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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06016842

Registration number

NCT06016842

Ethics application status

Date submitted

22/08/2023

Date registered

30/08/2023

Date last updated

28/06/2024

Titles & IDs

Public title

A Long-Term Study of Elafibranor in Adult Participants With Primary Biliary Cholangitis

Scientific title

A Phase III Randomised, Parallel-Group, Double-Blind, Placebo-Controlled, Two-Arm Study to Evaluate the Efficacy and Safety of Elafibranor 80 mg on Long-Term Clinical Outcomes in Adult Participants With Primary Biliary Cholangitis (PBC)

Secondary ID [1]

2023-505251-43-00

Secondary ID [2]

CLIN-60190-454

Universal Trial Number (UTN)

Trial acronym

ELFIDENCE

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Primary Biliary Cholangitis (PBC)

Condition category

Condition code

Oral and Gastrointestinal

Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon

Infection

Other infectious diseases

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

Treatment: Drugs - Elafibranor
Other interventions - Matched 80 mg placebo

Experimental: Elafibranor 80 mg - Participants will take 1 tablet of elafibranor 80 mg per day orally before breakfast with a glass of water at approximately the same time each morning.

Placebo comparator: Placebo - Participants will take 1 placebo tablet per day orally (matching the 80 mg elafibranor sized tablet) before breakfast with a glass of water at approximately the same time each morning.

Treatment: Drugs: Elafibranor
Duration: up to an estimated 84-month (7-year) double-blind treatment period during which elafibranor 80 mg tablet will be administered once daily

Other interventions: Matched 80 mg placebo
Duration: up to an estimated 84-month (7-year) double-blind treatment period during which matching placebo tablet will be administered once daily

Intervention code [1]

Treatment: Drugs

Intervention code [2]

Other interventions

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Event-free survival

Timepoint [1]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [1]

Percentage of participants experiencing Treatment Emergent Adverse Events (TEAEs), treatment-related TEAEs, Serious Adverse Events (SAEs), and Adverse Events of Special Interests (AESIs)

Timepoint [1]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [2]

Percentage of participants developing clinically significant changes in physical examination findings

Timepoint [2]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [3]

Percentage of participants developing clinically significant changes in vital signs

Timepoint [3]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [4]

Percentage of participants developing clinically significant changes in Electrocardiogram (ECG) readings.

Timepoint [4]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [5]

Percentage of participants with clinically significant changes in laboratory parameters (blood chemistry, hematology, coagulation and urinalysis)

Timepoint [5]

From baseline until 4 weeks after the end of treatment (maximum duration of 7 years)

Secondary outcome [6]

Change from baseline in Alkaline phosphatase (ALP)

Timepoint [6]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [7]

Change from baseline in Total Bilirubin (TB)

Timepoint [7]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [8]

Percentage of participants with ALP= 1.67x ULN and TB= ULN

Timepoint [8]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [9]

Percentage of participants with complete biochemical response

Timepoint [9]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [10]

Percentage of participants with normalisation of TB and ALP

Timepoint [10]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [11]

Percentage of participants with stabilisation in TB (i.e. no increase)

Timepoint [11]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [12]

Percentage of participants with a response based on albumin normalisation

Timepoint [12]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [13]

Change from baseline in liver stiffness measurement (LSM)

Timepoint [13]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [14]

Change from baseline in PBC risk scores based in Global PBC Study Group (GLOBE) score

Timepoint [14]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [15]

Change from baseline in PBC risk scores based on United Kingdom (UK)-PBC score.

Timepoint [15]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [16]

Percentage of participants with LSM =15 kPa

Timepoint [16]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [17]

Change in serum levels of Aspartate aminotransferase (AST) compared to the baseline

Timepoint [17]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [18]

Change in serum levels of ALT compared to the baseline

Timepoint [18]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [19]

Change in serum levels of Gamma-glutamyl transferase (GGT) compared to the baseline

Timepoint [19]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [20]

Change from baseline in hepatic function: Conjugated bilirubin

Timepoint [20]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [21]

Change in serum levels of Albumin compared to the baseline

Timepoint [21]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [22]

Change from baseline in hepatic function: international normalised ratio (INR)

Timepoint [22]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [23]

Change from baseline in hepatic function: fractionated ALP

Timepoint [23]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [24]

Percentage of participants with no worsening of LSM

Timepoint [24]

At Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [25]

Percentage of participants with ALP reduction of 40%

Timepoint [25]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [26]

Percentage of participants with ALP <1.5x ULN, ALP decrease =15% and TB =ULN

Timepoint [26]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [27]

Percentage of participants with ALP <1.5x ULN, ALP decrease =40% and TB =ULN

Timepoint [27]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [28]

Percentage of participants with ALP <1.67x ULN, ALP decrease =15% and TB =ULN

Timepoint [28]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [29]

Percentage of participants with ALP <3x ULN, AST <2x ULN and TB =1 mg/dL (Paris I)

Timepoint [29]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [30]

Percentage of participants with ALP =1.5x ULN, AST =1.5x ULN and TB =1 mg/dL (Paris II criteria)

Timepoint [30]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [31]

Percentage of participants with normalisation of abnormal TB

Timepoint [31]

Assessed at Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [32]

Percentage of participants with normalisation of abnormal TB and albumin (Rotterdam criteria)

Timepoint [32]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [33]

Percentage of participants with reduction in TB to =0.6x ULN in participants with TB >0.6x ULN at baseline

Timepoint [33]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [34]

Change from baseline in lipid parameters: total cholesterol (TC)

Timepoint [34]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [35]

Change from baseline in lipid parameters: high density lipoprotein cholesterol (HDL-C)

Timepoint [35]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [36]

Change from baseline in lipid parameters: calculated very low density lipoprotein cholesterol (VLDL-C)

Timepoint [36]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [37]

Change from baseline in lipid parameters: low density lipoprotein cholesterol (LDL-C)

Timepoint [37]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [38]

Change from baseline in lipid parameters: triglycerides (TG)

Timepoint [38]

At Month 6, Month 12, and every 12 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [39]

Percentage of participants with a response in PBC Worst Itch NRS score

Timepoint [39]

Through 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [40]

Percentage of participants with a response in PBC Worst Itch NRS

Timepoint [40]

Assessed through 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [41]

Change from baseline in 5D-Itch scale

Timepoint [41]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [42]

Change from baseline in Patient Global Impression of Severity (PGI-S)

Timepoint [42]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [43]

Change from baseline in Patient Global Impression of Change (PGI-C)

Timepoint [43]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [44]

Change from baseline in Patient Reported Outcome Measurement Information System (PROMIS) Fatigue Short Form 7a

Timepoint [44]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [45]

Change from baseline in the Epworth Sleepiness Scale (ESS)

Timepoint [45]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [46]

Change from baseline in PBC-40 score

Timepoint [46]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [47]

Change from baseline in PBC Worst Itch Numeric Rating Scale (NRS) score

Timepoint [47]

Assessed through 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [48]

Change from baseline in EuroQol 5-dimensional 5-level questionnaire (EQ-5D-5L)

Timepoint [48]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [49]

Change from baseline in Work Productivity and Activity Impairment General Health (WPAI-GH)

Timepoint [49]

Assessed at every 6 months up to end of treatment (maximum duration of 7 years)

Secondary outcome [50]

Time to the first occurrence of each of individual adjudicated clinical outcome events

Timepoint [50]

From baseline until 4 weeks after the end of treatment

Secondary outcome [51]

Area under the plasma concentration-time curve from time 0 to 24 hours: AUC0-24

Timepoint [51]

At Day 1/Baseline, Month 6 and Month 12 (during a dosing period of 24 hours)

Secondary outcome [52]

Maximum (peak) plasma drug concentration: Cmax

Timepoint [52]

At Day 1/Baseline, Month 6 and Month 12 (during a dosing period of 24 hours)

Secondary outcome [53]

Time to reach maximum (peak) plasma concentration following drug administration): Tmax

Timepoint [53]

At Day 1/Baseline, Month 6 and Month 12 (during a dosing period of 24 hours)

Secondary outcome [54]

Apparent clearance of drug from plasma (CL)

Timepoint [54]

At Day 1/Baseline, Month 6 and Month 12 (during a dosing period of 24 hours)

Secondary outcome [55]

Apparent volume of distribution (VZ)

Timepoint [55]

At Day 1/Baseline, Month 6 and Month 12 (during a dosing period of 24 hours)

Eligibility

Key inclusion criteria

Inclusion Criteria :

* Male or female participants must be =18 years of age at the time of signing the informed consent.
* Participants with a definite or probable diagnosis of primary biliary cholangitis (PBC)
* Participants taking ursodeoxycholic acid (UDCA) for at least 12 months (at a stable dose for =3 months) prior to screening period and expected to remain on stable dose during the study, or unable to tolerate UDCA treatment (no UDCA for =3 months) prior to screening period (per country standard-of-care dosing).
* Participants taking medications for management of pruritus (e.g. cholestyramine, rifampin, naltrexone, sertraline or colchicine) must be on a stable dose for =3 months prior to screening period.
* Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
* Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Minimum age

18 Years

Maximum age

No limit

Sex

Both males and females

Can healthy volunteers participate?

Key exclusion criteria

Exclusion Criteria :

* History or presence of other concomitant liver disease including but not limited to: i) Primary sclerosing cholangitis (PSC). ii) Autoimmune hepatitis (AIH) by simplified Diagnostic Criteria of the International Autoimmune Hepatitis Group (IAIHG) =6, or if treated for an overlap of PBC with AIH, or if there is clinical suspicion and evidence of overlap AIH features, that cannot be explained alone by insufficient response to UDCA. iii) Positive hepatitis B surface antigen (HBsAg). Participants with negative HBsAg and positive hepatitis B core antibody (HBcAb) may be eligible if hepatitis B virus deoxyribonucleic acid (HBV DNA) is negative. iv) Hepatitis C virus (HCV) infection defined by positive anti-HCV antibody and positive HCV ribonucleic acid (RNA) (Note: Participants with positive anti-HCV antibody due to previously treated HCV infection, may be enrolled if a confirmatory HCV RNA is undetectable and sustained viral response has been documented). v) Alcohol-associated liver disease (ALD). vi) Nonalcoholic steatohepatitis (NASH). vii) Other chronic liver diseases, such as alpha-1 antitrypsin deficiency.
* History or presence of clinically significant hepatic decompensation, including: i) History of liver transplantation, current placement on a liver transplant list, current model for end-stage liver disease including serum sodium (MELD)-Na score =12 due to hepatic impairment (MELD-Na will be calculated only when MELD >11). ii) Evidence of complications of cirrhosis, including hepatic decompensation or evidence of significant portal hypertension complications including presence of uncontrolled ascites; history of variceal bleeding or related interventions (e.g. variceal banding, or transjugular intrahepatic portosystemic shunt placement); presence of hepatic encephalopathy Grade 2 or higher per West-Haven criteria; history or presence of spontaneous bacterial peritonitis. Note: participants with low-risk varices (Grade I) without history of bleeding or other treatment may be eligible to enrol. iii) Hepatorenal syndrome (HRS) (type I or II).
* Known history of human immunodeficiency virus (HIV) infection or having a positive confirmatory test for HIV type 1 or 2.
* Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease).
* Evidence of any other unstable or untreated clinically significant immunological, endocrine, hematologic, gastrointestinal, neurological, or psychiatric disease as evaluated by the investigator; other clinically significant conditions that are not well controlled.
* Non-hepatic medical conditions that may diminish life expectancy to <2 years, including known cancers.
* History of hepatocellular carcinoma.
* Alpha-fetoprotein (AFP) >20 ng/mL with 4-phase liver computerised tomography (CT) or magnetic resonance imaging (MRI) imaging suggesting presence of 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.
* Administration of the following medications is prohibited during the study, and prior to the study as per the timelines specified below: i) 3 months prior to screening period: fibrates, seladelpar, glitazones, obeticholic acid, azathioprine, cyclosporine, methotrexate, mycophenolate, pentoxifylline, budesonide and other systemic corticosteroids (parenteral and oral chronic administration only); potentially hepatotoxic drugs (including a-methyl-dopa, sodium valproic acid, isoniazid or nitrofurantoin).
* Participants with previous exposure to elafibranor.
* Participants 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 5 half-lives, whichever is longer, prior to the screening period.

i) If the previous study was for an experimental therapy being studied for potential benefit in PBC, and the potential therapeutic agent was proven to have no beneficial effect in PBC and there are no safety concerns, the participant may enrol after 30 days or 5 half-lives from the last dose of the therapeutic agent, whichever is longer. ii) For therapeutic agents being studied for potential benefit in PBC for which it is still unclear if there may be a potential benefit, participants may enrol after 6 months from the last dose of the therapeutic agent.
* 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. For participants with bundle branch block or other intraventricular conduction delay, a longer QTcF >480 msec would be exclusionary.
* Total bilirubin (TB) >3x ULN. Participants with Gilbert's syndrome are eligible with a total bilirubin above 3× ULN if direct bilirubin is <30% of total bilirubin.
* Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) >5x ULN at SV1, or variability >40% based on two consecutive values. The interval between the two measurements should be of at least 2 weeks (and up to 4 weeks):
* Creatinine phosphokinase (CPK) >2x ULN.
* Platelet count <75,000/µL
* International normalised ratio (INR) >1.5 in the absence of anticoagulant therapy.
* Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73m2 per the Modification of Diet in Renal Disease (MDRD)-6 Study formula at SV1.
* Significant renal disease, including nephritic syndrome, chronic kidney disease (CKD) (defined as participants with evidence of significantly impaired kidney function or underlying kidney injury).
* For female participants: known current pregnancy, or has a positive serum pregnancy test, or is breastfeeding.
* Regular alcohol intake in excess of the recommended limit of 1 standard drink per day for men or women.
* History of alcohol abuse, or other substance abuse within 1 year prior to SV1.
* A positive drug screen at screening would be exclusionary unless it can be explained by a prescribed medication. Use of cannabidiol (CBD) or other cannabinoids is not exclusionary for this study.
* Known hypersensitivity to elafibranor or to any of the excipients of the investigational product(s).
* Mental instability or incompetence, such that the validity of informed consent or ability to be compliant with the study is uncertain.
* Any other condition that, in the opinion of the investigator, would interfere with study participation or completion, or would put the participant at risk, including a potential participant assessed as being at high risk of noncompliance with the study.

Study design

Purpose of the study

Treatment

Allocation to intervention

Randomised controlled trial

Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

Masking / blinding

Blinded (masking used)

Who is / are masked / blinded?

The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data

Intervention assignment

Parallel

Other design features

Phase

Phase 3

Type of endpoint/s

Statistical methods / analysis

Recruitment

Recruitment status

Recruiting

Data analysis

Reason for early stopping/withdrawal

Other reasons

Date of first participant enrolment

Anticipated

Actual

31/08/2023

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

18/10/2030

Actual

Sample size

Target

450

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

Recruitment hospital [1]

St George Hospital - Sydney

Recruitment hospital [2]

Westmead Hospital - Westmead

Recruitment postcode(s) [1]

- Sydney

Recruitment postcode(s) [2]

- Westmead

Recruitment outside Australia

Country [1]

United States of America

State/province [1]

Arizona

Country [2]

United States of America

State/province [2]

Arkansas

Country [3]

United States of America

State/province [3]

California

Country [4]

United States of America

State/province [4]

Colorado

Country [5]

United States of America

State/province [5]

Florida

Country [6]

United States of America

State/province [6]

Louisiana

Country [7]

United States of America

State/province [7]

Michigan

Country [8]

United States of America

State/province [8]

South Carolina

Country [9]

United States of America

State/province [9]

Tennessee

Country [10]

United States of America

State/province [10]

Texas

Country [11]

United States of America

State/province [11]

Virginia

Country [12]

Belgium

State/province [12]

Edegem

Country [13]

Belgium

State/province [13]

Gent

Country [14]

Belgium

State/province [14]

Limbourg

Country [15]

Belgium

State/province [15]

Roeselare

Country [16]

Czechia

State/province [16]

Hradec Králové

Country [17]

Czechia

State/province [17]

Liberec

Country [18]

Czechia

State/province [18]

Ostrava

Country [19]

Czechia

State/province [19]

Plzen

Country [20]

Czechia

State/province [20]

Prague

Country [21]

France

State/province [21]

Lille

Country [22]

France

State/province [22]

Lyon

Country [23]

France

State/province [23]

Marseille

Country [24]

France

State/province [24]

Nice

Country [25]

France

State/province [25]

Paris

Country [26]

France

State/province [26]

Poitiers

Country [27]

France

State/province [27]

Toulouse

Country [28]

France

State/province [28]

Villejuif

Country [29]

Italy

State/province [29]

Modena

Country [30]

Italy

State/province [30]

Monza

Country [31]

Italy

State/province [31]

Naples

Country [32]

Italy

State/province [32]

Padova

Country [33]

Italy

State/province [33]

Palermo

Country [34]

Italy

State/province [34]

Pavia

Country [35]

Italy

State/province [35]

Pisa

Country [36]

Italy

State/province [36]

San Giovanni Rotondo

Country [37]

Korea, Republic of

State/province [37]

Busan

Country [38]

Korea, Republic of

State/province [38]

Daegu

Country [39]

Korea, Republic of

State/province [39]

Daejeon

Country [40]

Korea, Republic of

State/province [40]

Gyeonggi-do

Country [41]

Korea, Republic of

State/province [41]

Junggu

Country [42]

Korea, Republic of

State/province [42]

Seongnam-si

Country [43]

Korea, Republic of

State/province [43]

Seoul

Country [44]

Korea, Republic of

State/province [44]

Uijeongbu

Country [45]

New Zealand

State/province [45]

Christchurch

Country [46]

Poland

State/province [46]

Elblag

Country [47]

Poland

State/province [47]

Katowice

Country [48]

Poland

State/province [48]

Kraków

Country [49]

Poland

State/province [49]

Malogoskie

Country [50]

Poland

State/province [50]

Sosnowiec

Country [51]

Poland

State/province [51]

Warszawa

Country [52]

Romania

State/province [52]

Bucharest

Country [53]

Romania

State/province [53]

Iasi

Country [54]

Spain

State/province [54]

Barcelona

Country [55]

Spain

State/province [55]

Madrid

Country [56]

Spain

State/province [56]

Majadahonda

Country [57]

Spain

State/province [57]

Málaga

Country [58]

Spain

State/province [58]

Pontevedra

Country [59]

Spain

State/province [59]

Sabadell

Country [60]

Spain

State/province [60]

Sevilla

Country [61]

Spain

State/province [61]

Valencia

Country [62]

Spain

State/province [62]

Valladolid

Country [63]

Spain

State/province [63]

Zaragoza

Funding & Sponsors

Primary sponsor type

Commercial sector/industry

Name

Ipsen

Address

Country

Ethics approval

Ethics application status

Summary

Brief summary

The participants of this study will have confirmed Primary Biliary Cholangitis (PBC).

Participants will also have inadequate response or intolerance to ursodeoxycholic acid (UDCA) a drug used to treat PBC.

PBC is a disease that progresses slowly. It causes damage to 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 people with PBC may require 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). Each participant will be in the study up to about 7 years.

The main aim of this study is to determine if elafibranor is better than placebo in preventing clinical outcome events showing disease worsening (including progression of disease leading to liver transplant or death).

This study will also study the safety of long-term treatment with elafibranor, as well as the impact on symptoms such as itching and tiredness.

Trial website

https://clinicaltrials.gov/study/NCT06016842

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Ipsen Medical Director

Address

Ipsen

Country

Phone

Fax

Contact person for public queries

Name

Ipsen Clinical Study Enquiries

Address

Country

Phone

See e mail

Fax

clinical.trials@ipsen.com

Contact person for scientific queries

Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06016842

Trial Review

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06016842