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 Phase II Study of Capecitabine and Cisplatin to Treat Metastatic Triple Negative Breast Cancer (NCT01928680), a na phase 2 study. The primary outcome is Overall Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01928680 |
| Phase | Phase 2 |
| Condition | Metastatic Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 33 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Chinese Academy of Medical Sciences |
| Results posted | Yes |
2. Clinical Question
Population
Patients with anthracycline- and taxane-pretreated metastatic triple negative breast cancer. The registry lists metastatic breast cancer as the condition and does not provide further eligibility details or a description of the outcome-analysis population.
Intervention
Cisplatin plus capecitabine. The regimen description lists capecitabine 1000mg/m2 orally Bid on day 1 to day 14, plus cisplatin 75mg/m2 on day1 of each 21 day cycle, until progression or untolerable toxicity.
Comparator
There is no separate comparator group. The registry describes a single arm phase II trial, with allocation listed as NA and masking as NONE.
Primary question
What overall response rate is observed at six months among patients receiving the combination? The registry identifies this endpoint but does not report its measured value or an analysis result.
This is a single-group question about outcomes during treatment, not a randomized comparison between treatment strategies. Accordingly, the record cannot provide a direct estimate of how outcomes would differ under another treatment or under a control condition. The stated aim is to explore efficacy and safety in the specified pretreated metastatic population.
The question is clinically focused, but the registered endpoint description is brief. It names overall response rate, gives the unit as percentage of response, and specifies a six-month timeframe. It does not describe response criteria, how response was assessed, or the population denominator used for the primary analysis. Those details are not reported in the registry record.
The clinical question is therefore narrower than a claim about long-term outcomes or comparative benefit. Progression free survival, overall survival, and adverse events are listed as secondary outcomes, but their results are not reported in the registry record. The primary endpoint result is also not reported, so the record does not establish the response rate observed in this study.
3. Trial Design
The registry describes NCT01928680 as a phase II study of cisplatin and capecitabine for anthracycline- and taxane-pretreated metastatic triple negative breast cancer. The sponsor is the Chinese Academy of Medical Sciences. The record lists a start date of 2012-11 and primary completion of 2015-10; its status is UNKNOWN.
The design is single group, with no masking and allocation listed as NA. The arm description explicitly calls this a single arm phase II clinical trial. Participants in the treatment group received cisplatin/capecitabine according to the listed regimen: capecitabine 1000mg/m2 orally Bid on day 1 to day 14, plus cisplatin 75mg/m2 on day1 of each 21 day cycle, until progression or untolerable toxicity. The record does not describe a comparator regimen.
The registry lists enrollment as 33. That entry should be read as the record's enrollment figure; it does not, by itself, establish how many participants contributed data to each outcome analysis. The number included in the primary endpoint analysis and the reasons for any exclusions are not reported in the registry record.
Overall response rate at six months is the registered primary outcome. Progression free survival, overall survival, and number and severity of adverse events are listed as secondary outcomes. Although the record lists four outcome measures, it reports no analysis results. The methods used to assess response, progression, survival, and adverse events are not reported in the registry record.
A single-group phase II design can describe outcomes observed among treated participants, but it does not create a concurrent control group. It therefore cannot separate treatment-associated outcomes from other explanations, including the natural course of illness or differences in patient characteristics. The record does not report a protocol-based sample-size justification, analysis plan, or interim analysis plan.
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/Capecitabine |
|---|---|
| STARTED | 33 |
| COMPLETED | 33 |
| 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/Capecitabine |
|---|---|---|
| Participants analysed | 33 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 32 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | years | 48 (SD 10) |
| Sex: Female, Male: Female | Participants | 33 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: China | participants | 33 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate, measured over 6 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of response |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is overall response rate, expressed in the registry as a percentage of response, with a six-month timeframe. In general, an overall response rate describes the proportion of an analyzed group meeting predefined criteria for response. The precise criteria and assessment procedures for this study are not reported in the registry record, so the endpoint cannot be interpreted more specifically from the record alone.
Response rate is used in clinical studies because it summarizes whether participants meet a defined response threshold during a specified assessment period. It is a proportion, rather than a measure of how long a response lasts or how long participants live. Those are different questions. The registry lists progression free survival and overall survival as secondary outcomes, but does not report their results.
The six-month timeframe is part of the registered endpoint definition. The record does not state how visits or assessments were scheduled within that period, how missing assessments were handled, or whether response confirmation was required. These details matter because endpoint definitions and assessment rules determine which observations count as responses and which participants enter the denominator.
No primary endpoint value, confidence interval, or statistical test is reported in the registry record. Thus, the endpoint is identifiable, but its observed estimate and precision cannot be evaluated from the recorded results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a treatment and a primary endpoint, but it does not provide the primary endpoint result. Overall response rate is specified for six months, yet no percentage, numerator, denominator, confidence interval, or test result is reported in the registry record. Consequently, the record does not establish what response rate was observed or how precisely it was estimated.
The same limitation applies to the secondary outcomes. Progression free survival and overall survival are listed, as is the number and severity of adverse events, but outcome results and analysis details for these endpoints are not reported in the registry record. The enrollment entry is 33, but that number should not be taken as proof that 33 participants contributed to every outcome analysis. Outcome-specific analysis populations are not reported.
The single-group structure is central to interpretation. The study describes outcomes for participants receiving cisplatin/capecitabine, but it has no separate control arm. Even if an outcome estimate were available, a within-group result alone would not provide a randomized estimate of the difference between this regimen and another strategy. Differences in patient characteristics, disease course, assessment, and other factors could influence outcomes. The registry does not report a comparative analysis.
The safety entries report 0 serious adverse events among 33 at risk and 27 other adverse events among 33 at risk. These figures describe the registry's recorded safety fields, not a comparison with another group. The record does not list serious adverse event terms, and deaths are not reported. The categories and methods used to collect or classify adverse events are not described in the registry record, so the figures should not be expanded into claims about particular event types or causes.
There are no analyses listed in the record. Statistical methods, prespecified decision criteria, and uncertainty estimates are not reported. General statistical interpretation depends not only on a point estimate, but also on its precision, the analysis population, and the design that generated it. Here, the absent endpoint estimates prevent assessment of magnitude and precision, while the absence of a comparator limits causal interpretation by design.
The appropriate reading is therefore descriptive: the registry records a phase II single-group study, its intended response endpoint and timeframe, the regimen, and selected safety counts. It does not report results sufficient to quantify response, survival, or comparative effects. No conclusion about whether the regimen works or does not work follows from the information recorded.
9. Statistical Methods Explained
The statistical information in the registry record is limited. It names an overall response rate endpoint and a six-month timeframe, lists secondary outcomes, and records some adverse event counts. It does not report statistical analyses, effect estimates, confidence intervals, p-values, or details of the methods used to analyze the outcomes. The concepts below explain how such quantities would generally be interpreted; they are not descriptions of methods confirmed for this study.
What does a response rate estimate?
In general, a response rate is a proportion: the number of participants meeting a predefined response definition relative to an analysis denominator. The estimate depends on the response criteria, the timing of assessments, and the rules for handling participants without an evaluable assessment. The registry names overall response rate and gives a six-month timeframe, but the response criteria, assessment procedures, denominator, and handling of missing assessments are not reported in the registry record. No response-rate estimate is reported either.
A proportion is often accompanied by a confidence interval. In general, a confidence interval expresses the uncertainty around an estimate under a specified statistical model and sampling process. A narrower interval indicates greater precision under those assumptions; a wider interval indicates less precision. It is not a guarantee that the true value lies within the interval. No confidence interval for the primary endpoint is reported in the registry record, so its precision cannot be assessed.
What can a single-group design estimate?
In general, a single-group study can describe the outcomes observed among participants receiving an intervention. Without a concurrent comparator, it does not directly estimate a treatment contrast. A difference between an observed outcome and a value from another population or source would depend on assumptions about comparability; the registry record does not identify such a comparison. The design is described as single group, and no randomized comparison is reported.
This distinction is separate from whether an outcome is clinically important or whether a statistical estimate is precise. A precisely estimated outcome in one group still does not, by itself, show what would have happened to similar participants under another strategy. The registry record has no comparative effect estimate to interpret.
What would a significance test mean?
In general, a statistical test evaluates how compatible the observed data are with a specified null hypothesis, given a statistical model. A p-value is not the probability that the null hypothesis is true, nor does it measure the size or importance of an effect. Its interpretation depends on the hypothesis, analysis method, data, and assumptions. No p-value, null hypothesis, or analysis method is reported in the registry record. A significance conclusion therefore cannot be drawn from the recorded information.
Effect estimates and their uncertainty are usually more informative together than a significance label alone. The estimate describes the observed magnitude, while the confidence interval conveys precision under the analysis assumptions. For the primary endpoint in this study, neither is reported. The absence of results should not be treated as evidence that the estimate is zero or that a test was nonsignificant.
How should sample-size planning be understood?
In general, sample-size planning is based on assumptions about the outcome, target effect, variability, statistical error rates, and design. A planned sample size is not the same as an observed result and does not establish that a study had a particular probability of finding an effect after the data are collected. The registry lists enrollment as 33, but the rationale for that number and the study's own sample-size assumptions are not reported in the registry record. No sample-size justification is reported.
A planning illustration based on an observed effect is conceptually different from the original design justification. Such an illustration describes hypothetical sample requirements under stated assumptions; it is not observed power and does not recover a missing trial result. No numerical planning estimates are reported in the registry record.
What do the safety counts convey?
In general, safety counts describe recorded events or participants with events within a defined observation period and population. Their interpretation depends on definitions, exposure, collection procedures, and whether the count represents participants or event episodes. The registry reports 0 serious adverse events among 33 at risk and 27 other adverse events among 33 at risk. It does not explain the operational definitions or collection period in the record. The counts should therefore be reported as registry entries, without inferring event rates over time or causes.
Overall, the record permits a basic description of the design and some safety counts, but not a reanalysis of efficacy. Analysis populations, missing-data methods, endpoint assessment rules, and statistical methods are not reported in the registry record. Those omissions prevent reconstruction of an estimate or its uncertainty.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists an overall response rate as the primary outcome and progression free survival, overall survival, and number and severity of adverse events as secondary outcomes. It does not report a statistical analysis plan, hypotheses, or procedures for handling multiple outcomes or repeated testing. Whether any multiplicity adjustment was planned or used is not reported in the registry record.
In general, multiplicity arises when several outcomes or statistical tests are considered. The more questions examined, the greater the chance that at least one result may appear notable by chance if no adjustment or interpretive strategy is used. This general issue cannot be resolved from the registry entry because no analyses or test results are reported. It would be inappropriate to assume either that adjustment was applied or that no adjustment was planned.
Missing-data rules are also not reported. The record does not specify how missing response assessments, incomplete follow-up, or unavailable survival information were handled. Nor does it report how many participants were included in each outcome analysis. Enrollment is listed as 33, but an enrollment figure does not establish the denominator used for every endpoint.
The analysis populations are not reported in the registry record. It is not stated whether analyses were based on all enrolled participants, participants who began treatment, or another defined group. In general, the chosen population and missing-data handling can affect an estimate, especially when participants have incomplete assessments or follow-up. Without those details and outcome results, the primary and secondary findings cannot be reproduced or fully appraised.
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/Capecitabine | 0 | 33 | 0.0% |
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/Capecitabine | 27 of 33 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cisplatin/Capecitabine |
|---|---|---|
| leukopenia | Blood and lymphatic system disorders | 27 of 33 |
The registry records 0 serious adverse events among 33 at risk in the cisplatin/capecitabine group. It also records 27 other adverse events among 33 at risk. These are the figures listed for the safety fields; they are not a comparison between treatment groups because the study is single group.
The categories should be kept distinct. The serious adverse event entry is not a count of all adverse events, and the other adverse event entry should not be relabeled as serious. The registry does not list serious adverse event terms. It also does not report deaths. These entries do not identify the event types, severity distribution for the other events, timing, duration, or attribution to treatment.
In general, safety counts are interpreted in light of how events were defined, collected, and monitored, as well as the period of observation and the population at risk. Those details are not reported in the registry record. The counts therefore support only a narrow description of what the record lists; they should not be used to infer that no unlisted harms occurred or to compare safety with another regimen.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study was registered to explore cisplatin plus capecitabine in anthracycline- and taxane-pretreated metastatic triple negative breast cancer. It specifies overall response rate at six months as the primary endpoint and lists progression free survival, overall survival, and adverse events as secondary outcomes. The regimen and enrollment figure are also recorded.
It does not report the primary endpoint estimate, the secondary outcome results, or statistical analyses. As a result, readers cannot determine the observed response rate, its precision, or the outcomes for survival from this record. The absence of results is not a result of zero effect and is not evidence of a particular effect in either direction.
The single-group design also places a limit on what the study could establish through direct comparison. It has no separate control group, so it cannot provide a randomized estimate of differences between the combination and another treatment. Descriptive outcomes in a treated group, even if fully reported, would not alone establish that treatment caused those outcomes.
The safety fields list 0 serious adverse events among 33 at risk and 27 other adverse events among 33 at risk. The record does not provide the event terms or additional detail needed to characterize those entries broadly. These figures are not a complete account of every possible safety question.
In general, clinical interpretation of a study result considers the outcome definition, estimate, uncertainty, analysis population, missing data, and design. Several of these elements are not reported in the registry record, and the endpoint estimates themselves are absent. The defensible conclusion is limited to describing the registered question, design, and recorded safety counts. The record does not support a conclusion that the treatment works or does not work.
13. Why This Trial Matters Statistically
This study is statistically important to read as a design-and-reporting record rather than as a comparative treatment estimate. Its single-group phase II structure defines the main interpretive boundary: it can be used to describe outcomes within the treated group, but it does not include a concurrent comparator from which to estimate a randomized difference.
The registry identifies an overall response rate endpoint and a six-month timeframe, which makes the intended primary question clear at a broad level. At the same time, the response criteria, denominator, assessment procedures, estimate, and uncertainty are not reported. These distinctions illustrate why naming an endpoint is not equivalent to reporting its result. A result requires an estimate and enough methodological detail to understand how it was obtained.
The entry also shows why enrollment and analysis populations should not be conflated. Enrollment is listed as 33, while the participants included in each outcome analysis are not specified. The safety fields have their own at-risk denominators, but the primary endpoint denominator is not given. An enrollment figure alone cannot establish that every enrolled participant contributed to every outcome.
Finally, statistical interpretation depends on both design and reporting. Confidence intervals, tests, and analysis populations help readers evaluate precision and robustness, but none is reported for the efficacy outcomes. The record therefore offers a useful example of how registry design details can clarify what a study set out to examine while leaving the magnitude and precision of its findings unknown.
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 most immediate limitation is that no primary endpoint result is reported. The registry identifies overall response rate at six months, but provides no response estimate, denominator, confidence interval, or test result. Results for the listed secondary outcomes are also not reported. Accordingly, this record cannot support an assessment of response magnitude, survival outcomes, or precision.
The single-group design has no control arm. This prevents a direct randomized comparison and limits causal interpretation of any within-group outcome. The record also does not describe eligibility criteria beyond the pretreated metastatic triple negative breast cancer population named in the study description, so the boundaries of the enrolled population cannot be evaluated in detail.
Important endpoint and analysis details are absent. Response criteria, assessment schedule, handling of missing data, outcome-specific analysis populations, and methods for survival analyses are not reported in the registry record. The protocol-based sample-size justification, interim analysis plan, and statistical analysis plan are also not reported.
The safety information is limited to the recorded counts: 0 serious adverse events among 33 at risk and 27 other adverse events among 33 at risk. Serious event terms, deaths, collection procedures, and further event descriptions are not reported in the record. These omissions make detailed characterization of safety entries impossible.
The registry status is UNKNOWN. This status does not itself explain whether follow-up, reporting, or study conduct was completed as planned. The record lists primary completion as 2015-10, but does not report a reason for the status or provide results that would resolve these uncertainties.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Study of Capecitabine and Cisplatin to Treat Metastatic Triple Negative Breast Cancer — NCT01928680.
- Registry record last updated 2015-02-23; status last verified 2015-02.
- Results first posted 2015-02-23.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01928680 is listed as a phase II, single-group study of cisplatin/capecitabine for anthracycline- and taxane-pretreated metastatic triple negative breast cancer. The sponsor is the Chinese Academy of Medical Sciences. The record lists enrollment as 33, a start date of 2012-11, primary completion of 2015-10, and status UNKNOWN.
The regimen description specifies capecitabine 1000mg/m2 orally Bid on day 1 to day 14 plus cisplatin 75mg/m2 on day1 of each 21 day cycle, until progression or untolerable toxicity. The primary endpoint is overall response rate at six months. Secondary outcomes include progression free survival, overall survival, and number and severity of adverse events.
No efficacy endpoint results or analyses are reported in the registry record. The safety fields list 0 serious adverse events among 33 at risk and 27 other adverse events among 33 at risk. The study has no separate comparator group, and the record does not report the statistical analysis plan or outcome-specific analysis populations.