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    Clinical Trial Results:
    Early treatment of pediatric patients with typical hemolytic uremic syndrome with the anti-C5 monoclonal antibody eculizumab: a phase III prospective, randomized, placebo-controlled clinical trial

    Summary
    EudraCT number
    		 2014-001169-28
    Trial protocol
    		 FR  
    Global end of trial date
    26 Jun 2019

    Results information
    Results version number
    		 v1(current)
    This version publication date
    27 Jun 2026
    First version publication date
    27 Jun 2026
    Other versions
    Summary report(s)
    		 Adverse event analysis report

    Trial information

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    Trial identification
    Sponsor protocol code
    13705201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02205541
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Direction de la Recherche et de l'Innovation
    Sponsor organisation address
    2 Rue Charles Viguerie, Toulouse, France, 31059
    Public contact
    Caroline PEYROT, Centre Hospitalier Universitaire de Toulouse, DRI, Hôtel-Dieu, +33 561778486, peyrot.c@chu-toulouse.fr
    Scientific contact
    Arnaud GARNIER, Hôpital des Enfants - Centre Hospitalier Universitaire Purpan , +33 534558594, garnier.a@chu-toulouse.fr
    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
    21 Apr 2022
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    26 Jun 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the impact of early eculizumab (ECZ) treatment on the progression of acute kidney injury (IRA) in children with STEC-associated hemolytic uremic syndrome (SHU-STEC) in the context of a prospective, placebo-controlled therapeutic trial.
    Protection of trial subjects
    The participants in the clinical trial were protected by a complete vaccination schedule including a tetravalent meningococcal vaccine, a meningococcal B vaccine, and oral antibiotic prophylaxis. The study was designed to limit eculizumab (ECZ) treatment to a short duration (maximum 5 weeks) in order to reduce the risks of side effects and infections. All the local regulatory requirements pertinent to safety of trial participants were followed.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    08 Jul 2015
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 100
    Worldwide total number of subjects
    100
    EEA total number of subjects
    100
    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)
    100
    Adolescents (12-17 years)
    0
    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
    		 Recruitment planned over 38 months in 18 pediatric nephrology units across France. Estimated 200 hospitalizations of eligible patients during this period to reach 100 inclusions. Recruitment coordinated via the Société de Néphrologie Pédiatrique (SNP).

    Pre-assignment
    Screening details
    		 Screening in hospitalized pediatric patients with STEC-HUS and AKI. Exclusion of patients with severe extra-renal involvement. Written consent from both parents required. Approximatively 200 patients screened to include 100. Vaccination and prophylaxis mandatory before treatment.

    Pre-assignment period milestones
    Number of subjects started
    		 100
    Number of subjects completed
    		 100

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Subject

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Eculizumab treatment arm
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    eculizumab
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Eculizumab IV infusion over 1 hour. Dosage based on weight: 300–1200 mg. Administered at D0, D7, D14, D21, D28 depending on patient weight. Pre-treatment includes mandatory meningococcal vaccination and oral antibiotic prophylaxis until 60 days post last dose.

    Arm title
    		 Placebo arm
    Arm description
    		 -
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Concentrate for solution for infusion
    Routes of administration
    Intravenous use
    Dosage and administration details
    Placebo: 5% glucose IV infusion over 1 hour, matching eculizumab schedule (D0, D7, D14, D21, D28) based on patient weight. Pre-treatment includes same vaccination and oral antibiotic prophylaxis as active arm, maintained for 60 days after last infusion.

    Number of subjects in period 1
    		Eculizumab treatment arm Placebo arm
    Started
    50
    50
    Completed
    44
    42
    Not completed
    6
    8
         Consent withdrawn by subject
    1
    1
         Physician decision
    1
    -
         Adverse event, non-fatal
    3
    2
         Reason not specified
    -
    1
         Protocol deviation
    1
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Eculizumab treatment arm
    Reporting group description
    		 -

    Reporting group title
    Placebo arm
    Reporting group description
    		 -

    Reporting group values
    		 Eculizumab treatment arm Placebo arm Total
    Number of subjects
    		 50 50 100
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 3.89 ( 3 ) 3.28 ( 2.32 ) -
    Gender categorical
    Units: Subjects
        Female
    		 31 23 54
        Male
    		 19 27 46

    End points

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    End points reporting groups
    Reporting group title
    Eculizumab treatment arm
    Reporting group description
    		 -

    Reporting group title
    Placebo arm
    Reporting group description
    		 -

    Primary: Efficacy analysis

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    End point title
    		 Efficacy analysis
    End point description
    Occurrence of renal replacement therapy (hemodialysis, hemofiltration, or peritoneal dialysis) lasting 48 hours or more (binary yes/no criterion) after treatment initiation
    End point type
    Primary
    End point timeframe
    From Visit 1 ( inclusion visit) through Visit 6 (last treatment visit)
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: Number of participants with EER ≥ 48h
        Success
    19
    24
        Failure
    31
    26
    Statistical analysis title
    		 Primary endpoint analysis
    Comparison groups
    Placebo arm v Eculizumab treatment arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.313
    Method
    		 Chi-squared
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    1.506
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 0.679
         upper limit
    		 3.339

    Secondary: Safety and tolerability profile of the treatment

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    End point title
    		 Safety and tolerability profile of the treatment
    End point description
    End point type
    Secondary
    End point timeframe
    Throughout the study , an average of 1 year
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50 [1]
    50 [2]
    Units: Number of adverse events per group
    297
    253
    Notes
    [1] - The actual number of people who have received Eculizumab treatment is 54
    [2] - The actual number of people who have received Placebo is 46
    No statistical analyses for this end point

    Secondary: Duration of acute kidney injury (creatinine)

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    End point title
    		 Duration of acute kidney injury (creatinine)
    End point description
    The result is then described by estimating the coefficient β with a 95% confidence interval, corresponding to the average difference in creatinine level between the groups (ECZ group and Placebo group) between V2 and V7.
    End point type
    Secondary
    End point timeframe
    From V2 to V7
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: µmol/L
        least squares mean (confidence interval 95%)
    9.72 (-9.65 to 29.09)
    9.72 (-9.65 to 29.09)
    Statistical analysis title
    		 SEP 2A analysis
    Statistical analysis description
    SEP : Secondary endpoint
    Comparison groups
    Placebo arm v Eculizumab treatment arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.325
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Duration of acute kidney injury (creatinine clairance)

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    End point title
    		 Duration of acute kidney injury (creatinine clairance)
    End point description
    The result is then described by estimating the coefficient β with a 95% confidence interval, corresponding to the average difference in creatinine clairance between the groups (ECZ group and Placebo group) between V2 and V7.
    End point type
    Secondary
    End point timeframe
    From V2 to V7
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: (mL/min/1.73m2)
        least squares mean (confidence interval 95%)
    -1.43 (-11.16 to 8.3)
    -1.43 (-11.16 to 8.6)
    Statistical analysis title
    		 SEP 2B analysis
    Statistical analysis description
    SEP : Secondary endpoint
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.773
    Method
    		 Mixed models analysis
    Confidence interval

    Secondary: Renal sequelae

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    End point title
    		 Renal sequelae
    End point description
    Evaluation of renal sequelae through the number of children with renal sequelae (definition of renal sequelae based on blood pressure measurement, creatinine clearance, proteinuria and microalbuminuria as ProtU/creatU>0.02) 6 months and 12 months after the last injection of ECZ or placebo. Renal sequelae : defined by a ProtU/creatU ratio > 0.02 .
    End point type
    Secondary
    End point timeframe
    at V8 (6 months post treatment) & at V9 (12 months post treatment)
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50 [3]
    50 [4]
    Units: participants with renal sequelae
    number (not applicable)
        12 months post treatment
    20
    29
        6 months post treatment
    26
    21
    Notes
    [3] - number of children with presence of renal sequelae
    [4] - number of children with presence of renal sequelae
    Statistical analysis title
    		 SEP 3 analysis (6 months)
    Statistical analysis description
    SEP : Secondary endpoint
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.394
    Method
    		 Chi-squared
    Confidence interval
    Statistical analysis title
    		 SEP 3 analysis (12 months)
    Statistical analysis description
    SEP : Secondary endpoint
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.045
    Method
    		 Chi-squared
    Confidence interval

    Secondary: Evolution of hematological abnormalities (LDH level)

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    End point title
    		 Evolution of hematological abnormalities (LDH level)
    End point description
    Evaluation of the evolution of hematological abnormalities based on the evolution of the difference in LDH levels between the experimental group and the placebo group between visits V2 and V7
    End point type
    Secondary
    End point timeframe
    from V2 to V7
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: UI/l
    arithmetic mean (confidence interval 95%)
        LDH level at V2
    3308.264 (2706.485 to 3910.043)
    2537.549 (2120.574 to 2954.523)
        LDH level at V7
    281.65 (203.641 to 359.66)
    340.663 (276.341 to 404.985)
    No statistical analyses for this end point

    Secondary: Evolution of hematological abnormalities (platelet count)

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    End point title
    		 Evolution of hematological abnormalities (platelet count)
    End point description
    Evaluation of the evolution of hematological abnormalities based on the platelet count in each group at visits V3 and V4
    End point type
    Secondary
    End point timeframe
    At V3 and V4
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: G/L
    arithmetic mean (standard deviation)
        Platelet count at V3
    278.71 ( 167.62 )
    266.84 ( 145.40 )
        Platelet count at V4
    393.98 ( 127.92 )
    366.1 ( 144.27 )
    Statistical analysis title
    		 SEP 4B analysis (V3)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.7223
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 SEP 4B analysis (V4)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.3507
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: Evolution of hematological abnormalities ( schistocytes count)

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    End point title
    		 Evolution of hematological abnormalities ( schistocytes count)
    End point description
    Evaluation of the evolution of hematological abnormalities based on the schistocytes count in each group at visits V3 and V4
    End point type
    Secondary
    End point timeframe
    at V3 & V4
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: percentage of schistocyte
    arithmetic mean (standard deviation)
        Schistocytes count at V3
    5.67 ( 9 )
    3.83 ( 2.17 )
        Schistocytes count at V4
    2.67 ( 1.36 )
    2.4 ( 1.36 )
    Statistical analysis title
    		 SEP 4C analysis (V3)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.333
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval
    Statistical analysis title
    		 SEP 4C analysis (V4)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.333
    Method
    		 Wilcoxon (Mann-Whitney)
    Confidence interval

    Secondary: Evolution of hematological abnormalities ( hemoglobin level)

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    End point title
    		 Evolution of hematological abnormalities ( hemoglobin level)
    End point description
    Evaluation of the evolution of hematological abnormalities based on the evolution of the difference in hemoglobin levels between the experimental group and the placebo group between visits V2 and V7
    End point type
    Secondary
    End point timeframe
    From V2 to V7
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: G/dL
    arithmetic mean (confidence interval 95%)
        Hemoglobin level at V2
    9.096 (8.572 to 9.620)
    8.329 (7.958 to 8.7)
        hemoglobin level at V7
    11.403 (11.131 to 11.674)
    11.73 (11.468 to 11.991)
    No statistical analyses for this end point

    Secondary: Evolution of hematological abnormalities (red blood cell transfusion)

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    End point title
    		 Evolution of hematological abnormalities (red blood cell transfusion)
    End point description
    Evaluation of the evolution of hematological abnormalities based on information regarding the use of red blood cell transfusion in each group after the initiation of treatment. The measures concern the number of participants who required a transfusion (Yes/No) after the initiation of treatment.
    End point type
    Secondary
    End point timeframe
    After the initiation of treatment
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: number of participants
        Yes
    36
    35
        No
    13
    13
    Statistical analysis title
    		 SEP 4E analysis
    Comparison groups
    Placebo arm v Eculizumab treatment arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.951
    Method
    		 Chi-squared
    Confidence interval

    Secondary: Evolution of hematological abnormalities (platelet transfusion)

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    End point title
    		 Evolution of hematological abnormalities (platelet transfusion)
    End point description
    Evaluation of the evolution of hematological abnormalities based on information regarding the use of platelet transfusion in each group after the initiation of treatment. The measures concern the number of participants who required a transfusion (Yes/No) after the initiation of treatment.
    End point type
    Secondary
    End point timeframe
    After the initiation of the treatment
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: number of participants
        Yes
    5
    6
        No
    44
    42
    Statistical analysis title
    		 SEP 4F analysis
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.721
    Method
    		 Chi-squared
    Confidence interval

    Secondary: Evolution of biological parameters of VAC activation (plasma dosage)

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    End point title
    		 Evolution of biological parameters of VAC activation (plasma dosage)
    End point description
    Evaluation of the biological parameters of VAC activation based on the plasma measurement of the C3 fraction in each group at V3 and V5
    End point type
    Secondary
    End point timeframe
    At V3 & V5
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: mg/ml
    arithmetic mean (standard deviation)
        C3 fraction at V3
    1345.86 ( 293.6 )
    1396.06 ( 381.71 )
        C3 fraction at V5
    1233.81 ( 274.78 )
    1157.5 ( 290.88 )
    Statistical analysis title
    		 SEP 5A analysis (V3)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.537
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 SEP 5A analysis (V4)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.274
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: Evolution of biological parameters of VAC activation (membrane expression of CD46)

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    End point title
    		 Evolution of biological parameters of VAC activation (membrane expression of CD46)
    End point description
    Evaluation of the biological parameters of VAC activation based on the plasma measurement of the membrane expression of CD46 in each group at V3 and V5
    End point type
    Secondary
    End point timeframe
    At V3 & V5
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: MFI
    arithmetic mean (standard deviation)
        Membrane expression of CD46 at V3
    14.96 ( 2.92 )
    15.43 ( 3.31 )
        Membrane expression of CD46 at V5
    15.89 ( 2.69 )
    14.88 ( 3.2 )
    Statistical analysis title
    		 SEP 5B analysis (V3)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.54
    Method
    		 t-test, 2-sided
    Confidence interval
    Statistical analysis title
    		 SEP 5B analysis (V5)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.207
    Method
    		 t-test, 2-sided
    Confidence interval

    Secondary: CAM inhibition (CH50 measurement)

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    End point title
    		 CAM inhibition (CH50 measurement)
    End point description
    CAM inhibition assessed by CH50 measurement in each group at visits V3 and V5. The measures concern the number of participants with a CH 50 ≤ to 20%.
    End point type
    Secondary
    End point timeframe
    At V3 & V5
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: Number of participants
        participants with a CH 50 ≤ to 20% at V3
    19
    1
        participants with a CH 50 ≤ to 20% at V5
    16
    2
    Statistical analysis title
    		 SEP 6A analysis (V3)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 > 0.001
    Method
    		 Chi-squared
    Confidence interval
    Statistical analysis title
    		 SEP 6A analysis (V5)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.001
    Method
    		 Chi-squared
    Confidence interval

    Secondary: CAM inhibition ( free ECZ)

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    End point title
    		 CAM inhibition ( free ECZ)
    End point description
    CAM inhibition assessed by free ECZ plasma assays in each group at visits V3 and V5. The evaluation of this endpoint was only performed for the Eculizumab group.
    End point type
    Secondary
    End point timeframe
    At V3 & V5
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: mg/l
    arithmetic mean (standard deviation)
        free ECZ plasma assays at V3
    252.63 ( 181.2 )
    0 ( 0 )
        free ECZ plasma assays at V5
    254.24 ( 191.49 )
    0 ( 0 )
    No statistical analyses for this end point

    Secondary: Incidence of extra-renal manifestations

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    End point title
    		 Incidence of extra-renal manifestations
    End point description
    The measures concern the number of cases of neurological, cardiac, and digestive disorders during initial hospitalization and after the initiation of treatment.
    End point type
    Secondary
    End point timeframe
    Throughout the study
    End point values
    		 Eculizumab treatment arm Placebo arm
    Number of subjects analysed
    50
    50
    Units: number of cases
        Neurological impairement
    4
    6
        Cardiac disorder
    1
    5
        Digestive disorder
    9
    12
    Statistical analysis title
    		 SEP 7 analysis (neurologic)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.52
    Method
    		 Fisher exact
    Confidence interval
    Statistical analysis title
    		 SEP 7 analysis (cardiac)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.111
    Method
    		 Fisher exact
    Confidence interval
    Statistical analysis title
    		 SEP 7 analysis (digestive)
    Comparison groups
    Eculizumab treatment arm v Placebo arm
    Number of subjects included in analysis
    100
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.459
    Method
    		 Chi-squared
    Confidence interval

    Adverse events

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    Adverse events information [1]
    Timeframe for reporting adverse events
    From first study drug administration up to the end of the 12-month follow-up period, including the safety visits at Months 1, 6, and 12.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.0
    Reporting groups
    Reporting group title
    AE Eculizumab arm
    Reporting group description
    		 The number of people in this reporting group corresponds to the number of patients who actually received eculizumab treatment.

    Reporting group title
    AE Placebo arm
    Reporting group description
    		 The number of people in this reporting group corresponds to the number of patients who actually received placebo.

    Notes
    [1] - There are no non-serious adverse events recorded for these results. It is expected that there will be at least one non-serious adverse event reported.
    Justification: Due to the large number of AEs, the list and the number of AEs (serious and non-serious) collected during the study is presented in the adverse event analysis report attached in the "summary attachment" section.
    Serious adverse events
    		 AE Eculizumab arm AE Placebo arm
    Total subjects affected by serious adverse events
         subjects affected / exposed
    11 / 54 (20.37%)
    7 / 46 (15.22%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Investigations
    Pancreatic enzymes increased
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Vascular disorders
    Venous thrombosis
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Cardiac disorder
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Paralysis
         subjects affected / exposed
    0 / 54 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Tonic clonic movements
         subjects affected / exposed
    0 / 54 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorder
         subjects affected / exposed
    1 / 54 (1.85%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Status epilepticus
         subjects affected / exposed
    2 / 54 (3.70%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    General disorders and administration site conditions
    Fever
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Oedema
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Gastrointestinal disorder
         subjects affected / exposed
    2 / 54 (3.70%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Abdominal discomfort
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Pulmonary disorder
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Hepatocellular injury
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Renal and urinary disorders
    Proteinuria
         subjects affected / exposed
    1 / 54 (1.85%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Otitis media acute
         subjects affected / exposed
    0 / 54 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastroenteritis
         subjects affected / exposed
    0 / 54 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pyelonephritis
         subjects affected / exposed
    1 / 54 (1.85%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Peritonitis
         subjects affected / exposed
    1 / 54 (1.85%)
    0 / 46 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 AE Eculizumab arm AE Placebo arm
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 54 (0.00%)
    0 / 46 (0.00%)

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    26 Jan 2016
    Update: - of the drug circuit (labeling) - of the conditions imposed by the risk management plan to which Soliris® is subject (vaccinations and antibiotic prophylaxis) - of the dosages performed at visits 7, 8, and 9 (harmonization between the flowchart and the visit descriptions) - of the randomization procedure - of the conditions for patient participation in another study and discontinuation of the research - of the timelines for reporting adverse events - of the list of investigators Removal of one investigation site (Réunion)
    23 Mar 2017
    Extension of the inclusion period and therefore of the study duration by 14 months.
    18 Sep 2017
    Addition of a co-coordinator due to the absence of the principal investigator coordinator of the study

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    Premature termination leading to a small number of analyzed subjects
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