Perioperative MVT-5873, a Fully Human Monoclonal Antibody Against a CA 19-9 Epitope, for Operable CA 19-9 Producing Pancreatic Cancers, Cholangiocarcinomas, and Metastatic Colorectal Cancers: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Perioperative MVT-5873, a Fully Human Monoclonal Antibody Against a CA 19-9 Epitope, for Operable CA 19-9 Producing Pancreatic Cancers, Cholangiocarcinomas, and Metastatic Colorectal Cancers (NCT03801915), a non-randomized phase 2 study. The primary outcome is Number of Participants With Disease Recurrence At 1 Year.

2. Clinical Question

3. Trial Design

This was a phase 2, non-randomized, unmasked, sequential study sponsored by the National Cancer Institute. The registry lists enrollment of 10 and a completed status. The study started on 2019-11-13, with primary completion on 2022-05-27. These design features describe the registered study; they do not supply an outcome result.

Participants were assigned to sequential cohorts rather than randomized between treatment groups. Cohort 1 received pre-operative escalation doses of MVT-5873, surgery by pancreatectomy or hepatectomy, and post-operative MVT-5873. Cohort 2 received the pre-operative recommended dose, followed by surgery and post-operative treatment. The registry record does not report the rationale for the cohort sequence or the criteria used to choose the recommended dose.

The study plan described a dose before surgery, surgery with tissue collection for research, post-operative recovery and monitoring, and subsequent MVT-5873 treatment. The brief description specifies weekly treatment for 1 month after hospital discharge, followed by treatment every other week for 2 months. The registry record does not report how many participants completed each part of this planned schedule.

Because the study was not randomized and had no masking, comparisons between cohorts are descriptive rather than controlled treatment comparisons. Cohort membership may reflect the sequential design and other factors that the record does not describe. The record does not report a separate untreated or standard-care comparison group, nor does it report a statistical plan for estimating differences between the cohorts.

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.

Population: One participant was enrolled but not treated. However, collected baseline data is reported here.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Disease Recurrence At 1 Year, measured over 1 year.

The primary endpoint was the number of participants with disease recurrence at 1 year. The registry defines recurrence as new disease measurable on computed tomography or magnetic resonance imaging that was not present on baseline imaging. This is a binary outcome at the participant level: a participant either meets that stated recurrence definition or does not, provided the relevant assessment is available.

In general, recurrence endpoints are used to describe whether disease returns after treatment and can offer a clinically interpretable summary of disease status at a specified follow-up point. The fixed assessment time makes clear which point in follow-up the endpoint concerns. It does not, by itself, describe when recurrence occurred, how long participants remained recurrence-free, or what happened after the assessment.

The registry record names the endpoint and its definition but does not report the observed number of participants with recurrence, the number assessed, or the result by cohort. It also does not report how missing imaging or incomplete follow-up was handled. Therefore, the endpoint can be described from the registry, but its observed frequency and precision cannot be evaluated from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary endpoint is a participant-level count of recurrence events at a defined follow-up time. In general, an endpoint of this kind is often analyzed as a binary outcome: each participant with an available assessment is classified according to whether the recurrence definition is met. A count is useful for stating how many participants met the criterion, while a proportion would additionally require a clearly defined denominator. The registry record supplies neither a recurrence count nor the number assessed at 1 year, so neither quantity can be interpreted from the record.

What does non-randomized, sequential allocation imply?

Random allocation is a general method for balancing known and unknown prognostic factors between comparison groups, on average. In this study, allocation was non-randomized and the model was sequential. The cohorts therefore do not constitute a randomized comparison. A difference in outcomes could reflect cohort composition, timing, dose approach, follow-up, or other factors; the registry record does not describe a method to separate these possibilities. Cohort comparisons should consequently be treated as descriptive unless a suitable analysis and its assumptions are reported.

The study was unmasked. In general, masking can reduce the influence of knowledge of treatment assignment on outcome assessment or reporting. The primary endpoint uses imaging-defined recurrence, but the registry record does not say whether image review was centralized or blinded, or how disagreements in assessment were handled. Those procedures are not reported in the registry record.

How should a confidence interval be understood?

In general, a confidence interval expresses statistical uncertainty around an estimate. A narrower interval indicates greater precision under the chosen analysis assumptions; a wider interval indicates less precision. It is not a guarantee that the true value lies in the interval, and it does not resolve biases created by study design or missing data. The registry record does not report a confidence interval for the primary endpoint, so no precision claim about recurrence can be made from the posted information.

What do the sample-size illustrations represent?

The planning illustrations use the observed effect as a starting point and consider detecting a stated fraction of that effect. Their assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are planning illustrations, not the study's own design justification and not observed power. In general, a planning calculation estimates the amount of information needed under specified assumptions; it does not show that a study actually had that amount of information or that an effect was detected. The record does not report the study's own sample-size justification or statistical plan.

For a binary endpoint, the calculation depends on the outcome rates assumed for the comparison and on how groups are allocated. A normal approximation is a mathematical shortcut for estimating uncertainty or required sample size. General statistical caution is warranted when event counts or groups are small, because approximations may not represent uncertainty well. The registry does not report enough endpoint data to assess how well any approximation would fit the primary outcome in this study.

Why does over-dispersion matter in general?

Over-dispersion means that observed variability is greater than a statistical model expects. It can lead a model to understate uncertainty if the extra variation is ignored. This concept is relevant to some count-based models, but the registry record does not identify an over-dispersion assessment or report a model for the primary endpoint. The endpoint is described as a count of participants with recurrence, and no analysis method is listed.

The registry reports no analyses. It does not report whether the primary endpoint was analyzed by cohort, whether covariates were included, how censoring or incomplete follow-up was treated, or which statistical test or model was used. These absences limit reproducibility: readers cannot evaluate whether the analytic approach matched the endpoint, whether assumptions were checked, or how estimates and uncertainty were obtained.

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("Cohort 1 - Pre-operative Escalation Doses of MVT-5", "Cohort 2 - Pre-operative Recommended Dose (RD) of ")
n      <- c(8, 1)
events <- c(2, 0)
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  Cohort 1 - Pre-operative Escalation Doses of MVT-5  8  2  25.0  3.2  65.1  7.1  59.1  7.5  60.0
ARM  Cohort 2 - Pre-operative Recommended Dose (RD) of   1  0  0.0  0.0  97.5  0.0  79.3  1.3  84.2

11. Multiplicity, Missing Data and Analysis Populations

The registry record does not report a statistical plan addressing multiplicity. The listed primary endpoint concerns recurrence at 1 year, and a secondary outcome concerns defining disease-free survival for participants treated with pre-operative MVT-5873. The record does not say whether other outcomes or cohort comparisons were tested together, or whether any adjustment was planned for multiple statistical tests. In general, testing several hypotheses can increase the chance of at least one apparently notable result; a prespecified approach helps clarify how results should be interpreted. No such approach is reported in the registry record.

The record also does not describe missing-data methods or analysis populations. It does not state how participants with no 1-year imaging, incomplete follow-up, or deviations from the planned treatment schedule were handled. The number assessed for the primary endpoint and the number included in any analysis are not reported. These details matter because an observed count is interpretable only alongside the population assessed and the handling of unavailable outcomes.

The record lists enrollment of 10, but that does not identify how many participants were evaluable for each outcome. It does not report whether the primary analysis used all enrolled participants, only those receiving treatment, or another population. No imputation method, censoring rule, or sensitivity analysis is reported. Consequently, the impact of missing outcome information cannot be assessed from 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: alkaline phosphatase increased (100.0%), hepatobiliary disorders - other, bile duct perforation (100.0%), gastric ulcer (12.5%), thromboembolic event (12.5%).

The registry reports serious and other adverse-event figures separately for Cohort 1 and Cohort 2. For serious adverse events, Cohort 1 lists 8 participants at risk and 2 affected; Cohort 2 lists 1 at risk and 1 affected. For other adverse events, Cohort 1 lists 8 at risk and 7 affected; Cohort 2 lists 1 at risk and 1 affected. Deaths are listed as 1 in Cohort 1 and 0 in Cohort 2. These are the posted counts and should be read within the cohort labels and at-risk figures rather than as a randomized comparison.

The record lists serious-event terms including “Alkaline phosphatase increased” and “Hepatobiliary disorders - Other, Bile duct perforation,” each with 100.0%, as well as “Gastric ulcer” and “Thromboembolic event,” each with 12.5%. The denominator and the detailed meaning of these percentages are not reported in the registry record, so they should not be interpreted as overall risks among all enrolled participants.

These figures do not establish that the study drug caused an event. The registry record does not provide event timing, attribution to treatment, duration, severity grades, or participant-level details. The cohorts are not randomized, and their reported at-risk figures differ. The counts therefore describe reported safety experience, not a controlled estimate of comparative harm.

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

The registry record describes a phase 2 study of MVT-5873 given around surgery for specified CA 19-9-producing cancers. It identifies disease recurrence at 1 year as the primary endpoint and gives an imaging-based definition. It also records serious and other adverse-event counts by cohort. These details clarify the study question and the outcomes the registry intended to capture.

However, the registry record does not report the primary endpoint result. It contains no recurrence count, no number assessed at 1 year, and no analysis result. It therefore cannot show from the posted information how frequently recurrence occurred or how precise any estimate might be. The absence of an analysis in the record is not evidence that no events occurred; it means the outcome is not reported there.

The safety counts likewise should not be taken as proof that MVT-5873 caused or prevented an event. Attribution, event timing, severity details, and complete follow-up information are not reported. The non-randomized, sequential design also means the cohorts do not provide a controlled comparison. Differences between them cannot be attributed to dose approach from these figures alone.

In general, a trial's result depends not only on its endpoint definition but also on which participants were assessed, how missing outcomes were handled, and how uncertainty was estimated. Those analysis details are not reported in the registry record. The sample-size illustrations are planning exercises under stated assumptions, not observed power and not evidence of efficacy. The record supports a description of the study design and its reported safety counts, but not a conclusion about treatment benefit or a causal treatment effect.

14. Why This Trial Matters Statistically

This study is statistically instructive because it pairs a clearly defined, imaging-based recurrence endpoint with a non-randomized sequential design. The distinction matters: a well-specified endpoint helps readers understand what was intended to be measured, but it cannot by itself make comparisons unbiased or provide an estimate when the result is not reported.

The cohorts represent different pre-operative dosing approaches, but they are not randomized control groups. This illustrates why treatment-group contrasts need to be interpreted in light of allocation. Without randomization or a reported adjustment strategy, differences may reflect factors other than the intervention. A comparison can be descriptively informative while remaining unsuitable as a causal estimate.

The registry record also highlights how much outcome interpretation depends on reporting. Naming recurrence at 1 year is not enough to judge its frequency or precision: readers also need the observed count, the number assessed, and a description of missing-data handling. Those details are not reported for the primary endpoint. In general, transparent reporting of denominators and analysis methods makes it possible to distinguish a result from an endpoint definition or a study plan.

Finally, the planning assumptions attached to sample-size illustrations should be kept separate from the evidence generated by the trial. A calculation based on an observed effect and a hypothetical allocation can help frame future planning, but it is not observed power and does not substitute for a reported endpoint analysis. The distinction between planning, observed data, and causal inference is central to sound clinical-trial interpretation.

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 leaves important uncertainties specific to this study. Although it lists enrollment of 10 and a completed status, it does not report the number assessed for disease recurrence at 1 year, the recurrence count, or the endpoint result by cohort. Without those details, the primary outcome cannot be quantified or its precision evaluated.

The study used non-randomized, sequential allocation and no masking. The registry record does not explain how participants entered each cohort, why the recommended dose was selected, or whether cohort differences were addressed analytically. Thus, cohort outcomes cannot be treated as a randomized comparison. The record also does not report whether imaging was reviewed centrally or without knowledge of treatment cohort.

The registry does not describe the statistical analysis plan, analysis populations, missing-data procedures, censoring rules, or multiplicity approach. It does not report a sample-size justification for the trial. The planning assumptions attached to sample-size illustrations are not a substitute for those missing design and analysis details.

Safety reporting is also limited in the available record. Counts are shown by cohort, but event-level timing, attribution, severity grading, duration, and clinical course are not reported. The denominator for the listed serious-event percentages is not reported in the registry record. These limitations restrict both the interpretation of the safety summaries and any attempt to compare cohorts.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Perioperative MVT-5873, a Fully Human Monoclonal Antibody Against a CA 19-9 Epitope, for Operable CA 19-9 Producing Pancreatic Cancers, Cholangiocarcinomas, and Metastatic Colorectal Cancers — NCT03801915.
• Registry record last updated 2023-03-21; status last verified 2023-02.
• Results first posted 2023-03-21.
• 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

NCT03801915 is a completed phase 2, non-randomized, unmasked, sequential study sponsored by the National Cancer Institute. The registry lists enrollment of 10, a start date of 2019-11-13, and primary completion on 2022-05-27. The study evaluated perioperative MVT-5873 alongside pancreatectomy or hepatectomy in specified CA 19-9-producing cancers.

The primary endpoint was the number of participants with disease recurrence at 1 year, defined by new disease measurable on CT or MRI that was absent on baseline imaging. The registry record does not report a result for that endpoint or list analyses. It reports serious and other adverse-event counts and deaths by cohort, but does not provide detailed event attribution or course. The cohorts were not randomized controls, so their figures do not support a like-for-like treatment comparison.