Scoring Clinical Index For Onychomycosis in Toenail Onychomycosis Treating With Itraconazole Capsules: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Scoring Clinical Index For Onychomycosis in Toenail Onychomycosis Treating With Itraconazole Capsules (NCT00871728), a na phase 4 study. The primary outcome is Percentage of Participants Showing 10 Percent or Higher Response in Scoring Clinical Index for Onychomycosis (SCIO) Score at Week 5.

2. Clinical Question

The study also sought to examine the relationship between SCIO score change, mycological cure, and clinical improvement. Mycological cure is named as a secondary outcome, but the record does not report its result or a measure of the relationship between outcomes. The primary question is therefore a within-group response question: it concerns how many participants met a specified score-change threshold after receiving itraconazole. Without a comparator, the study cannot isolate how much of any observed change was attributable to treatment rather than other influences, such as the course of the condition, measurement variability, or expectations. Those limitations do not change the endpoint’s descriptive purpose: it records whether a defined level of SCIO improvement was observed between baseline and Week 5.

3. Trial Design

The registry record describes a completed Phase 4, single-group study of itraconazole capsules in toenail onychomycosis. Enrollment was 132. The listed condition is onychomycosis of the toe, and the study summary describes participants with infection confirmed by positive KOH and bacterial identification tests. Janssen Korea, Ltd., Korea is listed as the sponsor. The study started in 2007-01 and had primary completion in 2009-05.

There was no randomized allocation: allocation is listed as not applicable, and the study model is single group. Itraconazole is the only intervention listed. Masking is listed as none, so the record does not describe blinding. No concurrent control group is listed. The results should therefore be read as observations within the treated group, not as a comparison of treatment effects between groups.

The primary outcome was a percentage: participants showing a 10 percent or higher response in SCIO score at Week 5 relative to baseline. The record defines the primary-outcome population as the FAS and excludes participants who violated major selection or exclusion criteria, never took study medication, or did not participate in the Week 5 assessment. The registry record does not describe the timing or details of treatment beyond the endpoint timeframe.

The brief summary says the study aimed to evaluate SCIO score change and its relationship to mycological cure rate and clinical improvement. Percentage of participants showing mycological cure is listed among the secondary outcomes. Seven outcome measures are recorded, but the registry record does not provide results for the primary endpoint or the other outcome measures in the available information. It lists zero analyses. Completion status indicates that the study is recorded as completed; it does not, by itself, supply evidence about the size or precision of any outcome.

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 Full analysis set (FAS) population included all participants except for those participants who violated the major selection and exclusion criteria or did not take the study medication even once or those participants who didn't participate in Week 5 assessment.

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 Percentage of Participants Showing 10 Percent or Higher Response in Scoring Clinical Index for Onychomycosis (SCIO) Score at Week 5, measured over Baseline and Week 5.

The primary endpoint is the percentage of participants whose SCIO score improved by 10 percent or higher at Week 5 compared with baseline. It is a threshold-based binary outcome: each participant either meets the defined response criterion or does not, and the endpoint expresses the responding participants as a percentage of the analysis population. The registry record identifies the measure and timeframe but does not report the observed percentage, response count, or an uncertainty interval.

SCIO is described as an index based on clinical state, including the clinical form of onychomycosis, the depth of the infected area, and subungual hyperkeratosis. Its score ranges from 1 to 30, with higher scores indicating greater severity. The record says the score is classified into seven steps and that treatment can be applied consistently according to the clinical state represented by those steps.

A threshold endpoint can be useful because it gives a clinically interpretable way to classify change: it distinguishes participants who meet a prespecified level of improvement from those who do not. That simplification also discards information about the degree of change within each category. Participants just below and well below the threshold are grouped together, as are participants who just meet and greatly exceed it. The percentage therefore answers a specific response question; it does not fully describe the distribution of score changes or establish that the index change corresponds to mycological cure. The registry record names mycological cure as a secondary outcome but does not report its result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary percentage outcome. Participants are classified according to whether their SCIO score improved by 10 percent or higher at Week 5 relative to baseline. The percentage is intended to represent the share of the analysis population meeting that rule. The registry record does not report the response count or percentage, so there is no result to interpret numerically. It also does not report a confidence interval or another measure of uncertainty for this outcome.

What does a percentage outcome convey?

A percentage is a summary of how often a defined event occurred in a specified population. Here, the event is meeting the SCIO response threshold, and the specified population is the FAS. The measure is straightforward to communicate, but its meaning depends on both the response rule and the population included. A change in the threshold or in the analysis population could alter the percentage, even if the underlying participant scores were unchanged.

Turning a score change into a yes-or-no classification also loses detail. The response percentage does not show the sizes of individual changes, how scores were distributed at baseline, or how many participants improved without reaching the cutoff. Those questions call for fuller reporting of the score data. The record describes the SCIO scale and the threshold but does not provide the corresponding distribution or individual change summaries.

Why does a confidence interval matter?

In general, a confidence interval communicates uncertainty around an estimated quantity. A narrow interval indicates greater precision, while a wide interval indicates less precision. It does not guarantee that the true value lies inside the interval, and it does not resolve limitations caused by study design or selection. No confidence interval for the primary response is reported in the registry record. Without the estimate and its uncertainty, the result’s precision cannot be evaluated.

What can a single-group result establish?

In general, a single-group study can describe outcomes observed among participants who received the intervention. It cannot provide the same treatment contrast as a randomized comparison, because there is no concurrent group to represent what might have happened under an alternative condition. A pre-to-post change can be informative as a description, but it does not isolate the cause of that change. This distinction is particularly important when interpreting an endpoint measured from baseline to a later assessment.

Allocation is listed as not applicable, the model is single group, and masking is listed as none. The record does not describe a comparator or blinding. Accordingly, the primary endpoint is best understood as a within-group response measure, rather than as an estimate of a difference between treatment groups. No causal effect estimate is reported.

How should the analysis population be read?

The FAS definition excludes participants who violated major selection or exclusion criteria, did not take study medication at least once, or did not participate in the Week 5 assessment. A defined population helps clarify whom an endpoint describes. It also means the endpoint need not describe every enrolled participant. The registry record does not state the FAS size, provide exclusion counts, or explain how the listed exclusions were determined in practice.

Missing-data methods matter when participants lack an outcome assessment. In general, different approaches to missing observations can produce different estimates, particularly if the likelihood of a missing assessment is related to participants’ outcomes. The Week 5 assessment requirement is part of the FAS definition, but the record does not report how missing values were handled beyond that population description. Reasons for missing assessments and sensitivity analyses are not reported in the registry record.

What do the planning illustrations mean?

Planning illustrations are not observed study results. The record describes illustrations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. These assumptions describe a planning exercise, not the design of this single-group study and not an estimate of observed power. The registry record does not report its own sample-size justification. Any planning value should therefore be read as an illustration under its stated assumptions, rather than as proof that the study was adequately powered or as a result from participants.

In general, power is a design-stage probability tied to assumptions about an effect, variability, analysis, and sample size. It is not a measure of how likely a study’s conclusion is to be correct after results are observed. Recalculated planning illustrations, when present, also do not replace the sponsor’s own analysis; where a discrepancy is identified, the sponsor’s own analysis takes precedence. The registry record does not report an outcome analysis or an observed-effect estimate from which the primary result could be interpreted.

Finally, multiple outcomes require care. The registry record lists seven outcome measures, but does not report a multiplicity adjustment, a hierarchy among outcomes, or the statistical analysis plan. In general, testing many outcomes can increase the chance of at least one apparently notable result if each is considered independently. Without a reported plan and analysis, the role of any individual secondary finding cannot be assessed. Here, the available record does not provide outcome estimates with which to make that assessment.

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("Itraconazole")
n      <- c(117)
events <- c(30)
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  Itraconazole  117  30  25.6  18.0  34.5  18.6  34.2  18.6  34.3

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists seven outcome measures and identifies the percentage with mycological cure as a secondary outcome. It does not report a multiplicity strategy, such as a prespecified hierarchy or adjustment for considering multiple outcomes. In general, examining several outcomes can make isolated findings harder to interpret because the chance of an apparently notable result may increase when multiple tests are considered. This is a general statistical concern, not evidence that any particular result in this study is false. No outcome analyses are reported in the registry record.

The record defines the FAS as excluding participants who violated major selection or exclusion criteria, never took study medication, or did not participate in the Week 5 assessment. It does not report the number included in the FAS, the number excluded for each reason, or the number with missing outcome assessments. Although the Week 5 assessment is part of the population definition, no additional missing-data method is reported. Reasons for nonparticipation and sensitivity analyses are not reported in the registry record.

The record does not describe the statistical analysis plan, the method for estimating the primary percentage, or how uncertainty was assessed. Sample-size justification and interim analyses are not reported in the registry record. The record’s planning assumptions describe an illustration, not the trial’s own design justification or observed power. With no reported endpoint result and no analysis details, the handling of multiple outcomes and missing information cannot be evaluated beyond the population definition.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: dyspepsia (0.8%).

The registry record reports serious adverse events for the itraconazole group with 130 participants at risk and 1 participant affected. It lists dyspepsia at 0.8% among the top serious terms. The record also lists other adverse events with 130 participants at risk and 35 affected. These are descriptive counts from the safety information; they are not a comparison with an untreated or alternative-treatment group. The registry record does not report deaths.

Counts affected and participants at risk describe the recorded safety population and should not be treated as a complete account of every possible safety outcome. The record does not provide further details about event timing, duration, severity beyond the serious-event classification, relationship to treatment, or event-specific outcomes. The serious-term listing does not establish causation. Because there is no comparison group, these figures cannot show whether the event frequency was higher or lower than it would have been under another condition. The counts characterize what the registry record reports, not a comparative safety effect.

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

The central contribution of the study record is its definition of a specific clinical-response question: whether participants met a 10 percent or higher improvement threshold on SCIO at Week 5 compared with baseline. The record also describes the SCIO domains, identifies the FAS population, and lists mycological cure as a secondary outcome. These details clarify what the study intended to measure and whom the primary outcome was intended to describe.

The registry record does not report the primary outcome result. It therefore does not establish what percentage met the response threshold, how many participants did so, or how precisely the response was estimated. The absence of an outcome value must not be interpreted as a zero response or as evidence for either a favorable or unfavorable result. Secondary outcome results and analyses of the relationship between SCIO change, cure, and clinical improvement are also not reported.

The single-group design further limits what any observed change could mean. Without a concurrent comparator, a baseline-to-Week 5 change would not distinguish an intervention-associated change from other possible influences. The study can describe outcomes among participants in its analysis population, but the design does not furnish a randomized treatment contrast. The lack of masking, as recorded, is also relevant context for interpreting a clinically assessed score, although the record does not provide enough detail to evaluate its influence.

The response threshold makes the endpoint easy to classify but compresses the underlying score information. It does not show the extent of change among responders or nonresponders, and it cannot by itself establish mycological cure. Nor does the record provide a reported association between SCIO response and cure. The endpoint definition and study purpose are informative, but they should not be mistaken for evidence of an observed treatment effect.

Overall, the available information supports a design-level and measurement-level interpretation. It does not support a quantitative claim about response, precision, comparative benefit, or the relationship between clinical score and mycological outcome. Those questions require results and analysis details that are not reported in the registry record.

14. Why This Trial Matters Statistically

This study is statistically notable for asking a clearly thresholded, within-group question about change in a clinical index. SCIO incorporates clinical form, depth of infected area, and subungual hyperkeratosis, and its score is intended to represent severity. Translating that score into a response threshold offers a direct summary of the proportion meeting a specified level of improvement. It also illustrates the trade-off involved in categorizing a continuous or ordered score: the rule is easy to interpret, but less information about the amount and pattern of change remains visible.

The study’s design makes the distinction between description and comparison especially important. It is a single-group study with no allocation to a control arm and no masking listed. A baseline-to-follow-up response can describe change in the analyzed group, but cannot provide the same inference as a randomized contrast. This is a useful example of why the endpoint and the design must be read together: a precise response definition does not, on its own, establish a causal treatment effect.

The FAS definition also highlights the role of analysis-population rules. Participants who did not attend the Week 5 assessment are excluded, so the target population for the primary endpoint is narrower than everyone enrolled. Without reported counts, missing-data methods, or sensitivity analyses, readers cannot assess how that rule affected the estimate.

Finally, the registry record describes a substantial set of outcome measures but reports no analyses or primary result in the available information. This demonstrates why a stated objective, a completed study status, and a defined endpoint are not substitutes for outcome estimates and their uncertainty. Statistical interpretation depends on both the design and the reported results; without the latter, the response question remains unanswered in the registry record.

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 most direct limitation for interpreting the primary question is that the registry record does not report the primary outcome estimate. There is no response count, percentage, or measure of uncertainty to evaluate. The record likewise does not provide results for the listed secondary outcomes, including mycological cure, or report analyses relating SCIO change to cure or clinical improvement.

The single-group design has no comparator, and allocation is listed as not applicable. Any reported pre-to-post change would therefore be descriptive rather than a randomized estimate of the difference attributable to treatment. Masking is listed as none. Because the primary measure reflects clinical state, lack of masking is relevant context, though the record does not provide detail that would quantify its impact.

The primary endpoint uses a threshold, which reduces a score change to whether or not a participant meets the response criterion. It does not convey the full distribution of change or distinguish smaller from larger improvements within either category. The record does not report score distributions or other summaries that would complement the threshold-based endpoint.

The FAS excludes participants who violated major selection or exclusion criteria, did not take medication, or did not participate in the Week 5 assessment. The record does not give the size of this population, exclusion counts, reasons for nonparticipation, or additional missing-data methods. This prevents assessment of how the population definition and missing assessments may affect interpretation.

The statistical analysis plan, multiplicity approach, sample-size justification, and interim analyses are not reported in the registry record. Seven outcome measures are listed, but no analyses are reported. The record also does not give a dose or treatment schedule. These limitations constrain assessment of the study’s analysis and reporting, rather than proving that any particular outcome or method was absent from the conduct of the study.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Scoring Clinical Index For Onychomycosis in Toenail Onychomycosis Treating With Itraconazole Capsules — NCT00871728.
• Registry record last updated 2013-07-02; status last verified 2013-06.
• Results first posted 2013-06-14.
• 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

This is a completed Phase 4, single-group study of itraconazole capsules for toenail onychomycosis. Enrollment was 132; the sponsor is Janssen Korea, Ltd., Korea. The study started in 2007-01 and had primary completion in 2009-05. Allocation is listed as not applicable, and masking as none.

The primary outcome was the percentage of participants with a 10 percent or higher SCIO score improvement at Week 5 compared with baseline. The FAS excluded participants for major selection or exclusion criteria violations, never taking study medication, or not participating in the Week 5 assessment. Mycological cure is listed as a secondary outcome. Seven outcome measures are recorded, and the record lists zero analyses.

The primary outcome result, secondary outcome results, and analysis details are not reported in the registry record. The safety record lists serious adverse events for 130 participants at risk and 1 affected, and other adverse events for 130 at risk and 35 affected. Deaths are not reported in the registry record.