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 Topical Avastin for Treatment of Corneal Neovascularization (NCT00559936), a na phase 1 study. The primary outcome is The Size and Extent of Corneal Neovascularization Will be Measured by Computerized Image Analysis of Corneal Photographs Taken Throughout the Study..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00559936 |
| Phase | Phase 1 |
| Condition | Corneal Neovascularization |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 24 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Massachusetts Eye and Ear Infirmary |
| Results posted | Yes |
2. Clinical Question
Population
Participants with corneal neovascularization who received the investigational treatment. The registry record reports enrollment of 24, but does not provide further eligibility details or a fuller description of the analyzed population.
Intervention
Topical Avastin 1% (bevacizumab). The arm description says each patient would receive topical Avastin in one eye. The registry record identifies this as a Phase 1 study.
Comparator
No comparator group is reported. The study is identified as single-group, with allocation listed as NA and no masking. It therefore does not provide a like-for-like control comparison.
Primary question
How did the size and extent of corneal neovascularization, measured from corneal photographs using computerized image analysis, change from baseline during follow-up? The primary measure is percent change since baseline, assessed at six months. The registry record does not report the observed mean change or its standard deviation.
3. Trial Design
The registry record describes a completed Phase 1 study of topical bevacizumab, identified by the intervention name Topical Avastin 1%. Its stated purpose was to assess the effectiveness and safety of topical bevacizumab for corneal neovascularization. The condition listed is corneal neovascularization, and the sponsor is Massachusetts Eye and Ear Infirmary.
This was a single-group study, not a randomized comparison. Allocation is listed as NA, and masking is listed as none. The arm description states that each patient would receive topical Avastin in one eye. There is no like-for-like control arm in the record, so the design does not directly compare the intervention with an untreated group or another treatment group.
Enrollment is reported as 24. The study start is recorded as 2007-02 and primary completion as 2010-10; the status is completed. These registry entries describe enrollment and study timing, not the timing or completeness of individual participants’ follow-up.
The primary outcome concerns change in the photographed size and extent of corneal neovascularization. The registry describes computerized image analysis of corneal photographs and specifies percent change from baseline at six months. The population description says that participants who received investigational treatment were analyzed. The record does not provide further detail about how many participants contributed primary-outcome data or how missing measurements were handled.
The registry lists two outcome measures but records no analyses. In particular, it does not report a primary-outcome mean, standard deviation, confidence interval, or statistical test. Those omissions limit interpretation of efficacy; the study design and outcome definition alone do not establish the magnitude or precision of change.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Only patients with superficial or deep corneal neovascularization that extends farther than 2 mm from the limbus will be considered.
Overall Study
| Stage | Topical Avastin 1.0% |
|---|---|
| STARTED | 24 |
| COMPLETED | 20 |
| NOT COMPLETED | 4 |
Analysis population for the primary outcome, as defined in the registry: Participants who received investigational treatment were analyzed.
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 | Topical Avastin 1.0% |
|---|---|---|
| Participants analysed | 24 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 14 |
| Age, Categorical: >=65 years | Participants | 10 |
| Age, Continuous | years | 52.5 (SD 3.3) |
| Sex: Female, Male: Female | Participants | 8 |
| Sex: Female, Male: Male | Participants | 16 |
| Region of Enrollment: United States | participants | 24 |
6. Primary Endpoint
The registry lists the primary outcome as The Size and Extent of Corneal Neovascularization Will be Measured by Computerized Image Analysis of Corneal Photographs Taken Throughout the Study., measured over Six Months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | percent change since baseline |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the percent change since baseline in the size and extent of corneal neovascularization, measured from corneal photographs through computerized image analysis. The registry identifies the measure as a mean with standard deviation and gives six months as the timeframe. It says baseline photographs were compared with photographs taken at follow-up visits.
As a general statistical concept, an image-based continuous endpoint can represent change in a feature that may be difficult to capture with a simple yes-or-no outcome. A mean describes the average change across the participants included in an analysis, while a standard deviation describes variation among their measurements. Neither statistic is reported for this outcome in the registry record, so the direction, magnitude, and variability of the observed changes cannot be assessed from the available result information.
Baseline comparison is relevant because participants can differ in their initial extent of disease. Measuring change from baseline focuses the endpoint on how the recorded image measure shifts over follow-up. That does not, by itself, distinguish change associated with treatment from change that might occur for other reasons. In a single-group design without a control comparison, the endpoint can describe observed change among analyzed participants but cannot establish a comparative treatment effect.
The registry does not report additional details about the image-analysis procedure, measurement reliability, or the exact handling of photographs from different follow-up visits. These details matter when interpreting a continuous image-derived measure, because the precision and consistency of measurement affect how confidently small or variable changes can be understood.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and design but does not provide a numerical result for the primary endpoint. Although the outcome is defined as mean percent change from baseline in the size and extent of corneal neovascularization, the observed mean and standard deviation are not reported in the registry record. No confidence interval or test result is reported, and the record lists no analyses. Consequently, the posted information does not allow readers to judge the direction, magnitude, or statistical precision of the primary outcome.
The design also shapes what any observed change could mean. This was a single-group study with no masking and no like-for-like control group. A baseline-to-follow-up change among treated participants is a within-group observation. In general, a within-group change can reflect the intervention, but it can also reflect other influences, including the course of the condition, measurement variability, or changes over time. Without a control comparison, those possibilities cannot be separated using the design described in the registry record.
The record reports enrollment of 24, while the primary population is described as participants who received investigational treatment. It does not state how many participants contributed usable photographs or primary-outcome measurements. Enrollment therefore should not be treated as the denominator for the primary endpoint without further information. The registry does not report the amount or pattern of missing outcome data, or the approach used to address missing measurements.
The safety record lists 24 participants at risk for serious adverse events and 0 affected; it also lists 24 at risk for other adverse events and 0 affected. These entries describe recorded counts in the registry. They do not provide a detailed account of event collection, follow-up completeness, or the specific definitions applied. The deaths field is null, which means deaths are not reported in the registry record rather than establishing a count of zero.
For these reasons, the record supports a description of the study’s objective, intervention, design, endpoint, and listed safety counts, but not a conclusion about the size or precision of an efficacy result. No comparative effect estimate is available, and the registry information does not establish whether an observed change would be attributable to treatment. The absence of a posted primary analysis is especially important: a study question and measurement plan are not themselves evidence of an outcome.
9. Statistical Methods Explained
Statistical interpretation depends on the kind of outcome, the study design, and what analysis results are available. The primary measure in this record is continuous: percent change from baseline in the size and extent of corneal neovascularization. The registry characterizes it using a mean and standard deviation. However, the numerical mean and standard deviation are not reported in the registry record, and no analysis is listed. Statistical methods can explain what such summaries would mean, but they cannot recover the unreported result.
What does a mean describe?
As a general explanation, a mean is an average across the measurements included in an analysis. For this study, it would summarize the percent changes recorded for the participants included in the primary-outcome analysis. Its interpretation depends on the analysis population and the measurements available. The registry says participants who received investigational treatment were analyzed, but does not state how many had evaluable photographs or how missing observations were handled. Enrollment of 24 is not necessarily the same as the number contributing to an endpoint analysis.
Why is a standard deviation useful?
In general, a standard deviation describes how spread out individual measurements are around their mean. It helps distinguish a mean change shared relatively consistently across participants from a mean that combines more varied individual changes. A standard deviation is not a confidence interval: it describes dispersion in the observed measurements, whereas a confidence interval expresses uncertainty about an estimated quantity under an analysis model. Neither the standard deviation nor a confidence interval for the primary outcome is reported in the registry record.
What would a confidence interval add?
As a general statistical concept, a confidence interval gives a range of values compatible with the observed data and the method used to calculate it. Its width reflects precision: a narrower interval indicates greater precision under the assumptions of that method, while a wider interval indicates less. A confidence interval would help readers assess plausible values for the average change, not simply whether a test crosses a significance threshold. No primary-outcome confidence interval is reported, so the precision of the estimated change cannot be assessed.
What can a single-group change show?
A comparison of follow-up measurements with baseline can describe change over time among participants who were treated and analyzed. In general, it does not provide the same causal information as a controlled comparison. Without a control group, there is no concurrent estimate of what would have happened without the intervention or under a different intervention. Natural course, measurement conditions, and other influences can contribute to within-group change. The registry identifies this study as single-group and does not list a comparator.
Allocation is listed as NA and masking as none. These design details are distinct from the statistical summary: they do not determine the observed mean, but they affect how findings should be interpreted. In particular, an unmasked, single-group design does not support a direct treatment-versus-control estimate. The posted information gives no adjusted analysis or comparative estimate.
What do the planning illustrations represent?
The record describes planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not a description of the trial’s own design; the actual study is single-group. The record also distinguishes participants per arm for ordinary outcomes from total events for time-to-event outcomes. No numerical planning results are reported in the record, and such illustrations would not be observed power or evidence that the trial had a particular power.
More generally, sample-size calculations depend on assumptions about the effect of interest, variability, significance level, allocation, and analysis. A planning illustration asks what sample size would be associated with specified assumptions. It does not show that an effect was observed, and it does not repair the absence of a reported outcome estimate. The registry does not report the trial’s own sample-size justification, so its design rationale should not be inferred from the planning assumptions.
Why does over-dispersion matter in general?
Over-dispersion is a general issue for count data when variation exceeds what a selected probability model assumes. If ignored, uncertainty can be understated and tests or intervals can be poorly calibrated. That concept is not directly the summary method specified for this primary endpoint, which is continuous and reported as a mean with standard deviation. The registry record does not report an over-dispersion analysis, and no such method should be presumed.
Overall, the available statistical information is descriptive of the planned endpoint and design, rather than a reported analysis result. The record gives no numerical primary estimate, dispersion, interval, or test. Without those results and details of the analyzed population, readers cannot evaluate the observed change’s magnitude, variability, or precision.
10. Confidence Intervals for Each Arm
No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.
| Arm | Participants | Mean | Standard deviation | 95% CI for the mean |
|---|---|---|---|---|
| Topical Avastin 1.0% | 20 | 27.900 | 9.200 | 23.594 to 32.206 |
R code and output
arm <- c("Topical Avastin 1.0%")
n <- c(20)
m <- c(27.9)
sp <- c(9.2)
sd <- sp # posted standard deviations
for (i in seq_along(n)) {
se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}
CARM Topical Avastin 1.0% 20 27.900 9.200 23.594 32.20611. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures, but no analyses are recorded. It does not report a multiplicity adjustment or explain how multiple outcomes or analyses were handled. Multiplicity is a general concern when several statistical hypotheses are tested: considering many results can increase the chance that at least one appears noteworthy by chance. The available record is insufficient to determine whether any adjustment was planned or used, so none should be assumed.
Missing-data handling is also not reported in the registry record. The primary population is described as participants who received investigational treatment, but the record does not state how many had primary-outcome photographs available, how missing follow-up measurements were treated, or whether any imputation or sensitivity analysis was performed. These details affect the interpretation of a mean change because the analyzed participants may not represent everyone enrolled if outcome data are incomplete.
The registry does not describe an intention-to-treat analysis or another named analysis population. Its brief population description should be read as written, without assuming that every enrolled participant contributed to the endpoint. Enrollment is reported as 24, but the endpoint denominator is not reported. The record also does not report interim analyses, a statistical analysis plan, or a sample-size justification. Their presence, absence, and methods cannot be inferred from the listed study characteristics.
12. 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 |
|---|---|---|---|
| Topical Avastin 1.0% | 0 | 24 | 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 |
|---|---|
| Topical Avastin 1.0% | 0 of 24 |
The registry lists 24 participants at risk for serious adverse events in the Topical Avastin 1.0% group and 0 affected. For other adverse events, it also lists 24 at risk and 0 affected. These are the counts recorded in the registry and should be read as such; they do not substitute for a full description of how events were collected or what follow-up was available.
No serious adverse-event terms are listed. The deaths field is null, so deaths are not reported in the registry record; null should not be interpreted as a reported zero. The record does not give event-level descriptions, severity gradations beyond the serious/other categories, or additional context for the safety counts.
As a general statistical point, an observed count of zero does not prove that an event cannot occur. Safety estimates are affected by the amount and duration of observation and by how events are identified and reported. The registry gives the listed at-risk counts but does not provide the follow-up detail needed to assess those issues fully. These counts are descriptive and do not establish a broader safety profile.
13. What the Result Does, and Does Not, Mean
The registry record identifies a completed Phase 1 study of topical bevacizumab for corneal neovascularization and specifies a photographic measure of change from baseline. It also records a single-group design, no masking, enrollment of 24, and safety counts showing 0 affected for serious adverse events and 0 affected for other adverse events among the listed at-risk group. These registry entries describe the study and posted record.
The record does not report the primary outcome’s mean percent change, standard deviation, confidence interval, or statistical test. It also lists no analyses. Therefore, it does not show the magnitude or precision of the measured change. It cannot support a conclusion about whether the measured outcome changed, how large any change was, or how consistent it was across analyzed participants.
Even if a within-group change estimate were available, the design would not provide a direct comparison with a control group. In general, change from baseline in a single group cannot isolate the effect of an intervention from other influences over time. The absence of a comparator is particularly important when interpreting a change in a clinical measure.
The safety entries likewise require a limited reading. They are counts recorded for the listed categories and at-risk group, not proof that adverse events are impossible. The deaths field is not reported, and details of event collection and follow-up are not included in the registry information.
In short, the registry documents the question, intervention, design, endpoint definition, and selected safety counts, but leaves the efficacy result and its statistical uncertainty unreported. The design and record should not be mistaken for evidence of a treatment effect or for a complete account of safety.
14. Why This Trial Matters Statistically
This study is statistically informative as an example of how an endpoint and a design must be considered together. It defines a continuous, image-based outcome as percent change from baseline in the size and extent of corneal neovascularization, assessed at six months. That definition clarifies what was intended to be measured, while the single-group design limits what such a measurement can establish about cause.
The distinction between a within-group change and a comparative effect is central. A mean change can describe the experience of participants with available measurements, but a control group is needed to make a direct comparison against an alternative course. Because the registry lists no comparator, any unreported change cannot be interpreted as a controlled treatment effect.
The record also illustrates why statistical completeness matters. A mean without a measure of variability or uncertainty would be difficult to judge; in this record, the mean, standard deviation, confidence interval, and test result are all unreported. The registry lists two outcome measures but no analyses, and does not describe missing-data handling or the analysis population in sufficient detail to establish an endpoint denominator.
The listed safety counts offer a separate lesson in cautious interpretation. Zero recorded events in the listed categories is a description of the posted data, not a guarantee about risk. The null deaths entry is not a reported zero. Clear distinctions between zero, missing, and not reported are essential when reading registry records.
15. 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 primary limitation for interpreting efficacy is that the registry record does not report the primary-outcome estimate or any associated statistical analysis. Although the outcome, measure, and six-month timeframe are described, there is no reported mean percent change, standard deviation, confidence interval, or test result. The record lists no analyses, leaving no numerical efficacy result to interpret.
The design is single-group, with allocation listed as NA and masking as none. No like-for-like control comparison is reported. Thus, the design cannot distinguish an intervention-associated change from other sources of change over time. This limitation applies even if a within-group endpoint result were available.
The registry reports enrollment of 24 but does not report the number contributing primary-outcome data. It describes the analyzed population as participants who received investigational treatment, without giving an endpoint denominator or explaining missing photographs and follow-up measurements. The handling of missing data is not reported in the registry record.
Further limitations include the absence of reported measurement-reliability information, detailed image-analysis procedures, multiplicity handling, an analysis plan, interim-analysis information, and a sample-size justification. These items should not be presumed to have been present or absent in the conduct of the study; their status is not reported in the registry record.
Safety interpretation is also limited by the concise registry entries. The at-risk counts and affected counts are reported, but event definitions, collection methods, and follow-up detail are not. The deaths field is null, not a reported zero. Together, these omissions mean the listed safety counts should be interpreted narrowly as registry entries rather than a comprehensive safety evaluation.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Topical Avastin for Treatment of Corneal Neovascularization — NCT00559936.
- Registry record last updated 2022-04-29; status last verified 2022-04.
- Results first posted 2012-09-24.
- 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.
19. Record Summary
The record describes a completed Phase 1, single-group study of Topical Avastin 1% for corneal neovascularization, sponsored by Massachusetts Eye and Ear Infirmary. Enrollment is reported as 24. Allocation is listed as NA, and masking as none. The arm description says each patient would receive topical Avastin in one eye.
The primary endpoint is mean percent change from baseline in the size and extent of corneal neovascularization, assessed through computerized analysis of corneal photographs at six months. The registry record does not report the primary-outcome estimate or a statistical analysis; it lists no analyses. Safety entries list 0 serious adverse events and 0 other adverse events among 24 at risk in each category. Deaths are not reported in the registry record.