This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Neoadjuvant Hormonal Therapy Combined With Chemoimmunotherapy (NCT02345772), a na phase 1, phase 2 study. The primary outcome is Pathological Complete Remission Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02345772 |
| Phase | Phase 1, Phase 2 |
| Condition | HER2-positive Breast Cancer, ER-positive Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 4 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Western Regional Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was patients with ER-positive and HER2-positive breast cancer who were to receive treatment before surgery. The registry does not report demographic or clinical characteristics of enrolled participants, and the primary-outcome population is described as “Data not collected.” The record therefore does not establish which patients enrolled or whether any had evaluable surgical pathology.
Intervention
The study planned neoadjuvant hormonal therapy with fulvestrant alongside chemoimmunotherapy with docetaxel, trastuzumab and pertuzumab. The protocol description says fulvestrant 500 mg was scheduled on specified days of the first cycle and then on day 1 of each 28-day cycle for up to 5 cycles before surgery. It also specifies docetaxel 75 mg/m2 every 3 weeks for four cycles. The registry’s arm description is truncated, so the complete treatment schedule is not reported in the registry record.
Comparator
There was no separate comparator group. The study is listed as a single-group design, and allocation is recorded as NA. It therefore does not provide a randomized or concurrent control comparison. Any assessment is limited to the planned outcome in the treated group, and causal contrasts against another treatment cannot be estimated from this design.
Primary question
The primary question was the pathological complete remission rate at surgery in patients receiving the combined neoadjuvant regimen. The record defines pathological complete remission as no invasive neoplastic cells in the primary breast tumor, while allowing remaining in-situ lesions. The timeframe is listed as one year. No rate, numerator, denominator, or analysis is reported in the registry record, so the question remains unanswered by the posted results.
3. Trial Design
This was a phase 1 and phase 2, single-group study listed as terminated. Its title describes neoadjuvant hormonal therapy combined with chemoimmunotherapy in patients with HER2-positive and ER-positive breast cancer. Enrollment is recorded as 4. The registry lists Western Regional Medical Center as the sponsor and gives a start date of 2014-07 and a primary completion date of 2015-11. The study status is terminated.
The planned treatment combined fulvestrant with docetaxel, trastuzumab and pertuzumab before surgery. The arm description specifies fulvestrant 500 mg by intramuscular administration on days 1 and 15 of the first cycle, then on day 1 of every 28-day cycle for up to 5 cycles before surgery. It also specifies docetaxel 75 mg/m2 every 3 weeks for four cycles. The remainder of the regimen description is truncated, so its complete dosing and scheduling details are not reported in the registry record.
Masking is listed as none. Allocation is recorded as NA, and the study model is single group. These features mean the record describes neither blinded assignment nor a randomized comparison between treatment groups. The study’s primary outcome was the pathological complete remission rate at the time of surgery, with a listed timeframe of one year. Partial pathological response rate and quantitative texture analysis are listed as secondary outcomes.
The registry states that the study was terminated because the principal investigator was no longer employed at CTCA and did not have rights to the trial information. It further states that, after much effort, results could not be retained. The primary outcome population is described as “Data not collected.” Thus, although planned enrollment and study features are recorded, outcome data and analyses are not available for evaluating the intended clinical question.
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
| Stage | Treatment Protocol |
|---|---|
| STARTED | 4 |
| COMPLETED | 0 |
| NOT COMPLETED | 4 |
Analysis population for the primary outcome, as defined in the registry: Data not collected. The study has been terminated due to the PI no longer being employed at CTCA and not having rights to the trial information."After much effort, results were not able to be retained.
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.
Population: Baseline data were not collected. No study data was collected
| Characteristic | Unit | Treatment Protocol |
|---|---|---|
| Participants analysed | 0 | |
| Age, Categorical: <=18 years | — | |
| Age, Categorical: Between 18 and 65 years | — | |
| Age, Categorical: >=65 years | — | |
| Sex: Female, Male: Female | — | |
| Sex: Female, Male: Male | — | |
| Region of Enrollment: United States | participants | — |
6. Primary Endpoint
The registry lists the primary outcome as Pathological Complete Remission Rate, measured over one year.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the pathological complete remission rate at surgery. The registry defines pathological complete remission as the absence of invasive neoplastic cells in the primary tumor in the breast; remaining in-situ lesions are allowed. The timeframe is listed as one year. This endpoint is a proportion: its interpretation ordinarily depends on identifying how many participants meet the pathology definition and how many participants were assessed.
As a general statistical concept, a pathology endpoint can describe tumor findings at a specified surgical assessment. A clearly defined pathology criterion is important because it determines which observations count as events in the endpoint. The distinction between invasive disease and in-situ lesions is part of the definition in this record and should not be replaced by a broader or narrower definition when interpreting the outcome.
The registry record does not report the number meeting the definition, the number assessed, an estimated remission rate, or an interval expressing uncertainty. It also says that primary-outcome data were not collected. Therefore, the endpoint is described as a planned measure, not as an observed result. No conclusion about the rate or its precision can be drawn from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a study intended to estimate pathological complete remission after a combined neoadjuvant regimen in ER-positive and HER2-positive breast cancer. It records enrollment as 4, but the primary-outcome population is explicitly described as “Data not collected.” No remission count, denominator of assessed participants, estimated rate, confidence interval, test statistic, or p-value is reported in the registry record. The posted results therefore do not provide an estimate of the primary endpoint.
Enrollment is not interchangeable with an endpoint denominator. To estimate a rate, the outcome status and assessment status of participants must be known. The record does not say whether any enrolled participants received the planned treatment, proceeded to surgery, or had evaluable pathology. Those details are not reported in the registry record. It would not be appropriate to infer outcome values from enrollment alone.
The study is listed as single group with allocation recorded as NA. It does not provide a concurrent control group or a randomized contrast. Even if a treated-group outcome estimate were available, this design would describe outcomes in the study group rather than establish a comparative treatment effect. In the current record, however, the absence of collected primary-outcome data means even that descriptive estimate cannot be made.
The registry reports no analyses, and the study is marked terminated. It attributes termination to the principal investigator no longer being employed at CTCA and lacking rights to the trial information, and says that results could not be retained after much effort. This account supplies a reason for the unavailable results, but it does not substitute for participant-level outcomes or an analysis. The status and explanation should be read as record information, not as evidence about the regimen’s effects.
There are also secondary outcomes listed: partial pathological response rate and quantitative texture analysis. Their listing indicates planned outcomes, not reported findings. No outcome values or analyses for them are reported in the registry record. Likewise, the adverse-event entry with zero values cannot be interpreted as evidence that no events occurred: the record does not provide a usable account of participants assessed for harms, and no adverse-event analyses are listed.
In general, an estimate and its precision answer different questions. An estimate describes the observed outcome in the analyzed group; an interval would show the range of values compatible with the data under the method used. Neither is available for the primary endpoint. There is consequently no basis in the posted results to characterize the remission rate as high, low, precise, or imprecise. The available information supports a description of the planned question and the reason outcome results were not retained, but not an efficacy or safety conclusion.
9. Statistical Methods Explained
The statistical methods that were actually used to analyze the primary outcome are not reported in the registry record. The registry lists zero analyses. It gives no outcome estimate, statistical test, confidence interval, model, adjustment strategy, or analysis population. Statistical concepts can help explain what would ordinarily be needed to interpret a remission-rate study, but they must not be mistaken for methods documented for this trial.
What an endpoint rate requires
As a general statistical concept, a rate for a binary endpoint is calculated from the number of participants who meet the endpoint definition and the number whose endpoint status is assessed. For this study, the endpoint definition concerns the absence of invasive neoplastic cells in the primary breast tumor at surgery, with in-situ lesions allowed. The registry does not give either the number meeting that definition or the number assessed. It also states that primary-outcome data were not collected. No trial-specific rate can therefore be reconstructed from the registry record.
The listed enrollment of 4 does not supply the missing endpoint denominator. Enrollment indicates the recorded study size, not how many participants completed treatment, underwent surgery, had pathology assessed, or were included in an analysis. Those statuses are not reported in the registry record. Treating all enrolled participants as assessed would require an assumption that the record does not support.
How uncertainty would usually be expressed
General explanation: a confidence interval communicates uncertainty around an estimate under a specified statistical method. For an outcome rate, its width depends on the observed data and the interval method. An interval is useful because a single point estimate does not show how much statistical uncertainty remains. Here, no rate or interval is reported, and the required outcome data are absent. There is therefore no trial-specific precision measure to interpret.
The registry also does not report a hypothesis test or p-value for the primary endpoint. In general, a p-value relates observed data to a specified null hypothesis under a statistical model; it does not by itself measure clinical importance or precision. Without a reported analysis and its underlying outcome data, there is no p-value to explain and no null hypothesis comparison to assess.
Why the design matters
The study is recorded as single group, with allocation listed as NA and masking listed as none. In general, a single-group design can describe outcomes observed among participants who receive a regimen, but it does not create a concurrent control contrast. It cannot, by itself, separate a treatment effect from other influences on observed outcomes. This limitation is distinct from missing outcome data: even a complete descriptive outcome result would not amount to a randomized comparison.
As no comparator group is recorded, comparative effect measures cannot be interpreted for this study. The record does not report a comparison with usual care, another regimen, or an untreated group. No adjusted comparison or causal analysis is described. These are absences in the registry record, not evidence that an unreported analysis was or was not conducted elsewhere.
Models and distributional assumptions
No statistical model for the primary or secondary outcomes is reported in the registry record. General explanation: statistical models encode assumptions about how observations relate to outcomes and, in some settings, how variability behaves. For a binary pathology endpoint, a rate-based method may be suitable, but the choice of estimator and interval procedure depends on the available data and prespecified analysis plan. Neither that plan nor its implementation is reported.
Over-dispersion is a general concern when observed variability is greater than a model assumes. It can affect standard errors and the reliability of uncertainty estimates in models that rely on restrictive variance assumptions. The registry does not report a model or data that would permit assessment of over-dispersion in this study. It would be speculative to attribute a particular model or diagnostic procedure to the investigators.
Multiplicity and planned outcomes
The registry lists the primary outcome and two secondary outcomes, but it does not report a multiplicity strategy. General explanation: when several outcomes or hypotheses are tested, the chance of at least one false-positive finding can increase if each is assessed without an appropriate plan. Approaches to multiplicity depend on the scientific questions and analysis plan. Since no analyses or results are reported, there are no trial-specific tests to evaluate for multiplicity, and no adjustment method can be attributed to this study.
The sample-size assumptions recorded for planning illustrations are not evidence of the trial’s own sample-size justification or an analysis conducted by the investigators. The registry does not report the study’s design rationale or a statistical analysis plan. In particular, no claim about a prespecified power calculation, interim analysis, or stopping rule is supported by the record.
Analysis populations and missing outcomes
The registry does not identify an intention-to-treat, per-protocol, or safety analysis population. It provides no participant flow or accounting of exclusions, missing pathology assessments, or withdrawals. General explanation: analysis populations determine which participants contribute to an estimate and can materially affect its interpretation. Missing outcomes can also introduce uncertainty or bias, especially when the reasons for missingness are related to outcomes. The record’s statement that data were not collected does not describe the reasons or pattern of missingness at the participant level.
Because the primary endpoint was not collected, there is no basis for an observed-data analysis, a missing-data adjustment, or a sensitivity analysis. The record does not report imputation or other methods for handling missing results. The appropriate interpretation is not that a particular statistical method failed, but that the registry does not provide outcome data or an analysis with which to evaluate the endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists pathological complete remission rate as the primary outcome, with partial pathological response rate and quantitative texture analysis as secondary outcomes. It does not report how these outcomes were prioritized in statistical testing, whether any formal hypotheses were tested, or whether multiplicity was addressed. This information is not reported in the registry record.
General explanation: multiplicity arises when multiple outcomes, comparisons, or analyses are considered. Without a plan, repeated testing can make isolated positive findings harder to interpret. A prespecified strategy can define which outcomes are confirmatory and how uncertainty or testing is handled. No such strategy can be attributed to this study from the available registry information. There are no analyses or outcome results to assess for multiplicity.
The record says the primary-outcome population was “Data not collected.” It does not report the number of participants with endpoint assessments, reasons for missing outcome information, or participant flow from enrollment through surgery. Nor does it describe an analysis population such as all enrolled participants or those with evaluable pathology. These items are not reported in the registry record.
General explanation: missing outcomes can affect both the precision and potential representativeness of an estimate. The consequences depend on how much is missing and why it is missing. Methods such as imputation or sensitivity analyses require information and assumptions that are not described. Since the endpoint data were not collected, the record offers no trial-specific missing-data analysis to evaluate.
The adverse-event record includes a treatment group with zero values for participants at risk and affected, and no serious-event terms. Those entries do not establish that no harms occurred. The record does not supply a participant-level safety population or an analysis, and the study’s outcome information is described as not retained. An absence of reported events must not be treated as a verified absence of events.
11. What the Result Does, and Does Not, Mean
The registry record establishes that a study was planned to assess pathological complete remission after neoadjuvant hormonal therapy combined with chemoimmunotherapy in ER-positive and HER2-positive breast cancer. It also records enrollment as 4, a single-group design, and terminated status. It states that primary-outcome data were not collected and that results could not be retained after much effort. These registry entries describe the availability and limitations of the record, not the outcome of treatment.
The posted results do not establish a pathological complete remission rate. There is no reported count of participants meeting the endpoint, no assessed denominator, and no estimate with which to describe statistical precision. As a result, the information does not show whether the observed endpoint was common or uncommon in the study group. It also cannot support a claim about how outcomes would compare with another regimen or with no treatment.
General explanation: a small enrolled group can leave substantial uncertainty in an estimated rate, but enrollment alone does not reveal the endpoint result or its precision. In this record, the more fundamental issue is that primary-outcome data are explicitly described as not collected. No statistical estimate can replace the missing endpoint observations.
The study’s single-group design is an additional interpretive constraint. In general, observations from one group do not provide a randomized control contrast, so they cannot isolate the effect of the regimen from other factors. Here, the absence of outcome data prevents even a descriptive estimate of the primary endpoint. The design limitation and the missing results are separate reasons that comparative conclusions are unavailable.
The listed secondary outcomes likewise do not have reported results. The adverse-event entries do not permit a safety conclusion because the record does not document a usable safety analysis or participant assessment. The registry’s explanation for termination and loss of results is relevant context, but it cannot stand in for clinical outcome data.
Accordingly, the record can be used to understand the intended question, planned treatment, and study status. It cannot be used to conclude that the treatment did or did not produce remission, to quantify any effect, or to characterize the regimen’s safety. Those conclusions are not reported in the registry record.
12. Why This Trial Matters Statistically
This trial matters statistically because its record illustrates the distinction between a study question and an answer supported by data. The primary endpoint is defined, the intended population and regimen are described, and enrollment is recorded. But the registry says that primary-outcome data were not collected and that results could not be retained. Without endpoint observations, a rate cannot be estimated, and statistical uncertainty cannot be quantified.
The case also shows why enrollment and analysis populations must not be conflated. Enrollment is recorded as 4, but that number does not identify how many participants had evaluable pathology or contributed to an endpoint analysis. The registry does not report participant flow, surgical assessment, or endpoint denominators. These distinctions matter for any interpretation of an outcome rate.
The single-group design is also important. A study can describe the outcomes of a treated group, but a concurrent randomized comparator serves a different purpose: it supports a direct comparison under the study design. This record lists no comparator and no randomized allocation. Even if a treated-group rate were available, it would not alone establish a comparative effect. Since the rate is also unavailable, neither descriptive nor comparative outcome claims are supported.
Finally, the record distinguishes planned endpoints from results. Partial pathological response rate and quantitative texture analysis are listed as secondary outcomes, but no analyses are listed. Naming an outcome does not establish that it was measured or analyzed. Clear reporting of the result, denominator, analysis population, and uncertainty would be needed to make the endpoint statistically interpretable; those details are not reported in the registry record.
13. 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 the absence of primary-outcome data. The registry explicitly describes the primary-outcome population as “Data not collected,” and says results could not be retained. There is no remission count, denominator, estimate, confidence interval, or analysis. The intended primary question therefore cannot be answered from the posted results.
Recorded enrollment is 4, but the registry does not show whether those participants received the planned regimen, completed treatment, proceeded to surgery, or had pathology assessed. It does not describe withdrawals, exclusions, or missing assessments. The size and composition of the endpoint analysis population are not reported in the registry record.
The single-group design provides no concurrent control comparison. Allocation is NA and masking is none. Even complete outcome data would not create a randomized estimate contrasting the regimen with another treatment. No comparator or adjusted analysis is reported.
The treatment description is incomplete: the arm description ends mid-sentence, so the full protocol schedule is not reported in the registry record. The registry also does not report a statistical analysis plan, sample-size justification, prespecified analysis population, handling of missing outcomes, multiplicity strategy, interim analyses, or detailed participant characteristics. These omissions limit assessment of how the study was intended to be conducted and analyzed.
Finally, the adverse-event entries cannot establish an absence of harms. The record does not provide participant-level safety assessments or an analysis, and results were not retained. These limitations concern the information available in the registry record; they do not establish what may have been documented elsewhere.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Neoadjuvant Hormonal Therapy Combined With Chemoimmunotherapy — NCT02345772.
- Registry record last updated 2018-02-20; status last verified 2018-01.
- Results first posted 2017-10-25.
- 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.
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17. Record Summary
NCT02345772 is a phase 1 and phase 2 study titled “Neoadjuvant Hormonal Therapy Combined With Chemoimmunotherapy.” It was designed as a single-group study of neoadjuvant fulvestrant with docetaxel, trastuzumab and pertuzumab in ER-positive and HER2-positive breast cancer. The registry records enrollment as 4, status as terminated, and Western Regional Medical Center as sponsor.
The primary endpoint was pathological complete remission at surgery, defined as absence of invasive neoplastic cells in the primary breast tumor while allowing remaining in-situ lesions. Partial pathological response rate and quantitative texture analysis are listed as secondary outcomes.
The registry says primary-outcome data were not collected and that results could not be retained. It reports no analyses. Consequently, no primary endpoint estimate, precision measure, comparative result, or interpretable safety conclusion is available in the registry record.