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 High-Dose Lucentis (Ranibizumab 2.0mg) for the Treatment of Nonproliferative Idiopathic Parafoveal Telangiectasia (NCT01205035), a randomized phase 2 study. The primary outcome is Visual Acuity Change From Baseline to Month 12 of the Study.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01205035 |
| Phase | Phase 2 |
| Condition | Retinal Diseases, Telangiectasis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 6 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Eye Center of Northern Colorado, P.C. |
| Results posted | Yes |
2. Clinical Question
Population
The registry describes people with nonproliferative idiopathic parafoveal telangiectasia, also called idiopathic perifoveal or juxtafoveal telangiectasia and Macular Telangiectasia. The condition is described as having an unknown cause and as bilateral, though not always symmetric. The registry record does not report detailed eligibility criteria or participant characteristics, so the population represented by the enrolled participants cannot be characterized further.
Intervention
The intervention was intravitreal ranibizumab. The arm description says the initial dose was 2.0mg and was changed to 1.0mg near the conclusion of the study. The registry record does not report the exact timing of the change, the injection schedule, or the number of injections received by each participant.
Comparator
The comparison group was observation, with no treatment given. The trial used a parallel design, so the registry presents separate arms rather than a within-participant comparison. The posted primary outcome includes one participant in the observation arm and three in the ranibizumab arm.
Primary question
The primary question concerned change in visual acuity from baseline to month 12, measured in LogMAR units. The registry posts a mean change of 0.1 for observation and -0.05 for ranibizumab, with full ranges of 0.1 to 0.1 and -0.12 to 0.0, respectively. These are descriptive arm summaries; the registry record does not report a statistical comparison or an estimate of uncertainty for the difference between arms.
The trial was registered as a phase 2 study of ranibizumab 2.0mg for this condition. Its posted results should be read as a small, descriptive record rather than as a precise estimate of a treatment contrast. The registry lists enrollment of 6, while the primary outcome summaries include 1 participant in the observation arm and 3 in the ranibizumab arm. How the remaining enrolled participants relate to the posted primary outcome is not reported in the registry record.
3. Trial Design
This was a randomized, open-label, parallel phase 2 trial sponsored by Eye Center of Northern Colorado, P.C. The registry reports enrollment of 6 and a completed status. The recorded study dates are 2010-10 for start and 2012-10 for primary completion. The two groups were observation, with no treatment, and intravitreal ranibizumab 2.0mg. The ranibizumab arm description specifies that the initial dose was switched to 1.0mg near the study’s conclusion.
Random allocation is intended to assign participants to groups by chance, rather than by clinician or participant choice. In general, randomization can reduce systematic differences between groups, but it does not guarantee comparable groups, particularly when the number of participants is very small. The method used to generate the allocation sequence, conceal allocation before assignment, and handle any changes after randomization is not reported in the registry record.
The trial was not masked. Participants and study personnel therefore knew the assigned group, according to the registry’s masking information. Visual acuity is assessed through a measurement procedure, but knowledge of assignment can still affect behavior, assessment, or other aspects of trial conduct. The registry record does not report whether outcome assessors were masked separately from participants or other personnel.
The primary endpoint was visual-acuity change from baseline to 12 months, in LogMAR units. Listed secondary outcomes cover visual-acuity change at month 6 and month 9; central subfield thickness measured by optical coherence tomography at 6, 9, and 12 months; adverse events associated with ranibizumab 2.0mg; and angiographic leakage at month 6 and month 12. Results for these secondary outcomes are not reported in the registry record. The registry does not describe the planned analysis population, follow-up procedures, or handling of participants who did not contribute outcome measurements.
Although the trial is marked completed, the posted results give limited information for evaluating how the planned study proceeded. The registry reports an enrollment total of 6, whereas the primary outcome arm summaries contain 1 and 3 participants. Reasons for that difference, if any, are not reported in the registry record. No protocol details beyond the recorded design and outcome descriptions should be inferred.
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 | Observation | Intravitreal Ranibizumab 2.0mg |
|---|---|---|
| STARTED | 3 | 3 |
| COMPLETED | 1 | 3 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Observation | Intravitreal Ranibizumab 2.0mg |
|---|---|---|
| Withdrawal by Subject | 1 | 0 |
| Physician Decision | 1 | 0 |
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 | Observation | Intravitreal Ranibizumab 2.0mg | Total |
|---|---|---|---|---|
| Participants analysed | 3 | 3 | 6 | |
| Age, Continuous | years | 68 (52 to 76) | 65 (52 to 76) | 66.5 (52 to 76) |
| Sex: Female, Male: Female | Participants | 2 | 2 | 4 |
| Sex: Female, Male: Male | Participants | 1 | 1 | 2 |
| Region of Enrollment: United States | participants | 3 | 3 | 6 |
6. Primary Endpoint
The registry lists the primary outcome as Visual Acuity Change From Baseline to Month 12 of the Study, measured over Baseline to 12 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Full Range |
| Unit | LogMAR Unit |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the change in visual acuity from baseline to month 12, expressed in LogMAR units. It is intended to describe how visual acuity changed over the stated follow-up interval in each study arm. The registry reports the endpoint as a mean with full range, rather than as a standard deviation, standard error, or confidence interval.
Visual acuity is a functional measure relevant to a disorder affecting the parafoveal and macular region. A change score uses each participant’s baseline measurement as a reference and describes the subsequent measurement relative to that starting point. Interpreting the direction of a change requires understanding the scale and how change was coded. The registry gives the unit as LogMAR but does not provide additional detail about the scoring or calculation of change.
The posted mean change is 0.1 in the observation arm, with a full range from 0.1 to 0.1, based on 1 participant. In the ranibizumab arm, the mean change is -0.05, with a full range from -0.12 to 0.0, based on 3 participants. These summaries describe the observed measurements in the participants included in the endpoint results. They do not by themselves estimate the uncertainty of an arm-to-arm contrast or establish whether a difference is attributable to assignment.
The registry does not report a confidence interval, hypothesis test, or clinical threshold for interpreting the change. It also does not report the individual baseline and follow-up measurements. The endpoint therefore supports only a limited descriptive reading of the posted arm summaries.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports a mean visual-acuity change of 0.1 LogMAR units for observation and -0.05 LogMAR units for intravitreal ranibizumab 2.0mg at month 12. The corresponding full ranges are 0.1 to 0.1 and -0.12 to 0.0. These values are summaries of the endpoint observations, not estimates accompanied by a measure of sampling uncertainty. The record does not report a statistical test or confidence interval for the contrast between groups.
The small numbers contributing to the posted results are central to interpretation: the observation summary has 1 participant and the ranibizumab summary has 3. A single participant determines the entire observation mean and range. In the ranibizumab arm, the mean and range summarize only three participants. Such summaries can be strongly affected by the particular observed values, and the observed spread cannot reliably characterize variation in a wider population. The registry reports enrollment of 6, but does not explain the relationship between that total and the four participants represented in the primary outcome summaries.
A confidence interval, when reported, expresses the statistical uncertainty around an estimate under the assumptions of the analysis. None is posted for this primary endpoint. Without one, readers cannot use the registry results to judge how compatible the observed summaries are with a broad range of possible underlying arm differences. The lack of a reported test also means there is no posted inferential result to interpret. The arm means alone should not be treated as evidence that a difference is statistically reliable.
The registry describes a randomized design, which is useful for reducing systematic allocation differences in expectation. However, randomization cannot ensure balance in a trial with very few participants. The study was also open-label. The registry does not state whether any outcome assessment was masked, and it does not give enough participant-level information to assess baseline comparability or follow-up completeness.
The intervention description adds another interpretive consideration: the initial ranibizumab dose was 2.0mg, then changed to 1.0mg near the conclusion of the study. The timing and participant-level exposure are not reported. The primary outcome is described for the ranibizumab arm as a whole, so the record does not allow changes to be evaluated separately by dose.
Overall, the posted results show the observed mean changes and ranges among the participants included in each arm summary. They do not establish the precision of an arm comparison, clarify what happened to all enrolled participants, or provide results for the listed secondary outcomes. No conclusion about treatment effect should be drawn from these descriptive figures alone.
9. Statistical Methods Explained
Randomization and comparison. In a randomized trial, assignment to study groups is determined by chance. In general, randomization is used to limit systematic differences in prognostic factors between groups, making a causal comparison more credible than a nonrandomized comparison. It does not guarantee that groups are balanced in every characteristic, and chance imbalances remain possible. That concern is especially important when few participants contribute outcomes. The registry identifies the study as randomized but does not report the sequence-generation method, allocation concealment, or stratification factors.
The parallel design means the arms are distinct groups followed under their assigned conditions. The observation group received no treatment; the intervention group received intravitreal ranibizumab, initially at 2.0mg and switched to 1.0mg near the conclusion. In general, an arm comparison should account for the randomized assignment and use a clearly stated analysis population. The registry record does not report the analysis population or a model for the primary comparison.
Open-label conduct. The registry reports no masking. In general, masking can reduce the influence of knowledge about assignment on participant behavior, co-interventions, outcome assessment, and reporting. Its relevance depends on the outcome and how it is measured. The record does not say whether outcome assessors were masked even though participants and study personnel were not, so the extent of any such protection is not reported.
Mean and full range. A mean is the arithmetic average of observed values. A full range gives the smallest and largest observed values; it describes the span of the observations but not how values are distributed within that span. The primary endpoint is reported as a mean with full range. For the observation arm, the registry gives 1 participant and a range of 0.1 to 0.1. For the ranibizumab arm, it gives 3 participants and a range of -0.12 to 0.0. With so few observations, these summaries provide little information about the population distribution or the stability of the means. The record does not report standard deviations, standard errors, or confidence intervals for the arm means.
Uncertainty and inference. General statistical inference distinguishes the observed estimate from uncertainty about the underlying quantity. A confidence interval is a range of estimates compatible with the data under a specified method and its assumptions; it is not a guarantee that the true value lies within the interval. A hypothesis test assesses data against a stated null model. The registry record does not post a confidence interval or test for the primary outcome. No inferential comparison is therefore available to interpret. The descriptive means should not be mistaken for a direct estimate with known precision.
In general, a treatment comparison may be expressed as a difference between group outcomes, with uncertainty calculated using an analysis appropriate to the design and data. The registry gives arm-level means but does not post an arm-difference estimate. No unadjusted recalculation is reported in the registry record. A reader should not infer a numerical contrast or its uncertainty from the posted summaries.
Repeated outcomes and time points. The listed secondary measures include visual acuity at more than one follow-up time, central subfield thickness measured by optical coherence tomography at several times, and angiographic leakage at specified visits. Repeated measurements from the same participant are related rather than independent. In general, an analysis of repeated measurements should account for that within-participant relationship and make clear how missing visits are handled. The registry record does not report models, covariance assumptions, or secondary outcome results.
For count outcomes such as adverse events, interpretation depends on whether the analysis counts participants with at least one event, total events, or event rates over exposure time. These are different quantities. The registry posts numbers of participants at risk and numbers affected for serious and other adverse events, but does not describe event definitions, exposure time, or a comparative statistical method. Those counts can be read descriptively, not as a complete analysis of safety.
Planning calculations. A sample-size calculation is a planning exercise based on assumptions about an effect, variability, significance level, allocation, and analysis. It is distinct from the evidence in the observed trial results. A planning illustration based on an observed effect does not measure the trial’s observed power, and it cannot repair the limited precision of a small study. The registry record does not report the trial’s own sample-size justification or the assumptions used to select its enrollment. Interim analyses and stopping rules are also not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and four secondary outcomes. The secondary outcomes include visual-acuity change at month 6 and month 9, central subfield thickness at several follow-up times, adverse events associated with ranibizumab 2.0mg, and angiographic leakage at month 6 and month 12. Considering multiple outcomes or time points can increase the chance of finding an apparently notable result by chance when many statistical tests are performed. In general, investigators may define one primary comparison, adjust for multiple testing, or treat additional analyses as exploratory. The registry record does not report a multiplicity strategy, hierarchy among secondary outcomes, or planned set of statistical tests.
No analysis results are posted in the registry record. In particular, results for the listed secondary outcomes are not reported in the registry record. The record does not describe whether tests were planned at each time point, how correlated measurements were to be handled, or whether any adjustment was intended. The absence of posted results should not be interpreted as a null result.
The registry reports enrollment of 6, while the primary outcome summaries include 1 participant in observation and 3 in ranibizumab. The record does not state why the outcome summaries include those numbers, whether other participants lacked the month-12 measurement, or how missing outcomes were treated. Reasons for missingness, imputation, sensitivity analyses, and the relationship between randomized and analyzed participants are not reported in the registry record.
The analysis population is also not reported in the registry record. It is therefore unclear whether the primary outcome summaries reflect all randomized participants with available measurements, a protocol-defined subset, or another population. The registry does not report baseline values by arm or a prespecified approach to adjustment. These omissions limit assessment of attrition, comparability, and possible bias in the posted endpoint summaries.
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 |
|---|---|---|---|
| Observation | 0 | 3 | 0.0% |
| Intravitreal Ranibizumab 2.0mg | 0 | 3 | 0.0% |
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 |
|---|---|
| Observation | 2 of 3 |
| Intravitreal Ranibizumab 2.0mg | 2 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Observation | Intravitreal Ranibizumab 2.0mg |
|---|---|---|---|
| Elevated Blood Pressure | Cardiac disorders | 0 of 3 | 1 of 3 |
| Sinusitis | Infections and infestations | 0 of 3 | 1 of 3 |
| Cold | Immune system disorders | 1 of 3 | 0 of 3 |
| Food Poisoning | General disorders | 1 of 3 | 0 of 3 |
| Diabetes | Endocrine disorders | 1 of 3 | 0 of 3 |
| Ear Infection | Infections and infestations | 1 of 3 | 0 of 3 |
| Allergies | Respiratory, thoracic and mediastinal disorders | 1 of 3 | 0 of 3 |
| Stiff Neck | Musculoskeletal and connective tissue disorders | 1 of 3 | 0 of 3 |
The adverse-event record lists 3 participants at risk for serious events in each arm and 0 participants affected by serious events in each arm. It lists 3 participants at risk for other adverse events in each arm and 2 participants affected in each arm. No serious-event terms are listed. Death counts are not reported in the registry record.
These are counts of participants, not counts of all event occurrences. A participant may have experienced more than one event, and the posted affected-participant count does not show event severity, duration, timing, or relationship to treatment. The registry does not provide exposure time or further event definitions, so the counts cannot be interpreted as rates over time. Nor do they establish that the groups had equivalent safety: the numbers at risk and affected are very small, and no comparative statistical analysis is posted.
The intervention arm is described as starting at 2.0mg and switching to 1.0mg near the study’s conclusion. The registry does not report which participants received each dose for how long, or whether events occurred before or after the change. The listed secondary outcome refers to adverse events associated with ranibizumab 2.0mg, but the posted adverse-event figures do not provide detailed event-level attribution. The record supports a narrow description of reported counts only, not a complete characterization of safety.
12. What the Result Does, and Does Not, Mean
The primary outcome record gives descriptive mean changes in visual acuity from baseline to month 12. Observation is reported with a mean change of 0.1 LogMAR units and a full range of 0.1 to 0.1 among 1 participant. Intravitreal ranibizumab 2.0mg is reported with a mean change of -0.05 and a full range of -0.12 to 0.0 among 3 participants. These values describe only the posted observations in the analyzed arm summaries.
The record does not report a confidence interval, hypothesis test, or arm-difference estimate. Consequently, the precision of a comparison is not quantified. A difference in observed arm means is not, on its own, evidence that the groups differ reliably or that assignment caused the difference. The registry also does not report an interpretation threshold for the endpoint, so the clinical importance of the observed changes cannot be established from these results alone.
The enrollment total is 6, whereas the posted primary outcome summaries account for 1 participant in observation and 3 in ranibizumab. The reasons for this distinction are not reported in the registry record. Without participant flow, missing-data methods, baseline characteristics, and a stated analysis population, it is not possible to determine how representative the endpoint summaries are of all enrolled participants.
The randomized design is relevant to the question, but its value depends on the allocation process, retention, outcome measurement, and analysis. The trial was open-label, and the registry does not describe whether outcome assessors were masked. In addition, the ranibizumab dose changed from 2.0mg to 1.0mg near the conclusion, with timing and participant-level details not reported. The posted outcome cannot be separated by dose exposure.
The record does not post results for the secondary outcomes, including central subfield thickness, angiographic leakage, and the additional visual-acuity time points. It therefore provides no basis for drawing conclusions about those measures. The appropriate reading is limited: the registry records small-sample descriptive endpoint and event counts, while important information needed for estimation, precision, and interpretation is not reported.
13. Why This Trial Matters Statistically
This trial is statistically instructive because it pairs a randomized design with very sparse posted outcome data. Randomization is an important design feature, but it does not make a small set of observations precise. The primary outcome summaries include 1 participant in observation and 3 in the ranibizumab arm, while enrollment is reported as 6. The registry does not explain the relationship between those counts. The example illustrates why readers need both the design description and the actual analysis population when interpreting trial results.
The registry reports means and full ranges, but no confidence interval, standard error, or test of the primary contrast. A mean can be calculated from observed values, yet its precision depends on the amount and variability of the data. When very few participants contribute, the mean and range offer limited information about what might be expected in a broader population. Descriptive summaries are not substitutes for uncertainty estimates.
The open-label design and dose change are also relevant to interpretation. General concerns about unmasked assessment and changes in exposure cannot be resolved from the posted record because assessor masking and participant-level dosing details are not reported. The registry also lists several secondary outcomes across multiple time points without posting their results or describing a multiplicity strategy. These features demonstrate the importance of prespecified analyses and transparent reporting, without implying that any particular unreported procedure was or was not used.
Finally, the record distinguishes a completed trial from a complete statistical account. It posts a primary outcome summary and adverse-event counts, but not the planned analysis, missing-data approach, sample-size justification, or results for secondary outcomes. The statistical value of the record lies in showing what can be described from these figures and where the limits of inference begin.
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 primary endpoint summaries are based on very few participants: 1 in observation and 3 in the ranibizumab arm. Enrollment is reported as 6, but the registry does not explain why the posted primary outcome counts differ from that total. Participant flow, reasons for non-contribution to the endpoint, and follow-up completeness are not reported in the registry record.
The registry gives arm means and full ranges but no confidence intervals, standard errors, standard deviations, or formal comparison. It does not provide individual outcome values, baseline measurements, or an estimate of the arm difference. The precision and stability of the means therefore cannot be assessed from the posted summaries. The record also does not state a threshold for judging the relevance of a change in LogMAR units.
The study was open-label. Whether outcome assessors were masked is not reported in the registry record. The method for sequence generation and allocation concealment, eligibility criteria, participant characteristics, and baseline balance are also not reported. These details would help assess how randomization was conducted and how applicable the enrolled group may be to the condition described.
The intervention changed from an initial dose of 2.0mg to 1.0mg near the conclusion. Exact timing, injection schedule, and individual exposure are not reported. This limits interpretation of the intervention arm summary as a result for a single, consistently applied dose. The registry does not post results for the listed secondary outcomes or describe the analysis plan, multiplicity handling, missing-data methods, interim analyses, or the trial’s sample-size justification. These limitations restrict both the precision of the primary comparison and the ability to evaluate the broader pattern of outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: High-Dose Lucentis (Ranibizumab 2.0mg) for the Treatment of Nonproliferative Idiopathic Parafoveal Telangiectasia — NCT01205035.
- Registry record last updated 2015-04-10; status last verified 2015-03.
- Results first posted 2015-04-10.
- 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
NCT01205035 is a completed randomized, open-label, parallel phase 2 trial sponsored by Eye Center of Northern Colorado, P.C. It enrolled 6 participants and compared observation with intravitreal ranibizumab, initially at 2.0mg and changed to 1.0mg near the study’s conclusion. The recorded start was 2010-10, and primary completion was 2012-10.
The primary outcome was mean visual-acuity change from baseline to month 12 in LogMAR units. The registry reports a mean of 0.1, with a full range of 0.1 to 0.1, for 1 observation participant, and a mean of -0.05, with a full range of -0.12 to 0.0, for 3 ranibizumab participants. No inferential comparison or uncertainty interval is posted.
Four secondary outcomes are listed, but their results are not reported in the registry record. Serious adverse events affected 0 of 3 participants at risk in each arm; other adverse events affected 2 of 3 in each arm. The record does not report the analysis population, missing-data approach, or reasons the primary summaries include fewer participants than the enrollment total.