Study to Evaluate the Efficacy and Safety of Elafibranor in Patients With Primary Biliary Cholangitis (PBC) and Inadequate Response to Ursodeoxycholic Acid

NCT ID: NCT03124108

Last Updated: 2019-09-24

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

45 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-04-05

Study Completion Date

2018-10-31

Brief Summary

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The primary objective of the study is to compare the effect of daily oral administration of elafibranor 80mg and 120 mg on change in serum alkaline phosphatase (ALP) to that of placebo in patients with PBC and inadequate response to Ursodeoxycholic acid (UDCA).

Detailed Description

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Conditions

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Primary Biliary Cholangitis (PBC)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Elafibranor 80 mg

Study subjects will take two tablets per day orally before breakfast with a glass of water each morning

Group Type ACTIVE_COMPARATOR

Elafibranor 80 mg

Intervention Type DRUG

Two coated tablets daily for 12 weeks

Elafibranor 120 mg

Study subjects will take two tablets per day orally before breakfast with a glass of water each morning

Group Type ACTIVE_COMPARATOR

Elafibranor 120 mg

Intervention Type DRUG

Two coated tablets daily for 12 weeks

Placebo

Study subjects will take two tablets per day orally before breakfast with a glass of water each morning

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Two coated tablets daily for 12 weeks

Interventions

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Elafibranor 80 mg

Two coated tablets daily for 12 weeks

Intervention Type DRUG

Elafibranor 120 mg

Two coated tablets daily for 12 weeks

Intervention Type DRUG

Placebo

Two coated tablets daily for 12 weeks

Intervention Type DRUG

Other Intervention Names

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GFT505 GFT505

Eligibility Criteria

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Inclusion Criteria

1. Must have provided written informed consent
2. Definite or probable PBC diagnosis as demonstrated by the presence of at least 2 of the following 3 diagnostic factors:

* History of elevated ALP levels for at least 6 months prior to Day 0 (randomization visit)
* Positive Anti-Mitochondrial Antibodies (AMA) titers (\> 1/40 on immunofluorescence or M2 positive by enzyme-linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies
* Liver biopsy consistent with PBC
3. ALP \>= 1.67x upper limit of normal (ULN)
4. Taking UDCA for at least 12 months (stable dose for ≥ 6 months) prior to screening visit
5. Contraception: Females participating in this study must be of non-childbearing potential or must be using highly efficient contraception for the full duration of the study and for 1 month after the end of treatment.

Exclusion Criteria

1. History or presence of other concomitant liver diseases
2. Screening creatine phosphokinase (CPK) \> upper limits of normal (ULN)
3. Screening alanine transaminase (ALT) or aspartate aminotransferase (AST) \> 5 ULN
4. Screening total bilirubin \> 2 ULN
5. Screening serum creatinine \> 1.5 mg/dl
6. Significant renal disease, including nephritic syndrome, chronic kidney disease (defined as patients with markers of kidney damage or estimated glomerular filtration rate \[eGFR\] of less than 60 mL/min/1.73 m\^2).
7. Patients with moderate or severe hepatic impairment (defined as Child-Pugh B/C)
8. Platelet count \<150 X 10\^3/microliter
9. Albumin \<3.5 g/dL
10. Presence of clinical complications of PBC or clinically significant hepatic decompensation
11. If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
12. Known history of human immunodeficiency virus (HIV) infection
13. Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Genfit

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Head

Role: STUDY_DIRECTOR

Genfit

Locations

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Mayo Clinic in Arizona

Phoenix, Arizona, United States

Site Status

Schiff Center for Liver Diseases

Miami, Florida, United States

Site Status

Cleveland Clinic Florida

Weston, Florida, United States

Site Status

Piedmont Research Institute

Atlanta, Georgia, United States

Site Status

Massachusetts General Hospital

Boston, Massachusetts, United States

Site Status

Beth Israel Deaconess Medical Center

Boston, Massachusetts, United States

Site Status

Northwell Health Institution

Manhasset, New York, United States

Site Status

Asheville Gastroenterology Associates

Asheville, North Carolina, United States

Site Status

UT Southwestern Medical Center

Dallas, Texas, United States

Site Status

Baylor College of Medicine

Houston, Texas, United States

Site Status

University of Virginia

Charlottesville, Virginia, United States

Site Status

Virginia Commonwealth University

Richmond, Virginia, United States

Site Status

Swedish Medical Center

Seattle, Washington, United States

Site Status

Hopital Saint-Antoine

Paris, , France

Site Status

Clinic for Gastroenterology and Hepatology

Cologne, , Germany

Site Status

University Hospital Frankfurt

Frankfurt, , Germany

Site Status

Johannes Gutenberg University

Mainz, , Germany

Site Status

Liver Unit, University of Barcelona

Barcelona, , Spain

Site Status

Hospital De La Sant Creu St. Pau

Barcelona, , Spain

Site Status

University of Birmingham Centre for Liver Research

Birmingham, , United Kingdom

Site Status

Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status

The Royal Liverpool University Hospital

Liverpool, , United Kingdom

Site Status

King's College Hospital

London, , United Kingdom

Site Status

The Newcastle Upon Tyne Hosptials NHS Foundation Trust

Newcastle upon Tyne, , United Kingdom

Site Status

Countries

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United States France Germany Spain United Kingdom

References

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Schattenberg JM, Pares A, Kowdley KV, Heneghan MA, Caldwell S, Pratt D, Bonder A, Hirschfield GM, Levy C, Vierling J, Jones D, Tailleux A, Staels B, Megnien S, Hanf R, Magrez D, Birman P, Luketic V. A randomized placebo-controlled trial of elafibranor in patients with primary biliary cholangitis and incomplete response to UDCA. J Hepatol. 2021 Jun;74(6):1344-1354. doi: 10.1016/j.jhep.2021.01.013. Epub 2021 Jan 21.

Reference Type DERIVED
PMID: 33484775 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-003817-80

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

GFT505B-216-1

Identifier Type: -

Identifier Source: org_study_id

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