An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris (NCT04148287), a na phase 2 study. The primary outcome is Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC).

2. Clinical Question

3. Trial Design

This completed Phase 2 study evaluated APX001 in patients with candidemia and/or invasive candidiasis caused by Candida auris. The registry describes it as multicenter, open-label, and single arm. Enrollment was 9. All participants were assigned to the APX001 group; there was no randomized allocation or control group. Treatment could be given intravenously or orally for up to 42 days.

The study began on 2019-12-13, and the recorded primary completion date is 2020-11-30. Its status is completed. These registry entries describe the conduct and administrative status of the study, not the clinical outcome. In particular, completion does not establish whether participants met the primary endpoint.

The primary outcome was treatment success at end of study treatment (EOST), assessed by a Data Review Committee (DRC). The endpoint timeframe was any day from Day 1 up to maximum of Day 42. The registry also lists secondary outcomes concerning time to first negative blood culture, mycological outcomes, investigator- and DRC-determined treatment success at later time points, all-cause mortality through Study Day 30, and treatment-emergent adverse events.

The registry lists 7 outcome measures and 0 analyses. The primary outcome’s observed result is not reported in the registry record. The record also does not report a comparator, masking, or a statistical analysis plan. With a single group, the study can describe outcomes among treated participants, but it cannot estimate a randomized treatment contrast.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The modified intent-to-treat (mITT) population included all participants who received at least 1 dose of study drug and had a confirmed diagnosis of candidemia and/or invasive candidiasis caused by Candida auris within 120 hours (for candidemia) or 168 hours (for invasive candidiasis without candidemia) of study qualification (as determined by the DRC).

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: Analysis was performed on all enrolled participants.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants With Treatment Success at End of Study Treatment (EOST) as Determined by Data Review Committee (DRC), measured over EOST: any day from Day 1 up to maximum of Day 42.

The primary endpoint is the percentage of participants with treatment success at EOST, as determined by the DRC. Success required all listed criteria: qualifying negative cultures or, for specified deep-seated infections, other evidence of resolution; being alive at EOST; and no concomitant use of other systemic antifungal therapies through EOST. The culture and clinical criteria differ according to infection site, so success is a composite judgment rather than a single laboratory measurement.

A DRC assessment provides a committee-based determination using the endpoint definition. That is distinct from the investigator assessments listed among the secondary outcomes. The endpoint combines microbiological evidence, survival at the assessment time, and absence of other systemic antifungal treatment. A participant had to satisfy every required component to count as a success under the stated definition.

Reporting a percentage describes the proportion meeting the definition in the analysis population. A 95% confidence interval is identified as the measure of precision. In general, a confidence interval conveys uncertainty around an estimated percentage; it does not by itself establish a treatment effect or resolve limitations from the study design. The observed percentage and interval are not reported in the registry record, so the primary endpoint’s magnitude and precision cannot be assessed from that record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary outcome as a percentage of participants with an event, specifically treatment success at EOST. In general, a percentage summarizes how many participants in an analysis population meet a defined outcome, expressed relative to the relevant population size. The record does not report the observed percentage, so no numerical estimate can be interpreted.

What does the confidence interval add?

The endpoint lists a 95% confidence interval. In general, a confidence interval communicates the uncertainty associated with an estimate under the statistical method used. It is not a guarantee that the true value lies within a particular interval, and it does not substitute for a comparison group. The interval’s endpoints and the method used to calculate it are not reported in the registry record. Thus neither the estimated precision nor the underlying calculation can be checked from the recorded information.

What does the endpoint count as success?

The primary outcome is a composite definition. It requires culture evidence, or specified alternative clinical and radiological evidence for some deep-seated infections; survival at EOST; and no other systemic antifungal therapy through EOST. Composite outcomes can capture several aspects of a clinical course, but their interpretation depends on all components and the rules for applying them. Since component-level results are not reported, it is not possible to determine which criteria participants met or which criterion most often prevented a success classification.

What is the role of the DRC?

The registry names the DRC as the assessor for the primary outcome. A committee assessment can apply a shared endpoint definition across participants. The record does not report the committee’s composition, review procedures, or whether its members were masked to treatment or other information. The study itself was open-label, and the DRC designation alone does not establish that outcome assessment was blinded.

What can a single-group design estimate?

In general, a single-group study can describe outcomes observed among participants receiving the study intervention. It has no concurrent control group against which to estimate a treatment contrast. As a result, differences attributable to natural disease course, participant selection, other care, or measurement cannot be separated from an intervention effect using a randomized comparison. This is a design limitation, not a statement about whether the observed outcomes were favorable or unfavorable.

How should the listed safety counts be read?

The safety record provides counts at risk and affected for serious adverse events and other adverse events, alongside a death count and selected serious-event terms. These counts describe what was recorded in the study group. Without a control group, they do not quantify excess risk attributable to APX001. The event-term percentages are reported, but the record does not give the event-term denominators or explain whether participants could appear under more than one term. These figures therefore should not be treated as mutually exclusive categories.

What analyses are recorded?

The registry lists 0 analyses. It does not report an analysis plan, a method for handling missing endpoint data, adjustment for multiple outcomes, or any adjusted or unadjusted estimates. General statistical practice may address these questions in a protocol or analysis plan, but such details are not reported in the registry record. No post hoc calculation or inference about those methods can replace the sponsor’s own analysis, which is also not reported in the record.

Planning calculations, when available, are distinct from trial findings: they illustrate assumptions for a hypothetical design and are not observed power. No resulting planning figures are reported in the registry record. The recorded enrollment alone does not reveal the study’s sample-size justification or whether enrollment met a prespecified target.

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("APX001 (Fosmanogepix)")
n      <- c(9)
events <- c(8)
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  APX001 (Fosmanogepix)  9  8  88.9  51.8  99.7  56.5  98.0  55.5  97.5

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint and several secondary outcomes, including blood-culture timing, mycological outcomes, treatment success assessed at different times or by different assessors, mortality, and treatment-emergent adverse events. When multiple outcomes are examined, general statistical practice considers whether and how to account for the possibility of chance findings across analyses. The registry record does not report a multiplicity strategy, an ordered testing plan, or a statistical analysis plan. It is therefore not possible to determine whether any adjustment was intended or used.

The record identifies the mITT population for the primary outcome as including participants who received at least 1 dose of study drug and had a confirmed diagnosis of candidemia and/or invasive candidiasis caused by Candida auris. It does not report the number included in that population, the number excluded, or participant-level endpoint availability. The complete handling of missing or unevaluable outcomes is not reported in the registry record.

Missing-data methods matter because the analysis population and the treatment of participants without an assessable endpoint can affect a percentage estimate. The registry does not explain whether missing outcomes were counted as failures, excluded, imputed, or handled another way. It also does not report whether analysis populations differed across the secondary outcomes. These omissions limit reproducibility and make it difficult to assess the scope of any eventual estimate.

The record lists 0 analyses, so it does not provide analysis results or clarify how outcomes were analyzed. No multiplicity correction, missing-data approach, or additional population definition should be inferred from the outcome list alone.

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 2% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: multiple organ dysfunction syndrome (22.2%), cardiac arrest (11.1%), pneumonia (11.1%), hypotension (11.1%).

The safety information lists 2 participants with serious adverse events among 9 at risk, and 9 participants with other adverse events among 9 at risk. It also lists 2 deaths. These are descriptive study-group counts; without a comparator, they cannot show whether event frequency differs from what would have occurred under another treatment or no treatment. The timeframe associated with the safety death count is not reported in these figures, so it should not be assumed to correspond to the separately listed all-cause mortality outcome through Study Day 30.

The most frequent serious-event term listed is multiple organ dysfunction syndrome at 22.2%. Cardiac arrest, pneumonia, and hypotension are each listed at 11.1%. These percentages describe the reported terms; the record does not specify the denominator for each term or whether a participant could have more than one term. They should not be added together or interpreted as mutually exclusive shares.

Adverse events occurring during a study are not necessarily caused by the study intervention. The registry record does not provide event-level timing, causality assessments, duration, or a comparative safety analysis. These counts therefore describe reported safety observations, not a causal estimate of harm.

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

The registry record establishes that APX001 was studied in a completed, open-label, single-group Phase 2 trial involving patients with Candida auris candidemia and/or invasive candidiasis. It defines treatment success at EOST using microbiological or specified clinical evidence, survival, and absence of other systemic antifungal therapy. It also records safety counts and selected serious adverse-event terms.

The record does not report the primary endpoint result. There is no observed treatment-success percentage or 95% confidence interval to interpret. The listed endpoint definition cannot be treated as evidence that participants did or did not meet that definition. Likewise, enrollment and study completion do not reveal the direction or size of an outcome.

The design cannot provide a randomized comparison because there is no comparator group. A single-group outcome, even if reported, would not establish how outcomes would have differed under another treatment. Open-label conduct adds another consideration for interpreting assessments, while the DRC role indicates that the primary endpoint was determined by a committee. Details of that review process are not reported.

The safety counts document events recorded among participants in the APX001 group, but they do not establish causation or comparative risk. The listed deaths should not be conflated with the separately named mortality outcome without a reported timeframe linking them. The record also does not provide component-level primary endpoint results, missing-data methods, or analysis results.

Accordingly, the information recorded supports a description of the study design, endpoint criteria, and selected safety observations. It does not support a conclusion about the magnitude of treatment success, the precision of an efficacy estimate, or a causal treatment effect.

14. Why This Trial Matters Statistically

This study is statistically notable for the gap between a clearly described composite endpoint and the absence of a reported endpoint estimate in the registry record. The definition specifies multiple requirements and a DRC assessment, but without the observed percentage and confidence interval, readers cannot evaluate either the outcome level or its precision.

The single-group design also defines the scope of inference. It can describe outcomes among treated participants, but it cannot supply a randomized contrast. This distinction is important when interpreting results from studies in populations with limited treatment options: descriptive observations and comparative effects answer different statistical questions.

The endpoint’s composite structure creates a further interpretive issue. Success depends on microbiological or specified alternative evidence, survival at EOST, and absence of other systemic antifungal treatment. A single overall percentage, if reported, would not by itself reveal the contribution of each component. Component-level reporting would help explain how participants arrived at the overall classification, but those results are not reported in the registry record.

The safety information likewise requires descriptive interpretation. Counts and event-term percentages can identify reported observations, but the lack of a comparator and missing details about denominators, timing, and overlap prevent a comparative risk assessment. The record’s 0 analyses and lack of a reported statistical plan also leave key questions about missing data and multiplicity unanswered.

These features make transparent reporting especially important: the endpoint result, its uncertainty, analysis population, missing-data handling, and safety definitions are needed to interpret a small single-group study responsibly. The registry record documents the study framework but does not report those central analytical details.

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 primary limitation for interpreting efficacy is that the registry record does not report the primary endpoint result or its 95% confidence interval. It also does not provide component-level outcomes for the composite definition. The result’s magnitude, precision, and clinical pattern therefore cannot be assessed from the recorded information.

The design was open-label and single-group, with allocation listed as not applicable. There is no like-for-like control comparison, so the study cannot estimate a randomized treatment contrast. The record does not state whether the DRC assessment was masked or describe its review procedures.

The enrollment was 9, and the record does not report the number included in the mITT analysis or the endpoint’s missing-data handling. It does not provide a sample-size justification, protocol-based statistical plan, interim-analysis details, or multiplicity strategy. These matters are not reported in the registry record and should not be inferred from the completed status or the outcome list.

Safety interpretation is also limited. The record lists event counts and selected serious-event terms, but does not give event timing, term-specific denominators, overlap rules, or the timeframe for the death count shown with safety information. With no comparator, these figures are descriptive and cannot establish treatment-attributable risk.

Finally, the listed secondary outcomes are not accompanied by analysis results in the registry record. Their definitions, participant availability, and handling of missing observations are not sufficient to support additional statistical conclusions.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: An Open-label Study of APX001 for Treatment of Patients With Candidemia/Invasive Candidiasis Caused by Candida Auris — NCT04148287.
• Registry record last updated 2025-09-16; status last verified 2025-08.
• Results first posted 2023-01-09.
• 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

NCT04148287 is a completed Phase 2, multicenter, open-label, single-group study of APX001 for candidemia and/or invasive candidiasis caused by Candida auris. Enrollment was 9. APX001 could be administered intravenously or orally for up to 42 days.

The primary endpoint was DRC-determined treatment success at EOST, assessed from Day 1 up to maximum of Day 42. The endpoint required specified microbiological or alternative clinical evidence, survival at EOST, and no concomitant systemic antifungal therapy through EOST. The observed primary result and its 95% confidence interval are not reported in the registry record.

The safety record lists serious and other adverse-event counts, 2 deaths, and selected serious-event terms. The study has no comparator, and the record lists 0 analyses. Its results therefore cannot support a randomized comparison, and the safety counts are descriptive rather than estimates of comparative risk.