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 elgible. 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.

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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.

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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.

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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: 

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• 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).