The VIADUCT - Issue 8
- PSC Partners Patient Registry
- Apr 9
- 5 min read
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
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 into reality. The time has now come for me to pass on the baton to teammate Mary P Vyas, serving as Registry Strategist to date and involved in numerous PSC Partners initiatives. A Berkeley, M.I.T., and Stanford University graduate and President of PSC Partners Canada, she brings her creativity and action-minded spirit to leading the team as Registry Director. I will continue as Senior Registry Advisor. The Registry is in great hands.
We continue to be very excited about the Registry’s move to our new and much-improved home. The new platform holds state-of-the-art features to support research and Registry participants, maintains tight security, and is managed by the PSC Partners Registry team of super experts (each with a connection to PSC) who guard your precious information and turn it into research.
Now that we’ve settled, the Registry Team hopes to receive those of you who haven’t yet made the move to the new Matrix platform. It is an easy task: on the Registry website pscpartnersregistry.org, take the simple steps to log in,* complete the updated 2025 Clinical Survey, and explore the Registry’s new features.
It is crucial that you make this move, that you complete the new surveys and enter your voice into research. We hope to show you the highlights and impact of your participation. With your enthusiastic participation, PSC research can only flourish.
Together, we are creating a world where a PSC diagnosis comes with a cure!
Rachel Gomel, Senior Advisor, PSC Partners Patient Registry
*The site supports Chrome, Edge, and Safari browsers. Please note that the first time that you log into the upgraded site, you will have to reset your password using the “Forgot your password?” link on the login page. You will be asked to review and complete a general profile information form.
Take the PSC and Cholangiocarcinoma (CCA) Survey
A new survey is available in the Registry, focusing on screening and surveillance for bile duct cancer (cholangiocarcinoma, or CCA) in people living with PSC. Anyone with a PSC diagnosis, including post-transplant, can and are urged to take the survey through the Registry. Log in and complete this survey today! Responses are requested by April 14th.
Trailblazing WIND-PSC Study is Enrolling Patients and Adding Study Sites
The 5-year prospective study, led by PSC Partners, collects data from enrolled patients to act as a "synthetic placebo" group for a clinical trial. This opens up new pathways to approving treatments for PSC and can reduce the enormous costs associated with clinical trials.
Investigational Drug for PSC Advances to Phase 3 Trial
Few investigational PSC drugs have, to date, moved forward to a Phase III clinical trial. For a drug to do so, it must first meet initial trial goals surrounding safety, efficacy, and identification of side effects. Chemomab Therapeutics, an Israeli clinical stage biotechnology company, has announced plans to move forward with a Phase III clinical trial of nebokitug (CM-101).
The planned Phase III trial will assess changes in the “time-to-first-event” to typical PSC clinical events, such as cholangitis attacks, strictures requiring intervention, portal hypertension, etc.. and will not require liver biopsies. Trial participants will stay on nebokitug until they experience such a clinical event endpoint.
PSC Partners issued a statement with more information about the announcement, including the Chemomab press release.
Trials and External Surveys in Need of Participants
Are you interested in helping to advance PSC research by participating in a trial or completing a survey?
Two prospective drug trials are in need of additional U.S. patients. The research team is led by Dr. Josh Korzenik, director of the Crohn's and Colitis Center and director of the Resnek Family Center for Primary Sclerosing Cholangitis Research at Brigham and Women's Hospital.
Additionally, two surveys are available for those living with PSC. These surveys are not hosted in the Registry, but are also offered through secure and confidential platforms. Read more about the surveys and – if you haven’t yet – complete them today!
Open globally: PSC and Quality of Healthcare Survey (University of Miami)
For Europeans: PSC and Diet Survey (European Reference Network)
The Symptom Assessment Project: Building New Endpoints for Clinical Trials
Three common PSC symptoms – fatigue, brain fog, and liver pain – lack research-quality assessment tools. The PSC Partners-led Symptom Assessment Project aims to fill that gap.
Registry Superstars
The Registry team would like to spotlight and thank those Registry participants who have been especially active in the Registry. Because identifying information NEVER leaves the Registry, we can’t share their names here, so we will email them individually to thank them for their loyal participation in the Registry.
19 Registry Superstars have completed 10 or more surveys as of December 2024. 180 Rising Stars have completed 5 or more surveys. With 4 currently active surveys in the Registry (not counting the Healthcare Provider Map Submission Form), a new participant can become a Rising Star as soon as two consecutive years of Clinical Surveys have been completed!
Active users demonstrate they recognize that patients CAN accelerate the trajectory of PSC research. There IS hope for better treatments and a cure for PSC, and every active Registry member plays a powerful role.
We encourage YOU to become one of our stars this year as, together, we are creating a world where a PSC diagnosis comes with a cure!
Learning from Registry Data: Relatives with PSC
People living with PSC often ask, “What is the chance that my child develops PSC?” Studies have shown that PSC does have a genetic component, but developing PSC is not likely even if a family member has PSC.
To better understand the family link in PSC, the Registry team analyzed Clinical Surveys from 2,182 Registry participants. Here’s what we found:
1.74% (38) of participants reported a parent or child with PSC.
4.48% (96) reported a blood relative with PSC (including immediate or extended family).
This does not mean that there is a 1.74% chance that a child of someone with PSC develops PSC: because this analysis does not account for the number of children in a family, the true chances are likely lower.
For comparison, 11.6% of Registry participants reported a parent or child with IBD, and 16.6% reported a blood relative with IBD.
Thank you to everyone who has completed a Clinical Survey to make these types of analyses possible! If you know a relative or acquaintance living with PSC who is not yet in the Registry, we encourage you to ask them to join today.
An EASY action to safeguard your account!
If this newsletter reached your inbox, the Registry has your correct email. To be sure that we can always reach you:
1) Log into your Registry account* and confirm all of your contact info in the General Information section.** Consider adding an optional email address, which is used in case your primary email address is no longer valid.
2) If your contact information changes, let us know! Please do not make a second account! If you have trouble accessing your account, the Registry team can help.
Thank you for helping the Registry team keep you connected and up to date.
*If you have an older account and haven’t logged in during 2024 or 2025, you will need to reset your password, as the Registry moved to an upgraded platform in early 2024. The Registry supports Chrome, Edge, and Safari browsers.
**You can edit all demographic info except your email address. To update an email, please contact us at registrycoordinator@pscpartners.org, and the Registry team will change it.
