Trial Outcomes & Findings for A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) (NCT NCT05543187)
NCT ID: NCT05543187
Last Updated: 2026-01-28
Results Overview
The ItchRO (Obs) scale measures severity of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Average baseline morning ItchRO (Obs) scores were calculated as sum of the morning scores divided by number of morning scores for the 4-week (28 days) time periods (that is \[i.e.\], Day -28 to Day -1). Average morning ItchRO (Obs) scores Week 15 through Week 26 were calculated as the sum of the morning scores divided by the number of morning scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
5 participants
Primary outcome timeframe
Baseline to Week 15 through Week 26
Results posted on
2026-01-28
Participant Flow
Participants took part in the study at 8 sites in Japan from 10 January 2023 to 22 July 2025.
Participants diagnosed with progressive familial intrahepatic cholestasis 2 (PFIC2) due to adenosine triphosphate (ATP) binding cassette subfamily B member 11 (ABCB11) mutation that predicted residual bile salt excretion pump (BSEP) function (non-truncating \[nt\]-PFIC2) were enrolled in Primary Cohort, and participants with other PFIC subtypes or post-surgical were enrolled in Supplemental Cohort, to receive TAK-625 twice daily (BID).
Participant milestones
| Measure |
Primary Cohort: TAK-625
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 micrograms per kilograms (mcg/kg), 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
2
|
|
Overall Study
Dosed TAK-625 150 mcg/kg
|
3
|
2
|
|
Overall Study
Dosed TAK-625 300 mcg/kg
|
3
|
2
|
|
Overall Study
Dosed TAK-625 450 mcg/kg
|
3
|
2
|
|
Overall Study
Dosed TAK-625 600 mcg/kg
|
3
|
2
|
|
Overall Study
COMPLETED
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
Baseline characteristics by cohort
| Measure |
Primary Cohort: TAK-625
n=3 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
7.7 years
STANDARD_DEVIATION 3.21 • n=158 Participants
|
8.0 years
STANDARD_DEVIATION 4.24 • n=157 Participants
|
7.8 years
STANDARD_DEVIATION 3.11 • n=315 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=158 Participants
|
1 Participants
n=157 Participants
|
3 Participants
n=315 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=158 Participants
|
1 Participants
n=157 Participants
|
2 Participants
n=315 Participants
|
|
Race/Ethnicity, Customized
Asian
|
3 Participants
n=158 Participants
|
2 Participants
n=157 Participants
|
5 Participants
n=315 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 15 through Week 26Population: ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
The ItchRO (Obs) scale measures severity of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Average baseline morning ItchRO (Obs) scores were calculated as sum of the morning scores divided by number of morning scores for the 4-week (28 days) time periods (that is \[i.e.\], Day -28 to Day -1). Average morning ItchRO (Obs) scores Week 15 through Week 26 were calculated as the sum of the morning scores divided by the number of morning scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).
Outcome measures
| Measure |
Primary Cohort: TAK-625
n=3 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26
|
-1.514 score on a scale
Interval -3.6541 to 0.6255
|
-0.202 score on a scale
Interval -3.6957 to 3.2911
|
SECONDARY outcome
Timeframe: Baseline to Week 15 through Week 26Population: ITT included all participants who received at least one dose of study drug. Here, "overall number of participants analyzed" signified those participants who were evaluable for this outcome measure. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
The ItchRO (Obs) scale measures frequency of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1= A little bit of the time, 2= Some of the time, 3= Most of the time, 4= Almost all of the time/constantly, I don't know) to describe their pruritus condition. 'I don't know' was categorized as missing data. A higher score indicated more severe pruritus. Baseline average morning ItchRO (Obs) frequency scores were calculated as the sum of the morning frequency scores divided by the number of morning severity scores for the 4-week (28 days) time periods. (i.e., Day -28 to Day -1). Average morning ItchRO (Obs) frequency scores of Week 15 through Week 26 were calculated as the sum of the morning frequency scores divided by the number of mornings frequency scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).
Outcome measures
| Measure |
Primary Cohort: TAK-625
n=2 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26
|
-0.740 score on a scale
Interval -4.8063 to 3.3254
|
-0.735 score on a scale
Interval -6.0922 to 4.6213
|
SECONDARY outcome
Timeframe: Baseline to Week 26Population: ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
Change from baseline was calculated as: Post-baseline observed value - Baseline (before first dosing) observed value. Change from baseline in total sBA levels to Week 26 was reported.
Outcome measures
| Measure |
Primary Cohort: TAK-625
n=3 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26
|
-149.900 micromoles per liter (mcmol/L)
Interval -561.4008 to 261.6008
|
-18.250 micromoles per liter (mcmol/L)
Interval -173.901 to 137.401
|
SECONDARY outcome
Timeframe: Baseline through Week 26Population: ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
Responders to sBA control were defined as participants who achieved a decrease to less than (\<) 102 mcmol/L, a decrease of greater than (\>) 75 percentage (%), or normalization at any timepoint from baseline through Week 26.
Outcome measures
| Measure |
Primary Cohort: TAK-625
n=3 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26
|
33.3 percentage of participants
Interval 0.84 to 90.57
|
0.0 percentage of participants
Interval 0.0 to 84.19
|
SECONDARY outcome
Timeframe: Baseline to Week 15 through Week 26Population: ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
ItchRO (Obs) scale measures severity of pruritus, score ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Weekly average severity was calculated based on daily maximum severity scores from both morning and evening. Average baseline morning and evening ItchRO (Obs) scores were calculated as sum of morning or evening scores divided by number of morning or evening scores for 4-week (28 days) time periods (i.e., Day -28 to Day -1). Average morning and evening ItchRO (Obs) scores Week 15 to Week 26 were calculated as sum of morning or evening scores divided by number of morning or evening scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1).
Outcome measures
| Measure |
Primary Cohort: TAK-625
n=3 Participants
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 Participants
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26
|
-1.629 score on a scale
Interval -3.5064 to 0.2481
|
-0.254 score on a scale
Interval -4.3906 to 3.8824
|
Adverse Events
Primary Cohort: TAK-625
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Supplemental Cohort: TAK-625
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Primary Cohort: TAK-625
n=3 participants at risk
Participants diagnosed with PFIC2 due to ABCB11 mutation that predicted residual BSEP function received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
Supplemental Cohort: TAK-625
n=2 participants at risk
Participants diagnosed with other PFIC subtypes or post-surgical received TAK-625 orally, BID. In dose escalation period, dose was increased weekly, 150 mcg/kg, 300 mcg/kg, 450 mcg/kg, and 600 mcg/kg for up to 4 weeks (Weeks 1 to 4) or extended to 6 weeks depending on the safety or tolerability concerns. After the dose escalation period, each participant continued dosing with TAK-625 600 mcg/kg, orally, BID dose level in the stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Blood and lymphatic system disorders
Anaemia
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Investigations
Aspartate aminotransferase increased
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Injury, poisoning and procedural complications
Auricular haematoma
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Investigations
Blood bilirubin increased
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
COVID-19
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
100.0%
2/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
66.7%
2/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Gastrointestinal disorders
Diarrhoea
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Injury, poisoning and procedural complications
Fall
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Focal nodular hyperplasia
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Gingivitis
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Metabolism and nutrition disorders
Hyperammonaemia
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Influenza
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Investigations
Liver function test increased
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Nasopharyngitis
|
66.7%
2/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Otitis media
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Pneumonia mycoplasmal
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
General disorders
Pyrexia
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Injury, poisoning and procedural complications
Rib fracture
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Immune system disorders
Seasonal allergy
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Injury, poisoning and procedural complications
Sunburn
|
33.3%
1/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
0.00%
0/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Upper respiratory tract infection
|
100.0%
3/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
100.0%
2/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
|
Infections and infestations
Viral pharyngitis
|
0.00%
0/3 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
50.0%
1/2 • From start of study drug administration up to follow-up (up to Week 133)
As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place