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 A Study of Abicipar Pegol in Japanese Patients With Neovascular Age-related Macular Degeneration (NCT02181504), a randomized phase 2 study. The primary outcome is Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02181504 |
| Phase | Phase 2 |
| Condition | Macular Degeneration |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 25 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Allergan |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned Japanese patients with neovascular age-related macular degeneration. The primary endpoint’s analysis population was defined as modified intent-to-treat: randomized and treated patients with at least 1 follow-up visit. The registry record does not report further eligibility details or a separate analysis comparing Japanese and non-Japanese patients.
Intervention
The abicipar pegol groups received 2 mg or 1 mg by intravitreal injection in the study eye at day 1, weeks 4 and 8, followed by a sham procedure at weeks 12 and 16. These are the treatment schedules described in the record; the endpoint results for either schedule are not reported in the registry record.
Comparator
The listed active comparator was ranibizumab 0.5 mg, given by intravitreal injection in the study eye every 4 weeks from day 1 through week 16. Sham procedures were also listed as an intervention and as part of the abicipar pegol schedules. The record does not report a result comparing the treatment groups.
Primary question
The primary endpoint was change from baseline in best corrected visual acuity in the study eye, measured in letters at baseline and Week 16. The study summary describes its broader purpose as assessing safety and efficacy and establishing comparability between Japanese and non-Japanese patients. The registry record does not report a primary endpoint estimate or a direct Japanese–non-Japanese comparison, so it cannot answer whether the groups were comparable on this endpoint.
3. Trial Design
This was a randomized, parallel-group, triple-masked phase 2 study sponsored by Allergan. The registry record lists an enrollment of 25 and identifies macular degeneration as the condition. The study title specifies Japanese patients with neovascular age-related macular degeneration. The record lists abicipar pegol at 2 mg and 1 mg, ranibizumab at 0.5 mg, and sham procedure among the interventions.
The treatment schedules differed. Abicipar pegol was injected into the study eye at day 1, weeks 4 and 8; sham procedures were scheduled at weeks 12 and 16. Ranibizumab was injected into the study eye every 4 weeks from day 1 through week 16. These schedule descriptions identify what the registry says participants were assigned to receive, but they do not establish adherence or describe any departures from the assigned schedules.
The study started in 2014-09, had primary completion in 2015-12, and is listed as completed. The registry record does not report details of the randomization method, allocation concealment, masking procedures, or the roles that were masked. It also does not provide a protocol-level account of visit procedures beyond the schedules summarized for the arms.
The study summary states that the aim was to assess safety and efficacy and establish comparability between Japanese and non-Japanese patients. The listed treatment groups are abicipar pegol and ranibizumab groups; a non-Japanese comparison group or a direct analysis of nationality is not reported in the registry record. Thus, the stated aim should not be mistaken for evidence that a cross-population comparison was conducted or that comparability was demonstrated.
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 | Abicipar Pegol 2 mg | Abicipar Pegol 1 mg | Ranibizumab 0.5 mg |
|---|---|---|---|
| STARTED | 10 | 10 | 5 |
| COMPLETED | 9 | 9 | 4 |
| NOT COMPLETED | 1 | 1 | 1 |
Reasons for not completing this period:
| Reason | Abicipar Pegol 2 mg | Abicipar Pegol 1 mg | Ranibizumab 0.5 mg |
|---|---|---|---|
| Adverse Event | 1 | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Modified Intent-to-Treat: all randomized and treated patients with at least 1 follow-up visit
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 | Abicipar Pegol 2 mg | Abicipar Pegol 1 mg | Ranibizumab 0.5 mg | Total |
|---|---|---|---|---|---|
| Participants analysed | 10 | 10 | 5 | 25 | |
| Age, Continuous | Years | 71.8 (SD 5.75) | 75.6 (SD 7.18) | 76.6 (SD 9.24) | 74.3 (SD 7.10) |
| Sex: Female, Male: Female | Participants | 2 | 1 | 1 | 4 |
| Sex: Female, Male: Male | Participants | 8 | 9 | 4 | 21 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye, measured over Baseline, Week 16.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Letters |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was change from baseline in best corrected visual acuity (BCVA) in the study eye, measured in letters, with assessments at baseline and Week 16. BCVA was measured using an eye chart and recorded as the number of letters read correctly; the scale ranges from 0 to 100 letters. The registry description states that a higher letter count represents better visual acuity. A positive change from baseline means more letters were read correctly, while a negative change means fewer letters were read correctly.
Change from baseline is used to express how an outcome at follow-up differs from its starting value for the same participant. For this endpoint, a group mean change can describe the average direction and size of visual-acuity change in the analyzed group. A standard deviation can describe how much individual changes vary around that average. These are general statistical interpretations, not results from this study.
The registry record identifies the endpoint as a mean with standard deviation, but does not report the group means, standard deviations, or a treatment contrast. It also does not report a single primary endpoint result in the posted results. The endpoint definition therefore explains what was intended to be measured, not what change was observed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies change from baseline in BCVA at Week 16 as the primary endpoint, but it does not report the numerical primary outcome results. No group mean changes, standard deviations, confidence intervals, p-values, or treatment-effect estimates are reported in the registry record. Without those results, the direction, magnitude, and precision of change in each treatment group cannot be assessed from this record.
The record also indicates that the primary outcome has several categories or time points and is not reduced to one comparison. Although the endpoint description names baseline and Week 16, the posted results do not supply a single comparison that can be interpreted as the primary treatment contrast. The registry record does not report why no single comparison is presented or how any possible multiple outcome categories or time points were handled.
There are no listed analyses in the registry record. This does not establish that no analyses were conducted; it means the record does not provide analysis results to evaluate. In particular, it is not possible to determine whether the observed group changes differed, whether any difference was compatible with no effect, or how broad the uncertainty around an estimate might be. No conclusion about treatment efficacy or comparative performance follows from the endpoint definition alone.
The adverse-event entries provide counts of participants at risk and affected, separately for serious and other adverse events. They are descriptive safety information, not substitutes for the missing primary endpoint results. The record lists serious adverse events for the ranibizumab group and none affected in the abicipar pegol groups, but interpretation is limited by the small group counts and the absence of further detail about event timing, attribution, or statistical comparisons. The reported counts alone do not establish whether events were caused by treatment.
The study summary’s aim of establishing comparability between Japanese and non-Japanese patients is not accompanied by a reported cross-population estimate or analysis. Consequently, the registry record cannot establish whether that aim was met. The results that would be needed for such an interpretation—including a defined comparison, its estimate, and its uncertainty—are not reported in the registry record.
Overall, the record describes a randomized design, treatment schedules, and an endpoint relevant to visual acuity. It does not provide enough outcome information to quantify the primary result or judge its precision. The absence of a reported result is not evidence that there was no change or no difference; it is a limit on what can be concluded from the posted results.
9. Statistical Methods Explained
The registry describes the primary measure as a mean of a continuous outcome, with standard deviation. The following points explain these statistical concepts generally; they are not a report of analyses conducted in this trial.
What does a mean change describe?
A mean is an average across the participants included in an analysis. For an outcome expressed as change from baseline, each participant’s follow-up value is compared with that participant’s baseline value, and the mean summarizes those individual changes. A mean can give a compact account of the group’s average direction and magnitude of change, but it does not show the full spread of individual responses. Participants may have changes that differ substantially even when their group mean is similar.
For BCVA, the unit is letters read correctly on an eye chart. The registry description says a positive change corresponds to reading more letters and a negative change to reading fewer. This scale-based interpretation clarifies the direction of the measure. It does not, on its own, indicate whether a particular mean change is clinically important, whether it is attributable to treatment, or whether treatment groups differ.
What does a standard deviation add?
A standard deviation describes how dispersed individual observations are around the group mean. A smaller standard deviation indicates that observed values are more concentrated around the average; a larger one indicates more spread. It is not the same as the uncertainty in the estimated mean. The standard error and confidence interval address uncertainty about an estimate, and their calculation depends on the data and analysis approach. Neither standard errors nor confidence intervals for the primary outcome are reported in the registry record.
In general, between-group interpretation focuses on a treatment contrast rather than on whether one group’s change is positive or negative by itself. Each group might change from baseline, but the contrast asks how the average change differs between assigned treatments. A confidence interval around that contrast can show the range of values compatible with the data under the analysis assumptions. A p-value addresses compatibility with a specified null hypothesis, but does not quantify the size or practical importance of a difference. The registry record does not report such a contrast, interval, or p-value for the primary endpoint.
Why does the analysis population matter?
The primary outcome population is defined as modified intent-to-treat: all randomized and treated patients with at least 1 follow-up visit. This definition excludes people who did not meet one or more of those conditions. In general, an analysis population should be interpreted using its stated inclusion rules because estimates can depend on which participants contribute data. The registry record does not provide participant-level inclusion details, reasons for exclusion, or a flow of participants through the analysis.
Randomized assignment can support a fair comparison when the analysis preserves the intended comparison and missing outcomes are handled appropriately. The term modified intent-to-treat signals that the stated population is not simply every randomized participant, because treatment and follow-up are also required. The record does not report the statistical rationale for this definition or how the analysis addressed any departures from it.
What can be inferred from planning illustrations?
The record describes a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect. Such a calculation is a planning exercise: it describes a hypothetical sample size under its assumptions, not power observed in the completed trial. The registry record does not report a sample-size justification for the original design, and a planning illustration should not be treated as evidence that the trial had adequate power.
For a continuous outcome, a planning calculation generally depends on the target difference, variability, chosen error rate, allocation, and statistical model. If the target is defined as a fraction of an observed effect, the illustration also depends on that effect estimate. Since the primary endpoint estimates and the numerical planning outputs are not reported in the registry record, no sample-size conclusion can be drawn from the stated assumptions alone. A normal approximation is a modeling choice, not a guarantee that the outcome data follow a normal distribution.
Why can over-dispersion matter?
Over-dispersion is a general concern for outcomes whose variability is greater than a statistical model assumes. When a model underestimates variability, uncertainty can be understated and statistical evidence can appear stronger than warranted. The primary endpoint is described as a continuous mean outcome, and the registry record does not report an over-dispersion analysis. This general concept should not be taken to mean that over-dispersion was present in this trial.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary endpoint and several secondary outcomes, including BCVA changes, BCVA gains of at least 15 letters and at least 10 letters, and change in central retinal thickness. It reports five outcome measures and says the primary outcome has several categories or time points and is not reduced to one comparison. When several outcomes, time points, or treatment comparisons are examined, the chance of at least one apparently notable result can increase. Statistical procedures or a prespecified hierarchy can address that issue, but the registry record does not report a multiplicity strategy.
Missing-data handling is also not reported in the registry record. The endpoint population requires randomized and treated participants with at least 1 follow-up visit, but the record does not say how missing Week 16 measurements were treated, whether any imputation was used, or what sensitivity analyses were performed. These details matter because estimates may depend on which participants have outcome measurements and on assumptions used for missing values.
The stated primary analysis population is modified intent-to-treat. Its definition is given, but the registry record does not report how many participants contributed to the primary endpoint analysis, whether that differed by treatment group, or the reasons any participants were not analyzed. It also does not report a per-protocol or safety analysis population definition.
No analysis results are listed in the registry record. Therefore, the record does not allow assessment of whether multiplicity adjustment, missing-data methods, or alternative analysis populations affected the reported findings. The absence of those details should be read as a reporting limitation, not proof that the methods were or were not used.
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 |
|---|---|---|---|
| Abicipar Pegol 2 mg | 0 | 10 | 0.0% |
| Abicipar Pegol 1 mg | 0 | 10 | 0.0% |
| Ranibizumab 0.5 mg | 2 | 5 | 40.0% |
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 |
|---|---|
| Abicipar Pegol 2 mg | 4 of 10 |
| Abicipar Pegol 1 mg | 8 of 10 |
| Ranibizumab 0.5 mg | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Abicipar Pegol 2 mg | Abicipar Pegol 1 mg | Ranibizumab 0.5 mg |
|---|---|---|---|---|
| Cataract | Eye disorders | 0 of 10 | 0 of 10 | 1 of 5 |
| Angle closure glaucoma | Eye disorders | 0 of 10 | 0 of 10 | 1 of 5 |
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 10 | 0 of 10 | 1 of 5 |
| Embolic stroke | Nervous system disorders | 0 of 10 | 0 of 10 | 1 of 5 |
| Pneumonia | Infections and infestations | 0 of 10 | 0 of 10 | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Abicipar Pegol 2 mg | Abicipar Pegol 1 mg | Ranibizumab 0.5 mg |
|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | 0 of 10 | 4 of 10 | 0 of 5 |
| Iritis | Eye disorders | 1 of 10 | 1 of 10 | 0 of 5 |
| Vitreous floaters | Eye disorders | 1 of 10 | 1 of 10 | 0 of 5 |
| Vitreous opacities | Eye disorders | 1 of 10 | 1 of 10 | 0 of 5 |
| Foreign body sensation in eyes | Eye disorders | 1 of 10 | 0 of 10 | 1 of 5 |
| Intraocular pressure increased | Investigations | 0 of 10 | 0 of 10 | 2 of 5 |
| Cataract | Eye disorders | 0 of 10 | 1 of 10 | 0 of 5 |
| Anterior chamber inflammation | Eye disorders | 0 of 10 | 1 of 10 | 0 of 5 |
| Asthenopia | Eye disorders | 0 of 10 | 1 of 10 | 0 of 5 |
| Cystitis | Infections and infestations | 0 of 10 | 1 of 10 | 0 of 5 |
Serious events with the highest arm-level rate: cataract (20.0%), angle closure glaucoma (20.0%), chronic respiratory failure (20.0%), embolic stroke (20.0%), pneumonia (20.0%).
The registry lists serious adverse-event counts separately from other adverse-event counts. For abicipar pegol 2 mg, 0 of 10 participants at risk were affected by a serious adverse event, and 4 of 10 were affected by other adverse events. For abicipar pegol 1 mg, the corresponding counts were 0 of 10 for serious events and 8 of 10 for other events. For ranibizumab 0.5 mg, they were 2 of 5 for serious events and 5 of 5 for other events. These are the posted counts, not adjusted comparisons between groups.
The record lists cataract, angle closure glaucoma, chronic respiratory failure, embolic stroke, and pneumonia among the top serious-event terms, each at 20.0%. It does not report the denominator or further context for these percentages in the adverse-event summary. The listed terms should not be read as proving that an event was caused by a study treatment.
Deaths are recorded as null for each group. Null does not provide a death count and should not be interpreted as a reported count of zero. The registry record does not give event timing, participant-level descriptions, attribution assessments, or statistical tests for the adverse-event counts. Differences in counts should therefore be treated as descriptive, particularly given the group sizes, and not as proof of comparative safety.
12. What the Result Does, and Does Not, Mean
This study record can establish what was planned and what the registry reports about the trial’s design. It describes a randomized, parallel-group phase 2 study in Japanese patients with neovascular age-related macular degeneration, with abicipar pegol and ranibizumab treatment schedules and a primary visual-acuity endpoint. It also identifies a modified intent-to-treat population and provides selected adverse-event counts.
It cannot establish the size or direction of the primary visual-acuity result because the primary endpoint estimates are not reported in the registry record. Nor can it establish whether the treatment groups differed, how precise any difference was, or whether a difference was compatible with no effect. Those questions require outcome estimates and measures of uncertainty that are absent from the posted results.
The record’s stated goal of establishing comparability between Japanese and non-Japanese patients should be interpreted as an aim, not as a demonstrated conclusion. A direct non-Japanese comparison or its results are not reported in the registry record. The trial’s title and population description alone do not provide evidence about cross-population comparability.
The adverse-event counts are also limited in what they can establish. They describe affected participants as recorded, but do not provide a causal assessment or a statistical comparison. The null entries for deaths do not supply death counts. Thus, the safety information should be read as descriptive registry reporting rather than a complete account of comparative safety.
In general, a trial result is more informative when the estimated effect is paired with uncertainty, clear analysis methods, and enough context to judge missing data and multiplicity. Those elements are not reported for the primary outcome in this record. This means conclusions should be restricted to the documented design and descriptive entries, without interpreting the unreported endpoint results as positive, negative, or equivalent.
13. Why This Trial Matters Statistically
Statistically, the study is notable for pairing an explicit treatment schedule with a continuous, patient-level visual-acuity endpoint. Change from baseline in BCVA is understandable in its units: the number of letters read correctly on an eye chart. The record also states the direction of interpretation, with positive change representing more letters read correctly. This makes the endpoint definition transparent even though its results are unavailable in the registry record.
The design includes two abicipar pegol dose schedules and a ranibizumab schedule, while the study summary frames the broader objective as establishing comparability between Japanese and non-Japanese patients. Those are different questions. A treatment-group comparison addresses differences among the listed assigned interventions; a cross-population comparison requires data and an analysis that compare populations. The registry record does not report the latter.
The record also illustrates why endpoint definitions, analysis populations, and outcome estimates must be distinguished. It specifies a modified intent-to-treat population and a mean-based primary outcome, but does not report the mean changes, variability, or a treatment contrast. Knowing the measure and population is valuable for understanding what an eventual estimate would represent, but it cannot replace the estimate or its uncertainty.
Finally, the adverse-event entries show the importance of keeping descriptive counts separate from causal or comparative claims. Counts are useful for describing what the registry records, yet group sizes and missing contextual details limit interpretation. The record therefore offers a case in which design and measurement are described more fully than the statistical evidence needed to evaluate the primary question.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main trial-specific limitation is that the registry record does not report numerical primary endpoint results. It gives the endpoint definition and its timeframe but no group means, standard deviations, treatment contrast, confidence interval, or p-value. As a result, neither the size nor the precision of the primary outcome can be assessed from the posted results.
The record identifies the analysis population as randomized and treated participants with at least 1 follow-up visit, but does not provide a participant flow, analysis counts for the endpoint, or reasons for missing follow-up. It does not report how missing measurements were handled or whether sensitivity analyses were used. These omissions restrict assessment of how representative the analyzed participants were of those randomized.
The record lists secondary outcomes and notes that the primary outcome has several categories or time points, but does not report a multiplicity strategy or a single primary comparison. This limits interpretation of any outcome claims that might otherwise be drawn from multiple measures or assessments. No analysis results are listed.
The study summary states an aim of establishing comparability between Japanese and non-Japanese patients, but a non-Japanese comparison group and direct cross-population results are not reported in the registry record. The record also does not provide detailed randomization or masking procedures. For safety, adverse-event context is limited: attribution, timing, denominator details for the listed serious-event percentages, and death counts are not reported. These limitations do not show that the methods or results were inadequate; they define what cannot be evaluated from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Abicipar Pegol in Japanese Patients With Neovascular Age-related Macular Degeneration — NCT02181504.
- Registry record last updated 2017-04-25; status last verified 2017-03.
- Results first posted 2017-04-25.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT02181504 is a completed randomized, triple-masked, parallel-group phase 2 study sponsored by Allergan. The registry lists an enrollment of 25, a start in 2014-09, and primary completion in 2015-12. The study concerned Japanese patients with neovascular age-related macular degeneration and listed abicipar pegol at 2 mg and 1 mg, ranibizumab at 0.5 mg, and sham procedure among its interventions.
The primary endpoint was change from baseline in BCVA in the study eye, measured in letters at baseline and Week 16. The specified analysis population was modified intent-to-treat. The registry record does not report primary endpoint estimates or a single comparison, and no analyses are listed. Selected serious and other adverse-event counts are reported, but further safety context is limited. The stated aim of comparing Japanese and non-Japanese patients is not accompanied by a reported cross-population analysis.