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 RAD001 Plus Bevacizumab in Metastatic Melanoma (NCT00591734), a na phase 2 study. The primary outcome is Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00591734 |
| Phase | Phase 2 |
| Condition | Metastatic Melanoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 57 |
| Arms in the primary outcome | n/a |
| Lead sponsor | SCRI Development Innovations, LLC |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic melanoma enrolled in a Phase II study. The registry record reports enrollment of 57; it does not describe further eligibility details or the population included in the primary endpoint analysis.
Intervention
All patients received bevacizumab 15 mg/kg by intravenous infusion on day 1 of each 21 day course, together with everolimus 10 mg orally each day.
Comparator
No separate comparator group is described. The design is listed as single group, with no masking and allocation recorded as NA. The registry brief summary uses the wording “comparing bevacizumab and everolimus,” but the arm description says that all patients received both drugs.
Primary question
The primary endpoint is progression-free survival: the time patients were alive from their first date of protocol treatment until their disease worsened. The registry lists a 13 months timeframe and a median with a 95% confidence interval, but the estimate and interval are not reported in the registry record.
In plain terms, the study was intended to describe progression-free survival among patients receiving the two-drug regimen. Because the registry lists one treatment group and no comparator, the design does not directly estimate how outcomes under the regimen differ from outcomes under another treatment or a control condition. The registry record identifies overall survival rate and objective response rate as secondary outcomes, but does not give their results in the information recorded for this analysis.
The clinical question is therefore about outcomes observed in a treated group, rather than a randomized comparison of treatment strategies. The record does not report an estimate of the primary endpoint, so its magnitude and precision cannot be assessed from the registry information available.
3. Trial Design
This is a completed Phase II study with a single-group design. The registry records enrollment of 57 and identifies metastatic melanoma as the condition. The study is listed as non-randomized and open label, with no masking and allocation recorded as NA. Its start is listed as 2008-01 and primary completion as 2010-10.
All patients in the intervention group received bevacizumab and everolimus. Bevacizumab was administered by intravenous infusion at 15 mg/kg on day 1 of each 21 day course; everolimus was taken orally at 10 mg daily. The registry does not describe a distinct control arm. Although the brief summary says the study is “comparing bevacizumab and everolimus,” the arm description specifies that both drugs were given together to all patients. That wording does not establish a head-to-head comparison.
The primary endpoint is progression-free survival, described as the length of time patients were alive from their first date of protocol treatment until disease worsening. The listed timeframe is 13 months. Overall survival rate and objective response rate are identified as secondary outcomes. The registry record does not report the methods used to assess worsening, the handling of deaths before documented worsening, or further details of endpoint assessment.
The record does not report the protocol’s sample-size justification, a planned statistical analysis strategy, interim analyses, or details of analysis populations. It records no analyses. These omissions limit what can be concluded about how the study’s planned methods relate to any observed outcomes.
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 | Intervention |
|---|---|
| STARTED | 57 |
| COMPLETED | 4 |
| NOT COMPLETED | 53 |
Reasons for not completing this period:
| Reason | Intervention |
|---|---|
| Lack of Efficacy | 35 |
| Adverse Event | 5 |
| Physician Decision | 5 |
| Intercurrent Illness | 8 |
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 | Intervention |
|---|---|---|
| Participants analysed | 57 | |
| Age, Continuous | years | 70 (36 to 88) |
| Sex: Female, Male: Female | Participants | 18 |
| Sex: Female, Male: Male | Participants | 39 |
| Region of Enrollment: United States | participants | 57 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival, measured over 13 months.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is a time-to-event endpoint. In this study, the registry describes it as the time patients were alive from their first date of protocol treatment until their disease worsened. The listed timeframe is 13 months, and the planned summary measure is a median with a 95% confidence interval.
As a general statistical concept, a time-to-event endpoint incorporates both whether an event occurs and how long it takes to occur. A median time-to-event estimate describes the point by which half of the analyzed group has experienced the defined event, subject to the analysis methods and follow-up available. A confidence interval expresses uncertainty around an estimate; it is not a range containing every individual patient’s outcome.
Progression-free survival is used in clinical research when the timing of disease worsening is of interest, rather than only whether worsening occurred by a single fixed point. Its interpretation depends on a clear event definition, reliable assessment, and appropriate handling of participants whose event status is not known during follow-up. Those methodological details are not reported in the registry record. The median estimate and its confidence interval are also not reported in the registry record, so the endpoint’s observed value and precision cannot be described.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies progression-free survival as the primary endpoint and describes a median estimate with a 95% confidence interval over a 13 months timeframe. However, neither the median nor its confidence interval is reported in the registry record. Consequently, the posted information does not establish the observed duration of progression-free survival or how precisely it was estimated.
The study design also determines what the result could mean. This is a single-group study in which all patients received bevacizumab plus everolimus. There is no separate comparator group in the arm description. Therefore, any endpoint estimate would describe outcomes in the treated group; by itself, it would not quantify a difference between the regimen and another treatment, nor establish that a treatment caused an observed outcome. The brief summary’s wording about “comparing” the drugs is not consistent with the arm description, which specifies their combined use.
The registry lists overall survival rate and objective response rate as secondary outcomes, but does not report their results in the available record. It records no analyses. No hazard ratio or other sponsor-reported comparative estimate is reported in the registry record. The record therefore does not support a comparison of event rates, times, or treatment effects.
As a general statistical concept, precision is reflected by the uncertainty around an estimate, often conveyed by a confidence interval. Without the primary endpoint estimate and interval, it is not possible to judge whether the estimate is narrow or wide, or to distinguish a precise estimate from one with substantial uncertainty. The enrollment count of 57 describes the study’s recorded enrollment, not necessarily the number included in the endpoint analysis; the analysis population is not reported in the registry record.
The registry records serious adverse events affecting 29 of 57 at risk and other adverse events affecting 56 of 57 at risk. These figures describe safety reporting in the intervention group; they do not provide a comparison with another group. The listed serious event terms and percentages give further descriptive information, but the record does not establish whether events were judged related to treatment. Taken together, the available information describes the design, dosing, endpoint definition, and some safety reporting, but it does not give the primary efficacy estimate needed to characterize the observed progression-free survival result.
9. Statistical Methods Explained
The registry lists a single-group, non-randomized, open-label design. In a single-group study, outcomes are observed in participants receiving the specified intervention without a separate study comparator described in the record. This differs from a randomized comparison, where assigned groups can be compared under the trial’s allocation procedure. Here, allocation is recorded as NA and masking as NONE. The record does not describe a randomization process or a masked outcome assessment.
What does a median time-to-event measure?
The primary outcome is progression-free survival, described as time from the first date of protocol treatment until disease worsening, while patients were alive. Its summary measure is a median, and the registry lists a 95% confidence interval. As a general statistical concept, the median time-to-event estimate marks the point at which the estimated event-free proportion reaches one half, when that point can be estimated from the available follow-up. It is a summary of the group’s event-time distribution, not a statement that every participant experienced the event at that time.
Time-to-event methods can use information from participants whose event has not been observed by the end of their available follow-up, provided the analysis appropriately handles that incomplete observation. Such participants are often described as censored in general statistical practice. The registry record does not report the censoring rules, follow-up procedures, or method used to calculate the median and its interval. It is therefore not possible to assess those details for this study.
How should the confidence interval be read?
In general, a confidence interval communicates uncertainty in an estimate under the statistical method used. A narrower interval indicates greater precision under that method, while a wider interval indicates less precision. It does not describe the range of individual outcomes, and it does not by itself establish a treatment effect. The registry specifies a 95% confidence interval for the primary endpoint but does not report its limits or the median estimate. The interval’s width and the estimate’s precision cannot therefore be evaluated.
What does the one-group design permit?
Descriptive estimates can characterize outcomes observed among the participants analyzed, subject to the completeness and methods of the data. Without a separate comparator group, the design does not directly provide an estimate of the difference in outcomes between the regimen and another strategy. Other factors may also influence outcomes in a single group; the registry information does not describe methods to account for them. The summary’s use of “comparing” does not change the listed single-group design or the description that every patient received both drugs.
How do planning illustrations differ from trial results?
As a general concept, sample-size planning asks how much information a future study might need to detect a prespecified effect under stated assumptions. The planning assumptions associated with this record describe two-sided significance level 0.05, equal allocation between two arms, and a normal approximation, with the effect expressed as a stated fraction of the observed effect. For time-to-event planning, the stated convention is to give the total number of events using the Schoenfeld approach; for other outcomes, it is participants per arm. Such planning illustrations are not observed study outcomes and are not observed power.
Those two-arm assumptions do not describe the listed single-group trial, whose allocation is NA and whose intervention group received both drugs. They should not be read as evidence that this trial randomized participants or had a two-arm control comparison. The registry record does not report the trial’s own sample-size justification or statistical plan. It also records no analyses, and no sponsor-reported hazard ratios are listed in the available information. The general planning framework cannot fill those gaps or substitute for the missing endpoint estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry identifies progression-free survival as the primary outcome and lists overall survival rate and objective response rate as secondary outcomes. The record does not describe a testing hierarchy, adjustment for multiple comparisons, or a strategy for interpreting several endpoints together. These multiplicity methods are not reported in the registry record.
As a general statistical concept, testing multiple outcomes can increase the chance that at least one result appears notable by chance, depending on the tests and decision rules used. A prespecified approach can clarify which comparisons are primary and how secondary findings should be interpreted. Without the analysis plan, it is not possible to determine whether or how multiplicity was addressed in this study. The registry also does not report outcome estimates for the listed secondary outcomes in the information available for this analysis.
The primary endpoint description states a median and 95% confidence interval, but the estimate and interval are not reported in the registry record. The record does not report missing-data rules, censoring rules, or the numbers of participants contributing to each endpoint analysis. Enrollment of 57 should not be assumed to equal the analysis population. The record also does not describe whether participants were excluded from analyses or how incomplete endpoint information was handled.
The listed design is single group, and the record records no analyses. As a result, the available information does not allow assessment of an analysis population definition, adjustment strategy, or missing-data sensitivity analysis. These are reporting gaps; they do not establish that a particular method was or was not used outside what the registry record states.
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 |
|---|---|---|---|
| Intervention | 29 | 57 | 50.9% |
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 |
|---|---|
| Intervention | 56 of 57 |
Most frequent serious adverse event terms
| Event term | Organ system | Intervention |
|---|---|---|
| Pain - Abdomen | General disorders | 5 of 57 |
| Death | General disorders | 3 of 57 |
| Infection - Pneumonia | Infections and infestations | 3 of 57 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 3 of 57 |
| Cardiac Ischemia/Infarction | Cardiac disorders | 2 of 57 |
| Fever | General disorders | 2 of 57 |
| Dehydration | Gastrointestinal disorders | 2 of 57 |
| Pain - Chest | General disorders | 2 of 57 |
| Anemia | Blood and lymphatic system disorders | 1 of 57 |
| Hypertension | Cardiac disorders | 1 of 57 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Intervention |
|---|---|---|
| Fatigue | General disorders | 38 of 57 |
| Mucositis/Stomatitis | Gastrointestinal disorders | 31 of 57 |
| Hemoglobin | Blood and lymphatic system disorders | 28 of 57 |
| Platelets | Blood and lymphatic system disorders | 28 of 57 |
| Anorexia | Gastrointestinal disorders | 27 of 57 |
| Rash/Desquamation | Skin and subcutaneous tissue disorders | 26 of 57 |
| Diarrhea | Gastrointestinal disorders | 25 of 57 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 23 of 57 |
| Proteinuria | Metabolism and nutrition disorders | 22 of 57 |
| Cough | Respiratory, thoracic and mediastinal disorders | 21 of 57 |
Serious events with the highest arm-level rate: pain - abdomen (8.8%), death (5.3%), infection - pneumonia (5.3%), dyspnea (5.3%), cardiac ischemia/infarction (3.5%).
The registry records 57 participants at risk for serious adverse events, with 29 affected. It also records 57 at risk for other adverse events, with 56 affected. These are group-level safety counts for the intervention group; the record does not provide a separate comparator group for interpreting them.
The listed serious adverse event terms and percentages are Pain - Abdomen, 8.8%; Death, 5.3%; Infection - Pneumonia, 5.3%; Dyspnea, 5.3%; Cardiac Ischemia/Infarction, 3.5%; and Fever, 3.5%. These entries describe reported event categories and percentages in the registry. They should not be interpreted as proof that the study treatment caused the events. The registry record does not report an attribution assessment, the event-level details, or whether any participant experienced more than one listed event.
As a general statistical concept, adverse-event counts are descriptive unless a suitable comparison and analysis support a comparative interpretation. The one-group design does not provide a like-for-like control group. The listed figures therefore describe safety reporting in this group, not an estimate of excess risk compared with another treatment.
12. What the Result Does, and Does Not, Mean
The registry record describes a Phase II, single-group study in which all participants received bevacizumab plus everolimus. It identifies progression-free survival as the primary endpoint, with a median and 95% confidence interval over a 13 months timeframe. The endpoint definition, intervention schedule, enrollment, and some adverse-event counts and categories are recorded.
The primary endpoint’s median and confidence interval are not reported in the registry record. Without them, the observed progression-free survival estimate and its precision cannot be stated. Results for the listed secondary outcomes, overall survival rate and objective response rate, are also not reported in the information available for this analysis. The record lists no analyses.
Even if a group-level endpoint estimate were available, the design would describe outcomes among participants receiving the combination; it would not alone establish what outcomes would have been under another treatment. The registry lists no separate control group, and the brief summary’s use of the word “comparing” is inconsistent with the description of a single intervention group receiving both drugs. The record also does not report the analysis population, missing-data methods, or endpoint assessment procedures.
As a general statistical principle, an observed outcome and a causal treatment effect are different quantities. A treatment effect requires a credible comparison that addresses how outcomes would differ under alternative strategies. The listed design does not directly make that comparison. Nor do the safety counts alone show whether the events were caused by treatment; the registry does not report attribution or comparative safety results.
Accordingly, the available information supports a description of the study design and the outcomes the registry intended to assess, but not a conclusion about the magnitude or precision of progression-free survival, comparative efficacy, or comparative safety.
13. Why This Trial Matters Statistically
This study illustrates why the design description is essential when interpreting a clinical endpoint. The brief summary says the study is “comparing bevacizumab and everolimus,” while the arm description says that every patient received both drugs and the study model is single group. Those descriptions do not support reading the study as a head-to-head comparison. Recognizing that distinction prevents a descriptive outcome from being mistaken for a comparative treatment effect.
It also highlights the importance of reporting estimates alongside endpoint definitions. The registry identifies progression-free survival, a median summary, and a 95% confidence interval, but does not report the estimate or interval. Without the numerical result and its uncertainty, readers cannot assess the outcome’s magnitude or precision. Enrollment alone cannot replace that information or establish how many participants contributed to the endpoint analysis.
The record also shows why safety counts need context. Serious and other adverse-event figures are reported for the intervention group, but no comparator is described and attribution is not reported. These counts are descriptive and cannot by themselves establish treatment causation or comparative risk.
More broadly, the record distinguishes planned or listed outcomes from posted results. It names primary and secondary outcomes, yet the available information does not include their estimates. It also records no analyses and does not report the statistical plan. These gaps constrain interpretation: they leave readers unable to assess analysis choices, missing-data handling, multiplicity, or the precision of the primary result. Clear separation of study design, endpoint definition, analysis methods, and numerical findings is fundamental to 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 main trial-specific limitation in interpreting the registry information is that the primary endpoint estimate is absent. Although progression-free survival is defined and its intended summary is identified as a median with a 95% confidence interval, neither the estimate nor the interval is reported in the registry record. The listed secondary outcomes also have no reported estimates in the information available for this analysis.
The study is listed as single group and non-randomized, with all patients receiving both bevacizumab and everolimus. No comparator group is described. The brief summary’s wording about comparing the drugs conflicts with the arm description, so it cannot support a head-to-head interpretation. The registry does not report how disease worsening was assessed, the censoring rules, or the analysis population. It also does not report missing-data procedures or the protocol’s statistical analysis plan.
The registry records enrollment of 57, but does not state how many participants were included in the primary endpoint analysis. It records no analyses. As a result, the available information does not permit assessment of the methods behind any endpoint estimate or whether analyses accounted for incomplete follow-up. The listed serious adverse event counts and terms are not accompanied by a comparative group or reported treatment-attribution information.
These limitations concern what can be concluded from the registry record. They do not establish that methods or results were absent from other study materials; those details are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: RAD001 Plus Bevacizumab in Metastatic Melanoma — NCT00591734.
- Registry record last updated 2021-11-24; status last verified 2021-11.
- Results first posted 2013-02-26.
- 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 completed Phase II study of bevacizumab plus everolimus in metastatic melanoma. It lists a single-group, non-randomized, open-label design and enrollment of 57. All patients received bevacizumab 15 mg/kg by intravenous infusion on day 1 of each 21 day course and everolimus 10 mg orally each day.
Progression-free survival is the primary endpoint, defined as time from first protocol treatment until disease worsening while alive. The listed summary is a median with a 95% confidence interval over 13 months. The median and interval are not reported in the registry record. Overall survival rate and objective response rate are listed as secondary outcomes, but their results are not reported in the available information. The registry records serious adverse events affecting 29 of 57 at risk and other adverse events affecting 56 of 57 at risk. It records no analyses.