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 Fulvestrant in Treating Patients With Recurrent Ovarian Epithelial Cancer (NCT00617188), a na phase 2 study. The primary outcome is Patients' Overall 90-Day Clinical Response as Measured by Response Evaluation Criteria in Solid Tumors (RECIST).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00617188 |
| Phase | Phase 2 |
| Condition | Ovarian Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 26 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Masonic Cancer Center, University of Minnesota |
| Results posted | Yes |
2. Clinical Question
Population
Patients with recurrent ovarian epithelial cancer were the intended population. The registry record lists ovarian cancer as the condition and records enrollment of 26 participants. It does not report further eligibility details, such as the clinical features required for entry or the characteristics of participants who actually contributed data to the primary endpoint.
Intervention
The intervention was fulvestrant. The recorded regimen was 500 milligrams on Day 1, followed by 250 mg on Day 1, 29 and every 28 days thereafter. The record does not describe treatment adherence, dose modifications, or how many participants completed the regimen.
Comparator
There was no comparison group: the trial is recorded as single-group, with allocation marked NA. The registry record therefore does not set fulvestrant against placebo, usual care, or another treatment in this trial. It cannot directly estimate a relative treatment effect against a concurrent control.
Primary question
The primary endpoint asks how many participants had a complete response, partial response, or stable disease by Day 90, using RECIST as recorded in the registry. The registry record defines the categories but does not report the number of participants in any category or the overall clinical-response result. Thus the clinical question is identifiable, but the posted registry information does not establish how participants responded.
3. Trial Design
This was a phase II, completed, single-group trial of fulvestrant for recurrent ovarian carcinoma. The registry record lists enrollment of 26 and gives a start date of 2007-06 and a primary completion date of 2008-04. Allocation is marked NA, and masking is recorded as none. There is no concurrent control group in the design, so the study is not a randomized comparison and its results cannot be read as a head-to-head estimate against another treatment.
The intervention was fulvestrant, with the regimen recorded as 500 milligrams on Day 1; 250 mg on Day 1, 29 and every 28 days thereafter. The record does not report how treatment was implemented in practice, whether doses changed, or how long participants remained on treatment. A secondary outcome is median days to treatment termination, but its result is not reported in the registry record.
The primary outcome is overall clinical response at Day 90, defined as the sum of complete responses, partial responses, and stable disease. Other outcomes listed include quality-of-life scores and serum and urine measures, as well as treatment-termination time. The record identifies 6 outcome measures, but it does not report the findings for the primary endpoint or these secondary measures. It records 0 analyses; details of a statistical analysis plan, analysis populations, and any interim analyses are not reported in the registry record.
Enrollment describes the trial record, not necessarily the number of participants with observed endpoint data. The registry record does not report endpoint-specific denominators, missing observations, or participant flow. Those omissions limit assessment of how completely the endpoint represents the enrolled group.
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.
Before assignment: 3 additional patients were enrolled, but were never treated.
Overall Study
| Stage | Fulvestrant Treatment |
|---|---|
| STARTED | 26 |
| COMPLETED | 26 |
| NOT COMPLETED | 0 |
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 | Fulvestrant Treatment |
|---|---|---|
| Participants analysed | 26 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 14 |
| Age, Categorical: >=65 years | Participants | 12 |
| Sex: Female, Male: Female | Participants | 26 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 26 |
6. Primary Endpoint
The registry lists the primary outcome as Patients' Overall 90-Day Clinical Response as Measured by Response Evaluation Criteria in Solid Tumors (RECIST), measured over Day 90.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with an overall clinical response by Day 90. The registry defines this as the sum of complete responses, partial responses, and stable disease recorded from treatment start through Day 90. Complete response means disappearance of all lesions. Partial response is defined by a decrease of 30% or more in the sum of target lesions. Progressive disease is an increase of 20% or more in the sum of target lesions or the appearance of any new lesion. Stable disease is defined as not meeting the complete-response, partial-response, or progressive-disease criteria.
In general, response categories provide a structured way to describe change in measurable disease. Combining complete response, partial response, and stable disease creates a broader clinical-response measure than counting tumor shrinkage alone: stable disease is included even when the criteria for response are not met. The endpoint therefore answers a defined question about disease status through the specified assessment period; it is not itself a measure of survival, quality of life, or duration of disease control.
The registry record identifies the assessment time and definitions but does not report the count with each response category, the total meeting the combined endpoint, or the denominator assessed at Day 90. Without those results, neither the observed response frequency nor its precision can be described. The endpoint definition alone is not evidence that a response occurred.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record makes the study question clear but does not provide the primary endpoint result. It defines clinical response by Day 90 and describes how complete response, partial response, stable disease, and progressive disease are classified. However, it does not state how many participants fell into any category, how many had the combined response, or how many were assessed. No effect estimate, confidence interval, or significance test for the primary outcome is reported in the registry record. The endpoint therefore cannot be interpreted as evidence for or against a treatment effect.
The trial is single-group, with allocation marked NA and no masking. As a result, there is no concurrent control group against which to compare the observed clinical course. In general, an outcome observed after treatment in a single group cannot by itself separate treatment effects from other explanations, including the course of the disease or differences in participant characteristics. This is a design limitation, not a reason to infer an unreported result.
The registry lists enrollment of 26, but that figure does not reveal how many participants had evaluable Day 90 assessments. A denominator for the primary outcome and information about missing endpoint data are not reported in the registry record. Without them, the completeness of outcome ascertainment and the potential influence of missing observations cannot be judged.
Secondary outcomes include quality-of-life scores, serum skeletal-specific alkaline phosphatase concentration, urine N-telopeptide concentration, and median days to treatment termination. Their results are not reported in the registry record. The record also lists serious and other adverse-event counts, but those safety figures do not fill the gap in the efficacy endpoint and should be interpreted separately.
Any sample-size planning illustrations associated with the posted results are not observed power and do not substitute for the trial's clinical findings. The stated planning assumptions describe hypothetical two-arm calculations, while this trial itself is recorded as a single-group study. Without the primary result and its denominator, the clinical-response question remains unanswered by the registry information.
9. Statistical Methods Explained
The primary outcome is a binary participant-level measure: a participant either meets the registry's combined clinical-response definition by Day 90 or does not. The registry describes the count as the endpoint quantity, but does not report that count. For a binary outcome, a useful summary ordinarily gives the number meeting the definition and the number assessed, alongside an estimate of the response proportion. The registry record does not report those values, so no response proportion can be stated.
In general, a confidence interval describes the uncertainty around an estimate. For a response proportion, it communicates a range of values compatible with the observed data under the method used. Its width depends in part on the amount of information and the observed outcome pattern. A narrow interval would indicate greater precision than a wide interval, but neither would address whether a single-group result is attributable to treatment. No confidence interval for the primary endpoint is reported in the registry record.
The endpoint combines complete response, partial response, and stable disease. This is a composite binary definition: the component categories are distinct, but are counted together for the overall endpoint. In general, composite outcomes can be easier to summarize than separate response categories, but they can conceal variation in the kinds of outcomes that make up the total. The separate category counts would help readers understand whether the combined result reflects tumor disappearance, shrinkage, or stable disease. Those counts are not reported in the registry record.
RECIST criteria define how changes in lesions are categorized. The record specifies complete response as disappearance of all lesions, partial response as a decrease of 30% or more in the sum of target lesions, and progressive disease as an increase of 20% or more or any new lesion. Stable disease is the remaining category under the stated definitions. In general, consistent criteria can make outcome classification more reproducible. The registry does not report assessment procedures, assessor training, or whether response classifications were independently reviewed.
Because this is a single-group study, there is no between-group contrast to estimate. In a randomized controlled trial, random allocation can help make treatment groups comparable in expectation, supporting interpretation of a difference between groups. That logic does not apply to this record as a comparative analysis: allocation is NA, masking is none, and the model is single-group. An observed outcome, if reported, would describe participants in the study and would not by itself supply a controlled estimate of what would have happened under an alternative treatment.
General statistical methods for binary outcomes may include a proportion with a confidence interval, or a model if covariates or repeated measurements are part of the analysis. The registry record does not report which method was used for the primary endpoint, whether any covariate adjustment was planned, or how response data were handled when assessments were missing. The record lists 0 analyses. Thus, the analysis approach and its assumptions are not reported in the registry record; they should not be inferred from the endpoint definition alone.
Planning calculations answer a different question from trial results. A sample-size calculation can illustrate how many participants per arm might be needed under specified assumptions to detect an effect at a chosen significance level and power. Such planning values are hypothetical and are not a measure of the probability that a completed trial found a real effect. The stated planning illustration uses two-sided significance level 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a fraction of the observed effect. That two-arm framework differs from the actual single-group design. These illustrations should therefore not be read as a design description or as evidence of efficacy.
For a binary outcome, general planning calculations often rely on assumptions about the event frequency in each group and the treatment contrast to be detected. A different assumed effect can change the required sample size. If a planning illustration is based on an observed effect, its value is conditional on that effect estimate, which can itself be uncertain. Such a calculation does not correct for the absence of a control group, missing endpoint data, or an unreported statistical analysis plan. The registry record does not report the trial's own sample-size justification.
Multiplicity concerns arise when several outcomes or analyses are examined. In general, testing many outcomes can increase the chance of at least one apparently notable result by chance, depending on the testing strategy. The record identifies a primary endpoint and several secondary outcomes, but does not report a multiplicity procedure or a hierarchy for interpreting them. Since endpoint results are also not reported, there is no basis to assess how multiple findings were handled in practice.
Finally, interpretation requires a clear analysis population and outcome ascertainment. The registry record does not report whether the primary analysis included all enrolled participants, only those who received treatment, or only those with an evaluable assessment. It also does not report the handling of participants who discontinued treatment or missed Day 90 assessment. In general, these choices can affect both the estimate and its uncertainty. The record does not contain enough detail to evaluate those choices.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists the Day 90 clinical-response endpoint as primary and identifies secondary outcomes for clinical response using modified criteria, treatment-termination time, quality of life, and serum and urine measures. It records 6 outcome measures and 0 analyses. The results for the listed outcomes and the analytic procedures are not reported in the registry record.
In general, multiplicity is relevant when investigators examine multiple outcomes, time points, or statistical tests. If many hypotheses are tested, the chance of finding at least one apparently unusual result can rise unless the analysis plan accounts for that. Common approaches include identifying a primary outcome in advance, specifying a testing hierarchy, or adjusting inference across tests. Which, if any, approach was used in this trial is not reported in the registry record. The presence of a designated primary outcome does not establish how the secondary outcomes were handled.
The registry record does not report missing-data counts for the Day 90 endpoint, the number assessed, reasons for missing assessments, or a method for handling missing outcomes. It also does not report the analysis population or whether participants who stopped treatment remained in the primary analysis. In general, missingness can matter if the likelihood of an unavailable assessment is related to the participant's outcome. Without a denominator and handling rules, the potential direction or magnitude of any effect cannot be evaluated.
Details of interim analyses, sample-size justification, and a prespecified statistical analysis plan are not reported in the registry record. The registry's planning assumptions for illustrative two-arm calculations do not establish that those assumptions formed the trial's own design or analysis plan.
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 |
|---|---|---|---|
| Fulvestrant Treatment | 10 | 26 | 38.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Fulvestrant Treatment | 2 of 26 |
Most frequent serious adverse event terms
| Event term | Organ system | Fulvestrant Treatment |
|---|---|---|
| Bowel obstruction, non-specific | Gastrointestinal disorders | 3 of 26 |
| Death, non-specific | General disorders | 2 of 26 |
| Pleural effusion (non-malignant) | Respiratory, thoracic and mediastinal disorders | 2 of 26 |
| Ascites (non-malignant) | Respiratory, thoracic and mediastinal disorders | 1 of 26 |
| Failure to thrive | General disorders | 1 of 26 |
| Fracture | Musculoskeletal and connective tissue disorders | 1 of 26 |
| Nausea | Gastrointestinal disorders | 1 of 26 |
| Pain | General disorders | 1 of 26 |
| Pain, abdomen | General disorders | 1 of 26 |
| Pericardial effusion (non-malignant) | Cardiac disorders | 1 of 26 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Fulvestrant Treatment |
|---|---|---|
| Bromidrosis | Skin and subcutaneous tissue disorders | 2 of 26 |
| Headache | Nervous system disorders | 1 of 26 |
Serious events with the highest arm-level rate: bowel obstruction, non-specific (11.5%), death, non-specific (7.7%), pleural effusion (non-malignant) (7.7%), ascites (non-malignant) (3.8%), failure to thrive (3.8%).
For the Fulvestrant Treatment group, the registry lists 26 participants at risk for serious adverse events and 10 with a serious adverse event. It also lists 26 at risk for other adverse events and 2 with an other adverse event. These are participant counts in the registry record, not measures of treatment benefit. The record does not provide further detail about the timing, severity grading, duration, or relationship of these events to treatment.
The listed serious-event terms include Bowel obstruction, non-specific (11.5%), Death, non-specific (7.7%), Pleural effusion (non-malignant) (7.7%), Ascites (non-malignant) (3.8%), Failure to thrive (3.8%), and Fracture (3.8%). These term-specific percentages should be read as the registry's reported values, not as estimates recalculated from the participant counts. The record does not report the event-specific counts alongside these percentages. Deaths as a separate count are not reported in the registry record, even though Death, non-specific appears in the serious-event terms.
In general, an adverse event occurring during a trial is not, by itself, proof that treatment caused it. A single-group safety record also does not supply a concurrent comparison for judging whether event frequency differs from what would occur without the intervention. The registry's safety figures describe reported events within this study; they do not establish causality or comparative risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase II, single-group trial of fulvestrant was completed in patients with recurrent ovarian carcinoma. It also defines a Day 90 primary clinical-response endpoint and lists secondary outcomes. These details show what the study set out to examine, but they do not establish what the participants experienced on the primary endpoint.
The record does not report the number with complete response, partial response, or stable disease, the combined response count, or the number assessed. It also does not report an effect estimate or confidence interval. Therefore, the endpoint's magnitude and precision cannot be described from the registry record. Neither a positive nor a negative efficacy conclusion can be drawn from a definition of an outcome without its result.
The single-group design sets an additional limit. Even if the outcome result were available, it would describe the participants treated in this study rather than a randomized contrast with another treatment. In general, an uncontrolled result cannot isolate the contribution of treatment from other influences on the observed course. The absence of a comparator is a design feature, not evidence that the intervention did or did not affect outcomes.
The listed adverse-event counts and terms provide safety information, but they should not be interpreted as proof of causation or as a comparison with another treatment. The registry record does not provide details such as event timing or a separate death count. Secondary outcome results and endpoint-specific missing-data information are also not reported.
Accordingly, the record supports a description of the study design, intervention, endpoint definitions, and some reported safety figures. It does not support a conclusion about clinical efficacy, the precision of a response estimate, or a comparative benefit-risk balance. Those questions require results and analysis details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial illustrates why an endpoint definition and an endpoint result are different kinds of information. The registry specifies a clinically interpretable Day 90 response measure and defines its response categories. But without a response count, an assessed denominator, or an estimate of uncertainty, readers cannot quantify what the study observed. Clear definitions are necessary for interpretation, but they cannot replace reported data.
It also shows the importance of design when interpreting a treatment outcome. The trial is single-group, with allocation marked NA and masking recorded as none. A result from such a design would not provide a randomized comparison with a control group. Statistical precision, even if reported, would not remove this structural limitation: precision describes uncertainty in an estimate, whereas design determines what comparison the estimate can support.
The registry also identifies multiple secondary measures, including quality-of-life and biochemical outcomes. In general, multiple outcomes can offer a broader view of a trial, while also raising questions about prespecification and multiplicity. Those questions cannot be resolved from outcome names alone. The statistical analysis plan and multiplicity handling are not reported in the registry record.
Finally, the record underscores why safety counts require careful interpretation alongside efficacy data. Serious-event counts and listed terms describe reported events, but do not establish that treatment caused them or show how they compare with an alternative. Taken together, these features make transparent reporting of outcome denominators, analysis methods, missing data, and comparative design essential to a sound statistical reading.
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 most direct limitation is that the registry record defines the primary endpoint without reporting its result. It does not give response-category counts, the combined Day 90 response count, an endpoint denominator, or an estimate with uncertainty. Consequently, the central clinical outcome cannot be evaluated from the available record.
The single-group design provides no concurrent control comparison. Allocation is marked NA and masking is recorded as none. The record therefore does not support a randomized estimate of treatment effect relative to another intervention. It also does not report detailed eligibility criteria or participant characteristics, limiting assessment of how well the enrolled group represents the broader population of patients with recurrent ovarian epithelial cancer.
Endpoint ascertainment is not fully described. The registry gives response definitions but does not report assessment procedures, independent review, or the number with evaluable Day 90 data. It does not describe treatment completion, adherence, dose modifications, or reasons for discontinuation. The median number of days to treatment termination is listed as an outcome, but its result is not reported in the registry record.
Analysis details are also limited. The record lists 0 analyses and does not report the analysis population, missing-data approach, confidence intervals, or statistical tests. A statistical analysis plan, interim analyses, and the trial's sample-size justification are not reported in the registry record. The listed secondary outcomes create further questions about multiplicity, but the approach to those outcomes is not specified.
Safety information includes participant counts and serious-event terms, but event-specific counts, timing, causal assessments, and a separate death count are not reported. These omissions limit interpretation of both efficacy and safety, and should not be resolved by assumptions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Fulvestrant in Treating Patients With Recurrent Ovarian Epithelial Cancer — NCT00617188.
- Registry record last updated 2017-12-28; status last verified 2017-12.
- Results first posted 2010-03-17.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record describes a completed phase II, single-group trial of fulvestrant for recurrent ovarian carcinoma. Enrollment was 26; the recorded start date was 2007-06 and primary completion date was 2008-04. Allocation is NA and masking is none. The recorded regimen was 500 milligrams on Day 1, followed by 250 mg on Day 1, 29 and every 28 days thereafter.
The primary endpoint is overall clinical response through Day 90, defined as the sum of complete response, partial response, and stable disease under the stated RECIST criteria. The primary endpoint result and results for secondary outcomes are not reported in the registry record. The record lists serious adverse events in 10 of 26 participants at risk and other adverse events in 2 of 26 at risk, alongside several serious-event terms and percentages. It does not report a separate death count.
The record identifies the study question and some safety information, but does not provide the primary response estimate, its denominator, or the analysis details needed to assess efficacy and precision.