Active not recruitingPHASE3INTERVENTIONAL

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)

This study is investigating a new medication called elafibranor for individuals diagnosed with Primary Biliary Cholangitis (PBC) who haven't responded well to usual treatments. PBC is a condition where the small bile ducts in the liver are damaged, which can lead to liver scarring and other health problems. The study aims to find out if elafibranor is better than a dummy treatment (placebo) at lowering a specific liver enzyme level in the blood. Researchers will also look at how safe the drug is over time and if it helps reduce common symptoms of PBC, such as itchy skin and fatigue. Participants will receive either elafibranor or a placebo for up to two years, followed by up to five years where everyone receives elafibranor.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Ipsen

Enrolment target

161

Start

24 Sep 2020

Estimated completion

01 Dec 2028

Primary Biliary Cirrhosis

Results

Results from this study

Posted September 2024

Results have been published for this study.

Primary outcome

Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52

Cholestasis response was defined as alkaline phosphatase (ALP) \< 1.67 x upper limit of normal (ULN) and total bilirubin (TB) \<= ULN and ALP decrease from baseline \>= 15% at Week 52 and based on the composite strategy imputing non-response for participants who experienced intercurrent events (ICEs) (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.

Full results on the registry

What is this study about?

This research study is looking into a new medication called elafibranor for people who have a liver condition called Primary Biliary Cholangitis (PBC). PBC is a long-term illness where tiny tubes in your liver, called bile ducts, get damaged. These ducts usually carry a fluid called bile that helps digest fats. When they're damaged, bile can build up in the liver, leading to further harm. Over time, this damage can cause scarring, known as cirrhosis, and may even require a liver transplant.

Many people with PBC also experience bothersome symptoms like very itchy skin and extreme tiredness. Current treatments, like a medication called ursodeoxycholic acid (UDCA), help some people, but not everyone responds well or can tolerate it. This study wants to see if elafibranor could be a better option for those individuals. The main goal is to find out if taking elafibranor can reduce the levels of a particular blood marker, called alkaline phosphatase, which doctors use to monitor PBC. Lower levels often suggest that the liver is doing better.

Beyond just the blood tests, the research team will also carefully check for any side effects of elafibranor to understand its safety. Importantly, they will also try to understand if the drug helps to make those uncomfortable symptoms, like itching and tiredness, any better. The study is divided into two parts: initially, some participants will receive the active drug and others a dummy drug (placebo) for one to two years. Then, for the second part, everyone in the study will have the opportunity to take elafibranor for several more years.

Key takeaways

The study tests a new drug, elafibranor, for Primary Biliary Cholangitis (PBC).
It's for people with PBC who haven't responded well to standard treatment (UDCA).
The study aims to improve liver health and reduce symptoms like itching and tiredness.
Participants will take either elafibranor or a dummy pill (placebo) initially.
The study involves regular visits and blood tests over several years.
Your safety and well-being are a priority, and you can withdraw at any time.

Who may be eligible?

To join this study, you generally need to be an adult between 18 and 75 years old and have a confirmed diagnosis of Primary Biliary Cholangitis (PBC). It's important that your PBC hasn't responded well to standard treatment with ursodeoxycholic acid (UDCA) or that you're unable to take UDCA. Your liver test results, specifically certain levels of alkaline phosphatase and bilirubin, also need to fall within specific ranges.

However, some conditions would mean you couldn't take part. This includes having other severe liver diseases or having serious complications from your PBC, such as advanced cirrhosis. If you have any other significant medical conditions that could shorten your life expectancy, or if you've been diagnosed with certain serious infections like HIV, you wouldn't be able to join. Additionally, pregnant or breastfeeding women cannot participate, and women who could become pregnant must use effective birth control throughout the study.

There are also restrictions on certain medications. If you are currently taking or have recently taken specific drugs for liver conditions or other illnesses, you might not be eligible. The study team will review all your medical history and current medications to ensure it's safe for you to participate.

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.

Are you between 18 and 75 years old?
Do you have a firm diagnosis of Primary Biliary Cholangitis (PBC)?
Has your PBC not responded well to usual UDCA treatment, or can you not take UDCA?
Do you *not* have other severe liver conditions?
Are you *not* pregnant or breastfeeding, and if you could get pregnant, are you willing to use effective birth control?
Are you *not* taking certain other specific medications that might interfere with the study drug?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, it will involve two main stages. In the first stage, which could last between one and two years, you would be randomly assigned to receive either the study drug, elafibranor, or a dummy treatment (placebo) every day. Neither you nor your doctor would know which one you are taking. You will need to attend regular appointments at the clinic for check-ups, blood tests, and to discuss any symptoms or side effects you might be experiencing. These visits help the researchers monitor your health and how the treatment is working.

After the first stage is complete, all participants will then move into the second stage. During this stage, everyone will receive elafibranor every day. This part of the study could last for up to five years, or until your total time in the study (including the first stage) reaches a maximum of six years, whichever comes first. Throughout both stages, you will continue to have regular check-ups and assessments to monitor your liver health, symptoms like itching and tiredness, and to ensure your safety. You will be provided with the study medication and instructions on how to take it.

Potential risks and benefits

Participating in this study might offer potential benefits, such as receiving a new treatment that could improve your liver condition or reduce your symptoms if elafibranor proves to be effective. However, it's also possible that you could receive the placebo (dummy treatment) during the first part of the study, meaning you might not experience any direct benefit from the study drug itself during that time. As with any medication, there are potential risks and side effects associated with taking elafibranor, which the study team will monitor closely. You will be fully informed about all known potential risks before you consent to participate. It's very important to remember that joining a clinical trial is always voluntary, and you have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (115)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

The Institute for Liver Health
Verified postcode
Chandler, United States
Keck Medical Center of USC
Verified postcode
Los Angeles, United States
Ruane Clinical Research Group Inc.
Verified postcode
Los Angeles, United States
Cedars-Sinai Medical Center
Verified postcode
Los Angeles, United States
University of California, Davis Medical Center
Verified postcode
Sacramento, United States
California Pacific Medical Center - Sutter Pacific Medical Foundation
Verified postcode
San Francisco, United States
University of Colorado Denver and Hospital
Verified postcode
Aurora, United States
South Denver Gastroenterology, P.C.
Verified postcode
Englewood, United States
Yale School of Medicine, Digestive Diseases
Verified postcode
New Haven, United States
Encore Borland-Groover Clinical Research
Verified postcode
Jacksonville, United States
Schiff Center for Liver Diseases/University of Miami
Verified postcode
Miami, United States
Digestive Healthcare of Georgia
Verified postcode
Atlanta, United States

Common questions

What is Primary Biliary Cholangitis (PBC)?

PBC is a long-term liver disease where the small tubes in your liver (bile ducts) become damaged, causing bile to build up and potentially leading to liver scarring.

What is elafibranor?

Elafibranor is a new medication being tested in this study to see if it can help people with PBC by improving liver health and symptoms.

What is a 'placebo'?

A placebo is a dummy treatment, often a pill that looks like the study drug but contains no active medicine. It helps researchers compare the real drug's effects.

How long will the study last?

The study has two parts; the first part is 1-2 years, and the second part can last for up to 5 more years, with a total study time of up to 6 years.

Will I know if I'm getting the real drug or the placebo?

During the first part of the study, neither you nor your doctor will know if you are receiving elafibranor or the placebo. In the second part, everyone receives elafibranor.