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 Loteprednol vs Prednisolone for the Treatment of Intraocular Inflammation Following Cataract Surgery in Children. (NCT01475643), a randomized phase 3 study. The primary outcome is Anterior Chamber Inflammation.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01475643 |
| Phase | Phase 3 |
| Condition | Cataract |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 107 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Bausch & Lomb Incorporated |
| Results posted | Yes |
2. Clinical Question
Population
Children undergoing surgery for cataract, with postoperative intraocular inflammation as the clinical context. The registry lists Cataract as the condition and describes the study as addressing inflammation following ocular surgery for childhood cataract. Specific participant ages and other eligibility criteria are not reported in the registry record.
Intervention
Loteprednol etabonate 0.5%. The official title describes topical treatment, but the registry record does not report a dosing schedule or treatment duration.
Comparator
Prednisolone acetate 1.0%, also described in the arm listing as Prednisolones Acetate. The record does not report details of how treatment was administered beyond the named intervention and strength.
Primary question
At Postoperative Day 29, what was the mean grade of anterior chamber inflammation in each treatment arm? The registry posts arm-specific means and 95% confidence intervals, but no estimated between-arm contrast or statistical test. The endpoint description also refers to a “Proportion of subjects” with inflammation, while the posted measure is a mean grade; the intended relationship between those descriptions is not clarified in the registry record.
3. Trial Design
The registry record describes a randomized, double-masked, parallel-group Phase 3 trial. The study compared loteprednol etabonate 0.5% with prednisolone acetate 1.0% for treating postoperative inflammation following surgery for childhood cataract. The listed sponsor is Bausch & Lomb Incorporated.
Enrollment is recorded as 107. The trial is marked COMPLETED, with a start date of 2013-06 and a primary completion date of 2017-06-02. The posted primary endpoint results include 53 participants in the loteprednol arm and 48 in the prednisolone arm. These arm-specific endpoint counts do not sum to the overall enrollment, and the registry record does not explain the difference. Safety counts use different denominators: 54 and 53 participants at risk in the respective arms.
Randomization and masking are identified at a high level, but the method used to generate the allocation sequence, allocation concealment, and the people kept masked are not reported in the registry record. The record does not state whether participants could switch treatments, whether additional postoperative care was standardized, or how long assigned treatment continued.
The primary endpoint is described as anterior chamber inflammation, measured as a grade of anterior chamber cells at Postoperative Day 29. A secondary outcome is listed as Anterior Chamber Cells & Flare. The registry identifies two outcome measures, but reports one secondary outcome by name and does not post analysis records. Details of the full protocol, statistical analysis plan, and the reasoning behind the planned enrollment 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.
Overall Study
| Stage | Loteprednol Etabonate | Prednisolones Acetate |
|---|---|---|
| STARTED | 54 | 53 |
| COMPLETED | 40 | 43 |
| NOT COMPLETED | 14 | 10 |
Analysis population for the primary outcome, as defined in the registry: Proportion of subjects with anterior chamber inflammation
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 | Loteprednol Etabonate | Prednisolones Acetate | Total |
|---|---|---|---|---|
| Participants analysed | 53 | 52 | 105 | |
| Age, Continuous | years | 3.7 (SD 3.22) | 4.3 (SD 3.39) | 4 (SD 3.305) |
| Sex: Female, Male: Female | Participants | 22 | 26 | 48 |
| Sex: Female, Male: Male | Participants | 31 | 26 | 57 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 24 | 20 | 44 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 29 | 32 | 61 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | 1 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 18 | 18 | 36 |
| Race (NIH/OMB): Black or African American | Participants | 8 | 10 | 18 |
| Race (NIH/OMB): White | Participants | 26 | 23 | 49 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 53 | 52 | 105 |
6. Primary Endpoint
The registry lists the primary outcome as Anterior Chamber Inflammation, measured over Postoperative Day 29.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | grade of anterior chamber cells |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is the mean grade of anterior chamber inflammation at Postoperative Day 29. The registry describes an ordinal scale from 0, meaning no visible clouding, through 4, meaning very severe clouding. Intermediate grades describe increasing clouding and changes in visibility of the iris pattern and red reflex. The endpoint description gives additional examination guidance for subjects who could only be examined with a pen light and a 20D magnifying lens.
Anterior chamber inflammation is a clinical sign of inflammation inside the front part of the eye. A graded measure can capture differences in severity rather than reducing all observations to whether inflammation is present or absent. A mean summarizes the arm's average grade, but it does not show the distribution of individual grades. In particular, a mean alone cannot reveal how many participants had no inflammation, mild inflammation, or higher grades.
There is a wording inconsistency in the registry: the primary population is called the “Proportion of subjects with anterior chamber inflammation,” whereas the measure is identified as a mean of a continuous measure and the unit is a grade. The posted results are means with 95% confidence intervals, not proportions. The record does not clarify whether the proportion wording refers to a separate intended analysis or is a labeling error. That uncertainty matters when interpreting exactly what the primary endpoint was intended to estimate.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
At Postoperative Day 29, the posted mean anterior chamber inflammation grade was 0.913 in the loteprednol etabonate arm, with a 95% confidence interval from 0.668 to 1.158. In the prednisolone acetate arm, the posted mean was 0.783, with a 95% confidence interval from 0.528 to 1.038. The endpoint counts were 53 and 48, respectively. Because lower grades on the registry's scale represent less visible inflammation, the arm means describe the average grade in each group on that scale.
These are arm-specific summaries, not a direct estimate of the difference between treatments. The registry record does not post a between-arm effect estimate, confidence interval for that contrast, or p-value. The available results therefore do not establish the size or precision of a comparative effect. It would be inappropriate to infer a treatment comparison by subtracting the means or by treating the separate arm confidence intervals as though they were a confidence interval for the difference.
A confidence interval describes uncertainty around an estimated quantity under the assumptions of the method used to construct it. Here the posted intervals are for the arm means. They indicate the range of values compatible with those estimates under the applicable statistical procedure, but the procedure itself is not described in the registry record. The intervals do not describe the range of individual participants' grades, and they do not by themselves show whether the arms differ statistically.
The endpoint is a grade with categories from 0 through 4, but the posted summary is a mean. A mean is compact and familiar, yet it can conceal the shape of the underlying grade distribution. Different patterns of participant grades can yield similar averages. The registry does not post grade counts or other distributional summaries, so the results do not show how frequently each severity category occurred.
There is also a mismatch between the endpoint's population wording, which refers to a proportion of subjects, and the reported mean-grade measure. The record does not resolve that inconsistency. The most direct reading of the numerical results is therefore limited to the posted arm means and their confidence intervals. No claim about comparative benefit, equivalence, or non-inferiority is supported by the registry results alone. A prespecified margin, analysis population, and comparative analysis would be needed to assess such questions; those details are not reported in the registry record.
9. Statistical Methods Explained
The registry describes a randomized, double-masked, parallel-group trial. Randomization is intended, in general, to make treatment groups comparable in expectation by assigning participants through a chance-based process. Masking is intended, in general, to limit the influence of knowledge of assigned treatment on participants, clinical assessments, or other decisions. The registry does not report the specific randomization procedure or identify who was masked, so the implementation of these safeguards cannot be evaluated from the record.
What does the mean summarize?
The posted primary measure is the mean grade of anterior chamber inflammation. The scale assigns grades from 0 to 4, with higher grades describing more clouding and more severe findings. Taking a mean treats grade values as numerical quantities and summarizes their central tendency. This can be useful for a concise comparison, but grades are ordered categories: the scale establishes an order of severity without necessarily establishing that every step represents an equal change in clinical state. The registry does not report an alternative ordinal analysis or participant-level grade frequencies.
The record identifies the measure as continuous and reports means with 95% confidence intervals. It does not describe the method used to estimate the means or intervals, whether any adjustment was applied, or whether the analysis accounted for features of the study design beyond the treatment groups. Those methods are not reported in the registry record, so the assumptions behind the posted intervals cannot be independently assessed from the registry entry.
What does a confidence interval communicate?
In general, a confidence interval expresses the uncertainty associated with an estimated quantity under a statistical method and its assumptions. The intervals posted apply separately to the mean grade in each arm. They are not intervals for the treatment difference. A direct comparison would ordinarily report an estimate of the contrast between arms and its uncertainty, possibly alongside a test statistic or p-value. No such comparative result is posted.
The reported intervals also should not be read as containing most participants' individual grades. They concern uncertainty about the arm means, not the spread of individual observations. The record gives no standard deviations, grade distributions, or other measure of participant-level variability. Without those summaries, the amount and pattern of variation within each treatment group remain unclear.
Why can a mean be incomplete for a grade scale?
For an ordered scale, the same mean can arise from different combinations of grades. A mean does not show whether observations are concentrated near one grade or spread across several grades. It can also make a small shift in the average seem like a complete account when differences in the tails of the distribution may be of interest. Reporting the frequency at each grade, or an appropriate ordinal summary, can help readers understand the pattern. Such details are not reported in the registry record.
The endpoint description adds a qualification about subjects who could only be examined with a pen light and a 20D magnifying lens. It does not state how many participants were assessed in that way, how this examination circumstance affected scoring, or whether the same assessment conditions applied across arms. The record also uses proportion language for the endpoint population while reporting a mean grade. This unresolved labeling issue limits certainty about the exact estimand—the quantity the analysis was intended to estimate.
What is absent from the comparative analysis?
The registry marks the continuous two-arm comparison as unavailable or not estimable and posts no analysis records. Consequently, there is no reported adjusted or unadjusted comparison, no p-value, and no confidence interval for a treatment contrast. The arm means can be described, but a comparative conclusion cannot be reconstructed without making calculations that the record does not post. No recalculated contrast is available in the registry record.
General statistical practice distinguishes the estimate from the decision rule. An estimate conveys the direction and magnitude of an observed contrast; an interval conveys its precision; and a test assesses compatibility with a specified null hypothesis under a model. None of those comparative components is reported. The record does not report a null hypothesis, a superiority or non-inferiority objective, or a decision threshold for the primary comparison.
How should planning illustrations be understood?
Any sample-size planning illustrations associated with the posted results are not the trial's original design justification. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation, a normal approximation, and an effect defined as a stated fraction of the observed effect. Such illustrations are hypothetical planning exercises, not evidence of the trial's actual power and not a replacement for the missing design rationale. The registry does not report the original sample-size justification or the assumptions used by the investigators when planning enrollment.
In general, power calculations describe the chance that a planned analysis would detect a specified effect under particular assumptions. They do not measure the probability that a result is true, and calculations based on an observed effect should not be described as observed power. The illustration assumptions alone do not establish which effect the investigators considered clinically important or whether the completed enrollment was adequate for their intended question.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists Anterior Chamber Cells & Flare as a secondary outcome and identifies two outcome measures overall. It does not post analysis records. The record does not describe a multiplicity strategy, such as prioritizing outcomes, adjusting significance thresholds, or treating secondary findings as exploratory. As a result, the interpretation of any potential collection of outcome tests cannot be evaluated from the registry record. No secondary numerical result is included in the registry entries.
The registry does not report how missing endpoint measurements were handled, how many participants lacked a Postoperative Day 29 assessment, or whether missingness differed between arms. It also does not state whether participants were analyzed according to their randomized assignment, according to treatment received, or within another analysis population. Those details are important because exclusions and missing measurements can affect the interpretation of an estimated treatment contrast.
The primary endpoint counts are 53 for loteprednol etabonate and 48 for prednisolone acetate, while enrollment is 107. The record does not explain why the endpoint counts differ from enrollment or whether the counts represent all randomized participants with an available endpoint. Safety counts use 54 and 53 participants at risk, respectively. These distinct denominators should not be assumed to represent the same analysis population. The registry does not provide a participant flow account or reasons for any differences in counts.
The endpoint description's “Proportion of subjects” wording alongside a mean-grade result adds another uncertainty about the planned analysis. The registry does not clarify whether the proportion wording corresponds to an outcome that was not posted or to a description of the population for the grade analysis. Without an analysis plan, missing-data information, and definitions of analysis populations, the robustness of the posted summaries cannot be assessed.
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 |
|---|---|---|---|
| Loteprednol Etabonate | 3 | 54 | 5.6% |
| Prednisolones Acetate | 0 | 53 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Loteprednol Etabonate | 0 of 54 |
| Prednisolones Acetate | 0 of 53 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Loteprednol Etabonate | 34 of 54 |
| Prednisolones Acetate | 23 of 53 |
Most frequent serious adverse event terms
| Event term | Organ system | Loteprednol Etabonate | Prednisolones Acetate |
|---|---|---|---|
| Glaucoma | Eye disorders | 2 of 54 | 0 of 53 |
| Vitreous Disorder | Eye disorders | 1 of 54 | 0 of 53 |
| Bronchiolitis | Eye disorders | 1 of 54 | 0 of 53 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Loteprednol Etabonate | Prednisolones Acetate |
|---|---|---|---|
| Eye Pain | Eye disorders | 6 of 54 | 4 of 53 |
| Eyelid Odema | Eye disorders | 4 of 54 | 4 of 53 |
| Nasopharyngitis | Infections and infestations | 2 of 54 | 4 of 53 |
| Discomfort | General disorders | 2 of 54 | 3 of 53 |
| Ocular hyperemia | Eye disorders | 3 of 54 | 0 of 53 |
| Pyrexia | General disorders | 1 of 54 | 2 of 53 |
| Ear Infection | Infections and infestations | 2 of 54 | 1 of 53 |
| Gastroenteritis Viral | Infections and infestations | 2 of 54 | 1 of 53 |
| Cough | Respiratory, thoracic and mediastinal disorders | 2 of 54 | 1 of 53 |
| Amblyopia | Eye disorders | 1 of 54 | 1 of 53 |
Serious events with the highest arm-level rate: glaucoma (3.7%), vitreous disorder (1.9%), bronchiolitis (1.9%).
The registry reports serious adverse events among 54 participants at risk in the loteprednol etabonate group, with 3 affected, and among 53 participants at risk in the prednisolone acetate group, with 0 affected. It reports 0 deaths in each group. These are counts recorded in the registry; they do not, on their own, establish that an event was caused by a study treatment.
The record also reports other adverse events affecting 34 participants in the loteprednol group and 23 in the prednisolone group. The registry does not provide further detail in the registry entries about their definitions, event-level counts, severity, timing, or attribution. The serious adverse event terms listed are Glaucoma at 3.7%, Vitreous Disorder at 1.9%, and Bronchiolitis at 1.9%. The record does not specify in the posted information how these listed terms are distributed by treatment arm.
Safety counts should be read with their stated denominators and kept distinct from the primary endpoint counts. The registry does not report an inferential comparison of adverse events, methods for collecting them, or a causal assessment. The figures describe what was recorded; they do not provide a complete assessment of comparative safety.
12. What the Result Does, and Does Not, Mean
The posted results describe average anterior chamber inflammation grades at Postoperative Day 29 in the two randomized groups. The loteprednol etabonate group had a mean grade of 0.913, with a 95% confidence interval from 0.668 to 1.158. The prednisolone acetate group had a mean grade of 0.783, with a 95% confidence interval from 0.528 to 1.038. These arm-level estimates offer a numerical account of the recorded endpoint, but they do not constitute a formal treatment comparison.
In particular, the registry does not post a between-group estimate, its confidence interval, or a p-value. Without these, the precision of the difference between treatments cannot be read directly from the record. The separate confidence intervals do not answer whether the treatments differ, and the registry does not report a prespecified criterion for superiority, equivalence, or non-inferiority. The displayed findings therefore cannot support a conclusion in any of those terms.
The grade scale ranges from 0 to 4, but means alone do not tell readers how participants were distributed across the categories. The registry also describes the endpoint population as a proportion of subjects, while reporting a mean grade. That inconsistency remains unresolved. It is therefore important to keep the interpretation close to the actual posted measure: arm-specific mean grades and their intervals.
The findings also do not establish how well the results apply to particular children or clinical settings. Eligibility criteria, age distribution, details of surgical practice, duration and schedule of study treatment, and information about care after surgery are not reported in the registry record. Nor does the entry explain differences between enrollment, endpoint counts, and safety denominators.
These limitations do not erase the descriptive information that is posted, but they define what it can answer. The registry offers a snapshot of the mean grade at the specified time point, alongside safety counts. It does not provide enough comparative statistical detail to determine the magnitude or precision of a treatment contrast, nor enough outcome-distribution detail to understand individual severity patterns.
13. Why This Trial Matters Statistically
This trial is statistically notable because it reports a graded clinical endpoint as an arm-specific mean while leaving the direct comparison unreported. A reader can see the estimated mean grade and confidence interval for each group, but cannot see the estimated difference between groups or its uncertainty. Those are distinct quantities, and the latter is central when the question is whether two randomized treatments produced different outcomes.
The endpoint illustrates a broader issue in analysis of ordered clinical scales. The categories have a meaningful order, from no visible clouding to very severe clouding, but a mean compresses the underlying pattern into one value. That summary may be useful, yet it does not show the number of participants at each grade. The registry's additional use of proportion language for a mean-grade outcome makes clear endpoint definition especially important.
Reporting an analysis population, missing-data approach, grade distribution, and between-arm estimate would help readers judge both the result and its precision. A confidence interval for each mean is not a substitute for a confidence interval for the treatment contrast. The difference is relevant even when both arms have estimates and intervals posted.
The study also illustrates why design labels do not answer every methodological question. Randomization and double masking are stated, but their implementation details are not. The registry identifies the trial as Phase 3 and completed, yet does not report the original sample-size justification or the statistical plan. These omissions constrain what can be learned from the registry entry about how the study was designed to answer its 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.
Several features of the registry entry limit interpretation beyond the absence of a comparative analysis. The endpoint is described both as a “Proportion of subjects with anterior chamber inflammation” and as a mean grade of anterior chamber cells. The numerical results are means with 95% confidence intervals, but the record does not explain the proportion wording. Readers cannot tell whether the wording reflects a separate intended endpoint, a population label, or a recording inconsistency.
The posted measure summarizes a scale from 0 to 4, but no counts by grade or other description of the outcome distribution are reported. The record therefore does not show whether the arm means arise from similar or different patterns of grades. It also does not explain the method used to calculate the confidence intervals or report a direct comparison, leaving uncertainty about both the model and the precision of the treatment contrast.
Participant counts vary by context: enrollment is 107, endpoint counts are 53 and 48, and safety denominators are 54 and 53. The registry does not explain the differences or provide reasons for missing endpoint data, participant exclusions, or changes in analysis populations. The missing-data method and analysis population are not reported in the registry record.
Other trial details that would support assessment of applicability and conduct are absent, including eligibility criteria, participant ages, the randomization method, which groups of people were masked, and the treatment schedule. The record also does not report the original sample-size justification, the statistical analysis plan, or a multiplicity strategy. These are limitations of the registry information and do not by themselves establish how the trial was conducted.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Loteprednol vs Prednisolone for the Treatment of Intraocular Inflammation Following Cataract Surgery in Children. — NCT01475643.
- Registry record last updated 2019-06-11; status last verified 2019-06.
- Results first posted 2019-06-11.
- 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
NCT01475643 is a completed, randomized, double-masked, parallel-group Phase 3 trial sponsored by Bausch & Lomb Incorporated. It enrolled 107 participants and compared loteprednol etabonate 0.5% with prednisolone acetate 1.0% for postoperative inflammation following childhood cataract surgery. The registry gives a start date of 2013-06 and a primary completion date of 2017-06-02.
The primary result is the mean anterior chamber inflammation grade at Postoperative Day 29: 0.913 (95% confidence interval 0.668 to 1.158) for loteprednol, and 0.783 (95% confidence interval 0.528 to 1.038) for prednisolone. No comparative estimate or test is posted. A secondary outcome is named, but no analysis records are reported. Safety entries include serious adverse events affecting 3 of 54 participants in the loteprednol group and 0 of 53 in the prednisolone group, with 0 deaths in each group. The registry does not report key analysis details, including missing-data handling, the analysis population, and the original sample-size justification.