A Phase 3 interventional study of Elafibranor in Primary Biliary Cholangitis, sponsored by Ipsen. Active, not recruiting at 16 sites in Japan. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-10-01.
Sponsored by Ipsen · Phase 3, Interventional, and Treatment
The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease.
PBC is a slowly progressive disease characterised by damage of 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 scarring (cirrhosis).
PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. In this study all participants will receive a daily dose of elafibranor (the study intervention).
The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease.
There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored.
Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to approximately 6 years
208 studies on the registry are indexed under Liver Cirrhosis, Biliary; 44 are open to participants now.
This study's enrollment of 18 is below the median of 60 across 150 interventional studies indexed under Liver Cirrhosis, Biliary.
Browse Liver Cirrhosis, Biliary studies →Ipsen is the lead sponsor of 282 studies on the registry; 16 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 19 (79%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria
Administration of the following medications are prohibited as specified below:
Participants will take the study intervention by mouth (swallow whole) once daily
Drug: Elafibranor
Tablet of 80 mg
Percentage of participants with Alkaline phosphatase (ALP) <1.67x ULN, ALP decrease ≥15% and Total Bilirubin (TB) ≤ ULN
Time frame: At Week 52
Percentage of Participants who normalised ALP Levels
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in ALP Levels
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of Participants With ALP Level response
Defined as 10%, 20% and 40% ALP reduction from baseline
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP <1.5×ULN, ALP decrease ≥40% and TB ≤ULN.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP <3×ULN, aspartate aminotransferase (AST) <2×ULN and Total Bilirubin (TB) <1 mg/dL (Paris I criteria).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP ≤1.5×ULN, AST ≤1.5×ULN and TB ≤ULN (Paris II criteria).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with TB response rate of 15% change.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with normalisation of abnormal TB and/or albumin (Rotterdam criteria).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with TB ≤0.6×ULN
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP ≤1.67×ULN and TB ≤1 mg/dL.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with no worsening of TB Levels
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with complete biochemical response
Defined as normal ALP; TB; AST; alanine aminotransferase (ALT); albumin; and INR.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP ≤1.67×ULN, ALP decrease ≥15% and TB ≤ULN.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP <1.5×ULN, ALP decrease ≥15% and TB ≤ULN.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants with ALP ≤ULN and TB ≤ULN.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC risk scores based in Global PBC Study Group (GLOBE) score
The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC risk scores based on United Kingdom (UK)-PBC score.
PBC risk score developed by United Kingdom (UK)-PBC Consortium is a scoring system and the calculation is based on laboratory test measurements and upper limits of normal (ULN) for the total bilirubin (BIL12); alanine transaminase or aspartate transaminase (TA12), and alkaline phosphatase (ALP12) after at least 12 months of UDCA, and the laboratory test measurements and lower limits of normal (LLN) for the serum albumin and platelet count in the same timeframe. A high number is indicative of a worse score.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Response based on the normalisation of bilirubin.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Response based on the normalisation of albumin Levels.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in hepatobiliary injury and liver function
Hepatobiliary injury and liver function will be measured by AST, ALT, gamma-glutamyl transferase (GGT), 5'nucleotidase (5' NT), total and conjugated bilirubin, albumin, INR, and ALP fractionated (hepatic).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in lipid parameters
Lipid parameters as measured by total cholesterol (TC), low-density lipoprotein cholesterol ( LDL-C), high-density lipoprotein cholesterol (HDL-C), calculated very low-density lipoprotein cholesterol (VLDL-C) and triglycerides (TG).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in liver stiffness measurement (LSM)
Assessed by vibration-controlled transient elastography (VCTE) using Fibroscan®
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in pruritus
Based on PBC Worst Itch Numeric Rating Scale (NRS) score in participants with baseline PBC Worst Itch NRS score ≥4.
Time frame: At Week 26, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Proportion of responders in PBC Worst Itch NRS according to clinically meaningful change from baseline
At least 30% reduction; and one point, two points or three points decrease in score in participants with a baseline NRS score ≥4.
Time frame: At Week 26 and at Week 52 and every 26 weeks until the end of study (up to 6 years)
Proportion of participants with no worsening of pruritus from baseline
Time frame: At Week 26 and at Week 52 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in 5D-Itch scale
Questionnaire that assesses symptoms in terms of 5 domains: degree, duration, direction, disability and distribution. Participants rate their symptoms over the preceding 2-week period on a 1 to 5 scale, with 5 being the most affected.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7a scores
PROMIS Fatigue Short Form 7a scores consists of 7 items that measure both the experience of fatigue and the interference of fatigue on daily activities over the past week. Response options are on a 5-point Likert scale, ranging from 1 to 5. Scores can range from 7 to 35, with higher scores indicating greater fatigue.
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Epworth Sleepiness Scale (ESS).
Self-administered questionnaire that consists of 8 questions asking to rate how likely it is to fall asleep in different situations commonly encountered in daily life (each question can be scored from 0 to 3 points; '0' indicates no sleepiness, '3' indicates significant sleepiness). It provides a total score which has been shown to relate to the participant's level of daytime sleepiness (total score range 0-24 points).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in PBC-40.
40-item questionnaire that assesses symptoms across 6 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'. Six items (3/3 in the itch domain, 2/10 in the social domain, and 1/7 in the general symptoms 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 to 5 per item (0 to 5 on items with a 'does not apply' option) with 5 being the most affected (greatest burden).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in EuroQol 5-dimensional 5-level questionnaire (EQ-5D-5L)
Self-administered standardised questionnaire that assesses the 5-dimensions of mobility, self-care, usual activities, pain/discomfort, anxiety/depression descriptively (each dimension has 5 levels) and the overall health state via an EQ Visual Analogue Scale (VAS).
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in Patient Global Impression of Severity (PGI-S)
A 1-item, 5-point scale designed to assess the participant's impression of change in disease severity since the baseline visit
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Patient Global Impression of Change (PGI-C)
A 1-item, 5-point scale designed to assess the participant's impression of change in disease severity since the baseline visit
Time frame: At Week 4, Week 13, Week 26, Week 39, and Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)]
Time to the first occurrence of each of individual adjudicated clinical outcome events
Time frame: At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)
Percentage of participants who experience Treatment Emergent Adverse Events (TEAEs), treatment related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs).
An Adverse Event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered 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.
Time frame: From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in physical examination findings
Clinically significant change in physical examination findings will be reported. The clinical significance will be graded by the investigator.
Time frame: From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in vital signs
Clinically significant change in vital signs will be reported. The clinical significance will be graded by the investigator.
Time frame: From baseline to end of study (up to 6 years)
Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, liver tests, renal tests (including urinalysis).
Clinically significant change in laboratory parameters will be reported. The clinical significance will be graded by the investigator.
Time frame: From baseline to end of study (up to 6 years)
Percentage of participants who develop clinically significant changes from baseline in ECG readings
Clinically significant change in ECG readings. The clinical significance will be graded by the investigator.
Time frame: From baseline to end of study (up to 6 years)
Change from baseline in fasting plasma glucose (FPG) Levels
Time frame: At Week 52 and every 26 weeks until the end of study (up to 6 years)
Change from baseline in serum markers of bone turnover and in bone mineral density (hip and lumbar)
Assessed by dual-energy X-ray absorptiometry (DEXA) scanning.
Time frame: At Week 52 and every 26 weeks until the end of study (up to 6 years)
Area under the plasma concentration-time curve from time 0 to 24 hours: AUC0-24
Time frame: At Week 4
Maximum (peak) plasma drug concentration: Cmax
Time frame: At Week 4
Time to reach maximum (peak) plasma concentration following drug administration): Tmax
Time frame: At Week 4
Plan to share: 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.
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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