Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Hepatic Injury: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Hepatic Injury (NCT01325584), a na phase 2 study. The primary outcome is Maximum Conjugated Bilirubin.

2. Clinical Question

3. Trial Design

The registry identifies NCT01325584 as a Phase 2, single-group, unmasked compassionate-use study sponsored by Midwestern Regional Medical Center. Allocation is listed as NA. The intervention description states that all participants would receive intravenous Omegaven, a 10% fish oil emulsion, with parenteral nutrition for 4 weeks. There is no control arm described, so the design does not provide a like-for-like comparison between people receiving Omegaven and people receiving another treatment or no Omegaven.

The registry lists enrollment as 12 and the study status as terminated. The recorded start is 2012-10 and primary completion is listed as 2016-12. These record fields do not establish how many participants completed treatment, contributed to each outcome analysis, or had complete follow-up. The reason for termination and the circumstances around it are not reported in the registry record.

The stated purpose was to make Omegaven available to cancer patients with liver disease and to examine whether it could improve or prevent further liver disease. The summary also says that immune-function effects would be examined. These are stated objectives, not evidence of findings. The registry lists five outcome measures and three analyses, but the details necessary to understand all of those measures or analyses are not included in the available record details.

Because this was a single-group study, any change over time would be a within-group observation. Without a concurrent control group, the study cannot separate a treatment-associated change from changes that could occur over time for other reasons. The registry record does not report a statistical analysis plan, the planned analysis population, or the reason the study was terminated.

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:

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 Maximum Conjugated Bilirubin, measured over Assessed at day 1, 2, 3, and weekly therafter, up to 4 weeks..

The primary endpoint was maximum conjugated bilirubin, measured in mg/dl. The registry describes the outcome as a mean with standard deviation and says the highest detected laboratory values would be summarized between baseline and the end of study participation. Assessments were specified at day 1, day 2, day 3, and weekly thereafter, up to 4 weeks.

Conjugated bilirubin is a laboratory measure relevant to liver function and injury. In general, repeated measurements can describe how a laboratory marker varies during follow-up. Focusing on the highest detected value captures the peak recorded level over the defined observation period, rather than describing every measurement separately. The mean and standard deviation are ways to describe the distribution of a continuous measure: the mean gives a central summary, while the standard deviation describes variation around that summary.

The endpoint definition does not itself show whether the measure changed. The registry record does not report the observed mean, standard deviation, participant-level values, or a numerical comparison between baseline and the end of participation. It also does not explain how a maximum was selected if a participant had more than one eligible measurement, or how incomplete measurement schedules were handled. Accordingly, the specified measure indicates what the study intended to summarize, not what the study found.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary measure as a continuous outcome summarized by a mean and standard deviation. This is a descriptive approach: the mean indicates the central level of the recorded values, and the standard deviation describes their spread. The endpoint is specifically maximum conjugated bilirubin, rather than a general summary of every bilirubin observation. The record says the highest detected laboratory values would be summarized between baseline and the end of study participation.

What does a mean and standard deviation describe?

In general, the mean is useful for describing the center of a set of measurements when a single average is an informative summary. The standard deviation gives a sense of how much individual measurements vary around that average. These statistics do not show the full shape of the data, and a mean can be affected by unusually high or low observations. Since the endpoint uses the highest detected values, the interpretation also depends on the measurement schedule and on which observations were available. The registry record does not report the observed mean or standard deviation, so neither the central level nor the variation can be assessed.

What does the assessment schedule imply?

The planned timeframe includes assessments at day 1, day 2, day 3, and weekly thereafter, up to 4 weeks. Repeated assessment can capture values over time, while use of the maximum focuses attention on the peak detected value during the stated period. In general, a maximum can depend on how often measurements are taken: more opportunities to observe a high value can affect the recorded peak. This is a general measurement consideration, not a claim about how observations differed in this study. The registry does not explain how the maximum was derived for participants with incomplete assessments or whether all scheduled measurements were obtained.

Why does the single-group design matter?

In general, a single-group study can describe what was observed among people who received the intervention, but it cannot provide a concurrent estimate of what would have occurred under a comparator condition. A before-and-after pattern may be informative as a description, yet other changes during the same period can also influence the outcome. This means that a change during treatment cannot automatically be attributed to treatment. Here, the registry lists a single-group model, allocation as NA, and masking as none. No randomized comparator is described.

Unmasked measurement means that the registry does not describe masking of treatment assignment. In general, lack of masking can affect some aspects of study conduct or interpretation, depending on the outcome and how it is measured. The primary endpoint is a laboratory measure, but the registry record does not provide enough detail to assess how laboratory testing or outcome processing was performed. No assumptions about those procedures should be made.

What do the planning illustrations represent?

The sample-size assumptions describe a planning illustration computed from the observed effect, not the trial's own design. They specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect stated as a fraction of the observed effect. For non-time-to-event outcomes, the illustration is expressed as participants per arm for 80% power or other displayed power levels; time-to-event illustrations use a total event count. These are hypothetical planning quantities, not observed trial results and not evidence that the study had a particular power.

This illustration assumes two arms even though the recorded study is single-group. It therefore should not be read as a description of the actual trial's allocation or as evidence that a comparator arm existed. The registry does not report a sample-size justification for the actual study, and the planning illustration is not a substitute for one. It is also not observed power: general statistical practice does not treat a calculation based on an observed effect as proof that a study was adequately powered or that an effect is real.

What can be concluded from an absent estimate?

Inference about an effect ordinarily depends on an estimate and an assessment of its uncertainty. A confidence interval, in general, communicates the range of effect values compatible with the data under the stated model and assumptions. A test statistic or p-value may address a specified hypothesis, but neither is interpretable without knowing the analysis, data, and assumptions. The registry record does not report a primary endpoint estimate, confidence interval, p-value, or the analysis method used for the primary outcome. No recalculation from summary statistics is possible from the recorded details.

The registry lists five outcome measures and three analyses. The record details do not identify all the analyses, their statistical models, or whether adjustments were made for multiple outcomes or comparisons. In general, testing multiple hypotheses can increase the chance of observing a nominally unusual result by chance if no appropriate approach to multiplicity is used. Whether that concern applies to a particular reported analysis depends on what was tested and how the analyses were planned; those details are not reported in the registry record.

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("Omegaven (Compassionate Use)")
n   <- c(11)
m   <- c(1)
sp  <- c(0.1)
sd  <- sp      # posted standard deviations
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  Omegaven (Compassionate Use)  11  1.000  0.100  0.933  1.067

11. Multiplicity, Missing Data and Analysis Populations

The registry lists five outcome measures and three analyses, but the available record details do not specify the full set of outcomes, the hypotheses associated with the analyses, or how the analyses relate to one another. A multiplicity adjustment or other strategy for interpreting multiple comparisons is not reported in the registry record. In general, when multiple hypotheses are examined, the chance of at least one apparently unusual result may increase unless the analysis plan accounts for the number and structure of the tests. It is not possible to determine from the listed counts alone whether an adjustment was needed or used.

Missing-data handling is also not reported in the registry record. The planned primary endpoint involves repeated laboratory assessments, but there is no information about the number of measurements available, how missing visits were handled, or whether an analysis required complete follow-up. No imputation method, sensitivity analysis, or other missing-data procedure is specified in the record details.

The analysis populations are not reported in the registry record. Although the enrollment field lists 12, that field does not say how many participants were included in an outcome analysis or whether the analysis population followed a particular rule. The record also does not report participant flow, treatment exposure, or the number with evaluable baseline and end-of-participation data. These omissions limit interpretation of both the endpoint and the safety figures; the enrollment listing should not be substituted for an analysis denominator.

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 non-serious adverse event terms

The registry reports 0 participants affected by serious adverse events among 11 at risk in the Omegaven compassionate-use group. It reports 1 participant affected by other adverse events among 11 at risk, and lists 1 death. No serious adverse event terms are listed. These are the recorded safety figures and should be read as descriptive counts, not as estimates of comparative risk.

The record does not explain whether the death occurred during treatment or follow-up, what caused it, or whether it was considered related to the intervention. It also does not report whether the death overlaps with the other adverse-event entry or how adverse events were collected and classified. The serious adverse event figure and the other adverse event figure refer to separate registry categories, but the available information does not provide event-level detail.

In general, an adverse event occurring during a study is not necessarily caused by the study treatment. Determining relatedness requires clinical assessment and event details. The registry record does not provide enough information to attribute or rule out a relationship between Omegaven and any reported event. The listed counts also do not support a comparison with another treatment, because no comparator group is recorded.

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

The registry record describes a compassionate-use study of Omegaven with parenteral nutrition and specifies maximum conjugated bilirubin as its primary outcome. It also lists objectives concerning liver disease and immune function. These details establish what the study set out to examine; they do not establish what happened to participants. Numerical results for the primary endpoint are not reported in the registry record.

As a result, the record cannot show whether the maximum conjugated bilirubin values increased, decreased, or remained similar during participation. It does not give an effect estimate or a measure of precision. No conclusion about the size or certainty of a change can be drawn from the endpoint definition alone. The registry's enrollment and study status fields do not supply the missing outcome data or establish how many participants contributed to the primary analysis.

Even if a within-group change were available, a single-group design could describe change over time but would not identify what would have happened without Omegaven. In general, a concurrent comparison helps distinguish a treatment-associated effect from other influences that may change over the same period. This record lists no comparator and no random allocation. It therefore cannot support a randomized treatment-effect comparison.

The safety entries are also limited. They report serious and other adverse event counts and a death, but do not provide event descriptions, timing, cause, or relationship to treatment. They cannot establish whether Omegaven caused or prevented any safety outcome.

The most defensible interpretation is that the registry records the study's intended intervention, outcome, and design, along with limited safety counts, while leaving the main efficacy results and important analysis details unreported. The absence of numerical results is not evidence of benefit, lack of benefit, or harm. It is a limit on what can be concluded from this registry record.

14. Why This Trial Matters Statistically

This study illustrates an important distinction between an outcome being specified and an outcome being reported. The registry defines a clinically relevant laboratory endpoint and a schedule for observing it, but the numerical endpoint results are not reported in the registry record. Readers therefore cannot assess the direction, size, or precision of the outcome from the registry entries.

Its single-group compassionate-use design also highlights the difference between describing a clinical course and estimating a comparative treatment effect. A change over time can be documented, but without a concurrent comparator it is difficult to distinguish the intervention's contribution from other influences. This is a design limitation, not a reason to treat any unreported result as favorable or unfavorable.

The record also shows why sample-size planning illustrations must be kept separate from the actual design. The stated illustration uses hypothetical equal allocation between two arms, while the registered study is single-group. Such planning values are not observed outcomes, do not describe actual enrollment by arm, and are not observed power. Keeping those interpretations separate prevents a planning calculation from being mistaken for trial evidence.

Finally, safety counts require context. The registry reports event counts, but not details that would explain timing, severity beyond the listed categories, or attribution. The record's limitations are therefore relevant to both outcome interpretation and safety assessment. In statistical reporting, clear definitions, analysis populations, uncertainty estimates, and transparent missing-data methods are important because they allow readers to understand what the data can and cannot support.

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.

Several limitations specific to this record constrain interpretation. It is registered as a single-group, unmasked compassionate-use study, with allocation marked NA and no comparator described. Consequently, the design does not provide a concurrent basis for separating treatment-associated changes from other changes over time. The registry does not report the reason for termination, so the status cannot be interpreted as evidence about the intervention or about the completeness of data collection.

The primary outcome is defined as maximum conjugated bilirubin, with the highest detected values summarized between baseline and the end of participation. The registry does not report the actual outcome values, the number of observations available, or how maxima were handled when measurements were missing. The record also does not provide an analysis population, statistical model, confidence interval, or p-value for the endpoint. These omissions prevent assessment of the magnitude and precision of the outcome.

The registry lists enrollment as 12, but does not say how many participants completed the planned treatment or contributed data to each analysis. The stated 4-week treatment period does not establish that every participant received treatment for that duration. The start and primary-completion fields do not describe individual follow-up or explain why the study ended.

Safety interpretation is limited by the absence of adverse-event terms and details about the listed death. Timing, cause, treatment relationship, and any overlap between categories are not reported in the registry record. The record also does not state how missing data, multiplicity, or analysis populations were handled. These are limits of the information recorded, rather than proof that any specific method was or was not used.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Compassionate Use of Omegaven® in the Treatment of Parenteral Nutrition Induced Hepatic Injury — NCT01325584.
• Registry record last updated 2018-08-07; status last verified 2018-07.
• Results first posted 2018-06-27.
• 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

NCT01325584 is a Phase 2, single-group, unmasked compassionate-use study sponsored by Midwestern Regional Medical Center. The intervention was intravenous Omegaven, described as a 10% fish oil emulsion with parenteral nutrition for 4 weeks. The registry lists enrollment as 12 and status as terminated; the reason for termination is not reported in the registry record.

The primary outcome was maximum conjugated bilirubin in mg/dl, summarized as a mean and standard deviation over the stated assessment period. Numerical primary outcome results, analysis methods, and analysis populations are not reported in the registry record. Safety fields list 0 participants with serious adverse events among 11 at risk, 1 with other adverse events among 11 at risk, and 1 death. Event details and treatment relationships are not reported. With no comparator group and no numerical primary outcome results, the registry record does not establish a treatment effect.