SABR-PSC Pilot Study
Research type
Research Study
Full title
A Single Arm pilot study of BRodalumab in the treatment of Primary Sclerosing Cholangitis
IRAS ID
1006951
Contact name
Amera Elzubeir
Contact email
Sponsor organisation
Norfolk and Norwich University Hospital Foundation Trust
Research summary
Primary sclerosing cholangitis (PSC) is a rare liver disease where the body attacks itself, causing inflammation and scarring of the bile ducts and liver. This in turn leads to repeated infections, liver failure, and in some cases cancer. There is no recognised PSC medication that improves survival. For some, liver transplantation is the only lifesaving option. Recent research shows that the immune system (T cells & B cells) is responsible for bile duct damage in PSC. Research has shown us that in people with PSC, these T cells release a chemical called Interleukin 17 (IL-17). Importantly, powerful drugs called biologics that target the specific parts of the immune system have already been developed to treat these diseases. One such biologic is Brodalumab. It is used for psoriasis (a skin condition) and reduces the effects of IL-17. Therefore, given the potential importance of IL-17 in PSC, we will study the effect of brodalumab in people with PSC. Before brodalumab can be recommended as a treatment for PSC, larger studies (randomised control trial) are required to prove its effect. Before a clinical trial we need to understand how to design a larger study; recruitment and retention rates; whether participants will be willing to take the medication; side-effects experienced; quality of life & if brodalumab reduces or slows the damage caused by PSC by measuring known markers of liver damage with blood tests and scans. 20 volunteers with PSC from four large PSC centres (Norwich, Oxford, Cambridge and Birmingham) will take part. They will all be given brodalumab for 12 weeks (once a week for three weeks, then every other week). Brodalumab will be given using an injection pen and participants will be able to do this themselves at home after training. We will monitor participants with blood tests, liver scans, a colonoscopy and questionnaires. A group of PSC patients from across the UK helped us improve the design of our study and highlight patients’ needs and concerns.
REC name
London - London Bridge Research Ethics Committee
REC reference
23/LO/0718
Date of REC Opinion
25 Oct 2023
REC opinion
Further Information Favourable Opinion