Proof of concept - safety and efficacy of baricitinib in PBC
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Proof-of-Concept Study Evaluating the Efficacy and Safety of Baricitinib (LY3009104) in Patients with Primary Biliary Cholangitis Who Have an Inadequate Response or are Intolerant to UDCA
IRAS ID
256145
Contact name
Steve Ryder
Contact email
Sponsor organisation
Eli Lilly and Company
Eudract number
2018-003365-34
Duration of Study in the UK
0 years, 18 months, 0 days
Research summary
Primary biliary cholangitisis (PBC) is a chronic and progressive liver disease that is caused when the body’s immune systems mistakenly attack the bile ducks. PBC is typically diagnosed during routine blood tests while the patient has no symptoms or shows early signs of the disease. Common symptoms include persistent itching, sore joints, dry eyes/mouth and fatigue. When left untreated PBC can progress resulting in liver transplant or death.
Current standard of care for treatment of PBC is Ursodeoxycholic acid (UDCA). UCDA has been shown to significantly reduce disease progression, liver transplant and death, but anywhere from 40 to 50% of patients will have an inadequate response. In 2016 Obeticholic acid was approved for use in patients with PBC as an add on to UCDA or as a monotherapy when the patient is intolerant to UCDA. But maintaining an effective does is difficult as Obeticholic is associated with increased itching.
The study medication Baricitinib works by inhibiting JAK enzyme activity which in turn reduces inflammation, cellular activity and increases important immune cells. Clinical efficacy and safety have been shown in 4 completed phase 3 studies in patients with rheumatoid arthritis, while a phase 2 study demonstrated efficacy in patients with moderate to severe plaque psoriasis.
This proof of concept Phase II study is sponsored by Lilly and will evaluate the safety and efficacy of Baricitinib in patients with PBC who have an inadequate or are intolerant to UDCA. Approximately 52 participants from the US, UK and Italy will be selected to take part with 12 participants selected from the UK. Patients will be randomized to receive either placebo, 2-mg baricitinib or 4-mg baricitinib. The study will be double-blind and placebo-controlled to minimize bias. Patients will be allowed to take UCDA if able to tolerate.REC name
East Midlands - Nottingham 2 Research Ethics Committee
REC reference
19/EM/0061
Date of REC Opinion
2 May 2019
REC opinion
Further Information Favourable Opinion