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 the Treatment of Vitiligo (NCT02809976), a na phase 2 study. The primary outcome is Percent Change in Vitiligo Area Severity Index (VASI) Score From Baseline to Week 20.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02809976 |
| Phase | Phase 2 |
| Condition | Vitiligo |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 11 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Tufts Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
Adults with vitiligo. The registry record lists an enrollment of 11 participants.
Intervention
Ruxolitinib 1.5% phosphate cream applied twice daily to vitiligo patches.
Comparator
No separate comparator group is listed. This was a single-group, open-label study.
Primary question
What was the percent change in Vitiligo Area Severity Index (VASI) score from baseline to Week 20?
The study was framed as a proof-of-concept pilot examining whether topical ruxolitinib could be associated with repigmentation in vitiligo lesions. The primary endpoint was the percent change in VASI score from baseline to Week 20. The registry record names this outcome and its measurement interval, but its numerical estimate and confidence interval are not reported in the registry record.
The question is therefore about change over time among participants receiving the cream, not a comparison of outcomes between treatment and control groups. Without a separate comparator, any change observed during the study could not by itself distinguish an intervention effect from other explanations for change over time. The study's open-label design also means masking was not used, according to the registry record.
The clinical question is narrow: it concerns change in a measure of depigmented skin area and extent over the stated interval. The registry also lists secondary outcomes covering repigmentation, physician assessment, VETF assessments, and dermatology-related quality of life. Numerical results for those outcomes are not reported in the registry record. The record therefore defines what the pilot set out to assess, but does not provide enough outcome data to describe the magnitude or precision of change.
3. Trial Design
This was a phase 2, open-label, single-group proof-of-concept pilot study of topical ruxolitinib in adults with vitiligo. The listed intervention was ruxolitinib 1.5% phosphate cream, applied twice daily to vitiligo patches. The registry lists enrollment of 11 and study status as completed. The recorded start and primary completion dates are 2016-01 and 2017-01.
There was no separate control arm in the listed design. Allocation is recorded as not applicable, and masking is recorded as none. These design features matter when interpreting change in the primary outcome: the study can describe change in the treated group, but its design does not supply a concurrent comparison that would help separate treatment-associated change from other changes over time. An open-label design also means participants and study personnel were not masked to treatment assignment.
The study's stated purpose was to determine whether the cream would provide repigmentation in vitiligo lesions. The primary outcome was percent change in VASI score from baseline to Week 20. Six secondary outcomes are listed: change in body surface area of repigmentation; the number of participants reaching a Physician Global Vitiligo Assessment of clear or almost clear; VETF body surface area, disease staging, and disease progression assessments; and mean Dermatology Life Quality Index scores.
The registry identifies the study as a pilot, but a protocol rationale for the enrollment size, formal sample-size justification, eligibility details, treatment duration beyond the primary outcome interval, and analysis populations are not reported in the registry record. The registry record also does not report an allocation procedure, which is consistent with the single-group design. These omissions limit how fully the conduct and analysis can be reconstructed from the record alone.
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 1.5% Phosphate Cream |
|---|---|
| STARTED | 11 |
| COMPLETED | 9 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Ruxolitinib 1.5% Phosphate Cream |
|---|---|
| Lost to Follow-up | 1 |
| Withdrawal by Subject | 1 |
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 1.5% Phosphate Cream |
|---|---|---|
| Participants analysed | 11 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 11 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 52 (33 to 65) |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | Participants | 6 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 4 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 7 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 4 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 4 |
| Region of Enrollment: United States | Participants | 11 |
6. Primary Endpoint
The registry lists the primary outcome as Percent Change in Vitiligo Area Severity Index (VASI) Score From Baseline to Week 20, measured over Baseline to Week 20.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | percentage of change in VASI |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is percent change in Vitiligo Area Severity Index (VASI) score from baseline to Week 20. VASI combines the area of skin affected with the extent of depigmentation. The registry description divides assessment across body regions and uses hand-print units to estimate area; the degree of depigmentation is assessed on a scale from no depigmentation to complete depigmentation. The endpoint therefore reflects both how much skin is involved and how much pigment loss is present in the assessed area.
As a general measurement principle, a continuous score can retain gradations of change rather than reducing a participant's status to a simple yes-or-no category. VASI is intended to represent the extent of vitiligo across specified body regions, making it relevant to a study concerned with repigmentation. The percent-change form expresses change relative to a participant's baseline score. The registry record identifies the endpoint as a mean and specifies a 95% confidence interval, but it does not report the estimate or interval.
The direction and size of an observed change would need to be interpreted using the study's precise calculation and reporting conventions. Those numerical results and further details of the calculation are not reported in the registry record. Thus, the endpoint definition explains what the study intended to measure, but cannot establish how much change occurred or how precisely it was estimated.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a primary outcome and a measurement interval, but does not report the primary outcome's numerical result. In particular, the mean percent change in VASI score and its 95% confidence interval are not reported in the registry record. Without those values, the magnitude, direction, and precision of the primary outcome cannot be described from the posted results.
A confidence interval is a statistical range intended to convey uncertainty around an estimate. As a general explanation, a narrower interval indicates greater precision than a wider interval, while the interval's position helps readers understand which values are compatible with the data under the analysis assumptions. In this case, the registry names a 95% confidence interval as the dispersion measure, but gives no interval to interpret. It is therefore not possible to assess precision or determine whether the estimate is compatible with a wide range of changes.
The single-group design also constrains interpretation independently of whether an outcome estimate is available. A change from baseline would describe the experience of participants in the treated group during the study. Without a control group, it would not provide a direct estimate of how outcomes would have differed without treatment. The open-label design is another relevant feature when assessing outcomes that involve observation or assessment. The registry record does not report further details that would allow the influence of these features to be quantified.
The record lists six secondary outcomes in addition to the primary endpoint. Their numerical results are not reported in the registry record. The listed outcomes cover clinical assessments and quality of life, but the outcome names alone do not show whether or how participants changed. Likewise, an outcome's presence in the registry does not establish that a particular result was observed.
The safety information has a different scope from the primary efficacy endpoint. The record lists zero participants with serious adverse events among 11 at risk, zero deaths, and 9 participants with other adverse events among 11 at risk. The serious-event terms are not listed. These counts describe the recorded safety figures; they do not fill the gap in the primary outcome result, nor do they establish the absence of possible harms.
Overall, the record supports a description of the study question, design, and reported adverse-event counts, but not a quantitative conclusion about the primary outcome. Any claim about the amount of repigmentation, the uncertainty around that change, or the relation between treatment and change would go beyond the numerical results reported in the registry record.
9. Statistical Methods Explained
The registry describes the primary endpoint as the mean percent change in VASI score from baseline to Week 20, with a 95% confidence interval. This identifies the intended summary measure and a way of expressing its uncertainty. The numerical mean and interval are not reported in the registry record, so the estimate cannot be interpreted or checked against a clinically meaningful threshold from this record.
What does a mean describe?
As a general statistical explanation, a mean combines individual measurements into a single summary of their central level. For change-from-baseline outcomes, it can describe the average change among participants whose measurements are included in the analysis. That summary does not reveal how similar or different the participants' individual changes were. A mean may be accompanied by a measure of uncertainty, but the confidence interval listed for this endpoint is not numerically reported.
The study is described as a single-group study, so the primary endpoint concerns change within the treated group over time. A within-group change is not the same quantity as a treatment-versus-control contrast. General statistical interpretation of change over time must distinguish the observed course in a group from the causal effect of an intervention. The design has no listed control group to provide a concurrent estimate of what might have happened otherwise.
How should a confidence interval be read?
In general, a confidence interval expresses the uncertainty associated with an estimate under the assumptions of the statistical procedure used. It is not a guarantee that the true value lies in the interval, and it does not describe the distribution of individual participant outcomes. A reader would use the interval's endpoints and width to judge the range of estimates reasonably compatible with the analysis. Here, the registry reports the intended confidence level but not the endpoints, so this interpretation cannot be carried out for the primary result.
The registry lists the primary measure as a mean and the dispersion as a 95% confidence interval. It does not report the calculation method for the interval, the treatment of missing observations, whether the analysis adjusted for other variables, or the analysis population. Those details are not reported in the registry record. They should not be inferred from the endpoint description alone.
What does the design imply for comparisons?
As a general principle, random assignment and a concurrent comparator can support a treatment contrast by balancing or accounting for influences on outcomes. This study is not described as randomized and has no separate comparator arm. Its allocation is recorded as not applicable. Therefore, a statistical comparison between treatment and control groups is not available from the stated design. Any change from baseline would remain a description of the treated group, rather than a randomized estimate of a treatment effect.
The study is open label, with masking recorded as none. As a general matter, lack of masking may be relevant to the conduct of assessments and the reporting of subjective outcomes. The registry record does not report whether or how such influences were addressed. The endpoint includes an assessment of depigmentation and area, but the record does not provide enough detail to assess the impact of the open-label design on measurement.
How do planning illustrations differ from observed results?
Planning illustrations are not outcomes observed in participants. The registry's described planning assumptions use a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation, with the effect to detect defined as a stated fraction of the observed effect. These assumptions describe a planning calculation, not the study's single-group design. They do not establish the trial's original sample-size justification, and that justification is not reported in the registry record.
Any sample-size illustration based on an observed effect is especially dependent on the estimate used as its input. It should be read as a planning exercise under stated assumptions, not as evidence that the study had a particular probability of detecting an effect. Such an illustration is not observed power. The registry record does not report a trial-specific sample-size calculation or a rationale for enrolling 11 participants.
General statistical considerations also apply to a small pilot: estimates can be imprecise, and a few participant outcomes may have substantial influence on a group summary. That principle does not establish the precision of this study's primary result; the result and interval are not reported. The record's enrollment count alone cannot supply the missing estimate, its variability, or the analysis details needed to interpret it.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome and six secondary outcomes. The secondary outcomes include body surface area of repigmentation, a physician global assessment threshold, VETF assessments of body surface area, disease staging and disease progression, and mean DLQI scores. The registry record identifies these outcomes but does not report their numerical results.
When multiple outcomes are assessed, a general statistical concern is multiplicity: the more hypotheses or comparisons examined, the greater the chance that some results may appear noteworthy by chance. Statistical approaches can address this concern, but the registry record does not report whether multiplicity adjustments were planned or used. It also does not report a hierarchy among the secondary outcomes or a broader statistical analysis plan. No adjustment method should be assumed.
Missing-data handling is also not reported in the registry record. The record does not state how many participants contributed to each endpoint, how missing measurements were treated, or whether any imputation or other method was used. Enrollment of 11 does not establish that all 11 contributed to every analysis. The analysis populations, including whether the primary analysis used all enrolled participants or a subset, are not reported in the registry record.
These omissions matter because the choice of outcome population and missing-data method can affect an estimate and its uncertainty. They also limit reproducibility: readers cannot reconstruct the primary analysis or assess how incomplete measurements might have influenced it. The endpoint names and study enrollment count alone do not resolve these questions.
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 1.5% Phosphate Cream | 0 | 11 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Ruxolitinib 1.5% Phosphate Cream | 0 of 11 |
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 1.5% Phosphate Cream | 9 of 11 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ruxolitinib 1.5% Phosphate Cream |
|---|---|---|
| Rim of hyperpigmentation surrounding vitiligo lesion | Skin and subcutaneous tissue disorders | 9 of 11 |
| Intermittent erythema over affected lesions | Skin and subcutaneous tissue disorders | 8 of 11 |
| URI symptoms | Respiratory, thoracic and mediastinal disorders | 4 of 11 |
| Transient acne | Skin and subcutaneous tissue disorders | 2 of 11 |
| Sore throat | Respiratory, thoracic and mediastinal disorders | 1 of 11 |
| Muscle aches | Musculoskeletal and connective tissue disorders | 1 of 11 |
| hypercholesterolemia | Metabolism and nutrition disorders | 1 of 11 |
| Traumatic blister | Social circumstances | 1 of 11 |
| Bee sting | Social circumstances | 1 of 11 |
| Dental pain | General disorders | 1 of 11 |
The registry lists 11 participants at risk for serious adverse events and zero participants affected. It lists 11 participants at risk for other adverse events and 9 affected. The death count is zero. These figures are counts of participants, not a detailed description of event types, severity beyond the serious-event category, timing, or relationship to treatment.
No serious adverse-event terms are listed in the record. The meaning of the other adverse-event count is limited by the absence of event descriptions: the record does not report which events occurred or how many individual events there were. A participant count does not necessarily correspond to an event count, because a participant can experience more than one event; event-level detail is not reported in the registry record.
As a general statistical point, observing zero serious adverse events in a study does not prove that the risk is zero. A small study can describe what was recorded among its participants, but it may not be able to characterize uncommon events precisely. The zero count should therefore be read as a registry-reported observation in this study, not as evidence that serious events cannot occur. The listed figures do not establish a comparison of safety with another treatment, because no comparator group is listed.
12. What the Result Does, and Does Not, Mean
The record establishes that this completed phase 2 pilot examined topical ruxolitinib 1.5% phosphate cream in adults with vitiligo. It specifies twice-daily application to vitiligo patches and identifies percent change in VASI score from baseline to Week 20 as the primary endpoint. It also lists secondary clinical and quality-of-life outcomes and reports adverse-event participant counts.
The record does not report the numerical primary outcome, its 95% confidence interval, or numerical results for the secondary outcomes. Consequently, it does not allow a reader to quantify change in VASI, assess the precision of that change, or determine what the listed secondary assessments found. The absence of a numerical result in the registry record should not be treated as evidence of no change or as evidence of a particular change.
The single-group, open-label design also defines what the study can establish. As a general interpretation principle, observed change within a group is not by itself a controlled estimate of what would have happened without the intervention. There is no separate comparator in the listed design, and the registry does not describe random allocation. This means the record cannot support a like-for-like treatment comparison.
The safety counts report zero participants with serious adverse events among 11 at risk, 9 participants with other adverse events among 11 at risk, and zero deaths. These are descriptive counts. The serious-event terms are not listed, and the record does not provide enough detail to assess event types, timing, or comparative safety.
The study can be understood as a small proof-of-concept investigation with a clearly named primary measure, but the posted information is insufficient to evaluate the measured outcome quantitatively. A fuller interpretation would require the outcome estimates, uncertainty measures, analysis methods, and information on missing observations and analysis populations, which are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of the difference between a defined research question and a reported result. The registry specifies a population, intervention, primary endpoint, and measurement interval. That structure tells readers what the investigators intended to assess. However, without the numerical primary estimate and its confidence interval, the central result cannot be assessed for magnitude or precision.
The design also makes clear why a within-group outcome and a controlled treatment contrast are not interchangeable. In a single-group study, the primary question concerns change over time among people receiving the intervention. General statistical reasoning cautions that such change does not isolate the intervention's contribution. The absence of a separate control group is therefore central to interpretation, not a minor technical detail.
The study's pilot designation and enrollment of 11 underscore the distinction between exploratory evidence and a definitive comparative estimate. The registry does not report the sample-size justification or a trial-specific analysis plan. Planning illustrations based on an observed effect are separate from the study's observed results and should not be interpreted as proof of adequate power or as a substitute for outcome reporting.
The record also illustrates why outcome reporting must include more than endpoint names. A stated confidence-interval method is useful only when the estimate and interval are reported; a list of secondary outcomes does not reveal their findings; and adverse-event counts need context to describe event types and completeness. These gaps limit statistical interpretation even when the study question and design are clear.
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 registry record gives limited information for reconstructing the study methods. It identifies an open-label, single-group phase 2 pilot and lists enrollment of 11, but eligibility criteria, participant characteristics, recruitment details, and the sample-size rationale are not reported in the registry record. The record does not report the primary outcome estimate or its 95% confidence interval.
Although the primary endpoint is defined, the record does not report how missing measurements were handled, which participants were included in each analysis, or the statistical procedure used to calculate the mean and confidence interval. These omissions prevent assessment of whether the reported analysis, if any, followed a prespecified plan. Details of outcome-assessment procedures beyond the VASI description are not reported in the registry record.
The design has no separate comparator group and is not masked. These features restrict causal interpretation and may matter for outcome assessment. The registry record does not describe procedures to address possible measurement or reporting influences associated with the open-label design.
Numerical results for the listed secondary outcomes are not reported in the registry record, and no multiplicity approach is described. Safety reporting includes participant counts, but serious-event terms and detailed event descriptions are not listed. The record therefore supports a limited account of the study's aims, structure, and reported safety counts, rather than a complete evaluation of efficacy, safety, or analysis quality.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Topical Ruxolitinib for the Treatment of Vitiligo — NCT02809976.
- Registry record last updated 2020-08-31; status last verified 2020-08.
- Results first posted 2020-08-31.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record identifies NCT02809976 as a completed phase 2, open-label, single-group pilot study of topical ruxolitinib for adults with vitiligo. Tufts Medical Center is listed as sponsor. The recorded study start is 2016-01, and the primary completion date is 2017-01. Enrollment is listed as 11.
Participants were assigned ruxolitinib 1.5% phosphate cream, applied twice daily to vitiligo patches. The primary outcome was percent change in VASI score from baseline to Week 20, summarized as a mean with a 95% confidence interval. The numerical primary result is not reported in the registry record. Six secondary outcomes are listed, but their numerical results are not reported.
The safety record lists zero participants with serious adverse events among 11 at risk, 9 participants with other adverse events among 11 at risk, and zero deaths. Serious-event terms are not listed. The record does not report the sample-size justification, missing-data methods, analysis populations, or a detailed statistical analysis plan.