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 Patient Handling Study of Mometasone Furoate/Formortoral Fumarate (MF/F) Metered Dose Inhaler (MDI) With an Integrated Dose Counter in Adolescent and Adult Subjects With Asthma or Chronic Obstructive Pulmonary Disease (Study P04703AM1) (NCT00604500), a na phase 3 study. The primary outcome is Overall Discrepancy Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00604500 |
| Phase | Phase 3 |
| Condition | Asthma, COPD |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 272 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Organon and Co |
| Results posted | Yes |
2. Clinical Question
Population
Adolescents and adults aged 12 years or older with persistent asthma or chronic obstructive pulmonary disease (COPD) of at least 12 months’ duration were eligible. The registry record reports enrollment of 272 participants. The primary endpoint used a narrower completer population: participants who recorded using at least 90% of the labeled actuations during the 4-week Treatment Period.
Intervention
Participants used mometasone furoate/formoterol fumarate (MF/F) through a metered dose inhaler with an integrated dose counter. The regimen was 100/10 mcg twice daily, given as two inhalations of the 50/5 mcg inhaler per dose. The study assessed the device during normal patient handling conditions.
Comparator
There was no comparator group. The registry describes a single-group, open-label study, with allocation listed as not applicable and masking listed as none. Therefore, the study does not provide a randomized, like-for-like comparison with another inhaler, device, or treatment condition.
Primary question
Across the 4-week Treatment Period, how often did the participant-recorded number of actuations differ from the participant-recorded dose-counter readout? The endpoint was expressed as overall discrepancies per 100 actuations among completers who recorded at least 90% of labeled actuations. The observed discrepancy rate is not reported in the registry record, so the magnitude and precision of the result cannot be assessed from the posted information.
3. Trial Design
This was a phase 3, open-label, multicenter, single-group study. The registry record lists enrollment of 272 and a completed status. The study start date was 2008-03-01, and the primary completion date was 2008-11-01. The treatment period described for the primary endpoint was 4 weeks. Participants were adolescents and adults aged 12 years or older with persistent asthma or COPD of at least 12 months’ duration.
All participants in the study group used MF/F MDI 100/10 mcg twice daily. The regimen was administered as two inhalations of MF/F MDI 50/5 mcg per dose, approximately 12 hours apart. The inhaler included an integrated dose counter. The stated purpose was to evaluate the counter’s performance under normal patient handling conditions, rather than to compare clinical outcomes between assigned treatment groups.
Masking was listed as none, and allocation was not applicable. The registry therefore describes an unmasked study without a randomized control group. The design can describe the recorded agreement or discrepancy between participant logs and counter readouts in the eligible completer population. By itself, it cannot establish how that performance compares with another device or with a different study condition.
The registry record names the sponsor as Organon and Co and describes the study as Study P04703AM1. Details such as the recruitment process, site count, specific device-training procedures, and handling of protocol deviations are not reported in the registry record. The record also does not explain the rationale for the chosen treatment period or provide a sample-size justification.
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 | MF/F MDI 100/10 mcg BID (With Dose Counter) |
|---|---|
| STARTED | 272 |
| COMPLETED | 261 |
| NOT COMPLETED | 11 |
Reasons for not completing this period:
| Reason | MF/F MDI 100/10 mcg BID (With Dose Counter) |
|---|---|
| Adverse Event | 2 |
| Protocol Violation | 2 |
| Treatment Failure | 1 |
| Withdrawal by Subject | 4 |
| Administrative | 1 |
| Did not meet protocol eligibility | 1 |
Analysis population for the primary outcome, as defined in the registry: Completer population, defined as participants who recorded use of at least 90% of the labeled actuations during the 4-week Treatment Period.
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 | MF/F MDI 100/10 mcg BID (With Dose Counter) |
|---|---|---|
| Participants analysed | 272 | |
| Age, Continuous: Age 12 to <18 years of age | years | 14.69 (SD 1.64) |
| Age, Continuous: Age 18 to <65 years of age | years | 44.22 (SD 12.12) |
| Age, Continuous: Age ≥ 65 years of age | years | 70.43 (SD 4.77) |
| Sex: Female, Male: Female | Participants | 178 |
| Sex: Female, Male: Male | Participants | 94 |
| Number of Participants with Asthma and Chronic Obstructive Pulmonary Disease (COPD): Asthma | Participants | 222 |
| Number of Participants with Asthma and Chronic Obstructive Pulmonary Disease (COPD): Chronic Obstructive Pulmonary Disease (COPD) | Participants | 50 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Discrepancy Rate, measured over 4-week Treatment Period.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Overall discrepancies per 100 actuations |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the overall discrepancy rate, expressed as discrepancies per 100 actuations during the 4-week Treatment Period. A discrepancy was the difference between the participant-recorded number of actuations and the participant-recorded dose-counter readout. The endpoint calculation used the total number of discrepancies and the total number of actuations among completers who recorded use of at least 90% of labeled actuations.
This measure addresses whether the inhaler’s counter readout and participants’ own actuation records aligned during routine handling. Expressing discrepancies relative to actuations provides a rate rather than only a raw count, which can make results more interpretable when participants contribute different numbers of actuations. That is a general statistical rationale for rate measures; it does not establish the size or importance of any discrepancy in this study.
The primary endpoint’s observed value, uncertainty estimate, and any inferential test are not reported in the registry record. Consequently, the endpoint definition is available, but its observed magnitude and precision cannot be described from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record specifies what the primary endpoint was intended to measure, but does not report the observed overall discrepancy rate. It therefore does not support a numerical description of how often participant records differed from counter readouts in the primary analysis population. A confidence interval, standard error, or hypothesis-test result for that endpoint is also not reported in the registry record. Without an estimate and its uncertainty, the degree of precision cannot be judged.
The endpoint was defined for completers who recorded at least 90% of labeled actuations during the 4-week Treatment Period. This means the primary analysis population was not simply every enrolled participant. The number of participants meeting the completer definition is not reported in the registry record. Enrollment of 272 should not be treated as the endpoint denominator, because the record does not state that all enrolled participants met the completion criterion or contributed to the primary calculation.
The study’s single-group, open-label design is consistent with a descriptive assessment of device handling in the enrolled population. It does not provide a concurrent comparison group, so the results cannot distinguish device performance from differences that might arise under another device or study condition. The endpoint also concerns concordance between two participant-recorded sources; it is not a direct measure of clinical benefit, disease control, or the accuracy of inhaled medication delivery.
The registry’s endpoint description says that the rate was calculated as 100 multiplied by the total discrepancies divided by total actuations in the completer population. That definition clarifies the intended scale and denominator. However, because the numerator, denominator, and resulting rate are not reported in the registry record, the calculation cannot be checked from the posted information. No recalculated estimate is available to compare with a sponsor analysis.
The posted record identifies four outcome measures and lists zero analyses. It does not report a statistical analysis plan, inferential results, or an explanation of how the primary result was summarized. The appropriate reading is therefore limited: the registry defines the question and analysis population, but the primary numerical finding and its precision are not reported in the registry record.
9. Statistical Methods Explained
What a rate means. The primary endpoint is a rate: discrepancies are related to the number of actuations contributed by the completer population and expressed per 100 actuations. In general, a rate uses an exposure or opportunity denominator so that counts can be interpreted in relation to how much observation occurred. Here, an actuation is the stated denominator. A rate does not by itself describe the distribution of discrepancies among individual participants, and a pooled rate can be influenced by participants who contribute more actuations.
What the denominator includes. The registry defines the completer population as participants who recorded at least 90% of labeled actuations during the 4-week Treatment Period. The intended calculation uses total discrepancies and total actuations in that population. This definition makes adherence to recorded use part of eligibility for the primary calculation. The number of completers and the totals used in the calculation are not reported in the registry record, so the endpoint arithmetic cannot be independently reproduced from the posted results.
How to think about precision. A point estimate gives a single summary of the observed rate, while a confidence interval describes the range of values reasonably compatible with the data under the interval’s statistical assumptions. A narrow interval generally indicates greater precision than a wide interval. Neither an endpoint estimate nor a confidence interval is reported in the registry record. It is therefore not possible to assess precision, distinguish a stable estimate from a highly uncertain one, or judge whether the observed rate is compatible with a particular benchmark.
Why participant-level information can matter. A pooled rate combines information across participants. In general, participants may contribute different numbers of actuations, and discrepancies may cluster within a participant rather than occur independently. If observations are more variable than a simple model assumes, over-dispersion can make conventional uncertainty estimates too small. Methods that account for clustering or extra variability may be relevant in some rate analyses. The registry record does not report the statistical model, variance assumptions, or whether clustering or over-dispersion was assessed, so it is not possible to determine which approach was used.
Single-group description versus comparison. With no randomized comparator, the primary endpoint can be read as a description of recorded counter-versus-participant discrepancies in the study population. It is not a treatment contrast. General statistical comparisons require a defined contrast and an appropriate comparison group; neither is identified for this primary question. No comparative effect estimate or hypothesis test is reported in the registry record.
Planning illustrations. Any sample-size figures associated with the posted material are planning illustrations based on an observed effect, not observed trial outcomes or evidence of the trial’s achieved power. The stated planning assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those assumptions describe a hypothetical planning exercise, not this single-group study’s design. The registry record does not report the trial’s own sample-size justification, and the planning illustration should not be interpreted as observed power.
What cannot be inferred. The registry record does not report an analysis method for the primary rate, a prespecified benchmark, or an inferential framework. General statistical concepts can explain how rates and uncertainty are often assessed, but they cannot fill in those missing study-specific details. The posted endpoint definition alone does not reveal the observed result or whether any analytic assumptions were satisfied.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies four outcome measures, but it does not report how many were designated primary, how they were ordered, or whether statistical testing was planned across multiple endpoints. The record lists zero analyses. A multiplicity strategy, such as a prespecified adjustment or hierarchy for interpreting multiple tests, is not reported in the registry record. Without those details, the impact of multiple testing cannot be assessed. This does not establish that no such planning occurred; it means the registry record does not describe it.
The primary endpoint population is defined using a completion threshold: participants had to record use of at least 90% of labeled actuations during the 4-week Treatment Period. The number meeting that threshold is not reported in the registry record, nor is the number excluded from the primary population. The handling of missing actuation records, incomplete counter readings, or other missing endpoint data is not reported. It is also not reported whether missing values were imputed, whether records were treated as incomplete, or whether sensitivity analyses were conducted.
The registry record does not report interim analyses, stopping rules, or an analysis plan for the primary endpoint. A sample-size justification for the trial is also not reported in the registry record. These omissions limit assessment of how the endpoint, missing data, and any multiple analyses were handled. They should be treated as unavailable reporting details, not as evidence that a particular method was or was not used.
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 |
|---|---|---|---|
| MF/F MDI 100/10 mcg BID | 1 | 272 | 0.4% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| MF/F MDI 100/10 mcg BID | 0 of 272 |
Most frequent serious adverse event terms
| Event term | Organ system | MF/F MDI 100/10 mcg BID |
|---|---|---|
| Oedema Peripheral | General disorders | 1 of 272 |
Serious events with the highest arm-level rate: oedema peripheral (0.4%).
The registry’s adverse-event figures list 272 participants at risk for the MF/F MDI 100/10 mcg twice-daily group. One participant was recorded as affected by a serious adverse event, and zero participants were recorded as affected by an other adverse event. These counts describe the registry entries for this study group; they do not establish whether an event was caused by the inhaler.
The leading serious adverse-event term is listed as Oedema Peripheral, with 0.4% shown in the registry record. The record does not provide further detail about the event’s timing, duration, outcome, or assessed relationship to treatment. A death count is not reported in the registry record. The figures should be read as descriptive safety reporting for a single-group study, not as a comparison of risk against another treatment or device.
12. What the Result Does, and Does Not, Mean
This study was designed to examine an operational question: whether participants’ actuation records agreed with the readout from an integrated dose counter during the 4-week Treatment Period. That is distinct from a clinical efficacy question. The endpoint concerns recorded use and counter readings; it does not directly measure asthma or COPD symptoms, lung function, medication delivery, or changes in disease status.
The registry record gives the endpoint definition and the completer criterion, but does not report the observed discrepancy rate or its precision. As a result, readers cannot determine from the posted record how frequent discrepancies were, how variable the result was, or whether the estimate was compatible with any practical standard. The absence of a reported rate should not be interpreted as a rate of zero.
The design also limits comparative interpretation. Because the study was open-label and single-group, it does not estimate a difference from another inhaler or a control condition. The registry record does not support a conclusion that the device performed better or worse than an alternative. Nor can the adverse-event counts establish comparative safety.
The findings that can be described are therefore about the study’s purpose, endpoint definition, treatment regimen, and analysis population. The numerical primary result is not reported in the registry record, so no conclusion about the observed level of counter-record discrepancy can be drawn from the posted information alone.
13. Why This Trial Matters Statistically
This trial illustrates that a clinical study can focus on measurement and device handling rather than a comparison of clinical outcomes. Its primary endpoint connects two forms of participant-recorded information: actuation records and dose-counter readouts. Defining the discrepancy in relation to total actuations makes the intended summary understandable, while the completer rule clarifies which participants were meant to contribute to that summary.
It also shows why an endpoint definition is not a substitute for an endpoint result. Without the observed rate, denominator, and uncertainty, readers cannot assess the size or precision of the finding. Clear reporting of analysis populations and missing-data handling is particularly relevant when an endpoint depends on participant records and a completion threshold.
The single-group design is suited to a descriptive question but does not answer comparative questions. Keeping that distinction clear prevents a device-handling measure from being read as evidence of clinical effect or as a comparison with another option. The posted record is informative about the study’s intended measurement, but its missing primary numerical result limits statistical interpretation.
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 most consequential limitation for statistical interpretation is that the primary discrepancy rate is not reported in the registry record. The record also does not report its numerator, denominator, or uncertainty estimate. This prevents verification of the calculation and assessment of precision. The number of completers who met the at-least-90% recorded-use criterion is not reported, so the size of the primary analysis population cannot be established from the posted information.
The study had no comparator group and was open-label. It can describe the stated endpoint within the study group, but cannot provide a randomized comparison with another device or condition. The endpoint relies on participant-recorded actuations and participant-recorded counter readouts; the registry record does not describe validation procedures, data-quality checks, or how discordant or incomplete records were handled.
Important analysis details are not reported in the registry record, including the method used to estimate the rate, its variance assumptions, the approach to missing data, and any sensitivity analyses. A sample-size justification, interim analysis plan, and multiplicity strategy are also not reported. Enrollment of 272 does not resolve these gaps or establish how many participants contributed to the primary endpoint.
Finally, the registry’s safety entries are descriptive and do not provide a control group or detailed event narratives. The posted information does not support attribution of events to the inhaler or a comparative safety assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Patient Handling Study of Mometasone Furoate/Formortoral Fumarate (MF/F) Metered Dose Inhaler (MDI) With an Integrated Dose Counter in Adolescent and Adult Subjects With Asthma or Chronic Obstructive Pulmonary Disease (Study P04703AM1) — NCT00604500.
- Registry record last updated 2024-05-20; status last verified 2022-02.
- Results first posted 2011-04-12.
- 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, open-label, multicenter, single-group study with enrollment of 272 participants aged 12 years or older with persistent asthma or COPD of at least 12 months’ duration. Participants used MF/F MDI 100/10 mcg twice daily, administered as two inhalations of the 50/5 mcg inhaler per dose. The primary endpoint was overall discrepancies per 100 actuations during the 4-week Treatment Period, among completers who recorded at least 90% of labeled actuations.
The observed primary endpoint value and its precision are not reported in the registry record. The record lists one participant with a serious adverse event among 272 at risk and zero with an other adverse event; the death count is not reported. The design provides no randomized comparator, and key analysis details, including missing-data handling and a sample-size justification, are not reported in the registry record.