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 Microplasmin Intravitreal Administration in Participants With Uveitic Macular Edema (NCT01194674), a na phase 1, phase 2 study. The primary outcome is Number of Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01194674 |
| Phase | Phase 1, Phase 2 |
| Condition | Uveitis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institutes of Health Clinical Center (CC) |
| Results posted | Yes |
2. Clinical Question
Population
Participants with uveitic macular edema were the intended study population. Uveitis is the listed condition. The registry record lists enrollment as 2; it does not report further eligibility details or a demographic description.
Intervention
The intervention was Microplasmin, identified as a drug and described in the title as administered intravitreally. The registry record does not report the dose, schedule, or detailed administration procedures.
Comparator
No comparator group is listed. The study is classified as single-group, with allocation marked NA and masking listed as none. This means the recorded study design does not offer a concurrent control for comparison.
Primary question
The primary outcome was the number of adverse events over 24 weeks. The study summary describes an objective of investigating Microplasmin’s safety and efficacy, while the listed primary endpoint focuses on adverse events. The registry record does not report a primary endpoint result as a total event count.
Other questions listed for assessment concerned changes in central macular thickness measured by optical coherence tomography, posterior vitreous detachment, best-corrected visual acuity, and retino-vascular leakage seen on fluorescein angiography. The registry specifies measurements at 4 weeks for several outcomes and at 12 weeks for visual acuity. Those outcome descriptions indicate what investigators planned to assess, not whether a change occurred or how large it was.
The clinical question is therefore narrower than a comparison of treatment with an alternative: the record describes observations in a small group receiving Microplasmin, with adverse events as the primary endpoint and ocular measures as secondary outcomes. Without a comparator, outcome patterns cannot by themselves distinguish an intervention-related effect from changes that might also occur without the intervention.
3. Trial Design
The registry identifies NCT01194674 as a Phase 1 and Phase 2 study of Microplasmin intravitreal administration in participants with uveitic macular edema. Its design is classified as single-group. Allocation is listed as NA and masking as none. Enrollment is listed as 2. The study status is terminated.
The registry lists a start date of 2011-01 and a primary completion date of 2011-12. It names the National Institutes of Health Clinical Center (CC) as sponsor. No additional explanation for the termination is reported in the registry record, so the status alone does not establish why the study ended or whether the planned assessments were completed.
A single-group design can describe outcomes observed among participants treated in the study, but it does not provide a randomized or concurrent control group. The record’s skip note also cautions that the arms are not a like-for-like control comparison. Accordingly, the results cannot be interpreted as a controlled estimate of the difference between Microplasmin and another treatment or no treatment.
The primary outcome is titled “Number of Adverse Events” and has a timeframe of 24 weeks. The registry lists nine outcome measures and zero analyses. Secondary outcomes include changes in central macular thickness at 4 weeks, posterior vitreous detachment at 4 weeks, visual acuity at 4 weeks and 12 weeks, and retino-vascular leakage at 4 weeks. The registry record does not provide the statistical analysis plan, analysis populations, detailed eligibility criteria, intervention dose, or an account of which planned assessments were completed.
Phase labels describe the study’s registered phase, but they do not establish what procedures, dose-escalation rules, or monitoring plan were used. Those details are not reported in the registry record. The small listed enrollment and single-group structure are central to interpreting the amount of information these results can offer.
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 | Microplasmin |
|---|---|
| STARTED | 2 |
| COMPLETED | 2 |
| NOT COMPLETED | 0 |
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 | Microplasmin |
|---|---|---|
| Participants analysed | 2 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 40.50 (full range 17.68) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Adverse Events, measured over 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Adverse Events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of adverse events over 24 weeks. An adverse-event count is intended to capture events observed during a stated follow-up period. It is different from a count of participants who experienced at least one event: a participant can contribute more than one event, so participant-level affected counts do not necessarily reveal the total number of events.
Safety endpoints are commonly included to describe the kinds and frequency of undesirable events observed during a study. Such information can help characterize what was recorded during follow-up, but it does not by itself establish that an intervention caused an event. Attribution requires information about timing, clinical assessment, and alternative explanations; these details are not reported in the registry record.
The registry lists the primary outcome’s unit as adverse events and its timeframe as 24 weeks. The posted adverse-event figures report participants at risk and participants affected for serious and other adverse events. These participant figures do not supply the total number of events needed to read the primary endpoint literally as an event count. A primary endpoint result reported as an event total is not reported in the registry record.
In general, the interpretation of a count endpoint depends on how events are defined, collected, and handled when participants have different observation times. The registry record does not report event definitions, exposure-time methods, or details of event ascertainment. The endpoint is therefore identifiable by its title and timeframe, but the information available does not support a detailed reconstruction of its measurement or analysis.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry lists enrollment as 2 and describes a single-group Microplasmin study. The adverse-event figures show 2 at risk for serious adverse events, with 0 affected, and 2 at risk for other adverse events, with 1 affected. These are participant-level figures. They should not be read as counts of total events: the record does not state how many separate events occurred among affected participants.
The serious adverse-event figures indicate that no affected participants are recorded in that category. The other-adverse-event figures indicate that 1 participant is recorded as affected among 2 at risk. The record lists no serious adverse-event terms. Death figures are not reported in the registry record. These entries describe what the registry records; they do not, on their own, establish whether an event was caused by Microplasmin or how complete event reporting was.
The small enrollment limits precision. In general, results based on very few observations can change substantially with each additional observation, and estimates may be highly uncertain. The registry record does not report confidence intervals or another uncertainty measure for these adverse-event figures. It is therefore not possible to quantify their precision from the posted results. The observed figures should be read as descriptive counts rather than as precise estimates of the frequency of events in a broader population.
The study had no listed comparator group. Without a concurrent comparison, these observations cannot separate outcomes associated with study participation from background events, underlying disease, or other influences. Nor can the registry entries establish a comparative risk. The allocation field is NA, the design is single-group, and the record specifically cautions against treating the arms as a like-for-like control comparison.
The registry’s stated objective includes safety and efficacy, but the primary endpoint is adverse-event count. Secondary outcomes address retinal thickness, posterior vitreous detachment, visual acuity, and retino-vascular leakage. Their descriptions name the measures and assessment times, but outcome results for those measures are not reported in the registry record. The study’s efficacy objective therefore cannot be evaluated from the information recorded in the registry record.
The record lists nine outcome measures and zero analyses. No statistical analysis plan or explanation of the absent analyses is reported. This prevents assessment of planned hypothesis tests, adjustment for repeated outcome assessments, or model choice. The posted safety figures can be described directly, but they do not establish a treatment effect or rule out effects that the study was too limited to detect.
The status is terminated, with primary completion dated 2011-12. The reason for termination and its implications for follow-up are not reported in the registry record. Termination alone does not show whether the study stopped because of safety, feasibility, recruitment, or another reason. Conclusions should remain confined to the listed counts and the limitations imposed by the design and available reporting.
9. Statistical Methods Explained
What does a count outcome represent?
In general, a count outcome records how many events meet a defined criterion during a specified observation period. The primary endpoint in this study is titled “Number of Adverse Events,” with a 24-week timeframe. An event count and the number of participants affected are related but distinct quantities. A participant can experience multiple events, while a participant-level affected figure records whether a participant is represented as having an event in the category.
The posted figures give the number at risk and number affected for serious and other adverse events. They do not give a total number of separate events. As a result, they allow a descriptive reading of participants affected but do not reconstruct the primary endpoint as an event total. The registry does not report a definition of an adverse event, rules for counting recurring events, or event-level detail. Those measurement choices matter because different counting rules can produce different totals from the same participant experiences.
In general, count data are often analyzed with models designed for non-negative event counts. A Poisson model is one common starting point, but it assumes a relationship between the mean and variance that may be unsuitable when the data are over-dispersed. Over-dispersion means the event counts vary more than the model expects; if not accounted for, uncertainty can be understated. Other models or descriptive methods may be appropriate depending on event rates, follow-up, and the study question. The registry record does not report which method, if any, was used.
Why does follow-up matter for event counts?
In general, a count observed over a fixed interval can be interpreted differently from a rate that accounts for time under observation. Participants may have different lengths of follow-up, and participants who leave observation earlier have less opportunity for events to be recorded. A rate analysis can incorporate person-time, while a simple count does not automatically do so. The listed primary endpoint specifies 24 weeks, but the registry record does not report individual follow-up duration, person-time, censoring rules, or how incomplete follow-up was handled. The timeframe alone does not establish that every participant contributed the same amount of observed time.
For the secondary outcomes, the registry describes change from baseline in central macular thickness and visual acuity, as well as assessments of posterior vitreous detachment and retino-vascular leakage. In general, change scores compare a follow-up measurement with a baseline measurement for the same participant. Interpreting them requires knowing how measurements were collected, whether readings were repeated or centrally assessed, and how missing measurements were handled. These procedural and analytic details are not reported in the registry record.
What does a confidence interval convey?
In general, a confidence interval communicates uncertainty around an estimated quantity under a specified statistical method. A narrow interval indicates greater precision than a wide interval, while an interval that includes values compatible with different conclusions may leave substantial uncertainty. Confidence intervals do not correct for bias, poor measurement, or the absence of a control group. No confidence interval for the primary adverse-event endpoint is reported in the registry record, and the available participant counts do not provide enough information to reproduce one appropriately.
Small samples generally yield limited information about uncommon events and unstable estimates. With enrollment listed as 2, the counts should be treated as a description of this study record, not as a precise estimate of event frequency for a wider population. No inferential analysis for the safety outcome is reported. The registry’s zero analyses entry means the record does not list an analysis count; it does not tell readers which calculations investigators may have conducted outside the registry.
How should planning illustrations be understood?
In general, a sample-size calculation asks how much information a hypothetical study might need under stated assumptions. The planning assumptions associated with the record specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect expressed as a stated fraction of an observed effect. Such calculations are planning illustrations, not observed power and not evidence about the treatment’s performance.
Those assumptions describe a hypothetical two-arm planning setup, whereas this trial is registered as single-group. Any resulting illustration cannot be treated as the actual design of NCT01194674 or as an estimate of what its enrolled group could establish. The sample-size assumptions do not replace a registered design or analysis plan. No sample-size justification for the trial itself is reported in the registry record, and no actual planning values need be inferred from the assumptions.
More broadly, statistical significance, precision, and clinical importance are different concepts. A significance test evaluates data against a statistical model and a null hypothesis; a confidence interval expresses uncertainty around an estimate; and clinical importance depends on the meaning of the outcome. None can be fully assessed without outcome estimates, methods, and context. Here, no analysis is listed, and no primary event total or secondary outcome estimates are reported in the registry record. The appropriate statistical reading is therefore descriptive and cautious rather than inferential.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures and zero analyses. It names a primary adverse-event endpoint and several secondary assessments, including measures collected at 4 weeks and visual acuity measured at 4 weeks and 12 weeks. Considering multiple outcomes and assessment times can create multiplicity: the more comparisons examined, the more opportunities there may be for an apparently notable result by chance. In general, a prespecified analysis plan can clarify which outcomes are confirmatory and whether any adjustment or hierarchy is intended.
No multiplicity strategy, statistical analysis plan, or explanation of analysis populations is reported in the registry record. It is therefore not possible to determine whether investigators planned adjusted tests, selected a primary contrast, or treated secondary results as exploratory. The listed outcome descriptions alone do not establish how results would have been analyzed or interpreted.
The registry record also does not report missing-data counts, reasons for missingness, imputation methods, or rules for excluding observations. Missing data can affect interpretation when the availability of measurements is related to participants’ health or outcomes. For change-from-baseline measures, both baseline and follow-up data are needed to calculate a change; for event outcomes, incomplete observation can reduce opportunities to record events. These are general considerations, not claims about what occurred in this trial.
There is no reported analysis population specifying whether results were based on all enrolled participants, those who received treatment, or those with available outcome measurements. Enrollment is listed as 2, but the registry does not describe participant flow or completeness of each assessment. The absence of these details limits reproducibility and makes it difficult to know precisely which observations would underlie any outcome estimate.
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 |
|---|---|---|---|
| Microplasmin | 0 | 2 | 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 |
|---|---|
| Microplasmin | 1 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Microplasmin |
|---|---|---|
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 2 |
| Blood glucose increased | Investigations | 1 of 2 |
| Conjunctivitis infective | Infections and infestations | 1 of 2 |
| Rash | Skin and subcutaneous tissue disorders | 1 of 2 |
The registry records 2 participants at risk for serious adverse events and 0 affected. For other adverse events, it records 2 at risk and 1 affected. These figures describe participants affected in each category, not the total number of separate events. A participant affected by an event category may have experienced more than one event, but event-level counts are not reported in the registry record.
No serious adverse-event terms are listed. The record does not report death figures. The primary endpoint is titled “Number of Adverse Events” over 24 weeks, but the posted at-risk and affected figures do not supply a total event count. They should not be substituted for that endpoint without additional event-level information.
In general, a zero recorded count in a small study does not demonstrate that an event is impossible or that risk is absent. Sparse safety observations provide limited information about uncommon events, and the absence of a comparator also limits attribution. The figures describe what was registered for this study; they do not establish causation, comparative safety, or completeness of follow-up. The registry does not report event definitions, severity gradings beyond the serious/other categories, or details of relatedness assessments.
12. What the Result Does, and Does Not, Mean
The registry record documents a terminated, single-group study of intravitreal Microplasmin in participants with uveitic macular edema. Enrollment is listed as 2. Its primary safety outcome was the number of adverse events over 24 weeks, and the posted safety figures record 0 affected among 2 at risk for serious adverse events and 1 affected among 2 at risk for other adverse events. These figures describe recorded participants affected, not total events.
The record also identifies planned secondary measures involving macular thickness, posterior vitreous detachment, visual acuity, and retino-vascular leakage. It does not report results for those measures in the registry record. The stated objective included safety and efficacy, but the available outcome information does not allow an assessment of the listed efficacy measures. Nor does it provide a total adverse-event count for the primary endpoint.
These observations do not establish that Microplasmin caused or prevented an event. A single-group study has no listed concurrent comparator, so it cannot separate outcomes associated with treatment from the disease course, background events, or other influences. The record does not report a statistical analysis plan or analyses, and it provides no uncertainty interval for the adverse-event figures. With the small listed enrollment, the safety counts are descriptive and provide limited precision.
The status “terminated” does not reveal why the study ended. The reason for termination, the completeness of follow-up, and whether all planned assessments were performed are not reported in the registry record. The appropriate conclusion is therefore limited: the record contains a small set of participant-level adverse-event counts and describes intended ocular outcomes, but it does not support a controlled estimate of treatment effect or a broad estimate of safety.
In general, absence of a reported result should not be confused with evidence that an outcome did not occur. Likewise, a count of zero affected participants in a category is not proof that the event cannot occur. The available information supports a description of the registered observations, not a clinical recommendation or a definitive conclusion about efficacy or safety.
13. Why This Trial Matters Statistically
This trial illustrates why study design and outcome reporting matter as much as the labels attached to endpoints. The registry describes a single-group study with no comparator, enrollment of 2, and a primary endpoint framed as an adverse-event count. Those features sharply limit what the observations can establish. Counts from a small treated group can describe recorded experiences, but they cannot provide a controlled contrast or a precise estimate of event frequency.
The record also shows why participant-level safety summaries must be distinguished from event counts. The affected figures indicate whether participants appear in a category, while the primary endpoint is defined as the number of adverse events. These are not interchangeable quantities. Clear event-level reporting, including definitions and handling of repeated events, is important when the endpoint concerns counts.
Multiplicity and missing-data reporting are also relevant because the registry lists multiple outcomes and assessment times. Without a statistical analysis plan, analysis populations, and reporting of missingness, readers cannot determine how planned comparisons were prioritized or how incomplete measurements were handled. This is a general issue in clinical research, not evidence that a particular analytic choice was made in this study.
Finally, planning calculations should be kept separate from evidence generated by the trial. The recorded planning assumptions describe a hypothetical two-arm setup, whereas the trial itself is registered as single-group. This distinction helps prevent a design illustration from being mistaken for observed power or for the study’s actual analysis.
The statistical importance of this record lies less in a conclusive estimate than in the questions it exposes: what was counted, who contributed observations, how complete follow-up was, and what the analysis intended to establish. These details are necessary for interpreting even straightforward descriptive figures.
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 main limitation is the study’s small listed enrollment of 2. With so few observations, the recorded safety counts provide little precision and limited information about events that may occur infrequently. The registry does not report confidence intervals or another measure of uncertainty for these figures.
The single-group design has no concurrent comparator. It cannot support a controlled estimate of differences in adverse events or ocular outcomes, and it cannot isolate the role of Microplasmin from other influences. The registry explicitly cautions that the arms are not a like-for-like control comparison. Allocation is listed as NA and masking as none.
The endpoint and safety summaries are not aligned in the information reported: the primary endpoint is the number of adverse events, while the posted safety figures give participants at risk and affected. The registry does not report the total number of separate events. Definitions, event-level records, attribution assessments, follow-up completeness, and death figures are not reported in the registry record.
Results for the listed secondary outcomes are not reported in the registry record. Although the outcomes specify measurement concepts and assessment times, there are no listed analyses and no reported analysis populations, missing-data methods, or multiplicity approach. The reason for termination is also not reported, so the status cannot be used to infer why the study ended or whether planned assessments were completed.
The intervention’s dose and schedule, detailed eligibility criteria, and further conduct procedures are not reported. These omissions limit reproducibility and contextual interpretation. The trial’s registered phase does not fill in those details. Taken together, the design and reporting support a narrow descriptive reading of the listed adverse-event figures, not a definitive evaluation of efficacy or comparative safety.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Microplasmin Intravitreal Administration in Participants With Uveitic Macular Edema — NCT01194674.
- Registry record last updated 2018-07-31; status last verified 2018-07.
- Results first posted 2012-08-14.
- 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
NCT01194674 is a Phase 1 and Phase 2, single-group study of intravitreal Microplasmin in participants with uveitic macular edema. Enrollment is listed as 2. The study is marked terminated, with a start date of 2011-01 and primary completion date of 2011-12. The sponsor is the National Institutes of Health Clinical Center (CC).
The primary endpoint is the number of adverse events over 24 weeks. The registry records 0 affected among 2 at risk for serious adverse events and 1 affected among 2 at risk for other adverse events. These are participant-level figures, not a reported total event count. No serious adverse-event terms are listed, and death figures are not reported in the registry record.
Secondary outcomes include retinal thickness, posterior vitreous detachment, visual acuity, and retino-vascular leakage. The registry lists nine outcome measures and zero analyses; results for the secondary outcomes, statistical analysis details, missing-data handling, and the reason for termination are not reported in the registry record. The single-group design and limited enrollment restrict interpretation to cautious description of the recorded counts.