Sunitinib Non Small Cell Lung Cancer Patients Over 70: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib Non Small Cell Lung Cancer Patients Over 70 (NCT00864721), a na phase 2 study. The primary outcome is Disease Control Rate (DCR).

2. Clinical Question

3. Trial Design

This completed Phase II trial examined sunitinib malate in previously untreated patients over the age of 70 with non-small cell lung cancer. US Oncology Research is listed as the sponsor. The registry reports enrollment of 63, a start date of 2009-02, and primary completion in 2012-07. Its status is COMPLETED.

The design is identified as single-group. Allocation is listed as NA and masking as none. The record describes one intervention, sunitinib malate, and does not identify a randomized comparator. This means the central statistical description is a result for the study group, rather than an estimate of a difference between treatment groups. The design alone cannot separate treatment-associated outcomes from the course of disease or other influences.

The planned regimen is described as outpatient oral sunitinib malate (Sutent) at 37.5 mg/day, taken on Days 1-42 of each 42-day cycle. The primary outcome was assessed every 6 weeks until progressive disease or death due to any cause, up to 36 month. The registry does not report actual treatment duration, adherence, dose interruptions, or changes in dose.

The primary outcome is disease control rate in the evaluable population. Other listed outcomes are overall response rates, progression-free survival, 1-year overall survival rate, and time to progression. Although the record lists five outcome measures, it reports zero analyses. No outcome estimates or analysis details are present. In particular, the enrollment figure should not be substituted for the unreported evaluable population size or treated as the denominator for the primary outcome.

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: Evaluable 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.

Population: ITT population

6. Primary Endpoint

The registry lists the primary outcome as Disease Control Rate (DCR), measured over Every 6 weeks until progressive disease or death due to any cause, up to 36 month..

The primary endpoint is disease control rate, expressed as a percentage of participants in the evaluable population. The registry defines disease control as complete response, partial response, or stable disease lasting at least 6 weeks. It describes complete response as disappearance of all target lesions and partial response as at least a 30% decrease in the sum of the longest diameter of target lesions, using RECIST v1.0. Assessment is described as by MRI. The record defines stable disease as persistence of one or more non-target lesions and/or maintenance of a tumor marker level above the normal limits.

As a general statistical concept, a composite response endpoint can capture more than tumor shrinkage alone: it combines specified response categories with a duration requirement for stable disease. A rate is useful for describing the proportion of an analysis population meeting that endpoint definition over the stated assessment schedule. The accompanying confidence interval is intended to express uncertainty around the estimated rate.

The registry specifies a 95% confidence interval but does not report the disease control rate, its interval, or the number of evaluable participants. The observed result and its precision therefore are not available in the registry record. The endpoint definition also does not, by itself, supply information about overall survival, progression-free survival, or patient experience.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a single-group Phase II study and names disease control rate as its primary outcome. The outcome is a percentage of participants, and its population is described as evaluable. The record specifies a 95% confidence interval, but it does not report the estimate, interval limits, or the evaluable denominator. The statistical ideas below are general explanations of how such summaries are usually interpreted; the registry record does not identify the specific analysis procedure used in this trial.

What does a percentage endpoint represent?

For a binary outcome, each participant in the analysis population is classified according to whether the endpoint definition was met. The percentage describes the share classified as having disease control. Here, the definition combines complete response, partial response, and stable disease lasting at least 6 weeks. The endpoint is therefore not simply a measure of tumor shrinkage: stable disease meeting the stated duration criterion also contributes.

A percentage depends on both the number meeting the endpoint and the population used as its denominator. This study specifies the evaluable population, but does not report its size or the number meeting the endpoint. The enrollment figure cannot be assumed to equal the evaluable population. Without the numerator and denominator, the observed percentage cannot be reconstructed, and no recalculated estimate is available.

How should a confidence interval be read?

In general, a confidence interval communicates the statistical uncertainty around an estimate. A narrower interval indicates greater precision under the method’s assumptions; a wider interval indicates less precision. It is not a guarantee that the true value lies within the particular interval, nor does it address sources of bias such as selection into an evaluable population or the absence of a comparator. The registry specifies a 95% confidence interval for disease control rate, but no interval values are reported. Its precision therefore cannot be judged.

For a binary rate, uncertainty is influenced by the amount of information in the analysis population and by the observed rate. General methods make assumptions about how participant outcomes vary and how the analysis population was formed. The record does not name an interval construction method, discuss missing endpoint assessments, or state how evaluability was determined. These omissions prevent a method-specific assessment of the interval that was intended.

What does the single-group design imply?

A single-group analysis can describe the proportion of the study population meeting an endpoint. It does not supply a randomized contrast with an untreated or alternative-treatment group. General comparisons between a study result and an external benchmark may be affected by differences in participant characteristics, assessment practices, and follow-up. No such benchmark or comparison is reported in the registry record. Accordingly, the design should be interpreted as descriptive rather than as a controlled treatment-effect estimate.

Masking is listed as none. In general, lack of masking can matter when clinical judgment affects assessment or when knowledge of treatment could influence reporting or follow-up. The registry describes disease status assessment using MRI and RECIST v1.0, but does not report details of assessor masking, independent review, or adjudication. It is not possible to determine from this record how assessment processes addressed potential influence.

Why can over-dispersion matter?

As a general concept, a simple binomial model treats participants’ binary outcomes as having a common underlying event probability and assumes independent observations. Over-dispersion refers to more variability than such a model expects. If important differences among participants or clustering create extra variability, a model that ignores it may understate uncertainty. The registry does not report a model-based analysis, an over-dispersion assessment, or enough outcome detail to assess this issue for the trial. This is a general consideration, not a claim that over-dispersion occurred.

What do planning calculations mean?

The stated planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it describes the effect to detect as a stated fraction of the observed effect. It is explicitly a planning illustration based on an observed effect, not the trial’s own design. The actual study is single-group, and the registry record does not report the observed primary effect. Such a hypothetical two-arm planning framework does not describe the trial’s analysis and cannot establish observed power. A sample-size illustration is a planning quantity, not a finding about the probability that the completed study detected an effect.

The record does not report the trial’s own sample-size justification, statistical analysis plan, interim analyses, or inferential testing procedure. No result should be inferred from those missing details. The listed endpoint, study design, and summary specification are the available basis for statistical interpretation.

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("Sunitinib Malate")
n      <- c(60)
events <- c(38)
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  Sunitinib Malate  60  38  63.3  49.9  75.4  50.7  74.4  50.6  74.4

11. Multiplicity, Missing Data and Analysis Populations

The registry lists five outcome measures: disease control rate, overall response rates, progression-free survival, 1-year overall survival rate, and time to progression. It reports zero analyses. The primary endpoint is identified, but the record does not describe an analysis hierarchy for the secondary outcomes, an adjustment for multiple testing, or whether any outcomes were designated as exploratory. These matters are not reported in the registry record.

Multiplicity is a general statistical concern when several outcomes or hypotheses are assessed: the chance of at least one chance finding can increase as the number of tests grows. Whether adjustment is appropriate depends on the study’s objectives and analysis plan. Because no analysis plan or test results are reported, the role of multiplicity control cannot be evaluated for this trial. The listed outcome measures should not be treated as evidence that all were analyzed or that any particular significance threshold was applied.

The primary endpoint population is described as evaluable, but the record does not define evaluability or report the size of that population. It also does not report missing assessments, exclusions, withdrawals, or rules for handling incomplete endpoint data. These omissions matter because the calculated rate could depend on which participants were included and how unavailable assessments were classified.

The registry’s enrollment figure is not a substitute for the unreported evaluable denominator. There is no basis in the record to determine whether all enrolled participants contributed to the primary analysis, whether the analysis followed an intention-to-treat principle, or whether missing outcomes were imputed. The analysis population and missing-data procedures are not reported in the registry record.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: diarrhea (5.0%), thrombocytopenia (5.0%), appetite decreased (3.3%), dehydration (3.3%), fatigue (3.3%).

The registry adverse-event record lists 60 participants at risk for serious adverse events and 11 affected. It also lists 60 at risk for other adverse events and 45 affected. These are descriptive counts for the Sunitinib Malate group. They do not constitute a comparison with another group, and the record does not explain whether the categories overlap at the participant level.

The listed serious adverse-event terms and percentages are diarrhea, 5.0%; thrombocytopenia, 5.0%; appetite decreased, 3.3%; dehydration, 3.3%; fatigue, 3.3%; and abdominal pain, 1.7%. These entries identify selected serious-event terms in the registry. The record does not give counts for each term or describe event timing, duration, recurrence, or severity grading beyond the serious classification. The listed percentages should not be added together: the registry does not establish that the event categories are mutually exclusive.

Deaths are not reported in the registry record. The safety figures do not establish how risk compares with another treatment or with no treatment. They also do not describe all aspects of tolerability, such as dose changes or reasons for discontinuation, which are not reported in the registry record.

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

The registry record describes a study of sunitinib malate in previously untreated patients over the age of 70 with non-small cell lung cancer. It identifies a single-group Phase II design and defines disease control rate as the primary endpoint. The endpoint includes complete response, partial response, and stable disease lasting at least 6 weeks, in the evaluable population.

However, the registry does not report the primary endpoint result, its 95% confidence interval, or the evaluable-population denominator. It therefore does not show what disease control rate was observed or how precisely it was estimated. The enrollment figure cannot answer those questions because it is not identified as the analysis denominator.

The single-group design also limits what any reported rate could establish. A rate describes observed outcomes in the study group; without a concurrent randomized comparator, it is not a controlled estimate of how outcomes differ under another treatment or condition. The registry does not report comparative analyses, nor does it provide results for the listed secondary outcomes.

The safety record lists serious and other adverse-event counts and selected serious-event terms. These are descriptive registry figures. They do not provide a comparison of safety, and they do not capture every detail needed to characterize events or treatment exposure. Deaths are not reported in the registry record.

In general, a trial’s endpoint definition, population, analysis method, and uncertainty all contribute to interpretation. Here, the endpoint definition and intended confidence-interval summary are available, but the result and key analysis details are not. The record therefore supports understanding what the trial set out to measure, but not a conclusion about the magnitude or precision of the primary outcome, comparative effects, or the balance of benefits and harms.

14. Why This Trial Matters Statistically

This trial is statistically notable for the distinction between an outcome definition and an outcome result. The registry gives a detailed primary endpoint definition and specifies that disease control rate was to be expressed as a percentage with a 95% confidence interval. Yet it does not report the rate, interval, or evaluable denominator. Those missing quantities are essential for judging both the observed outcome and its precision.

The single-group design also shapes the appropriate interpretation. It can characterize outcomes observed under the study regimen, but does not create a randomized comparison. This distinction matters because a percentage in one group is not the same as a treatment contrast. The record’s allocation entry is NA, masking is none, and no comparator is identified.

The case also illustrates why the analysis population matters. The registry names the evaluable population but does not define it or report its size. Enrollment and endpoint analysis populations need not be interchangeable; readers need the actual denominator and participant flow to understand who contributed to an estimate. Those details are not reported in the registry record.

Finally, multiple outcome topics are listed, while the record reports zero analyses. Without results or a statistical analysis plan, it is not possible to assess how the secondary outcomes were analyzed or how multiplicity was handled. The record is therefore useful for identifying intended questions and design features, while also demonstrating the limits of interpreting a trial when estimates, uncertainty, and analysis details are absent.

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 for interpreting this trial record is that it does not report the primary disease control rate, its 95% confidence interval, or the evaluable-population denominator. The enrollment of 63 does not resolve that omission, because the record identifies the evaluable population as the endpoint population but does not say how many participants were included.

The study is single-group and has no comparator identified in the registry. Its design therefore does not support a randomized comparison. The absence of masking is reported, but the record does not explain who assessed response, whether assessment was independently reviewed, or how potential assessment influence was addressed.

The endpoint is defined using response categories and an assessment schedule, but the registry does not report the criteria for evaluability, participant flow, missing assessments, or handling of incomplete follow-up. It also does not report treatment duration, adherence, interruptions, dose modifications, or analysis methods. Sample-size justification, interim analyses, and a statistical analysis plan are not reported in the registry record.

Although secondary outcomes are listed, the registry reports zero analyses and gives no outcome estimates. The listed adverse-event counts and selected serious-event terms offer limited descriptive information; deaths are not reported, and additional details about timing, duration, grading, and participant-level overlap are unavailable. These gaps restrict interpretation of both outcomes and safety, and prevent a fuller assessment of the trial’s precision and analysis choices.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sunitinib Non Small Cell Lung Cancer Patients Over 70 — NCT00864721.
• Registry record last updated 2018-10-16; status last verified 2018-10.
• Results first posted 2018-10-16.
• 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

NCT00864721 is a completed Phase II, single-group trial sponsored by US Oncology Research. It studied outpatient oral sunitinib malate (Sutent) in previously untreated patients over the age of 70 with non-small cell lung cancer. The registry reports enrollment of 63, a start date of 2009-02, and primary completion in 2012-07. Allocation is listed as NA and masking as none.

The primary endpoint is disease control rate in the evaluable population, with assessment every 6 weeks until progressive disease or death due to any cause, up to 36 month. The registry specifies a 95% confidence interval but does not report the endpoint result, interval, or analysis-population size. Four secondary outcomes are listed, and zero analyses are reported. Safety entries include counts for serious and other adverse events and selected serious-event terms; deaths are not reported in the registry record.