Spesolimab in Pyoderma Gangrenosum: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Spesolimab in Pyoderma Gangrenosum (NCT06092216), a na phase 2 study. The primary outcome is Change in Global Pyoderma Gangrenosum (GPG) Severity Score.

2. Clinical Question

The study’s stated purpose was to assess the feasibility of using spesolimab in this population and to investigate the body’s immune response to it. The registry record names lesion improvement and patient-reported measures among the secondary outcomes, including pain severity and dermatology-related quality of life. It also lists lesion recurrence after complete re-epithelialization and treatment cessation as an outcome. These are outcome topics, not evidence that a particular result occurred.

The registry status is terminated. That status does not, by itself, explain why the study ended or establish whether the intended assessments were completed. A reason for termination is not reported in the registry record. Most importantly for the primary clinical question, a numerical change in the severity score and its precision are not reported in the registry record. The stated research question can be identified, but the posted record does not establish the size or direction of the score change.

3. Trial Design

This is a phase 2, single-group study of spesolimab for pyoderma gangrenosum. The registry lists enrollment as 5 and identifies the study status as terminated. The listed study start is 2023-09-23, and the primary completion date is 2025-01-14. These dates describe the registry record; they do not explain the study’s duration or the reason it was terminated.

The study has no listed allocation to separate groups and no listed masking. Allocation is recorded as not applicable, and the design is identified as single group. Thus, the record describes participants receiving spesolimab, not a randomized comparison with a placebo or another treatment. The intervention description specifies 900 mg of spesolimab intravenously. A dosing schedule is not reported in the registry record.

The primary outcome is change in Global Pyoderma Gangrenosum Severity Score in a target lesion from baseline to Week 16. The analysis population is described as including all participants who did not withdraw. The registry record does not state how many participants met that description, whether all planned assessments were completed, or how missing Week 16 observations were handled.

The study also lists outcomes concerning complete re-epithelialization, pain severity, dermatology-related quality of life, recurrence after treatment cessation, and a low GPG severity score. The registry lists 6 outcome measures in total and reports no analyses. The available record does not supply numerical results for the primary endpoint or the listed secondary outcomes.

Because there is no concurrent comparator group, changes from baseline can describe the participants’ observed course but cannot isolate a treatment effect from other explanations for change over time. The registry record does not report the protocol rationale, sample-size justification, interim analyses, or a detailed statistical plan. The termination reason is also 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Includes all participants who did not withdraw

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.

6. Primary Endpoint

The registry lists the primary outcome as Change in Global Pyoderma Gangrenosum (GPG) Severity Score, measured over Baseline and Week 16.

The primary endpoint is change in the Global Pyoderma Gangrenosum Severity Score for a target lesion between baseline and Week 16. The registry describes the score using categories from 0, completely clear, through 4, severe. The categories reflect lesion features such as ulceration, granulation tissue, re-epithelialization, borders, drainage, and necrotic tissue. A lower score corresponds to a category with less severe lesion features in the scale description.

The endpoint is intended to capture change in lesion severity over time using a defined clinical scoring framework. A score provides a structured way to describe lesion status; summarizing change can make the direction and average magnitude of observed score movement easier to communicate. The registry specifies the mean as the summary parameter and standard deviation as the dispersion measure. In general, a mean describes the average of participant-level values, while a standard deviation describes their spread around that mean.

These summaries do not replace the underlying clinical descriptions of the score categories. As a general statistical consideration, score categories are ordered, but the distance between adjacent categories need not represent equal clinical change. The registry record does not explain how this issue was addressed in analysis.

The endpoint definition alone does not show the observed change. The numerical mean change, its standard deviation, the number contributing data, and any confidence interval are not reported in the registry record. The record therefore identifies what was to be measured and when, but not the size or precision of the observed primary result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The design is identified as single group, with allocation recorded as not applicable and masking listed as none. In general, an uncontrolled study can describe what happened to participants over time, but it cannot use a concurrent randomized group to separate treatment-associated change from other possible causes. This is a design limitation, not a result about the participants.

What does a mean change represent?

The primary outcome is a mean change in GPG severity score between baseline and Week 16. In general, a mean change summarizes participant-level changes with one average. It can communicate whether the group’s average score moved upward or downward, provided the result and its direction are reported. The registry record does not report the numerical mean change, so its direction and size are unknown from the posted results.

The listed dispersion measure is the standard deviation. In general, a standard deviation describes how spread out individual observations are around their mean. It is not the same as a confidence interval: the standard deviation describes variability among observed values, whereas a confidence interval is an inferential range used to express uncertainty about an estimated quantity. A confidence interval for the primary endpoint is not reported in the registry record.

The GPG scale is described through ordered categories from 0 to 4, with clinical features distinguishing levels of lesion severity. A mean can be a compact summary of a scored outcome, but, as a general consideration, the scale’s ordered categories do not necessarily have equal distances between them. A mean alone may therefore conceal which category transitions occurred or whether participant-level changes varied. The registry record does not report participant-level transitions or an analysis addressing the scale’s categorical structure.

What can a single-group comparison establish?

A comparison of baseline with Week 16 is a within-group description. In general, such a comparison asks how measurements changed in the same study group over time. It does not estimate the difference between the treatment and a concurrent control. Without a comparator, changes can also reflect the natural course of illness, measurement variation, or other influences. This means that even a numerical change would need to be interpreted as observed change in this group, rather than as a controlled estimate of treatment effect.

The registry identifies the primary population as all participants who did not withdraw. The record does not report the number included in the endpoint analysis, the number with measurements at both time points, or whether participants who withdrew contributed any data. It also does not state how missing values were handled. These are important details because a mean calculated only from participants with available follow-up can describe a subset rather than everyone enrolled.

What does the record say about planning?

The listed planning assumptions describe an illustration using 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 the observed effect. The record characterizes this as a planning illustration, not the trial’s own design. Since the actual study is single group, an equal-allocation two-arm illustration is not a description of its treatment assignment. The registry record does not report a sample-size justification for the study itself.

In general, a planning calculation asks what sample size might be needed under chosen assumptions about an effect, variability, significance level, power, and allocation. It is not evidence that a study achieved a particular level of power, and it does not turn an observed result into a controlled treatment comparison. The registry record does not report the primary observed effect needed to interpret a numerical illustration. No numerical planning result or recalculated primary estimate is stated.

More broadly, a confidence interval can help show how precisely an estimate is known: a narrower interval generally indicates greater precision under the analysis assumptions, while a wider interval indicates more uncertainty. A small study may produce imprecise estimates, but the actual precision cannot be assessed without the relevant estimate and uncertainty measure. No confidence interval or standard error for the primary result is reported in the registry record.

Why does over-dispersion matter?

Over-dispersion is a general concern when observed variability exceeds what a statistical model assumes. If a model understates variability, its uncertainty estimates may be too small and inferential conclusions too confident. The registry record does not report a model, a dispersion assessment, or an analysis of over-dispersion for this endpoint. The primary measure is specified as a mean with standard deviation, and a more detailed statistical method is not reported.

The record lists 6 outcome measures and reports no analyses. It does not report an inferential procedure, adjustment for multiple outcomes, handling of repeated measurements, or a prespecified hierarchy among secondary outcomes. These missing details limit the ability to evaluate how any possible findings were selected or how uncertainty was quantified. Because numerical results are not reported for the primary endpoint, statistical significance, precision, and consistency cannot be assessed from the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Spesolimab")
n   <- c(3)
m   <- c(-3)
sp  <- c(2)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Spesolimab  3  -3.000  2.000  -7.968  1.968

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 6 outcome measures, including the primary change in GPG severity score and secondary outcomes concerning lesion re-epithelialization, pain, dermatology-related quality of life, recurrence, and a low GPG severity score. It reports no analyses. The registry record does not describe a multiplicity strategy, such as a prespecified order for testing outcomes or an adjustment to account for multiple statistical tests.

As a general statistical principle, examining multiple outcomes can increase the chance of observing an apparently notable result by chance if each is tested separately. A prespecified analysis plan can clarify which outcome is primary and how additional outcomes are to be interpreted. The record identifies a primary outcome, but it does not report how multiplicity was addressed or whether any inferential tests were planned. No numerical outcome results are reported, so the practical effect of any multiplicity approach cannot be assessed.

Missing-data handling is also not reported in the registry record. The primary population is described as including all participants who did not withdraw, but the record does not state how withdrawals were defined for analysis, whether Week 16 measurements were available for each participant, or what method would be used for missing observations. The number contributing to the primary analysis is not reported. These omissions matter because the estimate may depend on which participants have usable follow-up data and how missingness is handled.

The record does not describe whether analyses followed an intention-to-treat approach, a per-protocol approach, or another analysis population definition beyond the statement about participants who did not withdraw. It also does not report sensitivity analyses for missing data. Without these details, it is not possible to evaluate whether the primary analysis population and assumptions were robust to incomplete follow-up.

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 non-serious adverse event terms

The registry reports safety figures for the spesolimab group. Serious adverse events affected 0 participants among 3 at risk. Other adverse events affected 2 participants among 3 at risk. The recorded death count is 0. These are counts, not estimates of risk for a broader population, and the record does not report further event-level detail for the other adverse events.

The serious-event and other-event figures have separate at-risk counts. They should be read as the registry’s reported summaries rather than combined into a new total. The record does not list serious adverse-event terms. It also does not describe the nature, timing, duration, severity, or treatment relationship of the other adverse events. The counts alone cannot resolve those questions.

With a small listed at-risk group, each participant’s experience can have substantial influence on a descriptive count. In general, absence of a reported serious event in a small group does not establish that serious events cannot occur. Nor do the other-event counts, without event descriptions or a comparison group, establish whether those events were caused by spesolimab.

These safety figures should be interpreted separately from the primary severity-score outcome. The registry record does not report the numerical primary outcome result, and the safety counts do not provide evidence about lesion-score change. A more complete safety interpretation would require information that is not reported in the registry record.

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

The record establishes that a phase 2, single-group study of spesolimab was registered for participants with pyoderma gangrenosum. It identifies an intravenous intervention described as 900 mg and a primary endpoint based on change in target-lesion GPG severity score from baseline to Week 16. It also records enrollment as 5, identifies the study status as terminated, and reports limited adverse-event counts.

It does not establish the size or direction of the primary score change. The mean change and standard deviation are not reported in the registry record, nor is the number of participants contributing to the primary analysis. A confidence interval or other estimate of precision is not reported. The endpoint definition is not itself a result; it explains what the study intended to measure.

Even if a baseline-to-Week 16 change were available, the single-group design would describe change in the study group rather than provide a randomized comparison. In general, without a concurrent control, it is not possible to distinguish treatment-associated change from other explanations for change over time. The record does not support a comparative conclusion about spesolimab versus another treatment or no treatment.

The listed safety counts are similarly limited. They describe the reported serious and other adverse-event counts in the at-risk group, but do not provide detailed event descriptions or a comparison group. The registry record does not state why the study was terminated, whether all planned assessments were completed, or how missing outcome data were handled. Those uncertainties should not be filled in by inference.

Accordingly, the posted information defines the research question and provides a small amount of descriptive safety information, but it does not provide a numerical efficacy result or enough detail to assess its precision and completeness. It cannot establish a treatment benefit, lack of benefit, or comparative safety conclusion.

14. Why This Trial Matters Statistically

This study is statistically notable because it asks about change in a scored lesion-severity measure in a single group. Such designs can be useful for describing feasibility and the course of measured outcomes over time. They also make the distinction between descriptive change and comparative treatment effect especially important. Without a control group, statistical summaries cannot remove the alternative explanations that may influence before-and-after change.

The primary score has ordered categories tied to lesion features, while the registered summary is a mean with standard deviation. This raises a general interpretive point: a numerical average can compress a range of participant-level experiences and may not communicate which transitions between clinical categories occurred. The registry record does not report participant-level changes or the details of how the score was analyzed.

Enrollment is listed as 5, but the primary analysis population size is not reported. In a small study, incomplete follow-up or variation between participants can be particularly consequential for the estimated mean and its precision. The record does not provide the primary outcome estimate, uncertainty interval, or analysis sample size, so the degree of uncertainty cannot be quantified from the posted results.

The study’s terminated status also underscores the importance of transparent reporting. The status is known, but the reason for termination and the completeness of outcome collection are not reported in the registry record. These registry entries are necessary for understanding how closely the posted information reflects the planned study.

Finally, the record lists several outcome measures but no analyses. Without numerical findings or a reported multiplicity approach, readers cannot assess outcome patterns or distinguish primary from exploratory statistical evidence beyond the registered endpoint designation. The statistical value of the record lies in defining the question and design; it does not provide the results needed to estimate the observed primary change.

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 primary limitation for interpreting this trial record is the absence of a numerical result for the registered primary endpoint. The mean change and standard deviation are identified as intended summaries, but neither is reported. The record also does not report a confidence interval, the number included in the primary analysis, or participant-level outcome information. The observed magnitude, direction, variability, and precision of change therefore cannot be assessed.

The single-group, unmasked design has no concurrent comparator. As a general design limitation, an observed change over time cannot by itself distinguish treatment-associated change from the natural course of the condition, measurement variation, or other influences. The registry record does not report details that would address these alternative explanations.

Enrollment is listed as 5, while the primary population is described as participants who did not withdraw. The number who did not withdraw and the number with complete baseline and Week 16 assessments are not reported. The record does not describe missing-data methods, sensitivity analyses, or the analysis population beyond that population statement.

The status is terminated, but the reason for termination is not reported in the registry record. The record does not clarify whether all planned outcome assessments were completed. It also does not report a detailed statistical analysis plan, a sample-size justification for the actual single-group design, or how multiple outcomes were handled.

Safety information is limited to counts, with no serious adverse-event terms and no description of the other adverse events. The registry record does not provide their timing, severity, duration, or relationship to treatment. These limitations leave substantial uncertainty about both the primary outcome and the context needed to interpret the safety summaries.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Spesolimab in Pyoderma Gangrenosum — NCT06092216.
• Registry record last updated 2026-03-31; status last verified 2026-03.
• Results first posted 2026-03-31.
• 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

NCT06092216 is a phase 2, single-group study of spesolimab for pyoderma gangrenosum. The intervention is described as 900 mg intravenously. Enrollment is listed as 5, the study status is terminated, and the listed primary completion date is 2025-01-14. The primary endpoint is mean change in target-lesion GPG severity score from baseline to Week 16, with standard deviation as the dispersion measure.

The registry record does not report the numerical primary outcome, the number included in its analysis, or its precision. It reports 0 serious adverse events among 3 at risk, 2 participants with other adverse events among 3 at risk, and 0 deaths. The reason for termination, missing-data handling, and detailed statistical plan are not reported in the registry record. Because there is no comparator group, the design is descriptive rather than a randomized treatment comparison.