Sunitinib in Treating Patients With Kidney Cancer That Cannot Be Removed by Surgery: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib in Treating Patients With Kidney Cancer That Cannot Be Removed by Surgery (NCT00459979), a na phase 2 study. The primary outcome is Response to Sunitinib Therapy.

2. Clinical Question

The clinical question is whether treatment with sunitinib malate can reduce tumor burden in patients with renal cell carcinoma and an unresectable primary tumor enough to permit nephrectomy within 1 year of starting treatment. The registry frames this as a phase II study of sunitinib, with response defined by that treatment-related change in surgical eligibility rather than by tumor shrinkage alone.

This question links a biological change in the primary tumor to a clinically consequential decision about whether surgery can be undertaken. It is not equivalent to asking whether tumors shrink by a fixed percentage, whether all disease disappears, or whether survival improves. Those are distinct outcomes. The record lists tumor-size changes and progression-free survival among secondary outcomes, but their results are not reported in the registry record. The primary response count and its uncertainty are also not reported in the registry record, so the record does not establish how often the defined response occurred.

3. Trial Design

The registry describes a phase II study sponsored by Case Comprehensive Cancer Center. It is a single-group study of sunitinib malate in patients with renal cell carcinoma and unresectable primary tumors. Enrollment is listed as 30, and the study status is completed. The recorded start is 2007-03 and the primary completion is 2011-03.

There is no randomized allocation and no concurrent control arm described. Masking is listed as none. Therefore, the design does not support a like-for-like comparison between sunitinib and another assigned treatment. The intervention list includes sunitinib malate and conventional surgery, while the arm description identifies a Sunitinib group. The presence of surgery in the intervention list does not establish that surgery was a separate randomized arm; the record describes nephrectomy as part of the primary response definition, where eligibility for the procedure is the outcome criterion.

The primary endpoint uses a per-protocol population defined as participants who had received at least one cycle of sunitinib. The registry does not report how many participants met that criterion, how many began treatment, or how participants who did not receive a cycle were handled in any other analysis. Although enrollment is listed as 30, it should not be assumed that this is the denominator for the primary endpoint or for the adverse-event summaries.

The registry lists six secondary outcomes: complications or adverse events, progression-free survival, change in primary-tumor diameter, tumors that decreased in size, tumors with a 30% reduction in size, and median size reduction among tumors with some shrinkage. Results for these outcomes are not reported in the registry record. The number of analyses is listed as 0. Details of the protocol’s statistical plan, sample-size justification, interim analyses, and procedures for handling deviations 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.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: per protocol - analysis after at least one cycle of Sunitinib

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 Response to Sunitinib Therapy, measured over 1 year from start of treatment.

The primary endpoint is the number of participants classified as responding to sunitinib therapy. Response is defined as a reduction in tumor burden sufficient to permit nephrectomy within 1 year from the start of treatment. The registry’s no-response definition includes both failure to reach that surgical eligibility criterion within 1 year and removal from the study for any reason. The stated analysis population is per protocol, after at least one cycle of sunitinib.

This endpoint captures a treatment-related change with a practical surgical consequence: whether the primary tumor becomes operable according to the study’s definition. It differs from a simple measurement of tumor diameter. A tumor may change in size without meeting the criterion for surgery, and the endpoint does not require a specified percentage reduction. The registry separately lists tumor-size reduction outcomes, which indicates that they are not interchangeable with the primary response definition.

For a binary endpoint, the response count is commonly interpreted alongside the number of participants assessed and, when available, an interval estimate describing uncertainty. Those results are not reported in the registry record. The registry also does not report the clinical criteria used to decide that nephrectomy was permitted, beyond the stated reduction in tumor burden. Consequently, the endpoint definition is available, but the observed response frequency and the precision of that estimate cannot be assessed from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation starts with the endpoint, the population counted, and the design that generated the observations. In this study, the endpoint is a participant count: response is defined by tumor reduction sufficient to permit nephrectomy within 1 year. The analysis population is described as per protocol after at least one cycle of sunitinib. The registry does not report the number in that population or the number meeting the response definition, so the response proportion cannot be evaluated from the registry record.

What a participant count can tell us

A binary outcome places each assessed participant into one of two categories under the study definition: response or no response. The count of responses is meaningful only alongside the number assessed. A proportion expresses the share meeting the criterion, while a confidence interval communicates the uncertainty associated with estimating that proportion from a study sample. A narrow interval generally indicates greater precision than a wide interval. Neither a response count nor an interval is reported in the registry record, so there is no numerical estimate of response or precision to interpret.

The definition also determines classification. Here, a participant is classified as having no response if nephrectomy is not permitted within 1 year or if the participant is removed from the study for any reason. That rule combines the surgical eligibility outcome with a study-removal condition. The record does not explain how removal reasons were assessed or whether any participants were removed. It also does not describe an independent review process for deciding whether nephrectomy was permitted. These details matter because outcome classification depends on the operational rule and its application.

Why the design matters

In general, a single-group study estimates outcomes in the treated group but lacks a concurrent comparison group. Without such a group, the observed course cannot be directly contrasted with the course under another assigned strategy within the same trial. Randomization is one method used in comparative studies to reduce systematic differences between groups; no randomized allocation is described. The record lists allocation as not applicable, no masking, and a single-group model. Consequently, the design should be read as a study of outcomes in the sunitinib group, not as a comparative treatment-effect estimate.

The arm description identifies Sunitinib. Conventional surgery is also listed as an intervention, but the record does not describe a surgery comparison arm. In the primary definition, nephrectomy is the procedure that becomes permitted if the response criterion is met. The intervention listing alone does not establish that participants were assigned to surgery or that surgery was performed.

Analysis population and missing information

The primary endpoint population is per protocol after at least one cycle. This is not automatically the same as all enrolled participants. In general, restricting analysis to participants who started or continued treatment can change the population being described; interpretation depends on how exclusions and missing outcomes are handled. The registry does not report the number receiving at least one cycle, the number with an endpoint assessment, reasons for missing assessments, or the method used to handle missing data. Therefore, the extent and potential influence of missingness cannot be assessed.

The registry lists zero analyses. It does not report an estimate, confidence interval, hypothesis test, or detailed statistical analysis plan for the primary endpoint. The record also does not report a sample-size justification, stopping rule, interim analysis plan, or methods for adjustment for multiple outcomes. These omissions should not be filled by assumptions about standard practice.

Planning calculations and observed power

Planning illustrations based on an observed effect answer a different question from the study’s observed result. The stated assumptions use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the target effect is described as a stated fraction of the observed effect. The registry specifies that such calculations are illustrations rather than the trial’s own design. It also describes total events for time-to-event planning and participants per arm for other planning calculations.

Those assumptions do not match the recorded single-group design, which has no described two-arm comparison. Moreover, the observed primary effect is not reported in the registry record. No planning count should therefore be treated as the number enrolled, an observed outcome, or evidence that the study had a particular probability of detecting an effect. Sample-size planning is prospective; observed power is not a substitute for reporting an effect estimate and its uncertainty.

Multiple outcomes and time-to-event measures

The registry lists outcomes concerning adverse events, progression-free survival, and several measures of tumor-size change. Each outcome has its own definition and may require different statistical methods. For instance, a time-to-event analysis generally accounts for both the timing of an event and participants whose event status is not yet observed at the end of follow-up. A count or percentage of tumors shrinking does not answer the same question as progression-free survival or the primary surgical-eligibility endpoint. The registry does not report results or analysis methods for these secondary outcomes, so their estimates and precision cannot be assessed.

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(28)
events <- c(13)
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  28  13  46.4  27.5  66.1  29.5  64.2  29.4  64.3

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary response outcome and multiple secondary outcomes, including adverse events, progression-free survival, and several measures of tumor-size change. When multiple outcomes are examined, general statistical interpretation must distinguish a prespecified primary question from additional questions. Testing many outcomes can increase the chance of at least one apparently notable finding by chance; methods such as prioritizing outcomes or adjusting for multiple comparisons can address that issue. The registry does not report whether any multiplicity procedure was planned or used, and it does not report analyses for the listed outcomes.

Missing-data handling is also not reported in the registry record. The primary analysis population is described as per protocol after at least one cycle, but the number meeting that criterion is not reported. There is no reported account of participants lacking endpoint assessments, treatment discontinuations, reasons for removal, or methods for handling unavailable outcomes. The endpoint definition classifies removal from the study for any reason as no response, but the record does not say how that rule was applied in practice or how many participants it affected.

The enrollment figure is 30, while the serious and other adverse-event summaries each list 28 participants at risk. The record does not explain the difference between these listed populations. It also does not report whether all enrolled participants contributed to each secondary outcome or how incomplete follow-up was handled. Without denominators and missingness details for each analysis, readers cannot determine whether outcome summaries use consistent populations.

The registry does not report a statistical analysis plan, interim analyses, or endpoint-specific analysis populations beyond the primary population description. These are limits on what can be inferred from the registry record, not evidence that particular procedures were or were not used outside the 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: thrombosis/thrombus/embolism (21.4%), hemoglobin (14.3%), hemorrhage, gu - urinary nos (10.7%), pain - abdomen nos (10.7%), dyspnea (shortness of breath) (10.7%).

The serious adverse-event summary lists 28 participants at risk and 16 affected in the Sunitinib group. The other-adverse-event summary lists 28 at risk and 28 affected. These are participant-level summary counts as recorded; they do not reveal the number of separate events per participant. A participant may have experienced more than one event, and the record does not describe whether the summaries count participants, event episodes, or how repeated events were treated beyond the listed participant figures.

The serious-event terms include Thrombosis/thrombus/embolism at 21.4%, Hemoglobin at 14.3%, Hemorrhage, GU - Urinary NOS at 10.7%, Pain - Abdomen NOS at 10.7%, Dyspnea (shortness of breath) at 10.7%, and Supraventricular and nodal arrhythmia - Atrial fibrillation at 7.1%. These percentages are reported as terms in the registry, but the record does not provide accompanying event counts for each term or explain whether categories can overlap. The listed terms should not be read as mutually exclusive categories.

The record does not report event timing, severity grades, attribution to sunitinib, duration, or the number of deaths. It also has no concurrent control group for safety comparison. Therefore, these figures describe the recorded safety summaries but do not establish comparative risk or causation.

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

The study is a completed phase II, single-group investigation of sunitinib in renal cell carcinoma with unresectable primary tumors. Its primary endpoint asks whether tumor reduction permits nephrectomy within 1 year, with a per-protocol analysis population after at least one treatment cycle. This is a defined clinical outcome that connects tumor burden to surgical eligibility.

The registry record does not report the primary response count, the analysis denominator, or an estimate with an uncertainty interval. As a result, the record does not show how many participants met the endpoint, how frequent response was, or how precise an estimate would be. The listed enrollment of 30 cannot be assumed to equal the primary analysis population. The record also does not report results for progression-free survival or the listed tumor-size outcomes.

The design has no described randomized comparison group, and masking is listed as none. Even if an outcome estimate were available, a single-group result would describe the experience of participants in that group rather than directly establish a comparative effect against another treatment. The record’s surgical intervention listing does not establish a separate comparator arm; nephrectomy is part of the response definition.

The safety summaries report 16 affected among 28 participants at risk for serious adverse events and 28 affected among 28 at risk for other adverse events. Several serious-event terms and percentages are listed. These data do not by themselves establish causation, comparative safety, or the frequency of all event episodes; important details such as timing and attribution are not reported.

Accordingly, the registry supports understanding what the investigators intended to measure and provides limited safety counts, but it does not provide the primary outcome result needed to quantify response. The study should not be characterized as demonstrating a response rate or a treatment effect on the basis of the endpoint definition, enrollment figure, or planning assumptions alone.

14. Why This Trial Matters Statistically

This trial raises a useful statistical distinction between a tumor-response measure and a clinical decision threshold. The primary outcome is not simply whether a tumor became smaller. It asks whether the change was sufficient for nephrectomy to be permitted within 1 year. That makes the endpoint dependent both on tumor burden and on the assessment of surgical eligibility. The endpoint definition therefore needs to be read carefully before interpreting any response count.

It also illustrates why a denominator is essential. The registry lists enrollment of 30, but defines the primary analysis population as participants who received at least one cycle. The size of that population and the response count are not reported in the registry record. Without both, readers cannot estimate the response frequency or assess its precision. Enrollment alone does not fill that gap.

The design also determines the scope of conclusions. A single-group phase II study can describe outcomes in its treated group, but it does not supply a concurrent randomized comparison. This is not a minor technical detail: comparative claims require a basis for distinguishing outcomes under the study treatment from outcomes that might occur under another strategy. The record’s inclusion of conventional surgery among interventions does not change the listed single-group structure.

Finally, the registry juxtaposes a clinically framed primary endpoint with several secondary outcomes, including progression-free survival and tumor-size changes. These outcomes answer different questions and should not be treated as interchangeable. The absence of reported analyses, a documented multiplicity approach, and missing-data details further limits statistical interpretation. The case is a reminder that a clearly defined endpoint and completed status are not substitutes for posted outcome estimates and uncertainty measures.

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 principal limitation of interpretation is that the registry record does not report the primary response result. It also does not report the response-analysis denominator, an effect estimate, or an uncertainty interval. Consequently, neither the frequency of response nor the precision of an estimate can be evaluated.

The primary analysis is described as per protocol after at least one cycle, but the record does not state how many participants received a cycle or how many were assessed. It does not report reasons for removal, missing endpoint data, or the handling of participants without an assessment. Because removal for any reason is included in the no-response definition, knowing how that rule was applied would be important to understanding the outcome.

The study is single-group, with no randomized comparator described and no masking. This limits the kinds of comparative conclusions that could be drawn from outcomes, even if results were available. The record does not provide detailed criteria or procedures for determining that nephrectomy was permitted, beyond the stated tumor-burden reduction criterion.

The safety summaries give participant counts and selected serious-event terms but do not report deaths, event timing, severity grades, attribution, or the handling of repeated events. The different listed enrollment and safety at-risk counts are not reconciled in the registry record. Results for the listed secondary outcomes are also not reported, nor are methods for multiplicity, missing data, interim analyses, or the primary statistical analysis plan.

The stated planning assumptions use a two-arm framework that does not match the listed single-group design. The observed effect needed as the basis for the planning illustration is not reported in the registry record. These illustrations should not be treated as evidence about the trial’s actual design or statistical power.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sunitinib in Treating Patients With Kidney Cancer That Cannot Be Removed by Surgery — NCT00459979.
• Registry record last updated 2014-03-07; status last verified 2014-02.
• Results first posted 2014-01-07.
• 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

NCT00459979 is a completed phase II, single-group study sponsored by Case Comprehensive Cancer Center. It lists enrollment of 30, with a recorded start of 2007-03 and primary completion of 2011-03. The study concerns sunitinib malate in renal cell carcinoma with an unresectable primary tumor. Allocation is listed as not applicable, and masking as none.

The primary outcome is the number of participants whose tumor reduction permits nephrectomy within 1 year. The defined population is per protocol after at least one cycle of sunitinib. The primary response count and analysis denominator are not reported in the registry record. The record lists six secondary outcomes and zero analyses.

Safety summaries list 28 participants at risk for serious adverse events, with 16 affected, and 28 at risk for other adverse events, with 28 affected. Selected serious-event terms and percentages are recorded. The registry does not report the primary response result, secondary-outcome results, or deaths.