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 Palonosetron for the Treatment of Nausea and Vomiting in Terminally Ill Patients (NCT00982995), a na phase 2 study. The primary outcome is To Determine the Complete Response (no Vomiting and no Need for Nausea Rescue Medication) in Terminally Ill Patients Suffering From Nausea and/or Vomiting, Treated With Palonosetron..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00982995 |
| Phase | Phase 2 |
| Condition | Nausea, Vomiting, Terminally Ill |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Michigan Rogel Cancer Center |
| Results posted | Yes |
2. Clinical Question
The study asked whether palonosetron could be associated with complete relief of nausea and vomiting in terminally ill patients experiencing nausea and/or vomiting. Its primary question focused on a complete response: no vomiting and no need for nausea rescue medication, assessed 96 hours after dosing. The study also intended to assess a partial response, defined in the record as relief to the extent that the patient wished to continue palonosetron.
Population
Terminally ill patients suffering from nausea and/or vomiting. The registry record does not report further eligibility details in the outcome description.
Intervention
Palonosetron 0.25 mg I.V. bolus.
Comparator
No separate comparator group is listed. The study is recorded as a single-group study with allocation listed as NA.
Primary question
Whether patients had no vomiting and needed no nausea rescue medication 96 hours after dosing.
These are the intended clinical questions, not demonstrated treatment effects. The registry record says the study was unable to accrue the required number of patients to analyze the primary outcome. It lists no analyses and provides no numerical primary-outcome result. Accordingly, the record does not establish how many participants met either response definition. The absence of an analyzable primary result also means that the intended response question cannot be answered from the posted results.
3. Trial Design
The registry describes a phase 2, single-group study of palonosetron. It lists no randomized allocation and no masking. The planned intervention was palonosetron 0.25 mg I.V. bolus; no control intervention or separate comparison group is listed. The study is therefore not a randomized comparison, and the record does not support a like-for-like contrast between palonosetron and another treatment or control.
The study was sponsored by University of Michigan Rogel Cancer Center. The record lists a start date of 2010-11 and primary completion in 2013-05. Its current status is TERMINATED. The reason for termination is not reported in the registry record, so the status alone does not establish why the study ended.
Enrollment is listed as 3. The primary-outcome description states that the study was unable to accrue the required number of patients to analyze that outcome. The number of patients required, the planned enrollment target, and the justification for that target are not reported in the registry record. These omissions prevent an assessment of how far recruitment fell short of the intended target or whether the target was based on a particular statistical calculation.
A single-group design can describe outcomes among people receiving an intervention, if outcome data are available. Without a separate comparison group, it cannot by itself distinguish an intervention-associated outcome from changes that might also occur without the intervention or under another treatment. That is a general limitation of the design, not a claim about what happened to these participants. In this record, the central limitation is more direct: the primary outcome was not analyzed, and no primary-response estimate is posted.
The registry lists one primary outcome and one secondary outcome, the latter concerning partial response. It lists no analyses. The statistical methods used or intended for evaluating either outcome 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.
Overall Study
| Stage | Palonosetron |
|---|---|
| STARTED | 3 |
| COMPLETED | 3 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: The study was unable to accrue the required number of patients to analyze the primary outcome.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Palonosetron |
|---|---|---|
| Participants analysed | 3 | |
| Age, Continuous | years | 65 (55 to 84) |
| Sex: Female, Male: Female | Participants | 2 |
| Sex: Female, Male: Male | Participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as To Determine the Complete Response (no Vomiting and no Need for Nausea Rescue Medication) in Terminally Ill Patients Suffering From Nausea and/or Vomiting, Treated With Palonosetron., measured over 96 hours after dosing.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is complete response 96 hours after dosing. The registry defines this as no vomiting and no need for nausea rescue medication. It combines two conditions: the participant must have had no vomiting and must not have needed rescue medication for nausea. Under this definition, satisfying only one condition would not constitute complete response.
As a general statistical concept, a binary endpoint such as this gives each assessed participant a response classification: meeting the stated definition or not meeting it. A response proportion can then describe how often the endpoint occurred in the analyzed group. Such a summary depends on having endpoint assessments and a clearly specified analysis population. The registry record provides no analyzed participant count or response count, so no response proportion can be reported from it.
A defined assessment time matters because symptoms and rescue-medication use can vary over time. The specified 96-hour timeframe identifies when the complete-response status was meant to be assessed; it does not establish that all enrolled participants had an assessment at that time. Whether assessments were made at other times, how missing assessments were handled, and how rescue medication use was collected are not reported in the registry record.
The endpoint targets a patient-experienced symptom outcome and incorporates use of additional medication into the definition. The registry gives no primary-outcome result, however, so its intended clinical meaning should not be mistaken for evidence about the frequency of response.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record supports a narrow description of study conduct: it was a phase 2, single-group study of palonosetron 0.25 mg I.V. bolus in terminally ill patients with nausea and/or vomiting; enrollment was 3; and the study is listed as terminated. The primary outcome was complete response at 96 hours after dosing. The registry explicitly says recruitment was insufficient to analyze that outcome.
No primary-outcome estimate is reported. The record therefore does not tell readers how many enrolled participants had no vomiting and needed no nausea rescue medication at the specified time. It also does not provide a confidence interval or another measure of precision for that outcome. This is not a result showing that the response rate was low, high, or absent; it is an unavailable primary analysis.
There is also no posted numerical result for the secondary partial-response outcome. Although the registry describes the intended definition, it lists zero analyses. The intended outcome definitions and the observed outcome results are distinct: the former are present, while numerical findings for response are not.
The absence of a comparator limits interpretation independently of the missing primary analysis. Even if a response count had been available, a single-group result would describe outcomes in the treated group and would not directly estimate how outcomes would differ under a comparator. General statistical reasoning cannot supply that missing contrast. No treatment effect relative to another group can be inferred from this registry record.
The safety record lists 0 serious adverse events among 3 participants at risk and 1 other adverse event among 3 participants at risk. These counts describe the recorded categories; they do not fill the gap in the efficacy outcome. The specific serious adverse event terms are not listed, and the number of deaths is not reported in the registry record. With only the posted counts, the scope and detail of the safety experience are limited.
Overall, the registry supports describing the study's design, intended endpoints, enrollment, status, and the limited adverse-event counts. It does not support a numerical conclusion about complete response or partial response, nor a comparison of palonosetron with an alternative. The reason for termination, the trial's statistical analysis plan, and the planned sample-size justification are not reported in the registry record.
9. Statistical Methods Explained
The registry labels this as a phase 2, single-group study, with allocation listed as NA and masking listed as NONE. It reports no analyses. Consequently, there is no posted primary-outcome estimate or inferential analysis to interpret. The methods below explain general statistical concepts relevant to the design and endpoint; they are not a description of analyses performed in this study.
What does a single-group design estimate?
In general, a single-group study can describe the outcomes observed among participants who receive the intervention, provided the outcome data are collected and analyzed. For a binary response, a descriptive analysis might report the number meeting the response definition and the corresponding proportion in a defined analysis population. The registry does not report those response counts or an analyzed population for the primary endpoint. It states that the required number of patients was not accrued to analyze that endpoint.
A single-group description is not the same as a comparative treatment effect. A treatment effect in a comparative sense asks how outcomes differ between intervention conditions. Without a comparator group, there is no concurrent contrast in this record. General considerations such as the natural course of symptoms, other care, or participant characteristics cannot be separated from the intervention using a single-group outcome alone. These are design considerations, not claims about the participants in this study.
What would a confidence interval add?
In general, a confidence interval communicates the uncertainty around an estimated quantity, such as a response proportion. A narrower interval indicates greater statistical precision under the assumptions of the analysis; a wider interval indicates less precision. An interval does not correct for a missing comparator, resolve bias, or create information when an outcome was not analyzed. Since the registry record gives no primary-response estimate, it also gives no confidence interval for that response.
For small groups, response estimates can be especially imprecise because each participant's outcome has substantial influence on the group summary. That is a general statistical principle. The record's enrollment of 3 and lack of an analyzed primary outcome mean readers should not treat enrollment alone as evidence about the response rate. No observed response count is available for evaluating the endpoint.
What does the endpoint definition require?
The primary endpoint combines no vomiting with no need for nausea rescue medication at 96 hours after dosing. In general, a composite definition requires the analysis to apply all stated conditions consistently. A participant who meets one component but not the other would not satisfy the stated complete-response definition. The registry does not report component-level results, endpoint ascertainment details, or a rule for handling an unavailable assessment. It is therefore not possible to assess whether each component was recorded or how the intended definition would have been applied in an analysis.
The secondary outcome concerns partial response, described as relief of nausea and vomiting to the extent that the patient wishes to continue palonosetron. This definition includes a participant preference component. The registry record does not report a numerical result, an operational assessment procedure, or an analysis method for that secondary outcome. A stated outcome definition does not itself show that the outcome was measured or analyzed.
Why do design and analysis details matter?
Statistical interpretation depends on knowing which participants were included, how outcomes were defined and measured, and what analysis was planned. The registry does not report the statistical analysis plan, an analysis population, a missing-data method, or inferential procedures for the endpoints. It lists no analyses. Readers should not assume that a particular model, testing procedure, confidence-interval method, or missing-data approach was used.
Similarly, the trial's sample-size justification is not reported in the registry record. Any sample-size calculation presented as an illustration based on an observed effect is a planning illustration, not an estimate of the trial's observed power and not evidence that the trial met its recruitment target. The registry's statement that the required number of patients was not accrued is the available explanation for why the primary outcome could not be analyzed; the target number and its rationale are not stated.
Multiplicity refers generally to the statistical consequences of analyzing multiple outcomes or making multiple comparisons. The record lists a primary and a secondary outcome but does not report a multiplicity plan or any analyses. It is not possible to determine whether or how multiplicity was addressed. This matters for interpreting inferential claims when several statistical tests are conducted, but no such claims are posted for this study.
These general concepts clarify what would be needed to interpret a completed analysis. They do not substitute for the absent outcome results or establish that any particular method was used.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary complete-response outcome and a secondary partial-response outcome. It does not report a multiplicity strategy, such as a rule for how conclusions across multiple outcomes would be controlled or prioritized beyond their primary and secondary labels. Since it lists no analyses, there are no posted tests or outcome estimates for which multiplicity handling can be assessed. The analysis plan is not reported in the registry record.
The primary-outcome description says that the study was unable to accrue the required number of patients to analyze the endpoint. The record does not give the number required or explain how the recruitment target was determined. It also does not report the number of participants with usable primary-outcome assessments, the number included in any analysis population, or a missing-data procedure. These details should not be inferred from the enrollment count.
Failure to accrue the required number is not the same as a report that enrolled participants had missing endpoint data. The registry statement concerns the ability to analyze the primary outcome because recruitment was insufficient; it does not specify whether individual assessments were missing, incomplete, or available. The data status for the secondary outcome is likewise not described in detail.
Analysis-population definitions can affect a response estimate because they determine who contributes to its denominator. The registry record does not specify an intention-to-treat population, a per-protocol population, or another analysis set. With no reported response count or denominator, no endpoint proportion can be reconstructed. The reasons for termination and any planned interim analyses are not reported in the registry record.
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 |
|---|---|---|---|
| Palonosetron | 0 | 3 | 0.0% |
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 |
|---|---|
| Palonosetron | 1 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Palonosetron |
|---|---|---|
| Thromboembolic Event | Vascular disorders | 1 of 3 |
The registry's safety grouping lists 3 participants at risk for serious adverse events and 0 participants affected. For other adverse events, it lists 3 participants at risk and 1 affected. These are counts, not comparative risks: the study has no separate control group, and the registry does not provide a like-for-like safety comparison.
The serious adverse event count of 0 does not mean that all aspects of safety were fully characterized. The registry does not list specific serious adverse event terms, and the number of deaths is not reported in the registry record. The other-adverse-event count identifies one affected participant, but the event term, severity, timing, and relationship to palonosetron are not reported in the information available for this study record.
In general, adverse-event counts describe events recorded under the study's reporting framework; they do not establish causation. A small enrolled group also gives limited information about the range of possible events. The posted counts should therefore be read as a limited description of recorded events among the listed participants, not as a comprehensive estimate of safety or as evidence for comparison with another treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a phase 2, single-group study evaluated palonosetron 0.25 mg I.V. bolus in terminally ill patients with nausea and/or vomiting. It identifies complete response at 96 hours after dosing as the primary outcome and describes partial response as a secondary outcome. It also records enrollment of 3 and states that recruitment was insufficient to analyze the primary outcome.
What the record does not establish is the frequency of complete or partial response. No response count, response proportion, confidence interval, or endpoint analysis is reported. The study's intended clinical question is therefore not answered by the posted primary-outcome information. That absence should not be interpreted as evidence that palonosetron did or did not produce a response.
The design also does not provide a comparison with another intervention or a control condition. In general, outcomes observed in one group cannot by themselves quantify how outcomes would differ under a different condition. This distinction would remain important even if an outcome estimate were available.
The safety counts are limited to the registry's reported categories: no serious adverse events among the 3 at risk and 1 other adverse event among the 3 at risk. The record does not provide the details needed to characterize the other event, and deaths are not reported. These counts do not supply the missing response analysis or a comparative safety estimate.
For interpretation, the central distinction is between a planned question and an analyzed result. The registry contains the intervention, outcome definitions, and design information needed to understand what the investigators intended to study. It also explicitly explains that the required recruitment was not achieved for analysis of the primary outcome. The record therefore supports a description of the study and its reporting limits, but not a conclusion about the treatment's effect.
13. Why This Trial Matters Statistically
This study illustrates why statistical interpretation begins with the design and the availability of analyzable outcomes, rather than with an outcome label alone. The registry defines a clinically meaningful endpoint, complete response, but also states that insufficient accrual prevented its analysis. Without an endpoint estimate, there is no basis for discussing the magnitude or precision of response in this study.
It also illustrates the difference between enrollment and an analysis population. Enrollment is listed as 3, but the record does not report a primary-outcome denominator or response count. A reader cannot assume that everyone enrolled contributed an endpoint assessment or infer the result from the enrollment figure. The registry's explicit statement about insufficient accrual is essential context for reading the outcome record.
The single-group design raises a separate interpretive issue. Descriptive outcomes in one group, when available, do not provide a direct comparison with another care condition. This distinction helps prevent a response definition from being mistaken for evidence of a comparative treatment effect. In this record, there is neither a comparator nor a reported primary-response analysis.
Finally, the sparse safety counts show why counts need context. The serious-event count is zero among the listed participants at risk, and one participant is listed with another adverse event. Those observations are useful as limited registry information, but they do not characterize all possible safety outcomes or provide a comparison. Together, these features make the record a useful example of how design, recruitment, endpoint analysis, and reporting determine what conclusions statistics can support.
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 is stated directly in the registry: the study did not accrue the required number of patients to analyze its primary outcome. The required target and the reason for it are not reported in the registry record. As a result, no primary-response estimate or measure of its precision is available.
Enrollment is 3, but the registry does not report how many participants had an endpoint assessment at 96 hours, how many were included in an analysis population, or whether any data were missing. It also gives no outcome results for the secondary partial-response measure. The record therefore does not allow readers to evaluate outcome ascertainment or reconstruct either endpoint.
The single-group design has no listed comparator, and allocation is recorded as NA. Consequently, the study cannot provide a direct treatment comparison from the design described. Masking is listed as NONE. The registry does not report further details about outcome assessment or measures intended to address potential bias.
The reason for termination, detailed statistical analysis plan, sample-size justification, handling of missing data, multiplicity approach, and any interim analyses are not reported in the registry record. The safety information is also limited: no serious events are listed among the participants at risk, one participant is listed with another adverse event, event details are not provided, and deaths are not reported. These limitations define the boundaries of what can be concluded from this record; they do not establish unreported events or outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Palonosetron for the Treatment of Nausea and Vomiting in Terminally Ill Patients — NCT00982995.
- Registry record last updated 2015-12-04; status last verified 2015-11.
- Results first posted 2014-09-15.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
This is a phase 2, single-group study of palonosetron for nausea and vomiting in terminally ill patients. The intervention is palonosetron 0.25 mg I.V. bolus. The record lists enrollment of 3, no masking, allocation NA, a start date of 2010-11, primary completion in 2013-05, and status TERMINATED.
The primary endpoint was complete response, defined as no vomiting and no need for nausea rescue medication 96 hours after dosing. Partial response was listed as a secondary outcome. The record states that the required number of patients was not accrued to analyze the primary outcome and lists no analyses. No numerical response results are reported.
Safety counts list 0 serious adverse events among 3 at risk and 1 other adverse event among 3 at risk. The reason for termination and further statistical and outcome-assessment details are not reported in the registry record.