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 Phase II Capecitabine, Oxaliplatin & Bevacizumab for Metastatic / Unresectable Neuroendocrine Tumors (NCT00398320), a na phase 2 study. The primary outcome is 12-month Progression Free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00398320 |
| Phase | Phase 2 |
| Condition | Neuroendocrine Tumors |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 40 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pamela L. Kunz |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns participants with metastatic or unresectable neuroendocrine tumors. The registry record lists neuroendocrine tumors as the condition, but does not report further eligibility details or a more specific description of the analyzed population.
Intervention
The treatment regimen combines capecitabine, oxaliplatin and bevacizumab. The registry record describes a single-group study and lists these drugs as the interventions. It does not report dose or schedule details in the record summarized for this analysis.
Comparator
There is no concurrent comparator group identified. The allocation is listed as not applicable, and the model is single group. Consequently, the study does not provide a randomized, like-for-like contrast against another treatment or control condition.
Primary question
The primary endpoint asks what percentage of participants had progression-free survival at 12 months. PFS is defined as time from enrollment until documented disease progression or death, whichever occurred first. The endpoint result itself is not reported in the registry record.
3. Trial Design
The registry record describes a completed Phase II, single-group study of capecitabine, oxaliplatin and bevacizumab for metastatic or unresectable neuroendocrine tumors. Enrollment is recorded as 40. The study began in 2006-11, and primary completion is recorded as 2012-10. The record identifies Pamela L. Kunz as sponsor.
Allocation is listed as not applicable, masking as none, and the model as single group. These features mean the study is not a randomized comparison. Its design can describe outcomes among participants treated in the study, but it cannot by itself isolate the effect of the regimen from other influences or establish how outcomes compare with a concurrent control group.
The registry’s brief summary frames the study as an investigation of the regimen in the setting of metastatic neuroendocrine tumors. The treatment combination is listed as the intervention. No dose, schedule, treatment duration, or detailed eligibility criteria are reported in the registry record.
The primary outcome is 12-month progression-free survival, assessed every 3 months through 12 months. Secondary outcomes listed are response rates, overall survival and biochemical markers. The record lists 5 outcome measures, but the endpoint result and analysis details are not reported in the registry record. The record also lists zero analyses.
Planning assumptions associated with the record describe a two-arm illustration with equal allocation, a two-sided significance level of 0.05 and a normal approximation. Those assumptions do not describe the actual single-group study design. The observed endpoint effect needed to interpret such an illustration is 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 | Capecitabine / Oxaliplatin / Bevacizumab |
|---|---|
| STARTED | 40 |
| COMPLETED | 34 |
| NOT COMPLETED | 6 |
Reasons for not completing this period:
| Reason | Capecitabine / Oxaliplatin / Bevacizumab |
|---|---|
| Death | 1 |
| Adverse Event | 4 |
| Physician Decision | 1 |
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 | Capecitabine / Oxaliplatin / Bevacizumab |
|---|---|---|
| Participants analysed | 40 | |
| Age, Continuous | years | 55 (32 to 76) |
| Gender: Female | Participants | 18 |
| Gender: Male | Participants | 22 |
| Region of Enrollment: United States | participants | 40 |
6. Primary Endpoint
The registry lists the primary outcome as 12-month Progression Free Survival (PFS), measured over PFS assessed every 3 months through 12 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants w/ 12 mo PFS |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the percentage of participants with progression-free survival at 12 months. The registry defines PFS as the time from enrollment until documented disease progression or death, whichever occurs first, and says PFS was assessed every 3 months through 12 months. This is a time-to-event outcome expressed at a particular follow-up point: it describes the proportion who have not yet had a documented progression event or death by that point, subject to the trial’s endpoint and analysis definitions.
The registry describes disease response using RECIST criteria, version 1. Complete response means disappearance of all target lesions; partial response means a decrease in the sum of longest diameters; progressive disease means an increase in that sum; and stable disease falls between the stated response and progression thresholds. These definitions help classify tumor status, while PFS also includes death as an event.
As a general statistical matter, a fixed-time PFS percentage can be clinically interpretable because it summarizes event-free status at a defined follow-up point. Its precision depends on the amount and follow-up of observed data and on how incomplete follow-up is handled. The registry record does not report the 12-month percentage, its uncertainty interval, the analysis method, or the handling of censoring and missing follow-up. The endpoint’s numerical result therefore cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the question the study set out to address, but it does not report the primary endpoint result. Although the primary outcome is defined as the percentage of participants with PFS at 12 months, there is no posted value for that percentage in the registry record. There is also no reported confidence interval, test statistic or p-value for that endpoint. Without an estimate and its precision, the magnitude and uncertainty of the result cannot be described.
The trial is single group, with allocation listed as not applicable and masking listed as none. Therefore, any outcome observed among participants in the study would be an outcome under the study regimen, not a randomized estimate of the regimen’s effect relative to a concurrent control. In a single-group study, interpretation is especially sensitive to the participants selected, the course of the disease, follow-up, and other factors that could influence outcomes. The registry record does not supply enough detail to evaluate those factors.
The enrollment field reports 40. That is a recorded enrollment figure, not a substitute for the number included in a particular endpoint analysis. The record does not state how many participants had evaluable follow-up for the primary endpoint or how incomplete observations were handled. It also does not report an analysis population definition. Those omissions prevent a reader from determining the denominator behind a 12-month estimate even if a percentage were available.
As general statistical guidance, a confidence interval would show the range of values compatible with the observed estimate under the method used. A narrow interval would indicate greater precision than a wide interval, though neither would remove limitations from the single-group design. No interval is reported, so precision cannot be judged. A p-value alone would not address the absence of a control group or provide the missing effect estimate; no primary-outcome p-value is reported in the registry record.
Secondary outcomes include response rates, overall survival and biochemical markers. Their names indicate additional areas of interest, but the record does not provide their results or analysis methods. It would be inappropriate to infer benefit, lack of benefit, or a comparative effect from the endpoint definition, study completion status or adverse-event counts. The posted registry information supports a description of design and outcome definitions, not a quantitative conclusion about efficacy.
9. Statistical Methods Explained
What does a single-group study estimate?
The design is listed as single group, with allocation not applicable and no masking. In general, a single-group study estimates outcomes observed among participants who received the study regimen. Without a concurrent comparator, it does not directly estimate the difference between outcomes under the regimen and outcomes under another treatment or control condition. Differences between the observed outcome and an external expectation may reflect treatment as well as participant characteristics, disease course, follow-up, or other influences. Those concerns are about design and interpretation, not proof that any particular bias occurred in this trial.
The registry record labels the study Phase II and records enrollment as 40. It does not report a protocol-based sample-size justification or a statistical analysis plan. The planning assumptions associated with the record describe a two-arm illustration, equal allocation, a two-sided significance level of 0.05 and a normal approximation. They should not be read as the design of this single-group study, nor as evidence that an analysis using those assumptions was performed.
How should PFS be understood?
The primary outcome is the percentage of participants with PFS at 12 months. PFS is defined as time from enrollment to documented disease progression or death, whichever happens first. The record says PFS was assessed every 3 months through 12 months. In general, time-to-event outcomes retain information about when an event occurs, whereas a fixed-time percentage focuses on status by a specified follow-up point. The registry does not state how the time-to-event information was converted into the reported percentage, and it does not provide the percentage itself.
In general, participants whose event status is not observed through the follow-up point may be censored rather than treated as if they remained event-free. The analysis method needs to account for such incomplete follow-up. Different approaches can make different assumptions about censoring and the timing of assessment. The registry record does not report the censoring rules, the method used to estimate the 12-month PFS percentage, or how missing follow-up was handled. No particular method, such as a Kaplan–Meier estimate, should be attributed to the study on the basis of the available record.
What do response definitions add?
The outcome description refers to RECIST criteria, version 1, and defines complete response, partial response, progressive disease and stable disease by changes in target lesions. These categories standardize how tumor response or progression is classified. In general, standardized response definitions can help make assessment more consistent, but the record does not give details of assessment review, assessor masking, or adjudication. Masking is listed as none for the study. The endpoint description does not report how disagreements or unevaluable assessments were handled.
What can precision statistics tell readers?
In general, an estimate describes the observed quantity, while a confidence interval communicates uncertainty around that estimate under a statistical model. A p-value evaluates compatibility between data and a specified null hypothesis under assumptions; it does not measure the size or clinical importance of an effect. Neither statistic replaces an effect estimate, and neither resolves the lack of a concurrent comparator in a single-group design. The registry record does not report a confidence interval or p-value for the primary outcome, and it does not report a primary endpoint estimate.
The planning assumptions specify a two-sided significance level of 0.05. This value belongs to the stated planning illustration; the record does not establish that it was the trial’s own inferential threshold. The assumptions also refer to an effect expressed as a fraction of an observed effect. Because the primary outcome result is not reported, the observed effect needed to interpret such an illustration is unavailable in the registry record. A planning illustration is not observed power and does not show that a study achieved a particular degree of power.
How should the listed outcomes be considered?
The record lists 5 outcome measures and names response rates, overall survival and biochemical markers as secondary outcomes. Listing outcomes does not establish that results were reported, that all outcomes were analyzed, or that a particular statistical procedure was used. The record lists zero analyses. It does not report which comparisons, if any, were planned for secondary outcomes, how continuous or categorical measures were summarized, or whether time-to-event methods were used for overall survival.
As a general matter, several outcome measures can create multiplicity: the more inferential claims considered, the greater the opportunity for chance findings if each is evaluated separately. Methods to address this depend on the inferential goals and analysis plan. The registry record does not report a multiplicity strategy. It also does not report missing-data methods, analysis populations, or a detailed statistical analysis plan. These gaps limit reproducibility and prevent a complete assessment of how the endpoint definitions were translated into statistical results.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 5 outcome measures, with response rates, overall survival and biochemical markers named as secondary outcomes. It lists zero analyses. These entries describe the scope of registered outcomes, but they do not show whether every outcome was analyzed, which outcomes were emphasized, or what statistical procedures were used. The primary endpoint result is not reported.
Multiplicity arises in general when multiple outcomes or multiple comparisons are examined. If each is assessed separately, the chance of finding an apparently notable result by chance can increase. Whether and how to adjust depends on the trial’s inferential aims and prespecified analysis strategy. The registry record does not report a multiplicity adjustment, a hierarchy of outcomes, or a statistical analysis plan. It is therefore not possible to determine how the family of outcome questions was handled.
The record does not report missing-data methods, censoring rules, or an analysis population definition. The primary outcome is assessed through 12 months, but no details are given about participants without complete follow-up, unevaluable assessments, or whether analyses used an intention-to-treat or another population. These are unreported methods, not evidence that data were missing or that a particular population was excluded.
In general, incomplete follow-up can affect time-to-event estimates if censoring is handled inappropriately or if the assumptions behind the method are not reasonable. A clear analysis plan would explain how event times, censoring, missing assessments and outcome populations were defined. Since those details and the endpoint result are not reported in the registry record, readers cannot reconstruct the primary analysis or assess its sensitivity to alternative choices.
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 |
|---|---|---|---|
| Capecitabine / Oxaliplatin / Bevacizumab | 14 | 40 | 35.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 3% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Capecitabine / Oxaliplatin / Bevacizumab | 20 of 40 |
Most frequent serious adverse event terms
| Event term | Organ system | Capecitabine / Oxaliplatin / Bevacizumab |
|---|---|---|
| Hypokalemia | Metabolism and nutrition disorders | 3 of 40 |
| Thrombosis/Embolism | Respiratory, thoracic and mediastinal disorders | 2 of 40 |
| Syncope | Nervous system disorders | 1 of 40 |
| Death | General disorders | 1 of 40 |
| Confusion | Nervous system disorders | 1 of 40 |
| Wound dehiscence | Surgical and medical procedures | 1 of 40 |
| Right tongue numbness/deviation | Nervous system disorders | 1 of 40 |
| Weight loss | General disorders | 1 of 40 |
| Anorexia | General disorders | 1 of 40 |
| Thrombocytopenia | Blood and lymphatic system disorders | 1 of 40 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Capecitabine / Oxaliplatin / Bevacizumab |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 8 of 40 |
| Hypertension | Cardiac disorders | 5 of 40 |
| Hand foot skin reaction | Skin and subcutaneous tissue disorders | 4 of 40 |
| Sensory neuropathy | Nervous system disorders | 3 of 40 |
| Low hemoglobin | Blood and lymphatic system disorders | 2 of 40 |
| Low neutrophils | Blood and lymphatic system disorders | 2 of 40 |
| Hemorrhage, GI Upper | Gastrointestinal disorders | 2 of 40 |
| Abdominal pain | Gastrointestinal disorders | 2 of 40 |
| Tinnitus | Ear and labyrinth disorders | 1 of 40 |
| Low leukocytes | Blood and lymphatic system disorders | 1 of 40 |
Serious events with the highest arm-level rate: hypokalemia (7.5%), thrombosis/embolism (5.0%), syncope (2.5%), death (2.5%), confusion (2.5%).
The adverse-event record lists 40 at risk for the capecitabine, oxaliplatin and bevacizumab group, with 14 affected by serious adverse events and 20 affected by other adverse events. These are registry counts for the study group; they are not a comparison with another treatment group. The record does not report event-level exposure time, the rules used for attribution, or whether the same participant could appear in both categories. The counts should not be added or interpreted as separate sets of participants.
The listed serious terms include hypokalemia at 7.5%, thrombosis/embolism at 5.0%, syncope at 2.5%, death at 2.5%, confusion at 2.5% and wound dehiscence at 2.5%. These percentages are reported as registry entries. They describe the listed terms in this record and do not by themselves establish causality, severity beyond the registry’s serious-event classification, duration, or clinical course.
As a general statistical matter, adverse-event frequencies are descriptive and depend on how events are collected, coded and attributed. The registry record does not provide those details or comparative safety data. The entries should therefore be read as reported safety counts and terms for this single-group study, not as estimates of relative risk or proof that a particular event was caused by a study drug.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed Phase II study of capecitabine, oxaliplatin and bevacizumab in metastatic or unresectable neuroendocrine tumors. Its primary outcome asks for the percentage with PFS at 12 months, with PFS defined as time from enrollment to documented progression or death. This identifies the clinical question and the outcome definition, but the record does not report the resulting percentage.
Without the primary estimate, readers cannot describe the magnitude of the observed PFS outcome. Without a confidence interval or another uncertainty measure, they cannot assess its precision. Without a reported analysis method, they cannot evaluate how the estimate accounted for event timing, censoring or incomplete follow-up. The record also does not provide results for the named secondary outcomes.
The study’s single-group design places an important limit on interpretation. Even if a favorable or unfavorable outcome estimate were available, a single-group result would describe participants in the study rather than provide a randomized comparison against another treatment. The registry record does not identify a concurrent control group. Therefore it cannot support a like-for-like estimate of comparative treatment effect.
Completion status and enrollment do not substitute for clinical outcome results. Nor do the listed adverse-event frequencies answer the efficacy question. They offer descriptive safety information for the recorded study group, but do not establish causality or relative safety compared with a control.
In general, a registry record can document intended outcomes and aspects of study design, while leaving important analytic details and results unavailable. Here, the definitions and design are informative, but the primary result, its precision, missing-data handling and analysis populations are not reported in the registry record. The defensible interpretation is therefore limited to what was registered: a single-group study with a defined 12-month PFS endpoint and recorded adverse-event entries. No conclusion about whether the regimen improved outcomes, failed to improve outcomes, or differed from another treatment follows from these registry entries alone.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of why design determines the kinds of conclusions an outcome can support. The registry identifies a single-group Phase II study, not a randomized comparison. A single-group estimate can describe outcomes in the enrolled study population, but it does not directly measure a contrast against concurrent control outcomes. That distinction remains important even when an endpoint is clearly defined.
The primary endpoint is a fixed-time PFS percentage, with PFS defined by progression or death. This definition makes the target outcome understandable, but an estimate is needed to interpret the observed result, and an uncertainty measure is needed to assess its precision. Neither is reported in the registry record. A well-specified endpoint without a posted result cannot answer the quantitative question the endpoint was intended to address.
The record also illustrates the difference between outcome definitions and analysis transparency. RECIST criteria, version 1, are named, and the PFS assessment schedule is stated. However, details about censoring, missing data, analysis populations, multiplicity and statistical procedures are not reported. In general, these choices can affect the estimate and how confidently it can be interpreted. Their absence makes it difficult to reproduce or critically assess the analysis.
Finally, the record’s planning assumptions should be kept distinct from the actual trial design. They describe a two-arm planning illustration, whereas the trial is recorded as single group. The primary outcome result needed to interpret an observed-effect-based illustration is not reported. This distinction helps avoid treating a planning exercise as evidence about achieved power or as a result of the clinical study.
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 main trial-specific limitation in interpreting the registry record is that the primary endpoint result is not reported. The record gives the definition and timing of 12-month PFS assessment, but not the percentage of participants meeting the endpoint. It also does not report a confidence interval or other measure of uncertainty. Consequently, neither the outcome magnitude nor its precision can be evaluated.
The study is single group, with no randomized comparator identified. This limits any causal or comparative interpretation, because outcomes cannot be contrasted with a concurrent control group. The registry record does not report an external comparator or provide information that would support an adjusted comparison. The absence of a control is a design feature, not evidence for or against a treatment effect.
Several details needed to understand the endpoint analysis are not reported in the registry record: the analysis population, the number with evaluable follow-up, the handling of censoring and missing data, and the statistical method used to obtain the fixed-time PFS percentage. Although the endpoint description specifies assessments every 3 months through 12 months, it does not explain how incomplete or unevaluable assessments were treated.
The registry names secondary outcomes and lists 5 outcome measures, but does not report their results, analysis methods or any strategy for multiplicity. It also lists zero analyses. No protocol-based sample-size justification or detailed statistical analysis plan is reported. The planning assumptions associated with the record describe a two-arm illustration, which does not match the registered single-group model; the observed endpoint effect needed to interpret that illustration is unavailable.
Safety interpretation is also limited to descriptive registry entries. The record does not report event attribution rules, exposure time, event duration or comparative safety data. The listed serious-event terms should therefore not be treated as causal estimates or as a comparison with another regimen.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Capecitabine, Oxaliplatin & Bevacizumab for Metastatic / Unresectable Neuroendocrine Tumors — NCT00398320.
- Registry record last updated 2017-03-01; status last verified 2017-01.
- Results first posted 2013-12-10.
- 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
The registry record describes a completed Phase II, single-group study of capecitabine, oxaliplatin and bevacizumab for metastatic or unresectable neuroendocrine tumors. Enrollment is recorded as 40. The study began in 2006-11, with primary completion in 2012-10. Allocation is listed as not applicable and masking as none.
The primary endpoint is the percentage of participants with PFS at 12 months, assessed every 3 months through 12 months. PFS is defined as time from enrollment to documented progression or death, whichever occurred first. Secondary outcomes include response rates, overall survival and biochemical markers. The record lists 5 outcome measures and zero analyses.
The primary endpoint result, its precision, analysis populations, missing-data methods and statistical analysis plan are not reported in the registry record. Adverse-event entries include 14 affected by serious events and 20 affected by other events among the 40 at risk. These are descriptive entries from a single-group study, not comparative estimates.