Active — Not RecruitingPhase 3

A Study of Elafibranor in Adults With Primary Biliary Cholangitis and Inadequate Response or Intolerance to Ursodeoxycholic Acid.

United States69 participantsStarted 2024-07-09

Plain-language summary

The participants in this study will have confirmed PBC with inadequate response or intolerance to Ursodeoxycholic acid (UDCA), which is a medication used in the management and treatment of cholestatic liver disease. Primary biliary cholangitis is a slowly progressive disease characterised by damage of the bile ducts in the liver, leading to a build-up of bile acids which causes further damage. The liver damage in PBC may lead to scarring (cirrhosis). PBC may also be associated with multiple symptoms. Many patients with PBC may require a liver transplant or may die if the disease progresses and a liver transplant is not done. This study will compare a daily dose of elafibranor (the study drug) to a daily dose of placebo (a dummy treatment). The main aim of this study is to determine if elafibranor is better than placebo in reducing ALP levels to a normal value. High ALP levels in the blood can indicate liver disease. There will be three periods in this study: A screening period (up to 8 weeks) to assess whether the participant can take part; a treatment period (up to 52 weeks) where eligible participants will be grouped as per their blood ALP levels and randomly assigned to either receive elafibranor or placebo, and a follow-up period (4 weeks) where participants' health will be monitored. Participants will be twice as likely to receive elafibranor than placebo (2:1 ratio). Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a noninvasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (a noninvasive test that passes a probe on skin to measure stiffness of the liver). They will also be asked to fill in questionnaires. Each participant will be in this study for up to 64 weeks (15 months).

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria * Male or female participants age ≥18 years of age. * Participants with a historical diagnosis of PBC as demonstrated by the presence of ≥2 of the following three historical diagnostic criteria: * i. History of elevated ALP levels for ≥6 months prior to the first screening visit (SV1). * ii. Positive Antimitochondrial antibody (AMA) titres (≥1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay) or positive PBC-specific antinuclear antibodies. * iii. Liver biopsy consistent with PBC. * ALP \>1 × ULN and \<1.67 × ULN. * Participants taking UDCA should have been on this medication for at least 6 months and at a stable dose for ≥3 months. Participants who are intolerant to UDCA should have taken the last dose of UDCA ≥3 months prior. * Participants taking medications for management of pruritus must be on a stable dose for ≥3 months. * Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies \* (a) Male participants must agree that, if their partner is at risk of becoming pregnant, they will use an effective method of contraception. The participant must agree to use contraception during the whole period of the study and for 30 days after the last dose of study intervention. * Capable of giving signed informed consent Exclusion Criteria * History or presence of other concomitant liver diseases. * Participants with known ci…

What they're measuring

Percentage of participants with normalisation of Alkaline Phosphate (ALP) Levels

Timeframe: At Week 52

Trial details

NCT IDNCT06383403

SponsorIpsen

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06-26