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 Effect of Montelukast Therapy in Obstructive Sleep Apnea(OSA) Children (NCT01027806), a randomized phase 4 study. The primary outcome is The Percentage of Children Experiencing a Reduction in Apnea-Hypopnea Index (API) From Baseline (Before Treatment) to After Treatment..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01027806 |
| Phase | Phase 4 |
| Condition | Obstructive Sleep Apnea |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 64 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Chicago |
| Results posted | Yes |
2. Clinical Question
Population
Children with obstructive sleep apnea. The registry summary frames the leading hypothesis around children with mild to moderate obstructive sleep apnea syndrome who require removal of the adenoids and tonsils for the condition. Whether those statements define the trial’s actual eligibility criteria is not reported in the registry record.
Intervention
Montelukast, listed as a drug intervention. The registry summary describes oral therapy. Dose, schedule, and treatment adherence details are not reported in the registry record.
Comparator
Placebo. The record lists Montelukast and Placebo as the two arms, but does not provide further descriptions of the arms.
Primary question
Among children with obstructive sleep apnea, does assignment to montelukast rather than placebo correspond to a reduction in the apnea-hypopnea index from baseline to after treatment? The listed timeframe is 16 weeks. The registry record does not report the outcome counts or a single comparison that would answer this question.
The registry summary also states a hypothesis that treatment could reduce the severity of obstructive sleep apnea and remove the need for surgical intervention. That is a stated hypothesis, not a reported trial finding. The primary outcome listed in the record concerns reduction in the index, and a result about surgical need is not reported in the registry record. The record identifies the study as terminated, but does not report why it ended or whether the stated hypotheses were evaluated as planned.
3. Trial Design
The record identifies NCT01027806 as a Phase 4, randomized, triple-masked, parallel-group trial. It lists montelukast and placebo as the arms. These design labels describe the planned structure, but the record does not identify which groups of people were masked or give details of the allocation procedure. Although randomization is listed, the allocation ratio is not reported in the registry record. Equal allocation appears as an assumption for planning illustrations, not as a reported feature of the trial’s actual assignment.
The registered enrollment is 64. That figure is not necessarily the number of participants included in the outcome analysis: the registry record does not report how many were randomized, received treatment, completed follow-up, or entered the primary analysis. For the adverse-event entries, the numbers listed at risk are 28 for Montelukast and 29 for Placebo. The record does not explain how those denominators relate to registered enrollment or to the outcome population.
The condition is obstructive sleep apnea, and the listed intervention is montelukast. The registry summary describes a hypothesis involving children with mild to moderate obstructive sleep apnea syndrome who require adenoid and tonsil removal. Specific eligibility criteria, participant ages, recruitment setting, and baseline characteristics are not reported in the registry record. The treatment description is oral therapy in the summary, but dose, duration beyond the outcome timeframe, adherence procedures, and co-interventions are not reported.
The listed timeframe for the primary outcome is 16 weeks. The record gives a start of 2010-07 and a primary completion of 2014-08, and the study status is terminated. A termination reason, planned follow-up schedule, and details of any changes to the design are not reported. The dates alone do not establish why recruitment or follow-up ended, or how much outcome information was collected.
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 | Montelukast | Placebo |
|---|---|---|
| STARTED | 32 | 32 |
| COMPLETED | 28 | 29 |
| NOT COMPLETED | 4 | 3 |
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: Patients who completed the study
| Characteristic | Unit | Montelukast | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 28 | 29 | 57 | |
| Age, Continuous | years | 5.5 (SD 2.5) | 5.6 (SD 2.4) | 5.6 (SD 2.5) |
| Sex: Female, Male: Female | Participants | 14 | 15 | 29 |
| Sex: Female, Male: Male | Participants | 14 | 14 | 28 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 12 | 12 | 24 |
| Race (NIH/OMB): White | Participants | 16 | 17 | 33 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 28 | 29 | 57 |
6. Primary Endpoint
The registry lists the primary outcome as The Percentage of Children Experiencing a Reduction in Apnea-Hypopnea Index (API) From Baseline (Before Treatment) to After Treatment., measured over 16 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is titled as the percentage of children experiencing a reduction in the Apnea-Hypopnea Index (API) from baseline, before treatment, to after treatment. The stated timeframe is 16 weeks. In practical terms, the endpoint asks whether a participant’s index was lower after treatment than at baseline, rather than describing only a single measurement at one time. The registry record identifies the measure as a participant count, while its title describes a percentage. The outcome counts and the percentage are not reported in the registry record.
A change from baseline can describe whether participants experienced the direction of change specified by the endpoint. In general, a binary endpoint classifies participants according to whether they meet a stated condition, here a reduction. It does not, by itself, describe how large each participant’s change was. The registry record does not define the threshold or calculation used to decide whether an individual experienced a reduction, nor does it give the underlying index values.
The endpoint is relevant to the stated hypothesis because the summary proposes that montelukast may improve sleep-study findings and reduce obstructive sleep apnea severity. However, the endpoint title alone does not establish how an index reduction relates to surgical decisions or symptoms. A result about the need for surgery is not reported as the primary endpoint result. The record indicates that the endpoint has multiple categories or time points and is not reduced to one comparison, so a single treatment contrast cannot be interpreted from the posted information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the trial’s question and design, but it does not provide a single primary-outcome result that can be interpreted as a comparison between montelukast and placebo. The primary outcome is described as a reduction in the apnea-hypopnea index from baseline after treatment, with a 16 weeks timeframe. However, the record indicates that the outcome has several categories or time points and is not reduced to one comparison. The outcome counts, group-specific percentages, effect estimate, and uncertainty around an estimate are not reported in the registry record.
Consequently, the posted information does not establish how often a reduction occurred in either arm, how the arms compared, or how precisely any difference was estimated. A statement that one treatment group had more participants meeting the endpoint would require group outcome counts, which are not reported. No conclusion about the treatment effect can be drawn from the endpoint title or from the hypotheses in the summary. The study’s terminated status also does not reveal whether the missing comparison reflects incomplete data, a reporting choice, or another circumstance; a reason for termination is not reported.
The enrolled number is 64, but enrollment alone does not indicate the number analyzed. The registry record does not report the analysis population or the number of participants with outcome data. The adverse-event denominators are listed separately and should not be substituted for primary-outcome denominators. No confidence interval, p-value, or other uncertainty measure for the primary endpoint is reported in the registry record.
In general, an estimate describes the magnitude and direction of a contrast, while a confidence interval expresses the range of values reasonably compatible with the data under the method used. Without an estimate and its uncertainty, statistical precision cannot be judged. A missing single comparison is not evidence of no difference; it means the posted record does not permit that comparison to be evaluated. Likewise, the study’s stated hypotheses do not constitute evidence that the predicted outcomes occurred.
The safety entries show zero serious adverse events recorded for each arm, with other adverse-event counts of 2 for Montelukast and 3 for Placebo. These are descriptive counts in the registry record. They do not supply a primary efficacy result and, without additional definitions and analysis details, do not establish comparative safety. Deaths are not reported in the registry record.
9. Statistical Methods Explained
The registered design is randomized, triple-masked, and parallel-group, with montelukast and placebo arms. In a randomized trial, assignment is determined through a random process intended to reduce systematic differences between groups at the start. That general purpose does not guarantee that the groups will be identical, and it does not replace a description of baseline characteristics. The registry record does not report the allocation sequence, allocation ratio, implementation procedures, or baseline balance. The label triple-masked indicates masking at three levels, but which participants, clinicians, assessors, or other personnel were masked is not reported in the registry record.
What does the endpoint measure?
The endpoint is a participant count for a reduction in the Apnea-Hypopnea Index (API) between baseline and after treatment. The title describes the endpoint as a percentage, while the registered measure is a count of participants. In general, a binary outcome records whether each participant meets a defined condition, then summarizes the number meeting it. The registry record does not report the counts or percentages by arm, and the rule used to classify a reduction is not reported. The record also notes multiple categories or time points and does not reduce them to one comparison, which prevents a clear arm-to-arm interpretation.
Why are an effect estimate and uncertainty important?
In general, an effect estimate expresses the observed contrast between trial groups on a defined outcome. The estimate’s uncertainty indicates how much the data support a range of possible values, rather than only one point estimate. A confidence interval is one common way to display that uncertainty. A p-value, when reported, addresses compatibility between observed data and a specified null model; it does not measure the size or clinical importance of an effect. No primary-outcome effect estimate, confidence interval, or p-value is reported in the registry record. It is therefore not possible to describe the magnitude, precision, or statistical evidence for a treatment contrast.
What does the analysis plan tell readers?
A statistical analysis plan ordinarily identifies the outcome definition, analysis population, model or test, handling of missing observations, and approach to multiplicity. Those details help readers understand which comparison was intended and how estimates were produced. The registry record lists one analysis, but the analysis method, analysis population, covariate adjustment, and missing-data approach are not reported. The listing of an analysis does not reveal which participants were included or which outcome categories and time points were selected.
For a binary outcome, methods may compare event proportions or model the probability of meeting the endpoint. These are general possibilities, not methods identified for this trial. Different methods can produce different estimands and uncertainty estimates, particularly when outcome data are incomplete or analysis populations differ. No specific method should be attributed to this trial on the basis of the registry record alone.
Why can over-dispersion matter?
In general, over-dispersion occurs when the variability in data is greater than a model assumes. It can make standard errors too small if the model does not account for the extra variability, leading to uncertainty that is understated. This issue is relevant only when the outcome and analysis model call for a variance model susceptible to over-dispersion. The registry record does not identify a model for the primary endpoint or report a dispersion estimate, so whether over-dispersion was relevant to this trial is not reported in the registry record.
How should planning illustrations be read?
The record describes sample-size planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. It states that the effect to detect is a specified fraction of the observed effect. These are planning assumptions, not evidence that the trial was designed using those assumptions, and any sample-size illustration is not observed power. The record does not report the trial’s own sample-size justification. Because the primary outcome is not reduced to one comparison and no interpretable effect estimate is reported, the planning assumptions should not be mistaken for evidence about the trial’s observed treatment effect.
10. Multiplicity, Missing Data and Analysis Populations
The primary outcome is flagged in the registry record as having several categories or time points and not being reduced to one comparison. That matters because interpreting multiple tests or selected time points can create multiplicity: the more comparisons considered, the greater the chance that an apparently notable finding arises by chance if no adjustment or prespecified selection approach is used. In general, a multiplicity strategy may define a primary contrast, adjust inference across tests, or specify a hierarchy of outcomes. The registry record does not report such a strategy, the particular categories and time points, or which comparison was intended to be primary.
The registry record does not report missing-data counts for the primary outcome, reasons for missingness, or methods for handling missing observations. In general, missingness can affect both the number of participants analyzed and the validity of an estimate, depending on why observations are absent and how the analysis handles them. Registered enrollment of 64 does not establish that all enrolled participants contributed primary-outcome data. The analysis population and numbers analyzed are not reported in the registry record.
Similarly, the record does not report whether the primary analysis followed randomized assignment, included only participants who began treatment, or used another population. It lists one analysis, but this does not specify its population or methods. Without these details and the outcome results, readers cannot assess the effects of multiplicity choices, missing data, or analysis-population definitions on the primary comparison.
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 |
|---|---|---|---|
| Montelukast | 0 | 28 | 0.0% |
| Placebo | 0 | 29 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Montelukast | 2 of 28 |
| Placebo | 3 of 29 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Montelukast | Placebo |
|---|---|---|---|
| Nausea | General disorders | 1 of 28 | 2 of 29 |
| Headache | General disorders | 1 of 28 | 1 of 29 |
The registry lists serious adverse events for 28 participants at risk in the Montelukast group and 29 at risk in the Placebo group. The recorded number affected by a serious adverse event is 0 in each group. For other adverse events, 28 participants are listed at risk in the Montelukast group, with 2 affected, and 29 are listed at risk in the Placebo group, with 3 affected. These are counts as recorded; no further event descriptions or analysis are reported in the registry record.
The at-risk denominators in these safety entries are not the registered enrollment of 64, and the registry record does not explain the relationship between them. They should be read as the denominators attached to the specific adverse-event entries, not assumed to represent the number randomized or analyzed for the primary outcome. The term “other” does not provide details about event type, severity, timing, or whether events were considered related to treatment.
Deaths are not reported in the registry record; a blank death entry should not be read as a recorded count of zero. These safety counts are descriptive and do not by themselves establish a comparative safety effect. Follow-up duration for safety, event-collection methods, and statistical comparisons are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record describes a randomized comparison of montelukast with placebo in children with obstructive sleep apnea, and identifies reduction in the apnea-hypopnea index after treatment as the primary outcome. It also records a 16 weeks timeframe, a triple-masked parallel design, and a terminated status. These registry entries establish what the trial was intended to examine and some features of its design. They do not establish the outcome of the comparison.
The primary result cannot be interpreted from the posted record because outcome counts and a single comparison are not reported. The record specifically indicates multiple categories or time points without reducing them to one comparison. There is no reported group-specific endpoint result, effect estimate, confidence interval, or p-value. It is not possible to say from this record whether participants in either arm experienced a reduction more often, whether the groups differed, or how precise an estimated difference would be.
The summary’s statements about possible improvements in sleep-study findings and avoiding surgery are hypotheses. They should not be read as findings. The listed primary endpoint is a reduction in an index, and a result for surgical intervention is not reported in the registry record. The record also does not explain why the study was terminated, so termination alone cannot be interpreted as evidence for or against a treatment effect.
The adverse-event entries report no serious adverse events recorded in either arm and list other adverse-event counts of 2 and 3, respectively. These observations are limited to the registry’s counts and denominators. They do not establish that no other events occurred beyond those recorded, and they do not provide a complete comparative safety analysis. Deaths are not reported in the registry record.
In general, a trial result is most informative when the outcome definition, analysis population, group-specific results, estimated contrast, and uncertainty are available together. The record leaves these elements incomplete for the primary question. The appropriate interpretation is therefore limited: the trial was registered to study the stated comparison, but the posted results do not provide an interpretable primary-outcome conclusion.
13. Why This Trial Matters Statistically
This trial illustrates the difference between a registered research question and an interpretable statistical result. The record identifies a randomized, triple-masked, parallel-group design and names montelukast and placebo as the arms. It also states a primary endpoint and timeframe. Those details help define what investigators planned to study, but they do not substitute for group-specific outcome data and an analysis that connects the outcome to the randomized comparison.
The endpoint’s wording raises a useful reporting issue: it is titled as a percentage of children experiencing a reduction, while its measure is listed as a participant count. A count can be converted to a percentage only with a clearly specified denominator. The record gives no primary-outcome counts or denominators, and it also indicates that multiple categories or time points were not reduced to one comparison. Without a clear outcome definition and comparison, readers cannot tell what an apparent difference would represent.
The record also underscores why enrollment should not be confused with analysis size. Enrollment is 64, while the adverse-event entries list separate at-risk counts of 28 and 29. The primary-outcome analysis population is not reported. Knowing the registered enrollment alone cannot show how many participants contributed outcome information or how missing observations were addressed.
Finally, statistical interpretation depends on both an estimate and its uncertainty. Neither is reported for the primary comparison. The registered hypotheses and design features cannot fill that gap, and the terminated status does not explain it. Careful reading separates what is known about the trial’s structure from what the posted record can support about its results.
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.
Several trial-specific details needed to interpret the primary outcome are not reported in the registry record. These include the outcome counts by arm, a single comparison across the listed categories or time points, the definition used to classify a participant as experiencing a reduction, and the primary-outcome analysis population. The record therefore does not support an estimate of the treatment contrast or its precision.
The registry reports enrollment of 64, but does not report the number randomized, treated, followed, or included in the primary analysis. It does not describe baseline characteristics, eligibility criteria in detail, participant ages, or the setting. The summary’s description of children with mild to moderate disease who require adenoid and tonsil removal is framed as part of the investigators’ hypothesis; whether it represents the full enrolled population is not reported.
Although the study is identified as randomized, triple-masked, and parallel-group, the allocation ratio and masking details are not reported. The record does not give the treatment dose, administration schedule, adherence information, or co-intervention details. It also does not provide the statistical method, missing-data approach, multiplicity strategy, or rationale for the sample size. The planning assumptions described in the record do not establish the trial’s own design justification.
The status is terminated, but the reason is not reported in the registry record. The listed adverse-event counts are accompanied by at-risk denominators, but event descriptions, collection methods, timing, and an explanation of the denominators are not reported. Deaths are not reported. These limitations prevent a full appraisal of both efficacy and safety from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effect of Montelukast Therapy in Obstructive Sleep Apnea(OSA) Children — NCT01027806.
- Registry record last updated 2019-05-03; status last verified 2019-04.
- Results first posted 2019-05-03.
- 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
NCT01027806 is a Phase 4 randomized, triple-masked, parallel-group trial of montelukast versus placebo for children with obstructive sleep apnea. Registered enrollment is 64. The study status is terminated; the listed start is 2010-07 and primary completion is 2014-08.
The primary outcome is described as the percentage of children with a reduction in the Apnea-Hypopnea Index (API) from baseline after treatment, with a timeframe of 16 weeks. The measure is also listed as a participant count. The registry indicates multiple categories or time points and does not reduce the outcome to one comparison. Primary outcome counts and an interpretable treatment contrast are not reported in the registry record.
Safety entries record 0 serious adverse events in each arm; other adverse-event counts are 2 for Montelukast and 3 for Placebo. Deaths are not reported in the registry record. The reason for termination, primary analysis population, missing-data handling, and statistical analysis methods are not reported.