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 Suprachoroidal Injection of CLS-TA in Patients With Non-infectious Uveitis (NCT03097315), a na phase 3 study. The primary outcome is Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03097315 |
| Phase | Phase 3 |
| Condition | Uveitis, Uveitis, Posterior, Uveitis, Anterior |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 38 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Clearside Biomedical, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
People with non-infectious uveitis, including forms affecting the anterior, intermediate, or posterior eye and panuveitis. The record describes the safety population as patients who entered the study and received at least one dose of CLS-TA. The posted safety results give a denominator of 38 participants.
Intervention
A suprachoroidal injection of 4 mg CLS-TA, described as 40 mg/mL in a 100 μL injection. CLS-TA is triamcinolone acetonide injectable suspension. The study was open-label, so masking was not used.
Comparator
There was no comparator group in the registry design. The study is recorded as a single-group study with allocation listed as not applicable. Its results therefore describe outcomes among participants receiving CLS-TA; they do not provide a like-for-like comparison with another treatment or with no treatment.
Primary question
How many participants experienced treatment-emergent adverse events or serious adverse events from baseline to 24 weeks? The primary endpoint is a count of participants, not a comparative estimate of benefit or harm. The registry record reports 24 participants with treatment-emergent adverse events and 1 with a serious adverse event, among 38 at risk.
The study concerns safety in patients with non-infectious uveitis, with or without macular edema, according to its brief description. The central statistical question is descriptive: what adverse-event experience was recorded in the treated group during the stated follow-up period? Because the study has no control group and is unmasked, these results cannot distinguish events related to the injection or drug from events that might have occurred for other reasons. The record also lists measures of intraocular pressure and inflammatory findings as secondary outcomes, but their results are not reported in the registry record.
3. Trial Design
This was a Phase 3, open-label, single-group safety study of suprachoroidal CLS-TA in patients with non-infectious uveitis. The registry lists allocation as not applicable and masking as none. There was one intervention group: a suprachoroidal injection of 4 mg CLS-TA, described as 40 mg/mL in 100 μL. The registry enrollment was 38 participants. The study status is completed; the recorded start date is 2017-04-04 and the primary completion date is 2018-01-24.
The primary outcome was the number of patients with treatment-emergent adverse events and serious adverse events, measured from baseline to 24 weeks. The safety population is defined in the registry as all patients who entered the study and received at least one dose of CLS-TA. The posted safety group has 38 participants at risk for both the serious and other adverse-event counts. These details describe the population and observation window for the safety summary, but they do not establish that every participant had identical follow-up or complete outcome ascertainment.
A single-group design can describe what was observed after treatment, but it cannot estimate a treatment contrast against a concurrent control. Open-label conduct also means participants and study personnel were not masked to treatment. The registry record does not report a randomized allocation procedure, a control arm, or a masking procedure; those features are not part of the recorded design. The record does not report a sample-size justification or a detailed statistical analysis plan. It lists five outcome measures, including the primary safety outcome and secondary measures concerning intraocular pressure, anterior chamber cells and flare, and vitreous haze. Results for those secondary measures are not reported in the registry record.
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 | 4 mg CLS-TA Suprachoriodal Injection |
|---|---|
| STARTED | 38 |
| Intention-to-Treat Population | 38 |
| Safety Population | 38 |
| COMPLETED | 37 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | 4 mg CLS-TA Suprachoriodal Injection |
|---|---|
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: The Safety Population included all patients who entered the study and were administered at least one dose of CLS-TA
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: The Safety Population included all patients who entered the study and were administered at least one dose of CLS-TA
| Characteristic | Unit | 4 mg CLS-TA Suprachoriodal Injection |
|---|---|---|
| Participants analysed | 38 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 25 |
| Age, Categorical: >=65 years | Participants | 13 |
| Age, Continuous | years | 52.4 (SD 15.8) |
| Sex: Female, Male: Female | Participants | 25 |
| Sex: Female, Male: Male | Participants | 13 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 5 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 33 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 7 |
| Race (NIH/OMB): White | Participants | 31 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 38 |
| Type of Uveitis in the Study Eye: Anterior uveitis | Participants | 12 |
| Type of Uveitis in the Study Eye: Intermediate uveitis | Participants | 15 |
| Type of Uveitis in the Study Eye: Posterior uveitis | Participants | 10 |
| Type of Uveitis in the Study Eye: Panuveitis | Participants | 10 |
6. Primary Endpoint
The registry lists the primary outcome as Adverse Events, measured over Baseline to 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants who experienced treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) during the period from baseline to 24 weeks. A TEAE is defined in the record as an adverse event that begins on or after the first dose of study drug, or a worsening of a pre-existing condition relative to the pre-treatment state. The definition does not require that the event be judged causally related to the product.
An SAE is an event meeting one or more seriousness criteria described in the registry, such as resulting in death, being immediately life-threatening, requiring hospitalization or prolongation of hospitalization, causing persistent or significant disability or incapacity, involving a congenital abnormality or birth defect, or constituting an important medical event that could jeopardize the participant or require intervention to prevent a listed outcome. These criteria classify seriousness; they do not by themselves establish that treatment caused an event.
Counting participants with events is a direct way to describe how many people in the safety population had at least one qualifying event. It is useful for summarizing observed safety experience in a study, especially when the question concerns the occurrence of events rather than a change in a continuous measurement. The endpoint does not convey how many events each participant had, their duration, or their severity beyond the separate serious-event classification. Nor does a count in a single treatment group show how the event experience compares with what would have happened under another treatment or without treatment.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record reports 24 participants with treatment-emergent adverse events among 38 participants at risk, and 1 participant with a serious adverse event among 38 at risk. It reports 0 deaths. The leading serious-event term listed is “Visual acuity reduced,” with 2.6% shown in the record. These are descriptive safety findings for the study group and the stated follow-up period, not estimates of a difference between treatments.
The distinction between an adverse event and a treatment-emergent adverse event matters. An event is classified as treatment-emergent based on its timing relative to the first dose or worsening from the pre-treatment state. That timing definition does not establish causation. Similarly, serious describes an event’s seriousness under the listed criteria, not its relationship to CLS-TA. The registry definition explicitly allows adverse events whether or not considered causally related to the product.
These counts alone do not describe the full distribution of events. The count of participants with at least one TEAE does not indicate how many separate events occurred, whether some participants experienced repeated events, or how long events lasted. The registry also does not provide a detailed listing of all non-serious event types in the registry record for this analysis. The one serious-event participant and the serious-event term should therefore be interpreted as a concise summary, not a complete clinical account.
Precision is limited by the size and structure of the study. In general, estimates based on a small group can be uncertain, and a count may change noticeably when a small number of participants have or do not have an event. No confidence intervals or inferential analysis are reported in the registry record. A confidence interval, in general, expresses the statistical uncertainty around an estimate under a specified method; without one, the counts should not be read as precisely locating the event frequency in a broader population.
The single-group, open-label design further limits interpretation. Without a concurrent comparator, the registry results cannot establish whether the observed events were more or less common than under another approach. They also cannot isolate the contribution of treatment from underlying disease, other care, or other circumstances. The findings describe the safety experience recorded for participants in this study; they do not establish a causal treatment effect or settle questions beyond the study population and observation period.
9. Statistical Methods Explained
The primary outcome is a participant count. The registry reports how many participants had TEAEs and how many had SAEs during baseline to 24 weeks, with 38 participants at risk for each summary. This is a descriptive method: it organizes observed outcomes without estimating a contrast between study groups. Since this study has one group, there is no between-group treatment effect to estimate from the reported primary endpoint.
What does a participant count tell the reader?
A count records the number of people meeting an outcome definition at least once. It is not the same as a count of all event episodes. If one person experiences multiple qualifying events, a participant-level count still identifies that person once for the relevant outcome. The registry describes the primary outcome as the number of patients with TEAEs and SAEs, and the posted safety summaries are presented as participant counts. They do not provide a complete account of event recurrence, duration, or severity for every event.
The denominator is important to interpreting a count. Here, the record identifies 38 participants at risk for the serious-event count and 38 for the other adverse-event count. It defines the safety population as participants who entered the study and received at least one dose. The record does not provide further detail in the registry record about participant-level follow-up completion or how missing outcome information, if any, was handled. A denominator should therefore be understood as the group specified for that registry summary, not as proof that every participant contributed identical observation time.
How do TEAEs and SAEs differ?
TEAE classification is based on timing: an adverse event must begin on or after the first dose or represent a worsening relative to the pre-treatment state. The registry definition allows the event to be counted whether or not it is considered causally related to the product. SAE classification is based on seriousness criteria, including outcomes such as death, immediate life threat, hospitalization, significant disability, or an important medical event. The two categories answer different questions. Treatment-emergent describes when an event occurred relative to treatment; serious describes the event’s consequence or importance under the listed definition. Neither label alone establishes causation.
What can a confidence interval add?
In general, a confidence interval gives a range of values compatible with the observed data under a statistical model and a chosen method. It helps communicate uncertainty that a point estimate or count cannot express by itself. The registry record does not report confidence intervals for the primary safety outcome. Without such intervals, readers should avoid treating the observed counts as exact estimates of the event experience that would occur in every similar population. The study’s group size and single-group design also mean that uncertainty about the broader population cannot be resolved by the raw count alone.
Why does the design matter for statistical interpretation?
A randomized controlled comparison can, under suitable design and analysis, help distinguish outcomes between assigned groups. This study is recorded as single-group, with allocation not applicable and no masking. Its posted safety results therefore describe the treated participants, rather than a treatment contrast. No statistical adjustment can create a concurrent comparison group that the design did not include. Likewise, the open-label design means the record does not describe masked assessment; the possible influence of awareness on reporting or assessment cannot be evaluated from the information reported.
The record lists a sample-size planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a fraction of the observed effect. Those assumptions concern a planning illustration, not the design of this single-group safety study. The registry does not report that this planning exercise was the study’s own design rationale. It must not be interpreted as observed power or as evidence that the study was planned around a particular comparative hypothesis. No numerical planning result is stated in the registry record for this analysis.
The record reports no analyses. It does not report a statistical model, an adjusted analysis, or an inferential comparison for the primary endpoint. For the safety endpoint, the most defensible reading is therefore descriptive: identify the outcome definition, the number of participants at risk, the number with the outcome, and the stated observation period, while keeping the design limitations in view.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists five outcome measures: the primary adverse-event outcome and secondary measures for mean intraocular pressure, anterior chamber cell grade, anterior chamber flare grade, and vitreous haze grade. The record lists no analyses, and results for the secondary measures are not reported in the registry record. A multiplicity strategy for interpreting several outcomes is not reported in the registry record.
In general, multiplicity arises when several outcomes or statistical tests are considered together. If each is assessed separately, the chance of at least one apparently unusual result can increase; statistical plans may address this through prespecified hierarchies or adjustments. No such approach is reported in the registry record, so it is not possible to determine how the listed outcomes were intended to be interpreted collectively.
The registry identifies the safety population as patients who entered the study and received at least one dose. It does not report a missing-data method for the primary outcome, rules for incomplete follow-up, or how participants with unavailable outcome information were handled. The registry entries also do not describe the completeness of follow-up for each participant. These omissions matter because counts and denominators are easiest to interpret when outcome ascertainment and missingness are clearly described.
The registry does not report interim analyses or a detailed statistical analysis plan. Those matters are not reported in the registry record and should not be inferred from the posted counts. The analysis population definition and event counts are the available basis for describing the primary safety findings.
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 |
|---|---|---|---|
| 4 mg CLS-TA Suprachoriodal Injection | 1 | 38 | 2.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 4 mg CLS-TA Suprachoriodal Injection | 0 of 38 |
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 |
|---|---|
| 4 mg CLS-TA Suprachoriodal Injection | 24 of 38 |
Most frequent serious adverse event terms
| Event term | Organ system | 4 mg CLS-TA Suprachoriodal Injection |
|---|---|---|
| Visual acuity reduced | Eye disorders | 1 of 38 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 4 mg CLS-TA Suprachoriodal Injection |
|---|---|---|
| Conjunctival haemorrhage | Eye disorders | 5 of 38 |
| Intraocular pressure increased | Investigations | 4 of 38 |
| Intraocular pressure increased | Investigations | 4 of 38 |
| Eye pain | Eye disorders | 3 of 38 |
| Procedure headache | Injury, poisoning and procedural complications | 3 of 38 |
| Eye inflammation | Eye disorders | 2 of 38 |
| Eye inflammation | Eye disorders | 2 of 38 |
| Ocular hypertension | Eye disorders | 2 of 38 |
| Visual acuity reduced | Eye disorders | 2 of 38 |
| Cataract | Eye disorders | 2 of 38 |
Serious events with the highest arm-level rate: visual acuity reduced (2.6%).
The safety summary reports 1 participant with a serious adverse event among 38 at risk, 24 participants with other adverse events among 38 at risk, and 0 deaths. “Visual acuity reduced” is listed as the leading serious-event term, with 2.6% reported. The record gives counts of affected participants; these figures are not a complete description of event episodes or participant-level clinical courses.
The serious-event count refers to participants meeting the registry’s SAE definition during baseline to 24 weeks. Seriousness is defined by criteria such as life threat, hospitalization, significant disability, or an important medical event. It is not synonymous with treatment-related. The record’s TEAE definition also makes clear that an event can qualify based on timing or worsening whether or not it is considered causally related to the product.
The 2.6% associated with “Visual acuity reduced” is reported as a serious-event term. It should not be treated as a comparative risk or as evidence that CLS-TA caused the event. Because the study has no comparator group, the figures describe the observed safety experience in this group only. The registry does not provide further detail about the event’s course or causal assessment in the registry record.
12. What the Result Does, and Does Not, Mean
The results establish that adverse events were recorded among participants who received CLS-TA in this open-label, single-group study. The registry reports 24 participants with TEAEs and 1 with an SAE among 38 at risk over baseline to 24 weeks, with 0 deaths. These are the posted descriptive safety findings. The listed serious-event term is “Visual acuity reduced,” reported as 2.6%.
They do not establish that CLS-TA caused any particular event. TEAE status reflects timing or worsening relative to the first dose or pre-treatment state, while SAE status reflects seriousness criteria. Neither classification alone proves a causal link. Without a concurrent comparator, there is no registry-based estimate of how the event counts differ from those under another treatment or no treatment.
The counts also do not determine how often events occurred in total, how long they lasted, or whether individual participants had recurrent events. Nor do the registry entries provide inferential estimates or confidence intervals for the primary safety outcome. The results should therefore be read as observations in this study rather than precise estimates that necessarily apply to a wider population.
The study’s scope is bounded by its recorded population, intervention, design, and follow-up period. It enrolled 38 participants and evaluated a 4 mg suprachoroidal CLS-TA injection in patients with non-infectious uveitis. Secondary outcomes were listed, but their results are not reported in the registry record. The record also does not report a detailed analysis plan, missing-data procedures, or a sample-size justification. These limits leave important questions about precision, outcome completeness, and comparative interpretation unanswered.
In general, safety evidence from a single-group study can describe events observed after exposure and can identify topics for further study. It cannot, by itself, separate treatment effects from background events, disease-related events, or other influences. The findings do not establish a benefit or lack of benefit, and they do not support a treatment recommendation.
13. Why This Trial Matters Statistically
This study is statistically informative because it illustrates the difference between describing safety outcomes and estimating a comparative effect. Its primary endpoint counts participants with TEAEs and SAEs, while its single-group design has no concurrent control group. The result is a record of observed events after treatment, not an estimate of how outcomes would differ under an alternative.
Clear definitions are central to interpreting such counts. A TEAE is identified by timing or worsening from the pre-treatment state, whereas an SAE is identified by seriousness criteria. These labels are not interchangeable, and neither label alone establishes that the intervention caused an event. Keeping the definitions distinct prevents a descriptive safety count from being overread as a causal finding.
The study also highlights why denominators and follow-up windows matter. The record states that 38 participants were at risk for each adverse-event summary and defines the safety population as those who entered the study and received at least one dose. The primary timeframe is baseline to 24 weeks. These details anchor the counts, while the absence of reported missing-data handling and participant-level follow-up detail limits assessment of completeness.
Finally, the record lists secondary clinical measures but does not report their results, and it reports no analyses. This makes clear that registering outcomes and reporting outcome findings are distinct. For readers assessing evidence, the design, endpoint definitions, denominators, missingness information, and availability of comparative analysis all shape what can reasonably be concluded. Here, the defensible statistical conclusion is descriptive: the record documents the stated adverse-event counts in this study group, with substantial limits on comparative and causal interpretation.
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 single-group design. There is no concurrent comparator, so the observed counts cannot show whether adverse events were more or less frequent than they would have been under another treatment or without treatment. The study is also open-label, with masking listed as none. The registry record does not describe masked outcome assessment.
The primary endpoint is a count of participants with events. It does not provide the total number of event episodes, event duration, recurrence, or a full severity profile. Although one serious-event term is listed, the registry entries do not provide detailed participant-level descriptions or causal assessments. The reported counts are limited to the baseline-to-24-week timeframe.
The registry defines the safety population as participants who entered the study and received at least one dose, and reports 38 at risk for the posted safety outcomes. However, participant-level follow-up completeness and handling of missing outcome information are not reported in the registry record. No confidence intervals or inferential analyses are reported, and the record lists no analyses. Precision beyond the descriptive counts is therefore not quantified.
The registry lists secondary measures for intraocular pressure and inflammatory findings, but their results are not reported in the registry record. A multiplicity approach, interim analyses, detailed statistical analysis plan, and sample-size justification are also not reported in the registry record. The sample-size assumptions described for a planning illustration should not be mistaken for the trial’s design rationale or for observed power. These limitations restrict assessment of uncertainty, completeness, and the broader applicability of the findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Suprachoroidal Injection of CLS-TA in Patients With Non-infectious Uveitis — NCT03097315.
- Registry record last updated 2021-06-23; status last verified 2021-06.
- Results first posted 2021-06-15.
- 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
NCT03097315 is a completed Phase 3, open-label, single-group study of suprachoroidal CLS-TA in patients with non-infectious uveitis. The intervention was a 4 mg injection, described as 40 mg/mL in 100 μL. Registry enrollment was 38; the recorded study dates are 2017-04-04 to primary completion on 2018-01-24.
The primary safety outcome counted participants with TEAEs and SAEs from baseline to 24 weeks. The record reports 24 participants with TEAEs and 1 with an SAE among 38 at risk, and 0 deaths. “Visual acuity reduced” is listed as a serious-event term at 2.6%. The safety population comprised patients who entered the study and received at least one dose.
The record lists secondary outcomes for intraocular pressure and inflammatory findings, but their results, inferential analyses, missing-data procedures, and detailed statistical plan are not reported in the registry record. Because there is no comparator group, the posted findings describe observed safety outcomes and do not estimate a comparative or causal effect.