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 Trastuzumab, Docetaxel, and Carboplatin in Treating Women With Stage II, Stage III, or Inflammatory Breast Cancer (NCT00118053), a na phase 2 study. The primary outcome is Antitumor Activity as Measured by Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00118053 |
| 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 | University of Medicine and Dentistry of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
Women with stage II, stage III, or inflammatory breast cancer were the intended population. Breast cancer is the condition listed in the registry record. Enrollment is listed as 5. The record does not report further eligibility details, such as age criteria or the clinical characteristics required for participation.
Intervention
The trial studied trastuzumab, docetaxel, and carboplatin. The arm description specifies six cycles of TCH administered every 3 weeks, followed by restaging 3 weeks after the sixth cycle. It gives docetaxel as 75 mg/m2, carboplatin as AUC = 6, and trastuzumab as 2 mg/kg weekly after a 4 mg/kg load on Day 1. Conventional surgery and radiation therapy are also listed as interventions, but the available arm description does not provide their full use or timing.
Comparator
There was no separate comparator group. The registry describes a single-group study, with allocation listed as NA and masking as none. Consequently, the trial does not make a randomized, like-for-like comparison between the regimen and another treatment or control condition.
Primary question
The primary outcome was antitumor activity measured by response rate, with a timeframe of 5 years. The registry record states that early termination left insufficient data to evaluate this outcome. It does not report the response-rate result, the criteria used to define response, or a statistical estimate of response. The study question can therefore be described, but the posted registry information does not answer it with an evaluable outcome.
3. Trial Design
This was a phase II, single-group trial sponsored by the University of Medicine and Dentistry of New Jersey. The registry lists the condition as breast cancer and describes the intended population as women with stage II, stage III, or inflammatory breast cancer. Enrollment is listed as 5. The study started in 2005-04, had a primary completion date of 2008-12, and is marked terminated.
The intervention group was named Docetaxel, Carboplatin and Trastuzumab. Its description specifies six cycles of TCH every 3 weeks and restaging 3 weeks after the sixth cycle. The regimen details listed are docetaxel 75 mg/m2, carboplatin AUC = 6, and trastuzumab 2 mg/kg weekly after a 4 mg/kg load on Day 1. Conventional surgery and radiation therapy appear in the intervention list as well. The available description does not fully specify how those procedures were incorporated for participants, so their role should not be inferred from the list alone.
Allocation is listed as NA, masking as none, and the model as single group. This means the registry does not describe random assignment or a masked comparison. It is not a comparative trial with a control arm, so any outcome observed within the group would not by itself establish how outcomes would differ under another treatment. The registry also lists pathological complete response, disease-free survival, and pathologic and molecular markers for predicting efficacy as secondary outcomes.
The record lists four outcome measures and zero analyses. It does not report a sample-size justification, a detailed statistical analysis plan, or interim analyses. It states that the trial was terminated early and that insufficient data were collected to evaluate the primary response-rate outcome. These details set limits on what can be concluded from the registry results.
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 | Docetaxel, Carboplatin and Trastuzumab |
|---|---|
| STARTED | 5 |
| COMPLETED | 5 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Study was terminated early and insufficient data were collected to evaluate this outcome measure.
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 | Docetaxel, Carboplatin and Trastuzumab |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age Continuous | years | 46.4 (SD 9.9) |
| 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 Antitumor Activity as Measured by Response Rate, measured over 5 years.
| 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 is titled antitumor activity as measured by response rate, with a timeframe of 5 years. In general, a response-rate endpoint counts how many participants meet a prespecified definition of tumor response during a stated assessment period. It is used to describe whether measurable disease changes according to those criteria. The criteria, assessment schedule, and precise response-rate definition are not reported in the registry record, so the endpoint cannot be interpreted more specifically from the posted information.
A response rate is a proportion-based outcome. In general, its estimate depends on both the number of participants who meet the response definition and the number assessed under the endpoint rules. The registry record does not provide either an evaluable response count or an estimate. Instead, it explicitly says the study was terminated early and insufficient data were collected to evaluate this outcome measure.
That statement means there is no posted primary response-rate result to interpret for this trial. The stated timeframe does not establish that participants were followed for the full period or that a response assessment was completed for each participant. The actual assessment duration and the handling of participants without an assessment are not reported in the registry record. The endpoint identifies what the study intended to measure; it does not supply evidence about the size or precision of a response rate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the primary outcome was not evaluable in the registry record. The primary endpoint was antitumor activity measured by response rate over a timeframe of 5 years, but the record states that the study was terminated early and insufficient data were collected to evaluate it. No response estimate, confidence interval, or primary-outcome analysis is reported. The posted information therefore does not quantify response in this study.
The registry lists enrollment as 5. A small enrolled group can make estimates imprecise even when an outcome is measured, because each participant's result can have a substantial influence on the group summary. In this case, however, the main limitation is more fundamental: the primary outcome itself is reported as unevaluable. There is no basis in the record for reconstructing a response estimate or its uncertainty.
The single-group design also affects what could be inferred even if an outcome estimate were available. In general, an outcome observed in one group describes that group under the study conditions. Without a comparator, the result cannot establish how outcomes differ from an alternative treatment or control group. The registry lists no random allocation and no masking. It is therefore not a randomized comparison, and causal attribution to the regimen would require more than a within-group outcome description.
The safety information is limited but not absent. In the Docetaxel, Carboplatin and Trastuzumab group, 1 participant is listed as affected by a serious adverse event and 4 as affected by other adverse events, with 5 participants at risk in each category. Diarrhea and febrile neutropenia are each listed among the top serious terms at 20.0%. These entries describe reported safety categories; they do not replace the missing efficacy endpoint. The record does not report a death count.
The registry lists pathological complete response, disease-free survival, and pathologic and molecular markers for predicting efficacy as secondary outcomes, but does not provide outcome estimates for them in the posted information. It lists four outcome measures and zero analyses. The definitions, analysis populations, missing-data procedures, and statistical methods for outcome evaluation are not reported in the registry record. Thus, the posted evidence supports a description of the study design, early termination, enrollment, and selected safety counts, but not an efficacy conclusion.
Any numerical planning illustration based on an observed effect should be understood as a planning exercise, not as observed power or a substitute for a trial result. Here, the registry does not report an evaluable primary effect to interpret. More broadly, an illustration based on assumed effect sizes and design assumptions would not change the fact that this trial has no reported primary response-rate analysis. Precision and evidential strength must be judged from observed outcome data and their analysis, neither of which is available for the primary endpoint in this record.
9. Statistical Methods Explained
Statistical interpretation starts with identifying the design and the outcome actually reported. This record describes a phase II, single-group study, lists enrollment as 5, and marks the trial terminated. The primary outcome is response rate, but the record says that early termination left insufficient data to evaluate it. The registry lists zero analyses. Because no primary result is posted, statistical methods can explain what would ordinarily be needed to interpret such an outcome, but they cannot recover a trial estimate that the record does not contain.
What does a response rate measure?
In general, response rate is a proportion: the numerator consists of participants who satisfy the trial's response definition, and the denominator consists of participants included under the endpoint's analysis rules. The result depends on how response is defined, when it is assessed, and which participants count in the denominator. Those details are essential because different definitions or analysis populations can produce different estimates. The registry record names the response-rate endpoint and gives a timeframe of 5 years, but the response criteria, assessment schedule, denominator rules, and result are not reported.
A proportion estimate is usually interpreted alongside its uncertainty. In general, a confidence interval describes a range of values compatible with the data under a specified statistical method. A narrow interval indicates greater precision, while a wide interval indicates less precision. An interval does not remove design limitations, and it cannot be calculated meaningfully without the relevant outcome count and denominator. Neither a primary response estimate nor an interval is reported in this record.
Why does the single-group design matter?
In general, a single-group study can describe outcomes among participants receiving the studied intervention, but it does not directly estimate a contrast against a concurrent control group. The registry lists one group, allocation as NA, and masking as none. Without a comparator, differences that might arise from participant characteristics, disease course, assessment practices, or other care cannot be separated through a randomized between-group analysis. This limitation applies even when an outcome is fully observed; here, the primary endpoint is additionally unevaluable.
Randomization is useful in comparative studies because it aims to balance known and unknown participant characteristics across groups. Masking can reduce some forms of influence in outcome assessment or other trial processes. These are general design concepts, not claims that the registry specifies a particular unreported procedure. This study is recorded as unmasked and single-group, so an interpretation should not imply a randomized or masked comparison.
What can precision mean with limited enrollment?
In general, estimates based on fewer observations tend to be less precise than estimates based on more observations, all else being equal. With enrollment listed as 5, any complete-case summary could be sensitive to individual outcomes. That is a general statistical concern, not a recalculation or a claim about an unreported result. The primary obstacle remains that the registry says insufficient data were collected to evaluate the response-rate endpoint. No estimate can be made more precise by describing a method when its necessary outcome data are unavailable.
Confidence intervals, hypothesis tests, and effect estimates answer different questions. An estimate describes the observed magnitude; an interval conveys uncertainty under a method; and a hypothesis test evaluates compatibility with a specified null model. None is reported for the primary outcome. A p-value cannot stand in for an effect estimate, and a planning calculation cannot stand in for observed data. The registry does not report a hypothesis test or a detailed inferential procedure for the response endpoint.
What do sample-size illustrations mean?
In general, a sample-size calculation asks how much information a planned study might need under stated assumptions. It depends on choices such as the target effect, significance level, power, allocation, outcome variability, and analysis method. Such a calculation is a planning illustration, not an observed trial finding and not observed power. The registry record does not report the trial's own sample-size justification, and the posted primary outcome was not evaluable. Any planning illustration must therefore remain distinct from an analysis of what happened in this study.
For outcomes that are counts or proportions, the assumptions used for planning should match the intended endpoint and design. For time-to-event outcomes, planning may depend on event counts and follow-up assumptions. These are general considerations; the registry does not report a detailed statistical plan that would establish which methods were chosen for its secondary outcomes. The record's sample-size assumptions describe an illustration based on an observed effect, but the primary result itself is stated to be insufficient for evaluation. The illustration should not be read as evidence that the study achieved a particular power or detected an effect.
Why do analysis populations and missing outcomes matter?
In general, an analysis population defines which participants contribute to an endpoint estimate. Possible approaches can include analyzing participants according to their original assignment, according to treatment received, or according to whether outcome data are available. The choice needs to be prespecified and clearly described because exclusions can affect interpretation. The registry does not report the analysis population used for the primary endpoint, and the lack of an evaluable result prevents assessing how participants were handled.
Missing outcome data can create both reduced precision and potential bias. If participants without an assessment differ systematically from those assessed, a summary among the assessed participants may not represent the intended study population. Appropriate methods depend on why data are missing and on the endpoint and design. The registry record does not report reasons for missing primary outcome data, a missing-data method, or an imputation analysis. The explicit statement that insufficient data were collected is not enough to determine the pattern or cause of missingness.
Overall, the appropriate statistical reading is bounded: the design and intended endpoint are identifiable, but the primary endpoint analysis is not available. General statistical methods clarify what an estimate would mean and what information is needed to assess it; they do not supply a missing effect, confidence interval, or test result.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures. The named secondary outcomes are pathological complete response, disease-free survival, and pathologic and molecular markers for predicting efficacy. The primary outcome is antitumor activity measured by response rate. The record lists zero analyses and does not provide outcome estimates for the secondary measures in the posted information.
Multiplicity is the general issue that arises when a study evaluates several outcomes, time points, subgroups, or statistical tests. If many hypotheses are tested, the chance of at least one apparently notable result can increase unless the analysis plan accounts for the number and relationship of tests. Methods for addressing multiplicity can include prespecifying a hierarchy of outcomes or adjusting inference, but the appropriate approach depends on the design and aims. The registry record does not report a multiplicity strategy, a hierarchy beyond naming the primary outcome, or a detailed statistical analysis plan. It is not possible to determine how the listed secondary outcomes were intended to be analyzed or interpreted together.
The primary endpoint is explicitly described as unevaluable because the trial was terminated early and insufficient data were collected. Reasons for termination, the timing of termination relative to outcome assessments, and any interim analyses are not reported in the registry record. Missing-data rules and analysis populations are also not reported. These omissions mean the record cannot establish whether secondary analyses were performed, how missing outcome information was handled, or whether any adjustment for multiple comparisons was planned. The absence of these details should be treated as uncertainty in the reporting, not as evidence that a particular procedure was or was not used.
In general, early termination can reduce the amount of information available and make estimates less precise. Its implications depend on why and when a study stopped, which are not stated. No inferential conclusion about multiplicity or missing-data bias can be drawn from the registry entries alone.
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 |
|---|---|---|---|
| Docetaxel, Carboplatin and Trastuzumab | 1 | 5 | 20.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Docetaxel, Carboplatin and Trastuzumab | 4 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Docetaxel, Carboplatin and Trastuzumab |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 1 of 5 |
| Febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infe | General disorders | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Docetaxel, Carboplatin and Trastuzumab |
|---|---|---|
| Fatigue (asthenia, lethargy, malaise) | General disorders | 4 of 5 |
| Hair loss/alopecia (scalp or body) | Skin and subcutaneous tissue disorders | 2 of 5 |
| Glucose, serum-high (hyperglycemia) | Investigations | 2 of 5 |
| Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | 2 of 5 |
| Diarrhea | Gastrointestinal disorders | 2 of 5 |
| Pain - Extremity-limb | General disorders | 2 of 5 |
| Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L) | General disorders | 1 of 5 |
| Bruising (in absence of Grade 3 or 4 thrombocytopenia) | Skin and subcutaneous tissue disorders | 1 of 5 |
| Rash: acne/acneiform | Skin and subcutaneous tissue disorders | 1 of 5 |
| ALT, SGPT (serum glutamic pyruvic transaminase) | Investigations | 1 of 5 |
Serious events with the highest arm-level rate: diarrhea (20.0%), febrile neutropenia (fever of unknown origin without clinically or microbiologically documented infe (20.0%).
The safety group is Docetaxel, Carboplatin and Trastuzumab. The registry lists 5 participants at risk for serious adverse events and 5 at risk for other adverse events. It lists 1 participant affected by a serious adverse event and 4 affected by other adverse events. These are participant-level counts in the posted safety record; the categories should not be assumed to be mutually exclusive, and the counts do not establish how many distinct event episodes occurred.
The top serious terms listed are diarrhea at 20.0% and febrile neutropenia at 20.0%. The febrile neutropenia term is accompanied by a truncated description in the registry. The percentages are reported as listed; the record does not provide enough detail in this entry to establish additional information about event timing, duration, severity grading, recurrence, or attribution. A term listed among the top serious events is not a complete account of all safety observations.
The death count is not reported in the registry record. Safety counts should be read as descriptive entries for this small, single-group study, not as comparative estimates against another treatment. The available data do not support conclusions about relative safety, and they should not be combined with the unevaluable primary endpoint to make an efficacy claim.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group study evaluated a regimen of trastuzumab, docetaxel, and carboplatin in women with stage II, stage III, or inflammatory breast cancer. It also establishes that the study was terminated and that the primary response-rate outcome could not be evaluated because insufficient data were collected. These registry entries describe the study and its reporting status; they do not quantify the regimen's effect.
The record does not show a response-rate estimate, an uncertainty interval, or a statistical test for the primary outcome. It therefore cannot show how often participants met a response definition or how precisely such a rate could be estimated. The outcome criteria and assessment details are not reported either. No efficacy conclusion should be inferred from the trial title, rationale, planned intervention, or the existence of a named endpoint.
The single-group design also limits comparisons. In general, a within-group outcome cannot provide the same evidence about a treatment contrast as a randomized comparison with a control group. The registry lists no separate comparator and no random allocation. Even if a response estimate had been available, the design would not by itself establish how outcomes differed from those under another treatment or control condition.
The safety entries document participants affected by serious and other adverse events, as well as two serious-event terms with reported percentages. They are descriptive entries, not comparative safety estimates, and the registry does not report a death count. The record also names secondary outcomes, but does not post their estimates in the information available.
Thus, what the registry can support is a limited account of design, enrollment, status, intended outcomes, and selected safety data. It does not support a claim that the studied regimen did or did not achieve a particular response, nor a claim about benefit relative to another option. The absence of an evaluable result is an information limitation, not evidence of either effect or no effect.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between an outcome being named and an outcome being measured well enough to analyze. The primary endpoint is clearly identified as antitumor activity measured by response rate, but the registry says early termination left insufficient data for evaluation. A protocol question can remain scientifically relevant while the available record does not produce an answer to it.
The study also shows why design and endpoint information need to be read together. It is a single-group, unmasked phase II study with enrollment listed as 5. A response-rate estimate, had one been reported, would describe the observed group and would need careful interpretation for precision. The absence of a comparator would still prevent a direct randomized contrast. These are distinct issues: limited information affects precision, while the design determines which comparisons the study can support.
Registry transparency matters because readers need to distinguish planned outcomes from reported results. The listed secondary outcomes, the intended 5-year primary timeframe, and the regimen details describe what the study set out to address. They do not supply outcome data. Likewise, a planning illustration is not an observed analysis and cannot repair an unevaluable endpoint.
Finally, safety reporting is not interchangeable with efficacy reporting. The registry provides some adverse-event counts and serious-event terms, but these entries do not answer the primary response question. Keeping outcome domains separate prevents a descriptive safety record from being mistaken for a treatment-effect estimate. The statistical lesson is to state what the record supports, identify what is missing, and avoid turning an unreported or unevaluable outcome into a positive or negative finding.
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 direct limitation is stated in the registry itself: the trial was terminated early and insufficient data were collected to evaluate the primary response-rate outcome. No response estimate, confidence interval, or primary analysis is reported. The reason for termination is not reported in the registry record, so its relationship to recruitment, feasibility, safety, or other considerations cannot be inferred.
Enrollment is listed as 5, and the study has a single group. The small listed enrollment limits the amount of information available for outcome estimation, while the absence of a comparator prevents direct between-group assessment. Allocation is listed as NA and masking as none. The record does not report a response definition, assessment schedule, analysis population, missing-data method, or detailed statistical analysis plan. Consequently, the reader cannot determine how endpoint eligibility or incomplete assessments were to be handled.
The record names pathological complete response, disease-free survival, and pathologic and molecular markers for predicting efficacy as secondary outcomes, but does not post their results in the available information. It lists four outcome measures and zero analyses. Multiplicity procedures and interim analyses are not reported in the registry record. The stated 5-year timeframe for the primary outcome does not establish the actual duration of follow-up or completion of assessments.
The arm description includes regimen details and says participants were to be restaged after the sixth cycle, but the available description is incomplete. Although conventional surgery and radiation therapy are listed as interventions, their use and timing are not fully reported in the record excerpt. The safety entries also have limits: a death count is not reported, and details about event timing, grading, recurrence, and attribution are not given. These gaps constrain interpretation of both outcomes and treatment delivery.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trastuzumab, Docetaxel, and Carboplatin in Treating Women With Stage II, Stage III, or Inflammatory Breast Cancer — NCT00118053.
- Registry record last updated 2013-11-20; status last verified 2013-09.
- Results first posted 2013-11-20.
- 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
This registry record describes a phase II, single-group study of trastuzumab, docetaxel, and carboplatin in women with stage II, stage III, or inflammatory breast cancer. Enrollment is listed as 5. The study started in 2005-04, had a primary completion date of 2008-12, and is marked terminated.
The primary outcome was antitumor activity measured by response rate, with a timeframe of 5 years. The registry states that early termination left insufficient data to evaluate it. The record lists pathological complete response, disease-free survival, and pathologic and molecular markers for predicting efficacy as secondary outcomes, but their results are not reported in the posted information.
The study has no comparator group, allocation is listed as NA, and masking as none. Safety entries list 1 participant affected by a serious adverse event and 4 affected by other adverse events, among 5 at risk in each category. Diarrhea and febrile neutropenia are each listed at 20.0% among the top serious terms. A death count and detailed statistical analysis plan are not reported in the registry record.