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 Phase 2, Multi-Center Study To Compare The Efficacy And Safety Of A Chemokine CCR2/5 Receptor Antagonist With Ranibizumab In Adults With Diabetic Macular Edema (NCT01994291), a randomized phase 2 study. The primary outcome is Mean Letter Change From Baseline at Week 12 in Best Corrected Visual Acuity (BCVA).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01994291 |
| Phase | Phase 2 |
| Condition | Macular Edema, Diabetic |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 199 |
| Arms in the primary outcome | 4 |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
The study asked whether oral PF-04634817 could produce a Week 12 change in best corrected visual acuity comparable to ranibizumab-based treatment in adults with diabetic macular edema. The primary outcome was the mean change in visual-acuity letters from baseline, assessed using ETDRS charts. The registry reports results for PF-04634817 and for ranibizumab comparator groups receiving either the 0.3 mg or 0.5 mg regimen, as well as a combined 0.3 mg/0.5 mg comparator group.
Population
Adults with diabetic macular edema. The primary-outcome population included randomized subjects who received at least one dose of randomized treatment and had at least one post-baseline BCVA measurement.
Intervention
PF-04634817 200 mg once daily by mouth, paired with masked sham therapy given monthly.
Comparator
Placebo once daily paired with intravitreal ranibizumab, reported in 0.3 mg, 0.5 mg, and combined 0.3 mg/0.5 mg groups.
Primary question
How did mean BCVA letter change from baseline to Week 12 compare between PF-04634817 and the ranibizumab-based groups?
The registry’s analysis is framed as non-inferiority or equivalence, but the margin and the precise decision rule are not reported in the registry record. The reported estimates therefore describe the observed adjusted contrasts; they do not, on their own, establish whether a non-inferiority criterion was met. The study was terminated, and the reason is not reported in the registry record.
3. Trial Design
This was a randomized, parallel-group, phase 2, multicenter study of adults with diabetic macular edema. The registry reports enrollment of 199 and lists the study status as terminated. The start is recorded as 2013-11 and primary completion as 2015-08. The reason for termination is not reported in the registry record.
The treatment comparison was between oral PF-04634817 200 mg once daily with monthly masked sham therapy and oral placebo once daily with monthly intravitreal ranibizumab. The registry describes the ranibizumab regimen as either 0.3 mg or 0.5 mg, administered monthly according to the prescribing information and label content approved for the country governing the site. Results are reported separately for the two ranibizumab doses and for a combined 0.3 mg/0.5 mg group.
The registry fields classify the trial as randomized, triple-masked, and parallel-group. The official title calls it double-masked. The record does not explain this difference in masking descriptions. Details of the randomization procedure, allocation concealment, or the roles masked are not reported in the registry record.
The primary outcome was mean change from baseline in BCVA letters at Week 12. The posted primary analysis population consisted of randomized subjects who had received at least one dose and had at least one post-baseline BCVA measurement. The registry lists 13 outcome measures, five secondary outcomes, and 15 analyses. The study’s planned sample-size justification, detailed analysis plan, and the reason enrollment ended at the recorded level are not reported in 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: In total, 199 participants were randomized, with 99 randomized to receive PF 04634817 200 mg and 99 to placebo + ranibizumab 0.3/0.5 mg. One participant was randomized but discontinued prior to dosing as no longer willing to participate.
Overall Study
| Stage | PF-04634817 200 mg QD | Placebo QD + Ranibizumab 0.3 mg | Placebo QD + Ranibizumab 0.5 mg |
|---|---|---|---|
| STARTED | 99 | 43 | 56 |
| Received Treatment | 99 | 43 | 56 |
| COMPLETED | 84 | 36 | 47 |
| NOT COMPLETED | 15 | 7 | 9 |
Reasons for not completing this period:
| Reason | PF-04634817 200 mg QD | Placebo QD + Ranibizumab 0.3 mg | Placebo QD + Ranibizumab 0.5 mg |
|---|---|---|---|
| Adverse Event | 4 | 1 | 3 |
| Lost to Follow-up | 1 | 1 | 0 |
| Does not meet entrance criteria | 1 | 0 | 0 |
| Medication error without associated AE | 1 | 0 | 1 |
| Protocol Violation | 1 | 0 | 0 |
| Withdrawal by Subject | 6 | 3 | 5 |
| Other | 1 | 2 | 0 |
Analysis population for the primary outcome, as defined in the registry: all subjects randomized and who had received at least one dose of randomized treatment and had at least one post-baseline measurement of BCVA.
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 | PF-04634817 200 mg QD | Placebo QD + Ranibizumab 0.3 mg | Placebo QD + Ranibizumab 0.5 mg | Total |
|---|---|---|---|---|---|
| Participants analysed | 99 | 43 | 56 | 198 | |
| Age, Continuous | years | 62.5 (SD 8.8) | 60.4 (SD 7.5) | 63.4 (SD 8.4) | 62.3 (SD 8.4) |
| Sex: Female, Male: Female | Participants | 40 | 17 | 18 | 75 |
| Sex: Female, Male: Male | Participants | 59 | 26 | 38 | 123 |
6. Primary Endpoint
The registry lists the primary outcome as Mean Letter Change From Baseline at Week 12 in Best Corrected Visual Acuity (BCVA), measured over Baseline (Day 0) and Week 12.
| Element | Registry entry |
|---|---|
| Result type | Least Squares Mean |
| Measure of spread | 80% Confidence Interval |
| Unit | Letters |
| Endpoint classification | not reduced to a single comparison |
Best corrected visual acuity, or BCVA, measures visual acuity after refraction. In this study it was assessed using retro-illuminated ETDRS charts, with distance visual acuity expressed as the number of letters correctly read. The primary endpoint was the mean change in that letter score from baseline, Day 0, to Week 12.
A change score describes how an outcome differs from a participant’s baseline measurement over the specified follow-up period. A group mean summarizes those changes, while a comparison between group means describes the estimated difference in average change. The registry reports least-squares means, which are model-based estimates rather than simple arithmetic averages. The model specification and any covariate adjustments beyond the stated mixed-model analysis are not reported in the registry record.
BCVA is a clinical measurement that can be expressed numerically, allowing group outcomes to be compared on the same scale. The endpoint is limited to the measurement and time point recorded: it does not by itself describe other aspects of diabetic macular edema, later outcomes, or the full range of patient experience. The primary outcome was measured at Week 12, and the registry’s other listed outcomes include retinal thickness, fluorescein leakage, diabetic retinopathy severity, and drug concentration.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports least-squares mean BCVA changes at Week 12 of 1.55 letters for PF-04634817, 3.87 letters for placebo plus ranibizumab 0.3 mg, 4.03 letters for placebo plus ranibizumab 0.5 mg, and 3.96 letters for the combined 0.3 mg/0.5 mg group. The corresponding reported 80% confidence intervals are -2.21 to 5.3, 0.09 to 7.65, 0.17 to 7.9, and 0.28 to 7.64, respectively. These are model-based group estimates, not individual participant outcomes.
The mixed-model comparisons report differences in least-squares means of -2.32 letters for PF-04634817 versus the 0.3 mg comparator, -2.48 versus the 0.5 mg comparator, and -2.41 versus the combined comparator. Each reported difference is negative, indicating a lower estimated mean change in the PF-04634817 group on the registry’s stated contrast scale. The 80% confidence intervals are -4.27 to -0.37, -4.24 to -0.73, and -3.91 to -0.91, respectively. The confidence intervals for all three reported contrasts lie below zero.
The associated p values are 0.127, 0.070, and 0.040, respectively. The analyses are categorized in the registry as non-inferiority or equivalence analyses. Those p values cannot be interpreted as a complete answer to that question without knowing the tested hypothesis, the non-inferiority margin, and the decision rule. These details are not reported in the registry record. In particular, a p value by itself does not establish whether a non-inferiority or equivalence criterion was satisfied.
An 80% confidence interval conveys uncertainty under the analysis model, but is less stringent than the commonly discussed 95% interval. General explanation: a confidence interval describes a range of values compatible with the data and model at its stated confidence level; it is not a guarantee that the true effect lies in that interval. The registry reports no 95% confidence interval for these primary comparisons.
The estimates should be read in the context of the stated analysis population, which required treatment and a post-baseline BCVA measurement. The registry does not report how missing outcome measurements were handled, so the effect of missingness on the estimates cannot be assessed from the record. Nor does the Week 12 endpoint describe longer-term outcomes. The reported results are estimates from this study, not a basis for a treatment recommendation.
9. Statistical Methods Explained
The registry reports least-squares means for the primary outcome and mixed-model analyses for the comparisons. Least-squares means are model-based estimates of group outcomes. A mixed model is a statistical framework that can account for multiple measurements or other structured sources of variation, depending on how it is specified. General explanation: repeated-measures models can use information across visits and represent within-participant correlations, but the exact structure and assumptions depend on the model. The registry does not report the model formula, covariance structure, covariates, or other implementation details.
What does the least-squares mean represent?
The posted group estimates are 1.55 letters for PF-04634817, 3.87 letters for placebo plus ranibizumab 0.3 mg, 4.03 letters for placebo plus ranibizumab 0.5 mg, and 3.96 letters for the combined comparator group. The estimates are reported as least-squares means, so they should not automatically be treated as raw sample means. The record does not give enough model detail to identify precisely how the estimates were adjusted.
The reported analysis population included randomized subjects who had received at least one dose and had at least one post-baseline BCVA measurement. This definition excludes randomized subjects without a post-baseline measurement from the stated primary analysis population. The number excluded for that reason and the handling of missing measurements among included subjects are not reported in the registry record.
How should the confidence intervals be read?
The group estimates have reported 80% confidence intervals: -2.21 to 5.3 for PF-04634817, 0.09 to 7.65 for the 0.3 mg comparator, 0.17 to 7.9 for the 0.5 mg comparator, and 0.28 to 7.64 for the combined comparator. The comparison intervals are also reported at 80%: -4.27 to -0.37, -4.24 to -0.73, and -3.91 to -0.91.
General explanation: a confidence interval communicates uncertainty around an estimate under the model and analysis assumptions. A narrower interval indicates greater precision than a wider interval on the same scale and at the same confidence level. An interval is not a statement about the range of individual outcomes, nor does it show how outcomes are distributed among participants. The registry does not report standard deviations or individual-level data for this endpoint.
What does the comparison estimate mean?
The mixed-model analysis reports differences in least-squares means. The contrast values are -2.32 for PF-04634817 versus the 0.3 mg group, -2.48 versus the 0.5 mg group, and -2.41 versus the combined group. The negative signs indicate that the estimated mean change in the PF-04634817 group was lower on the contrast scale used in the registry. The record does not provide the model’s full contrast specification, though the listed comparisons identify the groups involved.
The registry labels these analyses as non-inferiority or equivalence. For such a conclusion, the size of the estimated contrast must be assessed against a prespecified margin and a stated decision rule. Neither the margin nor a complete testing procedure is reported in the registry record. The analysis p values—0.127, 0.070, and 0.040—therefore cannot be interpreted as ordinary tests of whether the two group means are equal, or as proof of non-inferiority or equivalence.
Why do the confidence level and p value need context?
The reported comparison intervals use an 80% confidence level. General explanation: intervals at different confidence levels have different coverage properties and typically different widths, so they should not be compared as if they were interchangeable. The p values and confidence intervals answer related but not necessarily identical questions when a non-inferiority margin or other specialized null hypothesis is involved. The precise relationship cannot be reconstructed without the analysis plan and margin.
The registry also describes planning illustrations based on the observed effect, equal allocation, a two-sided significance level of 0.05, and a normal approximation. Such calculations are planning illustrations rather than observed power; the registry’s sample-size assumptions do not establish the original study’s design justification. The underlying design rationale and any target sample size are not reported in the registry record.
10. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| Mixed Models Analysis | Difference in LS means -2.32 | -4.27 to -0.37 | 0.127 |
| Mixed Models Analysis | Difference in LS means -2.48 | -4.24 to -0.73 | 0.070 |
| Mixed Models Analysis | Difference in LS means -2.41 | -3.91 to -0.91 | 0.040 |
The registry reports mixed-model analyses for the primary BCVA outcome. The reported contrasts are -2.32 letters for PF-04634817 versus placebo plus ranibizumab 0.3 mg, -2.48 versus the 0.5 mg group, and -2.41 versus the combined 0.3 mg/0.5 mg group. Their respective 80% confidence intervals are -4.27 to -0.37, -4.24 to -0.73, and -3.91 to -0.91.
No separate recalculated comparison is reported in the registry record, so a numerical reconciliation between an independent recalculation and the registry analysis cannot be made from the record. The reported mixed-model results should be distinguished from any simple, unadjusted calculation based on summary statistics: they are presented as model-based differences in least-squares means. The analysis details needed to reproduce the mixed model are not reported in the registry record.
The non-inferiority or equivalence label also matters when reading the reported p values. The margin and the exact hypothesis-testing procedure are not reported in the registry record, so the p values cannot independently confirm a non-inferiority or equivalence conclusion. The reported estimates and intervals describe the posted analysis; interpretation of the study’s formal objective requires those missing design and analysis details.
11. Multiplicity, Missing Data and Analysis Populations
The registry lists 13 outcome measures, five secondary outcomes, and 15 analyses. The secondary outcomes include the proportion gaining 15 ETDRS letters at Week 12, changes in central subfield retinal thickness, fluorescein leakage area and diabetic retinopathy severity at Week 12, and plasma concentration of PF-04634817 up to Week 12. Multiple outcomes and comparisons raise a general multiplicity issue: when many statistical questions are evaluated, the chance of at least one apparently notable result can increase unless the analysis plan addresses that issue.
Whether multiplicity was controlled, and by what method, is not reported in the registry record. The record does not identify a hierarchy of outcomes, a family of hypotheses, an adjustment procedure, or which analyses were confirmatory versus exploratory. The presence of multiple listed analyses is not itself evidence that an adjustment was or was not applied.
The primary outcome population was restricted to randomized subjects who had received at least one dose and had at least one post-baseline BCVA measurement. The number of randomized subjects who did not meet that definition, the reasons measurements were unavailable, and any sensitivity analyses are not reported in the registry record. The method for handling missing outcomes—such as a model-based approach, imputation, or another strategy—is also not reported.
General explanation: missing data can affect an estimate when the availability of an outcome is related to participant characteristics or outcomes, and different assumptions about missingness can lead to different results. Without the missing-data approach and relevant sensitivity analyses, the robustness of the reported estimate to missing measurements cannot be evaluated from the registry record.
The analysis population is not described as all randomized subjects regardless of treatment received. The record does not report an intention-to-treat analysis or a per-protocol analysis for the primary endpoint. This limits assessment of how the chosen population relates to the randomized comparison, especially for an analysis categorized as non-inferiority or equivalence.
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 |
|---|---|---|---|
| PF-04634817 200 mg QD | 7 | 99 | 7.1% |
| Placebo QD + Ranibizumab 0.3 mg | 2 | 43 | 4.7% |
| Placebo QD + Ranibizumab 0.5 mg | 3 | 56 | 5.4% |
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 |
|---|---|
| PF-04634817 200 mg QD | 17 of 99 |
| Placebo QD + Ranibizumab 0.3 mg | 15 of 43 |
| Placebo QD + Ranibizumab 0.5 mg | 7 of 56 |
Most frequent serious adverse event terms
| Event term | Organ system | PF-04634817 200 mg QD | Placebo QD + Ranibizumab 0.3 mg | Placebo QD + Ranibizumab 0.5 mg |
|---|---|---|---|---|
| Aortic valve stenosis | Cardiac disorders | 1 of 99 | 0 of 43 | 0 of 56 |
| Cardiac failure | Cardiac disorders | 1 of 99 | 0 of 43 | 0 of 56 |
| Coronary artery disease | Cardiac disorders | 1 of 99 | 0 of 43 | 0 of 56 |
| Pancreatitis chronic | Gastrointestinal disorders | 0 of 99 | 0 of 43 | 1 of 56 |
| Vomiting | Gastrointestinal disorders | 1 of 99 | 0 of 43 | 0 of 56 |
| Paronychia | Infections and infestations | 0 of 99 | 0 of 43 | 1 of 56 |
| Sepsis | Infections and infestations | 1 of 99 | 0 of 43 | 0 of 56 |
| Head injury | Injury, poisoning and procedural complications | 1 of 99 | 0 of 43 | 0 of 56 |
| Rib fracture | Injury, poisoning and procedural complications | 1 of 99 | 0 of 43 | 0 of 56 |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | 0 of 99 | 0 of 43 | 1 of 56 |
Most frequent non-serious adverse event terms
| Event term | Organ system | PF-04634817 200 mg QD | Placebo QD + Ranibizumab 0.3 mg | Placebo QD + Ranibizumab 0.5 mg |
|---|---|---|---|---|
| Diabetic retinal oedema | Eye disorders | 4 of 99 | 7 of 43 | 3 of 56 |
| Headache | Nervous system disorders | 3 of 99 | 4 of 43 | 0 of 56 |
| Conjunctival haemorrhage | Eye disorders | 5 of 99 | 1 of 43 | 0 of 56 |
| Eye irritation | Eye disorders | 6 of 99 | 0 of 43 | 0 of 56 |
| Vitreous haemorrhage | Eye disorders | 2 of 99 | 3 of 43 | 1 of 56 |
| Blepharitis | Eye disorders | 1 of 99 | 3 of 43 | 0 of 56 |
| Intraocular pressure increased | Investigations | 0 of 99 | 1 of 43 | 3 of 56 |
| Retinal haemorrhage | Eye disorders | 0 of 99 | 3 of 43 | 0 of 56 |
Serious events with the highest arm-level rate: hyperuricaemia (2.3%), personality disorder (2.3%), pancreatitis chronic (1.8%), paronychia (1.8%), diabetic ketoacidosis (1.8%).
The registry reports serious adverse events for 7 of 99 subjects in the PF-04634817 group, 2 of 43 in the placebo plus ranibizumab 0.3 mg group, and 3 of 56 in the placebo plus ranibizumab 0.5 mg group. Other adverse events are reported for 17 of 99, 15 of 43, and 7 of 56, respectively. These are posted counts with their stated at-risk denominators; they should not be combined across groups or interpreted as comparative effects without an appropriate analysis.
The listed serious adverse-event terms include hyperuricaemia and personality disorder, each reported as 2.3%, and pancreatitis chronic, paronychia, diabetic ketoacidosis, and VIIth nerve paralysis, each reported as 1.8%. The record does not provide further detail in the registry record about event timing, severity beyond the serious-event classification, recurrence, or attribution to treatment.
Death counts are not reported in the registry record. A missing death count should not be read as a reported count of zero. The safety counts also use group denominators that differ from the primary efficacy analysis counts; the registry does not explain the relationship between those analysis populations. These descriptive figures do not by themselves establish whether an event rate differs between treatments.
13. What the Result Does, and Does Not, Mean
The posted primary results show estimated Week 12 BCVA changes of 1.55 letters for PF-04634817 and estimates from 3.87 to 4.03 letters across the listed ranibizumab-based groups. The reported mixed-model contrasts are negative for PF-04634817 relative to each comparator group. Their 80% confidence intervals lie below zero, while the recorded analysis type is non-inferiority or equivalence.
Those findings describe the registry’s estimates and their uncertainty under the reported analysis. They do not establish whether PF-04634817 met a non-inferiority or equivalence criterion, because the relevant margin and complete decision rule are not reported in the registry record. Nor do the results answer questions about outcomes beyond Week 12, other measures of eye health, or how individual participants experienced treatment.
The efficacy estimates apply to the stated analysis population: randomized subjects who received at least one dose and had at least one post-baseline BCVA measurement. The registry does not report enough information to judge how missing measurements, departures from assigned treatment, or other exclusions may have affected the comparison. The primary analysis is therefore best read as a model-based summary for the defined population, rather than a complete account of every randomized subject.
The adverse-event counts provide a descriptive view of serious and other events in the listed safety groups. They do not provide a formal comparative safety analysis, and the registry record does not report death counts. The event counts should not be interpreted as proof of a treatment-related cause or as a comprehensive account of safety.
Overall, the registry gives a Week 12 outcome comparison and selected safety counts for a terminated phase 2 study. It does not report the termination reason, a detailed statistical analysis plan, the non-inferiority margin, or a full account of missing-data methods. These omissions constrain the conclusions that can be drawn from the posted results. No treatment recommendation follows from these statistics alone.
14. Why This Trial Matters Statistically
This trial illustrates why the label attached to a statistical analysis matters. The primary results are reported as mixed-model differences in least-squares means, and the registry categorizes the comparisons as non-inferiority or equivalence. That framing requires more than a direction of effect or a p value: the margin and decision rule are central to judging the stated objective, yet they are not reported in the registry record.
The confidence level also affects interpretation. The registry reports 80% confidence intervals for both group estimates and contrasts. Readers should keep that stated level in mind rather than treating the intervals as though they were at another confidence level. General explanation: uncertainty intervals are meaningful only alongside their confidence level, model assumptions, and target estimand.
The record also shows the distinction between model-based estimates and simple summaries. Least-squares means and mixed-model contrasts depend on model choices, but details such as the model structure, covariates, and missing-data approach are not reported. Without them, the analysis cannot be fully reproduced or assessed for sensitivity to modeling assumptions.
Finally, several outcomes and analyses are listed, while the multiplicity strategy is not reported. That omission matters when interpreting a collection of statistical findings, because the number and relationship of hypotheses affect how evidence should be evaluated. The trial is therefore a useful example of how registry results can provide estimates and uncertainty while leaving important elements of the inferential framework unresolved.
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.
Several limitations specific to this record restrict interpretation. The study status is listed as terminated, but the reason is not reported in the registry record. The record gives enrollment of 199, but the planned sample-size rationale and whether the study reached its intended enrollment are not reported. The trial’s start and primary completion are recorded as 2013-11 and 2015-08, but this does not explain the termination or the duration of follow-up for individual subjects.
The primary endpoint was measured at Week 12, so the posted estimate addresses that time point rather than longer-term visual acuity. Although the registry lists other outcomes, the methods and complete results for all of them are not included in the registry record. The record also does not report the non-inferiority margin, a detailed analysis plan, model specification, multiplicity procedure, missing-data methods, or sensitivity analyses.
The analysis population required receipt of at least one dose and a post-baseline BCVA measurement. The record does not report how many randomized subjects were not included in that population or why measurements were unavailable. It also does not describe adherence, treatment discontinuation, or how those events were handled analytically.
The masking descriptions differ: the registry fields classify the study as triple-masked, while the official title calls it double-masked. The record does not explain the difference. Safety information is limited to the posted adverse-event counts and selected serious-event terms; death counts are not reported in the registry record. These limitations do not negate the reported estimates, but they leave important uncertainties about design, analysis, and interpretation.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: A Phase 2, Multi-Center Study To Compare The Efficacy And Safety Of A Chemokine CCR2/5 Receptor Antagonist With Ranibizumab In Adults With Diabetic Macular Edema — NCT01994291.
- Registry record last updated 2016-10-17; status last verified 2016-08.
- Results first posted 2016-10-17.
- 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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19. Record Summary
NCT01994291 is a randomized, parallel-group phase 2 study of PF-04634817 compared with ranibizumab-based treatment in adults with diabetic macular edema. The registry lists enrollment of 199 and study status as terminated. The primary outcome was mean change from baseline in BCVA letters at Week 12, assessed with ETDRS charts.
The reported least-squares mean changes were 1.55 letters for PF-04634817 and 3.87, 4.03, and 3.96 letters for the ranibizumab 0.3 mg, 0.5 mg, and combined groups. The mixed-model contrasts were negative, with reported 80% confidence intervals below zero. The registry categorizes these as non-inferiority or equivalence analyses, but does not report the margin or full decision rule.
Serious and other adverse-event counts are reported by group; death counts are not reported in the registry record. The termination reason, detailed statistical plan, multiplicity strategy, and missing-data methods are also not reported.