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 Efficacy and Safety of Topically Applied Medical Therapy for the Treatment of Pediatric Glaucoma (NCT01527682), a na phase 2 study. The primary outcome is Percentage of Eyes With Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01527682 |
| Phase | Phase 2 |
| Condition | Childhood Glaucoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Azienda Socio Sanitaria Territoriale degli Spedali Civili di Brescia |
| Results posted | Yes |
2. Clinical Question
Population
Children with primary pediatric glaucoma described as refractory to surgical procedures. The record lists childhood glaucoma as the condition and reports enrollment of 37 participants. It does not give further eligibility details or describe the characteristics of participants included in the primary endpoint analysis.
Intervention
Topical latanoprost, dorzolamide, or both. The treatment received by an eye was to depend on intraocular pressure (IOP) assessment. The record does not describe a fixed treatment sequence or provide more detail about how the choice among these options was made.
Comparator
There was no like-for-like control group: the study is listed as single-group, with allocation recorded as not applicable. The registry record therefore does not describe a randomized comparison against placebo, usual care, or another treatment.
Primary question
Over the 3-year study period, what percentage of eyes achieved and maintained an IOP decrease of at least 20% relative to baseline? The endpoint is defined at the eye level, while enrollment is reported in participants; the record does not state how many eyes contributed to the primary analysis or report the observed response percentage.
This is a question about change in IOP under a treatment approach in a selected pediatric glaucoma population, not a comparative question about which treatment option is better. Because the treatment could be latanoprost, dorzolamide, or both according to IOP assessment, the study represents a treatment strategy rather than one uniform drug exposure. The registry record does not report details of the treatment decisions or outcomes by treatment received.
3. Trial Design
The registry record describes a completed Phase 2, single-group study. Allocation is listed as not applicable and masking as none. These design features mean that the record does not describe random assignment to treatment groups or masking of participants, clinicians, or outcome assessors. It also explicitly indicates that the study is not a randomized comparison and that its arms should not be treated as like-for-like controls.
The intervention is listed as latanoprost and dorzolamide. According to the description, an eye could receive latanoprost, dorzolamide, or both depending on IOP assessment. This makes interpretation different from a trial in which every participant receives the same fixed regimen. The record does not report a protocol for selecting treatment, changing treatment, or managing treatment failure.
Enrollment is listed as 37. The study summary discusses planned eye counts: an earlier protocol forecast 96 eyes to complete enrollment, and an amended protocol set a target of 68 eyes. It also states that reaching the amended eye target could require recruiting from 34 to 68 patients, because both eyes would not necessarily be eligible for every participant. These are protocol-planning figures, not a report of the number of eyes actually analyzed. The registry record does not report the final number of eligible eyes or the number included in the primary endpoint analysis.
The study start is recorded as 2009-07, primary completion as 2016-11, and status as completed. The record identifies a selected population with primary pediatric glaucoma described as refractory to surgical procedures. It does not provide further details about the surgical history, eligibility rules, recruitment process, follow-up completeness, or the statistical analysis plan. Those omissions limit assessment of how closely the analyzed population and conduct corresponded to the stated study objective.
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: Patients affected with Primary congenital glaucoma will be recruited if after a single surgical procedure Intraocular pressure will be ranging from 22 and 27 mm Hg
Overall Study
| Stage | Latanoprost, Dorzolamide |
|---|---|
| STARTED | 37 |
| COMPLETED | 29 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Latanoprost, Dorzolamide |
|---|---|
| Protocol Violation | 8 |
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 | Latanoprost, Dorzolamide |
|---|---|---|
| Participants analysed | 37 | |
| Age, Categorical: <=18 years | Participants | 37 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 4.1 (SD 3.8) |
| Sex: Female, Male: Female | Participants | 15 |
| Sex: Female, Male: Male | Participants | 22 |
| Region of Enrollment: Italy | Participants | 37 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Eyes With Response, measured over 3 years.
| Element | Registry entry |
|---|---|
| Result type | Count Of Units |
| Measure of spread | not applicable |
| Unit | eyes |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the percentage of eyes with a response. A responding eye is defined as one in which IOP decreases by at least 20% relative to its baseline assessment and that decrease is achieved and maintained during the 3-year study period. The endpoint therefore combines the magnitude of change from baseline with persistence over time; a temporary decrease alone would not meet the stated definition.
Using a percentage gives a way to describe how frequently eyes met a prespecified response criterion, rather than reporting only an average pressure change. The threshold also makes the endpoint clinically interpretable as a binary classification for each eye: response or no response under the definition. As a general statistical point, a binary endpoint can be summarized by the number of responding units and the total number evaluated, as well as by an estimated response proportion and its uncertainty.
The registry record does not report the number of eyes meeting the definition, the total number assessed for this outcome, or an estimated percentage. It also does not describe how repeated IOP measurements were used to determine that a decrease was maintained, what happened when measurements were missing, or how the response criterion was applied to eyes with changes in treatment. The title and definition identify the intended outcome, but the posted record does not provide enough outcome detail to evaluate its observed frequency or precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record identifies an important descriptive question: whether eyes in a selected group of children with primary pediatric glaucoma met a sustained IOP response definition while receiving treatment selected from latanoprost, dorzolamide, or both. However, the registry record does not report the observed primary endpoint result. It gives no count of responding eyes, no number evaluated for the endpoint, and no response percentage or interval estimate. The primary outcome therefore cannot be interpreted quantitatively from the posted record.
The absence of a comparator is central to interpretation. A single-group study can describe outcomes observed during a treatment approach, but it does not provide a randomized contrast with an untreated group or another regimen. Changes from baseline may be described within the group if those results are reported, but without a concurrent control they cannot by themselves isolate the contribution of treatment from other explanations. The registry record does not report a primary outcome estimate in any case.
The outcome is defined per eye, whereas enrollment is stated in participants. The summary explains that not every participant necessarily had both eyes eligible. Since the number of eyes actually analyzed is not reported, the effective denominator and the relationship between participant and eye counts remain unclear. When both eyes from one participant are included, their outcomes may be related. As a general statistical consideration, analyses that treat such eyes as independent can overstate precision; the record does not report how dependence between eyes was addressed.
The intervention description also matters: treatment could vary among eyes according to IOP assessment. This means the study is not a straightforward estimate for one fixed drug regimen. The registry record does not report outcomes separated by treatment received, or explain whether treatment changes were part of the response assessment. It is therefore not possible to attribute any unreported outcome to one component of the treatment approach.
The safety record lists 37 at risk for serious adverse events and 0 affected, and 37 at risk in the other adverse-event category with 22 affected; deaths are listed as 0. These counts describe what the record reports, but do not replace the missing efficacy result. The record does not state the observation time contributed by each eye or participant, provide event descriptions for the other category, or explain how events were collected. Overall, the registry supports a description of the design and recorded safety counts, but not a numerical conclusion about the primary response endpoint or a comparative treatment effect.
9. Statistical Methods Explained
Interpreting this study requires separating the endpoint definition, the design, and the availability of results. The registry record describes a single-group study with an eye-level response endpoint. It does not report the primary result or enough analysis details to reconstruct how the result was estimated.
What does a response proportion represent?
The primary outcome is the percentage of eyes meeting a defined response condition: an IOP decrease of at least 20% from baseline, achieved and maintained during the 3-year study period. In general, a proportion is formed by counting units that meet a condition and relating that count to the number of units evaluated. Here, the relevant unit is the eye. Both pieces—the responding-eye count and the evaluated-eye denominator—are needed to interpret the percentage. Neither is reported in the registry record.
A response threshold simplifies a continuous measurement into two categories. This can make a result easier to communicate, but it also means that eyes just on opposite sides of the threshold are classified differently even if their measured values are close. The endpoint additionally requires that the decrease be maintained, so its application depends on how repeated assessments are interpreted. The record gives the threshold and follow-up duration but does not describe the measurement schedule, the rule for confirming persistence, or the handling of missing assessments.
Why does the single-group design matter?
In a randomized comparison, groups are assigned to different strategies so that a between-group contrast can estimate how outcomes differ under those strategies, subject to the trial's conduct and assumptions. This record instead lists a single-group model and no applicable allocation. It does not provide a randomized control comparison. A result from this design would describe outcomes in the enrolled group, not establish what would have happened to comparable participants under a different treatment strategy.
The intervention could be latanoprost, dorzolamide, or both according to IOP assessment. The treatment strategy is therefore conditional on clinical assessment as described in the record. If treatment selection is related to participants' or eyes' clinical status, outcomes across treatment options would not automatically be directly comparable. The registry record does not report treatment-specific response estimates or the details needed to assess such comparisons.
What can be said about precision?
As a general statistical concept, an estimate is more informative when accompanied by an uncertainty interval. A confidence interval describes a range of values compatible with the data and the method used, under that method's assumptions; it is not a guarantee that the true value lies in the interval. For a response proportion, precision depends in part on the number of independent units contributing information. The record gives participant enrollment but not the number of eyes evaluated for the primary endpoint, the response estimate, or an interval. Consequently, the precision of the primary result cannot be assessed.
Participants may contribute one or two eligible eyes. As a general matter, two eyes belonging to the same participant can have correlated outcomes because they share characteristics and care. Analyses may need to account for this within-participant dependence. The protocol summary acknowledges that not all participants would necessarily contribute both eyes, but the registry record does not report the analysis unit, a method for accounting for paired eyes, or the final eye count. No conclusion about the adequacy of the analysis for this structure can be drawn.
How should the planning illustrations be read?
The record describes sample-size illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These assumptions describe planning illustrations, not the design reported for this single-group study: there is no randomized two-arm allocation in the registry record. The record does not provide the numerical illustration results in the information available for this analysis. Such illustrations should not be treated as observed power, as evidence that the study was adequately powered, or as the trial's own sample-size justification.
More generally, power calculations address the chance that a specified design and analysis would detect a specified effect under assumed conditions. They do not describe the probability that a finding is true, and a retrospective calculation based on an observed effect is not a substitute for a prespecified design rationale. The registry record does not report the original sample-size justification or the assumptions behind the protocol's eye targets beyond the stated enrollment figures.
What analysis details are missing?
The record lists 3 outcome measures and 1 analysis, but does not describe the statistical method used for the primary endpoint, any adjustment for repeated measurements, or how participants with incomplete follow-up were handled. It also does not report an analysis population, such as whether analyses included all enrolled participants or only eligible and evaluable eyes. Because these details are absent, the posted record does not allow independent assessment of the primary endpoint analysis, its assumptions, or the precision of any estimate.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 3 outcome measures and 1 analysis. It identifies the percentage of eyes with response as the primary outcome and lists time to treatment failure and the number of eyes with an adverse event as secondary outcomes. The record does not describe whether there were additional planned comparisons, repeated analyses, or subgroup analyses, nor does it report a procedure for controlling the chance of false-positive findings across multiple outcomes. The presence or absence of such procedures cannot be inferred from the listed outcome count alone.
Missing-data handling is not reported in the registry record. In particular, it does not say how an eye was classified if one or more IOP assessments were absent, how failure to complete follow-up affected the sustained-response definition, or whether any imputation or sensitivity analysis was planned. These omissions matter because the primary outcome requires a response to be achieved and maintained over the 3-year period. Without the analysis rules and follow-up accounting, readers cannot determine how incomplete observations influenced the endpoint.
The record also does not identify the analysis population or provide the number of participants and eyes included in each outcome analysis. It reports enrollment of 37 participants and discusses an amended target of 68 eyes, but does not state the final number of eyes contributing data. The unit of analysis and any method for accounting for two eyes from one participant are not reported. No assumptions about intention-to-treat analysis, per-protocol analysis, or handling of ineligible eyes should be made from the available description.
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 |
|---|---|---|---|
| Latanoprost / Dorzolamide | 0 | 37 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Latanoprost / Dorzolamide | 0 of 37 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Latanoprost / Dorzolamide | 22 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Latanoprost / Dorzolamide |
|---|---|---|
| Hypertrichosis | Eye disorders | 17 of 37 |
| Corneal epitheliopathy | Eye disorders | 17 of 37 |
| Allergic conjunctivitis | Eye disorders | 9 of 37 |
| Iris color modification | Eye disorders | 6 of 37 |
| Eyelid skin hyperpigmentation | Eye disorders | 1 of 37 |
The registry record lists 37 at risk for serious adverse events and 0 affected. It separately lists 37 at risk in the other adverse-event category and 22 affected. Deaths are listed as 0. These are counts in the record, not rates adjusted for duration of observation. The record does not provide event terms for the other category in the information, so the nature, severity, timing, and recurrence of those events cannot be described.
Zero serious events recorded does not mean that the possibility of serious events has been ruled out. The interpretation of a zero count depends on how many people or eyes were monitored, the duration and completeness of follow-up, and how events were defined and collected. The registry record does not report those details, nor does it explain whether the at-risk unit is consistently the participant or the eye across the safety categories. The endpoint descriptions refer to eyes with adverse events, while enrollment is reported in participants, so the unit should not be assumed without further detail.
These safety counts should be read as registry-recorded observations in a single-group study. They do not provide a comparison of adverse-event frequency against another treatment or control group. They also do not establish that an event was caused by treatment. The record does not report an adjudication process or additional safety-analysis methods.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study was designed to assess an eye-level response to treatment in a selected pediatric glaucoma population. It defines response as an IOP decrease of at least 20% from baseline that was achieved and maintained during the 3-year study period. It also reports participant enrollment, protocol eye-count targets, the intervention options, and safety counts. These details describe the study question and some aspects of its conduct.
The record does not report the observed primary endpoint result. Without the number of eyes meeting the response definition, the number assessed, or an estimated percentage, it is not possible to state how often the defined response occurred or how precise an estimate would be. The treatment-failure outcome is listed, but its result is not reported in the available record. The safety figures are counts, and the record does not provide enough detail to interpret the other adverse-event category beyond its reported count.
Even if a response estimate were available, this single-group design would describe outcomes during a treatment approach rather than provide a randomized comparison. The treatment received could vary according to IOP assessment, and the registry record does not report outcomes separately by treatment option. Thus, the design cannot establish a comparative effect of latanoprost versus dorzolamide or their combination from the information recorded.
As a general statistical principle, an observed before-and-after change does not by itself distinguish a treatment effect from other influences that change over time or affect measurement. A concurrent comparison can help address such questions, while also requiring attention to design and analysis assumptions. The registry record does not report a control group or an estimate that could be contrasted with one.
The posted information therefore supports a limited conclusion: the study had a defined sustained-response endpoint and recorded safety outcomes, but the primary result and key analysis details are not reported in the registry record. This is not evidence that the endpoint was or was not met. Nor does it permit a conclusion about the treatment's effectiveness or comparative safety.
13. Why This Trial Matters Statistically
This study is statistically notable because its primary outcome is defined for eyes, while enrollment is reported for participants. The study summary explicitly distinguishes the planned number of eyes from the number of patients needed to reach that target, since not every participant would necessarily contribute both eyes. This is an important design feature: participant totals and eye totals are not interchangeable, and the analysis must make clear which units contributed outcome information.
When a participant contributes two eyes, the observations may be related. As a general statistical matter, accounting for such dependence is relevant to uncertainty estimates; treating correlated eye outcomes as independent may make an estimate appear more precise than warranted. The registry record does not state how this issue was handled or how many eyes were analyzed, so it illustrates why a clear analysis description is essential for eye-level outcomes.
The response endpoint also combines a numerical threshold with a requirement that the change be maintained over time. That structure makes the definition more informative than a single measurement, but requires transparent rules for repeated assessments and missing visits. The registry record gives the duration and threshold without reporting those operational details or the observed response result.
Finally, the treatment strategy allows different options according to IOP assessment, while the design is single-group. This distinction matters for interpretation: a result under a variable treatment approach is not the same as a randomized estimate for one fixed regimen. The absence of a reported primary outcome and analysis details prevents evaluation of the result's precision or of how treatment variation was incorporated.
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 immediate limitation of the registry record is that it does not report the primary endpoint result. No count of responding eyes, evaluable-eye denominator, response percentage, or uncertainty interval is available. The same record lists secondary outcomes but does not provide their results in the information analyzed.
Further detail about the analyzed population is absent. Enrollment is reported as 37 participants, while the study summary describes an amended target of 68 eyes and an earlier forecast of 96 eyes. The record does not report the final number of eligible eyes, the number completing follow-up, or the number contributing to each outcome. It also does not describe whether both eyes from any participant were included or how within-participant dependence was handled.
The treatment approach is not uniform: an eye could receive latanoprost, dorzolamide, or both based on IOP assessment. The record does not state how treatment choices were made, whether they changed over time, or how such changes affected endpoint classification. There is no randomized control group, and the record specifically cautions that the arms are not a like-for-like control comparison. These features limit comparative interpretation.
Methods important to the endpoint are not reported, including the measurement schedule, the operational rule for a response being maintained, missing-data handling, and the analysis population. The registry record also does not report a sample-size justification, the full statistical analysis plan, or the method used to account for eye-level data. Safety information is limited to counts without event descriptions for the other adverse-event category or details about observation time and ascertainment.
These limitations concern what can be concluded from the registry record; they do not establish that omitted procedures were not performed. They mean that those procedures and results are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety of Topically Applied Medical Therapy for the Treatment of Pediatric Glaucoma — NCT01527682.
- Registry record last updated 2019-09-27; status last verified 2019-09.
- Results first posted 2019-09-27.
- 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 record describes a completed Phase 2, single-group study of topical latanoprost, dorzolamide, or both for selected patients with primary pediatric glaucoma described as refractory to surgical procedures. Treatment for an eye was to depend on IOP assessment. Enrollment is listed as 37 participants; the summary describes an amended target of 68 eyes and an earlier forecast of 96 eyes.
The primary endpoint is the percentage of eyes achieving and maintaining an IOP decrease of at least 20% from baseline during the 3-year study period. The record does not report the primary endpoint result or the number of eyes analyzed. It lists 3 outcome measures and 1 analysis but does not describe key analysis methods, missing-data handling, or the analysis population.
Safety counts include 0 serious adverse events among 37 at risk, 22 affected in the other adverse-event category among 37 at risk, and 0 deaths. The study is not a randomized comparison, and the registry record does not support a comparative treatment-effect conclusion.