Efficacy and Safety of Sunitinib in Metastatic Gastric Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy and Safety of Sunitinib in Metastatic Gastric Cancer (NCT00411151), a non-randomized phase 2 study. The primary outcome is Objective Response Rate (ORR).

2. Clinical Question

The clinical context in the brief summary is the search for second-line options after first-line chemotherapy failure. It describes a need for additional options for patients with advanced or metastatic gastric cancer who were refractory to cisplatinum- or irinotecan-combination treatment. That rationale explains the study question, but it is not evidence of the trial’s outcome.

The design addresses response in a treated group, not whether sunitinib improves outcomes compared with another treatment or with no treatment. Without a randomized comparator, the trial cannot make a randomized estimate of a treatment contrast. The registry record also does not report response counts, survival results, or the detailed eligibility criteria. Conclusions about the clinical effect therefore cannot be drawn from the recorded primary-endpoint description alone.

3. Trial Design

The registry describes an open-label, multicenter phase II trial of sunitinib for patients with chemo-refractory metastatic gastric cancer. It lists a single-group model, non-randomized allocation, and no masking. Enrollment is recorded as 52. The study status is completed; the listed start is 2006-12 and primary completion is 2009-07.

Because there was one treatment group and no random assignment, the study is not a randomized comparison. Its results, if reported, would describe outcomes in the participants who received sunitinib rather than estimate a randomized difference between treatment arms. Comparisons with outcomes from other studies or historical expectations would require additional assumptions and are not reported as part of this registry record.

The brief summary frames sunitinib as a second-line palliative therapy and discusses the lack of established randomized second-line protocols at the time of the study rationale. That rationale should be kept distinct from the design: the registry record identifies a phase II, single-group study, not a randomized trial establishing comparative efficacy. It does not report a control regimen or a treatment allocation procedure beyond the non-randomized designation.

The listed condition terms are Gastric Adenocarcinoma and Barrett Esophagus. The registry record does not provide enough detail to clarify the role of each condition in participant eligibility. It also does not report the full protocol, inclusion and exclusion criteria, treatment schedule, dose modifications, follow-up procedures, or statistical analysis plan. Those omissions limit how specifically the design can be reconstructed from the registry entry.

The total enrollment is a registry count and should not be confused with the number included in the primary analysis. The endpoint description says the intention-to-treat population excluded one registered patient whose tumor diagnosis was not confirmed, but the analysis-population count itself is not reported in the registry record. No number of participants with evaluable response data is reported either.

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: Intention-to-treat (ITT) population comprises all patients registered, except one patient whose tumor diagnosis was not confirmed.

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 one year.

The primary endpoint is objective response rate (ORR), expressed as participants with a confirmed complete response or partial response. The description specifies that response is assessed within the first 6 treatment cycles and follows RECIST. A complete response means disappearance of all target lesions. A partial response means a 30% decrease in the sum of the longest dimensions of target lesions, using the baseline sum as the reference.

ORR is a binary-response summary: each participant is classified according to whether a qualifying confirmed response occurred, and the rate describes the proportion meeting that definition in the analysis population. The registry lists the endpoint unit as participants (%) and specifies a 95% confidence interval. However, it does not report the number of responders, the observed rate, or the interval limits.

General statistical context: response rate is used in clinical trials because it provides a defined measure of tumor shrinkage over a specified assessment period. It does not by itself describe how long a response lasts, how long participants remain free of progression, or overall survival. Those are different outcomes; progression-free survival and overall survival are listed as secondary outcomes in this trial.

The endpoint metadata also lists a timeframe of one year, while the endpoint description specifies response within the first 6 treatment cycles. Both descriptions are part of the registry record; it does not explain their relationship. The endpoint’s stated analysis population is all registered patients except one whose tumor diagnosis was not confirmed. The count for that population is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods are only partly described in the registry record. The endpoint is defined, its unit and confidence-interval type are named, and the trial is characterized as a single-group, non-randomized phase II study. A detailed statistical analysis plan is not reported in the registry record. The method explanations below distinguish the recorded design details from general statistical context.

What does a single-group design estimate?

The trial assigns no concurrent comparison group in the registry description. Its primary endpoint is a response rate within the treated population. General statistical context: a one-group rate estimates the frequency of an outcome under the observed study conditions. It does not, on its own, estimate the difference between treatments. Even if a response rate were available, interpreting it as an effect caused by treatment would require caution because other influences on the outcome are not controlled through random allocation.

Randomization is generally used to make treatment groups comparable, on average, with respect to measured and unmeasured baseline factors. This trial is listed as non-randomized and single-group, so that design-based protection is not available for a treatment-versus-control comparison. The registry record does not describe a matched control, external control, or other comparative analysis.

How should the response definition be read?

The primary outcome counts confirmed complete or partial responses under RECIST during the first 6 treatment cycles. The stated complete-response criterion is disappearance of all target lesions. The stated partial-response criterion is a 30% decrease in the sum of the longest dimensions of target lesions relative to the baseline sum. A participant who does not meet either response definition would not meet the primary response endpoint as described, though the registry record does not provide the rules for handling unevaluable assessments or missing scans.

The registry specifies a 95% confidence interval for the response rate. General statistical context: an interval communicates sampling uncertainty around an estimate, and its width depends in part on the amount of information available and the observed data. The interval does not show that the estimate is free from bias, and it does not compensate for the absence of a control group. Since neither the response count nor the interval limits are reported, no numerical interpretation of precision is possible.

What can be said about planning illustrations?

The statistical assumptions described for planning illustrations are two-sided significance level 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those are planning assumptions, not reported features of the trial’s design. The record’s own study is a single-group trial, so the two-arm illustration should not be read as a description of its enrollment or allocation.

General statistical context: a sample-size illustration asks how much information a hypothetical future design might require under stated assumptions. It is not an observed outcome and does not establish the probability that this completed study detected a true effect. The registry record does not report the trial’s own sample-size justification or planning assumptions. No numerical planning results are reported in the registry record, so no sample-size values can be interpreted.

The note accompanying the planning assumptions distinguishes time-to-event calculations, which use total events, from other calculations, which express participants per arm. That distinction concerns the interpretation of planning quantities. It does not imply that the present single-group trial had two arms or that any specific number of events was observed.

Why can over-dispersion matter?

General statistical context: over-dispersion occurs when observed variability exceeds what a chosen statistical model expects. If a model assumes less variability than the data have, standard errors can be too small and confidence intervals can be too narrow. Appropriate methods depend on the outcome and model. The registry record does not report an over-dispersion assessment or an outcome model for this trial, so no claim about whether it arose or how it was handled is warranted.

What does the analysis population mean?

The primary endpoint description identifies an intention-to-treat population consisting of all patients registered except one patient whose tumor diagnosis was not confirmed. This is the registry’s stated population rule. The analysis count is not reported in the registry record. General statistical context: analysis-population definitions affect the denominator and therefore the interpretation of a rate. Without the analysis count and the response count, the ORR cannot be reproduced or checked.

The trial is open-label with no masking. General statistical context: knowledge of treatment assignment can influence some aspects of assessment or care, depending on the outcome and procedures. A standardized response definition can make assessment more explicit, but the registry record does not describe independent review, assessor masking, or adjudication. These procedures are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The trial lists several secondary outcomes: progression-free survival, overall survival, one-year survival, adverse events, serious adverse events, and adverse events reported in at least 10% of patients. The primary outcome is ORR. The registry record does not report a multiplicity strategy, such as an adjustment for testing several outcomes or a hierarchy governing interpretation. It also does not report the number of statistical analyses performed; the recorded analysis count is zero. That entry should not be taken to mean that no analyses were conducted outside the registry fields.

General statistical context: when several outcomes or hypotheses are examined, the chance of at least one apparently unusual result can increase if each is assessed without accounting for the others. Multiplicity methods can address particular testing goals, but the appropriate approach depends on the analysis plan. Since no such plan is reported, the interpretation of any secondary-outcome tests cannot be assessed from the registry record. The secondary outcome results themselves are not reported in the registry record.

The endpoint definition identifies the intention-to-treat population as all registered patients except one whose tumor diagnosis was not confirmed. The registry does not state how missing response assessments were handled, whether any participants were unevaluable, or whether any additional analysis populations were used. Those details are not reported in the registry record. The primary analysis denominator is also not reported.

General statistical context: missing outcome data can affect a rate if the probability of missingness is related to response or other participant characteristics. An analysis may use different rules for missing data, and the choice can influence the estimate. Without the number of missing assessments and the analysis rule, readers cannot evaluate this issue for the ORR. The record does not provide these details, so no missing-data method or assumption should be attributed to the trial.

The registry record also does not report interim analyses, stopping rules, or a prespecified statistical analysis plan. Their presence or absence cannot be inferred from the completed status or the endpoint description.

11. 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.

The recorded serious-adverse-event group is Sunitinib. The registry lists 52 participants at risk and zero affected by serious adverse events. This is the serious-event count recorded for that group. It is not a complete account of safety, because the record also lists other safety outcomes and does not report their results.

The deaths figure is not reported in the registry record. The list of top serious-event terms is empty, and the record does not provide further serious-event descriptions. The serious-adverse-event count should therefore be interpreted only as the specific posted count, not as a statement that no deaths occurred or that no adverse events of any kind occurred.

General statistical context: when an event is uncommon, a count of zero in a study group does not establish that the event is impossible or that its underlying risk is zero. Precision depends on the amount and completeness of safety follow-up, among other factors. The registry record does not provide enough information about follow-up, event ascertainment, or the broader safety results to assess those aspects.

The registry separately lists adverse events, serious adverse events, and adverse events occurring in at least 10% of patients as secondary outcomes. Their results are not reported in the registry record. The serious-event count alone should not be used as a substitute for those unreported summaries.

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

This trial’s registry entry documents a phase II evaluation of sunitinib in a single-group, open-label, non-randomized study of chemo-refractory metastatic gastric cancer. It defines ORR as the primary endpoint and gives the response criteria and intended analysis population. Those details clarify what the study set out to measure, but they do not establish the observed response outcome.

The primary response estimate and its 95% confidence interval are not reported in the registry record. The registry also does not report results for progression-free survival, overall survival, one-year survival, or the listed adverse-event outcomes beyond the serious-adverse-event count. Consequently, the available record does not support a quantitative conclusion about efficacy, response precision, survival, or the full safety profile.

The design further limits the type of inference possible. General statistical context: a response rate in a single treated group is descriptive of that group and does not provide the same evidence as a randomized comparison. It cannot isolate a treatment effect from differences in prognosis, participant selection, or other influences. No comparator arm is listed, and there is no randomized treatment contrast to interpret.

The posted serious-adverse-event count is zero affected among 52 at risk in the Sunitinib group. This count is useful as a narrow safety observation, but it does not establish that no other adverse events occurred, nor does it supply the unreported deaths figure or other safety outcomes. The absence of those results limits a broader benefit-risk interpretation.

The trial can therefore be understood as a study with a clearly described response question and a completed status, but the registry record does not include the main outcome results needed to judge that question. No conclusion that sunitinib did or did not produce a particular response rate, survival outcome, or overall clinical effect follows from the information recorded.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a defined endpoint and a reported result. The registry specifies ORR, the response criteria, the assessment period, and a confidence-interval type. Yet it does not report the response estimate, responder count, or interval limits. A well-described outcome does not substitute for the data needed to interpret that outcome.

The design also makes the estimand important. A single-group study can describe outcomes among treated participants, but it does not directly estimate a randomized difference between treatments. The non-randomized, open-label structure should therefore shape how any reported outcomes are interpreted. A response result, if available, would need to be read as a group outcome rather than a controlled causal contrast.

The registry’s population definition is another consequential detail. It says the intended primary analysis excludes one registered patient whose tumor diagnosis was not confirmed. Because the analysis denominator and response count are not reported, the rate cannot be checked or reconstructed. That illustrates why both the endpoint definition and the analysis population matter for interpreting a proportion.

The entry also separates efficacy and safety questions. ORR is the primary endpoint; progression-free survival, overall survival, one-year survival, and safety measures are listed as secondary outcomes. The registry does not report those results, while it does record zero participants affected by serious adverse events among 52 at risk. That narrow safety field should not be mistaken for a complete safety analysis.

Finally, planning assumptions should not be confused with the design of the completed study. The registry describes hypothetical two-arm sample-size illustrations, while the actual trial is listed as single-group. Keeping planning calculations, observed estimates, and trial design distinct is essential to avoid attributing results or methods to the study that the registry does not report.

14. 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 the registry information is that it does not report the primary ORR result or its 95% confidence interval. It also does not report the primary analysis denominator, response count, or number with missing or unevaluable assessments. The primary outcome therefore cannot be quantified or independently checked from the registry record.

The single-group, non-randomized design has no concurrent control arm. Even if outcome estimates were available, the design would not provide a randomized comparison with another treatment. The open-label designation and absence of masking are recorded, but details about response assessment, independent review, or adjudication are not reported in the registry record.

The population is described as chemo-refractory metastatic gastric cancer, while the condition list includes Gastric Adenocarcinoma and Barrett Esophagus. The detailed eligibility criteria and the relationship of each listed condition to enrollment are not reported in the registry record. Treatment dose and schedule, follow-up procedures, and the handling of treatment discontinuation are also not reported.

The endpoint description refers to response within the first 6 treatment cycles, while its timeframe is listed as one year. The registry record does not explain how those time descriptions relate. The analysis plan, missing-data methods, multiplicity handling, sample-size justification, and interim-analysis procedures are not reported in the registry record.

Finally, safety reporting is incomplete in the available record. It lists zero participants affected by serious adverse events among 52 at risk, but the deaths figure is not reported, and other listed safety outcomes lack reported results. This limits any interpretation of the overall safety experience.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy and Safety of Sunitinib in Metastatic Gastric Cancer — NCT00411151.
• Registry record last updated 2011-01-20; status last verified 2011-01.
• Results first posted 2011-01-14.
• 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.

18. Record Summary

The registry record describes NCT00411151, titled “Efficacy and Safety of Sunitinib in Metastatic Gastric Cancer,” as a completed, open-label, multicenter phase II trial. It lists non-randomized allocation and a single-group model, with enrollment of 52. The recorded start is 2006-12 and primary completion is 2009-07. Sunitinib-malate is the intervention.

The primary endpoint is ORR, defined by confirmed complete or partial response under RECIST. Its description specifies assessment during the first 6 treatment cycles and an intention-to-treat population excluding one registered patient whose tumor diagnosis was not confirmed. The registry does not report the ORR estimate or confidence interval.

Progression-free survival, overall survival, one-year survival, and safety outcomes are listed as secondary outcomes. The Sunitinib safety group records 52 at risk and zero affected by serious adverse events; the deaths figure is not reported. The registry record does not provide the other outcome results or a detailed statistical analysis plan.