Cisplatin and Nab-paclitaxel for (N2) Defined NSCLC: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Cisplatin and Nab-paclitaxel for (N2) Defined NSCLC (NCT02276560), a non-randomized phase 2 study. The primary outcome is Rate of N2 Nodal Clearance.

2. Clinical Question

3. Trial Design

This was a phase II, non-randomized, single-group study with no masking. Its official title describes a multicenter trial of neoadjuvant cisplatin and nab-paclitaxel for N2-defined Stage IIIA non-small cell lung cancer. The intended treatment sequence placed chemotherapy before surgery, with further adjuvant treatment options also described in the registry. The record lists cisplatin and nab-paclitaxel as neoadjuvant drugs and lists adjuvant regimens involving cisplatin with nab-paclitaxel, pemetrexed or gemcitabine.

The study began in 2015-01, and its primary completion date is recorded as 2016-06. The status is terminated. Enrollment was 1, and the record says that the only patient registered before funding was withdrawn could not complete treatment due to adverse events. This is a crucial design and interpretation detail: the planned phase II study did not produce a completed treatment cohort from which the primary endpoint could be assessed.

Because there was no random allocation and the model was single group, the registry does not support a treatment-versus-control estimate. The listed adjuvant options are not evidence of distinct randomized arms or of a comparison between strategies. No participant allocation details beyond the single registered patient are reported in the registry record.

The registry record does not report a detailed statistical analysis plan, a sample-size justification for the intended trial, or a planned interim analysis. The available record also does not state how the proposed study would have handled deviations from assigned treatment, assessment of nodal clearance, or missing endpoint observations. These omissions matter because a trial's design and analysis plan determine how an endpoint is measured and how uncertainty is evaluated. With one registered participant who could not complete treatment and no endpoint analysis, the record is descriptive of the study's status and the patient's treatment completion, not an efficacy evaluation.

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: The only patient registered before funding was withdrawn could not complete treatment due to adverse events.

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 Rate of N2 Nodal Clearance, measured over 3 Months.

The primary endpoint was the rate of N2 nodal clearance at 3 Months. The registry defines N2 disease as involvement of the ipsilateral mediastinal and/or subcarinal lymph nodes. Under the endpoint definition, clearance means that disease is no longer present in those locations. The measure was intended to assess disease status at the specified nodal sites following neoadjuvant treatment and by the time of surgery.

A nodal-clearance endpoint focuses on a defined anatomical finding rather than an overall clinical outcome. In general, a trial may use such an endpoint to assess whether a treatment sequence is associated with a change in disease observed at surgery. That rationale is general; the registry record does not provide evidence about how well this endpoint predicts later outcomes, nor does it report a validation analysis.

No clearance rate or patient-level endpoint result is reported in the registry record. The record states that the only registered patient could not complete treatment due to adverse events. Consequently, there is no reported completed treatment assessment from which to describe N2 clearance, and no estimate of the endpoint's precision. The planned endpoint and its definition should therefore be distinguished from an observed result: a stated study objective is not evidence that the outcome was measured or that clearance occurred.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a phase II study with non-randomized allocation, no masking and a single-group model. These terms describe the structure of the intended study, not a completed comparative analysis. The observed enrollment was 1, and the record lists 0 analyses. As a result, statistical methods that would normally summarize and compare endpoint data cannot be evaluated from a reported primary outcome analysis.

What does a single-group design estimate?

In general, a single-group study describes outcomes among participants who receive the study intervention. It does not create a concurrent randomized control group. A rate could be estimated if participants had outcome assessments and the record specified how assessed outcomes were counted. Here, the primary endpoint result is not reported in the registry record, and the sole registered patient could not complete treatment. There is therefore no reported endpoint rate to interpret.

Non-randomized allocation means that a comparison between groups, if one were attempted, would not have the protection against systematic baseline differences that random allocation is designed to provide. In this record, however, there is no reported comparative analysis. The adjuvant regimens in the intervention descriptions should not be treated as observed comparison groups: the study is identified as single group, and the registry does not report enrollment into those regimens.

What would a rate and its uncertainty mean?

Generally, a rate summarizes the share of assessed participants who meet an endpoint definition. Its interpretation depends on knowing who was assessed, how the endpoint was determined, and how missing assessments were handled. A confidence interval expresses statistical uncertainty around an estimate under a stated model and method. It does not resolve problems caused by an incomplete study or make an unobserved endpoint measurable. No primary endpoint estimate or confidence interval is reported in the registry record.

For this endpoint, assessment would concern whether N2 disease remained in the specified lymph-node locations at the relevant time. The registry supplies the endpoint definition and a timeframe of 3 Months, but it does not report an assessment result or detailed procedures for determining clearance. No adjudication process or measurement agreement information is reported in the registry record.

What can the safety counts tell readers?

Safety counts describe the number of participants recorded as at risk and affected for each category. They are not a substitute for the primary endpoint analysis. The neoadjuvant group has one participant listed at risk for serious adverse events and zero affected; for other adverse events, one is listed at risk and one affected. Counts from such a small group provide only a narrow account of recorded events. They do not characterize the range of possible event rates in a larger group, and the record does not include serious adverse-event terms.

How should planning illustrations be understood?

Sample-size calculations are planning exercises that describe how many participants might be needed under chosen assumptions to detect a specified effect with a chosen level of statistical power. They are not results from enrolled participants and are not observed power. The stated assumptions for the planning illustrations include a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of the observed effect. These assumptions describe the illustrations, not a documented design rationale for this single-group trial. The trial's own sample-size justification is not reported in the registry record. The actual trial enrolled 1, so any hypothetical sample-size planning should remain separate from the evidence generated by this study.

Over-dispersion is a general statistical issue that arises when observed variation exceeds what a simple model assumes. In general, accounting for it can affect standard errors and uncertainty estimates. The registry record does not report an over-dispersion analysis or enough endpoint data to assess whether such a method was relevant. Likewise, no adjusted model, subgroup analysis, or sensitivity analysis is reported for the primary endpoint.

What is not available for statistical review?

The registry record does not report an analysis population definition, detailed endpoint assessment procedures, missing-data methods, multiplicity control, or an inferential statistical plan. These elements ordinarily clarify which participants contribute to each analysis, how incomplete observations are treated, and how multiple statistical claims are controlled. Their absence is especially important when treatment was not completed and no endpoint result is listed. Without those details or endpoint data, readers should not infer a completed analysis from the study's stated phase or endpoint.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity concerns the risk of drawing misleading conclusions when several outcomes, comparisons, time points or subgroups are tested. In general, a prespecified analysis plan can identify which result is primary and how additional tests will be interpreted. The registry identifies one primary endpoint, Rate of N2 Nodal Clearance, with a timeframe of 3 Months. It does not report a multiplicity adjustment or a broader statistical plan for secondary analyses. The number of outcome measures is listed as 1, and the record lists 0 analyses.

Missing data are particularly consequential for a study in which the only registered patient could not complete treatment. The record does not report a primary endpoint observation, nor does it state whether an endpoint assessment was attempted or how a participant who did not complete treatment would be handled analytically. No imputation method, missing-data sensitivity analysis, or other missing-data strategy is reported in the registry record. The absence of an endpoint result should not be treated as a negative or positive endpoint observation.

The analysis population is also not defined in the registry record. General possibilities include analyzing participants according to enrollment, treatment received, or availability of endpoint assessment, but none should be assumed to be the method used for this trial. There is no reported intention-to-treat, per-protocol, or safety-population definition. Given the single registration and no primary outcome analysis, the record does not permit comparison of results across analysis populations.

These gaps limit assessment of whether the planned endpoint would have been analyzed as intended. They do not establish that a particular method was inappropriate; they indicate that the relevant methods are not reported in the registry record.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports one participant at risk for serious adverse events in the neoadjuvant cisplatin and nab-paclitaxel group, with zero serious events affected. For other adverse events, one participant was at risk and one was affected. The record reports no deaths. No serious adverse-event terms are listed.

These are counts for the one participant registered in the neoadjuvant group, not a broad estimate of safety in a larger treated population. The listed adjuvant groups have zero participants at risk and zero affected, so their entries do not provide safety experience for people receiving those regimens. In general, small counts can describe what was recorded but cannot precisely characterize the frequency or range of adverse events in a wider population.

The registry also states that the sole patient could not complete treatment due to adverse events. It does not report detailed event descriptions or timing in the registry record for this analysis. The overall adverse-event category and the treatment-completion statement should therefore be read separately from the primary endpoint, for which no result is reported.

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

The result of statistical interest—the primary rate of N2 nodal clearance—is not reported. The registry record states that the sole patient enrolled before funding was withdrawn could not complete treatment due to adverse events. It lists no primary endpoint analysis. Thus, the study's stated objective does not translate into an observed estimate of nodal clearance.

This record does establish several details about the trial: it was registered as a phase II, non-randomized, single-group study; enrollment was 1; the status is terminated; and the planned outcome concerned N2 nodal clearance at 3 Months. It also records the limited safety counts for the neoadjuvant group. Those details describe what happened to the registered study and participant, but they do not answer whether the regimen reduced disease in the specified lymph-node locations.

The record does not establish that treatment did or did not produce clearance. It does not show how the regimen compares with surgery alone, another chemotherapy regimen, or any other strategy. Because no randomized comparator is reported and no endpoint result is available, there is no treatment contrast or effect estimate to interpret. Nor can any unreported outcome be inferred from termination, non-completion, or the absence of a published endpoint value in this record.

In general, an estimate is more informative when its uncertainty and the process producing the data can also be examined. Here there is no reported endpoint estimate or interval, and the registry does not report an analysis plan or missing-data approach. This prevents an assessment of statistical precision as well as the outcome itself. Planning illustrations, if considered, are hypothetical calculations and must not be read as observed power or as a substitute for trial results.

The most defensible conclusion is narrow: the intended phase II evaluation did not yield a reported primary endpoint analysis in the registry record. It should be understood as an incomplete evidence record, not as a finding for or against the treatment strategy.

13. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a protocol question and an analyzable result. The registry sets out a clinically focused endpoint—N2 nodal clearance at 3 Months—but the study enrolled 1 participant and reports no primary endpoint analysis. A well-defined endpoint alone cannot yield an estimate; participants must complete the relevant assessment, and those observations must be analyzed and reported.

It also illustrates why design labels must be interpreted precisely. The trial was described as phase II, but it was non-randomized and single group. Those features do not create a controlled comparison, and the listed adjuvant options are not evidence of comparative arms. Even a completed single-group endpoint assessment would answer a narrower descriptive question than a randomized comparison could answer.

The sole participant's inability to complete treatment is relevant to both feasibility and interpretation. It means there is no reported completed treatment cohort for the stated primary analysis. It also makes the distinction between safety reporting and efficacy evaluation important: adverse-event counts can be recorded even when an efficacy endpoint cannot be assessed, but the two types of information answer different questions.

Finally, the record demonstrates how incomplete reporting constrains statistical appraisal. Without an endpoint result, analysis population, missing-data approach, or stated inferential plan, readers cannot assess the estimate, precision, or robustness of a primary conclusion. The statistical lesson is not that a treatment effect was found or absent, but that the planned question remained unanswered in the registry record.

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 main limitation specific to this trial is that only 1 participant was registered before funding was withdrawn, and that patient could not complete treatment due to adverse events. The record is therefore not a completed evaluation of the planned neoadjuvant regimen. No N2 nodal-clearance result is reported, so the primary endpoint cannot be estimated or interpreted.

The status is terminated, and no analysis is listed. There is no randomized comparator, and the single-group design cannot provide a controlled between-group treatment contrast. The adjuvant regimens included in the intervention descriptions do not supply comparison data: the record reports zero participants at risk in those listed groups.

Important methodological details are not reported in the registry record, including the intended sample-size justification, analysis population, missing-data methods, endpoint assessment procedures, and detailed statistical analysis plan. No interim analysis is reported. Without these details, readers cannot determine how incomplete treatment or an unavailable endpoint assessment would have been handled in the planned analysis.

Safety interpretation is also limited by the small number of participants at risk. The registry lists one participant at risk in the neoadjuvant group and does not provide serious adverse-event terms. These records describe the registered experience but cannot establish how often events might occur in a larger group. Taken together, the study record supports a description of enrollment, termination, treatment non-completion and recorded adverse-event counts, but not a conclusion about nodal clearance or comparative treatment effects.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Cisplatin and Nab-paclitaxel for (N2) Defined NSCLC — NCT02276560.
• Registry record last updated 2017-05-11; status last verified 2017-03.
• Results first posted 2017-05-11.
• 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

NCT02276560 is a phase II, non-randomized, single-group study of neoadjuvant cisplatin and nab-paclitaxel for N2-defined Stage IIIA non-small cell lung cancer. The planned primary endpoint was the rate of N2 nodal clearance at 3 Months. Enrollment was 1, and the study status is terminated.

The registry states that the only patient registered before funding was withdrawn could not complete treatment due to adverse events. The primary endpoint result is not reported, and the record lists 0 analyses. In the neoadjuvant group, one participant was listed at risk for serious adverse events with zero affected; one was at risk for other adverse events and one was affected. No deaths were reported. The record does not provide an efficacy estimate or a randomized comparison.