PANVAC-V and PANVAC-F Vaccines Plus Sargramostim to Treat Advanced Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for PANVAC-V and PANVAC-F Vaccines Plus Sargramostim to Treat Advanced Cancer (NCT00088413), a registered phase 1, phase 2 study. The primary outcome is Number of Participants With Complete Responses (CRs), Partial Responses (PRs,) Stable Disease and Progressive Disease in the Ovarian Cancer and Breast Cancer Cohorts.

2. Clinical Question

The study asked whether a vaccination regimen combining PANVAC-V, PANVAC-F, and sargramostim could be evaluated for safety and tolerability in adults with advanced cancer, and whether immune or anti-tumor responses could be documented. The registry describes an open-label pilot study, not a randomized comparison of the regimen against a control treatment.

Participants were adults with advanced cancer whose tumors produced CEA or MUC-1, according to the eligibility description. The listed conditions include adenocarcinoma, colorectal cancer, ovarian cancer, and breast cancer. The study was organized into colorectal and non-colorectal cancer cohorts, a breast cancer cohort, and an ovarian cancer cohort. The intervention description says all cohorts received the same intervention.

The intervention comprised PANVAC-V, described as the priming vaccine, followed by PANVAC-F, described as the boosting vaccine, with sargramostim. The registry explains that the vaccines contain human genes associated with CEA and MUC-1 production, as well as genes intended to enhance immune activity. This is the registry's account of the intervention's rationale, not evidence that an immune or tumor response occurred.

The stated primary outcome focuses on the breast and ovarian cancer cohorts. It counts participants classified as having complete response, partial response, stable disease, or progressive disease by RECIST during approximately 6 months while on trial. The registry also lists safety and immune-response outcomes. The posted results do not include the primary outcome counts or analyses, so the clinical question is described by the planned outcome rather than answered by reported comparative estimates.

3. Trial Design

The registry classifies the study as Phase 1 and Phase 2, open label, and sequential. Enrollment is listed as 51, and the study status is completed. The start date is 2004-07-21 and the primary completion date is 2018-04-01. The sponsor is the National Cancer Institute (NCI).

The design description identifies three cohorts: colorectal and non-colorectal cancer, breast cancer, and ovarian cancer. It gives planned cohort descriptions of 10 patients with advanced colorectal cancer and 10 to 15 patients with advanced non-colorectal cancer in the first cohort, 12 patients with advanced breast cancer in the second cohort, and 14 patients with advanced ovarian cancer in the third cohort. The registry's intervention description states that all cohorts received the same intervention. These descriptions do not establish a randomized or concurrent control group.

The vaccination schedule begins with PANVAC-V on study day 1, followed by PANVAC-F on days 15, 29 and 43 and then every 28 days for up to 12 vaccines, followed by every 3 months until disease progression or toxicity. The vaccines are described as injections under the skin. The brief description also says sargramostim is injected at the vaccination site, but the available description ends before the complete administration details.

Because masking is listed as none and the study is open label, treatment assignment was not masked according to the record. Allocation is not stated. The record does not describe a like-for-like control comparison, and the listed cohort groupings should not be interpreted as randomized arms. The sample-size justification, detailed protocol for cohort enrollment, and any interim analysis plan 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:

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 Complete Responses (CRs), Partial Responses (PRs,) Stable Disease and Progressive Disease in the Ovarian Cancer and Breast Cancer Cohorts, measured over Approximately 6 months while on trial.

The primary endpoint is a count of participants in the ovarian and breast cancer cohorts classified as having complete response, partial response, stable disease, or progressive disease. Assessment is described as using the Response Evaluation Criteria in Solid Tumors (RECIST), with a timeframe of approximately 6 months while on trial. The outcome therefore describes the distribution of recorded disease-response categories in those cohorts; it is not a single continuous measure of tumor size or a time-to-event endpoint.

The registry defines complete response as disappearance of all clinical and laboratory signs and symptoms of disease for a minimum of 4 weeks, with no new lesions. Partial response requires a minimum 30% decrease in the sum of the longest diameter of target lesions relative to baseline. Stable disease means neither enough shrinkage for partial response nor progressive disease, using the smallest sum of longest diameters since treatment started as reference. Progressive disease requires a minimum 20% increase using that smallest recorded sum as reference, or the appearance of one or more new measurable lesions.

These categories provide a structured way to describe tumor status under the stated criteria. They are useful for distinguishing degrees of change and stability, but a category count alone does not describe how long a response lasts beyond the stated assessment context, nor does it establish that the intervention caused the observed status. Actual counts for these categories are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation depends first on the type of question and outcome. This study is described as an open-label, sequential Phase 1/Phase 2 pilot study. The primary outcome is a count of participants classified into RECIST response categories, rather than a continuous measurement or a time-to-event outcome. The posted record does not include outcome analyses, so there is no reported estimate to interpret statistically.

What does an open-label, sequential design imply?

General: In an open-label study, participants and study personnel are not masked to the intervention. This may matter when outcomes involve clinical assessment or decisions about whether a participant remains under observation. Sequential designs can describe how study groups or cohorts are enrolled over time, but the term alone does not reveal the detailed enrollment rules or whether later decisions depended on earlier results. Those protocol details are not reported in the registry record.

The study is not described as randomized, and allocation is not stated. The record also says the cohorts receive the same intervention. Therefore, the cohort labels do not create a randomized treatment contrast. A difference in recorded outcomes across colorectal, non-colorectal, breast, and ovarian cancer groups could reflect differences between the populations or their disease contexts, and would not by itself estimate a treatment effect.

How should response categories be read?

General: A categorical outcome classifies each assessed participant into one of several mutually distinguished states. RECIST response categories use measurements of target lesions and the appearance of new lesions, according to the definitions listed in the record. A count in each category describes how many participants were assigned that classification, if counts are available. It does not by itself convey the duration of the classification, the uncertainty around the count, or the counterfactual outcome that would have occurred without the regimen.

The listed primary endpoint combines the response categories in its title, but no category counts are posted. No estimate, confidence interval, or statistical test is given for the primary outcome. General: A confidence interval, when reported, describes statistical uncertainty around an estimate under the assumptions of the analysis; it does not eliminate limitations in the study design or measurement. Since no interval or estimate is reported, the precision of the response findings cannot be evaluated from this record.

What can the safety summaries establish?

The record reports participants at risk and affected for serious and other adverse events, together with death counts, in two cohort groupings. These are useful descriptive quantities for understanding what the registry lists, but they are not a randomized comparison. The record does not provide the event-level detail needed to evaluate timing, recurrence, severity distributions, or attribution. It also does not report a statistical comparison of safety between treatment groups.

General: An adverse-event count and the number of participants with at least one event answer different questions. The record's group fields use participant counts, while its top-term list gives percentages for named terms. The definition of the percentage denominator and whether a participant could contribute more than one event are not reported in the registry record. For that reason, the percentages should not be converted into counts or interpreted as the proportion of all enrolled participants without further definition.

Why do missing outcomes matter?

The record lists enrollment but does not report the number assessed for the primary endpoint or the handling of participants without an assessment. General: If outcome availability is related to disease status, treatment tolerance, or follow-up, analyzing only participants with available measurements can produce a result that differs from the result for the full enrolled group. An analysis plan may specify how such cases are handled, but no such plan is reported in the registry record.

The sample-size assumptions describe planning illustrations computed from an observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. These are planning illustrations, not observed power, and they do not establish the trial's original sample-size justification. The trial's own sample-size justification is not reported in the registry record. The posted record also lists no analyses, so there are no reported statistical methods for an outcome estimate to assess.

General: Over-dispersion matters for count data when variability is greater than the model assumes; ignoring it can make uncertainty estimates too narrow. This study's primary outcome is a count of participants in response categories, not a reported event-rate model, and the record provides no fitted model or dispersion estimate. There is therefore no basis to claim that over-dispersion was present or that a particular adjustment was used.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary outcome and several secondary outcomes, covering response categories, adverse events, and immune responses. It does not report how these outcomes were prioritized in the analysis, whether there were additional endpoints, or whether any adjustment for multiple testing was planned or applied. A multiplicity plan is not reported in the registry record.

General: When several outcomes or subgroup analyses are examined, the chance of obtaining at least one apparently unusual result can increase if each is tested separately without accounting for the set of tests. Different approaches to multiplicity can address different aims, such as controlling the chance of a false positive across a family of confirmatory tests or treating results as exploratory. Without a reported analysis plan, the intended role of the listed secondary outcomes cannot be determined.

The record does not report the number of participants with an observed primary outcome, missing primary outcome data, reasons for missingness, or rules for including participants in each analysis. It also does not identify an intention-to-treat, per-protocol, or other analysis population. These details are not reported in the registry record.

General: Missing outcome data can affect both the estimated distribution of response categories and its uncertainty. The consequences depend on how much data are unavailable and why they are unavailable, as well as on the method used to handle them. No imputation method, censoring rule, or sensitivity analysis is reported in the registry record. Because the actual primary counts and analysis population are absent, neither the extent nor the statistical impact of missingness can be assessed.

The registry's count of 51 enrolled participants does not establish that 51 participants contributed to every listed outcome. Enrollment and analysis denominators are distinct quantities. The number assessed for each endpoint and the disposition of participants are not reported in the registry record.

11. 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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: dermatology/skin:: injection site reaction/extravasation changes*3 (100.0%), metabolic/laboratory:: albumin, serum-low (hypoalbuminemia)*3 (76.9%), syndromes:: flu-like syndrome*3 (69.2%), blood/bone marrow:: hemoglobin*3 (52.0%), constitutional symptoms:: fatigue (asthenia, lethargy, malaise)*3 (52.0%).

The serious adverse-event summary is organized into two groups: the colorectal cancer versus non-colorectal cancer cohort, and the breast and ovarian cohorts. For the first group, 25 participants are listed as at risk and 25 as affected for serious adverse events; the same 25 and 25 are listed for other adverse events. The recorded death count is 1. For the breast and ovarian cohorts, the corresponding at-risk and affected counts are 26 and 26 for serious adverse events and 26 and 26 for other adverse events; the recorded death count is 0.

These are descriptive registry entries, not estimates comparing the regimen with a control treatment. The record does not report event timing, participant-level details, duration, seriousness criteria for individual events, or attribution to a particular intervention component. The listed groupings should not be treated as separate treatment arms.

The record also lists serious-event terms with percentages: injection site reaction/extravasation changes, 100.0%; hypoalbuminemia, 76.9%; flu-like syndrome, 69.2%; hemoglobin, 52.0%; fatigue, 52.0%; and fever, 50.0%. The denominator and precise interpretation of these term percentages are not reported in the registry record. The percentages do not, on their own, indicate whether events were treatment-related or how severe or prolonged they were.

General: Safety summaries are most informative when their denominators, event definitions, follow-up period, severity, and attribution are clear. These details help distinguish participants with at least one event from repeated events and clarify which events were considered serious. The available record does not report all of these details, so the entries should be interpreted as limited descriptive reporting.

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

The registry record establishes that a completed open-label pilot study evaluated PANVAC-V and PANVAC-F with sargramostim in adults with advanced cancer, with cohorts defined by cancer type. It specifies safety, immune-response, and anti-tumor-response objectives. It also identifies a RECIST-based primary outcome for the breast and ovarian cohorts over approximately 6 months while on trial.

However, the primary response-category counts are not reported in the registry record. Immune-response results are also not reported, and no statistical analyses are listed. Consequently, the posted information does not quantify how many participants had each response category, how uncertain any estimate might be, or whether a response differed across groups. It cannot support a numerical conclusion about treatment effect.

The safety information documents cohort-level participant counts and deaths, alongside named adverse-event terms and percentages. It does not provide a control group, full event-level data, or a clear denominator for the term percentages. These entries describe what the registry records but do not isolate the contribution of the vaccines, sargramostim, the underlying cancer, or other factors.

General: An observed outcome in a single-arm or nonrandomized study is not the same as a causal effect. A causal comparison asks how outcomes under the intervention differ from outcomes that would have occurred under a suitable alternative. Without a control comparison, that contrast is not available from the design. Cohort-to-cohort differences are also not a substitute when cohorts represent different cancer types.

The study's objectives and definitions therefore matter, but they do not replace outcome results. The record can be used to understand the intervention schedule, cohort structure, and intended measurements. It does not establish whether the regimen produced an immune response, a tumor response, or a specific safety profile relative to another treatment. Those conclusions are not reported in the registry record.

13. Why This Trial Matters Statistically

Statistically, this study illustrates the difference between an outcome definition and an outcome result. The registry specifies a clinically interpretable primary outcome: participant counts across RECIST response categories in the breast and ovarian cohorts. It also states a timeframe and definitions for complete response, partial response, stable disease, and progressive disease. Yet the outcome counts and statistical analyses are not reported in the registry record. A clear endpoint cannot be interpreted quantitatively without the results and their denominators.

The design also highlights the importance of separating cohort descriptions from comparative treatment groups. The study is open label and sequential, and all cohorts are described as receiving the same intervention. The different cancer cohorts are not a like-for-like treatment comparison. Recognizing this prevents disease-specific outcome differences from being mistaken for evidence that one treatment strategy outperformed another.

The safety summaries demonstrate why denominators and event definitions matter. The registry records at-risk and affected participant counts, deaths, and percentages for selected event terms. Without the denominator definition and fuller event-level detail, those figures remain descriptive. They cannot answer questions about comparative risk, timing, or treatment attribution.

The record also lists several secondary outcome areas, including adverse events and immune responses. When multiple outcomes are considered, the analysis plan is important for interpreting the strength of evidence. The multiplicity plan, missing-data handling, analysis populations, and detailed statistical plan are not reported in the registry record. This limits the ability to assess how robust or exploratory any result would be, even if estimates were available.

Finally, planning illustrations should be distinguished from observed evidence. Sample-size illustrations based on an observed effect are planning calculations, not observed power and not the trial's own design justification. Overall, the statistical value of this record lies in its description of the intended questions and the limits of the posted results, rather than in a numerical estimate of efficacy or comparative safety.

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 most direct limitation is that the registry record does not report the primary response counts or any analyses. Without the numbers assessed in each RECIST category, there is no result estimate to interpret and no way to judge its statistical precision. Secondary immune-response outcome counts are also not reported.

The study is described as open label and sequential, with all cohorts receiving the same intervention. No randomized control comparison is described, and allocation is not stated. Consequently, the record does not provide a like-for-like basis for estimating a comparative treatment effect. Cohorts are defined by cancer type, so differences across them should not be interpreted as treatment differences.

Although enrollment is listed as 51, the number contributing data to each outcome is not reported. The registry does not describe outcome completeness, reasons for missing assessments, analysis populations, or handling of missing data. It also does not report a detailed statistical analysis plan, multiplicity approach, interim analysis plan, or sample-size justification.

The safety summaries have additional interpretive limits. The record lists participant counts at risk and affected, death counts, and percentages for selected adverse-event terms, but does not report the denominator definition for those percentages or the event-level timing, duration, severity distribution, and attribution needed for a fuller assessment. The available descriptions do not establish which specific intervention component, if any, was associated with an event.

The brief intervention description is truncated after stating that sargramostim is injected at the vaccination site, so complete administration details are not available in the registry record. The listed outcome timeframe is approximately 6 months while on trial, but the record does not report the assessment schedule or follow-up completeness. These limits constrain both clinical interpretation and statistical evaluation.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: PANVAC-V and PANVAC-F Vaccines Plus Sargramostim to Treat Advanced Cancer — NCT00088413.
• Registry record last updated 2019-04-16; status last verified 2019-04.
• Results first posted 2019-04-16.
• 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.

18. Record Summary

NCT00088413 is a completed, open-label, sequential Phase 1/Phase 2 pilot study sponsored by the National Cancer Institute (NCI). Enrollment is listed as 51. The study evaluated PANVAC-V and PANVAC-F with sargramostim in adults with advanced cancer, organized into colorectal and non-colorectal, breast, and ovarian cancer cohorts. All cohorts are described as receiving the same intervention.

The primary outcome specified for the breast and ovarian cohorts is the number of participants classified by RECIST as having complete response, partial response, stable disease, or progressive disease during approximately 6 months while on trial. The record also lists safety and immune-response outcomes. Primary response counts, immune-response results, and statistical analyses are not reported in the registry record.

The safety summary lists cohort-level counts for participants at risk and affected, deaths, and selected adverse-event terms with percentages. The denominator and further event-level details are not reported. The study design and posted outcomes therefore support a description of the planned questions and recorded safety summaries, but not a quantitative conclusion about comparative treatment effects.