Elafibranor in Primary Biliary Cholangitis

What is the Purpose of this Study?

This study focuses on individuals who have been diagnosed with primary biliary cholangitis (PBC), a chronic liver disease resulting from progressive destruction of the liver bile ducts (intrahepatic bile ducts) and buildup of bile acids leading to inflammation and scarring of the liver (fibrosis). The purpose of the study is to evaluate the safety and effectiveness of an investigational drug called elafibranor in patients with PBC who have inadequate response to or cannot tolerate standard therapy for PBC. Researchers will compare the effects of elafibranor against the effects of placebo (inactive substance). Participants will be randomly assigned to one of two treatment groups. One group will receive 80 mg of elafibranor, and the other will receive placebo.

Eligibility

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

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)
Must have PBC Worst Itch NRS collected prior to randomization
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 complications of cirrhosis/portal hypertension
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 HIV Type 1 or 2 at screening, or patient is known to have tested positive for 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 OCA, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); 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
ALT and/or AST values > 5 x ULN
For patients with AT or TB>ULN at SV1, variability of AT or TB > 40% (see section 3.5.1)
Albumin<3.0 g/dl
Severely advanced patients according to Rotterdam criteria (TB > ULN and albumin
INR > 1.3 due to altered hepatic function
CPK > 2 x ULN
Screening serum creatinine > 1.5 mg/dl
Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as patients with markers of kidney failure damage or eGFR < 60 mL/min/1,73 m2) calculated by MDRD
Platelet count < 150 x 103/μL
AFP > 20 ng/mL with 4-phase liver CT or MRI imaging suggesting presence of liver cancer
Known hypersensitivity to the investigational product or to any of the formulation excipients of the elafibranor or placebo tablet Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain

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Where can I participate?

Beverly

More about this Clinical Trial

What is the full name of this clinical trial?

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

Study Details

Disease Type/Condition

Biliary Disease

Principal Investigator

Ayoub, Walid

Age Group

Adult

Phase

III

IRB Number

STUDY00000987

ClinicalTrials.gov ID

NCT04526665

Key Eligibility

ClinicalTrials.gov

How do I learn more about this study?

clinicaltrials@cshs.org