Primary Biliary CholangitisELFINITYNCT06447168
A Study Observing Everyday Effectiveness and Safety of the Drug Elafibranor in Participants With Primary Biliary Cholangitis Who Are Receiving Ongoing Treatment
An observational study of Primary Biliary Cholangitis, run by Ipsen at 72 sites in 11 countries.
Trial summary
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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Primary Biliary Cholangitis
- How many people
- 424 participants
- Where
- 72 sitesin 11 countries
- Study dates
- 14 Oct 2024to 15 Jul 2032
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
72 sites in 11 countries.
72 sites in 11 countries
United States19 sites
Beth Israel Deaconess Medical Center, Liver Research Center
Boston, Massachusetts, 02215
UNC Hospitals, The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425
Virtua Center for Liver Disease - Cherry Hill
Cherry Hill, New Jersey, 08034
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Southern California Research Center
Coronado, California, 92118
Liver Center of Texas
Dallas, Texas, 75234
South Denver Gastroenterology,P.C.
Englewood, Colorado, 80113
Baylor College of Medicine - Advanced Liver Therapies
Houston, Texas, 77030
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Northwell Health Inc, Center for Liver Disease and Transplantation
Manhasset, New York, 11030
Texas Digestive Specialists
McAllen, Texas, 78504
Schiff Center for Liver Diseases - University of Miami
Miami, Florida, 33136
Intermountain Medical Center
Murray, Utah, 84107
Yale University School of Medicine
New Haven, Connecticut, 06510
Bon Secours Richmond Community Hospital LLC. d/b/a Bon Secours Liver Institute of Richmond
Richmond, Virginia, 23226
Virginia Commonwealth University Medical Center - West Hospital
Richmond, Virginia, 23298
University of California Davis Medical Center
Sacramento, California, 95817
Velocity Clinical Research
Seattle, Washington, 98105
United Kingdom9 sites
Aberdeen Royal Infirmary NHS Grampian Grampian Health Board
Aberdeen
Belfast Health and Social Care Trust - Royal Victoria Hospital
Belfast
Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
Birmingham
Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
Bradford
Hull Royal Infirmary - Hull University Teaching Hospitals NHS Trust
Hull
King's College Hospital NHS Foundation Trust
London
The Newcastle upon Tyne Hospitals NHS Foundation Trust - Freeman Hospital
Newcastle
Queen's Medical Centre - Nottingham University Hospitals NHS Trust
Nottingham
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
Oxford
Germany7 sites
Charite Universitatsmedizin Berlin
Berlin
DRK Kliniken Berlin Mitte
Berlin
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt
Studiengesellschaft BSF
Halle
Gastroenterologsiche Studiengesellschaft Herne
Herne
Universitaet des Saarlandes
Homburg
Universitaetsklinikum Leipzig
Leipzig
Italy7 sites
ASST-Ospedale Papa Giovanni XXIII
Bergamo
Ospedale Garibaldi Nesima
Catania
Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)
Florence
ASST Grande Ospedale Metropolitano Niguarda
Milan
Ospedale Maggiore Della Carita
Novara
A. Gemelli University Hospital, Catholic University of the Sacred Heart
Roma
Fondazione Policlinico Universitario Campus Bio-Medico
Roma
Spain6 sites
Hospital de Torrecardenas
Almería
Hospital Clinic de Barcelona (Hospital Clinic i Provincial)
Barcelona
Hospital Universitario Vall d'Hebron
Barcelona
Hospital General Universitario Gregorio Maranon (HGUGM)
Madrid, 28007
Complexo Hospitalario Universitario de Pontevedra
Pontevedra
Instituto De Investigacion Marques De Valdecilla (IDIVAL)
Santander
Canada5 sites
University of Calgary
Calgary
London Health Sciences Centre (LHSC) - University Hospital
London
Centre de Recherche du Centre Hospitalier de l'Universite de Montreal (CRCHUM)
Montreal, Quebec, H2X 0A9
The Ottawa Hospital - General Campus
Ottawa
(G.I.R.I) GI Research Institute Foundation
Vancouver
Greece5 sites
General Hospital of Athens Laiko
Athens
University Hospital of Heraklion
Heraklion
University General Hospital of Larissa
Larissa
University General Hospital of Patras
Pátrai
Ippokratio General Hospital of Thessaloniki
Thessaloniki
South Korea5 sites
Pusan National University Hospital (PNUH)
Busan
Seoul National University Bundang Hospital (SNUBH)
Seongnam-si
Samsung Medical Center
Seoul
Seoul National University Hospital
Seoul
Severance Hospital, Yonsei University Health System
Seoul
United Arab Emirates4 sites
Clemenceau Medical Center Hospital
Dubai
Mediclinic Airport Road Hospital
Dubai
Rashid Hospital
Dubai
Sheikh Shakhbout Medical City
Dubai
Austria3 sites
Medizinische Universitaetsklinik Graz
Graz
Medical University Innsbruck
Innsbruck
Ordensklinikum Linz GmbH Barmherzige Schwestern
Linz
Australia2 sites
Royal Prince Alfred Hospital
Camperdown
St Vincent's Hospital
Fitzroy, 3065
About this study
Official title: 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.
Read more
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).
- Conditions
- Primary Biliary Cholangitis
- Medical subject headings
- Liver Cirrhosis, Biliary
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: At month 6
Secondary outcomes (22)
Percentage of participants with normalization of ALP levels
Measured: 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.
Measured: 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)
Measured: 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)
Measured: 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)
Measured: Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of TB
Measured: Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Conjugated (direct) bilirubin
Measured: Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of creatinine
Measured: Up to 60 months following enrollment, based on routine physician follow-up visit
Change from baseline in liver function parameters: Serum levels of albumin
Measured: 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).
Measured: 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'.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Up to 60 months following enrollment, based on routine physician follow-up visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Jun 2024
- Study start14 Oct 2024
- Primary completion15 Jul 2032 (estimated)
- Study completion15 Jul 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Biliary CholangitisStudies of the same condition, open and under way.
- More trials run by IpsenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 424 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ipsen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ipsen Medical Director, Study director, Ipsen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06447168
- Sponsor's protocol ID
- CLIN-60190-461
- Acronym
- ELFINITY
- Status last verified
- Oct 2026
- First submitted
- 3 Jun 2024
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.