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

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)

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.

Study Overview

• Status: Completed
• Conditions: Primary Immunodeficiency Disease
• Intervention / Treatment: Biological: IGSC 20% infusion

Detailed Description

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)

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 3

Contacts and Locations

Study Locations

• Japan
  • Fukuoka, Japan
    • Kyushu University Hospital
  • Gifu, Japan
    • Gifu University Hospital
  • Hiroshima, Japan
    • Hiroshima University Hospital
  • Tokyo, Japan
    • Tokyo Medical Dental University Hospital
  • Aichi
    • Nagoya, Aichi, Japan
      • Nagoya University Hospital
  • Fukuoka
    • Kurume, Fukuoka, Japan
      • Kurume University Hospital
  • Ishikawa
    • Kanazawa, Ishikawa, Japan
      • Kanazawa University Hospital
  • Saitama
    • Tokorozawa, Saitama, Japan
      • National Defense Medical College hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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).

2. 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.
3. Participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria:

1. 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.
2. 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.
3. 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.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: IGSC, 20%; Participants who completed Epoch 2 of core study TAK-664-3001 (NCT04346108), received between 50 to 200 mg/kg of Immune globulin subcutaneous (IGSC) infusion, 20% infusion once a week (Epoch 2 regimen) and 100 to 400 mg/kg of IGSC infusion, 20% biweekly (Epoch 3 regimen) until the study drug becomes commercially available.	Biological: IGSC 20% infusion; IGSC 20% infusion,; Other Names: Immune Globulin Infusion (Human)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Non-serious TEAEs	TEAEs were defined as adverse events (AEs) with onset after date-time of first dose of study drug (intravenous immunoglobulin [IGIV] or IGSC), or medical conditions present prior to the start of study drug (IGIV or IGSC) but increased in severity or relationship after date-time of first dose of study drug (IGIV or IGSC). A serious TEAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important.	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Number of Participants With Drug-related and Non-related TEAEs	TEAEs were defined as adverse events (AEs) with onset after date-time of first dose of study drug (intravenous immunoglobulin [IGIV] or IGSC), or medical conditions present prior to the start of study drug (IGIV or IGSC) but increased in severity or relationship after date-time of first dose of study drug (IGIV or IGSC). Any TEAE that was recorded by the investigator as "probably related" or "possibly related" to study drug was considered as study drug related AE, and any AE recorded as "unlikely related" or "not related" was considered as unrelated AE.	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Number of Participants With Severe, Local and Systemic TEAEs	A severe TEAE was an AE that caused considerable interference with the participant's usual activities. Local TEAEs were defined as AEs that were included in the MedDRA Higher Level Group Term "administration site reactions" or contained the phrase "injection site" or "infusion site". Systemic TEAEs were defined as AEs that were not included in the Medical Dictionary for Regulatory Activities (MedDRA) Higher Level Group Term "administration site reactions" and did not contain the phrase "injection site" or "infusion site".	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Number of Participants With TEAEs Leading to Premature Discontinuation From Study and Infusion-associated TEAEs	Infusion associated TEAEs were defined as any TEAE that began during study drug infusion or within 72 hours of completion of study drug infusion. TEAEs leading to premature discontinuation from study and infusion-associated TEAEs were reported.	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serum Trough Levels of Total Immune Globulin G (IgG) and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Weekly Administration of IGSC, 20%	Serum trough levels of total IgG and IgG1, IgG2, IgG3, IgG4 antibodies subclasses were determined by using standard assay methods.	At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)
Serum Trough Levels of IgG and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Biweekly Administration of IGSC, 20%	Serum trough levels of IgG and IgG1, IgG2, IgG3, IgG4 antibodies subclasses were determined by using standard assay methods.	At end of treatment in the current extension study TAK-664-3002 (i.e. 3 years)
Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)	The ASBI rate was calculated as the mean number of acute serious bacterial infections per participants per year. Annual rate of validated acute serious bacterial infections per participant was assessed. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Annual Rate of All Infections	The annual rate of infections was calculated as the mean number of infections per participant per year. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 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 are standardized per year (365.25 days). Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Number of Days Participants on Antibiotics	Number of days on antibiotics was defined as the number of days those antibiotics were taken as concomitant medications and was standardized to per year (365.25 days). Number of days participants on antibiotics were reported. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Number of Hospitalizations Due to Illness or Infection	Number of hospitalizations were standardized to per year (365.25 days). Hospitalizations were measured by asking participants to report the number of nights they have stayed overnight in the hospital during the year, for something related to their own health. Number of hospitalizations due to illness or infection were reported. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Length of Hospital Stay Due to Illness or Infection	Length of hospital stay per stay was standardized to per year (365.25 days). Number of days due to illness or infection were reported. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Number of Acute Physician Visits Due to Illness/Infection	Number of acute physician visits is standardized to per year (365.25 days). Number of acute (urgent or unscheduled) physician visits due to illness/infection were reported. Reported timeframe included timeframe of core study TAK-664-3001 (NCT04346108) (Max approximately 1.5 year) and of current extension study (Max approximately 3 years) as data of this outcome measure was collected from first dose of core study to end of current extension study and the data is presented for participants who entered in current study from core study as per plan.	From first dose of study drug in core study TAK-664-3001 up to end of current extension study TAK-664-3002 (up to approximately 4.5 years)
Number of Participants With Their Response for Treatment Preference Questionnaire	Treatment preference questionnaire is a self-administered questionnaire developed to assess participant's 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. For First question "Before participation in the trial(s), where did you receive your immunoglobulin therapy" participants were allowed to select multiple answers for options ("At the hospital"; "At home"; "Other") for their treatment. As a result, the sum of responders in the arm "IGSC, 20%: >=14 Years" for the first question were higher than the total number of participants analyzed in the category.	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose
Number of Participants With Tolerability Events Related to the Infusion of Study Drug	An infusion was considered tolerable if the infusion rate was not reduced, or the infusion was not interrupted or stopped, due to a TEAE related to study drug infusion. A tolerability event was considered to have occurred if an infusion was not tolerable.	From the first dose of the study drug in the current extension study TAK-664-3002, up to 3 years post-dose

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): April 27, 2021
• Primary Completion (Actual): April 25, 2024
• Study Completion (Actual): April 25, 2024

Study Registration Dates

• First Submitted: April 11, 2021
• First Submitted That Met QC Criteria: April 11, 2021
• First Posted (Actual): April 13, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Immune System Diseases; Primary Immunodeficiency Diseases; Immunologic Deficiency Syndromes; Immunologic Factors; Physiological Effects of Drugs; Immunoglobulins
• Other Study ID Numbers: TAK-664-3002; U1111-1265-9447 (Other Identifier: WHO); jRCT2041210006 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared as there is a reasonable likelihood that individual patients could be re-identified (due to the limited number of study participants/study sites).

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No