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| ID | Type | Description | Link |
|---|---|---|---|
| jRCT2051220098 | Registry Identifier | jRCT |
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The main aim of the study is to check if TAK-625 improves symptoms of Alagille Syndrome (ALGS), side effect from the study treatment or TAK-625, and how much TAK-625 stays in their blood over time. This will help the study sponsor (Takeda) to work out the best dose to give people in the future.
The participants will be treated with TAK-625 for up to the end of study (about 34 months).
Participants will visit their study clinic 9 times from the start of study. After 9 times visits, participants will visit their study clinic every 12 weeks up to the end of study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TAK-625 | Experimental | TAK-625 200 mcg per kilogram, orally, once daily for 1 week. After that, TAK-625 400 mcg per kilogram, orally, once daily after Week 1. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| TAK-625 | Drug | TAK-625 200 mcg or 400 mcg per kilogram, orally, once daily |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 | Change in fasting sBA levels from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22- observed value at Week 18. | From Week 18 to Week 22 |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Fasting sBA Levels From Baseline to Week 18 | Change in fasting sBA levels from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | Baseline to Week 18 |
| Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 |
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Inclusion Criteria:
The participant is Japanese male or female with a body weight >=3.0 kilograms (kg) and who is >=1 month of age at the time of informed consent.
The participant is diagnosed with ALGS.
The participant has one or more of the following evidences of cholestasis:
The participant is expected to have a consistent caregiver(s) for the duration of the study.
The participant has an access to phone for scheduled calls from study site.
Both a caregiver and participant above the age of assent are capable of reading and understanding the questionnaires.
Caregivers (and age-appropriate participants) must be willing and able to use an eDiary device during the study.
Caregivers (and age-appropriate participants) must complete at least 10 eDiary reports (morning or evening) during each of 2 consecutive weeks of the screening period (maximum possible reports=14 per week), even if the participant is an adult (over 18 years old).
Average daily score >2 on the ItchRO questionnaire (maximum possible daily score of 4) for 2 consecutive weeks in the screening period, prior to dosing. A daily score is the higher of the scores for the morning and evening ItchRO. The average daily score is the sum of all daily scores divided by the number of days the ItchRO was completed. Since it is difficult to evaluate pruritus in infants, participants <12 months of age at screening whose pruritus is unavoidably difficult to be evaluated are not necessarily required to meet the above score.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Tsukuba Hospital | Tsukuba | Ibaraki | Japan | |||
| Yokohamashi Tobu Hospital |
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| Label | URL |
|---|---|
| To obtain more information on the study, click here/on this link | View source |
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De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).
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Participants diagnosed with Alagille Syndrome (ALGS) were enrolled to receive TAK-625 once daily (QD). The study consisted of the screening period, 2-week dose escalation period, 46-week stable dosing period, a follow-up dosing period after Week 48, and a safety follow-up period.
Participants took part in the study at 8 sites in Japan.
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| ID | Title | Description |
|---|---|---|
| FG000 | TAK-625 | Participants diagnosed with ALGS received TAK-625 orally, QD. In dose escalation period, dose was increased weekly, 200 micrograms per kilograms (mcg/kg) and 400 mcg/kg for up to 2 weeks. After the dose escalation period, each participant continued dosing with TAK-625 400 mcg/kg, orally, QD dose level in the stable dosing period for 46 weeks and a follow-up dosing period after Week 48, followed by a safety follow-up period. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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|
Intention-to-treat (ITT) included all participants who received at least one dose of study drug. As per-planned analysis, the data for baseline measures were collected, analyzed and reported as a single cohort.
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| ID | Title | Description |
|---|---|---|
| BG000 | TAK-625 | Participants diagnosed with ALGS received TAK-625 orally, QD. In dose escalation period, dose was increased weekly, 200 mcg/kg and 400 mcg/kg for up to 2 weeks. After the dose escalation period, each participant continued dosing with TAK-625 400 mcg/kg, orally, QD dose level in the stable dosing period for 46 weeks and a follow-up dosing period after Week 48, followed by a safety follow-up period. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 | Change in fasting sBA levels from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22- observed value at Week 18. | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | micromoles per liter (mcmol/L) | From Week 18 to Week 22 |
|
From the first dose of study drug up to Week 133
As per-planned analysis, the data for adverse events section were collected, analyzed and reported as a single cohort.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | TAK-625 | Participants diagnosed with ALGS received TAK-625 orally, QD. In dose escalation period, dose was increased weekly, 200 mcg/kg and 400 mcg/kg for up to 2 weeks. After the dose escalation period, each participant continued dosing with TAK-625 400 mcg/kg, orally, QD dose level in the stable dosing period for 46 weeks and a follow-up dosing period after Week 48, followed by a safety follow-up period. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Liver transplant | Surgical and medical procedures | MedDRA 28.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Administration site extravasation | General disorders | MedDRA 28.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Takeda | +1-877-825-3327 | TrialDisclosures@takeda.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 8, 2022 | Sep 10, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 9, 2025 | Jan 12, 2026 | SAP_002.pdf |
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| ID | Term |
|---|---|
| D016738 | Alagille Syndrome |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
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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. Weekly average severity of Pruritus is calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). |
| Baseline to Week 18 |
| Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | 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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | Baseline to Week 18 |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity were calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | From Week 18 to Week 22 |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | From Week 18 to Week 22 |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | Baseline to Week 18 |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | Baseline to Week 18 |
| Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18 | Change in ALT and ALP levels from baseline to Week 18 were reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | Baseline to Week 18 |
| Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18 | Change in bilirubin (total and direct) from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | Baseline to Week 18 |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | From Week 18 to 22 |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | From Week 18 to 22 |
| Change in ALT and ALP Levels From Week 18 to 22 | Change in ALT and ALP from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18. | From Week 18 to 22 |
| Change in Bilirubin (Total and Direct) Levels From Week 18 to 22 | Change in bilirubin (total and direct) from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18. | From Week 18 to 22 |
| Yokohama |
| Kanagawa |
| Japan |
| Miyagi Children's Hospital | Sendai | Miyagi | Japan |
| Kindai University Nara Hospital | Ikoma | Nara | Japan |
| Osaka University Hospital | Suita | Osaka | Japan |
| Juntendo University Hospital | Bunkyo-ku | Tokyo | Japan |
| Kyoto University Hospital | Kyoto | Japan |
| Saitama Prefectural Children's Medical Center | Saitama | Japan |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
|
|
| Secondary | Change in Fasting sBA Levels From Baseline to Week 18 | Change in fasting sBA levels from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | mcmol/L | Baseline to Week 18 |
|
|
|
| Secondary | Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | 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. Weekly average severity of Pruritus is calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | Baseline to Week 18 |
|
|
|
| Secondary | Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | 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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | Baseline to Week 18 |
|
|
|
| Secondary | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Obs). Weekly average severity were calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | From Week 18 to Week 22 |
|
|
|
| Secondary | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | From Week 18 to Week 22 |
|
|
|
| Secondary | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. Here, "overall number of participants analyzed" signified those participants who were evaluable for this outcome measure. | Posted | Mean | 95% Confidence Interval | score on a scale | Baseline to Week 18 |
|
|
|
| Secondary | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 18 (Day 120 to Day 126) - average value of baseline (Day -7 to Day -1). | 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 data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | Baseline to Week 18 |
|
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| Secondary | Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18 | Change in ALT and ALP levels from baseline to Week 18 were reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | Units per liter (U/L) | Baseline to Week 18 |
|
|
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| Secondary | Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18 | Change in bilirubin (total and direct) from baseline to Week 18 was reported. Change was calculated as: observed value at Week 18 - observed value at baseline (before dosing). | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | mcmol/L | Baseline to Week 18 |
|
|
|
| Secondary | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average severity of Pruritus was calculated based on daily maximum of morning and evening severity scores measured by ItchRO (Pt). Weekly average scores was calculated as the average of the daily maximum of morning and evening over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | 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 data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | From Week 18 to 22 |
|
|
|
| Secondary | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | The ItchRO (Pt) scale measures severity of pruritus. The score on ItchRO (Pt) scale ranged from 0 to 4, where 0= Not felt itchy, 1= Felt a little bit itchy, 2= Felt pretty itchy, 3= Felt very itchy, 4= Felt very, very itchy. A higher score indicated more severe pruritus. Weekly average morning severity was calculated as the average of the daily morning scores over the visit consisting of the 7 days on or before the scheduled Study Day (i.e., the sum of the scores divided by the number of non-missing scores). Change was calculated as: average value of Week 22 (Day 148 to Day 154) - average value of Week 18 (Day 120 to Day 126). | 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 data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | score on a scale | From Week 18 to 22 |
|
|
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| Secondary | Change in ALT and ALP Levels From Week 18 to 22 | Change in ALT and ALP from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18. | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | U/L | From Week 18 to 22 |
|
|
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| Secondary | Change in Bilirubin (Total and Direct) Levels From Week 18 to 22 | Change in bilirubin (total and direct) from Week 18 to 22 was reported. Change was calculated as: observed value at Week 22 - observed value at Week 18. | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the data for outcome measure were collected, analyzed and reported as a single cohort. | Posted | Mean | 95% Confidence Interval | mcmol/L | From Week 18 to 22 |
|
|
|
| 0 |
| 7 |
| 1 |
| 7 |
| 7 |
| 7 |
| Malnutrition | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Weight gain poor | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 28.0 | Systematic Assessment |
|
| Anal rash | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Arthropod bite | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | MedDRA 28.0 | Systematic Assessment |
|
| Bronchitis chronic | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Chapped lips | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Congenital arterial malformation | Congenital, familial and genetic disorders | MedDRA 28.0 | Systematic Assessment |
|
| Defaecation urgency | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dermatitis atopic | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dermatitis diaper | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Eyelid injury | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Gastroenteritis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Hand-foot-and-mouth disease | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA 28.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Ocular hyperaemia | Eye disorders | MedDRA 28.0 | Systematic Assessment |
|
| Otitis externa | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Otitis media | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 28.0 | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Skin abrasion | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Upper limb fracture | Injury, poisoning and procedural complications | MedDRA 28.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 28.0 | Systematic Assessment |
|
| Upper respiratory tract inflammation | Respiratory, thoracic and mediastinal disorders | MedDRA 28.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 28.0 | Systematic Assessment |
|
Not provided
| D004066 |
| Digestive System Diseases |
| D008107 | Liver Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |