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 Open Label Study of Acthar SQ Gel Injection in Patients With Active Anterior Uveitis (NCT02769702), a na phase 4 study. The primary outcome is Change From Baseline in Eye With Uveitis of Anterior Chamber Cellularity Graded From 0-4 on a Likert Scale Determined by Slit Lamp Examination.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02769702 |
| Phase | Phase 4 |
| 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 | Washington University School of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with active anterior uveitis who were not well controlled with, or were intolerant of, topical or systemic corticosteroids. The registry record describes uveitis as an inflammatory condition and identifies uveitis as the condition studied. Further eligibility criteria and participant characteristics are not reported in the registry record.
Intervention
Participants received Acthar 80 IU by subcutaneous injection twice weekly. The study was open-label, so masking was listed as none. The registry record does not report further details about treatment adherence, dose changes, or concomitant therapies.
Comparator
There was no comparator group. The study is listed as a single-group trial with allocation marked not applicable. It therefore does not provide a direct comparison between Acthar and placebo, usual care, or another treatment.
Primary question
The primary endpoint asked how anterior chamber cellularity in the affected eye changed from baseline to 12 weeks, as assessed by slit-lamp examination and graded on a Likert scale from 0 to 4. Higher scores indicate more cellularity. The registry record does not report the observed change, its variability, or a statistical analysis.
In practical terms, the study was intended to describe change in a clinical measure of anterior eye inflammation during treatment in the specified patient group. Because it had one treatment group and no masking, the design cannot separate change during treatment from changes that might have occurred over time for other reasons. Its registry record also does not contain outcome results with which to quantify the observed course.
3. Trial Design
This was a Phase 4, prospective, open-label, single-group study sponsored by Washington University School of Medicine. The registry lists allocation as not applicable and masking as none. Participants received Acthar 80 IU by subcutaneous injection twice weekly. No comparison arm is recorded, so the design does not support a randomized between-group treatment contrast.
The registry lists an enrollment of 2 and a status of terminated. The study start is recorded as 2016-06, and primary completion as 2017-01-06. The reason for termination and the circumstances of recruitment or follow-up are not reported in the registry record. The status alone does not establish why the study ended or whether participants completed the intended follow-up.
The primary measure was change from baseline in anterior chamber cellularity in the eye with uveitis, graded from 0 to 4 by slit-lamp examination; the stated timeframe was baseline and 12 weeks. A secondary outcome was change from baseline in anterior chamber protein, also graded from 0 to 4 by slit-lamp evaluation. Results for these outcomes are not reported in the registry record.
The record lists two outcome measures and zero analyses. It does not report the statistical analysis plan, analysis populations, missing-data procedures, or whether any interim analyses were planned or performed. Although the registry describes the trial as prospective, additional operational details such as the number of sites and the timing of individual visits are not reported. The study design and record therefore permit a description of the intended clinical question, but not an estimate of treatment-associated change from the posted results.
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 | Acthar 80 IU |
|---|---|
| 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 | Acthar 80 IU |
|---|---|---|
| Participants analysed | 2 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Eye With Uveitis of Anterior Chamber Cellularity Graded From 0-4 on a Likert Scale Determined by Slit Lamp Examination, measured over Baseline and 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | units on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change from baseline in anterior chamber cellularity in the eye with uveitis, assessed at baseline and 12 weeks. Slit-lamp examination is the specified assessment method, and cellularity is graded on a Likert scale from 0 to 4. Higher scores represent more cellularity. The registry describes this measure as a standard assessment of uveitis activity.
In general, an endpoint is chosen to represent an outcome relevant to the clinical question. Here, the endpoint focuses on an observable sign in the anterior chamber rather than a patient-reported outcome. A change score describes how the recorded measurement differs from its baseline value; its interpretation depends on having the paired measurements and a clear account of how missing observations were handled. Those details and the actual endpoint results are not reported in the registry record.
The scale is ordered: higher categories indicate greater cellularity, but the distance between adjacent categories is not necessarily equal. The registry specifies a mean and standard deviation for this continuous measure. That summary can describe the distribution numerically, but does not by itself show each participant’s course or establish that equal spacing between scale categories is clinically meaningful. No mean change, standard deviation, confidence interval, or test result is reported in the registry record.
The secondary outcome concerns anterior chamber protein graded from 0 to 4 by slit-lamp evaluation. Its result is also not reported in the registry record. Without the posted results, neither endpoint can be used to quantify the observed change.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the planned primary measure and the study’s single-group design, but it does not report the primary outcome result. There is no posted mean change in anterior chamber cellularity, standard deviation, confidence interval, or statistical test result. The secondary outcome, change in anterior chamber protein, also has no result reported. Consequently, the record does not quantify the direction, size, or precision of change on either measure.
The recorded enrollment was 2. This is a very limited basis for describing outcome patterns, even if individual measurements were available. In a study with a single group, any measured change over time would not by itself distinguish an effect associated with treatment from natural variation, regression to the mean, changes in other care, or measurement variation. Those are general considerations for interpreting an uncontrolled before-and-after design; the registry does not report whether any of them occurred in this study.
The absence of a comparator also means there is no direct estimate of how outcomes might have differed under another treatment or no study treatment. Open-label status and no masking are recorded. These design features can matter when assessments involve clinical grading, although the registry does not report who performed the examinations or whether outcome assessors were masked.
The status is terminated, but the reason for termination is not reported in the registry record. It would be inappropriate to infer from that status alone whether termination was related to recruitment, feasibility, safety, efficacy, or another factor. Nor does the status establish whether the listed follow-up was completed.
The safety record lists 0 participants affected by serious adverse events among 2 at risk, and 0 affected by other adverse events among 2 at risk. These are the registry’s recorded figures; they do not replace detailed event descriptions or establish the safety profile beyond the reported record. Deaths are not reported in the registry record.
Overall, the posted record documents the study question, the intervention, a small enrolled group, and a terminated status, but it supplies no efficacy outcome estimates. It therefore supports neither a numerical estimate of change nor a conclusion about the precision of such an estimate. General caution is warranted when considering an uncontrolled study with very limited enrollment, but no treatment conclusion can be drawn from the missing outcome results.
9. Statistical Methods Explained
What does the endpoint summary mean?
The primary endpoint is a continuous-style summary of change in an ordered clinical scale. The registry describes the parameter as a mean and the dispersion as a standard deviation. In general, a mean represents the arithmetic average of measurements, while a standard deviation describes how spread out measurements are around that average. For a change endpoint, the underlying quantity is the difference between a follow-up measurement and baseline for each participant, summarized across the analysis group. The registry record does not report the mean change or standard deviation, so neither can be interpreted for this study.
The cellularity score runs from 0 to 4, with higher scores indicating more cellularity. Although the record specifies a mean, the scale is ordered rather than necessarily equally spaced. In general, treating an ordered scale numerically may be convenient, but interpretation should consider what the score categories mean and whether numerical summaries adequately describe the data. With very limited enrollment, individual observations can strongly influence a mean and spread. The registry does not provide participant-level scores or category distributions.
What would a confidence interval add?
In general, a confidence interval expresses uncertainty around an estimate under the assumptions of a statistical method. A narrower interval indicates greater precision within that method; a wider interval indicates more uncertainty. It is not a range that guarantees the true value lies inside it, nor does it correct for problems such as missing data, bias, or an unsuitable design. No confidence interval is reported for either outcome in the registry record. Without an estimate and its uncertainty, readers cannot judge the precision of a treatment-associated change from the posted results.
What can a single-group design estimate?
A single-group study can describe measurements observed in participants receiving the study intervention. If baseline and follow-up measurements are available, an analysis can describe within-person change. In general, this is different from estimating a comparative treatment effect. A comparison group helps account for changes that could occur without the intervention, including time trends and other influences. Because this study has no comparator, a before-and-after difference, even if it had been reported, would not by itself establish that the intervention caused the difference.
Open-label status means masking was listed as none. In general, knowledge of treatment can influence behavior, reporting, clinical management, or assessments. Slit-lamp cellularity grading is a clinical assessment, so assessor procedures can matter; however, the registry does not identify who assessed the endpoint or describe any safeguards against assessment bias.
How should the planning assumptions be read?
The registry includes assumptions for a planning illustration based on an observed effect: a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. The illustration is described as detecting a stated fraction of the observed effect. It is not the design of this single-group trial, which has no two-arm allocation. No numerical planning results are reported in the registry record, and the record does not describe this illustration as the trial’s own sample-size justification.
In general, a planning calculation asks how many participants per arm might be needed under specified assumptions to detect a target effect with a chosen level of power. Such a calculation is hypothetical and depends on the effect size, variability, statistical method, and other assumptions. It is not observed power and does not tell readers what the study actually found. The registry’s assumptions do not provide a basis for reconstructing the missing endpoint results, and no new estimates can be derived from the posted record.
Why do dispersion and scale properties matter?
Standard deviation is useful for describing variability in a numerical measure, but it does not show the full distribution. In general, distributions can be asymmetric, concentrated in a few scale categories, or affected by extreme observations. This is especially relevant when the underlying measurement is a bounded score with ordered categories. A mean and standard deviation can therefore be supplemented by category counts or individual-level summaries, if available. The registry record does not report such information.
Over-dispersion is a general concern when observed variability exceeds what a chosen statistical model assumes. If present, a model that understates variability may produce confidence intervals that are too narrow or tests that are too optimistic. This is a general methodological point, not a finding about this study: the registry provides no outcome distribution or analysis with which to assess over-dispersion.
The analysis method, assumptions about the scale, handling of repeated measurements, and approach to incomplete observations are not reported in the registry record. Since the record lists zero analyses, there is no posted inferential result to evaluate against the endpoint definition or planning assumptions.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome for anterior chamber cellularity and a secondary outcome for anterior chamber protein. When a study evaluates multiple outcomes or performs multiple statistical tests, the chance of a false-positive finding can increase. In general, a statistical analysis plan may specify which outcome is primary, how secondary outcomes will be interpreted, and whether any adjustment for multiple testing will be used. The registry record does not report a multiplicity strategy, and it reports no outcome analyses. No claim about statistical significance or adjustment can therefore be assessed.
Missing-data handling is also not reported in the registry record. In general, an analysis should clarify how participants with absent baseline or follow-up measurements are treated, whether the analysis uses available cases or another approach, and what assumptions are made about why measurements are missing. These choices can affect a change-from-baseline estimate. The record does not say whether any measurements were missing or how they were handled.
The analysis population is not reported in the registry record. For example, the record does not specify whether the intended analysis included all enrolled participants, only participants with follow-up measurements, or another defined group. No participant-level outcome data or analysis denominators are posted, so the composition of an outcome analysis cannot be established.
The record does not report an interim-analysis plan, stopping criteria, or the reason for termination. These omissions do not establish that such procedures were or were not used; they mean that the information is not reported in the registry record. These details would be relevant to understanding how the study was conducted and how any eventual results should be interpreted.
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 |
|---|---|---|---|
| Acthar 80 IU | 0 | 2 | 0.0% |
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 |
|---|---|
| Acthar 80 IU | 0 of 2 |
The safety record lists 0 participants affected by serious adverse events among 2 at risk. It also lists 0 participants affected by other adverse events among 2 at risk. These figures describe what the registry records for the Acthar 80 IU group. They should be read as counts of participants affected, not as detailed descriptions of event types, timing, severity, or relatedness.
The registry lists no serious adverse-event terms. It does not provide event narratives or further information on monitoring. Deaths are not reported in the registry record. A recorded zero for affected participants is not the same as a complete account of all safety observations, and the small enrolled group limits how much can be learned about uncommon events. These figures describe the posted safety record and do not establish a broader safety profile.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase 4, open-label, single-group study of Acthar was conducted in patients with active anterior uveitis who were not well controlled with, or were intolerant of, topical or systemic corticosteroids. It identifies the intended primary endpoint as change in anterior chamber cellularity from baseline to 12 weeks, assessed by slit-lamp examination on a scale from 0 to 4. It also lists a secondary anterior chamber protein measure.
The registry record does not report results for either endpoint. There is no posted estimate of mean change, variability, confidence interval, or statistical test. As a result, the record does not say how cellularity or protein scores changed among participants, or how precisely any change was estimated. The absence of reported outcomes should not be read as evidence that there was no change.
The study has no comparator group, and masking is listed as none. Consequently, even a reported change over time would not, by itself, establish that the treatment caused that change or show how outcomes compare with an alternative. The record’s terminated status does not explain why the study ended and should not be used to infer an efficacy or safety finding.
The safety figures record 0 participants affected by serious adverse events and 0 affected by other adverse events among 2 at risk. Those entries are limited to the registry record; deaths are not reported. They do not provide a complete event-level account or establish what might be observed in a larger or different population.
Thus, the posted information is primarily descriptive of the study’s design and registry status. It does not support a quantified treatment effect, a comparative conclusion, or a conclusion about the precision of clinical outcomes. General statistical interpretation must remain separate from claims about treatment, because the outcome estimates needed to evaluate change are not reported.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between a clearly stated clinical question and an answerable treatment-effect question. The registry identifies a clinical measure, an assessment method, a timeframe, and a treatment group. But the absence of reported endpoint results means that readers cannot quantify even the within-group course, let alone compare it with an alternative.
The single-group design also clarifies why baseline-to-follow-up change and comparative effect are not interchangeable. In general, a before-and-after description can show how measurements vary over time among treated participants. Without a comparator, it cannot isolate the treatment’s contribution from other influences. The open-label status adds another consideration for a clinically assessed endpoint, although the registry does not describe assessor procedures.
The enrollment of 2 highlights how limited enrollment constrains what a study can describe. In general, estimates based on very few observations are sensitive to individual measurements, and rare safety events cannot be characterized reliably from such a record. Here, the missing outcome results further prevent assessment of variability or precision.
Finally, the terminated status and absent analysis details show why registry completeness matters. The record does not state why the study was terminated, how missing measurements were handled, or what analysis population was used. These gaps prevent a transparent account of how the intended question was translated into an analysis. The statistical importance of the study record lies less in a reported estimate than in the limits imposed by its design and the absence of posted outcome analyses.
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 record does not report results for the primary or secondary outcome. Without endpoint estimates and variability, readers cannot assess the magnitude, direction, or precision of change. The record also lists zero analyses, so there are no posted statistical results to evaluate.
The study was single-group, with allocation marked not applicable and no comparator recorded. Any within-group change would therefore lack a concurrent control for interpreting what might have happened over the same period without the intervention. Masking is listed as none. Since the primary measure involves clinical grading, the absence of details about who assessed it and how assessments were conducted limits assessment of potential measurement bias.
Enrollment was 2, which restricts the amount of information the study could provide about outcome variation and safety. The registry status is terminated, but the reason for termination and the circumstances of follow-up are not reported. The record does not establish whether the listed 12-week assessment was completed for all enrolled participants.
The registry does not report participant characteristics, detailed eligibility criteria, analysis populations, missing-data procedures, or the statistical analysis plan. It does not describe whether there were protocol deviations, treatment discontinuations, or changes in concomitant care. Those matters cannot be inferred from the available record. In combination, the limited enrollment, lack of comparator, open-label design, and missing outcome analyses mean the record supports only a narrow description of the intended study and its status, not an estimate of treatment effect.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Open Label Study of Acthar SQ Gel Injection in Patients With Active Anterior Uveitis — NCT02769702.
- Registry record last updated 2018-10-01; status last verified 2018-09.
- Results first posted 2018-10-01.
- 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 Phase 4, prospective, open-label, single-group study sponsored by Washington University School of Medicine. It evaluated Acthar 80 IU by subcutaneous injection twice weekly in patients with active anterior uveitis who were not well controlled with, or were intolerant of, topical or systemic corticosteroids. Enrollment was 2. The study began in 2016-06, had a primary completion date of 2017-01-06, and is listed as terminated.
The primary outcome was change from baseline in anterior chamber cellularity at 12 weeks, graded from 0 to 4 by slit-lamp examination. A secondary outcome assessed change in anterior chamber protein on a 0-to-4 scale. The registry record reports no analyses and no results for either measure. It lists 0 participants affected by serious adverse events and 0 affected by other adverse events among 2 at risk; deaths are not reported. The reason for termination and further analysis details are not reported in the registry record.