MW151-101: First-in-human Study of MW151: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for MW151-101: First-in-human Study of MW151 (NCT04120233), a randomized phase 1 study. The primary outcome is Percentage of Participants Experiencing Drug-related Serious Adverse Events..

2. Clinical Question

3. Trial Design

The registry record describes MW151-101 as a Phase 1a, first-in-human study of MW151. Its official title characterizes it as a double-blind, randomized, placebo-controlled, single ascending dose study in healthy volunteers. Allocation is listed as randomized, masking as single, and the model as sequential. The record does not explain how the sequential dose progression was operationalized, or how advancement between dose levels was decided; those details are not reported in the registry record.

The listed intervention arms are placebo and five single-dose levels of MW151: 10 mg, 20mg, 40mg, 80mg, and 160mg. The arm descriptions state that participants receive placebo or the specified dose. The registry lists 10 participants in the placebo arm and 6 participants in each dose arm. Total enrollment is recorded as 40. The record does not report a separate flow of participants through screening, dosing, follow-up, or analysis populations.

The study was completed. Its recorded start date is 2019-10-22 and its primary completion date is 2021-09-16. The study's stated purpose was to obtain safety and pharmacokinetic information on single ascending doses to inform decisions about continued clinical development. The listed pharmacokinetic outcomes include maximum drug concentration, time to maximum drug concentration, overall drug exposure, drug half-life, and elimination rate constant. Numerical results for those outcomes are not reported in the registry record.

This design is suited to describing short-term safety and drug disposition after single doses in the enrolled volunteers. It is not a test of clinical benefit in people with cognitive disorders: the participants are described as healthy volunteers, and treatment outcomes for cognitive disorders are not listed as trial endpoints. The registry record does not report a treatment duration beyond the single dose, nor does it describe follow-up beyond the seven-day timeframe specified for the primary endpoint.

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: A total of 97 subjects were screened and 40 subjects were randomized.

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 7 groups are shown.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants Experiencing Drug-related Serious Adverse Events., measured over Seven days.

The primary endpoint is the percentage of participants experiencing drug-related serious adverse events from the start of study drug administration through seven-day follow-up. It is a binary outcome at the participant level: each participant either meets the endpoint definition during that timeframe or does not. The registry expresses the result as a percentage, rather than as a severity score or a count of event episodes.

Serious adverse events are used in early clinical studies to capture important safety events, while the attribution of an event as drug-related addresses whether it is considered related to the study drug. These are distinct aspects of the endpoint: seriousness concerns the event's classification, while relatedness concerns its assessed connection to treatment. The record does not provide a detailed definition of seriousness or explain how relatedness was assessed, so those criteria are not reported in the registry record.

The short follow-up makes the endpoint specifically about events recorded within the stated period after dosing. It does not measure all possible adverse outcomes over longer periods, and it does not directly measure pharmacokinetics, tolerability in general, or effectiveness for a cognitive disorder. The primary endpoint can describe events observed in the study arms, but a percentage based on the small arm sizes may be imprecise. The registry record does not provide confidence intervals or a formal comparison for this endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The main result is a percentage for a binary participant-level outcome. A binary outcome records whether an event occurred or did not occur for each participant. For the primary endpoint, the event is a drug-related serious adverse event within seven days after study drug administration began. The posted percentages are 0.0% in the placebo group and 0.0% in every MW151 dose group. The arm sizes differ: 10 participants are listed for placebo and 6 participants for each dose arm.

What does a zero observed percentage mean?

It means no qualifying events were recorded among the participants included in that arm's posted result. It does not mean the true probability of an event is known to be zero. General statistical principle: when an event is rare and the group is small, observing no cases can still be compatible with a range of underlying risks. The uncertainty around a zero estimate is especially important in early-phase studies, which are generally informative about events that occur in the observed participants but are limited in their ability to characterize uncommon events.

Why is there no formal arm comparison?

The registry record reports no statistical analyses for the posted outcomes and marks the two-arm binary endpoint comparison as missing or not estimable. With zero primary events in each arm, the record does not provide an inferential comparison, a p-value, or an estimated contrast. A p-value, in general, summarizes how compatible data are with a specified statistical model and null hypothesis; it is not a measure of clinical importance or proof that groups are equivalent. Since no p-value or model-based estimate is reported, none can be interpreted for this outcome.

A confidence interval, in general, gives a range of values compatible with the observed data under a specified method. It communicates estimation uncertainty rather than guaranteeing that a parameter lies within the interval. The registry record does not report confidence intervals for the primary endpoint. Without those intervals or a stated analysis method, readers should not infer a precise upper bound on the underlying event frequency from the posted zero percentages.

How should the other adverse-event counts be read?

The record separately lists counts for other adverse events: 6 of 10 in the placebo arm, 0 of 6 at 10 mg, 4 of 6 at 20mg, 3 of 6 at 40mg, 3 of 6 at 80mg, and 2 of 6 at 160mg. These should be read as descriptive counts in a category separate from the primary endpoint. They are not a substitute for the drug-related serious-event outcome, and the record does not include descriptions in this adverse-event summary that would clarify the type or attribution of those events. Comparisons of such small groups are also vulnerable to substantial random variation.

What can the dose structure tell us?

The study lists sequential single-dose arms ranging from 10 mg to 160 mg, alongside placebo. The arm labels show the dose levels, but the registry record does not report pharmacokinetic measurements or a statistical analysis of trends across dose levels. General statistical principle: a dose-response analysis requires appropriate outcome data and a specified method; a sequence of dose groups alone does not establish a trend. No pattern in pharmacokinetic exposure can be inferred from the arm labels or the primary safety percentages.

The record names five pharmacokinetic outcomes: maximum drug concentration (Cmax), time to maximum drug concentration (Tmax), overall drug exposure (AUC), drug half-life (T1/2), and elimination rate constant (Kel). Numerical results, estimates of variability, and analyses for these outcomes are not reported in the registry record. Their inclusion as planned or listed outcomes does not reveal the observed values.

What do the planning assumptions mean?

The registry describes sample-size calculations as planning illustrations based on the observed effect, not as the design basis for this trial. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the planning illustration uses a stated fraction of the observed effect. Such calculations, in general, explore how many participants per arm might be needed under chosen assumptions. They are not observed power, do not describe the number of participants actually enrolled, and do not establish that the trial had adequate power for safety comparisons. The registry's comparison is marked missing or not estimable, and the record does not report usable planning estimates for this binary endpoint.

For this outcome, the appropriate reading is therefore descriptive: note the observed percentages and arm sizes, recognize the limited precision of small groups, and avoid treating the absence of a reported test as evidence of no difference. The sponsor's own analysis does not appear as a formal endpoint comparison in the registry record.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Placebo", "Dose 1", "Dose 2", "Dose 3", "Dose 4", "Dose 5")
n      <- c(10, 6, 6, 6, 6, 6)
events <- c(0, 0, 0, 0, 0, 0)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Placebo  10  0  0.0  0.0  30.8  0.0  27.8  0.2  28.5
ARM  Dose 1  6  0  0.0  0.0  45.9  0.0  39.0  0.4  41.0
ARM  Dose 2  6  0  0.0  0.0  45.9  0.0  39.0  0.4  41.0
ARM  Dose 3  6  0  0.0  0.0  45.9  0.0  39.0  0.4  41.0
ARM  Dose 4  6  0  0.0  0.0  45.9  0.0  39.0  0.4  41.0
ARM  Dose 5  6  0  0.0  0.0  45.9  0.0  39.0  0.4  41.0

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists a primary safety endpoint and five pharmacokinetic outcomes: Cmax, Tmax, AUC, T1/2, and Kel. It does not report a statistical analysis plan, a hierarchy among the secondary outcomes, or a multiplicity adjustment. The record also lists zero analyses for the posted outcomes. Without a reported plan for testing multiple outcomes, it is not possible to determine how the trial would have controlled the chance of false-positive findings across a set of formal comparisons. No numerical secondary-outcome results or inferential tests are reported in the registry record.

The record does not describe missing-data handling, the number of participants with missing endpoint data, or whether all enrolled participants were included in each analysis. A missing-data method can affect estimates when observations are unavailable; its appropriateness depends on why data are missing and on the analysis being conducted. Those details are not reported in the registry record, so no assumptions about missingness or imputation should be made.

The analysis population is also not described beyond the arm sizes and the posted outcome counts. The registry does not specify whether results use all randomized participants, only participants who received study drug, or another population. It does not report participant flow or exclusions from analysis. Thus, the posted results can be described using the arm denominators recorded, but the exact analysis-population definition is not reported in the registry record.

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

For the primary endpoint, the registry reports 0.0% experiencing drug-related serious adverse events in placebo and 0.0% in each MW151 dose arm. The adverse-event record lists zero serious events among those at risk in each group, with 10 at risk for placebo and 6 at risk in each dose arm. It also records zero deaths in every group. These counts describe events recorded in the trial; they do not establish that serious events cannot occur.

Other adverse events are listed separately, with counts of 6 in placebo, 0 at 10 mg, 4 at 20mg, 3 at 40mg, 3 at 80mg, and 2 at 160mg. The registry summary does not provide event descriptions alongside these counts. They should not be interpreted as serious-event counts or assumed to be drug-related. Small arm sizes make descriptive comparisons uncertain, and the record does not report confidence intervals or a statistical comparison for these safety results.

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

The posted results establish that no drug-related serious adverse events were recorded for the listed participants during the seven-day period used for the primary endpoint. The same observed percentage, 0.0%, appears in placebo and in each MW151 dose group. This is a description of what happened in the enrolled groups over the specified follow-up, not a precise estimate of the frequency of serious events in a larger population.

The result does not show that the risk of a serious adverse event is zero, nor does it rule out uncommon events. Each dose arm contains 6 participants, and the placebo arm contains 10 participants. The registry record provides no confidence intervals or formal comparison for this endpoint. It therefore does not support a conclusion that the arms are equivalent or that risk is unchanged across dose levels.

The record's other adverse-event counts are distinct from the primary endpoint, and their descriptions and attribution are not reported in the posted summary. Those counts alone cannot establish a causal relationship to MW151 or convey the nature or clinical importance of the events. In addition, the registry lists pharmacokinetic outcomes but does not report their numerical results. It is not possible to draw conclusions about measured drug concentrations, exposure, or dose-related pharmacokinetics from the available outcome figures.

This study was conducted in healthy volunteers and used single doses. Its registry description does not report a trial of treatment for cognitive disorders, and the primary endpoint is a short-term safety measure rather than a measure of clinical benefit. The results therefore should not be taken as evidence that MW151 improves cognitive outcomes, or as evidence about safety during repeated or longer-term use. The record's stated purpose was to inform decisions about continued clinical development; the posted results alone do not establish what those decisions were.

14. Why This Trial Matters Statistically

Early-phase, first-in-human studies address a different statistical question from trials designed to estimate clinical benefit. In this study, the central posted outcome is the occurrence of drug-related serious adverse events after a single dose, with follow-up through seven days. Describing such events by dose group can contribute to an initial safety picture, while the small group sizes mean that estimates remain imprecise for uncommon outcomes.

The trial also illustrates why a zero event count needs careful interpretation. The observed primary percentages are 0.0% across placebo and all MW151 dose arms, but this does not make the underlying risks known or identical. Without a reported interval or formal comparison, the evidence is descriptive rather than a demonstration of equivalence. The registry's statement that the binary comparison is missing or not estimable is important: no inferential result should be inferred from the matching observed percentages.

The protocol's listed pharmacokinetic outcomes also highlight the distinction between what a study intends to measure and what the registry reports as results. Cmax, Tmax, AUC, T1/2, and Kel are named, but their numerical findings are not reported in the registry record. Consequently, the available figures do not show how exposure varied among dose groups. This separation between listed endpoints and posted estimates is central to transparent interpretation.

Finally, the record offers limited detail on analysis populations, missing data, multiplicity, and statistical methods. Those omissions constrain what can be concluded from the results, especially when several outcomes are listed. Statistically, the trial matters as a small, sequential single-dose safety and pharmacokinetic study whose posted primary result describes observed events, while leaving uncertainty about less common events and unreported outcome analyses.

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 arm sizes are small: the registry lists 10 participants for placebo and 6 participants for each MW151 dose. This limits the precision of the observed safety percentages, particularly for rare serious events. The absence of a recorded event does not rule out an underlying risk.

The primary endpoint covers drug-related serious adverse events during the seven-day follow-up. The record does not describe longer-term safety monitoring or provide details on how seriousness and drug relatedness were determined. The findings should therefore be read within the stated endpoint definition and follow-up period.

The registry record does not report confidence intervals, a formal comparison, or an analysis method for the primary endpoint. It marks the two-arm binary comparison as missing or not estimable. The record also lacks details about participant flow, analysis populations, missing data, and multiplicity. These gaps limit assessment of the precision and robustness of the posted results.

Although pharmacokinetic outcomes are listed, their numerical results are not reported in the registry record. The available results consequently cannot characterize exposure across the dose levels. The study is described as a single ascending dose study in healthy volunteers, so the record does not establish effects of repeated dosing, long-term use, or treatment in people with cognitive disorders. Its posted outcome is safety-focused and does not measure cognitive benefit.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: MW151-101: First-in-human Study of MW151 — NCT04120233.
• Registry record last updated 2022-12-30; status last verified 2022-12.
• Results first posted 2022-12-30.
• 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

MW151-101 is a completed Phase 1a randomized, placebo-controlled, double-blind, sequential single ascending dose study in healthy volunteers. The registry records enrollment of 40 and lists placebo plus MW151 doses of 10 mg, 20mg, 40mg, 80mg, and 160mg. The primary endpoint is the percentage experiencing drug-related serious adverse events through seven-day follow-up.

The posted primary result is 0.0% in placebo and 0.0% in every dose arm. No deaths are listed. The adverse-event record separately reports other adverse-event counts, but the posted summary does not describe those events. The registry lists five pharmacokinetic outcomes, without numerical results. It reports no formal endpoint analyses and marks the two-arm binary comparison as missing or not estimable. These findings describe the recorded short-term outcomes and do not establish the absence of uncommon risks or clinical benefit.