A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID)
- Conditions
- Primary Immunodeficiency Diseases (PID)
- Interventions
- Drug: TAK-771
- Registration Number
- NCT05150340
- Lead Sponsor
- Takeda
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 16
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Epoch 1: TAK-771 Ramp up Period TAK-771 TAK-771 included IGI 10% and Recombinant Human Hyaluronidase (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. Epoch 2: TAK-771 Full Dose Treatment Period TAK-771 TAK-771 included Immune Globulin Infusion (IGI) 10% and Recombinant Human Hyaluronidase (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, every 3, or 4 weeks for up to Week 27 for participants with 4-Week dosing interval or Week 25 for participants with 3-Week dosing interval.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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. 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 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 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 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.
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).
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.
Trial Locations
- Locations (12)
Saitama Prefectual Children's Medical Center
🇯🇵Saitama, Japan
Shizuoka Childrens Hospital
🇯🇵Shizuoka, Japan
Nagoya University Hospital
🇯🇵Nagoya, Aichi, Japan
Kurume University Hospital
🇯🇵Kurume, Fukuoka, Japan
Hospital of University of Occupational and Environmental Health
🇯🇵Kitakyushu, Fukuoka, Japan
Kanagawa Children's Medical Center
🇯🇵Yokohama, Kanagawa, Japan
Shinshu University Hospital
🇯🇵Matsumoto, Nagano, Japan
National Center for Child Health and development
🇯🇵Setagaya-ku, Tokyo, Japan
Gifu University Hospital
🇯🇵Gifu, Japan
Kyushu University Hospital
🇯🇵Fukuoka, Japan
Hiroshima University Hospital
🇯🇵Hiroshima, Japan
Tokyo Medical Dental University Hospital
🇯🇵Bunkyo-ku, Tokyo, Japan