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    Clinical Trial Results:
    An open label, multi-center trial of eculizumab in patients with Shiga-toxin producing Escherichia Coli Hemolytic-Uremic Syndrome (STEC-HUS)

    Summary
    EudraCT number
    		 2011-002691-17
    Trial protocol
    		 DE  
    Global end of trial date
    20 Apr 2012

    Results information
    Results version number
    		 v1(current)
    This version publication date
    24 Jul 2016
    First version publication date
    24 Jul 2016
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    C11-001
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT01410916
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Incorporated
    Sponsor organisation address
    352 Knotter Drive, Cheshire, CT, United States, 06410
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, clinicaltrials.eu@alxn.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, clinicaltrials.eu@alxn.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000876-PIP02-11
    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
    21 May 2014
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Apr 2012
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Apr 2012
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Safety and efficacy of eculizumab treatment in patients with STEC-HUS
    Protection of trial subjects
    Vaccination against N. meningitidis at least 14 days prior to study drug initiation or prophylactic antibiotics protection
    Background therapy
    		 -
    Evidence for comparator
    		 None
    Actual start date of recruitment
    22 Jul 2011
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    		 Safety, Efficacy
    Long term follow-up duration
    		 2 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Germany: 198
    Worldwide total number of subjects
    198
    EEA total number of subjects
    198
    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)
    2
    Adolescents (12-17 years)
    7
    Adults (18-64 years)
    163
    From 65 to 84 years
    25
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    		 196 patients were retrospectively enrolled after signing ICF. All received commercially available eculizumab prior to enrollment and at least 1 dose of eculizumab as investigational product following study entry. Two patients were enrolled prospectively. This represents the IIT/safety population.

    Pre-assignment
    Screening details
    		 At screening, the following to be collected: medical history, demographics, historical data review, administration/confirmation of N. meningiditis vaccination and prophylactic antibiotics; neurology assessments, clinical laboratories, safety, seizure assessment, disease-specific information.

    Pre-assignment period milestones
    Number of subjects started
    		 198
    Number of subjects completed
    		 198

    Period 1
    Period 1 title
    Treatment Period (28 weeks) (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded

    Arms
    Arm title
    		 eculizumab
    Arm description
    		 A total of 196 patients were retrospectively enrolled after signing ICF. All received commercially available eculizumab prior to enrollment and at least 1 dose of eculizumab as investigational product following study entry. Two patients were enrolled prospectively. This represents the IIT/safety population. Fixed dosing of eculizumab based on body weight cohorts were administered. Adjustment of dose to accommodate patient growth was possible.
    Arm type
    		 Experimental

    Investigational medicinal product name
    eculizumab
    Investigational medicinal product code
    eculizumab
    Other name
    Soliris
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eculizumab was to be administered intravenously (IV) according to the regimens described below: - If weight ≥ 40 kg: Induction: 900 mg weekly x 4; Maintenance: 1200 mg Wk5; then 1200 mg every 2 weeks - If weight 30 - < 40 kg: Induction: 600 mg weekly x 2; Maintenance: 900 mg Wk3; then 900 mg every 2 weeks - If weight 20 - < 30 kg: Induction: 600 mg weekly x 2 ; Maintenance: 600 mg Wk3; then 600 mg every 2 weeks - If weight 10 - < 20 kg: Induction: 600 mg weekly x 1; Maintenance: 300 mg Wk2; then 300 mg every 2 weeks - If weight 5 - < 10 kg: Induction: 300 mg weekly x 1; Maintenance: 300 mg Wk2; then 300 mg every 3 weeks Induction: 300 mg weekly x 1 Maintenance: 300 mg Wk2; then 300 mg every 2 weeks

    Number of subjects in period 1
    		eculizumab
    Started
    198
    Completed
    184
    Not completed
    14
         Patient condition improvement
    2
         Adverse Event
    2
         Lost to follow-up
    7
         Patient/parent decision
    3

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    eculizumab
    Reporting group description
    		 A total of 196 patients were retrospectively enrolled after signing ICF. All received commercially available eculizumab prior to enrollment and at least 1 dose of eculizumab as investigational product following study entry. Two patients were enrolled prospectively. This represents the IIT/safety population. Fixed dosing of eculizumab based on body weight cohorts were administered. Adjustment of dose to accommodate patient growth was possible.

    Reporting group values
    		 eculizumab Total
    Number of subjects
    		 198 198
    Age categorical
    Units: Subjects
        Pediatric patients (< 18)
    		 9 9
        Adults (18 ≤ 45)
    		 112 112
        Adults (45 ≤ 65)
    		 51 51
        ≥ 65
    		 26 26
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 42.1 ± 17.06 -
    Gender categorical
    Units: Subjects
        Female
    		 142 142
        Male
    		 56 56
    Race
    Units: Subjects
        White
    		 198 198
    PE/PI at Baseline
    Units: Subjects
        Yes
    		 181 181
        No
    		 17 17
    Ever on PE/PI
    Units: Subjects
        Yes
    		 186 186
        No
    		 12 12
    Ventilator use at Baseline
    Units: Subjects
        Yes
    		 47 47
        No
    		 151 151
    Baseline Seizure Status
    Seizure status is presented in patients with brain involvement at baseline. Reporting group (IIT/Safety population): Data available for 166 subjects
    Units: Subjects
        Yes
    		 43 43
        No
    		 123 123
        Not brain involvement
    		 32 32
    Therapeutic Coma at Baseline
    Reporting group (IIT/Safety population): Data available for 166 subjects.
    Units: Subjects
        Yes
    		 35 35
        No
    		 131 131
        Not brain involvement
    		 32 32
    Hospitalization at Baseline
    Units: Subjects
        Yes
    		 197 197
        No
    		 1 1
    WBC category
    Units: Subjects
        > 20 x 10^9/L
    		 33 33
        ≤ 20 x 10^9/L
    		 165 165
    Kidney involvement at Baseline
    Units: Subjects
        Kidney involvement
    		 190 190
        No kidney involvement
    		 8 8
    Brain involvement at Baseline
    Units: Subjects
        Brain involvement
    		 166 166
        No brain involvement
    		 32 32
    Thrombosis at Baseline
    Units: Subjects
        Thrombosis
    		 0 0
        No thrombosis
    		 198 198
    Brain and kidney involvement at Baseline
    Units: Subjects
        Brain and kidney involvement
    		 158 158
        No brain and kidney involvement
    		 40 40
    No organ involvement at Baseline
    Units: Subjects
        No organ involvement
    		 0 0
        Any organ involvement
    		 198 198
    Dialysis prior to treatment
    Units: Number of days
        arithmetic mean (standard deviation)
    		 5 ± 11.27 -
    PE/PI prior to treatment
    Units: Number of Days
        arithmetic mean (standard deviation)
    		 4.3 ± 2.3 -
    Duration from Onset of PE/PI to initiation of eculizumab at baseline
    Units: days
        arithmetic mean (standard deviation)
    		 6.7 ± 9.06 -
    Duration from onset of diarrhea to onset of PE/PI at baseline
    Units: days
        arithmetic mean (standard deviation)
    		 7 ± 2.54 -
    Duration from onset of diarrhea to initiation of eculizumab at baseline
    Units: days
        arithmetic mean (standard deviation)
    		 12.7 ± 9.36 -
    Modified Rankin Score at Baseline
    Units: Modified Rankin Score
        arithmetic mean (standard deviation)
    		 3.7 ± 1.18 -
    Baseline Creatinine
    Units: micromole(s)/litre
        arithmetic mean (standard deviation)
    		 217.3 ± 102.57 -
    Baseline eGFR
    eGFR are presented for patients with kidney involvement who were not on dialysis at baseline.
    Units: mL/min/1.73m^2
        arithmetic mean (standard deviation)
    		 34 ± 16.29 -
    Baseline haemoglobin
    Units: gram(s)/litre
        arithmetic mean (standard deviation)
    		 84.5 ± 15.94 -
    Baseline Lactate Dehydrogenase
    Units: U/L
        arithmetic mean (standard deviation)
    		 844.4 ± 459.84 -
    Baseline Platelets
    Units: 10^9/L
        arithmetic mean (standard deviation)
    		 78.2 ± 65.33 -
    Baseline White Blood Cells
    Units: 10^9/L
        arithmetic mean (standard deviation)
    		 14 ± 6.86 -

    End points

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    End points reporting groups
    Reporting group title
    eculizumab
    Reporting group description
    		 A total of 196 patients were retrospectively enrolled after signing ICF. All received commercially available eculizumab prior to enrollment and at least 1 dose of eculizumab as investigational product following study entry. Two patients were enrolled prospectively. This represents the IIT/safety population. Fixed dosing of eculizumab based on body weight cohorts were administered. Adjustment of dose to accommodate patient growth was possible.

    Primary: Improvement in systemic TMA and vital organ at Wk 8

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    End point title
    		 Improvement in systemic TMA and vital organ at Wk 8 [1]
    End point description
    It consists of complete and partial responders, as defined below: - Complete Responder: (i) Hematologic Normalization (platelet count ≥150 x 10^9/L at any 2 consecutive measures); (ii) Clinically important improvement in all of the affected major vital organs: brain, kidney, thrombosis when abnormal at baseline and with baseline abnormality plausibly related to EHEC event; and (iii) no clinically important worsening in Brain, Kidney, Thrombosis. - Partial Responder: (i) Hematologic Improvement (>25% increase in platelet count at any 2 consecutive measures) or Hematologic Normalization; (ii) Clinically important improvement in none, one, or more affected major organs: brain, kidney and thrombosis when abnormal at baseline and when baseline abnormality plausibly related to the STEC event; and (iii) no clinically important worsening in Brain, Kidney, Thrombosis.
    End point type
    Primary
    End point timeframe
    Through Week 8
    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: The system does not support statistical analyses for a single arm trial.
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    number (confidence interval 95%)
        Complete response
    80.3 (74.1 to 85.6)
        Partial response
    94.4 (90.3 to 97.2)
    No statistical analyses for this end point

    Primary: Improvement in systemic TMA and vital organ at Wk 28

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    End point title
    		 Improvement in systemic TMA and vital organ at Wk 28 [2]
    End point description
    Evidence for sustained response to eculizumab continuing after the end of the dosing period was evaluated by the global assessment of efficacy defined as response rate (CR+PR) at Week 28.
    End point type
    Primary
    End point timeframe
    28 weeks
    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: The system does not support statistical analyses for a single arm trial.
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    number (confidence interval 95%)
        Complete response
    88.9 (83.7 to 92.9)
        Partial response
    94.4 (90.3 to 97.2)
    No statistical analyses for this end point

    Secondary: New Ventilator Requirement

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    End point title
    		 New Ventilator Requirement
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: percent
    number (confidence interval 95%)
        Week 8
    6.1 (3.2 to 10.3)
        Week 28
    6.1 (3.2 to 10.3)
    No statistical analyses for this end point

    Secondary: New Dialysis After Day 14 of eculizumab treatment

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    End point title
    		 New Dialysis After Day 14 of eculizumab treatment
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    53
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    0 (0 to 6.7)
        Week 28
    0 (0 to 6.7)
    No statistical analyses for this end point

    Secondary: Hematological Normalization and No New Organ Involvement

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    End point title
    		 Hematological Normalization and No New Organ Involvement
    End point description
    End point type
    Secondary
    End point timeframe
    Week 8 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    91.4 (86.6 to 94.9)
        Week 28
    92.9 (88.4 to 96.1)
    No statistical analyses for this end point

    Secondary: Hematological Normalization

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    End point title
    		 Hematological Normalization
    End point description
    Platelet ≥ 150x10^9/L for any two measures (all patients)
    End point type
    Secondary
    End point timeframe
    Week 8 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    97 (93.5 to 98.9)
        Week 28
    98.5 (95.6 to 99.7)
    No statistical analyses for this end point

    Secondary: Hematological Normalization

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    End point title
    		 Hematological Normalization
    End point description
    Platelet ≥ 150x10^9/L for any two measures (patients with platelets <150 x 10^9/L at Baseline)
    End point type
    Secondary
    End point timeframe
    At week 8 and 28
    End point values
    		 eculizumab
    Number of subjects analysed
    174
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    96.6 (92.6 to 98.7)
        Week 28
    98.3 (95 to 99.6)
    No statistical analyses for this end point

    Secondary: Global Assessement of Neurological Function : Clinically Important Improvement

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    End point title
    		 Global Assessement of Neurological Function : Clinically Important Improvement
    End point description
    Clinically important improvement is assessed in patients with the associated organ involvement at baseline. Results in the PP population were found to be similar to those of the ITT population, so only the ITT results are shown for all secondary endpoint assessments.
    End point type
    Secondary
    End point timeframe
    At week 8, week 16 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    152
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    88.8 (82.7 to 93.3)
        Week 16
    94.7 (89.9 to 97.7)
        Week 28
    96.1 (91.6 to 98.5)
    No statistical analyses for this end point

    Secondary: Global Assessment of Neurological Function : Clinically Important Worsening

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    End point title
    		 Global Assessment of Neurological Function : Clinically Important Worsening
    End point description
    Results in the PP population were found to be similar to those of the ITT population, so only the ITT results are shown for all secondary endpoint assessments. Clinically important worsening is assessed in all patients.
    End point type
    Secondary
    End point timeframe
    At week 8, week 16 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    193
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    2.1 (0.6 to 5.2)
        Week 16
    2.1 (0.6 to 5.2)
        Week 28
    2.1 (0.6 to 5.2)
    No statistical analyses for this end point

    Secondary: Global Assessment of Renal Function : Clinically Important Improvement

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    End point title
    		 Global Assessment of Renal Function : Clinically Important Improvement
    End point description
    Clinically important improvement is assessed in patients with the associated organ involvement at baseline.
    End point type
    Secondary
    End point timeframe
    At week 8, week 16 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    182
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    96.2 (92.2 to 98.4)
        Week 16
    98.4 (95.3 to 99.7)
        Week 28
    98.9 (96.1 to 99.9)
    No statistical analyses for this end point

    Secondary: Global Assessment of Renal Function : Clinically Important Worsening

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    End point title
    		 Global Assessment of Renal Function : Clinically Important Worsening
    End point description
    Clinically important worsening is assessed in all patients. In the ITT population, 137 patients could not be assessed for clinically important worsening in renal function as they were on dialysis at baseline.
    End point type
    Secondary
    End point timeframe
    At week 8, week 16 and week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    61
    Units: Percentage of patients
    number (confidence interval 95%)
        Week 8
    4.9 (1 to 13.7)
        Week 16
    4.9 (1 to 13.7)
        Week 28
    4.9 (1 to 13.7)
    No statistical analyses for this end point

    Secondary: TMA event-free statut for > 6 weeks

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    End point title
    		 TMA event-free statut for > 6 weeks
    End point description
    TMA event-free status for >6 weeks: defined as no plasma therapy, no ≥25% decrease in platelet count, and no new dialysis. The cumulative incidence was estimated using the cumulative distribution function (CDF).
    End point type
    Secondary
    End point timeframe
    Up to Week 28
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    number (not applicable)
        No TMA
    99.5
        Censored
    0.5
    No statistical analyses for this end point

    Secondary: TMA intervention rate

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    End point title
    		 TMA intervention rate
    End point description
    TMA intervention rate defined as the number of PE/PI interventions plus the number of dialysis events per patient per day. The TMA intervention rate was calculated pretreatment and post-treatment. Pre-treatment is from the onset of diarrhea to just prior to the first dose of eculizumab (day -1). Post-treatment is from the first dose of eculizumab (Day 0) to the end of study follow up (or discontinuation day for discontinued patients). P-value is calculated using a two-sided Wilcoxon signed rank test.
    End point type
    Secondary
    End point timeframe
    Up to 28 weeks
    End point values
    		 eculizumab
    Number of subjects analysed
    198
    Units: Percentage of patients
    arithmetic mean (standard deviation)
        Pretreatment TMA intervention rate
    0.5192 ± 0.2906
        Post-treatment TMA intervention rate
    0.0549 ± 0.0784
    No statistical analyses for this end point

    Other pre-specified: Improvement in systemic TMA and vital organ at Wk 8 for Patients Dosed Beyond 8 Weeks

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    End point title
    		 Improvement in systemic TMA and vital organ at Wk 8 for Patients Dosed Beyond 8 Weeks
    End point description
    Global assessment of efficacy (CR + PR) at Week 8 for patients who were dosed beyond Week 8 This analysis is a sub-group analysis (Intent-to-Treat Population)
    End point type
    Other pre-specified
    End point timeframe
    8 weeks
    End point values
    		 eculizumab
    Number of subjects analysed
    27
    Units: Percentage of patients
    number (confidence interval 95%)
        Complete response
    74.1 (53.7 to 88.9)
        Partial response
    85.2 (66.3 to 95.8)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Through end of study
    Adverse event reporting additional description
    All AEs that occurred after the patient had given consent must have been reported and followed to satisfactory resolution or until the Principal Investigator deems the event to be chronic or the patient to be stable. In the non SAE section below, non SAE and SAE are reported.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    14.0
    Reporting groups
    Reporting group title
    eculizumab
    Reporting group description
    		 A total of 196 patients were retrospectively enrolled after signing ICF. All received commercially available eculizumab prior to enrollment and at least 1 dose of eculizumab as investigational product following study entry. Two patients were enrolled prospectively. This represents the IIT/safety population. Fixed dosing of eculizumab based on body weight cohorts were administered. Adjustment of dose to accommodate patient growth was possible.

    Serious adverse events
    		 eculizumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    66 / 198 (33.33%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Acute myeloid leukaemia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Prostate cancer
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Vascular disorders
    Haematoma
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemorrhage
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hypertensive crisis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Venous thrombosis
         subjects affected / exposed
    3 / 198 (1.52%)
         occurrences causally related to treatment / all
    3 / 3
         deaths causally related to treatment / all
    0 / 0
    Pregnancy, puerperium and perinatal conditions
    Amniotic cavity disorder
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Complication of pregnancy
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Immune system disorders
    Anaphylactic reaction
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Cervical dysplasia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Acute respiratory distress syndrome
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pleural effusion
         subjects affected / exposed
    3 / 198 (1.52%)
         occurrences causally related to treatment / all
    1 / 3
         deaths causally related to treatment / all
    0 / 0
    Pneumothorax
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Pulmonary embolism
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pulmonary oedema
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Respiratory failure
         subjects affected / exposed
    4 / 198 (2.02%)
         occurrences causally related to treatment / all
    4 / 4
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Transient psychosis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Blood creatinine increased
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Ejection fraction decreased
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Platelet count decreased
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    Post procedural haemorrhage
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Shunt occlusion
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Suture rupture
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Left ventricular failure
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Myocardial infarction
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Myocardial ischaemia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tachyarrhythmia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Aphasia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Brain oedema
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cerebral infarction
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Convulsion
         subjects affected / exposed
    24 / 198 (12.12%)
         occurrences causally related to treatment / all
    2 / 30
         deaths causally related to treatment / all
    0 / 0
    Dysaesthesia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Epilepsy
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemorrhagic stroke
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hemianopia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Hemiparesis
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Neurological symptom
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Partial seizures
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemolytic uraemic syndrome
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Heparin-induced thrombocytopenia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Optic nerve disorder
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal adhesions
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Colitis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Colonic stenosis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemorrhoidal haemorrhage
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Ileus
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Subileus
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Vomiting
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Cholangitis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis acute
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholelithiasis
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Liver disorder
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Skin and subcutaneous tissue disorders
    Angioedema
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Dermatitis allergic
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Urethral prolapse
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Device related infection
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haematoma infection
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences causally related to treatment / all
    7 / 10
         deaths causally related to treatment / all
    0 / 0
    Pneumonia herpes viral
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Pseudomonas infection
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    2 / 198 (1.01%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Staphylococcal sepsis
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Hyperkalaemia
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Type 1 diabetes mellitus
         subjects affected / exposed
    1 / 198 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 eculizumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    196 / 198 (98.99%)
    Vascular disorders
    Hypertension
         subjects affected / exposed
    74 / 198 (37.37%)
         occurrences all number
    81
    Hypotension
         subjects affected / exposed
    12 / 198 (6.06%)
         occurrences all number
    12
    General disorders and administration site conditions
    Asthenia
         subjects affected / exposed
    12 / 198 (6.06%)
         occurrences all number
    12
    Chest pain
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Fatigue
         subjects affected / exposed
    30 / 198 (15.15%)
         occurrences all number
    32
    Oedema
         subjects affected / exposed
    29 / 198 (14.65%)
         occurrences all number
    30
    Oedema peripheral
         subjects affected / exposed
    64 / 198 (32.32%)
         occurrences all number
    80
    Pyrexia
         subjects affected / exposed
    25 / 198 (12.63%)
         occurrences all number
    28
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    19 / 198 (9.60%)
         occurrences all number
    21
    Dyspnoea
         subjects affected / exposed
    32 / 198 (16.16%)
         occurrences all number
    34
    Pleural effusion
         subjects affected / exposed
    48 / 198 (24.24%)
         occurrences all number
    51
    Alopecia
         subjects affected / exposed
    69 / 198 (34.85%)
         occurrences all number
    69
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    19 / 198 (9.60%)
         occurrences all number
    19
    Anxiety
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    12
    Depression
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    11
    Disorientation
         subjects affected / exposed
    15 / 198 (7.58%)
         occurrences all number
    16
    Restlessness
         subjects affected / exposed
    12 / 198 (6.06%)
         occurrences all number
    12
    Sleep disorder
         subjects affected / exposed
    17 / 198 (8.59%)
         occurrences all number
    18
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    11
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    12 / 198 (6.06%)
         occurrences all number
    12
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    13 / 198 (6.57%)
         occurrences all number
    13
    Nervous system disorders
    Aphasia
         subjects affected / exposed
    21 / 198 (10.61%)
         occurrences all number
    24
    Convulsion
         subjects affected / exposed
    28 / 198 (14.14%)
         occurrences all number
    35
    Disturbance in attention
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    12
    Dizziness
         subjects affected / exposed
    18 / 198 (9.09%)
         occurrences all number
    20
    Headache
         subjects affected / exposed
    95 / 198 (47.98%)
         occurrences all number
    102
    Myoclonus
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    11
    Paraesthesia
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Tremor
         subjects affected / exposed
    28 / 198 (14.14%)
         occurrences all number
    29
    Insomnia
         subjects affected / exposed
    20 / 198 (10.10%)
         occurrences all number
    21
    Blood and lymphatic system disorders
    Anaemia
         subjects affected / exposed
    13 / 198 (6.57%)
         occurrences all number
    13
    Leukopenia
         subjects affected / exposed
    13 / 198 (6.57%)
         occurrences all number
    13
    Ear and labyrinth disorders
    Vertigo
         subjects affected / exposed
    22 / 198 (11.11%)
         occurrences all number
    24
    Eye disorders
    Diplopia
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Vision blurred
         subjects affected / exposed
    12 / 198 (6.06%)
         occurrences all number
    12
    Visual impairment
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    26 / 198 (13.13%)
         occurrences all number
    27
    Abdominal pain upper
         subjects affected / exposed
    14 / 198 (7.07%)
         occurrences all number
    14
    Ascites
         subjects affected / exposed
    15 / 198 (7.58%)
         occurrences all number
    15
    Constipation
         subjects affected / exposed
    20 / 198 (10.10%)
         occurrences all number
    20
    Diarrhoea
         subjects affected / exposed
    21 / 198 (10.61%)
         occurrences all number
    23
    Flatulence
         subjects affected / exposed
    13 / 198 (6.57%)
         occurrences all number
    13
    Nausea
         subjects affected / exposed
    61 / 198 (30.81%)
         occurrences all number
    66
    Vomiting
         subjects affected / exposed
    44 / 198 (22.22%)
         occurrences all number
    46
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Renal and urinary disorders
    Nocturia
         subjects affected / exposed
    17 / 198 (8.59%)
         occurrences all number
    17
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    22 / 198 (11.11%)
         occurrences all number
    26
    Back pain
         subjects affected / exposed
    21 / 198 (10.61%)
         occurrences all number
    22
    Myalgia
         subjects affected / exposed
    14 / 198 (7.07%)
         occurrences all number
    19
    Pain in extremity
         subjects affected / exposed
    10 / 198 (5.05%)
         occurrences all number
    10
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    31 / 198 (15.66%)
         occurrences all number
    33
    Pneumonia
         subjects affected / exposed
    19 / 198 (9.60%)
         occurrences all number
    21
    Urinary tract infection
         subjects affected / exposed
    22 / 198 (11.11%)
         occurrences all number
    23
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    11 / 198 (5.56%)
         occurrences all number
    11

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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