Single Dose ADME Study of [14C]-Rencofilstat in Healthy Male Subjects: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Single Dose ADME Study of [14C]-Rencofilstat in Healthy Male Subjects (NCT05737433), a na early/phase 1 study. The primary outcome is To Determine the Mass Balance Recovery After a Single Oral Dose of [14C]-Rencofilstat.

2. Clinical Question

3. Trial Design

This was an open-label, single-center, single-period, single-dose, non-randomized, single-group study. It is classified as EARLY PHASE1 and is marked completed in the registry record. The listed enrollment is 6 healthy male subjects. The study summary says that 6 subjects were planned for enrollment in one group.

Participants were to receive one oral dose of 225 mg of [14C]-rencofilstat. The radiolabel makes it possible to track drug-related radioactivity for the mass-balance objective. The official title also describes assessment of the metabolite profile and metabolite identification. The registry record states these objectives, but does not report metabolite findings.

There was no randomized allocation, masking, or comparison arm. Consequently, the design does not support a direct between-group treatment comparison. Its focus is descriptive characterization after dosing, rather than estimating a relative effect against a control group. The absence of masking is consistent with the registry’s open-label designation; further details about procedures for collecting or assessing samples are not reported in the registry record.

The registry gives a start date of 2023-03-01 and a primary completion date of 2023-05-01. The primary outcome timeframe is 22 Days. Although the study is marked completed, the primary outcome result itself is not reported in the registry record. The record also gives no explanation of the sample-size rationale or additional detail about the conduct of the study.

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: After subjects consented for study, they went through the screening process. Subjects that qualified for study were assigned to the single treatment arm.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Mass balance population

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: Baseline population contains 6 six subjects. The same subjects are in the pharmacokinetic population and safety population.

6. Primary Endpoint

The registry lists the primary outcome as To Determine the Mass Balance Recovery After a Single Oral Dose of [14C]-Rencofilstat, measured over 22 Days.

The primary endpoint is the cumulative percentage of total radioactivity recovered after a single oral dose of [14C]-rencofilstat, assessed over 22 Days. The registry identifies the unit as percentage of total radioactivity, the outcome parameter as NUMBER, and the population as the mass balance population. No observed value, measure of variability, or interval estimate is reported in the registry record.

In general, a mass-balance endpoint describes how much drug-related material is accounted for over a defined observation period. A radiolabel allows radioactivity associated with the administered compound and its derived material to be tracked. The recovery measure is used to characterize disposition and to inform an understanding of how completely the administered radioactivity has been accounted for. It does not, by itself, identify every chemical form or establish the clinical effect of a drug.

The endpoint’s interpretation depends on the observation period, the material collected and analyzed, and the completeness of those procedures. The registry identifies the timeframe but does not report collection procedures, the recovery result, or details about handling incomplete observations. Therefore, the endpoint’s intended meaning can be described, but its observed magnitude and precision cannot be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the study design estimate?

This is a single-group study, not a controlled comparison. In general, a single-group design describes measurements made after participants receive an intervention. It does not isolate the intervention’s effect from other influences as a randomized comparator design aims to do. For this study, the primary question concerns recovery of total radioactivity after a single dose, rather than a difference between treatment groups.

The open-label designation means the study was not masked. The registry also identifies the study as non-randomized, with allocation listed as not applicable. These features are part of the design description; they do not tell the reader how laboratory measurements were performed or whether analysts were blinded to samples. Those procedural details are not reported in the registry record.

What does the endpoint summarize?

The endpoint is a cumulative percentage of total radioactivity recovered over 22 Days. In general, a cumulative measure combines recovery across the observation period into a single endpoint. The percentage scale expresses recovered radioactivity relative to total radioactivity, while the cumulative feature concerns recovery through the stated timeframe. The registry does not report the observed percentage, the underlying measurements, or a distribution of participant-level values.

With no reported endpoint value or variability measure, there is no basis for assessing the observed magnitude or precision. In general, an estimate describes a quantity derived from observed data, while a confidence interval conveys uncertainty around that estimate under the analysis assumptions. Neither an estimate nor a confidence interval for the primary endpoint is reported in the registry record.

How should planning calculations be read?

The registry describes planning illustrations based on an observed effect. Their stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. The registry further distinguishes illustrations expressed as participants per arm from time-to-event illustrations expressed as total events using the Schoenfeld method.

These assumptions do not describe this trial’s actual design: the study has one group and no comparator. Planning illustrations based on a two-arm allocation are hypothetical planning scenarios, not the enrollment plan or an analysis of this single-group study. The primary outcome value is not reported in the registry record, so the basis for interpreting any illustration in relation to an observed effect is not available. Such calculations are not observed power and do not establish the probability that the study detected an effect.

More generally, planning calculations depend on assumptions about the target effect, variability, allocation, statistical model, and error rate. If those assumptions do not match the eventual study design or outcome distribution, the illustration may not describe the precision or operating characteristics of the actual study. The registry record does not report a study-specific sample-size justification or a statistical analysis plan that would clarify this relationship.

Why can dispersion matter?

General statistical interpretation of an outcome depends not only on its average or cumulative estimate but also on how observations vary. When data are more variable than an analysis model assumes, uncertainty can be understated and inferential results can be unreliable. This is a general reason to examine dispersion and model fit where relevant. For this endpoint, the registry record does not report participant-level data, a dispersion measure, or a fitted model, so those features cannot be assessed.

The outcome is identified as a number with a percentage unit, but the registry does not specify a statistical test, confidence-interval method, transformation, or model for the primary result. No analysis is listed in the record. It would therefore be inappropriate to attribute a particular inferential method to the study or to interpret a test statistic that is not reported.

What can be inferred from the safety counts?

Adverse-event counts are descriptive observations. General interpretation of such counts requires attention to the number at risk, event definitions, follow-up, ascertainment, and the distinction between serious and other events. The registry records the at-risk counts and event counts, but does not provide event-level descriptions for the other adverse events or explain collection procedures. With no comparator and no reported inferential analysis, the counts do not establish a comparative safety effect.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists one primary outcome measure: cumulative percentage of total radioactivity recovered after a single oral dose, assessed over 22 Days. It also states study objectives related to metabolite profiling and identification, but does not list outcome results for those objectives. The record does not report whether additional hypotheses or endpoints were tested, or whether any multiplicity adjustment was planned. The number and handling of interim analyses are not reported in the registry record.

Multiplicity is a general concern when several outcomes or statistical tests are considered: repeated opportunities to declare a result can affect the interpretation of inferential evidence. Whether that issue arose in this study cannot be determined from the registry entry. No statistical analysis plan or analysis results are listed.

The registry names the mass balance population for the primary endpoint but does not define its membership or state how many participants were included in the analysis. Enrollment is listed as 6, but that does not establish the analyzed population. The record does not describe missing-data rules, exclusions, protocol deviations, or sensitivity analyses. It is therefore not possible to assess how incomplete observations, if any, were handled, or whether the analysis population differed from enrollment.

In general, missing data can affect an estimate when the probability of an observation being absent is related to the underlying outcome. The direction and size of any such effect depend on the reasons for missingness and the analysis approach. As those details are not reported in the registry record, no conclusion about missing-data impact is supported.

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 1% reporting threshold.

Most frequent non-serious adverse event terms

The registry records 0 serious adverse events among 6 participants at risk in the [14C]-RCF 225mg group. It records 3 other adverse events among 6 participants at risk and 0 deaths. These are counts within the study group, not comparisons against a control group.

The count of other adverse events does not identify the events themselves. The registry record does not report their terms, severity, timing, duration, or relationship to study drug. It also does not describe the methods used to collect or classify adverse events. No serious adverse-event terms are listed.

These figures are descriptive and should be read in the context of the study’s listed enrollment and design. They do not establish that an event was caused by the study drug, nor do they provide a controlled estimate of safety. The registry record’s small single-group enrollment and lack of event-level detail limit what can be concluded from the counts. The absence of recorded serious events in this study is not evidence that serious events cannot occur in other settings.

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

The registry record establishes that this was a completed early-phase, open-label study of a single 225 mg oral dose of [14C]-rencofilstat in healthy male subjects. Its stated primary objective was to determine cumulative mass-balance recovery of total radioactivity over 22 Days. The design and endpoint definition clarify what the study set out to measure.

The record does not report the observed primary endpoint result. It therefore cannot show the amount of radioactivity recovered or the precision of that estimate. It also does not report metabolite profile or identification findings, despite naming these among the study objectives. No conclusion about the study’s ADME findings follows from the stated objectives alone.

Because there was no comparator, the study does not estimate a difference between rencofilstat and another intervention or between dosing and no dosing. The descriptive design can address questions about observations following the dose if the results are reported, but the registry entry does not contain the main outcome result needed for that interpretation.

The listed adverse-event counts describe recorded events in the study group. They do not identify the other adverse events or establish causation, and the absence of recorded serious events in this group does not establish the absence of risk in a broader population.

Thus, the record is informative about the study’s design, planned dose, population description, endpoint, and some safety counts. It is not sufficient to evaluate the magnitude or uncertainty of mass-balance recovery, characterize metabolites, or draw a comparative treatment conclusion.

13. Why This Trial Matters Statistically

Mass-balance studies address a distinct statistical and scientific question: how much drug-related radioactivity is accounted for over a defined period after dosing. A radiolabelled dose makes tracking total radioactivity possible, while metabolite profiling and identification address the chemical forms associated with that radioactivity. These objectives are related but not interchangeable; a recovery percentage alone would not describe the identity of every measured component.

The design also illustrates why the estimand matters. This study is a single-group assessment, so a comparative treatment effect is not its target. Interpreting it as if it were a randomized efficacy trial would misstate the question the study was designed to address. Its stated endpoint concerns recovery after a single dose in healthy male subjects.

Statistical transparency is especially important for descriptive studies. An endpoint definition tells readers what was intended to be measured, but the observed result, variability, and analysis population are needed to assess the finding’s magnitude and precision. The registry record contains the endpoint definition but not its observed value or a reported analysis.

The planning assumptions also illustrate the need to distinguish a design-specific calculation from the actual study design. The listed planning illustration assumes equal allocation between two arms, whereas this study has a single group. Such an illustration should not be mistaken for the trial’s enrollment rationale or evidence of observed power.

For these reasons, the record contributes useful design context but leaves the central statistical result unresolved. Clear reporting of outcome values, analysis populations, and uncertainty would be needed to evaluate the study’s mass-balance findings.

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 main limitation for interpreting this study from the registry record is the absence of the primary outcome result. The endpoint is defined, but its observed value and any measure of uncertainty are not reported. The metabolite objectives are also stated without reported findings, preventing assessment of the intended ADME characterization.

The design is single-group, open-label, and non-randomized, with no comparator. It can describe observations after the dose but cannot support a controlled comparison. The participants are described as healthy male subjects, so the record does not establish how findings would apply to people with NASH or fibrosis, despite NASH with fibrosis being listed as a condition.

The registry lists enrollment of 6 but does not report the number included in the mass-balance analysis, the definition of the analysis population, participant-level results, or missing-data handling. Collection and laboratory procedures are not described in the registry record, so completeness and measurement details cannot be evaluated.

Safety information is limited to counts. Terms for the other adverse events, severity, timing, causality assessments, and ascertainment methods are not reported. The planning assumptions describe a two-arm illustration that does not match the single-group study design; the registry does not report a study-specific sample-size justification or statistical analysis plan.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Single Dose ADME Study of [14C]-Rencofilstat in Healthy Male Subjects — NCT05737433.
• Registry record last updated 2024-10-03; status last verified 2024-06.
• Results first posted 2024-10-03.
• 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.

18. Record Summary

NCT05737433 is a completed EARLY PHASE1, single-center, open-label, non-randomized, single-group study of [14C]-rencofilstat. The registry lists 6 healthy male subjects and describes a single oral dose of 225 mg in a self-micro-emulsifying drug delivery system oral emulsion. The listed start date is 2023-03-01, and the primary completion date is 2023-05-01.

The primary endpoint is cumulative percentage of total radioactivity recovered after dosing, with a timeframe of 22 Days and the mass balance population named for analysis. The registry record does not report the observed endpoint value, an analysis, or metabolite findings. Safety entries record 0 serious adverse events among 6 at risk, 3 other adverse events among 6 at risk, and 0 deaths. No control group is included.