Comparing the Availability of AQ280 in the Body After Single Oral Doses of a Capsule Formulation Versus a Tablet for Oral Suspension Formulation: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Comparing the Availability of AQ280 in the Body After Single Oral Doses of a Capsule Formulation Versus a Tablet for Oral Suspension Formulation (NCT07093008), a randomized phase 1 study. The primary outcome is Area Under the Concentration-time Curve From Time 0 Extrapolated to Infinity (AUC0-∞).

2. Clinical Question

The central question is whether the amount of AQ280 reaching the body after a single oral dose differs between the capsule formulation and the tablet for oral suspension formulation. The registry describes a Phase 1, randomized, investigator- and participant-blinded, placebo-controlled crossover study in healthy participants. The primary endpoint is AUC0-∞, a measure of drug exposure over time that includes an estimate of exposure beyond the last measured concentration.

This is a formulation comparison, not a comparison of AQ280 with an active standard treatment. The registry record describes single-dose relative bioavailability in healthy participants; it does not report a clinical efficacy question. The conditions field names Eosinophilic Esophagitis, but the study description identifies the participants as healthy. These two registry entries should be read as distinct pieces of record information rather than as evidence that participants had the condition.

3. Trial Design

The registry identifies NCT07093008 as a completed Phase 1 study sponsored by AQILION AB. It is described as randomized, double-blind, placebo-controlled, and crossover. The stated purpose is to compare the relative bioavailability of two AQ280 formulations after single oral doses in healthy participants. The listed enrollment is 9.

A crossover design gives a participant different treatments in separate treatment periods, so the comparison can use within-participant information rather than relying only on comparisons between separate groups. The active-treatment sequences listed in the record reverse the order of the two formulations: one sequence gives AQ280 capsule first and tablet for oral suspension second, and another gives the tablet first and capsule second. Two additional sequences use the corresponding placebo forms in reversed order. The registry record does not report the allocation ratio among sequences, period duration, washout interval, or the reason placebo sequences were included.

The primary endpoint description specifies single oral doses of 100 mg AQ280 for both formulations. It names the Pharmacokinetic Analysis Set as the analysis population and gives the measurement window as predose and up to 48 hours post dose, with follow-up described as up to the end of study, approximately 7 weeks. The listed primary endpoint is AUC0-∞.

The registry lists 9 participants enrolled, while the arm-level primary summaries give n=6 for AQ280 Capsule and n=6 for AQ280 Tablet for Oral Suspension. In a crossover study, those arm summaries may represent observations under each treatment rather than independent groups. The registry record does not explain the counts or state how many participants completed both active periods. The dates of study start and primary completion are listed in the record, but timing alone does not establish how participants were followed or how any incomplete measurements were handled.

Randomization and blinding are design features intended to reduce bias, but the randomization procedure, blinding implementation, and any assessment of blinding are not reported in the registry record. A detailed protocol, sample-size justification, and prespecified statistical analysis plan are also 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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Pharmacokinetic Analysis Set

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 Area Under the Concentration-time Curve From Time 0 Extrapolated to Infinity (AUC0-∞), measured over Pre dose and up to 48 hours post dose (up to end of study - approximately 7 weeks).

AUC0-∞ means the area under the concentration-time curve from time zero, extrapolated to infinity. It is a pharmacokinetic measure of overall drug exposure: it combines measured concentrations over time and an estimate of the remaining exposure after the final measurement. In this study, concentrations were assessed predose and up to 48 hours post dose. The endpoint is reported in h*ng/mL and is described for single oral doses of 100 mg AQ280 in each formulation.

For a formulation comparison, AUC0-∞ is useful because it reflects total systemic exposure rather than a concentration at just one sampling time. A difference in exposure could arise from differences in how a formulation releases or makes the drug available for absorption. This endpoint does not, by itself, measure symptom change, disease control, or long-term safety. The registry describes the participants as healthy, and identifies the purpose as relative bioavailability.

The primary endpoint is characterized as a geometric mean, with standard deviation listed as the dispersion measure for the arm summaries. The posted comparison is a ratio of GLSMs rather than a difference in arithmetic means. In general, a ratio expresses the relative scale of the two modeled exposure summaries. Its interpretation depends on which formulation is in the numerator and which is in the denominator; the analysis lists AQ280 Capsule and AQ280 Tablet for Oral Suspension in that order. Details of the underlying model and the exact construction of the GLSM estimates are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The principal comparative statistic is a ratio of GLSMs. GLSM refers to a geometric least-squares mean, a model-based summary often used when measurements are analysed on a logarithmic scale. In general, taking a ratio makes the comparison relative: a value of 1 represents equal modeled summaries, values below 1 indicate a smaller numerator summary, and values above 1 indicate a larger numerator summary. For this record, the listed comparison order is AQ280 Capsule and then AQ280 Tablet for Oral Suspension. The posted ratio is 0.976.

The registry record does not name the statistical model, specify whether log-transformed AUC values were used, or describe the terms included in the analysis. It also does not identify how period, sequence, or participant effects were handled. Those details matter in a crossover study because observations from the same participant are correlated, and treatment comparisons should account for the paired structure rather than treat every treatment-period observation as independent. These are general design considerations; the particular model used for this analysis is not reported in the registry record.

What does the confidence interval convey?

The 90% confidence interval for the ratio runs from 0.943 to 1.01. In general, a confidence interval gives a range of values reasonably compatible with the data under the statistical model and its assumptions. It is useful to assess both the location of that range and its precision. Here, the interval includes 1, so it does not isolate a ratio different from equality at the level represented by this interval. The interval is not a guarantee about the range of individual responses, and it does not establish equivalence. Establishing equivalence normally requires a prespecified acceptable range and an analysis designed to test whether the interval lies within that range; such a margin is not reported in the registry record.

The confidence level and interval should not be confused with a p-value. The registry lists no p-value for the primary comparison. A p-value, when reported, addresses how unusual the observed data or more extreme data would be under a specified null model; it does not quantify the size or clinical importance of an effect. In this record, the point estimate and confidence interval are the available comparative results.

Why use a ratio and geometric summaries?

Pharmacokinetic measures such as AUC can have a distribution in which values are more naturally compared multiplicatively than additively. In general, a geometric mean summarizes values on a multiplicative scale, and a ratio can be easier to interpret for relative exposure than an absolute difference. The record identifies the primary parameter as a geometric mean and reports the contrast as a GLSM ratio. The arm summaries list standard deviations, but the registry does not describe how those dispersion summaries were calculated or whether they are on the original or transformed scale. They should therefore be read as the posted descriptive summaries, not as a substitute for the comparative model.

The reported summary for AQ280 Capsule is 5070.0 h*ng/mL with standard deviation 933.0, based on 6 participants; the corresponding tablet-for-oral-suspension summary is 4950.0 h*ng/mL with standard deviation 1040.0, based on 6 participants. No new contrast should be calculated from those values without the underlying paired data and model details. In a crossover trial, the variability of within-person differences is important, and it cannot be recovered from arm-level means and standard deviations alone.

How does crossover structure affect interpretation?

In general, crossover comparisons can be statistically efficient because each participant receives more than one treatment and can serve as their own reference. That advantage relies on suitable design and analysis. Period effects, treatment sequence, carryover, missing periods, and the correlation between measurements may affect estimates. The registry lists treatment sequences in which the order of the two active formulations is reversed, but it does not report a washout interval, a carryover assessment, period-specific results, or the model used to account for these features. It is therefore not possible to assess those details from the registry record.

The total enrollment is 9, whereas each active treatment summary has n=6. Because the study is crossover, those two arm counts may describe the same participants measured under each treatment; they should not be interpreted as a total of 12 distinct participants. The record does not state whether all 6 in each summary are the same people, how many completed both treatment periods, or why the analysis set differs from total enrollment.

What does over-dispersion mean in general?

Over-dispersion occurs when observed variability is greater than a model assumes. In general, ignoring it can make uncertainty estimates too small and confidence intervals too narrow. The posted endpoint uses a continuous exposure measure, and no over-dispersion diagnostic or adjustment is reported in the registry record. There is no basis in the posted information to assert that over-dispersion was present or that a particular correction was used.

The registry record names the Pharmacokinetic Analysis Set, but its membership rules and missing-measurement procedures are not reported. It lists 16 outcome measures and 3 analyses, while no detailed multiplicity procedure is stated. Those omissions limit reconstruction of the full analysis and should temper interpretation of the single posted primary comparison.

10. Analyses Reported by the Sponsor

The registry posts the sponsor's comparative result as a ratio of GLSMs of 0.976, with a 90% confidence interval from 0.943 to 1.01. No p-value or model method is listed for this analysis. The arm summaries are 5070.0 h*ng/mL for AQ280 Capsule and 4950.0 h*ng/mL for AQ280 Tablet for Oral Suspension, but a ratio recalculated from arm summaries would not necessarily reproduce a model-based crossover comparison. The paired participant data and analysis specification would be needed to reconstruct that comparison reliably.

No separate recalculated comparative estimate is reported in the registry record, so there is no recalculated value to set against the sponsor's result. The record does not flag a discrepancy. The posted sponsor analysis therefore remains the available comparative analysis, while its method and assumptions cannot be fully assessed from the registry entries. Its confidence interval includes 1, and the record does not report an equivalence or noninferiority margin. The sponsor's result should be interpreted as an estimate with uncertainty, not as proof that the formulations are identical or that one is superior.

The raw arm summaries are descriptive statistics, not an independent confirmation of the sponsor's modeled estimate. In a crossover design, within-participant pairing can affect the standard error and interval. Neither the pairing information nor the fitted model is included in the registry record.

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 16 outcome measures, 3 analyses, and 7 posted secondary outcomes. The primary outcome is AUC0-∞, while the secondary outcome list includes adverse events, electrocardiograms, vital signs, and physical examinations. Considering multiple outcomes or analyses raises the general issue of multiplicity: when many hypotheses are tested, the chance of at least one apparently notable result can increase. The registry record does not report a multiplicity adjustment, a hierarchy of testing, or which outcomes were treated as confirmatory versus exploratory. The listed count alone does not establish that every measure was tested as a separate hypothesis.

The primary analysis population is identified as the Pharmacokinetic Analysis Set. The inclusion criteria for this set, the handling of participants with incomplete periods, and the approach to missing concentrations or AUC values are not reported in the registry record. The record does not describe whether any imputation was used, whether analyses were restricted to complete crossover pairs, or whether sensitivity analyses were performed. Those details are important because a crossover comparison may depend on paired measurements from the same participant.

The enrollment is 9, and the primary arm summaries each report n=6. The record does not explain how these counts relate, so the available information does not identify the number of participants with complete data in both active treatment periods. The number of analyses and the outcome counts do not fill that gap.

The registry indicates that values were missing or not estimable for a two-arm comparison for another endpoint. It does not identify the endpoint in this note or explain whether the issue reflects unavailable data, an estimability problem, or another cause. No conclusion about that endpoint can be drawn from the note alone.

The protocol, statistical analysis plan, prespecified interim analyses, and sample-size justification are not reported in the registry record. A planning illustration based on an observed effect is a design-planning exercise, not observed power and not evidence that the study was adequately powered. The assumptions described for such planning include a two-sided significance level of 0.05, equal allocation, a normal approximation, and an effect defined as a fraction of the observed effect. The actual trial design justification is not reported.

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 registry reports serious adverse events for three groups. In the Placebo group, 0 serious events were recorded among 3 participants at risk. In the AQ280 Capsule group, 0 were recorded among 6 at risk. In the AQ280 Tablet for Oral Suspension group, 0 were recorded among 6 at risk. No deaths are reported in any of these groups, and no serious adverse-event terms are listed.

These counts describe the posted safety record, not proof that serious adverse events cannot occur. The number of participants at risk is limited, and the record does not provide a time-at-risk comparison or enough detail to characterize uncommon events. The placebo group has a different at-risk count from the active formulation groups, and the study uses crossover sequences; those group counts should not be treated as if they necessarily represent separate, independent sets of participants.

For other adverse events, the registry reports 2 affected participants among 3 at risk for Placebo, 0 among 6 for AQ280 Capsule, and 2 among 6 for AQ280 Tablet for Oral Suspension. The record lists incidence and severity of adverse events as a secondary outcome, but these group counts do not give the event descriptions, severity breakdown, timing, or relation to treatment. The absence of serious events in these counts should be read narrowly as a description of what is posted, not as a general safety conclusion.

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

The posted primary result is a ratio of GLSMs of 0.976 for AQ280 Capsule relative to AQ280 Tablet for Oral Suspension, with a 90% confidence interval from 0.943 to 1.01. The estimate is close to 1, and the interval includes 1. This describes a central exposure comparison whose uncertainty includes equality; it does not demonstrate that the formulations are identical.

The registry record does not report an equivalence margin or a noninferiority margin. Without such a prespecified criterion and a corresponding analysis, a result near 1 cannot be called proof of equivalence or noninferiority. Likewise, the analysis is labelled superiority, but the posted interval includes the equality value and no p-value is reported. The data should be described by their estimate and uncertainty rather than by a categorical claim about superiority.

The endpoint is AUC0-∞ after single oral doses of 100 mg, measured predose and up to 48 hours post dose. It concerns pharmacokinetic exposure in the Pharmacokinetic Analysis Set, not symptom improvement or disease outcomes. The registry describes healthy participants, even though Eosinophilic Esophagitis appears in the condition field. The record does not provide a basis for extending this exposure comparison to clinical benefit in people with that condition.

The primary summaries are based on 6 participants per formulation, while total enrollment is 9. The crossover design means these arm counts may involve the same participants under different treatments; the record does not explain the relation between the counts. That small set of posted observations and the interval's uncertainty limit precision. The registry does not report the exact crossover model, missing-data rules, or a detailed analysis plan, so full independent reconstruction is not possible.

The result is therefore specific: it is a posted relative bioavailability estimate for two AQ280 formulations in a Phase 1 crossover study. It does not establish longer-term safety, efficacy, dose equivalence beyond the tested dose, or performance in a patient population. The secondary safety counts are also limited descriptions and should not be used to infer that adverse events will or will not occur in other settings.

14. Why This Trial Matters Statistically

This study illustrates why pharmacokinetic formulation comparisons are often expressed as ratios rather than as simple differences. A ratio compares relative exposure, while a geometric least-squares mean reflects a model-based summary. The posted estimate of 0.976 is near the equality reference of 1, but the 90% confidence interval from 0.943 to 1.01 is essential for judging uncertainty. A point estimate alone would conceal that the interval includes values on either side of equality.

The crossover design also highlights an important analytical distinction. In general, measurements from the same participant across treatment periods are related. A model that accounts for that pairing can produce a different uncertainty estimate from an analysis that treats treatment groups as independent. The registry gives the sequence structure and arm summaries, but it does not describe the fitted model or covariance handling. That gap makes the posted comparative result more informative than a hand comparison of arm summaries, while also limiting the ability to audit its derivation.

The record's numbers bring attention to the difference between enrollment and analysis counts. It reports 9 enrolled and n=6 for each active formulation summary. Without an explanation of who contributed paired observations, readers cannot determine from these entries alone how complete the crossover data were. Clear reporting of analysis-set rules and missingness would help distinguish the planned design from the observations included in the primary analysis.

Finally, a near-unity estimate is not equivalent to a formal equivalence conclusion. Equivalence requires a stated acceptable range and an analysis evaluated against that range; neither is reported in the registry record. This is a useful example of how the question a study is designed to answer, the estimate it reports, and the conclusion supported by the interval must remain distinct.

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 does not report the statistical model used for the ratio of GLSMs, the analysis terms, or whether and how the crossover pairing was represented. It also does not describe a washout interval, period effects, carryover assessment, or treatment-sequence balance. These omissions prevent a full assessment of the comparative analysis from the record alone.

The record lists 9 participants enrolled and 6 participants in each active formulation summary, but does not explain the difference or state how many participants contributed observations for both formulations. It does not report the analysis-set inclusion criteria, completion counts, missing concentration handling, or sensitivity analyses. The limited number of participants in the posted summaries means that precision and generalizability warrant caution, although the exact impact of any individual observation cannot be assessed without participant-level data.

The primary endpoint window is stated as predose and up to 48 hours post dose, with the timeframe extending up to end of study, approximately 7 weeks. The registry does not describe sampling times within that interval, assay details, or the method for extrapolating AUC0-∞. It also does not report whether the variability summaries are on the original or transformed scale.

The registry identifies the study as Phase 1 and describes healthy participants, while Eosinophilic Esophagitis is listed as a condition. The record does not explain that difference. The primary result concerns pharmacokinetic exposure after single doses, not clinical outcomes. The listed safety counts are limited and do not give event descriptions or a detailed severity account. The protocol, sample-size justification, interim analysis plan, and full statistical analysis plan are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Comparing the Availability of AQ280 in the Body After Single Oral Doses of a Capsule Formulation Versus a Tablet for Oral Suspension Formulation — NCT07093008.
• Registry record last updated 2026-06-01; status last verified 2026-02.
• Results first posted 2026-06-01.
• 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

NCT07093008 is a completed Phase 1 randomized, double-blind, placebo-controlled crossover study sponsored by AQILION AB. It enrolled 9 participants and compared single oral doses of 100 mg AQ280 as a capsule and as a tablet for oral suspension in healthy participants.

The primary endpoint was AUC0-∞, measured predose and up to 48 hours post dose, in the Pharmacokinetic Analysis Set. The posted ratio of GLSMs was 0.976, with a 90% confidence interval from 0.943 to 1.01. Each active formulation summary reports n=6. The registry record does not explain the relation between those analysis counts and total enrollment or provide the full statistical model.

The record lists 7 secondary outcomes, including adverse-event and clinical safety measures. It reports no serious adverse events and no deaths in the listed groups. The available primary comparison is a pharmacokinetic result; the registry does not report an equivalence margin, noninferiority margin, or clinical efficacy result.