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 2 Study of Trastuzumab and Etoposide for Her2 Positive Breast Cancer (NCT00810017), a na phase 2 study. The primary outcome is To Determine ORR of Trastuzumab Combined With Etoposide in Patients With HER2 Positive Metastatic Breast Cancer, and to Assess Toxicity of the Combination of Trastuzumab With Intravenous Etoposide..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00810017 |
| Phase | Phase 2 |
| Condition | HER-2 Positive Metastatic Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Medstar Health Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
Women and men with previously treated HER2-positive metastatic breast cancer were the intended population. The registry lists the condition as HER-2 Positive Metastatic Breast Cancer. The primary outcome description refers to patients with HER2-positive metastatic breast cancer. The record lists enrollment as 2, but its primary outcome population statement says the study was terminated and no results were collected; it therefore does not provide outcome data for evaluating response or toxicity.
Intervention
The planned treatment combined intravenous etoposide and trastuzumab. The arm description specifies etoposide 100mg/m2 daily for 3 days every 3 weeks, with trastuzumab given as an 8mg/kg loading dose followed by 6mg/kg. After the combination treatment, the plan was to continue trastuzumab alone until disease progression. The brief summary describes the combination for 6 cycles, followed by trastuzumab alone until worsening of disease.
Comparator
There was no separate comparator group. The registry classifies the study as single group, with allocation listed as NA and masking listed as NONE. Thus, the clinical question is not a randomized comparison of the combination against another treatment or a control condition. Any treatment response observed in a single-group study would not, by itself, establish how outcomes compare with an alternative.
Primary question
The stated primary objective was to determine the overall response rate (ORR) for trastuzumab combined with etoposide in HER2-positive metastatic breast cancer and to assess toxicity of the combination. The outcome timeframe describes best overall response from the start of first treatment until the first progression or death from any cause, assessed up to 24 months. The registry record says no results were collected, so it does not answer whether participants responded or what toxicities occurred.
3. Trial Design
NCT00810017 is listed as a Phase 2, single-group study sponsored by Medstar Health Research Institute. Its title describes trastuzumab and etoposide for HER2-positive breast cancer; the official title specifies HER2-positive metastatic breast cancer. The registry classifies allocation as NA and masking as NONE. There was no randomized allocation to distinct treatment groups, and the record describes a single arm rather than a like-for-like control comparison.
The intended participants were women and men with previously treated metastatic HER2-positive breast cancer. The registry lists enrollment as 2. The study status is TERMINATED, and the primary outcome population statement says the study was terminated and no results were collected. The reason for termination is not reported in the registry record, and the record does not establish which planned procedures or treatments, if any, were completed.
The planned regimen was etoposide 100mg/m2 daily for 3 days every 3 weeks, together with trastuzumab at an 8mg/kg loading dose followed by 6mg/kg. The brief summary describes this combination during 6 cycles, with trastuzumab alone afterward until worsening of disease. The arm description says trastuzumab alone would continue until disease progression. The record also describes laboratory evaluation of previously collected surgical tissue for Top2A, in the context of possible genetic differences related to drug response. Whether that testing was performed is not reported in the registry record.
The registry lists a start date of 2009-02 and a primary completion date of 2010-05. It does not report a comparator, a masking procedure, or a completed treatment comparison. The intended primary objective combined an efficacy question about ORR with an assessment of toxicity. Because no results were collected, the design information describes the planned investigation rather than evidence about response, safety, or treatment effects.
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 | Single Arm Study |
|---|---|
| STARTED | 2 |
| COMPLETED | 0 |
| NOT COMPLETED | 2 |
Analysis population for the primary outcome, as defined in the registry: Study was terminated and no results were collected.
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: Study was terminated due to difficulties enrolling
| Characteristic | Unit | Single Arm Study |
|---|---|---|
| Participants analysed | 2 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as To Determine ORR of Trastuzumab Combined With Etoposide in Patients With HER2 Positive Metastatic Breast Cancer, and to Assess Toxicity of the Combination of Trastuzumab With Intravenous Etoposide., measured over The best overall response is the best response recorded from the start of first treatment until the date of first progression or date of death from any cause whichever came first, assessed up to 24 months.
| 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 outcome is described as determining the overall response rate (ORR) for trastuzumab combined with etoposide and assessing toxicity of the combination. The outcome timeframe defines best overall response as the best response recorded from the start of first treatment until the first progression or death from any cause, assessed up to 24 months. The registry record does not specify the response categories, the criteria used to classify a response, or a numerical result.
In general, ORR is used to describe the fraction of an evaluated group whose disease meets prespecified criteria for response. It can provide a concise measure of antitumor activity in a clinical study, but its interpretation depends on clear response definitions, consistent assessment, and an identified analysis population. Those details are not reported in the registry record. A response-rate endpoint also does not by itself describe how long a response lasts or whether outcomes differ from those under another treatment.
The toxicity objective addresses harms associated with the combination, but the record does not give a defined toxicity measure or a reported toxicity result. The endpoint is therefore a planned efficacy and safety assessment, not a result that can be estimated from the posted information. The registry states that no results were collected.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a planned Phase 2 investigation of trastuzumab with etoposide in previously treated HER2-positive metastatic breast cancer. Its primary objective was to determine ORR and assess toxicity. However, the primary outcome population statement explicitly says the study was terminated and no results were collected. There is consequently no posted estimate of response, no uncertainty interval around an effect, and no reported toxicity estimate to interpret.
The study is classified as single group, with allocation listed as NA and masking listed as NONE. It does not contain a concurrent control group for comparing the combination with another treatment. Even if a response result had been posted, a single-group result would describe outcomes observed under the study regimen; it would not by itself isolate the regimen's effect from other explanations or establish comparative benefit. In this record, the absence of collected results means even a descriptive response estimate is unavailable.
The registry lists enrollment as 2. That entry does not supply an outcome denominator or show that participants contributed evaluable response or toxicity data. The primary outcome statement instead reports that no results were collected. These statements should not be conflated: a listed enrollment value is not a substitute for an analyzed population, and it cannot support a response-rate calculation in the absence of outcome counts.
The endpoint timeframe refers to best response from treatment initiation until first progression or death, assessed up to 24 months. This describes the intended observation window; it is not evidence that follow-up reached that point. The record does not report actual follow-up, response assessments, event counts, or the circumstances of termination. The reason the study stopped is also not reported in the registry record.
Accordingly, the posted information supports conclusions about the study's stated question, planned regimen, single-group design, and termination status. It does not support conclusions about the frequency of response, the precision of an efficacy estimate, the occurrence or severity of toxicity, or whether the regimen is better or worse than an alternative. The absence of results is central to interpretation, rather than a finding that the treatment had no effect or caused no harms.
9. Statistical Methods Explained
The registry classifies this investigation as a single-group Phase 2 study, rather than a randomized comparison. That design distinction matters because the question addressed by a single-group study is mainly descriptive: what outcomes were observed among participants who received the planned regimen? A randomized controlled comparison instead evaluates differences between assigned groups. There is no randomized contrast in this record, and allocation is listed as NA. Masking is listed as NONE.
What can an ORR estimate describe?
In general, an overall response rate is a proportion: the number of participants meeting a response definition relative to the number evaluated under the stated analysis rules. The value depends on what counts as response and who is included in the denominator. A complete analysis description would explain response criteria, assessment timing, handling of participants without an evaluable assessment, and whether responses required confirmation. These details are not reported in the registry record. More importantly, the record states that no results were collected, so there is no response count or evaluated denominator from which to estimate ORR.
A response rate is also not the same as duration of response, time to progression, or survival. The registry gives a timeframe for best overall response that runs from the start of first treatment until first progression or death from any cause, assessed up to 24 months. That timeframe identifies the intended window for determining best response. It does not provide the number of participants who responded, the time any response lasted, or observed progression or death data. The registry lists duration of response among its secondary outcomes, but does not report an estimate.
Why does the single-group design limit comparison?
In general, a concurrent control group helps distinguish a treatment-associated outcome from what might have occurred under another treatment or condition. Without such a group, comparisons with outside populations may be affected by differences in eligibility, disease characteristics, follow-up, and outcome assessment. Those concerns are general design considerations, not claims about what happened to participants in this study. Since this record reports no outcomes, it does not support even a descriptive estimate of response under the regimen, much less a causal or comparative effect.
The registry's planning assumptions describe an illustrative sample-size calculation based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The assumptions state that the effect to detect is a stated fraction of the observed effect. They are planning illustrations, not the trial's own design or observed power. The record does not report an observed treatment effect, and no trial results were collected, so those assumptions cannot be treated as evidence of efficacy or as a justification for the enrollment listed. In general, a significance level sets a threshold for a specified testing procedure; it does not measure the probability that a result is true or the size of a treatment effect.
For general sample-size planning, calculations depend on the outcome type, the target effect, variability or event assumptions, the testing approach, and the design. The record's assumptions distinguish time-to-event illustrations, which use a total event count, from other illustrations, which express participants per arm. Such outputs are planning quantities, not observed data and not proof that a study had adequate power. No numerical planning estimates or study-specific statistical justification are reported in the registry record.
What does uncertainty mean?
In general, a confidence interval describes a range of effect estimates compatible with data under a stated statistical procedure. Its width reflects the information in the data and the method used. A narrow interval generally indicates greater precision than a wide interval, although precision does not resolve design limitations or establish causality. Here, there is no posted primary outcome estimate or interval to interpret. The registry record does not report a confidence-interval method or a statistical model.
Likewise, a p-value summarizes how incompatible the observed data are with a specified null model, under the assumptions of a test. It is not an effect-size measure, and it cannot be inferred from a study objective or design label. No primary outcome analysis or p-value is reported in the registry record. The planning significance level is an assumption for an illustrative calculation; it is not a trial result.
For count outcomes, general methods sometimes account for over-dispersion, when observed variability exceeds the level expected under a simpler probability model. Choosing such a model requires outcome data and a defined analysis plan. The registry record does not report an over-dispersion assessment, a model, or analysis details. More generally, the record does not report the statistical methods used for any completed outcome analysis. The available design and endpoint descriptions therefore establish what investigators intended to assess, but cannot be used to reconstruct analyses or results that are not reported.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 5 outcome measures and repeats duration of response among the listed secondary outcomes. The primary objective names both ORR and toxicity, while the primary outcome description emphasizes determining ORR. A registry listing alone does not establish how investigators prioritized tests or interpreted several outcomes together. A multiplicity plan, including any adjustment or hierarchy for multiple statistical tests, is not reported in the registry record.
In general, when several outcomes or hypotheses are tested, the chance of at least one apparently unusual finding can increase if each is assessed separately without accounting for the family of tests. Multiplicity methods can control or describe that issue, but the appropriate approach depends on the planned questions and analysis strategy. Here, no outcome results or analysis plan are reported, so there is no basis to assess the consequences of multiple testing or to identify a prespecified primary comparison beyond the registry's stated primary objective.
The registry says the study was terminated and no results were collected. It does not report missing-data counts, reasons for missing measurements, or procedures for handling missing outcomes. It also does not identify an analysis population, such as all enrolled participants or participants with evaluable response assessments. The enrollment entry is not enough to determine an analysis denominator.
In general, missing outcome data can affect both the size and interpretation of an estimate, particularly when the reasons for missingness are related to a participant's outcome or treatment experience. Methods for addressing missing data depend on what is missing and why. No such assessment or method is reported in the registry record. With no collected results and no analysis population described, neither missing-data bias nor the robustness of any estimate can be evaluated. The absence of these details should not be interpreted as evidence that outcomes were complete or that missingness was handled in a particular way.
11. What the Result Does, and Does Not, Mean
The record establishes that investigators planned to study trastuzumab combined with etoposide in previously treated HER2-positive metastatic breast cancer, with ORR and toxicity as the central questions. It describes a single-group design, the intended treatment schedule, and a timeframe for best overall response. It also records that the study was terminated and no results were collected.
These registry entries do not show that the treatment produced a response, failed to produce a response, or caused or avoided toxicity. No response estimate, toxicity result, confidence interval, or statistical test is available in the registry record. In particular, the absence of results is not a zero response rate and is not evidence of an absence of adverse events. The registry status and outcome statement describe the reporting record, not the clinical effect of the regimen.
The listed enrollment value does not establish how many participants had an evaluable outcome or were included in an analysis. It should not be used as a denominator for an ORR calculation because the record does not report response counts and explicitly states that no results were collected. The reason for termination, actual treatment exposure, outcome-assessment completion, and follow-up are not reported in the registry record.
The design also does not support a direct comparison with another treatment. As a single-group study, it lacks a randomized control group. General limitations of that design would apply even if outcomes were available; in this registry record, the lack of results further prevents descriptive assessment. The planned timeframe of up to 24 months is not proof that participants were followed for that period.
The appropriate interpretation is therefore narrow: the registry describes the intended study question and design, but supplies no empirical answer to the primary question. It cannot establish efficacy, lack of efficacy, comparative effectiveness, or a safety profile. Nor can planning assumptions be read as observed power or as outcome evidence. Any conclusion about treatment effects would go beyond what the registry record reports.
12. Why This Trial Matters Statistically
This study is statistically informative as an example of the difference between a registered research question and an analyzable result. The registry gives a specific primary objective: estimate ORR for a combination regimen and assess toxicity. It also gives a single-group design and a planned response timeframe. Those details clarify what investigators intended to learn, but the record states that the study was terminated and no results were collected. Without outcome observations, an estimate and its uncertainty cannot be calculated or interpreted.
The distinction between enrollment and analysis is also important. The registry lists enrollment as 2, but does not report an outcome denominator, evaluable population, or response data. In general, a registered enrollment figure does not automatically indicate that every listed participant completed treatment, had an outcome assessment, or entered a particular analysis. The record does not supply those details, and the enrollment value cannot stand in for them.
The single-group design highlights another general statistical principle: a within-study outcome description is different from a controlled treatment comparison. A control group can support estimation of a contrast under the trial's design, while a single group has no concurrent comparison. This trial record does not report results for either kind of inference. Its classification is nevertheless essential for understanding what the planned study could have addressed.
Finally, the registry's planning assumptions should be kept separate from empirical evidence. A significance level, allocation assumption, or normal approximation belongs to a planning calculation when used for that purpose; it is not a treatment result, an observed power calculation, or proof that a completed analysis was conducted. The absence of posted results makes this distinction especially important. The record matters statistically because it shows how design information can be described while the outcome evidence needed to answer the clinical question remains unavailable.
13. 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 central trial-specific limitation is that the registry reports termination and no collected results. There is no posted ORR, toxicity estimate, response count, or uncertainty measure. The reason for termination is not reported in the registry record, so the status cannot be attributed to a particular operational, safety, efficacy, or administrative cause.
The study is listed as single group, with no randomized comparator. As a result, the design does not provide a controlled treatment contrast. The registry also lists allocation as NA and masking as NONE. These design features should be kept distinct from the lack of results: even a completed single-group study would have limitations for comparative inference, while this record additionally lacks outcome data for descriptive assessment.
The enrollment entry is 2, but the record does not identify an analysis population or a denominator for the primary outcome. It does not say whether participants received the planned regimen, completed the planned cycles, had response assessments, or contributed toxicity data. The stated timeframe of up to 24 months is an endpoint description, not a report of actual follow-up. These details are not reported in the registry record.
The registry does not specify response criteria, toxicity definitions, a statistical analysis plan, missing-data procedures, multiplicity handling, or methods for assessing response. It lists duration of response as a secondary outcome but gives no result. The record also describes planned tissue testing for Top2A; whether testing occurred or how it was to be analyzed is not reported in the registry record.
These limitations prevent estimates of treatment response, precision, safety, or comparative effect. They also prevent assessment of whether the planned endpoint definitions and analysis choices were implemented. The registry supports description of the intended study and its status, but not conclusions about outcomes.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Phase 2 Study of Trastuzumab and Etoposide for Her2 Positive Breast Cancer — NCT00810017.
- Registry record last updated 2022-05-19; status last verified 2022-04.
- Results first posted 2022-05-19.
- 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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17. Record Summary
NCT00810017 is a Phase 2, single-group study sponsored by Medstar Health Research Institute. It planned to combine trastuzumab with etoposide for previously treated HER2-positive metastatic breast cancer. The primary objective was to determine ORR and assess toxicity. The planned regimen used etoposide and trastuzumab, followed by trastuzumab alone until disease progression or worsening of disease.
The registry lists enrollment as 2, a start date of 2009-02, and a primary completion date of 2010-05. The study status is TERMINATED. The primary outcome population statement says no results were collected. No response or toxicity estimate, analysis population, or statistical analysis is reported in the registry record. Because the study is single group and has no comparator, it was not a randomized treatment comparison.