Epirubicin and Vinorelbine in Treating Patients With Stage II, Stage III, or Stage IV Breast Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Epirubicin and Vinorelbine in Treating Patients With Stage II, Stage III, or Stage IV Breast Cancer (NCT00176488), a na phase 2 study. The primary outcome is Efficacy of the Sequential Use of a DNA Damaging Drug (Epirubicin) Followed by a Vinca Alkaloid (Vinorelbine) in the Treatment of Breast Cancer..

2. Clinical Question

In practical terms, the registry frames a question about whether the sequential regimen could be evaluated for efficacy in the described patient population. The record does not support an estimate of how often a defined clinical outcome occurred, how long it took to occur, or how outcomes differed from another treatment. Because the trial has one group and lacks a posted efficacy analysis, the clinical question remains unresolved in this record. The secondary aims concern biological response in tumor biopsies and peripheral blood mononuclear cells, and the relationship between tumor response and changes in MAP4 gene expression; the registry does not report results for those aims.

3. Trial Design

This is a phase II, single-group study of sequential epirubicin and vinorelbine in patients with breast cancer. The registry lists the allocation as not applicable and masking as none. The record therefore describes an unmasked treatment group rather than a randomized design with a concurrent control. Enrollment is listed as 31. The study status is terminated; its start is recorded as 2003-06 and primary completion as 2009-10.

The treatment group is titled Sequential epirubicin/vinorelbine. According to the arm description, patients with stage IIB (T3N0), IIIA, or IIIB breast cancer were to receive the drugs for up to 5 cycles. For patients with stage IV disease, treatment was described as continuing while there was evidence of response or stable disease; the full stopping details are not reported in the registry record. The record does not provide the drug doses or a complete administration schedule.

The registry lists 31 participants at risk in the adverse-event group. That safety denominator is not, by itself, evidence that every enrolled participant contributed data to every efficacy outcome. The primary outcome entry states that the study closed prematurely and insufficient data was collected. The registry does not report the number analyzed for efficacy, the reasons for termination, or the specific timing and rules used to determine analysis populations.

A single-group phase II design can describe outcomes among treated participants and may help characterize feasibility or safety. Without a concurrent comparison group, however, observed outcomes cannot establish how they compare with outcomes under another treatment or with the course of disease in the absence of this regimen. The registry record does not report a protocol-level statistical analysis plan, allocation procedure beyond the single-group designation, or details of interim monitoring.

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: Study was closed prematurely and insufficient data was collected.

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 Efficacy of the Sequential Use of a DNA Damaging Drug (Epirubicin) Followed by a Vinca Alkaloid (Vinorelbine) in the Treatment of Breast Cancer., measured over 10 years.

The primary outcome is described as the efficacy of sequential use of epirubicin followed by vinorelbine in the treatment of breast cancer. Its stated timeframe is 10 years. The registry does not identify the unit, parameter, or dispersion for this outcome, and the outcome description does not specify the clinical measure or its definition. In particular, it does not state whether efficacy was to be represented by tumor response, time to an event, survival, or another measure. The registry record therefore does not allow the primary endpoint to be reconstructed as a precise statistical quantity.

In general, a prespecified primary endpoint focuses a trial on an outcome intended to answer its main research question. A clear endpoint definition specifies what is measured, how it is assessed, and the time period or event rule used. This supports consistent measurement and interpretation. A timeframe alone does not define an endpoint: the record also needs to identify the outcome and how it is calculated or classified.

Here, the registry explicitly says the study closed prematurely and insufficient data was collected. It does not report a primary efficacy estimate or an analysis of that endpoint. Consequently, the stated aim and timeframe describe the intended question, not an observed efficacy result. The biological aims listed as secondary outcomes are distinct from the primary efficacy question, and results for those measures are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a phase II study with one treatment group. There is no randomized allocation or masked comparison listed. This design determines the kinds of conclusions the data can support: outcomes can be described for the treated group, but treatment effects relative to a concurrent control cannot be estimated from this trial alone. The registry record does not report a detailed statistical analysis plan, so the intended analytic methods for the efficacy question are not known.

What does a single-group design show?

In general, a single-group study records outcomes among people who receive the study regimen. Such observations can describe the experience of that group under the study conditions. A comparison with another treatment requires a suitable reference group and a defined contrast. Without that, differences from historical expectations can be difficult to interpret because patient selection, disease characteristics, supportive care, assessment practices, and other factors may differ. This is a general design limitation, not evidence that any particular factor affected this trial.

In a randomized comparison, assignment helps balance known and unknown prognostic factors on average between groups. This record lists no random allocation and no comparator. Consequently, no randomized treatment contrast is available. The absence of masking also means treatment assignment was not concealed from participants or investigators as part of the recorded design. The registry does not describe whether outcome assessors were masked separately.

Why does endpoint definition matter?

A statistical analysis requires a measurable outcome. For example, a response endpoint needs criteria for deciding what counts as response and when it is assessed; a time-to-event endpoint needs a defined event and a rule for handling participants who have not experienced it by the end of follow-up. These are general requirements, not claims about the endpoint chosen for this study. The primary outcome is titled as efficacy of sequential drug use and has a timeframe of 10 years, but the registry does not report the operational endpoint definition, unit, or parameter. As a result, a reader cannot determine which statistical model or summary would be appropriate from the registry entry alone.

In general, an estimate should be interpreted together with its uncertainty. A confidence interval describes a range of values compatible with the data and the assumptions of the analysis; a wider interval indicates less precision. A significance test addresses a different question, evaluating compatibility with a specified null hypothesis under model assumptions. Neither an interval nor a test can supply a missing endpoint definition or overcome insufficient outcome data. The registry does not report an efficacy estimate, confidence interval, or analysis result for the primary outcome.

How should the safety counts be read?

The adverse-event entry gives the number at risk and the number affected for serious and other adverse events. These counts are useful descriptive information, but their interpretation depends on definitions and follow-up. A participant-based count differs from a count of repeated events. A rate also depends on the time participants were observed. The registry does not report enough detail to determine whether the term percentages represent participants or events, how recurrent events were handled, or whether follow-up duration was comparable across participants. In general, safety summaries should distinguish the number of people affected from the total number of events and should specify the observation period and event definitions.

The listed percentages for serious-event terms are not necessarily additive. In general, a participant may experience more than one type of event, and a single event may be coded under a specific term within a broader safety system. Without the underlying definitions and counting rules, summing term percentages or treating them as separate portions of a whole would not be justified. The record also does not report a treatment attribution assessment for the listed events.

What do the planning illustrations represent?

General planning calculations can illustrate the sample sizes that might be needed under stated assumptions. The registry-related planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. For time-to-event outcomes, the planning approach uses a total number of events; for other outcomes, it uses participants per arm. These are planning illustrations, not the trial's own design, and they do not describe observed power. The actual study is single-group, so an equal-allocation two-arm illustration should not be confused with the design that was conducted. The record does not report a sample-size justification from the trial protocol or explain whether the planning assumptions were used by the investigators.

More generally, a planning calculation is conditional on the assumed effect, variability, significance level, allocation, and analysis method. If those assumptions differ from the eventual data or design, the illustration may not match the study's actual information. It cannot compensate for the lack of a reported primary endpoint analysis. No calculated planning result or effect estimate is reported in this record.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary efficacy aim and secondary biological aims, including biological response in sequential tumor biopsies and peripheral blood mononuclear cells, and a relationship between tumor response and changes in MAP4 gene expression. It does not report which outcomes were formally tested, how many tests were planned, or whether any adjustment for multiple testing was specified. The registry record lists no analysis entries.

In general, multiplicity arises when investigators make several statistical comparisons or test several outcomes. The more questions addressed, the greater the chance that at least one result may appear statistically notable by chance if each is treated as a separate test. Methods such as prespecifying a primary endpoint, controlling an overall error rate, or labeling secondary analyses as exploratory can help readers distinguish confirmatory from exploratory findings. The registry does not report whether any such approach was used.

The primary outcome entry says the study closed prematurely and insufficient data was collected. The record does not report how missing outcome data were defined, how many participants lacked each assessment, whether data were censored, or what methods were used to address missingness. In general, missing-data methods rely on assumptions about why observations are unavailable; different assumptions can lead to different estimates. Without a reported analysis plan and outcome-level accounting, those assumptions cannot be assessed.

The registry also does not report the efficacy analysis population, such as whether analyses were intended to include all enrolled participants, only treated participants, or participants meeting other criteria. The adverse-event group lists 31 at risk, but that safety denominator does not establish the analysis population for the primary endpoint. These omissions prevent assessment of whether analyses followed a prespecified population definition or how incomplete follow-up affected the findings.

11. 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.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infe (38.7%), hemoglobin (3.2%), platelets (3.2%), thrombosis/embolism (3.2%), fatigue (lethargy, malaise, asthenia) (3.2%).

The adverse-event record lists 31 participants at risk in the Sequential Epirubicin/Vinorelbine group. It records 14 participants affected by serious adverse events and 31 affected by other adverse events. These are counts as recorded; they do not by themselves describe how many individual events occurred, how long events lasted, or whether any participant experienced more than one event.

The most frequently listed serious-event term is febrile neutropenia, with 38.7% recorded. Hemoglobin, Platelets, Thrombosis/embolism, Fatigue (lethargy, malaise, asthenia), and Fever in the absence of neutropenia are each listed at 3.2%. The registry does not report the counting denominator or clarify whether these percentages are participant-level or event-level, so they should not be read as a directly comparable set of risks without that context. The serious-event term description for febrile neutropenia is incomplete in the registry record.

In general, safety figures are easier to interpret when the record defines event severity, observation time, attribution, and whether repeated events are counted. Those details are not reported in the registry record. The single-group design also means these safety entries do not provide a comparative estimate against another treatment. The figures describe the entries recorded for this study; they do not, on their own, establish causation.

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

The record documents a phase II trial of sequential epirubicin and vinorelbine in a single group of patients with breast cancer. It records enrollment of 31 and includes adverse-event counts for 31 participants at risk. It also records selected serious-event terms and their percentages. These registry entries describe the study and its reported safety information; they are not a complete account of all outcomes or a comparison of treatment options.

The registry's primary outcome entry says the study closed prematurely and insufficient data was collected. No primary efficacy estimate or analysis is reported. Thus the record cannot answer the stated efficacy question with an estimate of response, survival, or another defined endpoint. The primary measure itself is not operationally specified in the record, so the intended outcome cannot be identified precisely from the title alone.

Because this is a single-group study, the reported data cannot establish how outcomes compare with those under another treatment. In general, an outcome observed after treatment does not by itself show that the treatment caused it; a relevant comparison and a sufficiently defined analysis are needed to assess a treatment effect. The adverse-event counts likewise describe reported events in the group but do not provide a comparative safety estimate or prove that an event was caused by treatment.

The record does not provide details on efficacy analysis populations, missing outcomes, endpoint assessment, or statistical methods. These omissions, together with premature closure and insufficient data, limit the precision and scope of interpretation. The absence of an efficacy result in the registry should not be read as evidence that an effect was absent; it means the record does not report an evaluable result for the stated primary question. The secondary biological aims are listed, but their results are not reported either.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between an intended research question and an answer supported by reported data. The registry states an efficacy aim and a timeframe, but does not define the primary measure in operational terms or report an estimate. It also explicitly notes premature closure and insufficient data collection. Those limitations make it important to separate the study's purpose from its available findings.

The design matters as well. A single-group study can describe outcomes among participants receiving a regimen, but it cannot provide a randomized comparison with a control group. That distinction is especially important when interpreting safety counts or any observed clinical outcomes: descriptive data are not equivalent to a treatment contrast. The registry's lack of masking and absence of a comparator are features of the recorded design, not details that can be repaired by further calculation.

The safety entries also illustrate why denominators, event definitions, and counting rules matter. Counts of participants affected and percentages attached to specific terms answer different questions, and the registry record does not clarify all the relevant units. General statistical interpretation depends on knowing whether figures count people or events and how long participants were observed.

Finally, the record illustrates why missing analysis information matters. Without a defined endpoint, analysis population, or account of missing outcome data, readers cannot assess the assumptions that would support an efficacy estimate. Statistical methods can clarify uncertainty when outcomes are adequately defined and observed; they cannot create an answer where the registry reports insufficient data. Careful reading therefore means recognizing both the information the record contains and the questions it leaves unresolved.

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.

The most direct limitation is stated in the primary outcome entry: the study closed prematurely and insufficient data was collected. The registry does not report an efficacy analysis or estimate, so the intended primary question cannot be answered from the posted outcome information. The reason for termination is not reported in the registry record.

The primary outcome is described broadly as efficacy, with a timeframe of 10 years, but the record does not define the endpoint, unit, or analysis parameter. It is not possible to determine from the registry whether the intended measure was a response classification, time-to-event outcome, or another endpoint. The record also does not report the assessment schedule or criteria.

The single-group design has no concurrent comparator, and allocation is listed as not applicable. The record therefore does not provide a randomized treatment contrast. Masking is listed as none; whether outcome assessors were masked separately is not reported in the registry record. The detailed treatment description is incomplete for stage IV disease, and doses and full administration details are not reported.

For safety, the record gives the number at risk and affected counts, along with selected term percentages, but does not clarify all counting units, follow-up time, recurrent-event handling, or attribution. The complete analysis population, outcome-level missingness, and methods for handling missing data are also not reported. The registry does not describe multiplicity procedures, interim analyses, or a protocol-based sample-size justification. These gaps limit assessment of precision, potential bias, and the scope of the recorded findings.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Epirubicin and Vinorelbine in Treating Patients With Stage II, Stage III, or Stage IV Breast Cancer — NCT00176488.
• Registry record last updated 2017-03-23; status last verified 2017-02.
• Results first posted 2013-11-25.
• 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

The registry describes a phase II, single-group study of sequential epirubicin and vinorelbine for breast cancer. It lists 31 enrolled participants, no masking, and a terminated status. The listed dates are 2003-06 for study start and 2009-10 for primary completion.

The primary outcome concerns efficacy over a stated timeframe of 10 years, but the endpoint is not defined in measurable terms and no efficacy analysis is reported. The registry says the study closed prematurely and insufficient data was collected. Secondary aims concern biological response and MAP4 gene expression; results for these aims are not reported.

The adverse-event group lists 31 participants at risk, 14 affected by serious adverse events, and 31 affected by other adverse events. Selected serious-event terms and percentages are reported, but the counting basis is not fully described. The record does not provide a concurrent comparator, detailed analysis methods, or enough information to evaluate efficacy.