Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43977   clinical trials with a EudraCT protocol, of which   7312   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A Multi-centre, Phase I, Open-label, Single-dose Study to Investigate Pharmacokinetics (PK) of Ticagrelor in Infants and Toddlers, Aged 0 to less than 24 Months, with Sickle Cell Disease (HESTIA4)

    Summary
    EudraCT number
    2017-003641-14
    Trial protocol
    BE   ES   GB   IT  
    Global end of trial date
    07 May 2019

    Results information
    Results version number
    v1(current)
    This version publication date
    06 Nov 2019
    First version publication date
    06 Nov 2019
    Other versions

    Trial information

    Close Top of page
    Trial identification
    Sponsor protocol code
    D5136C00010
    Additional study identifiers
    ISRCTN number
    -
    US NCT number
    NCT03492931
    WHO universal trial number (UTN)
    -
    Sponsors
    Sponsor organisation name
    AstraZeneca
    Sponsor organisation address
    950 Wind River Ln, Gaithersburg, MD, United States, 20878
    Public contact
    Global Clinical Lead, AstraZeneca, +1 3028851180, ClinicalTrialTransparency@astrazeneca.com
    Scientific contact
    Global Clinical Lead, AstraZeneca, +1 3028851180, ClinicalTrialTransparency@astrazeneca.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    EMEA-000480-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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    07 May 2019
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    07 May 2019
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    D5136C00010 is part of a paediatric development programme and is the first study with ticagrelor in paediatric patients aged 0 months to <24 months with sickle cell disease (SCD) to characterise pharmacokinetic (PK) properties after single doses.
    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 Council for Harmonisation/Good Clinical Practice, applicable regulatory requirements and the AstraZeneca policy on Bioethics.
    Background therapy
    -
    Evidence for comparator
    -
    Actual start date of recruitment
    28 Mar 2018
    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
    Belgium: 3
    Country: Number of subjects enrolled
    Italy: 1
    Country: Number of subjects enrolled
    Kenya: 7
    Country: Number of subjects enrolled
    Lebanon: 7
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    United Kingdom: 2
    Worldwide total number of subjects
    21
    EEA total number of subjects
    7
    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)
    21
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    Close Top of page
    Recruitment
    Recruitment details
    Paediatric patients aged 0 to <24 months with SCD were recruited to this Phase I open-label, single-dose study at 8 study centres in Belgium, Italy, Kenya, Lebanon, Spain and the United Kingdom. The first patient started in March 2018 and the last patient completed in May 2019.

    Pre-assignment
    Screening details
    Patients were diagnosed with homozygous sickle cell anaemia or sickle beta-zero-thalassaemia and had a body weight of at least 5 kilograms (kg) at screening. Patients participating in this study were not withheld from any other standard of care treatments that may be used in SCD.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    Ticagrelor 0.1 mg/kg: <6 months old
    Arm description
    Patients in the age group <6 months old received a single oral dose of 0.1 milligrams per kg (mg/kg) ticagrelor.
    Arm type
    Experimental

    Investigational medicinal product name
    Ticagrelor
    Investigational medicinal product code
    Other name
    BRILINTA™
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    A single oral 0.1 mg/kg dose of ticagrelor. Before administration, ticagrelor granules were constituted with 10 millilitres (mL) of purified water to form a homogenous suspension of 1 mg/mL ticagrelor.

    Arm title
    Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Arm description
    Patients in the age group 6 to <12 months old received a single oral dose of 0.2 mg/kg ticagrelor.
    Arm type
    Experimental

    Investigational medicinal product name
    Ticagrelor
    Investigational medicinal product code
    Other name
    BRILINTA™
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    A single oral 0.2 mg/kg dose of ticagrelor. Before administration, ticagrelor granules were constituted with 10 mL of purified water to form a homogenous suspension of 1 mg/mL ticagrelor.

    Arm title
    Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Arm description
    Patients in the age group 12 to <24 months old received a single oral dose of 0.2 mg/kg ticagrelor.
    Arm type
    Experimental

    Investigational medicinal product name
    Ticagrelor
    Investigational medicinal product code
    Other name
    BRILINTA™
    Pharmaceutical forms
    Granules for oral suspension
    Routes of administration
    Oral use
    Dosage and administration details
    A single oral 0.2 mg/kg dose of ticagrelor. Before administration, ticagrelor granules were constituted with 10 mL of purified water to form a homogenous suspension of 1 mg/mL ticagrelor.

    Number of subjects in period 1
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <12 months old Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Started
    2
    6
    13
    Received ticagrelor
    2
    6
    13
    Completed
    2
    6
    13

    Baseline characteristics

    Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Ticagrelor 0.1 mg/kg: <6 months old
    Reporting group description
    Patients in the age group <6 months old received a single oral dose of 0.1 milligrams per kg (mg/kg) ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Reporting group description
    Patients in the age group 6 to <12 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Reporting group description
    Patients in the age group 12 to <24 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Reporting group values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <12 months old Ticagrelor 0.2 mg/kg: 12 to <24 months old Total
    Number of subjects
    2 6 13 21
    Age categorical
    Units: Subjects
        In utero
    0 0 0 0
        Preterm newborn infants (gestational age < 37 wks)
    0 0 0 0
        Newborns (0-27 days)
    0 0 0 0
        Infants and toddlers (28 days-23 months)
    2 6 13 21
        Children (2-11 years)
    0 0 0 0
        Adolescents (12-17 years)
    0 0 0 0
        Adults (18-64 years)
    0 0 0 0
        From 65-84 years
    0 0 0 0
        85 years and over
    0 0 0 0
    Age Continuous
    Units: Months
        arithmetic mean (standard deviation)
    3.5 ( 0.71 ) 9.0 ( 1.26 ) 16.2 ( 3.00 ) -
    Sex: Female, Male
    Units: Subjects
        Female
    1 4 5 10
        Male
    1 2 8 11
    Race (NIH/OMB)
    Units: Subjects
        American Indian or Alaska Native
    0 0 0 0
        Asian
    0 0 0 0
        Native Hawaiian or Other Pacific Islander
    0 0 0 0
        Black or African American
    0 6 8 14
        White
    2 0 5 7
        More than one race
    0 0 0 0
        Unknown or Not Reported
    0 0 0 0
    Ethnicity (NIH/OMB)
    Units: Subjects
        Hispanic or Latino
    0 0 0 0
        Not Hispanic or Latino
    2 6 13 21
        Unknown or Not Reported
    0 0 0 0

    End points

    Close Top of page
    End points reporting groups
    Reporting group title
    Ticagrelor 0.1 mg/kg: <6 months old
    Reporting group description
    Patients in the age group <6 months old received a single oral dose of 0.1 milligrams per kg (mg/kg) ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Reporting group description
    Patients in the age group 6 to <12 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Reporting group description
    Patients in the age group 12 to <24 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Subject analysis set title
    Ticagrelor 0.2 mg/kg: 6 to <24 months old
    Subject analysis set type
    Sub-group analysis
    Subject analysis set description
    Patients in the age group 6 to <24 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Subject analysis set title
    All Patients
    Subject analysis set type
    Full analysis
    Subject analysis set description
    Patients received single oral doses of 0.1 mg/kg ticagrelor (<6 months old) or 0.2 mg/kg ticagrelor (6 to <24 months old).

    Primary: Ticagrelor Mean Observed Plasma Concentrations

    Close Top of page
    End point title
    Ticagrelor Mean Observed Plasma Concentrations [1] [2]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean observed plasma concentration of ticagrelor is presented for each timepoint of sampling after patients had received a single oral dose of ticagrelor. Data is presented for each of the 2 doses of ticagrelor as well as for all patients. The lower limit of quantification (LLOQ) of ticagrelor was 1.00 nanograms per millilitre (ng/mL). Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient. '(n=x,y,z)' indicates the number of patients analysed at each timepoint.
    End point type
    Primary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    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: No formal statistical comparisons were carried out for this study. All endpoints were evaluated using standard summary descriptive statistics.
    [2] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms 'Ticagrelor 0.2 mg/kg: 6 to <12 months old' and 'Ticagrelor 0.2 mg/kg: 12 to <24 months old' represented within the analysis set 'Ticagrelor 0.2 mg/kg: 6 to <24 months old'.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2 [3]
    19
    21
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        1 hour post-dose (n=1,19,20)
    99999999 ( 99999999 )
    18.51 ( 154.70 )
    18.59 ( 147.75 )
        2 hours post-dose (n=2,19,21)
    99999999 ( 99999999 )
    21.82 ( 127.79 )
    21.86 ( 118.47 )
        4 hours post-dose (n=2,19,21)
    99999999 ( 99999999 )
    16.54 ( 61.78 )
    16.12 ( 59.75 )
        6 hours post-dose (n=2,18,20)
    99999999 ( 99999999 )
    10.39 ( 60.68 )
    10.25 ( 58.23 )
    Notes
    [3] - '9999999' indicates the data was not calculated due to the sample size.
    No statistical analyses for this end point

    Primary: Ticagrelor Maximum Observed Plasma Concentrations (Cmax)

    Close Top of page
    End point title
    Ticagrelor Maximum Observed Plasma Concentrations (Cmax) [4] [5]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean Cmax for ticagrelor is presented for each of the 2 doses of ticagrelor and for all patients. Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient.
    End point type
    Primary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    Notes
    [4] - 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: No formal statistical comparisons were carried out for this study. All endpoints were evaluated using standard summary descriptive statistics.
    [5] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms ‘Ticagrelor 0.2 mg/kg: 6 to <12 months old’ and ‘Ticagrelor 0.2 mg/kg: 12 to <24 months old’ represented within the analysis set ‘Ticagrelor 0.2 mg/kg: 6 to <24 months old’.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2
    19
    21
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    23.80 ( 23.78 )
    34.44 ( 74.75 )
    33.25 ( 71.67 )
    No statistical analyses for this end point

    Primary: Ticagrelor Area Under the Plasma Concentration-Time Curve from Zero to 6 Hours (AUC[0-6])

    Close Top of page
    End point title
    Ticagrelor Area Under the Plasma Concentration-Time Curve from Zero to 6 Hours (AUC[0-6]) [6] [7]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean AUC(0-6) for ticagrelor is presented for each of the 2 doses of ticagrelor and for all patients. Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient.
    End point type
    Primary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    Notes
    [6] - 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: No formal statistical comparisons were carried out for this study. All endpoints were evaluated using standard summary descriptive statistics.
    [7] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms ‘Ticagrelor 0.2 mg/kg: 6 to <12 months old’ and ‘Ticagrelor 0.2 mg/kg: 12 to <24 months old’ represented within the analysis set ‘Ticagrelor 0.2 mg/kg: 6 to <24 months old’.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2
    19
    21
    Units: ng*hours/mL (ng*h/mL)
        geometric mean (geometric coefficient of variation)
    87.22 ( 32.72 )
    112.39 ( 52.21 )
    109.71 ( 50.59 )
    No statistical analyses for this end point

    Secondary: Active Metabolite (AR-C124910XX) Mean Observed Plasma Concentrations

    Close Top of page
    End point title
    Active Metabolite (AR-C124910XX) Mean Observed Plasma Concentrations [8]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean observed plasma concentration of AR-C124910XX is presented for each timepoint of sampling after patients had received a single oral dose of ticagrelor. Data is presented for each of the 2 doses of ticagrelor as well as for all patients. The LLOQ of AR-C124910XX was 2.50 ng/mL. Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient. '(n=x,y,z)' indicates the number of patients analysed at each timepoint.
    End point type
    Secondary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    Notes
    [8] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms ‘Ticagrelor 0.2 mg/kg: 6 to <12 months old’ and ‘Ticagrelor 0.2 mg/kg: 12 to <24 months old’ represented within the analysis set ‘Ticagrelor 0.2 mg/kg: 6 to <24 months old’.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2 [9]
    19
    21
    Units: ng/mL
    geometric mean (geometric coefficient of variation)
        1 hour post-dose (n=1,19, 20)
    99999999 ( 99999999 )
    4.65 ( 88.79 )
    4.70 ( 85.91 )
        2 hours post-dose (n=2,19,21)
    99999999 ( 99999999 )
    7.40 ( 81.31 )
    7.35 ( 76.39 )
        4 hours post-dose (n=2,19,21)
    99999999 ( 99999999 )
    7.33 ( 53.57 )
    7.16 ( 52.08 )
        6 hours post-dose (n=2,18,20)
    99999999 ( 99999999 )
    5.89 ( 49.02 )
    5.77 ( 47.77 )
    Notes
    [9] - '99999999' indicates the data was not calculated due to the sample size.
    No statistical analyses for this end point

    Secondary: Active Metabolite (AR-C124910XX) Cmax

    Close Top of page
    End point title
    Active Metabolite (AR-C124910XX) Cmax [10]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean Cmax for AR-C124910XX is presented for each of the 2 doses of ticagrelor and for all patients. Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient.
    End point type
    Secondary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    Notes
    [10] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms ‘Ticagrelor 0.2 mg/kg: 6 to <12 months old’ and ‘Ticagrelor 0.2 mg/kg: 12 to <24 months old’ represented within the analysis set ‘Ticagrelor 0.2 mg/kg: 6 to <24 months old’.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2
    19
    21
    Units: ng/mL
        geometric mean (geometric coefficient of variation)
    6.89 ( 22.55 )
    9.17 ( 61.25 )
    8.93 ( 58.76 )
    No statistical analyses for this end point

    Secondary: Active Metabolite (AR-C124910XX) AUC(0-6)

    Close Top of page
    End point title
    Active Metabolite (AR-C124910XX) AUC(0-6) [11]
    End point description
    Blood samples were taken up to 6 hours post-dose so that the number and volume of blood samples were compliant with European Ethical Considerations in a Paediatric Population 2008. The geometric mean AUC(0-6) for AR-C124910XX is presented for each of the 2 doses of ticagrelor and for all patients. Data is presented for the PK analysis set which included all patients who received at least 1 dose of ticagrelor and provided at least 1 post-dose analysable plasma sample without any important protocol deviations or events that would exclude the patient.
    End point type
    Secondary
    End point timeframe
    Blood samples were collected on Day 1 at 1, 2, 4 and 6 hours post-dose.
    Notes
    [11] - The end point is not reporting statistics for all the arms in the baseline period. It is expected all the baseline period arms will be reported on when providing values for an end point on the baseline period.
    Justification: Data is presented for all patients in the baseline period, with data for patients in the arms ‘Ticagrelor 0.2 mg/kg: 6 to <12 months old’ and ‘Ticagrelor 0.2 mg/kg: 12 to <24 months old’ represented within the analysis set ‘Ticagrelor 0.2 mg/kg: 6 to <24 months old’.
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <24 months old All Patients
    Number of subjects analysed
    2
    19
    21
    Units: ng*h/mL
        geometric mean (geometric coefficient of variation)
    29.76 ( 22.84 )
    34.97 ( 58.67 )
    34.44 ( 55.80 )
    No statistical analyses for this end point

    Secondary: Observer Assessment of Acceptability and Palatability of Ticagrelor

    Close Top of page
    End point title
    Observer Assessment of Acceptability and Palatability of Ticagrelor
    End point description
    An observer's assessment of the patient's behaviour was performed directly as the patient was administered the single dose of ticagrelor on Day 1. The patient's willingness to swallow was recorded as one of the following: swallowed without a problem, some resistance but did swallow, spat out some/all of medication, vomited up the medication. The patient's negative response to palatability was assessed and the following outcomes were recorded: turned head to reject intake of the medication, twisted face or mouth in an expression of displeasure or other negative response. Data is presented for the safety analysis set which included all patients who received at least 1 single dose of ticagrelor, and for whom any post-dose data was available.
    End point type
    Secondary
    End point timeframe
    Day 1 (directly after treatment administration)
    End point values
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 6 to <12 months old Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Number of subjects analysed
    2
    6
    13
    Units: Participants
        Swallowed without problem
    2
    5
    10
        Some resistance but did swallow
    0
    0
    3
        Spat out some/all
    0
    1
    0
        Vomited up medication
    0
    0
    0
        Turned head to reject intake
    0
    1
    3
        Twisted face/mouth in displeasure
    0
    1
    0
        Other negative response
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

    Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    On-treatment adverse events (AEs) were collected from Day 1 up to Day 8 (approximately 1 week).
    Adverse event reporting additional description
    AEs are presented for the safety analysis set which included all patients who received at least 1 single dose of ticagrelor, and for whom any post-dose data was available.
    Assessment type
    Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    Ticagrelor 0.1 mg/kg: <6 months old
    Reporting group description
    Patients in the age group <6 months old received a single oral dose of 0.1 mg/kg ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 12 to <24 months old
    Reporting group description
    Patients in the age group 12 to <24 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Reporting group title
    Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Reporting group description
    Patients in the age group 6 to <12 months old received a single oral dose of 0.2 mg/kg ticagrelor.

    Serious adverse events
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 12 to <24 months old Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Infections and infestations
    Bronchiolitis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    Ticagrelor 0.1 mg/kg: <6 months old Ticagrelor 0.2 mg/kg: 12 to <24 months old Ticagrelor 0.2 mg/kg: 6 to <12 months old
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    0 / 2 (0.00%)
    3 / 13 (23.08%)
    3 / 6 (50.00%)
    Injury, poisoning and procedural complications
    Fall
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Injury
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Vascular disorders
    Pallor
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    1 / 6 (16.67%)
         occurrences all number
    0
    1
    1
    Blood and lymphatic system disorders
    Sickle cell anaemia with crisis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Splenomegaly
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Respiratory, thoracic and mediastinal disorders
    Nasal congestion
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Infections and infestations
    Gastroenteritis
         subjects affected / exposed
    0 / 2 (0.00%)
    1 / 13 (7.69%)
    0 / 6 (0.00%)
         occurrences all number
    0
    1
    0
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 2 (0.00%)
    0 / 13 (0.00%)
    2 / 6 (33.33%)
         occurrences all number
    0
    0
    2

    More information

    Close Top of page

    Substantial protocol amendments (globally)

    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    05 Jan 2018
    - The synopsis (Investigational product, dosage and mode of administration) and sections of the protocol relating to the rationale for study design, doses, and control groups, and interim analysis were changed to declare that any dose adjustments would require a protocol amendment and regulatory approval. The prediction level of reduction in platelet reactivity unit text was amended. These updates reflected the Medicines and Healthcare products Regulatory Agency request.

    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.
    For the endpoint 'Ticagrelor Mean Plasma Concentrations', '9999999' indicates the data was not calculable for the sample size.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA