This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Sunitinib in Treating Patients With Relapsed or Refractory Esophageal or Gastroesophageal Junction Cancer (NCT00702884), a na phase 2 study. The primary outcome is Progression-free Survival Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00702884 |
| Phase | Phase 2 |
| Condition | Esophageal Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Tony Bekaii-Saab |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory esophageal or gastroesophageal junction cancer were the intended population. The registry condition is listed as esophageal cancer; the study title and summary also include cancer of the gastroesophageal junction. The record lists an enrollment of 25. Further eligibility details and characteristics of enrolled participants are not reported in the registry record.
Intervention
The study used sunitinib malate, described as 37.5 mg daily for a 4 week cycle. The record characterizes the study as a phase 2, single-agent investigation. It does not describe a treatment schedule beyond that arm description.
Comparator
There was no separate comparison group: the design is listed as single group, with allocation recorded as not applicable and no masking. The study therefore does not provide a randomized, like-for-like comparison with another treatment or a control group.
Primary question
The primary outcome is titled “Progression-free Survival Rate,” with a median parameter and a 95% confidence interval, over a timeframe of up to 24 weeks. Its description refers to complete response, partial response, and stable disease assessed by RECIST criteria at 24 weeks. The record’s wording does not fully clarify how that description relates to the titled outcome or median parameter. A numerical primary result is not reported in the registry record.
3. Trial Design
This completed phase 2 study evaluated sunitinib malate as a single agent in patients with relapsed or refractory esophageal or gastroesophageal junction cancer. The registry lists a single-group model, allocation as not applicable, and no masking. The listed enrollment is 25. With no separate control arm, the design can describe outcomes among people treated in the study, but it cannot by itself estimate a randomized treatment contrast.
The intervention is described as sunitinib 37.5 mg daily for a 4 week cycle. The registry lists the condition as esophageal cancer, while the study title and summary refer to both esophageal and gastroesophageal junction cancer. Additional information about eligibility, baseline characteristics, treatment duration, adherence, or dose changes is not reported in the registry record.
The primary outcome title is Progression-free Survival Rate. It is described using a median parameter and a 95% confidence interval, with a timeframe of up to 24 weeks. Its description refers to complete response, partial response, and stable disease assessed by RECIST criteria at 24 weeks. These elements do not specify a single unambiguous outcome definition, and the record does not provide a numerical result to resolve that ambiguity.
Secondary outcomes include overall response rate, median overall survival time, median progression-free survival time, frequency and severity of adverse events, change in mean vessel density, and quantitative assessment of proliferating tumor cells and apoptosis. The registry lists 7 outcome measures and 0 analyses. The study start is recorded as 2008-06-30, primary completion as 2013-09-17, and status as completed. A detailed protocol, statistical analysis plan, and explanation of the enrollment target 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
| Stage | Sunitinib |
|---|---|
| STARTED | 25 |
| COMPLETED | 25 |
| NOT COMPLETED | 0 |
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.
| Characteristic | Unit | Sunitinib |
|---|---|---|
| Participants analysed | 25 | |
| Age, Customized | years | 61 (38 to 74) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 22 |
| Race/Ethnicity, Customized: White | patients | 23 |
| Race/Ethnicity, Customized: Black | patients | 2 |
| Region of Enrollment: United States | patients | 25 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival Rate, measured over up to 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | weeks |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is titled “Progression-free Survival Rate.” Progression-free survival generally describes the time until disease progression or death, while a rate at a specified time describes the proportion meeting a defined status at that point. A median is a time-to-event summary: it identifies the point at which the estimated event-time distribution reaches its midpoint. A confidence interval expresses uncertainty around an estimate; a wider interval generally indicates less precision than a narrower one.
The registry describes the primary measure with a median parameter and a 95% confidence interval, over a timeframe of up to 24 weeks. However, the endpoint description refers to complete response, partial response, and stable disease assessed by RECIST criteria at 24 weeks. Those response categories describe tumor status at an assessment, whereas a median progression-free survival measure concerns event timing. The registry record does not explain how these definitions were combined, or which event and censoring rules were used.
Time-to-event endpoints are often used when the timing of progression or death is important and follow-up may differ among participants. Their interpretation depends on clear definitions of the event, follow-up period, and handling of participants whose event has not been observed. Those details, along with the numerical primary estimate and its interval, are not reported in the registry record. The endpoint’s stated purpose can therefore be described, but its observed magnitude and precision cannot be evaluated from the record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a completed phase 2 study of sunitinib in a single group of patients with relapsed or refractory esophageal or gastroesophageal junction cancer. It lists 25 participants enrolled and describes the primary outcome, but a numerical primary efficacy estimate is not reported in the registry record. Consequently, the record does not establish the observed median progression-free survival, the proportion meeting a response or disease-control definition at 24 weeks, or the precision of any such estimate.
The primary outcome wording warrants caution. Its title refers to a progression-free survival rate, its parameter is median, and its description refers to RECIST response categories at 24 weeks. These formulations concern related but nonidentical statistical summaries. Without a clear operational definition and a posted estimate, a reader cannot determine which interpretation was used for the primary result. The 95% confidence interval is specified as the dispersion measure, but its limits are not reported in the registry record.
The single-group design has an important interpretive consequence. Outcomes observed among participants receiving sunitinib could describe the course of disease and events in this study population, but there is no concurrent control group to separate treatment-associated outcomes from other influences. The record does not support a randomized comparison, and no comparative treatment effect should be inferred from its design.
The registry lists secondary outcomes, including response rate, overall survival, progression-free survival, adverse events, vessel density, and tumor-cell measures. It lists 0 analyses. This means the record does not include a set of listed statistical analyses for those outcomes; it does not establish that no analyses were conducted outside the registry record. Numerical efficacy results and analysis details are not reported in the registry record.
The safety listing contains serious-event terms with percentages, including neutropenia, anemia, and fatigue. These are descriptive safety figures, not evidence from a controlled comparison. They do not, on their own, establish whether an event was caused by treatment or how its frequency compares with another treatment. The record also lists aggregate counts for serious and other adverse-event categories, but does not provide enough detail to reconstruct participant-level overlap, timing, or causal attribution.
The planning illustrations are not observed trial outcomes. They are explicitly based on an observed effect and hypothetical design assumptions; they should not be read as observed power or as proof that the study could detect a particular effect. The underlying efficacy estimate and its uncertainty are not reported in the registry record, so the planning scenarios cannot substitute for the missing trial result.
9. Statistical Methods Explained
The registry identifies a median time-to-event outcome and a 95% confidence interval, but does not report a numerical primary estimate or list analyses. The statistical concepts below are general explanations; they do not establish which procedures were actually used in this study.
What does a median time-to-event estimate describe?
In general, a time-to-event analysis uses both the occurrence and timing of a defined event. The event must be specified—for example, progression or death—and the rules for deciding when it occurred must be clear. A median event time summarizes the estimated distribution rather than the average follow-up time. It is meaningful only in relation to a defined event, population, and follow-up approach.
Time-to-event data can include participants whose event was not observed during their available follow-up. Such observations are commonly treated as censored: they contribute information up to their last known follow-up, without being treated as though an event occurred then. The analysis depends on when follow-up ended and on assumptions about censoring. Event definitions, censoring rules, and follow-up handling are not reported in the registry record.
What does a confidence interval communicate?
In general, a confidence interval conveys statistical uncertainty around an estimate. It is not a range containing a fixed proportion of individual participant outcomes, and it does not remove uncertainty about study design, outcome definition, or bias. An interval’s width reflects precision under the analysis assumptions; interpreting it also requires the estimate itself and the interval limits. Although the primary outcome lists a 95% confidence interval, neither the estimate nor its limits are reported in the registry record.
A confidence interval for a single-group outcome would describe uncertainty in that group’s estimate. It would not create a comparison with an untreated group or another therapy. In a nonrandomized, single-group study, the observed outcome can also reflect factors beyond the intervention. A comparative effect requires a suitable comparator and an analysis that directly estimates the contrast.
How should the endpoint wording be handled?
The registry’s primary outcome combines a title referring to a progression-free survival rate, a median parameter, and a description based on complete response, partial response, and stable disease assessed by RECIST criteria at 24 weeks. In general, a response assessment at a fixed time and a median time-to-event summary answer different questions. A rate would need a stated numerator, denominator, and assessment rule; a median time-to-event estimate would need an event definition and follow-up method. The registry record does not explain the relationship among these elements. Readers should not treat them as interchangeable.
What do the planning illustrations represent?
The planning illustrations are hypothetical design calculations, not a measure of what the completed study achieved. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. Time-to-event illustrations use a total number of events under the Schoenfeld approach, while other illustrations express participants per arm. These scenarios describe planning under assumed conditions. They are not observed power, do not change the single-group design of the actual study, and do not demonstrate that a treatment effect exists.
Because the actual trial has one group, the assumed equal allocation between two arms belongs to a hypothetical planning comparison, not to the study’s treatment assignment. Any sample-size illustration must also be interpreted in light of its assumed effect size and statistical model. The registry record does not report a sample-size justification or the actual numerical planning results.
What can be learned from the absence of listed analyses?
The registry lists 0 analyses. That signals that analysis details are not available in the record, rather than specifying an analytic method. The record does not describe model selection, covariate adjustment, handling of missing data, analysis populations, or interim analyses. It also does not give a statistical plan against which to assess whether outcomes were analyzed as intended. These omissions limit reproducibility and make it difficult to assess how the planned endpoint was translated into an analysis.
The record states that only sponsor-reported hazard ratios are used for time-to-event tables. No hazard ratio estimate is included in the registry entries, and a hazard ratio should not be inferred from a median or from an endpoint title. In general, a hazard ratio compares event rates over follow-up under a specified model; it is not the same as a difference in median times or a fixed-time response rate. Without a reported estimate, confidence interval, and analysis context, there is no hazard-ratio result to interpret.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 7 outcome measures, spanning efficacy, safety, and biologic measures, and lists 0 analyses. It does not report a multiplicity strategy, such as a prespecified hierarchy among outcomes or an adjustment to account for testing several hypotheses. Without such information, the chance of a false-positive finding across multiple analyses cannot be assessed. This is a general concern when several outcomes are examined; it does not imply that any particular analysis was performed or that a particular result is false.
Missing-data handling and analysis populations are not reported in the registry record. The record does not state how many participants contributed to each outcome, how incomplete follow-up was addressed, or whether analyses followed an intention-to-treat, per-protocol, or other population definition. Those details matter because missing measurements and exclusions can affect the population represented by an estimate.
The registry record also does not report interim analyses, a statistical analysis plan, or a sample-size justification. These absences prevent assessment of prespecification, outcome-selection decisions, and planned monitoring. They should be read as limits of the information in the record, not as proof that procedures were absent from the study itself. The primary endpoint wording further leaves uncertainty about whether the intended analysis concerned a fixed-time response status or a time-to-event summary.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Sunitinib | 25 | 25 | 100.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Sunitinib | 25 of 25 |
Most frequent serious adverse event terms
| Event term | Organ system | Sunitinib |
|---|---|---|
| Neutropenia | Blood and lymphatic system disorders | 8 of 25 |
| Anemia | Blood and lymphatic system disorders | 5 of 25 |
| Fatigue | General disorders | 4 of 25 |
| Abdominal Pain | Gastrointestinal disorders | 2 of 25 |
| Leukopenia | Blood and lymphatic system disorders | 2 of 25 |
| GI Hemorrhage | Gastrointestinal disorders | 2 of 25 |
| Nausea | Gastrointestinal disorders | 1 of 25 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 25 |
| Hypertension | Vascular disorders | 1 of 25 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Sunitinib |
|---|---|---|
| Fatigue | General disorders | 15 of 25 |
| Nausea | Gastrointestinal disorders | 13 of 25 |
| Anemia | Blood and lymphatic system disorders | 12 of 25 |
| Anorexia | Metabolism and nutrition disorders | 12 of 25 |
| Diarrhoea | Gastrointestinal disorders | 11 of 25 |
| Leukopenia | Investigations | 10 of 25 |
| Abdominal pain | Gastrointestinal disorders | 10 of 25 |
| Vomiting | Gastrointestinal disorders | 9 of 25 |
| Neutropenia | Investigations | 8 of 25 |
| Hyperglycaemia | Metabolism and nutrition disorders | 8 of 25 |
Serious events with the highest arm-level rate: neutropenia (32.0%), anemia (20.0%), fatigue (16.0%), abdominal pain (8.0%), leukopenia (8.0%).
The registry lists serious adverse-event terms with percentages: neutropenia, 32.0%; anemia, 20.0%; fatigue, 16.0%; abdominal pain, 8.0%; leukopenia, 8.0%; and GI hemorrhage, 8.0%. These are presented as serious-event figures. They are descriptive entries from a single-group study, not comparisons against another treatment or a control group. The record also lists 25 at risk and 25 affected in the serious adverse-event category, and the same counts for the other adverse-event category.
The percentages should be read as registry-reported values, not recalculated estimates. The record does not specify the number of events per participant, timing, duration, severity grading rules, or whether events were judged related to sunitinib. It also does not clarify overlap among the listed terms. Therefore, the percentages do not by themselves describe the number of unique participants experiencing any event or establish causation.
A deaths value is not reported in the registry record. The listed terms and counts do not indicate how these findings compare with expected risks or with another treatment. Safety interpretation is limited by the single-group design and the absence of additional detail on ascertainment and analysis.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed phase 2, single-group study evaluated sunitinib malate in relapsed or refractory esophageal or gastroesophageal junction cancer. It identifies the intervention schedule, a primary outcome description, secondary outcomes, and selected serious adverse-event figures. It does not report a numerical primary efficacy estimate or the analyses used to generate one. Accordingly, the record does not allow readers to assess the observed magnitude or precision of the primary outcome.
The listed primary endpoint is not fully resolved by its wording. A progression-free survival rate, a median time-to-event measure, and the proportion with complete response, partial response, or stable disease at a fixed assessment are not identical summaries. Without a clarified definition and a result, no one of these should be substituted for another. The absence of a result in the registry record is not evidence that the outcome was zero or that the study found no effect.
The single-group design also limits what can be concluded. It offers no randomized control comparison, so outcomes in the treated group cannot isolate the effect of sunitinib from other influences. The safety percentages describe reported events in the study context, but do not establish treatment causation or comparative safety. Likewise, planning illustrations based on assumed effects are hypothetical and are not evidence that the intervention did or did not produce a particular outcome.
These limits do not erase the study’s descriptive value. The registry documents a clinical investigation and its intended outcome areas, including radiographic and biologic measures. But conclusions about efficacy, comparative benefit, or the precision of an effect require posted results and sufficient analysis details, which are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because its registry record brings together an explicit single-group design, a complex primary endpoint description, and an absence of listed analyses. That combination illustrates why an outcome’s label alone is not enough to interpret a clinical result. The intended measure, event definition, assessment timing, and statistical summary need to align before a result can be understood.
The endpoint description refers to response categories at 24 weeks, while its title names progression-free survival and its parameter is median. That distinction matters because a fixed-time status and a time-to-event summary answer different questions and require different analytic details. The registry does not clarify the intended relationship or report a numerical estimate, making transparent endpoint specification especially important.
The study’s single-group structure is also central to statistical interpretation. A treatment-group outcome can be described, but a concurrent control is needed for a direct randomized comparison. This design distinction prevents a descriptive result from being mistaken for a comparative treatment effect.
Finally, the record lists multiple outcome areas and selected safety figures but does not describe multiplicity handling, missing-data procedures, or analysis populations. These are not merely technical details: they help readers judge how robustly the evidence was generated and which participants and outcomes an estimate represents. The record’s limitations therefore provide a practical example of how registry transparency supports responsible interpretation.
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 most immediate limitation is that the registry record does not report a numerical primary efficacy result, even though it names a primary outcome and a 95% confidence interval. The endpoint is also described in terms that do not clearly align: its title refers to progression-free survival rate, its parameter is median, and its description refers to response categories assessed at 24 weeks. The event definition, assessment rules, and handling of follow-up are not reported in the registry record.
The study is listed as single group, with no masking and allocation not applicable. There is no control arm for a direct comparison, so the design cannot separate outcomes associated with treatment from other influences. The registry lists 25 enrolled, but further participant characteristics and outcome-specific analysis populations are not reported in the registry record.
The registry lists 7 outcome measures and 0 analyses. It does not report a statistical analysis plan, multiplicity approach, missing-data method, interim analyses, or sample-size justification. A death count is not reported in the registry record. The safety listings also do not supply event timing, causal attribution, participant-level overlap, or a detailed account of how adverse events were collected and graded.
These limitations concern what the registry record documents. They do not establish that unreported procedures or analyses were never conducted. They do, however, restrict independent assessment of the endpoint, efficacy, precision, and safety findings from the information recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sunitinib in Treating Patients With Relapsed or Refractory Esophageal or Gastroesophageal Junction Cancer — NCT00702884.
- Registry record last updated 2017-03-21; status last verified 2017-01.
- Results first posted 2017-03-21.
- 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.
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18. Record Summary
NCT00702884 is a completed phase 2, single-group study of sunitinib malate in relapsed or refractory esophageal or gastroesophageal junction cancer. The registry lists 25 enrolled and describes sunitinib 37.5 mg daily for a 4 week cycle. No randomized comparator or masking is listed.
The primary outcome is titled Progression-free Survival Rate and is described with a median parameter and 95% confidence interval over up to 24 weeks, while its description refers to RECIST response categories at 24 weeks. A numerical primary result is not reported in the registry record. The record lists 7 outcome measures and 0 analyses. It includes serious adverse-event terms with percentages, but does not report a death count. Further statistical and outcome details are not reported in the registry record.