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 Interleukin-2 in Metastatic Melanoma (NCT01702896), a na phase 2 study. The primary outcome is Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01702896 |
| Phase | Phase 2 |
| Condition | Melanoma Metastatic |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Western Regional Medical Center |
| Results posted | Yes |
2. Clinical Question
Population
People with metastatic melanoma. The primary-outcome population field says “Data not collected,” and further eligibility details are not reported in the registry record.
Intervention
Interleukin-2, described in the official title as moderate-dose bolus interleukin-2. Specific dose and administration details are not reported in the registry record.
Comparator
There is no comparator group. This is a single-group trial, and allocation is listed as NA.
Primary question
The stated objective was to determine whether interleukin-2 at the trial dose and schedule would help increase tumor shrinkage. The primary endpoint was response rate assessed with RECIST v1.0. The registry record does not report a response-rate result.
The study is identified as NCT01702896 and as a Phase II trial. Its record describes interleukin-2 as the intervention and lists metastatic melanoma as the condition. The enrollment field reports 8. Because there is no control group, the clinical question is about responses observed among participants who received interleukin-2, not about a randomized comparison between treatments.
Radiographic assessment was planned after every 2 cycles, with each cycle described as 6 weeks. Assessments were to continue until disease progression or death from any cause, up to 2 years. The record also lists median duration of response and median survival as secondary outcomes, but does not provide results for those outcomes. The primary question is therefore clear as a planned endpoint, while its observed answer is not available in the registry record.
3. Trial Design
This is a Phase II, single-group trial of interleukin-2 in metastatic melanoma. The registry record lists an enrollment of 8, identifies Western Regional Medical Center as the sponsor, and gives the trial status as terminated. The recorded start is 2012-09 and the primary completion is 2014-12. These dates and status describe the registry record; they do not establish why the trial ended or what results prompted that status.
The intervention is interleukin-2. The official title describes the regimen as moderate-dose bolus interleukin-2, while the intervention and arm description do not give a specific dose or detailed administration schedule. Such details are not reported in the registry record. The arm description says interleukin-2 will be used in the group.
Allocation is listed as NA, masking as none, and the model as single group. There is no like-for-like control arm. Consequently, this design can describe outcomes recorded among those treated, but it cannot by itself distinguish an intervention effect from other explanations for an observed outcome. In particular, a single-group response rate would not provide the same direct treatment contrast as a randomized comparison.
The primary endpoint is response rate assessed using RECIST v1.0. Radiographic studies were scheduled after every 2 cycles (6 weeks), until progression or death from any cause, up to 2 years. Median duration of response and median survival are listed as secondary outcomes. The registry record does not give the analysis plan, detailed eligibility criteria, specific dose, or reasons for termination. It also does not report outcome results or analysis records. Thus, the design and planned measurements can be described, but their observed findings cannot be reconstructed from this 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.
Overall Study
| Stage | Interleukin-2 |
|---|---|
| STARTED | 8 |
| COMPLETED | 0 |
| NOT COMPLETED | 8 |
Analysis population for the primary outcome, as defined in the registry: Data not collected
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 | Number of Participants |
|---|---|---|
| Participants analysed | 8 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 8 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Response Rate, measured over Measured until Disease Progression or death from any cause up to 2 years.
| 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 Response Rate. It is intended to capture the proportion of participants receiving interleukin-2 whose tumors meet the response criteria, based on radiographic assessment. The registry description specifies RECIST v1.0 for target lesions assessed by CT scan. Complete response means disappearance of all target lesions. Partial response means a >=30% decrease in the sum of the longest diameter of target lesions. The stated overall response rate combines complete and partial responses among the total number of patients receiving interleukin-2.
Response rate is used as a measure of observable tumor shrinkage under the specified criteria. It differs from outcomes such as survival because it classifies radiographic change rather than time until death. The assessment schedule was after every 2 cycles (6 weeks), continuing until disease progression or death from any cause, up to 2 years. The registry record does not report how many participants had evaluable scans, how response categories were assigned, or whether independent review was used.
Most importantly, the primary outcome population is recorded as “Data not collected.” No response counts, response rate, confidence interval, or analysis result are reported in the registry record. The endpoint definition explains what the trial intended to measure; it is not evidence that a particular response occurred.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a trial with an enrollment of 8, but it does not report the primary response-rate result. The outcome population field explicitly says “Data not collected,” and the record lists 0 analyses. Without response counts or a reported estimate, there is no observed response rate to interpret, and no estimate of its statistical precision can be drawn from the record.
This absence must not be read as a response rate of zero. A zero response rate would require outcome data showing that no participants met the response criteria. The record instead says the primary outcome data were not collected. Nor does the absence of a result imply that responses occurred. Neither conclusion is supported by the registry record.
The single-group design is also important for interpretation. Even if a response rate had been reported, the trial would describe outcomes in the interleukin-2 group without a concurrent control group. It would not directly estimate the difference between interleukin-2 and another treatment or no treatment. Tumor response can be described under the trial's RECIST definition, but causal attribution is more limited without a comparator.
The registry identifies median duration of response and median survival as secondary outcomes. It provides no values, event counts, or analyses for them. The timeframe for the primary assessment is specified, but reporting a timeframe is not the same as reporting follow-up completeness or participant-level outcomes. Details on missing assessments and the analysis population are not reported in the registry record.
The trial status is terminated. That status alone does not explain whether termination was due to recruitment, safety, efficacy, operational considerations, or another reason. The reason is not reported in the registry record. The recorded enrollment and completion dates likewise do not establish why the study ended or how many participants contributed to each endpoint.
Accordingly, the statistically supportable interpretation is limited: this was a small, single-group Phase II trial with a planned RECIST response-rate endpoint, but the registry record contains no collected primary-outcome result. The endpoint definition and study design can be evaluated; the magnitude, direction, and precision of a clinical response cannot be determined from the reported record. No conclusion about the intervention's effectiveness follows from the absence of results.
9. Statistical Methods Explained
Statistical methods are difficult to assess when an outcome has no reported data or analysis. The registry record names a response-rate endpoint and gives the response criteria, but lists 0 analyses and says the primary-outcome population was “Data not collected.” It does not report an analysis plan, a point estimate, uncertainty interval, or hypothesis test. The explanations below distinguish general statistical principles from methods documented for this trial.
What does a response rate estimate?
In general, a response rate is a proportion: the number of participants meeting a prespecified response definition relative to the relevant analysis population. Here, the registry describes the numerator categories as complete response and partial response and defines the denominator as the total number of patients receiving interleukin-2. The criteria are based on RECIST v1.0 assessments of target lesions by CT scan. Complete response requires disappearance of all target lesions; partial response requires a >=30% decrease in the sum of the longest diameter of target lesions.
Those definitions specify the intended classification, but the record does not state any observed counts. It also does not report whether every treated participant had a scan, how unevaluable cases would be handled, or whether the analysis denominator was in fact all participants who received treatment. These details matter because the estimated proportion can depend on which participants are counted and how missing assessments are classified. The handling of these issues is not reported in the registry record.
Why would an uncertainty interval matter?
In general, a confidence interval describes the statistical uncertainty around an estimate under a specified model and sampling framework. It is not a guarantee that the true value lies within the interval, and it does not resolve bias or limitations in the design. For a response-rate endpoint, an interval would help readers judge how precisely the observed proportion was estimated. No estimate or confidence interval is reported, so precision cannot be evaluated.
Small samples commonly yield imprecise estimates, particularly when the outcome is a proportion. That general consideration is relevant to the enrollment field of 8, but it does not permit a numerical interval to be created without response data and an explicit method. No such recalculation is possible from the registry record.
What can a single-group study establish?
In general, a single-group study can describe outcomes among participants receiving an intervention. It does not contain a concurrent comparator for estimating a between-group contrast. Changes in disease status may reflect the intervention, the natural course of disease, participant selection, assessment processes, or other factors. Without a control group, these explanations cannot be separated through a randomized treatment comparison. This design feature remains relevant regardless of the statistical method used to summarize the response rate.
Allocation is listed as NA, masking as none, and the model as single group. These fields are consistent with the lack of a randomized, masked comparison. The registry record does not describe any adjusted analysis or other approach intended to address the absence of a comparator.
What does the assessment schedule contribute?
Repeated radiographic assessments were planned after every 2 cycles (6 weeks), until disease progression or death from any cause, up to 2 years. In general, repeated measurements can establish when a response is first observed and whether it is maintained, provided assessments and follow-up are adequately recorded. The registry lists median duration of response and median survival as secondary outcomes, but gives no event definitions beyond the listed outcome names, no follow-up summaries, and no outcome results. No time-to-event method is reported in the registry record.
How should planning assumptions be read?
In general, a sample-size calculation is a planning exercise based on assumptions, not an observed result and not a measure of the achieved power of a completed trial. The registry's planning-illustration description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it also says the effect to detect is a stated fraction of the observed effect. For time-to-event illustrations, it specifies total events, while other illustrations are expressed as participants per arm.
Those assumptions describe hypothetical planning calculations, not the actual single-group design, which has no two-arm allocation. The registry record does not report an observed primary response effect from which the illustrations could be clinically interpreted. Any such planning quantities should therefore not be confused with enrollment, observed power, or evidence that a treatment effect was found. The trial's actual enrollment is recorded as 8; the record does not supply a sample-size justification or its own design calculation.
More broadly, a statistical analysis plan would clarify endpoint derivation, analysis populations, missing data, interim monitoring, confidence intervals, and testing procedures. Those details are not reported in the registry record. The available record supports explanation of the intended endpoint and general statistical concepts, but not a reconstruction of the trial's analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists Response Rate as the primary outcome and Median Duration of Response and Median Survival as secondary outcomes. It does not report a statistical analysis plan explaining how these outcomes would be tested or interpreted together. Procedures for controlling multiplicity, such as adjusting inference when several outcomes are examined, are not reported in the registry record. No p-values or outcome analyses are listed.
The record states “Data not collected” for the primary-outcome population and lists 0 analyses. It does not report the number of participants with evaluable radiographic assessments, whether any participants were lost to follow-up, or how missing scans or incomplete follow-up would be handled. A missing-data method, sensitivity analysis, or imputation approach is not reported in the registry record. Because the primary endpoint data are absent, the amount and consequences of missing outcome information cannot be assessed.
The analysis populations are also not described. The primary outcome description refers to the total number of patients receiving interleukin-2, but the record does not provide a defined intention-to-treat, treated, or evaluable population or confirm which denominator was used in an analysis. Rules for treatment discontinuation, protocol deviations, or inclusion in secondary-outcome analyses are not reported.
Interim analyses and stopping rules are not reported in the registry record. The terminated status does not establish that an interim analysis occurred or explain the decision to stop. Without an analysis plan and outcome data, the record cannot show how multiplicity, missingness, or analysis-population choices affected the results.
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 |
|---|---|---|---|
| Adverse Events | 0 | 8 | 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 |
|---|---|
| Adverse Events | 0 of 8 |
The registry's adverse-event group reports 8 at risk and 0 affected for serious adverse events. It also reports 8 at risk and 0 affected for other adverse events. These are the figures recorded for the adverse-event group; they should not be interpreted as a complete account of all possible safety outcomes without additional reporting details.
The deaths field is null, rather than a reported count. A null field does not mean that no deaths occurred; the number of deaths is not reported in the registry record. No top serious adverse-event terms are listed. The record does not give event-level descriptions, timing, severity grades, treatment relatedness, or details about how adverse events were collected.
In general, a participant count labeled “affected” describes people with at least one event in the relevant reporting category, while an event count may count multiple occurrences in one person. The record presents affected counts, not a detailed tally of event episodes. The zero affected values therefore describe what is entered in these registry fields; they do not establish that no adverse experiences occurred or that the intervention is safe. The small enrolled group and absence of detailed safety reporting limit interpretation.
12. What the Result Does, and Does Not, Mean
This record establishes that a Phase II study of interleukin-2 in metastatic melanoma was registered as a single-group trial, with an enrollment of 8 and a planned primary endpoint of RECIST response rate. It describes the intended radiographic criteria and schedule. The trial status is terminated, and the record includes secondary outcomes of median duration of response and median survival.
It does not establish how many participants experienced complete or partial response, the response-rate estimate, or its precision. The primary outcome population is marked “Data not collected,” and there are 0 analyses listed. Secondary-outcome values are also not reported. The record therefore cannot support a conclusion that participants did or did not respond, nor can it quantify survival or duration of response.
Even a reported response rate from this single-group design would be a description of outcomes among participants receiving interleukin-2, not a randomized estimate of the difference between interleukin-2 and a comparator. A single-group result does not by itself establish that the intervention caused a response. In this record, the lack of primary outcome data makes even that descriptive assessment unavailable.
The adverse-event fields list 0 affected for both serious and other adverse events, with 8 at risk for each category. The deaths field is null, and no top serious terms are listed. These entries should be read as registry reporting fields, not as proof that no safety events or deaths occurred. The record does not explain the status of safety ascertainment or provide detailed event information.
The most defensible conclusion is about reporting, not treatment effect: the trial's design and intended endpoint are described, but the registry record does not provide the results needed to judge the observed response or its uncertainty. The reason for termination and the trial's analysis plan are also not reported. No conclusion about benefit, lack of benefit, or comparative effectiveness can be drawn from the absent outcome data.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined endpoint and a reported result are both necessary for statistical interpretation. The registry specifies a RECIST-based response-rate outcome, including complete and partial response definitions and a radiographic assessment schedule. Those details make the intended measurement understandable. But the primary-outcome population is marked “Data not collected,” and the record lists 0 analyses. Without observed response data, neither the estimated response rate nor its precision can be evaluated.
The single-group design is another central statistical feature. It can describe outcomes among participants receiving interleukin-2, but it offers no concurrent control group for a direct treatment contrast. That limitation is separate from the missing results: even a complete outcome report would not turn this design into a randomized comparison.
The record also highlights the difference between a registry status and an explanation of study conduct. “Terminated” is recorded, but the reason is not. The dates and enrollment field provide descriptive information, not a substitute for an analysis plan, outcome data, or a transparent account of missing assessments. Secondary outcomes are named but not reported.
Finally, safety entries require careful reading. Zero affected is recorded in the serious and other adverse-event fields, while the deaths field is null. These values are not enough to characterize the completeness of safety ascertainment. Taken together, the record is useful for identifying the question the study intended to address and the limits of what can be inferred from its reported information. It demonstrates that endpoint definitions, comparison structure, data completeness, and analysis reporting each shape the strength of statistical conclusions.
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 principal limitation is that the registry record reports no collected primary-outcome data and no analyses. It therefore gives no response count, response-rate estimate, confidence interval, or test result. The same record does not provide results for median duration of response or median survival. These omissions prevent assessment of the observed outcomes and their statistical precision.
The study is single group, with allocation listed as NA and masking as none. No comparator is available for estimating a direct treatment contrast. The enrollment field reports 8, but the number included in any endpoint analysis is not reported. The record does not state whether participants had evaluable scans or how missing assessments would have been handled.
Important operational and methodological details are absent. Specific dose and schedule details beyond the title's description are not reported. Eligibility criteria, analysis populations, the statistical analysis plan, missing-data methods, multiplicity procedures, interim analyses, and stopping rules are not reported in the registry record. The reason for termination is likewise not reported.
Safety information is limited to aggregate at-risk and affected fields. Event details and ascertainment methods are not described; the deaths field is null. As a result, the adverse-event entries cannot establish the completeness of safety reporting. These limitations concern what the registry record contains and should not be mistaken for evidence about unreported trial conduct or outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Interleukin-2 in Metastatic Melanoma — NCT01702896.
- Registry record last updated 2018-04-05; status last verified 2018-04.
- Results first posted 2018-02-07.
- 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
NCT01702896 is a Phase II, single-group trial of interleukin-2 in metastatic melanoma, sponsored by Western Regional Medical Center. The registry records enrollment of 8, status as terminated, a start of 2012-09, and primary completion of 2014-12. The official title describes moderate-dose bolus interleukin-2.
The primary endpoint is Response Rate using RECIST v1.0, with radiographic assessment after every 2 cycles (6 weeks), until progression or death from any cause, up to 2 years. Median duration of response and median survival are listed as secondary outcomes. The primary-outcome population is marked “Data not collected,” and 0 analyses are listed. No efficacy outcome results are reported in the registry record.
The adverse-event fields list 0 affected among 8 at risk for serious events and 0 affected among 8 at risk for other events. The deaths field is null, and no top serious-event terms are listed. The reason for termination and the detailed analysis plan are not reported in the registry record.