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 Navelbine, Taxol, Herceptin and Neupogen in Stage IV Breast Cancer: A Phase I - II Trial (NCT00041470), a na phase 1, phase 2 study. The primary outcome is To Measure Response Rates, Time to Progression and Survival in Patients so Treated..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00041470 |
| Phase | Phase 1, Phase 2 |
| Condition | Breast Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 38 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Washington |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerns patients with Stage IV, or metastatic, breast cancer. The registry record lists Breast Cancer as the condition and states that Herceptin was included for patients with HER-2/neu positive disease. Further eligibility details, such as how participants were selected or what prior treatment they had received, are not reported in the registry record.
Intervention
The regimen combined weekly intravenous paclitaxel and vinorelbine with daily G-CSF support. The listed paclitaxel dose levels were 50 mg/m2, 60 mg/m2, 70 mg/m2, and 80 mg/m2; weekly vinorelbine was described at 20 mg/m2. Herceptin was included for patients with HER-2/neu positive disease. The record describes dose-finding and toxicity aims as well as assessment of clinical outcomes.
Comparator
No separate control group is listed. The trial is recorded as single-group, with allocation marked NA and no masking. Although the brief summary asks whether the regimen is more effective than standard therapy, the registry record does not describe a concurrent standard-therapy arm. That intended question therefore cannot be answered as a randomized, like-for-like comparison using the recorded design.
Primary question
The stated primary objective is to measure response rates, time to progression, and survival in treated patients. The outcome record describes a participant count with a timeframe of 1 year, but does not provide a reported count or define the response criteria, progression rules, or survival analysis. Consequently, the registry record does not establish what response, progression, or survival results were observed.
3. Trial Design
This was a Phase I–II, single-group trial sponsored by the University of Washington. The registry record lists 38 participants as the enrollment, allocation as NA, and masking as none. The study began in 2001-03, had a primary completion date of 2008-08, and is marked terminated. The reason for termination is not reported in the registry record.
The treatment description combines weekly intravenous paclitaxel and vinorelbine with daily G-CSF support. Vinorelbine was described as being given one hour after paclitaxel. Paclitaxel dose levels were listed as 50 mg/m2, 60 mg/m2, 70 mg/m2, and 80 mg/m2, while the regimen description gives weekly vinorelbine at 20 mg/m2. Herceptin was included for patients with HER-2/neu positive disease. The record does not report the number of participants treated at each dose level or provide further dosing details for Herceptin or G-CSF.
The stated aims include identifying the highest doses of paclitaxel and vinorelbine that could be given safely, describing side effects, and measuring response, time to progression, and survival. A secondary objective concerns qualitative and quantitative toxicity. No separate comparator group is recorded, so the design does not support a direct concurrent comparison with standard therapy. Details of the dose-escalation procedure, treatment duration, follow-up schedule, and rules for assigning dose levels are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Paclitaxel, Vinorelbine, G-CSF, Herceptin - no Placebo |
|---|---|
| STARTED | 38 |
| COMPLETED | 0 |
| NOT COMPLETED | 38 |
Reasons for not completing this period:
| Reason | Paclitaxel, Vinorelbine, G-CSF, Herceptin - no Placebo |
|---|---|
| Loss of Funding | 38 |
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 | Phase I/II |
|---|---|---|
| Participants analysed | 38 | |
| Age, Continuous | years | 46.5 (27 to 73) |
| Sex: Female, Male: Female | Participants | 38 |
| Sex: Female, Male: Male | Participants | 0 |
| Baseline Participants | participants | 38 |
6. Primary Endpoint
The registry lists the primary outcome as To Measure Response Rates, Time to Progression and Survival in Patients so Treated., measured over 1 year.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The stated primary objective is to measure response rates, time to progression, and survival among treated participants. These outcomes address different aspects of disease course: response concerns whether disease meets a defined improvement criterion, time to progression concerns how long it takes for disease to worsen under a specified definition, and survival concerns time until death under a stated endpoint definition. These are general descriptions; the registry record does not give operational definitions or analysis rules for these outcomes.
The primary outcome is recorded as a count of participants with a timeframe of 1 year. That description does not specify how the count maps to response, progression, or survival, whether the outcomes were assessed separately, or how participants without a recorded assessment were handled. No primary outcome result or analysis is reported in the registry record. Thus, the stated endpoint aims explain what the investigators intended to measure, but the posted record does not supply the results needed to estimate response, progression, or survival in this trial.
Such endpoints are used in clinical research because they describe whether participants experience clinically relevant events over treatment and follow-up. Interpreting them requires clear event definitions, an analysis population, a follow-up method, and appropriate handling of incomplete observation. Those details are not reported in the registry record, so no further endpoint interpretation can be made from the posted information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes an investigational combination regimen and lists 38 participants as enrolled. It does not report a primary response, time-to-progression, or survival result, and the analysis count is recorded as zero. Accordingly, the posted information does not quantify the regimen’s efficacy on the stated primary objectives. It also does not provide estimates of uncertainty, such as confidence intervals, or a statistical test for those outcomes.
The design is single-group, with no masking and no separate control arm listed. The brief summary expresses an interest in whether the combination is more effective than standard therapy, but the recorded design does not include a concurrent standard-therapy group. Without such a comparison, outcomes in this group cannot establish a treatment difference relative to standard therapy. General differences in patient characteristics, disease course, and care over time could affect any comparison with results from elsewhere; the record does not describe an external comparison.
The posted safety information lists 38 participants at risk for other adverse events, with 38 affected, and 38 at risk for serious adverse events, with 10 affected. The listed leading serious adverse event terms include leukopenia at 10.5%, mucositis at 5.3%, febrile neutropenia at 5.3%, aspiration pneumonia at 2.6%, and congestive heart failure at 2.6%. These figures describe the recorded safety observations. They do not show whether an event was caused by a particular drug, and the registry record does not give a comparison group for assessing relative risk. Deaths are not reported in the registry record.
The status is terminated, but the reason for termination is not reported in the registry record. Termination alone does not explain whether recruitment, treatment, follow-up, or reporting was affected. Nor does it establish why primary outcome analyses are absent. The record does not report a statistical analysis plan, analysis population, handling of missing outcome data, or whether any interim analyses were conducted. These omissions limit how fully the study’s stated questions can be assessed.
Overall, the registry information documents the treatment approach, enrollment, intended outcomes, and selected adverse event summaries. It does not provide the primary outcome estimates required to judge the observed response, progression, or survival results, and its single-group design does not support a controlled claim of comparative effectiveness.
9. Statistical Methods Explained
General statistical explanation: a study design determines which comparisons can be supported by the data. This trial is recorded as a single-group study, with allocation marked NA and no masking. Participants received the described combination regimen; no concurrent control arm is listed. As a result, outcome counts can describe what was recorded among treated participants, but they cannot by themselves estimate how outcomes would have differed under another treatment.
What does the Phase I–II designation indicate?
The registry record labels the study Phase I and Phase II and describes both dose-finding and clinical outcome aims. In general, early-phase studies may investigate tolerability and dose levels, while also collecting information about outcomes. The record lists paclitaxel dose levels of 50 mg/m2, 60 mg/m2, 70 mg/m2, and 80 mg/m2, and weekly vinorelbine at 20 mg/m2. It does not report the dose-escalation rules, the number of participants assigned to each level, dose-limiting toxicity criteria, or a recommended dose determination. Those aspects therefore cannot be reconstructed from the registry record.
How should the primary outcome description be read?
The primary objective names response rates, time to progression, and survival, while the outcome description records a count of participants and a timeframe of 1 year. A count is a descriptive quantity: it tells how many participants meet an event definition, if that definition and the count are available. The posted record does not provide the primary count or specify the criteria used to classify response, progression, or survival. It also does not say whether each was analyzed as a separate endpoint or explain censoring and follow-up rules for time-to-event outcomes.
General statistical explanation: for a binary outcome, a confidence interval expresses uncertainty around an estimated event proportion. For a time-to-event outcome, methods generally account for participants whose event time is not observed during follow-up. Such methods depend on clearly specified event definitions, follow-up, and censoring. No estimate, confidence interval, or time-to-event analysis is reported in the registry record, so there is no numerical precision assessment to interpret for the primary outcomes.
What can the safety counts tell a reader?
The safety record gives a group at risk and an affected count for serious and other adverse events. For serious events, 10 of 38 participants are recorded as affected; for other events, 38 of 38 are recorded as affected. These are descriptive counts, not controlled treatment effects. The listed serious event terms are accompanied by percentages, but the record does not provide event-specific participant counts for each term or clarify whether participants could appear under multiple terms. General statistical explanation: adverse event records can count participants, event occurrences, or both; the distinction matters because one participant can experience more than one event.
The record does not state whether adverse events were graded, how attribution to treatment was determined, or the observation period used for the safety summaries. It gives no comparator group. Therefore, the counts describe the registry’s recorded safety observations but cannot establish causation or a relative safety difference.
How should the planning illustrations be interpreted?
General statistical explanation: a sample-size calculation estimates how many observations might be needed to detect a specified effect under stated assumptions. The planning illustrations associated with this record are described as using the observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a stated fraction of the observed effect. They are planning illustrations, not the trial’s own design or an estimate of observed power. The trial itself is recorded as single-group, so the equal-allocation two-arm assumption does not describe its recorded enrollment structure. No sample-size justification from the trial protocol is reported in the registry record.
More generally, power depends on the assumed effect, variability or event frequency, significance threshold, allocation, and analysis method. A planning calculation does not guarantee that a study will detect an effect, and it does not replace a reported estimate with uncertainty. Since no primary outcome estimate or analysis is posted for this trial, any planning illustration cannot supply evidence about the trial’s observed response, progression, or survival.
The registry record reports zero analyses. It does not report the statistical plan, the analysis population, missing-data procedures, interim analyses, or multiplicity adjustments. General statistical explanation: a prespecified plan helps readers understand which outcomes and analyses were intended, while adjustment for multiple testing may be relevant when several outcomes or comparisons are assessed. Without those details or results, the intended inferential procedure and the strength of any conclusions cannot be evaluated.
10. Multiplicity, Missing Data and Analysis Populations
The registry record names several primary outcome aims: response rates, time to progression, and survival. It also lists a toxicity objective. When a study examines multiple endpoints, multiplicity can matter because the chance of at least one apparently notable finding may increase as more questions are tested. This is a general statistical consideration; the registry record does not report whether the investigators adjusted for multiplicity, prioritized one endpoint, or specified a testing sequence.
No primary outcome analysis is reported, and the analysis count is zero. The registry record does not report an analysis population, such as whether outcomes were analyzed among all enrolled participants or only those who received treatment. It also does not describe how missing assessments, withdrawal, or incomplete follow-up were handled. These details matter because the amount and pattern of missing data can affect both estimates and their interpretation. The reason for the terminated status is not reported in the registry record, so its relationship to recruitment, follow-up, or data completeness is unknown.
Interim analyses and stopping rules are not reported in the registry record. Nor is a statistical analysis plan reported. These absences do not establish that such procedures were or were not used; they mean the posted record does not document them. Without endpoint definitions, analysis rules, and information about missing observations, readers cannot determine how the stated outcomes were analyzed or whether the reported safety observations represent complete follow-up.
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 |
|---|---|---|---|
| Weekly Paclitaxel and Vinorelbine With GCSF Support | 10 | 38 | 26.3% |
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 |
|---|---|
| Weekly Paclitaxel and Vinorelbine With GCSF Support | 38 of 38 |
Most frequent serious adverse event terms
| Event term | Organ system | Weekly Paclitaxel and Vinorelbine With GCSF Support |
|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | 4 of 38 |
| Mucositis | Infections and infestations | 2 of 38 |
| Febrile Neutropenia | Infections and infestations | 2 of 38 |
| Aspiration Pneumonia | Respiratory, thoracic and mediastinal disorders | 1 of 38 |
| Congestive Heart Failure | Cardiac disorders | 1 of 38 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Weekly Paclitaxel and Vinorelbine With GCSF Support |
|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | 20 of 38 |
| Thrombocytopenia | Blood and lymphatic system disorders | 16 of 38 |
| Vomiting | Gastrointestinal disorders | 4 of 38 |
Serious events with the highest arm-level rate: leukopenia (10.5%), mucositis (5.3%), febrile neutropenia (5.3%), aspiration pneumonia (2.6%), congestive heart failure (2.6%).
The safety summary records 38 participants at risk for serious adverse events, of whom 10 were affected. For other adverse events, 38 were at risk and 38 were affected. The listed leading serious event terms are leukopenia at 10.5%, mucositis at 5.3%, febrile neutropenia at 5.3%, aspiration pneumonia at 2.6%, and congestive heart failure at 2.6%. These percentages are reported as written in the registry record.
These figures are descriptive summaries for the recorded treatment group. They do not show whether an event was caused by treatment, and there is no listed control group against which to compare event frequency. The registry record does not explain whether each percentage represents participants or event occurrences, whether participants could have multiple listed events, or what observation period applies to these summaries. It also does not provide event grades or detailed attribution rules. Deaths are not reported in the registry record; this should not be read as evidence that no deaths occurred.
General statistical explanation: serious adverse events and other adverse events are distinct reporting categories, and a participant may experience more than one event. Counts and percentages should therefore be interpreted according to the study’s definitions and reporting rules. Those definitions are not reported in the registry record, limiting interpretation beyond the recorded totals and terms.
12. What the Result Does, and Does Not, Mean
The record documents a Phase I–II investigation of weekly paclitaxel and vinorelbine with daily G-CSF support, with Herceptin for patients with HER-2/neu positive disease. It lists 38 enrolled participants, describes several paclitaxel dose levels, and gives selected adverse event counts and terms. These details show what regimen and objectives were registered and what safety information was posted.
The record does not report a primary result for response, time to progression, or survival. It therefore does not quantify how many participants met a response definition, how long participants remained free from progression, or what survival was observed. No confidence intervals or inferential analyses are reported for the primary outcomes. This is an absence of reported results in the registry record, not evidence that the outcomes had any particular value.
The study is single-group and has no listed concurrent standard-therapy arm. Although the brief summary describes an interest in comparison with standard therapy, the recorded design cannot provide a randomized treatment contrast. Any comparison with a separate study or historical experience would require additional information and assumptions not reported in the registry record.
The safety counts describe events recorded among participants in the treatment group, but they do not establish that the regimen caused those events or whether its event frequency differs from another treatment. Further information about event definitions, follow-up, attribution, and completeness is not reported in the registry record.
The terminated status is recorded, but the reason is not reported. Without the primary outcome analyses, the study’s effect estimates and their precision cannot be assessed. The record supports a description of the registered design and posted safety observations, not a conclusion about comparative effectiveness or the magnitude of clinical benefit or harm.
13. Why This Trial Matters Statistically
This trial illustrates why a stated clinical question and the recorded design need to be considered together. The brief summary asks whether a combination regimen is more effective than standard therapy, but the study is registered as single-group and lists no concurrent control arm. A study can describe outcomes in a treated group, but a direct comparative claim requires a suitable comparison and an analysis that addresses differences between groups.
The registry also combines dose-finding aims with response, progression, survival, and toxicity objectives. These questions require different kinds of information. Dose decisions depend on explicit dose-level rules and toxicity definitions; response requires assessment criteria; and time-to-event outcomes require event and follow-up definitions. Those details are not reported in the registry record, and no primary outcome analyses are posted.
The safety record provides useful descriptive information: 10 participants are recorded as affected by serious adverse events, while 38 are recorded as affected by other adverse events. However, event counts without a control group and without full reporting definitions do not establish causation or a relative safety profile. This distinction is central to interpreting adverse event summaries responsibly.
The record’s terminated status, missing primary outcome results, and absent statistical plan also show how registry reporting affects interpretability. A reader needs outcome estimates, their uncertainty, analysis populations, missing-data methods, and endpoint definitions to evaluate results. When these are not reported, conclusions must remain limited to documented design features and descriptive safety information. The trial therefore matters statistically as an example of the boundary between registered aims, descriptive observations, and evidence for a comparative treatment effect.
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 important trial-specific limitation in the registry record is the absence of reported primary outcome analyses. Although response rates, time to progression, and survival are named as objectives, no results or estimates are reported for them. The record also reports zero analyses, so the intended outcome questions cannot be evaluated from the posted results.
The single-group design has no listed concurrent comparator, despite the stated interest in comparison with standard therapy. It cannot by itself establish a treatment difference relative to standard therapy. The record lists 38 enrolled participants but does not describe how many received treatment, how many completed follow-up, or how many contributed to each endpoint.
Important methodological details are not reported in the registry record, including eligibility criteria beyond the condition, dose-escalation rules, response and progression definitions, follow-up procedures, the analysis population, missing-data handling, and the statistical analysis plan. The reason for termination is also not reported. The safety summaries do not specify event grading, attribution, observation period, or whether participant counts or event counts are used for each listed term. Deaths are not reported in the registry record.
These limitations prevent assessment of outcome precision, endpoint ascertainment, and the completeness of follow-up. They also restrict interpretation of the safety counts to the descriptive information recorded. The gaps do not establish that procedures were absent or that outcomes were unfavorable; they indicate that those details and results are not documented in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Navelbine, Taxol, Herceptin and Neupogen in Stage IV Breast Cancer: A Phase I - II Trial — NCT00041470.
- Registry record last updated 2017-07-17; status last verified 2017-06.
- Results first posted 2017-06-06.
- 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
The registry record identifies NCT00041470 as a Phase I–II, single-group trial sponsored by the University of Washington. It lists 38 participants, no masking, a start date of 2001-03, a primary completion date of 2008-08, and status terminated.
The treatment description is weekly intravenous paclitaxel and vinorelbine with daily G-CSF support, with Herceptin for patients with HER-2/neu positive disease. The stated aims include dose finding, toxicity assessment, response, time to progression, and survival. No separate control group is listed.
Safety information records 10 of 38 participants affected by serious adverse events and 38 of 38 affected by other adverse events. The reason for termination, primary outcome results, statistical analysis plan, and analysis population are not reported in the registry record.