A Study of Elafibranor in Adult Japanese Participants With Primary Biliary Cholangitis (PBC)

NCT06730061 | Active Not Recruiting Phase 3

• 1 other identifier: CLIN-60190-462
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 16

Brief Summary

The purpose of this study is to find out about the safety and how well the study intervention (elafibranor) works in participants with PBC. The participants in this study will have confirmed PBC with inadequate response or intolerance to UDCA, which is a medication used in the management and treatment of cholestatic liver disease. PBC 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. In this study all participants will receive a daily dose of elafibranor (the study intervention). The main aim of this study is to determine if elafibranor reduces alkaline phosphatase (ALP) and total bilirubin levels. High ALP and bilirubin levels in the blood can indicate liver disease. There will be 4 periods in this study: A screening period (up to 10 weeks) to assess whether the participant can take part. A treatment period (52 weeks) where all eligible participants will receive elafibranor. A variable treatment extension period (2-5 years) from End Of Treatment (EOT) period up to the commercial availability of elafibranor in Japan. A follow-up period (4 weeks) where participants' health will be monitored. Participants will undergo blood sampling, urine collections, physical examinations, clinical evaluations, electrocardiograms (ECG: recording of the electrical activity of heart), ultrasound examinations (a non-invasive test that passes a probe over skin to look at the bladder, urinary tract, and liver), and Fibroscan® examinations (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 approximately 6 years

Conditions

Primary Biliary Cholangitis

Outcome Measures

Primary Outcomes (1)

• Percentage of participants with Alkaline phosphatase (ALP) <1.67x ULN, ALP decrease ≥15% and Total Bilirubin (TB) ≤ ULN

  At Week 52

Secondary Outcomes (43)

• Percentage of Participants who normalised ALP Levels

  At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

• Change from baseline in ALP Levels

  At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

• Percentage of Participants With ALP Level response

  At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

• Percentage of participants with ALP <1.5×ULN, ALP decrease ≥40% and TB ≤ULN.

  At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

• Percentage of participants with ALP <3×ULN, aspartate aminotransferase (AST) <2×ULN and Total Bilirubin (TB) <1 mg/dL (Paris I criteria).

  At Week 4, Week 13, Week 26, Week 39, Week 52, Week 78 and every 26 weeks until the end of study (up to 6 years)

Study Arms (1)

Elafibranor

EXPERIMENTAL

Participants will take the study intervention by mouth (swallow whole) once daily

Drug: Elafibranor

Interventions

Elafibranor DRUG

Tablet of 80 mg

Elafibranor

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must have provided written informed consent and agree to comply with the study protocol.
• Japanese male or female participants aged 18 to 75 years inclusive at Screening Visit 1 (SV1).
• PBC diagnosis as described in the study protocol
• ALP ≥1.67×ULN (mean value based on samples collected at SV1 and SV2).
• TB ≤2×ULN at SV1 and SV2.
• Must have at least 4 available values for PBC Worst Itch Numeric Rating Scale (NRS) during each of the 7-day intervals in the 14 days prior to visit (V)1, for a total of at least 8 values for PBC Worst Itch NRS in the last 14 days prior to V1.
• Participants taking 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).
• If on colchicine, must be on a stable dose for ≥3 months prior to screening.
• Medications for management of pruritus (for example, cholestyramine, rifampicin, naltrexone, sertraline or nalfurafine hydrochloride) must be on a stable dose for ≥3 months prior to screening.
• Participants taking statins or ezetimibe must be on a stable dose for ≥2 months prior to screening.
• Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies

You may not qualify if:

• History or presence of other concomitant liver disease
• Participants with known cirrhosis who have a Child-Pugh B or C classification.
• Participants with cirrhosis with Child-Pugh A classification are allowed.
• History or presence of clinically significant hepatic decompensation,
• Medical conditions that may cause non-hepatic increases in ALP (for example, Paget's disease) or which may diminish life expectancy to \<2 years, including known cancers.
• 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.
• Participant has a positive test for human immunodeficiency virus (HIV) Type 1 or 2 at screening, or participant is known to have tested positive for HIV.
• Evidence of any other unstable or untreated clinically significant immunological, endocrine, haematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant medical conditions that are not well controlled.
• History of alcohol abuse, defined as consumption of more than 30 g pure alcohol per day for men, and more than 20 g pure alcohol per day for women, or other substance abuse within 1 year prior to SV1.
• For female participants: known pregnancy, or has a positive serum pregnancy test, or breastfeeding.
• Administration of the following medications are prohibited as specified below:
• month prior to screening: fibrates.
• months prior to screening: glitazones.
• For participants with previous exposure to obeticholic acid (OCA), OCA should be discontinued 3 months prior to screening.
• months prior to screening: azathioprine, cyclosporine (systemic), methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including α-methyldopa, sodium valproate/valproic acid isoniazid, or nitrofurantoin).

Sponsors & Collaborators

• Ipsen: lead

Study Sites (16)

Nippon Medical School - Chiba Hokusoh Hospital

Chiba, Japan

Location

Fukushima Medical University Hospital

Fukushima, Japan

Location

Chugoku Rosai Hospital

Hiroshima, Japan

Location

Teine Keijinkai Hospital

Hokkaido, Japan

Location

Kagawa University Hospital

Kagawa, Japan

Location

Kagoshima University Hospital

Kagoshima, Japan

Location

Shinshu University Hospital

Nagano, Japan

Location

National Hospital Organization Nagasaki Medical Center

Nagasaki, Japan

Location

Nara Medical University Hospital

Nara, Japan

Location

Nagaoka Red Cross Hospital

Niigata, Japan

Location

Niigata University Medical & Dental Hospital

Niigata, Japan

Location

National Hospital Organization Osaka National Hospital

Osaka, Japan

Location

Hamamatsu University Hospital

Shizuoka, Japan

Location

Juntendo University Hospital

Tokyo, Japan

Location

Teikyo University Hospital

Tokyo, Japan

Location

Tokyo Metropolitan Komagome Hospital

Tokyo, Japan

Location

MeSH Terms

Conditions

Liver Cirrhosis, Biliary

Interventions

2-(2,6-dimethyl-4-(3-(4-(methylthio)phenyl)-3-oxo-1-propenyl)phenoxyl)-2-methylpropanoic acid

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Liver Cirrhosis; Fibrosis; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Ipsen Medical, Director

  Ipsen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 3, 2024
• First Posted: December 12, 2024
• Study Start: January 16, 2025
• Primary Completion (Estimated): December 17, 2026
• Study Completion (Estimated): April 8, 2032
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Time Frame: Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
• Access Criteria: Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

Locations

JP (16)