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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
350
Round and orange film coated tablet of 120 mg
Round and orange film coated tablet of placebo
Arizona Liver Health
Chandler, Arizona, United States
RECRUITINGSouthern California Research Center
Coronado, California, United States
RECRUITINGPeak Gastroenterology Associates
Colorado Springs, Colorado, United States
RECRUITINGSouth Denver Gastroenterology,P.C.
Englewood, Colorado, United States
RECRUITINGRocky Mountain Gastroenterology (RMG)
Littleton, Colorado, United States
RECRUITINGIndiana University School of Medicine
Indianapolis, Indiana, United States
RECRUITINGMercy Medical Center, Inc
Baltimore, Maryland, United States
RECRUITINGUniversity Gastroenterology
Providence, Rhode Island, United States
RECRUITINGMedical University of South Carolina (MUSC)
Charleston, South Carolina, United States
RECRUITINGGastrointestinal Associates of Northeast Tennessee
Johnson City, Tennessee, United States
RECRUITING...and 3 more locations
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 the end of treatment (estimated up to 5 years)
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.
Time frame: From baseline to Week 48
Change from baseline in severity of a patient-reported cholestatic symptom: Pruritus
Symptom will be assessed using a patient-reported outcome scale
Time frame: From baseline to Week 24
Change from baseline in severity of a patient-reported cholestatic symptom: Fatigue
Symptom will be assessed using a patient-reported outcome scale
Time frame: From baseline to Week 48
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
Time frame: From baseline until the end of treatment (estimated up to 5 years)
Percentage of participants developing clinically significant changes in physical examination findings
The clinical significance will be graded by the investigator
Time frame: From baseline until the end of treatment (estimated up to 5 years)
Percentage of participants developing clinically significant changes in vital signs
The clinical significance will be graded by the investigator
Time frame: From baseline until the end of treatment (estimated up to 5 years)
Percentage of participants developing clinically significant changes in Electrocardiogram (ECG) readings.
The clinical significance will be graded by the investigator
Time frame: From baseline until the end of treatment (estimated up to 5 years)
Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, coagulation and urinalysis)
The clinical significance will be graded by the investigator
Time frame: From baseline until the end of treatment (estimated up to 5 years)
Percentage of participants developing cholangiocarcinoma (CCA), hepatocellular carcinoma (HCC), or colorectal cancer (CRC)
Time frame: From baseline until the end of treatment (estimated up to 5 years)
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.