An Extension Study of TAK-664 for Japanese People With Primary Immunodeficiency Disease

Study Overview
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Brief Summary
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This study is an extension study for participants with primary immunodeficiency disorders who were previously treated with IGSC, 20% in the TAK-664-3001 study. They must have completed that study or be about to complete it before joining this study. Participants will continue treatment with IGCS, 20% in this study. The main aim of this study is to check for side effects from long-term treatment with IGSC, 20% . This medicine is not yet licensed in Japan, so participants will be treated with IGSC, 20% until it becomes commercially available.

Official Title
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A Phase 3, Open-label, Non-controlled, Multi-dose, Extension Study to Evaluate the Long-term Safety and Tolerability of IGSC, 20% in Japanese Subjects With Primary Immunodeficiency Disease (PID)

Conditions
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Primary Immunodeficiency Disease

Intervention / Treatment
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* Biological: IGSC 20% infusion


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participant has completed or is about to complete Takeda Clinical Study TAK-664-3001. A participant is considered to have completed Study TAK-664-3001 successfully if they fulfill the following criterion: Completed Epoch 2, in which IGSC, 20% is administered weekly (completion of Epoch 3, in which IGSC, 20% is administered biweekly, is not mandatory for participation in TAK-664-3002 study). Written informed consent is obtained from either the Participant or the Participant's legally authorized representative prior to any study-related procedures and study product administration. Participant is willing and able to comply with the requirements of the protocol. Exclusion Criteria: Participant has developed a new serious medical condition during participation in Study TAK-664-3001 such that the Participant's safety or medical care would be impacted by participation in the extension study TAK-664-3002. Participant is scheduled to participate in another non-Takeda clinical study involving an Investigational Product or device-used-in-clinical-trial in the course of this study. If a female of childbearing potential, Participant is pregnant or has a negative pregnancy test but does not agree to employ adequate birth control measures for the duration of the study.

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Epoch 2 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)<br>Participants will receive between 50 and 200 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once a week until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 2 of previous study (TAK-664-3001). The dose of IGSC will be established in previous study. | Biological: IGSC 20% infusion<br>* IGSC 20% infusion, The dose of IGSC will be established in previous study (TAK-664-3001)<br>* Other names: Immune Globulin Infusion (Human);|
| Experimental: Epoch 3 in previous study: Immune Globulin Subcutaneous 20% Solution (IGSC)<br>Participants will receive between 100 and 400 mg/kg of Immunoglobulin Globulin subcutaneous (IGSC) infusion, 20 percent (%) once every 2 weeks until the study drug becomes commercially available (approximately 3 years). All participants of this arm will have assigned Epoch 3 of previous study (TAK-664-3001).The dose of IGSC will be established in previous study (TAK-664-3001). For participants who discontinue Epoch 3 and enter Study TAK-664-3002, the dose regimen will be determined on a case-by-case basis. | Biological: IGSC 20% infusion<br>* IGSC 20% infusion, The dose of IGSC will be established in previous study (TAK-664-3001)<br>* Other names: Immune Globulin Infusion (Human);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants with Treatment Emergent Adverse Events (TEAEs) | Treatment emergent adverse events are defined as AEs with onset after date/time of first dose of study drug in Study-TAK-664-3001, or medical conditions present prior to the start of study drug Study-TAK-664-3001 but increased in severity or relationship after date/time of first dose of study drug in Study-TAK-664-3001. An AE is defined as any untoward medical occurrence in a clinical investigation subject administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (eg, a clinically significant abnormal laboratory value), symptom, or disease temporally associated with the use of a drug whether or not it is considered related to the drug. Number of participants with TEAEs will be assessed. | Up to 3 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Serum Trough Levels of Immune Globulin G (IgG) Antibodies Following Weekly Administration of IGSC, 20% | Serum trough levels of IgG antibodies measured during treatment after Epoch 2 of the previous study will be assessed. | Baseline, Every 12 weeks after first dose up to 3 years | 
| Serum Trough Levels of IgG Subclasses Following Weekly Administration of IGSC, 20% | Serum trough levels of IgG subclasses measured during treatment after Epoch 2 of the previous study will be assessed. | Baseline, Every 12 weeks after first dose up to 3 years | 
| Serum Trough Levels of IgG Antibodies Following Biweekly Administration of IGSC, 20% | Serum trough levels of IgG antibodies measured during treatment after Epoch 3 of the previous study will be assessed. | Baseline, Every 12 weeks after first dose up to 3 years | 
| Serum Trough Levels of IgG Subclasses Following Biweekly Administration of IGSC, 20% | Serum trough levels of IgG subclasses measured during treatment after Epoch 3 of the previous study will be assessed. | Baseline, Every 12 weeks after first dose up to 3 years | 
| Annual Rate of Validated Acute Serious Bacterial Infections (ASBI) | The ASBI rate will be calculated as the mean number of ASBI per participants per year. Annual rate of validated ASBI per participant will be assessed. | Baseline, Up to 3 years | 
| Annual Rate of All Infections | Annual rate is the number of participants reporting any infection per year. | Baseline, Up to 3 years | 
| Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection | Number of days not able to attend school or work to perform normal daily activities due to illness/infection will be assessed. | Baseline, Up to 3 years | 
| Number of Days Participants on Antibiotics | Number of days participants on antibiotics will be assessed. | Baseline, Up to 3 years | 
| Number of Hospitalizations due to Illness or Infection | Number of hospitalizations due to illness or infection will be assessed. | Baseline, Up to 3 years | 
| Length of Hospital Stay due to Illness or Infection | Number of days due to illness or infection will be assessed. | Baseline, Up to 3 years | 
| Number of Acute Physician Visits due to Illness/Infection | Number of acute (urgent or unscheduled) physician visits due to illness/infection will be reported. | Baseline, Up to 3 years | 
| Treatment Preference | Treatment preference questionnaire is a self-administered questionnaire developed to assess participants' preference towards the administration of new IGSC therapy. There are 4-items on the questionnaire, which investigate a participant's preference on the clinic/hospital/home setting of receiving the immunoglobulin therapy, the participant's rating on the frequency and method of administration, and the participant's preference to continue receiving the IGSC treatment. | Baseline, End of study (3 years) |