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    Clinical Trial Results:
    Eculizumab in Shiga-Toxin producing E. Coli Haemolytic Uraemic Syndrome (ECUSTEC): A Randomised, Double-Blind, Placebo-Controlled Trial

    Summary
    EudraCT number
    		 2016-000997-39
    Trial protocol
    		 GB  
    Global end of trial date
    01 Jul 2021

    Results information
    Results version number
    		 v1(current)
    This version publication date
    25 Nov 2021
    First version publication date
    25 Nov 2021
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    7837
    Additional study identifiers
    ISRCTN number
    		 ISRCTN89553116
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 REC Ref No: 16/NE/0325, ISRCTN: 89553116, MHRA CTA: 17136/0282/001-0001
    Sponsors
    Sponsor organisation name
    Newcastle upon Tyne Hospitals
    Sponsor organisation address
    Freeman Hospital, Freeman Road, High Heaton, Newcastle upon Tyne, NE7 7DN, Newcastle, United Kingdom,
    Public contact
    Sean Scott, Newcastle upon Tyne Hospitals NHS Foundation Trust, +44 01912825969, Tnu-tr.sponsormanagement@nhs.net
    Scientific contact
    Sean Scott, Newcastle upon Tyne Hospitals NHS Foundation Trust, +44 01912825969, Tnu-tr.sponsormanagement@nhs.net
    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
    01 Jul 2020
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Jul 2021
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    To determine whether the severity of Shiga-toxin producing Escherichia Coli Haemolytic Syndrome (STEC HUS) is less in patients who receive eculizumab, compared to those given placebo.
    Protection of trial subjects
    All sites were provided with the ECUSTEC protocol that contained specific instruction relating to inclusion/exclusion criteria and trial patient safety. There was clear instruction relating to trial intevention safety and considerations detailed within the protocol. ECUSTEC had a Data Monitoring Committee to monitor patient safety throughout the trial.
    Background therapy
    		 The use of Ecu for the treatment of severe STEC HUS is increasing internationally, with no objective evidence of efficacy or safety in children or adults, and at a huge cost to the NHS and other health services. It is therefore important that the efficacy and safety of Ecu in STEC HUS is properly evaluated in a prospective randomised controlled trial. In our study, ECUSTEC, we assesed giving Ecu early in the disease course, have a wider objective, to consider reduction in the overall disease severity, rather than just renal disease severity, have a double-blind design and are examining fewer doses of Ecu. ECUSTEC also provide a health economic analysis to allow further assessment of the role of Ecu in managing STEC HUS in children.
    Evidence for comparator
    		 Eculizumab in Shiga-Toxin producing E. Coli Haemolytic Uraemic Syndrome (ECUSTEC): A Randomised, Double-Blind, Placebo-Controlled Trial. Recruited patients will receive either Eculizumab, formulation 10mg/ml concentrate for solution for infusion (30ml vial) or placebo (Sodium chloride 0.9%) formulation intravenous infusion bags. Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur. To reduce the risk of meningococcal disease, all ECUSTEC trial participants are given: 1. Antibiotic prophylaxis 2. Vaccination against meningococcus 3. Information on the early features of meningococcal diseases Patients will be followed-up for the duration of the treatment.
    Actual start date of recruitment
    11 Aug 2017
    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
    United Kingdom: 36
    Worldwide total number of subjects
    36
    EEA total number of subjects
    0
    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)
    7
    Children (2-11 years)
    26
    Adolescents (12-17 years)
    3
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 First participant was randomised on the 11th August 2017 and the last patient was randomised on the 7th February 2020. A total of 36 participants were randomised into ECUSTEC. Patients were equally recruited with 17 patient in the Eculizumab arm and 19 participants in the placebo arm.

    Pre-assignment
    Screening details
    		 A total of 108 participants were identified, of these identified 87 participants were approach and 36 were randomised.

    Period 1
    Period 1 title
    Baseline
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    All site personnel were blinded apart from those responsible for preparing the IMP (e.g. pharmacy), who made sure that no other person, had access to the study drugs and pharmacy documentation, and remained independent to the treatment of all trial participants. After randomisation, the unblinded staff received the treatment allocation electronically from BCTU. The unblinded staff prepared an intravenous (IV) infusion bag containing either sodium chloride 0.9% with Ecu o placebo (NaCl, 0.9%)

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab
    Arm description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur. To reduce the risk of meningococcal disease, all ECUSTEC trial participants are given: 1. Antibiotic prophylaxis 2. Vaccination against meningococcus 3. Information on the early features of meningococcal disease
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Arm title
    		 Placebo
    Arm description
    		 Placebo, Sodium chloride 0.9% Brand: any brand with marketing authorisation within EEA Formulation: Intravenous Infusion bags
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo, Sodium chloride 0.9%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for dispersion for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    5 to <10kg-60 ml of 0.9% Saline 10 to <20 Kg-120 ml of 0.9% Saline 20 to <40 kg-120 ml of 0.9% Saline ≥40kg -180 ml of 0.9% Saline

    Number of subjects in period 1
    		Eculizumab Placebo
    Started
    17
    19
    Completed
    17
    19
    Period 2
    Period 2 title
    30 days
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    All site personnel were blinded apart from those responsible for preparing the IMP (e.g. pharmacy), who made sure that no other person, had access to the study drugs and pharmacy documentation, and remained independent to the treatment of all trial participants. After randomisation, the unblinded staff received the treatment allocation electronically from BCTU. The unblinded staff prepared an intravenous (IV) infusion bag containing either sodium chloride 0.9% with Ecu o placebo (NaCl, 0.9%)]

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab
    Arm description
    		 Randomised, parallel group, double blind, placebo-controlled trial
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for injection/infusion
    Routes of administration
    Infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Arm title
    		 Placebo
    Arm description
    		 NaCL 0.9% saline infusion
    Arm type
    		 Placebo

    Investigational medicinal product name
    NaCl 0.9% saline infusion
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    0.9% saline

    Investigational medicinal product name
    Placebo, Sodium chloride 0.9%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for dispersion for infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    5 to <10kg-60 ml of 0.9% Saline 10 to <20 Kg-120 ml of 0.9% Saline 20 to <40 kg-120 ml of 0.9% Saline ≥40kg -180 ml of 0.9% Saline

    Number of subjects in period 2
    		Eculizumab Placebo
    Started
    17
    19
    Completed
    16
    19
    Not completed
    1
    0
         Adverse event, serious fatal
    1
    -
    Period 3
    Period 3 title
    60 days
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    All site personnel were blinded apart from those responsible for preparing the IMP (e.g. pharmacy), who made sure that no other person, had access to the study drugs and pharmacy documentation, and remained independent to the treatment of all trial participants. After randomisation, the unblinded staff received the treatment allocation electronically from BCTU. The unblinded staff prepared an intravenous (IV) infusion bag containing either sodium chloride 0.9% with Ecu o placebo (NaCl, 0.9%).

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab
    Arm description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Arm title
    		 Placebo
    Arm description
    		 Placebo, Sodium chloride 0.9%
    Arm type
    		 Placebo

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Investigational medicinal product name
    Placebo, Sodium chloride 0.9%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for dispersion for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    5 to <10kg-60 ml of 0.9% Saline 10 to <20 Kg-120 ml of 0.9% Saline 20 to <40 kg-120 ml of 0.9% Saline ≥40kg -180 ml of 0.9% Saline

    Number of subjects in period 3
    		Eculizumab Placebo
    Started
    16
    19
    Completed
    15
    19
    Not completed
    1
    0
         Consent withdrawn by subject
    1
    -
    Period 4
    Period 4 title
    26 Weeks
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    All site personnel were blinded apart from those responsible for preparing the IMP (e.g. pharmacy), who made sure that no other person, had access to the study drugs and pharmacy documentation, and remained independent to the treatment of all trial participants. After randomisation, the unblinded staff received the treatment allocation electronically from BCTU. The unblinded staff prepared an intravenous (IV) infusion bag containing either sodium chloride 0.9% with Ecu o placebo (NaCl, 0.9%)

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab
    Arm description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Arm title
    		 Placebo
    Arm description
    		 Placebo, Sodium chloride 0.9%
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo, Sodium chloride 0.9%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for dispersion for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    5 to <10kg-60 ml of 0.9% Saline 10 to <20 Kg-120 ml of 0.9% Saline 20 to <40 kg-120 ml of 0.9% Saline ≥40kg -180 ml of 0.9% Saline

    Number of subjects in period 4
    		Eculizumab Placebo
    Started
    15
    19
    Completed
    15
    19
    Period 5
    Period 5 title
    52 Weeks
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Carer, Assessor
    Blinding implementation details
    All site personnel were blinded apart from those responsible for preparing the IMP (e.g. pharmacy), who made sure that no other person, had access to the study drugs and pharmacy documentation, and remained independent to the treatment of all trial participants. After randomisation, the unblinded staff received the treatment allocation electronically from BCTU. The unblinded staff prepared an intravenous (IV) infusion bag containing either sodium chloride 0.9% with Ecu o placebo (NaCl, 0.9%)

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab
    Arm description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.
    Arm type
    		 Active comparator

    Investigational medicinal product name
    Eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Solution for injection/infusion
    Routes of administration
    Concentrate for solution for infusion
    Dosage and administration details
    The dose of Ecu and volume given will be dependent on the individual participant's bodyweight. 5 to <10kg-Dose of Eculizumab 300mg 10 to <20 Kg-Dose of Eculizumab 600mg 20 to <40 kg-Dose of Eculizumab 600 mg ≥40kg -Dose of Eculizumab 900mg

    Arm title
    		 Placebo
    Arm description
    		 Placebo, Sodium chloride 0.9%
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo, Sodium chloride 0.9%
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for dispersion for infusion
    Routes of administration
    Infusion
    Dosage and administration details
    5 to <10kg-60 ml of 0.9% Saline 10 to <20 Kg-120 ml of 0.9% Saline 20 to <40 kg-120 ml of 0.9% Saline ≥40kg -180 ml of 0.9% Saline

    Number of subjects in period 5
    		Eculizumab Placebo
    Started
    15
    19
    Completed
    15
    19

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Eculizumab
    Reporting group description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur. To reduce the risk of meningococcal disease, all ECUSTEC trial participants are given: 1. Antibiotic prophylaxis 2. Vaccination against meningococcus 3. Information on the early features of meningococcal disease

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo, Sodium chloride 0.9% Brand: any brand with marketing authorisation within EEA Formulation: Intravenous Infusion bags

    Reporting group values
    		 Eculizumab Placebo Total
    Number of subjects
    		 17 19 36
    Age categorical
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 3 4 7
        Children (2-11 years)
    		 13 13 26
        12 years-17 years
    		 1 2 3
    Gender categorical
    Units: Subjects
        Female
    		 10 10 20
        Male
    		 7 9 16
    pRIFLE category
    Units: Subjects
        Injury
    		 2 1 3
        Failure
    		 15 18 33
    Volume of 0.9% saline (ml/Kg)
    Units: Subjects
        <=20
    		 13 13 26
        >20
    		 4 6 10

    End points

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    End points reporting groups
    Reporting group title
    Eculizumab
    Reporting group description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur. To reduce the risk of meningococcal disease, all ECUSTEC trial participants are given: 1. Antibiotic prophylaxis 2. Vaccination against meningococcus 3. Information on the early features of meningococcal disease

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo, Sodium chloride 0.9% Brand: any brand with marketing authorisation within EEA Formulation: Intravenous Infusion bags
    Reporting group title
    Eculizumab
    Reporting group description
    		 Randomised, parallel group, double blind, placebo-controlled trial

    Reporting group title
    Placebo
    Reporting group description
    		 NaCL 0.9% saline infusion
    Reporting group title
    Eculizumab
    Reporting group description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo, Sodium chloride 0.9%
    Reporting group title
    Eculizumab
    Reporting group description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo, Sodium chloride 0.9%
    Reporting group title
    Eculizumab
    Reporting group description
    		 IMPs-Eculizumab Brand: Soliris Formulation: 10mg/ml Concentrate for solution for infusion (30ml vial). Ecu increases children’s susceptibility to meningococcal disease, particularly due to uncommon serogroups (e.g. Y, W and X), although meningococcal disease due to any serogroup (including B or C) may occur.

    Reporting group title
    Placebo
    Reporting group description
    		 Placebo, Sodium chloride 0.9%

    Primary: Clinical severity score (CSS)

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    End point title
    		 Clinical severity score (CSS)
    End point description
    End point type
    Primary
    End point timeframe
    Day 60
    End point values
    		 Eculizumab Placebo
    Number of subjects analysed
    15
    19
    Units: Score
        arithmetic mean (standard deviation)
    11.5 ( 8.4 )
    14.6 ( 7.7 )
    Statistical analysis title
    		 Mean CSS comparison
    Comparison groups
    Placebo v Eculizumab
    Number of subjects included in analysis
    34
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.05
    Method
    		 Regression, Linear
    Parameter type
    		 Mean difference (final values)
    Point estimate
    -2.5
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -7.8
         upper limit
    		 2.8

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    If an AE meets the criteria of a SAE for ECUSTEC and occurs within 90 days of the first dose of meningococcal vaccination or prophylactic antibiotic (whichever occurs first) it is reported to the trial office.
    Adverse event reporting additional description
    Events identified as SAEs require completion of an SAE form. A trial-specific SAE form is forwarded to BCTU within 24 hours of the research staff becoming aware of the event. Events categorised as Suspected Unexpected Serious Adverse Reactions (SUSARs) are reported to the Main REC and MHRA within the required timeframes.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 CTCAE
    Dictionary version
    4
    Reporting groups
    Reporting group title
    Eculizumab
    Reporting group description
    		 -

    Reporting group title
    Placebo
    Reporting group description
    		 -

    Serious adverse events
    		 Eculizumab Placebo
    Total subjects affected by serious adverse events
         subjects affected / exposed
    5 / 17 (29.41%)
    1 / 19 (5.26%)
         number of deaths (all causes)
    1
    0
         number of deaths resulting from adverse events
    0
    0
    Surgical and medical procedures
    Dyalisis
    Additional description: Serum amylase increased haemolytic uremic syndrome, general anaesthetic for dialysis central line
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 19 (5.26%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Brain injury
    Additional description: Death due to severe brain injury
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 19 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Horner syndrome
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 19 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood and lymphatic system disorders
    Anaemia
    Additional description: Anaemic
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 19 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Prologed NAT feeding post discharge
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 19 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Skin and subcutaneous tissue disorders
    rash
    Additional description: Rash to arm and legs
         subjects affected / exposed
    1 / 17 (5.88%)
    0 / 19 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Eculizumab Placebo
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 17 (17.65%)
    2 / 19 (10.53%)
    Immune system disorders
    Infection
    Additional description: The development of any significal infections (grade 3 or above)
         subjects affected / exposed
    0 / 17 (0.00%)
    1 / 19 (5.26%)
         occurrences all number
    0
    0
    Gastrointestinal disorders
    STEC
    Additional description: The presence of STEC in a stool sample which is collected at day 30
         subjects affected / exposed
    3 / 17 (17.65%)
    1 / 19 (5.26%)
         occurrences all number
    0
    0

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    12 Dec 2016
    Version 2.0 -Changes to the initial protocol requested by the MHRA including information about contraception, pregnancy testing, more frequent CNS examinations and SUSAR reporting.
    01 Apr 2017
    Version 3.0-Changes to incorporate those requested by REC for Version 1.0 7th September 2016 and MHRA requested changes for Version 2.0 12th December 2016 reviewed the updated stool SOP 016. Additional inclusion criteria and wording of an exclusion criteria. Further detail added regarding confirmation of vaccinations. Amendments to the assessments schedule, data collection, samples guidance and AE reporting sections. Other minor changes.
    18 Jan 2018
    Version 4.0-The treatment window has been extended by 12 hours due to the operational difficulty of treating patients. Other minor changes.
    24 Jun 2019
    Version 5.0- The wording for inclusion criteria 4 has been amended to include “OR Passage of blood per rectum within 14 days prior to diagnosis of HUS”. Also refined household contact to: Stool culture or shiga toxin polymerase chain reaction (PCR) or STEC serology result indicating STEC in a close contact (household or institutional). Other changes include an update to the UK Data Protection Act 2018, re-wording of events that do not require expedited reporting and other minor changes.
    26 May 2020
    Version 6.0- The treatment window has been extended by 24 hours due to the operational difficulty of treating patients within the current window. Other minor changes.

    Interruptions (globally)
    Were there any global interruptions to the trial? Yes
    Date
    Interruption
    Restart date
    27 Mar 2020
    COVID 19 Pandemic. Notification of suspension of recruitment. Due to the COVID-19 pandemic rapidly evolving situation within Trusts in the UK in March 2020, the strain that it was putting on our clinical collaborators, and current government adviced to stop unnecessary contact with other people. The trial team and trial steering committee chair agreed to suspend temporarily the ECUSTEC trial. The ECUSTEC trial team did not have confidence that children recruited in the remaining sites would have been able to attend their follow-up visits, particularly the crucial day 60 visit to collect the primary outcome data. For this reason, we proposed to suspend recruitment for an initial two month period. The situation was reviewed after the two month suspension and a further extension suspension was implemented.
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    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
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