Safety and Blood Levels After a Single Injection of UPB-101 in Healthy Japanese and Non-Japanese Non-East Asian Adults: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Safety and Blood Levels After a Single Injection of UPB-101 in Healthy Japanese and Non-Japanese Non-East Asian Adults (NCT05653479), a randomized phase 1 study. The primary outcome is Maximum Observed Concentration (Cmax) of UPB-101.

2. Clinical Question

3. Trial Design

The registry describes a completed, randomized, open-label, parallel-group Phase 1 study in healthy Japanese and non-Japanese non-East Asian adults. Participants were assigned to one of four planned treatment groups, and the groups were run in parallel. Each group is listed with 8 participants. The registry reports enrollment of 32 participants.

Three groups consisted of Japanese adults receiving a single subcutaneous injection of UPB-101 at low, medium, or high dose. The remaining group consisted of NJNEA adults receiving a single subcutaneous injection at the high dose. The amounts corresponding to low, medium, and high dose are not reported in the registry record. The high-dose groups therefore offer a descriptive comparison between the named populations at the same stated dose level; the record does not describe a separate control treatment.

The study was open label, meaning masking was not used according to the registry. The allocation is described as randomized, but the method used to generate or implement the allocation is not reported in the registry record. The study started on 2022-12-05, reached primary completion on 2023-04-07, and is listed as completed. Its stated focus was safety, tolerability, and blood levels after a single dose. The registry does not report further details on follow-up visits, dose escalation procedures, or the conduct of the study beyond these descriptions.

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.

6. Primary Endpoint

The registry lists the primary outcome as Maximum Observed Concentration (Cmax) of UPB-101, measured over Baseline through Day 85.

The primary endpoint is maximum observed concentration, or Cmax, of UPB-101, reported in ug/mL. It represents the highest concentration recorded in the blood measurements used for the pharmacokinetic assessment. The registry gives the timeframe as baseline through Day 85 and states that non-compartmental analysis was used to estimate pharmacokinetic parameters.

Cmax is used to describe the peak concentration observed after administration. In pharmacokinetic work, it helps characterize the concentration profile and can be compared descriptively across dose groups or populations. It is not, by itself, a measure of a health outcome or proof of a treatment effect. Interpretation depends on how and when blood samples were collected, how concentrations were measured, and how the endpoint was derived. Those operational details are not reported in the registry record.

The reported group summaries are means with standard deviations. A mean describes the group’s average Cmax, while the standard deviation describes the spread of individual observations around that mean. Neither quantity directly gives the precision of a comparison between groups. The registry does not report confidence intervals or inferential tests for these group means. Consequently, the endpoint results support descriptive reading of the posted values, but do not establish whether apparent differences between groups are statistically distinguishable.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary endpoint is a continuous measurement, and the registry reports it using the mean and standard deviation for each treatment group. These summaries serve different purposes. The mean describes the group’s central value on the measured concentration scale. The standard deviation describes how dispersed individual measurements are around that mean. Neither summary alone describes the precision of a comparison between groups.

What does Cmax represent?

Cmax is the maximum concentration observed in the blood measurements for a participant during the stated assessment period. The study record identifies baseline through Day 85 as the timeframe. The exact blood-sampling schedule, assay procedures, and handling of measurements are not reported in the registry record. Because Cmax is based on observed measurements, its estimate can depend on when samples are collected relative to the concentration peak. This is a general feature of observed pharmacokinetic endpoints, not a claim about the unreported schedule in this study.

The registry says non-compartmental analysis was used to estimate pharmacokinetic parameters. In general, non-compartmental analysis summarizes concentration-time data without requiring a specified compartment structure for the body. That broad description does not reveal the details of the calculations used for this endpoint. The record does not report the software, analysis conventions, or rules for handling missing or unusual measurements.

How should the mean and standard deviation be read?

The mean is a summary of the values within a group, expressed in the same units as the endpoint. Here, the reported unit is ug/mL. The standard deviation is also expressed in ug/mL and gives a measure of within-group spread. A larger standard deviation indicates greater observed dispersion relative to the group mean, but it does not tell whether a difference between two group means is precise or statistically persuasive.

A confidence interval is a general statistical tool for expressing uncertainty around an estimated quantity. A confidence interval for a mean or a between-group contrast would account for sampling variability under a stated method. The registry does not report confidence intervals for the Cmax results. It also does not report a standard error or a formal test for the group comparisons. As a result, the posted means and standard deviations are descriptive rather than a complete inferential analysis.

What can the group layout support?

The parallel-group design assigns participants to a treatment group and collects results within those groups. The Japanese groups differ in the stated dose level, while the NJNEA group is listed at the high dose. Comparing the high-dose groups descriptively focuses attention on the population labels while holding the stated dose category constant. However, the record does not report an adjusted analysis or a statistical test of a population effect. Nor does it provide a formal dose-response model for the Japanese groups.

Random allocation is reported, but the registry does not describe the allocation sequence, concealment, or stratification. Randomization is generally intended to reduce systematic differences between assigned groups, but the record’s brief design description does not establish how allocation was carried out in practice. The study is open label, and the registry does not report masking of participants or investigators. For an objective laboratory concentration measure, the potential influence of knowledge of assignment may differ from its influence on subjective outcomes; that general consideration does not substitute for study-specific safeguards, which are not reported.

How should planning illustrations be interpreted?

Planning illustrations based on an observed effect are not observed power and are not the trial’s original sample-size justification. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. Such illustrations describe hypothetical future planning under their assumptions. They do not show that the study was designed with those assumptions, and they should not be read as evidence that a result is adequately powered. The trial’s own sample-size justification is not reported in the registry record.

In general, over-dispersion means that observed variation is greater than a statistical model assumes. If a model underestimates variation, its uncertainty estimates may be too small and its inferential results too optimistic. The registry does not report a model-based analysis or an assessment of over-dispersion for this continuous endpoint, so that issue cannot be evaluated from the posted summaries. For these results, the main practical caution is that group means and standard deviations do not provide the full distribution or uncertainty for a contrast.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Low Dose, Japanese", "Medium Dose, Japanese", "High Dose, Japanese", "High Dose, NJNEA")
n   <- c(8, 8, 8, 8)
m   <- c(11.6, 24.4, 35.1, 34.9)
sp  <- c(2.21, 4.77, 12.2, 6.31)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Low Dose, Japanese  8  11.600  2.210  9.752  13.448
CARM  Medium Dose, Japanese  8  24.400  4.770  20.412  28.388
CARM  High Dose, Japanese  8  35.100  12.200  24.901  45.299
CARM  High Dose, NJNEA  8  34.900  6.310  29.625  40.175

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 5 outcome measures and identifies 2 secondary outcomes: the number of participants with treatment-emergent adverse events and serious adverse events, and an immunogenicity assessment. It does not report a multiplicity adjustment, a hierarchy for testing endpoints, or a family-wise analysis plan. The primary endpoint is clearly identified as maximum observed concentration, but the registry does not describe whether or how multiple comparisons among dose groups or populations were considered. Without that information, any set of exploratory comparisons should be interpreted cautiously.

The registry analysis listings report 0 analyses. No inferential statistical plan, prespecified contrast, confidence interval method, or model for the primary endpoint is reported in the registry record. This limits assessment of whether the posted descriptive results correspond to a prespecified analysis or whether additional analyses were planned.

Missing-data handling is not reported in the registry record. The record does not state whether all enrolled participants contributed an endpoint value, how incomplete concentration profiles were handled, or whether any observations were excluded. It also does not define an analysis population for the Cmax results. The group summaries list 8 participants in each arm, but that alone does not describe the completeness of measurements or establish which participants were included in the endpoint calculation.

These reporting gaps matter because missingness and analysis-population choices can affect descriptive summaries as well as formal comparisons. They do not show that data were missing or that an analysis was biased; they mean that the registry does not provide the information needed to assess those possibilities.

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 0 serious adverse events in each treatment group and 0 deaths in each group. Each group is listed with 8 participants at risk for serious adverse events. These are the counts reported in the registry record; they do not establish that serious risks are absent. With a limited group size, an event that was not observed in the study could still occur in a broader population or with different exposure.

The registry also reports other adverse events affecting 4 participants in the low-dose Japanese group, 4 in the medium-dose Japanese group, 7 in the high-dose Japanese group, and 8 in the high-dose NJNEA group. Each group is listed with 8 participants at risk for these events. These counts should be read as group-level safety summaries, not as a comparison adjusted for dose or population. The record does not provide event terms for the other adverse events in the posted summary, nor does it describe severity, duration, relationship assessment, or the rules used to assign events to categories.

Absence of a serious event in the reported groups is not proof of absence of risk. The safety results are limited to the study setting, its follow-up, and the reporting available in the registry. Treatment-emergent adverse event outcomes are listed, but further detail on how they were collected and analyzed is not reported in the registry record.

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

The results describe pharmacokinetic and safety observations after a single subcutaneous injection of UPB-101 in healthy Japanese and NJNEA adults. The Cmax means rise across the listed Japanese dose groups, and the high-dose means in the Japanese and NJNEA groups are numerically close. These patterns describe the posted group summaries; they are not formal demonstrations of a dose-response relationship or equivalence between populations.

The study is a Phase 1 trial focused on safety, tolerability, and blood levels. Cmax is a pharmacokinetic endpoint, not a direct measure of clinical benefit. No clinical efficacy outcome is identified in the primary endpoint information. Therefore, the concentration results cannot establish whether UPB-101 improves health outcomes or how it compares with an alternative treatment.

Likewise, the safety counts should not be interpreted as a guarantee about safety beyond the study. The record reports no serious adverse events or deaths in the listed groups, while also reporting other adverse events. The available summaries do not give event-level descriptions or enough detail to assess the clinical importance of individual events. They also do not establish how the findings would apply beyond the healthy adults and study conditions described.

Important uncertainties remain: inferential comparisons, confidence intervals, dose amounts, blood-sampling details, and missing-data procedures are not reported in the registry record. Without those details, the results are best understood as descriptive evidence about the observations recorded in this study. They can inform questions for further pharmacokinetic or safety evaluation, but they do not settle those questions on their own.

14. Why This Trial Matters Statistically

This study is statistically informative because it separates dose-level summaries in Japanese adults from a high-dose comparison involving NJNEA adults. The group structure makes the intended descriptive contrasts understandable: variation across the Japanese dose groups and comparison of the high-dose groups across populations. Presenting the observed mean and standard deviation for each group gives readers a basic view of central tendency and dispersion.

The results also illustrate why a visually similar pair of means is not the same as a demonstrated equivalence. The high-dose means are 35.1 ug/mL and 34.9 ug/mL, but the registry does not report a confidence interval for their contrast, an equivalence margin, or a formal analysis. A precise conclusion about similarity requires more than comparing the displayed averages. Similarly, an increasing sequence of means across dose labels is a descriptive pattern, not a statistical dose-response analysis.

The limited group sizes make transparent reporting of variability and uncertainty particularly relevant. Standard deviations help show that observations vary within groups, but they do not provide the uncertainty around a between-group comparison. The absence of inferential analyses in the registry record means readers should distinguish the useful descriptive summaries from conclusions that would require additional analysis.

Finally, this study separates pharmacokinetic characterization from clinical outcomes. The primary endpoint describes blood concentration, while the listed safety outcomes address adverse events and immunogenicity. Keeping those endpoint types distinct helps prevent concentration summaries from being mistaken for evidence of clinical benefit. The study’s value is in describing early pharmacokinetic and safety observations, with the limits of its reporting kept explicit.

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 actual amounts corresponding to low, medium, and high dose. This restricts interpretation of the concentration summaries in relation to dose magnitude and prevents assessment of dose proportionality from the listed labels alone. The record also does not provide concentration-time data or the blood-sampling schedule, so readers cannot assess how the observed maximum relates to the timing and density of measurements.

Although the trial is described as randomized, details of sequence generation, allocation concealment, and any stratification are not reported in the registry record. The study is open label, and the record does not describe any masking of laboratory personnel or other safeguards. No inferential analysis, confidence interval, or formal comparison is listed for the primary endpoint. The registry also does not provide individual observations or distribution plots in its reported details, so the influence of unusual values cannot be assessed from the summary statistics alone.

Information about participant characteristics beyond the healthy population labels is not reported in the registry record. The record does not describe eligibility criteria in detail, the analysis population, missingness, protocol deviations, or the handling of incomplete concentration profiles. These omissions limit evaluation of generalizability and the reliability of group summaries.

The safety summary does not include event terms or detailed event characteristics. The record lists immunogenicity as a secondary outcome but provides no posted result detail for that outcome in the registry record described. Overall, these limitations constrain interpretation to the reported group-level concentration and safety summaries after a single dose; they do not establish that unreported procedures or analyses were absent.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Safety and Blood Levels After a Single Injection of UPB-101 in Healthy Japanese and Non-Japanese Non-East Asian Adults — NCT05653479.
• Registry record last updated 2024-09-03; status last verified 2024-04.
• Results first posted 2024-09-03.
• 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

NCT05653479 is a completed randomized, open-label, parallel-group Phase 1 study of a single subcutaneous dose of UPB-101 in healthy Japanese and NJNEA adults. The registry reports enrollment of 32 participants across four treatment groups, with 8 participants in each group. The Japanese groups received low, medium, or high dose; the NJNEA group received high dose.

The primary endpoint was mean maximum observed concentration from baseline through Day 85, with standard deviation as the measure of dispersion. The posted means were 11.6 ug/mL, 24.4 ug/mL, 35.1 ug/mL, and 34.9 ug/mL across the groups in the listed order. The registry also reports no serious adverse events or deaths in the groups, and reports other adverse events affecting 4, 4, 7, and 8 participants, respectively.

Formal analyses, confidence intervals, dose amounts, and key details of endpoint and missing-data handling are not reported in the registry record. The available results therefore support descriptive interpretation rather than definitive claims about differences between groups.