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 This is a Phase 1 Study of Eribulin Mesylate in Pediatric Participants With Recurrent or Refractory Solid Tumors (Excluding [Central Nervous System] CNS), Including Lymphomas (NCT02171260), a na phase 1 study. The primary outcome is Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02171260 |
| Phase | Phase 1 |
| Condition | Pediatrics, Solid Tumors |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eisai Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Children with recurrent or refractory solid tumors, including lymphomas and excluding CNS tumors, were eligible for the study. Part A1 covered participants aged at least 12 months and younger than 18 years. Part A2 was intended for infants older than 6 months and younger than 12 months. The registry describes the primary safety population as participants who received at least one dose of study drug; enrollment was 23.
Intervention
The intervention was intravenous eribulin mesylate, planned on Days 1 and 8 of each 21-day cycle. The study’s dose-finding aim was to determine the maximum tolerated dose and/or recommended Phase 2 dose for the regimen. The posted safety groups include Part A1 dose levels of 1.1 mg/m^2, 1.4 mg/m^2, and 1.8 mg/m^2, as well as a pharmacokinetic expansion group.
Comparator
There was no randomized control group. The record describes a single-group study, with allocation listed as not applicable and masking listed as none. The dose groups are sequential or expansion cohorts within a Phase 1 investigation, not like-for-like randomized treatment arms. Differences between their observed event counts should therefore not be interpreted as controlled comparisons of dose effects.
Primary question
The primary endpoint counted participants with treatment-emergent adverse events and serious adverse events. Events were counted from the first dose through 30 days after the last dose. The study also aimed to describe toxicity and pharmacokinetics and to characterize antitumor effects preliminarily. The posted safety figures describe event occurrence in the listed groups; they do not by themselves establish comparative safety, dose-response, or antitumor benefit.
3. Trial Design
This was a completed Phase 1, single-group study of eribulin mesylate in pediatric participants with recurrent or refractory solid tumors, including lymphomas and excluding CNS tumors. The registry lists enrollment as 23. The study began on 2014-07-31, and primary completion was recorded as 2016-01-28. The intervention was administered intravenously on Days 1 and 8 of each 21-day cycle.
The dose-finding program included Part A1 for participants aged at least 12 months and younger than 18 years, and Part A2 for infants older than 6 months and younger than 12 months. Part A2 was planned to enroll one dose level behind the Part A1 level then enrolling, as a safety measure for infants. The posted adverse-event groups are labeled Part A1: eribulin mesylate 1.1 mg/m^2, 1.4 mg/m^2, and 1.8 mg/m^2, followed by a Part A1 pharmacokinetic expansion group. The registry summary says the starting dose was 1.1 mg/m^2 and approximately 80% of the adult maximum tolerated dose. The available arm description is incomplete, so further dose-escalation rules are not reported in the registry record.
The study aimed to determine the maximum tolerated dose and/or recommended Phase 2 dose, describe toxicities and pharmacokinetics, and preliminarily describe antitumor effects. The design is appropriate to read as an early-phase, safety- and dose-finding investigation. There is no randomized comparator, and the groups are not control arms. The record lists no masking. Results across groups are descriptive; they do not isolate the effect of dose from differences in cohort membership, timing, or other factors.
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: In Part A1, a total of 23 participants of greater than or equal to (\>=) 12 months were enrolled, of which 22 were treated in the study. In Part A2, the study was open for the enrolment of infant participants of less than 12 months of age, however no participants were enrolled into this part.
Overall Study
| Stage | Part A1: Eribulin Mesylate 1.1 mg/m^2 | Part A1: Eribulin Mesylate 1.4 mg/m^2 | Part A1: Eribulin Mesylate 1.8 mg/m^2 | Part A1: Eribulin Mesylate PK Expansion |
|---|---|---|---|---|
| STARTED | 6 | 6 | 5 | 5 |
| COMPLETED | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 6 | 6 | 5 | 5 |
Reasons for not completing this period:
| Reason | Part A1: Eribulin Mesylate 1.1 mg/m^2 | Part A1: Eribulin Mesylate 1.4 mg/m^2 | Part A1: Eribulin Mesylate 1.8 mg/m^2 | Part A1: Eribulin Mesylate PK Expansion |
|---|---|---|---|---|
| Death | 0 | 0 | 0 | 1 |
| Physician Decision | 1 | 0 | 1 | 0 |
| Withdrawal by Subject | 1 | 0 | 0 | 0 |
| Evidence of progressive disease | 4 | 6 | 4 | 4 |
Analysis population for the primary outcome, as defined in the registry: The dose evaluable set (DES) included all participants who were judged as DLT evaluable as recorded in the database. In order to be DLT evaluable, all participants had to complete Cycle 1.
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 safety analysis set (SAS) included all participants who received at least one dose of study drug.
| Characteristic | Unit | Part A1: Eribulin Mesylate 1.1 mg/m^2 | Part A1: Eribulin Mesylate 1.4 mg/m^2 | Part A1: Eribulin Mesylate 1.8 mg/m^2 | Part A1: Eribulin Mesylate PK Expansion | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 6 | 6 | 5 | 5 | 22 | |
| Age, Continuous | years | 14.0 (SD 3.52) | 10.7 (SD 2.25) | 13.0 (SD 3.24) | 12.6 (SD 5.50) | 12.5 (SD 3.69) |
| Sex: Female, Male: Female | Participants | 1 | 4 | 3 | 2 | 10 |
| Sex: Female, Male: Male | Participants | 5 | 2 | 2 | 3 | 12 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 2 | 1 | 1 | 4 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 6 | 4 | 4 | 4 | 18 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 1 | 1 | 3 |
| Race (NIH/OMB): White | Participants | 5 | 5 | 4 | 2 | 16 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 1 | 0 | 2 | 3 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs), measured over First dose of study drug (Baseline) up to 30 days after last dose of study drug (Cycle 8 Day 38).
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs). A TEAE was an event that occurred, or worsened if already present at baseline, after the first study-drug dose through 30 days after the last dose. An adverse event was defined as an untoward medical occurrence in a participant receiving an investigational product and did not necessarily have to be caused by the product.
The registry defined an SAE by specified consequences, including death, a life-threatening event, inpatient hospitalization or prolonged hospitalization, persistent or significant incapacity, substantial disruption of normal life functions, or a congenital anomaly or birth defect. These definitions distinguish seriousness from whether an event is judged treatment-related: an event can meet the seriousness criteria without a causal relationship being established.
Counting participants with events is useful in an early-phase study because it gives a direct description of how many treated participants experienced safety events during the defined observation window. It can support dose-finding and safety characterization, but a count does not convey event severity beyond the separate serious-event classification, event duration, or causal certainty. The registry record does not report a treatment-effect estimate for this endpoint or a comparative statistical test.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted adverse-event results divide participants into four Part A1 groups: the 1.1 mg/m^2 group, the 1.4 mg/m^2 group, the 1.8 mg/m^2 group, and the pharmacokinetic expansion group. For serious adverse events, the record lists 2 participants among 6 at risk in the 1.1 mg/m^2 group, 1 among 6 in the 1.4 mg/m^2 group, 3 among 5 in the 1.8 mg/m^2 group, and 4 among 5 in the pharmacokinetic expansion group. These are descriptive counts within small cohorts, not estimates from a randomized comparison.
For other adverse events, the record lists 6 participants among 6 at risk in each of the 1.1 mg/m^2 and 1.4 mg/m^2 groups, 5 among 5 in the 1.8 mg/m^2 group, and 5 among 5 in the pharmacokinetic expansion group. These figures indicate that every participant listed as at risk in each group had at least one event in this category. They should not be read as counts of all events: the endpoint reports participants with events, so a participant may have experienced more than one event.
The record lists deaths as 0 in each of the first three groups and 1 in the pharmacokinetic expansion group. This is a count within the posted safety groups; it does not establish the cause of death or whether a study drug caused it. The registry definition of an adverse event does not require a causal relationship, and the displayed death count alone does not provide an attribution.
The most frequently listed serious-event terms include hypoxia at 40.0%, and febrile neutropenia, gingival pain, pyrexia, device related infection, and lung infection at 20.0% each. The denominator and method used to calculate these term percentages are not reported in the registry record. They should be treated as descriptive registry percentages, not as comparisons between treatment groups or as estimates of risk for other populations.
Because this was a single-group Phase 1 study, observed differences between dose cohorts cannot be interpreted as dose effects without a suitable comparative design and analysis. Cohort sizes are small, and the record does not report confidence intervals or adjusted estimates for the safety counts. The findings characterize what was recorded among treated participants, rather than establishing that eribulin caused each event or that one dose is safer than another.
9. Statistical Methods Explained
The main statistical output for this study is a count of participants who experienced safety events. The registry identifies the primary endpoint as the number of participants with TEAEs and SAEs. A count is a direct summary of observed participants, not a model-based estimate of treatment effect. It is particularly understandable in an early-phase safety study, where the purpose includes documenting adverse events and supporting dose-finding.
What does a participant count represent?
A participant count records whether each person met the event definition during the specified observation period. It is different from an event count: one participant can have multiple events, but the participant is counted once for a participant-level endpoint. The registry groups safety outcomes as serious adverse events and other adverse events, with the number at risk and the number affected listed for each dose group. The counts should therefore be interpreted as numbers of participants, not as totals of every occurrence or episode.
The primary endpoint observation window starts with the first study-drug dose and continues through 30 days after the last dose. The definition includes events that begin during treatment and events already present at baseline that worsen after the first dose. A consistent window helps define what qualifies as treatment-emergent, but the time window does not prove causation. The registry expressly defines an adverse event as not necessarily having a causal relationship with the medicinal product.
Why separate serious and other events?
Seriousness is classified by consequences, such as death, a life-threatening event, hospitalization, or substantial and persistent impairment, rather than simply by how often an event occurs. Reporting serious events separately highlights events meeting those criteria. The other-event counts describe participants affected by events not included in the serious category used in the posted summaries. Neither category alone conveys the full clinical context of each event, its duration, whether it recurred, or the investigators’ causal assessment.
The serious-event terms are also presented as descriptive percentages. The registry record lists hypoxia at 40.0%, and febrile neutropenia, gingival pain, pyrexia, device related infection, and lung infection at 20.0% each. The denominator and percentage calculation method are not reported in the registry record. Without that information, the percentages should not be reverse-engineered into counts or compared with the group-specific participant counts. A percentage is not a substitute for a clear denominator and definition.
What can comparisons between groups show?
General statistical principle: a randomized comparison helps balance, on average, factors other than assigned treatment. This study did not use randomized allocation and is recorded as a single-group study. Its dose groups are therefore descriptive cohorts rather than randomized arms. A difference in counts between cohorts may reflect cohort composition or other differences as well as dose; the registry does not report a controlled analysis to separate these explanations.
When group sizes are small, a single participant can materially change a proportion. General statistical principle: uncertainty intervals can communicate the range of values compatible with observed data, but no confidence intervals for these event counts are reported in the registry record. The raw counts remain useful for describing the observed participants, while not supporting precise estimates of event frequency beyond the groups themselves.
How should planning calculations be read?
General statistical principle: a planning calculation asks how many participants might be needed under specified assumptions to detect a chosen effect with a chosen statistical procedure. Such a calculation is not evidence that a study had a particular probability of detecting its true effect after results are observed. Any planning illustrations associated with the results are hypothetical design calculations based on an observed effect and stated assumptions: a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They are not the trial’s own sample-size justification, and they are not observed power.
The registry record does not report the trial’s sample-size justification, statistical plan, or a randomized two-arm comparison. The planning assumptions therefore should not be mistaken for features of the actual study design. In particular, equal allocation between two arms does not describe the single-group, dose-cohort design. The record also does not report a treatment-effect analysis for the primary safety endpoint. Results are best read as descriptive evidence from the posted groups, with interpretation bounded by the study design and the event definitions.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report how multiplicity was handled across the safety outcomes, dose groups, or secondary outcomes. Multiplicity arises when several outcomes or comparisons are examined; without a prespecified strategy, repeated testing can increase the chance of apparently notable findings by chance. No adjustment method or hierarchy for interpreting outcomes is reported in the registry record. The descriptive event counts can be read without assuming that formal hypothesis tests were conducted.
Missing-data procedures are not reported in the registry record. The record does not describe how participants with incomplete follow-up, missing event information, or early treatment discontinuation were handled for the endpoint. The stated safety analysis population included participants who received at least one dose of study drug. That definition identifies who was eligible for the safety analysis, but does not explain missing observations or provide details about follow-up completeness.
The registry record does not report an analysis population for efficacy outcomes or a statistical analysis plan. It also does not describe interim analyses, imputation, sensitivity analyses, or prespecified subgroup analyses. These elements should not be inferred from the observed counts or from general Phase 1 practice. Consequently, the safety summaries are most appropriately read as posted descriptive results for the listed groups and outcome definitions, rather than as results from a documented multiplicity-controlled inferential analysis.
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 |
|---|---|---|---|
| Part A1: Eribulin Mesylate 1.1 mg/m^2 | 2 | 6 | 33.3% |
| Part A1: Eribulin Mesylate 1.4 mg/m^2 | 1 | 6 | 16.7% |
| Part A1: Eribulin Mesylate 1.8 mg/m^2 | 3 | 5 | 60.0% |
| Part A1: Eribulin Mesylate PK Expansion | 4 | 5 | 80.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Part A1: Eribulin Mesylate 1.1 mg/m^2 | 0 of 6 |
| Part A1: Eribulin Mesylate 1.4 mg/m^2 | 0 of 6 |
| Part A1: Eribulin Mesylate 1.8 mg/m^2 | 0 of 5 |
| Part A1: Eribulin Mesylate PK Expansion | 1 of 5 |
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 |
|---|---|
| Part A1: Eribulin Mesylate 1.1 mg/m^2 | 6 of 6 |
| Part A1: Eribulin Mesylate 1.4 mg/m^2 | 6 of 6 |
| Part A1: Eribulin Mesylate 1.8 mg/m^2 | 5 of 5 |
| Part A1: Eribulin Mesylate PK Expansion | 5 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Part A1: Eribulin Mesylate 1.1 mg/m^2 | Part A1: Eribulin Mesylate 1.4 mg/m^2 | Part A1: Eribulin Mesylate 1.8 mg/m^2 | Part A1: Eribulin Mesylate PK Expansion |
|---|---|---|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 6 | 0 of 6 | 0 of 5 | 2 of 5 |
| Device related infection | Infections and infestations | 0 of 6 | 0 of 6 | 1 of 5 | 1 of 5 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 1 of 6 | 1 of 6 | 0 of 5 | 0 of 5 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 6 | 0 of 6 | 1 of 5 | 0 of 5 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 6 | 1 of 6 | 0 of 5 | 0 of 5 |
| Gingival pain | Gastrointestinal disorders | 0 of 6 | 0 of 6 | 0 of 5 | 1 of 5 |
| Fatigue | General disorders | 0 of 6 | 1 of 6 | 0 of 5 | 0 of 5 |
| Pyrexia | General disorders | 0 of 6 | 0 of 6 | 1 of 5 | 0 of 5 |
| Lung infection | Infections and infestations | 0 of 6 | 0 of 6 | 0 of 5 | 1 of 5 |
| Pneumonia | Infections and infestations | 0 of 6 | 0 of 6 | 0 of 5 | 1 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Part A1: Eribulin Mesylate 1.1 mg/m^2 | Part A1: Eribulin Mesylate 1.4 mg/m^2 | Part A1: Eribulin Mesylate 1.8 mg/m^2 | Part A1: Eribulin Mesylate PK Expansion |
|---|---|---|---|---|---|
| White blood cell count decreased | Investigations | 5 of 6 | 5 of 6 | 5 of 5 | 4 of 5 |
| Lymphocyte count decreased | Investigations | 4 of 6 | 3 of 6 | 3 of 5 | 4 of 5 |
| Anaemia | Blood and lymphatic system disorders | 4 of 6 | 4 of 6 | 2 of 5 | 3 of 5 |
| Neutrophil count decreased | Investigations | 5 of 6 | 2 of 6 | 4 of 5 | 2 of 5 |
| Nausea | Gastrointestinal disorders | 3 of 6 | 3 of 6 | 4 of 5 | 1 of 5 |
| Decreased appetite | Metabolism and nutrition disorders | 3 of 6 | 4 of 6 | 2 of 5 | 2 of 5 |
| Haemoglobin decreased | Investigations | 3 of 6 | 3 of 6 | 4 of 5 | 0 of 5 |
| Vomiting | Gastrointestinal disorders | 3 of 6 | 2 of 6 | 2 of 5 | 2 of 5 |
| Aspartate aminotransferase increased | Investigations | 4 of 6 | 2 of 6 | 2 of 5 | 1 of 5 |
| Platelet count decreased | Investigations | 1 of 6 | 2 of 6 | 4 of 5 | 2 of 5 |
Serious events with the highest arm-level rate: hypoxia (40.0%), febrile neutropenia (20.0%), gingival pain (20.0%), pyrexia (20.0%), device related infection (20.0%).
The serious adverse event figures are participant counts within each posted Part A1 group. The 1.1 mg/m^2 group lists 2 participants among 6 at risk; the 1.4 mg/m^2 group lists 1 among 6; the 1.8 mg/m^2 group lists 3 among 5; and the pharmacokinetic expansion group lists 4 among 5. These values describe participants meeting the serious-event definition, not the number of separate serious-event episodes.
The registry lists the other adverse event counts separately: 6 among 6, 6 among 6, 5 among 5, and 5 among 5 for those same groups. A person can be included in both serious and other-event summaries if they experienced events in both categories; the record does not state that these categories are mutually exclusive at participant level. The counts should not be added to create a total number of affected participants.
Deaths are listed as 0 in the 1.1 mg/m^2, 1.4 mg/m^2, and 1.8 mg/m^2 groups, and 1 in the pharmacokinetic expansion group. No causal attribution for the death is reported in the registry record. The serious-event term percentages are descriptive; their denominators are not reported in the registry record. The absence of a randomized comparator, small cohort sizes, and unreported uncertainty intervals limit interpretation beyond these observed counts.
12. What the Result Does, and Does Not, Mean
The results describe safety events recorded among pediatric participants who received eribulin mesylate in an early-phase dose-finding study. The registry provides group-specific participant counts for serious and other adverse events, a death count in each group, and a list of serious-event terms with percentages. These data are relevant to describing the safety experience in the enrolled cohorts and to the study’s stated aims of characterizing toxicity and informing dose selection.
The results do not establish that eribulin mesylate caused every reported event. The registry’s adverse-event definition explicitly allows events without a causal relationship to study treatment. Nor do the counts establish that one dose group is safer than another. Participants were not randomized among the groups, and the posted record does not provide a comparative analysis, uncertainty intervals, or an adjustment for differences between cohorts.
The study also aimed to determine a maximum tolerated dose and/or recommended Phase 2 dose, describe pharmacokinetics, and preliminarily describe antitumor effects. The safety counts alone do not answer all of those questions. The registry’s listed secondary outcome includes best overall response, but detailed response results are not included in the registry record for this analysis. A conclusion about antitumor activity cannot be drawn from the safety figures.
These findings should be understood within the population and treatment schedule studied: children with recurrent or refractory solid tumors excluding CNS tumors, treated intravenously on Days 1 and 8 of each 21-day cycle. They do not automatically generalize to other ages, diagnoses, regimens, or clinical settings. The record gives descriptive observations, not proof of benefit or lack of benefit.
13. Why This Trial Matters Statistically
Early-phase trials have a different statistical purpose from large comparative studies. This investigation was designed to study eribulin mesylate in children, characterize toxicity and pharmacokinetics, and determine a maximum tolerated dose and/or recommended Phase 2 dose. Participant-level safety counts help make the observed experience visible during dose-finding, while the separation of serious and other events distinguishes event categories with different definitions.
The design also illustrates why numerical differences between groups require context. The posted dose groups are small cohorts within a single-group study, not randomized treatment arms. A larger count in one cohort does not by itself show that its dose caused more events. Differences can be difficult to interpret when allocation is not randomized and when estimates are based on few participants. The record does not provide confidence intervals or a controlled dose comparison.
Statistically, the endpoint’s denominator and observation window matter as much as the event count. The safety population includes participants who received at least one dose, and TEAEs are defined from the first dose through 30 days after the last dose. Those definitions tell readers which participants and period the reported counts concern. They also help distinguish observed safety summaries from broader claims about all eligible children or later outcomes.
The record’s limits are informative too: it does not report missing-data methods, multiplicity procedures, or a statistical analysis plan. Recognizing what is and is not documented prevents descriptive safety findings from being mistaken for causal or comparative evidence. The appropriate statistical reading is specific to these cohorts and outcomes.
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 study was not randomized and had no control group, so the dose cohorts do not support a like-for-like comparison. Any differences in observed event counts may reflect cohort characteristics or other factors, and the registry record does not report an adjusted analysis. The posted groups are small, which makes participant counts sensitive to individual events and limits the precision of conclusions about event frequency.
The enrollment figure is 23, but the record does not establish that every enrolled participant received treatment or contributed to each listed safety group. The primary safety population is defined as participants who received at least one dose. The registry record does not provide a complete account of participant disposition, missing follow-up, or how incomplete event information was handled.
The record does not report confidence intervals for the safety counts, a statistical analysis plan, multiplicity handling, or a sample-size justification. It also does not report causal attribution for each event or the denominator and calculation method for the percentages attached to serious-event terms. The available arm description is incomplete, so details beyond the stated regimen and listed groups are not reported in the registry record.
Although the study included antitumor activity as a preliminary descriptive aim and lists best overall response as a secondary outcome, response results are not present in the registry record. The safety summaries therefore cannot answer the efficacy question. Generalizability is also limited to the pediatric population, tumor eligibility, and regimen described in the registry; applicability to other groups or treatment settings is not established by these results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: This is a Phase 1 Study of Eribulin Mesylate in Pediatric Participants With Recurrent or Refractory Solid Tumors (Excluding [Central Nervous System] CNS), Including Lymphomas — NCT02171260.
- Registry record last updated 2019-01-15; status last verified 2018-06.
- Results first posted 2018-12-24.
- 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 a completed Phase 1 study of intravenous eribulin mesylate in pediatric participants with recurrent or refractory solid tumors, including lymphomas but excluding CNS tumors. Enrollment was 23. The regimen was planned on Days 1 and 8 of each 21-day cycle, with dose-finding in Part A1 and an infant cohort planned in Part A2.
The primary endpoint counted participants with treatment-emergent and serious adverse events. Posted safety summaries list four Part A1 groups: 1.1 mg/m^2, 1.4 mg/m^2, 1.8 mg/m^2, and a pharmacokinetic expansion group. Serious-event, other-event, and death counts are descriptive. The record does not report a randomized comparator or a formal comparative analysis.
The study’s aims included dose selection, toxicity and pharmacokinetic description, and preliminary description of antitumor effects. Details on missing-data handling, multiplicity, the statistical analysis plan, and uncertainty intervals are not reported in the registry record.