Completed Digestion, Kidneys & Other Organs NIHR-supported project

SABR- PSC Pilot study A single arm pilot study of Brodalumab in the treatment of Primary Sclerosing Cholangitis

In plain English

AI plain-English summary

A small group of patients with a rare liver disease will test a drug that blocks a specific immune signal, to see if it can be given safely and practically. This matters because primary sclerosing cholangitis (PSC) is a chronic condition where the bile ducts become inflamed and scarred, often leading to liver failure. There is no approved treatment that slows the disease. Many patients also have inflammatory bowel disease (IBD), and existing options are limited. The drug, brodalumab, inhibits a protein called IL-17 that drives inflammation in several autoimmune diseases, but its potential in PSC has not been tested in a clinical setting. If the pilot shows that brodalumab is safe, tolerable, and feasible to administer, it would open the door to a larger randomised trial. Success could eventually give patients a first-ever treatment that targets the underlying inflammation in PSC, not just its symptoms. The study also collects patient-reported outcomes and liver health markers, providing early data on whether the drug might slow liver damage. Because PSC is rare and poorly served by current medicine, even a modestly effective therapy would change clinical practice.

View original technical description
Hypothesis Brodalumab is a safe and feasible treatment to administer to patients with PSC and concomitant IBD. Objectives IL-17 inhibition has shown promise in several other autoimmune diseases. However,its potential benefit in PSC patients requires clarification. This pilot study will assess if Brodalumab (an IL-17 inhibitor) could represent a new,safe,tolerable,practicable and acceptable treatment in PSC. Trial Design This study is a multi-centre,single arm,open label pilot study to determine the feasibility of using brodalumab as a treatment for PSC in a future phase II RCT. Exploratory data will be analysed for the potential efficacy of brodalumab in PSC using biochemical and surrogate markers of liver fibro-inflammation,in addition to disease specific patient reported outcome measures. 20 patients will be enrolled,across 4 sites: Norfolk and Norwich University hospital,John Radcliffe Hospital-Oxford,Cambridge University Hospital,and Queen Elizabeth Hospital-Birmingham,which will utilise interdisciplinary collaborative expertise in the PSC field.

Researchers

Guruprasad P Aithal (Principal Investigator)

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Original classification

Gastrointestinal and Liver Disorders

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