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    Clinical Trial Results:
    One year study to evaluate the long-term safety and tolerability of dupilumab in pediatric patients with asthma who participated in a previous dupilumab asthma clinical study

    Summary
    EudraCT number
    		 2017-003317-25
    Trial protocol
    		 Outside EU/EEA   LT   HU   PL   IT   ES  
    Global end of trial date
    01 Apr 2025

    Results information
    Results version number
    		 v1(current)
    This version publication date
    23 Oct 2025
    First version publication date
    23 Oct 2025
    Other versions

    Trial information

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    Trial identification
    Sponsor protocol code
    LTS14424
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT03560466
    WHO universal trial number (UTN)
    		 U1111-1200-1757
    Sponsors
    Sponsor organisation name
    Sanofi aventis recherche & développement
    Sponsor organisation address
    1 avenue Pierre Brossolette, Chilly-Mazarin, France, 91380
    Public contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Scientific contact
    Trial Transparency Team, Sanofi aventis recherche & développement, Contact-US@sanofi.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001501-PIP02-13
    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
    29 Apr 2025
    Is this the analysis of the primary completion data?
    No
    Global end of trial reached?
    Yes
    Global end of trial date
    01 Apr 2025
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    Main study: To evaluate the long-term safety and tolerability of dupilumab in pediatric participants with asthma who participated in a previous dupilumab asthma clinical study EFC14153 (2016-001607-23). Japan sub-study: To evaluate the efficacy of dupilumab in children 6 to <12 years of age with uncontrolled persistent asthma in the Japan sub-study.
    Protection of trial subjects
    The study was conducted by investigators experienced in the treatment of pediatric participants. The parent(s) or guardian(s) as well as the children were fully informed of all pertinent aspects of the clinical trial as well as the possibility to discontinue at any time. In addition to the consent form for the parent(s)/guardian(s), an assent form in child-appropriate language was provided and explained to the child. Repeated invasive procedures were minimized. The number of blood samples as well as the amount of blood drawn were adjusted according to age and weight. A topical anesthesia may have been used to minimize distress and discomfort.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    21 Jun 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
    Argentina: 54
    Country: Number of subjects enrolled
    Chile: 29
    Country: Number of subjects enrolled
    Colombia: 7
    Country: Number of subjects enrolled
    United States: 44
    Country: Number of subjects enrolled
    Hungary: 25
    Country: Number of subjects enrolled
    Italy: 6
    Country: Number of subjects enrolled
    Poland: 23
    Country: Number of subjects enrolled
    Spain: 1
    Country: Number of subjects enrolled
    Türkiye: 7
    Country: Number of subjects enrolled
    Lithuania: 17
    Country: Number of subjects enrolled
    Ukraine: 51
    Country: Number of subjects enrolled
    Australia: 1
    Country: Number of subjects enrolled
    Brazil: 19
    Country: Number of subjects enrolled
    Canada: 4
    Country: Number of subjects enrolled
    Japan: 13
    Country: Number of subjects enrolled
    Mexico: 55
    Country: Number of subjects enrolled
    Russian Federation: 10
    Country: Number of subjects enrolled
    South Africa: 12
    Worldwide total number of subjects
    378
    EEA total number of subjects
    72
    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)
    378
    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
    		 The main study was conducted at 70 centers in 17 countries, and the sub-study was conducted at 11 centers in Japan. 365 participants who rolled over from parent study EFC14153 were enrolled in the main study and 13 participants (not included in parent study EFC14153) were enrolled in Japan sub-study.

    Pre-assignment
    Screening details
    		 All participants received dupilumab dose based on body weight in the main study and Japan sub-study. As pre-specified in the protocol and statistical analysis plan (SAP), the results are presented by study and treatment group, regardless of the dose regimen.

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

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Main Study: Placebo-Dupilumab
    Arm description
    		 Participants who received placebo matched to dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 milligrams (mg) once every 2 weeks (q2w) as a subcutaneous (SC) injection or 300 mg once every 4 weeks (q4w) as an SC injection for participants with body weight ≤30 kilograms (kg). - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose inhaled corticosteroids (ICS) with a second controller medication (i.e., long-acting beta-2 agonists [LABA], long-acting muscarinic antagonists [LAMA], leukotriene receptor antagonists [LTRA] or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    Dupixent, REGN668
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered as an SC injection at a dose 100 mg q2w or 300mg q4w for participants with body weight ≤30 kg and 200 mg q2w for participants with body weight >30 kg for 52 weeks.

    Arm title
    		 Main Study: Dupilumab-Dupilumab
    Arm description
    		 Participants who received dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    Dupixent, REGN668
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered as an SC injection at a dose 100 mg q2w or 300mg q4w for participants with body weight ≤30 kg and 200 mg q2w for participants with body weight >30 kg for 52 weeks.

    Arm title
    		 Japan Sub-study: Dupilumab
    Arm description
    		 Participants received dupilumab for 52 weeks in the Japan sub-study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≥15 kg and ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Dupilumab
    Investigational medicinal product code
    SAR231893
    Other name
    Dupixent, REGN668
    Pharmaceutical forms
    Solution for injection in pre-filled syringe
    Routes of administration
    Subcutaneous use
    Dosage and administration details
    Dupilumab was administered as an SC injection at a dose 100 mg q2w or 300 mg q4w for participants with body weight ≥15 kg and ≤30 kg and 200 mg q2w for participants with body weight >30 kg for 52 weeks.

    Number of subjects in period 1
    		Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Started
    125
    240
    13
    Completed
    118
    228
    12
    Not completed
    7
    12
    1
         Adverse event, non-fatal
    1
    2
    -
         Unspecified
    6
    8
    1
         Poor compliance to protocol
    -
    2
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    Main Study: Placebo-Dupilumab
    Reporting group description
    		 Participants who received placebo matched to dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 milligrams (mg) once every 2 weeks (q2w) as a subcutaneous (SC) injection or 300 mg once every 4 weeks (q4w) as an SC injection for participants with body weight ≤30 kilograms (kg). - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose inhaled corticosteroids (ICS) with a second controller medication (i.e., long-acting beta-2 agonists [LABA], long-acting muscarinic antagonists [LAMA], leukotriene receptor antagonists [LTRA] or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Main Study: Dupilumab-Dupilumab
    Reporting group description
    		 Participants who received dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Japan Sub-study: Dupilumab
    Reporting group description
    		 Participants received dupilumab for 52 weeks in the Japan sub-study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≥15 kg and ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab Total
    Number of subjects
    		 125 240 13 378
    Age Categorical
    Units: Participants
    Age Continuous
    Units: years
        arithmetic mean (standard deviation)
    		 9.9 ( 1.6 ) 9.9 ( 1.7 ) 9.69 ( 1.11 ) -
    Gender Categorical
    Units: Participants
        Female
    		 46 85 4 135
        Male
    		 79 155 9 243
    Race
    Units: Subjects
        Caucasian/White
    		 111 215 0 326
        Black/of African Descent
    		 6 9 0 15
        Asian
    		 0 1 13 14
        American Indian or Alaska Native
    		 0 1 0 1
        Other
    		 8 14 0 22

    End points

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    End points reporting groups
    Reporting group title
    Main Study: Placebo-Dupilumab
    Reporting group description
    		 Participants who received placebo matched to dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 milligrams (mg) once every 2 weeks (q2w) as a subcutaneous (SC) injection or 300 mg once every 4 weeks (q4w) as an SC injection for participants with body weight ≤30 kilograms (kg). - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose inhaled corticosteroids (ICS) with a second controller medication (i.e., long-acting beta-2 agonists [LABA], long-acting muscarinic antagonists [LAMA], leukotriene receptor antagonists [LTRA] or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Main Study: Dupilumab-Dupilumab
    Reporting group description
    		 Participants who received dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Japan Sub-study: Dupilumab
    Reporting group description
    		 Participants received dupilumab for 52 weeks in the Japan sub-study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≥15 kg and ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Subject analysis set title
    Main Study: Dupilumab 100 mg q2w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the main study who received dupilumab 100 mg q2w were included in this arm.

    Subject analysis set title
    Main Study: Dupilumab 200 mg q2w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the main study who received dupilumab 200 mg q2w were included in this arm.

    Subject analysis set title
    Main Study: Dupilumab 300 mg q4w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the main study who received dupilumab 300 mg q4w were included in this arm.

    Subject analysis set title
    Japan Sub-study: Dupilumab 100 mg q2w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the Japan sub-study who received dupilumab 100 mg q2w were included in this arm.

    Subject analysis set title
    Japan Sub-study: Dupilumab 200 mg q2w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the Japan sub-study who received dupilumab 200 mg q2w were included in this arm.

    Subject analysis set title
    Japan Sub-study: Dupilumab 300 mg q4w
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    All participants from the Japan sub-study who received dupilumab 300 mg q4w were included in this arm.

    Primary: Main Study: Number of Participants With Any Treatment-Emergent Adverse Events (TEAEs)

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    End point title
    		 Main Study: Number of Participants With Any Treatment-Emergent Adverse Events (TEAEs) [1] [2]
    End point description
    An adverse event (AE) was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. TEAEs were AEs that developed or worsened or became serious during the TEAE period. Safety population included all participants exposed to at least 1 dose or part of a dose of the study treatment during current study regardless of the amount of treatment administered.
    End point type
    Primary
    End point timeframe
    From first dose of study treatment (Day 1) up to 112 days post last dose of study treatment, approximately 64 weeks
    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: As the endpoint is descriptive in nature, no statistical analysis is provided.
    [2] - 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: Only participants in the Main study arms were analyzed in this endpoint.
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab
    Number of subjects analysed
    125
    240
    Units: participants
    85
    147
    No statistical analyses for this end point

    Primary: Japan Sub-study: Change From Baseline to Week 12 in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)

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    End point title
    		 Japan Sub-study: Change From Baseline to Week 12 in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [3] [4]
    End point description
    FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline was defined as the last available measurement prior to the first study treatment dose if the participant was treated, or the last available value up to enrollment if the participant was not exposed to study treatment in the current study. Intent-to-treat (ITT) population included all participants in the enrolled population in the current study.
    End point type
    Primary
    End point timeframe
    Baseline (Day 1) to Week 12
    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: As the endpoint is descriptive in nature, no statistical analysis is provided.
    [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: Only participants in the Japan sub-study arms were analyzed in this endpoint.
    End point values
    		 Japan Sub-study: Dupilumab
    Number of subjects analysed
    13
    Units: percent predicted FEV1
        arithmetic mean (standard deviation)
    7.08 ( 6.78 )
    No statistical analyses for this end point

    Secondary: Japan Sub-study: Number of Participants With Any Treatment-Emergent Adverse Events

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    End point title
    		 Japan Sub-study: Number of Participants With Any Treatment-Emergent Adverse Events [5]
    End point description
    An AE was any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and which did not necessarily have to have a causal relationship with this treatment. TEAEs were AEs that developed or worsened or became serious during the TEAE period. Safety population included all enrolled participants who took at least 1 dose of study treatment in the current study, regardless of the amount of treatment administered.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) up to 112 days post last dose of study treatment, approximately 64 weeks
    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: Only participants in the Japan sub-study arms were analyzed in this endpoint.
    End point values
    		 Japan Sub-study: Dupilumab
    Number of subjects analysed
    13
    Units: participants
    13
    No statistical analyses for this end point

    Secondary: Annualized Rate of Severe Asthma Exacerbation Events During the 52-Week Treatment Period

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    End point title
    		 Annualized Rate of Severe Asthma Exacerbation Events During the 52-Week Treatment Period
    End point description
    A severe exacerbation event during study was defined as a deterioration of asthma requiring use of systemic corticosteroid (SCS) for ≥3 days or hospitalization/emergency room visit because of asthma, requiring SCS. Annualized severe exacerbation event rate was defined as total number of events that occurred during 52-week treatment period divided by total number of participant-years followed in 52-week treatment period. Main study: safety population included all participants exposed to at least 1 dose or part of a dose of study treatment during current study regardless of amount of treatment administered. Sub-study: ITT population included all participants in enrolled population in the current study.
    End point type
    Secondary
    End point timeframe
    Up to Week 52
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    125
    240
    13
    Units: exacerbations per participant-years
        number (not applicable)
    0.114
    0.120
    0.462
    No statistical analyses for this end point

    Secondary: Change From Baseline in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second Over Time

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    End point title
    		 Change From Baseline in Pre-Bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second Over Time
    End point description
    FEV1 was volume of air (in liters) exhaled from the lungs in first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study. Main study: safety population included all participants exposed to at least 1 dose or part of a dose of study treatment during current study regardless of amount of treatment administered. Sub-study: ITT population included all participants in enrolled population in current study. Only participants with data collected are reported. n=number of participants with data collected at specified timepoints. 99999=no data as there were no participants analyzed for that timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    122
    231
    13
    Units: percent predicted FEV1
    arithmetic mean (standard deviation)
        Week 2 (n=122, 231, 12)
    8.06 ( 15.71 )
    11.03 ( 18.74 )
    7.50 ( 9.29 )
        Week 4 (n=0, 0, 13)
    99999 ( 99999 )
    99999 ( 99999 )
    7.00 ( 7.12 )
        Week 8 (n=120, 222, 13)
    7.93 ( 14.88 )
    12.47 ( 18.60 )
    6.31 ( 8.97 )
        Week 12 (n=118, 226, 13)
    8.31 ( 14.70 )
    10.95 ( 17.33 )
    7.08 ( 6.78 )
        Week 24 (n=118, 219, 13)
    8.37 ( 15.48 )
    11.23 ( 18.24 )
    4.08 ( 8.75 )
        Week 52 (n=116, 212, 11)
    8.81 ( 16.18 )
    12.19 ( 17.88 )
    6.73 ( 8.09 )
        Week 64 (n=112, 212, 12)
    7.98 ( 17.14 )
    10.67 ( 17.19 )
    1.17 ( 8.03 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Absolute Forced Expiratory Volume in 1 Second Over Time

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    End point title
    		 Change From Baseline in Absolute Forced Expiratory Volume in 1 Second Over Time
    End point description
    FEV1 was volume of air (in liters) exhaled from the lungs in first second of a forced expiration as measured by spirometer. Spirometry was performed after a wash out period of bronchodilators according to their action duration. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study. Main study: safety population included all participants exposed to at least 1 dose or part of a dose of study treatment during current study regardless of amount of treatment administered. Sub-study: ITT population included all participants in enrolled population in current study. Only participants with data collected are reported. n=number of participants with data collected at specified timepoints. 99999=no data as there were no participants analyzed for that timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    122
    231
    13
    Units: liter
    arithmetic mean (standard deviation)
        Week 2 (n=122, 231, 12)
    0.36 ( 0.32 )
    0.42 ( 0.38 )
    0.13 ( 0.15 )
        Week 4 (n=0, 0, 13)
    99999 ( 99999 )
    99999 ( 99999 )
    0.13 ( 0.13 )
        Week 8 (n=120, 222, 13)
    0.38 ( 0.32 )
    0.47 ( 0.39 )
    0.12 ( 0.15 )
        Week 12 (n=118, 226, 13)
    0.41 ( 0.31 )
    0.45 ( 0.36 )
    0.17 ( 0.13 )
        Week 24 (n=118, 219, 13)
    0.46 ( 0.35 )
    0.51 ( 0.39 )
    0.15 ( 0.15 )
        Week 52 (n=116, 212, 11)
    0.62 ( 0.41 )
    0.66 ( 0.41 )
    0.34 ( 0.21 )
        Week 64 (n=112, 212, 12)
    0.66 ( 0.45 )
    0.70 ( 0.44 )
    0.25 ( 0.14 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Vital Capacity (FVC) Over Time

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    End point title
    		 Change From Baseline in Forced Vital Capacity (FVC) Over Time
    End point description
    FVC was defined as volume of air (in liters) that could be forcibly blown out after full inspiration in upright position as measured by spirometer. Spirometry was performed after wash out period of bronchodilators according to their action duration. Baseline(main study):original baseline of parent study EFC14153. Baseline(sub-study): last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study. Main study: safety population=all participants exposed to at least 1 dose or part of a dose of study treatment during current study regardless of amount of treatment administered. Sub-study: ITT population=all participants in enrolled population in current study. Only participants with data collected are reported. n=number of participants with data collected at specified timepoints. 99999=no data as there were no participants analyzed for that timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    122
    231
    13
    Units: liter
    arithmetic mean (standard deviation)
        Week 2 (n=122, 231, 12)
    0.35 ( 0.29 )
    0.38 ( 0.38 )
    0.11 ( 0.15 )
        Week 4 (n=0, 0, 13)
    99999 ( 99999 )
    99999 ( 99999 )
    0.09 ( 0.12 )
        Week 8 (n=120, 222, 13)
    0.37 ( 0.30 )
    0.45 ( 0.42 )
    0.11 ( 0.14 )
        Week 12 (n=118, 226, 13)
    0.40 ( 0.30 )
    0.43 ( 0.39 )
    0.12 ( 0.11 )
        Week 24 (n=118, 219, 13)
    0.47 ( 0.36 )
    0.50 ( 0.40 )
    0.17 ( 0.15 )
        Week 52 (n=116, 212, 11)
    0.68 ( 0.42 )
    0.70 ( 0.45 )
    0.36 ( 0.22 )
        Week 64 (n=112, 212, 12)
    0.74 ( 0.49 )
    0.75 ( 0.48 )
    0.41 ( 0.24 )
    No statistical analyses for this end point

    Secondary: Change From Baseline in Forced Expiratory Flow [FEF] 25% to 75% Over Time

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    End point title
    		 Change From Baseline in Forced Expiratory Flow [FEF] 25% to 75% Over Time
    End point description
    FEF:amount of air which can be forcibly exhaled from lungs in first second of forced exhalation.FEF 25-75%:mean FEF between 25% and 75% of FVC,FVC:volume of air that could be forcibly blown out after full inspiration in upright position. Spirometry was performed after wash out period of bronchodilators. Baseline(main study):original baseline of parent study. Baseline(sub-study): last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study. Main study:safety population=all participants exposed to at least 1 dose or part of dose of study treatment during current regardless of amount of treatment administered. Sub-study:ITT population=all participants in enrolled population in current study.n=number of participants with data collected at specified timepoints.99999=no data as there were no participants analyzed for that timepoint.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4 (Japan sub-study), 8, 12, 24, 52 and 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    122
    231
    13
    Units: liter per second
    arithmetic mean (standard deviation)
        Week 2 (n=122, 231, 12)
    0.49 ( 0.63 )
    0.58 ( 0.66 )
    0.21 ( 0.26 )
        Week 4 (n=0, 0, 13)
    99999 ( 99999 )
    99999 ( 99999 )
    0.25 ( 0.23 )
        Week 8 (n=120, 222, 13)
    0.48 ( 0.60 )
    0.62 ( 0.63 )
    0.15 ( 0.30 )
        Week 12 (n=118, 226, 13)
    0.54 ( 0.60 )
    0.59 ( 0.61 )
    0.29 ( 0.31 )
        Week 24 (n=118, 219, 13)
    0.58 ( 0.68 )
    0.66 ( 0.65 )
    0.15 ( 0.37 )
        Week 52 (n=116, 212, 11)
    0.73 ( 0.75 )
    0.77 ( 0.67 )
    0.40 ( 0.46 )
        Week 64 (n=112, 212, 12)
    0.77 ( 0.78 )
    0.79 ( 0.70 )
    0.11 ( 0.34 )
    No statistical analyses for this end point

    Secondary: Main Study: Change From Baseline in Percent Predicted Forced Vital Capacity Over Time

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    End point title
    		 Main Study: Change From Baseline in Percent Predicted Forced Vital Capacity Over Time [6]
    End point description
    FVC was defined as volume of air (in liters) that could be forcibly blown out after full inspiration in upright position as measured by spirometer. Spirometry was performed after wash out period of bronchodilators according to their action duration. Baseline was defined as the original baseline of parent study EFC14153. Safety population included all participants exposed to at least 1 dose or part of a dose of study treatment during current study regardless of amount of treatment administered. Only participants with data collected are reported. n=number of participants with data collected at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 8, 12, 24, 52 and 64
    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: Only participants in the Main study arms were analyzed in this endpoint.
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab
    Number of subjects analysed
    122
    231
    Units: percent predicted FVC
    arithmetic mean (standard deviation)
        Week 2 (n=122, 231)
    2.98 ( 12.91 )
    5.02 ( 16.33 )
        Week 8 (n=120, 222)
    2.88 ( 12.17 )
    6.64 ( 17.42 )
        Week 12 (n=118, 226)
    3.23 ( 12.49 )
    5.03 ( 16.45 )
        Week 24 (n=118, 219)
    3.19 ( 13.35 )
    5.28 ( 16.20 )
        Week 52 (n=116, 212)
    3.74 ( 13.10 )
    6.43 ( 16.40 )
        Week 64 (n=112, 212)
    3.13 ( 15.14 )
    4.83 ( 16.09 )
    No statistical analyses for this end point

    Secondary: Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-Question Version (ACQ-7-IA) Over Time

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    End point title
    		 Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-Question Version (ACQ-7-IA) Over Time [7]
    End point description
    ACQ-7-IA had 7 questions with first 5 items of ACQ-7 addressing most common asthma symptoms:frequency in past week awoken by asthma during night,severity of asthma symptoms in morning,limitation of daily activities due to asthma,shortness of breath due to asthma, wheeze; 2 questions on short-acting bronchodilator use and predicted bronchodilator use of FEV1.Participants/parents/guardians recalled child's previous week asthma and responded to questions on 7-point scale: 0=no impairment, 6=maximum impairment. Global ACQ-7-IA score: mean of 7 questions; ranged 0(totally controlled) to 6(severely uncontrolled).Higher scores=lower asthma control. Baseline:last available measurement prior to first study treatment dose if participant was treated or last available value up to enrollment if participant was not exposed to study treatment in current study.ITT population=all participants in enrolled population in current study.n=number of participants with data collected at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4, 8, 12, 24, 36, 52 and 64
    Notes
    [7] - 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: Only participants in the Japan sub-study arms were analyzed in this endpoint.
    End point values
    		 Japan Sub-study: Dupilumab
    Number of subjects analysed
    13
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 2 (n=13)
    -0.65 ( 0.64 )
        Week 4 (n=13)
    -0.68 ( 0.75 )
        Week 8 (n=13)
    -0.84 ( 0.59 )
        Week 12 (n=13)
    -1.03 ( 0.66 )
        Week 24 (n=13)
    -0.96 ( 0.61 )
        Week 36 (n=13)
    -1.01 ( 0.47 )
        Week 52 (n=13)
    -1.21 ( 0.37 )
        Week 64 (n=12)
    -0.99 ( 0.52 )
    No statistical analyses for this end point

    Secondary: Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-Question Version (ACQ-5-IA) Over Time

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    End point title
    		 Japan Sub-study: Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-Question Version (ACQ-5-IA) Over Time [8]
    End point description
    The ACQ-5-IA had 5 questions addressing most common asthma symptoms: frequency in past week awoken by asthma during night, severity of asthma symptoms in morning, limitation of daily activities due to asthma, shortness of breath due to asthma and wheeze. Participants/parents/guardians recalled the child's previous week asthma and responded to symptom and bronchodilator use questions on a 7-point scale: 0=no impairment, 6=maximum impairment. Global ACQ-5-IA score was mean of 5 questions ranging from 0 (totally controlled) to 6 (severely uncontrolled). Higher score indicated lower asthma control. Baseline: last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study. ITT population included all participants in enrolled population in current study. n=number of participants with data collected at specified timepoints.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) and Weeks 2, 4, 8, 12, 24, 36, 52 and 64
    Notes
    [8] - 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: Only participants in the Japan sub-study arms were analyzed in this endpoint.
    End point values
    		 Japan Sub-study: Dupilumab
    Number of subjects analysed
    13
    Units: score on a scale
    arithmetic mean (standard deviation)
        Week 2 (n=13)
    -0.68 ( 0.87 )
        Week 4 (n=13)
    -0.75 ( 1.06 )
        Week 8 (n=13)
    -0.92 ( 0.82 )
        Week 12 (n=13)
    -1.20 ( 0.84 )
        Week 24 (n=13)
    -1.15 ( 0.68 )
        Week 36 (n=13)
    -1.25 ( 0.54 )
        Week 52 (n=13)
    -1.49 ( 0.54 )
        Week 64 (n=12)
    -1.28 ( 0.59 )
    No statistical analyses for this end point

    Secondary: Serum Concentrations of Dupilumab

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    End point title
    		 Serum Concentrations of Dupilumab
    End point description
    Blood samples were collected at the specified timepoints for determination of functional dupilumab concentration in serum. Pharmacokinetic population (main and sub-study) included all the participants in the safety population with at least 1 non-missing and evaluable pre-dose serum concentration value after the first dose of dupilumab in the current study. Only participants with data collected are reported. n=number of participants with data collected at specified timepoints. As pre-specified in the SAP, serum concentration is presented by dose regimen for main and sub-study.
    End point type
    Secondary
    End point timeframe
    Weeks 12, 24, 52 and 64
    End point values
    		 Main Study: Dupilumab 100 mg q2w Main Study: Dupilumab 200 mg q2w Main Study: Dupilumab 300 mg q4w Japan Sub-study: Dupilumab 100 mg q2w Japan Sub-study: Dupilumab 200 mg q2w Japan Sub-study: Dupilumab 300 mg q4w
    Number of subjects analysed
    52
    299
    16
    3
    7
    3
    Units: nanogram per milliliter
    arithmetic mean (standard deviation)
        Week 12 (n=52, 296, 6, 3, 7, 3)
    60740.38 ( 23885.77 )
    80450.15 ( 39865.24 )
    79600.00 ( 25509.84 )
    38600.00 ( 20850.18 )
    79128.57 ( 24513.31 )
    53066.67 ( 18569.96 )
        Week 24 (n=38, 293, 12, 3, 6, 3)
    64201.03 ( 22890.02 )
    81849.66 ( 42824.93 )
    80675.00 ( 26921.27 )
    41300.00 ( 22138.65 )
    98316.67 ( 18066.81 )
    55166.67 ( 14558.96 )
        Week 52 (n=27, 299, 16, 3, 7, 3)
    52808.85 ( 21526.05 )
    71910.74 ( 42290.54 )
    81289.94 ( 31957.07 )
    41000.00 ( 23157.94 )
    91128.57 ( 28739.33 )
    59300.00 ( 18107.46 )
        Week 64 (n=28, 298, 16, 3, 6, 3)
    1884.04 ( 9763.01 )
    3391.86 ( 16196.36 )
    184.69 ( 582.75 )
    39.00 ( 0.00 )
    39.00 ( 0.00 )
    39.00 ( 0.00 )
    No statistical analyses for this end point

    Secondary: Number of Participants With Anti-Drug Antibodies (ADAs) Against Dupilumab

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    End point title
    		 Number of Participants With Anti-Drug Antibodies (ADAs) Against Dupilumab
    End point description
    ADA positive was defined as either treatment-boosted or treatment-emergent response in the ADA assay. Treatment-boosted response was defined as a positive response in the ADA assay post first dose in the current study that was greater than or equal to 4-fold of the baseline titer levels, when baseline results were positive. Treatment-emergent response was defined as a positive response in the ADA assay post first dose in the current study, when baseline results were negative or missing. Baseline (main study): original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study. ADA population (main and sub-study) included all the participants in the safety population with at least 1 non-missing ADA result following the first dose of dupilumab in the current study.
    End point type
    Secondary
    End point timeframe
    From first dose of study treatment (Day 1) to Week 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    124
    239
    13
    Units: participants
        Treatment-boosted ADA Response
    1
    0
    0
        Treatment-emergent ADA Response
    10
    7
    2
    No statistical analyses for this end point

    Secondary: Main Study: Percent Change From Baseline in Blood Eosinophil Count at Week 64

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    End point title
    		 Main Study: Percent Change From Baseline in Blood Eosinophil Count at Week 64 [9]
    End point description
    Blood samples were collected at specified timepoints to assess percent change from baseline in eosinophil count. Baseline was defined as the original baseline of parent study EFC14153. Safety population included all participants exposed to at least 1 dose or part of a dose of the study treatment during current study regardless of the amount of treatment administered. Only participants with data collected are reported.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 64
    Notes
    [9] - 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: Only participants in the Main study arms were analyzed in this endpoint.
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab
    Number of subjects analysed
    103
    186
    Units: percent change
        arithmetic mean (standard deviation)
    34.528 ( 207.067 )
    29.769 ( 323.837 )
    No statistical analyses for this end point

    Secondary: Percent Change From Baseline in Total Immunoglobulin (Ig) E in Serum at Week 64

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    End point title
    		 Percent Change From Baseline in Total Immunoglobulin (Ig) E in Serum at Week 64
    End point description
    Blood samples were collected at specified timepoints to assess percent change from baseline in total IgE in serum. Baseline (main study): the original baseline of parent study EFC14153. Baseline (sub-study): last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in current study. Main study: safety population included all participants exposed to at least 1 dose or part of a dose of the study treatment during current study regardless of the amount of treatment administered. Sub-study: safety population included all enrolled participants who took at least 1 dose of study treatment in the current study, regardless of the amount of treatment administered. Only participants with data collected are reported.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 64
    End point values
    		 Main Study: Placebo-Dupilumab Main Study: Dupilumab-Dupilumab Japan Sub-study: Dupilumab
    Number of subjects analysed
    113
    219
    12
    Units: percent change
        median (inter-quartile range (Q1-Q3))
    -76.9 (-85.2 to -63.4)
    -86.8 (-91.4 to -77.3)
    -75.0 (-81.1 to -70.0)
    No statistical analyses for this end point

    Secondary: Japan Sub-study: Change From Baseline in Fractional Exhaled Nitric Oxide (FeNO) at Week 64

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    End point title
    		 Japan Sub-study: Change From Baseline in Fractional Exhaled Nitric Oxide (FeNO) at Week 64 [10]
    End point description
    FeNO was analyzed using a NIOX instrument or similar analyzer using a flow rate of 50 milliliter per second. FeNO assessment was conducted prior to spirometry and following a fast of ≥1 hour. Baseline was defined as the last available measurement prior to first study treatment dose if participant was treated, or last available value up to enrollment if participant was not exposed to study treatment in the current study. Safety population included all enrolled participants who took at least 1 dose of study treatment in the current study, regardless of the amount of treatment administered. Only participants with data collected are reported.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1) to Week 64
    Notes
    [10] - 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: Only participants in the Japan sub-study arms were analyzed in this endpoint.
    End point values
    		 Japan Sub-study: Dupilumab
    Number of subjects analysed
    12
    Units: parts per billion
        arithmetic mean (standard deviation)
    -8.3 ( 27.5 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    AEs: From first dose of study treatment (Day 1) up to 112 days post last dose of study treatment, approximately 64 weeks for main study and sub-study each. Deaths: From first dose of study treatment (Day 1) up to end of follow-up, approximately 353 weeks.
    Adverse event reporting additional description
    Analysis was performed on safety population. As pre-specified in the protocol and SAP, the results are presented by study and treatment group, regardless of the dose regimen.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    27.1
    Reporting groups
    Reporting group title
    Main Study: Placebo-Dupilumab
    Reporting group description
    		 Participants who received placebo matched to dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Japan Sub-study: Dupilumab
    Reporting group description
    		 Participants received dupilumab for 52 weeks in the Japan sub-study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≥15 kg and ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Reporting group title
    Main Study: Dupilumab-Dupilumab
    Reporting group description
    		 Participants who received dupilumab in the parent study EFC14153 received dupilumab for 52 weeks in the main study as follows: - 100 mg q2w as an SC injection or 300 mg q4w as an SC injection for participants with body weight ≤30 kg. - 200 mg q2w as an SC injection for participants with body weight >30 kg. Participants also received a stable-dose background therapy of medium-dose ICS with a second controller medication (i.e., LABA, LAMA, LTRA or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller medication. Albuterol/salbutamol or levalbuterol/levosalbutamol was given as reliever medication as instructed by Investigator.

    Serious adverse events
    		 Main Study: Placebo-Dupilumab Japan Sub-study: Dupilumab Main Study: Dupilumab-Dupilumab
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 125 (0.80%)
    4 / 13 (30.77%)
    6 / 240 (2.50%)
         number of deaths (all causes)
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    Injury, poisoning and procedural complications
    Radius Fracture
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Respiratory, thoracic and mediastinal disorders
    Asthma
         subjects affected / exposed
    0 / 125 (0.00%)
    2 / 13 (15.38%)
    0 / 240 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 5
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Atelectasis
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Infections and infestations
    Bronchitis
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Complicated Appendicitis
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Influenza
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 2
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pneumonia
         subjects affected / exposed
    1 / 125 (0.80%)
    0 / 13 (0.00%)
    0 / 240 (0.00%)
         occurrences causally related to treatment / all
    0 / 1
    0 / 0
    0 / 0
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Pulmonary Tuberculosis
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Upper Respiratory Tract Infection
         subjects affected / exposed
    0 / 125 (0.00%)
    0 / 13 (0.00%)
    1 / 240 (0.42%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 Main Study: Placebo-Dupilumab Japan Sub-study: Dupilumab Main Study: Dupilumab-Dupilumab
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    63 / 125 (50.40%)
    13 / 13 (100.00%)
    108 / 240 (45.00%)
    Injury, poisoning and procedural complications
    Accidental Overdose
         subjects affected / exposed
    2 / 125 (1.60%)
    1 / 13 (7.69%)
    5 / 240 (2.08%)
         occurrences all number
    2
    1
    5
    Contusion
         subjects affected / exposed
    2 / 125 (1.60%)
    1 / 13 (7.69%)
    2 / 240 (0.83%)
         occurrences all number
    2
    1
    2
    Eyelid Injury
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Scratch
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    1 / 240 (0.42%)
         occurrences all number
    0
    1
    2
    Wrist Fracture
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Ligament Sprain
         subjects affected / exposed
    2 / 125 (1.60%)
    1 / 13 (7.69%)
    3 / 240 (1.25%)
         occurrences all number
    2
    1
    3
    Nervous system disorders
    Tension Headache
         subjects affected / exposed
    0 / 125 (0.00%)
    2 / 13 (15.38%)
    0 / 240 (0.00%)
         occurrences all number
    0
    2
    0
    Headache
         subjects affected / exposed
    3 / 125 (2.40%)
    1 / 13 (7.69%)
    8 / 240 (3.33%)
         occurrences all number
    4
    1
    15
    Blood and lymphatic system disorders
    Eosinophilia
         subjects affected / exposed
    7 / 125 (5.60%)
    1 / 13 (7.69%)
    8 / 240 (3.33%)
         occurrences all number
    8
    1
    8
    General disorders and administration site conditions
    Injection Site Erythema
         subjects affected / exposed
    3 / 125 (2.40%)
    1 / 13 (7.69%)
    7 / 240 (2.92%)
         occurrences all number
    6
    4
    11
    Injection Site Induration
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    1 / 240 (0.42%)
         occurrences all number
    0
    1
    2
    Injection Site Reaction
         subjects affected / exposed
    9 / 125 (7.20%)
    0 / 13 (0.00%)
    8 / 240 (3.33%)
         occurrences all number
    73
    0
    46
    Pyrexia
         subjects affected / exposed
    3 / 125 (2.40%)
    6 / 13 (46.15%)
    5 / 240 (2.08%)
         occurrences all number
    3
    7
    5
    Gastrointestinal disorders
    Constipation
         subjects affected / exposed
    1 / 125 (0.80%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    1
    1
    0
    Dental Caries
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    1 / 240 (0.42%)
         occurrences all number
    0
    2
    1
    Vomiting
         subjects affected / exposed
    1 / 125 (0.80%)
    1 / 13 (7.69%)
    5 / 240 (2.08%)
         occurrences all number
    1
    1
    7
    Enterocolitis
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Diarrhoea
         subjects affected / exposed
    7 / 125 (5.60%)
    2 / 13 (15.38%)
    10 / 240 (4.17%)
         occurrences all number
    7
    3
    10
    Respiratory, thoracic and mediastinal disorders
    Rhinitis Allergic
         subjects affected / exposed
    9 / 125 (7.20%)
    1 / 13 (7.69%)
    7 / 240 (2.92%)
         occurrences all number
    9
    1
    10
    Skin and subcutaneous tissue disorders
    Acne
         subjects affected / exposed
    0 / 125 (0.00%)
    2 / 13 (15.38%)
    3 / 240 (1.25%)
         occurrences all number
    0
    2
    3
    Dermatitis Contact
         subjects affected / exposed
    2 / 125 (1.60%)
    1 / 13 (7.69%)
    3 / 240 (1.25%)
         occurrences all number
    2
    1
    3
    Miliaria
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    1 / 240 (0.42%)
         occurrences all number
    0
    1
    1
    Eczema
         subjects affected / exposed
    0 / 125 (0.00%)
    2 / 13 (15.38%)
    0 / 240 (0.00%)
         occurrences all number
    0
    2
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    1 / 240 (0.42%)
         occurrences all number
    0
    1
    1
    Musculoskeletal and connective tissue disorders
    Osteochondrosis
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Arthralgia
         subjects affected / exposed
    1 / 125 (0.80%)
    2 / 13 (15.38%)
    2 / 240 (0.83%)
         occurrences all number
    1
    2
    2
    Infections and infestations
    Hordeolum
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    2 / 240 (0.83%)
         occurrences all number
    0
    1
    2
    Herpes Zoster
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Covid-19
         subjects affected / exposed
    1 / 125 (0.80%)
    2 / 13 (15.38%)
    3 / 240 (1.25%)
         occurrences all number
    1
    2
    3
    Bronchitis
         subjects affected / exposed
    5 / 125 (4.00%)
    3 / 13 (23.08%)
    7 / 240 (2.92%)
         occurrences all number
    6
    4
    10
    Oral Herpes
         subjects affected / exposed
    1 / 125 (0.80%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    1
    2
    0
    Nasopharyngitis
         subjects affected / exposed
    12 / 125 (9.60%)
    5 / 13 (38.46%)
    21 / 240 (8.75%)
         occurrences all number
    17
    12
    25
    Lower Respiratory Tract Infection
         subjects affected / exposed
    0 / 125 (0.00%)
    1 / 13 (7.69%)
    0 / 240 (0.00%)
         occurrences all number
    0
    1
    0
    Pharyngitis
         subjects affected / exposed
    12 / 125 (9.60%)
    5 / 13 (38.46%)
    15 / 240 (6.25%)
         occurrences all number
    13
    7
    16
    Influenza
         subjects affected / exposed
    7 / 125 (5.60%)
    5 / 13 (38.46%)
    13 / 240 (5.42%)
         occurrences all number
    9
    8
    16
    Sinusitis
         subjects affected / exposed
    3 / 125 (2.40%)
    1 / 13 (7.69%)
    5 / 240 (2.08%)
         occurrences all number
    5
    1
    6
    Viral Upper Respiratory Tract Infection
         subjects affected / exposed
    6 / 125 (4.80%)
    1 / 13 (7.69%)
    9 / 240 (3.75%)
         occurrences all number
    7
    2
    12
    Upper Respiratory Tract Infection
         subjects affected / exposed
    5 / 125 (4.00%)
    2 / 13 (15.38%)
    19 / 240 (7.92%)
         occurrences all number
    7
    6
    28
    Tonsillitis
         subjects affected / exposed
    3 / 125 (2.40%)
    1 / 13 (7.69%)
    6 / 240 (2.50%)
         occurrences all number
    3
    1
    6

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    09 Jul 2018
    Changed study sample size due to the increase in the sample size of EFC14153 (parent study of the LTS14424), removed secondary safety endpoint, removed optional pharmacogenetic informed consent/assent form mention, changed pregnancy outcome follow-up period, added LAMA and ultra LABA (vilanterol) withhold period before spirometry, included the total volume of blood collected at a single visit, corrected the information that data for current study Visit (V) 1 coming from end of treatment (EOT) visit from parent study would not be reported in current study electronic case report form.
    12 Dec 2019
    Changed dupilumab dose for children with body weight ≤30 kg from 100 mg q2w to 300 mg q4w, removed immunoglobulins as other secondary endpoints, clarified when an asthma exacerbation was to be reported as AE, added relatedness to study treatment in the adverse event of special interest anaphylactic reaction and systemic allergic reaction, clarified that hepatitis serologies were not repeated for determining participants' eligibility in the current study, changed study sample size, updated analysis populations according to the dose regimen, updated retention of study documents in study sites from 15 to 25 years.
    15 Apr 2020
    Allowed later entrance in current study for EFC14153 participants who were not able to perform the EFC14153 EOT visit onsite to rollover at V28/EOT, or to receive last study treatment doses in parent study EFC14153 due to the coronavirus disease 2019 pandemic, removed the monthly 300 mg dose specific analysis set, allowed at home administration of 300 mg monthly doses from the second administration onwards (following first 300 mg administered onsite).

    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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