This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma (NCT00589784), a na phase 2 study. The primary outcome is Overall Objective Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00589784 |
| Phase | Phase 2 |
| Condition | CNS Cancer, Meningioma, Intracranial Hemangiopericytoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 50 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The study title identifies patients with recurrent or inoperable meningioma. The registry also lists CNS cancer, intracranial hemangiopericytoma, hemangioblastoma, and neurofibromatosis among the conditions. Enrollment was 50.
Intervention
The intervention was sunitinib. The treatment description specifies 50 mg orally once daily for four consecutive weeks, followed by a two-week rest period. It notes that intra-patient dose reduction may be required depending on the type and severity of toxicity.
Comparator
This was a single-group study, with allocation listed as not applicable and masking listed as none. The registry record does not identify a concurrent control group. The study therefore does not provide a like-for-like comparison between sunitinib and another treatment or control condition.
Primary question
The primary outcome was overall objective response, measured as a number of participants, with a timeframe of 1.5 years. The registry record does not report the number of participants who had an objective response or define the response criteria.
In statistical terms, the central question is how many participants met the study’s objective-response definition during the stated timeframe. A response count can describe what happened among participants in a single group, but without a concurrent comparator it cannot by itself estimate how outcomes would have differed under another treatment or under no study treatment. The response definition, assessment rules, and analysis population are not reported in the registry record, so the endpoint’s operational details cannot be assessed from this record.
3. Trial Design
This was a Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists enrollment of 50, a start date of 2007-10, a primary completion date of 2014-10, and a completed status. Allocation is listed as not applicable, and masking as none. These features describe a study without a listed randomized, masked comparison group; the registry explicitly indicates that its arms are not a like-for-like control comparison.
The intervention was sunitinib, administered orally at 50 mg once daily for four consecutive weeks followed by a two-week rest period. The treatment description says dose reduction within a participant might be needed according to the type and severity of toxicity. It also mentions imaging studies after every other, but the description ends before specifying what follows. Further imaging details are not reported in the registry record.
The title focuses on recurrent or inoperable meningioma, while the registry’s condition list also includes CNS cancer, intracranial hemangiopericytoma, hemangioblastoma, and neurofibromatosis. The precise eligibility criteria and the number of enrolled participants in each condition group are not reported in the registry record. Consequently, the enrolled population’s composition cannot be determined from the listed information alone.
In a single-group design, the observed response count describes the participants who received the study intervention, subject to the endpoint definition and analysis population. It does not supply a direct estimate of the difference between intervention and control outcomes. Changes in participants’ status over time, assessment practices, or other influences cannot be separated from intervention effects using a concurrent comparison in this design. Those are general limitations of interpreting an uncontrolled group, not findings about what occurred in this study.
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 | Aggressive Memingioma | Exploratory Cohort |
|---|---|---|
| STARTED | 36 | 14 |
| COMPLETED | 35 | 4 |
| NOT COMPLETED | 1 | 10 |
Reasons for not completing this period:
| Reason | Aggressive Memingioma | Exploratory Cohort |
|---|---|---|
| Withdrawal by Subject | 0 | 6 |
| Patient inevaluable | 1 | 0 |
| Adverse Event | 0 | 4 |
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 | Aggressive Memingioma | Exploratory Cohort | Total |
|---|---|---|---|---|
| Participants analysed | 36 | 13 | 49 | |
| Age, Customized: Median Age | years | 61 (27 to 85) | 48 (32 to 79) | 59 (27 to 85) |
| Sex: Female, Male: Female | Participants | 14 | 5 | 19 |
| Sex: Female, Male: Male | Participants | 22 | 8 | 30 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Objective Response, measured over 1.5 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is overall objective response, recorded as a number of participants, with a timeframe of 1.5 years. It is a binary-type outcome at the participant level: each participant either meets the study’s response criteria or does not, and the outcome can be summarized by counting participants who meet those criteria. The registry record gives the endpoint title and timeframe but does not report a response count, response proportion, response criteria, or assessment schedule.
As a general statistical concept, an objective-response endpoint is used to describe whether participants meet prespecified criteria for a response rather than relying only on a subjective impression. Such an endpoint can provide a clinically interpretable summary of tumor response, provided the response rules and assessment process are clearly defined. Those operational details are not reported in the registry record, so readers cannot assess how response was classified or whether assessments were independently reviewed.
A count alone would describe the number of participants with response, while an estimate of the response probability would also require a denominator and a defined analysis population. A confidence interval, if reported, would express uncertainty around an estimated response probability; it would not establish a comparison with another treatment. Neither an endpoint result nor its uncertainty interval is reported in the registry record. The available record therefore identifies what the primary endpoint was intended to measure, but does not establish the observed response outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies overall objective response as the primary endpoint and states a timeframe of 1.5 years. It does not report the observed number of participants with a response, the response proportion, or an uncertainty interval. As a result, the posted record does not allow a quantitative estimate of the primary endpoint to be interpreted. The endpoint’s response criteria and assessment procedures are also not reported in the registry record.
The study enrolled 50 participants and used a single-group design. Enrollment is not the same as the number included in an endpoint analysis: the analysis population and the number assessed for response are not reported in the registry record. Without those details, it is not possible to determine how much of the enrolled group contributed to the primary endpoint result. No primary endpoint analysis result is listed in the record.
General interpretation: a response count in a single group can describe the observed experience of participants assessed under a study’s rules. A confidence interval around a response estimate would communicate the statistical precision of that estimate. A wider interval indicates greater uncertainty; a narrower interval indicates greater precision under the assumptions of the analysis. Neither an interval nor its width can substitute for a comparison group when the question is whether outcomes differ between treatments.
There is no concurrent control group identified in this study. Accordingly, even if a response estimate were available, it could not be interpreted as a randomized estimate of the difference between sunitinib and another treatment. External comparisons can be affected by differences in eligibility, disease characteristics, follow-up, and outcome assessment. Whether any external comparison was planned or performed is not reported in the registry record.
The registry lists serious adverse event information separately from the primary endpoint. For the all-patients group, 50 participants were at risk and 27 were affected by serious events. These safety figures do not supply the missing objective-response result. Nor should serious-event percentages be combined into a single total: the registry does not indicate whether participants could experience more than one listed term or how terms were grouped for reporting.
For the primary question, the appropriate conclusion is limited: the trial was designed to assess overall objective response in a single group, but the registry record does not report the response result. No conclusion about the magnitude or precision of response can be drawn from the endpoint entry alone. The absence of a reported result is not evidence of either a favorable or an unfavorable outcome.
9. Statistical Methods Explained
The design is single-group, and the primary outcome is a participant count. These features determine what statistical summaries would be relevant, but the registry record does not report the primary endpoint analysis. The following are general explanations of the methods and concepts that could apply to a binary participant-level outcome; they are not claims about analyses actually performed in this study.
What does a binary endpoint summarize?
A binary endpoint classifies each participant into one of two categories under a defined rule, such as meeting or not meeting an objective-response definition. A count reports how many participants fall into the event category. A proportion relates that count to the number of participants included in the endpoint analysis. To interpret either quantity, the outcome definition, the analysis denominator, and the handling of participants without an assessment need to be clear. The registry record gives the endpoint title and timeframe, but does not report those further analysis details.
What would a confidence interval add?
As a general concept, a confidence interval gives a range of values compatible with the observed data under a statistical model and its assumptions. It describes uncertainty in an estimate; it is not a guarantee that the true value lies in the interval, nor does it correct bias in study design or measurement. For a single-group response estimate, an interval would address uncertainty about the response probability in the population represented by the study. It would not quantify a treatment-versus-control difference when no concurrent comparator is present. No confidence interval for the primary endpoint is reported in the registry record.
Why does the design matter?
A single-group study can document outcomes among participants who received the intervention. However, observed outcomes can also reflect other influences, including participant selection, disease course, and how outcomes are measured. General statistical reasoning therefore distinguishes a descriptive estimate within one group from a causal comparison between groups. Random allocation to a concurrent comparator can support a different kind of inference by helping make groups comparable on average, but this study’s registry entry identifies a single-group model and no like-for-like control comparison.
What do planning calculations mean?
General sample-size planning asks how many participants or events might be needed to detect an effect of a chosen size under specified assumptions. The planning illustration associated with this record is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it defines the effect to detect as a stated fraction of the observed effect. Those assumptions describe a planning calculation, not the single-group design listed for the trial. Any such sample-size illustration is hypothetical and is not observed power, a result of the trial, or evidence that the study achieved a particular level of statistical sensitivity. No numerical sample-size illustration or observed-effect estimate is reported in the registry record.
Why can over-dispersion matter?
General explanation: for count outcomes, a statistical model may assume that variability follows a specified relationship to the average count. Over-dispersion occurs when the observed variability is greater than that assumed by the model. If not addressed, standard errors can be too small and intervals or tests can appear more precise than warranted. The primary outcome in this record is a participant count for a binary-type endpoint, not a reported rate of recurrent events. The registry record does not report a model, a dispersion estimate, or an over-dispersion assessment.
What can be inferred from the listed analysis metadata?
The registry lists one outcome measure and zero analysis entries. This does not provide an endpoint estimate or explain what analysis methods were used. The statistical analysis plan, including any covariate adjustment, hypothesis test, confidence-interval method, or sensitivity analysis, is not reported in the registry record. It is therefore not possible to attribute a particular inferential method to the study or to assess whether a reported analysis followed a prespecified plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure and zero analysis entries. It names overall objective response as the primary outcome, but does not report its result. The number of outcomes listed in the record does not establish how many endpoints, time points, subgroups, or analyses were considered in the full study. The statistical plan for choosing among analyses or controlling false-positive findings is not reported in the registry record.
As a general concept, multiplicity arises when several hypotheses or analyses are evaluated. If many opportunities to find a statistically notable result are considered, the chance of at least one misleading finding can increase. Prespecified primary outcomes and analysis plans can help clarify which result carries the main inferential focus. Whether multiplicity adjustments or a prespecified hierarchy were used in this study is not reported in the registry record.
The registry does not report how missing response assessments, withdrawals, or participants without evaluable imaging were handled. The number of participants included in the primary endpoint analysis is also not reported in the registry record. These omissions prevent assessment of whether the endpoint result, if available elsewhere, would use all enrolled participants or only a subset.
In general, missing-data choices can affect a binary endpoint when the likelihood of being assessed is related to response. Different assumptions or analysis populations can lead to different estimates. The registry record does not describe imputation, censoring, missing-data sensitivity analyses, or the definition of an evaluable population. No particular approach should therefore be inferred.
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 | 27 | 50 | 54.0% |
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 |
|---|---|
| All Patients | 50 of 50 |
Most frequent serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Fatigue | General disorders | 9 of 50 |
| Thrombocytopenia | Respiratory, thoracic and mediastinal disorders | 6 of 50 |
| Lymphopenia | Blood and lymphatic system disorders | 5 of 50 |
| CNS Hemorrhage | Nervous system disorders | 4 of 50 |
| Neutropenia | Blood and lymphatic system disorders | 4 of 50 |
| Hypertension | Cardiac disorders | 4 of 50 |
| Headache | General disorders | 4 of 50 |
| Lueukopenia | Blood and lymphatic system disorders | 3 of 50 |
| Thrombic Microangiopathy | Respiratory, thoracic and mediastinal disorders | 2 of 50 |
| Hypophosphatemia | Metabolism and nutrition disorders | 2 of 50 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Patients |
|---|---|---|
| Leukopenia | Blood and lymphatic system disorders | 31 of 50 |
| Fatigue | General disorders | 29 of 50 |
| Thrombocytopenia | Respiratory, thoracic and mediastinal disorders | 23 of 50 |
| Diarrhea | Gastrointestinal disorders | 22 of 50 |
| Hypoalbuminemia | Metabolism and nutrition disorders | 19 of 50 |
| AST | Blood and lymphatic system disorders | 19 of 50 |
| ALT | Blood and lymphatic system disorders | 17 of 50 |
| Nausea | Gastrointestinal disorders | 17 of 50 |
| Hyperglycemia | Metabolism and nutrition disorders | 16 of 50 |
| Rash, Hand-Foot Reaction | Skin and subcutaneous tissue disorders | 16 of 50 |
Serious events with the highest arm-level rate: fatigue (18.0%), thrombocytopenia (12.0%), lymphopenia (10.0%), cns hemorrhage (8.0%), neutropenia (8.0%).
The registry lists an all-patients group with 50 participants at risk and 27 affected by serious events. It also lists serious-event terms with percentages: fatigue, 18.0%; thrombocytopenia, 12.0%; lymphopenia, 10.0%; CNS hemorrhage, 8.0%; neutropenia, 8.0%; and hypertension, 8.0%. These are safety descriptions and should be read separately from the primary objective-response endpoint.
The record does not specify whether participants could contribute more than one serious-event term, whether the percentages refer to participants or events, or the detailed coding and follow-up rules behind the term-level figures. General caution: categories of adverse events are not necessarily mutually exclusive, so adding their percentages would not necessarily give a meaningful overall rate. No such total is calculated.
The deaths value is not reported in the registry record. That absence does not establish that there were no deaths. The registry also reports 50 at risk and 50 affected for other events in the all-patients group, but does not provide the further definitions needed to interpret those fields. These safety data describe reported events; they do not establish a comparison with another treatment or determine whether an event was caused by sunitinib.
12. What the Result Does, and Does Not, Mean
The record documents a completed Phase II, single-group study of sunitinib with enrollment of 50. Its named primary endpoint is overall objective response over a timeframe of 1.5 years. However, the registry record does not report the primary endpoint result, response criteria, or an uncertainty interval. The central efficacy outcome therefore cannot be quantified or interpreted from this record.
The single-group design is important to the scope of any interpretation. General statistical reasoning distinguishes an outcome observed in one group from a comparison of outcomes under alternative treatments. Without a concurrent control group, an observed outcome cannot by itself show how participants would have fared under another treatment or without the study intervention. This is a design limitation, not a claim about the treatment’s effect.
The registry describes sunitinib dosing and notes that dose reduction could be required based on toxicity. It lists serious adverse event terms and group-level affected counts, but these safety entries do not answer the primary response question. The registry record does not provide enough detail to establish the causal relationship of particular events to treatment or to compare safety with another group.
General explanation: a confidence interval, when available, helps convey the precision of an estimate, but precision and validity are different. A narrowly estimated response probability could still be affected by selection, incomplete assessment, or an unclear response definition. Here, the endpoint result and key assessment details are not reported in the registry record, so neither the size nor the precision of the response outcome can be evaluated.
Accordingly, the record supports a description of the study design, intervention, primary endpoint, and selected safety information. It does not support a quantitative conclusion about objective response or a comparative conclusion about treatment effects. The absence of a registry result should not be read as evidence that the endpoint was positive, negative, or unchanged.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of how design determines the claims an outcome can support. The registry identifies a Phase II, single-group study and an objective-response endpoint. Those elements allow the intended outcome to be described, but they do not create a comparison with another treatment. A within-group response result, if reported, would answer a descriptive question about participants in the study rather than directly estimate a randomized treatment difference.
The distinction between an endpoint definition and an endpoint result also matters. The registry names overall objective response and gives a 1.5-year timeframe, but does not report the number of participants meeting the response criteria or the criteria themselves. Statistical interpretation depends not only on the label of an outcome but also on how it was assessed, who was included, and how uncertainty was quantified. Those details are not reported in the registry record.
The study also illustrates why safety summaries and efficacy endpoints should not be conflated. Serious adverse event terms and affected counts describe a different aspect of participants’ experience than objective response. Without a comparator and fuller reporting details, those safety entries cannot establish relative safety or attribute each event to the intervention.
General statistical lesson: a study’s design, outcome definition, denominator, and analysis plan jointly determine what can be learned. A confidence interval can clarify precision, while a control group can support a comparative question; neither can compensate for an endpoint result that is not reported. The registry record is useful for identifying the intended question and design, while leaving the primary response outcome unresolved.
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 limitation for interpreting this specific trial from the registry record is that the primary endpoint result is not reported. The record names overall objective response, gives a timeframe of 1.5 years, and identifies the outcome unit as participants, but does not give the response count, response proportion, or confidence interval. The response criteria and assessment rules are not reported in the registry record either.
The study is listed as single-group, with allocation not applicable and masking none. It therefore lacks a concurrent control comparison in the registry description. This limits comparative interpretation independently of whether an outcome estimate is available. The registry does not report the number of participants included in the primary analysis, missing-assessment handling, or the analysis population.
The title focuses on recurrent or inoperable meningioma, while the condition list includes several additional diagnoses. Eligibility criteria and the distribution of enrolled participants by condition are not reported in the registry record. The treatment description includes a dosing schedule and possible dose reduction, but its final imaging sentence is incomplete. Further details about assessment timing and dose management are not reported in the registry record.
The record lists serious adverse-event terms and percentages but does not report detailed definitions, attribution rules, or whether participants could appear under multiple terms. Deaths are not reported in the registry record. The statistical analysis plan, including any adjustments, sensitivity analyses, and missing-data methods, is also not reported. These limits prevent a full assessment of outcome ascertainment, precision, and robustness.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma — NCT00589784.
- Registry record last updated 2016-01-25; status last verified 2015-12.
- Results first posted 2016-01-25.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT00589784 is a completed Phase II, single-group trial sponsored by Memorial Sloan Kettering Cancer Center. The registry lists enrollment of 50, a start date of 2007-10, and a primary completion date of 2014-10. The study title concerns sunitinib in patients with recurrent or inoperable meningioma. The intervention description specifies 50 mg orally once daily for four consecutive weeks followed by a two-week rest period, with possible dose reduction based on toxicity.
The primary endpoint is overall objective response, measured in participants over 1.5 years. The registry record does not report the endpoint result, response criteria, or analysis population. It also lists serious adverse-event information for all patients, including 27 affected among 50 at risk and several term-specific percentages. The deaths value is not reported in the registry record. No like-for-like control comparison is identified.