The Study Of Fluconazole For Vulvovaginal Candidiasis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for The Study Of Fluconazole For Vulvovaginal Candidiasis (NCT01806623), a na phase 3 study. The primary outcome is Therapeutic Outcome: Response Rate.

2. Clinical Question

This question combines clinical assessment with evidence about Candida. That is distinct from asking whether symptoms improved alone or whether a laboratory test became negative alone. The registry lists separate secondary outcomes for clinical cure, cure or improvement, mycological eradication, symptom scores, and fluconazole concentration measurements.

The record describes the study purpose as confirming efficacy and safety of a single oral dose in Japanese patients. That stated purpose is not itself an efficacy result. The registry record does not report the response-rate estimate, its confidence interval, or the number of participants classified as effective, ineffective, or indeterminate. It is therefore possible to describe what was intended to be measured, but not to conclude from this record how often the combined response occurred.

3. Trial Design

This was a phase 3, multicenter, open-label, non-comparative study of fluconazole for vulvovaginal candidiasis. The registry lists a parallel model, no masking, and allocation as not applicable. It records enrollment of 157 participants, a start date of 2013-03-05, primary completion on 2013-11-22, and a completed status. The official title identifies the study as multicenter and open-label, while its stated purpose focuses on Japanese patients.

The registry lists one intervention arm, Fluconazole, with no arm description. The study summary describes the regimen as a single oral administration of 150 mg. No control intervention or randomized allocation is listed. As a result, the study design can describe outcomes observed after treatment in the enrolled study population, but it cannot by itself estimate a randomized difference against another treatment or against no treatment.

Open-label means that masking was not used, consistent with the registry entry of none. In general, lack of masking can influence subjective assessments or participant reporting, though it does not establish that such influence occurred in this study. The combined endpoint included clinical efficacy, and the record does not report further details about who assessed it or what procedures were used to reduce assessment bias.

The registry record does not report a formal sample-size justification, a planned statistical analysis protocol, or interim analyses. The sample-size illustrations shown elsewhere on the page are hypothetical planning calculations, not evidence that this trial was designed with those assumptions. In particular, the record identifies a non-comparative study while those illustrations use equal allocation between two arms. They should not be read as the study's actual design or as observed power.

The record reports 14 outcome measures and lists secondary clinical, mycological, symptom-score, and pharmacokinetic outcomes. It reports zero analyses in its analysis listing. That absence means the registry record does not present the analyses; it does not establish what analyses may have been conducted elsewhere.

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 (m-ITT) included participants with vulvovaginal candidiasis who received the investigational products, who tested positive for Candida in the vulva and/or vagina at Day 1 (before dosing), and whose clinical efficacy was evaluated.

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 Therapeutic Outcome: Response Rate, measured over Day 7, Day 14 and Day 28.

The primary endpoint is the therapeutic response rate, expressed as the percentage of participants meeting a combined definition of effectiveness. For each participant, therapeutic outcome was classified as Effective, Ineffective, or Indeterminate using clinical efficacy together with mycological efficacy. Effective required both Cure for clinical efficacy and Eradication for mycological efficacy. A clinical cure without mycological eradication, or eradication without clinical cure, would not satisfy the stated definition of Effective.

The response rate denominator excludes participants assessed as indeterminate. The numerator is the number assessed as effective. The endpoint was specified at Day 7, Day 14, and Day 28, with the primary evaluation on Day 28. The record defines a modified-Intent to Treat population: participants had to receive the investigational product, test positive for Candida in the vulva and/or vagina before dosing on Day 1, and have clinical efficacy evaluated.

Using a combined endpoint can represent success across more than one dimension of disease assessment. It also makes the result dependent on both component assessments and on how indeterminate cases are handled. The endpoint does not equal a symptom-only response, a laboratory-only response, or a comparison with another treatment. The registry record does not report the observed response rate or its confidence interval, so the endpoint definition is available but its numerical result is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary classification at the participant level: the therapeutic outcome is either Effective or not Effective for calculation of the response rate, with Indeterminate handled separately. The underlying clinical and mycological assessments are combined according to the rule in the registry. This is not simply a tally of participants who improved on either component. Effective requires clinical Cure together with mycological Eradication.

How a response rate is formed

For a binary outcome, a response rate is the proportion of eligible participants who meet the success definition. The stated formula uses the number assessed as effective in the numerator and excludes those assessed as indeterminate from the denominator. The resulting proportion is presented as a percentage. Excluding indeterminate cases means the rate answers a specific question about participants with classifiable outcomes; it is not automatically the percentage among everyone enrolled.

This denominator rule makes the number and disposition of indeterminate assessments important to interpretation. If the count of indeterminate cases is not shown, a reader cannot determine how much the analysis population differs from enrollment. Nor can a reader assess whether indeterminate status was associated with participant characteristics or outcomes. Those counts and details are not reported in the registry record.

What a confidence interval would convey

In general, a confidence interval expresses uncertainty around an estimate arising from sampling variation under a statistical model. A relatively narrow interval indicates more precision under that model than a wider interval. It does not guarantee that the estimate is unbiased, and it does not address every limitation of the design. For a response rate, the interval would be interpreted alongside the count of responses, the analysis denominator, and the endpoint definition. Although the registry specifies a 95% confidence interval as the dispersion measure, the numerical interval is not reported in the registry record.

A confidence interval is not a range in which the true value is known to lie with certainty. Its usual interpretation concerns the long-run behavior of the interval procedure under repeated sampling and its assumptions. Readers should also distinguish a confidence interval from a comparison test: an interval around a single-arm rate does not create a treatment contrast when there is no control arm.

Why the study design matters

In general, random allocation to comparison groups helps balance measured and unmeasured factors on average, while masking can reduce the influence of treatment knowledge on assessment or reporting. This study is described as non-comparative, has allocation listed as not applicable, and has masking listed as none. Its results, if reported, would therefore be descriptive of the treated study population rather than a randomized estimate of a difference between treatments. The record does not establish that bias occurred, but the design does not remove those possible influences.

The primary outcome combines clinical and mycological assessments. Combining endpoints can make the success criterion more demanding and can align the result with a broader outcome than either component alone. At the same time, the final classification depends on both component assessments. The registry does not report component-level primary counts or the observed combined response, so the contribution of each component to the overall endpoint cannot be examined.

Planning calculations are not trial results

General sample-size planning asks how many participants a future design might need for a specified statistical objective. The planning illustrations on the page are based on the observed effect and use 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. Such illustrations are hypothetical design scenarios. They are not counts enrolled in this trial, are not observed power, and do not establish the rationale used to plan this study.

The assumptions also describe a two-arm comparison, whereas this record describes a non-comparative study with one listed fluconazole arm. A two-arm planning scenario should therefore not be mistaken for the actual trial design. No planning result can substitute for the unreported outcome estimates. A formal sample-size justification for this study is not reported in the registry record.

What the analysis listing does and does not show

The registry lists 14 outcome measures and zero analyses. The outcomes include clinical and mycological efficacy, symptom scores, and plasma or vaginal-fluid fluconazole concentrations. An outcome listing identifies measures of interest; it does not provide their observed values. Likewise, an analysis listing with no entries does not supply an inferential result. The statistical analysis plan, including details such as the method used to calculate the interval and any planned handling beyond the stated indeterminate rule, is not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The record lists a primary therapeutic outcome at three assessment times and 14 outcome measures overall, including secondary efficacy, symptom-score, and pharmacokinetic measures. When several outcomes or assessment times are examined, there can be more opportunities to observe an apparently notable result by chance. Statistical methods sometimes address this issue through a prespecified hierarchy or adjustment. The registry record does not report whether any multiplicity strategy was planned or used, so it is not possible to tell whether or how results across outcomes and time points were handled together.

The Day 28 assessment is identified as the primary evaluation. That designation clarifies which time point is primary in the record, but it does not reveal a full analysis plan, nor does it establish that other assessments were secondary in a formal testing hierarchy. The record also does not report interim analyses. No conclusion about interim monitoring or multiplicity adjustment should be inferred from the absence of an analysis listing.

The response-rate calculation excludes participants assessed as indeterminate, while the primary population has defined eligibility criteria. The registry does not report how many enrolled participants entered that population, how many had indeterminate outcomes, or the reasons for such classifications. It also does not report a broader missing-data plan, such as how missing clinical or mycological assessments were classified. Consequently, the effect of unavailable or indeterminate assessments on the estimate cannot be evaluated from this record.

The definition of the modified-Intent to Treat population is available, but the population count and outcome analysis are not. Enrollment of 157 is not enough to reconstruct the analysis denominator. The registry record does not report the analysis populations used for the secondary outcomes or whether those differed from the primary population.

11. 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 non-serious adverse event terms

The registry lists 157 participants at risk for serious adverse events in the Fluconazole group and zero participants affected. It also lists 157 at risk for other adverse events and 37 affected. These are distinct categories in the posted safety summary. The record does not list serious adverse event terms, and deaths are not reported in the registry record.

These counts describe the entries in the registry safety summary. They do not provide a comparison with a control group, because none is listed, and they do not establish whether an event was caused by the study treatment. A count of participants affected is also not necessarily the same as a count of individual event episodes; the registry summary gives participant counts.

The safety figures should be read alongside the study design and reporting scope. The study was open-label and non-comparative, and the record does not supply further detail about event definitions, severity, duration, exposure time, or attribution. Zero reported serious adverse events in this summary should not be interpreted as evidence that serious events are impossible or that no such events could occur in other settings. The registry record reports the displayed counts, not a comparative safety estimate.

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

This registry entry is informative about the question the study set out to examine: a single oral administration of fluconazole 150 mg for vulvovaginal candidiasis in Japanese patients. It also specifies a combined therapeutic endpoint, assessment times, a modified-Intent to Treat population, study design, enrollment, and selected safety counts. These details help readers understand what the intended outcomes mean and what kind of evidence the design could generate.

The record does not report the primary response-rate results. It therefore does not show how many participants met the combined definition of clinical Cure and mycological Eradication, what the Day 28 percentage was, or how precisely that percentage was estimated. It also does not provide a reported comparison against another treatment. No claim about a treatment effect or comparative benefit follows from the record alone.

Even a reported response rate in a single-arm study would describe the observed proportion in the defined study population; it would not establish what would have happened to comparable participants under a different treatment. The open-label design also leaves the possibility that knowledge of treatment could influence subjective components of assessment, though this record does not show whether such influence occurred. The endpoint's combined definition and indeterminate-case exclusion are important parts of interpreting any eventual estimate.

The safety summary reports zero participants affected by serious adverse events and 37 affected by other adverse events among 157 at risk in each category. These counts do not provide a randomized safety comparison, causal attribution, or detailed event descriptions. The appropriate reading is limited to the registry entries, not a general conclusion about safety.

Overall, the record supplies useful design and endpoint definitions but not the efficacy result needed to answer the primary question numerically. The numerical response estimate, its confidence interval, and the detailed analysis populations are not reported in the registry record.

13. Why This Trial Matters Statistically

This study illustrates why knowing the endpoint definition is not enough to interpret an efficacy result. Here, success requires both clinical Cure and mycological Eradication, and the rate excludes participants assessed as indeterminate. Those choices determine what the response percentage would represent. Without the numerator, denominator, and indeterminate count, even a clearly stated formula cannot yield an interpretable study estimate.

It also shows why design labels matter. A multicenter, open-label, non-comparative study can describe outcomes in participants receiving the intervention, but it does not make a randomized comparison with another treatment. Statistical language about response rates and confidence intervals must be read in light of that structure. A precise single-arm estimate, if one were available, would still answer a different question from a treatment-effect comparison.

The registry includes a safety summary, several secondary outcomes, and a primary time point, but the analysis results are absent. This distinction between outcomes registered and results reported is important for statistical reading. A record may define what investigators intended to measure without presenting the data required to assess the estimate, uncertainty, or consistency across outcomes.

Finally, hypothetical planning calculations should be kept separate from evidence generated by the study. Planning scenarios describe assumptions for a possible future design; they are not observed power and do not retroactively establish this trial's design rationale. The registry record does not report that rationale. For biostatisticians and clinical researchers, this example underscores the need to connect an endpoint's operational definition, its analysis population, the design, and the reported results before drawing statistical conclusions.

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 record is that it does not report the primary endpoint estimate or confidence interval. The counts classified as effective, ineffective, or indeterminate are also not reported. Accordingly, the response rate cannot be assessed from the registry entry, and the precision of an estimate is unknown.

The study has no listed control group and is explicitly described as non-comparative. It therefore cannot supply a randomized estimate of a difference between fluconazole and another treatment. The open-label design is another consideration for an outcome incorporating clinical assessment; the record does not provide details about assessor roles or procedures intended to limit potential assessment effects.

The enrollment figure does not give the size of the primary analysis population. The modified-Intent to Treat definition requires treatment receipt, a positive Candida test at baseline, and clinical efficacy evaluation, while indeterminate assessments are excluded from the response-rate denominator. The number meeting these conditions and the handling of other missing assessments are not reported in the registry record.

The statistical analysis plan, any multiplicity approach, interim analyses, and sample-size justification are not reported in the registry record. The listing of 14 outcome measures does not provide the corresponding results or clarify how secondary outcomes were analyzed. Although the safety summary gives participant counts, it does not report event terms for serious events, deaths, causal attribution, or a comparison group. These limitations restrict the conclusions that can be drawn from the registry record rather than establishing that the study itself lacked additional analyses or documentation.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: The Study Of Fluconazole For Vulvovaginal Candidiasis — NCT01806623.
• Registry record last updated 2021-07-19; status last verified 2021-07.
• Results first posted 2014-12-03.
• 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

This completed phase 3 study was multicenter, open-label, and non-comparative, with fluconazole as the only listed intervention. The study summary describes a single oral administration of 150 mg in Japanese patients with vulvovaginal candidiasis. The registry records enrollment of 157 participants, a start date of 2013-03-05, and primary completion on 2013-11-22.

The primary outcome was therapeutic response at Day 7, Day 14, and Day 28, with Day 28 designated as the primary evaluation. Effectiveness required clinical Cure and mycological Eradication, and indeterminate assessments were excluded from the rate denominator. The registry record does not report the response-rate results or confidence interval.

The safety summary lists zero participants affected by serious adverse events and 37 affected by other adverse events, each among 157 at risk. Serious event terms and deaths are not reported in the registry record. The design and outcome definitions can be described, but the posted information does not provide a numerical efficacy conclusion or a controlled treatment comparison.