Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
NCT ID: NCT04526665
Last Updated: 2025-12-03
Study Results
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View full resultsBasic Information
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ACTIVE_NOT_RECRUITING
PHASE3
161 participants
INTERVENTIONAL
2020-09-24
2028-12-01
Brief Summary
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PBC is a slowly progressive disease characterized by damage of the bile ducts in the liver, leading to a buildup of bile acids which causes further damage. The liver damage in PBC may lead to scarring (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.
The main aim of this study is to determine if elafibranor (the study drug) is better than placebo (a dummy treatment) at decreasing the levels of a specific blood test (alkaline phosphatase) that provides information about participant's disease. This study will also evaluate the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itchy skin (pruritus) and tiredness (fatigue).
This study has two main parts:
Part 1 will compare a daily dose of elafibranor to a daily dose of placebo and will last between a minimum of one year and a maximum of two years.
Part 2, all participants will receive elafibranor for a period of up to 5 years or until the total treatment duration (part 1 and part 2) reaches 6 years, whichever occurs first.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Elafibranor 80mg double-blind
Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
Elafibranor 80mg
Elafibranor 80mg daily
Placebo
Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
Placebo
Placebo daily
Elafibranor 80mg open label
Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
Elafibranor 80mg
Elafibranor 80mg daily
Interventions
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Elafibranor 80mg
Elafibranor 80mg daily
Placebo
Placebo daily
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
75 Years
ALL
No
Sponsors
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Ipsen
INDUSTRY
Responsible Party
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Principal Investigators
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Ipsen Medical Director
Role: STUDY_DIRECTOR
Ipsen
Locations
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The Institute for Liver Health
Chandler, Arizona, United States
Keck Medical Center of USC
Los Angeles, California, United States
Ruane Clinical Research Group Inc.
Los Angeles, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
University of California, Davis Medical Center
Sacramento, California, United States
California Pacific Medical Center - Sutter Pacific Medical Foundation
San Francisco, California, United States
University of Colorado Denver and Hospital
Aurora, Colorado, United States
South Denver Gastroenterology, P.C.
Englewood, Colorado, United States
Yale School of Medicine, Digestive Diseases
New Haven, Connecticut, United States
Encore Borland-Groover Clinical Research
Jacksonville, Florida, United States
Schiff Center for Liver Diseases/University of Miami
Miami, Florida, United States
Digestive Healthcare of Georgia
Atlanta, Georgia, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Beth Israel Deaconess Medical Center (BIDMC)
Boston, Massachusetts, United States
Henry Ford Health System
Novi, Michigan, United States
Saint Louis University
St Louis, Missouri, United States
UPMC Center for Liver Diseases
New Hyde Park, New York, United States
NYU Langone Health / NYU Grossman School of Medicine
New York, New York, United States
Columbia University Medical Center - Center for Liver Disease and Transplantation
New York, New York, United States
The New York-Presbyterian Hospital, David H. Koch Center
New York, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Carolinas Centre for Liver disease/ Atrium Health
Huntersville, North Carolina, United States
Consultants for Clinical Research
Cincinnati, Ohio, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
The Ohio State University Wexner Medical Center
Columbus, Ohio, United States
Investigational Drug Service Pharmacy Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Thomas Jefferson University
Philadelphia, Pennsylvania, United States
Medical University of South Carolina- College of Medicine
Charleston, South Carolina, United States
Vanderbilt Digestive Disease Center
Nashville, Tennessee, United States
Texas Clinical Research Institute, LLC
Arlington, Texas, United States
The Liver Institute at Methodist Dallas Medical Center
Dallas, Texas, United States
Liver Center of Texas, PLLC
Dallas, Texas, United States
The University of Texas Southwestern Medical Center-IDS Aston Pharmacy
Dallas, Texas, United States
Texas Digestive Disease Consultants dba GI Alliance
Fort Worth, Texas, United States
Liver Associates of Texas, P.A.
Houston, Texas, United States
St. Luke's Health-Baylor St Luke's Medical center - Advanced Liver Therapies Research
Houston, Texas, United States
American Research Corporation
San Antonio, Texas, United States
Gastro health & Nutrition-Victoria
Victoria, Texas, United States
Intermountain Medical Center - Transplant Services
Murray, Utah, United States
University of Utah Hospital-Division of Gastroenterology, Hepatology, and Nutrition
Salt Lake City, Utah, United States
University of Virginia Medical Center
Charlottesville, Virginia, United States
Maryview Hospital Inc, Bon Secours Liver Institute of Hampton Roads
Newport News, Virginia, United States
Richmond Community Hospital LLC, Bon Secours Liver Institute of Richmond
Richmond, Virginia, United States
Virginia Commonwealth University Clinical Research Services Unit (CRSU)
Richmond, Virginia, United States
Liver Institute Northwest
Seattle, Washington, United States
Hospital Italiano de Buenos Aires
CABA, Buenos Aires, Argentina
Hospital Alemán
Caba, Buenos Aires, Argentina
Hospital Británico de Buenos Aires
CABA, Buenos Aires, Argentina
Hospital Italiano de La Plata
La Plata, Buenos Aires, Argentina
Hospital Espanol De Mendoza
Godoy Cruz, Mendoza Province, Argentina
Hospital Provincial del Centenario
Rosario, Santa Fe Province, Argentina
Hôpital Erasme
Brussels, , Belgium
Antwerp University Hospital
Edegem, , Belgium
Universitair Ziekenhuis Gent
Ghent, , Belgium
University Hospital Leuven
Leuven, , Belgium
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo-HCFMUSP
São Paulo, São Paulo, Brazil
University of Calgary Liver Unit - Heritage Medical Research Clinic (HMRC)
Calgary, Alberta, Canada
Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
Vancouver, British Columbia, Canada
Shared Health Inc.-Operating as Health Sciences Centre-John Buhler Research Centre
Winnipeg, Manitoba, Canada
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, , Canada
Hospital de La Serena
La Serena, Coquimbo Region, Chile
Centro de Investigaciones Clinicas (CIC)
Viña del Mar, Región de Valparaíso, Chile
Clínica Universidad de los Andes
Santiago, , Chile
Hospital Clínico Universidad de Chile
Santiago, , Chile
Centro de Investigacion Clinica Universidad Catolica CICUC
Santiago, , Chile
CHU Amiens Picardie
Amiens, , France
Hôpital Henri Mondor
Créteil, , France
CHU Grenoble Alpes - Hôpital Albert Michallon
Grenoble, , France
Centre Hospitalier Régional d'Orléans
Orléans, , France
Hôpital Saint-Antoine
Paris, , France
Hôpital Cochin
Paris, , France
Hôpital Pitié-Salpétrière
Paris, , France
Hôpital Haut Lévêque
Pessac, , France
Hôpital Robert Debré - CHU de Reims
Reims, , France
Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre
Strasbourg, , France
Gastro - Sudien
Berlin, , Germany
Charite - Universitätsmedizin Berlin- CVK - Medizinische Klinik
Berlin, , Germany
Universitätsklinikum Frankfurt
Frankfurt, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Center for Gastroenterology and Hepatology (GHZ) Kiel
Kiel, , Germany
EUGASTRO GmbH
Leipzig, , Germany
Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Mainz, , Germany
Ospedale Civile di Baggiovara-Struttura Complessa di Medicina ad indririzzo Metabolico Nutrizionale
Modena, Località Baggiovara, Italy
Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
Bologna, , Italy
Azienda Ospedaliera San Paolo, Dipartimento di Scienza della Salute, UO Medicina VI e Pathologia e Gastroenterologia
Milan, , Italy
ASST Monza - Ospedale San Gerardo, Gastroenterologia
Monza, , Italy
Azienda Ospedale- Università degli Studi di Padova UOC Gastroenterologia
Padua, , Italy
Azienda Ospedaliera Universitaria Policlinico P. Giaccone, UOC di Gastroenterologia-Dip.Medicina Interna e Specialistica
Palermo, , Italy
Tiervlei Trial Centre
Cape Town, Western Cape, South Africa
Groote Schuur Hospital, University of Cape Town - Clinical Research Centre
Cape Town, Western Cap, South Africa
Mediclinic Constantiaberg, North Suites
Cape Town, , South Africa
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Clinic de Barcelona
Barcelona, , Spain
Hospital General Universitario Gregorio Marañón
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Puerta de Hierro -Madrid
Madrid, , Spain
Complexo Hospitalario Universitario de Pontevedra
Pontevedra, , Spain
Hospital Universitario Donostia
San Sebastián, , Spain
Hospital Universitario Marqués de Valdecilla
Santander, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Inselspital, Universitätsspital Bern Universitätsklinik für Viszerale chirurgie und Medizin Hepatologie
Bern, , Switzerland
Fondazione Epatocentro Ticino
Lugano, , Switzerland
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, Bornova, Turkey (Türkiye)
Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi, Gastroentoloji Bilim Dali
Istanbul, Pendik, Turkey (Türkiye)
Hacette Universitesi Hastenesi IC, Hastaliklari Anabilim Dali, Gastronenteroloji Bilim Dali, Mithapasa Cad. Hacettepe Mah.
Ankara, , Turkey (Türkiye)
The Newcastle upon Tyne Hospitals NHS Foundation Trust-Freeman Hospital
Newcastle upon Tyne, Newcastle, United Kingdom
Belfast Health and Social Care Trust-Royal Victoria Hospital
Belfast, , United Kingdom
Frimley Health NHS Foundation Trust - Frimley Park Hospital
Camberley, , United Kingdom
Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
Cambridge, , United Kingdom
Hull University Teaching Hospitals NHS Trust , Hull Royal Infirmary
Hull, , United Kingdom
King's College Hospital. King's College NHS Foundation Trust
London, , United Kingdom
Nottingham University Hospitals NHS Trust - Queen's Medical Centre (QMC)
Nottingham, , United Kingdom
Plymouth Hospitals NHS Trust, Derriford Hospital
Plymouth, , United Kingdom
Countries
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References
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Kowdley KV, Bowlus CL, Levy C, Akarca US, Alvares-da-Silva MR, Andreone P, Arrese M, Corpechot C, Francque SM, Heneghan MA, Invernizzi P, Jones D, Kruger FC, Lawitz E, Mayo MJ, Shiffman ML, Swain MG, Valera JM, Vargas V, Vierling JM, Villamil A, Addy C, Dietrich J, Germain JM, Mazain S, Rafailovic D, Tadde B, Miller B, Shu J, Zein CO, Schattenberg JM; ELATIVE Study Investigators' Group; ELATIVE Study Investigators' Group. Efficacy and Safety of Elafibranor in Primary Biliary Cholangitis. N Engl J Med. 2024 Feb 29;390(9):795-805. doi: 10.1056/NEJMoa2306185. Epub 2023 Nov 13.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2019-004941-34
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
GFT505B-319-1
Identifier Type: -
Identifier Source: org_study_id
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