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 Docetaxel and Lapatinib in Metastatic Transitional Cell Carcinoma in Bladder (NCT01382706), a na phase 2 study. The primary outcome is Progression-free Survival Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01382706 |
| Phase | Phase 2 |
| Condition | Recurrent Bladder Cancer, Stage III Bladder Cancer, Stage IV Bladder Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Southern California |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic transitional cell carcinoma of the bladder were the target population. The registry lists recurrent, stage III and stage IV bladder cancer among the conditions, while the brief summary describes stage IV disease that cannot be removed by surgery and second-line treatment. Enrollment was 15. The eligibility criteria and characteristics of enrolled participants are not reported in the registry record.
Intervention
The treatment group received docetaxel and lapatinib ditosylate. The registry describes docetaxel by intravenous infusion over 1 hour on day 1 and lapatinib ditosylate by mouth once daily on days 1–21. Courses were scheduled to repeat every 21 days until disease progression or unacceptable toxicity.
Comparator
There was no control group. The study is recorded as a single-group, non-randomized trial, so it does not provide a concurrent comparison with another treatment, placebo or usual care. An effect estimate comparing the regimen with an alternative cannot be derived from this study design alone.
Primary question
The primary outcome was progression-free survival, described as the time from treatment start to documented progression or death without documented progression. The registry lists a median measure and a 95% confidence interval, with a timeframe of “At 12 weeks.” It does not report the median, confidence interval or a treatment-effect estimate.
In statistical terms, the study asks how progression-free survival was characterized among participants receiving this combination in the specified treatment setting. Because only one group is described, the question is about outcomes observed under the study regimen, not whether the regimen performs better than another option. The registry record also lists incidence of Grade 3 or higher serious adverse events as a secondary outcome. It does not report a numerical result for that secondary outcome as a distinct endpoint. The record does list serious adverse-event figures, which require careful interpretation because timing, attribution and the details of event counting are not given.
3. Trial Design
This was a phase II, single-group study sponsored by the University of Southern California. It was designed to examine docetaxel and lapatinib ditosylate as second-line treatment for metastatic transitional cell carcinoma of the bladder. Allocation is recorded as not applicable, and masking as none. The registry lists enrollment of 15 and the study status as terminated. The recorded start date is 2011-06-13, and the primary completion date is 2013-07-15.
Participants in the treatment group were scheduled to receive docetaxel intravenously over 1 hour on day 1 and lapatinib ditosylate orally once daily on days 1–21. Courses were planned to repeat every 21 days until disease progression or unacceptable toxicity. The registry also lists immunohistochemistry staining, fluorescence in situ hybridization and laboratory biomarker analysis among the interventions. It does not give further detail about their role or how their results were analyzed.
The single-group structure is central to interpreting the evidence. Every participant was assigned to the same treatment group, and no concurrent control group is described. Consequently, outcomes within the group can be described, but the design does not support a randomized, like-for-like comparison with another treatment. Differences from external groups, if considered, would be vulnerable to differences in patient selection, care and follow-up; the registry record does not report such a comparison.
The study is marked terminated. The reason for termination, the number who completed treatment, follow-up duration and disposition of participants are not reported in the registry record. The record indicates 15 enrolled, but enrollment alone does not establish how many participants contributed data to each endpoint. Analysis populations and exclusions are also not reported. These omissions limit the ability to assess whether the primary outcome reflects all enrolled participants or a subset.
The registry record gives a primary outcome definition and planned statistical summary but does not report a primary result. It also records no analyses. Accordingly, the study design and measurement plan can be described, but the treatment-group outcome cannot be quantified from 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 | Treatment |
|---|---|
| STARTED | 15 |
| COMPLETED | 13 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Treatment |
|---|---|
| Adverse Event | 1 |
| Withdrawal by Subject | 1 |
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 | Treatment |
|---|---|---|
| Participants analysed | 15 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 7 |
| Age, Categorical: >=65 years | Participants | 8 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 12 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 14 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 11 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival Rate, measured over At 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
The primary outcome is titled “Progression-free Survival Rate,” and is described as the time from the start of treatment to documented disease progression or death without documented progression. This is a time-to-event endpoint: each participant contributes time until the event occurs or, if no event is observed during their available follow-up, until their observation is censored. The registry says the endpoint is summarized with Kaplan–Meier curves and that the median is estimated using a non-parametric method.
Time-to-event outcomes are used when both whether an event occurred and when it occurred matter. They can incorporate participants whose follow-up ends before an event is documented, rather than treating all such participants as if they had the same follow-up. This approach depends on a valid definition of the event and appropriate handling of censoring. General statistical considerations include whether censoring is independent of the chance of the event, and whether the event dates and follow-up are recorded consistently.
The registry lists the timeframe as “At 12 weeks,” while also describing a median time-to-event measure. It does not provide a numerical median, confidence interval, number of events, or Kaplan–Meier estimate. Nor does it explain whether the timeframe refers to an assessment point, a planned follow-up period or another convention. The exact interpretation of that timeframe therefore cannot be clarified from the registry record.
A median and its confidence interval, if reported, would describe the time-to-event distribution and its precision in the study group. They would not, by themselves, establish a comparative treatment effect in this single-group study. No such numerical endpoint result is reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a phase II study of docetaxel and lapatinib ditosylate in one treatment group, with enrollment of 15. It identifies progression-free survival as the primary outcome and specifies a time-to-event definition and Kaplan–Meier summary. However, no numerical primary endpoint result appears in the registry record. There is no reported median progression-free survival, 95% confidence interval, event count or hazard ratio. The record also lists no analyses. As a result, the primary outcome cannot be quantitatively interpreted from the registry information.
The lack of a concurrent comparator sets a second boundary on interpretation. Even if the registry included a numerical outcome for this group, a single-group estimate would describe participants receiving the regimen, not the difference between the regimen and another treatment. It would not distinguish a treatment-associated outcome from the course of disease, participant selection, other care or other influences. The design therefore cannot answer a comparative efficacy question on its own.
The study status is terminated. The reason for termination and its relationship, if any, to enrollment, safety, feasibility or efficacy are not reported in the registry record. It would be inappropriate to infer a reason from the status alone. Similarly, enrollment of 15 does not tell how many participants were evaluable for progression-free survival, how much follow-up was available, or how many events occurred.
The record does provide serious adverse-event information for the treatment group. It lists 15 participants at risk and 15 affected for serious events, and separately records 11 deaths. Serious-event terms include diarrhea at 33.3%, nausea and vomiting each at 26.7%, and anemia, decreased neutrophil count and decreased white blood cell count each at 13.3%. These are descriptive entries, not a comparison with another group. The registry record does not give event timing, duration, recurrence, attribution to treatment, or the relationship of the listed deaths to treatment or adverse events. The death count therefore must not be interpreted as a treatment-caused death count.
For all outcomes, precision and completeness matter. A confidence interval, when available, indicates the range of values compatible with the data under the analysis assumptions; a wide interval signals uncertainty. Here, the primary confidence interval is listed as a planned summary but its limits are not reported. The small enrollment makes detailed characterization difficult, but no precision estimate can be reconstructed from the record without inventing data.
The reasonable statistical conclusion is limited: the registry describes the intended study question, design, outcome definition and some safety entries, but it does not report the primary result or analysis. It does not establish the magnitude or precision of progression-free survival in the study group, and it cannot support a controlled comparison.
9. Statistical Methods Explained
The registry describes a time-to-event endpoint, a single treatment group and a planned Kaplan–Meier summary. These details indicate the general analytic framework, but the record does not give the numerical results or a complete statistical analysis plan. General explanations of the methods clarify what the planned measures mean; they do not fill in missing trial results.
What does a time-to-event endpoint measure?
A time-to-event endpoint combines information about whether an event occurred with the time from a defined starting point to that event. In this study, the starting point is treatment initiation. The event is documented progression or death without documented progression. A participant with no recorded event by the end of available observation may be censored at their last relevant follow-up. Censoring means the exact event time is not known beyond that point; it does not mean the participant had no event ever.
Time-to-event methods are useful when participants have different follow-up durations or when not all have an event during observation. They use the observed follow-up rather than reducing the outcome to a simple event/no-event count. Their validity depends on sound event definitions, accurate dates and appropriate assumptions about censoring. The registry gives the endpoint definition but does not report event counts, follow-up details, censoring information or how these elements were checked.
What does Kaplan–Meier estimation do?
Kaplan–Meier estimation is a non-parametric method for estimating the event-free proportion over time while accounting for censoring. At each observed event time, the estimate reflects the proportion of participants still event-free among those under observation just before that time. The resulting curve describes the estimated progression-free survival experience in the group. It is a descriptive estimate for this study population; without a comparator it does not quantify a relative treatment effect.
The median time-to-event is the time at which the estimated event-free proportion reaches one half, if the curve reaches that point. A median may not be estimable if the observed curve does not reach that threshold. The registry says a median is to be estimated using a non-parametric method, but it does not report whether the median was estimable or give its value. The endpoint title includes “Rate,” while the stated parameter is a median time. The record does not clarify this naming distinction.
How should a confidence interval be read?
A confidence interval expresses statistical uncertainty around an estimate under the assumptions of the method. It is not a guarantee that the true value lies within the particular interval, nor does it account automatically for every source of bias or missing information. For a median, the confidence interval would indicate the precision of the estimated time-to-event summary. The registry lists a 95% confidence interval as the dispersion measure, but its limits are not reported. Therefore, the precision of the primary estimate cannot be assessed.
Precision is not the same as clinical importance, and a confidence interval is not a substitute for a control group. A narrow interval around a descriptive estimate would not remove the design limitation of having no concurrent comparison. Conversely, a broad interval would indicate substantial statistical uncertainty even about the group’s own outcome. No interval can be characterized for this trial from the registry record.
Why does the single-group design matter?
In a randomized comparative trial, assignment to separate groups is intended to make treatment groups comparable, subject to the design and conduct. This study is recorded as single-group, with allocation not applicable and no masking. There is no treatment contrast within the trial. Consequently, no between-group difference, relative risk or treatment hazard ratio can be estimated from the described design. External comparisons would require additional data and assumptions; the registry does not report any.
Unmasked treatment can also affect how care, outcome assessment or reporting occurs, depending on the outcome and procedures. The record does not state whether progression was assessed by blinded reviewers or describe assessment procedures. This is a limitation of the available description, not evidence that assessment was biased.
What can be inferred from the analysis record?
The registry records zero analyses and does not report a primary endpoint estimate. It does not provide the statistical plan, analysis population, missing-data approach, censoring rules, subgroup analyses, or methods for secondary outcomes. The endpoint description says Kaplan–Meier curves and a non-parametric median estimate, but this is not enough to establish exactly how the analysis was implemented. No hazard ratio is reported; therefore no hazard-ratio interpretation applies.
The registry also includes a generic sample-size planning illustration based on an observed effect and states assumptions for a two-arm comparison. Such planning illustrations are not observed trial results, and they are not evidence that the study had a particular level of power. The study is single-group. No numerical illustration values appear in the registry record, and the record does not report the trial’s own sample-size justification. It is therefore not possible to assess whether enrollment was selected to achieve a specified precision or power.
In general, over-dispersion occurs when variation in data is greater than expected under a chosen model; it can make standard errors too small if ignored. That concept does not establish a problem in this trial. The registry does not describe a count model or report a method that would permit an over-dispersion assessment. Likewise, no recalculated estimates are available to compare with a sponsor-reported analysis, and there is no reported analysis to take precedence over a recalculation.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome and one secondary outcome: incidence of Grade 3 or higher serious adverse events. It does not report a multiplicity strategy, such as a hierarchy of hypotheses or an adjustment for testing several outcomes. It also does not report p-values or analyses. Accordingly, there is no basis for judging how multiple testing was handled or for interpreting statistical significance across endpoints. The outcome list alone does not show that formal hypothesis tests were conducted.
Missing-data methods and analysis populations are not reported in the registry record. The record does not state whether the primary analysis was intended to include all enrolled participants, only those who received treatment, or another defined group. It does not describe how incomplete progression assessments, withdrawals, missed visits or other unavailable data were handled. For a time-to-event endpoint, the distinction between an event and censoring is important, but the relevant censoring details are not available.
The registry records enrollment of 15, but does not state how many participants contributed to the primary endpoint analysis or how much follow-up was available. The number enrolled should not be assumed to equal the number analyzed. The status is terminated, but reasons for termination and participant disposition are not reported. These gaps prevent assessment of attrition, missingness, selective reporting or the degree to which the analyzed population represents everyone enrolled.
The registry also does not report a complete statistical analysis plan, interim analyses, stopping rules or outcome-specific analysis methods beyond the stated Kaplan–Meier approach for the primary endpoint. These matters are not evidence of a particular practice; they are simply not reported in the registry record. Without those details and without outcome estimates, inferential claims about statistical significance or robustness are not possible.
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 |
|---|---|---|---|
| Treatment | 15 | 15 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment | 11 of 15 |
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 |
|---|---|
| Treatment | 15 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 5 of 15 |
| Nausea | Gastrointestinal disorders | 4 of 15 |
| Vomiting | Gastrointestinal disorders | 4 of 15 |
| Anemia | Blood and lymphatic system disorders | 2 of 15 |
| Neutrophil count decreased | Investigations | 2 of 15 |
| White blood cell decreased | Investigations | 2 of 15 |
| Fatigue | General disorders | 1 of 15 |
| Anorexia | Metabolism and nutrition disorders | 1 of 15 |
| Dehydration | Investigations | 1 of 15 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment |
|---|---|---|
| Fatigue | General disorders | 10 of 15 |
| Alopecia | Skin and subcutaneous tissue disorders | 9 of 15 |
| Diarrhea | Gastrointestinal disorders | 7 of 15 |
| Nausea | Gastrointestinal disorders | 6 of 15 |
| Anorexia | Metabolism and nutrition disorders | 6 of 15 |
| Anemia | Blood and lymphatic system disorders | 5 of 15 |
| Insomnia | Psychiatric disorders | 4 of 15 |
| White blood cell decreased | Investigations | 3 of 15 |
| Back pain | Musculoskeletal and connective tissue disorders | 3 of 15 |
| Dry skin | Skin and subcutaneous tissue disorders | 3 of 15 |
Serious events with the highest arm-level rate: diarrhea (33.3%), nausea (26.7%), vomiting (26.7%), anemia (13.3%), neutrophil count decreased (13.3%).
The registry lists 15 participants at risk and 15 affected for serious adverse events in the treatment group. It also lists 11 deaths. These entries describe the recorded group; they do not establish that every event was caused by treatment or that every death was related to an adverse event. The registry record does not report the timing of events, their duration, recurrence, treatment attribution or the timing and causes of the deaths.
Among the listed serious-event terms, diarrhea is reported at 33.3%; nausea and vomiting are each reported at 26.7%; anemia, decreased neutrophil count and decreased white blood cell count are each reported at 13.3%. These percentages are descriptive registry entries. They should not be read as a comparison against another treatment, because no control group is described. The record does not clarify whether these entries count participants with at least one event, individual events, or another reporting unit.
The secondary outcome is described as incidence of Grade 3 or higher serious adverse events, but a separate numerical result for that outcome is not reported. Seriousness and severity are distinct concepts in adverse-event reporting; the registry does not provide enough detail to characterize the event grades or the criteria underlying the listed terms. The entries should therefore be read as safety information recorded for the study group, not as a complete causal assessment.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II, single-group study of docetaxel and lapatinib ditosylate for second-line treatment of metastatic transitional cell carcinoma of the bladder. It specifies a progression-free survival endpoint, a time-to-event definition and a planned Kaplan–Meier summary. It also lists serious adverse-event entries for the treatment group. Those details establish what the study set out to examine and what information is recorded, but not the size or precision of the primary outcome.
The primary endpoint result is not reported in the registry record. There is no median progression-free survival, confidence interval, event count or hazard ratio to interpret. The record also lists no analyses. Therefore, the registry information does not quantify progression-free survival for the enrolled group, and it does not show whether a particular estimate is precise or uncertain.
The study has no concurrent comparison group. This means the record cannot establish how outcomes under the regimen compare with outcomes under another treatment or with no treatment. A single-group observation cannot separate the effect of treatment from other factors that influence outcomes. The design also cannot support a randomized treatment contrast.
The safety entries should likewise be interpreted descriptively. Serious adverse events and deaths are recorded for the treatment group, but the registry does not state whether they were attributed to treatment. It also does not provide event timing or enough detail to assess the full safety profile. Neither the counts nor listed percentages alone establish causation.
More broadly, the study status, enrollment and outcome definitions do not reveal why the study was terminated, how many participants completed follow-up, or how missing observations were handled. Those details are not reported in the registry record. Statistical interpretation must remain bounded by these omissions: the record describes a study and selected safety data, but it does not provide the primary results needed to estimate the observed progression-free survival or its uncertainty.
13. Why This Trial Matters Statistically
This trial illustrates why design and reporting details are essential to statistical interpretation. The study has a clearly identified treatment group and a time-to-event primary outcome, but there is no concurrent comparator. That distinction changes the question that can be answered: a single-group study can describe outcomes in its participants, whereas a controlled comparison is needed to estimate a treatment contrast within the trial.
It also shows the difference between defining an outcome and reporting a result. The registry specifies progression-free survival as time from treatment start to progression or death without documented progression, and names Kaplan–Meier estimation and a median. Those methodological details do not provide an estimate. Without the median, confidence interval, event count and follow-up information, the outcome cannot be assessed quantitatively.
Safety reporting needs similar care. The registry lists affected participants, deaths and serious-event terms, but does not report timing or causal attribution. Descriptive event figures are useful to identify what was recorded, yet they do not determine whether the regimen caused an event or how its safety compares with another option.
Finally, the record’s terminated status and enrollment of 15 do not explain the study’s completion or analysis. Without participant disposition, analysis populations, missing-data procedures and the reason for termination, readers cannot evaluate potential sources of bias or how representative the analyzed data might be. This is a useful reminder that statistical conclusions depend not only on the planned method, but also on transparent reporting of what was observed and how it was analyzed.
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 registry record leaves important trial-specific information unreported. It does not give the eligibility criteria in full, baseline characteristics, participant disposition, treatment exposure, adherence, follow-up duration or the reason the study was terminated. Enrollment of 15 is recorded, but the number contributing to each outcome is not stated. These omissions prevent a detailed assessment of who was studied and how completely outcomes were observed.
The primary outcome is defined, but its numerical result is absent. The median and 95% confidence interval are not reported, and the record gives no event count or Kaplan–Meier estimates. Although the timeframe is listed as “At 12 weeks,” the record does not explain how that phrase relates to the median time-to-event measure. This leaves uncertainty about the intended reporting point as well as the outcome itself.
The design is single-group, with no comparator and no masking. This precludes a randomized treatment comparison. The registry does not explain whether progression assessment was independently reviewed or whether outcome assessors were blinded. No claim about the presence or absence of assessment bias can be made from that omission.
Analysis details are also limited. The record does not report an analysis population, missing-data or censoring procedures, a complete statistical plan, interim analyses, or a multiplicity approach. It lists no analyses and provides no primary estimate. The stated method is therefore a description of the intended summary, not evidence about how the data were ultimately analyzed.
Safety information is incomplete for causal interpretation. Serious adverse-event entries and deaths are recorded, but event timing, attribution, grading detail and the relationship between deaths and treatment are not reported. The record does not provide a comparator safety profile. These limitations mean that the listed entries should not be treated as a complete assessment of treatment-related risk.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Docetaxel and Lapatinib in Metastatic Transitional Cell Carcinoma in Bladder — NCT01382706.
- Registry record last updated 2025-10-09; status last verified 2025-09.
- Results first posted 2025-10-09.
- 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 describes a phase II, single-group study of docetaxel and lapatinib ditosylate as second-line treatment for metastatic transitional cell carcinoma of the bladder. Enrollment was 15. The study is marked terminated; the recorded start date is 2011-06-13, and the primary completion date is 2013-07-15. There was no randomized comparator or masking.
The primary outcome was progression-free survival, defined from treatment start to documented progression or death without documented progression, with a planned Kaplan–Meier summary and median estimate. The primary result and its 95% confidence interval are not reported in the registry record, which also lists no analyses. Serious adverse-event information is recorded for the treatment group, including 11 deaths and listed event terms, but timing and treatment attribution are not reported. The reason for termination, participant disposition and analysis populations are also not reported.