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    Clinical Trial Results:
    A Phase III, open-label, extension study of eculizumab in patients with transfusion-dependent, haemolytic Paroxysmal Nocturnal Haemoglobinuria (PNH) who have participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 studies.

    Summary
    EudraCT number
    2005-000043-28
    Trial protocol
    GB   IE   SE   DE   ES   IT  
    Global end of trial date
    12 Sep 2008

    Results information
    Results version number
    v1(current)
    This version publication date
    06 Jan 2017
    First version publication date
    06 Jan 2017
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    E05-001
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT00122317
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    Alexion Pharmaceuticals Incorporated
    Sponsor organisation address
    100 College Street, New Haven, CT, United States, 06510
    Public contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, clinicaltrials.eu@alexion.com
    Scientific contact
    European Clinical Trial Information, Alexion Europe SAS, +33 1 47 10 06 06, 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
    31 Mar 2010
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 Sep 2008
    Global end of trial reached?
    Yes
    Global end of trial date
    12 Sep 2008
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective was to evaluate the long-term safety of eculizumab in patients with haemolytic PNH, who had completed the eculizumab TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 studies. The TRIUMPH study was a double-blind, placebo-controlled study in which haemolytic, transfusion-dependent patients received eculizumab (N=43) or placebo (N=44) administered by intravenous (IV) infusion for 26 weeks. The SHEPHERD study was an open-label study in which patients (N=97) received eculizumab treatment for 52 weeks. The X03-001 study was an open-label extension study of eculizumab in which patients with transfusion dependent, haemolytic, PNH continued to receive treatment for an additional 104 weeks.
    Protection of trial subjects
    Patients must have been vaccinated for Neisseria meningitidis 14 days before the first investigational product infusion in the parent studies (C04-001 [TRIUMPH], C04-002 [SHEPHERD], or X03-001 studies).
    Background therapy
    No background therapy was used.
    Evidence for comparator
    This was an open-label extension study, opened to patients who had completed the eculizumab TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 studies, and had consented to participate. Patients who had completed the randomised, double-blind, placebo-controlled TRIUMPH study were required to enroll in a 4-week blind induction period to preserve the blinded treatment before inclusion in the open-label treatment period ofstudy E05-001.
    Actual start date of recruitment
    09 May 2005
    Long term follow-up planned
    Yes
    Long term follow-up rationale
    Safety
    Long term follow-up duration
    30 Months
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Spain: 9
    Country: Number of subjects enrolled
    Sweden: 5
    Country: Number of subjects enrolled
    United Kingdom: 44
    Country: Number of subjects enrolled
    Belgium: 3
    Country: Number of subjects enrolled
    France: 2
    Country: Number of subjects enrolled
    Germany: 24
    Country: Number of subjects enrolled
    Italy: 21
    Country: Number of subjects enrolled
    Australia: 14
    Country: Number of subjects enrolled
    Canada: 2
    Country: Number of subjects enrolled
    Ireland: 5
    Country: Number of subjects enrolled
    Netherlands: 12
    Country: Number of subjects enrolled
    Switzerland: 1
    Country: Number of subjects enrolled
    United States: 45
    Worldwide total number of subjects
    187
    EEA total number of subjects
    125
    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)
    173
    From 65 to 84 years
    13
    85 years and over
    1

    Subject disposition

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    Recruitment
    Recruitment details
    A total of 46 clinical sites in the United States, Canada, Australia, Belgium, France, Germany, Ireland, Italy, the Netherlands, Sweden, Switzerland, and the United Kingdom participated in this study. Patients must have had fully completed the TRIUMPH (C04-001), SHEPHERD (C04-002), or X03-001 studies to enter this extension study.

    Pre-assignment
    Screening details
    A total of 187/195 patients from the 3 previous PNH studies of eculizumab elected to continue treatment in the E05-001 extension study (85 [41 eculizumab-treated patients and 44 placebo-treated patients]), 92, and 10 patients from the TRIUMPH, SHEPHERD, and X03-001 studies, respectively).

    Period 1
    Period 1 title
    2-yr treatment period (overall period)
    Is this the baseline period?
    Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    Not blinded
    Blinding implementation details
    This study was not blinded. Of note, patients who completed the TRIUMPH study, a 4-week blind induction period was applied to preserve the blinded treatment before inclusion in study E05-001. Patients who completed the SHEPHERD or X03-001 studies entered directly into this open-label treatment extension study because there was no blind to be maintained.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    eculizumab (PNH studies eculizumab-treated patients)
    Arm description
    This arm consisted of patients who had received eculizumab in any of the 3 previous PNH studies of eculizumab (TRIUMPH, SHEPHERD, or X03-001).
    Arm type
    Experimental

    Investigational medicinal product name
    ecullizumab
    Investigational medicinal product code
    eculizumab
    Other name
    Soliris
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    - Patients randomised to eculizumab in the TRIUMPH study: Eculizumab at a dose of 900 mg at Weeks 0, 2, and 4, and then 900 mg and then every 2 weeks through the end of the study. These patients also received placebo at Weeks 1 and 3. - All other patients were infused with eculizumab at a dose of 900 mg biweekly beginning at Week 0 and through the end of the study.

    Arm title
    eculizumab (TRIUMPH placebo-treated patients)
    Arm description
    This arm consisted of patients who had received placebo in the TRIUMPH study.
    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
    Patients randomised to placebo in the TRIUMPH study: Eculizumab at a dose of 600 mg once a week for 4 weeks, followed by 900 mg of eculizumab 1 week later for one dose, then 900 mg of eculizumab every 2 weeks through the end of the study.

    Number of subjects in period 1
    eculizumab (PNH studies eculizumab-treated patients) eculizumab (TRIUMPH placebo-treated patients)
    Started
    143
    44
    Completed
    137
    39
    Not completed
    6
    5
         Protocol deviation
    1
    -
         Physician decision
    1
    1
         Adverse event, serious fatal
    1
    2
         Adverse event, non-fatal
    3
    1
         Consent withdrawn by subject
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    eculizumab (PNH studies eculizumab-treated patients)
    Reporting group description
    This arm consisted of patients who had received eculizumab in any of the 3 previous PNH studies of eculizumab (TRIUMPH, SHEPHERD, or X03-001).

    Reporting group title
    eculizumab (TRIUMPH placebo-treated patients)
    Reporting group description
    This arm consisted of patients who had received placebo in the TRIUMPH study.

    Reporting group values
    eculizumab (PNH studies eculizumab-treated patients) eculizumab (TRIUMPH placebo-treated patients) Total
    Number of subjects
    143 44 187
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    131 42 173
        From 65-84 years
    11 2 13
        85 years and over
    1 0 1
    Age continuous
    Units: years
        median (inter-quartile range (Q1-Q3))
    42.1 (30.8 to 53.5) 36 (30.4 to 46.2) -
    Gender categorical
    Units: Subjects
        Female
    72 29 101
        Male
    71 15 86
    Blood type
    Units: Subjects
        A-
    14 1 15
        A+
    41 20 61
        B-
    6 0 6
        B+
    9 5 14
        AB+
    8 2 10
        O-
    9 6 15
        O+
    55 10 65
        AB-
    1 0 1
    Race
    Units: Subjects
        Asian
    5 1 6
        Black
    7 0 7
        Caucasian
    127 41 168
        Hispanic
    0 1 1
        Latin
    1 0 1
        Saudi
    1 0 1
        Other
    1 1 2
        Jamaican
    1 0 1
    LDH at baseline in parent trial
    Units: U/L
        median (inter-quartile range (Q1-Q3))
    2139 (1488 to 2829) 2166.5 (1701 to 2965) -
    LDH Prior to First Dose in E05-001
    Units: U/L
        median (inter-quartile range (Q1-Q3))
    270 (215 to 326) 2166.5 (1701 to 2965) -
    red blood count at baseline in parent trial
    Units: x10*12/L
        median (inter-quartile range (Q1-Q3))
    2.93 (2.58 to 3.34) 2.78 (2.55 to 3.12) -
    RBC Prior to First Dose in E05-001
    Units: x10*12/L
        median (inter-quartile range (Q1-Q3))
    2.92 (2.56 to 3.2) 2.78 (2.55 to 3.12) -

    End points

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    End points reporting groups
    Reporting group title
    eculizumab (PNH studies eculizumab-treated patients)
    Reporting group description
    This arm consisted of patients who had received eculizumab in any of the 3 previous PNH studies of eculizumab (TRIUMPH, SHEPHERD, or X03-001).

    Reporting group title
    eculizumab (TRIUMPH placebo-treated patients)
    Reporting group description
    This arm consisted of patients who had received placebo in the TRIUMPH study.

    Primary: Intravascular haemolysis measured by LDH AUC

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    End point title
    Intravascular haemolysis measured by LDH AUC [1]
    End point description
    A quantitative assessment of haemolysis was obtained by calculating the AUC for LDH from Baseline to Month 24, and the data were analysed using a Wilcoxon signed rank test.
    End point type
    Primary
    End point timeframe
    Change from baseline through 24 months
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The system EudraCT does not allow entering for statistical analysis for single arm studies.
    End point values
    eculizumab (PNH studies eculizumab-treated patients) eculizumab (TRIUMPH placebo-treated patients)
    Number of subjects analysed
    140
    44
    Units: U/L* day
    median (inter-quartile range (Q1-Q3))
        6 Months
    -316018 (-420072 to -183916)
    -317196 (-429293 to -248130)
        12 Months
    -344930 (-478143 to -208390)
    -337482 (-451391 to -240626)
        18 Months
    -343846 (-485016 to -203676)
    -322356 (-461607 to -240964)
        24 Months
    -341977 (-492407 to -208304)
    -326061 (-469426 to -237221)
    No statistical analyses for this end point

    Secondary: Levels of fatigue

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    End point title
    Levels of fatigue
    End point description
    The Quality-of-Life (QoL) instrument FACIT-Fatigue scale version 4 was utilised to collect QoL data. The scoring guideline for the FACIT-Fatigue scale version 4 instrument was used to calculate the QoL score. Per the corresponding scoring guideline, scores can range from 0 to 52, with higher scores indicating improvement in fatigue.
    End point type
    Secondary
    End point timeframe
    through 24 months
    End point values
    eculizumab (PNH studies eculizumab-treated patients) eculizumab (TRIUMPH placebo-treated patients)
    Number of subjects analysed
    140
    44
    Units: Total FACIT-Fatigue score
    median (inter-quartile range (Q1-Q3))
        Change from baseline at 6 months
    7 (-18 to 38)
    7.5 (0 to 13.5)
        Change from baseline at 12 months
    7 (2 to 16)
    4 (0 to 14)
        Change from baseline at 18 months
    8 (1 to 14)
    7.5 (0 to 15.5)
        Change from baseline at 24 months
    7 (1 to 7)
    7 (1 to 15)
    No statistical analyses for this end point

    Secondary: Thrombosis events

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    End point title
    Thrombosis events
    End point description
    This endpoint reports the number of patients reporting thrombosis events in the present study.
    End point type
    Secondary
    End point timeframe
    Through 24 months
    End point values
    eculizumab (PNH studies eculizumab-treated patients) eculizumab (TRIUMPH placebo-treated patients)
    Number of subjects analysed
    140
    44
    Units: Total number of events
        Number of events
    9
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Information regarding AEs was collected from the time the patient signed the informed consent form up to 30 days after the last dose of eculizumab was administered.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    6.1
    Reporting groups
    Reporting group title
    Eculizumab (overall)
    Reporting group description
    This group reports safety data in the safety population, consisting of all patients who received any amount of eculizumab istudy E05-001. Overall, patients who had received eculizumab in the parent trials, TRIUMPH, SHEPHERD or X03-001, were exposed to eculizumab for a 30.3-month median duration (vs 24-month median duration for those who had received placebo in the parent trial, TRIUMPH).

    Serious adverse events
    Eculizumab (overall)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    57 / 187 (30.48%)
         number of deaths (all causes)
    3
         number of deaths resulting from adverse events
    0
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    phlebothrombosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombophlebitis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Thrombosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 1
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Adenocarcinoma
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Chronic myelomonocytic leukaemia
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 1
    Malignant melanoma
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Metastases to bone
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    General disorders and administration site conditions
    Chest pain
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pyrexia
         subjects affected / exposed
    5 / 187 (2.67%)
         occurrences causally related to treatment / all
    2 / 6
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Suicidal ideation
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Reproductive system and breast disorders
    Erectile dysfunction
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Ovarian cyst ruptured
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Injury, poisoning and procedural complications
    drug toxicity
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Wrist fracture
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Investigations
    Haemoglobin decreased
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cardiac disorders
    Cardiac failure
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pleurisy
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Blood and lymphatic system disorders
    Agranulocytosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Anaemia
         subjects affected / exposed
    3 / 187 (1.60%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    Aplastic anaemia
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Neutropenia
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Haemolysis
         subjects affected / exposed
    5 / 187 (2.67%)
         occurrences causally related to treatment / all
    0 / 5
         deaths causally related to treatment / all
    0 / 0
    Paroxysmal nocturnal haemoglobinuria
         subjects affected / exposed
    4 / 187 (2.14%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Thrombocytopenia
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Nervous system disorders
    Cerebral haemorrhage
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    convulsion
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Headache
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolic encephalopathy
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Eye disorders
    Retinal vein thrombosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    5 / 187 (2.67%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Ascites
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Colitis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Diarrhoea
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastrointestinal haemorrhage
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    sigmoiditis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Renal and urinary disorders
    Calculus urinary
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Nephrolithiasis
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    renal failure acute
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Renal impairment
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hepatobiliary disorders
    Bile duct stone
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis
         subjects affected / exposed
    3 / 187 (1.60%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Cholecystitis acute
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Cholelithiasis
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Hepatic cirrhosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Hepatic vein thrombosis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Portal vein thrombosis
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Musculoskeletal and connective tissue disorders
    Intervertebral disc protrusion
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Tenosynovitis stenosans
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Hypokalaemia
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Hyponatraemia
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Cellulitis
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Cellulitis gangrenous
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Empyema
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Endocarditis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Enterococcal sepsis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Gallbladder abscess
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Gastroenteritis viral
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Haemophilus infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Liver abscess
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Lower respiratory tract infection
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Meningococcal sepsis
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Bacterial sepsis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Bronchitis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Necrotising fasciitis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Penile infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Pneumonia
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Respiratory tract infection
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    1 / 2
         deaths causally related to treatment / all
    0 / 0
    Sepsis
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Septic shock
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    2 / 2
         deaths causally related to treatment / all
    0 / 0
    Staphylococcal infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    2 / 187 (1.07%)
         occurrences causally related to treatment / all
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    Viral infection
         subjects affected / exposed
    3 / 187 (1.60%)
         occurrences causally related to treatment / all
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    Viral upper respiratory tract infection
         subjects affected / exposed
    1 / 187 (0.53%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    Eculizumab (overall)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    182 / 187 (97.33%)
    Vascular disorders
    Haematoma
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    23
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    22 / 187 (11.76%)
         occurrences all number
    37
    Cardiac disorders
    Chest pain
         subjects affected / exposed
    10 / 187 (5.35%)
         occurrences all number
    17
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    30 / 187 (16.04%)
         occurrences all number
    47
    Epistaxis
         subjects affected / exposed
    15 / 187 (8.02%)
         occurrences all number
    16
    Pharyngolaryngeal pain
         subjects affected / exposed
    26 / 187 (13.90%)
         occurrences all number
    32
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    23 / 187 (12.30%)
         occurrences all number
    30
    Headache
         subjects affected / exposed
    69 / 187 (36.90%)
         occurrences all number
    155
    General disorders and administration site conditions
    Influenza like illness
         subjects affected / exposed
    29 / 187 (15.51%)
         occurrences all number
    56
    Pyrexia
         subjects affected / exposed
    22 / 187 (11.76%)
         occurrences all number
    33
    Oedema peripheral
         subjects affected / exposed
    14 / 187 (7.49%)
         occurrences all number
    15
    Fatigue
         subjects affected / exposed
    16 / 187 (8.56%)
         occurrences all number
    16
    Psychiatric disorders
    Depression
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    13
    Insomnia
         subjects affected / exposed
    16 / 187 (8.56%)
         occurrences all number
    35
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    44 / 187 (23.53%)
         occurrences all number
    70
    Abdominal pain
         subjects affected / exposed
    28 / 187 (14.97%)
         occurrences all number
    49
    Abdominal pain upper
         subjects affected / exposed
    18 / 187 (9.63%)
         occurrences all number
    41
    Dyspepsia
         subjects affected / exposed
    16 / 187 (8.56%)
         occurrences all number
    22
    Nausea
         subjects affected / exposed
    39 / 187 (20.86%)
         occurrences all number
    54
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    24
    Rash
         subjects affected / exposed
    13 / 187 (6.95%)
         occurrences all number
    19
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    39 / 187 (20.86%)
         occurrences all number
    65
    Back pain
         subjects affected / exposed
    35 / 187 (18.72%)
         occurrences all number
    45
    muscle cramp
         subjects affected / exposed
    13 / 187 (6.95%)
         occurrences all number
    17
    Myalgia
         subjects affected / exposed
    18 / 187 (9.63%)
         occurrences all number
    22
    Neck pain
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    18
    Pain in extremity
         subjects affected / exposed
    26 / 187 (13.90%)
         occurrences all number
    40
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    10 / 187 (5.35%)
         occurrences all number
    17
    Gastroenteritis
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    14
    Gastroenteritis viral
         subjects affected / exposed
    10 / 187 (5.35%)
         occurrences all number
    11
    Herpes simplex
         subjects affected / exposed
    12 / 187 (6.42%)
         occurrences all number
    16
    Influenza
         subjects affected / exposed
    10 / 187 (5.35%)
         occurrences all number
    11
    Nasopharyngitis
         subjects affected / exposed
    74 / 187 (39.57%)
         occurrences all number
    148
    Respiratory tract infection
         subjects affected / exposed
    10 / 187 (5.35%)
         occurrences all number
    10
    Sinusitis
         subjects affected / exposed
    15 / 187 (8.02%)
         occurrences all number
    16
    Upper respiratory tract infection
         subjects affected / exposed
    58 / 187 (31.02%)
         occurrences all number
    102
    Urinary tract infection
         subjects affected / exposed
    22 / 187 (11.76%)
         occurrences all number
    31
    Viral infection
         subjects affected / exposed
    19 / 187 (10.16%)
         occurrences all number
    30

    More information

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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

    Online references

    http://www.ncbi.nlm.nih.gov/pubmed/17702897
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