Elafibranor for Primary Sclerosing Cholangitis

(ELASCOPE Trial)

This trial aims to evaluate the efficacy and safety of elafibranor for treating individuals with primary sclerosing cholangitis (PSC). PSC is a rare disease that causes inflammation and scarring in the liver's bile ducts, potentially leading to liver damage. Participants will receive either elafibranor or a placebo (a pill with no active medicine) to compare the effects. Those with PSC, stable liver function, and no other chronic liver diseases may be suitable candidates for this trial. The study will monitor liver health, symptoms, and overall safety through regular check-ups over several years. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment nearing widespread availability.

The trial requires stopping certain medications at least 3 months before starting, including norucholic acid, fibrates, seladelpar, glitazones, cyclosporine, mycophenolate, pentoxifylline, and some corticosteroids. Check with the study team to see if your current medications are on this list.

Previous studies found that elafibranor was well tolerated by people with primary sclerosing cholangitis (PSC). Specifically, research showed that it improved liver function more than a placebo over 12 weeks. Most participants experienced some side effects, similar to those in other groups studied. This suggests that while some side effects may occur, elafibranor generally has a favorable safety profile for people with PSC.¹²³⁴⁵

Elafibranor is unique because it targets a different pathway compared to current treatments for Primary Sclerosing Cholangitis (PSC). Most treatments for PSC focus on managing symptoms or complications, but Elafibranor is a dual agonist of the peroxisome proliferator-activated receptors alpha and delta (PPARα/δ), which are involved in reducing inflammation and fibrosis in the liver. This mechanism could offer a more direct approach to tackling the underlying disease process, which is why researchers are excited about its potential to provide a more effective treatment option for PSC.

Research has shown that elafibranor offers promising results for people with Primary Sclerosing Cholangitis (PSC). In this trial, participants will receive either elafibranor or a placebo. Previous studies found that elafibranor improved liver health more than a placebo over 12 weeks, including a reduction in alkaline phosphatase (ALP), an important indicator of liver damage. The treatment was well-tolerated, with side effects similar to those of the placebo. Overall, early findings suggest that elafibranor could effectively improve liver function in PSC patients.¹²³⁴⁶