A new international clinical trial funded by medical research organisation LifeArc will test whether oral vancomycin, an existing antibiotic, could improve outcomes for people living with a rare liver condition called primary sclerosing cholangitis (PSC), which develops in people with a specific type of inflammatory bowel disease. 

Primary sclerosing cholangitis is a rare, chronic disease affecting approximately 3,500 people in the UK. It causes inflammation and scarring of the bile ducts, eventually leading to liver damage. There are currently no treatments that can stop the disease progressing, and liver transplantation remains the only life-extending treatment. 

Sam Barrell, Chief Executive Officer at LifeArc, said: “For too many people living with rare diseases, getting a diagnosis can take years and effective treatments simply don’t exist.

“Clinical trials are one of the ways we can change that. They generate the evidence needed to support the approval and adoption of new diagnostics and treatments into routine care, but for many rare diseases those studies are difficult to deliver because patient populations are small and funding is limited. 

“Through our Rare Disease Clinical Trials Programme, we’re supporting ambitious and innovative studies that have the potential to improve diagnosis, create and expand treatment options and deliver meaningful benefits for patients within the next five years. This project shows exactly the kind of research we want to help accelerate, and we want to hear from researchers, clinicians and organisations with promising clinical-stage interventions that could make a real difference for people living with rare diseases.”  

Most people with PSC also have an associated type of inflammatory bowel disease (IBD). This can be more difficult to treat than other forms of IBD not associated with this rare condition. As a result, people who are affected are more likely to require bowel surgery and have a higher lifetime risk of bowel cancer. 

Previous research suggests changes in the gut microbiome may play an important role in PSC-IBD. A pilot study led by Professor Trivedi found that oral vancomycin was associated with remission of bowel inflammation and improvements in liver blood tests. 

The trial, titled VITALITY, is one of the first to be funded through LifeArc’s Rare Disease Clinical Trials Programme. It will be led by Professor Palak Trivedi at the University of Birmingham and delivered through the National Institute for Health and Care Research (NIHR) Biomedical Research Centre in Birmingham.  

It will compare two doses of oral vancomycin against a placebo. If successful, it could provide the robust clinical evidence needed for the approval of a new treatment option for people living with PSC-IBD. The trial is also receiving contributions from PSC Support, Children’s PSC Foundation and UHB Charities.

Professor Palak Trivedi, who leads the trial, said: “People living with PSC-IBD face a particularly challenging disease. At present, there are no medicines that have been proven to slow down or stop the disease. As a result, people with PSC can develop serious complications affecting both the liver and the bowel. Previous research has suggested that the gut microbiome may play a key role in driving disease development and its progression, and early findings show that an oral antibiotic called vancomycin could have meaningful benefits for some patients. 

“The VITALITY trial is an important step towards determining whether these initial results can be confirmed in a rigorous international trial. We hope this research will help us better understand the disease and, ultimately, bring us closer to an effective treatment for people living with PSC-IBD.”