Trial Outcomes & Findings for This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2 (NCT NCT03566238)
NCT ID: NCT03566238
Last Updated: 2025-11-28
Results Overview
Fasting s-BA baseline was calculated as the average of the last 2 values prior to the first dose. The end value was the average of the values at Weeks 22 and 24 after the start of double-blind treatment. Participants who had at least 70% reduction in fasting s-BA from baseline to the end of treatment or reached \<=70 mcmol/L after 24 weeks of treatment were considered as responder. Participants with missing average at the end of treatment were classified as non-responder. Percentages are rounded to hundredth decimal.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
62 participants
Primary outcome timeframe
From Baseline (Day 1) up to Week 24
Results posted on
2025-11-28
Participant Flow
This Phase 3, double-blind, placebo-controlled study was conducted in children with progressive familial intrahepatic cholestasis (PFIC) at 33 study centers in 12 countries between 16 May 2018 and 28 July 2020.
The study included up to an 8-week screening period, a 24-week treatment period, and a 4-week follow-up period. A total of 62 participants were enrolled in the study.
Participant milestones
| Measure |
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
A4250 Low Dose
Capsules for oral administration \[40 microgram per kilogram (mcg/kg)\] once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
|
A4250 High Dose
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
|---|---|---|---|
|
Overall Study
STARTED
|
20
|
23
|
19
|
|
Overall Study
Received Treatment
|
20
|
23
|
19
|
|
Overall Study
COMPLETED
|
15
|
18
|
16
|
|
Overall Study
NOT COMPLETED
|
5
|
5
|
3
|
Reasons for withdrawal
| Measure |
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
A4250 Low Dose
Capsules for oral administration \[40 microgram per kilogram (mcg/kg)\] once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of ileal bile acid transporter (IBAT).
|
A4250 High Dose
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
0
|
1
|
|
Overall Study
Lack of Efficacy
|
5
|
4
|
2
|
|
Overall Study
Other
|
0
|
1
|
0
|
Baseline Characteristics
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Baseline characteristics by cohort
| Measure |
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Total
n=62 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
20 Participants
n=30 Participants
|
23 Participants
n=30 Participants
|
19 Participants
n=60 Participants
|
62 Participants
n=92 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
0 Participants
n=92 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
0 Participants
n=92 Participants
|
|
Age, Continuous
|
3.75 years
STANDARD_DEVIATION 3.853 • n=30 Participants
|
3.86 years
STANDARD_DEVIATION 3.660 • n=30 Participants
|
5.24 years
STANDARD_DEVIATION 4.188 • n=60 Participants
|
4.25 years
STANDARD_DEVIATION 3.883 • n=92 Participants
|
|
Age, Customized
Age Category 1 · 6 months to 5 years
|
16 Participants
n=30 Participants
|
17 Participants
n=30 Participants
|
14 Participants
n=60 Participants
|
47 Participants
n=92 Participants
|
|
Age, Customized
Age Category 1 · 6 to 12 years
|
3 Participants
n=30 Participants
|
5 Participants
n=30 Participants
|
4 Participants
n=60 Participants
|
12 Participants
n=92 Participants
|
|
Age, Customized
Age Category 1 · 13 to 18 years
|
1 Participants
n=30 Participants
|
1 Participants
n=30 Participants
|
1 Participants
n=60 Participants
|
3 Participants
n=92 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=30 Participants
|
12 Participants
n=30 Participants
|
11 Participants
n=60 Participants
|
31 Participants
n=92 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=30 Participants
|
11 Participants
n=30 Participants
|
8 Participants
n=60 Participants
|
31 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
17 Participants
n=30 Participants
|
18 Participants
n=30 Participants
|
17 Participants
n=60 Participants
|
52 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · Black or African American
|
0 Participants
n=30 Participants
|
2 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
2 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
1 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
1 Participants
n=60 Participants
|
2 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · American Indian or Alaska Native
|
0 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
0 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
0 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
2 Participants
n=30 Participants
|
3 Participants
n=30 Participants
|
1 Participants
n=60 Participants
|
6 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Hispanic or Latino
|
1 Participants
n=30 Participants
|
0 Participants
n=30 Participants
|
0 Participants
n=60 Participants
|
1 Participants
n=92 Participants
|
|
Race/Ethnicity, Customized
Ethnicity · Not Hispanic or Latino
|
19 Participants
n=30 Participants
|
23 Participants
n=30 Participants
|
19 Participants
n=60 Participants
|
61 Participants
n=92 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=30 Participants
|
2 participants
n=30 Participants
|
3 participants
n=60 Participants
|
8 participants
n=92 Participants
|
|
Region of Enrollment
United Kingdom
|
5 participants
n=30 Participants
|
6 participants
n=30 Participants
|
2 participants
n=60 Participants
|
13 participants
n=92 Participants
|
|
Region of Enrollment
Saudi Arabia
|
1 participants
n=30 Participants
|
0 participants
n=30 Participants
|
2 participants
n=60 Participants
|
3 participants
n=92 Participants
|
|
Region of Enrollment
Canada
|
1 participants
n=30 Participants
|
1 participants
n=30 Participants
|
0 participants
n=60 Participants
|
2 participants
n=92 Participants
|
|
Region of Enrollment
Netherlands
|
1 participants
n=30 Participants
|
0 participants
n=30 Participants
|
2 participants
n=60 Participants
|
3 participants
n=92 Participants
|
|
Region of Enrollment
Turkey
|
1 participants
n=30 Participants
|
7 participants
n=30 Participants
|
3 participants
n=60 Participants
|
11 participants
n=92 Participants
|
|
Region of Enrollment
Poland
|
2 participants
n=30 Participants
|
0 participants
n=30 Participants
|
1 participants
n=60 Participants
|
3 participants
n=92 Participants
|
|
Region of Enrollment
Italy
|
1 participants
n=30 Participants
|
1 participants
n=30 Participants
|
0 participants
n=60 Participants
|
2 participants
n=92 Participants
|
|
Region of Enrollment
Israel
|
1 participants
n=30 Participants
|
0 participants
n=30 Participants
|
1 participants
n=60 Participants
|
2 participants
n=92 Participants
|
|
Region of Enrollment
France
|
1 participants
n=30 Participants
|
3 participants
n=30 Participants
|
1 participants
n=60 Participants
|
5 participants
n=92 Participants
|
|
Region of Enrollment
Australia
|
1 participants
n=30 Participants
|
0 participants
n=30 Participants
|
0 participants
n=60 Participants
|
1 participants
n=92 Participants
|
|
Region of Enrollment
Germany
|
2 participants
n=30 Participants
|
3 participants
n=30 Participants
|
4 participants
n=60 Participants
|
9 participants
n=92 Participants
|
|
Type of PFIC
Type 1
|
5 Participants
n=30 Participants
|
7 Participants
n=30 Participants
|
5 Participants
n=60 Participants
|
17 Participants
n=92 Participants
|
|
Type of PFIC
Type 2
|
15 Participants
n=30 Participants
|
16 Participants
n=30 Participants
|
14 Participants
n=60 Participants
|
45 Participants
n=92 Participants
|
PRIMARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment.
Fasting s-BA baseline was calculated as the average of the last 2 values prior to the first dose. The end value was the average of the values at Weeks 22 and 24 after the start of double-blind treatment. Participants who had at least 70% reduction in fasting s-BA from baseline to the end of treatment or reached \<=70 mcmol/L after 24 weeks of treatment were considered as responder. Participants with missing average at the end of treatment were classified as non-responder. Percentages are rounded to hundredth decimal.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid (s-BA) Concentration From Baseline to the End of Treatment or Reaching a Level <=70 Micromoles Per Liter (Mcmol/L) After 24 Weeks of Treatment
|
43.5 percentage of participants
Interval 23.19 to 65.51
|
21.1 percentage of participants
Interval 6.05 to 45.57
|
0 percentage of participants
Interval 0.0 to 16.84
|
PRIMARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment.
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period
|
58.31 percentage of assessments
Standard Error 6.205
|
47.69 percentage of assessments
Standard Error 8.110
|
28.74 percentage of assessments
Standard Error 5.209
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 12 and 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported.
Blood samples for analysis of s-BA were drawn at all visits. Participants were to fast (water intake only was permissible) for at least 4 hours prior to the collection of samples. Exceptions could be made for infants \<12 months of age if they were unable to fast for the full 4 hours. Baseline was the average of the last 2 non-missing values of fasting s-BA concentration prior to the first dose of study treatment.
Outcome measures
| Measure |
A4250 Low Dose
n=20 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=16 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=17 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24
Week 12
|
-113.70 mcmol/L
Standard Error 38.011
|
-106.44 mcmol/L
Standard Error 41.201
|
7.44 mcmol/L
Standard Error 24.588
|
|
Change From Baseline in Fasting Serum Bile Acid at Weeks 12 and 24
Week 24
|
-145.03 mcmol/L
Standard Error 41.951
|
-72.90 mcmol/L
Standard Error 52.617
|
18.64 mcmol/L
Standard Error 31.559
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 12 and 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported.
Blood samples were collected to determine the ALT concentration. Baseline was the last available assessment before the first dose of study treatment.
Outcome measures
| Measure |
A4250 Low Dose
n=22 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=18 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=18 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Change From Baseline in Serum Alanine Aminotransferase (ALT) Concentration at Weeks 12 and 24
Week 12
|
-25.9 units/L
Standard Error 23.36
|
-13.8 units/L
Standard Error 19.42
|
1.7 units/L
Standard Error 10.50
|
|
Change From Baseline in Serum Alanine Aminotransferase (ALT) Concentration at Weeks 12 and 24
Week 24
|
-27.9 units/L
Standard Error 17.97
|
-25.3 units/L
Standard Error 22.47
|
3.7 units/L
Standard Error 4.95
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 12 and 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at Baseline and Weeks 12 and 24 are reported.
The change in the growth parameters was assessed using linear growth deficit \[height (centimeter), weight (kilogram) and body mass index (BMI) (kg/meter square)\] compared to standard growth curve (Z-score) calculated by using the software or methods from the centers for disease control (CDC) website for participants with age \>=2 years old and from the world health organization website for participants with age \<2 years old. Participants whose accurate age was not available, Z-score was not calculated. A Z-score indicated how many standard deviation's (SD) a participant's measurement (like height, weight and BMI), was from the average for their age and sex. A Z-score of 0 represents the median or 50th percentile, while positive or negative values show how far above or below average a measurement was. Baseline was the last available assessment before the first dose of study treatment.
Outcome measures
| Measure |
A4250 Low Dose
n=22 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=16 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=18 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
Height deficit: Week 12
|
0.01 z-score
Standard Error 0.108
|
-0.06 z-score
Standard Error 0.100
|
-0.03 z-score
Standard Error 0.127
|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
Height deficit: Week 24
|
0.05 z-score
Standard Error 0.105
|
0.00 z-score
Standard Error 0.163
|
-0.16 z-score
Standard Error 0.104
|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
Weight deficit: Week 12
|
0.20 z-score
Standard Error 0.078
|
0.00 z-score
Standard Error 0.100
|
0.13 z-score
Standard Error 0.066
|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
Weight deficit: Week 24
|
0.29 z-score
Standard Error 0.106
|
0.15 z-score
Standard Error 0.124
|
0.10 z-score
Standard Error 0.102
|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
BMI deficit: Week 12
|
0.23 z-score
Standard Error 0.114
|
0.08 z-score
Standard Error 0.147
|
0.18 z-score
Standard Error 0.148
|
|
Change From Baseline in Growth Parameters at Weeks 12 and 24
BMI deficit: Week 24
|
0.36 z-score
Standard Error 0.113
|
0.20 z-score
Standard Error 0.203
|
0.26 z-score
Standard Error 0.156
|
SECONDARY outcome
Timeframe: Weeks 12 and 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported.
The responder for pruritus scores was defined as a participant who achieved at least a 1-point reduction in the ObsRO pruritus score. Percentages are rounded to hundredth decimal.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM score: Week 24 (Bi-weekly score)
|
39.1 percentage of participants
Interval 19.71 to 61.46
|
33.3 percentage of participants
Interval 13.34 to 59.01
|
11.8 percentage of participants
Interval 1.46 to 36.44
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM score: Week 24 (Monthly score)
|
52.2 percentage of participants
Interval 30.59 to 73.18
|
42.1 percentage of participants
Interval 20.25 to 66.5
|
15.8 percentage of participants
Interval 3.38 to 39.58
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
PM score: Week 12 (Bi-weekly score)
|
65.2 percentage of participants
Interval 42.73 to 83.62
|
36.8 percentage of participants
Interval 16.29 to 61.64
|
10.0 percentage of participants
Interval 1.23 to 31.7
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
PM score: Week 12 (Monthly score)
|
60.9 percentage of participants
Interval 38.54 to 80.29
|
36.8 percentage of participants
Interval 16.29 to 61.64
|
10.0 percentage of participants
Interval 1.23 to 31.7
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
PM score: Week 24 (Bi-weekly score)
|
56.5 percentage of participants
Interval 34.49 to 76.81
|
33.3 percentage of participants
Interval 13.34 to 59.01
|
11.8 percentage of participants
Interval 1.46 to 36.44
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
PM score: Week 24 (Monthly score)
|
52.2 percentage of participants
Interval 30.59 to 73.18
|
31.6 percentage of participants
Interval 12.58 to 56.55
|
10.5 percentage of participants
Interval 1.3 to 33.14
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM and PM scores combined: Week 24 (Monthly score)
|
52.2 percentage of participants
Interval 30.59 to 73.18
|
31.6 percentage of participants
Interval 12.58 to 56.55
|
10.5 percentage of participants
Interval 1.3 to 33.14
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM score: Week 12 (Bi-weekly score)
|
60.9 percentage of participants
Interval 38.54 to 80.29
|
42.1 percentage of participants
Interval 20.25 to 66.5
|
10.0 percentage of participants
Interval 1.23 to 31.7
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM score: Week 12 (Monthly score)
|
56.5 percentage of participants
Interval 34.49 to 76.81
|
42.1 percentage of participants
Interval 20.25 to 66.5
|
10.0 percentage of participants
Interval 1.23 to 31.7
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM and PM scores combined: Week 12 (Bi-weekly score)
|
60.9 percentage of participants
Interval 38.54 to 80.29
|
42.1 percentage of participants
Interval 20.25 to 66.5
|
5.0 percentage of participants
Interval 0.13 to 24.87
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM and PM scores combined: Week 12 (Monthly score)
|
52.2 percentage of participants
Interval 30.59 to 73.18
|
42.1 percentage of participants
Interval 20.25 to 66.5
|
10.0 percentage of participants
Interval 1.23 to 31.7
|
|
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument at Weeks 12 and 24
AM and PM scores combined: Week 24 (Bi-weekly score)
|
43.5 percentage of participants
Interval 23.19 to 65.51
|
33.3 percentage of participants
Interval 13.34 to 59.01
|
11.8 percentage of participants
Interval 1.46 to 36.44
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported.
The sleep disturbance were recorded twice daily via the electronic diary (eDiary). Participants and/or caregivers completed the eDiary every day in the morning and in the evening. The morning diary was completed shortly after the participant woke up and was used to record nighttime itching and scratching severity, aspects of sleep disturbance, and tiredness upon waking (AM scores). The evening/bedtime diary was completed just before the participant went to bed and recorded participant's itching and scratching severity, and tiredness during the day (PM scores). Both morning and bedtime diaries included Albireo ObsRO and PRO items. Baseline was the average of 14-day scores before the first dose of study treatment.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 1 to 4
|
-14.42 percentage of days
Standard Error 5.854
|
-12.97 percentage of days
Standard Error 6.036
|
-15.79 percentage of days
Standard Error 4.700
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 5 to 8
|
-26.22 percentage of days
Standard Error 7.427
|
-13.99 percentage of days
Standard Error 7.770
|
-12.10 percentage of days
Standard Error 6.353
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 9 to 12
|
-29.22 percentage of days
Standard Error 7.593
|
-16.89 percentage of days
Standard Error 9.258
|
-13.49 percentage of days
Standard Error 6.320
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 13 to 16
|
-33.38 percentage of days
Standard Error 8.219
|
-18.16 percentage of days
Standard Error 9.403
|
-15.75 percentage of days
Standard Error 6.264
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 17 to 20
|
-32.14 percentage of days
Standard Error 8.531
|
-18.02 percentage of days
Standard Error 9.967
|
-22.02 percentage of days
Standard Error 7.999
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Seeing blood due to scratching: Weeks 21 to 24
|
-30.82 percentage of days
Standard Error 8.184
|
-15.52 percentage of days
Standard Error 10.465
|
-23.72 percentage of days
Standard Error 8.563
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 1 to 4
|
-14.73 percentage of days
Standard Error 5.683
|
-20.37 percentage of days
Standard Error 6.390
|
-0.91 percentage of days
Standard Error 1.596
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 5 to 8
|
-25.03 percentage of days
Standard Error 7.527
|
-31.49 percentage of days
Standard Error 10.209
|
-2.35 percentage of days
Standard Error 2.397
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 9 to 12
|
-31.03 percentage of days
Standard Error 8.061
|
-36.72 percentage of days
Standard Error 11.154
|
-0.90 percentage of days
Standard Error 1.152
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 13 to 16
|
-46.10 percentage of days
Standard Error 9.677
|
-37.76 percentage of days
Standard Error 12.013
|
-0.86 percentage of days
Standard Error 1.392
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 17 to 20
|
-45.65 percentage of days
Standard Error 9.524
|
-40.51 percentage of days
Standard Error 12.566
|
-6.27 percentage of days
Standard Error 3.862
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Help falling asleep: Weeks 21 to 24
|
-51.75 percentage of days
Standard Error 9.857
|
-32.58 percentage of days
Standard Error 14.573
|
-3.19 percentage of days
Standard Error 2.890
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 1 to 4
|
-12.92 percentage of days
Standard Error 5.911
|
-21.88 percentage of days
Standard Error 6.228
|
-1.15 percentage of days
Standard Error 1.338
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 5 to 8
|
-17.89 percentage of days
Standard Error 7.562
|
-35.97 percentage of days
Standard Error 9.795
|
-3.76 percentage of days
Standard Error 2.773
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 9 to 12
|
-29.79 percentage of days
Standard Error 8.570
|
-41.21 percentage of days
Standard Error 10.796
|
-5.00 percentage of days
Standard Error 2.819
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 13 to 16
|
-42.79 percentage of days
Standard Error 10.364
|
-40.23 percentage of days
Standard Error 11.611
|
-2.12 percentage of days
Standard Error 3.006
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 17 to 20
|
-44.96 percentage of days
Standard Error 10.485
|
-40.13 percentage of days
Standard Error 12.324
|
-4.73 percentage of days
Standard Error 3.742
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Soothing: Weeks 21 to 24
|
-51.48 percentage of days
Standard Error 10.323
|
-34.87 percentage of days
Standard Error 13.369
|
-7.64 percentage of days
Standard Error 6.182
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 1 to 4
|
-15.30 percentage of days
Standard Error 6.396
|
-24.57 percentage of days
Standard Error 7.024
|
-4.49 percentage of days
Standard Error 1.961
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 5 to 8
|
-19.73 percentage of days
Standard Error 7.061
|
-35.58 percentage of days
Standard Error 10.053
|
-5.93 percentage of days
Standard Error 3.649
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 9 to 12
|
-26.12 percentage of days
Standard Error 9.543
|
-36.00 percentage of days
Standard Error 10.361
|
-5.74 percentage of days
Standard Error 3.585
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 13 to 16
|
-40.55 percentage of days
Standard Error 10.501
|
-37.52 percentage of days
Standard Error 11.083
|
-4.96 percentage of days
Standard Error 3.481
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 17 to 20
|
-42.20 percentage of days
Standard Error 10.247
|
-35.14 percentage of days
Standard Error 11.563
|
-2.59 percentage of days
Standard Error 3.688
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Sleeping with the caregiver: Weeks 21 to 24
|
-49.35 percentage of days
Standard Error 10.466
|
-33.14 percentage of days
Standard Error 11.801
|
-5.45 percentage of days
Standard Error 4.844
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 1 to 4
|
-0.61 percentage of days
Standard Error 1.913
|
0.98 percentage of days
Standard Error 2.320
|
-1.78 percentage of days
Standard Error 1.921
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 5 to 8
|
0.18 percentage of days
Standard Error 3.018
|
-2.43 percentage of days
Standard Error 3.036
|
1.44 percentage of days
Standard Error 4.292
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 9 to 12
|
1.31 percentage of days
Standard Error 3.656
|
-5.74 percentage of days
Standard Error 4.967
|
2.40 percentage of days
Standard Error 4.271
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 13 to 16
|
-1.61 percentage of days
Standard Error 2.556
|
-4.58 percentage of days
Standard Error 4.681
|
0.16 percentage of days
Standard Error 4.647
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 17 to 20
|
-1.99 percentage of days
Standard Error 2.141
|
-3.26 percentage of days
Standard Error 6.403
|
3.53 percentage of days
Standard Error 5.384
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument Over the 24-Week Treatment Period
Taking medications to induce sleep: Weeks 21 to 24
|
-0.03 percentage of days
Standard Error 2.251
|
2.04 percentage of days
Standard Error 8.413
|
5.50 percentage of days
Standard Error 5.307
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported.
The ObsRO and electronic diary (eDiary) PRO scale called PRUCISION was used to assess pruritus and sleep outcomes. Participants and/or caregivers completed the eDiary every day in the morning (AM scores) and in the evening (PM scores). Specifically, for difficulty falling or staying asleep, the following 2 respective questions were asked in the Morning Daily eDiary: 1) How hard was it to fall asleep last night because of your itching. and 2) How hard was it to stay asleep last night because of your itching. The PRO was a 5-point scale, and scores ranged from 0 (no itching) to 28 (the worst itching), where higher scores indicated a greater amount of itching, sleep disturbance, and tiredness. No subscales were used. Change in sleep parameters measured with the Albireo PRO and ObsRO instruments by each 4-week interval over the 24-week treatment period was assessed. Baseline was the average of 14-day scores before the first dose of study treatment.
Outcome measures
| Measure |
A4250 Low Dose
n=2 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=5 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=2 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 1 to 4
|
-0.72 scores on a scale
Standard Error 0.494
|
-0.59 scores on a scale
Standard Error 0.394
|
-0.46 scores on a scale
Standard Error 0.143
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 5 to 8
|
-1.40 scores on a scale
Standard Error 1.009
|
-0.65 scores on a scale
Standard Error 0.571
|
-0.34 scores on a scale
Standard Error 0.018
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 9 to 12
|
-2.31 scores on a scale
Standard Error 0.499
|
-0.95 scores on a scale
Standard Error 0.650
|
-0.38 scores on a scale
Standard Error 0.339
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 13 to 16
|
-2.07 scores on a scale
Standard Error 0.863
|
-1.01 scores on a scale
Standard Error 0.665
|
-0.16 scores on a scale
Standard Error 0.268
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 17 to 20
|
-2.12 scores on a scale
Standard Error 0.974
|
-1.02 scores on a scale
Standard Error 0.710
|
-0.52 scores on a scale
Standard Error 0.228
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty falling asleep: Weeks 21 to 24
|
-2.22 scores on a scale
Standard Error 0.823
|
-0.82 scores on a scale
Standard Error 0.658
|
-0.25 scores on a scale
Standard Error 0.179
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 1 to 4
|
-0.70 scores on a scale
Standard Error 0.548
|
-0.43 scores on a scale
Standard Error 0.440
|
-0.13 scores on a scale
Standard Error 0.411
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 5 to 8
|
-1.48 scores on a scale
Standard Error 0.932
|
-0.50 scores on a scale
Standard Error 0.553
|
0.09 scores on a scale
Standard Error 0.518
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 9 to 12
|
-2.51 scores on a scale
Standard Error 0.352
|
-0.77 scores on a scale
Standard Error 0.670
|
0.02 scores on a scale
Standard Error 0.661
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 13 to 16
|
-2.46 scores on a scale
Standard Error 0.464
|
-0.89 scores on a scale
Standard Error 0.626
|
0.13 scores on a scale
Standard Error 0.625
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 17 to 20
|
-2.36 scores on a scale
Standard Error 0.637
|
-0.95 scores on a scale
Standard Error 0.644
|
-0.15 scores on a scale
Standard Error 0.147
|
|
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome (PRO) Instrument Over the 24-Week Treatment Period
Difficulty staying asleep: Weeks 21 to 24
|
-2.55 scores on a scale
Standard Error 0.448
|
-0.78 scores on a scale
Standard Error 0.633
|
0.25 scores on a scale
Standard Error 0.429
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment.
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 4
|
51.40 percentage of assessments
Standard Error 5.818
|
42.48 percentage of assessments
Standard Error 7.508
|
33.48 percentage of assessments
Standard Error 5.881
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 8
|
54.23 percentage of assessments
Standard Error 5.887
|
48.21 percentage of assessments
Standard Error 7.479
|
32.72 percentage of assessments
Standard Error 5.871
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 12
|
56.06 percentage of assessments
Standard Error 5.922
|
52.26 percentage of assessments
Standard Error 7.693
|
31.07 percentage of assessments
Standard Error 5.374
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 12
|
58.85 percentage of assessments
Standard Error 5.738
|
49.15 percentage of assessments
Standard Error 7.428
|
32.35 percentage of assessments
Standard Error 5.945
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 16
|
58.83 percentage of assessments
Standard Error 5.925
|
48.31 percentage of assessments
Standard Error 7.624
|
30.67 percentage of assessments
Standard Error 5.522
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 18
|
58.80 percentage of assessments
Standard Error 6.004
|
48.25 percentage of assessments
Standard Error 7.809
|
29.88 percentage of assessments
Standard Error 5.462
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 20
|
58.68 percentage of assessments
Standard Error 6.079
|
48.10 percentage of assessments
Standard Error 7.975
|
29.45 percentage of assessments
Standard Error 5.376
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 4
|
48.14 percentage of assessments
Standard Error 6.161
|
47.37 percentage of assessments
Standard Error 8.439
|
31.07 percentage of assessments
Standard Error 5.436
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 8
|
51.16 percentage of assessments
Standard Error 6.129
|
51.79 percentage of assessments
Standard Error 8.001
|
31.07 percentage of assessments
Standard Error 5.213
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 16
|
56.41 percentage of assessments
Standard Error 6.183
|
50.47 percentage of assessments
Standard Error 7.660
|
29.91 percentage of assessments
Standard Error 5.246
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 18
|
56.35 percentage of assessments
Standard Error 6.249
|
49.92 percentage of assessments
Standard Error 7.800
|
28.93 percentage of assessments
Standard Error 5.224
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 20
|
56.27 percentage of assessments
Standard Error 6.293
|
49.74 percentage of assessments
Standard Error 7.993
|
28.50 percentage of assessments
Standard Error 5.206
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 24
|
55.73 percentage of assessments
Standard Error 6.396
|
48.92 percentage of assessments
Standard Error 8.197
|
28.17 percentage of assessments
Standard Error 5.276
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 4
|
54.66 percentage of assessments
Standard Error 6.594
|
37.59 percentage of assessments
Standard Error 7.629
|
35.89 percentage of assessments
Standard Error 7.055
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 8
|
57.30 percentage of assessments
Standard Error 6.478
|
44.64 percentage of assessments
Standard Error 7.804
|
34.38 percentage of assessments
Standard Error 7.290
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 12
|
61.65 percentage of assessments
Standard Error 6.330
|
46.05 percentage of assessments
Standard Error 7.884
|
33.63 percentage of assessments
Standard Error 7.404
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 16
|
61.26 percentage of assessments
Standard Error 6.205
|
46.15 percentage of assessments
Standard Error 8.178
|
31.43 percentage of assessments
Standard Error 7.006
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 18
|
61.25 percentage of assessments
Standard Error 6.225
|
46.57 percentage of assessments
Standard Error 8.363
|
30.83 percentage of assessments
Standard Error 6.898
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 20
|
61.09 percentage of assessments
Standard Error 6.284
|
46.47 percentage of assessments
Standard Error 8.434
|
30.39 percentage of assessments
Standard Error 6.812
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 24
|
60.89 percentage of assessments
Standard Error 6.385
|
46.47 percentage of assessments
Standard Error 8.456
|
29.31 percentage of assessments
Standard Error 6.592
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment.
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo PRO instrument, only participants \>=8 years of age completed the Albireo PRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Outcome measures
| Measure |
A4250 Low Dose
n=2 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=5 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=2 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 16
|
68.30 percentage of assessments
Standard Error 23.661
|
51.25 percentage of assessments
Standard Error 18.751
|
45.54 percentage of assessments
Standard Error 5.357
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 18
|
68.65 percentage of assessments
Standard Error 24.206
|
52.06 percentage of assessments
Standard Error 19.230
|
43.65 percentage of assessments
Standard Error 1.587
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 20
|
68.57 percentage of assessments
Standard Error 25.000
|
52.43 percentage of assessments
Standard Error 19.568
|
40.71 percentage of assessments
Standard Error 0.000
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 4
|
43.75 percentage of assessments
Standard Error 25.893
|
38.93 percentage of assessments
Standard Error 15.272
|
64.29 percentage of assessments
Standard Error 8.929
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 8
|
52.68 percentage of assessments
Standard Error 25.893
|
41.96 percentage of assessments
Standard Error 16.178
|
46.88 percentage of assessments
Standard Error 12.946
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 12
|
63.39 percentage of assessments
Standard Error 18.155
|
45.00 percentage of assessments
Standard Error 16.939
|
41.96 percentage of assessments
Standard Error 10.417
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 16
|
66.74 percentage of assessments
Standard Error 18.080
|
46.96 percentage of assessments
Standard Error 17.477
|
38.62 percentage of assessments
Standard Error 3.348
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 18
|
67.66 percentage of assessments
Standard Error 18.849
|
47.14 percentage of assessments
Standard Error 17.558
|
37.50 percentage of assessments
Standard Error 0.198
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 20
|
67.32 percentage of assessments
Standard Error 20.179
|
48.07 percentage of assessments
Standard Error 18.010
|
34.82 percentage of assessments
Standard Error 0.179
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 0 to 24
|
68.88 percentage of assessments
Standard Error 19.772
|
47.65 percentage of assessments
Standard Error 17.997
|
31.70 percentage of assessments
Standard Error 1.637
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 4
|
41.07 percentage of assessments
Standard Error 23.214
|
37.86 percentage of assessments
Standard Error 16.115
|
55.36 percentage of assessments
Standard Error 8.929
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 8
|
50.00 percentage of assessments
Standard Error 21.429
|
38.93 percentage of assessments
Standard Error 16.243
|
39.29 percentage of assessments
Standard Error 14.286
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 12
|
61.31 percentage of assessments
Standard Error 11.310
|
41.43 percentage of assessments
Standard Error 16.562
|
33.93 percentage of assessments
Standard Error 10.119
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 16
|
65.18 percentage of assessments
Standard Error 12.500
|
42.68 percentage of assessments
Standard Error 16.554
|
31.70 percentage of assessments
Standard Error 1.339
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 18
|
66.67 percentage of assessments
Standard Error 13.492
|
42.22 percentage of assessments
Standard Error 16.343
|
31.35 percentage of assessments
Standard Error 1.190
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 20
|
66.07 percentage of assessments
Standard Error 15.357
|
43.71 percentage of assessments
Standard Error 16.860
|
28.93 percentage of assessments
Standard Error 0.357
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 0 to 24
|
66.72 percentage of assessments
Standard Error 16.718
|
43.24 percentage of assessments
Standard Error 16.835
|
26.19 percentage of assessments
Standard Error 1.190
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 4
|
46.43 percentage of assessments
Standard Error 28.571
|
40.00 percentage of assessments
Standard Error 15.042
|
73.21 percentage of assessments
Standard Error 8.929
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 8
|
55.36 percentage of assessments
Standard Error 30.357
|
45.00 percentage of assessments
Standard Error 16.545
|
54.46 percentage of assessments
Standard Error 11.607
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 12
|
65.48 percentage of assessments
Standard Error 25.000
|
48.57 percentage of assessments
Standard Error 17.715
|
50.00 percentage of assessments
Standard Error 10.714
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 0 to 24
|
71.04 percentage of assessments
Standard Error 22.825
|
52.06 percentage of assessments
Standard Error 19.595
|
37.20 percentage of assessments
Standard Error 2.083
|
SECONDARY outcome
Timeframe: Baseline (Day 1) and Weeks 1 to 4, Weeks 5 to 8, Weeks 9 to 12, Weeks 13 to 16, Weeks 17 to 20, and Weeks 21 to 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected at specific timepoints are reported.
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100. At each assessment, the AM or PM score was compared to the baseline AM or PM average, respectively.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 5 to 8
|
57.07 percentage of assessments
Standard Error 6.714
|
53.95 percentage of assessments
Standard Error 8.423
|
31.96 percentage of assessments
Standard Error 6.515
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 1 to 4
|
51.40 percentage of assessments
Standard Error 5.818
|
42.48 percentage of assessments
Standard Error 7.508
|
33.48 percentage of assessments
Standard Error 5.881
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 5 to 8
|
54.19 percentage of assessments
Standard Error 6.783
|
56.20 percentage of assessments
Standard Error 8.483
|
31.07 percentage of assessments
Standard Error 5.812
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 9 to 12
|
68.09 percentage of assessments
Standard Error 6.357
|
53.67 percentage of assessments
Standard Error 9.404
|
31.61 percentage of assessments
Standard Error 6.794
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 13 to 16
|
67.59 percentage of assessments
Standard Error 7.779
|
54.35 percentage of assessments
Standard Error 9.802
|
28.36 percentage of assessments
Standard Error 7.534
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 17 to 20
|
66.79 percentage of assessments
Standard Error 7.805
|
56.14 percentage of assessments
Standard Error 10.655
|
32.62 percentage of assessments
Standard Error 8.583
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM and PM scores combined: Weeks 21 to 24
|
68.57 percentage of assessments
Standard Error 6.926
|
55.12 percentage of assessments
Standard Error 10.047
|
35.42 percentage of assessments
Standard Error 8.783
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 1 to 4
|
48.14 percentage of assessments
Standard Error 6.161
|
47.37 percentage of assessments
Standard Error 8.439
|
31.07 percentage of assessments
Standard Error 5.436
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 9 to 12
|
65.84 percentage of assessments
Standard Error 6.854
|
55.95 percentage of assessments
Standard Error 9.383
|
31.07 percentage of assessments
Standard Error 6.747
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 13 to 16
|
66.07 percentage of assessments
Standard Error 8.042
|
53.57 percentage of assessments
Standard Error 9.550
|
29.62 percentage of assessments
Standard Error 8.502
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 17 to 20
|
64.11 percentage of assessments
Standard Error 8.121
|
55.58 percentage of assessments
Standard Error 11.171
|
30.24 percentage of assessments
Standard Error 8.733
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
AM score: Weeks 21 to 24
|
64.37 percentage of assessments
Standard Error 7.324
|
54.35 percentage of assessments
Standard Error 10.801
|
37.30 percentage of assessments
Standard Error 10.909
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 1 to 4
|
54.66 percentage of assessments
Standard Error 6.594
|
37.59 percentage of assessments
Standard Error 7.629
|
35.89 percentage of assessments
Standard Error 7.055
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 5 to 8
|
59.94 percentage of assessments
Standard Error 7.355
|
51.69 percentage of assessments
Standard Error 9.051
|
32.86 percentage of assessments
Standard Error 8.138
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 9 to 12
|
70.34 percentage of assessments
Standard Error 6.816
|
51.39 percentage of assessments
Standard Error 10.015
|
32.14 percentage of assessments
Standard Error 8.078
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 13 to 16
|
69.11 percentage of assessments
Standard Error 7.783
|
55.13 percentage of assessments
Standard Error 10.664
|
27.10 percentage of assessments
Standard Error 8.997
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 17 to 20
|
69.46 percentage of assessments
Standard Error 7.848
|
56.70 percentage of assessments
Standard Error 10.554
|
35.00 percentage of assessments
Standard Error 10.323
|
|
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument Over the 24-Week Treatment Period
PM score: Weeks 21 to 24
|
72.76 percentage of assessments
Standard Error 7.185
|
55.90 percentage of assessments
Standard Error 9.976
|
33.54 percentage of assessments
Standard Error 9.699
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment.
The number of participants underwent biliary diversion surgery and liver transplantation was determined.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Number of Participants Underwent Biliary Diversion Surgery and Liver Transplantation
Biliary diversion surgery
|
0 Participants
|
0 Participants
|
0 Participants
|
|
Number of Participants Underwent Biliary Diversion Surgery and Liver Transplantation
Liver transplantation
|
0 Participants
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: From Baseline (Day 1) up to Week 24Population: The FAS included all randomized participants who received at least 1 dose of study treatment. Only participants with data collected for specific instrument are reported.
A positive pruritus assessment was defined as a scratching score of \<=1 or at least 1 point drop from baseline based on the Albireo ObsRO and PRO instruments. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments only when more than 50% of planned assessment recorded by each participant multiplied by 100.
Outcome measures
| Measure |
A4250 Low Dose
n=23 Participants
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 Participants
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
Placebo
n=20 Participants
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
|---|---|---|---|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo ObsRO Instrument: AM score
|
13 Participants
|
10 Participants
|
3 Participants
|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo ObsRO Instrument: AM and PM scores combined
|
17 Participants
|
9 Participants
|
4 Participants
|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo ObsRO Instrument: PM score
|
16 Participants
|
9 Participants
|
5 Participants
|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo PRO Instrument: AM and PM scores combined
|
1 Participants
|
3 Participants
|
0 Participants
|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo PRO Instrument: AM score
|
1 Participants
|
3 Participants
|
0 Participants
|
|
Number of Participants Achieved Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO and PRO Instruments Over the 24-Week Treatment Period
Albireo PRO Instrument: PM score
|
1 Participants
|
3 Participants
|
0 Participants
|
Adverse Events
Placebo
Serious events: 5 serious events
Other events: 17 other events
Deaths: 0 deaths
A4250 Low Dose
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
A4250 High Dose
Serious events: 3 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=20 participants at risk
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
A4250 Low Dose
n=23 participants at risk
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 participants at risk
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
|---|---|---|---|
|
Cardiac disorders
Supraventricular tachycardia
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
General disorders
Pyrexia
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Gastroenteritis adenovirus
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
H1N1 influenza
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Influenza
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Urinary tract infection
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral infection
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Auricular haematoma
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Liver function test increased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Weight gain poor
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Psychiatric disorders
Insomnia
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Neurodermatitis
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
Other adverse events
| Measure |
Placebo
n=20 participants at risk
Capsules for oral administration (to match active) once daily for 24 weeks.
Placebo: Placebo identical in appearance to active drug (A4250).
|
A4250 Low Dose
n=23 participants at risk
Capsules for oral administration (40 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
A4250 High Dose
n=19 participants at risk
Capsules for oral administration (120 mcg/kg) once daily for 24 weeks.
A4250 (odevixibat): A4250 is a small molecule and selective inhibitor of IBAT.
|
|---|---|---|---|
|
Ear and labyrinth disorders
Otorrhoea
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Blood and lymphatic system disorders
Splenomegaly
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Ear and labyrinth disorders
Ear pain
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Eye disorders
Eye discharge
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
8.7%
2/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Constipation
|
20.0%
4/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Diarrhoea
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
39.1%
9/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
21.1%
4/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Frequent bowel movements
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Toothache
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
17.4%
4/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
15.8%
3/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
General disorders
Influenza like illness
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
General disorders
Injection site swelling
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
General disorders
Pyrexia
|
25.0%
5/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
30.4%
7/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
26.3%
5/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Hepatobiliary disorders
Cholelithiasis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Hepatobiliary disorders
Hepatomegaly
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Hepatobiliary disorders
Jaundice
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Adenovirus infection
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Bronchitis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Conjunctivitis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Ear infection
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Gastroenteritis norovirus
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Gastroenteritis viral
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
H1N1 influenza
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Influenza
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Klebsiella infection
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Nasopharyngitis
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Otitis media
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Parotitis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Post procedural infection
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
2/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral diarrhoea
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral infection
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral rash
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
8.7%
2/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Auricular haematoma
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Scar
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Scratch
|
10.0%
2/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Alanine aminotransferase increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
13.0%
3/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
15.8%
3/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Aspartate aminotransferase increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
8.7%
2/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Bilirubin conjugated increased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Blood alkaline phosphatase increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Blood bilirubin increased
|
10.0%
2/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
13.0%
3/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Blood creatine phosphokinase increased
|
10.0%
2/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Blood creatinine decreased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Blood creatinine increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
International normalised ratio increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Liver palpable
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Platelet count increased
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Product residue present
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Vitamin D decreased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Vitamin E increased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
Weight decreased
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Investigations
White blood cell count increased
|
10.0%
2/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Vitamin A deficiency
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Metabolism and nutrition disorders
Vitamin E deficiency
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Musculoskeletal and connective tissue disorders
Neck mass
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Nervous system disorders
Dizziness
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Nervous system disorders
Headache
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Psychiatric disorders
Irritability
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Psychiatric disorders
Selective eating disorder
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Renal and urinary disorders
Chromaturia
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Renal and urinary disorders
Cystitis haemorrhagic
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Reproductive system and breast disorders
Genital rash
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.0%
3/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
10.5%
2/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Nail discolouration
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Neurodermatitis
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
8.7%
2/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Rash
|
15.0%
3/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Skin and subcutaneous tissue disorders
Rash vesicular
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
4.3%
1/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Surgical and medical procedures
Cardiac ablation
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
8.7%
2/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Sinusitis bacterial
|
0.00%
0/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
5.3%
1/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Skin candida
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Tonsillitis
|
5.0%
1/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
0.00%
0/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
|
Infections and infestations
Upper respiratory tract infection
|
15.0%
3/20 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
13.0%
3/23 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
26.3%
5/19 • All serious and non-serious adverse events (AEs) were collected once the caregiver/participant had signed the informed consent form (ICF) and until the post-treatment follow-up (Visit 10) or 28 days after the last dose of study treatment, approximately 28 weeks
Treatment emergent adverse events were defined as any AE that occurred after first dose or AE occurred before first dose but worsened in severity on or after first dose. Treatment emergent serious adverse events and treatment emergent non-serious AEs are reported here.
|
Additional Information
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