Trial Outcomes & Findings for Study of Efficacy and Safety of QAW039 When Added to Standard-of-care Asthma Therapy in Patients With Uncontrolled Asthma (NCT NCT03226392)

Last Updated: 2026-01-13

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

704 participants

Primary outcome timeframe

Baseline and Week 12

Results posted on

2026-01-13

Participant Flow

Participants were recruited from centers in Brazil (8), Bulgaria (4), Canada (3), Colombia (4), Germany (5), Hungary (7), India (5), Israel (5), Italy (4), Peru (6), Republic of Korea (5), Russian Federation (14), Spain (8), United States (36), Vietnam (3).

Participant milestones

Participant milestones
Measure	QAW039 QAW039 150mg once daily	Placebo Placebo once daily
Overall Study STARTED	352	352
Overall Study FAS	352	350
Overall Study COMPLETED	341	344
Overall Study NOT COMPLETED	11	8

Reasons for withdrawal

Reasons for withdrawal
Measure	QAW039 QAW039 150mg once daily	Placebo Placebo once daily
Overall Study Protocol Deviation	1	0
Overall Study Physician Decision	1	0
Overall Study Lost to Follow-up	1	0
Overall Study Adverse Event	1	0
Overall Study Technical Problems	0	2
Overall Study Subject/Guardian Decision	7	6

Baseline Characteristics

Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	QAW039 n=352 Participants QAW039 150mg once daily	Placebo n=350 Participants Placebo once daily	Total n=702 Participants Total of all reporting groups
Age, Continuous	50.4 Years STANDARD_DEVIATION 14.87 • n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	50.2 Years STANDARD_DEVIATION 14.39 • n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	50.3 Years STANDARD_DEVIATION 14.62 • n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Sex: Female, Male Female	216 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	218 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	434 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Sex: Female, Male Male	136 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	132 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	268 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Black	26 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	18 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	44 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Asian	41 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	46 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	87 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Native American	12 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	12 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	24 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Pacific Islander	2 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	0 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	2 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Other	55 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	58 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	113 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"
Race/Ethnicity, Customized Caucasian	216 Participants n=210 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	216 Participants n=19 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"	432 Participants n=123 Participants • Two patients in the placebo group were randomized in error and did not receive any study drug; for these patients the reason for discontinuation is given as "technical problems"

PRIMARY outcome

Timeframe: Baseline and Week 12

Population: Full Analysis Set (FAS): all randomized patients who received at least one dose of study medication. Patients in the FAS were analyzed according to the treatment they were assigned to at randomization.

Outcome measures

Outcome measures
Measure	QAW039 n=352 Participants QAW039 150mg once daily	Placebo n=350 Participants Placebo once daily
Change From Baseline in Pre-dose FEV1	0.126 Liters Standard Error 0.00177	0.157 Liters Standard Error 0.0177

SECONDARY outcome

Timeframe: Baseline and Week 12

Daytime asthma symptoms are evaluated through four questions and each of them will be rated on a scale of 0 to 6. Higher scores indicate more severe asthma-related symptoms. A mean score is calculated for the responses to 4 questions.

Outcome measures

Outcome measures
Measure	QAW039 n=352 Participants QAW039 150mg once daily	Placebo n=350 Participants Placebo once daily
Change From Baseline in Daytime Asthma Symptom Score	-0.55 Score Standard Error 0.034	-0.45 Score Standard Error 0.034

SECONDARY outcome

Timeframe: Baseline (daily mean for up to three weeks prior to baseline visit) and Week 12 (daily mean post baseline up to Week 12)

Daily use of SABA (the number of rescue medication puffs taken in the previous 12 hours) was recorded using a patient electronic diary (referred to as eDiary or eDiary/ePEF). Patients were instructed to routinely complete the patient diary twice daily - at the same time each morning and each evening, approximately 12 hours apart.

Outcome measures

Outcome measures
Measure	QAW039 n=352 Participants QAW039 150mg once daily	Placebo n=350 Participants Placebo once daily
Change From Baseline in Number of Puffs of SABA Taken Per Day	-0.89 Puffs per day Standard Error 0.066	-0.88 Puffs per day Standard Error 0.066

SECONDARY outcome

Timeframe: Baseline and Week 12

AQLQ is a 32-item instrument administered as a self-assessment. AQLQ+12 is a modified version of AQLQ developed to measure functional impairments of participants aged 12-70 years. It is divided into 4 domains: activity limitation, symptoms, emotional function, and environmental stimuli. Participants were asked to recall their experiences during the last 2 weeks and respond to each question on a 7-point scale (1=severe impairment, 7=no impairment), where higher scores indicated "better quality of life." Overall AQLQ+12 score is the mean of all 32 responses.

Outcome measures

Outcome measures
Measure	QAW039 n=352 Participants QAW039 150mg once daily	Placebo n=350 Participants Placebo once daily
Change From Baseline in Asthma Quality of Life Questionnaire for 12 Years and Older (AQLQ+12) Score	0.77 units on a scale Standard Error 0.043	0.72 units on a scale Standard Error 0.043

Adverse Events

QAW039 150 mg

Serious events: 7 serious events

Other events: 94 other events

Deaths: 0 deaths

Placebo

Serious events: 3 serious events

Other events: 116 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	QAW039 150 mg n=352 participants at risk QAW039 150 mg	Placebo n=350 participants at risk Placebo
Cardiac disorders Acute myocardial infarction	0.00% 0/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.29% 1/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Gastrointestinal disorders Upper gastrointestinal haemorrhage	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
General disorders Non-cardiac chest pain	0.00% 0/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.29% 1/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Hepatobiliary disorders Cholecystitis acute	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Cellulitis	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Urinary tract infection	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Injury, poisoning and procedural complications Femur fracture	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Injury, poisoning and procedural complications Hip fracture	0.00% 0/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.29% 1/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Prostate cancer	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Asthma	0.85% 3/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.00% 0/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.

Other adverse events

Additional Information

Study Director

Novartis pharmaceutical

Phone: 862-778-8300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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 (ie, data from all sites) in the clinical trial.
Publication restrictions are in place

Restriction type: OTHER

Measure	QAW039 150 mg n=352 participants at risk QAW039 150 mg	Placebo n=350 participants at risk Placebo
Infections and infestations Bronchitis	2.6% 9/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.7% 6/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Influenza	1.1% 4/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.29% 1/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Nasopharyngitis	2.8% 10/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	6.9% 24/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Pharyngitis	1.4% 5/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.57% 2/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Respiratory tract infection viral	1.4% 5/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	0.29% 1/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Upper respiratory tract infection	2.0% 7/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	2.9% 10/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Upper respiratory tract infection bacterial	0.85% 3/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.7% 6/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Infections and infestations Viral upper respiratory tract infection	3.4% 12/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	2.3% 8/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Injury, poisoning and procedural complications Contusion	0.00% 0/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.4% 5/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Musculoskeletal and connective tissue disorders Back pain	2.0% 7/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	2.0% 7/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Nervous system disorders Headache	1.4% 5/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	4.0% 14/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Asthma	13.1% 46/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	17.4% 61/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Cough	0.57% 2/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.4% 5/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.85% 3/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.4% 5/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	1.7% 6/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.1% 4/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	0.85% 3/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.4% 5/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.
Respiratory, thoracic and mediastinal disorders Wheezing	0.28% 1/352 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.	1.1% 4/350 • After signing informed consent to 30 days after last dose, assessed up to 16 weeks.