Study To Evaluate The Efficacy Of Tofacitinib In Patients With SJS/TEN: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study To Evaluate The Efficacy Of Tofacitinib In Patients With SJS/TEN (NCT06474078), a na na study. The primary outcome is Complete Skin Healing Time, Day, Medium [Full Range].

2. Clinical Question

The study is titled as an evaluation of tofacitinib in SJS/TEN and is described as an open-label pilot assessing safety, tolerability, and efficacy. Its primary outcome focuses on the time taken for skin to heal. Secondary outcomes described in the study summary include mortality, length of hospitalization, adverse events, time to the beginning of epithelization, time to halting of disease progression, ocular complications, and infections. The registered secondary outcome list specifically names length of hospitalization and mortality.

Because there is no control group, the central question is descriptive: what outcomes were recorded for the treated group? A single-group result cannot isolate the effect of tofacitinib from other factors that may influence recovery. The registry record does not report numerical results for the primary or secondary outcomes, so the clinical question cannot be answered from the posted outcome information.

3. Trial Design

This is a completed, open-label pilot study sponsored by Chang Gung Memorial Hospital. The registry lists the study as a single-group design, with no masking and allocation recorded as not applicable. The registered intervention is tofacitinib. Enrollment is listed as 20. The registry records the study start as 2022-08-01 and primary completion as 2025-06-20.

The single-group structure means that participants received the study intervention without a separately registered control arm. Open-label means the study is not recorded as masked. Consequently, the record does not support a randomized treatment comparison. Outcomes can describe what was observed in the study group, but cannot by themselves establish how those outcomes would compare with an alternative treatment or with no study intervention.

The primary endpoint is time to complete skin healing. Healing is defined as complete re-epithelialization, meaning the complete absence of erosions. The study summary also identifies mortality, hospitalization duration, adverse events, the beginning of epithelization, cessation of SJS/TEN progression, ocular complications, and infections as outcomes of interest. The registered secondary outcome list names length of hospitalization and mortality. Four outcome measures are recorded, but the registry reports zero analyses.

The record does not report a detailed statistical analysis plan, a sample-size justification, rules for handling missing outcome data, or a definition of the analysis population. It also does not report whether any interim analyses were conducted. The status is completed, but that status alone does not establish that every planned outcome was measured or that results were posted. Numerical outcome results 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: SJS/TEN (n=20)

Overall Study

Analysis population for the primary outcome, as defined in the registry: We determined if the enrolled participants had SJS/TEN using the criteria and histopathological examinations.

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: We totally recruited 20 SJS/TEN participants to analyze.

6. Primary Endpoint

The registry lists the primary outcome as Complete Skin Healing Time, Day, Medium [Full Range], measured over days.

The primary endpoint is the time taken for the skin to heal, measured in days. The registry defines healing as complete re-epithelialization, specifically the complete absence of erosions. This endpoint is intended to capture the duration of the skin-healing process rather than only whether healing occurred.

Time-to-event outcomes are useful when the timing of an event matters and participants may reach that event at different times. In general, the event is the prespecified outcome—in this case, complete healing. An analysis of time to event can make use of both event timing and the period during which participants were observed. How participants who do not reach the event during observation are handled depends on the analysis approach and censoring rules.

The registry identifies the primary outcome summary as a median with a full range. In general, the median describes the middle of the observed time distribution, while the full range describes its lowest and highest observed values. These summaries can be informative when outcome times are unevenly distributed or include unusually long observations. They do not, on their own, describe uncertainty as a confidence interval would.

The record does not report the median, the full range, a confidence interval, or other numerical results for this endpoint. It therefore does not show the observed healing-time distribution or its precision.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registered primary outcome is a time-to-event measure: time from the relevant study starting point to complete skin healing. The event is defined as complete re-epithelialization, or complete absence of erosions. The record identifies the summary as a median with a full range. It does not report an inferential analysis for this endpoint, an estimate of uncertainty, or the individual event times.

What does the median describe?

In general, the median is the middle value after observations are ordered by their measured outcome. For healing time, it gives a central description of the observed times, rather than an average that can be strongly affected by unusually long or short observations. The full range gives the span between the smallest and largest recorded values. Neither summary alone explains how precise the estimated median is. A confidence interval, when calculated and reported, expresses uncertainty around an estimate under the assumptions of the method used. No confidence interval for the primary endpoint is reported in the registry record.

Why is healing treated as a time-to-event outcome?

In general, time-to-event methods account for both whether an event occurred and when it occurred. They can also accommodate censoring: a participant may leave observation or reach the end of follow-up before the event is recorded. Such a participant's event time is not known in full, but their observed follow-up can still contribute information under suitable methods. The treatment of censoring and the follow-up rules are important because they affect interpretation. These rules are not reported in the registry record, so the handling of participants without a recorded healing event cannot be assessed.

A median and full range may be descriptive summaries of observed event times, but a time-to-event analysis can use other methods to account for censored observations. The registry does not report whether a survival curve, a hazard ratio, or another time-to-event estimator was used for the primary endpoint. The record's general note says that only sponsor-reported hazard ratios are used for time-to-event calculations, but it reports no hazard ratio for this study. No hazard-ratio estimate should therefore be inferred from the endpoint description.

What can a hazard ratio communicate?

In general, a hazard ratio compares the event rates over time between groups, subject to the model and its assumptions. It is not a direct comparison of median times and is not a risk ratio. In a single-group study, a hazard ratio would require a defined comparator or reference framework to provide a comparative interpretation. No such treatment-control comparison is registered here, and no sponsor-reported hazard ratio is listed. A comparative effect estimate cannot be recovered from the design description alone.

What do the design and analysis fields imply?

The study is recorded as open-label and single-group, with allocation not applicable and no masking. These features describe the registered design; they do not specify the statistical methods used. A single-group study can report descriptive outcomes, but it does not provide a randomized control group for estimating a between-group effect. The registry records four outcome measures and zero analyses. It does not report a statistical analysis plan, adjustment variables, missing-data methods, or an analysis population definition.

In general, an analysis plan clarifies how outcomes are summarized, how uncertainty is quantified, and how deviations from complete follow-up are handled. Without those details, even posted descriptive values would need cautious interpretation. For this record, the absence of numerical outcome results means that neither an estimate nor its precision can be evaluated.

How should planning calculations be read?

The record describes planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It states that the effect to detect is a stated fraction of the observed effect. For time-to-event illustrations, the stated quantity is the total number of events using a Schoenfeld approach; other illustrations use participants per arm. These are planning assumptions, not descriptions of the registered single-group design. In particular, equal allocation between two arms does not match the recorded single-group study.

In general, sample-size calculations estimate how much information a future study might need under chosen assumptions. They are not a measure of the probability that a completed study's finding is true, and they are not observed power. The record does not report planning results or an observed effect estimate on which to base a numerical illustration. No sample-size figure or power conclusion can be drawn from the available registry information.

What does over-dispersion mean in general?

For count outcomes, over-dispersion means that observed variability exceeds what a simple model assumes. In general, ignoring it can make uncertainty appear smaller than it is. That consideration may matter for some count-data analyses, but the registry record does not report a count-model analysis or a method addressing over-dispersion. It would be inappropriate to assume that such an analysis was used.

10. Multiplicity, Missing Data and Analysis Populations

The registry describes multiple outcomes. The study summary names time to complete re-epithelialization as primary and lists mortality, length of hospitalization, adverse events, time to beginning of epithelization, time to halting of SJS/TEN progression, ocular complications, and infections as secondary outcomes. The registered secondary outcome list names length of hospitalization and mortality. Four outcome measures are recorded. The record does not explain how these outcome lists relate to the four measures or provide the detailed outcome analysis plan.

When a study evaluates multiple outcomes, multiplicity can arise because many statistical tests create more opportunities for chance findings. In general, an analysis plan may specify which outcome is primary, whether secondary analyses are confirmatory or descriptive, and whether any adjustment for multiple testing is used. No multiplicity strategy is reported in the registry record. Since no analyses or numerical outcome results are posted, it is not possible to determine whether testing was conducted or how multiplicity was handled.

Missing-data methods are also not reported. The registry does not describe the amount or pattern of missing outcome data, how participants without a recorded healing event were handled, or whether imputation or another approach was used. These details matter because incomplete follow-up can affect estimates, particularly for time-to-event outcomes. No assumption about complete follow-up or the treatment of missing observations is justified by the record.

The definition of the analysis population is not reported. Although enrollment is listed as 20, the record does not state how many participants contributed to each outcome analysis or whether the analysis included all enrolled participants. The registered analysis count is zero. A completed study status does not resolve these reporting gaps.

11. 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 records zero serious adverse events among 20 at risk in the tofacitinib treatment group. It also records three other adverse events among 20 at risk and zero deaths. The serious adverse event term list is empty, so no serious-event types are listed in the registry record.

These counts should be read as the adverse-event information recorded for this group, not as comparisons between treatments. There is no separate control group in the registered design, and the record does not provide comparative rates or estimates. The counts alone do not establish whether an event was caused by tofacitinib, how events were defined or collected, or how long participants were observed for safety. Descriptions of the three other adverse events are not reported in the registry record.

In general, adverse-event counts depend on the definitions, observation period, and completeness of reporting. Zero recorded serious events does not demonstrate that serious events cannot occur; it reports only that none are listed for this study group in the registry. The safety data are limited to the posted counts and should not be interpreted as a controlled safety comparison.

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

The record establishes that a completed, open-label, single-group pilot study of tofacitinib in SJS/TEN was registered, with enrollment listed as 20. It identifies time to complete skin healing as the primary outcome and defines healing as complete re-epithelialization, or the absence of erosions. It also records limited adverse-event counts for the treatment group.

It does not report a numerical primary outcome result. There is no posted median healing time, full range, confidence interval, or hazard ratio. The record also lists zero analyses and does not provide numerical results for the other named outcomes. As a result, the registry record cannot show the observed healing-time distribution, the precision of an estimate, or the findings for hospitalization, mortality, and other outcomes.

The design also limits what could be concluded from any single-group outcome. Without a control group, there is no registered randomized comparison that could separate treatment-associated outcomes from other influences. General improvement or recovery in a treated group, if reported, would not by itself establish that the intervention caused that outcome. No claim that tofacitinib is effective or ineffective follows from the information in this record.

The adverse-event counts describe what is listed for the study group but do not estimate a comparative safety effect. Event details, comparative data, and safety follow-up duration are not reported in the registry record. These gaps mean the counts cannot support a broader conclusion about safety.

The distinction is between a registered research question and a reported result. The record describes what the investigators set out to evaluate and gives some design and safety information. It does not provide the outcome estimates required to judge the observed clinical results or their uncertainty.

13. Why This Trial Matters Statistically

This study raises statistical questions common to small, early-stage clinical research. Its primary outcome is a time-to-event measure, for which timing, censoring, and follow-up matter. A clearly defined event—complete re-epithelialization—helps make the outcome interpretable, but a result still needs an estimate and information about its uncertainty and analysis population.

The single-group design is central to interpretation. It can describe outcomes among participants enrolled in the study, but it does not supply a concurrent randomized comparison. That distinction is important when readers consider whether outcomes can be attributed to the intervention. The open-label design and lack of masking are also part of the registered study context, though the record does not describe their effects on outcome assessment.

The registry identifies a primary outcome and several other outcomes of interest, but does not report an analysis plan or multiplicity strategy. These omissions make it difficult to understand how results would have been prioritized and interpreted if numerical results were posted. Likewise, missing-data handling is not described, which is particularly relevant for a time-to-event endpoint when follow-up may not capture the event for every participant.

Finally, the record illustrates why registration, study completion, and results reporting are distinct. Registration identifies the intended question; completion records a status; neither supplies an outcome estimate. Here, the design and primary endpoint are described, but numerical efficacy results are not reported. Statistical interpretation must therefore remain limited to the study structure and the counts that are posted.

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 for interpreting outcomes is that numerical results for the primary endpoint are not reported in the registry record. The record gives the endpoint definition and intended summary, but not the observed median or full range. Numerical results for the other listed outcomes are also not reported, and the registered analysis count is zero.

The single-group design has no registered control arm. This prevents a randomized comparison and limits causal interpretation. The study is open-label, and the record does not describe masking of outcome assessment. No information about co-interventions or other factors that might influence healing is reported in the registry record.

Important analysis details are absent. The record does not report an analysis population, missing-data approach, censoring rules, follow-up distribution, or statistical analysis plan. It also does not report whether interim analyses occurred. These details are needed to assess how well outcome estimates represent the enrolled group and how precisely they were estimated.

The safety record is limited to event counts. Descriptions of the three other adverse events, safety observation duration, and comparative safety estimates are not reported. The empty serious-event term list does not provide further detail about event ascertainment.

Enrollment is listed as 20, but the record does not report how many participants contributed to each outcome. The completed status and the presence of a registered endpoint do not establish that all outcome data are available. Together, these limitations mean the posted record is insufficient to characterize efficacy outcomes or make comparative conclusions.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study To Evaluate The Efficacy Of Tofacitinib In Patients With SJS/TEN — NCT06474078.
• Registry record last updated 2025-09-16; status last verified 2025-08.
• Results first posted 2025-09-16.
• 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.

18. Record Summary

The registry describes NCT06474078, an open-label pilot study of tofacitinib in patients with Stevens-Johnson Syndrome or Toxic Epidermal Necrolysis. Chang Gung Memorial Hospital is listed as sponsor. The study is recorded as completed, with enrollment of 20, a start date of 2022-08-01, and primary completion on 2025-06-20.

The registered design is single-group, with no masking and allocation recorded as not applicable. The primary outcome is time to complete skin healing, defined as complete re-epithelialization and the complete absence of erosions. The record lists four outcome measures and zero analyses. It does not report a numerical primary outcome result.

The safety information records zero serious adverse events, three other adverse events, and zero deaths in the tofacitinib group. No serious adverse event terms are listed. These counts are not comparative results.