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    Clinical Trial Results:
    A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

    Summary
    EudraCT number
    		 2017-004794-13
    Trial protocol
    		 Outside EU/EEA  
    Global end of trial date
    29 Oct 2021

    Results information
    Results version number
    		 v2(current)
    This version publication date
    26 Jan 2023
    First version publication date
    14 May 2022
    Other versions
    		 v1
    Version creation reason
    • New data added to full data set
    Addition of secondary outcomes

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    VX16-809-122
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03601637
    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?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    19 Nov 2021
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    29 Oct 2021
    Global end of trial reached?
    Yes
    Global end of trial date
    29 Oct 2021
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than (<) 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
    Protection of trial subjects
    The study was conducted in accordance with the ethical principles stated in the Declaration of Helsinki and the International Conference on Harmonization (ICH) Guideline for Good Clinical Practice (GCP).
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Sep 2018
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United States: 49
    Country: Number of subjects enrolled
    Canada: 12
    Worldwide total number of subjects
    61
    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)
    61
    Children (2-11 years)
    0
    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
    		 -

    Pre-assignment
    Screening details
    		 This study was conducted in subjects with CF aged 1 through less than 2 years of age who are homozygous for F508del.

    Period 1
    Period 1 title
    Overall Trial (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Part A: LUM/IVA
    Arm description
    		 Subjects weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Subjects weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/VX-770
    Other name
    Lumacaftor/Ivacaftor
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received LUM/IVA fixed-dose combination (FDC) every 12 hours.

    Arm title
    		 Part B: LUM/IVA
    Arm description
    		 Subjects weighing 7 to <9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
    Arm type
    		 Experimental

    Investigational medicinal product name
    LUM/IVA
    Investigational medicinal product code
    VX-809/VX-770
    Other name
    Lumacaftor/Ivacaftor
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received LUM/IVA FDC every 12 hours.

    Number of subjects in period 1 [1]
    		Part A: LUM/IVA Part B: LUM/IVA
    Started
    14
    46
    Completed
    13
    43
    Not completed
    1
    3
         Adverse Event
    1
    1
         Other
    -
    1
         Withdrawal of Consent (not due to AE)
    -
    1
    Notes
    [1] - The number of subjects reported to be in the baseline period are not the same as the worldwide number enrolled in the trial. It is expected that these numbers will be the same.
    Justification: A total of 61 subjects were enrolled in Parts A and B of the study. One subject in Part B was enrolled but not dosed in this study. Therefore, data for 60 subjects are reported in the subject disposition and baseline characteristics sections.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Part A: LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Subjects weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.

    Reporting group title
    Part B: LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to <9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.

    Reporting group values
    		 Part A: LUM/IVA Part B: LUM/IVA Total
    Number of subjects
    		 14 46 60
    Age categorical
    Units: Subjects
    Age continuous
    Units: months
        arithmetic mean (standard deviation)
    		 17.2 ( 3.6 ) 18.1 ( 3.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 7 24 31
        Male
    		 7 22 29
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 1 2 3
        Not Hispanic or Latino
    		 13 37 50
        Unknown or Not Reported
    		 0 7 7
    Race
    Units: Subjects
        White
    		 14 34 48
        Black or African American
    		 0 1 1
        Asian
    		 0 0 0
        American Indian or Alaska Native
    		 0 1 1
        Native Hawaiian or Other Pacific Islander
    		 0 0 0
        Not collected per local regulations
    		 0 7 7
        Other
    		 0 3 3

    End points

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    End points reporting groups
    Reporting group title
    Part A: LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Subjects weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.

    Reporting group title
    Part B: LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to <9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.

    Subject analysis set title
    Part A: LUM 75 mg/IVA 94 mg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received LUM 75 mg/IVA 94 mg dose every 12 hours for 15 days.

    Subject analysis set title
    Part A: LUM 100 mg/IVA 125 mg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received LUM 100 mg/IVA 125 mg dose every 12 hours for 15 days.

    Subject analysis set title
    Part B: LUM 75 mg/IVA 94 mg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received LUM 75 mg/IVA 94 mg dose every 12 hours for 24 weeks.

    Subject analysis set title
    Part B: LUM 100 mg/IVA 125 mg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received LUM 100 mg/IVA 125 mg dose every 12 hours for 24 weeks.

    Subject analysis set title
    Part B: LUM 150 mg/IVA 188 mg
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Subjects received LUM 150 mg/IVA 188 mg dose every 12 hours for 24 weeks.

    Primary: Part A: Observed Plasma Concentrations From 3-4 hours (C3-4hr) of LUM and IVA

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    End point title
    		 Part A: Observed Plasma Concentrations From 3-4 hours (C3-4hr) of LUM and IVA [1]
    End point description
    PK set included subjects who received at least 1 dose of study drug. Here “n” signifies those subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Primary
    End point timeframe
    Day 1 and Day 15
    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 this endpoint.
    End point values
    		 Part A: LUM 75 mg/IVA 94 mg Part A: LUM 100 mg/IVA 125 mg
    Number of subjects analysed
    7
    7
    Units: nanograms per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        Day 1: LUM (n=7,7)
    14600 ( 5560 )
    12600 ( 7190 )
        Day 15: LUM (n=7,5)
    16600 ( 9590 )
    13900 ( 5800 )
        Day 1: IVA (n=7,7)
    1620 ( 648 )
    1320 ( 804 )
        Day 15: IVA (n=7,5)
    718 ( 352 )
    496 ( 268 )
    No statistical analyses for this end point

    Primary: Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA

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    End point title
    		 Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA [2]
    End point description
    PK set included subjects who received at least 1 dose of study drug. Here “n” signifies those subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Primary
    End point timeframe
    Pre-dose at Day 8 and Day 15.
    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 this endpoint.
    End point values
    		 Part A: LUM 75 mg/IVA 94 mg Part A: LUM 100 mg/IVA 125 mg
    Number of subjects analysed
    7
    7
    Units: nanograms per milliliter (ng/mL)
    arithmetic mean (standard deviation)
        Day 8: LUM (n=7,6)
    12000 ( 8880 )
    12800 ( 3900 )
        Day 15: LUM (n=5,6)
    8380 ( 7790 )
    10500 ( 3070 )
        Day 8: IVA (n=7,6)
    169 ( 75.5 )
    185 ( 101 )
        Day 15: IVA (n=5,6)
    78.9 ( 19.1 )
    120 ( 60.5 )
    No statistical analyses for this end point

    Primary: Part B : Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

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    End point title
    		 Part B : Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [3] [4]
    End point description
    Safety set included all subjects who received at least 1 dose of study drug in the treatment period. The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part B: LUM/IVA arm.
    End point type
    Primary
    End point timeframe
    From Day 1 up to Week 26
    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 planned. No statistical comparisons were planned for this endpoint.
    [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: This primary endpoint is only applicable for Part B.
    End point values
    		 Part B: LUM/IVA
    Number of subjects analysed
    46
    Units: subjects
        Subjects with TEAEs
    44
        Subjects with SAEs
    5
    No statistical analyses for this end point

    Secondary: Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events

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    End point title
    		 Part A: Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events [5]
    End point description
    Safety set included all subjects who received at least 1 dose of study drug in the treatment period. The safety and tolerability analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part A: LUM/IVA arm.
    End point type
    Secondary
    End point timeframe
    From Day 1 up to Day 25
    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: This primary endpoint is only applicable for Part A.
    End point values
    		 Part A: LUM/IVA
    Number of subjects analysed
    14
    Units: Subjects
        Subjects with TEAEs
    12
        Subjects with SAEs
    0
    No statistical analyses for this end point

    Secondary: Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)

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    End point title
    		 Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
    End point description
    PK set included subjects who received at least 1 dose of study drug. Here “n” signifies those subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Secondary
    End point timeframe
    Pre-dose at Day 8 and Day 15
    End point values
    		 Part A: LUM 75 mg/IVA 94 mg Part A: LUM 100 mg/IVA 125 mg
    Number of subjects analysed
    7
    7
    Units: nanogram(s)/millilitre
    arithmetic mean (standard deviation)
        Day 8: M28-LUM (n=7,6)
    1410 ( 791 )
    1470 ( 365 )
        Day 15: M28-LUM (n=5,6)
    1460 ( 1060 )
    1370 ( 468 )
        Day 8: M1-IVA (n=7,6)
    606 ( 298 )
    842 ( 527 )
        Day 15: M1-IVA (n=5,6)
    284 ( 70.2 )
    604 ( 394 )
        Day 8: M6-IVA (n=7,6)
    2380 ( 843 )
    3290 ( 1830 )
        Day 15: M6-IVA (n=5,6)
    1460 ( 763 )
    2800 ( 2300 )
    No statistical analyses for this end point

    Secondary: Part B: Absolute Change in Sweat Chloride

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    End point title
    		 Part B: Absolute Change in Sweat Chloride [6]
    End point description
    The Full Analysis Set (FAS) included all enrolled subjects who were exposed to any amount of study drug in Part B. The pharmacodynamic analyses were assessed for the overall treatment arm, irrespective of weight-based dose regimen. Therefore, the analysis is reported in a single Part B: LUM/IVA arm.
    End point type
    Secondary
    End point timeframe
    From Baseline at Week 24
    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: This secondary endpoint is only applicable for Part B.
    End point values
    		 Part B: LUM/IVA
    Number of subjects analysed
    46
    Units: millimole(s)/litre
        arithmetic mean (standard deviation)
    -29.1 ( 13.5 )
    No statistical analyses for this end point

    Secondary: Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)

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    End point title
    		 Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)
    End point description
    PK set included subjects who received at least 1 dose of study drug. Here “n” signifies those subjects who were evaluable at specified time points for each reporting group respectively.Here 99999 represents "not applicable" categories for Ctrough.
    End point type
    Secondary
    End point timeframe
    Pre-dose at Day 15, Week 4, Week 12 and Week 24
    End point values
    		 Part B: LUM 75 mg/IVA 94 mg Part B: LUM 100 mg/IVA 125 mg Part B: LUM 150 mg/IVA 188 mg
    Number of subjects analysed
    1
    44
    1
    Units: nanogram(s)/millilitre
    arithmetic mean (standard deviation)
        Day 15: LUM (n=1,43,1)
    13500 ( 99999 )
    9030 ( 4390 )
    887 ( 99999 )
        Week 4: LUM (n=1,43,1)
    21900 ( 99999 )
    9370 ( 5030 )
    1000 ( 99999 )
        Week 12: LUM (n=1,41,1)
    12100 ( 99999 )
    9160 ( 4400 )
    566 ( 99999 )
        Week 24: LUM (n=1,35,1)
    11200 ( 99999 )
    8930 ( 5310 )
    288 ( 99999 )
        Day 15: M28-LUM (n=1,43,1)
    1460 ( 99999 )
    1260 ( 528 )
    786 ( 99999 )
        Week 4: M28-LUM (n=1,43,1)
    1570 ( 99999 )
    1340 ( 579 )
    602 ( 99999 )
        Week 12: M28-LUM (n=1,41,1)
    1740 ( 99999 )
    1440 ( 599 )
    501 ( 99999 )
        Week 24: M28-LUM (n=1,35,1)
    1870 ( 99999 )
    1470 ( 548 )
    569 ( 99999 )
        Day 15: IVA (n=1,43,1)
    213 ( 99999 )
    112 ( 68.3 )
    3.68 ( 99999 )
        Week 4: IVA (n=1,43,1)
    267 ( 99999 )
    112 ( 82.9 )
    11.4 ( 99999 )
        Week 12: IVA (n=1,41,1)
    286 ( 99999 )
    91.7 ( 44.7 )
    2.18 ( 99999 )
        Week 24: IVA (1,34,1)
    37.1 ( 99999 )
    144 ( 296 )
    2.86 ( 99999 )
        Day 15: M1-IVA (n=1,43,1)
    1420 ( 99999 )
    437 ( 291 )
    18.1 ( 99999 )
        Week 4: M1-IVA (n=1,43,1)
    1930 ( 99999 )
    466 ( 359 )
    58.0 ( 99999 )
        Week 12: M1-IVA (n=1,41,1)
    759 ( 99999 )
    418 ( 236 )
    18.3 ( 99999 )
        Week 24: M1-IVA (n=1,35,1)
    256 ( 99999 )
    499 ( 613 )
    18.5 ( 99999 )
        Day 15: M6-IVA (n=1,43,1)
    4740 ( 9999 )
    1930 ( 1460 )
    43.9 ( 99999 )
        Week 4: M6-IVA (n=1,43,1)
    9090 ( 99999 )
    1970 ( 1490 )
    131 ( 99999 )
        Week 12: M6-IVA (n=1,41,1)
    3520 ( 99999 )
    1950 ( 1430 )
    46.5 ( 99999 )
        Week 24: M6-IVA (n=1,35,1)
    1320 ( 99999 )
    1740 ( 1130 )
    63.3 ( 99999 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    From Day 1 Through Safety Follow-up Period (up to Day 25 for Part A and up to Week 26 for Part B)
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    24.1
    Reporting groups
    Reporting group title
    Part A : LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to <10 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Subjects >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.

    Reporting group title
    Part B: LUM/IVA
    Reporting group description
    		 Subjects weighing 7 to <9 kg at screening received LUM 75 mg/ IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg FDC q12h for 24 weeks. Subjects weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.

    Serious adverse events
    		 Part A : LUM/IVA Part B: LUM/IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 14 (0.00%)
    5 / 46 (10.87%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Injury, poisoning and procedural complications
    Post procedural fever
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    0 / 14 (0.00%)
    1 / 46 (2.17%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    0 / 14 (0.00%)
    3 / 46 (6.52%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 4
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Part A : LUM/IVA Part B: LUM/IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 14 (85.71%)
    42 / 46 (91.30%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 14 (0.00%)
    4 / 46 (8.70%)
         occurrences all number
    0
    4
    Pseudomonas test positive
         subjects affected / exposed
    0 / 14 (0.00%)
    5 / 46 (10.87%)
         occurrences all number
    0
    5
    Injury, poisoning and procedural complications
    Lip injury
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 46 (0.00%)
         occurrences all number
    1
    0
    General disorders and administration site conditions
    Crying
         subjects affected / exposed
    1 / 14 (7.14%)
    0 / 46 (0.00%)
         occurrences all number
    1
    0
    Pyrexia
         subjects affected / exposed
    1 / 14 (7.14%)
    10 / 46 (21.74%)
         occurrences all number
    1
    15
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 14 (0.00%)
    3 / 46 (6.52%)
         occurrences all number
    0
    3
    Constipation
         subjects affected / exposed
    1 / 14 (7.14%)
    5 / 46 (10.87%)
         occurrences all number
    1
    7
    Diarrhoea
         subjects affected / exposed
    0 / 14 (0.00%)
    3 / 46 (6.52%)
         occurrences all number
    0
    3
    Vomiting
         subjects affected / exposed
    0 / 14 (0.00%)
    8 / 46 (17.39%)
         occurrences all number
    0
    9
    Flatulence
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 46 (2.17%)
         occurrences all number
    1
    1
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    4 / 14 (28.57%)
    16 / 46 (34.78%)
         occurrences all number
    4
    27
    Nasal congestion
         subjects affected / exposed
    1 / 14 (7.14%)
    4 / 46 (8.70%)
         occurrences all number
    1
    6
    Rhinorrhoea
         subjects affected / exposed
    5 / 14 (35.71%)
    5 / 46 (10.87%)
         occurrences all number
    5
    9
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    3 / 14 (21.43%)
    4 / 46 (8.70%)
         occurrences all number
    4
    4
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 46 (2.17%)
         occurrences all number
    1
    1
    Infections and infestations
    Ear infection
         subjects affected / exposed
    0 / 14 (0.00%)
    5 / 46 (10.87%)
         occurrences all number
    0
    5
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    0 / 14 (0.00%)
    8 / 46 (17.39%)
         occurrences all number
    0
    12
    Influenza
         subjects affected / exposed
    2 / 14 (14.29%)
    0 / 46 (0.00%)
         occurrences all number
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    0 / 14 (0.00%)
    4 / 46 (8.70%)
         occurrences all number
    0
    4
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 14 (7.14%)
    6 / 46 (13.04%)
         occurrences all number
    1
    9
    Viral upper respiratory tract infection
         subjects affected / exposed
    0 / 14 (0.00%)
    5 / 46 (10.87%)
         occurrences all number
    0
    6
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 14 (7.14%)
    1 / 46 (2.17%)
         occurrences all number
    1
    1

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    04 Dec 2019
    Amended to update the planned dosing regimen to add lower dose of LUM/IVA in Parts A and B and, to adjust the weight bound of LUM/IVA in Part B.

    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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