Ubiquinol (Reduced COQ10) for Patients With Sepsis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ubiquinol (Reduced COQ10) for Patients With Sepsis (NCT01948063), a randomized phase 2 study. The primary outcome is CoQ10 Levels.

2. Clinical Question

The registry describes a study intended to assess whether ubiquinol might attenuate mitochondrial injury and decrease inflammatory response in patients with sepsis. The posted primary endpoint is a measurement of total CoQ10 levels, rather than a direct measure of mitochondrial injury, inflammation, or a patient-centered clinical outcome. It therefore addresses a narrower question: how the recorded CoQ10 concentrations compare between the randomized groups at the specified time.

The reported median was 0.87 mcg/mL in the ubiquinol group and 0.38 mcg/mL in the placebo group. The registry also gives an inter-quartile range for each group. These summaries describe the observed concentrations; they do not, by themselves, establish whether the groups differ beyond what could occur by chance or indicate the precision of an estimated treatment effect. No statistical comparison or analysis result is reported in the registry record.

3. Trial Design

This completed phase 2 study is registered as NCT01948063 and is titled “Ubiquinol (Reduced COQ10) for Patients With Sepsis.” Beth Israel Deaconess Medical Center is listed as the sponsor. The registry records a randomized, quadruple-masked, parallel design and an enrollment of 38. The start is recorded as 2012-10 and primary completion as 2014-04.

The two reported groups are Ubiquinol and Placebo, with 19 participants in each group in the primary outcome results. The ubiquinol arm description specifies 200mg, delivered as a pill or liquid according to the patient's ability to swallow pills. Its liquid form was mixed with 50 milliliters of Ensure to support blinding. The arm description is incomplete in the registry record, so the complete dosing schedule and duration for ubiquinol are not reported in the registry record.

The placebo description likewise allows a pill or liquid, according to the patient's ability to swallow pills. The liquid placebo is described as 50 milliliters of Ensure. The registry says the control treatment was given every 12 hours for 7 days or until hospital discharge. It does not state which individuals received pills or liquid, or report adherence to the assigned regimen.

Random allocation and masking are design features intended to reduce bias in group assignment and outcome assessment. The registry records quadruple masking, but the identities of the masked parties and the allocation procedure are not reported in the registry record. The record reports one primary outcome measure, CoQ10 levels, assessed 24 hours after study-drug administration. It does not report a statistical analysis result for that outcome.

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: 1 patient was randomized in the placebo group but the blood was accidentally thawed why this patient was excluded. 2 patients were randomized in the Ubiquinol group but never received the treatment (one because of an exclusion criteria and one because of a Boston-wide mass casualty event). These were also excluded.

Overall Study

Reasons for not completing this period:

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 CoQ10 Levels, measured over 24 hours after study drug administration.

The primary endpoint is total CoQ10 concentration, measured in mcg/mL, 24 hours after study-drug administration. The registry describes the result using a median and an inter-quartile range. The median represents the middle of the ordered observations, while the inter-quartile range describes the spread of the central portion of the observed values. These summaries can be useful when measurements are skewed or include extreme observations, because the median is less influenced by extremes than the arithmetic mean.

CoQ10 is the substance named in the outcome, and the endpoint supplies a biochemical measurement at a defined follow-up time. Such a measurement can be used to examine whether the groups' recorded levels differ after treatment assignment. It is not itself a direct clinical outcome, nor does its value alone establish whether mitochondrial injury or inflammation changed. The registry's stated study aim refers to those processes, but no corresponding injury or inflammatory endpoint result is included in the reported outcome.

The placebo group's median was 0.38 mcg/mL, with an inter-quartile range from 0.26 to 0.66. The ubiquinol group's median was 0.87 mcg/mL, with an inter-quartile range from 0.57 to 1.26. An inter-quartile range describes variation among observed values; it is not a confidence interval for the difference between groups. The registry record does not report a confidence interval, hypothesis test, or adjusted estimate for this endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the median describe?

The median is the central value after measurements are ordered. It can describe a typical observation without being pulled as strongly by unusually high or low values as an arithmetic mean can be. This is general statistical context. In this trial, the primary outcome is explicitly summarized using medians, with the placebo median reported as 0.38 mcg/mL and the ubiquinol median as 0.87 mcg/mL.

A median is a description of observations in one group. It is not automatically the best estimate of a treatment effect, because a treatment effect is a comparison between groups and may be defined in several ways. For example, an analysis could estimate a difference in medians or use a model suited to the outcome distribution. The registry record does not report which comparative estimand or method was used.

What does the inter-quartile range describe?

The inter-quartile range describes the central spread of observed values, from the lower quartile to the upper quartile. It gives information about variability within a group, rather than uncertainty around a treatment-effect estimate. The reported range is 0.26 to 0.66 in the placebo group and 0.57 to 1.26 in the ubiquinol group. The two ranges overlap, but overlap or lack of overlap between such summaries is not, on its own, a formal test of a group difference.

As general statistical context, the choice of summary and analysis can depend on the shape of the data and the scientific question. If values are strongly skewed, a mean-based method may be sensitive to extreme observations; other methods may be more suitable. The registry record does not report the distribution of individual measurements or the method used to compare the groups. The summary statistics therefore do not identify the analysis assumptions or establish whether those assumptions were checked.

How is uncertainty in a comparison assessed?

A statistical comparison typically estimates a contrast between the assigned groups and quantifies its uncertainty. A confidence interval is one common way to describe that uncertainty. It indicates a range of values compatible with the data under the method's assumptions; it is not a range expected to contain most individual measurements. A p-value, when reported, addresses the compatibility of the data with a specified null hypothesis under a statistical model. Neither a confidence interval nor a p-value is reported for the primary endpoint in the registry record.

Because the registry supplies group medians and inter-quartile ranges but no analysis result, a reader should not infer a formal level of evidence from the apparent separation of the medians. Group summaries do not account explicitly for sampling uncertainty, baseline values, missing measurements, or any covariates. Whether such considerations were addressed in the sponsor's analysis is not reported in the registry record.

Why can over-dispersion matter?

As general statistical context, over-dispersion occurs when observed variability is greater than a statistical model assumes. If a model understates variability, its standard errors can be too small and its confidence intervals too narrow; inferential conclusions can then be overconfident. The appropriate response depends on the outcome type and analysis model and might involve a different variance specification or another model. The registry record does not report a model for the primary outcome, so it is not possible to assess whether over-dispersion was relevant or how variability was handled in an analysis.

What do the planning assumptions mean?

The registry describes planning illustrations based on the observed effect, not on the trial's own design. The stated assumptions include a two-sided significance level of 0.05, equal allocation between groups, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. A planning calculation estimates the sample size needed under its assumptions; it does not show that a study with its actual enrollment had a particular probability of detecting an effect. It is not observed power and should not be used as a substitute for the unreported endpoint analysis.

Such illustrations also depend on how the effect is defined and on assumptions about variability and the statistical test. When the observed effect is used as the starting point, the calculation is conditional on that estimate and does not independently validate it. The registry record does not report the trial's sample-size justification, planned target effect, or underlying variance assumptions. No numerical sample-size results are described because those values are not reported in the registry record.

What can be inferred from the record?

The registry reports a randomized, quadruple-masked, parallel study and provides descriptive primary outcome results. Randomization is generally intended to make treatment groups comparable on average, while masking is generally intended to reduce biases in care or outcome assessment. These are general purposes of the design features; their success in a particular trial cannot be judged from the design labels alone. Allocation concealment details, the masked roles, the analysis method, confidence intervals, and hypothesis tests are not reported in the registry record.

The absence of a posted analysis result is an important boundary on interpretation. Descriptive measurements can be stated precisely as reported, but a comparative conclusion requires an analysis that addresses uncertainty and specifies the comparison. The registry record does not show whether a separate analysis was conducted outside the posted results.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity arises when several outcomes, time points, groups, or statistical tests are examined. As general statistical context, conducting many tests can increase the chance that at least one result appears noteworthy by chance. A prespecified analysis plan may address that issue by identifying a primary endpoint and defining how additional comparisons will be interpreted or adjusted.

The registry lists one outcome measure, CoQ10 levels, and reports zero analyses and zero posted secondary outcomes. Those registry entries do not establish that no additional outcomes or analyses were considered outside the record. A multiplicity adjustment strategy or complete statistical analysis plan is not reported in the registry record. Consequently, the handling of multiple comparisons cannot be assessed.

Missing-data methods are also not reported in the registry record. The primary outcome results include 19 participants in each group, but the record does not specify whether these are all randomized participants, whether any measurements were unavailable, or how missing measurements were handled. It also does not report an analysis population, such as whether the analysis followed assigned treatment regardless of adherence. Without participant flow and analysis-population details, readers cannot determine how closely the reported summaries correspond to the randomized groups as a whole.

These omissions do not show that missing data or multiplicity affected the results. They limit what can be evaluated from the registry entry. The registry record also does not report whether interim analyses were planned or performed.

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 reports serious adverse events for 0 of 19 participants in the ubiquinol group and 0 of 19 participants in the placebo group. It reports other adverse events for 1 of 19 participants in the ubiquinol group and 0 of 19 participants in the placebo group. Deaths are listed for 4 participants in the ubiquinol group and 2 in the placebo group.

These are event counts as recorded, not estimates of a causal effect. A count alone does not establish whether an event was caused by treatment, whether the groups differed beyond chance, or whether events were collected and classified in comparable ways. The record does not give further detail about the reported deaths, their timing, causes, or relationship to study treatment. It also does not provide a statistical comparison of safety outcomes or additional descriptions of the other adverse event.

Zero reported serious adverse events in a group does not establish that the risk is zero. General statistical context: when events are uncommon, a study can record no events while uncertainty about the underlying event rate remains. The registry record does not provide confidence intervals for these safety counts. The counts should therefore be read as descriptive reports for the recorded groups, not as proof of safety or evidence that one regimen is safer.

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

The posted primary result describes total CoQ10 concentrations 24 hours after study-drug administration. The median was 0.87 mcg/mL in the ubiquinol group and 0.38 mcg/mL in the placebo group; the reported inter-quartile ranges were 0.57 to 1.26 and 0.26 to 0.66, respectively. Those values state what the registry reports for the endpoint. They are not a formal estimate of the uncertainty in a treatment effect.

The result does not, by itself, answer whether ubiquinol attenuated mitochondrial injury or reduced inflammatory response. Although those processes appear in the study aim, the reported primary outcome is CoQ10 level, and the registry does not include results for a direct measure of either process. The biomarker finding also cannot establish an effect on clinical outcomes such as survival or recovery; those results are not reported in the registry record.

The record does not provide a between-group statistical test, confidence interval, or adjusted analysis for the primary endpoint. It is therefore not possible to judge the precision of the comparison or assess its statistical uncertainty from the posted summaries alone. The group medians should not be treated as proof of a treatment effect, and the lack of an analysis result should not be treated as proof that there is no effect.

Safety counts are similarly descriptive. The record lists no serious adverse events in either group, other adverse events for 1 participant in the ubiquinol group and none in the placebo group, and deaths for 4 and 2 participants, respectively. These entries do not establish causation or comparative safety, and the registry does not provide further death details or an analysis of these outcomes.

Overall, the registry supports a narrow description of the primary biomarker summaries and recorded adverse-event counts. Broader conclusions about biological mechanisms, clinical outcomes, or treatment effects would require information and analyses not reported in the registry record.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the difference between descriptive outcome reporting and comparative inference. The registry gives a median and inter-quartile range for CoQ10 in each randomized group, which allows readers to describe the observed measurements. A claim about a group effect, however, requires an estimate of the contrast and an assessment of its uncertainty. The registry record does not report that analysis.

The endpoint also illustrates why the outcome being measured matters. A biomarker can be relevant to a biological hypothesis, but it is not interchangeable with a measure of clinical benefit or harm. Here, the stated study aim concerns mitochondrial injury and inflammatory response, while the reported primary endpoint is total CoQ10 at a single specified time. The posted data therefore speak directly to that measurement, not automatically to the broader processes or to patient outcomes.

The design labels—randomized, quadruple-masked, and parallel—identify features intended to support a fair comparison. Yet labels do not replace information about implementation, participant flow, missingness, analysis populations, or statistical methods. Those details are not reported in the registry record. Their absence makes it difficult to evaluate the strength and robustness of an inference based on the posted summaries.

The registry's planning assumptions also provide a useful distinction between design calculations and observed results. A sample-size illustration based on an observed effect is conditional on its assumptions and is not a measurement of the trial's realized power. It cannot supply a missing test or confidence interval. Taken together, these features show why accurate interpretation requires separating the observed biomarker summaries, the planned design, and the unreported comparative analysis.

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 trial-specific limits constrain interpretation. The registry lists one primary outcome, total CoQ10 levels at 24 hours, and does not report a comparative statistical analysis, confidence interval, or p-value. The primary result is therefore descriptive in the registry record. The study aim also refers to mitochondrial injury and inflammatory response, but results for direct measures of those processes are not reported in the registry record.

The record gives 19 participants per group for the primary outcome but does not explain whether measurements were missing, whether every randomized participant was included, or how the analysis population was defined. Missing-data procedures and participant flow are not reported in the registry record. Baseline characteristics and individual outcome values are also not reported in the information, limiting assessment of group comparability and distributional shape beyond the medians and inter-quartile ranges.

The ubiquinol arm description is incomplete in the registry record. It specifies the dose and pill-or-liquid delivery, but the complete dosing schedule and duration are not reported in the registry record. The identity of the masked parties and details of allocation procedures are likewise not reported.

Safety reporting is limited to counts. The record does not provide further descriptions of the deaths, their causes or timing, or a treatment-relatedness assessment. It also does not report statistical comparisons for adverse events. The trial's sample-size justification, interim-analysis procedures, and full statistical plan are not reported in the registry record. These limitations restrict assessment of precision, bias, and the scope of conclusions that can be drawn from the posted information.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Ubiquinol (Reduced COQ10) for Patients With Sepsis — NCT01948063.
• Registry record last updated 2017-04-24; status last verified 2017-03.
• Results first posted 2017-04-24.
• 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

NCT01948063 is a completed phase 2 randomized, quadruple-masked, parallel study sponsored by Beth Israel Deaconess Medical Center. The condition is sepsis; the recorded start is 2012-10 and primary completion is 2014-04. The registry records enrollment of 38.

The primary outcome is total CoQ10 levels in mcg/mL at 24 hours after study-drug administration, summarized by median and inter-quartile range. The placebo median was 0.38, with a range of 0.26 to 0.66; the ubiquinol median was 0.87, with a range of 0.57 to 1.26. No statistical analysis result is reported in the registry record.

The registry reports no serious adverse events in either group, other adverse events for 1 participant in the ubiquinol group and none in the placebo group, and deaths for 4 and 2 participants, respectively. Further clinical details and comparative safety analyses are not reported in the registry record.