AsthmaPhase 3VOYAGENCT02948959
Evaluation of Dupilumab in Children With Uncontrolled Asthma
A Phase 3 study testing Dupilumab and Asthma Reliever Therapies in people with Asthma, run by Sanofi at 99 sites in 18 countries.
Trial summary
Primary Objective: To evaluate the efficacy of dupilumab in children 6 to less than (<) 12 years of age with uncontrolled persistent asthma. Secondary Objective: To evaluate in children 6 to <12 years of age with uncontrolled persistent asthma: The safety and tolerability of dupilumab. Read more about this study
- Who is it for?
- Ages 6 to 11 years
- Conditions the study is for
- Asthma
- What is being tested?
- Dupilumab and Asthma Reliever Therapies
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 408 participants
- Where
- 99 sitesin 18 countries
- Study dates
- 21 Apr 2017to 26 Aug 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 11 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Children 6 to <12 years of age, with a physician diagnosis of persistent asthma for greater than or equal to (>=)12 months prior to screening, based on clinical history and examination, pulmonary function parameters according to Global initiative for asthma (GINA) 2015 Guidelines and the following criteria:
- Existing background therapy of medium-dose ICS with second controller medication (i.e., long-acting β2 agonist , leukotriene receptor antagonist, long acting muscarinic antagonist, or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller, for at least 3 months with a stable dose >=1 month prior to Screening Visit 1.
- Pre-bronchodilator forced expiratory volume in 1 second (FEV1) <=95 percentage (%) of predicted normal or pre bronchodilator FEV1/forced vital capacity ratio <0.85 at Screening and Baseline Visits.
- Reversibility of at least 10% in FEV1 after the administration of 200 to 400 micrograms (mcg; 2 to 4 puff inhalations with metered-dose inhaler [MDI]) of albuterol/salbutamol or 45 to 90 mcg (2 to 4 puffs with MDI) of levalbuterol/levosalbutamol reliever medication before randomization (up to 3 opportunities during the same visit were allowed with a maximum of 12 puffs of reliever medication if tolerated by the participant).
- Must had experienced, within 1 year prior to Screening Visit 1, any of the following events:
- Treatment with a systemic corticosteroid (oral or parenteral), as prescribed by a healthcare professional for worsening asthma at least once or,
Show all 12Show fewer
- Hospitalization or emergency visit for worsening asthma.
- Evidence of uncontrolled asthma, with at least one of the following criteria during the 4 (±1) weeks Screening Period:
- Asthma Control Questionnaire-Interviewer Administered (ACQ-IA) ACQ-5 score >=1.5 on at least one day of the Screening Period.
- Use of reliever medication (i.e., albuterol/salbutamol or levalbuterol/levosalbutamol), other than as a preventive for exercise induced bronchospasm, on 3 or more days per week, in at least one week during the Screening Period.
- Sleep awakening due to asthma symptoms requiring use of reliever medication at least once during the Screening Period.
- Asthma symptoms 3 or more days per week in at least one week during the Screening Period.
Who cannot join
- Participants <6 or >=12 years of age.
- Participants with <16 kg bodyweight.
- Any other chronic lung disease (cystic fibrosis, bronchopulmonary dysplasia, etc.), which may impair lung function.
- A participant with any history of life threatening asthma (ie, extreme exacerbation that requires intubation).
- Co-morbid disease that might interfere with the evaluation of investigational medicinal product.
- The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Trial locations and contacts
99 sites in 18 countries.
99 sites in 18 countries
United States18 sites
Investigational Site Number 840016
Ann Arbor, Michigan, 48106
Investigational Site Number 840031
Birmingham, Alabama, 35209
Investigational Site Number 840004
Charlotte, North Carolina, 28277
Investigational Site Number 840008
Cincinnati, Ohio, 45229
Investigational Site Number 840021
Durham, North Carolina, 27710
Investigational Site Number 840024
Edmond, Oklahoma, 73034
Investigational Site Number 840002
Gilbert, Arizona, 85234
Investigational Site Number 840023
Great Neck, New York, 11021
Investigational Site Number 840018
La Crosse, Wisconsin, 54601
Investigational Site Number 840022
Lincoln, Nebraska, 68505
Investigational Site Number 840035
New York, New York, 00000
Investigational Site Number 840013
New York, New York, 10032
Investigational Site Number 840036
Owensboro, Kentucky, 42301
Investigational Site Number 840007
Rochester, New York, 14607
Investigational Site Number 840001
Rolling Hills Estates, California, 90274
Investigational Site Number 840003
San Antonio, Texas, 78229
Investigational Site Number 840006
St Louis, Missouri, 63110-1077
Investigational Site Number 840012
Tucson, Arizona, 85724-5030
Hungary8 sites
Investigational Site Number 348006
Budapest, 1089
Investigational Site Number 348002
Gyula, 5700
Investigational Site Number 348012
Mezőkövesd, 3400
Investigational Site Number 348005
Szeged, 6720
Investigational Site Number 348001
Székesfehérvár, 8000
Investigational Site Number 348008
Szigetvár, 7900
Investigational Site Number 348003
Töröbálint, 2045
Investigational Site Number 348007
Zalaegerszeg, 8900
Ukraine8 sites
Investigational Site Number 804007
Chernivtsi, 58023
Investigational Site Number 804004
Dnipro, 49101
Investigational Site Number 804011
Ivano-Frankivsk, 76014
Investigational Site Number 804005
Kharkiv, 61093
Investigational Site Number 804008
Kryvyi Rig, 50082
Investigational Site Number 804001
Kyiv, 03115
Investigational Site Number 804002
Zaporizhzhya, 69063
Investigational Site Number 804003
Zaporizhzhya, 69076
Brazil7 sites
Investigational Site Number 076008
Blumenau, 89030-100
Investigational Site Number 076001
Porto Alegre, 90020-090
Investigational Site Number 076007
Porto Alegre, 90610-000
Investigational Site Number 076003
São Paulo, 02189-010
Investigational Site Number 076002
São Paulo, 04037-002
Investigational Site Number 076004
São Paulo, 05403-000
Investigational Site Number 076006
Sorocaba, 18040-425
Chile6 sites
Investigational Site Number 152003
Santiago, 7560994
Investigational Site Number 152005
Santiago, 838-0418
Investigational Site Number 152009
Santiago, 8380453
Investigational Site Number 152001
Valdivia
Investigational Site Number 152007
Viña del Mar, 2520024
Investigational Site Number 152002
Viña del Mar, 2520594
Russia6 sites
Investigational Site Number 643006
Moscow, 119333
Investigational Site Number 643004
Perm, 614066
Investigational Site Number 643005
Saint Petersburg, 191144
Investigational Site Number 643002
Saint Petersburg, 193312
Investigational Site Number 643001
Saint Petersburg, 194100
Investigational Site Number 643003
Saint Petersburg, 196240
Turkey (Türkiye)6 sites
Investigational Site Number 792005
Adana
Investigational Site Number 792008
Ankara, 06500
Investigational Site Number 792001
Ankara
Investigational Site Number 792006
Bursa
Investigational Site Number 792003
Istanbul
Investigational Site Number 792004
Istanbul
Argentina5 sites
Investigational Site Number 032004
Buenos Aires, B1602DQD
Investigational Site Number 032003
Buenos Aires, C1121ABE
Investigational Site Number 032002
Caba, C1122AAK
Investigational Site Number 032001
Caba, C1414AIF
Investigational Site Number 032006
Mendoza, 5500
Italy5 sites
Investigational Site Number 380007
Catania, 95123
Investigational Site Number 380003
Florence, 50139
Investigational Site Number 380004
Padova, 35128
Investigational Site Number 380005
Roma, 00146
Investigational Site Number 380001
Verona, 37126
Lithuania5 sites
Investigational Site Number 440002
Kaunas, LT-50161
Investigational Site Number 440005
Šiauliai, LT-76231
Investigational Site Number 440003
Utena, LT-28151
Investigational Site Number 440001
Vilnius, LT-08406
Investigational Site Number 440004
Vilnius, LT-09108
Mexico5 sites
Investigational Site Number 484006
Chihuahua City, 31000
Investigational Site Number 484004
Chihuahua City, 31200
Investigational Site Number 484003
Durango, 34080
Investigational Site Number 484001
Monterrey, 64460
Investigational Site Number 484002
Veracruz, 91910
Spain5 sites
Investigational Site Number 724001
Barcelona, 08035
Investigational Site Number 724006
Esplugues de Llobregat, 08950
Investigational Site Number 724005
Pozuelo de Alarcón, 28223
Investigational Site Number 724002
Santiago de Compostela, 15706
Investigational Site Number 724003
Valencia, 46017
Australia4 sites
Investigational Site Number 036001
Campbelltown, 2560
Investigational Site Number 036005
North Adelaide, 5006
Investigational Site Number 036003
Parkville/Melbourne, 3052
Investigational Site Number 036002
South Brisbane, 4101
Canada4 sites
Investigational Site Number 124004
Edmonton, T6G 2B7
Investigational Site Number 124002
Hamilton, L8S1G5
Investigational Site Number 124001
Montreal, H3T 1C5
Investigational Site Number 124003
Québec, G1V 4W2
Colombia2 sites
Investigational Site Number 170004
Antioquia, 050010
Investigational Site Number 170002
Cali, 760043
Poland2 sites
Investigational Site Number 616001
Lodz, 90-329
Investigational Site Number 616002
Poznan, 60-693
South Africa2 sites
Investigational Site Number 710004
Cape Town, 7500
Investigational Site Number 710001
Cape Town, 7700
About this study
Official title: A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Dupilumab in Children 6 to <12 Years of Age With Uncontrolled Persistent Asthma
Primary Objective:
To evaluate the efficacy of dupilumab in children 6 to less than (<) 12 years of age with uncontrolled persistent asthma.
Secondary Objective:
To evaluate in children 6 to <12 years of age with uncontrolled persistent asthma:
- The safety and tolerability of dupilumab.
- The evaluate the effect of dupilumab in improving participant reported outcomes including health related quality of life.
- The dupilumab systemic exposure and incidence of anti-drug antibodies.
- The evaluate the association between dupilumab treatment and pediatric immune responses to vaccines: any vaccination for tetanus, diphtheria, pertussis and/or seasonal trivalent/quadrivalent influenza vaccine.
Read the detailed description
The total study duration per participant was up to 69 weeks, consisted of a screening period of 3-5 weeks, a randomized treatment period of 52 weeks and a post-treatment period of 12 weeks.
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DupilumabDrug
Also called SAR231893 (REGN668)
Pharmaceutical form: Solution
Route of administration: Subcutaneous
PlaceboDrug
Pharmaceutical form: Solution
Route of administration: Subcutaneous
Asthma Controller TherapiesDrug
Pharmaceutical form: Aerosol, capsules, tablets, oral solution
Route of administration: Inhaled, oral
Asthma Reliever TherapiesDrug
Pharmaceutical form: Nebulized, aerosol
Route of administration: Inhaled
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo (for Dupilumab), subcutaneous (SC) injection every 2 weeks (q2w) for 52 weeks in combination with stable-dose background therapy of medium-dose inhaled corticosteroids (ICS) with a second controller medication (i.e., long-acting β2 agonist [LABA], long acting muscarinic antagonist [LAMA], leukotriene receptor antagonist [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. Participants were followed up for 12 weeks after last dose (i.e. up to Week 64). Receives: Drug: Placebo; Drug: Asthma Controller Therapies; Drug: Asthma Reliever Therapies |
| Dupilumab | Experimental | Dupilumab 200 milligrams (mg) (in 1.14 milliliters [mL] for >30 kilograms [kg] bodyweight [BW]) or 100 mg (in 0.67 mL for less than or equal to (<=) 30 kg BW), SC injection q2w for 52 weeks in combination with 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. Participants were followed up for 12 weeks after last dose (i.e. up to Week 64). Receives: Drug: Dupilumab; Drug: Asthma Controller Therapies; Drug: Asthma Reliever Therapies |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized Rate of Severe Exacerbation Events During the 52-Week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization or emergency room visit because of asthma requiring systemic corticosteroid treatment. Annualized event rate was defined as the total number of severe exacerbation events that occurred during the 52-week treatment period divided by the total number of participant-years followed in the 52-week treatment period.
Measured: Baseline to Week 52
Annualized Rate of Severe Exacerbation Events During the 52-Week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization or emergency room visit because of asthma requiring systemic corticosteroid treatment. Annualized event rate was defined as the total number of severe exacerbation events that occurred during the 52-week treatment period divided by the total number of participant-years followed in the 52-week treatment period.
Measured: Baseline to Week 52
Secondary outcomes (48)
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 (FEV1) Second at Week 12: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator % predicted FEV1 value up to Week 12 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 12
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 12: Type 2 Inflammatory Asthma Phenotype Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. Least square (LS) means and standard error (SE) were derived from mixed-effect model with repeated measures (MMRM) model with change from baseline in pre-bronchodilator % predicted FEV1 value up to Week 12 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 12
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version (ACQ-7-IA) at Week 24: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
ACQ-7-IA had 7 questions, which assessed: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities, shortness of breath due to asthma and wheeze, reliever medication use, and FEV1 (% predicted). Participants recalled their previous week asthma and answered 5 symptom questions on 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). Total score: mean of scores of all 7 questions; ranging from 0 (totally controlled) to 6 (severely uncontrolled), higher score indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-7-IA values up to Week 52 as response variable and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-7-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 24
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Week 24: Type 2 Inflammatory Asthma Phenotype Population
ACQ-7-IA had 7 questions, which assessed: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities due to asthma, shortness of breath due to asthma and wheeze, reliever medication use, and FEV1 (% predicted). Participants recalled their previous week asthma and answered 5 symptom questions on 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). Total score: mean of scores of all 7 questions; ranging from 0 (totally controlled) to 6 (severely uncontrolled), higher score indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-7-IA values up to Week 52 as response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-7-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 24
Change From Baseline in Fractional Exhaled Nitric Oxide Level at Week 12: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FeNO is a surrogate marker for airway inflammation. FeNO was analyzed using a NIOX instrument or similar analyzer using a flow rate of 50 mL/second, and reported in ppb. LS means and SE were derived from MMRM model with change from baseline in FeNO up to Week 12 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline ICS level, visit, treatment by-visit interaction, baseline FeNO value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 12
Change From Baseline in Fractional Exhaled Nitric Oxide Level at Week 12: Type 2 Inflammatory Asthma Phenotype Population
FeNO is a surrogate marker for airway inflammation. FeNO was analyzed using a NIOX instrument or similar analyzer using a flow rate of 50 mL/second, and reported in ppb. LS means and SE were derived from MMRM model with change from baseline in FeNO up to Week 12 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline ICS level, visit, treatment by-visit interaction, baseline FeNO value and baseline-by-visit interaction as covariates.
Measured: Baseline, Week 12
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second at Weeks 2, 4, 8, 24, 36 and 52: Type 2 Inflammatory Asthma Phenotype Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator % predicted FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 24, 36, 52
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second at Weeks 2, 4, 8, 24, 36 and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator % predicted FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 24, 36, 52
Time to First Severe Exacerbation Event: Kaplan-Meier Estimates During 52-week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
The time to first severe exacerbation was defined as date of the first severe exacerbation event - randomization date +1. A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization related to asthma symptoms or emergency room visit because of asthma requiring systemic corticosteroid treatment. Kaplan-Meier method was used for analysis.
Measured: Baseline up to Week 52
Time to First Severe Exacerbation Event: Kaplan-Meier Estimates During 52-week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The time to first severe exacerbation was defined as date of the first severe exacerbation event - randomization date +1. A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization related to asthma symptoms or emergency room visit because of asthma requiring systemic corticosteroid treatment. Kaplan-Meier method was used for analysis.
Measured: Baseline up to Week 52
Time to First Loss of Asthma Control (LOAC) Event: Kaplan-Meier Estimates During 52-week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
Time to first LOAC event was date of first LOAC event - first dose date +1. A LOAC event was defined as deterioration of asthma during 52-week treatment period that resulted in any of the following: >= 6 additional reliever puffs of salbutamol/albuterol or levosalbutamol/levalbuterol in 24-hour period (compared to baseline) on 2 consecutive days; increase in ICS dose >=4 times than dose at Visit 2 (Week 0); a decrease in ante meridiem (AM)/post meridiem (PM) peak flow of 30% or more on 2 consecutive days of treatment, based on defined stability limit (defined as respective mean AM/PM peak expiratory flow obtained over last 7 days prior to randomization (Day 1); severe exacerbation event. Kaplan-Meier method was used for analysis.
Measured: Baseline up to Week 52
Time to First Loss of Asthma Control Event: Kaplan-Meier Estimates During 52-week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time to first LOAC event was date of first LOAC event - first dose date +1. A LOAC event was defined as deterioration of asthma during 52-week treatment period that resulted in any of the following: >= 6 additional reliever puffs of salbutamol/albuterol or levosalbutamol/levalbuterol in 24-hour period (compared to baseline) on 2 consecutive days; increase in ICS dose >=4 times than dose at Visit 2 (Week 0); a decrease in AM/PM peak flow of 30% or more on 2 consecutive days of treatment, based on defined stability limit (defined as respective mean AM/PM peak expiratory flow obtained over last 7 days prior to randomization (Day 1); severe exacerbation event. Kaplan-Meier method was used for analysis.
Measured: Baseline up to Week 52
Absolute Change From Baseline in Pre-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator FEV1 values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline pre-bronchodilator FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Absolute Change From Baseline in Pre-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator FEV1 values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline pre-bronchodilator FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Percent Change From Baseline in Pre-Bronchodilator Percent Predicted FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator % predicted FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Percent Change From Baseline in Pre-Bronchodilator Percent Predicted FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FEV1 was the volume of air (in liters) exhaled from the lungs in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in pre-bronchodilator % predicted FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline % predicted FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning (AM) Peak Expiratory Flow (PEF) at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter. Peak flow testing for AM PEF was performed in morning prior to taking any salbutamol/albuterol or levosalbutamol/levalbuterol reliever medication. Baseline AM PEF was the mean AM measurement recorded for the 7 days prior to the first dose of investigational product. LS means and SE were derived from MMRM model with change from baseline in AM PEF (liters/minute) values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline AM PEF (liters/minute) value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening (PM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter. Peak flow testing for PM PEF was performed in evening prior to taking any salbutamol/albuterol or levosalbutamol/levalbuterol reliever medication. Baseline PM PEF was the mean PM measurement recorded for the 7 days prior to the first dose of investigational product. LS means and SE were derived from MMRM model with change from baseline in PM PEF (liters/minute) values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PM PEF (liters/minute) value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning (AM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter. Peak flow testing for AM PEF was performed in morning prior to taking any salbutamol/albuterol or levosalbutamol/levalbuterol reliever medication. Baseline AM PEF was the mean AM measurement recorded for the 7 days prior to the first dose of investigational product. LS means and SE were derived from MMRM model with change from baseline in AM PEF (liters/minute) values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline AM PEF (liters/minute) value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening (PM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The PEF is a participant's maximum speed of expiration, as measured with a peak flow meter. Peak flow testing for PM PEF was performed in evening prior to taking any salbutamol/albuterol or levosalbutamol/levalbuterol reliever medication. Baseline PM PEF was the mean PM measurement recorded for the 7 days prior to the first dose of investigational product. LS means and SE were derived from MMRM model with change from baseline in PM PEF (liters/minute) values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PM PEF (liters/minute) value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Vital Capacity (FVC) at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position, measured in liters. LS means and SE were derived from MMRM model with change from baseline in FVC values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline FVC value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Vital Capacity at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position, measured in liters. LS means and SE were derived from MMRM model with change from baseline in FVC values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline FVC value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Expiratory Flow (FEF) 25-75% at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
FEF is the amount of air (in liters) which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF 25-75% was defined as the mean FEF between 25% and 75% of the FVC, where FVC was defined as the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. LS means and SE were derived from MMRM model with change from baseline in FEF 25-75% values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline FEF 25-75% value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Expiratory Flow 25-75% at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
FEF is the amount of air (in liters) which can be forcibly exhaled from the lungs in the first second of a forced exhalation. FEF 25-75% was defined as the mean FEF between 25% and 75% of the FVC, where FVC was defined as the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. LS means and SE were derived from MMRM model with change from baseline in FE F25-75% values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, sex, ethnicity, baseline height, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline FEF 25-75% value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Post-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Participants were assessed for post-bronchodilator FEV1 30 minutes after bronchodilator administration (200 to 400 mg [2 to 4 puffs] of albuterol/salbutamol or 45 to 90 micrograms [2 to 4 puffs] of levalbuterol/levosalbutamol). FEV1 was the volume of air (in liters) exhaled in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in post-bronchodilator FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline post-bronchodilator FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Post-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Participants were assessed for post-bronchodilator FEV1 30 minutes after bronchodilator administration (200 to 400 mg [2 to 4 puffs] of albuterol/salbutamol or 45 to 90 micrograms [2 to 4 puffs] of levalbuterol/levosalbutamol). FEV1 was the volume of air (in liters) exhaled in the first second of a forced expiration as measured by spirometer. LS means and SE were derived from MMRM model with change from baseline in post-bronchodilator FEV1 values up to Week 52 as the response variable, and treatment, baseline weight group, region, ethnicity, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline post-bronchodilator FEV1 value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
The morning asthma symptom score evaluated participant's overall asthma symptoms experienced during the previous night. It ranged from 0 (no asthma symptoms, slept through the night) to 4 (bad night, awake most of the night because of asthma), where lower scores indicate more mild symptoms and higher scores indicate more severe symptoms. LS means and SE were derived from MMRM model with change from baseline in AM asthma symptom score values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline AM asthma symptom score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
The evening asthma symptom score evaluated participant's overall asthma symptoms experienced during the day. It ranged from 0 (very well, no asthma symptoms) to 4 (asthma very bad, unable to carry out daily activities as usual), where lower scores indicate more mild symptoms and higher scores indicate more severe symptoms. LS means and SE were derived from MMRM model with change from baseline in PM asthma symptom score values up to Week 52 as response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PM asthma symptom score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The morning asthma symptom score evaluated participant's overall asthma symptoms experienced during the previous night. It ranged from 0 (no asthma symptoms, slept through the night) to 4 (bad night, awake most of the night because of asthma), where lower scores indicate more mild symptoms and higher scores indicate more severe symptoms. LS means and SE were derived from MMRM model with change from baseline in AM asthma symptom score values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline AM asthma symptom score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The evening asthma symptom score evaluated participant's overall asthma symptoms experienced during the day. It ranged from 0 (very well, no asthma symptoms) to 4 (asthma very bad, unable to carry out daily activities as usual), where lower scores indicate more mild symptoms and higher scores indicate more severe symptoms. LS means and SE were derived from MMRM model with change from baseline in PM asthma symptom score values up to Week 52 as response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PM asthma symptom score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-question Version (ACQ-5-IA) at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
ACQ-5-IA has 5 questions, reflecting top-scoring 5 asthma symptoms: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities, shortness of breath due to asthma and wheeze. Participants were asked to recall how their asthma had been during the previous week and to respond to each of the five symptom questions on a 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). ACQ-5-IA total score was mean of the scores of all 5 questions and, therefore, ranged from 0 (totally controlled) to 6 (severely uncontrolled), higher scores indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-5-IA values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-5-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-question Version at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
ACQ-5-IA has 5 questions, reflecting top-scoring 5 asthma symptoms: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities, shortness of breath due to asthma and wheeze. Participants were asked to recall how their asthma had been during the previous week and to respond to each of the five symptom questions on a 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). ACQ-5-IA total score was mean of the scores of all 5 questions and, therefore, ranged from 0 (totally controlled) to 6 (severely uncontrolled), higher scores indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-5-IA values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-5-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Weeks 2, 4, 8, 12, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
ACQ-7-IA had 7 questions, assessed: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities, shortness of breath due to asthma and wheeze, reliever medication use, and FEV1 (% predicted). Participants recalled their previous week asthma and answered 5 symptom questions on 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). Total score:mean of scores of all 7 questions; ranging from 0 (totally controlled) to 6 (severely uncontrolled), higher score indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-7-IA values up to Week 52 as response variable and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-7-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8,12, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Weeks 2, 4, 8, 12, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
ACQ-7-IA had 7 questions, assessed: frequency of nocturnal awakenings, severity of asthma symptoms in the mornings, limitation of daily activities due to asthma, shortness of breath due to asthma and wheeze, reliever medication use, and FEV1 (% predicted). Participants recalled their previous week asthma and answered 5 symptom questions on 7-point scale ranging from 0 (no impairment) to 6 (maximum impairment). Total score: mean of scores of all 7 questions; ranging from 0 (totally controlled) to 6 (severely uncontrolled), higher score indicated lower asthma control. LS means and SE were derived from MMRM model with change from baseline in ACQ-7-IA values up to Week 52 as response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline ACQ-7-IA value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8,12, 36, 52
Change From Baseline in Number of Puffs of Reliever Medication Used Per 24 Hours at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Participants might be administered salbutamol/albuterol or levosalbutamol/levalbuterol as reliever medication as needed. Number of reliever medication inhalations were recorded daily in electronic diary/PEF meter. When Nebulizer solutions were used as alternative delivery method, nebulizer dose was converted to number of puffs as per conversion factor: salbutamol/albuterol nebulizer solution (2.5 mg) and levosalbutamol/levalbuterol (1.25 mg) corresponds to 4 puffs. Change From Baseline in number of puffs of reliever medication used per 24 hours at specified weeks was reported. LS means and SE were derived from MMRM model with change from baseline in number of puffs of reliever medication/24 hours values up to Week 52 as response variable and treatment, age, baseline: weight group, region, eosinophil level, FeNO level, ICS dose level, visit, treatment by-visit interaction, baseline number of puffs of reliever medication/24 hours value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Puffs of Reliever Medication Used Per 24 Hours at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Participants might be administered salbutamol/albuterol or levosalbutamol/levalbuterol as reliever medication as needed. Number of reliever medication inhalations were recorded daily in electronic diary/PEF meter. When Nebulizer solutions were used as alternative delivery method, nebulizer dose was converted to number of puffs as per conversion factor: salbutamol/albuterol nebulizer solution (2.5 mg) and levosalbutamol/levalbuterol (1.25 mg) corresponds to 4 puffs. Change From Baseline in number of puffs of reliever medication used per 24 hours at specified weeks was reported. LS means and SE were derived from MMRM model with change from baseline in number of puffs of reliever medication/24 hours values up to Week 52 as response variable and treatment, age, baseline: weight group, region, eosinophil level, FeNO level, ICS dose level, visit, treatment by-visit interaction, baseline number of puffs of reliever medication/24 hours value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Nocturnal Awakenings Per Night at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Participants recorded every morning the number of asthma-related nocturnal awakenings requiring use of rescue medication that occurred during the previous night. Change from baseline in number of nocturnal awakenings per night at specified weeks was reported. LS means and SE were derived from MMRM model with change from baseline in number of nocturnal awakenings values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline number of nocturnal awakenings value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Nocturnal Awakenings Per Night at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Participants recorded every morning the number of asthma-related nocturnal awakenings requiring use of rescue medication that occurred during the previous night. Change from baseline in number of nocturnal awakenings per night at specified weeks was reported. LS means and SE were derived from MMRM model with change from baseline in number of nocturnal awakenings values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline number of nocturnal awakenings value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Pediatric Asthma Quality of Life (QoL) Questionnaire With Standardized Activities-Interviewer Administered (PAQLQ[S] IA) Scores at Weeks 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
PAQLQ(S)-IA, a disease-specific, interviewer-administered QoL questionnaire designed to measure functional impairments that are most important to children >=7 years with asthma. The PAQLQ(S)-IA comprises of 23 items in 3 domains: symptoms (10 items), activity limitation (5 items) and emotional function (8 items). Each item was scored on a 7-point likert scale (1=maximal impairment to 7=no impairment). 23 items of questionnaire were averaged to produce 1 overall quality of life score ranging from 1 (severely impaired) to 7 (not impaired at all), higher scores indicated better quality of life. LS means and SE were derived from MMRM model with change from baseline in PAQLQ(S)-IA global score values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PAQLQ(S)-IA global score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 12, 24, 36, 52
Change From Baseline in Pediatric Asthma Quality of Life Questionnaire With Standardized Activities-Interviewer Administered Scores at Weeks 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
PAQLQ(S)-IA, a disease-specific, interviewer-administered QoL questionnaire designed to measure functional impairments that are most important to children >=7 years with asthma. The PAQLQ(S)-IA comprises of 23 items in 3 domains: symptoms (10 items), activity limitation (5 items) and emotional function (8 items). Each item was scored on a 7-point likert scale (1=maximal impairment to 7=no impairment). 23 items of questionnaire were averaged to produce 1 overall quality of life score ranging from 1 (severely impaired) to 7 (not impaired at all), higher scores indicated better quality of life. LS means and SE were derived from MMRM model with change from baseline in PAQLQ(S)-IA global score values up to Week 52 as the response variable, and treatment, age, baseline weight group, region, baseline eosinophil level, baseline FeNO level, baseline ICS dose level, visit, treatment by-visit interaction, baseline PAQLQ(S)-IA global score value and baseline-by-visit interaction as covariates.
Measured: Baseline, Weeks 12, 24, 36, 52
Healthcare Resource Utilization (HCRU): Number of School and Work Days Missed Due to LOAC: Type 2 Inflammatory Asthma Phenotype Population
The number of days missed from school by the participant and the number of days missed from work by the caregiver of participant due to a LOAC were collected in the electronic-case report form (eCRF). Cumulative number of missed days (school days and work days) up to week 52 were computed and summarized using mean and standard deviation (SD).
Measured: Baseline to Week 52
Healthcare Resource Utilization: Number of School and Work Days Missed Due to LOAC: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The number of days missed from school by the participant and the number of days missed from work by the caregiver of participant due to a LOAC were collected in the eCRF. Cumulative number of missed days (school days and work days) up to week 52 were computed and summarized using mean and SD.
Measured: Baseline to Week 52
Healthcare Resource Utilization: Percentage of Participants Who Had Missed Greater Than or Equal to 5 School/Work Days Due to LOAC: Type 2 Inflammatory Asthma Phenotype Population
The number of days missed from school for the participant and the missed number of days from work for the caregiver due to a LOAC were collected in the eCRF. The percentage of participants who had at least 5 days (school days and work days) missed due to LOAC over the study period was reported.
Measured: Baseline to Week 52
Healthcare Resource Utilization: Percentage of Participants Who Had Missed Greater Than or Equal to 5 School/Work Days Due to LOAC: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
The number of days missed from school for the participant and the missed number of days from work for the caregiver due to a LOAC were collected in the eCRF. The percentage of participants who had at least 5 days (school days and work days) missed due to LOAC over the study period was reported.
Measured: Baseline to Week 52
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)
Adverse event (AE) was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and did not necessary have to had a causal relationship with treatment.TEAEs were defined as AEs that developed or worsened in grade or became serious during TEAE period which was defined as the period from the time of first dose of study drug to the end of post-treatment period. A serious adverse events (SAE) was any untoward medical occurrence that at any dose resulted in: death; or life-threatening experience; or required inpatient hospitalization or prolongation of existing hospitalization; or resulted in persistent or significant disability/incapacity; or was a congenital anomaly/birth defect or a medically important event. TEAEs included both SAEs and non-SAEs.
Measured: From Baseline up to Week 64
Pharmacokinetics (PK) Assessment: Functional Dupilumab Concentration in Serum
Data for this outcome measure was planned to be collected and analyzed separately for dupilumab 100 mg and 200 mg dose and not planned to be collected and analyzed for placebo arm.
Measured: Baseline, Weeks 6, 12, 24, 52, 64
Percentage of Participants With Treatment Emergent Antidrug Antibodies (ADA) Response
ADA response was categorized as: treatment emergent and treatment boosted response. 1) Treatment emergent was defined as an ADA positive response in the assay post first dose, when baseline results were negative or missing. 2) Treatment boosted was defined as: an ADA positive response in the assay post first dose that was greater-than or equal to 4-fold over baseline titer levels, when baseline results were positive. The criteria for positive was defined as "30 to > 10,000", where low titer (< 1,000); moderate (1,000 <= titer <= 10,000) and high titer (> 10,000).
Measured: From Baseline up to Week 64
Percentage of Participants With Seroconversion
Seroconversion was defined as a post-vaccination titer >=40 (1/dilution) for those with a pre-vaccination titer <10 (1/dilution), or a >= 4-fold increase in post-vaccination titer for those with a pre-vaccination titer >=10 (1/dilution).
Measured: From Baseline up to Week 64
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2016 (estimated)
- Study start21 Apr 2017
- Primary completion26 Aug 2020
- Study completion26 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (11)Papers the sponsor has linked to this study.
- Other trials for AsthmaStudies of the same condition, open and under way.
- More trials run by SanofiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 408 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sanofi
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Regeneron Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Clinical Sciences & Operations, Study director, Sanofi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02948959
- Sponsor's protocol ID
- EFC14153
- Other IDs
- 2016-001607-23, U1111-1179-4851, 2016
- Acronym
- VOYAGE
- Status last verified
- Mar 2022
- First submitted
- 27 Oct 2016
- Last updated
- 28 Mar 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Publications
Dell S, Guilbert TW, Deschildre A, Phipatanakul W, Papadopoulos NG, Murray CS, Xia C, Moraco A, Shams M, Bacharier LB. Dupilumab outcomes in pediatric asthma by early eosinophil status: Post hoc analysis of VOYAGE/EXCURSION. J Allergy Clin Immunol. 2026 Sep;158(3):727-738. doi: 10.1016/j.jaci.2026.06.012. Epub 2026 Jun 30.
PubMed 42379525Jackson DJ, Hamelmann E, Roberts G, Bacharier LB, Xia C, Gall R, Ledanois O, Coleman A, Tawo K, Jacob-Nara JA, Radwan A, Rowe PJ, Deniz Y. Dupilumab Efficacy and Safety in Children With Moderate to Severe Asthma and High Blood Eosinophils: A Post Hoc Analysis of VOYAGE. J Allergy Clin Immunol Pract. 2025 Mar;13(3):568-575. doi: 10.1016/j.jaip.2024.11.014. Epub 2024 Nov 28.
PubMed 39613097Maspero JF, Antila MA, Deschildre A, Bacharier LB, Altincatal A, Laws E, Mortensen E, Radwan A, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M. Dupilumab Efficacy in Children With Type 2 Asthma Receiving High- to Medium-Dose Inhaled Corticosteroids (VOYAGE). J Allergy Clin Immunol Pract. 2024 Dec;12(12):3303-3312. doi: 10.1016/j.jaip.2024.08.038. Epub 2024 Aug 28.
PubMed 39209068Phipatanakul W, Vogelberg C, Bacharier LB, Dell S, Altincatal A, Gall R, Ledanois O, Sacks H, Jacob-Nara JA, Deniz Y, Rowe PJ. Dupilumab 200 mg was efficacious in children (6-11 years) with moderate-to-severe asthma for up to 2 years: EXCURSION open-label extension study. Pediatr Pulmonol. 2024 Nov;59(11):2976-2983. doi: 10.1002/ppul.27167. Epub 2024 Jul 17.
PubMed 39016623Guilbert TW, Murphy KR, Hamelmann E, Ross KR, Gupta A, Fiocchi A, Xia C, Gall R, Ledanois O, Radwan A, Jacob-Nara JA, Rowe PJ, Deniz Y. Impact of Lung Function on Asthma Exacerbation Rates in Children Treated with Dupilumab: The VOYAGE Study. J Asthma Allergy. 2024 Feb 8;17:81-87. doi: 10.2147/JAA.S425101. eCollection 2024.
PubMed 38347908Bacharier LB, Pavord ID, Maspero JF, Jackson DJ, Fiocchi AG, Mao X, Jacob-Nara JA, Deniz Y, Laws E, Mannent LP, Amin N, Akinlade B, Staudinger HW, Lederer DJ, Hardin M. Blood eosinophils and fractional exhaled nitric oxide are prognostic and predictive biomarkers in childhood asthma. J Allergy Clin Immunol. 2024 Jul;154(1):101-110. doi: 10.1016/j.jaci.2023.09.044. Epub 2024 Jan 23.
PubMed 38272375Bacharier LB, Guilbert TW, Katelaris CH, Deschildre A, Phipatanakul W, Liu D, Altincatal A, Mannent LP, Amin N, Laws E, Akinlade B, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M. Dupilumab Improves Lung Function Parameters in Pediatric Type 2 Asthma: VOYAGE Study. J Allergy Clin Immunol Pract. 2024 Apr;12(4):948-959. doi: 10.1016/j.jaip.2023.12.006. Epub 2023 Dec 11.
PubMed 38092225Fiocchi AG, Phipatanakul W, Zeiger RS, Durrani SR, Cole J, Msihid J, Gall R, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M, Zhang Y, Khan AH. Dupilumab leads to better-controlled asthma and quality of life in children: the VOYAGE study. Eur Respir J. 2023 Nov 2;62(5):2300558. doi: 10.1183/13993003.00558-2023. Print 2023 Nov.
PubMed 37734856Papadopoulos NG, Szefler SJ, Bacharier LB, Maspero JF, Domingo C, Fiocchi A, Lee JK, Daizadeh N, Lederer DJ, Hardin M, Gall R, Djandji M, Siddiqui S, Jacob-Nara JA, Deniz Y, Rowe PJ. Assessment of dupilumab in children with moderate-to-severe type 2 asthma with or without evidence of allergic asthma. Allergy. 2023 Aug;78(8):2157-2167. doi: 10.1111/all.15743. Epub 2023 May 25.
PubMed 37059696Jackson DJ, Bacharier LB, Phipatanakul W, Sher L, Domingo C, Papadopoulos N, Modena B, Li N, Xia C, Kamal MA, Dillon M, Wolfe K, Gall R, Amin N, Mannent LP, Laws E, Rowe PJ, Jacob-Nara JA, Deniz Y, Lederer DJ, Hardin M, Xu C. Dupilumab pharmacokinetics and effect on type 2 biomarkers in children with moderate-to-severe asthma. Ann Allergy Asthma Immunol. 2023 Jul;131(1):44-51.e4. doi: 10.1016/j.anai.2023.03.014. Epub 2023 Mar 22.
PubMed 36958470Bacharier LB, Maspero JF, Katelaris CH, Fiocchi AG, Gagnon R, de Mir I, Jain N, Sher LD, Mao X, Liu D, Zhang Y, Khan AH, Kapoor U, Khokhar FA, Rowe PJ, Deniz Y, Ruddy M, Laws E, Patel N, Weinreich DM, Yancopoulos GD, Amin N, Mannent LP, Lederer DJ, Hardin M; Liberty Asthma VOYAGE Investigators. Dupilumab in Children with Uncontrolled Moderate-to-Severe Asthma. N Engl J Med. 2021 Dec 9;385(24):2230-2240. doi: 10.1056/NEJMoa2106567.
PubMed 34879449
Study at a glance
From the registry record, last updated by the sponsor on 28 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Dupilumab in Children 6 to <12 Years of Age With Uncontrolled Persistent Asthma
- ClinicalTrials.gov ID
- NCT02948959
- Acronym / sponsor ID
- VOYAGE / EFC14153
- Conditions
- Asthma
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 408 participants (actual)
- Lead sponsor
- SanofiIndustry (pharma, biotech, devices)
- Collaborators
- Regeneron Pharmaceuticals
- Study start
- 21 Apr 2017 (actual)
- Primary completion
- 26 Aug 2020 (actual)
- Study completion
- 26 Aug 2020 (actual)
- First posted
- 31 Oct 2016 (estimated)
- Results
- Posted 20 Oct 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Primary Objective:
To evaluate the efficacy of dupilumab in children 6 to less than (<) 12 years of age with uncontrolled persistent asthma.
Secondary Objective:
To evaluate in children 6 to <12 years of age with uncontrolled persistent asthma:
- The safety and tolerability of dupilumab.
- The evaluate the effect of dupilumab in improving participant reported outcomes including health related quality of life.
- The dupilumab systemic exposure and incidence of anti-drug antibodies.
- The evaluate the association between dupilumab treatment and pediatric immune responses to vaccines: any vaccination for tetanus, diphtheria, pertussis and/or seasonal trivalent/quadrivalent influenza vaccine.
Detailed description
The total study duration per participant was up to 69 weeks, consisted of a screening period of 3-5 weeks, a randomized treatment period of 52 weeks and a post-treatment period of 12 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo, Drug: Asthma Controller Therapies, Drug: Asthma Reliever TherapiesPlacebo (for Dupilumab), subcutaneous (SC) injection every 2 weeks (q2w) for 52 weeks in combination with stable-dose background therapy of medium-dose inhaled corticosteroids (ICS) with a second controller medication (i.e., long-acting β2 agonist [LABA], long acting muscarinic antagonist [LAMA], leukotriene receptor antagonist [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. Participants were followed up for 12 weeks after last dose (i.e. up to Week 64). |
| Dupilumab | Experimental | Drug: Dupilumab, Drug: Asthma Controller Therapies, Drug: Asthma Reliever TherapiesDupilumab 200 milligrams (mg) (in 1.14 milliliters [mL] for >30 kilograms [kg] bodyweight [BW]) or 100 mg (in 0.67 mL for less than or equal to (<=) 30 kg BW), SC injection q2w for 52 weeks in combination with 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. Participants were followed up for 12 weeks after last dose (i.e. up to Week 64). |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- Children 6 to <12 years of age, with a physician diagnosis of persistent asthma for greater than or equal to (>=)12 months prior to screening, based on clinical history and examination, pulmonary function parameters according to Global initiative for asthma (GINA) 2015 Guidelines and the following criteria:
- Existing background therapy of medium-dose ICS with second controller medication (i.e., long-acting β2 agonist , leukotriene receptor antagonist, long acting muscarinic antagonist, or methylxanthines) or high-dose ICS alone or high-dose ICS with second controller, for at least 3 months with a stable dose >=1 month prior to Screening Visit 1.
- Pre-bronchodilator forced expiratory volume in 1 second (FEV1) <=95 percentage (%) of predicted normal or pre bronchodilator FEV1/forced vital capacity ratio <0.85 at Screening and Baseline Visits.
- Reversibility of at least 10% in FEV1 after the administration of 200 to 400 micrograms (mcg; 2 to 4 puff inhalations with metered-dose inhaler [MDI]) of albuterol/salbutamol or 45 to 90 mcg (2 to 4 puffs with MDI) of levalbuterol/levosalbutamol reliever medication before randomization (up to 3 opportunities during the same visit were allowed with a maximum of 12 puffs of reliever medication if tolerated by the participant).
- Must had experienced, within 1 year prior to Screening Visit 1, any of the following events:
Key exclusion criteria (6 listed)
- Participants <6 or >=12 years of age.
- Participants with <16 kg bodyweight.
- Any other chronic lung disease (cystic fibrosis, bronchopulmonary dysplasia, etc.), which may impair lung function.
- A participant with any history of life threatening asthma (ie, extreme exacerbation that requires intubation).
- Co-morbid disease that might interfere with the evaluation of investigational medicinal product.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DupilumabAlso: SAR231893 (REGN668)Pharmaceutical form: Solution Route of administration: Subcutaneous | Drug | Dupilumab |
| PlaceboPharmaceutical form: Solution Route of administration: Subcutaneous | Drug | Placebo |
| Asthma Controller TherapiesPharmaceutical form: Aerosol, capsules, tablets, oral solution Route of administration: Inhaled, oral | Drug | Dupilumab, Placebo |
| Asthma Reliever TherapiesPharmaceutical form: Nebulized, aerosol Route of administration: Inhaled | Drug | Dupilumab, Placebo |
Primary outcome
Annualized Rate of Severe Exacerbation Events During the 52-Week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline to Week 52
A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization or emergency room visit because of asthma requiring systemic corticosteroid treatment. Annualized event rate was defined as the total number of severe exacerbation events that occurred during the 52-week treatment period divided by the total number of participant-years followed in the 52-week treatment period.
Annualized Rate of Severe Exacerbation Events During the 52-Week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline to Week 52
A severe asthma exacerbation event was defined as a deterioration of asthma during the 52-week treatment period requiring: use of systemic corticosteroids for >=3 days; and/or hospitalization or emergency room visit because of asthma requiring systemic corticosteroid treatment. Annualized event rate was defined as the total number of severe exacerbation events that occurred during the 52-week treatment period divided by the total number of participant-years followed in the 52-week treatment period.
48 secondary outcomes
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 (FEV1) Second at Week 12: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Week 12
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 12: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Week 12
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version (ACQ-7-IA) at Week 24: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Week 24
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Week 24: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Week 24
Change From Baseline in Fractional Exhaled Nitric Oxide Level at Week 12: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Week 12
Change From Baseline in Fractional Exhaled Nitric Oxide Level at Week 12: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Week 12
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second at Weeks 2, 4, 8, 24, 36 and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 24, 36, 52
Change From Baseline in Pre-bronchodilator Percent Predicted Forced Expiratory Volume in 1 Second at Weeks 2, 4, 8, 24, 36 and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 24, 36, 52
Time to First Severe Exacerbation Event: Kaplan-Meier Estimates During 52-week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline up to Week 52
Time to First Severe Exacerbation Event: Kaplan-Meier Estimates During 52-week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline up to Week 52
Time to First Loss of Asthma Control (LOAC) Event: Kaplan-Meier Estimates During 52-week Treatment Period: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline up to Week 52
Time to First Loss of Asthma Control Event: Kaplan-Meier Estimates During 52-week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline up to Week 52
Absolute Change From Baseline in Pre-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Absolute Change From Baseline in Pre-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Percent Change From Baseline in Pre-Bronchodilator Percent Predicted FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Percent Change From Baseline in Pre-Bronchodilator Percent Predicted FEV1 at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning (AM) Peak Expiratory Flow (PEF) at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening (PM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning (AM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening (PM) Peak Expiratory Flow at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Vital Capacity (FVC) at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Vital Capacity at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Expiratory Flow (FEF) 25-75% at Weeks 2, 4, 8, 12, 24, 36, 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Forced Expiratory Flow 25-75% at Weeks 2, 4, 8, 12, 24, 36, 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Post-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Post-Bronchodilator FEV1 at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Morning Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Evening Asthma Symptom Score at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-question Version (ACQ-5-IA) at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 5-question Version at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Weeks 2, 4, 8, 12, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8,12, 36, 52
Change From Baseline in Asthma Control Questionnaire-Interviewer Administered, 7-question Version at Weeks 2, 4, 8, 12, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8,12, 36, 52
Change From Baseline in Number of Puffs of Reliever Medication Used Per 24 Hours at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Puffs of Reliever Medication Used Per 24 Hours at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Nocturnal Awakenings Per Night at Weeks 2, 4, 8, 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Number of Nocturnal Awakenings Per Night at Weeks 2, 4, 8, 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 2, 4, 8, 12, 24, 36, 52
Change From Baseline in Pediatric Asthma Quality of Life (QoL) Questionnaire With Standardized Activities-Interviewer Administered (PAQLQ[S] IA) Scores at Weeks 12, 24, 36, and 52: Type 2 Inflammatory Asthma Phenotype Population
Time frame: Baseline, Weeks 12, 24, 36, 52
Change From Baseline in Pediatric Asthma Quality of Life Questionnaire With Standardized Activities-Interviewer Administered Scores at Weeks 12, 24, 36, and 52: Baseline Blood Eosinophils >=300 Cells Per Microliter Population
Time frame: Baseline, Weeks 12, 24, 36, 52
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Mar 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42379525Dell S, Guilbert TW, Deschildre A, Phipatanakul W, Papadopoulos NG, Murray CS, Xia C, Moraco A, Shams M, Bacharier LB. Dupilumab outcomes in pediatric asthma by early eosinophil status: Post hoc analysis of VOYAGE/EXCURSION. J Allergy Clin Immunol. 2026 Sep;158(3):727-738. doi: 10.1016/j.jaci.2026.06.012. Epub 2026 Jun 30.
- Derived from this study: PubMed 39613097Jackson DJ, Hamelmann E, Roberts G, Bacharier LB, Xia C, Gall R, Ledanois O, Coleman A, Tawo K, Jacob-Nara JA, Radwan A, Rowe PJ, Deniz Y. Dupilumab Efficacy and Safety in Children With Moderate to Severe Asthma and High Blood Eosinophils: A Post Hoc Analysis of VOYAGE. J Allergy Clin Immunol Pract. 2025 Mar;13(3):568-575. doi: 10.1016/j.jaip.2024.11.014. Epub 2024 Nov 28.
- Derived from this study: PubMed 39209068Maspero JF, Antila MA, Deschildre A, Bacharier LB, Altincatal A, Laws E, Mortensen E, Radwan A, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M. Dupilumab Efficacy in Children With Type 2 Asthma Receiving High- to Medium-Dose Inhaled Corticosteroids (VOYAGE). J Allergy Clin Immunol Pract. 2024 Dec;12(12):3303-3312. doi: 10.1016/j.jaip.2024.08.038. Epub 2024 Aug 28.
- Derived from this study: PubMed 39016623Phipatanakul W, Vogelberg C, Bacharier LB, Dell S, Altincatal A, Gall R, Ledanois O, Sacks H, Jacob-Nara JA, Deniz Y, Rowe PJ. Dupilumab 200 mg was efficacious in children (6-11 years) with moderate-to-severe asthma for up to 2 years: EXCURSION open-label extension study. Pediatr Pulmonol. 2024 Nov;59(11):2976-2983. doi: 10.1002/ppul.27167. Epub 2024 Jul 17.
- Derived from this study: PubMed 38347908Guilbert TW, Murphy KR, Hamelmann E, Ross KR, Gupta A, Fiocchi A, Xia C, Gall R, Ledanois O, Radwan A, Jacob-Nara JA, Rowe PJ, Deniz Y. Impact of Lung Function on Asthma Exacerbation Rates in Children Treated with Dupilumab: The VOYAGE Study. J Asthma Allergy. 2024 Feb 8;17:81-87. doi: 10.2147/JAA.S425101. eCollection 2024.
- Derived from this study: PubMed 38272375Bacharier LB, Pavord ID, Maspero JF, Jackson DJ, Fiocchi AG, Mao X, Jacob-Nara JA, Deniz Y, Laws E, Mannent LP, Amin N, Akinlade B, Staudinger HW, Lederer DJ, Hardin M. Blood eosinophils and fractional exhaled nitric oxide are prognostic and predictive biomarkers in childhood asthma. J Allergy Clin Immunol. 2024 Jul;154(1):101-110. doi: 10.1016/j.jaci.2023.09.044. Epub 2024 Jan 23.
- Derived from this study: PubMed 38092225Bacharier LB, Guilbert TW, Katelaris CH, Deschildre A, Phipatanakul W, Liu D, Altincatal A, Mannent LP, Amin N, Laws E, Akinlade B, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M. Dupilumab Improves Lung Function Parameters in Pediatric Type 2 Asthma: VOYAGE Study. J Allergy Clin Immunol Pract. 2024 Apr;12(4):948-959. doi: 10.1016/j.jaip.2023.12.006. Epub 2023 Dec 11.
- Derived from this study: PubMed 37734856Fiocchi AG, Phipatanakul W, Zeiger RS, Durrani SR, Cole J, Msihid J, Gall R, Jacob-Nara JA, Deniz Y, Rowe PJ, Lederer DJ, Hardin M, Zhang Y, Khan AH. Dupilumab leads to better-controlled asthma and quality of life in children: the VOYAGE study. Eur Respir J. 2023 Nov 2;62(5):2300558. doi: 10.1183/13993003.00558-2023. Print 2023 Nov.
- Derived from this study: PubMed 37059696Papadopoulos NG, Szefler SJ, Bacharier LB, Maspero JF, Domingo C, Fiocchi A, Lee JK, Daizadeh N, Lederer DJ, Hardin M, Gall R, Djandji M, Siddiqui S, Jacob-Nara JA, Deniz Y, Rowe PJ. Assessment of dupilumab in children with moderate-to-severe type 2 asthma with or without evidence of allergic asthma. Allergy. 2023 Aug;78(8):2157-2167. doi: 10.1111/all.15743. Epub 2023 May 25.
- Derived from this study: PubMed 36958470Jackson DJ, Bacharier LB, Phipatanakul W, Sher L, Domingo C, Papadopoulos N, Modena B, Li N, Xia C, Kamal MA, Dillon M, Wolfe K, Gall R, Amin N, Mannent LP, Laws E, Rowe PJ, Jacob-Nara JA, Deniz Y, Lederer DJ, Hardin M, Xu C. Dupilumab pharmacokinetics and effect on type 2 biomarkers in children with moderate-to-severe asthma. Ann Allergy Asthma Immunol. 2023 Jul;131(1):44-51.e4. doi: 10.1016/j.anai.2023.03.014. Epub 2023 Mar 22.
- Derived from this study: PubMed 34879449Bacharier LB, Maspero JF, Katelaris CH, Fiocchi AG, Gagnon R, de Mir I, Jain N, Sher LD, Mao X, Liu D, Zhang Y, Khan AH, Kapoor U, Khokhar FA, Rowe PJ, Deniz Y, Ruddy M, Laws E, Patel N, Weinreich DM, Yancopoulos GD, Amin N, Mannent LP, Lederer DJ, Hardin M; Liberty Asthma VOYAGE Investigators. Dupilumab in Children with Uncontrolled Moderate-to-Severe Asthma. N Engl J Med. 2021 Dec 9;385(24):2230-2240. doi: 10.1056/NEJMoa2106567.
- Individual participant dataSharing planned: Yes. Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Sciences & Operations study director
Sanofi
99 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dupilumab and Asthma Reliever Therapies in people with Asthma, run by Sanofi at 99 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 99 sites in United States, Hungary, Ukraine, Brazil, Chile and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Oct 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Annualized Rate of Severe Exacerbation Events During the 52-Week Treatment Period: Baseline Blood Eosinophils >=300 Cells Per Microliter Population (time frame: Baseline to Week 52). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02948959, which also lists 11 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Mar 2022.