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Trial Outcomes & Findings for Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis (NCT NCT05642468)

NCT ID: NCT05642468

Last Updated: 2026-05-04

Results Overview

An adverse event (AE) was any untoward medical occurrence in an enrolled participant regardless of causal relationship with study drug. An SAE was defined as any untoward medical occurrence that, at any dose, resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or an important medical event. A TEAE was any AE or worsening of an existing disease that occurred after the first dose of study drug and within the 14-day follow-up.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

From first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days

Results posted on

2026-05-04

Participant Flow

This Phase II, open-label study was conducted at 8 investigational sites in 4 countries in adults with primary sclerosing cholangitis (PSC). A total of 18 participants were enrolled in the study.

The study consisted of a 2-week screening period followed by administration of a single dose of A3907 followed by a 2-week period to confirm if the target exposure was reached before beginning a treatment period of 12 weeks. The study was terminated early due to Sponsor decision, not due to any safety concerns. No participant was enrolled in A3907 30 milligram (mg) twice daily (BID) without clinically relevant stricture (CRS) arm as the study was terminated prior to enrollment in that arm.

Participant milestones

Participant milestones
Measure
A3907 10 mg QD
Participants received A3907 10 mg (1x10 mg tablet) once daily (QD) on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Overall Study
STARTED
8
6
4
Overall Study
COMPLETED
6
3
1
Overall Study
NOT COMPLETED
2
3
3

Reasons for withdrawal

Reasons for withdrawal
Measure
A3907 10 mg QD
Participants received A3907 10 mg (1x10 mg tablet) once daily (QD) on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Overall Study
Adverse Event
0
0
1
Overall Study
Withdrawal by Subject
0
2
0
Overall Study
Exposure level exceeded safety margins
2
0
0
Overall Study
Study terminated by Sponsor
0
1
2

Baseline Characteristics

Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
A3907 10 mg QD
n=8 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Total
n=18 Participants
Total of all reporting groups
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Age, Continuous
42.3 years
STANDARD_DEVIATION 9.82 • n=54 Participants
43.0 years
STANDARD_DEVIATION 11.08 • n=60 Participants
37.8 years
STANDARD_DEVIATION 14.57 • n=114 Participants
41.5 years
STANDARD_DEVIATION 10.85 • n=1 Participants
Sex: Female, Male
Female
0 Participants
n=54 Participants
1 Participants
n=60 Participants
0 Participants
n=114 Participants
1 Participants
n=1 Participants
Sex: Female, Male
Male
8 Participants
n=54 Participants
5 Participants
n=60 Participants
4 Participants
n=114 Participants
17 Participants
n=1 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=54 Participants
1 Participants
n=60 Participants
0 Participants
n=114 Participants
2 Participants
n=1 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
7 Participants
n=54 Participants
5 Participants
n=60 Participants
4 Participants
n=114 Participants
16 Participants
n=1 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Race (NIH/OMB)
Asian
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Race (NIH/OMB)
White
8 Participants
n=54 Participants
6 Participants
n=60 Participants
4 Participants
n=114 Participants
18 Participants
n=1 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=54 Participants
0 Participants
n=60 Participants
0 Participants
n=114 Participants
0 Participants
n=1 Participants

PRIMARY outcome

Timeframe: From first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days

Population: The safety analysis set included all participants who received at least 1 dose of A3907.

An adverse event (AE) was any untoward medical occurrence in an enrolled participant regardless of causal relationship with study drug. An SAE was defined as any untoward medical occurrence that, at any dose, resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or an important medical event. A TEAE was any AE or worsening of an existing disease that occurred after the first dose of study drug and within the 14-day follow-up.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=8 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
TEAEs
5 Participants
6 Participants
3 Participants
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
TESAEs
0 Participants
1 Participants
0 Participants

SECONDARY outcome

Timeframe: Pre-dose and 0.5, 1, 1.5, 2, 4, 6, 8, 10 hours post-dose on Days 0 and 98

Population: The PK analysis set included all participants who received at least 1 dose of A3907 and had evaluable PK data (i.e. with no major protocol deviation affecting the PK variables). During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported.

Blood samples were collected at specified timepoints to assess Cmax of A3907. The pharmacokinetic (PK) analysis was conducted after single dose (Day 0) and repeated dose administration (Day 98).

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=8 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Maximum Observed Plasma Concentration (Cmax) of A3907
Day 98
35.9 nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation 71.9
134 nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation 22.4
NA nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation NA
NA indicates that values are not estimable for a single participant.
Maximum Observed Plasma Concentration (Cmax) of A3907
Day 0
47.1 nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation 67.9
142 nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation 53.7
78.3 nanogram/milliliter (ng/mL)
Geometric Coefficient of Variation 240.2

SECONDARY outcome

Timeframe: Pre-dose and 0.5, 1, 1.5, 2, 4, 6, 8, 10 hours post-dose on Days 0 and 98

Population: The PK analysis set included all participants who received at least 1 dose of A3907 and had evaluable PK data (i.e. with no major protocol deviation affecting the PK variables). During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported.

Blood samples were collected at specified timepoints to assess AUC0-t of A3907.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=8 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Area Under the Plasma Concentration-time Curve From Time Zero to Time t (Time of Last Quantifiable Plasma Concentration) (AUC0-t) of A3907
Day 98
223 hour*ng/mL
Geometric Coefficient of Variation 61.1
936 hour*ng/mL
Geometric Coefficient of Variation 9.3
NA hour*ng/mL
Geometric Coefficient of Variation NA
NA indicates that values are not estimable for a single participant.
Area Under the Plasma Concentration-time Curve From Time Zero to Time t (Time of Last Quantifiable Plasma Concentration) (AUC0-t) of A3907
Day 0
309 hour*ng/mL
Geometric Coefficient of Variation 76.4
866 hour*ng/mL
Geometric Coefficient of Variation 58.0
604 hour*ng/mL
Geometric Coefficient of Variation 189.4

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 12

Population: The pharmacodynamic (PD) analysis set included all participants who received at least 1 dose of A3907 and for whom at least 1 PD marker was evaluated. During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported. Unconjugated bile acids include CA, LCA, DCA, CDCA, UDCA. Sulfated bile acids include respective conjugated and unconjugated urine sulfated bile acids.

Blood and urine samples were collected at specified timepoints to assess serum and urine individual and total bile acid levels. Baseline=average of all screening results and results on day of planned single dose if both results were available. Otherwise, baseline=last non-missing assessment prior to study drug administration of planned single dose. CA=Cholic Acid; LCA=lithocholic acid; DCA=deoxycholic acid; CDCA=chenodeoxycholic acid; UDCA= ursodeoxycholic acid; GCA=glycocholic acid; GLCA=glycolithocholic acid; GDCA=glycodeoxycholic acid; GCDCA=glycochenodeoxycholic acid; GUDCA= glycoursodeoxycholic acid; TCA=taurocholic acid; TCDCA=taurochenodeoxycholic acid; TDCA=taurodeoxycholic acid; TLCA=taurolithocholic acid; TUDCA=tauroursodeoxycholic acid; S=sulfate. Calculated total bile acids without all UDCA bile acids is the sum of all individual bile acids excluding all UDCAs (UDCA, GUDCA, TUDCA). Conjugated bile acids include GCA, GLCA, GDCA, GCDCA, GUDCA, TCA, TCDCA, TDCA, TLCA, TUDCA.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=6 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=4 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=2 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine DCA-S
0.0954 micromole/liter (umol/L)
Standard Deviation 0.2697
0.0327 micromole/liter (umol/L)
Standard Deviation 0.0915
-0.0315 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Unconjugated urine sulfated bile acids
-0.6305 micromole/liter (umol/L)
Standard Deviation 5.8407
1.5471 micromole/liter (umol/L)
Standard Deviation 3.8482
0.0225 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum CA
0.4885 micromole/liter (umol/L)
Standard Deviation 0.9331
-0.1753 micromole/liter (umol/L)
Standard Deviation 0.5502
0.0560 micromole/liter (umol/L)
Standard Deviation 0.0550
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum LCA
0.0151 micromole/liter (umol/L)
Standard Deviation 0.0484
0.0261 micromole/liter (umol/L)
Standard Deviation 0.0655
-0.0104 micromole/liter (umol/L)
Standard Deviation 0.0146
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum DCA
0.2717 micromole/liter (umol/L)
Standard Deviation 0.4695
0.2679 micromole/liter (umol/L)
Standard Deviation 0.3591
-0.0456 micromole/liter (umol/L)
Standard Deviation 0.0528
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum CDCA
0.8167 micromole/liter (umol/L)
Standard Deviation 1.1989
0.0039 micromole/liter (umol/L)
Standard Deviation 0.1233
0.1613 micromole/liter (umol/L)
Standard Deviation 0.2870
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum UDCA
4.6122 micromole/liter (umol/L)
Standard Deviation 6.0338
-3.5851 micromole/liter (umol/L)
Standard Deviation 5.7702
-1.1110 micromole/liter (umol/L)
Standard Deviation 1.4305
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum GCA
1.3351 micromole/liter (umol/L)
Standard Deviation 2.3384
-1.8013 micromole/liter (umol/L)
Standard Deviation 5.2396
0.3603 micromole/liter (umol/L)
Standard Deviation 0.0326
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum GLCA
0.0175 micromole/liter (umol/L)
Standard Deviation 0.1967
0.0325 micromole/liter (umol/L)
Standard Deviation 0.3491
-0.0212 micromole/liter (umol/L)
Standard Deviation 0.0300
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum GDCA
2.1247 micromole/liter (umol/L)
Standard Deviation 2.8012
5.5493 micromole/liter (umol/L)
Standard Deviation 13.3255
-0.3383 micromole/liter (umol/L)
Standard Deviation 0.8731
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum GCDCA
5.0845 micromole/liter (umol/L)
Standard Deviation 7.6696
2.4643 micromole/liter (umol/L)
Standard Deviation 13.6973
4.2567 micromole/liter (umol/L)
Standard Deviation 6.7475
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum GUDCA
11.5201 micromole/liter (umol/L)
Standard Deviation 14.0272
4.2602 micromole/liter (umol/L)
Standard Deviation 28.0844
-4.6380 micromole/liter (umol/L)
Standard Deviation 1.2898
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum TCA
0.2912 micromole/liter (umol/L)
Standard Deviation 0.7627
-3.0008 micromole/liter (umol/L)
Standard Deviation 4.1924
-0.7998 micromole/liter (umol/L)
Standard Deviation 1.5993
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum TCDCA
0.4840 micromole/liter (umol/L)
Standard Deviation 0.9149
-1.3761 micromole/liter (umol/L)
Standard Deviation 3.6052
0.1130 micromole/liter (umol/L)
Standard Deviation 0.3271
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum TDCA
0.1295 micromole/liter (umol/L)
Standard Deviation 0.2088
-0.1607 micromole/liter (umol/L)
Standard Deviation 1.4477
-0.1464 micromole/liter (umol/L)
Standard Deviation 0.3922
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum TLCA
-0.0002 micromole/liter (umol/L)
Standard Deviation 0.0385
-0.0508 micromole/liter (umol/L)
Standard Deviation 0.1124
-0.0054 micromole/liter (umol/L)
Standard Deviation 0.0077
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum TUDCA
0.9627 micromole/liter (umol/L)
Standard Deviation 1.5129
-1.8607 micromole/liter (umol/L)
Standard Deviation 4.4925
-1.2759 micromole/liter (umol/L)
Standard Deviation 0.1619
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum total bile acids-KI
28.1533 micromole/liter (umol/L)
Standard Deviation 36.3683
0.5934 micromole/liter (umol/L)
Standard Deviation 65.2963
-3.4447 micromole/liter (umol/L)
Standard Deviation 6.4230
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum total bile acids-GCL
32.7500 micromole/liter (umol/L)
Standard Deviation 48.0778
8.3750 micromole/liter (umol/L)
Standard Deviation 86.8028
0.6667 micromole/liter (umol/L)
Standard Deviation 4.2426
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Serum total bile acids (calculated) without all UDCAs bile acid
11.0582 micromole/liter (umol/L)
Standard Deviation 16.8346
1.7790 micromole/liter (umol/L)
Standard Deviation 38.0268
3.5803 micromole/liter (umol/L)
Standard Deviation 3.8647
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Conjugated serum bile acids
21.9490 micromole/liter (umol/L)
Standard Deviation 29.8683
4.0558 micromole/liter (umol/L)
Standard Deviation 69.7236
-2.4951 micromole/liter (umol/L)
Standard Deviation 4.6887
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Unconjugated serum bile acids
6.2043 micromole/liter (umol/L)
Standard Deviation 8.1301
-3.4624 micromole/liter (umol/L)
Standard Deviation 6.1819
-0.9497 micromole/liter (umol/L)
Standard Deviation 1.7343
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine CA
0.4831 micromole/liter (umol/L)
Standard Deviation 0.3881
0.0657 micromole/liter (umol/L)
Standard Deviation 0.0842
0.1562 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine LCA
0.0088 micromole/liter (umol/L)
Standard Deviation 0.0140
0.0049 micromole/liter (umol/L)
Standard Deviation 0.0104
0.0000 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine DCA
0.0248 micromole/liter (umol/L)
Standard Deviation 0.0298
0.0254 micromole/liter (umol/L)
Standard Deviation 0.0348
0.0000 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine CDCA
0.0103 micromole/liter (umol/L)
Standard Deviation 0.0087
0.0020 micromole/liter (umol/L)
Standard Deviation 0.0087
0.0000 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine UDCA
0.1338 micromole/liter (umol/L)
Standard Deviation 0.1513
0.0935 micromole/liter (umol/L)
Standard Deviation 0.2295
0.0183 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GCA
0.4344 micromole/liter (umol/L)
Standard Deviation 0.6883
-0.6828 micromole/liter (umol/L)
Standard Deviation 1.8381
0.2483 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GLCA
0.0058 micromole/liter (umol/L)
Standard Deviation 0.0091
-0.0001 micromole/liter (umol/L)
Standard Deviation 0.0002
0.0000 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GDCA
0.1260 micromole/liter (umol/L)
Standard Deviation 0.1397
0.0191 micromole/liter (umol/L)
Standard Deviation 0.4760
-0.0102 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GCDCA
0.0606 micromole/liter (umol/L)
Standard Deviation 0.0939
-0.0682 micromole/liter (umol/L)
Standard Deviation 0.2017
0.0162 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GUDCA
17.6985 micromole/liter (umol/L)
Standard Deviation 24.4586
-2.6098 micromole/liter (umol/L)
Standard Deviation 22.9701
0.6844 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine CA-S
-0.0656 micromole/liter (umol/L)
Standard Deviation 0.1611
-0.0072 micromole/liter (umol/L)
Standard Deviation 0.0076
0.0041 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine LCA-S
0.0933 micromole/liter (umol/L)
Standard Deviation 0.0903
0.2142 micromole/liter (umol/L)
Standard Deviation 0.4239
0.0000 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine CDCA-S
-0.0176 micromole/liter (umol/L)
Standard Deviation 0.1047
0.1086 micromole/liter (umol/L)
Standard Deviation 0.2254
-0.0116 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine UDCA-S
-0.7360 micromole/liter (umol/L)
Standard Deviation 5.5327
1.1988 micromole/liter (umol/L)
Standard Deviation 3.4228
0.0616 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GCA-S
-0.1996 micromole/liter (umol/L)
Standard Deviation 0.9580
-2.1408 micromole/liter (umol/L)
Standard Deviation 3.5941
-0.0137 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GLCA-S
-2.2804 micromole/liter (umol/L)
Standard Deviation 21.4492
-11.3853 micromole/liter (umol/L)
Standard Deviation 13.7351
-0.0568 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GDCA-S
6.3690 micromole/liter (umol/L)
Standard Deviation 16.0747
-10.6307 micromole/liter (umol/L)
Standard Deviation 25.1248
-1.0202 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GCDCA-S
1.4066 micromole/liter (umol/L)
Standard Deviation 4.4791
-3.9908 micromole/liter (umol/L)
Standard Deviation 7.1694
-0.0223 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine GUDCA-S
-1.6910 micromole/liter (umol/L)
Standard Deviation 126.5646
14.1540 micromole/liter (umol/L)
Standard Deviation 37.1691
-2.1189 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine total bile acids
21.9601 micromole/liter (umol/L)
Standard Deviation 179.9461
-15.5970 micromole/liter (umol/L)
Standard Deviation 71.0133
-2.0963 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Urine total bile acids (calculated) without all UDCAs bile acid
6.5548 micromole/liter (umol/L)
Standard Deviation 39.9565
-28.4334 micromole/liter (umol/L)
Standard Deviation 51.0251
-0.7417 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Conjugated urine bile acids
18.3253 micromole/liter (umol/L)
Standard Deviation 25.3490
-3.3419 micromole/liter (umol/L)
Standard Deviation 25.4237
0.9387 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Unconjugated urine bile acids
0.6608 micromole/liter (umol/L)
Standard Deviation 0.5074
0.1914 micromole/liter (umol/L)
Standard Deviation 0.3603
0.1745 micromole/liter (umol/L)
Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels
Conjugated urine sulfated bile acids
3.6045 micromole/liter (umol/L)
Standard Deviation 157.4674
-13.9937 micromole/liter (umol/L)
Standard Deviation 47.9874
-3.2320 micromole/liter (umol/L)

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 12

Population: The safety analysis set included all participants who received at least 1 dose of A3907. During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported.

Blood samples were collected at specified timepoints to assess LBTs. Baseline was defined as the average of all screening results and results on the day of the planned single dose if both results were available. Otherwise, baseline was defined as the last non-missing assessment prior to the study drug administration of planned single dose.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=6 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=4 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=2 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Change From Baseline to Week 12 in Liver Biochemical Tests (LBTs): Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Gamma-glutamyl Transferase (GGT) and Alkaline Phosphatase (ALP)
ALT
1.500 Units/liter
Standard Deviation 37.3698
-14.500 Units/liter
Standard Deviation 47.0815
0.333 Units/liter
Standard Deviation 41.0122
Change From Baseline to Week 12 in Liver Biochemical Tests (LBTs): Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Gamma-glutamyl Transferase (GGT) and Alkaline Phosphatase (ALP)
AST
0.833 Units/liter
Standard Deviation 33.4644
-13.000 Units/liter
Standard Deviation 25.8618
-7.167 Units/liter
Standard Deviation 22.3917
Change From Baseline to Week 12 in Liver Biochemical Tests (LBTs): Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Gamma-glutamyl Transferase (GGT) and Alkaline Phosphatase (ALP)
GGT
50.500 Units/liter
Standard Deviation 49.8849
-39.500 Units/liter
Standard Deviation 132.6292
68.500 Units/liter
Standard Deviation 37.9481
Change From Baseline to Week 12 in Liver Biochemical Tests (LBTs): Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Gamma-glutamyl Transferase (GGT) and Alkaline Phosphatase (ALP)
ALP
55.417 Units/liter
Standard Deviation 78.7359
-16.375 Units/liter
Standard Deviation 53.3547
22.667 Units/liter
Standard Deviation 61.2826

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 12

Population: The safety analysis set included all participants who received at least 1 dose of A3907. During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported.

Blood samples were collected at specified timepoints to assess LBTs. Baseline was defined as the average of all screening results and results on the day of the planned single dose if both results were available. Otherwise, baseline was defined as the last non-missing assessment prior to the study drug administration of planned single dose.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=6 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=4 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=2 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Change From Baseline to Week 12 in Liver Biochemical Tests: Total and Direct Bilirubin Levels
Total bilirubin
-0.700 umol/L
Standard Deviation 2.6414
-3.988 umol/L
Standard Deviation 4.1003
-2.117 umol/L
Standard Deviation 0.3064
Change From Baseline to Week 12 in Liver Biochemical Tests: Total and Direct Bilirubin Levels
Direct bilirubin
0.425 umol/L
Standard Deviation 1.7745
-1.600 umol/L
Standard Deviation 2.8534
-0.283 umol/L
Standard Deviation 0.1179

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 12

Population: The PD analysis set included all participants who received at least 1 dose of A3907 and for whom at least 1 PD marker was evaluated. During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported

Blood samples were collected at specified timepoints to assess C4. Baseline was defined as the assessment performed on the day of the planned single dose.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=6 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=5 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=3 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Change From Baseline to Week 12 in 7α-hydroxy-4-cholesten-3-one (C4)
-2.333 microgram/liter
Standard Deviation 11.8162
-3.860 microgram/liter
Standard Deviation 18.2600
17.233 microgram/liter
Standard Deviation 27.8011

SECONDARY outcome

Timeframe: Baseline (Day 1) and Week 12

Population: The PD analysis set included all participants who received at least 1 dose of A3907 and for whom at least 1 PD marker was evaluated. During the study, participants missed few scheduled site visits for sample collection, and only participants with data collected at specific timepoints are reported.

Blood samples were collected at specified timepoints to assess FGF-19. Baseline was defined as the assessment performed on the day of the planned single dose.

Outcome measures

Outcome measures
Measure
A3907 10 mg QD
n=3 Participants
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=4 Participants
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=2 Participants
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Change From Baseline to Week 12 in Fibroblast Growth Factor 19 (FGF-19)
-23.7 ng/L
Standard Deviation 31.56
-129.3 ng/L
Standard Deviation 190.72
-6.5 ng/L
Standard Deviation 21.92

Adverse Events

A3907 10 mg QD

Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths

A3907 30 mg QD

Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths

A3907 30 mg BID With CRS

Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
A3907 10 mg QD
n=8 participants at risk
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 participants at risk
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 participants at risk
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Hepatobiliary disorders
Cholangitis
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.

Other adverse events

Other adverse events
Measure
A3907 10 mg QD
n=8 participants at risk
Participants received A3907 10 mg (1x10 mg tablet) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 10 mg QD for 12 weeks.
A3907 30 mg QD
n=6 participants at risk
Participants received A3907 30 mg (3x10 mg tablets) QD on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg QD for 12 weeks.
A3907 30 mg BID With CRS
n=4 participants at risk
Participants with CRS received A3907 30 mg (3x10 mg tablets) BID 12 hours apart on Day 0, followed by a 2-week study enrollment confirmation period, before being administered 30 mg BID for 12 weeks.
Gastrointestinal disorders
Diarrhoea
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
50.0%
3/6 • Number of events 6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
50.0%
2/4 • Number of events 3 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Gastrointestinal disorders
Abdominal pain
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
33.3%
2/6 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Gastrointestinal disorders
Flatulence
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Gastrointestinal disorders
Nausea
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Gastrointestinal disorders
Abdominal pain upper
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Gastrointestinal disorders
Haemorrhoids
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Alanine aminotransferase increased
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Aspartate aminotransferase increased
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Crystal urine present
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
50.0%
2/4 • Number of events 3 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Bile acids increased
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Blood urine present
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
Eosinophil count increased
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Investigations
White blood cells urine positive
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
General disorders
Asthenia
25.0%
2/8 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
General disorders
Pyrexia
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Infections and infestations
Bronchitis
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Infections and infestations
Sinusitis
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Infections and infestations
Urinary tract infection bacterial
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Infections and infestations
Viral upper respiratory tract infection
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Hepatobiliary disorders
Cholangitis acute
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
25.0%
1/4 • Number of events 2 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Musculoskeletal and connective tissue disorders
Arthralgia
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Musculoskeletal and connective tissue disorders
Neck pain
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Nervous system disorders
Headache
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 3 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Skin and subcutaneous tissue disorders
Urticaria
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Psychiatric disorders
Insomnia
12.5%
1/8 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/6 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
Hepatobiliary disorders
Bile duct stenosis
0.00%
0/8 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
16.7%
1/6 • Number of events 1 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.
0.00%
0/4 • Adverse events were collected from first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days. Deaths (all-cause mortality) were collected from first dose of study drug (Day 1) up to end of follow-up per participant, maximum of 30 months
Analysis was performed on the safety analysis set.

Additional Information

Medical Director

Ipsen

Phone: see email

Results disclosure agreements

  • Principal investigator is a sponsor employee Albireo will retain the ownership of all data. All proposed publications based on this study must be subject to the sponsor's approval requirements.
  • Publication restrictions are in place

Restriction type: OTHER