Elafibranor for adults with primary sclerosing cholangitis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

About 350 adults with large-duct primary sclerosing cholangitis and compensated liver disease will be enrolled and randomized to receive either elafibranor 120 mg once daily or a matching placebo. The protocol includes a screening period, a treatment period with routine clinical assessments, laboratory testing and imaging, and a post-treatment safety follow-up. Participants will complete symptom and quality-of-life questionnaires and undergo evaluations to track liver disease progression and safety. Key exclusions include other chronic liver diseases, prior liver transplant, current hepatic decompensation, recent biliary intervention, and Child-Pugh B/C cirrhosis.

Why it matters

Who can join: the full rules

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.

Where it's running

• Arizona Liver Health
  Chandler, Arizona, United States Recruiting
• Southern California Research Center
  Coronado, California, United States Recruiting
• California Liver Research Institute
  Pasadena, California, United States Recruiting
• University of California, San Francisco Medical Center
  San Francisco, California, United States Recruiting
• Peak Gastroenterology Associates
  Colorado Springs, Colorado, United States Recruiting
• South Denver Gastroenterology,P.C.
  Englewood, Colorado, United States Recruiting

+181 more sites. See ClinicalTrials.gov for the full list.

Study contacts

• Study coordinator: Ipsen Clinical Study Enquiries
• Email: clinical.trials@ipsen.com
• Phone: see email

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.