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 Pilot Study of Etanercept in Dermatomyositis (NCT00112385), a randomized phase 1 study. The primary outcome is Occurrence of at Least One Adverse Event.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00112385 |
| Phase | Phase 1 |
| Condition | Dermatomyositis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 16 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Brigham and Women's Hospital |
| Results posted | Yes |
2. Clinical Question
Population
Participants with dermatomyositis were eligible for this pilot study. The registry record reports an enrollment of 16 and identifies the primary outcome population as intention-to-treat. Further eligibility criteria and participant characteristics are not reported in the registry record.
Intervention
The intervention was etanercept, administered by subcutaneous injection once a week for 52 weeks. The study’s stated purpose was to obtain preliminary information about safety and tolerability, and to evaluate outcome measures for use in future trials.
Comparator
The comparator was placebo, administered by subcutaneous injection once a week for 52 weeks. Both arms were described as part of a randomized, double-masked, parallel study.
Primary question
The primary endpoint was the occurrence of at least one adverse event, assessed at each visit during the 12 month study. The registry describes adverse events using CTCAE version 3.0 and includes severity categories. The posted results do not give a single arm-level comparison for the primary endpoint, so the record does not establish a comparative estimate of adverse-event occurrence.
The study also aimed to assess prednisone safety and to examine the variability, reliability, and responsiveness of outcome measures recommended by IMACS. The secondary outcomes listed concern average prednisone dosage after Week 24 and average daily prednisone dose from baseline to Week 52. The specific prednisone dosing schedule and results for those outcomes are not reported in the registry record.
3. Trial Design
This was a Phase 1, randomized, double-masked, parallel-group pilot study sponsored by Brigham and Women's Hospital. It enrolled 16 participants with dermatomyositis and is listed as completed. The registry record gives a study start of 2006-03 and a primary completion of 2010-06. These dates describe the registered study timeline; they do not provide details about the timing of individual assessments or analysis decisions.
Participants were assigned to etanercept or placebo. The etanercept arm received weekly subcutaneous injections of 50 mg for 52 weeks. The placebo arm received weekly subcutaneous injections for 52 weeks. The registry does not describe the randomization sequence, allocation concealment, masking procedures, or the handling of treatment changes and discontinuations.
The primary outcome was occurrence of at least one adverse event, assessed at each visit during the 12 month study. Adverse events were assessed using CTCAE version 3.0, with mild, moderate, and severe categories described in relation to the CTCAE dictionary. The primary outcome population is identified as intention-to-treat. Although the registry lists 42 outcome measures, it does not give a single summarized primary comparison in the posted results.
The study was framed as preliminary work on safety, tolerability, and outcome-measure properties to inform design of future trials in inflammatory myopathies. It also included aims relating to prednisone and listed two prednisone dosage outcomes. The protocol details for those aims, including the proposed prednisone schedule, are not reported in the registry record. A detailed statistical analysis plan, sample-size justification, and information about interim analyses are also not reported in the registry record.
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: Most patient were initially ineligible because they were not new dermatomyositis patients. Due to slow recruitment, we modified the protocol. We allowed enrollment of refractory dermatomyositis patients receiving prednisone for greater than 2 months. These subjects could also receive second line agents, as long as they were on a stable dose.
Overall Study
| Stage | Etanercept | Placebo |
|---|---|---|
| STARTED | 11 | 5 |
| COMPLETED | 10 | 4 |
| NOT COMPLETED | 1 | 1 |
Reasons for not completing this period:
| Reason | Etanercept | Placebo |
|---|---|---|
| Lost to Follow-up | 1 | 0 |
| Withdrawal by Subject | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Intention- to -Treat (ITT)
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 | Etanercept | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 11 | 5 | 16 | |
| Age Categorical: < 18 years | participants | 0 | 0 | 0 |
| Age Categorical: Between 18 and 65 years | participants | 11 | 5 | 16 |
| Age Categorical: >=65 years | participants | 0 | 0 | 0 |
| Age Continuous | years | 43.36 (SD 14.8) | 44.2 (SD 11.9) | 43.6 (SD 13.39) |
| Sex: Female, Male: Female | Participants | 6 | 4 | 10 |
| Sex: Female, Male: Male | Participants | 5 | 1 | 6 |
| Region of Enrollment: Canada | participants | 0 | 1 | 1 |
| Region of Enrollment: United States | participants | 11 | 4 | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Occurrence of at Least One Adverse Event, measured over at each visit during the 12 month study.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the occurrence of at least one adverse event. The registry says adverse events were assessed at each visit during the 12 month study and classified using CTCAE version 3.0. Its descriptions distinguish mild events, for which intervention is not indicated, from moderate events, which may require minimal or local intervention and may limit activities of daily living, and severe events, which are medically significant and may involve hospitalization or disability.
Counting participants with an event can provide a direct description of how often an adverse event was reported in each arm. It is a binary outcome at the participant level: a participant either meets the event definition or does not. Such a count does not by itself describe the number of events experienced by each participant, their duration, or their timing. Those details are not reported for the primary endpoint.
Safety is central to the stated purpose of this pilot study, which sought preliminary information about etanercept and prednisone tolerability as well as information useful for planning future therapeutic trials. The endpoint is therefore relevant to the study's stated aims. However, the registry record does not present a single comparison for the primary endpoint across treatment arms, and it does not report an effect estimate or confidence interval for that comparison. The available safety counts should not be treated as a substitute for that missing primary-outcome summary.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small pilot study focused on safety, tolerability, and the performance of outcome measures. Its posted safety summaries list serious adverse events affecting 3 of 11 participants at risk in the etanercept arm and 3 of 5 participants at risk in the placebo arm. The record also lists other adverse-event counts affecting 11 of 11 participants at risk in the etanercept arm and 5 of 5 participants at risk in the placebo arm. These are separate reported summaries; the record does not explain whether or how the categories overlap. They should not be combined into a new count of participants with any adverse event.
The primary endpoint is occurrence of at least one adverse event, but the registry does not reduce its multiple categories or time points to a single arm-level comparison. No comparative effect estimate, confidence interval, or p-value for that endpoint is reported in the registry record. Consequently, the posted results do not quantify the precision of a treatment contrast or support a formal conclusion about between-arm differences in the primary endpoint.
The counts are descriptive and come from a study identified as a pilot. The difference in the number at risk between arms also means that the raw counts alone are not a direct comparison of event frequency. A statistical comparison would require a clearly defined outcome, a specified analysis population and method, and appropriate uncertainty estimates. Those elements are not reported for a single primary comparison.
The registry lists serious adverse-event terms with percentages, including Viral Gastroenteritis at 20.0%, Dermatomyositis Flare at 20.0%, Ovarian Cancer at 20.0%, Partner Pregnant at 9.1%, Urinary Tract Infection at 9.1%, and Fever at 9.1%. The denominators and the way these term percentages were calculated are not reported in the registry record. The percentages should therefore be read as registry-listed descriptors, not as comparative risks or rates.
The posted results are not evidence about efficacy: no primary clinical efficacy outcome is presented in the material described by this record. The study's stated goals were preliminary safety assessment and evaluation of outcome measures, not a definitive comparative test. Neither the absence of a reported comparative analysis nor the observed descriptive counts establish that one treatment is safer or less safe than the other. The interpretation is limited to what the registry reports and to the lack of a single primary-outcome analysis.
9. Statistical Methods Explained
The registry identifies the primary outcome as a participant-level event count: occurrence of at least one adverse event. It also indicates that the outcome was assessed at each visit and describes multiple severity categories. The posted results do not reduce these categories and time points to one comparison. That matters because a statistical analysis needs a clearly defined outcome rule: for example, whether the comparison concerns any event, a particular severity category, or an event within a specified assessment period. The registry record does not report a single rule and associated comparison for the primary endpoint.
General: What does a binary count describe?
A binary outcome classifies each participant according to whether a prespecified event occurred. Reporting affected participants alongside the number at risk gives a descriptive account of observed events. It does not, by itself, show how uncertain an arm-to-arm comparison is. A confidence interval is a range of values compatible with the data and the assumptions of the analysis; it communicates the precision of an estimate. No confidence interval for the primary treatment comparison is reported in the registry record.
General: Why does the analysis definition matter?
Repeated assessment can produce more than one event or more than one record for a participant. An analysis of participants who ever had an event differs from an analysis of event counts, timing, or severity. These approaches answer different questions and may require different statistical methods. The registry's primary endpoint wording refers to at least one adverse event, but the posted results do not provide a single comparison that resolves the listed categories and time points. The methods used to address repeated observations, if any, are not reported in the registry record.
General: What do uncertainty and over-dispersion mean?
General statistical interpretation requires separating the observed result from its uncertainty. A point estimate describes the observed comparison, while an uncertainty interval communicates how much precision the data support under the analysis assumptions. For count outcomes, over-dispersion means that variability exceeds what a chosen model expects. Accounting for it can affect uncertainty estimates. This is general statistical context; the registry does not report an over-dispersion assessment or a model for the primary outcome.
The registry lists an intention-to-treat population for the primary outcome. In general, intention-to-treat analyses aim to retain participants in the groups to which they were randomized, regardless of later treatment adherence. The registry does not provide the participant-level disposition or explain how missing outcome assessments were handled, so the operational details of the intention-to-treat analysis are not reported in the registry record.
The record lists 42 outcome measures, while no analyses are listed in the registry record. This does not establish that no analyses were conducted; it means that an analysis plan or set of reported comparisons is not available in the registry entry. The prespecified status of each outcome, the choice of any primary contrast, and any adjustment for multiple testing are not reported. When many outcomes or comparisons are considered, multiplicity can increase the chance of a chance finding if results are selected or interpreted without an appropriate plan. This is general statistical context, not a claim about analyses performed in this study.
The registry's sample-size assumptions describe planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. The stated effect to detect is a fraction of the observed effect. For time-to-event illustrations, the record describes total events using the Schoenfeld method; for other illustrations, it describes participants per arm. These are planning illustrations, not observed power and not the trial's own sample-size justification. The original sample-size rationale is not reported in the registry record. A planning calculation based on an observed effect is conditional on its assumptions and does not establish that a future study will achieve a particular result.
No inferential analysis for the primary endpoint is reported. The registry therefore supplies no reported test statistic, p-value, treatment-effect estimate, or confidence interval for that comparison. Without those results and the analysis details, readers should treat the listed counts as descriptive rather than as a completed statistical test of the primary question.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 42 outcome measures and identifies occurrence of at least one adverse event as the primary endpoint. However, the posted results do not give a single primary comparison across the endpoint's multiple categories or assessment times. The record does not report which comparisons were prespecified, whether a hierarchy of outcomes was used, or whether any adjustment for multiple testing was planned or performed. A multiplicity strategy is therefore not reported in the registry record.
The primary population is identified as intention-to-treat, but the participant-level analysis details are not reported. The registry does not describe missing visits, incomplete adverse-event information, withdrawals, or the method used to handle missing data. It also does not report sensitivity analyses that might examine whether conclusions change under different assumptions about missing outcomes.
These omissions limit interpretation of the posted counts. When multiple outcomes, categories, or time points are available, selective emphasis can affect how results are understood. Missing data can also affect a participant-level event summary, depending on how incomplete follow-up is treated. These are general statistical concerns; they do not establish that a particular bias occurred in this study.
The registry record does not report an interim-analysis plan, a detailed statistical analysis plan, or a sample-size justification. Without these details and a defined primary contrast, the listed results cannot be used to reconstruct the intended analysis or to assess whether the reported summaries follow a prespecified plan.
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 |
|---|---|---|---|
| Etanercept | 3 | 11 | 27.3% |
| Placebo | 3 | 5 | 60.0% |
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 |
|---|---|
| Etanercept | 11 of 11 |
| Placebo | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Etanercept | Placebo |
|---|---|---|---|
| Partner Pregnant | Pregnancy, puerperium and perinatal conditions | 1 of 11 | 0 of 5 |
| Urinary Tract Infection | Infections and infestations | 1 of 11 | 0 of 5 |
| Fever | General disorders | 1 of 11 | 0 of 5 |
| Post Herpetic Neuralgia | Nervous system disorders | 1 of 11 | 0 of 5 |
| Acute Psychosis | Psychiatric disorders | 1 of 11 | 0 of 5 |
| Viral Gastroenteritis | Infections and infestations | 0 of 11 | 1 of 5 |
| Dermatomyositis Flare | Nervous system disorders | 0 of 11 | 1 of 5 |
| Ovarian Cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 11 | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Etanercept | Placebo |
|---|---|---|---|
| Musculoskeletal Pains | Musculoskeletal and connective tissue disorders | 6 of 11 | 3 of 5 |
| Headache | Nervous system disorders | 3 of 11 | 4 of 5 |
| Diarrhea | Gastrointestinal disorders | 3 of 11 | 3 of 5 |
| Infection Upper Airway | Infections and infestations | 5 of 11 | 1 of 5 |
| Pruritus | Skin and subcutaneous tissue disorders | 5 of 11 | 1 of 5 |
| Worsening Skin Rash | Skin and subcutaneous tissue disorders | 5 of 11 | 1 of 5 |
| Nausea | Gastrointestinal disorders | 3 of 11 | 2 of 5 |
| Hypertension | Vascular disorders | 3 of 11 | 1 of 5 |
| Injection Site Reaction | General disorders | 2 of 11 | 2 of 5 |
| Neuropathy | Nervous system disorders | 3 of 11 | 1 of 5 |
Serious events with the highest arm-level rate: viral gastroenteritis (20.0%), dermatomyositis flare (20.0%), ovarian cancer (20.0%), partner pregnant (9.1%), urinary tract infection (9.1%).
The registry lists serious adverse events affecting 3 of 11 participants at risk in the etanercept arm and 3 of 5 participants at risk in the placebo arm. It separately lists other adverse events affecting 11 of 11 participants at risk in the etanercept arm and 5 of 5 participants at risk in the placebo arm. The registry does not define the relationship between these two summaries or state whether participants appear in both. They cannot be added together to obtain a count of participants with any adverse event.
Serious adverse-event terms are listed with percentages: Viral Gastroenteritis, Dermatomyositis Flare, and Ovarian Cancer at 20.0% each; Partner Pregnant, Urinary Tract Infection, and Fever at 9.1% each. The record does not report the denominators or calculation method for these term percentages, so they should not be interpreted as arm-specific risks. The death counts are not reported in the registry record.
These figures describe events recorded in the trial; they do not establish causation or provide a complete comparative safety analysis. The registry does not report a single primary-endpoint comparison, uncertainty intervals, or details about event overlap, timing, or exposure-adjusted rates.
12. What the Result Does, and Does Not, Mean
The record documents a randomized, double-masked pilot study of etanercept and placebo in dermatomyositis. Its stated purpose was to collect preliminary safety and tolerability information, assess prednisone safety, and examine outcome measures for future inflammatory-myopathy trials. The posted adverse-event counts give descriptive information for the two arms, while the listed serious-event terms provide additional event labels and percentages.
Those descriptions do not amount to a definitive comparative estimate. The registry does not provide a single analysis of the primary endpoint, a treatment-effect estimate, a confidence interval, or a p-value. The posted results also do not supply the denominators or calculation method for the serious-event term percentages. The record therefore does not show the precision of an arm-to-arm contrast or resolve whether the reported event categories overlap.
The study's pilot purpose is important context. Preliminary data may help inform the design and measurement choices of later research, but the registry record does not report a sample-size justification or a statistical plan that would establish the ability to detect a particular effect. The planning illustrations described in the record are not observed power and are not a replacement for the original design rationale.
No conclusion about efficacy follows from these safety summaries. Nor do they establish that etanercept is safer or less safe than placebo. The defensible interpretation is limited to the reported counts and the fact that the registry does not provide a unified primary-outcome comparison. Further details on endpoint definitions, analysis populations, missing observations, event overlap, and uncertainty would be needed for a fuller statistical assessment.
13. Why This Trial Matters Statistically
This trial is statistically relevant because it illustrates the role of pilot studies in evaluating both safety outcomes and the measurement tools intended for future research. The stated aims include assessing variability, reliability, and responsiveness of IMACS-recommended measures. Those properties can inform whether an outcome is suitable for a later trial and how it should be analyzed. The registry record does not report results for those measurement properties, so their findings cannot be evaluated from the posted information.
The study also highlights why a clearly defined estimand and analysis are needed for safety outcomes. The primary endpoint refers to at least one adverse event, while the registry describes assessment at each visit and multiple severity categories. A single comparison requires clarity about how those elements translate into an arm-level result. The posted results do not provide that unified comparison.
Small pilot studies can produce useful descriptive information, but descriptive counts should not be mistaken for a precise treatment contrast. Reporting a defined estimate alongside its uncertainty would help readers distinguish the observed data from what they can support statistically. The registry does not provide such an estimate for the primary endpoint.
Finally, the study's stated intention to inform future trials makes transparency about outcome selection, missing data, and sample-size rationale especially useful. Those details are not reported in the registry record. The available entry nevertheless makes the study's preliminary aims and basic randomized design clear, while showing where a later or fuller report would be needed to interpret the primary comparison.
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 main limitation for interpretation is that the registry does not report a single comparison for the primary endpoint, despite identifying occurrence of at least one adverse event as primary. It lists adverse-event summaries, but the relationship between serious and other events is not described. The listed serious-event term percentages lack reported denominators and a calculation method.
The registry record does not report confidence intervals, p-values, treatment-effect estimates, or the statistical method used for the primary outcome. It also does not report participant-level disposition, missing-data handling, event timing, or whether event counts reflect unique participants or repeated events. The primary outcome population is labeled intention-to-treat, but implementation details are not reported.
The record lists 42 outcome measures, but does not describe an analysis hierarchy, multiplicity adjustment, interim analyses, or a detailed statistical analysis plan. Results for the stated prednisone aims and for outcome-measure properties are not reported in the material described by the registry entry. The eligibility criteria, baseline characteristics, and specific prednisone schedule are not reported in the registry record.
These limitations prevent reconstruction of the intended primary analysis and constrain assessment of precision, missing-data effects, and selective reporting. They do not show that a particular analysis was inappropriate or that bias occurred; they mark information that is not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Pilot Study of Etanercept in Dermatomyositis — NCT00112385.
- Registry record last updated 2011-06-21; status last verified 2011-05.
- Results first posted 2011-06-21.
- 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
NCT00112385 is a completed Phase 1 randomized, double-masked, parallel pilot study of etanercept versus placebo in dermatomyositis, sponsored by Brigham and Women's Hospital. The registry reports enrollment of 16. Etanercept and placebo were administered by weekly subcutaneous injection for 52 weeks.
The primary endpoint was occurrence of at least one adverse event, assessed at each visit during the 12 month study using CTCAE version 3.0. The registry lists serious and other adverse-event counts, but does not report a single primary-outcome comparison or inferential analysis. It also lists prednisone dosage outcomes and states aims concerning safety and outcome-measure properties; corresponding results are not reported in the registry record.
The posted information is descriptive. Comparative estimates, uncertainty, missing-data methods, and the detailed statistical plan are not reported in the registry record.