Not recruitingRare disease

Study of Elafibranor in Adults with Primary Biliary Cholangitis Who Don't Respond Well to or Cannot Tolerate Ursodeoxycholic Acid Treatment

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What is this trial about?

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

This study focuses on Primary Biliary Cholangitis (PBC), a long-term liver disease that causes the bile ducts in the liver to become damaged. The research evaluates a medication called elafibranor in patients who have not responded well to or cannot tolerate standard treatment with ursodeoxycholic acid. The purpose is to determine if elafibranor can help normalize liver enzyme levels in adults with this condition.

The study will test film-coated tablets containing 80 mg of elafibranor compared to placebo in adult patients. The treatment will be given as a daily oral dose for 52 weeks. During this time, researchers will monitor changes in liver function, particularly focusing on levels of alkaline phosphatase, an enzyme that indicates liver health.

Throughout the study, participants will have their symptoms assessed, including itching severity and quality of life. The research will also track any changes in liver function tests and monitor the overall safety of the treatment. The study uses various questionnaires to measure how participants feel and function during their daily activities while receiving the treatment.

The research process

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

Step 1
Initial medication assignment
You will be randomly assigned to receive either elafibranor (80 mg film-coated tablet) or a matching placebo tablet
The medication needs to be taken orally once daily
The study will last for 52 weeks (1 year)
Step 2
Regular check-ups - Week 4
Your first follow-up visit will occur 4 weeks after starting treatment
Medical staff will measure your alkaline phosphatase (ALP) levels
You will complete questionnaires about your symptoms, including any itching you may experience
Step 3
Follow-up visits - Weeks 12 and 24
Additional check-ups will take place at weeks 12 and 24
Your blood will be tested to monitor liver function
Your overall health status will be evaluated
At week 24, you will complete quality of life assessments
Step 4
Continued monitoring - Weeks 36 and 52
Further evaluations will occur at weeks 36 and 52
Final blood tests will be performed to measure ALP and bilirubin levels
The study will conclude at week 52 with final health assessments
Step 5
Safety monitoring
Throughout the study, you will be monitored for any side effects
Regular physical examinations will be conducted
Your vital signs and heart activity (ECG) will be checked
Laboratory tests will continue to monitor your health status

Who can join the trial?

13 criteria

Must be at least 18 years old
Must have a confirmed diagnosis of Primary Biliary Cholangitis (PBC) with at least 2 of these conditions:
- History of elevated alkaline phosphatase (ALP) levels for 6 months or more
- Positive antimitochondrial antibody test
- Liver biopsy showing PBC
- Must have ALP levels above normal but not more than 1.67 times the upper normal limit
- If taking UDCA (a medication for PBC):
  - Must have been taking it for at least 12 months
  - Must be on a stable dose for at least 6 months
  - If unable to take UDCA, must have stopped it at least 3 months ago
  - If taking medications for itching, must be on a stable dose for at least 3 months
  - Must follow specific contraception requirements:
    
    Men must agree to use condoms and not donate sperm during the study and for 30 days after
    Women who can become pregnant must use effective contraception methods and have a negative pregnancy test
    Women must not be pregnant or breastfeeding
    Must be able to understand and sign the informed consent form

Who cannot join the trial?

15 criteria

Age below 18 years old
Pregnant or breastfeeding women
History of liver transplantation
Current diagnosis of cirrhosis (severe scarring of the liver) with complications
Presence of other liver diseases besides Primary Biliary Cholangitis (PBC)
Current or recent use of medications that could interact with the study drug
Severe kidney problems (as measured by blood tests)
Uncontrolled high blood pressure
History of heart problems in the past 6 months
Active cancer or cancer treatment within the past 5 years
History of alcohol or drug abuse within the past 2 years
Participation in another clinical trial within the past 30 days
Known allergies to the study medication or its components
Abnormal blood test results that could indicate other health problems
Mental health conditions that could interfere with study participation

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Verified sites

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Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain
Futuremeds Sp. z o.o.
Responsive
Wroclaw, Poland
Krakowskie Centrum Medyczne Sp. z o.o.
Responsive
Cracow, Poland

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Investigated drugs

Elafibranor
is a medication being studied for treating Primary Biliary Cholangitis (PBC), a liver condition. It works by targeting certain receptors in the liver that help reduce inflammation and improve liver function. This medication is taken orally and is being tested in patients who haven't responded well to standard treatments or couldn't tolerate them.
Ursodeoxycholic Acid
is a standard medication used to treat Primary Biliary Cholangitis. It's a naturally occurring bile acid that helps improve liver function by reducing the buildup of harmful bile acids. This medication is mentioned as the previous treatment that patients in the study either didn't respond well to or couldn't tolerate.

Elafibranor

What is already known about the treatment

Elafibranor
An oral medication being investigated in Phase IIIb clinical trials for the treatment of Primary Biliary Cholangitis (PBC), particularly in patients who have not responded adequately to or cannot tolerate Ursodeoxycholic Acid. This drug belongs to the class of dual PPARα/δ (peroxisome proliferator-activated receptor alpha and delta) agonists, which work by regulating genes involved in bile acid metabolism and inflammation in the liver. The medication is administered as an 80 mg oral dose taken daily, and its primary goal is to normalize alkaline phosphatase (ALP) levels in PBC patients, potentially offering a new therapeutic option for this chronic liver disease.
Ursodeoxycholic Acid
A naturally occurring bile acid medication that is the current standard first-line treatment for Primary Biliary Cholangitis (PBC), administered orally in tablet form. It works by improving bile flow from the liver, reducing inflammation, and protecting liver cells from damage caused by toxic bile acids. This medication has been well-established in medical literature and clinical practice as an effective treatment for PBC, though some patients may show inadequate response or intolerance to the therapy.

Investigated diseases

Primary Biliary Cholangitis (PBC) - A chronic liver disease that primarily affects women and slowly damages the bile ducts in the liver. The condition causes inflammation and scarring of the small bile ducts, which leads to bile buildup in the liver. Over time, this progressive disease causes the bile ducts to be destroyed, allowing harmful substances to build up in the liver. The disease typically develops slowly over many years, gradually affecting the liver's ability to function properly. PBC often begins with inflammation that may cause fatigue and itching, while the bile ducts become increasingly damaged.