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    Clinical Trial Results:
    A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

    Summary
    EudraCT number
    		 2019-003076-39
    Trial protocol
    		 DE   PL   HU   ES   LV   CZ   GB   BE   NL   BG   DK   AT   IT   RO  
    Global end of trial date
    11 May 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 May 2024
    First version publication date
    25 May 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ARGX-113-1802
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04281472
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    argenx BV
    Sponsor organisation address
    Industriepark Zwijnaarde 7, Zwijnaarde (Ghent), Belgium, 9052
    Public contact
    Regulatory, argenx, regulatory@argenx.com
    Scientific contact
    Regulatory, argenx, regulatory@argenx.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?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    21 Nov 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 May 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    11 May 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Stage A: To assess the activity of efgartigimod PH20 SC (efgartigimod co-formulated with recombinant human hyaluronidase PH20 [rHuPH20]) based on the percentage of patients classified as treatment responders. Stage B: To determine the efficacy of efgartigimod PH20 SC compared to placebo based on the time needed for the first evidence of clinical deterioration.
    Protection of trial subjects
    This study was conducted in accordance with the protocol and consensus ethical principles derived from international guidelines, including the Declaration of Helsinki, applicable ICH GCP guidelines, and applicable laws and regulations. The participant’s informed consent was documented by the dated signature of the participant (and assent, if applicable) and the dated signature of the investigator or investigator’s delegate.
    Background therapy
    		 -
    Evidence for comparator
    		 This study is placebo-controlled and Placebo PH20 SC (placebo) is used for comparator.
    Actual start date of recruitment
    14 Apr 2020
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 5
    Country: Number of subjects enrolled
    Poland: 18
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    United Kingdom: 13
    Country: Number of subjects enrolled
    Austria: 8
    Country: Number of subjects enrolled
    Belgium: 7
    Country: Number of subjects enrolled
    Bulgaria: 9
    Country: Number of subjects enrolled
    Denmark: 17
    Country: Number of subjects enrolled
    France: 17
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Italy: 14
    Country: Number of subjects enrolled
    China: 58
    Country: Number of subjects enrolled
    Georgia: 16
    Country: Number of subjects enrolled
    Israel: 1
    Country: Number of subjects enrolled
    Japan: 24
    Country: Number of subjects enrolled
    Romania: 11
    Country: Number of subjects enrolled
    Russian Federation: 16
    Country: Number of subjects enrolled
    Serbia: 9
    Country: Number of subjects enrolled
    Taiwan: 5
    Country: Number of subjects enrolled
    Türkiye: 3
    Country: Number of subjects enrolled
    Ukraine: 12
    Country: Number of subjects enrolled
    United States: 48
    Worldwide total number of subjects
    322
    EEA total number of subjects
    117
    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)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    247
    From 65 to 84 years
    75
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Up to 360 participants were planned to be enrolled in Stage A. A total of 322 participants received efgartigimod PH20 SC in Stage A, and 221 of those participants continued to Stage B where they were randomized in a 1:1 ratio to efgartigimod PH20 SC (N=111) or placebo (N=110) in Stage B.

    Pre-assignment
    Screening details
    		 In total, 629 participants were screened, and 342 entered the study: 36 entered Stage A directly, and 306 started the run-in period, of whom 286 entered Stage A. Overall, 322 participants entered Stage A. 221 participants from Stage A were randomized in Stage B.

    Period 1
    Period 1 title
    Stage A period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    Stage A was open-label.

    Arms
    Arm title
    		 efgartigimod PH20 SC - Stage A
    Arm description
    		 Up to 360 participants were planned to be enrolled in Stage A. Open-label efgartigimod PH20 SC was administered once weekly in Stage A for up to 12 weeks (with an optional additional week for ECI confirmation), with a minimum of 4 administrations. ECI (evidence of clinical improvement) was achieved through improvement of the INCAT score, or improvement on I-RODS or mean grip strength. Participants remained in Stage A until ECI was confirmed at 2 consecutive visits. Participants who had ECI at 2 consecutive visits during Stage A entered Stage B. Participants without confirmed ECI during Stage A were withdrawn from the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    efgartigimod PH20 SC
    Investigational medicinal product code
    Other name
    Efgartigimod (ARGX-113)
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Stage A: up to 12 weeks (with an optional additional week). Efgartigimod PH20 SC 1000 mg was administered once weekly for at least 4 and up to 13 administrations.

    Number of subjects in period 1
    		efgartigimod PH20 SC - Stage A
    Started
    322
    Completed
    221
    Not completed
    101
         death
    1
         physician decision
    1
         AEs
    20
         prohibited medications
    2
         Rollover to OLE
    22
         other
    5
         Lost to follow-up
    2
         withdrawal by participant
    11
         IMP noncompliance
    1
         Lack of efficacy
    36
    Period 2
    Period 2 title
    Stage B period
    Is this the baseline period?
    		 Yes [1]
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    Stage B was double-blinded.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 efgartigimod PH20 SC - Stage B
    Arm description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 111 subjects were randomized to receive the efgartigimod PH20 SC.
    Arm type
    		 Experimental

    Investigational medicinal product name
    efgartigimod PH20 SC
    Investigational medicinal product code
    Other name
    Efgartigimod (ARGX-113)
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Stage B: Efgartigimod PH20 SC 1000 mg once weekly for up to 48 weeks.

    Arm title
    		 placebo PH20 SC - Stage B
    Arm description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 110 subjects were randomized to receive the placebo.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo PH20 SC
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in vial
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Stage B: 1000 mg placebo PH20 SC (placebo) once weekly for up to 48 weeks.

    Notes
    [1] - Period 1 is not the baseline period. It is expected that period 1 will be the baseline period.
    Justification: Stage B is the baseline period.
    Number of subjects in period 2 [2]
    		efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Started
    111
    110
    Completed
    64
    74
    Not completed
    47
    36
         death
    -
    1
         AEs
    3
    -
         prohibited medications
    2
    1
         Rollover after 88th event
    35
    26
         other
    3
    -
         Sponsor decision
    -
    1
         Lost to follow-up
    -
    2
         withdrawal by participant
    3
    3
         Protocol deviation
    1
    1
         Lack of efficacy
    -
    1
    Notes
    [2] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Reported results are for Stage B period which is baseline period of the study.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    efgartigimod PH20 SC - Stage B
    Reporting group description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 111 subjects were randomized to receive the efgartigimod PH20 SC.

    Reporting group title
    placebo PH20 SC - Stage B
    Reporting group description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 110 subjects were randomized to receive the placebo.

    Reporting group values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B Total
    Number of subjects
    		 111 110 221
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 86 88 174
        From 65-84 years
    		 25 22 47
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 54.5 ( 13.18 ) 51.3 ( 14.47 ) -
    Gender categorical
    Units: Subjects
        Female
    		 38 41 79
        Male
    		 73 69 142

    End points

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    End points reporting groups
    Reporting group title
    efgartigimod PH20 SC - Stage A
    Reporting group description
    		 Up to 360 participants were planned to be enrolled in Stage A. Open-label efgartigimod PH20 SC was administered once weekly in Stage A for up to 12 weeks (with an optional additional week for ECI confirmation), with a minimum of 4 administrations. ECI (evidence of clinical improvement) was achieved through improvement of the INCAT score, or improvement on I-RODS or mean grip strength. Participants remained in Stage A until ECI was confirmed at 2 consecutive visits. Participants who had ECI at 2 consecutive visits during Stage A entered Stage B. Participants without confirmed ECI during Stage A were withdrawn from the study.
    Reporting group title
    efgartigimod PH20 SC - Stage B
    Reporting group description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 111 subjects were randomized to receive the efgartigimod PH20 SC.

    Reporting group title
    placebo PH20 SC - Stage B
    Reporting group description
    		 In Stage B, participants were randomized in a 1:1 ratio to receive once-weekly injections of efgartigimod PH20 SC or placebo. 110 subjects were randomized to receive the placebo.

    Primary: Stage B -Time to First Occurrence of Clinical Deterioration: Hazard ratio

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    End point title
    		 Stage B -Time to First Occurrence of Clinical Deterioration: Hazard ratio
    End point description
    Hazard ratio for time to first adjusted INCAT deterioration, comparing efgartigimod PH20 SC over placebo PH20 SC
    End point type
    Primary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111 [1]
    110 [2]
    Units: Hazard ratio rate
    number (confidence interval 95%)
        Hazard ratio
    0.394 (0.253 to 0.614)
    999 (999 to 999)
    Notes
    [1] - The hazard ratio compares efgartigimod(111) with placebo(110) in one value. 999 is a dummy value.
    [2] - The hazard ratio compares efgartigimod(111) with placebo(110) in one value. 999 is a dummy value.
    Statistical analysis title
    		 Cox Proportional Hazard Model
    Comparison groups
    efgartigimod PH20 SC - Stage B v placebo PH20 SC - Stage B
    Number of subjects included in analysis
    221
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.000039
    Method
    		 Cox Proportional Hazard Model
    Parameter type
    		 Cox proportional hazard
    Point estimate
    0.394
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.253
         upper limit
    		 0.614
    Variability estimate
    Standard deviation

    Primary: Stage A - Percentage of participants with confirmed ECI

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    End point title
    		 Stage A - Percentage of participants with confirmed ECI [3]
    End point description
    Percentage of participants in Stage A with confirmed ECI (Evidence of clinical Improvement)
    End point type
    Primary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    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: No statistical analysis for this Primary End Point.
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    322
    Units: percent
    number (not applicable)
        Number of participants with confirmed ECI
    66.5
    No statistical analyses for this end point

    Primary: Stage B mITT: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline: Hazard Ratio

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    End point title
    		 Stage B mITT: Time to First Adjusted INCAT Deterioration Compared to Stage B Baseline: Hazard Ratio [4]
    End point description
    Hazard ratio for time to first adjusted INCAT deterioration, comparing efgartigimod PH20 SC over placebo PH20 SC
    End point type
    Primary
    End point timeframe
    Up to 48 weeks during randomized placebo-controlled Stage B
    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: Cox Proportional Hazard Model has been added as Statistical analysis.
    End point values
    Number of subjects analysed
    Units: Hazard Ratio rate
    number (confidence interval 95%)
        Time to first aINCAT deterioration
    No statistical analyses for this end point

    Secondary: Stage A - Time to initial confirmed ECI

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    End point title
    		 Stage A - Time to initial confirmed ECI
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    322
    Units: days
    median (confidence interval 95%)
        25th percentile (95% CI) (days)
    22.0 (22.0 to 23.0)
        50th percentile (95% CI) (days)
    43.0 (31.00 to 51.0)
        75th percentile (95% CI) (days)
    71.0 (70.0 to 78.0)
    No statistical analyses for this end point

    Secondary: Stage A - Change from Stage A baseline over time during Stage A in aINCAT score, MRC sum score, 24-item I-RODS score, TUG score and Mean grip strength

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    End point title
    		 Stage A - Change from Stage A baseline over time during Stage A in aINCAT score, MRC sum score, 24-item I-RODS score, TUG score and Mean grip strength
    End point description
    INCAT: Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability. MRC: The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness. I-RODS: The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability. TUG: The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    322
    Units: number
    arithmetic mean (standard deviation)
        aINCAT score
    -0.9 ( 1.71 )
        I-RODS score
    7.7 ( 15.48 )
        Mean grip strength – dominant hand (kPa)
    12.3 ( 18.68 )
        Mean grip strength – nondominant hand (kPa)
    11.2 ( 21.12 )
        MRC Sum Score
    3.8 ( 0.41 )
        TUG score
    -4.3 ( 0.83 )
    No statistical analyses for this end point

    Secondary: Stage A - Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time

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    End point title
    		 Stage A - Changes From Stage A Baseline to Last Assessment in Stage A, in EQ-5D-5L Visual Analog Scale (VAS) Over Time
    End point description
    Scores range from 0-100 with 100 indicating the best health state. Therefore, positive changes indicate higher health-related quality of life reported by the patient.
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    322
    Units: number
    arithmetic mean (standard error)
        Baseline Stage A
    50.8 ( 1.17 )
        Last assessment Stage A
    61.7 ( 1.23 )
    No statistical analyses for this end point

    Secondary: Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events

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    End point title
    		 Stage A: Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
    End point description
    Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    322
    Units: events/100 PYFU
    number (not applicable)
        treatment-emergent adverse events
    1343.1
        treatment-emergent serious adverse events
    51.2
    No statistical analyses for this end point

    Secondary: Stage A - Predose efgartigimod PH20 SC serum concentrations over time

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    End point title
    		 Stage A - Predose efgartigimod PH20 SC serum concentrations over time
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    298
    Units: number
    arithmetic mean (standard deviation)
        Week 1
    14.9 ( 6.92 )
        Week 2
    19.6 ( 8.55 )
        Week 3
    19.7 ( 9.62 )
        Week 4
    18.9 ( 9.96 )
        Week 5
    18.4 ( 8.38 )
        Week 6
    19.2 ( 9.62 )
        Week 7
    17.3 ( 8.89 )
        Week 8
    18.8 ( 8.93 )
        Week 9
    17.8 ( 8.84 )
        Week 10
    17.3 ( 7.62 )
        Week 11
    20.1 ( 9.64 )
        Week 12
    20.0 ( 6.89 )
        Week 13
    14.9 ( 7.24 )
    No statistical analyses for this end point

    Secondary: Stage A - Percent Changes From Stage A Baseline of Serum IgG Levels Over Time

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    End point title
    		 Stage A - Percent Changes From Stage A Baseline of Serum IgG Levels Over Time
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    321
    Units: percent
    arithmetic mean (standard error)
        Week 1
    -36.1 ( 0.58 )
        Week 2
    -54.6 ( 0.80 )
        Week 3
    -63.5 ( 0.71 )
        Week 4
    -66.2 ( 0.89 )
        Week 5
    -67.9 ( 0.74 )
        Week 6
    -67.7 ( 1.42 )
        Week 7
    -66.4 ( 1.96 )
        Week 8
    -64.5 ( 4.28 )
        Week 9
    -68.8 ( 1.17 )
        Week 10
    -67.1 ( 1.90 )
        Week 11
    -70.0 ( 1.41 )
        Week 12
    -67.3 ( 4.44 )
        Week 13
    -51.0 ( 10.00 )
    No statistical analyses for this end point

    Secondary: Stage A - Incidence of Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20

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    End point title
    		 Stage A - Incidence of Binding Antidrug Antibodies (ADA) Towards Efgartigimod or Antibodies (Ab) Against rHuPH20 and Neutralizing Antibodies (NAb) Against Efgartigimod and/or rHuPH20
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 12 weeks during the open-label stage A
    End point values
    		 efgartigimod PH20 SC - Stage A
    Number of subjects analysed
    317
    Units: Incidence
    number (not applicable)
        ADA towards Efgartigimod incidence
    20
        Ab towards rHuPH20 incidence
    45
        NAb against Efgartigimod incidence
    1
        NAb against rHuPH20 incidence
    0
    No statistical analyses for this end point

    Secondary: Stage B - Time to CIDP Disease Progression

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    End point title
    		 Stage B - Time to CIDP Disease Progression
    End point description
    Time to chronic inflammatory demyelinating polyneuropathy (CIDP) disease progression is defined by the time from first dose of double-blind IMP to the first I-RODS score decrease ≥4 points compared to Stage B baseline using the centile metric. '00' is reported as the median (95% CI) time to CIDP Disease Progression could not be calculated because less than 50% of the participants showed CIDP Disease Progression in the efgartigimod PH20 SC group
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111 [5]
    110
    Units: days
    median (confidence interval 95%)
        Time to CIDP Disease Progression
    999 (247.0 to 999)
    85 (50.0 to 253.0)
    Notes
    [5] - '999' is a dummy value as it should be 'NA'
    No statistical analyses for this end point

    Secondary: Stage B - Percentage of participants with improved functional level compared to Stage B baseline, as measured by an increase in the 24-item I-RODS score up to week 48

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    End point title
    		 Stage B - Percentage of participants with improved functional level compared to Stage B baseline, as measured by an increase in the 24-item I-RODS score up to week 48
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    110
    Units: percent
    number (not applicable)
        Improved Functional Level
    45.0
    36.4
    No statistical analyses for this end point

    Secondary: Stage B - Change from Stage B baseline over time in aINCAT score, MRC sum score, 24-item I-RODS score, TUG score and Mean grip strength

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    End point title
    		 Stage B - Change from Stage B baseline over time in aINCAT score, MRC sum score, 24-item I-RODS score, TUG score and Mean grip strength
    End point description
    aINCAT: Adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) scores range from 0-10 with a score of 10 indicating the greatest degree of disability. MRC: The Medical Research Council (MRC) Sum scores range from 0 to 60 with a lower score indicating greater muscle weakness. I-RODS: The Inflammatory Rasch-built Overall Disability Scale (I-RODS) score ranges from 0-100, with lower scores indicating the greatest degree of disability. TUG: The Timed Up and Go (TUG) score is calculated as the number of seconds needed to complete a series of actions. The longer time needed to complete this test (expressed in seconds) indicates lower mobility.
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    110
    Units: number
    arithmetic mean (standard deviation)
        aINCAT score
    0.1 ( 1.08 )
    0.9 ( 1.98 )
        I-RODS score
    0.8 ( 12.33 )
    -7.0 ( 19.10 )
        Mean grip strength – dominant hand (kPa)
    2.1 ( 13.29 )
    -8.2 ( 20.69 )
        Mean grip strength – nondominant hand (kPa)
    2.0 ( 17.33 )
    -6.9 ( 21.30 )
        MRC Sum score
    -0.3 ( 0.43 )
    -3 ( 0.86 )
        TUG score
    0.8 ( 0.36 )
    1.9 ( 0.60 )
    No statistical analyses for this end point

    Secondary: Stage B - Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events

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    End point title
    		 Stage B - Exposure Adjusted Occurrence of Treatment-emergent Adverse Events and Serious Adverse Events
    End point description
    Treatment-emergent (serious) AEs expressed in number of events/100 PYFU (participant years of follow-up)
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    110
    Units: Events/100 PYFU
    number (not applicable)
        treatment-emergent adverse events
    347.6
    510.9
        treatment-emergent serious adverse events
    14.1
    19.0
    No statistical analyses for this end point

    Secondary: Stage B - Predose efgartigimod PH20 SC serum concentrations over time

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    End point title
    		 Stage B - Predose efgartigimod PH20 SC serum concentrations over time
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    110
    Units: number
    arithmetic mean (standard deviation)
        Week 4
    18.4 ( 10.3 )
    0.267 ( 0.365 )
        Week 8
    17.2 ( 9.11 )
    0 ( 0 )
        Week 12
    18.1 ( 9.48 )
    0 ( 0 )
        Week 16
    16.9 ( 9.03 )
    0 ( 0 )
        Week 20
    16.8 ( 8.36 )
    0 ( 0 )
        Week 24
    16.0 ( 7.89 )
    0 ( 0 )
        Week 28
    18.5 ( 10.2 )
    0 ( 0 )
        Week 32
    18.0 ( 9.50 )
    0 ( 0 )
        Week 36
    17.9 ( 10.7 )
    0 ( 0 )
        Week 40
    16.2 ( 8.20 )
    0 ( 0 )
        Week 44
    18.1 ( 10.2 )
    0 ( 0 )
        Week 48
    16.3 ( 8.18 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Stage B - Percent Changes of Serum IgG Levels Over Time

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    End point title
    		 Stage B - Percent Changes of Serum IgG Levels Over Time
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    110
    Units: percent
    arithmetic mean (standard error)
        Week 4
    -68.2 ( 1.14 )
    -36.7 ( 1.91 )
        Week 8
    -68.2 ( 1.24 )
    -10.9 ( 2.41 )
        Week 12
    -69.8 ( 1.09 )
    -7.1 ( 2.54 )
        Week 16
    -67.4 ( 1.49 )
    -2.1 ( 3.09 )
        Week 20
    -67.8 ( 1.30 )
    2.0 ( 3.61 )
        Week 24
    -67.2 ( 1.52 )
    1.7 ( 4.22 )
        Week 28
    -67.9 ( 1.42 )
    -2.3 ( 4.31 )
        Week 32
    -68.5 ( 1.63 )
    0.6 ( 5.18 )
        Week 36
    -64.7 ( 2.82 )
    -3.6 ( 4.12 )
        Week 40
    -68.0 ( 1.91 )
    -4.2 ( 4.98 )
        Week 44
    -67.8 ( 1.92 )
    -3.6 ( 6.50 )
        Week 48
    -66.6 ( 1.85 )
    -0.9 ( 7.36 )
    No statistical analyses for this end point

    Secondary: Stage B - Incidence of ADA against efgartigimod and antibodies against rHuPH20

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    End point title
    		 Stage B - Incidence of ADA against efgartigimod and antibodies against rHuPH20
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 48 weeks during the randomized placebo-controlled stage B
    End point values
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B
    Number of subjects analysed
    111
    109
    Units: Incidence
    number (not applicable)
        ADA towards Efgartigimod incidence
    2
    64
        Ab towards rHuPH20 incidence
    52
    32
        NAb against efgartigimod incidence
    0
    13
        NAb against rHuPH20 incidence
    5
    2
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs reported from the first dose of IMP until 28 days after the last dose of IMP were considered treatment-emergent and were summarized descriptively.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    efgartigimod PH20 SC - Stage B
    Reporting group description
    		 Participants who completed stage A and received efgartigimod PH20 SC in stage B

    Reporting group title
    placebo PH20 SC - Stage B
    Reporting group description
    		 Participants who completed stage A and received placebo PH20 SC in stage B

    Reporting group title
    efgartigimod PH20 SC - Stage A
    Reporting group description
    		 Participants receiving efgartigimod PH20 SC in stage A

    Serious adverse events
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B efgartigimod PH20 SC - Stage A
    Total subjects affected by serious adverse events
         subjects affected / exposed
    6 / 111 (5.41%)
    6 / 110 (5.45%)
    21 / 322 (6.52%)
         number of deaths (all causes)
    0
    1
    2
         number of deaths resulting from adverse events
    0
    1
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Lipoma
         subjects affected / exposed
    0 / 111 (0.00%)
    1 / 110 (0.91%)
    0 / 322 (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
    Prostate cancer
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Transitional cell carcinoma
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Squamous cell carcinoma of skin
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Concussion
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Foot fracture
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac arrest
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Chronic inflammatory demyelinating polyradiculoneuropathy
         subjects affected / exposed
    0 / 111 (0.00%)
    1 / 110 (0.91%)
    14 / 322 (4.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 16
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Quadriparesis
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Ear and labyrinth disorders
    Deafness unilateral
         subjects affected / exposed
    0 / 111 (0.00%)
    1 / 110 (0.91%)
    0 / 322 (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
    Hepatobiliary disorders
    Cholelithiasis
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urethral stenosis
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary bladder polyp
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Calculus urinary
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Appendicitis
         subjects affected / exposed
    0 / 111 (0.00%)
    1 / 110 (0.91%)
    0 / 322 (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
    COVID-19 pneumonia
         subjects affected / exposed
    1 / 111 (0.90%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 111 (0.00%)
    2 / 110 (1.82%)
    0 / 322 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    1 / 1
    0 / 0
    Clostridium difficile colitis
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Suspected COVID-19
         subjects affected / exposed
    0 / 111 (0.00%)
    0 / 110 (0.00%)
    1 / 322 (0.31%)
         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
    0 / 111 (0.00%)
    1 / 110 (0.91%)
    0 / 322 (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
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 efgartigimod PH20 SC - Stage B placebo PH20 SC - Stage B efgartigimod PH20 SC - Stage A
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    31 / 111 (27.93%)
    15 / 110 (13.64%)
    60 / 322 (18.63%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 111 (3.60%)
    2 / 110 (1.82%)
    16 / 322 (4.97%)
         occurrences all number
    6
    2
    28
    General disorders and administration site conditions
    Injection site bruising
         subjects affected / exposed
    6 / 111 (5.41%)
    1 / 110 (0.91%)
    4 / 322 (1.24%)
         occurrences all number
    6
    1
    5
    Injection site erythema
         subjects affected / exposed
    6 / 111 (5.41%)
    0 / 110 (0.00%)
    33 / 322 (10.25%)
         occurrences all number
    6
    0
    53
    Infections and infestations
    COVID-19
         subjects affected / exposed
    19 / 111 (17.12%)
    14 / 110 (12.73%)
    6 / 322 (1.86%)
         occurrences all number
    20
    14
    7
    Upper respiratory tract infection
         subjects affected / exposed
    2 / 111 (1.80%)
    11 / 110 (10.00%)
    11 / 322 (3.42%)
         occurrences all number
    3
    11
    12

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Jan 2020
    The primary endpoint of Stage B was updated: 1. The time window for confirmation of the first occurrence of clinical deterioration (defined by an increase of ≥1 point in aINCAT score compared with Stage B baseline) was shortened to reduce the time of clinical deterioration and offer follow-up treatment as soon as possible. 2. A clarification was added that to prevent further deterioration, no confirmation was needed for a clinical deterioration (increase in aINCAT score of ≥2 points compared with baseline). Clarification of the eligibility criterion was added: The lowest possible INCAT score of 2 had to be exclusively from leg disability.
    04 May 2020
    COVID-19 mitigation measures were added. Additional IMP blinding measures were added (using blinded IMP vials and masked syringes). A withdrawal criterion was added for Stage A. The requirement of a 4-week minimum in the run-in period before entering Stage A was removed.
    30 Nov 2020
    The definition of ECI was updated to include I-RODS and/or grip strength (for participants with no change in aINCAT during run-in). Inclusion criterion #6 was updated to specify that “current” CIDP treatment at screening means “within the last 6 months.” Based on nonclinical teratogenicity and reproductive toxicity data, female participants could use acceptable contraception methods (in addition to highly effective methods) and male contraception was no longer required; however, male participants had to agree not to donate sperm during the study and for 90 days after. Clarification was added for INCAT scores at baseline (with different baselines during the study) and aINCAT scores at postbaseline visits. The safety section was updated based on the current Investigator’s Brochure at the time of the amendment. Further clarification was provided on how to perform a home visit during the COVID-19 pandemic.
    12 Oct 2022
    EQ-5D-5L was escalated from an exploratory endpoint to a secondary endpoint. As an additional blinding precaution, local IgG measurements were no longer allowed, and total protein and albumin results were not sent to the sites (or not measured if a local laboratory was used) after the Stage B baseline. IgG subtype measurements were removed from the protocol. A change was made to allow participants in the run-in period at the time the study stopped (ie, at the 88th event) to roll over to the OLE study. The maximum number of 180 randomized participants in Stage B was removed. Clarification was added that additional tests could be performed to confirm the CIDP diagnosis. The list of prohibited medications was updated.Based on nonclinical teratogenicity and reproductive toxicity data, the inclusion and exclusion criteria were updated: 1) Female participants could stop their contraception method at the date of the last IMP dose; 2) Female participants could become pregnant immediately after the study; 3) Male participants could donate sperm. Integration of country-specific requirements (except for China) in the global protocol.

    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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