RV001V, a RhoC Anticancer Vaccine, Against Metastasis From Solid Tumours: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for RV001V, a RhoC Anticancer Vaccine, Against Metastasis From Solid Tumours (NCT03199872), a na phase 1, phase 2 study. The primary outcome is Proportions of Participants With Treatment-related Adverse Events.

2. Clinical Question

The clinical question is about safety and immunological response to a RhoC peptide vaccine in patients with prostate cancer. The registry lists proportions of participants with treatment-related adverse events as the primary outcome, and immunological response as a secondary outcome. It does not report a comparative question about RV001V versus another treatment or a control group.

The primary endpoint is framed around the occurrence of treatment-related adverse events, rather than a measure of tumour response or metastasis. The available registry information does not give a numerical result for that endpoint, an estimate of the immunological response, or an analysis linking either outcome to participants’ characteristics. Accordingly, the record supports describing the study’s questions and design, but not drawing a conclusion about the size or precision of an observed response.

3. Trial Design

This is a Phase I/II, single-group study of RV001V in patients with prostate cancer. The registry records enrollment of 22 and lists RhoVac APS as the sponsor. The study status is completed. The recorded start date is 2017-03-30, and the primary completion date is 2018-08-01.

Allocation is listed as not applicable, and masking is listed as none. The design therefore has no randomized comparator arm and no masking recorded. The intervention is RV001V, described as a vaccine containing the RV001 peptide and Montanide ISA 51 adjuvant. The registry’s brief summary says the study evaluates safety and immunological response to the RhoC peptide vaccine in patients with prostate cancer.

A single-group design can describe outcomes among people receiving the intervention, but it does not create a concurrent control group for separating treatment effects from other explanations. Changes over time, participant selection, and other influences cannot be distinguished by a randomized between-group comparison in this design. These are general design considerations, not claims about what happened to participants in this study.

The registry record does not report a more detailed protocol description, participant characteristics, a control regimen, or a statistical analysis plan. It also does not report why the enrollment target was chosen. The Phase I/II label and the listed outcome descriptions identify the broad aims, but do not establish the precise procedures used for every assessment or the basis for any formal conclusions.

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 Proportions of Participants With Treatment-related Adverse Events, measured over 8 month.

The primary endpoint is titled “Proportions of Participants With Treatment-related Adverse Events.” Its unit is participants, and its description says adverse events were assessed using CTCAE v4.0. The stated timeframe is 8 month. In general, a participant-level binary endpoint records whether each participant did or did not meet an event definition during the specified period. The resulting proportion expresses how often the defined event occurred in the assessed group.

Safety endpoints are used to characterize unwanted events observed during a study and can help describe the tolerability profile recorded in that study. The phrase “treatment-related” indicates that the endpoint concerns attribution to treatment, rather than simply any event occurring during follow-up. CTCAE v4.0 is the assessment system named in the record; the registry does not report further details about how relatedness was decided or how event severity was handled for this primary outcome.

The endpoint’s title does not itself provide a result. Although the registry contains adverse-event summary counts, it does not report an analyzed estimate for this primary endpoint, such as the number meeting the treatment-related definition, a proportion, or a confidence interval. The record also does not clarify how missing assessments, if any, were handled. Therefore, the endpoint can be described, but its observed frequency and precision cannot be established from the primary-outcome information reported.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes the primary outcome as a participant-level count for a binary endpoint: participants with treatment-related adverse events. In general, binary data classify each assessed participant according to whether an event definition was met. A proportion is commonly formed by relating the number meeting the definition to the number assessed. That description does not establish which participants were included in an analysis or how incomplete follow-up was handled; those details are not reported in the registry record.

What does a confidence interval add?

In general, a confidence interval expresses the uncertainty around an estimate under a specified statistical method. A narrow interval indicates greater precision under that method, while a wide interval indicates less precision. The interval does not eliminate bias or establish that an endpoint was measured correctly. The registry record does not report a confidence interval for the primary outcome, and it gives no analyzed primary-endpoint proportion to accompany one. Consequently, neither the estimated frequency nor its statistical precision can be assessed from the primary outcome entry.

How should a single-group result be interpreted?

For a single-group study, an event proportion describes participants observed in that study. It does not, by itself, describe the counterfactual outcome those same participants would have had under a different intervention. General statistical reasoning therefore separates descriptive safety reporting from comparative treatment-effect estimation. Here, the allocation is recorded as not applicable and the model as single group. There is no randomized control comparison in the registry record from which to estimate a between-group difference.

That limitation matters even if an event proportion is available. The frequency can reflect the intervention, the underlying condition, other care, participant selection, or event ascertainment. A single-group description cannot isolate those influences. This is a general limitation of the design, not evidence that any particular influence affected the reported participants.

What do the safety counts represent?

The registry reports adverse-event counts by category, including participants at risk and affected. Such counts are descriptive summaries. The serious-event entry records 4 affected among 22 at risk; the other-adverse-event entry records 22 affected among 22 at risk. The listed deaths count is 0. These figures do not supply the missing primary endpoint analysis because the record does not say that the categories correspond exactly to the primary endpoint’s treatment-related definition. The specific attribution rules and outcome-analysis population are not reported in the registry record.

General interpretation of event counts also depends on the observation period and event definitions. A participant-level count does not reveal, by itself, how many events each participant experienced, how long each person was observed, or whether follow-up was complete. The primary endpoint states an eight-month timeframe, but the registry does not report further information on exposure time, missing observations, or the handling of events outside the assessment period.

What is over-dispersion, and does it apply?

In general, over-dispersion arises when observed variability in count data is greater than expected under a chosen statistical model. It matters when a model’s uncertainty estimates rely on assumptions about how counts vary. A binary participant-level endpoint is not the same as a recurrent-event count: each participant is classified by whether the endpoint occurred, whereas recurrent-event analyses may count multiple events for a person. The record describes the primary endpoint as participants with an event and reports no model or recurrent-event analysis. It therefore does not establish that over-dispersion was assessed or relevant to the reported primary outcome.

How should planning illustrations be read?

The registry-associated planning description says the illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect stated as a fraction of the observed effect. It also distinguishes participant-per-arm illustrations from total-event calculations for time-to-event outcomes. These are planning assumptions, not a description of the actual single-group study design. A hypothetical two-arm planning framework does not mean that this trial randomized participants between two arms.

In general, a sample-size planning calculation asks how much information a future study might need under specified assumptions. It is not an observed result and is not “observed power.” The registry record does not report the trial’s own sample-size justification or the statistical basis for its enrollment. Although the planning description refers to an observed effect, the registry does not report an analyzed primary-endpoint effect that allows readers to evaluate that input. Any planning illustration should therefore be treated as conditional on its stated assumptions, not as evidence about the trial’s achieved precision or design rationale.

More broadly, a statistical analysis plan would specify such matters as endpoint definitions, analysis populations, missing-data handling, model choice, and any planned interim analyses. Those details are not reported in the registry record. They should not be inferred from the Phase I/II designation, the completed status, or the existence of descriptive safety counts.

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("Experimental: RV001V")
n      <- c(22)
events <- c(22)
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  Experimental: RV001V  22  22  100.0  84.6  100.0  85.1  100.0  85.2  99.9

11. Multiplicity, Missing Data and Analysis Populations

The registry lists two outcome measures and identifies immunological response as a secondary outcome. The primary outcome is the proportion of participants with treatment-related adverse events. The record does not report analyses for either outcome. It also does not report a multiplicity strategy, such as how inference across multiple outcomes or multiple tests would be controlled. In general, testing multiple hypotheses can increase the chance of at least one apparently notable finding, so a prespecified approach helps readers interpret inferential results. No inferential outcome results are available to evaluate under such an approach.

Information about missing data is also not reported in the registry record. The record does not state whether any outcome assessments were missing, how participants with incomplete follow-up were handled, or whether imputation or sensitivity analyses were used. These are distinct issues: the number enrolled does not establish how many contributed data to each outcome analysis. The adverse-event counts include participants at risk, but they do not describe the handling of missing primary endpoint assessments.

The analysis population is not reported in the registry record. There is no description of whether analysis included all enrolled participants, participants who received the intervention, or another defined population. The record also does not report a protocol-based statistical analysis plan, interim analyses, or the prespecified treatment of secondary outcomes. The absence of these details does not establish that a particular method was or was not used; it means the registry record does not permit that determination.

These omissions limit reproducibility and interpretation. Without the analysis population and missing-data rules, the denominator and inclusion criteria behind a primary endpoint estimate would be difficult to assess even if an estimate were listed. Without a multiplicity plan, a collection of inferential results could be hard to interpret as a coherent set. In this case, no analyzed estimates or tests are reported, so the appropriate conclusion is that multiplicity and missing-data methods are not documented in the registry record.

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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: arthralgia (9.1%), impaired healing (4.5%), pneumonia (4.5%).

The registry’s safety summary records serious adverse events for 4 participants among 22 at risk, other adverse events for 22 participants among 22 at risk, and 0 deaths. These counts are descriptive. They do not by themselves show whether an event was attributed to RV001V, whether it met the primary endpoint definition, or how long each participant was observed. The primary endpoint is specifically treatment-related adverse events over an eight-month timeframe; the record does not establish that the summary categories are identical to that endpoint.

The serious-event terms listed are Arthralgia (9.1%), Impaired healing (4.5%), and Pneumonia (4.5%). These percentages are reported as term-level summaries. The registry does not say whether one participant could contribute to more than one term, so the terms should not be treated as separate, non-overlapping participant groups or added together. Nor does the listing establish that each term was judged treatment-related.

Safety counts are best read alongside event definitions, attribution procedures, follow-up completeness, and analysis populations. Those details are not reported in the registry record. These counts therefore describe the recorded adverse-event categories without determining cause, comparative risk, or the primary endpoint’s estimated proportion.

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

The record establishes that a completed single-group Phase I/II study of RV001V was registered for patients with prostate cancer. Its stated aims were to evaluate safety and immunological response. The primary outcome concerns treatment-related adverse events assessed using CTCAE v4.0 over 8 month, and immunological response is listed as a secondary outcome. The record also contains descriptive safety counts and serious-event terms.

Those details do not establish the frequency of the primary endpoint. The registry does not report a primary outcome analysis, a treatment-related adverse-event estimate, or a confidence interval. It does not report an immunological-response result either. The adverse-event counts cannot fill this gap because the record does not establish that they use the same treatment-related definition and timeframe as the primary endpoint. They also do not provide a comparison with another treatment.

In general, a single-group study can document what was observed among participants in that group, but it cannot make a randomized comparison with an alternative. The record therefore does not estimate a causal effect of RV001V on adverse events, immune response, tumour outcomes, or metastasis. The study title refers to metastasis from solid tumours, but the listed condition is prostate cancer and the reported primary endpoint is a safety measure. No metastasis outcome result is reported in the registry record.

The safety summary should not be translated into a broad judgment of benefit or harm. Event attribution, severity details for the primary endpoint, follow-up completeness, and analysis populations are not reported. Without those details and an analyzed endpoint result, the reported counts do not establish how precisely the treatment-related event question was answered.

The most defensible reading is narrow: the registry records the study’s design, aims, an eight-month safety endpoint, and certain adverse-event summaries. It does not report outcome analyses sufficient to quantify the primary endpoint or immunological response, and its non-comparative design does not support a control-based treatment-effect estimate.

14. Why This Trial Matters Statistically

This trial illustrates why statistical interpretation depends on more than an endpoint label or an enrollment figure. The registry identifies a participant-level safety endpoint and a stated assessment timeframe, but does not report its analyzed result. Without an estimate and an indication of its precision, readers cannot determine how frequent the defined treatment-related events were or how uncertain that estimate might be.

The single-group design is also important. In general, descriptive counts answer what was recorded in the study group, while a randomized comparison can address how outcomes differ between assigned interventions under the design’s assumptions. Allocation is listed as not applicable, so the reported adverse-event counts do not form a like-for-like control comparison. The distinction guards against reading an observed event count as a treatment effect.

The record further shows why endpoint definitions and category labels matter. The primary question concerns treatment-related adverse events, whereas the adverse-event summary separates serious events from other events. The registry does not state that these are equivalent classifications. An accurate statistical reading keeps the primary definition separate from the available descriptive summaries rather than treating them as interchangeable.

Finally, the record lists immunological response as a secondary outcome but reports no analysis. This makes the difference between a planned or named outcome and a reported result explicit. A complete interpretation needs the outcome estimate, its analysis population, missing-data handling, and precision—not only the study’s objective. Those details are not reported in the registry record, so conclusions must remain limited to the design and descriptive information that is recorded.

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 trial-specific limitations constrain interpretation. The registry describes a single-group study with allocation listed as not applicable and masking listed as none. Without a concurrent comparison group, the adverse-event summaries cannot establish how outcomes would compare under another intervention. The record’s design does not support a randomized estimate of a treatment difference.

The primary endpoint is defined as proportions of participants with treatment-related adverse events, assessed using CTCAE v4.0 over 8 month. However, an analyzed primary endpoint result is not reported. The registry does not report a numerical treatment-related event proportion, confidence interval, or statistical test. It also does not state how treatment relatedness was adjudicated or whether the adverse-event summary counts follow the primary endpoint’s definition and timeframe.

Participant characteristics and detailed eligibility criteria are not reported in the registry record. The analysis population, missing-data handling, and outcome-specific denominators are not described beyond the adverse-event at-risk counts. These gaps limit assessment of who contributed to each analysis and how representative the findings might be. No immunological-response result is reported despite its listing as a secondary outcome.

The record does not report the study’s sample-size justification, a statistical analysis plan, or any interim analysis procedures. The planning description uses hypothetical equal allocation between two arms, whereas the registered study is single group; those planning assumptions should not be mistaken for the trial’s actual allocation. Finally, the registry title references metastasis, but no metastasis result is reported in the available outcome information. These limitations do not imply unreported procedures or outcomes; they define what cannot be determined from the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: RV001V, a RhoC Anticancer Vaccine, Against Metastasis From Solid Tumours — NCT03199872.
• Registry record last updated 2020-06-09; status last verified 2020-06.
• Results first posted 2020-06-09.
• 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

NCT03199872 is recorded as a completed Phase I/II, single-group study sponsored by RhoVac APS. The registry lists enrollment of 22 patients with prostate cancer and the intervention RV001V, a vaccine described as RV001 peptide with Montanide ISA 51 adjuvant. Allocation is not applicable and masking is none. The recorded start date is 2017-03-30, and primary completion is dated 2018-08-01.

The primary outcome is the proportion of participants with treatment-related adverse events assessed using CTCAE v4.0 over 8 month. Immunological response is listed as a secondary outcome. The registry reports adverse-event summaries, including serious adverse events for 4 participants among 22 at risk, other adverse events for 22 among 22 at risk, and 0 deaths. Serious-event terms include Arthralgia, Impaired healing, and Pneumonia.

No outcome analyses are reported in the registry record. In particular, it does not provide an analyzed primary endpoint estimate, its precision, or an immunological-response result.