Trial Outcomes & Findings for Seladelpar (MBX-8025) in Subjects With Primary Biliary Cholangitis (PBC) (NCT NCT02955602)
NCT ID: NCT02955602
Last Updated: 2022-07-14
Results Overview
Relative change from baseline in serum ALP levels at Week 8 (endpoint). The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. n, denotes number of subjects evaluable for the respective timepoints
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
119 participants
Primary outcome timeframe
8 weeks
Results posted on
2022-07-14
Participant Flow
A total of 192 subjects were screened of which 121 subjects randomized (2 subjects were not treated) into the study and 71 were screen failures. Subjects were randomized to the 5 and 10 mg treatment groups for entry to the 8-week initial treatment period study, while those in the 2 mg treatment group entered after being sequentially assigned their dose
Participant milestones
| Measure |
MBX-8025 (2 mg)
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Overall Study
STARTED
|
11
|
53
|
55
|
|
Overall Study
COMPLETED
|
10
|
46
|
49
|
|
Overall Study
NOT COMPLETED
|
1
|
7
|
6
|
Reasons for withdrawal
| Measure |
MBX-8025 (2 mg)
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
3
|
0
|
|
Overall Study
Withdrawal of informed consent
|
1
|
3
|
2
|
|
Overall Study
Lost to Follow-up
|
0
|
0
|
1
|
|
Overall Study
Other
|
0
|
1
|
3
|
Baseline Characteristics
Seladelpar (MBX-8025) in Subjects With Primary Biliary Cholangitis (PBC)
Baseline characteristics by cohort
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=53 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=55 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
Total
n=119 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
41 Participants
n=7 Participants
|
41 Participants
n=5 Participants
|
90 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
3 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
29 Participants
n=4 Participants
|
|
Age, Continuous
|
55.2 years
STANDARD_DEVIATION 9.6 • n=5 Participants
|
57.5 years
STANDARD_DEVIATION 8.1 • n=7 Participants
|
57.4 years
STANDARD_DEVIATION 9.7 • n=5 Participants
|
56.4 years
STANDARD_DEVIATION 9.6 • n=4 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
51 Participants
n=7 Participants
|
50 Participants
n=5 Participants
|
112 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
7 Participants
n=4 Participants
|
|
Race/Ethnicity, Customized
White
|
10 Subjects
n=5 Participants
|
50 Subjects
n=7 Participants
|
49 Subjects
n=5 Participants
|
109 Subjects
n=4 Participants
|
|
Race/Ethnicity, Customized
Black or African-American
|
0 Subjects
n=5 Participants
|
1 Subjects
n=7 Participants
|
3 Subjects
n=5 Participants
|
4 Subjects
n=4 Participants
|
|
Race/Ethnicity, Customized
Asian
|
0 Subjects
n=5 Participants
|
2 Subjects
n=7 Participants
|
1 Subjects
n=5 Participants
|
3 Subjects
n=4 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska native
|
0 Subjects
n=5 Participants
|
0 Subjects
n=7 Participants
|
1 Subjects
n=5 Participants
|
1 Subjects
n=4 Participants
|
|
Race/Ethnicity, Customized
Other
|
1 Subjects
n=5 Participants
|
0 Subjects
n=7 Participants
|
0 Subjects
n=5 Participants
|
1 Subjects
n=4 Participants
|
|
Race/Ethnicity, Customized
Multiple
|
0 Subjects
n=5 Participants
|
0 Subjects
n=7 Participants
|
1 Subjects
n=5 Participants
|
1 Subjects
n=4 Participants
|
|
Region of Enrollment
Canada
|
0 participants
n=5 Participants
|
5 participants
n=7 Participants
|
1 participants
n=5 Participants
|
6 participants
n=4 Participants
|
|
Region of Enrollment
United States
|
0 participants
n=5 Participants
|
39 participants
n=7 Participants
|
43 participants
n=5 Participants
|
82 participants
n=4 Participants
|
|
Region of Enrollment
United Kingdom
|
11 participants
n=5 Participants
|
5 participants
n=7 Participants
|
4 participants
n=5 Participants
|
20 participants
n=4 Participants
|
|
Region of Enrollment
Germany
|
0 participants
n=5 Participants
|
4 participants
n=7 Participants
|
7 participants
n=5 Participants
|
11 participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 8 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Relative change from baseline in serum ALP levels at Week 8 (endpoint). The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. n, denotes number of subjects evaluable for the respective timepoints
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=47 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=51 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 8
|
-26.06 percentage change from baseline
Standard Deviation 9.15
|
-33.38 percentage change from baseline
Standard Deviation 17.81
|
-41.42 percentage change from baseline
Standard Deviation 13.05
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population: The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Absolute change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Absolute Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 12 and Week 52
Absolute Change at Week 12
|
-68.318 U/L
Standard Deviation 63.276
|
-135.902 U/L
Standard Deviation 150.954
|
-127.867 U/L
Standard Deviation 60.284
|
|
Absolute Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 12 and Week 52
Absolute Change at Week 52
|
-101.150 U/L
Standard Deviation 107.956
|
-158.310 U/L
Standard Deviation 143.668
|
-133.760 U/L
Standard Deviation 76.151
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from baseline in AST levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change in Aspartate Aminotransferase (AST) From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-2.50 U/L
Standard Deviation 16.72
|
-1.13 U/L
Standard Deviation 24.28
|
-3.35 U/L
Standard Deviation 10.55
|
|
Change in Aspartate Aminotransferase (AST) From Baseline to 12 Weeks and 52 Weeks
Week 52
|
-7.05 U/L
Standard Deviation 10.50
|
-6.18 U/L
Standard Deviation 13.98
|
-6.14 U/L
Standard Deviation 9.18
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from baseline in ALT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change in Alanine Aminotransferase (ALT) From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-5.59 U/L
Standard Deviation 16.07
|
-10.48 U/L
Standard Deviation 21.41
|
-10.87 U/L
Standard Deviation 20.25
|
|
Change in Alanine Aminotransferase (ALT) From Baseline to 12 Weeks and 52 Weeks
Week 52
|
-14.30 U/L
Standard Deviation 25.12
|
-17.29 U/L
Standard Deviation 17.46
|
-15.32 U/L
Standard Deviation 13.89
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from baseline in GGT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change in Gamma-glutamyl Transferase (GGT) From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-43.68 U/L
Standard Deviation 56.87
|
-75.35 U/L
Standard Deviation 87.57
|
-80.82 U/L
Standard Deviation 109.06
|
|
Change in Gamma-glutamyl Transferase (GGT) From Baseline to 12 Weeks and 52 Weeks
Week 52
|
-78.60 U/L
Standard Deviation 81.59
|
-91.37 U/L
Standard Deviation 102.23
|
-88.08 U/L
Standard Deviation 122.14
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from baseline in TB levels at endpoint is being reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change in Bilirubin - Total Bilirubin (TB) From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-0.010 mg/dL
Standard Deviation 0.121
|
-0.055 mg/dL
Standard Deviation 0.161
|
-0.067 mg/dL
Standard Deviation 0.199
|
|
Change in Bilirubin - Total Bilirubin (TB) From Baseline to 12 Weeks and 52 Weeks
Week 52
|
0.002 mg/dL
Standard Deviation 0.152
|
-0.028 mg/dL
Standard Deviation 0.263
|
-0.068 mg/dL
Standard Deviation 0.190
|
SECONDARY outcome
Timeframe: 12 Weeks and 52 WeeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Participant meets composite endpoint is defined by participant meets all of the following criteria: * ALP \< 1.67 × upper limit of normal (ULN) * Total Bilirubin within normal limit * \> 15% decrease in ALP Endpoint of Alkaline Phosphatase and Total Bilirubin by Visit (mITT Population)
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Composite Endpoint Criteria of ALP and Total Bilirubin
Week 12
|
45.5 percentage of participants
|
48.9 percentage of participants
|
66.7 percentage of participants
|
|
Percentage of Participants Meet Composite Endpoint Criteria of ALP and Total Bilirubin
Week 52
|
63.6 percentage of participants
|
53.3 percentage of participants
|
67.3 percentage of participants
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percentage of participants with response based on Paris I risk score was defined as ALP less than or equal to (≤) 3x ULN and aspartate aminotransferase (AST) less than or equal to (≤) 2 x ULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Published PBC Response Criteria - Paris I
Week 12
|
81.8 percentage of participants
|
76.1 percentage of participants
|
75.5 percentage of participants
|
|
Percentage of Participants Meet Published PBC Response Criteria - Paris I
Week 52
|
90 percentage of participants
|
81.0 percentage of participants
|
79.2 percentage of participants
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percentage of participants with response based on Paris II risk score was defined as ALP≤1.5xULN and AST≤1.5xULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Published PBC Response Criteria - Paris II
Week 12
|
36.4 percentage of participants
|
37.0 percentage of participants
|
59.2 percentage of participants
|
|
Percentage of Participants Meet Published PBC Response Criteria - Paris II
Week 52
|
50.0 percentage of participants
|
54.8 percentage of participants
|
60.4 percentage of participants
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percentage of participants with response based on Toronto I risk score defined as ALP ≤ 1.67 x ULN. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Published PBC Response Criteria - Toronto I
Week 12
|
63.6 percentage of subjects
|
51.1 percentage of subjects
|
78.4 percentage of subjects
|
|
Percentage of Participants Meet Published PBC Response Criteria - Toronto I
Week 52
|
63.6 percentage of subjects
|
57.8 percentage of subjects
|
71.4 percentage of subjects
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
The UK-PBC Risk Score at endpoint is defined by the mean percentage risk that a PBC patient treated with ursodeoxycholic acid (UDCA) would develop liver failure requiring liver transplantation in 5, 10 and 15 years from diagnosis. The higher the score might indicate higher risk to death or live transplantation. Formula used for UK-PBC risk score = 1- 0.982 \^EXP(0.0287854\*(ALP12 x ULN-1.722136304) - 0.0422873\*(((TA12 xULN/10)\^-1) - 8.675729006) + 1.4199 \* (LN(BIL12 x ULN/10)+2.709607778)-1.960303\*(Albumin x LLN-1.17673001)-0.4161954\*(Platelet x LLN-1.873564875)). Where, Baseline survivor function = 0.982, 0.941, and 0.893 for 5 years, 10 years and 15 years respectively. ALP12, TA12 and BIL12 refers to the ALP, transaminases (ALT, AST), and total bilirubin assessments, respectively. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
UK-PBC Risk Score Value
5 years, week 12
|
1.3929 units on a scale
Standard Deviation 0.6976
|
2.2553 units on a scale
Standard Deviation 2.5145
|
1.8124 units on a scale
Standard Deviation 1.7267
|
|
UK-PBC Risk Score Value
5 years, week 52
|
1.3145 units on a scale
Standard Deviation 0.7704
|
2.3485 units on a scale
Standard Deviation 3.432
|
1.7244 units on a scale
Standard Deviation 1.6121
|
|
UK-PBC Risk Score Value
10 years week 12
|
4.5705 units on a scale
Standard Deviation 2.2479
|
7.1229 units on a scale
Standard Deviation 7.4904
|
5.8293 units on a scale
Standard Deviation 5.3605
|
|
UK-PBC Risk Score Value
10 years, week 52
|
4.3128 units on a scale
Standard Deviation 2.4649
|
7.2322 units on a scale
Standard Deviation 9.5794
|
5.5607 units on a scale
Standard Deviation 5.0092
|
|
UK-PBC Risk Score Value
15 years, week 12
|
8.3008 units on a scale
Standard Deviation 3.9932
|
12.4002 units on a scale
Standard Deviation 12.1785
|
10.3469 units on a scale
Standard Deviation 9.1269
|
|
UK-PBC Risk Score Value
15 years, week 52
|
7.8325 units on a scale
Standard Deviation 4.3413
|
12.3028 units on a scale
Standard Deviation 14.5701
|
9.9008 units on a scale
Standard Deviation 8.5396
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
VAS is the commonly used graphic tool for self-reporting of pruritus intensity in patients. VAS is a simple to use, validated, reliable and widely applicable tool that does not determine the impact of pruritus to quality of life. It comprises of a 100-mm horizontal line labelled as "no symptom" on left end and "worst imaginable symptom" on right end. Based on the intensity of the itch patient is instructed to draw a vertical line on the horizontal scale having a range \[VAS values (unit: mm) ranging from 0 to 100, where 0 represents "no itching" and 100 "worst possible itching"\]. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change From Baseline in Pruritus Visual Analog Score (VAS) at Week 12 and Week 52
Week 12
|
-3.7 score on a scale
Standard Deviation 6.4
|
-5.5 score on a scale
Standard Deviation 25.0
|
-12.3 score on a scale
Standard Deviation 22.3
|
|
Change From Baseline in Pruritus Visual Analog Score (VAS) at Week 12 and Week 52
Week 52
|
-3.3 score on a scale
Standard Deviation 11.7
|
-9.6 score on a scale
Standard Deviation 22.5
|
-16.5 score on a scale
Standard Deviation 23.0
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
The PBC-40 QoL questionnaire is a disease-specific health-related tool developed for measuring the psychometric profile in PBC patients. It has 10 domains and 43 questions relevant to PBC, including Cognitive, Social, Emotional Function, Fatigue, Itch, and Other Symptoms. Questions in domains: 1) digestion and diet (questions 1-3); 2) experiences (questions 4-7); 3) itching (questions 8-10); 4) fatigue (questions 11-18); 5) effort and planning (questions 19-21); 6) memory and concentration (questions 22-27); 7) affects to you as person (questions 28-33); 8) affects to your social life (questions 34-37); 9) overall impact on your life (questions 38-40); 10) general health and well-being (questions A-C). Within a domain, items are scored from 1 to 5 and the individual item scores are summed to give a total domain score. High scores represent high impact and low scores low impact of PBC on QoL (mITT Population).
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
General Symptoms, Week 12
|
1.9 units on a scale
Standard Deviation 2.7
|
-0.7 units on a scale
Standard Deviation 5.1
|
-0.5 units on a scale
Standard Deviation 3.6
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
General Symptoms, Week 52
|
0.3 units on a scale
Standard Deviation 2.4
|
-1.4 units on a scale
Standard Deviation 4.4
|
-0.1 units on a scale
Standard Deviation 4.4
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Itch, Week 12
|
-0.4 units on a scale
Standard Deviation 2.0
|
-0.5 units on a scale
Standard Deviation 3.4
|
-0.8 units on a scale
Standard Deviation 3.1
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Itch, Week 52
|
0.4 units on a scale
Standard Deviation 2.7
|
-1.3 units on a scale
Standard Deviation 3.2
|
-1.4 units on a scale
Standard Deviation 3.7
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Fatigue, Week 12
|
-1.5 units on a scale
Standard Deviation 6.1
|
-2.1 units on a scale
Standard Deviation 10.4
|
-3.0 units on a scale
Standard Deviation 5.2
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Fatigue, Week 52
|
-1.2 units on a scale
Standard Deviation 7.8
|
-3.0 units on a scale
Standard Deviation 8.5
|
-3.4 units on a scale
Standard Deviation 6.3
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Cognitive Function, Week 12
|
-0.9 units on a scale
Standard Deviation 2.4
|
-0.6 units on a scale
Standard Deviation 5.7
|
-0.7 units on a scale
Standard Deviation 2.8
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Cognitive Function, Week 52
|
-0.7 units on a scale
Standard Deviation 7.6
|
-1.4 units on a scale
Standard Deviation 5.3
|
-0.6 units on a scale
Standard Deviation 2.5
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Social, Week 12
|
0.8 units on a scale
Standard Deviation 2.8
|
-0.5 units on a scale
Standard Deviation 7.9
|
-1.0 units on a scale
Standard Deviation 6.3
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Social, Week 52
|
-3.3 units on a scale
Standard Deviation 5.2
|
-1.6 units on a scale
Standard Deviation 7.2
|
-1.6 units on a scale
Standard Deviation 6.2
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Emotional, Week 12
|
-0.9 units on a scale
Standard Deviation 1.6
|
-1.0 units on a scale
Standard Deviation 2.6
|
-0.8 units on a scale
Standard Deviation 2.1
|
|
Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52
Emotional, Week 52
|
-2.1 units on a scale
Standard Deviation 2.3
|
-1.3 units on a scale
Standard Deviation 2.7
|
-0.9 units on a scale
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percentage of participants with response based on Barcelona risk scores was defined as Normalization of ALP or a Decrease of ALP ≥ 40%. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Published PBC Response Criteria - Barcelona
Week 12
|
9.1 percentage of participants
|
42.6 percentage of participants
|
62.7 percentage of participants
|
|
Percentage of Participants Meet Published PBC Response Criteria - Barcelona
Week 52
|
45.5 percentage of participants
|
66.7 percentage of participants
|
65.3 percentage of participants
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from baseline to 12 weeks and 52 weeks in Model for End-stage Liver Disease (MELD) Score (mITT Population) The MELD score ranges from 6 to 40 and is a measure of how severe a patient's liver disease is. The higher the score, the more likely the patients will need a liver transplant. A calculated prognostic risk factor used to assess the potential need for a liver transplant. MELD(i) score = 10\*\[0.957\*ln(creatinine mg/dL) + 0. 378\*ln(total bilirubin mg/dL) + 1.120\*ln (INR) + 0.643\]. If MELD(i) is less than or equal to 11 then MELD = MELD(i). If MELD(i) is greater than 11 then MELD = MELD(i) + (1.32 \*(137 - (Na)) - (0.033\*MELD(i)\*(137 - Na))
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Absolute Change in MELD Score From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-0.3 Change from Baseline
Standard Deviation 1.6
|
0.1 Change from Baseline
Standard Deviation 1.2
|
0.1 Change from Baseline
Standard Deviation 1.0
|
|
Absolute Change in MELD Score From Baseline to 12 Weeks and 52 Weeks
Week 52
|
-0.7 Change from Baseline
Standard Deviation 1.6
|
0.3 Change from Baseline
Standard Deviation 1.1
|
0.2 Change from Baseline
Standard Deviation 1.2
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Change from Baseline to 12 weeks and 52 weeks in Global PBC Study Group (GLOBE) score (mITT Population) The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk. It is calculated from the following equation: GLOBE score = (0.044378 \* age + 0.93982 \* LN(total bilirubin/ULN) +(0.335648 \* LN(alkaline phosphatase/ULN)) - 2.266708 \* albumin /LLN -0.002581 \* platelet count per 109/L) + 1.216865
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Change in GLOBE PBC Score From Baseline to 12 Weeks and 52 Weeks
Week 12
|
-0.079 units on a scale
Standard Deviation 0.260
|
-0.292 units on a scale
Standard Deviation 0.304
|
-0.346 units on a scale
Standard Deviation 0.269
|
|
Change in GLOBE PBC Score From Baseline to 12 Weeks and 52 Weeks
Week 52
|
-0.180 units on a scale
Standard Deviation 0.217
|
-0.271 units on a scale
Standard Deviation 0.354
|
-0.404 units on a scale
Standard Deviation 0.298
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
participants with Response Based on Rotterdam Criteria at Weeks 12 and 52 Rotterdam Published PBC Response Criteria by Visit (mITT Population) Rotterdam criteria: Early (normal total bilirubin and normal albumin), Moderately advanced (either abnormal albumin or abnormal total bilirubin), and Advanced (both abnormal albumin and abnormal total bilirubin). From Early stage to Moderate Stage and to Advanced Stage, it becomes worse and worse in abnormality.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Participants Meet Rotterdam Criteria
Early Stage,12 Weeks
|
11 participants
|
38 participants
|
40 participants
|
|
Participants Meet Rotterdam Criteria
Early Stage, 52 weeks
|
10 participants
|
36 participants
|
42 participants
|
|
Participants Meet Rotterdam Criteria
Moderately advanced at 12 Weeks: Either abnormal Albumin or abnormal Total Bilirubin
|
0 participants
|
7 participants
|
9 participants
|
|
Participants Meet Rotterdam Criteria
Moderately advanced: 52 Weeks, Either abnormal Albumin or abnormal Total Bilirubin
|
0 participants
|
3 participants
|
5 participants
|
|
Participants Meet Rotterdam Criteria
Advanced: 12 Weeks, Both abnormal Albumin and abnormal Total Bilirubin
|
0 participants
|
1 participants
|
0 participants
|
|
Participants Meet Rotterdam Criteria
Advanced: 52 Weeks Both abnormal Albumin and abnormal Total Bilirubin
|
0 participants
|
3 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percentage of participants with Response Defined by Composite Endpoint (ALP\< 1.67 \* Upper Limit of Normal \[ULN\] at Endpoint, Total Bilirubin \[BIL\] within Normal Limits at Endpoint, and Greater Than Equal To \[≥\] 15% ALP Reduction) from Baseline to Week 12 and Week 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percentage of Participants Meet Composite Endpoint of AP and Total Bilirubin Criteria at Week 12 and Week 52
Week 12
|
45.5 percentage of subjects
|
48.9 percentage of subjects
|
66.7 percentage of subjects
|
|
Percentage of Participants Meet Composite Endpoint of AP and Total Bilirubin Criteria at Week 12 and Week 52
Week 52
|
63.6 percentage of subjects
|
53.3 percentage of subjects
|
67.3 percentage of subjects
|
SECONDARY outcome
Timeframe: 12 weeks and 52 weeksPopulation: mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC.
Percent change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment.
Outcome measures
| Measure |
MBX-8025 (2 mg)
n=11 Participants
MBX-8025 2 mg capsule once daily
MBX-8025 2 mg Capsule: Initial 8-week treatment:
• MBX-8025 2 mg
Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg.
|
MBX-8025 (5 mg)
n=49 Participants
MBX-8025 5 mg capsule once daily
MBX-8025 5 mg Capsule: Initial 8-week treatment:
• MBX-8025 5 mg
Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 (10 mg)
n=52 Participants
MBX-8025 10 mg capsule once daily
MBX-8025 10 mg Capsule: Initial 8-week treatment:
• MBX-8025 10 mg
Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Percent Change in Serum Alkaline Phosphatase (ALP)
Relative Change at Week 12
|
-22.56 percentage Change
Standard Deviation 13.92
|
-34.49 percentage Change
Standard Deviation 20.62
|
-43.20 percentage Change
Standard Deviation 12.39
|
|
Percent Change in Serum Alkaline Phosphatase (ALP)
Relative Change at Week 52
|
-32.72 percentage Change
Standard Deviation 22.48
|
-40.09 percentage Change
Standard Deviation 24.23
|
-44.19 percentage Change
Standard Deviation 15.52
|
Adverse Events
MBX-8025 2 mg
Serious events: 1 serious events
Other events: 11 other events
Deaths: 0 deaths
MBX-8025 5 mg
Serious events: 8 serious events
Other events: 45 other events
Deaths: 0 deaths
MBX-8025 10 mg
Serious events: 5 serious events
Other events: 47 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
MBX-8025 2 mg
n=11 participants at risk
MBX-8025 2 mg capsule once dailyMBX-8025 2 mg Capsule: Initial 8-week treatment:• MBX-8025 2 mgSubjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg
|
MBX-8025 5 mg
n=53 participants at risk
MBX-8025 5 mg capsule once dailyMBX-8025 5 mg Capsule: Initial 8-week treatment:• MBX-8025 5 mgSubjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 10 mg
n=55 participants at risk
MBX-8025 10 mg capsule once dailyMBX-8025 10 mg Capsule: Initial 8-week treatment:• MBX-8025 10 mgSubjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Febrile neutropenia
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Cardiac disorders
Angina pectoris
|
0.00%
0/11 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Cardiac disorders
Atrial fibrillation
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Cardiac disorders
Supraventricular tachycardia
|
0.00%
0/11 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Pneumonia
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Infections and infestations
Pyonephrosis
|
0.00%
0/11 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Femoral neck fracture
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Procedural pain
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Rib fracture
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Metabolism and nutrition disorders
Metabolic acidosis
|
0.00%
0/11 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Intervertebral disc protrusion
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Nervous system disorders
Syncope
|
0.00%
0/11 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
Other adverse events
| Measure |
MBX-8025 2 mg
n=11 participants at risk
MBX-8025 2 mg capsule once dailyMBX-8025 2 mg Capsule: Initial 8-week treatment:• MBX-8025 2 mgSubjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg
|
MBX-8025 5 mg
n=53 participants at risk
MBX-8025 5 mg capsule once dailyMBX-8025 5 mg Capsule: Initial 8-week treatment:• MBX-8025 5 mgSubjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment.
|
MBX-8025 10 mg
n=55 participants at risk
MBX-8025 10 mg capsule once dailyMBX-8025 10 mg Capsule: Initial 8-week treatment:• MBX-8025 10 mgSubjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
0.00%
0/11 • Up to Week 56
|
5.7%
3/53 • Number of events 3 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Cardiac disorders
Palpitations
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Eye disorders
Dry eye
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
7.3%
4/55 • Number of events 4 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal discomfort
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
5.7%
3/53 • Number of events 3 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal distension
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
7.5%
4/53 • Number of events 4 • Up to Week 56
|
7.3%
4/55 • Number of events 4 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal pain
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
5.7%
3/53 • Number of events 3 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal pain upper
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
13.2%
7/53 • Number of events 8 • Up to Week 56
|
9.1%
5/55 • Number of events 5 • Up to Week 56
|
|
Gastrointestinal disorders
Abdominal tenderness
|
0.00%
0/11 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/11 • Up to Week 56
|
7.5%
4/53 • Number of events 5 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Gastrointestinal disorders
Diarrhoea
|
36.4%
4/11 • Number of events 4 • Up to Week 56
|
13.2%
7/53 • Number of events 7 • Up to Week 56
|
16.4%
9/55 • Number of events 9 • Up to Week 56
|
|
Gastrointestinal disorders
Dry mouth
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
7.3%
4/55 • Number of events 4 • Up to Week 56
|
|
Gastrointestinal disorders
Dyschezia
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Gastrointestinal disorders
Dyspepsia
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
3.8%
2/53 • Number of events 4 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Gastrointestinal disorders
Flatulence
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
9.4%
5/53 • Number of events 7 • Up to Week 56
|
9.1%
5/55 • Number of events 5 • Up to Week 56
|
|
Gastrointestinal disorders
Haematochezia
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Gastrointestinal disorders
Nausea
|
36.4%
4/11 • Number of events 8 • Up to Week 56
|
17.0%
9/53 • Number of events 11 • Up to Week 56
|
10.9%
6/55 • Number of events 6 • Up to Week 56
|
|
Gastrointestinal disorders
Vomiting
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
7.5%
4/53 • Number of events 6 • Up to Week 56
|
9.1%
5/55 • Number of events 5 • Up to Week 56
|
|
General disorders
Asthenia
|
9.1%
1/11 • Number of events 2 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
General disorders
Chest discomfort
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 2 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
General disorders
Fatigue
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
17.0%
9/53 • Number of events 10 • Up to Week 56
|
10.9%
6/55 • Number of events 6 • Up to Week 56
|
|
General disorders
Feeling of body temperature change
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
General disorders
Influenza like illness
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
General disorders
Oedema peripheral
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 3 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
General disorders
Peripheral swelling
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
General disorders
Pyrexia
|
0.00%
0/11 • Up to Week 56
|
5.7%
3/53 • Number of events 3 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Hepatobiliary disorders
Biliary colic
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Hepatobiliary disorders
Cholelithiasis
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Immune system disorders
Allergy to arthropod sting
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Immune system disorders
Allergy to vaccine
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Bronchitis
|
0.00%
0/11 • Up to Week 56
|
5.7%
3/53 • Number of events 3 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Infections and infestations
Cellulitis
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Gastrointestinal viral infection
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Influenza
|
0.00%
0/11 • Up to Week 56
|
9.4%
5/53 • Number of events 5 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Infections and infestations
Nasopharyngitis
|
36.4%
4/11 • Number of events 5 • Up to Week 56
|
9.4%
5/53 • Number of events 7 • Up to Week 56
|
10.9%
6/55 • Number of events 9 • Up to Week 56
|
|
Infections and infestations
Rash pustular
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Sinusitis
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 2 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Infections and infestations
Upper respiratory tract infection
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
9.4%
5/53 • Number of events 6 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Infections and infestations
Urinary tract infection
|
9.1%
1/11 • Number of events 2 • Up to Week 56
|
15.1%
8/53 • Number of events 11 • Up to Week 56
|
14.5%
8/55 • Number of events 12 • Up to Week 56
|
|
Infections and infestations
Viral infection
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Infections and infestations
Viral upper respiratory tract infection
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Contusion
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Fall
|
9.1%
1/11 • Number of events 2 • Up to Week 56
|
1.9%
1/53 • Number of events 2 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Injury, poisoning and procedural complications
Muscle strain
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
7.3%
4/55 • Number of events 4 • Up to Week 56
|
|
Investigations
Troponin increased
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Metabolism and nutrition disorders
Decreased appetite
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
11.3%
6/53 • Number of events 6 • Up to Week 56
|
12.7%
7/55 • Number of events 8 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
27.3%
3/11 • Number of events 3 • Up to Week 56
|
9.4%
5/53 • Number of events 5 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
5.7%
3/53 • Number of events 6 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
7.3%
4/55 • Number of events 5 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/11 • Up to Week 56
|
9.4%
5/53 • Number of events 6 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Melanocytic naevus
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Nervous system disorders
Dizziness
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
9.4%
5/53 • Number of events 5 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Nervous system disorders
Headache
|
18.2%
2/11 • Number of events 5 • Up to Week 56
|
11.3%
6/53 • Number of events 9 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Nervous system disorders
Memory impairment
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Nervous system disorders
Paraesthesia
|
27.3%
3/11 • Number of events 5 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Psychiatric disorders
Insomnia
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Psychiatric disorders
Nightmare
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Renal and urinary disorders
Chromaturia
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Renal and urinary disorders
Pollakiuria
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Renal and urinary disorders
Urine odour abnormal
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Reproductive system and breast disorders
Pruritus genital
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
7.5%
4/53 • Number of events 4 • Up to Week 56
|
5.5%
3/55 • Number of events 3 • Up to Week 56
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
18.2%
2/11 • Number of events 2 • Up to Week 56
|
1.9%
1/53 • Number of events 2 • Up to Week 56
|
7.3%
4/55 • Number of events 4 • Up to Week 56
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
1.9%
1/53 • Number of events 1 • Up to Week 56
|
3.6%
2/55 • Number of events 2 • Up to Week 56
|
|
Skin and subcutaneous tissue disorders
Erythema
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
0.00%
0/53 • Up to Week 56
|
1.8%
1/55 • Number of events 1 • Up to Week 56
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
54.5%
6/11 • Number of events 9 • Up to Week 56
|
20.8%
11/53 • Number of events 13 • Up to Week 56
|
21.8%
12/55 • Number of events 12 • Up to Week 56
|
|
Vascular disorders
Hypertension
|
9.1%
1/11 • Number of events 1 • Up to Week 56
|
3.8%
2/53 • Number of events 2 • Up to Week 56
|
0.00%
0/55 • Up to Week 56
|
Additional Information
Elaine Watkins, DO, MSPH, Vice President of Clinical Development
CymaBay Therapeutics, Inc.
Phone: 510-293-8800
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place