Pre-Surgical Intervention for Targeted Therapies for Breast Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pre-Surgical Intervention for Targeted Therapies for Breast Cancer (NCT01004744), a na na study. The primary outcome is Number of Subjects That Completed Oral Anastrozole 1mg Daily for Two Weeks in the Interval Between Diagnostic Breast Biopsy and Definitive Breast Surgery.

2. Clinical Question

3. Trial Design

This was a completed, single-group study sponsored by Columbia University. The registry lists enrollment as 10 and identifies the intervention as oral anastrozole. The intervention description specifies 1mg daily for two weeks between diagnostic breast biopsy and definitive breast surgery. The study started in 2007-07, and its primary completion date is 2009-08.

The allocation field is listed as NA, the model is single group, and masking is NONE. There is therefore no randomized allocation or masking described, and no control group for a direct treatment comparison. The design is suited to describing whether participants completed the presurgical regimen, but it does not separate treatment-related changes from changes that might occur over time or for other reasons.

The registry summary describes a feasibility study in women with newly diagnosed early invasive or non-invasive breast cancer. It says the short-term intervention was intended to evaluate biologic effects on the PI3K/AKT signaling pathway. The registered primary outcome is narrower: the number of participants who completed the two-week course. The record does not report a primary-outcome value or a pathway result.

The registry lists one outcome measure and zero analyses. It does not report a statistical analysis plan, the analysis population, eligibility criteria in detail, or the method used to assess pathway changes. These omissions limit what can be inferred from the registration about how the study was conducted and how results were intended to be interpreted. The completed status indicates the registry’s status classification; it does not supply the missing outcome result.

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

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 Subjects That Completed Oral Anastrozole 1mg Daily for Two Weeks in the Interval Between Diagnostic Breast Biopsy and Definitive Breast Surgery, measured over Two weeks.

The primary endpoint is the number of participants who completed oral anastrozole 1mg daily for two weeks during the interval between diagnostic breast biopsy and definitive breast surgery. It is measured in participants and refers to finishing the prescribed length of the study intervention. The timeframe is two weeks.

A completion endpoint is relevant to feasibility because it addresses whether participants can carry out the planned short-term regimen in the presurgical setting. In general, feasibility outcomes describe practical aspects of delivering or completing a study intervention; they are not automatically measures of clinical benefit or of a biological mechanism.

The registry record does not report the observed number who completed treatment. It also does not give a completion definition beyond finishing the study’s stated treatment length, explain how discontinuation or partial adherence was handled, or report an analysis of reasons for non-completion. Enrollment is listed as 10, but the record does not state whether that number is the denominator used for the primary outcome. Without the outcome value and its denominator, the registered endpoint cannot be quantitatively interpreted from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design allow?

In general, a single-group study observes participants who receive the intervention without a concurrent comparison group. Such a design can describe the treatment course and outcomes in the enrolled group. It does not directly estimate what would have happened to those participants under a different treatment or without the intervention. Differences over time may reflect the intervention, the underlying condition, other care, measurement variation, or other influences. Without a comparison group, those explanations cannot be separated by a treatment contrast.

For this study, the registry lists a single-group model, allocation as NA, and masking as NONE. These design features matter when interpreting the primary completion outcome and any intended biologic assessment. They do not make a descriptive observation invalid; they define what kind of inference the study can support. The record does not report a comparative analysis.

How should the completion endpoint be understood?

In general, a count records how many participants meet a defined event criterion, while a proportion relates that count to a specified denominator. The count and denominator answer related but different questions. A count alone does not indicate the fraction of an eligible or enrolled group that completed, unless the relevant denominator is known and appropriate. This distinction is especially important when participants can have missing outcome information or leave a study before the endpoint is assessed.

The registered endpoint is the number who completed oral anastrozole for the stated two-week period. Enrollment is listed as 10, but the record does not report the completion count, the analysis denominator, or how missing information was handled. No completion rate can therefore be read from the registry record. The primary endpoint is described as a number of participants, while the record’s outcome classification also identifies it as binary and as participants with an event counted. Those labels do not supply an observed result or further specify the completion assessment.

What can a statistical analysis add?

In general, statistical analysis can quantify uncertainty around an observed result and can assess how compatible the data are with a specified model or comparison. Those interpretations depend on the analysis method, the data included, and the assumptions made. A confidence interval, for example, gives a range of estimates compatible with a procedure’s stated coverage under its assumptions; it is not a guarantee that the true value lies in that range. A p-value, when used, describes the compatibility of data with a statistical null model and is not the probability that a hypothesis is true.

The registry record lists zero analyses and does not report an analysis method, confidence interval, p-value, or model for the primary outcome. It is therefore not possible to describe a sponsor analysis of that endpoint or to assess its precision statistically. The record does not report an interim-analysis plan or sample-size justification. Their absence from the registry record should not be taken as evidence about whether such work occurred outside the record.

How do planning illustrations differ from study results?

In general, a sample-size calculation is a planning exercise: it estimates the amount of information a future study might need under stated assumptions. It is not an observed result and does not establish the probability that a completed study found an effect. The registry’s planning illustration assumptions specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of an observed effect. These are planning assumptions, not the design of this single-group trial.

Where an illustration is based on a fraction of an observed effect, it asks how planning requirements change if the target effect is smaller than the reference effect. Such an illustration depends on the effect estimate and statistical assumptions used. It does not show that the target effect is real, nor does it provide evidence about treatment performance in the trial. In general, an effect estimate from a small study can be imprecise, so calculations based on it should be read as conditional planning examples, not as a replacement for a prospective design rationale.

Power is a property of a planned statistical test under assumptions. It is not a measure of whether a treatment works, and a planning calculation is not observed power. No trial-specific analysis or observed primary endpoint is reported in the registry record, so the planning assumptions cannot resolve the missing outcome information. The record does not report the study’s own sample-size justification.

Why does the event definition matter?

In general, an event count is interpretable only when the event definition, observation period, and population at risk are clear. For completion, it matters whether the criterion requires taking every planned dose, completing the scheduled interval, or satisfying another prespecified rule. For adverse events, it matters which events are included and whether the count refers to participants or event occurrences. A participant can experience more than one event, so participant counts and event counts are not interchangeable.

The registry describes the completion criterion as finishing oral anastrozole for the length of the study and gives a two-week timeframe. It does not describe additional adherence rules. For adverse events, it reports participants at risk and participants affected in serious and other categories, but does not report the event terms in the other category. These details constrain interpretation and should not be filled in by assumption.

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("Presurgical Oral Anastrozole")
n      <- c(10)
events <- c(10)
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  Presurgical Oral Anastrozole  10  10  100.0  69.2  100.0  72.2  100.0  71.5  99.8

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and zero analyses. It names treatment completion as the primary outcome, but does not report a statistical analysis plan or the observed primary outcome value. The record does not state whether additional outcomes were prespecified outside the registry entry, how any multiple outcomes would have been prioritized, or whether multiplicity adjustments were planned. Those matters are not reported in the registry record.

In general, multiplicity arises when several statistical hypotheses or outcomes are examined. Testing many questions can increase the chance of at least one apparently unusual result under a null model. A prespecified analysis plan can make clear which outcome is primary, which analyses are exploratory, and whether any adjustment is used. The registry entry does not provide enough information to assess these issues for this study. No adjustment method should be inferred.

The record does not report an analysis population, participant-level outcome availability, or a missing-data method. It does not say whether completion was assessed for all enrolled participants or whether any participants were excluded from an analysis. Enrollment is listed as 10, but the endpoint denominator is not reported. In general, missing outcomes can affect both the estimated event count and how representative the analyzed group is; the direction of any effect cannot be determined without information about the missingness and analysis approach.

The record also does not report an interim-analysis plan or stopping rules. These details are distinct from whether the study is marked completed. Their absence in the registry record limits assessment of the prespecification and completeness of statistical reporting.

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 non-serious adverse event terms

The registry lists 10 participants at risk in the serious adverse-event category and 0 affected. It also lists 10 at risk in the other adverse-event category and 10 affected. These entries are participant counts as recorded; they should not be read as a comparison between treatment groups because the study has one group and no control.

The record does not identify the terms included in the other adverse-event category, explain how events were collected, or report their severity, timing, duration, or relationship to anastrozole. The serious-event entry is limited to the reported count and at-risk figure. The death count is not reported in the registry record.

In general, a zero count in a study group does not establish that an event is impossible or that there is no risk. It describes the events reported during the study’s observation and under the record’s reporting definitions. The record does not provide enough detail to evaluate the completeness of event ascertainment or make a broader safety comparison. These figures should be treated as descriptive entries, not as a comprehensive safety profile.

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

The registry record supports a limited description of the study: it was a completed, single-group investigation of oral anastrozole given before breast surgery, and its registered primary question concerned completion of a two-week treatment course. The study’s stated purpose also included examining biologic effects on the PI3K/AKT signaling pathway. The intervention and purpose are described, but the primary completion result and pathway result are not reported.

This distinction matters because a feasibility endpoint and a biological endpoint address different questions. A count of participants completing treatment would describe the practical completion outcome in the study group. A pathway measure would require its own definition, measurement method, and result. The registry record does not report the values needed to interpret either outcome. The completed status alone does not establish that participants completed the regimen or that a particular biological change occurred.

The single-group design does not provide a control comparison. Even with an observed outcome, the study would not by itself show how the same participants would have fared under another intervention or no intervention. It also cannot isolate a treatment effect from other influences on changes observed between biopsy and surgery.

The adverse-event entries are limited descriptive figures: 0 serious events among 10 at risk, and 10 affected in the other adverse-event category among 10 at risk. The record does not supply the event terms or enough context to interpret the latter count in detail. These figures should not be converted into broader claims about treatment safety.

Accordingly, the registry information outlines a feasibility-oriented study question and some design details, but leaves the central outcome result and analysis methods unavailable in the record. It does not support a claim for or against clinical benefit, nor does it establish a pathway effect. The warranted interpretation is bounded by what the registry actually reports.

14. Why This Trial Matters Statistically

This study illustrates why the registered endpoint should be read alongside the study’s stated purpose. The summary describes interest in the biologic effects of a short presurgical course on the PI3K/AKT signaling pathway, while the primary registered outcome measures the number of participants who completed treatment. Completion can inform feasibility, but it is not itself a measure of molecular response or clinical benefit. Keeping these questions distinct avoids treating a practical outcome as evidence for a biological mechanism.

The study also illustrates the limits of a single-group design. A single group can provide descriptive information about an intervention’s delivery and outcomes among those enrolled. Without a comparator, the design cannot provide a controlled estimate of how outcomes differ under another approach. This is particularly important for short-term observations made during a period in which participants are also receiving care around biopsy and surgery.

Finally, the record shows why results and analysis details matter for interpretation. It lists enrollment as 10 and reports adverse-event counts, but it does not report the primary completion value, a pathway result, or statistical analyses. A completed status cannot replace those results. The missing endpoint value prevents assessment of the central feasibility question from the registry record, while the absence of analysis details prevents an evaluation of precision and analytic choices.

Statistically, the study is a reminder to distinguish design intent, measured outcomes, and reported evidence. A study may be designed around an important biological question while its registered primary outcome captures a different feature of conduct. Transparent outcome definitions, denominators, analysis methods, and results are necessary for readers to judge what an investigation establishes and what remains unresolved.

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 registry record does not report the observed value for the primary completion outcome. This is the main limitation for interpreting the registered feasibility question. Although enrollment is listed as 10, the record does not state the endpoint denominator, the number who completed treatment, or how participants with incomplete or unavailable outcome information were handled.

The design is single group, with allocation listed as NA and masking as NONE. There is no concurrent control group for distinguishing treatment-related changes from other changes over the presurgical interval. The record therefore does not support a comparative treatment estimate.

The summary describes evaluation of biologic effects on the PI3K/AKT signaling pathway, but no pathway outcome value, measurement method, or analysis is reported. The registered primary outcome concerns completion rather than a reported molecular measure. The record does not explain how the completion criterion was operationalized beyond the stated treatment duration, or report adherence details.

The registry lists zero analyses and does not report the statistical plan, analysis population, missing-data approach, sample-size justification, or interim-analysis plan. These omissions limit assessment of prespecification and statistical precision. The record also provides limited safety detail: the terms for the other adverse events, their timing, severity, and relationship to treatment are not reported. The death count is not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Pre-Surgical Intervention for Targeted Therapies for Breast Cancer — NCT01004744.
• Registry record last updated 2021-01-11; status last verified 2021-01.
• Results first posted 2021-01-11.
• 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

NCT01004744 is a completed, single-group study sponsored by Columbia University. The registry lists enrollment as 10, masking as NONE, and allocation as NA. The study evaluated oral anastrozole, described as 1mg daily for two weeks between diagnostic breast biopsy and definitive breast surgery. The start date is 2007-07, and the primary completion date is 2009-08.

The registered primary outcome is the number of participants who completed the treatment course. The study summary also describes an aim to evaluate biologic effects on the PI3K/AKT signaling pathway. The primary outcome value and pathway results are not reported in the registry record. The record lists one outcome measure and zero analyses.

For adverse events, the record lists 0 serious events among 10 at risk and 10 affected in the other adverse-event category among 10 at risk. Event terms for the latter category and the death count are not reported in the registry record.