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    Clinical Trial Results:
    A Phase III, Open-Label, Extension Trial of ECU-NMO-301 To Evaluate The Safety And Efficacy of Eculizumab in Patients With Relapsing Neuromyelitis Optica (NMO)

    Summary
    EudraCT number
    		 2013-001151-12
    Trial protocol
    		 DE   IT   GB   ES   AT   CZ   DK   FR   HR  
    Global end of trial date
    12 Jul 2021

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

    Trial information

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    Trial identification
    Sponsor protocol code
    ECU-NMO-302
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02003144
    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 787148158, clinicaltrials.eu@alexion.com
    Scientific contact
    Alexion Europe SAS European Clinical Trial Information, Alexion Pharmaceuticals Inc., +33 787148158, clinicaltrials.eu@alexion.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    06 Oct 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Jul 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Jul 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The purpose of this study is to determine whether eculizumab long-term use is safe and effective in participants with relapsing NMO.
    Protection of trial subjects
    This study was conducted in accordance with International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Good Clinical Practice, and the principles of the Declaration of Helsinki, in addition to following the laws and regulations of the country or countries in which a study is conducted.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    12 Jan 2015
    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
    Argentina: 6
    Country: Number of subjects enrolled
    Australia: 2
    Country: Number of subjects enrolled
    Czechia: 1
    Country: Number of subjects enrolled
    Germany: 5
    Country: Number of subjects enrolled
    Denmark: 2
    Country: Number of subjects enrolled
    Spain: 5
    Country: Number of subjects enrolled
    United Kingdom: 2
    Country: Number of subjects enrolled
    Hong Kong: 1
    Country: Number of subjects enrolled
    Croatia: 3
    Country: Number of subjects enrolled
    Italy: 5
    Country: Number of subjects enrolled
    Japan: 12
    Country: Number of subjects enrolled
    Korea, Republic of: 10
    Country: Number of subjects enrolled
    Malaysia: 4
    Country: Number of subjects enrolled
    Russian Federation: 11
    Country: Number of subjects enrolled
    Thailand: 7
    Country: Number of subjects enrolled
    Turkey: 10
    Country: Number of subjects enrolled
    Taiwan: 5
    Country: Number of subjects enrolled
    United States: 28
    Worldwide total number of subjects
    119
    EEA total number of subjects
    21
    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)
    108
    From 65 to 84 years
    11
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Participants who completed ECU-NMO-301 (NCT01892345) were eligible to participate in ECU-NMO-302. This is an open-label study in which all participants were administered intravenous eculizumab. However, to maintain the blind of ECU-NMO-301, all participants underwent a 4-week Blind Induction Phase before entering the Open-label Maintenance Phase.

    Period 1
    Period 1 title
    Blind Induction Phase
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo/Eculizumab
    Arm description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
    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 matched to eculizumab at prespecified dose and timepoints.

    Arm title
    		 Eculizumab/Eculizumab
    Arm description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
    Arm type
    		 Experimental

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

    Number of subjects in period 1
    		Placebo/Eculizumab Eculizumab/Eculizumab
    Started
    41
    78
    Received at Least 1 Dose of Study Drug
    41
    78
    Completed
    41
    78
    Period 2
    Period 2 title
    Open Label Maintenance Phase
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo/Eculizumab
    Arm description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
    Arm type
    		 Experimental

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

    Arm title
    		 Eculizumab/Eculizumab
    Arm description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
    Arm type
    		 Experimental

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

    Number of subjects in period 2
    		Placebo/Eculizumab Eculizumab/Eculizumab
    Started
    41
    78
    Received at least 1 dose of study drug
    41
    78
    Completed
    32
    64
    Not completed
    9
    14
         Consent withdrawn by subject
    4
    5
         Physician decision
    1
    1
         Adverse event, non-fatal
    2
    1
         Pregnancy
    -
    2
         Other than specified
    2
    4
         Lost to follow-up
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Placebo/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group title
    Eculizumab/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group values
    		 Placebo/Eculizumab Eculizumab/Eculizumab Total
    Number of subjects
    		 41 78 119
    Age categorical
    Units: Subjects
        In utero
    		 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0 0
        Newborns (0-27 days)
    		 0 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0 0
        Children (2-11 years)
    		 0 0 0
        Adolescents (12-17 years)
    		 0 0 0
        Adults (18-64 years)
    		 37 71 108
        From 65-84 years
    		 4 7 11
        85 years and over
    		 0 0 0
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 46.0 ( 13.82 ) 46.6 ( 13.77 ) -
    Sex: Female, Male
    Units: participants
        Female
    		 36 74 110
        Male
    		 5 4 9
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    		 2 11 13
        Not Hispanic or Latino
    		 37 63 100
        Unknown or Not Reported
    		 2 4 6
    Race/Ethnicity, Customized
    Units: Subjects
        American Indian or Alaska Native
    		 0 1 1
        Asian
    		 13 32 45
        Black or African American
    		 7 3 10
        White
    		 21 40 61
        Other
    		 0 1 1
        Unknown or Not Reported
    		 0 1 1

    End points

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    End points reporting groups
    Reporting group title
    Placebo/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group title
    Eculizumab/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.
    Reporting group title
    Placebo/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 milligrams [mg]) plus matching placebo via intravenous (IV) infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group title
    Eculizumab/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [1]
    End point description
    An AE was defined as any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. TEAEs were defined as an AE with onset on or after the first study drug dose in Study ECU-NMO-302. A SAE was defined as an untoward medical occurrence that at any dose either results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or is an important medical event. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302.
    End point type
    Primary
    End point timeframe
    Baseline up to end of study (up to 6.5 years)
    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: Only descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: participants
        TEAEs
    41
    70
        SAEs
    14
    26
    No statistical analyses for this end point

    Primary: Percentage of Participants With At Least 1 Post Baseline C-SSRS Assessment (Suicide-Related Thoughts or Behaviours) Abnormality

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    End point title
    		 Percentage of Participants With At Least 1 Post Baseline C-SSRS Assessment (Suicide-Related Thoughts or Behaviours) Abnormality [2]
    End point description
    The C-SSRS is a validated questionnaire to capture occurrence, severity, and frequency of suicide-related thoughts and behaviours, and has a binary response (yes/no). Suicidal Ideation: a "yes" answer to any one of 5 suicidal ideation questions: Wish to be Dead; Non-specific Active Suicidal Thoughts; Active Suicidal Ideation with Any Methods (Not Planned) without Intent to Act; Active Suicidal Ideation with Some Intent to Act, without Specific Plan; and Active Suicidal Ideation with Specific Plan and Intent. Suicidal Behaviour: a "yes" answer to any of 5 suicidal behaviour questions: Preparatory Acts or Behaviour, Aborted Attempt, Interrupted Attempt, Actual Attempt (non-fatal), and Completed Suicide. Suicidal Ideation or Behaviour: a "yes" answer to the following question: Self-injurious behaviour without suicidal intent. The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302.
    End point type
    Primary
    End point timeframe
    Baseline up to end of study (up to 6.5 years)
    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: Only descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: percentage of participants
    number (not applicable)
        Suicidal Ideation
    9.8
    6.4
        Suicidal Behavior
    0.0
    1.3
        Suicidal Ideation or Behavior
    9.8
    6.4
    No statistical analyses for this end point

    Primary: On-Trial Annualized Relapse Rate (ARR) as Determined by the Treating Physician

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    End point title
    		 On-Trial Annualized Relapse Rate (ARR) as Determined by the Treating Physician [3]
    End point description
    The On-trial ARR was computed as the total number of relapses divided by the total number of participant years in the study period. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to end of study (up to 6.5 years)
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: relapses/years on study
        arithmetic mean (standard deviation)
    0.128 ( 0.4576 )
    0.061 ( 0.2186 )
    No statistical analyses for this end point

    Primary: Number of Participants With An On-trial Relapse as Determined by the Treating Physician

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    End point title
    		 Number of Participants With An On-trial Relapse as Determined by the Treating Physician [4]
    End point description
    An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined as an On-trial Relapse that was positively adjudicated by the relapse adjudication committee. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment.
    End point type
    Primary
    End point timeframe
    Baseline up to end of study (up to 6.5 years)
    Notes
    [4] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: Only descriptive analysis was planned to be reported for this endpoint.
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: participants
    5
    8
    No statistical analyses for this end point

    Secondary: Change From Baseline in Expanded Disability Status Scale (EDSS) score

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    End point title
    		 Change From Baseline in Expanded Disability Status Scale (EDSS) score
    End point description
    Disease-related disability was measured by the EDSS. The EDSS quantifies disability in 8 Functional Systems (FS) and allows neurologists to assign a Functional System Score (FSS) in each of these. The Functional Systems are pyramidal, cerebellar, brainstem, sensory, bowel and bladder, visual, cerebral, and other. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 52, 104 and 156
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=41, 78)
    4.34 ( 1.879 )
    3.97 ( 1.736 )
        Change from Baseline at Week 52 (n=35, 73)
    -0.24 ( 0.721 )
    0.01 ( 0.571 )
        Change from Baseline at Week 104 (n=22, 36)
    -0.39 ( 0.830 )
    -0.11 ( 0.536 )
        Change from Baseline at Week 156 (n=13, 16)
    -0.38 ( 1.003 )
    -0.38 ( 1.057 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Modified Rankin Scale (mRS) Score

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    End point title
    		 Change From Baseline in Modified Rankin Scale (mRS) Score
    End point description
    Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no symptoms at all) to 6 (death) in whole-point increments. A decrease in score indicates improvement. Baseline was defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 52, 104 and 156
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=41, 78)
    2.39 ( 1.358 )
    1.88 ( 1.269 )
        Change from Baseline at Week 52 (n=37, 72)
    -0.27 ( 0.932 )
    -0.04 ( 0.458 )
        Change from Baseline at Week 104 (n=22, 36)
    -0.41 ( 1.182 )
    -0.14 ( 0.543 )
        Change from Baseline at Week 156 (n=13, 16)
    -0.62 ( 1.446 )
    -0.31 ( 0.602 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function

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    End point title
    		 Change from Baseline in Hauser Ambulation Index (HAI) in Participants With Abnormal Baseline Ambulatory Function
    End point description
    The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully active) and 9 being the worst (restricted to wheelchair; unable to transfer self independently). A decrease in score indicates improvement. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 52, 104 and 156
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    40
    66
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=40, 66)
    2.83 ( 2.123 )
    2.35 ( 2.257 )
        Change from Baseline at Week 52 (n=36, 59)
    -0.44 ( 1.132 )
    0.08 ( 0.816 )
        Change from Baseline at Week 104 (n=21, 29)
    -0.57 ( 1.777 )
    0.07 ( 1.033 )
        Change from Baseline at Week 156 (n=13, 14)
    -1.08 ( 1.706 )
    0.07 ( 1.269 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Questionnaire (EQ-5D) Health Status Score

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    End point title
    		 Change From Baseline in European Quality of Life (EuroQoL) 5-Dimension Questionnaire (EQ-5D) Health Status Score
    End point description
    The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. The EQ-5D comprises 5 dimensions of health: mobility, pain/discomfort, anxiety/depression, and overall health (each dimension consists of 3 levels ranging from no problems to extreme problems) and self-care (ranging of 6 levels ranging from no problems to extreme problems). From these scores, a summary index score is derived ranging from less than 0 to 1, with higher scores representing a better health status. Baseline is defined as the last available assessment prior to the first study drug infusion in Study ECU-NMO-302. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number Analyzed signifies those participants who were evaluable at specified time points.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 52, 104 and 156
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    41
    78
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=41, 78)
    62.00 ( 22.012 )
    72.27 ( 20.941 )
        Change from Baseline at Week 52 (n=37, 73)
    2.22 ( 13.294 )
    -0.78 ( 12.388 )
        Change from Baseline at Week 104 (n=22, 36)
    0.05 ( 18.867 )
    1.28 ( 11.295 )
        Change from Baseline at Week 156 (n=13, 16)
    11.00 ( 19.374 )
    -4.13 ( 18.421 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function

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    End point title
    		 Change From Baseline in Kurtzke Visual Functional System Scores (FSS) in Participants With Abnormal Baseline Visual Function
    End point description
    The EDSS assesses multiple Kurtzke functional systems in the context of a standard neurological exam, including visual function. The visual score ranges from 0 to 6. A score of 0 implies the participant has normal visual function. Higher scores represent worse disability. Baseline is defined as the last available assessment prior to the first study drug infusion in Study EC-NMO-302. The Extension Full Analysis Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302 and had a post-IP-infusion efficacy assessment. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure and Number Analyzed signifies those participants who were evaluable at specified time points.
    End point type
    Secondary
    End point timeframe
    Baseline, Weeks 52, 104 and 156
    End point values
    		 Placebo/Eculizumab Eculizumab/Eculizumab
    Number of subjects analysed
    28
    67
    Units: units on a scale
    arithmetic mean (standard deviation)
        Baseline (n=28, 67)
    3.75 ( 2.030 )
    3.60 ( 2.031 )
        Change from Baseline at Week 52 (n=26, 62)
    -0.08 ( 0.392 )
    -0.06 ( 0.569 )
        Change from Baseline at Week 104 (n=15, 31)
    -0.13 ( 0.352 )
    -0.10 ( 0.651 )
        Change from Baseline at Week 156 (n=11, 14)
    0.00 ( 0.000 )
    -0.29 ( 0.994 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Baseline up to end of study (up to 6.5 years)
    Adverse event reporting additional description
    The Extension Safety Set consisted of all participants who had received at least 1 dose of eculizumab in Study ECU-NMO-302.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.0
    Reporting groups
    Reporting group title
    Placebo/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-NMO-301 were administered eculizumab (900 mg) plus matching placebo via IV infusion on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group title
    Eculizumab (Combined Total)
    Reporting group description
    		 All participants who received at least 1 dose of eculizumab in the extension study. Participants received open-label eculizumab (1200 mg) every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Reporting group title
    Eculizumab/Eculizumab
    Reporting group description
    		 Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-NMO-301 were administered eculizumab (1200 mg) via IV infusion on Day 1 and Week 2 and placebo at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (1200 mg) via IV infusion every 2 weeks starting at Week 4 and continued for up to 6.5 years.

    Serious adverse events
    		 Placebo/Eculizumab Eculizumab (Combined Total) Eculizumab/Eculizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    14 / 41 (34.15%)
    40 / 119 (33.61%)
    26 / 78 (33.33%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acrochordon
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cholesteatoma
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Vascular disorders
    Venous thrombosis limb
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Anembryonic gestation
         subjects affected / exposed [1]
    0 / 36 (0.00%)
    1 / 110 (0.91%)
    1 / 74 (1.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Chest pain
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Oedema peripheral
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Reproductive system and breast disorders
    Endometriosis
    Additional description: The N for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event.
         subjects affected / exposed [2]
    0 / 36 (0.00%)
    1 / 110 (0.91%)
    1 / 74 (1.35%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Bronchiectasis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Chronic obstructive pulmonary disease
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nasal septum deviation
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psychiatric disorders
    Catatonia
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Delirium
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Investigations
    Blood pressure increased
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Femoral neck fracture
         subjects affected / exposed
    1 / 41 (2.44%)
    2 / 119 (1.68%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Clavicle fracture
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Head injury
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Spinal compression fracture
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Wrist fracture
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Cardiac disorders
    Ventricular fibrillation
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Nervous system disorders
    Neuromyelitis optica spectrum disorder
         subjects affected / exposed
    2 / 41 (4.88%)
    5 / 119 (4.20%)
    3 / 78 (3.85%)
         occurrences causally related to treatment / all
    1 / 4
    2 / 8
    1 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Optic neuritis
         subjects affected / exposed
    1 / 41 (2.44%)
    3 / 119 (2.52%)
    2 / 78 (2.56%)
         occurrences causally related to treatment / all
    1 / 1
    2 / 3
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Aphasia
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hemiparaesthesia
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Mental impairment
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Muscle spasticity
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Neuromuscular blockade
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Neuromyelitis optica pseudo relapse
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Eye disorders
    Glaucoma
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Irritable bowel syndrome
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis acute
         subjects affected / exposed
    2 / 41 (4.88%)
    3 / 119 (2.52%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 3
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Renal and urinary disorders
    Urinary retention
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Musculoskeletal and connective tissue disorders
    Intervertebral disc protrusion
         subjects affected / exposed
    0 / 41 (0.00%)
    2 / 119 (1.68%)
    2 / 78 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Osteonecrosis
         subjects affected / exposed
    1 / 41 (2.44%)
    2 / 119 (1.68%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Muscular weakness
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pain in extremity
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Spondylolisthesis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Systemic lupus erythematosus
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Tenosynovitis
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Urinary tract infection
         subjects affected / exposed
    2 / 41 (4.88%)
    5 / 119 (4.20%)
    3 / 78 (3.85%)
         occurrences causally related to treatment / all
    1 / 2
    1 / 5
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 41 (2.44%)
    2 / 119 (1.68%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 2
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pyelonephritis acute
         subjects affected / exposed
    0 / 41 (0.00%)
    2 / 119 (1.68%)
    2 / 78 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 2
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urosepsis
         subjects affected / exposed
    0 / 41 (0.00%)
    2 / 119 (1.68%)
    2 / 78 (2.56%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Abscess limb
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Bacterial sepsis
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    COVID-19 pneumonia
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Device related infection
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Escherichia pyelonephritis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gonorrhoea
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infective tenosynovitis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Osteomyelitis
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia bacterial
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia influenzal
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Psoas abscess
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Staphylococcal bacteraemia
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Streptococcal infection
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Urinary tract infection enterococcal
         subjects affected / exposed
    1 / 41 (2.44%)
    1 / 119 (0.84%)
    0 / 78 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hypophosphataemia
         subjects affected / exposed
    0 / 41 (0.00%)
    1 / 119 (0.84%)
    1 / 78 (1.28%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Notes
    [1] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed to this adverse event. These numbers are expected to be equal.
    Justification: The number of subjects exposed for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event.
    [2] - The number of subjects exposed to this adverse event is less than the total number of subjects exposed to this adverse event. These numbers are expected to be equal.
    Justification: The number of subjects exposed for this adverse event has been adjusted to the number of females in the study as it is a sex-specific event.
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Placebo/Eculizumab Eculizumab (Combined Total) Eculizumab/Eculizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    40 / 41 (97.56%)
    110 / 119 (92.44%)
    70 / 78 (89.74%)
    Investigations
    Blood pressure increased
         subjects affected / exposed
    3 / 41 (7.32%)
    3 / 119 (2.52%)
    0 / 78 (0.00%)
         occurrences all number
    3
    3
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    6 / 41 (14.63%)
    10 / 119 (8.40%)
    4 / 78 (5.13%)
         occurrences all number
    10
    16
    6
    Fall
         subjects affected / exposed
    3 / 41 (7.32%)
    3 / 119 (2.52%)
    0 / 78 (0.00%)
         occurrences all number
    14
    14
    0
    Thermal burn
         subjects affected / exposed
    3 / 41 (7.32%)
    6 / 119 (5.04%)
    3 / 78 (3.85%)
         occurrences all number
    8
    11
    3
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    3 / 41 (7.32%)
    8 / 119 (6.72%)
    5 / 78 (6.41%)
         occurrences all number
    3
    10
    7
    Headache
         subjects affected / exposed
    12 / 41 (29.27%)
    27 / 119 (22.69%)
    15 / 78 (19.23%)
         occurrences all number
    34
    174
    140
    Hypoaesthesia
         subjects affected / exposed
    2 / 41 (4.88%)
    7 / 119 (5.88%)
    5 / 78 (6.41%)
         occurrences all number
    3
    9
    6
    Paraesthesia
         subjects affected / exposed
    3 / 41 (7.32%)
    6 / 119 (5.04%)
    3 / 78 (3.85%)
         occurrences all number
    3
    7
    4
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    3 / 41 (7.32%)
    9 / 119 (7.56%)
    6 / 78 (7.69%)
         occurrences all number
    3
    10
    7
    Iron deficiency anaemia
         subjects affected / exposed
    2 / 41 (4.88%)
    7 / 119 (5.88%)
    5 / 78 (6.41%)
         occurrences all number
    2
    7
    5
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    6 / 41 (14.63%)
    18 / 119 (15.13%)
    12 / 78 (15.38%)
         occurrences all number
    8
    27
    19
    Fatigue
         subjects affected / exposed
    4 / 41 (9.76%)
    11 / 119 (9.24%)
    7 / 78 (8.97%)
         occurrences all number
    13
    20
    7
    Oedema peripheral
         subjects affected / exposed
    1 / 41 (2.44%)
    5 / 119 (4.20%)
    4 / 78 (5.13%)
         occurrences all number
    1
    5
    4
    Peripheral swelling
         subjects affected / exposed
    3 / 41 (7.32%)
    5 / 119 (4.20%)
    2 / 78 (2.56%)
         occurrences all number
    4
    6
    2
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    8 / 41 (19.51%)
    14 / 119 (11.76%)
    6 / 78 (7.69%)
         occurrences all number
    17
    28
    11
    Dyspepsia
         subjects affected / exposed
    4 / 41 (9.76%)
    6 / 119 (5.04%)
    2 / 78 (2.56%)
         occurrences all number
    5
    14
    9
    Constipation
         subjects affected / exposed
    4 / 41 (9.76%)
    10 / 119 (8.40%)
    6 / 78 (7.69%)
         occurrences all number
    5
    12
    7
    Nausea
         subjects affected / exposed
    7 / 41 (17.07%)
    10 / 119 (8.40%)
    3 / 78 (3.85%)
         occurrences all number
    17
    21
    4
    Toothache
         subjects affected / exposed
    5 / 41 (12.20%)
    6 / 119 (5.04%)
    1 / 78 (1.28%)
         occurrences all number
    7
    8
    1
    Dental caries
         subjects affected / exposed
    1 / 41 (2.44%)
    5 / 119 (4.20%)
    4 / 78 (5.13%)
         occurrences all number
    1
    6
    5
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    7 / 41 (17.07%)
    12 / 119 (10.08%)
    5 / 78 (6.41%)
         occurrences all number
    10
    19
    9
    Oropharyngeal pain
         subjects affected / exposed
    4 / 41 (9.76%)
    9 / 119 (7.56%)
    5 / 78 (6.41%)
         occurrences all number
    4
    13
    9
    Asthma
         subjects affected / exposed
    3 / 41 (7.32%)
    5 / 119 (4.20%)
    2 / 78 (2.56%)
         occurrences all number
    5
    7
    2
    Rhinorrhoea
         subjects affected / exposed
    2 / 41 (4.88%)
    6 / 119 (5.04%)
    4 / 78 (5.13%)
         occurrences all number
    2
    7
    5
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 41 (4.88%)
    6 / 119 (5.04%)
    4 / 78 (5.13%)
         occurrences all number
    3
    8
    5
    Dermatitis
         subjects affected / exposed
    3 / 41 (7.32%)
    3 / 119 (2.52%)
    0 / 78 (0.00%)
         occurrences all number
    3
    3
    0
    Skin lesion
         subjects affected / exposed
    3 / 41 (7.32%)
    3 / 119 (2.52%)
    0 / 78 (0.00%)
         occurrences all number
    4
    4
    0
    Renal and urinary disorders
    Urinary incontinence
         subjects affected / exposed
    3 / 41 (7.32%)
    4 / 119 (3.36%)
    1 / 78 (1.28%)
         occurrences all number
    3
    4
    1
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    3 / 41 (7.32%)
    6 / 119 (5.04%)
    3 / 78 (3.85%)
         occurrences all number
    4
    8
    4
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    8 / 41 (19.51%)
    23 / 119 (19.33%)
    15 / 78 (19.23%)
         occurrences all number
    13
    38
    25
    Back pain
         subjects affected / exposed
    5 / 41 (12.20%)
    16 / 119 (13.45%)
    11 / 78 (14.10%)
         occurrences all number
    9
    33
    24
    Pain in extremity
         subjects affected / exposed
    5 / 41 (12.20%)
    12 / 119 (10.08%)
    7 / 78 (8.97%)
         occurrences all number
    13
    35
    22
    Muscular weakness
         subjects affected / exposed
    1 / 41 (2.44%)
    5 / 119 (4.20%)
    4 / 78 (5.13%)
         occurrences all number
    1
    5
    4
    Muscle spasms
         subjects affected / exposed
    5 / 41 (12.20%)
    10 / 119 (8.40%)
    5 / 78 (6.41%)
         occurrences all number
    7
    13
    6
    Musculoskeletal pain
         subjects affected / exposed
    0 / 41 (0.00%)
    5 / 119 (4.20%)
    5 / 78 (6.41%)
         occurrences all number
    0
    5
    5
    Infections and infestations
    Upper respiratory tract infection
         subjects affected / exposed
    11 / 41 (26.83%)
    28 / 119 (23.53%)
    17 / 78 (21.79%)
         occurrences all number
    24
    51
    27
    Nasopharyngitis
         subjects affected / exposed
    16 / 41 (39.02%)
    29 / 119 (24.37%)
    13 / 78 (16.67%)
         occurrences all number
    35
    67
    32
    Influenza
         subjects affected / exposed
    9 / 41 (21.95%)
    19 / 119 (15.97%)
    10 / 78 (12.82%)
         occurrences all number
    14
    25
    11
    Urinary tract infection
         subjects affected / exposed
    11 / 41 (26.83%)
    24 / 119 (20.17%)
    13 / 78 (16.67%)
         occurrences all number
    29
    59
    30
    Cystitis
         subjects affected / exposed
    4 / 41 (9.76%)
    9 / 119 (7.56%)
    5 / 78 (6.41%)
         occurrences all number
    6
    12
    6
    Oral herpes
         subjects affected / exposed
    4 / 41 (9.76%)
    8 / 119 (6.72%)
    4 / 78 (5.13%)
         occurrences all number
    17
    21
    4
    Bronchitis
         subjects affected / exposed
    3 / 41 (7.32%)
    7 / 119 (5.88%)
    4 / 78 (5.13%)
         occurrences all number
    6
    12
    6
    Herpes zoster
         subjects affected / exposed
    3 / 41 (7.32%)
    5 / 119 (4.20%)
    2 / 78 (2.56%)
         occurrences all number
    3
    5
    2
    Pneumonia
         subjects affected / exposed
    3 / 41 (7.32%)
    4 / 119 (3.36%)
    1 / 78 (1.28%)
         occurrences all number
    4
    6
    2
    Periodontitis
         subjects affected / exposed
    0 / 41 (0.00%)
    4 / 119 (3.36%)
    4 / 78 (5.13%)
         occurrences all number
    0
    5
    5
    Pharyngitis
         subjects affected / exposed
    4 / 41 (9.76%)
    5 / 119 (4.20%)
    1 / 78 (1.28%)
         occurrences all number
    4
    5
    1
    Sinusitis
         subjects affected / exposed
    3 / 41 (7.32%)
    4 / 119 (3.36%)
    1 / 78 (1.28%)
         occurrences all number
    4
    8
    4

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    25 Oct 2013
    Incorporated changes in the amended protocol for Study ECU-NMO-301 version 4.0 dated 16 Oct 2013, which addressed issues/concerns from the Investigators as well as recommended changes from Institutional Review Board, Independent Ethics Committee, and Competent Authorities.
    01 Jun 2015
    • Aligned with amended protocol for Study ECU-NMO-301 version 5.0 dated 25 Feb 2015: Allowed qualified non-physician healthcare professionals to conduct the EDSS rating Expanded the number of investigational sites Extended the estimated date of first participant enrolled from 2014 to 2015 and estimated date of last participant completed from 2018 to 2019 Provided flexibility for the supplemental investigational product dose administration time from “within 60 minutes” to “preferably within 1-2 hours” after each plasma exchange cycle Changed the definition of the Per Protocol population from participants who have “no major protocol deviations or inclusion/exclusion criteria deviations” to “no major protocol deviations or key inclusion/exclusion criteria deviations” Allowed the Treating Physician’s designee to perform the mRS assessment • Detailed instructions for supplemental dosing after PE that take into account the possibility of PE during the Blind Induction Phase.
    03 Aug 2016
    • Established an Adjudication Committee to perform independent blinded reviews of all On-trial Relapses as determined by the Treating Physician • Added a sensitivity analysis for the change in ARR between the baseline ARR and Adjudicated On-trial ARR including all relapses when on eculizumab treatment in Study ECU-NMO-302 using a Wilcoxon signed rank test.
    22 Mar 2018
    • Extended the study duration from 4 years to 5.5 years • Reduced the number of visits at which pharmacokinetics/pharmacodynamics (PK/PD) samples were to be collected • Added an appendix to describe the procedures for collection of post-study follow-up information from participants who withdrew from the study.

    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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