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 Romidepsin in Treating Patients With Steroid-Refractory Graft-versus-Host Disease (NCT02203578), a na na study. The primary outcome is Incidence of aGVHD.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02203578 |
| Phase | NA |
| Condition | Graft Versus Host Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Rutgers, The State University of New Jersey |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with graft-versus-host disease that had not responded to steroid treatment. The registry summary describes this condition in the setting of bone marrow or stem cell transplantation. More detailed eligibility criteria, such as disease severity or prior therapies, are not reported in the registry record.
Intervention
Patients received romidepsin intravenously over 4 hours on days 1, 8, and 15. Treatment was scheduled to repeat every 28 days for up to 6 courses, unless disease progression or unacceptable toxicity occurred. Laboratory biomarker analysis was also listed as an intervention. The registry record does not report the biomarker procedures or their results.
Comparator
There was no separate comparator group. The record describes a single-group study with allocation listed as NA and masking listed as none. Its supportive-care arm name includes romidepsin, but it does not describe a like-for-like control arm or a comparison against another treatment.
Primary question
The primary outcome was the incidence of acute graft-versus-host disease at 28 days after romidepsin initiation. The registry record states that the study ended early because accrual was slow and insufficient data were collected to assess this measure. It therefore does not report an estimate of the primary outcome or an answer to the clinical question.
3. Trial Design
This was a pilot clinical trial of romidepsin for patients with steroid-refractory graft-versus-host disease. The registry classifies the design as single group, with allocation listed as NA and masking as none. The phase is also listed as NA. Because there is no separate control group, the design does not support a randomized treatment comparison.
The record lists enrollment as 1. The treatment schedule was intravenous romidepsin over 4 hours on days 1, 8, and 15, with treatment repeating every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis was also listed. The registry record does not describe how treatment exposure, dose changes, or biomarker measurements were handled in practice.
The study began in 2014-11, and the primary completion date is listed as 2016-06-14. Its status is terminated. The record attributes early termination to slow accrual and says there were insufficient data to assess the primary outcome. It does not report a sample-size justification, a recruitment target, or reasons beyond those stated for the termination.
The sponsor is Rutgers, The State University of New Jersey. The record lists three secondary outcomes: total duration of immunosuppressive therapy, rate of documented infection, and T cell kinetics - reconstitution. It indicates nine outcome measures overall, but does not report analyses for them; the number of analyses listed is 0. Further details about outcome definitions and analysis populations are 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: We are reporting results on 1 eligible participant.
Overall Study
| Stage | Supportive Care (Romidepsin) |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Study was terminated early due to slow accrual and insufficient data was collected to assess this outcome measure.
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.
Population: Participants \> 18 years old with steroid (or IST) refractory aGVHD or cGVHD were enrolled.
| Characteristic | Unit | Supportive Care (Romidepsin) |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 49 (49 to 49) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Incidence of aGVHD, measured over At 28 days after initiation of romidepsin.
| 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 is the incidence of acute graft-versus-host disease, abbreviated aGVHD, at 28 days after initiation of romidepsin. An incidence endpoint asks whether the specified condition occurred within the stated assessment period. In general, such an endpoint can describe how often an event is observed in a defined study population over a specified period. The registry record does not give a more detailed case definition, grading system, assessment procedure, or rule for handling events when follow-up is incomplete.
Incidence was the stated primary measure for this pilot study. It focuses on whether acute GVHD was observed after treatment began, rather than on a continuous measurement such as a laboratory value or on time until an event. However, the record says that early termination due to slow accrual left insufficient data to assess this outcome. It therefore does not report an incidence estimate, event count for this endpoint, or measure of precision such as a confidence interval.
The endpoint must be interpreted as a planned question, not as a reported finding. The absence of an assessable result does not indicate that the outcome occurred or did not occur. Additional details about the outcome's operational definition and the methods intended for its analysis are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical fact is that the registry record does not provide an assessable result for the primary endpoint. It identifies incidence of aGVHD at 28 days after romidepsin initiation as the outcome, then states that the trial was terminated early because accrual was slow and insufficient data were collected to assess it. No endpoint estimate, confidence interval, or analysis result is listed. The record also lists 0 analyses.
Enrollment is listed as 1, and the design is single group. These features sharply limit what can be learned from the record. A single-group study can describe observations among enrolled participants if outcomes are available, but it cannot by itself separate a treatment-associated observation from the course of the condition or other influences. Here, the primary outcome is not assessable in the registry record, so even a descriptive estimate for that outcome is unavailable.
The record gives no effect estimate for romidepsin and no comparative estimate against another intervention or usual care. It is therefore not possible to quantify a treatment effect, assess its precision, or judge whether any observed difference is compatible with a range of possibilities. No claim that romidepsin did or did not change the incidence of aGVHD follows from the reported primary-outcome information.
The safety record requires a separate, equally cautious reading. In the supportive-care (romidepsin) group, 1 participant is listed at risk for serious adverse events and 1 as affected; the record lists 1 at risk and 1 affected for other adverse events, and 0 deaths. The top serious term is endocarditis infective at 100.0%. With the listed group size and the absence of further context, these entries are descriptive registry counts, not a stable estimate of event frequency in a wider population. They do not establish that the intervention caused an event.
Overall, the posted information characterizes a very small, terminated pilot study with no assessable primary endpoint result. It can document the planned question, treatment schedule, termination reason, and listed safety entries. It cannot provide a reliable estimate of efficacy, comparative benefit, or the frequency of adverse events beyond the participants recorded.
9. Statistical Methods Explained
Statistical interpretation starts with the design and the question the endpoint was intended to answer. This registry record describes a single-group pilot study, not a randomized comparison. The primary endpoint was incidence of aGVHD at 28 days after romidepsin initiation. The record says the study was terminated early because of slow accrual and insufficient data to assess that endpoint. It lists 0 analyses. Consequently, there is no reported primary-outcome estimate to interpret with inferential methods.
What does an incidence estimate mean?
In general, an incidence estimate describes the proportion of an observed study population in which a specified event occurs over a defined period. Its interpretation depends on a clear event definition, a relevant population, and adequate ascertainment during follow-up. If follow-up is incomplete, the method for treating participants whose outcome status is unknown can affect the estimate. The registry record does not report an operational definition for aGVHD, endpoint ascertainment details, or a missing-outcome approach. It also explicitly states that insufficient data were collected to assess this endpoint. No incidence value can therefore be inferred from the planned endpoint description.
Why does the single-group design matter?
In general, a single-group study can describe outcomes observed among participants who receive the intervention. Without a separate comparison group, however, it cannot directly estimate how those outcomes differ from what would have occurred under another treatment or without the study intervention. A change or event observed after treatment begins does not alone identify its cause. This limitation is distinct from precision: even a precisely measured single-group outcome would not, by itself, establish a comparative treatment effect. In this record, the primary outcome is not assessable, so neither a descriptive estimate nor a comparative effect is reported.
What would a confidence interval add?
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the statistical method. A narrow interval indicates greater statistical precision than a wide interval, but an interval cannot compensate for a design that does not answer the desired comparison. It also cannot be interpreted without an underlying estimate and an analysis population. The registry record reports no confidence interval or primary endpoint estimate. No precision claim can be made about the primary question.
How should sparse observations be considered?
In general, estimates based on very sparse observations can be highly sensitive to the outcome status of individual participants. For event outcomes, a small denominator can make the observed proportion an unstable guide to what might be seen in a broader population. Statistical methods can represent uncertainty, but they cannot create information that was not collected. Enrollment is listed as 1, and the record itself says there were insufficient data for the primary endpoint. The posted record therefore supports no generalizable estimate of the primary outcome.
What is not reported about the analysis?
The registry record does not report the planned statistical model, an analysis population, a confidence-interval method, an approach to missing data, interim analyses, or a sample-size justification. These details should not be inferred from the fact that the study was described as a pilot. A pilot label does not, by itself, determine whether an analysis was descriptive or inferential, what assumptions were used, or how many participants were intended for enrollment.
The record includes a treatment schedule and lists secondary outcomes, but does not report analysis results for those outcomes. General statistical concepts can explain how such outcomes might be analyzed, but they cannot establish which methods were planned or used in this study. Any recalculation would require posted summary statistics and an endpoint estimate; none is reported for the primary outcome. Statistical interpretation should therefore remain focused on what the registry documents: the planned endpoint, the single-group design, early termination, insufficient primary-outcome data, and the absence of listed analyses.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists incidence of aGVHD as the primary outcome and names three secondary outcomes: total duration of immunosuppressive therapy, rate of documented infection, and T cell kinetics - reconstitution. It indicates nine outcome measures overall, but the remaining measure names or details are not reported in the registry record. The number of analyses listed is 0.
When a study evaluates multiple outcomes, the chance of at least one apparently unusual result can increase if each is tested separately. In general, a prespecified analysis plan can identify which outcomes are primary, how secondary outcomes will be interpreted, and whether any adjustment for multiple testing will be used. An adjustment method, testing hierarchy, or multiplicity plan is not reported in the registry record. Because no analyses are listed, there are no reported outcome tests to interpret as adjusted or unadjusted.
The record says insufficient data were collected to assess the primary outcome, but it does not describe participant-level missingness, reasons for missing outcome information, follow-up completeness, or an imputation or other missing-data method. The number and definition of analysis populations are also not reported. The enrollment entry alone does not establish which participants would have been included in a particular analysis.
Early termination and inadequate data collection mean that the intended primary question remains unanswered in the posted record. They do not reveal how unobserved outcomes should be classified, nor do they establish that secondary outcomes were measured or analyzed. Details about the analysis plan, missing data, and populations 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 |
|---|---|---|---|
| Supportive Care (Romidepsin) | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Supportive Care (Romidepsin) | 0 of 1 |
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 |
|---|---|
| Supportive Care (Romidepsin) | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Supportive Care (Romidepsin) |
|---|---|---|
| Endocarditis infective | Infections and infestations | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Supportive Care (Romidepsin) |
|---|---|---|
| Nausea | Gastrointestinal disorders | 1 of 1 |
Serious events with the highest arm-level rate: endocarditis infective (100.0%).
For the supportive-care (romidepsin) group, the registry lists 1 participant at risk and 1 affected by serious adverse events. It also lists 1 at risk and 1 affected for other adverse events, with 0 deaths. These entries are registry counts; they should not be read as comparative rates because the study has no separate control group.
The top serious adverse-event term is endocarditis infective, listed at 100.0%. That percentage is tied to the registry's reported serious-event listing and the very limited group information; it is not a dependable estimate of how often the event would occur in a broader population. The record does not report further details about event timing, severity, duration, outcome, or the basis for judging a relationship to treatment. It also does not provide a comparison that could help distinguish background events from events associated with treatment.
Serious adverse-event and other-adverse-event entries are separate registry categories. Their affected counts should not be added together or interpreted as separate people. The record does not report whether categories overlap or provide participant-level event histories. The listed safety information is descriptive and limited; it does not establish that romidepsin caused an event or quantify risk for other patients.
12. What the Result Does, and Does Not, Mean
The study asked whether incidence of aGVHD could be assessed at 28 days after romidepsin initiation in patients with steroid-refractory graft-versus-host disease. The registry record says the study terminated early because accrual was slow and insufficient data were collected to assess that primary outcome. The primary result is therefore unavailable in the record.
This absence is not evidence that aGVHD did or did not occur, and it does not show that romidepsin was effective or ineffective. It means the registry does not report an endpoint estimate from which to draw a conclusion. No confidence interval or other measure of precision is listed. The available information cannot quantify the outcome or indicate how uncertain an estimate might be.
The single-group design is another important boundary. Even if outcome observations had been reported, without a separate comparator group the study would not directly establish how outcomes differed from those under another treatment or no study intervention. The record does not report a treatment-effect estimate, and no causal conclusion should be inferred from the treatment schedule or the safety entries.
The safety information records adverse-event categories for a very limited group and identifies endocarditis infective as the top serious term at 100.0%. These entries are not a robust estimate of risk in a wider population and do not demonstrate causation. The registry does not provide the contextual detail needed to interpret event timing or attribution.
The defensible conclusion is narrow: this terminated pilot study planned to assess aGVHD incidence at 28 days, but the registry reports that insufficient data were collected to assess it. The record documents the study design, intervention schedule, selected secondary outcomes, and limited safety entries. It does not answer whether romidepsin changes the primary outcome, establish comparative benefit or harm, or support a general estimate of event frequency.
13. Why This Trial Matters Statistically
This trial matters statistically because its record illustrates the difference between a planned outcome and an outcome that can actually be estimated. The primary question is clearly identified: incidence of aGVHD at 28 days after romidepsin initiation. Yet the registry says slow accrual led to early termination and insufficient data for assessment. A stated endpoint is not itself a result; without assessable outcome information, there is no estimate to interpret.
The study also illustrates why design and precision are separate concerns. A single-group design has no concurrent comparator, so it cannot directly estimate a difference between treatment strategies. Separately, the listed enrollment is 1, and the primary endpoint is not assessable. Increasing statistical complexity cannot resolve the absence of outcome information or supply the missing comparison.
Its safety entries show a related issue: percentages based on very sparse observations can appear definitive while offering little information about what might occur in a broader population. The listed serious term, endocarditis infective at 100.0%, must be read in the context of the reported group information, not as a stable population rate or proof of causation.
Finally, the record indicates nine outcome measures but lists 0 analyses. This makes clear why readers need to distinguish outcome registration from analyzed results. The registry can preserve the study's planned questions and basic conduct even when the available data cannot answer them. Here, the most statistically responsible interpretation is to identify what was intended, what the record says could not be assessed, and which conclusions remain unsupported.
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 primary limitation is stated directly in the registry record: the trial was terminated early because accrual was slow, and insufficient data were collected to assess the primary endpoint. Enrollment is listed as 1. As a result, the registry does not report an assessable estimate for incidence of aGVHD at 28 days after romidepsin initiation.
The single-group design offers no separate control arm for a direct treatment comparison. Allocation is listed as NA and masking as none. The record therefore does not provide a randomized or blinded comparison that could address differences between treatment strategies. This constrains causal interpretation even apart from the lack of a primary-outcome result.
Important outcome details are also absent. The registry does not report a detailed aGVHD definition, the planned statistical analysis, analysis populations, a missing-data approach, or the handling of incomplete follow-up. It does not report a sample-size justification or interim analyses. Those details should not be inferred from the pilot designation or from the treatment schedule.
The record lists nine outcome measures but names only three secondary outcomes in the available information, and it lists 0 analyses. It does not give results for those secondary outcomes. The safety entries are limited, with no reported event histories, timing, attribution detail, or comparison group. These limitations mean that neither efficacy nor population-level safety can be estimated from the registry information.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Romidepsin in Treating Patients With Steroid-Refractory Graft-versus-Host Disease — NCT02203578.
- Registry record last updated 2017-03-30; status last verified 2017-02.
- Results first posted 2017-03-30.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT02203578 is a pilot study of romidepsin for steroid-refractory graft-versus-host disease, sponsored by Rutgers, The State University of New Jersey. The registry describes a single-group design, with phase and allocation listed as NA and masking listed as none. Enrollment is listed as 1. Romidepsin was scheduled intravenously over 4 hours on days 1, 8, and 15, repeating every 28 days for up to 6 courses unless progression or unacceptable toxicity occurred.
The primary outcome was incidence of aGVHD at 28 days after treatment initiation. The study status is terminated; the record states that slow accrual and insufficient data prevented assessment of this outcome. The listed secondary outcomes are total duration of immunosuppressive therapy, rate of documented infection, and T cell kinetics - reconstitution. The record indicates nine outcome measures and 0 analyses.
The safety record lists 1 at risk and 1 affected for serious adverse events, 1 at risk and 1 affected for other adverse events, and 0 deaths. Endocarditis infective is the top serious term at 100.0%. The record does not report an assessable primary result or a comparative treatment effect.