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 Generate Real World Data On Tofacitinib Induction Therapy and Changes In Clinical and Patient Reported Outcomes. (NCT05069259), a registered phase not stated study. The primary outcome is Percentage of Participants Who Achieved Clinical Response at Week 8.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05069259 |
| Phase | Phase not stated |
| Condition | Ulcerative Colitis |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 18 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
Participants with moderately to severely active ulcerative colitis in Switzerland. The registry record describes a full analysis set as participants who had a baseline visit and received a study identification number; it also clarifies that the analyzed number for an outcome means those evaluable for that measure. Enrollment was 18, but the number evaluable for the primary endpoint is not reported in the registry record.
Intervention
The study concerns tofacitinib induction therapy and real-world clinical, biochemical, and patient-reported responses. The intervention listing itself names stool sample collection. The registry record does not give a dose or further treatment administration details.
Comparator
No like-for-like control comparison is described. The record does not state an allocation method, and its note cautions that the study is not a randomized comparison. The arm description refers to patients with active ulcerative colitis.
Primary question
The primary endpoint asks what percentage of participants achieved clinical response at Week 8. Response was defined by reduction in partial Mayo score from baseline or achieving clinical remission. The registry record does not report the observed percentage or its confidence interval, so the clinical response rate cannot be assessed from the registry information alone.
3. Trial Design
This is described as a low-interventional, prospective, multicenter study in Switzerland. Its stated focus is real-world clinical, biochemical, and patient-reported responses to tofacitinib induction therapy among people with moderately to severely active ulcerative colitis. The study title and summary frame the work as post-marketing evidence intended to characterize clinical outcomes, biomarker measurements, and participants’ experiences during induction treatment.
The registry lists enrollment of 18 and a status of terminated. The start date is 2022-03-28, and the primary completion date is 2024-04-30. These registry entries do not establish why the study was terminated or whether the listed dates reflect the timing of all participant follow-up. The reason for termination is not reported in the registry record.
The record lists stool sample collection as the intervention. It does not describe a randomized allocation, a control treatment, or a dose. Allocation is not stated, and the record warns that this is not a randomized comparison and that arms are not a like-for-like control comparison. The arm description is “Patients with active Ulcerative Colitis,” rather than a description of separate treatment groups.
Clinical outcomes are specified at Week 8 and Week 16, alongside patient-reported outcomes and biochemical remission. The registry lists 47 outcome measures, but reports 0 analyses. Thus, the design and endpoint definitions can be described, but treatment response estimates and comparisons cannot be reconstructed from 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
| Stage | All Participants |
|---|---|
| STARTED | 18 |
| COMPLETED | 16 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | All Participants |
|---|---|
| Withdrawal by Subject | 1 |
| Sponsor decision | 1 |
Analysis population for the primary outcome, as defined in the registry: Full analysis set (FAS) included all participants who were included in the study (i.e., performed baseline visit and received a study identification number). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure.
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 | All Participants |
|---|---|---|
| Participants analysed | 18 | |
| Age, Continuous | Years | 41 (30 to 56) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 15 |
| Race and Ethnicity Not Collected | Participants | — |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants Who Achieved Clinical Response at Week 8, measured over Week 8.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants who achieved clinical response at Week 8. It is a binary outcome: each evaluable participant is classified as having met or not met the response definition, and the percentage expresses the share meeting that definition. The registry record does not report the observed percentage, the number evaluable, or a confidence interval.
Clinical response is defined as a reduction in partial Mayo score from baseline of >=2 points or achieving clinical remission. Remission is defined as a partial Mayo score of <= 2 with no subscore >1. The partial Mayo score has three components: rectal bleeding, stool frequency, and physician global assessment of disease activity. Each subscore ranges from 0, normal, to 3, extreme severity; the summed score ranges from 0 to 9, with higher scores indicating greater disease severity.
A response endpoint is used to summarize whether participants meet a prespecified clinical threshold over the induction period. It offers a direct description of the proportion classified as responders, but it does not show the size of each participant’s score change or describe outcomes outside the response definition. Without the result and its uncertainty, the endpoint definition alone cannot establish how frequently response occurred in this study.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a clinically relevant question: the proportion of participants with moderately to severely active ulcerative colitis who achieved clinical response at Week 8 during a real-world tofacitinib induction study. It defines the response using change in partial Mayo score or achievement of remission. However, the registry record does not report the primary endpoint percentage, the number evaluable for that endpoint, or its 95% confidence interval. There is therefore no primary result to interpret as an estimate of response in this study.
Enrollment was 18, but enrollment is not interchangeable with the denominator for an outcome. The registry defines the full analysis set using baseline participation and receipt of a study identification number, and says that the analyzed number for an outcome refers to participants evaluable for that measure. The outcome-specific denominator is not reported in the registry record. Without it, the precision and completeness of the primary endpoint estimate cannot be assessed.
The registry lists 47 outcome measures and 0 analyses. It describes secondary outcomes covering clinical response and remission, IBDQ response and remission, and biochemical remission at specified follow-up weeks. Those endpoint definitions and titles indicate the planned areas of assessment, not the observed findings. Actual estimates for these outcomes are not reported in the registry record.
The study is not described as a randomized comparison, and allocation is not stated. Its record also cautions that arms are not a like-for-like control comparison. Consequently, the available details do not support a causal comparison between treatment strategies. The study’s real-world framing may be useful for describing outcomes in the enrolled population, but the missing endpoint results mean that even a descriptive response rate cannot be read from the registry record.
Confidence intervals are used generally to express uncertainty around an estimate, with wider intervals indicating less precision under the statistical method used. Although the primary endpoint specifies a 95% confidence interval, no interval estimate is reported. Neither the direction nor the magnitude of response, and neither statistical significance nor precision, can be inferred from an endpoint definition, study status, or enrollment figure alone.
The serious adverse event information is separate from the efficacy endpoint. The record lists a serious adverse event count and a leading serious event term, but does not provide enough detail to connect those events to the primary response result or to establish causation. The results should therefore be read as incomplete registry reporting rather than as evidence for or against a treatment effect.
9. Statistical Methods Explained
A percentage endpoint begins with a binary classification. For this study, the classification is whether an evaluable participant met the Week 8 clinical response definition. The percentage is calculated from the number meeting the definition relative to the number evaluable for that outcome. The registry describes this endpoint but does not report either count or the resulting percentage, so no response estimate can be verified or interpreted from the registry record.
What does the endpoint definition capture?
The partial Mayo score combines rectal bleeding, stool frequency, and physician global assessment. The response rule allows either a reduction from baseline of >=2 points or clinical remission, defined as a score of <= 2 with no subscore >1. These are threshold-based criteria: participants on different sides of a threshold can be classified differently even if their underlying clinical changes are similar. The endpoint condenses several clinical features into a responder status; it does not preserve every detail of the score trajectory.
The scale runs from 0 to 9, and a higher score indicates greater disease severity. The registry also gives the range for each subscore. Those details help explain what the measure represents, but they do not reveal the baseline score distribution, individual changes, or how many participants crossed the response threshold. Such information is not reported in the registry record.
How should a confidence interval be understood?
In general, a confidence interval describes uncertainty around an estimated quantity under the assumptions of the statistical method. For a percentage, the interval communicates how precisely the observed proportion is estimated. It does not guarantee that the true value lies within a particular interval, nor does it resolve limitations in study design or measurement. The endpoint specifies a 95% confidence interval, but no interval values are reported in the registry record. Consequently, precision cannot be judged.
For binary outcomes, the amount of information depends on both the number meeting the endpoint and the number evaluated. A small evaluation group generally offers less precision than a larger group, but the study’s endpoint denominator is not reported. Enrollment of 18 alone does not establish how many participants contributed data to the Week 8 measure. It would be inappropriate to substitute enrollment for the outcome-specific denominator without registry evidence.
What does the study design allow?
Prospective collection means outcomes are specified for assessment over follow-up, while a real-world study aims to describe outcomes in a clinical setting beyond a tightly controlled efficacy comparison. These general features do not, by themselves, ensure complete follow-up or eliminate confounding. The record calls the study low-interventional and multicenter, but does not give further detail about site-level procedures, treatment exposure, or the reasons for termination.
The record does not describe a randomized comparison, and allocation is not stated. Its own caution says this is not a randomized comparison and that arms are not a like-for-like control comparison. In general, a nonrandomized comparison may reflect differences between groups beyond treatment exposure. Since no valid control comparison is described, the results, if reported, would need to be interpreted according to the study’s observational context rather than as a randomized treatment contrast.
What are the planning illustrations?
Sample-size calculations are planning tools, not outcomes observed in the trial. The stated illustration assumptions use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. The assumptions distinguish time-to-event illustrations, which use a total event count, from other illustrations, which use participants per arm. These are general planning conventions, not evidence that the trial used those assumptions in its design.
The registry record does not report a sample-size justification, and the study is not described as a randomized two-arm comparison. The observed primary effect needed to interpret an effect-based planning illustration is not reported in the registry record. Any such sample-size illustration should therefore be understood as conditional planning arithmetic rather than as observed power, a design feature, or evidence that the study could detect a specified clinical effect.
Why can an analysis not be reconstructed?
Reconstructing an endpoint estimate would require the outcome count and the evaluable denominator, along with the applicable analysis method. The registry record reports 0 analyses and does not provide these primary outcome details. No unadjusted recalculation can be made from the information reported. There is also no basis for inferring missing observations, imputing outcomes, or selecting a statistical model. Those choices require information about the actual data and analysis plan, which is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 47 outcome measures. The outcomes described include clinical response and remission, IBDQ response and remission, and biochemical remission at Week 8 or Week 16. Assessing multiple outcomes can raise a multiplicity issue: when many hypotheses are tested, the chance of at least one apparently notable finding can increase. In general, an analysis plan may address this by identifying a primary outcome, specifying a testing hierarchy, or adjusting inference. A multiplicity adjustment or testing strategy is not reported in the registry record.
The registry identifies clinical response at Week 8 as the primary endpoint. It does not report its estimate or statistical analysis, and it lists 0 analyses. No p-values or confidence interval values are reported for the endpoint. As a result, there is no set of statistical tests from which to assess whether multiplicity affected the interpretation.
Missing-data methods are also not reported in the registry record. The record defines the full analysis set as participants who had a baseline visit and received a study identification number. It further explains that the analyzed number for an outcome is the number evaluable for that measure. This distinction matters because enrollment, inclusion in the full analysis set, and evaluability for a particular follow-up outcome need not refer to the same denominator. The primary outcome denominator and the handling of missing Week 8 data are not reported.
Reasons for missing assessments, methods for handling incomplete questionnaires or biomarker samples, and any sensitivity analyses are not reported in the registry record. Without these details, it is not possible to assess whether missingness could have changed the reported endpoint estimate. No assumptions about imputation, exclusion, or follow-up completion should be made.
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 |
|---|---|---|---|
| All Participants | 1 | 18 | 5.6% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| All Participants | 0 of 18 |
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 |
|---|---|
| All Participants | 4 of 18 |
Most frequent serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Worsening Ulcerative Colitis | Gastrointestinal disorders | 1 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants |
|---|---|---|
| Worsening Ulcerative Colitis | Gastrointestinal disorders | 2 of 18 |
| Colitis | Gastrointestinal disorders | 1 of 18 |
| Enterocolitis infectious | Infections and infestations | 1 of 18 |
| Shingles | Infections and infestations | 1 of 18 |
Serious events with the highest arm-level rate: worsening ulcerative colitis (5.6%).
The registry safety entry lists All Participants as the group, with 18 at risk. It reports 1 affected by serious adverse events and 4 affected by other adverse events, with 0 deaths. These are the posted counts; the registry record does not provide the detailed event-level information needed to interpret their timing, duration, severity beyond the serious classification, or relationship to treatment.
The leading serious event term is Worsening Ulcerative Colitis, listed at 5.6%. That entry is not a complete account of all serious events, and its percentage should not be treated as an efficacy result. The registry does not explain in the available information how the term-level percentage relates to the affected count or whether participants experienced more than one event. The serious and other affected counts also should not be added to infer a count of distinct participants, because overlap is not reported.
These figures describe events reported in this registry entry. They do not establish that tofacitinib caused an event or that an event was unrelated to treatment. A comparison group and further event details are not described, so comparative safety conclusions cannot be drawn from these counts alone.
12. What the Result Does, and Does Not, Mean
The record establishes that a prospective, multicenter study of tofacitinib induction therapy in ulcerative colitis was registered, that enrollment was 18, and that the study status is terminated. It also specifies a Week 8 clinical response endpoint and several clinical, biochemical, and patient-reported outcomes. These details show what the study set out to assess.
The record does not report the primary response percentage, the number evaluable for that endpoint, or its confidence interval. It therefore does not show how often participants met the response definition or how precisely that proportion was estimated. The listed endpoint definition is not itself a result. Likewise, the secondary outcome titles do not reveal whether response, remission, or patient-reported improvements were observed.
The record also does not establish a treatment effect. It does not describe random allocation or a like-for-like comparator, and it cautions that arms are not a comparable control comparison. Even if a descriptive outcome estimate were available, interpretation would depend on the population assessed, follow-up completeness, and analysis methods. Those details are not fully reported.
The safety entry reports serious and other adverse event counts and 0 deaths, but does not establish causality or permit comparative safety assessment. The reason for termination is not reported in the registry record, so termination should not be taken as evidence about efficacy or harm.
Overall, the available registry information supports a description of the research question, design labels, endpoint definitions, and limited safety counts. It does not support a conclusion that treatment worked or did not work. The absence of posted analyses is a reporting limitation, not a result in either direction.
13. Why This Trial Matters Statistically
This study raises statistical issues that commonly arise when evaluating real-world treatment outcomes. Its primary measure is a binary clinical response at a fixed follow-up time, while its stated outcome set spans clinical, biochemical, and patient-reported measures. Together, those endpoints illustrate why clear definitions and outcome-specific denominators matter: a response percentage is only interpretable when the event rule and the number evaluated are known.
The partial Mayo response definition combines score change and a remission threshold. This makes the clinical meaning of the endpoint depend on both baseline and follow-up assessment, and it compresses changes into a responder classification. Statistical reporting would ideally allow readers to understand the estimate and its precision, not only the threshold used. The registry specifies a 95% confidence interval but does not report its values.
The real-world, nonrandomized framing also matters. Such studies can describe outcomes in a clinical population, but a treatment comparison requires a credible comparator and consideration of differences between groups. The registry cautions that the study is not a randomized comparison and that arms are not like-for-like controls. That caveat limits causal interpretation regardless of how an outcome is defined.
Finally, the record lists multiple outcomes but reports 0 analyses. Multiplicity, missing follow-up, and the analysis population could all affect interpretation, yet the corresponding statistical details are not reported. This makes the study a useful example of the distinction between a registered endpoint and a reported result: a precise question can be specified even when the information needed to estimate its answer is absent from the registry record.
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 this trial is that the registry record does not report the primary endpoint result. It gives no Week 8 response percentage, outcome-specific denominator, or confidence interval. The listed enrollment of 18 cannot substitute for those details, since the record distinguishes enrollment from participants evaluable for an outcome.
The record lists 47 outcome measures and 0 analyses. It does not provide results for the secondary outcomes, statistical methods for the endpoint, or a multiplicity strategy. Missing-data handling, reasons for incomplete assessment, and sensitivity analyses are also not reported in the registry record. These omissions prevent assessment of precision, robustness, and the possible influence of missing observations.
The design is described as low-interventional, prospective, multicenter, and real-world, but allocation is not stated. The registry warns that the study is not a randomized comparison and that arms are not like-for-like controls. A causal treatment comparison is therefore not supported by the described design information. The reason for the terminated status is not reported, and no inference about the reason is warranted.
The intervention listing names stool sample collection, while the study title and official title concern tofacitinib induction therapy. The record does not supply dosing or fuller treatment administration details. Finally, the safety figures lack event-level context and do not establish attribution. These limits apply to what can be concluded from the registry record, not necessarily to information collected by the investigators outside it.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Generate Real World Data On Tofacitinib Induction Therapy and Changes In Clinical and Patient Reported Outcomes. — NCT05069259.
- Registry record last updated 2025-12-19; status last verified 2025-12.
- Results first posted 2025-04-18.
- 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
NCT05069259 is a prospective, multicenter, low-interventional study concerning real-world responses to tofacitinib induction therapy in moderately to severely active ulcerative colitis in Switzerland. Enrollment is listed as 18; status is terminated. The primary endpoint is the percentage achieving clinical response at Week 8, defined using change in partial Mayo score or clinical remission.
The registry lists 47 outcome measures and 0 analyses. The primary result, its evaluable denominator, and its confidence interval are not reported in the registry record. Safety entries list 1 affected by serious adverse events, 4 affected by other adverse events, 0 deaths, and Worsening Ulcerative Colitis as the leading serious event term at 5.6%. Allocation, the reason for termination, and the statistical handling of missing data are not reported in the registry record.