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 A Multicenter, Open-label Study for E7040 in Japanese Subjects With Hypervascular Tumor and Subjects With Arteriovenous Malformation (NCT01677624), a na phase 3 study. The primary outcome is Success Rate of Embolization in the Target Vessel.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01677624 |
| Phase | Phase 3 |
| Condition | Hypervascular Tumor and Arteriovenous Malformation |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 28 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eisai Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned Japanese subjects with a hypervascular tumor or an arteriovenous malformation. The registry describes a total enrollment of 28. For the primary efficacy analysis, the stated Full Analysis Set included participants who received embolization therapy with E7040 and had at least one evaluable efficacy datum after the procedure; the number meeting that definition is not reported in the registry record.
Intervention
The intervention was transcatheter arterial embolization with E7040, a device. The study aimed to evaluate efficacy and safety. The registry’s primary outcome assessed embolization performance in the target vessel during the period from Day 1, when embolization took place, up to Day 30 after treatment.
Comparator
There was no control group. The registry identifies the study as single-group, with allocation recorded as not applicable and masking as none. The design therefore does not provide a randomized or masked comparison against another treatment or a control condition.
Primary question
The primary question was how often embolization in the target vessel met the registry’s definition of success. Success combined complete embolization and intensive embolization, assessed using post-embolization digital subtraction angiography. The registry lists both an Imaging Evaluation Committee assessment and an Investigator or Subinvestigator assessment. The actual success-rate result and its 95% confidence interval are not reported in the registry record.
3. Trial Design
This was a Phase 3, multicenter, open-label, single-group study of E7040 in Japanese subjects with hypervascular tumor or arteriovenous malformation. The registry lists 28 participants enrolled and the study status as completed. The recorded start was 2012-08 and primary completion was 2013-12. The intervention was transcatheter arterial embolization with E7040, categorized in the record as a device.
Because the design was single-group, there was no randomized control arm. Allocation is recorded as not applicable, and masking as none. The study can describe outcomes observed among participants who received the intervention, but its design does not furnish a like-for-like comparison with an untreated group or another intervention. In particular, the registry does not support attributing differences between groups, because no comparison group is listed.
The primary outcome was the success rate of embolization in the target vessel, with the timeframe stated as Day 1 (embolization) up to Day 30 after treatment. The registry also lists success rate for operability of embolization as a secondary outcome. It reports two outcome measures and zero analyses. Detailed analysis results, including the primary outcome estimate, are not reported in the registry record.
The record defines a Full Analysis Set for the primary efficacy analysis as participants who received embolization therapy with E7040 and had at least one evaluable efficacy datum after the procedure. It does not state how many participants qualified for that set. The sample-size justification, any interim analyses, and further details of the statistical plan are not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: 'Insufficient response' = Insuf Resp
Overall Study
| Stage | Device E7040 |
|---|---|
| STARTED | 28 |
| COMPLETED | 27 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Device E7040 |
|---|---|
| Insuf Resp: intra-articular hemorrhage | 1 |
Analysis population for the primary outcome, as defined in the registry: The analysis was performed using Full Analysis Set defined as all participants who received embolization therapy with E7040 and had at least one evaluable efficacy data after the procedure.
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: The analysis was performed using the Safety Analysis Set defined as all participants who received the embolization therapy with E7040 and had at least 1 evaluable safety data after the procedure.
| Characteristic | Unit | Device E7040 |
|---|---|---|
| Participants analysed | 28 | |
| Age, Continuous | Years | 54.6 (SD 12.87) |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 17 |
6. Primary Endpoint
The registry lists the primary outcome as Success Rate of Embolization in the Target Vessel, measured over Day 1 (embolization) up to Day 30 after treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint was the success rate of embolization in the target vessel, expressed as a percentage of participants. Embolization performance was graded using four levels based on disappearance of contrast enhancement in the target vessel on post-embolization digital subtraction angiography. Complete embolization meant 100% disappearance; intensive embolization meant 80% or more; moderate embolization meant 50% or more but less than 80%; and mild embolization meant less than 50%. The registry defined success as the combined percentage of complete and intensive cases.
The endpoint is used to summarize whether the procedure achieved the specified angiographic level in the target vessel. Combining the two highest performance grades into a success measure gives a binary outcome for analysis: each evaluable participant either meets the success definition or does not. That summary is easier to interpret as a proportion than the full grading scale, but it also conceals differences between complete and intensive embolization. The registry states that performance was evaluated by both the Imaging Evaluation Committee and the Investigator or Subinvestigator. It does not report the resulting success percentage or 95% confidence interval.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a study designed to assess embolization performance and safety, but it does not report the numerical primary endpoint result. Although the primary outcome is named and its definition is detailed, the success rate and 95% confidence interval are not reported in the registry record. Consequently, the posted results do not establish how many participants met the endpoint, how large the estimated success proportion was, or how precise that estimate was.
The denominator also needs care. The registry reports enrollment of 28, while defining the Full Analysis Set as participants who received E7040 embolization and had at least one evaluable efficacy datum after the procedure. It does not report the number in that analysis population. Enrollment alone therefore cannot be treated as the primary endpoint denominator. The same caution applies to interpreting any plotted or tabulated analysis: the population and denominator should be checked rather than assumed to equal enrollment.
General statistical interpretation: a percentage estimates the share of an analysis population meeting a specified outcome. A confidence interval describes the uncertainty around that estimate under the method used to construct it. It does not show the range of individual participant outcomes, and it does not resolve potential limitations in the design or data collection. Here, the registry identifies a 95% confidence interval as the measure of dispersion but does not provide the interval or report the method used to calculate it.
The study is single-group and open-label, with no control group or masking. Its outcome can therefore be read as a descriptive result among eligible participants, if reported, rather than as a randomized estimate of a difference between treatments. The registry does not give a primary efficacy estimate to interpret in that way. Nor does it provide details on how missing efficacy data were handled, how disagreements between evaluators were resolved, or whether a prespecified statistical plan governed the analysis.
Safety information is presented separately. The record reports 2 participants affected by serious adverse events among 28 at risk, and 28 participants affected by other adverse events among 28 at risk. These are descriptive counts in a study without a control group; they do not by themselves establish the cause of an event or permit comparative safety conclusions. The registry lists cholangitis and hepatic failure among the top serious adverse event terms, each at 3.6%. Deaths are not reported in the registry record.
Overall, the available record gives useful definitions of the target outcome, the study population criteria, and some adverse-event counts, but it leaves the primary efficacy result and several analytical details unavailable. Conclusions about the measured success proportion should not be drawn from the design description alone.
9. Statistical Methods Explained
The registry presents a binary percentage endpoint: the percentage of participants whose embolization met the stated success definition. The success categories were complete embolization and intensive embolization. Participants graded as moderate or mild did not meet that definition. The record says that angiographic performance was evaluated after embolization using digital subtraction angiography, and that both the Imaging Evaluation Committee and the Investigator or Subinvestigator evaluated performance.
What does a percentage summarize?
General statistical explanation: A percentage for a binary endpoint summarizes the proportion of an analysis population classified as having the event of interest. For this endpoint, the event is success as defined by the two higher embolization grades. To interpret such a percentage, readers need both the number meeting the definition and the relevant denominator. The registry provides an enrollment of 28 and a Full Analysis Set definition, but it does not report the number of participants in that analysis set or the primary success result. Enrollment should not automatically be substituted for the endpoint denominator.
What does the confidence interval add?
General statistical explanation: A confidence interval conveys statistical uncertainty around an estimate. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. Its width is affected by the amount of information in the data and by the method used to calculate it. The registry specifies a 95% confidence interval for the primary outcome, but the interval itself and the calculation method are not reported in the registry record. It is therefore not possible to assess the precision of the primary estimate from the registry information.
Why does the analysis population matter?
The stated Full Analysis Set consists of participants who received embolization therapy with E7040 and had at least one evaluable efficacy datum after the procedure. This definition indicates that eligibility for the efficacy analysis depends on treatment receipt and availability of efficacy information. The number of participants included under that definition is not reported. The record does not describe how participants with no evaluable post-procedure data were treated, whether partial data were used, or what methods, if any, were used to address missing values.
General statistical explanation: Analysis populations can differ from enrollment when not every enrolled participant contributes outcome data under the stated criteria. Reporting the population size and the reasons participants are excluded or unevaluable helps readers understand the denominator and judge whether the analyzed group represents the enrolled group. Without those details, a reported percentage would need to be interpreted with attention to the stated population definition. In this record, the primary percentage itself is also absent.
What can evaluator assessments tell us?
The registry says that performance was evaluated by an Imaging Evaluation Committee and by the Investigator or Subinvestigator. It does not state whether one assessment was designated as primary, how discrepancies were handled, or whether evaluators were independent of each other. The study was open-label and masking was recorded as none. These design features are relevant when considering how assessments were made, but the registry does not provide enough detail to characterize assessment procedures beyond the descriptions given.
General statistical explanation: When an endpoint depends on classifying images or observations into categories, the clarity and reproducibility of the classification process matter. Describing evaluator roles, masking, and any approach to disagreement helps readers understand the reliability of the measurement. Those additional procedures are not reported in the registry record, so they should not be assumed.
How should the planning illustrations be read?
The sample-size assumptions describe a planning illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These assumptions are not the study’s own design justification. They also describe two-arm planning, whereas this study is listed as single-group. Any sample sizes shown in planning materials should therefore be understood as hypothetical planning illustrations, not as participants actually enrolled under that design and not as observed power. The registry’s own sample-size justification is not reported.
General statistical explanation: A planning calculation explores how much information a hypothetical study might need under specified assumptions. It depends on the assumed effect, allocation, statistical test, and other design choices. It is not a measure of the probability that a completed study has found a true effect. In this case, the record does not report the observed primary effect needed to evaluate the illustration’s assumptions, nor does the single-group study itself match the two-arm setup described for that planning exercise.
What analyses are documented?
The registry lists two outcome measures and zero analyses. It identifies a secondary outcome, success rate for operability of embolization, but does not present analysis results for it. No statistical test, adjustment method, or detailed primary analysis procedure is reported in the registry record. General statistical methods can explain how proportions and confidence intervals are commonly interpreted, but they cannot fill in an analysis method or result that the record does not document.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures: the primary success rate of embolization in the target vessel and the secondary success rate for operability of embolization. It reports zero analyses. The record does not provide a hierarchy of hypotheses, explain how the primary and secondary outcomes were handled together, or report whether any multiplicity adjustment was used. These details are not reported in the registry record, so no adjustment method or confirmatory testing strategy should be inferred.
The Full Analysis Set is defined as participants who received E7040 embolization and had at least one evaluable efficacy datum after the procedure. The number in that set is not reported. The registry does not describe how missing efficacy observations were handled, whether any imputation was used, or whether other analysis populations were considered. The definition therefore identifies who was intended to contribute to the primary efficacy analysis, but it does not answer how missing data affected the result.
The primary endpoint result and 95% confidence interval are also absent. Without them, readers cannot assess the estimate, its precision, or the contribution of different analysis choices. The record does not report an interim analysis plan, stopping rules, a statistical analysis plan, or a sample-size justification. The planning assumptions described elsewhere are illustrative and do not establish the study’s own planned analysis. These gaps limit assessment of the analysis framework, but they should be distinguished from evidence that a particular method was or was not used.
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 |
|---|---|---|---|
| Device E7040 | 2 | 28 | 7.1% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Device E7040 | 28 of 28 |
Most frequent serious adverse event terms
| Event term | Organ system | Device E7040 |
|---|---|---|
| cholangitis | Hepatobiliary disorders | 1 of 28 |
| hepatic failure | Hepatobiliary disorders | 1 of 28 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Device E7040 |
|---|---|---|
| Post embolisation syndrome | Injury, poisoning and procedural complications | 22 of 28 |
| Lymphocyte count decreased | Investigations | 9 of 28 |
| Constipation | Gastrointestinal disorders | 8 of 28 |
| Aspartate aminotransferase increased | Investigations | 7 of 28 |
| Nausea | Gastrointestinal disorders | 6 of 28 |
| Vomiting | Gastrointestinal disorders | 6 of 28 |
| Back pain | Musculoskeletal and connective tissue disorders | 6 of 28 |
| Alanine aminotransferase increased | Investigations | 5 of 28 |
| Decreased appetite | Metabolism and nutrition disorders | 5 of 28 |
| Pelvic pain | Reproductive system and breast disorders | 4 of 28 |
Serious events with the highest arm-level rate: cholangitis (3.6%), hepatic failure (3.6%).
The registry reports 28 participants at risk for both serious and other adverse events. It records 2 participants affected by serious adverse events and 28 affected by other adverse events. Cholangitis and hepatic failure are listed among the top serious adverse event terms, each at 3.6%. The number of deaths is not reported in the registry record.
These figures are counts from a single-group study, not a comparison between E7040 and a control treatment. The record does not provide a comparison group, and the counts alone do not establish whether an event was caused by the device or procedure. The registry also does not explain the relationship between the listed serious event terms and the number of participants affected, so those details should not be inferred from the summary.
General statistical explanation: Adverse-event counts describe what was recorded in the study population over the reporting period or periods captured by the registry. Their interpretation depends on definitions, observation time, event coding, and completeness of reporting. Those details are not fully. The figures are therefore best read as registry-reported descriptive safety information, not as a comparative risk estimate.
12. What the Result Does, and Does Not, Mean
The study asks a clearly defined procedural question: whether embolization in the target vessel reached the registry’s success threshold, based on angiographic disappearance of contrast enhancement. It also records a secondary outcome concerning operability of embolization and reports adverse-event counts. The registry’s endpoint categories and stated Full Analysis Set definition make the intended efficacy measure understandable.
However, the primary success percentage and its 95% confidence interval are not reported in the registry record. Without those results, the posted registry information does not show the observed level of success or its statistical precision. The enrollment of 28 cannot be used as a substitute for an endpoint result, nor can it be assumed to be the denominator for the Full Analysis Set. The count meeting the primary endpoint and the analysis-set size are not reported.
The single-group, open-label design also defines what can be learned from any observed outcomes. Such a design can describe outcomes among participants treated in the study, but it does not provide a randomized comparison with another treatment or control group. The record does not support attributing an outcome to E7040 by comparison with an alternative, and no causal or comparative conclusion should be inferred from the study structure alone.
The safety section gives descriptive counts, including 2 participants affected by serious adverse events and 28 affected by other adverse events among 28 at risk. The listed serious terms include cholangitis and hepatic failure, each at 3.6%. These entries do not establish causation or comparative safety, and deaths are not reported in the registry record.
In short, the record supports an account of the study’s question, endpoint definition, population criteria, and reported safety counts. It does not provide the primary numerical efficacy finding, its confidence interval, or several details needed to evaluate analysis and missing data. Those absences are important boundaries on interpretation, not evidence for a particular efficacy or safety conclusion.
13. Why This Trial Matters Statistically
This study is statistically notable for the gap between a detailed endpoint definition and the absence of the endpoint result. The registry specifies how success was classified, which evaluators assessed performance, the timeframe, and the intended efficacy population. Yet it does not report the success proportion or its 95% confidence interval. This makes clear that a well-described measurement is not, by itself, an interpretable result: readers also need the estimate, denominator, and uncertainty.
The single-group design is another central consideration. Without a control group, the study can summarize outcomes among participants who received embolization with E7040, but it cannot provide a randomized treatment contrast. That distinction matters when reading an efficacy question, because descriptive performance and comparative effect are different statistical targets.
The registry’s Full Analysis Set definition further highlights the importance of transparent analysis populations. The definition is stated, but the number of participants who met it and the handling of missing efficacy data are not reported. That leaves the endpoint denominator and possible influence of unavailable data unclear. Likewise, the registry lists two outcome measures and zero analyses, without describing multiplicity handling or the detailed statistical plan.
Finally, the sample-size assumptions describe a two-arm planning illustration, while the trial itself is listed as single-group. The illustration should not be confused with the study’s actual design or treated as observed power. Taken together, these features make the record useful for understanding the planned measurement and design, while showing why complete results and analysis documentation are essential to assess precision and interpret the evidence.
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 most consequential limitation of the registry record for interpretation is that it does not report the primary success-rate result or its 95% confidence interval. The number of participants in the defined Full Analysis Set is also not reported, so the primary endpoint denominator cannot be established from the record. Although enrollment is listed as 28, enrollment and the efficacy analysis population are not interchangeable without further information.
The study is single-group, open-label, and unmasked. No control group is listed, so the record does not offer a randomized comparison. The registry does not provide further detail on how the Imaging Evaluation Committee and Investigator or Subinvestigator assessments were reconciled or which assessment determined the result. It also does not report missing-data procedures, multiplicity handling, a detailed statistical analysis plan, interim analyses, or the study’s sample-size justification.
Safety results are summarized as affected-participant counts and selected serious event terms. The registry does not give additional context about event attribution or the relationship between the terms and the affected-participant count. Deaths are not reported in the registry record. These limitations restrict what can be concluded from the available summary; they do not establish that a particular analysis or procedure was absent from the study itself.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Multicenter, Open-label Study for E7040 in Japanese Subjects With Hypervascular Tumor and Subjects With Arteriovenous Malformation — NCT01677624.
- Registry record last updated 2016-02-22; status last verified 2016-01.
- Results first posted 2016-02-22.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
This completed Phase 3 study evaluated transcatheter arterial embolization with E7040 in Japanese subjects with hypervascular tumor or arteriovenous malformation. It was multicenter, open-label, and single-group, with 28 participants enrolled. The primary outcome was the success rate of embolization in the target vessel from Day 1 (embolization) up to Day 30 after treatment. Success combined complete and intensive embolization, assessed by the Imaging Evaluation Committee and by the Investigator or Subinvestigator.
The registry defines a Full Analysis Set for efficacy but does not report its size. The primary success rate and 95% confidence interval are not reported in the registry record. A secondary outcome concerned operability of embolization. Safety reporting lists 2 participants affected by serious adverse events and 28 affected by other adverse events among 28 at risk; deaths are not reported.