Multi OIT to Test Immune Markers After Minimum Maintenance Dose: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Multi OIT to Test Immune Markers After Minimum Maintenance Dose (NCT03181009), a randomized phase 2 study. The primary outcome is Change in Allergen-specific Serum IgG4 and IgE.

2. Clinical Question

The practical comparison in the posted arm descriptions is between the two maintenance-dose groups. Both descriptions include initial omalizumab therapy, so the group comparison is not a comparison of omalizumab against no omalizumab. The record also describes interest in safety and in whether a lower maintenance dose could be used, particularly in younger participants, but it does not provide an age-specific result or a separate registered outcome for that question.

The primary outcome is framed as change from baseline to Week 18, the end of study. However, the posted arm summaries give median total serum IgE values and full ranges without baseline values, changes, or a statistical comparison. They therefore do not answer whether the groups differed in change in allergen-specific IgG4 and IgE. The central registered question is identifiable, but the posted results do not establish an estimated treatment contrast for that question.

3. Trial Design

This is a Phase 2, randomized, double-masked, parallel study with reported enrollment of 60. The registry record lists the start date as 2017-07-31, primary completion as 2018-12-01, and status as completed. Andrew Long, PharmD is listed as the sponsor.

The two arm descriptions begin with initial therapy using omalizumab. Group A then escalated food flour allergens to a 300 mg maintenance dose over 18 weeks. Group B escalated food flour allergens to a 1200 maintenance dose over 18 weeks. The record reports 30.0 participants in each arm for the posted primary outcome summaries. It does not describe the randomization procedure, the allocation sequence, or the practical implementation of masking. Those details are not reported in the registry record.

A parallel design assigns participants to a group and compares outcomes between groups rather than having each participant receive both dose regimens in sequence. Randomization is intended, as a general design principle, to reduce systematic differences between groups at the point of assignment. The registry record does not give enough detail to assess how randomization was conducted or whether it achieved balance on particular participant characteristics.

The posted outcome is described as change in allergen-specific serum IgG4 and IgE from baseline to Week 18. Its stated unit, however, is total serum IgE (kU/L), and the outcome summaries report medians and full ranges. This mismatch leaves uncertainty about how the reported values relate to the stated change outcome. The record does not give the planned statistical analysis or a sample-size justification. A protocol, detailed analysis plan, eligibility criteria, and treatment adherence information are 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: Sixty-five participants were initially screened for eligibility with 60 ultimately randomized 1:1 to the 300 mg and 1200 mg arms at two sites. Two participants withdrew early and 11 did not reach maintenance, but continued in the study throughout duration.

6. Primary Endpoint

The registry lists the primary outcome as Change in Allergen-specific Serum IgG4 and IgE, measured over 18 weeks.

The primary endpoint is titled “Change in Allergen-specific Serum IgG4 and IgE.” Its description specifies change from baseline to Week 18, identified as the end of study. These are immune-marker measurements, which can be used in research to characterize biological responses during treatment. As a general statistical concept, measuring a marker at baseline and again after treatment can describe how the observed marker changes over time; comparing changes between randomized groups can address whether the groups followed different patterns.

The registry record does not post baseline measurements, participant-level changes, a group difference in change, or an analysis of either marker’s change. Instead, it gives total serum IgE (kU/L) as the unit and reports a median with a full range for each arm: 578.5, range 64.6 to 6538.0, for Group A; and 738.5, range 61.0 to 4367.0, for Group B. These summaries cannot be treated as changes from baseline because the record does not identify them as change estimates or provide the baseline values needed to interpret change.

The difference between the endpoint title and description on one hand, and the unit and posted summaries on the other, limits interpretation. It is also not clear from the record whether the posted total serum IgE summaries represent the intended allergen-specific measures. This ambiguity matters because the meaning of an endpoint depends on what was measured, at what time, and whether the number describes a level or a change.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Randomization, masking, and a parallel-group structure describe aspects of how a trial is organized; they do not by themselves specify how an outcome was analyzed. The registry record identifies the study as randomized, double-masked, and parallel. In general, random assignment aims to make groups comparable on average at the start of a study, including on factors that may not have been measured. Masking is intended to limit the influence of knowledge about group assignment on participant experience, care, or assessment. The record does not describe the randomization method, who was masked, or how masking was maintained, so those details cannot be assessed.

What does the reported median describe?

A median is a measure of the center of a distribution: it is the point that divides ordered observations into lower and upper portions. It is less affected by very large or very small observations than an arithmetic mean. The record reports medians of 578.5 for Group A and 738.5 for Group B, each with a full range. A range gives the minimum and maximum reported values. It does not reveal the density of observations across that span, the shape of the distribution, or how many observations lie near the median.

These statistics describe each group separately. A treatment comparison ordinarily requires an estimate of the contrast between groups, together with an assessment of its uncertainty. A difference in group summaries is not automatically an estimate of the primary endpoint, especially when the endpoint is defined as change from baseline and the posted summaries are not identified as changes. No inferential comparison is posted for the medians or for the stated change outcome.

Why does the endpoint definition matter?

An endpoint’s name, measurement, time point, and summary must align for its analysis to be interpretable. Here, the title names allergen-specific serum IgG4 and IgE change, and the description sets the comparison from baseline to Week 18. The unit instead names total serum IgE (kU/L), while the results give medians and full ranges by group. The record does not explain whether these summaries are Week 18 levels, another summary, or the intended change measure. This is not merely a choice of statistical method: without clarity about the measured quantity, readers cannot determine exactly which clinical research question the numbers address.

For a change outcome, an analysis might compare within-participant changes between groups, or model follow-up measurements while accounting for baseline. These are general examples of approaches, not claims about the analysis used in this study. The registry record does not report the chosen approach, how baseline values were incorporated, or whether any adjustment variables were planned. The posted data do not permit reconstruction of the change analysis.

What does uncertainty add?

An estimate describes the result in the observed study; uncertainty describes how much the estimate may vary under the statistical model and sampling process. Confidence intervals are one common way to express uncertainty around an estimate. A narrow interval indicates greater precision under the model, while a wide interval indicates less precision. A hypothesis test addresses compatibility with a specified null hypothesis under its assumptions. Neither a confidence interval nor a test for the primary outcome is reported in the registry record, so the precision and statistical compatibility of any group contrast cannot be evaluated.

As a general principle, interpreting a result requires attention to both the size of an estimate and its uncertainty. A statistical test alone would not indicate whether an observed difference is important in a research or clinical context. Conversely, a difference between two descriptive medians does not establish a reliable group effect. The record does not specify a clinically meaningful threshold for the immune-marker endpoint.

How should the planning illustrations be read?

Sample-size planning is distinct from observed trial results. A planning illustration based on an observed effect uses assumptions to show how many participants per arm might be needed to detect a stated fraction of that effect under the selected statistical framework. The record describes such illustrations as using a two-sided significance level of 0.05, equal allocation, and a normal approximation; the effect to detect is defined as a stated fraction of the observed effect. Such figures are planning illustrations, not evidence that the study had a particular probability of detecting an effect, and not a substitute for a prespecified design justification.

The registry record does not report the trial’s own sample-size justification or statistical plan. It also lists no analysis for the outcome and identifies values as missing or not estimable for a two-arm comparison. Therefore, the planning assumptions cannot be used to infer what the investigators planned or to treat any planning estimate as an observed result. The assumption of equal allocation in a planning calculation is not itself proof of the actual randomization process; the posted summaries do report 30.0 participants in each arm.

Dispersion refers to variation among observations. When outcome data are more variable than a statistical model assumes, standard errors and confidence intervals based on that model can be too small, producing overconfident inference. This general issue is one reason an analysis should fit the distribution and design of the outcome. The record reports full ranges but no model, standard errors, confidence intervals, or distributional diagnostics. No specific conclusion about over-dispersion or model adequacy is possible from the posted summaries alone.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists one outcome measure and zero posted analyses. It does not report secondary outcomes. It does not state whether multiple markers, time points, subgroup analyses, or other comparisons were prespecified, nor whether any multiplicity adjustment was planned. Multiplicity refers, in general, to conducting multiple statistical tests or analyses; when many comparisons are examined, the chance of at least one apparently unusual result can increase. A prespecified analysis plan can clarify which comparisons are primary and how multiple testing is handled. That plan is not reported in the registry record, so no adjustment strategy can be described.

The primary outcome description names allergen-specific serum IgG4 and IgE, but the posted summaries give total serum IgE only. The record does not provide baseline values, participant-level data, changes, or a two-arm analysis. The outcome population field is blank. It does not report how many participants contributed measurements at each time point, how missing measurements were handled, or whether any analysis population excluded participants after randomization.

Missing-data methods can affect estimates when outcomes are unavailable. As a general concept, an analysis that uses only observed measurements may be biased if the likelihood of a measurement being missing is related to participant characteristics or outcomes. Other approaches require assumptions about the missingness process. The appropriate method depends on the design and reasons for missingness, which are not reported. It is therefore not possible to assess attrition, completeness, or the robustness of the endpoint analysis.

The record does not report an intention-to-treat analysis, a per-protocol analysis, or another defined analysis population. Those labels should not be inferred from the arm counts. Likewise, no interim analyses, stopping rules, or prespecified statistical plan are reported in the registry record.

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 non-serious adverse event terms

For Group A, 30 participants were listed as at risk for serious adverse events and 0 as affected. For Group B, 30 were listed as at risk and 0 as affected. No deaths were reported in either group. The registry record lists no top serious adverse-event terms. These figures describe what was recorded; they do not establish that no adverse experience of any kind occurred.

For other adverse events, the record lists 19 affected among 30 at risk in Group A and 16 affected among 30 at risk in Group B. The record does not provide event names, severity breakdowns, timing, duration, or attribution to treatment for these other events. The affected counts should not be interpreted as counts of distinct event types or as evidence that an event was caused by a study intervention unless the record specifies that relationship.

Adverse-event counts are descriptive. Without a prespecified comparative analysis, uncertainty estimates, and detailed definitions, they do not establish whether safety differed between the maintenance-dose groups. Zero serious events recorded in these groups is also not proof that the risk of a serious event is absent. The study description says safety was an aim, but detailed safety methods and analysis are not reported in the registry record.

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

The posted results establish that the registry record contains arm-level summaries for total serum IgE and adverse-event counts. They do not provide an analyzed estimate of change in allergen-specific serum IgG4 or IgE from baseline to Week 18. No confidence interval, test result, or two-arm contrast is posted for the primary outcome. Accordingly, the results cannot establish the direction, magnitude, or precision of a difference between the 300 mg and 1200 maintenance-dose groups on the registered change outcome.

The medians and ranges are descriptive summaries. They do not show within-participant change, and their relationship to the endpoint description is not explained. The record does not support a conclusion that either maintenance-dose regimen produced a greater immune-marker response, or that the lower dose achieved a response comparable with the higher dose. It also does not report age-specific findings, despite noting younger subjects in the study rationale.

The adverse-event figures indicate zero participants affected by a serious adverse event in each group as recorded, and other adverse events affecting 19 participants in Group A and 16 in Group B. With no event detail or comparative analysis, these counts do not establish a safety advantage for either group. They also cannot be used to conclude that serious adverse-event risk was absent.

More generally, a completed randomized study can inform a research question only to the extent that outcomes and analyses are reported in a form that addresses it. The design description identifies the intended dose comparison, but the posted primary result does not quantify that comparison. The record therefore supports a description of study structure and reported summaries, not a treatment-effect conclusion or a dose recommendation.

13. Why This Trial Matters Statistically

This trial is statistically interesting because its design and registered question point toward a comparison of two maintenance-dose strategies, while its posted primary outcome summaries do not quantify the comparison the endpoint describes. Separating a study’s design, outcome definition, descriptive results, and inferential analysis is essential when interpreting a registry record. A randomized design can support a fair group comparison, but that advantage depends on reporting an appropriate outcome analysis and enough detail to interpret it.

The distinction between a level and a change is especially important in this record. The primary outcome is described as change from baseline to Week 18, yet the reported values are medians for total serum IgE with full ranges. Without baseline values or a stated change estimate, those values answer a different or at least unclear descriptive question. The record does not resolve that ambiguity.

The study also illustrates why a result needs an uncertainty estimate, not only a central summary. Group medians and ranges describe observations, but they do not express the precision of a between-group treatment contrast. Without an analysis and its uncertainty, readers cannot determine how compatible the posted results are with materially different group effects.

Finally, registry completeness is part of statistical interpretability. The record does not include an analysis plan, sample-size justification, missing-data approach, analysis population, or multiplicity strategy. Their absence does not prove that these methods were not used; it means they are not reported in the registry record. This limits independent assessment of whether the completed analysis followed a prespecified plan and whether its results address the stated research question.

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 important limitation is the gap between the registered primary outcome and the posted summaries. The endpoint is described as change in allergen-specific serum IgG4 and IgE from baseline to Week 18, while the unit identifies total serum IgE (kU/L) and the results provide arm medians and full ranges. No explanation reconciles these descriptions. The posted results do not include separate IgG4 results, baseline values, or changes.

No two-arm analysis is posted, and the record indicates that values are missing or not estimable for a two-arm comparison. There are no reported confidence intervals or hypothesis-test results for the primary outcome. As a result, neither the magnitude nor the precision of a group contrast can be evaluated. The primary outcome population is also left blank, and the record does not explain whether all randomized participants contributed outcome data.

Important design and analysis details are not reported in the registry record, including eligibility criteria, randomization implementation, masking details, adherence, the statistical plan, sample-size justification, handling of missing outcomes, and analysis populations. The record does not report an age-specific analysis, despite mentioning younger subjects as a focus in the study rationale. Safety results lack event descriptions and detailed definitions.

These limitations concern the interpretability of the registry record. They do not establish that relevant procedures or analyses did not occur; they indicate that they cannot be evaluated from what is reported. The results should therefore be read as limited descriptive reporting rather than as a complete analysis of the study’s primary question.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Multi OIT to Test Immune Markers After Minimum Maintenance Dose — NCT03181009.
• Registry record last updated 2026-04-27; status last verified 2020-04.
• Results first posted 2019-09-20.
• 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

NCT03181009 is a completed Phase 2 randomized, double-masked, parallel study of multiple-food oral immunotherapy with initial omalizumab therapy. Reported enrollment was 60. Group A escalated food flour allergens to a 300 mg maintenance dose over 18 weeks; Group B escalated to a 1200 maintenance dose over 18 weeks. The registered primary outcome concerns change in allergen-specific serum IgG4 and IgE from baseline to Week 18.

The posted primary summaries are medians and full ranges for total serum IgE, without baseline measurements, changes, or a two-arm analysis. Serious adverse events affected 0 participants in either group; other adverse events were recorded for 19 in Group A and 16 in Group B. No statistical analysis is posted for the primary outcome, and key analysis details are not reported in the registry record.