TARCEVA (Erlotinib) in Combination With Chemoradiation in Patients With Stage IIIA/B Non-Small Cell Lung Cancer (NSCLC): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for TARCEVA (Erlotinib) in Combination With Chemoradiation in Patients With Stage IIIA/B Non-Small Cell Lung Cancer (NSCLC) (NCT00563784), a na phase 2 study. The primary outcome is Time To First Disease Progression.

2. Clinical Question

The stated research aims also include examining safety and testing earlier biopsy tissue for levels of epidermal growth factor receptor (EGFR), to learn about possible links between EGFR forms and response to erlotinib. Those aims describe questions for the study, not findings. The registry record does not report results for the EGFR analysis or a numerical result for the primary endpoint. Because the trial has a single group, its outcome data cannot by themselves distinguish the contribution of the combined regimen from other influences on participants’ outcomes. The study’s stated question is about the regimen as a whole, rather than a randomized comparison of erlotinib with another treatment.

3. Trial Design

This is a Phase II, single-group study sponsored by M.D. Anderson Cancer Center. The registry record lists the study as completed, with enrollment of 68. Allocation is recorded as NA, and masking as NONE. The listed intervention combines erlotinib with paclitaxel, carboplatin, and radiation therapy. No separate control arm is described, so outcomes are not presented as a randomized comparison between treatment groups.

The regimen description specifies oral erlotinib 150 mg daily, paclitaxel 45 mg/m^2 by vein weekly, carboplatin 2 AUC by vein weekly, and radiation therapy of 63 GY/35 fractions for 7 weeks. These are the registry’s listed treatment details; information about adherence, treatment modifications, or delivered doses is not reported in the registry record.

The primary endpoint is time to first disease progression, with a median and 95% confidence interval as the stated summary. The endpoint timeframe starts at registration and lists first documented progression, death from any cause, or loss to follow-up as possible stopping points, with assessment up to 5 years. The endpoint description says 46 out of 48 patients completed treatment under the protocol and were evaluable for data analysis. It also says 2 patients did not complete treatment and went off-study: 1 for diarrhea described as unrelated to treatment, and the other for chemotherapy-induced chest pain. How the registry’s enrollment of 68 relates to this 48-patient endpoint account is not explained in the record.

Secondary outcomes are listed as overall survival and disease local control rate. The registry record does not report their numerical results. It also does not report the protocol’s sample-size justification, an analysis plan, or interim analyses. The number of analyses recorded is 0; the record does not give details about the statistical procedures used to produce endpoint estimates.

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.

Before assignment: There were 20 patients off-study after enrollment due to screen failure. Two patients were taken off protocol: 1 for diarrhea unrelated to treatment and the other for chemotherapy-induced chest pain. Only 46 patients are evaluable for this study.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: There were 46 out of 48 patients completed treatment under the protocol and evaluable for data analysis. 2 patients cannot complete the treatment and off-study. 1 for diarrhea unrelated to treatment and the other for chemotherapy-induced chest pain.

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.

Population: 48 patients registered and treated under the protocol.

6. Primary Endpoint

The registry lists the primary outcome as Time To First Disease Progression, measured over From date of registration until the date of first documented progression or death from any cause, or lost to follow up, whichever came first, assessed up to 5 years..

Time to first disease progression measures the elapsed time from a specified starting point to an event of interest. In this record, the stated starting point is the date of registration, and the endpoint is summarized by its median and a 95% confidence interval. The registry’s description lists first documented progression, death from any cause, or loss to follow-up as possible endpoint stopping points, and says assessment was planned up to 5 years.

The progression definition refers to RECIST v1.0 and a 20% increase in the sum of the longest diameter of target lesions. In general, a time-to-event endpoint can account for differing follow-up durations: participants may be observed for different lengths of time, and some may not have a documented event by the end of their follow-up. Statistical methods for these data must specify how such incomplete follow-up is handled. The registry record does not explain that handling in detail.

A median time-to-event result gives a central summary of observed event timing, while its confidence interval conveys uncertainty in the estimated median. In general, a narrower interval indicates greater precision and a wider interval indicates less precision. The registry record specifies the intended summary but does not report the median or its 95% confidence interval. Consequently, neither the timing of progression in this study nor the precision of its estimate can be described from the posted endpoint information.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Time-to-event summaries. Time to first disease progression is a time-to-event outcome: the measurement combines whether an event was documented with how long participants were followed before that event or another endpoint stopping point. The registry specifies the median as the summary. In general, the median is the point in the estimated event-time distribution at which half of the population is estimated to have experienced the event. It is not the same as an average, and it does not describe the experience of every participant.

The registry also specifies a 95% confidence interval for the median. In general, a confidence interval communicates uncertainty around an estimate arising from the available data and analysis. It is not a guarantee about an individual participant’s outcome. Because the median and interval are not reported in the registry record, their magnitude and precision cannot be interpreted for this study.

Progression definition and assessment. The endpoint description defines progression using RECIST v1.0 as a 20% increase in the sum of the longest diameter of target lesions. It describes follow-up after treatment, including regular assessments, and states that assessment could continue up to 5 years. Consistent assessment criteria are important in general because they make the event definition clearer and support more comparable evaluations. The registry does not provide actual assessment records, timing of observed events, or information about whether progression was confirmed through any additional process.

Incomplete follow-up. Time-to-event analyses commonly encounter participants who leave observation before an event is documented. In general, statistical methods may account for such follow-up by treating the participant’s observed time as incomplete rather than as a confirmed event time. The endpoint timeframe in this record lists loss to follow-up among the stopping points, but the registry does not explain whether or how censoring was used, how loss to follow-up was handled, or whether other missing-data methods were applied. Those details affect interpretation because estimates depend on the relationship between follow-up and event occurrence.

Single-group design. The design is recorded as single group, with allocation NA and no masking. In general, a single-group analysis can describe outcomes within the enrolled group, but it lacks a concurrent control group for separating treatment-associated outcomes from other influences. As a result, the study design does not provide a randomized comparison between the combination regimen and an alternative. The registry does not report an adjusted comparative estimate or a sponsor-reported hazard ratio for the primary endpoint.

Planning calculations. The record describes a planning illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says time-to-event illustrations use a total number of events, while other illustrations use participants per arm. These are planning assumptions, not a description of this trial’s single-group design or an observed power calculation. The numerical planning outputs and the effect used for those illustrations are not reported in the registry record. A planning illustration cannot establish the amount of power actually achieved after a study is completed.

Safety summaries. The safety entry lists participants at risk and affected, along with selected serious adverse-event terms and percentages. Such counts are descriptive. In general, counts can indicate how many participants in the recorded safety group were affected, but without a comparator they do not estimate a relative increase or decrease in risk. The record does not explain the detailed event-coding, attribution, or observation rules used for those summaries. Its primary endpoint and safety summaries address different questions: time to progression concerns disease events over follow-up, while adverse-event counts concern reported harms.

Multiplicity and analysis detail. Multiple outcomes or repeated statistical tests can raise a multiplicity issue: in general, the chance of at least one false-positive finding can increase when many tests are considered without an appropriate plan. The registry lists a primary endpoint and secondary outcomes, but does not report a multiplicity strategy, hypothesis-testing procedure, or interim-analysis plan. The number of recorded analyses is 0. That entry does not reveal whether analyses were conducted outside the registry record; it means the posted record does not provide analysis details sufficient to evaluate those methods.

10. Multiplicity, Missing Data and Analysis Populations

The record identifies time to first disease progression as the primary endpoint and lists overall survival and disease local control rate as secondary outcomes. It also describes safety and EGFR tissue testing as study aims. The registry record does not report a plan for controlling multiplicity across outcomes or analyses. In general, multiplicity matters when several outcomes or statistical tests are examined, because interpreting results without a prespecified approach can make isolated findings harder to assess. No numerical primary or secondary efficacy results are reported, so the record does not permit a review of how such results were handled.

Missing-data methods are not reported in the registry record. The primary endpoint account says 46 out of 48 patients completed treatment under the protocol and were evaluable, while 2 did not complete treatment and went off-study. The reasons are given as diarrhea unrelated to treatment for 1 patient and chemotherapy-induced chest pain for the other. The registry does not explain whether these participants contributed to any endpoint analysis, how follow-up after stopping treatment was handled, or how loss to follow-up was treated in the time-to-event calculation.

The record lists enrollment of 68, but the endpoint description refers to 48 patients. It does not explain the relationship between these figures or define the analysis population for the primary endpoint. The statistical analysis plan and any interim-analysis plan are not reported in the registry record. The number of analyses recorded is 0. Without those details, it is not possible to determine from the record which participants were included in each analysis or whether the methods addressed missing outcomes and multiple comparisons.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: skin toxicity, any (13.0%), pneumonititis (6.5%), acneform rash (4.3%), esophagitis (2.2%).

The registry safety entry lists 46 participants at risk for the serious-event summary and 10 affected. It also lists 46 at risk for the other adverse-event summary and 36 affected. The entry reports 0 deaths. These are descriptive counts in the posted safety record; they are not a comparison against a separate control group, because the trial is recorded as single group.

The selected serious adverse-event terms are Skin toxicity, any (13.0%), Pneumonititis (6.5%), Acneform rash (4.3%), and Esophagitis (2.2%). These percentages are quoted as recorded. The entry does not provide further detail about event timing, duration, severity grading, treatment attribution, or whether participants could contribute to more than one listed term. The registry record also does not explain how these terms were selected for display.

In general, an adverse-event count is not by itself evidence that an event was caused by a treatment. A single-group safety summary also cannot show how the counts compare with another treatment group. The figures describe the safety information recorded for this study and should not be read as a comparative estimate.

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

The registry record describes a Phase II, single-group study of erlotinib combined with paclitaxel, carboplatin, and radiation therapy in patients with Stage IIIA/B non-small cell lung cancer. It identifies time to first disease progression as the primary endpoint and states that the result was to be summarized by a median and 95% confidence interval. Those numerical results are not reported in the registry record.

Accordingly, the posted information does not establish the observed median time to progression or how precisely it was estimated. It also does not report numerical results for the listed secondary outcomes of overall survival and disease local control rate, or for the stated EGFR tissue-testing aim. The registry’s listed safety counts and selected serious-event terms provide descriptive information, but do not compare the regimen with a control group.

The design is single group, with allocation recorded as NA and masking as NONE. In general, the absence of a separate comparator means that outcomes in the study group cannot be interpreted as a randomized difference from another treatment. The registry record therefore cannot, on its own, attribute observed outcomes to any one component of the combination or quantify a comparative treatment effect.

The record lists enrollment of 68, while the primary endpoint description accounts for 48 patients, of whom 46 completed treatment under the protocol and were evaluable and 2 went off-study. The relationship between the enrollment figure and the endpoint population is not explained. This limits understanding of which participants are represented in the endpoint account.

These points distinguish the study’s aims from its reported findings. The aims include efficacy, safety, and possible relationships between EGFR forms and response; the registry entries do not provide numerical primary endpoint findings or EGFR results. The posted data therefore describe the design, endpoint definition, participant account, and selected safety information, but do not provide enough outcome information to characterize the primary time-to-progression result.

13. Why This Trial Matters Statistically

This trial illustrates why the design and reporting of an endpoint matter alongside the clinical question. Time to first disease progression is a time-to-event measure, and the registry specifies a median and 95% confidence interval. Those summaries would communicate both the estimated timing of progression and uncertainty around that estimate. Because neither is reported in the registry record, readers cannot assess the magnitude or precision of the primary endpoint from the posted results.

The single-group design is also important statistically. It allows outcomes to be described for participants in the study group, but it does not provide a concurrent randomized comparator. In general, this means that a within-group outcome is not equivalent to a comparative treatment effect. This is especially relevant for a regimen combining several treatment components, because the design does not isolate the contribution of any one component.

The participant accounting makes analysis populations another key issue. The registry lists enrollment of 68, while its primary endpoint description refers to 48 patients, including 46 who completed treatment under the protocol and were evaluable. The relationship between these figures is not explained. Clear reporting of who was included in each outcome analysis is important for understanding how the estimates represent the enrolled group.

The record also shows why outcome definitions and follow-up handling should be explicit. It defines progression using RECIST v1.0 and describes follow-up up to 5 years, but does not report the endpoint estimate or explain in detail how loss to follow-up was handled. Safety counts are listed, but there is no control group for comparison. Taken together, the design and reporting details identify what the study set out to examine while underscoring the limits of interpretation when numerical outcomes and analysis methods are absent.

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 for statistical interpretation is that the registry record does not report the primary endpoint median or its 95% confidence interval. The endpoint is defined, but there is no numerical time-to-progression result to assess. Numerical results for the listed secondary outcomes are also not reported in the registry record.

The study is single group, with allocation listed as NA and no masking. There is no separate comparator arm, so the study does not provide a like-for-like control comparison. This limits the ability to interpret the outcomes as a comparative treatment effect or to separate the contribution of the regimen’s components.

There is an unexplained difference between the enrollment of 68 listed in the record and the primary endpoint description of 48 patients, including 46 who completed treatment and were evaluable. The registry record does not clarify the relationship between those counts or specify the analysis population for the primary endpoint. It also does not report the statistical analysis plan, missing-data methods, multiplicity approach, or interim-analysis plan. Although loss to follow-up is included in the endpoint timeframe, the handling of such follow-up is not described in detail.

The posted safety information is limited to the listed at-risk and affected counts, deaths, and selected serious adverse-event terms. Further event-level details and the method used to select those terms are not reported in the registry record. The EGFR testing aim is described, but its results are not reported. These omissions constrain assessment of both outcome precision and the scope of the analyses.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: TARCEVA (Erlotinib) in Combination With Chemoradiation in Patients With Stage IIIA/B Non-Small Cell Lung Cancer (NSCLC) — NCT00563784.
• Registry record last updated 2019-11-20; status last verified 2019-11.
• Results first posted 2019-11-20.
• 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

The registry describes a completed Phase II, single-group study of erlotinib with paclitaxel, carboplatin, and radiation therapy for Stage IIIA/B non-small cell lung cancer. Enrollment is listed as 68. The primary endpoint is time to first disease progression, specified as a median with a 95% confidence interval, using a progression definition based on RECIST v1.0. The endpoint result is not reported in the registry record.

The primary endpoint description says 46 out of 48 patients completed treatment under the protocol and were evaluable, while 2 went off-study. The relationship between this account and the listed enrollment is not explained. Overall survival and disease local control rate are listed as secondary outcomes, without numerical results. The safety entry lists 10 affected among 46 at risk for serious events, selected serious-event terms, and 0 deaths. No comparator arm is described, and analysis methods are not reported in detail.