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Elafibranor 在原发性胆汁性胆管炎 (PBC) 患者中的研究 (ELATIVE)

2026年10月20日 更新者：Ipsen

一项双盲、随机、安慰剂对照研究和开放标签长期扩展，以评估 Elafibranor 80 mg 在对熊去氧胆酸反应不足或不耐受的原发性胆汁性胆管炎患者中的疗效和安全性

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC) with inadequate response or intolerance to ursodeoxycholic acid (which is a medication used in the management and treatment of cholestatic liver disease).

PBC is a slowly progressive disease characterized by damage of the bile ducts in the liver, leading to a buildup 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 liver transplant or may die if the disease progresses and a liver transplant is not done.

The main aim of this study is to determine if elafibranor (the study drug) is better than placebo (a dummy treatment) at decreasing the levels of a specific blood test (alkaline phosphatase) that provides information about participant's disease. This study will also evaluate the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itchy skin (pruritus) and tiredness (fatigue).

This study has two main parts:

Part 1 will compare a daily dose of elafibranor to a daily dose of placebo and will last between a minimum of one year and a maximum of two years.

Part 2, all participants will receive elafibranor for a period of up to 5 years or until the total treatment duration (part 1 and part 2) reaches 6 years, whichever occurs first.

研究概览

地位

主动，不招人

条件

干预/治疗

研究类型

介入性

注册 (实际的)

161

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 加拿大
      • Montreal、加拿大、H2X 0A9
        • Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
    • Alberta
      • Calgary、Alberta、加拿大、T2N 4Z6
        • University of Calgary Liver Unit - Heritage Medical Research Clinic (HMRC)
    • British Columbia
      • Vancouver、British Columbia、加拿大、V5Z IM9
        • Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology
    • Manitoba
      • Winnipeg、Manitoba、加拿大、R3E 3P4
        • Shared Health Inc.-Operating as Health Sciences Centre-John Buhler Research Centre
  • 南非
      • Cape Town、南非、7800
        • Mediclinic Constantiaberg, North Suites
    • Western Cap
      • Cape Town、Western Cap、南非、7925
        • Groote Schuur Hospital, University of Cape Town - Clinical Research Centre
    • Western Cape
      • Cape Town、Western Cape、南非、7530
        • Tiervlei Trial Centre
  • 土耳其（türkiye）
      • Ankara、土耳其（türkiye）、06230
        • Hacette Universitesi Hastenesi IC, Hastaliklari Anabilim Dali, Gastronenteroloji Bilim Dali, Mithapasa Cad. Hacettepe Mah.
    • Bornova
      • Izmir、Bornova、土耳其（türkiye）、35100
        • Ege Universitesi Tip Fakultesi Hastanesi
    • Pendik
      • Istanbul、Pendik、土耳其（türkiye）、34890
        • Marmara Universitesi Pendik Egitim ve Arastirma Hastanesi, Gastroentoloji Bilim Dali
  • 巴西
    • Rio Grande do Sul
      • Porto Alegre、Rio Grande do Sul、巴西、90035-903
        • Hospital de Clínicas de Porto Alegre
    • São Paulo
      • São Paulo、São Paulo、巴西、05403-000
        • Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo-HCFMUSP
  • 德国
      • Berlin、德国、10825
        • Gastro - Sudien
      • Berlin、德国、13353
        • Charite - Universitätsmedizin Berlin- CVK - Medizinische Klinik
      • Frankfurt、德国、60590
        • Universitätsklinikum Frankfurt
      • Hanover、德国、30625
        • Medizinische Hochschule Hannover
      • Kiel、德国、24146
        • Center for Gastroenterology and Hepatology (GHZ) Kiel
      • Leipzig、德国、04103
        • EUGASTRO GmbH
      • Mainz、德国、55131
        • Universitätsmedizin der Johannes Gutenberg-Universität Mainz
  • 意大利
      • Bologna、意大利、40138
        • Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
      • Milan、意大利、20142
        • Azienda Ospedaliera San Paolo, Dipartimento di Scienza della Salute, UO Medicina VI e Pathologia e Gastroenterologia
      • Monza、意大利、20900
        • ASST Monza - Ospedale San Gerardo, Gastroenterologia
      • Padova、意大利、35128
        • Azienda Ospedale- Università degli Studi di Padova UOC Gastroenterologia
      • Palermo、意大利、90127
        • Azienda Ospedaliera Universitaria Policlinico P. Giaccone, UOC di Gastroenterologia-Dip.Medicina Interna e Specialistica
    • Località Baggiovara
      • Modena、Località Baggiovara、意大利、41126
        • Ospedale Civile di Baggiovara-Struttura Complessa di Medicina ad indririzzo Metabolico Nutrizionale
  • 智利
      • Santiago、智利、7550000
        • Clínica Universidad de los Andes
    • Coquimbo Region
      • La Serena、Coquimbo Region、智利、1700000
        • Hospital de La Serena
    • Santiago Metropolitan
      • Santiago、Santiago Metropolitan、智利、8330034
        • Centro de Investigacion Clinica Universidad Catolica CICUC
      • Santiago、Santiago Metropolitan、智利、8320000
        • Hospital Clínico Universidad de Chile
    • Valparaiso
      • Viña del Mar、Valparaiso、智利、2540364
        • Centro de Investigaciones Clinicas (CIC)
  • 比利时
      • Brussels、比利时、1070
        • Hôpital Erasme
      • Edegem、比利时、2650
        • Antwerp University Hospital
      • Ghent、比利时、9000
        • Universitair Ziekenhuis Gent
      • Leuven、比利时、3000
        • University Hospital Leuven
  • 法国
      • Amiens、法国、80054
        • CHU Amiens Picardie
      • Créteil、法国、94000
        • Hôpital Henri Mondor
      • Grenoble、法国、38043
        • CHU Grenoble Alpes - Hôpital Albert Michallon
      • Orléans、法国、45067
        • Centre Hospitalier Régional d'Orléans
      • Paris、法国、75012
        • Hôpital Saint-Antoine
      • Paris、法国、75014
        • Hôpital Cochin
      • Paris、法国、75651
        • Hôpital Pitié-Salpétrière
      • Pessac、法国、33604
        • Hôpital Haut Lévêque
      • Reims、法国、51092
        • Hôpital Robert Debré - CHU de Reims
      • Strasbourg、法国、67200
        • Hôpitaux Universitaires de Strasbourg - Hôpital de Hautepierre
  • 瑞士
      • Bern、瑞士、3010
        • Inselspital, Universitätsspital Bern Universitätsklinik für Viszerale chirurgie und Medizin Hepatologie
      • Lugano、瑞士、6900
        • Fondazione Epatocentro Ticino
  • 美国
    • Arizona
      • Chandler、Arizona、美国、85224
        • The Institute for Liver Health
    • California
      • Los Angeles、California、美国、90048
        • Cedars-Sinai Medical Center
      • Los Angeles、California、美国、90036
        • Ruane Clinical Research Group Inc.
      • Los Angeles、California、美国、90033
        • Keck Medical Center of USC
      • Sacramento、California、美国、95817
        • University of California, Davis Medical Center
      • San Francisco、California、美国、94109
        • California Pacific Medical Center - Sutter Pacific Medical Foundation
    • Colorado
      • Aurora、Colorado、美国、840045
        • University of Colorado Denver and Hospital
      • Englewood、Colorado、美国、80113
        • South Denver Gastroenterology, P.C.
    • Connecticut
      • New Haven、Connecticut、美国、06510
        • Yale School of Medicine, Digestive Diseases
    • Florida
      • Jacksonville、Florida、美国、32256
        • Encore Borland-Groover Clinical Research
      • Miami、Florida、美国、33136
        • Schiff Center for Liver Diseases/University of Miami
    • Georgia
      • Atlanta、Georgia、美国、30309
        • Digestive Healthcare of Georgia
    • Kansas
      • Kansas City、Kansas、美国、66160
        • University of Kansas Medical Center
    • Massachusetts
      • Boston、Massachusetts、美国、02114
        • Massachusetts General Hospital
      • Boston、Massachusetts、美国、02215
        • Beth Israel Deaconess Medical Center (BIDMC)
    • Michigan
      • Novi、Michigan、美国、48377
        • Henry Ford Health System
    • Missouri
      • St Louis、Missouri、美国、63104
        • Saint Louis University
    • New York
      • New Hyde Park、New York、美国、11042-11114
        • UPMC Center for Liver Diseases
      • New York、New York、美国、10016
        • NYU Langone Health / NYU Grossman School of Medicine
      • New York、New York、美国、10032
        • Columbia University Medical Center - Center for Liver Disease and Transplantation
      • New York、New York、美国、10065
        • The New York-Presbyterian Hospital, David H. Koch Center
    • North Carolina
      • Durham、North Carolina、美国、27710
        • Duke University Medical Center
      • Huntersville、North Carolina、美国、28078
        • Carolinas Centre for Liver disease/ Atrium Health
    • Ohio
      • Cincinnati、Ohio、美国、45249
        • Consultants for Clinical Research
      • Cleveland、Ohio、美国、44106
        • University Hospitals Cleveland Medical Center
      • Columbus、Ohio、美国、43210-1240
        • The Ohio State University Wexner Medical Center
    • Pennsylvania
      • Hershey、Pennsylvania、美国、17033
        • Investigational Drug Service Pharmacy Penn State Milton S. Hershey Medical Center
      • Philadelphia、Pennsylvania、美国、19107
        • Thomas Jefferson University
    • South Carolina
      • Charleston、South Carolina、美国、29425
        • Medical University of South Carolina- College of Medicine
    • Tennessee
      • Nashville、Tennessee、美国、37232
        • Vanderbilt Digestive Disease Center
    • Texas
      • Arlington、Texas、美国、76012
        • Texas Clinical Research Institute, LLC
      • Dallas、Texas、美国、75203
        • The Liver Institute at Methodist Dallas Medical Center
      • Dallas、Texas、美国、75234
        • Liver Center of Texas, PLLC
      • Dallas、Texas、美国、75390-8575
        • The University of Texas Southwestern Medical Center-IDS Aston Pharmacy
      • Fort Worth、Texas、美国、76104
        • Texas Digestive Disease Consultants dba GI Alliance
      • Houston、Texas、美国、77030
        • Liver Associates of Texas, P.A.
      • Houston、Texas、美国、77030
        • St. Luke's Health-Baylor St Luke's Medical center - Advanced Liver Therapies Research
      • San Antonio、Texas、美国、78215
        • American Research Corporation
      • Victoria、Texas、美国、77904
        • Gastro health & Nutrition-Victoria
    • Utah
      • Murray、Utah、美国、84107
        • Intermountain Medical Center - Transplant Services
      • Salt Lake City、Utah、美国、84132
        • University of Utah Hospital-Division of Gastroenterology, Hepatology, and Nutrition
    • Virginia
      • Charlottesville、Virginia、美国、22908
        • University of Virginia Medical Center
      • Newport News、Virginia、美国、23602
        • Maryview Hospital Inc, Bon Secours Liver Institute of Hampton Roads
      • Richmond、Virginia、美国、23298
        • Virginia Commonwealth University Clinical Research Services Unit (CRSU)
      • Richmond、Virginia、美国、23226
        • Richmond Community Hospital LLC, Bon Secours Liver Institute of Richmond
    • Washington
      • Seattle、Washington、美国、98105
        • Liver Institute Northwest
  • 英国
      • Belfast、英国、BT12 6BA
        • Belfast Health and Social Care Trust-Royal Victoria Hospital
      • Camberley、英国、GU16 7UJ
        • Frimley Health NHS Foundation Trust - Frimley Park Hospital
      • Cambridge、英国、CB2 0QQ
        • Cambridge University Hospitals NHS Foundation Trust - Addenbrookes Hospital
      • Hull、英国、HU32J
        • Hull University Teaching Hospitals NHS Trust , Hull Royal Infirmary
      • London、英国、SE5 9RS
        • King's College Hospital. King's College NHS Foundation Trust
      • Nottingham、英国、NG72UH
        • Nottingham University Hospitals NHS Trust - Queen's Medical Centre (QMC)
      • Plymouth、英国、PL6 8DH
        • Plymouth Hospitals NHS Trust, Derriford Hospital
    • Newcastle
      • Newcastle upon Tyne、Newcastle、英国、NE7 7DN
        • The Newcastle upon Tyne Hospitals NHS Foundation Trust-Freeman Hospital
  • 西班牙
      • Barcelona、西班牙、08036
        • Hospital Clinic de Barcelona
      • Barcelona、西班牙、08035
        • Hospital Universitario Vall d'Hebron
      • Madrid、西班牙、28046
        • Hospital Universitario La Paz
      • Madrid、西班牙、28007
        • Hospital General Universitario Gregorio Marañón
      • Madrid、西班牙、C.P 28222
        • Hospital Universitario Puerta de Hierro -Madrid
      • Pontevedra、西班牙、36071
        • Complexo Hospitalario Universitario de Pontevedra
      • San Sebastián、西班牙、20014
        • Hospital Universitario Donostia
      • Santander、西班牙、39010
        • Hospital Universitario Marqués de Valdecilla
      • Seville、西班牙、41013
        • Hospital Universitario Virgen del Rocio
  • 阿根廷
    • Buenos Aires
      • CABA、Buenos Aires、阿根廷、1199
        • Hospital Italiano de Buenos Aires
      • CABA、Buenos Aires、阿根廷、C1280AEB
        • Hospital Británico de Buenos Aires
      • Caba、Buenos Aires、阿根廷、C1118AAT
        • Hospital Alemán
      • La Plata、Buenos Aires、阿根廷、1900
        • Hospital Italiano de La Plata
    • Mendoza Province
      • Godoy Cruz、Mendoza Province、阿根廷、5547
        • Hospital Espanol De Mendoza
    • Santa Fe Province
      • Rosario、Santa Fe Province、阿根廷、S2002KDS
        • Hospital Provincial del Centenario

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

18年 至 75年 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Inclusion Criteria

  • Males or females age of 18 to 75 years (inclusive)
  • Definite or probable Primary biliary cholangitis (PBC) diagnosis
  • Alkaline phosphatase (ALP) ≥ 1.67x upper limit of normal (ULN)
  • Total bilirubin (TB) ≤ 2x ULN
  • Ursodeoxycholic acid (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)
  • Must have PBC Worst Itch Numeric rating scale (NRS) collected prior to randomization
  • Females participating in this study must be of non-child bearing potential or must be using highly efficient contraception for the full duration of the study and for 1 month after the last drug intake

Exclusion Criteria:

  • History or presence of other concomitant liver disease
  • Clinically significant hepatic decompensation, including patients with complications of cirrhosis/portal hypertension
  • Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease) or which may diminish life expectancy to < 2 years, including known cancers
  • Patient has a positive test for HIV Type 1 or 2 at screening, or patient is known to have tested positive for HIV
  • Evidence of any other unstable or untreated clinically significant disease
  • History of alcohol abuse
  • For female patients: known pregnancy or lactating
  • Use of fibrates and glitazones within 2 months prior to screening
  • Use of Obeticholic acid (OCA), azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs
  • (including α-methyl-dopa, sodium valproic acid isoniazid, or nitrofurantoin) within 3 months prior to screening
  • Use of antibodies or immunotherapy directed against interleukins (ILs) or other cytokines or chemokines within 12 months prior to screening
  • For patients with previous exposure to OCA, OCA should be discontinued 3 months prior to screening
  • Patients who are currently participating in, plan to participate in, or have participated in an investigational drug study or medical device study containing active substance within 30 days or five half-lives, whichever is longer, prior to screening; for patients with previous exposure to seladelpar, seladelpar should be discontinued 3 months prior to screening
  • Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) values > 5 x ULN
  • For patients with AT or TB>ULN at SV1, variability of AT or TB > 40% (see section 3.5.1)
  • Albumin<3.0 g/dl
  • Severely advanced patients according to Rotterdam criteria (TB > ULN and albumin <LLN)
  • INR > 1.3 due to altered hepatic function
  • CPK > 2 x ULN
  • Screening serum creatinine > 1.5 mg/dl
  • Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as patients with markers of kidney failure damage or eGFR < 60 mL/min/1.73 m^2) calculated by Modification of diet in renal disease (MDRD)
  • Platelet count < 150 x 10^3/μL
  • AFP > 20 ng/mL with 4-phase liver CT or MRI imaging suggesting presence of liver cancer
  • Known hypersensitivity to the investigational product or to any of the formulation excipients of the elafibranor or placebo tablet Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：四人间

武器和干预

参与者组/臂
干预/治疗
实验性的：Elafibranor 80mg double-blind

Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
药品：Elafibranor 80mg

Elafibranor 80mg daily

其他名称：
  • Iqirvo®
安慰剂比较：Placebo

Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
药品：Placebo

Placebo daily
实验性的：Elafibranor 80mg open label

Study participants will take 1 tablet per day orally before breakfast with a glass of water each morning
药品：Elafibranor 80mg

Elafibranor 80mg daily

其他名称：
  • Iqirvo®

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52
大体时间：At Week 52

Cholestasis response was defined as alkaline phosphatase (ALP) < 1.67 x upper limit of normal (ULN) and total bilirubin (TB) <= ULN and ALP decrease from baseline >= 15% at Week 52 and based on the composite strategy imputing non-response for participants who experienced intercurrent events (ICEs) (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52

次要结果测量

结果测量
措施说明
大体时间
Key Secondary Endpoint: Percentage of Participants With Response to Treatment Based on ALP Normalization at Week 52
大体时间：At Week 52

Response to treatment based on normalization of ALP at Week 52 was defined as percentage of participants with ALP =<1.0× ULN and based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch Numeric Rating Scale (NRS) Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 52
大体时间：Baseline (up to 14 days pre-dose) and Week 52

The PBC Worst Itch NRS is a simple, self-administered patient reported outcome (PRO) questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake).
Baseline (up to 14 days pre-dose) and Week 52
Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch NRS Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 24
大体时间：Baseline (up to 14 days pre-dose) and Week 24

The PBC Worst Itch NRS is a simple, self-administered PRO questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake).
Baseline (up to 14 days pre-dose) and Week 24
Change From Baseline in ALP at Weeks 4, 13, 26, 39 and 52
大体时间：Baseline (Day 1) and at Weeks 4, 13, 26, 39 and 52

Blood samples were collected at specified timepoints for assessment of ALP. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Weeks 4, 13, 26, 39 and 52
Percentage of Participants With ALP Response From Baseline at Week 52
大体时间：Baseline (Day 1) and at Week 52

ALP response was defined as ALP decrease from baseline >= 10%; ALP decrease from baseline >= 20% and ALP decrease from baseline >= 40% at Week 52 and based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Percentage of Participants With Response to Treatment According to ALP < 1.5x ULN, ALP Decrease From Baseline >= 40% and TB =<ULN at Week 52
大体时间：At Week 52

Response to treatment was according to ALP < 1.5x ULN, ALP decrease from baseline >= 40% and TB =<ULN at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
At Week 52
Percentage of Participants With Response to Treatment According to ALP < 3x ULN, Aspartate Aminotransferase (AST) < 2x ULN and TB =< 1 Milligrams Per Deciliter (mg/dL) (Paris I) at Week 52
大体时间：At Week 52

Response to treatment was according to ALP < 3x ULN, AST < 2x ULN and TB =< 1 mg/dL (Paris I) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to ALP =< 1.5x ULN, AST =< 1.5x ULN and TB =< 1 mg/dL (Paris II) at Week 52
大体时间：At Week 52

Response to treatment was according to ALP =< 1.5x ULN, AST =< 1.5x ULN and TB =< 1 mg/dL (Paris II) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to TB Decrease of 15% Change From Baseline at Week 52
大体时间：At Week 52

Response to treatment was according to TB decrease of 15% change from baseline at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
At Week 52
Percentage of Participants With Response to Treatment According to Normalization of TB (TB =< ULN) and/or Albumin (ALB >= Lower Limit of Normal [LLN]) (Rotterdam) at Week 52
大体时间：At Week 52

Response to treatment was according to TB (TB =< ULN) and/or albumin (ALB >= LLN) (Rotterdam) at week 52. Participant was considered as responder at Week 52 only if total bilirubin and albumin were normal. The endpoint was analyzed in participants with abnormal total bilirubin (TB > ULN) or albumin (ALB < LLN) at baseline. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to TB ≤0.6 x ULN at Week 52
大体时间：At Week 52

Response to treatment was according to TB ≤0.6 x ULN at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to ALP ≤1.67 x ULN and TB ≤1 mg/dL (Momah/ Lindor) at Week 52
大体时间：At Week 52

Response to treatment was according to ALP ≤1.67 x ULN and TB ≤1 mg/dL (Momah/ Lindor) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to no Worsening of TB at Week 52
大体时间：At Week 52

Response to treatment was according to no worsening of TB, which was defined as level of TB <ULN or no increase from baseline of more than 0.1 x ULN at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
Percentage of Participants With Response to Treatment According to Complete Biochemical Response at Week 52
大体时间：At Week 52

Response to treatment according to complete biochemical response which was defined as <= ULN values of ALP, TB, AST, ALT and ALB, and international normalized ratio (INR) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52.
At Week 52
PBC 5-, 10- and 15-year Risk Scores Based on United Kingdom (UK)-PBC Score at Week 52
大体时间：At Week 52

The UK-PBC risk score was defined by the mean percentage risk that a PBC participant treated with UDCA would develop liver failure requiring liver transplantation in 5, 10, and 15 years from diagnosis. It is calculated from the following equation at week 52 as follows: UK-PBC score = 0.0287854*(alpEPxULN-1.722136304) - 0.0422873*([{altastEP x ULN/10}^-1] - 8.675729006) + 1.4199 * (LN(bilEPxULN /10)+2.709607778) - 1.960303*(alb x LLN -1.17673001)-0.4161954*(plt x LLN -1.873564875). The survival S(t) for any given participants was then calculated by S(t) = S0(t) exp(UK-PBC score). Here, alpEP x ULN=ALP /Upper Level Normal ALP at the timepoint, altastEPxULN=(ALT, AST or TA) /upper level normal of the value at the timepoint, bilEP x ULN=bilirubin /upper level normal bilirubin at the timepoint, alb x LLN=alb /alb lower level normal at baseline and plt x LLN=plt/plt lower level normal at baseline. Lower UK-PBC score predicts lower risk and better prognosis.
At Week 52
Global PBC Study Group (GLOBE) Score at Week 52
大体时间：At Week 52

The GLOBE score was a validated risk assessment tool providing an estimate of transplant-free survival for participants with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 participants with PBC. It is calculated from the following equation at week 52 as follows: GLOBE score = 0.044378 * age at start of ursodeoxycholic acid (UDCA) therapy + 0.93982 * LN (TB x ULN) + 0.335648 * LN (ALP x ULN) - 2.266708 * ALB x LLN - 0.002581 * platelet count per 10^9/L) + 1.216865; where TB x ULN = bilirubin/ULN at the timepoint, ALP x ULN = ALP/ULN at the timepoint, ALB x LLN = ALB/LLN at the timepoint, platelet count per 10^9/L = platelet count per 10^9/L at the timepoint. The Lower GLOBE PBC score predicts lower risk and better prognosis.
At Week 52
Change From Baseline in Hepatobiliary Injury and Liver Function as Assessed by AST, ALT, Gamma-glutamyl Transferase (GGT), 5 Prime Nucleotidase (5'-NT), and Fractionated ALP (Hepatic) (H1 and H2) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Hepatobiliary injury and liver function were assessed as measured by AST, ALT, GGT, 5'-NT, and fractionated ALP (hepatic) (H1 and H2). Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Hepatobiliary Injury and Liver Function as Measured by Total and Conjugated Bilirubin at Week 52
大体时间：Baseline (Day 1) and at Week 52

Hepatobiliary injury and liver function were assessed as measured by total and conjugated bilirubin. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Hepatobiliary Injury and Liver Function as Measured by Albumin at Week 52
大体时间：Baseline (Day 1) and at Week 52

Hepatobiliary injury and liver function were assessed as measured by albumin. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Hepatobiliary Injury and Liver Function as Measured by INR at Week 52
大体时间：Baseline (Day 1) and at Week 52

Hepatobiliary injury and liver function were assessed as measured by INR. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers of Inflammation as Measured by High-sensitivity C-reactive Protein (hsCRP) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers of inflammation were assessed as measured by hsCRP. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers of Inflammation as Measured by Fibrinogen and Haptoglobin at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers of inflammation were assessed as measured by fibrinogen and haptoglobin. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers of Inflammation as Measured by Tumor Necrosis Factor Alpha at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers of inflammation were assessed as measured by TNF-alpha. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Immune Response as Measured by Immunoglobulin (Ig)G and IgM at Week 52
大体时间：Baseline (Day 1) and at Week 52

Immune response was assessed as measured by IgG and IgM. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers and Non-invasive Measures of Hepatic Fibrosis as Measured by Enhanced Liver Fibrosis (ELF) and Plasminogen Activator Inhibitor-1 (PAI-1) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers and non-invasive measures of hepatic fibrosis were assessed as measured by ELF: hyaluronic acid [HA], type 2 procollagen peptide [PIINP], tissue inhibitor of metalloproteinases-1[TIMP-1]) and PAI-1. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers and Non-invasive Measures of Hepatic Fibrosis as Measured by Cytokeratin-18 (CK-18) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers and non-invasive measures of hepatic fibrosis were assessed as measured by CK-18 (M30 and M65). Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Biomarkers and Non-invasive Measures of Hepatic Fibrosis as Measured by Transforming Growth Factor Beta (TGF-beta) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Biomarkers and non-invasive measures of hepatic fibrosis were assessed as measured by TGF-beta. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Liver Stiffness Measured by Transient Elastography (TE) at Week 52
大体时间：Baseline (Day 1) and at Week 52

FibroScan® TE device (Echosens, Paris, France) is a non-invasive technique used to measure liver stiffness, which correlated with fibrosis. Baseline was defined as the last non-missing value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Lipid Parameters at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment of total cholesterol, low-density lipoprotein (LDL) cholesterol, high-density lipoprotein (HDL) cholesterol, very low-density lipoprotein (VLDL) cholesterol and triglycerides (TG). Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Fasting Plasma Glucose (FPG) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment of FPG. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Bile Acids and Biomarkers of Bile Acid Synthesis at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment bile acids and biomarkers of bile acid synthesis. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline at Week 52 in Bile Acids and Biomarkers of Bile Acid Synthesis as Measured by 7 Alpha-hydroxy-4-cholesten-3-one (C4) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment of bile acids and biomarkers of bile acid synthesis as measured by C4. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Bile Acids and Biomarkers of Bile Acid Synthesis as Measured Fibroblast Growth Factor 19 at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment bile acids and biomarkers of bile acid synthesis as measured by Fibroblast Growth Factor 19. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Percentage of Participants With Response in PBC Worst Itch NRS Score at Weeks 24 and 52
大体时间：Baseline (up to 14 days pre-dose) and at Week 24 and Week 52

The PBC Worst Itch NRS is a simple, self-administered PRO questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Response to PBC Worst Itch NRS was according to clinically meaningful change at least 30% reduction; one point, two points or three points decrease in score from baseline in participants with a baseline NRS score ≥4. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake).
Baseline (up to 14 days pre-dose) and at Week 24 and Week 52
Percentage of Participants With no Worsening of Pruritus as Measured by the PBC Worst Itch NRS Score at Weeks 24 and 52
大体时间：Baseline (up to 14 days pre-dose) and at Week 24 and Week 54

The PBC Worst Itch NRS is a simple, self-administered PRO questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake). A worsening of Pruritus was defined by a positive change from baseline in PBC Worst Itch NRS score greater than 2 at week 24 and 52.
Baseline (up to 14 days pre-dose) and at Week 24 and Week 54
Change From Baseline in 5-D Itch at Week 52
大体时间：Baseline (Day 1) and at Week 52

The 5-D Itch scale is a questionnaire that has been validated in several different diseases. It assesses symptoms in terms of 5 domains: duration: score range 1 (less than 6 hours) to 5 (all day); degree: score range 1 (not present) to 5 (unbearable); duration: score range 1 (less than 6 hours) and 5 (all day); direction: score range 1(completely resolved) and 5 (getting worse); disability: score range 1 (never affects or not applicable) and 5 (delays falling asleep and frequently wakes up at night or always affects this activity) and distribution: where the question was to mark where itching is present in body parts over the last two weeks. Participants rated their symptoms over preceding 2-week period on a 1 to 5 scale, with 5 being the most affected. The 5-D Itch total score ranged between 5 (no pruritus) and 25 (most severe pruritus). Higher scores indicated worse outcomes. Baseline= last non-missing value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue Short Form 7a at Week 52
大体时间：Baseline (Day 1) and at Week 52

The PROMIS Fatigue Short Form 7a consists of seven items that measure both the experience of fatigue and the interference of fatigue on daily activities over the past week. Participants completed the PROMIS Fatigue Short Form 7a and answered following questions on a scale from 1 (never) to 5 (always) : "How often did you feel tired"; "How often did you experience extreme exhaustion"; "How often did you run out of energy"; "How often did your fatigue limit you at work"; "How often were you too tired to think clearly"; "How often were you too tired to take a bath or shower" and 'How often did you have enough energy to exercise strenuously". T-score values include mean of 50 and standard deviation of 10. The PROMIS Fatigue T-score ranged from 29.4 (best) to 83.2 (worst). Higher scores indicated worse outcomes. Baseline was defined as the last non-missing value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Epworth Sleepiness Scale (ESS) at Week 52
大体时间：Baseline (Day 1) and at Week 52

The ESS is a short, self-administered questionnaire that consists of 8 questions asking to rate how likely it is to fall asleep in everyday situations (each question can be scored from 0 to 3 points; '0' indicates no chance of dozing and '3' indicates high chance of dozing). It provides a total score which has been shown to relate to the participant's level of daytime sleepiness (total score range 0 [no sleepiness] to 24 points [significant sleepiness]). The total score is the sum of all item scores and ranged from 0 (best) to 24 (worst). Higher scores indicated more chances of sleepiness. Participants were asked to complete the ESS with regard to the level of sleepiness they experienced over approximately the past 7 days. Baseline was defined as the last non-missing value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in PBC-40 at Week 52
大体时间：Baseline (Day 1) and at Week 52

PBC-40 assess symptoms across 6 domains:symptoms,itch,fatigue,cognitive,emotional and social. Participants responded on verbal response scale,depending on section options range from 'never'/'not at all'/'strongly disagree' to 'always'/'very much'/'strongly agree'.5 items (3/3 in itch domain and 2/10 in social domain)also included a 'does not apply' option.Domains:Symptoms(7 questions)score range 7-35,Itch(3 questions)score range 3-15,Fatigue(11 questions)score range 11-55,Cognitive(6 questions)score range 6-30,Emotional(3 questions)score range 3-15,Social(10 questions)score range 10-50.Score for each domain was provided(total score was not calculated),with each verbal response scale correlating to score of 1-5 per item(0-5 on items with a 'does not apply' option);5=most affected.Individual item scores are summed to give total domain score.Higher scores=worse outcomes.PBC-40 had 4-week recall period.Baseline=last non-missing value on or before first dose of randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Health Utility as Measured by the European Quality of Life 5 Dimensions 5 Level Version (EQ-5D-5L) at Week 52
大体时间：Baseline (Day 1) and at Week 52

The EQ-5D-5L is a 6-item, standardized questionnaire that assesses mobility, self-care, usual activities, pain/discomfort, anxiety/depression, and overall health state. The visual analog scale (VAS) of EQ-5D-5L questionnaire is numbered from 0 (worst) to 100 (best). Higher scores indicated better outcomes. Baseline was defined as the last non-missing value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in T-Scores for Bone Mineral Density Assessed by Dual-energy X-ray Absorptiometry (DEXA) Scanning at Week 52
大体时间：Baseline (Day 1) and at Week 52

DEXA scanning (hip and lumbar) was performed in participants to assess bone mineral density (femoral neck, lumbar and total hip). T-scores were calculated based on actual measured bone density value. The T-score compared a participant's bone density against that of a healthy 30-year-old adult. T-scores were used to determine primary osteoporosis, which exists on its own without any other cause and were divided into 3 categories: low risk, medium risk, and high risk (osteoporosis). T-scores measure the number of minerals in a participant's bone. A participant's level of bone loss was compared to that of a typical, healthy 30-year-old adult. A score ≥-1.0 indicates low risk, medium risk is a score between-2.5 and -1.0, and a high risk/osteoporosis is a score ≤-2.5. A positive change in T-score indicates an improvement in bone mineral density. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Serum Markers of Bone Turnover Carboxy Terminal Crosslinked Telopeptides of Type 1 Collagen [CTX] at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment of serum markers of bone turnover, CTX. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Change From Baseline in Serum Markers of Bone Turnover Type 1 Procollagen Peptide [P1NP]) at Week 52
大体时间：Baseline (Day 1) and at Week 52

Blood samples were collected at specified timepoints for assessment of serum markers of bone turnover, P1NP. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication.
Baseline (Day 1) and at Week 52
Percentage of Participants With Onset of Clinical Outcomes
大体时间：From Day 1 up to Week 52

Onset of clinical outcomes were described as a composite endpoint composed of the following: model for end stage liver disease sodium (MELD-Na) >14 for participants with baseline MELD-Na <12; liver transplant; uncontrolled ascites requiring treatment; hospitalization for new onset or recurrence of any including, variceal bleed, hepatic encephalopathy defined as West-Haven score of 2 or more and spontaneous bacterial peritonitis; and death.
From Day 1 up to Week 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs), Treatment-emergent Serious Adverse Events (TESAEs) and Adverse Events of Special Interest (AESIs)
大体时间：TEAEs were collected from the start of study treatment administration (Day 1) up to DCO date of 01 June 2023, approximately 980 days.

An AE was any untoward medical occurrence in a participant or clinical investigation participant who was administered an investigational product, and which does not necessarily have a causal relationship with treatment. A TEAE was defined as any AE with onset during TEAE assessment period, or any event started prior to first dose of treatment and worsened or became serious during the TEAE assessment period. An SAE was any AE that resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality/birth defect. An AESI was an AE that might not be serious but was of special importance to a particular treatment or class.
TEAEs were collected from the start of study treatment administration (Day 1) up to DCO date of 01 June 2023, approximately 980 days.
Plasma Concentrations of Elafibranor and GFT1007
大体时间：Week 4: pre-dose, 0.5 hour, 1.5 hours, between 2-3 hours, 4 hours and 6 hours post-dose

Blood samples were collected at specified timepoints to evaluate plasma concentration of Elafibranor and its metabolite GFT1007.
Week 4: pre-dose, 0.5 hour, 1.5 hours, between 2-3 hours, 4 hours and 6 hours post-dose

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研究主要日期

学习开始 (实际的)

2020年9月24日

初级完成 (实际的)

2023年6月1日

研究完成 (估计的)

2028年12月1日

研究注册日期

首次提交

2020年8月21日

首先提交符合 QC 标准的

2020年8月21日

首次发布 (实际的)

2020年8月26日

研究记录更新

最后更新发布 (实际的)

2026年11月5日

上次提交的符合 QC 标准的更新

2026年10月20日

最后验证

2026年10月1日

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其他研究编号

  • GFT505B-319-1
  • 2019-004941-34 (EudraCT编号)
  • 2019 (美国 NIH 拨款/合同)

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