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 Indocyanine Angiographic Changes of Choroidal Neovascularization by Ranibizumab (NCT01810042), a na phase 4 study. The primary outcome is Caliber of Choroidal New Vessel (CNV).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01810042 |
| Phase | Phase 4 |
| Condition | Exudative Age-related Macular Degeneration |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 49 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pusan National University Hospital |
| Results posted | Yes |
2. Clinical Question
Population
Patients with exudative age-related macular degeneration and choroidal neovascularization (CNV). The study description focuses on eyes in which the vascular structure was clearly visible on indocyanine green angiography (ICGA). The registry record reports 49 enrolled participants and says 31 patients were included in the baseline analysis.
Intervention
Ranibizumab injected into the vitreous cavity. The described regimen is 0.5mg monthly 3 times for the 3 months, followed by pro-re-nata (PRN) treatment for the following 3 months.
Comparator
There is no separate comparator group. The registry lists a single-group study with allocation recorded as NA and no masking. Any interpretation must therefore be about the observed course in the treated group, not a randomized treatment contrast.
Primary question
At 6 months, what is the caliber of the largest CNV vessel, measured in micrometers from ICGA images? The registry describes a method for measuring vessel caliber, but the endpoint result is not reported in the registry record. The stated motivation is that vascular structure may provide information about recurrence; the registry does not report a recurrence outcome.
The clinical focus is an imaging measure of CNV structure following ranibizumab treatment, rather than a comparison of treatments. Secondary outcomes listed in the registry are CNV lesion size, visual acuity in ETDRS letters, and visual acuity changes. Their numerical results are not reported in the registry record, so the record does not establish how those measures changed during the study.
3. Trial Design
This is a prospective, interventional case series listed as a phase 4 study. It is a single-group study, with allocation recorded as NA and masking recorded as none. The study enrolled 49 participants and is marked completed. Its listed condition is exudative age-related macular degeneration, and the sponsor is Pusan National University Hospital.
The intervention is ranibizumab. The registry describes an injection of 0.5mg into the vitreous cavity monthly 3 times for the 3 months, then PRN for the following 3 months. The primary endpoint is assessed at 6 months. The registry record lists the study start as 2010-10 and primary completion as 2013-02.
As a single-group case series, the design can describe measurements observed among participants receiving the intervention. It does not include an untreated or alternative-treatment group for estimating a between-group effect. Changes over time could be described if the measurements and analysis were reported, but the registry record does not report the numerical primary endpoint result or a statistical analysis of it.
The baseline analysis included 31 patients. The primary endpoint description also notes that 7 patients had the thickest vessels under the limitation of measurement. The record does not explain how this measurement limitation affected the analysis or whether the baseline analysis population was used for the endpoint assessment. A protocol, sample-size justification, interim analysis plan, and prespecified statistical analysis plan are not reported in the registry record.
The study lists 4 outcome measures in total: the primary CNV caliber measure and secondary measures for lesion size and visual acuity. The registry record lists no analyses. Without reported outcome values and analysis details, the design can be characterized, but the magnitude and precision of any observed change cannot be assessed from the registry record.
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: This study has a single arm, and there was no assignment process.
Overall Study
| Stage | Ranibizumab |
|---|---|
| STARTED | 49 |
| COMPLETED | 39 |
| NOT COMPLETED | 10 |
Reasons for not completing this period:
| Reason | Ranibizumab |
|---|---|
| Lost to Follow-up | 10 |
Analysis population for the primary outcome, as defined in the registry: Of 31 patients included in the baseline analysis, 7 patients had the thickest vessels under limitation of measurement.
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.
Population: Of 39 patients completed the study, 8 patients were excluded because 6 patients had polypoidal choroidal vasculopathy, and 2 patients had poor quality of indocyanine green angiography.
| Characteristic | Unit | Ranibizumab |
|---|---|---|
| Participants analysed | 31 | |
| Age, Continuous | years | 69 (51 to 85) |
| Sex: Female, Male: Female | Participants | 12 |
| Sex: Female, Male: Male | Participants | 19 |
| Region of Enrollment: Korea, Republic of | participants | 31 |
| Median visual acuity (ETDRS) | ETDRS letter | 46 (0 to 81) |
| Central subfield macular thickness (um) | micrometer | 317 (168 to 793) |
6. Primary Endpoint
The registry lists the primary outcome as Caliber of Choroidal New Vessel (CNV), measured over 6 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Full Range |
| Unit | micrometer |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the caliber of the largest choroidal new vessel (CNV), expressed in micrometers and assessed at 6 months. The registry describes measuring vessel caliber from an indocyanine green angiography (ICGA) image. The image is processed by inverting black and white; the CNV arteriolar course is indicated manually in IVAN software, and the software calculates the average thickness of the vascular segment.
As a general statistical and clinical concept, vessel caliber is a structural imaging measure. It may characterize the appearance of the CNV vascular network, which the registry describes as potentially informative about recurrence. That rationale does not make caliber itself a recurrence measure: recurrence is not listed as an outcome in the registry record.
The endpoint uses the mean as its summary parameter and full range as its stated dispersion. A mean describes the average measured value, while a full range describes the span from the smallest to the largest recorded value. The registry does not report the mean, full range, number of endpoint measurements, or any confidence interval. It also notes measurement limitations for the thickest vessels in 7 patients. The effect of that limitation on the endpoint estimate is not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a prospective case series in which ranibizumab was given to participants with exudative age-related macular degeneration and the primary outcome was CNV vessel caliber at 6 months. It does not report the numerical caliber result. Consequently, the amount or direction of any change in vessel caliber cannot be established from the record. No estimate of precision, such as a confidence interval, is reported either.
The registry describes the endpoint as a mean with full-range dispersion. Those summaries would describe the distribution of measured vessel calibers, but they are not present in the record. There are also no posted p-values or analysis results: the registry lists no analyses. The record therefore does not support a conclusion about whether the measured caliber changed over time or how consistent the measurements were across participants.
The study has no separate comparison group. This matters for interpretation because an observed change in a single group, even if it had been reported, would not by itself distinguish an intervention-associated change from other explanations such as natural variation, changes in measurement, or factors that changed over time. Those are general considerations for uncontrolled studies, not findings about this study. A randomized comparison is not part of this design.
The stated motivation is that CNV vascular structure may provide information regarding recurrence. However, recurrence is not listed among the outcomes, and no recurrence results are reported in the registry record. The primary endpoint should therefore be read as an imaging measurement, not as evidence that recurrence was predicted or prevented. The listed secondary outcomes concern lesion size and visual acuity; their results are also not reported.
The baseline analysis included 31 patients, and the endpoint description says 7 patients had the thickest vessels under measurement limitation. The record does not state how many measurements contributed to the six-month endpoint, how those limitations were handled, or whether the endpoint analysis used the same participants as the baseline analysis. Without that information, the completeness and comparability of measurements cannot be assessed.
Overall, the registry supports a description of the research question, treatment schedule, and imaging method, but not a quantitative interpretation of efficacy or change. The sample-size illustrations associated with an observed effect cannot be grounded in this record because no observed endpoint effect is reported. Such planning illustrations, when available, are not observed power and do not substitute for a reported estimate and its uncertainty.
9. Statistical Methods Explained
The registry record describes the endpoint and design, but does not report the statistical analysis methods or numerical outcome estimates. The points below explain how the listed design and summaries are generally interpreted, without implying that particular analyses were conducted in this study.
What a single-group design estimates
In a single-group study, measurements can describe the participants who received the intervention. If measurements are collected at multiple times, a within-group change can be summarized. Such a change is not the same as a treatment effect estimated against a comparator. General statistical reasoning is that other changes over time and measurement variability can contribute to a within-group difference. Because this study has no separate comparator, it cannot directly estimate a between-group treatment contrast.
What the mean and full range convey
The registry identifies the primary endpoint parameter as a mean and its dispersion as the full range. The mean is a measure of the center of a set of values. The range reports the minimum and maximum, giving a sense of the spread but not showing how observations are distributed between those extremes. A range can be sensitive to unusually small or large observations. As a general matter, a mean and range alone do not provide all the information needed to judge precision; a confidence interval or other uncertainty estimate would help convey how uncertain an estimated mean is. No such estimate is reported in the registry record.
Why imaging measurement matters
The endpoint is derived from ICGA images using a semiautomatic software method. The vessel course is indicated manually, after the image is processed by inverting black and white, and the software calculates average thickness for the indicated segment. General measurement principles apply: results can depend on image quality, the selection of the vessel segment, and the consistency of the person tracing it. The registry describes the procedure but does not report reader blinding, repeatability, or a reliability assessment. These details are not reported in the registry record.
The endpoint description notes a measurement limitation for the thickest vessels in 7 patients. This is relevant because a measurement constraint can affect the values recorded and the population represented by an analysis. The registry does not state whether these observations were excluded, adjusted, treated as censored, or handled in another way. No particular handling method should be assumed.
Why confidence intervals and p-values are different
In general, a confidence interval gives a range of values compatible with the data and the assumptions of the statistical model. It conveys the precision of an estimate, whereas a p-value summarizes how compatible the observed data are with a specified null hypothesis under a model. Neither replaces the estimated effect itself, and neither establishes clinical importance. The registry record does not report a confidence interval, p-value, or test for the primary endpoint.
For an uncontrolled case series, a test of change from baseline, if conducted, would address a within-group question. It would not answer the same question as a randomized comparison. A statistical test can also be difficult to interpret without the estimate, measurement details, and analysis population. The registry record lists no analyses, so no test or model can be attributed to this study.
Why over-dispersion can matter in general
Over-dispersion describes more variability in observed data than a simple statistical model expects. In general, it can make standard errors too small if ignored, causing uncertainty to be understated. This concept is especially relevant to some count or clustered outcomes. The primary outcome is a vessel-caliber measurement summarized as a mean, and the registry does not report an analysis model or a finding of over-dispersion. It would therefore be inappropriate to claim that over-dispersion was observed or that a particular correction was used.
The registry mentions a sample-size illustration based on an observed effect and specifies assumptions for that planning exercise. A planning illustration is a way to explore the participant count that might be needed to detect an assumed effect under stated conditions. It is not a record of the study's actual design justification and does not show the probability that the completed study detected an effect. The registry record does not report a sample-size justification for this trial, and no observed primary effect is reported to anchor such an illustration.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 4 outcome measures: the primary measure of CNV vessel caliber and secondary measures of CNV lesion size, visual acuity in ETDRS letters, and visual acuity changes. The registry record does not report numerical results for these outcomes or analyses of them. It also does not report a statistical analysis plan specifying which outcomes or time points were designated for formal testing.
Multiplicity is a general statistical issue that arises when several hypotheses or outcomes are tested. As the number of tests increases, the chance of at least one apparently notable result can increase unless the analysis accounts for the testing strategy. This does not establish that multiplicity affected this study; the registry does not report whether formal tests were conducted or whether any adjustment was planned or used.
Missing-data information is limited. The record reports 49 enrolled participants and says 31 patients were included in the baseline analysis. It also notes measurement limitations for the thickest vessels in 7 patients. It does not give the number with a six-month endpoint measurement, reasons for absent measurements, or a missing-data method. It is therefore not possible to determine whether the primary result represents all enrolled participants or a subset.
The analysis population is not defined beyond the description of the baseline analysis. The registry does not say whether analyses followed an intention-to-treat principle, used a per-protocol population, or included another specified set of participants. It also does not report how deviations from the treatment schedule were handled. These details are not reported in the registry record and should not be inferred from the enrollment count or intervention description.
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 |
|---|---|---|---|
| Ranibizumab | 2 | 49 | 4.1% |
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 |
|---|---|
| Ranibizumab | 0 of 49 |
Most frequent serious adverse event terms
| Event term | Organ system | Ranibizumab |
|---|---|---|
| Cerebral infarction | Nervous system disorders | 1 of 49 |
| Liver cirrhosis | Gastrointestinal disorders | 1 of 49 |
Serious events with the highest arm-level rate: cerebral infarction (2.0%), liver cirrhosis (2.0%).
The registry reports 2 serious adverse events in the ranibizumab group, with 49 participants at risk. The serious event terms listed are cerebral infarction and liver cirrhosis, each reported as 2.0%. The record reports 0 other adverse events affecting participants in this group, with 49 at risk. Deaths are not reported in the registry record.
These figures describe the events recorded during the study; they do not, by themselves, establish whether an event was caused by the intervention. A serious adverse event is a safety reporting category, not a determination of treatment causality. The registry does not provide case-level descriptions, timing, duration, investigator assessments of relatedness, or a comparison group for judging whether event frequency differed from another treatment or background risk.
Because the study has one group, the serious event figures are descriptive. They should not be interpreted as a comparative safety estimate. The registry also does not report how adverse events were monitored over time or whether all participants completed the same period of safety follow-up. The event counts therefore need to be read alongside the limited design and reporting information, rather than as a causal conclusion.
12. What the Result Does, and Does Not, Mean
The record documents a prospective, single-group investigation of ranibizumab and ICGA-based measurement of CNV vessel caliber. It gives the treatment schedule, endpoint definition, and measurement approach. It also identifies secondary outcomes relating to CNV lesion size and visual acuity. These details clarify what the study set out to examine.
The record does not report the numerical primary endpoint result, secondary outcome results, or statistical analyses. It therefore does not show whether vessel caliber, lesion size, or visual acuity changed. It also does not provide an estimate of the size of any change or its precision. The study's stated interest in vascular structure as a possible source of information about recurrence should not be mistaken for a reported recurrence result; recurrence is not listed as an outcome.
Even if a within-group change were available, a case series without a comparator would describe change among treated participants rather than isolate the effect of treatment from other influences. That is a general limitation of the design, not evidence that any particular alternative explanation occurred. There is no randomized treatment contrast in this study.
The enrollment count and the baseline analysis count are not interchangeable. The registry reports 49 enrolled participants and a baseline analysis of 31 patients, but does not explain the difference or state how many contributed to the six-month endpoint. The measurement limitation noted for 7 patients adds uncertainty about how the thickest vessels were represented. The registry record does not describe the impact of that limitation on the analysis.
Accordingly, the record is informative about the study question and methods, but it does not support a quantitative conclusion about efficacy, the magnitude of change, or the precision of an estimate. The serious adverse event entries are descriptive and do not establish causation or comparative safety.
13. Why This Trial Matters Statistically
This study is statistically notable for focusing on a structural imaging measure within a prospective case series. CNV vessel caliber is defined through a particular ICGA processing and measurement procedure, rather than through a broad clinical outcome. Such a design highlights the importance of clear operational definitions: image processing, manual tracing, and software-based thickness calculation all contribute to how the endpoint is obtained.
The record also makes visible a practical measurement concern: the thickest vessels were subject to measurement limitation in 7 patients. For quantitative interpretation, it matters whether such limitations affect the values, availability of measurements, or inclusion in the analysis. The registry describes the concern but does not report its handling. This is a useful reminder that measurement constraints are part of the statistical problem, not merely technical details.
The single-group design defines what the study can estimate. It can describe observations among participants receiving ranibizumab, but it does not provide a randomized contrast. This distinction matters when separating a measured trajectory from a causal treatment effect. No result is reported in the registry record, so the structural endpoint cannot be quantitatively evaluated from this record.
Finally, the record illustrates the difference between a study's motivation and its measured outcomes. The rationale connects vascular structure with recurrence, while the registered primary outcome is vessel caliber and recurrence is not listed as an outcome. Keeping those concepts separate prevents an imaging measure from being interpreted as a direct recurrence result. Complete reporting of outcome values, analysis populations, uncertainty, and measurement handling would be needed to assess the endpoint statistically.
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 most important limitation for interpreting results is that the numerical primary endpoint estimate is not reported in the registry record. No uncertainty estimate or analysis result is reported either. The secondary outcome values are also absent, so the record does not permit an assessment of the magnitude or direction of any outcome change.
The study has no separate comparator group and is not a randomized comparison. This restricts interpretation to a description of the treated group and does not support a direct estimate of a between-group effect. The absence of masking is recorded, but the registry does not describe whether image assessors were masked or how measurement consistency was assessed.
The endpoint is based on image processing and a manually indicated vessel course, with software calculating average vessel-segment thickness. The record notes that 7 patients had the thickest vessels under measurement limitation, but does not describe the consequences for their measurements or the analysis. Measurement reproducibility, image quality criteria, and procedures for resolving uncertain traces are not reported in the registry record.
The registry reports 49 enrolled participants and a baseline analysis including 31 patients, without explaining the analysis-population definition or reporting the number with a six-month endpoint measurement. Reasons for missing measurements and methods for handling missing data are not reported. The record also does not describe protocol deviations or adherence beyond the planned treatment schedule.
Further limitations include the absence of a reported statistical analysis plan, sample-size justification, or multiplicity strategy. These details are not reported in the registry record. The registry's motivation mentions possible information about recurrence, but recurrence is not a listed outcome. Thus the record does not establish whether vessel caliber is associated with recurrence in this study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Indocyanine Angiographic Changes of Choroidal Neovascularization by Ranibizumab — NCT01810042.
- Registry record last updated 2016-05-04; status last verified 2016-03.
- Results first posted 2014-04-07.
- 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
NCT01810042 is a completed, prospective, interventional, single-group case series of ranibizumab for exudative age-related macular degeneration. The record reports 49 enrolled participants and identifies Pusan National University Hospital as the sponsor. The described regimen is 0.5mg injected into the vitreous cavity monthly 3 times for the 3 months, then PRN for the following 3 months.
The primary outcome is mean caliber of the largest CNV vessel, measured in micrometers at 6 months from ICGA images using a semiautomatic software procedure. The baseline analysis included 31 patients; the record notes measurement limitation for the thickest vessels in 7 patients. Secondary outcomes concern CNV lesion size and visual acuity.
Numerical outcome results, statistical analyses, and analysis-population details are not reported in the registry record. The record lists 2 serious adverse events, cerebral infarction and liver cirrhosis, each reported as 2.0%; deaths are not reported in the registry record.