A Study of the Safety of INCB054707 in Participants With Hidradenitis Suppurativa: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of the Safety of INCB054707 in Participants With Hidradenitis Suppurativa (NCT03569371), a na phase 2 study. The primary outcome is Number of Participants With Treatment-emergent Adverse Events (TEAEs).

2. Clinical Question

The study’s stated purpose was to assess safety in participants with moderate to severe HS. Its primary outcome is a participant count, rather than a comparison of event rates between treatments. The registry defines a treatment-emergent adverse event as an adverse event reported for the first time, or a worsening of a pre-existing event, after the first dose of study drug.

This question is descriptive: it asks what adverse events were recorded among participants receiving the study drug. With no control group, the design cannot directly distinguish events that might occur without treatment from events associated with treatment. The registry also lists secondary outcomes concerning drug disposition, HS clinical response, lesion counts, pain scores and disease severity. Numerical results for those outcomes are not reported in the registry record.

3. Trial Design

This is a phase 2, open-label, single-arm study of INCB054707 in participants with HS. The registry describes an enrollment of 10 and lists the study as completed. The study began on 2018-07-17, and primary completion was 2019-04-22. The primary outcome timeframe is up to approximately 12 weeks.

In a single-arm design, participants receive the study intervention without a concurrent control group. Open-label means the study is not masked. The registry lists allocation as not applicable and masking as none. These features make the study suitable for describing recorded outcomes in the treated group, but they do not provide a randomized treatment contrast. Differences from an external population or from an assumed background rate would not be controlled by the design.

The primary outcome population is defined as all enrolled subjects who received at least 1 dose of INCB054707. The registry’s adverse-event information lists 10 participants at risk for the serious-adverse-event summary and 10 at risk for the other-adverse-event summary. The relationship between those summaries and the primary analysis population is not described beyond the information in the record.

The registry lists secondary outcomes that include apparent oral clearance, apparent oral volume of distribution, absorption and inter-compartmental clearance, as well as HS clinical response, lesion-count status, pain-score change and modified Sartorius Scale change. These outcomes suggest that the study record covers both safety and other clinical or pharmacokinetic measures. The detailed assessment schedule, treatment regimen, study procedures and statistical analysis plan 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.

Before assignment: A total of 10 participants were assigned to treatment with INCB054707.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The Full Analysis Set population include all subjects enrolled in the study who received at least 1 dose of INCB054707

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 Number of Participants With Treatment-emergent Adverse Events (TEAEs), measured over Up to approximately 12 weeks..

The primary endpoint is the number of participants with treatment-emergent adverse events (TEAEs), assessed over a timeframe of up to approximately 12 weeks. The registry defines a TEAE as an adverse event first reported after the first dose of study drug, or a worsening of an event that was already present before treatment. The endpoint therefore classifies events according to their timing relative to treatment initiation.

Counting participants with an event answers how many people experienced at least one event under the endpoint definition. It is not the same as counting every event: one participant may have more than one event, but a participant-level count does not express that event burden. Nor does the count alone establish that treatment caused an event. In a single-arm study, the observed count describes events recorded among those receiving the intervention, without a concurrent group to provide a direct comparison.

Adverse-event endpoints are used in safety assessment because they capture unfavorable events observed during treatment and follow-up, including events that may not be anticipated in advance. Interpretation depends on the event definition, the observation period, the population included and how completely events were collected. The registry record names this primary endpoint, but its numerical result is not reported in the registry record. The separate serious and other adverse-event participant figures should not be substituted for a TEAE result unless the record explicitly equates those categories, which it does not.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods should be interpreted in light of the design and the type of outcome. This study is single-arm, so its core safety information is descriptive: it records the number of participants affected in the treated group. A descriptive count answers how many participants met a stated event definition in the observed group. A comparative treatment effect would require a suitable comparator and a defined analysis, neither of which is reported for this study.

What does a participant count represent?

The primary outcome is a count of participants with TEAEs, not a count of all adverse-event episodes. If a participant experiences multiple events, a participant-level endpoint still counts that person as a participant with an event, rather than representing the number or duration of every episode. This distinction matters because participant frequency and total event burden are different summaries. The endpoint definition also sets a timing rule: the event must first be reported, or worsen, after the first dose.

For an event count to be interpreted as a frequency, its denominator and observation period matter. The registry states the primary outcome timeframe as up to approximately 12 weeks. Follow-up can differ between participants in practice, but whether it did so in this study is not reported in the registry record. The record defines the Full Analysis Set as enrolled subjects who received at least 1 dose. The number included in that population is not separately reported in the registry record.

Why does the lack of a comparator matter?

In general, a concurrent control group helps distinguish outcomes observed during treatment from outcomes that may also occur in the absence of that treatment. A single-arm study does not make that contrast. Observed adverse events can be described, but attributing them to the intervention requires more than temporal ordering. The registry definition establishes that TEAEs occur after first dose or worsen after first dose; it does not establish causation.

Similarly, a p-value or confidence interval would address a particular statistical question only if an analysis and its assumptions were specified. The registry lists 0 analyses. It does not report an inferential model, a comparison hypothesis, a confidence interval for the primary outcome, or an adjusted estimate. General explanation: a confidence interval represents uncertainty around an estimate under the assumptions of the method used. It is not a guarantee about an individual outcome, and it cannot compensate for a lack of a comparison group.

How should the planning illustrations be understood?

Any sample-size calculations associated with this record are planning illustrations, not results from the trial’s design process. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For time-to-event calculations, the illustration concerns the total number of events; for other outcomes, it concerns participants per arm. These assumptions describe a hypothetical planning framework. They do not change the actual study into a two-arm trial, and they are not observed power.

In general, over-dispersion means that outcome variation exceeds what a simpler statistical model assumes. It can affect uncertainty estimates and planning calculations when the model does not adequately represent the data. The registry record does not report whether over-dispersion was assessed or whether any such model was used. Likewise, the specific model, estimation method and uncertainty calculations for this trial are not reported in the registry record.

What can the secondary outcomes add?

The registry lists pharmacokinetic outcomes and clinical measures, including response, lesion counts, pain and modified Sartorius Scale change. These are distinct questions and may have different outcome types and analysis needs. Their presence in the outcome list does not establish that a particular statistical model was used or that a result was statistically significant. Numerical results and analysis details for those measures are not reported in the registry record. The record therefore supports a design-based reading and descriptive interpretation of the reported safety counts, rather than reconstruction of an unstated analysis.

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("INCB054707")
n      <- c(10)
events <- c(7)
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  INCB054707  10  7  70.0  34.8  93.3  39.7  89.2  39.0  89.1

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 16 outcome measures, including the primary safety endpoint and secondary outcomes concerning pharmacokinetics and HS measures. When multiple outcomes are assessed, multiplicity can arise because several statistical questions are considered at once. In general, testing many hypotheses without an appropriate strategy can increase the chance of at least one apparently notable result arising by chance. A prespecified hierarchy, adjustment procedure or clearly descriptive approach can help readers understand how multiple outcomes were handled.

The registry record does not report a multiplicity strategy, a hierarchy among secondary outcomes or a statistical analysis plan. It lists 0 analyses. The number of outcome measures alone does not establish that every outcome was formally tested, or that any adjustment was or was not applied; those details are not reported in the registry record.

Missing-data methods are also not reported in the registry record. The record does not describe whether outcome data were incomplete, how missing observations were handled, or whether participants with incomplete follow-up were included in particular analyses. The primary analysis population is defined as enrolled subjects who received at least 1 dose, but the number meeting that definition is not separately reported.

These omissions affect interpretation because the analyzed population, completeness of follow-up and handling of missing observations can influence descriptive counts and estimates. The adverse-event summaries include their own at-risk counts, but they do not supply a complete account of missingness or of the analysis populations for every outcome. Readers should therefore treat the population and missing-data procedures as not reported in the registry record, rather than infer them from the listed outcomes or event 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 5% reporting threshold.

Most frequent non-serious adverse event terms

The registry lists 0 participants affected by serious adverse events among 10 at risk. Its other-adverse-event summary lists 7 participants affected among 10 at risk. The record also lists 0 deaths. These figures describe participants in the categories recorded; the serious and other categories should be read separately.

The “other” summary is not defined in the registry record as the primary TEAE endpoint, so its count should not be presented as the TEAE result. The primary endpoint is the number of participants with TEAEs, but that numerical result is not reported in the registry record. Nor do the serious-adverse-event figures state the total number of event episodes or their duration.

These are observed counts, not a controlled comparison and not proof of causation. The absence of participants recorded with serious adverse events in this summary does not establish that serious events are impossible or that no risk exists. Likewise, the other-adverse-event count should not be used to infer severity or treatment attribution. Event definitions, ascertainment details and uncertainty intervals for these counts are not reported in the registry record.

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

The results describe what the registry records for participants in a completed, open-label, single-arm study of INCB054707 for HS. The available safety summary reports 0 participants affected by serious adverse events among 10 at risk, 7 participants affected in the other-adverse-event summary among 10 at risk, and 0 deaths. These counts provide a limited descriptive account of recorded events in this group.

They do not establish that the intervention caused or prevented an event. Without a concurrent comparator, the study cannot directly contrast event occurrence under treatment with what would have occurred under another treatment or without the study intervention. The event counts also do not by themselves describe all aspects of safety, such as event duration, recurrence, severity within the other category, or how events changed over time.

The named primary endpoint is the number of participants with TEAEs, but its numerical result is not reported in the registry record. The available serious and other event summaries should not be treated as substitutes for that endpoint. Likewise, the registry lists secondary clinical and pharmacokinetic outcomes, but their numerical results are not reported in the registry record. The registry entries therefore do not support conclusions about the size or precision of changes in those measures.

In general, statistical evidence is strongest when the endpoint, analysis population, follow-up, uncertainty and comparison are all clear. Here, the design and reported counts allow a description of the observed safety summaries, but not a controlled estimate of treatment effect. The record does not report confidence intervals or a detailed analysis plan. These limitations define what the findings can mean; they do not negate the value of recording outcomes in a clinical study.

14. Why This Trial Matters Statistically

This trial is statistically informative in part because it illustrates the distinction between describing outcomes and estimating comparative effects. A single-arm study can document what was recorded among participants receiving an intervention. It cannot, by design alone, show how those observations compare with outcomes under a different treatment or without treatment. That distinction is especially important when a study’s stated purpose is safety assessment.

The primary endpoint is participant-based, which focuses attention on how many people experience a treatment-emergent event rather than on the total number of event episodes. The registry’s definition also makes timing part of the endpoint: an event must be new after first dose or represent worsening of a pre-existing event. Such definitions are central to interpreting safety summaries, but they do not establish causation.

The record also demonstrates why endpoint names and reported results must be kept distinct. It names TEAEs as the primary outcome but does not report a numerical result for that outcome in the registry record. Separate serious and other adverse-event counts are available, but they should not be silently substituted for the primary endpoint. Keeping those categories separate prevents an overly broad interpretation of a count.

Finally, the record lists a range of secondary outcomes without numerical results or analysis details in the registry entries. That makes transparency about missing methods and results part of sound interpretation. Readers can identify the study design, population definition, outcome focus and reported safety counts, while recognizing that the record does not support a more detailed inferential conclusion.

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 main limitation is the single-arm, open-label design. There is no concurrent comparator, and the record lists no masking. The observed safety summaries are therefore descriptive and cannot provide a direct treatment comparison. The design also limits causal interpretation: an event occurring after treatment begins is not necessarily caused by treatment.

The registry reports an enrollment of 10, but the primary analysis population is defined by enrollment and receipt of at least 1 dose. The number in that population is not separately reported. The adverse-event summaries each list 10 participants at risk, but these figures do not resolve the population details for every endpoint.

The primary TEAE endpoint is defined, yet its numerical result is not reported in the registry record. The available serious and other adverse-event counts do not fully describe event type, timing, duration or severity, and the other-event category is not defined as equivalent to TEAEs. The record lists 0 deaths, but does not report further detail on the methods used to collect or classify events.

Numerical results for the listed secondary outcomes, confidence intervals, statistical models, multiplicity procedures and missing-data methods are not reported in the registry record. The detailed dosing schedule, study procedures and statistical analysis plan are also not reported. These gaps constrain assessment of precision, completeness and the handling of multiple outcomes. As a result, interpretation should remain close to the reported design and adverse-event counts.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study of the Safety of INCB054707 in Participants With Hidradenitis Suppurativa — NCT03569371.
• Registry record last updated 2022-09-26; status last verified 2022-09.
• Results first posted 2020-05-20.
• 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

NCT03569371 is a completed phase 2, open-label, single-arm study of INCB054707 in participants with HS. The registry lists an enrollment of 10, a study start of 2018-07-17 and primary completion on 2019-04-22. The stated purpose was to assess safety in men and women with moderate to severe HS.

The primary endpoint is the number of participants with TEAEs over up to approximately 12 weeks. Its numerical result is not reported in the registry record. The adverse-event summaries report 0 participants affected by serious adverse events among 10 at risk, 7 affected in the other-adverse-event summary among 10 at risk, and 0 deaths. The registry also lists secondary pharmacokinetic and clinical outcomes, but their numerical results and detailed analysis methods are not reported in the registry record.