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

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ClinicalTrials.gov Identifier: NCT03124108
Recruitment Status : Completed
First Posted : April 21, 2017
Results First Posted : September 24, 2019
Last Update Posted : September 24, 2019
Sponsor:
Information provided by (Responsible Party):
Genfit

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Primary Biliary Cholangitis (PBC)
Interventions Drug: Elafibranor 80 mg
Drug: Elafibranor 120 mg
Drug: Placebo
Enrollment 45
Recruitment Details  
Pre-assignment Details A total of 68 participants were screened, out of which 45 participants were randomized, 15 participants in each of the 3 treatment groups.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks. Participants received elafibranor 120 mg tablets orally once daily for 12 weeks. Participants received matching placebo tablets orally once daily for 12 weeks.
Period Title: Overall Study
Started 15 15 15
Completed 15 14 15
Not Completed 0 1 0
Reason Not Completed
Adverse Event             0             1             0
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo Total
Hide Arm/Group Description Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks. Participants received elafibranor 120 mg tablets orally once daily for 12 weeks. Participants received matching placebo tablets orally once daily for 12 weeks. Total of all reporting groups
Overall Number of Baseline Participants 15 15 15 45
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 15 participants 45 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
13
  86.7%
10
  66.7%
11
  73.3%
34
  75.6%
>=65 years
2
  13.3%
5
  33.3%
4
  26.7%
11
  24.4%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 15 participants 15 participants 15 participants 45 participants
56.5  (8.7) 60.4  (6.9) 60.5  (8.6) 59.1  (8.2)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 15 participants 45 participants
Female
14
  93.3%
15
 100.0%
14
  93.3%
43
  95.6%
Male
1
   6.7%
0
   0.0%
1
   6.7%
2
   4.4%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 15 participants 45 participants
Hispanic or Latino
3
  20.0%
2
  13.3%
5
  33.3%
10
  22.2%
Not Hispanic or Latino
12
  80.0%
13
  86.7%
10
  66.7%
35
  77.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 15 participants 15 participants 45 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
1
   6.7%
1
   2.2%
White
15
 100.0%
15
 100.0%
14
  93.3%
44
  97.8%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1.Primary Outcome
Title Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) Levels at Week 12 (Endpoint)
Hide Description 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.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (mITT) analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: Percent change
-48.264  (14.7676) -40.640  (17.3624) 3.190  (14.8059)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Elafibranor 80mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference in percentage
Estimated Value -52.0
Confidence Interval (2-Sided) 95%
-62.5 to -41.5
Parameter Dispersion
Type: Standard Error of the Mean
Value: 5.4
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Elafibranor 120mg, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference in percentage
Estimated Value -43.9
Confidence Interval (2-Sided) 95%
-55.7 to -32.1
Parameter Dispersion
Type: Standard Error of the Mean
Value: 6
Estimation Comments [Not Specified]
2.Secondary Outcome
Title 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)
Hide Description Percentage of participants with response defined by Composite Risk Scores (ALP Less than [<] 1.67 * ULN at endpoint, Total BIL within normal limits at endpoint, and > 15% ALP reduction from baseline to Endpoint) was reported.
Time Frame Up to Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
66.7 78.6 6.7
3.Secondary Outcome
Title 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)
Hide Description Percentage of participants with response defined by composite risk scores (ALP < 2 * ULN at endpoint, Total BIL within normal limits at endpoint, and > 40% ALP reduction from baseline to endpoint) was reported.
Time Frame Up to Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
73.3 42.9 0
4.Secondary Outcome
Title Percentage of Participants With Response Based on PARIS I Risk Score at Endpoint
Hide Description Percentage of participants with response based on Paris I risk score was defined as ALP less than or equal to (<=) 3 * ULN and aspartate aminotransferase (AST) <= 2 * ULN and bilirubin within normal limits.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
80.0 78.6 53.3
5.Secondary Outcome
Title Percentage of Participants With Response Based on PARIS II Risk Score at Endpoint
Hide Description Percentage of participants with response based on Paris II risk score was defined as ALP <= 1.5 * ULN and AST <= 1.5 * ULN and bilirubin within normal limits.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
53.3 50.0 0
6.Secondary Outcome
Title Percentage of Participants With Response Based on Toronto I Risk Score at Endpoint
Hide Description Percentage of participants with response based on Toronto I risk score was defined as ALP <= 1.67 *ULN.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
66.7 78.6 6.7
7.Secondary Outcome
Title Percentage of Participants With Response Based on Toronto II Risk Score at Endpoint
Hide Description Percentage of participants with response based on Toronto II risk scores was defined as ALP <= 1.75 * ULN.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
66.7 78.6 6.7
8.Secondary Outcome
Title Median Percentage Risk as Assessed by United Kingdom-Primary Biliary Cholangitis (UK-PBC) Risk Total Score at Endpoint
Hide Description UK-PBC risk score at endpoint estimated that the median percentage risk that a participant treated with ursodeoxycholic acid (UDCA) will develop liver failure requiring liver transplant in 5, 10 and 15 years. UK-PBC score was calculated at each of the 3 survivor functions 1-baseline survival function^exp(0.0287854*[alpEPxuln-1.722136304] - 0.0422873*[{(altastEPxuln/10)^-1} - 8.675729006] + 1.4199 * [ln{bilEPxuln /10}+2.709607778] -1.960303*[albxlln -1.17673001]-0.4161954*[ pltxlln -1.873564875]). Where: Baseline survivor function=0. 982 (at 5 years); 0. 941 (at 10 years); 0.893 (at 15 years). alpEPxuln = ALP at endpoint/upper level normal ALP; altastEPxuln=(ALT, AST) at endpoint/upper level normal of the value; bilEPxuln=bilirubin at endpoint/upper level normal bilirubin; albxlln=albumin at baseline/albumin lower level normal; pltxlln=platelet count at baseline/ platelet count lower level normal.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Median (Full Range)
Unit of Measure: Percentage risk
5 year risk at Week 12
0.80
(0.1 to 6.0)
0.95
(0.2 to 5.8)
1.30
(0.1 to 3.7)
10 year risk at Week 12
2.60
(0.3 to 18.8)
3.05
(0.8 to 18.1)
4.40
(0.5 to 12.0)
15 year risk at Week 12
4.70
(0.6 to 32.1)
5.55
(1.5 to 31.0)
8.00
(0.9 to 21.1)
9.Secondary Outcome
Title Percentage of Participants With Response Defined by 10, 20 and 40 Percent Reduction in Alkaline Phosphatase
Hide Description Percentage of participants with response (defined by at least 10%, 20%, and 40% decrease in ALP from baseline to Endpoint) reported.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
10 Percent Reduction 93.3 92.9 13.3
20 Percent Reduction 93.3 92.9 6.7
40 Percent Reduction 86.7 57.1 0
10.Secondary Outcome
Title Percentage of Participants With Response Defined by Normalized Alkaline Phosphatase Levels at Endpoint
Hide Description The response was defined by normalized ALP levels (ALP ULN 105 units per liter [U/L] for females, 129 U/L for males) at endpoint.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
13.3 21.4 0
11.Secondary Outcome
Title Percentage of Participants With Response Defined by Normalized Bilirubin (BIL) at Endpoint
Hide Description The response was defined by normalized BIL levels (BIL ULN <1.20 milligram per deciliter [mg/dL]) at endpoint.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
86.7 92.9 93.3
12.Secondary Outcome
Title Percentage of Participants With Response Defined by Normalized Albumin (ALB) Levels at Endpoint
Hide Description The response was defined by normalized ALB levels (3.5-5.2 gram per deciliter [g/dL] for ages 18-60 years; 3.2-4.6 g/dL for ages 61-91 years) at endpoint.
Time Frame At Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Measure Type: Number
Unit of Measure: Percentage of participants
100 100 100
13.Secondary Outcome
Title Change From Baseline in Alanine Aminotransferase (ALT) Levels at Endpoint
Hide Description Change from baseline in ALT levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: U/L
-0.5  (57.38) 7.3  (29.13) -1.2  (8.57)
14.Secondary Outcome
Title Change From Baseline in Aspartate Aminotransferase (AST) Levels at Endpoint
Hide Description Change from baseline in AST levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: U/L
6.0  (55.29) 11.1  (27.96) -4.3  (7.97)
15.Secondary Outcome
Title Change From Baseline in Gamma-glutamyl Transferase (GGT) Levels at Endpoint
Hide Description Change from baseline in GGT levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: U/L
-91.5  (95.30) -61.9  (70.82) 0.6  (54.40)
16.Secondary Outcome
Title Change From Baseline in 5 Prime (’) Nucleotidase Levels at Endpoint
Hide Description Change from baseline in 5' nucleotidase levels at endpoint was reported. 5’ nucleotidase is an enzyme used as a biomarker of hepatobiliary cholestasis and is less sensitive but more specific than GGT and ALP.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: U/L
-7.81  (8.279) -4.59  (13.067) -0.47  (3.491)
17.Secondary Outcome
Title Change From Baseline in Total Bilirubin (BIL) Levels at Endpoint
Hide Description Change from baseline in total BIL levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: micromole per liter (mcmol/L)
-0.23  (3.425) -0.51  (2.821) -0.01  (3.548)
18.Secondary Outcome
Title Change From Baseline in Conjugated Bilirubin Levels at Endpoint
Hide Description Change from baseline in conjugated bilirubin levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: mcmol/L
0.34  (2.229) -0.06  (0.596) 0.45  (1.526)
19.Secondary Outcome
Title Change From Baseline in Albumin Levels at Endpoint
Hide Description Change from baseline in albumin levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: gram per liter (g/L)
2.2  (2.54) 2.3  (2.73) 0.0  (2.20)
20.Secondary Outcome
Title Change From Baseline in Cholesterol Levels at Endpoint
Hide Description Change from baseline in cholesterol levels at endpoints was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: millimole per liter (mmol/L)
-0.455  (0.7479) -0.387  (0.6308) 0.043  (0.3706)
21.Secondary Outcome
Title Change From Baseline in Low-density Lipoprotein (LDL) Cholesterol Levels at Endpoint
Hide Description Change from baseline in LDL-cholesterol at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: mmol/L
-0.366  (0.5919) -0.334  (0.4848) 0.061  (0.3272)
22.Secondary Outcome
Title Change From Baseline in High-density Lipoprotein (HDL) Cholesterol Levels at Endpoint
Hide Description Change from baseline in HDL-cholesterol levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: mmol/L
-0.017  (0.3898) 0.059  (0.3391) -0.007  (0.2988)
23.Secondary Outcome
Title Change From Baseline in Triglycerides Levels at Endpoint
Hide Description Change from baseline in triglycerides levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: mmol/L
-0.155  (0.3460) -0.253  (0.2085) -0.019  (0.3776)
24.Secondary Outcome
Title Change From Baseline in Total Free Bile Acid Levels at Endpoint
Hide Description Change from baseline in total free bile acid levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: 10^-9 mole per liter (mol/L)
-248.88  (2496.672) -673.71  (2962.097) -135.20  (6777.727)
25.Secondary Outcome
Title Change From Baseline in Total Conjugated Bile Acid Levels at Endpoint
Hide Description Change from baseline in total conjugated bile acid levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: 10^-9 mol/L
5008.99  (17844.304) -3280.16  (10941.769) 1873.22  (21795.349)
26.Secondary Outcome
Title Change From Baseline in Total Bile Acid Levels at Endpoint
Hide Description Change from baseline in total bile acid levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: 10^-9 mol/L
4760.11  (18919.661) -3953.86  (12008.620) 1738.02  (26521.746)
27.Secondary Outcome
Title Change From Baseline in 7 Alpha-hydroxy-4-cholesten-3-one Levels at Endpoint
Hide Description Change from baseline in 7 alpha-hydroxy-4-cholesten-3-one levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: 10^-9 mol/L
-16.29  (27.584) -10.04  (28.606) 5.22  (10.848)
28.Secondary Outcome
Title Change From Baseline in Fibroblast Growth Factor-19 Levels at Endpoint
Hide Description Change from baseline in fibroblast growth factor-19 levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: nanogram per liter (ng/L)
-21.67  (52.588) -16.96  (38.933) -47.08  (69.560)
29.Secondary Outcome
Title Change From Baseline in Immunoglobulin M (IgM) Levels at Endpoint
Hide Description Change from baseline in IgM levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: g/L
-0.339  (0.5846) -0.472  (0.5507) -0.076  (0.7227)
30.Secondary Outcome
Title Change From Baseline in Tumor Necrosis Factor Levels at Endpoint
Hide Description Change from baseline in tumor necrosis factor levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: ng/L
0.066  (0.7829) 0.154  (1.1374) 0.053  (0.8329)
31.Secondary Outcome
Title Change From Baseline in Transforming Growth Factor Beta Levels at Endpoint
Hide Description Change from baseline in transforming growth factor beta levels at endpoint was reported,
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: ng/L
734.7  (2103.75) 297.2  (2762.61) -1163.0  (4295.49)
32.Secondary Outcome
Title Change From Baseline in Interleukin 6 Levels at Endpoint
Hide Description Change from baseline in interleukin 6 levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: ng/L
-0.021  (0.8337) -0.261  (0.5213) -0.165  (0.5624)
33.Secondary Outcome
Title Change From Baseline in Plasminogen Activator Inhibitor-1 Antigen (AG) Levels at Endpoint
Hide Description Change from baseline in plasminogen activator inhibitor-1 AG levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: microgram per liter (mcg/L)
-0.483  (2.9839) -1.739  (4.6587) -1.456  (4.6448)
34.Secondary Outcome
Title Change From Baseline in Cytokeratin-18 Levels at Endpoint
Hide Description Change from baseline in cytokeratin-18 (M30 and M65) levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: picomole per liter (pmol/L)
Cytokeratin-18 M30 26.12  (472.247) 163.33  (499.500) 17.93  (307.531)
Cytokeratin-18 M65 114.31  (627.068) 238.97  (611.520) -53.16  (131.934)
35.Secondary Outcome
Title Change From Baseline in Autotaxin Levels at Endpoint
Hide Description Change from baseline in autotaxin levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: mcg/L
4.6  (156.92) 49.9  (77.25) 35.1  (161.58)
36.Secondary Outcome
Title C-reactive Protein Level at Endpoint
Hide Description C-reactive protein level at endpoint was reported.
Time Frame Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Geometric Mean (95% Confidence Interval)
Unit of Measure: milligram per liter (mg/L)
2.74
(1.81 to 4.14)
2.84
(1.68 to 4.78)
4.01
(2.52 to 6.37)
37.Secondary Outcome
Title Change From Baseline in Haptoglobin Levels at Endpoint
Hide Description Change from baseline in haptoglobin levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: g/L
-0.265  (0.4271) -0.254  (0.1088) 0.025  (0.2244)
38.Secondary Outcome
Title Change From Baseline in Fibrinogen Levels at Endpoint
Hide Description Change from baseline in fibrinogen levels at endpoint was reported.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: g/L
-0.865  (0.9472) -0.452  (0.5780) -0.072  (1.0936)
39.Secondary Outcome
Title Change From Baseline in 5D-Itch Scale Total Score
Hide Description 5 dimensional (5D)-Itch Scale is a reliable, multidimensional measure of itching that has been validated in participants with chronic pruritus to detect changes over time. It consists of 5 domains: duration, degree, direction, disability, and distribution. The duration, degree and direction domains each include one item, while the disability domain has four items (sleep, leisure/social, housework/errands, work/school). All items of the first four domains were measured on a 5-point Likert scale. The distribution domain included 16 potential locations of itch, including 15 body part items (head/scalp, soles, face, palms, chest, abdomen, back, buttocks, thighs, lower legs, tops of feet/toes, tops of hands/fingers, upper arms, groin, forearms) and one point of contact with clothing or bandages. Scores of each of five domains are achieved separately and then summed together to obtain a total 5-D score. 5-D scores can potentially range between 5 (no pruritus) and 25 (most severe pruritus).
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint. Here 'N' (overall number of participants analyzed) signifies number of participants evaluable for this outcome measure.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 12 15
Mean (Standard Deviation)
Unit of Measure: Units on a scale
-2.1  (5.15) -0.1  (2.19) 0.8  (4.93)
40.Secondary Outcome
Title Change From Baseline in Pruritus as Assessed by Visual Analogue Scale (VAS) Total Score
Hide Description The VAS is a reliable and validated method of pruritus assessment. The VAS is adequate in assessing the severity of the symptom; it does not take into account other aspects of pruritus, such as the relative impact of pruritus on quality of life. The VAS, for pruritus assessment, requires the participant to use abstract thought processes to convert their itch severity to a mark on a continuum. A participant draws a line anywhere on the scale ranging from 0 to 10 (where 0 represents ‘no itching’ and 10 represents ‘worst possible itching’) that best represents the severity of participant’s itching and the scoring involves manual measuring of the mark with a ruler on range of 0 to 100 millimeter (mm). Higher scores indicate worse itching.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint. Here 'N' (number of participants analyzed) signifies number of participants evaluable for this outcome measure.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 14 14 15
Mean (Standard Deviation)
Unit of Measure: Units on a scale
-4.4  (22.80) -4.7  (11.81) 9.3  (35.93)
41.Secondary Outcome
Title Change From Baseline in Primary Biliary Cholangitis -40 (PBC-40) Quality of Life (QoL) Questionnaire Scores
Hide Description PBC-40 QoL Questionnaire is a patient-derived, disease-specific QoL measure developed and validated for use in PBC. It consists of 9 domains with total 40 questions as: 1) digestion and diet (questions 1-3, total score range: 3-15); 2) experiences (questions 4-7, total score range: 4-20); 3) itching (questions 8-10, total score range: 3-15); 4) fatigue (questions 11-18, total score range: 8-40); 5) effort and planning (questions 19-21, total score range: 3-15); 6) memory and concentration (questions 22-27, total score range: 6-30); 7) affects you as a person (questions 28-33, total score range: 6-30); 8) affects your social life (questions 34-37, total score range: 4-20); 9) overall impact on your life (questions 38-40, total score range: 3-15). PBC-40 QoL Questionnaire has 40 questions, each scored on scale of 1-5 (1 = least impact, 5 = greatest impact). For each domain, scoring involved summing individual question response scores. Higher scores indicate poorer quality of life.
Time Frame Baseline, Week 12 (Endpoint)
Hide Outcome Measure Data
Hide Analysis Population Description
The mITT analysis set included all randomized participants who received at least one study drug dose with available baseline value and at least one post baseline value for the primary endpoint.
Arm/Group Title Elafibranor 80 mg Elafibranor 120 mg Placebo
Hide Arm/Group Description:
Participants received orally a dose of 80 milligram (mg) elafibranor as tablets once daily for 12 weeks.
Participants received orally a dose of 120 mg elafibranor as tablets once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 14 15
Mean (Standard Deviation)
Unit of Measure: Units on a scale
Digestion and Diet -0.3  (2.74) -0.6  (2.24) -0.6  (3.00)
Experiences 0.6  (2.64) -1.3  (1.77) -0.7  (3.69)
Itching -0.9  (6.19) -4.1  (6.56) 2.1  (5.78)
Fatigue -1.9  (4.10) -1.4  (2.71) -1.5  (5.04)
Effort and Planning -0.9  (2.03) -0.8  (1.31) -0.9  (1.98)
Memory and concentration 0.1  (3.26) -1.5  (3.33) -0.5  (3.25)
Affecting you as a Person -1.8  (3.78) -2.5  (2.85) -1.3  (3.22)
Affects your Social Life 1.3  (3.08) 0.0  (1.47) 1.4  (4.79)
Impact on your Life -0.1  (3.04) 0.6  (0.63) -1.0  (3.30)
42.Secondary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events
Hide Description An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical (investigational) product and which does not necessarily have to have a causal relationship with this treatment. A Serious adverse event (SAE) is any untoward medical occurrence that at any dose: results in death, is life-threatening, requires inpatient hospitalization/prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is another medically important condition. TEAEs is defined as (1) it is not present when active phase of study (time of first dose) begins and is not a chronic condition that is part of patient’s medical history, or it is present at start of active phase or as part of patient’s medical history, but severity/frequency increases during active phase.
Time Frame Up to Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Set included all randomized participants who were administered at least one dose of study medication.
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description:
Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks.
Participants received elafibranor 120 mg tablets orally once daily for 12 weeks.
Participants received matching placebo tablets orally once daily for 12 weeks.
Overall Number of Participants Analyzed 15 15 15
Measure Type: Count of Participants
Unit of Measure: Participants
TEAEs
12
  80.0%
13
  86.7%
12
  80.0%
Serious TEAEs
0
   0.0%
2
  13.3%
0
   0.0%
Time Frame Up to 16 Weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Elafibranor 80mg Elafibranor 120mg Placebo
Hide Arm/Group Description Participants received elafibranor 80 milligram (mg) tablets orally once daily for 12 weeks. Participants received elafibranor 120 mg tablets orally once daily for 12 weeks. Participants received matching placebo tablets orally once daily for 12 weeks.
All-Cause Mortality
Elafibranor 80mg Elafibranor 120mg Placebo
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   13/15 (86.67%)      13/15 (86.67%)      12/15 (80.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Elafibranor 80mg Elafibranor 120mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/15 (0.00%)      2/15 (13.33%)      0/15 (0.00%)    
Hepatobiliary disorders       
Autoimmune Hepatitis  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Injury, poisoning and procedural complications       
Post Procedural Stroke  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Nervous system disorders       
Ischaemic Stroke  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
1
Term from vocabulary, MedDra 20.0
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Elafibranor 80mg Elafibranor 120mg Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   12/15 (80.00%)      13/15 (86.67%)      12/15 (80.00%)    
Ear and labyrinth disorders       
Vertigo  1  1/15 (6.67%)  2 0/15 (0.00%)  0 0/15 (0.00%)  0
Eye disorders       
Dry Eye  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Eye ulcer  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Scleral Haemorrhage  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Gastrointestinal disorders       
Abdominal Distension  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Abdominal Pain  1  1/15 (6.67%)  1 1/15 (6.67%)  1 0/15 (0.00%)  0
Abdominal Pain Upper  1  0/15 (0.00%)  0 1/15 (6.67%)  1 1/15 (6.67%)  1
Constipation  1  0/15 (0.00%)  0 1/15 (6.67%)  1 1/15 (6.67%)  1
Diarrhoea  1  0/15 (0.00%)  0 2/15 (13.33%)  2 2/15 (13.33%)  2
Dry Mouth  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Dyspepsia  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Nausea  1  0/15 (0.00%)  0 3/15 (20.00%)  3 1/15 (6.67%)  1
Rectal Haemorrhage  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
General disorders       
Fatigue  1  1/15 (6.67%)  1 3/15 (20.00%)  3 0/15 (0.00%)  0
Influenza Like Illness  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Local Swelling  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Peripheral Swelling  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Pyrexia  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Immune system disorders       
Hypersensitivity  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Infections and infestations       
Bronchitis  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Escherichia Urinary Tract Infection  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Gastroenteritis Viral  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Influenza  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Labyrinthitis  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Otitis Externa  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Sinusitis  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Urinary Tract Infection  1  1/15 (6.67%)  1 3/15 (20.00%)  3 0/15 (0.00%)  0
Viral Upper Respiratory Tract Infection  1  3/15 (20.00%)  3 0/15 (0.00%)  0 2/15 (13.33%)  2
Vulvovaginal Candidiasis  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Injury, poisoning and procedural complications       
Post-traumatic Neck Syndrome  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Investigations       
Blood Bilirubin Increased  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Blood Cholesterol Increased  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Blood Urine Present  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Cystoscopy  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Electrocardiogram Abnormal  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Gamma-glutamyltransferase Increased  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Liver Palpable  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Transaminases Increased  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Urine Albumin/Creatinine Ratio Increased  1  0/15 (0.00%)  0 1/15 (6.67%)  2 0/15 (0.00%)  0
Urobilinogen Urine Increased  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
White Blood Cells Urine  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Metabolism and nutrition disorders       
Decreased Appetite  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Musculoskeletal and connective tissue disorders       
Back Pain  1  1/15 (6.67%)  1 0/15 (0.00%)  0 1/15 (6.67%)  1
Bone Pain  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Musculoskeletal Discomfort  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Pain in Extremity  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Nervous system disorders       
Aphasia  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Cerebral Amyloid Angiopathy  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Dizziness  1  2/15 (13.33%)  2 0/15 (0.00%)  0 1/15 (6.67%)  1
Dysgeusia  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Headache  1  2/15 (13.33%)  3 2/15 (13.33%)  2 1/15 (6.67%)  1
Lumbar Radiculopathy  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Psychiatric disorders       
Insomnia  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Sleep Disorder  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Renal and urinary disorders       
Albuminuria  1  0/15 (0.00%)  0 1/15 (6.67%)  3 0/15 (0.00%)  0
Chromaturia  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Nephrolithiasis  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Nitrituria  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Polyuria  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Proteinuria  1  0/15 (0.00%)  0 1/15 (6.67%)  2 0/15 (0.00%)  0
Renal Colic  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Renal Pain  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Respiratory, thoracic and mediastinal disorders       
Chronic Obstructive Pulmonary Disease  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  2
Cough  1  0/15 (0.00%)  0 1/15 (6.67%)  1 0/15 (0.00%)  0
Skin and subcutaneous tissue disorders       
Alopecia  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Photosensitivity Reaction  1  0/15 (0.00%)  0 0/15 (0.00%)  0 1/15 (6.67%)  1
Pruritus  1  3/15 (20.00%)  3 3/15 (20.00%)  3 2/15 (13.33%)  3
Skin Disorder  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
Surgical and medical procedures       
Stent Removal  1  1/15 (6.67%)  1 0/15 (0.00%)  0 0/15 (0.00%)  0
1
Term from vocabulary, MedDra 20.0
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor retains exclusive ownership of all data, results, reports, findings, discoveries, and any other information collected during this study; these may not be published, given, or disclosed, either in part or in whole, by the Investigator or by any person under his/her authority to any third party without the prior express consent of the Sponsor.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Head
Organization: Genfit SA
Phone: +33320164000
EMail: clinicaltrial@genfit.com
Layout table for additonal information
Responsible Party: Genfit
ClinicalTrials.gov Identifier: NCT03124108     History of Changes
Other Study ID Numbers: GFT505B-216-1
2016-003817-80 ( EudraCT Number )
First Submitted: March 28, 2017
First Posted: April 21, 2017
Results First Submitted: August 16, 2019
Results First Posted: September 24, 2019
Last Update Posted: September 24, 2019