Personalized Infliximab Induction Strategy With Model-informed Dosing in Patients With Crohn's Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Personalized Infliximab Induction Strategy With Model-informed Dosing in Patients With Crohn's Disease (NCT04974099), a na phase 2, phase 3 study. The primary outcome is Obtain Safety Data for Optimal Dosing Strategy and Sample Size Estimation.

2. Clinical Question

The research question centers on using patient blood-testing results to inform infliximab dosing, rather than assigning participants to different dosing strategies for a controlled comparison. The brief summary frames the aim as determining whether a computer program can better achieve an optimal drug level than standard dosing. However, the registered design is single-group, with allocation listed as not applicable and no masking. The record therefore does not describe a like-for-like comparison that could estimate a difference between personalized dosing and standard dosing.

The intervention combines a device, the RoadMAB precision dashboard, and the drug, infliximab. The record describes use of the software to guide the initial dose and later dose and interval decisions. Its description of induction begins with infusions at 0 and 2, then ends before completing the schedule. The full induction schedule is not reported in the registry record.

The primary question is framed around safety and sample-size estimation, not a confirmatory test of clinical benefit. Secondary outcome titles refer to accuracy of infliximab concentration targets, clinical response, clinical remission, and sustained remission. The registry record does not report results for these outcomes. The central interpretive issue is therefore the distinction between the study’s stated dosing rationale and the limited outcome evidence recorded for this single group.

3. Trial Design

This is a single-group interventional study listed as phase 2 and phase 3. The allocation field is not applicable, masking is none, and the recorded enrollment is 6. These design features mean the record describes participants receiving the precision-dosing intervention without a separate randomized or masked comparison group.

The intervention consists of the RoadMAB precision dashboard and infliximab precision dosing. According to the arm description, the software guides selection of the first infliximab dose and later dose and interval decisions during maintenance. The description states that three infusions occur during induction and gives the first infusion times as 0 and 2, but the available description stops before specifying the full schedule. Additional details about how dosing decisions were made are not reported in the registry record.

The listed primary outcome is titled “Obtain Safety Data for Optimal Dosing Strategy and Sample Size Estimation.” It measures the percentage of participants with adverse and/or serious adverse events over 10 months. This framing is consistent with a study intended to collect preliminary safety information and inform future planning, but the registry record does not report a sample-size justification or explain the rationale for the recorded enrollment.

The record lists 20 outcome measures, including secondary outcomes on drug concentration targets, clinical response, clinical remission, and sustained remission. It lists 0 analyses. The study status is terminated. The start and primary completion dates are recorded as 2021-10-01 and 2024-08-01, respectively. The registry record does not explain why the study was terminated, whether follow-up was complete for all participants, or whether the termination changed the planned analyses.

Because the study has no control group, its design can describe observations among participants who received the intervention, but it cannot separate the effects of the dosing approach from other explanations through a concurrent comparison. The registry record does not report a protocol-level statistical analysis plan, an analysis population, or procedures for handling incomplete observations.

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 Obtain Safety Data for Optimal Dosing Strategy and Sample Size Estimation, measured over 10 months.

The primary endpoint is the percentage of participants with adverse and/or serious adverse events during a timeframe of 10 months. It is intended to summarize the proportion of the study group experiencing events relevant to safety while using the dosing strategy. Reporting a proportion can make event frequency easier to interpret than a count alone, because it relates events to the number of participants at risk. The registry record identifies the unit as a percentage of participants, but it does not report a primary endpoint percentage.

Adverse events and serious adverse events are not interchangeable categories. Seriousness is a classification of an event’s severity or consequences under clinical research reporting conventions; it does not by itself establish that the intervention caused the event. The endpoint description includes adverse and/or serious adverse events, while the posted safety figures separately report serious events and other adverse events. The registry record does not specify the exact rules used to classify or attribute events.

A safety endpoint is useful in an early or exploratory investigation because it records harms observed during exposure and can help inform subsequent research planning. It does not, on its own, establish how the intervention compares with another dosing strategy, nor does it determine whether any event was caused by treatment. That distinction is especially important in a single-group study, where there is no concurrent control group for comparison.

Although the endpoint title mentions sample-size estimation, the registry record does not report a sample-size calculation based on the safety endpoint or a justification for the enrollment. It also does not provide the primary endpoint result as a percentage or explain how participants with missing follow-up were handled. The posted event counts offer limited safety information, but they are not a complete account of the specified primary endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation depends on the design, endpoint, and quality of the recorded observations. In this study, the design is single-group, and the primary outcome is a percentage of participants with adverse and/or serious adverse events over 10 months. The registry record lists 0 analyses, so it does not document a statistical analysis of the primary endpoint or of the secondary outcomes.

What does a single-group design allow?

A single-group study can describe what was observed among participants receiving an intervention. It has no concurrent comparator group in which outcomes are observed under a different dosing strategy. Without that comparison, a difference attributable to the intervention cannot be distinguished from other influences, such as the course of the underlying condition, participant characteristics, or the circumstances of care. This is a general limitation of uncontrolled designs; it does not imply that any particular alternative explanation occurred in this study.

Allocation is listed as not applicable and masking as none. These entries are consistent with the absence of a randomized comparison and blinding. The record does not describe further procedures that might reduce observer or measurement bias. The intervention combines a precision dashboard with infliximab dosing, so observed outcomes relate to the study’s use of that approach as a whole; the record does not provide a method for isolating the contribution of the software from other components of care.

How should a percentage endpoint be understood?

A percentage endpoint expresses how many participants experienced a defined event relative to the participants included in the calculation. Its interpretation depends on a clear event definition, a clear observation period, and a clearly specified denominator. The registry record states the unit and the 10-month timeframe, but does not report a primary percentage or provide the detailed rules for combining adverse and serious adverse events. It also does not state whether the endpoint counts participants with at least one event or counts individual events; those are different summaries and should not be assumed to be equivalent.

Event counts are descriptive. A count does not show whether events were related to treatment, how severe non-serious events were, or when they occurred. Seriousness and causality are distinct judgments: an event may be serious without being caused by the intervention, and an event judged related need not be serious. The registry record does not give event-level descriptions or causality assessments.

Why does precision matter?

General statistical principle: an estimate from a small group can have substantial uncertainty, even when its observed value is straightforward to state. A confidence interval is a range calculated using a statistical model to convey uncertainty around an estimate. It is not a guarantee that the true value lies in the interval, and its interpretation depends on the model and data. The registry record does not report a confidence interval for the primary endpoint. Accordingly, readers cannot use a posted interval to judge the precision of the event-frequency estimate.

General statistical principle: when event data are sparse, conventional approximations can perform poorly, and methods appropriate for small samples may be needed. The record does not report which method, if any, was intended for estimating uncertainty around the safety endpoint. No unreported interval or test should be reconstructed from the event counts when the registry record does not state the analysis approach.

What do the planning assumptions mean?

The registry record describes planning illustrations based on an observed effect. It states that the illustrations assume a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a fraction of the observed effect. These are planning assumptions, not a description of this study’s single-group design. Any resulting sample-size figure would be an illustration for a hypothetical comparative study under those assumptions, not the enrollment target or sample-size justification for this trial.

Such planning values are not observed power. General statistical principle: observed power calculated from an observed effect does not add a separate measure of evidence beyond the effect estimate and its uncertainty. Sample-size planning is more informative when based on a prespecified effect of scientific interest, a chosen error rate, and a suitable design and outcome model. The registry record does not report the original sample-size justification, a prespecified effect for future planning, or a statistical plan connecting the illustrations to a later study.

Normal approximations are general methods that rely on conditions about the behavior of estimates. With binary outcomes and sparse observations, their suitability depends on the data and the specific calculation. The assumptions listed for an illustration do not establish that a normal approximation was used for the study’s own safety analysis. In addition, a two-arm planning calculation cannot create a control group that was absent from the trial.

What is not documented?

The registry record lists 20 outcome measures and 0 analyses. It does not describe a multiplicity strategy, missing-data procedures, an analysis population, or a prespecified statistical model. The primary endpoint description does not explain how overlapping categories of adverse and serious adverse events would be combined. The record also does not report confidence intervals, hypothesis tests, or adjusted estimates. These omissions limit evaluation of the precision and robustness of any outcome interpretation.

General statistical principle: over-dispersion matters when the variability in observed data exceeds what a chosen model expects. In event-count settings, ignoring excess variability can make uncertainty appear too small. The registry record does not report a count-model analysis or evidence of over-dispersion, so this issue cannot be evaluated as a feature of the study’s actual analysis. It is a general consideration, not a claim about these data.

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("RoadMAB Dashboard System")
n      <- c(6)
events <- c(3)
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  RoadMAB Dashboard System  6  3  50.0  11.8  88.2  18.8  81.2  18.4  81.6

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 20 outcome measures, including measures related to concentration targets, clinical response, clinical remission, and sustained remission. It lists 0 analyses. No multiplicity procedure or hierarchy among outcomes is reported in the registry record. Without an analysis plan, it is not possible to determine whether any outcomes were designated for formal testing, whether secondary outcomes were exploratory, or how the risk of chance findings across multiple evaluations was to be handled.

General statistical principle: when many outcomes or comparisons are examined, the chance of at least one apparently unusual result can increase unless the analysis is planned and interpreted with multiplicity in mind. This does not establish that a false-positive result occurred in this study. No analysis results are reported for the listed secondary outcomes, so there are no posted significance tests to interpret.

Missing-data methods are not reported in the registry record. The record does not state whether all participants completed the 10-month observation, how incomplete follow-up was handled, or whether the endpoint denominator included everyone enrolled or only participants with available outcome information. The participant population used for each analysis is also not described. These details matter because the interpretation of a percentage depends on who was observed and included in its denominator.

The registered enrollment is 6, but the record does not provide a flow of participants through treatment and follow-up or explain whether any outcome data were unavailable. No assumption about complete follow-up, imputation, or exclusion should be made. The posted safety figures give participants at risk for the listed group, but they do not substitute for a stated missing-data plan or a complete analysis-population definition.

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 non-serious adverse event terms

The RoadMAB Dashboard System group has 0 serious adverse events among 6 at risk. The record also reports 3 other adverse events among 6 at risk and 0 deaths. These are the posted descriptive safety figures. The record does not provide event names, severity details for the other adverse events, timing, or judgments about whether an event was related to the intervention.

The categories should be read as reported rather than combined into a new total. Serious adverse events and other adverse events are shown separately, and the registry record does not clarify whether participants could appear in both categories or how repeated events were handled. It would therefore be inappropriate to infer a number of participants with any adverse event by adding the category counts.

Observed absence of a serious event in this group is not proof that the intervention carries no serious-event risk. A small group offers limited information about events that may be uncommon, and the record does not report an uncertainty interval for the safety estimate. Conversely, the reported other adverse events do not establish that the intervention caused them. Event attribution, clinical context, and comparison with an appropriate control are not reported.

The primary endpoint calls for the percentage of participants with adverse and/or serious adverse events over 10 months, but the registry record does not report that combined endpoint as a percentage. The safety counts are therefore best understood as a limited descriptive account, rather than a complete estimate of comparative safety or causation.

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

The record documents a single-group investigation of a model-informed infliximab dosing strategy using the RoadMAB precision dashboard. It reports limited safety counts in the intervention group: 0 serious adverse events among 6 at risk, 3 other adverse events among 6 at risk, and 0 deaths. These observations describe what the registry record posts for the group; they do not establish what would have occurred under a different dosing strategy.

The primary outcome is framed as the percentage of participants with adverse and/or serious adverse events over 10 months. The registry record does not report that percentage, its uncertainty, or a detailed analysis. It also does not explain the reason for termination. The safety counts should therefore not be treated as a complete evaluation of the primary endpoint or as an explanation for the study’s status.

The record does not provide results for its secondary outcome titles concerning concentration targets, clinical response, clinical remission, or sustained remission. Those titles indicate intended areas of evaluation, not evidence that any target was reached or any clinical outcome changed. The brief summary describes the rationale for studying personalized dosing, but rationale is not a result.

This design cannot support a controlled estimate of how personalized dosing compares with standard dosing because no concurrent control group is described. It also cannot establish that an event was caused by the dosing approach. Even a reported absence of a serious event in a small group would not rule out events that were not observed during the study.

Planning illustrations based on an observed effect are not evidence of efficacy, safety, or observed power. The statistical assumptions listed for those illustrations concern a hypothetical planning exercise and do not alter the design or findings of the registered study. In short, the registry record offers a limited descriptive safety account and documents the intended questions, while leaving the comparative and clinical conclusions unresolved.

14. Why This Trial Matters Statistically

This study is statistically notable because its title and rationale concern individualized dosing, while the registered design is single-group. That distinction matters: a personalized strategy may be evaluated descriptively in one group, but claims about improvement over standard dosing require a valid basis for comparison. The brief summary refers to standard dosing as a point of comparison in the research motivation; the registered design does not describe a standard-dosing control group.

The primary endpoint also highlights a common challenge in early clinical research. Safety information can be useful for planning further study, but the usefulness of an estimate depends on its precision, event definitions, follow-up completeness, and a transparent analysis. Here, the endpoint is defined as a percentage over 10 months, while a primary percentage, confidence interval, and detailed analysis are not reported in the registry record. The posted counts are informative as a narrow description, but they do not resolve the uncertainty around event frequency.

The record lists secondary measures spanning drug concentration targets and clinical outcomes. Such measures can help connect dosing decisions with pharmacologic and clinical questions, but outcome titles alone cannot establish what the study found. The absence of listed analyses and results makes it especially important to distinguish intended measurement from reported evidence.

Finally, sample-size planning should be separated from the trial’s observed results. The record describes assumptions for planning illustrations, but not the original sample-size justification. Illustrative values based on an observed effect do not amount to observed power and do not remedy the absence of a comparator. These distinctions help readers assess what the study can contribute: descriptive experience and potential planning information, rather than a controlled estimate of treatment differences.

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 gives limited information for evaluating this study’s results. It describes a single-group design, with allocation not applicable and masking none, so there is no concurrent control for interpreting outcomes relative to another dosing approach. The record does not report a statistical analysis plan, and it lists 0 analyses. It therefore does not explain how the primary or secondary outcomes were to be analyzed.

The recorded enrollment is 6, which limits the amount of information available for estimating event frequency and makes rare outcomes difficult to characterize. The safety data report serious events, other adverse events, and deaths, but do not identify event types, timing, severity details for other events, or causal attribution. The primary endpoint percentage and its uncertainty are not reported.

The record lists 20 outcome measures but does not report a multiplicity strategy, hierarchy, or results for the secondary outcomes. It also does not provide an analysis population, participant flow, missing-data procedures, or confirmation that all participants completed the 10-month observation. The denominator and event-counting rules for the primary endpoint are not fully specified.

The intervention description is incomplete: it identifies the dashboard and infliximab dosing, and gives infusion times of 0 and 2, but does not finish the induction schedule. The record does not report detailed dosing algorithms or the exact decision rules used by the software. The reason for the terminated status is also not reported in the registry record.

These limitations do not show that the intervention was ineffective or unsafe. They identify why the posted record cannot support a precise estimate of safety, a causal conclusion about adverse events, or a comparative claim about dosing strategies.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Personalized Infliximab Induction Strategy With Model-informed Dosing in Patients With Crohn's Disease — NCT04974099.
• Registry record last updated 2026-03-11; status last verified 2026-02.
• Results first posted 2026-03-11.
• 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

NCT04974099 is a phase 2 and phase 3, single-group interventional study of RoadMAB-guided infliximab dosing for Crohn disease. The registry record lists enrollment of 6, no masking, and a terminated status. Its primary outcome is the percentage of participants with adverse and/or serious adverse events over 10 months, framed as supporting safety assessment and sample-size estimation.

The posted safety figures report 0 serious adverse events among 6 at risk, 3 other adverse events among 6 at risk, and 0 deaths. The record does not report the primary endpoint as a percentage, an uncertainty interval, or results for the listed secondary outcomes. It lists 20 outcome measures and 0 analyses.

Because the design has no separate control group, the record supports descriptive interpretation of the posted observations, not a controlled comparison with standard dosing. The statistical analysis plan, missing-data procedures, and reason for termination are not reported in the registry record.