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 A Study of the Safety and Efficacy of Bimatoprost Ophthalmic Solution in Paediatric Patients With Glaucoma (NCT01426113), a randomized phase 3 study. The primary outcome is Change From Baseline in Intraocular Pressure (IOP) in the Study Eye.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01426113 |
| Phase | Phase 3 |
| Condition | Glaucoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Triple |
| Enrollment | 6 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Allergan |
| Results posted | Yes |
2. Clinical Question
Population
Paediatric patients with glaucoma were eligible for the study. The registry record reports a target of 120 patients and enrollment of 6, with 3 in each group. Further eligibility details are not reported in the registry record.
Intervention
The intervention was bimatoprost ophthalmic solution formulation A, used with bimatoprost vehicle. The arm description reports a morning vehicle dose and evening formulation A dose for 6 weeks, followed by a formulation A regimen. The remaining description of that later regimen is incomplete in the registry record.
Comparator
The comparator was timolol ophthalmic solution. The registry describes 1 drop in the affected eye or eyes in the morning and evening for 12 weeks.
Primary question
The intended primary question concerned change from baseline in intraocular pressure in the study eye, with the recorded timeframe of baseline to Week 6. The registry explains that a negative change represents a reduction in pressure and a positive change represents an increase. Because of limited enrollment, the analysis was not performed and no outcome summaries were generated. The record therefore does not establish a comparative estimate of the endpoint.
This is a description of the study question, not evidence that either treatment had a particular effect. The registry identifies glaucoma as the condition and lists the two treatment strategies, but does not report baseline characteristics or participant-level outcome values. The trial’s early termination and lack of outcome analysis leave the primary comparison unanswered.
3. Trial Design
The registry describes a Phase 3, randomized, triple-masked, parallel-group study sponsored by Allergan. It began in 2011-09, had a recorded primary completion date of 2014-10, and is marked terminated. These design labels describe the planned structure; they do not mean the intended comparison was completed or analyzed.
The study compared bimatoprost ophthalmic solution formulation A with timolol ophthalmic solution in paediatric patients with glaucoma. The bimatoprost arm also lists bimatoprost vehicle. Its treatment description includes a vehicle dose in the morning and formulation A in the evening for 6 weeks, followed by a formulation A regimen, but the latter part of the description is cut off. The timolol arm is described as 1 drop in the affected eye or eyes in the morning and evening for 12 weeks. Further details of treatment conduct are not reported in the registry record.
The registry gives a target enrollment of 120 and reports that 6 patients enrolled, with 3 in each group. It states that early termination and lack of enrollment led to no statistical analysis and no data summaries for the primary outcome. The reasons for termination beyond the stated enrollment limitation, details of random-sequence generation, allocation concealment, and masking procedures are not reported in the registry record.
Randomization is intended to reduce systematic differences between groups, while masking is intended to limit the influence of treatment knowledge on care, assessment, or reporting. These are general design principles, not evidence that the procedures were implemented successfully in this study. With no reported outcome analysis, the registry cannot show whether the planned design yielded a comparative estimate.
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 | Bimatoprost Ophthalmic Solution Formulation A and Vehicle | Timolol Ophthalmic Solution |
|---|---|---|
| STARTED | 3 | 3 |
| COMPLETED | 2 | 1 |
| NOT COMPLETED | 1 | 2 |
Reasons for not completing this period:
| Reason | Bimatoprost Ophthalmic Solution Formulation A and Vehicle | Timolol Ophthalmic Solution |
|---|---|---|
| Study Discontinued | 1 | 1 |
| Adverse Event | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Due to early termination of the study, no statistical analysis was performed and no data summaries were generated. From a target of 120 patients, only 6 patients (3 in each group) were enrolled.
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 | Bimatoprost Ophthalmic Solution Formulation A and Vehicle | Timolol Ophthalmic Solution | Total |
|---|---|---|---|---|
| Participants analysed | 3 | 3 | 6 | |
| Age, Customized: 12 to 15 years | Participants | 3 | 3 | 6 |
| Sex: Female, Male: Female | Participants | 3 | 0 | 3 |
| Sex: Female, Male: Male | Participants | 0 | 3 | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Intraocular Pressure (IOP) in the Study Eye, measured over Baseline, Week 6.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was change from baseline in intraocular pressure (IOP) in the study eye, with the registry listing baseline and Week 6 as the timeframe. IOP is the fluid pressure inside the eye. The registry’s description interprets a negative change from baseline as a reduction in IOP and a positive change as an increase.
In general, an endpoint is a defined outcome used to assess the question a study is designed to address. Measuring change from baseline focuses on how the recorded value differs from its starting point, rather than relying only on a value at follow-up. IOP was the selected measure for the study’s stated efficacy question. The endpoint definition alone does not establish what happened to participants or whether the treatments differed.
The registry states that lack of enrollment meant analysis was not performed for this outcome measure. It also says no statistical analysis was performed and no data summaries were generated. Consequently, there is no reported group estimate of change, estimate of the difference between groups, measure of variability, or confidence interval for this endpoint. The registry record does not report the method used to measure IOP, rules for selecting the study eye, or how a participant’s endpoint value would have been summarized.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the primary endpoint was not analyzed. The record reports an enrollment target of 120 and actual enrollment of 6, with 3 in each group, and attributes the absence of analysis to early termination and lack of enrollment. There is no reported estimate of change in IOP for either group and no comparative estimate between bimatoprost and timolol. Therefore, the posted results do not quantify an effect or its precision.
This distinction matters. A randomized trial can, in principle, estimate a difference between assigned groups, but randomization by itself does not create an outcome estimate when the outcome analysis has not been performed. The study design identifies the comparison that was intended; it does not supply evidence about the direction or size of the comparison. The registry’s description of negative and positive changes explains how the endpoint would be interpreted, but no such changes are reported.
There is also no confidence interval or statistical test result for the primary endpoint. In general, a confidence interval describes the range of values compatible with the data and the method used, and helps communicate precision. Without an analyzed endpoint and an estimate, there is no interval to interpret. The registry record does not report a p-value for the primary comparison. This is not evidence that the groups were equivalent, nor is it evidence that one treatment was superior; the intended comparison remains unresolved in the posted record.
The adverse-event figures are separate descriptive safety counts and do not replace the missing efficacy analysis. The record reports no serious adverse events in either group, while other adverse events are recorded for participants in both groups. These counts have very small group denominators and are not accompanied by inferential comparisons or fuller event descriptions. They should be read as limited registry reporting, not as a comprehensive estimate of comparative safety.
In general, limited enrollment can make estimates unstable and reduce the ability to distinguish a real difference from random variation. In this case, however, the issue is more fundamental: the registry says the primary outcome was not analyzed and provides no outcome summaries. There is thus no observed efficacy effect in the record to characterize as large, small, beneficial, harmful, or uncertain. The proper interpretation is that the planned primary question cannot be answered from the posted outcome data.
The termination status and the stated enrollment limitation are relevant context, but the registry does not give a detailed account of the termination decision or any interim assessment. No conclusions about the conduct or reasons beyond what is recorded should be inferred. The available information describes the planned trial and a small set of adverse-event counts; it does not provide an analyzed comparison of IOP change.
9. Statistical Methods Explained
The study is registered as randomized, triple-masked, and parallel-group. These terms describe design features rather than analysis results. Randomization assigns participants to treatment groups by a chance-based process, which is intended to reduce systematic differences at the time of assignment. A parallel design means groups follow their assigned treatment strategies rather than crossing over between them. Masking is intended to limit the effects of knowing treatment assignment on participants, clinical staff, or outcome assessment. The registry does not describe the specific procedures used to implement these features.
What comparison was planned?
The stated primary outcome was change from baseline in IOP in the study eye, with baseline and Week 6 listed as the timeframe. A treatment comparison would ordinarily use outcome information from both assigned groups to estimate how their changes differ, under a specified analysis approach. The record does not report such estimates or identify the statistical method planned for the primary analysis. It says that lack of enrollment meant the analysis was not performed and no summaries were generated.
In general, an estimate and its uncertainty should be considered together. An estimate gives the observed difference or change, while a standard error or confidence interval conveys how much uncertainty is associated with it under the analysis assumptions. A p-value, when used, addresses compatibility between observed data and a specified null model; it does not measure the size or clinical importance of an effect. None of these primary-outcome quantities is reported for this trial, so none can be interpreted in relation to the bimatoprost and timolol comparison.
Why do enrollment and analysis matter?
The record reports a target of 120 patients, but enrollment was 6, with 3 in each group. A target enrollment is a planning figure, not an outcome result. The registry does not report the trial’s original sample-size justification or the assumptions used in its design. It also does not report a completed primary analysis. General statistical reasoning suggests that a small amount of outcome information can yield imprecise estimates, but the record gives no estimate from which to assess precision in this particular comparison.
Planning sample sizes are distinct from observed power. A sample-size calculation is a forward-looking planning exercise based on assumptions about the effect to detect, variability, significance level, allocation, and analysis. Observed power is not a substitute for presenting an effect estimate and its uncertainty. The registry record does not report the original trial’s sample-size justification or its planned statistical analysis. Any planning illustration based on an observed effect would require an analyzed effect estimate; no primary outcome estimate is reported for this trial.
What does masking add?
As a general principle, masking can reduce the risk that expectations influence subjective decisions or assessments. The registry’s “triple” label does not specify which roles were masked or how masking was maintained. IOP is a measured clinical quantity, but the record does not describe the measurement protocol, assessor procedures, or whether any masking was broken. These implementation details are not reported in the registry record.
What can be inferred from adverse-event counts?
Adverse-event counts are descriptive summaries. The registry lists serious events and other adverse events separately for the two groups. Counts alone do not account for event types, timing, exposure duration, or whether an event was judged related to treatment. General comparisons of safety also need careful interpretation when groups are small and follow-up or reporting may differ. The registry provides no inferential safety analysis, detailed event listings, or deaths count; deaths are not reported in the registry record.
Overall, the methods information supports an account of the design and intended endpoint, but not a reconstruction of the planned analysis. The statistical analysis plan, handling of missing outcome data, analysis populations, adjustment factors, and any interim analyses are not reported in the registry record. No specific model or test should therefore be attributed to the study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one outcome measure and identifies change from baseline in IOP in the study eye as the primary outcome. It does not report a completed analysis for that endpoint. The registry record does not report a multiplicity strategy, such as how statistical error would be handled if several outcomes, time points, or comparisons were tested. Because no primary analysis was performed, there are no reported tests to interpret in light of multiplicity.
The registry’s timeframe for the primary outcome is baseline and Week 6. It does not provide outcome data at those time points or describe how missing measurements would have been handled. The reason given for the absence of summaries is lack of enrollment and early termination; specific missing-data methods, imputation procedures, or sensitivity analyses are not reported in the registry record.
The record also does not identify analysis populations, such as whether an intended primary analysis would include all randomized participants or a defined subset. The count of enrolled participants is reported, but the registry does not provide a participant flow or detail treatment completion and withdrawal. These omissions prevent assessment of how the intended analysis population might relate to enrollment or treatment exposure.
These are reporting gaps, not evidence that a particular method was or was not used outside the registry record. The posted record states that no statistical analysis was performed for the primary outcome, so it offers no adjusted or unadjusted efficacy result to assess.
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 |
|---|---|---|---|
| Bimatoprost Ophthalmic Solution Formulation A and Vehicle | 0 | 3 | 0.0% |
| Timolol Ophthalmic Solution | 0 | 3 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Bimatoprost Ophthalmic Solution Formulation A and Vehicle | 3 of 3 |
| Timolol Ophthalmic Solution | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Bimatoprost Ophthalmic Solution Formulation A and Vehicle | Timolol Ophthalmic Solution |
|---|---|---|---|
| Growth of Eyelashes | Eye disorders | 1 of 3 | 1 of 3 |
| Conjunctival Hyperaemia | Eye disorders | 2 of 3 | 0 of 3 |
| Eyelash Hyperpigmentation | Eye disorders | 0 of 3 | 1 of 3 |
| Intraocular Pressure Increased | Investigations | 0 of 3 | 1 of 3 |
| Eyelid Oedema | Eye disorders | 1 of 3 | 0 of 3 |
| Headache | Nervous system disorders | 1 of 3 | 0 of 3 |
| Influenza | Infections and infestations | 1 of 3 | 0 of 3 |
| Diarrhoea | Gastrointestinal disorders | 1 of 3 | 0 of 3 |
| Pyrexia | General disorders | 1 of 3 | 0 of 3 |
| Blepharal Pigmentation | Eye disorders | 1 of 3 | 0 of 3 |
The adverse-event record reports 0 serious adverse events among 3 participants at risk in the bimatoprost formulation A and vehicle group, and 0 among 3 at risk in the timolol group. For other adverse events, it reports 3 affected among 3 at risk in the bimatoprost group and 1 affected among 3 at risk in the timolol group. These are counts as recorded, not an adjusted comparison.
In general, adverse-event counts describe what was recorded in the groups; they do not by themselves establish that treatment caused an event or that one treatment is safer. Interpretation depends on such details as event type, timing, severity, follow-up, and how events were collected. Those details are not reported in the registry record. The group sizes are limited, and the registry does not report a statistical comparison of these counts.
Deaths are not reported in the registry record. The absence of a reported death count should not be treated as a confirmed count of zero. These safety data are therefore narrow descriptive information and do not resolve the study’s comparative safety question.
12. What the Result Does, and Does Not, Mean
The record documents a randomized, triple-masked comparison that was intended to assess bimatoprost ophthalmic solution formulation A against timolol ophthalmic solution in paediatric patients with glaucoma. Its primary endpoint was change from baseline in IOP in the study eye, with the stated timeframe of baseline and Week 6. The study was terminated, and the registry attributes the lack of outcome analysis to insufficient enrollment.
What the posted results do show is limited: enrollment was 6, with 3 in each group, against a target of 120; no statistical analysis or primary-outcome summaries were generated; and adverse-event counts are reported. The posted results do not show the amount of IOP change in either group, a difference between groups, or the precision of any such difference. They do not establish whether either treatment reduced or increased IOP in this study.
In general, a study that has no analyzed primary endpoint cannot support an efficacy conclusion from that endpoint. The absence of an estimate is not the same as an estimate of no effect, and it does not demonstrate equivalence. Likewise, the serious adverse-event counts do not establish comparative safety, particularly without event details and a reported safety analysis.
The registry record does not report the detailed reasons for termination beyond the stated enrollment limitation, the original sample-size rationale, analysis populations, missing-data procedures, or a statistical analysis plan. Those unknowns limit reconstruction of what analyses were planned. The conclusions should remain confined to what the registry records: a planned comparison, limited enrollment, no primary outcome analysis, and descriptive adverse-event counts.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a planned randomized comparison and an informative estimate from that comparison. Randomization and masking describe how a study is intended to reduce bias. They cannot replace the outcome information needed to estimate a treatment contrast. Here, the registry records the design and primary question but reports that the primary analysis was not performed.
Enrollment is central to the context: the target was 120, while 6 patients enrolled, with 3 in each group. The registry explicitly links lack of enrollment to the absence of outcome summaries. This underscores a general point: a target sample size and a realized analysis are different things. A planned sample size does not guarantee that the planned comparison can be estimated if the trial does not generate analyzed outcome data.
The record also illustrates why absence of statistical significance cannot be inferred when no test was reported. There is no primary estimate, confidence interval, or p-value in the registry record. It would be incorrect to read the lack of an analysis as evidence of no difference or as proof that treatments perform similarly. Statistical conclusions depend on results and their uncertainty, not merely on a trial’s design labels.
Finally, safety counts require their own context. The registry records serious and other adverse events, but not a detailed event-level account or inferential comparison. Those figures can describe what was reported, yet they do not answer the missing efficacy question and should not be treated as a complete safety assessment.
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 is that the registry reports no primary-outcome analysis and no data summaries. There is consequently no IOP change estimate, between-group contrast, or measure of uncertainty to evaluate. The record states that early termination and lack of enrollment prevented analysis; the target was 120 and enrollment was 6, with 3 in each group.
Other important details are not reported in the registry record. These include baseline participant characteristics, participant flow after enrollment, treatment adherence, actual follow-up, reasons for termination beyond the stated enrollment limitation, and detailed eligibility criteria. The bimatoprost arm description is incomplete after the description of its later regimen, so that regimen cannot be fully reconstructed from the record.
The registry does not report the planned statistical analysis, analysis populations, handling of missing data, multiplicity procedures, or original sample-size justification. It also does not provide IOP measurement procedures, participant-level outcome values, or a confidence interval or test result. These gaps mean the intended analysis cannot be independently assessed from the registry record.
Safety reporting is limited to group counts of serious and other adverse events. Event types and timing, treatment attribution, and a deaths count are not reported in the registry record. The counts therefore offer only a narrow descriptive view and do not support a detailed comparative safety interpretation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of the Safety and Efficacy of Bimatoprost Ophthalmic Solution in Paediatric Patients With Glaucoma — NCT01426113.
- Registry record last updated 2015-10-02; status last verified 2015-09.
- Results first posted 2015-10-02.
- 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
The registry describes a randomized, triple-masked, parallel-group Phase 3 study sponsored by Allergan in paediatric patients with glaucoma. It compared bimatoprost ophthalmic solution formulation A, with bimatoprost vehicle, against timolol ophthalmic solution. The primary endpoint was change from baseline in IOP in the study eye, with baseline and Week 6 listed as the timeframe.
The study is marked terminated. Against a target enrollment of 120, 6 patients enrolled, with 3 in each group. The registry states that no statistical analysis was performed and no primary-outcome summaries were generated because of early termination and lack of enrollment. It reports 0 serious adverse events in each group and other adverse events affecting 3 participants in the bimatoprost group and 1 in the timolol group. Deaths are not reported in the registry record. The posted record does not provide a comparative efficacy estimate.