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    Clinical Trial Results:
    CLINICAL PHASE 3 STUDY TO MONITOR THE SAFETY, TOLERABILITY, AND EFFICACY OF SUBCUTANEOUS HUMAN IMMUNOGLOBULIN (CUTAQUIG®) ADMINISTERED AT MODIFIED DOSING REGIMENS IN PATIENTS WITH PRIMARY IMMUNODEFICIENCY DISEASES

    Summary
    EudraCT number
    		 2019-002999-13
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    03 Jan 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    20 Jul 2023
    First version publication date
    20 Jul 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SCGAM-06
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03939533
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Octapharma
    Sponsor organisation address
    Oberlaaer Straße 235, Vienna, Austria,
    Public contact
    Clinical Trials Information, CRMG, 43 610 321 220, ctgov@clinicalresearchmgt.com
    Scientific contact
    Clinical Trials Information, CRMG, 1 4136865213, ctgov@clinicalresearchmgt.com
    Sponsor organisation name
    Octapharma
    Sponsor organisation address
    Oberlaaer St 235, 1100, Vienna, Austria,
    Public contact
    Patrick Murphy, CRMG, 1 4136865213, p.murphy@crmg-usa.com
    Scientific contact
    Elisabeth Sussitz, Octapharma, 43 1610320,
    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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Nov 2022
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    03 Jan 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    03 Jan 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The co-primary objectives of this study are to assess CUTAQUIG administered using the following infusion parameters: • Compare total IgG trough levels from weekly infusions to every other week infusions • Safety and tolerability when administered at increased infusion volumes at each infusion site • Safety and tolerability when administered at increased infusion flow rates at each infusion site • Safety and tolerability when administered on an every other week dosing regimen
    Protection of trial subjects
    IRB Reviewed and approved
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    17 Oct 2019
    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
    United States: 64
    Worldwide total number of subjects
    64
    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)
    3
    Adolescents (12-17 years)
    2
    Adults (18-64 years)
    45
    From 65 to 84 years
    14
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Subjects with a history of primary immunodeficiency (PI) disease that were currently on a stable does of SCIG treatment were enrolled at 16 research sites across the US between October 2019 and January 2022

    Pre-assignment
    Screening details
    		 Subjects with a history of primary immunodeficiency (PI) disease that were currently on a stable does of SCIG treatment were enrolled at 16 research sites across the US between October 2019 and January 2022

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 : Increased Volume Cohort
    Arm description
    		 Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    CUTAQUIG Human normal immunoglobulin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Arm title
    		 Increased Infusion Rate Cohort - Cohort 2
    Arm description
    		 Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks
    Arm type
    		 Experimental

    Investigational medicinal product name
    CUTAQUIG Human normal immunoglobulin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Arm title
    		 Every Other Week Dosing Cohort - Cohort 3
    Arm description
    		 Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose
    Arm type
    		 Experimental

    Investigational medicinal product name
    CUTAQUIG Human normal immunoglobulin
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Infusion
    Routes of administration
    Infusion
    Dosage and administration details
    Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Number of subjects in period 1
    		Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Started
    15
    15
    34
    Completed
    12
    13
    30
    Not completed
    3
    2
    4
         Terminated due to patient decision
    3
    2
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 : Increased Volume Cohort
    Reporting group description
    		 Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks

    Reporting group title
    Increased Infusion Rate Cohort - Cohort 2
    Reporting group description
    		 Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Reporting group title
    Every Other Week Dosing Cohort - Cohort 3
    Reporting group description
    		 Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose

    Reporting group values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3 Total
    Number of subjects
    		 15 15 34 64
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0 0
        Newborns (0-27 days)
    		 0 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0 0
        Children (2-11 years)
    		 0 1 2 3
        Adolescents (12-17 years)
    		 0 1 1 2
        Adults (18-64 years)
    		 10 9 26 45
        From 65-84 years
    		 5 4 5 14
        85 years and over
    		 0 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 51.20 ± 17.27 47.88 ± 20.53 50.81 ± 18.54 -
    Gender categorical
    Units: Subjects
        Female
    		 10 11 27 48
        Male
    		 5 4 7 16
    Type of PI Disease
    Units: Subjects
        CVID
    		 14 13 30 57
        XLA
    		 0 1 0 1
        OTHER
    		 1 1 4 6

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 : Increased Volume Cohort
    Reporting group description
    		 Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks

    Reporting group title
    Increased Infusion Rate Cohort - Cohort 2
    Reporting group description
    		 Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Reporting group title
    Every Other Week Dosing Cohort - Cohort 3
    Reporting group description
    		 Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose

    Primary: IgG Trough Levels

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    End point title
    		 IgG Trough Levels
    End point description
    Mean change from baseline in individual total IgG trough levels in cohort 3 from weekly infusions to end of study every other week infusions, and for cohort 1 and cohort 2 (weekly infusions) change from baseline to end of study
    End point type
    Primary
    End point timeframe
    24 Weeks
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: g/L
        log mean (standard deviation)
    0.144 ± 0.7303
    0.065 ± 1.1046
    -0.593 ± 1.0791
    Statistical analysis title
    		 Primary Endpoint Analysis Cohort 3
    Statistical analysis description
    For subjects in Cohort 3, the mean total IgG trough levels were maintained with every other week dosing (mean [SD] = 9.927 [2.0146] g/L) compared to weekly dosing (mean [SD] = 10.364 [1.96322] g/L) for the FAS. A decrease of <1g/L total IgG trough levels is not considered to be clinically meaningful, confirmed by the statistically significant difference (p = 0.0017, 97.5% CI = -0.799, Infinity) supporting the primary endpoint that the decrease is not >1g/L.
    Comparison groups
    Every Other Week Dosing Cohort - Cohort 3 v Cohort 1 : Increased Volume Cohort
    Number of subjects included in analysis
    49
    Analysis specification
    Pre-specified
    Analysis type
    		 other [1]
    P-value
    		 = 0.0017
    Method
    		 t-test, 1-sided
    Parameter type
    		 Mean difference (net)
    Confidence interval
         level
    		 97.5%
         sides
    1-sided
         lower limit
    		 -0.0799
         upper limit
    		 -
    Variability estimate
    Standard deviation
    Dispersion value
    1.424
    Notes
    [1] - Pre-specified threshold

    Secondary: Serious Bacterial Infection Rates

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    End point title
    		 Serious Bacterial Infection Rates
    End point description
    Number of subjects who reported SBIs during the study
    End point type
    Secondary
    End point timeframe
    Duration of Study
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: Participants
        Participants
    0
    0
    0
    No statistical analyses for this end point

    Secondary: Rates of Infections

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    End point title
    		 Rates of Infections
    End point description
    Infection Rates per Person Year in the treatment period of 24 weeks
    End point type
    Secondary
    End point timeframe
    Treatment Period of 24 Weeks
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: Infections per person-year
    arithmetic mean (standard deviation)
        Infections per person-year
    3.16 ± 4.082
    2.22 ± 2.555
    1.66 ± 1.918
    No statistical analyses for this end point

    Secondary: Time to Resolution of Infections

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    End point title
    		 Time to Resolution of Infections
    End point description
    The amount of days it took for infectious disease occurrence and resolution for subjects during the treatment period of 24 weeks
    End point type
    Secondary
    End point timeframe
    24 Weeks
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: Days
    median (full range (min-max))
        Days
    23.5 (1 to 160)
    20.0 (11 to 85)
    16.0 (6 to 65)
    No statistical analyses for this end point

    Secondary: Antiobiotic Usage

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    End point title
    		 Antiobiotic Usage
    End point description
    Amount of subjects treated with antibiotics during the study
    End point type
    Secondary
    End point timeframe
    Duration of Study
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: Participants
        Participants
    10
    8
    21
    No statistical analyses for this end point

    Secondary: Number of Antibiotic Treatment Episodes Annualized

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    End point title
    		 Number of Antibiotic Treatment Episodes Annualized
    End point description
    Total number of treatment episodes annualized calculated as the sum of all unique episodes of antibiotics of all subjects from first dose day of cutaquig to last study visit/number of person years exposure
    End point type
    Secondary
    End point timeframe
    Duration of Study
    End point values
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Number of subjects analysed
    15
    15
    34
    Units: Treatment Episodes
    number (not applicable)
        Treatment Episodes
    4.53
    1.96
    2.38
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Duration of Study
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    19.0
    Reporting groups
    Reporting group title
    Cohort 1 : Increased Volume Cohort
    Reporting group description
    		 Increased volume at each infusion site - patients will receive CUTAQUIG weekly and increase infusion volumes every 4 weeks

    Reporting group title
    Increased Infusion Rate Cohort - Cohort 2
    Reporting group description
    		 Increased infusion rate - patients will receive CUTAQUIG weekly and increase infusion rates every 4 weeks

    Reporting group title
    Every Other Week Dosing Cohort - Cohort 3
    Reporting group description
    		 Every other week dosing - patients will receive CUTAQUIG every other week at the equivalent of twice their body-weight dependent [mg/kg] weekly dose

    Serious adverse events
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    1 / 34 (2.94%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Rheumatoid arthritis
         subjects affected / exposed
    0 / 15 (0.00%)
    1 / 15 (6.67%)
    0 / 34 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19
         subjects affected / exposed
    0 / 15 (0.00%)
    0 / 15 (0.00%)
    1 / 34 (2.94%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Dehydration
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    0 / 34 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Cohort 1 : Increased Volume Cohort Increased Infusion Rate Cohort - Cohort 2 Every Other Week Dosing Cohort - Cohort 3
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 15 (86.67%)
    12 / 15 (80.00%)
    30 / 34 (88.24%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 15 (0.00%)
    3 / 15 (20.00%)
    7 / 34 (20.59%)
         occurrences all number
    0
    4
    16
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    2 / 15 (13.33%)
    1 / 15 (6.67%)
    1 / 34 (2.94%)
         occurrences all number
    2
    7
    1
    Infusion site erythema
         subjects affected / exposed
    4 / 15 (26.67%)
    8 / 15 (53.33%)
    8 / 34 (23.53%)
         occurrences all number
    7
    54
    12
    Infusion site pain
         subjects affected / exposed
    2 / 15 (13.33%)
    4 / 15 (26.67%)
    3 / 34 (8.82%)
         occurrences all number
    2
    10
    7
    Infusion site pruritus
         subjects affected / exposed
    4 / 15 (26.67%)
    5 / 15 (33.33%)
    6 / 34 (17.65%)
         occurrences all number
    15
    19
    10
    Pyrexia
         subjects affected / exposed
    1 / 15 (6.67%)
    1 / 15 (6.67%)
    2 / 34 (5.88%)
         occurrences all number
    1
    4
    3
    Ear and labyrinth disorders
    Ear infection
         subjects affected / exposed
    2 / 15 (13.33%)
    0 / 15 (0.00%)
    2 / 34 (5.88%)
         occurrences all number
    2
    0
    2
    Gastrointestinal disorders
    Diarrhea
         subjects affected / exposed
    1 / 15 (6.67%)
    0 / 15 (0.00%)
    3 / 34 (8.82%)
         occurrences all number
    1
    0
    4
    Nausea
         subjects affected / exposed
    0 / 15 (0.00%)
    4 / 15 (26.67%)
    2 / 34 (5.88%)
         occurrences all number
    0
    9
    5
    Infections and infestations
    Acute sinusitis
         subjects affected / exposed
    2 / 15 (13.33%)
    2 / 15 (13.33%)
    2 / 34 (5.88%)
         occurrences all number
    2
    3
    2
    Sinusitis
         subjects affected / exposed
    4 / 15 (26.67%)
    5 / 15 (33.33%)
    6 / 34 (17.65%)
         occurrences all number
    4
    8
    9
    Urinary tract infection
         subjects affected / exposed
    3 / 15 (20.00%)
    1 / 15 (6.67%)
    1 / 34 (2.94%)
         occurrences all number
    3
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    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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