A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
ELFIDENCE
A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)
2 other identifiers
interventional
276
26 countries
180
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Aug 2023
Longer than P75 for phase_3
180 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 22, 2023
CompletedFirst Posted
Study publicly available on registry
August 30, 2023
CompletedStudy Start
First participant enrolled
August 31, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 31, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 31, 2029
April 30, 2026
April 1, 2026
5.8 years
August 22, 2023
April 29, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Event-free survival
Event-free survival is defined as the time from randomisation to either adjudicated disease progression or death, whichever occurs first.
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
Secondary Outcomes (57)
Percentage of participants experiencing Treatment Emergent Adverse Events (TEAEs), treatment-related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs)
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
Percentage of participants developing clinically significant changes in physical examination findings
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
Percentage of participants developing clinically significant changes in vital signs
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
Percentage of participants developing clinically significant changes in Electrocardiogram (ECG) readings.
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, coagulation and urinalysis)
From baseline until 4 weeks after the end of treatment (maximum duration of 3.5 years)
- +52 more secondary outcomes
Study Arms (2)
Elafibranor 80 mg
EXPERIMENTALParticipants 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.
Placebo
PLACEBO COMPARATORParticipants 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.
Interventions
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
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
You may qualify if:
- 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.
You may not qualify if:
- 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.
- +22 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Ipsenlead
Study Sites (180)
Arizona Liver Health
Tucson, Arizona, 85641, United States
Arkansas Diagnostic Center, PA
Little Rock, Arkansas, 55130, United States
Southern California Research Center
Coronado, California, 92118, United States
Cedars-Sinai Medical Center
Los Angeles, California, 90048, United States
GastroIntestinal BioSciences
Los Angeles, California, 90067, United States
University of California Los Angeles
Los Angeles, California, 90404, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
University of Colorado
Aurora, Colorado, 80045, United States
Peak Gastroenterology Associates
Colorado Springs, Colorado, 80829, United States
South Denver Gastroenterology, P.C.
Englewood, Colorado, 80113, United States
Rocky Mountain Gastroenterology
Littleton, Colorado, 80120, United States
University Of Miami School Of Medicine, Center For Liver Diseases
Miami, Florida, 33136, United States
Bolanos Clinical Research
Pembroke Pines, Florida, 12105, United States
International Center for Research
Tampa, Florida, 33614, United States
University of Kansas Medical Center (KUMC) - University of Kansas Liver Center - Hepatology Clinic
Kansas City, Kansas, 66160, United States
Louisiana Research Center, LLC
Shreveport, Louisiana, 71103, United States
University of Michigan Health System
Ann Arbor, Michigan, 48109, United States
Huron Gastroenterology Associates - Center for Digestive Care
Ypsilanti, Michigan, 48197, United States
Southwest Gastroenterology Associates, PC (SWGA)
Albuquerque, New Mexico, 87109, United States
NYU Langone Gastroenterology and Hepatology Associates
New York, New York, 10016, United States
University of Pittsburgh
Pittsburgh, Pennsylvania, 15213, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Gastroenterology Center of the Midsouth
Cordova, Tennessee, 38138, United States
Texas Clinical Research Institute
Arlington, Texas, 22201, United States
American Research Corporation
Austin, Texas, 78757, United States
Rush University Medical Center - University Cardiovascular Surgeons
Dallas, Texas, 60612, United States
Liver Center of Texas
Dallas, Texas, 75203, United States
Methodist Transplant Physicians
Dallas, Texas, 75203, United States
University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390, United States
Baylor Scott & White All Saints Medical Center - Forth Worth
Fort Worth, Texas, 76104, United States
Liver Associates of Texas
Houston, Texas, 77030, United States
Houston Methodist Cancer Center
Houston, Texas, 77584, United States
Gastro health & Nutrition
Katy, Texas, 77904, United States
American Research Corporation at The Texas Liver Institute
San Antonio, Texas, 78015, United States
Impact Research Tx
Waco, Texas, 76710, United States
University of Virginia Medical Center
Charlottesville, Virginia, 22903, United States
Bon Secours St. Mary's Hospital of Richmond, Inc
Richmond, Virginia, 23226, United States
Virginia Commonwealth University Medical Center - West Hospital
Richmond, Virginia, 23298, United States
Medstar Georgetown Transplant Institute University Hospital (MGUH)
Columbia, Washington, 20007, United States
Velocity Clinical Research at Liver Institute Northwest
Seattle, Washington, 98105, 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
Hospital Henri Mondor
Créteil, 94000, France
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
Hopital Pitie-Salpetriere - APHP
Paris, 75013, 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
Rambam Health Care Campus (RHCC)
Haifa, 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
Padova, 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
Hospital Sultanah Aminah
Johor Bahru, Malaysia
Hospital Queen Elizabeth II
Kota Kinabalu, Malaysia
University of Malaya Medical Centre
Kuala Lumpur, Malaysia
Centro de Investigacion Clinica y Medicina Traslacional
Guadalajara, Mexico
Centro de Investigacion Medico Biologica y Terapia Avanzada
Jalisco, Mexico
Centro de Investigacion y Gastroenterologia SC
Mexico City, Mexico
Hospital Angeles Valle Oriente
Monterrey, 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
Hospital Dr. Nelio Mendonca
Funchal, 9004-514, Portugal
Unidade Local de Saúde de Trás-os-Montes e Alto Douro, E. P. E
Vila Real, 5000-508, Portugal
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, 700111, 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
Hanyang University Seoul 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, 46026, Spain
Hospital Universitario Rio Hortega
Valladolid, Spain
Hospital Universitario Miguel Servet
Zaragoza, Spain
Chiang Mai University
Chiang Mai, Thailand
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Ipsen Medical Director
Ipsen
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 22, 2023
First Posted
August 30, 2023
Study Start
August 31, 2023
Primary Completion (Estimated)
May 31, 2029
Study Completion (Estimated)
May 31, 2029
Last Updated
April 30, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- Time Frame
- Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
- Access Criteria
- Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.