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    Clinical Trial Results:
    A Randomized, Double-Blind, Placebo-Controlled, Multicenter Phase 3 Trial to Evaluate the Efficacy, Safety and Tolerability of ARGX-113 in Patients with Myasthenia Gravis Having Generalized Muscle Weakness (ADAPT)

    Summary
    EudraCT number
    		 2018-002132-25
    Trial protocol
    		 NL   DK   FR   CZ   DE   HU   BE   IT  
    Global end of trial date
    06 Apr 2020

    Results information
    Results version number
    		 v1(current)
    This version publication date
    22 Apr 2021
    First version publication date
    22 Apr 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ARGX-113-1704
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03669588
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    argenx BVBA
    Sponsor organisation address
    Industriepark Zwijnaarde 7, Zwijnaarde, Belgium, 9052
    Public contact
    Regulatory Manager, argenx BVBA, regulatory@argenx.com
    Scientific contact
    Regulatory Manager, argenx BVBA, 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
    06 Apr 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    06 Apr 2020
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of ARGX-113 (efgartigimod) as assessed by the percentage of Myasthenia Gravis Activities of Daily Living (MG-ADL) responders after the first treatment cycle (TC) in the acetylcholine receptor (AChR)-antibody (Ab) seropositive (AChR-Ab seropositive) population. The study included both AChR-Ab seropositive and seronegative patients. AChR-Ab seronegative patients were patients in whom AChR-Ab was undetectable in serum during the screening period, using a radioimmunoassay.
    Protection of trial subjects
    This study was conducted 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
    		 Patients were required to be on a stable dose of standard of care (SoC) (concomitant generalized myasthenia gravis [gMG] treatment) prior to screening. Concomitant gMG treatment was limited to acetylcholinesterase inhibitors, steroids and nonsteroidal immunosuppressive drugs (NSIDs).
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Aug 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Netherlands: 1
    Country: Number of subjects enrolled
    Poland: 30
    Country: Number of subjects enrolled
    Belgium: 6
    Country: Number of subjects enrolled
    Czechia: 13
    Country: Number of subjects enrolled
    Denmark: 1
    Country: Number of subjects enrolled
    France: 1
    Country: Number of subjects enrolled
    Germany: 2
    Country: Number of subjects enrolled
    Hungary: 1
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    Japan: 15
    Country: Number of subjects enrolled
    Russian Federation: 6
    Country: Number of subjects enrolled
    Serbia: 19
    Country: Number of subjects enrolled
    United States: 40
    Country: Number of subjects enrolled
    Georgia: 22
    Worldwide total number of subjects
    167
    EEA total number of subjects
    61
    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)
    142
    From 65 to 84 years
    25
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Study was conducted in gMG patients at 56 sites worldwide. Patients were randomized 1:1 within each stratum (Japanese/non-Japanese, AChR-Ab status and SoC) to receive ARGX-113 intravenous (IV) 10 milligrams/kilogram (mg/kg) or placebo, in addition to SoC. Patients completing the study were eligible to roll over into a follow-up study ARGX-113-1705.

    Pre-assignment
    Screening details
    		 Total study duration was up to 28 weeks, including a 2-week screening period, an initial 8-week TC and intertreatment cycle (ITC) of variable length depending on the patient. Patients had to be on a stable dose of SoC and not have received immunoglobulins by IV, subcutaneous or intramuscular route, or plasma exchange, < 1 month prior to screening.

    Period 1
    Period 1 title
    Overall study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 ARGX-113
    Arm description
    		 Patients received ARGX-113 IV 10 mg/kg administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ARGX-113
    Investigational medicinal product code
    Other name
    Efgartigimod
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    ARGX-113 was provided as a 20 mg/milliliter (mL) sterile, colorless and clear concentrate solution for IV administration. Appropriate dilutions in a 0.9% saline solution were made on site prior to administration. For each administration, patients were administered a dose of 10 mg/kg of body weight in a total volume of 125 mL, infused over 1 hour. The maximum permitted ARGX-113 dose per infusion was 1200 mg.

    Arm title
    		 Placebo
    Arm description
    		 Patients received matching placebo administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Matched placebo was provided as a sterile, colorless and clear concentrate solution for IV administration. Appropriate dilutions in a 0.9% saline solution were made on site prior to administration. For each administration, patients were administered a total volume of 125 mL, infused over 1 hour.

    Number of subjects in period 1
    		ARGX-113 Placebo
    Started
    84
    83
    Completed
    80
    76
    Not completed
    4
    7
         Consent withdrawn by subject
    1
    4
         Physician decision
    -
    1
         Adverse event, non-fatal
    1
    -
         Sponsor decision
    1
    1
         Rescue therapy needed
    -
    1
         Protocol deviation
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ARGX-113
    Reporting group description
    		 Patients received ARGX-113 IV 10 mg/kg administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients received matching placebo administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.

    Reporting group values
    		 ARGX-113 Placebo Total
    Number of subjects
    		 84 83 167
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 73 69 142
        From 65-84 years
    		 11 14 25
        85 years and over
    		 0 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 45.9 ( 14.41 ) 48.2 ( 14.97 ) -
    Gender categorical
    Units: Subjects
        Female
    		 63 55 118
        Male
    		 21 28 49
    Race
    Units: Subjects
        American Indian or Alaska native
    		 2 0 2
        Asian
    		 9 7 16
        Black or African American
    		 3 3 6
        White
    		 69 72 141
        Multiple
    		 1 0 1
        Not reported
    		 0 1 1
    Ethnicity, Customized
    Units: Subjects
        Japanese
    		 8 7 15
        Hispanic or Latino
    		 7 2 9
        Not Hispanic or Latino
    		 69 73 142
        Not reported
    		 0 1 1
    Concomitant gMG treatment
    Units: Subjects
        NSIDs
    		 51 51 102
        No NSIDs
    		 33 32 65
    AChR-Ab status
    Units: Subjects
        Positive
    		 65 64 129
        Negative
    		 19 19 38

    End points

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    End points reporting groups
    Reporting group title
    ARGX-113
    Reporting group description
    		 Patients received ARGX-113 IV 10 mg/kg administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients received matching placebo administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle. Each 8-week TC comprised a 3-week treatment period and a 5-week follow-up period. At the end of each TC, patients entered the ITC period consisting of visits every 2 weeks. At each ITC visit, retreatment criteria were evaluated to determine if the patient was eligible to enter the next cycle for retreatment, based on clinical response as measured by the MG-ADL scale.

    Primary: Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population

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    End point title
    		 Percentage of MG-ADL Responders During Cycle 1 (C1); Analyzed in the AChR-Ab Seropositive Population
    End point description
    The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 [C1B]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of investigational medicinal product (IMP) in C1. Analysis was performed in the AChR-Ab seropositive population using the modified intention-to-treat (mITT) analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Primary
    End point timeframe
    Baseline up to Day 63 (end of TC1)
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    65
    64
    Units: percentage of patients
        number (not applicable)
    67.7
    29.7
    Statistical analysis title
    		 Comparison of ARGX-113 versus (vs) Placebo
    Statistical analysis description
    MG-ADL responders after the first TC: 2-sided exact test (using logistic regression) at the 2-sided 5% significance level in the AChR-Ab seropositive population, stratified by Japanese vs non-Japanese and NSID vs no NSID as concomitant gMG treatment, with cycle baseline MG-ADL total score as covariate. The treatment effect was presented as the odds ratio. An odds ratio of more than 1 represents a higher response rate for ARGX-113 compared to placebo.
    Comparison groups
    ARGX-113 v Placebo
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    4.951
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2.213
         upper limit
    		 11.528

    Secondary: Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population

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    End point title
    		 Percentage of Quantitative Myasthenia Gravis (QMG) Responders During C1; Analyzed in the AChR-Ab Seropositive Population
    End point description
    The QMG scale quantifies disease severity based on impairments of body functions and structures as defined by the International Classification of Disability and Health. The QMG scale consists of 13 items that measure endurance or fatigability, and accounts for fluctuations in disease state. The QMG total score range is 0-39, with higher scores indicative of greater disease severity. A patient was considered a QMG responder during C1 if there was a reduction of ≥3-points on the QMG total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1. Analysis was performed in the AChR-Ab seropositive population using the mITT analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 63 (end of TC1)
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    65
    64
    Units: percentage of patients
        number (not applicable)
    63.1
    14.1
    Statistical analysis title
    		 Comparison of ARGX-113 vs Placebo
    Statistical analysis description
    QMG responders after the first TC: 2-sided exact test (using logistic regression) at the 2-sided 5% significance level in the AChR-Ab seropositive population, stratified by Japanese vs non-Japanese and NSID vs no NSID as concomitant gMG treatment, with cycle baseline QMG total score as covariate. The treatment effect was presented as the odds ratio. An odds ratio of more than 1 represents a higher response rate for ARGX-113 compared to placebo.
    Comparison groups
    ARGX-113 v Placebo
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    10.842
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 4.179
         upper limit
    		 31.2

    Secondary: Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population

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    End point title
    		 Percentage of MG-ADL Responders During C1; Analyzed in the Overall Population
    End point description
    The percentage of MG-ADL responders during C1 in the overall population is reported for this secondary end point; percentage of MG-ADL responders during C1 in the AChR-Ab seropositive population is reported previously as a primary end point. Analysis was performed in the overall population (including both AChR-Ab seropositive and seronegative patients) using the mITT analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 63 (end of TC1)
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    84
    83
    Units: percentage of patients
        number (not applicable)
    67.9
    37.3
    Statistical analysis title
    		 Comparison of ARGX-113 vs Placebo
    Statistical analysis description
    MG-ADL responders after the first TC: 2-sided exact test (using logistic regression) at the 2-sided 5% significance level in the overall population, stratified by AChR-Ab status (seropositive vs seronegative), Japanese vs non-Japanese and NSID vs no NSID as concomitant gMG treatment, with cycle baseline MG-ADL total score as covariate. The treatment effect was presented as the odds ratio. An odds ratio of more than 1 represents a higher response rate for ARGX-113 compared to placebo.
    Comparison groups
    ARGX-113 v Placebo
    Number of subjects included in analysis
    167
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    3.699
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.854
         upper limit
    		 7.578

    Secondary: Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population

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    End point title
    		 Percentage of Time That Patients Had a Clinically Meaningful Improvement (CMI) in MG-ADL Total Score up to and Including Day 126; Analyzed in the AChR-Ab Seropositive Population
    End point description
    An MG-ADL CMI was defined as a reduction of ≥2 points on the total MG-ADL score compared to study entry baseline (SEB). Analysis was performed in the AChR-Ab seropositive population using the mITT analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 126
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    65
    64
    Units: percentage of time (in days)
        least squares mean (standard error)
    48.714 ( 6.163 )
    26.649 ( 6.316 )
    Statistical analysis title
    		 Comparison of ARGX-113 vs Placebo
    Statistical analysis description
    Percentage of time patients had a CMI in MG-ADL total score up to and including Day 126: Analysis of covariance (ANCOVA) in the AChR-Ab seropositive population with treatment, baseline MG-ADL total score, Japanese vs non-Japanese, and NSID vs no NSID as concomitant gMG treatment as the covariates.
    Comparison groups
    ARGX-113 v Placebo
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0001
    Method
    		 ANCOVA
    Parameter type
    		 Least square means difference
    Point estimate
    22.065
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 10.949
         upper limit
    		 33.181
    Variability estimate
    Standard error of the mean
    Dispersion value
    5.616

    Secondary: Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population

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    End point title
    		 Time From Week 4 to Qualify for Retreatment; Analyzed in the AChR-Ab Seropositive Population
    End point description
    Time to qualify for retreatment was defined as time from the Week 4 assessment until the first visit with a <2-point reduction compared to SEB in the MG-ADL total score and MG-ADL total score ≥5 points with >50% of the total score attributable to nonocular symptoms. Analysis was performed in the AChR-Ab seropositive population using the mITT analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Secondary
    End point timeframe
    Week 4 up to Day 182 (end of study [EoS])
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    65
    64
    Units: days
        median (confidence interval 95%)
    35.0 (29.0 to 43.0)
    8.0 (1.0 to 30.0)
    Statistical analysis title
    		 Comparison of ARGX-113 vs Placebo
    Statistical analysis description
    Time from Week 4 to qualify for retreatment: Analysis was performed in the AChR-Ab seropositive population and the p-value was calculated using the log-rank test, stratified by Japanese vs non-Japanese and NSID vs no NSID as concomitant gMG treatment.
    Comparison groups
    Placebo v ARGX-113
    Number of subjects included in analysis
    129
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.2604
    Method
    		 Logrank
    Confidence interval

    Secondary: Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population

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    End point title
    		 Percentage of Early MG-ADL Responders During C1; Analyzed in the AChR-Ab Seropositive Population
    End point description
    A patient was considered an early MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to C1B) for ≥4 consecutive weeks with the first reduction occurring no later than Week 2 (ie, after 1 or maximum 2 infusions of IMP in C1). Analysis was performed in the AChR-Ab seropositive population using the mITT analysis set which included all randomized patients with a value for the MG-ADL total score at baseline and at least 1 postbaseline timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline up to Day 63 (end of TC1)
    End point values
    		 ARGX-113 Placebo
    Number of subjects analysed
    65
    64
    Units: percentage of patients
        number (not applicable)
    56.9
    25.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Treatment-emergent adverse events were monitored from first administration of IMP on Day 1 until the EoS (up to a maximum of approximately 182 days).
    Adverse event reporting additional description
    The safety analysis set included patients in the randomized population who received at least a partial dose of IMP.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    23.0
    Reporting groups
    Reporting group title
    ARGX-113
    Reporting group description
    		 Patients received ARGX-113 IV 10 mg/kg administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle.

    Reporting group title
    Placebo
    Reporting group description
    		 Patients received matching placebo administered as a 1-hour infusion every 7 days for 4 infusions (Days 1, 8, 15, and 22) in each cycle.

    Serious adverse events
    		 ARGX-113 Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 84 (4.76%)
    7 / 83 (8.43%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Rectal adenocarcinoma
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 83 (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
    Procedural pain
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Spinal compression fracture
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myocardial ischaemia
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Myasthenia gravis
         subjects affected / exposed
    1 / 84 (1.19%)
    2 / 83 (2.41%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myasthenia gravis crisis
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Thrombocytosis
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Therapeutic product ineffective
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Depression
         subjects affected / exposed
    1 / 84 (1.19%)
    0 / 83 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Spinal ligament ossification
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 84 (0.00%)
    1 / 83 (1.20%)
         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
    		 ARGX-113 Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    49 / 84 (58.33%)
    48 / 83 (57.83%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    3 / 84 (3.57%)
    6 / 83 (7.23%)
         occurrences all number
    4
    6
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 84 (3.57%)
    5 / 83 (6.02%)
         occurrences all number
    5
    5
    Headache
         subjects affected / exposed
    24 / 84 (28.57%)
    23 / 83 (27.71%)
         occurrences all number
    40
    39
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    6 / 84 (7.14%)
    9 / 83 (10.84%)
         occurrences all number
    6
    14
    Nausea
         subjects affected / exposed
    7 / 84 (8.33%)
    9 / 83 (10.84%)
         occurrences all number
    7
    15
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    3 / 84 (3.57%)
    5 / 83 (6.02%)
         occurrences all number
    3
    5
    Oropharyngeal pain
         subjects affected / exposed
    3 / 84 (3.57%)
    7 / 83 (8.43%)
         occurrences all number
    3
    7
    Musculoskeletal and connective tissue disorders
    Myalgia
         subjects affected / exposed
    5 / 84 (5.95%)
    1 / 83 (1.20%)
         occurrences all number
    6
    3
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    5 / 84 (5.95%)
    2 / 83 (2.41%)
         occurrences all number
    6
    2
    Nasopharyngitis
         subjects affected / exposed
    10 / 84 (11.90%)
    15 / 83 (18.07%)
         occurrences all number
    12
    17
    Upper respiratory tract infection
         subjects affected / exposed
    9 / 84 (10.71%)
    4 / 83 (4.82%)
         occurrences all number
    11
    4
    Urinary tract infection
         subjects affected / exposed
    8 / 84 (9.52%)
    4 / 83 (4.82%)
         occurrences all number
    9
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    28 Nov 2018
    Changes encompassed by this amendment included updates for clarification or to apply minor corrections.
    11 Jul 2019
    Changes encompassed by this amendment included updates for clarification or to apply minor corrections. A new section for Adverse Events of Special Interest was 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.
    None reported
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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
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