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 A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis (NCT01140503), a na na study. The primary outcome is The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01140503 |
| Phase | NA |
| Condition | Dermatomyositis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Stanford University |
| Results posted | Yes |
2. Clinical Question
Population
Patients with dermatomyositis and skin involvement were the intended study population. The registry record lists dermatomyositis as the condition and an enrollment of 5. Further eligibility details, including the clinical criteria used to define the skin disease, are not reported in the registry record.
Intervention
The intervention was oral apremilast. The arm description specifies apremilast 20mg bid. The study was open-label, so treatment masking is listed as none.
Comparator
There was no separate comparator group. The registry record identifies a single-group model and does not describe a randomized allocation. Consequently, the study does not directly compare apremilast with placebo, usual care, or another treatment.
Primary question
The primary endpoint concerned safety: the number of adverse events and serious adverse events during 12 weeks of therapy and 4 weeks of follow-up. Efficacy was also listed as a secondary focus, including CDASI-activity outcomes at 12 weeks, but efficacy results are not reported in the registry record.
The central question was whether apremilast could be evaluated for safety and efficacy in patients with cutaneous dermatomyositis within an open-label, single-group study. The design can describe outcomes observed during treatment and follow-up in the enrolled group. Without a comparator, however, it cannot establish how those outcomes would differ under another treatment or without apremilast. That distinction matters especially for efficacy: changes over time can reflect treatment, the natural course of the condition, other care, measurement variability, or other influences. The record does not report enough efficacy results to characterize changes in CDASI-activity. Nor does it provide details of eligibility or analysis populations that would define how broadly these observations apply.
3. Trial Design
This registry record describes an open-label study of apremilast for cutaneous disease in patients with dermatomyositis. The study model is single-group, the allocation field is listed as NA, and masking is listed as none. The arm description specifies apremilast 20mg bid. Stanford University is identified as the sponsor. The record lists enrollment of 5 and status as terminated. The phase is listed as NA.
The planned primary endpoint was safety, measured as the number of adverse events and serious adverse events during 12 weeks of therapy and 4 weeks of follow-up. The endpoint timeframe is described as 16 weeks. Secondary outcomes concerned efficacy at 12 weeks: the number of participants with a 30% decrease in CDASI-a score, and mean change in CDASI-activity. The registry record does not report numerical results for those secondary outcomes.
A single-group study can document what was observed among participants receiving the intervention. It does not create a concurrent control group, and the record does not describe randomization. The absence of a comparator limits causal interpretation: observed outcomes cannot be separated from changes that might have occurred for other reasons. Open-label treatment also means participants and study personnel were not masked to treatment assignment, which can affect reporting or assessment, depending on the outcome and its measurement process. The registry record does not report further details about outcome assessment procedures.
The record lists 3 outcome measures and 0 analyses. It does not report the protocol’s statistical analysis plan, reasons for termination, recruitment progress, or whether all intended outcome assessments were completed. The start is recorded as 2010-02 and primary completion as 2011-08. Those registry fields provide context for the study record, but they do not explain why the study ended or establish the completeness of follow-up.
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 | Apremilast |
|---|---|
| STARTED | 5 |
| COMPLETED | 4 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Apremilast |
|---|---|
| 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 | Apremilast |
|---|---|---|
| Participants analysed | 5 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 5 |
| Sex: Female, Male: Male | 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 | 5 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Physician Global Assessment of Skin Activity (VAS) | units on a scale | 3.8 (SD 0.98) |
| Physician Global Assessment of Skin Activity (Likert) | units on a scale | 2 (SD 0) |
| CDASI Activity Score | units on a scale | 31 (SD 6.8) |
| Manual Muscle Testing (MMT-8) score | units on a scale | 147 (SD 4.21) |
| Patient Pruritis Scale (VAS) | units on a scale | 2.9 (SD 2.40) |
| Dermatology Life Quality Index (DLQI) | units on a scale | 9 (SD 3.89) |
6. Primary Endpoint
The registry lists the primary outcome as The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup., measured over 16 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | adverse events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was a safety measure: the number of adverse events and serious adverse events occurring during 12 weeks of therapy and 4 weeks of follow-up. The registry record gives the endpoint timeframe as 16 weeks. Safety endpoints are used to describe unwanted events observed while participants receive an intervention and during a specified follow-up period. Serious events are distinguished from other adverse events because seriousness is a separate clinical and regulatory classification; it is not simply a measure of how often an event occurs.
For this study, the endpoint’s stated focus is the number of events. The posted safety summary, however, reports participants affected and participants at risk. Those are participant-level counts, which are not necessarily the same as the total number of events: a participant may experience more than one event. The registry record does not provide the total event count, event-level descriptions, or the criteria used to classify events. It therefore supports a limited account of how many participants were listed as affected, but not a complete reconstruction of the primary endpoint as worded.
In general, safety summaries help describe what was observed in the study group over the period monitored. They do not by themselves establish whether events were caused by the intervention. In a single-group, open-label study, interpretation also lacks a concurrent comparison that could help contextualize event frequency. Safety findings should therefore be read as descriptive observations from this group, not as a comparative estimate of risk.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety summary lists 5 participants at risk for serious adverse events and 0 affected. For other adverse events, it lists 5 participants at risk and 5 affected. These figures describe participant counts, not necessarily the total number of individual events. The registry record does not report event terms, the total number of events, or a death count. The serious adverse event terms list is empty, which does not supply additional detail about event-level ascertainment.
The figures should be interpreted as descriptive observations from a small, single-group study. The record lists enrollment of 5. With no comparator group, the observed safety experience cannot be contrasted with a concurrent group receiving another treatment or no study intervention. It also cannot establish whether any event was caused by apremilast. The open-label design means treatment masking was absent, although the registry record does not describe how this affected event reporting or assessment.
The primary endpoint was framed as the number of adverse events and serious adverse events over 12 weeks of therapy and 4 weeks of follow-up. The posted safety summary instead gives the number of participants affected. Because a participant can have more than one event, participant counts do not necessarily reveal the endpoint’s total event count. The record does not state whether the participant-level figures cover the entire planned timeframe for every participant, or how missing safety observations were handled. Thus, the counts are useful as a narrow description of the recorded safety summary, but they do not resolve those details.
The secondary outcomes were efficacy measures involving CDASI-activity at 12 weeks. The record names a participant response defined by a 30% decrease in CDASI-a score and a mean change in CDASI-activity. It does not report the observed values, estimates of uncertainty, or analyses for those outcomes. Accordingly, no efficacy conclusion can be drawn from the information in the registry record.
More generally, a confidence interval describes a range of estimates compatible with the data under a specified statistical procedure. It can communicate precision as well as the direction and scale of an estimate. The registry record does not report confidence intervals for these outcomes. With limited participant-level information and no comparison group, the posted safety figures provide little basis for estimating comparative effects or characterizing uncertainty beyond the counts themselves. No inference about treatment benefit or lack of benefit follows from the absence of reported efficacy results.
The status is listed as terminated, but the reason for termination is not reported in the registry record. Termination alone does not establish that the study stopped for safety, lack of efficacy, recruitment difficulty, or any other particular reason. The record also does not report whether the intended follow-up was completed for all participants. These omissions constrain interpretation of both the safety counts and the unreported efficacy outcomes.
9. Statistical Methods Explained
Statistical methods determine how observations are summarized and what kinds of conclusions can be supported. The registry record lists 0 analyses and does not report a statistical analysis plan. As a result, the planned analytic approach, any adjustment strategy, and the handling of incomplete observations are not reported in the registry record. The endpoint descriptions alone do not establish which statistical procedures were intended or used.
What does a single-group design estimate?
In general, a single-group study describes outcomes observed among participants who receive the intervention. It does not directly estimate a treatment contrast against a concurrent control group. A treatment contrast requires outcomes under comparison conditions; without such a group, differences between what happened and what might have happened otherwise cannot be measured directly. This is why the design can provide descriptive safety information while offering limited support for causal claims about treatment effects.
Here, the registry record identifies a single-group model, no masking, and no randomized allocation. The posted safety summary can therefore be read as a description of the group’s recorded experience. It cannot show whether the frequency of events was higher or lower than it would have been under another condition. For the secondary efficacy outcomes, the record does not report results that could be assessed even descriptively.
Why distinguish participants from events?
Participant counts and event counts answer different questions. A participant-level count indicates how many people experienced at least one event of a specified category. An event count includes repeated events in the same participant if they are counted separately. General safety reporting may include either or both, but the endpoint’s wording and the reported statistic need to be distinguished. In this record, the endpoint is described as a number of adverse events, while the safety summary gives the number of participants affected. The total event count is not reported in the registry record.
Likewise, serious adverse events are not interchangeable with all adverse events. Seriousness is a classification, while severity describes intensity; the registry record does not give event-level details that would permit further characterization. The reported count of participants affected by serious adverse events is a descriptive statistic, not a comparative risk estimate.
What does precision mean?
General statistical interpretation considers both an estimate and its uncertainty. A confidence interval is a way to express uncertainty around an estimate under a stated model and procedure. Narrower intervals generally indicate greater precision, whereas wider intervals indicate less precision. The record does not report confidence intervals or other uncertainty intervals for the safety or efficacy outcomes. It is therefore not possible to use the registry record to assess the precision of an estimated treatment effect.
In general, sparse observations can make estimates unstable: a small change in observed outcomes can materially change a summary. That consideration is relevant to interpreting this study’s limited enrollment, but it does not justify calculating an unreported effect or interval. The appropriate reading remains descriptive, with the limits of the available counts made explicit.
What is over-dispersion, and is it relevant?
General count-data methods sometimes assume that counts vary according to a particular probability model. Over-dispersion means that observed variation is greater than the model assumes. When present, it can make standard errors too small and intervals too narrow if the model does not account for the extra variation. Whether over-dispersion is relevant depends on the event data and analytic model. The registry record does not report event-level counts or a model for the primary endpoint, so it is not possible to assess over-dispersion or identify a method used to address it.
How should planning illustrations be read?
Sample-size planning is distinct from analysis of observed outcomes. The planning assumptions described for the posted illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; they describe the effect to detect as a stated fraction of the observed effect. The record characterizes these as planning illustrations, not as the study’s own design. The study itself is listed as single-group, so the two-arm equal-allocation assumption is not a description of its treatment structure. Such illustrations are hypothetical planning aids and do not represent observed power. Their numerical outputs and the observed effect they draw on are not reported in the registry record.
More broadly, statistical power is a design-stage probability under specified assumptions, not a score that can be inferred from whether a result is statistically significant. A planning calculation cannot repair the absence of a comparator or supply missing efficacy estimates. Nor does the registry record report a sample-size justification for this study. Its enrollment and the planning assumptions should not be conflated.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 3 outcome measures: the primary safety endpoint and secondary efficacy outcomes relating to CDASI-activity at 12 weeks. It does not report an analysis for these outcomes, and the count of analyses is 0. A prespecified statistical plan, including any strategy for handling multiple outcomes or repeated tests, is not reported in the registry record.
Multiplicity arises when several hypotheses or outcomes are assessed. In general, testing more hypotheses can increase the chance of at least one false-positive result if each is treated as a separate confirmatory test without an appropriate plan. This does not mean that every set of multiple outcomes requires the same adjustment; the approach depends on the scientific aims and analysis plan. Since no inferential results or multiplicity strategy are reported, the registry record does not allow readers to determine whether the secondary outcomes were intended to be exploratory or confirmatory, or how any multiplicity was addressed.
Missing-data methods and analysis populations are also not reported in the registry record. The record does not specify how participants with incomplete follow-up, missed assessments, or unavailable measurements were handled. It does not define whether analyses included all enrolled participants, participants who started treatment, or another population. The participant-level safety counts are reported, but the record does not describe completeness of observation across the stated timeframe or whether all participants contributed the same follow-up.
These omissions matter because the set of participants analyzed and the treatment of missing measurements can affect summaries and inferences. They do not authorize an assumption that data were complete, that a particular analysis population was used, or that missing outcomes were handled in a specific way. Those details are not reported in the registry record.
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 |
|---|---|---|---|
| Apremilast | 0 | 5 | 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 |
|---|---|
| Apremilast | 5 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Apremilast |
|---|---|---|
| headache | Nervous system disorders | 2 of 5 |
| Myalgia | Musculoskeletal and connective tissue disorders | 1 of 5 |
| Urinary Tract Infection | Infections and infestations | 1 of 5 |
| Bruising | Skin and subcutaneous tissue disorders | 1 of 5 |
| Indigestion | Gastrointestinal disorders | 1 of 5 |
| Back Pain | Musculoskeletal and connective tissue disorders | 1 of 5 |
| Migraine headache | Nervous system disorders | 1 of 5 |
| Upper respiratory tract infection | Infections and infestations | 1 of 5 |
| Left Knee Injury | Musculoskeletal and connective tissue disorders | 1 of 5 |
| Dizziness | Nervous system disorders | 1 of 5 |
The registry safety summary lists 0 participants affected by serious adverse events among 5 at risk. For other adverse events, it lists 5 affected among 5 at risk. These are participant-level figures. They do not necessarily equal the number of separate adverse events, because a participant can experience multiple events. The primary endpoint is worded as a count of adverse events and serious adverse events, but the total event count is not reported in the registry record.
The serious adverse event terms list contains no entries, and a death count is not reported in the registry record. The absence of listed serious event terms should not be extended into claims about unreported event categories or ascertainment details. The record does not state whether the safety figures include complete observation for every participant across 12 weeks of therapy and 4 weeks of follow-up.
These figures describe the recorded experience in this single group. They do not establish that the intervention caused or prevented any event, and they do not provide a comparison with another treatment. The small enrollment and lack of a comparator mean the counts should be read as limited descriptive safety information, not as a precise estimate of risk.
12. What the Result Does, and Does Not, Mean
The registry record documents an open-label, single-group study of apremilast for skin disease in patients with dermatomyositis. It identifies a safety endpoint covering 12 weeks of therapy and 4 weeks of follow-up, and lists secondary efficacy outcomes at 12 weeks. The posted safety summary records 0 participants affected by serious adverse events among 5 at risk, and 5 participants affected by other adverse events among 5 at risk.
These observations do not determine whether apremilast caused an event. Without a comparator, the study cannot directly distinguish outcomes associated with treatment from outcomes that might have occurred under a different condition. Participant counts also do not provide the total number of events. The record does not report the event-level details or total count needed to fully characterize the primary endpoint as worded.
The record does not report numerical findings for the secondary efficacy outcomes. It therefore does not establish the size, direction, or precision of any change in CDASI-activity, nor does it report how many participants met the stated response definition. The absence of these results should not be interpreted as evidence that no change occurred or that treatment was ineffective.
In general, a study can contribute descriptive information even when it cannot support a comparative conclusion. Here, that contribution is constrained by the limited enrollment, single-group design, open-label status, and gaps in reported analyses and outcome details. The study status is terminated, but the reason is not reported in the registry record; the status alone does not explain the study’s results or its termination.
The record also does not report the statistical plan, analysis populations, missing-data handling, confidence intervals, or efficacy estimates. Without those elements, readers cannot evaluate the precision or robustness of efficacy findings. The appropriate interpretation is narrow: the registry lists specific safety counts and planned outcome descriptions, but it does not provide enough evidence to infer a comparative effect or make a treatment recommendation.
13. Why This Trial Matters Statistically
Statistically, this study is useful for illustrating the difference between describing an observed group and estimating a comparative treatment effect. A single-group design can record events among participants receiving apremilast, but it lacks a concurrent comparison that would show how outcomes differ under another condition. The open-label design adds a further reason to keep conclusions descriptive when outcomes depend on reporting or assessment.
The record also illustrates why endpoint definitions and reported summaries need to be checked against each other. The primary endpoint is expressed as a number of adverse events, while the safety figures identify participants affected. Those summaries are related but are not interchangeable. A participant may experience more than one event, so participant counts alone do not recover the total event count.
Another statistical lesson is the importance of reporting uncertainty and analysis methods. The registry record does not report analyses, confidence intervals, missing-data handling, or analysis populations. Without those details, the precision and robustness of estimates cannot be assessed. The secondary outcomes are named, but their numerical results are not reported, so their descriptive or inferential contribution cannot be evaluated.
Finally, planning calculations must be separated from the design and results of the study. The planning assumptions describe a two-arm, equal-allocation illustration, whereas the registry lists a single-group study. Such planning illustrations are not evidence of observed power and do not create a comparison that the study did not have. This distinction helps prevent hypothetical design calculations from being mistaken for findings.
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 study’s single-group structure is a central limitation for interpretation. It has no concurrent comparator, and the registry does not describe randomized allocation. Consequently, the recorded outcomes cannot be directly contrasted with outcomes under another treatment or no study intervention. Open-label status, with masking listed as none, also means treatment assignment was not concealed from participants and study personnel.
The registry lists enrollment of 5, limiting the scope of the descriptive safety record. The safety summary reports participants affected rather than the total number of events specified in the primary endpoint. Event terms and the death count are not reported in the registry record. The record also does not clarify whether all participants completed observation across the stated treatment and follow-up periods.
Efficacy outcomes are described but their numerical results are not reported. This prevents assessment of the CDASI-activity response measure and mean change, including their magnitude and precision. The registry record lists 0 analyses and does not report an analysis plan, confidence intervals, missing-data methods, or analysis populations. The handling of incomplete follow-up and unavailable outcome measurements is therefore unknown.
The status is terminated, but the reason for termination is not reported in the registry record. The record does not state whether termination affected recruitment, follow-up, or the completeness of outcome reporting. No sample-size justification for the study is reported. Planning assumptions described for a two-arm illustration do not resolve these limitations or describe the study’s single-group design.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis — NCT01140503.
- Registry record last updated 2015-03-04; status last verified 2015-03.
- Results first posted 2015-03-04.
- 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 describes an open-label, single-group study of oral apremilast 20mg bid for cutaneous disease in patients with dermatomyositis. Stanford University is listed as sponsor, enrollment is 5, and the study status is terminated. The phase is listed as NA.
The primary endpoint concerns the number of adverse events and serious adverse events during 12 weeks of therapy and 4 weeks of follow-up. The safety summary lists 0 participants affected by serious adverse events among 5 at risk and 5 participants affected by other adverse events among 5 at risk. The total event count and event terms are not reported in the registry record.
Secondary outcomes concern CDASI-activity at 12 weeks, including a participant response defined by a 30% decrease and mean change. Numerical efficacy results, analyses, the statistical plan, and the reason for termination are not reported in the registry record. The single-group design supports descriptive observations but not a direct comparison with another treatment.