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    Clinical Trial Results:
    A MULTI-CENTRE, SINGLE DOSE TRIAL TO EVALUATE PHARMACOKINETICS OF PITOLISANT (BF2.649) IN CHILDREN FROM 6 TO LESS THAN 18 YEARS WITH NARCOLEPSY

    Summary
    EudraCT number
    		 2013-001505-93
    Trial protocol
    		 IT   FR  
    Global end of trial date
    16 Sep 2014

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 Jun 2022
    First version publication date
    26 Jun 2022
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    P11-11
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Bioprojet Pharma
    Sponsor organisation address
    9, rue Rameau, 75002 - Paris, France,
    Public contact
    Clinical Development Director, Bioprojet Pharma, 33 147036633, contact@bioprojet.com
    Scientific contact
    Clinical Development Director, Bioprojet Pharma, 33 147036633, contact@bioprojet.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001176-PIP01-11
    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
    09 Jul 2015
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    16 Sep 2014
    Global end of trial reached?
    Yes
    Global end of trial date
    16 Sep 2014
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To determine the pharmacokinetic parameters of pitolisant in narcoleptic children aged from 6 to less than 18 years including at least maximum concentration (Cmax), time of occurrence of maximum concentration (tmax), t1/2, AUC0-t and AUC0-∞ using 6 samples per participant.
    Protection of trial subjects
    Physical examination, vital sign, ECG evaluation and haematology/blood chemistry were performed on trial subjects to ensure safety. Adverse events were continuously monitored. Topical anaesthesia was offered for phlebotomy/ venipuncture. Blood samples were taken using a catheter.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    16 Jan 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    No
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    France: 15
    Country: Number of subjects enrolled
    Italy: 10
    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)
    0
    Children (2-11 years)
    12
    Adolescents (12-17 years)
    13
    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
    		 The study was carried out in 25 narcoleptic children with or without cataplexy (12 males and 13 females), who met all the inclusion criteria and none of the non-inclusion criteria. One patient withdrew from the study. Therefore, 24 patients duly completed the study.

    Pre-assignment
    Screening details
    		 Occurred within 2 weeks before starting study medication. Each patient underwent a medical history and physical examination. Oral and written information about the study was given followed by a questionnaire corresponding to the study inclusion/non-inclusion criteria. Parents or the patient (if old enough), signed the Informed Consent Form.

    Pre-assignment period milestones
    Number of subjects started
    		 25
    Number of subjects completed
    		 25

    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
    Yes

    Arm title
    		 Sub-group I
    Arm description
    		 from 6 to less than 12 years of age
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pitolisant
    Investigational medicinal product code
    PF2.649
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Single dose of 20 mg

    Arm title
    		 Sub-group II
    Arm description
    		 from 12 to less than 18 years of age
    Arm type
    		 Experimental

    Investigational medicinal product name
    Pitolisant
    Investigational medicinal product code
    PF2.649
    Other name
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Single dose of 20 mg

    Number of subjects in period 1
    		Sub-group I Sub-group II
    Started
    12
    13
    Completed
    12
    12
    Not completed
    0
    1
         Protocol deviation
    -
    1

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sub-group I
    Reporting group description
    		 from 6 to less than 12 years of age

    Reporting group title
    Sub-group II
    Reporting group description
    		 from 12 to less than 18 years of age

    Reporting group values
    		 Sub-group I Sub-group II Total
    Number of subjects
    		 12 13 25
    Age categorical
    Units: Subjects
        from 6 to less than 12 years of age
    		 12 0 12
        from 12 to less than 18 years of age
    		 0 13 13
    Gender categorical
    Units: Subjects
        Female
    		 7 6 13
        Male
    		 5 7 12

    End points

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    End points reporting groups
    Reporting group title
    Sub-group I
    Reporting group description
    		 from 6 to less than 12 years of age

    Reporting group title
    Sub-group II
    Reporting group description
    		 from 12 to less than 18 years of age

    Primary: tmax

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    End point title
    		 tmax [1]
    End point description
    Determination of the PK parameters of pitolisant in narcoleptic children from 6 to less than 18 years of age
    End point type
    Primary
    End point timeframe
    PK sampling at Pre-dose, 1, 2, 3, 6, and 10 h 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: Categorical data were presented using frequency and percentages of patients, while continuous variables were presented using the arithmetic mean, standard deviation (SD), median, minimum (Min), maximum (Max), and number observations.
    End point values
    		 Sub-group I Sub-group II
    Number of subjects analysed
    12
    12
    Units: hours
        arithmetic mean (full range (min-max))
    2.50 (1.00 to 6.00)
    2.00 (1.00 to 3.00)
    No statistical analyses for this end point

    Primary: Cmax

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    End point title
    		 Cmax [2]
    End point description
    Determination of the PK parameters of pitolisant in narcoleptic children from 6 to less than 18 years of age
    End point type
    Primary
    End point timeframe
    PK sampling at Pre-dose, 1, 2, 3, 6, and 10 h post-dose
    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: Categorical data were presented using frequency and percentages of patients, while continuous variables were presented using the arithmetic mean, standard deviation (SD), median, minimum (Min), maximum (Max), and number observations.
    End point values
    		 Sub-group I Sub-group II
    Number of subjects analysed
    12
    12
    Units: ng/mL
        arithmetic mean (standard deviation)
    55.50 ( 26.47 )
    36.47 ( 19.61 )
    No statistical analyses for this end point

    Primary: AUC0-t

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    End point title
    		 AUC0-t [3]
    End point description
    Determination of the PK parameters of pitolisant in narcoleptic children from 6 to less than 18 years of age
    End point type
    Primary
    End point timeframe
    PK sampling at Pre-dose, 1, 2, 3, 6, and 10 h post-dose
    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: Categorical data were presented using frequency and percentages of patients, while continuous variables were presented using the arithmetic mean, standard deviation (SD), median, minimum (Min), maximum (Max), and number observations.
    End point values
    		 Sub-group I Sub-group II
    Number of subjects analysed
    12
    12
    Units: ng.h/mL
        arithmetic mean (standard deviation)
    316.06 ( 151.82 )
    182.19 ( 92.48 )
    No statistical analyses for this end point

    Primary: AUC0-∞

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    End point title
    		 AUC0-∞ [4]
    End point description
    Determination of the PK parameters of pitolisant in narcoleptic children from 6 to less than 18 years of age
    End point type
    Primary
    End point timeframe
    PK sampling at Pre-dose, 1, 2, 3, 6, and 10 h 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: Categorical data were presented using frequency and percentages of patients, while continuous variables were presented using the arithmetic mean, standard deviation (SD), median, minimum (Min), maximum (Max), and number observations.
    End point values
    		 Sub-group I Sub-group II
    Number of subjects analysed
    2
    2
    Units: ng.h/mL
        arithmetic mean (standard deviation)
    276.50 ( 23.33 )
    155.50 ( 28.99 )
    No statistical analyses for this end point

    Primary: t1/2

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    End point title
    		 t1/2 [5]
    End point description
    Determination of the PK parameters of pitolisant in narcoleptic children from 6 to less than 18 years of age
    End point type
    Primary
    End point timeframe
    PK sampling at Pre-dose, 1, 2, 3, 6, and 10 h post-dose
    Notes
    [5] - 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: Categorical data were presented using frequency and percentages of patients, while continuous variables were presented using the arithmetic mean, standard deviation (SD), median, minimum (Min), maximum (Max), and number observations.
    End point values
    		 Sub-group I Sub-group II
    Number of subjects analysed
    2
    2
    Units: hours
        arithmetic mean (standard deviation)
    3.55 ( 0.21 )
    3.75 ( 0.07 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Continuous reporting
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    16.1
    Reporting groups
    Reporting group title
    Sub-group I
    Reporting group description
    		 from 6 to less than 12 years of age

    Reporting group title
    Sub-group II
    Reporting group description
    		 from 12 to less than 18 years of age

    Serious adverse events
    		 Sub-group I Sub-group II
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (0.00%)
    0 / 13 (0.00%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Frequency threshold for reporting non-serious adverse events: 0%
    Non-serious adverse events
    		 Sub-group I Sub-group II
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    2 / 12 (16.67%)
    2 / 13 (15.38%)
    Nervous system disorders
    Dizziness
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Headache
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 12 (8.33%)
    2 / 13 (15.38%)
         occurrences all number
    1
    2
    Gastrointestinal disorders
    Diarrhoea
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 13 (0.00%)
         occurrences all number
    1
    0
    Vomiting
    alternative assessment type: Non-systematic
         subjects affected / exposed
    1 / 12 (8.33%)
    0 / 13 (0.00%)
         occurrences all number
    0
    0

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Nov 2013
    This amendment presented adaptation of inclusion criteria No. 4 (enlargement of the BMI range in favor of low values with lower limit brought from 22 to 18 kg/m² and for Italian site clarification of laboratory tests results) and No. 8 (change of necessary hormonal contraception at screening to a birth control deemed appropriate by the Investigator) as well as minor changes to maintain consistency across sections of the protocol.

    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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