Inhibition of Oral Tumorigenesis by Antitumor B: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Inhibition of Oral Tumorigenesis by Antitumor B (NCT04278989), a na early/phase 1 study. The primary outcome is Percent of Ki-67 Positive Cells.

2. Clinical Question

3. Trial Design

This is an early-phase 1, single-group, window-of-opportunity study of Anti-tumor B in squamous cell cancer of the oral cavity. The record lists the Medical College of Wisconsin as sponsor, an enrollment of 30, and a completed status. The recorded study start is 2022-04-22 and the primary completion date is 2025-05-19. These record dates describe study administration; they do not supply endpoint findings.

The intervention arm is described as Anti-tumor B at 1,200 mg three times a day. Masking is listed as none and allocation as NA. Although the design is labeled single group, the primary outcome description refers to a control group composed of seven placebo recipients and seven matched controls. The registry also explicitly cautions that this is not a randomized comparison and that the arms are not a like-for-like control comparison. The record does not clarify the relationship between the stated model and these control observations.

The primary endpoint is a continuous measure assessed at baseline and after surgery, seven to 28 days after baseline. The outcome description says that 16 subjects received Anti-tumor B and that one subject with only one dose was removed for analysis. It does not give a separate analysis population or explain how the placebo and matched-control observations were handled. The record lists one outcome measure and zero analyses. It does not report a protocol-level analysis plan, sample-size justification, or interim-analysis plan.

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.

Before assignment: This began as a randomized study with a parallel design. Part way through it, the investigator decided to make it an open label, single arm study that would just recruit subjects taking ATB. The control group represents seven placebo subjects and seven samples taken from the local tissue bank.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Sixteen subjects received ATB, however, one subject had only one dose and was removed for analysis. The control group consisted of seven subjects who received placebos and seven matched controls.

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: The untreated group consists of seven placebo subjects and seven samples taken from a local tissue bank. We have no demographics or AE information about the seven subjects whose samples were taken. Also, for the ATB group, we are counting 16 subjects who began although one didn't complete and wasn't used for analysis.

6. Primary Endpoint

The registry lists the primary outcome as Percent of Ki-67 Positive Cells, measured over Baseline and after surgery (seven to 28 days after baseline).

The primary endpoint is the percent of Ki-67-positive cells, expressed as the percentage of stained tumor cell nuclei. The registry describes an immunohistochemical evaluation using the antihuman Ki-67 monoclonal antibody MIB-1 and counting positively stained malignant cells. Measurements were scheduled at baseline and after surgery, with the later assessment occurring seven to 28 days after baseline.

Ki-67 staining is used as a measure of cellular proliferation. In general, a continuous endpoint retains information about the measured value rather than reducing it to a yes-or-no category. Comparing measurements over time can describe whether the measured marker changes during a study period, while comparing groups may help contextualize that change. Those interpretations depend on how the observations are collected and analyzed.

The registry record identifies the endpoint type as a mean with standard deviation, but it does not provide the mean, standard deviation, or any numerical comparison. It also does not report whether the primary analysis used individual changes, a comparison of measurements at each time, or another approach. Thus, the endpoint definition is available, but the magnitude, variability, and precision of the results are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a continuous primary measure summarized as a mean with standard deviation. It lists one outcome measure and zero analyses. The outcome is defined, but its numerical results and the methods used to compare measurements are not reported in the registry record. The discussion below explains general statistical concepts relevant to this type of endpoint; it does not describe an analysis that the record confirms was performed.

What does a continuous endpoint represent?

In general, a continuous endpoint can take a range of measured values. A mean describes the center of a set of observations, while a standard deviation describes how spread out individual observations are around that mean. Reporting both can give a basic account of the measured distribution, although the mean and standard deviation alone do not show every feature of that distribution. For the Ki-67 endpoint, the record identifies the measure as a percentage of stained tumor cell nuclei, but does not report either summary statistic.

Because measurements were specified at baseline and after surgery, analysis might focus on changes within participants, on measurements at a particular time, or on a contrast between groups. These approaches answer different questions and rely on different assumptions. The registry record does not state which approach was used, whether measurements were paired for analysis, or whether covariates were included. Those details should not be inferred from the endpoint title or time frame.

What would a confidence interval communicate?

In general, a confidence interval gives a range of values compatible with an estimate under the chosen statistical method and its assumptions. A narrower interval indicates greater precision for that estimate, while a wider interval indicates more uncertainty. The interval does not repair limitations in study design, address unmeasured differences between groups, or show that a result is important in practice. No confidence interval for the primary endpoint is reported in the registry record.

What is the role of a p-value?

In general, a p-value assesses how unusual the observed data would be under a specified null model, assuming that model and the analysis assumptions are appropriate. It does not state the probability that the null hypothesis is true, and it does not measure the size or importance of an effect. A p-value is most interpretable alongside an effect estimate and its uncertainty. The record lists zero analyses and reports no primary-endpoint p-value.

Why does the design matter?

In general, random allocation can reduce systematic differences between groups in expectation, while masking can reduce some forms of measurement or behavior-related bias. This record lists allocation as NA and masking as none, and explicitly says the comparison is not randomized or like-for-like. It labels the model single group, but the outcome description includes placebo recipients and matched controls. Without an explanation of how these control observations were formed and analyzed, a group contrast cannot automatically be interpreted as an effect attributable to the intervention.

What does over-dispersion mean in general?

In general, over-dispersion occurs when observed variability is greater than a statistical model assumes. If a model understates variability, standard errors may be too small and confidence intervals too narrow. Choice of a suitable model depends on the outcome scale, data distribution, and study design. The primary outcome is described as continuous and summarized with a mean and standard deviation; the registry record does not report a model or diagnostics, so there is no basis to say that over-dispersion was assessed or was a concern.

How should planning calculations be understood?

The record states that its sample-size assumptions are a planning illustration computed from the observed effect, not the trial's own design. It describes a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. For time-to-event outcomes, its general note says planning totals refer to events; for other outcomes they refer to participants per arm. The primary outcome in this trial is continuous, not time-to-event. No numerical sample-size illustration is included in the registry record for this interpretation, and such an illustration would not be observed power or evidence that the study was planned around that effect.

More generally, planning estimates depend on assumptions about the effect, variability, allocation, and analysis. If those assumptions differ from the eventual study data, the resulting planning requirement may not describe the study's actual precision. The registry record does not report the original sample-size justification or enough primary endpoint statistics to evaluate a trial-specific planning calculation.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and zero analyses. It names the percentage of Ki-67-positive cells as the primary endpoint, but the actual numerical results are not reported. There is no reported primary analysis model, estimate, uncertainty interval, or hypothesis test to assess.

Multiplicity concerns arise when several outcomes, time points, subgroups, or statistical tests are examined. In general, testing multiple hypotheses without an appropriate strategy can increase the chance of a chance finding. The record identifies a baseline and a post-surgery measurement, but does not describe the planned contrast, additional analyses, or any adjustment for multiple testing. An interim-analysis plan is not reported in the registry record. These absences do not establish that such procedures did or did not occur; they mean the record does not provide the information needed to assess them.

The outcome description states that one participant who had only one dose was removed for analysis. It does not provide a complete account of the analysis population, the handling of other missing measurements, or the reason for excluding participants beyond that description. Missing-data methods, an intention-to-treat analysis, and a per-protocol analysis are not reported in the registry record. The study's single-group label also sits alongside descriptions of placebo recipients and matched controls, without a clear account of which participants were included in which analysis.

In general, missingness can affect an estimate when the chance that an outcome is unavailable is related to participant characteristics or the unobserved outcome. Complete-case analysis, imputation, and sensitivity analyses have different assumptions. No missing-data strategy or sensitivity analysis is reported, so the effect of missing observations on the primary result cannot be evaluated.

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: neck abscess (14.3%), dysphagia (14.3%), lung infection (6.2%), pleural infection (6.2%), anemia (6.2%).

The registry lists safety information for an Anti-tumor B group and an Untreated Group. For Anti-tumor B, 16 were at risk for serious adverse events and 4 were affected; 16 were at risk for other adverse events and 14 were affected. For the Untreated Group, 7 were at risk for serious adverse events and 1 was affected; 7 were at risk for other adverse events and 7 were affected. The record reports zero deaths in each group.

The registry also lists serious adverse-event terms with percentages: Neck abscess and dysphagia at 14.3% each, and Lung infection, Pleural infection, Anemia, and Vomiting at 6.2% each. The record does not specify in the registry record how those term percentages map to the listed groups or whether the percentages refer to participants or events. They should therefore be interpreted as reported terms and percentages, not as a fully described group-specific comparison.

In general, adverse-event counts describe events recorded during a study, but do not by themselves establish causation. The untreated group is not described as a randomized, like-for-like comparator, and the record explicitly cautions against treating the arms as a randomized comparison. The counts should not be interpreted as proof that Anti-tumor B caused or prevented any event.

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

The record establishes that a study of Anti-tumor B was conducted in the setting of squamous cell cancer of the oral cavity, with a primary measure based on Ki-67 staining before and after surgery. It also records the intervention dose, the planned assessment window, and limited participant and safety information. Those details describe the study question and its recorded conduct, but do not provide a numerical primary endpoint result.

The registry record does not report means, standard deviations, changes, group contrasts, confidence intervals, or p-values for Ki-67-positive cells. Consequently, it does not support a statement about the size, direction, or precision of a change in that marker. Nor does the absence of a numerical result establish that there was no change; the result is simply not reported in the registry record.

Even if a numerical group difference were available, the design information would matter. The record labels the study single group, lists allocation as NA and masking as none, and describes placebo recipients and matched controls. It also cautions that the comparison is not randomized and that the arms are not like-for-like. In general, differences between non-randomized groups can reflect differences in who entered each group as well as differences associated with an intervention. The registry record does not describe methods that would resolve that issue.

The safety counts provide a record of reported adverse events, not a causal conclusion. The listed group sizes and event counts cannot, on their own, establish why events occurred. The serious-event terms are reported with percentages, but the registry entries do not fully explain their group attribution or denominator.

Accordingly, the record supports a description of the study and its endpoint, but not a conclusion about an intervention effect on Ki-67 or a causal safety comparison. The biological marker outcome should also not be treated as a direct measure of a clinical outcome; the registry entries do not report clinical efficacy results.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of why a clearly named endpoint is not the same as a reported result. The record specifies a continuous Ki-67 measure, its staining method, and its assessment timing. It does not include the numerical summaries needed to understand the observed endpoint values or their precision. Readers therefore need to distinguish a defined outcome from an available analysis.

The design descriptions also illustrate why group labels alone do not establish a valid comparison. The model is listed as single group, while the outcome description includes placebo recipients and matched controls. Allocation is NA, masking is none, and the registry cautions that the arms are not a randomized, like-for-like comparison. These details limit what can be concluded from any between-group contrast and make clarity about group formation and analysis especially important.

Another statistical issue is transparency about who contributed data. The endpoint description notes that one participant with only one dose was removed for analysis, but the record does not fully describe the analysis population or missing-data handling. Such information helps readers understand whether the analyzed participants differ from those initially enrolled and how robust an estimate might be to unavailable observations.

Finally, the record separates safety counts from the primary biological endpoint, but provides limited context for interpreting the serious-event terms. Clear denominators, group attribution, and event definitions matter when readers compare safety reports. The study therefore highlights the value of reporting estimates, uncertainty, analysis populations, and design details alongside an endpoint definition.

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 primary limitation for statistical interpretation is that numerical results for the primary endpoint are not reported in the registry record. The record provides no means, standard deviations, changes, contrasts, confidence intervals, or p-values for Ki-67-positive cells. The listed count of zero analyses reinforces that a completed endpoint analysis cannot be evaluated from these registry entries.

The design is difficult to interpret fully. It is labeled single group, but the outcome description includes placebo recipients and matched controls. Allocation is NA, masking is none, and the registry explicitly cautions that the comparison is not randomized and the arms are not like-for-like. The record does not explain control selection, group comparability, or the analysis linking these groups.

The analysis population is also incompletely described. The record says that one subject who had only one dose was removed for analysis, but does not report the handling of missing endpoint observations or a full accounting of participants contributing data at each assessment. Baseline group characteristics, outcome distributions, and any adjusted analysis are not reported in the registry record.

The safety information has limitations as well. The serious-event terms have percentages, but the record does not specify in the registry record their group attribution or denominator. Counts of events do not establish causation, especially in a comparison that is not randomized and is not described as like-for-like.

Finally, the record does not report a sample-size justification, interim-analysis plan, or detailed statistical analysis plan. Although it states general assumptions for planning illustrations, those assumptions are not the trial's own design justification. These gaps limit assessment of precision, multiplicity, missing-data bias, and the intended analysis.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Inhibition of Oral Tumorigenesis by Antitumor B — NCT04278989.
• Registry record last updated 2026-03-25; status last verified 2026-03.
• Results first posted 2026-03-25.
• 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

The record concerns NCT04278989, titled Inhibition of Oral Tumorigenesis by Antitumor B. It lists an early-phase 1, single-group study of Anti-tumor B for squamous cell cancer of the oral cavity, sponsored by the Medical College of Wisconsin. Enrollment is 30, and status is completed. The recorded start date is 2022-04-22 and primary completion date is 2025-05-19.

Anti-tumor B is described as a six-herb botanical agent, administered at 1,200 mg three times a day. The primary outcome is the percentage of Ki-67-positive tumor cell nuclei, assessed at baseline and after surgery, seven to 28 days after baseline. The record describes an intervention group and placebo and matched controls, while warning that the comparison is not randomized or like-for-like.

Numerical primary endpoint results and analyses are not reported in the registry record. Safety counts and serious-event terms are listed, but do not establish causation.