Inflammatory Cytokine Quantification in Infants: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Inflammatory Cytokine Quantification in Infants (NCT01155830), a registered phase not stated study. The primary outcome is TNF-alpha, Baseline.

2. Clinical Question

In clinical terms, the question is descriptive: what baseline TNF-alpha levels are recorded among infants in these different clinical circumstances? The study also lists maximum TNF-alpha as a secondary outcome. The registry description refers to neonatal sepsis with cardiovascular instability, congenital diaphragmatic hernia, and cardiopulmonary failure requiring ECMO. These are distinct clinical categories, not necessarily exchangeable populations. A difference between their measured levels, if reported, would therefore need to be interpreted in light of those underlying group distinctions rather than as a direct effect of one assigned treatment versus another.

The registry record does not state a hypothesis about which group should have higher or lower levels, nor does it identify a specific treatment effect as the primary target. It also does not report an analysis comparing the groups. The available information therefore defines the population and measurement question, but not a numerical answer to that question.

3. Trial Design

NCT01155830 is listed as a completed study sponsored by the University of Utah. Its title is Inflammatory Cytokine Quantification in Infants, and the official title describes infants with documented sepsis, congenital diaphragmatic hernia, and/or ECMO therapy. The registry lists 21 participants enrolled. The recorded start is 2010-06, and primary completion is listed as 2013-05.

The study description proposes quantifying inflammatory cytokine profiles in three neonatal disease states. The arm descriptions identify infants with congenital diaphragmatic hernia, infants with culture-positive sepsis requiring vasopressor support, and infants with cardiopulmonary failure severe enough to require ECMO. These descriptions identify clinical groups; they do not establish that participants were randomly assigned to groups. Allocation is not stated in the registry record.

The registry information explicitly cautions that the arms are not a like-for-like control comparison. Consequently, the design should not be read as a randomized comparison of an intervention against a control. The record does not state how participants entered each group, whether any group was intended as a reference, or whether the study planned adjusted comparisons. Those design details are not reported in the registry record.

The primary outcome is baseline TNF-alpha, expressed in pg/mL and summarized as a mean with standard error. The listed secondary outcome is maximum TNF-alpha. The record lists two outcome measures and zero analyses. It does not include numerical outcome estimates or a reported comparison between groups. Thus, the registry describes the intended measurements and the clinical categories but does not provide the analysis results needed to assess differences in cytokine levels.

The record labels the study completed, but completion status alone does not establish that every planned measurement was obtained or that an outcome analysis was conducted. The registered enrollment figure and the safety denominators should also be read as separate pieces of information: the record does not provide a participant count for each of the three clinical groups in the outcome information.

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

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 TNF-alpha, Baseline, measured over Baseline.

The primary endpoint is baseline TNF-alpha, a continuous measurement expressed in pg/mL. The registry specifies that it is summarized as a mean with standard error. The study description frames this measurement as part of quantifying inflammatory cytokine profiles in infants with neonatal sepsis and cardiovascular instability, congenital diaphragmatic hernia, or cardiopulmonary failure requiring ECMO.

In general, measuring a cytokine at baseline can describe the level of that marker at the stated baseline assessment. TNF-alpha is the specific marker named for the primary outcome; the registry record does not give a broader biological interpretation, a threshold defining an abnormal result, or a stated clinical decision rule. The outcome is therefore best understood as a laboratory measurement intended to characterize the groups, rather than as a direct measure of a clinical event or treatment benefit.

The mean summarizes the group’s measured values as a central tendency. The standard error describes the statistical precision of an estimated mean, not the spread of individual infants’ measurements. In general, a standard error is affected by both variability in the observations and the number of observations contributing to the estimate. The registry record gives no primary outcome values, standard errors, group-specific outcome sample sizes, or confidence intervals, so the observed levels and their precision cannot be assessed from the record.

Baseline is the outcome timeframe stated for this primary measure. The record separately lists maximum TNF-alpha as a secondary outcome, but does not provide its numerical results or further detail about how the maximum was defined. No conclusions about changes over time or group differences can be drawn from the endpoint description alone.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a mean describe?

For a continuous outcome such as TNF-alpha measured in pg/mL, the mean is a summary of the values in a group. It gives a central estimate but does not show the full distribution. In general, a mean can be influenced by unusually high or low observations. A reader would ordinarily consider the measurement distribution and the individual-level spread alongside the mean, particularly when group sizes are small or when values are skewed. The registry record does not report the observed values or distributions, so those features cannot be assessed for this study.

The registry names standard error as the dispersion measure for the primary mean. In general, standard error quantifies the sampling uncertainty of an estimated mean. It is not the same as standard deviation: standard deviation describes variability among individual observations, while standard error describes uncertainty in the estimated mean. A standard error alone also does not show the complete range of individual measurements. Here, no numerical standard error is reported, so the precision of the baseline mean is unknown.

How should group comparisons be understood?

In general, a randomized comparison can support a causal interpretation when assignment, follow-up, and analysis are appropriate. This registry record does not state allocation and explicitly notes that the arms are not a like-for-like control comparison. The group definitions reflect different clinical states and support needs. A difference in a biomarker across such groups, if estimated, would be an association between group membership and the measured level; it would not by itself establish that one condition or treatment caused the difference.

The record does not state whether the intended analysis was descriptive, comparative, or adjusted for other factors. It does not report a statistical model, covariates, transformation of measurements, handling of repeated measures, or a hypothesis test. These analysis details are not reported in the registry record. Without them, the planned inferential method and the assumptions underlying any comparison cannot be reconstructed.

What does uncertainty mean?

In general, a confidence interval expresses uncertainty around an estimate under a specified statistical method. A narrow interval indicates greater precision than a wide interval, all else being comparable. It does not establish that the estimate is free from bias, and it does not turn a nonrandomized group comparison into a causal result. A p-value, if reported, addresses compatibility between data and a specified null model; it does not measure the size or clinical importance of an effect. No confidence intervals, p-values, or effect estimates for the primary outcome are reported in the registry record.

The primary outcome is a mean, but an estimate of the difference between group means would require outcome information and a clearly defined comparison. The registry does not state which groups were to be compared or report such estimates. No new comparison can be inferred from the registered endpoint name alone.

What do the planning assumptions mean?

The record describes a sample-size planning illustration based on the observed effect, not the study’s own design. It specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. For outcomes other than time-to-event outcomes, the planning quantities are participants per arm for 80% power or participants per arm for 90% power. Time-to-event illustrations instead give the total number of events.

These assumptions are planning conventions, not evidence about how this study was designed or analyzed. They do not establish that the actual study used equal allocation, a two-arm comparison, or that it was designed around an observed effect. A planning sample size is not observed power and cannot establish whether the study had adequate power after the fact. The registry record does not report its own sample-size justification. Numerical planning outputs are not included in the registry information summarized in this record.

Why can over-dispersion matter?

In general, over-dispersion means that observed variation is greater than a statistical model assumes. For continuous measurements, the relevant issue may be a distribution with greater spread or heavier tails than an analysis method accommodates. In other settings, over-dispersion is used for count data when variation exceeds a model’s expected level. If a model fails to accommodate extra variation, its uncertainty estimates can be too small and statistical evidence can appear more precise than it is. The registry record does not report a model or an assessment of dispersion for TNF-alpha, so there is no basis to say whether over-dispersion was examined or relevant to the analysis actually used.

Because the outcome values and statistical analyses are not reported, the statistical methods can only be explained in general terms and in relation to the endpoint and planning assumptions named in the registry. The record does not provide enough information to verify model assumptions or reproduce an outcome analysis.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Infants Treated With ECMO")
n   <- c(21)
m   <- c(8.88)
sp  <- c(4.58)
sd  <- sp * sqrt(n)      # standard errors converted to SDs
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Infants Treated With ECMO  21  8.880  20.988  -0.674  18.434

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures: baseline TNF-alpha as the primary outcome and maximum TNF-alpha as a secondary outcome. It reports zero analyses. No numerical results, p-values, confidence intervals, or analysis descriptions are available for either outcome. The record does not state whether the two outcomes were included in a formal testing hierarchy or whether any adjustment for multiple comparisons was planned. Multiplicity handling is not reported in the registry record.

When several outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase. In general, a prespecified testing strategy or adjustment can address that issue, but the suitable approach depends on the questions and analyses planned. The record does not identify a set of group comparisons, so it is not possible to determine how many tests were intended or whether multiplicity correction was relevant to a particular analysis.

The registry does not report missing outcome counts, reasons for missing measurements, or methods for handling missing data. It also does not state whether all enrolled infants contributed to the baseline or maximum TNF-alpha outcomes. Missing-data handling is therefore not reported in the registry record.

Similarly, the record does not define an analysis population, such as a population based on enrollment, outcome availability, or another criterion. The total enrollment is 21, but the number included in any primary or secondary outcome analysis is not reported. The safety denominators should not be substituted for outcome populations. The registry record also does not describe exclusions, withdrawals, or how infants who might have had incomplete measurements were treated analytically.

These omissions limit assessment of both the completeness and the inferential reliability of any result. No conclusions about multiplicity control, missing-data bias, or the population actually analyzed can be made from the registry information.

12. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

The serious adverse event entries list zero participants affected in each of the three groups. The at-risk counts differ: zero for infants with congenital diaphragmatic hernia, zero for infants with sepsis, and 21 for infants treated with ECMO. The entries for other adverse events likewise list zero affected, with the same at-risk counts. Death counts are recorded as null rather than as a numerical count. No serious adverse event terms are listed.

These entries require attention to denominators. A group with zero participants recorded as at risk cannot be interpreted as showing an absence of events among a group that was followed; the registry provides no at-risk participants for those groups in these safety entries. For the ECMO group, the record lists 21 at risk and zero affected in the displayed categories, but the registry does not give further detail about safety surveillance, reporting periods, or event definitions.

In general, a zero count does not demonstrate that risk is absent. It describes the recorded count within the reported safety information, and its interpretation depends on who was monitored, for how long, and how events were collected. Those details are not reported in the registry record. Null death entries should not be read as zero deaths. The safety entries also do not establish a comparative safety profile across the clinical groups.

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

The registry record establishes that NCT01155830 was intended to quantify inflammatory cytokine profiles in three neonatal disease states and identifies baseline TNF-alpha as the primary outcome. It also specifies pg/mL as the unit and mean with standard error as the summary. These details clarify what the study set out to measure, but the record does not report the measured values or an analysis of them.

As a result, the registry information does not show whether baseline TNF-alpha levels were high or low, how much they varied, or whether the groups differed. It does not provide estimates with which to judge precision. The secondary outcome, maximum TNF-alpha, is named but has no reported numerical result or analysis either.

The three arms should not be interpreted as treatment and control groups. They represent different clinical circumstances, and the record states that they are not a like-for-like control comparison. Allocation is not stated. Even if a group difference were reported elsewhere, a comparison across these groups would need careful consideration of how their clinical characteristics differ and what analysis was used. The registry record does not support a causal claim about the effect of ECMO, sepsis, or any other treatment.

The study’s completed status and enrollment of 21 describe registry information about the trial, not the availability or reliability of an outcome result. The outcome analysis count is zero, and outcome-specific sample sizes are not reported. The safety counts are separate and cannot fill in those missing endpoint results.

In general, a biomarker measurement can contribute to scientific understanding without directly answering whether an intervention improves a clinical outcome. The registry description is focused on cytokine quantification, and it does not state a clinical endpoint or treatment-effect objective. The appropriate interpretation is therefore limited: the record documents a study objective and planned outcome measures, but it does not provide results from which to assess the magnitude or meaning of TNF-alpha levels in these infants.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the difference between a registered measurement objective and an analyzable result. The registry identifies the biomarker, unit, summary measure, timeframe, and clinical groups. Those elements help define what a study is intended to observe. At the same time, the absence of outcome values and analyses means readers cannot assess the observed level, its uncertainty, or any group pattern.

The study also illustrates why the nature of the comparison matters. The clinical groups are not described as randomized arms or like-for-like controls. Their definitions involve distinct disease states and levels of clinical support. In general, group contrasts in such settings describe differences between the groups as observed; they do not, without appropriate design and analysis, isolate a causal effect of a treatment or condition.

The use of a mean with standard error highlights an important reporting distinction. A mean describes a group’s central value, while its standard error concerns precision of that estimate. Neither gives a complete account of individual variation. Reporting the estimate, its uncertainty, and relevant group sizes would help readers evaluate how informative a biomarker summary is. Those numerical outcome details are not reported in the registry record.

Finally, the record distinguishes the planning illustration from the study’s own design. Sample-size calculations based on an observed effect and stated assumptions are planning exercises; they do not establish actual power or describe the study’s original sample-size rationale. The registry does not report that rationale. This distinction is important when interpreting small clinical datasets: planning calculations, enrollment totals, outcome denominators, and analyzed results answer different questions and should not be treated as interchangeable.

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 principal limitation for interpretation is that the registry record lists no analyses and provides no numerical primary or secondary outcome results. There are no group means, standard errors, confidence intervals, or reported comparisons for baseline TNF-alpha. The maximum TNF-alpha outcome is also listed without a result. Thus, neither the size nor precision of any observed cytokine pattern can be evaluated.

The total enrollment is 21, but the record does not report the number contributing to each outcome or the distribution of participants across the three clinical groups for outcome analysis. The safety denominator of 21 in the ECMO group does not establish that all 21 enrolled participants contributed to that outcome, or that the other groups had no outcome data.

The record does not state the allocation process, a comparator, a prespecified group contrast, or an analysis plan. It explicitly notes that the arms are not a like-for-like control comparison. The clinical groups differ by condition and support status, so any comparison would require an interpretation that accounts for those group distinctions. Whether analyses adjusted for other characteristics is not reported in the registry record.

Definitions and measurement details beyond the named endpoint are limited. The registry does not report how baseline timing was operationalized, how maximum TNF-alpha was defined, whether there were repeated measurements, or how any missing observations were handled. It also does not report a sample-size justification or a multiplicity strategy.

The safety information has zero at-risk counts for two groups, a denominator of 21 for the ECMO group, null death entries, and no listed serious adverse event terms. Without further information on monitoring and reporting, those entries do not provide a complete comparative safety assessment. Overall, the registry record supports description of the intended study but leaves substantial uncertainty about its conduct and outcomes.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Inflammatory Cytokine Quantification in Infants — NCT01155830.
• Registry record last updated 2015-05-04; status last verified 2014-12.
• Results first posted 2015-05-04.
• 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

NCT01155830, Inflammatory Cytokine Quantification in Infants, is listed as completed and sponsored by the University of Utah. Enrollment is listed as 21. The recorded start is 2010-06, with primary completion in 2013-05. The study objective is to quantify inflammatory cytokine profiles in infants with sepsis, congenital diaphragmatic hernia, or cardiopulmonary failure requiring ECMO.

The primary outcome is baseline TNF-alpha in pg/mL, summarized as a mean with standard error. Maximum TNF-alpha is listed as a secondary outcome. The registry record reports zero analyses and provides no numerical outcome results. Allocation is not stated, and the arms are not identified as a like-for-like control comparison. The record therefore describes the study question and registered outcome measures, but does not provide evidence to assess the observed cytokine levels or their precision.