Recruiting
Phase 3

Elafibranor

Sponsor:

Ipsen

Code:

NCT06016842

Conditions

Primary Biliary Cholangitis (PBC)

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Elafibranor

Matched 80 mg placebo

Study Details

Brief summary:

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) and cirrhosis (scarring of the liver).

PBC is a slowly progressive disease, characterised by damage to the bile ducts in the liver, leading to a build-up of bile acids which causes further damage.

The liver damage in PBC may lead to cirrhosis. PBC may also be associated with multiple symptoms. Many patients with PBC may require liver transplant or may die if the disease progresses and a liver transplant is not done.

This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment) and will last up to 3.5 years for each participant.

The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).

This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

Conditions

Primary Biliary Cholangitis (PBC)

Study ID

NCT06016842

Start date

Aug 31, 2023

Status verified date

Aug, 2026

Completion date

May 31, 2029

Anticipated

Primary completion date

May 31, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Inclusion Criteria :

  • 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)
  • Participants with cirrhosis at SV1. • Participants must be Child Pugh A or Child Pugh B.
  • Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
  • Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Exclusion Criteria :

  • 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.
  • History or presence of clinically significant hepatic decompensation, including:

  • i) History of liver transplantation, current placement on a liver transplant list, current model for end-stage liver disease including (MELD) 3.0 score >12 due to hepatic impairment.
  • ii) Evidence of complications of cirrhosis, including hepatic decompensation or evidence of significant portal hypertension complications including presence of uncontrolled ascites; history of variceal bleeding or related interventions (e.g. variceal banding, or transjugular intrahepatic portosystemic shunt placement); presence of hepatic encephalopathy Grade 2 or higher per West-Haven criteria; history or presence of spontaneous bacterial peritonitis. Note: participants with low-risk varices (Grade I) without history of bleeding or other treatment may be eligible to enrol.
  • iii) Hepatorenal syndrome (HRS) (type I or II ). • vi) Hospitalisation for liver-related complication within 12 weeks prior to SV1.
  • Known history of human immunodeficiency virus (HIV) infection or having a positive confirmatory test for HIV type 1 or 2.
  • Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).
  • Evidence of any other unstable or untreated clinically significant immunological, endocrine, hematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant conditions that are not well controlled.
  • Non-hepatic medical conditions that may diminish life expectancy to <2 years, including known cancers.
  • History of hepatocellular carcinoma.
  • Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of hepatocellular carcinoma.
  • Known malignancy or history of malignancy within the last 5 years. Participants with non-melanoma skin cancer, or carcinoma in situ (e.g. breast carcinoma, cervical cancer in situ) that have undergone potentially curative therapy are not excluded.
  • Administration of the following medications is prohibited during the study, and prior to the study as per the timelines specified below: • i) 3 months prior to baseline: fibrates, seladelpar, glitazones, obeticholic acid, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid or nitrofurantoin).
  • Participants who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or 5 half-lives, whichever is longer, prior to the screening period.

i) If the previous study was for an experimental therapy being studied for potential benefit in PBC, and the potential therapeutic agent was proven to have no beneficial effect in PBC and there are no safety concerns, the participant may enrol after 30 days or 5 half-lives from the last dose of the therapeutic agent, whichever is longer.ii) For therapeutic agents being studied for potential benefit in PBC for which it is still unclear if there may be a potential benefit, participants may enrol after 6 months from the last dose of the therapeutic agent.
  • Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) >450 msec in males or QTcF >470 msec in females for participants without bundle branch block. For participants with bundle branch block or other intraventricular conduction delay, a longer QTcF >480 msec would be exclusionary.
  • Total bilirubin (TB) >5x ULN
  • Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >5x ULN at SV1
  • Creatinine phosphokinase (CPK) >2x ULN.
  • Platelet count <50,000/μL
  • International normalised ratio (INR) >1.8 in the absence of anticoagulant therapy.
  • Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m2 per the Modification of Diet in Renal Disease (MDRD)-6 Study formula at SV1.
  • Significant renal disease, including nephritic syndrome, chronic kidney disease (CKD) (defined as participants with evidence of significantly impaired kidney function or underlying kidney injury).
  • For female participants: known current pregnancy, or has a positive serum pregnancy test, or is breastfeeding.
  • Participants unwilling or unable to be abstinent from alcohol during the study.
  • History of alcohol abuse, or other substance abuse within 1 year prior to SV1.
  • Known hypersensitivity to elafibranor or to any of the excipients of the investigational product(s).
  • Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
  • Any other condition that, in the opinion of the investigator, would interfere with study participation or completion, or would put the participant at risk, including a potential participant assessed as being at high risk of noncompliance with the study.
  • Alkaline phosphatase (ALP) ≥10x ULN.
  • Albumin <2.8 g/dL due to impaired hepatic function.

Study Design

Enrollment

276 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Elafibranor 80 mg

Participants will take 1 tablet of elafibranor 80 mg per day orally with a glass of water at approximately the same time each morning, with or without food.

placebo comparator: Placebo

Participants will take 1 placebo tablet per day orally (matching the 80 mg elafibranor sized tablet) with a glass of water at approximately the same time each morning, with or without food.

Interventions

Elafibranor

Duration: up to an estimated 42-month (3.5-year) double-blind treatment period during which elafibranor 80 mg tablet will be administered once daily

Matched 80 mg placebo

Duration: up to an estimated 42-month (3.5-year) double-blind treatment period during which matching placebo tablet will be administered once daily

Primary outcome measure

  • Event-free survival [ Time Frame: From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years) ]

Central Contacts and Locations

Central contacts

Ipsen Clinical Study Enquiries

See e mailclinical.trials@ipsen.com

Locations

Arizona Liver Health

Recruiting

Tucson, Arizona, United States, 85641

Southern California Research Center

Recruiting

Coronado, California, United States, 92118

University of California Davis Medical Center

Recruiting

Sacramento, California, United States, 95817

University of Colorado

Recruiting

Aurora, Colorado, United States, 80045

Peak Gastroenterology Associates

Recruiting

Colorado Springs, Colorado, United States, 80829

South Denver Gastroenterology, P.C.

Recruiting

Englewood, Colorado, United States, 80113

Rocky Mountain Gastroenterology

Recruiting

Littleton, Colorado, United States, 80120

University Of Miami School Of Medicine, Center For Liver Diseases

Recruiting

Miami, Florida, United States, 33136

Bolanos Clinical Research

Recruiting

Pembroke Pines, Florida, United States, 12105

International Center for Research

Recruiting

Tampa, Florida, United States, 33614

Louisiana Research Center, LLC

Recruiting

Shreveport, Louisiana, United States, 71103

University of Michigan Health System

Recruiting

Ann Arbor, Michigan, United States, 48109

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

NYU Langone Gastroenterology and Hepatology Associates

Recruiting

New York, New York, United States, 10016

University of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

Medical University of South Carolina

Recruiting

Charleston, South Carolina, United States, 29425

Gastroenterology Center of the Midsouth

Recruiting

Cordova, Tennessee, United States, 38138

Texas Clinical Research Institute

Recruiting

Arlington, Texas, United States, 22201

American Research Corporation

Recruiting

Austin, Texas, United States, 78757

Methodist Transplant Physicians

Recruiting

Dallas, Texas, United States, 75203

University of Texas Southwestern Medical Center at Dallas

Recruiting

Dallas, Texas, United States, 75390

Liver Associates of Texas

Recruiting

Houston, Texas, United States, 77030

American Research Corporation at The Texas Liver Institute

Recruiting

San Antonio, Texas, United States, 78015

University of Virginia Medical Center

Recruiting

Charlottesville, Virginia, United States, 22903

Bon Secours St. Mary's Hospital of Richmond, Inc

Recruiting

Richmond, Virginia, United States, 23226

Velocity Clinical Research at Liver Institute Northwest

Recruiting

Seattle, Washington, United States, 98105

University Physicians and Surgeons Inc, dba Marshall Health

Recruiting

Huntington, West Virginia, United States, 25701

University of Alberta - Faculty of Medicine & Dentistry - The Centre of Excellence for Gastrointestinal Inflammation and Immunity Research (CEGIIR)

Recruiting

Edmonton, Canada

University Health Network (UHN) - Toronto General Hospital (TGH) - Toronto General Research Institute (TGRI)

Recruiting

Toronto, Canada

More Information

Sponsor

Ipsen

Last update posted

Aug 31, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ipsen on 2026-08-31.