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 Ipilimumab Administered to Stage IIIC Stage IV Melanoma After Reg. T Cell Depletion With Denileukin Diftitox (NCT02009384), a na phase 2 study. The primary outcome is Change From Baseline Using Immune-related Response Criteria (irRC).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02009384 |
| Phase | Phase 2 |
| Condition | Metastatic Melanoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 2 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Louisville |
| Results posted | Yes |
2. Clinical Question
The study was designed to examine change in melanoma status among people with stage IIIC or stage IV melanoma who had recently received denileukin diftitox. The intervention was intravenous ipilimumab. The primary question was whether measurements using immune-related response criteria (irRC) changed from baseline across the planned assessment times.
The registry describes eligibility as including radiographically measurable melanoma and at least one cycle of denileukin diftitox. Its brief summary anticipated approximately 42 participants, while the enrollment entry reports 2. The record says the study was terminated because of low subject enrollment, related to the requirement for prior denileukin diftitox treatment.
Population
People with stage IIIC or stage IV melanoma, radiographically measurable disease, and prior treatment with at least one cycle of denileukin diftitox.
Intervention
Intravenous ipilimumab after recent denileukin diftitox treatment.
Comparator
No comparator group is described. The registry identifies a single-group study.
Primary question
How did measurements under irRC change from baseline at 12 weeks, 24 weeks, 36 weeks, and 48 weeks?
This is a question about observed change over time within the treated group, not a randomized comparison between treatments. Since the registry says the data were not analyzed and provides no primary outcome values, it does not establish what changes occurred or how precisely they could be estimated.
3. Trial Design
The registry identifies NCT02009384 as a phase II, open-label study sponsored by the University of Louisville. Its design is listed as single-group, with allocation recorded as not applicable and masking as none. The intervention is ipilimumab, described as intravenous. The study concerned stage IIIC and stage IV melanoma after prior denileukin diftitox treatment.
The brief summary anticipated approximately 42 participants with radiographically measurable melanoma who had received at least one cycle of denileukin diftitox. The enrollment entry reports 2. The registry marks the study terminated and attributes termination to low subject enrollment, related to the prior-treatment requirement. The recorded start is 2012-01 and primary completion is 2014-10.
A single-group design can describe what happened to participants receiving the intervention, but it does not by itself separate changes associated with treatment from changes that might have occurred for other reasons. Open-label status means masking was not used; the record does not describe any blinded assessment procedure. No randomized control group or allocation comparison is reported.
The primary outcome was planned as change from baseline using irRC, with assessments at baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks. The registry states that no outcomes were available, the study was terminated for low enrollment, and the data were not analyzed. Further protocol details, including the dosing schedule and any sample-size justification, 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: Study was Terminated
Overall Study
| Stage | Ipilimumab |
|---|---|
| STARTED | 2 |
| COMPLETED | 0 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Ipilimumab |
|---|---|
| Death | 2 |
Analysis population for the primary outcome, as defined in the registry: no outcomes available, Study was Terminated due to low subject enrollment \[r/t requirement of prior treatment with denileukin diftitox\] Data was not analyzed
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 | Ipilimumab |
|---|---|---|
| Participants analysed | 2 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 2 |
| Age, Continuous | years | 67 (65 to 69) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline Using Immune-related Response Criteria (irRC), measured over baseline, 12 weeks, 24 weeks, 36 weeks, 48 weeks.
| 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 outcome is titled change from baseline using immune-related response criteria (irRC). The planned time points were baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks. In broad terms, a change-from-baseline endpoint compares a participant’s later measurement with a starting measurement. It can describe whether the measured disease status moved over time within the study group.
The registry does not specify a unit, numerical parameter, or dispersion measure for this endpoint. It also does not provide the actual irRC measurements or an analyzed summary. The record explicitly states that no outcomes were available and that the data were not analyzed. Thus, the endpoint’s intended role can be described, but its observed values, direction of change, variability, and precision cannot be assessed from the registry record.
As a general statistical point, repeated assessments can help characterize the course of an outcome at more than one follow-up time. Interpretation depends on knowing how the measure was defined, which participants contributed data at each time, and how missing assessments were handled. Those details and results are not reported in the registry record. The planned endpoint therefore remains a question the study intended to address, rather than a reported finding.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The main interpretive fact is that the registry reports no analyzed primary outcome. Although the study planned to assess change from baseline using irRC through 48 weeks, the record says outcomes were unavailable and the data were not analyzed. There is consequently no reported treatment-group estimate of change, no measure of uncertainty for that estimate, and no basis in the posted results for describing the direction or size of an efficacy outcome.
The enrollment entry is 2, whereas the brief summary anticipated approximately 42 participants. The record attributes termination to low enrollment, related to the requirement for prior denileukin diftitox treatment. This context matters because a small enrolled group and unavailable outcome analyses leave the intended clinical question unanswered in the registry results. They do not show that the intervention had no effect, nor do they demonstrate an effect.
The design also limits what an analyzed change could have established. This was an open-label, single-group study, with no comparator group described. In general, a before-and-after change in a single group does not isolate the effect of an intervention from other influences on the outcome. A concurrent comparison can help address that question, but none is reported for this study.
The safety entries are separate from the missing efficacy analysis. The registry lists 2 at risk for serious adverse events and 1 affected, with Grade: 3 Encephalopathy listed as a serious term at 50.0%. It also lists 2 at risk and 0 affected in the other adverse-event category. These entries should be read as the registry’s recorded safety counts, not as a complete characterization of all possible harms. Deaths are not reported in the safety entry.
Because the primary outcome was not analyzed, conventional measures of statistical evidence for that outcome, such as an estimate with a confidence interval or a test result, are not reported in the registry record. No conclusion about treatment efficacy can be drawn from the absence of those results. The registry documents a terminated study with limited enrollment and unavailable analyses, not a demonstrated positive or negative primary outcome.
9. Statistical Methods Explained
The design information identifies a phase II, open-label, single-group study. These labels describe different aspects of the research. Phase II identifies the registered phase; open-label indicates that masking was not used; and single-group means the record does not describe separate intervention and control groups. Allocation is listed as not applicable. These design labels do not substitute for outcome data or an analysis plan.
What does a single-group design estimate?
In general, a single-group study can describe outcomes observed among participants who received the intervention. If measurements are collected at baseline and follow-up, an analysis may summarize within-participant change or group-level change over time. Such a summary is descriptive of the enrolled group. Without a concurrent comparator, it cannot by itself establish what would have happened to similar participants without the intervention. Changes may reflect the intervention, the course of the condition, other care, measurement variation, or a mixture of influences. This is a general limitation of the design, not a finding about the study’s outcome.
What does open-label mean for interpretation?
Open-label means masking was not used. In general, knowledge of treatment assignment can affect how participants report experiences, how clinicians make decisions, or how outcomes are assessed, depending on the outcome and procedures. The registry does not describe additional safeguards or blinded assessments. It would therefore be inappropriate to assume that such safeguards were or were not used beyond what the record states.
How should change from baseline be understood?
A change-from-baseline outcome uses an initial measurement as a reference for later measurements. The planned irRC assessments were at baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks. In general, an analysis could report change at each follow-up or model measurements across time; those approaches answer related but not identical questions. The registry does not say which approach was planned, how the irRC value was represented numerically, or what summary measure would be used. It provides no endpoint values because the data were not analyzed.
A confidence interval, as a general concept, expresses the uncertainty around an estimated quantity under the assumptions of the analysis. It does not repair limitations such as the absence of a comparator, and its width depends on the data and analysis. No confidence interval for the primary outcome is reported. Likewise, the registry does not report a primary-outcome test statistic or p-value. There is no result to which those measures could be attached.
What can the enrollment information establish?
The registry reports enrollment of 2, while the brief summary anticipated approximately 42. It also states that the study was terminated due to low subject enrollment, related to the prior denileukin diftitox requirement. These registry entries describe recruitment and termination; they do not explain all reasons participants may or may not have enrolled. The recruitment barrier identified in the record is the requirement for prior treatment. Further recruitment details are not reported in the registry record.
Sample-size planning is generally tied to a specified outcome, a target effect, variability, and assumptions about the analysis. The statistical plan and sample-size justification are not reported in the registry record. The record’s planned enrollment statement is not an analyzed result, and the enrolled group does not provide a reported primary-outcome estimate. Any planning illustration based on an observed effect should not be confused with observed power or with evidence about the study’s clinical outcome.
Why do analysis details matter?
For repeated measurements, an analysis plan typically specifies the outcome definition, the time points to be emphasized, the participants included, and the handling of incomplete observations. These choices can affect the estimate and its interpretation. The record supplies planned assessment times but does not report the statistical model, the primary analysis population, missing-data procedures, or multiplicity strategy. Since the data were not analyzed, no assumptions or choices can be inferred from an outcome analysis.
General statistical methods can explain how results would ordinarily be interpreted, but they cannot generate missing study findings. Here, the appropriate reading is limited to the registered design, planned endpoint, enrollment, termination reason, and recorded safety entries. The primary outcome remains unreported rather than estimated with low precision or shown to be unchanged.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary outcome assessed at baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks. It does not report whether one follow-up time was designated as the principal comparison, whether all time points were intended to be interpreted together, or how any multiple-testing issue would be addressed. The multiplicity strategy is not reported in the registry record.
As a general matter, examining results at multiple time points can create several opportunities to find an apparent difference. A prespecified analysis plan can clarify which comparisons are primary and how uncertainty should be interpreted. No such details are available in this record, and there are no analyzed outcome results to evaluate.
The record states that the data were not analyzed and that no outcomes were available. It does not report how missing measurements would have been handled, whether participants with incomplete follow-up would have been included in particular analyses, or how analysis populations were defined. Those procedures are not reported in the registry record. The enrollment entry and termination explanation do not establish how many scheduled assessments were completed.
In general, missing-data methods rely on assumptions about why values are absent and how that absence relates to the unobserved outcome. Without participant-level follow-up information or a stated method, those assumptions cannot be assessed. Since there is no primary outcome analysis, it is not possible to determine whether missingness affected an estimate or its precision. The registry record supports describing these analytic details as unreported, not guessing how the investigators would have handled them.
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 |
|---|---|---|---|
| Ipilimumab | 1 | 2 | 50.0% |
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 |
|---|---|
| Ipilimumab | 0 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Ipilimumab |
|---|---|---|
| Grade: 3 Encephalopathy | Nervous system disorders | 1 of 2 |
Serious events with the highest arm-level rate: grade: 3 encephalopathy (50.0%).
The registry lists 2 participants at risk for serious adverse events and 1 affected. The listed serious term is Grade: 3 Encephalopathy, reported as 50.0%. The record also lists 2 at risk and 0 affected for the other adverse-event category. These are the safety figures recorded for the ipilimumab group.
These entries are counts within a small enrolled study group, not estimates that can be generalized confidently to a broader population. A percentage based on a small group can be particularly sensitive to each recorded event. The serious-event listing should not be interpreted as a complete description of severity, duration, relatedness, or outcome; those details are not reported in the registry record.
Deaths are not reported in the safety entry. That is different from a reported count of zero. The safety data also do not answer the primary efficacy question: the registry says the primary outcome data were not analyzed. In general, adverse-event summaries describe recorded safety events under the categories used, and interpretation depends on the definitions, observation period, and completeness of reporting. Further details are not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, open-label, single-group study of intravenous ipilimumab after denileukin diftitox was registered for people with stage IIIC or stage IV melanoma. It planned to examine change from baseline using irRC at baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks. It also reports enrollment of 2 and termination due to low subject enrollment related to the prior-treatment requirement.
The primary outcome does not have an analyzed result in the registry. Therefore, the record does not show whether participants’ irRC measurements improved, worsened, or remained stable. It does not provide an estimate of the size of any change, a confidence interval, or an analysis of differences over time. The lack of a reported result is not evidence that there was no effect; it means the intended outcome question cannot be answered from the posted results.
Even if an outcome change had been reported, the single-group design would describe change among participants in the study rather than provide a randomized comparison with another treatment or control. In general, that distinction matters when asking whether observed change can be attributed to an intervention. The registry does not report a comparator group.
The safety record lists a serious adverse event affecting 1 participant and a Grade: 3 Encephalopathy term at 50.0%. It also lists 0 affected for the other adverse-event category, while deaths are not reported. These entries are descriptive registry data and should not be treated as a comprehensive assessment of safety beyond what is recorded.
Overall, the record documents the study’s intended question, limited enrollment, termination, and absence of analyzed outcome data. It does not establish that ipilimumab did or did not produce a clinical benefit, and it does not settle the safety profile of the intervention.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction between a study’s planned question and the result available for interpretation. The primary endpoint and assessment schedule are recorded, but the registry states that no outcomes were available and the data were not analyzed. A clearly stated endpoint does not itself provide evidence about the outcome.
It also shows why recruitment is part of the evidential context. The brief summary anticipated approximately 42 participants, while the enrollment entry reports 2. The registry attributes termination to low enrollment related to the requirement for prior denileukin diftitox. This recruitment history helps explain why the planned clinical question remains unanswered in the record, without implying a particular result.
The single-group, open-label design offers another useful statistical lesson. Within-group changes can be informative descriptions, but without a comparator they cannot isolate the intervention’s contribution to those changes. Design structure and analysis results answer different questions: the former describes what comparisons were possible, while the latter would describe what the data showed. Here, the primary analysis is unavailable.
Finally, the safety entries demonstrate why counts, percentages, and missing reporting need to be read in context. A recorded percentage does not remove the uncertainty associated with a small group, and an unreported death count is not the same as a reported absence of deaths. Careful statistical interpretation distinguishes what the registry records from what it leaves unresolved.
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 for interpreting efficacy is explicit: the registry says no outcomes were available and the data were not analyzed. No numerical primary endpoint result, estimate of change, or measure of precision is reported. The intended outcome therefore cannot support an inference about the direction or magnitude of change.
Enrollment was 2, compared with approximately 42 anticipated in the brief summary. The study was terminated due to low subject enrollment, related to the prior denileukin diftitox requirement. The record does not report a sample-size justification, so the reason for the planned enrollment target and its statistical assumptions cannot be assessed.
The study was open-label and single-group, with no comparator described. In general, this structure limits causal interpretation of any within-group change. The registry also does not report the statistical analysis plan, analysis populations, handling of incomplete assessments, or multiplicity strategy. Those omissions matter for evaluating how repeated measurements would have been analyzed, but no procedures should be inferred.
The safety entry includes a serious-event count and term, but details such as event relatedness, duration, follow-up completeness, and death count are not reported in the registry record. The record’s safety categories should consequently be read as limited reported summaries, not as exhaustive evidence about harms. The registry does not provide additional information that would resolve these limitations.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Ipilimumab Administered to Stage IIIC Stage IV Melanoma After Reg. T Cell Depletion With Denileukin Diftitox — NCT02009384.
- Registry record last updated 2021-10-25; status last verified 2021-10.
- Results first posted 2021-10-25.
- 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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18. Record Summary
NCT02009384 is registered as a phase II, open-label, single-group study of intravenous ipilimumab in stage IIIC and stage IV melanoma after denileukin diftitox. The planned primary endpoint was change from baseline using irRC at baseline, 12 weeks, 24 weeks, 36 weeks, and 48 weeks.
The brief summary anticipated approximately 42 participants; the enrollment entry reports 2. The study is marked terminated because of low subject enrollment related to the prior-treatment requirement. The registry states that no outcomes were available and the data were not analyzed.
The safety record lists 1 participant affected by a serious adverse event and reports Grade: 3 Encephalopathy at 50.0%. Deaths are not reported. The registry does not provide an analyzed primary outcome or a comparator-based estimate.