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 Pilot Study to Evaluate the Efficacy of Ruxolitinib in Alopecia Areata (NCT01950780), a na phase 2 study. The primary outcome is Change in SALT Score.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01950780 |
| Phase | Phase 2 |
| Condition | Alopecia Areata |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Columbia University |
| Results posted | Yes |
2. Clinical Question
The study asked whether hair loss, measured with the Severity of Alopecia Tool (SALT) score, changed during treatment with ruxolitinib in people with moderate to severe alopecia areata. It was a pilot study with a single treatment group and no placebo or other control group. That design can describe changes observed during treatment, but it cannot distinguish a treatment-associated change from changes that might have occurred for other reasons.
Population
People with moderate to severe alopecia areata. The registry lists 12 participants enrolled. Further eligibility details and characteristics of the enrolled group are not reported in the registry record.
Intervention
Ruxolitinib, described as a fixed oral dose of 20mg twice daily for 12 to 24 weeks. The arm description says dosing could be decreased or held if needed because of adverse effects.
Comparator
There was no separate comparator group. The study is listed as single-group, open-label, and without masking; all enrolled participants were intended to receive active medication.
Primary question
How did mean SALT score change from baseline to the scheduled assessments at 3 months and 6 months? The registry describes the measure and timing, but the change estimates and their uncertainty are not reported in the registry record.
The question is descriptive as registered: it concerns change in a continuous score within one group. Without a concurrent control group, the study cannot establish how much of any observed change was attributable to ruxolitinib rather than natural fluctuation, other influences, or measurement variation. The absence of reported outcome estimates also means that the direction, magnitude, and precision of the measured change cannot be evaluated from the registry record.
3. Trial Design
This was a phase 2 pilot study sponsored by Columbia University. The registry lists the study as completed, with a start date of 2013-08 and a primary completion date of 2016-04. Enrollment was 12. The study used a single-group model, had no masking, and did not use a randomized allocation. The registry description says all participants would receive active medication and that there would be no placebo group.
The intervention was oral ruxolitinib, with a planned fixed dose of 20mg twice daily for 12 to 24 weeks. The arm description allows for dosing to be decreased or held if adverse effects required it. The brief summary describes treatment for a minimum of 3 months and up to 6 months. The primary outcome timeframe specifies baseline, 3 months, and 6 months. These descriptions indicate planned assessment points, but the number of participants assessed at each time point is not reported in the registry record.
An open-label design means participants and study personnel were not masked to treatment assignment. In a single-group study, that design can support a description of outcomes during treatment, but it does not provide a randomized comparison against an untreated or alternative-treatment group. The registry record does not report whether outcome assessment itself involved any masking or independent review.
The registry does not report a statistical analysis plan, a sample-size justification, or details of how missing measurements were handled. It also does not report the number of participants completing each assessment, adherence to the intended schedule, or reasons for any dose changes. These omissions limit evaluation of how the planned design was implemented and how representative the outcome measurements were of all enrolled participants.
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 |
|---|---|
| STARTED | 12 |
| COMPLETED | 12 |
| 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 |
|---|---|---|
| Participants analysed | 12 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 12 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 7 |
| Sex: Female, Male: Male | Participants | 5 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 9 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 |
| Race (NIH/OMB): White | Participants | 8 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Change in SALT Score, measured over Baseline, 3 months and 6 months.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | score on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change in SALT score. SALT is a continuous measure of scalp hair loss. The scalp is divided into four areas, each assigned a share of the scalp surface area: vertex, 40%; right profile, 18%; left profile, 18%; and posterior aspect, 24%. For each area, the percentage of hair loss is weighted by that area's share of the scalp, and the weighted values are summed.
The reported score ranges from 0, indicating no hair loss, to 100, indicating 100% hair loss. Lower scores therefore indicate less hair loss. The registry specifies a mean and standard deviation as the summary measures and gives baseline, 3 months, and 6 months as the assessment times. This makes the endpoint suitable for describing the group’s average score and the spread of individual scores at those scheduled assessments. A change in the mean can describe the group-level pattern, while the standard deviation conveys how much individual scores vary around that mean.
The registry record does not report the mean scores, standard deviations, or changes between assessment times. It therefore does not show the observed direction or magnitude of change, nor whether participants’ scores varied widely or clustered near the group mean. The clinical interpretation of a score change also cannot be assessed from the registry record because no threshold for an important change is reported.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies change in SALT score as the primary outcome, measured at baseline, 3 months, and 6 months. It does not report the outcome values or an analysis of change. As a result, there is no registry-reported estimate of the mean change, no standard deviation for change, and no confidence interval or p-value with which to assess uncertainty. The record supports no conclusion about the direction or size of change in SALT score.
The safety counts are available separately from the primary outcome. In the Ruxolitinib group, 12 participants were listed as at risk for serious adverse events and 0 as affected. For other adverse events, 12 were listed as at risk and 9 as affected. These counts describe the registry entries, not the primary endpoint, and do not supply information about SALT-score change. Deaths are recorded as null, so a death count is not reported in the registry record. No serious adverse-event terms are listed.
The design also limits what a change could establish even if the outcome estimates were available. In a single-group study without a control group, a before-and-after change would be an observed within-group pattern. It would not, by itself, separate the influence of treatment from the natural course of alopecia areata, changes in other care, regression toward a participant’s usual level after enrollment at a time of more severe disease, or measurement variation. Open-label conduct can also influence behavior and assessment, although the registry record does not report whether the outcome assessors were masked.
Precision cannot be judged without estimates of variability and uncertainty. A mean score alone would not show whether the observed pattern was consistent across participants; the standard deviation and an interval estimate would help describe that uncertainty. Neither the outcome summaries nor inferential results are reported in the registry record. The enrollment of 12 indicates that this was a small pilot study, but sample size alone does not reveal the direction, magnitude, or precision of the outcome.
The registry includes planning illustrations based on a stated effect fraction, two-sided significance level 0.05, equal allocation, and a normal approximation. These assumptions describe hypothetical planning calculations, not an observed comparison in this single-group trial. No planning values are reported in the registry record. Such illustrations should not be read as observed power or as evidence that an effect was demonstrated. Overall, the available registry information describes the question, design, endpoint, and adverse-event counts, but does not provide the primary outcome result needed to judge the observed SALT-score pattern.
9. Statistical Methods Explained
The primary outcome is a continuous score, summarized as a mean and standard deviation. General statistical explanation: a mean describes the average score in a group, while a standard deviation describes the spread of individual scores around that average. Those summaries answer different questions. The mean describes the group’s central level; the standard deviation describes how similar or different participants’ scores are. The registry record specifies these summary measures but does not report their values at baseline, 3 months, or 6 months.
What does change in a continuous score represent?
For a repeated-measures outcome, change describes how scores differ across assessment times. In this study, the planned assessments are baseline, 3 months, and 6 months. A group mean at each time can show the average score at each assessment, while an analysis of within-person change would use each participant’s paired measurements. General statistical explanation: paired measurements account for the fact that repeated observations from the same participant are related. The registry record does not state which specific model or calculation was used to analyze change, and it does not report the number of paired observations available.
For SALT, lower scores represent less hair loss, so the direction of a score difference has an interpretable scale meaning. However, direction alone would not establish the size or importance of a change. The registry record does not report a threshold for a clinically important change. Nor does it report whether the primary analysis compared each follow-up assessment with baseline, assessed a trend over time, or used another prespecified contrast.
How should uncertainty be understood?
General statistical explanation: an estimate is more informative when accompanied by a measure of uncertainty. A confidence interval gives a range of values compatible with the data and the method used to calculate it; it communicates precision, not the probability that a particular treatment effect is true. A narrow interval indicates greater precision than a wide interval, all else being equal. The registry record does not report a confidence interval for SALT-score change, a standard error, or another uncertainty interval. Therefore, the precision of the primary outcome cannot be assessed.
A p-value, if reported, would address how compatible the data are with a specified statistical model and null hypothesis. It would not measure the size or clinical importance of a change. No p-value or inferential test for the primary outcome is reported in the registry record. The record also does not report the null hypothesis, the selected significance level for the primary outcome analysis, or whether any adjustment was planned for multiple comparisons across assessment times.
Why does the study design matter for interpretation?
General statistical explanation: random allocation to a concurrent control group helps balance, on average, factors that can affect outcomes, including factors that are known and unknown at the start of a trial. This study is listed as a single group with no randomized allocation and no placebo group. It can describe the scores observed among participants during the study, but a within-group before-and-after comparison does not isolate a causal treatment effect. Changes over time may also reflect the disease’s course, concurrent influences, selective follow-up, or measurement variability.
Open-label conduct means treatment assignment is known rather than masked. General statistical explanation: knowledge of treatment may affect participant reports, behavior, adherence, or assessment. The registry record does not describe any masking of SALT assessment, so whether outcome measurement was independently protected from knowledge of treatment is not reported in the registry record. This uncertainty does not establish that assessment was biased; it means the record does not allow that safeguard to be evaluated.
What do planning illustrations say?
The registry describes planning illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is expressed as a stated fraction of the observed effect. General statistical explanation: a sample-size calculation estimates the number of participants needed under specified assumptions to have a chosen probability of detecting an effect of a defined size at a chosen significance level. It is a planning exercise, not a result from the trial.
The illustrations are not the design of this study: the trial is single-group and has no randomized comparator, whereas the listed assumptions describe equal allocation between two arms. The calculated planning values are not reported in the registry record. They cannot be interpreted as the observed power of the completed trial. General statistical explanation: observed power is not a substitute for an effect estimate and its uncertainty; the actual data and their precision are the relevant basis for interpreting an outcome. Here the registry does not report the primary outcome estimates needed for that assessment.
What can be inferred from the reported summaries?
The registry record gives the endpoint definition, scale range, scheduled assessments, and summary type. It does not give the mean score, standard deviation, change estimate, confidence interval, or test result. Consequently, no quantitative inference about primary-outcome change can be drawn from the registry record. The adverse-event counts are descriptive safety entries and do not supply a statistical test or estimate for the SALT endpoint.
10. Multiplicity, Missing Data and Analysis Populations
The primary outcome is one continuous measure, SALT score, with assessments scheduled at baseline, 3 months, and 6 months. Comparing multiple follow-up times with baseline can create a multiplicity question: when several statistical tests are conducted, the chance of at least one apparently notable result can increase. General statistical explanation: an analysis plan may address this by defining one primary comparison, adjusting for multiple tests, or using a method that models the repeated measurements together. The registry record does not report which comparisons were considered primary or whether a multiplicity procedure was planned or used.
The record lists a secondary outcome, Percentage of Regrowth, but does not report its result or its analysis. The number of outcome measures is listed as 2, and the number of analyses is listed as 0. The registry record does not explain the analysis count or identify a statistical analysis plan. It is therefore not possible to determine how outcomes or time points were selected for formal testing, or whether any adjustments were made.
Missing data matter when scheduled measurements are unavailable. If participants miss follow-up or stop treatment, the observed participants may differ from those without measurements. General statistical explanation: complete-case analyses can be biased when missingness is related to the outcome, while other methods require assumptions about why data are missing. The registry record does not report how many participants contributed SALT scores at each time, whether any values were missing, why values might be missing, or how missing measurements were handled.
The registry record also does not describe the analysis population, such as whether all enrolled participants were included in the primary analysis or whether analysis depended on treatment exposure or follow-up availability. It does not report protocol deviations or dose changes actually made. Without those details and the outcome data, the impact of missingness, exclusions, or treatment changes cannot be assessed.
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 | 0 | 12 | 0.0% |
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 | 9 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ruxolitinib |
|---|---|---|
| upper respiratory infection | Infections and infestations | 5 of 12 |
| acne | Skin and subcutaneous tissue disorders | 2 of 12 |
| low hemoglobin | Blood and lymphatic system disorders | 1 of 12 |
| pneumonia | Infections and infestations | 1 of 12 |
The registry lists 12 participants at risk for serious adverse events in the Ruxolitinib group, with 0 affected. For other adverse events, it lists 12 at risk and 9 affected. These are counts of participants, not rates calculated across different follow-up durations. The record does not specify event timing, severity for the other adverse events, or whether an individual experienced more than one event.
No serious adverse-event terms are listed. The death entry is null; a death count is therefore not reported in the registry record, rather than being presented as a reported zero. The listed counts do not describe the type, duration, or clinical course of the other adverse events, and the registry record does not report whether any led to a dose decrease or hold.
These safety entries should be read as descriptive observations in a small, single-group study. Without a comparator group, they do not show how the observed counts compare with what would have occurred without ruxolitinib or with another treatment. They also do not provide a complete characterization of risk. The registry record does not report exposure-adjusted rates, detailed event narratives, or a causal assessment attributing events to treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed, open-label, single-group phase 2 pilot study evaluated ruxolitinib in alopecia areata, with change in SALT score designated as the primary outcome. It describes the score’s construction, its range from 0 to 100, and assessments planned at baseline, 3 months, and 6 months. It also lists enrollment of 12 participants and provides adverse-event counts.
It does not report the primary outcome values. No mean SALT scores, standard deviations, changes, confidence intervals, or p-values are available in the registry record. That means the direction and size of any observed score change, and the precision of any estimate, cannot be determined from this information. No conclusion about whether the outcome changed can be drawn from the endpoint description alone.
Even if within-group changes were reported, the single-group design would support a descriptive account of change during the study rather than a controlled estimate of treatment effect. Without a comparator, differences over time could reflect treatment, natural fluctuation, other influences, or measurement variation. Open-label conduct adds a further consideration because treatment knowledge may influence behavior or assessment; whether SALT assessors were masked is not reported in the registry record.
The safety entries show 0 serious adverse events among 12 listed at risk and 9 participants with other adverse events among 12 listed at risk. These are registry counts, not a comparison of risks between treatment groups. A death count is not reported in the registry record. The entries do not provide enough detail to characterize event types, timing, or attribution.
Accordingly, the most defensible reading is limited to what the registry records: the study design, intended intervention, planned endpoint, and listed safety counts. It does not provide the primary outcome results needed to characterize observed SALT-score change or its uncertainty. The study’s pilot and uncontrolled nature would also require careful qualification of any within-group outcome pattern.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined endpoint and its results are both necessary for statistical interpretation. The registry specifies SALT score, explains how scalp areas contribute to the score, gives the score range, and identifies assessment times. That information makes the intended measurement understandable. But without the observed means, variability, and change estimates, readers cannot determine what happened to the measured outcome or how precisely it was estimated.
The design also shows the distinction between describing change and estimating a comparative effect. A single-group study can document outcomes over time among enrolled participants. It cannot provide the same causal contrast as a randomized controlled comparison, because there is no concurrent group showing what might have happened without the intervention. This is a central issue when interpreting before-and-after results in clinical research.
The registry’s planning assumptions offer another statistical lesson. Sample-size calculations depend on assumptions about the effect to detect, allocation, significance level, and analysis method. The listed illustrations assume equal allocation between two arms, while the trial is single-group. Planning calculations are not outcomes and should not be mistaken for observed power or evidence that a treatment effect was demonstrated. The registry record does not report their numerical values.
Finally, reporting of analysis populations, missing data, and outcome uncertainty is important even in a pilot study. Those details help readers judge who contributed data, how incomplete follow-up was handled, and how much confidence to place in an estimate. They are not reported in the registry record for the primary outcome. The record therefore provides useful design and measurement information but insufficient outcome reporting for a statistical assessment of SALT-score change.
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 most important trial-specific limitation in the registry record is the absence of primary outcome results. Although change in SALT score is identified as the primary endpoint and its assessment times are specified, no scores, changes, variability estimates, or inferential results are reported. The effectiveness question therefore cannot be evaluated from the registry outcome information.
The study is single-group, open-label, and has no randomized allocation or control group. Any reported before-and-after change would not, on its own, distinguish a treatment-related effect from the disease’s natural course, other influences, or measurement variation. The registry record does not state whether SALT assessments were masked, leaving that aspect of outcome measurement unclear.
Enrollment was 12, limiting the amount of participant-level information available for describing variability and assessing uncommon events. The registry record does not report the number of participants with outcome measurements at each scheduled assessment, the amount or pattern of missing data, or how missing measurements were handled. It also does not report the analysis population, adherence, actual dose modifications, or reasons for treatment discontinuation.
The registry does not report a statistical analysis plan, sample-size justification, prespecified primary comparison across follow-up times, multiplicity approach, or a threshold for an important SALT-score change. It lists a secondary outcome but does not report its analysis. Safety reporting is also limited: the record gives participant counts but no event descriptions or timing, and a death count is not reported in the registry record. These limitations restrict interpretation of both outcome patterns and safety observations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pilot Study to Evaluate the Efficacy of Ruxolitinib in Alopecia Areata — NCT01950780.
- Registry record last updated 2019-05-07; status last verified 2019-04.
- Results first posted 2019-05-07.
- 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 describes a completed phase 2, open-label, single-group pilot study sponsored by Columbia University, with 12 participants enrolled. The intervention was oral ruxolitinib, described as 20mg twice daily for 12 to 24 weeks, with possible dose decrease or hold for adverse effects. The primary endpoint was change in SALT score at baseline, 3 months, and 6 months.
The primary outcome results are not reported in the registry record. For safety, 0 of 12 participants at risk were listed as affected by serious adverse events, and 9 of 12 were listed as affected by other adverse events. A death count is not reported in the registry record. The single-group design permits descriptive observation but does not provide a controlled treatment comparison.