Principal Investigator
Enrollment Status
Clinical Trial ID
Clinical Trial Summary
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). In
the DB period, patients will be randomized in a 2:1 ratio to receive Elafibranor 80 mg or
placebo, once daily.
Phase
Phase 3
Funding Agency/Sponsor
Industry
Disease
PBC (Primary Biliary Cholangitis)
Enrollment Eligibility
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