Oxulumis® Suprachoroidal Microcatherization of Triesence® in Diabetic Macular Edema: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Oxulumis® Suprachoroidal Microcatherization of Triesence® in Diabetic Macular Edema (NCT05512962), a randomized phase 2 study. The primary outcome is Frequency of Ocular Adverse Events, Systemic Adverse Events, Serious, and Treatment-emergent Non-serious Adverse Events.

2. Clinical Question

This is principally a safety and tolerability question. It concerns the occurrence of events among participants receiving either dose level, not whether treatment improves diabetic macular edema. The registry identifies a randomized, two-arm design, so the planned comparison is between participants assigned to the 2.4mg and 4.0mg dose levels. Both arms use the same administration device and route described in the record.

The posted counts describe how many participants in each arm were recorded as having events. They do not, by themselves, establish whether one dose level has a higher or lower underlying risk. Interpretation also depends on event definitions, observation, missing information, and the uncertainty around the estimates. Those details are not all reported in the registry record. No efficacy outcome results or formal comparative analysis are reported in the material available for this trial.

3. Trial Design

This was a Phase 2, randomized, multicenter, parallel-group trial with single masking. It evaluated two dose levels of suprachoroidal triamcinolone acetonide administered with the Oxulumis® ophthalmic administration device in participants with diabetic macular edema. The registry lists an enrollment of 25 and a completed status. The recorded study start was 2022-08-31, and the primary completion date was 2023-11-30.

The two arms were single-treatment regimens. One used 2.4mg/60µl Triesence® and the other used 4.0mg/100µl Triesence®. Random allocation is intended, in general, to make treatment groups comparable with respect to both measured and unmeasured characteristics on average. In a trial of this size, however, chance differences between groups can remain, and the posted record does not describe participant characteristics in enough detail to assess their balance.

Single masking means that at least one party was masked, but the registry record does not identify which party or describe how masking was maintained. The allocation ratio, sequence-generation procedure, concealment procedures, and any stratification factors are not reported in the registry record. The record also does not give a sample-size justification or identify any planned interim analyses.

The primary outcome concerns the frequency of ocular and systemic adverse events, including serious and treatment-emergent non-serious events. Its stated timeframe is Day 0 up to Week 24, described as the per-protocol individual trial duration per participant. The record defines treatment-emergent ocular adverse events as ocular events emerging following the start of administration at Visit 2, Baseline, Day 0. A corresponding definition for systemic events is not reported in the registry record.

The registry lists six outcome measures, but the primary outcome combines several categories or time points and is not reduced to one comparison in the posted analysis. No analyses are listed. Consequently, the design description establishes the randomized dose comparison and the safety focus, but does not supply a complete statistical analysis plan or a single inferential estimate for the primary outcome.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Safety Analysis set of subjects enrolled - Number of Patients with at least 1 event

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: Safety Population (all subjects eligible and randomized)

6. Primary Endpoint

The registry lists the primary outcome as Frequency of Ocular Adverse Events, Systemic Adverse Events, Serious, and Treatment-emergent Non-serious Adverse Events, measured over Day 0 up to Week 24 (per protocol individual trial duration per participant).

The primary endpoint is titled as the frequency of ocular adverse events, systemic adverse events, serious events, and treatment-emergent non-serious adverse events. The unit is participants, and the record describes the population as the safety analysis set of enrolled subjects, counted as the number of patients with at least one event. The timeframe is Day 0 up to Week 24, corresponding to the stated individual trial duration per participant.

Counting participants with at least one event is a way to describe how many people experienced an event during observation, rather than how many separate event episodes occurred. This can be useful in safety monitoring because it gives a participant-level view of events during exposure. It does not describe the number of recurrences, how long events lasted, their severity beyond the stated categories, or their relationship to treatment unless those details are separately collected and reported.

The registry defines a treatment-emergent ocular event as an ocular event emerging after administration begins at Visit 2, Baseline, Day 0. It does not report a parallel definition for systemic events in the record. The posted results distinguish serious adverse events from other adverse events, but do not provide a single combined primary-endpoint estimate or a formal comparison across the listed categories. The endpoint is therefore best read as a set of safety counts, not as a unified measure of benefit or a complete account of all clinical outcomes.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count measure?

The endpoint is expressed as the number of participants with at least one event. This participant-level measure answers whether an event was recorded for a person during the observation period. It is different from counting every event episode: a participant with repeated events may still contribute to a count of participants with at least one event only once. The registry record does not report episode counts or event-level details alongside the posted group counts.

Participant counts are straightforward to understand, but they are not a complete description of risk on their own. Interpretation depends on the group being observed, the event definition, and the period over which events are collected. The risk sets are listed as 13 for the 2.4mg arm and 12 for the 4.0mg arm. These denominators are the context for the posted counts; no percentages or comparative estimates are reported in the registry record.

Why does randomization matter?

Random allocation is a general method for reducing systematic differences between treatment groups. If carried out as intended, it helps make the groups comparable on average at the time of assignment, including for factors that were not measured. It does not guarantee that small groups will be identical, and it does not eliminate uncertainty in the outcome estimates. The registry identifies the study as randomized but does not report the sequence-generation or allocation-concealment details.

The parallel-group design assigns participants to one dose-level arm and follows them in that group. This design permits a comparison between the assigned regimens, but the reported results remain descriptive unless an appropriate analysis is specified and presented. The record lists zero analyses and does not report a model, adjustment factors, a treatment-effect estimate, a confidence interval, or a p-value for the primary outcome.

How should uncertainty be understood?

A confidence interval is a general statistical tool for expressing the uncertainty around an estimated quantity. A narrower interval indicates greater precision under the assumptions of the method; a wider interval indicates less precision. An interval is not a guarantee that the true value lies within its limits, and it does not by itself establish clinical importance. No confidence intervals are reported for these adverse-event counts in the registry record, so their statistical precision cannot be quantified from the posted results.

A hypothesis test is another general method for assessing how compatible data are with a specified null hypothesis under a statistical model. A p-value does not measure the size or importance of an effect, and a non-significant result would not establish that two treatments are equivalent. Here, no p-value or formal test for the reported safety counts is listed. The descriptive counts should therefore not be recast as evidence from a completed significance test.

Why is endpoint structure important?

The primary outcome title names ocular and systemic events as well as serious and treatment-emergent non-serious events. These are distinct categories that may have different definitions and clinical interpretations. Combining several categories into one outcome can require explicit rules about which events count, whether a participant can appear in more than one category, and how time points are handled. The registry record does not provide a single reduced comparison or a complete analysis plan for the categories. It is therefore appropriate to keep the reported serious and other event counts distinct rather than construct a new summary.

Multiplicity is a general concern when several outcomes, categories, time points, or comparisons are tested. Performing many tests can increase the chance of at least one apparently notable result even when no true differences exist. Statistical plans may address this through prespecified primary comparisons or adjustment procedures, but the registry record does not report a multiplicity strategy. No multiplicity adjustment should be inferred from the available counts.

What can planning calculations tell us?

Sample-size calculations are planning tools. They describe how many participants might be needed under specified assumptions about the outcome, effect to detect, significance level, allocation, and statistical method. They are not estimates of what the trial observed and are not observed power. The registry record does not report a sample-size justification for the trial. Any planning illustration based on an observed effect is not a substitute for the study's original design rationale and should not be interpreted as a retrospective test of whether the trial was adequately powered.

General planning calculations can be particularly sensitive when the observed outcome is rare or when counts are small. The assumptions used to translate an effect into a target sample size may not hold in a later study. The registry's stated planning assumptions refer to a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the effect expressed as a fraction of the observed effect. Those assumptions describe an illustration, not a reported trial design justification. No planning estimates are included in the registry record reported for this analysis.

How do missing data and analysis populations affect interpretation?

Missing outcome information can affect safety counts if participants do not complete follow-up or if events are not fully recorded. A complete-case analysis, an approach that treats participants with missing outcomes in a particular way, or another strategy can yield different summaries. The registry record does not report a missing-data method, the amount of missing outcome information, or details about withdrawals and follow-up completeness. The stated safety analysis set is enrolled subjects, but the handling of any incomplete observations is not described.

The posted counts should thus be read as the recorded observations in the registry, not as a complete reconstruction of every participant's follow-up. General statistical explanations cannot resolve what happened to participants with unreported or unavailable outcome data. Those details, along with the event definitions and prespecified analysis, would be needed for a fuller assessment of the safety evidence.

10. Multiplicity, Missing Data and Analysis Populations

The registry identifies a primary outcome that includes several event categories and lists six outcome measures overall. The primary outcome was not reduced to one comparison, and no analyses are listed. A multiplicity procedure, such as a prespecified hierarchy or adjustment for multiple comparisons, is not reported in the registry record. Without such information, the separate safety counts should not be interpreted as a collection of confirmatory tests or combined into a new primary result.

The described safety analysis set consists of enrolled subjects, and the outcome is counted as participants with at least one event. The record does not state how missing observations were handled, how many participants lacked follow-up information, or whether all enrolled participants contributed the same observation period. The timeframe is Day 0 up to Week 24 per protocol individual trial duration per participant, but follow-up completeness is not reported in the registry record.

The record does not report an analysis population beyond the stated safety set, nor does it describe exclusions from that set. It also does not identify an intention-to-treat or per-protocol analysis for a comparative endpoint. A complete statistical interpretation would require the planned analysis populations, missing-data rules, definitions for each outcome category, and the prespecified approach to multiplicity. These items are not reported in the registry record, so no assumptions about them should be made.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

For serious adverse events, the posted count is 0 affected among 13 at risk in the 2.4mg arm and 0 affected among 12 at risk in the 4.0mg arm. Deaths are recorded as 0 in both arms. These figures describe what was recorded during the trial's stated observation period; they do not prove that serious events cannot occur or establish a zero underlying risk.

For other adverse events, 12 participants were recorded as affected among 13 at risk in the 2.4mg arm, while 9 were recorded as affected among 12 at risk in the 4.0mg arm. These are counts of participants, not event episodes. The registry does not list the specific other events, their severity, or their timing in the information, so the counts should not be interpreted as a detailed clinical profile.

Because the groups are small and no comparative estimates or confidence intervals are reported, the counts alone do not establish that one dose level has a different safety profile from the other. The empty list of top serious event terms should not be read as a separate analysis; the group counts themselves record no serious events.

12. What the Result Does, and Does Not, Mean

The posted results describe adverse-event participant counts for two randomized dose-level groups receiving suprachoroidal triamcinolone acetonide with the Oxulumis® device. No participants were recorded as having a serious adverse event in either arm, and deaths were recorded as 0 in each arm. Other adverse events were recorded in both arms. These observations concern the trial participants and the stated follow-up period, not all people with diabetic macular edema or every possible treatment setting.

The results do not show whether either dose level improves diabetic macular edema. The primary question is framed around safety and tolerability, and no efficacy estimate is reported in the registry record. They also do not establish that the two dose levels are equivalent, or that one has a lower risk of adverse events. Such conclusions would require an appropriate prespecified comparative analysis and estimates with uncertainty, neither of which is reported.

Zero serious events in a small study is not proof that the true risk is zero. Likewise, the observed counts for other adverse events are not a complete account of severity, duration, recurrence, or causality. The record does not report event-level details, missing-data handling, or a confidence interval around the counts. Those limitations restrict how precisely the results can be generalized or compared.

The trial can be understood as a randomized safety evaluation of two dose levels, with the device and route used in both arms. Its observations are useful as a record of what was reported during the trial, but they are not a substitute for a larger body of evidence or a complete statistical analysis. No treatment recommendation follows from these data alone.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between a randomized design and a definitive comparative result. Randomization supports a planned comparison between the two dose levels, but random allocation does not remove uncertainty, particularly when the enrollment is 25. The posted safety counts are useful descriptions, while the absence of a reported comparative analysis limits what can be concluded about differences between groups.

The endpoint also illustrates why clear outcome definitions matter. The primary outcome names several safety categories, yet the posted information gives separate counts for serious and other adverse events and does not reduce the endpoint to one comparison. Readers need to know exactly how events are classified, how overlapping categories are handled, and how the analysis was planned before treating a collection of counts as a single result. Those details are not fully reported in the registry record.

Zero serious events can be reassuring as an observation but remain statistically imprecise in a small trial. Rare outcomes are difficult to characterize when few participants are observed, and a zero count does not imply absence of underlying risk. Reporting uncertainty, follow-up completeness, and event-level detail would help readers understand the boundaries of the evidence.

Finally, the record separates the trial's safety focus from claims about efficacy. Keeping that distinction clear is important when interpreting early clinical research. The study provides a record of adverse events observed after two dose-level regimens, but does not provide a formal estimate of comparative safety or an efficacy result in the posted analyses.

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 enrollment was 25, and the posted risk sets are 13 and 12. These small groups limit the precision with which uncommon adverse events can be characterized. The absence of serious events in the recorded counts does not exclude the possibility of rare events, and the record provides no confidence intervals to show uncertainty around these observations.

The primary endpoint includes several categories, but the registry indicates that it was not reduced to a single comparison. No analyses are listed. Consequently, there is no reported treatment-effect estimate or formal test for the primary outcome. The analysis model, any covariate adjustment, and the handling of multiple categories or comparisons are not reported in the registry record.

The event details are limited. The registry reports counts for serious and other adverse events but does not list the other event terms, severity, timing, duration, recurrence, or attribution in the information reported for this analysis. It defines treatment-emergent ocular adverse events, but a corresponding systemic-event definition is not reported in the registry record.

Follow-up is described as Day 0 up to Week 24, but the record does not report how complete follow-up was, how missing observations were handled, or whether participants had different amounts of observation. It also does not describe withdrawals or exclusions from the safety analysis set. The single-masked design is reported, but which parties were masked and how masking was maintained are not stated.

The trial compares two dose levels, with no placebo or untreated arm described in the registry record. The posted information therefore does not provide a comparison with no treatment, and it does not report an efficacy result for diabetic macular edema. The record also does not provide a sample-size justification, so the rationale for the enrollment target cannot be assessed.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Oxulumis® Suprachoroidal Microcatherization of Triesence® in Diabetic Macular Edema — NCT05512962.
• Registry record last updated 2024-11-20; status last verified 2024-10.
• Results first posted 2024-11-20.
• 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.

18. Record Summary

NCT05512962 is a completed Phase 2, multicenter, randomized, single-masked, parallel-group trial of suprachoroidal triamcinolone acetonide administered using the Oxulumis® device in participants with diabetic macular edema. The registry records enrollment of 25, a study start of 2022-08-31, and a primary completion date of 2023-11-30.

The two arms received a single treatment with 2.4mg/60µl or 4.0mg/100µl Triesence®. The primary outcome concerns ocular and systemic adverse events, including serious and treatment-emergent non-serious events, from Day 0 up to Week 24. The posted counts record 0 serious events in each arm and other adverse events in both arms; deaths were recorded as 0 in both arms.

The registry does not list analyses, a single reduced comparison for the primary outcome, or a sample-size justification. The posted counts are descriptive and do not establish a comparative treatment effect or provide an efficacy result.