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    Clinical Trial Results:
    An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients with Pemphigus (ADDRESS+)

    Summary
    EudraCT number
    		 2020-002917-16
    Trial protocol
    		 DE   HU   ES   GR   BG   FR   IT  
    Global end of trial date
    25 Mar 2024

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Jan 2025
    First version publication date
    04 Jan 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ARGX-113-1905
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04598477
    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
    31 Oct 2024
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Mar 2024
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To assess the long-term safety of treatment, tolerability, and efficacy of efgartigimod PH20 SC in participants with pemphigus vulgaris (PV) or pemphigus foliaceus (PF) who participated in ARGX-113-1904
    Protection of trial subjects
    The protocol, protocol amendments, ICFs, Investigator Brochure, and participant recruitment information were approved by the IEC/IRB 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 were required to sign a statement of informed consent that met the requirements of the local regulations, ICH guidelines, Health Insurance Portability and Accountability Act requirements, where applicable, and the IRB/IEC or study center.
    Background therapy
    		 Similar to the antecedent study ARGX-113-1904, participants could also receive concomittant prednisone. Investigators could increase or decrease the dose based on protocol-specified criteria.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    25 Oct 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
    Australia: 4
    Country: Number of subjects enrolled
    China: 25
    Country: Number of subjects enrolled
    Georgia: 6
    Country: Number of subjects enrolled
    India: 11
    Country: Number of subjects enrolled
    Japan: 8
    Country: Number of subjects enrolled
    Serbia: 1
    Country: Number of subjects enrolled
    Türkiye: 3
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    Ukraine: 12
    Country: Number of subjects enrolled
    United States: 13
    Country: Number of subjects enrolled
    Russian Federation: 15
    Country: Number of subjects enrolled
    Poland: 17
    Country: Number of subjects enrolled
    Romania: 2
    Country: Number of subjects enrolled
    Spain: 4
    Country: Number of subjects enrolled
    Bulgaria: 13
    Country: Number of subjects enrolled
    France: 8
    Country: Number of subjects enrolled
    Germany: 13
    Country: Number of subjects enrolled
    Greece: 9
    Country: Number of subjects enrolled
    Hungary: 6
    Country: Number of subjects enrolled
    Italy: 11
    Worldwide total number of subjects
    183
    EEA total number of subjects
    83
    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)
    159
    From 65 to 84 years
    24
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted at 76 sites that enrolled participants in 20 countries.

    Pre-assignment
    Screening details
    		 A total of 183 participants rolled over from ARGX-113-1904. Of these, 57 participants had a CRmin status (complete remission on minimal prednisone therapy) at rollover of which 34 participants did not receive efgartigimod PH20 SC in this ARGX-113-1905 study.

    Period 1
    Period 1 title
    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
    Are arms mutually exclusive
    Yes

    Arm title
    		 Efgartigimod-efgartigimod PH20 SC
    Arm description
    		 Participants who received efgartigimod PH20 SC in antecedent study ARGX-113-1904.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod PH20 SC
    Investigational medicinal product code
    ARGX-113 PH20 SC
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    At baseline, eligible participants received efgartigimod PH20 SC according to their clinical status at the rollover visit and was administered until participants achieved CRmin. Participants could continue to receive a prednisone (or equivalent) dose according to their clinical status at the rollover visit, and the dose was tapered or escalated based on clinical status at the investigator’s discretion following protocol-specified instructions.

    Arm title
    		 Placebo-efgartigimod PH20 SC
    Arm description
    		 Participants who received placebo PH20 SC in antecedent study ARGX-113-1904.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod PH20 SC
    Investigational medicinal product code
    ARGX-113 PH20 SC
    Other name
    Pharmaceutical forms
    Solution for injection
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    At baseline, eligible participants received efgartigimod PH20 SC according to their clinical status at the rollover visit and was administered until participants achieved CRmin. Participants could continue to receive a prednisone (or equivalent) dose according to their clinical status at the rollover visit, and the dose was tapered or escalated based on clinical status at the investigator’s discretion following protocol-specified instructions.

    Number of subjects in period 1
    		Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Started
    123
    60
    Completed
    64
    23
    Not completed
    59
    37
         Adverse event, serious fatal
    -
    1
         Required prohibited medication
    -
    1
         Consent withdrawn by subject
    16
    12
         Physician decision
    7
    2
         Adverse event, non-fatal
    2
    -
         Not specified
    15
    8
         Pregnancy
    -
    1
         Study terminated by sponsor
    18
    12
         Lost to follow-up
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Efgartigimod-efgartigimod PH20 SC
    Reporting group description
    		 Participants who received efgartigimod PH20 SC in antecedent study ARGX-113-1904.

    Reporting group title
    Placebo-efgartigimod PH20 SC
    Reporting group description
    		 Participants who received placebo PH20 SC in antecedent study ARGX-113-1904.

    Reporting group values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC Total
    Number of subjects
    		 123 60 183
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 108 51 159
        From 65-84 years
    		 15 9 24
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 50.1 ( 11.40 ) 52.4 ( 13.02 ) -
    Gender categorical
    Units: Subjects
        Female
    		 61 32 93
        Male
    		 62 28 90

    End points

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    End points reporting groups
    Reporting group title
    Efgartigimod-efgartigimod PH20 SC
    Reporting group description
    		 Participants who received efgartigimod PH20 SC in antecedent study ARGX-113-1904.

    Reporting group title
    Placebo-efgartigimod PH20 SC
    Reporting group description
    		 Participants who received placebo PH20 SC in antecedent study ARGX-113-1904.

    Subject analysis set title
    Rollover Analysis Set
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The rollover analysis set included all participants who rolled over from study ARGX-113-1904, regardless of whether or not they received efgartigimod PH20 SC treatment as part of this study. Additional restrictions to the analysis set might apply in the different outcome measures. These are described as notes to the number of subjects analyzed.

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    The safety analysis set included participants who received at least 1 dose of efgartigimod PH20 SC during this study. Additional restrictions to the analysis set might apply in the different outcome measures. These are described in the number of subjects analyzed.

    Primary: Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)

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    End point title
    		 Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE) [1]
    End point description
    Incidence rates were calculated as 100 × n/PYFU. PYFU=participant-years of follow-up. The safety data sets includes participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF).
    End point type
    Primary
    End point timeframe
    Up to Week 60
    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: No statistical analysis was applied to this end point
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    101 [2]
    48 [3]
    Units: number
    number (not applicable)
        TEAE
    116.4
    113.0
        AESI
    72.0
    69.3
        SAE
    24.5
    14.6
    Notes
    [2] - Safety set
    [3] - Safety set
    No statistical analyses for this end point

    Secondary: Proportion of participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF) who achieve CRmin

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    End point title
    		 Proportion of participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF) who achieve CRmin
    End point description
    CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
    End point type
    Secondary
    End point timeframe
    Up to 60 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    101 [4]
    48 [5]
    Units: participants, n
    55
    26
    Notes
    [4] - Safety set
    [5] - Safety set
    No statistical analyses for this end point

    Secondary: Proportion of participants with pemphigus vulgaris (PV) participants who achieve CRmin

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    End point title
    		 Proportion of participants with pemphigus vulgaris (PV) participants who achieve CRmin
    End point description
    CRmin (complete clinical remission on minimal prednisone therapy) defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
    End point type
    Secondary
    End point timeframe
    Up to 60 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    86 [6]
    41 [7]
    Units: participants, n
    47
    22
    Notes
    [6] - Safety set - Participants with PV only
    [7] - Safety set - Participants with PV only
    No statistical analyses for this end point

    Secondary: Time to Disease Control (DC) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Time to Disease Control (DC) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    Disease Control (DC) defined as absence of new lesions and the start of healing of established lesions.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    26 [8]
    17 [9]
    Units: days
        median (confidence interval 95%)
    8.5 (8.0 to 15.0)
    15.0 (8.0 to 22.0)
    Notes
    [8] - Safety set – Participants with status DC at the roll-over visit were not included in the analysis
    [9] - Safety set – Participants with status DC at the roll-over visit were not included in the analysis
    No statistical analyses for this end point

    Secondary: Time to Complete clinical remission (CR) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Time to Complete clinical remission (CR) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    Measure Description CR (Complete clinical remission) defined as the absence of new lesions and complete healing of established lesions.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    61 [10]
    34 [11]
    Units: days
        median (confidence interval 95%)
    66.0 (43.0 to 182.0)
    71.0 (41.0 to 100.0)
    Notes
    [10] - Safety set – Participants with status CR at the roll-over visit were not included in this analysis
    [11] - Safety set – Participants with status CR at the roll-over visit were not included in this analysis
    No statistical analyses for this end point

    Secondary: Time to Complete remission on minimal prednisone therapy (CRmin) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Time to Complete remission on minimal prednisone therapy (CRmin) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    CRmin defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks. * The value '9999' is a dummy number to indicate the number was not calculable.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    83 [12]
    43 [13]
    Units: days
        median (confidence interval 95%)
    229.0 (161.0 to 9999)
    169.0 (141.0 to 322.0)
    Notes
    [12] - Safety set – Participants with status CRmin at the rollover visit were not included in this analysis
    [13] - Safety set – Participants with status CRmin at the rollover visit were not included in this analysis
    No statistical analyses for this end point

    Secondary: Time to Complete remission off therapy (CRoff) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Time to Complete remission off therapy (CRoff) in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    Complete remission off therapy (CRoff) is defined as the absence of new and established lesions completely healed while the patient is receiving no prednisone therapy for at least 8 weeks. * The value '9999' is a dummy number to indicate the number was not calculable.
    End point type
    Secondary
    End point timeframe
    up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    83 [14]
    43 [15]
    Units: days
        median (confidence interval 95%)
    9999 (9999 to 9999)
    9999 (9999 to 9999)
    Notes
    [14] - Safety set – Participants with status CRoff at the rollover visit were not included in this analysis
    [15] - Safety set – Participants with status CRoff at the rollover visit were not included in this analysis
    No statistical analyses for this end point

    Secondary: Time to flare after CRmin in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Time to flare after CRmin in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    CRmin defined as defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    42 [16]
    23 [17]
    Units: days
        median (confidence interval 95%)
    339.0 (223.0 to 9999)
    168.0 (64.0 to 9999)
    Notes
    [16] - Safety set - Only participants who achieved CRmin were considered for the analysis
    [17] - Safety set - Only participants who achieved CRmin were considered for the analysis
    No statistical analyses for this end point

    Secondary: Rate of treatment failure in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)

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    End point title
    		 Rate of treatment failure in participants with pemphigus vulgaris (PV) and pemphigus foliaceus (PF)
    End point description
    The absence of DC with oral prednisone 1.5 mg/kg/day for a minimum of 3 weeks, or absence of DC due to prednisone-related SAE, or flare before CRmin resulting in withdrawal of the participant.
    End point type
    Secondary
    End point timeframe
    Up to 52 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    101 [18]
    48 [19]
    Units: number
    3
    1
    Notes
    [18] - Safety set
    [19] - Safety set
    No statistical analyses for this end point

    Secondary: Number of Flares in participants with PV and PF

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    End point title
    		 Number of Flares in participants with PV and PF
    End point description
    A flare is defined as the appearance of 3 or more new lesions in a 4-week period that do not heal spontaneously within 1 week or the extension, of established lesions in a participant who had achieved DC.
    End point type
    Secondary
    End point timeframe
    Up to 60 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    122 [20]
    59 [21]
    Units: number
        arithmetic mean (standard deviation)
    0.8 ( 1.03 )
    0.7 ( 0.79 )
    Notes
    [20] - Roll-over set - Only participants who achieved DC were considered for the analysis
    [21] - Roll-over set - Only participants who achieved DC were considered for the analysis
    No statistical analyses for this end point

    Secondary: Normalized Cumulative Prednisone Dose in participants with PV and PF

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    End point title
    		 Normalized Cumulative Prednisone Dose in participants with PV and PF
    End point description
    Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study
    End point type
    Secondary
    End point timeframe
    up to 60 weeks
    End point values
    		 Efgartigimod-efgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Number of subjects analysed
    123 [22]
    60 [23]
    Units: mg/kg/day
        arithmetic mean (standard error)
    0.212 ( 0.2018 )
    0.241 ( 0.2401 )
    Notes
    [22] - For participants that do not achieve CRmin/CRoff, NCPD until CRmin/CRoff is not calculated
    [23] - For participants that do not achieve CRmin/CRoff, NCPD until CRmin/CRoff is not calculated
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Up to 60 weeks
    Adverse event reporting additional description
    Participants who rolled over from the antecedent study ARGX-113-1904, and received at least 1 dose of efgartigimod PH20 SC during this ARGX-113-1905 study (Safety set). Laboratory abnormalities were reported as AEs.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.0
    Reporting groups
    Reporting group title
    Efgartigimodefgartigimod PH20 SC
    Reporting group description
    		 Participants who received efgartigimod PH20 SC in antecedent study ARGX-113-1904.

    Reporting group title
    Placebo-efgartigimod PH20 SC
    Reporting group description
    		 Participants who received placebo PH20 SC in antecedent study ARGX-113-1904.

    Serious adverse events
    		 Efgartigimodefgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    16 / 101 (15.84%)
    4 / 48 (8.33%)
         number of deaths (all causes)
    0
    1
         number of deaths resulting from adverse events
    0
    1
    Investigations
    Blood immunoglobulin G decreased
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood pressure increased
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Patella fracture
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tibia fracture
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Wound
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Deep vein thrombosis
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypertension
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Shock haemorrhagic
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Disease progression
         subjects affected / exposed
    2 / 101 (1.98%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Anal fistula
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastritis erosive
         subjects affected / exposed
    2 / 101 (1.98%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Intestinal obstruction
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Reflux gastritis
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Respiratory failure
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Pemphigus
         subjects affected / exposed
    3 / 101 (2.97%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    1 / 3
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Musculoskeletal pain
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Osteonecrosis
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Anal abscess
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    2 / 101 (1.98%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Epiglottitis
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Lung abscess
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Septic shock
         subjects affected / exposed
    1 / 101 (0.99%)
    0 / 48 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin infection
         subjects affected / exposed
    0 / 101 (0.00%)
    1 / 48 (2.08%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Efgartigimodefgartigimod PH20 SC Placebo-efgartigimod PH20 SC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    46 / 101 (45.54%)
    19 / 48 (39.58%)
    Investigations
    Blood lactate dehydrogenase increased
         subjects affected / exposed
    7 / 101 (6.93%)
    1 / 48 (2.08%)
         occurrences all number
    8
    1
    Blood uric acid increased
         subjects affected / exposed
    5 / 101 (4.95%)
    0 / 48 (0.00%)
         occurrences all number
    5
    0
    Glycosylated haemoglobin increased
         subjects affected / exposed
    6 / 101 (5.94%)
    0 / 48 (0.00%)
         occurrences all number
    6
    0
    Low density lipoprotein increased
         subjects affected / exposed
    5 / 101 (4.95%)
    0 / 48 (0.00%)
         occurrences all number
    8
    0
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 101 (0.99%)
    3 / 48 (6.25%)
         occurrences all number
    1
    3
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 101 (2.97%)
    4 / 48 (8.33%)
         occurrences all number
    11
    4
    General disorders and administration site conditions
    Injection site erythema
         subjects affected / exposed
    4 / 101 (3.96%)
    3 / 48 (6.25%)
         occurrences all number
    30
    5
    Blood and lymphatic system disorders
    Increased tendency to bruise
         subjects affected / exposed
    0 / 101 (0.00%)
    3 / 48 (6.25%)
         occurrences all number
    0
    3
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    2 / 101 (1.98%)
    3 / 48 (6.25%)
         occurrences all number
    2
    5
    Renal and urinary disorders
    Haematuria
         subjects affected / exposed
    2 / 101 (1.98%)
    3 / 48 (6.25%)
         occurrences all number
    2
    4
    Musculoskeletal and connective tissue disorders
    Myopathy
         subjects affected / exposed
    2 / 101 (1.98%)
    3 / 48 (6.25%)
         occurrences all number
    2
    3
    Infections and infestations
    COVID-19
         subjects affected / exposed
    12 / 101 (11.88%)
    5 / 48 (10.42%)
         occurrences all number
    12
    5
    Folliculitis
         subjects affected / exposed
    5 / 101 (4.95%)
    1 / 48 (2.08%)
         occurrences all number
    6
    1
    Nasopharyngitis
         subjects affected / exposed
    5 / 101 (4.95%)
    1 / 48 (2.08%)
         occurrences all number
    7
    4
    Oral candidiasis
         subjects affected / exposed
    6 / 101 (5.94%)
    0 / 48 (0.00%)
         occurrences all number
    8
    0
    Urinary tract infection
         subjects affected / exposed
    5 / 101 (4.95%)
    2 / 48 (4.17%)
         occurrences all number
    5
    2
    Metabolism and nutrition disorders
    Hypercholesterolaemia
         subjects affected / exposed
    5 / 101 (4.95%)
    0 / 48 (0.00%)
         occurrences all number
    5
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Feb 2021
    Protocol, Version 2.0 • A secondary objective and endpoints were added to explore the feasibility of efgartigimod PH20 SC self-administration or caregiver-supported administration. Instructions were added and the schedule of activities updated to include self-administration or caregiver-supported administration and training. • A secondary objective and endpoint were added to measure the health impact of glucocorticoid use in participants with pemphigus. • Evaluating antibodies against rHuPH20 was changed from a secondary objective and endpoint to an exploratory objective and endpoint due to safety concerns. • Lymphocyte dynamic changes was added as an endpoint to the exploratory objective to evaluate the disease-specific genetic background and effects of efgartigimod PH20 SC on the serological and immunological profiles. • Assessments of vaccine-induced immunity in the context of efgartigimod PH20 SC administration were added. • Changes to the contraceptive requirements based on new data about efgartigimod PH20 SC were implemented. • A transition in efgartigimod concentration from 165 mg/mL to 180 mg/mL was implemented to reduce the volume for each 1000-mg SC injection. • A new criterion was added allowing the withdrawal from the study of a participant for whom a severe AE, SAE, or clinically significant change in a laboratory test parameter was reported.
    05 Sep 2022
    Protocol, Version 3.0 • The upper limit of the number of participants who could enter the study (originally up to 150) was removed after the sample size for ARGX-113-1904 was increased. • The timing of samples collected for lymphocyte populations was updated to allow for long-term immunological profiling. • Based on nonclinical teratogenicity and reproductive toxicity data, the inclusion and exclusion criteria were updated: (1) Female participants could stop their contraception method after the last IMP dose, (2) female participants and the female partners of male participants could become pregnant immediately after the study, and (3) male participants could donate sperm. • Information on injection-site reactions and instructions on monitoring and reporting injection-site reactions were added.

    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.
    Study was terminated prematurely by the Sponsor.
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    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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