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 Study of Nasonex® for the Treatment of Nasal Polyps in Pediatric Subjects Between Ages of 6 and Less Than 18 Years Old (P04292) (NCT00378378), a randomized phase 3 study. The primary outcome is Change From Baseline 24-hour Urinary Free Cortisol Level.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00378378 |
| Phase | Phase 3 |
| Condition | Nasal Polyps |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 127 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Organon and Co |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric subjects aged 6 to less than 18 years with nasal polyps. The study enrolled 127 participants. For the primary endpoint, only participants with both a baseline and an endpoint urine sample were included in the analyzed population.
Intervention
Mometasone furoate nasal spray (MFNS), with the registry listing once-daily and twice-daily groups and age-specific dose groups. The arm descriptions include MFNS 100 mcg once per day for subjects 6 to less than 12 years of age; other listed group names describe 100 mcg or 200 mcg schedules.
Comparator
Placebo nasal spray, listed in once-daily and twice-daily groups for the two age ranges. The registry record does not report numerical primary-endpoint results comparing MFNS with placebo.
Primary question
How did 24-hour urinary free cortisol change from baseline to endpoint among pediatric subjects receiving MFNS, with safety as the study’s primary focus? This biomarker was used to assess a safety objective; the registry record does not provide the estimates needed to describe the observed change or compare groups statistically.
The study’s stated purpose was to evaluate the safety and efficacy of MFNS for nasal polyps in pediatric subjects. Its primary objective, however, was safety, assessed by determining 24-hour urinary free cortisol. A secondary outcome was change from baseline in 24-hour urinary free cortisol corrected for creatinine. The available record therefore identifies a safety-focused biomarker question, but does not establish the numerical size or precision of any treatment-group difference.
3. Trial Design
NCT00378378 is a completed Phase 3 randomized, double-masked, parallel-group study sponsored by Organon and Co. The registry records a start in 2006-07 and primary completion in 2008-06. Enrollment was 127. The population was pediatric subjects aged 6 to less than 18 years with nasal polyps.
The listed interventions were mometasone furoate nasal spray (MFNS) and placebo nasal spray. The registry lists groups with once-daily and twice-daily schedules, with dose groups associated with the age ranges 6 to less than 12 years and 12 to less than 18 years. One arm description specifies MFNS 100 mcg once per day for subjects 6 to less than 12 years of age. The other arm descriptions are not detailed beyond their group names in the registry record.
Randomization means assignment to study groups was intended to occur by chance; double masking indicates that the record describes the study as masked for two parties, but it does not identify those parties. A parallel-group design assigns participants to a group that is followed alongside the other groups, rather than assigning each participant to receive every intervention in sequence. These are general descriptions of the design terms, not details of the study’s implementation.
The registry record does not report the randomization procedure, allocation concealment, masking procedures, adherence, or the numbers assigned to each intervention group. It also does not report the protocol’s sample-size justification or a detailed statistical analysis plan. Those omissions limit how much can be concluded about the conduct and analysis beyond the design labels and endpoint descriptions that are recorded.
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 | MFNS 100 or 200 mcg BID for Subjects 6 to Less Than 18 Years | MFNS 100 or 200 mcg QD for Subjects 6 to Less Than 18 Years | Pooled Placebo |
|---|---|---|---|
| STARTED | 51 | 50 | 26 |
| COMPLETED | 51 | 49 | 24 |
| NOT COMPLETED | 0 | 1 | 2 |
Reasons for not completing this period:
| Reason | MFNS 100 or 200 mcg BID for Subjects 6 to Less Than 18 Years | MFNS 100 or 200 mcg QD for Subjects 6 to Less Than 18 Years | Pooled Placebo |
|---|---|---|---|
| Withdrawal by Subject | 0 | 1 | 0 |
| Treatment Failure | 0 | 0 | 1 |
| Non-compliance with protocol | 0 | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Not all participants that were randomized had both a baseline and an endpoint urine sample. Only participants with both were included in the participants analyzed.
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 | MFNS 100 or 200 mcg BID for Subjects 6 to Less Than 18 Years | MFNS 100 or 200 mcg QD for Subjects 6 to Less Than 18 Years | Pooled Placebo | Total |
|---|---|---|---|---|---|
| Participants analysed | 51 | 50 | 26 | 127 | |
| Age, Continuous | years | 9.6 (SD 1.4) | 14.4 (SD 1.8) | 12.7 (SD 3.2) | 12.7 (SD 2.9) |
| Age, Customized: 6 to < 12 years | participants | 18 | 18 | 10 | 46 |
| Age, Customized: 12 to < 18 years | participants | 33 | 32 | 16 | 81 |
| Sex: Female, Male: Female | Participants | 20 | 22 | 12 | 54 |
| Sex: Female, Male: Male | Participants | 31 | 28 | 14 | 73 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline 24-hour Urinary Free Cortisol Level, measured over Baseline to Endpoint.
| Element | Registry entry |
|---|---|
| Result type | Least Squares Mean |
| Measure of spread | Standard Deviation |
| Unit | mcg/hour |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was change from baseline to endpoint in 24-hour urinary free cortisol, measured in mcg/hour. Urinary free cortisol is a biomarker measured in a urine collection over 24 hours. In this study, the registry identifies it as the measure used to assess the primary safety objective for MFNS in pediatric subjects.
Using a baseline-to-endpoint change focuses the endpoint on how a measurement differs from a participant’s starting value over the study period. A biomarker can be used to monitor a physiologic measure relevant to safety, although the registry description alone does not establish how changes should be interpreted clinically. This is a general point about biomarker endpoints, not a conclusion about the study’s results.
The registry characterizes the reported parameter as a least-squares mean and the dispersion as a standard deviation. It states that not all randomized participants had both a baseline and endpoint urine sample, and only participants with both samples were included in the analyzed population. The actual endpoint estimates, standard deviations, confidence intervals, and statistical tests are not reported in the registry record. Consequently, the direction, magnitude, and precision of the observed change cannot be described from this record.
A secondary outcome was change from baseline in 24-hour urinary free cortisol corrected for creatinine. That is distinct from the primary endpoint, which is recorded without the creatinine correction in its title. The registry record does not provide numerical results for either measure.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies the study’s primary safety measure and describes how the analyzed population was selected, but it does not report numerical results for the primary endpoint. There is no recorded least-squares mean change, group-specific estimate, treatment contrast, confidence interval, or p-value for the 24-hour urinary free cortisol endpoint. Without those statistics, the direction and magnitude of the observed changes, and the precision of any comparison, are not reported in the registry record.
This absence matters for interpretation. A study’s design and endpoint specify what investigators intended to assess; they do not reveal what was observed. The fact that the study was randomized, double-masked, and parallel-group does not by itself establish whether groups differed on the biomarker. Nor does enrollment alone tell how much information was available for the primary analysis, because the registry says that participants needed both baseline and endpoint samples to be analyzed.
The adverse-event information is separately recorded. In the MFNS 100 or 200 mcg once-daily group, 0 participants were recorded with serious adverse events among 50 at risk; the registry records 24 with other adverse events among 50 at risk. In the MFNS 100 or 200 mcg twice-daily group, 0 were recorded with serious adverse events among 51 at risk, and 23 with other adverse events among 51 at risk. In the placebo group, 0 were recorded with serious adverse events among 26 at risk, and 13 with other adverse events among 26 at risk. These are descriptive counts. They do not supply a statistical comparison, and the registry record does not report deaths.
Counts of people with events do not describe event severity beyond the serious-versus-other grouping, the timing of events, or the number of events per participant. The record also does not report an analysis relating the adverse-event counts to the urinary cortisol endpoint. Thus, the safety information available in the registry consists of a limited set of event counts and an endpoint definition without endpoint results. It should not be treated as a complete account of the study’s findings.
The registry lists 2 outcome measures and 6 analyses, but does not report the analysis details needed to understand how those analyses were selected or interpreted. No conclusion about statistical significance or the clinical importance of a difference can be drawn from the primary endpoint description alone.
9. Statistical Methods Explained
The registry describes the primary result as a least-squares mean for a continuous outcome, with standard deviation as the dispersion measure. Those terms describe a way to summarize a numerical measurement, but they are not a complete account of the statistical model. The model specification, covariates, handling of repeated or incomplete measurements, and group contrast are not reported in the registry record.
What does a least-squares mean represent?
In general, a least-squares mean is a model-based estimate of a group mean. Depending on the model, it can account for specified factors rather than simply taking the arithmetic average of observed values. The factors included and the exact interpretation depend on the analysis model. Although the registry names a least-squares mean, it does not state which model or adjustments were used. It is therefore not possible to determine from the record whether or how the primary estimate was adjusted.
A standard deviation describes the spread of individual measurements around a mean. It is not the same as a standard error, which describes uncertainty in an estimated mean. Nor does a standard deviation alone give the uncertainty around a difference between groups. To assess that uncertainty, readers generally need an estimate of the contrast and a confidence interval or another inferential statistic. Those primary-endpoint statistics are not reported in the registry record.
How should change from baseline be understood?
Change from baseline compares a later measurement with the participant’s starting measurement. As a general statistical concept, the analysis might summarize individual changes, compare group mean changes, or use a model that incorporates baseline values. These approaches can have different assumptions and interpretations. The endpoint title specifies change from baseline to endpoint, but the registry record does not describe the calculation or model used for that change.
Baseline and endpoint measurements were required for inclusion in the primary analysis. This means the analyzed population excluded randomized participants who did not have both samples. The record does not give the number excluded for this reason, the reasons samples were unavailable, or whether any methods were used to account for missing measurements. Without such information, the potential influence of missing data on the endpoint estimates cannot be evaluated.
What would a confidence interval and p-value add?
In general, a confidence interval expresses uncertainty around an estimate and shows a range of values compatible with the data and analysis assumptions. A narrow interval indicates greater precision than a wide interval, while the interval’s location helps readers assess the possible direction and size of an effect. A p-value, when used, summarizes how compatible the data are with a specified null hypothesis under the assumptions of the statistical test. Neither statistic gives the probability that a treatment works, and neither replaces consideration of the estimated effect and its practical meaning.
The registry record does not report confidence intervals or p-values for the primary endpoint. It also does not state the null hypothesis, the statistical test, or the threshold used for the primary analysis. As a result, statistical significance and precision cannot be assessed from the recorded endpoint information.
Why can variability affect interpretation?
For continuous measurements, participants can vary in their underlying values and in their changes over time. Greater variation can make a group mean less precise, all else being equal. The standard deviation is one description of observed spread, but the registry gives no primary-endpoint standard deviation value. General concepts about variability therefore cannot be used to infer how variable the cortisol measurements were in this study.
Over-dispersion is a general concern for count data when variability exceeds what a chosen statistical model expects. It can make standard errors and tests unreliable if the model does not allow for the extra variability. That issue is not identified as a feature of this continuous cortisol endpoint in the registry record, and there is no basis to claim that over-dispersion affected this study’s analysis. The general concept is relevant when choosing methods for count outcomes, not as a reported finding.
What do the planning assumptions mean?
The record describes planning illustrations based on the observed effect, not the trial’s own design. It states that these illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event settings, the stated planning quantity is the total number of events; for other settings, it is participants per arm. These assumptions describe illustrations for planning, not observed power or evidence about the study’s achieved precision. Numerical planning results and the fractions used are not reported in the registry record.
The record does not report the sample-size justification used when the study was designed, whether any interim analyses were planned or conducted, or the full statistical plan. The presence of planning assumptions does not fill those gaps. Any interpretation of the primary endpoint must therefore remain limited to the recorded design and population description, rather than presumed model choices or unreported analyses.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures and 6 analyses. Multiple outcomes or analyses can raise multiplicity concerns: when several hypotheses are tested, the chance of at least one apparently unusual result can increase if each test is interpreted independently. In general, investigators may address this through a prespecified testing hierarchy, adjustment of significance thresholds, or careful designation of primary and secondary questions. The registry record does not report a multiplicity procedure, a testing hierarchy, or which analyses were considered confirmatory.
There is also an important limitation in the primary analysis population. The registry states that not all randomized participants had both a baseline and endpoint urine sample, and that only participants with both were included in the participants analyzed. It does not report how many participants lacked one or both samples, why the samples were unavailable, or whether the missingness differed across groups. It also does not report imputation, sensitivity analyses, or other approaches to assess the effect of missing data.
As a general matter, analyzing only participants with complete measurements can be vulnerable to bias if having both measurements is related to treatment assignment, outcome values, or other factors associated with the outcome. That is a general methodological concern, not evidence that bias occurred in this trial. The registry record does not contain enough information to assess the missing-data mechanism or its consequences.
The record does not report a full analysis population definition beyond the requirement for both urine samples for the primary endpoint. It does not specify whether analyses followed randomized assignment regardless of treatment received, how protocol deviations were handled, or whether any additional populations were used. These details are not reported in the registry record.
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 |
|---|---|---|---|
| MFNS 100 or 200 mcg QD | 0 | 50 | 0.0% |
| MFNS 100 or 200 mcg BID | 0 | 51 | 0.0% |
| Placebo | 0 | 26 | 0.0% |
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 |
|---|---|
| MFNS 100 or 200 mcg QD | 24 of 50 |
| MFNS 100 or 200 mcg BID | 23 of 51 |
| Placebo | 13 of 26 |
Most frequent non-serious adverse event terms
| Event term | Organ system | MFNS 100 or 200 mcg QD | MFNS 100 or 200 mcg BID | Placebo |
|---|---|---|---|---|
| HEADACHE | Nervous system disorders | 12 of 50 | 10 of 51 | 8 of 26 |
| NASOPHARYNGITIS | Infections and infestations | 6 of 50 | 6 of 51 | 6 of 26 |
| EPISTAXIS | Respiratory, thoracic and mediastinal disorders | 3 of 50 | 6 of 51 | 1 of 26 |
| INFLUENZA | Infections and infestations | 4 of 50 | 4 of 51 | 1 of 26 |
| PYREXIA | General disorders | 1 of 50 | 3 of 51 | 1 of 26 |
| VIRAL UPPER RESPIRATORY TRACT INFECTION | Infections and infestations | 1 of 50 | 2 of 51 | 2 of 26 |
| PHARYNGOLARYNGEAL PAIN | Respiratory, thoracic and mediastinal disorders | 3 of 50 | 1 of 51 | 1 of 26 |
| EAR PAIN | Ear and labyrinth disorders | 3 of 50 | 1 of 51 | 0 of 26 |
| NASAL DISCOMFORT | Respiratory, thoracic and mediastinal disorders | 1 of 50 | 0 of 51 | 2 of 26 |
The registry records serious adverse-event counts of 0 in each listed group. The corresponding numbers at risk are 50 for MFNS 100 or 200 mcg once daily, 51 for MFNS 100 or 200 mcg twice daily, and 26 for placebo. For other adverse events, the recorded counts are 24 among 50 at risk, 23 among 51 at risk, and 13 among 26 at risk, respectively.
These figures are counts of participants recorded as affected, not a description of every event or its timing. The label “other adverse events” distinguishes them from serious adverse events in the registry’s grouping, but the record does not provide the specific terms in this summary. Counts alone do not show severity within the other-events category, duration, recurrence, or whether one participant experienced multiple events. The registry record does not report deaths. These descriptive safety counts do not establish a treatment effect or provide the primary urinary cortisol results.
12. What the Result Does, and Does Not, Mean
The record establishes what the study set out to assess: safety of MFNS in pediatric subjects with nasal polyps, with change in 24-hour urinary free cortisol designated as the primary endpoint. It also records a randomized, double-masked, parallel-group design and gives limited adverse-event counts. Those details help define the study question and the kinds of data collected.
The registry record does not report the numerical primary-endpoint results. It therefore cannot show how urinary free cortisol changed from baseline, how estimates compared across groups, or how precisely any differences were estimated. No conclusion about statistical significance or the size of an effect follows from the endpoint description. The missing numerical endpoint results also mean that the listed safety counts cannot be used as a substitute for the primary safety analysis.
Even when outcome estimates are available, a statistical result and a clinical interpretation are not interchangeable. A general interpretation would consider the estimated magnitude, uncertainty, relevance of the measurement, and the completeness of follow-up. Here, the numerical estimates and uncertainty measures are not reported in the registry record, and the analyzed population excluded those without both baseline and endpoint urine samples.
The reported adverse-event counts are descriptive and do not convey event timing, event-level details, or comparative statistical analyses. They should not be read as a complete safety profile. The registry also does not report the reasons for missing urine samples, the statistical handling of missing data, or the multiplicity approach for the listed outcomes and analyses.
Accordingly, the record supports a description of the trial design, the planned safety endpoint, the complete-sample requirement for the primary analysis, and the recorded adverse-event counts. It does not support a quantitative account of the primary endpoint’s observed results or a broad conclusion about the study’s findings.
13. Why This Trial Matters Statistically
This trial illustrates why an endpoint definition and a result are different kinds of information. The registry identifies a safety-focused biomarker, its unit, and the time period over which change was assessed. Those details make the target of the primary analysis understandable, but they do not tell readers what the analysis found. Without the numerical estimate and its uncertainty, the central statistical result cannot be evaluated.
The record also illustrates how analysis populations affect interpretation. Although enrollment was 127, the primary analysis included only participants with both baseline and endpoint urine samples. The registry does not state how many met that requirement. A randomized design helps structure comparisons, but missing outcome measurements can still affect the analyzed population and its representativeness. Assessing that issue requires information about the missing measurements and the analysis approach, which is not reported in the registry record.
Finally, the available safety counts show why descriptive counts should not be confused with a complete statistical comparison. Serious adverse-event counts are recorded as 0 for each listed group, while other adverse-event counts are also reported. These summaries do not provide the primary biomarker estimates, event-level detail, or uncertainty around a group comparison.
For clinical researchers and biostatisticians, the key lesson is to separate design information, outcome definitions, analyzed populations, and numerical findings. The registry record is useful for identifying the intended safety question and some features of the study, while leaving important elements of the statistical interpretation unreported.
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 primary limitation of interpretation is that the registry record does not report numerical results for the primary endpoint. It gives no group-specific least-squares mean changes, comparison estimate, standard deviation values, confidence intervals, or p-values. Consequently, the magnitude, direction, and precision of the primary safety measure cannot be assessed.
The primary analysis required both baseline and endpoint urine samples, but the record does not state how many randomized participants lacked one or both samples or why. It does not report a missing-data method or sensitivity analysis. This prevents assessment of how the complete-sample requirement may have affected the results.
The arm descriptions are incomplete in the registry record. Group names indicate age ranges, dose levels, and once-daily or twice-daily schedules, but most descriptions do not give further details. The record also does not specify the randomization procedure, masking procedures, adherence, or the specific statistical model underlying the least-squares mean.
Further limitations include the absence of a reported multiplicity approach despite the listing of 2 outcome measures and 6 analyses, and the lack of a reported sample-size justification or detailed statistical analysis plan. The adverse-event summary provides counts but not event-level details, timing, or deaths; deaths are not reported in the registry record. These limitations restrict interpretation to the study features and counts that are explicitly recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Nasonex® for the Treatment of Nasal Polyps in Pediatric Subjects Between Ages of 6 and Less Than 18 Years Old (P04292) — NCT00378378.
- Registry record last updated 2024-05-20; status last verified 2022-02.
- Results first posted 2010-07-20.
- 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
NCT00378378 is a completed Phase 3 randomized, double-masked, parallel-group study sponsored by Organon and Co. It enrolled 127 pediatric subjects with nasal polyps, aged 6 to less than 18 years, and compared listed groups receiving mometasone furoate nasal spray with placebo nasal spray.
The primary safety endpoint was change from baseline to endpoint in 24-hour urinary free cortisol, measured in mcg/hour. The analyzed population for this endpoint required both a baseline and endpoint urine sample. The registry record does not report numerical primary-endpoint results, confidence intervals, or p-values. It records serious and other adverse-event counts by group, but does not report deaths.