Universal Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)-Producing and CD40L Expressing Bystander Cell Line for Tumor Vaccine in Melanoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Universal Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)-Producing and CD40L Expressing Bystander Cell Line for Tumor Vaccine in Melanoma (NCT00101166), a na phase 2 study. The primary outcome is Number of Participants With Partial Response.

2. Clinical Question

3. Trial Design

This was a Phase II, single-group trial of a vaccine for patients with malignant melanoma. The registry lists H. Lee Moffitt Cancer Center and Research Institute as sponsor, gives the status as completed, and records enrollment of 43. The primary outcome description, however, specifies an analysis population of all 28 participants who were vaccinated. The registry record does not explain why those figures differ, so the enrollment figure should not be treated as the number evaluated for the primary outcome.

The intervention was an autologous tumor cell-based vaccine formulated with a GM-CSF-producing and CD40L-expressing bystander cell line. The treatment description specifies intradermal injections at 28-day intervals for a total of 3 immunizations, on Days 1, 29, and 57. The listed primary outcome concerns partial response. Secondary outcomes include serious adverse events related to treatment, stable disease, time to progression, and overall survival.

The study was not randomized and had no control arm. Masking was recorded as none. These features are important when interpreting any response or safety count: outcomes in a single group describe what occurred among treated participants, but cannot by themselves establish what would have happened to comparable patients without the intervention or under another treatment. The registry record does not report a protocol-level statistical plan, a sample-size justification, or an analysis comparing the vaccine group with a control group.

The study began in 2004-10 and reached primary completion in 2010-03. The registry lists 5 outcome measures and no analysis entries. Although the primary outcome definition and analysis population are recorded, the primary response count and associated statistical estimate 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.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All 28 participants who were vaccinated on this study.

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Partial Response, measured over Average of 14 months.

The primary endpoint is a count: the number of vaccinated participants who had a partial response. It is not a change in an average tumor measurement, nor is it a comparison between treatment groups. The registry says response and progression were evaluated using international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) Committee. Its definition of partial response requires at least a 30% decrease in the sum of the longest diameter of target lesions, using the baseline sum as the reference.

A response count is useful because it translates a prespecified tumor-response definition into an outcome that can be tallied across participants. In general, a clear operational definition helps readers understand which clinical observations qualify as events. The registry identifies the outcome population as all 28 participants vaccinated in the study and gives an average timeframe of 14 months.

The count is not reported in the registry record. Without it, readers cannot determine how many vaccinated participants met the response definition, estimate the response proportion, or assess the precision of that estimate. No such estimate should be inferred from the endpoint definition, enrollment, or safety results. The registry also does not report the timing or individual pattern of response assessments beyond the stated average timeframe.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a single-group Phase II study, not a randomized comparison. Statistical interpretation therefore begins with the distinction between describing an outcome in a treated group and estimating a difference between treatments. A single-group count can describe the participants observed, but there is no concurrent comparison group in this design to provide a direct estimate of what might have happened under an alternative. General statistical adjustment cannot create a control group that the design did not include.

What does the primary count represent?

The primary endpoint is the number of vaccinated participants meeting the partial-response definition. The registry defines that response using RECIST criteria: at least a 30% decrease in the sum of the longest diameter of target lesions relative to baseline. The outcome is thus a participant count based on a stated classification rule. It is not a continuous tumor measurement or a time-to-event estimate. The stated primary population is all 28 participants vaccinated in the study, and the timeframe is an average of 14 months.

For a binary outcome, each participant is generally classified as having or not having the event under the endpoint definition. A response proportion can be estimated when both the event count and the relevant denominator are available. Uncertainty around such an estimate is commonly expressed with a confidence interval. In general, a confidence interval describes the range of values compatible with the data under the method’s assumptions; it does not remove design limitations or establish causation. Here, the response count is not reported in the registry record, so a response proportion or its confidence interval cannot be stated.

Why does the design matter?

In general, random allocation helps make comparison groups similar in expectation with respect to factors that could influence outcomes. This trial has no randomized comparator: allocation is listed as NA, the model is single group, and the masking entry is none. Accordingly, any observed outcome would be descriptive of the participants studied. It would not, on its own, show that the vaccine caused the outcome or quantify how results differ from another treatment.

Masking and randomization address different concerns. Randomization concerns how participants are assigned to groups; masking concerns awareness of treatment or assessment. The record reports no masking and no comparator group. The registry does not provide further details about outcome-assessment procedures, so no additional safeguards should be assumed.

How should safety counts be read?

The safety information lists participants at risk and participants with events in separate categories. The registry reports 0 participants with serious adverse events related to study treatment among 28 at risk. It also reports 10 participants with other adverse events among 28 at risk. These counts should be read according to their recorded labels; they do not establish the absence of serious events unrelated to treatment, and they do not indicate how many events each participant experienced.

In general, event counts and participant counts are not interchangeable. A participant may have more than one event, while a count of participants with at least one event records each person in that category once. The registry’s labels specify numbers of participants, so they should not be restated as numbers of individual event occurrences. Without definitions for the “other” category or additional event details, its clinical seriousness and composition cannot be inferred.

What is not documented?

The registry lists 5 outcome measures but no analysis entries. It does not report the primary response count, an estimate with uncertainty, a p-value, or a statistical model for the primary outcome. It also does not report a sample-size justification, interim analyses, or a detailed statistical analysis plan. These absences limit assessment of how the endpoint was analyzed and whether the analysis followed a prespecified plan.

General planning calculations based on an observed effect are not the same as a trial’s original design justification. A sample-size illustration is a hypothetical planning aid, not observed power and not evidence that the trial was adequately or inadequately powered. The registry record does not report its own sample-size rationale. Likewise, no model-based adjustment, subgroup analysis, or handling of incomplete observations should be presumed when it is not reported.

Over-dispersion is a concern in some count-data settings when variability exceeds what a chosen probability model expects. It is not a reason to invent a model for this endpoint: the primary outcome is a participant count for a binary response classification, and the registry record gives no fitted analysis. More generally, statistical methods should match the outcome and design, but the actual method cannot be identified from the outcome definition alone.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Vaccine Therapy")
n      <- c(28)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Vaccine Therapy  28  1  3.6  0.1  18.3  0.6  17.7  0.8  17.8

11. Multiplicity, Missing Data and Analysis Populations

The registry lists the primary partial-response outcome alongside secondary outcomes for serious adverse events related to treatment, stable disease, time to progression, and overall survival. It also records 5 outcome measures and no analysis entries. The registry record does not report a multiplicity plan, such as whether any adjustment was intended for interpreting several outcomes, or which outcomes were considered confirmatory. Without that information, the relative inferential status of the listed outcomes cannot be established.

The primary response count itself is not reported in the registry record. No missing-data method, imputation approach, or sensitivity analysis is reported. The record identifies all 28 vaccinated participants as the primary outcome population, but does not say whether every participant had an evaluable response assessment or how incomplete assessments were handled. It would be inappropriate to assume that participants without reported response data were classified as nonresponders or excluded.

Enrollment is listed as 43, while the primary outcome description refers to 28 vaccinated participants. The registry record does not explain this difference or provide a participant flow accounting for enrollment, vaccination, follow-up, and endpoint availability. The reason for the difference is therefore not reported in the registry record.

The registry’s analysis entries are absent, and it does not report a detailed statistical plan, interim analyses, or a sample-size justification. These omissions prevent a reader from checking whether analysis populations and methods were prespecified or how multiple outcomes and incomplete observations were addressed. They do not establish that no such procedures were used; they establish only that they are not reported in the registry record.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent non-serious adverse event terms

The registry reports 0 participants with serious adverse events related to study treatment among 28 at risk. It separately reports 10 participants with other adverse events among 28 at risk. These are participant counts in distinct categories, not a complete account of every event occurrence. The record does not list serious adverse event terms, and the serious-event term list is empty.

The qualifier “related to study treatment” matters: the serious-event count concerns events classified as related, not necessarily every serious event from any cause. A zero in this category should therefore not be paraphrased as proof that no serious adverse events occurred. The registry also does not give the event types, timing, severity details beyond the category, or the criteria used to judge relatedness.

The other adverse-event count does not indicate how many events occurred per participant, what those events were, or whether they were mild or severe. The record does not provide a comparison group, so these counts cannot be used to estimate a relative safety difference. They are registry-reported counts for the vaccine group and should be interpreted within those limits.

13. What the Result Does, and Does Not, Mean

The registry record describes a completed Phase II study of a bystander-based autologous tumor cell vaccine in melanoma, including the treatment schedule, a RECIST-based partial-response definition, and selected safety counts. It also makes clear that the design was single group, with no randomized comparator and no masking. Those details help define the context in which the outcomes should be read.

The record does not report the primary partial-response count. Consequently, it cannot establish from the reported primary outcome how many vaccinated participants met the response criterion, what the response frequency was, or how precise an estimate might be. The endpoint definition and stated population do not supply the missing result. No effect estimate or statistical significance result for the primary endpoint is reported in the registry record.

The safety figures show 0 participants with serious adverse events related to treatment and 10 participants with other adverse events, each reported among 28 at risk. These are descriptive counts, not proof that all serious adverse events were absent or that one treatment is safer than another. The record gives no control group for such a comparison.

More generally, an outcome observed in a single group cannot by itself determine what caused that outcome. Even a fully reported response count would describe what was observed among the studied participants, while the lack of a comparator would limit causal interpretation. The registry also does not explain why enrollment was 43 while the primary outcome population was 28 vaccinated participants.

Accordingly, the strongest reading is a careful account of what the registry documents and what it leaves unresolved. The study’s purpose and outcome definitions are recorded, along with selected safety information. The primary response result, its statistical uncertainty, and several details needed to assess the analysis are not reported in the registry record.

14. Why This Trial Matters Statistically

This trial is statistically instructive because its outcome definition is available while its primary outcome count is not. The distinction matters: a well-defined endpoint tells readers what investigators intended to measure, but it does not reveal the result. Naming a RECIST-based partial-response criterion and a vaccinated analysis population cannot substitute for reporting the number meeting that criterion.

The design also illustrates the difference between an outcome description and a treatment comparison. A single-group study can document observations among treated participants, but without a comparator it cannot directly estimate a difference attributable to treatment. That limitation remains even if the outcome is measured consistently and the count is reported.

The record gives both an overall enrollment figure and a smaller vaccinated population for the primary outcome. Without participant flow information, readers cannot tell why those populations differ or how the difference affects interpretation. Reporting the analysis population and accounting for participants at each stage are important for understanding who contributes to an outcome estimate.

Finally, the safety information separates serious events related to treatment from other adverse events. Preserving that distinction avoids overstating what a zero related serious-event count means. The record is a useful reminder that transparent outcome reporting requires not only endpoint definitions, but also results, denominators, analysis methods, and clear descriptions of missing observations.

15. 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 that the registry record does not report the number of participants with partial response. It therefore provides no primary response estimate or associated measure of precision. The RECIST-based definition and average timeframe of 14 months clarify the intended endpoint but do not provide the result.

The single-group design offers no concurrent control for comparison, and masking is recorded as none. Outcomes cannot be interpreted as a controlled estimate of the vaccine’s effect. The registry also gives no details about response-assessment timing beyond the average timeframe, or about assessor procedures.

The enrollment figure is 43, whereas the primary outcome population is described as all 28 vaccinated participants. The registry record does not explain the difference or report participant flow, reasons for non-vaccination, follow-up completeness, or the handling of missing assessments. These details are necessary to understand how the outcome population relates to enrollment.

For safety, the record reports 0 participants with serious adverse events related to treatment and 10 with other adverse events, but does not provide event terms or a fuller account of seriousness, timing, or relatedness assessment. The empty serious-event term list does not supply those missing details.

The record lists no analysis entries and does not report a statistical plan, sample-size justification, interim analyses, multiplicity handling, or missing-data methods. These aspects of the trial’s statistical conduct are not reported in the registry record, so no conclusions about them should be inferred.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Universal Granulocyte-Macrophage Colony-Stimulating Factor (GM-CSF)-Producing and CD40L Expressing Bystander Cell Line for Tumor Vaccine in Melanoma — NCT00101166.
• Registry record last updated 2018-02-28; status last verified 2012-09.
• Results first posted 2012-11-16.
• 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.

19. Record Summary

The registry describes a completed Phase II, single-group study of a GM-CSF-producing and CD40L-expressing bystander cell line used in an autologous tumor cell-based vaccine for melanoma. The listed treatment schedule was intradermal injections at 28-day intervals on Days 1, 29, and 57. Enrollment was 43; the primary outcome population was all 28 vaccinated participants.

The primary endpoint was the number of participants with partial response under a RECIST-based definition, with an average timeframe of 14 months. The response count is not reported in the registry record. Safety information lists 0 participants with serious adverse events related to treatment and 10 participants with other adverse events among 28 at risk.

There was no randomized comparator, masking was none, and the registry lists no analysis entries. The record does not report the primary result, statistical analysis plan, sample-size justification, or explanation for the difference between enrollment and the vaccinated outcome population.