Ph II Adjuvant Carboplatin/Docetaxel in Curatively Resected Stage I-IIIA NSCLC: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ph II Adjuvant Carboplatin/Docetaxel in Curatively Resected Stage I-IIIA NSCLC (NCT00280735), a na phase 2 study. The primary outcome is Number of Participants Who Completed Four Cycles of the Carboplatin/Docetaxel Regimen.

2. Clinical Question

3. Trial Design

This was a phase II study listed as completed, with enrollment of 75. The sponsor was UNC Lineberger Comprehensive Cancer Center. The registry record gives a start date of 2004-05 and a primary completion date of 2011-07. These dates describe the study record; they do not by themselves establish when every participant was treated or followed.

The design is recorded as single group, with allocation listed as not applicable and masking listed as none. There was no randomized treatment contrast within the study. All participants were considered in relation to the adjuvant carboplatin/docetaxel regimen, but the number who actually received treatment or entered each analysis population is not reported in the registry record.

The regimen description specifies carboplatin AUC = 6 intravenously on day 1 every 3 weeks for 4 cycles, together with docetaxel 75 mg/m² intravenously on day 1 every 3 weeks for 4 cycles. The primary outcome timeframe was 12 weeks from initiating adjuvant therapy. The registry record does not report dose modifications, treatment delays, or the rules used to determine whether a cycle counted as completed.

The study focused on feasibility after curative resection, rather than a randomized comparison of treatment strategies. Secondary outcomes named in the record include patterns of recurrence, toxicity, progression-free survival, and overall survival. The record lists five outcome measures in total, but does not provide numeric results for the named secondary outcomes. It also contains no analysis entries. Details of the statistical analysis plan, including any planned subgroup or adjusted analyses, are not reported in 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.

Before assignment: Of the 75 participants screened for eligibility, 72 went on to treatment and 3 participants withdrew consent prior to treatment.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Patients assigned to treatment

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 Who Completed Four Cycles of the Carboplatin/Docetaxel Regimen, measured over 12 weeks from initiating adjuvant therapy.

The primary endpoint is the number of participants who completed four cycles of the carboplatin/docetaxel regimen. The registry sets the timeframe at 12 weeks from initiating adjuvant therapy and describes feasibility in terms of the percentage completing the regimen. The endpoint is therefore a treatment-completion measure: it asks whether participants reached the specified course of therapy within the specified period.

Completion can be useful in a feasibility study because it describes whether a regimen can be delivered as planned in the study population. It is distinct from an endpoint that measures tumor response, recurrence, or survival. As a general statistical point, a count of completers needs an interpretable denominator and a clear definition of completion. The registry record identifies the population as patients assigned to treatment, but does not report the observed completion count, percentage, or participant-level reasons for not completing treatment.

The endpoint name and definition do not establish that a particular completion threshold was met. The registry record does not report a numeric primary outcome or a threshold against which the observed result was judged. Consequently, the endpoint can be described, but the feasibility finding itself cannot be assessed from the reported primary outcome information.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What kind of outcome is the primary endpoint?

The primary measure is a count of participants completing the specified treatment course. The registry classifies the outcome as binary and describes the underlying meaning as participants with an event (counts). In general, a binary outcome places each participant into one of two categories for the endpoint, such as completion or non-completion. Reporting a count is informative only when the denominator and the rules for classifying participants are also clear. The registry record gives the population as patients assigned to treatment and sets a 12-week timeframe, but the observed count, denominator, and classification details are not reported.

The endpoint description says feasibility was based on the percentage completing four cycles. A percentage would communicate the share of a defined group meeting the completion criterion, but no observed percentage is reported. No completion threshold or decision rule is stated in the registry record. In general, a feasibility endpoint can be used to evaluate whether treatment delivery is practicable; it does not, by itself, quantify comparative benefit or establish an effect on recurrence or survival.

What does a single-group design allow?

This study is recorded as single group, with allocation not applicable and masking none. In general, a single-group study can describe outcomes among participants receiving a regimen. It does not estimate a within-trial difference between that regimen and a concurrent control group, because no separate comparator group is described. Without a comparison group, differences from an external population may reflect differences in eligibility, clinical setting, follow-up, or other factors; no such external comparison is reported in the registry record.

For this study, the most direct interpretation would be descriptive: how many participants completed the regimen, and what adverse events were recorded. The primary completion result is not reported, so even that descriptive feasibility assessment is unavailable from the posted primary outcome information. Recurrence and survival are named secondary outcomes, but their results and any analysis methods are not reported.

What is reported about planning assumptions?

The registry record describes a planning illustration based on the observed effect, not the trial’s own design. Its stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. The record says that time-to-event planning tables give the total number of events using the Schoenfeld method, while other planning tables give participants per arm.

Those assumptions do not describe the actual single-group trial design: the record does not identify two randomized arms in this study. No numerical planning results are included in the registry information available for this analysis, and no observed effect estimate is reported to anchor the illustration. Thus, the assumptions cannot be used to infer the study’s planned enrollment, its achieved precision, or its ability to detect an effect. Any sample-size illustrations are planning calculations, not observed power. Observed power is not a substitute for reporting an endpoint estimate and its uncertainty.

How should uncertainty and over-dispersion be understood?

As a general statistical explanation, a confidence interval communicates uncertainty around an estimated outcome under a specified model. For a completion proportion, interval width depends on the number of observations and the observed outcome pattern, among other assumptions. The registry does not report a primary estimate or interval, so the precision of the feasibility result cannot be assessed. A p-value, if reported, would address compatibility with a null hypothesis under a defined analysis; it would not by itself describe the size or practical meaning of the result. No p-value is reported for the primary endpoint.

Over-dispersion is a general concern when a statistical model assumes less variation than is present in the data. For example, participants may have outcomes that are more alike within clinical groups than a simple model assumes, making uncertainty estimates too narrow if that clustering is ignored. The registry record does not report a model for the primary endpoint or information that would establish whether over-dispersion was assessed. It would therefore be inappropriate to claim that a particular model or correction was used.

What analysis details are absent?

No analysis entries are reported. The registry record does not specify a statistical test or model for the primary outcome, an approach to confidence intervals, an interim analysis plan, or adjustments for covariates. It also does not report how treatment completion was handled for participants who withdrew, had treatment delayed, or lacked an outcome assessment. These details are relevant because analysis choices determine which participants contribute to an estimate and how uncertainty is quantified. Their absence prevents a fuller methodological appraisal, rather than showing that any particular method was or was not used during the study.

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("Single Arm Trial")
n      <- c(72)
events <- c(57)
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  Single Arm Trial  72  57  79.2  68.0  87.8  68.4  86.9  68.4  86.9

11. Multiplicity, Missing Data and Analysis Populations

The registry record identifies a primary completion endpoint and names secondary outcomes concerning recurrence patterns, toxicity, progression-free survival, and overall survival. It lists five outcome measures in total, but does not provide numeric results for the named secondary outcomes or analysis entries. The record does not state which secondary outcomes were formally tested, whether any were considered exploratory, or how results across multiple outcomes were interpreted.

Multiplicity is a general statistical issue that arises when several hypotheses or outcomes are examined. If many tests are conducted, the chance of at least one apparently unusual result can increase unless the analysis plan accounts for that testing. The registry record does not report whether a multiplicity adjustment or hierarchy among outcomes was planned. It is therefore not possible to determine how any secondary findings were intended to be interpreted or whether their uncertainty reflects multiple testing.

Missing-data handling is also not reported. In particular, the registry record does not say how participants with incomplete treatment, missing completion status, or incomplete follow-up were included in the primary or secondary analyses. Enrollment is 75, while the serious adverse event group lists 72 participants at risk; reasons for that difference and its relation to the endpoint populations are not reported. The figures alone do not establish that the same participants were used for each analysis.

Analysis populations are not described beyond the primary endpoint’s reference to patients assigned to treatment. The record does not identify an intention-to-treat, per-protocol, or other analysis population. These omissions limit assessment of selection and missingness; they do not establish that the study used an inappropriate approach.

12. 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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: febrile neutropenia (11.1%), neutrophils/granulocytes (anc/agc) (8.3%), diarrhea (4.2%), cardiac ischemia/infarction (2.8%), dehydration (2.8%).

The registry’s serious adverse event group lists 72 participants at risk and 19 affected. It separately lists 72 participants at risk and 63 affected for other adverse events. These are participant counts as recorded, not a comparison between treatment groups. The registry also lists serious-event terms with percentages: febrile neutropenia, 11.1%; neutrophils/granulocytes (ANC/AGC), 8.3%; diarrhea, 4.2%; cardiac ischemia/infarction, 2.8%; dehydration, 2.8%; and nausea, 2.8%.

Those term-specific percentages should be read as registry-reported values. The record does not provide further information about event definitions, timing, duration, severity grades, or whether one participant could contribute to more than one term. Therefore, the categories should not be assumed to be mutually exclusive, and the percentages should not be added together. The record lists deaths as null, so a death count is not reported in the registry record. The safety figures describe events in this study and cannot establish comparative risk without a separate comparison group.

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

The registry record defines a clinically relevant feasibility question: whether participants completed four cycles of adjuvant carboplatin/docetaxel within 12 weeks from initiating therapy. However, it does not report the primary endpoint result. Without the number completing treatment, the relevant denominator, and an uncertainty estimate, readers cannot tell from this record what proportion completed the planned course or how precisely that proportion was estimated.

The record does report serious and other adverse event counts, along with several serious-event terms and percentages. These figures describe events recorded among participants at risk in the single study group. They do not compare this regimen with another treatment, and the record does not provide enough information to determine overlap among event categories or the circumstances of each event.

The study’s single-group design is important to the scope of any conclusion. A descriptive result among treated participants is not the same as a treatment effect relative to an alternative. The record names recurrence, progression-free survival, and overall survival among its secondary outcomes, but does not report their numeric results. It therefore does not support a conclusion about comparative effects on those outcomes.

In general, a phase II study can contribute information about treatment delivery and generate evidence for further evaluation. Its designation as phase II, however, does not supply missing endpoint results or establish a particular level of efficacy. Here the registry record documents the question, treatment schedule, and some safety information, but the primary feasibility result is not reported. The appropriate interpretation is limited to those recorded details and the substantial uncertainty created by absent outcome and analysis details.

14. Why This Trial Matters Statistically

This study illustrates why a trial’s endpoint definition and its observed result must be considered separately. The registry provides a clear treatment-completion question, a four-cycle regimen, and a 12-week timeframe. Those details indicate what the primary endpoint was intended to capture. They are not a substitute for the number of participants meeting the endpoint, the denominator used, or an estimate of uncertainty.

The design also matters statistically. A single-group study can describe treatment delivery and adverse events among its participants, but it does not provide a randomized internal comparison. The safety counts should therefore be read as descriptive, not as evidence that event rates were higher or lower than they would have been under another treatment. The named recurrence and survival outcomes would require reported results and defined analyses to support interpretation; those results are not reported in the registry record.

Finally, the record distinguishes the trial from the planning illustration described alongside its assumptions. The stated illustration uses equal allocation between two arms, whereas the actual study is recorded as single group. This is a reminder that a planning scenario should not be confused with the design or results of the trial itself. The absence of analysis entries, endpoint estimates, and missing-data methods limits reproducibility and precision assessment. These are statistical limitations of the available record, not evidence that the underlying study did not collect the information.

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 central limitation is that the primary completion result is not reported in the registry record. The record gives an endpoint definition and timeframe, but not the observed count, percentage, denominator, or uncertainty. As a result, the main feasibility question cannot be answered from the posted outcome information.

The single-group design has no concurrent treatment comparator. It can describe observed treatment completion and adverse events, but cannot establish a comparative effect within the trial. The record does not report numeric results for the named recurrence, toxicity, progression-free survival, or overall survival outcomes, nor does it contain analysis entries.

Several details needed to assess the primary analysis are not reported: the analysis population, handling of missing completion data, definition of a completed cycle, and treatment of withdrawals or delays. The serious adverse event data list 72 participants at risk, while enrollment is 75; the reason for that difference is not reported. The record also does not describe event timing or whether safety categories overlap.

The registry wording is not fully consistent about stage: the official title specifies stage I-IIIA, the brief summary describes stage I, stage II, or stage III, and the primary endpoint description refers to stage IIIIA. The record does not explain this wording difference. It also does not report a statistical analysis plan, multiplicity approach, sample-size justification for the actual study, or interim analysis details. These limitations constrain interpretation of the registry record and should not be taken as proof that such information was absent from other study documents.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Ph II Adjuvant Carboplatin/Docetaxel in Curatively Resected Stage I-IIIA NSCLC — NCT00280735.
• Registry record last updated 2017-07-11; status last verified 2017-06.
• Results first posted 2017-06-12.
• 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

NCT00280735 is a completed phase II, single-group study sponsored by UNC Lineberger Comprehensive Cancer Center, with enrollment of 75. The registry record gives a start date of 2004-05 and a primary completion date of 2011-07. It describes adjuvant carboplatin and docetaxel after curative resection of non-small cell lung cancer.

The primary endpoint is the number of participants completing four cycles within 12 weeks from initiating adjuvant therapy. The observed completion result is not reported in the registry record. Serious adverse event data list 19 affected participants among 72 at risk, and other adverse event data list 63 among 72 at risk. Recurrence patterns, toxicity, progression-free survival, and overall survival are named as secondary outcomes, but their numeric results are not reported. No analysis entries are listed.