Phase 3 study for people with Primary Biliary Cholangitis

Reproduced exactly, unedited. Written for researchers rather than for patients.

Inclusion Criteria:

• Adults participants aged 18 years or older
• Confirmed diagnosis of primary sclerosing cholangitis based on standard clinical, biochemical, and imaging criteria
• Compensated liver disease at screening
• Stable background therapy, where applicable prior to study entry
• Women of childbearing potential have to apply during the entire duration of the study a highly effective method of birth control
• Ability to provide written informed consent and comply with study procedures.

Exclusion Criteria:

- History or presence of other concomitant chronic liver disease

- History of hepatic decompensation, including: i) History of liver transplantation, current MELD 3.0 score ≥12 due to hepatic impairment.

ii) Evidence of complications of cirrhosis

• Participants with cirrhosis who are also classified as Child-Pugh B or C based on the Child Pugh score.

• History of biliary intervention within 60 days prior to the screening period, and/or presence of percutaneous drain or bile duct stent at SV.

• History of bacterial cholangitis, and/or participant on antibiotics for prophylaxis of recurrent cholangitis within 60 days prior to the SV.

• History or any current suspicion of cholangiocarcinoma or hepatocellular carcinoma

• Known malignancy or history of malignancy within the last 5 years, with the exception of local, successfully treated basal cell carcinoma or in-situ carcinoma of the uterine cervix.

• Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).

• Administration of the following medications are prohibited as specified below:

  i) 3 months prior to baseline: norucholic acid, fibrates, seladelpar and glitazones.

ii) 3 months prior to baseline: cyclosporine, mycophenolate, pentoxifylline, and chronic systemic corticosteroids (except as part of management of IBD at an ongoing stable dose); potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin).

• Participants who are currently participating in, plan to participate in, or have participated in an investigational drug or medical device study containing active substance within 30 days or five half-lives, whichever is longer, prior to the SV. - Participants with previous exposure to elafibranor.
• Electrocardiogram (ECG) with QT interval corrected by Fridericia's formula (QTcF) >450 msec in males or QTcF >470 msec in females for participants without bundle branch block.
• Significant renal disease,
• For female participants: known pregnancy, or has a positive serum pregnancy test, or lactating.
• Regular alcohol intake in excess of the recommended limit of 2 standard drinks per day for men or 1 standard drink per day for women
• History of alcohol abuse, or other substance abuse within 1 year prior to SV.
• Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that contraindicates participation in the study.
• Mental instability or incompetence
• Participant has or is known to have tested positive for human immunodeficiency virus (HIV) type 1 or 2 at SV.
• Medical conditions that may diminish life expectancy to <2 years.