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 Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus (NCT01313923), a na early/phase 1 study. The primary outcome is Improvement of ABSIS Score While Reducing Steroid Dosage.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01313923 |
| Phase | EARLY/Phase 1 |
| Condition | Pemphigus |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of California, Irvine |
| Results posted | Yes |
2. Clinical Question
Population
The intended participants were people with stable pemphigus vulgaris who were already receiving prednisone. The registry lists pemphigus as the condition and an enrollment of 3, but says the primary outcome was not assessed because no participant completed any study visits. The registry does not report participant characteristics beyond the eligibility description.
Intervention
The intervention was oral sirolimus, also identified as rapamycin. The planned approach was to increase the sirolimus dose while decreasing prednisone, with disease state monitored during that process. The registry record does not report delivered doses, duration of exposure, adherence, or participant-level treatment histories.
Comparator
This was a single-group study with no masking and no randomized comparator. Prednisone reduction was part of the planned treatment approach, not a separate control arm. The registry record therefore does not support a like-for-like comparison between sirolimus and another treatment or between sirolimus and a control group.
Primary question
The primary question was whether disease control, measured with the Autoimmune Bullous Skin Disorder Intensity Score (ABSIS), could be maintained or improved while steroid dosage was reduced. The planned timeframe was described as an expected 24 months. The study was terminated early by the investigator, and the registry says no participant completed any visits. Consequently, the primary question was not answered by reported outcome data.
3. Trial Design
The registry record describes an early phase 1, single-group study sponsored by the University of California, Irvine. Allocation is listed as not applicable, and masking is listed as none. The study is not a randomized comparison, and its arm description does not describe a concurrent control group. Results from this design, had they been collected, would principally have described the course of participants receiving the planned regimen rather than isolated a treatment effect against a comparator.
The intended participants had stable pemphigus vulgaris and were already taking prednisone. They were to begin oral sirolimus, increase its dose, and decrease prednisone while their disease state was monitored. This treatment sequence is the planned intervention described in the registry; the record does not state how much treatment was actually delivered to any participant.
The listed enrollment is 3. The study start is recorded as 2011-02, and primary completion as 2014-10. Its status is terminated. The primary outcome description says the investigator terminated the study early and that no participant completed any study visits. The expected outcome timeline of 24 months is therefore a planned timeframe, not evidence that follow-up continued for that duration.
The record lists two outcome measures, but the named secondary outcome is only “Statistical Measures,” without a defined clinical measure or reported result. It lists zero analyses. The registry does not report the detailed protocol, a statistical analysis plan, the rationale for the enrollment target, or whether any analysis was planned at interim stages. Such details cannot be inferred from the early phase designation or the single-group design.
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.
Before assignment: Only major consideration for the pre-assignment is to exclude any patient with underlying immunodeficiency or hematological disorder. Investigational product itself can suppress hematopoiesis and is an immuno-modulator. All three patients screened were enrolled in the study.
Overall Study
| Stage | Sirolimus |
|---|---|
| STARTED | 3 |
| COMPLETED | 0 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Sirolimus |
|---|---|
| Physician Decision | 3 |
Analysis population for the primary outcome, as defined in the registry: This outcome was not assessed because no participant completed any visits of the study
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 | Sirolimus |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 59 (57 to 60) |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Improvement of ABSIS Score While Reducing Steroid Dosage, measured over Expected time line 24 months.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was titled “Improvement of ABSIS Score While Reducing Steroid Dosage.” ABSIS means Autoimmune Bullous Skin Disorder Intensity Score. As described in the registry, disease severity was to be measured with ABSIS, and improvement in disease control was defined as maintaining or improving the score while steroid dosage was reduced. The endpoint thus joined a disease-severity measure with a change in prednisone use; it was not simply a measure of sirolimus exposure or steroid reduction alone.
In general, a clinical endpoint makes a study question observable by specifying what is measured and how a result is interpreted. A disease-activity score can provide a structured way to track the condition over time. Considering the score alongside a reduction in steroid dosage was intended to describe whether the planned treatment transition could occur without worsening measured disease control. The registry does not report the scoring schedule, the precise rule for determining improvement, the required extent of steroid reduction, or how repeated measurements were to be combined.
The stated timeframe was an expected 24 months. However, the registry says the endpoint was not assessed because no participant completed any study visits. No ABSIS values, steroid-dose results, or endpoint estimates are reported. The endpoint therefore describes the question the study intended to assess, not an observed finding.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the primary outcome was not assessed. The registry states that the study was terminated early by the investigator and that no participant completed any visits. It reports no ABSIS measurements or steroid-reduction results, and it lists zero analyses. There is therefore no observed primary endpoint result from which to estimate the course of disease control during the planned sirolimus and prednisone regimen.
This is not a result showing that ABSIS improved, worsened, or remained stable. It is also not a result demonstrating that steroid dosage was reduced or that such a reduction was achieved without a change in disease control. Those conclusions would require measurements that the registry says were not collected through completed visits. The enrollment listing of 3 does not change that interpretation: enrollment is recorded, but the outcome description explicitly says no participant completed any study visits.
There is no reported treatment-effect estimate, uncertainty interval, or hypothesis test for the primary outcome. In general, an estimate describes the observed effect according to a defined analysis, while a confidence interval expresses the precision of that estimate under the analysis assumptions. Neither can be interpreted for the primary endpoint when there are no endpoint observations. The registry record also does not report a prespecified clinical threshold for a meaningful ABSIS change.
The study had a single group, no masking, and no randomized comparator. Even if measurements had been available, a change observed over time in this design would not by itself separate the effect of sirolimus from other explanations, such as the course of the disease or changes accompanying prednisone reduction. The record does not support a controlled comparison, and the listed design should not be interpreted as one.
The adverse-event record lists the sirolimus group with zero at risk and zero affected for serious events, and zero at risk and zero affected for other events. Deaths are recorded as null, and no serious-event terms are listed. These entries do not establish that treatment was free of adverse events: with zero participants recorded as at risk, they provide no meaningful description of safety during exposure. The record does not report participant-level exposure or an adverse-event analysis.
Planning illustrations based on an observed effect are not observed power. In this case, the primary endpoint has no reported observations from which an empirical effect can be estimated. Any planning quantity should therefore be read as a hypothetical illustration, not as evidence about the study's achieved precision, its ability to detect an effect, or the clinical result.
9. Statistical Methods Explained
What can a single-group study estimate?
In general, a single-group study can describe outcomes among participants who receive the study regimen, provided measurements are collected and the analysis is defined. It does not directly compare outcomes under that regimen with outcomes under a concurrent control. Here, the registry lists a single-group model, allocation as not applicable, and no masking. It does not report primary outcome observations or analyses, so even a descriptive estimate of change is unavailable.
A before-and-after description, if it had been collected, would compare measurements over time in the same treatment group. Such a comparison can show a temporal pattern, but it cannot by itself establish that the intervention caused that pattern. Changes in disease activity, prednisone use, and other factors could coincide with treatment. A randomized comparator is one general way clinical studies address some competing explanations, but this record does not describe such a comparison. No causal effect can be recovered from absent measurements.
What does the endpoint require?
The registry defines improvement in disease control through maintenance or improvement of ABSIS while steroid dosage is reduced. This requires both disease-severity information and a record of steroid dosage over the relevant period. In general, if either component is missing, the combined clinical question cannot be evaluated as written. The registry says the outcome was not assessed because no participant completed any visits; it supplies no scores, dosage trajectories, or endpoint classification.
The registry gives an expected timeframe of 24 months, but does not report the planned visit schedule, measurement frequency, or endpoint assessment rules. These details matter because repeated measurements can be summarized in different ways, and the interpretation can depend on when measurements are taken and how missing assessments are handled. The record does not state whether the endpoint would have used a final measurement, a change from baseline, a pattern across visits, or another summary.
How should statistical significance assumptions be read?
In general, a significance level is a design or analysis convention used to control the probability of a specified type of false-positive conclusion under a statistical model. The planning assumptions listed in the registry material use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. Those are assumptions for a planning illustration; they are not evidence that this single-group study used a two-arm design or carried out a hypothesis test. The trial record itself lists no analyses.
A normal approximation is a way to estimate a sampling distribution using a normal model. Whether such an approximation is suitable depends on the data and analysis, among other factors. The registry does not report an analysis dataset or an endpoint estimate with which to assess its suitability. Equal allocation between two arms is likewise a planning assumption, not a description of the actual single-group study.
What do the planning illustrations mean?
In general, a sample-size calculation asks how much information a future study might need under stated assumptions about the effect to detect, the error rate, allocation, and statistical model. The planning assumptions describe the effect to detect as a stated fraction of an observed effect. They also distinguish participant counts per arm for non-time-to-event outcomes from total event counts for time-to-event outcomes. These are planning conventions, not observed trial outcomes.
Because no participant completed a study visit and no primary result is reported, there is no observed ABSIS effect in the registry record to interpret as the empirical basis for a planning calculation. Any such illustration cannot be treated as a trial-derived estimate of the effect or as an assessment of the study's achieved power. Sample-size illustrations are not observed power. They cannot supply evidence about efficacy, precision, or the number of participants that would be needed under a different design unless the assumptions and effect target are independently justified.
The stated planning setup also assumes two arms and equal allocation, whereas the registered design is single-group and has no randomized comparator. This makes it especially important to read the planning setup as hypothetical rather than as a description of the study's design. The registry does not report a trial-specific sample-size justification or the assumptions used to choose the listed enrollment.
What can be said about uncertainty and safety?
A confidence interval is a general way to express the uncertainty around an estimate under a specified statistical method. It does not remedy missing outcome data: without a reported ABSIS estimate, there is no primary endpoint interval to interpret. The registry does not report a confidence interval, standard error, or other measure of primary-outcome precision.
The adverse-event entries list zero participants at risk and zero affected in the sirolimus group for both serious and other events; deaths are null, and no serious-event terms are listed. In general, adverse-event counts need an interpretable exposure population and observation period. The zero at-risk entries mean these counts do not characterize event frequency among exposed participants. They should not be interpreted as evidence of no risk or as a comparative safety result.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures, but its secondary outcome is named only “Statistical Measures.” It does not identify a specific secondary clinical endpoint or report results for that entry. The primary outcome is also unassessed, and the record lists zero analyses. Consequently, there are no reported endpoint estimates or hypothesis tests to evaluate as a family of statistical comparisons.
Multiplicity refers, in general, to the interpretive issue that can arise when several outcomes, time points, subgroups, or hypotheses are examined. A prespecified analysis plan can explain which outcomes are primary, how tests are prioritized, and whether any adjustment or other strategy is used. The registry record does not report a multiplicity strategy, a detailed statistical analysis plan, or whether analyses of multiple measures or time points were planned. It is not possible to determine from the record how multiplicity would have been addressed.
Missing-data methods are also not reported in the registry record. In general, a missing-data plan describes how incomplete measurements are handled and what assumptions support the analysis. The primary outcome was not assessed because no participant completed any study visits, so the record does not offer observed endpoint data for an imputation or missingness analysis. It also does not report whether the planned analysis would have used imputation, a complete-case approach, or another method. None should be presumed.
The record does not define an analysis population, such as a population based on enrollment, treatment initiation, or completion of specified assessments. The enrollment listing of 3 is not an analysis population, and the statement that no participant completed visits precludes interpreting it as a group with observed primary outcomes. The registry does not describe treatment exposure or participant-level inclusion in any analysis. Thus, no analysis population can be reconstructed from the reported information.
11. What the Result Does, and Does Not, Mean
The result of this registry record is a lack of assessable primary outcome data, not a positive or negative estimate of the regimen's effect. The study intended to assess whether ABSIS could be maintained or improved while prednisone dosage was reduced during sirolimus treatment. The record says no participant completed any study visits and that the endpoint was not assessed. It therefore gives no observed evidence about change in disease severity or steroid dosage.
That distinction is important. An absent result is not equivalent to a finding of no change, and it is not evidence that the planned treatment did or did not affect disease control. It also does not establish that participants could or could not reduce prednisone. No scores, dosage measurements, treatment-effect estimates, or uncertainty intervals are reported. The study's early termination explains why the registry gives no primary result, but does not supply the missing clinical measurements.
The record also cannot establish comparative effectiveness. The design was single-group, with allocation listed as not applicable and masking listed as none. There was no randomized control comparison in the registry description. Even a measured change in a single group would require cautious interpretation because the design alone cannot distinguish the intervention from other influences on outcomes over time.
The adverse-event entries should be read narrowly. They list zero at risk and zero affected in the sirolimus group, with deaths recorded as null. Since the group is recorded as having zero participants at risk, these counts do not provide an interpretable estimate of safety during treatment exposure. They should not be taken to mean that no adverse effects occurred in an exposed population.
In general, a small or incomplete study may contribute information about feasibility only when the relevant participation, treatment-delivery, or follow-up data are reported. This registry does not report completed visits, delivered doses, adherence, or a feasibility analysis. The enrollment listing alone does not answer those questions. The appropriate conclusion is limited: the planned clinical question remains unanswered by the results in this record.
12. Why This Trial Matters Statistically
This study illustrates a distinction central to clinical biostatistics: a registered objective is not the same as an analyzed outcome. The endpoint was framed around maintaining or improving a disease score while reducing steroid dosage, but the record states that it was not assessed because no participant completed any visits. Without observed measurements, there is no effect estimate to interpret, no precision to quantify, and no statistical test to support a conclusion about the clinical question.
The record also makes clear why design labels matter. A single-group study can be appropriate for descriptive or early-stage questions, but it does not furnish a randomized comparison. The planned sirolimus titration and prednisone reduction are part of the same treatment sequence, not separate experimental and control conditions. Any change, if measured, would need interpretation within those design limits. Here, no such change is reported.
It also demonstrates why enrollment, exposure, follow-up, and analysis are distinct concepts. The registry lists an enrollment of 3, while the outcome description says no participant completed a visit. Those details should not be collapsed into a claim that the primary endpoint was observed in the enrolled group. The adverse-event record's zero at-risk counts likewise cannot be treated as safety evidence for participants exposed to the intervention.
Finally, planning calculations need an interpretable effect target and assumptions that match the design being planned. The listed planning assumptions describe a hypothetical two-arm setup, while the registered study is single-group; the primary outcome has no reported observations. This is a useful reminder that sample-size illustrations are planning tools, not trial results or observed power. Their meaning depends on the assumptions and the availability of a defensible effect target.
13. 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 consequential limitation is that the study was terminated early and the registry says no participant completed any study visits. The primary endpoint was consequently not assessed. There are no reported ABSIS observations, prednisone-dose trajectories, or endpoint estimates. The registry lists an enrollment of 3, but that figure does not indicate completed follow-up or an analyzable primary outcome.
The design offers no concurrent control group. It is described as single-group, with no masking and allocation listed as not applicable. Accordingly, the registry cannot support a randomized treatment comparison. The intended intervention also involved increasing sirolimus while reducing prednisone, so the treatment sequence would not isolate the contribution of either component even if outcome data were available.
Important details are not reported in the registry record, including actual treatment exposure, dosing, adherence, participant characteristics beyond the eligibility description, visit schedule, baseline measures, and the rule used to classify endpoint improvement. The record does not report a trial-specific sample-size justification, statistical analysis plan, missing-data approach, multiplicity strategy, or analysis populations. No such details should be inferred.
Safety interpretation is limited as well. The adverse-event listing records zero participants at risk and zero affected for serious and other events, while deaths are null and no serious-event terms are listed. These entries do not describe the frequency of events in an exposed participant population. The record also does not report an adverse-event analysis or exposure duration.
The listed expected timeframe of 24 months is not evidence of completed follow-up, given the report that no participant completed any visits. Together, the early termination, absent endpoint data, lack of comparator, and missing analysis details prevent assessment of efficacy, precision, and safety from the posted results.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus — NCT01313923.
- Registry record last updated 2017-06-09; status last verified 2017-05.
- Results first posted 2016-03-25.
- 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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17. Record Summary
The registry record describes NCT01313923, “Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus,” sponsored by the University of California, Irvine. It is listed as early phase 1, single-group, with no masking and allocation not applicable. The registered enrollment is 3. The study started in 2011-02, primary completion is recorded as 2014-10, and status is terminated.
The planned intervention was oral sirolimus in people with stable pemphigus vulgaris already receiving prednisone. Sirolimus was to be increased while prednisone was decreased. The primary endpoint concerned maintaining or improving ABSIS while reducing steroid dosage, with an expected timeframe of 24 months.
The registry states that the investigator terminated the study early and that no participant completed any visits. The primary endpoint was not assessed, and zero analyses are listed. The record therefore reports no primary outcome estimate or interpretable comparison. Adverse-event entries list zero at risk and zero affected, with deaths recorded as null; these entries do not establish safety during exposure.