Vaccination of Patients With Renal Cell Cancer With Dendritic Cell Tumor Fusions and GM-CSF: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Vaccination of Patients With Renal Cell Cancer With Dendritic Cell Tumor Fusions and GM-CSF (NCT00458536), a non-randomized phase 1, phase 2 study. The primary outcome is Number of Participants With Adverse Events Associated With Vaccination With Mature DC/Tumor Fusion and GM-CSF.

2. Clinical Question

3. Trial Design

This completed study is registered as a Phase 1 and Phase 2 trial of a dendritic cell/tumor fusion vaccine with GM-CSF in renal cancer. The registry lists Beth Israel Deaconess Medical Center as the sponsor, enrollment of 36, a start date of 2004-10, and primary completion in 2014-12. It identifies the allocation as non-randomized, the masking as none, and the model as single group.

The intervention descriptions name the Dendritic Cell Tumor Fusion Vaccine and Granulocyte Macrophage Colony Stimulating Factor (GM-CSF). The registry describes two cohorts: Cohort 1 was treated with the vaccine to evaluate treatment-limiting toxicity, and Cohort 2 was treated with the vaccine to evaluate response. The descriptions indicate differing evaluation aims, but do not report random allocation, a control treatment, or a direct comparison between cohorts. Cohort sizes and the details of how participants entered each cohort are not reported in the registry record.

The registry’s stated rationale is to test safety and characterize side effects. It describes creating the vaccine by fusing cells removed from a participant’s tumor with dendritic cells obtained from that participant’s blood. The registry also notes animal-study rationale for the approach; that rationale is not a clinical outcome from this trial.

The primary endpoint covers the number of participants with vaccination-associated adverse events over “6 months following last dose of treatment, up to 9 months.” A secondary outcome is the number of participants demonstrating a two-fold increase in interferon gamma. The registry record does not report the detailed schedule of assessments, the precise analysis populations, or the statistical plan for comparing outcomes. With no randomized control group, the study is principally descriptive for the reported safety counts; its design does not provide a randomized treatment-effect comparison.

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: Adverse events potentially related to vaccination were largely restricted to injection site reactions. 12 of the 19 patients experienced vaccine site reactions.

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 Adverse Events Associated With Vaccination With Mature DC/Tumor Fusion and GM-CSF, measured over 6 months following last dose of treatment, up to 9 months.

The primary endpoint is the number of participants with adverse events associated with vaccination with mature dendritic cell/tumor fusion and GM-CSF. It is a count of participants with an event, rather than a measurement of how many events each participant experienced or how long an event lasted. Its timeframe is “6 months following last dose of treatment, up to 9 months.”

A safety endpoint of this kind helps describe the occurrence of adverse events during a defined observation period. Counting participants with events can communicate how many people in the assessed group were affected, while a separate account of event type and severity would be needed to characterize the clinical nature of those events more fully. The registry’s endpoint description says events potentially related to vaccination were largely restricted to injection-site reactions and reports such reactions in 12 of the 19 patients.

The endpoint title specifies events associated with vaccination, but that wording does not by itself establish that the vaccine caused every reported event. The registry record does not provide event-level timing, severity grades, duration, or a full breakdown by type for the primary endpoint. It also does not report an inferential estimate, confidence interval, or comparison group for this outcome. The endpoint therefore supports a descriptive reading of the recorded safety experience, not a causal comparison or a precise estimate of risk beyond the reported group.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a count endpoint describe?

A count of participants with an event records whether each participant was reported to have experienced at least one event within the specified outcome and observation period. It is different from a count of all event episodes: one participant with repeated episodes may still contribute as one participant to a participant-count endpoint. The registry defines the primary outcome in terms of participants, and its reported vaccine-site reaction figure is likewise phrased as a number of patients. The record does not clarify how repeated events were handled in every adverse-event category.

Counts are useful for safety description because they are direct and interpretable. They do not, by themselves, convey event duration, severity, recurrence, or the relationship between an event and treatment. Nor do they show how outcomes were distributed across the two cohort descriptions. Those details require additional reporting, which is not reported in the registry record for the primary endpoint.

What can a single-group result establish?

General statistical principle: a comparison group helps distinguish changes associated with an intervention from changes that might occur for other reasons. Random allocation is one way to make groups comparable in expectation, while masking can reduce some influences on measurement and reporting. This registry lists non-randomized allocation, no masking, and a single-group model. It therefore does not describe a randomized, masked comparison of the vaccine-plus-GM-CSF regimen with a control.

The two cohort descriptions refer to evaluation of treatment-limiting toxicity and evaluation of response. They do not make the trial a randomized comparison. Differences in outcomes between cohorts, if reported, could reflect differences in who entered each cohort or how they were assessed, rather than an intervention effect. Cohort-specific outcome results and the basis for assigning participants to cohorts are not reported in the registry record.

How should uncertainty be understood?

General statistical principle: a confidence interval expresses the uncertainty around an estimate under a stated statistical model. A narrow interval generally indicates greater precision than a wide interval, while an interval for a single-group event frequency would not remove concerns about selection, event ascertainment, or generalizability. The registry record does not report a confidence interval for the primary endpoint or for the separate adverse-event counts. It therefore does not quantify sampling uncertainty for these results.

A p-value addresses compatibility between data and a specified statistical hypothesis under assumptions; it is not a measure of clinical importance or proof of causation. No p-value or inferential analysis for the primary safety outcome is reported in the registry record. The record lists 0 analyses, so there is no reported analysis from which to infer a testing procedure or statistical significance.

Why do definitions and observation windows matter?

General statistical principle: an event count depends on the outcome definition, the population assessed, and the time during which events are collected. A longer observation period can offer more opportunity to record an event, but comparing counts across studies or groups requires compatible definitions and follow-up. Here, the primary endpoint timeframe is “6 months following last dose of treatment, up to 9 months.” The registry does not report participant-level follow-up duration or whether all participants completed that window.

Attribution also matters. An event described as associated with vaccination is not necessarily proven to have been caused by vaccination. The registry’s summary says adverse events potentially related to vaccination were largely restricted to injection-site reactions, but it does not report a detailed adjudication procedure or an event-level causal assessment. The separate adverse-event summary distinguishes serious from other events; the categories should not be taken as a complete narrative of severity or attribution.

What do planning assumptions mean?

General statistical principle: a sample-size calculation for a future study depends on assumptions about the effect to detect, the significance level, allocation, and statistical method. Such a calculation is a planning illustration, not an observation from the trial and not a measure of the trial’s achieved power. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as stated fractions of an observed effect. The record explicitly frames those assumptions as a planning illustration computed from an observed effect, not as the trial’s own design.

Those assumptions do not change the actual study design recorded as non-randomized and single group. In particular, an equal-allocation assumption for a planning illustration does not establish that this study randomized participants equally between arms. The record does not report a protocol-based sample-size justification, the effect estimate used in the illustration, or planning results in this statistical record. The assumptions therefore cannot be used to infer the intended enrollment rationale or the precision of the observed safety results.

General statistical principle: over-dispersion matters when count outcomes vary more than a chosen statistical model expects. A model that understates this variation can make uncertainty appear too small. The primary endpoint is a participant count, and no model-based analysis is reported in the registry record; therefore, no claim about over-dispersion handling can be made. In general, the appropriate analysis depends on whether the endpoint counts participants with any event, counts repeated event episodes, or measures event rates over person-time. The registry’s primary endpoint is phrased as participants with an event, and person-time methods are not described.

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("Patients Treated With Vaccine")
n      <- c(19)
events <- c(12)
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  Patients Treated With Vaccine  19  12  63.2  38.4  83.7  41.0  80.9  40.8  80.9

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures and identifies a secondary outcome: the number of participants demonstrating a two-fold increase in interferon gamma. The primary outcome concerns participants with adverse events associated with vaccination. A result for the interferon gamma outcome is not reported in the registry record. The second outcome measure beyond the named secondary outcome is not described in the record in a way that permits a fuller account of all planned endpoints.

When a study assesses multiple outcomes or conducts multiple statistical tests, the chance of at least one apparently unusual result can increase. General statistical principle: a prespecified testing hierarchy or adjustment procedure can address some multiplicity concerns, but the choice depends on the study’s goals and analysis plan. A multiplicity strategy is not reported in the registry record. Since no analyses are listed, it is not possible to determine whether any adjustment was used or how outcomes were prioritized beyond the primary and secondary labels.

Missing-data procedures and analysis populations are also not reported in the registry record. The enrollment figure is 36, while the adverse-event summary lists 19 patients at risk. The record does not explain this difference, state how missing observations were handled, or specify whether the safety counts use all treated participants or another population. It also does not report how many participants completed the primary endpoint observation window.

These omissions limit interpretation of completeness and analysis choices. They do not establish that data were missing or that participants were excluded; they mean that the registry record does not describe those matters. The reported counts should therefore be read with their stated denominators and without assumptions about unreported analysis populations or multiplicity procedures.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 1% reporting threshold.

Most frequent non-serious adverse event terms

The registry’s serious adverse-event summary lists 19 patients at risk and 0 with serious adverse events. The separate other-adverse-event summary lists 19 at risk and 16 affected. The record also lists 0 deaths. These are registry counts, not comparative estimates against a control group.

The primary endpoint description separately states that 12 of the 19 patients experienced vaccine-site reactions and says potentially related adverse events were largely restricted to injection-site reactions. This specific result should not be treated as interchangeable with the broader other-adverse-event count. The record does not provide enough event-level detail to determine which individual events overlap between those descriptions, how events were graded, or how attribution was assessed.

A reported count of 0 serious events does not prove that serious events cannot occur, and it does not describe risk beyond the patients and follow-up captured in the registry summary. Likewise, the other-event count does not establish causation. The record does not explain why the safety at-risk group is 19 despite enrollment of 36, or provide a complete account of the safety population. Read the figures as the posted registry summaries, with those denominator and reporting limitations in mind.

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

The reported safety observations give a limited description of events recorded in this study. The primary endpoint description reports vaccine-site reactions in 12 of the 19 patients and characterizes potentially related adverse events as largely restricted to injection-site reactions. The separate adverse-event summary records 0 serious adverse events among 19 at risk, 16 with other adverse events among 19 at risk, and 0 deaths. The record does not provide enough detail to reconstruct the full event experience or reconcile the safety denominator with enrollment of 36.

These counts do not show that the intervention caused or prevented an event. There is no randomized control group, and the study is listed as non-randomized, unmasked, and single group. Without a suitable comparator, background event occurrence and other influences cannot be separated from events potentially associated with vaccination. The phrase “associated with vaccination” in the endpoint title should not be read as proof of causation.

The record also does not report a comparative estimate of response, an inferential analysis of the primary outcome, or a result for the interferon gamma secondary outcome. It therefore does not support a statistical conclusion about efficacy or a comparative treatment effect. The cohort description for response indicates an evaluation aim, not a reported response result.

Nor do these results establish the full safety profile for every patient with renal cancer or for settings beyond the study. The registry record does not report detailed event grades, event timing, participant-level follow-up, or the reasons the safety summary covers 19 patients. Those omissions constrain how broadly the counts can be interpreted. The findings mean only that the record reports the stated events and counts in its listed safety summary, within the described study and timeframe.

14. Why This Trial Matters Statistically

This trial is statistically notable because its central question is safety, but its registry design is non-randomized and single group. That design is compatible with describing events observed during treatment, while it offers no randomized control comparison for estimating how many events would have occurred without the intervention. Keeping description separate from causal inference is essential when interpreting safety counts.

The record also makes clear that different safety summaries answer different questions. The primary endpoint describes participants with vaccination-associated adverse events and highlights vaccine-site reactions. A separate summary classifies serious and other adverse events and lists deaths. These figures should retain their own definitions and denominators rather than being combined or treated as duplicate versions of one outcome.

Another statistical issue is the relationship between the enrollment figure and the safety at-risk group. Enrollment is listed as 36, while the adverse-event summary uses 19. Because the reason is not reported in the registry record, readers cannot determine from this record whether the difference reflects cohort scope, treatment exposure, follow-up, or another aspect of reporting. That uncertainty matters for judging completeness and for understanding the population to which the counts apply.

Finally, the record names a secondary immune-response outcome but does not report its result, and it lists no analyses. This illustrates why outcome definitions, analysis populations, missing-data methods, and statistical procedures are important parts of interpreting a trial. Without those details, the posted information supports a narrow descriptive reading rather than a broader claim about response, comparative effectiveness, or precision.

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 registry record provides limited detail on the safety population. It lists enrollment of 36 but gives 19 patients at risk in the adverse-event summary. The reason for this difference, cohort-specific denominators, and the relationship between enrollment and the participants included in each safety count are not reported in the registry record.

The study is listed as non-randomized, unmasked, and single group. Although two cohorts are described, one for evaluation of treatment-limiting toxicity and one for response, the record does not describe a randomized control group or a like-for-like comparison. This limits causal interpretation and prevents a controlled estimate of comparative treatment effect.

For the primary endpoint, the registry reports a timeframe and a brief description of vaccine-site reactions, but does not report detailed event-level data, severity grades, duration, recurrence, or a full account of event attribution. Participant-level follow-up and completion of the observation window are not reported in the registry record. The record also does not report confidence intervals or other inferential estimates for the safety counts.

The secondary interferon gamma outcome is named without a reported result. The statistical plan, analysis populations, missing-data procedures, multiplicity approach, and protocol-based sample-size justification are not reported in the registry record. The planning assumptions described for an illustration do not establish the trial’s own design or analysis. Together, these gaps restrict interpretation to the reported descriptive counts and do not support broader conclusions about comparative effects or the complete safety experience.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Vaccination of Patients With Renal Cell Cancer With Dendritic Cell Tumor Fusions and GM-CSF — NCT00458536.
• Registry record last updated 2026-07-29; status last verified 2026-07.
• Results first posted 2016-02-17.
• 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

NCT00458536 is a completed Phase 1 and Phase 2 study sponsored by Beth Israel Deaconess Medical Center. The registry describes a dendritic cell/tumor fusion vaccine with GM-CSF for patients with renal cancer, with enrollment of 36 and a single-group, non-randomized, unmasked design. The listed start date is 2004-10 and primary completion is 2014-12.

The primary endpoint counts participants with adverse events associated with vaccination over “6 months following last dose of treatment, up to 9 months.” The registry reports vaccine-site reactions in 12 of the 19 patients. Its separate adverse-event summary lists 0 serious adverse events among 19 at risk, 16 with other adverse events among 19 at risk, and 0 deaths. The record names a secondary interferon gamma outcome but does not report its result. It does not explain why the safety summary’s at-risk group differs from enrollment or report inferential analyses.