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Study on Bezafibrate for Patients with Primary Biliary Cholangitis Not Responding Well to Ursodeoxycholic Acid

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effectiveness and safety of the medication bezafibrate in patients with Primary Biliary Cholangitis (PBC), a liver disease. The study is specifically for those who have not had an optimal response to a standard treatment called ursodeoxycholic acid (UDCA). The trial will test two different doses of bezafibrate: 200 mg and 400 mg, and compare them to a placebo. The purpose of the study is to see if bezafibrate can help improve the condition of patients with PBC who are not responding well to UDCA.

The study will last for a total of 24 months. During the first 12 months, participants will receive either bezafibrate or a placebo in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication. After this period, there will be a 12-month extension phase where all participants will receive bezafibrate, but without the use of a placebo. This approach helps to ensure that the results are reliable and that any improvements can be attributed to bezafibrate.

Throughout the study, participants will be monitored for changes in their liver function and any side effects they may experience. The main goal is to determine if bezafibrate can lead to a complete biochemical response, which means normalizing certain liver enzyme levels. Additionally, the study will look at other factors such as the intensity of itching, which is a common symptom of PBC, and any serious adverse events that may occur. This research aims to provide more information on whether bezafibrate can be a beneficial addition to the treatment of PBC.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, eligibility is confirmed based on specific criteria, including age and medical history related to primary biliary cholangitis (PBC).

    Participants must have been on a stable dose of ursodeoxycholic acid (UDCA) therapy for at least 12 months prior to joining.

  2. Step 2

    Randomization and initial treatment

    Participants are randomly assigned to receive either bezafibrate 200 mg, bezafibrate 400 mg, or a placebo. This is a double-blind process, meaning neither the participants nor the researchers know who receives which treatment.

    The medication is taken orally in the form of tablets. The 200 mg dose is a film-coated tablet, while the 400 mg dose is a prolonged-release tablet.

  3. Step 3

    Treatment phase

    The treatment phase lasts for 12 months. During this time, participants continue taking their assigned medication daily.

    Regular monitoring occurs to assess the biochemical response, focusing on liver function tests such as alkaline phosphatase (ALP) and other enzymes.

  4. Step 4

    Assessment of primary outcomes

    At the end of 48 weeks, the primary outcome is evaluated. This involves checking if participants have achieved normal levels of specific liver enzymes, indicating a complete biochemical response.

    Secondary outcomes include monitoring for any adverse events and changes in symptoms such as itch intensity.

  5. Step 5

    Extension phase

    After the initial 12-month period, participants enter a 12-month extension phase where the placebo is no longer used.

    This phase continues to monitor the safety and efficacy of bezafibrate as an adjunctive treatment.

Who can join the trial?

9 criteria

  • Must be between 18 and 79 years old.
  • Must have a diagnosis of **PBC** (Primary Biliary Cholangitis) based on at least two of the following:
    • High **ALP** (Alkaline Phosphatase) level, which is a type of enzyme in the blood.
    • Presence of **antimitochondrial antibodies**, which are proteins made by the immune system that mistakenly target the body's own cells.
    • Specific **antinuclear antibodies** or positive tests for **anti-gp210** or **anti-Sp100 antibodies**, which are other types of proteins made by the immune system.
    • Medical records showing liver tissue features that suggest or match PBC.
    • Must have been on **UDCA** (Ursodeoxycholic Acid) treatment for the past 12 months, with a stable dose of at least 12 mg per kg per day for at least 3 months before joining the study.
    • Women who can have children must use at least one barrier method of birth control during the study and for at least 90 days after the last dose.
    • Must be part of a social security system, except for AME (State Medical Aid).
    • Must have signed an informed consent form, which means agreeing to participate in the study after understanding all the details.

Who cannot join the trial?

3 criteria

  • Patients who do not have a condition called PBC (Primary Biliary Cholangitis) with a non-optimal response to UDCA (Ursodeoxycholic Acid) cannot participate.
  • Patients who are not within the specified age range for the study cannot participate.
  • Patients who are part of a vulnerable population, which means they might need special protection or care, cannot participate.
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Investigated drugs

Bezafibrate is being studied as an additional treatment for patients with primary biliary cholangitis (PBC) who do not respond well to the standard therapy. It is used to help improve liver function in these patients. The trial is testing its effectiveness and safety over a period of 12 months.

What is already known about the treatment

Bezafibrate – Bezafibrate is administered orally in tablet form and is currently being studied in clinical trials for its effectiveness as an additional treatment for patients with primary biliary cholangitis (PBC) who do not respond optimally to ursodeoxycholic acid (UDCA) therapy. It is classified as a fibrate, which is a type of medication primarily used to lower lipid levels in the blood. The main therapeutic indication for bezafibrate in this context is to improve liver function in PBC patients. At the molecular level, bezafibrate works by activating peroxisome proliferator-activated receptors (PPARs), which play a role in regulating lipid metabolism and inflammation.

Investigated diseases

Primary Biliary Cholangitis (PBC) – This is a chronic liver disease where the bile ducts in the liver become damaged, leading to a buildup of bile and potential liver damage. Over time, this can cause scarring of the liver tissue, known as fibrosis, and eventually lead to cirrhosis. The disease often progresses slowly, and symptoms may not appear until significant liver damage has occurred. Common symptoms include fatigue, itchy skin, and dry eyes and mouth. The exact cause of PBC is not well understood, but it is believed to involve an autoimmune response. It primarily affects middle-aged women and can be associated with other autoimmune conditions.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-504086-23-00Protocol codeAPHP220822Estimated enrolment130 patientsSponsorAssistance Publique Hopitaux De Paris

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