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 Descriptive Study of Cardiac Output During Rehydration With Recombinant Human Hyaluronidase in Pediatrics (NCT01139294), a randomized phase 4 study. The primary outcome is Cardiac Output Trends.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01139294 |
| Phase | Phase 4 |
| Condition | Dehydration, Cardiac Output, Pediatrics |
| Allocation | Randomized |
| Design model | Factorial |
| Masking | None (open label) |
| Enrollment | 17 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Vanderbilt University Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric patients with moderate dehydration receiving care in an Emergency Department. The registry record does not report further eligibility details or baseline characteristics.
Intervention
Hylenex-augmented subcutaneous rehydration. The arm description specifies 1ml subcutaneously with initiation of intravenous fluids, then every 24 hours, with a maximum of 3 injections in 72 hours.
Comparator
Standard IV therapy, described as the control arm. The record calls the comparison routine or standard IV rehydration.
Primary question
How do noninvasively measured cardiac output trends, including onset and changes in slope over time, compare during rehydration with Hylenex-augmented subcutaneous rehydration or routine IV rehydration?
The question concerns a physiological response during fluid therapy rather than a direct comparison of symptoms, recovery, or other patient-important outcomes. Cardiac output was to be measured in liters per minute while patients received care in the Emergency Department. The outcome description emphasizes the pattern of measurements over time, including when changes begin and whether the slope changes.
The registry also lists Gorelick Assessment as a secondary outcome. It does not report the measure's results or explain how it was assessed. No cardiac output values, estimates of change, or between-arm comparisons are available: the registry record states that the study data were lost. Therefore, the clinical question can be described from the planned comparison, but the posted record cannot answer it with outcome evidence.
3. Trial Design
This was a Phase IV randomized study of Hylenex or standard IV hydration in the pediatric population with moderate dehydration. The registry lists a factorial model and no masking. The study is marked completed, with enrollment of 17. It lists Vanderbilt University Medical Center as sponsor.
The record describes two arms: standard-of-care IV therapy and Hylenex. The Hylenex arm description specifies 1ml administered subcutaneously when intravenous fluids were initiated, followed by administration every 24 hours, with a maximum dose of 3 injections in 72 hours. Standard IV therapy is identified as the control arm. The study summary describes the purpose as examining reflective changes in cardiac output during fluid therapy, comparing Hylenex-augmented subcutaneous infusion of fluids and electrolytes with intravenous methods.
Random allocation can reduce systematic differences between groups on average, but the record does not report the randomization sequence, allocation concealment, or any stratification. The factorial label is recorded, but the factors, their levels, and the planned comparisons are not reported in the registry record. It is therefore not possible to determine from the record how that design label relates to the two described arms.
Masking is listed as none. The record does not explain whether outcome assessors were also unmasked or whether measurement procedures were standardized. The study began in 2010-02 and had primary completion in 2011-11. These design details describe the intended trial structure, but they do not substitute for results. The primary outcome data were lost, and no analyses are reported.
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.
Before assignment: 17 participants were enrolled into the study per the final report to the Institutional Review Board. Actual study data was lost, so arm assignments are unknown.
Overall Study
| Stage | Standard of Care IV Therapy | Hylenex |
|---|---|---|
| STARTED | 0 | 0 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: No outcome measure data available. Study data was lost. Data was left behind when PI left institution in 2013. Unable to find anyone with knowledge of data location.
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: No baseline data available. Study data was lost. Data was left behind when PI left institution in 2013. Unable to find anyone with knowledge of data location.
| Characteristic | Unit | Standard of Care IV Therapy | Hylenex | Total |
|---|---|---|---|---|
| Participants analysed | 0 | 0 | 0 | |
| Age, Categorical: <=18 years | — | — | — | |
| Age, Categorical: Between 18 and 65 years | — | — | — | |
| Age, Categorical: >=65 years | — | — | — | |
| Sex: Female, Male: Female | — | — | — | |
| Sex: Female, Male: Male | — | — | — | |
| Region of Enrollment: United States | participants | — | — | — |
6. Primary Endpoint
The registry lists the primary outcome as Cardiac Output Trends, measured over Measurements and data are recorded only while patient is receiving care in the Emergency Department (ED). A f/u call is made on day 3 and/or day 7 post enrollment into the study..
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled Cardiac Output Trends. It was intended to describe cardiac output measured in liters per minute, focusing on the onset of changes and changes in slope over time. The stated measurement approach was noninvasive monitoring in pediatric patients receiving either Hylenex-augmented subcutaneous rehydration or routine IV rehydration. Measurements and data were to be recorded while the patient was receiving care in the Emergency Department. The record also mentions a follow-up call on day 3 and/or day 7 after enrollment, but does not explain how that call contributed to the primary endpoint.
In general, cardiac output is a measure of the volume of blood pumped by the heart over time. Tracking measurements across treatment can describe a physiological pattern, rather than relying on a single measurement. A slope can represent the direction and rate of change across observations, while the onset describes when a change begins. These features may be useful for characterizing the response observed during rehydration, but they do not by themselves establish a benefit in symptoms or other clinical outcomes.
The registry record gives no endpoint values, units beyond the planned liters-per-minute measure, or analysis results. It explicitly says no outcome measure data are available because the study data were lost. Consequently, neither the observed cardiac output trends nor their precision can be evaluated from the posted record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record does not provide a primary outcome result. It states that study data were lost after the principal investigator left the institution in 2013 and that the data location could not be identified. There are no cardiac output measurements, estimates of change over time, uncertainty intervals, or statistical comparisons reported. The planned endpoint therefore cannot be interpreted as evidence favoring either rehydration approach.
The study is listed as randomized, which describes how treatment assignment was intended to occur. Randomization is useful because, when properly implemented, it can limit systematic differences between groups at baseline. It does not guarantee that groups will be balanced in a small study, and it cannot compensate for missing outcome data. Because the record includes no baseline or outcome data, it is not possible to assess balance, follow-up, or the treatment contrast.
The enrollment is listed as 17, but this number alone does not reveal how many participants contributed to a specific endpoint, whether any participants withdrew, or whether measurements were available at each planned time. Such details are not reported in the registry record. The number enrolled cannot be treated as the denominator for an outcome analysis when outcome records are unavailable.
The adverse-event fields list zero participants at risk and zero affected for serious and other events in both groups. These entries do not establish that no adverse events occurred. With zero participants listed at risk, there is no usable denominator from which to estimate event frequency or compare safety between groups. Death counts are not reported. The registry record therefore does not support a safety-rate conclusion.
The study lists Gorelick Assessment as a secondary outcome, but provides no results for it. It also reports no analyses. With no reported outcome data or analyses, there is no basis for judging the magnitude, direction, or precision of an effect. The record describes a research question and design, but does not preserve the evidence needed to answer that question.
Any planning illustration based on an observed effect must be distinguished from evidence about the trial. In general, sample-size calculations use an assumed effect and statistical design to estimate the number of participants needed under those assumptions. They are not observed power and do not recover missing results. Because the primary outcome data and the observed effect are unavailable in the registry record, no trial-specific effect estimate can be interpreted from them.
9. Statistical Methods Explained
The record identifies a randomized study with no masking and a factorial model. These labels describe different aspects of design. Randomization concerns how participants are assigned to study groups. Masking concerns whether participants, clinicians, or people assessing outcomes know the assigned group. A factorial design generally evaluates more than one intervention factor within the same study, but the factors and comparisons are not reported in the registry record. The label alone is not enough to reconstruct the design or the intended analysis.
What would a trend analysis describe?
For an outcome measured repeatedly, a trend analysis describes how values change over time rather than comparing only one measurement. A model may represent the average starting level and the rate of change, and may allow the rate to differ across periods. In this trial, the endpoint description refers to onset and changes in slope. That wording indicates interest in the time pattern, but the registry record does not specify the planned model, measurement schedule, or exact contrasts.
Repeated measurements from one participant are related to one another. They are not equivalent to independent observations from separate participants. A statistical analysis must account for that within-participant association to avoid overstating the amount of information. In general, longitudinal methods can model this dependence and describe average trajectories, but the specific method planned for this study is not reported in the registry record.
What does a confidence interval convey?
In general, a confidence interval expresses the uncertainty around an estimate under a specified statistical method. A relatively narrow interval indicates greater precision than a wider interval, while the interval's location describes which effect sizes are compatible with the data and method. A confidence interval is not a guarantee that the true effect lies within its limits. No confidence interval is reported for this study, because the outcome data were lost.
A p-value, when reported, describes how unusual data at least as incompatible with a null hypothesis would be under the assumptions of a statistical test. It does not measure the probability that the null hypothesis is true, and it does not measure clinical importance. No p-value or analysis result is reported in the registry record. It would therefore be inappropriate to infer statistical significance or a null finding from the absence of results.
Why can over-dispersion matter?
In general, over-dispersion occurs when observed variability is greater than a statistical model assumes. If ignored, standard errors may be too small and confidence intervals too narrow. It is especially relevant to some count-data models, where the assumed relationship between the mean and variability may not fit the data. The endpoint is a continuous cardiac output measure, and no count-based primary analysis is reported. The registry record does not state whether over-dispersion was assessed or whether any model adjustments were planned.
What does a planning calculation mean?
A sample-size calculation is a planning exercise based on assumptions such as the target effect, variability, allocation, significance level, and analysis method. The record describes an illustrative calculation using a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the effect framed as a stated fraction of an observed effect. Those are planning assumptions, not findings from the trial. The record also distinguishes illustrations expressed as participants per arm from time-to-event illustrations expressed as total events. No trial-specific outcome estimates or numeric planning values are reported in the registry record.
When the observed effect used as the planning reference is not available, the illustration cannot be interpreted as evidence about the actual treatment contrast. A calculated sample size is not the number of participants observed in the trial, and it is not observed power. In general, observed power calculated from an observed effect does not replace an effect estimate and its uncertainty. Here, the missing outcome data prevent such interpretation.
The registry lists zero analyses. This means the record does not present an analysis result; it does not establish what analyses were conducted outside the record. The statistical plan, including the intended model for the cardiac output trajectory, handling of repeated measurements, covariates, and any planned subgroup or sensitivity analyses, is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures and identifies Cardiac Output Trends as primary and Gorelick Assessment as secondary. It does not report a multiplicity strategy. In general, evaluating several outcomes, time points, or contrasts can increase the chance of at least one apparently unusual result if each is tested separately. Prespecified prioritization, adjusted thresholds, or a hierarchy of tests can address that issue, but the registry record does not describe any such approach.
The primary endpoint itself concerns changes over time, including onset and changes in slope. The record notes that the primary outcome has several categories or time points and was not reduced to one comparison. It does not specify which time points or contrasts were planned for inference. The analysis plan for handling multiple measurements and comparisons is not reported in the registry record.
Missing outcome information is the central limitation. The registry says no outcome measure data are available because study data were lost; it describes the data as left behind when the principal investigator left the institution in 2013, with no one able to identify their location. It does not report participant-level completeness, withdrawals, missed visits, or an imputation method. Those details cannot be inferred from the total enrollment.
The analysis populations are also not described. The record does not state whether analyses were intended to include all randomized participants, only participants who received assigned treatment, or another group. It does not report how protocol deviations or unavailable measurements would have been handled. With no outcome dataset and no reported analyses, neither the impact of missingness nor the use of an analysis population can be assessed.
11. What the Result Does, and Does Not, Mean
The registry record establishes that a randomized Phase IV study was conducted to examine cardiac output trends during two approaches to rehydration in pediatric patients with moderate dehydration. It describes the intervention, comparator, intended outcome, and study status. It does not report the primary outcome data or a statistical comparison. Accordingly, it does not show the direction or size of any difference between the groups.
Absence of results is not evidence that the groups had the same cardiac output trends. It is also not evidence that one approach produced a better or worse trend. Without measurements and uncertainty estimates, the range of effects compatible with the study cannot be assessed. The missing data leave the clinical research question unanswered in this registry record.
The record also cannot establish a safety comparison. Although the adverse-event entries display zeros, they also show zero participants at risk. Those entries do not provide interpretable event rates. The record does not report death counts or secondary-outcome results.
In general, a physiological endpoint can provide information about a biological response, but it is not automatically equivalent to a patient-important outcome. Cardiac output trends alone would not establish a difference in symptoms, recovery, or other outcomes unless those outcomes were measured and analyzed. The registry lists Gorelick Assessment as secondary, but does not provide its results.
The enrollment figure describes the study's recorded enrollment, not the amount of usable endpoint information. The number of participants with outcome measurements, the distribution across arms, and the extent of follow-up are not reported in the registry record. No conclusion about precision, statistical significance, or the clinical importance of an effect can be drawn from the available results because no results are posted.
12. Why This Trial Matters Statistically
This study illustrates an important distinction between a trial's design and its evidence. Randomization and a clearly stated physiological question can define a useful comparison, but the scientific value of the comparison depends on retaining outcome data and reporting analyses. In this case, the registry records a completed study and an intended cardiac output endpoint, while also stating that the study data were lost. The design remains describable, but the treatment contrast is not evaluable from the posted record.
The endpoint also raises a common statistical issue: repeated measurements are intended to capture a trajectory, not merely a single result. Analyses of trajectories need to account for the relationship among observations from the same participant and to specify which changes or time points are of interest. The registry's reference to onset and changes in slope conveys the scientific aim, but the statistical plan is not reported.
Study records are essential for interpreting null, positive, or uncertain findings. If measurements are unavailable, readers cannot distinguish a genuinely small effect from a large but imprecisely estimated effect, or determine whether the planned comparison was carried out. This is not a statistical finding about rehydration; it is a limitation on the evidence that can be recovered from the registry record.
The case also shows why enrollment and adverse-event entries should not be treated as substitutes for outcome reporting. Enrollment does not identify the number analyzed, and zero participants at risk does not establish the absence of harms. Clear denominators, analysis populations, estimates, and uncertainty are needed for interpretable comparisons.
13. 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 primary limitation is explicit: the registry reports that no outcome measure data are available because study data were lost. It states that the data were left behind when the principal investigator left the institution in 2013 and that the data location could not be found. No cardiac output values, secondary outcome results, or analysis outputs are reported. This prevents assessment of the planned treatment comparison.
Several design and analysis details are also absent. The factorial model is listed, but its factors and intended contrasts are not described. The record does not report allocation concealment, the randomization procedure, measurement timing beyond the Emergency Department care period, the statistical model, or how repeated measurements were to be handled. Masking is listed as none, but the roles of outcome assessors are not specified.
The registry does not report baseline characteristics, the number of participants contributing measurements, follow-up completeness, protocol deviations, or analysis populations. It mentions follow-up calls on day 3 and/or day 7, without describing their use in outcome assessment. The handling of missing measurements and the multiplicity approach are not reported in the registry record.
Safety interpretation is limited as well: the fields list zero participants at risk and zero affected, while death counts are not reported. These entries do not support estimates of event frequency. The record's enrollment figure cannot resolve these gaps. Together, these limitations mean that the study's planned question is identifiable, but its results and precision are not.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Descriptive Study of Cardiac Output During Rehydration With Recombinant Human Hyaluronidase in Pediatrics — NCT01139294.
- Registry record last updated 2018-02-01; status last verified 2018-01.
- Results first posted 2017-06-26.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
17. Record Summary
The registry identifies NCT01139294 as a completed Phase IV randomized study comparing Hylenex-augmented subcutaneous rehydration with standard IV hydration in pediatric patients with moderate dehydration. It lists Vanderbilt University Medical Center as sponsor, enrollment of 17, no masking, and a factorial model. The study began in 2010-02, with primary completion in 2011-11.
The primary outcome was Cardiac Output Trends: noninvasive measurements in liters per minute, with interest in onset and changes in slope during Emergency Department care. Gorelick Assessment is listed as a secondary outcome. The record reports no analyses and no available outcome measure data, stating that the study data were lost after the principal investigator left the institution in 2013.
Consequently, the registry describes the intended design and endpoints but does not provide results for efficacy, precision, or safety comparisons.