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 A Long-term Safety Study of QMF149 in Japanese Participants With Asthma (NCT03100500), a na phase 3 study. The primary outcome is Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03100500 |
| Phase | Phase 3 |
| Condition | Asthma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 51 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
The registry describes a long-term safety study of QMF149 in Japanese participants with asthma. Its stated purpose was to provide long-term safety data for Japanese participants with inadequately controlled asthma. The study evaluated participants during treatment with QMF149 150/320 μg once daily over 52 weeks. It was a single-group study, so the registry record does not describe a randomized control group or a like-for-like comparison with another treatment.
Population
The study enrolled 51 Japanese participants with asthma. The primary endpoint population was the Safety Set, defined as participants who received at least one dose of study medication. The registry record does not report further eligibility details in the record summarized for this analysis.
Intervention
Eligible participants took QMF149 150/320 μg once daily over 52 weeks. The study was open label, meaning the registry describes no masking.
Comparator
There was no separate comparator group in this single-group study. The observed safety counts therefore describe participants receiving QMF149, not a contrast between QMF149 and another treatment or control.
Primary question
The primary question concerned the number of participants with treatment-emergent adverse events and serious adverse events up to 52 weeks. The registry reports 37 participants with other adverse events, 0 with serious adverse events, and 0 deaths among 51 at risk.
The registry also lists changes in lung function, peak expiratory flow, asthma control, and rescue medication use as secondary outcomes. It lists an asthma control responder outcome based on achieving a minimal important difference of at least 0.5. Results for these secondary outcomes are not reported in the registry record analyzed here, so the safety figures do not answer questions about changes in asthma symptoms or lung function.
3. Trial Design
This was a Phase 3, multicenter, open-label, single-arm treatment study. Its official title describes a 52-week study in Japanese patients with asthma, and the registry lists asthma as the condition. The study enrolled 51 participants. The registry reports that it started on 2017-04-25, had primary completion on 2018-07-19, and is completed.
All eligible participants received QMF149 150/320 μg once daily over 52 weeks. The registry describes no masking and no allocation to distinct treatment groups. With one treatment group, the study can report the number of observed events during treatment, but it cannot estimate a randomized between-group treatment contrast. The registry explicitly identifies the study as not being a randomized comparison, and its treatment group should not be read as a like-for-like control comparison.
The primary outcome was the number of participants with treatment-emergent adverse events and serious adverse events up to 52 weeks. The record defines a treatment-emergent adverse event as one beginning on or after the first inhalation and no later than 7 days after the last administration, with a 30-day period after the last administration for a serious adverse event. It defines serious adverse events by specified consequences, including death, life-threatening events, persistent or significant disability or incapacity, hospitalization or its prolongation, congenital anomaly, and other medically significant events.
The Safety Set consisted of participants who received at least one dose of study medication. The adverse-event record lists 51 participants at risk in the QMF149 group. The registry lists secondary outcomes concerning FEV1, morning and evening PEF, ACQ-7, an ACQ-7 responder outcome, and rescue medication use. The registry record does not report a detailed analysis plan, a sample-size justification, or results for those secondary outcomes.
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.
Overall Study
| Stage | QMF-149 150/320 μg |
|---|---|
| STARTED | 51 |
| COMPLETED | 51 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety Set consisted of all participants who received at least one dose of study medication during this study.
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.
Population: Safety Set consisted of all participants who received at least one dose of study medication during this study.
| Characteristic | Unit | QMF-149 150/320 μg |
|---|---|---|
| Participants analysed | 51 | |
| Age, Continuous | years | 51.9 (SD 12.45) |
| Sex: Female, Male: Female | Participants | 28 |
| Sex: Female, Male: Male | Participants | 23 |
| Race/Ethnicity, Customized: Asian | Participants | 51 |
| Race/Ethnicity, Customized: East Asian | Participants | 51 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), measured over Up to 52 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants who experienced treatment-emergent adverse events and serious adverse events during follow-up of up to 52 weeks. It is a participant count, not a measure of how many separate events occurred or how long participants remained event-free. The registry defines the time window for treatment-emergent events in relation to the first inhalation and the last administration. Its serious-event definition includes events with specified severe consequences or other medically significant events.
Safety endpoints of this kind are used to describe adverse events observed during exposure to a study treatment. Counts can communicate how many participants in the analyzed group had an event under the registry’s definitions. Here, the record reports 37 participants with other adverse events and 0 with serious adverse events; it also reports 0 deaths. These counts are descriptive. Because the study has one treatment group and no randomized comparator, the endpoint does not by itself establish whether an event was caused by QMF149 or whether event frequency differs from what would have occurred under another treatment.
The primary endpoint population is the Safety Set: participants who received at least one dose. The adverse-event record lists 51 participants at risk. The registry’s event definitions and follow-up window are important when interpreting the counts, since events outside the defined treatment-emergent window are not captured by that endpoint definition. The registry record does not report further detail about event timing, severity categories beyond serious versus other events, or individual event terms.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results describe 51 participants at risk in the QMF149 group. The record reports 37 participants with other adverse events, 0 participants with serious adverse events, and 0 deaths. These are observed counts in a single treatment group over a study with treatment lasting up to 52 weeks. They do not constitute a comparison with an untreated group, placebo group, or alternative treatment.
The absence of reported serious adverse events in this group is a descriptive finding for this study. It does not demonstrate that serious events cannot occur, nor does it provide a comparative estimate of serious-event risk. Similarly, the count of participants with other adverse events indicates that such events were recorded, but the registry record does not provide the specific terms, timing, or severity breakdown for those events. It is therefore not possible to characterize the types of other events from these counts alone.
In general, a participant count is useful for describing the number of people with at least one event, but it does not state how many total event episodes occurred. A participant who experienced more than one event is still counted once in a count of participants with an event. The registry does not report total event episodes or person-time information in the record. No rate or time-to-event comparison can be inferred from the participant counts alone.
The open-label, single-arm design also limits interpretation. Without a separate control group, changes in event counts cannot be attributed to treatment by comparison with a concurrent alternative. The study’s stated purpose was to provide long-term safety data for Japanese participants with inadequately controlled asthma, but the registry record does not report a causal analysis. The counts should therefore be read as observations among participants who received QMF149, not as proof that the treatment caused or prevented an event.
The registry lists secondary outcomes related to FEV1, PEF, ACQ-7, the ACQ-7 minimal important difference responder outcome, and rescue medication use. Their results are not reported in the registry record. No inference about improvement, worsening, or lack of change in these outcomes follows from the adverse-event counts. Likewise, the record does not report confidence intervals or inferential comparisons for the primary safety counts. The data are descriptive, and their precision cannot be fully assessed from the posted counts alone.
In general, confidence intervals express uncertainty around an estimated quantity, with wider intervals indicating less precision. No confidence interval for these safety counts is reported in the registry record, so the interpretation should remain focused on the observed number of participants rather than an unreported estimate of underlying event frequency. The record also does not report a detailed analysis plan or sample-size justification, which prevents assessment of whether the study was designed to estimate specific safety quantities with a prespecified level of precision.
9. Statistical Methods Explained
The central statistical feature of this study is its single-group design. The primary safety outcome is a count of participants with events, and the adverse-event results are reported for the QMF149 group. The registry lists 51 participants at risk. No randomized comparison group is described, so the reported numbers are not treatment-effect estimates comparing QMF149 with another arm.
What does a participant count represent?
In general, a participant count records how many individuals meet an outcome definition at least once during the stated observation period. It does not count repeated episodes separately. For this endpoint, the registry reports numbers of participants with treatment-emergent adverse events and serious adverse events. It reports 37 participants with other adverse events and 0 with serious adverse events. The record also reports 0 deaths. These counts communicate what was observed in the study group; they do not reveal how many separate events each participant experienced.
The registry defines treatment-emergent events by their timing relative to study treatment: an event must begin on or after the first inhalation and within the specified period after the last administration. It gives a 7-day period for adverse events and a 30-day period for serious adverse events. These operational definitions determine which events are included in the primary outcome. An event outside the applicable window would not meet that definition solely by virtue of occurring during a different period.
Why does the study design matter?
In general, a randomized comparator helps distinguish outcomes associated with one assigned treatment from outcomes occurring under another assigned condition. This study has one group and is open label, with no masking reported. As a result, its event counts are descriptive rather than a randomized estimate of a difference between treatments. There is no concurrent control count in the registry record to use as a comparison. The design also means that the posted figures alone cannot separate treatment effects from other influences on event occurrence.
The study is described as a Phase 3 multicenter treatment study, but those labels do not change the interpretation of the single-group safety counts. The registry identifies the Safety Set as participants who received at least one dose of study medication. This is an exposure-based population definition: participants must have received treatment to enter that set. The adverse-event record lists 51 participants at risk. The registry record does not report additional details about participant-level analysis procedures or any alternate analysis populations.
How should precision be assessed?
In general, an estimate based on a limited number of participants has sampling uncertainty. A confidence interval is one way to express that uncertainty around an estimated quantity. The registry record does not report confidence intervals for the safety counts. It also does not report a comparative effect estimate or an inferential test for the primary endpoint. Accordingly, the posted counts should not be presented as if they were precise estimates of event frequency in a broader population, nor as evidence of a statistically tested treatment difference.
In general, over-dispersion matters when event data are analyzed as counts of repeated episodes because the variability can exceed what a simple count model assumes. That issue is distinct from a participant-level endpoint, which counts people with an event rather than event episodes. The registry’s primary endpoint is a count of participants, and the record does not provide total repeated-event counts or an event-rate analysis. No over-dispersion adjustment is reported in the registry record.
What can be concluded from the listed outcomes?
The registry lists secondary outcomes involving change from baseline in pre-dose FEV1 after 26 and 52 weeks, morning and evening PEF during treatment, ACQ-7 after 26 and 52 weeks, an ACQ-7 responder definition based on a minimal important difference of at least 0.5, and rescue medication use. Listing an outcome does not establish its result. The registry record does not report numerical results for those secondary outcomes in the information, so conclusions about them cannot be drawn from the safety counts.
The record lists 6 outcome measures and 0 analyses. It does not report a detailed statistical analysis plan, an approach to multiplicity, or a missing-data method. In general, an analysis plan specifies how outcomes and data will be handled, while multiplicity procedures address the increased chance of false-positive findings when many hypotheses are tested. Since the registry record does not report these procedures or inferential results, it is not possible to assess how such issues were handled for the study.
What is not an analysis result?
In general, planning calculations estimate the sample size that might be needed under stated assumptions; they are not observed outcomes or evidence of power achieved by a completed study. The registry record does not report a study-specific sample-size justification. It also does not report interim analyses. Neither a protocol rationale nor an interim monitoring procedure should be inferred from the study’s phase, duration, or participant count.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple outcomes: the primary count of participants with treatment-emergent adverse events and serious adverse events, along with secondary measures of FEV1, PEF, ACQ-7, ACQ-7 responder status, and rescue medication use. The record lists 6 outcome measures. It does not report a multiplicity strategy or a detailed statistical analysis plan. Since numerical results for the secondary outcomes are not reported in the registry record, there are no posted secondary-outcome tests in this record to interpret for multiplicity.
In general, multiplicity arises when several hypotheses or outcomes are tested: even if each test is individually conducted at a chosen significance level, the chance of at least one false-positive finding across tests can increase. Prespecified procedures can address this, but the registry record does not report whether any were planned or used. No adjustment method should be assumed.
The registry defines the Safety Set as participants who received at least one dose of study medication. The adverse-event listing shows 51 participants at risk. The record does not report how missing observations were handled for the primary or secondary outcomes, whether any participants discontinued, or whether there were incomplete follow-up data. It also does not provide a detailed account of missingness by outcome. These omissions mean that the available record does not support an assessment of the extent or potential influence of missing data.
In general, missing-data methods depend on what is missing, why it is missing, and the outcome being analyzed. Different assumptions can lead to different interpretations, so the analysis method matters. Here, the relevant assumptions and procedures are not reported in the registry record. The endpoint population definition and at-risk count can be stated, but they do not establish that every outcome was observed for every participant throughout the planned treatment period.
11. 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 |
|---|---|---|---|
| QMF149 150/320 µg | 0 | 51 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| QMF149 150/320 µg | 0 of 51 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| QMF149 150/320 µg | 37 of 51 |
Most frequent non-serious adverse event terms
| Event term | Organ system | QMF149 150/320 µg |
|---|---|---|
| Asthma | Respiratory, thoracic and mediastinal disorders | 28 of 51 |
| Nasopharyngitis | Infections and infestations | 15 of 51 |
| Bronchitis | Infections and infestations | 6 of 51 |
| Upper respiratory tract infection bacterial | Infections and infestations | 3 of 51 |
| Back pain | Musculoskeletal and connective tissue disorders | 3 of 51 |
| Myalgia | Musculoskeletal and connective tissue disorders | 3 of 51 |
| Gastritis | Gastrointestinal disorders | 2 of 51 |
| Otitis media | Infections and infestations | 2 of 51 |
| Pharyngitis | Infections and infestations | 2 of 51 |
| Electrocardiogram QT prolonged | Investigations | 2 of 51 |
The safety listing reports 51 participants at risk in the QMF149 150/320 μg group. It records 0 participants with serious adverse events, 37 with other adverse events, and 0 deaths. The primary endpoint covers treatment-emergent events up to 52 weeks, using the registry’s stated timing definitions. These figures are participant counts, not counts of event episodes.
A count of 0 serious adverse events means none were recorded for the listed group under the registry’s outcome definition and reporting. It should not be read as proof that serious adverse events are impossible or that the treatment has no serious risks. Because there is no control group, the count cannot be compared with a concurrent alternative treatment. The registry also does not report confidence intervals for the counts, so their precision is not quantified in the record.
The 37 participants with other adverse events indicate that events in this category were recorded among participants at risk. The registry information does not list the specific event terms, number of recurrent episodes, or severity distribution for these events. The counts also do not establish that the events were caused by study treatment. The study’s open-label, single-arm design and the absence of a comparator should remain central when interpreting the safety listing.
12. What the Result Does, and Does Not, Mean
The results describe adverse events recorded among participants receiving QMF149 in a 52-week, open-label, single-arm study. The registry lists 51 participants at risk, 37 participants with other adverse events, 0 with serious adverse events, and 0 deaths. These are the posted safety findings for the study group. They offer a descriptive account of what was recorded in this study under its event definitions and follow-up window.
The results do not establish that QMF149 caused the reported other adverse events. Nor does the absence of serious adverse events in this group establish that such events cannot occur. A single-arm study has no concurrent randomized comparison from which to estimate how event counts might differ under another treatment or control condition. The record also does not report an inferential analysis or confidence intervals for the safety counts, so their statistical precision is not quantified.
The registry’s stated purpose was to provide long-term safety data for Japanese participants with inadequately controlled asthma. This result set does not answer every safety question: the record does not report specific terms for the other adverse events, their timing, or the number of repeated episodes. It also does not report a detailed missing-data account or a sample-size justification.
The listed secondary outcomes concern lung function, peak expiratory flow, asthma control, responder status, and rescue medication use. Their results are not reported in the registry record. Therefore, the adverse-event counts do not establish whether those measures changed during treatment. They also do not establish comparative efficacy, because no comparator group is described.
In general, statistical conclusions are bounded by the design, outcome definitions, and information reported. The appropriate reading is therefore specific: the registry records 37 participants with other adverse events and no participants with serious adverse events or deaths among 51 at risk in the QMF149 group. Broader claims about comparative safety, causation, or outcomes beyond those posted counts are not supported by this record alone.
13. Why This Trial Matters Statistically
This study illustrates why design and endpoint structure matter when reading clinical-trial statistics. A participant count can directly describe how many people in a treatment group experienced an event, and the registry reports such counts for adverse events in the QMF149 group. But a single-group count answers a narrower question than a randomized comparison: it describes observations during treatment without estimating a contrast against another group.
The study also makes clear that an endpoint’s definition controls what its count means. The registry specifies the treatment-emergent timing window and gives a definition for serious adverse events. Readers need those rules to understand which events are included. The count is of participants, not repeated event episodes, so it should not be interpreted as a total event burden or event rate.
Safety information over a 52-week treatment period can be relevant to the stated purpose of providing long-term safety data for Japanese participants with inadequately controlled asthma. The registry reports no serious adverse events or deaths among 51 at risk, alongside 37 participants with other adverse events. These counts are important descriptive results, but they do not provide a control-based estimate or a measure of precision such as a confidence interval.
The record also lists several secondary outcomes, while their results are not reported in the registry record analyzed. This is a useful reminder to distinguish outcomes named in a trial record from outcomes with posted numerical results. Statistical interpretation depends not only on what a study set out to measure, but on what results, analysis methods, and uncertainty estimates are available to assess.
14. 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 principal limitation for interpreting the safety counts is the single-arm design. The study has no randomized control group, so the registry cannot support a like-for-like comparison of event counts with another treatment. Its open-label design also means masking is not reported. The posted counts describe events in the QMF149 group and do not by themselves establish causation.
The record reports participant counts but does not give confidence intervals, comparative estimates, or inferential analyses for the primary safety endpoint. It does not list specific terms for the 37 participants with other adverse events, nor does it report the number of repeated event episodes or a severity breakdown beyond the serious-event category. Thus, the detail needed to characterize the nature and frequency of individual events is not reported in the registry record.
The study enrolled 51 participants, and the event listing reports 51 at risk. The record does not report a sample-size justification, so the intended precision for safety estimates cannot be assessed. It also does not report a detailed statistical analysis plan, interim analyses, missing-data methods, or a participant-level account of follow-up and discontinuation. These omissions limit assessment of how complete the outcome data were and how analyses were specified.
Although several secondary outcomes are listed, their numerical results are not reported in the registry record analyzed. The safety findings therefore cannot be used to assess lung function, peak expiratory flow, asthma control, responder status, or rescue medication use. The findings apply to the study population and observation described in the registry; the record does not provide evidence for a comparative effect or a broader population estimate.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Long-term Safety Study of QMF149 in Japanese Participants With Asthma — NCT03100500.
- Registry record last updated 2020-02-07; status last verified 2020-01.
- Results first posted 2020-02-07.
- 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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18. Record Summary
The registry record describes a completed Phase 3, multicenter, open-label, single-arm study of QMF149 in Japanese participants with asthma. The study enrolled 51 participants and used once-daily QMF149 150/320 μg over 52 weeks. Its stated purpose was to provide long-term safety data for Japanese participants with inadequately controlled asthma.
The primary outcome was the number of participants with treatment-emergent adverse events and serious adverse events up to 52 weeks. The Safety Set included participants who received at least one dose. The adverse-event listing reports 37 participants with other adverse events, 0 with serious adverse events, and 0 deaths among 51 at risk. The registry also lists secondary outcomes concerning lung function, peak expiratory flow, asthma control, responder status, and rescue medication use, but their results are not reported in the registry record analyzed.
Because the study had one group and no randomized comparator, the safety counts are descriptive and do not establish comparative effects. The registry record does not report confidence intervals, a detailed analysis plan, a sample-size justification, or missing-data methods.