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 Targeted T Cells After Neoadjuvant Chemotherapy in Treating Women With Stage II or III Breast Cancer Undergoing Surgery (NCT01147016), a na phase 2 study. The primary outcome is Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01147016 |
| Phase | Phase 2 |
| Condition | Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Barbara Ann Karmanos Cancer Institute |
| Results posted | Yes |
2. Clinical Question
Population
Women with stage II–III breast cancer described in the primary outcome population as HER2/neu (0-2+) negative, treated with neoadjuvant chemotherapy. The official title also specifies hormone receptor negative disease. The registry record reports enrollment of 8 participants.
Intervention
HER2Bi-armed activated T cells after neoadjuvant chemotherapy. The intervention description specifies 4 intravenous T-cell infusions over 1 month. Cyclophosphamide, doxorubicin hydrochloride, and paclitaxel are described as physician-prescribed standard of care.
Comparator
There is no concurrent comparator group in the registry record. The study is listed as single-group, with allocation recorded as not applicable; it is not a randomized comparison.
Primary question
The primary endpoint is progression-free survival, measured in months from randomization until documented progression or death from any cause, whichever occurs first. The record defines progression using RECIST v1.0 and gives an assessment period of up to 7 years. It does not report the median progression-free survival or its 95% confidence interval.
In practical terms, the study was intended to describe outcomes after adding laboratory-treated T cells to treatment involving neoadjuvant chemotherapy and surgery. The registry rationale proposes that this sequence might improve pathologic complete response, but the listed primary endpoint is progression-free survival, not pathologic complete response. The record does not give a pathologic complete response result.
Because there is no concurrent control group, the study cannot directly estimate a treatment contrast against chemotherapy alone or another comparator. Its posted information can describe the study’s design and the outcomes recorded in the registry, but it does not establish what would have happened to the same participants under a different treatment strategy.
3. Trial Design
This is a phase II, single-group study sponsored by Barbara Ann Karmanos Cancer Institute. The registry lists allocation as not applicable and masking as none. Enrollment was 8, and the study status is terminated. The registry record does not report the reason for termination.
The study concerns women with stage II or III breast cancer. Its official title specifies HER2/Neu (0-2+), hormone receptor negative disease, while the primary endpoint population description refers to HER2/neu (0-2+) negative disease. The brief description places HER2Bi-armed activated T cells after neoadjuvant chemotherapy and before surgery. The listed treatment includes cyclophosphamide, doxorubicin hydrochloride, and paclitaxel, described as physician-prescribed standard of care, as well as therapeutic conventional surgery.
The intervention description states that participants received a total of 4 intravenous T-cell infusions over 1 month. The registry does not report further details about treatment delivery, adherence, or changes to therapy. Laboratory biomarker analysis is also listed as an intervention, but the record does not provide biomarker results.
The study start is listed as 2010-07, and primary completion is listed as 2017-07-01. These dates do not by themselves explain why the study was terminated or how follow-up proceeded for individual participants. That information is not reported in the registry record.
With one group and no allocation to a concurrent control, the design is descriptive rather than a randomized comparative trial. Any outcomes among the enrolled participants would not, on their own, separate the effects of the T-cell treatment from chemotherapy, surgery, the course of the disease, or other factors. The registry record does not report a protocol-based sample-size justification, detailed statistical plan, or reasons for selecting the enrollment target.
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 | Treated |
|---|---|
| STARTED | 8 |
| COMPLETED | 8 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Women with stage II-III, HER2/neu (0-2+) negative breast cancer treated with anti-CD3 x anti-HER2neu (Her2BI) armed, activated T cells after neoadjuvant chemotherapy
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 | Treated |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 44.5 (39 to 52) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 0 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| 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 | 4 |
| Race (NIH/OMB): White | Participants | 4 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 8 |
| enrollment | Participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival, measured over From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 7 years...
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is progression-free survival, reported in months. It is defined as the time from randomization until the first documented progression or death from any cause, whichever comes first. The stated assessment period is up to 7 years. Progression is defined using RECIST v1.0: an increase of 20% in the sum of the longest diameters of target lesions, a measurable increase in a non-target lesion, or the appearance of a new lesion.
Progression-free survival combines disease progression and death into one time-to-event outcome. In general, this kind of endpoint can capture how long participants remain alive without documented progression, while accounting for the timing of events rather than only whether an event occurred by a single cutoff. A median is the time by which half of the analyzed group has had an event, if the data permit that estimate. A 95% confidence interval describes uncertainty around the estimated median; it is not a range that contains the outcome time for 95% of individual participants.
The registry record names the median and its 95% confidence interval as the primary outcome measure, but it does not report either estimate. It also does not report the number of progression events, the number censored, or individual follow-up information. The endpoint definition therefore identifies what was intended to be measured, but the posted record does not allow the outcome’s magnitude or precision to be assessed.
The brief description mentions pathologic complete response as part of the rationale. That is distinct from the listed primary endpoint, and no pathologic complete response result is reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes that this was a small, single-group phase II study with enrollment of 8 and a terminated status. It identifies progression-free survival as the primary endpoint and defines how progression was assessed. It does not report the median progression-free survival, the 95% confidence interval, or a sponsor-reported hazard ratio. Consequently, the posted results do not provide a numerical estimate of progression-free survival or its precision.
The distinction between a defined endpoint and a reported result matters. A detailed endpoint definition explains what investigators intended to measure, but it does not reveal how participants fared. Without an estimate and its uncertainty, readers cannot judge the observed duration of progression-free survival from this registry entry. The record also does not report counts of progression events or follow-up, which limits assessment of how much information contributed to the endpoint.
The study has no concurrent control group. Even if an outcome estimate were available, a single-group result would describe outcomes among participants receiving the study regimen, not the difference attributable to that regimen compared with another treatment. Such a comparison would require a suitable control and an analysis designed to estimate the contrast. The registry lists allocation as not applicable and masking as none, and it explicitly does not describe a randomized comparison.
The enrollment of 8 also means that any participant-level events can have a substantial influence on a group summary. This is a general consideration for small studies, not a recalculation of the trial’s results. A confidence interval, if reported, would be important for conveying uncertainty; the record does not provide one for the primary endpoint. No conclusion about statistical significance can be drawn from the absence of a reported estimate or analysis.
The record lists 1 death in the treated group, but that safety count is not a substitute for the primary progression-free survival analysis. The time of death, relationship to progression, follow-up duration, and analysis population are not reported in the registry record. Similarly, the listed count of participants affected by other adverse events does not explain their nature or severity.
The registry status is terminated, but the reason for termination is not reported. Termination alone does not establish whether the study stopped for safety, recruitment, administrative, efficacy, or other reasons. The record also does not report a sample-size justification, interim analysis plan, or complete analysis methods. The available evidence therefore supports a description of the design and missing outcome information, not an estimate of treatment effect or a conclusion about comparative benefit.
9. Statistical Methods Explained
Progression-free survival is a time-to-event outcome: each participant contributes information about the time until progression or death, or about the period for which they are known to remain without either event. The registry defines the event as the first documented progression or death from any cause, whichever comes first. It specifies RECIST v1.0 criteria for progression and lists the endpoint unit as months.
What does a median time-to-event estimate mean?
In general, the median time-to-event estimate is the point at which the estimated event-free proportion reaches one half. It is not an arithmetic average of participant times. Estimating it typically uses methods that can incorporate participants whose event has not been observed by the end of their recorded follow-up. Such observations are commonly described as censored. This is a general explanation; the registry record does not report how censoring was handled in this study or provide participant-level follow-up.
The registry names a median and a 95% confidence interval for the primary outcome. In general, a confidence interval communicates uncertainty in an estimate under the assumptions of the analysis. A narrower interval indicates greater precision than a wider one, but it does not indicate that individual participants’ event times fall within that interval. Here, neither the median nor the interval is reported, so neither the endpoint’s estimated level nor its precision can be evaluated.
Why does the endpoint definition matter?
Combining progression and death makes the endpoint sensitive to either type of event. The event definition also depends on how progression is identified. The registry specifies target-lesion growth, measurable growth in a non-target lesion, or a new lesion under RECIST v1.0. General interpretation of a time-to-event outcome depends on consistent assessment and complete follow-up. The record does not report assessment schedules, review procedures, or whether progression was assessed by investigators or an independent reviewer.
What can a single-group design estimate?
In general, a single-group study can describe outcomes in the enrolled group. It does not supply a concurrent estimate of outcomes under an alternative intervention. Differences between participants who receive a study regimen and historical or external groups can reflect differences in eligibility, care, follow-up, or other characteristics. No external comparison is reported in this registry record. The study is listed as single-group, with allocation not applicable and no masking.
The registry’s statistical interpretation note says that only sponsor-reported hazard ratios are used for time-to-event outcomes. However, the record lists no analyses and does not report a hazard ratio. A hazard ratio is generally a relative measure comparing event rates over time between groups; it is not the same as a difference between medians. Since this study has no concurrent comparison group and no hazard ratio is reported, there is no posted hazard-ratio estimate to interpret.
What do planning illustrations represent?
Sample-size calculations are planning tools, not evidence that a study had a particular probability of detecting an effect after it was conducted. General planning calculations depend on assumptions, including the target effect, significance level, allocation, and analysis method. Any planning illustration based on an observed effect would be especially difficult to interpret when the registry does not report the primary outcome estimate. No numeric planning result or protocol-based sample-size justification is reported in the registry record. A planning illustration must not be read as observed power or as a trial result.
What is missing for a full statistical assessment?
The registry reports enrollment and the intended primary endpoint but not the endpoint estimate, confidence interval, event count, censoring details, or analysis population. It also lists 2 outcome measures and 0 analyses. The secondary outcome is overall deaths, but no corresponding outcome estimate is included in the record. The statistical plan, handling of missing outcome data, and any interim analyses are not reported. Without those details, the analysis choices and the completeness of the reported results cannot be assessed.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study tests or estimates several outcomes, time points, subgroups, or treatment comparisons. In general, looking across many analyses can increase the chance that an apparently notable result arises by chance. A prespecified analysis plan can clarify which endpoint is primary and how other analyses should be interpreted. The registry identifies progression-free survival as the primary outcome and overall deaths as a secondary outcome, but the analysis plan and any multiplicity approach are not reported in the registry record.
The record lists 2 outcome measures and 0 analyses. It gives the primary endpoint definition but does not provide an estimate for that endpoint or a result for overall deaths. It also does not report subgroup analyses, additional time points, or interim analyses. Their presence or absence in study conduct cannot be inferred from the available registry entry; these details are not reported in the registry record.
Missing-data methods are also not reported. For a time-to-event endpoint, relevant information can include how follow-up ends, how participants without an observed event are handled, and whether data are censored at particular times. The registry record does not give participant-level follow-up or explain how missing assessments were treated.
The analysis population is not specified in the posted outcome information. Enrollment is 8, but the record does not state how many participants were included in the primary endpoint analysis or whether any participants were excluded from it. It also does not report reasons for exclusions or deviations from the planned treatment. These omissions prevent assessment of whether the analyzed group matched the enrolled group and how missing information might affect the outcome estimate.
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 |
|---|---|---|---|
| Treated | 0 | 8 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treated | 1 of 8 |
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 |
|---|---|
| Treated | 8 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treated |
|---|---|---|
| chills/rigors | General disorders | 7 of 8 |
| Nausea | Gastrointestinal disorders | 7 of 8 |
| fatigue | General disorders | 7 of 8 |
| fever | General disorders | 6 of 8 |
| hypotension | General disorders | 5 of 8 |
| Anemia | Blood and lymphatic system disorders | 5 of 8 |
| Low platelets | Blood and lymphatic system disorders | 5 of 8 |
| Vomiting | Gastrointestinal disorders | 4 of 8 |
| neutropenia | General disorders | 4 of 8 |
| Rash | General disorders | 3 of 8 |
The registry lists one treated group. It reports 8 participants at risk for serious adverse events and 0 affected by serious adverse events. It also reports 8 at risk for other adverse events and 8 affected by other adverse events. The record lists 1 death. The serious adverse-event terms are not listed.
These are counts recorded for the group, not comparative estimates. With no concurrent control group, the figures do not establish whether events were more or less frequent than under another treatment. The record does not report the names, grades, timing, duration, or treatment relatedness of the other adverse events, nor does it describe how adverse events were collected. The serious-event term list is empty, so the registry entry does not identify specific serious events.
The death count should not be interpreted as a cause-specific outcome or as evidence about treatment causation. The registry record does not report the cause or timing of death, or whether the death was related to treatment. It also does not state whether all participants completed safety follow-up. These limitations mean that the counts describe the posted safety fields but do not provide a complete characterization of the safety experience.
12. What the Result Does, and Does Not, Mean
The record documents a phase II, single-group study of HER2Bi-armed activated T cells after neoadjuvant chemotherapy in women with stage II or III breast cancer. It reports enrollment of 8, a terminated status, and a primary endpoint of progression-free survival. The endpoint definition and the regimen components are described, including 4 intravenous T-cell infusions over 1 month and physician-prescribed standard-of-care chemotherapy.
The posted information does not report a median progression-free survival, a 95% confidence interval, or a hazard ratio. It therefore does not quantify the primary outcome or how precisely it was estimated. The registry rationale refers to a possible improvement in pathologic complete response, but no such result is reported. No conclusion about the magnitude of an outcome can be drawn from the endpoint definition alone.
The study’s single-group design also limits what an outcome could establish. Without a concurrent comparator, the record cannot show how outcomes differ from those under another treatment strategy. Outcomes among participants in a single group cannot by themselves identify the contribution of any one treatment component.
The safety fields report 0 participants affected by serious adverse events, 8 affected by other adverse events, and 1 death. These figures do not describe event types or establish causality, and the record does not report the reason the study was terminated. They should be read as the registry’s posted counts, not as a complete safety analysis.
In general, a registry entry is most informative when it includes outcome estimates, uncertainty, analysis populations, and enough methods to understand how results were obtained. Those details are not reported. The record can support a description of the study and its intended endpoint, but it does not establish comparative efficacy, treatment benefit, or the reason for study termination.
13. Why This Trial Matters Statistically
This study illustrates why trial design and outcome reporting must be considered together. A time-to-event endpoint such as progression-free survival can describe when progression or death occurs, while its estimate and confidence interval communicate the observed outcome and its precision. The registry record defines that endpoint but does not report its median or confidence interval. Readers therefore cannot assess the primary outcome from the posted results.
The single-group design is also central to interpretation. It can provide descriptive information about participants receiving the regimen, but it lacks a concurrent comparator for estimating a treatment contrast. That limitation remains even when a study has a clearly defined endpoint. A design’s ability to answer a comparative question depends on how comparison groups are formed, not only on the endpoint selected.
The enrollment of 8 highlights the importance of reporting the number analyzed, event counts, and follow-up. In general, summaries based on small groups can be sensitive to individual observations, making uncertainty and transparent accounting especially relevant. The registry does not provide the event and analysis details needed to evaluate the primary estimate.
The record also separates a proposed rationale from a reported outcome. The brief description discusses pathologic complete response, whereas the registered primary endpoint is progression-free survival. No pathologic complete response result is reported. Keeping those outcomes distinct prevents a study’s stated motivation from being mistaken for evidence that the intended outcome was achieved.
Finally, the terminated status is a relevant feature, but the reason is not reported. Statistical interpretation should not infer a cause from that status alone. This record is therefore useful as an example of how endpoint definitions, study structure, outcome reporting, and uncertainty each shape what can be concluded from a clinical trial registry.
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.
Several trial-specific limitations constrain interpretation. The study is single-group, with allocation not applicable and no masking. There is no concurrent control group in the registry record, so it does not support a direct estimate of how the regimen compares with another treatment strategy.
Enrollment was 8. The registry does not report the number included in the primary endpoint analysis, the number of progression events, the number censored, or individual follow-up. It also does not report the median progression-free survival or the 95% confidence interval, leaving the primary result and its precision unavailable.
The primary endpoint refers to time from randomization, but the record lists a single-group design and does not explain how the time origin was applied. This does not establish that the endpoint was defined incorrectly; it identifies a detail that cannot be evaluated from the registry entry. The assessment schedule and procedures for applying RECIST v1.0 are also not reported.
The study is marked terminated, but the reason is not reported. The record does not provide a detailed statistical analysis plan, sample-size justification, interim analysis information, missing-data methods, or a description of the analysis population. Although overall deaths is listed as a secondary outcome, no corresponding result is reported. The registry also does not provide pathologic complete response results despite discussing that outcome in its rationale.
Safety reporting is limited to aggregate counts. The record does not identify the other adverse events, report their grades or timing, or state whether events were judged related to treatment. It lists no serious adverse-event terms. These omissions limit the clinical and statistical interpretation of the safety information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Targeted T Cells After Neoadjuvant Chemotherapy in Treating Women With Stage II or III Breast Cancer Undergoing Surgery — NCT01147016.
- Registry record last updated 2023-04-27; status last verified 2023-04.
- Results first posted 2023-04-27.
- 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
This is a phase II, single-group study sponsored by Barbara Ann Karmanos Cancer Institute, with enrollment of 8 and status listed as terminated. It concerns HER2Bi-armed activated T cells after neoadjuvant chemotherapy in women with stage II or III breast cancer. The intervention description specifies 4 intravenous infusions over 1 month, alongside physician-prescribed cyclophosphamide, doxorubicin hydrochloride, and paclitaxel.
The primary endpoint is progression-free survival in months, defined from randomization to documented progression or death, whichever comes first, with progression assessed using RECIST v1.0. The registry record does not report the median, its 95% confidence interval, or a hazard ratio. Overall deaths is listed as a secondary outcome, but no result is reported. The reason for termination and the detailed statistical analysis plan are not reported in the registry record.