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

Purpose

The main objective of the study is to evaluate the effect of daily oral administration of 80mg elafibranor on cholestasis (impairment of bile formation and/or bile accumulation) in patients with PBC and inadequate response or intolerance to Ursodeoxycholic Acid (UCDA)

Condition

  • Primary Biliary Cirrhosis

Eligibility

Eligible Ages
Between 18 Years and 75 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Males or females age of 18 to 75 years (inclusive) - Definite or probable PBC diagnosis - ALP ≥ 1.67x upper limit of normal (ULN) - Total bilirubin (TB) ≤ 2x ULN - UDCA for at least 12 months (stable dose ≥ 3 months) prior to screening, or unable to tolerate UDCA treatment (no UDCA for ≥ 3 months) prior to screening (per country standard-of-care dosing) - Females participating in this study must be of non-child bearing potential or must be using highly efficient contraception for the full duration of the study and for 1 month after the last drug intake

Exclusion Criteria

  • History or presence of other concomitant liver disease - Clinically significant hepatic decompensation, including patients with cirrhosis/portal hypertension complications - Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease) or which may diminish life expectancy to < 2 years, including known cancers - Patient has a positive test for Human Immunodeficiency Virus (HIV) - Evidence of any other unstable or untreated clinically significant disease - History of alcohol abuse - For female patients: known pregnancy or lactating - Use of fibrates and glitazones within 2 months prior to screening - Use of azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid isoniazid, or nitrofurantoin) within 3 months prior to screening - Use of antibodies or immunotherapy directed against interleukins (ILs) or other cytokines or chemokines within 12 months prior to screening - For patients with previous exposure to obeticholic acid (OCA), OCA should be discontinued 3 months prior to screening - Patients who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or five half-lives, whichever is longer, prior to screening; for patients with previous exposure to seladelpar, seladelpar should be discontinued 3 months prior to screening. - Patients with previous exposure to elafibranor - Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 5x ULN - Albumin < 3.0 g/dL - PBC Severely advanced patients according to Rotterdam criteria (TB > ULN and albumin < LLN) - International normalized ratio (INR) > 1.3 due to altered hepatic function - Creatine phosphokinase CPK > 2X ULN - Serum creatinine > 1.5 mg/dL - Significant renal disease

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Intervention Model Description
This is a double-blind (DB), randomized, placebo-controlled study followed by an open-label long term extension (LTE)
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Elafibranor 80mg
Study subjects will take 1 tablet per day orally before breakfast with a glass of water each morning
  • Drug: Elafibranor 80mg
    Elafibranor 80mg for double blind period (52 to 104 weeks). After completion of the double blind period, participants will be offered the opportunity to enter an open label long term extension study for up to 5 years. Participants will continue the elafibranor dose received during the double blind period.
Placebo Comparator
Placebo
Study subjects will take 1 tablet per day orally before breakfast with a glass of water each morning
  • Drug: Placebo
    Placebo for double blind period (52 to 104 weeks). After completion of the double blind period, participants will be offered the opportunity to enter an open label long term extension study and to switch to elafibranor 80mg for up to 5 years.

Recruiting Locations

Vanderbilt Digestive Disease Center
Nashville, Tennessee 37232

More Details

Status
Recruiting
Sponsor
Genfit

Study Contact

Genfit
+33(0)320164000
clinicaltrial@genfit.com