Docetaxel and Liposomal Doxorubicin Chemotherapy With Enoxaparin in Patients With Advanced Pancreatic Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Docetaxel and Liposomal Doxorubicin Chemotherapy With Enoxaparin in Patients With Advanced Pancreatic Cancer (NCT00426127), a na phase 2 study. The primary outcome is Tumor Response Measured by CT Scans After Each Set of 3 Cycles of Chemotherapy.

2. Clinical Question

This question is about an observed tumor response under a treatment combination, not a direct comparison with another treatment. In general, a comparative question needs outcomes measured under both the intervention and a suitable comparator; the registry record does not describe such a comparator group. The study’s stated objective and its primary outcome are related, but the objective does not establish that tumor shrinkage was observed. The record does not report a response count or a statistical analysis of response. Its description of progression for one participant is not, by itself, a complete account of the primary outcome for all enrolled participants.

3. Trial Design

The registry identifies this as a Phase II, single-group trial of docetaxel and liposomal doxorubicin combined with enoxaparin in patients with advanced pancreatic cancer. The sponsor is the University of Iowa. The record lists enrollment as 2, allocation as NA, and masking as none. It reports a start date of 2006-11, a primary completion date of 2009-01, and a status of terminated.

The listed regimen combines docetaxel 75 mg/m², liposomal doxorubicin (Doxil) 30 mg/m² and enoxaparin 1.5 mg/kg. The record does not describe a separate control arm. Because the trial is single-group and allocation is listed as NA, it is not a randomized comparison, and there is no like-for-like control group in the registry description. The absence of masking means that the record does not report blinding.

The stated purpose was to assess the treatment combination and whether it increased the number of individuals whose tumors shrink. The primary outcome specifies CT-based tumor response after each set of 3 chemotherapy cycles, with a timeframe of 9 weeks. The outcome description says that one participant underwent 3 cycles and progressed as assessed by CT.

The record also lists secondary outcomes involving D-dimer: the number of blood draws with elevated D-dimer measured every cycle, and the safety and effect of the chemotherapy regimen on D-dimer, also measured every cycle. It does not report numerical results for those outcomes. Further details about eligibility, treatment delivery, follow-up procedures, or reasons for termination are not reported in the registry record.

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:

Analysis population for the primary outcome, as defined in the registry: One participant underwent 3 cycles of therapy and progressed as assessed by CT.

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 Tumor Response Measured by CT Scans After Each Set of 3 Cycles of Chemotherapy, measured over 9 weeks.

The primary endpoint is titled tumor response measured by CT scans after each set of 3 cycles of chemotherapy. It is expressed as a count of participants, and its timeframe is 9 weeks. The registry describes the outcome as assessing whether the combination increases the number of individuals whose tumors shrink. It also records that one participant underwent 3 cycles and progressed as assessed by CT.

In general, imaging-based tumor response is used to describe how a tumor changes during treatment. A count of participants can communicate how many people met a defined response category, but the registry record does not specify the response criteria, the categories used, or the exact rule for counting a response. It does not give a complete response count or a statistical analysis of this endpoint. The participant-level progression statement should therefore be read as the specific outcome information reported, not as a complete estimate of the response rate or a comparison of treatments.

The record names CT assessment after a set of treatment cycles, but does not report details such as independent image review, assessment masking, handling of scans that were not completed, or confirmation procedures. Those details matter because endpoint definitions and assessment procedures affect how consistently a response outcome can be interpreted. They are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods describe how trial questions are translated into outcomes, comparisons, and uncertainty estimates. The registry record identifies a primary outcome and gives some design information, but reports no analyses. The methods below explain how to read the available information and distinguish general statistical principles from analyses documented for this trial.

What does a single-group design allow?

The trial is listed as single-group, with allocation NA and no masking. In general, a single-group study can describe outcomes observed among participants receiving the studied regimen. A comparative treatment effect requires a comparator and a defined contrast between groups or conditions. Since the record does not describe a control group, there is no randomized between-group contrast to interpret. The outcome description can be reported as an observation, but it cannot establish what would have happened to comparable participants under a different treatment.

Random allocation, when used, helps make comparison groups comparable on average with respect to measured and unmeasured factors. That general protection does not apply to a comparison that is absent. The record gives no basis for attributing the outcome described to the regimen rather than to other influences, and no basis for estimating a comparative effect.

What is the primary endpoint measuring?

The endpoint is a count of participants with tumor response measured by CT after each set of 3 cycles; the stated timeframe is 9 weeks. The record says one participant underwent 3 cycles and progressed by CT. In general, an endpoint needs a clear definition of what qualifies as a response, when it is assessed, and how incomplete assessments are handled. Those specifications help ensure that the same outcome is applied consistently to each participant. The registry record does not report response criteria, a complete count of response categories, or how missing scans would be handled.

Counts are direct descriptions of events or outcomes. A proportion can sometimes be estimated from a count and a defined denominator, but a sound interpretation requires knowing who was eligible for that denominator and whether each participant had an assessable outcome. The record does not provide a complete endpoint summary or an analysis population. No response proportion should be inferred from the brief outcome description alone.

How should uncertainty be understood?

In general, a confidence interval describes the range of effect values compatible with the data and a specified statistical procedure. Its width reflects both the amount of information and the variability in the data. A narrow interval indicates greater precision than a wide interval, while neither interval alone resolves questions of bias or applicability. The registry record reports no confidence interval for the primary endpoint.

A p-value, in general, quantifies how incompatible the observed data are with a specified null model, under the assumptions of the test. It is not the probability that a treatment works, and it does not measure the size or importance of an effect. No p-value or primary-outcome test is reported in this registry record. Without a reported test, a significance claim cannot be made from the record.

What do the planning calculations represent?

The planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It considers detecting a stated fraction of the observed effect. Except for time-to-event calculations, which are described as total event counts using the Schoenfeld method, the planning quantities are participants per arm for 80% power or other stated power levels. These are planning assumptions, not the trial’s observed results, and not evidence that the completed study had a particular power.

General sample-size planning asks how much information a future study would need to detect a specified effect under stated assumptions. Such calculations depend on the effect used for planning and the design, allocation, significance threshold, and statistical method. A planning illustration is not a substitute for a trial’s protocol or its statistical analysis plan. The registry record does not report the trial’s own sample-size justification, planned target enrollment, or protocol-based analysis plan. The numerical planning assumptions do not establish that the trial used a two-arm design; the reported trial itself is single-group.

Why can over-dispersion matter?

In general, over-dispersion occurs when count outcomes vary more than a statistical model assumes. If a model ignores that extra variation, uncertainty may be understated and tests may be poorly calibrated. This consideration can be relevant for some count outcomes, but the registry record does not report a model for the primary endpoint, a dispersion estimate, or any analysis of over-dispersion. It would be inappropriate to claim that such a model was used in this trial.

The listed secondary outcomes involve repeated D-dimer measurements every cycle. Repeated observations on the same participant are generally related, so analyses need to account for within-participant dependence when making inferential claims. The registry does not report an analysis method, the number of D-dimer observations, or the results of those secondary outcomes. No model or inferential result for them can be attributed to the trial.

Finally, statistical interpretation depends on transparent reporting of the outcome definition, analysis population, missing-data approach, and analysis plan. The record provides a limited outcome description and no analyses. Accordingly, general statistical concepts can explain what would be needed for estimation and inference, but they cannot fill in results or methods absent from the registry record.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Docetaxel and Liposomal Doxorubicin Combined With ")
n      <- c(1)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Docetaxel and Liposomal Doxorubicin Combined With   1  1  100.0  2.5  100.0  20.7  100.0  15.8  98.7

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one primary outcome and secondary outcomes concerning elevated D-dimer and the effect and safety of the chemotherapy regimen on D-dimer. It reports 3 outcome measures and 0 analyses. No statistical analysis results or plan for controlling multiplicity are reported in the registry record.

In general, multiplicity arises when several outcomes, comparisons, time points, or subgroup questions are tested. Considering many tests can increase the chance of at least one apparently notable result when there is no underlying effect. Statistical plans may address this through a prespecified hierarchy or an adjustment, but the registry does not report whether either approach was planned or used. No adjustment should be assumed.

The record gives a primary endpoint timeframe of 9 weeks and describes CT assessment after each set of 3 cycles. It does not specify whether all planned assessments were completed, how incomplete scans or withdrawals were handled, or how participants were assigned to an analysis population. Missing-data methods and analysis populations are not reported in the registry record.

These omissions matter because the interpretation of a count depends on which participants were included and whether outcome information was available for all of them. The reported enrollment and brief progression statement do not provide a complete participant flow or endpoint accounting. The record also does not explain the reason for termination or how termination affected follow-up and analysis. Without those details, the completeness and intended scope of the outcome summary cannot be assessed.

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 serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: disease progression (50.0%).

The registry reports serious adverse events for a group titled Docetaxel and Liposomal Doxorubicin Combined With Enoxaparin. It lists 2 at risk and 1 affected for serious adverse events. For other adverse events, it lists 2 at risk and 2 affected. The record also lists 2 deaths. Disease progression is the top serious term, reported as 50.0%.

These are descriptive counts in the registry. They do not, by themselves, show that an event was caused by the treatment. The record does not report the cause of death, an attribution assessment, event timing, severity details beyond the serious-event classification, or a comparison with an untreated or control group. It also does not clarify the denominator underlying the 50.0% term entry. The percentage should therefore be read as the registry’s reported figure, not as a treatment-risk estimate that can be independently interpreted from the available details.

In general, safety comparisons require clear definitions, systematic event collection, and appropriate comparison groups or contextual information. The record’s small reported enrollment and absence of a comparator limit descriptive interpretation. The counts do not establish whether the regimen increases or decreases risk, and they should not be taken as a causal safety conclusion.

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

The registry record establishes that a Phase II, single-group trial of docetaxel, liposomal doxorubicin and enoxaparin was conducted for advanced pancreatic cancer, with enrollment reported as 2 and status listed as terminated. Its primary outcome concerned CT-measured tumor response after each set of 3 chemotherapy cycles over a timeframe of 9 weeks. The outcome description states that one participant completed 3 cycles and progressed by CT.

That information does not provide a complete response result. The record does not give a total count of participants with tumor shrinkage, the criteria for defining response, a confidence interval, or a statistical test. The brief progression statement cannot stand in for a complete outcome summary, and it does not allow a response rate or treatment effect to be established.

The single-group design also means there is no randomized comparator for judging what would have happened under another treatment. Even a complete description of outcomes in the treated group would not, on its own, establish a causal treatment effect. The registry record does not report a comparison or an analysis that quantifies such an effect.

The adverse-event entries describe serious and other adverse events and deaths, but do not establish that these events were caused by treatment. The record does not explain the reason for termination, the completeness of follow-up, or the handling of missing outcomes. Those gaps further limit interpretation.

In general, a trial result is most informative when its endpoint is fully defined, participant flow is clear, analyses are reported with uncertainty, and the design supports the intended comparison. The available registry entries do not meet that standard for estimating comparative efficacy or safety. They document the study design and a limited outcome observation; they do not prove that the regimen does or does not alter tumor response, or that it does or does not cause harm.

14. Why This Trial Matters Statistically

This trial is statistically notable because the gap between a treatment question and an interpretable treatment effect is visible in the registry record. The stated objective concerns whether the combination increases the number of individuals whose tumors shrink, but the listed design is single-group and no comparator is reported. A question about an increase implies a comparison; without a comparator and a reported contrast, the record cannot quantify an increase.

The primary endpoint is described as a participant count based on CT response after treatment cycles, but the registry offers only a brief participant-level progression statement rather than a complete response summary. This illustrates why an endpoint title and a timeframe do not, on their own, provide an analyzable result. A full interpretation needs a clear response definition, complete outcome accounting, and an estimate with its uncertainty.

The record also shows why planning calculations must be kept separate from observed evidence. Planning illustrations depend on assumptions about effect size and design. They do not establish that the completed study was adequately powered, nor do they replace an analysis of observed outcomes. In this case, the posted record reports 0 analyses.

Finally, the safety entries illustrate the difference between counts and causal conclusions. Event counts are important descriptive information, but attribution and comparison are needed to interpret risk. The registry does not report those details. The statistical lesson is not a conclusion about the regimen; it is that clear design, outcome reporting, and analysis are essential before a clinical question can be answered with precision.

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 registry record constrain interpretation. The study is listed as single-group, with allocation NA and no masking, and no comparator is described. It therefore does not provide a randomized treatment comparison. The registry reports enrollment as 2, which leaves little information for estimating outcomes, but it does not state the planned enrollment or explain whether the reported enrollment was the intended target.

The status is terminated, yet the reason for termination is not reported in the registry record. The record does not describe participant flow, explain whether all enrolled participants completed follow-up, or report the analysis population. It is not possible to determine from the posted information how termination affected the planned outcome assessment.

The primary outcome is identified as CT-measured tumor response after each set of 3 cycles, with a timeframe of 9 weeks. However, response criteria, a complete set of outcome counts, and an inferential analysis are absent. The record’s statement that one participant underwent 3 cycles and progressed does not provide a complete accounting of primary outcomes. Missing-data methods, handling of unevaluable scans, and the analysis plan are not reported.

The record lists secondary D-dimer outcomes but provides no numerical results or analyses for them. It also reports adverse-event counts and deaths without details on event timing, treatment attribution, or a comparison group. The registry does not explain the reason for termination or provide information that would resolve these interpretive gaps. These limitations apply to what can be concluded from the registry record; they do not establish what may or may not have been reported elsewhere.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Docetaxel and Liposomal Doxorubicin Chemotherapy With Enoxaparin in Patients With Advanced Pancreatic Cancer — NCT00426127.
• Registry record last updated 2017-12-29; status last verified 2017-12.
• Results first posted 2017-06-02.
• 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

The registry describes NCT00426127 as a terminated Phase II, single-group study sponsored by the University of Iowa. It evaluated docetaxel, liposomal doxorubicin and enoxaparin in patients with advanced pancreatic cancer. The listed regimen was docetaxel 75 mg/m², Doxil 30 mg/m² and enoxaparin 1.5 mg/kg. Enrollment was 2; masking was listed as none, and allocation as NA.

The primary outcome concerned CT-measured tumor response after each set of 3 chemotherapy cycles, with a timeframe of 9 weeks. The outcome description says one participant underwent 3 cycles and progressed by CT. Secondary outcomes concerned D-dimer measurements. The registry reports 0 analyses and does not provide a complete primary-outcome summary or statistical results.

The adverse-event record lists 1 affected for serious events, 2 affected for other events, 2 deaths, and disease progression as the top serious term at 50.0%. The record does not report a comparator, the reason for termination, or enough analysis and attribution detail to establish treatment effects or causes of the reported events.