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 Palonosetron for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) in Participants Who Have Experienced CINV(Chemotherapy-Induced Nausea and Vomiting) During the Previous Cycle of Low Emetogenic Chemotherapy (LEC) (NCT01054456), a na phase 2 study. The primary outcome is Percentage of Participants With Complete Response (CR) During Acute Period (0 to 24 Hours) After Receiving Treatment on Day 1.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01054456 |
| Phase | Phase 2 |
| Condition | Patients With Confirmed Malignant Disease to Receive Low Emetogenic Chemotherapy (LEC) or Who Experienced at Least Nausea and Vomiting During Last Cycle of LEC |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 36 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Eisai Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Participants with confirmed malignant disease who were to receive low emetogenic chemotherapy (LEC) and had experienced vomiting or at least moderate nausea during their last LEC cycle. The primary endpoint’s intent-to-treat population was defined more narrowly: participants had to receive palonosetron, receive at least one dose of the qualifying LEC agents on Day 1, and have at least one post-chemotherapy efficacy assessment.
Intervention
Palonosetron 0.25 mg/5 mL by intravenous injection 30 minutes before an LEC agent on Day 1.
Comparator
There was no control group. This was a single-group study, and the allocation field is recorded as NA. The registry record therefore does not describe a randomized, like-for-like comparison.
Primary question
What percentage of participants had a complete response during the acute period, defined as 0 to 24 hours after treatment on Day 1? Complete response meant no emetic episodes and no use of rescue medication during that period. The percentage and its 95% confidence interval are not reported in the registry record.
The study addressed prevention of chemotherapy-induced nausea and vomiting (CINV) in a group selected because they had experienced CINV during a previous cycle of LEC. Its primary endpoint was an acute-period response measure, not a comparison of palonosetron with another treatment. The registry describes the study as a multicenter, open-label evaluation and identifies it as phase 2.
The clinical question is therefore descriptive: how many participants in the defined study population met the complete-response criteria after receiving palonosetron before LEC? Without a comparator group, the study cannot directly distinguish an outcome associated with palonosetron from outcomes that might occur under another treatment or without it. The registry record also does not report the primary endpoint result, so the response rate and its precision cannot be assessed from the posted information.
3. Trial Design
This was a phase 2, multicenter, open-label, single-group study. The registry lists enrollment of 36 participants and marks the study completed. The recorded start date is 2009-10-27 and the primary completion date is 2010-12-08. Allocation is recorded as NA, and masking as none. There was no parallel control arm in the design described by the registry.
Participants received palonosetron 0.25 mg/5 mL by intravenous injection 30 minutes before receiving a low emetogenic chemotherapy agent on Day 1. The study focused on participants who had experienced vomiting or at least moderate nausea during their last cycle of LEC. This history defines the clinical context of the evaluation: the study was not described as enrolling an unselected population receiving chemotherapy.
The primary outcome was the percentage of participants with complete response during 0 to 24 hours after treatment on Day 1. The registry defines the intent-to-treat population for that endpoint as participants who received palonosetron, received at least one dose of qualifying LEC agents on Day 1, and had at least one post-chemotherapy efficacy assessment. The count in this endpoint population is not reported in the registry record; it should not be assumed to equal the enrollment figure.
The registry lists 15 outcome measures, including measures of complete response, complete control, and absence of emetic episodes across acute, delayed, and overall periods, as well as across specified intervals. It lists zero analyses. The primary endpoint’s observed percentage and confidence interval are not reported in the registry record. The protocol’s sample-size justification, detailed statistical plan, and any interim analyses are also not reported in the registry record. The study’s design supports a description of outcomes among enrolled and assessed participants, but does not supply a concurrent treatment comparison.
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: A total of 38 participants were screened, of which 02 were screen failures and 36 participants were enrolled and received the study treatment.
Overall Study
| Stage | All Participants: Palonosetron 0.25 mg/5 mL |
|---|---|
| STARTED | 36 |
| COMPLETED | 35 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | All Participants: Palonosetron 0.25 mg/5 mL |
|---|---|
| Other | 1 |
Analysis population for the primary outcome, as defined in the registry: The intent-to-treat (ITT) population included all participants who received palonosetron, received at least one dose of the qualifying LEC agents on Day 1, and had at least 1 post-chemotherapy efficacy assessment.
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 population included all participants who received study medication (palonosetron) and had at least 1 safety assessment after treatment.
| Characteristic | Unit | All Participants: Palonosetron 0.25 mg/5 mL |
|---|---|---|
| Participants analysed | 36 | |
| Age, Continuous | years | 64.6 (SD 13.82) |
| Sex: Female, Male: Female | Participants | 25 |
| Sex: Female, Male: Male | Participants | 11 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 5 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 31 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 5 |
| Race (NIH/OMB): White | Participants | 30 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Complete Response (CR) During Acute Period (0 to 24 Hours) After Receiving Treatment on Day 1, measured over From 0 to 24 hours after receiving treatment on Day 1.
| 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 percentage of participants with complete response (CR) during the acute period, defined as 0 to 24 hours after receiving treatment on Day 1. CR required both no emetic episodes and no rescue medication during that period. It is a binary participant-level outcome: each participant either meets the stated criteria for the period or does not.
The registry gives a detailed definition of an emetic episode. It includes one occurrence of vomiting; closely succeeding occurrences not interrupted by at least 1 minute of relaxation; unproductive emesis, or retches, occurring within a unique 5-minute period; and a short retching episode combined with vomiting without the specified relaxation period. Participants who completed the diary for the period and had no emetic episodes and no rescue medication were considered to have achieved CR.
This endpoint combines vomiting-related events with rescue-medication use. That can make the outcome more informative than counting emetic episodes alone, because a participant with no episode but who used rescue medication does not meet the CR definition. It is also a time-bounded measure: it describes the acute period, rather than the full later course of symptoms.
The endpoint is expressed as a percentage, with a 95% confidence interval specified as its dispersion measure. The observed percentage and interval are not reported in the registry record. The record also does not report the number of participants contributing to this endpoint, beyond defining the intent-to-treat population.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group assessment of palonosetron before LEC in participants who had experienced CINV during the previous cycle. It specifies a primary endpoint and its definition, but does not report the endpoint’s observed percentage or 95% confidence interval. As a result, the posted record does not establish how many participants achieved complete response, nor how precisely that percentage was estimated.
In general, a percentage for a binary endpoint describes the proportion of participants meeting the outcome definition in the analysis population. A confidence interval expresses uncertainty around an estimate; it reflects the range of values compatible with the data and the statistical method, rather than a guarantee that the true value lies within the interval. Here, the interval is named in the endpoint description, but its limits are not reported in the registry record. No numerical interpretation of the response rate or its precision is possible without those results.
The single-arm design is central to interpretation. Outcomes among participants who received palonosetron can be described, but there is no concurrent comparison group to show what would have happened under an alternative treatment or another care strategy. Differences from prior experience would not substitute for a randomized control, because participant characteristics, care, and assessment conditions may differ. The open-label design also means masking was absent; the registry does not report details about who assessed outcomes or how any potential influence of awareness was addressed.
The primary outcome depends on both diary information and rescue-medication use. A participant had to complete the diary for the period and have neither an emetic episode nor rescue-medication use to count as a responder. The record does not provide counts for diary completion, endpoint evaluability, or missing observations. Consequently, it is not possible to assess how incomplete diary data affected the reported outcome, or whether missing outcomes were handled in any particular way.
The registry lists several additional outcome measures covering complete response, complete control, and absence of emetic episodes over different intervals. Such measures describe distinct aspects or periods of CINV; they should not be treated as interchangeable with the primary acute-period CR endpoint. The record lists zero analyses and does not report results for those secondary outcomes. It also does not report a multiplicity plan, so the approach to interpreting several outcomes or time periods is unknown.
The adverse-event record lists 36 participants at risk for serious adverse events and 3 affected, along with 36 at risk for other adverse events and 13 affected. It records 0 deaths. These are safety counts in the registry record, not comparative estimates. The recorded serious-event terms each have a listed percentage of 2.8%; the record does not establish whether these terms represent distinct participants or whether any event was caused by treatment. In a study without a control group, event counts cannot by themselves estimate how risk differs from another treatment.
Overall, the registry supports a description of the study question, design, endpoint definitions, and posted safety counts. It does not provide the efficacy result needed to quantify the acute complete-response rate, and the design does not permit a direct randomized comparison.
9. Statistical Methods Explained
The statistical information in the registry record is limited. It identifies the primary endpoint as a percentage and names a 95% confidence interval as the dispersion measure. It does not report the endpoint estimate, interval limits, a detailed analysis plan, or the sample-size justification. The concepts below are general explanations of the methods relevant to the stated endpoint and design, not claims about analyses that were performed in this study.
What a percentage measures
For a binary outcome, a percentage represents the share of participants who meet a prespecified definition. For this endpoint, the definition is complete response during 0 to 24 hours: no emetic episodes and no rescue medication. The percentage depends on both the number meeting that definition and the number included in the analysis. The registry defines the intent-to-treat population, but does not report its size or the number meeting the endpoint.
A percentage is easy to interpret as a response frequency in the studied group. It is not, by itself, evidence that treatment caused the outcome. That distinction is particularly important in a single-group study, where there is no concurrent comparison group to account for outcomes that might occur under other conditions.
What a confidence interval conveys
In general, a confidence interval describes statistical uncertainty around an estimate. A narrower interval indicates greater precision under the method used, while a wider interval indicates less precision. The interval does not measure all sources of uncertainty: it does not correct for bias from selection, measurement, missing data, or lack of a comparator. Although the registry names a 95% confidence interval for the primary endpoint, neither the interval nor the percentage is reported in the registry record.
For a proportion, the particular interval method can matter, especially when the sample is limited or the observed proportion lies near the boundaries. The registry record does not identify which method was used. No method should therefore be attributed to the study based only on the endpoint description.
Single-group estimation and comparison
A single-group design can estimate the frequency of an outcome among participants meeting the study’s eligibility and analysis criteria. It cannot directly estimate a treatment contrast because it has no control arm. A comparison with an external or historical rate would require a suitable reference and assumptions about comparability; the registry record does not describe such a comparison. Allocation is recorded as NA, consistent with the listed single-group model.
Open-label conduct means masking was absent. In general, awareness of treatment can affect reporting, use of rescue medication, diary completion, or assessment. The endpoint includes both symptom events and rescue-medication use, so consistent outcome ascertainment is important. The registry does not report procedures designed to limit assessment or reporting effects.
Outcome definitions and time windows
The primary endpoint is restricted to the acute period, 0 to 24 hours after treatment on Day 1. The additional outcomes listed in the registry use other time windows and definitions, including complete control and no emetic episodes. An endpoint based only on the absence of emetic episodes differs from CR, because CR also requires no rescue medication. Complete control is separately named, but its detailed definition is not included in the registry record. Results across different intervals answer different descriptive questions and should not be combined as if they were one outcome.
Planning calculations
The registry describes a planning illustration based on an observed effect, not the trial’s own design. Its stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For non-time-to-event outcomes, the resulting quantity is participants per arm for a specified power level. These are planning illustrations, not observed power, and they do not change the fact that this trial was single-arm. The registry record does not report a protocol-based sample-size justification. No planning sample-size result or effect estimate is reported in the registry record, so none can be interpreted.
Dispersion and over-dispersion
In general, over-dispersion means that observed variation is greater than expected under a statistical model’s assumptions. It is often considered for count outcomes or clustered data. The primary endpoint is a participant-level binary percentage, and the registry record does not report an over-dispersion assessment or a model that addresses it. It would be inappropriate to infer that such an assessment was performed.
More broadly, the record does not report an inferential model, covariate adjustment, interim analyses, or a detailed plan for handling multiple outcomes. The absence of those details prevents reconstruction of the statistical analysis. The stated endpoint definition and confidence-interval label describe what the study intended to summarize, but do not reveal how the numerical result was calculated.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 15 outcome measures, including the primary acute-period complete-response endpoint and secondary measures for complete response, complete control, and absence of emetic episodes over several time windows. When several endpoints or time periods are examined, the chance of at least one apparently unusual result can increase if each is interpreted independently. In general, multiplicity methods may control that risk or distinguish a prespecified primary question from exploratory outcomes.
The registry record does not report a multiplicity plan, the order in which outcomes were tested, or adjustments to statistical significance levels. It lists zero analyses, and results for the named outcomes are not reported in the registry record. Therefore, no claim can be made about how multiple outcomes were handled or whether any secondary result was considered confirmatory.
The record defines the intent-to-treat population for the primary endpoint, but does not report its size. It also does not report the number of participants with missing diaries, incomplete assessments, or unavailable outcome data, nor does it describe imputation, exclusion, or sensitivity analyses. Since the CR definition requires a completed diary for a given period, missingness could affect who is classifiable as a responder. The registry record does not state how unclassifiable participants were handled.
The registry’s population definition requires receipt of palonosetron, at least one qualifying LEC dose on Day 1, and at least one post-chemotherapy efficacy assessment. This is the stated analysis population; it should not be assumed that all enrolled participants satisfied those criteria. The record does not report a separate safety analysis population definition beyond the adverse-event counts at risk. These omissions limit assessment of analysis-population selection and missing-data sensitivity.
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 |
|---|---|---|---|
| All Participants: Palonosetron 0.25 mg/5 mL | 3 | 36 | 8.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| All Participants: Palonosetron 0.25 mg/5 mL | 0 of 36 |
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 |
|---|---|
| All Participants: Palonosetron 0.25 mg/5 mL | 13 of 36 |
Most frequent serious adverse event terms
| Event term | Organ system | All Participants: Palonosetron 0.25 mg/5 mL |
|---|---|---|
| Dysphagia | Gastrointestinal disorders | 1 of 36 |
| Melaena | Gastrointestinal disorders | 1 of 36 |
| Pneumonia | Infections and infestations | 1 of 36 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 1 of 36 |
Most frequent non-serious adverse event terms
| Event term | Organ system | All Participants: Palonosetron 0.25 mg/5 mL |
|---|---|---|
| Headache | Nervous system disorders | 2 of 36 |
| Decreased appetite | Metabolism and nutrition disorders | 2 of 36 |
| Pyrexia | General disorders | 2 of 36 |
| Fatigue | General disorders | 2 of 36 |
| Respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | 1 of 36 |
| Dehydration | Metabolism and nutrition disorders | 1 of 36 |
| Chills | General disorders | 1 of 36 |
| Asthenia | General disorders | 1 of 36 |
| Nausea | Gastrointestinal disorders | 1 of 36 |
| Haemorrhoidal haemorrhage | Gastrointestinal disorders | 1 of 36 |
Serious events with the highest arm-level rate: dysphagia (2.8%), melaena (2.8%), pneumonia (2.8%), dyspnoea (2.8%).
The registry lists 36 participants at risk for serious adverse events and 3 affected. For other adverse events, it lists 36 at risk and 13 affected. The recorded number of deaths is 0. These counts describe the safety information posted for the palonosetron group; they are not comparative estimates against a control group.
The serious-event terms listed are dysphagia, melaena, pneumonia, and dyspnoea, each with a recorded percentage of 2.8%. The registry record does not specify whether those terms occurred in distinct participants, whether a participant could contribute more than one term, or the timing and duration of the events. It also does not report a causal assessment for individual events. The listed terms should therefore be read as reported event categories, not as evidence that treatment caused them.
In general, adverse-event counts are affected by the number of participants observed, the observation period, event definitions, and reporting practices. With no control group, the counts cannot show whether event frequency was higher or lower than it would have been under another treatment. The registry record does not report further detail about event severity, exposure duration, or withdrawals due to adverse events.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase 2, open-label, single-arm study evaluated palonosetron before LEC in participants who had experienced CINV during the previous cycle. It specifies a clinically interpretable primary endpoint: complete response during 0 to 24 hours after treatment, requiring no emetic episodes and no rescue medication. It also reports safety counts for serious and other adverse events and records 0 deaths.
Those details do not provide the primary efficacy result. The percentage with complete response and its 95% confidence interval are not reported in the registry record. Without them, the observed response frequency and its precision cannot be described. The record also does not report results for the secondary outcomes, so their findings cannot be summarized.
Even if an outcome percentage were available, a single-group study would describe what occurred among participants who received palonosetron, not the difference between palonosetron and a comparator. There was no control arm, and allocation is recorded as NA. The design therefore cannot establish a treatment effect through a randomized comparison. Open-label conduct and the endpoint’s dependence on diary reporting and rescue-medication use are additional considerations when interpreting an outcome estimate.
The safety counts are similarly descriptive. They do not show comparative risk, and the registry record does not establish that an event was caused by treatment. The listed serious-event terms and the count of participants affected should be understood within the study’s reported group and available detail.
In general, estimates from a study apply most directly to the population and conditions actually studied. Here, participants had a history of CINV during a prior LEC cycle, and the primary population had to meet the stated treatment and assessment criteria. The registry does not report the count in that endpoint population or detailed missing-data handling. The results cannot be extended beyond the information and design recorded.
13. Why This Trial Matters Statistically
This trial illustrates an important distinction between describing an outcome and estimating a treatment effect. A single-group study can ask how frequently a defined outcome occurred in a selected group receiving an intervention. Without a concurrent comparator, it cannot directly estimate how that frequency differs from outcomes under another strategy. That distinction is essential when interpreting percentages from early-phase clinical evaluations.
The study also uses a composite operational definition for complete response: no emetic episodes and no rescue medication within a specified acute period. This definition makes clear that response is not simply the absence of vomiting. It depends on both event reporting and medication use, as well as diary completion. Clear outcome definitions are necessary for reproducible measurement, while information about missing observations is needed to understand how fully the definition could be applied.
Time windows matter too. The registry lists outcomes for acute, delayed, and overall periods, and for several specific intervals. A result over one period answers a different question from a result over another. Keeping those estimands distinct helps avoid treating all measures of nausea and vomiting as equivalent.
Finally, the record illustrates the limits of a registry entry when outcomes and analysis details are absent. The endpoint and confidence-interval type are stated, but the primary estimate, interval, analysis population size, and multiplicity approach are not reported. Statistical interpretation requires both a defined estimand and its numerical result, along with enough methodological detail to judge precision and potential bias. The posted record supplies the design context but not the efficacy result needed for that assessment.
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 primary limitation for statistical interpretation is that the registry record does not report the primary endpoint percentage or its 95% confidence interval. It also does not report results for the secondary outcomes. As a result, efficacy estimates and their precision cannot be evaluated from the posted record.
The study was single-arm and open-label, with no comparator group and no masking. This design permits descriptive reporting of outcomes among participants who received palonosetron, but does not provide a randomized comparison. Awareness of treatment may also be relevant for outcomes based partly on diary entries and rescue-medication use; the registry record does not describe procedures addressing this issue.
Enrollment is listed as 36, while the number in the primary endpoint’s intent-to-treat population is not reported in the registry record. The record does not give counts for endpoint completion or missing diary data, nor does it describe missing-data methods or sensitivity analyses. It is therefore not possible to assess the effect of incomplete outcome information or determine whether the analyzed population differed from those enrolled.
The registry lists 15 outcome measures but does not report a multiplicity strategy, detailed statistical plan, interim analyses, or the protocol’s sample-size justification. It lists zero analyses. These gaps prevent assessment of the analysis choices behind any numerical findings. The safety information is also limited to counts and listed terms, without a control group or reported causal assessment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Palonosetron for the Prevention of Chemotherapy-Induced Nausea and Vomiting (CINV) in Participants Who Have Experienced CINV(Chemotherapy-Induced Nausea and Vomiting) During the Previous Cycle of Low Emetogenic Chemotherapy (LEC) — NCT01054456.
- Registry record last updated 2020-12-23; status last verified 2011-01.
- Results first posted 2020-12-23.
- 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
NCT01054456 is a completed phase 2, multicenter, open-label, single-arm evaluation of palonosetron before low emetogenic chemotherapy. The registry lists enrollment of 36 participants. Palonosetron 0.25 mg/5 mL was given intravenously 30 minutes before an LEC agent on Day 1.
The primary endpoint was the percentage with complete response during 0 to 24 hours, defined as no emetic episodes and no rescue medication. The record specifies a 95% confidence interval, but the primary percentage and interval are not reported in the registry record. It lists 15 outcome measures and zero analyses.
The safety record lists 3 participants affected by serious adverse events among 36 at risk, 13 affected by other adverse events among 36 at risk, and 0 deaths. The design has no concurrent control group, so efficacy and safety information is descriptive rather than comparative.