This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Sunitinib Malate in Treating Patients With Recurrent Ovarian Epithelial, Fallopian Tube, or Primary Peritoneal Cancer (NCT00388037), a na phase 2 study. The primary outcome is Objective Response (Partial Response or Complete Response) as Per the Response Evaluation Criteria in Solid Tumors (RECIST) Criteria.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00388037 |
| Phase | Phase 2 |
| Condition | Recurrent Fallopian Tube Cancer, Recurrent Ovarian Epithelial Cancer, Recurrent Primary Peritoneal Cavity Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 31 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. The registry record also lists stage IIIA, IIIB, IIIC, and IV disease for these cancer types. The primary endpoint population is described as all response evaluable patients, but the number of patients who were response evaluable is not reported in the registry record.
Intervention
Sunitinib malate, taken by mouth once daily on days 1-28. Treatment courses were scheduled to repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis is also listed as an intervention, but further details are not reported in the registry record.
Comparator
There is no separate comparator group. The study is described as a single-group trial with no allocation and no masking. Its treatment experience therefore cannot be read as a direct, randomized comparison against another treatment or a control group.
Primary question
The primary question was whether patients receiving sunitinib malate had an objective response, defined as a complete or partial response under RECIST criteria. The registry record states that response was to be expressed as a percentage of response evaluable patients and that a 95% confidence interval would be calculated. The actual response estimate and confidence interval are not reported in the registry record.
This phase II study focused on recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. Its stated purpose included studying side effects and how well sunitinib malate works in these patients. The registry summary gives a biological rationale: the drug may inhibit a cancer’s ability to grow blood vessels. That is the trial’s stated rationale, not evidence of a measured biological effect in this study.
The question is best understood as a treatment activity question within a single group. It asks whether objective tumor responses were observed among evaluable participants receiving sunitinib. It does not directly answer whether the response rate differed from that under another treatment, because no concurrent comparator group is described. Nor does the registry record establish how the treatment compares with other care.
The primary endpoint description includes response criteria and a planned confidence interval, but the actual result is absent. The number enrolled was 31; the number included in the response-evaluable population is not reported in the registry record. Without that population count and the response result, the primary question cannot be answered quantitatively from this record.
3. Trial Design
This was a phase II study sponsored by the National Cancer Institute (NCI). The registry lists an enrollment of 31 and describes a single-group model, with allocation recorded as NA and masking as none. The study status is completed. Its recorded start was 2007-01 and its primary completion was 2012-01.
Participants received sunitinib malate by mouth once daily on days 1-28. Courses were scheduled to repeat every 28 days unless disease progression or unacceptable toxicity occurred. The registry also lists laboratory biomarker analysis, without further information about the analyses or their role in the study. The listed treatment schedule describes the planned regimen; details about actual treatment exposure, adherence, dose changes, and treatment duration are not reported in the registry record.
The single-group design has an important interpretive consequence: all described treatment observations belong to the sunitinib group, with no randomized control group for a like-for-like comparison. Outcomes in this group can describe what was recorded during the study, but they cannot by themselves isolate a treatment effect from other explanations. The registry does not provide a comparison against a historical or external group either.
The primary endpoint was objective response, defined as partial response or complete response under RECIST criteria, assessed through up to 3 years. The endpoint population is described as all response evaluable patients. Although the record specifies a planned 95% confidence interval and mentions assessing median and range of response duration, no numerical primary response result, confidence interval, response duration summary, or analysis is reported in the registry record. Details of the protocol’s statistical plan, including any planned interim analyses or sample-size justification, are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Treatment (Sunitinib Malate) |
|---|---|
| STARTED | 31 |
| COMPLETED | 30 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Treatment (Sunitinib Malate) |
|---|---|
| Protocol Violation | 1 |
Analysis population for the primary outcome, as defined in the registry: All response evaluable patients
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 eligible patients
| Characteristic | Unit | Treatment (Sunitinib Malate) |
|---|---|---|
| Participants analysed | 30 | |
| Age, Continuous | years | 57 (37 to 82) |
| Sex: Female, Male: Female | Participants | 30 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: Canada | participants | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response (Partial Response or Complete Response) as Per the Response Evaluation Criteria in Solid Tumors (RECIST) Criteria, measured over Up to 3 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of evalubale patients |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is objective response: partial response or complete response according to RECIST criteria. The registry defines partial response as a 30% decrease in the sum of the longest diameters of target lesions, maintained for at least 4 weeks. Complete response is defined as complete disappearance of disease and cancer-related symptoms, maintained for at least 4 weeks. The intended summary is a percentage of response evaluable patients, with a 95% confidence interval.
Response is a clinically interpretable measure of tumor change during treatment. It distinguishes a specified reduction or disappearance of disease from outcomes that do not meet these response definitions. General statistical context: a confidence interval describes the uncertainty around an estimated response rate; a wider interval indicates less precision, while a narrower interval indicates greater precision, conditional on the method and data used.
The registry says the endpoint was assessed up to 3 years and that the median and range of response duration would be assessed. However, it does not report the observed response percentage, its confidence interval, the count of response evaluable participants, or the duration-of-response results. The endpoint definition therefore clarifies what the study intended to measure, but the recorded information does not establish how many participants responded or how precisely that response rate was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record gives the design and endpoint definition, but not the numerical primary endpoint outcome. It states that objective response was to include partial or complete response under RECIST criteria, and that the response percentage would be calculated among all response evaluable patients. The observed response percentage and its 95% confidence interval are not reported in the registry record. Consequently, the primary endpoint cannot be interpreted as a numerical estimate from this record.
The enrollment was 31. This is not necessarily the denominator for the primary endpoint: the record defines the endpoint population as response evaluable patients, and does not report how many of the enrolled participants met that definition or had outcome data available. It would be incorrect to treat enrollment as the response denominator without that information.
The study was single-group and unmasked, with no allocation to a separate control arm. This structure allows a descriptive account of response and safety among treated participants, if results are reported. It does not provide a randomized comparison to estimate a difference attributable to treatment. General statistical context: random assignment helps balance measured and unmeasured prognostic factors between groups on average; without a concurrent randomized comparison, observed outcomes can also reflect patient characteristics, disease course, assessment practices, and other influences.
The registry summary says the study examined side effects and how well sunitinib malate works. That statement describes its purpose, not its finding. There is no basis in the record to state that the treatment did or did not produce responses, nor to characterize the magnitude of an effect. It also does not provide a result for response duration, despite describing median and range as planned assessments.
The adverse-event record includes a treatment group with 30 participants at risk for serious adverse events and 15 affected. The record also lists other adverse events with 30 at risk and 30 affected. These are separate fields and should not be combined into an overall patient count. The listed serious-event terms and percentages describe recorded safety summaries, not a comparison with an untreated or alternative-treatment group. The death count is not reported in the registry record.
Planning illustrations based on an observed effect are not trial results. Because an observed primary effect estimate is not reported, any illustration tied to a fraction of that effect cannot be interpreted as evidence about the study’s response rate. Even when available, such planning calculations describe hypothetical sample-size requirements under stated assumptions, not observed power or an observed treatment benefit.
9. Statistical Methods Explained
The registry record identifies an objective-response endpoint and states that a 95% confidence interval for the response rate would be calculated. It does not report the numerical estimate, interval, or analysis output. The method description can therefore clarify the intended statistical quantities, but it cannot reconstruct a result that is absent from the record.
What does a response percentage represent?
A response percentage is a proportion: the number of response evaluable participants meeting the response definition relative to the number of response evaluable participants assessed. Here, a response means partial response or complete response under the stated RECIST definitions. The registry’s use of “all response evaluable patients” makes the evaluable population, rather than enrollment alone, the stated analysis population for this endpoint. The size and composition of that population are not reported in the registry record.
General statistical context: a proportion is a sample estimate of an underlying response probability in a defined population. Its interpretation depends on clear outcome criteria, complete and consistent assessment, and a clearly stated denominator. If participants without an evaluable assessment are excluded, the resulting estimate may not represent all enrolled participants. The record does not state how unevaluable participants were handled or why some participants might not have been evaluable.
What does the confidence interval add?
The endpoint description specifies a 95% confidence interval. General statistical context: a confidence interval presents a range of values compatible with the data under a statistical procedure and its assumptions. It communicates precision, which a point estimate alone does not. A confidence interval is not a guarantee that the true response probability lies within one particular calculated interval, and its width depends on the amount and pattern of information available as well as the method used.
The actual interval and the procedure used to calculate it are not reported in the registry record. The record states the intended confidence level, but it does not identify a specific interval method. It is therefore not possible to assess the interval’s width, reproduce it, or determine whether a particular method was used.
Why does the single-group design matter?
With a single-group design, the study can describe outcomes among participants receiving the study treatment. It cannot produce a randomized between-group contrast because the registry lists no separate comparator arm. General statistical context: a treatment comparison requires a defined contrast, such as a difference in outcome between groups, and uncertainty around that contrast. A within-group response percentage is not itself a comparison and cannot establish how outcomes would have differed without the treatment.
The record lists no allocation and no masking. These design features matter when reading outcomes: an unmasked study can leave assessments more susceptible to knowledge of treatment, depending on how outcomes are measured and adjudicated. The endpoint uses RECIST criteria, but procedures for blinded review or independent assessment are not reported in the registry record. No claim about whether such procedures were used is warranted.
How should the planning illustrations be read?
The stated planning assumptions describe illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They further define the effect to detect as a stated fraction of the observed effect. These are assumptions for a planning calculation, not a description of the actual single-group design. The registry record does not report the trial’s own sample-size justification or protocol assumptions.
Because no observed primary effect is reported, an illustration that depends on that effect does not provide an interpretable numeric planning result from the registry record. Such illustrations are hypothetical planning quantities, not observed power. General statistical context: power calculations depend on the assumed effect, variability or event rate, significance threshold, allocation, and analysis method. A calculated sample size is conditional on those assumptions and does not establish what the study was capable of detecting after the fact.
What is over-dispersion, and is it relevant?
General statistical context: over-dispersion occurs when observed variability in count or rate data exceeds what a chosen model assumes. If a model ignores over-dispersion, uncertainty may be understated and inferential results can be too optimistic. The registry lists a count-type outcome classification but does not report a model-based estimate or analysis. It is not reported in the registry record whether over-dispersion was assessed or whether any count model was used. The primary endpoint is described as a response percentage, and no further modeling details are available.
What can be said about response duration?
The endpoint description says median and range of duration of response would be assessed. General statistical context: duration measures depend on when response begins, how follow-up is handled, and whether some responses are still ongoing when observation ends. The record does not report a duration estimate, range, follow-up details, censoring rules, or a time-to-event analysis. No conclusion about duration can be drawn from the registry entry.
10. Multiplicity, Missing Data and Analysis Populations
The registry record identifies objective response as the primary endpoint and states that the response rate would have a 95% confidence interval. It does not report a statistical analysis plan, hypothesis-testing procedure, or adjustment for multiple outcomes. Whether multiplicity was considered in the protocol is not reported in the registry record. Although one primary outcome measure is listed, that alone does not establish what other analyses were prespecified or how they were handled.
Missing-data handling is also not described. The endpoint population is all response evaluable patients, but the registry does not report how response evaluability was determined, how many enrolled participants were evaluable, or how participants without an assessment were treated in the analysis. It does not state whether missing assessments were counted as nonresponse, excluded, imputed, or handled by another approach. Those choices can affect a response percentage, so the denominator and missing-outcome rules are needed to interpret the estimate.
The record reports enrollment of 31, but enrollment should not be substituted for the response-evaluable denominator. The number of participants who started treatment, completed treatment, discontinued, or had follow-up assessments is not reported in the registry record. Reasons for discontinuation and the amount of missing outcome information are not reported either.
The trial is described as a single group, with no allocation and no masking. The record does not describe an analysis population beyond the primary endpoint’s response-evaluable population, nor does it report whether a separate safety population was used. Interim analyses, subgroup analyses, sensitivity analyses, and the handling of protocol deviations are not reported in the registry record. These absences limit assessment of how the posted endpoint was selected and how robust an analysis might be to alternative assumptions.
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 |
|---|---|---|---|
| Treatment (Sunitinib Malate) | 15 | 30 | 50.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Treatment (Sunitinib Malate) | 30 of 30 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Sunitinib Malate) |
|---|---|---|
| Death- NOS | General disorders | 3 of 30 |
| Pain-abdominal | General disorders | 3 of 30 |
| Small Bowel Obstruction | Gastrointestinal disorders | 3 of 30 |
| Dizziness | Nervous system disorders | 2 of 30 |
| Neuropathy-Sensory | Nervous system disorders | 1 of 30 |
| Dry eye | Eye disorders | 1 of 30 |
| Cerebrovascular ischemia | Cardiac disorders | 1 of 30 |
| Hypomagnesemia | Investigations | 1 of 30 |
| Distension/Bloating and Flatulence | Gastrointestinal disorders | 1 of 30 |
| Watery eye | Eye disorders | 1 of 30 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Sunitinib Malate) |
|---|---|---|
| Fatigue | General disorders | 28 of 30 |
| Nausea | Gastrointestinal disorders | 24 of 30 |
| Pain Abdomen NOS | General disorders | 20 of 30 |
| Constipation | Gastrointestinal disorders | 19 of 30 |
| Heartburn | Gastrointestinal disorders | 18 of 30 |
| Taste alteration | Gastrointestinal disorders | 18 of 30 |
| Vomiting | Gastrointestinal disorders | 18 of 30 |
| Mucositis (clinical exam) Oral cavity | Gastrointestinal disorders | 17 of 30 |
| Diarrhea | Gastrointestinal disorders | 16 of 30 |
| Distension | Gastrointestinal disorders | 16 of 30 |
Serious events with the highest arm-level rate: death- nos (10.0%), pain-abdominal (10.0%), small bowel obstruction (10.0%), dizziness (6.7%), neuropathy-sensory (3.3%).
The adverse-event record identifies a treatment group titled Treatment (Sunitinib Malate). It reports 30 participants at risk for serious adverse events and 15 affected. For other adverse events, it reports 30 at risk and 30 affected. These are distinct summaries; the fields do not establish that the participants in each category are mutually exclusive, and the figures should not be added together.
The listed serious adverse-event terms and percentages are Death- NOS at 10.0%, Pain-abdominal at 10.0%, Small Bowel Obstruction at 10.0%, Dizziness at 6.7%, Neuropathy-Sensory at 3.3%, and Dry eye at 3.3%. These are the recorded terms and percentages; the registry record does not provide a comparison group, event timing, duration, severity breakdown beyond the serious-event listing, or a causal assessment for these terms. The death count is not reported in the registry record.
General statistical context: safety percentages are descriptive and depend on the population at risk, follow-up time, event definitions, and how repeated events are counted. The record does not provide enough detail to determine whether the percentages refer to participants with at least one event of each type or to event episodes under another counting rule. These summaries should not be interpreted as comparative risk estimates or as evidence that an event was caused by treatment.
12. What the Result Does, and Does Not, Mean
The registry entry documents a phase II, single-group study of sunitinib malate in patients with recurrent ovarian epithelial, fallopian tube, or primary peritoneal cancer. It records enrollment of 31, describes the treatment schedule, and defines objective response as the primary endpoint. It also contains adverse-event summaries for the treatment group. These elements describe the study’s scope and the kinds of outcomes it recorded.
The key missing element for interpreting the primary question is the numerical response result. The registry states that response would be summarized as a percentage among response evaluable patients, with a 95% confidence interval, but neither the percentage nor interval is reported in the registry record. The response-evaluable denominator is also absent. As a result, the record does not establish the frequency of response or the precision of that estimate.
The single-group design also limits what any within-group response result could establish, even if reported. A response percentage would describe outcomes among participants treated in this study; without a concurrent randomized comparator, it would not quantify a treatment-versus-control difference. General statistical context: observed outcomes in a single group may reflect treatment as well as participant characteristics, disease course, assessment, and other factors.
The serious adverse-event percentages provide descriptive safety information, but the record does not include comparative safety estimates or enough detail to attribute listed events to treatment. The listed serious terms should be read as recorded summaries within the treatment group, not as proof of causation. The death count is not reported in the registry record.
Therefore, the record supports a description of the study design, planned response measure, treatment schedule, enrollment, and listed adverse-event summaries. It does not support a conclusion about the magnitude or precision of the primary response result, comparative effectiveness, or comparative safety. The registry’s rationale that sunitinib may inhibit blood-vessel growth is not a reported clinical finding from this study.
13. Why This Trial Matters Statistically
This study illustrates the distinction between an endpoint definition and an endpoint result. The registry provides substantial detail about what counted as partial and complete response, names the response-evaluable population, and states that a 95% confidence interval was planned. Yet it does not report the response estimate or the number of participants evaluated. Without those pieces, readers cannot determine the observed response frequency or its precision.
It also illustrates why design context is integral to statistical interpretation. A single-group phase II study can provide descriptive evidence about outcomes among treated participants, but no randomized comparator is available in this record. A response percentage, even if reported, would not by itself quantify a treatment effect relative to another treatment or control. Statistical summaries must be interpreted in light of the contrast the design can support.
The safety summaries reinforce the need to keep denominators and event categories clear. The record distinguishes participants at risk and affected for serious adverse events from those for other adverse events, and it lists specific serious-event terms with percentages. These summaries describe recorded events within the treatment group; they do not constitute comparative safety estimates.
Finally, the record shows why planning calculations should not be mistaken for trial findings. The stated illustration assumptions depend on an observed effect, while the primary response estimate is not reported. Such calculations are hypothetical planning aids, not observed power and not evidence of a result. In clinical trial interpretation, transparent reporting of the analysis population, outcome estimate, uncertainty, and analysis rules is essential to connect a protocol question with an evidence-based answer.
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 principal limitation for statistical interpretation is that the registry record does not report the primary response estimate, its confidence interval, or the number of response evaluable participants. Enrollment of 31 cannot replace the missing endpoint denominator. The record therefore does not permit a quantitative reading of the primary outcome.
The study has a single-group design, with no allocation and no masking. No like-for-like randomized comparison is available. Any outcome reported for this group would be descriptive and would not isolate the treatment’s contribution from other potential influences. The registry does not describe a separate comparator or an external comparison.
Important analysis details are absent. The record does not report the statistical analysis plan, sample-size justification, interim analyses, the specific confidence-interval method, or handling of missing response assessments. It also does not report how response evaluability was determined, the number of participants with incomplete assessments, or the rules for protocol deviations. These omissions limit assessment of the analysis population and the sensitivity of any result to analytic choices.
The endpoint description names a maximum assessment timeframe of up to 3 years and says duration of response would be summarized, but no response-duration result or follow-up detail is reported. The registry does not provide treatment exposure, adherence, dose modifications, discontinuation reasons, or outcome-assessment procedures such as independent review. The listed safety summaries do not include a comparator, event timing, or a reported death count. Each of these gaps narrows the conclusions that can be drawn from the registry entry.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Sunitinib Malate in Treating Patients With Recurrent Ovarian Epithelial, Fallopian Tube, or Primary Peritoneal Cancer — NCT00388037.
- Registry record last updated 2015-01-27; status last verified 2014-04.
- Results first posted 2015-01-15.
- 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
NCT00388037 is a completed phase II, single-group study sponsored by the National Cancer Institute (NCI), with enrollment of 31. Its recorded start was 2007-01 and primary completion was 2012-01. Participants received sunitinib malate by mouth once daily on days 1-28, with courses repeating every 28 days unless progression or unacceptable toxicity occurred.
The primary endpoint was objective response under RECIST criteria among response evaluable patients, with a planned 95% confidence interval and assessment up to 3 years. The numerical response result, confidence interval, and response-evaluable count are not reported in the registry record. The record lists serious adverse-event summaries for the treatment group, but no comparator-based safety analysis. Because the study has no separate control group, it does not provide a randomized treatment comparison.