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    Clinical Trial Results:
    An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants with Generalized Myasthenia Gravis

    Summary
    EudraCT number
    		 2020-003230-20
    Trial protocol
    		 GB   DE   CZ   FR   PL   HU   DK   BE   IT  
    Global end of trial date
    25 Jan 2024

    Results information
    Results version number
    		 v2
    This version publication date
    28 Mar 2025
    First version publication date
    07 Feb 2025
    Other versions
    		 v1 , v3
    Version creation reason
    • Correction of full data set
    Alignment with final posting on ClinicalTrials.gov after NIH review.

    Trial information

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    Trial identification
    Sponsor protocol code
    MG0007
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04650854
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    UCB Biopharma SRL
    Sponsor organisation address
    Allée de la Recherche 60, Brussels, Belgium, 1070
    Public contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.com
    Scientific contact
    Clin Trial Reg & Results Disclosure, UCB BIOSCIENCES GmbH, clinicaltrials@ucb.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
    12 Apr 2024
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    25 Jan 2024
    Global end of trial reached?
    Yes
    Global end of trial date
    25 Jan 2024
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Assess the safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)
    Protection of trial subjects
    During the conduct of the study all participants were closely monitored.
    Background therapy
    		 Background therapy as permitted in the protocol.
    Evidence for comparator
    		 Not applicable
    Actual start date of recruitment
    03 Feb 2021
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 19
    Country: Number of subjects enrolled
    Czechia: 2
    Country: Number of subjects enrolled
    Denmark: 5
    Country: Number of subjects enrolled
    France: 13
    Country: Number of subjects enrolled
    Georgia: 13
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Italy: 11
    Country: Number of subjects enrolled
    Japan: 12
    Country: Number of subjects enrolled
    Poland: 26
    Country: Number of subjects enrolled
    Russian Federation: 12
    Country: Number of subjects enrolled
    Serbia: 5
    Country: Number of subjects enrolled
    Taiwan: 3
    Country: Number of subjects enrolled
    United States: 32
    Country: Number of subjects enrolled
    Spain: 5
    Worldwide total number of subjects
    165
    EEA total number of subjects
    69
    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)
    123
    From 65 to 84 years
    40
    85 years and over
    2

    Subject disposition

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    Recruitment
    Recruitment details
    		 Study started to enroll participants in February 2021 and concluded in January 2024. Participant Flow refers to the Full Analysis Set (FAS).

    Pre-assignment
    Screening details
    		 Participants who completed MG0003 (NCT03971422) or required rescue therapy during observation period (OP) in MG0003 (except who received intravenous immunoglobulin/plasma exchange) or completed at least 6 treatment visits in MG0004 (NCT04124965) were enrolled in study.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Enrolled, but not Treated
    Arm description
    		 Participants were enrolled but did not receive a treatment cycle of Rozanolixizumab (RLZ) in MG0007 per the Investigator’s discretion.
    Arm type
    		 No intervention

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Arm title
    		 Rozanolixizumab ~7 mg/kg
    Arm description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    RLZ
    Pharmaceutical forms
    Infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received rozanolixizumab at pre-specified time-points.

    Arm title
    		 Rozanolixizumab ~10 mg/kg
    Arm description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Rozanolixizumab
    Investigational medicinal product code
    UCB7665
    Other name
    RLZ
    Pharmaceutical forms
    Infusion
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants received rozanolixizumab at pre-specified time-points.

    Number of subjects in period 1
    		Enrolled, but not Treated Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Started
    8
    80
    77
    Completed
    3
    15
    16
    Not completed
    5
    65
    61
         End Of Study
    -
    2
    1
         Enrollment Cup
    -
    -
    1
         Discontinued Due to Chronic Heart Failure
    -
    -
    1
         Withdraw Per Sponsor's Directive
    -
    1
    -
         Participant Withdraw After Drug Approved By FDA
    -
    -
    1
         Rescue Medication
    -
    -
    1
         Adverse event, non-fatal
    1
    12
    14
         Subject Performed PEOT, Not a Completed Subject
    -
    1
    -
         Last Treatment Per Sponsor
    -
    -
    2
         Last Treatment Per Sponsor Lplv Timelines- Japan
    -
    3
    3
         Withdrawal Due to Prohibited Treatment
    -
    -
    1
         Withdraw Based on Sponsor Decision
    -
    -
    1
         Participant Screened For MG0020
    -
    2
    3
         Use of Rescue Medication
    -
    -
    1
         Participant to Be Screened for MG0020 Study
    -
    4
    5
         Patient Withdrawn; Lack of IMP Shipments
    -
    2
    1
         Wish To Continue Study IMP in Patient Programme
    -
    1
    -
         Participant Received Rescue Medication
    -
    1
    -
         Participant Want to Start a Family
    -
    -
    1
         Consent withdrawn by subject
    2
    17
    9
         Last Treatment Per Lplv, Screened For MG0020
    -
    2
    2
         Last Treatment Per Sponsor Lplv Timelines
    -
    12
    6
         Pi Withdrew the Patient From the Study
    -
    -
    1
         Lost to follow-up
    1
    1
    -
         Patient Withdraw Due To Inappropriate Behavior
    -
    1
    -
         Lack of efficacy
    1
    3
    6

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Enrolled, but not Treated
    Reporting group description
    		 Participants were enrolled but did not receive a treatment cycle of Rozanolixizumab (RLZ) in MG0007 per the Investigator’s discretion.

    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Reporting group values
    		 Enrolled, but not Treated Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg Total
    Number of subjects
    		 8 80 77 165
    Age Categorical
    Units: participants
        18 to <65 years
    		 5 61 57 123
        65 to <85 years
    		 2 19 19 40
        >=85 years
    		 1 0 1 2
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 59.6 ( 17.6 ) 52.5 ( 15.7 ) 52.2 ( 17.0 ) -
    Sex/Gender, Customized
    Units: participants
        Female
    		 3 45 48 96
        Male
    		 5 35 29 69

    End points

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    End points reporting groups
    Reporting group title
    Enrolled, but not Treated
    Reporting group description
    		 Participants were enrolled but did not receive a treatment cycle of Rozanolixizumab (RLZ) in MG0007 per the Investigator’s discretion.

    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Subject analysis set title
    Rozanolixizumab ~7 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Subject analysis set title
    Rozanolixizumab ~10 mg/kg
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Primary: Percentage of participants with treatment-emergent adverse events (TEAEs)

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    End point title
    		 Percentage of participants with treatment-emergent adverse events (TEAEs) [1]
    End point description
    An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which did not necessarily had a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. TEAE was defined as any event that was not present prior to first dose of investigational medicinal product (IMP), or any unresolved event already present that worsened in intensity following treatment, up to 8 weeks after end of Treatment Period or after last dose of IMP in participants who discontinued study or IMP. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both Rozanolixizumab (RLZ) doses.
    End point type
    Primary
    End point timeframe
    From Baseline (Day 1) to End of Study (up to 34 months)
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    102
    102
    Units: percentage of participants
        number (not applicable)
    78.4
    94.1
    No statistical analyses for this end point

    Primary: Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP)

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    End point title
    		 Percentage of participants with TEAEs leading to withdrawal of investigational medicinal product (IMP) [2]
    End point description
    An adverse event (AE) was any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Participants who switched doses were counted in both RLZ doses.
    End point type
    Primary
    End point timeframe
    From Baseline (Day 1) to End of Study (up to 34 months)
    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 formal statistical hypothesis testing was planned for this study. Results were summarized as descriptive statistics only.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    102
    102
    Units: percentage of participants
        number (not applicable)
    9.8
    16.7
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle (Cycle 1, 2, and 3) [3]
    End point description
    The MG-ADL is an 8-item PRO instrument developed on basis of QMG. The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0= no symptoms or impaired performance and 3= most severe symptoms or impaired performance. The total score ranges from 0 to 24, with higher score indicating more disability. A positive change in score indicates worsening and negative change indicates improvement. For analyses done by study cycle, Baseline values were last available values prior to or on the same date of first administration of IMP at each cycle (Baseline [Day 1]) value for that cycle. Safety Set (SS): All study participants in FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for assessment. 'n' included those participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [3] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    74
    66
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=74,66)
    -3.7 ( 3.5 )
    -3.1 ( 2.9 )
        Cycle 2: Treatment (Day 43) (n=56,65)
    -2.9 ( 3.1 )
    -3.8 ( 3.9 )
        Cycle 3: Treatment (Day 43) (n=41,58)
    -3.3 ( 2.7 )
    -3.2 ( 3.3 )
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle (Cycle 1, 2, and 3) [4]
    End point description
    The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change in the score indicates worsening and a negative change indicates improvement. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed included those participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [4] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    73
    64
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=73,64)
    -4.5 ( 5.0 )
    -4.1 ( 4.2 )
        Cycle 2: Treatment (Day 43) (n=55,64)
    -3.9 ( 4.1 )
    -4.9 ( 5.5 )
        Cycle 3: Treatment (Day 43) (n=41,57)
    -5.1 ( 4.8 )
    -4.2 ( 4.4 )
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle (Cycle 1, 2, and 3) [5]
    End point description
    MG-C scale consists of 10 items: ptosis (0 to 3), double vision on lateral gaze left/right/both (0 to 4), eye closure (0 to 2), talking (0 to 6), chewing (0 to 6), swallowing (0 to 6), breathing (0 to 9), neck flexion or extension (0 to 4), shoulder abduction (0 to 5) and hip flexion (0 to 5), lower scores indicates lower disease activity. Total MG-C score obtained by summing responses to each individual item and score ranges from 0 - 50, (lower scores= lower disease activity). A positive change indicates worsening and a negative change improvement. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed included participants who were evaluable for the assessment. 'n' included participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    73
    66
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=73,66)
    -7.6 ( 7.3 )
    -4.7 ( 5.7 )
        Cycle 2: Treatment (Day 43) (n=56,65)
    -5.7 ( 5.4 )
    -7.5 ( 7.0 )
        Cycle 3: Treatment (Day 43) (n=41,56)
    -6.8 ( 5.9 )
    -6.1 ( 7.2 )
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle (Cycle 1, 2, and 3) [6]
    End point description
    The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular symptoms (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option that best described severity of ocular, bulbar, and respiratory symptoms over past 7 days using a 4-point Likert scale (“none” to “severe”) and frequency of experiencing physical fatigue and muscle weakness fatigability over past 7 days using a 5-point Likert scale (1=“none of the time” – 5= “all of the time”), for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100, higher scores indicates more severe symptoms. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for assessment. 'n' included participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [6] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    75
    66
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=75,66)
    -17.6 ( 21.0 )
    -14.6 ( 17.2 )
        Cycle 2: Treatment (Day 43) (n=56,65)
    -13.2 ( 19.2 )
    -19.2 ( 21.6 )
        Cycle 3: Treatment (Day 43) (n=41,58)
    -12.8 ( 14.4 )
    -15.4 ( 17.7 )
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle (Cycle 1, 2, and 3) [7]
    End point description
    The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Study participants had to choose response option based on frequency of experiencing physical fatigue (19-33) over past 7 days using a 5-point Likert scale (1=“none of time” - 5=“all of time”) for each item. Sum of each item score is linearly transformed to have all domain scores and total score ranging from 0 to 100, higher scores indicated severe symptoms. Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    75
    66
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=75,66)
    -16.5 ( 18.6 )
    -14.8 ( 18.5 )
        Cycle 2: Treatment (Day 43) (n=56,65)
    -13.6 ( 21.6 )
    -16.0 ( 18.2 )
        Cycle 3: Treatment (Day 43) (n=41,58)
    -15.3 ( 16.9 )
    -15.0 ( 18.4 )
    No statistical analyses for this end point

    Secondary: Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' score within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' score within one treatment cycle (Cycle 1, 2, and 3) [8]
    End point description
    The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (1-5); bulbar muscle weakness (6-15); respiratory muscle weakness (16-18); physical fatigue (19-33) and muscle weakness fatigability (34-42). Bulbar symptoms are now recognised as bulbar muscle weakness. Study participants were asked to choose response option that best described severity of bulbar muscle weakness (6-15) symptoms over past 7 days using a 4-point Likert scale (1=“none” to 4=“severe”) for each item. Sum of each item score is linearly transformed to have all domain scores and total score ranging from 0 to 100, higher scores indicated severe symptoms. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [8] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    75
    66
    Units: score on a scale
    arithmetic mean (standard deviation)
        Cycle 1: Treatment (Day 43) (n=75,66)
    -13.4 ( 19.8 )
    -11.6 ( 16.2 )
        Cycle 2: Treatment (Day 43) (n=56,65)
    -10.4 ( 16.2 )
    -15.5 ( 18.1 )
        Cycle 3: Treatment (Day 43) (n=41,58)
    -14.0 ( 16.8 )
    -13.0 ( 17.9 )
    No statistical analyses for this end point

    Secondary: MG-ADL responder rate (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3 [Day 43])

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    End point title
    		 MG-ADL responder rate (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3 [Day 43]) [9]
    End point description
    The MG-ADL is an 8-item PRO instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A MG-ADL responder was defined as achieving at least 2.0-point improvement (decrease) in the MG-ADL score from Baseline. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number of Participants Analyzed: participants who were evaluable for the assessment. 'n' included those participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    Day 43 of Cycle 1, 2, and 3
    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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    74
    66
    Units: percentage of participants
    number (not applicable)
        Cycle 1: Treatment (Day 43) (n=74,66)
    74.3
    63.6
        Cycle 2: Treatment (Day 43) (n=56,65)
    62.5
    67.7
        Cycle 3: Treatment (Day 43) (n=41,58)
    73.2
    67.2
    No statistical analyses for this end point

    Secondary: Time to MG-ADL response (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3)

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    End point title
    		 Time to MG-ADL response (>=2.0-point improvement from Baseline [Day 1]) within one treatment cycle (Cycle 1, 2, and 3) [10]
    End point description
    Time to achieve MG-ADL response, defined as at least 2.0-point improvement from Baseline. Time to first MG-ADL response (in days) by study cycle was defined as date of first MG-ADL Response within study cycle − date of MG-ADL Baseline within study cycle + 1. Safety Set (SS): All study participants in the FAS who received at least one dose of IMP. Number Analyzed included those participants who were evaluable at specified timepoint.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Day 43 of Cycle 1, 2, and 3
    Notes
    [10] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    80
    77
    Units: days
    median (confidence interval 95%)
        Cycle 1 (n=80,77)
    9.00 (8.00 to 15.00)
    22.00 (15.00 to 36.00)
        Cycle 2 (n=59,67)
    15.00 (10.00 to 17.00)
    15.00 (9.00 to 22.00)
        Cycle 3 (n=44,61)
    15.00 (9.00 to 15.00)
    15.00 (9.00 to 17.00)
    No statistical analyses for this end point

    Secondary: Time between consecutive treatment cycles

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    End point title
    		 Time between consecutive treatment cycles [11]
    End point description
    Time between consecutive treatment cycles: Study participants were assessed for MG worsening prior to repeated cycles. In case of symptom worsening (eg, an increase of 2.0 points on MG-ADL or 3.0 points on QMG scale) between assessments, resulted additional treatment, study participants undergone another 6-week treatment cycle followed by an Observation Period, based on Investigator's discretion. Time between treatment cycles was calculated as: date of first sc infusion in consecutive cycle - date of last sc infusion before new cycle + 1 (or date of censoring - date of last sc infusion before potential new cycle + 1). Safety Set (SS): All study participants in FAS who received at least 1 dose of IMP. 'n' included participants who were evaluable for specified category. 99999 indicates median and full range were not estimated as no participants were analysed at specific timepoint.
    End point type
    Secondary
    End point timeframe
    From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years
    Notes
    [11] - 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: The endpoint was assessed only for participants who received treatment and the arm ‘Enrolled, but not treated’ did not receive any treatment during this study.
    End point values
    		 Rozanolixizumab ~7 mg/kg Rozanolixizumab ~10 mg/kg
    Number of subjects analysed
    80
    77
    Units: days
    median (full range (min-max))
        Between Cycle 1 and Cycle 2 (n=80,77)
    64.0 (9.0 to 883)
    51.0 (2.0 to 856)
        Between Cycle 2 and Cycle 3 (n=59,67)
    58.0 (1.0 to 631)
    50.0 (15.0 to 575)
        Between Cycle 3 and Cycle 4 (n=44,61)
    42.5 (27 to 399)
    43.0 (5 to 194)
        Between Cycle 4 and Cycle 5 (n=40,55)
    43.0 (9 to 181)
    43.0 (17 to 242)
        Between Cycle 5 and Cycle 6 (n=36,51)
    44.0 (22 to 192)
    43.0 (15 to 175)
        Between Cycle 6 and Cycle 7 (n=33,49)
    36.0 (8 to 348)
    43.0 (7 to 106)
        Between Cycle 7 and Cycle 8 (n=31,40)
    37.0 (22 to 87)
    37.0 (6 to 84)
        Between Cycle 8 and Cycle 9 (n=27,36)
    36.0 (8 to 120)
    36.0 (7 to 134)
        Between Cycle 9 and Cycle 10 (n=21,31)
    29.0 (7 to 97)
    29.0 (8 to 72)
        Between Cycle 10 and Cycle 11 (n=15,26)
    29.0 (8 to 64)
    29.0 (8 to 99)
        Between Cycle 11 and Cycle 12 (n=11,19)
    23.0 (8 to 52)
    22.0 (7 to 37)
        Between Cycle 12 and Cycle 13 (n=9,13)
    22.0 (6 to 36)
    22.0 (7 to 38)
        Between Cycle 13 and Cycle 14 (n=5,9)
    29.0 (22 to 35)
    16.0 (8 to 65)
        Between Cycle 14 and Cycle 15 (n=4,5)
    29.0 (29 to 64)
    9.0 (8 to 16)
        Between Cycle 15 and Cycle 16 (n=3,2)
    23.0 (8 to 29)
    26.5 (9 to 44)
        Between Cycle 16 and Cycle 17 (n=1,0)
    29.0 (29 to 29)
    99999 (99999 to 99999)
        Between Cycle 17 and Cycle 18 (n=1,0)
    8.0 (8 to 8)
    99999 (99999 to 99999)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Baseline (Day 1) until End of Study (up to 34 months)
    Adverse event reporting additional description
    TEAEs were reported. Participants who switched doses were counted in both RLZ doses. Participants of ‘Enrolled, but not Treated’ arm (N=8) did not receive RLZ treatment in MG0007 study as per Investigator’s discretion hence, no TEAEs were reported.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Rozanolixizumab ~10 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of sc dose of RLZ equivalent to approximately 10 mg/kg QW, followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, PEOT Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Reporting group title
    Rozanolixizumab ~7 mg/kg
    Reporting group description
    		 Eligible participants from MG0003 were randomized to receive an initial fixed 6-week treatment cycle of subcutaneous (sc) dose of Rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg) once weekly (QW), followed by OP that began after last dose of that treatment cycle. Eligible participants from MG0004 who completed at least 6 scheduled visits for RLZ treatment, premature end of treatment (PEOT) Visit and who were in OP could complete End of Study Visit could move directly into OP in MG0007. These participants underwent their first MG0007 treatment with RLZ upon worsening of MG symptoms. Participants continued their last treatment dose from MG0004. Dose adjustments could be applied in further cycles as per Investigator’s discretion. If symptom worsens between assessments, resulting in need for additional treatment, participants underwent another 6-week treatment cycle followed by OP, based on Investigator’s discretion.

    Serious adverse events
    		 Rozanolixizumab ~10 mg/kg Rozanolixizumab ~7 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    31 / 102 (30.39%)
    16 / 102 (15.69%)
         number of deaths (all causes)
    3
    1
         number of deaths resulting from adverse events
    0
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Prostate cancer stage II
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Thymoma
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Intestinal adenocarcinoma
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Retroperitoneal neoplasm
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Invasive ductal breast carcinoma
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Shock haemorrhagic
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hypertension
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Surgical and medical procedures
    Thymectomy
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General physical health deterioration
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyrexia
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Haemothorax
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Bronchitis chronic
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Arteriovenous fistula thrombosis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Atrial fibrillation
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Angina unstable
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac failure
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Sciatica
         subjects affected / exposed
    1 / 102 (0.98%)
    1 / 102 (0.98%)
         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
    4 / 102 (3.92%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 4
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myasthenia gravis
         subjects affected / exposed
    11 / 102 (10.78%)
    5 / 102 (4.90%)
         occurrences causally related to treatment / all
    0 / 17
    0 / 8
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Transient global amnesia
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Iron deficiency anaemia
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Macular hole
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Duodenal ulcer haemorrhage
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Oesophageal food impaction
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Large intestine polyp
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Subacute cutaneous lupus erythematosus
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Dysuria
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Endocrine disorders
    Adrenal disorder
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Osteoarthritis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Sinusitis aspergillus
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 102 (0.98%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Liver abscess
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    COVID-19
         subjects affected / exposed
    2 / 102 (1.96%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    0 / 0
    Meningitis aseptic
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Endocarditis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia bacterial
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Diverticulitis
         subjects affected / exposed
    0 / 102 (0.00%)
    1 / 102 (0.98%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Complicated appendicitis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urosepsis
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Septic shock
         subjects affected / exposed
    1 / 102 (0.98%)
    0 / 102 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Rozanolixizumab ~10 mg/kg Rozanolixizumab ~7 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    80 / 102 (78.43%)
    68 / 102 (66.67%)
    Investigations
    Blood cholesterol increased
         subjects affected / exposed
    7 / 102 (6.86%)
    0 / 102 (0.00%)
         occurrences all number
    10
    0
    Blood immunoglobulin G decreased
         subjects affected / exposed
    15 / 102 (14.71%)
    6 / 102 (5.88%)
         occurrences all number
    27
    13
    Blood triglycerides increased
         subjects affected / exposed
    6 / 102 (5.88%)
    0 / 102 (0.00%)
         occurrences all number
    10
    0
    Lymphocyte count decreased
         subjects affected / exposed
    7 / 102 (6.86%)
    3 / 102 (2.94%)
         occurrences all number
    8
    8
    Low density lipoprotein increased
         subjects affected / exposed
    8 / 102 (7.84%)
    0 / 102 (0.00%)
         occurrences all number
    9
    0
    Vascular disorders
    Hypertension
         subjects affected / exposed
    8 / 102 (7.84%)
    3 / 102 (2.94%)
         occurrences all number
    11
    4
    Nervous system disorders
    Headache
         subjects affected / exposed
    46 / 102 (45.10%)
    36 / 102 (35.29%)
         occurrences all number
    218
    99
    Dizziness
         subjects affected / exposed
    10 / 102 (9.80%)
    2 / 102 (1.96%)
         occurrences all number
    11
    2
    Myasthenia gravis
         subjects affected / exposed
    6 / 102 (5.88%)
    2 / 102 (1.96%)
         occurrences all number
    6
    2
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    7 / 102 (6.86%)
    1 / 102 (0.98%)
         occurrences all number
    8
    1
    Oedema peripheral
         subjects affected / exposed
    8 / 102 (7.84%)
    7 / 102 (6.86%)
         occurrences all number
    9
    8
    Pyrexia
         subjects affected / exposed
    19 / 102 (18.63%)
    9 / 102 (8.82%)
         occurrences all number
    34
    20
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    28 / 102 (27.45%)
    20 / 102 (19.61%)
         occurrences all number
    55
    49
    Abdominal pain upper
         subjects affected / exposed
    6 / 102 (5.88%)
    2 / 102 (1.96%)
         occurrences all number
    8
    2
    Abdominal pain
         subjects affected / exposed
    7 / 102 (6.86%)
    10 / 102 (9.80%)
         occurrences all number
    11
    11
    Vomiting
         subjects affected / exposed
    8 / 102 (7.84%)
    2 / 102 (1.96%)
         occurrences all number
    10
    3
    Nausea
         subjects affected / exposed
    18 / 102 (17.65%)
    9 / 102 (8.82%)
         occurrences all number
    26
    17
    Respiratory, thoracic and mediastinal disorders
    Oropharyngeal pain
         subjects affected / exposed
    9 / 102 (8.82%)
    5 / 102 (4.90%)
         occurrences all number
    13
    5
    Dyspnoea
         subjects affected / exposed
    6 / 102 (5.88%)
    3 / 102 (2.94%)
         occurrences all number
    6
    4
    Cough
         subjects affected / exposed
    6 / 102 (5.88%)
    3 / 102 (2.94%)
         occurrences all number
    7
    5
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    12 / 102 (11.76%)
    5 / 102 (4.90%)
         occurrences all number
    13
    6
    Myalgia
         subjects affected / exposed
    6 / 102 (5.88%)
    1 / 102 (0.98%)
         occurrences all number
    9
    2
    Back pain
         subjects affected / exposed
    8 / 102 (7.84%)
    5 / 102 (4.90%)
         occurrences all number
    8
    7
    Infections and infestations
    COVID-19
         subjects affected / exposed
    24 / 102 (23.53%)
    15 / 102 (14.71%)
         occurrences all number
    26
    15
    Upper respiratory tract infection
         subjects affected / exposed
    11 / 102 (10.78%)
    7 / 102 (6.86%)
         occurrences all number
    22
    13
    Nasopharyngitis
         subjects affected / exposed
    11 / 102 (10.78%)
    10 / 102 (9.80%)
         occurrences all number
    14
    16
    Urinary tract infection
         subjects affected / exposed
    2 / 102 (1.96%)
    6 / 102 (5.88%)
         occurrences all number
    3
    12

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    03 Mar 2021
    Protocol Amendment 1 (dated 03 Mar 2021) was primarily implemented to incorporate an additional study objective and endpoint on post vaccination biomarkers in study participants who had received a coronavirus disease 2019 (COVID-19) vaccine. Other required changes included aligning the protocol with the updates for the rozanolixizumab generalized myasthenia gravis (gMG) clinical program, and to incorporate specific local ethics committees and/or agency requirements into the global protocol.
    30 Jun 2022
    Protocol Amendment 2 (dated 30 Jun 2022) was primarily implemented to incorporate the option to administer investigational medicinal product (IMP) via manual push instead of using a syringe driver, implement changes specific to the Schedule of Activities, update the temporary investigational medicinal product (IMP) discontinuation criteria, and update the study medication permanent discontinuation criteria, as well as provide additional information on the benefit-risk for study participants who had received a full COVID-19 vaccination. Protocol Amendment 2 was approved and submitted to Food and Drug Administration (FDA) but was not implemented at the study sites.
    03 Oct 2022
    Protocol Amendment 3 (dated 03 Oct 2022) was primarily implemented to provide an update on the safety information in line with the updated Investigator’s Brochure dated Sep 2022 and to update the list of adverse events of special monitoring (adverse events of special monitoring [AESM]; remove severe gastrointestinal [GI] disorders [ie, abdominal pain, diarrhea, vomiting] and opportunistic infections and include serious headache and suspected aseptic meningitis). The possibility for participants receiving rescue therapy during the Observation Period to continue in the study was also added. The criteria for study medication discontinuation and the requirements for male contraception were also updated. Additional study assessments were specified in case of AESM, and a full neurological examination was added as part of the physical examination.

    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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    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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