Safety and Efficacy of Bimatoprost/Timolol (Ganfort®) Ophthalmic Solution in Korea: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Safety and Efficacy of Bimatoprost/Timolol (Ganfort®) Ophthalmic Solution in Korea (NCT01976624), a registered phase not stated study. The primary outcome is Number of Participants With Adverse Events and Adverse Drug Reactions.

2. Clinical Question

In statistical terms, the record describes observed event counts in a treated safety population. It does not report an estimated treatment effect against a control, and the primary endpoint combines safety information rather than measuring a comparative benefit. The study also lists change from baseline in intraocular pressure as a secondary outcome, but an IOP result is not reported in the registry record included for this analysis.

The central question is therefore descriptive: what adverse-event counts were recorded among the listed participants treated with Ganfort®, and how much can those counts establish about safety? Counts can identify events recorded during surveillance, but interpretation depends on how events were collected, how complete follow-up was, and what comparison is available. Those details are not reported in the registry record. The findings should be read as observations from this surveillance record, not as a randomized estimate of the drug’s effect or risk.

3. Trial Design

This was a post-marketing surveillance study of bimatoprost/timolol (Ganfort®) in Korea for open-angle glaucoma or ocular hypertension. The registry lists Allergan as the sponsor, enrollment of 756, and a completed status. The listed start is 2009-08 and the primary completion is 2014-01. These dates describe the registry record; they do not establish the follow-up duration for each participant.

The treatment description says that participants received Ganfort® for the stated conditions as per local standard of care in clinical practice. The outcome population is described as all participants treated for on-label indications. The primary safety outcome has a timeframe of up to 51 months. The record does not explain whether that timeframe applied uniformly to every participant or how individual observation periods varied.

The registry describes no randomized comparison. Allocation is not stated, and the available arm information describes Ganfort® without a like-for-like control group. Consequently, the reported event counts are descriptive and cannot by themselves separate treatment-associated events from events that might occur in the underlying population or during routine care.

The primary outcome is the number of participants with adverse events and adverse drug reactions. The registry also lists change from baseline in intraocular pressure as a secondary outcome and indicates two outcome measures, but the IOP result is not reported in the registry record. The record lists zero analyses. It does not report a statistical analysis plan, the method of event ascertainment, handling of missing observations, or a sample-size justification. These omissions constrain interpretation; they should not be filled in by assuming procedures typical of other studies.

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 population included all participants treated for on-label indications.

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 included all participants treated for on-label indications.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Adverse Events and Adverse Drug Reactions, measured over Up to 51 months.

The primary endpoint counts participants with adverse events and adverse drug reactions. An adverse event was defined broadly as an unfavorable and unintended sign, symptom, or disease associated with use of the study drug, regardless of whether it was considered related to the drug. An adverse drug reaction was a harmful and unintended reaction incurred during routine administration or use for which a causal relationship with the drug could not be excluded. The endpoint therefore distinguishes events by the stated relationship criteria, while including events that were not judged treatment-related in the adverse-event definition.

Counting participants is useful in safety surveillance because it describes how many people in the recorded safety population experienced an event under the study’s definitions. It is not the same as counting all event episodes: a participant may experience more than one event, but the primary outcome is expressed as participants. The registry record does not report event-level totals or explain how repeated events were handled.

Safety outcomes of this kind are used to characterize events observed during exposure and follow-up. Without a control group and a reported comparative analysis, these counts do not establish how much of the observed event burden was caused by treatment. They also do not alone establish the frequency of events in a broader population. The outcome’s timeframe is up to 51 months, but the registry record does not report the distribution of individual follow-up time or the approach used for participants with incomplete observation.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant count represent?

The primary outcome is expressed as participants with an event. This is a person-level summary: it reports whether participants were counted as having an event under the registry’s definitions. It does not necessarily represent the number of event episodes, because a participant can have multiple episodes. The registry record does not report the total number of episodes or explain how recurrent events were treated in the analysis.

Person-level counts are often easier to interpret than episode counts because they focus on how many people were affected at least once during the observation period. However, they discard information about recurrence, duration, and severity unless those features are reported separately. Here, the record gives a serious-event count and a count of other adverse events, but does not provide a detailed event-level analysis.

Why does the event definition matter?

The adverse-event definition is broad: it includes unfavorable and unintended signs, symptoms, or diseases associated with study-drug use, whether or not considered related to the drug. The adverse-drug-reaction definition is narrower in its causal wording: it concerns a harmful and unintended reaction for which a causal relationship cannot be excluded. These definitions affect which observations enter each category. A reader should not assume that an adverse event was caused by treatment merely because it occurred during the study.

General statistical explanation: outcome definitions should be specified before results are interpreted because different rules for attribution, seriousness, or recurrence can produce different summaries from the same clinical experience. The registry record supplies definitions for adverse events and adverse drug reactions, but does not describe the detailed event-review process or how judgments were made.

What can a single treatment group tell us?

The posted information describes a Ganfort® group and does not describe a comparator. Its counts can characterize events recorded among treated participants, but they cannot directly estimate a contrast with another treatment or with no treatment. In general, a comparator helps account for events that might occur regardless of the intervention, including events related to the underlying condition or ordinary background risk. Without that comparison, the source of observed events cannot be separated by the posted counts alone.

The registry says allocation was not stated and flags that this is not a randomized comparison. It does not report a basis for treating any other group as a control. Therefore, no causal interpretation should be attached to a between-group effect that is not reported.

How should uncertainty be understood?

General statistical explanation: a confidence interval describes a range of values compatible with an estimate under the assumptions of the analysis. It communicates precision as well as the direction and scale of uncertainty. The registry record does not report confidence intervals for these safety counts, so their statistical precision cannot be assessed from the posted outcome alone.

General statistical explanation: event counts can be sparse, especially for serious outcomes. When counts are sparse, estimates may be imprecise, and a small number of observations can materially affect a summary. This is a general property of sparse data, not a further result calculated from this record. No inferential model or interval estimate is reported.

What do planning illustrations mean?

Any sample-size illustrations associated with these results are planning examples, not observed power. The stated assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the detectable effect is defined as a stated fraction of the observed effect. The sample-size assumptions also distinguish total events for time-to-event outcomes from participants per arm for other outcomes. These are assumptions for hypothetical planning, not a description of the study’s actual design or analysis.

General statistical explanation: a planning calculation asks how much information a future study might need under selected assumptions. It does not show that a completed study had a particular probability of detecting an effect, and it does not turn a descriptive outcome into a randomized comparison. The record does not report that its original enrollment was justified by these illustrations, and the illustrations should not be treated as the trial’s own design rationale.

The registry entries do not include a recalculated estimate or a discrepancy flag. No additional effect estimate or sample-size value can therefore be interpreted from the record. The key methodological conclusion is that the posted safety counts are descriptive, while the registry does not report the comparative or inferential analyses needed to estimate a treatment effect or its precision.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures, including the primary safety outcome and a secondary outcome of change from baseline in intraocular pressure. It does not report a multiplicity strategy, such as how interpretation would account for testing or selecting results across outcomes. General statistical explanation: when several outcomes are examined, repeated testing can increase the chance of a misleading positive finding if the analysis does not address multiplicity. No such adjustment or testing procedure is reported in the registry record.

The record lists zero analyses. It does not provide a statistical analysis plan, identify prespecified analysis populations beyond the safety population, or report how missing outcome data were handled. The safety population is described as all participants treated for on-label indications. The registry does not explain the difference between enrollment of 756 and 742 participants at risk for the safety outcome.

General statistical explanation: missing observations can affect an estimate if the likelihood of being observed is related to participant characteristics or outcomes. The consequences depend on why data are missing and on the methods used to address them. Since the registry record does not report missing-data methods or the extent and reasons for missingness, the direction or magnitude of any resulting bias cannot be assessed.

The reported timeframe is up to 51 months, but the registry does not describe participant-level follow-up or censoring. These details matter when comparing event risks over time. No time-to-event analysis is reported, and no assumptions about such an analysis should be inferred from the stated timeframe.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: neoplasm metastatic (0.1%).

The serious adverse-event outcome lists 1 participant with a serious event among 742 at risk in the Ganfort® group. The top serious term is reported as “Neoplasm metastatic” with 0.1%. These are registry-reported safety summaries, not a comparison with another group. The record does not report deaths; the death count is not reported in the registry record.

The serious-event figure should be read separately from the 86 participants listed with other adverse events. The record does not explain whether event categories overlap, and the counts should not be added together. Nor does the serious-term percentage establish the cause of the event or show whether the term represents all serious events. The registry provides no term-specific participant count in the registry record for this analysis.

General statistical explanation: a serious event is a classification, not a judgment that the treatment caused the event. Attribution and seriousness are distinct concepts. The registry’s broad adverse-event definition includes events whether or not they were considered related to the study drug, while an adverse drug reaction requires that a causal relationship could not be excluded. The posted serious-event summary alone does not provide the information needed to determine causality.

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

The results describe adverse events recorded in a Korean post-marketing surveillance study of Ganfort® for open-angle glaucoma or ocular hypertension. The primary safety outcome reports participants with events in the described safety population, with follow-up stated as up to 51 months. Those counts are relevant to understanding what the registry record captured during routine clinical practice.

They do not establish that Ganfort® caused the reported events. The registry describes no randomized comparator, and allocation is not stated. Without an appropriate comparison, observed events cannot be distinguished from events that could arise from the conditions being treated, other factors, or background occurrence. The record also does not report confidence intervals or another measure of statistical precision for the counts.

The findings do not establish that events not listed did not occur. They reflect the posted outcome summary and its definitions; the registry does not report the full event-ascertainment process, recurrence information, missing-data handling, or detailed follow-up. The difference between enrollment of 756 and the 742 participants at risk is not explained in the registry record.

The secondary IOP outcome is named, but its result is not reported in the registry record. Therefore, the listed safety figures do not answer whether intraocular pressure changed, nor do they quantify a comparative treatment effect. The completed status indicates the study is recorded as completed, but it does not substitute for unreported results or analyses.

In practical statistical terms, these are descriptive surveillance findings. They can be read as a record of reported events among treated participants, with substantial limits on causal and comparative interpretation. They should not be taken as a universal estimate of event frequency or as evidence for a treatment decision.

13. Why This Trial Matters Statistically

Post-marketing surveillance addresses a different statistical question from a randomized comparison. It records experience in clinical practice, where treatment is given according to local standard of care. Such records can contribute descriptive information about events observed after a drug is in use, while their design may not support causal comparisons without an appropriate control and a clearly specified analysis.

This study makes that distinction important. The registry identifies a safety population and reports adverse-event counts, but describes no like-for-like comparator and no randomized allocation. The counts therefore retain value as surveillance observations while leaving the treatment-attributable component uncertain. Recognizing that boundary prevents descriptive safety data from being mistaken for a comparative effect estimate.

The endpoint definitions also matter statistically. Adverse events include observations whether or not considered related to treatment, whereas adverse drug reactions use a causal-relationship criterion. Keeping those concepts separate helps avoid treating every event during exposure as a drug reaction. The record’s broad outcome and serious-event summary provide a framework for reading the reported counts, but do not replace detailed event-level information.

Finally, the record illustrates the importance of completeness in registry reporting. Enrollment, the safety population, outcome timeframe, and listed secondary outcome are present, while the reason for the difference between enrollment and the safety denominator, the IOP result, and analysis details are not reported. For biostatisticians and clinical researchers, these omissions shape what can be concluded just as much as the event counts do. A disciplined interpretation distinguishes what is observed from what remains unestimated.

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 most important limitation is the absence of a described comparator. The registry itself indicates that this is not a randomized comparison and does not state allocation. The event counts therefore cannot provide a like-for-like estimate of how outcomes differ under another treatment or no treatment.

The relationship between enrollment of 756 and the 742 participants at risk for the safety outcome is not explained in the registry record. The outcome population is described as participants treated for on-label indications, but the record does not specify why the numbers differ or whether all outcome information was available for everyone in the safety population.

Follow-up is described as up to 51 months, but the registry does not report how long each participant was observed, how follow-up ended, or how incomplete observation was handled. It also does not describe event ascertainment, recurrent-event handling, or the process used to classify seriousness and treatment relationship. These omissions limit interpretation of the counts and their comparability with other settings.

The registry lists change from baseline in IOP as a secondary outcome, but its result is not reported in the registry record. It lists zero analyses and provides no reported statistical analysis plan, confidence intervals, or inferential results. No multiplicity or missing-data approach is reported. The record also does not report a sample-size justification. These specific reporting gaps prevent assessment of precision, outcome selection, and the robustness of any comparative inference.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Safety and Efficacy of Bimatoprost/Timolol (Ganfort®) Ophthalmic Solution in Korea — NCT01976624.
• Registry record last updated 2015-09-28; status last verified 2015-08.
• Results first posted 2015-07-21.
• 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 post-marketing surveillance study of bimatoprost/timolol (Ganfort®) in Korea for open-angle glaucoma or ocular hypertension. It lists Allergan as sponsor, enrollment of 756, a start of 2009-08, and primary completion of 2014-01. The primary safety outcome counted participants with adverse events and adverse drug reactions over a timeframe of up to 51 months.

The Ganfort® safety outcome lists 742 participants at risk, 1 participant with a serious adverse event, and 86 with other adverse events. “Neoplasm metastatic” is listed among the top serious terms with 0.1%. The record does not report deaths. It lists IOP change as a secondary outcome but does not report that result, and it lists zero analyses.

No randomized comparison is described, and allocation is not stated. The record does not explain the difference between enrollment and the safety population or report analysis, missing-data, or multiplicity methods. The posted results are descriptive safety observations, not a comparative treatment-effect estimate.