Sorafenib, Docetaxel, and Cisplatin in Treating Patients With Metastatic or Advanced Gastric or Gastroesophageal Junction Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Sorafenib, Docetaxel, and Cisplatin in Treating Patients With Metastatic or Advanced Gastric or Gastroesophageal Junction Cancer (NCT00253370), a na phase 2 study. The primary outcome is The Proportion of Patients With Objective Response (Complete Response or Partial Response).

2. Clinical Question

3. Trial Design

This is a phase II, single-group study sponsored by the National Cancer Institute (NCI). Its status is completed. The record lists a start of 2005-10 and primary completion of 2008-05. Enrollment was 44. Allocation is recorded as NA, and masking as none; the design does not include a randomized control arm.

The enrolled population had metastatic or advanced unresectable gastric or gastroesophageal junction adenocarcinoma. The listed treatment group received BAY 43-9006 (sorafenib), docetaxel, and cisplatin. In the regimen description, sorafenib is taken orally on days 1-21, while docetaxel and cisplatin are administered intravenously on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. The registry record names oxaliplatin as an alternative in the official study title, but the listed regimen and interventions specify cisplatin.

The primary endpoint was the proportion of patients with an objective response, defined as complete response or partial response. Response was assessed every 6 weeks until disease progression or up to 3 years. Progression-free survival and overall survival are listed as secondary outcomes. The posted record does not report their outcome estimates.

All enrolled patients reportedly started treatment and were considered eligible for inclusion in the primary analysis. There is no comparator arm against which to interpret response, survival, or adverse-event frequencies. The registry record does not report the rationale for the enrollment target, details of any interim analyses, or a fuller statistical analysis plan.

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: All enrolled patients started treatment and were considered eligible and hence were all included in the analysis.

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 44 patients started treatment and were considered eligible and hence were included in the analysis.

6. Primary Endpoint

The registry lists the primary outcome as The Proportion of Patients With Objective Response (Complete Response or Partial Response), measured over Assessed every 6 weeks until disease progression or up to 3 years.

The primary endpoint is the proportion of patients with an objective response: the number with a complete or partial response considered relative to the analyzed patient group. It combines two kinds of tumor response into one outcome. Under the registry's RECIST 1.0 definition, complete response requires disappearance of all target and non-target lesions. Partial response requires a decrease of at least 30% in the sum of the longest diameters of target lesions from baseline, together with the stated requirements concerning non-target lesions or tumor-marker levels. The registry record describes objective response as complete response plus partial response.

Response is used as a measure of tumor change during treatment. It differs from outcomes such as progression-free survival and overall survival, which concern time to an event or death. A response proportion can communicate how often the specified tumor-response criteria were met, but it does not by itself describe how long a response lasted or whether it translated into longer survival.

The endpoint was assessed every 6 weeks until disease progression or up to 3 years. The record specifies a 90% confidence interval for the response proportion. However, neither the observed proportion nor its interval is reported in the registry record. The definition and assessment schedule are available, but the primary result cannot be quantified from the posted record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The trial is described as phase II and single-group. In a single-group design, the primary outcome is measured among participants receiving the study regimen. There is no concurrent control group in this record. The study therefore describes outcomes in the enrolled group, rather than estimating a randomized difference between treatment strategies. This distinction matters because outcomes observed during treatment can have more than one explanation; a single-group result alone cannot identify the effect attributable to the regimen.

The primary outcome is binary at the participant level: a patient either meets the stated objective-response criteria or does not. The registry defines objective response as complete response or partial response under RECIST 1.0. A proportion summarizes the fraction of analyzed patients meeting those criteria. The registry record specifies that the primary endpoint is a proportion and that its dispersion measure is a 90% confidence interval. The estimate and interval themselves are not reported in the registry record.

A confidence interval expresses uncertainty around an estimate. In general, an interval gives a range of values compatible with the observed data under the method used to construct it. Its width reflects statistical precision: narrower intervals indicate greater precision, while wider intervals indicate more uncertainty. The interval does not remove design limitations, and it does not guarantee that the true value lies within its limits. Since the response estimate and interval are absent from the registry record, the precision of this trial's response estimate cannot be evaluated.

The response assessment schedule was every 6 weeks until disease progression or up to 3 years. Repeated assessment provides opportunities to determine whether lesions meet the response criteria over the specified follow-up. The registry record does not describe how results from assessments were combined for the primary analysis, how missing assessments were handled, or whether response confirmation was required. Those details should not be inferred from the schedule or endpoint definition.

The population for the primary analysis is described as all enrolled patients. The record says all enrolled patients started treatment and were considered eligible and hence were all included in the analysis. This is the stated analysis population. It does not, on its own, explain the operational rules for classifying a patient with an unavailable assessment, or how protocol deviations were handled. Those rules are not reported in the registry record.

There is one listed analysis and three outcome measures, but the record does not give enough detail to infer the statistical procedures used for each outcome. It would be inappropriate to assume a particular model, adjustment strategy, hypothesis test, or method for dealing with censoring for the secondary time-to-event outcomes. The statistical analysis plan is not reported in the registry record.

The registry lists progression-free survival and overall survival as secondary outcomes. These are time-to-event concepts: generally, such outcomes concern the time until a defined event, rather than whether an event occurred by a single assessment. In general, time-to-event analyses may account for participants whose event has not been observed by the end of their follow-up through censoring. The record does not specify the event definitions, censoring rules, analysis method, or numerical results for these outcomes, so no particular approach should be attributed to this trial.

Multiplicity arises when several endpoints, time points, or comparisons are tested. In general, making many statistical tests can increase the chance of at least one apparently notable result even when there is no underlying effect. This study lists a primary response outcome and secondary progression-free and overall survival outcomes. The record does not report a multiplicity adjustment, a hierarchy among secondary outcomes, or details about any interim analyses. It is not possible to determine from the record whether or how multiplicity was addressed.

The sample-size material describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It specifies planning for stated fractions of the observed effect. Those assumptions do not describe the design of this single-group trial, and the record explicitly characterizes the illustration as not the trial's own design. It should be read as planning context, not as observed power or evidence about the trial's planned enrollment. The registry record does not report the study's own sample-size justification.

In general, power is a design-stage probability of detecting a specified effect under stated assumptions. It is not a measure of how likely a result is true after the data have been observed. A retrospective power calculation based on an observed effect does not add information beyond the estimate and its uncertainty. Any sample-size figures framed as planning illustrations are hypothetical planning quantities, not observed trial outcomes. No numerical sample-size illustration results are reported in the registry record.

The record reports serious adverse events separately from other adverse events. These are event categories, not efficacy endpoints. In general, event frequencies depend on the definitions, observation period, and completeness of reporting. A participant count affected by an event does not indicate how many total event episodes occurred unless that is explicitly reported, and it does not alone establish causation. The posted safety summaries should therefore be read as descriptive counts and percentages for the treatment group.

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("BAY 43-9006, Docetaxel, Cisplatin")
n      <- c(44)
events <- c(0)
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  BAY 43-9006, Docetaxel, Cisplatin  44  0  0.0  0.0  8.0  -0.0  8.0  0.1  7.9

11. Multiplicity, Missing Data and Analysis Populations

The registry names one primary endpoint, the proportion of patients with objective response, and lists progression-free survival and overall survival as secondary outcomes. It does not report a statistical plan describing how these outcomes were prioritized for inference, whether any adjustment for multiple testing was used, or how the secondary outcomes were analyzed. Details of interim analyses are also not reported in the registry record.

Multiplicity is a general concern when several outcomes or statistical tests are considered: without an appropriate strategy, the chance of at least one apparently notable result can increase. This does not establish that the trial's analyses were invalid; it means the available record does not provide enough detail to assess how multiplicity was handled. The absence of reported procedures should not be replaced with an assumption that no such procedures existed.

The primary-analysis population is stated clearly at a broad level: all enrolled patients started treatment, were considered eligible, and were included in the analysis. However, the registry record does not explain how missing or unevaluable response assessments were treated, whether any outcome data were unavailable, or how protocol deviations were addressed. No missing-data method is reported.

These gaps affect interpretation in different ways. Multiplicity details would help readers understand the scope of statistical testing, while missing-data rules would clarify how the analyzed outcome was constructed. The available record defines the endpoint and named analysis population but does not report these additional analytic details or the primary response result.

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: neutrophils decreased (63.6%), leukocytes decreased (40.9%), hand-foot reaction (22.7%), dehydration (20.5%), fatigue (15.9%).

The serious adverse-event summary lists 44 participants at risk and 40 affected in the treatment group. The other adverse-event summary lists 44 at risk and 44 affected. The registry also gives percentages for the most frequent listed serious adverse-event terms: neutrophils decreased, 63.6%; leukocytes decreased, 40.9%; hand-foot reaction, 22.7%; dehydration, 20.5%; fatigue, 15.9%; and nausea, 15.9%.

These are descriptive safety figures for the treated group, not comparisons with a control group. The participant counts indicate how many people were recorded as affected in the respective summary; they should not be read as counts of total event episodes. The listed percentages are event-term frequencies as recorded, and the registry excerpt does not give further detail on severity grades, duration, or attribution for each term.

The deaths field is not reported in the registry record. That absence must not be interpreted as evidence that no deaths occurred. In a single-group trial, safety frequencies also cannot show how event rates would compare under another treatment. The summaries describe what was recorded in this group without establishing that every event was caused by the regimen.

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

The record documents a phase II investigation of sorafenib with docetaxel and cisplatin in patients with metastatic or advanced unresectable gastric or gastroesophageal junction adenocarcinoma. It specifies objective response as the primary outcome and defines response using RECIST 1.0 criteria. It also gives the assessment schedule and identifies progression-free survival and overall survival as secondary outcomes.

However, the primary response proportion and its 90% confidence interval are not reported in the registry record. Thus, the available information does not quantify how often objective response occurred or how precise the estimate was. The secondary survival results are also not reported. Without those outcome estimates, the record cannot establish the magnitude of the observed efficacy outcomes.

The design has no randomized comparator. Even if outcome estimates were available, a single-group description would not provide a randomized estimate of the difference between this regimen and another treatment. The record cannot establish comparative benefit or comparative risk. The response definition is an endpoint rule, not an outcome result.

The safety summary does report affected-participant counts and percentages for selected serious adverse-event terms. These figures describe the treatment group; they do not show how frequencies compare with another group, and they do not by themselves establish causation. The deaths field is not reported in the registry record, which is distinct from a report of zero deaths.

The enrollment of 44 and the statement that all enrolled patients started treatment and were included in the primary analysis provide context for the analysis population. They do not substitute for a response estimate, nor do they establish that every response assessment was complete. The record does not report the missing-data procedures or a full statistical analysis plan.

In short, the registry supports understanding of the trial's design, intervention, endpoint definition, and selected safety summaries. It does not provide the primary efficacy result needed to assess the observed response frequency or its precision. No treatment recommendation or conclusion about comparative effects follows from the information recorded.

14. Why This Trial Matters Statistically

This trial illustrates why design and outcome reporting must be read together. A clear endpoint definition explains what counts as an objective response, while a numerical estimate and an uncertainty interval are needed to understand how often that outcome occurred and how precisely it was estimated. The registry gives the former but does not report the latter.

Its single-group design is also important statistically. A treatment-group outcome can describe what occurred among enrolled patients, but the absence of a randomized comparator prevents a direct estimate of differences between treatment strategies. The design therefore places a limit on the kinds of conclusions the results could support, even if all outcome estimates were available.

The record also distinguishes endpoint types. Objective response is a proportion based on tumor-response criteria; progression-free survival and overall survival are listed as secondary outcomes with a time-to-event character. These measures answer different questions and require different interpretation. The registry does not report the secondary outcome estimates or the analytic details needed to evaluate them.

Finally, the safety summary shows why event frequencies need context. Counts and percentages in a single treatment group can describe what was recorded, but without a comparator they do not establish relative risk. Reporting of analysis populations, missing-data handling, and statistical procedures would further clarify how results were derived. The record supplies some of that context but leaves key efficacy and analysis details unreported.

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 posted record is that it does not report the primary response proportion or its 90% confidence interval. The endpoint is defined, but the result and its precision cannot be evaluated. The listed progression-free survival and overall survival outcomes also have no numerical results reported in the registry record.

The trial is single-group, with no randomized comparator. The observed outcomes therefore cannot be interpreted as a controlled estimate of the difference between this regimen and another treatment. The design also offers no like-for-like comparison for the listed adverse-event frequencies.

The record says all 44 enrolled patients started treatment, were considered eligible, and were included in the primary analysis. It does not report how missing or unevaluable response assessments were handled, how protocol deviations were addressed, or whether all scheduled assessments were available. These details matter when interpreting a proportion-based endpoint.

The record does not provide the statistical analysis plan, the study's own sample-size justification, or details of interim analyses. It also does not report how multiplicity was addressed across the primary and secondary outcomes. The sample-size material is explicitly a planning illustration rather than the trial's own design, and should not be used as a substitute for the missing design rationale or outcome results.

Safety reporting is also limited in the posted information. The adverse-event summary includes affected-participant counts and selected serious adverse-event percentages, but does not provide the deaths count; that value is not reported in the registry record. Further details on event grading, timing, duration, and attribution are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Sorafenib, Docetaxel, and Cisplatin in Treating Patients With Metastatic or Advanced Gastric or Gastroesophageal Junction Cancer — NCT00253370.
• Registry record last updated 2014-11-24; status last verified 2013-12.
• Results first posted 2014-11-24.
• 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

NCT00253370 is a completed phase II, single-group study sponsored by the National Cancer Institute (NCI), with enrollment of 44. It evaluated BAY 43-9006 (sorafenib) with docetaxel and cisplatin in metastatic or advanced unresectable gastric or gastroesophageal junction adenocarcinoma. The record lists no masking and no separate comparator group.

The primary endpoint was the proportion of patients with complete or partial objective response under RECIST 1.0, assessed every 6 weeks until progression or up to 3 years. Progression-free survival and overall survival were listed as secondary outcomes. All enrolled patients reportedly started treatment and were included in the primary analysis.

The primary response estimate, its 90% confidence interval, and secondary outcome results are not reported in the registry record. Safety summaries include affected-participant counts and selected serious adverse-event percentages. The deaths field is not reported in the registry record.