A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
NCT ID: NCT06016842
Last Updated: 2025-12-31
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE3
276 participants
INTERVENTIONAL
2023-08-31
2029-05-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
TRIPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Elafibranor 80 mg
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.
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
Placebo
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.
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
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.
* 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 screening period: 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.
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Ipsen
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Ipsen Medical Director
Role: STUDY_DIRECTOR
Ipsen
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Arizona Liver Health
Tucson, Arizona, United States
Arkansas Diagnostic Center, PA
Little Rock, Arkansas, United States
Southern California Research Center
Coronado, California, United States
Cedars-Sinai Medical Center
Los Angeles, California, United States
GastroIntestinal BioSciences
Los Angeles, California, United States
University of California Los Angeles
Los Angeles, California, United States
University of California Davis Medical Center
Sacramento, California, United States
University of Colorado
Aurora, Colorado, United States
Peak Gastroenterology Associates
Colorado Springs, Colorado, United States
South Denver Gastroenterology, P.C.
Englewood, Colorado, United States
Rocky Mountain Gastroenterology
Littleton, Colorado, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, United States
Bolanos Clinical Research
Pembroke Pines, Florida, United States
International Center for Research
Tampa, Florida, United States
University of Kansas Medical Center (KUMC) - University of Kansas Liver Center - Hepatology Clinic
Kansas City, Kansas, United States
Louisiana Research Center, LLC
Shreveport, Louisiana, United States
University of Michigan Health System
Ann Arbor, Michigan, United States
Huron Gastroenterology Associates - Center for Digestive Care
Ypsilanti, Michigan, United States
Southwest Gastroenterology Associates, PC (SWGA)
Albuquerque, New Mexico, United States
NYU Langone Gastroenterology and Hepatology Associates
New York, New York, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Gastroenterology Center of the Midsouth
Cordova, Tennessee, United States
Texas Clinical Research Institute
Arlington, Texas, United States
American Research Corporation
Austin, Texas, United States
Rush University Medical Center - University Cardiovascular Surgeons
Dallas, Texas, United States
Liver Center of Texas
Dallas, Texas, United States
Methodist Transplant Physicians
Dallas, Texas, United States
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, United States
Baylor Scott & White All Saints Medical Center - Forth Worth
Fort Worth, Texas, United States
Liver Associates of Texas
Houston, Texas, United States
Houston Methodist Cancer Center
Houston, Texas, United States
Gastro health & Nutrition
Katy, Texas, United States
American Research Corporation at The Texas Liver Institute
San Antonio, Texas, United States
Impact Research Tx
Waco, Texas, United States
University of Virginia Medical Center
Charlottesville, Virginia, United States
Bon Secours St. Mary's Hospital of Richmond, Inc
Richmond, Virginia, United States
Virginia Commonwealth University Medical Center - West Hospital
Richmond, Virginia, United States
Medstar Georgetown Transplant Institute University Hospital (MGUH)
Columbia, Washington, United States
Velocity Clinical Research at Liver Institute Northwest
Seattle, Washington, United States
DIM Centro Medico
Buenos Aires, , Argentina
Fundacion Respirar (Centro Medico Dra. De Salvo) - Instituto Argentino de Investigaciones Clinicas (IAIC) S.R.L
Buenos Aires, , Argentina
Hospital Britanico de Buenos Aires
Buenos Aires, , Argentina
Hospital Italiano de Buenos Aires
Buenos Aires, , Argentina
Centro Medico Colon
Córdoba, , Argentina
Hospital Espanol de Mendoza
Mendoza, , Argentina
Hospital Universitario Austral
Pilar, , Argentina
Hospital El Cruce
San Juan Bautista, , Argentina
Sunshine Coast University Hospital
Birtinya, , Australia
Footscray Hospital, Western Health
Footscray, , Australia
Nepean Clinical School
Kingswood, , Australia
Monash University - Monash Health -Monash Medical Centre
Melbourne, , Australia
St George Hospital
Sydney, , Australia
Westmead Hospital
Westmead, , Australia
Universitair Ziekenhuis Antwerpen
Edegem, , Belgium
Ziekenhuis Oost-Limburg
Genk, , Belgium
Gent University Hospital
Ghent, , Belgium
Algemeen Ziekenhuis Delta
Roeselare, , Belgium
NUPEC Cardio
Belo Horizonte, , Brazil
Universidade Federal de Goias
Goiânia, , Brazil
Hospital de Clinicas de Porto Alegre (HCPA)
Porto Alegre, , Brazil
Instituto D'Or de Pesquisa e Ensino - Hospital Aliança
Salvador, , Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo
São Paulo, , Brazil
Hospital de Base - Centro Integrado de Pesquisa ( CIP )
São Paulo, , Brazil
Universidade Estadual Paulista Julio De Mesquita Filho (UNESP) Hospital das Clinicas Faculdade de Medicina de Botucatu (HCFMB)
São Paulo, , Brazil
Diagnostic-Consultative Center Aleksandrovska EOOD
Sofia, , Bulgaria
Tokuda Hospital, Dept. of Internal Medicine
Sofia, , Bulgaria
University Hospital City Clinic Cancer Center
Sofia, , Bulgaria
University of Alberta - Faculty of Medicine & Dentistry - The Centre of Excellence for Gastrointestinal Inflammation and Immunity Research (CEGIIR)
Edmonton, , Canada
University Health Network (UHN) - Toronto General Hospital (TGH) - Toronto General Research Institute (TGRI)
Toronto, , Canada
Universidad De Los Andes
Las Condes, , Chile
Centro de Estudios Clinicos e Investigaciones Medicas (CECIM)
Santiago, , Chile
Enroll SpA
Santiago, , Chile
Hospital Clinico de la Pontificia Universidad Catolica de Chile
Santiago, , Chile
University of Chile Clinical Hospital (Hospital Clinico de la Universidad de Chile)
Santiago, , Chile
Centro Medico Cedid - Centro de Diagnostico Digestivo
Viña del Mar, , Chile
Hepato-Gastroenterologie HK, s.r.o.
Hradec Králové, , Czechia
Krajska Nemocnice Liberec
Liberec, , Czechia
Artroscan
Ostrava, , Czechia
Research Site s.r.o.
Pilsen, , Czechia
Institute for Clinical and Experimental Medicine - IKEM
Prague, , Czechia
Bispebjerg Hospital
Copenhagen, , Denmark
Centre Hospitalier Regional Universitaire de Lille - Hopital Claude Huriez
Lille, , France
Hospices Civils de Lyon (HCL) - Hopital de la Croix-Rousse
Lyon, , France
Hopital Saint Joseph - Marseille
Marseille, , France
CHU de Nice, Hopital de l'Archet
Nice, , France
Assistance Publique - Hopitaux de Paris (AP-HP) - Hopital Universitaire Pitie Salpetriere (Hopital Pitie-Salpetriere)
Paris, , France
CHU Poitiers
Poitiers, , France
Clinique Pasteur
Toulouse, , France
Hopital Paul-Brousse - APHP
Villejuif, , France
Agios Savvas Regional Cancer Hospital
Athens, , Greece
General Hospital of Athens Laiko
Athens, , Greece
General Oncological Hospital of Kifisia Oi Agioi Anargyroi
Athens, , Greece
GeneIppokratio General Hospital of Thessaloniki
Thessaloniki, , Greece
Central Hospital of Northern Pest - Military Hospital
Budapest, , Hungary
Kenezy County Hospital
Debrecen, , Hungary
Szegedi Tudomanyegyetem AOK, I. sz Belgyogyaszati Klinika
Szeged, , Hungary
Institute of Gastroenterology and Liver Diseases - Soroka University Medical Center
Beersheba, , Israel
Hadassah University Hospital (HUH) - Ein-Kerem
Jerusalem, , Israel
Shaare Zedek Medical Center
Jerusalem, , Israel
Galilee Medical Center, ZIV Medical Center
Nahariya, , Israel
Galilee Medical Center
Nahariya, , Israel
Rabin Medical Center - Beilinson Hospital - Liver Institute
Petah Tikva, , Israel
Azienda Ospedaliero Universitaria Modena
Modena, , Italy
Ospedale San Gerardo
Monza, , Italy
Azienda Ospedaliero Universitaria Federico II di Napoli
Naples, , Italy
Azienda Ospedaliera di Padova - U.O.C. di Gastroenterologia
Padua, , Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Palermo, , Italy
Fondazione I.R.C.C.S. Policlinico San Matteo
Pavia, , Italy
Azienda Ospedaliero Universitaria Pisana
Pisa, , Italy
Ospedale Casa Sollievo della Sofferenza
San Giovanni Rotondo, , Italy
Hospital of Lithuanian University of Health Sciences Kaunas
Kaunas, , Lithuania
Vilnius University Hospital Santaros Klinikos
Vilnius, , Lithuania
Centro de Investigacion Medico Biologica y Terapia Avanzada
Jalisco, , Mexico
Centro de Investigacion y Gastroenterologia SC
Mexico City, , Mexico
Medical Care & Research
Yucatán, , Mexico
Canterbury District Health Board
Christchurch, , New Zealand
Waikato Hospital
Hamilton, , New Zealand
NZOZ Twoje Zdrowie EL Sp. z o.o.
Elblag, , Poland
Uniwersyteckie Centrum Kliniczne im. Prof. K. Gibinskiego Slaskiego Uniwersytetu Medycznego
Katowice, , Poland
Krakowskie Centrum Medyczne Sp.z.o.o - FutureMeds
Krakow, , Poland
Szpital Uniwersytecki w Krakowie
Krakow, , Poland
Medrise Sp. z o.o.
Lublin, , Poland
Krakowskie Centrum Medyczne Sp.z.o.o. - FutureMeds
Małogoskie, , Poland
Centrum Medyczne Medyk Sp. z o.o. Sp. K.
Rzeszów, , Poland
Niepubliczny Zaklad Opieki Zdrowotnej (NZOZ) - Poliklinika Doktora Bessera
Sosnowiec, , Poland
FutureMeds Warszawa Centrum
Warsaw, , Poland
FutureMeds sp. z o. o
Wroclaw, , Poland
Nzoz Centrum BadanKlinicznych
Wroclaw, , Poland
PlanetMed Sp. z o.o.
Wroclaw, , Poland
Centrul Pentru Studiul Metabolismului
Bucharest, , Romania
Sana S.R.L
Bucharest, , Romania
Spital Clinic Dr I Cantacuzino
Bucharest, , Romania
Cluj County Clinical Emergency Hospital
Cluj-Napoca, , Romania
Gastromedica Srl
Iași, , Romania
Gastroenterological Centre Thalion
Bratislava, , Slovakia
Univerzitna nemocnica L. Pasteura Kosice
Košice, , Slovakia
Fakultna nemocnica Nitra
Nitra, , Slovakia
Pusan National University Hospital (PNUH)
Busan, , South Korea
Keimyung University Dongsan Hospital
Daegu, , South Korea
The Catholic University of Korea Daejeon St.Mary's Hospital
Daejeon, , South Korea
Inje University Ilsan Paik Hospital
Goyang-si, , South Korea
Korea University Ansan Hospital
Gyeonggi-do, , South Korea
Inha University Hospital
Incheon, , South Korea
Kyungpook National University Hospital (KNUH)
Junggu, , South Korea
CHA Bundang Medical Center, CHA University
Seongnam-si, , South Korea
Chung-Ang University Hospital
Seoul, , South Korea
Hallym University Kangnam Sacred Heart Hospital
Seoul, , South Korea
Korea University Guro Hospital
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Seoul National University Bundang Hospital (SNUBH)
Seoul, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital, Yonsei University Health System
Seoul, , South Korea
The Catholic University of Korea, Eunpyeong St. Mary's Hospital
Seoul, , South Korea
The Catholic University of Korea, Seoul St. Mary's Hospital
Seoul, , South Korea
The Catholic University of Korea, Uijeongbu St. Mary's Hospital
Uijeongbu-si, , South Korea
Hospital Clinic i Provincial de Barcelona
Barcelona, , Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Clinica Universidad Navarra-Sede Madrid
Madrid, , Spain
Hospital General Universitario Gregorio Maranon (HGUGM)
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario Puerta de Hierro de Majadahonda
Majadahonda, , Spain
Hospital Universitario Virgen de la Victoria
Málaga, , Spain
Hospital De Montecelo
Pontevedra, , Spain
Institut d Investigacio i Innovacio Parc Tauli, Hospital Universitari Parc Tauli
Sabadell, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Hospital Universitario y Politecnico La Fe
Valencia, , Spain
Hospital Universitario Rio Hortega
Valladolid, , Spain
Hospital Universitario Miguel Servet
Zaragoza, , Spain
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2023-505251-43-00
Identifier Type: CTIS
Identifier Source: secondary_id
CLIN-60190-454
Identifier Type: -
Identifier Source: org_study_id