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 2 Trial of Regorafenib in Patients With Recurrent Ovarian, Primary Peritoneal and Fallopian Tube Cancer (NCT02278783), a na phase 2 study. The primary outcome is 6 Month Progression Free Survival (PFS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02278783 |
| Phase | Phase 2 |
| Condition | Ovarian Cancer, Primary Peritoneal Cancer, Fallopian Tube Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Utah |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with recurrent ovarian, primary peritoneal, or fallopian tube cancer. The registry does not give further eligibility details or describe the characteristics of the enrolled patient, so the population represented by the recorded enrollment cannot be characterized in greater detail.
Intervention
The intervention was regorafenib, recorded as a drug. The brief summary describes a non-blinded, single-arm study intended to test its efficacy in the specified recurrent gynecological cancers. Dose, schedule, treatment duration beyond the endpoint wording, and rules for changing or stopping treatment are not reported in the registry record.
Comparator
There was no comparison arm: the registry describes a single-group study, with allocation recorded as not applicable and masking as none. The study therefore does not provide a like-for-like control group for separating the observed course from what might have occurred without regorafenib or under another treatment.
Primary question
The primary question was whether participants with recurrent gynecological cancers had progression-free survival at 6 months on treatment, as a measure of regorafenib’s anti-tumor activity. The registry names 6 Month Progression Free Survival (PFS) as the primary endpoint and says patients would be checked after 6 months on treatment. It does not report the endpoint result, so the clinical question cannot be answered from the posted outcome information.
3. Trial Design
This was a phase 2, non-blinded, single-group study of regorafenib in patients with recurrent ovarian, primary peritoneal, or fallopian tube cancer. The registry identifies the University of Utah as sponsor. It records no allocation because the study had no randomized comparison, and masking was none. The arm description identifies a regorafenib treatment arm for all patients.
The registry lists enrollment as 1. That is the recorded enrollment figure, not evidence of a completed efficacy analysis or a basis for generalizing to a broader population. The study status is terminated. Its start is recorded as 2015-03 and primary completion as 2016-07; the reason for termination and the circumstances around recruitment or follow-up are not reported in the registry record.
The primary endpoint was 6 Month Progression Free Survival (PFS), with a timeframe stating that patients would be checked for PFS after 6 months on treatment. Secondary outcomes listed in the record were to estimate progression-free survival and the frequency of clinical benefit, defined there as stable disease, partial response, and complete response. The record notes 4 outcome measures but does not identify all of them in the available outcome descriptions.
There is no control arm against which to compare the outcome. Consequently, even if a treatment outcome were reported, this design would not by itself distinguish the treatment’s effect from other explanations for a participant’s course. The registry record lists 0 analyses and does not report an analysis plan, detailed eligibility criteria, the treatment regimen, or the reason the study was terminated. These gaps limit interpretation of both the intended design and the posted results.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | All Patients |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| 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 | All Patients |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | years | 71 (71 to 71) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as 6 Month Progression Free Survival (PFS), measured over Patients will be checked for PFS after 6 months on treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is named 6 Month Progression Free Survival (PFS). Its stated purpose was to evaluate regorafenib’s anti-tumor activity in patients with recurrent gynecological cancers, and the timeframe says that patients would be checked for PFS after 6 months on treatment. The registry records the outcome unit as participants and the parameter as a number. It does not provide a numerical result, a definition of the exact event counted, or details of how the assessment was conducted.
In general, progression-free survival is used to describe how long patients remain alive without their disease worsening. A fixed-time PFS measure asks whether that status is present at a specified assessment point, rather than describing the full course of time to an event. The exact operational definition and assessment rules for this study are not reported in the registry record, so this general description should not be taken as confirmation of the trial’s specific event criteria.
A six-month assessment can be clinically relevant because it focuses attention on disease control over a defined treatment interval. Its interpretation depends on clear outcome definitions, complete follow-up, and a result with enough detail to understand who was assessed and how missing assessments were handled. Those details, along with the primary endpoint result, are not reported in the registry record. Thus the endpoint’s stated purpose is clear, but its observed value and precision cannot be evaluated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a phase 2 study of regorafenib in recurrent ovarian, primary peritoneal, and fallopian tube cancer, but it does not post the numerical result for the primary endpoint, 6 Month Progression Free Survival (PFS). It also lists 0 analyses. The record therefore does not establish how many participants met the endpoint, what proportion did so, or how uncertain an estimate might be. No efficacy conclusion can be drawn from the outcome information recorded.
Enrollment is listed as 1, and the study is marked terminated. These registry entries describe the record, but do not explain why the study ended or how the enrolled participant’s follow-up was completed. The reason for termination, follow-up duration, and handling of any incomplete assessment are not reported in the registry record. Without those details, it is not possible to determine whether the primary endpoint was assessed as planned or whether the available record reflects a final analysis.
The absence of a comparator is also important. In general, a single-group study describes outcomes among treated participants but cannot provide a randomized estimate of how those outcomes differ from an appropriate control group. Disease course, patient selection, and other aspects of care can affect observed outcomes. The design therefore cannot establish a treatment effect by itself, even if an outcome value were available. Here, the primary outcome value is not reported in the registry record in any case.
The record lists secondary outcomes concerning progression-free survival and clinical benefit, including stable disease, partial response, and complete response. It does not provide their numerical results. It also does not report the outcome definitions or assessment procedures in enough detail to assess how those categories were assigned. The fact that 4 outcome measures are recorded does not substitute for the missing outcome values or definitions.
Precision cannot be assessed because there is no posted primary estimate, interval, or analysis. In general, a confidence interval communicates the range of estimates compatible with the data under a statistical model; it helps readers distinguish an imprecise estimate from one estimated more precisely. No confidence interval is reported for this endpoint. The enrollment figure alone cannot be converted into a meaningful endpoint estimate, and no such calculation is appropriate from the information in the record.
The study’s status and the lack of outcome analyses should be kept separate from claims about benefit or lack of benefit. Termination does not explain what happened clinically, and missing results are not equivalent to a negative result. The record supports a description of the intended question and the limited design information, but not a conclusion about regorafenib’s efficacy in this population.
9. Statistical Methods Explained
Statistical methods help specify how outcomes are defined, summarized, compared, and interpreted. For this study, the registry provides the primary endpoint name and timeframe, describes a single-group design, and records 0 analyses. A detailed statistical analysis plan is not reported in the registry record. The distinctions below explain general statistical concepts; they do not claim that any particular method was used in this study.
What does the single-group design allow?
A single-group study observes outcomes among participants who receive the intervention, without a concurrent control arm. It can describe what happened in the observed group when outcomes and follow-up are fully reported. It cannot, on its own, determine what would have happened to comparable participants under a different treatment or no treatment. In general, randomization helps make comparison groups comparable with respect to both measured and unmeasured factors. This study has no randomized comparison, so a treatment-versus-control effect is not available from its design.
Masking is recorded as none. In general, masking can reduce the influence of expectations on care, assessment, or reporting. The registry does not describe additional safeguards for outcome assessment, nor does it report whether the endpoint was assessed by a blinded reviewer. No details beyond the recorded absence of masking should be inferred.
What is a fixed-time progression-free survival endpoint?
The primary endpoint is 6 Month Progression Free Survival (PFS), with patients to be checked after 6 months on treatment. In general, time-to-event analysis uses information about when an event occurs and can account for differing follow-up times, including censoring when a participant’s event status is not observed through the end of follow-up. A fixed-time endpoint instead focuses on status at a specified time. How this study classified progression, death, or an unevaluable assessment is not reported in the registry record. The record also gives no event counts or time-to-event estimates.
The outcome is recorded in participants and as a number. That description indicates a count-oriented outcome entry, but does not supply the count. It should not be mistaken for a rate, a percentage, or a time-to-event estimate. No numerator, denominator beyond the enrollment figure, or uncertainty measure for the primary endpoint is posted. Therefore, no endpoint rate or confidence interval can be read from the record.
Why do estimates need uncertainty measures?
General statistical interpretation considers both the estimate and its uncertainty. A confidence interval gives a range of values consistent with the observed data under the assumptions of the analysis. Its width depends on factors such as the amount of information and the statistical model. An interval cannot be reconstructed when the endpoint result and its analysis are absent. The registry record does not report a confidence interval or other measure of precision for the primary outcome.
For count outcomes, a statistical model may need to account for variability beyond what a simple model expects. In general, over-dispersion means that observed variation exceeds the model’s assumed variation; ignoring it can make uncertainty appear too small. There is no reported count analysis for this study, and there is no basis to say that over-dispersion was present or that any particular model was used.
What do planning calculations mean?
In general, sample-size planning asks how much information a future study may need to detect a prespecified effect with a chosen error rate and power. Planning illustrations are not observed results and are not the same as a retrospective assessment of how likely the completed study was to find an effect. The registry’s planning description refers to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect expressed as a stated fraction of the observed effect. It also says that time-to-event illustrations use a total number of events, while other illustrations use participants per arm.
Those assumptions are described as a planning illustration computed from the observed effect, not as the trial’s own design. They do not turn this single-group study into a two-arm trial, and the record gives no calculated planning values in the registry record for interpretation. The planned illustration should not be described as the study’s actual enrollment target, a trial result, or observed power. The record does not report the original sample-size justification or design assumptions for the trial itself.
What can be concluded from the analysis record?
The registry lists 0 analyses and does not report an analysis population, a statistical plan, a primary endpoint estimate, or uncertainty measures. The number of outcome measures is recorded as 4, but outcome values are not reported. Without those elements, there is no statistical result to reproduce or evaluate. A reader can identify the research question and broad design, but cannot assess the endpoint estimate, model fit, missing-data assumptions, or the robustness of any finding.
10. Multiplicity, Missing Data and Analysis Populations
Multiplicity arises when a study evaluates more than one outcome, time point, subgroup, or hypothesis. In general, testing multiple questions can increase the chance that at least one result appears notable by chance. Researchers may address this with prespecified priorities or statistical adjustments, but the appropriate approach depends on the design and aims. The registry identifies a primary outcome and secondary outcomes, and records 4 outcome measures. It does not report a multiplicity strategy, so whether any adjustment or hierarchy was planned is not reported in the registry record.
The listed secondary outcomes include estimating progression-free survival and the frequency of clinical benefit, with clinical benefit described as stable disease, partial response, and complete response. Numerical results for these outcomes are not reported in the registry record. The identities of all recorded outcome measures, beyond those described, are not reported in the registry record.
Missing-data handling is also not described. In general, missing outcome data can affect interpretation when participants cannot be assessed or when follow-up ends before the planned assessment. Methods may depend on why data are missing and on assumptions about the unobserved outcomes. The record does not say whether the primary assessment was missing, how such data would be handled, or whether any sensitivity analysis was planned.
The analysis population is not reported in the registry record. For example, the record does not specify which participants would be included in the primary analysis or how deviations from treatment or assessment plans would be handled. The statistical analysis plan, prespecified stopping or interim procedures, and any rules for subgroup analyses are not reported in the registry record. With 0 analyses listed, there is no posted analysis from which to assess these 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 |
|---|---|---|---|
| All Patients | 0 | 1 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| All Patients | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| anemia | Blood and lymphatic system disorders | 1 of 1 |
| tachycardia | Cardiac disorders | 1 of 1 |
| oral mucositis | Gastrointestinal disorders | 1 of 1 |
| diarrhea | Gastrointestinal disorders | 1 of 1 |
| chills | General disorders | 1 of 1 |
| platelet count decrease | Investigations | 1 of 1 |
| creatinine increased | Investigations | 1 of 1 |
| AST increased | Investigations | 1 of 1 |
| weight loss | Investigations | 1 of 1 |
| hyponatremia | Metabolism and nutrition disorders | 1 of 1 |
The registry’s adverse-event entry is for the group titled All Patients. It records 1 participant at risk for serious adverse events and 0 affected. For other adverse events, it records 1 at risk and 1 affected. These are the figures posted for the registry group; they do not provide event descriptions or severity details for the other adverse event.
No serious adverse-event terms are listed. The death field is null, so a death count is not reported in the registry record; null should not be read as a recorded count of zero. The record does not provide enough detail to determine the nature, timing, duration, or relationship to regorafenib of the other adverse event. It also does not describe how adverse events were collected or define the reporting period.
In general, adverse-event counts describe events recorded under the study’s reporting procedures. They do not by themselves establish that an intervention caused an event, and small amounts of observed safety information cannot characterize uncommon or delayed harms. Here, the recorded at-risk and affected figures should be read as registry entries, not as a comprehensive account of safety. The absence of listed serious terms does not establish that no unreported events occurred.
12. What the Result Does, and Does Not, Mean
The registry record identifies the intended question: whether patients with recurrent ovarian, primary peritoneal, or fallopian tube cancer had progression-free survival at 6 months while receiving regorafenib. It records a phase 2, non-blinded, single-group study and enrollment of 1. It also records that the study is terminated. These details describe the study’s registered design and status, not the clinical outcome.
The primary endpoint result is not reported in the registry record, and the record lists 0 analyses. Secondary outcome results are also not reported. As a result, the posted information does not show how the participant’s disease course was classified, whether the primary assessment was completed, or what statistical uncertainty surrounds any outcome estimate. Missing outcome information cannot be interpreted as evidence that the treatment did or did not have an effect.
Even with a reported outcome, a single-group design would not provide a randomized control comparison. In general, observed outcomes in a treated group can reflect the intervention as well as patient characteristics, disease course, and other factors. The design can describe outcomes under the study conditions, but it cannot isolate the effect of treatment from these alternative explanations. The reason for termination and the details of follow-up are not reported in the registry record, so the status cannot be interpreted as an explanation of efficacy or safety.
The serious adverse-event entry records 0 affected among 1 at risk, while the other-adverse-event entry records 1 affected among 1 at risk. No serious terms are listed, and the death count is not reported. Those safety entries are limited records and do not establish a complete safety profile or causation. They should be considered alongside the absence of event descriptions and collection details.
In general, a result is more informative when its outcome definition, analysis population, estimate, and precision are all available. Those elements are not present for the primary endpoint in the registry record. The study therefore documents an intended research question and a limited set of design and safety details, but does not support a conclusion about clinical benefit, lack of benefit, or comparative effectiveness.
13. Why This Trial Matters Statistically
This study illustrates why a registered research question and a reported result are not the same thing. The registry specifies a primary endpoint, 6 Month Progression Free Survival (PFS), and a population with recurrent ovarian, primary peritoneal, and fallopian tube cancer. But the primary endpoint result is not reported, and the record lists 0 analyses. Statistical interpretation depends on the outcome data and the analysis that connects those data to a claim; without them, the central question remains unanswered by the posted results.
The single-group design is also statistically important. It can describe outcomes among treated participants if those outcomes are reported, but it has no randomized comparator for estimating a treatment contrast. A comparison with historical expectations or other external data would require suitable information and explicit methods, neither of which is reported in the registry record. The design should therefore be distinguished from a controlled efficacy comparison.
Enrollment is recorded as 1 and the study is marked terminated. These registry entries make careful attention to what the record does and does not contain especially important. They do not reveal the reason for termination, the completeness of follow-up, or the primary endpoint status. A small recorded enrollment cannot replace the missing outcome data or quantify uncertainty by itself.
The record also demonstrates why safety figures require context. The adverse-event entries give counts at risk and affected, but event terms, collection details, and a reported death count are not available. Counts without definitions and follow-up context provide only a narrow description. In general, clear outcome definitions, transparent analyses, and complete reporting are essential for readers to judge both efficacy and safety evidence.
Statistically, the most responsible conclusion is bounded: the registry documents the intended study question and some design information, but not the results needed to answer that question. Separating documented details from unavailable evidence prevents an absent result from being mistaken for either a favorable or unfavorable finding.
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.
Several limitations specific to this study restrict what can be learned from the registry record. It describes a single-group study with no randomized comparator and no masking. Consequently, there is no concurrent control outcome for a direct treatment comparison, and the design cannot isolate an intervention effect from other influences on observed outcomes.
The registry lists enrollment as 1 and marks the study terminated. It does not report why the study was terminated, whether follow-up was complete, or whether the planned 6-month assessment occurred. The primary endpoint result is not reported, and 0 analyses are listed. These gaps prevent evaluation of the endpoint outcome, its precision, and the analysis used to produce it.
The record names secondary outcomes but does not provide their results. Although it records 4 outcome measures, not all are identified in the outcome descriptions available for interpretation. Detailed outcome definitions, assessment procedures, eligibility criteria, treatment dose and schedule, and analysis-population rules are not reported in the registry record.
Statistical planning for the trial itself is also unclear. The registry describes planning illustrations based on an observed effect, but those illustrations are not identified as the trial’s own design justification. The original sample-size justification, statistical analysis plan, multiplicity approach, missing-data methods, and interim-analysis procedures are not reported in the registry record.
The safety information is similarly limited: serious and other adverse-event counts are posted, but serious terms are not listed, the other event is not described, and the death count is not reported. These limitations mean that the registry record supports a description of the study’s registered question and selected entries, not a full assessment of efficacy or safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase 2 Trial of Regorafenib in Patients With Recurrent Ovarian, Primary Peritoneal and Fallopian Tube Cancer — NCT02278783.
- Registry record last updated 2017-10-06; status last verified 2017-09.
- Results first posted 2016-12-02.
- 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 describes a phase 2, non-blinded, single-group study of regorafenib for recurrent ovarian, primary peritoneal, and fallopian tube cancer. The University of Utah is listed as sponsor. Enrollment is recorded as 1, the status is terminated, the start is 2015-03, and primary completion is 2016-07.
The primary outcome is 6 Month Progression Free Survival (PFS), with assessment after 6 months on treatment. Secondary outcomes concern progression-free survival and clinical benefit. The record lists 4 outcome measures and 0 analyses. The primary endpoint result and secondary outcome results are not reported in the registry record.
The adverse-event entry records 0 serious adverse events among 1 at risk and 1 other adverse event among 1 at risk. No serious terms are listed, and a death count is not reported. The record does not explain the study’s termination or provide the analysis and follow-up details needed for a fuller interpretation.