Alagille Syndrome (ALGS)
Conditions
Brief summary
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.
Interventions
DRUGTAK-625
TAK-625 200 mcg or 400 mcg per kilogram, orally, once daily
Sponsors
Takeda
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Months to No maximum
Healthy volunteers
No
Inclusion criteria
1. 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. 2. The participant is diagnosed with ALGS. 3. The participant has one or more of the following evidences of cholestasis: 1. Total serum bile acid (sBA) \>3\^ upper limit of the normal range (ULN) for age. 2. Direct bilirubin (conjugated) \>1 mg/dL. 3. Lipid soluble vitamin (LSV) deficiency otherwise unexplainable. 4. Gamma-glutamyl transferase (GGT) \>3\^ ULN for age. 5. Intractable pruritus explainable only by liver disease. 4. The participant is expected to have a consistent caregiver(s) for the duration of the study. 5. The participant has an access to phone for scheduled calls from study site. 6. Both a caregiver and participant above the age of assent are capable of reading and understanding the questionnaires. 7. Caregivers (and age-appropriate participants) must be willing and able to use an eDiary device during the study. 8. 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). 9. 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
1. The participant has chronic diarrhea requiring ongoing intravenous (IV) fluid or nutritional intervention. 2. The participant has a previous history of surgical interruption of the enterohepatic circulation. 3. The participant has a previous liver transplant. 4. The participant decompensated cirrhosis (ALT \>15\^ ULN, international normalized ratio \[INR\] \>1.5 \[unresponsive to vitamin K therapy\], albumin \<3.0 g/dL, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy). 5. The participant has a history or presence of other concomitant liver disease. 6. The participant has a history or presence of any other disease or condition known to interfere with the absorption, distribution, metabolism, or excretion of drugs, including bile salt metabolism in the intestine (eg, inflammatory bowel disease). 7. The participant has a history or presence of gallstones or kidney stones. 8. The participant has a possible malignant liver mass in imaging, including screening ultrasound. 9. The participant has cancers, except for in situ carcinoma, or cancers treated at least 5 years prior to screening with no evidence of recurrence. 10. The participant has received bile acid/lipid binding resins or IBAT inhibitors within 28 days prior to screening and throughout the trial. 11. The participant who has received sodium phenylbutyrate for less than 6 months at the initiation of screening.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Fasting sBA Levels From Baseline to Week 18 | 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). |
| Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | 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). |
| Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | 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). |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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). |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | 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). |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | 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). |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | 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). |
| Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18 | 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). |
| Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18 | 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). |
| Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | From Week 18 to 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). |
| Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | From Week 18 to 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). |
| Change in ALT and ALP Levels From Week 18 to 22 | 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. |
| Change in Bilirubin (Total and Direct) Levels From Week 18 to 22 | 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. |
Countries
Japan
Contacts
STUDY_DIRECTORStudy Director
Takeda
Participant flow
Recruitment details
Participants took part in the study at 8 sites in Japan.
Pre-assignment details
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 by arm
| Arm | Count |
|---|---|
| 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 20 weeks. | 7 |
| Total | 7 |
Baseline characteristics
| Characteristic | TAK-625 |
|---|---|
| Age, Continuous | 6.143 years STANDARD_DEVIATION 3.8675 |
| Race/Ethnicity, Customized Asian | 7 Participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 7 |
| other Total, other adverse events | 7 / 7 |
| serious Total, serious adverse events | 1 / 7 |
Outcome results
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.
Time frame: From Week 18 to Week 22
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Fasting Serum Bile Acid (sBA) Levels From Week 18 to Week 22 | 17.643 micromoles per liter (mcmol/L) |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| TAK-625 | Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18 | Change in ALT | 17.7 Units per liter (U/L) |
| TAK-625 | Change in Alanine Aminotransferase (ALT) and Alkaline Phosphatase (ALP) Levels From Baseline to Week 18 | Change in ALP | 75.6 Units per liter (U/L) |
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.
Time frame: From Week 18 to 22
Population: 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.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| TAK-625 | Change in ALT and ALP Levels From Week 18 to 22 | Change in ALT | 7.9 U/L |
| TAK-625 | Change in ALT and ALP Levels From Week 18 to 22 | Change in ALP | -32.3 U/L |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| TAK-625 | Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18 | Change in total bilirubin | 11.3619 mcmol/L |
| TAK-625 | Change in Bilirubin (Total and Direct) Levels From Baseline to Week 18 | Change in direct bilirubin | 8.6009 mcmol/L |
Secondary
Change in Bilirubin (Total and Direct) Levels From Week 18 to 22
Change in bilrubin (total and direct) from Week 18 to 22 was reported. . Change was calculated as: observed value at Week 22 - observed value at Week 18.
Time frame: From Week 18 to 22
Population: 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.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| TAK-625 | Change in Bilirubin (Total and Direct) Levels From Week 18 to 22 | Change in total bilirubin | 0.6842 mcmol/L |
| TAK-625 | Change in Bilirubin (Total and Direct) Levels From Week 18 to 22 | Change in direct bilirubin | 1.2217 mcmol/L |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Fasting sBA Levels From Baseline to Week 18 | 8.300 mcmol/L |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Morning Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | -1.327 score on a scale |
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).
Time frame: From Week 18 to Week 22
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | -0.280 score on a scale |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | -1.238 score on a scale |
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).
Time frame: From Week 18 to 22
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Morning Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | -0.032 score on a scale |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Severity of Pruritus Measured by Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) From Baseline to Week 18 | -1.531 score on a scale |
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).
Time frame: From Week 18 to Week 22
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Obs) From Week 18 to Week 22 | -0.286 score on a scale |
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).
Time frame: Baseline to Week 18
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Baseline to Week 18 | -1.381 score on a scale |
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).
Time frame: From Week 18 to 22
Population: 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.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| TAK-625 | Change in Weekly Average Severity of Pruritus Measured by ItchRO (Pt) From Week 18 to Week 22 | 0.000 score on a scale |