Study of DA-9501 In Pediatric Subjects In The Intensive Care Unit: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of DA-9501 In Pediatric Subjects In The Intensive Care Unit (NCT02757625), a na phase 3 study. The primary outcome is Percentage of Participants Who Did Not Require a Rescue Sedative Within 24 Hours of Dosing of Study Drug.

2. Clinical Question

The study was designed to evaluate efficacy, safety, and pharmacokinetics in pediatric intensive care patients. Its primary endpoint focuses on whether rescue sedative medication was needed, rather than on a direct comparison of sedation scores between treatment groups. That makes the endpoint a measure of observed rescue-medication use in the study group under the stated assessment conditions; it does not, on its own, establish how outcomes would compare under a different treatment.

3. Trial Design

This was a phase 3, multicenter, single-arm, open-label study of dexmedetomidine hydrochloride in pediatric intensive care patients. The registry lists Pfizer as the sponsor, an enrollment of 63, and a completed status. The recorded start date is 2016-07-20 and the primary completion date is 2017-05-24.

The intervention was dexmedetomidine hydrochloride, administered as a continuous intravenous infusion according to the study summary. The registry describes a vial containing 2 mL of a solution with 100 µg/mL of dexmedetomidine in physiological saline. The record does not report a full dosing schedule in the study details summarized for this analysis.

There was no control arm or randomized allocation. The study therefore describes outcomes among participants receiving the study drug, rather than an estimated treatment difference between groups. Its open-label design also means that treatment masking was absent. These design features matter when interpreting outcomes that can depend on clinical judgment, including the decision to give rescue sedation.

The primary endpoint was assessed from the start of study drug administration on Day 1 until the earliest of 24 hours of study drug dosing, the conclusion of mechanical ventilation, or the end of study drug administration, with a maximum timeframe of 28 days. The endpoint was the percentage who did not require rescue sedative medication in that period. The full analysis set was defined as participants who received at least one dose of study drug. The registry record does not report the primary endpoint result, the confidence interval, or detailed statistical analysis procedures.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: FAS included all participants who received at least one dose of the study drug.

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: The Full Analysis Set (FAS) included all participants who have received at least one dose of the study drug.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants Who Did Not Require a Rescue Sedative Within 24 Hours of Dosing of Study Drug, measured over From start of study drug administration on Day 1 up to 24 hours of study drug dosing or at the conclusion of mechanical ventilation or the end of study drug administration, whichever is earliest (up to maximum of 28 days).

The primary endpoint measures the percentage of participants who did not require rescue sedation with midazolam during the defined observation period. The period began with study drug administration on Day 1 and ended at the earliest of 24 hours of study drug dosing, the conclusion of mechanical ventilation, or the end of study drug administration; the maximum timeframe was 28 days. The endpoint description says that rescue use was assessed using investigator judgment and the State Behavioral Scale for intubated participants.

The scale ranges from 2 to -3: 2 indicates agitation, 1 indicates restlessness and difficulty calming, 0 indicates awake and able to calm, -1 indicates responsiveness to gentle touch or voice, -2 indicates responsiveness to noxious stimuli, and -3 indicates unresponsiveness. The target sedation range was -2 to 0 during intubation and -1 to 0 after extubation. Higher scores indicated greater responsiveness.

As a general statistical interpretation, a “no rescue medication” endpoint can describe how often additional sedative treatment was not needed under the study’s clinical assessment and observation rules. It is not the same as a direct measure of a participant’s sedation state at every moment. The registry record does not report the observed percentage or its 95% confidence interval, so the frequency and precision of this endpoint cannot be assessed from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The key statistical distinction is between estimating an outcome in one group and comparing outcomes between groups. This study had a single group and no randomized comparator. Its primary endpoint is a percentage: the number of participants meeting the “did not require rescue sedative” definition relative to the relevant analysis population. The registry record does not give that percentage, its numerator or denominator, or its confidence interval.

What does a confidence interval describe?

General explanation: a confidence interval expresses uncertainty around an estimate, using a procedure intended to produce intervals that capture the underlying quantity at the stated rate over repeated applications. A 95% confidence interval is a measure of statistical precision, not a guarantee that a particular interval contains the true value. For a percentage, the interval’s width depends in part on the amount of information available and on the observed proportion. The registry identifies a 95% confidence interval for the primary endpoint but does not report its limits or the method used to construct it.

A confidence interval around a single-group percentage would describe uncertainty in that group’s endpoint rate under the study’s definitions. It would not become a treatment-effect interval, because there is no second group and no between-group contrast. The distinction matters: a precise estimate of a single-group rate can still leave the causal effect of treatment unknown.

Why does a single-group design matter?

General explanation: random allocation to a concurrent comparator can help balance both measured and unmeasured factors between groups, supporting a more direct comparison. In a single-group study, changes or outcomes may also reflect the illness course, other care, patient characteristics, or decisions made during follow-up. Without a comparator, these influences cannot be separated from the study treatment using a randomized contrast. The record identifies allocation as not applicable and the model as single group; it does not describe another comparative design.

The endpoint is based on rescue medication use, with investigator judgment and the State Behavioral Scale informing assessment. The record states that target sedation was -2 to 0 during intubation and -1 to 0 after extubation. General explanation: an endpoint that incorporates clinical decisions may reflect both patient state and the care process, so the operational definitions and assessment context are important when interpreting its rate. The record does not report masking, which is listed as none, or any procedure to reduce assessment influence.

What can be said about the analysis population?

The full analysis set included all participants who received at least one dose of study drug. This definition identifies who was intended to contribute to that population, but it does not establish how many participants were included in the primary endpoint analysis or whether all had complete endpoint observations. The enrolled total was 63. The registry does not report a primary endpoint denominator, exclusions from analysis, or the handling of missing data.

General explanation: when a proportion is reported, its denominator is essential. If some participants lack endpoint information, the method used to address those observations can affect both the estimated proportion and its uncertainty. Common approaches have different assumptions; no particular approach can be attributed to this study because the registry record does not report one.

What is not described statistically?

The record does not report the statistical method for calculating the primary estimate or its confidence interval, any covariate adjustment, an analysis plan, interim analyses, or a sample-size justification. It lists 29 outcome measures and no analyses. That does not establish whether analyses were conducted outside the registry record; it means the record does not report them. Statistical conclusions about the posted endpoint therefore cannot be reconstructed from the registry information alone.

General explanation: when multiple outcomes are assessed, the chance of at least one apparently unusual result can rise if many hypothesis tests are performed. Whether multiplicity adjustment is needed depends on the intended inferential claims and analysis plan. No testing strategy or multiplicity procedure is reported, and no outcome analyses are listed. The presence of multiple registered outcomes is not itself evidence that any result is significant or that an adjustment was or was not used.

The sample-size assumptions in the record describe a planning illustration based on an observed effect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. Such assumptions are not evidence of the study’s original design rationale, and the illustration does not correspond to the registered single-group design. The registry record does not report the study’s own sample-size justification. Any planning figures should be understood as illustrations, not as observed power or as a substitute for trial results.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Dexmedetomidine: >=45 Weeks CGA to <12 Months", "Dexmedetomidine: >=12 Months to <24 Months", "Dexmedetomidine: >=2 Years to <6 Years", "Dexmedetomidine: >=6 Years to <17 Years", "Dexmedetomidine: All Participants")
n      <- c(14, 18, 19, 12, 63)
events <- c(11, 12, 15, 11, 49)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Dexmedetomidine: >=45 Weeks CGA to <12 Months  14  11  78.6  49.2  95.3  52.4  92.4  51.9  92.2
ARM  Dexmedetomidine: >=12 Months to <24 Months  18  12  66.7  41.0  86.7  43.7  83.7  43.4  83.7
ARM  Dexmedetomidine: >=2 Years to <6 Years  19  15  78.9  54.4  93.9  56.7  91.5  56.3  91.3
ARM  Dexmedetomidine: >=6 Years to <17 Years  12  11  91.7  61.5  99.8  64.6  98.5  64.0  98.1
ARM  Dexmedetomidine: All Participants  63  49  77.8  65.5  87.3  66.1  86.3  66.0  86.2

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 29 outcome measures, including the primary measure of avoiding rescue sedation and secondary measures involving rescue sedative use, rescue analgesic use, and maintenance of the target sedation level. It lists no analyses. Results for these outcomes and the statistical plan for interpreting them are not reported in the registry record.

General explanation: assessing many outcomes can raise multiplicity concerns when each is tested as a separate hypothesis. A prespecified testing hierarchy or adjustment can be used to control the chance of misleading findings, but an appropriate approach depends on the study’s inferential goals. The record does not report a multiplicity strategy, a hierarchy among outcomes, or whether formal hypothesis tests were planned. No conclusion about adjustment can therefore be drawn.

The full analysis set included participants who received at least one dose of study drug. The record does not give the number included in the primary endpoint analysis, the number with missing endpoint data, or the handling of missing observations. It also does not report whether other analysis populations were defined. These omissions matter because the denominator and missing-data method affect interpretation of a percentage and its precision.

General explanation: missing outcomes can make a reported rate less representative if the likelihood of missingness is related to participant status or outcome. Methods for dealing with missing information rely on assumptions, and the assumptions should be made clear. No missing-data assumptions or sensitivity analyses are reported for this study. The absence of these details in the registry record should not be taken as evidence that missing data occurred, nor as evidence that they did not.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cardiac tamponade (5.3%).

For all participants, the registry reports 63 at risk for serious adverse events, 1 participant affected, and 0 deaths. For other adverse events, it reports 63 at risk and 55 affected. These are counts reported in the registry record; the record does not provide a comparative safety estimate because the study had no control group.

The age-stratified serious adverse event counts were 0 among 14 participants aged at least 45 weeks corrected gestational age to under 12 months, 0 among 18 participants aged at least 12 months to under 24 months, 1 among 19 participants aged at least 2 years to under 6 years, and 0 among 12 participants aged at least 6 years to under 17 years. The corresponding other adverse event counts were 11 of 14, 16 of 18, 16 of 19, and 12 of 12. The registry lists cardiac tamponade (5.3%) among the top serious terms.

These counts describe events recorded in this study; without a comparator, they do not estimate excess risk attributable to the intervention. The registry record does not provide event-level timing, duration, severity details beyond the serious classification, or a causal assessment in the information. Affected-participant counts also do not describe the total number of events, since a participant may experience more than one event.

13. What the Result Does, and Does Not, Mean

The study record establishes that a phase 3, open-label, single-group study evaluated continuous intravenous dexmedetomidine hydrochloride in pediatric intensive care patients. It defines a primary endpoint based on whether rescue sedation with midazolam was required during a specified period, and it describes the sedation assessment criteria used for intubated participants. It also records the enrolled population and safety counts.

The record does not report the primary endpoint estimate or its 95% confidence interval. It therefore does not show what percentage avoided rescue sedation or how precisely that percentage was estimated. The listed outcome measures do not fill this gap: no analyses are listed, and the registry record does not report outcome results. The absence of a reported result is not evidence that the endpoint was high, low, or unchanged.

Even a reported single-group percentage would not establish a comparative treatment effect. Without a control group, differences from another treatment or from an expected course cannot be estimated through randomized comparison. The open-label design and the role of investigator judgment in rescue medication decisions are additional considerations when assessing an observed rate; they do not demonstrate that the decisions were biased.

The safety counts describe participants affected during the study and include no deaths in the registry record. They should not be interpreted as showing how risks compare with other care, since there is no concurrent comparator. The record also does not provide enough detail to assess how complete event ascertainment was or how missing safety observations were handled.

In short, the registry describes the design, endpoint definitions, and some adverse event counts, but not the efficacy results or statistical analysis needed to quantify the primary outcome. Conclusions about treatment differences, precision of the endpoint estimate, or consistency among outcomes are not supported by the information reported in the registry record.

14. Why This Trial Matters Statistically

This study illustrates why trial design and reported results need to be read together. A phase 3 label indicates the study phase recorded in the registry, but it does not by itself show whether an endpoint was achieved or how uncertain an estimate may be. For this study, the record defines a clinically interpretable endpoint—whether rescue sedation was needed—but does not report the percentage or confidence interval that would quantify it.

The single-group design is central to statistical interpretation. An estimate from participants receiving study drug can describe their observed outcome under the study’s assessment rules. It cannot, without a comparator, answer how that outcome would differ under another treatment. This distinction applies even when enrollment and follow-up are clearly documented.

The endpoint also highlights the importance of operational definitions. The rescue-medication measure depends on clinical judgment and uses the State Behavioral Scale for intubated participants, with distinct target ranges during intubation and after extubation. Those details clarify what the endpoint represents and help prevent it from being mistaken for a direct measure of a treatment contrast.

Finally, the registry lists multiple outcome measures but no analyses. This makes transparency about results, missing data, analysis populations, and multiplicity especially relevant: without those details, readers cannot assess the primary estimate, its precision, or the relationship between primary and secondary findings. The study is statistically informative as an example of how registry design information can define a question while still leaving the answer unquantified in the record.

15. 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 main limitation for interpreting the registry results is that no primary endpoint estimate or confidence interval is reported. Although the endpoint definition and full analysis set are described, the record does not provide the endpoint numerator, denominator, or analysis result. It also lists no analyses, so efficacy findings cannot be assessed from the registry record.

The single-group design provides no randomized comparator. As a result, the study cannot estimate a between-group treatment difference from the design described. Its open-label structure is relevant because the primary endpoint includes rescue medication use judged by investigators; however, the record does not establish that this affected assessments.

The observation period could end at different times for different participants, depending on the end of study drug dosing or mechanical ventilation, as well as the stated 24-hour period and maximum timeframe of 28 days. The record does not report individual observation duration or how participants with incomplete observation contributed to the endpoint.

The record does not report the primary analysis method, confidence interval construction, missing-data procedures, prespecified multiplicity handling, interim analyses, or the study’s sample-size justification. It also lists 29 outcome measures without reporting their results. These gaps prevent assessment of the statistical plan and the precision or robustness of outcome estimates. The registry does not report additional information that would resolve these uncertainties.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study of DA-9501 In Pediatric Subjects In The Intensive Care Unit — NCT02757625.
• Registry record last updated 2018-12-17; status last verified 2018-05.
• Results first posted 2018-12-17.
• 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.

19. Record Summary

NCT02757625 is a completed phase 3, multicenter, single-group, open-label study of dexmedetomidine hydrochloride in pediatric intensive care patients requiring sedation. The registry lists 63 enrolled participants and defines the full analysis set as those who received at least one dose of study drug. The primary endpoint was the percentage who did not require rescue sedation with midazolam during the specified observation period.

The registry record does not report the primary endpoint result, its 95% confidence interval, or analyses of the 29 outcome measures. It reports 1 participant with a serious adverse event among 63 at risk, 55 with other adverse events among 63 at risk, and 0 deaths. Because the study had no comparator group, these data do not provide a randomized estimate of treatment differences.