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    Clinical Trial Results:
    A Long-term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia Gravis

    Summary
    EudraCT number
    		 2020-004086-38
    Trial protocol
    		 DE   HU   CZ   BE   NL   IT   ES  
    Global end of trial date
    31 Dec 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Dec 2025
    First version publication date
    26 Dec 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ARGX-113-2002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04818671
    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 BV, 0032 93103400, regulatory@argenx.com
    Scientific contact
    Regulatory, argenx BV, 0032 93103400, 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
    05 Jun 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Dec 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the long-term safety and tolerability of efgartigimod PH20 SC in participants with Generalized Myasthenia Gravis (gMG)
    Protection of trial subjects
    The protocol, protocol amendments, ICFs and participant recruitment information were approved by the IRB, IEC and regulatory agency before participants were enrolled. 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. Participants or their legally authorized representative were required to sign a statement of informed consent that met the requirements of the local regulations, ICH guidelines, privacy and data protection requirements, where applicable, and the IRB/IEC or study site.
    Background therapy
    		 Participants must have been receiving a stable dose of concomitant gMG therapy at study entry. During year 1 of the study, participants could reduce their dose of steroid, NSID, or AChE inhibitor after week 4 of a cycle and before the next cycle. Changes to concomitant gMG therapy were unrestricted from year 2 onward and based on the discretion of the investigator. The medications and vaccinations allowed or prohibited during and/or after efgartigimod PH20 SC administration are described in the protocol.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    05 Apr 2021
    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
    Georgia: 33
    Country: Number of subjects enrolled
    Japan: 16
    Country: Number of subjects enrolled
    Russian Federation: 7
    Country: Number of subjects enrolled
    United States: 33
    Country: Number of subjects enrolled
    Netherlands: 3
    Country: Number of subjects enrolled
    Poland: 60
    Country: Number of subjects enrolled
    Spain: 3
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Czechia: 3
    Country: Number of subjects enrolled
    Germany: 4
    Country: Number of subjects enrolled
    Hungary: 4
    Country: Number of subjects enrolled
    Italy: 8
    Worldwide total number of subjects
    180
    EEA total number of subjects
    91
    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)
    139
    From 65 to 84 years
    41
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted at 47 sites that enrolled participants in 12 countries. A total of 184 participants rolled over from ARGX-113-2001 and ARGX-113-1705, of whom 180 participants were exposed to efgartigimod PH20 SC treatment.

    Pre-assignment
    Screening details
    		 All participants were adults with gMG who participated in ARGX-113-2001 or ARGX-113-1705. No randomization or blinding was performed because this was an open-label study.

    Period 1
    Period 1 title
    Treatment period (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    This is an open-label study.

    Arms
    Arm title
    		 Efgartigimod PH20 SC
    Arm description
    		 All participants who were exposed to efgartigimod PH20 SC in this study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod PH20 SC
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Efgartigimod PH20 SC 1000 mg was administered in cycles. Each cycle comprising a treatment period (TP) and an intertreatment period (IP). 4 once-weekly injections of efgartigimod PH20 SC were administrated over 3 weeks during the TP. The duration of the IP was based on clinical evaluation with a minimum of 28 days in year 1 and 7 days in year 2. Once considered capable for (self-)administration, participants and caregivers could perform SC injections at home except for the first administration in a cycle’s TP.

    Number of subjects in period 1
    		Efgartigimod PH20 SC
    Started
    180
    Completed
    138
    Not completed
    42
         Physician decision
    4
         Consent withdrawn by subject
    16
         Adverse event, non-fatal
    5
         Death
    4
         Requires prohibited medication
    4
         Sponsor request
    1
         Approved drug available for indication
    1
         Lost to follow-up
    2
         Lack of efficacy
    5

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Efgartigimod PH20 SC
    Reporting group description
    		 All participants who were exposed to efgartigimod PH20 SC in this study.

    Reporting group values
    		 Efgartigimod PH20 SC Total
    Number of subjects
    		 180 180
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 139 139
        From 65-84 years
    		 41 41
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 50.8 ( 15.50 ) -
    Gender categorical
    Units: Subjects
        Female
    		 119 119
        Male
    		 61 61
    Race
    Units: Subjects
        Asian (Japanese)
    		 16 16
        Black or African American
    		 2 2
        White
    		 161 161
        Multiple
    		 1 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 8 8
        Not Hispanic or Latino
    		 172 172

    End points

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    End points reporting groups
    Reporting group title
    Efgartigimod PH20 SC
    Reporting group description
    		 All participants who were exposed to efgartigimod PH20 SC in this study.

    Primary: Number of AEs, SAEs and AESIs

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    End point title
    		 Number of AEs, SAEs and AESIs [1]
    End point description
    Adverse events, Serious Adverse event and Adverse events of special interest. Adverse events in the 'Infections and infestations' SOC were defined as AESIs because efgartigimod causes a transient reduction in total IgG levels.
    End point type
    Primary
    End point timeframe
    Up to 3.5 years
    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: Since this is an open-label extension study, inferential statistics are not applicable.
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    180
    Units: events
        Number of AEs
    3325
        Number of SAEs
    107
        Number of AESIs
    420
    No statistical analyses for this end point

    Secondary: MG-ADL total score changes from baseline

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    End point title
    		 MG-ADL total score changes from baseline
    End point description
    The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported scale that assesses MG symptoms and their effects on daily activities. It evaluates a participant’s capacity to perform different activities in their daily life. The total score ranges from 0 to 24 with higher scores indicating more impairment.
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    180
    Units: score on scale
    arithmetic mean (standard error)
        Cycle 1, Week 1 (n=180)
    -2.2 ( 0.20 )
        Cycle 1, Week 2 (n=176)
    -3.4 ( 0.21 )
        Cycle 1, Week 3 (n=176)
    -3.9 ( 0.22 )
        Cycle 1, Week 4 (n=170)
    -4.0 ( 0.23 )
    No statistical analyses for this end point

    Secondary: Percent Change in total IgG levels from baseline

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    End point title
    		 Percent Change in total IgG levels from baseline
    End point description
    IgG: immunoglobulin G
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    164 [2]
    Units: percent
    arithmetic mean (standard error)
        Cycle 1, Week 4
    -62.6 ( 0.93 )
    Notes
    [2] - Only participants with an assessment at baseline and week 4 of first cycle are reported here.
    No statistical analyses for this end point

    Secondary: Percent Change in AChR-Ab From Baseline in AChR-Ab Seropositive Participants

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    End point title
    		 Percent Change in AChR-Ab From Baseline in AChR-Ab Seropositive Participants
    End point description
    AchR-Ab: anti-acetylcholine receptor antibodies.
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    124 [3]
    Units: percent change
    arithmetic mean (standard error)
        Cycle 1, Week 4
    -57.6 ( 2.29 )
    Notes
    [3] - Only AChR-Ab seropositive participants assessed at baseline and week 4 of first cycle are reported.
    No statistical analyses for this end point

    Secondary: Efgartigimod serum concentrations

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    End point title
    		 Efgartigimod serum concentrations
    End point description
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    141 [4]
    Units: ng/mL
    arithmetic mean (standard deviation)
        Cycle 1, Week 4 (n=141)
    21693 ( 8242 )
    Notes
    [4] - Only participants with an assessment at week 4 of first cycle are reported here.
    No statistical analyses for this end point

    Secondary: Incidence of ADAs Against Efgartigimod Over Time

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    End point title
    		 Incidence of ADAs Against Efgartigimod Over Time
    End point description
    ADA: Anti-drug antibodies
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    178 [5]
    Units: Participants
    35
    Notes
    [5] - The number analyzed is the total number of ADA evaluable participants.
    No statistical analyses for this end point

    Secondary: Incidence of NAbs Against Efgartigimod Over Time

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    End point title
    		 Incidence of NAbs Against Efgartigimod Over Time
    End point description
    NAbs: neutralizing antibodies
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    178 [6]
    Units: Participants
    11
    Notes
    [6] - The number analyzed is the total number of NAb-evaluable participants.
    No statistical analyses for this end point

    Secondary: Incidence of Antibodies Against rHuPH20 Over Time

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    End point title
    		 Incidence of Antibodies Against rHuPH20 Over Time
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    178 [7]
    Units: Participants
    49
    Notes
    [7] - The number analyzed is the total number of rHuPH20 Ab evaluable participants.
    No statistical analyses for this end point

    Secondary: Incidence of NAbs Against rHuPH20 Over Time

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    End point title
    		 Incidence of NAbs Against rHuPH20 Over Time
    End point description
    NAbs: neutralizing antibodies
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    167 [8]
    Units: Participants
    1
    Notes
    [8] - The number analyzed is the total number of rHuPH20 NAb-evaluable participants.
    No statistical analyses for this end point

    Secondary: Changes in Total MG-QoL15r From Baseline

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    End point title
    		 Changes in Total MG-QoL15r From Baseline
    End point description
    Myasthenia Gravis Quality of Life 15 item scale revised (MG-QoL 15r) scores range from 0 to 30 with a higher score representing more severe symptoms.
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    180
    Units: score
    arithmetic mean (standard error)
        Cycle 1, Week 1 (n=179)
    -2.5 ( 0.29 )
        Cycle 1, Week 2 (n=175)
    -3.7 ( 0.34 )
        Cycle 1, Week 3 (n=175)
    -4.5 ( 0.38 )
        Cycle 1, Week 4 (n=168)
    -4.7 ( 0.39 )
    No statistical analyses for this end point

    Secondary: Changes in EQ-5D-5L VAS Score From Baseline

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    End point title
    		 Changes in EQ-5D-5L VAS Score From Baseline
    End point description
    EuroQoL 5 Dimensions 5-Level (EQ-5D-5L) visual analog scale (VAS) scores range from 0 to 100 with a higher score representing better health.
    End point type
    Secondary
    End point timeframe
    Up to week 4 of the first cycle
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    169 [9]
    Units: score on a scale
    arithmetic mean (standard error)
        Cycle 1, Week 4
    13.9 ( 1.32 )
    Notes
    [9] - Only participants with an assessment at baseline and week 4 of first cycle are reported here.
    No statistical analyses for this end point

    Secondary: Percent of Participants or Caregivers by Number of Training Visits Needed to be Competent to Start Self- or Caregiver-Supported Administration

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    End point title
    		 Percent of Participants or Caregivers by Number of Training Visits Needed to be Competent to Start Self- or Caregiver-Supported Administration
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    166 [10]
    Units: percent
    number (not applicable)
        0 visits
    4.8
        1 visit
    45.8
        2 visits
    13.9
        3 visits
    11.4
        4 visits
    12.7
        5 visits
    1.8
        6 visits
    5.4
        9 visits
    1.2
        11 visits
    0.6
        12 visits
    0.6
        22 visits
    0.6
        37 visits
    0.6
        42 visits
    0.6
    Notes
    [10] - Only participants/caregivers trained and capable of self- or caregiver-administrations are reported.
    No statistical analyses for this end point

    Secondary: Percent of Self- or Caregiver-supported Study Drug Administration Among All Study Treatment Visits at Home

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    End point title
    		 Percent of Self- or Caregiver-supported Study Drug Administration Among All Study Treatment Visits at Home
    End point description
    End point type
    Secondary
    End point timeframe
    Up to 3.5 years
    End point values
    		 Efgartigimod PH20 SC
    Number of subjects analysed
    180
    Units: percent
    number (not applicable)
        Participants at home
    47.5
        Caregivers at home
    1.7
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 3.5 years
    Adverse event reporting additional description
    Any clinically significant changes occurring during the study were reported as adverse events.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    Total
    Reporting group description
    		 All participants who were exposed to efgartigimod PH20 SC in this study.

    Serious adverse events
    		 Total
    Total subjects affected by serious adverse events
         subjects affected / exposed
    55 / 180 (30.56%)
         number of deaths (all causes)
    5
         number of deaths resulting from adverse events
    5
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Bladder transitional cell carcinoma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metastases to liver
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Ovarian adenoma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Rectal cancer stage IV
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal cancer metastatic
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal cell carcinoma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Thymoma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Uterine leiomyoma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Brachiocephalic vein stenosis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypertension
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Surgical and medical procedures
    Umbilical hernia repair
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Uterine polypectomy
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory failure
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Chronic respiratory failure
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Hydrothorax
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Interstitial lung disease
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary mass
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory failure
         subjects affected / exposed
    4 / 180 (2.22%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 3
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Post procedural complication
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Rib fracture
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Shunt occlusion
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Shunt stenosis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spinal fracture
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tendon injury
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Acute myocardial infarction
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Angina unstable
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Cardiac arrest
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Cardiac failure congestive
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Coma
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Myasthenia gravis
         subjects affected / exposed
    11 / 180 (6.11%)
         occurrences causally related to treatment / all
    1 / 17
         deaths causally related to treatment / all
    0 / 0
    Myasthenia gravis crisis
         subjects affected / exposed
    4 / 180 (2.22%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    Syncope
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Diplopia
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Retinal detachment
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vitreous haemorrhage
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Gastric ulcer haemorrhage
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Inguinal hernia
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Intestinal perforation
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Terminal ileitis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Renal failure
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Enthesopathy
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Muscular weakness
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Osteoarthritis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spinal osteoarthritis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Spondyloarthropathy
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Anal abscess
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Bacteraemia
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cellulitis
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Cellulitis gangrenous
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    COVID-19
         subjects affected / exposed
    4 / 180 (2.22%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 1
    COVID-19 pneumonia
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diarrhoea infectious
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diverticulitis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Herpes zoster
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Orchitis
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    7 / 180 (3.89%)
         occurrences causally related to treatment / all
    1 / 7
         deaths causally related to treatment / all
    0 / 0
    Rotavirus infection
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    2 / 180 (1.11%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 180 (0.56%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Viral infection
         subjects affected / exposed
    1 / 180 (0.56%)
         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
    		 Total
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    167 / 180 (92.78%)
    Investigations
    Activated partial thromboplastin time prolonged
         subjects affected / exposed
    12 / 180 (6.67%)
         occurrences all number
    15
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    9 / 180 (5.00%)
         occurrences all number
    13
    Vascular disorders
    Hypertension
         subjects affected / exposed
    14 / 180 (7.78%)
         occurrences all number
    17
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    13 / 180 (7.22%)
         occurrences all number
    18
    Headache
         subjects affected / exposed
    48 / 180 (26.67%)
         occurrences all number
    205
    Myasthenia gravis
         subjects affected / exposed
    18 / 180 (10.00%)
         occurrences all number
    23
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    18 / 180 (10.00%)
         occurrences all number
    33
    Iron deficiency anaemia
         subjects affected / exposed
    13 / 180 (7.22%)
         occurrences all number
    15
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    10 / 180 (5.56%)
         occurrences all number
    20
    Influenza like illness
         subjects affected / exposed
    14 / 180 (7.78%)
         occurrences all number
    20
    Injection site bruising
         subjects affected / exposed
    24 / 180 (13.33%)
         occurrences all number
    80
    Injection site erythema
         subjects affected / exposed
    55 / 180 (30.56%)
         occurrences all number
    727
    Injection site haematoma
         subjects affected / exposed
    10 / 180 (5.56%)
         occurrences all number
    16
    Injection site pain
         subjects affected / exposed
    22 / 180 (12.22%)
         occurrences all number
    47
    Injection site pruritus
         subjects affected / exposed
    21 / 180 (11.67%)
         occurrences all number
    63
    Injection site rash
         subjects affected / exposed
    15 / 180 (8.33%)
         occurrences all number
    32
    Injection site reaction
         subjects affected / exposed
    10 / 180 (5.56%)
         occurrences all number
    47
    Injection site swelling
         subjects affected / exposed
    11 / 180 (6.11%)
         occurrences all number
    100
    Pyrexia
         subjects affected / exposed
    9 / 180 (5.00%)
         occurrences all number
    12
    Eye disorders
    Cataract
         subjects affected / exposed
    9 / 180 (5.00%)
         occurrences all number
    13
    Gastrointestinal disorders
    Abdominal pain upper
         subjects affected / exposed
    11 / 180 (6.11%)
         occurrences all number
    14
    Diarrhoea
         subjects affected / exposed
    32 / 180 (17.78%)
         occurrences all number
    45
    Nausea
         subjects affected / exposed
    16 / 180 (8.89%)
         occurrences all number
    25
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    12 / 180 (6.67%)
         occurrences all number
    14
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    17 / 180 (9.44%)
         occurrences all number
    22
    Back pain
         subjects affected / exposed
    22 / 180 (12.22%)
         occurrences all number
    28
    Muscle spasms
         subjects affected / exposed
    11 / 180 (6.11%)
         occurrences all number
    14
    Pain in extremity
         subjects affected / exposed
    11 / 180 (6.11%)
         occurrences all number
    14
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    9 / 180 (5.00%)
         occurrences all number
    13
    COVID-19
         subjects affected / exposed
    52 / 180 (28.89%)
         occurrences all number
    62
    Influenza
         subjects affected / exposed
    9 / 180 (5.00%)
         occurrences all number
    9
    Nasopharyngitis
         subjects affected / exposed
    36 / 180 (20.00%)
         occurrences all number
    55
    Upper respiratory tract infection
         subjects affected / exposed
    32 / 180 (17.78%)
         occurrences all number
    75
    Urinary tract infection
         subjects affected / exposed
    16 / 180 (8.89%)
         occurrences all number
    23

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 May 2021
    Protocol v2.0 • Added optional PK sample collection visits with an associated objective and endpoint • Defined “caregiver” and provided information about the caregiver ICF • Added vaccination history collection for participants entering the study from ARGX-113-1705 • Added a retreatment condition to ensure participants can complete a full cycle before the end of the study • Required site staff to review the administration log and participant diary • Added specialty laboratory tests • Defined sexual abstinence as a contraceptive method
    02 Feb 2023
    Protocol v3.0 • Extended the study duration to collect data past 2 years of efgartigimod PH20 SC exposure. Participants can remain in the study until, at the latest, 31 Dec 2024 • Reduced the number of assessments from year 2 onward • Removed the 28-day restriction between TPs and the limit of 14 TPs from year 2 onward • Clarified inclusion criterion 2a so that participants who discontinued from an antecedent study for a non–life-threatening SAE are eligible • Removed the requirement that participants receive concurrent gMG therapy • Clarified in informed consent language that participants who are enrolled in another study with no therapeutic intervention are not to be excluded or discontinued • Added a requirement to permanently discontinue efgartigimod PH20 SC for participants diagnosed with a malignancy (except basal cell carcinoma of the skin) • Clarified the prohibited therapies requiring study discontinuation and prohibited activities during year 1 that do not require study discontinuation • Removed the requirement for male contraception

    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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