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    Clinical Trial Results:
    Study of the pharmacokinetics and pharmacodynamics of desmopressin oral lyophilisate - route of administration in the pediatric patient population - SAFEPEDRUG

    Summary
    EudraCT number
    		 2014-005200-13
    Trial protocol
    		 BE  
    Global end of trial date
    31 Dec 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    19 Aug 2021
    First version publication date
    19 Aug 2021
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    SafePed002
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02584231
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Ghent University Hospital
    Sponsor organisation address
    C. Heymanslaan 10, Ghent, Belgium, 9000
    Public contact
    HIRUZ CTU, Ghent University Hospital, +32 93320500, hiruz.ctu@uzgent.be
    Scientific contact
    HIRUZ CTU, Ghent University Hospital, +32 93320500, hiruz.ctu@uzgent.be
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    01 Feb 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    19 Mar 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    31 Dec 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the influence of the weight, length and gender on the pharmacokinetcs of desmopressin
    Protection of trial subjects
    Ethics review and approval, informed consent, supportive care and routine monitoring.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    01 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
    Belgium: 25
    Worldwide total number of subjects
    25
    EEA total number of subjects
    25
    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)
    3
    Children (2-11 years)
    22
    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
    		 25 patients were recruited between 09-09-2015 and 19-03-2018. End of trial notification was dated 31-12-2018 (last patient last visit) and submitted to EC and CA on 09-01-2019. There were 2 dropouts due to technical problems with blood sampling.

    Pre-assignment
    Screening details
    		 The following groups were included in the study: between 6 months and 2 years (n=3): dosing with 60µg of PO; between 2 and 4 years (n=5): dosing with 120µg PO and between 4 and 8 years (n=17): dosage with 240 µg PO.

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

    Arms
    Are arms mutually exclusive
    No

    Arm title
    		 Desmopressine arm
    Arm description
    		 Treatment of enuresis nocturna in paediatric patients, with desmopressine lyophilisaat (MELT) 60 or 120µg for oral use.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Desmopressine
    Investigational medicinal product code
    CAS 16679-58-6
    Other name
    Minirin Melt
    Pharmaceutical forms
    Oral lyophilisate, Tablet
    Routes of administration
    Sublingual use
    Dosage and administration details
    Doses are administered according to the patient's age: between 6 months and 2 years: dosing with 60µg of PO; between 2 and 4 years: dosing with 120µg PO and between 4 and 8 years: dosage with 240 µg PO.

    Arm title
    		 Baseline arm
    Arm description
    		 Baseline data for the study, as the study only has 1 arm
    Arm type
    		 Baseline arm

    Investigational medicinal product name
    No investigational medicinal product assigned in this arm
    Number of subjects in period 1
    		Desmopressine arm Baseline arm
    Started
    25
    25
    Completed
    23
    25
    Not completed
    2
    0
         technical problems with blood sampling.
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall trial
    Reporting group description
    		 -

    Reporting group values
    		 Overall trial Total
    Number of subjects
    		 25 25
    Age categorical
    Treatment of enuresis nocturna in paediatric patients (age 0,9 - 7,3 years).
    Units: Subjects
        Infants and toddlers (28 days-23 months)
    		 3 3
        Children (2-11 years)
    		 22 22
    Gender categorical
    All 25 patients, 9 girls and 16 boys, completed the renal concentrating test.
    Units: Subjects
        Female
    		 9 9
        Male
    		 16 16
    Weight
    Units: kg
        median (inter-quartile range (Q1-Q3))
    		 17.8 (13.0 to 25.5) -
    Length
    Units: cm
        median (inter-quartile range (Q1-Q3))
    		 112 (95.6 to 123) -
    BMI
    Units: kg/m²
        median (inter-quartile range (Q1-Q3))
    		 1.56 (1.51 to 1.66) -
    Dose/weight
    Units: µg/kg
        median (inter-quartile range (Q1-Q3))
    		 9.41 (8.57 to 11.8) -

    End points

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    End points reporting groups
    Reporting group title
    Desmopressine arm
    Reporting group description
    		 Treatment of enuresis nocturna in paediatric patients, with desmopressine lyophilisaat (MELT) 60 or 120µg for oral use.

    Reporting group title
    Baseline arm
    Reporting group description
    		 Baseline data for the study, as the study only has 1 arm

    Primary: Desmopressine concentrations

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    End point title
    		 Desmopressine concentrations [1] [2]
    End point description
    End point type
    Primary
    End point timeframe
    0, 1/4, 1/2, 1, 2, 3, 5, 6 and 7h
    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 statistical analysis available.
    [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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    20 [3]
    Units: pg*h/mL
    median (full range (min-max))
        AUC (0 - infinity)
    105 (66 to 146)
    Notes
    [3] - No PK data available for the first 5 of the 25 patients (technical problem storage conditions)
    No statistical analyses for this end point

    Secondary: Efficacy - osmolality in urine

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    End point title
    		 Efficacy - osmolality in urine [4]
    End point description
    PD of desmopressin in children with nocturnal enuresis that is resistant to treatment with desmopressin tablet. Second PD parameter is urinary concentration capacity. This is osmolality in urine.
    End point type
    Secondary
    End point timeframe
    24 hours
    Notes
    [4] - 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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    10
    Units: mOsm/kg
        arithmetic mean (standard deviation)
    859 ± 67
    No statistical analyses for this end point

    Secondary: Safety of desmopressin in children as assessed by registration of adverse events.

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    End point title
    		 Safety of desmopressin in children as assessed by registration of adverse events. [5]
    End point description
    Registration of adverse events
    End point type
    Secondary
    End point timeframe
    24 hours
    Notes
    [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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    10
    Units: adverse events
        occurence of adverse events
    4
        occurence of serious adverse events
    0
    No statistical analyses for this end point

    Secondary: Safety of desmopressin in children as assessed by the measurement of natremia

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    End point title
    		 Safety of desmopressin in children as assessed by the measurement of natremia [6]
    End point description
    End point type
    Secondary
    End point timeframe
    24 hours
    Notes
    [6] - 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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    10
    Units: sodium (plasma, mmol/L)
    arithmetic mean (standard deviation)
        difference in sodium (plasma)
    0.5 ± 2.2
    No statistical analyses for this end point

    Secondary: Efficacy - urinary volume.

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    End point title
    		 Efficacy - urinary volume. [7]
    End point description
    Antidiuretic effect was defined in this study if the diuresis rate dropped below 2mL/kg/h (age c 2 y) or 1 mL/kg/h (age > 2y)
    End point type
    Secondary
    End point timeframe
    24 hours
    Notes
    [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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    10
    Units: patients with antidiuretic effect (n)
        drop below 2mL/kg/h ( <2y) or 1 mL/kg/h ( >2y)
    10
    No statistical analyses for this end point

    Secondary: Urinary concentration test

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    End point title
    		 Urinary concentration test [8]
    End point description
    Evaluation of the oral lyophilisate formulation of desmopressin for the urinary concentration test
    End point type
    Secondary
    End point timeframe
    24 hours
    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: Statistics are not reported for the baseline arm (baseline arm for the study, as the study only has 1 arm).
    End point values
    		 Desmopressine arm
    Number of subjects analysed
    20 [9]
    Units: mL/min/kg
        arithmetic mean (standard deviation)
    0.76 ± 0.46
    Notes
    [9] - No PK data available for the first 5 of the 25 patients (technical problem storage conditions)
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events will be reported between the first dose administration of trial medication and the last trial related activity.
    Assessment type
    		 Non-systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24
    Reporting groups
    Reporting group title
    Overall Trial
    Reporting group description
    		 Treatment of enuresis nocturna in paediatric patients, with desmopressine lyophilisaat (MELT) 60 or 120µg for oral use.

    Serious adverse events
    		 Overall Trial
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 25 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Overall Trial
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    4 / 25 (16.00%)
    General disorders and administration site conditions
    sore throat
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences all number
    1
    Gastrointestinal disorders
    Diarrhoea
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences all number
    1
    abdominal pain/stomach pain
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences all number
    1
    Renal and urinary disorders
    pain during voiding
         subjects affected / exposed
    1 / 25 (4.00%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    21 Jan 2016
    Reason for substantial amendment: • Removal of Tanner stage determination from protocol. • Correction of typing error • Extra measurement of Desmopressin concentration in urine

    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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