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 Effect of Chemotherapy and Radiation Prior to Surgery for Triple Negative Breast Cancer (NCT00603408), a na phase 2 study. The primary outcome is Overall Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00603408 |
| Phase | Phase 2 |
| Condition | Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Washington University School of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
People with locally advanced triple-negative breast cancer were the intended study population. The registry record does not provide further eligibility details or a populated description of the primary-outcome population.
Intervention
The study evaluated cisplatin given with radiation therapy before surgery. The listed regimen describes cisplatin 75 mg/m^2 IV on Day 1 of Week 1, Day 1 of Week 2, Day 1 of Week 7, and Day 1 of Week 10. Radiation was planned to the breast or chest wall and regional nodal areas, followed by recommended surgery.
Comparator
There was no separate comparator group. The trial is recorded as single-group, with allocation listed as NA and masking as none. It therefore does not provide a randomized, like-for-like comparison with another treatment or with no treatment.
Primary question
The primary question was whether cisplatin-based chemoradiation before surgery was associated with an overall response at the time of surgery, specified as week 13. The registry record does not report the number of participants in each response category or an overall-response result.
The stated purpose was to assess response to treatment and to collect tumor, blood, and bone marrow samples for biological investigations. The brief description says investigators were interested in whether cisplatin could alter tumor DNA and affect tumor-cell growth and death. These biological aims do not replace the clinical primary endpoint, and the registry record does not report results for those aims.
3. Trial Design
This was a phase 2, single-group study of neoadjuvant cisplatin-based chemoradiation for locally advanced triple-negative breast cancer. The registry lists Washington University School of Medicine as the sponsor, enrollment of 5, a start date of 2007-12, and primary completion in 2009-12. The status is recorded as terminated. The reason for termination is not reported in the registry record.
The treatment group is named “Cisplatin + Radiation + Recommended Surgery.” The description specifies cisplatin 75 mg/m^2 IV on Day 1 of Week 1, Day 1 of Week 2, Day 1 of Week 7, and Day 1 of Week 10. Radiation to the breast or chest wall was specified as a total dose of 50-60 Gy in 1.8-2.0 Gy daily fractions. Internal mammary nodes, supraclavicular fossa nodes, and axillary nodal basins were to receive 45-50 Gy over 5-6 weeks. Surgery followed the neoadjuvant treatment. The intervention list includes cisplatin, radiation therapy, and mastectomy.
Because the design has a single treatment group, the study can describe outcomes among its participants but cannot isolate the contribution of cisplatin from radiation or surgery through a randomized comparison. Allocation is listed as NA, and masking as none. Further details on eligibility criteria, treatment delivery, deviations from the intended regimen, and how many participants underwent surgery are not reported in the registry record.
The registry lists secondary aims concerning time to disease progression, overall survival, surgical complications, medical toxicities, disseminated cancer cells in bone marrow and their correlation with tumor response, development of animal models, and collection of samples for an FNA assay. The record lists 8 outcome measures, but does not report results for these secondary aims in the information available.
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 | Cisplatin + Radiation + Recommended Surgery |
|---|---|
| STARTED | 5 |
| COMPLETED | 5 |
| NOT COMPLETED | 0 |
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 | Cisplatin + Radiation + Recommended Surgery |
|---|---|---|
| Participants analysed | 5 | |
| Age, Continuous | years | 52 (42 to 62) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response, measured over At the time of surgery (week 13).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is “Overall Response,” expressed as the number of participants, with assessment specified at the time of surgery (week 13). The registry describes response categories using changes in target and non-target lesions and tumor-marker status. Complete response is defined as disappearance of all target and non-target lesions and normalization of the tumor-marker level. Partial response requires at least a 30% decrease in the sum of the longest diameters of target lesions relative to baseline. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progressive disease, using the smallest sum of longest diameters since treatment began as reference. Progressive disease includes at least a 20% increase from that smallest recorded sum, new lesions, or unequivocal progression of existing non-target lesions.
Response at surgery is used to describe the tumor’s status after the neoadjuvant treatment and before or at the time of surgery. It is a clinical outcome, distinct from the biological sample aims and the later outcomes such as disease progression and survival. The registry record does not state how the response categories were combined into an overall-response summary or report the participant counts for any category. Consequently, the endpoint definition is available, but the primary result cannot be interpreted from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry record identifies a primary endpoint and a planned assessment time, but does not report the observed overall-response result. It gives no number of participants with complete, partial, stable, or progressive disease and no overall-response estimate. Enrollment is listed as 5, but enrollment alone does not establish how many participants completed treatment, underwent surgery, or had an evaluable endpoint. Those analysis counts are not reported in the registry record.
The study’s single-group structure also limits what can be concluded. Without a concurrent comparator, an observed response pattern would describe outcomes in the treated group, not the difference attributable to the treatment regimen relative to another approach. The intervention combined cisplatin, radiation, and recommended surgery, so the design does not separately estimate the effect of each component. No causal comparison between treatment strategies is available from this design.
The registry status is terminated. That status is important context, but the reason for termination is not reported in the registry record. It would be inappropriate to infer whether termination reflected recruitment, safety, operational, efficacy, or other considerations. The record also does not state whether the planned enrollment was completed, why enrollment was 5, or whether follow-up was complete.
The described response categories are clinically interpretable definitions, but definitions are not findings. In particular, the listed threshold for partial response and the criteria for progressive disease do not indicate how participants actually fared. Similarly, the brief summary’s biological rationale and plans for tumor, blood, and bone marrow sampling do not establish that a biological change occurred. Results for those investigations are not reported in the registry record.
The safety figures are separate from the primary efficacy endpoint. The record reports serious adverse events among participants at risk and provides a list of serious-event terms with percentages. These figures should be read as descriptive observations in a small single-group cohort, not as a controlled estimate of excess risk. The registry record does not give a comparator, person-time, or information sufficient to attribute events to a particular treatment component. The listed affected-participant counts and event terms do not resolve the missing primary-response result.
Overall, the record documents the study question, regimen, intended endpoint, and some safety summaries, while leaving the main clinical outcome unreported. The evidence in this registry entry therefore supports description of the study design and reporting gaps, not an estimate of treatment benefit or a comparison with other care.
9. Statistical Methods Explained
Interpreting a clinical trial requires separating its design, endpoint definition, analysis, and reported results. In this study, the design is single-group and the stated primary endpoint is overall response at surgery. The registry record supplies the response-category definitions but does not report the outcome counts or a statistical analysis of those counts. The following concepts explain what such methods would mean in general; they should not be read as claims that a particular analysis was performed in this trial.
What does a single-group design estimate?
A single-group study observes outcomes among participants receiving the study regimen. Its results can describe the outcomes in that cohort, provided the analysis population and outcome data are reported. Without a concurrent comparison group, it cannot directly estimate how outcomes would differ under another regimen or under no study treatment. Differences from outcomes in another population may reflect differences in eligibility, disease characteristics, follow-up, outcome assessment, or other factors, rather than a treatment effect.
Here, the registry identifies cisplatin, radiation therapy, and recommended surgery as the treatment sequence. Since the design does not compare separate groups, it cannot disentangle the contribution of those treatment components. Allocation is listed as NA and masking as none. Further randomization or blinding procedures are not reported in the registry record.
What does a binary outcome count mean?
A count endpoint records how many participants meet a specified condition. For an outcome such as response, interpretation depends on the precise definition of response and on knowing the denominator: which participants were assessed and how missing or unevaluable assessments were handled. A count without a clear denominator can be difficult to interpret, while a denominator without a reported outcome count does not establish the outcome rate. The registry lists the primary endpoint as a participant count, but does not provide the response counts or analysis population.
The record gives definitions for complete, partial, stable, and progressive disease. It does not say how the categories were combined for the reported overall-response endpoint. That distinction matters because an analysis could report each category separately or define a particular combination as response. No such combination should be assumed from the endpoint label alone.
How should an estimate and confidence interval be read?
In general, an estimate summarizes the observed outcome in a study, while a confidence interval describes the statistical uncertainty around that estimate under the assumptions of the analysis. A narrower interval indicates greater precision, and a wider interval indicates less precision. With a small cohort, estimates can be especially sensitive to the outcome of individual participants. These are general principles, not reported calculations for this study: the registry record does not provide a primary-response estimate or confidence interval.
A confidence interval does not address every source of uncertainty. It reflects sampling uncertainty under a statistical model, not uncertainty from selection, missing outcome assessments, changes in treatment, outcome measurement, or lack of a comparison group. Even a precise estimate in a single group would not by itself establish a comparative treatment effect.
Why does over-dispersion matter?
For count outcomes, statistical models often rely on assumptions about how variable counts are across participants or groups. Over-dispersion means the observed variability is greater than a model expects. If a model ignores that extra variability, uncertainty can be understated and inferential results may appear more precise than warranted. This is a general consideration for count-data methods. The registry record does not report a count model, an over-dispersion assessment, or a model-based primary analysis for this trial.
What does a p-value address?
In general, a p-value measures how compatible observed data are with a specified null model, assuming that model and the analysis assumptions hold. It does not measure the probability that the null hypothesis is true, the size or importance of an effect, or the probability that a result will replicate. For this single-group study, an inferential test would also need a stated reference or null value. The statistical plan and any p-value for the primary endpoint are not reported in the registry record.
How should planning calculations be distinguished from trial results?
Sample-size calculations are planning tools. They use assumptions about an effect, variability, significance level, allocation, and desired power to illustrate how much information a future comparison might require. Such calculations are not observed trial outcomes and do not establish the probability that a treatment worked. The registry record does not report the trial’s sample-size justification, target enrollment rationale, or a prospective statistical plan. Any planning illustration based on an observed effect should not be mistaken for an analysis of this trial’s efficacy.
In general, recalculations from summary statistics can differ from a sponsor’s prespecified analysis because they may use unadjusted methods, rounded inputs, or different assumptions. If a discrepancy is identified between a sponsor-reported analysis and a simple recalculation, the sponsor’s own analysis takes precedence for describing the trial’s reported analysis. The registry record does not provide a sponsor analysis of the primary endpoint.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when several outcomes, comparisons, time points, or subgroup analyses are tested. The more analyses performed, the greater the chance that at least one result may appear noteworthy by chance if no adjustment or prespecified hierarchy is used. Appropriate interpretation depends on which outcomes were planned as primary or secondary, which analyses were actually conducted, and whether any multiplicity procedure was specified. The registry lists one primary outcome and 8 outcome measures, but the statistical plan, testing hierarchy, and any adjustment for multiple analyses are not reported in the registry record.
The record lists 0 analyses. No primary endpoint estimate, inferential test, or analysis details are given in the registry entry. This absence does not show that analyses were or were not conducted outside the registry; it means analysis results are not reported in the registry record. Interim analyses and any rules for early stopping are also not reported in the registry record, so no such procedures should be assumed.
Missing-data handling is another important part of a clinical outcome analysis. The record does not state how participants without a surgery assessment, those who withdrew, or those with unevaluable response would be handled. It also does not report the number of missing primary outcomes. The enrolled count should therefore not be treated as the denominator for the response endpoint without an analysis population and outcome count.
Analysis populations can include all enrolled participants, all participants who started treatment, or participants meeting specified criteria, depending on the prespecified plan. The record does not identify an intention-to-treat, per-protocol, safety, or other analysis population for the primary outcome. The endpoint population field is not populated. These omissions limit both reproducibility and assessment of possible bias due to exclusions or missing assessments.
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 |
|---|---|---|---|
| Cisplatin + Radiation + Recommended Surgery | 3 | 5 | 60.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Cisplatin + Radiation + Recommended Surgery | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Cisplatin + Radiation + Recommended Surgery |
|---|---|---|
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 5 |
| Hypoglycemia | Metabolism and nutrition disorders | 1 of 5 |
| Viral hepatitis | Infections and infestations | 1 of 5 |
| Infection unknown ANC - general wound | Infections and infestations | 1 of 5 |
| Burn due to radiation therapy | Skin and subcutaneous tissue disorders | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cisplatin + Radiation + Recommended Surgery |
|---|---|---|
| Fatigue | General disorders | 5 of 5 |
| Rash: dermatitis associated with radiation therapy | Skin and subcutaneous tissue disorders | 5 of 5 |
| Hemoglobin | Blood and lymphatic system disorders | 4 of 5 |
| Lymphopenia | Investigations | 4 of 5 |
| Hypertension | Vascular disorders | 3 of 5 |
| Dermatology - other | Skin and subcutaneous tissue disorders | 3 of 5 |
| Nausea | Gastrointestinal disorders | 3 of 5 |
| Edema: limb | General disorders | 3 of 5 |
| Alkaline phosphatase | Investigations | 3 of 5 |
| ALT | Investigations | 3 of 5 |
Serious events with the highest arm-level rate: hyperglycemia (20.0%), hypoglycemia (20.0%), viral hepatitis (20.0%), infection unknown anc - general wound (20.0%), burn due to radiation therapy (20.0%).
The registry lists 5 participants at risk for serious adverse events and 3 participants affected. For other adverse events, it lists 5 participants at risk and 5 affected. These are participant counts as recorded; they are distinct from the primary overall-response endpoint and should not be interpreted as evidence about tumor response.
The serious-event terms listed include hyperglycemia, hypoglycemia, viral hepatitis, infection unknown ANC - general wound, and burn due to radiation therapy. Each term is accompanied by 20.0%. These percentages are reported as registry figures, not as an independently calculated rate. The record does not clarify whether participants could contribute to more than one term, how the terms were coded, or the event-specific severity, timing, or duration. Therefore the terms should not be summed or treated as mutually exclusive categories.
Safety observations in a single-group study are descriptive. Without a control group and additional context on exposure and follow-up, they cannot establish how much risk is attributable to cisplatin, radiation, surgery, the underlying disease, or other causes. The registry record does not provide the reason for termination or a causal assessment of the listed events. The figures describe what is posted, not a comparative safety estimate.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase 2 study was designed to examine cisplatin-based chemoradiation before surgery for locally advanced triple-negative breast cancer. It identifies the treatment components, gives a regimen description, and defines an overall-response endpoint assessed at surgery. It also lists safety summaries and several secondary outcome aims. These elements describe the clinical question and the intended observations.
The record does not establish the primary clinical result. No response count, response proportion, estimate of uncertainty, or primary analysis is reported. The enrollment of 5 does not substitute for these missing outcome data. The record also does not identify how many participants completed treatment, had surgery, or were included in an endpoint analysis.
Because this was a single-group study, even a reported response result would describe outcomes in the study cohort rather than provide a randomized comparison with another treatment. The combined regimen also prevents the design from separating the effects of cisplatin, radiation, and surgery. The posted safety figures likewise have no control group for comparative interpretation.
The registry status is terminated, but the reason for termination is not reported in the registry record. It is not possible to infer why the study stopped, whether the termination was related to recruitment or outcomes, or whether follow-up was complete. The biological rationale and sample-collection aims are not evidence that the hypothesized tumor changes occurred; those results are not reported in the registry record.
Accordingly, the record is informative about the planned study and its reporting limits, but it does not provide enough primary-outcome information to estimate response or support a comparative conclusion about treatment effects. This statistical interpretation is not medical advice.
13. Why This Trial Matters Statistically
This trial is statistically notable because its registry entry combines a clearly named primary clinical question with a very small enrolled cohort and no reported primary-outcome result. The stated outcome is a count of participants with overall response at surgery, but the record does not report the counts needed to interpret that endpoint. The distinction between an outcome definition and an outcome finding is fundamental: a detailed definition improves clarity, but it cannot replace observed data.
The single-group design also makes the limits of causal interpretation visible. A cohort’s observed outcomes can be described, but without a concurrent comparator the study cannot directly estimate how outcomes differ under another strategy. Since cisplatin, radiation, and surgery are part of the same treatment sequence, their separate contributions cannot be estimated from this design.
The registry includes both clinical and biological aims, as well as safety outcomes. Keeping these outcome families distinct matters: a plan to collect samples does not demonstrate a biological effect, and adverse-event counts do not answer the primary response question. Transparent reporting of denominators, missing outcomes, analysis populations, and statistical methods would help readers judge each outcome on its own terms.
Finally, the terminated status and absence of analysis results leave important questions unresolved. The reason for termination, planned sample-size rationale, handling of missing outcomes, and analysis plan are not reported in the registry record. These gaps illustrate why trial registration is most useful when outcome definitions are accompanied by complete results and sufficient methods detail to interpret them.
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 registry record has substantial limitations for evaluating the clinical findings. It reports enrollment of 5, but no primary overall-response counts or analysis population. Although the endpoint is specified at surgery (week 13), the record does not report how many participants reached that assessment, whether any assessments were missing, or how unevaluable cases were handled. It also does not explain how the response categories were combined into the overall-response outcome.
The design is single-group, with allocation listed as NA and masking as none. It therefore lacks a randomized comparator and cannot support a direct treatment contrast. The combined treatment sequence further limits attribution to any individual component. Eligibility criteria, participant characteristics, treatment completion, deviations from the stated regimen, and the number undergoing surgery are not reported in the registry record.
The status is terminated, but the reason and timing of termination are not reported in the registry record. The record does not provide a sample-size justification, statistical analysis plan, interim analysis information, multiplicity approach, or analysis populations. It also does not report primary-outcome estimates, uncertainty intervals, or tests. Secondary outcome results are not reported in the registry record.
Safety information is limited to the posted affected-participant counts and listed terms with percentages. Details on event timing, duration, severity by term, participant overlap, exposure, and causal assessment are not provided. Without a comparator and fuller follow-up information, these figures are descriptive rather than comparative. These limitations do not establish that the study had a particular outcome; they constrain what can be concluded from its registry entry.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effect of Chemotherapy and Radiation Prior to Surgery for Triple Negative Breast Cancer — NCT00603408.
- Registry record last updated 2016-08-19; status last verified 2016-07.
- Results first posted 2016-08-19.
- 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
NCT00603408 is a phase 2, single-group study sponsored by Washington University School of Medicine. The registry lists 5 participants enrolled, a start date of 2007-12, primary completion in 2009-12, and status as terminated. It evaluated cisplatin-based chemoradiation before recommended surgery for locally advanced triple-negative breast cancer.
The primary endpoint was overall response at surgery (week 13), with definitions for complete, partial, stable, and progressive disease. The primary result is not reported in the registry record. The entry lists 8 outcome measures and 0 analyses. It reports serious adverse events affecting 3 of 5 participants at risk and other adverse events affecting 5 of 5 at risk; listed serious-event terms each carry 20.0%. The reason for termination, statistical plan, missing-data approach, and secondary outcome results are not reported in the registry record.