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    Clinical Trial Results:
    A sequential, open-label, two-period study to assess the pharmacokinetics, safety and tolerability of two dose levels of AFQ056 in male, adolescent patients with Fragile X Syndrome (12 to 18 years inclusive)

    Summary
    EudraCT number
    		 2010-019353-18
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    29 Dec 2010

    Results information
    Results version number
    		 v1(current)
    This version publication date
    13 Jul 2016
    First version publication date
    26 Jul 2015
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    CAFQ056B2131
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Novartis Pharma AG
    Sponsor organisation address
    CH-4002, Basel, Switzerland,
    Public contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    Scientific contact
    Clinical Disclosure Office, Novartis Pharma AG, +41 613241111,
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001003-PIP01-10
    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
    29 Dec 2010
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Dec 2010
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    The main objective of the study was to evaluate the single-dose pharmacokinetics of mavoglurant (25 mg, 50 mg or 100 mg) capsules in male adolescent subjects (aged 12-18 years) suffering with Fragile X Syndrome (FXS).
    Protection of trial subjects
    The study was in compliance with the ethical principles derived from the Declaration of Helsinki and in compliance with all International Conference on Harmonization (ICH) Good Clinical Practice (GCP) Guidelines. All the local regulatory requirements pertinent to safety of trial subjects were followed. No rescue medications were permitted during the study as there are no approved treatments for FXS currently. If the investigator deemed that rescue medication for specific symptoms was necessary, the subject had to be discontinued from the study.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    06 Sep 2010
    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
    Switzerland: 12
    Worldwide total number of subjects
    12
    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)
    12
    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 conducted at single centre in Switzerland.

    Pre-assignment
    Screening details
    		 A total of 12 subjects were enrolled in the study.

    Period 1
    Period 1 title
    First Intervention Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    As the current study was an open label study, this section was not applicable.

    Arms
    Arm title
    		 AFQ056 25 mg
    Arm description
    		 Subjects received one capsule of AFQ056 25 mg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mavoglurant
    Investigational medicinal product code
    AFQ056
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    One capsule of AFQ056 25 mg was administered.

    Number of subjects in period 1
    		AFQ056 25 mg
    Started
    12
    Completed
    11
    Not completed
    1
         Consent withdrawn by subject
    1
    Period 2
    Period 2 title
    Second Intervention Period
    Is this the baseline period?
    		 No
    Allocation method
    Not applicable
    Blinding used
    		 Not blinded
    Blinding implementation details
    As the current study was an open label study, this section was not applicable.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 AFQ056 50 mg
    Arm description
    		 Subjects received two capsules of AFQ056 25 mg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mavoglurant
    Investigational medicinal product code
    AFQ056
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Two capsules of AFQ056 25 mg were administered.

    Arm title
    		 AFQ056 100 mg
    Arm description
    		 Subjects received one capsule of AFQ056 100 mg.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Mavoglurant
    Investigational medicinal product code
    AFQ056
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    One capsule of AFQ056 100 mg was administered.

    Number of subjects in period 2
    		AFQ056 50 mg AFQ056 100 mg
    Started
    4
    7
    Completed
    4
    7

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    AFQ056 25 mg
    Reporting group description
    		 Subjects received one capsule of AFQ056 25 mg.

    Reporting group values
    		 AFQ056 25 mg Total
    Number of subjects
    		 12 12
    Age categorical
    Units: Subjects
        Adolescents (12- 17 years)
    		 12 12
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 13.8 ± 1.71 -
    Gender categorical
    Units: Subjects
        Female
    		 0 0
        Male
    		 12 12

    End points

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    End points reporting groups
    Reporting group title
    AFQ056 25 mg
    Reporting group description
    		 Subjects received one capsule of AFQ056 25 mg.
    Reporting group title
    AFQ056 50 mg
    Reporting group description
    		 Subjects received two capsules of AFQ056 25 mg.

    Reporting group title
    AFQ056 100 mg
    Reporting group description
    		 Subjects received one capsule of AFQ056 100 mg.

    Primary: AUC From Time Zero to Extrapolated Infinite Time (AUCinf)

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    End point title
    		 AUC From Time Zero to Extrapolated Infinite Time (AUCinf) [1]
    End point description
    AUCinf was the area under the plasma concentration versus time curve (AUC) from time zero (pre-dose) to infinity. Analysis was performed in pharmacokinetic (PK) set population which included all the subjects who received at least one dose of study drug and had data for at least one of the primary PK variables in at least one period and no major protocol deviations with impact on PK data.
    End point type
    Primary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 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: Only descriptive summary statistics was planned for this primary outcome measure.
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    11 [2]
    4
    7
    Units: hour.nanograms/millilitre(hr.ng/mL)
        arithmetic mean (standard deviation)
    364.8 ± 169.52
    871.2 ± 351.8
    1131 ± 560.28
    Notes
    [2] - Result for one subject was excluded, since terminal phase was not evaluable.
    No statistical analyses for this end point

    Primary: AUC From Time Zero to Last Measurable Concentration [AUClast]

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    End point title
    		 AUC From Time Zero to Last Measurable Concentration [AUClast] [3]
    End point description
    Area under the plasma concentration-time curve from time zero to the time of the last quantifiable concentration. It was calculated as the sum of linear trapezoids using non-compartmental analysis. Analysis was performed in PK set population.
    End point type
    Primary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 hours 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: Only descriptive summary statistics was planned for this primary outcome measure.
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    12
    4
    7
    Units: hr.ng/mL
        arithmetic mean (standard deviation)
    357.9 ± 176.25
    790.2 ± 287.36
    1022 ± 494.89
    No statistical analyses for this end point

    Primary: Maximum Observed Plasma Concentration (Cmax)

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    End point title
    		 Maximum Observed Plasma Concentration (Cmax) [4]
    End point description
    Maximum observed plasma concentration following drug administration from the raw plasma concentration-time data. Analysis was performed in PK set population.
    End point type
    Primary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 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: Only descriptive summary statistics was planned for this primary outcome measure.
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    12
    4
    7
    Units: ng/mL
        arithmetic mean (standard deviation)
    68.01 ± 20.801
    143 ± 39.741
    185.8 ± 110.5
    No statistical analyses for this end point

    Secondary: Time to Reach Maximum Observed Plasma Concentration (Tmax)

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    End point title
    		 Time to Reach Maximum Observed Plasma Concentration (Tmax)
    End point description
    Tmax was defined as the time required to reach maximum observed plasma concentration. Tmax was directly determined from the raw plasma concentration-time data. Analysis was performed in PK set population.
    End point type
    Secondary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 hours post-dose
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    12
    4
    7
    Units: Hours (hr)
        median (full range (min-max))
    1 (1 to 2.75)
    1 (1 to 1)
    1 (0.92 to 4)
    No statistical analyses for this end point

    Secondary: Apparent Plasma Clearance (CL/F)

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    End point title
    		 Apparent Plasma Clearance (CL/F)
    End point description
    The apparent body clearance of drug from the plasma (CL/F) and it was calculated as Dose/AUCinf, where CL was the clearance of the drug and F was the absolute oral bioavailability. Analysis was performed in PK set population.
    End point type
    Secondary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 hours post-dose
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    11 [5]
    4
    7
    Units: Litre/hour(L/hr)
        arithmetic mean (standard deviation)
    94.1 ± 73
    65.3 ± 26.9
    111 ± 57.4
    Notes
    [5] - Result for one subject was excluded, since terminal phase was not evaluable.
    No statistical analyses for this end point

    Secondary: Elimination half-life (T1/2)

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    End point title
    		 Elimination half-life (T1/2)
    End point description
    The elimination half-life associated with the terminal slope of a semi logarithmic concentration-time curve. Analysis was performed in PK set population.
    End point type
    Secondary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 hours post-dose
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    11 [6]
    4
    7
    Units: Hour (hr)
        arithmetic mean (standard deviation)
    6.96 ± 4.2
    6.6 ± 1.93
    7.21 ± 1.93
    Notes
    [6] - Result for one subject was excluded, since terminal phase was not evaluable.
    No statistical analyses for this end point

    Secondary: Apparent Volume of Distribution (Vz/F)

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    End point title
    		 Apparent Volume of Distribution (Vz/F)
    End point description
    Apparent volume of distribution was calculated by using the formula (CL/F)/λz, where CL/F was oral total plasma clearance and λz was terminal elimination rate constant. Analysis was performed in PK set population.
    End point type
    Secondary
    End point timeframe
    Pre-dose (0), 1, 2.5, 4, 8, 12, 16 and 24 hours post-dose
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    11 [7]
    4
    7
    Units: Litre (L)
        arithmetic mean (standard deviation)
    713 ± 324
    599 ± 278
    1163 ± 657
    Notes
    [7] - Result for one subject was excluded, since terminal phase was not evaluable.
    No statistical analyses for this end point

    Secondary: Number of subjects with adverse events (AEs) and serious adverse events(SAEs)

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    End point title
    		 Number of subjects with adverse events (AEs) and serious adverse events(SAEs)
    End point description
    An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug. A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the subject or require medical or surgical intervention to prevent one of the aforementioned outcomes. All subjects who received at least one dose of study drug were included in the safety analysis set.
    End point type
    Secondary
    End point timeframe
    From start of study treatment to end of study
    End point values
    		 AFQ056 25 mg AFQ056 50 mg AFQ056 100 mg
    Number of subjects analysed
    12
    4
    7
    Units: Number of subjects
        AEs
    3
    0
    0
        SAEs
    0
    0
    0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    13.1
    Reporting groups
    Reporting group title
    25 mg AFQ056
    Reporting group description
    		 Subjects received one capsule of AFQ056 25 mg.

    Serious adverse events
    		 25 mg AFQ056
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 12 (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
    		 25 mg AFQ056
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    3 / 12 (25.00%)
    Investigations
    Blood amylase increased
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Lipase increased
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    1 / 12 (8.33%)
         occurrences all number
    1
    Infections and infestations
    Nail infection
         subjects affected / exposed
    1 / 12 (8.33%)
         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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