Phase 3 Study of Elafibranor to Treat Adults With Primary Sclerosing Cholangitis PSC

What this Trial Is About

This research aims to evaluate how well and safely elafibranor works compared to a placebo in adults with Primary Sclerosing Cholangitis PSC, a rare liver disease that causes inflammation and scarring of bile ducts. PSC can lead to serious liver damage, including the need for liver transplant or death. The study is a Phase III trial involving about 350 adults with large duct PSC, conducted to understand treatment effects over an extended period. Participants will be randomly assigned to take either 120 mg of elafibranor or a placebo tablet once daily. The study includes a screening period, a treatment phase expected to last about five years until enough health events occur, and a post-treatment safety follow-up approximately four weeks after treatment ends. The study is double-blinded and placebo-controlled, ensuring unbiased evaluation of elafibranors effects. During the study, participants will undergo regular clinical assessments, laboratory tests, imaging, and patient-reported evaluations to monitor liver disease progression, symptoms like fatigue and itching, quality of life, and safety. The primary outcome measured is event-free survival from baseline up to five years. Participants may withdraw at any time, and the total duration for each participant may span several years including treatment and follow-up.

A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis