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    Clinical Trial Results:
    An Open-label, Single Arm Study to Evaluate the Pharmacokinetics of a Single Dose of Intravenous Difelikefalin in Adolescents Aged 12 to 17 Years on Haemodialysis

    Summary
    EudraCT number
    		 2021-000894-94
    Trial protocol
    		 IT  
    Global end of trial date
    30 May 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    15 Dec 2023
    First version publication date
    15 Dec 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    KOR-PED-201
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vifor Fresenius Medical Care Renal Pharma Ltd.
    Sponsor organisation address
    Rechenstrasse 37, St. Gallen, Switzerland, CH-9014
    Public contact
    Clinical Trial Information Desk, Vifor Fresenius Medical Care Renal Pharma Ltd., +41 588518000, clinicaltrials@cslbehring.com
    Scientific contact
    Clinical Trial Information Desk, Vifor Fresenius Medical Care Renal Pharma Ltd., +41 588518000, clinicaltrials@cslbehring.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-002565-PIP02-19
    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
    03 Aug 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    30 May 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    30 May 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the pharmacokinetic (PK) profile of a single dose of difelikefalin in adolescent subjects aged 12 to 17 years on haemodialysis (HD).
    Protection of trial subjects
    The study was conducted according to the principles of the World Medical Association’s Declaration of Helsinki, and the ICH guidelines for Good Clinical Practices (GCP) as amended.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Nov 2021
    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
    Lebanon: 5
    Country: Number of subjects enrolled
    United Kingdom: 3
    Worldwide total number of subjects
    8
    EEA total number of subjects
    0
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    8
    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
    		 -

    Pre-assignment
    Screening details
    		 The screening visit occurred within 21 calendar days prior to the start of study drug administration.

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

    Arms
    Arm title
    		 Difelikefalin
    Arm description
    		 -
    Arm type
    		 Experimental

    Investigational medicinal product name
    Difelikefalin solution (IV formulation)
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Injection
    Routes of administration
    Intravenous use
    Dosage and administration details
    Individual IV doses of difelikefalin were based on subject body weight (0.5 μg/kg dry body weight) and prepared by withdrawing subject-specific volume of study drug with sterile, single-use 1 ml Plastipak syringe (or equivalent) and sterile single-use needles. Difelikefalin was administered by IV bolus injection within 15 minutes following the end of the dialysis on the scheduled drug administration day. Difelikefalin administration could be done by injection into the dialysis venous line (e.g., into the venous port) or by direct injection into a vein. If the dialysis line was used, following the bolus, the venous line was flushed with at least 10 ml of normal saline.

    Number of subjects in period 1
    		Difelikefalin
    Started
    8
    Completed
    8

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Difelikefalin
    Reporting group description
    		 -

    Reporting group values
    		 Difelikefalin Total
    Number of subjects
    		 8 8
    Age categorical
    Units: Subjects
        In utero
    		 0 0
        Preterm newborn infants (gestational age < 37 wks)
    		 0 0
        Newborns (0-27 days)
    		 0 0
        Infants and toddlers (28 days-23 months)
    		 0 0
        Children (2-11 years)
    		 0 0
        Adolescents (12-17 years)
    		 8 8
        Adults (18-64 years)
    		 0 0
        From 65-84 years
    		 0 0
        85 years and over
    		 0 0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 15.0 ( 2.14 ) -
    Gender categorical
    Units: Subjects
        Female
    		 5 5
        Male
    		 3 3
    Subject analysis sets

    Subject analysis set title
    Pharmacokinetic Analysis Population
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK evaluable population includes all subjects who received the dose of study drug and have sufficient plasma concentrations for PK analysis

    Subject analysis sets values
    		 Pharmacokinetic Analysis Population
    Number of subjects
    7
    Age categorical
    Units: Subjects
        In utero
    0
        Preterm newborn infants (gestational age < 37 wks)
    0
        Newborns (0-27 days)
    0
        Infants and toddlers (28 days-23 months)
    0
        Children (2-11 years)
    0
        Adolescents (12-17 years)
    7
        Adults (18-64 years)
    0
        From 65-84 years
    0
        85 years and over
    0
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    15.1 ( 2.27 )
    Gender categorical
    Units: Subjects
        Female
    4
        Male
    3

    End points

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    End points reporting groups
    Reporting group title
    Difelikefalin
    Reporting group description
    		 -

    Subject analysis set title
    Pharmacokinetic Analysis Population
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    The PK evaluable population includes all subjects who received the dose of study drug and have sufficient plasma concentrations for PK analysis

    Primary: Cmax

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    End point title
    		 Cmax [1]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 to 3
    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: Only descriptive statistics were included for this PK parameter.
    End point values
    		 Pharmacokinetic Analysis Population
    Number of subjects analysed
    7
    Units: ng/mL
        arithmetic mean (standard deviation)
    6.03 ( 1.81 )
    No statistical analyses for this end point

    Primary: AUC0-48

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    End point title
    		 AUC0-48 [2]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 to 3
    Notes
    [2] - 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: Only descriptive statistics were included for this PK parameter.
    End point values
    		 Pharmacokinetic Analysis Population
    Number of subjects analysed
    7
    Units: h*ng/mL
        arithmetic mean (standard deviation)
    63.4 ( 17.4 )
    No statistical analyses for this end point

    Primary: AUCinf

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    End point title
    		 AUCinf [3]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 to 3
    Notes
    [3] - 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: Only descriptive statistics were included for this PK parameter.
    End point values
    		 Pharmacokinetic Analysis Population
    Number of subjects analysed
    7
    Units: h*ng/mL
        arithmetic mean (standard deviation)
    72.5 ( 23.7 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    from Baseline to the End of the trial
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    Difelikefalin
    Reporting group description
    		 -

    Serious adverse events
    		 Difelikefalin
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 8 (0.00%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Difelikefalin
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    1 / 8 (12.50%)
    Nervous system disorders
    Dizziness
         subjects affected / exposed
    1 / 8 (12.50%)
         occurrences all number
    1
    Headache
         subjects affected / exposed
    1 / 8 (12.50%)
         occurrences all number
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? No

    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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