NCT03124108

Brief Summary

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).

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
45

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2017

Geographic Reach
5 countries

24 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 28, 2017

Completed
8 days until next milestone

Study Start

First participant enrolled

April 5, 2017

Completed
16 days until next milestone

First Posted

Study publicly available on registry

April 21, 2017

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2018

Completed
11 months until next milestone

Results Posted

Study results publicly available

September 24, 2019

Completed
Last Updated

September 24, 2019

Status Verified

September 1, 2019

Enrollment Period

1.6 years

First QC Date

March 28, 2017

Results QC Date

August 16, 2019

Last Update Submit

September 20, 2019

Conditions

Primary Biliary Cholangitis (PBC)

Keywords

ElafibranorPrimary Biliary CholangitisAlkaline phosphatase

Outcome Measures

Primary Outcomes (1)

  • Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) Levels at Week 12 (Endpoint)

    Relative change from baseline is in serum ALP levels at Week 12 (endpoint) were reported. Relative change from baseline is defined as percentage (%) change from baseline to endpoint.

    Baseline, Week 12 (Endpoint)

Secondary Outcomes (41)

  • Percentage of Participants With Response Defined by Composite Risk Scores (ALP< 1.67 * Upper Limit of Normal [ULN] at Endpoint, Total Bilirubin [BIL] Within Normal Limits at Endpoint, and Greater Than [>] 15% ALP Reduction From Baseline to Endpoint)

    Up to Week 12 (Endpoint)

  • Percentage of Participants With Response Defined by Composite Risk Scores (ALP < 2 * Upper Limit of Normal at Endpoint, Total Bilirubin Within Normal Limits at Endpoint, and > 40% ALP Reduction From Baseline to Endpoint)

    Up to Week 12 (Endpoint)

  • Percentage of Participants With Response Based on PARIS I Risk Score at Endpoint

    At Week 12 (Endpoint)

  • Percentage of Participants With Response Based on PARIS II Risk Score at Endpoint

    At Week 12 (Endpoint)

  • Percentage of Participants With Response Based on Toronto I Risk Score at Endpoint

    At Week 12 (Endpoint)

  • +36 more secondary outcomes

Study Arms (3)

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Elafibranor 80 mg

ACTIVE COMPARATOR

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

Drug: Elafibranor 80 mg

Elafibranor 120 mg

ACTIVE COMPARATOR

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

Drug: Elafibranor 120 mg

Interventions

Elafibranor 80 mgDRUG

Two coated tablets daily for 12 weeks

Also known as: GFT505
Elafibranor 80 mg
Elafibranor 120 mgDRUG

Two coated tablets daily for 12 weeks

Also known as: GFT505
Elafibranor 120 mg
PlaceboDRUG

Two coated tablets daily for 12 weeks

Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Must have provided written informed consent
  • 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
  • ALP \>= 1.67x upper limit of normal (ULN)
  • Taking UDCA for at least 12 months (stable dose for ≥ 6 months) prior to screening visit
  • 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.

You may not qualify if:

  • History or presence of other concomitant liver diseases
  • Screening creatine phosphokinase (CPK) \> upper limits of normal (ULN)
  • Screening alanine transaminase (ALT) or aspartate aminotransferase (AST) \> 5 ULN
  • Screening total bilirubin \> 2 ULN
  • Screening serum creatinine \> 1.5 mg/dl
  • 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).
  • Patients with moderate or severe hepatic impairment (defined as Child-Pugh B/C)
  • Platelet count \<150 X 10\^3/microliter
  • Albumin \<3.5 g/dL
  • Presence of clinical complications of PBC or clinically significant hepatic decompensation
  • If female: known pregnancy, or has a positive urine pregnancy test (confirmed by a positive serum pregnancy test), or lactating
  • Known history of human immunodeficiency virus (HIV) infection
  • Medical conditions that may cause non-hepatic increases in ALP (e.g., Paget's disease)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (24)

Mayo Clinic in Arizona

Phoenix, Arizona, 85054, United States

Location

Schiff Center for Liver Diseases

Miami, Florida, 33136, United States

Location

Cleveland Clinic Florida

Weston, Florida, 33331, United States

Location

Piedmont Research Institute

Atlanta, Georgia, 30309, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

Northwell Health Institution

Manhasset, New York, 11030, United States

Location

Asheville Gastroenterology Associates

Asheville, North Carolina, 28801, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

University of Virginia

Charlottesville, Virginia, 22903, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

Swedish Medical Center

Seattle, Washington, 98122, United States

Location

Hopital Saint-Antoine

Paris, 75012, France

Location

Clinic for Gastroenterology and Hepatology

Cologne, 50937, Germany

Location

University Hospital Frankfurt

Frankfurt, 60590, Germany

Location

Johannes Gutenberg University

Mainz, 55131, Germany

Location

Liver Unit, University of Barcelona

Barcelona, 08036, Spain

Location

Hospital De La Sant Creu St. Pau

Barcelona, 08041, Spain

Location

University of Birmingham Centre for Liver Research

Birmingham, B15 2TT, United Kingdom

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

The Royal Liverpool University Hospital

Liverpool, L7 8XP, United Kingdom

Location

King's College Hospital

London, SE5 9RS, United Kingdom

Location

The Newcastle Upon Tyne Hosptials NHS Foundation Trust

Newcastle upon Tyne, NE7 7DN, United Kingdom

Location

Related Publications (1)

  • 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.

    PMID: 33484775DERIVED

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, IntrahepaticCholestasisBile Duct DiseasesBiliary Tract DiseasesDigestive System DiseasesLiver DiseasesLiver CirrhosisFibrosisPathologic ProcessesPathological Conditions, Signs and Symptoms

Results Point of Contact

Title
Clinical Head
Organization
Genfit SA
clinicaltrial@genfit.com
+33320164000

Study Officials

  • Clinical Head

    Genfit

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 28, 2017

First Posted

April 21, 2017

Study Start

April 5, 2017

Primary Completion

October 31, 2018

Study Completion

October 31, 2018

Last Updated

September 24, 2019

Results First Posted

September 24, 2019

Record last verified: 2019-09

Locations

FR(1)GB(5)US(13)DE(3)ES(2)