Sunitinib and Surgery in Treating Patients With Localized or Metastatic Kidney Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib and Surgery in Treating Patients With Localized or Metastatic Kidney Cancer (NCT00849186), a na na study. The primary outcome is Safety of Sunitinib Malate (SM).

2. Clinical Question

The study also records a secondary response-rate outcome after 90 days of treatment with sunitinib malate. The registry description frames the study as examining side effects and whether preoperative treatment may affect tumor size and the amount of normal tissue removed. Those aims describe the rationale and questions; they are not evidence that a particular response or surgical effect occurred.

The intervention is described in relation to nephrectomy, but the registry record does not report a comparison between patients receiving preoperative sunitinib and patients receiving surgery without it. With a single-group design, any observed outcome would describe the treated group over the specified follow-up, rather than estimate a treatment contrast against a concurrent control group. The registry record does not report the primary endpoint result, the response-rate result, or analyses that would quantify either outcome.

3. Trial Design

This is a completed pilot study of neoadjuvant sunitinib malate in patients with renal cell carcinoma undergoing nephrectomy. It is registered as a single-group study, with allocation listed as NA and masking listed as NONE. The listed enrollment is 20. The sponsor is Roswell Park Cancer Institute. The registry dates are 2007-08 for study start and 2009-07 for primary completion.

The treatment description includes sunitinib malate, neoadjuvant therapy, and therapeutic conventional surgery. The study title refers to localized or metastatic kidney cancer, while the official title describes patients with renal cell carcinoma undergoing nephrectomy. The brief summary explains the rationale for giving sunitinib before surgery and states that the trial studies side effects and treatment response. The intervention is therefore studied in a surgical context, not as a randomized comparison of alternative treatment strategies.

The record lists a 90-day timeframe for the primary safety outcome and a secondary outcome assessing response rate after 90 days of treatment with sunitinib malate. The primary outcome population is all treated and eligible patients. The adverse-event listing records 20 at risk for the sunitinib group, but the registry does not provide a flow description connecting enrollment, eligibility, treatment, follow-up, and endpoint assessment.

Details that would explain how treatment was administered, how surgery was timed, which eligibility criteria were applied, and how outcomes were assessed are not reported in the registry record. A protocol-level statistical plan, sample-size justification, and explanation of the phase designation are also not reported in the registry record. The listed status is completed, but that status alone does not establish that every planned outcome was analyzed or posted.

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: All treated and eligible patients

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: All treated and eligible patients

6. Primary Endpoint

The registry lists the primary outcome as Safety of Sunitinib Malate (SM), measured over 90 days.

The primary endpoint is a safety measure: the proportion of all treated and eligible patients who experienced a grade 3 or higher endpoint-relevant toxic event within 3 months of beginning treatment. It is expressed as a proportion of participants, with a 95% confidence interval identified as the dispersion measure. This type of endpoint focuses on whether a specified level of toxicity occurred during a defined observation period, rather than on the number of events or their timing.

In general, a proportion is useful for describing how common an event was in the population observed. The confidence interval conveys uncertainty around that estimated proportion; it is not a guarantee that the true value lies within its limits. Its width depends in part on the amount of information available and on the number of participants with and without the event. The interval is especially important when a study is small, because a point estimate alone can suggest more precision than the data support.

The registry record does not report the primary endpoint estimate or its confidence interval. The serious adverse-event count in the adverse-event listing is a separate summary and should not automatically be treated as the primary endpoint result: the primary definition is based on grade 3 or higher endpoint-relevant toxic events, while the listing is categorized by seriousness. The registry record does not report how those categories were mapped to the primary endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The main statistical feature is the single-group design. The study has no listed concurrent control group, so an analysis of outcomes within the treated group would be descriptive rather than a direct estimate of the difference between treatment strategies. In general, a comparison with a control group helps separate the treatment contrast from other influences on outcomes, such as patient characteristics, the course of disease, and changes in care over time. Without that comparison, observed outcomes cannot be attributed to sunitinib alone.

What does a proportion measure?

The primary outcome is a binary event summarized as a proportion: each eligible treated participant either meets the endpoint definition during the specified period or does not. A proportion uses the number meeting the definition relative to the number assessed for that endpoint. The registry record states the intended population and timeframe, but does not report the primary endpoint count, denominator used in analysis, or estimate. The adverse-event listing has its own at-risk and affected counts; those values should be interpreted within that listing’s categories rather than assumed to be the primary endpoint data.

In general, binary outcomes can be reported as counts and proportions. Such summaries are straightforward to read, but they do not show the timing, duration, recurrence, or clinical context of events. If participants have different amounts of observation or if some outcomes are missing, the proportion can also depend on how the analysis handles those circumstances. The registry record does not report missing-outcome handling or follow-up completeness for the primary endpoint.

Why is a confidence interval relevant?

A confidence interval communicates uncertainty around an estimated outcome. It gives a range of values compatible with the data under the chosen statistical method and assumptions. It is not a measure of clinical importance, and a narrow interval does not remove bias from a study design. Conversely, a wide interval indicates that the estimate is imprecise. The primary endpoint lists a 95% confidence interval as its dispersion measure, but the interval and point estimate are not reported in the registry record. The interval method is not reported in the registry record.

In general, uncertainty for a proportion depends on the amount of information and on how common the event is. With limited observations, even an estimate near an endpoint of the possible range may leave substantial uncertainty. Statistical methods can differ in their behavior in such settings. The registry record does not identify which method would be used, so no method-specific interpretation is appropriate.

What can a single-group result establish?

In general, a single-group estimate can describe the outcomes observed under the study conditions. It cannot, on its own, identify what would have happened to the same population under a different strategy. Differences between this group and an external population may reflect differences in eligibility, disease characteristics, care, or outcome definitions, rather than a treatment effect. The registry record does not report an external comparison or an adjusted comparative analysis.

The planned clinical sequence also matters for interpretation. Sunitinib is described as neoadjuvant treatment before nephrectomy, and the safety endpoint begins at treatment initiation. To interpret an endpoint fully, readers generally need operational definitions, assessment schedules, and rules for attributing events to treatment. The registry record gives the broad event threshold and observation timeframe, but detailed event adjudication and assessment procedures are not reported in the registry record.

What is over-dispersion, and is it relevant?

In general, over-dispersion means that observed variability is greater than a statistical model expects. It is often discussed for count outcomes when events cluster within people or when participants have differing underlying event rates. It can make standard errors too small if a model assumes less variability than the data contain. The stated primary outcome is a participant-level proportion, not a reported event-rate model, and the registry record does not report an analysis that assesses over-dispersion. It should not be presumed to have been assessed or to affect a particular estimate.

For this trial, the more immediate interpretive issue is that no primary endpoint analysis is reported. The adverse-event counts alone do not show the primary endpoint’s grade threshold, endpoint relevance, or confidence interval. They also do not describe repeated events or how such events were handled. The registry record does not report a statistical analysis plan, model, adjustment strategy, or interim analyses. Those details cannot be inferred from the broad endpoint description.

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 (Sunitinib)")
n      <- c(20)
events <- c(0)
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 (Sunitinib)  20  0  0.0  0.0  16.8  0.0  16.1  0.1  16.1

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 3 outcome measures, including the primary safety outcome and a secondary response-rate outcome after 90 days of treatment with sunitinib malate. It reports 0 analyses. The record does not explain how the outcomes were prioritized beyond identifying the primary endpoint, or whether any adjustment for multiple outcome testing was planned. Multiplicity matters in general because examining several outcomes can increase the chance of finding an apparently notable result by chance. The registry record does not report whether this issue was addressed.

Missing data and analysis populations also affect interpretation. The primary endpoint population is described as all treated and eligible patients. The record does not report the number of participants included in the primary analysis, whether every participant had endpoint follow-up, or how incomplete assessments were handled. It does not report whether participants who discontinued treatment, missed visits, or had unevaluable outcomes were included or excluded. Those details are needed to understand the denominator and potential effects of missingness.

The adverse-event listing records 20 at risk for the sunitinib group, but that value does not establish the analysis denominator for the primary endpoint. The registry record does not report a participant flow or a reconciliation of enrollment, treatment, eligibility, and outcome availability. It also does not report an intention-to-treat approach or another named analysis population. As a result, the posted adverse-event counts should not be treated as evidence that all outcomes were complete or analyzed under a particular population rule.

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 non-serious adverse event terms

The adverse-event listing separates serious events from other adverse events for the sunitinib group. It reports 0 affected among 20 at risk for serious adverse events, and 20 affected among 20 at risk in the other-adverse-event category. These figures describe affected participants in the listed categories; they do not give the number of individual events, their duration, or event-specific details. The record does not list serious adverse-event terms.

The categories should not be merged with the primary endpoint definition without further information. The primary outcome concerns grade 3 or higher endpoint-relevant toxic events within 3 months of beginning treatment, while the adverse-event listing is organized by seriousness. Seriousness and grade are not interchangeable descriptions, and the registry record does not report how the categories relate. Therefore, the serious adverse-event count is not a reported estimate of the primary endpoint.

Deaths are not reported in the registry record. The adverse-event counts also do not establish whether all treated and eligible patients were followed for the same duration. The record does not report the event collection method or the severity and timing of the other adverse events. These limitations restrict what the counts can say about the overall safety profile.

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

The registry record identifies a pilot study asking about safety and response when sunitinib is given before nephrectomy for renal cell carcinoma. It records a single-group design, enrollment of 20, and a primary safety endpoint defined over 3 months. It also includes adverse-event counts for the sunitinib group. These details describe the study and its posted safety summaries; they do not supply a complete analysis of the primary endpoint.

The serious adverse-event listing reports 0 affected among 20 at risk, while the other-adverse-event listing reports 20 affected among 20 at risk. Neither figure by itself establishes the frequency of grade 3 or higher endpoint-relevant toxic events, because the registry record does not state that the adverse-event categories correspond to the primary endpoint definition. The primary endpoint estimate and its 95% confidence interval are not reported in the registry record.

The secondary response outcome is named, but its result is not reported. There is no reported estimate comparing sunitinib with another strategy. The record therefore does not establish whether treatment changed response, tumor size, surgical tissue removal, or any other outcome relative to what would have occurred without preoperative treatment. The brief summary describes intended questions and biological rationale, not findings.

In general, absence of a reported analysis is not evidence that an outcome did or did not occur. It means the registry record does not permit readers to quantify that outcome from the posted information. The completed status indicates the study is recorded as completed; it does not fill in missing outcome estimates, analysis methods, or follow-up details. Interpretation should remain limited to the design and counts explicitly recorded.

14. Why This Trial Matters Statistically

This study is statistically informative as an example of why endpoint definitions and design features need to be kept distinct. The primary question concerns a grade-based, endpoint-relevant toxicity proportion over a stated period. The adverse-event listing, by contrast, reports counts under serious and other categories. Even when categories concern related aspects of safety, one cannot substitute for another unless the definitions and analysis rules are reported.

The single-group design also illustrates the difference between describing an outcome and estimating a comparative effect. A proportion in a treated group can characterize what was recorded in that group, but a comparator is needed for a direct treatment contrast. The registry record does not list one. That distinction is important when study rationales refer to possible benefits: an intended benefit is a reason to conduct a study, not a statistical result.

Finally, the record demonstrates the value of reporting estimates with their uncertainty and analysis populations. A point estimate alone would not indicate precision; a count without a clear endpoint match would not answer the primary question. Here, the primary estimate, confidence interval, detailed outcome analyses, and participant flow are not reported in the registry record. Readers can identify the intended safety endpoint and the posted adverse-event counts, but cannot reconstruct a complete efficacy or primary safety analysis.

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.

Several trial-specific limitations constrain interpretation. The study is single-group, with allocation listed as NA and no comparator arm listed. It therefore does not provide a concurrent basis for estimating the difference between neoadjuvant sunitinib and another treatment strategy. The record describes a pilot study, but does not report a sample-size justification or explain the phase designation.

The principal reporting limitation is that the registry record does not report the primary endpoint estimate or its 95% confidence interval. Although a response-rate outcome after 90 days is listed, its result is also not reported. The record lists 3 outcome measures and 0 analyses, leaving the outcome-level analysis details unavailable. It does not report the statistical analysis plan, endpoint-specific denominators, or handling of missing outcomes.

The adverse-event listing provides affected and at-risk counts but no event terms for serious events, and deaths are not reported in the registry record. It does not establish that the serious-event category matches the grade 3 or higher primary endpoint. The record also does not report detailed follow-up completeness, event assessment procedures, or how safety events were attributed to treatment.

Finally, the title and summary describe kidney cancer in localized or metastatic settings, while the official title focuses on renal cell carcinoma patients undergoing nephrectomy. The registry record does not provide enough eligibility detail to characterize the enrolled population more narrowly. These limitations do not imply a particular outcome; they mark what cannot be concluded from the recorded information.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sunitinib and Surgery in Treating Patients With Localized or Metastatic Kidney Cancer — NCT00849186.
• Registry record last updated 2015-02-06; status last verified 2015-01.
• Results first posted 2015-02-06.
• 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

The registry record describes a completed, single-group pilot study of neoadjuvant sunitinib malate in patients with renal cell carcinoma undergoing nephrectomy. Enrollment is listed as 20, the sponsor as Roswell Park Cancer Institute, and the study dates as 2007-08 to 2009-07. Masking is listed as NONE.

The primary endpoint is the proportion of treated and eligible patients with a grade 3 or higher endpoint-relevant toxic event within 3 months of beginning treatment, with a 95% confidence interval specified. The record does not report the endpoint estimate or interval. A secondary response-rate outcome after 90 days is listed, but its analysis is not reported. The adverse-event listing reports 0 affected for serious events and 20 affected for other adverse events among 20 at risk in each category. The record does not report how these categories map to the primary endpoint.