Asthma
Conditions
Keywords
Asthma
Brief summary
This study planned to assess the effect on the reduction in rate of severe asthma exacerbations of different dose levels of QGE031 in asthma patients that are inadequately controlled with inhaled steroid plus beta-2 agonist medication with or without oral steroid. However, this study was terminated due to the efficacy results from an interim analysis (at the end of treatment epoch) of the Phase II study CQGE031B2201 (NCT01716754). Planned data analyses were not performed for this study due to the early termination and the very limited dataset (only 10 participants received study medication of the 440 participants planned).
Interventions
DRUGQGE031
QGE031 120 mg per 1 mL liquid in vial
DRUGPlacebo
Placebo to QGE031 0 mg per 1 mL liquid in vial
Sponsors
Novartis Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * A diagnosis of allergic asthma, uncontrolled on current medication. * History of at least 2 asthma exacerbations during the last 1 year * Forced Expiratory Volume in one second (FEV1) of ≥ 40% and ≤ 80% of the predicted normal value; reversibility following administration of bronchodilator. Key
Exclusion criteria
* Use of tobacco products within the previous 6 months (Social occasional smokers may be included). * Recent asthma attack/exacerbation or asthma worsening/ respiratory infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| QGE031 Compared to Placebo in Atopic Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations | Week 52 |
Secondary
| Measure | Time frame |
|---|---|
| QGE031 Compared to Placebo in Non-atopic Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations | Week 52 |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on the Reduction in Rate of Asthma Exacerbations (by Severity) | Week 52 |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Time to First Asthma Exacerbations (by Severity) | Week 52 |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Change From Baseline in Asthma Control Questionnaire (ACQ) | Baseline, Treatment (Weeks 4, 8, 12, 16, 24, 36, 52), follow up (Weeks 60 and 72) |
| QGE031 Compared to Placebo in All Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations | Week 52 |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Forced Expiratory Volume in 1 Second (FEV1) | Baseline, Treatment (Weeks 4, 8, 12, 16, 24, 36, 52), follow up (Weeks 60 and 72) |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Peak Expiratory Flow (PEF) in the Morning and Evening | Over 52 weeks (Treatment) and 20 weeks (follow-up) |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Total Daily Symptom Score | Over 52 weeks (Treatment) and 20 weeks (follow-up) |
| Response to QGE031 Between Atopic Asthma and Non-atopic Asthma | Over 52 weeks (treatment) and 20 weeks (follow up) |
| QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Change From Baseline in Asthma Control Diary (ACD) | Over 52 weeks (Treatment) and 20 weeks (follow-up) |
Countries
Belgium, Canada, France, Germany, Hungary, Italy, Japan, Netherlands, Slovakia, United States
Participant flow
Pre-assignment details
Participants were randomized to one of the four treatment arms.
Participants by arm
| Arm | Count |
|---|---|
| QGE031 240 mg QGE031 240 mg subcutaneous injection every 4 weeks | 2 |
| QGE031 72 mg QGE031 72 mg subcutaneous injection every 4 weeks | 2 |
| QGE031 24 mg QGE031 24 mg subcutaneous injection every 4 weeks | 3 |
| Placebo to QGE031 Placebo subcutaneous injection every 4 weeks | 3 |
| Total | 10 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 |
|---|---|---|---|---|---|
| Overall Study | Study terminated. | 2 | 2 | 3 | 3 |
Baseline characteristics
| Characteristic | QGE031 240 mg | QGE031 72 mg | QGE031 24 mg | Placebo to QGE031 | Total |
|---|---|---|---|---|---|
| Age, Continuous | 46.5 Years STANDARD_DEVIATION 16.26 | 47.0 Years STANDARD_DEVIATION 1.41 | 53.7 Years STANDARD_DEVIATION 11.59 | 47.7 Years STANDARD_DEVIATION 2.89 | 49.1 Years STANDARD_DEVIATION 8.45 |
| Sex: Female, Male Female | 2 Participants | 0 Participants | 3 Participants | 2 Participants | 7 Participants |
| Sex: Female, Male Male | 0 Participants | 2 Participants | 0 Participants | 1 Participants | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — | — / — | — / — |
| other Total, other adverse events | 1 / 2 | 2 / 2 | 1 / 3 | 2 / 3 |
| serious Total, serious adverse events | 0 / 2 | 0 / 2 | 0 / 3 | 0 / 3 |
Outcome results
Primary
QGE031 Compared to Placebo in Atopic Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations
Time frame: Week 52
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in All Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations
Time frame: Week 52
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Change From Baseline in Asthma Control Diary (ACD)
Time frame: Over 52 weeks (Treatment) and 20 weeks (follow-up)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Change From Baseline in Asthma Control Questionnaire (ACQ)
Time frame: Baseline, Treatment (Weeks 4, 8, 12, 16, 24, 36, 52), follow up (Weeks 60 and 72)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Forced Expiratory Volume in 1 Second (FEV1)
Time frame: Baseline, Treatment (Weeks 4, 8, 12, 16, 24, 36, 52), follow up (Weeks 60 and 72)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Peak Expiratory Flow (PEF) in the Morning and Evening
Time frame: Over 52 weeks (Treatment) and 20 weeks (follow-up)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on the Reduction in Rate of Asthma Exacerbations (by Severity)
Time frame: Week 52
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Time to First Asthma Exacerbations (by Severity)
Time frame: Week 52
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Asthma Patients (All and Either Atopic or Non-atopic) on Total Daily Symptom Score
Time frame: Over 52 weeks (Treatment) and 20 weeks (follow-up)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
QGE031 Compared to Placebo in Non-atopic Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations
Time frame: Week 52
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.
Secondary
Response to QGE031 Between Atopic Asthma and Non-atopic Asthma
Time frame: Over 52 weeks (treatment) and 20 weeks (follow up)
Population: Due to the small number of patients randomized and limited treatment duration (not more than 22 days in QGE031 and 29 days in Placebo), the planned statistical analysis was not performed.