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    Clinical Trial Results:
    An Open-label, Non-controlled, Phase II Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, Efficacy and Conditions of Use of ARGX-113 in Patients with Mild to Moderate Pemphigus (Vulgaris or Foliaceus)

    Summary
    EudraCT number
    		 2017-002333-40
    Trial protocol
    		 DE   HU   IT   RO  
    Global end of trial date
    28 Oct 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Nov 2021
    First version publication date
    13 Nov 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ARGX-113-1701
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03334058
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    argenx BV
    Sponsor organisation address
    Industriepark Zwijnaarde 7, Zwijnaarde, Belgium, 9052
    Public contact
    Regulatory Manager, argenx BV, regulatory@argenx.com
    Scientific contact
    Regulatory Manager, argenx BV, 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
    28 Oct 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    28 Oct 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of efgartigimod in Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) subjects.
    Protection of trial subjects
    This study was performed according to the International Conference on Harmonisation of Good Clinical Practice, the principles of the Declaration of Helsinki, and other applicable local ethical and legal requirements.
    Background therapy
    		 Relapsing subjects under tapered prednisone therapy were kept in the study at the same dosage during the Induction treatment period. Then prednisone dosage could be tapered from the beginning of the Maintenance treatment period (Visit 5) up to study end (Follow-up Visit 3), according to standard of care (SoC). Newly diagnosed subjects or relapsing subjects off therapy, who were already on a first course of oral prednisone and for whom efgartigimod monotherapy was considered not clinically acceptable, were kept at the same dosage during the Induction treatment period. From the beginning of the Maintenance treatment period (Visit 5) up to study end (Follow-up Visit 3), prednisone dosage could be tapered according to SoC. Newly diagnosed subjects naïve to any treatment and relapsing subjects off therapy, or with a first course of oral prednisone (≤ 4 weeks), for whom an initial period of efgartigimod monotherapy was judged clinically acceptable, were not administered any SoC (e.g. oral prednisone). Any adjuvant conventional immunosuppressant (e.g. mycophenolate mofetil, azathioprine) was discontinued at any time during the screening period. SoC in additional Cohort 4: • All subjects off therapy were associated with oral prednisone 20 mg/day at baseline. In relapsing subjects who were under oral prednisone therapy at tapered dose, oral prednisone was maintained at the same dosage, while any adjuvant conventional immunosuppressant was discontinued at any time during the screening period. • Oral prednisone dose could be tapered as of end of consolidation (EoC) (the time at which no new lesions have developed for a minimum of 2 weeks, and approximately 80% of lesions have healed). For all subjects with a clinically active disease (e.g. worsening of the clinical signs) upon Investigator’s judgment, a rescue treatment of oral prednisone could be implemented at any post-baseline visit.
    Evidence for comparator
    		 -
    Actual start date of recruitment
    02 Nov 2017
    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
    Israel: 4
    Country: Number of subjects enrolled
    Ukraine: 5
    Country: Number of subjects enrolled
    Germany: 6
    Country: Number of subjects enrolled
    Hungary: 11
    Country: Number of subjects enrolled
    Italy: 8
    Worldwide total number of subjects
    34
    EEA total number of subjects
    25
    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)
    27
    From 65 to 84 years
    6
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 This Phase 2 study was conducted in subjects with mild to moderate PV or PF at 17 centers worldwide. Of the 53 subjects who were screened, 19 subjects were screen failures and 34 subjects were enrolled.

    Pre-assignment
    Screening details
    		 The study comprised a screening period of up to 3 weeks, treatment periods ranging from 9 to 34 weeks, and a treatment-free follow-up period of 8 (Cohort 1) or 10 weeks (Cohorts 2-4).

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Cohort 1 (10 mg/kg)
    Arm description
    		 Efgartigimod intravenous (IV) 10 milligrams per kilogram (mg/kg) was administered as 4 weekly infusions during the induction period and during the maintenance period at weeks 2 and 6.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod
    Investigational medicinal product code
    ARGX-113
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Efgartigimod (10 mg/kg), 250 milliliter (mL) was infused IV over a period of 2 hours. Subjects remained at the site for 2 hours minimum after the end of infusion during these visits as part of routine safety monitoring.

    Arm title
    		 Cohort 2 (10 mg/kg)
    Arm description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period once every 2 weeks (q2w) for 8 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod
    Investigational medicinal product code
    ARGX-113
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Efgartigimod (10 mg/kg), 250 mL was infused IV over a period of 2 hours. Subjects remained at the site for 2 hours minimum after the end of infusion during these visits as part of routine safety monitoring.

    Arm title
    		 Cohort 3 (10 mg/kg)
    Arm description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period q2w for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod
    Investigational medicinal product code
    ARGX-113
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Efgartigimod (10 mg/kg), 250 mL was infused IV over a period of 2 hours. Subjects remained at the site for 2 hours minimum after the end of infusion during these visits as part of routine safety monitoring.

    Arm title
    		 Cohort 4 (25 mg/kg)
    Arm description
    		 Efgartigimod IV 25 mg/kg was administered as 5 weekly infusions (minimum) during the induction period until EoC, and during the maintenance period q2w until Week 34.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Efgartigimod
    Investigational medicinal product code
    ARGX-113
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Efgartigimod (25 mg/kg), 250 mL was infused IV over a period of 2 hours. Subjects remained at the site for 2 hours minimum after the end of infusion during these visits as part of routine safety monitoring.

    Number of subjects in period 1
    		Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Started
    6
    5
    8
    15
    Completed
    3
    2
    7
    10
    Not completed
    3
    3
    1
    5
         Other
    1
    -
    -
    -
         Investigator termination
    2
    3
    -
    2
         Withdrawal of informed consent
    -
    -
    1
    2
         Lost to follow-up
    -
    -
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Cohort 1 (10 mg/kg)
    Reporting group description
    		 Efgartigimod intravenous (IV) 10 milligrams per kilogram (mg/kg) was administered as 4 weekly infusions during the induction period and during the maintenance period at weeks 2 and 6.

    Reporting group title
    Cohort 2 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period once every 2 weeks (q2w) for 8 weeks.

    Reporting group title
    Cohort 3 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period q2w for 12 weeks.

    Reporting group title
    Cohort 4 (25 mg/kg)
    Reporting group description
    		 Efgartigimod IV 25 mg/kg was administered as 5 weekly infusions (minimum) during the induction period until EoC, and during the maintenance period q2w until Week 34.

    Reporting group values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg) Total
    Number of subjects
    		 6 5 8 15 34
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (full range (min-max))
    		 42.0 (29 to 63) 64.6 (43 to 78) 46.9 (30 to 65) 53.3 (22 to 85) -
    Gender categorical
    Units: Subjects
        Female
    		 3 4 7 8 22
        Male
    		 3 1 1 7 12
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 0 0 0 0
        Not Hispanic or Latino
    		 6 5 8 15 34
    Race
    Units: Subjects
        White
    		 6 5 8 14 33
        Black or African American
    		 0 0 0 0 0
        Asian
    		 0 0 0 1 1
        American Indian or Alaska
    		 0 0 0 0 0
        Native
    		 0 0 0 0 0
        Native Hawaiian or Other
    		 0 0 0 0 0
        Pacific Islander
    		 0 0 0 0 0
        Multiple Race
    		 0 0 0 0 0
    Pemphigus Type
    Units: Subjects
        PV: Mucosal-dominant
    		 1 3 3 2 9
        PV: Mucocutaneous
    		 4 2 4 4 14
        PV: Cutaneous
    		 1 0 0 2 3
        PF
    		 0 0 1 7 8

    End points

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    End points reporting groups
    Reporting group title
    Cohort 1 (10 mg/kg)
    Reporting group description
    		 Efgartigimod intravenous (IV) 10 milligrams per kilogram (mg/kg) was administered as 4 weekly infusions during the induction period and during the maintenance period at weeks 2 and 6.

    Reporting group title
    Cohort 2 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period once every 2 weeks (q2w) for 8 weeks.

    Reporting group title
    Cohort 3 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period q2w for 12 weeks.

    Reporting group title
    Cohort 4 (25 mg/kg)
    Reporting group description
    		 Efgartigimod IV 25 mg/kg was administered as 5 weekly infusions (minimum) during the induction period until EoC, and during the maintenance period q2w until Week 34.

    Subject analysis set title
    Cohorts 1-3
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period in Cohorts 1, 2, and 3; and during the maintenance period at weeks 2 and 6 (Cohort 1), q2w for 8 weeks (Cohort 2), and q2w for 12 weeks (Cohort 3).

    Subject analysis set title
    Cohorts 1-4
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period in Cohorts 1, 2, and 3; and during the maintenance period at weeks 2 and 6 (Cohort 1), q2w for 8 weeks (Cohort 2), and q2w for 12 weeks (Cohort 3). Efgartigimod IV 25 mg/kg was administered as 5 weekly infusions (minimum) during the induction period until EoC in Cohort 4; and during the maintenance period q2w until Week 34.

    Primary: Number of Subjects who Experienced Treatment-emergent Adverse Events (TEAEs)

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    End point title
    		 Number of Subjects who Experienced Treatment-emergent Adverse Events (TEAEs) [1]
    End point description
    A TEAE was an undesirable event not present prior to medical treatment, or an already present event that worsened either in intensity or frequency following the treatment. A serious adverse event (SAE), experience or reaction, was any untoward medical occurrence (whether considered to be related to investigational medicinal product [IMP] or not) that at any dose: • Resulted in death; • Was life-threatening; • Required in subject hospitalization or prolongation of existing hospitalization; • Resulted in persistent or significant disability or incapacity; • Was a congenital abnormality or birth defect; • Medically significant events, which did not meet any of the criteria above, but may have jeopardized the subject and may have required medical or surgical intervention to prevent one of the other serious outcomes listed in the definition above. The Safety analysis set (SAS) included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    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 additional statistical analysis was prespecified for this end point.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Number of subjects analysed
    6
    5
    8
    15
    Units: subjects
        TEAE
    6
    4
    6
    13
        SAE
    1
    1
    0
    0
    No statistical analyses for this end point

    Primary: Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Vital Sign Measurements

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    End point title
    		 Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Vital Sign Measurements [2]
    End point description
    At each visit, vital signs (supine blood pressure, pulse rate, and oral body temperature) were assessed. Supine blood pressure and pulse rate were measured using standard equipment after approximately 10 minutes in a supine position. The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No additional statistical analysis was prespecified for this end point.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Number of subjects analysed
    6
    5
    8
    15
    Units: subjects
    1
    0
    0
    2
    No statistical analyses for this end point

    Primary: Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Electrocardiograms (ECG)

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    End point title
    		 Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Electrocardiograms (ECG) [3]
    End point description
    ECG was taken at a paper speed of 25 millimeter/second and should be obtained with the subject in the supine position after they have rested in this position for at approximately 10 minutes. Three consecutive ECG recordings was taken with an interval of approximately 5 minutes at each occasion to obtain reliable and interpretable data. The ECG parameters that were collected are heart rate, PR, QRS, QT whereas QTcF was calculated. The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    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 additional statistical analysis was prespecified for this end point.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Number of subjects analysed
    6
    5
    8
    15
    Units: subjects
    0
    0
    0
    0
    No statistical analyses for this end point

    Primary: Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Physical Examinations (Cohorts 1-3)

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    End point title
    		 Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Physical Examinations (Cohorts 1-3) [4] [5]
    End point description
    At each visit, physical examinations were carried out. Only physical examinations and visits where a clinically significant change from baseline occurred are included below. A clinically significant change from baseline was defined as a change from normal or abnormal (not clinically significant) to abnormal (clinically significant) post-baseline. The complete physical examination included an assessment of general appearance, skin, head and neck (including ears, eyes, nose and throat), lymph nodes, thyroid, musculoskeletal/extremities, abdomen, breast, and cardiovascular, respiratory, neurological, and genital/rectal systems. The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    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: No additional statistical analysis was prespecified for this end point.
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    6
    5
    8
    Units: subjects
        Head and Neck: Day 15
    1
    0
    0
        Respiratory: Day 15
    1
    0
    0
        Musculoskeletal/Extremities: Day 22
    0
    1
    0
    No statistical analyses for this end point

    Primary: Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Physical Examinations (Cohort 4)

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    End point title
    		 Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Physical Examinations (Cohort 4) [6] [7]
    End point description
    At each visit, physical examinations were carried out. Only physical examinations and visits where a clinically significant change from baseline occurred are included below. A clinically significant change from baseline was defined as a change from normal or abnormal (not clinically significant) to abnormal (clinically significant) post-baseline. The complete physical examination included an assessment of general appearance, skin, head and neck (including ears, eyes, nose and throat), lymph nodes, thyroid, musculoskeletal/extremities, abdomen, breast, and cardiovascular, respiratory, neurological, and genital/rectal systems. The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [6] - 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 additional statistical analysis was prespecified for this end point.
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: subjects
        Abdomen: Day 99
    1
        General Appearance: Day 211
    1
        General Appearance: Day 239
    1
        General Appearance: Day 260
    1
        General Appearance: Day 281
    1
        General Appearance: Day 309
    1
        Genital/Rectal: Day 85
    1
        Genital/Rectal: Day 99
    1
        Genital/Rectal: Day 190
    1
        Genital/Rectal: Day 204
    1
        Genital/Rectal: Day 218
    1
        Genital/Rectal: Day 232
    1
        Genital/Rectal: Day 246
    1
        Genital/Rectal: Day 274
    1
        Genital/Rectal: Day 288
    1
        Genital/Rectal: Day 323
    1
        Head and Neck: Day 78
    1
        Head and Neck: Day 120
    1
        Head and Neck: Day 134
    1
        Head and Neck: Day 148
    1
        Head and Neck: Day 162
    1
        Head and Neck: Day 176
    1
        Head and Neck: Day 190
    1
        Head and Neck: Day 204
    1
        Head and Neck: Day 218
    1
        Head and Neck: Day 232
    1
        Head and Neck: Day 253
    1
        Head and Neck: Day 274
    1
        Head and Neck: Day 302
    1
        Neurological: Day 127
    1
        Neurological: Day 141
    1
        Neurological: Day 155
    1
        Neurological: Day 169
    1
        Neurological: Day 183
    1
        Neurological: Day 197
    1
        Neurological: Day 211
    1
        Neurological: Day 225
    1
        Neurological: Day 239
    1
        Neurological: Day 253
    1
        Neurological: Day 288
    1
        Neurological: Day 309
    1
        Respiratory: Day 50
    1
        Respiratory: Day 85
    1
        Respiratory: Day 127
    1
        Respiratory: Day 141
    1
        Respiratory: Day 155
    1
        Respiratory: Day 169
    1
        Respiratory: Day 183
    1
        Respiratory: Day 197
    1
        Respiratory: Day 211
    1
        Respiratory: Day 225
    1
        Respiratory: Day 239
    1
        Respiratory: Day 253
    1
        Respiratory: Day 288
    1
        Respiratory: Day 309
    1
        Skin: Day 211
    1
        Skin: Day 239
    1
        Skin: Day 260
    1
        Skin: Day 281
    1
        Skin: Day 309
    1
    No statistical analyses for this end point

    Primary: Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Clinical Laboratory Evaluations

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    End point title
    		 Number of Subjects Who Experienced a Clinically Significant Change from Baseline in Clinical Laboratory Evaluations [8]
    End point description
    At each visit, routine blood samples were taken for determination of: • Hematology: hemoglobin, hematocrit, mean corpuscular volume, red blood cell count, platelet count, white blood cell count with differential; • Blood chemistry: sodium, potassium, calcium, glucose, creatinine, creatinine clearance, blood urea nitrogen, alanine aminotransferase, aspartate aminotransferase, total bilirubin, gamma-glutamyl transferase, C-reactive protein, alkaline phosphatase, lactate dehydrogenase, uric acid, total protein and albumin. Urinalysis was performed by dipstick method and included: color, clarity/appearance, specific gravity, pH, protein, glucose, ketones, blood, bilirubin, urobilinogen, nitrite, leukocyte esterase, and microscopic examination including red blood cells, white blood cells, cast crystals, and bacteria. Subjects need to be fasting for at least 8 hours prior to blood glucose assessment. The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    End point type
    Primary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [8] - 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 additional statistical analysis was prespecified for this end point.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Number of subjects analysed
    6
    5
    8
    15
    Units: subjects
    0
    0
    3
    3
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Total Immunoglobulin G (IgG) (Cohorts 1-3)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Total Immunoglobulin G (IgG) (Cohorts 1-3) [9]
    End point description
    Levels of total IgG were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1), end of Induction Period (Day 29), end of Maintenance Period (Cohort 1: Day 64; Cohort 2: Day 78; Cohort 3: Day 106), and end of Treatment-free Follow-up (Cohort 1: Day 120; Cohort 2: Day 148; Cohort 3: Day 176)
    Notes
    [9] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    3 [10]
    3 [11]
    7 [12]
    Units: percentage change (%)
    arithmetic mean (standard error)
        End of Induction Period (n = 2, 3, 7)
    -55.00 ( 1.400 )
    -67.90 ( 3.005 )
    -63.44 ( 3.192 )
        End of Maintenance Period (n = 3, 3, 7)
    -4.37 ( 4.834 )
    -50.67 ( 3.176 )
    -49.21 ( 3.782 )
        End of Treatment-free Follow-up (n = 3, 2, 7)
    23.30 ( 13.141 )
    -9.65 ( 21.550 )
    -8.54 ( 6.762 )
    Notes
    [10] - 'n' in category title denotes number of subjects analyzed for that category.
    [11] - 'n' in category title denotes number of subjects analyzed for that category.
    [12] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Total IgG (Cohort 4)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Total IgG (Cohort 4) [13]
    End point description
    Levels of total IgG were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of Induction Period (Day 29)
    Notes
    [13] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: percentage change (%)
        arithmetic mean (standard error)
    -63.53 ( 3.285 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of IgG Subtypes (IgG1, IgG2, IgG3, IgG4) (Cohorts 1-3)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of IgG Subtypes (IgG1, IgG2, IgG3, IgG4) (Cohorts 1-3) [14]
    End point description
    Levels of IgG subtypes (IgG1, IgG2, IgG3, IgG4) were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1), end of Induction Period (Day 29), and the end of Maintenance Period (Cohort 1: Day 64; Cohort 2: Day 78; Cohort 3: Day 106), and end of Treatment-free Follow-up (Cohort 1: Day 120; Cohort 2: Day 148; Cohort 3: Day 176)
    Notes
    [14] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    3
    3
    7
    Units: percentage change (%)
    arithmetic mean (standard error)
        IgG1: End of Induction Period
    -45.83 ( 13.715 )
    -69.47 ( 1.785 )
    -69.14 ( 3.051 )
        IgG1: End of Maintenance Period
    -7.83 ( 5.210 )
    -48.63 ( 1.281 )
    -55.67 ( 3.907 )
        IgG2: End of Induction Period
    -45.00 ( 12.501 )
    -63.70 ( 5.524 )
    -57.11 ( 2.474 )
        IgG2: End of Maintenance Period
    -25.63 ( 6.542 )
    -50.17 ( 1.185 )
    -49.33 ( 3.729 )
        IgG3: End of Induction Period
    -43.07 ( 16.689 )
    -70.30 ( 1.320 )
    -65.43 ( 4.153 )
        IgG3: End of Maintenance Period
    -4.37 ( 1.354 )
    -45.67 ( 1.955 )
    -45.36 ( 5.201 )
        IgG4: End of Induction Period
    -40.43 ( 8.842 )
    -61.07 ( 5.374 )
    -56.80 ( 5.382 )
        IgG4: End of Maintenance Period
    7.17 ( 12.856 )
    1.97 ( 58.640 )
    -46.94 ( 5.862 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of IgG Subtypes (IgG1, IgG2, IgG3, IgG4) (Cohort 4)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of IgG Subtypes (IgG1, IgG2, IgG3, IgG4) (Cohort 4) [15]
    End point description
    Levels of IgG subtypes (IgG1, IgG2, IgG3, IgG4) were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of Induction Period (Day 29)
    Notes
    [15] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: percentage change (%)
    arithmetic mean (standard error)
        IgG1: End of Induction Period
    -66.95 ( 3.067 )
        IgG2: End of Induction Period
    -62.54 ( 2.722 )
        IgG3: End of Induction Period
    -69.15 ( 2.753 )
        IgG4: End of Induction Period
    -55.85 ( 4.986 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-1 Autoantibodies (Cohorts 1-3)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-1 Autoantibodies (Cohorts 1-3) [16]
    End point description
    Levels of anti-desmoglein-1 were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC. '99999' denotes standard error could not be calculated as only one subject was analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1), end of Induction Period (Day 29), end of Maintenance Period (Cohort 1: Day 64; Cohort 2: Day 78; Cohort 3: Day 106), and end of Treatment-free Follow-up (Cohort 1: Day 120; Cohort 2: Day 148; Cohort 3: Day 176)
    Notes
    [16] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    1 [17]
    2 [18]
    4 [19]
    Units: percentage change (%)
    arithmetic mean (standard error)
        End of Induction Period (n = 1, 2, 4)
    -64.70 ( 99999 )
    11.85 ( 82.050 )
    -50.08 ( 22.920 )
        End of Maintenance Period (n = 1, 1, 4)
    -14.10 ( 99999 )
    -88.80 ( 99999 )
    -50.23 ( 19.924 )
        End of Treatment-free Follow-up (n = 1, 1, 4)
    25.60 ( 99999 )
    -88.20 ( 99999 )
    -30.13 ( 51.025 )
    Notes
    [17] - 'n' in category title denotes number of subjects analyzed for that category.
    [18] - 'n' in category title denotes number of subjects analyzed for that category.
    [19] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-1 Autoantibodies (Cohort 4)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-1 Autoantibodies (Cohort 4) [20]
    End point description
    Levels of anti-desmoglein-1 were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of Induction Period (Day 29)
    Notes
    [20] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    13
    Units: percentage change (%)
        arithmetic mean (standard error)
    -57.81 ( 7.701 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-3 Autoantibodies (Cohorts 1-3)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-3 Autoantibodies (Cohorts 1-3) [21]
    End point description
    Levels of anti-desmoglein-3 were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1), end of Induction Period (Day 29), and the end of Maintenance Period (Cohort 1: Day 64; Cohort 2: Day 78; Cohort 3: Day 106), and end of Treatment-free Follow-up (Cohort 1: Day 120; Cohort 2: Day 148; Cohort 3: Day 176)
    Notes
    [21] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    3 [22]
    3 [23]
    6 [24]
    Units: percentage change (%)
    arithmetic mean (standard error)
        End of Induction Period (n = 3, 3, 6)
    -20.77 ( 4.100 )
    -56.90 ( 12.468 )
    -52.63 ( 12.314 )
        End of Maintenance Period (n = 3, 3, 6)
    72.27 ( 79.268 )
    -48.37 ( 19.804 )
    -40.62 ( 17.492 )
        End of Treatment-free Follow-up (n = 3, 2, 6)
    213.03 ( 155.219 )
    -59.45 ( 25.050 )
    -46.52 ( 4.873 )
    Notes
    [22] - 'n' in category title denotes number of subjects analyzed for that category.
    [23] - 'n' in category title denotes number of subjects analyzed for that category.
    [24] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-3 Autoantibodies (Cohort 4)

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    End point title
    		 Mean Percentage Change from Baseline in Serum Levels of Anti-Desmoglein-3 Autoantibodies (Cohort 4) [25]
    End point description
    Levels of anti-desmoglein-3 were measured from the blood sampled at Baseline and each visit using a validated assay. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of Induction Period (Day 29)
    Notes
    [25] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    10
    Units: percentage change (%)
        arithmetic mean (standard error)
    -35.32 ( 11.279 )
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in Pemphigus Disease Area Index (PDAI) (Cohorts 1-3)

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    End point title
    		 Mean Percentage Change from Baseline in Pemphigus Disease Area Index (PDAI) (Cohorts 1-3) [26]
    End point description
    PDAI is a cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions. The score was weighted for the number and size of lesions with score of 0 (absent) to 10 (> 3 lesions, and/or at least one lesion > 16 cm diameter or entire area) given for skin at 12 anatomic locations, scalp, and mucous membrane showing disease activity (erosions/blisters or new erythema). Damage from post inflammatory hyperpigmentation or erythema from the resolving lesions was scored separately from the main score as absent (0) or present (1) for each body area or scalp resulting in a score of 0 to 12 and 0 to 1. The PDAI total activity score ranged from 0 to 263, with 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity) and 13 points representing disease damage. Higher total activity scores indicate more severe pemphigus symptoms. The Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1), end of Induction Period (Day 29), and the end of Maintenance Period (Cohort 1: Day 64; Cohort 2: Day 78; Cohort 3: Day 106), and end of Treatment-free Follow-up (Cohort 1: Day 120; Cohort 2: Day 148; Cohort 3: Day 176)
    Notes
    [26] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohort 4 is presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg)
    Number of subjects analysed
    3 [27]
    3 [28]
    7 [29]
    Units: percentage change (%)
    arithmetic mean (standard error)
        End of Induction Period (n = 3, 3, 7)
    -53.77 ( 10.791 )
    88.20 ( 161.816 )
    -58.26 ( 24.617 )
        End of Maintenance Period (n = 3, 3, 7)
    -23.83 ( 17.629 )
    -56.20 ( 24.906 )
    -15.49 ( 69.638 )
        End of Treatment-free Follow-up (n = 3, 2, 7)
    9.90 ( 46.279 )
    -76.30 ( 6.300 )
    -37.86 ( 45.237 )
    Notes
    [27] - 'n' in category title denotes number of subjects analyzed for that category.
    [28] - 'n' in category title denotes number of subjects analyzed for that category.
    [29] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Mean Percentage Change from Baseline in PDAI (Cohort 4)

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    End point title
    		 Mean Percentage Change from Baseline in PDAI (Cohort 4) [30]
    End point description
    PDAI is a cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions. The score was weighted for the number and size of lesions with score of 0 (absent) to 10 (> 3 lesions, and/or at least one lesion > 16 cm diameter or entire area) given for skin at 12 anatomic locations, scalp, and mucous membrane showing disease activity (erosions/blisters or new erythema). Damage from post inflammatory hyperpigmentation or erythema from the resolving lesions was scored separately from the main score as absent (0) or present (1) for each body area or scalp resulting in a score of 0 to 12 and 0 to 1. The PDAI total activity score ranged from 0 to 263, with 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity) and 13 points representing disease damage. Higher total activity scores indicate more severe pemphigus symptoms. The Efficacy Analysis Set.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and End of Induction Period (Day 29)
    Notes
    [30] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analyses for Cohorts 1-3 are presented in a separate end point due to the different timeframe.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: percentage change (%)
        arithmetic mean (standard error)
    -55.16 ( 5.474 )
    No statistical analyses for this end point

    Secondary: Median Time to Disease Control (DC)

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    End point title
    		 Median Time to Disease Control (DC) [31]
    End point description
    Time to DC, defined as when new lesions cease to form and established lesions begin to heal, evaluated from visit 2 until control was achieved. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [31] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohorts 1-3 is pooled using a subject analysis set.
    End point values
    		 Cohort 4 (25 mg/kg) Cohorts 1-3
    Number of subjects analysed
    15
    16
    Units: days
        median (full range (min-max))
    29 (6 to 64)
    15 (8 to 92)
    No statistical analyses for this end point

    Secondary: Median Time to EoC (Cohort 4)

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    End point title
    		 Median Time to EoC (Cohort 4) [32]
    End point description
    Time to EoC was defined as the time at which no new lesions have developed for a minimum of 2 weeks, and approximately 80% of lesions have healed. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [32] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis was prespecified for Cohort 4 only.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: days
        median (full range (min-max))
    43 (34 to 99)
    No statistical analyses for this end point

    Secondary: Median Time Until Relapse

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    End point title
    		 Median Time Until Relapse [33]
    End point description
    Time until relapse, relapse being defined as the appearance of 3 or more new lesions a month that do not heal spontaneously within 1 week, or as the extension of established lesions, evaluated from any visit after DC. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [33] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis for Cohorts 1-3 is pooled using a subject analysis set.
    End point values
    		 Cohort 4 (25 mg/kg) Cohorts 1-3
    Number of subjects analysed
    14 [34]
    14
    Units: days
        median (full range (min-max))
    82 (63 to 211)
    141 (10 to 169)
    Notes
    [34] - Median value represents the lower 95% confidence interval as the median could not be calculated.
    No statistical analyses for this end point

    Secondary: Median Time to Complete Clinical Remission (CR) (Cohort 4)

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    End point title
    		 Median Time to Complete Clinical Remission (CR) (Cohort 4) [35]
    End point description
    Time to calculated and Investigator-assessed CR, with CR being defined as the absence of new lesions and complete healing of established lesions for Cohort 4. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [35] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis was prespecified for Cohort 4 only.
    End point values
    		 Cohort 4 (25 mg/kg)
    Number of subjects analysed
    15
    Units: days
    median (full range (min-max))
        Calculated CR
    72 (41 to 287)
        Investigator-assessed CR
    92 (41 to 287)
    No statistical analyses for this end point

    Secondary: Median Time to Complete CR Under Minimal Therapy (Cohort 4)

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    End point title
    		 Median Time to Complete CR Under Minimal Therapy (Cohort 4) [36]
    End point description
    CR under minimal therapy was defined as a prednisone dose of 10 mg/day or less for at least 8 weeks. The Efficacy Analysis Set included the population evaluable for efficacy assessment. Subjects evaluable for efficacy were determined by the IDMC.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Notes
    [36] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Statistical analysis was prespecified for Cohort 4 only.
    End point values
    		 Cohort 4 (25 mg/kg) Cohorts 1-3
    Number of subjects analysed
    0 [37]
    0 [38]
    Units: days
        median (full range (min-max))
    ( to )
    ( to )
    Notes
    [37] - No data was collected for this end point.
    [38] - No data was collected for this end point.
    No statistical analyses for this end point

    Secondary: Mean Maximum Serum Concentration (Cmax) of Efgartigimod (Cohorts 1-3)

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    End point title
    		 Mean Maximum Serum Concentration (Cmax) of Efgartigimod (Cohorts 1-3)
    End point description
    In order to assess the pharmacokinetic (PK) parameters of efgartigimod, blood samples were collected from each subject at each visit from Baseline. Concentrations of serum efgartigimod were determined using a validated assay. The PK Analysis Set included all subjects in the SAS who have at least one evaluable time-point of efgartigimod PK concentration data. No PK parameters were derived from Cohort 4.
    End point type
    Secondary
    End point timeframe
    Within 30 minutes prior to start of infusion for the predose sample and within 30 minutes after end of infusion for the postdose sample at Baseline (Day 1) and Days 8, 15, 22, 36, 50, 64, 78, 92, and 106
    End point values
    		 Cohorts 1-3
    Number of subjects analysed
    19 [39]
    Units: micrograms (µg)/mL
    arithmetic mean (standard deviation)
        Day 1 (n = 19)
    168.748 ( 74.4491 )
        Day 8 (n = 19)
    171.079 ( 51.0213 )
        Day 15 (n = 17)
    147.829 ( 55.9076 )
        Day 22 (n = 15)
    209.147 ( 212.4485 )
        Day 36 (n = 11)
    173.209 ( 49.3980 )
        Day 50 (n = 10)
    164.590 ( 59.4498 )
        Day 64 (n = 12)
    229.333 ( 251.7157 )
        Day 78 (n = 10)
    227.100 ( 189.2784 )
        Day 92 (n = 7)
    195.286 ( 73.6698 )
        Day 106 (n = 7)
    168.071 ( 80.1428 )
    Notes
    [39] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Median Time to Cmax (Tmax) of Efgartigimod (Cohorts 1-3)

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    End point title
    		 Median Time to Cmax (Tmax) of Efgartigimod (Cohorts 1-3)
    End point description
    In order to assess the PK parameters of efgartigimod, blood samples were collected from each subject at each visit from Baseline. Concentrations of serum efgartigimod were determined using a validated assay. The PK Analysis Set included all subjects in the SAS who have at least one evaluable time-point of efgartigimod PK concentration data. No PK parameters were derived from Cohort 4.
    End point type
    Secondary
    End point timeframe
    Within 30 minutes prior to start of infusion for the predose sample and within 30 minutes after end of infusion for the postdose sample at Baseline (Day 1) and Days 8, 15, 22, 36, 50, 64, 78, 92, and 106
    End point values
    		 Cohorts 1-3
    Number of subjects analysed
    19 [40]
    Units: hours (h)
    median (full range (min-max))
        Day 1 (n = 19)
    2.500 (2.50 to 2.50)
        Day 8 (n = 19)
    2.317 (2.03 to 2.55)
        Day 15 (n = 17)
    2.250 (2.03 to 2.50)
        Day 22 (n = 15)
    2.300 (2.13 to 2.50)
        Day 36 (n = 11)
    2.250 (2.05 to 2.50)
        Day 50 (n = 10)
    2.267 (2.08 to 2.50)
        Day 64 (n = 12)
    2.450 (2.08 to 3.33)
        Day 78 (n = 10)
    2.425 (2.08 to 2.50)
        Day 92 (n = 7)
    2.333 (2.08 to 2.50)
        Day 106 (n = 7)
    2.283 (2.08 to 2.50)
    Notes
    [40] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Mean Concentration of Efgartigimod at the End of the Dosing Interval (Ctrough) (Cohorts 1-3)

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    End point title
    		 Mean Concentration of Efgartigimod at the End of the Dosing Interval (Ctrough) (Cohorts 1-3)
    End point description
    In order to assess the PK parameters of efgartigimod, blood samples were collected from each subject at each visit from Baseline. Concentrations of serum efgartigimod were determined using a validated assay. The PK Analysis Set included all subjects in the SAS who have at least one evaluable time-point of efgartigimod PK concentration data. No PK parameters were derived from Cohort 4.
    End point type
    Secondary
    End point timeframe
    Within 30 minutes prior to start of infusion for the predose sample and within 30 minutes after end of infusion for the postdose sample on Days 8, 15, 22, 36, 50, 64, 78, 92, and 106
    End point values
    		 Cohorts 1-3
    Number of subjects analysed
    19 [41]
    Units: µg/mL
    arithmetic mean (standard deviation)
        Day 8 (n = 19)
    9.395 ( 5.2388 )
        Day 15 (n = 18)
    10.742 ( 5.9878 )
        Day 22 (n = 15)
    11.164 ( 7.5341 )
        Day 29 (n = 14)
    12.993 ( 6.5653 )
        Day 36 (n = 12)
    3.519 ( 2.3757 )
        Day 50 (n = 10)
    3.044 ( 2.1179 )
        Day 64 (n = 12)
    1.883 ( 1.8393 )
        Day 78 (n = 10)
    3.257 ( 1.9766 )
        Day 92 (n = 7)
    2.581 ( 1.2742 )
        Day 106 (n = 7)
    2.469 ( 1.5568 )
    Notes
    [41] - 'n' in category title denotes number of subjects analyzed for that category.
    No statistical analyses for this end point

    Secondary: Number of Subjects Experiencing Postdose Antidrug Antibodies (ADA) to Efgartigimod

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    End point title
    		 Number of Subjects Experiencing Postdose Antidrug Antibodies (ADA) to Efgartigimod
    End point description
    In Cohorts 1-3, blood samples to assess ADA were collected at Baseline, Visit 3, Visit 5, all Maintenance treatment visits, Follow-up 1, Follow-up 2 and Follow-up 3. In Cohort 4, blood samples to assess ADA were collected at Baseline, then biweekly until End of Treatment, and at each Follow-up visit. The Full Analysis Set included all subjects who received at least one dose of study drug.
    End point type
    Secondary
    End point timeframe
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    End point values
    		 Cohorts 1-4
    Number of subjects analysed
    31
    Units: subjects
    3
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From the start of the first IMP (Day 1) up to end of the follow-up period, approximately 44 weeks
    Adverse event reporting additional description
    The SAS included all enrolled subjects who received at least 1 dose of efgartigimod.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.1
    Reporting groups
    Reporting group title
    Cohort 1 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period at weeks 2 and 6.

    Reporting group title
    Cohort 2 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period once q2w for 8 weeks.

    Reporting group title
    Cohort 3 (10 mg/kg)
    Reporting group description
    		 Efgartigimod IV 10 mg/kg was administered as 4 weekly infusions during the induction period and during the maintenance period q2w for 12 weeks.

    Reporting group title
    Cohort 4 (25 mg/kg)
    Reporting group description
    		 Efgartigimod IV 25 mg/kg was administered as 5 weekly infusions (minimum) during the induction period until EoC, and during the maintenance period q2w until Week 34.

    Serious adverse events
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 6 (16.67%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Tibia fracture
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Pneumonia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Cohort 1 (10 mg/kg) Cohort 2 (10 mg/kg) Cohort 3 (10 mg/kg) Cohort 4 (25 mg/kg)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    6 / 6 (100.00%)
    3 / 5 (60.00%)
    6 / 8 (75.00%)
    13 / 15 (86.67%)
    Vascular disorders
    Flushing
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Hypertension
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    1
    Influenza like illness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    4
    Pyrexia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    3
    0
    0
    0
    Infusion site swelling
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Hernia pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Oedema peripheral
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Nasal dryness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Cough
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    2
    1
    Respiratory failure
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    2
    Aspartate aminotransferase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Blood creatine phosphokinase increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Blood creatinine increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Weight increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Cystatin C increased
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Injury, poisoning and procedural complications
    Traumatic ulcer
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    1 / 15 (6.67%)
         occurrences all number
    3
    0
    1
    1
    Headache
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    3 / 15 (20.00%)
         occurrences all number
    1
    0
    0
    3
    Paraesthesia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Syncope
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    2
    Neutrophilia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Anaemia macrocytic
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Ear and labyrinth disorders
    Excessive cerumen production
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Middle ear inflammation
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Eye disorders
    Blindness transient
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    2 / 8 (25.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    3
    Abdominal pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    1
    2
    Vomiting
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    1
    1
    Dyspepsia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Abdominal pain upper
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    2
    Nausea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Gastrointestinal disorder
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Oral pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Toothache
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Oral disorder
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Dry skin
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    1
    1
    Alopecia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Psoriasis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Pruritus
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Rash erythematous
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Urticaria
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Renal and urinary disorders
    Renal cyst
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Renal pain
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    2
    0
    0
    1
    Endocrine disorders
    Cushingoid
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Back pain
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Muscular weakness
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Pain in extremity
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    4 / 15 (26.67%)
         occurrences all number
    0
    0
    0
    4
    Urinary tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    2
    2
    Rhinitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    3
    Bronchitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    1
    0
    Gastroenteritis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    1
    0
    1
    Impetigo
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    1
    0
    0
    1
    Pustule
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    2
    0
    Bacteriuria
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Candida infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Oral herpes
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Conjunctivitis
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    0
    1
    0
    0
    Folliculitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Pulpitis dental
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Respiratory tract infection
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Sialoadenitis
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Tonsillitis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Skin infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Tooth infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Metabolism and nutrition disorders
    Type 2 diabetes mellitus
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    20 Oct 2017
    The following sections of the protocol were updated: - Schedule of Assessments - Section 5.1 Summary of Study Design - Section 5.3 Selection of Study Population - Section 5.3.1 Inclusion Criteria - Section 5.3.2 Exclusion Criteria - Section 6.3.4 Visit 6 and Visit 7 (Maintenance treatment period) - Section 6.6 Unscheduled Visit - Section 8.1.2.2 Other Laboratory Assessments - Section 9.2 Quality Control of Data - Section 10.2 Statistical Methods
    28 Feb 2018
    The following sections of the protocol were updated: - Section 3.1 Primary Objectives - Section 5.3 Selection of Study Population - Synopsis Section 5.3.1 Inclusion Criteria - Synopsis Methodology - Section 11.4 Patient Data Protection
    11 Jun 2018
    The following sections of the protocol were updated: - Section 5.1 Summary of Study Design - Schedule of Assessments - Section 5.3.1 Inclusion Criteria - Section 5.3.6 Sample Size Increase - Section 7.7 Prior and Concomitant Treatments - Section 7.8.1 Rescue Therapy - Section 8.1.2.2 Other Laboratory Assessments
    06 Feb 2019
    The following sections of the protocol were updated: - Section 3 Study Objectives - Section 4.2 Secondary endpoints - Section 5.1 Summary of Study Design - Section 5.3.6 Sample Size Increase - Section 5.3.1 Inclusion Criteria - Section 5.3.8 Screen Failures and Rescreening - Section 6.2 Screening - Sections 6.4, 6.5.1, 8.1.2.2 Assessments - Section 7.1 Treatments Administered - Section 7.7 Prior and Concomitant Treatments - Section 7.8.1 Rescue Therapy - Section 8.1.2.2 Other Laboratory Assessments - Section 8.5 Antidrug Antibodies - Section 10.4 Interim Analysis
    26 Jun 2019
    The following sections of the protocol were updated: - Section 6.4.2 Visit 2 to EoC - Section 7.1 Treatments Administered

    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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