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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02555683




Registration number
NCT02555683
Ethics application status
Date submitted
18/09/2015
Date registered
21/09/2015
Date last updated
18/05/2020

Titles & IDs
Public title
Study of Efficacy and Safety of QAW039 in Patients With Severe Asthma Inadequately Controlled With Standard of Care Asthma Treatment.
Scientific title
A 52-week, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy and Safety of QAW039 When Added to Existing Asthma Therapy in Patients With Uncontrolled Severe Asthma.
Secondary ID [1] 0 0
2015-002553-35
Secondary ID [2] 0 0
CQAW039A2307
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Asthma 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Asthma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - QAW039
Treatment: Drugs - QAW039
Treatment: Drugs - Placebo

Experimental: QAW039 150 mg - QAW039 150 mg once daily

Experimental: QAW039 450 mg - QAW039 450 mg once daily

Placebo Comparator: Placebo - Placebo once daily


Treatment: Drugs: QAW039
QAW039 150 mg once daily

Treatment: Drugs: QAW039
QAW039 450 mg once daily

Treatment: Drugs: Placebo
Placebo once daily

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation - A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours). The high eosinophils subpopulation consists of all patients with blood eosinophil count = 250 cells/µL at baseline.
Timepoint [1] 0 0
Baseline, Week 52
Primary outcome [2] 0 0
Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in Overall Population - A severe asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and hospitalization; or treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days and emergency department visit (greater than 24 hours*); or death due to asthma. A moderate asthma exacerbation is defined as treatment with 'rescue' systemic corticosteroids for greater than or equal to 3 days either as an outpatient or in emergency department visits (Emergency department visit less than or equal to 24 hours).
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [1] 0 0
Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in High Eosinophils Subpopulation - The AQLQ+12 is comprised of a total of 32 individual questions that span a total of four domains: symptoms, activity limitation, emotional function, and environmental stimuli.
Patients were asked to recall their experiences during the previous 2 weeks and to score each item on a 7-point scale (7 = not at all impaired to 1 = severely impaired). The AQLQ+12 yields individual domain scores, which is the mean of all items in each domain, and an overall score, which is the mean of all 32 individual responses. Higher scores indicate less impairment in health-related quality of life. The high eosinophils subpopulation consists of all patients with blood eosinophil count = 250 cells/µL at baseline.
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Change From Baseline to Week 52 in Asthma Control Questionnaire-5(ACQ-5) Score in High Eosinophils Subpopulation - The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. Patients were asked to recall how their asthma had been during the previous week and to respond to the symptom questions on a 7-point scale (0=no impairment, 6=maximum impairment). The questions are equally weighted and the ACQ-5 score is the mean of the 5 questions: therefore, between 0 (totally controlled) and 6 (severely uncontrolled). The high eosinophils subpopulation consists of all patients with blood eosinophil count = 250 cells/µL at baseline.
Timepoint [2] 0 0
Baseline, Week 52
Secondary outcome [3] 0 0
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in High Eosinophils Subpopulation - Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug. The high eosinophils subpopulation consists of all patients with blood eosinophil count = 250 cells/µL at baseline.
Timepoint [3] 0 0
Baseline, Week 52
Secondary outcome [4] 0 0
Change From Baseline to Week 52 in Asthma Quality of Life Questionnaire for Participants 12 Years and Older (AQLQ+12) Score in Overall Population - The AQLQ+12 is comprised of a total of 32 individual questions that span a total of four domains: symptoms, activity limitation, emotional function, and environmental stimuli.
Patients were asked to recall their experiences during the previous 2 weeks and to score each item on a 7-point scale (7 = not at all impaired to 1 = severely impaired). The AQLQ+12 yields individual domain scores, which is the mean of all items in each domain, and an overall score, which is the mean of all 32 individual responses. Higher scores indicate less impairment in health-related quality of life.
Timepoint [4] 0 0
Baseline, Week 52
Secondary outcome [5] 0 0
Change From Baseline to Week 52 in Asthma Control Questionnaire-5(ACQ-5) Score in Overall Population - The ACQ-5 is a five-item, self-completed questionnaire, which is used as a measure of asthma control of a participant. Patients were asked to recall how their asthma had been during the previous week and to respond to the symptom questions on a 7-point scale (0=no impairment, 6=maximum impairment). The questions are equally weighted and the ACQ-5 score is the mean of the 5 questions: therefore, between 0 (totally controlled) and 6 (severely uncontrolled).
Timepoint [5] 0 0
Baseline, Week 52
Secondary outcome [6] 0 0
Change From Baseline to Week 52 in Pre-dose Forced Expiratory Volume in 1 Second (FEV1) in Overall Population - Forced Expiratory Volume in one second (FEV1) is calculated as the volume of air forcibly exhaled in one second as measured by a spirometer. Baseline is defined as the last available FEV1 measurement taken prior to the first dose of randomized study drug.
Timepoint [6] 0 0
Baseline, Week 52

Eligibility
Key inclusion criteria
- Written informed consent and assent (if applicable).

- Male and female patients aged =12 years (or =lower age limit allowed by health
authority and/or ethics committee/institutional review board approvals).

- A diagnosis of severe asthma, uncontrolled on GINA 4/5 asthma medication.

- Evidence of airway reversibility or airway hyper- reactivity.

- FEV1 of =80% of the predicted normal value for patients aged =18 years; FEV1 of =90%
for patients aged 12 to <18 years

- An ACQ score =1.5.

- A history of 2 or more asthma exacerbations within the 12 months prior to entering the
study.
Minimum age
12 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Use of other investigational drugs within 5 half-lives of study entry, or within 30
days, whichever is longer.

- Subjects who have participated in another trial of QAW039.

- A QTcF (Fridericia) =450 msec (male) or =460 msec (female).

- History of malignancy with the exception of local basal cell carcinoma of the skin.

- Pregnant or nursing (lactating) women.

- Serious co-morbidities.

- Patients on greater than 20 mg of simvastatin, greater than 40 mg of atorvastatin,
greater than 40 mg of pravastatin, or greater than 2 mg of pitavastatin

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Bedford Park
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Novartis Investigative Site - Clayton
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Novartis Investigative Site - Footscray
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Novartis Investigative Site - Heidelberg
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Novartis Investigative Site - Melbourne
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5041 - Bedford Park
Recruitment postcode(s) [2] 0 0
3168 - Clayton
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3011 - Footscray
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3084 - Heidelberg
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3004 - Melbourne
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Ho Chi Minh

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This study aimed to determine the efficacy and safety of QAW039 150 mg and QAW039 450 mg,
compared with placebo, when added to GINA (Global Initiative for Asthma) steps 4 and 5
standard-of- care (SoC) asthma therapy (GINA 2016) in the following two populations:

- patient with inadequately controlled severe asthma and high eosinophil counts at
baseline (eosinophil count at Visit 1 =250 cells/ µl) (sub-population)

- patients with inadequately controlled severe asthma (overall study population)

Inadequate control is defined as partly controlled or uncontrolled asthma (GINA 2016).
Trial website
https://clinicaltrials.gov/show/NCT02555683
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications