A Phase IIIb Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Effect of Elafibranor 80 mg on Normalisation of Alkaline Phosphatase in Adult Participants with Primary Biliary Cholangitis (PBC) and Inadequate Response or Intolerance to Ursodeoxycholic Acid
Primary biliary cholangitis (PBC) is a long-term condition where the body's immune system slowly damages the tiny bile ducts inside the liver. The standard treatment is a medicine known as UDCA (ursodeoxycholic acid). While UDCA helps many people, it does not work well enough for everyone, and some people cannot tolerate it. This trial is testing whether a new tablet called elafibranor can help. Researchers want to find out if taking elafibranor for one year can return key liver proteins, especially one called alkaline phosphatase (ALP), to normal ranges. Lowering ALP is an important sign that the liver is healthier. The study will also measure whether the treatment helps ease common, distressing symptoms like persistent skin itching. To ensure fair results, participants will be randomly chosen to receive either elafibranor or a dummy pill (placebo).
At a glance
What is this study about?
Primary biliary cholangitis (PBC) is an autoimmune condition affecting the liver. Over time, swelling and inflammation damage the small tubes that carry digestive fluid (bile) out of the liver. When bile builds up, it causes liver irritation and can lead to scarring. A high level of a liver protein called alkaline phosphatase (ALP) in the blood is one of the main signs that the liver is under stress.
For many years, the main treatment has been ursodeoxycholic acid (UDCA). UDCA helps improve bile flow, but a significant number of people find that their liver tests remain too high despite taking it, or they experience side effects that make it difficult to continue. When this happens, doctors need safe, effective second-line medicines to step in and protect the liver from further damage.
This study evaluates elafibranor, a tablet designed to calm inflammation and improve how the liver processes bile acids and fats. By reducing liver stress, doctors hope elafibranor will bring ALP and other liver markers back down to safe levels. Researchers also want to see if the treatment can give people meaningful relief from severe itching (pruritus), which is often one of the most frustrating daily symptoms of PBC.
Key takeaways
- Tests a new daily tablet called elafibranor for primary biliary cholangitis (PBC).
- Aimed at adults whose current PBC treatment is not working well enough or causes side effects.
- Main goal is to return liver blood tests, particularly ALP, back to healthy levels.
- Also evaluates whether the medicine relieves severe skin itching.
- Involves taking a tablet daily and attending regular clinic visits for about one year.
Who may be eligible?
This study is open to men and women aged 18 and older who have been diagnosed with primary biliary cholangitis (PBC).
To be suitable, you must have tried the standard treatment, UDCA, and found that your liver blood tests (such as ALP) did not improve enough, or you were unable to keep taking UDCA because of troublesome side effects.
Your study doctor will also run health checks to ensure your liver and kidneys are functioning safely enough for you to take part. People with certain other liver conditions or severe health problems may not be able to join.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- I am an adult aged 18 or older.
- I have a confirmed diagnosis of primary biliary cholangitis (PBC).
- I have taken ursodeoxycholic acid (UDCA) but my liver numbers are still higher than normal.
- OR I had to stop taking UDCA because it caused side effects.
- I am willing to attend regular hospital appointments and have routine blood tests for a year.
What does participation involve?
Taking part in this study lasts for at least one year (52 weeks). During this time, you will be asked to take a study tablet once every day. You will have regular appointments at a hospital clinic, typically starting every few weeks and then spacing out over the year. At these visits, doctors will monitor your health through routine blood tests, urine tests, and physical examinations. You will also be asked to complete short questionnaires to track how you are feeling, particularly regarding energy levels and any skin itching. Travel expenses for clinic visits are usually reimbursed.
Potential risks and benefits
Locations (7)
- —UnverifiedGermany
- —UnverifiedPoland
- —UnverifiedCzechia
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedRomania
Common questions
What is a placebo and why is it used?
A placebo is a dummy pill that looks and tastes identical to the real medicine but contains no active ingredients. It helps researchers see whether changes in health are truly caused by the study drug or by other factors.
Will I know if I am getting the real medicine or the placebo?
No. Neither you nor your study doctor will know which tablet you are receiving during the trial. This is called a 'double-blind' study and ensures the results are completely unbiased.
Do I have to stop taking my usual PBC medication?
If you are currently taking UDCA and can tolerate it, you will usually continue taking your standard dose alongside the study tablet. If you cannot tolerate UDCA, you may take the study tablet on its own.
What happens if my liver tests worsen during the study?
Your safety is the top priority. Your blood tests will be checked regularly, and if your liver health shows signs of worsening, the study team will step in and discuss the safest treatment options with you.
Do I have to pay to take part?
No. The study medication, clinic visits, and all trial tests are provided free of charge, and reasonable travel costs to and from the hospital are usually covered.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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