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| ID | Type | Description | Link |
|---|---|---|---|
| 2012-002298-69 | EudraCT Number |
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This study assessed the effect on asthma control of different dose levels and regimens of QGE031 in asthma patients that are inadequately controlled with inhaled steroid and beta-2 agonist medication. Safety was assessed also. Comparison was to placebo and omalizumab. Information from this study was planned to support the design of future studies.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| QGE031 240 mg every 2 weeks (q2w) | Experimental | Participants received QGE031 240 mg subcutaneously (s.c.) q2w for 16 weeks. |
|
| QGE031 240 mg q4w | Experimental | Participants received QGE031 240 mg s.c. q4w for 16 weeks. |
|
| QGE031 180 mg q2w | Experimental | Participants received QGE031 180 mg s.c. q2w for 16 weeks. |
|
| QGE031 120 mg q2w | Experimental | Participants received QGE031 120 mg s.c. q2w for 16 weeks. |
|
| QGE031 36 mg q2w | Experimental | Participants received QGE031 36 mg s.c. q2w for 16 weeks. |
|
| QGE031 12 mg q2w | Experimental | Participants received QGE031 12 mg s.c. q2w for 16 weeks. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| QGE031 | Drug | QGE031 was supplied as 120 mg liquid in vial per 1 mL |
|
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of QGE031 Participants With Clinically Important Improvement of <= -0.5 in the Asthma Control Questionnaire 7 (ACQ-7) Score Compared to Placebo | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. | Week 16 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in ACQ-7 Score | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. A negative change from baseline indicates improvement. |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Fresno | California | 93720 | United States | ||
| Novartis Investigative Site |
The treatment arms for QGE031 and placebo were pooled into high dose QGE031 (240 mg q2w, 240 mg q4w, 180 mg q2w and 120 q2w), low dose QGE031 (36 mg q2w and 18 mg 2qw) and Placebo Total (all QGE031 placebo and Omalizumab placebo arms).
A total of 471 participants were randomized to one of the 14 treatment groups. Of these, 5 participants did not receive study treatment. Therefore, the full analysis set (FAS) and safety set included 466 participants.
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| ID | Title | Description |
|---|---|---|
| FG000 | QGE031 High Dose | Participants received QGE031 240 mg q2w, 240 mg q4w, 180 mg q2w or 120 mg q2w. |
| FG001 | QGE031 Low Dose | Participants received QGE031 36 mg q2w or 18 mg q2w. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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|
| Omalizumab (as per locally approved dosing table) | Active Comparator | Participants received omalizumab as per locally approved dosing table s.c. q2w or q4w for 16 weeks. |
|
| Placebo to QGE031 240 mg q2w | Placebo Comparator | Participants received matching placebo to QGE031 240 mg s.c. q2w for 16 weeks. |
|
| Placebo to QGE031 240 mg q4w | Placebo Comparator | Participants received placebo to QGE031 240 mg s.c. q2w for 16 weeks. |
|
| Placebo to QGE031 180 mg q2w | Placebo Comparator | Participants received QGE031 180 mg s.c. q2w for 16 weeks. |
|
| Placebo to QGE031 120 mg q2w | Placebo Comparator | Participants received QGE031 120 mg s.c. q2w for 16 weeks. |
|
| Placebo to QGE031 36 mg q2w | Placebo Comparator | Participants received QGE031 36 mg s.c. q2w for 16 weeks. |
|
| Placebo to QGE031 12 mg q2w | Placebo Comparator | Participants received QGE031 12 mg s.c. q2w for 16 weeks. |
|
| Placebo to omalizumab | Placebo Comparator | Participants received placebo to omalizumab s.c. q2w or q4w for 16 weeks. |
|
| Omalizumab | Drug | Omalizumab was supplied as 150 mg lyophilisate in vial |
|
|
| Placebo | Drug | Placebo to QGE031 and Omalizumab was supplied as QGE031 0 mg in vial per 1 mL |
|
| Baseline, Weeks 4, 8, 12, 16 and 28 |
| Percentage of Participants With a Change From Baseline in ACQ-7 Score Less Than -1.1 | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. | Week 16 |
| Change From Baseline in Asthma Quality of Life Questionnaire (AQLQ) Score | The AQLQ is a 32-item disease specific questionnaire designed to measure functional impairments that are most important to participants with asthma. The 32 items in the AQLQ were divided into four domain-specific scores and a total score as follows: Activity limitations = Mean of Items 1, 2, 3, 4, 5, 11, 19, 25, 28, 31, 32 (11 items); Symptoms = Mean of Items 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, 29, 30 (12 items); Emotional function = Mean of Items 7, 13, 15, 21, 27 (5 items); Environmental stimuli = Mean of Items 9, 17, 23, 26 (4 items); and Overall Score = Mean of Items 1 to 32 (32 items). Each item of the AQLQ was equally weighted and scored along a 7-point scale, where 1 indicates maximal impairment and 7 indicates no impairment. Thus, higher scores indicate better asthma-related quality of life. The mean overall score ranged from 1 to 7. A positive change from baseline indicates improvement. | Baseline, Week 16, Week 28 |
| Change From Baseline in Mean Number of Puffs of Morning, Evening and Total Daily Asthma Rescue Medication | Participants recorded their use of rescue medication into an electronic diary (eDiary). A negative change from baseline indicates improvement. | Baseline, Week 16 |
| Hawaiian Gardens |
| California |
| 90716 |
| United States |
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| FG002 | Omalizumab | Participants received omalizumab as per locally approved dosing table q2w or q4w. |
| FG003 | Placebo Total | Participants received matching placebo to QGE031 or Omalizumab. |
| FAS |
|
| Safety Set |
|
| COMPLETED |
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| NOT COMPLETED |
|
|
Baseline characteristics were based on the FAS.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | QGE031 High Dose | Participants received QGE031 240 mg q2w, 240 mg q4w, 180 mg q2w or 120 mg q2w. |
| BG001 | QGE031 Low Dose | Participants received QGE031 36 mg q2w or 18 mg q2w. |
| BG002 | Omalizumab | Participants received omalizumab as per locally approved dosing table q2w or q4w. |
| BG003 | Placebo Total | Participants received matching placebo to QGE031 or Omalizumab. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage of QGE031 Participants With Clinically Important Improvement of <= -0.5 in the Asthma Control Questionnaire 7 (ACQ-7) Score Compared to Placebo | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. | The full analysis set (FAS) for the QGE031 240 mg q2w, placebo to QGE031 240 mg q2w and Omalizumab groups (n=120,49,131) was considered for the analysis. Only participants who had week 16 values were analyzed. The FAS included randomized participants who received at least one dose of study drug. | Posted | Number | Percentage of participants | Week 16 |
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| Secondary | Change From Baseline in ACQ-7 Score | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. A negative change from baseline indicates improvement. | The FAS for the QGE031 240 mg q2w, placebo to QGE031 240 mg q2w and Omalizumab groups (n=120,49,131) was considered for the analysis. Participants who had values at both baseline and the post baseline time point were analyzed for that post baseline time point. The FAS included randomized participants who received at least one dose of study drug. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Weeks 4, 8, 12, 16 and 28 |
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| Secondary | Percentage of Participants With a Change From Baseline in ACQ-7 Score Less Than -1.1 | The ACQ-7 measures asthma symptom control and consisted of 7 items: 5 on symptom assessment, 1 on rescue bronchodilator use and 1 on airway caliber (FEV1 % predicted). All 7 questions of the ACQ were equally weighted. Items 1-6 scored along a 7-point response scale, where 0 = good controlled and 6 = poor controlled. The 7th item on % predicted FEV1 (pre-bronchodilator) was scored by clinic staff on a 7-point scale (0 - > 95%; 1 - 90-95%; 2 - 80-89%; 3 - 70-79%; 4 - 60-69%; 5 - 50-59%; 6 - < 50%). The average score of the 7 questions was calculated as the sum of scores divided by the number of questions that were answered by the participants, as long as there were at least 6 questions answered and the missing items were neither question 1 nor question 7. | The full analysis set (FAS) for the QGE031 240 mg q2w, placebo to QGE031 240 mg q2w and Omalizumab groups (n=120,49,131) was considered for the analysis. Only participants who had week 16 values were analyzed. The FAS included randomized participants who received at least one dose of study drug. | Posted | Number | Percentage of participants | Week 16 |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Asthma Quality of Life Questionnaire (AQLQ) Score | The AQLQ is a 32-item disease specific questionnaire designed to measure functional impairments that are most important to participants with asthma. The 32 items in the AQLQ were divided into four domain-specific scores and a total score as follows: Activity limitations = Mean of Items 1, 2, 3, 4, 5, 11, 19, 25, 28, 31, 32 (11 items); Symptoms = Mean of Items 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, 29, 30 (12 items); Emotional function = Mean of Items 7, 13, 15, 21, 27 (5 items); Environmental stimuli = Mean of Items 9, 17, 23, 26 (4 items); and Overall Score = Mean of Items 1 to 32 (32 items). Each item of the AQLQ was equally weighted and scored along a 7-point scale, where 1 indicates maximal impairment and 7 indicates no impairment. Thus, higher scores indicate better asthma-related quality of life. The mean overall score ranged from 1 to 7. A positive change from baseline indicates improvement. | The FAS for the QGE031 240 mg q2w, placebo to QGE031 240 mg q2w and Omalizumab groups (n=120,49,131) was considered for the analysis. Participants who had values at both baseline and the post baseline time point were analyzed for that post baseline time point. The FAS included randomized participants who received at least one dose of study drug. | Posted | Mean | Standard Deviation | Score on a scale | Baseline, Week 16, Week 28 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Mean Number of Puffs of Morning, Evening and Total Daily Asthma Rescue Medication | Participants recorded their use of rescue medication into an electronic diary (eDiary). A negative change from baseline indicates improvement. | The full analysis set (FAS) for the QGE031 240 mg q2w, placebo to QGE031 240 mg q2w and Omalizumab groups (n=120,49,131) was considered for the analysis. Only participants who had both baseline and week 16 values were analyzed. The FAS included randomized participants who received at least one dose of study drug. | Posted | Least Squares Mean | Standard Error | Number of puffs | Baseline, Week 16 |
|
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | QGE031 High Dose | Participants received QGE031 240 mg q2w, 240 mg q4w, 180 mg q2w or 120 mg q2w. | 9 | 199 | 91 | 199 | ||
| EG001 | QGE031 Low Dose | Participants received QGE031 36 mg q2w or 18 mg q2w. | 3 | 40 | 17 | 40 | ||
| EG002 | Omalizumab | Participants received omalizumab as per locally approved dosing table q2w or q4w. | 1 | 131 | 43 | 131 | ||
| EG003 | Placebo Total | Participants received matching placebo to QGE031 or Omalizumab | 5 | 96 | 35 | 96 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Haemorrhagic anaemia | Blood and lymphatic system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Coronary artery disease | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Ventricular arrhythmia | Cardiac disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Non-cardiac chest pain | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Drug hypersensitivity | Immune system disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Vaginal laceration | Injury, poisoning and procedural complications | MedDRA (18.0) | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA (18.0) | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA (18.0) | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA (18.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Renal colic | Renal and urinary disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Laryngeal dysplasia | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (18.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Injection site reaction | General disorders | MedDRA (18.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA (18.0) | Systematic Assessment |
| |
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (18.0) | Systematic Assessment |
|
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Novartis Pharmaceuticals | 862-778-8300 |
| ID | Term |
|---|---|
| D001249 | Asthma |
| D006967 | Hypersensitivity |
| ID | Term |
|---|---|
| D001982 | Bronchial Diseases |
| D012140 | Respiratory Tract Diseases |
| D008173 | Lung Diseases, Obstructive |
| D008171 | Lung Diseases |
| D012130 | Respiratory Hypersensitivity |
| D006969 | Hypersensitivity, Immediate |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000598891 | ligelizumab |
| D000069444 | Omalizumab |
| ID | Term |
|---|---|
| D000888 | Antibodies, Anti-Idiotypic |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D012712 | Serum Globulins |
| D005916 | Globulins |
Not provided
Not provided
| Male |
|
| Odds Ratio (OR) |
| 0.84 |
| 2-Sided |
| 95 |
| 0.46 |
| 1.52 |
| Superiority or Other |
| OG002 | Omalizumab | Participants received omalizumab as per locally approved dosing table q2w or q4w. |
|
|
| OG002 |
| Omalizumab |
Participants received omalizumab as per locally approved dosing table q2w or q4w. |
|
|
|
Participants received placebo to QGE031 240 mg q2w |
| OG002 | Omalizumab | Participants received omalizumab as per locally approved dosing table q2w or q4w. |
|
|
| Counts |
|---|
| Participants |
|
|
|