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 Sunitinib Malate, Paclitaxel, Doxorubicin Hydrochloride, and Cyclophosphamide Before Surgery in Treating Patients With Stage IIB-IIIC Breast Cancer (NCT00513695), a non-randomized phase 2 study. The primary outcome is Microscopic Pathologic CR (pCR) Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00513695 |
| Phase | Phase 2 |
| Condition | Inflammatory Breast Cancer, Male Breast Cancer, Stage II Breast Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 68 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Washington |
| Results posted | Yes |
2. Clinical Question
Population
Participants had locally advanced or inflammatory breast cancer, described in the registry title as stage IIB-IIIC disease. The recorded conditions include inflammatory breast cancer, male breast cancer, and stage II and stage III breast cancer categories. Enrollment was 68. The registry record does not report the number who ultimately underwent surgery or were evaluable for the primary endpoint.
Intervention
The study evaluated neoadjuvant treatment with sunitinib malate and chemotherapy before surgery. The regimen included weekly paclitaxel followed by doxorubicin and daily oral cyclophosphamide with G-CSF, as described in the official title. The intervention list also includes filgrastim, surgery, laboratory biomarker analysis, and flow cytometry. The arm description in the registry record is incomplete, so additional details of the treatment schedule are not reported in the registry record.
Comparator
There was no concurrent comparison group. The study is recorded as non-randomized and single-group, with no masking. Its results therefore describe outcomes among participants receiving the study regimen; they do not provide a randomized estimate of how that regimen compares with another treatment.
Primary question
The primary endpoint was the microscopic pathologic complete response rate at surgery. It was defined as no evidence of microscopic invasive tumor at the primary tumor site in the surgical specimen. The question was how often this endpoint occurred among evaluable participants. The registry record does not report the observed rate or the number of evaluable participants.
The study was registered as NCT00513695 and sponsored by the University of Washington. It is a phase II evaluation of a treatment sequence before surgery, rather than a head-to-head trial. The posted endpoint definition focuses on the primary tumor site and microscopic invasive disease; it does not say that the endpoint requires absence of disease in every tissue or site. The record also lists clinical complete response and biomarker correlations, relapse rate, time to disease progression, overall survival, and adverse events as secondary outcomes. Results for those outcomes are not reported in the registry record.
3. Trial Design
NCT00513695 was a phase II, non-randomized, single-group study with no masking. The registry records enrollment of 68 participants and a completed status. The study start is recorded as 2007-06 and primary completion as 2012-08. These dates describe the registry record and do not, by themselves, establish when each participant received treatment or completed follow-up.
The treatment was given before surgery. The official title describes sunitinib maleate with weekly paclitaxel, followed by doxorubicin and daily oral cyclophosphamide plus G-CSF, as neoadjuvant chemotherapy for locally advanced or inflammatory breast cancer. The arm description states that sunitinib and weekly paclitaxel were administered for 8-12 weeks in the absence of disease progression or unacceptable toxicity. It says that doxorubicin treatment began within 3 weeks of completing that portion, but the arm description ends before giving the full schedule. Details beyond those recorded are not reported in the registry record.
The primary endpoint was assessed at surgery and concerned microscopic pathologic complete response in the primary tumor specimen. Because the design has one treatment group, the main endpoint is a proportion rather than a randomized between-group contrast. The registry record does not report a control group, random assignment, or masking. It also does not report eligibility details beyond the disease descriptions, the analysis population definition, or how participants who did not reach surgery would be handled.
The record lists six outcome measures in total: the primary endpoint and five secondary outcome categories. The secondary categories include response and biomarker correlations, relapse, disease progression, overall survival, and adverse events. The registry records no analyses. Statistical analysis procedures beyond the endpoint description, including any prespecified adjustment or subgroup approach, 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 | Treatment (Neoadjuvant Chemotherapy Before Surgery) |
|---|---|
| STARTED | 68 |
| COMPLETED | 63 |
| NOT COMPLETED | 5 |
Reasons for not completing this period:
| Reason | Treatment (Neoadjuvant Chemotherapy Before Surgery) |
|---|---|
| Discontinued therapy | 2 |
| Progression prior to surgery | 3 |
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 | Treatment (Neoadjuvant Chemotherapy Before Surgery) |
|---|---|---|
| Participants analysed | 68 | |
| Age, Continuous | years | 50 (33 to 79) |
| Sex: Female, Male: Female | Participants | 68 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 4 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 56 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 8 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 4 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 2 |
| Race (NIH/OMB): White | Participants | 50 |
| Race (NIH/OMB): More than one race | Participants | 4 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Microscopic Pathologic CR (pCR) Rate, measured over At the time of surgery.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percent of evaluable participants |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the microscopic pathologic complete response rate at surgery. A participant met the endpoint definition if the surgical specimen showed no evidence of microscopic invasive tumor at the primary tumor site. The measure is expressed as a percentage of evaluable participants. In general, a pathologic endpoint uses examination of tissue removed at surgery to assess disease at the specified site; it is distinct from a clinical response assessment based on examination or imaging.
For a single-group treatment study, this rate describes the observed frequency of the defined tissue finding among the participants included in the endpoint analysis. It can provide a direct summary of response at surgery, but it is not a comparison with an untreated or alternative-treatment group. The registry record does not report the observed rate, the number of participants included in its denominator, or the interval estimate.
The endpoint description says that the rate was to be calculated with an exact 90% binomial confidence interval. Elsewhere, the record labels the dispersion as a 95% confidence interval. These descriptions do not agree. Which confidence level was used for the reported analysis is not clear from the registry record, and no interval result is reported there. The distinction matters because the confidence level determines the width of an interval for the same observed data.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase II study of neoadjuvant sunitinib and chemotherapy in a single group of participants, with microscopic pathologic complete response at surgery as the primary endpoint. Enrollment was 68. However, the registry record does not report the primary endpoint result, its confidence interval, or the evaluable denominator. The central efficacy statistic therefore cannot be interpreted from the posted registry results. It is not possible to state from this record how many participants met the endpoint or how precisely the rate was estimated.
In general, a response rate is a descriptive proportion within the analyzed group. Its precision depends on both the number of observed responses and the number of participants contributing endpoint data. A confidence interval expresses uncertainty around that estimated proportion; it does not make the result a comparison with another treatment. Here, the registry record gives inconsistent confidence-level descriptions: the endpoint definition specifies an exact 90% binomial interval, while the dispersion label says 95%. The result and the interval itself are not reported in the registry record, so neither the estimate nor the source of that inconsistency can be checked from the posted information.
The one-group design also limits what can be concluded. There is no randomized comparator, so the record cannot distinguish the treatment’s contribution from other explanations for outcomes or establish how outcomes would differ under another treatment. The study’s completed status does not fill this gap in the registry results. Likewise, the listed secondary outcomes are not accompanied by results or analyses. Relapse, time to disease progression, overall survival, clinical response, biomarker correlations, and adverse-event outcomes cannot be assessed as efficacy findings from the registry record.
Safety information is more detailed in the record than the primary endpoint information. In the treatment group, 47 of 68 participants were recorded as having a serious adverse event, and 67 of 68 were recorded in the other adverse-event category. The listed serious adverse-event terms include neutropenia (ANC) at 69.1%, leucopenia at 33.8%, anemia at 22.1%, mucositis at 10.3%, ALT elevation at 5.9%, and fatigue at 4.4%. These figures describe reported events in this group; they do not establish that the regimen caused every event, and the record does not provide event-level detail or a comparison group. Deaths are not reported in the registry record.
Any sample-size illustrations based on the observed effect are planning calculations, not observed trial outcomes or observed power. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect set as a stated fraction of the observed effect. Those assumptions describe an illustration, not the actual single-group design. The registry record does not report the trial’s own sample-size justification. Since the primary observed effect is not reported, the figures cannot be independently interpreted as an estimate of what the study found.
9. Statistical Methods Explained
The endpoint is a binary outcome at the participant level: the surgical specimen either meets the specified definition of microscopic pathologic complete response or it does not. A rate summarizes the number meeting the definition relative to the participants included in the analysis. The registry record labels the unit as a percent of evaluable participants, but does not report the number evaluable or the observed rate.
What does a binomial confidence interval convey?
General statistical explanation: when an outcome is recorded as yes or no for each participant, a binomial model is a common way to describe uncertainty in the underlying response proportion. A confidence interval gives a range of values compatible with the observed data under the method’s assumptions. It is not a guarantee that the underlying rate lies in the interval, and it is not a probability that the treatment is effective. A wider interval indicates less precision; a narrower interval indicates greater precision. The width depends on the number of participants contributing data and the observed response frequency.
The endpoint description specifies an exact binomial interval, while the registry’s dispersion label identifies a 95% confidence interval. The description specifies 90%. These are inconsistent entries in the record. An exact binomial method is generally designed to maintain coverage properties for a binary outcome without relying on a normal approximation to the response proportion. The exact method does not resolve the disagreement about the intended confidence level. Neither interval limits nor an endpoint estimate are reported in the registry record.
Why does the single-group design matter?
General statistical explanation: a one-group estimate describes outcomes among participants who received the study regimen. Without a concurrent control group, the estimate does not directly quantify a treatment contrast. A comparison with historical results, if one were made, would depend on additional assumptions about similarity of populations, care, outcome definitions, and follow-up. Such a comparison is not reported in the registry record. Randomization is also not part of this design, so the study does not use random assignment to balance measured and unmeasured factors across treatment groups.
Non-randomized and unmasked are the registry’s design classifications. The record does not report a blinding procedure for assessing the surgical specimens. In general, an endpoint based on pathology may require judgment in specimen assessment, and masking can help limit the influence of knowledge about treatment when judgment is involved. The record does not say whether any safeguards were used, so no specific assessment procedure should be assumed.
How should sample-size illustrations be read?
The sample-size assumptions associated with the posted illustrations are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. These are planning assumptions for hypothetical two-arm calculations, whereas this trial is recorded as a single-group study. Any such calculations are illustrations, not the actual study design, and they are not observed power. Participants per arm for 80% power is a planning quantity, not a count of participants observed in this trial. Time-to-event illustrations are described as using a total number of events, while other illustrations are described as participants per arm. The registry record does not report the trial’s own sample-size justification. It also does not report the observed primary effect needed to interpret an illustration tied to that effect.
What does over-dispersion mean in general?
General statistical explanation: over-dispersion occurs when variation in observed data is greater than expected under a statistical model. In count outcomes, for example, a simple model may assume that counts vary around their mean in a particular way; real data may show more spread because participants differ or events cluster. If over-dispersion is ignored, standard errors can be too small and intervals or tests can appear more precise than warranted. No over-dispersion assessment or count-model analysis is reported in the registry record. The primary endpoint is described as a binary response rate, and the record does not specify an over-dispersion method.
What analysis details are absent?
The record identifies the primary endpoint and its timing but does not report a statistical analysis plan, the primary analysis population, handling of missing endpoint observations, or criteria for excluding participants from the evaluable group. No analyses are recorded. It is therefore not possible to confirm whether the reported endpoint, had it been posted, used all enrolled participants, only those who underwent surgery, or another defined group. The registry record also does not describe any interim analyses, model adjustments, subgroup analyses, or sensitivity analyses. These methods should not be inferred from the phase designation or from the endpoint description.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary endpoint and five secondary outcome categories, including clinical complete response and biomarker correlations, relapse rate, time to disease progression, overall survival, and adverse events. It records no analyses and does not report results for these outcomes. A plan for controlling multiplicity across endpoints or across repeated statistical tests is not reported in the registry record.
General statistical explanation: when many outcomes or comparisons are examined, the chance of at least one apparently notable finding can rise if each is interpreted separately. A prespecified strategy can address that issue, for example by defining a primary outcome or adjusting inference across multiple tests. The registry record identifies a primary endpoint, but does not report whether or how multiplicity was addressed for secondary outcomes, biomarker associations, or any additional analyses. No particular adjustment should be assumed.
The primary endpoint denominator is described as evaluable participants, but its definition and size are not reported in the registry record. The record gives enrollment of 68 but does not report how many participants received treatment, reached surgery, had an assessable specimen, or had missing endpoint data. A missing-data method and an analysis population such as intention-to-treat or per-protocol are not reported in the registry record. Consequently, the effect of missing or unevaluable observations on the response estimate cannot be assessed.
The registry record also does not report whether interim analyses were planned or conducted, or whether any stopping rules were used. The completed status and primary completion date do not establish those details. General statistical explanation: missing outcome data can alter a rate if participants with unobserved outcomes differ from those with observed outcomes. Transparent reporting of the analysis population, missingness, and any sensitivity analysis helps readers understand how dependent a result is on those choices. Those details are not reported in the registry 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 |
|---|---|---|---|
| Treatment (Neoadjuvant Chemotherapy Before Surgery) | 47 | 68 | 69.1% |
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 |
|---|---|
| Treatment (Neoadjuvant Chemotherapy Before Surgery) | 67 of 68 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Neoadjuvant Chemotherapy Before Surgery) |
|---|---|---|
| Neutropenia (ANC) | Blood and lymphatic system disorders | 47 of 68 |
| Leucopenia | Blood and lymphatic system disorders | 23 of 68 |
| Anemia | Blood and lymphatic system disorders | 15 of 68 |
| Mucositis | Respiratory, thoracic and mediastinal disorders | 7 of 68 |
| ALT elevation | Investigations | 4 of 68 |
| Fatigue | General disorders | 3 of 68 |
| Diarrhea | Gastrointestinal disorders | 3 of 68 |
| Sensory Neuropathy | Nervous system disorders | 3 of 68 |
| Pain | General disorders | 2 of 68 |
| Nail changes | Skin and subcutaneous tissue disorders | 2 of 68 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Neoadjuvant Chemotherapy Before Surgery) |
|---|---|---|
| Fatigue | General disorders | 37 of 68 |
| Leucopenia | Blood and lymphatic system disorders | 34 of 68 |
| Anemia | Blood and lymphatic system disorders | 32 of 68 |
| Neutropenia (ANC) | Blood and lymphatic system disorders | 22 of 68 |
| Pain | General disorders | 15 of 68 |
| Nail changes | Skin and subcutaneous tissue disorders | 12 of 68 |
| Diarrhea | Gastrointestinal disorders | 9 of 68 |
| Rash | Skin and subcutaneous tissue disorders | 8 of 68 |
| Hypertension | Vascular disorders | 8 of 68 |
| Heartburn | Gastrointestinal disorders | 8 of 68 |
Serious events with the highest arm-level rate: neutropenia (anc) (69.1%), leucopenia (33.8%), anemia (22.1%), mucositis (10.3%), alt elevation (5.9%).
The registry records 68 participants at risk in the treatment group. It reports 47 participants affected by serious adverse events and 67 affected in the other adverse-event category. These are participant counts, not counts of distinct event episodes; the record does not provide episode-level detail. The serious-event terms listed most prominently are neutropenia (ANC) at 69.1%, leucopenia at 33.8%, anemia at 22.1%, mucositis at 10.3%, ALT elevation at 5.9%, and fatigue at 4.4%.
These term percentages should be read as reported safety summaries, not as mutually exclusive categories. One participant may have experienced more than one event, so the percentages should not be added. The registry record does not give the event grades, timing, duration, management, or attribution for each event. With a single treatment group and no randomized comparator, these figures describe events recorded during the study but do not establish how event rates would compare under another treatment. Deaths are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The study’s stated primary question concerns the frequency of microscopic pathologic complete response at surgery after neoadjuvant treatment with sunitinib and chemotherapy. The registry record defines that outcome clearly: no microscopic invasive tumor at the primary tumor site in the surgical specimen. But it does not post the observed response rate, its confidence interval, or the number of evaluable participants. The endpoint definition therefore explains what the study intended to measure, not what the study found.
The record does establish the broad design: a phase II, non-randomized, single-group study with enrollment of 68. It describes a treatment sequence before surgery and lists several secondary outcomes. It does not provide results for those secondary outcomes or a comparison group. As a result, the registry information cannot establish whether the regimen improved response relative to another approach, nor can it quantify a comparative effect. The single-group design does not support a like-for-like treatment comparison.
The reported safety counts and serious adverse-event terms offer descriptive information about the enrolled treatment group. They do not on their own prove that each event was caused by treatment, and the record lacks details needed to assess severity over time or compare risk with another group. The deaths field has no reported value; deaths are not reported in the registry record.
More generally, a confidence interval would communicate precision around the response estimate, but none is posted. The endpoint description and dispersion label also disagree about whether the interval was exact 90% or 95%. Without a result and a clear interval, readers cannot assess the precision of the primary finding. The registry record’s completed status does not substitute for posted outcome data. The appropriate interpretation is therefore limited to the study design, endpoint definition, enrollment, and recorded safety summaries; the treatment’s effect on the primary endpoint is not reported in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why a clear endpoint definition and a posted endpoint result are both necessary for statistical interpretation. The registry identifies a specific surgical pathology outcome and defines the tissue finding that qualifies. That makes the intended measurement understandable. But without the observed rate, evaluable denominator, and interval estimate, the central result cannot be assessed for either magnitude or precision.
It also illustrates the difference between estimating a rate and estimating a treatment effect. A single-group response proportion can describe the observed experience in the study group. It does not, by itself, estimate what would have happened under a different regimen. Non-randomized, single-group evidence therefore has a different inferential scope from a randomized comparison. This distinction matters when interpreting any response rate, regardless of its size.
The record’s confidence-interval inconsistency is another statistical reporting concern. The endpoint description specifies an exact 90% binomial interval, while the dispersion label says 95%. Confidence level is part of the interpretation of an interval, so inconsistent labeling leaves an important method detail unresolved. The absent interval result compounds that uncertainty.
Finally, the record shows that trial registration fields can contain useful design and safety information while still omitting results and analysis details needed for a complete assessment. Enrollment, outcome definitions, adverse-event counts, and study status are informative, but they do not replace a transparent report of the analyzed population, missing observations, statistical methods, and endpoint estimates. Those omissions make this trial particularly useful as an example of the boundary between what a registry record can describe and what it can support statistically.
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 efficacy is that the registry record does not report the primary endpoint result. It also does not report the number of evaluable participants or the confidence interval. The endpoint is defined for the surgical specimen and is expressed among evaluable participants, but the record does not explain how evaluability was determined or how participants without an assessable specimen were handled.
The design is recorded as non-randomized and single-group, with no masking. There is no concurrent control group for a like-for-like comparison. The registry record does not report a comparison with another treatment or provide an analysis that could support a comparative estimate. The record also does not state whether pathology assessment was blinded to treatment or describe procedures intended to limit assessment bias.
The confidence-interval description is internally inconsistent: the endpoint description names an exact 90% binomial interval, while the dispersion label states 95%. Neither interval result is posted. The statistical analysis plan, analysis population, missing-data handling, multiplicity strategy, interim analyses, and sample-size justification are not reported in the registry record.
The arm description is truncated after the start of the later treatment component. Although the official title names doxorubicin, daily oral cyclophosphamide, and G-CSF, the full treatment schedule is not reported in the registry record. Safety reporting is also limited: the record gives affected-participant counts and selected serious-event percentages, but does not report detailed event grades, timing, duration, attribution, or deaths. Results for the listed secondary outcomes are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sunitinib Malate, Paclitaxel, Doxorubicin Hydrochloride, and Cyclophosphamide Before Surgery in Treating Patients With Stage IIB-IIIC Breast Cancer — NCT00513695.
- Registry record last updated 2019-08-07; status last verified 2019-07.
- Results first posted 2017-05-10.
- 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
NCT00513695 is a completed phase II, non-randomized, single-group study sponsored by the University of Washington. It enrolled 68 participants and evaluated sunitinib with chemotherapy before surgery for locally advanced or inflammatory breast cancer. The primary endpoint was microscopic pathologic complete response at surgery, defined by no microscopic invasive tumor at the primary tumor site in the surgical specimen.
The registry record does not report the primary response rate, evaluable denominator, confidence interval, or results for the listed secondary outcomes. Its endpoint description specifies an exact 90% binomial interval, while its dispersion label says 95%. Safety summaries report 47 of 68 participants with serious adverse events and 67 of 68 in the other adverse-event category; deaths are not reported in the registry record. The available information supports description of the design and posted safety figures, but not an assessment of the primary efficacy result or a comparison with another treatment.