Dasatinib in Treating Patients With Stage IIIB, Stage IV, or Recurrent Non-Small Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Dasatinib in Treating Patients With Stage IIIB, Stage IV, or Recurrent Non-Small Cell Lung Cancer (NCT00459342), a na phase 2 study. The primary outcome is Number of Participants With Objective Response (Complete Response (CR) or Partial Response (PR)).

2. Clinical Question

The study examined dasatinib in people with advanced or recurrent non-small cell lung cancer. Its primary endpoint was an objective-response count: participants were classified as having a complete response or partial response if their tumor findings met the relevant RECIST definition. The record describes assessment using tumor size on computed tomography scans and metabolic activity on positron emission tomography scans.

Because the study had a single group, its primary question concerns the response observed among participants who received dasatinib. It does not directly compare outcomes against a concurrent control group. A response count, even when reported, would describe the observed participants and would not by itself establish what would have happened to comparable participants without the study treatment.

The registry record gives some information about the population available for response assessment: 34 participants were eligible, and four were not evaluable for response. It does not report the number who achieved complete response or partial response, nor does it report a response percentage or uncertainty interval. Therefore, the main endpoint cannot be quantified from the registry record.

3. Trial Design

This was a phase II study of dasatinib in non-small cell lung cancer. The registry lists a single-group model, no allocation, and no masking. The study status is completed. Enrollment was 35, and the listed intervention was dasatinib. The registry gives a start date of 2007-03 and a primary completion date of 2012-04.

Participants received oral dasatinib twice daily on days 1–21, with treatment repeating every 21 days in the absence of disease progression or unacceptable toxicity. The population described in the study title and conditions included stage IIIB, stage IV, and recurrent non-small cell lung cancer. The registry's primary-outcome description states that 34 enrolled participants were eligible and that four were not evaluable for response.

A single-group design means that the reported treatment group is not accompanied by a concurrent group assigned to a different treatment or to no study treatment. This describes the structure, not the reason the investigators selected it. The reason for the design, the intended role of any external comparison, and the detailed protocol rationale are not reported in the registry record.

The record lists six outcome measures and no analyses. It does not report a randomization procedure, a masking procedure, or an allocation ratio; allocation is listed as not applicable and masking as none. No sample-size justification, interim-analysis plan, or full statistical analysis plan is reported in the registry record. These absences limit what can be concluded about how the study was planned and how its endpoints were intended to be analyzed.

For this design, the primary result would ordinarily be interpreted as a description of response within the treated group, not as an estimated contrast between treatments. General statistical interpretation of a single-group outcome must account for the lack of a randomized comparator and for which participants contributed evaluable outcome data. The response result itself is not reported in the registry record, so those features cannot be used to quantify response in this study.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Of the 34 eligible study participants enrolled, four participants were not evaluable for response.

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Objective Response (Complete Response (CR) or Partial Response (PR)), measured over 12 weeks.

The primary endpoint is the number of participants with an objective response, defined as complete response or partial response, assessed at 12 weeks. The registry describes response assessment under RECIST. A complete response means disappearance of all target lesions. A partial response means at least a 30% decrease in the sum of the longest diameters of target lesions, using the baseline sum as the reference. The record also defines progressive disease as at least a 20% increase from the smallest sum recorded since treatment started, or the appearance of one or more new lesions. Stable disease is neither enough shrinkage for partial response nor enough increase for progressive disease, using the stated reference.

The endpoint converts tumor assessments into a response classification and then counts participants meeting either of the two response categories. In general, a binary endpoint can make the outcome straightforward to describe, while losing detail about the degree and pattern of change within each category. The registry describes assessment using tumor size on computed tomography scans and metabolic activity on positron emission tomography scans.

The response count is not reported in the registry record. Nor is a response proportion or confidence interval reported. The stated assessment time is 12 weeks, but the record does not give additional details about assessment timing, handling of assessments outside that time, or how discordant imaging information would be resolved.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

General statistical explanation: a clinical endpoint is a defined outcome used to describe a study question. Here, the primary endpoint is a count of participants with complete or partial response by 12 weeks. The registry's definition ties those categories to RECIST criteria and describes assessment by computed tomography tumor size and positron emission tomography metabolic activity. The endpoint is binary at the participant level: each assessed participant either meets the objective-response definition or does not. The posted endpoint is then expressed as a count.

What does a count tell the reader?

A count gives the number of participants classified as having an event or outcome. For a response endpoint, the count alone is not the same as a response rate. A rate or proportion requires a clearly specified denominator, such as the number of participants included in the response analysis. In this record, 34 participants are described as eligible and four of them as not evaluable for response, but the response count and final analysis denominator are not reported. Those statements should not be combined to create an unreported estimate.

General statistical explanation: a confidence interval describes a range of values compatible with the observed data under the method used, reflecting sampling uncertainty. It helps readers assess the precision of an estimate; it does not repair limitations in the design or guarantee that the range contains a true value. No confidence interval for objective response is reported in the registry record. No inferential analysis is reported, so no associated test or model can be evaluated.

Why does the single-group design matter?

In a single-group study, outcomes are observed among participants receiving the study intervention without a concurrent randomized comparison group. The design can describe the experience of the enrolled group, but it does not directly estimate a treatment contrast. General statistical explanation: random allocation, when used, can help balance measured and unmeasured participant characteristics between groups on average. Without such a comparison, differences between the observed outcome and an external expectation can be difficult to attribute to treatment. This trial's record lists no allocation and a single-group model, so no randomized between-group estimate is available.

General statistical explanation: masking is intended to reduce the influence of knowledge about treatment assignment on aspects of care, assessment, or reporting. The registry lists masking as none. This fact describes the record's design classification; it does not show whether or how assessment decisions were affected. The record does not report additional safeguards or procedures for response assessment.

How should evaluability be considered?

Participants who cannot be evaluated for an endpoint may affect both the denominator and interpretation of a response estimate. The registry states that four eligible participants were not evaluable for response, but does not report why, how their outcomes were handled, or which analysis population was used. General statistical explanation: excluding participants can change the population represented by an estimate, while treating missing outcomes as nonresponse is a different analytic choice. Neither approach should be assumed unless it is stated. The record does not specify either approach.

General statistical explanation: binary outcomes can be analyzed with methods for proportions or with regression models, but the choice depends on the question and the design. A single-group count could be described directly if the numerator and denominator were available. More complex adjustment would require a stated model and relevant data. The registry reports no analyses, so it is not possible to identify which methods were used, whether any adjustment was applied, or whether any assumptions were checked.

What does the safety summary represent?

General statistical explanation: adverse-event summaries typically distinguish the number of participants at risk from the number who experienced an event, and may separately list event terms. Those quantities are not interchangeable. A participant may experience more than one event, so a participant count does not necessarily represent the total number of event episodes. The serious-event summary lists participants at risk and affected; the record does not provide event-level totals or information sufficient to reconstruct them. The listed event percentages should be read as the values recorded for those terms, not as a comparative risk estimate.

General statistical explanation: over-dispersion is relevant when observed variability exceeds what a chosen statistical model expects. It can affect uncertainty estimates in analyses of counts. The primary endpoint is described as a participant count, but no analysis model or variance estimate is reported, and the record does not provide the outcome count needed to assess variability. Therefore, over-dispersion cannot be evaluated for this result from the registry record.

Finally, a sample-size justification and analysis plan are important for understanding what a study was designed to estimate and how outcomes were to be evaluated. Neither is reported in the registry record. The record lists enrollment of 35, but that number alone does not reveal the target effect, planned precision, or rationale for enrollment. No observed power should be inferred from enrollment, and no sample-size calculation can be reconstructed from the information reported.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists six outcome measures, but no analyses are reported. It does not report whether the primary endpoint was tested against a prespecified benchmark, whether secondary outcomes were analyzed inferentially, or whether any multiplicity adjustment was planned or applied. The registry record also does not give a statistical analysis plan or identify any hierarchy among the outcomes beyond naming the primary endpoint.

General statistical explanation: when several outcomes or hypotheses are tested, the chance of at least one apparently notable result can increase if each is judged separately without accounting for the number of tests. Methods to address multiplicity depend on the questions and analysis plan. Since no analyses or multiplicity approach are reported, no claim about adjusted or unadjusted inference can be made for this study.

The record notes that 34 participants were eligible and four were not evaluable for response. It does not explain the reasons for non-evaluability, report a missing-data method, or specify how those participants were treated in the primary analysis. It also does not state whether the primary analysis included all enrolled participants, eligible participants, or another defined population. No intention-to-treat or per-protocol analysis population is reported.

General statistical explanation: missing outcomes can affect an estimate when the probability of missingness is related to participant characteristics or outcomes. Different assumptions and methods can lead to different interpretations. Without the reasons for missingness and the analytic approach, the effect of the four unevaluable responses cannot be assessed. The response count itself is not reported in the registry record, so neither the endpoint estimate nor its sensitivity to missing data can be evaluated.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 2% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: dyspnea (42.9%), pleural effusion (17.1%), lung infection (8.6%), dehydration (5.7%), fatigue (5.7%).

The registry reports 35 participants at risk for serious adverse events, of whom 25 were affected. For other adverse events, it reports 35 at risk and 34 affected. These are participant counts in the dasatinib group. The record lists serious adverse-event terms and their recorded percentages: dyspnea, 42.9%; pleural effusion, 17.1%; lung infection, 8.6%; dehydration, 5.7%; fatigue, 5.7%; and thromboembolic event, 5.7%.

These summaries should be read as safety descriptions, not as estimates of comparative risk. The study has no concurrent comparator, so the figures do not show how often these events would have occurred under another treatment or without study treatment. The registry record does not report a causal assessment for the listed events. It also does not report the total number of event episodes, timing, duration, severity grades, or participant-level overlap among event terms in the information.

The record does not report a death count: the death value is not reported in the registry record. The affected-participant counts and listed terms should not be added together, since the same participant could be represented in more than one category and the record does not give the overlap. These details constrain interpretation of the safety summaries.

12. What the Result Does, and Does Not, Mean

The registry record establishes that a phase II, single-group study of dasatinib was completed in people with stage IIIB, stage IV, or recurrent non-small cell lung cancer. It identifies objective response at 12 weeks as the primary endpoint and defines complete and partial response using RECIST criteria. It also reports enrollment, eligibility and evaluability information, and selected adverse-event counts.

The key limitation for interpreting the primary question is that the objective-response result is not reported in the registry record. Without the count of participants meeting the response definition, the response proportion, and an uncertainty estimate, the endpoint cannot be quantified. The endpoint definition describes how response was to be classified; it does not disclose how many participants met that classification.

Even if a response count were available, a single-group result would describe the observed group rather than establish a causal contrast against another treatment. General statistical explanation: an observed outcome does not by itself isolate the effect of an intervention from other influences on outcome. A randomized comparison can support a different type of inference, but the registry lists a single-group model and no allocation.

The safety information similarly describes reported events in the treated group. It does not provide a comparison that would allow the listed counts or percentages to be interpreted as excess risk attributable to dasatinib. The record's counts should therefore be read as registry safety summaries, with the stated denominators and limitations, rather than as evidence for or against a treatment effect.

In short, the record is informative about the trial's design and endpoint definition, but it does not report the primary outcome result or analyses. It cannot support a numerical conclusion about objective response, its precision, or a between-treatment difference. Those absences are distinct from a finding of no response or no effect; the registry record does not report such a finding.

13. Why This Trial Matters Statistically

This trial illustrates why an endpoint definition and an endpoint result are separate parts of a statistical report. The registry describes objective response in detail, including the RECIST categories and assessment timeframe, but does not report the observed response count. Without the result and its denominator, readers cannot estimate the response proportion or assess its precision.

It also demonstrates the interpretive limits of a single-group design. A treated-group outcome can be described, but it does not supply a randomized comparison. This matters because treatment-effect questions concern a contrast between outcomes under different conditions, while a single-group study records one group's experience. The registry lists no allocation and no comparator.

The eligibility and evaluability statements highlight another central issue: an analysis population must be defined to interpret a count. The record reports 34 eligible participants and four not evaluable for response, but does not state how response analysis handled these participants. Reporting this information clearly, together with the numerator and denominator, is essential for understanding what an estimate represents.

Finally, the record lists six outcomes, no analyses, and no reported statistical analysis plan or sample-size justification. These omissions prevent assessment of multiplicity handling, analytic choices, and the intended precision or target effect. The trial therefore offers a useful example of how registry design and outcome definitions can be present while key elements needed for quantitative interpretation remain unreported.

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 primary limitation is that the objective-response result is not reported in the registry record. The response count, response proportion, confidence interval, and any inferential result are absent. The record's detailed endpoint definition cannot replace those observed results.

The primary analysis population is also unclear. The record states that 34 participants were eligible and four were not evaluable for response, but it does not explain why they were not evaluable, define the final response denominator, or describe how those participants were handled. The number of evaluable participants should not be inferred from these statements alone.

The single-group design has no concurrent comparator, and the registry lists no allocation and no masking. Thus, even a reported response count would not provide a randomized comparison. The record does not describe an external comparison or a rationale for interpreting outcomes against a benchmark.

Other limitations include the absence of reported analyses, a statistical analysis plan, a sample-size justification, a multiplicity approach, and an interim-analysis plan. The registry lists six outcome measures but does not report how they were analyzed. The safety summary does not report a death count, event-level totals, timing, or overlap among the listed event terms. These omissions constrain both efficacy and safety interpretation.

These limitations reflect information not reported in the registry record; they do not establish that a particular analysis was not conducted outside the record. No additional results or analytic details can be inferred from the study status or enrollment count.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Dasatinib in Treating Patients With Stage IIIB, Stage IV, or Recurrent Non-Small Cell Lung Cancer — NCT00459342.
• Registry record last updated 2019-03-05; status last verified 2019-02.
• Results first posted 2013-12-24.
• 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.

18. Record Summary

This completed phase II study evaluated dasatinib in a single group of people with stage IIIB, stage IV, or recurrent non-small cell lung cancer. Enrollment was 35. Participants received oral dasatinib twice daily on days 1–21, with treatment repeating every 21 days in the absence of disease progression or unacceptable toxicity. The primary endpoint was the number of participants with complete or partial response at 12 weeks, assessed using RECIST.

The registry states that 34 participants were eligible and four were not evaluable for response. The objective-response result and analyses are not reported in the registry record. Safety summaries list 25 of 35 participants affected by serious adverse events and 34 of 35 affected by other adverse events. The record has no comparator group, and the primary response result cannot be quantified from the information reported.