Women's MoonShot: Neoadjuvant Treatment With PaCT for Patients With Locally Advanced TNBC: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Women's MoonShot: Neoadjuvant Treatment With PaCT for Patients With Locally Advanced TNBC (NCT02593175), a na phase 2 study. The primary outcome is Number of Participants With Residual Disease.

2. Clinical Question

3. Trial Design

This was a phase II, single-group treatment study sponsored by M.D. Anderson Cancer Center. The registry lists 43 participants as enrolled. Allocation is recorded as NA, masking as none, and the model as single group. These design features mean that participants were not assigned between randomized study arms and that the treatment was not masked.

The regimen combined panitumumab with the chemotherapy drugs carboplatin and paclitaxel before surgery. The treatment description specifies panitumumab and paclitaxel intravenously on days 1, 8, and 15, and carboplatin intravenously on day 1. Treatment repeated every 21 days for up to 4 cycles in the absence of disease progression or unacceptable toxicity.

The registry describes the intended population as patients with newly diagnosed triple-negative breast cancer that was localized to the breast, possibly with nearby lymph-node involvement. The official title describes tumors predicted to be insensitive to standard neoadjuvant chemotherapy. The brief summary says the treatment study was linked to protocol NCI-2015-00191, which used a baseline biopsy to determine neoadjuvant therapy matched to a TNBC subtype.

The study started on 2016-11-03, had a primary completion date of 2025-04-03, and is listed as completed. A detailed protocol, statistical analysis plan, eligibility criteria, and rationale for the enrollment target are not reported in the registry record. The record does not report a randomized control group, so the design can describe outcomes among people receiving this regimen but cannot provide a like-for-like treatment comparison.

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: All patients were evaluable for data analysis

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 Number of Participants With Residual Disease, measured over At the time of surgery.

The primary endpoint is the number of participants with residual disease at the time of surgery. It is a count outcome: the registry identifies the measure as a participant count and describes the outcome type as participants with an event. In this study, the relevant event is residual disease at surgery. The registry states that all patients were evaluable for data analysis, but it does not give the number with residual disease or the corresponding number without it.

Residual disease is used as an outcome because the registry description says it can help predict disease recurrence and survival across breast cancer subtypes. It reflects disease status assessed at the surgical time point after neoadjuvant treatment. That makes it a clinically relevant measure of what remained at surgery, but it is not itself a direct report of later recurrence or survival in this record.

For a count endpoint, the observed count would describe how many participants met the outcome definition within the analyzed group. A count alone is most interpretable alongside the number assessed and a clearly specified outcome definition. The registry record does not report the observed primary count, a denominator for the outcome, or further details about how residual disease was classified. Therefore, it does not allow a numerical estimate of the primary endpoint or its precision.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

In a single-group study, outcomes are recorded among participants receiving the study regimen. The direct descriptive target is what happened in that treated group, subject to the outcome definition and the completeness of assessment. Such a design does not create a concurrent randomized group receiving a different treatment. As a general statistical principle, randomization helps balance measured and unmeasured prognostic factors on average; without a randomized comparator, differences from an external expectation cannot be cleanly attributed to treatment.

Here, the registry records the model as single group, allocation as NA, and masking as none. It identifies 43 participants as enrolled. Those details establish the broad design, but do not reveal the recruitment process, baseline characteristics, or whether all enrolled participants contributed to every analysis. The primary outcome description says all patients were evaluable for data analysis; details about the analysis population and any exclusions are not reported in the registry record.

How should a count endpoint be read?

The primary measure is a count of participants with residual disease at surgery. For a count to be interpreted fully, readers need the observed event count, the number assessed, and the rule used to decide whether the event occurred. The registry names the outcome and its time point, but does not report its observed value or classification details. It therefore does not permit a numerical estimate of the endpoint.

In general, an interval estimate expresses uncertainty around an estimated quantity. A narrow interval indicates greater precision under the statistical model used, while a wide interval indicates less precision. No confidence interval or other precision estimate for the primary outcome is reported in the registry record. The precision of the study's primary result therefore cannot be assessed from the available registry information.

What can safety counts tell readers?

The safety record lists participants at risk and participants affected in serious and other adverse-event categories. These are descriptive counts for the treatment group. A participant count is not necessarily the same as an event count: a participant may experience more than one event, but whether that occurred is not detailed in the registry record. The listed serious adverse-event terms and their percentages add detail, but do not provide timing, severity grading, duration, or individual-level attribution.

In general, adverse events observed during a clinical trial do not by themselves establish that treatment caused them. A comparison group, event details, and clinical assessment can help contextualize potential treatment-related risk. Since this trial has no comparator group in the registry record, the posted counts should be read as descriptions of events among treated participants rather than comparative estimates.

What is not available about the statistical plan?

The registry record does not report the planned statistical methods for the primary endpoint, a hypothesis test, a confidence interval method, or a sample-size justification. It also does not report interim analyses or a stopping rule. These details should not be inferred from the phase label, enrollment count, or the fact that an endpoint was designated primary.

In general, a statistical analysis plan specifies how outcomes and analysis populations will be defined, how missing observations will be handled, and how estimates and uncertainty will be calculated. Prespecification can reduce the risk of selectively emphasizing favorable analyses. Without the plan or analysis results, readers cannot determine whether the analysis followed a prespecified method or how sensitive the result might be to analytic choices.

Multiplicity refers to the increased chance of a false-positive finding when many hypotheses or outcomes are tested. The record identifies one primary outcome measure, but does not report a multiplicity strategy or the broader set of analyses, if any. This does not establish that multiple testing occurred; it means the handling of multiplicity is not reported in the registry record.

The registry contains no analysis entries for the primary outcome. General statistical concepts can explain what an estimate or test would mean, but they cannot replace the missing observed result. No efficacy estimate, test statistic, p-value, or interval should be inferred from the enrollment or safety information.

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("Panitumumab Plus Carboplatin and Paclitaxel")
n      <- c(24)
events <- c(24)
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  Panitumumab Plus Carboplatin and Paclitaxel  24  24  100.0  85.8  100.0  86.2  100.0  86.3  99.9

11. Multiplicity, Missing Data and Analysis Populations

The registry identifies one primary outcome measure: the number of participants with residual disease at surgery. It does not report a multiplicity adjustment or explain how multiple outcomes, subgroups, or statistical tests were handled. The absence of a reported strategy does not show that multiple testing occurred, and it does not establish that no additional analyses were conducted. The relevant analysis plan is not reported in the registry record.

The primary outcome result itself is not reported, and the registry contains no analysis entries. The record says all patients were evaluable for data analysis, but does not provide the analyzed count for the primary outcome, describe missing observations, or state how any missing outcome data were handled. It also does not report whether participants who discontinued treatment or did not undergo surgery were included in the endpoint analysis.

In general, missing outcome data can affect both the estimate and its precision, especially when the likelihood of missingness is related to prognosis or treatment experience. A transparent report would specify the number assessed, reasons for unavailable outcomes, and any assumptions or sensitivity analyses. Those details are not reported in the registry record, so the impact of missingness cannot be evaluated.

The analysis population is described only as all patients evaluable for data analysis. Further details about its definition, eligibility for analysis, or relationship to the enrolled group are not reported. No sample-size justification, interim-analysis plan, or primary-endpoint statistical method is reported either. These omissions limit assessment of prespecification and robustness; they do not establish that the study lacked such procedures outside the registry record.

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 3% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: cystitis (2.3%), vertigo (2.3%).

The registry reports safety counts for the group receiving panitumumab plus carboplatin and paclitaxel. For serious adverse events, 43 participants were at risk and 2 were affected. For other adverse events, 43 participants were at risk and 40 were affected. The number of deaths is recorded as 0. The serious event terms listed are cystitis and vertigo, each reported as 2.3%.

These are descriptive observations in the treated group, not comparisons against another treatment. The registry does not report whether each event was considered related to treatment, when it occurred, how long it lasted, or whether participants experienced more than one event. The record also does not provide details needed to assess event severity beyond the serious-versus-other categories. The listed term percentages should be read as registry-reported figures; no additional event rate or comparison can be inferred from them.

In general, adverse-event counts summarize what was recorded under the study's reporting definitions. They do not on their own establish causality, and a single-group record cannot quantify how these counts compare with those under an alternative regimen. The available information supports a description of the recorded safety figures, not a comparative safety conclusion.

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

The record describes a phase II study of a three-drug neoadjuvant regimen in patients with triple-negative breast cancer. It states that 43 participants enrolled and identifies residual disease at surgery as the primary outcome. It also gives selected adverse-event counts and reports 0 deaths. These details are useful for understanding the study's purpose, general design, treatment schedule, and the safety information entered in the registry.

The record does not report the primary residual-disease count or any analysis result for that endpoint. As a result, the primary outcome cannot be quantified, and no statement about its precision can be made. The absence of a posted result does not mean that no result exists; the reason it is absent is not reported in the registry record.

Even if a primary outcome count were available, this single-group design would describe outcomes in the treated participants rather than establish how outcomes compare with another treatment. Without a concurrent comparator, other influences on outcome cannot be disentangled through a randomized comparison. The design therefore does not support a causal treatment comparison on its own.

The safety counts likewise describe recorded events among participants in the study group. They do not establish that the regimen caused an event or how its safety compares with another option. No medical decision should be inferred from these registry entries alone.

The distinction is between a study's question and the evidence available in its record. The study was designed to examine residual disease following neoadjuvant treatment, but the posted registry information gives no numerical answer to that primary question. The sound interpretation is therefore limited: the design and some safety observations are documented, while the primary result and comparative evidence are not reported.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of how design determines the scope of an answer. A single-group phase II study can document treatment delivery and describe outcomes among the enrolled participants. It cannot provide a randomized estimate of the difference between this regimen and another treatment because no comparator group is recorded.

The primary endpoint is a count of participants with residual disease at surgery, a clearly timed outcome that the registry says can help predict recurrence and survival across breast cancer subtypes. Yet a named endpoint is not the same as a reported result. Without the count, denominator, and an uncertainty estimate, readers cannot evaluate the magnitude or precision of the primary finding.

The record also illustrates why the completeness of reporting matters. It identifies enrollment and gives selected safety figures, but offers no primary outcome analysis, no stated statistical method, and no description of missing-data handling. Those gaps prevent readers from checking how the endpoint was analyzed or how robust the result might be. They should be treated as limits of the registry record, not as proof that analyses or procedures were absent elsewhere.

Finally, the distinction between descriptive data and comparative evidence is important in clinical research. Counts observed in one treatment group can inform a description of that group, but without a comparison they cannot isolate a treatment effect. For this record, the most defensible statistical reading is precise about what is documented and equally precise about what remains unavailable.

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 most consequential limitation for interpreting the primary question is that the registry does not report the residual-disease count or an analysis result. It provides no estimate of the primary outcome and no measure of its precision. The stated endpoint time is surgery, but additional details of the outcome definition and assessment are not reported in the registry record.

The single-group design has no like-for-like control arm. Any outcomes among treated participants would therefore be descriptive and would not, by themselves, establish what would have happened under another treatment. Masking is recorded as none, and the record does not describe procedures that might reduce the influence of knowledge of treatment on outcome assessment.

The registry lists 43 participants as enrolled and says all patients were evaluable for data analysis, but does not give the primary endpoint denominator or explain whether any participants lacked an assessment. Reasons for missing outcomes, exclusions, and the definition of the analysis population are not reported. These omissions prevent assessment of potential selection or missing-data effects.

Further limitations of the registry information include the absence of a reported statistical analysis plan, sample-size justification, multiplicity approach, and interim-analysis details. These items are not reported in the registry record; their absence from the record does not establish that they were not specified elsewhere. Safety reporting is also limited to the posted categories, counts, and selected event terms, without detailed timing, severity grading, or comparative data.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Women's MoonShot: Neoadjuvant Treatment With PaCT for Patients With Locally Advanced TNBC — NCT02593175.
• Registry record last updated 2025-11-06; status last verified 2025-10.
• Results first posted 2025-11-06.
• 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

NCT02593175 is a completed phase II, single-group study sponsored by M.D. Anderson Cancer Center. It enrolled 43 participants and evaluated neoadjuvant panitumumab, carboplatin and paclitaxel for localized triple-negative breast cancer. Treatment was described for up to 4 cycles before surgery.

The primary outcome was the number of participants with residual disease at surgery. The registry record does not report that outcome's observed count or an analysis result. It records serious adverse events in 2 of 43 participants at risk, other adverse events in 40 of 43 at risk, and 0 deaths. Cystitis and vertigo are listed as serious event terms, each at 2.3%.

Because the design has no comparator group and the primary result is not reported, the registry supports a limited description of study design and safety counts, not a numerical primary-outcome conclusion or a comparative treatment estimate.