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The VIADUCT Newsletter Issue 6

Issue 6 - June 2023

In this issue: From the Registry Director • New CZI Grant Awarded to PSC Partners • Patient Focus Groups Provide Feedback • Oral Vancomycin & Insurance Coverage • European Clinicians Survey Results • Call to Action: What is the Registry Not Asking? • Highlights: Clinical Trials & Studies

From the Registry Director

Dear Registry Participants,

PSC Partners and the PSC Partners Patient Registry are moving at rapid speed through involvement in large international projects; increased patient engagement in producing new patient data through the Registry; and in growing clinical trial participation.

Nothing happens in a vacuum. Your involvement, your participation is creating a snowball effect that transcends geographic boundaries.

In this issue of the VIADUCT, we bring to you the news of a large grant received by PSC Partners in collaboration with a Toronto multicenter research team that is studying PSC at the cellular level.

You will find a report on a survey implemented in the Registry regarding insurance coverage of oral vancomycin. We’ve also reviewed the thought-provoking results from a European study and expand on the impact of the Registry on the PSC research community.

You will find an overview of several clinical trials for PSC that are currently enrolling.

We need your help as the Registry team plans for a future Clinical Survey update. You’ll find a link to an anonymous Google survey where you can provide your suggestions. What questions aren’t being asked in the Registry surveys that you think should be asked?

We cannot emphasize enough the value and the importance of your participation. We wish we were together in person, openly expressing our yearning for a cure. As you quietly complete surveys and participate in trials, we hear your voice loud and clear. Never hesitate to ask us your questions. We’re together in our relentless search for a cure.

On behalf of the Registry team, Rachel Gomel, Director, PSC Partners Patient Registry

New CZI grant awarded to PSC Partners for Collaborative Research Project

In December 2022, PSC Partners was selected for new four-year funding by the Chan Zuckerberg Initiative (CZI) for a unique patient-partnered study to be conducted by PSC Partners and a Toronto multi-center research team. The grant will support a research initiative aimed at advancing understanding of the fundamental biology underlying pediatric PSC and PSC-IBD.

This CZI award is truly unique for PSC Partners, as this is the first time that the organization has applied for funding for a scientific proposal as partners with an academic research team.The project is led by two principal investigators (PIs). PSC Partners Canada President and Registry team member, Mary Vyas, will serve as the patient organization PI, and Sonya MacParland, PhD, senior scientist, Ajmera Transplant Centre in Toronto (part of the Canada-wide University Health Network), will be the Canadian research team’s coordinating PI.

Members of the team leading this innovative research project include, (L to R), Mary Vyas, President at PSC Partners Canada, Drs. Sonya MacParland, Senior Scientist at UHN's Ajmera Transplant Centre, Amanda Ricciuto staff gastroenterologist at SickKids, and Gary Bader, affiliate scientist at the Princess Margaret Cancer Centre. (Photo: UHN)

The project, “Understanding the Cellular Ecosystem of Primary Sclerosing Cholangitis,” will focus on clarifying the cellular mechanisms of pediatric PSC-IBD and identifying potential cellular targets for effective therapies.

Up to two million dollars will be awarded for this initiative. This includes a budget for PSC Partners to provide patient-focused input at every step of the project, support participant recruitment, and to oversee knowledge translation to the patient community. Most of the funding will pay for advanced and expensive technology to execute this single-cell research study. The research funds will go to the collaborating Toronto institutions: University of Toronto, The Hospital for Sick Children, University Health Network, Toronto Centre for Liver Disease, Ajmera Transplant Centre, and Mt. Sinai Hospital.

Your participation and the large number of participants in the Registry attracted attention to PSC, despite it being such a rare disease, and, in doing so, played a vital role in the granting of this competitive CZI award. When Dr. MacParland first learned of the PSC Partners Patient Registry about five years ago, she became excited about the research possibilities. Having access to the de-identified data of over 2,000 patients supported her decision to focus her research efforts on PSC and led to an exciting relationship between PSC Partners and her team of researchers. 

For several years, PSC Partners Canada President Mary Vyas, Registry Director Rachel Gomel, and PSC Partners volunteer Katie Bingham, PhD, have been serving as integrated patient collaborators¹ on a large-scale PSC study led by Dr. MacParland that compares PSC liver tissue to healthy liver tissue.

Click on the video to view a 3-minute interview from 2021 with Dr. Sonya MacParland, whose work focuses on translating the immune biology of the liver into clinical applications - and how the PSC Partners Patient Registry helps advance that goal.

“Beyond incorporating the patient perspective in research design, our partners ensure that our work is grounded in the patient experience,” said Dr. MacParland.²

The relationship existing between the research team and PSC Partners laid the foundation for the application for the most recent Patient-Partners Collaboration CZI grant by providing evidence of our ability to work well as a collaborative team.³

The award is a major milestone for the PSC patient community. To date, PSC Partners has pursued its mission to drive research to identify treatments and a cure for PSC through the WIND-PSC and PROM initiatives and through awarding seed grants to promising researchers. This is the first time that PSC Partners is a grant recipient for a project in which the patient organization plays a formal collaborative role in a scientific project. PSC Partners' new role of being a grantee, besides being a grantor, represents a momentous change in PSC research.

¹An integrated patient collaborator is the title given by the Canadian Institutes of Health Research. ²As quoted in a UHN press release about the grant award. ³Further details about the CZI “Patient-Partnered Collaborations for Single-Cell Analysis of Rare Inflammatory Pediatric Disease” grant can be found here.

Patient Focus Groups Provide Feedback for WIND-PSC Initiative

Last fall, as part of the planning for the WIND-PSC initiative, PSC Partners recruited volunteers to participate in focus groups to discuss existing patient symptom surveys. The volunteers all belong to the Patient Registry and have personal experience with common PSC symptoms, including fatigue, sleep disturbance, cognitive dysfunction (brain fog), itch, abdominal or liver pain, and depression or anxiety.

PSC Partners staff members, Medical Liaison Joanne Hatchett, and Research Program manager Sarah Curup Callif, organized the recruitment effort and were encouraged by the number of people who showed interest in participating. “It really reaffirmed that our community is so excited. They want to make a difference. They want to be involved,” Joanne said.

Participants represent the U.S. and Canada and were assigned to one of two focus groups. Prior to meeting, each person received copies of various existing symptom surveys to review and compare. Such survey instruments, called Patient Reported Outcome Measures (PROMs), will be used in the WIND-PSC registry to capture participants’ symptoms over time.

In November, each focus group met separately via Zoom with qualitative  research expert Donna Evon, PhD, of the University of North Carolina, to begin the process of selecting patient symptom surveys to be included in the WIND-PSC study.

The focus groups reviewed, discussed, and compared the various PROMs to determine which they thought best capture and reflect PSC symptoms. They also reflected on the process of administering PROMS and similar surveys.

Both Joanne and Sarah were extremely pleased with the quality of the input received. “The people who were there were amazing,” said Joanne. “They were so attentive to the details of what they were looking at, how they felt, and how they expressed their thoughts.”

“It helps us move forward with confidence,” said Sarah.

The participants’ input will form the basis for a larger, future community and Registry survey that will be submitted for publication and will help make decisions about which existing PROMs to use in the WIND-PSC initiative.

“Their impact made a difference, whether it was filling out an interest form, attending a focus group, or even just being aware,” said Sarah, "It really helps us to move forward…it seems like within the PSC community, people are so eager to help and to participate. We are thankful. We just can’t thank them enough!”

WIND-PSC is one of two multi-year projects being developed by PSC Partners International Collaborative Research Network (ICRNetwork). The other is the development of an improved PSC-specific PROM that will better inform clinical trial research, a need confirmed by the focus group members.

Dr. Evon and Director of the Center for Health Measurement at Duke University Bryce Reeve, PhD, are collaborating with PSC Partners and the ICRNetwork on the PROM project.

The WIND-PSC and PROM initiatives are concurrently moving forward.

Oral Vancomycin and Insurance Coverage: Data Results From Recent PSC Partners Survey

In late February, the Registry launched a short-term survey, “Oral Vancomycin and Insurance,” in which respondents were asked to share their experiences with oral vancomycin and insurance coverage. Emails were sent to all Registry participants who had indicated on their Clinical Survey that they are or were taking vancomycin for PSC, and the survey was promoted on the PSC Partners social media pages.

The de-identified data was aggregated and is available for you to view in the Resources/News section of the Registry website

The purpose of this short-term, focused survey was to provide data to PSC community members who were meeting with lawmakers and others on Rare Disease Day, February 28. This is the reason it was only available for a short period of time. Many thanks to Registry Strategist Mary Vyas for implementing the survey and for the quick turnaround of the data analysis!

By launching this survey through the Registry, data integrity, informed consent, and patient privacy in an IRB-approved environment were maintained. Collecting data in the way that the Registry does, with IRB oversight, increases the scientific merit of the data, especially with respect to the ability to publish in a peer-reviewed journal.

The PSC Partners Registry team is very grateful to the many community members who have joined the Registry and who respond to calls for participation.

Your participation is even more valuable when your Registry account is complete and up to date. If you take oral vancomycin for PSC and did not receive an email invitation to complete the survey, perhaps your Clinical Survey or your email address needs to be updated.

Please return regularly to your Registry account and add or update anything that is missing. Check your profile information for accuracy, upload a document confirming your PSC diagnosis if you have not already, and update your Clinical Survey and the SF-36 (quality of life measure) yearly or when you have changes in medical status.

Please email if you have any questions, are unable to access your account, or would like help uploading a document. Please do not create a duplicate account – we are happy to help you access and update your original account.

European Clinician Survey: PSC Treatment Varies Among Providers

Results of a survey published in late 2022 show that there is “substantial variation” among European clinicians in the treatment of PSC and “considerable discrepancies” between practice guidelines and clinical management.

PSC researchers from the European Research Network Rare-Liver group (ERN RARE-LIVER) surveyed European hepatologists affiliated with the ERN and/or the International PSC Study Group (IPSCSG) to find out about differences in PSC management and whether clinicians adhered to the clinical practice guidelines set forth by the European Association for the Study of the Liver (EASL).

In the full-text research publication, “Survey uncovering variations in the management of primary sclerosing cholangitis across Europe,” the authors state, “There is a scarcity of data that consistently support the use of medical therapy to prevent disease progression and prolong transplant-free survival in PSC.”

The researchers believe that identifying the differences in PSC management is essential for optimizing treatment and, therefore, they conducted the survey to “uncover differences in real-life clinical practices.”

The study report gives the following summary highlights, as quoted below:

  • Substantial variations in the treatment and monitoring of patients with PSC was seen across Europe.

  • Considerable discrepancies between practice and published guidelines in the management of patients with PSC existed.

  • Despite no robust evidence or clear recommendations, most [European] physicians [surveyed] treated all their patients with UDCA [ursodiol].

  • Regular screening for cholangiocarcinoma was performed by 90% of physicians. A variety of screening methods were used.

  • In PSC without IBD detected at diagnosis of the bile duct disease, most physicians would repeat colonoscopy on a regular basis.

The report also provides details and graphics of their findings. In the table below, a few of the study findings are compared to aggregate PSC Partners Patient Registry data.

European Study Report

Registry Data

UDCA (Ursodiol)

82% of physicians who responded prescribe UDCA either routinely (50%) or selectively (32%) to their PSC patients.

80.6% of survey respondents have taken UDCA at some point. 

Oral Vancomycin

16% of physicians who responded treat selected PSC patients with oral vancomycin.

18.5% of survey respondents have taken oral vancomycin for PSC at some point.


34% of physicians who responded ordered a colonoscopy every 5 years for patients with PSC but no IBD at diagnosis, and another 34% repeated the test within 3-5 years. 27% of physicians only referred patients for repeat colonoscopy if they developed symptoms of IBD.

93% of survey respondents have had one or more colonoscopies, and 68.4% are expecting to have one annually or biannually.

Authors of the study conjecture that PSC management may be even more disparate than the study findings, since all respondents belong to the ERN-RARE and/or the IPSCSG and thus are likely to be somewhat more aligned in their clinical practices.

They concluded that their results “confirm that existing data and recommendations for clinicians are inadequate for uniform patient management” and proposed that “established international networks and research associations should work to coordinate guideline recommendations in order to generate a better basis for developing clinical trials and to improve the daily management of patients with PSC.”

EASL and AASLD practice recommendations

In 2022, the U.S. and the European professional hepatology associations each published updated recommendations for the diagnosis and management of PSC. You may wish to review these informative documents.

Click on images below to read the EASL guidelines and AASLD guidance.

Call to action for all patients and caregivers: What is the Registry not asking?

Periodically, the Patient Registry’s main Clinical Survey is updated to include new questions posed by PSC research. Questions that can provide additional insight into the PSC experience are added, and prior survey responses are carried over to maintain continuity. This is the time for you, patients and caregivers, to provide input as new versions of the survey are developed.

What is the Registry currently not asking? This Google survey prompts you to consider areas of possible concern and offers a way to share your ideas on the Registry survey.

Your perspective about how PSC impacts you is invaluable and unique. If you would like to provide suggestions, they will be considered very seriously. TO REMIND YOURSELF OF THE QUESTIONS IN THE CURRENT CLINICAL SURVEY:

Login to your Registry account,* go to your PROFILE dashboard, and either:

  • Update the Clinical Survey, if you haven't updated in the past year (click on “Update survey”).

  • OR, if you've already updated your information within the last year, click on "Download Results" next to the survey name to view questions and your responses.

While plans for an update to the Clinical Survey are still in the early stages, your input is needed now. The Google survey can be completed here.

Thank you in advance for sharing your thoughts and for engaging in shaping PSC research!

* Do you need help accessing your account? Just reply to this email ( ) and we will help you. Please do not create a new account! 

Highlights: Clinical Trials and Studies

An important function of the Registry is to assist in connecting PSC patients with clinical trials or studies for which they may be eligible or simply interested in following, and the Registry team strives to do so through emails, publications, and a dedicated web page. Many PSC trials and studies are underway. Some are highlighted below, and you are encouraged to visit the Registry’s clinical trial page often to learn about others. 

The ELMWOOD Study - enrolling patients now: The Elmwood study, sponsored by Ipsen Pharmaceutical, is an interventional study of the drug Elafibranor, a drug originally tested for use in steatohepatitis (NASH) and cardiometabolic conditions. It is now being tested for safety and efficacy in PSC. Study centers are located in the U.S., Canada, and Spain. For full information, including contact information, click here

The PACIFIC study - enrolling patients now: There is an increased interest in researching PSC pruritus (itching), as it is frequently mentioned by patients as one of the most difficult PSC symptoms. The PACIFIC study, sponsored by Escient Pharmaceuticals, is focusing on pruritus and is a phase 2 interventional trial of the drug EP547 for use in PSC. The Escient website states that EP547 has a “novel, targeted mechanism of action” and “blocks the activation of itch neurons by all bile acids and bilirubin.” Seven U.S. study locations are located in California, Mississippi, New York, and Texas, and are open to patients with PSC, ages 18 to 80, who have moderate to severe pruritus. Additional information and qualifying criteria can be found here.

The SPRING study - enrolling patients now: Sponsored by Chemomab Therapeutics, the phase 2a SPRING study is an interventional trial that is currently recruiting for 45 study centers in the U.S., U.K., Israel, Germany, and Spain. The Chemomab website states, “Chemomab has established strong pre-clinical evidence that demonstrates the potent anti-cholestatic and anti-fibrotic effects of CM-101 in PSC.” CM-101 is a monoclonal antibody delivered by intravenous infusion every three weeks.The web page above includes a video link that explains CM-101’s mechanism of action in PSC.

The VISTAS study - enrolling patients now: Sponsored by Mirum Pharmaceuticals, the VISTAS study is testing their drug volixibat for pruritus. The pediatric version of the drug, Livmarli, was recently approved by the FDA for Alagille Syndrome, a rare pediatric bile duct disease. Over 50 trial sites have opened throughout the US, in Canada, Germany, UK, and Israel. All patients are given the opportunity to take the drug. You can find out more about the trial here.

The ItCH-PSC study - enrolling patients now: The ItCH-PSC study is a basic research study with an aim of understanding more about the frequency, severity, and persistence of itching in patients with PSC.

The principal investigator is Dr. Chris Bowlus of UC Davis in Sacramento, and six study centers¹ are participating throughout the U.S. Participants will need to travel to the study center up to three times. Inclusion criteria shared by the researchers state that the study is open to all adult patients with PSC (18 years of age and older) who have not had a liver transplant and who do not have concomitant liver diseases other than IBD and autoimmune hepatitis. There is no experimental drug intervention involved; participants will have blood draws and fill out questionnaires. If you are interested in learning more, please fill out the ItCH-PSC interest form.

¹ItCH-PSC participating study sites are located in the U.S. at UC Davis, Sacramento, University of Colorado, Aurora; Henry Ford University, Michigan, Indiana University, Indianapolis; University of Miami, Massachusetts General Hospital, Boston. If you live near or can travel to one of these sites and are interested in learning more about becoming involved with this study, please complete above interest form.

How do you know if you meet the criteria for a trial?

Most interventional studies (clinical trials) are listed at . Interventional studies are testing a drug, device, procedure, or activity, and both the inclusion and exclusion criteria are posted on the site. For the studies listed above, most have complete details listed on the ClinicalTrials site. Links are embedded above.

Every trial or study has inclusion and exclusion criteria for participation. Though patients are sometimes frustrated if they don’t meet the criteria, there are important reasons for these, including the need for uniformity in the test group and, especially, safety. Certain medical conditions might put a patient at greater risk.

If you don’t qualify for an interventional trial, you can still help further research by joining other types of studies and by participating fully in the Patient Registry. A strong, “A+” Registry profile has every survey completed; the Clinical Survey and SF-36 Quality-of-Life Survey updated within the last year; an attached medical record confirming that you have PSC, your healthcare provider added to your record, and, of course, complete and accurate contact information.

Are you receiving Registry emails about trials?

Is your Registry profile information correct? If you received this newsletter via email, congratulations – your email address is up to date! If you accessed this through the Registry website or social media, please check the email address listed on your Registry profile to ensure that it is correct. Whenever the Registry team sends out emails about trial opportunities, about 5-10% of them are returned to us because the email address is no longer valid.

Depending upon your answers to the patient surveys in the PSC Partners Patient Registry, you may receive occasional emails with information about clinical trials that are recruiting PSC patients. Have you filled out each survey and kept them up to date?

Receiving an email does not obligate you to participate or to contact the researchers. It is for your information.

Whether or not you are able or choose to participate personally, you are urged to visit the Registry’s Clinical Trials page frequently to learn about the many ongoing research efforts geared towards better treatments for PSC.

The research process is lengthy, difficult, and sometimes disappointing; however, without study participants, it would go nowhere. Patients willing to participate in research studies are key to the ultimate goal of treatments and a cure for PSC. On behalf of the PSC community, we thank you for having participated or for planning to participate.


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)

© 2023 PSC Partners Seeking a Cure


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