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 Study of E7080 in Subjects With Advanced Thyroid Cancer (NCT01728623), a na phase 2 study. The primary outcome is Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01728623 |
| Phase | Phase 2 |
| Condition | Thyroid Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 51 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eisai Co., Ltd. |
| Results posted | Yes |
2. Clinical Question
Population
Participants with advanced thyroid cancer were enrolled. The registry record reports enrollment of 51 participants and defines the safety population as those who received at least one dose of study drug and had at least one postbaseline safety evaluation. The arm label names DTC, MTC, and ATC, but the registry record does not report how participants were distributed among those categories or give further eligibility details.
Intervention
The study evaluated E7080 capsules administered orally once daily. The registry record describes evaluation of safety, efficacy, and pharmacokinetics. It does not report a dose in the information available for this analysis, nor does it describe dose changes or treatment duration beyond the endpoint timeframe.
Comparator
There was no comparator group: the study is classified as single-group, with no allocation and no masking. Its results therefore describe participants receiving E7080 in this study; they do not provide a randomized, like-for-like comparison with another treatment or with no treatment.
Primary question
The primary endpoint asks what percentage of participants in the safety population experienced at least one treatment-emergent adverse event (TEAE) during the specified observation period. The posted adverse-event figures include 51 participants at risk and 51 with other adverse events, while the serious-event figures list 51 at risk and 27 affected. These are descriptive safety counts, not evidence from a controlled comparison. Efficacy outcomes are listed among the study outcomes, but their results are not reported in the registry record.
3. Trial Design
This was a completed Phase 2 study of E7080 in subjects with advanced thyroid cancer. The registry record identifies the design as single-group, with no allocation and no masking. Enrollment was 51. The sponsor was Eisai Co., Ltd.; the study start date was 2012-09-03 and the primary completion date was 2015-07-09.
Participants received E7080 capsules orally once daily. Because there was no comparator group, the study can describe outcomes among participants receiving the study drug, but cannot estimate a randomized treatment contrast. The registry record does not report a control arm, a randomization procedure, or masking procedures beyond the classification of no masking.
The primary outcome was the percentage of participants with TEAEs. The stated timeframe ran from the screening visit to 30 days after the last dose of study drug, or was assessed up to 3 years. The safety population included participants who received at least one dose and had at least one postbaseline safety evaluation. The registry record also lists progression-free survival, overall survival, best overall response, objective response rate, disease control rate, and clinical benefit rate as secondary outcomes. Results for those efficacy outcomes are not reported in the registry record.
The registry record does not report the dose, detailed eligibility criteria, recruitment setting, treatment exposure by participant, or the statistical plan. It also does not report the rationale for the enrollment target. These omissions limit how fully the design and its intended analyses can be reconstructed from the registry information.
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 | Arm 4: Lenvatinib (MTC, DTC, ATC) |
|---|---|
| STARTED | 51 |
| DTC | 25 |
| MTC | 9 |
| ATC | 17 |
| COMPLETED | 23 |
| NOT COMPLETED | 28 |
Reasons for not completing this period:
| Reason | Arm 4: Lenvatinib (MTC, DTC, ATC) |
|---|---|
| Disease progression | 26 |
| Adverse Event | 1 |
| Withdrawal by Subject | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety population included all participants who received at least one dose of study drug and had at least one postbaseline safety evaluation.
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: Safety analysis set included all participants who received at least one dose of study drug and had at least one postbaseline safety evaluation.
| Characteristic | Unit | Lenvatinib (Arm 1) | Lenvatinib (Arm 2) | Lenvatinib (Arm 3) | Total |
|---|---|---|---|---|---|
| Participants analysed | 25 | 9 | 17 | 51 | |
| Age, Continuous | years | 57.1 (SD 12.34) | 60.6 (SD 13.70) | 63.4 (SD 11.40) | 59.8 (SD 12.36) |
| Sex: Female, Male: Female | Participants | 16 | 3 | 11 | 30 |
| Sex: Female, Male: Male | Participants | 9 | 6 | 6 | 21 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Treatment-Emergent Adverse Events (TEAEs), measured over Screening visit to 30 days after the last dose of study drug, or assessed up to 3 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with treatment-emergent adverse events. A TEAE is an adverse event occurring in the study’s treatment-emergent observation period; the registry describes that period as screening through 30 days after the last dose, or assessment up to 3 years. The safety population consisted of participants who received at least one dose and had at least one postbaseline safety evaluation.
For this summary, a participant was counted only once within the same adverse-event category. If multiple events in that category had different CTCAE v 4.0 grades, only the event with the highest grade was reported. The registry states that alopecia and infertility were exceptions to the use of CTCAE v 4.0 for grading.
General statistical context: a participant-level percentage describes how common an event was in the observed safety population. It is useful for summarizing the frequency of harms, but it does not by itself show whether the study drug caused an event. Without a comparator group, the endpoint also cannot establish how the observed frequency differs from what would have occurred under another treatment or without the study drug. The registry record does not report a confidence interval or an inferential analysis for the primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety results describe a single group of participants, not a treatment comparison. The adverse-event record lists 51 participants at risk for serious events and 27 affected. It also lists 51 at risk and 51 affected for other events, with 4 deaths recorded. These counts provide a description of events recorded during the study; they do not determine whether an event was caused by E7080.
The listed serious adverse-event terms include decreased appetite at 13.7%, malignant neoplasm progression at 7.8%, pneumonia at 5.9%, nausea at 3.9%, laryngeal stenosis at 3.9%, and diarrhoea at 2.0%. These percentages describe the registry’s listed event terms. The record’s rule that a participant is counted once within the same category, and that the highest grade is used when grades differ, matters when interpreting the summaries. The figures should not be treated as the number of all individual event episodes.
No efficacy estimates or analyses are reported in the registry record. Although progression-free survival, overall survival, best overall response, objective response rate, disease control rate, and clinical benefit rate are listed as secondary outcomes, their results are not available in the registry information used for this analysis. It is therefore not possible to describe the observed efficacy outcomes, their precision, or their relation to the safety findings.
There are no reported confidence intervals, p-values, or analysis results in the registry record. A percentage is a point estimate: it gives the observed frequency in the analyzed group, but does not quantify uncertainty around that frequency. General statistical context: confidence intervals can express the range of values compatible with data under a specified method; without one, readers cannot use the registry summary to assess that uncertainty directly.
The single-group design is central to interpretation. Event frequencies can be useful descriptive information, but comparison with an external rate would require an appropriate comparator and careful consideration of differences in population, event definitions, follow-up, and ascertainment. The registry record does not report such a comparison. The results should therefore be read as observations from this study’s safety population, rather than as evidence of a causal effect or a comparative benefit-risk estimate.
9. Statistical Methods Explained
The registry record describes a Phase 2, single-group study and lists a binary percentage as the primary outcome. The available information supports a descriptive reading of the safety summaries. It does not report a statistical analysis plan, inferential test, confidence interval, or model result. The explanations below give general statistical context for interpreting this type of outcome and distinguish that context from the analyses documented in the registry.
What does a participant percentage describe?
General statistical context: for a binary outcome, each participant is classified according to whether the defined event occurred during the relevant observation period. The percentage is the proportion of eligible participants classified as having the event. Its interpretation depends on the event definition, the population included in the denominator, and the observation period. Here, the registry defines the safety population as participants who received at least one dose and had at least one postbaseline safety evaluation. That is narrower than simply referring to everyone enrolled, and the registry record does not give a separate count for those who did not meet the safety-population definition.
For the TEAE summary, the record says only TEAEs are included, participants are counted once in the same category, and the highest grade is reported when multiple events in a category have different grades. This means the category-level summary is not a count of every episode. The record says CTCAE v 4.0 was used for grading, except for alopecia and infertility. Consistent definitions matter because a frequency can change when the category, grading approach, or period of observation changes.
What does uncertainty mean for a percentage?
General statistical context: an observed percentage is subject to sampling variability. Another group drawn from the same underlying population could have a different observed frequency, even if the underlying event probability were unchanged. A confidence interval is one conventional way to show uncertainty around an estimate, under assumptions about sampling and the calculation method. A narrower interval generally indicates greater precision, while a wider interval indicates less precision. The registry record does not report a confidence interval for the primary outcome, so its precision cannot be evaluated from that measure.
The number of participants, event frequency, follow-up completeness, and analysis method can all affect precision. These ideas do not permit a new uncertainty estimate without the necessary calculations and assumptions. No such estimate is reported in the registry record, and none should be inferred from the percentages alone.
Why does the single-group design matter?
General statistical context: a controlled comparison estimates how outcomes differ between groups under a study design intended to make those groups comparable. A single-group study has no concurrent control group, so its observed event percentages do not directly estimate a treatment effect relative to another option. Other factors, including participants’ underlying illness, other care, and the way events are observed, may contribute to the events recorded. This is a design limitation, not something that can be corrected by interpreting a percentage differently.
The registry classifies allocation as NA, masking as NONE, and the model as SINGLE GROUP. Thus, the registry record does not describe a randomized or masked comparison. It does not report an adjusted analysis or a method for addressing differences from any external population. The adverse-event figures should be treated as descriptive observations in the study group.
What can be said about planning calculations?
General statistical context: sample-size calculations for a hypothetical comparison depend on assumptions about the effect to detect, the significance level, allocation, and the chosen statistical approximation. Such calculations are planning illustrations, not observed power and not evidence that a study did or did not detect an effect. The registry record does not report the trial’s sample-size justification or a numerical calculation for the study’s own design. No numerical planning result is reported in the registry information used for this analysis.
Although a planning description references a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a fraction of an observed effect, those are assumptions for a hypothetical two-arm illustration. They do not describe this single-group trial’s allocation or establish that its design used those assumptions. The registry record does not provide the resulting planning values in the information available for this analysis. They should not be presented as actual trial findings.
Why are no inferential results described?
The registry record lists zero analyses. It contains no reported p-value, confidence interval, adjusted estimate, or model output for the primary endpoint. General statistical context: inferential results require a specified analysis and assumptions about the data. Without those reported details, readers cannot assess whether an analysis accounted for differing follow-up, repeated events, or other features of the data. The descriptive safety counts remain interpretable as reported, but they do not substitute for an inferential analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary safety outcome and six secondary outcomes: progression-free survival, overall survival, best overall response, objective response rate, disease control rate, and clinical benefit rate. It does not report results for those secondary outcomes. It also does not report a multiplicity strategy, such as a prespecified ordering of tests or an adjustment for making multiple statistical comparisons. Since no analyses are listed, the record offers no inferential results on which to assess how multiple testing was handled.
General statistical context: when several outcomes are tested, the chance of at least one apparently notable result can increase if each is interpreted without accounting for the set of tests. A multiplicity approach may control that risk, but it depends on the planned hypotheses and analysis. The registry record does not report whether any such approach was planned or used. It would be inappropriate to infer one from the outcome list alone.
The record also does not report a missing-data strategy, outcome-specific analysis populations, or how incomplete follow-up was handled for efficacy outcomes. The primary safety population is defined as participants who received at least one dose and had at least one postbaseline safety evaluation. That definition identifies who was eligible for the safety summary, but does not explain how missing safety assessments were managed or whether all eligible participants contributed to every endpoint.
General statistical context: missingness can affect interpretation when participants with unavailable outcomes differ from those with observed outcomes. The extent and direction of any such effect depend on why data are missing and on the analysis choices. The registry record does not report enough information to characterize missingness or its possible impact. It also does not report a protocol-level analysis population for the listed efficacy outcomes.
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 |
|---|---|---|---|
| Arm 4: Lenvatinib (DTC, MTC, ATC) | 27 | 51 | 52.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Arm 4: Lenvatinib (DTC, MTC, ATC) | 4 of 51 |
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 |
|---|---|
| Arm 4: Lenvatinib (DTC, MTC, ATC) | 51 of 51 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm 4: Lenvatinib (DTC, MTC, ATC) |
|---|---|---|
| Decreased appetite | Metabolism and nutrition disorders | 7 of 51 |
| Malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 4 of 51 |
| Pneumonia | Infections and infestations | 3 of 51 |
| Nausea | Gastrointestinal disorders | 2 of 51 |
| Laryngeal stenosis | Respiratory, thoracic and mediastinal disorders | 2 of 51 |
| Diarrhoea | Gastrointestinal disorders | 1 of 51 |
| Dysphagia | Gastrointestinal disorders | 1 of 51 |
| Gastric ulcer | Gastrointestinal disorders | 1 of 51 |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | 1 of 51 |
| Fatigue | General disorders | 1 of 51 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm 4: Lenvatinib (DTC, MTC, ATC) |
|---|---|---|
| Hypertension | Vascular disorders | 46 of 51 |
| Decreased appetite | Metabolism and nutrition disorders | 39 of 51 |
| Palmar-plantar erythrodysaesthesia syndrome | Skin and subcutaneous tissue disorders | 39 of 51 |
| Fatigue | General disorders | 37 of 51 |
| Proteinuria | Renal and urinary disorders | 31 of 51 |
| Stomatitis | Gastrointestinal disorders | 29 of 51 |
| Diarrhoea | Gastrointestinal disorders | 27 of 51 |
| Nausea | Gastrointestinal disorders | 21 of 51 |
| Dysphonia | Respiratory, thoracic and mediastinal disorders | 21 of 51 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 19 of 51 |
Serious events with the highest arm-level rate: decreased appetite (13.7%), malignant neoplasm progression (7.8%), pneumonia (5.9%), nausea (3.9%), laryngeal stenosis (3.9%).
The serious-event figures list 51 participants at risk and 27 affected. The other-event figures list 51 at risk and 51 affected. The registry also records 4 deaths. These are separate posted summaries; the record does not provide enough detail in the information used to establish whether the death count overlaps with the affected counts or to attribute deaths to a particular cause.
The listed serious terms and percentages are decreased appetite, 13.7%; malignant neoplasm progression, 7.8%; pneumonia, 5.9%; nausea, 3.9%; laryngeal stenosis, 3.9%; and diarrhoea, 2.0%. These are reported as serious-event terms, not as a complete accounting of every adverse event or as proof of causation. The endpoint description also specifies category-level counting and selection of the highest grade when a participant had events of different grades in the same category.
General statistical context: safety counts are descriptive, and their interpretation depends on the definitions used, the participants included, and the duration and completeness of observation. A serious adverse event is not interchangeable with a severe event; the registry’s detailed classification criteria are not reported in the information used for this analysis. With no comparator group, the figures do not quantify an excess risk attributable to E7080.
12. What the Result Does, and Does Not, Mean
The record documents a completed Phase 2, single-group study of E7080 in advanced thyroid cancer, with enrollment of 51 participants. The primary outcome concerns treatment-emergent adverse events in a defined safety population. The posted summaries include serious-event counts, other-event counts, deaths, and selected serious-event terms. Those observations describe what was recorded in this study under the registry’s definitions.
They do not establish that E7080 caused the recorded events. A single-group design does not show what event frequency would have occurred in a comparable group receiving another treatment or no study drug. The study is also unmasked according to the registry classification; the record does not explain whether or how that might have affected event reporting or assessment.
The registry lists efficacy outcomes, including progression-free survival, overall survival, and response measures, but does not report their results in the information available for this analysis. Consequently, the record cannot support a description of observed efficacy, an estimate of treatment effect, or a benefit-risk comparison. It also contains no reported inferential analysis or confidence interval for the primary safety endpoint, so the precision of the percentage estimates cannot be assessed from the reported material.
General statistical context: a descriptive event frequency can help characterize the experience of participants in a study, but it is not equivalent to a causal conclusion. A causal or comparative interpretation requires an appropriate design and analysis, as well as attention to population, follow-up, outcome definitions, and missing information. The results should be understood within those limits: they are observations from the study’s safety population and are not, on their own, a comparison with another care strategy.
13. Why This Trial Matters Statistically
This study illustrates why design labels are essential to interpreting clinical-trial statistics. A single-group study can report the frequency of observed events in participants who received the study drug, but it does not create a randomized treatment contrast. The distinction helps prevent descriptive safety figures from being read as estimates of comparative harm or benefit.
The primary endpoint also highlights how outcome definitions shape a result. The registry specifies the observation period, the safety-population criteria, category-level counting, and the approach to grading. These details affect what the percentage represents. A participant-level summary is not a tally of every event episode, and the stated grading rule means the recorded category reflects the highest grade when grades differ.
The registry lists several efficacy outcomes but does not report their results or any analysis output. This makes the boundary between an outcome being named and an outcome being quantitatively interpretable especially important. A listed endpoint alone is not evidence of an observed effect; estimates and their uncertainty are needed to assess results, while a suitable design is needed for comparative interpretation.
General statistical context: clear reporting of analysis populations, missing-data handling, multiplicity, and uncertainty helps readers judge how robust an estimate may be. Those details are not reported in the registry record used for this analysis. Their absence does not change the recorded event counts, but it limits what can be concluded about precision and analysis choices. This trial is therefore a useful example of reading safety summaries as descriptive evidence while keeping design and reporting limitations in view.
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 important limitation is the single-group design. There is no concurrent comparator, and the record classifies the study as having no allocation and no masking. The observed safety frequencies therefore cannot establish a treatment contrast or show that E7080 caused the events. Comparisons with another population would require evidence about how comparable the participants, event definitions, follow-up, and ascertainment were; such a comparison is not reported in the registry record.
The registry record does not report the statistical analysis plan, inferential analyses, confidence intervals, p-values, or a sample-size justification. It also does not report a missing-data approach, the handling of incomplete follow-up, or whether outcomes other than the primary safety measure used the same analysis population. The listed efficacy outcomes have no reported results in the information used here, so they cannot be interpreted quantitatively.
The safety population definition excludes enrolled participants who did not receive at least one dose or did not have a postbaseline safety evaluation. The registry record does not give further detail on such participants or indicate whether any safety assessments were missing among those eligible for the analysis. The endpoint description gives a broad observation period, from screening to 30 days after the last dose or assessment up to 3 years, but does not report participant-level exposure or follow-up duration.
The arm label names DTC, MTC, and ATC, but the record does not provide subgroup counts or results. The available information also does not include detailed eligibility criteria or event-level data. These limitations constrain assessment of subgroup patterns, event timing, and the precision of the reported summaries.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of E7080 in Subjects With Advanced Thyroid Cancer — NCT01728623.
- Registry record last updated 2020-08-14; status last verified 2020-07.
- Results first posted 2020-08-14.
- 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.
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18. Record Summary
The registry record describes NCT01728623, a completed Phase 2, single-group study of E7080 in advanced thyroid cancer, sponsored by Eisai Co., Ltd. Enrollment was 51. Participants received E7080 capsules orally once daily. The primary endpoint was the percentage of participants with treatment-emergent adverse events in a defined safety population. Its timeframe ran from screening to 30 days after the last dose, or was assessed up to 3 years.
Posted safety figures list 27 participants affected by serious events among 51 at risk, 51 affected by other events among 51 at risk, and 4 deaths. Efficacy outcomes are listed, but their results are not reported in the registry record. The record also lists no analyses and does not report confidence intervals, p-values, or the statistical plan. Findings are therefore descriptive and cannot establish a comparative treatment effect or causal attribution.