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 Azithromycin for Children Hospitalized With Asthma (NCT02003911), a randomized phase 4 study. The primary outcome is Length of Stay.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02003911 |
| Phase | Phase 4 |
| Condition | Asthma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 159 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Montefiore Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Children hospitalized with acute asthma exacerbations. The registry record identifies asthma as the condition but does not report further participant characteristics, such as an age range, eligibility details, or baseline clinical profile.
Intervention
Azithromycin suspension, described as 10mg/kg/dose with a maximum of 500mg, given once daily for 3 days.
Comparator
Placebo suspension, described as the same volume as the active drug and given once daily for 3 days.
Primary question
The trial proposed to assess whether azithromycin shortened hospital length of stay for children admitted with an acute asthma exacerbation. The endpoint was the median time from admission to discharge. The registry record gives arm-level medians and interquartile ranges, but a two-arm inferential comparison for this endpoint is not reported in the registry record.
3. Trial Design
The registry describes a Phase 4, randomized, quadruple-masked, parallel-group trial comparing azithromycin suspension with placebo suspension. Its official title identifies it as a double-blind, randomized, placebo-controlled trial; the registry's masking classification is quadruple. The record does not identify which specific people or roles were masked, so the exact meaning of that classification cannot be expanded from the available information.
The registry lists enrollment as 159. The arm-level length-of-stay results are reported for 80 participants in the azithromycin arm and 79 in the placebo arm. These are the reported arm counts; the record does not explain how many participants were included in each analysis or whether any participants were excluded from particular analyses.
The intervention descriptions specify once-daily dosing for 3 days. The placebo was described as the same volume as the active suspension. The study began in 2013-10, and primary completion is recorded as 2018-06-01. Its current status is terminated. The reason for termination and any relationship between termination and the posted results are not reported in the registry record.
Montefiore Medical Center is named as sponsor. The number of study sites, the allocation procedure, the intended recruitment target beyond the recorded enrollment, and any detailed protocol deviations are not reported in the registry record. Randomization and masking describe aspects of the design, but do not by themselves establish how the outcome data were analyzed.
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 | Azithromycin Suspension | Placebo Suspension |
|---|---|---|
| STARTED | 80 | 79 |
| COMPLETED | 78 | 72 |
| NOT COMPLETED | 2 | 7 |
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 | Azithromycin Suspension | Placebo Suspension | Total |
|---|---|---|---|---|
| Participants analysed | 80 | 79 | 159 | |
| Age, Continuous | years | 7.5 (5.3 to 9.4) | 7.1 (5.2 to 9.4) | 7.3 (5.2 to 9.4) |
| Sex: Female, Male: Female | Participants | 29 | 26 | 55 |
| Sex: Female, Male: Male | Participants | 51 | 53 | 104 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 55 | 56 | 111 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 25 | 23 | 48 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| 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 | 40 | 33 | 73 |
| Race (NIH/OMB): White | Participants | 11 | 14 | 25 |
| Race (NIH/OMB): More than one race | Participants | 29 | 32 | 61 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| PASS Score on enrollment | Score on a scale | 2.35 (SD 1.25) | 2.71 (SD 1.29) | 2.53 (SD 1.28) |
6. Primary Endpoint
The registry lists the primary outcome as Length of Stay, measured over Admission time to discharge time (average LOS is 3 days).
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is hospital length of stay, measured from admission time to discharge time. The registry specifies the median as the summary measure and days as the unit. It also includes the phrase “average LOS is 3 days” in the timeframe description. That phrase does not change the stated primary summary from median to mean.
Length of stay is relevant to the study question because it directly records the interval between admission and discharge. The median gives the middle value when observed stays are ordered, making it a measure of a typical stay that is less influenced by unusually long stays than the mean can be. The interquartile range describes the span from the lower to the upper quartile and conveys variability among observed stays; it is not a confidence interval for the median or for a between-arm effect.
The reported median was 1.69 days in the azithromycin arm, with an interquartile range of 1.33 to 2.48, and 1.86 days in the placebo arm, with an interquartile range of 1.33 to 2.63. A formal comparison of the two medians, including an effect estimate and uncertainty interval, is not reported in the registry record. The arm-level summaries therefore describe the observed length-of-stay distributions but do not by themselves quantify the precision of a treatment contrast.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary results show arm-level medians of 1.69 days for azithromycin and 1.86 days for placebo. The respective interquartile ranges are 1.33 to 2.48 and 1.33 to 2.63. These summaries describe the observed length of stay in each arm. They do not establish the size or precision of a difference between arms.
The interquartile ranges overlap, but overlap between ranges of individual observations is not a test of a difference in medians. In general, a confidence interval for an effect estimates the range of values compatible with the data under an analysis, whereas an interquartile range describes the spread of participant outcomes. The registry does not report a confidence interval, p-value, hazard ratio, or other inferential comparison for the primary endpoint. It states that values were missing or not estimable for a two-arm comparison of this median-time endpoint. Thus the posted primary result is descriptive rather than a reported test of the trial's primary question.
Three secondary outcomes have numerical results posted: readmission, emergency room visits, and physician office visits, all assessed one month after discharge. Their unadjusted risk-difference intervals include zero, and their unadjusted p-values do not establish clear evidence of a between-arm difference. These are secondary comparisons, and the registry does not report an adjustment for examining multiple outcomes. They should therefore be interpreted as exploratory descriptions rather than as definitive evidence about individual outcomes.
For all of these outcomes, uncertainty matters. A non-significant or inconclusive comparison is not proof that the arms are equivalent, and the observed arm summaries do not show what would happen in a different population or under a different study design. The registry does not report a primary inferential result that can resolve the main question. The appropriate reading is limited to the recorded descriptive results and the uncertainty in the secondary estimates, not a conclusion that azithromycin does or does not alter hospital stay or post-discharge outcomes.
9. Statistical Methods Explained
What does the median describe?
For a time measure such as length of stay, the median is the midpoint of the ordered observations: half of the recorded stays fall at or below it and half at or above it, subject to ties. It summarizes the center of the observed distribution. The interquartile range runs from the lower quartile to the upper quartile and describes the central spread of individual observations. In this trial, the registry reports medians and interquartile ranges for each arm. These quantities are not measures of uncertainty around an arm-to-arm effect.
General statistical context: the mean can be pulled by a small number of unusually long stays, while the median is less sensitive to such values. That can make a median a useful summary when a time outcome is skewed. However, choosing a median does not by itself specify the statistical test or model used to compare arms. The method for a formal primary comparison is not reported in the registry record.
Why is an effect estimate useful?
A between-arm effect estimate expresses the size and direction of a contrast using a defined measure. A confidence interval communicates uncertainty around that estimate. For a median-based endpoint, the analysis might report a suitable contrast and interval, but the choice depends on the analysis plan and data. The registry's posted primary results do not include such an estimate or interval, and it notes that a two-arm comparison was not estimable from the posted median-time endpoint values.
General statistical context: a p-value, when available, measures how unusual data at least as incompatible with a specified null hypothesis would be under the assumptions of a statistical test. It does not measure the probability that the null hypothesis is true, the size of an effect, or its practical importance. No primary p-value is reported in the registry record, so one should not infer a primary test result from the difference between the two arm medians.
How should the secondary comparisons be read?
The posted secondary comparisons use risk differences for binary outcomes. A risk difference is the contrast between the proportions with an outcome in the two arms, expressed on the probability scale. A confidence interval that includes zero is compatible with no risk difference as well as with differences in either direction at the interval's stated level, subject to the method's assumptions. The registry presents these secondary p-values as unadjusted. Unadjusted results do not account for the number of outcomes examined.
General statistical context: estimates based on relatively few observed events can be imprecise. The width of a confidence interval is a useful guide to that imprecision; a point estimate alone can give a misleading impression of certainty. The registry's secondary intervals are broad enough that their endpoints allow effects in either direction. The posted results should therefore be read with their uncertainty rather than as precise estimates of an enduring effect.
What do the planning illustrations mean?
The posted sample-size illustrations are calculations based on observed secondary effects, not the design assumptions or sample-size justification for the trial itself. They use a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation, and consider detecting stated fractions of the observed effects. These are planning illustrations, not observed power and not evidence that the trial was designed to detect those effects.
General statistical context: a planning calculation depends on assumptions about the effect worth detecting, outcome frequency or variability, significance level, allocation, and analysis. If an observed effect is small or unstable, using it as the basis for a future calculation can produce highly variable planning estimates. The registry does not report the original sample-size justification or the assumptions used in the trial's own design. The posted illustrations cannot substitute for that missing design information.
For binary outcomes, the illustrations express required sample sizes as participants per arm. The registry also explains that time-to-event planning illustrations, when present, use total event counts; that convention does not supply a primary event-based comparison for this trial. The primary length-of-stay comparison remains unreported as an inferential analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry names seven secondary outcomes, covering readmission, school missed, work missed, emergency room visits, physician office visits, recurrence of asthma symptoms, and steroid courses. Numerical results are posted for readmission, emergency room visits, and physician office visits. Results for school missed, work missed, recurrence of asthma symptoms, and steroid courses are not reported in the registry record. The record does not explain why numerical results are absent for those outcomes.
A statistical analysis plan, a prespecified hierarchy among outcomes, and a method for addressing multiplicity are not reported in the registry record. The three posted secondary comparisons are unadjusted. Without a reported plan for controlling or otherwise interpreting the number of comparisons, individual secondary p-values should not be treated as if they were the sole planned test. The registry also does not report a primary hypothesis test for length of stay.
Missing-data handling is not reported in the registry record. The record does not describe whether outcomes were missing, how missing observations were handled, or whether any sensitivity analyses were performed. Analysis populations—such as whether analyses followed assigned treatment regardless of adherence—are not reported in the registry record. The arm counts associated with the posted results do not, by themselves, establish the analysis population or completeness of follow-up.
The record reports a terminated status, but the reason for termination, whether recruitment stopped as planned, and whether termination affected outcome collection are not reported in the registry record. Interim analyses, stopping rules, and changes to the analysis plan are also not reported in the registry record. These omissions limit assessment of how the posted outcomes were selected and analyzed; they do not justify assuming that any particular procedure 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 |
|---|---|---|---|
| Azithromycin Suspension | 0 | 80 | 0.0% |
| Placebo Suspension | 1 | 79 | 1.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Azithromycin Suspension | 0 of 80 |
| Placebo Suspension | 0 of 79 |
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 |
|---|---|
| Azithromycin Suspension | 2 of 80 |
| Placebo Suspension | 7 of 79 |
Most frequent serious adverse event terms
| Event term | Organ system | Azithromycin Suspension | Placebo Suspension |
|---|---|---|---|
| Transfer to PICU | Respiratory, thoracic and mediastinal disorders | 0 of 80 | 1 of 79 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Azithromycin Suspension | Placebo Suspension |
|---|---|---|---|
| Study Exit | Social circumstances | 2 of 80 | 7 of 79 |
Serious events with the highest arm-level rate: transfer to picu (1.3%).
The registry reports serious adverse events for 0 of 80 participants in the azithromycin arm and 1 of 79 participants in the placebo arm. It records no deaths in either arm. The top serious-event term listed is “Transfer to PICU,” recorded as 1.3%. The registry does not provide additional detail in the posted serious-event summary about the clinical course or outcome of that event.
Other, non-serious adverse events are reported for 2 of 80 participants in the azithromycin arm and 7 of 79 participants in the placebo arm. These counts should be kept distinct from the serious-event counts. The registry summary does not establish whether each participant had only one event, describe event severity beyond the serious/non-serious categories, or provide a detailed account of how events were collected.
These figures are descriptive counts within the posted arm results. With few serious events, the record does not support a precise comparison of serious-event risk. The counts alone do not establish causation, and the registry's safety summary should not be read as a complete account of every possible adverse effect.
12. What the Result Does, and Does Not, Mean
The record describes a randomized, masked comparison of azithromycin suspension with placebo suspension in children hospitalized with acute asthma exacerbations. Its primary outcome was median time from admission to discharge. The reported arm medians and interquartile ranges show the observed summaries, but the registry does not report an inferential two-arm comparison for this endpoint. The central question about a between-arm difference in length of stay therefore remains unresolved by the posted primary analysis.
The three numerical secondary outcomes provide limited post-discharge information. Their unadjusted estimates are uncertain, and the registry does not report a multiplicity procedure. Outcomes named in the record but lacking numerical results cannot be interpreted as showing either a difference or no difference. The same caution applies to the safety counts: they describe recorded events but are too sparse to provide a precise comparative safety estimate.
General statistical context: absence of a statistically clear result does not prove equivalence, and a numerical difference between arm summaries does not alone establish a meaningful effect. A strong interpretation would require an appropriate effect estimate, its uncertainty, a clear analysis population, and information about how outcomes and missing data were handled. For this trial, several of those elements are not reported in the registry record.
The results should not be extended beyond the population, intervention, comparator, outcomes, and follow-up described in the record. In particular, the secondary outcomes are specified for one month after discharge, and the record does not provide numerical results for all named secondary measures. The posted information supports a description of the trial and its recorded summaries, not a general conclusion about treatment effects in other settings or populations.
13. Why This Trial Matters Statistically
This trial is statistically instructive because the primary endpoint is reported using a median and interquartile range, while the primary between-arm inferential comparison is not reported. Arm-level summaries can show the distribution's center and spread, but they cannot substitute for an effect estimate with uncertainty. The distinction matters whenever readers are tempted to treat a visible numerical contrast as a tested treatment effect.
The record also illustrates why outcome hierarchy and multiplicity matter. Several secondary outcomes are named, some have numerical results, and the posted comparisons are unadjusted. A collection of individual comparisons can be harder to interpret than a single prespecified primary analysis, particularly when a correction or interpretation plan is not reported. The secondary findings therefore need to be read as exploratory rather than as a set of independent confirmations.
Finally, the sample-size illustrations are not the trial's original design justification and do not report observed power. They are based on observed secondary effects under stated assumptions. Keeping planning calculations separate from the original trial design and from observed results prevents a common interpretive error: treating a post hoc illustration as evidence that the study was adequately powered or that a particular effect was ruled out.
More broadly, this record shows how completeness of reporting affects what can be learned. The design and arm-level summaries are available, but analysis populations, missing-data methods, multiplicity handling, and a primary comparison are not reported in the registry record. Those omissions constrain the precision and scope of statistical conclusions, even when the descriptive outcome values themselves are clear.
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 limitations are specific to the record. The study status is terminated, but the reason for termination and its implications for recruitment or follow-up are not reported in the registry record. Termination alone does not establish that the study stopped early or that it affected the analysis, but the missing explanation prevents readers from assessing those possibilities.
The primary endpoint is summarized by arm, yet a formal comparison is not reported. There is no posted primary effect estimate, confidence interval, or p-value to quantify the contrast. In addition, the registry does not report the analysis population, the handling of missing observations, or any sensitivity analyses. These gaps prevent assessment of whether the posted summaries represent all randomized participants or a subset.
Numerical secondary results are available for only some of the outcomes named in the record. The listed post-discharge timeframe is one month after discharge for the outcomes with posted comparisons. Numerical findings for the other named secondary measures are not reported in the registry record, and outcomes beyond the stated timeframe are not characterized by those results.
Participant characteristics beyond the description of children hospitalized with acute asthma exacerbations are not reported in the registry record. The number of sites and details of the allocation process are also not reported. The sponsor is named, but that does not establish the setting or breadth of recruitment. These reporting limits constrain judgments about how well the results might apply outside the trial population described in the record.
Finally, the secondary comparisons are unadjusted, and the registry does not report a multiplicity plan. The combination of uncertain estimates, incomplete numerical results across named outcomes, and absent analysis details means that the posted results should be interpreted cautiously without inferring unreported procedures or conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Azithromycin for Children Hospitalized With Asthma — NCT02003911.
- Registry record last updated 2020-08-13; status last verified 2020-08.
- Results first posted 2020-08-13.
- 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
NCT02003911 is a Phase 4, randomized, quadruple-masked, parallel-group trial sponsored by Montefiore Medical Center. It compared azithromycin suspension with placebo suspension in children hospitalized with acute asthma exacerbations. The registry lists enrollment as 159 and records the study status as terminated.
The primary outcome was median hospital length of stay from admission to discharge. Arm-level medians and interquartile ranges are posted, but a two-arm inferential comparison is not reported in the registry record. Numerical results are also posted for three post-discharge secondary outcomes; other named secondary outcomes lack numerical results in the registry record. The record does not report the reason for termination, the analysis populations, missing-data handling, or a multiplicity plan.