Study of Safety and Performance of a Novel Ocular Lubricant in Subjects With Dry Eye Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study of Safety and Performance of a Novel Ocular Lubricant in Subjects With Dry Eye Disease (NCT06444516), a na na study. The primary outcome is Change From Baseline in Impact of Dry Eye on Everyday Life - Symptom Bother (IDEEL-SB) Questionnaire Score at Day 30.

2. Clinical Question

The study was intended to evaluate and demonstrate safety and performance of the ocular lubricant in adult subjects with dry eye disease. Its single-arm structure means the primary outcome is a change over time within the treated group, not a difference between FID123300 and a control group. Such a change can describe participants’ reported experience during the study, but without a comparator it cannot by itself separate a treatment-associated change from other explanations, such as changes over time or the influence of study participation.

3. Trial Design

This was a single-arm study of FID123300 ocular lubricant in adults with dry eye disease. The registry lists allocation as not applicable and masking as none. There was no randomized control arm, and the study model was recorded as a single group. The registry lists an enrollment of 188. The study status is completed.

The treatment schedule began with one drop in each eye on the morning of Day 1. On Days 2-30, the instructions were at least one drop in each eye four times a day. The registered primary outcome was assessed at baseline and Day 30. Baseline was defined as the last non-missing value before the start of study treatment.

The primary outcome population was the Safety Analysis Set with data at both visits. The registry also reports safety-event figures for a group of 161 participants at risk. These figures do not establish that the primary outcome analysis included every participant listed in the enrollment field. Further details about participant flow, reasons for missing visits, and how participants entered or left the analysis population are not reported in the registry record.

The registry lists the study start as 2024-09-04 and primary completion as 2025-02-07. It identifies Alcon Research as the sponsor and lists the phase as not applicable. Because the study was unmasked and had no control group, outcome assessments were made without a masked comparison. The registry record does not report additional details about the study protocol, the rationale for the design, or a statistical plan specifying how the primary outcome was to be analyzed.

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: Of the 188 subjects enrolled in the study (consented), 27 were exited as screen failures. This reporting group includes all subjects exposed to the study product (161).

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Safety Analysis Set with data at both visits

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 Analysis Set

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline in Impact of Dry Eye on Everyday Life - Symptom Bother (IDEEL-SB) Questionnaire Score at Day 30, measured over Baseline (Day 1); Day 30.

The primary endpoint was change from baseline in the Impact of Dry Eye on Everyday Life Symptom Bother questionnaire score at Day 30. The module contains 20 patient-reported questions about dry-eye symptoms. For each question, the participant selected one response for both eyes. Response options ranged from no symptom or not applicable to a symptom that bothered the participant very much, with intermediate options describing different degrees of bother.

The overall score was calculated as the mean of the non-missing item scores multiplied by 25, on a scale from 0 to 100. The registry defines baseline as the last non-missing value before treatment began, and defines change as the Day 30 score minus the baseline score. Therefore, a negative change represents improvement in the score, or less symptom bother; the sign should not be reversed when interpreting the endpoint.

A patient-reported symptom measure addresses participants’ experience directly, rather than relying only on an examination or an observer’s assessment. It is relevant to a study asking about symptom bother in everyday life. However, the registry record does not report the observed mean change, its standard deviation, a confidence interval, or a test result. The direction of the endpoint is defined, but the magnitude and precision of the observed result cannot be described from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary measure is continuous: it is a score on a scale, and the registered summary parameter is the mean. For a continuous outcome, a mean change describes the average difference between each participant’s Day 30 score and baseline score in the analysis population. The score itself is constructed from questionnaire responses; it is not a count of events. The registered definition makes negative values favorable in the limited sense that they correspond to less symptom bother on this measure.

What does a mean change describe?

A mean summarizes the center of a set of numeric observations. In a change-from-baseline analysis, each participant’s change is defined by subtracting the baseline score from the Day 30 score. The reported mean change, if available, would describe the average of those individual changes for the included participants. It would not mean that every participant changed by that amount, nor would it show the distribution of individual experiences.

For example, participants can have different starting scores and different changes while contributing to one group mean. The mean alone does not show whether changes were similar across participants, whether some improved while others worsened, or whether a small number of observations influenced the average. Those questions require distributional detail that is not reported in the registry record.

Why report a standard deviation?

The registered dispersion measure is the standard deviation. As a general statistical concept, a standard deviation describes how spread out individual observations are around their mean. It is different from a standard error, which describes uncertainty in an estimated mean. A standard deviation therefore helps characterize variability among participant scores, while a confidence interval for the mean would address precision in estimating the group average. Neither measure replaces the other.

The registry record identifies standard deviation as the dispersion measure for the continuous outcome but does not report its value. It also does not report a confidence interval or the details of an inferential test. Consequently, the mean’s precision and the variability of individual changes cannot be assessed from the record.

How should the score be read?

The IDEEL Symptom Bother module has 20 questions and a score range from 0 to 100. Responses are recorded for both eyes together, and the overall score uses the mean of non-missing item scores multiplied by 25. This scoring rule turns the item responses into a single score. The registered change is Day 30 minus baseline, so the order of subtraction determines the sign. A negative value is defined as an improvement; a positive value would indicate greater symptom bother compared with baseline.

The score is a patient-reported outcome. In general, patient-reported outcomes capture a participant’s report of symptoms or experience, which can complement other forms of clinical assessment. A score should be interpreted in light of its measurement scale and the specific questions it represents. The registry does not report a threshold for a clinically meaningful change, so no threshold-based interpretation can be made from this record.

What can a single-group analysis establish?

A single-group analysis can describe the observed outcome over time among the participants included in the analysis. A baseline-to-follow-up change is not a randomized treatment contrast. In general, a control group helps estimate what might have happened over the same period without the intervention under study. Without such a group, change may also reflect time, measurement variation, study participation, or other influences. This design limitation applies even when the outcome is measured carefully.

Masking is listed as none. In general, masking can reduce the influence of knowledge about treatment assignment on behavior, reporting, or assessment. This study had one treatment group and no masked assignment. The registry does not report further methods to address potential influences on a patient-reported outcome.

What do planning illustrations mean?

Planning calculations based on an observed effect are not evidence that an effect was observed with a particular probability, and they are not observed power. The stated planning assumptions describe a hypothetical two-arm design with equal allocation, a two-sided significance level of 0.05, and a normal approximation; the effect to detect is a stated fraction of the observed effect. The registry record does not report the observed primary-outcome effect needed to interpret such planning values. Moreover, those assumptions describe a two-arm planning scenario, whereas this trial was single-arm and had no randomized comparison. Such calculations should therefore be understood as illustrations for a hypothetical design, not as a description of the trial’s actual design or findings.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("FID123300")
n   <- c(158)
m   <- c(-16.95)
sp  <- c(13.12)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  FID123300  158  -16.950  13.120  -19.012  -14.888

11. Multiplicity, Missing Data and Analysis Populations

The registry lists one outcome measure and one analysis. It does not report whether other analyses were conducted, whether outcomes were ranked in a prespecified testing sequence, or whether any adjustment for multiple comparisons was planned. Multiplicity refers, in general, to the increased chance of at least one false-positive result when several hypotheses are tested. Whether adjustment is needed depends on the analysis plan and the set of tests being interpreted; the registry record does not provide enough detail to assess that issue beyond its listing of one outcome measure and one analysis.

The primary outcome population is the Safety Analysis Set with data at both visits. This definition indicates that both baseline and Day 30 data were required for the primary outcome population. The registry does not report the missing-data method, how partially completed questionnaires were handled beyond the score’s use of non-missing items, or whether any sensitivity analyses were performed. It also does not report the number of participants with missing primary outcome data or the reasons for missingness. Missingness matters because the available observations may differ from those that are unavailable; without details, the extent and possible impact cannot be evaluated.

The enrollment figure is 188, while the safety-event group lists 161 at risk. The primary outcome population is described separately and does not include a reported count. These figures should not be treated as interchangeable denominators. The registry record does not provide a participant-flow account linking enrollment, safety assessment, and the primary outcome analysis.

The record does not report a statistical analysis plan, an imputation approach, interim analyses, or a sample-size justification. Those details should not be inferred from the outcome listing or from planning illustrations. The primary result’s numerical estimate and uncertainty are also not reported in the registry record.

12. 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 safety figures list 161 participants at risk in the FID123300 group. For that group, the registry reports zero participants with a serious adverse event, zero with other adverse events, and zero deaths. No serious adverse event terms are listed. These are counts for the safety group stated in the registry; they are not a statement about the primary outcome population.

A count of zero means that no events of the specified kind were recorded in the reported group. It does not demonstrate that the underlying risk is absent, and it does not show what would have occurred in a comparison group. In general, event counts are interpreted in relation to the number of participants monitored, the duration and completeness of follow-up, and the definitions used to classify events. The registry record does not report additional detail about event definitions, monitoring completeness, or follow-up beyond the listed outcome information.

There is no comparator arm, so these safety figures cannot support a controlled comparison of event rates. They describe the posted observations in the FID123300 group only. The record also does not report additional breakdowns by event type, severity, or timing beyond the serious-event, other-event, and death counts.

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

The registry record identifies a completed, single-arm study of FID123300 ocular lubricant in adults with dry eye disease. It defines the primary outcome as change from baseline to Day 30 in IDEEL Symptom Bother score and explains how that score is constructed and interpreted. In particular, negative change represents less symptom bother. These details clarify what the outcome was intended to measure.

The registry record does not report a numerical primary-outcome result. It therefore does not show whether the mean score increased or decreased, the size of any change, how variable participant changes were, or how precise an estimate might be. No conclusion about the magnitude of symptom change can be drawn from the endpoint definition alone.

Even a reported within-group change would not, by itself, establish that the intervention caused the change. The study had one treatment group, no randomized comparator, and no masking. A before-and-after result describes what happened among participants with the required observations; it does not provide the same evidence as a comparison against a concurrent control group.

The posted safety counts report no serious adverse events, other adverse events, or deaths in the listed group. Those counts describe the observations for the reported safety denominator. They do not establish that risks are absent, and the lack of a comparator prevents a direct comparative safety interpretation.

The record also leaves important analysis details unspecified, including missing-data handling, the numerical primary result, and its uncertainty. As a result, the registry entry provides information about the question, design, scoring, and posted safety counts, but not enough information to quantify the primary outcome or judge its precision.

14. Why This Trial Matters Statistically

This study illustrates why a clear endpoint definition is important. The registry specifies the questionnaire, its response scale, how item responses contribute to the overall score, the baseline definition, the timing of follow-up, and the direction of change associated with less symptom bother. These details make it possible to understand what a reported change would mean and avoid misreading a negative value as worsening.

It also demonstrates the distinction between describing change and estimating a comparative effect. In a single-arm study, a baseline-to-follow-up analysis can describe participants’ reported experience during the study. Without a comparator, it cannot separate an intervention-associated change from other influences over time. That distinction is central when interpreting clinical research, particularly for a patient-reported measure.

The record also shows why estimates need uncertainty information. A mean change without a standard deviation or confidence interval would give limited information about variability and precision; in this case, the registry record does not report the mean change itself. The endpoint’s scale and direction alone cannot establish the observed result.

Finally, the safety counts need their own context. They are reported for a specified group and should not be confused with the primary outcome analysis population. A zero count is an observation, not proof that an event is impossible. Reading the design, endpoint, analysis population, and safety denominator separately helps keep descriptive findings distinct from causal or population-wide claims.

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.

The main limitation for interpreting the primary outcome is that its numerical result is not reported in the registry record. The mean change, standard deviation, confidence interval, and p-value are absent, so neither the direction and size of the group-level change nor its precision can be assessed.

The study was single-arm, with allocation listed as not applicable and no masking. There was no randomized control group for comparison. Any change from baseline to Day 30 would therefore be descriptive of the observed group and would not isolate the effect of the lubricant from other possible influences. Because the outcome was patient-reported and the study was unmasked, knowledge of treatment could also be relevant to how responses were reported; the registry does not describe methods addressing this possibility.

The primary outcome population required data at both visits, but the registry does not report its size, the amount of missing outcome data, reasons for missingness, or the method used to handle missing observations. The outcome score allows calculation from non-missing questionnaire items, but further details about incomplete questionnaires are not reported. The enrollment figure and the safety denominator do not resolve these uncertainties.

The registry also does not report a clinically meaningful change threshold, a statistical analysis plan, or a sample-size justification. It does not provide the information needed to evaluate the practical importance of a score change or the planned analysis in detail. The listed safety counts are descriptive and have no control-group comparison. These limitations restrict interpretation to the registered design and posted information.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study of Safety and Performance of a Novel Ocular Lubricant in Subjects With Dry Eye Disease — NCT06444516.
• Registry record last updated 2026-02-18; status last verified 2026-01.
• Results first posted 2026-02-18.
• 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.

19. Record Summary

NCT06444516 is a completed, single-arm study sponsored by Alcon Research. It enrolled 188 participants and evaluated FID123300 ocular lubricant in adults with dry eye disease. The registered schedule was one drop in each eye on the morning of Day 1, then at least one drop in each eye four times a day on Days 2-30. The study was unmasked and had no randomized comparator.

The primary outcome was change from baseline to Day 30 in IDEEL Symptom Bother score, with negative change defined as less symptom bother. The primary outcome population was the Safety Analysis Set with data at both visits. The numerical primary result and its uncertainty are not reported in the registry record.

For the listed safety group of 161 participants at risk, the registry reports zero serious adverse events, zero other adverse events, and zero deaths. The record does not report further analysis details or a controlled comparison.