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    Clinical Trial Results:
    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)

    Summary
    EudraCT number
    		 2022-003400-32
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    25 Apr 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Nov 2024
    First version publication date
    03 Nov 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    TAK-664-3002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04842643
    WHO universal trial number (UTN)
    		 U1111-1265-9447
    Other trial identifiers
    		 JRCT: jRCT2041210006
    Sponsors
    Sponsor organisation name
    Takeda
    Sponsor organisation address
    95 Hayden Avenue, Lexington, United States, MA 02421
    Public contact
    Study Director, Takeda, TrialDisclosures@takeda.com
    Scientific contact
    Study Director, Takeda, TrialDisclosures@takeda.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    25 Apr 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Apr 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of the study is to evaluate the long-term safety and tolerability of subcutaneous immunoglobulin (IGSC), 20% in Japanese participants with primary immunodeficiency disease (PID).
    Protection of trial subjects
    All study participants were required to read and sign an Informed Consent Form.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    27 Apr 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Japan: 12
    Worldwide total number of subjects
    12
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    2
    Adolescents (12-17 years)
    1
    Adults (18-64 years)
    8
    From 65 to 84 years
    1
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants took part in the study at 8 investigative sites in Japan from 27 April 2021 to 25 April 2024.

    Pre-assignment
    Screening details
    		 Participants with PID who completed core study TAK-664-3001 (2022- 001873-29) enrolled to receive IGCS, 20 percent (%) in current extension study (TAK-664-3002 [2022-003400-32]). As per planned analysis, the data was collected, analyzed and reported for a single arm only and per dose level wise data was not collected in this study.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 IGSC, 20%
    Arm description
    		 Participants who completed Epoch 2 of core study TAK-664-3001 (2022-001873-29), 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.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
    Investigational medicinal product code
    Other name
    Immune Globulin Infusion (Human)
    Pharmaceutical forms
    Infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received IGSC 20% infusion once a week or two weeks.

    Number of subjects in period 1
    		IGSC, 20%
    Started
    12
    Completed
    10
    Not completed
    2
         Physician decision
    1
         Not specified
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    IGSC, 20%
    Reporting group description
    		 Participants who completed Epoch 2 of core study TAK-664-3001 (2022-001873-29), 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.

    Reporting group values
    		 IGSC, 20% Total
    Number of subjects
    		 12
    Age Categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 36.0 ( 21.68 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 7
        Male
    		 5 5
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 12 12
        Native Hawaiian or Other Pacific Islander
    		 0 0
        Black or African American
    		 0 0
        White
    		 0 0
        More than one race
    		 0 0
        Unknown or Not Reported
    		 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 0 0
        Not Hispanic or Latino
    		 12 12
        Unknown or Not Reported
    		 0 0

    End points

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    End points reporting groups
    Reporting group title
    IGSC, 20%
    Reporting group description
    		 Participants who completed Epoch 2 of core study TAK-664-3001 (2022-001873-29), 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.

    Subject analysis set title
    IGSC: 2-13 Years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged 2-13 years and who completed Epoch 2 of core study TAK-664-3001 (2022-001873-29), received between 50 to 200 mg/kg of 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.

    Subject analysis set title
    IGSC: >=14 Years
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants aged >=14 years and who completed Epoch 2 of core study TAK-664-3001 (2022-001873-29), received between 50 to 200 mg/kg of 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.

    Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Non-serious TEAEs

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    End point title
    		 Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Non-serious TEAEs [1]
    End point description
    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. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be analyzed for this endpoint.
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: participants
        TEAEs
    12
        Serious TEAEs
    3
        Non-Serious TEAEs
    12
    No statistical analyses for this end point

    Primary: Number of Participants With Drug-related and Non-related TEAEs

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    End point title
    		 Number of Participants With Drug-related and Non-related TEAEs [2]
    End point description
    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. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be analyzed for this endpoint.
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: participants
        Drug-related TEAEs
    9
        Non-related TEAEs
    12
    No statistical analyses for this end point

    Primary: Number of Participants With Severe, Local and Systemic TEAEs

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    End point title
    		 Number of Participants With Severe, Local and Systemic TEAEs [3]
    End point description
    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”. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be analyzed for this endpoint.
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: participants
        Severe TEAEs
    3
        Local TEAEs
    8
        Systemic TEAEs
    12
    No statistical analyses for this end point

    Primary: Number of Participants With TEAEs Leading to Premature Discontinuation From Study and Infusion-associated TEAEs

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    End point title
    		 Number of Participants With TEAEs Leading to Premature Discontinuation From Study and Infusion-associated TEAEs [4]
    End point description
    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. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study.
    End point type
    Primary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive data was planned to be analyzed for this endpoint.
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: participants
        TEAEs Leading to Premature Discontinuation
    0
        Infusion-associated TEAEs
    12
    No statistical analyses for this end point

    Secondary: Serum Trough Levels of Total Immune Globulin G (IgG) and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Weekly Administration of IGSC, 20%

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    End point title
    		 Serum Trough Levels of Total Immune Globulin G (IgG) and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Weekly Administration of IGSC, 20%
    End point description
    Serum trough levels of total IgG and IgG1, IgG2, IgG3, IgG4 antibodies subclasses were determined by using standard assay methods. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study. Here” number of subjects analyzed" signified participants who were evaluable for this endpoint. Here "99999" means geometric mean and 95% confidence interval (CI) could not be calculated because the values were below lower limit of quantification (LLOQ).
    End point type
    Secondary
    End point timeframe
    At End of treatment (i.e. 3 years) in current extension study
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    5
    Units: grams per liter (g/L)
    geometric mean (confidence interval 95%)
        IgG 1
    6.14 (4.25 to 8.87)
        IgG 2
    3.52 (2.35 to 5.28)
        IgG 3
    99999 (99999 to 99999)
        IgG 4
    0.305 (0.165 to 0.567)
        IgG Total
    11.0 (7.29 to 16.5)
    No statistical analyses for this end point

    Secondary: Serum Trough Levels of IgG and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Biweekly Administration of IGSC, 20%

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    End point title
    		 Serum Trough Levels of IgG and IgG1, IgG2, IgG3, IgG4 Antibodies Subclasses Following Biweekly Administration of IGSC, 20%
    End point description
    Serum trough levels of IgG and IgG1, IgG2, IgG3, IgG4 antibodies subclasses were determined by using standard assay methods. All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study. Here” number of subjects analyzed" signified participants who were evaluable for this endpoint. Here "99999" means geometric mean and 95% CI could not be calculated because the values were below LLOQ.
    End point type
    Secondary
    End point timeframe
    At End of treatment (i.e. 3 years) in current extension study
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    7
    Units: g/L
    geometric mean (confidence interval 95%)
        IgG 1
    5.36 (4.55 to 6.33)
        IgG 2
    3.42 (3.03 to 3.86)
        IgG 3
    99999 (99999 to 99999)
        IgG 4
    0.250 (0.219 to 0.284)
        IgG Total
    8.98 (7.78 to 10.4)
    No statistical analyses for this end point

    Secondary: Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)

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    End point title
    		 Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: infections per year
        arithmetic mean (standard deviation)
    0.00 ( 0.000 )
    No statistical analyses for this end point

    Secondary: Annual Rate of All Infections

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    End point title
    		 Annual Rate of All Infections
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: infections per year
        arithmetic mean (standard deviation)
    1.42 ( 1.119 )
    No statistical analyses for this end point

    Secondary: Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection

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    End point title
    		 Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: days
        median (full range (min-max))
    1.91 (0.0 to 31.9)
    No statistical analyses for this end point

    Secondary: Number of Days Participants on Antibiotics

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    End point title
    		 Number of Days Participants on Antibiotics
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: days
        median (full range (min-max))
    2.55 (0.0 to 57.8)
    No statistical analyses for this end point

    Secondary: Number of Hospitalizations due to Illness or Infection

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    End point title
    		 Number of Hospitalizations due to Illness or Infection
    End point description
    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. 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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: hospitalization
        arithmetic mean (standard deviation)
    0.21 ( 0.370 )
    No statistical analyses for this end point

    Secondary: Length of Hospital Stay due to Illness or Infection

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    End point title
    		 Length of Hospital Stay due to Illness or Infection
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001. Here” number of subjects analyzed” signified participants who were evaluable for this endpoint.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    11
    Units: days
        median (full range (min-max))
    0.00 (0.0 to 3.9)
    No statistical analyses for this end point

    Secondary: Number of Acute Physician Visits due to Illness/Infection

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    End point title
    		 Number of Acute Physician Visits due to Illness/Infection
    End point description
    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. The Core Study All-Treated Set consisted of participants who received at least 1 dose of study drug (IGIV or IGSC) in TAK-664-3001.
    End point type
    Secondary
    End point timeframe
    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)
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: visits per year
        arithmetic mean (standard deviation)
    2.66 ( 2.652 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Their Response for Treatment Preference Questionnaire

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    End point title
    		 Number of Participants With Their Response for Treatment Preference Questionnaire
    End point description
    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 questionnaire, which investigate participant's preference on 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. All-Treated Set. 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. As pre-specified in protocol, data was planned to be reported as per age criteria (2-13 years and >=14 years).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    End point values
    		 IGSC: 2-13 Years IGSC: >=14 Years
    Number of subjects analysed
    3
    9
    Units: participants
        Before participation: Received therapy at home
    0
    0
        Before participation:Received therapy at hospital
    3
    8
        Before participation:Received therapy: Other
    0
    4
        Where you prefer to receive therapy:At Hospital
    1
    1
        Where you prefer to receive therapy:At Home
    0
    6
        Where you prefer to receive therapy:NoPreference
    2
    2
        Frequency of Administration: Like very much
    1
    1
        Frequency of Administration: Like
    1
    4
        Frequency of Administration: No preference
    1
    3
        Frequency of Administration: Dislike
    0
    1
        Frequency of Administration: Dislike very much
    0
    0
        Number of needlesticks per month: Like very much
    1
    1
        Number of needlesticks per month: Like
    1
    5
        Number of needlesticks per month: No preference
    1
    2
        Number of needlesticks per month:Dislike
    0
    1
        Number of needlesticks per month:Dislike verymuch
    0
    0
        Time spent for treatment per month:Like verymuch
    0
    3
        Time spent for treatment per month: Like
    1
    2
        Time spent for treatment per month: No preference
    1
    1
        Time spent for treatment per month: Dislike
    1
    3
        Timespent for treatment permonth:Dislike verymuch
    0
    0
        The ease of administration: Like very much
    1
    3
        The ease of administration: Like
    2
    4
        The ease of administration: No preference
    0
    1
        The ease of administration: Dislike
    0
    1
        The ease of administration: Dislike very much
    0
    0
        Ability to fit treatment: Like very much
    1
    5
        Ability to fit treatment: Like
    2
    1
        Ability to fit treatment: No preference
    0
    3
        Ability to fit treatment: Dislike
    0
    0
        Ability to fit treatment: Dislike very much
    0
    0
        The overall convenience: Like very much
    0
    4
        The overall convenience: Like
    3
    3
        The overall convenience: No preference
    0
    2
        The overall convenience: Dislike
    0
    0
        The overall convenience: Dislike very much
    0
    1
        Total Time administration took: Like very much
    1
    1
        Total Time administration took: Like
    1
    5
        Total Time administration took: No preference
    1
    1
        Total Time administration took: Dislike
    1
    2
        Total Time administration took: Dislike very much
    0
    0
        Complexity of administration: Like very much
    0
    2
        Complexity of administration process: Like
    3
    4
        Complexity of administration: No preference
    0
    2
        Complexity of administration: Dislike
    0
    1
        Complexity of administration: Dislike verymuch
    0
    0
        Ability to self-administer: Like very much
    2
    6
        Ability to self-administer: Like
    1
    3
        Ability to self-administer: No preference
    0
    0
        Ability to self-administer: Dislike very much
    0
    0
        Ability to self-administer: Dislike
    0
    0
        Choose to continue receiving IGSC: Yes
    3
    7
        Choose to continue receiving IGSC: No
    0
    2
        Factor influenced decision: Twoway needle needed
    0
    1
        Factor influenced decision:Canbe selfadministered
    1
    0
        Factor influenced decision: Easy administration
    0
    1
        Factor influenced decision: Feel relieved
    0
    1
        Factor influenced decision: IgG levels stabilized
    0
    1
        Factorinfluencedecision:Timeto clean affected area
    0
    1
        Factor influence decision:Atopicdermatitisimprove
    1
    0
        Factor influenced decision: No answer
    1
    0
        Factor influenced decision: No more coughing
    0
    1
        Factor influenced decision: The values are stable
    0
    1
        Factor influenced decision: Time is short
    0
    1
        Factor influencedecision:Stable medicalcondition
    0
    1
        Potential to selfadminister: Like very much
    2
    5
        Potential to selfadminister: Like
    0
    4
        Potential to selfadminister: No preference
    0
    0
        Potential to selfadminister:Dislike very much
    1
    0
        Potential to selfadminister: Dislike
    0
    0
    No statistical analyses for this end point

    Secondary: Number of Participants With Tolerability Events Related to the Infusion of Study Drug

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    End point title
    		 Number of Participants With Tolerability Events Related to the Infusion of Study Drug
    End point description
    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. The Safety Analysis Set (SAS) consisted of all participants who received at least 1 dose of study drug (IGIV or IGSC).
    End point type
    Secondary
    End point timeframe
    From first dose of study drug up to 3 years in current extension study
    End point values
    		 IGSC, 20%
    Number of subjects analysed
    12
    Units: participants
        Infusion rate was reduced
    1
        Infusion was interrupted
    4
        Infusions was stopped
    1
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug up to 3 years in current extension study
    Adverse event reporting additional description
    As per planned analysis, the data for this AE section was collected, analyzed and reported for a single arm only and per dose level wise data was not collected . All-Treated Set consisted of all enrolled participants who received IGSC, 20% administration at least once in this study.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    IGSC, 20%
    Reporting group description
    		 Participants who completed Epoch 2 of core study TAK-664-3001 (NCT04346108), received between 50 to 200 mg/kg of 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.

    Serious adverse events
    		 IGSC, 20%
    Total subjects affected by serious adverse events
         subjects affected / exposed
    3 / 12 (25.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Colorectal adenocarcinoma
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Rhegmatogenous retinal detachment
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Diabetic ketoacidosis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 IGSC, 20%
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 12 (100.00%)
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Colon adenoma
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin papilloma
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    3
    Vascular disorders
    Hypertension
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Chills
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Administration site discolouration
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infusion site bruising
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Malaise
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Injection site swelling
         subjects affected / exposed
    4 / 12 (33.33%)
         occurrences all number
    12
    Injection site reaction
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Injection site pain
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    4
    Injection site haemorrhage
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Injection site erythema
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    7
    Injection site bruising
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infusion site swelling
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    7
    Infusion site pruritus
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    5
    Infusion site pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infusion site erythema
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    4
    Puncture site pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Vaccination site joint erythema
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Vaccination site pain
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Pyrexia
         subjects affected / exposed
    5 / 12 (41.67%)
         occurrences all number
    15
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Drug hypersensitivity
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Allergy to arthropod bite
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Investigations
    Occult blood positive
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Platelet count decreased
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Injury, poisoning and procedural complications
    Heat illness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Lip injury
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Post-traumatic pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Procedural dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Procedural pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin abrasion
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin injury
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Wound
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Vaccination complication
         subjects affected / exposed
    5 / 12 (41.67%)
         occurrences all number
    12
    Cardiac disorders
    Angina pectoris
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nervous system disorders
    Sinus headache
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Retrograde amnesia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Post herpetic neuralgia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Dizziness
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Headache
         subjects affected / exposed
    6 / 12 (50.00%)
         occurrences all number
    26
    Hypoaesthesia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Orthostatic intolerance
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Ear and labyrinth disorders
    Vertigo positional
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Vertigo
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Sudden hearing loss
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Eye disorders
    Asthenopia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Blepharitis
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Chorioretinopathy
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Conjunctivitis allergic
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Dry eye
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gastrointestinal disorders
    Abdominal discomfort
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Abdominal pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Diarrhoea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Dental caries
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    6
    Constipation
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Aphthous ulcer
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gingival pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nausea
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Haemorrhoids thrombosed
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Haemorrhoids
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Haematochezia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Stomatitis
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    8
    Toothache
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Tooth resorption
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Skin and subcutaneous tissue disorders
    Hyperkeratosis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Eczema asteatotic
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Dyshidrotic eczema
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Acne
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Drug eruption
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Dermatitis atopic
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Cutaneous amyloidosis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Dry skin
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Urticaria
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Seborrhoeic dermatitis
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Rash pruritic
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Rash
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Prurigo
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Musculoskeletal and connective tissue disorders
    Arthritis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Back pain
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Myalgia
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Nodal osteoarthritis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Pain in extremity
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Pain in jaw
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Rheumatoid arthritis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Spinal osteoarthritis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infections and infestations
    Influenza
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Impetigo
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Hordeolum
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Herpes zoster
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Gastroenteritis
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    6
    Cystitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Conjunctivitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    COVID-19
         subjects affected / exposed
    5 / 12 (41.67%)
         occurrences all number
    6
    Bronchitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Laryngitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Nasopharyngitis
         subjects affected / exposed
    3 / 12 (25.00%)
         occurrences all number
    5
    Oral herpes
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    3
    Otitis externa
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Paronychia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Periodontitis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Post-acute COVID-19 syndrome
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    2
    Pulpitis dental
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    2
    Rhinovirus infection
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Sinusitis
         subjects affected / exposed
    4 / 12 (33.33%)
         occurrences all number
    6
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 12 (16.67%)
         occurrences all number
    9
    Metabolism and nutrition disorders
    Diabetic ketoacidosis
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Hyperlipidaemia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    02 Nov 2021
    Amendment 1 -Changed the inclusion criteria #1 to allow participants who have completed Epoch 2 to participate in this study even if they have not completed Epoch 3 in TAK-664-3001 study. - Extended the visit window from 28 days (±1 day) to 28 days (±2 days). - Extend the estimated duration of this study from approximately 2 years to approximately 3 years. - Added statement, For participants who discontinue Epoch 3 and enter Study TAK-664-3002, the dose regimen will be determined on a case-by-case basis. - Added the few criteria for discontinuation or withdrawal of the participants.
    19 Jul 2022
    Amendment 2: - Relationship of adverse event to study drug. - Investigational device term was replaced with device-use in-clinical trial. - A new section for description of ‘Device-used-in-clinical-trial’ is added. - The study schema is updated. - Infusions may be performed at home or at the study site, at the investigator’s discretion. - Added ‘body weight’ in the below sentence: “The dose can be modified based on participants IgG level/condition/body weight”. -Vital signs can be measured at participant’s home. -Vital signs at home will be recorded. in subject diary and will be reviewed by investigators. -Participants should come to the study sites for visits when laboratory test samples are to be collected every 12 weeks. Subjects are not required to come to the study site if all procedures/assessments can be performed at home. -The decision where the SC injection is administered is made during the Study TAK-664-3001, however the location of injection administration can be changed based on the Investigator’s and subject’s agreement -Laboratory tests including assessment of IgG trough levels will be performed every 12 weeks from Visit 1 -Hemolysis test will be done every 24 weeks from Visit 1. -Text added for physical examination, vital signs, assessment of non-drug therapies, assessment of concomitant medications, assessment of AEs, collection/review of diary, administration of study drug, and healthcare resource utilization -Minor grammatical, editorial and/or administrative changes have been made.
    28 Nov 2022
    Amendment 3: Interim analysis will be conducted during the study. The interim analysis data will be submitted during Japanese New Drug Application process as requested by Pharmaceuticals and Medical Devices Agency (PMDA).

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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