A Study of Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KAN-101 in Celiac Disease (ACeD-it): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study of Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KAN-101 in Celiac Disease (ACeD-it) (NCT05574010), a randomized phase 1, phase 2 study. The primary outcome is Incidence and Severity of TEAEs as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) in Part A.

2. Clinical Question

More broadly, the study was intended to evaluate pharmacodynamic activity, pharmacokinetics, safety, tolerability and plasma biomarker response in participants with celiac disease. Its registered primary endpoint is a safety measure rather than a direct measure of clinical benefit. The record lists pharmacokinetic outcomes for Part A and for Part B and Part C, including measures of plasma exposure. It also lists a total of 15 outcome measures, but outcome-level results for pharmacodynamic activity, biomarkers and pharmacokinetics are not included in the registry record for this statistical interpretation. The clinical question that can be addressed from the posted data is therefore limited: what adverse events were recorded in the listed study groups, and how should those counts be interpreted without a reported primary-outcome analysis? The registry record does not report the reason the study was terminated, the detailed analysis populations or the final interpretation by the sponsor.

3. Trial Design

The registry identifies NCT05574010 as a randomized, parallel-group study with triple masking, covering Phase 1 and Phase 2. Its official title describes a Phase 1B open-label component and a Phase 2 double-blind, placebo-controlled component. The distinction matters: Part A is described as open-label, whereas the later double-blind component included placebo and KAN-101 groups. The registry record does not explain how the masking terminology applies across each part, or which roles were masked.

Part A included two cohorts. Cohort 1 received KAN-101 Dose 1, identified in the adverse-event results as 1.2 mg/kg, and Cohort 2 received Dose 2, identified as 3.0 mg/kg. Each arm description specifies 3 intravenous infusions. Part B and Part C included placebo and KAN-101 groups. The adverse-event results list KAN-101 doses of 0.6 mg/kg, 1.2 mg/kg and 3.0 mg/kg, as well as placebo; the corresponding arm descriptions specify 3 intravenous infusions. The registry record does not describe the allocation process or explain the differing group labels across the intervention and adverse-event listings.

The record lists enrollment as 128. This is a registry enrollment figure and should not be treated as the denominator for any particular posted result. The study began on 2022-11-15, had a primary completion date of 2024-11-29, and is marked terminated. The reason for termination is not reported in the registry record. The registry record does not state whether all planned participants were enrolled or whether the listed completion date reflects all follow-up and analysis activity. The adverse-event counts are reported separately by study part and group, and should be read using their own at-risk denominators rather than the overall enrollment figure.

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: Safety Analysis Set includes all participants who received any portion of study intervention in Parts A, B and C. Participants were analyzed according to the intervention they actually received. In Part B, a total of 11 participants were assigned to the KAN-101 treatment groups, and only 10 received study intervention. In Part C, a total of 80 participants were assigned to the KAN-101 treatment groups, and only 79 received study intervention.

Only the first 6 of 10 groups are shown.

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.

Only the first 6 of 11 groups are shown.

6. Primary Endpoint

The registry lists the primary outcome as Incidence and Severity of TEAEs as Assessed by Common Terminology Criteria for Adverse Events (CTCAE) in Part A, measured over From screening until the safety follow-up visit on Day 28.

The primary endpoint is the incidence and severity of treatment-emergent adverse events (TEAEs) in Part A, assessed using the Common Terminology Criteria for Adverse Events (CTCAE). The endpoint counts participants with events and covers the period from screening until the safety follow-up visit on Day 28. An adverse event is defined as an untoward medical occurrence temporally associated with study intervention, whether or not it is considered related to the intervention. The endpoint includes serious and non-serious adverse events.

Severity is classified using NCI CTCAE version 5. The registry describes Grade 1 as mild, Grade 2 as moderate, Grade 3 as severe, Grade 4 as life-threatening with urgent intervention indicated, and Grade 5 as death related to an adverse event. These grades describe severity, not necessarily whether the event was caused by treatment. Seriousness is a separate classification: the registry definition includes outcomes such as death, life-threatening events, inpatient hospitalization or its prolongation, persistent or significant disability, congenital anomaly or birth defect, and other important medical events.

A safety endpoint of this kind is used to describe the types and frequency of events observed during treatment and follow-up. It is useful for organizing safety observations, but counts alone do not establish causation. The registry record does not provide a single primary-outcome comparison, a severity breakdown for the Part A primary endpoint, or an analysis of the relationship between treatment and events. The posted adverse-event counts therefore give context on reported events, but do not constitute a complete analysis of the registered primary endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation begins by distinguishing the endpoint from the analysis result. The registered primary endpoint is a count of participants with treatment-emergent adverse events, organized by incidence and severity. The record includes adverse-event counts by group, but it does not report a single primary-endpoint analysis or a statistical comparison. Counts can describe what was observed in each listed group; they cannot, on their own, quantify uncertainty or establish a treatment effect.

What does a participant count represent?

A count-of-participants endpoint records whether participants experienced an event, rather than how many total events occurred. A participant with repeated occurrences may still contribute as a participant with an event, depending on the endpoint definition and analysis rules. The registry record gives participant counts affected for serious and other adverse events, but it does not report event-level totals, rules for repeat events, or the statistical handling of participants with more than one event. The at-risk group sizes are the relevant denominators for reading each reported count. The overall enrollment figure is not a substitute for those group-specific denominators.

How do incidence and severity differ?

Incidence describes how many participants experienced an event. Severity describes how intense or serious an event was under the stated classification system. The endpoint title refers to both incidence and severity, while the posted group results available for interpretation distinguish serious adverse events from other adverse events. The CTCAE grades describe severity, whereas seriousness refers to outcomes or characteristics such as hospitalization or life-threatening events. An event can be severe without meeting the regulatory definition of serious, and seriousness does not itself indicate the event's CTCAE grade. The record does not provide a grade-by-grade primary-endpoint result.

Why are descriptive counts limited?

Counts from small groups are sensitive to the experience of each participant. A single additional or fewer participant with an event can change the apparent pattern, particularly when denominators are small. Without confidence intervals or a specified inferential analysis, the reader cannot judge the precision of a group estimate or the uncertainty around a comparison. The record reports no analyses, so no p-value or model-based estimate is available to assess whether observed group differences are distinguishable from random variation. This is especially important when interpreting the Part B groups, whose at-risk counts are small.

Even when a randomized design is used, the existence of randomization does not turn every descriptive difference into a causal estimate. A causal interpretation depends on appropriate analysis, the population included, adherence to the analysis plan and the completeness of follow-up. Those details are not reported in the registry record. The listed adverse-event counts can be described as observations in the study groups, but they should not be treated as a definitive comparison of the safety of KAN-101 and placebo.

What does a confidence interval add?

In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified method. It helps distinguish an estimate that is precise from one that is compatible with a broad range of values. No confidence intervals are reported for these adverse-event counts in the registry record used for this interpretation. Consequently, the precision of the group estimates cannot be quantified from the registry record. A lack of a reported interval does not mean there is no uncertainty; it means that the uncertainty is not numerically characterized in the posted information.

How should the sample-size illustrations be read?

In general, a planning illustration based on an observed effect asks how many participants might be needed to detect an effect of a specified size under stated assumptions. Such an illustration is not an observed power calculation and is not the trial's original sample-size justification. The registry record states that its planning approach uses a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. It also distinguishes participant-per-arm planning for non-time-to-event outcomes from total-event planning for time-to-event outcomes. These are methodological assumptions, not evidence that the study was designed or powered in that way. The trial's own sample-size justification is not reported in the registry record, and no specific planning result is stated in the registry record for this explanation.

Finally, pharmacokinetic measures such as AUCinf, AUClast, Cmax, Tmax and t½ are listed as secondary outcomes. In general, these measures characterize drug exposure or the timing and persistence of plasma concentrations. The registry record lists them but does not report their numerical results or an analysis. The same distinction applies to the planned pharmacodynamic and biomarker objectives: their presence in the study description does not establish what they showed.

10. Multiplicity, Missing Data and Analysis Populations

The study lists 15 outcome measures, including a safety primary endpoint and secondary pharmacokinetic outcomes. Multiple outcomes create a multiplicity issue: when many hypotheses are tested, the chance of at least one apparently unusual result can increase unless the testing strategy is defined and interpreted appropriately. This is a general statistical concern, not a claim that the study conducted a particular set of tests. The registry record does not report a multiplicity adjustment, hierarchy of confirmatory outcomes, or a statistical analysis plan. It also reports 0 analyses, so there are no posted inferential results from which to evaluate the handling of multiple comparisons.

Missing-data methods are not reported in the registry record. The available information does not describe whether any participants were missing endpoint assessments, how incomplete safety follow-up was handled, or whether any imputation or sensitivity analysis was used. Adverse-event monitoring and participant counts do not remove the need to understand follow-up completeness. Without that information, the listed at-risk denominators and affected-participant counts should be read as the reported summaries rather than as proof that all planned observations were available.

Analysis populations are also not reported in the registry record. The record does not specify whether analyses were based on all randomized participants, participants who received treatment, or another defined group. It does not state how participants were assigned to a population after discontinuation or protocol deviations. Randomization is relevant to design, but a reader needs the analysis-population definition to assess how the randomized comparison was preserved. These gaps prevent a full evaluation of multiplicity control, missing-data bias and the relationship between the posted adverse-event counts and the registered primary endpoint.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: syncope (33.3%), bladder perforation (33.3%), pulmonary embolism (33.3%), diarrhoea (25.0%), vomiting (25.0%).

The adverse-event listing separates serious adverse events from other adverse events and gives the number at risk and affected in each group. Read each count with its own group denominator: for example, the Part C 1.2 mg/kg KAN-101 group had 1 of 26 participants with a serious adverse event, while the Part C placebo group had 0 of 28. These are descriptive counts, not a statistical comparison. The record does not report a test or confidence interval for the difference.

In the listed Part A groups, no serious adverse events were recorded, while other adverse events affected 3 of 3 participants in each cohort. In Part B, serious events were recorded in the 0.6 mg/kg, 1.2 mg/kg and 3.0 mg/kg groups, as well as in the placebo group only where indicated by the counts; the posted figures are 1 of 4, 1 of 3, 2 of 3 and 0 of 5, respectively. Part C counts are separately listed for each dose and placebo. No deaths are listed in any group.

Serious-event terms are accompanied by reported percentages: Syncope, Bladder perforation and Pulmonary embolism are each listed as 33.3%; Diarrhoea and Vomiting as 25.0%; and Infusion related hypersensitivity reaction as 3.8%. The registry record does not report the event-level details needed to connect each term to a particular group, participant, timing or treatment attribution. These percentages should therefore be retained as descriptive registry entries, not treated as evidence of a rate comparison or causation.

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

The registry record documents a randomized, parallel-group study of KAN-101 in celiac disease with open-label and double-blind components, and it provides adverse-event counts by listed group. Those counts show that both serious and other adverse events were recorded during the study. The figures can be used to describe the posted observations and to identify that the registry includes particular serious-event terms. They do not provide a complete inferential analysis of safety.

The counts do not establish that KAN-101 caused or prevented any listed event. An adverse event is defined as occurring in temporal association with study intervention whether or not it is considered related. Causation requires careful assessment of timing, alternative explanations, event characteristics and comparative evidence. Those details are not available in the registry record. Likewise, the absence of a serious adverse event in a group does not demonstrate that such events cannot occur; it describes the posted observations for that group.

The posted information also does not show whether an apparent difference between a KAN-101 group and placebo is statistically precise or likely to persist in another study. There are no reported confidence intervals, p-values or primary-outcome analysis, and the registry lists 0 analyses. The registered primary endpoint concerns the incidence and severity of TEAEs in Part A, but the available counts are not presented as a single analysis of that endpoint. A comparison of counts across different study parts should be especially cautious because the parts have distinct designs and group sizes.

Finally, these data do not answer the study's pharmacodynamic, pharmacokinetic or plasma biomarker questions. Those objectives and several pharmacokinetic outcomes are listed, but their results are not reported in the information available for this interpretation. Nor does the registry record explain why the study was terminated. The appropriate conclusion is limited to describing the reported safety counts and the gaps in the posted analysis, without inferring efficacy, overall safety or the reason for termination.

13. Why This Trial Matters Statistically

This trial is statistically notable because it combines early-phase safety and pharmacology objectives with a placebo-controlled component. The registered goals include pharmacodynamic activity, pharmacokinetics, safety, tolerability and plasma biomarker response. These objectives answer different kinds of questions. Safety counts describe participant experiences; pharmacokinetic outcomes characterize plasma exposure; pharmacodynamic and biomarker outcomes concern biological response. A result for one objective would not automatically answer the others.

The Part A endpoint focuses on treatment-emergent adverse events and their severity, while the record also lists placebo and multiple KAN-101 doses in Part B and Part C. This structure makes clear why the study should not be reduced to one simple comparison without regard to study part, masking and endpoint. The adverse-event counts are presented for individual groups, and the Part A endpoint is not accompanied by a single reported analysis. Preserving those distinctions is important for avoiding overinterpretation.

The registry also illustrates the difference between registration and complete reporting. It records a randomized, parallel design and a registered enrollment figure of 128, but lists 0 analyses and does not report a statistical analysis plan, analysis populations, missing-data methods or the reason for termination. Those omissions limit what can be concluded from the posted counts. Statistical rigor depends not only on the trial design but also on transparent reporting of how participants and outcomes were analyzed.

For clinical researchers and biostatisticians, the central lesson is that descriptive safety data are informative but bounded. Group-specific counts preserve useful detail, particularly in a study with different parts and dose groups. At the same time, they are not a substitute for estimates of uncertainty, a prespecified comparison or a clear description of follow-up and analysis populations. The registry record is therefore most useful as a structured account of design and observed adverse-event counts, rather than as a basis for definitive comparative conclusions.

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 key limitation is that the registry record does not report a primary-outcome analysis. Although the primary endpoint is defined as incidence and severity of TEAEs in Part A, the posted information does not provide one reduced comparison or a grade-specific summary for that endpoint. It reports adverse-event counts by group, but does not include confidence intervals, p-values or model-based estimates. The precision of the observations and the statistical evidence for between-group differences therefore cannot be assessed from these results.

Group sizes vary across the listed study parts, and several groups contain only a small number of participants. Such counts are useful as direct descriptions, but estimates based on small groups can be imprecise. The record does not provide event-level data, participant-level follow-up or detail on whether events recurred. It also does not report treatment relatedness for each event or explain how the serious-event terms and percentages map to specific groups.

Other limitations concern analysis transparency. The statistical analysis plan, analysis populations, multiplicity handling and missing-data methods are not reported in the registry record. The record lists 0 analyses. It also does not explain the reason for termination or whether enrollment and follow-up proceeded as planned. The registry enrollment figure of 128 should not be used as the denominator for group-specific results.

Finally, the registry entries available do not include numerical results for the listed pharmacokinetic, pharmacodynamic or plasma biomarker outcomes. The adverse-event counts cannot answer those questions. Together, these limitations mean that the posted information supports a descriptive account of selected safety observations, but not a comprehensive assessment of efficacy, comparative safety or the study's full scientific objectives.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Study of Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KAN-101 in Celiac Disease (ACeD-it) — NCT05574010.
• Registry record last updated 2026-03-10; status last verified 2025-05.
• Results first posted 2026-03-10.
• 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

NCT05574010, titled A Study of Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of KAN-101 in Celiac Disease (ACeD-it), is registered as a randomized, triple-masked, parallel Phase 1 and Phase 2 study. Its official title describes a Phase 1B open-label and Phase 2 double-blind, placebo-controlled design. The registry lists enrollment as 128, a start date of 2022-11-15, a primary completion date of 2024-11-29, and status as terminated.

The registered primary endpoint assesses the incidence and severity of TEAEs in Part A through the safety follow-up visit on Day 28. The posted results include serious and other adverse-event counts for Part A, Part B and Part C groups. The registry lists 15 outcome measures and 0 analyses. It does not report a primary-outcome analysis, the reason for termination, the statistical analysis plan, analysis populations or missing-data methods. The available safety counts are descriptive and do not establish a comparative treatment effect.