What side effects are associated with this type of intervention?

Common treatments for Primary Biliary Cirrhosis (PBC) include ursodeoxycholic acid (UDCA), obeticholic acid, and fibrates, which are similar to Elafibranor (a PPAR alpha/delta agonist). UDCA is generally well-tolerated but can cause weight gain and diarrhea. Obeticholic acid may lead to pruritus (itching), fatigue, and abdominal pain. Fibrates, such as bezafibrate and fenofibrate, can cause gastrointestinal disturbances, increased liver enzymes, and muscle pain. Elafibranor, being studied for PBC, may have similar side effects, including gastrointestinal issues and potential liver enzyme elevations.

What prior FDA approvals exist?

The FDA has approved several treatments for Primary Biliary Cirrhosis (PBC). Ursodeoxycholic acid (UDCA) was the first drug approved and remains a standard treatment. Obeticholic acid, a farnesoid X receptor agonist, is another FDA-approved drug for PBC, particularly for patients who have an inadequate response to UDCA. While Elafibranor, a PPAR alpha/delta agonist, is currently being studied, fibrates like bezafibrate and fenofibrate, which also target PPAR pathways, have shown promise in managing PBC symptoms and biochemical markers, although they are not yet FDA-approved specifically for PBC.

What other types of research exist?

Promising alternatives to Elafibranor for PBC patients include Bezafibrate and Fenofibrate. Bezafibrate has shown improvement in GLOBE and UK-PBC scores and long-term outcomes in patients with PBC who are refractory to ursodeoxycholic acid (UDCA). Fenofibrate has demonstrated significant reductions in liver biochemistries such as alkaline phosphatase (ALP) and alanine aminotransferase (ALT) levels, which could translate into better disease prognosis. Both fibrates are supported by observational evidence and clinical trials indicating their efficacy and safety in PBC treatment.

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Elafibranor for Primary Biliary Cholangitis (ELFIDENCE Trial)

Phase 3

Recruiting

Research Sponsored by Ipsen

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female participants must be ≥18 years of age at the time of signing the informed consent.

Participants with a definite or probable diagnosis of primary biliary cholangitis (PBC).

Must not have

History or presence of other concomitant liver disease including but not limited to: i) Primary sclerosing cholangitis (PSC). ii) Autoimmune hepatitis (AIH) by simplified Diagnostic Criteria of the International Autoimmune Hepatitis Group (IAIHG) ≥6, or if treated for an overlap of PBC with AIH, or if there is clinical suspicion and evidence of overlap AIH features, that cannot be explained alone by insufficient response to UDCA. iii) Positive hepatitis B surface antigen (HBsAg). Participants with negative HBsAg and positive hepatitis B core antibody (HBcAb) may be eligible if hepatitis B virus deoxyribonucleic acid (HBV DNA) is negative. iv) Hepatitis C virus (HCV) infection defined by positive anti-HCV antibody and positive HCV ribonucleic acid (RNA) (Note: Participants with positive anti-HCV antibody due to previously treated HCV infection, may be enrolled if a confirmatory HCV RNA is undetectable and sustained viral response has been documented). v) Alcohol-associated liver disease (ALD). vi) Nonalcoholic steatohepatitis (NASH). vii) Other chronic liver diseases, such as alpha-1 antitrypsin deficiency.

Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of hepatocellular carcinoma.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)

Awards & highlights

Pivotal Trial

Summary

This trial is testing elafibranor, a medication for people with Primary Biliary Cholangitis (PBC) who do not respond to or cannot take the usual treatment. PBC is a liver disease that can lead to severe damage if untreated. Elafibranor aims to reduce harmful substances in the liver to prevent further damage. The study will also check if the medication is safe and helps with symptoms like itching and tiredness. Elafibranor is being tested as a new treatment option for PBC patients who do not respond to or cannot tolerate the standard treatments.

Who is the study for?

Adults over 18 with Primary Biliary Cholangitis (PBC) who haven't responded well to or can't tolerate the standard treatment, UDCA. They should be stable on any itch-management meds for at least 3 months and not have other significant health issues that could affect their participation. People with a history of certain liver diseases, substance abuse, cancer within the last five years, or specific medical conditions are excluded.

What is being tested?

The trial is testing Elafibranor against a placebo in patients with PBC who don’t respond to UDCA. It aims to see if Elafibranor can prevent disease worsening better than a dummy pill over up to seven years while also monitoring long-term safety and effects on symptoms like itching and fatigue.

What are the potential side effects?

While specific side effects for Elafibranor aren't listed here, common ones in trials may include digestive discomfort, potential liver enzyme changes, fatigue, headache, skin reactions or itching. The study will closely monitor participants for any adverse reactions throughout its duration.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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I have been diagnosed with primary biliary cholangitis.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have other liver diseases like hepatitis or fatty liver disease.

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My AFP levels are above 20 ng/mL and imaging shows signs of liver cancer.

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I have previously taken elafibranor.

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My liver enzymes are significantly higher than normal.

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I am not pregnant, do not have a positive pregnancy test, and am not breastfeeding.

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I have a serious kidney condition.

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I haven't taken medications that could harm my liver or certain other drugs in the last 3 months.

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I am mentally capable of understanding and following the study's requirements.

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I do not have any health conditions, including other cancers, that could shorten my life to less than 2 years.

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I have had liver cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Event-free survival

Secondary study objectives

Change from baseline in 5D-Itch scale

Change from baseline in Child Pugh grade

Change from baseline in EuroQol 5-dimensional 5-level questionnaire (EQ-5D-5L)

+24 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Elafibranor 80 mgExperimental Treatment1 Intervention

Participants will take 1 tablet of elafibranor 80 mg per day orally before breakfast with a glass of water at approximately the same time each morning.

Group II: PlaceboPlacebo Group1 Intervention

Participants will take 1 placebo tablet per day orally (matching the 80 mg elafibranor sized tablet) before breakfast with a glass of water at approximately the same time each morning.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Elafibranor

2022

Completed Phase 1

~110

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Primary Biliary Cirrhosis (PBC) include ursodeoxycholic acid (UDCA) and fibrates. UDCA reduces bile acid toxicity, improves bile flow, and protects liver cells. Fibrates, like bezafibrate, activate peroxisome proliferator-activated receptors (PPARs) to reduce bile acid synthesis and inflammation. Elafibranor, a PPAR alpha/delta agonist, is being studied for its potential to further reduce bile acid levels and inflammation, slowing disease progression. These mechanisms are crucial for managing symptoms, preventing liver damage, and improving quality of life for PBC patients.

Fibrate treatment for primary biliary cirrhosis.