This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Efficacy and Safety Study of QAW039 in Patients With Severe Asthma Inadequately Controlled With Standard of Care Asthma Treatment. (NCT02555683), a randomized phase 3 study. The primary outcome is Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02555683 |
| Phase | Phase 3 |
| Condition | Asthma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 894 |
| Arms in the primary outcome | 3 |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The primary analysis concerned participants in the Full Analysis Set with inadequately controlled severe asthma and a high blood eosinophil count at baseline: at least 250 cells/μL. The overall study population also included patients with inadequately controlled severe asthma. Inadequate control was defined as partly controlled or uncontrolled asthma under GINA 2016 criteria.
Intervention
Participants received QAW039 150 mg once daily or QAW039 450 mg once daily, added to existing asthma therapy. The study assessed these doses alongside GINA steps 4 and 5 standard-of-care asthma treatment.
Comparator
The comparator was placebo once daily, added to existing asthma therapy. The registry lists comparisons of each QAW039 dose with placebo.
Primary question
During the 52-week treatment period, did either QAW039 dose differ from placebo in the rate of moderate-to-severe asthma exacerbations in the high-eosinophil subpopulation? The registry reports annualized rate estimates and rate ratios with confidence intervals and p-values. The estimates describe the posted analyses; they do not by themselves establish that a treatment works or does not work.
3. Trial Design
This was a phase 3, randomized, double-blind, parallel-group, placebo-controlled study of QAW039 in patients with uncontrolled severe asthma. The official title describes a 52-week, multicenter study assessing QAW039 when added to existing asthma therapy. The registry lists three once-daily arms: QAW039 150 mg, QAW039 450 mg, and placebo. The study was completed, with enrollment recorded as 894.
The stated purpose was to assess efficacy and safety in two populations: participants with inadequately controlled severe asthma and high eosinophil counts at baseline, and the overall study population with inadequately controlled severe asthma. The high-eosinophil subgroup was defined by a baseline blood eosinophil count of at least 250 cells/μL. Inadequate control was defined as partly controlled or uncontrolled asthma according to GINA 2016. Treatment was added to GINA steps 4 and 5 standard-of-care asthma therapy.
The primary endpoint focused on moderate-to-severe asthma exacerbations over the 52-week treatment period in the high-eosinophil subpopulation. Secondary endpoints included asthma quality of life, asthma control, and pre-dose FEV1 changes at Week 52, assessed in the high-eosinophil and overall populations. The registry record lists 8 outcome measures and 16 analyses, and identifies 6 secondary outcomes as posted. Detailed eligibility criteria, the allocation ratio, the method used to generate the randomization sequence, and procedures for maintaining masking are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: The study included a Screening period of up to 2 weeks and a Placebo Run-in period of 2 to 6 weeks, during which eligibility for randomization was determined.
Overall Study
| Stage | QAW039 150 mg | QAW039 450 mg | Placebo |
|---|---|---|---|
| STARTED | 301 | 295 | 298 |
| FAS/SAF: High eosinophil subpopulation | 200 | 201 | 201 |
| FAS/SAF: Overall population | 299 | 293 | 298 |
| COMPLETED | 282 | 278 | 269 |
| NOT COMPLETED | 19 | 17 | 29 |
Reasons for not completing this period:
| Reason | QAW039 150 mg | QAW039 450 mg | Placebo |
|---|---|---|---|
| Physician Decision | 1 | 0 | 2 |
| Lost to Follow-up | 2 | 1 | 2 |
| Technical Problems | 1 | 1 | 0 |
| Protocol deviation | 0 | 1 | 0 |
| Lack of Efficacy | 2 | 0 | 0 |
| Death | 0 | 1 | 2 |
| Adverse Event | 1 | 0 | 1 |
| Subject/guardian decision | 12 | 12 | 22 |
| Death post Treatment/safety period | 0 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Full Analysis Set (FAS) in high eosinophils subpopulation (count ≥ 250 cells/μL)
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | QAW039 150 mg | QAW039 450 mg | Placebo | Total |
|---|---|---|---|---|---|
| Participants analysed | 301 | 295 | 298 | 894 | |
| Age, Continuous | years | 49.9 (SD 14.97) | 51.1 (SD 14.29) | 50.3 (SD 14.48) | 50.4 (SD 14.58) |
| Sex: Female, Male: Female | Participants | 192 | 214 | 177 | 583 |
| Sex: Female, Male: Male | Participants | 109 | 81 | 121 | 311 |
| Race/Ethnicity, Customized: Caucasian | Participants | 214 | 215 | 213 | 642 |
| Race/Ethnicity, Customized: Black | Participants | 11 | 6 | 12 | 29 |
| Race/Ethnicity, Customized: Asian | Participants | 45 | 38 | 44 | 127 |
| Race/Ethnicity, Customized: Native American | Participants | 24 | 29 | 24 | 77 |
| Race/Ethnicity, Customized: Unknown | Participants | 1 | 2 | 3 | 6 |
| Race/Ethnicity, Customized: Other | Participants | 6 | 5 | 2 | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Rate of Moderate-to-severe Asthma Exacerbations During the 52-week Treatment Period in High Eosinophils Subpopulation, measured over Baseline, Week 52.
| Element | Registry entry |
|---|---|
| Result type | Least Squares Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | events/year |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the rate of moderate-to-severe asthma exacerbations during the 52-week treatment period in the high-eosinophil subpopulation. It is reported as a least-squares mean rate in events per year, with a 95% confidence interval, and as a rate ratio comparing each QAW039 dose with placebo. An annualized rate expresses the event frequency relative to time, allowing a rate-based comparison over the treatment period.
The registry defines a severe exacerbation as rescue systemic corticosteroid treatment for at least 3 days together with hospitalization, or with an emergency department visit lasting more than 24 hours, or death due to asthma. A moderate exacerbation is defined as rescue systemic corticosteroid treatment for at least 3 days, either as an outpatient or during an emergency department visit lasting no more than 24 hours. The high-eosinophil population consists of patients with a baseline blood eosinophil count of at least 250 cells/μL.
Exacerbation rates are useful because they summarize recurrent episodes over a treatment period rather than only whether a participant had any episode. The rate ratio expresses the estimated rate in one treatment group relative to placebo; a value of 1 represents equal estimated rates. The registry reports 95% confidence intervals to convey uncertainty around each comparison. The endpoint definition and analysis apply to the specified subgroup, so the primary result should not be treated as an estimate for every patient with severe asthma.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
In the high-eosinophil subpopulation, the reported least-squares mean exacerbation rate was 0.97 events/year for QAW039 150 mg, with a 95% confidence interval of 0.78 to 1.2. For QAW039 450 mg, it was 0.77 events/year, with a 95% confidence interval of 0.62 to 0.97. The placebo estimate was 0.93 events/year, with a 95% confidence interval of 0.75 to 1.16. These are model-based group estimates; their confidence intervals describe uncertainty in the estimates, not the range of individual participants' outcomes.
The reported negative binomial regression comparison for QAW039 150 mg versus placebo gave a rate ratio of 1.04, with a 95% confidence interval of 0.77 to 1.41 and p=0.800. For QAW039 450 mg versus placebo, the reported rate ratio was 0.83, with a 95% confidence interval of 0.61 to 1.14 and p=0.510. Both confidence intervals include 1, the value corresponding to equal rates. The posted comparisons therefore do not provide clear statistical evidence of a difference from placebo at the conventional 0.05 significance level. That is not proof that the rates are identical: the intervals include values on either side of 1 and reflect uncertainty in both directions.
The point estimates differ in direction: the 150 mg estimate is slightly above the placebo estimate, while the 450 mg estimate is below it. A point estimate alone does not establish a reliable treatment difference. Interpretation rests on the comparison estimate together with its uncertainty and the reported analysis. The registry identifies superiority comparisons, but the statistical plan and any multiplicity procedure are not reported in the registry record.
The secondary outcomes add context by assessing quality of life, asthma control, and lung function, but they do not replace the primary exacerbation endpoint. Several posted secondary comparisons have confidence intervals that include no difference, and their unadjusted re-calculations are exploratory rather than substitutes for the sponsor's analyses. The number of endpoints and comparisons makes isolated secondary p-values especially difficult to interpret without a reported multiplicity strategy. Taken together, the posted results should be read as estimates with uncertainty, not as definitive proof for or against an effect.
9. Statistical Methods Explained
The primary endpoint is a count-based rate: moderate or severe exacerbations can occur more than once during follow-up. The registry reports least-squares mean rates and compares the treatment groups with placebo using negative binomial regression. This approach is commonly used for count outcomes when the variability is greater than would be expected under a simple Poisson model. General statistical context: extra variation can arise when event counts differ substantially across participants, and a model that allows over-dispersion can avoid treating the data as more precise than they are. The registry record does not report model diagnostics, the covariates included, or the handling of exposure time in detail.
A rate ratio compares estimated event rates between groups. A ratio of 1 corresponds to equal rates; a ratio below 1 indicates a lower estimated rate in the numerator group, while a ratio above 1 indicates a higher estimated rate. The primary reported 95% confidence intervals include 1 for both dose comparisons. In general, a confidence interval gives a range of values compatible with the observed data under the analysis assumptions; it is not a guarantee that the true effect lies within that particular interval. The p-value evaluates the data against a specified null hypothesis under the model, rather than measuring the probability that the null hypothesis is true.
The secondary measures are continuous outcomes. For these, the registry reports least-squares mean changes from baseline and standard errors. A least-squares mean is a model-based adjusted mean; it need not equal a simple arithmetic average. A standard error describes the estimated precision of a mean, rather than the spread of individual observations. The posted sponsor comparisons use ANCOVA and report mean differences (net), confidence intervals, and p-values. ANCOVA is a regression-based method that can compare outcomes while accounting for specified covariates, but the covariates and detailed model specification are not reported in the registry record.
Separate from the sponsor-reported analyses, unadjusted re-calculations are available for the continuous secondary endpoints. These use posted summary statistics to estimate treatment-versus-placebo mean differences and confidence intervals. For some calculations, the standard deviation is approximated from the reported standard error and group size; that makes the result a simple summary-statistic re-calculation, not a reconstruction of the sponsor's ANCOVA. The re-calculations do not include model covariates or any adjustment for multiple comparisons. Where they differ from the sponsor's reported analysis, the sponsor's own analysis takes precedence; the registry record does not identify a discrepancy for this trial.
Planning illustrations relate hypothetical sample sizes to an assumed effect drawn from the observed effect. They are not observed power and do not show that the trial was or was not adequately powered. Their values depend on the stated planning assumptions, including two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. The illustrated effects are fractions of the observed effect, and the resulting sample sizes are planning scenarios rather than observed trial results. A sample-size justification for the actual study is not reported in the registry record.
Statistical significance and clinical importance are distinct. A p-value reflects compatibility with a null model under assumptions; it does not determine whether an estimated difference matters to patients. A confidence interval helps communicate both direction and precision, but clinical interpretation also requires context about the outcome and a meaningful difference threshold. A threshold for meaningful change is not reported in the registry record. Likewise, the posted analyses do not establish that every planned analysis was completed or explain how missing outcomes were handled.
The registry identifies the primary analysis population as the Full Analysis Set in the high-eosinophil subpopulation. The meaning and inclusion rules for the Full Analysis Set are not reported in the registry record. This matters because analysis populations can differ according to eligibility, treatment received, and availability of outcomes. Without further details, the reported population label should be retained rather than assigning it a more specific definition.
10. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| negative binomial regression model | rate ratio 1.04 | 0.77 to 1.41 | 0.800 |
| negative binomial regression model | Rate Ratio 0.83 | 0.61 to 1.14 | 0.510 |
The sponsor-reported primary comparisons use negative binomial regression and present rate ratios with 95% confidence intervals and p-values. For QAW039 150 mg versus placebo, the rate ratio was 1.04 (95% CI 0.77 to 1.41; p=0.800). For QAW039 450 mg versus placebo, it was 0.83 (95% CI 0.61 to 1.14; p=0.510). These are the registry's reported inferential results and should be the principal basis for interpreting the primary endpoint.
For secondary continuous outcomes, the sponsor reports ANCOVA comparisons, while the simple re-calculations use posted summary means and standard errors. Such re-calculations can differ because they do not reproduce the sponsor's model adjustments and may use approximations. The sponsor's reported analyses take precedence if a difference arises; no discrepancy is identified in the registry record. The re-calculated estimates are useful as transparent, unadjusted checks of the posted summaries, not as replacements for the sponsor's analyses. The registry record does not provide enough detail to reconstruct the full analysis models or determine every reason for any numerical difference between a re-calculation and a reported result.
11. Multiplicity, Missing Data and Analysis Populations
The registry record lists multiple outcomes, populations, and dose-versus-placebo comparisons. It reports one primary endpoint in the high-eosinophil subpopulation, along with secondary outcomes covering quality of life, asthma control, and FEV1 in that subgroup and in the overall population. A prespecified testing hierarchy, family-wise error procedure, or other multiplicity adjustment is not reported in the registry record. Consequently, secondary p-values should not be interpreted as though they were necessarily protected against the increased chance of a false-positive finding when many comparisons are made.
Missing-data handling is not reported in the registry record. The record does not describe how participants with incomplete follow-up, unavailable Week 52 measures, or incomplete exacerbation observation were treated in the analyses. It also does not report sensitivity analyses for missing outcomes or the assumptions used to address missingness. Those details can affect the interpretation of estimates, especially when outcomes are measured over time.
The primary population is identified as the Full Analysis Set in the high-eosinophil subpopulation, but the precise Full Analysis Set definition is not reported in the registry record. The record also does not describe whether all randomized participants were included in each analysis, how subgroup membership was confirmed for analysis, or whether any participants were excluded after randomization.
Interim analyses and stopping rules are not reported in the registry record. A statistical analysis plan, the primary endpoint's sample-size justification, model covariates, and detailed model diagnostics are also not reported. These absences do not demonstrate that such procedures were not used; they mean the registry record does not permit their assessment. The reported estimates can be interpreted on their stated terms, while conclusions about multiplicity control, missingness, and analysis conduct remain limited by the available registry detail.
12. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| QAW039 150 mg | 33 | 299 | 11.0% |
| QAW039 450 mg | 30 | 293 | 10.2% |
| Placebo | 32 | 298 | 10.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| QAW039 150 mg | 0 of 299 |
| QAW039 450 mg | 1 of 293 |
| Placebo | 2 of 298 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| QAW039 150 mg | 204 of 299 |
| QAW039 450 mg | 193 of 293 |
| Placebo | 214 of 298 |
Most frequent serious adverse event terms
| Event term | Organ system | QAW039 150 mg | QAW039 450 mg | Placebo |
|---|---|---|---|---|
| Asthma | Respiratory, thoracic and mediastinal disorders | 10 of 299 | 8 of 293 | 14 of 298 |
| Pneumonia | Infections and infestations | 3 of 299 | 5 of 293 | 1 of 298 |
| Influenza | Infections and infestations | 1 of 299 | 2 of 293 | 1 of 298 |
| Acute kidney injury | Renal and urinary disorders | 1 of 299 | 2 of 293 | 0 of 298 |
| Coronary artery disease | Cardiac disorders | 1 of 299 | 0 of 293 | 1 of 298 |
| Gastroenteritis | Infections and infestations | 1 of 299 | 0 of 293 | 1 of 298 |
| Viral upper respiratory tract infection | Infections and infestations | 2 of 299 | 0 of 293 | 0 of 298 |
| Radius fracture | Injury, poisoning and procedural complications | 2 of 299 | 0 of 293 | 0 of 298 |
| Uterine leiomyoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 299 | 0 of 293 | 1 of 298 |
| Headache | Nervous system disorders | 1 of 299 | 0 of 293 | 1 of 298 |
Most frequent non-serious adverse event terms
| Event term | Organ system | QAW039 150 mg | QAW039 450 mg | Placebo |
|---|---|---|---|---|
| Asthma | Respiratory, thoracic and mediastinal disorders | 151 of 299 | 135 of 293 | 155 of 298 |
| Nasopharyngitis | Infections and infestations | 43 of 299 | 37 of 293 | 49 of 298 |
| Upper respiratory tract infection | Infections and infestations | 35 of 299 | 35 of 293 | 31 of 298 |
| Viral upper respiratory tract infection | Infections and infestations | 29 of 299 | 26 of 293 | 26 of 298 |
| Bronchitis | Infections and infestations | 29 of 299 | 22 of 293 | 26 of 298 |
| Headache | Nervous system disorders | 28 of 299 | 22 of 293 | 24 of 298 |
| Sinusitis | Infections and infestations | 18 of 299 | 19 of 293 | 18 of 298 |
| Upper respiratory tract infection bacterial | Infections and infestations | 13 of 299 | 14 of 293 | 21 of 298 |
| Urinary tract infection | Infections and infestations | 11 of 299 | 15 of 293 | 18 of 298 |
| Back pain | Musculoskeletal and connective tissue disorders | 16 of 299 | 16 of 293 | 11 of 298 |
Serious events with the highest arm-level rate: asthma (4.7%), pneumonia (1.7%), influenza (0.7%), acute kidney injury (0.7%), viral upper respiratory tract infection (0.7%).
The registry reports serious adverse events and other adverse events for each treatment arm. Serious adverse events affected 33 of 299 participants in the QAW039 150 mg group, 30 of 293 in the QAW039 450 mg group, and 32 of 298 in the placebo group. Other adverse events affected 204 of 299, 193 of 293, and 214 of 298 participants, respectively. These are descriptive counts and denominators, not adjusted comparisons of risk.
Deaths recorded were 0 in the QAW039 150 mg group, 1 in the QAW039 450 mg group, and 2 in the placebo group. The registry lists asthma as the most frequent serious adverse event term at 4.7%, followed by pneumonia at 1.7%; influenza, acute kidney injury, viral upper respiratory tract infection, and radius fracture are each listed at 0.7%. The record does not establish that an adverse event was caused by study treatment, and the listed terms should not be interpreted as causal findings.
Safety counts are best read alongside the trial's definitions, follow-up, and participant exposure. Those detailed definitions and exposure-adjusted comparisons are not reported in the registry record. The counts alone cannot establish comparative safety or rule out uncommon harms.
13. What the Result Does, and Does Not, Mean
The primary result is an estimate of exacerbation rates during the 52-week treatment period among participants with high baseline eosinophil counts. The sponsor-reported rate ratios compare each QAW039 dose with placebo, and both 95% confidence intervals include 1. The estimates therefore leave uncertainty about whether the underlying rates differ and in which direction. A non-significant result is not evidence that the treatments and placebo are equivalent; demonstrating equivalence would require a prespecified equivalence margin and an analysis designed for that purpose, neither of which is reported in the registry record.
Nor does the result establish that QAW039 has no effect. The confidence intervals include a range of possible rate ratios, including values below and above 1. The p-values describe the reported model-based comparisons under the null hypothesis; they are not probabilities that a treatment is effective or ineffective. The outcome is also restricted to the high-eosinophil subgroup, so these estimates should not be generalized automatically to the overall population or to patients outside the study definition.
The secondary measures add information about quality of life, asthma control, and lung function, but they cannot independently settle the primary question. The registry does not report a multiplicity strategy, detailed missing-data methods, or thresholds for clinically meaningful change. Safety counts describe events recorded in the treatment groups but do not establish causality or comparative risk by themselves.
Overall, the posted data describe model-based estimates and their uncertainty rather than a definitive answer about treatment benefit or harm. Interpretation should remain specific to the studied population, doses, background therapy, endpoints, and follow-up. The registry record does not report enough methodological detail to resolve every question about the analysis or its assumptions.
14. Why This Trial Matters Statistically
This trial illustrates why event-rate outcomes require more than a comparison of group averages. Moderate-to-severe exacerbations can recur, so the primary measure accounts for event frequency over the treatment period. A rate ratio gives a direct contrast between groups, while its confidence interval shows how imprecisely that contrast is estimated. Here, both reported intervals include the value corresponding to equal rates, so the point estimates alone should not drive the conclusion.
It also demonstrates the importance of keeping the target population explicit. The primary endpoint applies to the high-eosinophil subpopulation, defined by a baseline count of at least 250 cells/μL. Results for that group are not automatically results for the overall study population. The presence of multiple secondary outcomes and populations further illustrates why a clear analysis hierarchy and multiplicity plan matter; those procedures are not reported in the registry record.
Finally, the study is a useful example of distinguishing sponsor-reported model-based results from simple unadjusted re-calculations. Summary statistics can support transparent checks and planning illustrations, but they do not recreate an adjusted analysis. Re-calculation-based sample sizes are planning scenarios, not observed power or evidence that a trial was adequately powered. Careful interpretation depends on respecting those distinctions, attending to uncertainty, and avoiding causal or clinical claims that the posted estimates do not establish.
15. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The registry record gives limited detail about the statistical analysis. It does not report the covariates in the negative binomial or ANCOVA models, model diagnostics, the approach to missing data, or sensitivity analyses. The precise Full Analysis Set definition is also not reported. These omissions make it difficult to assess the robustness of the estimates or reproduce the sponsor's analyses from the registry entry alone.
Multiplicity procedures are not reported despite several outcomes, populations, and comparisons. Without a stated hierarchy or adjustment, secondary p-values should be treated cautiously. The registry record also does not report a sample-size justification for the actual study, interim analyses, stopping rules, or a threshold for clinically important change. The planning illustrations use observed effects and specified assumptions; they do not establish the trial's original design assumptions or observed power.
The secondary re-calculations use posted summary statistics and are unadjusted. In some cases, variability is approximated from standard errors, so the calculations do not recover the full participant-level models. They are not a substitute for the sponsor's ANCOVA analyses. The registry record does not provide enough detail to evaluate the impact of departures from the model assumptions or to compare results under alternative analysis choices.
Safety results are also limited to descriptive counts, denominators, deaths, and selected serious adverse event terms. The registry entry does not provide causal attribution or exposure-adjusted comparisons in the recorded safety details. These limitations constrain what can be concluded from the registry results; they do not show that unreported analyses or procedures were not performed.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Efficacy and Safety Study of QAW039 in Patients With Severe Asthma Inadequately Controlled With Standard of Care Asthma Treatment. — NCT02555683.
- Registry record last updated 2026-10-07; status last verified 2020-04.
- Results first posted 2020-05-18.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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19. Record Summary
NCT02555683 was a completed phase 3 randomized, double-blind, parallel-group, placebo-controlled study of once-daily QAW039 150 mg or 450 mg added to existing asthma therapy, compared with placebo. Enrollment was recorded as 894. The primary endpoint was the rate of moderate-to-severe asthma exacerbations over 52 weeks in the high-eosinophil subpopulation, defined by a baseline blood eosinophil count of at least 250 cells/μL.
The reported rate-ratio confidence intervals for both dose comparisons included 1. Secondary outcomes assessed quality of life, asthma control, and pre-dose FEV1. The registry also reports adverse event counts and deaths. Detailed information on multiplicity, missing-data handling, analysis-set definitions, and model specification is not reported in the registry record, limiting assessment of those aspects.