Sunitinib Malate in Treating Patients With Recurrent or Metastatic Endometrial Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sunitinib Malate in Treating Patients With Recurrent or Metastatic Endometrial Cancer (NCT00478426), a na phase 2 study. The primary outcome is Objective Response Rate.

2. Clinical Question

3. Trial Design

This was a phase 2 clinical study of sunitinib malate for recurrent or metastatic endometrial cancer. The registry describes its design as a single-group study, with no masking and allocation listed as not applicable. All participants were assigned to the treatment described in the record; there was no listed control arm. This design can describe outcomes observed among treated participants, but it does not provide a randomized comparison that separates treatment effects from other influences on outcomes.

The treatment schedule was sunitinib malate by mouth once daily on days 1–28, with courses repeating every 6 weeks in the absence of disease progression or unacceptable toxicity. The study status is completed. The registry lists enrollment as 34, while the primary outcome description states that 33 participants were considered for response and that 28 were evaluable for response. These figures refer to different recorded populations; the registry record does not explain the difference or provide a participant flow account for the response analysis.

The primary endpoint was objective response rate, defined using complete and partial response under RECIST v1.0 and assessed by MRI and/or CT. The listed timeframe is up to 7 years. Secondary outcomes include prolonged stable disease, overall survival, adverse effects assessed using CTCAE Version 3.0, and time to progression. The information reported does not include numerical results for the primary endpoint or the secondary outcomes.

The record does not report a statistical analysis plan, a rationale for the enrollment target, or details about interim analyses. It also does not report a prespecified control rate or a benchmark against which the single-group response outcome was to be judged. Without a comparator and without the primary response count, the available registry information supports a description of the intended design and endpoint, but not a treatment-effect estimate.

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: Of the 33 participants, only 28 were considered evaluable for response.

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: 34 women with with endometrial cancer (subtypes included endometrioid, serous and carcinosarcoma) were enrolled onto the study. One participant with endometroid endometrial cancer withdrew consent prior to treatment, resulting in 33 participants receiving at least one dose of the study drug and were thus considered eligible for analysis.

6. Primary Endpoint

The registry lists the primary outcome as Objective Response Rate, measured over Up to 7 years.

The primary endpoint is objective response rate: the rate of complete or partial response assessed using RECIST v1.0. The record describes imaging by MRI and/or CT and gives a timeframe of up to 7 years. Under the stated criteria, complete response means disappearance of all target lesions. Partial response requires a decrease of >=30% in the sum of the longest diameter of target lesions. Stable disease does not meet the criteria for sufficient shrinkage to qualify as partial response or sufficient increase to qualify as progressive disease. Progressive disease is defined as a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions.

Response is commonly used in oncology trials to describe whether measurable tumors shrink or disappear according to defined assessment rules. A prespecified response definition makes the outcome more consistent than an informal judgment, although imaging assessments and the choice of evaluable population still matter to interpretation. A response rate is a count-based endpoint: it depends on how many participants meet the response definition and on the analysis population used as its denominator.

For this study, the registry says 33 participants were considered for response and 28 were evaluable. It does not report the counts with complete response or partial response, or the objective response rate. Thus, the endpoint’s definition is available, but its observed result and precision are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods determine how an outcome is summarized, which participants contribute to that summary, and how uncertainty is represented. For this study, the registry record gives a categorical primary endpoint and its clinical assessment criteria, but does not report the statistical analysis plan or the numerical response outcome. The explanations below describe general statistical considerations relevant to this design; they are not claims about analyses that were conducted.

What does a response count represent?

Objective response is a binary outcome for each participant under the stated response definition: a participant either meets the criteria for complete or partial response, or does not. The study-level response rate is a proportion formed from the number meeting the response definition and a defined denominator. Its interpretation therefore depends on both the response classification and the population included in the calculation. The registry says 33 participants were considered for response and 28 were evaluable, but does not report the response count or clarify the denominator used for a response-rate calculation.

RECIST provides operational rules for assigning response categories from imaging. Such rules help make assessments more reproducible by specifying how target lesions, non-target lesions, and new lesions contribute to response or progression. The record names MRI and/or CT as assessment methods and describes the criteria, but does not report details such as independent review, assessor blinding, or the timing and handling of individual assessments. Those details are not reported in the registry record.

Why does the single-group design matter?

In a single-group study, the observed outcome can be described for participants receiving the study treatment. There is no concurrent randomized group to provide a direct estimate of what would have occurred under a different treatment or without the study treatment. A comparison with historical experience, if one were made, would require assumptions about whether populations, care, eligibility, assessment, and follow-up were sufficiently comparable. Such a comparison is not reported in the registry record.

Randomization in a controlled trial is generally used to reduce systematic differences between groups, while masking can reduce some forms of bias in treatment delivery or outcome assessment. This record lists allocation as not applicable, identifies a single-group model, and lists no masking. These features do not invalidate a descriptive account of outcomes, but they limit the kinds of causal comparisons the design can support.

How should uncertainty be understood?

A confidence interval is a statistical range intended to convey the uncertainty around an estimate under a specified method and assumptions. For a response proportion, its width depends on the number of observations and the number of responses, among other methodological choices. A narrow interval indicates greater statistical precision than a wide interval, but precision is not the same as freedom from bias. The registry record does not report a response estimate or confidence interval, so neither the level nor the uncertainty of the primary result can be assessed from that record.

A p-value, when used, measures how incompatible observed data are with a specified null hypothesis under the statistical model. It does not measure the size or clinical importance of an effect. No primary-endpoint p-value or null hypothesis is reported in the registry information. In a single-group study, interpretation of a p-value would also depend on the benchmark or hypothesis chosen; the record does not report such a benchmark.

What does the planning information mean?

The planning assumptions describe an illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The illustration is not the trial’s own design: the actual study is recorded as single-group. The stated fraction of the observed effect describes a planning scenario rather than a new observed result. Sample-size illustrations are planning aids, not observed power, and their usefulness depends on assumptions about the effect, outcome variability, significance level, allocation, and analysis method.

The registry information does not include the effect estimate needed to interpret such an illustration, nor does it report resulting sample-size values. It also does not report that the trial was planned as a two-arm study. The illustration should therefore not be read as the study’s sample-size justification or as evidence about the certainty of its results. A design calculation cannot replace an observed endpoint estimate and its uncertainty.

Why can over-dispersion matter?

Over-dispersion is a general issue for count outcomes when variability exceeds what a chosen probability model expects. If a count model assumes less variation than the data contain, standard errors can be too small and uncertainty can be understated. The primary endpoint in this study is defined as participants with an event, a binary count, rather than a rate of repeated events. The registry record does not report a model or an over-dispersion assessment. This general concern should not be taken to mean that over-dispersion was present or that a particular model was used.

For safety outcomes, one participant may experience more than one adverse effect, and categories may overlap. Counts of affected participants or listed terms therefore need not add to a total of unique participants. The record reports safety counts and selected terms, but does not give enough information to reconstruct participant-level event patterns or the methods used to summarize them.

In short, the registry describes a measurable endpoint and a single-group design, but does not report the analysis details needed to evaluate statistical uncertainty or to reproduce the primary result. The methodological concepts above explain what would ordinarily be considered; they do not fill the reporting gaps.

10. Multiplicity, Missing Data and Analysis Populations

The study lists several outcomes: objective response rate as the primary outcome, along with prolonged stable disease, overall survival, adverse effects assessed by CTCAE Version 3.0, and time to progression as secondary outcomes. When a study evaluates multiple outcomes or makes multiple statistical comparisons, the chance of at least one apparently noteworthy result can increase. Statistical plans may address this through a prespecified hierarchy or other multiplicity approach. The registry record does not report a multiplicity strategy, a testing hierarchy, or a statistical analysis plan. It also does not report inferential results for the secondary outcomes.

Missing outcome information is a separate concern from missing participants. The record says 33 participants were considered for response and 28 were evaluable, but does not explain the difference. It does not report reasons for non-evaluability, missing imaging assessments, withdrawals, or how missing assessments were handled. It is therefore not possible to determine whether participants without evaluable response data were excluded, assigned a particular response category, or handled through another method.

The record does not describe an intention-to-treat analysis population, a per-protocol population, or another analysis-population rule. The enrollment listing of 34 does not establish which participants were included in any particular outcome analysis. Although the primary outcome description identifies the response-evaluable count, it does not provide a complete accounting of the analysis population. These limitations matter because changing the denominator or handling of missing outcomes can affect a response proportion.

These omissions do not show that multiplicity or missing data were mishandled; they mean that the relevant procedures are not reported in the registry record. The available information does not support a determination of how robust the outcome analyses were to these issues.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: fatigue (42.4%), neutropenia (24.2%), hypertension (18.2%), anemia (9.1%), thrombocytopenia (9.1%).

The registry safety summary lists 33 participants at risk for serious adverse effects and 30 affected, as well as 33 at risk for other adverse effects and 30 affected. It records 0 deaths. These are counts for the treatment group, not comparisons against a control group. The record also lists fatigue at 42.4%, neutropenia at 24.2%, hypertension at 18.2%, anemia at 9.1%, and thrombocytopenia at 9.1% among the top serious adverse-effect terms.

The listed percentages describe terms, not necessarily mutually exclusive groups of participants. One participant can experience more than one adverse effect, so the listed terms should not be added together or interpreted as separate, non-overlapping portions of the group. The registry summary does not provide event-level detail, severity grades for each term, duration, timing, or information about attribution to treatment. The outcome list says adverse effects were assessed using CTCAE Version 3.0, but the safety summary does not explain the full analysis or reporting procedure.

The figures indicate what the registry reports for this treated group; without a comparator, they do not show how these outcomes differ from those under another treatment or no treatment. They also cannot by themselves characterize all aspects of safety or establish a causal relationship for each event. Safety counts should be considered alongside the incompletely reported primary response outcome and the limits of the single-group design.

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

The registry record describes a phase 2, single-group evaluation of sunitinib malate in recurrent or metastatic endometrial cancer. It specifies a treatment schedule, an objective response endpoint, and RECIST v1.0 criteria for classifying response. It also gives some safety counts and selected serious adverse-effect terms. These details help define what the study set out to observe and how its primary endpoint was intended to be classified.

The record does not report the objective response count or rate. It therefore does not show how many participants had complete or partial response, nor how precisely a response estimate was measured. The primary outcome description states that 33 participants were considered and 28 were evaluable, but does not explain the difference. Without the response result, the study’s main endpoint cannot be summarized as an observed numerical finding from the registry information.

Even if a response proportion were available, this design would describe outcomes in the treated group rather than provide a randomized treatment comparison. A single-group study cannot directly establish what would have happened to comparable participants under a different treatment or no treatment. Other influences, including participant selection and outcome assessment, can shape observed outcomes. The record does not report a control group or a comparator-based analysis.

The posted safety counts likewise describe observations in the treated group. They do not provide a controlled estimate of excess risk, and the selected adverse-effect terms may overlap. Their meaning is limited by the lack of detailed event reporting in the registry record.

Accordingly, the study record is informative about design, outcome definitions, and selected safety summaries, but it does not provide enough primary outcome information to judge the observed response result or its uncertainty. It should not be read as proof for or against treatment benefit. The absence of a reported result in the registry record is a reporting limitation, not evidence that no response occurred.

13. Why This Trial Matters Statistically

This study illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry gives a detailed definition of objective response using RECIST v1.0, including imaging modalities and rules for complete response, partial response, stable disease, and progressive disease. Those criteria clarify what investigators intended to count. But the response count and response rate are not reported, so a reader cannot evaluate the central outcome from the registry information alone.

It also highlights the importance of transparent analysis populations. The record lists enrollment as 34, says 33 participants were considered for response, and identifies 28 as evaluable. Those figures cannot be treated as interchangeable. Reporting the reasons for exclusions or missing assessments, along with the population used for the endpoint, would help readers understand the denominator and assess potential bias.

The single-group design is another important statistical feature. Such a study can provide descriptive evidence about outcomes among treated participants, but lacks a concurrent randomized comparator. This distinction is essential: a response observation is not the same as an estimate of a difference caused by treatment. Statistical interpretation must account for the design before considering any numerical result.

Finally, the record shows that safety summaries and effectiveness outcomes have different reporting needs. Selected adverse-effect counts do not replace the primary endpoint, and no single summary can convey all dimensions of benefit, harm, and uncertainty. The missing response result, absent analysis details, and unexplained evaluability figures limit how much can be concluded from this registry entry. These issues demonstrate why complete outcome reporting is central to meaningful statistical 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.

Several limitations specific to this record constrain interpretation. First, the primary endpoint is defined but its numerical result is not reported. The registry record does not give the objective response count, response rate, or an interval describing uncertainty. This prevents assessment of the magnitude and precision of the main outcome.

Second, the response population is not fully explained. The record lists enrollment as 34, says 33 participants were considered for the primary outcome, and identifies 28 as evaluable. It does not state why these populations differ, how non-evaluable participants were handled, or which denominator was used for any response analysis. The reasons for missing or unusable outcome data are not reported in the registry record.

Third, the design is single-group, with no masking and no concurrent comparator. The observed outcomes cannot be interpreted as a randomized contrast, and potential influences apart from treatment cannot be separated through a control-group comparison. The record does not describe a historical benchmark or other comparator analysis.

Fourth, the registry information does not report the statistical analysis plan, any multiplicity approach, a sample-size justification, or details of interim analyses. It does not report primary-outcome inferential analyses or confidence intervals. The planning assumptions described for an illustration do not establish the study’s own design rationale or supply the missing outcome result.

Finally, the safety summary is limited to selected counts and listed terms. It does not provide detailed event-level descriptions, timing, duration, or full information about severity and attribution. These gaps do not prove that the study was conducted or analyzed incorrectly, but they limit independent appraisal of its results.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Sunitinib Malate in Treating Patients With Recurrent or Metastatic Endometrial Cancer — NCT00478426.
• Registry record last updated 2020-02-17; status last verified 2020-02.
• Results first posted 2020-02-17.
• 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 a completed phase 2, single-group study of sunitinib malate for recurrent or metastatic endometrial cancer. Treatment was given by mouth once daily on days 1–28, with courses repeating every 6 weeks in the absence of progression or unacceptable toxicity. The primary endpoint was objective response rate under RECIST v1.0, assessed by MRI and/or CT, with a timeframe of up to 7 years.

The record lists enrollment as 34 and states that 33 participants were considered for response, of whom 28 were evaluable. It does not report the primary response count or rate, or explain the difference between these populations. There was no concurrent comparator group. Safety information includes 30 participants with serious adverse effects among 33 at risk, 30 with other adverse effects among 33 at risk, and 0 deaths, together with selected serious adverse-effect terms. The registry record does not report a statistical analysis plan or the numerical primary outcome result.