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 Pilot hu14.18-IL2 in Resectable Recurrent Stage III or Stage IV Melanoma (NCT00590824), a randomized phase 2 study. The primary outcome is Ganglioside Expressed by Tumor Cells (GD2).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00590824 |
| Phase | Phase 2 |
| Condition | Melanoma |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Wisconsin, Madison |
| Results posted | Yes |
2. Clinical Question
Population
People with completely resectable recurrent stage III or stage IV melanoma were eligible for the study described in the title. The registry lists melanoma as the condition and records enrollment of 23 participants. For the primary GD2 assessment, 12 participants had evaluable tumor samples.
Intervention
Both study arms involved hu14.18-IL2 and surgical resection, but the order differed. In Group A, hu14.18-IL2 preceded resection, followed by hu14.18-IL2. The registry describes Group B as resection followed by hu14.18-IL2 and then hu14.18-IL2.
Comparator
The comparison was between those two treatment sequences. The registry records a randomized, parallel-group design with no masking. It does not describe a placebo or a group receiving no hu14.18-IL2.
Primary question
The primary endpoint was histological assessment of antitumor activity using tumor-cell GD2 expression after surgical resection. Results were classified as positive when GD2 expression was high or low/moderate, and negative when expression was undetectable. The registry also states that the study aimed to evaluate time to recurrence and overall survival, but results for these aims are not reported in the registry record.
3. Trial Design
The registry describes a phase 2 randomized trial with a parallel design and no masking. Enrollment was 23. The sponsor was the University of Wisconsin, Madison, and the listed condition was melanoma. The record gives a start date of 2007-12-17, a primary completion date of 2018-09-20, and a status of completed. These dates describe the registry record; they do not by themselves explain how long an individual participant was followed.
The two arms differed in the sequence of hu14.18-IL2 and resection. Group A is described as hu14.18-IL2, then resection, then hu14.18-IL2. Group B is described as resection followed by hu14.18-IL2 and then hu14.18-IL2. The registry does not report dosing details, the interval between study procedures, or how allocation was generated or concealed. Randomization is recorded, but the allocation ratio is not reported in the registry record.
The primary endpoint was a histological assessment after resection, with a stated timeframe of up to 1 week. Twelve participants had evaluable tumor samples for GD2 analysis. Other listed outcomes included C-reactive protein, lymphocyte count, anti-idiotypic antibodies, anti-Fc-IL2 antibodies, and in vitro soluble IL2 receptor alpha levels. The registry lists 16 outcome measures overall. The analysis population definitions, participant flow by arm, and details of follow-up 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: Protocol amended to drop cilengitide groups (C and D), proceed with enrollment and randomization into hu14.18- IL2 groups.
Overall Study
| Stage | Group A | Group B | Group C | Group D |
|---|---|---|---|---|
| STARTED | 11 | 9 | 2 | 1 |
| COMPLETED | 11 | 7 | 1 | 0 |
| NOT COMPLETED | 0 | 2 | 1 | 1 |
Reasons for not completing this period:
| Reason | Group A | Group B | Group C | Group D |
|---|---|---|---|---|
| Disease could not be resected | 0 | 2 | 1 | 0 |
| Adverse Event | 0 | 0 | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: There were 12 participants with evaluable tumor samples for GD2 analysis.
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: Groups C and D were suspended due to toxicity and removed from the study design in a subsequent protocol amendment.
| Characteristic | Unit | Group A | Group B | Group C | Group D | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 11 | 9 | 2 | 1 | 23 | |
| Age, Continuous | years | 46 (21 to 65) | 52 (38 to 69) | 56 (51 to 61) | 67 (67 to 67) | 47 (21 to 69) |
| Sex: Female, Male: Female | Participants | 5 | 2 | 1 | 0 | 8 |
| Sex: Female, Male: Male | Participants | 6 | 7 | 1 | 1 | 15 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 0 | 0 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 | 9 | 2 | 1 | 22 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 10 | 9 | 2 | 1 | 22 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 0 | 0 | 1 |
| Region of Enrollment: United States | participants | 11 | 9 | 2 | 1 | 23 |
| ECOG PS: 0 | Participants | 9 | 7 | 2 | 1 | 19 |
| ECOG PS: 1 | Participants | 2 | 2 | 0 | 0 | 4 |
| Disease Extent: Extensive | Participants | 4 | 2 | 1 | 0 | 7 |
| Disease Extent: Non-extensive | Participants | 7 | 7 | 1 | 1 | 16 |
| Disease Stage: III | Participants | 7 | 6 | 0 | 1 | 14 |
| Disease Stage: IV | Participants | 4 | 3 | 2 | 0 | 9 |
| Histology: Cutaneous melanoma | Participants | 10 | 8 | 2 | 1 | 21 |
| Histology: Unknown melanoma primary | Participants | 1 | 0 | 0 | 0 | 1 |
| Histology: Subungual melanoma | Participants | 0 | 1 | 0 | 0 | 1 |
| Prior Therapy: Granulocyte-macrophage colony stimulating factor | participants | 1 | 0 | 0 | 0 | 1 |
| Prior Therapy: Chemotherapy multiple agent | participants | 0 | 1 | 0 | 0 | 1 |
| Prior Therapy: Interferon alpha-2b | participants | 7 | 5 | 1 | 1 | 14 |
| Prior Therapy: No prior therapy | participants | 1 | 0 | 0 | 0 | 1 |
| Prior Therapy: Radiation therapy | participants | 1 | 2 | 0 | 0 | 3 |
| Prior Therapy: Surgery | participants | 10 | 9 | 2 | 1 | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Ganglioside Expressed by Tumor Cells (GD2), measured over up to 1 week.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint assessed GD2 expression in tumor cells using histological analysis after surgical resection. The registry describes the assessment as an indicator of antitumor activity. It grouped findings into positive, meaning GD2 expression was high or low/moderate, and negative, meaning expression was undetectable. The stated timeframe was up to 1 week.
This kind of endpoint turns a tissue observation into categories that can be counted. The categories describe whether GD2 expression was detected at the levels specified in the record; they are not, by themselves, a measure of time to recurrence or overall survival. Those outcomes were mentioned among the study aims, but their results are not reported in the registry record.
Twelve participants had evaluable tumor samples for the GD2 analysis, while trial enrollment was 23. The registry does not report the positive and negative counts, or explain why samples were not evaluable for the remaining participants. Without the category counts, the direction and size of any difference between study arms cannot be described from the registry record. The endpoint definition is available, but the numerical primary result is not.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The study record establishes the design and the intended primary measurement, but it does not provide the GD2 result counts. The primary endpoint classified post-resection tumor samples as positive or negative according to the stated GD2 criteria. Although 12 participants had evaluable samples, the numbers in each category and their distribution by study arm are not reported in the registry record. No numerical comparison of the two sequences can therefore be interpreted from the recorded primary result.
The trial enrolled 23 participants and is marked completed. Completion status does not establish that all planned outcomes were analyzed or reported. The registry lists 16 outcome measures and no analysis entries. The listed study aims also included time to recurrence and overall survival, but estimates or event counts for those outcomes are not reported in the registry record. Likewise, the listed secondary measures do not have numerical results in the information recorded.
In statistical interpretation, an effect estimate describes the observed difference or association, while uncertainty measures such as a confidence interval indicate how compatible the data are with a range of values under the analysis assumptions. Neither an effect estimate nor a confidence interval for GD2 is reported in the registry record. There is also no reported p-value or arm-specific primary outcome count. Consequently, the precision of an estimated treatment-sequence difference cannot be assessed.
The small number of evaluable specimens relative to total enrollment makes clear that the primary endpoint population is not identical to the enrolled group. That distinction matters when reading any result: the tissue endpoint can only describe participants with evaluable samples, and the record does not explain the missing evaluability or provide a broader analysis population. It would be inappropriate to treat the evaluable participants as representative of all enrolled participants without supporting information.
The posted safety information gives event counts in groups labeled A through D, along with a list of serious-event terms and percentages. The relationship between these safety groups and the randomized treatment arms is not reported in the registry record. Their denominators and the basis for the percentages attached to the serious-event terms are also not specified. These entries can be described as registry-reported safety counts, but they do not supply the missing primary efficacy results.
9. Statistical Methods Explained
The registry identifies the study as randomized, parallel-group, and unmasked. These design labels describe how the trial was organized, but they do not provide the details needed to reproduce its statistical analysis. The method used to generate the random sequence, conceal allocation before assignment, or handle departures from assigned treatment is not reported in the registry record.
What does randomization contribute?
In general, randomization assigns participants to study groups by chance. When implemented and maintained appropriately, it can reduce systematic differences between groups at the start of a trial. It does not guarantee that groups will be identical, particularly in a small study, and it does not address missing outcome measurements by itself. The registry records randomization but does not report baseline characteristics by arm or the allocation procedure.
How should the endpoint be read?
The primary outcome is categorical: the histological GD2 finding was summarized as positive or negative using the thresholds described in the record. A comparison of categorical outcomes would ordinarily require the category counts in each group and a clearly stated analysis method. Those counts and the method are not reported in the registry record. The endpoint was assessed after surgical resection, with a stated timeframe of up to 1 week; the record does not give more detail about the exact assessment schedule or handling of specimens that could not be evaluated.
What can a confidence interval tell a reader?
In general, a confidence interval expresses uncertainty around an estimated quantity under a specified statistical model and procedure. A narrow interval indicates greater precision than a wide interval, while an interval that spans values compatible with materially different conclusions may leave the result uncertain. No confidence interval or primary effect estimate is reported in the registry record, so precision cannot be judged for the GD2 comparison. A p-value, even if available, would not replace an effect estimate or its uncertainty.
What does over-dispersion mean?
In general, over-dispersion occurs when observed variability is greater than a statistical model expects. It can make standard errors too small if a model assumes less variability than the data contain. The registry does not report an over-dispersion assessment or a count model for this endpoint. The primary outcome is described as participant counts in categories, but no model or dispersion estimate is reported.
What do the planning assumptions mean?
The sample-size planning assumptions describe an illustration, not a result observed in participants and not the trial's own design justification. They specify a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The planning effect is described as a stated fraction of the observed effect. For time-to-event planning, the stated quantity is the total number of events using the Schoenfeld method; for other outcomes, the stated quantity is participants per arm. These are planning conventions, not evidence that a particular power was achieved. The registry record does not report the trial's own sample-size justification, and the planning values cannot be interpreted as observed power.
The record does not report a statistical analysis plan, model specifications, adjustment variables, interim analyses, or multiplicity procedures. It lists 16 outcome measures and no analysis entries. Without a defined analysis and the endpoint values, no inferential result can be reconstructed from the record. Any recalculated estimates, if available from posted summary statistics, would be unadjusted recalculations and would not substitute for the sponsor's own analysis when a discrepancy is recorded. No such primary analysis is reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 16 outcome measures, including the primary GD2 endpoint and secondary laboratory or immune measures. When a study examines multiple outcomes, the chance of at least one apparently unusual result can increase if each is assessed separately without an appropriate analysis plan. In general, multiplicity procedures may control that risk, but the choice depends on the scientific questions and planned comparisons. A multiplicity strategy is not reported in the registry record.
The record contains no analysis entries. It therefore does not document which outcomes were analyzed, which comparisons were prespecified, or whether any adjustments were used. The primary endpoint is described in two categories, and the record notes that the outcome has several categories or time points and was not reduced to a single comparison. The numerical category results are not reported in the registry record.
Missingness is also important. Twelve participants had evaluable tumor samples for GD2 analysis, out of 23 enrolled. The record does not explain why other samples were not evaluable, whether missingness differed by arm, or whether any statistical method was used to address it. It does not report analysis populations such as intention-to-treat or per-protocol sets, nor does it give participant flow by arm. These omissions limit assessment of how the endpoint population relates to all randomized participants.
The planned or actual handling of missing secondary outcomes, withdrawals, and incomplete follow-up is not reported in the registry record. Results for time to recurrence and overall survival are also not reported. Thus neither the impact of missing data nor the potential for selective outcome reporting can be assessed from the recorded information.
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 |
|---|---|---|---|
| Group A | 2 | 11 | 18.2% |
| Group B | 2 | 9 | 22.2% |
| Group C | 0 | 2 | 0.0% |
| Group D | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Group A | 4 of 11 |
| Group B | 5 of 9 |
| Group C | 1 of 2 |
| Group D | 1 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 |
|---|---|
| Group A | 10 of 11 |
| Group B | 7 of 9 |
| Group C | 2 of 2 |
| Group D | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Group A | Group B | Group C | Group D |
|---|---|---|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | 1 of 11 | 1 of 9 | 0 of 2 | 0 of 1 |
| Pain - Head / Headache | General disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Vomiting | Gastrointestinal disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Infection | Infections and infestations | 0 of 11 | 1 of 9 | 0 of 2 | 0 of 1 |
| Neuropathy | Nervous system disorders | 0 of 11 | 0 of 9 | 0 of 2 | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Group A | Group B | Group C | Group D |
|---|---|---|---|---|---|
| Lymphopenia | Blood and lymphatic system disorders | 10 of 11 | 7 of 9 | 2 of 2 | 1 of 1 |
| Hyponatremia | Metabolism and nutrition disorders | 1 of 11 | 1 of 9 | 0 of 2 | 0 of 1 |
| Pruritus/itching | Skin and subcutaneous tissue disorders | 0 of 11 | 2 of 9 | 0 of 2 | 0 of 1 |
| Pain | General disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Seroma | Musculoskeletal and connective tissue disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Hypocalcemia | Metabolism and nutrition disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Metabolic / Lab | Metabolism and nutrition disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Metabolic / Lab | Metabolism and nutrition disorders | 1 of 11 | 0 of 9 | 0 of 2 | 0 of 1 |
| Syncope | Nervous system disorders | 0 of 11 | 1 of 9 | 0 of 2 | 0 of 1 |
| Hyperbilirubinemia | Metabolism and nutrition disorders | 0 of 11 | 1 of 9 | 0 of 2 | 0 of 1 |
Serious events with the highest arm-level rate: neuropathy (100.0%), lymphopenia (11.1%), infection (11.1%), pain - head / headache (9.1%), vomiting (9.1%).
The registry safety entries use groups labeled A, B, C, and D. For serious events, the number affected and the number at risk are listed as Group A: 2 and 11; Group B: 2 and 9; Group C: 0 and 2; and Group D: 1 and 1. For other events, the corresponding affected and at-risk counts are Group A: 10 and 11; Group B: 7 and 9; Group C: 2 and 2; and Group D: 1 and 1. Death counts are listed as 4, 5, 1, and 1, respectively. The registry does not report how these safety groups correspond to the randomized arms or give further context for the death counts.
The listed serious-event terms include Neuropathy at 100.0%, Lymphopenia at 11.1%, Infection at 11.1%, Pain - Head / Headache at 9.1%, and Vomiting at 9.1%. The denominator and calculation basis for these percentages are not reported in the registry record. They should not be treated as arm-specific risks or compared directly without that information. Safety counts describe events recorded in the registry; they do not establish causation or provide a complete account of timing, severity, or resolution.
12. What the Result Does, and Does Not, Mean
The record describes a randomized comparison of two sequences involving hu14.18-IL2 and surgical resection in people with completely resectable recurrent stage III or stage IV melanoma. It defines the primary endpoint as a post-resection histological assessment of GD2 expression and states that 12 participants had evaluable samples. These details clarify what the trial set out to measure and the population for which the tissue result was available.
They do not show the primary outcome result. The positive and negative GD2 counts, arm-specific results, and a treatment-sequence effect estimate are not reported in the registry record. As a result, the record cannot establish the direction, size, or precision of any difference in the primary endpoint. The stated aims of assessing time to recurrence and overall survival also cannot be evaluated from the recorded information because their results are not reported.
In general, the absence of reported results is not evidence that an outcome was absent, unchanged, or statistically inconclusive. It means the result cannot be assessed from this record. Similarly, a completed status does not establish that every planned analysis was completed or published. The listing of safety events is informative as a record of reported counts, but the group mapping and percentage denominators are unspecified, limiting direct comparisons.
The sample-size assumptions describe planning illustrations rather than evidence about the observed study's statistical power. A planning calculation based on an observed effect is conditional on that effect and the stated assumptions; it does not establish how likely the trial was to detect a true effect. No trial-specific sample-size justification or primary inferential analysis is reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because its primary question concerns a biological measurement in tumor tissue, while the registry also describes clinical aims involving recurrence and survival. These are distinct kinds of outcomes. A histological classification can document GD2 expression in evaluable specimens, whereas time-to-event outcomes require information about follow-up and events. The record identifies these aims but does not report their numerical results.
The contrast between enrollment of 23 participants and 12 evaluable tumor samples highlights why analysis populations matter. A result based on evaluable tissue would not automatically describe every enrolled participant. Understanding the difference requires participant flow, reasons for unevaluable samples, and a prespecified approach to missing data, none of which is reported in the registry record.
Randomization provides a framework for comparing the two treatment sequences, but interpretation also depends on the allocation procedure, outcome assessment, and statistical analysis. The record reports no masking and does not report a detailed analysis plan. The absence of primary category counts and analysis entries prevents readers from assessing the comparison's magnitude and uncertainty.
Finally, the safety record illustrates the importance of denominators and group definitions. Counts and percentages may appear precise, but their interpretation depends on knowing which participants are represented and how each percentage was calculated. The registry entries allow a limited description of recorded events, while leaving those contextual details unresolved. This makes careful separation of design details, endpoint definitions, and reported results essential.
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 registry record leaves several trial-specific questions unresolved. It does not report the positive and negative GD2 counts, an arm-by-arm primary endpoint summary, an effect estimate, or uncertainty for the primary comparison. Although the record states that 12 participants had evaluable samples, it does not explain why the other enrolled participants lacked evaluable samples or whether this differed across arms.
The record does not give a participant-flow account, baseline characteristics by randomized group, or definitions of the analysis populations. It also does not describe the randomization procedure, allocation concealment, treatment dosing, or detailed timing of interventions. No statistical analysis plan, interim-analysis information, or sample-size justification is reported in the registry record. The sample-size assumptions are planning illustrations and cannot fill those gaps.
Results for the stated aims of time to recurrence and overall survival are not reported in the registry record. The record lists secondary outcomes but does not provide their numerical results or analysis details. It lists 16 outcome measures and no analysis entries, limiting assessment of which analyses were carried out and how multiple outcomes were handled.
Safety interpretation has separate limitations. The relationship between the safety groups labeled A through D and the randomized arms is not reported, and the denominators for the listed serious-event percentages are not specified. The record also provides no event timing, duration, or resolution details. These limitations prevent a complete assessment of comparative safety or the broader clinical meaning of the posted counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Pilot hu14.18-IL2 in Resectable Recurrent Stage III or Stage IV Melanoma — NCT00590824.
- Registry record last updated 2019-11-21; status last verified 2019-11.
- Results first posted 2019-10-22.
- 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
This completed phase 2 trial enrolled 23 participants with melanoma and is recorded as randomized, parallel-group, and unmasked. It compared two sequences involving hu14.18-IL2 and surgical resection. The sponsor was the University of Wisconsin, Madison. The record lists a start date of 2007-12-17 and a primary completion date of 2018-09-20.
The primary endpoint was post-resection histological assessment of tumor-cell GD2 expression, classified as positive or negative. Twelve participants had evaluable tumor samples. The registry record does not report the primary category counts or a statistical comparison. It lists 16 outcome measures, including secondary laboratory and immune measures, and no analysis entries. Results for recurrence and overall survival are not reported in the registry record. Safety counts are listed for four groups, but their relation to the randomized arms and the denominators for serious-event percentages are not specified.