A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis
ConditionPrimary Biliary Cholangitis (PBC)
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorIpsen
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
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).
Trial design
Design model
Parallel
Treatments tested in this trial
Elafibranor
DrugDuration: 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
Other interventionDuration: up to an estimated 42-month (3.5-year) double-blind treatment period during which matching placebo tablet will be administered once daily
Treatment groups
276 Participants
are divided into 2 treatment groupsGroup A: Elafibranor 80 mgExperimental treatment 1 intervention
Group B: PlaceboPlacebo comparator 1 intervention
Trial outcomes
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.
Time frame
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)Secondary outcomes
1
Percentage of participants experiencing Treatment Emergent Adverse Events (TEAEs), treatment-related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs)
An adverse event (AE) is any unfavourable medical occurrence in a trial participant administered the investigational product. The AE does not necessarily have a causal relationship with the treatment. AESIs are AEs that may or may not be serious but are of special importance to a particular drug or class of drugs.
Time frame
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)2
Percentage of participants developing clinically significant changes in physical examination findings
Complete physical examination at screening and targeted examination at all other clinical visit timepoints.
Time frame
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)3
Percentage of participants developing clinically significant changes in vital signs
Percentage of participants with clinically significant changes in Vital Signs will be reported. The clinical significance will be graded by the investigator.
Time frame
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years)4
Percentage of participants developing clinically significant changes in Electrocardiogram (ECG) readings.
Percentage of participants with clinically significant changes in ECG readings will be reported. The clinical significance will be graded by the investigator.
Time frame
From baseline until 4 weeks after the last dose of study intervention (maximum duration of 3.5 years) Source ClinicalTrials.gov