Phase 3 Primary Biliary Cholangitis Trial, Active, Not Recruiting NCT04526665 Sponsor: Ipsen Condition: Primary Biliary Cholangitis
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Trial results

AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

According to the results reported on ClinicalTrials.gov, this trial (NCT04526665) enrolled 161 people with primary biliary cholangitis (PBC), a liver condition. Of these, 108 were assigned to take elafibranor 80 mg daily and 53 received a placebo (a dummy tablet with no active ingredient). The trial ran for 52 weeks. It was measuring changes in certain liver-related blood markers — mainly a substance called alkaline phosphatase (ALP) and total bilirubin — to see how many participants reached pre-set target levels by the end of the year. Around 94 people in the elafibranor group and 46 in the placebo group completed the full study period. The reported data shows that for the main outcome — reaching a combined target for ALP and bilirubin blood levels at 52 weeks — about 50.9% of participants in the elafibranor group met that target, compared with 3.8% in the placebo group. For a stricter measure (ALP returning fully to the normal range), the reported figures were 14.8% in the elafibranor group and 0% in the placebo group. The trial also measured itching (a common symptom in PBC) in participants who had moderate-to-severe itch at the start, using an 11-point self-rated scale (0 = no itch, 10 = worst imaginable). The reported data shows average itch scores fell by 1.93 points in the elafibranor group and 1.15 points in the placebo group by week 52. In terms of ALP blood levels (measured in units per litre), the reported average reduction at 52 weeks was approximately 118 units/L in the elafibranor group compared with approximately 9 units/L in the placebo group. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.

These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.

Phase 3 Primary Biliary Cholangitis Trial, Active, Not Recruiting

NCT04526665
Active, Not Recruiting Phase 3

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • You are between 18 and 75 years old (inclusive)
  • You have been diagnosed with Primary Biliary Cholangitis (PBC), either confirmed or probable
  • Your blood test shows a liver enzyme called alkaline phosphatase (ALP) is at least 1.67 times above the normal upper limit
  • Your blood test shows your total bilirubin level is no more than 2 times the normal upper limit
  • You have been taking a medication called UDCA (ursodeoxycholic acid) for at least 12 months at a stable dose for 3 or more months before screening — or you have not been able to tolerate UDCA and have not taken it for at least 3 months before screening
  • You have completed a questionnaire about your worst itching symptoms before being assigned to a treatment group
  • If you are a woman who could become pregnant, you must be using highly effective contraception throughout the study and for 1 month after your last dose

Who may not be able to join:

  • You have, or have had, another liver disease alongside PBC
  • Your liver disease has reached a serious stage causing complications such as those related to cirrhosis or high blood pressure in the liver
  • You have a medical condition (other than liver disease) that raises your ALP levels, such as Paget's disease, or a condition (including certain cancers) that may limit your life expectancy to less than 2 years
  • You tested positive for HIV, or are known to be HIV positive
  • You have another significant medical condition that is unstable or untreated
  • You have a history of alcohol abuse
  • You are pregnant or breastfeeding (for women)
  • You have taken certain medications called fibrates or glitazones within the 2 months before screening
  • You have taken certain medications including obeticholic acid (OCA), azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide, other systemic steroids, or certain drugs that can harm the liver (such as α-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin) within 3 months before screening
  • You have used antibody treatments or immunotherapy targeting substances involved in inflammation (such as interleukins or other immune signalling proteins) within 12 months before screening
  • You have previously taken OCA and have not stopped taking it at least 3 months before screening
  • You are currently in, planning to join, or have recently taken part in another clinical drug or medical device study within 30 days (or five half-lives of the drug, whichever is longer) before screening; if you have previously taken a drug called seladelpar, you must have stopped it at least 3 months before screening
  • Your blood tests show certain liver enzymes (ALT and/or AST) are more than 5 times the normal upper limit
  • Your liver enzyme or bilirubin levels have varied by more than 40% between tests (confirm with trial site)
  • Your blood albumin level is below 3.0 g/dl
  • Your results meet a definition of severely advanced PBC based on both bilirubin being above normal and albumin being below normal (Rotterdam criteria)
  • Your blood clotting measure (INR) is above 1.3 due to reduced liver function
  • Your CPK (a muscle enzyme) level is more than 2 times the normal upper limit
  • Your blood creatinine level is above 1.5 mg/dl at screening
  • You have significant kidney disease, including conditions such as nephrotic syndrome or chronic kidney disease with reduced kidney function (eGFR below 60 mL/min/1.73 m²)
  • Your platelet count is below 150 x 10³ per microlitre
  • Your AFP (a liver tumour marker) is above 20 ng/mL and a liver scan suggests possible liver cancer
  • You have a known allergy or sensitivity to the study drug (elafibranor) or any of its ingredients
  • There are concerns about your ability to give informed consent or to follow the study requirements due to mental health or cognitive difficulties

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 28 July 2026
Phase 3: approximately ~65% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

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Trial details

Status
Active, Not Recruiting
Phase
Phase 3
Sponsor
Ipsen
Registry
ClinicalTrials.gov
Start date
24 September 2020
Est. completion
1 June 2023

Where this trial is recruiting

🇦🇷 Argentina 🇧🇪 Belgium 🇧🇷 Brazil 🇨🇦 Canada 🇨🇱 Chile 🇫🇷 France 🇩🇪 Germany 🇮🇹 Italy 🇿🇦 South Africa 🇪🇸 Spain 🇨🇭 Switzerland Turkey (Türkiye) 🇬🇧 United Kingdom 🇺🇸 United States

Primary endpoints

Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov