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    Clinical Trial Results:
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Ravulizumab in Complement-Inhibitor-Naïve Adult Patients With Generalized Myasthenia Gravis

    Summary
    EudraCT number
    		 2018-003243-39
    Trial protocol
    		 DE   AT   NL   DK   ES   FR   CZ   BE   GB   PT   IT  
    Global end of trial date

    Results information
    Results version number
    		 v1(current)
    This version publication date
    03 Jun 2022
    First version publication date
    03 Jun 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    ALXN1210-MG-306
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03920293
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Inc.
    Sponsor organisation address
    100 College Street, New Haven, CT, United States, 06510
    Public contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 7 87148158, clinicaltrials.eu@alexion.com
    Scientific contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 7 87148158, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    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
    Interim
    Date of interim/final analysis
    30 Jun 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    11 May 2021
    Global end of trial reached?
    No
    General information about the trial
    Main objective of the trial
    The primary purpose of this study is to evaluate the safety and efficacy of ravulizumab for the treatment of participants with generalized myasthenia gravis (gMG).
    Protection of trial subjects
    This study was conducted in accordance with the protocol and with the following: • Consensus ethical principles derived from international guidelines including the Declaration of Helsinki and Council for International Organizations of Medical Sciences (CIOMS) International Ethical Guidelines • Applicable International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice (GCP) guidelines • Applicable laws and regulations
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    26 Mar 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: 6
    Country: Number of subjects enrolled
    Czechia: 8
    Country: Number of subjects enrolled
    Denmark: 8
    Country: Number of subjects enrolled
    France: 7
    Country: Number of subjects enrolled
    Germany: 14
    Country: Number of subjects enrolled
    Israel: 2
    Country: Number of subjects enrolled
    Italy: 13
    Country: Number of subjects enrolled
    Japan: 13
    Country: Number of subjects enrolled
    Netherlands: 4
    Country: Number of subjects enrolled
    Korea, Republic of: 18
    Country: Number of subjects enrolled
    Spain: 6
    Country: Number of subjects enrolled
    Switzerland: 2
    Country: Number of subjects enrolled
    United States: 74
    Worldwide total number of subjects
    175
    EEA total number of subjects
    60
    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)
    115
    From 65 to 84 years
    60
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Participants were randomized 1:1 to either the ravulizumab or placebo group during the Randomized-Controlled Period. Following the placebo-controlled part of the study, participants were transitioned to the ongoing Open-Label Extension Period to receive treatment with ravulizumab.

    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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Randomized-Controlled Period: Ravulizumab
    Arm description
    		 Participants received a weight-based single loading dose (2400 to 3000 milligrams [mg]) of ravulizumab intravenously (IV) on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 once every 8 weeks (q8w), during the 26-week Randomized-Controlled Period of the study.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Ravulizumab
    Investigational medicinal product code
    Other name
    ALXN1210 Ultomiris
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received ravulizumab at prespecified dose and timepoints.

    Arm title
    		 Randomized-Controlled Period: Placebo
    Arm description
    		 Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Participants received placebo at prespecified dose and timepoints.

    Number of subjects in period 1
    		Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Started
    86
    89
    Received at Least 1 Dose of Study Drug
    86
    89
    Completed
    79
    83
    Not completed
    7
    6
         Adverse event, serious fatal
    2
    -
         Consent withdrawn by subject
    2
    1
         Physician decision
    1
    2
         Adverse event, non-fatal
    -
    2
         Sponsor Decision
    -
    1
         Protocol deviation
    1
    -
         Noncompliance
    1
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Randomized-Controlled Period: Ravulizumab
    Reporting group description
    		 Participants received a weight-based single loading dose (2400 to 3000 milligrams [mg]) of ravulizumab intravenously (IV) on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 once every 8 weeks (q8w), during the 26-week Randomized-Controlled Period of the study.

    Reporting group title
    Randomized-Controlled Period: Placebo
    Reporting group description
    		 Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study.

    Reporting group values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo Total
    Number of subjects
    		 86 89 175
    Age categorical
    Units: Subjects
        In utero
        Preterm newborn infants (gestational age < 37 wks)
        Newborns (0-27 days)
        Infants and toddlers (28 days-23 months)
        Children (2-11 years)
        Adolescents (12-17 years)
        Adults (18-64 years)
        From 65-84 years
        85 years and over
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 58.0 ± 13.82 53.3 ± 16.05 -
    Sex: Female, Male
    Units: participants
        Female
    		 44 45 89
        Male
    		 42 44 86
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 2 5 7
        Not Hispanic or Latino
    		 79 78 157
        Unknown or Not Reported
    		 5 6 11
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    		 0 1 1
        Asian
    		 15 16 31
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Black or African American
    		 2 4 6
        White
    		 67 61 128
        Other
    		 0 1 1
        Unknown or Not Reported
    		 2 6 8

    End points

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    End points reporting groups
    Reporting group title
    Randomized-Controlled Period: Ravulizumab
    Reporting group description
    		 Participants received a weight-based single loading dose (2400 to 3000 milligrams [mg]) of ravulizumab intravenously (IV) on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 once every 8 weeks (q8w), during the 26-week Randomized-Controlled Period of the study.

    Reporting group title
    Randomized-Controlled Period: Placebo
    Reporting group description
    		 Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study.

    Primary: Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26

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    End point title
    		 Change From Baseline In Myasthenia Gravis-Activities Of Daily Living (MG-ADL) Total Score At Week 26
    End point description
    MG-ADL: 8-point questionnaire focusing on relevant symptoms/functional performance of activities of daily living in participants with MG. The 8 items of MGADL questionnaire derived from symptom-based components of original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response was graded 0 (normal)-3 (most severe). Range of total MG-ADL score: 0-24. Decrease in score indicated improvement. Estimates based on MMRM that included treatment group, stratification factor region, and MG-ADL total score at baseline, study visit, and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: units on a scale
        least squares mean (standard error)
    -3.1 ± 0.38
    -1.4 ± 0.37
    Statistical analysis title
    		 Ravulizumab versus Placebo
    Comparison groups
    Randomized-Controlled Period: Ravulizumab v Randomized-Controlled Period: Placebo
    Number of subjects included in analysis
    175
    Analysis specification
    Pre-specified
    Analysis type
    P-value
    		 = 0.0009 [1]
    Method
    		 MMRM
    Parameter type
    		 Least Square (LS) Mean Difference
    Point estimate
    -1.6
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -2.6
         upper limit
    		 -0.7
    Variability estimate
    Standard error of the mean
    Dispersion value
    0.49
    Notes
    [1] - Statistical significance was tested at α=0.05.

    Secondary: Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26

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    End point title
    		 Change From Baseline In The Quantitative Myasthenia Gravis (QMG) Total Score At Week 26
    End point description
    The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. The QMG scoring system was considered to be an objective evaluation of therapy for MG and was based on quantitative testing of sentinel muscle groups. A decrease in score indicated improvement. Estimates were based on MMRM that included treatment group, stratification factor region, and QMG total score at baseline, study visit, and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: units on a scale
        least squares mean (standard error)
    -2.8 ± 0.46
    -0.8 ± 0.45
    No statistical analyses for this end point

    Secondary: Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26

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    End point title
    		 Percentage of Participants With a Quantitative Myasthenia Gravis (QMG) Total Score Reduction of at Least 5 Points At Week 26
    End point description
    The QMG scoring system consisted of 13 items: ocular (2 items), facial (1 item), bulbar (2 items), gross motor (6 items), axial (1 item), and respiratory (1 item); each graded 0 to 3, with 3 being the most severe. The range of total QMG score is 0 to 39. A decrease in score indicated improvement. Percentage of participants with a ≥5-point reduction in the QMG total score are reported. Estimates were based on a generalized linear mixed model (GLMM) that included treatment group, stratification factor region and QMG total score at baseline, study visit and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: percentage of participants
        number (confidence interval 95%)
    30.0 (19.2 to 43.5)
    11.3 (5.6 to 21.5)
    No statistical analyses for this end point

    Secondary: Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26

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    End point title
    		 Change From Baseline In the Revised 15 Component Myasthenia Gravis Quality of Life (MG-QOL15r) At Week 26
    End point description
    The revised Myasthenia Gravis Qualify of Life 15-item scale (MG-QOL15r) is a health-related QoL evaluative instrument specific to participants with MG. MG-QOL15r was designed to provide information about participants’ perception of impairment and disability, determine the degree to which disease manifestations are tolerated, and to be administered and interpreted easily. Each item was graded on a scale of 0 to 2, with 2 being the most severe. The range of MG-QOL15r score is 0 to 30. Higher scores indicated greater extent of and dissatisfaction with MG-related dysfunction. Estimates are based on MMRM that included treatment group, stratification factor region and MG-QOL15r score at baseline, study visit and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: score on a scale
        least squares mean (standard error)
    -3.3 ± 0.71
    -1.6 ± 0.70
    No statistical analyses for this end point

    Secondary: Change from Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26

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    End point title
    		 Change from Baseline in Neurological Quality of Life (Neuro-QoL) Fatigue Score at Week 26
    End point description
    The Neuro-QOL Fatigue is a reliable and validated brief 19-item survey of fatigue, completed by the participant. Each items was rated on a scale of 1 to 5, with 5 being the most severe. The range of total score is 19 to 95. Higher scores indicated greater fatigue and greater impact of MG on activities. Estimates were based on MMRM that included treatment group, stratification factor region and Neuro-QoL Fatigue score at baseline, study visit, and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Baseline, Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: units on a scale
        least squares mean (standard error)
    -7.0 ± 1.92
    -4.8 ± 1.87
    No statistical analyses for this end point

    Secondary: Percentage of Participants with a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26

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    End point title
    		 Percentage of Participants with a Myasthenia Gravis Activities of Daily Living (MG-ADL) Total Score Reduction of at Least 3 Points At Week 26
    End point description
    MG-ADL is an 8-point questionnaire that focused on relevant symptoms and functional performance of activities of daily living in participants with MG. The 8 items of the MGADL questionnaire derived from symptom-based components of the original 13-item QMG scale to assess disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. In this functional status instrument, each response was graded 0 (normal)-3 (most severe). Range of total MG-ADL score was 0-24. A decrease in score indicated improvement. Percentage of participants with a ≥3-point reduction in the MG-ADL total score are reported. Estimates based on a GLMM that included treatment group, stratification factor region and MG-ADL total score at baseline, study visit and study visit by treatment group interaction. Full Analysis Set: All randomized participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    Week 26
    End point values
    		 Randomized-Controlled Period: Ravulizumab Randomized-Controlled Period: Placebo
    Number of subjects analysed
    86
    89
    Units: percentage of participants
        number (confidence interval 95%)
    56.7 (44.3 to 68.3)
    34.1 (23.8 to 46.1)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 (after dosing) through Week 26
    Adverse event reporting additional description
    Treatment-emergent adverse events reported during the 26-week randomized-controlled period of the study are presented. The Open-Label Extension Period is ongoing, and results will be presented when the study is completed.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Randomized-Controlled Period: Placebo
    Reporting group description
    		 Participants received a weight-based single loading dose of placebo IV on Day 1, followed by regular maintenance IV weight-based doses of placebo beginning on Day 15 q8w, during the 26-week Randomized-Controlled Period of the study. Following the placebo-controlled part of the study, participants were transitioned to the ongoing Open-Label Extension Period to receive treatment with ravulizumab.

    Reporting group title
    Randomized-Controlled Period: Ravulizumab
    Reporting group description
    		 Participants received a weight-based single loading dose (2400 to 3000 mg) of ravulizumab IV on Day 1, followed by regular maintenance IV weight-based doses (3000 to 3600 mg) of ravulizumab beginning on Day 15 q8w during the 26-week Randomized-Controlled Period of the study. Following the placebo-controlled part of the study, participants were transitioned to the ongoing Open-Label Extension Period to receive treatment with ravulizumab.

    Serious adverse events
    		 Randomized-Controlled Period: Placebo Randomized-Controlled Period: Ravulizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    14 / 89 (15.73%)
    20 / 86 (23.26%)
         number of deaths (all causes)
    0
    2
         number of deaths resulting from adverse events
    0
    2
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Squamous cell carcinoma of skin
         subjects affected / exposed
    0 / 89 (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
    Ureteral neoplasm
         subjects affected / exposed
    0 / 89 (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
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    0 / 89 (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
    General physical health deterioration
         subjects affected / exposed
    0 / 89 (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
    Non-cardiac chest pain
         subjects affected / exposed
    0 / 89 (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
    Respiratory, thoracic and mediastinal disorders
    Dyspnoea
         subjects affected / exposed
    0 / 89 (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
    Lung infiltration
         subjects affected / exposed
    0 / 89 (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
    Dyspnoea exertional
         subjects affected / exposed
    0 / 89 (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
    Psychiatric disorders
    Suicide attempt
         subjects affected / exposed
    0 / 89 (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
    Injury, poisoning and procedural complications
    Multiple fractures
         subjects affected / exposed
    0 / 89 (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
    Infusion-related reaction
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Congestive cardiomyopathy
         subjects affected / exposed
    0 / 89 (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
    Transient ischaemic attack
         subjects affected / exposed
    0 / 89 (0.00%)
    2 / 86 (2.33%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Myasthenia gravis crisis
         subjects affected / exposed
    0 / 89 (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
    Cerebral haemorrhage
         subjects affected / exposed
    0 / 89 (0.00%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Syncope
         subjects affected / exposed
    0 / 89 (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
    Facial paresis
         subjects affected / exposed
    1 / 89 (1.12%)
    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
    3 / 89 (3.37%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Trigeminal neuralgia
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Eye disorders
    Visual impairment
         subjects affected / exposed
    0 / 89 (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
    Dysphagia
         subjects affected / exposed
    0 / 89 (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
    Enteritis
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nausea
         subjects affected / exposed
    0 / 89 (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
    Granuloma skin
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Renal failure
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nephrotic syndrome
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Intervertebral disc protrusion
         subjects affected / exposed
    0 / 89 (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
    Nodal osteoarthritis
         subjects affected / exposed
    0 / 89 (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
    Tendonitis
         subjects affected / exposed
    0 / 89 (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
    Spinal stenosis
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    COVID-19 pneumonia
         subjects affected / exposed
    0 / 89 (0.00%)
    2 / 86 (2.33%)
         occurrences causally related to treatment / all
    0 / 0
    2 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 1
    Diverticulitis
         subjects affected / exposed
    0 / 89 (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
    Arthritis bacterial
         subjects affected / exposed
    0 / 89 (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
    Gastroenteritis viral
         subjects affected / exposed
    0 / 89 (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
    Gangrene
         subjects affected / exposed
    0 / 89 (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
    Pneumonia respiratory syncytial viral
         subjects affected / exposed
    0 / 89 (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
    Infected skin ulcer
         subjects affected / exposed
    0 / 89 (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 zoster
         subjects affected / exposed
    1 / 89 (1.12%)
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Staphylococcal sepsis
         subjects affected / exposed
    0 / 89 (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
    COVID-19
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cellulitis
         subjects affected / exposed
    2 / 89 (2.25%)
    0 / 86 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Steroid diabetes
         subjects affected / exposed
    0 / 89 (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
    Diabetic ketoacidosis
         subjects affected / exposed
    1 / 89 (1.12%)
    0 / 86 (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
    		 Randomized-Controlled Period: Placebo Randomized-Controlled Period: Ravulizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    75 / 89 (84.27%)
    77 / 86 (89.53%)
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 89 (3.37%)
    8 / 86 (9.30%)
         occurrences all number
    3
    9
    Headache
         subjects affected / exposed
    23 / 89 (25.84%)
    16 / 86 (18.60%)
         occurrences all number
    27
    19
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    5 / 89 (5.62%)
    1 / 86 (1.16%)
         occurrences all number
    6
    1
    Fatigue
         subjects affected / exposed
    6 / 89 (6.74%)
    6 / 86 (6.98%)
         occurrences all number
    6
    7
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    11 / 89 (12.36%)
    13 / 86 (15.12%)
         occurrences all number
    15
    14
    Abdominal pain
         subjects affected / exposed
    0 / 89 (0.00%)
    5 / 86 (5.81%)
         occurrences all number
    0
    6
    Nausea
         subjects affected / exposed
    9 / 89 (10.11%)
    9 / 86 (10.47%)
         occurrences all number
    10
    12
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    5 / 89 (5.62%)
    7 / 86 (8.14%)
         occurrences all number
    5
    7
    Arthralgia
         subjects affected / exposed
    7 / 89 (7.87%)
    6 / 86 (6.98%)
         occurrences all number
    8
    8
    Infections and infestations
    COVID-19
         subjects affected / exposed
    3 / 89 (3.37%)
    5 / 86 (5.81%)
         occurrences all number
    3
    5
    Urinary tract infection
         subjects affected / exposed
    4 / 89 (4.49%)
    5 / 86 (5.81%)
         occurrences all number
    5
    7
    Nasopharyngitis
         subjects affected / exposed
    5 / 89 (5.62%)
    3 / 86 (3.49%)
         occurrences all number
    7
    3

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    11 Dec 2018
    The purpose of this amendment was to change duration of safety follow-up after last dose; add additional details on assessments, align pregnancy and clinical laboratory testing frequency with infusions; change supplemental dosing recommendations and sample collection when rescue therapy is provided; and update adverse event and pregnancy/contraception language.
    25 Oct 2019
    The purpose of this amendment was to revise secondary and exploratory endpoints, to decrease burden to participants by reduction in assessment and visit frequency, to provide additional guidance for supplemental dosing, and to clarify minor operational aspects of the protocol.

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    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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