Efficacy and Safety of SHJ002 Ophthalmic Solution in the Treatment of Corneal Erosion in Sjogren's Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy and Safety of SHJ002 Ophthalmic Solution in the Treatment of Corneal Erosion in Sjogren's Patients (NCT06381986), a randomized phase 2 study. The primary outcome is Change From Baseline to Week 12 in Total Corneal Fluorescein Staining.

2. Clinical Question

The central statistical question is whether the posted staining outcomes describe a different pattern of change for SHJ002 and vehicle over the study period. The primary outcome is a continuous score on the NEI grading scale, with higher scores indicating more severe staining. A negative change therefore denotes a reduction in the score, while the size and precision of the arm-level estimates matter when describing the observed pattern.

The record also lists change in inferior corneal fluorescein staining and change in SANDE score as secondary outcomes. Results for those outcomes are not reported in the registry record. The available results therefore address only the primary staining measure and do not provide a complete account of all planned or recorded clinical assessments.

The study was randomized and used a vehicle comparator, but the result record does not supply a direct statistical comparison between the arms. The group estimates alone do not establish the magnitude or statistical uncertainty of an arm-to-arm contrast. The clinical question can be framed, but the posted results answer it only in part.

3. Trial Design

This was a randomized, parallel-group, phase 2 study, marked completed in the registry record. The listed enrollment was 116 participants. The primary outcome is reported for the FAS population, with 58 participants listed in each arm. The registry record does not explain the relationship between enrollment and the analysis population or give a participant flow account.

Participants were assigned to SHJ002 or vehicle. The SHJ002 arm received SHJ002 ophthalmic solution, and the comparator arm received vehicle; both were administered topically to each eye twice daily for 12 weeks. The masking designation is quadruple. The record does not identify which groups of people were masked or describe how masking was implemented.

The study start date is 2024-06-28, and the primary completion date is 2025-04-10. These dates describe the registry record's study milestones; they do not provide information about individual follow-up duration or the timing of each participant's assessments beyond the stated 12-week endpoint timeframe.

The listed primary outcome is change from baseline to Week 12 in total corneal fluorescein staining. Two secondary outcomes are named: change in inferior corneal fluorescein staining and change in SANDE score. Three outcome measures are listed in total, but results for the secondary measures are not reported in the registry record.

The registry record does not report the method used to generate or conceal the random allocation, the planned sample-size justification, or the detailed analysis plan. It also does not report whether interim analyses were planned or performed. The listed enrollment and allocation therefore describe the study's recorded size and broad design, not the rationale for those choices.

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

Analysis population for the primary outcome, as defined in the registry: FAS population

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.

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline to Week 12 in Total Corneal Fluorescein Staining, measured over Baseline to Week 12.

The primary endpoint is change from baseline to Week 12 in total corneal fluorescein staining. The score is assessed using the NEI grading scale, which ranges from 0 to 15 points; higher scores indicate more severe staining. The endpoint is continuous, so its result describes a degree of change rather than a count of participants who did or did not meet a threshold.

Fluorescein staining is used to assess corneal surface staining. Repeating the assessment at baseline and Week 12 allows the analysis to describe how the score changed during the treatment period. A negative change indicates a lower score at Week 12 than at baseline, consistent with less severe staining on this scale. This interpretation concerns the score itself and does not, on its own, establish the clinical importance of a particular change.

The registry record reports least squares mean changes for the FAS population, with 95% confidence intervals for each arm. Least squares means are a statistical summary that can be model-based; the specific model, covariates, and handling of repeated measurements are not reported in the registry record. The record does not provide a direct between-arm estimate for this endpoint.

The endpoint is relevant to the study's stated focus on corneal erosion in patients with Sjogren's syndrome, but its result is only one measure of outcome. The registry record also names inferior corneal fluorescein staining and SANDE score as secondary measures, with their results not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a continuous measure, and the registry record reports a least squares mean change for each arm. The estimates describe average change in total corneal fluorescein staining from baseline to Week 12. They are not individual participant values, and they do not show how widely responses varied between participants. A measure of participant-level dispersion is not reported in the registry record.

What does a least squares mean represent?

In general, a least squares mean is an estimate produced from a statistical model. Depending on the model, it may adjust for specified factors or account for features of the data. That is a general description, not a statement about the model used in this study. The registry record does not report the model specification, any adjustment variables, assumptions, or method for accounting for repeated measurements. It is therefore not possible to determine from the record whether or how the arm estimates were adjusted.

The outcome is change from baseline, which compares the Week 12 score with the baseline score for the analysis. Because the measure is a score on a defined scale, a negative change means the measured score decreased. In this study's scale description, higher scores indicate more severe staining. Interpretation should remain tied to this measure: a change in a score is not itself a description of every aspect of the condition or a complete account of participants' experience.

How should the confidence intervals be read?

A 95% confidence interval expresses the uncertainty associated with an estimate under the statistical method used. In general, if the same procedure were repeated across many comparable samples, intervals constructed in that way would cover the quantity being estimated at the stated rate. The interval does not mean that there is a 95% probability that the particular fixed effect is inside it, and it is not a range of individual participant outcomes.

The intervals in the registry record are attached to the arm-level least squares mean changes. They describe uncertainty around each arm's estimated mean change. They do not directly describe uncertainty around the contrast between arms. Comparing two separate confidence intervals is not equivalent to estimating the randomized treatment contrast, because that contrast has its own estimate and uncertainty. Neither a contrast estimate nor its confidence interval is reported in the registry record.

Why is a direct comparison important?

In a randomized parallel-group study, the treatment question concerns the difference in outcomes between the assigned groups. Arm-specific summaries can show what was observed within each group, but the comparative effect requires a direct analysis of the groups. That analysis should report the contrast and its uncertainty, using the trial's stated statistical approach. Here, values for a two-arm comparison of the continuous endpoint are identified as missing or not estimable, and no analyses are listed in the posted results. A comparative conclusion cannot be reconstructed from the arm estimates without computing an unreported result.

This distinction also matters when interpreting changes from baseline. A within-arm change can reflect multiple influences over the follow-up period, while randomization is intended to support a fair comparison between assigned groups. The group-specific change estimates alone do not separate treatment-related differences from changes that may also occur in the comparator arm.

What does the sample-size illustration mean?

The sample-size calculations described for the record are planning illustrations based on the observed effect, not the trial's own design justification. Their stated assumptions include a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For non-time-to-event outcomes, the planning quantity is participants per arm. These calculations are not observed power and do not show the probability that this completed study detected an effect. The registry record does not report the original sample-size rationale.

More generally, a planning calculation asks how many participants might be needed under chosen assumptions to detect a target effect with a specified statistical design. Such a calculation depends on the target effect, variability, analysis method, and other assumptions. It cannot repair a missing comparative result or establish what the trial was designed to detect unless the actual protocol assumptions are reported. No additional assumptions should be attributed to this study beyond those stated in the record.

What can be said about significance?

The registry record does not report a p-value or a direct treatment-versus-vehicle test for the primary outcome. A confidence interval for an arm's change is not a test of the difference between arms. Therefore, the posted values support a description of arm-level estimates and their precision, but do not give a reported significance test for the primary comparative question. The exact analysis plan and the method used to derive the least squares means are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists one primary outcome and two secondary outcomes. It does not report a multiplicity strategy, such as whether the primary and secondary outcomes were tested in a hierarchy or whether the error rate was adjusted across multiple tests. Without that information, the statistical interpretation of any collection of outcome tests cannot be assessed. The two secondary outcomes are named, but their results are not reported in the registry record.

Missing-data methods are also not reported. The registry record does not describe how missing Week 12 measurements were handled, whether participants with incomplete assessments were included, or whether sensitivity analyses were conducted. Enrollment and the arm-level analysis counts do not explain whether all randomized participants contributed outcome data or how missing observations affected the estimates.

The primary outcome is described for the FAS population, but the registry record does not define the FAS or state the inclusion rules used for it. It does not report a separate per-protocol or safety analysis population, nor does it describe deviations from assigned treatment. Thus, the extent to which the outcome estimates represent all randomized participants cannot be determined from the registry record.

The record lists 58 participants in each arm and marks the study completed, but does not report a detailed participant flow, withdrawals, or reasons for discontinuation. These are important context for understanding endpoint availability and potential bias. The absence of these details does not establish that data were missing; it means the registry record does not report how completeness was evaluated or handled.

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 5% reporting threshold.

The posted safety figures report zero participants with serious adverse events in the SHJ002 arm and zero in the vehicle arm. The figures also report zero participants affected by other adverse events in each arm, and zero deaths in either arm. Each arm lists 58 participants at risk for the serious and other adverse event summaries.

These are counts for the safety categories reported in the registry record. A zero count means no events were recorded in that category for the listed group in the posted results; it does not establish that the underlying risk is zero. The record does not provide event terms for serious adverse events, so it does not describe the types of serious events considered. The list of top serious-event terms is empty.

The registry record does not report the adverse-event collection procedures, severity grading, relatedness assessments, exposure-adjusted summaries, or the timing and duration of safety follow-up. It also does not provide a narrative explaining whether the other adverse event counts include all events or participants with at least one event. As a result, the figures give a limited count-based description rather than a comprehensive account of safety.

These findings concern the recorded study participants and observation period; they should not be taken as proof that no adverse events can occur. The registry record does not report a statistical comparison of safety outcomes between arms.

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

The posted primary outcome results show a negative estimated change in total corneal fluorescein staining in both arms, with a more negative arm-level estimate for SHJ002 than for vehicle. Because higher values on the NEI grading scale indicate more severe staining, negative change corresponds to a lower score at Week 12 than at baseline. The confidence intervals describe uncertainty around each arm's estimated mean change.

The results do not include a direct estimate of the difference between SHJ002 and vehicle, a confidence interval for that difference, or a p-value for the primary comparison. Therefore, the group-specific estimates do not establish the size or precision of the treatment contrast. No efficacy conclusion should be inferred from comparing arm estimates alone.

The record also does not report results for the named secondary outcomes, change in inferior corneal fluorescein staining and change in SANDE score. It consequently does not show whether those measures align with the primary outcome pattern. The primary endpoint is a single assessment at Week 12, and the registry record does not report outcomes beyond that timeframe.

The result does not describe all participants' experiences or the range of individual responses. A mean change can coexist with variation between participants, but a dispersion measure is not reported. The record also does not describe missing-data handling, the detailed definition of the analysis population, or the model used for the least squares means. Those gaps constrain assessment of the statistical analysis.

Finally, the study is identified as phase 2 and completed, but the registry record does not report the trial's sample-size justification or detailed statistical plan. The planning illustrations based on the observed effect are not evidence of observed power or a substitute for the trial's original design rationale. The most defensible interpretation is limited to the posted arm-level outcome estimates and the specific measurement period described.

13. Why This Trial Matters Statistically

This study raises a useful statistical distinction between describing changes within randomized groups and estimating a difference between those groups. The registry record reports least squares mean changes for SHJ002 and vehicle, with confidence intervals for each. However, the direct comparison—the quantity that addresses how outcomes differed between randomized arms—is not reported. This makes clear why arm-level results, even when precise, do not by themselves answer the comparative question.

The endpoint also illustrates how scale direction affects interpretation. The NEI grading scale is described as ranging from 0 to 15 points, with higher scores indicating more severe staining. Accordingly, a negative change is a decrease in the score. Explaining the direction of a measure is essential before interpreting estimates, particularly when results are expressed as change from baseline.

The record identifies an analysis population and a model-based summary, but leaves important analytic details unspecified. The FAS population is not defined, and the least squares mean model is not described. Missing-data handling and multiplicity methods are also not reported. These features are not peripheral: they help readers understand which observations contribute to an estimate and what inferential claims are justified.

Safety reporting provides another lesson in scope. The posted counts for serious and other adverse events, and deaths, are zero in each arm, but the registry record does not describe event ascertainment or follow-up procedures in detail. A zero count is a description of the recorded data, not proof of no possible risk.

Overall, the record is an example of why statistical interpretation depends not only on numerical estimates, but also on the estimand, analysis population, direct comparison, and transparency about methods. Its randomized design is important context, yet the available registry results leave the central comparative estimate and several supporting details unreported.

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 principal limitation of the posted efficacy results is the absence of a direct between-arm estimate for the primary continuous outcome. The registry record gives arm-level least squares mean changes and confidence intervals, but no treatment contrast, interval for that contrast, or p-value. It specifically indicates that values for a two-arm comparison of the continuous endpoint are missing or not estimable. Comparative efficacy therefore cannot be fully assessed from these results.

The analysis methods are not described in enough detail to reproduce or evaluate the primary estimates. The registry record does not report the model specification, adjustment variables, or assumptions underlying the least squares means. It names the FAS population without defining it, and does not describe missing-data handling or sensitivity analyses. A detailed participant flow and reasons for discontinuation are also not reported.

Although two secondary outcomes are listed, their results are not reported in the registry record. A multiplicity approach is not reported, so the relationship between the primary and secondary analyses is unclear. The record also does not report an original sample-size justification, interim-analysis plan, or detailed protocol-level statistical plan.

The endpoint timeframe is baseline to Week 12. Results beyond that period are not reported in the registry record, and the available outcome measure is a staining score rather than a comprehensive description of participants' symptoms or function. The record provides no dispersion measure for individual outcome values. Safety summaries contain zero counts for the listed categories, but the event definitions and collection procedures are not described in detail.

These limitations concern the registry information available for interpretation. They do not establish that the unreported procedures or data were absent from the study itself; they mean that the registry record does not report them.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Efficacy and Safety of SHJ002 Ophthalmic Solution in the Treatment of Corneal Erosion in Sjogren's Patients — NCT06381986.
• Registry record last updated 2025-08-22; status last verified 2025-08.
• Results first posted 2025-08-22.
• 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

The registry record describes a completed, randomized, parallel-group phase 2 study of SHJ002 ophthalmic solution versus vehicle in patients with Sjogren's syndrome and corneal erosion. Enrollment was 116, with 58 participants listed in each arm. Both study solutions were administered topically to each eye twice daily for 12 weeks, and masking is designated quadruple.

The primary outcome was change from baseline to Week 12 in total corneal fluorescein staining on the NEI grading scale. Arm-level least squares mean changes and 95% confidence intervals are reported for the FAS population. A direct comparison between arms is not reported. Two secondary outcomes are listed, but their results are not reported in the registry record.

The posted safety counts show zero participants with serious or other adverse events and zero deaths in each arm. The registry record does not report detailed analysis methods, missing-data handling, multiplicity procedures, or the trial's sample-size justification.