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 Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD) (NCT03395340), a non-randomized phase 2 study. The primary outcome is Percent Change in the Surface Area Measurement of the Target Lesions for Ruxolitinib Treated Versus Placebo Treated Lesions.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03395340 |
| Phase | Phase 2 |
| Condition | Graft Versus Host Disease, JNS Kinase, Topical Administration |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | Triple |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS) |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was people ages 12 and older with epidermal skin chronic graft versus host disease (cGVHD). The registry describes cGVHD as a condition in which donor immune cells may attack the recipient’s tissues; skin is identified as the most commonly affected organ. The record reports enrollment of 1 participant, so the population intended for the study and the population actually represented in its record are very different in scale.
Intervention
The intervention was topical ruxolitinib 1.5% cream. The study description says participants would apply ruxolitinib cream and vehicle cream to separate areas of disease twice a day for 6 weeks, provided serious side effects did not occur. The registry does not report how much cream was applied or the treatment exposure actually received by the participant.
Comparator
The comparator was vehicle cream applied to another area of disease. This is described as a comparison between treated skin locations in the same participant, rather than a comparison between separate groups of participants. The registry labels the study non-randomized and single-group; it does not report a randomized assignment procedure for the skin locations.
Primary question
The primary question was whether the percent change in surface area differed between target lesions treated with ruxolitinib and lesions treated with placebo or vehicle, from baseline to week 6. Surface area was to be measured by tracing lesions. The record does not report a numerical result for this endpoint, so it cannot establish the size, direction, or precision of any observed difference.
3. Trial Design
This is a phase II study of topical ruxolitinib for cutaneous cGVHD, registered as NCT03395340. The registry identifies the allocation as non-randomized, the masking as triple, and the model as single-group. Its description says the participant would receive both ruxolitinib cream and vehicle cream on separate areas of disease. Thus, the intended comparison is at the lesion level within a participant, not a conventional comparison of independent treatment groups.
The registry describes screening with medical history, physical examination, blood and urine tests, and a skin biopsy to confirm the diagnosis. At baseline, skin disease was to be measured using rulers, photographs, and lesion tracings, with symptom questionnaires and blood and urine tests also planned. Some participants might also have a skin biopsy or total-body photographs. The description says both creams would be applied twice a day for 6 weeks if serious side effects did not occur. These are descriptions of the planned study procedures; the record does not establish which procedures were completed.
The primary endpoint was the percent change in surface area measurement of target lesions for ruxolitinib-treated versus placebo-treated lesions, from baseline to week 6. The registry also lists changes in overall severity, pain, and pruritus visual analog scales, along with pharmacokinetic measures of ruxolitinib. Although the record describes a local lesion comparison, its non-randomized designation means the comparison should not be treated as a randomized control contrast. The registry explicitly cautions that the listed arms are not a like-for-like control comparison.
The recorded enrollment was 1, and the study status is terminated. The registry lists a start date of 2018-11-19 and a primary completion date of 2019-01-31. It does not report why the study was terminated or whether the planned treatment period and assessments were completed.
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 | Ruxolitinib Cream |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
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 | Ruxolitinib Cream |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 51.9 (SD 0) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 1 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percent Change in the Surface Area Measurement of the Target Lesions for Ruxolitinib Treated Versus Placebo Treated Lesions, measured over Baseline to week 6.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percent change |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint concerns change in the surface area of target skin lesions. The registry specifies percent change from baseline to week 6, comparing lesions treated with ruxolitinib cream against lesions treated with placebo or vehicle cream. Lesion area was to be measured by tracing the lesion on transparency paper and measuring the area from the tracing. The record states that a decrease in active surface area would signify improvement in skin disease; it does not report whether such a decrease was observed.
This endpoint is relevant to the stated study question because it quantifies the extent of active skin involvement at the treated locations. A lesion-level measurement can be used to contrast the two topical preparations within the same participant, avoiding a comparison across separate participant groups. That general advantage does not by itself remove the limitations of a non-randomized comparison: differences between selected skin sites, measurement variability, or other influences may affect the contrast. The registry does not report details of site selection or a method for addressing those issues.
No numerical primary endpoint result, estimate of variability, confidence interval, or analysis is reported in the registry record. Consequently, the endpoint definition indicates what the study intended to measure, but it cannot support a conclusion about the observed difference or its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive fact is that the registry records enrollment of 1 participant but lists no analyses. It does not report a numerical result for the primary endpoint. There is therefore no posted estimate of the percent change in lesion surface area, no reported contrast between ruxolitinib-treated and vehicle-treated sites, and no confidence interval or other measure of uncertainty for that comparison. The endpoint description explains how the outcome was intended to be measured; it is not evidence that a particular change occurred.
The design was described as a within-participant comparison of different skin locations, but the registry classifies the study as non-randomized and single-group. This distinction matters. A paired lesion comparison may reduce some between-person variation in a general statistical sense, because both sites belong to the same participant. However, the registry does not report how the locations were selected or assigned, or how differences between lesions were handled. It also cautions that the listed arms are not a like-for-like control comparison. The observed contrast, if one were available, would need to be interpreted in light of these design features.
There is no basis in the record to assess the size or direction of a treatment-associated difference, whether the estimate is precise, or whether the observed data are compatible with a range of possible effects. General statistical interpretation relies on estimates and uncertainty measures; neither is reported for the primary endpoint. No inferential result or p-value is listed, and no analysis is recorded. The registry status is terminated, but the reason for termination is not reported in the registry record. It should not be inferred from the status alone.
The enrollment count also sharply limits what can be learned about variation across participants. The record does not give enough information to characterize outcomes across a broader cGVHD population, and the study cannot be read as establishing a population-level pattern. This is a limitation of what the posted record can support, not evidence that a treatment effect is absent or present.
Accordingly, the registry supports a description of the intended endpoint, design, enrollment, and status. It does not support a numerical judgment about efficacy, the magnitude or precision of an effect, or a comparison of benefits and harms. Any such judgment would require outcome and analysis information not reported in the registry record.
9. Statistical Methods Explained
Statistical methods can clarify what a study was designed to estimate and how much uncertainty surrounds its findings. In this record, the endpoint and broad design are described, but the registry does not report a statistical analysis plan, an endpoint result, or any analyses. The distinctions below therefore explain the relevant concepts generally and identify what cannot be assessed from the record.
What contrast was intended?
The intended primary contrast was percent change in target-lesion surface area for ruxolitinib-treated sites versus placebo- or vehicle-treated sites from baseline to week 6. In general, an estimand is the clearly specified treatment comparison a study aims to quantify, including the outcome, population, time point, and handling of events that affect interpretation. Here, the record names the outcome and timeframe, and describes a participant with separate treated locations. It does not report a complete estimand definition, such as how changes in treatment because of side effects or missing week 6 measurements would be incorporated.
Because both preparations were intended for separate areas in the same participant, the comparison is naturally understood as a paired, lesion-level contrast. In general, paired analyses account for the fact that two measurements come from the same individual rather than treating them as independent observations. This can be useful when participant characteristics influence both measurements. It does not guarantee that the skin sites are otherwise comparable. The registry says allocation was non-randomized and does not explain how sites were chosen or assigned, so the possible influence of lesion location or baseline differences cannot be evaluated.
How should uncertainty be assessed?
In general, a confidence interval gives a range of effect estimates compatible with the data and the statistical method, and indicates the precision of the estimate. A narrow interval generally represents greater precision than a wide interval, while neither interval width nor an estimate alone establishes clinical importance. No confidence interval or other uncertainty measure for the primary endpoint is reported in the registry record. With no endpoint estimate listed, the magnitude and precision of the intended contrast cannot be assessed.
In general, a p-value measures how unusual data at least as inconsistent with a specified null hypothesis would be under the assumptions of a statistical model. It does not measure the probability that a treatment is effective, and it does not describe the size or clinical importance of an effect. The registry does not list a primary endpoint p-value or an analysis. There is therefore no inferential test to interpret.
What does the sample-size illustration represent?
The planning illustration is described as a calculation from an observed effect, not as the study’s own design justification. It uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. For time-to-event outcomes, the illustration concerns total events; for other outcomes, it concerns participants per arm. These are planning concepts, not observed results, and they do not represent observed power. The registry record does not report the study’s own sample-size justification. It also does not report a primary effect estimate from which the planning illustration could be interpreted in context.
In general, a sample-size calculation depends on assumptions about the effect to detect, outcome variability, design, allocation, and error rates. A planning result is conditional on those assumptions; it cannot substitute for an analysis of observed data. In this study, the single-group, non-randomized, lesion-level description also means that a simple two-arm planning illustration may not capture all features of the intended comparison. The record does not report a study-specific calculation or the assumptions used for one.
Why does over-dispersion matter?
General explanation: over-dispersion occurs when count data vary more than a statistical model assumes. If that variation is ignored, standard errors can be too small and uncertainty may appear narrower than warranted. The primary endpoint is a lesion surface-area measure, not a count outcome, and the registry does not report an analysis model or a dispersion estimate. Over-dispersion therefore cannot be identified as an issue in the posted primary analysis; no such analysis is listed.
Other design choices also matter in general. A model might need to account for repeated measurements, pairing, baseline outcome values, or clustering when observations are related. The record describes baseline and week 6 measurements and separate skin sites, but does not report whether an adjusted or paired model was planned or used. It does not give the number of lesions measured or the distribution of their baseline areas. No statistical model can be reconstructed from the registry description alone.
The registry lists 9 outcome measures, including the primary surface-area endpoint, symptom visual analog scale changes, and pharmacokinetic measures. Listing outcomes does not show which were analyzed, how they were analyzed, or what estimates resulted. The record reports 0 analyses. Without analysis details and results, the method and uncertainty of any outcome comparison remain unreported.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 9 outcome measures, including a designated primary endpoint and secondary outcomes covering symptom scales and ruxolitinib pharmacokinetics. When a study examines multiple outcomes, the chance of at least one apparently notable result can increase if each is tested independently. General approaches to multiplicity include identifying a primary outcome in advance, adjusting error rates, or treating secondary findings as exploratory. The registry does not report a multiplicity strategy, a hierarchy among secondary outcomes, or an analysis plan. No analyses are listed, so there are no reported results to assess for multiplicity.
Missing-data handling is also not reported in the registry record. The description says the intended treatment period was 6 weeks if serious side effects did not occur, but does not specify how a participant who stopped treatment early, missed a measurement, or had an unusable lesion tracing would be included in an analysis. These are general issues that can affect interpretation when outcome data are incomplete; the registry does not report whether any such data were missing.
The analysis population is not reported. The record does not say whether the planned analysis would include all enrolled participants, only participants completing treatment, or another defined group. With enrollment of 1 and no analyses listed, no analysis population or handling of deviations from the planned procedures can be evaluated. The record also does not report interim analyses or stopping rules. The terminated status alone does not establish why the study ended or whether a particular statistical rule affected that decision.
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 |
|---|---|---|---|
| Ruxolitinib Cream | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Ruxolitinib Cream | 0 of 1 |
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 |
|---|---|
| Ruxolitinib Cream | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ruxolitinib Cream |
|---|---|---|
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
| Urine discoloration | Renal and urinary disorders | 1 of 1 |
The registry lists safety figures for the Ruxolitinib Cream group, with 1 participant at risk for serious adverse events and 0 participants affected by serious adverse events. It reports 1 participant at risk for other adverse events and 1 participant affected by other adverse events. Deaths are reported as 0. These are participant counts, not rates, and the record does not identify the other adverse event or describe its severity, timing, or relationship to treatment.
These counts should be read in the context of enrollment of 1 participant. They describe what the registry records for this very small enrolled group; they do not provide a stable estimate of how often events might occur in a larger population. The absence of a reported serious adverse event or death in this record does not establish that such events cannot occur. Likewise, the other adverse event count does not show that the cream caused the event. The registry does not report an assessment of causality or additional event details.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II study was registered to examine topical ruxolitinib 1.5% cream for cutaneous cGVHD. It describes a comparison of treated skin locations, with ruxolitinib cream at one location and vehicle cream at another, and identifies change in lesion surface area from baseline to week 6 as the primary endpoint. It also records enrollment of 1 participant and a terminated status.
Those details describe the study’s intent and its recorded conduct; they are not a result about whether the treatments differed. The registry does not report the primary endpoint estimate, a confidence interval, a p-value, or any analyses. It therefore cannot show the direction, size, or precision of a lesion-area difference. The absence of a reported result should not be interpreted as a finding of no effect, just as the study’s objective should not be interpreted as proof of an effect.
The design also limits how a hypothetical contrast should be interpreted. The registry describes a same-participant, different-location comparison, but classifies the study as non-randomized and single-group. It explicitly cautions that the listed arms are not a like-for-like control comparison. In general, non-randomized site comparisons can be affected by differences between the selected lesions, and the record does not explain site assignment or adjustment methods.
The recorded enrollment is too limited to characterize variation across a broad population of people with cGVHD. The safety counts are likewise descriptive of the recorded group and do not establish broader event frequencies. The record does not report why the study was terminated, whether the planned assessments were completed, or how missing information was handled. These gaps prevent conclusions about treatment effect, safety beyond the recorded counts, or the reason the study ended.
13. Why This Trial Matters Statistically
This trial is statistically notable because its stated comparison is at the level of separate skin lesions within a participant, while the registry classifies the study as non-randomized and single-group. Such a design raises a useful distinction between a within-person comparison and a randomized controlled comparison. Pairing can account for some shared participant characteristics in general, but it does not automatically balance differences between lesions or eliminate bias from non-random assignment.
The endpoint also illustrates how measurement choices shape interpretation. The study planned to trace target lesions and compare percent change in surface area from baseline to week 6. A defined, measurable outcome can make a clinical question more concrete, but interpretation still depends on the observed estimate, its uncertainty, and details of how measurements were collected and analyzed. Those results and methods are not reported in the registry record.
Finally, the record demonstrates why outcome definitions and study intentions should be distinguished from evidence generated by analysis. Enrollment was 1, the study status is terminated, and no analyses are listed. Without an estimate or uncertainty measure, statistical significance, effect magnitude, and precision cannot be evaluated. The recorded safety counts also require cautious reading because they describe a very small group and lack event-level detail. Together, these features make the registry useful for understanding the planned question and design, while sharply limiting what can be concluded from the posted results.
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 is the absence of reported outcome analyses. The registry defines the primary endpoint but gives no numerical result for lesion surface-area change, and reports 0 analyses. There is no estimate or uncertainty measure with which to assess the direction, magnitude, or precision of the intended comparison.
Enrollment was 1, and the study is marked terminated. The registry does not report why it was terminated, whether the participant completed the planned treatment and assessments, or whether the primary endpoint was measured at week 6. It would be inappropriate to infer the reason for termination or the completeness of follow-up from the status alone.
The study is described as non-randomized and single-group, although separate lesions were intended to receive ruxolitinib and vehicle cream. The record does not describe how lesion sites were selected or assigned, whether their baseline characteristics were compared, or how within-participant pairing was handled analytically. It also explicitly cautions that the listed arms are not a like-for-like control comparison. These design details constrain causal interpretation even if a lesion-level estimate were available.
The statistical analysis plan, study-specific sample-size justification, missing-data approach, analysis population, and multiplicity strategy are not reported in the registry record. The record does not give detailed adverse-event descriptions or an assessment of whether events were related to treatment. As a result, neither the efficacy question nor broader safety questions can be evaluated from the registry information alone.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD) — NCT03395340.
- Registry record last updated 2022-07-12; status last verified 2022-06.
- Results first posted 2022-07-12.
- 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
NCT03395340 is a phase II study of topical ruxolitinib for cutaneous cGVHD, sponsored by the National Institute of Arthritis and Musculoskeletal and Skin Diseases. The registry describes a non-randomized, triple-masked, single-group study in which ruxolitinib and vehicle cream were intended for separate areas of disease. The primary endpoint was percent change in target-lesion surface area from baseline to week 6.
The record lists enrollment of 1, a start date of 2018-11-19, a primary completion date of 2019-01-31, and status as terminated. It reports no analyses and no numerical primary endpoint result. Safety counts list 0 serious adverse events, 1 other adverse event, and 0 deaths among 1 participant at risk. The reason for termination, endpoint findings, and analysis details are not reported in the registry record.