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    Clinical Trial Results:
    A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-121 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for F508del and a Minimal Function Mutation (F/MF)

    Summary
    EudraCT number
    		 2021-000712-31
    Trial protocol
    		 SE   DE   CZ   IE   ES   PT   HU  
    Global end of trial date
    21 Nov 2023

    Results information
    Results version number
    		 v1(current)
    This version publication date
    26 May 2024
    First version publication date
    26 May 2024
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    VX20-121-102
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT05033080
    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-003052-PIP01-21
    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
    14 Dec 2023
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    12 May 2023
    Global end of trial reached?
    Yes
    Global end of trial date
    21 Nov 2023
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in cystic fibrosis (CF) subjects who are heterozygous for F508del and a minimal function mutation (F/MF subjects)
    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 Sep 2021
    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
    Ireland: 7
    Country: Number of subjects enrolled
    United States: 199
    Country: Number of subjects enrolled
    Germany: 63
    Country: Number of subjects enrolled
    United Kingdom: 42
    Country: Number of subjects enrolled
    Spain: 26
    Country: Number of subjects enrolled
    Sweden: 18
    Country: Number of subjects enrolled
    Australia: 19
    Country: Number of subjects enrolled
    New Zealand: 17
    Country: Number of subjects enrolled
    Israel: 17
    Country: Number of subjects enrolled
    Hungary: 13
    Country: Number of subjects enrolled
    Portugal: 8
    Country: Number of subjects enrolled
    Czechia: 6
    Worldwide total number of subjects
    435
    EEA total number of subjects
    141
    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)
    61
    Adults (18-64 years)
    372
    From 65 to 84 years
    2
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 This study was conducted in cystic fibrosis (CF) subjects aged 12 years or older. It was pre-specified in the protocol to combine the data from this study with study VX20-121-103 (NCT05076149) for selected endpoints.

    Pre-assignment
    Screening details
    		 A total of 435 subjects were enrolled in this study, of which 37 were included in the run-in period but were not dosed in treatment period. Therefore, results are presented for only 398 subjects dosed in the treatment period.

    Period 1
    Period 1 title
    Overall Period
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Carer, Assessor

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 ELX/TEZ/IVA
    Arm description
    		 Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, subjects received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.
    Arm type
    		 Active comparator

    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 (FDC) 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.

    Arm title
    		 VX-121/TEZ/D-IVA
    Arm description
    		 Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    VX-121/TEZ/D-IVA
    Investigational medicinal product code
    VX-121/VX-661/VX-561
    Other name
    VX-121/tezacaftor/deutivacaftor
    Pharmaceutical forms
    Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    Subjects received VX-121/TEZ/D-IVA fixed-dose combination (FDC) once daily in the morning.

    Number of subjects in period 1 [1]
    		ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Started
    202
    196
    Completed
    191
    184
    Not completed
    11
    12
         Physician decision
    -
    1
         Other
    1
    1
         Adverse event
    4
    4
         Lost to follow-up
    1
    -
         Other non-compliance
    -
    1
         Withdrawal of consent (not due to AE)
    5
    5
    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 435 subjects were enrolled in the study, of which 37 were included in the run-in period but were not dosed in treatment period. Therefore, results are presented for only 398 subjects dosed in the treatment period.

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, subjects received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.

    Reporting group title
    VX-121/TEZ/D-IVA
    Reporting group description
    		 Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.

    Reporting group values
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA Total
    Number of subjects
    		 202 196 398
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 30.9 ( 11.4 ) 30.8 ( 10.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 83 80 163
        Male
    		 119 116 235
    Race
    Units: Subjects
        Hispanic or Latino
    		 11 13 24
        Not Hispanic or Latino
    		 190 183 373
        Not Collected per Local Regulations
    		 1 0 1
    Ethnicity
    Units: Subjects
        White
    		 197 191 388
        Black or African American
    		 1 4 5
        Asian
    		 0 1 1
        Other
    		 1 0 1
        More than one race
    		 3 0 3
    Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
    Units: Percentage points
        arithmetic mean (standard deviation)
    		 67.2 ( 14.6 ) 67.0 ( 15.3 ) -

    End points

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    End points reporting groups
    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Following elexacftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) run-in period of 4 weeks, subjects received ELX 200 milligram (mg) once daily (qd) /TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period for 52 weeks.

    Reporting group title
    VX-121/TEZ/D-IVA
    Reporting group description
    		 Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.

    Primary: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)

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    End point title
    		 Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
    End point description
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. The Full Analysis Set (FAS) included all all randomized subjects who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point.
    End point type
    Primary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Number of subjects analysed
    193
    187
    Units: percentage points
        least squares mean (confidence interval 95%)
    0.3 (-0.3 to 0.9)
    0.5 (-0.1 to 1.1)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    ELX/TEZ/IVA v VX-121/TEZ/D-IVA
    Number of subjects included in analysis
    380
    Analysis specification
    Pre-specified
    Analysis type
    		 non-inferiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    0.2
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.7
         upper limit
    		 1.1

    Secondary: Absolute Change in Sweat Chloride (SwCl)

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    End point title
    		 Absolute Change in Sweat Chloride (SwCl)
    End point description
    Sweat samples were collected using an approved collection device. FAS.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Number of subjects analysed
    194
    185
    Units: millimole per liter (mmol/L)
        least squares mean (confidence interval 95%)
    0.9 (-0.6 to 2.3)
    -7.5 (-9.0 to -6.0)
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    ELX/TEZ/IVA v VX-121/TEZ/D-IVA
    Number of subjects included in analysis
    379
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.0001
    Method
    		 Mixed Model Repeated Measures
    Parameter type
    		 LS Mean difference
    Point estimate
    -8.4
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -10.5
         upper limit
    		 -6.3

    Secondary: Percentage of Subjects With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103)

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    End point title
    		 Percentage of Subjects With SwCl <60 mmol/L (Pooled With Data From Study VX20-121-103)
    End point description
    The Pooled Full Analysis Set (PFAS) included all randomized subjects from this study (VX20-121-102) and from Study VX20-121-103 who carried the intended CFTR mutation(s) and received at least 1 dose of study drug during the Treatment Period. Here “Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point. Here “n” signifies pooled analysis subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Number of subjects analysed
    202 [1]
    196 [2]
    Units: percentage of subjects
    number (not applicable)
        n=491,480
    76.6
    85.8
    Notes
    [1] - Out of 491 subjects, 202 subjects were enrolled in the 121–102 and 289 subjects in the 121–103 study
    [2] - Out of 491 subjects, 196 subjects were enrolled in the 121–102 and 284 subjects in the 121–103 study
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    VX-121/TEZ/D-IVA v ELX/TEZ/IVA
    Number of subjects included in analysis
    398
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 < 0.0001
    Method
    		 Generalized Estimated Equation Model
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.21
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 1.55
         upper limit
    		 3.15

    Secondary: Percentage of Subjects With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103)

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    End point title
    		 Percentage of Subjects With SwCl <30 mmol/L (Pooled With Data From Study VX20-121-103)
    End point description
    PFAS. Here “ Number of Subjects Analyzed” signifies those subjects who were evaluated for this specific end point. Here “n” signifies pooled analysis subjects who were evaluable at specified time points for each reporting group respectively.
    End point type
    Secondary
    End point timeframe
    From Baseline Through Week 24
    End point values
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Number of subjects analysed
    202 [3]
    196 [4]
    Units: percentage of subjects
    number (not applicable)
        n=491,480
    22.5
    30.5
    Notes
    [3] - Out of 491 subjects, 202 subjects were enrolled in the 121–102 and 289 subjects in the 121–103 study
    [4] - Out of 480 subjects, 196 subjects were enrolled in the 121–102 and 284 subjects in the 121–103 study
    Statistical analysis title
    		 Statistical Analysis 1
    Comparison groups
    ELX/TEZ/IVA v VX-121/TEZ/D-IVA
    Number of subjects included in analysis
    398
    Analysis specification
    Pre-specified
    Analysis type
    		 other
    P-value
    		 < 0.0001
    Method
    		 Generalized Estimated Equation Model
    Parameter type
    		 Odds ratio (OR)
    Point estimate
    2.87
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 2
         upper limit
    		 4.12

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Day 1 up to Safety follow-up (up to 56 weeks)
    Adverse event reporting additional description
    Safety set include all subjects who received at least 1 dose of study drug during the Treatment Period.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    26.1
    Reporting groups
    Reporting group title
    ELX/TEZ/IVA
    Reporting group description
    		 Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 52 weeks.

    Reporting group title
    VX-121/TEZ/D-IVA
    Reporting group description
    		 Following ELX/TEZ/IVA run-in period of 4 weeks, subjects received VX-121 20 mg qd/TEZ 100 mg qd/D-IVA 250 mg qd in the treatment period for 52 weeks.

    Serious adverse events
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Total subjects affected by serious adverse events
         subjects affected / exposed
    41 / 202 (20.30%)
    28 / 196 (14.29%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    Neoplasms benign, malignant and unspecified (incl cysts and polyps)
    Adenocarcinoma
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Immune system disorders
    Hypersensitivity
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Haemoptysis
         subjects affected / exposed
    2 / 202 (0.99%)
    2 / 196 (1.02%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nasal polyps
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Psychiatric disorders
    Depression suicidal
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Mixed anxiety and depressive disorder
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Suicidal ideation
         subjects affected / exposed
    1 / 202 (0.50%)
    2 / 196 (1.02%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Depression
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Behaviour disorder
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    2 / 202 (0.99%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    2 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Aspartate aminotransferase increased
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood alkaline phosphatase increased
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pulmonary function test decreased
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gamma-glutamyltransferase increased
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Injury, poisoning and procedural complications
    Postoperative ileus
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Alcohol poisoning
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Congenital, familial and genetic disorders
    Cerebrovascular arteriovenous malformation
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cardiac disorders
    Arteriospasm coronary
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Nervous system disorders
    Headache
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Epilepsy
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Serotonin syndrome
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Syncope
         subjects affected / exposed
    0 / 202 (0.00%)
    2 / 196 (1.02%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    2 / 202 (0.99%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Subileus
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Small intestinal obstruction
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pancreatitis
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Mechanical ileus
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Dysphagia
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Distal intestinal obstruction syndrome
         subjects affected / exposed
    1 / 202 (0.50%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Hepatobiliary disorders
    Cholecystitis
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cholelithiasis
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cholestasis
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infections and infestations
    Diverticulitis
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Cellulitis
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    23 / 202 (11.39%)
    11 / 196 (5.61%)
         occurrences causally related to treatment / all
    0 / 27
    0 / 12
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Appendicitis
         subjects affected / exposed
    1 / 202 (0.50%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    COVID-19
         subjects affected / exposed
    3 / 202 (1.49%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 3
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    0 / 202 (0.00%)
    2 / 196 (1.02%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Meningitis aseptic
         subjects affected / exposed
    1 / 202 (0.50%)
    0 / 196 (0.00%)
         occurrences causally related to treatment / all
    1 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    1 / 202 (0.50%)
    3 / 196 (1.53%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 3
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Urinary tract infection
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Viral myocarditis
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Metabolism and nutrition disorders
    Hyperphosphatasaemia
         subjects affected / exposed
    0 / 202 (0.00%)
    1 / 196 (0.51%)
         occurrences causally related to treatment / all
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 ELX/TEZ/IVA VX-121/TEZ/D-IVA
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    184 / 202 (91.09%)
    177 / 196 (90.31%)
    Investigations
    Blood creatine phosphokinase increased
         subjects affected / exposed
    23 / 202 (11.39%)
    18 / 196 (9.18%)
         occurrences all number
    27
    22
    Aspartate aminotransferase increased
         subjects affected / exposed
    8 / 202 (3.96%)
    12 / 196 (6.12%)
         occurrences all number
    10
    15
    Alanine aminotransferase increased
         subjects affected / exposed
    10 / 202 (4.95%)
    13 / 196 (6.63%)
         occurrences all number
    11
    17
    Nervous system disorders
    Headache
         subjects affected / exposed
    22 / 202 (10.89%)
    25 / 196 (12.76%)
         occurrences all number
    40
    32
    General disorders and administration site conditions
    Pyrexia
         subjects affected / exposed
    21 / 202 (10.40%)
    24 / 196 (12.24%)
         occurrences all number
    30
    27
    Fatigue
         subjects affected / exposed
    16 / 202 (7.92%)
    18 / 196 (9.18%)
         occurrences all number
    21
    23
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    14 / 202 (6.93%)
    10 / 196 (5.10%)
         occurrences all number
    18
    11
    Abdominal distension
         subjects affected / exposed
    10 / 202 (4.95%)
    5 / 196 (2.55%)
         occurrences all number
    11
    5
    Abdominal pain upper
         subjects affected / exposed
    6 / 202 (2.97%)
    12 / 196 (6.12%)
         occurrences all number
    9
    14
    Nausea
         subjects affected / exposed
    17 / 202 (8.42%)
    7 / 196 (3.57%)
         occurrences all number
    20
    7
    Diarrhoea
         subjects affected / exposed
    15 / 202 (7.43%)
    21 / 196 (10.71%)
         occurrences all number
    15
    30
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    41 / 202 (20.30%)
    45 / 196 (22.96%)
         occurrences all number
    57
    59
    Productive cough
         subjects affected / exposed
    5 / 202 (2.48%)
    12 / 196 (6.12%)
         occurrences all number
    7
    12
    Haemoptysis
         subjects affected / exposed
    10 / 202 (4.95%)
    13 / 196 (6.63%)
         occurrences all number
    15
    16
    Nasal congestion
         subjects affected / exposed
    24 / 202 (11.88%)
    19 / 196 (9.69%)
         occurrences all number
    30
    25
    Oropharyngeal pain
         subjects affected / exposed
    23 / 202 (11.39%)
    24 / 196 (12.24%)
         occurrences all number
    29
    34
    Sputum increased
         subjects affected / exposed
    21 / 202 (10.40%)
    18 / 196 (9.18%)
         occurrences all number
    28
    24
    Sinus congestion
         subjects affected / exposed
    5 / 202 (2.48%)
    14 / 196 (7.14%)
         occurrences all number
    8
    21
    Rhinorrhoea
         subjects affected / exposed
    14 / 202 (6.93%)
    19 / 196 (9.69%)
         occurrences all number
    18
    25
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    9 / 202 (4.46%)
    12 / 196 (6.12%)
         occurrences all number
    10
    16
    Musculoskeletal and connective tissue disorders
    Back pain
         subjects affected / exposed
    16 / 202 (7.92%)
    9 / 196 (4.59%)
         occurrences all number
    18
    11
    Arthralgia
         subjects affected / exposed
    11 / 202 (5.45%)
    10 / 196 (5.10%)
         occurrences all number
    14
    11
    Infections and infestations
    COVID-19
         subjects affected / exposed
    53 / 202 (26.24%)
    49 / 196 (25.00%)
         occurrences all number
    54
    53
    Infective pulmonary exacerbation of cystic fibrosis
         subjects affected / exposed
    64 / 202 (31.68%)
    53 / 196 (27.04%)
         occurrences all number
    114
    70
    Influenza
         subjects affected / exposed
    10 / 202 (4.95%)
    17 / 196 (8.67%)
         occurrences all number
    10
    17
    Nasopharyngitis
         subjects affected / exposed
    35 / 202 (17.33%)
    45 / 196 (22.96%)
         occurrences all number
    63
    74
    Viral upper respiratory tract infection
         subjects affected / exposed
    18 / 202 (8.91%)
    17 / 196 (8.67%)
         occurrences all number
    22
    19
    Upper respiratory tract infection
         subjects affected / exposed
    27 / 202 (13.37%)
    17 / 196 (8.67%)
         occurrences all number
    35
    24
    Sinusitis
         subjects affected / exposed
    10 / 202 (4.95%)
    10 / 196 (5.10%)
         occurrences all number
    13
    12
    Respiratory tract infection
         subjects affected / exposed
    12 / 202 (5.94%)
    8 / 196 (4.08%)
         occurrences all number
    21
    14

    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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