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 A Study of Herceptin (Trastuzumab) in Combination With Xeloda (Capecitabine) in Patients With Metastatic or Recurrent HER2-positive Breast Cancer After First-Line or (Neo)Adjuvant Therapy. (NCT01290718), a non-randomized phase 2 study. The primary outcome is Percentage of Participants With Overall Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01290718 |
| Phase | Phase 2 |
| Condition | Breast Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 4 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
People with HER2-positive metastatic or recurrent breast cancer after prior therapy. The study description focuses on disease refractory to, or relapsing after, chemotherapy with trastuzumab and taxanes, or relapse after (neo)adjuvant trastuzumab and taxanes.
Intervention
Capecitabine (Xeloda) with trastuzumab (Herceptin). The described regimen was capecitabine 900mg/m2 twice daily by mouth on days 1-14 of each 3-week cycle, with trastuzumab 8mg/kg intravenously on day 1 of the first cycle and 6mg/kg intravenously every 3 weeks afterward.
Comparator
There was no comparator group. The study was non-randomized and used a single-group design, so it does not provide a like-for-like control comparison.
Primary question
The primary outcome was the percentage of participants with an overall tumor response, assessed using RECIST Version 1.1. The registry record says these data were not analyzed because of early termination, so it does not report an overall response result.
This question concerns whether tumor response could be assessed in the stated clinical setting while participants received the combination. The design can describe outcomes observed in the treated group if those outcomes are analyzed and reported. Without a control group, it cannot separate outcomes associated with the combination from outcomes that might have occurred under another treatment or without the study regimen. The registry record lists time to disease progression, overall survival, and progression-free survival as secondary outcomes, but it reports no analyses for them. The enrollment was 4, and the record marks the study as terminated. These details set the scope of interpretation: the posted record identifies the intended population, regimen, and outcome, but does not provide an analyzed estimate of response or other efficacy outcomes.
3. Trial Design
This was a Phase 2, non-randomized, open-label, single-group study sponsored by Hoffmann-La Roche. Its title and description concern trastuzumab combined with capecitabine in HER2-positive metastatic or recurrent breast cancer following prior treatment. The registry record lists an enrollment of 4 and a terminated status. The recorded study start is 2011-12 and primary completion is 2012-12.
All participants were assigned to the combination group; there was no randomized allocation and no masking. The treatment description specifies capecitabine 900mg/m2 twice daily orally on days 1-14 of each 3-week cycle. Trastuzumab was described as 8mg/kg intravenously on day 1 of the first cycle, followed by 6mg/kg intravenously every 3 weeks. Treatment was anticipated to continue until disease progression or unacceptable toxicity. The registry record does not report whether all enrolled participants received treatment or how long any participant was treated.
The primary outcome was the percentage of participants with overall response, assessed by RECIST Version 1.1 at baseline and Day 1 of Cycles 4 and 8, then every 3 weeks until unacceptable toxicity or death. The record states that these data were not analyzed due to early termination. It lists time to disease progression, overall survival, and progression-free survival as secondary outcomes, but reports no analyses. The detailed statistical analysis plan, reasons for termination, and any intended decision criteria are not reported in the registry record.
Because this was a single-group study, the design could describe outcomes in the treated participants but could not directly compare the combination with an alternative. Open-label status means masking was absent; the registry record does not describe any additional steps to limit assessment bias. The small enrollment and absence of analyzed primary outcome data sharply limit what can be concluded from the posted 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 | Capecitabine Plus (+) Trastuzumab |
|---|---|
| STARTED | 4 |
| COMPLETED | 0 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Capecitabine Plus (+) Trastuzumab |
|---|---|
| Early termination of the study | 4 |
Analysis population for the primary outcome, as defined in the registry: Data were not analyzed due to early termination of the study.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: All randomized participants
| Characteristic | Unit | Capecitabine + Trastuzumab |
|---|---|---|
| Participants analysed | 4 | |
| Age, Continuous | years | 53.75 (SD 9.03) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Overall Response, measured over Baseline and Day 1 of Cycles 4 and 8 and every 3 weeks until unacceptable toxicity or Death.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the percentage of participants with an overall tumor response. The registry specifies that tumor response was measured using Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1. In general, an overall response endpoint classifies whether a participant's tumor meets the stated response criteria during assessment. Expressing the outcome as a percentage is intended to describe how common response was in the study group, rather than the amount of time until an event.
The assessment schedule began at baseline and included Day 1 of Cycles 4 and 8, followed by assessments every 3 weeks until unacceptable toxicity or death. Repeated assessments can establish whether a response is observed over the specified follow-up, according to the chosen criteria. The registry record does not report further details of the response definitions, confirmation rules, or handling of participants whose assessments were unavailable.
Response is used as a tumor activity endpoint in studies of cancer treatment. It is distinct from time-to-event outcomes such as progression-free survival, which concern how long participants remain without a defined event. A response percentage does not, by itself, describe duration of response, survival, symptoms, or safety. Those questions require their own outcomes and analyses.
In this study, the registry explicitly says the primary response data were not analyzed due to early termination. It therefore reports neither a response percentage nor an estimate of its uncertainty. The endpoint identifies what the study intended to measure, but the posted record does not establish the observed response in the enrolled group.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the primary outcome data were not analyzed because the study terminated early. Although the registry identifies overall response as the primary endpoint and describes RECIST Version 1.1 assessment, it supplies no response count, percentage, effect estimate, or confidence interval. The posted record therefore does not support a numerical conclusion about tumor response in the study group.
The listed enrollment was 4. That figure describes enrollment, not necessarily the number who received treatment, completed assessments, or contributed to any particular outcome. The registry does not report those analysis populations. For the same reason, enrollment alone cannot be used to reconstruct a response percentage or to determine how many participants had evaluable tumor assessments.
The design also limits the kind of inference that would have been possible even if outcomes had been reported. A single-group study has no concurrent control group, so any observed outcome would describe participants in that group rather than estimate a treatment contrast against another option. Non-randomized allocation and open-label conduct are consistent with that limitation. The registry record does not report an adjusted comparison or another analysis that would address it.
Secondary outcomes are named as time to disease progression, overall survival, and progression-free survival. The record reports no analyses for these outcomes. It provides no event counts, time estimates, censoring details, or uncertainty intervals. Naming an outcome does not mean that a result for it is available.
The adverse-event record reports 0 serious adverse events among 2 participants at risk and 0 other adverse events among 2 participants at risk for the listed treatment group. These figures refer to the posted risk sets; they do not establish that all enrolled participants were represented in those sets. The record does not report death counts for that group. These limited entries are not a complete description of safety.
Overall, the registry record documents the intended treatment, study design, outcomes, early termination, and a small set of safety counts. It does not report analyzed efficacy results or enough outcome detail to quantify effects precisely. No conclusion about comparative benefit can be drawn from a study without a control group, and the absence of an analyzed primary result leaves the planned response question unanswered in the posted record.
9. Statistical Methods Explained
What does a single-group study estimate?
As general statistical context, an outcome measured in a single treatment group is a description of that group. For a binary outcome such as response, the basic summary may be a proportion: the number classified as responders relative to the number included in the analysis. The interpretation depends on the definition of response, the analysis population, and whether outcome assessments are available. A comparison between treatment groups requires a comparator; a single-group proportion is not itself a treatment effect relative to another regimen.
For this study, the registry names the response endpoint and the RECIST Version 1.1 criteria, but says the data were not analyzed due to early termination. It does not report the numerator, denominator, estimate, or confidence interval for response. It also does not report which participants, if any, had evaluable assessments. No response calculation can therefore be reconstructed from the registry record.
What would a confidence interval communicate?
As general statistical context, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the analysis. It is not a guarantee that the true value lies within a particular interval, nor does it describe individual participant outcomes. The width of an interval depends on the information in the data and the method used. With no analyzed primary endpoint estimate or interval reported, the precision of the response result cannot be assessed. No interval should be inferred from enrollment alone.
How do time-to-event outcomes differ?
Time to disease progression, overall survival, and progression-free survival are listed as secondary outcomes. In general, time-to-event methods account for both the time until an event and the fact that some participants may not have an observed event before their follow-up ends. Such participants may be censored under a defined analysis approach. The methods, event definitions, follow-up duration, and censoring rules for this study are not reported in the registry record. There are no posted analyses from which to interpret a time-to-event estimate.
As general context, a hazard ratio, if reported, would compare event rates over time under specified modeling assumptions; it would not be the same as a difference in survival percentages at a particular time. No hazard ratio or other time-to-event estimate is reported for this study. This distinction matters because the secondary outcome names alone do not indicate what statistical method was planned or used.
Why does the absence of a comparator matter?
Random assignment to comparison groups can help balance measured and unmeasured characteristics on average, while a control group provides a concurrent reference for outcome comparisons. This study was non-randomized and single-group, so neither a randomized contrast nor a concurrent control estimate is available. General statistical adjustment can sometimes address measured differences, but it cannot automatically remove all confounding, and the registry does not report an adjusted analysis. Any hypothetical outcome estimate from this group would not be a randomized comparative effect.
What do the planning assumptions mean?
The registry describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect, and that time-to-event planning uses a total event count while other planning uses participants per arm. These are planning conventions, not evidence that the study itself used two randomized arms. The actual study is described as single-group and non-randomized.
Planning calculations address how much information a hypothetical design might require under specified assumptions. They are not observed study outcomes, and a sample-size illustration is not observed power. The registry record does not report the actual effect or planning values needed to interpret a particular illustration. Given that the primary data were not analyzed, no observed primary response effect is available in the record to assess against a planning target.
What can be learned from the posted analysis status?
The record lists no analyses and explicitly states that the primary data were not analyzed due to early termination. This is different from a reported estimate that is imprecise or statistically inconclusive: no primary estimate is posted to evaluate. The statistical analysis plan, rules for missing observations, analysis populations, and any interim analyses are not reported in the registry record. Without them, readers cannot assess whether outcome handling followed a prespecified plan or determine how unobserved assessments would have affected an analysis.
These distinctions separate the intended methods from the available evidence. The endpoint and assessment schedule describe what investigators planned to measure. The terminated status and lack of analyses describe what the registry record makes available. Statistical interpretation must rest on the latter when judging whether an effect estimate, its precision, or a comparison can be supported.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome, percentage of participants with overall response, and secondary outcomes of time to disease progression, overall survival, and progression-free survival. It does not report a multiplicity strategy, such as how interpretation would account for testing more than one outcome or making repeated comparisons. Any prespecified hierarchy, adjustment procedure, or threshold for declaring results notable is not reported in the registry record.
The primary response data were not analyzed due to early termination, and the record lists no analyses. As a result, there are no posted estimates to assess across the primary and secondary outcomes, and no reported analysis set for any endpoint. The enrollment of 4 should not be treated as the denominator for every outcome: the number who received treatment, had evaluable tumor assessments, or contributed follow-up is not reported.
Missing-data methods are also not reported in the registry record. It does not describe how unavailable response assessments, incomplete follow-up, treatment discontinuation, or participants lost to observation would have been handled. These details matter because an analysis can depend on whether missing outcomes are excluded, imputed, or addressed through another method. No such approach should be assumed.
The registry identifies assessment times for the primary endpoint and describes treatment as continuing until progression or unacceptable toxicity, but it does not provide participant-level follow-up or explain how assessments after discontinuation would be handled. It also does not report analysis populations such as all enrolled participants or participants with evaluable outcomes. Consequently, neither the scope of any intended analysis nor the effect of missing information can be established from the posted 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 |
|---|---|---|---|
| Capecitabine + Trastuzumab | 0 | 2 | 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 |
|---|---|
| Capecitabine + Trastuzumab | 0 of 2 |
The registry reports a Capecitabine + Trastuzumab group with 2 participants at risk for serious adverse events and 0 affected. It separately lists 2 participants at risk for other adverse events and 0 affected. These are counts, not percentages; no percentage or rate is stated in the record. They describe the listed risk sets and should not automatically be read as covering every enrolled participant.
The deaths field is null, so a death count is not reported in the registry record. No serious adverse-event terms are listed. The figures therefore do not provide event descriptions, timing, duration, severity breakdowns, or information about treatment relatedness. The registry also does not describe how adverse events were collected or the period during which participants were monitored for them.
Zero events in the posted counts means no affected participants were recorded in those particular entries. It does not establish that the regimen has no safety risks or that no events occurred outside the reported risk sets or reporting period. With limited enrollment and sparse safety detail, these entries cannot characterize the full safety profile. The record's counts should be read narrowly, alongside its missing death count and lack of broader event information.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase 2, open-label, non-randomized, single-group study was intended to examine trastuzumab plus capecitabine in HER2-positive metastatic or recurrent breast cancer after prior therapy. It identifies overall response by RECIST Version 1.1 as the primary outcome and names several secondary outcomes. These details define the clinical question and planned measurements.
The record does not report an analyzed primary response result. It says the data were not analyzed because the study terminated early. It also lists no analyses for the secondary outcomes. Accordingly, the posted record does not establish the response percentage, provide an uncertainty estimate, or show a time-to-event result. The absence of an analysis is not evidence of no response or no change; it means the outcome question is unanswered by the reported data.
The single-group design means the study would not, on its own, establish how outcomes compare with another treatment. Even if a response estimate had been reported, it would describe participants in the study group rather than isolate the effect of the combination against a concurrent control. The registry contains no randomized comparison or comparator group.
The safety counts are also limited. The record lists 0 serious and 0 other adverse events among 2 participants at risk in each category, while a death count is not reported. Those counts do not provide a complete safety account or show that adverse events could not occur.
The registry can therefore be used to understand the study's intent, design, treatment description, status, and limited reported safety entries. It cannot be used to claim a measured response rate, quantify treatment effects, establish comparative efficacy, or draw a complete safety conclusion. The reasons for early termination and detailed analysis procedures are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically instructive because its intended question and available results are distinct. The registry specifies a primary endpoint, an assessment framework, and an assessment schedule, but states that the primary data were not analyzed due to early termination. A clearly defined endpoint does not by itself produce an estimate: readers need analyzed outcome data, an analysis population, and information about uncertainty to interpret the result.
The design also illustrates the limits of single-group evidence. A single treatment group can describe outcomes among its participants, but it cannot provide a direct concurrent comparison. Randomization and a comparator address a different question from simply observing what happened in a treated group. This distinction remains important even when a study names a clinically relevant endpoint.
The enrollment of 4 and the safety risk sets of 2 underline why denominators and analysis populations matter. Enrollment is not interchangeable with the number assessed for an endpoint or followed for safety. The record does not report those populations or explain how incomplete observations were handled. Without that information, readers cannot determine which participants contributed to each potential estimate.
The record also separates outcomes from planning. Its stated planning assumptions describe an illustration, not the design actually used and not observed power. Since no primary outcome analysis is reported, planning language cannot substitute for an observed effect or its precision.
More broadly, this example shows why registry entries should be read for both the intended methods and the results actually posted. Here, the record documents a terminated study and limited safety counts, but not analyzed efficacy outcomes. That distinction helps prevent an unreported result from being mistaken for a negative result, and a small set of event counts from being mistaken for a complete safety assessment.
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 consequential limitation is that early termination left the primary response data unanalyzed, according to the registry record. No response estimate or confidence interval is posted, so the primary endpoint cannot be interpreted quantitatively. The record also lists no analyses for the secondary outcomes.
Enrollment was 4, but the number of participants included in each outcome assessment is not reported. The registry does not state how many received treatment, had post-baseline tumor assessments, completed follow-up, or discontinued treatment. This prevents readers from identifying the relevant denominators for efficacy outcomes or the extent of follow-up.
The study had no comparator and was non-randomized and open-label. Thus, even an analyzed outcome would not constitute a randomized comparison with another treatment. The record does not report whether independent response review was used, how response assessments were confirmed, or how missing assessments were to be handled.
The reasons for termination, detailed statistical analysis plan, sample-size justification, interim analyses, multiplicity approach, and analysis populations are not reported in the registry record. Although planning assumptions are described, the specific planning values and their interpretation are not available in the listed details. The registry also does not provide participant-level outcome data or follow-up details.
Safety reporting is limited to the listed risk sets and event counts. A death count is not reported, and no serious adverse-event terms are listed. The record does not give event timing, severity detail, attribution, or monitoring duration. These gaps mean that both efficacy and safety remain incompletely characterized by the posted information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Herceptin (Trastuzumab) in Combination With Xeloda (Capecitabine) in Patients With Metastatic or Recurrent HER2-positive Breast Cancer After First-Line or (Neo)Adjuvant Therapy. — NCT01290718.
- Registry record last updated 2014-12-17; status last verified 2014-12.
- Results first posted 2014-12-17.
- 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
NCT01290718 is a terminated Phase 2 study sponsored by Hoffmann-La Roche. It was open-label, non-randomized, and single-group, with an enrollment of 4. The registry describes trastuzumab plus capecitabine for HER2-positive metastatic or recurrent breast cancer following prior therapy.
The primary outcome was percentage of participants with overall response by RECIST Version 1.1, assessed from baseline at specified treatment-cycle visits and then every 3 weeks. The registry states that primary data were not analyzed due to early termination. Time to disease progression, overall survival, and progression-free survival were listed as secondary outcomes; the record reports no analyses.
For the listed treatment group, 0 of 2 participants at risk were affected by serious adverse events, and 0 of 2 were affected by other adverse events. A death count is not reported. The registry does not provide an analyzed efficacy estimate, a comparator, or a complete safety account.