Telavancin Pediatric PK Study (Ages >12 Months to 17 Years): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Telavancin Pediatric PK Study (Ages >12 Months to 17 Years) (NCT02013141), a na phase 1 study. The primary outcome is Pharmacokinetics- Area Under the Curve Extrapolated to Infinity for the Plasma Concentration Versus Time Curves for Telavancin.

2. Clinical Question

This is a description of drug exposure after administration, not a randomized evaluation of clinical benefit. Safety was also included among the secondary outcomes, but the listed outcomes do not establish a treatment comparison.

3. Trial Design

The registry describes a phase 1, multicenter, open-label study of the pharmacokinetics of a single telavancin dose in pediatric subjects aged 12 months to 17 years. It lists a single-group model, no masking, and allocation as not applicable. The listed intervention was telavancin, given at 10 mg/kg intravenously over approximately 60 minutes one time. No comparator arm is described.

The enrollment field reports 22. The registry lists the study as terminated, with a start in 2014-12 and primary completion in 2021-03. It does not report why the study was terminated. That status alone does not explain whether termination affected enrollment, follow-up, or the availability of outcome data.

The primary outcome was an area-under-the-curve pharmacokinetic measure, calculated from plasma telavancin concentrations measured at 1.0, 1.5, 2.0, 6.0, 12.0, and 24.0 hours after infusion. The outcome was specified as a mean with standard deviation, in h*mcg/ml. The pharmacokinetic population excluded one subject from Cohort 1 and one from Cohort 2 because plasma samples were missing at >1 time point.

The registry lists safety outcomes concerning the number of treatment-emergent adverse events and the number of subjects with such events. It reports seven outcome measures and zero analyses. Numerical results for the primary pharmacokinetic outcome are not reported in the registry record. The record also does not report a statistical analysis plan, a target sample size rationale, or details explaining how the terminated study's enrollment and collected data were handled in the analysis.

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

Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic population- one subject from Cohort 1 and one subject from Cohort 2 were excluded due to lack of pharmacokinetic plasma samples at \>1 time point

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.

6. Primary Endpoint

The registry lists the primary outcome as Pharmacokinetics- Area Under the Curve Extrapolated to Infinity for the Plasma Concentration Versus Time Curves for Telavancin, measured over 1.0, 1.5, 2.0, 6.0, 12.0, 24.0 hours.

The primary endpoint was the area under the curve extrapolated to infinity for plasma telavancin concentration versus time, expressed in h*mcg/ml. Concentration measurements were taken at 1.0, 1.5, 2.0, 6.0, 12.0, and 24.0 hours following the single intravenous dose. The registry specifies that the endpoint is summarized as a mean with standard deviation.

In general, an area under a concentration-time curve represents drug exposure over time. Extending the curve to infinity aims to include exposure beyond the last measured concentration, rather than limiting the summary to the observed sampling window. The registry record does not describe the specific calculation method or assumptions used for that extrapolation.

Pharmacokinetic measures are used to characterize how drug concentrations change after administration. They address exposure, not whether a treatment improves a clinical condition. In a study without a comparator arm, the endpoint can describe exposure observed in the study population but cannot by itself estimate a comparative treatment effect.

The registry identifies the endpoint and its unit but does not report a mean, standard deviation, confidence interval, or other numerical result for it. Thus, the posted record does not show the estimated exposure or how precisely it was measured. It also does not report cohort-specific endpoint values.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary measure summarize?

The endpoint is the area under the plasma concentration-versus-time curve extrapolated to infinity. In general, a concentration-time curve traces measured drug concentrations at successive sampling times. The area under that curve is a summary of exposure over time; extrapolation extends the summary beyond the final measured sample. Here, the listed sampling times were 1.0, 1.5, 2.0, 6.0, 12.0, and 24.0 hours after infusion. The registry record does not describe the formula, interpolation approach, or assumptions used to estimate the portion beyond the last sample.

The stated unit, h*mcg/ml, reflects concentration accumulated over time. The endpoint is defined as a continuous measure, with the mean and standard deviation specified as its summary statistics. The registry record does not post numerical values for either statistic.

What do the mean and standard deviation tell a reader?

In general, the mean provides a central summary of observed values. The standard deviation describes how dispersed individual values are around that mean. A larger standard deviation indicates more spread among measurements, while a smaller one indicates less spread. This describes variability in the observations; it does not, on its own, quantify uncertainty in the mean. A confidence interval is a different summary that represents uncertainty about an estimated population value. No confidence interval for the primary endpoint is reported in the registry record.

The primary endpoint is a pharmacokinetic measure rather than a clinical outcome. Its interpretation concerns exposure after administration. In general, exposure summaries may help describe drug disposition, but an exposure estimate alone does not establish clinical effectiveness or comparative safety.

Why does the design affect interpretation?

The registry identifies a single-group model, no masking, and allocation as not applicable. There is no control arm against which to estimate a between-group difference. The age cohorts are described by age, not as randomized treatment groups. Accordingly, any cohort-level differences would be descriptive unless an appropriate analysis and its assumptions were specified; such cohort-specific primary results are not reported in the registry record.

Open-label means masking was not used. In general, lack of masking can affect outcomes that depend on judgment or reporting. Concentration measurements are more objective than subjective assessments, but the registry does not report laboratory procedures, assay details, or quality-control methods. Those details cannot be evaluated from the record.

What does missing sampling imply?

The pharmacokinetic population excluded one subject from Cohort 1 and one subject from Cohort 2 because plasma samples were absent at >1 time point. A curve-based exposure measure relies on measurements across time, so unavailable samples can affect whether an individual endpoint can be calculated. The record reports exclusion for this reason, but does not describe the cause of missing samples, whether any additional measurements were unavailable, or whether any missing-data sensitivity analysis was performed.

In general, a complete-case analysis uses only subjects with the measurements required for the endpoint. Such an analysis can be difficult to interpret if missingness is related to characteristics that also affect the outcome. The registry does not report whether missingness was considered likely to be informative, nor does it report imputation or other missing-data methods. No conclusion about the impact of these exclusions can be drawn without additional information.

What can be inferred from the analysis record?

The registry lists seven outcome measures and zero analyses. That means the record does not present a statistical analysis for the outcomes. It does not report a model, an inferential test, a confidence interval, a multiplicity adjustment, or an interim-analysis plan. In general, a statistical analysis plan helps readers understand how outcomes, missing observations, and multiple comparisons were handled. The plan is not reported in the registry record, so those choices cannot be assessed.

The planning illustrations, where applicable, should be read as planning scenarios rather than observed power. In general, sample-size calculations depend on assumptions about an effect, variability, allocation, and the chosen significance level. The stated assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a fraction of an observed effect. These are planning assumptions, not evidence that this single-group study used such a design or achieved a particular power. Because the registry does not report numerical primary endpoint results and there is no comparator arm, the record does not establish an observed comparative effect for interpreting such a calculation.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists seven outcome measures, including the primary pharmacokinetic endpoint and secondary safety outcomes. It reports zero analyses. A statistical plan describing which outcomes were confirmatory, how multiple outcomes were handled, or whether any adjustment was used is not reported in the registry record.

In general, when several outcomes or statistical tests are considered, multiplicity can increase the chance of finding an apparently notable result by chance. Methods for addressing this depend on the study's objectives and analysis plan. Without that plan and without posted analyses, it is not possible to determine how multiplicity was considered or to interpret any outcome as a prespecified confirmatory finding.

For the primary pharmacokinetic population, the record says that one subject from Cohort 1 and one subject from Cohort 2 were excluded because plasma samples were missing at >1 time point. It does not report the reasons for missing samples, the concentration data that were unavailable, any imputation method, or a sensitivity analysis. It also does not explain whether other missing data affected the safety outcomes.

The registry does not describe an analysis population beyond the stated pharmacokinetic population or explain how subjects were handled for each secondary outcome. In general, analysis-population definitions clarify which observations contribute to a given estimate and why. The available description identifies the exclusions for the primary endpoint but does not provide enough detail to assess other population rules. Numerical results and a fuller statistical plan are not reported in the registry record.

11. 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 non-serious adverse event terms

The registry's safety display lists serious adverse events for three age cohorts: Cohort 1 (Age 12 to 17 Years), Cohort (Age 6 to 11 Years), and Cohort 3 (Age 2 to 5 Years). The number of subjects with serious adverse events is 0 in each cohort. The number of deaths is also 0 in each cohort.

The display reports 14 subjects at risk in Cohort 1, 7 in Cohort (Age 6 to 11 Years), and 1 in Cohort 3. It lists 6 other adverse events in Cohort 1, 4 in Cohort (Age 6 to 11 Years), and 0 in Cohort 3. These are counts as recorded, not evidence that the same number of subjects experienced events; the registry distinguishes the number of events from the number of subjects with treatment-emergent events.

In general, zero recorded serious events means none were reported in that cohort in the registry display; it does not prove that the risk is absent. These descriptive safety counts come from a single-group study with no comparator, so they do not estimate a comparative safety effect. The registry does not report additional context such as event details, severity categories beyond the serious-event designation, or an analysis comparing age cohorts.

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

The record establishes that a phase 1, open-label, single-group study examined the pharmacokinetics of a single 10 mg/kg intravenous dose of telavancin in pediatric age cohorts. It specifies the primary exposure measure, its unit, the concentration sampling times, and the pharmacokinetic population exclusions. It also reports safety counts for the displayed cohorts.

The record does not report numerical results for the primary area-under-the-curve endpoint. It therefore does not show the observed mean exposure, the spread of individual values, or the precision of an estimate. Without those results, the primary pharmacokinetic findings cannot be quantitatively characterized from the registry record.

The study has no comparator arm. Its design cannot provide a randomized estimate of how telavancin compares with another treatment or with no treatment. The listed age cohorts are not described as randomized comparison groups, and the registry does not report cohort-specific primary endpoint results. The record also does not present a clinical efficacy outcome that would support a conclusion about clinical benefit.

The safety display reports zero serious adverse events and zero deaths in each listed cohort, alongside counts of other adverse events. Such counts describe what was recorded in the study; they do not establish that serious events cannot occur or that the safety profile is better or worse than an alternative. The record does not provide a comparative safety analysis.

Finally, the registry lists zero analyses and does not report a statistical analysis plan, detailed missing-data methods, or an explanation for termination. These gaps limit assessment of the analytic process and the completeness of the evidence. They should not be filled by assumptions about what may have occurred outside the registry record.

13. Why This Trial Matters Statistically

This study illustrates a distinction central to clinical biostatistics: describing exposure is not the same as estimating a treatment effect. The primary endpoint is a continuous pharmacokinetic measure, area under the concentration-time curve extrapolated to infinity. It addresses measured drug exposure after a single dose, while a treatment comparison would require an appropriate comparator and an analysis designed to estimate a difference.

The registry's single-group design makes that distinction especially important. A single-group study can describe observed values, but it cannot separate the intervention's effects from other explanations through a concurrent randomized comparison. The age cohorts do not serve as control arms simply because they contain different age ranges.

The record also demonstrates why the availability of a clearly named endpoint is not enough for interpretation. The registry identifies the measure, unit, time points, and summary statistics, but does not report the numerical endpoint values or analyses. Without results, readers cannot assess the magnitude, variability, or precision of exposure estimates. A complete statistical interpretation depends on those quantities as well as on the design.

Missing concentration samples are relevant because the endpoint relies on measurements over time. The record documents exclusions from the pharmacokinetic population but does not report enough information to assess whether missingness could have affected the results. Similarly, the listing of several outcomes makes the unreported statistical plan relevant: readers cannot evaluate the handling of multiple outcomes or the criteria used for analysis.

These features make the registry record useful for understanding the study question and design, while also defining the limits of what can be concluded from its posted information. The absence of numerical results is not evidence for a particular pharmacokinetic finding; it means that finding is not reported in the registry record.

14. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The most important limitation for interpreting the posted record is that it does not report numerical results for the primary pharmacokinetic endpoint. Although the endpoint, unit, summary measure, and sampling times are specified, the mean and standard deviation are absent. The registry also reports zero analyses, so no inferential results or interval estimates are available for review.

The design is single-group, open-label, and without a comparator. It can describe exposure following the administered dose, but cannot support a randomized comparison with another treatment or placebo. The record does not report cohort-specific primary endpoint estimates, so it also does not support quantitative comparisons among the age cohorts.

The primary pharmacokinetic population excluded one subject from Cohort 1 and one subject from Cohort 2 because plasma samples were unavailable at >1 time point. Reasons for missing samples, the handling of other missing data, and sensitivity analyses are not reported in the registry record. The impact of these exclusions therefore cannot be assessed.

The registry states that the study was terminated but does not report the reason or explain how termination affected data collection or analysis. It does not provide a detailed statistical analysis plan, a multiplicity approach, assay information, or the calculation method for extrapolating the area under the curve to infinity. Those omissions limit evaluation of analysis choices and measurement procedures.

The displayed safety counts are descriptive and lack a comparator. They do not estimate comparative risks. These limits apply to interpretation of the registry record and do not establish what may or may not be available outside it.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Telavancin Pediatric PK Study (Ages >12 Months to 17 Years) — NCT02013141.
• Registry record last updated 2024-06-24; status last verified 2024-01.
• Results first posted 2024-06-24.
• 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.

18. Record Summary

The registry describes NCT02013141 as a phase 1, multicenter, open-label, single-group pharmacokinetic study of a single 10 mg/kg intravenous telavancin dose in pediatric subjects aged 12 months to 17 years. Enrollment is listed as 22; the study status is terminated.

The primary outcome was area under the plasma concentration-time curve extrapolated to infinity, measured in h*mcg/ml and specified as a mean with standard deviation. Concentrations were measured at 1.0, 1.5, 2.0, 6.0, 12.0, and 24.0 hours. One subject from Cohort 1 and one from Cohort 2 were excluded from the pharmacokinetic population because samples were missing at >1 time point.

The registry lists safety outcomes and reports cohort-level adverse-event counts. It lists seven outcome measures and zero analyses. Numerical results for the primary pharmacokinetic endpoint, a detailed statistical plan, and the reason for termination are not reported in the registry record.