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    Clinical Trial Results:
    A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects with Generalized Myasthenia Gravis

    Summary
    EudraCT number
    		 2019-001564-30
    Trial protocol
    		 DE   NO   GB   ES   IT  
    Global end of trial date
    30 Dec 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Jan 2023
    First version publication date
    04 Jan 2023
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    RA101495-02.301
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT04115293
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Ra Pharmaceuticals, Inc.
    Sponsor organisation address
    87 Cambridge Park Drive, Cambridge, Massachusetts, United States, 02140
    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
    18 Jan 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    30 Dec 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To confirm the efficacy, safety and tolerability of zilucoplan in subjects with 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
    17 Sep 2019
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Canada: 3
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 3
    Country: Number of subjects enrolled
    Italy: 4
    Country: Number of subjects enrolled
    Japan: 16
    Country: Number of subjects enrolled
    Norway: 6
    Country: Number of subjects enrolled
    Poland: 23
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 19
    Country: Number of subjects enrolled
    United States: 88
    Worldwide total number of subjects
    174
    EEA total number of subjects
    48
    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)
    126
    From 65 to 84 years
    48
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 The study started to enroll participants in September 2019 and concluded in December 2021.

    Pre-assignment
    Screening details
    		 The Participant flow refers to the Randomized Set.

    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
    		 Placebo
    Arm description
    		 Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants self-administered zilucoplan matching placebo as SC injection during 12-week Treatment Period or at pre-specified timepoints.

    Arm title
    		 Zilucoplan 0.3 mg/kg
    Arm description
    		 Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Zilucoplan
    Investigational medicinal product code
    Other name
    RA101495
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Participants self-administered zilucoplan 0.3 mg/kg SC injection during 12-week Treatment Period or at pre-specified timepoints.

    Number of subjects in period 1
    		Placebo Zilucoplan 0.3 mg/kg
    Started
    88
    86
    Completed
    84
    82
    Not completed
    4
    4
         Adverse event, serious fatal
    1
    1
         Consent withdrawn by subject
    2
    1
         Physician decision
    1
    -
         Adverse event, non-fatal
    -
    2

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.

    Reporting group title
    Zilucoplan 0.3 mg/kg
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.

    Reporting group values
    		 Placebo Zilucoplan 0.3 mg/kg Total
    Number of subjects
    		 88 86 174
    Age Categorical
    Units: participants
        <=18 years
    		 0 0 0
        Between 18 and 65 years
    		 62 64 126
        >=65 years
    		 26 22 48
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 53.3 ± 15.7 52.6 ± 14.6 -
    Sex: Female, Male
    Units: participants
        Female
    		 47 52 99
        Male
    		 41 34 75

    End points

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    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.

    Reporting group title
    Zilucoplan 0.3 mg/kg
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period.

    Primary: Change from Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score

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    End point title
    		 Change from Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score
    End point description
    The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Primary
    End point timeframe
    From Baseline to End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -2.30 (-3.17 to -1.43)
    -4.39 (-5.28 to -3.50)
    Statistical analysis title
    		 RA101495 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 MMRM ANCOVA
    Parameter type
    		 LS Mean Difference
    Point estimate
    -2.09
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -3.24
         upper limit
    		 -0.95

    Secondary: Change from Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score

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    End point title
    		 Change from Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score
    End point description
    The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    From Baseline to End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -3.25 (-4.32 to -2.17)
    -6.19 (-7.29 to -5.08)
    Statistical analysis title
    		 RA101495 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 MMRM ANCOVA
    Parameter type
    		 LS Mean Difference
    Point estimate
    -2.94
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -4.39
         upper limit
    		 -1.49

    Secondary: Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score

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    End point title
    		 Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score
    End point description
    The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    From Baseline to End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -5.42 (-6.98 to -3.86)
    -8.62 (-10.22 to -7.01)
    Statistical analysis title
    		 RA101495 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0023
    Method
    		 MMRM ANCOVA
    Parameter type
    		 LS Mean Difference
    Point estimate
    -3.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -5.24
         upper limit
    		 -1.16

    Secondary: Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QoL15r) Scale Total Score

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    End point title
    		 Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QoL15r) Scale Total Score
    End point description
    The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient’s life. A decrease from Baseline score indicated improvement. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    From Baseline to End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: score on a scale
        least squares mean (confidence interval 95%)
    -3.16 (-4.65 to -1.67)
    -5.65 (-7.17 to -4.12)
    Statistical analysis title
    		 RA101495 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0128
    Method
    		 MMRM ANCOVA
    Parameter type
    		 LS Mean Difference
    Point estimate
    -2.49
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -4.45
         upper limit
    		 -0.54

    Secondary: Time to first receipt of rescue therapy over the 12-week Treatment Period

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    End point title
    		 Time to first receipt of rescue therapy over the 12-week Treatment Period
    End point description
    Time to to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    From Baseline to End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88 [1]
    86 [2]
    Units: Days
        median (full range (min-max))
    99999 (99999 to 99999)
    99999 (99999 to 99999)
    Notes
    [1] - 99999: Time to first receipt of rescue therapy was not estimated due to less number of events.
    [2] - 99999: Time to first receipt of rescue therapy was not estimated due to less number of events.
    No statistical analyses for this end point

    Secondary: Percentage of participants achieving Minimal Symptom Expression (MSE) at Week 12 without rescue therapy

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    End point title
    		 Percentage of participants achieving Minimal Symptom Expression (MSE) at Week 12 without rescue therapy
    End point description
    Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the Missing at Random (MAR) assumption. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: percentage of participants
        number (not applicable)
    5.8
    14.0
    Statistical analysis title
    		 Zilucoplan 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0885
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.608
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.866
         upper limit
    		 7.86

    Secondary: Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy

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    End point title
    		 Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy
    End point description
    Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: percentage of participants
        number (not applicable)
    46.1
    73.1
    Statistical analysis title
    		 Zilucoplan 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    3.184
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.662
         upper limit
    		 6.101

    Secondary: Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12

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    End point title
    		 Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12
    End point description
    Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
    End point type
    Secondary
    End point timeframe
    End of Treatment (Week 12)
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: percentage of participants
        number (not applicable)
    33.0
    58.0
    Statistical analysis title
    		 Zilucoplan 0.3 mg/kg vs Placebo
    Comparison groups
    Placebo v Zilucoplan 0.3 mg/kg
    Number of subjects included in analysis
    174
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.0012
    Method
    		 Regression, Logistic
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.865
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.518
         upper limit
    		 5.409

    Secondary: 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)
    End point description
    A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs. The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received.
    End point type
    Secondary
    End point timeframe
    From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])
    End point values
    		 Placebo Zilucoplan 0.3 mg/kg
    Number of subjects analysed
    88
    86
    Units: percentage of participants
        number (not applicable)
    70.5
    76.7
    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) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up])
    Adverse event reporting additional description
    A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period.

    Reporting group title
    RA101495 0.3 mg/kg
    Reporting group description
    		 Participants self-administered zilucoplan (RA101495) 0.3 mg/kg/day SC injection during 12-week Treatment Period.

    Serious adverse events
    		 Placebo RA101495 0.3 mg/kg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    13 / 88 (14.77%)
    11 / 86 (12.79%)
         number of deaths (all causes)
    1
    1
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Lipase increased
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Metastases to meninges
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Basal cell carcinoma
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Cerebral haemorrhage
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cerebrovascular accident
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myasthenia gravis
         subjects affected / exposed
    5 / 88 (5.68%)
    2 / 86 (2.33%)
         occurrences causally related to treatment / all
    0 / 6
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Hyperemesis gravidarum
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (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
    Anaemia
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Aphthous ulcer
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         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
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Oesophageal candidiasis
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Oral candidiasis
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    2 / 88 (2.27%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Herpes simplex meningoencephalitis
         subjects affected / exposed
    1 / 88 (1.14%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    1 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Sepsis
         subjects affected / exposed
    0 / 88 (0.00%)
    1 / 86 (1.16%)
         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
    		 Placebo RA101495 0.3 mg/kg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    34 / 88 (38.64%)
    41 / 86 (47.67%)
    Investigations
    Lipase increased
         subjects affected / exposed
    1 / 88 (1.14%)
    6 / 86 (6.98%)
         occurrences all number
    1
    6
    Amylase increased
         subjects affected / exposed
    2 / 88 (2.27%)
    5 / 86 (5.81%)
         occurrences all number
    2
    5
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    3 / 88 (3.41%)
    7 / 86 (8.14%)
         occurrences all number
    3
    8
    Nervous system disorders
    Headache
         subjects affected / exposed
    14 / 88 (15.91%)
    13 / 86 (15.12%)
         occurrences all number
    19
    16
    Myasthenia gravis
         subjects affected / exposed
    5 / 88 (5.68%)
    8 / 86 (9.30%)
         occurrences all number
    6
    10
    General disorders and administration site conditions
    Injection site bruising
         subjects affected / exposed
    8 / 88 (9.09%)
    14 / 86 (16.28%)
         occurrences all number
    11
    18
    Injection site pain
         subjects affected / exposed
    3 / 88 (3.41%)
    8 / 86 (9.30%)
         occurrences all number
    3
    9
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    2 / 88 (2.27%)
    9 / 86 (10.47%)
         occurrences all number
    2
    9
    Vomiting
         subjects affected / exposed
    5 / 88 (5.68%)
    3 / 86 (3.49%)
         occurrences all number
    5
    3
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    5 / 88 (5.68%)
    3 / 86 (3.49%)
         occurrences all number
    9
    3
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    4 / 88 (4.55%)
    7 / 86 (8.14%)
         occurrences all number
    4
    7
    Nasopharyngitis
         subjects affected / exposed
    3 / 88 (3.41%)
    5 / 86 (5.81%)
         occurrences all number
    3
    5

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    18 Dec 2020
    Protocol Amendment Version 2.0 was dated 18 Dec 2020 and had incorporated all the country-specific amendments into single protocol amendment: • The total sample size was increased from 130 study participants (65 study participants per treatment group) to 156 study participants (78 study participants per treatment group). This increase was made to account for higher variability in the primary endpoint than originally assumed, and to maintain the power of the study. • An unblinded interim analysis was added to be performed after the final study participant had completed the Week 12 Visit, or after the final study participant had prematurely discontinued prior to reaching Week 12. The purpose of this interim analysis was to perform a comprehensive evaluation of all available double-blind data to prepare regulatory submissions for approval of the gMG target indication. • Changes made in earlier country-specific protocol amendments (France, Germany, Italy, Japan, Norway, United Kingdom) were consolidated into a single global protocol. • The objectives and endpoints were revised to reflect current UCB practices for the categorization and description of study objectives, estimands, and endpoints.
    18 Dec 2020
    Protocol amendment version 2.0 Continued: • The following provisions for the coronavirus disease 2019; disease caused by severe acute respiratory syndrome coronavirus 2 (COVID-19) pandemic were included: − In situations when the study participant could not return to the study site, the Investigator assessed the study participant’s safety by telephone/video contact. If the study participant was suitable for IMP continuation, the Investigator or designee assessed if the study participant agreed to provide name, address, telephone number, and email to the appointed courier. If the shipment was agreed, the Investigator or designee clearly explained to the study participant everything needed regarding the handling (in case of inconsistencies at delivery) and administration of the IMP and how to return all unused IMP to the study site at the next on-site visit. Additional details regarding the shipment of IMP and deviations to data collection are provided in the protocol. − Ad hoc study participant contact may have been warranted to understand the current health status of the study participants, to follow up on AEs, and to inform them of any protective measures taken by the clinical site as a result of the COVID-19 pandemic. − If a study participant needed to be discontinued from the study and could not come into the clinic, a visit was scheduled to perform final safety assessments as soon as possible. − If a study participant visited another facility for a medical issue, the Investigator was to request contact with the physician providing care to provide a detailed explanation of the study participant’s condition and his/her participation in the clinical study. Study participants and/or caregivers were reminded to completely collect and keep records of this visit. • Administrative updates.

    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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