New hope for rare liver disease: drug shows promise in reducing damage and symptoms

NCT ID NCT04526665

Disease control Sponsor: Ipsen Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Sep 02, 2026 · Updated 5 times

Summary

This study tests a new drug called elafibranor in people with primary biliary cholangitis (PBC), a rare liver disease that can lead to scarring and liver failure. Participants either did not get enough help from or could not take the standard medicine. The goal is to see if elafibranor can lower a key liver enzyme and improve symptoms like itching and tiredness. The study lasts up to 6 years, with everyone eventually receiving the drug.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

161 people

The number who actually took part.

Started

Sep 2020

Expected to finish

Dec 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria * Males or females age of 18 to 75 years (inclusive) * Definite or probable Primary biliary cholangitis (PBC) diagnosis * Alkaline phosphatase (ALP) ≥ 1.67x upper limit of normal (ULN) * Total bilirubin (TB) ≤ 2x ULN * Ursodeoxycholic acid (UDCA) for at least 12 months (stable dose ≥ 3 months) prior to screening, or unable to tolerate UDCA treatment (no UDCA for ≥ 3 months) prior to screening (per country standard-of-care dosing) * Must have PBC Worst Itch Numeric rating scale (NRS) collected prior to randomization * Females participating in this study must be of non-child bearing potential or must be using highly efficient contraception for the full duration of the study and for 1 month after the last drug intake Exclusion Criteria: * History or presence of other concomitant liver disease * Clinically significant hepatic decompensation, including patients with complications of cirrhosis/portal hypertension * Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease) or which may diminish life expectancy to \< 2 years, including known cancers * Patient has a positive test for HIV Type 1 or 2 at screening, or patient is known to have tested positive for HIV * Evidence of any other unstable or untreated clinically significant disease * History of alcohol abuse * For female patients: known pregnancy or lactating * Use of fibrates and glitazones within 2 months prior to screening * Use of Obeticholic acid (OCA), 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) within 3 months prior to screening * Use of antibodies or immunotherapy directed against interleukins (ILs) or other cytokines or chemokines within 12 months prior to screening * For patients with previous exposure to OCA, OCA should be discontinued 3 months prior to screening * Patients 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 five half-lives, whichever is longer, prior to screening; for patients with previous exposure to seladelpar, seladelpar should be discontinued 3 months prior to screening * Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) values \> 5 x ULN * For patients with AT or TB\>ULN at SV1, variability of AT or TB \> 40% (see section 3.5.1) * Albumin\<3.0 g/dl * Severely advanced patients according to Rotterdam criteria (TB \> ULN and albumin \<LLN) * INR \> 1.3 due to altered hepatic function * CPK \> 2 x ULN * Screening serum creatinine \> 1.5 mg/dl * Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as patients with markers of kidney failure damage or eGFR \< 60 mL/min/1.73 m\^2) calculated by Modification of diet in renal disease (MDRD) * Platelet count \< 150 x 10\^3/μL * AFP \> 20 ng/mL with 4-phase liver CT or MRI imaging suggesting presence of liver cancer * Known hypersensitivity to the investigational product or to any of the formulation excipients of the elafibranor or placebo tablet Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain

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Conditions

The condition(s) this trial relates to.

primary biliary cholangitis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Primary biliary cirrhosis

Contacts and locations

Locations

ASST Monza - Ospedale San Gerardo, Gastroenterologia

Monza, 20900, Italy
American Research Corporation

San Antonio, Texas, 78215, United States
Antwerp University Hospital

Edegem, 2650, Belgium
Azienda Ospedale- Università degli Studi di Padova UOC Gastroenterologia

Padova, 35128, Italy
Azienda Ospedaliera San Paolo, Dipartimento di Scienza della Salute, UO Medicina VI e Pathologia e Gastroenterologia

Milan, 20142, Italy
Azienda Ospedaliera Universitaria Policlinico P. Giaccone, UOC di Gastroenterologia-Dip.Medicina Interna e Specialistica

Palermo, 90127, Italy
Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi

Bologna, 40138, Italy
Belfast Health and Social Care Trust-Royal Victoria Hospital

Belfast, BT12 6BA, United Kingdom
Beth Israel Deaconess Medical Center (BIDMC)

Boston, Massachusetts, 02215, United States
CHU Amiens Picardie

Amiens, 80054, France
CHU Grenoble Alpes - Hôpital Albert Michallon

Grenoble, 38043, France
California Pacific Medical Center - Sutter Pacific Medical Foundation

San Francisco, California, 94109, United States
Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital

Cambridge, CB2 0QQ, United Kingdom
Carolinas Centre for Liver disease/ Atrium Health

Huntersville, North Carolina, 28078, United States
Cedars-Sinai Medical Center

Los Angeles, California, 90048, United States
Center for Gastroenterology and Hepatology (GHZ) Kiel

Kiel, 24146, Germany
Centre Hospitalier Régional d'Orléans

Orléans, 45067, France
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)

Montreal, H2X 0A9, Canada
Centro de Investigacion Clinica Universidad Catolica CICUC

Santiago, Santiago Metropolitan, 8330034, Chile
Centro de Investigaciones Clinicas (CIC)

Viña del Mar, Valparaiso, 2540364, Chile
Charite - Universitätsmedizin Berlin- CVK - Medizinische Klinik

Berlin, 13353, Germany
Clínica Universidad de los Andes

Santiago, 7550000, Chile
Columbia University Medical Center - Center for Liver Disease and Transplantation

New York, New York, 10032, United States
Complexo Hospitalario Universitario de Pontevedra

Pontevedra, 36071, Spain
Consultants for Clinical Research

Cincinnati, Ohio, 45249, United States
Digestive Healthcare of Georgia

Atlanta, Georgia, 30309, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
EUGASTRO GmbH

Leipzig, 04103, Germany
Ege Universitesi Tip Fakultesi Hastanesi

Izmir, Bornova, 35100, Turkey (Türkiye)
Encore Borland-Groover Clinical Research

Jacksonville, Florida, 32256, United States
Fondazione Epatocentro Ticino

Lugano, 6900, Switzerland
Frimley Health NHS Foundation Trust - Frimley Park Hospital

Camberley, GU16 7UJ, United Kingdom
Gastro - Sudien

Berlin, 10825, Germany
Gastro health & Nutrition-Victoria

Victoria, Texas, 77904, United States
Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology

Vancouver, British Columbia, V5Z IM9, Canada
Groote Schuur Hospital, University of Cape Town - Clinical Research Centre

Cape Town, Western Cap, 7925, South Africa
Hacette Universitesi Hastenesi IC, Hastaliklari Anabilim Dali, Gastronenteroloji Bilim Dali, Mithapasa Cad. Hacettepe Mah.

Ankara, 06230, Turkey (Türkiye)
Henry Ford Health System

Novi, Michigan, 48377, United States
Hospital Alemán

Caba, Buenos Aires, C1118AAT, Argentina
Hospital Británico de Buenos Aires

CABA, Buenos Aires, C1280AEB, Argentina
Hospital Clinic de Barcelona

Barcelona, 08036, Spain
Hospital Clínico Universidad de Chile

Santiago, Santiago Metropolitan, 8320000, Chile
Hospital Espanol De Mendoza

Godoy Cruz, Mendoza Province, 5547, Argentina
Hospital General Universitario Gregorio Marañón

Madrid, 28007, Spain
Hospital Italiano de Buenos Aires

CABA, Buenos Aires, 1199, Argentina
Hospital Italiano de La Plata

La Plata, Buenos Aires, 1900, Argentina
Hospital Provincial del Centenario

Rosario, Santa Fe Province, S2002KDS, Argentina
Hospital Universitario Donostia

San Sebastián, 20014, Spain
Hospital Universitario La Paz

Madrid, 28046, Spain
Hospital Universitario Marqués de Valdecilla

Santander, 39010, Spain
Hospital Universitario Puerta de Hierro -Madrid

Madrid, C.P 28222, Spain
Hospital Universitario Vall d'Hebron

Barcelona, 08035, Spain
Hospital Universitario Virgen del Rocio

Seville, 41013, Spain
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo-HCFMUSP

São Paulo, São Paulo, 05403-000, Brazil
Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Hospital de La Serena

La Serena, Coquimbo Region, 1700000, Chile
Hull University Teaching Hospitals NHS Trust , Hull Royal Infirmary

Hull, HU32J, United Kingdom
Hôpital Cochin

Paris, 75014, France
Hôpital Erasme

Brussels, 1070, Belgium
Hôpital Haut Lévêque

Pessac, 33604, France
Hôpital Henri Mondor

Créteil, 94000, France
Hôpital Pitié-Salpétrière

Paris, 75651, France
Hôpital Robert Debré - CHU de Reims

Reims, 51092, France
Hôpital Saint-Antoine

Paris, 75012, France
Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre

Strasbourg, 67200, France
Inselspital, Universitätsspital Bern Universitätsklinik für Viszerale chirurgie und Medizin Hepatologie

Bern, 3010, Switzerland
Intermountain Medical Center - Transplant Services

Murray, Utah, 84107, United States
Investigational Drug Service Pharmacy Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States
Keck Medical Center of USC

Los Angeles, California, 90033, United States
King's College Hospital. King's College NHS Foundation Trust

London, SE5 9RS, United Kingdom
Liver Associates of Texas, P.A.

Houston, Texas, 77030, United States
Liver Center of Texas, PLLC

Dallas, Texas, 75234, United States
Liver Institute Northwest

Seattle, Washington, 98105, United States
Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi, Gastroentoloji Bilim Dali

Istanbul, Pendik, 34890, Turkey (Türkiye)
Maryview Hospital Inc, Bon Secours Liver Institute of Hampton Roads

Newport News, Virginia, 23602, United States
Massachusetts General Hospital

Boston, Massachusetts, 02114, United States
Medical University of South Carolina- College of Medicine

Charleston, South Carolina, 29425, United States
Mediclinic Constantiaberg, North Suites

Cape Town, 7800, South Africa
Medizinische Hochschule Hannover

Hanover, 30625, Germany
NYU Langone Health / NYU Grossman School of Medicine

New York, New York, 10016, United States
Nottingham University Hospitals NHS Trust - Queen's Medical Centre (QMC)

Nottingham, NG72UH, United Kingdom
Ospedale Civile di Baggiovara-Struttura Complessa di Medicina ad indririzzo Metabolico Nutrizionale

Modena, Località Baggiovara, 41126, Italy
Plymouth Hospitals NHS Trust, Derriford Hospital

Plymouth, PL6 8DH, United Kingdom
Richmond Community Hospital LLC, Bon Secours Liver Institute of Richmond

Richmond, Virginia, 23226, United States
Ruane Clinical Research Group Inc.

Los Angeles, California, 90036, United States
Saint Louis University

St Louis, Missouri, 63104, United States
Schiff Center for Liver Diseases/University of Miami

Miami, Florida, 33136, United States
Shared Health Inc.-Operating as Health Sciences Centre-John Buhler Research Centre

Winnipeg, Manitoba, R3E 3P4, Canada
South Denver Gastroenterology, P.C.

Englewood, Colorado, 80113, United States
St. Luke's Health-Baylor St Luke's Medical center - Advanced Liver Therapies Research

Houston, Texas, 77030, United States
Texas Clinical Research Institute, LLC

Arlington, Texas, 76012, United States
Texas Digestive Disease Consultants dba GI Alliance

Fort Worth, Texas, 76104, United States
The Institute for Liver Health

Chandler, Arizona, 85224, United States
The Liver Institute at Methodist Dallas Medical Center

Dallas, Texas, 75203, United States
The New York-Presbyterian Hospital, David H. Koch Center

New York, New York, 10065, United States
The Newcastle upon Tyne Hospitals NHS Foundation Trust-Freeman Hospital

Newcastle upon Tyne, Newcastle, NE7 7DN, United Kingdom
The Ohio State University Wexner Medical Center

Columbus, Ohio, 43210-1240, United States
The University of Texas Southwestern Medical Center-IDS Aston Pharmacy

Dallas, Texas, 75390-8575, United States
Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States
Tiervlei Trial Centre

Cape Town, Western Cape, 7530, South Africa
UPMC Center for Liver Diseases

New Hyde Park, New York, 11042-11114, United States
Universitair Ziekenhuis Gent

Ghent, 9000, Belgium
University Hospital Leuven

Leuven, 3000, Belgium
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University of Calgary Liver Unit - Heritage Medical Research Clinic (HMRC)

Calgary, Alberta, T2N 4Z6, Canada
University of California, Davis Medical Center

Sacramento, California, 95817, United States
University of Colorado Denver and Hospital

Aurora, Colorado, 840045, United States
University of Kansas Medical Center

Kansas City, Kansas, 66160, United States
University of Utah Hospital-Division of Gastroenterology, Hepatology, and Nutrition

Salt Lake City, Utah, 84132, United States
University of Virginia Medical Center

Charlottesville, Virginia, 22908, United States
Universitätsklinikum Frankfurt

Frankfurt, 60590, Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz

Mainz, 55131, Germany
Vanderbilt Digestive Disease Center

Nashville, Tennessee, 37232, United States
Virginia Commonwealth University Clinical Research Services Unit (CRSU)

Richmond, Virginia, 23298, United States
Yale School of Medicine, Digestive Diseases

New Haven, Connecticut, 06510, United States

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