Primary Immunodeficiency Diseases (PID)
Conditions
Brief summary
The main aim of the study is to check how much TAK-771 stays in their blood over time, side effect from the study treatment or TAK-771, how much TAK-771 participants can receive without getting side effects from it, and if TAK-771 improves symptoms of primary immunodeficiency diseases (PID). 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-771 for totally 27 or 30 weeks. Treatment period is consist of two periods called Epoch 1 and Epoch 2. In Epoch 1, different groups of participants will receive lower to higher doses of TAK-771 for 3 to 6 weeks. The study doctors will check for side effects from each dose of TAK-771. In Epoch 2, participants will receive TAK-771 once a 3 or 4 weeks until the end of 24 weeks. There will be many clinic visits. The number of visits will depend on the infusion cycles of study drug (every 3, or 4 weeks).
Interventions
DRUGTAK-771
Intervention description; Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (rHuPH20)
Sponsors
Takeda
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Be a Japanese person. 2. Participant must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies(IUIS) Committee 2017. The diagnosis must be confirmed by the Medical Director prior to TAK-771 treatment. 3. Participant has been receiving a stable clinical dose of intravenous immunoglobulin (IVIG) or conventional subcutaneous immunoglobulin (cSCIG), which is equivalent to approximately 200 to 600 mg/kg body weight per 3 to 4 week period for IVIG and approximately 50 to 200 mg/kg body weight per week for cSCIG based on the description in the package insert, consistently over a period of at least 3 months prior to screening, or Participant has been receiving of TAK-664 with fixed dose and dosing frequency at least 3 months prior to enrollment. That is, participant is about to complete Study TAK-664-3001 or participating in Study TAK-664-3002. 4. Participant who has been receiving IVIG or cSCIG had all serum trough levels of total immunoglobulin G (IgG) \>=5 g/L within 1 month prior to the screening/enrollment. 5. Serum trough levels at screening/enrollment meet one of the following: 1. IVIG-treated or cSCIG-treated participants Participant who had serum trough levels of IgG \>=5 g/L at the last 2 points in screening procedure before the first administration of TAK-771. 2. TAK-664-treated participants Participant who had serum trough levels of IgG \>=5 g/L at the last 2 points in TAK-664 studies before the first administration of TAK-771. 6. Participant is willing and able to comply with use of digital tools and applications.
Exclusion criteria
1. Participant has a known history of or is positive at screening/enrollment for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2 For participants who are switching from TAK-664 studies, the eligibility will be reconfirmed after result of the specialty test conducted at Week 1 become available. 2. Abnormal laboratory values at screening/enrollment meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent): * Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \>2.5 times the upper limit of normal (ULN) for the testing laboratory * Persistent severe neutropenia (defined as an absolute neutrophil count \[ANC\] =\<500/mm\^3) 3. Participant has presence of renal function impairment defined by eGFR \<60 mL/min/1.73m\^2. 4. Participant has been diagnosed with, or had a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix) unless the disease-free period prior to screening exceeds 5 years. 5. Participant is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident, pulmonary embolism) within 12 months prior to screening/enrollment or a history of thrombophilia. 6. Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome) 7. Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site. 8. Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IVIG, subcutaneous immunoglobulin (SCIG), and/or Immune Serum Globulin infusions 9. Participant has immunoglobulin A (IgA) deficiency (serum IgA less than 0.07g/L) and history of hypersensitivity, or history of confirmed anti-IgA antibodies, or both. 10. Participant is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the time of screening/enrollment. 11. Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening/enrollment or had a serious bacterial infection within the 3 months prior to screening/enrollment 12. Participant has a bleeding disorder, or a platelet count less than 20,000/microL, or in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous (SC) therapy. 13. Participant has total protein \>9 g/dL or myeloma, or macroglobulinemia (IgM) or paraproteinemia. 14. Participant has a known allergy to hyaluronidase 15. Participant has severe dermatitis that would preclude adequate sites for safe product administration.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval | The data was reported at Week 7, 11, 15, 19, 23, 27, and 31 for 4-Week interval and at Week 4, 7, 10, 13, 16, 19, 22, 25 and 28 for 3-Week interval. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | — |
| Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | AUC is expressed as grams\*day per liter/grams per kilograms \[(g\*day/L)/(g/kg)\]. |
| Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | At multiple time points post-infusion from Week 7 for participants with 4-Week or Week 4 with 3-Week dosing interval up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval | — |
| Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | — |
| Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | — |
| Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | — |
| Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | 4-Week dosing interval (Week 7, Week 11, Week 15, Week 19, Week 23, Week 27, and Week 31); 3-Week dosing interval (Week 4, week 7, Week 10, Week 16, Week 19, Week 22, Week 25, and Week 28) | — |
| Epoch 1 and 2: Trough Levels of Anti-Clostridium Tetani Toxoid Antibody After Administration of TAK-771 | From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval) | — |
| Epoch 1 and 2: Trough Levels of Anti-HBV Antibody After Administration of TAK-771 | From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval) | — |
| Epoch 1 and 2: Trough Levels of Anti-HIB Antibody After Administration of TAK-771 | From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval). | — |
| Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. |
| Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | TEAEs are defined as Adverse events (AEs) with onset after date-time of first dose of Investigational product (IP), or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. |
| Percentage of Participants With Serious and Non-serious TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Serious TEAE=any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) at any dose: results in death, was life-threatening, requires inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI=investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable AESI. |
| Percentage of Participants With Severe TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Percentage of Participants With Local and Systemic TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Percentage of Participants With TEAEs Leading to Premature Discontinuation From Study | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Percentage of Participants With Infusion-associated TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Percentage of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Percentage of Participants With Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Epoch 2: Percentage of Participants Who Develop Anti-rHuPH20 Binding Antibody Titers of Greater Than or Equal to 1:160 | 4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28) | — |
| Epoch 2: Percentage of Participants Who Develop Neutralizing Antibodies to rHuPH20 | 4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28) | — |
| Percentage of Participants Who Experienced Tolerability Events Related to the Infusion of TAK-771 | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | Tolerability events is defined as a case that the infusion rate is reduced, or that the infusion is interrupted or stopped, due to a TEAE related to TAK-771 infusion. |
| Epoch 1: Number of Weeks to Reach Final Dose Interval (3 Weeks or 4 Weeks) | Up to Week 4 for Participants with 4-Week Dosing Interval or Up to Week 3 for Participants with 3-Week Dosing Interval | The number of weeks to reach final dose interval is defined as treatment duration of Epoch 1. |
| Epoch 2: Percentage of Participants Who Achieve a Treatment Interval of 3 or 4 Weeks | Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Epoch 2: Percentage of Participants Who Maintain a Treatment Interval of 3 or 4 Weeks | Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Annual Rate of Validated Acute Serious Bacterial Infections (ASBIs) Per Participant Per Year | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | The annual rate of validated ASBIs is calculated as the mean number of ASBIs per participant per year. |
| Annual Rate of All Infections Per Participant Per Year | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | The annual rate of all infections is calculated as the mean number of infections per participant per year. |
| Healthcare Resource Utilization: Days Not Able To Attend School/Work or To Perform Normal Daily Activities Due to Illness/Infection | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Healthcare Resource Utilization: Days on Antibiotics | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Healthcare Resource Utilization: Number of Hospitalizations Due to Illness/Infection Per Year | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Healthcare Resource Utilization: Length of Stay in Days of Hospitalizations Due to Illness/Infection Per Participant Per Year | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Healthcare Resource Utilization: Number of Acute (Urgent or Unscheduled) Physician Visits Due to Illness/Infection | From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval | — |
| Infusion Parameters in Epoch 2: Number of Infusion Sites Per Infusion | From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval | Total number of infusion sites injected in Epoch 2 / Total number of infusions administered in Epoch 2. |
| Infusion Parameters in Epoch 2: Number of Infusion Sites Per Month | From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval | Total number of infusion sites injected in Epoch 2 / (duration of Epoch 2 / 30.4375), where duration of Epoch 2 is calculated as the end date of the Epoch 2 - the start date of the Epoch 2 + 1. |
| Infusion Parameters in Epoch 2: Duration of Individual Infusions | From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval | End date and time of infusion in Epoch 2 - Start date and time of infusion in Epoch 2, for each infusion per participant. |
| Infusion Parameters in Epoch 2: Maximum Infusion Rate Per Site | From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval | Maximum Infusion Rate results from CRF / number of infusion sites/body. |
| Infusion Parameters in Epoch 2: Infusion Volume Per Site | From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval | Infusion Volume per Site is scheduled Dose results from CRF / number of infusion sites/body. |
| Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | 4-Week Dosing Interval (Week 1 and Week 31); 3-Week Dosing Interval (Week 1 and Week 28) | The PEDS-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. In this study, 2-7 years (parent as observer), 8-13 years (participant as observer) for PEDS-QL health questionnaire will be analyzed. Higher scores indicate better quality of life (QOL) for all domains of the PEDS-QL. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Four dimensions (physical, emotional, social, & school functioning) are scored. |
| Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval. | — |
| QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28) | The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to all participants to assess the effect of the treatment on the participants' quality of life. Participants select answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-3L index score indicates improvement. |
| QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28) | The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to all participants to assess the effect of the treatment on the participants' quality of life. The EQ-5D-3L includes a visual analog scale (VAS), a vertical scale that allows the participants to indicate their health state that day, and ranges from 0 (worst imaginable) to 100 (best imaginable), with higher scores indicating better health state. |
| Treatment Preference | Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval | Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new subcutaneous immunoglobulin (SCIG) therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin (IG) therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment. |
| Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28) | Treatment Satisfaction Questionnaire for Medication (TSQM) is a global satisfaction scale used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. In this study, 2-12 years (parent as observer), 13 years and older (participant as observer) for TSQM health questionnaire will be analyzed. TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness, convenience, and global satisfaction. The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score indicated greater satisfaction in that domain. |
| QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | 4-Week Dosing Interval (Week 1 and Week 31); 3-Week Dosing Interval (Week 1 and Week 28) | The SF-36 is a generic quality-of-life instrument that has been widely used to assess Health-Related Quality of Life (HR QoL) of participants. In this study, 14 years and older (participant as observer) for SF-36 health questionnaire will be analyzed. Generic instruments are used in general populations to assess a wide range of domains applicable to a variety of health states, conditions, and diseases. The SF-36 consists of 36 items that are aggregated into 8 multi-item scales (physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health), with scores ranging from 0 to 100. Higher scores indicate better HR QoL. Physical component summary (PCS). |
Countries
Japan
Participant flow
Recruitment details
Participants with a confirmed diagnosis of primary immunodeficiency diseases (PID) took part in the study at 12 investigative sites in Japan from 24 January 2022 to 28 August 2023.
Pre-assignment details
Participants with PID, who had been receiving a consistent dose of intravenous immunoglobulin (IVIG), conventional subcutaneous immunoglobulin (cSCIG) or TAK-664 (immune globulin subcutaneous \[human\], 20% solution \[20%SCIG\]) for at least 3 months prior to screening were enrolled in the study to receive TAK-771.
Participants by arm
| Arm | Count |
|---|---|
| Epoch 1: TAK-771 TAK-771 included IGI 10% and rHuPH20. Participants received subcutaneous infusion of rHuPH20 solution at a dose of 80 U/g IgG first, followed by SC infusion of 10% IGI within 10 minutes of completion of the infusion of rHuPH20 solution. The dose of 10% IGI was increased from 1/3 of full dose to full dose in 3 weeks for participants who received TAK-771 once every 3 weeks, or from 1/4 of full dose to full dose in 6 weeks for participants who received TAK-771 once every 4 weeks. | 16 |
| Total | 16 |
Baseline characteristics
| Characteristic | Epoch 1: TAK-771 |
|---|---|
| Age, Continuous | 25.2 Years STANDARD_DEVIATION 16.86 |
| Body Mass Index (BMI) | 19.05 kilograms per meter square (kg/m^2) STANDARD_DEVIATION 2.747 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 16 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Height at Baseline | 149.26 Centimeters (cm) STANDARD_DEVIATION 25.892 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 16 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Region of Enrollment Japan | 16 Participants |
| Sex: Female, Male Female | 6 Participants |
| Sex: Female, Male Male | 10 Participants |
| Weight at Baseline | 44.33 Kilograms (kg) STANDARD_DEVIATION 16.509 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 16 | 0 / 16 |
| other Total, other adverse events | 13 / 16 | 15 / 16 |
| serious Total, serious adverse events | 1 / 16 | 1 / 16 |
Outcome results
Primary
Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771
The data was reported at Week 7, 11, 15, 19, 23, 27, and 31 for 4-Week interval and at Week 4, 7, 10, 13, 16, 19, 22, 25 and 28 for 3-Week interval.
Time frame: Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
Population: PK analysis set: all enrolled participants who received investigational drug once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect serum IgG concentration analysis results. PK set 1: analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Overall number analyzed: number of participants available for analysis. Number analyzed: participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 15, 4-Week Interval | 8.929 grams/Liter (g/L) | Geometric Coefficient of Variation 13 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 19, 4-Week Interval | 9.150 grams/Liter (g/L) | Geometric Coefficient of Variation 15.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 23, 4-Week Interval | 8.944 grams/Liter (g/L) | Geometric Coefficient of Variation 20.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 27, 4-Week Interval | 9.006 grams/Liter (g/L) | Geometric Coefficient of Variation 18.4 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 31, 4-Week Interval | 9.159 grams/Liter (g/L) | Geometric Coefficient of Variation 20.8 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 10, 3-Week Interval | 12.54 grams/Liter (g/L) | Geometric Coefficient of Variation 43.2 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 16, 3-Week Interval | 13.50 grams/Liter (g/L) | Geometric Coefficient of Variation 39.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 19, 3-Week Interval | 12.76 grams/Liter (g/L) | Geometric Coefficient of Variation 46.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 7, 4-Week Interval | 9.372 grams/Liter (g/L) | Geometric Coefficient of Variation 10.3 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 11, 4-Week Interval | 8.741 grams/Liter (g/L) | Geometric Coefficient of Variation 13.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 4, 3-Week Interval | 13.51 grams/Liter (g/L) | Geometric Coefficient of Variation 29.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 7, 3-Week Interval | 13.15 grams/Liter (g/L) | Geometric Coefficient of Variation 35.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 22, 3-Week Interval | 12.79 grams/Liter (g/L) | Geometric Coefficient of Variation 40.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 25, 3-Week Interval | 12.95 grams/Liter (g/L) | Geometric Coefficient of Variation 57.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of Total IgG Antibodies After Administration of TAK-771 | Week 28, 3-Week Interval | 12.70 grams/Liter (g/L) | Geometric Coefficient of Variation 43 |
Secondary
Annual Rate of All Infections Per Participant Per Year
The annual rate of all infections is calculated as the mean number of infections per participant per year.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Annual Rate of All Infections Per Participant Per Year | 2.69 infections per participant per year | Standard Deviation 3.133 |
Secondary
Annual Rate of Validated Acute Serious Bacterial Infections (ASBIs) Per Participant Per Year
The annual rate of validated ASBIs is calculated as the mean number of ASBIs per participant per year.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Annual Rate of Validated Acute Serious Bacterial Infections (ASBIs) Per Participant Per Year | 0.00 ASBIs per participant per year | Standard Deviation 0 |
Secondary
Epoch 1 and 2: Trough Levels of Anti-Clostridium Tetani Toxoid Antibody After Administration of TAK-771
Time frame: From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval)
Population: PK Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK Analysis Set included the analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-Clostridium Tetani Toxoid Antibody After Administration of TAK-771 | Week 1 | 1.334 IU/mL | Geometric Coefficient of Variation 84.4 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-Clostridium Tetani Toxoid Antibody After Administration of TAK-771 | End of Trial (EOS) | 1.578 IU/mL | Geometric Coefficient of Variation 68.4 |
Secondary
Epoch 1 and 2: Trough Levels of Anti-HBV Antibody After Administration of TAK-771
Time frame: From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval)
Population: Pharmacokinetic Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect serum IgG concentration analysis results. Pharmacokinetic Analysis Set included analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-HBV Antibody After Administration of TAK-771 | Week 1 | 278.38 mIU/mL | Geometric Coefficient of Variation 125 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-HBV Antibody After Administration of TAK-771 | End of Trial (EOS) | 383.37 mIU/mL | Geometric Coefficient of Variation 57.7 |
Secondary
Epoch 1 and 2: Trough Levels of Anti-HIB Antibody After Administration of TAK-771
Time frame: From Week 1, up to end of trial (EOS: Week 31 for participants with 4-Week dosing interval or Week 28 for participants with 3-Week dosing interval).
Population: PK Analysis Set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK Analysis Set included analysis of total serum IgG trough levels for total serum levels of IgG and IgG subclasses. Number analyzed are number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-HIB Antibody After Administration of TAK-771 | Week 1 | 1.958 ug/mL | Geometric Coefficient of Variation 45.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1 and 2: Trough Levels of Anti-HIB Antibody After Administration of TAK-771 | End of Trial (EOS) | 1.519 ug/mL | Geometric Coefficient of Variation 46.8 |
Secondary
Epoch 1: Number of Weeks to Reach Final Dose Interval (3 Weeks or 4 Weeks)
The number of weeks to reach final dose interval is defined as treatment duration of Epoch 1.
Time frame: Up to Week 4 for Participants with 4-Week Dosing Interval or Up to Week 3 for Participants with 3-Week Dosing Interval
Population: Safety Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 1: Number of Weeks to Reach Final Dose Interval (3 Weeks or 4 Weeks) | 6.00 weeks |
Secondary
Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: PK analysis set: all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK analysis set 2: the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number of participants analyzed is number of participants available for analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG | 76.84 milliliters per day (mL/day) | Geometric Coefficient of Variation 29.4 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1) | 117.0 milliliters per day (mL/day) | Geometric Coefficient of Variation 37.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2) | 219.6 milliliters per day (mL/day) | Geometric Coefficient of Variation 36.8 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3) | 6392 milliliters per day (mL/day) | Geometric Coefficient of Variation 176.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4) | 3234 milliliters per day (mL/day) | Geometric Coefficient of Variation 58 |
Secondary
Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: PK analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK analysis set 2=analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number participants analyzed: number of participants available for analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG | NA mL | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1) | 8193 mL | Geometric Coefficient of Variation 54.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2) | 14650 mL | Geometric Coefficient of Variation 57 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3) | NA mL | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4) | 165900 mL | Geometric Coefficient of Variation 88.3 |
Secondary
Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
AUC is expressed as grams\*day per liter/grams per kilograms \[(g\*day/L)/(g/kg)\].
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: PK analysis set: all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK analysis set 2: analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number of participants analyzed are number of participants available for analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG, AUClast/Dose | 625.6 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 55.2 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1), AUCtau/Dose | 432.4 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 36.2 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1), AUClast/Dose | 370.7 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 53 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2), AUCtau/Dose | 230.4 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 25.2 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2), AUClast/Dose | 197.4 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 40.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3), AUCtau/Dose | 7.916 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 165.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3), AUClast/Dose | 5.449 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 227.2 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4), AUCtau/Dose | 15.65 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 37.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG, AUCtau/Dose | 767.9 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 40 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4), AUClast/Dose | 12.08 (g*day/L)/(g/kg) | Geometric Coefficient of Variation 53.4 |
Secondary
Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
Time frame: At multiple time points post-infusion from Week 7 for participants with 4-Week or Week 4 with 3-Week dosing interval up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval
Population: PK analysis set: all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK analysis set 2: analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number of participants analyzed is number of participant available for analysis.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2) | 49.6 day |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3) | NA day |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4) | 35.9 day |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG | NA day |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Half-life of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1) | 59.7 day |
Secondary
Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | Total IgG | 12.72 g/L | Geometric Coefficient of Variation 23.4 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | IgG Subclass (IgG 1) | 7.694 g/L | Geometric Coefficient of Variation 16.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | IgG Subclass (IgG 2) | 4.140 g/L | Geometric Coefficient of Variation 23 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | IgG Subclass (IgG 3) | 0.2396 g/L | Geometric Coefficient of Variation 46.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG Subclasses | IgG Subclass (IgG 4) | 0.3200 g/L | Geometric Coefficient of Variation 36.3 |
Secondary
Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: PK analysis set: all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect serum IgG concentration analysis results. PK analysis set 2: analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number of participants analyzed: number of participants available for analysis.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3) | NA g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4) | 0.2255 g/L | Geometric Coefficient of Variation 8.4 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG | 9.347 g/L | Geometric Coefficient of Variation 17.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1) | 5.479 g/L | Geometric Coefficient of Variation 10.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2) | 2.941 g/L | Geometric Coefficient of Variation 19.8 |
Secondary
Epoch 2: Percentage of Participants Who Achieve a Treatment Interval of 3 or 4 Weeks
Time frame: Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Percentage of Participants Who Achieve a Treatment Interval of 3 or 4 Weeks | 100.0 percentage of participants |
Secondary
Epoch 2: Percentage of Participants Who Develop Anti-rHuPH20 Binding Antibody Titers of Greater Than or Equal to 1:160
Time frame: 4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28)
Population: Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Percentage of Participants Who Develop Anti-rHuPH20 Binding Antibody Titers of Greater Than or Equal to 1:160 | 0 percentage of participants |
Secondary
Epoch 2: Percentage of Participants Who Develop Neutralizing Antibodies to rHuPH20
Time frame: 4-Week Dosing Interval (Week 7, Week 19, and Week 31); 3-Week Dosing Interval (Week 4, Week 16, and Week 28)
Population: Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Percentage of Participants Who Develop Neutralizing Antibodies to rHuPH20 | 0 percentage of participants |
Secondary
Epoch 2: Percentage of Participants Who Maintain a Treatment Interval of 3 or 4 Weeks
Time frame: Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Percentage of Participants Who Maintain a Treatment Interval of 3 or 4 Weeks | 100.0 percenatge of participants |
Secondary
Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771
Time frame: 4-Week dosing interval (Week 7, Week 11, Week 15, Week 19, Week 23, Week 27, and Week 31); 3-Week dosing interval (Week 4, week 7, Week 10, Week 16, Week 19, Week 22, Week 25, and Week 28)
Population: PK analysis set: all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. PK analysis set 2: analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2. Number analyzed: participants with data available for analysis at given time point.
| Arm | Measure | Group | Value (GEOMETRIC_MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 7, 4-Week Interval | 0.1933 g/L | Geometric Coefficient of Variation 15.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 11, 4-Week Interval | 0.1965 g/L | Geometric Coefficient of Variation 22.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 25, 3-Week Interval | 1.910 g/L | Geometric Coefficient of Variation 73.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 28, 3-Week Interval | 1.948 g/L | Geometric Coefficient of Variation 88.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 4, 3-Week Interval | 0.1680 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 7, 3-Week Interval | 0.1680 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 7, 4-Week Interval | 5.478 g/L | Geometric Coefficient of Variation 12.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 11, 4-Week Interval | 5.106 g/L | Geometric Coefficient of Variation 15.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 15, 4-Week Interval | 4.987 g/L | Geometric Coefficient of Variation 11.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 19, 4-Week Interval | 5.063 g/L | Geometric Coefficient of Variation 10.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 23, 4-Week Interval | 4.873 g/L | Geometric Coefficient of Variation 10.8 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 27, 4-Week Interval | 5.066 g/L | Geometric Coefficient of Variation 16.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 31, 4-Week Interval | 5.104 g/L | Geometric Coefficient of Variation 14 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 7, 4-Week Interval | 3.147 g/L | Geometric Coefficient of Variation 20.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 11, 4-Week Interval | 2.931 g/L | Geometric Coefficient of Variation 15 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 15, 4-Week Interval | 2.912 g/L | Geometric Coefficient of Variation 13.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 19, 4-Week Interval | 3.043 g/L | Geometric Coefficient of Variation 16.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 23, 4-Week Interval | 2.976 g/L | Geometric Coefficient of Variation 16.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 27, 4-Week Interval | 3.078 g/L | Geometric Coefficient of Variation 15.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 31, 4-Week Interval | 3.091 g/L | Geometric Coefficient of Variation 18.3 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 3, Week 7, 4-Week Interval | 0.1380 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 3, Week 11, 4-Week Interval | 0.1780 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 3, Week 15, 4-Week Interval | 0.1390 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 3, Week 27, 4-Week Interval | 0.2280 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 15, 4-Week Interval | 0.2020 g/L | Geometric Coefficient of Variation 16.8 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 19, 4-Week Interval | 0.2057 g/L | Geometric Coefficient of Variation 16.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 23, 4-Week Interval | 0.2054 g/L | Geometric Coefficient of Variation 14.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 27, 4-Week Interval | 0.2228 g/L | Geometric Coefficient of Variation 24.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 31, 4-Week Interval | 0.2265 g/L | Geometric Coefficient of Variation 25.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 4, 3-Week Interval | 9.699 g/L | Geometric Coefficient of Variation 55.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 7, 3-Week Interval | 8.906 g/L | Geometric Coefficient of Variation 67 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 10, 3-Week Interval | 8.661 g/L | Geometric Coefficient of Variation 66.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 16, 3-Week Interval | 8.860 g/L | Geometric Coefficient of Variation 68 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 19, 3-Week Interval | 8.687 g/L | Geometric Coefficient of Variation 68.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 22, 3-Week Interval | 8.641 g/L | Geometric Coefficient of Variation 64.3 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 25, 3-Week Interval | 8.573 g/L | Geometric Coefficient of Variation 82.9 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 1, Week 28, 3-Week Interval | 8.845 g/L | Geometric Coefficient of Variation 68.3 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 4, 3-Week Interval | 1.936 g/L | Geometric Coefficient of Variation 105.6 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 7, 3-Week Interval | 1.859 g/L | Geometric Coefficient of Variation 89.7 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 10, 3-Week Interval | 1.852 g/L | Geometric Coefficient of Variation 83 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 16, 3-Week Interval | 1.964 g/L | Geometric Coefficient of Variation 82.5 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 19, 3-Week Interval | 1.944 g/L | Geometric Coefficient of Variation 82.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 2, Week 22, 3-Week Interval | 1.957 g/L | Geometric Coefficient of Variation 89.1 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 10, 3-Week Interval | 0.2010 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 16, 3-Week Interval | 0.2280 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 19, 3-Week Interval | 0.2120 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 22, 3-Week Interval | 0.2230 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 25, 3-Week Interval | 0.2060 g/L | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Serum Trough Levels of IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) After Administration of TAK-771 | IgG 4, Week 28, 3-Week Interval | 0.2390 g/L | — |
Secondary
Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4)
Time frame: Pre-infusion at Week 27 for participants with 4-Week or Week 25 with 3-Week dosing interval and post infusion at multiple time points up to Week 31 for participants with 4-Week dosing interval and up to Week 28 with 3-Week dosing interval.
Population: Pharmacokinetic analysis set included all enrolled participants who received investigational drug at least once, have had at least 1 evaluable serum IgG concentration, and no major protocol deviations or events that would affect the serum IgG concentration analysis results. Pharmacokinetic analysis set 2 included the analysis of PK parameters for total serum levels of IgG, for baseline-corrected total serum levels of IgG, and for IgG subclasses in Epoch 2.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | Total IgG | 6.94 days |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 1) | 3.92 days |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 2) | 3.92 days |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 3) | 2.99 days |
| Epoch 2: TAK-771 Full Dose Treatment Period | Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG Subclasses (IgG1, IgG2, IgG3, and IgG4) | IgG Subclass (IgG 4) | 3.88 days |
Secondary
Healthcare Resource Utilization: Days Not Able To Attend School/Work or To Perform Normal Daily Activities Due to Illness/Infection
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Healthcare Resource Utilization: Days Not Able To Attend School/Work or To Perform Normal Daily Activities Due to Illness/Infection | 0 number of days |
Secondary
Healthcare Resource Utilization: Days on Antibiotics
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Healthcare Resource Utilization: Days on Antibiotics | 0 number of days |
Secondary
Healthcare Resource Utilization: Length of Stay in Days of Hospitalizations Due to Illness/Infection Per Participant Per Year
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Healthcare Resource Utilization: Length of Stay in Days of Hospitalizations Due to Illness/Infection Per Participant Per Year | 0 hospitalization days/participant/year |
Secondary
Healthcare Resource Utilization: Number of Acute (Urgent or Unscheduled) Physician Visits Due to Illness/Infection
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Healthcare Resource Utilization: Number of Acute (Urgent or Unscheduled) Physician Visits Due to Illness/Infection | 1.74 Acute Physician Visits |
Secondary
Healthcare Resource Utilization: Number of Hospitalizations Due to Illness/Infection Per Year
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Healthcare Resource Utilization: Number of Hospitalizations Due to Illness/Infection Per Year | 0.22 hospitalizations per year | Standard Deviation 0.591 |
Secondary
Infusion Parameters in Epoch 2: Duration of Individual Infusions
End date and time of infusion in Epoch 2 - Start date and time of infusion in Epoch 2, for each infusion per participant.
Time frame: From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Infusion Parameters in Epoch 2: Duration of Individual Infusions | 74.0 minutes |
Secondary
Infusion Parameters in Epoch 2: Infusion Volume Per Site
Infusion Volume per Site is scheduled Dose results from CRF / number of infusion sites/body.
Time frame: From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2. Number analyzed are the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Infusion Parameters in Epoch 2: Infusion Volume Per Site | 117.3 mL/site | Standard Deviation 77.73 |
Secondary
Infusion Parameters in Epoch 2: Maximum Infusion Rate Per Site
Maximum Infusion Rate results from CRF / number of infusion sites/body.
Time frame: From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Infusion Parameters in Epoch 2: Maximum Infusion Rate Per Site | 222.3 mL/hour per site | Standard Deviation 83.77 |
Secondary
Infusion Parameters in Epoch 2: Number of Infusion Sites Per Infusion
Total number of infusion sites injected in Epoch 2 / Total number of infusions administered in Epoch 2.
Time frame: From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Infusion Parameters in Epoch 2: Number of Infusion Sites Per Infusion | 1.77 infusion sites per infusion | Standard Deviation 0.394 |
Secondary
Infusion Parameters in Epoch 2: Number of Infusion Sites Per Month
Total number of infusion sites injected in Epoch 2 / (duration of Epoch 2 / 30.4375), where duration of Epoch 2 is calculated as the end date of the Epoch 2 - the start date of the Epoch 2 + 1.
Time frame: From Week 7 up to Week 31 for Participants with 4-Week Dosing Interval or From Week 4 up to Week 28 for Participants with 3-Week Dosing Interval
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Infusion Parameters in Epoch 2: Number of Infusion Sites Per Month | 2.02 infusion sites per month | Standard Deviation 0.514 |
Secondary
Percentage of Participants Who Experienced Tolerability Events Related to the Infusion of TAK-771
Tolerability events is defined as a case that the infusion rate is reduced, or that the infusion is interrupted or stopped, due to a TEAE related to TAK-771 infusion.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety Analysis Set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants Who Experienced Tolerability Events Related to the Infusion of TAK-771 | 0 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants Who Experienced Tolerability Events Related to the Infusion of TAK-771 | 0 percentage of participants |
Secondary
Percentage of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs | 0 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs | 0 percentage of participants |
Secondary
Percentage of Participants With Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs | 0 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs | 0 percentage of participants |
Secondary
Percentage of Participants With Infusion-associated TEAEs
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Infusion-associated TEAEs | 37.5 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Infusion-associated TEAEs | 50.0 percentage of participants |
Secondary
Percentage of Participants With Local and Systemic TEAEs
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Local and Systemic TEAEs | Local TEAEs | 43.8 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Local and Systemic TEAEs | Systemic TEAEs | 75.0 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Local and Systemic TEAEs | Local TEAEs | 43.8 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Local and Systemic TEAEs | Systemic TEAEs | 87.5 percentage of participants |
Secondary
Percentage of Participants With Serious and Non-serious TEAEs
TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP. Serious TEAE=any untoward clinical manifestation of signs, symptoms, outcomes (related to IP or not) at any dose: results in death, was life-threatening, requires inpatient/prolongation of hospitalization, resulted in persistent/significant disability/incapacity, congenital abnormality/birth defect, important medical event. AESI=investigator-reported hypersensitivity reactions, events of disordered coagulation as bleeding/hypercoagulable AESI.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Serious and Non-serious TEAEs | Serious TEAEs | 6.3 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Serious and Non-serious TEAEs | Non-serious TEAEs | 81.3 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Serious and Non-serious TEAEs | Serious TEAEs | 6.3 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Serious and Non-serious TEAEs | Non-serious TEAEs | 93.8 percentage of participants |
Secondary
Percentage of Participants With Severe TEAEs
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Severe TEAEs | 0 Percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Severe TEAEs | 6.3 Percentage of participants |
Secondary
Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs
TEAEs are defined as AEs with onset after date-time of first dose of IP, or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | TEAE Related | 56.3 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | TEAE Non-Related | 62.5 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | TEAE Related | 68.8 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TAK-771-Related and TAK-771-Non-Related TEAEs | TEAE Non-Related | 81.3 percentage of participants |
Secondary
Percentage of Participants With TEAEs Leading to Premature Discontinuation From Study
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TEAEs Leading to Premature Discontinuation From Study | 0 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With TEAEs Leading to Premature Discontinuation From Study | 0 percentage of participants |
Secondary
Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs)
TEAEs are defined as Adverse events (AEs) with onset after date-time of first dose of Investigational product (IP), or medical conditions present prior to the start of IP but increased in severity or relationship after date-time of first dose of IP.
Time frame: From Week 1 up to Week 31 for Participants with 4-Week Dosing Interval or up to Week 28 for Participants with 3-Week Dosing Interval
Population: Safety analysis set included all enrolled participants who received investigational drug at least once. Epoch 2 Safety Analysis Set included all participants who received investigational drug at least once in Epoch 2.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | 81.3 percentage of participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs) | 93.8 percentage of participants |
Secondary
QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score
The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to all participants to assess the effect of the treatment on the participants' quality of life. Participants select answer for each of the following 3-level dimensions: 1) mobility; 2) self-care; 3) usual activities; 4) pain/discomfort; 5) anxiety/depression used to compute an index score ranging from 0 (worst imaginable health state) to 1 (best imaginable health state). An increase in the EQ-5D-3L index score indicates improvement.
Time frame: 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28)
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants available for analysis in the specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Baseline (Week 1), 2-11 years, EQ-5D index score, 4-week interval | 1.0000 score on a scale | Standard Deviation 0 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Baseline (Week 1), 2-11 years, EQ-5D index score, 3-week interval | 0.6750 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Baseline (Week 1), 12 years and older, EQ-5D index score, 4-week interval | 0.9271 score on a scale | Standard Deviation 0.12655 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Baseline (Week 1), 12 years and older, EQ-5D index score, 3-week interval | 1 score on a scale | Standard Deviation 0 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Change from Baseline (EOS/ET), 2-11 years, EQ-5D index score, 4-week interval | 0.0000 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Change from Baseline (EOS/ET), 2-11 years, EQ-5D index score, 3-week interval | 0.3250 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Change from Baseline (EOS/ET), 12 years and older, EQ-5D index score, 4-week interval | 0.0729 score on a scale | Standard Deviation 0.12655 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Index Score | Change from Baseline (EOS/ET), 12 years and older, EQ-5D index score, 3-week interval | 0.0000 score on a scale | Standard Deviation 0 |
Secondary
QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score
The EQ-5D-3L is a standardized instrument for use as a measure of health outcome and was administered to all participants to assess the effect of the treatment on the participants' quality of life. The EQ-5D-3L includes a visual analog scale (VAS), a vertical scale that allows the participants to indicate their health state that day, and ranges from 0 (worst imaginable) to 100 (best imaginable), with higher scores indicating better health state.
Time frame: 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28)
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants available for analysis in the specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Baseline (Week 1), 12 years and older, EQ-5D VAS score, 3-week interval | 85.0 score on a scale | Standard Deviation 7.07 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Baseline (Week 1), 2-11 years, EQ-5D VAS score, 4-week interval | 95.0 score on a scale | Standard Deviation 7.07 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Baseline (Week 1), 2-11 years, EQ-5D VAS score, 3-week interval | 100.0 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Baseline (Week 1), 12 years and older, EQ-5D VAS score, 4-week interval | 76.3 score on a scale | Standard Deviation 13.24 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Change from Baseline (EOS/ET), 2-11 years, EQ-5D VAS score, 4-week interval | -5.0 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Change from Baseline (EOS/ET), 2-11 years, EQ-5D VAS score, 3-week interval | -10 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Change from Baseline (EOS/ET), 12 years and older, EQ-5D VAS score, 4-week interval | 6.3 score on a scale | Standard Deviation 9.78 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire Visual Analogue Scale (VAS) Score | Change from Baseline (EOS/ET), 12 years and older, EQ-5D VAS score, 3-week interval | -2.5 score on a scale | Standard Deviation 3.54 |
Secondary
QOL: Short Form-36 Health Survey Version 2 (SF-36 v2)
The SF-36 is a generic quality-of-life instrument that has been widely used to assess Health-Related Quality of Life (HR QoL) of participants. In this study, 14 years and older (participant as observer) for SF-36 health questionnaire will be analyzed. Generic instruments are used in general populations to assess a wide range of domains applicable to a variety of health states, conditions, and diseases. The SF-36 consists of 36 items that are aggregated into 8 multi-item scales (physical functioning, role - physical, bodily pain, general health, vitality, social functioning, role - emotional, and mental health), with scores ranging from 0 to 100. Higher scores indicate better HR QoL. Physical component summary (PCS).
Time frame: 4-Week Dosing Interval (Week 1 and Week 31); 3-Week Dosing Interval (Week 1 and Week 28)
Population: Epoch 2 Full Analysis Set included all participants who received investigational drug at least once in Epoch 2. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants available for analysis in the specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), 14 years and older, Physical component summary (PCS) score, 4-week interval | 51.85 score on a scale | Standard Deviation 3.458 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), 14 years and older, Physical component summary (PCS) score, 3-week interval | 53.30 score on a scale | Standard Deviation 2.546 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), 14 years and older, Mental component summary (MCS) score 4-week interval | 51.38 score on a scale | Standard Deviation 14.06 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), >=14 years, Role/social component summary (RCS) score, 3-week interval | 53.50 score on a scale | Standard Deviation 8.768 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), 14 years and older, Mental component summary (MCS) score 3-week interval | 59.90 score on a scale | Standard Deviation 10.465 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Baseline (Week 1), >=14 years, Role/social component summary (RCS) score, 4-week interval | 49.67 score on a scale | Standard Deviation 8.495 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Physical component summary (PCS) score, 4-week interval | 0.20 score on a scale | Standard Deviation 8.709 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Physical component summary (PCS) score, 3-week interval | -1.85 score on a scale | Standard Deviation 1.202 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Mental component summary (MCS) score 4-week interval | -3.12 score on a scale | Standard Deviation 5.605 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Mental component summary (MCS) score 3-week interval | -3.55 score on a scale | Standard Deviation 0.636 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Role/social component summary (RCS) score 4-week interval | -0.80 score on a scale | Standard Deviation 6.592 |
| Epoch 2: TAK-771 Full Dose Treatment Period | QOL: Short Form-36 Health Survey Version 2 (SF-36 v2) | Change from Baseline (EOS/ET), >=14 years, Role/social component summary (RCS) score,3-week interval | 4.90 score on a scale | Standard Deviation 3.394 |
Secondary
Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL)
The PEDS-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. In this study, 2-7 years (parent as observer), 8-13 years (participant as observer) for PEDS-QL health questionnaire will be analyzed. Higher scores indicate better quality of life (QOL) for all domains of the PEDS-QL. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Four dimensions (physical, emotional, social, & school functioning) are scored.
Time frame: 4-Week Dosing Interval (Week 1 and Week 31); 3-Week Dosing Interval (Week 1 and Week 28)
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants available for analysis in the specified category.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Baseline (Week 1), 2-7 years, Total score, 3-week interval | 76.09 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Baseline (Week 1), 2-7 years, Total score, 4-week interval | 93.48 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Baseline (Week 1), 8-13 years, Total score, 4-week interval | 77.17 score on a scale | Standard Deviation 18.091 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Change from Baseline (EOS/ET),2-7 years, Total score, 4-week interval | 2.17 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Change from Baseline (EOS/ET),2-7 years, Total score, 3-week interval | 11.96 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL) | Change from Baseline (EOS/ET), 8-13 years, Total score, 4-week interval | 11.41 score on a scale | Standard Deviation 3.843 |
Secondary
Treatment Preference
Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new subcutaneous immunoglobulin (SCIG) therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin (IG) therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment.
Time frame: Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Before your participation in trial, where did you receive your IG therapy?=At hospital | 4 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, total time spent for my treatment per month=Like | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, ability to fit my treatment into my own schedule= Like | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Where do you prefer to receive your IG therapy? = At home | 7 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, frequency of administration=Like very much | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, potential to self-administer=Like | 8 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, The potential to self-administer = No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, potential to self-administer=Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ability to fit my treatment into my own schedule= Like | 7 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ability to fit my treatment into my own schedule= No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ability to fit my treatment into my own schedule= Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, overall convenience = Like | 8 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, overall convenience = No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, complexity of administration process=Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, complexity of administration process=Like | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, complexity of administration process=Dislike | 6 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, complexity of administration process=Dislike very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, My ability to self-administer without medical supervision = Dislike very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Before your participation in the trial, where did you receive your IG therapy?=At home | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Where do you prefer to receive your immunoglobulin therapy?=At hospital | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Where do you prefer to receive your IG therapy?=At home | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Where do you prefer to receive your IG therapy?=No Preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, frequency of administration=Like very much | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, frequency of administration=Like | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, Frequency of administration=No Preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, number of needlesticks per month=Like very much | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, number of needlesticks per month=Like | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, number of needlesticks per month=No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, total time spent for my treatment per month=No Preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, ease of administration= Like | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, ease of administration= No Preference | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, The ease of administration= Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, potential to self-administer=Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, potential to self-administer=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, potential to self-administer=Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, potential to self-administer=Dislike very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, ability to fit my treatment into my own schedule= No Preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, overall convenience=Like | 4 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, overall convenience=No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, amount of time administration takes=Like | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, amount of time administration takes=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, amount of time administration takes=Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, complexity of administration process=Like | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, complexity of administration process=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, complexity of administration process=Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, My ability to self-administer without medical supervision=Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, My ability to self-administer without medical supervision=No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, My ability to self-administer without medical supervision=Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, My ability to self-administer without medical supervision=Dislike very much | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 2-13 years, would you choose to continue receiving your treatment using IGg10% with rHuPH20 SC?=Yes | 5 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Before your participation, where did you receive your IG therapy?=At hospital | 8 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Before your participation, where did you receive your IG therapy?=At home | 7 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Before your participation, where did you receive your IG therapy?=Other | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Where do you prefer to receive your IG therapy? = At hospital | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, Where do you prefer to receive your IG therapy? = No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, frequency of administration=Like | 7 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, frequency of administration=No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, number of needlesticks per month=Like | 8 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, number of needlesticks per month=No preference | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, total time spent for my treatment per month = Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, total time spent for my treatment per month=Like | 6 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, total time spent for my treatment per month=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, total time spent for my treatment per month=Dislike | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ease of administration=Like | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ease of administration=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ease of administration=Dislike | 6 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ease of administration=Dislike very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, potential to self-administer=Dislike very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, ability to fit my treatment into my own schedule= Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, overall convenience = Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, amount of time administration takes = Like | 7 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, amount of time administration takes = No preference | 3 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, amount of time administration takes = Dislike | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, complexity of administration process=No preference | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, My ability to self-administer without medical supervision=Like very much | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, My ability to self-administer without medical supervision=Like | 6 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, My ability to self-administer without medical supervision = No preference | 1 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, My ability to self-administer without medical supervision = Dislike | 2 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, choose to continue receiving your treatment using IGg 10% with rHuPH20 SC?=Yes | 9 Participants |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Preference | 14 years and older, choose to continue receiving your treatment using IGg 10% with rHuPH20 SC?= No | 2 Participants |
Secondary
Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9)
Treatment Satisfaction Questionnaire for Medication (TSQM) is a global satisfaction scale used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. In this study, 2-12 years (parent as observer), 13 years and older (participant as observer) for TSQM health questionnaire will be analyzed. TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are effectiveness, convenience, and global satisfaction. The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score indicated greater satisfaction in that domain.
Time frame: 4-Week dosing interval (Week 1 and Week 31); 3-Week dosing interval (Week 1 and Week 28)
Population: Full Analysis Set included all enrolled participants who received investigational drug at least once. Overall number analyzed is the number of participants with data available for analyses. Number analyzed is the number of participants with data available for analysis at given timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Effectiveness, 3-week interval | 94.44 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Convenience, 4-week interval | 44.44 score on a scale | Standard Deviation 19.245 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Global satisfaction, 4-week interval | 59.52 score on a scale | Standard Deviation 10.911 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Convenience, 3-week interval | 44.44 score on a scale | Standard Deviation 0 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Effectiveness, 3-week interval | -27.78 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Convenience, 4-week interval | 13.89 score on a scale | Standard Deviation 3.928 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Effectiveness, 4-week interval | 64.81 score on a scale | Standard Deviation 3.208 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Convenience, 3-week interval | 38.89 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 2-12 years, Global satisfaction, 3-week interval | 100.00 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Effectiveness, 4-week interval | 64.81 score on a scale | Standard Deviation 18.812 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Effectiveness, 3-week interval | 63.89 score on a scale | Standard Deviation 3.928 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Convenience, 4-week interval | 62.04 score on a scale | Standard Deviation 19.694 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Global satisfaction, 4-week interval | 60.71 score on a scale | Standard Deviation 8.748 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Baseline (Week 1), 13 years and older, Global satisfaction, 3-week interval | 64.29 score on a scale | Standard Deviation 10.102 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Effectiveness, 4-week interval | 0.00 score on a scale | Standard Deviation 0 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Convenience, 3-week interval | 0.00 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Global satisfaction, 4-week interval | 10.71 score on a scale | Standard Deviation 5.051 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 2-12 years, Global satisfaction, 3-week interval | -28.57 score on a scale | — |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Effectiveness, 4-week interval | -12.04 score on a scale | Standard Deviation 26.156 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Effectiveness, 3-week interval | 2.78 score on a scale | Standard Deviation 3.928 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Convenience, 4-week interval | -3.70 score on a scale | Standard Deviation 20.688 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Convenience, 3-week interval | 0.00 score on a scale | Standard Deviation 7.857 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Global satisfaction, 4-week interval | -13.10 score on a scale | Standard Deviation 26.885 |
| Epoch 2: TAK-771 Full Dose Treatment Period | Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9) | Change from Baseline (EOS/ET), 13 years and older, Global satisfaction, 3-week interval | -3.57 score on a scale | Standard Deviation 5.051 |