Washington, D.C. There, we first heard the invigorating message that well-organized patient organizations could acquire the power to speed up research by creating their own patient-driven registries. These would be different from registries established by physicians and managed by medical centers. They would be organized by patients, run by patients, and the data would be provided by patients.  We couldn’t believe that patient-driven data could have a place in research. But it was the NIH, the Food and Drug Administration (FDA), clinicians, and researchers themselves who were showing us the way. PSC Partners was then a tiny team of passionate, eager patient and caregiver volunteers who believed that a cure for PSC could be within our reach if we brought awareness of our unmet needs to those positioned to help us stimulate research.  At that large workshop 14 years ago, many speakers shared their perspective on the huge hurdles that prevented rare disease research from moving forward. One story vividly persisted with us through the years and motivated us to keep pursuing the registry initiative. On the podium stood an elderly doctor who shared how she had zealously collected data from patients seriously impacted by a very rare disease. She described the devastating disease and the hours she had spent collecting each patient’s clinical information and recording their data into a registry. And yet, since her retirement, all the data she had painstakingly gathered was collecting dust on a shelf in her office.  No one was interested in the data, and no one wanted to maintain her registry. She burst into an impassioned plea, asking us - patient organizations - to take charge of our patients’ data, since patients would be the best and most responsible stewards of their data and would never allow this valuable information to die. We started our journey not knowing where the Registry would take us.  In 2012, the NIH selected PSC Partners for its pilot rare disease registry program. After two years of work with the NIH, the PSC Partners Patient Registry was launched in 2014. A steady stream of PSC community members came forward to share their medical information. Through the years, we’ve watched the transformation of the medical field and of the patient voice. It is no longer a surprise that the FDA, a U.S. institution that assesses and approves drugs, requires patient involvement in every step of research and drug development. Whereas blood tests, MRIs, and clinical tests were previously the primary objective measures of disease, now the patient voice describing how the patient feels and functions is becoming an equally important consideration. The 2020 “Our Voices Survey”, which so many of you completed and the results of which were shared in a meeting with the FDA, was a step towards highlighting the ways people with PSC feel and function in different phases of disease. If a new drug improves how a patient feels and functions, this improvement matters to the patient.  Researchers and the FDA are looking for ways to quantify how patients feel and function through standardized and disease-specific surveys that measure patient-reported outcomes (PRO). PSC Partners has hired two PRO experts to follow the lengthy scientific process in building a PSC-specific PRO for use in clinical trials. Some of you have participated in focus groups and interviews with these experts; your input is greatly appreciated. Accelerating Drug Development Hearing that the Registry now holds well over 2500 participants, drug developers consider our rare disease as a possible area to research. One memorable example of the impact of the Registry took place in Paris, at the 2018 International Liver Congress organized by the European Association for the Study of the Liver (EASL). A drug developer, curious about our modest patient booth that was surrounded by large, elaborate pharma booths, eagerly listened to our description of the Registry. A few hours later, he returned with his colleagues, who were excited to have discovered a rare disease in their field with easy access to patients. The Registry has pulled patients into the clinical trial space, which previously had been closed to us. Thanks in part to the Registry, PSC Partners has developed relationships with dozens of pharmaceutical and biotech companies. Based on data you provided, studies and posters have been presented in large international hepatology conferences. Pharmaceutical companies are coming to us earlier and earlier in the process of planning their clinical trials. What is your contribution to this process? Through the Registry, you have been telling us why you join clinical trials, what prevents you from joining, and how clinical trials could be improved to make them easy to join. We bring them your suggestions and concerns and ask them to make specific changes. Most have never met a PSC patient. We organize meetings so that those who develop the drugs and run the trials hear our stories and can ask questions. They tell us that meeting patients and understanding their unmet needs have added meaning and urgency to their daily work. And, very importantly, we help them recruit patients from the Registry so they can conduct their clinical trial. Researchers at the PSC Center of the University of California in San Francisco (UCSF) described the Registry data as “a gold mine” and are collaborating with us to publish your data. The publications represent an important milestone: they fill an important gap, as very few academic papers exist on the PSC patients’ voice. Through this collaboration, the patient voice is being translated into scientific, peer-reviewed literature, which we will continue to make available to you as soon as studies are published. Thanks to your participation in the Registry, there is more interest in PSC research, greater awareness of PSC and of patient needs, and better clinical trials. Thank you for responding to our requests. YOU are making an impact! Together, we are creating a world where a PSC diagnosis comes with a cure!

The 20th Anniversary PSC Partners Annual Conference took place in Phoenix, Arizona, on October 18-20, 2024. Throughout the conference weekend, more than 360 attendees were reminded of how far PSC Partners has come as an organization in the past 20 years and of the accelerating progress of PSC research. Evidence of that progress included the second gathering of the International Collaborative Research Network (ICRNetwork), which met prior to the general patient/caregiver conference. The ICRNetwork, launched in June 2022, is a group of key stakeholders, including researchers, clinicians, patients, and industry members, that was formed in response to PSC Partners’ focused efforts to forge a strategic research plan. Many of the ICRN members were speakers for the patient/caregiver sessions and presented up-to-date information on PSC management and research advances. One of the general sessions, “Industry Updates: Ongoing PSC Clinical Trials,” included reports by pharmaceutical representatives about several trials for which the Registry has assisted with trial design and/or recruitment, and in which some of you have participated. Presenters sharing updates about their PSC drug trials included Chemomab Therapeutics, Intercept Pharmaceuticals, Ipsen Biopharmaceuticals, Mirum Pharmaceuticals, and Pliant Therapeutics. You can find more information about their trials on the Clinical Trials page. Chemomab and Pliant gave similar presentations a few weeks later at “The Liver Meeting,” an annual global convening of hepatologists and other experts from around the world, sponsored by the American Association for the Study of Liver Diseases. Each presented late-breaking posters about their trial drug results. You can read the news releases and view the posters at the links below. Chemomab/SPRING trial: News Release Chemomab’s AASLD Poster Pliant/ INTEGRIS-PSC trial:  News Release Pliant's AASLD Poster The conference weekend was filled with enthusiasm as both PSC Partners and its Registry celebrated their respective anniversaries amid a growing sense of hope for the future of PSC research and much improved PSC treatments.

Thanks to your active participation in the Registry, significant progress has been made in PSC research. Four publications using Registry data were released in 2024  - two journal articles and two scientific posters. Topics include improving patient-centered clinical trial development, special considerations of pediatric PSC patients, and better understanding PSC-related fatigue and cholangitis. Click on each title to access the abstract and full article/poster (if available). Journal Article: Patient-focused drug development in primary sclerosing cholangitis: Insights on patient priorities and involvement in clinical trials Using the Registry’s 2021-2023 “Our Voices” survey results, researchers looked at patient priorities in drug development and factors/barriers contributing to clinical trial participation. While 61% of patients with PSC reported interest in trial participation, only 26% have ever been asked. Conversations and education with/from hepatologists and gastroenterologists “are vital to closing the gap between trial interest and participation”. Journal Article: Caregiver-reported symptom burden and preferences for therapeutic goals in pediatric primary sclerosing cholangitis Also drawn from the “Our Voices” survey, this study summarizes responses from 51 pediatric PSC patients and caregivers. The responses reveal “a substantial patient/caregiver-reported symptom burden for children with PSC that impacts quality of life and limits access to clinical trials.” Conference Abstract/Poster: 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 A series of in-depth interviews were conducted via recruitment from Registry participants. Analysis of presence, frequency, severity, and distress related to PSC symptoms led to fatigue emerging as a “prominent and distressing symptom.” These interviews inform development of a symptom assessment tool (survey) for use in clinical trials to measure the effectiveness of a treatment in improving patient-reported outcomes, including fatigue. Conference Abstract/Poster: 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 Using the “PSC Flare or Cholangitis Attack Patient Survey” , the Registry Team worked directly with an international team of researchers to characterize patient experiences with cholangitis attacks (acute cholangitis). This survey demonstrated a “broad dynamic range of the frequency and severity of symptoms”, and is a first step in developing a precise definition of acute cholangitis for use in clinical trials as a potential outcome of interest. Many thanks again to the Registry participants who have taken the surveys to make these publications possible! The “PSC Flare or Cholangitis Attack Patient Survey” is currently active in the Registry. Log in and complete this survey today! Data is always de-identified and your responses remain confidential while advancing PSC research.

A huge THANK YOU! to everyone who has kept the PSC Partners Patient Registry an active and constantly growing beacon of the patient voice in PSC research! Thanks to your time and effort, 179 new participants living with PSC  joined the Registry, and 416 surveys  were completed between the Registry’s re-launch in March and August 31. As part of the Registry’s 10th anniversary celebration, a drawing for three $100 gift cards was held for anyone who joined as a new participant, logged in and updated their profile information, and/or completed a survey by August 31. The winners of these gift cards have been randomly selected and contacted via email.  To maintain the confidentiality of Registry participants, the Registry will not be announcing their names. Congratulations to the winners, and thank you to each and every person who participated in the Registry to enter this drawing! Each actively engaged Registry participant and each completed survey provides a wealth of information to address pressing research questions and provide a better understanding of the impact of PSC on people living with the disease. There is always more to do – if you haven’t yet updated your profile information or taken the 2024 Clinical Survey, please log in and complete these tasks today! Stay tuned for two new surveys that will be launching in the coming weeks… Together, we are creating a world where a PSC diagnosis comes with a cure.

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 relative, friend, or caregiver of one, you probably never stop searching to understand how research into PSC treatments is progressing.  In this newsletter, you will find a review of PSC Partners’ recent webinar about current trials; highlights of the annual conference session on the international PSC research landscape; information about a current, exciting study for children and young adults; an invitation to submit ideas and concerns about trials that we can take to those conducting clinical trials; and more. You can also explore trial information at your convenience on the Registry website. The Registry team maintains a list of currently enrolling PSC clinical trials and studies on the “Clinical Trials” webpage and provides links to detailed information about them. On the same page, you can find links to more general information, such as a map of trial locations and an explanation of clinical trial “phases.” A demonstration of how to find the trial page and navigate the map resource is included in a short Registry video tour that can be found here (time stamp 3:40). An important function of the Registry is to assist in connecting PSC patients with clinical trials or studies for which they may be eligible. Based on your answers to the patient surveys, you may receive occasional emails with information about a clinical trial that is recruiting PSC patients. PSC Partners encourages all patients to learn as much as possible when making decisions about joining a study or trial. That is why we created a new Registry feature, the “Publications Related to Clinical Trials in PSC” page , located in the Resources section of the website. Here we suggest ways of evaluating studies, articles, and information related to ongoing clinical trials. Regardless of whether you are interested in or eligible for a trial, staying abreast of PSC research progress will keep you informed about new treatments and discoveries. Your engagement and your input about study design and methods is valued. “Together, we are creating a world where a PSC diagnosis comes with a cure!” On behalf of the Registry team, Rachel Gomel, Director, PSC Partners Patient Registry The Clinical Trial Landscape in PSC - Overview of a PSC Partners Webinar One of the recent PSC Partners post-conference webinars brought together the current clinical trial perspectives in PSC. Numerous clinical trial updates were presented at the September 21 event , “The Clinical Trial Landscape in PSC: Current and New Opportunities for Patients.” A panel of experts discussed ongoing PSC studies and clinical trials. The goal of this webinar was to familiarize the PSC community with what clinical trial participation entails, with the different types of clinical trials that are being conducted, and on ways we can advance towards new treatments and the much anticipated cure. Trials/studies presented were: Brigham and Women’s Hospital (DINER nutrition study) Chemomab Therapeutics (SPRING study of novel antifibrotic, anti-inflammatory, biologic drug CM-101) CymaBay Therapeutics (study of Seladelpar for impact on PSC disease progression) Escient Pharmaceuticals (trial of drug EP547 for pruritus) Gilead Sciences (PRIMIS study of Cilofexor for fibrosis) - This trial was terminated after the webinar; see details below. HighTide Therapeutics (study of HTD180 drug, also known as berberine ursodeoxycholate, with anticipated anti-inflammatory, anti-cholestatic, and antifibrotic properties) Mayo Clinic (oral vancomycin trial) Mirum Pharmaceuticals (VISTAS trial of volixibat for pruritus) Pliant Therapeutics (INTEGRIS-PSC trial of the antifibrotic drug PLN-74809) You are invited to watch the recorded presentations and learn about these ongoing or recently completed trials. Current International PSC Research: Updates from the 2022 PSC Partners Conference An update on international PSC research activity was one of the most popular sessions at the June 2022 PSC Partners conference. Conference co-host Dr. Joshua Korzenik  moderated the presentation, titled “International PSC Research: Updates Across Spectrum – From IBD & Fibrosis to Genetics & Environment.” This informative conference session is online and includes the following three presentations and a question-and-answer segment. “Fibrosis and Fibrotic Mechanisms in Primary Sclerosing Cholangitis,” presented by Richard Green, MD, of Northwestern University Feinberg School of Medicine (time stamp: 3:19 ) “The Norwegian PSC Research Center…an Update from Oslo and Bergen,” presented by Tom Hemming Karlsen, MD, PhD, Norwegian PSC Research Center (time stamp: 20:19 ) “PSC and IBD. A Close Relationship. Siblings or Distant Cousins? What We Can Learn from It,” presented by Joshua R. Korzenik, MD, Brigham and Women’s Hospital (time stamp: 38:57 ) The Q&A portion was moderated by Joel Pekow, MD, of University of Chicago Medicine. Kirsten Muri Boberg, MD, PhD, of the Norwegian PSC Research Center, joined Drs. Green and Korzenik to answer questions submitted by conference participants (time stamp: 58:03 ). You are encouraged to check out what’s new in PSC research and find out the ways in which it is gaining momentum towards developing better treatments for people living with PSC. The ChiLDReN Study: an Opportunity for Children and Young Adults with PSC The Childhood Liver Disease Research Network (ChiLDReN) is a collaborative team of medical professionals, patient support organizations, and the National Institutes of Health (NIH). With clinical sites and research laboratories in the U.S., Canada, and the United Kingdom, ChiLDReN sponsors a number of natural history studies focused on rare cholestatic liver diseases, including a new study on PSC in the pediatric and young adult PSC population. “Primary Sclerosing Cholangitis in Children,” is an observational, 10-year prospective natural history study funded by the NIH.* Study investigators are seeking 700 young PSC patients at nine North American sites. Those aged 2 – 25 at time of screening with a diagnosis of either large-duct PSC or small-duct-only PSC may be eligible if they meet the other specific criteria. Those currently taking ursodeoxycholic acid or oral vancomycin for treatment of their PSC may remain on it. The purpose of the ChiLDReN study is to collect medical and other data to learn more about PSC, how it progresses, and identify factors that may cause the disease to progress more quickly; and to ask questions about how PSC symptoms affect a child's life to learn more about its impact on their daily functioning. Also, participants who are seen at one of the clinical sites will be asked to contribute information, DNA, and other specimens. The information and specimens will be available to investigators to carry out approved research aimed at learning more about the possible causes and long-term effects of PSC. Detailed information about the study and participating sites can be found at clinicaltrials.gov , including site locations, the site’s contact person, and their contact information (scroll down to “Locations”). Please contact them directly for specific study information. Given the rarity of PSC in young people, it is imperative that investigators work together across treatment centers in order to have study cohorts that are large enough to draw statistically sound conclusions. PSC Partners is extremely grateful to the investigators working on this study for their collaborative approach to gathering this important data, and to the NIH for funding the study. *through the National Institute of Diabetes and Digestive and Kidney Diseases program (NIDDK) Gilead's PRIMUS trial is terminated early On September 27, 2022, Gilead Sciences announced the early termination of the PRIMUS study, its phase 3 study of the investigational drug Cilofexor. Although there were no reported safety issues, the trial evidence to date did not show that the drug was effective for PSC. The decision to stop the trial was based on the results reached after the first 160 participants had completed Week 96 of the trial. The primary endpoint was improvement of fibrosis measured with a biopsy. Investigators saw no benefit to continuing the trial. You can read the Gilead statement here . This is certainly disappointing news. However, a “failed” trial is not a failure if lessons are learned that prove helpful going forward. Especially when a disease mechanism is unclear, such as in PSC, an unsuccessful trial can provide clarity regarding the disease process. Other times, the trial design may prove faulty even though carefully planned; such mistakes are informative to future trials and may even provide new information about the disease itself. PSC Partners continues its collaboration with Gilead and hopes that they will persist in seeking a treatment for PSC. If you took part in the PRIMUS study, you are deeply appreciated. The research process is lengthy, difficult, and sometimes disappointing; however, without study participants, it would go nowhere. Patients willing to participate in clinical trials are key to the ultimate goal of treatments and a cure for PSC. For more information, please contact Gilead Public Affairs at public_affairs@gilead.com . How can clinical trials become more patient friendly? Your input is needed! PSC Partners frequently communicates with PSC researchers and drug developers about patient-friendly trial design. Too often, the burden of travel, invasive testing, too-stringent inclusion/exclusion criteria, and more, limit the number of patients willing to enroll in a trial, which naturally slows the pace of research. Have you ever participated in a clinical trial? Wanted to but didn’t meet the inclusion criteria? Met the criteria but found the requirements too burdensome or the potential risk too high? We want to hear from you! Some of you may have participated in a trial and had a positive and rewarding experience. We would love to hear from you, too. It would help to know what the trial team did right. Your comments about both positive and negative trial experiences as well as concerns or limitations that may keep you from enrolling in a clinical trial are valued. You can contribute to the patient voice in shaping patient-friendly trials. You can share your thoughts by email at registrycoordinator@pscpartners.org OR, if you prefer to be anonymous, you can fill out this two-question Google Survey . If you email, Sharon or Rachel will be responding to you. Some points you may want to address:                         Considerations about inclusion and exclusion criteria, which are meant to ensure trial safety and uniformity in testing Thoughts on the trial drug Thoughts about frequency and type of testing Logistics such as travel frequency and time spent at the trial site Comments on health impact incurred by the trial Communication quality regarding consent documents, your role in the trial, and/or availability of the trial team to answer questions Compensation for expenses incurred or payment for your time Access to study results Fears, concerns, and/or positive experiences The chart below shows the percentage of Registry Clinical Survey respondents who were currently participating in a clinical trial related to PSC at the time they completed the survey (blue bar) or who had previously participated in a PSC clinical trial or donated a biospecimen for PSC research (red bars). The green bars signify the percentages of respondents who indicated that they are willing to be contacted in the future about PSC trials and biospecimen donation for PSC research. Receiving information about trials and studies does not obligate participants to enroll or donate.  The relatively low figures for current or past participation raise some questions: Are too many patients not a match for clinical trials? Are researchers unable to reach those who could be a match? Are clinical trials not easily accessible to people with PSC? No matter what the reasons are, these figures show that people with PSC are very willing to participate in research. Thank you for taking a few minutes to email us at registrycoordinator@pscpartners.org OR respond to the anonymous Google survey with your comments. The PSC community has been sharing thoughts and concerns through surveys and discussions. PSC Partners brings your de-identified input to the attention of researchers and trial developers when reviewing trial protocols, with the intent of helping them design trials that are as patient friendly as possible. Highlights of the 2022 International Liver Congress PSC Partners Board Member Jesse Kirkpatrick, PhD, has chosen to pursue a career focused on PSC, following a family member's PSC diagnosis. Now a Harvard medical student and postdoctoral researcher at the Massachusetts Institute of Technology, Dr. Kirkpatrick is conducting PSC research with the goal of answering key unsolved questions in PSC. He recently attended the 2022 International Liver CongressTM (ILC), which was hosted in London by the European Association for the Study of the Liver (EASL), and shared a few of the highlights with the Registry team. Dr. Kirkpatrick described the recently updated EASL Clinical Practice Guidelines* on sclerosing cholangitis as “one of the most relevant” topics discussed. The EASL PSC guidelines  were published in the Journal of Hepatology in September 2022 and a brief summary, i ncluding EASL's strong recommendations, can be found here . Dr. Kirkpatrick also noted that the conference included a symposium on bile acids featuring three cholestatic liver disease experts. He particularly highlighted Dr. Binita Kamath’s talk about the work being conducted around ileal bile acid transporter (IBAT) disruption. IBATs are molecules involved in the reabsorption of bile acids from the intestine, and novel drugs targeting IBATs show promise for treating the itch associated with liver disease. “It sounds like relief is coming for PSC patients with pruritus,” he said. A poster about a multinational pruritus study in which PSC Partners participated was presented at the ILC by Dr. Kris Kowdley of Washington State University. PSC Partners CEO Ricky Safer, Registry Director Rachel Gomel, and Medical Liaison Joanne Hatchett are among the co-authors. You can find a link to the poster abstract on the Registry’s new “Publications Related to Clinical Trials” page , which is accessible to both Registry participants and other patients with PSC interested in joining clinical trials. Look under the Mirum trial heading for the link to the 2022 Kowdley article in the Journal of Hepatology . The 6th Biennial Meeting of the International PSC Study Group (IPSCSG) also took place at the 2022 ILC. The IPSCSG’s aim is to coordinate PSC research projects between leading institutions worldwide. Jesse Kirkpatrick is a member of the IPSCSG, as is Ruth-Anne Pai of PSC Partners. Dr. Kirkpatrick is an integral part of PSC Partners’ efforts to expand its global reach through the International Collaborative Research Network initiative and informational webinars featuring international research and advocacy partners. *The American Association for the Study of Liver Diseases (AASLD) has also recently published a PSC Guidance, which can be found here . PLEASE NOTE: While meetings such as the recent EASL ILC are for scientists, professional organizations are increasingly becoming more accessible for patients. If you are interested in following EASL and accessing its scientific publications and online resources, EASL has announced a new patient membership option at a cost of €25 (Euros, about $24 US). Visit their membership page for more information. If you are interested in attending scientific meetings, the AASLD is hosting The Liver Meeting® in Washington, DC, November 4-8, 2022, and registration for patients/caregivers is available for both in-person and virtual attendance at a very reduced rate of $50. Most of the content is aimed at researchers and clinicians, but there is a patient/caregiver track as well. The sessions cover all things liver-related; cholestatic liver disease is a small part of the agenda. Register here . Meet Director of Research Strategy Ruth-Anne Pai, PhD When doctoral candidate Ruth-Anne Pai was matched with PSC Partners as a scientific advisor through the Chan Zuckerberg Initiative’s Rare As One (RAO) project, little did she know that she had found her next professional home. After advising the organization for a few months, Ruth-Anne discovered that PSC Partners was planning to hire a director of research strategy.  “The role aligned so well with what I hoped to do after my PhD [studies] and I was thrilled when offered the opportunity to position my scientific training to support the PSC community,” Ruth-Anne said. She joined the PSC Partners staff in April 2021. A native of eastern Pennsylvania, Ruth-Anne earned a BA in Biological Sciences at Cornell University and a PhD in Immunology from the University of Pennsylvania. Her doctoral dissertation included identification of two new treatments for an idiopathic form of Castleman disease – another rare disease with liver involvement. Her mentor at UPenn, Dr. David Fajgenbaum – himself a Castleman patient – was the keynote speaker at the 2022 PSC Partners annual conference. “I’ve had the amazing honor of working with Ruth-Anne Langan Pai over the years on Castleman’s Disease,” he said in his conference address , “and I’m just so thrilled that she is the research director for PSC Partners doing such important work.” Ruth-Anne’s focus at PSC Partners is on several new initiatives. She has a leadership role in crafting a Strategic Research Plan and launching the International Collaborative Research Network ( ICRNetwork ); and she is integrally involved developing the Worldwide Integration of Natural History Databases ( WIND ) project and a new PSC-specific patient-reported outcome measure (PROM). “Having Ruth-Anne Pai among us has been a game changer!” said Registry Director Rachel Gomel. “She has led the PSC Partners leadership team to embark in patient-driven research and has helped us bring the patient voice to the forefront." Ruth-Anne’s dedication to research was inspired by her ninth-grade biology teacher, who instilled in her “a love for scientific exploration through science fair.” Ruth-Anne developed a specific interest in immunology at Cornell, where she decided to focus on human disease. As a new PhD student at UPenn, Ruth-Anne was offered a lab rotation opportunity working with Dr. Fajgenbaum on Castleman disease research. “Dr. David Fajgenbaum taught me to always remember the needs of the patient community I serve and to work with urgency and purpose every day,” she said. When asked what insights into PSC her background in immunology provides, she replied, “There are many parallels between the work and skills I gained during my PhD [studies], and the research landscape in PSC, from the challenges of studying a heterogeneous rare disease to the importance of utilizing a multiomic approach to study early disease mechanisms.” “At PSC Partners, my ultimate goal is to provide all of the support I possibly can to accelerate the discovery and development of treatments and a cure for PSC,” said Ruth-Anne. “While one aspect of this is to develop key research projects and collaborations, I aim to support the PSC community in any way that leads to improved quality of life and outcomes.” Ruth-Anne Pai presented a poster illustrating PSC Partners' Roadmap Initiative at the CZI Rare As One meeting in June 2022.  As a researcher, Ruth-Anne understands the significance of the PSC Partners Registry. “Participation from the patient and caregiver community is so critical in that it enables the completion of clinical studies and is an incredible resource for PSC researchers,” she said. “We are so grateful to current and future participants in the Registry, who are active and crucial contributors to our quest for a cure for PSC.” Ruth-Anne presently lives near Seattle, Washington, with her husband, where they enjoy trail-hiking and traveling to visit family. Don't forget to check the Registry Clinical Trials page for updates on active clinical trials! Click image or  here to visit the Registry list of PSC ongoing clinical trials. “Together, we are creating a world where a PSC diagnosis comes with a cure!” Established in collaboration with the Office of Rare Diseases Research ( ORDR ), the National Center for Advancing Translational Sciences (NCATS), and the National Institutes of Health (NIH) © 2022 PSC Partners Seeking a Cure

A new survey is available in the Registry: the PSC Flare or Cholangitis Attack Patient Survey . Why take a survey about PSC flares/cholangitis? Though those who live with PSC may know what a cholangitis attack feels like, surprisingly, the medical world has not agreed on a definition. That is why researchers need information from adult PSC patients about their experience with PSC flares/cholangitis. We request that patients, not caregivers, take this survey. If you have ever experienced a PSC flare or cholangitis attack, whether your symptoms were mild or severe, please log in today to complete this survey! Note: If you have never experienced a flare, or if you have previously completed this survey via Google Forms (see below), you don’t have to take this survey. If in the first few questions you respond that you took the survey before, or that you’ve never had a flare/cholangitis attack, the survey will stop there. If you are a Registry participant, by taking a couple of minutes to let us know that you have taken the survey already, we will know not to send you reminder emails to complete the survey. If you’re not sure whether you previously took this survey, here is what the start of the survey looks like in Google Forms. If you are not a Patient Registry participant, you are encouraged to join today  and take this survey in the Registry after completing your account setup. If you do not wish to at this time, you can instead complete the PSC Flare or Cholangitis Attack Patient Survey  through Google Forms. No identifying information (such as name, phone number, address, date of birth) is collected. Click on the SINGLE USE SURVEY button above to leave the Patient Registry site and complete the PSC Flare or Cholangitis Attack Patient Survey   outside the Registry . About the Survey The survey will capture your symptoms before, during, and after your most recent PSC flare/cholangitis attack. This information will help characterize the prevalence and frequency of symptoms and the impact of these episodes on people living with PSC.  The survey will also include questions about the treatment and care you received, if sought, during your most recent PSC flare/cholangitis attack. Understanding your symptoms through your first-hand experience and from treatments you received during these episodes will help inform clinical trial design and is an important first step in establishing a better standard of care for those living with PSC. To access the survey, log into the Registry here  and find it on the Dashboard or in the Surveys tab on the left menu. Again,   patients should complete the survey for themselves. Caregivers should not complete it on behalf of their patient. This survey has been developed by PSC Partners  (based in the U.S.) and PSC Support  (based in the U.K.) as part of their work with the PSC Forum Acute Cholangitis Working Group. Thank you for your participation, which is critical in advancing PSC research.  Rachel Gomel, Patient Registry Director Together, we are creating a world where a PSC diagnosis comes with a cure!