Study in Healthy Adults Evaluating PF-07202954: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study in Healthy Adults Evaluating PF-07202954 (NCT04857437), a randomized phase 1 study. The primary outcome is Number of Participants With Treatment Emergent Adverse Events (TEAEs): Part 1.

2. Clinical Question

3. Trial Design

This is a phase 1, randomized study of PF-07202954 in healthy adults. The registry describes a planned three-part, sequential design. Part 1 was intended to evaluate single escalating doses of PF-07202954 or placebo, and Part 2 repeated escalating doses from Day 1 to Day 14, inclusive. The registry describes Part 3 as conditional: a randomized, open-label assessment of food effects involving a dose with a high-fat/high-calorie meal and a dose after an overnight fast of at least 10 hours. Whether Part 3 was conducted is not reported in the registry record.

The brief study description says participants and investigators were blinded, with the sponsor unblinded, for the placebo-controlled dose escalation in Parts 1 and 2. At the same time, the registry’s masking classification is listed as none. These descriptions do not align cleanly, so the degree of masking should be read with that qualification rather than treated as unambiguous. Part 3 is described as open-label if conducted.

The registry lists an overall enrollment of 12 and a completed status. It gives a study start of 2021-05-13 and a primary completion of 2021-09-17. The posted Part 1 outcome groups are reported with separate counts by dose and administration condition. Those counts should not be added to reconstruct an overall participant total: the record does not explain how arm-level counts across the study parts relate to the enrollment figure.

The primary outcome concerns treatment-emergent adverse events in Part 1. The record also lists pharmacokinetic secondary outcomes, including maximum observed plasma concentration, time to maximum concentration, area under concentration-time profiles, and terminal half-life. These outcomes address drug exposure and disposition rather than clinical benefit. The registry describes conditions of non-alcoholic fatty liver disease and liver fibrosis, but the study title and design identify the participants as healthy adults; the record does not establish that this trial evaluated treatment effects in people with those conditions.

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.

Only the first 6 of 8 groups are shown.

Part 1 Period 1

Part 1 Period 2

Part 1 Period 3

Part 1 Period 4

Analysis population for the primary outcome, as defined in the registry: Safety population included all participants who were randomly assigned to study treatment and who took at least 1 dose of study treatment. Treatment groups with same dose and administration were combined as planned.

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: Data has been presented for all participants in Cohort 1 and 2 as presenting the data per sequence would risk re-identification of participants due to low enrollment of participants per sequence. The study was concluded following Part 1; hence, no participants were enrolled in Part 2 and 3.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Treatment Emergent Adverse Events (TEAEs): Part 1, measured over From start of study treatment on Day 1 up to 28 days post last dose of study treatment (maximum up to 10 weeks).

The primary endpoint is the number of participants with treatment-emergent adverse events in Part 1. The registry defines an adverse event as an untoward medical occurrence temporally associated with study treatment, whether or not it is considered related to treatment. A treatment-emergent adverse event is one that occurs after treatment begins and through approximately 28 days after the final dose. The stated timeframe runs from the start of treatment on Day 1 to 28 days after the last dose, with a maximum of up to 10 weeks.

This endpoint is used in early-phase studies to describe the observed safety experience during and after exposure. Counting participants with at least one event gives a participant-level summary; it does not count every event separately. Nor does the count convey how serious, persistent, or clinically important events were. Those questions require additional detail, such as event descriptions, severity, timing, and judgments about relatedness, which are not included in the primary outcome values summarized in the registry.

The primary safety population is defined as randomized participants who took at least one dose. The registry says treatment groups with the same dose and administration were combined as planned. The endpoint is reported by dose or administration group, but no formal statistical comparison or confidence interval is posted for these counts. In a small, escalating-dose study, the endpoint is primarily descriptive: it records events observed in the studied groups and observation period, rather than establishing a precise comparative risk estimate.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is binary at the participant level: each participant either did or did not have at least one treatment-emergent adverse event during the specified observation window. The registry presents the number of participants with the event and the number of participants in each group. This is a count outcome in the sense that it counts affected participants; it is not a continuous measure such as a concentration or a time-to-event estimate.

What does a confidence interval add?

General statistical explanation: a confidence interval expresses the uncertainty around an estimate under a specified statistical method. For an event count or event proportion, an interval can show how much uncertainty remains when the number of participants is limited. A narrow interval would indicate greater precision than a wide interval, but an interval is not a guarantee about the true value. No confidence intervals are posted for this endpoint, so the precision of the group estimates cannot be assessed from an interval reported in the registry.

Why are the counts descriptive?

The posted primary-outcome values consist of counts and group sizes, without an inferential comparison. General statistical explanation: a comparison between groups typically requires an analysis method suited to the endpoint and the design, along with an estimate of the difference or another contrast and its uncertainty. The registry record does not report such an analysis for this endpoint. The counts can therefore be described group by group, but they do not by themselves establish whether observed differences exceed what might arise from sampling variability.

The record labels the study randomized, but it is a sequential dose-escalation study with several dose and administration groups. The primary outcome data are not presented as one treatment group against one control group for a single statistical contrast. The registry does not report a model, a test, covariate adjustment, or a prespecified comparison for the primary outcome. Those analysis details are not reported in the registry record and should not be inferred from the study design label alone.

How should zero counts be read?

General statistical explanation: observing no events in a group does not show that the event is impossible or that the underlying event rate is exactly zero. With few observations, even a genuine but uncommon event may not appear in the group studied. The same general caution applies to small nonzero counts: they may be imprecise and are sensitive to the experience of individual participants. This is why event counts are best read alongside group sizes and the study’s limited scope, rather than treated as precise comparative estimates.

What do the planning assumptions mean?

The sample-size planning illustration is described as a calculation based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is expressed as a stated fraction of the observed effect. Such illustrations are hypothetical planning scenarios, not observed results and not evidence that the trial had a particular probability of detecting an effect. The registry record does not report a sample-size justification for the trial’s original design. No planning count should be interpreted as the number enrolled or as observed power.

General statistical explanation: planning calculations depend on assumptions about the target effect, variability or event frequency, allocation, and analysis approach. A planning illustration based on an observed effect can be especially dependent on that observed estimate, which may be unstable in a small study. It does not replace a prospective design justification. The registry’s stated illustration assumptions do not establish what assumptions were used when the trial was designed.

For continuous pharmacokinetic outcomes, methods may summarize concentration measures and their variability or compare exposure under different conditions. The listed outcomes include Cmax, Tmax, AUClast, AUCinf, terminal half-life, and AUCtau. Their result values and analysis details are not reproduced in the primary safety outcome. The registry record does not provide an inferential analysis for the adverse-event counts, and no pharmacokinetic result should be used to fill that gap.

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("Cohort 1 and 2: Placebo: Part 1", "Cohort 1: PF-07202954 10 mg: Part 1", "Cohort 2: PF-07202954 30 mg: Part 1", "Cohort 1 and 2: PF-07202954 100 mg Standard Meal: ", "Cohort 2: PF-07202954 300 mg: Part 1", "Cohort 1: PF-07202954 600 mg: Part 1")
n      <- c(11, 4, 4, 9, 5, 4)
events <- c(3, 0, 1, 2, 1, 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  Cohort 1 and 2: Placebo: Part 1  11  3  27.3  6.0  61.0  9.7  56.6  9.9  57.2
ARM  Cohort 1: PF-07202954 10 mg: Part 1  4  0  0.0  0.0  60.2  0.0  49.0  0.5  52.2
ARM  Cohort 2: PF-07202954 30 mg: Part 1  4  1  25.0  0.6  80.6  4.6  69.9  5.3  71.6
ARM  Cohort 1 and 2: PF-07202954 100 mg Standard Meal:   9  2  22.2  2.8  60.0  6.3  54.7  6.7  55.6
ARM  Cohort 2: PF-07202954 300 mg: Part 1  5  1  20.0  0.5  71.6  3.6  62.4  4.3  64.1
ARM  Cohort 1: PF-07202954 600 mg: Part 1  4  0  0.0  0.0  60.2  0.0  49.0  0.5  52.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists multiple study parts, dose groups, administration conditions, and secondary pharmacokinetic outcomes. When many outcomes or comparisons are examined, general statistical explanation: the chance of at least one apparently unusual result can increase if the analyses are treated as independent discoveries without accounting for the number of questions asked. A prespecified testing strategy or multiplicity adjustment can help control that issue when confirmatory inference is intended. The statistical plan for handling multiplicity is not reported in the registry record.

The record identifies 28 outcome measures and 16 posted secondary outcomes, but it does not report how these outcomes were prioritized, whether they were analyzed together, or whether any adjustment was applied. The primary adverse-event outcome is presented descriptively, with no analysis entries. It would therefore be inappropriate to infer an adjustment strategy, an ordered testing procedure, or a hierarchy among the secondary outcomes.

Missing-data methods are not reported in the registry record. The primary outcome is a count of participants with an event, and the registry defines the safety population as randomized participants who received at least one dose. That definition identifies who is intended to contribute to the safety summary, but it does not describe how incomplete follow-up, unavailable event information, or withdrawal was handled. No assumptions about missing outcomes should be made.

The registry does not report the primary outcome analysis population separately by group beyond the posted group counts, nor does it describe an intention-to-treat analysis for this endpoint. The absence of those details limits assessment of how participants were handled after randomization. The registry also does not describe interim analyses, stopping rules, or an analysis plan for the primary safety outcome. These features are not reported in the registry record and should not be inferred from the sequential design.

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 each listed group, the registry reports zero participants with serious adverse events and zero deaths. The serious-event denominator matches the number at risk shown for that group: 11 for placebo; 4 at 10 mg; 4 at 30 mg; 9 at 100 mg with a standard meal; 5 at 300 mg; 4 at 600 mg; 5 at 100 mg with a high-fat/high-calorie meal; and 2 at 100 mg fasted. No serious adverse-event terms are listed.

These are observed counts in the listed groups, not estimates of the probability of a serious event in a larger population. General statistical explanation: when a study has limited observations, a zero count still leaves uncertainty about events that might occur in other participants or under other conditions. The finding also applies only to the trial groups and observation period represented in the registry.

The serious-event summary is distinct from the primary treatment-emergent adverse-event count. The primary outcome includes events whether or not they were considered related to treatment, and the registry’s safety summary separately reports serious events and deaths. Neither the zero serious-event counts nor the nonzero primary outcome counts establish causation or rule out future events.

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

The results describe treatment-emergent adverse events recorded in the Part 1 groups during the registry’s stated follow-up period. They show that the number of participants with at least one such event was 3 in the placebo group and ranged from 0 to 2 in the listed PF-07202954 groups. The registry also reports no serious adverse events and no deaths in any listed group. These are descriptive observations from the participants represented in the posted results.

The counts do not establish that PF-07202954 caused or prevented an event. The registry definition includes events regardless of whether they were judged related to treatment, and the record does not post a causal analysis or inferential comparison for this endpoint. Differences in observed counts across the groups could reflect chance, differences in group size or other factors; the posted record does not quantify that uncertainty with confidence intervals or a statistical test.

The findings do not establish the safety profile for wider populations, longer treatment, other doses, or different clinical settings. The study is described as a phase 1 investigation in healthy adults, and the endpoint is limited to the stated period after treatment. The record’s conditions list includes non-alcoholic fatty liver disease and liver fibrosis, but that listing does not show that the study evaluated treatment in people with those conditions.

Nor do the primary safety counts answer the study’s pharmacokinetic questions. The registry lists measures of drug concentration and exposure as secondary outcomes, but those measures address a different question from whether participants experienced adverse events. A complete interpretation of the study would require the relevant posted exposure results and additional detail about event types, severity, timing, and relatedness, as well as the analysis methods. Those details are not established by the primary count alone.

14. Why This Trial Matters Statistically

This trial is statistically instructive because it illustrates the limits of descriptive safety counts in a small, multi-group early-phase study. The outcome is readily understood: it records how many participants experienced at least one event after treatment began. But a count is not, by itself, a comparative effect estimate. Without a reported contrast and measure of uncertainty, the observed group values should not be read as proof of differences between doses or between PF-07202954 and placebo.

The design also highlights how dose escalation and distinct administration conditions complicate simple comparisons. The listed Part 1 groups vary in dose, meal condition, and group size. Some group labels involve food conditions even though the study description presents the food-effect assessment as a conditional Part 3. Preserving those labels rather than silently reconciling them is important for a transparent reading of the record.

For biostatisticians and clinical researchers, the record is a reminder that registry entries can offer outcome definitions and observed counts while leaving essential analysis details unreported. Here, no primary-outcome analysis entries, confidence intervals, p-values, or multiplicity strategy are reported. A planning illustration based on an observed effect should also be distinguished from prospective sample-size justification and from observed power.

The study’s value for statistical interpretation lies in identifying what can be stated carefully: the recorded counts, their group denominators, their endpoint definition, and the limits of inference. Keeping those elements separate helps readers avoid turning a small descriptive safety summary into a definitive comparative conclusion.

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 analysis method for the primary adverse-event outcome, a comparison estimate, confidence intervals, or p-values. It also does not report a prospective sample-size justification, interim-analysis procedures, stopping rules, or a multiplicity strategy. Those omissions prevent assessment of the intended inferential framework for the primary endpoint.

The posted primary outcome is limited to Part 1. The record does not establish whether Part 3 was conducted, although the planned design describes it as conditional. It also does not reconcile the Part 1 labels attached to meal-condition groups with the study description of a food-effect assessment in Part 3. The posted group labels should therefore be retained as recorded rather than treated as proof of which planned part was completed.

Group sizes are small and differ across the listed dose and administration conditions. The registry reports an overall enrollment of 12, but the posted group-level counts do not explain how participants across study parts relate to that enrollment total. The available record does not provide enough detail to reconstruct participant flow or determine whether people contributed to more than one group across parts.

Further limitations include the absence of event-level descriptions in the primary outcome summary and the lack of reported methods for incomplete follow-up or missing outcome information. The safety population definition is stated, but the handling of missing data is not. The study is described as involving healthy adults, so the posted safety counts do not directly characterize people with the conditions listed in the registry. These limits should shape any interpretation of the counts and their applicability.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study in Healthy Adults Evaluating PF-07202954 — NCT04857437.
• Registry record last updated 2024-09-20; status last verified 2024-05.
• Results first posted 2024-09-20.
• 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.

19. Record Summary

The registry record describes a completed phase 1 randomized study of PF-07202954 in healthy adults, with a planned sequential design involving single-dose escalation, repeated-dose escalation, and a conditional food-effect part. The primary outcome is the number of Part 1 participants with treatment-emergent adverse events during treatment and follow-up, defined as approximately 28 days after the last dose, up to a maximum of 10 weeks.

Posted Part 1 counts range from 0 to 3 participants across the placebo and PF-07202954 groups. No serious adverse events or deaths are recorded in the listed groups. The outcome is descriptive: the registry does not report an inferential comparison, confidence intervals, or primary-outcome analysis entries. Statistical-plan details, missing-data handling, and multiplicity methods are not reported in the registry record.