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    Clinical Trial Results:
    A Phase I, Randomised, Open-Label, Multi-National Study to Evaluate the Pharmacokinetics of Repeated Once-Daily Intravenous Doses of Esomeprazole in Paediatric Patients 0 to 17 Years Old, Inclusive

    Summary
    EudraCT number
    		 2007-000628-41
    Trial protocol
    		 SE   HU   BE   Outside EU/EEA  
    Global end of trial date
    23 Feb 2010

    Results information
    Results version number
    		 v1(current)
    This version publication date
    01 Feb 2017
    First version publication date
    06 Aug 2015
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    D9615C00021
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    AstraZeneca LP
    Sponsor organisation address
    1800 Concord Pike, Wilmington, DE, United States, 19850
    Public contact
    AZ Clinical Trial Transparency group, AstraZeneca R&D, ClinicalTrialTransparency@astrazeneca.com
    Scientific contact
    Kurt Brown, MD, AstraZeneca LP, 1 302-885-0954,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-000331-PIP01-08
    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
    23 Feb 2010
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    23 Feb 2010
    Global end of trial reached?
    Yes
    Global end of trial date
    23 Feb 2010
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The primary objective of the study was to evaluate the pharmacokinetics of repeated doses of esomeprazole given as a once daily (qd) injection over 3 minutes in pediatric patients 0 to 17 years old, inclusive, by assessment of the total area under the plasma concentration versus time curve within a dosing interval (AUCτ) on Day 4 of the study based on population PK modeling.
    Protection of trial subjects
    This study was performed in accordance with the ethical principles that have their origin in the Declaration of Helsinki and that are consistent with International Conference on Harmonisation (ICH)/Good Clinical Practice (GCP) and applicable regulatory requirements and the AstraZeneca policy on Bioethics. The final clinical study protocol (CSP), including the final version of the Informed Consent Form, was approved by an Institutional Review Board (IRB) associated with each study center. The principal investigator at each center was to ensure that the patient and patient’s parent/guardian was given full and adequate oral and written information about the nature, purpose, possible risk, and benefit of the study. The patient and the patient’s parent/guardian were also to be notified that they were free to discontinue his/her child from the study at any time. The patient and patient’s parent/guardian were to be given the opportunity to ask questions and were allowed time to consider the information provided. The patient’s signed and dated assent (if appropriate) and the patient’s parent/guardian’s signed and dated informed consent had to be obtained before conducting any procedure specifically for the study. Patients could be discontinued from study treatment and assessments at any time at the discretion of the investigator(s).
    Background therapy
    		 The study included patients who were considered, in the judgment of the investigator, to be a candidate for acid suppression therapy. Concomitant use of other PPIs were allowed up to but not including the day of randomization.
    Evidence for comparator
    		 No comparator
    Actual start date of recruitment
    13 Oct 2007
    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
    Australia: 15
    Country: Number of subjects enrolled
    Hungary: 10
    Country: Number of subjects enrolled
    Sweden: 7
    Country: Number of subjects enrolled
    United States: 27
    Worldwide total number of subjects
    59
    EEA total number of subjects
    17
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    4
    Newborns (0-27 days)
    3
    Infants and toddlers (28 days-23 months)
    11
    Children (2-11 years)
    23
    Adolescents (12-17 years)
    18
    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 subject enrolled: 13 October 2007 Last subject last visit: 20 October 2009

    Pre-assignment
    Screening details
    		 62 patients screened , 3 patients failed to be eligible (did not meet inclusion/exclusion criteria) and were not randomized. Of the 59 randomised subjects, two subjects ( 1 newborn gestational age 32 weeks, and 1 adolscent 13 years old) were never dosed. Note: Safety population N=57 PK evaluable N=50;

    Period 1
    Period 1 title
    Randomized Dosing period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Subject
    Blinding implementation details
    Single Blind

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 0 - 1 month, 0.5 mg/kg
    Arm description
    		 Zero to one month old subjects dosed at 0.5 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 1 - 11 months, 1 mg/kg
    Arm description
    		 1 to 11 month old subjects dosed at 1 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 1 - 5 years, 10 mg
    Arm description
    		 1 to 5 year olds dosed at 10 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 6 - 11 years, 10 mg
    Arm description
    		 6 to 11 year old subjects dosed at 10 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 6 - 11 years, 20 mg
    Arm description
    		 6 to 11 year olds dosed at 20 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 12 - 17 years, 20 mg
    Arm description
    		 12 to 17 year olds dosed at 20 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 12 - 17 years, 40 mg
    Arm description
    		 12 to 17 year olds dosed at 40 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Number of subjects in period 1 [1]
    		0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Started
    6
    9
    8
    8
    9
    8
    9
    Completed
    6
    8
    7
    8
    7
    6
    8
    Not completed
    0
    1
    1
    0
    2
    2
    1
         Adverse event, non-fatal
    -
    -
    1
    -
    1
    -
    -
         Not specified
    -
    1
    -
    -
    1
    2
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: Of the 59 randomised subjects, two subjects ( 1 newborn gestational age 32 weeks, and 1 adolscent 13 years old) were never dosed. Thus, the number of subjects in the baseline period (ITT) is 57 while the worldwide number enrolled is 59.
    Period 2
    Period 2 title
    Pharmacokinetic Evaluation
    Is this the baseline period?
    		 No
    Allocation method
    Randomised - controlled
    Blinding used
    		 Single blind
    Roles blinded
    		 Subject
    Blinding implementation details
    Single Blind

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 0 - 1 month, 0.5 mg/kg
    Arm description
    		 Zero to one month old subjects dosed at 0.5 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV administration

    Arm title
    		 1 - 11 months, 1 mg/kg
    Arm description
    		 1 to 11 month old subjects dosed at 1 mg/kg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Arm title
    		 1 - 5 years, 10 mg
    Arm description
    		 1 to 5 year olds dosed at 10 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for solution for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Arm title
    		 6 - 11 years, 10 mg
    Arm description
    		 6 to 11 year old subjects dosed at 10 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Arm title
    		 6 - 11 years, 20 mg
    Arm description
    		 6 to 11 year olds dosed at 20 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Arm title
    		 12 - 17 years, 20 mg
    Arm description
    		 12 to 17 year olds dosed at 20 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Arm title
    		 12 - 17 years, 40 mg
    Arm description
    		 12 to 17 year olds dosed at 40 mg
    Arm type
    		 Experimental

    Investigational medicinal product name
    Esomeprazole sodium
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Powder for injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    IV

    Number of subjects in period 2
    		0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Started
    6
    7
    7
    8
    8
    6
    8
    Completed
    6
    7
    7
    8
    8
    6
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    0 - 1 month, 0.5 mg/kg
    Reporting group description
    		 Zero to one month old subjects dosed at 0.5 mg/kg

    Reporting group title
    1 - 11 months, 1 mg/kg
    Reporting group description
    		 1 to 11 month old subjects dosed at 1 mg/kg

    Reporting group title
    1 - 5 years, 10 mg
    Reporting group description
    		 1 to 5 year olds dosed at 10 mg

    Reporting group title
    6 - 11 years, 10 mg
    Reporting group description
    		 6 to 11 year old subjects dosed at 10 mg

    Reporting group title
    6 - 11 years, 20 mg
    Reporting group description
    		 6 to 11 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 20 mg
    Reporting group description
    		 12 to 17 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 40 mg
    Reporting group description
    		 12 to 17 year olds dosed at 40 mg

    Reporting group values
    		 0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg Total
    Number of subjects
    		 6 9 8 8 9 8 9 57
    Age Categorical
    Subjects in Pharmacokinetic data set
    Units: Subjects
        Newborns (0-27 days)
    		 4 0 0 0 0 0 0 4
        Infants and toddlers (28 days-23 months)
    		 2 9 2 0 0 0 0 13
        Children (2-11 years)
    		 0 0 6 8 9 0 0 23
        Adolescents (12-17 years)
    		 0 0 0 0 0 8 9 17
    Age Continuous
    Median Age in months or year depending on treatment arm classification
    Units: years
        median (full range (min-max))
    		 4 (2 to 36) 5 (1 to 7) 2 (1 to 5) 7.5 (6 to 11) 8 (6 to 11) 15.5 (13 to 17) 15.5 (13 to 17) -
    Gender Categorical
    Units: Subjects
        Male
    		 4 6 5 5 5 5 3 33
        Female
    		 2 3 3 3 4 3 6 24
    Subject analysis sets

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects that received at least one dose of study drug

    Subject analysis sets values
    		 Safety Analysis Set
    Number of subjects
    57
    Age Categorical
    Subjects in Pharmacokinetic data set
    Units: Subjects
        Newborns (0-27 days)
    4
        Infants and toddlers (28 days-23 months)
    13
        Children (2-11 years)
    23
        Adolescents (12-17 years)
    17
    Age Continuous
    Median Age in months or year depending on treatment arm classification
    Units: years
        median (full range (min-max))
    6 (1 to 17)
    Gender Categorical
    Units: Subjects
        Male
    33
        Female
    24

    End points

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    End points reporting groups
    Reporting group title
    0 - 1 month, 0.5 mg/kg
    Reporting group description
    		 Zero to one month old subjects dosed at 0.5 mg/kg

    Reporting group title
    1 - 11 months, 1 mg/kg
    Reporting group description
    		 1 to 11 month old subjects dosed at 1 mg/kg

    Reporting group title
    1 - 5 years, 10 mg
    Reporting group description
    		 1 to 5 year olds dosed at 10 mg

    Reporting group title
    6 - 11 years, 10 mg
    Reporting group description
    		 6 to 11 year old subjects dosed at 10 mg

    Reporting group title
    6 - 11 years, 20 mg
    Reporting group description
    		 6 to 11 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 20 mg
    Reporting group description
    		 12 to 17 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 40 mg
    Reporting group description
    		 12 to 17 year olds dosed at 40 mg
    Reporting group title
    0 - 1 month, 0.5 mg/kg
    Reporting group description
    		 Zero to one month old subjects dosed at 0.5 mg/kg

    Reporting group title
    1 - 11 months, 1 mg/kg
    Reporting group description
    		 1 to 11 month old subjects dosed at 1 mg/kg

    Reporting group title
    1 - 5 years, 10 mg
    Reporting group description
    		 1 to 5 year olds dosed at 10 mg

    Reporting group title
    6 - 11 years, 10 mg
    Reporting group description
    		 6 to 11 year old subjects dosed at 10 mg

    Reporting group title
    6 - 11 years, 20 mg
    Reporting group description
    		 6 to 11 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 20 mg
    Reporting group description
    		 12 to 17 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 40 mg
    Reporting group description
    		 12 to 17 year olds dosed at 40 mg

    Subject analysis set title
    Safety Analysis Set
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects that received at least one dose of study drug

    Primary: AUCτ

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    End point title
    		 AUCτ [1]
    End point description
    Area Under the Curve (AUC) of esomeprazole
    End point type
    Primary
    End point timeframe
    Day 4 steady state
    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: NSAE includes those events that occurred in at least 2 patients during the study
    End point values
    		 0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Number of subjects analysed
    6
    7
    7
    8
    8
    6
    8
    Units: μmol*h/L
        geometric mean (full range (min-max))
    7.5 (4.5 to 20.5)
    10.5 (4.5 to 22.2)
    7.9 (2.9 to 16.6)
    6.9 (3.5 to 10.9)
    14.4 (7.2 to 42.3)
    8.1 (4.7 to 15.9)
    17.6 (13.1 to 19.8)
    No statistical analyses for this end point

    Secondary: Css,max

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    End point title
    		 Css,max
    End point description
    End point type
    Secondary
    End point timeframe
    Day 4 steady state
    End point values
    		 0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Number of subjects analysed
    6
    7
    7
    8
    8
    6
    8
    Units: μmol/L
        geometric mean (full range (min-max))
    3.71 (2.73 to 5.77)
    8.68 (4.51 to 14)
    9.37 (4.4 to 17.2)
    5.6 (3.13 to 13.2)
    8.83 (3.36 to 29.4)
    7.1 (4.76 to 9.02)
    10.5 (7.82 to 14.2)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dosing to day 5
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    12.0
    Reporting groups
    Reporting group title
    0 - 1 month, 0.5 mg/kg
    Reporting group description
    		 Zero to one month old subjects dosed at 0.5 mg/kg

    Reporting group title
    1 - 11 months, 1 mg/kg
    Reporting group description
    		 1 to 11 month old subjects dosed at 1 mg/kg

    Reporting group title
    1 - 5 years, 10 mg
    Reporting group description
    		 1 to 5 year olds dosed at 10 mg

    Reporting group title
    6 - 11 years, 10 mg
    Reporting group description
    		 6 to 11 year old subjects dosed at 10 mg

    Reporting group title
    6 - 11 years, 20 mg
    Reporting group description
    		 6 to 11 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 20 mg
    Reporting group description
    		 12 to 17 year olds dosed at 20 mg

    Reporting group title
    12 - 17 years, 40 mg
    Reporting group description
    		 12 to 17 year olds dosed at 40 mg

    Serious adverse events
    		 0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Total subjects affected by serious adverse events
         subjects affected / exposed
    2 / 6 (33.33%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    0
    Gastrointestinal disorders
    Gastroenteritis
    Additional description: Gastroenteritis with abdominal pain, dehydration, nausea, and vomiting
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Colitis ulcerative
    Additional description: Ulcerative colitis flare
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Bronchial hyper-reactivity
    Additional description: Reactive airway disease (secondary to Influenza A)
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Acute respiratory distress syndrome
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Acute respiratory failuree
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Candida sepsis
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 0 - 1 month, 0.5 mg/kg 1 - 11 months, 1 mg/kg 1 - 5 years, 10 mg 6 - 11 years, 10 mg 6 - 11 years, 20 mg 12 - 17 years, 20 mg 12 - 17 years, 40 mg
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 6 (33.33%)
    5 / 9 (55.56%)
    7 / 8 (87.50%)
    5 / 8 (62.50%)
    2 / 9 (22.22%)
    5 / 8 (62.50%)
    5 / 9 (55.56%)
    Cardiac disorders
    Tachycardia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    1
    0
    Blood and lymphatic system disorders
    Anemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    1
    0
    General disorders and administration site conditions
    Catheter related complication
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    1
    0
    0
    0
    0
    Infusion site extravasation
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    2 / 8 (25.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
         occurrences all number
    0
    2
    2
    1
    0
    1
    1
    Abdominal distension
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    0
    Abdominal pain
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    2 / 8 (25.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    2
    0
    0
    0
    0
    Diarrhea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    0
    Gastroesophageal reflux disease
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    0
    0
    1
    0
    Nausea
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    1 / 8 (12.50%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    0
    Vomiting
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    2
    0
    1
    0
    0
    Skin and subcutaneous tissue disorders
    Pruritus
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    1
    Erythema
         subjects affected / exposed
    0 / 6 (0.00%)
    3 / 9 (33.33%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    3
    0
    0
    0
    0
    0
    Rash
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    1
    1
    0
    0
    0
    1
    Psychiatric disorders
    Agitation
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 9 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    0 / 6 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    2 / 8 (25.00%)
    1 / 9 (11.11%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    1
    Infections and infestations
    Pyrexia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    2 / 8 (25.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    1
    0
    1
    2
    0
    Pneumonia
         subjects affected / exposed
    0 / 6 (0.00%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    1 / 9 (11.11%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    0
    Metabolism and nutrition disorders
    Hypokalemia
         subjects affected / exposed
    1 / 6 (16.67%)
    0 / 9 (0.00%)
    1 / 8 (12.50%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    1
    0
    1
    0
    0
    0
    0
    Hyponatremia
         subjects affected / exposed
    0 / 6 (0.00%)
    2 / 9 (22.22%)
    0 / 8 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
    0 / 8 (0.00%)
    0 / 9 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Jun 2007
    Incorporating multiple changes to CSP in response to FDA comments
    29 Jan 2008
    New centers added to help meet enrollment goals; amendment to exclusion criteria
    18 Dec 2008
    Broadening of some inclusion/exclusion criteria

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

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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