Temsirolimus in Treating Patients With Metastatic or Locally Advanced Recurrent Endometrial Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Temsirolimus in Treating Patients With Metastatic or Locally Advanced Recurrent Endometrial Cancer (NCT00072176), a na phase 2 study. The primary outcome is Objective Clinical Response Rate.

2. Clinical Question

3. Trial Design

This completed study is registered as a phase II, single-group trial of temsirolimus for patients with metastatic or locally advanced recurrent endometrial cancer. Enrollment was 62. Allocation is listed as not applicable, and masking is listed as none. Those design details matter for interpretation: the registry does not describe randomized treatment assignment or a concurrent control group for comparing outcomes.

The treatment description specifies intravenous temsirolimus over 30 minutes on days 1, 8, 15, and 22, with courses repeating every 28 days when disease progression or unacceptable toxicity was absent. Laboratory biomarker analysis is also listed. The registry gives no additional biomarker-analysis details, such as which biomarkers were measured or how they were related to the clinical endpoint.

The primary outcome was objective clinical response rate among patients evaluable for response. Its timeframe was up to 5 years. The definition required a partial response, based on a 30% decrease in the sum of the longest diameters of target lesions maintained for at least 4 weeks, or a complete disappearance of disease and cancer-related symptoms, confirmed on independent radiology review.

The record lists a start date of 2004-05, primary completion of 2009-05, and a completed status. These dates describe the registered study timeline; they do not supply response follow-up for each participant or explain how outcome assessments were scheduled. The protocol’s planned analysis methods, sample-size justification, interim analyses, and handling of protocol deviations are not reported in the registry record. The record lists two outcome measures, but provides no primary response result or analysis output. Consequently, the design and endpoint can be described, while the trial’s estimated response rate cannot be assessed from the posted registry results.

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: Patients who were 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: All patients received treatment

6. Primary Endpoint

The registry lists the primary outcome as Objective Clinical Response Rate, measured over Up to 5 years.

The primary endpoint was the objective clinical response rate, expressed as the percentage of patients evaluable for response who met the response definition. It combined two categories. Partial response required a 30% decrease in the sum of the longest diameters of target lesions that was maintained for at least 4 weeks. Complete response required disappearance of disease and cancer-related symptoms, with confirmation on independent radiology review.

A response endpoint is useful in a phase II study because it records whether a participant met a prespecified tumor-response criterion during the stated assessment period. It gives a proportion rather than a time-to-event measure: each evaluable participant is classified according to whether the response definition was met. In general, a confidence interval around that proportion communicates uncertainty in the estimated response frequency; it does not show the range of individual outcomes or establish a causal treatment effect.

The registry specifies a 95% confidence interval and a timeframe of up to 5 years. However, the observed percentage, the interval limits, and the number of participants evaluable for response are not reported in the registry record. Thus, the endpoint’s definition is available, but its observed value and statistical precision cannot be described from the posted results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a binary percentage outcome: whether an evaluable participant met the objective response definition. In general, a binary outcome assigns each participant to one of two categories for the analysis, such as response or no response. The response rate is the proportion classified in the response category. The registry specifies a 95% confidence interval for this measure, but does not report the estimate or interval limits.

What does a confidence interval communicate?

In general, a confidence interval is a range of values calculated from sample data to represent uncertainty about an underlying quantity. For a response percentage, the interval indicates how uncertain the estimated proportion is, given the observed responses and the statistical method used. It is not a range that contains a specified share of individual patients’ outcomes, and it does not describe how much a patient’s tumor measurement changed. The registry’s stated confidence level is 95%; the construction method is not reported in the registry record. Without the interval itself, its precision cannot be evaluated.

Why does the analysis population matter?

The primary outcome population is described as patients evaluable for response. This is not automatically the same as everyone enrolled. In general, an analysis population determines which participants contribute outcome information, and the rule for inclusion can affect the estimate. The registry does not state how many of the 62 enrolled participants were evaluable or give criteria for evaluability beyond naming that population. It also does not explain how participants without an assessable response outcome were handled. The reported response percentage therefore cannot be reconstructed or checked from the registry information.

What does the single-group design allow?

The trial is registered as single-group, with allocation not applicable and no masking. In general, a single-group study can describe outcomes observed among its participants, but it does not estimate a treatment-versus-control contrast through random allocation. A response proportion is therefore descriptive of the analyzed study population. It cannot, by itself, distinguish a treatment-associated outcome from other explanations for observed change or establish what would have occurred under another treatment. The record does not identify a control group for the primary endpoint.

What can be learned from the response definition?

The definition classifies partial response using a 30% decrease in the sum of the longest diameters of target lesions, maintained for at least 4 weeks. Complete response requires disappearance of disease and cancer-related symptoms and confirmation on independent radiology review. In general, operational criteria make an endpoint more reproducible by specifying what counts as an event. They do not eliminate uncertainty from measurement, missing assessments, or differences in how a criterion is applied. The record does not report further assessment procedures or the statistical analysis used to turn participant classifications into the final percentage.

Why are planning illustrations not trial results?

Planning illustrations based on an observed effect are calculations about hypothetical study size under stated assumptions. They are not observed outcomes, and they do not establish that a study had a particular probability of finding a result. The registry record does not report the observed primary response effect needed to assess any such illustration. In general, planning calculations depend on assumptions such as the target effect, significance level, allocation, and statistical approximation; results can change when those assumptions change. The described planning assumptions should not be interpreted as the trial’s own design justification. That justification is not reported in the registry record.

The record lists no analysis outputs for the outcome measures. It does not report a test statistic, p-value, adjusted estimate, model specification, or interim analysis. Those omissions limit any evaluation of how the primary outcome was analyzed. General statistical concepts can explain what such quantities would mean, but they cannot substitute for the missing trial estimates or analysis details.

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("Group A", "Group B")
n      <- c(29, 25)
events <- c(4, 1)
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  Group A  29  4  13.8  3.9  31.7  5.5  30.6  5.6  30.7
ARM  Group B  25  1  4.0  0.1  20.4  0.7  19.5  0.9  19.6

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures, but the primary response result and any analysis output are not reported in the registry record. The record does not describe a multiplicity plan: it does not say whether multiple endpoints or analyses were formally tested, whether any adjustment was planned, or how the primary endpoint was prioritized in statistical testing. The number and nature of planned statistical comparisons are not reported in the registry record.

In general, multiplicity arises when multiple hypotheses or outcomes are examined. Repeated testing can increase the chance of finding at least one apparently notable result by chance, so a prespecified strategy can help explain how a set of results should be interpreted. That general concern cannot be tied to a particular adjustment or testing burden in this trial, because its statistical plan and analysis results are not reported.

The primary outcome population is described as patients evaluable for response. The number evaluable, the criteria for evaluability, and the handling of participants without a response assessment are not reported in the registry record. Nor does the record state whether missing response assessments were excluded, classified in a particular way, or addressed through another method. In general, missing outcome information can affect both the analyzed population and the estimated response proportion, especially if the likelihood of missingness is related to participant outcomes.

The registry does not report an intention-to-treat analysis population or another population definition beyond patients evaluable for response. It also does not report sensitivity analyses assessing alternative missing-data assumptions. As a result, the robustness of the primary response estimate to participant exclusions or incomplete assessments cannot be evaluated from the posted registry information.

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: hospitalization for dehydration (7.4%), hospitalization for hypokalemia (7.4%), hospitalization for vomiting (7.4%), hospitalization for diarrhea (7.4%), hospitalization for spinal cord compression (3.7%).

The registry reports serious adverse-event summaries under Group A and Group B. Group A lists 33 participants at risk and 11 affected for serious events; Group B lists 27 at risk and 9 affected. For other events, the record lists 33 at risk and 33 affected in Group A, and 27 at risk and 27 affected in Group B. The meaning of the group labels and the precise relationship of these summaries to the treatment assignment are not reported in the registry record. They should not be treated as a randomized comparison.

The listed serious-event terms include hospitalization for dehydration, hypokalemia, vomiting, and diarrhea, each reported as 7.4%, as well as hospitalization for spinal cord compression and pneumonitis, each reported as 3.7%. These are the percentages attached to the named terms in the registry. The record does not provide further detail on event timing, recurrence, overlap among event categories, or attribution to temsirolimus. The term-level percentages should therefore be read as descriptive safety reporting, not as proof of cause or as a complete account of all harms.

In general, an adverse-event percentage is most interpretable when its denominator, event definition, observation period, and grouping are clear. The registry gives at-risk and affected counts for the two group summaries, but does not explain the group labels or provide those additional details for each listed term. The summaries cannot establish a comparative treatment risk in the absence of a documented like-for-like control group.

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

The registry record documents a phase II, single-group study of temsirolimus in metastatic or locally advanced recurrent endometrial cancer. It specifies an objective clinical response endpoint, a response definition, and a stated timeframe of up to 5 years. These details make the question and the intended measure understandable: the study sought to describe the proportion of patients evaluable for response who met prespecified partial- or complete-response criteria.

However, the primary endpoint result is not reported in the registry record. The response percentage, confidence interval, number evaluable, and number meeting the endpoint are absent. The record therefore does not support a conclusion about the observed response frequency or how precisely it was estimated. The existence of a defined outcome is not the same as a reported outcome result.

The study design also sets limits on what can be inferred. A single-group study has no randomized control comparison in the registry. An observed response proportion, if available, would describe the study population; it would not alone establish the difference attributable to temsirolimus compared with another treatment or no treatment. This distinction is important when describing what a trial can answer, rather than treating a within-group outcome as a comparative effect.

The serious adverse-event summaries provide selected event counts and named terms, but the registry does not explain the Group A and Group B labels or report enough information to establish a controlled safety contrast. The listed percentages describe registry entries; they do not demonstrate that temsirolimus caused an event, and they do not establish the absence of other adverse events.

Overall, the posted record supports a description of the design, treatment schedule, endpoint definition, and selected safety summaries. It does not provide the primary clinical response estimate or the statistical detail needed to assess its uncertainty, missing outcome handling, or comparative interpretation. Those boundaries should remain explicit when characterizing the study.

14. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a clearly defined endpoint and a reported result. The registry specifies objective clinical response rate, identifies the response-evaluable population, states a 95% confidence interval, and gives a timeframe of up to 5 years. Yet the response estimate and interval are not reported in the registry record. A reader can understand what the endpoint was intended to measure without being able to assess its observed value or precision.

It also highlights the interpretive limits of a single-group design. The record lists phase II, single-group, allocation not applicable, and no masking. Those features mean that the registry does not supply a randomized control contrast for the primary endpoint. In general, a single-group outcome can describe what happened among participants, but it cannot provide the same comparative evidence as a randomized treatment comparison.

The registry’s endpoint definition shows how operational details support statistical interpretation. A partial response had to meet a specified lesion-measurement threshold and persist for a minimum duration; complete response required disappearance of disease and cancer-related symptoms with independent radiology confirmation. Such criteria define classification, while the estimate’s uncertainty still depends on the observed participant outcomes and analysis population. The latter results are not reported.

The safety entries offer another useful statistical lesson: counts and percentages need context. The record gives Group A and Group B summaries and selected serious-event percentages, but does not explain the group labels or show that the groups form a comparable treatment contrast. Descriptive safety data should not be mistaken for a causal comparison. Together, these features underscore why design, analysis population, uncertainty, and completeness of registry results all matter when reading a clinical trial.

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 most consequential limitation of the registry information is that it does not report the primary response result. Although the endpoint and its 95% confidence interval are specified, the response percentage and interval limits are not reported in the registry record. The number evaluable for response and the number meeting the response definition are also not reported. This prevents assessment of the outcome’s magnitude, precision, and basic reproducibility from the registry entry.

The trial is recorded as single-group, with allocation not applicable and no masking. There is no randomized control group in the registry record against which to interpret the primary response proportion. The record therefore does not support a randomized estimate of a comparative treatment effect. The labels Group A and Group B appear in the safety summary, but their meaning and their relation to treatment assignment are not reported in the registry record.

The primary analysis population is patients evaluable for response, but the registry does not give the evaluability criteria or the population count. It does not state how missing or unassessable response outcomes were handled, whether sensitivity analyses were conducted, or whether a different analysis population was used. These gaps make it impossible to assess the potential influence of incomplete outcome data.

Statistical procedures are also sparsely documented. The registry does not report the method used to construct the confidence interval, a test or model for the primary endpoint, an interim-analysis plan, or a multiplicity strategy. A sample-size justification is not reported in the registry record. The listed serious-event percentages and group counts are descriptive entries, with no further information on timing, overlap, or attribution. These limitations concern what the registry record makes available; they do not establish what may or may not have been documented elsewhere.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Temsirolimus in Treating Patients With Metastatic or Locally Advanced Recurrent Endometrial Cancer — NCT00072176.
• Registry record last updated 2015-02-23; status last verified 2014-03.
• Results first posted 2015-02-23.
• Data retrieved from ClinicalTrials.gov on 2026-11-09.
• Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.

19. Record Summary

The registry identifies NCT00072176 as a completed phase II, single-group study of temsirolimus in metastatic or locally advanced recurrent endometrial cancer. Enrollment was 62. The registered treatment schedule was intravenous temsirolimus over 30 minutes on days 1, 8, 15, and 22, repeating every 28 days in the absence of progression or unacceptable toxicity. The listed primary endpoint was objective clinical response rate among patients evaluable for response, with a stated timeframe of up to 5 years and a 95% confidence interval.

The endpoint definition distinguishes partial response, based on a 30% decrease in the sum of the longest diameters of target lesions maintained for at least 4 weeks, from complete response, requiring disappearance of disease and cancer-related symptoms with independent radiology confirmation. The primary response estimate, its interval, and the number evaluable are not reported in the registry record. Serious adverse-event summaries are listed for Group A and Group B, but the group labels are not explained. The record does not provide a randomized comparison for efficacy or safety.