Pharmacokinetic Study in Healthy Lactating Women Exposed to Ibrexafungerp: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Pharmacokinetic Study in Healthy Lactating Women Exposed to Ibrexafungerp (NCT06954493), a na phase 1 study. The primary outcome is SCY-078 Breast Milk Concentrations..

2. Clinical Question

The clinical question is pharmacokinetic rather than comparative. The primary outcome concerns drug concentrations in collected breast milk at specified post-dose intervals. The record also lists potential infant exposure and treatment-emergent adverse events as secondary outcomes, but it does not report numerical results for those outcomes.

The setting is narrowly defined: healthy lactating participants with an established milk supply, after a single day of dosing. The question does not directly compare ibrexafungerp with another drug, placebo, or no treatment. Nor does the primary outcome, by itself, answer whether an infant received a particular amount of drug or experienced an effect. Those are separate questions, and the posted record does not give numerical findings for potential infant exposure.

The record identifies Vulvovaginal Candidiasis, Candida Infection, and Vaginal Candidiasis as conditions associated with the study. That listing does not change the stated study population: the brief summary describes healthy lactating women, and the trial's principal purpose is measurement of drug concentrations in milk. The record does not report a clinical efficacy question or an outcome measuring symptom improvement.

3. Trial Design

This is a completed Phase 1, open-label, single-group pharmacokinetic study. The registry lists enrollment of 5 and describes a single treatment group. Participants received ibrexafungerp twice daily on one day, with the two oral 300-mg doses given 12 hours apart. There was no randomized allocation and no masking. With no control group, the study is not designed as a like-for-like treatment comparison.

The primary outcome is the median SCY-078 concentration in breast milk, expressed in ng/mL, with dispersion described as the full range. The measurement window begins before dosing and extends to 108 hours after the first dose. Milk collection is specified at pre-dose and across intervals of 0-2 hours, 2-4 hours, 4-8 hours, 8-12 hours, 12-18 hours, 18-24 hours, 24-36 hours, 36-48 hours, 48-72 hours, and 72-108 hours after dosing.

The record gives a start date of 2023-07-12, a primary completion date of 2023-11-01, and a status of completed. It lists 5 outcome measures and no analyses in the registry analysis count. The record does not report a planned sample-size justification, an interim-analysis plan, or a detailed statistical analysis plan.

The primary pharmacokinetic population is defined by receipt of ibrexafungerp and at least one quantifiable pharmacokinetic parameter. That definition is distinct from enrollment: the record does not state how many participants contributed a quantifiable result at each collection interval. It also does not provide a participant flow account or explain whether all planned milk samples were collected. These omissions matter when interpreting a sequence of measurements, because the set of observations may differ across intervals.

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: This is an open-label study.

Overall Study

Analysis population for the primary outcome, as defined in the registry: Pharmokinetic Population - all subjects that received ibrexafungerp (SCY-078) and that had at least one quantifiable Pk parameter.

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 SCY-078 Breast Milk Concentrations., measured over Pre-dose up to 108 hours post first dose.

The primary endpoint concerns SCY-078 concentrations in breast milk, measured in ng/mL from pre-dose through 108 hours after the first dose. Concentrations are scheduled for measurement at pre-dose and at the specified post-dose collection intervals. The registry defines the summary as a median with the full range.

Pharmacokinetic measurement is used to describe how much drug is detected in a biological sample over time after dosing. For this study, breast milk concentration measurements characterize the presence and time course of SCY-078 in milk in the studied population. They are not, on their own, a measure of clinical benefit or harm, and they do not directly establish an infant's actual exposure or response.

As a general statistical point, the median describes the middle of the observed values and is less influenced by an extreme observation than the mean. The full range communicates the lowest and highest observed values, but does not show how observations are distributed between those endpoints. With a small study population, each participant's measurement can have substantial influence on the reported summaries. The actual concentrations and ranges are not reported in the registry record, so the endpoint's observed pattern and precision cannot be evaluated from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a median with a full range describe?

The registry specifies the median and full range for breast-milk concentrations. In general, the median is the central observed value when measurements are ordered. It is useful for describing a typical observation when values may be unevenly distributed or affected by extreme observations. The full range gives the lowest and highest observed measurements. It does not indicate how densely measurements cluster, whether there are gaps, or how much variation occurs among most participants.

These summaries are descriptive. They do not, by themselves, test a hypothesis or quantify the precision of an estimate. To assess uncertainty, an analysis might report a confidence interval or another uncertainty measure, but such a measure is not reported for the primary endpoint in the registry record. The actual medians and ranges are also not reported, so no concentration pattern can be inferred from the endpoint definition.

Why is the collection schedule important?

Pharmacokinetic measurements are interpreted in relation to dose timing. The record specifies samples from pre-dose through 108 hours after the first dose and gives separate post-dose collection intervals. In general, a sequence of concentrations over time can describe when measurements rise or fall in relation to dosing. The schedule alone does not tell whether concentrations rose, fell, or remained similar. Those conclusions require observed values and information about which samples were available at each interval.

The record does not report the number of participants contributing measurements at each interval, the handling of measurements below a quantification threshold, or how incomplete sampling was treated. Such details can affect summaries of concentration data, especially when the population is small. The primary pharmacokinetic population is defined as participants who received ibrexafungerp and had at least one quantifiable pharmacokinetic parameter, but this definition does not describe completeness at every time point.

What does a single-group design allow?

In general, a single-group study can describe measurements observed after an intervention in the studied group. It lacks a concurrent control group, so it cannot separate changes associated with treatment from changes that might occur for other reasons. This is why the design should not be read as a randomized comparison. The record lists allocation as not applicable, masking as none, and a single-group model. No treatment contrast or analysis estimate is reported.

Open-label means participants and study personnel were not masked to treatment assignment. In a study centered on laboratory concentration measurements, the relevance of masking depends on how samples are collected, processed, and analyzed. The registry record does not report procedures for laboratory blinding or assay validation. Those details should not be assumed.

How should sample-size illustrations be understood?

General sample-size calculations are planning tools: they explore how many participants per arm might be needed under specified assumptions to detect an effect. The stated illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect defined as a fraction of the observed effect. These are planning assumptions, not the design of this single-group study, and any resulting sample-size values are not observed outcomes or evidence of achieved power.

The record does not report a sample-size justification for this trial. It also does not report the observed effect values or the numerical outputs of planning illustrations in the registry record. A planning calculation based on an observed effect should not be confused with prospective justification: it cannot show that the original study was designed around those assumptions. Power is not a property that can be established after seeing a result simply by labeling a calculation as observed power.

What is not available for inferential interpretation?

The registry analysis count is 0. This means the record does not provide a registry-listed analysis result to interpret; it does not establish that no analysis was performed outside the record. No statistical test, model, adjustment method, or uncertainty interval for the primary endpoint is reported. General statistical methods cannot fill in those missing details. The appropriate interpretation is therefore descriptive of the registered objective and design, with outcome magnitude and precision left undetermined by the record.

10. Multiplicity, Missing Data and Analysis Populations

The record lists 5 outcome measures, including the primary breast-milk concentration outcome and secondary outcomes concerning potential infant exposure and treatment-emergent adverse events. It does not report a multiplicity strategy, such as how testing across outcomes or time points would be controlled. This matters in general because examining multiple outcomes or time points can increase the chance of at least one apparently notable finding when many statistical tests are performed. No numerical significance tests are reported in the registry record, so no multiplicity-adjusted or unadjusted finding can be assessed.

The record does not describe missing-data methods, sample-level exclusions, or how measurements that could not be quantified were handled. The stated pharmacokinetic population requires at least one quantifiable parameter, but it does not explain how participants with incomplete measurements were included at particular time points. The record also does not report the number of missing samples or the reasons for any missingness.

Analysis populations are only partly described. The primary pharmacokinetic population is defined in the record, but the number included in that population is not stated separately from the enrollment figure. The record does not give a participant flow account or indicate whether additional populations were used for secondary outcomes or safety summaries.

These gaps limit reproducibility and interpretation. In general, missing measurements can affect estimates if their absence is related to concentration or participant characteristics. Without details on availability and handling, the direction or size of any such effect cannot be assessed. The registry record does not report an analysis plan that resolves these questions.

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 0% reporting threshold.

Most frequent non-serious adverse event terms

The posted adverse-event figures list 0 serious adverse events among 5 participants at risk, one participant with another adverse event among 5 at risk, and 0 deaths. The record does not list serious adverse-event terms. These are descriptive counts from the single treatment group; there is no comparator group from which to estimate a relative difference.

The distinction between serious and other adverse events is important. The serious-event count is not the same as the count for all adverse events, and the record's separate count indicates an event in the other-adverse-event category. The record lists treatment-emergent adverse events as a secondary outcome, but it does not provide a detailed event narrative or numerical results by event type in the information available.

With a small study population and no control group, these counts cannot establish that an event was caused by treatment or quantify comparative risk. A count of zero serious events in this study is a description of the observed record, not proof that serious events cannot occur. The adverse-event figures should be read as limited safety observations from this study, not as a comprehensive estimate of safety.

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

This trial is designed to characterize ibrexafungerp concentrations in breast milk after two oral doses on a single day in healthy lactating women. It records an intended measurement schedule extending from pre-dose through 108 hours after the first dose, with the median and full range specified as summaries. These design features clarify the question the study set out to address.

The registry record does not report numerical concentration results. As a result, it does not show the observed median or range at any collection interval, nor does it provide an estimate of uncertainty around those summaries. The record also does not report numerical results for potential infant exposure. The study's objectives should not be mistaken for findings.

The single-group, open-label design does not provide a randomized comparison with another treatment or with no treatment. The posted adverse-event counts describe observations in the treatment group but cannot establish causation or a comparative safety difference. The record does not report a clinical efficacy outcome, so it does not answer whether treatment changes symptoms or disease outcomes.

Breast-milk concentration measurements and infant exposure are related questions, but they are not interchangeable endpoints. Concentrations in milk alone do not establish the amount an infant consumed or any effect on an infant. The registry lists infant exposure as a secondary outcome, but its numerical result is not reported in the registry record.

In short, the record is informative about the study population, dosing, measurement window, and planned summaries. It is not sufficient to determine the magnitude, variability, or time course of observed milk concentrations, or to draw conclusions about infant outcomes or comparative safety.

13. Why This Trial Matters Statistically

Pharmacokinetic studies in lactating populations address a measurement question that cannot be answered by looking only at a drug's dose or at clinical outcomes in a different population. Measuring concentrations in breast milk over time can describe the presence of a drug in milk after dosing. The study's specified sampling intervals make the timing of that question explicit.

Statistically, this is a setting where the structure and completeness of repeated measurements matter. Concentrations are collected at multiple intervals, while the primary summary is a median and full range. Knowing the number of observations contributing to each interval, how unquantifiable values were handled, and how uncertainty was characterized would help readers judge how stable the summaries are. Those details are not reported in the registry record.

The study also illustrates why design and endpoint should be kept distinct. A single-group pharmacokinetic study can describe observed concentrations, but it does not provide a randomized treatment contrast. Likewise, measurements in milk do not automatically answer questions about infant exposure or clinical effects. The registry lists these as separate outcomes, and numerical results are not reported for them.

The record therefore matters statistically as an example of a narrowly focused, small, descriptive pharmacokinetic study whose intended measurements are clear but whose outcome values are absent from the registry record. Interpretation must distinguish what was planned from what was observed and reported. That distinction is essential whenever the available record contains substantial design information but limited outcome data.

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 principal limitation for interpreting results is that numerical primary-outcome values are not reported in the registry record. The stated median and full-range summaries cannot be evaluated without their observed values. The record also does not report confidence intervals or another measure of precision for the breast-milk concentrations.

The study enrolled 5 participants, with no control group. This supports description of the study group but does not allow a randomized comparison. The small study population also means individual observations can materially affect descriptive summaries; the record does not provide participant-level concentrations to examine that variability.

Although the primary population definition requires at least one quantifiable pharmacokinetic parameter, the record does not state how many participants contributed data at each scheduled interval. It does not report missing-sample counts, reasons for missingness, or handling of measurements that could not be quantified. These omissions make it difficult to assess completeness across the measurement window.

The record does not report detailed assay methods, laboratory quality procedures, a full statistical analysis plan, or a multiplicity approach. It also gives no numerical result for potential infant exposure, despite listing that outcome, and no detailed account of adverse events beyond the posted counts. The absence of these details limits reproducibility and the scope of conclusions; it does not establish that the procedures or analyses were not conducted.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Pharmacokinetic Study in Healthy Lactating Women Exposed to Ibrexafungerp — NCT06954493.
• Registry record last updated 2025-08-08; status last verified 2025-04.
• Results first posted 2025-08-08.
• 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

NCT06954493 is a completed Phase 1, open-label, single-group pharmacokinetic study sponsored by Scynexis, Inc. It enrolled 5 healthy lactating women and evaluated breast-milk SCY-078 concentrations after two oral ibrexafungerp doses on a single day, given 12 hours apart. The primary measurement window ran from pre-dose through 108 hours after the first dose, with median and full range specified as summaries.

The registry lists potential infant exposure and treatment-emergent adverse events as secondary outcomes. It reports 0 serious adverse events, one participant with another adverse event, and 0 deaths. Numerical primary concentration results and numerical infant-exposure results are not reported in the registry record. The single-group design does not provide a randomized comparator.