Efficacy and Safety of QGE031 Compared With Placebo in Patients Aged 18-75 Years With Asthma

Study Overview
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Brief Summary
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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).

Official Title
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A Multi-Center, Randomized, Double-Blind, Placebo- Controlled Study to Investigate the Efficacy and Safety of 52 Weeks Treatment With QGE031 Subcutaneous (s.c). in Asthma Patients Not Adequately Controlled by Medium- or High-dose Inhaled Corticosteroid (ICS) Plus Long Acting β2-agonist (LABA) With or Without Oral Corticosteroid (OCS)

Conditions
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Asthma

Intervention / Treatment
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* Drug: QGE031
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: QGE031 240 mg<br>QGE031 240 mg subcutaneous injection every 4 weeks | Drug: QGE031<br>* QGE031 120 mg per 1 mL liquid in vial<br>|
| Experimental: QGE031 72 mg<br>QGE031 72 mg subcutaneous injection every 4 weeks | Drug: QGE031<br>* QGE031 120 mg per 1 mL liquid in vial<br>|
| Experimental: QGE031 24 mg<br>QGE031 24 mg subcutaneous injection every 4 weeks | Drug: QGE031<br>* QGE031 120 mg per 1 mL liquid in vial<br>|
| Placebo Comparator: Placebo to QGE031<br>Placebo subcutaneous injection every 4 weeks | Drug: Placebo<br>* Placebo to QGE031 0 mg per 1 mL liquid in vial<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| QGE031 Compared to Placebo in Atopic Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations |  | Week 52 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| QGE031 Compared to Placebo in All Asthma Patients on the Reduction in Rate of Severe Asthma Exacerbations |  | Week 52 | 
| 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 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) | 
| 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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Asthma