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 Treatment With Intravitreal Avastin for Large Uveal Melanomas (NCT00596362), a na na study. The primary outcome is The Response of Intravitreal Avastin in Causing a Clinically Significant Reduction in Uveal Melanoma Tumor Size (Base Height and Volume)..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00596362 |
| Phase | NA |
| Condition | Uveal Melanoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
The registry record concerns intravitreal Avastin for uveal melanoma. Its stated purpose was to test the safety and effectiveness of Avastin introduced into the inside of the eyeball, including whether it caused shrinkage of the tumor. Avastin is described in the record as an anti-cancer drug designed to shrink blood vessels within tumors.
Population
People with uveal melanoma are the condition-specific population. The record describes the disease as a tumor of the eye and labels the study population as large uveal melanomas. Enrollment was 1. Further eligibility details and participant characteristics are not reported in the registry record.
Intervention
The intervention was Avastin, listed as a drug and described as being introduced intravitreally, into the inside of the eyeball. The record does not provide additional treatment-administration details such as dose or schedule.
Comparator
No comparator group is described. The trial is listed as a single-group study, with allocation recorded as NA. Thus, the record does not describe a like-for-like control comparison.
Primary question
The primary question was whether intravitreal Avastin produced a clinically significant reduction in uveal melanoma tumor size, assessed by base height and volume. The listed response measure uses RECIST v1.0 categories assessed by MRI. A result for that measure is not reported in the registry record.
This question is about the observed response in the study group, not a comparison of Avastin with another treatment or with no treatment. Without a reported primary outcome result, the record does not establish whether the enrolled participant met a response category.
3. Trial Design
This was a completed, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists enrollment as 1 and the study phase as NA. Allocation is also listed as NA, and masking is listed as none. The intervention was Avastin; the arm description names AVASTIN. No comparator arm is described, so the design does not support a randomized, like-for-like treatment comparison.
The study start is recorded as 2007-04 and primary completion as 2008-06. The status is COMPLETED. These registry dates describe the study record; they do not establish the timing of any individual treatment or assessment beyond what the endpoint timeframe states.
The primary outcome is a participant-count measure, with the unit listed as participants and the parameter listed as NUMBER. Its timeframe is “At conclusion of study, up to 5 days.” The outcome description says response was assessed by MRI under RECIST v1.0 criteria for target lesions. The registry record does not report further details of visit schedules, treatment dose, administration schedule, eligibility criteria, or the procedures used to confirm measurements.
A single-group design can describe what was recorded among participants who received the intervention, but it does not isolate an intervention effect from other explanations for an outcome. In this record, the enrollment of 1 also means the study offers very limited information about how outcomes might vary across people with the condition. The registry lists 0 analyses and does not report a primary endpoint result. It is therefore not possible to interpret this record as evidence of a measured response or to assess the precision of an estimated treatment effect.
The record does not report a protocol-level statistical plan, a sample-size justification, interim analyses, or a planned comparator. Those details should not be inferred from the study status or from the listed endpoint definition.
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 | AVASTIN |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | AVASTIN |
|---|---|
| Progressive Disease | 1 |
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 | AVASTIN |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as The Response of Intravitreal Avastin in Causing a Clinically Significant Reduction in Uveal Melanoma Tumor Size (Base Height and Volume)., measured over At conclusion of study, up to 5 days.
| 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 a clinically significant reduction in uveal melanoma tumor size, described in relation to base height and volume. The registry specifies MRI assessment and RECIST v1.0 categories for target lesions. These categories distinguish complete response, partial response, progressive disease, and stable disease using changes in lesion measurements or the appearance of lesions.
As a general statistical concept, a categorical response endpoint turns observations into defined groups. Prespecified criteria can make response classification more consistent than an informal judgment, while imaging supplies a way to assess tumor dimensions. The listed RECIST thresholds are part of the endpoint definition; they are not themselves evidence that a participant experienced a response.
For this record, the listed partial-response criterion is a >=30% decrease in the sum of the longest diameter of target lesions. Progressive disease includes a 20% increase in that sum, a measurable increase in a non-target lesion, or the appearance of new lesions. Complete response is defined as disappearance of all target lesions. Stable disease is the category when the change does not meet the stated partial-response or progressive-disease criteria.
The outcome timeframe is at study conclusion, up to 5 days. The record does not report the observed count in any response category or a result for the primary endpoint. It also does not provide a separate result for base height or volume. Consequently, the endpoint definition explains what was to be measured, but the registry record does not show what was observed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary response endpoint but reports no result for it. Although the endpoint is expressed as a number of participants, the observed number meeting any response category is not reported in the registry record. The record also lists 0 analyses. There is therefore no posted primary outcome estimate, confidence interval, or statistical test to interpret.
This absence matters because an endpoint definition and an outcome result are different things. RECIST v1.0 specifies how response categories are assigned from imaging findings; it does not tell readers which category was observed. The record gives criteria for complete response, partial response, progressive disease, and stable disease, but provides no count for any of them. No conclusion about tumor-size change can be drawn from the criteria alone.
The enrollment of 1 and the single-group design sharply limit what this record can establish. Even if a participant-level result were reported, a single-group observation would not provide a concurrent control comparison. It could describe what happened in the study group, but could not by itself distinguish an intervention-related change from other explanations. In this registry record, the lack of a posted endpoint result means even that participant-level response description is unavailable.
Statistical precision is also not assessable for the primary endpoint. A confidence interval would describe uncertainty around an estimated response proportion or other defined quantity, but no primary outcome estimate or interval is reported. The record does not report an analysis method or statistical test. It would be inappropriate to reconstruct an efficacy estimate from the endpoint criteria or from safety counts.
The safety entries are separate from the tumor-response endpoint. For the AVASTIN group, the registry reports 1 participant at risk and 0 affected for serious adverse events, and 1 at risk and 0 affected for other adverse events. These counts describe the registry entries; they do not substitute for the missing tumor-response outcome. The record does not report a death count.
Overall, the posted information supports a description of the study’s intended question, design, endpoint definition, and recorded safety counts. It does not support a numerical estimate of response, a treatment-effect comparison, or a conclusion about the effectiveness of Avastin. The study’s completed status should not be mistaken for a posted result.
9. Statistical Methods Explained
What does a single-group design show?
In a single-group study, the intervention is given within the study group, and outcomes are described for that group. General statistical context: such a design can document observations, but without a comparator it cannot directly estimate how outcomes would have differed under another treatment or under no intervention. Differences between people, natural changes over time, and other influences cannot be separated from the intervention using a single-group comparison alone.
Here, the registry lists a single-group model, allocation as NA, and masking as none. No comparator is described. The study has enrollment of 1, and the record lists 0 analyses. These registry entries leave no posted comparative estimate and no primary outcome analysis to evaluate. The record does not report a statistical plan that would clarify whether analyses were intended beyond the outcome definition.
What does a binary response measure mean?
The outcome is classified as a participant count, and the analysis type is identified as binary. In general, a binary outcome assigns observations to categories such as whether a specified event or response occurred. A count is meaningful only alongside a clearly defined group and denominator. The record provides a primary endpoint definition but does not report the response count or the number assessed for that endpoint. As a result, a response proportion cannot be read from the posted results.
RECIST v1.0 supplies rules for classifying imaging findings. The record defines complete response as disappearance of all target lesions; partial response as a >=30% decrease in the sum of the longest diameter of target lesions; progressive disease as a 20% increase in that sum, a measurable increase in a non-target lesion, or new lesions; and stable disease as neither sufficient decrease for partial response nor sufficient increase for progressive disease. The rules describe classification, not the outcome observed in this study.
What would uncertainty measures add?
General statistical context: an estimate describes the result calculated from observed data, while a confidence interval communicates the uncertainty associated with that estimate under specified assumptions. A statistical test addresses a particular hypothesis and depends on an analysis plan and data. These tools do not repair the lack of a comparator, and their interpretation depends on the endpoint, denominator, and methods used.
No primary endpoint estimate, confidence interval, or test result is reported in the registry record. The registry also lists 0 analyses. It is therefore not possible to assess the precision of a response estimate or to evaluate a hypothesis test for the primary outcome. No recalculated result is available to compare with a sponsor analysis.
Why does over-dispersion matter in general?
General statistical context: over-dispersion occurs when observed variation is greater than a statistical model assumes. If a model underestimates variation, uncertainty can appear too small and inferential results can be misleading. Whether over-dispersion is relevant depends on the outcome structure and analysis model. The registry record does not report a model for the primary endpoint or an analysis of over-dispersion, so no model-based conclusion on this issue can be made for this study.
How should planning illustrations be understood?
General statistical context: sample-size planning asks how much information a future study would need to address a specified question under stated assumptions. A planning illustration is not an observed result and is not the same as observed power. The registry’s planning note describes illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also says the effect to detect is a stated fraction of the observed effect; time-to-event illustrations use total events, while other illustrations use participants per arm.
Those assumptions are not the design of this trial, which is recorded as single-group. The registry record does not report a primary endpoint result from which an observed effect could be assessed, nor does it report a trial-specific sample-size justification. Any planning illustration should therefore be read as a hypothetical planning exercise, not as evidence about the trial’s outcome or its ability to detect an effect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 1 outcome measure and 0 analyses. It does not report a multiplicity-adjustment method or explain whether any additional outcomes or comparisons were planned. Multiplicity concerns arise when several hypotheses or endpoints are tested, because the chance of a misleading positive result can increase across multiple tests. General statistical context: prespecified priorities and appropriate methods help readers understand how multiple analyses are handled. The record does not provide enough information to assess such procedures.
The primary endpoint definition is present, but an observed endpoint result is not reported in the registry record. The record does not state whether the endpoint was assessed for the enrolled participant, whether any imaging measurement was missing, or how missing measurements would have been handled. Missing-data methods can affect interpretation when outcome availability is related to participants’ characteristics or outcomes; no such method is reported.
The analysis population is not reported in the registry record. There is no description of an intention-to-treat population, a per-protocol population, or another analysis set. Because the record lists a single-group study with enrollment of 1 and 0 analyses, there is no reported analysis population or primary outcome analysis to inspect. The endpoint timeframe is at study conclusion, up to 5 days, but the record does not describe the timing or completeness of endpoint assessment.
These omissions limit interpretation; they do not establish that analyses, missing data, or multiplicity problems occurred. They indicate that the registry record does not report how these issues were addressed.
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 |
|---|---|---|---|
| AVASTIN | 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 |
|---|---|
| AVASTIN | 0 of 1 |
For the AVASTIN group, the registry reports 1 participant at risk for serious adverse events and 0 affected. It also reports 1 at risk for other adverse events and 0 affected. These are counts in the registry record. They should be read as the reported entries for the listed categories, not as evidence that all possible harms were assessed or that no adverse events could occur.
The registry record does not list serious adverse-event terms. It does not report a death count; the death entry is not populated. The time period and detailed methods for adverse-event collection are not reported in the registry record. The entries therefore do not show how events were solicited, classified, or followed over time.
With enrollment of 1, the safety counts offer limited information about the range or frequency of possible adverse events. A zero count in the listed entries does not establish the absence of risk beyond the recorded observations. The safety information is also distinct from the primary tumor-response endpoint, for which a result is not reported.
12. What the Result Does, and Does Not, Mean
This registry record establishes that a completed, single-group study examined intravitreal Avastin for large uveal melanomas, with a primary endpoint defined around clinically significant tumor-size reduction using MRI and RECIST v1.0. It also reports the intervention, enrollment, study dates, and safety counts. These details help characterize the study question and what the registry recorded.
The record does not report the primary endpoint result. It therefore does not show whether the participant met criteria for complete response, partial response, progressive disease, or stable disease. There is no reported response estimate, confidence interval, or test result. The listed RECIST thresholds cannot be used to infer what happened to the tumor.
The single-group design does not include a comparator, and allocation is listed as NA. Consequently, the record does not support a direct treatment comparison. More generally, an outcome observed in a single group cannot, on its own, separate an intervention effect from other possible explanations. The absence of a posted primary result further limits interpretation.
The safety entries report 0 affected for both serious and other adverse events, with 1 at risk in each category. These counts describe the registry entries; they do not establish that the treatment has no risks. The death count and detailed adverse-event collection methods are not reported in the registry record.
The appropriate reading is narrow: the record documents a study and defines its intended tumor-response outcome, but does not provide the outcome needed to evaluate response. It cannot establish benefit, lack of benefit, or comparative effectiveness. Nor can the missing result be treated as a negative result. The posted information should be distinguished from any conclusion that would require a reported endpoint analysis.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a registered research question and an interpretable result. The record clearly defines a tumor-response endpoint using MRI and RECIST v1.0, but does not report the observed response category or any primary analysis. A detailed endpoint definition is useful, yet it cannot replace the outcome data needed to estimate response.
The design also makes the limits of inference visible. This is a single-group study with allocation listed as NA and no comparator described. Even a reported outcome would be descriptive rather than a like-for-like comparison with another treatment group. Here, enrollment is 1 and the registry lists 0 analyses, so there is no reported estimate whose precision or statistical significance could be assessed.
The safety entries show why counts need context. The record reports 0 affected in both listed adverse-event categories, but does not provide a death count or detailed collection methods. A count is not a complete characterization of safety unless readers can understand its definition, observation period, and completeness. Those details are not reported in the registry record.
Finally, the record’s planning note is separate from the trial design. General sample-size illustrations can help frame future research, but they are not trial outcomes or observed power. The trial-specific sample-size justification is not reported in the registry record. Keeping design, endpoint definitions, observed results, safety reporting, and planning assumptions distinct is essential to a careful 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 central limitation is that the registry record does not report a result for the primary endpoint. It gives the response definitions and timeframe, but no count in the response categories, no primary analysis, and no estimate of uncertainty. The reader therefore cannot determine what response was observed.
The design is single-group, with no comparator described and allocation listed as NA. This prevents a like-for-like comparison. Enrollment is 1, which limits the amount of information the study can provide about variation across participants. The registry lists 0 analyses, and the analysis population is not reported in the registry record.
Important operational details are also not reported in the registry record, including dose, treatment schedule, eligibility criteria, participant characteristics, endpoint assessment completeness, and statistical methods. The record does not report how missing measurements would be handled, whether interim analyses were planned, or whether any multiplicity procedure was used. These absences should not be interpreted as proof that the procedures were not undertaken; they limit what can be assessed from the registry record.
Safety information is limited to the posted counts: 1 at risk and 0 affected for serious adverse events, and 1 at risk and 0 affected for other adverse events. Specific serious event terms, a death count, and detailed adverse-event collection methods are not reported in the registry record. These limitations make the entries insufficient for a comprehensive assessment of safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment With Intravitreal Avastin for Large Uveal Melanomas — NCT00596362.
- Registry record last updated 2016-06-17; status last verified 2016-05.
- Results first posted 2015-04-14.
- 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
NCT00596362 is a completed, single-group study titled “Treatment With Intravitreal Avastin for Large Uveal Melanomas,” sponsored by Memorial Sloan Kettering Cancer Center. The registry lists enrollment of 1, phase NA, allocation NA, and masking none. The study start is 2007-04, and primary completion is 2008-06.
The intervention was Avastin. The primary endpoint concerned clinically significant reduction in uveal melanoma tumor size, assessed by MRI using RECIST v1.0 criteria, at study conclusion, up to 5 days. The registry lists 1 outcome measure and 0 analyses, but does not report a primary endpoint result.
For the AVASTIN group, the safety record lists 1 at risk and 0 affected for serious adverse events, and 1 at risk and 0 affected for other adverse events. A death count is not reported in the registry record. No comparator is described, so the posted information does not provide a comparative treatment estimate.