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

• Sponsor: Genfit (Industry)
• Overall Status: Completed
• CT.gov ID: NCT03124108
• Collaborator: (none)
• Enrollment: 45
• Locations: 24
• Arms: 3
• Actual Duration (Months): 18.9
• Patients Per Site: 1.9
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Primary Biliary Cholangitis (PBC)	Drug: Elafibranor 80 mg; Drug: Elafibranor 120 mg; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• Actual Enrollment: 45 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of Elafibranor at Doses of 80 mg and 120mg After 12 Weeks of Treatment in Patients With Primary Biliary Cholangitis and Inadequate Response to Ursodeoxycholic Acid
• Actual Study Start Date: Apr 5, 2017
• Actual Primary Completion Date: Oct 31, 2018
• Actual Study Completion Date: Oct 31, 2018

Arms and Interventions

Arm	Intervention/Treatment
Placebo Comparator: Placebo Study subjects will take two tablets per day orally before breakfast with a glass of water each morning	Drug: Placebo; Two coated tablets daily for 12 weeks
Active Comparator: Elafibranor 80 mg Study subjects will take two tablets per day orally before breakfast with a glass of water each morning	Drug: Elafibranor 80 mg; Two coated tablets daily for 12 weeks; Other Names: GFT505
Active Comparator: Elafibranor 120 mg Study subjects will take two tablets per day orally before breakfast with a glass of water each morning	Drug: Elafibranor 120 mg; Two coated tablets daily for 12 weeks; Other Names: GFT505

Outcome Measures

Primary Outcome Measures

1. Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) Levels at Week 12 (Endpoint) [Baseline, 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.

Secondary Outcome Measures

1. 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 Less than [<] 1.67 * ULN at endpoint, Total BIL within normal limits at endpoint, and > 15% ALP reduction from baseline to Endpoint) was reported.

2. 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 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.

3. 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 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.

4. Percentage of Participants With Response Based on PARIS II Risk Score at Endpoint [At Week 12 (Endpoint)]

   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.

5. Percentage of Participants With Response Based on Toronto I Risk Score at Endpoint [At Week 12 (Endpoint)]

   Percentage of participants with response based on Toronto I risk score was defined as ALP <= 1.67 *ULN.

6. Percentage of Participants With Response Based on Toronto II Risk Score at Endpoint [At Week 12 (Endpoint)]

   Percentage of participants with response based on Toronto II risk scores was defined as ALP <= 1.75 * ULN.

7. Median Percentage Risk as Assessed by United Kingdom-Primary Biliary Cholangitis (UK-PBC) Risk Total Score at Endpoint [At Week 12 (Endpoint)]

   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.

8. Percentage of Participants With Response Defined by 10, 20 and 40 Percent Reduction in Alkaline Phosphatase [At Week 12 (Endpoint)]

   Percentage of participants with response (defined by at least 10%, 20%, and 40% decrease in ALP from baseline to Endpoint) reported.

9. Percentage of Participants With Response Defined by Normalized Alkaline Phosphatase Levels at Endpoint [At Week 12 (Endpoint)]

   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.

10. Percentage of Participants With Response Defined by Normalized Bilirubin (BIL) at Endpoint [At Week 12 (Endpoint)]

    The response was defined by normalized BIL levels (BIL ULN <1.20 milligram per deciliter [mg/dL]) at endpoint.

11. Percentage of Participants With Response Defined by Normalized Albumin (ALB) Levels at Endpoint [At Week 12 (Endpoint)]

    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.

12. Change From Baseline in Alanine Aminotransferase (ALT) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in ALT levels at endpoint was reported.

13. Change From Baseline in Aspartate Aminotransferase (AST) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in AST levels at endpoint was reported.

14. Change From Baseline in Gamma-glutamyl Transferase (GGT) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in GGT levels at endpoint was reported.

15. Change From Baseline in 5 Prime (') Nucleotidase Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    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.

16. Change From Baseline in Total Bilirubin (BIL) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in total BIL levels at endpoint was reported.

17. Change From Baseline in Conjugated Bilirubin Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in conjugated bilirubin levels at endpoint was reported.

18. Change From Baseline in Albumin Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in albumin levels at endpoint was reported.

19. Change From Baseline in Cholesterol Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in cholesterol levels at endpoints was reported.

20. Change From Baseline in Low-density Lipoprotein (LDL) Cholesterol Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in LDL-cholesterol at endpoint was reported.

21. Change From Baseline in High-density Lipoprotein (HDL) Cholesterol Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in HDL-cholesterol levels at endpoint was reported.

22. Change From Baseline in Triglycerides Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in triglycerides levels at endpoint was reported.

23. Change From Baseline in Total Free Bile Acid Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in total free bile acid levels at endpoint was reported.

24. Change From Baseline in Total Conjugated Bile Acid Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in total conjugated bile acid levels at endpoint was reported.

25. Change From Baseline in Total Bile Acid Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in total bile acid levels at endpoint was reported.

26. Change From Baseline in 7 Alpha-hydroxy-4-cholesten-3-one Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in 7 alpha-hydroxy-4-cholesten-3-one levels at endpoint was reported.

27. Change From Baseline in Fibroblast Growth Factor-19 Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in fibroblast growth factor-19 levels at endpoint was reported.

28. Change From Baseline in Immunoglobulin M (IgM) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in IgM levels at endpoint was reported.

29. Change From Baseline in Tumor Necrosis Factor Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in tumor necrosis factor levels at endpoint was reported.

30. Change From Baseline in Transforming Growth Factor Beta Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in transforming growth factor beta levels at endpoint was reported,

31. Change From Baseline in Interleukin 6 Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in interleukin 6 levels at endpoint was reported.

32. Change From Baseline in Plasminogen Activator Inhibitor-1 Antigen (AG) Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in plasminogen activator inhibitor-1 AG levels at endpoint was reported.

33. Change From Baseline in Cytokeratin-18 Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in cytokeratin-18 (M30 and M65) levels at endpoint was reported.

34. Change From Baseline in Autotaxin Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in autotaxin levels at endpoint was reported.

35. C-reactive Protein Level at Endpoint [Week 12 (Endpoint)]

    C-reactive protein level at endpoint was reported.

36. Change From Baseline in Haptoglobin Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in haptoglobin levels at endpoint was reported.

37. Change From Baseline in Fibrinogen Levels at Endpoint [Baseline, Week 12 (Endpoint)]

    Change from baseline in fibrinogen levels at endpoint was reported.

38. Change From Baseline in 5D-Itch Scale Total Score [Baseline, Week 12 (Endpoint)]

    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).

39. Change From Baseline in Pruritus as Assessed by Visual Analogue Scale (VAS) Total Score [Baseline, Week 12 (Endpoint)]

    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.

40. Change From Baseline in Primary Biliary Cholangitis -40 (PBC-40) Quality of Life (QoL) Questionnaire Scores [Baseline, Week 12 (Endpoint)]

    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.

41. Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events [Up to Week 12]

    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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 75 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

1. ALP >= 1.67x upper limit of normal (ULN)

2. Taking UDCA for at least 12 months (stable dose for ≥ 6 months) prior to screening visit

3. 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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic in Arizona	Phoenix	Arizona	United States	85054
2	Schiff Center for Liver Diseases	Miami	Florida	United States	33136
3	Cleveland Clinic Florida	Weston	Florida	United States	33331
4	Piedmont Research Institute	Atlanta	Georgia	United States	30309
5	Massachusetts General Hospital	Boston	Massachusetts	United States	02114
6	Beth Israel Deaconess Medical Center	Boston	Massachusetts	United States	02215
7	Northwell Health Institution	Manhasset	New York	United States	11030
8	Asheville Gastroenterology Associates	Asheville	North Carolina	United States	28801
9	UT Southwestern Medical Center	Dallas	Texas	United States	75390
10	Baylor College of Medicine	Houston	Texas	United States	77030
11	University of Virginia	Charlottesville	Virginia	United States	22903
12	Virginia Commonwealth University	Richmond	Virginia	United States	23298
13	Swedish Medical Center	Seattle	Washington	United States	98122
14	Hopital Saint-Antoine	Paris		France	75012
15	University Hospital Frankfurt	Frankfurt		Germany	60590
16	Clinic for Gastroenterology and Hepatology	Koln		Germany	50937
17	Johannes Gutenberg University	Mainz		Germany	55131
18	Liver Unit, University of Barcelona	Barcelona		Spain	08036
19	Hospital De La Sant Creu St. Pau	Barcelona		Spain	08041
20	University of Birmingham Centre for Liver Research	Birmingham		United Kingdom	B15 2TT
21	Addenbrooke's Hospital	Cambridge		United Kingdom	CB2 0QQ
22	The Royal Liverpool University Hospital	Liverpool		United Kingdom	L7 8XP
23	King's College Hospital	London		United Kingdom	SE5 9RS
24	The Newcastle Upon Tyne Hosptials NHS Foundation Trust	Newcastle upon Tyne		United Kingdom	NE7 7DN

Sponsors and Collaborators

• Genfit

Investigators

• Study Director: Clinical Head, Genfit

Study Documents (Full-Text)

• Study Protocol - Dec 4, 2017
• Statistical Analysis Plan - Nov 11, 2018

More Information

Publications

None provided.

• Responsible Party: Genfit
• ClinicalTrials.gov Identifier: NCT03124108
• Other Study ID Numbers: GFT505B-216-1; 2016-003817-80
• First Posted: Apr 21, 2017
• Last Update Posted: Sep 24, 2019
• Last Verified: Sep 1, 2019

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Genfit: Elafibranor; Primary Biliary Cholangitis; Alkaline phosphatase
• Additional relevant MeSH terms: Cholangitis; Liver Cirrhosis, Biliary

Study Results

Participant Flow

Pre-assignment Detail	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
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

Baseline Characteristics

Arm/Group Title	Elafibranor 80mg	Elafibranor 120mg	Placebo	Total
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 Participants	15	15	15	45
Age (Count of Participants)
<=18 years	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 (Years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [Years]	56.5 (8.7)	60.4 (6.9)	60.5 (8.6)	59.1 (8.2)
Sex: Female, Male (Count of Participants)
Female	14 93.3%	15 100%	14 93.3%	43 95.6%
Male	1 6.7%	0 0%	1 6.7%	2 4.4%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	3 20%	2 13.3%	5 33.3%	10 22.2%
Not Hispanic or Latino	12 80%	13 86.7%	10 66.7%	35 77.8%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	1 6.7%	1 2.2%
White	15 100%	15 100%	14 93.3%	44 97.8%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [Percent change]	-48.264 (14.7676)	-40.640 (17.3624)	3.190 (14.8059)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Elafibranor 80mg, Placebo
	Comments
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<0.001
	Comments
	Method	ANCOVA
	Comments
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

Statistical Analysis 2

Statistical Analysis Overview	Comparison Group Selection	Elafibranor 120mg, Placebo
	Comments
	Type of Statistical Test	Superiority
	Comments
Statistical Test of Hypothesis	p-Value	<0.001
	Comments
	Method	ANCOVA
	Comments
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

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)
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	66.7 444.7%	78.6 524%	6.7 44.7%

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)
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	73.3 488.7%	42.9 286%	0 0%

4. Secondary Outcome

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

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	80.0 533.3%	78.6 524%	53.3 355.3%

5. Secondary Outcome

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

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	53.3 355.3%	50.0 333.3%	0 0%

6. Secondary Outcome

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

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	66.7 444.7%	78.6 524%	6.7 44.7%

7. Secondary Outcome

Title	Percentage of Participants With Response Based on Toronto II Risk Score at Endpoint
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	66.7 444.7%	78.6 524%	6.7 44.7%

8. Secondary Outcome

Title	Median Percentage Risk as Assessed by United Kingdom-Primary Biliary Cholangitis (UK-PBC) Risk Total Score at Endpoint
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
5 year risk at Week 12	0.80	0.95	1.30
10 year risk at Week 12	2.60	3.05	4.40
15 year risk at Week 12	4.70	5.55	8.00

9. Secondary Outcome

Title	Percentage of Participants With Response Defined by 10, 20 and 40 Percent Reduction in Alkaline Phosphatase
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
10 Percent Reduction	93.3 622%	92.9 619.3%	13.3 88.7%
20 Percent Reduction	93.3 622%	92.9 619.3%	6.7 44.7%
40 Percent Reduction	86.7 578%	57.1 380.7%	0 0%

10. Secondary Outcome

Title	Percentage of Participants With Response Defined by Normalized Alkaline Phosphatase Levels at Endpoint
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	13.3 88.7%	21.4 142.7%	0 0%

11. Secondary Outcome

Title	Percentage of Participants With Response Defined by Normalized Bilirubin (BIL) at Endpoint
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	86.7 578%	92.9 619.3%	93.3 622%

12. Secondary Outcome

Title	Percentage of Participants With Response Defined by Normalized Albumin (ALB) Levels at Endpoint
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Number [Percentage of participants]	100 666.7%	100 666.7%	100 666.7%

13. Secondary Outcome

Title	Change From Baseline in Alanine Aminotransferase (ALT) Levels at Endpoint
Description	Change from baseline in ALT levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in AST levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in GGT levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in total BIL levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in conjugated bilirubin levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in albumin levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in cholesterol levels at endpoints was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in LDL-cholesterol at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in HDL-cholesterol levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in triglycerides levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in total free bile acid levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in total conjugated bile acid levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in total bile acid levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in 7 alpha-hydroxy-4-cholesten-3-one levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in fibroblast growth factor-19 levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in IgM levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in tumor necrosis factor levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in transforming growth factor beta levels at endpoint was reported,
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in interleukin 6 levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in plasminogen activator inhibitor-1 AG levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in cytokeratin-18 (M30 and M65) levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
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
Description	Change from baseline in autotaxin levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [mcg/L]	4.6 (156.92)	49.9 (77.25)	35.1 (161.58)

36. Secondary Outcome

Title	C-reactive Protein Level at Endpoint
Description	C-reactive protein level at endpoint was reported.
Time Frame	Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Geometric Mean (95% Confidence Interval) [milligram per liter (mg/L)]	2.74	2.84	4.01

37. Secondary Outcome

Title	Change From Baseline in Haptoglobin Levels at Endpoint
Description	Change from baseline in haptoglobin levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
Description	Change from baseline in fibrinogen levels at endpoint was reported.
Time Frame	Baseline, Week 12 (Endpoint)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
Mean (Standard Deviation) [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
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)

Outcome Measure Data

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
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.
Measure Participants	15	12	15
Mean (Standard Deviation) [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
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)

Outcome Measure Data

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
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.
Measure Participants	14	14	15
Mean (Standard Deviation) [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
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)

Outcome Measure Data

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
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.
Measure Participants	15	14	15
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
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

Outcome Measure Data

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
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.
Measure Participants	15	15	15
TEAEs	12 80%	13 86.7%	12 80%
Serious TEAEs	0 0%	2 13.3%	0 0%

Adverse Events

Time Frame	Up to 16 Weeks
Arm/Group Title	Elafibranor 80mg		Elafibranor 120mg		Placebo
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 (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	13/15 (86.7%)		13/15 (86.7%)		12/15 (80%)
Serious Adverse Events
	Elafibranor 80mg		Elafibranor 120mg		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/15 (0%)		2/15 (13.3%)		0/15 (0%)
Hepatobiliary disorders
Autoimmune Hepatitis	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Injury, poisoning and procedural complications
Post Procedural Stroke	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Nervous system disorders
Ischaemic Stroke	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Other (Not Including Serious) Adverse Events
	Elafibranor 80mg		Elafibranor 120mg		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	12/15 (80%)		13/15 (86.7%)		12/15 (80%)
Ear and labyrinth disorders
Vertigo	1/15 (6.7%)	2	0/15 (0%)	0	0/15 (0%)	0
Eye disorders
Dry Eye	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Eye ulcer	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Scleral Haemorrhage	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Gastrointestinal disorders
Abdominal Distension	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Abdominal Pain	1/15 (6.7%)	1	1/15 (6.7%)	1	0/15 (0%)	0
Abdominal Pain Upper	0/15 (0%)	0	1/15 (6.7%)	1	1/15 (6.7%)	1
Constipation	0/15 (0%)	0	1/15 (6.7%)	1	1/15 (6.7%)	1
Diarrhoea	0/15 (0%)	0	2/15 (13.3%)	2	2/15 (13.3%)	2
Dry Mouth	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Dyspepsia	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Nausea	0/15 (0%)	0	3/15 (20%)	3	1/15 (6.7%)	1
Rectal Haemorrhage	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
General disorders
Fatigue	1/15 (6.7%)	1	3/15 (20%)	3	0/15 (0%)	0
Influenza Like Illness	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Local Swelling	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Peripheral Swelling	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Pyrexia	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Immune system disorders
Hypersensitivity	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Infections and infestations
Bronchitis	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Escherichia Urinary Tract Infection	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Gastroenteritis Viral	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Influenza	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Labyrinthitis	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Otitis Externa	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Sinusitis	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Urinary Tract Infection	1/15 (6.7%)	1	3/15 (20%)	3	0/15 (0%)	0
Viral Upper Respiratory Tract Infection	3/15 (20%)	3	0/15 (0%)	0	2/15 (13.3%)	2
Vulvovaginal Candidiasis	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Injury, poisoning and procedural complications
Post-traumatic Neck Syndrome	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Investigations
Blood Bilirubin Increased	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Blood Cholesterol Increased	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Blood Urine Present	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Cystoscopy	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Electrocardiogram Abnormal	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Gamma-glutamyltransferase Increased	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Liver Palpable	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Transaminases Increased	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Urine Albumin/Creatinine Ratio Increased	0/15 (0%)	0	1/15 (6.7%)	2	0/15 (0%)	0
Urobilinogen Urine Increased	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
White Blood Cells Urine	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Metabolism and nutrition disorders
Decreased Appetite	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Musculoskeletal and connective tissue disorders
Back Pain	1/15 (6.7%)	1	0/15 (0%)	0	1/15 (6.7%)	1
Bone Pain	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Musculoskeletal Discomfort	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Pain in Extremity	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Nervous system disorders
Aphasia	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Cerebral Amyloid Angiopathy	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Dizziness	2/15 (13.3%)	2	0/15 (0%)	0	1/15 (6.7%)	1
Dysgeusia	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Headache	2/15 (13.3%)	3	2/15 (13.3%)	2	1/15 (6.7%)	1
Lumbar Radiculopathy	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Psychiatric disorders
Insomnia	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Sleep Disorder	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Renal and urinary disorders
Albuminuria	0/15 (0%)	0	1/15 (6.7%)	3	0/15 (0%)	0
Chromaturia	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Nephrolithiasis	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Nitrituria	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Polyuria	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Proteinuria	0/15 (0%)	0	1/15 (6.7%)	2	0/15 (0%)	0
Renal Colic	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Renal Pain	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary Disease	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	2
Cough	0/15 (0%)	0	1/15 (6.7%)	1	0/15 (0%)	0
Skin and subcutaneous tissue disorders
Alopecia	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Photosensitivity Reaction	0/15 (0%)	0	0/15 (0%)	0	1/15 (6.7%)	1
Pruritus	3/15 (20%)	3	3/15 (20%)	3	2/15 (13.3%)	3
Skin Disorder	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0
Surgical and medical procedures
Stent Removal	1/15 (6.7%)	1	0/15 (0%)	0	0/15 (0%)	0

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

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.