Chemoradiotherapy in Stage III Non-small Cell Lung Cancer (NSCLC): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Chemoradiotherapy in Stage III Non-small Cell Lung Cancer (NSCLC) (NCT01102231), a na phase 2 study. The primary outcome is Disease-Control Rate.

2. Clinical Question

3. Trial Design

This completed phase II study evaluated chemoradiotherapy for stage III non-small cell lung cancer. Its official title specifies pemetrexed, cetuximab and cisplatin with concurrent chemoradiotherapy. The registry lists chemotherapy, ERBITUX and radiotherapy as interventions, and names the study arm “Chemoradiotherapy.” Intergroupe Francophone de Cancerologie Thoracique is listed as the sponsor.

The registry records a single-group model, allocation as not applicable and masking as none. This is not a randomized comparison: participants were not assigned between a trial treatment and a concurrent control arm in the registry description. Consequently, the posted design does not support a direct randomized estimate of the treatment difference versus another regimen or usual care.

Enrollment is listed as 106. The study start is recorded as 2010-03, primary completion as 2014-01, and status as completed. Those record dates describe the trial’s registry milestones; they do not give the dates of individual participants’ treatment or follow-up.

The primary endpoint is disease-control rate in the eligible population, assessed 16 weeks after inclusion. The listed secondary outcomes are 18-month overall survival rate and progression-free survival. The registry lists three outcome measures and one analysis, but does not report the analysis methods or numerical results for these outcomes. Details such as treatment schedule, follow-up procedures, enrollment criteria and the planned statistical design are not reported in the registry record.

The study’s stated aim was to examine efficacy and toxicity for the combination. Its single-group structure means that interpretation must focus on the outcomes recorded in that group, rather than on a controlled comparison. The absence of a comparator also limits how confidently differences in outcomes could be attributed to the regimen rather than to other influences.

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: Eligible population

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 Disease-Control Rate, measured over 16 weeks after inclusion.

The primary endpoint is disease-control rate, assessed 16 weeks after inclusion in the eligible population. It includes participants whose response is classified as complete response, partial response or stable disease under RECIST 1.1 criteria. The registry describes disease control as a percentage, while the measurement type is recorded as a count of participants. The numerical endpoint result is not reported in the registry record.

The response categories distinguish disappearance of target lesions, meaningful shrinkage, and neither sufficient shrinkage nor sufficient growth to meet the stated response or progression definitions. Complete response is defined as disappearance of all target lesions. Partial response requires at least a 30% decrease in the sum of target-lesion diameters. Stable disease is neither enough shrinkage for partial response nor enough increase for progressive disease; progressive disease is defined using at least a 20% increase in that sum.

As a general statistical point, a disease-control endpoint combines response with stabilization. It can therefore describe whether participants meet any of the included disease-status categories at the specified assessment time. It is not the same as a measure of how long control lasts. The listed progression-free survival outcome addresses a different time-to-event question, but its numerical result is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

The registry describes a single-group study with no masking and allocation recorded as not applicable. In a single-group design, results can describe what happened among participants in the study group. Without a concurrent randomized comparator, the design does not directly estimate how outcomes would differ under another treatment. General statistical interpretation therefore separates a group’s observed outcome from a causal treatment contrast. The registry does not report a control group or a comparative estimate.

The phase II designation describes the trial phase, but it does not supply the detailed analysis plan or the criterion used to judge the primary endpoint. The registry record does not report a sample-size justification, a target disease-control rate, an interim analysis plan or a prespecified decision rule. These details cannot be inferred from the phase label or from the enrollment figure.

How should a binary endpoint be understood?

The primary outcome groups participants into disease-control categories: complete response, partial response or stable disease. A binary outcome reduces each participant’s assessment to whether the defined event or condition is present. The count of participants with disease control can be used to describe the group, and a percentage expresses that count relative to a defined denominator. The registry labels the endpoint’s measurement as a participant count while its description refers to a percentage. It does not report the observed count, percentage or denominator used in a result.

As a general statistical concept, uncertainty around a proportion depends on both the observed proportion and the amount of information in the data. A confidence interval gives a range of values compatible with the data under a specified statistical method; it is not a guarantee that the true value lies in that range. No confidence interval or method for calculating one is reported for the primary outcome in the registry record. Consequently, its precision cannot be assessed from the posted endpoint information.

What can the analysis count tell readers?

The registry lists one analysis. That count does not identify which statistical test, model, estimator or adjustment was used. It also does not establish whether the analysis was prespecified, whether it was the only analysis performed, or how any deviations from an analysis plan were handled. The particular statistical methods are not reported in the registry record.

The endpoint is assessed at a stated time point, 16 weeks after inclusion. For a fixed-time binary outcome, the result depends on how participants with unavailable assessments are treated and on the denominator chosen. The registry does not report the missing-data rules, the number of participants assessed at the time point, or any sensitivity analyses. Thus, even a clearly defined endpoint cannot be fully evaluated without the analysis population and missing-data handling.

What do the planning assumptions mean?

The registry’s sample-size assumptions describe a planning illustration based on an observed effect, not the trial’s own design. They specify a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those assumptions belong to the planning illustration; the registry does not report that the phase II trial used a two-arm design or those assumptions to plan its enrollment. No numerical illustration results or observed primary effect are reported in the registry record.

As a general concept, power is a design-stage probability tied to a specified effect, variability, analysis method and sample size. It is not a measure of whether a completed study’s result is true. A planning calculation based on a chosen fraction of an observed effect is a scenario for future-study sizing, not observed power and not evidence that the trial detected an effect. If the observed effect itself is unavailable, readers cannot assess the scale on which that planning scenario was based.

The equal-allocation and two-arm assumptions also do not match the registry’s listed single-group design. That difference matters: the illustration addresses a hypothetical comparison, while the registered study is described as having one treatment group. The illustration should not be interpreted as a description of the study’s actual allocation or analysis.

Why do methods affect interpretation?

General statistical practice distinguishes an estimate from its uncertainty and from the assumptions used to obtain it. For a binary endpoint, the choice of denominator, handling of missing assessments and response classification can affect the result. For survival outcomes, methods must account for follow-up and event timing; the registry names overall survival and progression-free survival but does not state their estimators, event definitions or censoring rules. These details are not reported in the registry record.

Over-dispersion is a general issue when observed variability exceeds what a particular model assumes, and it can make standard errors too small if ignored. It is not possible to assess whether it is relevant to these outcomes because the analysis model and outcome data are not reported. More broadly, no method should be attributed to this study merely because it is common for a particular endpoint. The posted record does not provide enough analysis detail to reproduce the primary or secondary results.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Chemoradiotherapy + Cetuximab")
n      <- c(99)
events <- c(89)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Chemoradiotherapy + Cetuximab  99  89  89.9  82.2  95.0  82.4  94.4  82.4  94.4

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary disease-control endpoint and two secondary outcomes: 18-month overall survival rate and progression-free survival. It also lists three outcome measures and one analysis. The results for the primary and secondary outcomes are not reported in the registry record, and the record does not describe a multiplicity strategy. It is therefore not possible to determine whether the analysis controlled the chance of false-positive findings across outcomes or how the outcomes were prioritized in interpretation.

As a general statistical concept, testing several outcomes can increase the chance that at least one result appears notable by chance. Prespecified outcome hierarchies, adjustment procedures or a clearly exploratory interpretation can address different aspects of this issue. None of those approaches is described in the registry record, so none should be assumed to have been used.

The primary population is identified as the eligible population. The record does not report a more detailed analysis-population definition, such as which participants were included in the endpoint analysis or how exclusions were handled. It also does not report how missing assessments, withdrawals, protocol deviations or unavailable survival follow-up were treated. These details matter because the assessed population and assumptions about missing information can influence estimates and their uncertainty.

The registry record does not report the number of participants with an evaluable primary assessment, a missing-data sensitivity analysis, or a plan for handling incomplete outcome data. Nor does it describe whether outcome assessments were made by blinded reviewers; masking is listed as none. These omissions limit evaluation of possible assessment and analysis biases. They do not establish that any particular bias occurred, but they leave important analytic decisions undocumented in the posted record.

12. 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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: general physical health deterioration (6.9%), febrile neutropenia (4.9%), pulmonary embolism (3.9%), oesophagitis (3.9%), vomiting (3.9%).

For the group titled “Chemoradiotherapy + Cetuximab,” the registry lists 102 participants at risk, 26 serious adverse events, 100 other adverse events and 51 deaths. These are posted safety counts; they are not estimates of the primary disease-control outcome. The registry lists general physical health deterioration at 6.9%, febrile neutropenia at 4.9%, pulmonary embolism at 3.9%, oesophagitis at 3.9%, vomiting at 3.9% and lung disorder at 2.9% among the leading serious-event terms.

The percentages describe the listed terms, but the denominator and counting method for those term-specific percentages are not reported in the registry record. The record also does not state in this information whether events were considered related to treatment, how severity was graded, or the duration of safety observation. These figures should therefore be read as registry-reported safety summaries, not as proof of causation or as a complete account of each participant’s experience.

The count of deaths is listed separately from the adverse-event term percentages. The registry information does not identify their causes or attribute them to treatment. The 102 participants at risk in the safety group should also not be silently substituted for the enrollment figure of 106; the registry does not explain the relationship between those counts.

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

The record describes a phase II, single-group study of chemoradiotherapy with pemetrexed, cetuximab and cisplatin for stage III non-small cell lung cancer. It defines disease control at 16 weeks after inclusion using complete response, partial response and stable disease. It also names 18-month overall survival rate and progression-free survival as secondary outcomes. These design and endpoint details explain what the study set out to assess.

They do not, by themselves, show what the participants’ outcomes were. The numerical primary endpoint result and numerical secondary outcome results are not reported in the registry record. Without those results, readers cannot determine the observed disease-control level, survival estimates or precision. Nor can the record support a statement that a prespecified efficacy threshold was met, because no such threshold or result is reported.

The single-group design also does not provide a randomized comparison with another treatment. Even if group outcomes were available, they would describe the group and would not alone establish what would have happened to comparable participants under a different treatment. No comparative treatment effect is reported in the registry record.

The safety counts and listed serious-event terms offer a separate description of reported safety information. They do not establish whether events were caused by the regimen, and the record does not report the event attribution, observation details or term-specific denominators in the information listed. The counts should not be interpreted as evidence for or against efficacy.

Accordingly, the registry record supports a careful account of the study’s design, intended endpoints, enrollment and posted safety summaries. It does not support a conclusion about the magnitude or precision of disease control, survival, comparative effectiveness or treatment-attributable harm. Missing results are an information limitation, not evidence that outcomes were favorable, unfavorable or absent.

14. Why This Trial Matters Statistically

This trial illustrates why statistical interpretation begins with design rather than with an outcome label. A phase II study can be informative about outcomes in its study group, but this registry record identifies a single-group model and no randomized comparator. That distinction determines the kind of inference the design can support: description of a group is not the same as a controlled estimate of a treatment difference.

It also shows why endpoint definitions and results need to be read together. Disease control is defined through RECIST 1.1 response categories at a specified assessment time, but the numerical result is not reported. A precise definition clarifies what would count as the endpoint; it cannot replace the observed count, denominator and estimate of uncertainty needed to understand the result.

The record names survival outcomes and supplies safety counts, yet omits numerical survival results and key details about the analysis. This makes it important to distinguish an outcome’s inclusion in a registry from evidence about its observed value. Likewise, safety-event counts need context about attribution, collection and observation before they can be interpreted as treatment-related risks.

Finally, the planning assumptions should not be confused with the actual trial design. The registry describes a single treatment group, whereas the planning illustration refers to equal allocation between two arms. Keeping those contexts separate prevents hypothetical sample-size scenarios from being mistaken for evidence about the completed study. Clear distinctions among design, endpoint, analysis and reported result are central to reading clinical trial statistics responsibly.

15. 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 principal limitation for interpreting outcomes is that the registry record does not report the numerical primary endpoint result or numerical results for the listed secondary outcomes. It also does not provide confidence intervals, test results or other estimates of precision. As a result, the record cannot establish the magnitude of disease control or survival outcomes.

The single-group design has no randomized comparator listed. This limits causal interpretation and does not permit a direct treatment contrast. The registry also does not report a detailed statistical analysis plan, sample-size justification, interim-analysis plan, target for the primary endpoint or decision rule. The planning illustration’s assumptions should not be treated as the trial’s protocol.

Important endpoint-analysis details are absent, including the number assessed at the primary time point, the endpoint denominator, missing-data handling, analysis-population rules and sensitivity analyses. For the survival outcomes, event definitions, estimation methods and censoring rules are not reported. The registry lists no multiplicity approach despite naming multiple outcomes.

The safety information lists a risk group, event counts and selected serious-event percentages, but the record does not report the term-specific denominators, event attribution, collection period or grading details in the listed information. It also does not explain the relationship between the enrollment count and the safety group’s at-risk count. These gaps limit interpretation of both efficacy and safety; they do not show that any specific result or bias occurred.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Chemoradiotherapy in Stage III Non-small Cell Lung Cancer (NSCLC) — NCT01102231.
• Registry record last updated 2021-02-16; status last verified 2021-01.
• Results first posted 2021-02-16.
• 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.

19. Record Summary

NCT01102231 is a completed phase II study of chemoradiotherapy in stage III non-small cell lung cancer, sponsored by Intergroupe Francophone de Cancerologie Thoracique. The registry lists enrollment of 106, a single-group design, no masking and one chemoradiotherapy arm. Its official title names pemetrexed, cetuximab and cisplatin with concurrent chemoradiotherapy.

The primary endpoint is disease-control rate at 16 weeks after inclusion, defined using RECIST 1.1 complete response, partial response or stable disease. The listed secondary outcomes are 18-month overall survival rate and progression-free survival. Numerical results for these outcomes are not reported in the registry record.

Safety information lists 102 participants at risk, 26 serious adverse events, 100 other adverse events and 51 deaths for the chemoradiotherapy plus cetuximab group. The record does not report a randomized comparator or enough analysis detail to support comparative conclusions.