A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment (ELFINITY)

Prospective Non-interventional, Phase IV Multicentre Study to Assess the Effectiveness, Safety and Tolerability of Elafibranor 80 mg/Day in Participants With Primary Biliary Cholangitis Receiving Treatment in a Real-world Setting.

This study will collect information from participants with Primary Biliary Cholangitis (PBC) as they use the drug elafibranor in real world setting.

PBC is a progressive rare liver disease in which tubes in the liver called bile ducts are damaged.

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

In this study the main aim is to observe the effectiveness, safety and tolerability of elafibranor in participants with PBC who are receiving treatment in real world setting. The total study duration for each participants will be 60 months (approximately 5 years).

Study Overview

• Status: Recruiting
• Conditions: Primary Biliary Cholangitis

• Study Type: Observational
• Enrollment (Estimated): 424

Contacts and Locations

• Study Contact: Name: Ipsen Clinical Study Enquiries; Phone Number: See e mail; Email: clinical.trials@ipsen.com

Study Locations

• Australia
  • Fitzroy, Australia, 3065
    • Not yet recruiting
    • St Vincent's Hospital
• Austria
  • Graz, Austria
    • Recruiting
    • Medizinische Universitaetsklinik Graz
  • Innsbruck, Austria
    • Recruiting
    • Medical University Innsbruck
  • Linz, Austria
    • Recruiting
    • Ordensklinikum Linz GmbH Barmherzige Schwestern
• Canada
  • Calgary, Canada
    • Recruiting
    • University of Calgary
  • London, Canada
    • Recruiting
    • London Health Sciences Centre (LHSC) - University Hospital
  • Ottawa, Canada
    • Recruiting
    • The Ottawa Hospital - General Campus
  • Vancouver, Canada
    • Recruiting
    • (G.I.R.I) GI Research Institute Foundation
  • Quebec
    • Montreal, Quebec, Canada, H2X 0A9
      • Not yet recruiting
      • Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
• Germany
  • Berlin, Germany
    • Recruiting
    • Charité Universitätsmedizin Berlin
  • Berlin, Germany
    • Recruiting
    • DRK Kliniken Berlin Mitte
  • Frankfurt, Germany
    • Not yet recruiting
    • Klinikum Der Johann Wolfgang Goethe-Universitaet
  • Halle, Germany
    • Recruiting
    • Studiengesellschaft BSF
  • Herne, Germany
    • Recruiting
    • Gastroenterologsiche Studiengesellschaft Herne
  • Homburg, Germany
    • Recruiting
    • Universitaet des Saarlandes
  • Leipzig, Germany
    • Recruiting
    • Universitaetsklinikum Leipzig
  • Trier, Germany
    • Withdrawn
    • Hospital of the Merciful Brothers Trier
• Greece
  • Athens, Greece
    • Recruiting
    • General Hospital of Athens Laiko
  • Heraklion, Greece
    • Recruiting
    • University Hospital of Heraklion
  • Larissa, Greece
    • Recruiting
    • University General Hospital of Larissa
  • Pátrai, Greece
    • Recruiting
    • University General Hospital of Patras
  • Thessaloniki, Greece
    • Recruiting
    • Ippokratio General Hospital of Thessaloniki
• Italy
  • Bergamo, Italy
    • Not yet recruiting
    • ASST-Ospedale Papa Giovanni XXIII
  • Catania, Italy
    • Recruiting
    • Ospedale Garibaldi Nesima
  • Florence, Italy
    • Recruiting
    • Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)
  • Milan, Italy
    • Recruiting
    • Asst Grande Ospedale Metropolitano Niguarda
  • Novara, Italy
    • Recruiting
    • Ospedale Maggiore della Carità
  • Roma, Italy
    • Recruiting
    • Fondazione Policlinico Universitario Campus Bio-medico
  • Roma, Italy
    • Not yet recruiting
    • A. Gemelli University Hospital, Catholic University of the Sacred Heart
• Spain
  • Almería, Spain
    • Recruiting
    • Hospital de Torrecardenas
  • Barcelona, Spain
    • Recruiting
    • Hospital Universitario Vall d'Hebron
  • Barcelona, Spain
    • Recruiting
    • Hospital Clinic de Barcelona (Hospital Clinic i Provincial)
  • Madrid, Spain, 28007
    • Recruiting
    • Hospital General Universitario Gregorio Maranon (HGUGM)
  • Pontevedra, Spain
    • Recruiting
    • Complexo Hospitalario Universitario de Pontevedra
  • Santander, Spain
    • Recruiting
    • Instituto De Investigacion Marques De Valdecilla (IDIVAL)
• United Arab Emirates
  • Dubai, United Arab Emirates
    • Not yet recruiting
    • Clemenceau Medical Center Hospital
  • Dubai, United Arab Emirates
    • Not yet recruiting
    • Rashid Hospital
  • Dubai, United Arab Emirates
    • Recruiting
    • Sheikh Shakhbout Medical City
  • Dubai, United Arab Emirates
    • Recruiting
    • Mediclinic Airport Road Hospital
• United Kingdom
  • Aberdeen, United Kingdom
    • Recruiting
    • Aberdeen Royal Infirmary NHS Grampian Grampian Health Board
  • Belfast, United Kingdom
    • Recruiting
    • Belfast Health and Social Care Trust - Royal Victoria Hospital
  • Birmingham, United Kingdom
    • Recruiting
    • Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
  • Bradford, United Kingdom
    • Recruiting
    • Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
  • Hull, United Kingdom
    • Recruiting
    • Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust
  • London, United Kingdom
    • Recruiting
    • King's College Hospital NHS Foundation Trust
  • Newcastle, United Kingdom
    • Recruiting
    • The Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital
  • Nottingham, United Kingdom
    • Not yet recruiting
    • Queen's Medical Centre - Nottingham University Hospitals NHS Trust
  • Oxford, United Kingdom
    • Recruiting
    • John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
• United States
  • California
    • Coronado, California, United States, 92118
      • Recruiting
      • Southern California Research Center
    • Los Angeles, California, United States, 90048
      • Not yet recruiting
      • Cedars-Sinai Medical Center
    • Sacramento, California, United States, 95817
      • Recruiting
      • University of California Davis Medical Center
  • Colorado
    • Englewood, Colorado, United States, 80113
      • Recruiting
      • South Denver Gastroenterology,P.C.
  • Connecticut
    • New Haven, Connecticut, United States, 06510
      • Recruiting
      • Yale University School Of Medicine
  • Florida
    • Miami, Florida, United States, 33136
      • Recruiting
      • Schiff Center for Liver Diseases - University of Miami
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Recruiting
      • Beth Israel Deaconess Medical Center, Liver Research Center
  • New Jersey
    • Cherry Hill, New Jersey, United States, 08034
      • Recruiting
      • Virtua Center for Liver Disease - Cherry Hill
  • New York
    • Manhasset, New York, United States, 11030
      • Recruiting
      • Northwell Health Inc, Center for Liver Disease and Transplantation
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • Recruiting
      • UNC Hospitals, The University of North Carolina at Chapel Hill
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Recruiting
      • Cleveland Clinic Foundation
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina (MUSC)
  • Texas
    • Dallas, Texas, United States, 75234
      • Recruiting
      • Liver Center of Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Baylor College of Medicine - Advanced Liver Therapies
    • Katy, Texas, United States, 77494
      • Withdrawn
      • Gastro health & Nutrition
    • McAllen, Texas, United States, 78504
      • Recruiting
      • Texas Digestive Specialists
  • Utah
    • Murray, Utah, United States, 84107
      • Recruiting
      • Intermountain Medical Center
  • Virginia
    • Richmond, Virginia, United States, 23226
      • Recruiting
      • Bon Secours Richmond Community Hospital LLC. d/b/a Bon Secours Liver Institute of Richmond
    • Richmond, Virginia, United States, 23298
      • Recruiting
      • Virginia Commonwealth University Medical Center - West Hospital
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Velocity Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with PBC for whom the treating physician has decided to start treatment with elafibranor 80 mg/day, as recommended in the approved indication.

Description

Inclusion Criteria:

• Participant has provided written informed consent and agrees to comply with the study protocol.
• Participant with PBC diagnosis.
• Participant for whom the treating physician has decided to start or participants who are currently receiving treatment with commercialized elafibranor.
• If a participant has a caregiver who agrees to complete the caregiver questionnaires, an informed consent should be collected from the caregiver before any data is collected.

Exclusion Criteria:

• Participant is currently participating or, plans to participate in an investigational drug study or medical device study containing active substance.
• Participant with known hypersensitivity to the product or to any of its excipients.
• Participant with mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of participants with response to treatment	Defined as alkaline phosphatase (ALP) <1.67 x upper limit of normal (ULN) and total bilirubin (TB) ≤ULN and ALP decrease ≥15% from baseline.	At month 6

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of participants with normalization of ALP levels		Up to 60 months following enrollment, based on routine physician follow-up visit
Percentage of participants with response to treatment	Defined as ALP<1.67 x ULN and TB≤ULN and ALP decrease ≥15% from baseline.	Up to 60 months following enrollment, based on routine physician follow-up visits
Change from baseline in liver function parameters: Serum levels of alanine aminotransferase (ALT)		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of Aspartate aminotransferase (AST)		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of Gamma-glutamyl transferase (GGT)		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of TB		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Conjugated (direct) bilirubin		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of creatinine		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of albumin		Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in pruritus based on PBC Itch score	The PBC Itch score is a simple, self-administered Patient Reported Outcome (PRO) questionnaire that measures itch intensity. It uses 7-day recall periods and asks participants to rate the intensity of their worst itch over the past 7-day period on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable).	Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in fatigue based on functional assessment of chronic illness therapy-fatigue (FACIT-Fatigue) scale	The FACIT-Fatigue is a 13-item measure that assesses self-reported fatigue and its impact upon daily activities and function. It is a subset of the longer (47-item) Functional Assessment of Cancer Therapy - Anemia (FACT-An), which includes the 27-item FACT-G and a 20-item subscale addressing additional concerns associated with the anemia of cancer and its treatment. This 20-item subscale, referred to as the anemia subscale, is comprised of 13-items that assess fatigue and its impact (the FACIT-Fatigue) and, 7 additional symptoms associated with anemia (e.g. shortness of breath, headache). participants rate their symptoms over the preceding seven days on a verbal response scale, the options range from 'not at all' / 'a little bit' / 'somewhat quite a bit' / very much'.	Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in sleep based on Pittsburgh sleep quality index (PSQI)	The PSQI was designed to evaluate overall sleep quality in the psychiatric disorders associated with sleep disturbances. Each of the questionnaire's 19-self-reported items belongs to one of seven subcategories: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. Five additional questions rated by the respondent's roommate or bed partner are included for clinical purposes and are not scored.	Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in Quality Of Life (QoL) based on PBC-40 questionnaire	The PBC-40 is a validated, PBC-specific, health-related Quality Of Life (QoL) questionnaire with 40 questions that assesses symptoms across six domains: fatigue, emotional and social, cognitive function, general symptoms and itch. Participants respond on a verbal response scale, depending on the section options range from 'never' / 'not at all' / 'strongly disagree' to 'always' / 'very much'/ 'strongly agree'. Five items (3/3 in the itch domain and 2/10 in the social domain) also include a 'does not apply' option. A score for each domain is provided (but a total score is not calculated), with each verbal response scale correlating to a score of 1-5 per item (0-5 on items with a 'does not apply' option) with 5 being the most affected.	Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in QoL based on 5-Dimensional Itch scale (5-D Itch, also known as 5-D pruritus scale)	The 5-D Itch scale assesses symptoms in terms of five domains: degree, duration, direction, disability and distribution. It is a 1 to 5 scale, with 5 being the most affected.	Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver stiffness	Measured by transient elastography (FibroScan®) and enhanced liver fibrosis (ELF) test	Up to 60 months following enrollment, based on routine physician follow-up visit
Event free survival	Event free survival from first intake of commercialized elafibranor treatment as prescribed by physician to the first occurrence of any of the following clinical outcome events: (1) death, (2) liver transplant, and (3) liver decompensation.	From baseline to up to 60 months
Percentage of participants experiencing Adverse Events (AEs), Adverse Events of Special Interests (AESIs) and special situations (SS).	An Adverse event (AE) is any untoward medical occurrence, temporally associated with the use of study intervention, whether or not related to the study intervention. AESIs are AEs that may not be serious but are of special importance to a particular drug or class of drugs.	From baseline to up to 60 months
Participant's satisfaction on treatment	Measured by treatment satisfaction questionnaire for medication (TSQM). The TSQM (version 1.4) has 14 questions divided into 4 subscales: effectiveness (items 1 to 3), side effects (items 4 to 8), convenience (items 9 to 11), and global satisfaction (items 12 to 14).	Up to 60 months following enrollment, based on routine physician follow-up visit
Participant's adherence to treatment	Adherence to the treatment will be measured based on the participant report of missed doses every month.	Up to 60 months following enrollment, based on routine physician follow-up visit
Percentage of participants with clinically significant changes in laboratory parameters	Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be graded by the investigator.	Up to 60 months following enrollment, based on routine physician follow-up visit
Percentage of participants with clinically significant changes in physical examination	Clinically significant changes in physical examination will be reported. The clinical significance will be graded by the investigator.	Up to 60 months following enrollment, based on routine physician follow-up visit
Percentage of participants developing clinically significant changes in vital signs	Clinically significant changes in vital signs will be reported. The clinical significance will be graded by the investigator.	Up to 60 months following enrollment, based on routine physician follow-up visit

Collaborators and Investigators

• Sponsor: Ipsen
• Investigators: Study Director: Ipsen Medical Director, Ipsen

Study record dates

Study Major Dates

• Study Start (Actual): October 14, 2024
• Primary Completion (Estimated): July 15, 2032
• Study Completion (Estimated): July 15, 2032

Study Registration Dates

• First Submitted: June 3, 2024
• First Submitted That Met QC Criteria: June 3, 2024
• First Posted (Actual): June 6, 2024

Study Record Updates

• Last Update Posted (Actual): May 28, 2026
• Last Update Submitted That Met QC Criteria: May 27, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Digestive System Diseases; Biliary Tract Diseases; Liver Diseases; Bile Duct Diseases; Fibrosis; Cholestasis, Intrahepatic; Cholestasis; Liver Cirrhosis; Pathological Conditions, Signs and Symptoms; Liver Cirrhosis, Biliary
• Other Study ID Numbers: CLIN-60190-461

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

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.

• IPD Sharing 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.
• IPD Sharing 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/).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No