Exploratory Study to Assess Delivery of LMN-201 Components Via Enteric Capsules in the Gut of Individuals With Ostomies: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Exploratory Study to Assess Delivery of LMN-201 Components Via Enteric Capsules in the Gut of Individuals With Ostomies (NCT04893239), a non-randomized phase 1 study. The primary outcome is Number of Participants With Presence of Capsules and/or Transit Markers in Ostomy Fluid by Visual Observation.

2. Clinical Question

The central question is about delivery and transit, rather than a clinical outcome such as infection resolution. The endpoint asks whether capsule material or markers can be seen in fluid collected from an ostomy. This makes the ostomy fluid a route for observing whether the study materials or markers appear after dosing.

That question is relevant to an exploratory assessment of enteric capsule delivery, but a visual observation does not by itself establish how much of a component reached a particular intestinal location, whether it remained intact, or whether it had a biological effect. Those questions would require measurements and outcomes beyond the endpoint definition stated in the registry record.

The study enrolled 12 participants and had four named cohorts: one for each of VHH-1, VHH-2, and VHH-3, and one for the combination. The record does not report a comparison hypothesis between these cohorts. Because there was no random allocation or control arm, the cohort structure does not support a controlled estimate of comparative effects.

3. Trial Design

The registry identifies this as a Phase 1, single-site, open-label, exploratory study. It was completed, with a recorded enrollment of 12. The study start date was 2021-08-18 and the primary completion date was 2022-02-23. Its stated purpose was to assess delivery of LMN-201 components via enteric capsules in the gut of individuals with ostomies.

Allocation was non-randomized, masking was none, and the model is listed as single group. The interventions and cohort descriptions identify three cohorts receiving individual anti-toxin B VHH components and a fourth cohort receiving VHH 1, 2, and 3 together. The record does not describe the sequence or rationale for assigning participants to cohorts, so the cohort labels should not be interpreted as randomized groups.

Open-label means that masking is not reported; visual observation was the specified endpoint method. This matters because an observer who knows that a participant received capsules may be more susceptible to expectation influencing a subjective observation than an observer who is unaware of treatment assignment. The record does not state whether observations were independently reviewed, whether standardized criteria were used, or whether assessors were blinded.

The registry record does not report the protocol’s sample-size justification, statistical analysis plan, or any interim-analysis plan. It also does not report a comparator intervention or a randomized control. Therefore, the design is best read as exploratory: it can describe the recorded observations for the enrolled cohorts, but it is not a controlled trial designed to establish comparative treatment effects.

The study lists one primary outcome measure, the number of participants with capsules and/or transit markers present in ostomy fluid by visual observation. Its timeframe includes arrival (baseline), predose (immediately before dosing), and up to 12 hours post dose. The outcome result itself is 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

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: Adults aged 19 and above with mature ileostomy.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Presence of Capsules and/or Transit Markers in Ostomy Fluid by Visual Observation, measured over Arrival (baseline), Predose (immediately before dosing), and up to 12 hours post dose..

The primary endpoint counts participants with presence or absence of capsules and/or transit markers in ostomy fluid, based on visual observation. Observations were specified at arrival (baseline), predose (immediately before dosing), and up to 12 hours post dose. The registry describes the outcome as a count of participants at each collection timepoint.

This endpoint is used to examine whether visible capsule material or a transit marker appears in fluid collected from the ostomy after dosing. In an exploratory delivery study, the observation can provide a direct, practical indication that material or marker has reached the output being collected. Repeated collection times can also describe when visible material is observed relative to dosing, provided the timepoint-specific results are available.

The endpoint is binary at the participant level: presence or absence. A count of participants with presence can summarize the observation within a cohort and timepoint, while the absence category indicates that visual identification was not made at that collection. Neither classification quantifies the amount of material, its integrity, its concentration, or the location at which release occurred.

Visual observation also depends on how collection and assessment were conducted. The registry record does not report operational definitions for a positive observation, assessor procedures, or methods to verify the visual finding. It does not give observed counts by cohort or collection time. Consequently, the endpoint’s definition is clear in broad terms, but its actual findings and measurement reliability are not reported in the registry record.

Presence of a capsule or marker is a delivery observation, not by itself evidence of a clinical benefit or harm. The endpoint does not measure infection outcomes, symptom change, or treatment efficacy.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registered primary outcome is a count, not a continuous measurement. For each collection timepoint, the underlying observation is whether a participant had visible capsules and/or transit markers in ostomy fluid. A count can then summarize the number of participants meeting that presence criterion. The registry record does not provide those counts, so no observed rate or contrast can be described.

What a presence-or-absence endpoint captures

A binary endpoint reduces an observation to two categories: present or absent. This can make a question about visible delivery straightforward to record. However, it discards gradations such as the amount seen, the number of markers, or the degree of capsule integrity. Since the registered measure is a participant count, it also does not specify how repeated observations for the same participant were combined into any overall result. No such summary method is reported in the registry record.

The stated assessment times include arrival (baseline), predose (immediately before dosing), and up to 12 hours post dose. Measurements at multiple timepoints can be informative because they describe observations over time. If the same participants contribute observations at several times, those observations are related rather than independent. A statistical analysis should account for that within-participant relationship when estimating a time pattern or comparing timepoints. The registry record does not report a longitudinal analysis method or the participant-level observation data needed to assess such a pattern.

What an estimate and its uncertainty would mean

In general, an estimate summarizes a quantity in the study sample, while a confidence interval describes uncertainty about the corresponding population quantity under the assumptions of the method. A narrower interval generally indicates greater precision, whereas a wider interval indicates greater uncertainty. Confidence intervals do not correct for biased measurement, non-random cohort assignment, or a mismatch between the study population and the population of interest. No confidence interval is reported for this endpoint in the registry record.

For a binary endpoint, uncertainty depends in part on the number of participants and how observations are distributed across the categories. Small groups can yield imprecise estimates even when the observed count seems clear. The registry reports total enrollment of 12, and its safety groups list two participants at risk in each individual-component cohort and six in the combination cohort. Those safety denominators should not be assumed to supply the primary endpoint’s analyzed denominators: the record does not separately state the number contributing to each endpoint timepoint.

Why a control comparison matters

A controlled comparison is designed to distinguish treatment-related differences from differences that can arise for other reasons. Random assignment helps balance measured and unmeasured factors on average, and masking can reduce some forms of observer or participant expectation. This study is listed as non-randomized and open label, with no control treatment reported. Thus, the cohort observations are descriptive and cannot be interpreted as a randomized treatment contrast. The registry record does not report a statistical adjustment strategy that would address cohort differences.

General statistical analyses for binary data may compare proportions, model the probability of an observation, or describe counts with uncertainty intervals. The appropriate approach depends on the question, design, data structure, and assumptions. If repeated observations are analyzed, methods that account for their dependence may be needed. If cohorts are compared, the non-randomized allocation and limited group sizes are important constraints. None of these methods is identified as the analysis used for this trial; no primary analysis is reported in the registry record.

Planning calculations and observed results

Planning sample-size calculations answer a design question: how many participants might be needed under stated assumptions to detect a specified effect with a chosen error rate and power. They are not results from the enrolled participants and are not evidence that a study achieved a particular power after completion. The registry’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation for the planning illustration. It also states that the effect to detect is a stated fraction of the observed effect. Time-to-event illustrations are described as total event counts using the Schoenfeld approach; other illustrations are described as participants per arm.

Those assumptions are generic planning details in the registry record; they do not establish that the actual trial used two randomized arms, equal allocation, or a normal-approximation test. The study itself is listed as non-randomized and single group, with four named cohorts. No sample-size justification for this trial, target effect, or endpoint analysis is reported in the registry record. Because the primary outcome result is also not reported, no study-specific recalculation or planning illustration can be interpreted from the available result information. Any planning calculation would remain an illustration, not observed power.

Multiplicity and analysis choices

Multiplicity arises when an analysis makes several statistical tests or estimates and the chance of a false-positive result is considered across that collection. Multiple cohorts, timepoints, endpoints, or subgroup analyses can raise this issue if formal inferences are made. The registry lists one primary outcome measure, but its observations span multiple timepoints and the study has four cohorts. It does not report a multiplicity adjustment, a prespecified hierarchy of comparisons, or a statistical analysis plan. Therefore, the record does not show how any potential collection of comparisons would have been handled.

Missing observations can also affect interpretation. A participant may not have a recorded observation at every timepoint, and the amount or pattern of missing data can influence a summary. The registry record does not report endpoint-specific denominators, missingness, exclusions, or imputation methods. General methods for missing data rely on assumptions about why data are absent; those assumptions cannot be assessed without the relevant information.

Finally, an analysis population defines which participants are included in a result—for example, all enrolled participants or only those with a particular level of treatment exposure. The registry does not describe an analysis population for the primary endpoint. It is therefore not possible to tell whether any unposted analysis used every enrolled participant, a subset, or a different denominator at particular timepoints.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists one primary outcome measure, with observations planned at arrival, predose, and up to 12 hours post dose. It also identifies four cohorts. Multiple timepoints and cohort summaries do not automatically imply that formal statistical tests were performed, but if several comparisons are tested, multiplicity becomes relevant: repeated testing can increase the chance of at least one apparently notable result arising by chance. The record does not report a testing hierarchy, multiplicity adjustment, or strategy for interpreting multiple comparisons.

No statistical analysis plan is reported in the registry record. It does not state whether the primary endpoint was to be analyzed descriptively or inferentially, how the timepoints were to be combined, or whether cohort comparisons were planned. It reports zero analysis records and no primary endpoint result. Thus, the analytical handling of the endpoint cannot be reconstructed from the registry information.

The record also does not provide endpoint denominators at each collection time, the number of missing observations, reasons for missingness, participant exclusions, or methods for handling unavailable data. These details matter because a count of participants with an observation is interpretable only in relation to the number assessed at that timepoint. The 12 enrolled participants and the safety group counts do not establish endpoint-specific denominators.

An analysis population describes who contributes to a result. The registry record does not state whether primary endpoint analyses were intended to include all enrolled participants, only participants who received a dose, or another defined group. No imputation method or missing-data assumption is reported. Without these details and the underlying results, neither the effect of missing observations nor the consistency of the analysis population across timepoints can be assessed.

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 1% reporting threshold.

Most frequent non-serious adverse event terms

The registry lists serious adverse events, other adverse events, and deaths by cohort. In the LMN-201 Anti-toxin B VHH-1 cohort, zero serious adverse events were reported among two participants at risk, zero other adverse events among two at risk, and zero deaths. The VHH-2 cohort has the same reported counts: zero serious adverse events among two at risk, zero other adverse events among two at risk, and zero deaths. The VHH-3 cohort also reports zero serious adverse events among two at risk, zero other adverse events among two at risk, and zero deaths.

For the cohort receiving LMN-201 VHH 1, 2, and 3, the registry reports zero serious adverse events among six participants at risk, one other adverse event among six at risk, and zero deaths. No serious adverse-event terms are listed. The record does not identify the other adverse event, indicate its severity or timing, or report whether investigators considered it related to treatment.

These are counts from small cohorts, not proof that an event cannot occur. The registry entries describe events recorded in this study and do not estimate risk with precision or establish causality. The absence of a listed serious event should be read as a reported count, not as evidence that all possible safety concerns have been ruled out.

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

The registry record supports a limited description of the study’s purpose and design. It was a Phase 1, open-label, non-randomized, exploratory study of LMN-201 components delivered by enteric capsules in individuals with ostomies. Its primary endpoint was visual presence or absence of capsules and/or transit markers in ostomy fluid, at arrival, predose, and through 12 hours post dose.

The record does not report the primary endpoint counts or any statistical analysis. It therefore does not show how many participants had visible material at any collection time, nor does it quantify uncertainty around such observations. Without those results, the main delivery question cannot be answered from the registry record. The definition of an outcome is not itself an outcome finding.

Even if visible capsule or marker observations were reported, this endpoint would not by itself establish how much of a component reached a particular site, whether the component remained intact or active, or whether it changed a clinical outcome. It is a delivery-related observation, not a measure of infection response or treatment efficacy. The registry lists C. Diff. Infections as a condition, but does not report clinical infection outcomes.

The cohort structure also does not support a causal comparison. Allocation was non-randomized, masking was none, and no control group is reported. Findings across the separate-component and combination cohorts, if available, would need to be interpreted as descriptive rather than as evidence that one regimen caused a different outcome from another. The record does not provide analysis methods or participant-level information that could address potential cohort differences.

The safety entries report zero serious adverse events and zero deaths in all cohorts, with one other adverse event recorded in the combination cohort. These counts offer a limited account of recorded events, but do not establish that risk is absent or identify the event’s cause or clinical significance. Overall, this study’s registry record documents an exploratory design and an intended observation, while leaving its primary results and important analytical details unreported.

13. Why This Trial Matters Statistically

This trial is statistically notable because its central measurement is a direct, binary observation tied to a delivery question. Recording whether capsules or transit markers are visible in ostomy fluid is conceptually different from measuring a clinical response. It can help frame an exploratory question about transit, but its interpretation must remain within the limits of what visual presence or absence captures.

The study also illustrates the distinction between a registered endpoint and a reported result. A clearly named outcome and timeframe can tell readers what investigators intended to measure. They cannot show the observed pattern without counts, denominators, and an account of how the observations were analyzed. Here, the primary outcome definition is present, while its results are not reported in the registry record.

Design features matter to the strength of an inference. Non-random allocation and no masking can complicate comparisons between cohorts, while the absence of a control group means there is no like-for-like benchmark for changes or observations. These issues do not make exploratory observations meaningless; they define what those observations can support. Descriptive evidence can guide questions for later studies, but it is not equivalent to a controlled estimate of comparative effect.

The record also shows why small cohort safety counts require careful wording. Zero recorded serious events in a cohort describes what was reported during the study; it does not demonstrate that the true risk is zero. Event counts without event details or causal assessments provide only a narrow view of safety.

Finally, the record does not report an analysis plan, missing-data handling, or endpoint-specific denominators. Those omissions prevent readers from evaluating precision, analysis choices, and the robustness of any result. For clinical researchers, the key statistical lesson is to separate the question a trial was designed to ask from the results it actually reports and from the broader conclusions its design can support.

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 consequential limitation in interpreting this trial record is that the primary endpoint results are not reported. The outcome is defined, but no timepoint-specific or cohort-specific counts are available. There is no reported effect estimate, uncertainty interval, or inferential analysis with which to assess the observed delivery pattern.

The study was non-randomized and open label, with no control intervention reported. The four named cohorts cannot be treated as randomized comparison groups. The registry does not explain how participants were assigned, whether cohort characteristics differed, or whether any methods were used to account for differences. Visual observation in an unmasked study may also be affected by observer expectations; assessor procedures and verification methods are not reported.

The endpoint is limited to visual presence or absence of capsules and/or transit markers in ostomy fluid. It does not quantify material, verify capsule integrity, establish a precise location of release, or measure a clinical outcome. The record does not report criteria for classifying a visual observation as present, nor does it describe collection and assessment procedures in enough detail to assess measurement consistency.

Enrollment was 12, while the safety groups list two participants at risk in each of three component cohorts and six in the combination cohort. These small cohort counts constrain how much can be learned from subgroup descriptions, particularly for uncommon events. Endpoint-specific denominators are not reported, so the number contributing at each collection time cannot be confirmed from the registry record.

Further limitations include the absence of a reported statistical analysis plan, sample-size justification, interim-analysis plan, missing-data methods, and defined analysis population. The registry record also lacks detail about eligibility criteria, dose regimen, cohort assignment procedures, and clinical outcomes. These omissions restrict reproducibility and prevent a fuller assessment of bias, precision, and generalizability.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Exploratory Study to Assess Delivery of LMN-201 Components Via Enteric Capsules in the Gut of Individuals With Ostomies — NCT04893239.
• Registry record last updated 2025-03-18; status last verified 2025-02.
• Results first posted 2025-03-18.
• 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

This completed Phase 1, single-site, open-label, non-randomized exploratory study enrolled 12 individuals with ostomies. It assessed delivery of LMN-201 components via enteric capsules, with cohorts for Anti-toxin B VHH-1, VHH-2, VHH-3, and the combination of VHH 1, 2, and 3.

The primary outcome was the number of participants with capsules and/or transit markers present in ostomy fluid by visual observation at arrival (baseline), predose, and up to 12 hours post dose. The registry record does not report the primary outcome results or statistical analyses.

Safety entries report zero serious adverse events and zero deaths in each cohort. One other adverse event was reported among six participants at risk in the combination cohort; none were reported among two participants at risk in each individual-component cohort. The event is not identified. The record does not report a control group, randomized comparison, analysis population, missing-data approach, or trial-specific statistical analysis plan.