Allogeneic Cellular Vaccine 1650-G for Non-Small Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Allogeneic Cellular Vaccine 1650-G for Non-Small Cell Lung Cancer (NCT00654030), a na phase 2 study. The primary outcome is Number of Participants Responding to the Vaccine.

2. Clinical Question

This is a question about a measured immune response, not a direct comparison of the vaccine with another treatment. The endpoint description says the number of people with a response would approximate the vaccine’s biologic efficacy. That framing is important: a response on an immune assay is the outcome specified in the record, while clinical outcomes such as tumor control or survival are not reported as the primary endpoint.

The registry record does not report how many participants met the response definition, the percentage who responded, or an estimate of uncertainty around either result. It also does not report an analysis population or explain how participants without an evaluable immune measurement were handled. As a result, the clinical question can be stated from the planned endpoint, but the posted record does not answer it with an observed response result.

3. Trial Design

This is a completed Phase 2 study of the 1650-G vaccine for non-small cell lung cancer. Its model is single group, and allocation is listed as not applicable. The record therefore describes participants receiving the vaccine without a separate control arm or a randomized allocation between treatments. The enrollment is 12.

The intended population was people with Stage I or Stage II non-small cell lung cancer after completion of initial definitive therapies. The intervention description specifies 2 injections given 4 weeks apart, with a total of 52 weeks on study. The primary endpoint is scheduled at 16 weeks after vaccination. The record does not give further details about visit timing, treatment adherence, or the timing of assessments other than the primary endpoint timeframe.

The study start is recorded as 2006-10, primary completion as 2009-11, and status as completed. Completion status means that the registry record marks the study as completed; it does not supply the missing primary outcome result or establish that all enrolled participants had evaluable endpoint data.

Because there is no control group, the design cannot provide a randomized, like-for-like comparison with another treatment or with no vaccine. A single-group result, if reported, would describe observations in participants receiving the vaccine, but other influences on those observations would not be separated through a concurrent control arm. The registry record does not report a protocol rationale for the design, a sample-size justification, or further details about enrollment and follow-up procedures.

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

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 Responding to the Vaccine, measured over 16 weeks after vaccination.

The primary endpoint is the number of participants responding to the vaccine, assessed 16 weeks after vaccination. The record describes the response as an immunologic response measured by IFN-ELISPOT. A responder is defined as having a >2 SD increase from pre-vaccine baseline levels. The endpoint is therefore based on a change in an immune assay relative to an individual’s baseline, rather than on a direct clinical outcome.

The endpoint title and unit describe a count of participants. Its description also says that the result will be reported as the percent of patients responding. These are two ways of expressing the same type of endpoint: a count gives the number meeting the response criterion, while a percentage expresses that count relative to a specified population. The registry record does not report the observed count, percentage, or denominator used for an analyzed result.

The stated rationale for using this measure is that the number of individuals with the specified immune response would provide an approximation of biologic efficacy. In general, an immune assay can indicate whether a measurable immune change occurred, but that measurement alone does not establish a clinical benefit. The record does not report a primary endpoint result or show how the assay result relates to clinical outcomes in this study.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods determine how measurements are converted into estimates and how uncertainty is represented. The registry record contains a defined primary endpoint and a study design, but it does not report an analysis method or an analysis result. A method can be explained in general terms without assuming that it was used in this study.

What does the primary endpoint count?

The endpoint is a count of participants meeting the IFN-ELISPOT response definition at 16 weeks after vaccination. The definition is a >2 SD increase from pre-vaccine baseline levels. To interpret a count, readers need to know which participants were eligible for the endpoint analysis and how many had an evaluable measurement. The record does not report those analysis-population details or an observed count.

When a count is expressed as a percentage, the denominator is essential. A percentage among all enrolled participants answers a different descriptive question from a percentage among only those with an evaluable assay. The record says the response is to be reported as a percent, but it does not report a percentage or specify an analyzed denominator. No rate can be reconstructed from the enrollment figure alone.

What would uncertainty mean?

In general, a confidence interval describes a range of values compatible with the observed data under a specified statistical method. For a participant response proportion, it would help show how much uncertainty remains around the estimated response frequency. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. An interval does not remove the limitations of the study design, and it cannot establish a causal treatment effect in a single-group study.

The registry record does not report a confidence interval or a method for calculating one. Since the endpoint result itself is not reported, the uncertainty around that result cannot be assessed. The enrollment count does not reveal the number of participants with a completed assay or the distribution of response outcomes.

Why does the single-group design matter?

In general, a concurrent control group allows a direct comparison under the same study conditions. Random allocation can help balance known and unknown participant characteristics across groups. Here, the registry describes a single-group model and allocation as not applicable. There is no randomized comparator in the record, so a treatment contrast cannot be estimated from a comparison between arms.

Without a control group, observed changes may reflect the intervention, the natural course of illness, prior or concurrent care, measurement variability, or other influences. These are general interpretive possibilities, not claims that any one influence occurred in this study. A single-group immune response count can describe the observed participants if reported, but it cannot isolate the vaccine’s effect from those alternatives.

How should planning illustrations be read?

In general, a sample-size calculation asks how many participants a future study might need under specified assumptions about the effect, significance level, allocation, and statistical model. The assumptions in the registry material describe a planning illustration using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The illustrated effect is described as a stated fraction of an observed effect. Such calculations are hypothetical planning tools, not observed power and not evidence that a study had a particular probability of detecting an effect.

Those assumptions describe a two-arm planning setup, whereas this trial is recorded as single group with no comparator arm. The registry record does not report the actual sample-size justification or the protocol’s statistical plan. Nor does it report an observed primary endpoint effect that would allow readers to interpret an effect-based illustration as a result from this trial.

Why can over-dispersion matter?

In general, over-dispersion occurs when variability in count data is greater than a statistical model assumes. If a method assumes less variation than the data contain, standard errors and uncertainty estimates can be too small. Appropriate modeling depends on the outcome, the design, and the data available. The registry record does not report an over-dispersion assessment or a count model. The primary endpoint is described as a participant count, but that description alone does not establish which statistical distribution or model was used.

For this study, the central methodological limitation is not a choice among reported models: no primary analysis is recorded. The endpoint definition and timeframe are available, but the observed result, analysis population, missing-data approach, uncertainty estimate, and inferential method are not reported in the registry record.

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("1650-G ARM")
n      <- c(11)
events <- c(6)
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  1650-G ARM  11  6  54.5  23.4  83.3  28.0  78.7  27.7  78.9

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and identifies the immune-response count as the primary endpoint. That is useful for identifying the stated focus, but it does not establish how many outcomes, time points, or statistical tests were considered in the full study. A multiplicity strategy—such as a prespecified approach for controlling the chance of false-positive findings across multiple tests—is not reported in the registry record. No p-values or outcome analyses are reported, so the effect of any multiplicity adjustment cannot be assessed.

Missing-data methods are also not reported in the registry record. The primary endpoint is set at 16 weeks after vaccination, but the record does not state how missing assays, missed visits, or measurements that could not be interpreted were handled. It does not report how many participants had an evaluable primary endpoint. Enrollment therefore should not be read as the endpoint denominator.

The analysis population is not described. The registry record does not state whether the primary endpoint was analyzed among all enrolled participants, those who received the vaccine, or those with a usable assay result. Those choices can affect the denominator and the interpretation of a response percentage. No sensitivity analysis addressing missing outcomes is reported.

These omissions limit assessment of both the completeness and robustness of any endpoint result. Because the record also lists zero analyses and provides no primary response result, readers cannot determine how missingness, population definitions, or multiple testing were handled in the study’s analysis.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: suicide attempt (100.0%), arthroplasty (100.0%), pneumonia (8.3%), dyspnea (8.3%).

The registry record lists 12 participants at risk for serious adverse events and reports 4 participants affected. For other adverse events, it lists 12 at risk and 12 affected. These are descriptive counts in the single vaccine arm, not comparisons against an untreated or alternative-treatment group. They should not be interpreted as evidence that the vaccine caused any particular event.

The record also lists serious adverse event terms with percentages: Suicide Attempt and Arthroplasty are each listed as 100.0%, while Pneumonia and Dyspnea are each listed as 8.3%. It does not provide event counts alongside these term-level percentages or explain their denominator. The percentages therefore cannot be translated into a reliable number of events from the term listing alone, and their meaning should not be inferred from the overall count of participants affected by serious events.

The registry record does not report deaths. Adverse-event summaries can describe what was recorded during a study, but interpretation depends on event definitions, reporting procedures, timing, and the population under observation. Those additional details are not reported in the registry record. With no comparator arm, these safety entries cannot establish whether event frequency differed from another treatment or from background risk.

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

The record’s primary question is whether participants had an IFN-ELISPOT immune response, defined as a >2 SD increase from baseline at 16 weeks after vaccination. The record explains why this endpoint was selected: the number responding was intended to approximate biologic efficacy. However, it does not report the number or percentage who responded. It therefore provides no observed primary result to interpret.

Even a reported immune response would need to be interpreted as an assay-based outcome. In general, evidence that an immune measurement changed is not equivalent to evidence that a clinical outcome changed. The registry record does not present a primary endpoint result or a clinical outcome result that would connect the assay response to patient outcomes.

The study is recorded as single group, with no comparator arm. Consequently, the design does not support a randomized comparison with another treatment or with no vaccine. A response observed in a single group, if available, could describe those participants but could not by itself distinguish an intervention effect from other possible influences. The record does not report a causal-effect estimate.

The safety figures describe recorded adverse events in the vaccine arm. They do not establish causation or comparative safety, and the term-level percentages are not accompanied by event counts or a denominator explanation. The record also does not report deaths.

In practical statistical terms, the registry record provides design and endpoint definitions but not the analysis needed to quantify the primary outcome. It reports enrollment of 12 and completed status, but those details do not establish how many participants contributed an evaluable assay result. The record does not provide an estimate, precision measure, or statistical test for the primary endpoint. The available information therefore supports description of what the study set out to measure, not a conclusion about the observed immune-response frequency or a treatment effect.

14. Why This Trial Matters Statistically

This trial illustrates why a clearly named endpoint is not the same as a reported result. The registry record defines a measurable immune response, specifies its threshold and assessment timeframe, and describes the population and vaccine schedule. Yet it does not report the primary response count, percentage, or analysis. Readers can identify the intended question but cannot judge its observed answer or precision.

The single-group model also matters for interpretation. Without a concurrent control, there is no randomized comparison that can separate the vaccine-associated observations from other influences. This limitation applies even when an endpoint is measured objectively: measurement precision and causal interpretation are different issues. The record’s framing of the assay as an approximation of biologic efficacy makes it especially important not to treat an immune measurement as a clinical outcome.

The safety entries provide another useful statistical lesson. Counts and percentages need clear definitions and denominators to be interpretable. The registry reports participants at risk and affected, as well as term-level percentages, but does not explain the denominator for those terms or give associated term counts. Such summaries can alert readers to recorded events, but they do not support a comparative risk estimate or establish causation.

Finally, planning calculations should be distinguished from observed evidence. The stated assumptions describe hypothetical planning under a two-arm allocation model, while the actual study is recorded as single group. Planning illustrations cannot replace a reported endpoint analysis, and they should not be read as observed power. This case underscores the value of reporting the analysis population, outcome count, uncertainty, missing-data handling, and statistical methods alongside a registry endpoint definition.

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.

Several limitations are specific to the information recorded for this study. The registry lists enrollment of 12, but does not report how many participants completed the primary immune assay or contributed to an analysis. The primary response count and percentage are absent, as are an effect estimate and measure of uncertainty. The record lists zero analyses, so the primary question cannot be answered from the posted results.

The single-group design has no randomized comparator. It cannot support a like-for-like comparison with another treatment, and any observed response would be descriptive of the participants measured rather than an isolated causal effect. The registry record does not report further details of eligibility, the analysis population, adherence, or the handling of missing assays. It also does not report the sample-size justification or the study’s statistical plan.

The record identifies an immune assay response rather than a clinical outcome as the primary endpoint. It describes the response as an approximation of biologic efficacy, but does not report a primary result or establish a link between that measure and clinical outcomes. The safety summaries are likewise limited: the record does not report deaths, and the term-level serious adverse event percentages are not accompanied by event counts or a denominator explanation.

Although the record marks the study completed, that status does not supply missing outcome analyses. The registry record does not report whether all enrolled participants completed the scheduled assessment or how any incomplete follow-up was addressed. These limitations prevent assessment of the observed endpoint frequency, its precision, and the robustness of any treatment-related interpretation.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Allogeneic Cellular Vaccine 1650-G for Non-Small Cell Lung Cancer — NCT00654030.
• Registry record last updated 2018-04-09; status last verified 2018-03.
• Results first posted 2013-05-20.
• 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

NCT00654030 is a completed Phase 2, single-group study of the 1650-G vaccine for non-small cell lung cancer. The intended population was patients with Stage I or Stage II disease after initial definitive therapies. The arm description specifies 2 injections 4 weeks apart and a total of 52 weeks on study.

The primary endpoint was the number of participants with an IFN-ELISPOT response at 16 weeks after vaccination, defined as a >2 SD increase from pre-vaccine baseline levels. The registry record lists enrollment of 12 but does not report a primary response count, percentage, analysis, or uncertainty estimate. It lists 4 participants affected by serious adverse events among 12 at risk and 12 affected by other adverse events among 12 at risk. Deaths are not reported in the registry record.

The record does not describe an analysis population, missing-data method, multiplicity strategy, or statistical plan. Its single-group design does not provide a randomized comparator, and the posted information does not establish a treatment effect.