Trial Outcomes & Findings for Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol. (NCT NCT03226067)
NCT ID: NCT03226067
Last Updated: 2022-06-29
Results Overview
Percent change in serum GGT from baseline to Week 24 (serum GGT was measured in U/L)
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
111 participants
Primary outcome timeframe
Baseline to week 24 (visit 7)
Results posted on
2022-06-29
Participant Flow
Recruitment started Sep 2017-Sep 2018
Participant milestones
| Measure |
GKT137831 400mg Twice Daily
GKT137831 400mg twice daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
|
GKT137831 400mg Once Daily
GKT137831 400mg once daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. The capsules in the evening will be placebos.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
Placebo oral capsule: Matching capsules.
|
Placebo Arm
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. All capsules will be placebos.
Placebo oral capsule: Matching capsules.
|
|---|---|---|---|
|
Overall Study
STARTED
|
36
|
38
|
37
|
|
Overall Study
COMPLETED
|
36
|
38
|
37
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Assess Safety & Efficacy of GKT137831 in Patients With Primary Biliary Cholangitis Receiving Ursodiol.
Baseline characteristics by cohort
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=36 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
Total
n=111 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
31 Participants
n=99 Participants
|
30 Participants
n=107 Participants
|
30 Participants
n=206 Participants
|
91 Participants
n=7 Participants
|
|
Age, Categorical
>=65 years
|
7 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
20 Participants
n=7 Participants
|
|
Age, Continuous
|
56.8 years
STANDARD_DEVIATION 9.1 • n=99 Participants
|
55.6 years
STANDARD_DEVIATION 9.04 • n=107 Participants
|
56.3 years
STANDARD_DEVIATION 9.19 • n=206 Participants
|
56.2 years
STANDARD_DEVIATION 9.04 • n=7 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=99 Participants
|
34 Participants
n=107 Participants
|
35 Participants
n=206 Participants
|
99 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
2 Participants
n=206 Participants
|
12 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
38 Participants
n=99 Participants
|
36 Participants
n=107 Participants
|
36 Participants
n=206 Participants
|
110 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Black
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Region of Enrollment
Greece
|
4 participants
n=99 Participants
|
3 participants
n=107 Participants
|
3 participants
n=206 Participants
|
10 participants
n=7 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=99 Participants
|
2 participants
n=107 Participants
|
1 participants
n=206 Participants
|
6 participants
n=7 Participants
|
|
Region of Enrollment
Belgium
|
5 participants
n=99 Participants
|
2 participants
n=107 Participants
|
7 participants
n=206 Participants
|
14 participants
n=7 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=99 Participants
|
7 participants
n=107 Participants
|
11 participants
n=206 Participants
|
27 participants
n=7 Participants
|
|
Region of Enrollment
Italy
|
5 participants
n=99 Participants
|
6 participants
n=107 Participants
|
1 participants
n=206 Participants
|
12 participants
n=7 Participants
|
|
Region of Enrollment
United Kingdom
|
2 participants
n=99 Participants
|
8 participants
n=107 Participants
|
6 participants
n=206 Participants
|
16 participants
n=7 Participants
|
|
Region of Enrollment
Israel
|
5 participants
n=99 Participants
|
3 participants
n=107 Participants
|
5 participants
n=206 Participants
|
13 participants
n=7 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=99 Participants
|
2 participants
n=107 Participants
|
3 participants
n=206 Participants
|
7 participants
n=7 Participants
|
|
Region of Enrollment
Spain
|
3 participants
n=99 Participants
|
3 participants
n=107 Participants
|
0 participants
n=206 Participants
|
6 participants
n=7 Participants
|
|
Disease severity level based on baseline serum GGT
<2.5 X ULN
|
8 participants
n=99 Participants
|
7 participants
n=107 Participants
|
10 participants
n=206 Participants
|
25 participants
n=7 Participants
|
|
Disease severity level based on baseline serum GGT
>= 2.5 X ULN
|
30 participants
n=99 Participants
|
29 participants
n=107 Participants
|
27 participants
n=206 Participants
|
86 participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Baseline to week 24 (visit 7)Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in serum GGT from baseline to Week 24 (serum GGT was measured in U/L)
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=37 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=30 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=35 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
The Percent Change in Serum GGT.
|
-4.9 percent change
Standard Deviation 59.58
|
-19 percent change
Standard Deviation 28.89
|
-8.4 percent change
Standard Deviation 21.45
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in serum GGT from baseline to each assessment.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Serum GGT
Absolute change W2
|
-14.3 U/L
Standard Deviation 112.66
|
-48.5 U/L
Standard Deviation 61.83
|
-11.2 U/L
Standard Deviation 45.96
|
|
Absolute Change in Serum GGT
Absolute change W6
|
-22.2 U/L
Standard Deviation 76.68
|
-53.5 U/L
Standard Deviation 67.57
|
-17.9 U/L
Standard Deviation 59.01
|
|
Absolute Change in Serum GGT
Absolute change W12
|
-3.9 U/L
Standard Deviation 115.13
|
-50.6 U/L
Standard Deviation 74.7
|
-8.6 U/L
Standard Deviation 70.11
|
|
Absolute Change in Serum GGT
Absolute change W18
|
-19.2 U/L
Standard Deviation 134.32
|
-37.9 U/L
Standard Deviation 93.88
|
-4.5 U/L
Standard Deviation 79.81
|
|
Absolute Change in Serum GGT
Absolute change W24
|
-17.9 U/L
Standard Deviation 117.62
|
-43.6 U/L
Standard Deviation 62.22
|
-10.7 U/L
Standard Deviation 78.2
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in serum GGT from baseline to each assessment (serum GGT was measured in U/L)
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Serum GGT
Percent change W2
|
-7 percent change
Standard Deviation 25.08
|
-17 percent change
Standard Deviation 17.25
|
-6.2 percent change
Standard Deviation 13.04
|
|
Percent Change in Serum GGT
Percent change W6
|
-11.8 percent change
Standard Deviation 21.59
|
-22 percent change
Standard Deviation 23.4
|
-7.5 percent change
Standard Deviation 16.89
|
|
Percent Change in Serum GGT
Percent change W12
|
1.7 percent change
Standard Deviation 66.84
|
-18.6 percent change
Standard Deviation 27.44
|
-5.5 percent change
Standard Deviation 19.44
|
|
Percent Change in Serum GGT
Percent change W18
|
-4.5 percent change
Standard Deviation 69.65
|
-18.7 percent change
Standard Deviation 28.55
|
-5.2 percent change
Standard Deviation 22.79
|
|
Percent Change in Serum GGT
Percent change W24
|
-4.9 percent change
Standard Deviation 59.58
|
-19 percent change
Standard Deviation 28.89
|
-8.4 percent change
Standard Deviation 21.45
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in serum ALP from baseline to each assessment (serum ALP was measured in U/L).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Serum ALP
Percent change W2
|
-5.5 percent change
Standard Deviation 10.95
|
-13.0 percent change
Standard Deviation 12.71
|
-3.6 percent change
Standard Deviation 11.51
|
|
Percent Change in Serum ALP
Percent change W6
|
-8.6 percent change
Standard Deviation 13.42
|
-16.3 percent change
Standard Deviation 13.32
|
-1.4 percent change
Standard Deviation 15.19
|
|
Percent Change in Serum ALP
Percent change W12
|
-3.9 percent change
Standard Deviation 22.17
|
-14.6 percent change
Standard Deviation 17.89
|
-0.6 percent change
Standard Deviation 16.41
|
|
Percent Change in Serum ALP
Percent change W18
|
-7.8 percent change
Standard Deviation 21.68
|
-15.8 percent change
Standard Deviation 21.42
|
-0.5 percent change
Standard Deviation 15.66
|
|
Percent Change in Serum ALP
Percent change W24
|
-9.7 percent change
Standard Deviation 21.1
|
-12.9 percent change
Standard Deviation 19.55
|
-3.1 percent change
Standard Deviation 15.99
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in serum ALP from baseline to each assessment.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Serum ALP
Absolute change W2
|
-19.8 U/L
Standard Deviation 35.95
|
-45.9 U/L
Standard Deviation 75.75
|
-17 U/L
Standard Deviation 44.4
|
|
Absolute Change in Serum ALP
Absolute change W6
|
-27.3 U/L
Standard Deviation 44.13
|
-58.6 U/L
Standard Deviation 63.88
|
-14.5 U/L
Standard Deviation 59.72
|
|
Absolute Change in Serum ALP
Absolute change W12
|
-15.3 U/L
Standard Deviation 67.79
|
-53 U/L
Standard Deviation 66.51
|
-7.1 U/L
Standard Deviation 52.75
|
|
Absolute Change in Serum ALP
Absolute change W18
|
-27.6 U/L
Standard Deviation 62.54
|
-53.2 U/L
Standard Deviation 80.01
|
-6 U/L
Standard Deviation 50.15
|
|
Absolute Change in Serum ALP
Absolute change W24
|
-32.5 U/L
Standard Deviation 65.3
|
-45.2 U/L
Standard Deviation 84.41
|
-12.4 U/L
Standard Deviation 53.48
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in serum conjugated bilirubin from baseline to each assessment.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=32 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=27 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=32 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Serum Conjugated Bilirubin.
Absolute change W2
|
0 μmol/L
Standard Deviation 1.32
|
0.3 μmol/L
Standard Deviation 1.61
|
-0.1 μmol/L
Standard Deviation 1.4
|
|
Absolute Change in Serum Conjugated Bilirubin.
Absolute change W6
|
-0.3 μmol/L
Standard Deviation 1.14
|
0.5 μmol/L
Standard Deviation 2.06
|
-0.3 μmol/L
Standard Deviation 1.29
|
|
Absolute Change in Serum Conjugated Bilirubin.
Absolute change W12
|
0.8 μmol/L
Standard Deviation 1.97
|
0.4 μmol/L
Standard Deviation 1.28
|
-0.1 μmol/L
Standard Deviation 1.55
|
|
Absolute Change in Serum Conjugated Bilirubin.
Absolue change W18
|
0.5 μmol/L
Standard Deviation 2.10
|
0.6 μmol/L
Standard Deviation 1.78
|
-0.0 μmol/L
Standard Deviation 1.91
|
|
Absolute Change in Serum Conjugated Bilirubin.
Absolute change W24
|
1.1 μmol/L
Standard Deviation 3.44
|
1.1 μmol/L
Standard Deviation 1.76
|
0.3 μmol/L
Standard Deviation 2.57
|
SECONDARY outcome
Timeframe: From baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in serum Conjugated bilirubin, from baseline to each assessment (serum conjugated bilirubin is measured in μmol/L).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=32 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=27 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=32 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Serum Conjugated Bilirubin.
Percent change W2
|
0.9 percent change
Standard Deviation 16.81
|
5.2 percent change
Standard Deviation 22.79
|
1.3 percent change
Standard Deviation 28.11
|
|
Percent Change in Serum Conjugated Bilirubin.
Percent change W6
|
-3.4 percent change
Standard Deviation 18.7
|
6.3 percent change
Standard Deviation 29.4
|
-3.5 percent change
Standard Deviation 17.99
|
|
Percent Change in Serum Conjugated Bilirubin.
Percent change W12
|
9.4 percent change
Standard Deviation 27.14
|
4.8 percent change
Standard Deviation 18.55
|
-0.7 percent change
Standard Deviation 20.81
|
|
Percent Change in Serum Conjugated Bilirubin.
Percent change W18
|
6.0 percent change
Standard Deviation 28.16
|
8.9 percent change
Standard Deviation 19.34
|
1.8 percent change
Standard Deviation 25.98
|
|
Percent Change in Serum Conjugated Bilirubin.
Percent change W24
|
12.7 percent change
Standard Deviation 36.35
|
16.1 percent change
Standard Deviation 24.85
|
6.4 percent change
Standard Deviation 32.90
|
SECONDARY outcome
Timeframe: from baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in serum total bilirubin, from baseline to each assessment.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Serum Total Bilirubin.
Absolute change W2
|
-0.2 μmol/L
Standard Deviation 2.51
|
0.1 μmol/L
Standard Deviation 2.76
|
-0.4 μmol/L
Standard Deviation 3.75
|
|
Absolute Change in Serum Total Bilirubin.
Absolute change W6
|
-0.5 μmol/L
Standard Deviation 2.35
|
0.5 μmol/L
Standard Deviation 3.39
|
-0.5 μmol/L
Standard Deviation 3.29
|
|
Absolute Change in Serum Total Bilirubin.
Absolute change W12
|
0.6 μmol/L
Standard Deviation 3.35
|
0.1 μmol/L
Standard Deviation 2.69
|
0.0 μmol/L
Standard Deviation 2.21
|
|
Absolute Change in Serum Total Bilirubin.
Absolute change W18
|
0.1 μmol/L
Standard Deviation 3.22
|
0.5 μmol/L
Standard Deviation 2.82
|
-0.1 μmol/L
Standard Deviation 3.81
|
|
Absolute Change in Serum Total Bilirubin.
Absolute change W24
|
0.5 μmol/L
Standard Deviation 3.77
|
1.2 μmol/L
Standard Deviation 2.71
|
0.7 μmol/L
Standard Deviation 3.23
|
SECONDARY outcome
Timeframe: from baseline to Weeks 2, 6, 12, 18 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in Serum Total Bilirubin from baseline to each assessment (serum total bilirubin is measured in μmol/L).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=35 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Serum Total Bilirubin.
Percent change W2
|
-0.6 percent change
Standard Deviation 23.62
|
5.3 percent change
Standard Deviation 32.42
|
3.3 percent change
Standard Deviation 39.98
|
|
Percent Change in Serum Total Bilirubin.
Percent change W6
|
-1.6 percent change
Standard Deviation 21.94
|
8.8 percent change
Standard Deviation 41.19
|
0.1 percent change
Standard Deviation 34.6
|
|
Percent Change in Serum Total Bilirubin.
Percent change W12
|
5.7 percent change
Standard Deviation 32.99
|
7.7 percent change
Standard Deviation 37.53
|
3.1 percent change
Standard Deviation 22.39
|
|
Percent Change in Serum Total Bilirubin.
Percent change W18
|
1.7 percent change
Standard Deviation 28.34
|
9.6 percent change
Standard Deviation 32.56
|
4.1 percent change
Standard Deviation 29.5
|
|
Percent Change in Serum Total Bilirubin.
Percent change W24
|
5.4 percent change
Standard Deviation 29.77
|
14.5 percent change
Standard Deviation 31.60
|
10.5 percent change
Standard Deviation 32.27
|
SECONDARY outcome
Timeframe: From baseline to Week 24, in patients with values at baseline and Week 24.Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=33 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=26 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=32 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
|
-0.5 kPa
Standard Deviation 5.24
|
-0.3 kPa
Standard Deviation 7.9
|
0.7 kPa
Standard Deviation 3.63
|
SECONDARY outcome
Timeframe: From baseline to Week 24, in patients with values at baseline and Week 24.Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in liver stiffness as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=33 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=26 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=32 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Liver Stiffness as Assessed by Transient Elastography (FibroScan® or Similar Technology).
|
3.3 percent change
Standard Deviation 34.95
|
7.9 percent change
Standard Deviation 43.68
|
10.1 percent change
Standard Deviation 33.11
|
SECONDARY outcome
Timeframe: From baseline to Weeks 12 and 24.Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in serum levels of collagen fragments indicative of collagen formation and degradation, from baseline to Weeks 12 and 24 (serum levels of collagen fragments are measured in ng/mL).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=33 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=34 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change ProC3 W12
|
-2.58 percent change
Standard Deviation 21.09
|
-1.31 percent change
Standard Deviation 18.23
|
3.64 percent change
Standard Deviation 27.86
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change ProC3 W24
|
-3.6 percent change
Standard Deviation 22.54
|
0.69 percent change
Standard Deviation 18.17
|
3.10 percent change
Standard Deviation 20.78
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change ProC5 W12
|
1.17 percent change
Standard Deviation 20.56
|
-6.65 percent change
Standard Deviation 22.99
|
0.35 percent change
Standard Deviation 22.52
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change ProC5 W24
|
2.26 percent change
Standard Deviation 30.36
|
-3.27 percent change
Standard Deviation 17.73
|
-0.51 percent change
Standard Deviation 37.78
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change C3M W12
|
-0.63 percent change
Standard Deviation 15.03
|
-3.06 percent change
Standard Deviation 17.73
|
1.31 percent change
Standard Deviation 20.17
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change C3M W24
|
-0.93 percent change
Standard Deviation 15.29
|
0.17 percent change
Standard Deviation 30.10
|
-1.06 percent change
Standard Deviation 23.88
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change C4M W12
|
1.59 percent change
Standard Deviation 15.76
|
-4.82 percent change
Standard Deviation 18.97
|
3.19 percent change
Standard Deviation 20.80
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change C4M W24
|
-4.43 percent change
Standard Deviation 17.72
|
3.33 percent change
Standard Deviation 37.63
|
0.73 percent change
Standard Deviation 21.55
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change BGM W12
|
-0.13 percent change
Standard Deviation 14.91
|
-4.3 percent change
Standard Deviation 18.81
|
-0.12 percent change
Standard Deviation 23.07
|
|
Percent Change in Serum Levels of Collagen Fragments Indicative of Collagen Formation and Degradation.
Percent change BGM W24
|
1.19 percent change
Standard Deviation 17.15
|
2.18 percent change
Standard Deviation 29.14
|
3.77 percent change
Standard Deviation 25.89
|
SECONDARY outcome
Timeframe: From baseline to Week 24, in patients with values at baseline and Week 24.Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute change in liver stiffness by subgroup (\>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=14 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=9 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=16 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
|
-1.9 kPa
Standard Deviation 7.49
|
-2.1 kPa
Standard Deviation 2.43
|
0.4 kPa
Standard Deviation 4.65
|
SECONDARY outcome
Timeframe: From baseline to Week 24, in patients with values at baseline and Week 24.Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent change in liver stiffness by subgroup (\>=9.6 kPa) as assessed by transient elastography (FibroScan® or similar technology), from baseline to Week 24, in subjects with values at baseline and Week 24 (liver stiffness is measured in kPa).
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=14 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=9 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=16 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Liver Stiffness as Assessed by Transient Elastography by Subgroup (FibroScan® or Similar Technology).
|
-5.3 percent change
Standard Deviation 35.10
|
-16.1 percent change
Standard Deviation 20.71
|
4.2 percent change
Standard Deviation 30.09
|
SECONDARY outcome
Timeframe: From baseline to Weeks 12 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=34 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in Pruritis Visual Analogue Scale (VAS) Scores
Absolute change W12
|
-0.1 score on a scale
Standard Deviation 3.1
|
0.3 score on a scale
Standard Deviation 2.33
|
0.5 score on a scale
Standard Deviation 1.79
|
|
Absolute Change in Pruritis Visual Analogue Scale (VAS) Scores
Absolute change W24
|
-1.0 score on a scale
Standard Deviation 2.32
|
0.1 score on a scale
Standard Deviation 2.3
|
0.7 score on a scale
Standard Deviation 1.83
|
SECONDARY outcome
Timeframe: From baseline to Weeks 12 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent Change in Pruritis Visual Analogue Scale (VAS) scores from baseline to weeks 12 and 24- Score from 0 to 10, 0= no itch and 10= severe itch, continuous, day and night intolerable itch.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=34 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in Pruritis Visual Analogue Scale (VAS) Scores
Percent change W12
|
-35.1 percent change
Standard Deviation 46.13
|
-11.7 percent change
Standard Deviation 73.87
|
-8.3 percent change
Standard Deviation 70.68
|
|
Percent Change in Pruritis Visual Analogue Scale (VAS) Scores
Percent change W24
|
-36.9 percent change
Standard Deviation 58.6
|
-0.3 percent change
Standard Deviation 92.71
|
27.3 percent change
Standard Deviation 72.88
|
SECONDARY outcome
Timeframe: From baseline to Weeks 12 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Absolute Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=34 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Symptoms W12
|
-0.4 score on a scale
Standard Deviation 3.34
|
-0.9 score on a scale
Standard Deviation 3.19
|
-0.2 score on a scale
Standard Deviation 3.52
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Symptoms W24
|
0.1 score on a scale
Standard Deviation 3.46
|
-1.4 score on a scale
Standard Deviation 4.23
|
-0.3 score on a scale
Standard Deviation 3.10
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Itch W12
|
-0.2 score on a scale
Standard Deviation 2.61
|
0.6 score on a scale
Standard Deviation 2.69
|
0.4 score on a scale
Standard Deviation 2.79
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Itch W24
|
-0.4 score on a scale
Standard Deviation 3.22
|
0.1 score on a scale
Standard Deviation 2.83
|
0.1 score on a scale
Standard Deviation 2.85
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Fatigue W12
|
-1.8 score on a scale
Standard Deviation 5.76
|
-3.2 score on a scale
Standard Deviation 8.25
|
0.6 score on a scale
Standard Deviation 4.91
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Fatigue W24
|
-0.8 score on a scale
Standard Deviation 6.31
|
-3.6 score on a scale
Standard Deviation 7.32
|
0.6 score on a scale
Standard Deviation 5.35
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Cognitive W12
|
-0.1 score on a scale
Standard Deviation 5.02
|
-2.4 score on a scale
Standard Deviation 5.68
|
1.0 score on a scale
Standard Deviation 4.35
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Cognitive W24
|
0.8 score on a scale
Standard Deviation 4.67
|
-1.4 score on a scale
Standard Deviation 5.34
|
0.3 score on a scale
Standard Deviation 3.67
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Emotional W12
|
-0.5 score on a scale
Standard Deviation 2.09
|
-1.8 score on a scale
Standard Deviation 3.2
|
0.4 score on a scale
Standard Deviation 2.35
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Emotional W24
|
-0.3 score on a scale
Standard Deviation 2.6
|
-2.0 score on a scale
Standard Deviation 3.0
|
0.4 score on a scale
Standard Deviation 1.78
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Social W12
|
0.6 score on a scale
Standard Deviation 4.69
|
-2.1 score on a scale
Standard Deviation 5.24
|
2.0 score on a scale
Standard Deviation 5.43
|
|
Absolute Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Absolute change Social W24
|
0.8 score on a scale
Standard Deviation 5.08
|
-2.2 score on a scale
Standard Deviation 4.93
|
1.7 score on a scale
Standard Deviation 5.01
|
SECONDARY outcome
Timeframe: From baseline to Weeks 12 and 24Population: Number of subjects analyzed in the Intent to Treat population who have evaluable results
Percent Change in PBC-40 Domain scores from baseline to weeks 12 and 24 Symptoms domain: items 1 to 7 of the PBC-40 questionnaire (score from 6 to 35). Itch domain: items 8 to 10 of the PBC-40 questionnaire (score from 0 to 15). Fatigue domain: items 11 to 21 of the PBC-40 questionnaire (score from 11 to 55). Cognitive domain: items 22 to 27 of the PBC-40 questionnaire (score from 6 to 30). Emotional domain: items 28, 30 and 33 of the PBC-40 questionnaire (score from 3 to 15). Social domain: items 29, 31, 32, 34 to 40 of the PBC-40 questionnaire (score from 8 to 50). Higher scores mean worse outcome.
Outcome measures
| Measure |
GKT137831 400mg Once Daily
n=38 Participants
Subjects randomized to the 400 mg OD dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 placebo capsules (PM dose)
|
GKT137831 400mg Twice Daily
n=34 Participants
Subjects randomized to the 400 mg BID dose arm self-administered IMP twice daily for 24 weeks: 4 active capsules (AM dose) and 4 active capsules (PM dose)
|
Placebo Arm
n=37 Participants
Subjects randomized to the placebo arm self-administered placebo twice daily for 24 weeks: 4 placebo capsules (AM dose) and 4 placebo capsules (PM dose)
|
|---|---|---|---|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Symptoms W12
|
-2.8 percent change
Standard Deviation 24.68
|
-2.7 percent change
Standard Deviation 24.48
|
3.1 percent change
Standard Deviation 37.21
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Symptoms W24
|
1.1 percent change
Standard Deviation 25.27
|
-3.7 percent change
Standard Deviation 25.19
|
1.1 percent change
Standard Deviation 35.03
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Itch W12
|
-5.6 percent change
Standard Deviation 62.92
|
-9.2 percent change
Standard Deviation 43.43
|
-2.5 percent change
Standard Deviation 48.47
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Itch W24
|
-11.4 percent change
Standard Deviation 52.75
|
-9.5 percent change
Standard Deviation 41.83
|
-6.8 percent change
Standard Deviation 40.62
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Fatigue W12
|
-3.4 percent change
Standard Deviation 21.11
|
-6.8 percent change
Standard Deviation 26.51
|
3.4 percent change
Standard Deviation 20.45
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Fatigue W24
|
0.3 percent change
Standard Deviation 24.89
|
-9.9 percent change
Standard Deviation 19.81
|
2.4 percent change
Standard Deviation 23.07
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Cognitive W12
|
6.1 percent change
Standard Deviation 56.79
|
-9.5 percent change
Standard Deviation 32.14
|
11.7 percent change
Standard Deviation 51.28
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Cognitive W24
|
16.0 percent change
Standard Deviation 62.27
|
-1.9 percent change
Standard Deviation 40.79
|
5.2 percent change
Standard Deviation 46.11
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Emotional W12
|
-1.2 percent change
Standard Deviation 35.5
|
-12.8 percent change
Standard Deviation 33.88
|
8.7 percent change
Standard Deviation 45.99
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Emotional W24
|
4.9 percent change
Standard Deviation 54.41
|
-16.9 percent change
Standard Deviation 26.85
|
8.7 percent change
Standard Deviation 34.49
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Social W12
|
4.8 percent change
Standard Deviation 24.14
|
-6.2 percent change
Standard Deviation 21.65
|
13.9 percent change
Standard Deviation 40.03
|
|
Percent Change in PBC-40 (Primary Biliary Cholongitis) Domain Scores
Percent change Social W24
|
8.1 percent change
Standard Deviation 27.94
|
-7.7 percent change
Standard Deviation 18.38
|
9.3 percent change
Standard Deviation 28.53
|
Adverse Events
GKT137831 400mg Twice Daily
Serious events: 1 serious events
Other events: 32 other events
Deaths: 0 deaths
GKT137831 400mg Once Daily
Serious events: 0 serious events
Other events: 34 other events
Deaths: 0 deaths
Placebo Arm
Serious events: 1 serious events
Other events: 31 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GKT137831 400mg Twice Daily
n=36 participants at risk
GKT137831 400mg twice daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
|
GKT137831 400mg Once Daily
n=38 participants at risk
GKT137831 400mg once daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. The capsules in the evening will be placebos.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
Placebo oral capsule: Matching capsules.
|
Placebo Arm
n=37 participants at risk
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. All capsules will be placebos.
Placebo oral capsule: Matching capsules.
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Multiple Fractures
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/37 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Infections and infestations
Urinary Infection
|
0.00%
0/36 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.7%
1/37 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
Other adverse events
| Measure |
GKT137831 400mg Twice Daily
n=36 participants at risk
GKT137831 400mg twice daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
|
GKT137831 400mg Once Daily
n=38 participants at risk
GKT137831 400mg once daily
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. The capsules in the evening will be placebos.
GKT137831: GKT137831 100mg capsules. To be taken as part of two dose arms which are 400mg twice daily or 400mg once daily.
Placebo oral capsule: Matching capsules.
|
Placebo Arm
n=37 participants at risk
Patients will self-administer 4 capsules in the morning and 4 capsules in the evening. All capsules will be placebos.
Placebo oral capsule: Matching capsules.
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
16.7%
6/36 • Number of events 7 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
7.9%
3/38 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
10.8%
4/37 • Number of events 6 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Abdominal Pain
|
8.3%
3/36 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
7.9%
3/38 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/37 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Abdominal Pain Upper
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.3%
2/38 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Constipation
|
8.3%
3/36 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.7%
1/37 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Gastrooesoghageal Reflux Disease
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
7.9%
3/38 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.7%
1/37 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Diarrhea
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
8.1%
3/37 • Number of events 5 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Dry Mouth
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Gastrointestinal disorders
Abdominal Distension
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/37 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
22.2%
8/36 • Number of events 10 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
21.1%
8/38 • Number of events 11 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
21.6%
8/37 • Number of events 8 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Immune system disorders
Nasopharyngitis
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
7.9%
3/38 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
8.1%
3/37 • Number of events 5 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Infections and infestations
Urinary Tract Infection
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
10.8%
4/37 • Number of events 5 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Infections and infestations
Oral Herpes
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Nervous system disorders
Headache
|
11.1%
4/36 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
13.2%
5/38 • Number of events 8 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
13.5%
5/37 • Number of events 5 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Nervous system disorders
Dizziness
|
11.1%
4/36 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
13.2%
5/38 • Number of events 8 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.7%
1/37 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
2.8%
1/36 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.3%
2/38 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
8.1%
3/37 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
General disorders
Fatigue
|
8.3%
3/36 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
21.6%
8/37 • Number of events 8 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
General disorders
Pyrexia
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 3 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
General disorders
Asthenia
|
5.6%
2/36 • Number of events 4 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/37 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal Pain
|
5.6%
2/36 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.6%
1/38 • Number of events 1 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
2.7%
1/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
|
Injury, poisoning and procedural complications
Arthropod Bite
|
0.00%
0/36 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
0.00%
0/38 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
5.4%
2/37 • Number of events 2 • .Approximately 6-7 months
Adverse events were collected after signing the informed consent form (ICF) and up to completion of the 28-day follow-up period after the last administration of IMP
|
Additional Information
Richard Philipson, MD Chief Medical Officer
Calliditas Therapeutics
Phone: Desk: +46 556659-9766
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place