This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for An Efficacy and Safety Study of Itraconazole Sequential Therapy (Intravenous Injection Followed by Oral Solution) in Invasive Pulmonary Fungal Infections (NCT01823289), a na phase 4 study. The primary outcome is Number of Participants With Clinical Efficacy.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01823289 |
| Phase | Phase 4 |
| Condition | Pulmonary Fungal Infection |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 71 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Xian-Janssen Pharmaceutical Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
Participants with invasive pulmonary fungal infections. The primary endpoint’s stated population was the Full analysis set: participants who received at least one intravenous infusion or oral solution of the study drug and completed at least one post-baseline visit.
Intervention
Itraconazole sequential therapy, using intravenous injection followed by oral solution.
Comparator
No separate comparator group is described. The registry record identifies a single-group study, so the treatment group does not have a concurrent control group for a like-for-like comparison.
Primary question
At Week 6, how many participants had clinical efficacy according to the study’s clinical and radiographic response categories?
The clinical question is descriptive in the posted record: it concerns the number of participants meeting the clinical efficacy outcome after sequential itraconazole therapy. The endpoint definition classifies response using signs and symptoms of invasive fungal infection and radiographic findings. It includes categories ranging from cure or marked improvement to failure.
The registry record does not report the number of participants meeting the primary endpoint, nor a denominator for that result. It therefore does not allow a reader to quantify the observed clinical response from the posted efficacy information. The study’s brief description states that efficacy and safety were evaluated, but that stated purpose is not itself evidence of an observed result.
Because there is no separate control group, this study cannot directly compare the treatment group’s outcome with an outcome under another treatment or a control condition. The record can describe the study’s intended population, treatment sequence, and endpoint; it does not, in the posted material, provide the primary outcome needed to characterize the observed Week 6 response.
3. Trial Design
This was a Phase 4, single-group study of itraconazole sequential therapy for invasive pulmonary fungal infections. The intervention was intravenous itraconazole followed by oral solution. The registry lists an enrollment of 71 participants and identifies Xian-Janssen Pharmaceutical Ltd. as the sponsor. The study status is completed; its recorded start was 2007-06 and its primary completion was 2008-12.
The allocation entry is NA, and the study model is single group. No masking was used. These design features mean that the record does not describe a randomized allocation or a masked comparison between treatment groups. There is no concurrent control arm in the posted study design. The treatment group should therefore be read as a group receiving the sequential therapy, not as one side of a randomized treatment contrast.
The registry’s primary endpoint is the number of participants with clinical efficacy at Week 6. Its stated analysis population is the Full analysis set (FAS), defined as participants who received at least one infusion or oral solution of study drug and completed at least one post-baseline visit. The enrollment figure does not establish how many participants met that population definition; the FAS size is not reported in the registry record.
The record lists three outcome measures and zero analyses. It does not report a statistical analysis plan, the method used to handle missing observations, or whether any interim analyses were planned. The record also does not explain why the study used a single-group design or how its enrollment was selected. Those details should not be inferred from the study’s phase, stated purpose, or eventual completion.
These features set the limits for interpretation: the design can describe outcomes among participants meeting the stated analysis-population criteria, but it does not provide a randomized comparison that would separate treatment-associated outcomes from other explanations.
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
| Stage | Itraconazole Sequential Therapy |
|---|---|
| STARTED | 71 |
| COMPLETED | 47 |
| NOT COMPLETED | 24 |
Reasons for not completing this period:
| Reason | Itraconazole Sequential Therapy |
|---|---|
| Lost to Follow-up | 5 |
| Adverse Event | 5 |
| Lack of Efficacy | 1 |
| Physician Decision | 9 |
| Administered contraindicated medicine | 1 |
| Transaminase increased more than 3 times | 1 |
| Drug affected efficacy and safety | 1 |
| Creatinine clearance<30milliliter/minute | 1 |
Analysis population for the primary outcome, as defined in the registry: The Full analysis set (FAS) population included all participants who received at least 1 intravenous infusion or oral solution of the study drug and completed at least 1 post-baseline visit.
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.
| Characteristic | Unit | Itraconazole Sequential Therapy |
|---|---|---|
| Participants analysed | 60 | |
| Age Continuous | years | 55.5 (33.34 to 79.45) |
| Sex: Female, Male: Female | Participants | 23 |
| Sex: Female, Male: Male | Participants | 37 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Clinical Efficacy, measured over Week 6.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with clinical efficacy at Week 6. The registry defines clinical efficacy using both clinical signs and symptoms of invasive fungal infection and radiographic findings. A participant could be classified as cured if signs and symptoms completely disappeared or radiographic manifestations fully or nearly resolved. Marked improvement required improvement or disappearance of signs and symptoms and at least 50 percent improvement in radiographic findings. The definition also includes an improved category for moderate clinical improvement with less than 50 percent radiographic improvement, and a failed category when clinical signs and symptoms were unchanged or worsened.
This kind of endpoint is used to summarize clinical and imaging response together, rather than relying on a single symptom or radiographic change alone. The category definitions describe how response was intended to be judged; they do not establish that a particular participant met a category. The endpoint is posted as a count of participants, not as an effect estimate comparing treatment groups.
The analysis population is the FAS: participants who received at least one dose by infusion or oral solution and completed at least one post-baseline visit. This definition matters because it specifies who could contribute to the endpoint result. The number of participants in that set and the number meeting the clinical efficacy definition are not reported in the registry record. Without those results, the endpoint’s observed frequency and precision cannot be assessed from the posted efficacy information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes that the study evaluated a sequential itraconazole regimen and prespecified a Week 6 clinical efficacy outcome. It does not report the primary endpoint count. Consequently, the posted efficacy information does not establish how many participants were classified as cured, markedly improved, improved, or failed. No efficacy estimate or confidence interval is given in the registry record, so the magnitude and precision of the observed response cannot be described from these data.
The enrollment of 71 refers to the study’s overall enrollment. It should not be treated as the denominator for the primary efficacy result: the endpoint’s stated population is the FAS, and the FAS size is not reported. The FAS definition additionally requires receipt of study drug and at least one post-baseline visit. The record does not say how many enrolled participants met those criteria or how participants without a qualifying post-baseline visit were handled.
The design is single group, with allocation listed as NA and no masking. Thus, there is no concurrent comparison to estimate how outcomes would differ under another treatment or a control condition. General statistical interpretation: in a single-group study, an observed outcome describes what occurred among the analyzed participants, but it does not by itself identify what would have occurred to the same participants under an alternative. This limitation is distinct from whether the outcome was measured carefully.
The registry also lists safety information. In the itraconazole sequential therapy group, 4 participants are listed as affected for serious adverse events among 61 at risk, and 13 are listed as affected for other adverse events among 61 at risk. Serious adverse event terms include Death at 3.3%, Edema at 1.6%, Fungal infection at 1.6%, and Hemoptysis at 1.6%. The record does not provide a death count in the group’s death field, nor does it state whether participants could contribute to more than one listed term. These figures describe the posted safety entries; they do not supply the missing efficacy result.
The registry’s zero listed analyses means no analyses are recorded in that field. It does not establish which calculations, if any, were performed outside the posted registry record. The number of outcome measures is three, but details for the other outcome measures are not included in the registry record described for the primary endpoint. A statistical analysis plan, multiplicity approach, and missing-data procedure are not reported in the registry record.
General statistical interpretation: estimates are more informative when the result, denominator, and uncertainty are all available. Here, the primary outcome count and its denominator are absent, so neither the response’s observed frequency nor its precision can be evaluated. The treatment design also lacks a control group, limiting what can be concluded about comparative effects. The registry record supports a description of the design, endpoint definition, analysis-population rule, and selected safety entries, but not a numerical conclusion about the primary efficacy outcome.
9. Statistical Methods Explained
The statistical information available describes the endpoint and analysis population, but does not give a primary efficacy count, an inferential analysis, or an analysis plan. The explanations below distinguish general statistical concepts from what the registry record states about this study.
What does a participant count tell the reader?
The primary outcome is expressed as the number of participants with clinical efficacy. A count conveys how many participants met the endpoint definition in the analysis population. To understand how common that outcome was within the analyzed group, a reader also needs the relevant denominator. To assess uncertainty around a response frequency, the result would generally be accompanied by an interval estimate. The registry record does not report the primary count, the FAS size, or an interval estimate, so those features cannot be evaluated for this endpoint.
General explanation: a confidence interval is a range of values compatible with the observed data under a specified statistical procedure. It communicates uncertainty in an estimate; it is not a guarantee that the true value lies within a particular realized interval. Its width depends on features such as the amount of information in the data and the variability of the outcome. No confidence interval for the primary efficacy outcome is reported in the registry record.
What does the response definition contribute?
The endpoint combines clinical and radiographic criteria. It distinguishes cure, marked improvement, improvement, and failure using the signs and symptoms of invasive fungal infection and the degree or direction of radiographic change. This gives the reader the intended rules for classifying response. However, a definition alone does not show how many participants fell into any category, how consistently categories were assigned, or whether assessment was blinded. The record states that masking was none, and it does not report further information about assessment procedures.
Why does the single-group structure matter?
General explanation: a control group can provide a contemporaneous reference for comparing outcomes under different assigned conditions. In a single-group study, a change or response observed after treatment cannot be contrasted with a concurrent control outcome. Other influences may also contribute to what is observed. This is why a single-group result is descriptive and does not provide the same kind of direct treatment contrast as a controlled comparison. The registry record does not describe a randomized comparison for this study.
Allocation is listed as NA, the model is single group, and no masking was used. These entries support the description of the study structure; they do not reveal additional allocation procedures or safeguards. Such details are not reported in the registry record.
How should planning illustrations be understood?
The sample-size assumptions describe planning illustrations computed from an observed effect, rather than the study’s own design. They specify a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a fraction of the observed effect. These are assumptions for an illustrative planning exercise, not evidence that this single-group study used those assumptions to select its enrollment.
General explanation: a sample-size calculation asks how many observations a hypothetical design might require under stated assumptions about the effect, variability, allocation, and statistical error rates. Such a calculation is conditional on those assumptions. It does not show that the study had a given probability of success after the data were observed. The planning illustrations should not be interpreted as observed power or as a record of the study’s own sample-size justification. That justification is not reported in the registry record.
The illustrations describe equal allocation between two arms, whereas the recorded study model is single group. Their design assumptions therefore should not be mistaken for a description of the trial’s actual allocation or a randomized comparison. The record does not report the protocol’s original sample-size calculation or its rationale.
What can be inferred from the posted analyses?
The registry lists zero analyses. This indicates that no analyses are recorded in that registry field; it does not establish what analyses may have been performed elsewhere. No p-value, effect estimate, confidence interval, or test procedure for the primary efficacy outcome is reported in the registry record. General explanation: a p-value, when used, describes how incompatible the observed data are with a specified null model under its assumptions. It does not measure the size or clinical importance of an effect. Since no such result is reported for the primary endpoint, there is no p-value to interpret.
The endpoint’s analysis population is defined, but its size and the handling of missing outcomes are absent. General explanation: a clear population definition and missing-data method help readers understand which observations contribute to an analysis and how incomplete records affect estimates. The FAS definition requires a post-baseline visit, but the registry record does not say how participants who did not meet that requirement were treated in other analyses, or whether any imputation or sensitivity analysis was used.
Over-dispersion is a general issue for count outcomes when observed variation exceeds what a chosen count model assumes. It can affect standard errors and uncertainty estimates if a model relies on a restrictive variance assumption. The primary endpoint is a participant count, but no model or inferential analysis is reported, so the registry record does not support an assessment of whether over-dispersion was relevant to the study’s analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists three outcome measures and zero analyses. The primary efficacy endpoint is described, but the available record does not identify the other outcome measures or report a plan for controlling multiplicity. It does not state whether the three outcomes were treated as co-primary, whether one was designated primary and the others secondary, or whether any adjustment was planned for multiple statistical tests. Those details are not reported in the registry record.
General explanation: when several outcomes or comparisons are tested, the chance of a false-positive result can increase if each is assessed without regard to the others. A prespecified testing hierarchy or other multiplicity procedure can help explain how findings should be interpreted. No such procedure is reported. Since no primary efficacy analysis is posted, there is also no reported test result to interpret in light of multiplicity.
The primary analysis population is defined as participants who received at least one infusion or oral solution and completed at least one post-baseline visit. The number meeting that definition is not reported. The record does not state how missing Week 6 assessments were handled, whether participants with incomplete follow-up were included in any other analysis population, or whether sensitivity analyses were performed. These details are not reported in the registry record.
General explanation: missing observations can affect an estimate when the participants with observed outcomes differ from those without them. The consequences depend on why data are missing and on the assumptions used in the analysis. A population definition alone does not specify a missing-data method. The available entry therefore identifies who was intended to contribute to the FAS, but does not explain the treatment of incomplete outcome data or the robustness of any efficacy conclusion.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Itraconazole Sequential Therapy | 4 | 61 | 6.6% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Itraconazole Sequential Therapy | 13 of 61 |
Most frequent serious adverse event terms
| Event term | Organ system | Itraconazole Sequential Therapy |
|---|---|---|
| Death | General disorders | 2 of 61 |
| Edema | General disorders | 1 of 61 |
| Fungal infection | Immune system disorders | 1 of 61 |
| Hemoptysis | Respiratory, thoracic and mediastinal disorders | 1 of 61 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Itraconazole Sequential Therapy |
|---|---|---|
| Hypokalaemia | Metabolism and nutrition disorders | 3 of 61 |
| Hepatic function abnormal | Hepatobiliary disorders | 3 of 61 |
| Nausea | Gastrointestinal disorders | 2 of 61 |
| Diarrhea | Gastrointestinal disorders | 2 of 61 |
| Granulocytopenia | Blood and lymphatic system disorders | 1 of 61 |
| Hyperbilirubinemia | Hepatobiliary disorders | 1 of 61 |
| GPT increased | Hepatobiliary disorders | 1 of 61 |
| Urinary tract infection | Renal and urinary disorders | 1 of 61 |
| Renal function abnormal | Renal and urinary disorders | 1 of 61 |
| Dermatoses | Skin and subcutaneous tissue disorders | 1 of 61 |
Serious events with the highest arm-level rate: death (3.3%), edema (1.6%), fungal infection (1.6%), hemoptysis (1.6%).
The registry lists safety entries for the itraconazole sequential therapy group. For serious adverse events, 4 participants were affected among 61 at risk. For other adverse events, 13 participants were affected among 61 at risk. These are separate posted summaries; the registry record does not explain whether the groups of participants overlap or how event terms were counted.
The listed serious adverse event terms are Death at 3.3%, Edema at 1.6%, Fungal infection at 1.6%, and Hemoptysis at 1.6%. The group’s death-count field is not populated, so the Death percentage should not be converted into a count. The registry record does not state whether participants could have experienced multiple terms, or provide the timing and duration of these events in the information.
General explanation: an “at risk” denominator identifies the group considered in a safety summary, while an affected-participant count describes how many people were recorded with an event under that summary. These entries do not establish causality. The registry record does not provide details that would support a comparison of event rates between treatment groups, and no separate control group is described. The safety figures should therefore be read as reported event summaries for this group, not as a controlled estimate of treatment-attributable risk.
12. What the Result Does, and Does Not, Mean
The record establishes that a completed, Phase 4, single-group study evaluated intravenous itraconazole followed by oral solution in participants with invasive pulmonary fungal infections. It defines a Week 6 clinical efficacy endpoint and specifies a Full analysis set based on receipt of study drug and completion of at least one post-baseline visit. It also includes selected safety entries for the treatment group.
The record does not report the primary efficacy count or the size of the FAS. It therefore does not show how many participants met the clinical efficacy definition, what fraction of the analysis population did so, or how precise an estimate might be. The outcome definition explains how response was to be classified; it is not a numerical result. The stated purpose of evaluating efficacy and safety likewise does not establish the outcome.
Because the trial is single group and has no separate comparator, the posted design does not support a direct comparison with another treatment or control condition. General explanation: a response observed in a treated group cannot, by itself, determine what would have happened under a different condition. This design limitation remains even if a response count is available. In this record, the primary count is absent as well.
The safety entries report affected-participant counts and serious adverse event terms, but do not provide a comparative safety analysis. The Death term is reported as a percentage, while the group death-count field is not populated. The registry does not clarify overlap among event terms or provide further context for interpreting their timing.
In short, the posted information supports a description of the study structure, endpoint definition, analysis-population criteria, and selected safety reports. It does not support a numerical conclusion about the primary efficacy result, a comparison with an alternative, or a conclusion about the cause of listed safety events. The protocol’s statistical plan, sample-size justification, and missing-data methods are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial illustrates why a clear endpoint definition and a reported endpoint result are both needed for statistical interpretation. The registry specifies a Week 6 clinical efficacy outcome and explains how clinical and radiographic findings define response categories. That information clarifies what the study intended to measure. The primary count and FAS size, however, are not reported, so the observed result and its precision cannot be assessed.
The single-group structure is also important. It permits outcomes to be described for participants receiving the sequential regimen, but the record contains no concurrent comparator for a treatment contrast. General explanation: comparative designs address a different question from descriptive studies because they provide outcomes under more than one assigned condition. The absence of a control group limits causal and comparative interpretation independently of the missing efficacy result.
The safety summary demonstrates another reporting distinction: the record presents affected-participant counts and selected event terms, but the timing, overlap among terms, and comparative context are not reported. A listed event should not be treated as proof that the intervention caused it. The safety information is descriptive within the posted study structure.
Finally, the planning assumptions should not be confused with the trial’s own design or with observed power. The registry record does not report the study’s sample-size justification or statistical analysis plan. This distinction matters whenever a planning calculation is displayed alongside results: planning assumptions describe a hypothetical design, whereas an observed analysis requires reported outcome data and methods. The record’s missing primary result prevents the central efficacy question from being quantified.
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 trial-specific limitation in the registry record is the absence of the primary efficacy result. Although the endpoint and its Week 6 timeframe are defined, the number meeting the clinical efficacy criteria and the FAS denominator are not reported. The number of participants who met the FAS criteria is also not reported, so the overall enrollment of 71 cannot be substituted as the endpoint denominator.
The study is single group, with allocation recorded as NA and no masking. No separate control group is described. This prevents a direct comparison within the trial and limits conclusions about how outcomes might differ under another condition. The registry record does not explain the rationale for the design.
The record lists three outcome measures but does not describe the other outcomes in the information available for this analysis. It records zero analyses and does not report a statistical analysis plan, a multiplicity approach, or interim analyses. Missing-data procedures and sensitivity analyses are also not reported in the registry record. The sample-size justification is not reported, and the planning assumptions are not evidence of the study’s original design calculations.
For safety, the registry gives affected-participant counts and serious adverse event terms, but does not clarify whether terms overlap or provide additional timing information. The death-count field is not populated. These omissions limit interpretation of the safety entries and do not permit a controlled safety comparison.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: An Efficacy and Safety Study of Itraconazole Sequential Therapy (Intravenous Injection Followed by Oral Solution) in Invasive Pulmonary Fungal Infections — NCT01823289.
- Registry record last updated 2013-05-31; status last verified 2013-04.
- Results first posted 2013-05-31.
- 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.
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18. Record Summary
This completed Phase 4 study evaluated intravenous itraconazole followed by oral solution in a single group of participants with invasive pulmonary fungal infections. Enrollment was 71. The recorded study dates are 2007-06 for the start and 2008-12 for primary completion. No masking was used, and allocation is listed as NA.
The primary endpoint was the number of participants with clinical efficacy at Week 6, defined using clinical signs and symptoms and radiographic findings. The stated FAS included participants who received at least one infusion or oral solution and completed at least one post-baseline visit. The primary efficacy count and FAS size are not reported in the registry record.
Safety entries list 4 participants with serious adverse events and 13 with other adverse events, each among 61 at risk. Serious event terms include Death, Edema, Fungal infection, and Hemoptysis. No separate comparator group is described; the posted efficacy information therefore does not provide a comparative result.