NCT04526665

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)

A Double-blind, Randomized, Placebo-Controlled Study and Open-label Long Term Extension to Evaluate the Efficacy and Safety of Elafibranor 80 mg in Patients With Primary Biliary Cholangitis With Inadequate Response or Intolerance to Ursodeoxycholic Acid

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 161 (actual)
• Sponsor: Ipsen · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid (which is a medication used in the management and treatment of cholestatic liver disease). PBC is a slowly progressive disease characterized by damage of the bile ducts in the liver, leading to a buildup of bile acids which causes further damage. The liver damage in PBC may lead to scarring (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. The main aim of this study is to determine if elafibranor (the study drug) is better than placebo (a dummy treatment) at decreasing the levels of a specific blood test (alkaline phosphatase) that provides information about participant's disease. This study will also evaluate the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itchy skin (pruritus) and tiredness (fatigue). This study has two main parts: Part 1 will compare a daily dose of elafibranor to a daily dose of placebo and will last between a minimum of one year and a maximum of two years. Part 2, all participants will receive elafibranor for a period of up to 5 years or until the total treatment duration (part 1 and part 2) reaches 6 years, whichever occurs first.

Conditions

• Primary Biliary Cirrhosis

Interventions

Type	Name	Description
DRUG	Elafibranor 80mg	Elafibranor 80mg daily
DRUG	Placebo	Placebo daily

Timeline

• Start date: 2020-09-24
• Primary completion: 2023-06-01
• Completion: 2028-12-01
• First posted: 2020-08-26
• Last updated: 2026-04-02
• Results posted: 2024-09-05

Locations

115 sites across 14 countries: United States, Argentina, Belgium, Brazil, Canada, Chile, France, Germany, Italy, South Africa, Spain, Switzerland, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study