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 Safety Study of Abatacept to Treat Refractory Sarcoidosis (NCT00739960), a na phase 2 study. The primary outcome is Safety of Abatacept in Progressive Pulmonary Sarcoidosis..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00739960 |
| Phase | Phase 2 |
| Condition | Sarcoidosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Chicago |
| Results posted | Yes |
2. Clinical Question
Population
People with progressive pulmonary sarcoidosis were the intended population. The registry describes the study as addressing refractory or progressive sarcoidosis, but eligibility criteria and the number screened are not reported in the registry record. The listed enrollment is 1.
Intervention
The intervention was abatacept, recorded as a drug. The study was open-label, so masking was listed as none. Dosing details and treatment duration are not reported in the registry record.
Comparator
There was no separate comparison group: the study model is single-group, and allocation is listed as not applicable. Outcomes therefore describe participants in the abatacept group, rather than a contrast between abatacept and a control treatment.
Primary question
The primary question concerned safety: whether participants experienced adverse events considered reasonably or probably related to abatacept. The primary outcome was a count of participants with such events, with timeframes of 24 weeks and 52 weeks. The registry also describes the study's purpose as assessing safety and effectiveness, but a comparative effectiveness question cannot be answered by this single-group design.
The registry identifies change from baseline in 6-minute walk distance as a secondary outcome. That measure concerns functional change over time; the record does not include an analysis result for it. With one listed enrollee and no comparator, the record can describe only the limited observations captured for this study. It cannot establish what would have happened to similar participants without abatacept, or separate treatment effects from the course of the disease, background care, or other influences.
3. Trial Design
This was a phase 2, prospective, open-label, single-group study of abatacept for progressive sarcoidosis. The registry identifies the sponsor as the University of Chicago. The study began in 2008-08, had a primary completion date of 2008-12, and is listed as terminated. Registry enrollment is 1. Allocation is not applicable, masking is none, and the model is single-group. These details identify a safety-oriented study without a randomized control arm or a masked comparison.
The design means that participants and investigators were not masked to treatment, and no concurrent control group is recorded. Open-label observation can be useful for describing events during treatment, but reports may be influenced by awareness of treatment and by investigators' judgments. Without a comparison group, an event observed during treatment cannot by itself be attributed to the drug. The primary outcome definition narrows the safety count to events the investigator considered reasonably or probably related, but that judgment does not create a controlled estimate of causation.
The registry says safety was selected as the primary outcome because the study was designed as a safety study. It also notes that symptom changes from baseline were considered possibly related to study drug, although unlikely. The record states that the study was discontinued due to funding withdrawal; the accompanying explanation is incomplete. Further details about the discontinuation process are not reported in the registry record.
Eligibility criteria, planned recruitment, treatment schedule, follow-up procedures, and the intended statistical analysis plan are not reported in the registry record. The listed primary outcome timeframes are 24 weeks and 52 weeks, but the record does not explain how these timepoints were to be handled for an enrolled participant or whether both were assessed. The listed enrollment of 1 is not evidence of the original recruitment target or the reason enrollment stopped.
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 | Abatacept |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Since this study was designed to be a safety study, we chose safety as the primary outcome. Any change of symptoms from baseline was considered possibly related to the study drug, although unlikely. The study was discontinued due to funding withdrawal caused by the principal investigator switching institutions.
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 | Open Label |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Safety of Abatacept in Progressive Pulmonary Sarcoidosis., measured over 24 weeks and 52 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participant adverse events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is a count of participants with adverse events considered by the investigator to be reasonably or probably related to abatacept. The record gives two timeframes, 24 weeks and 52 weeks. It describes safety as the primary outcome because the study was designed as a safety study. This endpoint focuses on the occurrence of potential treatment-related harm rather than on a change in disease activity or functional capacity.
Counting affected participants can make safety information easier to interpret than an unstructured list of event descriptions: it indicates how many people experienced an event under the endpoint definition. It is not necessarily a count of every event, since one person can experience more than one event. The endpoint's attribution requirement also matters. Investigator assessment of whether an event is reasonably or probably related to treatment is a judgment, not a randomized comparison or proof of causation.
Safety outcomes are important in a study of an intervention, particularly when the study's stated priority is safety. However, an interpretable safety estimate requires enough observation and clear reporting of who was assessed, for how long, and how events were defined and collected. The registry lists one enrollee and does not report an analysis result for the primary outcome. It therefore does not offer a precise estimate of the frequency of treatment-related adverse events, nor does it establish the absence of risks. The secondary outcome is change from baseline in 6-minute walk distance; it is distinct from the primary safety endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, open-label, single-group study and lists enrollment of 1. In the adverse-event information, 1 participant is listed as at risk for serious events and 0 as affected; 1 is listed as at risk for other events and 1 as affected. No serious adverse-event terms are listed. These are counts from a very limited observation base. They do not support a stable estimate of event frequency or a broad conclusion about safety.
The distinction between zero observed serious events and evidence of no serious risk is essential. A zero count means that no affected participant is recorded in that category for the observation captured; it does not show that the underlying risk is zero. With a single participant listed at risk, the data have little capacity to reveal uncommon events or variation between people. The registry's listed primary outcome also concerns events judged reasonably or probably related to treatment, while the adverse-event summary gives seriousness categories. The available information does not report a detailed event-level account that would permit those categories to be fully reconciled.
The count of 1 participant affected by other adverse events should likewise be read as a descriptive observation, not as an estimate that generalizes to other patients. The record does not identify the event term or terms for this category. It also does not provide a narrative establishing severity, duration, recurrence, management, or the investigator's attribution for the reported other event. Those details are not reported in the registry record.
The registry lists a secondary outcome of change from baseline in 6-minute walk distance, but it lists no analyses. There is consequently no reported numerical change, uncertainty interval, or statistical test to interpret for that outcome. The stated study purpose included effectiveness, but a purpose statement is not an effectiveness result. No treatment effect relative to a control can be estimated from a single-group design, even if a before-and-after measurement had been reported.
The study is listed as terminated, and the record says it was discontinued due to funding withdrawal. That context helps explain why the record may be limited, but it does not establish how much follow-up was completed or whether the listed timeframes were reached. The follow-up completeness and reason narrative beyond funding withdrawal are not reported in the registry record. Overall, the posted information is a sparse description of safety observations, not a definitive assessment of the intervention's safety or effectiveness.
9. Statistical Methods Explained
What the study design can estimate. This is a single-group study, not a randomized comparison. In general, a single-group study can describe outcomes among people observed while receiving an intervention. It cannot directly estimate a treatment contrast against a concurrent control group. Changes over time may reflect the intervention, the disease's natural course, other care, measurement variability, or other factors. These alternative explanations cannot be separated by the design recorded for this trial.
What open-label means. The registry lists masking as none. In general, open-label means participants and investigators know which intervention is being given. This matters especially for outcomes that depend on reporting symptoms, investigator judgment, or decisions about whether an event is related to treatment. The primary endpoint uses an investigator judgment of whether an adverse event was reasonably or probably related to abatacept. Such a judgment can be clinically meaningful, but it is not an objective test of causation and is not protected from expectation effects by masking.
How to read a participant count. The primary outcome is expressed as a number of participants with qualifying adverse events. A participant count is different from an event count: an individual may experience multiple events, while a count of affected participants records that person once under the relevant definition. The registry's adverse-event summary lists one participant at risk and zero affected for serious events, and one at risk and one affected for other events. These counts are descriptive. With such a limited observation base, they do not estimate a population rate with useful precision. No confidence interval is reported in the registry record.
Why uncertainty matters. General statistical explanation: a confidence interval describes the range of values compatible with observed data under a specified statistical model. Wider intervals indicate greater uncertainty. A small amount of information usually leaves substantial uncertainty, even when a count is zero. No interval can be interpreted for the primary outcome from the posted analysis information because no analysis result is listed. The absence of a reported interval is not evidence that the result is precise.
Over-dispersion and count data. General statistical explanation: for count outcomes, a simple model may assume that variability follows a particular relationship to the average. Over-dispersion occurs when the observed variability exceeds that assumption. It can make standard errors too small and statistical evidence appear stronger than warranted if the model does not account for it. The registry record does not report a count-data model, an over-dispersion assessment, or a statistical analysis for the primary endpoint. With one participant listed, the record cannot support a meaningful assessment of variability across participants.
Repeated timeframes. The primary outcome lists 24 weeks and 52 weeks. In general, repeated assessment can describe whether events arise over different periods of follow-up, but interpretation depends on how the observation windows and people at risk are defined. A participant may contribute information at more than one timepoint, so the observations are not necessarily independent. The registry does not report the timepoint-specific counts, follow-up completeness, or method used to combine or compare the timeframes. It is therefore not possible to infer a longitudinal analysis from the timeframe labels alone.
Analysis results and the secondary outcome. The record lists two outcome measures and zero analyses. It names change from baseline in 6-minute walk distance as a secondary outcome, but no estimate, dispersion measure, confidence interval, or test result is reported. General statistical explanation: a change-from-baseline analysis compares a follow-up measurement with a starting measurement for the same participant. Its interpretation depends on the baseline and follow-up values, timing, completeness, and the analysis method. None of those numerical results or methods is reported in the registry record.
Planning calculations are not trial results. General statistical explanation: sample-size planning asks how much information a future or planned study might need to detect a specified effect under stated assumptions. It is not a measure of the probability that an observed result is true. The planning assumptions associated with this record describe an illustration using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the detectable effect expressed as a fraction of an observed effect. Those assumptions do not describe this trial's single-group design. Any such sample-size figures are planning illustrations, not observed power, not the trial's actual enrollment target, and not evidence of an effect. A trial-specific sample-size justification is not reported in the registry record.
What cannot be reconstructed. No analysis method, model specification, significance test, adjustment strategy, or statistical analysis plan is reported in the registry record. There are no posted analysis results from which to recalculate an estimate. General recalculation of a standard error, confidence interval, or test statistic requires suitable summary data and a defined method; those cannot be inferred from the counts alone. The clinical interpretation should therefore remain descriptive and limited to the recorded counts and design.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures: the primary safety outcome and change from baseline in 6-minute walk distance as a secondary outcome. It lists zero analyses. No statistical analysis plan is reported in the registry record, including any approach to multiplicity. Multiplicity concerns arise when several outcomes, timepoints, or analyses are tested: the more opportunities there are to find a result, the harder it can be to interpret an isolated nominal statistical finding. Here, no statistical findings are listed, so there is no reported set of tests to assess or adjust.
The primary outcome includes timeframes of 24 weeks and 52 weeks. The record does not state whether these were separate analyses, a combined endpoint, or alternative follow-up descriptions. It also does not report which timepoint would be primary for formal inference, whether any hierarchy was planned, or how repeat assessments would be handled. Those details are not reported in the registry record; the timepoint labels alone do not establish the statistical plan.
Missing-data handling and analysis populations are also not reported in the registry record. There is no stated rule for incomplete follow-up, missed outcome assessments, withdrawals, or participants with partial event information. The record does not define an intention-to-treat, per-protocol, safety, or other analysis population. Because enrollment is listed as 1, these omissions sharply limit the ability to judge whether the outcomes were collected as intended or whether the displayed counts include all relevant follow-up.
The registry notes termination due to funding withdrawal, but does not report whether the discontinuation affected missingness, follow-up duration, or outcome collection. No conclusions about the direction or impact of missing data can be made from that reason alone.
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 |
|---|---|---|---|
| Abatacept | 0 | 1 | 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 |
|---|---|
| Abatacept | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Abatacept |
|---|---|---|
| Worsening shortness of breath | Respiratory, thoracic and mediastinal disorders | 1 of 1 |
The serious adverse-event summary lists 1 participant at risk and 0 affected. The other adverse-event summary lists 1 participant at risk and 1 affected. No serious adverse-event terms are listed. These are participant counts, not necessarily counts of all individual events. The record lists deaths as null; a death count is not reported in the registry record, which is distinct from an explicit count of zero.
A zero serious-event count in a study with one participant at risk is a limited observation, not proof that serious events cannot occur. It cannot rule out uncommon harms or establish a precise risk level. Similarly, the other-event count describes what was recorded for this study; the event details and their attribution are not reported in the registry record. The primary endpoint is restricted to events the investigator considered reasonably or probably related to abatacept, but the summary counts alone do not provide a detailed account of that judgment for each event.
Because the study is open-label and has no control group, these figures cannot separate treatment-related events from events that might have occurred for other reasons. They should be read as sparse descriptive safety observations, not as a comparative safety estimate.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase 2, open-label, single-group study of abatacept in progressive sarcoidosis was registered, with a stated safety focus and enrollment listed as 1. It also records one participant at risk for serious adverse events, none affected in that category, and one participant at risk and affected in the other-event category. No serious adverse-event terms are listed. These details describe what the registry reports; they do not provide a comprehensive safety profile.
The record does not show whether abatacept improves symptoms, lung function, walking distance, or any other clinical outcome. Although the study's purpose statement mentions effectiveness and the secondary outcome is change from baseline in 6-minute walk distance, no analysis results are listed. A purpose, an outcome definition, and an observed treatment effect are different things. The registry record includes the first two but not the last.
Even a reported before-and-after change in a single group would not, by itself, show what caused the change. Without a control group, changes could reflect natural variation, other care, measurement conditions, or the disease course. Here, the record does not supply a numerical change to assess in the first place. It also does not provide uncertainty estimates that would indicate the precision of any treatment-related estimate.
The zero serious-event count does not prove an absence of serious risk. A small observation base has little ability to identify uncommon events, and the record's deaths field is not reported rather than explicitly zero. The other-event count is similarly too limited to support a general frequency estimate. Further information about event descriptions, duration, follow-up, and attribution is not reported in the registry record.
Consequently, the record supports a narrow conclusion: it documents a very small, terminated safety study and sparse event counts, but does not report analysis results sufficient to assess safety or effectiveness with precision. It should not be interpreted as evidence that abatacept does or does not work, or that it is or is not safe. The limits arise from the size and design of the study and from missing analysis and follow-up detail in the registry record.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates how design and information volume constrain what a result can say. A single-group study can document events observed during treatment, but it cannot make the direct treatment-versus-control comparison that random allocation is intended to support. The open-label design also leaves investigator judgments and participant reports unmasked. Those features are particularly relevant when the main outcome depends on judging whether an event is related to treatment.
The registry lists enrollment of 1. Even a clear count from one participant contains little information about variation across people. A count of zero serious adverse events may look simple, but the amount of information behind that zero is crucial. The same count in a much larger, well-followed group would have a different level of precision. Statistical interpretation therefore depends not just on the event count but also on the number observed and the completeness of follow-up.
The record also distinguishes an outcome's planned role from an analyzed result. Safety is named as the primary outcome, and 6-minute walk distance as a secondary outcome, but no analyses are listed. That gap prevents assessment of effect estimates, uncertainty, or consistency across outcomes and timepoints. It also prevents independent evaluation of whether the registry's stated objectives were met.
Finally, the planning assumptions illustrate why numerical design calculations must be kept separate from trial evidence. An illustration based on a two-arm allocation does not describe a single-group study, and a planning calculation is not an observed-power statement. The record does not report a trial-specific sample-size justification. Together, these features show why a registry record may document a study and some safety counts without supporting a conclusive statistical claim.
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 central limitation is the very small amount of information: registry enrollment is 1. That number does not support a reliable estimate of how often adverse events occur in the broader population of people with sarcoidosis. It also cannot characterize variation in response or risk between participants. The adverse-event counts should therefore be understood as observations from a limited record, not as stable estimates.
The study has no comparator group and is open-label. This prevents a controlled estimate of treatment effect and leaves the reported observations open to influences from awareness of treatment and investigator judgment. Since the primary endpoint includes an investigator assessment of relatedness, the absence of masking is especially relevant to interpretation. There is no basis in the record for separating treatment effects from other explanations.
The record lists no analyses. Numerical results for the primary safety endpoint and the secondary 6-minute walk outcome, along with uncertainty estimates and analysis methods, are not reported in the registry record. Nor are the actual event descriptions for the other-event count or a death count. Missing-data procedures, outcome-assessment completeness, analysis populations, and the extent of follow-up are not reported in the registry record.
The study is listed as terminated, and the record attributes discontinuation to funding withdrawal. It does not establish how termination affected the planned follow-up periods of 24 weeks and 52 weeks, or whether the listed outcomes were assessed at those timepoints. The explanation accompanying the funding statement is incomplete. Eligibility criteria, dosing, treatment exposure, and the original recruitment target are also not reported in the registry record. These gaps prevent a fuller assessment of applicability, conduct, and the reasons for the limited information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety Study of Abatacept to Treat Refractory Sarcoidosis — NCT00739960.
- Registry record last updated 2020-03-23; status last verified 2020-03.
- Results first posted 2020-03-17.
- 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 phase 2, prospective, open-label, single-group study of abatacept for progressive sarcoidosis, sponsored by the University of Chicago. It lists enrollment of 1, a start date of 2008-08, primary completion in 2008-12, and status as terminated. Safety was the primary outcome, defined as participants with adverse events considered reasonably or probably related to abatacept, with timeframes of 24 weeks and 52 weeks. Change from baseline in 6-minute walk distance was listed as a secondary outcome.
The adverse-event summary lists 0 of 1 participants affected by serious events and 1 of 1 affected by other events; no serious-event terms are listed. Deaths are not reported in the registry record. The record lists zero analyses, so it provides no numerical analysis result for either outcome. Discontinuation is attributed to funding withdrawal. With no control group, one listed enrollee, and no reported analysis results, the record supports only a limited descriptive account, not a precise assessment of safety or effectiveness.