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 Trial of Eribulin Followed by Doxorubicin & Cyclophosphamide for Her2-negative, Locally Advanced Breast Cancer (NCT01498588), a na phase 2 study. The primary outcome is Pathologic Complete Response Rate at the Time of Surgery.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01498588 |
| Phase | Phase 2 |
| Condition | Breast Neoplasms, Breast Cancer, Breast Tumors |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Emory University |
| Results posted | Yes |
2. Clinical Question
Population
People with HER2-negative, locally advanced breast cancer were the study population. The registry record lists breast neoplasms and related breast cancer terms as conditions. Seven participants enrolled. Pathology information at definitive resection was available for six participants; one participant was ultimately lost to follow-up.
Intervention
The regimen was neoadjuvant eribulin followed by dose-dense doxorubicin and cyclophosphamide. Eribulin was scheduled on Day 1 and Day 8 of a 21 day cycle for 4 cycles. The subsequent doxorubicin and cyclophosphamide treatment was scheduled every 14 days for 4 cycles. Pegfilgrastim is also listed as an intervention.
Comparator
There was no separate comparison group. The study is recorded as a single-group trial, with allocation listed as not applicable and masking listed as none. Results therefore describe participants receiving the regimen, rather than a contrast between treatment groups.
Primary question
The primary question concerned the number of participants with a pathologic complete response at surgery. A response was defined as no invasive or in situ residual disease in the breast and lymph node, designated ypT0N0. The primary outcome count is not reported in the registry record.
The study rationale was to assess whether this pre-surgery chemotherapy sequence could shrink breast tumors and permit breast preservation, and to assess whether the cancer was responsive to chemotherapy. Those aims describe the clinical questions motivating the study; they are not themselves reported outcome findings. The record gives no comparison with surgery followed by chemotherapy or with another regimen, so it cannot establish a difference between treatment strategies.
3. Trial Design
This was a phase II, single-group neoadjuvant study sponsored by Emory University. It enrolled 7 participants and is recorded as terminated. The study began in 2011-11, with primary completion in 2015-06. The registry entry does not report why the study was terminated.
Participants were assigned to the sequence of eribulin followed by dose-dense doxorubicin and cyclophosphamide. Eribulin was described as intravenous treatment on Day 1 and Day 8 of each 21 day cycle for 4 cycles, at 1.4mg/m² per administration. Doxorubicin was described as intravenous treatment on Day 1 of each 14-day cycle for 4 cycles, at 60mg/m². Cyclophosphamide is listed in the regimen, but its dose is not reported in the registry record. Pegfilgrastim is listed among the interventions.
The study was open-label, with masking recorded as none. Allocation is listed as not applicable because the design had a single group rather than a randomized allocation among groups. There is no control group, so the design does not support a like-for-like treatment comparison. The registry record does not report a sample-size justification, a statistical analysis plan, or interim analyses.
The primary outcome was assessed at surgery, with the timeframe described as an average of 24 weeks. The registry description says treatment lasted 20 weeks and surgery typically occurred 4-6 weeks after chemotherapy. The primary outcome was the number of participants meeting the pathologic complete response definition. The record also lists toxicity, measured as the number of participants with any adverse events, as a secondary outcome. The status of the study and the small enrollment describe the conduct recorded; neither explains why recruitment ended nor establishes the reason for termination.
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 | Eribulin+Doxorubicin+Cyclophosphamide |
|---|---|
| STARTED | 7 |
| COMPLETED | 7 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Pathology information at the time of definitive resection was available for six of seven patients. One patient was ultimately lost to follow-up.
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 | Eribulin+Doxorubicin+Cyclophosphamide |
|---|---|---|
| Participants analysed | 7 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 7 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 1 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 2 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Pathologic Complete Response Rate at the Time of Surgery, measured over Average of 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the number of participants with a pathologic complete response at the time of surgery. The registry defines this response as no evidence of invasive or in situ residual disease in either the breast or lymph node, recorded as ypT0N0. The assessment was based on the pathologist’s standard-of-care evaluation of residual disease at definitive resection.
Pathologic response is used in neoadjuvant studies because it describes the cancer remaining in tissue after pre-surgery treatment, at the point when surgical specimens can be assessed. It is distinct from a measure of tumor shrinkage alone: the registry’s definition requires no invasive or in situ residual disease in the specified sites. The endpoint is also distinct from longer-term outcomes, which are not identified as the primary outcome in this record.
The registry describes the endpoint timeframe as an average of 24 weeks, reflecting treatment for 20 weeks and surgery typically 4-6 weeks after chemotherapy. Pathology information at definitive resection was available for six of the seven participants, while one participant was lost to follow-up. The number meeting the pCR definition is not reported in the registry record, so the endpoint’s observed result cannot be described from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase II study of one neoadjuvant regimen in a single group of 7 participants. It reports the study design, planned treatment sequence, primary outcome definition, and availability of pathology information, but does not report the primary outcome count. Consequently, the number of participants with a pathologic complete response, and the proportion represented by that count, are not reported in the registry record. No estimate of the regimen’s response rate can be stated from these registry entries.
Pathology information at definitive resection was available for six participants, and one participant was ultimately lost to follow-up. This means the record identifies incomplete outcome ascertainment for the enrolled group. Without the outcome count or the missing participant’s surgical pathology result, the extent to which missing follow-up could affect the endpoint is not quantifiable from the registry record. It would be inappropriate to assume that the missing participant did or did not have a response.
There is no control group. Any result reported for this study would describe outcomes among participants who received the regimen, rather than show how outcomes compare with a different treatment or with no treatment. A single-group study can offer descriptive information, but cannot separate treatment effects from other explanations for observed outcomes. The record does not report a randomized comparison or a masking procedure.
The study enrolled 7 participants and is listed as terminated. These registry entries limit how much can be inferred about the population beyond those enrolled. The registry does not state why the study was terminated, so the status should not be interpreted as evidence about the regimen’s activity, safety, feasibility, or the reason for stopping.
No confidence interval, p-value, or statistical analysis result is reported in the registry record. A confidence interval, in general, describes the precision and uncertainty around an estimate; without an endpoint estimate and interval, the precision of a response-rate estimate cannot be assessed. The registry also lists no statistical analyses. Accordingly, the most defensible reading is descriptive: the trial was designed to assess pCR after the specified treatment sequence, but its posted primary outcome result is unavailable in the registry record. The absence of a posted result is not evidence of either benefit or lack of benefit.
9. Statistical Methods Explained
The design determines what comparisons can be made. This study is recorded as single-group, with allocation not applicable and masking none. Participants received the same treatment sequence; there is no separate control arm. As a general statistical matter, a comparison requires outcomes from distinct groups or a clearly specified within-person contrast. Neither is described. The results, if reported, would therefore be interpreted as outcomes among treated participants, not as a randomized estimate of a difference between treatments.
What does the primary endpoint count?
The primary endpoint is a binary clinical classification summarized as a count: participants either met the registry’s pCR definition or did not. The definition requires no invasive or in situ residual disease in the breast and lymph node, or ypT0N0. The registry describes assessment at surgery by pathology. A count is a direct description of how many participants met the definition, while a rate would express that count relative to a specified analysis population. The count itself is not reported in the registry record, so neither the count nor a rate can be presented.
How should precision be considered?
In general, an estimate from a small study is subject to substantial sampling uncertainty: results in the enrolled participants may not closely represent outcomes in a broader patient population. A confidence interval is one way to express uncertainty around an estimate, with wider intervals indicating less precision. The registry record does not report a pCR estimate or confidence interval, so the precision of the primary endpoint cannot be evaluated. No new interval or significance test can be reconstructed from the available outcome details.
What can a significance test establish?
In general, a significance test evaluates compatibility between observed data and a specified null hypothesis under stated assumptions. It does not by itself establish clinical importance or prove that a treatment caused an outcome. In a single-group design, the choice of a meaningful reference or null value matters. The registry record does not report such a reference, a hypothesis test, a p-value, or a statistical analysis plan. There is therefore no basis to describe a statistical test result or to infer the planned testing strategy.
What does over-dispersion mean in general?
For count outcomes, over-dispersion means that variation in the data is greater than a statistical model expects. If it is present and ignored, standard errors and resulting uncertainty estimates may be misleading. This is a general issue for modeling counts, not a reported feature of this study. The registry supplies no fitted count model, dispersion estimate, or analysis result, and the primary endpoint is described as a participant count rather than a model-based rate.
How do planning calculations differ from observed results?
A sample-size calculation is a planning illustration: it asks what study size could be needed under specified assumptions about the effect to detect, significance level, allocation, and statistical method. Such a calculation does not describe the number enrolled, the number with an outcome, or the power observed after the study. The record’s methodological notes describe a general planning approach based on fractions of an observed effect and equal allocation between two arms, but this trial itself is single-group. No numerical planning results or trial-specific sample-size justification are reported in the registry record. Any planning illustration should therefore not be mistaken for a randomized design, an observed outcome, or observed power.
The registry lists two outcome measures and no statistical analyses. It does not report how outcomes were summarized beyond the primary endpoint’s count, whether any adjustments were planned, or how uncertainty would be quantified. Such details cannot be inferred from the endpoint label or the study phase. In general, analysis choices should follow the design and outcome definition, and readers need those choices to assess how an estimate was produced. Here, the posted information supports a description of the design and endpoint, but not an independent reconstruction of an efficacy analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures: the primary pCR count and a secondary toxicity outcome, defined as the number of participants with any adverse events. Multiple outcomes can raise multiplicity concerns when several hypotheses are tested, because interpreting a set of tests differs from interpreting one prespecified test. The registry record does not report a multiplicity adjustment, a hierarchy among statistical tests, or a testing plan. It also lists no statistical analyses, so there are no reported test results to interpret across outcomes.
Missing outcome information is relevant to the primary endpoint. Pathology information at definitive resection was available for six of seven participants, and one participant was ultimately lost to follow-up. The pCR status of that participant is not reported in the registry record, nor is an approach to handling missing endpoint data. The record does not state whether the primary outcome analysis used all enrolled participants or only those with available pathology.
In general, missing data can affect an estimate when outcome availability is related to participants’ characteristics or outcomes. Without the missing participant’s pathology result and a stated analysis approach, the direction and size of any effect on the endpoint cannot be determined. The analysis population, any imputation method, and any sensitivity analyses are not reported in the registry record. No assumption about the missing result should be substituted for the absent information.
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 |
|---|---|---|---|
| Eribulin+Doxorubicin+Cyclophosphamide | 0 | 7 | 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 |
|---|---|
| Eribulin+Doxorubicin+Cyclophosphamide | 2 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Eribulin+Doxorubicin+Cyclophosphamide |
|---|---|---|
| Neuropathy | Nervous system disorders | 2 of 7 |
| Fatigue | General disorders | 1 of 7 |
| Insomnia | General disorders | 1 of 7 |
| Rash | Skin and subcutaneous tissue disorders | 1 of 7 |
| Anorexia | Gastrointestinal disorders | 1 of 7 |
| Hemorrhage | Blood and lymphatic system disorders | 1 of 7 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 1 of 7 |
| Elevated ALT | Metabolism and nutrition disorders | 1 of 7 |
| Elevated AST | Metabolism and nutrition disorders | 1 of 7 |
| Hypercalcemia | Metabolism and nutrition disorders | 1 of 7 |
The safety record lists 7 participants at risk for serious adverse events and 0 participants affected in that category. It also lists 7 participants at risk for other adverse events and 2 participants affected. No serious adverse event terms are listed. The death count is not reported in the registry record.
These figures are counts of participants, not counts of separate adverse-event episodes. They describe the safety information recorded for this treatment group; there is no comparator group for assessing relative safety. The record does not give the specific other adverse events, their severity, timing, duration, or relationship to treatment. It also does not provide enough information to characterize safety beyond the recorded participant counts. The serious adverse-event count should be read as a registry entry for this small group, not as proof that serious events cannot occur.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study enrolled 7 participants to receive neoadjuvant eribulin followed by dose-dense doxorubicin and cyclophosphamide. It also defines the primary endpoint as pathologic complete response at surgery and explains how that response was assessed. Those details help clarify what the study set out to measure.
The record does not report the number of participants who achieved pCR. As a result, it does not support a response-rate estimate or a conclusion about the primary outcome. The fact that pathology information was available for six participants and one was lost to follow-up also means the endpoint is not fully documented for the enrolled group.
There is no control group, so the study cannot show how outcomes compare with another regimen or treatment strategy. Even if a pCR count were available, a single-group result would be descriptive and would not by itself establish that the regimen caused the observed outcomes. The study’s termination status does not explain why it ended, and should not be treated as an efficacy or safety finding.
The safety entries report no participants affected in the serious adverse-event category and 2 participants affected in the other adverse-event category. They do not provide event descriptions or a controlled comparison. Overall, the registry record documents the study question, treatment sequence, design, and limited safety counts, but leaves the primary outcome result and several analysis details unreported. It therefore supports a description of what was studied, not a conclusion that the regimen does or does not work.
13. Why This Trial Matters Statistically
This study illustrates why design and outcome reporting must be read together. A defined pathology endpoint gives a clear account of what investigators intended to measure, while the single-group design sets a limit on what comparisons the study can make. Without a control group, outcomes cannot be contrasted with another treatment under the same trial conditions.
The small enrollment of 7 participants and the unreported pCR count also make the distinction between a study’s question and its evidence especially important. The registry identifies the endpoint and records that pathology information was available for six participants, but does not post the endpoint result. A prespecified and clinically interpretable endpoint is useful, yet its definition alone is not evidence about how many participants met it.
The record also shows the value of transparent reporting about missingness and safety. One participant was lost to follow-up, and the safety entry provides participant counts without event descriptions. Those details can guide readers about what information is and is not available, but they do not explain missing outcomes or provide a comparative safety assessment.
Statistically, this is a reminder that an observed count, its denominator, uncertainty, and analysis population all matter. Here, the primary count and its analysis details are not reported in the registry record. The study can therefore be understood as an investigation of a treatment sequence and a pathology-based endpoint, while its posted record does not permit an estimate of the endpoint result or a comparative inference.
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 principal limitation for interpreting efficacy is that the registry record does not report the primary pCR count. Although the endpoint is defined and the record states that pathology information was available for six of seven participants, no result is posted for those participants. One participant was ultimately lost to follow-up, and the record does not explain how that missing outcome was handled.
The study had a single group and no randomized comparator. Therefore, it cannot provide a direct comparison of the regimen with another treatment. Its enrollment was 7 participants, which restricts the amount of information available for describing outcomes and safety. The record does not report a confidence interval or other uncertainty estimate for the primary endpoint.
The registry describes the study as terminated but does not report the reason. It does not state the sample-size justification, the statistical analysis plan, any interim analyses, or the population used for the endpoint analysis. The specific other adverse events are not reported, and the death count is not reported in the registry record. These gaps limit assessment of outcome completeness, analysis choices, and safety context. They should not be filled by assumptions about the protocol or the missing participant’s outcome.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial of Eribulin Followed by Doxorubicin & Cyclophosphamide for Her2-negative, Locally Advanced Breast Cancer — NCT01498588.
- Registry record last updated 2016-10-03; status last verified 2016-09.
- Results first posted 2016-10-03.
- 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
NCT01498588 is a phase II, single-group, open-label study sponsored by Emory University. It enrolled 7 participants and is listed as terminated. The treatment sequence was neoadjuvant eribulin followed by dose-dense doxorubicin and cyclophosphamide, with pegfilgrastim also listed as an intervention.
The primary endpoint was the number of participants with pCR at surgery, defined as no invasive or in situ residual disease in the breast and lymph node. Pathology information was available for six participants; one participant was lost to follow-up. The primary outcome count is not reported in the registry record. Safety entries list 0 participants with serious adverse events and 2 with other adverse events; the death count is not reported. There is no control group, and no statistical analyses are listed.