Paclitaxel, Carboplatin, and Panitumumab in Treating Patients With Metastatic Breast Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Paclitaxel, Carboplatin, and Panitumumab in Treating Patients With Metastatic Breast Cancer (NCT01009983), a na phase 2 study. The primary outcome is Antitumor Activity as Assessed by Objective Tumor Response According to RECIST Criteria.

2. Clinical Question

Secondary outcomes named in the record were time to progression, survival, and expression of EGFR and other protein markers. These outcomes address different questions from objective response: they concern disease timing, survival, and biomarker expression. Their definitions and numerical results are not reported in the registry record. The record therefore identifies the clinical questions the trial set out to examine, but does not contain enough outcome information to quantify the observed tumor response or evaluate those secondary outcomes.

3. Trial Design

This was a phase II, single-group clinical trial sponsored by Wake Forest University Health Sciences. The registry lists enrollment of 14 participants. The study began in 2010-03 and had a primary completion date of 2013-07; its status is terminated. The reason for termination is not reported in the registry record.

All participants in the described arm received the combination of paclitaxel, carboplatin, and panitumumab. Paclitaxel and carboplatin were administered intravenously on days 1, 8, and 15, while panitumumab was administered intravenously on days 1 and 15. Laboratory biomarker analysis and immunohistochemistry staining were also listed as procedures. The registry does not report further details about treatment duration, dose adjustments, or treatment discontinuation rules.

The trial was not a randomized comparison: the allocation field is NA, the model is single-group, and there is no listed control arm. Masking is recorded as none. Thus, the design can describe outcomes among participants receiving the combination, but it cannot directly contrast those outcomes with a concurrent group receiving another treatment. In general, a control group can help distinguish differences associated with treatment assignment from differences related to participant mix, disease course, or other factors. Without such a group, interpretation must remain descriptive and cannot isolate the effect of the regimen.

The record lists four outcome measures and no analysis entries. It identifies objective tumor response as the primary outcome, with time to progression, survival, and biomarker expression among the secondary outcomes. Statistical-analysis details, including the planned methods for estimating or comparing outcomes, are not reported in the registry record. The termination status and small enrollment are important context, but neither alone explains why the trial ended or what the unreported results would have been.

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 Antitumor Activity as Assessed by Objective Tumor Response According to RECIST Criteria, measured over every 28 days for a minimum of 84 days.

The primary endpoint is the number of participants with an objective tumor response: a complete or partial response defined by reduction in tumor size according to RECIST criteria. The record states that response was assessed every 28 days for a minimum of 84 days. It identifies the unit as participants, so the intended summary is a count of people meeting the response definition, rather than a continuous measurement of tumor size.

In general, a prespecified response definition gives researchers a consistent rule for classifying whether a participant meets an endpoint. RECIST is used to standardize tumor-response assessment so that response categories are tied to specified changes in tumor measurements rather than an informal impression. A binary endpoint such as response or no response can be summarized by counting participants in each category; interpretation also depends on knowing who was assessed and how missing assessments were handled.

The registry record does not report the observed count of complete or partial responses, the number assessed, or an uncertainty interval. It therefore does not permit a numerical estimate of the response proportion or its precision. The endpoint definition is reported, but the result needed to answer the primary question is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary outcome as a count of participants with an event, where the event is complete or partial tumor response under RECIST criteria. This is a binary outcome at the participant level: each assessed participant either meets the response definition or does not. In general, binary outcomes are often summarized by the number meeting the definition and the number assessed. A proportion can then express the count in relation to the assessed group, but no response count or assessed denominator is reported in the registry record, so no such summary can be stated for this trial.

Response was to be assessed every 28 days for a minimum of 84 days. Repeated assessment can establish whether a participant meets the endpoint during the observation schedule, but interpretation depends on the protocol’s response rules and the analysis definition. For example, investigators may need to specify how to handle a response that is not confirmed, changes across assessments, or is followed by withdrawal. Those operational rules are not reported in the registry record. The timing statement alone does not establish how the endpoint was ultimately classified.

In general, an estimate is more informative when accompanied by an uncertainty interval. A confidence interval describes a range of values compatible with the data under the analysis assumptions; it communicates precision rather than guaranteeing that the true value lies in the interval. For a binary endpoint, the interval’s width depends on the number assessed and the number meeting the response definition. Neither a response estimate nor a confidence interval is reported in the registry record. No inferential test or model for the primary endpoint is described.

The design also shapes which statistical methods can answer the clinical question. In a randomized comparison, an analysis can estimate a difference between assigned groups, subject to the design and analysis assumptions. Here, there is one treatment group and no randomized comparator. A descriptive estimate, if reported, would characterize response among participants in that group; it would not estimate a randomized treatment contrast. General statistical principle: statistical adjustment cannot fully recreate a missing concurrent control group, particularly when relevant differences between groups cannot be measured.

The registry lists four outcome measures and no analysis entries. The named secondary outcomes are time to progression, survival, and expression of EGFR and other protein markers. Time-to-event outcomes generally concern the time until a defined event, and their analysis can depend on censoring, follow-up length, and the event definition. In general, censoring occurs when the event time is not observed during a participant’s available follow-up; methods for such outcomes account for the incomplete timing information under stated assumptions. The record does not provide endpoint definitions, event counts, follow-up summaries, censoring rules, estimates, or analyses for time to progression or survival. Biomarker outcome methods and results are likewise not reported in the registry record.

The safety figures are counts of participants affected, alongside percentages for listed serious adverse-event terms. In general, safety summaries should be interpreted with attention to the event definition, observation period, severity grading, and whether participants can contribute more than one event or term. A participant may experience multiple kinds of events, so percentages for separate terms should not automatically be added to obtain a total. The record does not report the detailed observation period, event-collection procedures, grading criteria, or participant-level overlap among listed terms.

General statistical concept: over-dispersion matters when observed variability is greater than the variability assumed by a statistical model, and may make uncertainty estimates too optimistic if ignored. It is not possible to assess over-dispersion from the registry information because no model, repeated event counts, or analysis results are reported. Likewise, the record does not report an interim-analysis plan, a sample-size justification, a primary statistical test, or an adjustment method for multiple outcomes. None should be inferred from the phase designation or enrollment alone.

The record includes planning-illustration assumptions elsewhere in the statistical material, but those are not the trial’s own design justification. Any such illustration is a planning exercise, not observed evidence and not observed power. The registry record does not report the trial’s original sample-size rationale. In general, power is a design property calculated under assumptions about an effect, variability, and analysis; it cannot be reconstructed as a measure of what the completed study observed. With the primary result and design rationale unreported, no trial-specific power interpretation is available.

Finally, a complete analysis requires a defined analysis population and a transparent account of missing data. General approaches differ depending on whether participants are analyzed according to enrollment, treatment received, or endpoint availability. The registry record does not report which population was used, how missing or unevaluable assessments were handled, or whether any participants were excluded from the endpoint analysis. Those details are important because response estimates can depend on who is included and how incomplete follow-up is treated.

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("Arm 1")
n      <- c(13)
events <- c(6)
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  Arm 1  13  6  46.2  19.2  74.9  23.2  70.9  23.0  71.1

11. Multiplicity, Missing Data and Analysis Populations

The registry lists four outcome measures, including objective tumor response and secondary outcomes of time to progression, survival, and expression of EGFR and other protein markers. Examining several outcomes can create a multiplicity issue: in general, the more statistical questions asked, the greater the chance of at least one apparently notable result occurring by chance when each is tested separately. A prespecified hierarchy or adjustment can address some multiplicity concerns, but the registry record does not report a testing hierarchy, adjustment, or other multiplicity strategy. It also lists no analysis entries, so the approach used for these outcomes is not described.

Missing-data handling is also not reported in the registry record. The response endpoint depends on assessments scheduled every 28 days for a minimum of 84 days, but the record does not say how many participants had evaluable assessments, whether any assessments were missing, or how unevaluable cases were classified. General statistical principle: excluding participants with missing outcomes can bias an estimate if the likelihood of missingness is related to response or other participant characteristics. Different assumptions about missing outcomes can therefore lead to different interpretations.

The analysis population is not reported in the registry record. It is not possible to determine whether the analysis included all enrolled participants, only participants who began treatment, or only those with an evaluable response assessment. Nor are participant flow, withdrawals, or reasons for missing outcomes reported. The absence of these details limits evaluation of both the primary endpoint and the secondary outcomes. No particular missing-data method or multiplicity adjustment should be assumed.

12. 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: low anc (64.3%), lymphopenia (35.7%), low wbc (28.6%), low hemoglobin (14.3%), diarrhea (14.3%).

The registry reports that 9 of 14 participants were affected in the serious-adverse-event category and 14 of 14 in the other-adverse-event category. These are participant counts, not counts of individual event episodes. The record lists the following top serious adverse-event terms and percentages: Low ANC, 64.3%; Lymphopenia, 35.7%; Low WBC, 28.6%; Low Hemoglobin, 14.3%; Diarrhea, 14.3%; and hyperglycemia, 14.3%.

In general, percentages attached to event terms describe how often those terms were recorded in the study’s safety data; they should not be summed, because a participant may have experienced more than one term. The registry record does not report the detailed event definitions, severity grading, collection period, or whether the listed terms overlap at the participant level. The number of deaths is not reported in the registry record.

These figures describe reported events among participants in a single group. Without a control group and further follow-up and ascertainment details, they do not establish how the event experience compares with another treatment or what caused an event. They are a safety description, not a comparative estimate.

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

The trial’s record establishes that a phase II, single-group study evaluated paclitaxel, carboplatin, and panitumumab in metastatic triple-negative breast cancer. It names objective response according to RECIST criteria as the primary outcome and specifies the assessment schedule. It also records serious and other adverse-event counts and names several secondary outcomes.

What the record does not establish is the observed primary response result. The number of participants with complete or partial response, the number assessed, and any estimate of uncertainty are not reported in the registry record. The secondary outcome results and their analyses are also not reported. Therefore, the record cannot quantify the degree of tumor response or the precision of an estimate, and it cannot support a conclusion about time to progression, survival, or biomarker expression.

Even a reported response count from this single group would be descriptive rather than a randomized comparison. The study has no listed control arm, so the record cannot establish how outcomes would differ under another regimen. General statistical principle: outcomes observed in a treatment group can reflect treatment, participant characteristics, disease course, and other influences; a single-group design does not separate these explanations.

The reported safety counts show that serious and other adverse events were recorded among participants, with several serious-event terms listed. They do not provide a comparative safety estimate or establish causation. The termination status is recorded, but its reason is not reported in the registry record. These gaps should be kept distinct from evidence of no effect or no event: an unreported result is not the same as a result of zero.

14. Why This Trial Matters Statistically

This trial is statistically instructive because it makes the distinction between a defined endpoint and a reported result clear. The registry describes objective tumor response using RECIST criteria and gives an assessment schedule, but it does not report the response count or analysis. A well-defined outcome is necessary for interpretation, yet a definition alone cannot show what participants experienced or how precisely an outcome was estimated.

The single-group design also illustrates the limits of descriptive evidence. Outcomes in one treatment group may characterize what was observed among those participants, but without a randomized comparator they do not provide a direct estimate of a difference between treatment strategies. This distinction matters when readers encounter a study objective framed around how well a combination works: the design and the results available in the registry determine which statistical questions can actually be answered.

Small enrollment is relevant to precision, but it should not be treated as a result. In general, estimates based on fewer observations tend to carry more uncertainty, although the actual uncertainty depends on the outcome data and analysis. Because the response result and interval are not reported, precision cannot be assessed for this trial. Enrollment alone also does not reveal whether all participants were evaluated.

Finally, the record separates efficacy questions from safety reporting and names multiple secondary outcomes without providing their analyses. This underscores why transparent reporting of participant flow, analysis populations, missing-data handling, and multiplicity methods matters. Without those details, readers cannot fully judge the reliability or scope of conclusions. The trial’s record is useful for understanding the design and the questions posed, while its posted outcome information remains insufficient to answer the primary statistical question.

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.

The principal limitation for interpreting this trial is that the registry record does not report the primary endpoint result. It provides the response definition and assessment schedule, but not the number of participants with a complete or partial response, the number assessed, or an uncertainty estimate. The named secondary outcomes likewise lack reported results and analyses.

The study was single-group, with no randomized control arm. Any available outcome would therefore describe the treated group rather than provide a direct comparison with another treatment. The record lists enrollment of 14, but does not report participant characteristics, the analysis population, participant flow, treatment exposure, or reasons for missing or unevaluable assessments. These omissions limit assessment of how representative the analyzed participants were and how missing outcomes might affect interpretation.

The trial is marked terminated, but the reason is not reported in the registry record. The record does not describe the sample-size justification, interim analyses, statistical plan, multiplicity handling, or missing-data methods. It also does not report detailed safety ascertainment, event grading, or the death count. These reporting gaps do not explain the termination or imply any particular outcome; they restrict what can be concluded from the registry information.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Paclitaxel, Carboplatin, and Panitumumab in Treating Patients With Metastatic Breast Cancer — NCT01009983.
• Registry record last updated 2018-07-06; status last verified 2018-07.
• Results first posted 2014-06-18.
• 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

This is a phase II, single-group trial sponsored by Wake Forest University Health Sciences, with enrollment of 14 participants and status recorded as terminated. The study began in 2010-03, with primary completion in 2013-07. Participants received paclitaxel and carboplatin intravenously on days 1, 8, and 15, and panitumumab intravenously on days 1 and 15.

The primary outcome was the number of participants with complete or partial tumor response according to RECIST criteria, assessed every 28 days for a minimum of 84 days. The registry record does not report the response result or analysis entries. It names time to progression, survival, and expression of EGFR and other protein markers as secondary outcomes, without reporting their results. Safety information includes 9 of 14 participants affected in the serious-adverse-event category and 14 of 14 in the other-adverse-event category; the death count is not reported in the registry record.