Sunitinib in Never-Smokers With Lung Adenocarcinoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib in Never-Smokers With Lung Adenocarcinoma (NCT01829217), a na phase 2 study. The primary outcome is Objective Response Rate (ORR).

2. Clinical Question

The study asked whether objective tumor responses would be observed during treatment with sunitinib in the specified lung adenocarcinoma population. It was a Phase II trial, not a randomized comparison of sunitinib against another treatment or against no treatment. Accordingly, its primary question is about response observed in the treated group, rather than a difference in response between treatment groups.

The registry description gives a biological and clinical rationale for studying this drug and population, but rationale is not evidence of an outcome. The posted record does not establish how many participants had complete response, how many had partial response, or how many had neither. It also does not report a response estimate, a confidence interval for that estimate, or an analysis comparing subgroups. Those missing results prevent a quantitative assessment of the primary question from the registry record.

3. Trial Design

This was a Phase II, single-group trial sponsored by Dana-Farber Cancer Institute. The record lists enrollment of 13 and a completed status. The start is recorded as 2013-05 and primary completion as 2017-08. The study had one intervention, sunitinib, and no registered comparison arm. Allocation is listed as NA and masking as none.

The intervention description specifies oral sunitinib in a repeating 42 day cycle: daily treatment for the first 28 days, followed by 14 days off. This schedule describes the planned regimen in the record; it does not establish how much treatment any participant actually received, whether doses were changed, or whether treatment was stopped early. Those details are not reported in the registry record.

The population description identifies lung adenocarcinoma and two molecular or smoking-history categories: tumors without mutations in EGFR, KRAS, or ALK among never-smokers, or tumors with a RET mutation. Never-smoking is defined in the record as fewer than 100 cigarettes over a lifetime. The record does not provide the number enrolled in each category or a breakdown by other participant characteristics.

Objective response rate was assessed at 6 weeks post-registration and every 6 weeks until documented disease progression or death, up to January 23, 2017 (approximately 44 months). The registry does not specify whether all participants had assessments at every scheduled time, how missing or unevaluable assessments were handled, or which assessment determined each participant’s response classification.

The record does not report a protocol-level statistical analysis plan, a target sample size justification, interim monitoring procedures, or a detailed account of the analysis population. A completed trial status does not by itself indicate what the primary endpoint result was or whether any prespecified statistical criterion was met.

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

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 Objective Response Rate (ORR), measured over ORR was assessed at 6 weeks post-registration and every 6 weeks until date of documented disease progression or death, up to January 23, 2017 (approximately 44 months)..

The primary endpoint was objective response rate (ORR), expressed as the percentage of participants with evidence of complete or partial response under RECIST1.1 criteria. It is a binary-outcome summary: each participant is classified according to whether a qualifying response was recorded, and the rate describes the proportion classified as responders. The registry record identifies the outcome unit as a percentage of participants and specifies a 95% confidence interval as the measure of dispersion.

Response is used in clinical trials because it provides a defined way to summarize whether measurable disease met criteria for complete or partial shrinkage during assessment. Applying a named response standard aims to make classifications more consistent than an informal description of tumor change. The registry states that ORR was assessed at 6 weeks post-registration and every 6 weeks until documented disease progression or death, up to January 23, 2017 (approximately 44 months).

ORR is not the same as duration of response, progression-free survival, overall survival, symptom change, or treatment tolerability. A response-rate result alone would not describe how long a response lasted or establish a comparative treatment effect in a single-group study. The registry record does not report the observed ORR, the component counts for complete and partial response, or the stated confidence interval. Therefore, the endpoint definition and assessment schedule are available, but its numerical result cannot be evaluated from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a response rate summarize?

ORR is a binary-outcome measure. In general, an individual is classified as meeting or not meeting a response definition, and the response rate is the proportion classified as having a complete or partial response. This trial’s record names RECIST1.1 criteria and describes the endpoint as a percentage of participants. It does not give the numbers of participants in the response categories, so the response proportion cannot be reconstructed from the posted record.

A binary summary is useful because it reduces a complex set of tumor assessments to an interpretable endpoint. That simplicity does not mean the underlying assessment process is simple. The classification depends on the response criteria, the timing and availability of assessments, and rules for handling missing, conflicting, or unevaluable data. The registry record does not describe those analysis rules beyond the named criteria and assessment schedule.

What does a confidence interval contribute?

In general, a confidence interval gives a range of values compatible with the observed data under a specified statistical method. For a response proportion, it provides information about precision that a point estimate alone cannot convey. A wider interval indicates greater uncertainty in the estimate than a narrower interval, all else equal. A confidence interval is not a guarantee about the true value and does not remove bias arising from study design or measurement.

The record specifies a 95% confidence interval for ORR but does not post the interval, the point estimate, or the calculation method. It is therefore not possible to describe the estimated response rate’s precision. The confidence level alone does not supply a result. Nor does the record state whether the interval accounts for any particular assessment or analysis-population considerations.

Why does the single-group design matter?

In a single-group trial, the observed response rate is descriptive of outcomes in the enrolled and assessed group. Without a concurrent comparison group, it does not directly quantify a difference between sunitinib and another treatment, and it cannot isolate the treatment’s effect from other influences. This is a general design limitation. It does not establish that the observed outcomes, if reported, would be due to any specific alternative cause.

The record lists no allocation process and no masking. It identifies the model as single-group and describes no comparator. As a result, a two-arm treatment contrast is not part of the registered design. The design may still describe outcomes under the study regimen, but a causal or comparative interpretation would require evidence beyond a response proportion in one group.

What can repeated assessments tell us?

The endpoint schedule begins at 6 weeks post-registration, then repeats every 6 weeks until documented disease progression or death, up to January 23, 2017 (approximately 44 months). Repeated assessments can capture changes over time, but they also make the operational definition of response important. For example, analyses must specify how assessments are combined into a participant-level response status and how incomplete follow-up is treated. Such rules are not reported in the registry record.

The primary endpoint is ORR, not a time-to-event endpoint. The assessment window does not itself provide a response duration or survival estimate. Although progression and death are referenced as stopping points for repeated ORR assessment, the record does not report a progression-free survival or overall survival result as a primary outcome.

How should planning calculations be read?

General sample-size calculations estimate how many participants a future study might need under stated assumptions. They are planning illustrations, not results from the enrolled trial and not observed power. The registry’s stated illustration assumptions refer to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with an effect defined as a stated fraction of the observed effect. The record also says that time-to-event illustrations use total events, while other illustrations use participants per arm.

Those assumptions describe a hypothetical two-arm planning exercise, whereas this trial is registered as single-group. No illustrated sample-size values or observed response effect are reported in the record. Consequently, there is no basis for describing a particular future sample size or treating a planning scenario as evidence about the trial’s achieved statistical power.

What statistical details remain unavailable?

The registry record lists no analyses and does not report the primary endpoint estimate. The statistical analysis plan, analysis population, missing-data approach, confidence-interval method, handling of response assessments, and any prespecified hypothesis test are not reported in the registry record. The record also does not state whether interim analyses took place or how multiplicity was managed. These gaps limit reproducibility and prevent a detailed appraisal of the analysis beyond the endpoint definition and design information that are registered.

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")
n      <- c(13)
events <- c(1)
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  13  1  7.7  0.2  36.0  1.4  33.3  1.8  33.9

11. Multiplicity, Missing Data and Analysis Populations

The primary outcome named in the registry is ORR. The record lists one outcome measure and no analyses. It does not report a statistical analysis plan, so the prespecified testing strategy, if any, is not reported in the registry record. No interim analyses or stopping rules are described. The record also does not state whether additional outcomes or subgroup analyses were planned outside the listed primary outcome.

Multiplicity refers, in general, to the increased chance of a false-positive finding when many hypotheses or outcomes are tested. A prespecified strategy can clarify which comparisons are confirmatory and whether adjustments are used. The registry record does not describe a multiplicity strategy. Since no analysis results are listed, there are no posted hypothesis-test findings whose multiplicity adjustment can be assessed.

Missing data are also important for a response-rate endpoint. If response assessments are unavailable or participants stop being assessed, the analysis needs a rule for classifying or excluding those cases. The record describes assessment timing but does not say how missing, unevaluable, or incomplete assessments were handled. The analysis population—such as whether it includes all enrolled participants or only those with evaluable assessments—is not reported in the registry record.

The record reports enrollment of 13, but enrollment alone does not establish how many participants contributed to the primary endpoint analysis. No response counts or analysis denominator are posted. The record also does not report whether all enrolled participants began treatment, completed treatment, or had assessments at the scheduled times. These absences make it impossible to evaluate attrition or determine how missing assessments might affect the response estimate.

In general, transparent reporting of analysis populations, missing-data rules, and testing plans is needed to understand how a result was produced and how sensitive it may be to analytic choices. Those details, along with the numerical primary outcome, are not reported in the registry 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 non-serious adverse event terms

The registry lists safety figures for the Sunitinib group. Serious adverse events are recorded for 0 participants affected among 13 at risk. Other adverse events are recorded for 9 participants affected among 13 at risk. Deaths are recorded as 7. These are counts as listed in the record; no percentages are calculated.

The record does not list any serious adverse event terms. It does not explain the definitions or observation period for the non-serious category, provide event-level detail, or indicate whether an event was considered related to sunitinib. It also does not say whether deaths occurred during treatment or follow-up, or whether they were attributed to disease, treatment, or another cause. The death count should not be treated as a count of treatment-related deaths or as a serious-adverse-event count.

Safety counts describe recorded events within the registry’s categories, but their interpretation depends on the collection period, definitions, and attribution procedures. Those details are not reported in the registry record. With no comparison group, these figures also do not estimate whether event frequency differs from another treatment or from an untreated group. The counts should be read separately from the missing ORR result and do not establish the treatment’s benefit or harm.

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

The record establishes that a Phase II, single-group study of sunitinib was completed, with enrollment of 13, and that objective response rate was the primary endpoint. It defines response using complete or partial response under RECIST1.1 and gives a repeated assessment schedule. It also lists safety counts for the Sunitinib group.

It does not report the observed ORR, the number of complete or partial responses, or the 95% confidence interval. Therefore, the posted information does not allow readers to determine the response rate or its precision. A completed status is not an endpoint result, and the fact that ORR was assessed does not reveal what those assessments found.

Even if a response-rate estimate were available, a single-group result would describe the outcomes recorded in that group; it would not, by itself, show what would have happened under a different treatment or without sunitinib. The absence of a comparator limits direct comparative interpretation. This design feature does not invalidate a descriptive response measure, but it limits the claims that can be made from it.

The safety counts also have boundaries. The record reports 0 serious adverse events among 13 at risk, 9 participants with other adverse events among 13 at risk, and 7 deaths, but it does not report event terms, attribution, or the relationship between the deaths and adverse events. These figures cannot establish why an event occurred or whether it was caused by treatment.

Overall, the registry record documents the study question, design, endpoint definition, assessment schedule, and selected safety counts. It does not provide enough outcome information to quantify the primary endpoint or judge its statistical precision. It should not be read as evidence that sunitinib did or did not produce a particular response rate, nor as a controlled estimate of treatment effect.

14. Why This Trial Matters Statistically

This trial is statistically notable for the gap between a clearly stated clinical endpoint and the outcome information available in the registry record. ORR is defined, RECIST1.1 is named, and the assessment schedule is described. Yet the observed rate and its confidence interval are not reported. Without those results, readers cannot assess even the descriptive magnitude or precision of the primary endpoint.

The single-group structure also shapes what the study can answer. A response proportion in one treated group is not a randomized treatment contrast. Statistical interpretation must distinguish describing outcomes from estimating a comparative effect. This distinction is important whenever a study lacks a concurrent control group, regardless of how precisely an outcome is measured.

The record provides an example of why uncertainty measures and analysis details matter alongside endpoint definitions. It names a 95% confidence interval, but the interval itself is absent. It gives repeated assessment timing, but not rules for participant-level classification or missing observations. It lists safety counts, but not serious adverse event terms or attribution. Each omission limits a different part of interpretation.

Finally, planning calculations need to be kept separate from trial findings. The stated planning framework assumes equal allocation between two arms and a normal approximation, while the registered study is single-group. A hypothetical planning illustration cannot replace an observed outcome, establish achieved power, or resolve the lack of a comparator. Taken together, the record underscores the value of reporting outcome estimates, uncertainty, analysis populations, and analytic rules in a form that allows independent appraisal.

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 most consequential limitation in the registry record is that it does not report the primary ORR result or its 95% confidence interval. Without the response estimate or its component counts, the main endpoint cannot be evaluated quantitatively. The record lists no analyses, leaving unclear whether the result was analyzed but not posted or whether other analyses were conducted.

The single-group design has no registered comparator, so the study cannot provide a randomized estimate of the difference between sunitinib and another treatment. Allocation is listed as NA and masking as none. The registry record does not describe measures intended to reduce assessment bias or specify how the response criteria were applied in practice.

Further limitations arise from missing methodological details. The analysis population, handling of missing or unevaluable scans, response-confirmation rules, confidence-interval method, and statistical analysis plan are not reported in the registry record. Nor does it describe interim analyses, stopping rules, or multiplicity procedures. The enrollment of 13 is recorded, but the number contributing to the primary outcome analysis is not given.

The safety information is also limited: no serious adverse event terms are listed, and the record does not provide event timing, treatment attribution, or an explanation of how deaths relate to adverse events. The registry does not state how long safety events were collected or whether all participants had the same observation period.

These limitations concern what can be determined from the registry record. They do not establish that unreported analyses or details were absent from other study documents; those details are simply not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sunitinib in Never-Smokers With Lung Adenocarcinoma — NCT01829217.
• Registry record last updated 2018-10-31; status last verified 2018-10.
• Results first posted 2018-09-04.
• 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

NCT01829217 is a completed Phase II, single-group trial sponsored by Dana-Farber Cancer Institute, with enrollment of 13. The registered intervention was oral sunitinib on a 42 day cycle, with treatment during the first 28 days followed by 14 days off. The study focused on specified lung adenocarcinoma groups involving never-smoking history or RET mutation status.

The primary endpoint was ORR under RECIST1.1, assessed at 6 weeks post-registration and every 6 weeks until documented progression or death, up to January 23, 2017 (approximately 44 months). The registry record does not report the ORR estimate, response counts, or 95% confidence interval, and lists no analyses.

Safety figures list 0 serious adverse events among 13 at risk, other adverse events for 9 among 13 at risk, and 7 deaths. Serious adverse event terms and event attribution are not reported in the registry record. The design has no comparison group, so the posted information cannot provide a controlled treatment-effect estimate.