The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring of the bile ducts in the liver. Over time, this can lead to liver damage and serious health problems, including the need for a liver transplant and death.
In this study, about 350 participants with large duct PSC will take part. Participants will be randomized to receive either elafibranor 120 mg once daily or a placebo (a tablet with no active medicine). The study includes a screening period, an treatment period, and a post-treatment safety follow-up.
During the study, participants will undergo routine clinical assessments, laboratory testing, imaging evaluations, and complete patient-reassessments to evaluate liver disease progression, symptoms, quality of life and safety.
Following the end of treatment, participants will complete a safety follow-up period at approximately four weeks. Participants may withdraw from the study at any time. Each participant may be in the study for several years, as the treatment period will continue until the study reaches enough health events among participants, which is expected to take about 5 years.
Adults participants aged 18 years or older
Confirmed diagnosis of primary sclerosing cholangitis based on standard clinical, biochemical, and imaging criteria
Compensated liver disease at screening
Stable background therapy, where applicable prior to study entry
Participants with cirrhosis who are also classified as Child-Pugh B or C based on the Child Pugh score.
History of biliary intervention within 60 days prior to the screening period, and/or presence of percutaneous drain or bile duct stent at SV.
History of bacterial cholangitis, and/or participant on antibiotics for prophylaxis of recurrent cholangitis within 60 days prior to the SV.
History or any current suspicion of cholangiocarcinoma or hepatocellular carcinoma
Parallel
Elafibranor
DrugRound and orange film coated tablet of 120 mg
Placebo
Other interventionRound and orange film coated tablet of placebo
Event-Free Survival
Event-free survival is defined as the time from randomisation to either adjudicated disease progression or death, whichever occurs first.
Percentage of participants with ALP level within the pre-defined level
Difference between elafibranor and placebo in the percentage of participants with alkaline phosphatase (ALP) within the pre-defined level at Week 48.
Change from baseline in severity of a patient-reported cholestatic symptom: Pruritus
Symptom will be assessed using a patient-reported outcome scale
Change from baseline in severity of a patient-reported cholestatic symptom: Fatigue
Symptom will be assessed using a patient-reported outcome scale
Percentage of participants experiencing treatment-emergent adverse events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs)
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
