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 Taxotere and Adriamycin/Cytoxan (AC) Validation in Breast Cancer Patients (NCT00206518), a randomized phase 2 study. The primary outcome is Pathological Tumor Response to Neoadjuvant Chemotherapy (Taxotere and AC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00206518 |
| Phase | Phase 2 |
| Condition | Breast Cancer |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 167 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mothaffar Rimawi |
| Results posted | Yes |
2. Clinical Question
Population
People with breast cancer enrolled in a randomized multicenter trial. The registry records an enrollment of 167 participants. Further eligibility details and characteristics of the enrolled population are not reported in the registry record.
Intervention
Arm A received single-agent Taxotere, also called docetaxel, before surgery. The arm description specifies 100 mg/m2 every 3 weeks for 4 cycles. The registry lists Adriamycin/Cytoxan and doxorubicin among the interventions, and describes AC as the other neoadjuvant regimen.
Comparator
Arm B received AC before surgery: doxorubicin 60 mg/m2 and cyclophosphamide 600 mg/m2 every 3 weeks for 4 cycles. The registry says the Adriamycin dose was calculated using BSA = 2.0 m2 for patients whose BSA was greater than 2.0 m2.
Primary question
The trial asked whether pathological tumor response differed between the two neoadjuvant chemotherapy approaches, and its stated purpose also concerned whether biomarker information from earlier studies could indicate whether Taxotere and/or AC could cause tumors to become smaller. The registry does not report a biomarker-specific comparison or arm-specific pathological response results.
This is a randomized, parallel-arm comparison without masking. Its primary outcome is recorded as pathological tumor response, graded using Chevalier categories. That outcome has several categories, ranging from complete pathological response through partial response grades to no or few modifications of tumor appearance. The registry does not reduce those categories to a single comparison, and does not provide counts for each category by treatment arm. Consequently, the central statistical question is identifiable from the design and endpoint description, but the posted record does not establish the direction, size, or precision of any difference in response between the arms.
3. Trial Design
The registry classifies NCT00206518 as a phase 2, randomized, parallel trial with no masking. The recorded enrollment is 167, the condition is breast cancer, and the study status is completed. The recorded start is 2004-09 and primary completion is 2016-10. The sponsor is listed as Mothaffar Rimawi.
Participants were assigned to one of two neoadjuvant chemotherapy approaches. Arm A is described as single-agent Taxotere/docetaxel, 100 mg/m2 every 3 weeks for 4 cycles before surgery. Arm B is described as AC, doxorubicin 60 mg/m2 and cyclophosphamide 600 mg/m2 every 3 weeks for 4 cycles before surgery. The registry notes a dose calculation rule for Adriamycin when BSA was greater than 2.0 m2. Both arm descriptions refer to surgery following neoadjuvant treatment, if operable. Additional treatment details beyond the recorded descriptions are not reported in the registry record.
Random allocation supports a planned comparison between the two assigned approaches, while the parallel design means the arms are distinct groups rather than a crossover sequence. With no masking recorded, the registry does not indicate that treatment assignment was concealed from participants or investigators after allocation. The methods used to generate the random sequence, conceal allocation, manage departures from assigned treatment, or define the analysis population are not reported in the registry record.
The registry lists Disease Relapse and Overall Survival as secondary outcomes. It records three outcome measures but no statistical analyses. The primary outcome has multiple response categories and is not reduced to a single comparison in the posted results. A protocol-level statistical analysis plan, planned sample-size justification, interim analysis procedures, and details of follow-up are not reported in the registry record.
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 | A: Taxotere/Docetaxel | B: AC Adriamycin/Cytoxan |
|---|---|---|
| STARTED | 83 | 84 |
| COMPLETED | 73 | 78 |
| NOT COMPLETED | 10 | 6 |
Reasons for not completing this period:
| Reason | A: Taxotere/Docetaxel | B: AC Adriamycin/Cytoxan |
|---|---|---|
| Lack of Efficacy | 6 | 4 |
| Adverse Event | 2 | 1 |
| Physician Decision | 1 | 0 |
| Protocol Violation | 1 | 1 |
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.
| Characteristic | Unit | A: Taxotere/Docetaxel | B: AC Adriamycin/Cytoxan | Total |
|---|---|---|---|---|
| Participants analysed | 83 | 84 | 167 | |
| Age, Customized: <=50 years | participants | 56 | 49 | 105 |
| Age, Customized: >50 years | participants | 27 | 35 | 62 |
| Sex: Female, Male: Female | Participants | 83 | 84 | 167 |
| Sex: Female, Male: Male | Participants | 0 | 0 | 0 |
| Race/Ethnicity, Customized: White | participants | 21 | 27 | 48 |
| Race/Ethnicity, Customized: Hispanic | participants | 35 | 34 | 69 |
| Race/Ethnicity, Customized: Black | participants | 24 | 18 | 42 |
| Race/Ethnicity, Customized: Asian | participants | 3 | 5 | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Pathological Tumor Response to Neoadjuvant Chemotherapy (Taxotere and AC), measured over 10 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is pathological tumor response to neoadjuvant chemotherapy, assessed with Chevalier's system. It classifies response as category 1, disappearance of all tumor on macroscopic or microscopic assessment in both the breast and lymph nodes; category 2, in situ carcinoma in the breast with no invasive tumor in the breast and no tumor in lymph nodes; categories 3A through 3D, invasive cancer with stromal alteration such as sclerosis or fibrosis, graded by the stated subjective therapeutic-effect ranges; or category 4, no or few modifications of tumor appearance. The description associates categories 1 and 2 with pCR, category 3 with pPR, and category 4 with pNR.
Pathological response is used to describe tumor findings after treatment and surgery, providing a clinically interpretable assessment of the tumor's observed pathological state. It is not the same as a time-to-event outcome such as relapse or survival. The registry lists the endpoint timeframe as 10 years, but does not explain how that timeframe relates to the pathological assessment or specify a separate assessment schedule.
The categories are not interchangeable: they distinguish complete response, degrees of partial response, and little or no modification. Combining them into a binary outcome would require a defined cutoff and would discard distinctions. The registry does not report an analysis using such a cutoff, nor does it report the number of participants in any response category by arm. Therefore, the endpoint definition is available, but the posted record does not permit interpretation of a comparative response estimate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted record describes a randomized comparison and defines a multicategory pathological response endpoint, but it does not report the response counts or comparative analysis needed to quantify the trial's primary result. The endpoint is not summarized as a single treatment contrast. No arm-specific response distribution, effect estimate, confidence interval, or p-value is reported in the registry record. Without those data, the direction and magnitude of any difference in pathological response cannot be determined from the posted results.
The study's stated purpose concerns whether biomarker information obtained from earlier studies could tell whether Taxotere and/or AC can cause tumors to become smaller. However, the posted record does not identify a biomarker-specific endpoint, report biomarker strata, or present analyses relating biomarker information to response. The title's use of validation does not by itself establish that any biomarker was validated; the relevant results and validation criteria are not reported in the registry record.
Randomization describes the intended basis for comparing treatment groups, but it does not substitute for outcome data. Enrollment was 167, and the safety summaries list 83 participants at risk in the Taxotere/docetaxel arm and 84 in the AC arm. These safety denominators do not provide the primary-outcome denominators or response counts. The registry does not say whether all randomized participants were included in the endpoint analysis, whether any participants were excluded, or how missing pathological assessments were handled.
The record lists Disease Relapse and Overall Survival as secondary outcomes, but it does not give their results. It also records no statistical analyses. Thus, no conclusion about those outcomes, their precision, or consistency with pathological response can be drawn from this record.
In general, an effect estimate would describe the observed difference or other contrast between groups, while a confidence interval would describe the uncertainty around that estimate under the analysis assumptions. Neither is reported for the primary endpoint. A p-value, even if reported, would address compatibility with a specified statistical null model rather than establish the size or clinical importance of a difference. Here there is no reported primary-outcome estimate or inferential result to interpret. The available information supports describing the design, endpoint, and safety counts, not judging comparative efficacy.
9. Statistical Methods Explained
Randomized allocation is intended to make treatment groups comparable, on average, with respect to factors that could influence outcomes. This is a general property of randomization, not a guarantee that the groups were identical in a particular trial. The registry classifies this study as randomized but does not report how the allocation sequence was generated, whether allocation was concealed, or how assigned treatment was analyzed.
What does the endpoint's categorical structure mean?
The primary outcome uses ordered descriptions of pathological response: categories 1 and 2 are identified as pCR, categories 3A through 3D describe degrees of pPR, and category 4 is pNR. Such categories retain distinctions that a yes/no outcome would remove. An analysis could compare the full distribution, or could define a threshold and compare participants on either side, but those choices answer different questions. The registry does not report a selected statistical model, threshold, or category-specific comparison. No method should be assumed from the endpoint description alone.
In general, methods for categorical outcomes may compare category frequencies or model the probability of categories, depending on the scientific question and assumptions. An ordinal model, for example, would use the ordering of categories and require assumptions about how treatment relates to that ordering. A nominal approach would treat categories as distinct without relying on an ordering. These are general possibilities, not methods documented for this trial. The posted record reports no analysis results, so there is no basis to say which approach was used.
How should estimates and uncertainty be read?
In general, an effect estimate gives a numerical summary of a comparison between groups. A confidence interval conveys uncertainty about that estimate under the model and analysis assumptions; it does not show the range of outcomes for individual participants. A p-value summarizes how unusual the data would be under a specified null hypothesis and model. Those statistics need the endpoint data and a defined analysis. Neither an estimate nor its uncertainty is reported for the primary endpoint in the registry record.
A statistical result also depends on who is included in the analysis and how outcomes are defined. An intention-to-treat analysis generally analyzes participants according to randomized assignment, while a per-protocol analysis generally restricts or classifies participants according to adherence to specified treatment requirements. These are general descriptions only. The analysis population and treatment of deviations from assigned therapy are not reported in the registry record.
What do the planning assumptions describe?
The registry's planning illustration is described as based on the observed effect, with a two-sided significance level of 0.05, equal allocation between arms, and a normal approximation. The assumed effect to detect is a stated fraction of the observed effect. These are planning assumptions, not proof that the trial was designed using them. The trial's own sample-size justification is not reported in the registry record. Because the primary outcome has several categories and is not reduced to one comparison, the posted record does not provide a single primary effect for interpreting a planning illustration. Any sample-size display based on an observed effect is a planning illustration and must not be read as observed power.
In general, power calculations describe the probability of detecting a specified effect under a set of assumptions if that effect is present. They do not measure the probability that a study result is true, and post hoc power based on an observed estimate is not a substitute for an effect estimate and confidence interval. The registry does not report an independently specified effect target, variance assumptions, or a design rationale that would permit reconstruction of the original planning process.
The two-sided significance level describes a testing convention that allows departures in either direction under the alternative. A normal approximation is a mathematical approximation used in some tests or planning calculations; its suitability depends on the outcome structure and data conditions. Equal allocation is a planning assumption, whereas actual analyzed group sizes depend on enrollment and the analysis set. These general concepts do not establish the trial's final analysis method. No interim analysis plan, adjustment strategy, or statistical analysis plan is reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary pathological response outcome and the secondary outcomes Disease Relapse and Overall Survival. The primary response itself has multiple categories, including pCR, graded pPR, and pNR descriptions. When a study tests multiple outcomes, categories, time points, or subgroup contrasts, the chance of at least one false-positive finding can increase if each test is interpreted separately. General strategies include specifying a primary contrast in advance, adjusting for multiple testing, or treating secondary analyses as exploratory. The registry record does not report which strategy, if any, was planned or used.
The posted results do not provide category-specific response counts, a single primary comparison, or analyses for the secondary outcomes. No multiplicity adjustment, hierarchy of hypotheses, subgroup plan, or interim analysis procedure is reported in the registry record. It is therefore not possible to determine how any multiple comparisons were handled.
Missing outcome data can affect interpretation when participants lack an assessable response or follow-up. General approaches include describing missingness, defining analysis populations, and using methods appropriate to the assumed missing-data process. The registry does not report the amount or pattern of missing pathological assessments, missing relapse or survival follow-up, or any imputation or sensitivity analysis.
The registry also does not specify whether the primary analysis was intention-to-treat, per-protocol, or another analysis population. These are material details because exclusions or outcome availability can change who contributes to an estimate. Without these details and the underlying outcome results, the primary and secondary comparisons cannot be reconstructed from the record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| A: Taxotere/Docetaxel | 12 | 83 | 14.5% |
| B: AC Adriamycin/Cytoxan | 5 | 84 | 6.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| A: Taxotere/Docetaxel | 13 of 83 |
| B: AC Adriamycin/Cytoxan | 3 of 84 |
Most frequent serious adverse event terms
| Event term | Organ system | A: Taxotere/Docetaxel | B: AC Adriamycin/Cytoxan |
|---|---|---|---|
| NEUTROPENIA | Blood and lymphatic system disorders | 8 of 83 | 3 of 84 |
| INFECTION | Infections and infestations | 4 of 83 | 1 of 84 |
| FEVER | General disorders | 2 of 83 | 1 of 84 |
| ABDOMINAL PAIN | Gastrointestinal disorders | 0 of 83 | 1 of 84 |
| ALLERGIC/REACTION | Immune system disorders | 1 of 83 | 0 of 84 |
| GASTRITIS | Gastrointestinal disorders | 0 of 83 | 1 of 84 |
| RENAL FAILURE | Renal and urinary disorders | 0 of 83 | 1 of 84 |
Most frequent non-serious adverse event terms
| Event term | Organ system | A: Taxotere/Docetaxel | B: AC Adriamycin/Cytoxan |
|---|---|---|---|
| NEUTROPENIA | Blood and lymphatic system disorders | 13 of 83 | 3 of 84 |
Serious events with the highest arm-level rate: neutropenia (9.6%), infection (4.8%), fever (2.4%), allergic/reaction (1.2%), abdominal pain (1.2%).
The safety summary records serious adverse events in 12 of 83 participants at risk in the Taxotere/docetaxel arm and 5 of 84 participants at risk in the AC Adriamycin/Cytoxan arm. Other adverse events are recorded for 13 of 83 and 3 of 84 participants, respectively. These are the counts and denominators listed in the registry; no adjusted comparison or uncertainty estimate is reported. The record does not define the event-collection period or provide additional detail on how events were classified.
The registry lists neutropenia at 9.6%, infection at 4.8%, fever at 2.4%, allergic/reaction at 1.2%, abdominal pain at 1.2%, and gastritis at 1.2% among the top serious adverse-event terms. It does not provide arm-specific counts for these terms in the posted information. Those percentages should not be attributed to an individual arm or treated as a complete account of all events without further information.
In general, adverse-event counts describe observed events in the recorded population; they do not alone establish whether a treatment caused an event or how groups compare after accounting for uncertainty. The registry does not report deaths in either arm, and a blank death field should not be interpreted as proof that none occurred.
12. What the Result Does, and Does Not, Mean
The record establishes that a randomized phase 2 comparison was conducted between neoadjuvant docetaxel and neoadjuvant AC in people with breast cancer. It also establishes how the primary pathological response endpoint was categorized and lists counts for serious and other adverse events. Those details can inform an understanding of the design and what outcomes were intended to be assessed.
The record does not establish which regimen produced more pathological responses, whether the response distributions differed, or how precisely any difference was estimated. It contains no arm-specific primary response results, effect estimate, confidence interval, or p-value. The multicategory endpoint cannot be interpreted as a single treatment contrast without data and a defined analysis. Nor does the registry report the biomarker analysis suggested by the study's stated purpose.
The safety counts are descriptive. They do not by themselves establish causality, comparative risk, or statistical significance. The record does not state the event definitions, ascertainment details, or duration of safety follow-up. Listed serious-event terms lack arm-specific counts in the posted summary.
The registry lists relapse and overall survival as secondary outcomes but reports no results for either. It therefore does not support claims about long-term outcomes or consistency between pathological response and later outcomes. The completion status indicates that the study is marked completed in the registry, but completion alone does not provide evidence about the results.
In general, a trial's design and outcome definitions describe the question it set out to address; conclusions about comparative effects require the outcome data and analysis. The evidence visible in this record is insufficient to determine whether one treatment approach performed better on the primary endpoint or to assess the precision of a comparison. This distinction separates what the registry documents about trial conduct from what it does not report about treatment outcomes.
13. Why This Trial Matters Statistically
This trial illustrates an important statistical distinction between defining an endpoint and reporting an analyzable result. The registry gives a detailed categorical description of pathological response, but the posted results do not include the distribution of participants across those categories by arm. Without those counts or an analysis, a reader cannot estimate a comparative effect or its uncertainty.
The endpoint also highlights the consequences of analytic choices. Chevalier categories distinguish complete response, graded partial response, and little or no modification. A comparison of the full category distribution would preserve more information than a binary grouping, while a binary analysis would require a threshold and answer a narrower question. The registry does not identify a chosen contrast. This makes the outcome definition informative but insufficient to reconstruct the intended statistical test.
The study's stated interest in biomarker information raises a further design and analysis question: whether biomarker status predicts response, or whether it changes the relative response across treatment groups. Those questions require defined biomarker measures and corresponding analyses. Such results and criteria are not reported in the registry record, so the title's reference to validation cannot be evaluated statistically from the posted information.
Finally, the record separates trial completion and enrollment from evidence of an effect. Enrollment and randomized design describe the study, while response estimates and uncertainty are needed to assess the primary comparison. The absence of reported analyses makes the record a useful example of why outcome definitions, analysis populations, multiplicity plans, missing-data handling, and complete results all matter for interpreting a clinical trial.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The primary limitation for interpreting this specific trial is that the registry does not report arm-specific pathological response counts or a comparative analysis. The response endpoint has several categories and is not reduced to one comparison. As a result, no response effect estimate or measure of precision can be described from the posted record. The registry also does not report biomarker-specific findings despite the stated purpose concerning biomarker information from earlier studies.
The registry records 167 participants overall and provides safety denominators by arm, but does not state the number included in each primary-outcome analysis. Eligibility criteria, baseline characteristics, allocation methods, adherence, treatment discontinuations, and reasons for missing assessments are not reported in the registry record. Without those details, readers cannot assess the analyzed population or compare its characteristics with the enrolled population.
The listed timeframe for pathological response is 10 years, but the registry does not explain the timing of pathological assessment or reconcile that timeframe with the endpoint description. The record also does not give results for the listed secondary outcomes of Disease Relapse and Overall Survival. Statistical methods, multiplicity handling, interim analyses, and the original sample-size justification are not reported.
Safety summaries provide counts and selected serious-event terms, but the posted information does not specify event definitions, follow-up duration, arm-specific counts for the listed terms, or a comparative safety analysis. The registry's completed status does not resolve these reporting gaps. These limitations concern what can be interpreted from the registry record; they do not establish whether additional results exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Taxotere and Adriamycin/Cytoxan (AC) Validation in Breast Cancer Patients — NCT00206518.
- Registry record last updated 2020-07-28; status last verified 2020-07.
- Results first posted 2017-06-06.
- 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.
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18. Record Summary
NCT00206518 is a completed phase 2 randomized parallel trial in breast cancer, with no masking recorded and enrollment of 167. It compared neoadjuvant single-agent Taxotere/docetaxel with neoadjuvant AC. The primary outcome was pathological tumor response classified using Chevalier categories, with a recorded timeframe of 10 years.
The registry lists Disease Relapse and Overall Survival as secondary outcomes. It records safety counts for serious and other adverse events and selected serious-event terms. It does not report arm-specific pathological response results, secondary-outcome results, or statistical analyses. Biomarker-specific results, analysis populations, missing-data handling, multiplicity methods, interim analyses, and the trial's sample-size justification are not reported in the registry record. The posted information therefore describes the trial design, endpoint, and selected safety data but does not support a quantitative interpretation of the primary treatment comparison.