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    Clinical Trial Results:
    A Phase 1, Randomized, Open-label Study to Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Lumacaftor in Combination With Ivacaftor in Healthy Adult Subjects

    Summary
    EudraCT number
    		 2019-002254-23
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    01 Dec 2015

    Results information
    Results version number
    		 v1(current)
    This version publication date
    04 Oct 2019
    First version publication date
    04 Oct 2019
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX15-809-014
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, Massachusetts, United States,
    Public contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    Scientific contact
    Medical Monitor, Vertex Pharmaceuticals Incorporated, +1 617 341 6777, medicalinfo@vrtx.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001582-PIP01-13
    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
    26 Feb 2016
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    24 Nov 2015
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Dec 2015
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the relative bioavailability of the granule formulation compared to the tablet formulation of lumacaftor (LUM)/ivacaftor (IVA)
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Council on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    22 Sep 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
    United States: 16
    Worldwide total number of subjects
    16
    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)
    0
    Adults (18-64 years)
    16
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 A total of 16 subjects were enrolled and randomized in the study.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Sequence 1
    Arm description
    		 Subjects received LUM/IVA Dose 1 tablet formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 2 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/ VX-770
    Other name
    Lumacaftor/Ivacaftor fixed dose combination
    Pharmaceutical forms
    Tablet, Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received a single dose of LUM/IVA tablet or granule formulation in the fed or fasted state on Day 1 in treatment period 1, 2, 3 and 4 as per the sequence.

    Arm title
    		 Sequence 2
    Arm description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 2, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/ VX-770
    Other name
    Lumacaftor/Ivacaftor fixed dose combination
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received a single dose of LUM/IVA tablet or granule formulation in the fed or fasted state on Day 1 in treatment period 1, 2, 3 and 4 as per the sequence.

    Arm title
    		 Sequence 3
    Arm description
    		 Subjects received LUM/IVA Dose 2 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 3, and then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/ VX-770
    Other name
    Lumacaftor/Ivacaftor fixed dose combination
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received a single dose of LUM/IVA tablet or granule formulation in the fed or fasted state on Day 1 in treatment period 1, 2, 3 and 4 as per the sequence.

    Arm title
    		 Sequence 4
    Arm description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fasted state in treatment period 1, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/ VX-770
    Other name
    Lumacaftor/Ivacaftor fixed dose combination
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received a single dose of LUM/IVA tablet or granule formulation in the fed or fasted state on Day 1 in treatment period 1, 2, 3 and 4 as per the sequence.

    Number of subjects in period 1
    		Sequence 1 Sequence 2 Sequence 3 Sequence 4
    Started
    4
    4
    4
    4
    Completed
    4
    4
    4
    4

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Sequence 1
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 tablet formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 2 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 2
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 2, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 3
    Reporting group description
    		 Subjects received LUM/IVA Dose 2 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 3, and then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 4
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fasted state in treatment period 1, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group values
    		 Sequence 1 Sequence 2 Sequence 3 Sequence 4 Total
    Number of subjects
    		 4 4 4 4 16
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 30.8 ( 4.72 ) 36.0 ( 9.87 ) 37.0 ( 11.17 ) 34.0 ( 16.21 ) -
    Gender categorical
    Units: Subjects
        Female
    		 2 2 3 3 10
        Male
    		 2 2 1 1 6

    End points

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    End points reporting groups
    Reporting group title
    Sequence 1
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 tablet formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 2 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 2
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 2, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 3
    Reporting group description
    		 Subjects received LUM/IVA Dose 2 granule formulation in fed state in treatment period 1, then LUM/IVA Dose 1 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 granule formulation in fasted state in treatment period 3, and then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Reporting group title
    Sequence 4
    Reporting group description
    		 Subjects received LUM/IVA Dose 1 granule formulation in fasted state in treatment period 1, then LUM/IVA Dose 2 granule formulation in fed state in treatment period 2, then LUM/IVA Dose 1 tablet formulation in fed state in treatment period 3, and then LUM/IVA Dose 1 granule formulation in fed state in treatment period 4. A washout period of at least 10 days occurred between each dosing occasion.

    Subject analysis set title
    Treatment A: LUM/IVA Dose 1 Tablet (Fed)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All subjects who received LUM/IVA Dose 1 tablet formulation in fed state.

    Subject analysis set title
    Treatment B: LUM/IVA Dose 1 Granule (Fasted)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All Subjects who received LUM/IVA Dose 1 granule formulation in fasted state.

    Subject analysis set title
    Treatment B: LUM/IVA Dose 1 Granule (Fed)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All Subjects who received LUM/IVA Dose 1 granule formulation in fed state.

    Subject analysis set title
    Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Subject analysis set type
    		 Safety analysis
    Subject analysis set description
    All Subjects who received LUM/IVA Dose 2 granule formulation in fed state.

    Primary: Maximum Observed Plasma Concentration (Cmax) of LUM and IVA

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    End point title
    		 Maximum Observed Plasma Concentration (Cmax) of LUM and IVA [1]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 up to Day 6 for each treatment period
    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 planned. No statistical comparisons were planned for the primary pharmacokinetic (PK) endpoint. PK set included participants who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here “Number Analyzed” signifies those participants who were evaluable for this outcome measure in the specified treatment period.
    End point values
    		 Treatment A: LUM/IVA Dose 1 Tablet (Fed) Treatment B: LUM/IVA Dose 1 Granule (Fasted) Treatment B: LUM/IVA Dose 1 Granule (Fed) Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Number of subjects analysed
    16
    15
    16
    15
    Units: nanogram per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        LUM
    5590 ( 1150 )
    3060 ( 1020 )
    4460 ( 541 )
    7010 ( 1370 )
        IVA
    643 ( 201 )
    301 ( 142 )
    531 ( 111 )
    837 ( 178 )
    No statistical analyses for this end point

    Primary: Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity [AUC(0 - inf)] of LUM and IVA

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    End point title
    		 Area Under the Concentration Versus Time Curve From the Time of Dosing Extrapolated to Infinity [AUC(0 - inf)] of LUM and IVA [2]
    End point description
    End point type
    Primary
    End point timeframe
    Day 1 up to Day 6 for each treatment period
    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 planned. No statistical comparisons were planned for the primary PK endpoint. PK set included participants who received at least 1 dose of study drug and for whom the primary PK data were considered to be sufficient and interpretable. Here “Number Analyzed” signifies those participants who were evaluable for this outcome measure in the specified treatment period.
    End point values
    		 Treatment A: LUM/IVA Dose 1 Tablet (Fed) Treatment B: LUM/IVA Dose 1 Granule (Fasted) Treatment B: LUM/IVA Dose 1 Granule (Fed) Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Number of subjects analysed
    16
    15
    16
    15
    Units: Hours*nanogram per milliliter (h*ng/mL)
    arithmetic mean (standard deviation)
        LUM
    133000 ( 35100 )
    105000 ( 24600 )
    118000 ( 22400 )
    184000 ( 38800 )
        IVA
    7400 ( 2590 )
    4070 ( 1560 )
    7160 ( 1880 )
    11100 ( 2840 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From first dose of study drug up to 10 days after last dose of study drug in treatment period 4
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Treatment A: LUM/IVA Dose 1 Tablet (Fed)
    Reporting group description
    		 All subjects who received LUM/IVA Dose 1 tablet formulation in fed state.

    Reporting group title
    Treatment B: LUM/IVA Dose 1 Granule (Fasted)
    Reporting group description
    		 All Subjects who received LUM/IVA Dose 1 granule formulation in fasted state.

    Reporting group title
    Treatment B: LUM/IVA Dose 1 Granule (Fed)
    Reporting group description
    		 All Subjects who received LUM/IVA Dose 1 granule formulation in fed state.

    Reporting group title
    Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Reporting group description
    		 All Subjects who received LUM/IVA Dose 2 granule formulation in fed state.

    Serious adverse events
    		 Treatment A: LUM/IVA Dose 1 Tablet (Fed) Treatment B: LUM/IVA Dose 1 Granule (Fasted) Treatment B: LUM/IVA Dose 1 Granule (Fed) Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 15 (0.00%)
         number of deaths (all causes)
    0
    0
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Gun shot wound
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 15 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Treatment A: LUM/IVA Dose 1 Tablet (Fed) Treatment B: LUM/IVA Dose 1 Granule (Fasted) Treatment B: LUM/IVA Dose 1 Granule (Fed) Treatment C: LUM/IVA Dose 2 Granule (Fed)
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    5 / 16 (31.25%)
    2 / 15 (13.33%)
    3 / 16 (18.75%)
    6 / 15 (40.00%)
    Injury, poisoning and procedural complications
    Arthropod bite
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 16 (18.75%)
    2 / 15 (13.33%)
    1 / 16 (6.25%)
    2 / 15 (13.33%)
         occurrences all number
    3
    2
    1
    2
    Eye disorders
    Dry eye
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0
    Gastrointestinal disorders
    Nausea
         subjects affected / exposed
    1 / 16 (6.25%)
    1 / 15 (6.67%)
    2 / 16 (12.50%)
    0 / 15 (0.00%)
         occurrences all number
    1
    1
    2
    0
    Dyspepsia
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Toothache
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    1 / 15 (6.67%)
         occurrences all number
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Eczema
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    2 / 15 (13.33%)
         occurrences all number
    0
    0
    0
    2
    Erythema
         subjects affected / exposed
    0 / 16 (0.00%)
    0 / 15 (0.00%)
    1 / 16 (6.25%)
    0 / 15 (0.00%)
         occurrences all number
    0
    0
    1
    0
    Infections and infestations
    Folliculitis
         subjects affected / exposed
    1 / 16 (6.25%)
    0 / 15 (0.00%)
    0 / 16 (0.00%)
    0 / 15 (0.00%)
         occurrences all number
    1
    0
    0
    0

    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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