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    Clinical Trial Results:
    A Phase 3b Open-label Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis

    Summary
    EudraCT number
    		 2020-004885-21
    Trial protocol
    		 CZ   BE   NL   ES  
    Global end of trial date
    20 Dec 2022

    Results information
    Results version number
    		 v1(current)
    This version publication date
    05 Jul 2023
    First version publication date
    05 Jul 2023
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX20-445-121
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05111145
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Vertex Pharmaceuticals Incorporated
    Sponsor organisation address
    50 Northern Avenue, Boston, 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)
    No
    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
    06 Feb 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    20 Dec 2022
    Global end of trial reached?
    Yes
    Global end of trial date
    20 Dec 2022
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the safety and tolerability of Elexacaftor (ELX)/Tezacaftor (TEZ)/Ivacaftor (IVA) in subjects with Cystic Fibrosis (CF).
    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
    14 Jan 2022
    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
    Australia: 24
    Country: Number of subjects enrolled
    Canada: 1
    Country: Number of subjects enrolled
    Spain: 20
    Country: Number of subjects enrolled
    Belgium: 38
    Country: Number of subjects enrolled
    Czechia: 3
    Worldwide total number of subjects
    86
    EEA total number of subjects
    61
    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)
    27
    Adults (18-64 years)
    59
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 Subjects from Parent Studies VX19-445-117(NCT04599465) and VX20-445-126(NCT04969224) were enrolled in this study. A total of 86 subjects were enrolled in this study.

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

    Arms
    Arm title
    		 ELX/TEZ/IVA
    Arm description
    		 Subjects received Elexacaftor (ELX) 200 mg once daily (qd)/Tezacaftor (TEZ) 100 mg qd/Ivacaftor (IVA) 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    ELX/TEZ/IVA
    Investigational medicinal product code
    VX-445/VX-661/VX-770
    Other name
    Elexacaftor/Tezacaftor/Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received ELX/TEZ/IVA fixed-dose combination once daily in the morning.

    Investigational medicinal product name
    IVA
    Investigational medicinal product code
    VX-770
    Other name
    Ivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received IVA once daily in the evening.

    Number of subjects in period 1
    		ELX/TEZ/IVA
    Started
    86
    Completed
    0
    Not completed
    86
         Commercial drug available to the subject
    76
         Subject enrolled in another qualified Vertex study
    10

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Overall Trial (overall period)
    Reporting group description
    		 Baseline data is based on the parent study baseline, which is defined as the most recent non-missing measurement collected before the first dose of study drug in the treatment period of parent studies. Baseline data is presented for subjects who received at least 1 dose of study drug in this study.

    Reporting group values
    		 Overall Trial (overall period) Total
    Number of subjects
    		 86 86
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 25.3 ( 9.9 ) -
    Gender categorical
    Units: Subjects
        Female
    		 37 37
        Male
    		 49 49
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 11 11
        Not Hispanic or Latino
    		 68 68
        Not collected per local regulations
    		 7 7
    Race
    Units: Subjects
        White
    		 79 79
        Black or African American
    		 0 0
        Asian
    		 0 0
        American Indian or Alaska Native
    		 0 0
        Native Hawaiian or other Pacific Islander
    		 0 0
        Other
    		 0 0
        Not collected per local regulations
    		 7 7

    End points

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    End points reporting groups
    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Subjects received Elexacaftor (ELX) 200 mg once daily (qd)/Tezacaftor (TEZ) 100 mg qd/Ivacaftor (IVA) 150 mg every 12 hours (q12h) in the treatment period for up to 36 weeks.

    Primary: 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
    		 Safety and Tolerability as Assessed by Number of Subjects With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [1]
    End point description
    Safety set included all subjects who received at least 1 dose of study drug in the treatment period.
    End point type
    Primary
    End point timeframe
    Day 1 up to Week 36
    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
    		 ELX/TEZ/IVA
    Number of subjects analysed
    86
    Units: Subjects
        Subjects with TEAEs
    61
        Subjects with SAEs
    4
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Week 36
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    25.1
    Reporting groups
    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Subjects received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 36 weeks.

    Serious adverse events
    		 ELX/TEZ/IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    4 / 86 (4.65%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Psychiatric disorders
    Anxiety
         subjects affected / exposed
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Infections and infestations
    Gastroenteritis viral
         subjects affected / exposed
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Metabolism and nutrition disorders
    Malnutrition
         subjects affected / exposed
    1 / 86 (1.16%)
         occurrences causally related to treatment / all
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 ELX/TEZ/IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    33 / 86 (38.37%)
    Infections and infestations
    COVID-19
         subjects affected / exposed
    16 / 86 (18.60%)
         occurrences all number
    16
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    11 / 86 (12.79%)
         occurrences all number
    14
    Nasopharyngitis
         subjects affected / exposed
    9 / 86 (10.47%)
         occurrences all number
    9
    Upper respiratory tract infection
         subjects affected / exposed
    6 / 86 (6.98%)
         occurrences all number
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    14 Feb 2022
    Updated that study drug does not need to be discontinued for a male subject whose female partner becomes pregnant during study participation; Removed parent study VX19-445-114 and updated the number of planned subjects.

    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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