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A Multi-part, Double Blind Study to Assess Safety, Tolerability and Efficacy of Tropifexor (LJN452) in PBC Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02516605
Recruitment Status : Completed
First Posted : August 6, 2015
Results First Posted : November 13, 2019
Last Update Posted : January 5, 2021
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Triple (Participant, Care Provider, Investigator);   Primary Purpose: Treatment
Condition Primary Biliary Cholangitis
Interventions Drug: Part 1: LJN452
Drug: Part 1: Placebo
Drug: Part 2: LJN452 Dose level 1
Drug: Part 2: Placebo
Drug: Part 2: LJN452 Dose level 2
Enrollment 61
Recruitment Details  
Pre-assignment Details This study comprised of an escalating multiple dose design in PBC patients with incomplete biochemical response to, but still taking, ursodeoxycholic acid (UDCA).
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description Tropifexor 0.03 mg daily for 28 days Tropifexor 0.06 mg daily for 28 days Tropifexor 0.09 mg daily for 28 days Tropifexor 0.15 mg daily for 28 days Tropifexor placebo daily for 28 days
Period Title: Overall Study
Started 11 9 12 8 21
Completed 11 9 12 7 20
Not Completed 0 0 0 1 1
Reason Not Completed
Protocol Violation             0             0             0             0             1
Withdrawal by Subject             0             0             0             1             0
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd Total
Hide Arm/Group Description Tropifexor 0.03 mg daily for 28 days Tropifexor 0.06 mg daily for 28 days Tropifexor 0.09 mg daily for 28 days Tropifexor 0.15 mg daily for 28 days Tropifexor placebo daily for 28 days Total of all reporting groups
Overall Number of Baseline Participants 11 9 12 8 21 61
Hide Baseline Analysis Population Description
The safety analysis set included all subjects who received any study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants 61 participants
58.6  (12.42) 57.9  (11.21) 53.6  (7.42) 57.4  (13.81) 53.7  (10.19) 55.7  (10.70)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants 61 participants
Female
11
 100.0%
7
  77.8%
12
 100.0%
8
 100.0%
21
 100.0%
59
  96.7%
Male
0
   0.0%
2
  22.2%
0
   0.0%
0
   0.0%
0
   0.0%
2
   3.3%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants 61 participants
Caucasian 10 8 12 8 19 57
Asian 0 0 0 0 1 1
Other 1 1 0 0 1 3
1.Primary Outcome
Title Fold Change in Serum Gamma-glutamyl Transferase (GGT)
Hide Description Fold change in serum gamma-glutamyl transferase (GGT) from baseline to Day 28
Time Frame Baseline to Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
The PD analysis set included all subjects with any available PD data and no protocol deviations with relevant impact on PD data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Pooled Placebo
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Placebo data from all cohorts were pooled.
Overall Number of Participants Analyzed 10 9 12 8 20
Mean (90% Confidence Interval)
Unit of Measure: fold-change
0.74
(0.61 to 0.90)
0.41
(0.33 to 0.50)
0.28
(0.23 to 0.33)
0.31
(0.24 to 0.40)
0.86
(0.75 to 0.99)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Pooled Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.293
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted fold change from baseline
Estimated Value 0.86
Confidence Interval (2-Sided) 90%
0.68 to 1.09
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Pooled Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted fold change from baseline
Estimated Value 0.47
Confidence Interval (2-Sided) 90%
0.37 to 0.60
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Pooled Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted fold change from baseline
Estimated Value 0.32
Confidence Interval (2-Sided) 90%
0.26 to 0.40
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Pooled Placebo
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter adjusted fold change from baseline
Estimated Value 0.36
Confidence Interval (2-Sided) 90%
0.27 to 0.47
Estimation Comments [Not Specified]
2.Primary Outcome
Title Blood Pressure
Hide Description Vital signs - Systolic Blood pressure
Time Frame Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: mm Hg
Screening Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
122.5  (12.21) 125.8  (11.79) 129.3  (14.42) 129.5  (17.42) 123.0  (15.40)
Baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
124.7  (17.66) 128.4  (13.83) 122.0  (18.18) 132.3  (18.09) 118.7  (12.76)
Day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
129.6  (20.48) 122.4  (20.24) 119.9  (11.84) 129.1  (25.12) 123.0  (21.84)
Day 7 Number Analyzed 11 participants 9 participants 12 participants 8 participants 20 participants
127.2  (20.18) 123.7  (13.04) 125.6  (14.22) 128.0  (26.58) 120.9  (19.57)
Day 14 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
122.4  (16.30) 124.6  (20.67) 127.1  (16.45) 127.3  (12.45) 118.7  (12.88)
Day 21 Number Analyzed 11 participants 9 participants 12 participants 6 participants 20 participants
121.5  (11.25) 126.3  (27.65) 123.9  (13.14) 124.0  (14.23) 124.5  (19.8)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 5 participants 20 participants
118.0  (11.22) 127.1  (28.55) 121.4  (12.82) 126.0  (7.71) 120.1  (22.38)
Day 56 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
121.4  (10.76) 125.4  (18.48) 121.5  (11.55) 129.1  (26.45) 123.8  (19.74)
Day 84 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
131.3  (16.04) 124.6  (15.77) 122.3  (7.70) 130.4  (25.51) 123.6  (19.09)
3.Primary Outcome
Title Pulse Rate
Hide Description Vital signs
Time Frame Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: bpm
Screening Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
68.6  (10.63) 61.2  (7.05) 75.3  (8.22) 72.8  (8.26) 69.3  (10.69)
Baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
64.3  (7.79) 64.1  (8.70) 70.8  (8.63) 74.4  (5.15) 66.3  (9.30)
Day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
67.7  (11.93) 64.2  (8.07) 69.7  (7.50) 74.1  (11.62) 67.6  (8.35)
Day 7 Number Analyzed 11 participants 9 participants 12 participants 8 participants 20 participants
65.2  (9.98) 66.8  (11.31) 70.1  (11.64) 73.5  (8.33) 65.2  (8.92)
Day 14 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
62.9  (10.03) 64.8  (8.09) 70.5  (14.16) 75.1  (6.74) 66.1  (9.61)
Day 21 Number Analyzed 11 participants 9 participants 12 participants 6 participants 20 participants
65.5  (8.26) 65.8  (11.18) 73.2  (8.43) 72.5  (6.75) 67.7  (10.18)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 5 participants 20 participants
65.5  (10.83) 68.9  (10.60) 68.5  (9.01) 67.2  (3.42) 65.8  (9.29)
Day 56 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
66.8  (9.64) 67.7  (7.53) 69.7  (8.17) 69.7  (4.07) 68.1  (10.11)
Day 84 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
65.6  (10.24) 64.0  (9.11) 70.2  (8.14) 72.8  (8.12) 68.1  (9.82)
4.Primary Outcome
Title Body Temperature
Hide Description Vital signs
Time Frame Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: Celsius
Screening Number Analyzed 11 participants 9 participants 12 participants 8 participants 20 participants
36.605  (0.2813) 36.567  (0.2000) 36.424  (0.3429) 36.650  (0.1604) 36.460  (0.4500)
baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
36.582  (0.2960) 36.644  (0.2128) 36.398  (0.3385) 36.638  (0.1506) 36.376  (0.5118)
day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
36.436  (0.3139) 36.537  (0.3076) 36.482  (0.2636) 36.575  (0.1982) 36.390  (0.4867)
day 7 Number Analyzed 11 participants 9 participants 12 participants 8 participants 20 participants
36.582  (0.2040) 36.533  (0.1323) 36.258  (0.3965) 36.600  (0.1195) 36.345  (0.4639)
day 14 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
36.516  (0.1793) 36.444  (0.2007) 36.291  (0.4109) 36.500  (0.1826) 36.310  (0.4576)
day 21 Number Analyzed 11 participants 9 participants 12 participants 6 participants 20 participants
36.500  (0.2490) 36.267  (0.4637) 36.308  (0.3288) 36.433  (0.1966) 36.455  (0.4236)
day 28 Number Analyzed 11 participants 9 participants 12 participants 5 participants 20 participants
36.382  (0.2228) 36.444  (0.2128) 36.308  (0.3397) 36.520  (0.2950) 36.380  (0.4047)
day 56 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
36.527  (0.2102) 36.500  (0.2179) 36.350  (0.4602) 36.443  (0.3309) 36.285  (0.4246)
day 84 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
36.482  (0.2483) 36.500  (0.1500) 36.383  (0.2980) 36.638  (0.1506) 36.295  (0.4248)
5.Primary Outcome
Title ECG - Heart Rate
Hide Description Electrocardiogram (ECG)
Time Frame Screening, Baseline, day 1, day 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: bpm
Screening Number Analyzed 10 participants 9 participants 12 participants 8 participants 21 participants
65.8  (11.32) 61.8  (9.97) 67.3  (5.58) 67.8  (6.54) 63.4  (9.35)
Baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
60.5  (8.99) 61.4  (8.75) 64.6  (7.95) 67.9  (6.45) 63.8  (9.41)
Day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
61.5  (11.16) 63.4  (11.17) 63.9  (8.21) 66.9  (11.97) 63.5  (7.12)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 5 participants 20 participants
60.5  (11.39) 65.2  (11.95) 63.5  (9.95) 65.8  (4.55) 60.8  (7.03)
6.Primary Outcome
Title ECG Intervals - PR Interval
Hide Description Electrocardiogram (ECG)
Time Frame Screening, Baseline, day 1, day 28
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: msec
Screening Number Analyzed 10 participants 9 participants 12 participants 8 participants 21 participants
159.2  (27.34) 158.0  (23.71) 172.0  (24.12) 159.9  (17.24) 162.6  (19.03)
Baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
165.8  (20.81) 156.2  (18.99) 175.0  (34.96) 152.0  (22.21) 160.3  (19.77)
Day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
165.5  (21.76) 162.9  (27.06) 175.7  (28.29) 160.0  (20.32) 161.6  (20.28)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 5 participants 20 participants
164.9  (19.75) 157.1  (26.70) 180.4  (31.63) 155.4  (23.51) 160.2  (28.70)
7.Primary Outcome
Title Haemoglobin
Hide Description Hematology panel for safety laboratory assessments.
Time Frame Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Analysis Set
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8 21
Mean (Standard Deviation)
Unit of Measure: g/L
Screening Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
127.9  (9.24) 125.6  (11.70) 130.5  (9.73) 133.9  (12.70) 132.2  (8.81)
baseline Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
126.5  (8.58) 127.4  (11.85) 130.5  (11.90) 134.6  (13.24) 131.3  (8.02)
day 1 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
124.7  (8.21) 132.9  (10.05) 127.0  (9.03) 133.1  (11.37) 126.8  (7.63)
day 7 Number Analyzed 11 participants 9 participants 12 participants 8 participants 20 participants
124.0  (8.23) 128.8  (16.97) 132.4  (10.66) 134.0  (8.75) 128.6  (9.18)
day 14 Number Analyzed 11 participants 9 participants 12 participants 7 participants 20 participants
126.5  (9.83) 127.3  (12.64) 128.6  (7.82) 135.4  (13.50) 128.7  (10.84)
day 21 Number Analyzed 11 participants 9 participants 12 participants 6 participants 20 participants
127.5  (8.32) 128.0  (13.49) 130.5  (10.40) 134.2  (9.91) 128.8  (10.79)
day 28 Number Analyzed 10 participants 9 participants 12 participants 5 participants 20 participants
125.3  (9.18) 127.1  (14.67) 129.6  (10.02) 136.4  (13.50) 125.7  (9.71)
day 56 Number Analyzed 11 participants 8 participants 12 participants 5 participants 20 participants
126.7  (6.96) 125.8  (11.15) 128.6  (10.63) 133.6  (12.00) 129.3  (11.39)
day 84 Number Analyzed 11 participants 9 participants 12 participants 8 participants 21 participants
126.5  (8.78) 124.9  (9.75) 125.9  (11.19) 131.1  (10.91) 129.3  (9.24)
8.Secondary Outcome
Title Plasma PK Parameter - AUC 0-8h
Hide Description Tropifexor levels were determined in plasma using a validated LC-MS/MS method. AUC0-t=The area under the plasma concentration-time curve from time zero to time 't' where t is a defined time point after administration [mass x time / volume]
Time Frame Day 1, Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
The PK analysis set included all subjects with available PK data and no protocol deviations with relevant impact on PK data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Overall Number of Participants Analyzed 9 7 3 4
Mean (Standard Deviation)
Unit of Measure: hr*ng/mL
Day 1 Number Analyzed 7 participants 3 participants 0 participants 4 participants
4.98  (2.87) 12.1  (2.68) 24.5  (15.9)
Day 28 Number Analyzed 9 participants 7 participants 3 participants 3 participants
7.95  (4.21) 17.6  (5.30) 23.4  (8.70) 44.2  (25.8)
9.Secondary Outcome
Title Plasma PK Parameter - Cmax
Hide Description Tropifexor levels were determined in plasma using a validated LC-MS/MS method. Cmax=The observed maximum plasma concentration following drug administration [mass /volume]
Time Frame Day 1, Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
The PK analysis set included all subjects with available PK data and no protocol deviations with relevant impact on PK data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 10 participants 7 participants 12 participants 8 participants
1.04  (0.484) 1.80  (0.585) 2.37  (1.56) 4.84  (2.59)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 4 participants
1.25  (0.559) 2.55  (0.946) 4.30  (2.10) 6.37  (3.40)
10.Secondary Outcome
Title Plasma PK Parameter - Tmax
Hide Description Tropifexor levels were determined in plasma using a validated LC-MS/MS method. Tmax = The time to reach the maximum concentration after drug administration [time]
Time Frame Day 1, Day 28
Hide Outcome Measure Data
Hide Analysis Population Description
The PK analysis set included all subjects with available PK data and no protocol deviations with relevant impact on PK data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Overall Number of Participants Analyzed 11 9 12 8
Median (Inter-Quartile Range)
Unit of Measure: hr
Day 1 Number Analyzed 10 participants 7 participants 12 participants 8 participants
4.12
(2.00 to 8.00)
4.00
(3.70 to 6.00)
4.00
(0 to 7.83)
4.00
(4.00 to 4.18)
Day 28 Number Analyzed 11 participants 9 participants 12 participants 4 participants
4.08
(2.00 to 8.00)
4.00
(3.13 to 7.60)
4.00
(0 to 6.00)
5.00
(3.03 to 6.00)
11.Secondary Outcome
Title Changes From Baseline in Total PBC-40 Score
Hide Description Baseline is defined as the latest available predose value. The PBC-40 is a paper-based, patient-derived, disease-specific health-related quality of life (HRQOL) patient reported outcome (PRO) measure which was developed and validated for use in PBC patients (Jacoby et al 2005). It consists of 40 questions arranged in 6 domains with between 3 and 11 questions in each domain. Each question is scored from 1 to 5 in increasing order of severity. All questions within a domain are summed and all domain totals are summed to obtain a total score. The total score range is between a minimum of 40 and a maximum of 200. Higher scores represent a poorer quality of life. The median difference from baseline in total sum score for each treatment group is presented.
Time Frame Baseline, Day 28, Day 56, Day 84
Hide Outcome Measure Data
Hide Analysis Population Description
The PD analysis set included all subjects with any available PD data and no protocol deviations with relevant impact on PD data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days.
Tropifexor 0.09 mg daily for 28 days.
Tropifexor 0.15 mg daily for 28 days.
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 10 9 12 8 20
Median (Full Range)
Unit of Measure: units on a scale
Day 28 Number Analyzed 9 participants 9 participants 12 participants 5 participants 18 participants
2.0
(-10 to 7)
1.0
(-14 to 16)
3.5
(-7 to 13)
-1.0
(-5 to 43)
-2.0
(-23 to 26)
Day 56 Number Analyzed 9 participants 9 participants 12 participants 7 participants 18 participants
3.0
(-7 to 29)
3.0
(-15 to 23)
-2.5
(-15 to 16)
-3.0
(-28 to 3)
1.0
(-12 to 30)
Day 84 Number Analyzed 9 participants 9 participants 12 participants 8 participants 18 participants
-2.0
(-12 to 13)
-1.0
(-14 to 16)
-3.5
(-38 to 10)
-1.0
(-12 to 5)
1.5
(-38 to 30)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.898
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 1.0
Confidence Interval (2-Sided) 90%
-7.0 to 6.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.593
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 2.0
Confidence Interval (2-Sided) 90%
-4.0 to 10.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.509
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -3.0
Confidence Interval (2-Sided) 90%
-11.0 to 4.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.591
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 1.5
Confidence Interval (2-Sided) 90%
-5.0 to 7.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.702
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -2.0
Confidence Interval (2-Sided) 90%
-12.0 to 4.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.838
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -1.0
Confidence Interval (2-Sided) 90%
-10.0 to 8.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.297
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 4.0
Confidence Interval (2-Sided) 90%
-3.0 to 8.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.236
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -6.0
Confidence Interval (2-Sided) 90%
-14.0 to 1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.192
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -6.5
Confidence Interval (2-Sided) 90%
-15.0 to 1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.605
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 2.0
Confidence Interval (2-Sided) 90%
-2.0 to 9.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.037
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -11.0
Confidence Interval (2-Sided) 90%
-21.0 to -1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.397
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -3.5
Confidence Interval (2-Sided) 90%
-11.0 to 3.0
Estimation Comments [Not Specified]
12.Secondary Outcome
Title Change From Baseline in Itch Subdomain of PBC-40 Score
Hide Description Baseline is defined as the latest available predose value. The PBC-40 is a paper-based, patient-derived, disease-specific health-related quality of life (HRQOL) patient reported outcome (PRO) measure which was developed and validated for use in PBC patients (Jacoby et al 2005). It consists of 40 questions arranged in 6 domains with between 3 and 11 questions in each domain. Each question is scored from 1 to 5 in increasing order of severity. This dataset focuses on the itch subdomain which consists of 3 questions. These 3 questions within the itch subdomain are summed to obtain a total score for the itch subdomain. The total score range is between a minimum of 3 and a maximum of 15. Higher scores represent a poorer quality of life. The median change from baseline in total itch subdomain score in each treatment group is presented.
Time Frame Baseline, Day 28, Day 56, Day 84
Hide Outcome Measure Data
Hide Analysis Population Description
The PD analysis set included all subjects with any available PD data and no protocol deviations with relevant impact on PD data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days.
Tropifexor 0.09 mg daily for 28 days.
Tropifexor 0.15 mg daily for 28 days.
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 10 9 12 8 20
Median (Full Range)
Unit of Measure: UNITS ON A SCALE
Day 28 Number Analyzed 9 participants 9 participants 12 participants 5 participants 18 participants
1.0
(-2 to 7)
1.0
(0 to 2)
2.0
(-2 to 10)
2.0
(0 to 7)
0.0
(-4 to 9)
Day 56 Number Analyzed 9 participants 9 participants 12 participants 7 participants 18 participants
1.0
(-9 to 4)
1.0
(0 to 6)
0.5
(-3 to 5)
0.0
(0 to 1)
0.0
(-3 to 12)
Day 84 Number Analyzed 9 participants 9 participants 12 participants 8 participants 18 participants
0.0
(-6 to 1)
0.0
(-2 to 2)
0.0
(-4 to 8)
0.0
(-1 to 1)
0.0
(-3 to 8)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.342
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 1.0
Confidence Interval (2-Sided) 90%
-1.0 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.699
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-1.0 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.377
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -1.0
Confidence Interval (2-Sided) 90%
-3.0 to 0.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.132
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 1.0
Confidence Interval (2-Sided) 90%
0.0 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.292
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 1.0
Confidence Interval (2-Sided) 90%
0.0 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.979
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-2.0 to 1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.102
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 2.0
Confidence Interval (2-Sided) 90%
0.0 to 4.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.717
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-1.0 to 2.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 1.000
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-2.0 to 1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.142
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 2.0
Confidence Interval (2-Sided) 90%
0.0 to 5.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.975
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-1.0 to 1.0
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.602
Comments [Not Specified]
Method Wilcoxon rank-sum test
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.0
Confidence Interval (2-Sided) 90%
-2.0 to 1.0
Estimation Comments [Not Specified]
13.Secondary Outcome
Title Change From Baseline in Global Itch Visual Analogue Scale (VAS)
Hide Description Baseline is defined as the latest available predose value. The Global Itch Visual Analogue Scale, a 100 mm visual analogue scale (VAS), was used to assess the severity of patients itch (ranging from 0 = none at all to 100 = the worst imaginable itch). The score range is between a minimum of 0 and a maximum of 100. The score (distance in mm from left) on the VAS was recorded by the patient marking with a line and used to test for an effect of tropifexor over placebo. The mean change from baseline in itch VAS score in each treatment group is presented.
Time Frame Day 7, Day 14, Day 21, Day 28, Day 56, and Day 84
Hide Outcome Measure Data
Hide Analysis Population Description
The PD analysis set included all subjects with any available PD data and no protocol deviations with relevant impact on PD data.
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description:
Tropifexor 0.03 mg daily for 28 days.
Tropifexor 0.06 mg daily for 28 days
Tropifexor 0.09 mg daily for 28 days
Tropifexor 0.15 mg daily for 28 days
Tropifexor placebo daily for 28 days
Overall Number of Participants Analyzed 10 9 12 8 20
Mean (90% Confidence Interval)
Unit of Measure: mm
Day 7 Number Analyzed 9 participants 9 participants 12 participants 8 participants 20 participants
-2.04
(-13.84 to 9.75)
12.08
(0.33 to 23.83)
14.66
(4.16 to 25.16)
27.45
(15.00 to 39.89)
0.74
(-7.17 to 8.66)
Day 14 Number Analyzed 9 participants 9 participants 12 participants 7 participants 20 participants
-8.04
(-19.55 to 3.48)
13.77
(2.30 to 25.23)
6.51
(-3.76 to 16.78)
14.20
(1.26 to 27.13)
6.03
(-1.70 to 13.76)
Day 21 Number Analyzed 9 participants 9 participants 12 participants 6 participants 20 participants
10.53
(-1.66 to 22.72)
19.54
(7.40 to 31.68)
7.76
(-3.11 to 18.64)
8.65
(-5.90 to 23.20)
2.74
(-5.44 to 10.93)
Day 28 Number Analyzed 9 participants 9 participants 12 participants 5 participants 20 participants
8.62
(-4.24 to 21.48)
15.64
(2.83 to 28.44)
1.78
(-9.69 to 13.26)
10.50
(-5.52 to 26.51)
1.59
(-7.04 to 10.22)
Day 56 Number Analyzed 9 participants 9 participants 12 participants 7 participants 20 participants
-7.81
(-17.87 to 2.26)
5.69
(-4.33 to 15.72)
-6.38
(-15.35 to 2.59)
-3.64
(-14.58 to 7.30)
7.44
(0.68 to 14.20)
Day 84 Number Analyzed 9 participants 9 participants 12 participants 8 participants 20 participants
-5.14
(-17.15 to 6.88)
0.89
(-11.08 to 12.85)
-6.44
(-17.13 to 4.26)
-5.14
(-17.81 to 7.53)
11.79
(3.73 to 19.86)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.743
Comments [Not Specified]
Method ANCOVA
Comments Day 7
Method of Estimation Estimation Parameter Mean difference from baseline
Estimated Value -2.78
Confidence Interval (2-Sided) 90%
-16.91 to 11.34
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.436
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.093
Comments [Not Specified]
Method ANCOVA
Comments Day 14
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -14.07
Confidence Interval (2-Sided) 90%
-27.85 to -0.28
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.229
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.376
Comments [Not Specified]
Method ANCOVA
Comments Day 21
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 7.78
Confidence Interval (2-Sided) 90%
-6.81 to 22.38
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.710
Estimation Comments [Not Specified]
Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.448
Comments [Not Specified]
Method ANCOVA
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 7.03
Confidence Interval (2-Sided) 90%
-8.36 to 22.43
Parameter Dispersion
Type: Standard Error of the Mean
Value: 9.187
Estimation Comments [Not Specified]
Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.039
Comments [Not Specified]
Method ANCOVA
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -15.25
Confidence Interval (2-Sided) 90%
-27.30 to -3.19
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.192
Estimation Comments [Not Specified]
Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.054
Comments [Not Specified]
Method ANCOVA
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -16.93
Confidence Interval (2-Sided) 90%
-31.31 to -2.54
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.592
Estimation Comments [Not Specified]
Hide Statistical Analysis 7
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.185
Comments [Not Specified]
Method ANCOVA
Comments Day 7
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 11.34
Confidence Interval (2-Sided) 90%
-2.78 to 25.46
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.434
Estimation Comments [Not Specified]
Hide Statistical Analysis 8
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.351
Comments [Not Specified]
Method ANCOVA
Comments Day 14
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 7.74
Confidence Interval (2-Sided) 90%
-6.05 to 21.52
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.226
Estimation Comments [Not Specified]
Hide Statistical Analysis 9
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.03 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.059
Comments [Not Specified]
Method ANCOVA
Comments day 21
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 16.79
Confidence Interval (2-Sided) 90%
2.20 to 31.38
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.707
Estimation Comments [Not Specified]
Hide Statistical Analysis 10
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.132
Comments [Not Specified]
Method ANCOVA
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 14.05
Confidence Interval (2-Sided) 90%
-1.35 to 29.44
Parameter Dispersion
Type: Standard Error of the Mean
Value: 9.184
Estimation Comments [Not Specified]
Hide Statistical Analysis 11
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.809
Comments [Not Specified]
Method ANCOVA
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -1.75
Confidence Interval (2-Sided) 90%
-13.80 to 10.31
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.190
Estimation Comments [Not Specified]
Hide Statistical Analysis 12
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.06 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.210
Comments [Not Specified]
Method ANCOVA
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -10.90
Confidence Interval (2-Sided) 90%
-25.29 to 3.48
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.590
Estimation Comments [Not Specified]
Hide Statistical Analysis 13
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.086
Comments [Not Specified]
Method ANCOVA
Comments day 7
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 13.92
Confidence Interval 90%
0.58 to 27.25
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.963
Estimation Comments [Not Specified]
Hide Statistical Analysis 14
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.951
Comments [Not Specified]
Method ANCOVA
Comments Day 14
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.48
Confidence Interval (2-Sided) 90%
-12.57 to 13.53
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.787
Estimation Comments [Not Specified]
Hide Statistical Analysis 15
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.545
Comments [Not Specified]
Method ANCOVA
Comments day 21
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 5.02
Confidence Interval (2-Sided) 90%
-8.79 to 18.83
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.244
Estimation Comments [Not Specified]
Hide Statistical Analysis 16
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.982
Comments [Not Specified]
Method ANCOVA
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 0.19
Confidence Interval (2-Sided) 90%
-14.38 to 14.77
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.697
Estimation Comments [Not Specified]
Hide Statistical Analysis 17
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.047
Comments [Not Specified]
Method ANCOVA
Comments day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -13.82
Confidence Interval (2-Sided) 90%
-25.21 to -2.43
Parameter Dispersion
Type: Standard Error of the Mean
Value: 6.797
Estimation Comments [Not Specified]
Hide Statistical Analysis 18
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.09 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.029
Comments [Not Specified]
Method ANCOVA
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -18.23
Confidence Interval (2-Sided) 90%
-31.81 to -4.64
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.110
Estimation Comments [Not Specified]
Hide Statistical Analysis 19
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.004
Comments [Not Specified]
Method ANCOVA
Comments Day 7
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 26.70
Confidence Interval (2-Sided) 90%
11.97 to 41.44
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.799
Estimation Comments [Not Specified]
Hide Statistical Analysis 20
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.370
Comments [Not Specified]
Method ANCOVA
Comments Day 14
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 8.17
Confidence Interval (2-Sided) 90%
-6.96 to 23.29
Parameter Dispersion
Type: Standard Error of the Mean
Value: 9.032
Estimation Comments [Not Specified]
Hide Statistical Analysis 21
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.558
Comments [Not Specified]
Method ANCOVA
Comments Day 21
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 5.90
Confidence Interval (2-Sided) 90%
-10.86 to 22.66
Parameter Dispersion
Type: Standard Error of the Mean
Value: 10.014
Estimation Comments [Not Specified]
Hide Statistical Analysis 22
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.418
Comments [Not Specified]
Method ANCOVA
Comments Day 28
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value 8.91
Confidence Interval (2-Sided) 90%
-9.34 to 27.15
Parameter Dispersion
Type: Standard Error of the Mean
Value: 10.911
Estimation Comments [Not Specified]
Hide Statistical Analysis 23
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.156
Comments [Not Specified]
Method ANCOVA
Comments Day 56
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -11.08
Confidence Interval (2-Sided) 90%
-23.95 to 1.80
Parameter Dispersion
Type: Standard Error of the Mean
Value: 7.690
Estimation Comments [Not Specified]
Hide Statistical Analysis 24
Statistical Analysis Overview Comparison Group Selection LJN452 - 0.15 mg qd, Placebo qd
Comments [Not Specified]
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.064
Comments [Not Specified]
Method ANCOVA
Comments Day 84
Method of Estimation Estimation Parameter Median difference from baseline
Estimated Value -16.93
Confidence Interval (2-Sided) 90%
-31.94 to -1.92
Parameter Dispersion
Type: Standard Error of the Mean
Value: 8.961
Estimation Comments [Not Specified]
Time Frame Adverse events and serious adverse events were collected for the maximum actual duration of treatment exposure and follow up for a participant per the protocol for approximately 56 days post last dose.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Hide Arm/Group Description Tropifexor 0.03 mg daily for 28 days Tropifexor 0.06 mg daily for 28 days Tropifexor 0.09 mg daily for 28 days Tropifexor 0.15 mg daily for 28 days Tropifexor placebo daily for 28 days
All-Cause Mortality
LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)   0/9 (0.00%)   0/12 (0.00%)   0/8 (0.00%)   0/21 (0.00%) 
Hide Serious Adverse Events
LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/11 (0.00%)   0/9 (0.00%)   0/12 (0.00%)   0/8 (0.00%)   0/21 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
LJN452 - 0.03 mg qd LJN452 - 0.06 mg qd LJN452 - 0.09 mg qd LJN452 - 0.15 mg qd Placebo qd
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   9/11 (81.82%)   8/9 (88.89%)   11/12 (91.67%)   8/8 (100.00%)   16/21 (76.19%) 
Blood and lymphatic system disorders           
Anaemia  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Leukopenia  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Thrombocytopenia  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Cardiac disorders           
Trifascicular block  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Eye disorders           
Dry eye  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Eye pruritus  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Gastrointestinal disorders           
Abdominal discomfort  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Abdominal distension  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Abdominal pain lower  1  2/11 (18.18%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Abdominal pain upper  1  1/11 (9.09%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  3/21 (14.29%) 
Constipation  1  0/11 (0.00%)  1/9 (11.11%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Diarrhoea  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Dry mouth  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Dyspepsia  1  1/11 (9.09%)  2/9 (22.22%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Epigastric discomfort  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Flatulence  1  1/11 (9.09%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Gastrooesophageal reflux disease  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Haemorrhoidal haemorrhage  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Nausea  1  1/11 (9.09%)  1/9 (11.11%)  2/12 (16.67%)  0/8 (0.00%)  3/21 (14.29%) 
Varices oesophageal  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Vomiting  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  2/21 (9.52%) 
General disorders           
Fatigue  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  1/8 (12.50%)  1/21 (4.76%) 
Non-cardiac chest pain  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Oedema peripheral  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  2/21 (9.52%) 
Infections and infestations           
Fungal infection  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Nasopharyngitis  1  2/11 (18.18%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  1/21 (4.76%) 
Overgrowth bacterial  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Pneumonia  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Rash pustular  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Sinusitis  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Tooth abscess  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Upper respiratory tract infection  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Urinary tract infection  1  0/11 (0.00%)  0/9 (0.00%)  2/12 (16.67%)  0/8 (0.00%)  0/21 (0.00%) 
Viral infection  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Viral upper respiratory tract infection  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Injury, poisoning and procedural complications           
Arthropod bite  1  2/11 (18.18%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Muscle strain  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Investigations           
Alanine aminotransferase increased  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  2/8 (25.00%)  0/21 (0.00%) 
Aspartate aminotransferase increased  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Blood creatine phosphokinase increased  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Blood creatinine increased  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Low density lipoprotein increased  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Weight increased  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Metabolism and nutrition disorders           
Decreased appetite  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  2/21 (9.52%) 
Hypercholesterolaemia  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Hyperlipidaemia  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Iron deficiency  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Musculoskeletal and connective tissue disorders           
Costochondritis  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Muscle spasms  1  2/11 (18.18%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Pain in extremity  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Nervous system disorders           
Aphasia  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Dizziness  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Dysgeusia  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Headache  1  0/11 (0.00%)  0/9 (0.00%)  2/12 (16.67%)  1/8 (12.50%)  3/21 (14.29%) 
Hypoaesthesia  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Optic neuritis  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Psychiatric disorders           
Initial insomnia  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Insomnia  1  1/11 (9.09%)  0/9 (0.00%)  1/12 (8.33%)  1/8 (12.50%)  0/21 (0.00%) 
Sleep disorder  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  1/21 (4.76%) 
Renal and urinary disorders           
Dysuria  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Pollakiuria  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Proteinuria  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  0/21 (0.00%) 
Reproductive system and breast disorders           
Vulvovaginal discomfort  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Respiratory, thoracic and mediastinal disorders           
Cough  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Epistaxis  1  0/11 (0.00%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  1/21 (4.76%) 
Nasal congestion  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Oropharyngeal pain  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Productive cough  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Sinus congestion  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Skin and subcutaneous tissue disorders           
Pruritus  1  3/11 (27.27%)  6/9 (66.67%)  5/12 (41.67%)  7/8 (87.50%)  6/21 (28.57%) 
Pruritus generalised  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Psoriasis  1  0/11 (0.00%)  1/9 (11.11%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
Rash  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  1/8 (12.50%)  1/21 (4.76%) 
Rash maculo-papular  1  0/11 (0.00%)  0/9 (0.00%)  1/12 (8.33%)  0/8 (0.00%)  0/21 (0.00%) 
Vascular disorders           
Hypertension  1  1/11 (9.09%)  0/9 (0.00%)  0/12 (0.00%)  0/8 (0.00%)  0/21 (0.00%) 
1
Term from vocabulary, MedDRA (21.0)
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 terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (ie, data from all sites) in the clinical trial.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
Phone: 862-778-8300
EMail: Novartis.email@novartis.com
Layout table for additonal information
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02516605    
Other Study ID Numbers: CLJN452X2201
2015-001590-41 ( EudraCT Number )
First Submitted: August 4, 2015
First Posted: August 6, 2015
Results First Submitted: August 1, 2019
Results First Posted: November 13, 2019
Last Update Posted: January 5, 2021