Oral OKT3 for the Treatment of Active Ulcerative Colitis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Oral OKT3 for the Treatment of Active Ulcerative Colitis (NCT01287195), a na phase 1, phase 2 study. The primary outcome is Number of Participants With Adverse Events.

2. Clinical Question

The study concerned participants with active ulcerative colitis and assessed orally delivered short-term OKT3. The registry describes Oral OKT3 given with omeprazole once daily for 30 days. Its stated primary question was safety, measured as the number of participants with adverse events from baseline to Week 10. The registry also lists clinical activity and endoscopic histology outcomes, but their results are not reported in the registry record.

Because all participants were assigned to the same study group, the record cannot provide a between-group estimate of how outcomes differ under OKT3 versus another treatment or no study treatment. The posted safety information gives counts for serious and other adverse events and deaths. It does not report a complete overall primary-endpoint result or numerical results for the listed clinical outcomes. That distinction matters: the trial's stated purpose includes efficacy, but the available outcome information described in the registry does not allow a reader to assess efficacy from these data.

3. Trial Design

This was a completed Phase 1/Phase 2 clinical trial sponsored by Brigham and Women's Hospital. The registry records a single-group model, allocation as not applicable, and no masking. Enrollment was 6. The study start date was 2011-04-07 and the primary completion date was 2013-05-02. These design features describe a small, unmasked study without a concurrent control group.

Participants with ulcerative colitis were to receive Oral OKT3 with omeprazole once daily for 30 days. The primary outcome timeframe was from baseline to Week 10. That is the stated observation window for the primary safety endpoint; details about visit schedules, the timing of individual assessments, or how adverse events were collected are not reported in the registry record.

The registry lists Mayo Score, Simple Clinical Colitis Activity Index (SCCAI) Score, and a score in histologic evaluation of flexible sigmoidoscopy as secondary outcomes. It records 8 outcome measures overall, but numerical results for those secondary outcomes are not reported in the registry record. The existence of listed outcomes does not by itself show whether they were assessed for every participant or how they changed during follow-up.

A single-group design can describe outcomes among participants who received the study regimen, but it does not separate changes associated with treatment from changes that could occur for other reasons. With no randomized allocation and no comparator arm, treatment contrasts cannot be estimated from this design. The absence of masking also means the registry does not describe a blinded study; further information about outcome assessment procedures is not reported in the registry record. The record does not report a sample-size justification or explain how the enrollment target was chosen.

The registry status is completed. Completion indicates the recorded study status, not that every planned outcome or analysis has a posted numerical result. The record lists no analyses, so inferential results such as treatment-effect estimates, confidence intervals, or hypothesis-test results are not available in the posted information.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All participants enrolled in the study.

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: All participants enrolled in the study.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Adverse Events, measured over From baseline to Week 10.

The primary endpoint is the number of participants with adverse events from baseline to Week 10. It is a participant-count endpoint: the intended summary is how many enrolled people experienced an event during the specified period, rather than the total number of event episodes or the severity score of a symptom. The registry defines an adverse event broadly as an untoward medical occurrence after administration of a pharmaceutical product, whether or not considered related to that product. The definition includes unfavorable signs, abnormal laboratory findings, symptoms, or diseases; it also includes preexisting conditions that worsen during the study.

Safety endpoints are used to describe harms observed during a study and to establish what kinds of events occurred among participants under observation. A broad definition can capture events without presuming that the study treatment caused them. This is important when interpreting the count: an adverse event occurring during a study is not automatically an adverse reaction caused by the treatment.

The endpoint population is all participants enrolled in the study. The registry reports serious-event and other-event participant counts, along with deaths, but an overall primary-endpoint count is not reported in the registry record. The serious-event and other-event counts should not be combined to produce a total because the record does not explain whether participants overlap across those categories. Nor does a participant count indicate how many separate event episodes each participant experienced.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a participant-count endpoint describe?

The primary outcome is the number of participants with adverse events. This type of endpoint counts people who meet an event definition during a specified observation period. It differs from a count of all event episodes: a participant with more than one event may still contribute only once to a participant-level count, depending on the analysis definition. The registry names the endpoint as a participant count and defines adverse events broadly, but a detailed counting rule for repeated events is not reported in the registry record.

The observation period is from baseline to Week 10. A timeframe helps establish which events are included, but it does not tell readers how frequently participants were assessed or how events arising between visits were collected. Those operational details are not reported in the registry record. The stated population is all enrolled participants, which identifies the intended denominator for the primary outcome, but the registry does not report a full overall endpoint result.

Why distinguish serious from other adverse events?

Safety reporting commonly separates serious events from other events because seriousness describes an important classification of an event, not simply how often it happened. These categories should be read according to the registry's reported labels. The posted data give serious-event and other-event participant counts, and list deaths separately. They do not explain category overlap or provide a combined total. General statistical explanation: overlapping categories cannot be added to obtain a unique-person total unless the overlap is known or the categories are explicitly mutually exclusive.

The adverse-event definition in this record does not require a causal relationship with treatment. This is useful for broad surveillance because it captures medical occurrences during the study without first deciding whether the study regimen caused them. It also means that event counts are not, by themselves, evidence of causation. A causal assessment would require additional evidence and methods not described in the registry record.

What can a single-group design estimate?

A single-group study can report the outcomes observed among enrolled participants. It has no concurrent control group, so it cannot estimate a treatment contrast from randomized groups. Changes in symptoms or event counts could reflect multiple influences, including the underlying condition and events unrelated to treatment. General statistical explanation: randomization helps balance measured and unmeasured prognostic factors between comparison groups on average; without randomized comparison, that protection against confounding is absent. This study's registry entry records no allocation and a single-group model.

Masking is recorded as none. In general, masking can reduce the influence of expectations on care, reporting, and outcome assessment. The record does not report further procedures for reducing assessment or reporting bias. This limitation is especially relevant to outcomes that involve judgment, though no specific assessment procedure can be inferred from the outcome names alone.

How should uncertainty and significance be understood?

General statistical explanation: an estimate summarizes the observed data, while a confidence interval describes uncertainty around that estimate under the assumptions of the analysis. A hypothesis test evaluates data against a specified null hypothesis and statistical model. Neither a confidence interval nor a test result is reported for the primary endpoint in the registry record. No analysis results are listed, so there is no posted basis for describing statistical significance, an adjusted effect, or the precision of an effect estimate.

The record includes a sample-size planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. Such an illustration is a planning exercise, not an observed trial comparison and not observed power. The assumptions describe a hypothetical design that differs from the actual single-group trial. Numerical planning outputs are not reported in the registry record, and the registry does not report the trial's own sample-size justification.

General statistical explanation: planning calculations depend on the effect chosen for detection and on assumptions about variability, event frequency, significance level, power, and allocation. A planning illustration cannot establish that a study was adequately powered for its actual design, particularly when the assumed comparison does not match the study's design. The record does not report an interim-analysis plan or a statistical analysis plan.

Why does outcome type matter?

The primary endpoint is binary at the participant level: a participant either meets the adverse-event criterion during the defined period or does not. Counts can be informative descriptions, but estimating an event probability or comparing probabilities requires a clearly defined denominator and, for a treatment contrast, an appropriate comparison. The registry provides the enrollment count and safety-group counts but no inferential output. General statistical explanation: with sparse event data, estimates may be imprecise, and methods must reflect the data structure; no particular model or correction can be attributed to this trial because the analysis methods are not reported in the registry record.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Oral OKT3")
n      <- c(6)
events <- c(3)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Oral OKT3  6  3  50.0  11.8  88.2  18.8  81.2  18.4  81.6

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 8 outcome measures, including the primary adverse-event endpoint and secondary outcomes for Mayo Score, SCCAI Score, and histologic evaluation of flexible sigmoidoscopy. It records 0 analyses. Numerical results for the secondary outcomes and a multiplicity procedure are not reported in the registry record. Without an analysis plan, it is not possible to determine whether the outcomes were tested, how their timing or scoring was specified, or whether any adjustment for multiple comparisons was intended.

General statistical explanation: testing multiple outcomes can increase the chance of obtaining at least one apparently unusual result by chance. Prespecified outcome hierarchies or multiplicity adjustments can address that issue, but neither is described in the registry record. Since no inferential analyses are listed, claims about statistical significance across outcomes cannot be evaluated.

The primary endpoint population is described as all enrolled participants. The registry does not report how missing outcome data were handled, whether any participants lacked follow-up, or whether the analysis population differed from the enrolled group. It also does not report an imputation method, sensitivity analysis, or reasons for missingness. An absence of reported missing-data details does not establish that there were no missing data.

These omissions limit interpretation of both the safety endpoint and the listed clinical outcomes. The posted information does not establish whether each participant contributed complete Week 10 data or how incomplete observation affected the reported counts.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: ulcerative colitis (16.7%), anorectal abscess (16.7%).

For the Oral OKT3 group, the registry records 6 participants at risk for serious adverse events and 2 participants affected. It records 6 participants at risk for other adverse events and 1 participant affected. Deaths are recorded as 0. These are participant counts, not counts of all event episodes, and they describe this study group rather than a comparison between treatments.

The top serious-event terms are ulcerative colitis at 16.7% and anorectal abscess at 16.7%. The registry does not establish that these events were caused by either study drug. Its definition of an adverse event explicitly allows events to be recorded whether or not they are considered related to a pharmaceutical product. The terms and percentages should therefore be read as safety reporting, not as causal findings.

The serious and other adverse-event categories may not be mutually exclusive; whether a participant was counted in both is not reported in the registry record. The counts should not be added to claim a total number of participants with any adverse event. The record also does not give details about event timing, duration, resolution, or individual participant histories. In a group of this size, these counts are descriptive and do not precisely characterize event frequencies beyond the enrolled participants.

13. What the Result Does, and Does Not, Mean

The posted safety information establishes that the registry recorded serious adverse events in 2 participants, other adverse events in 1 participant, and deaths as 0 among the Oral OKT3 group. It also lists ulcerative colitis and anorectal abscess as top serious-event terms, each at 16.7%. These statements describe the recorded group and the outcome categories; they do not indicate that treatment caused any particular event.

The findings do not establish comparative safety because the trial had no control group and no randomized allocation. They do not establish efficacy because the numerical results for the listed clinical activity and histology outcomes are not reported in the registry record. A completed study status does not fill in missing outcome results, and the listed outcome names do not disclose whether scores improved, worsened, or remained unchanged.

The record also does not report confidence intervals or statistical tests for the safety results. It is therefore not possible to quantify precision using inferential statistics or to judge whether a treatment contrast differs from a null value. General statistical explanation: a descriptive count is a report of observed participants, whereas a treatment effect is a comparison that requires an appropriate counterfactual or control group. The single-group counts do not supply that comparison.

These limitations do not make the recorded counts meaningless. They identify the safety information documented for a small group during the stated follow-up and indicate which serious-event terms were listed. Their scope is narrow: they concern participants enrolled in this trial, not a broader population, and they cannot resolve causal questions. The record should be read as descriptive evidence, with unreported analyses and outcomes remaining unknown rather than implicitly favorable or unfavorable.

14. Why This Trial Matters Statistically

This study is statistically informative as an example of how design determines the questions that data can answer. The trial enrolled 6 participants in a single group, without masking or a comparator. That structure can document events among participants receiving the study regimen, but it cannot provide a randomized treatment contrast. The distinction is fundamental: an observed outcome in a treated group is not, by itself, an estimate of what would have happened under another treatment.

The primary endpoint also illustrates the difference between a broad safety definition and a causal conclusion. The registry defines adverse events to include medical occurrences whether or not considered related to the pharmaceutical product. Recording an event supports safety description and monitoring; attributing it to treatment requires additional evidence that the registry does not report.

The record names clinical activity and histology outcomes alongside the safety endpoint, yet their numerical results are not reported in the registry record. This makes outcome availability as important as outcome selection: a registered measure cannot inform readers about the observed clinical course unless results and methods are posted. The record lists no analyses, so the precision and statistical interpretation of results cannot be assessed.

Finally, the planning illustration uses a hypothetical two-arm setup, while the actual design is single-group. This is a useful reminder that sample-size illustrations depend on their assumptions and should not be mistaken for the design that was conducted or for observed power. The registry does not report the trial's own sample-size justification. Together, these features make the study a clear case for matching statistical claims to the design, endpoint, and results actually available.

15. 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 enrolled group comprised 6 participants, and the study had no comparator arm. Consequently, the posted data are descriptive and cannot estimate a treatment difference or distinguish treatment-related events from events that could have occurred for other reasons. The absence of masking may also affect reporting or assessment, but further details about assessment procedures are not reported in the registry record.

The primary endpoint was defined over baseline to Week 10, but the record does not report how frequently events were assessed, how repeated events were counted, or how event attribution was determined. It does not clarify whether participants could appear in both serious and other adverse-event categories. The reported counts therefore do not support a unique total across categories.

Although secondary outcomes are named, their numerical results are not reported in the registry record. The registry lists 8 outcome measures and 0 analyses, but does not describe outcome-specific analysis methods, a multiplicity approach, or missing-data handling. It also does not report confidence intervals, hypothesis tests, interim analyses, or the study's sample-size justification. The planning illustration is not the trial's own design and should not be treated as evidence of observed power.

The dates and completed status describe the study record but do not resolve these gaps. Without detailed results and analysis methods, readers cannot assess the precision of the safety counts, the clinical outcomes, or the effects of incomplete follow-up. These limitations constrain interpretation to the descriptive information actually posted.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Oral OKT3 for the Treatment of Active Ulcerative Colitis — NCT01287195.
• Registry record last updated 2019-02-11; status last verified 2019-01.
• Results first posted 2017-06-14.
• 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.

19. Record Summary

NCT01287195 is a completed Phase 1/Phase 2, single-group study titled Oral OKT3 for the Treatment of Active Ulcerative Colitis. Brigham and Women's Hospital is the sponsor. The registry records enrollment of 6 participants, no masking, and allocation as not applicable. The study began on 2011-04-07 and reached primary completion on 2013-05-02.

Participants with ulcerative colitis were to receive Oral OKT3 with omeprazole once daily for 30 days. The primary endpoint was the number of participants with adverse events from baseline to Week 10. The Oral OKT3 group records 2 participants affected by serious adverse events, 1 affected by other adverse events, and 0 deaths; ulcerative colitis and anorectal abscess are listed among top serious-event terms, each at 16.7%.

Mayo Score, SCCAI Score, and a histologic evaluation score are listed as secondary outcomes. Their numerical results, analysis methods, missing-data handling, and the trial's sample-size justification are not reported in the registry record. The single-group design supports descriptive safety reporting, not a randomized comparison or a causal treatment estimate.