Multi-day Doses in Prevention of Nausea and Emesis: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Multi-day Doses in Prevention of Nausea and Emesis (NCT00600353), a na phase 2 study. The primary outcome is Overall Emetic Response: Acute.

2. Clinical Question

3. Trial Design

The registry identifies this as a Phase 2 pilot study with a single-group model. Allocation is listed as NA, and masking is listed as none. The study status is completed. The recorded start is 2007-10, and the primary completion is 2010-01. The sponsor is the University of Kansas Medical Center. Enrollment is listed as 20.

The intended population was patients with multiple myeloma and lymphoma undergoing autologous HSCT. The brief summary describes assessment of emetic responses during multi-day antiemetic treatment. It names palonosetron and aprepitant with low-dose dexamethasone, with or without prochlorperazine. The intervention list names palonosetron, aprepitant and dexamethasone. A detailed arm description is present for subjects with multiple myeloma; it describes a conditioning regimen over a 7 day period that includes melphalan and the named antiemetic medications. A corresponding detailed lymphoma regimen is not reported in the registry record.

Because the study is recorded as single-group and not randomized, the design does not support a randomized comparison with a control arm. The record lists adverse-event groups for subjects with multiple myeloma and patients with lymphoma, but does not provide primary-outcome results for these groups. It also lists a secondary outcome concerning the impact of nausea and vomiting on quality of life during autologous HSCT. Details of its measurement and results are not reported in the registry record.

The registry lists five outcome measures and zero analyses. It does not report a statistical analysis plan, a sample-size justification, interim analyses, or the analysis populations. The enrollment value is a registry characteristic; it should not be interpreted as a reported primary-outcome denominator or as evidence that every enrolled participant contributed to every outcome.

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: Emetic responses were assessed by daily patient diaries and the Multinational Association for Supportive Care in Cancer Antiemetic Tool (MAT). Nausea was measured using a Nausea Visual Score (NVS) of 0 to 10 with score of 0 having no nausea. A "modified" Osoba module was used to assess quality of life (QOL).

Overall Study

Reasons for not completing this period:

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: 2 patients excluded from analysis

6. Primary Endpoint

The registry lists the primary outcome as Overall Emetic Response: Acute, measured over 24 hours after chemotherapy.

The primary endpoint is titled “Overall Emetic Response: Acute.” Its timeframe is 24 hours after chemotherapy, and its unit is participants. The endpoint description defines several response categories using emetic episodes, rescue medication use and nausea visual scale (NVS) values. Complete Control (CC) requires no emetic episode in 24 hours, no rescue medications and an NVS of ≤ 2.5. Complete Emetic Response (CR) requires zero emetic episodes and no rescue medications. Major Emetic Response (MR) is defined as zero to two emetic episodes within 24 hours, with or without rescue medications. Minor Emetic Response (mR) is three to five emetic episodes within 24 hours, with or without rescue medications. Failure is defined as more than five emetic episodes within 24 hours. No Significant Nausea (NSN) is defined as a maximum NVS of ≤ 5.

These definitions combine observable vomiting or emetic episodes with medication use and, for some categories, reported nausea. In general, composite or categorical endpoints can describe different aspects of a clinical response that a single event count would not capture. Their interpretation depends on clear definitions and on knowing how many participants were assessed in each category.

The registry record does not report observed counts for these response categories, the number assessed, missing outcome data, or an estimate of precision. Thus the endpoint definitions establish what the study intended to classify, but do not establish the response distribution or the degree of uncertainty around it.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

There is little inferential analysis available to interpret because the registry lists zero analyses and does not report primary-outcome results. The methods below explain how the stated design and endpoint definitions affect interpretation, and distinguish general statistical principles from methods documented for this study.

What does a single-group design estimate?

The study is recorded as single-group, with allocation listed as NA and masking as none. In general, a single-group study can describe outcomes observed among participants receiving a study regimen. Without a comparator group, it cannot directly estimate how those outcomes would have differed under another regimen or under no study regimen. Other influences on symptoms and outcomes may be present, and the record does not provide a design-based method for separating them from the regimen’s effects.

The registry includes separate group labels for multiple myeloma and lymphoma in the adverse-event information. That does not establish a randomized comparison between the groups. The conditions differ by definition, and the record does not report a planned comparative analysis. The intervention details for lymphoma are not reported in the registry record, so the extent to which treatment was the same across the condition groups is also unclear.

How should response categories be understood?

The primary endpoint is a set of defined acute response categories measured 24 hours after chemotherapy. The definitions use emetic episode counts, rescue medication and NVS thresholds. In general, a categorical endpoint converts participant-level observations into groups with distinct meanings. Reporting the count in each category, along with the number assessed, would show the observed distribution. The registry record gives the category rules but not those counts or denominators.

Some categories use overlapping criteria: for example, the description of MR and mR specifies episode ranges and allows responses with or without rescue medication, while CR requires no episodes and no rescue medication. The record does not explain how overlapping category definitions were handled in the reported outcome, or provide results to clarify their use. The endpoint definitions should therefore be read as the registry’s stated criteria, not as evidence that the categories were mutually exclusive in analysis.

What would a confidence interval mean?

In general, a confidence interval describes the statistical uncertainty around an estimate, such as an observed response proportion. A narrower interval indicates greater precision under the assumptions of the analysis; a wider interval indicates less precision. An interval does not remove bias from study design, and it does not create a randomized comparison where none exists. No primary-outcome estimate or confidence interval is reported in the registry record, so precision for the acute response endpoint cannot be assessed.

For counts of participants with an event, the denominator matters: a count is interpretable only alongside the number of participants evaluated and a clear definition of the analysis population. The registry lists enrollment as 20, but that is not identified as the primary-endpoint denominator. The number assessed for the endpoint and the handling of missing outcome measurements are not reported in the registry record.

What do the planning illustrations represent?

The registry’s sample-size assumptions describe a planning illustration computed from the observed effect, not the trial’s own design. The 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. These assumptions describe hypothetical planning, not the single-group pilot design recorded for this study.

Any participant-per-arm values in such planning illustrations are not observed participant counts, and they are not observed power. They depend on the stated assumptions and effect size used for the illustration. The record does not report the trial’s own sample-size justification, and the registry information does not provide primary-outcome estimates with which to assess their precision. The two-arm planning assumptions should not be mistaken for evidence that this trial randomized participants between two arms.

In general, power is a design-stage probability of detecting a specified effect under a stated statistical model and assumptions. It is not a measure of whether a result is true, and calculating power after observing data does not replace reporting the estimate and its uncertainty. No trial-specific power calculation or statistical plan is reported in the registry record.

Why can over-dispersion matter?

In general, count outcomes can vary more widely across participants than a simple probability model assumes. This is called over-dispersion. If present, it can make standard errors too small under a model that assumes less variability, leading to confidence intervals that are too narrow and tests that appear more decisive than warranted. A model that allows extra variability may be appropriate for some count outcomes. The primary endpoint is described as participant counts in response categories; the registry does not report a count-model analysis or a method addressing over-dispersion.

Overall, the record allows readers to understand the study’s broad structure, endpoint definitions and some safety counts. It does not provide enough analysis detail or primary-outcome data to reconstruct an inferential analysis. Statistical methods beyond the planning assumptions described in the registry are not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The record lists five outcome measures and zero analyses. It identifies acute overall emetic response as the primary outcome and lists a secondary outcome concerning the impact of nausea and vomiting on quality of life among patients undergoing autologous HSCT. The remaining outcome details and results are not reported in the registry record.

Multiplicity arises when several outcomes, time points, groups or statistical tests are examined. In general, the more inferential questions examined, the greater the chance that at least one result may appear notable by chance if the tests are considered without adjustment. A prespecified hierarchy or multiplicity adjustment can address some of this issue, but the approach depends on the analysis plan. A multiplicity strategy is not reported in the registry record. Because no analyses are listed, there are no posted test results for which an adjustment can be evaluated.

The registry record also does not report missing-data counts, reasons for missing measurements, imputation methods, or sensitivity analyses. In general, missing outcome data can affect interpretation when participants with and without measurements differ in ways related to the outcome. Without the number assessed and the approach to missingness, the completeness and representativeness of the primary endpoint cannot be judged.

Analysis populations are not reported in the registry record. It is not stated whether outcome summaries were intended for all enrolled participants, participants who received treatment, or another defined group. The enrollment entry should not be assumed to be the denominator for every outcome. The record also does not report a statistical analysis plan, interim analyses, or a sample-size justification for the trial.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent non-serious adverse event terms

The adverse-event information is reported by condition group. For subjects with multiple myeloma, the registry reports 0 serious adverse events among 9 at risk and 3 other adverse events among 9 at risk. For patients with lymphoma, it reports 0 serious adverse events among 9 at risk and 2 other adverse events among 9 at risk. These are separate group entries and should not be added together or treated as a comparative effect estimate.

The record does not list serious adverse-event terms. It reports no serious adverse events in the posted group counts, but this is a description of the registry entries and the recorded at-risk groups; it does not establish that serious events could not occur. The number of participants exposed for the full period of observation and the duration of safety follow-up are not reported in the registry record. Deaths are also not reported in the registry record.

“Other” adverse events are reported separately from serious adverse events. The record does not provide event descriptions, severity grades, attribution to treatment, or timing. These counts therefore do not show whether events were related to a study medication or how clinically important they were. Because the study is not a randomized comparison, the safety figures should be read descriptively rather than as evidence of differences between treatments.

12. What the Result Does, and Does Not, Mean

This registry record establishes that a completed Phase 2 pilot study was designed to assess acute emetic response during antiemetic treatment in patients with multiple myeloma and lymphoma undergoing autologous HSCT. It defines the primary endpoint’s response categories and timeframe, and provides some adverse-event counts. It does not report the primary response results or statistical analyses.

That distinction matters: an endpoint definition describes how an outcome is classified, while an outcome result describes what was observed. Without the response counts, denominators and uncertainty estimates, the record cannot establish the frequency of complete control, complete emetic response, major or minor response, failure, or no significant nausea. It also cannot show how precise such estimates would be.

The design is single-group, with no randomized comparator described. Consequently, even if response counts were available, they would describe observations within the study rather than establish a treatment effect relative to a control regimen. The posted adverse-event counts likewise do not provide a randomized comparison. The record does not report enough information to attribute an outcome to a particular medication or to compare the condition groups as if they were exchangeable.

The study’s enrollment entry and adverse-event at-risk counts are not interchangeable. The record does not state the primary endpoint denominator, the analyzed population, or how missing data were handled. These uncertainties limit interpretation independently of the absence of outcome results.

The registry information is useful for understanding the question the pilot study addressed, the design that was recorded, and the definitions intended for acute response. It does not establish that the treatment was effective or ineffective, nor does it determine the comparative safety of the medications. Those conclusions cannot be drawn from the results reported in the registry record.

13. Why This Trial Matters Statistically

This study raises several statistical issues that are common in clinical research. First, the design determines what kind of claim an analysis can support. A single-group study can describe outcomes in its participants, but a treatment comparison requires a suitable comparator and a design that supports interpreting differences. Here, the registry records a single-group model, allocation as NA and masking as none, so it does not describe a randomized control comparison.

Second, the endpoint combines multiple dimensions of emetic response. Its categories refer to episode counts, rescue medication and nausea scale thresholds. Such definitions can be clinically informative, but interpretation depends on knowing how categories were assigned, how many participants were assessed and how missing measurements were treated. The registry provides the definitions but not the observed category counts or the analysis details.

Third, denominators need to match the outcome being discussed. The enrollment entry is not identified as the primary-endpoint denominator, while adverse-event entries give their own at-risk counts by condition group. Treating these as interchangeable would obscure what was actually observed. The record does not explain the relationship between those counts.

Finally, planning assumptions should not be confused with the trial design or with observed evidence. The registry describes a two-arm planning illustration based on a fraction of an observed effect, while the study itself is recorded as single-group. Such illustrations are hypothetical planning calculations, not observed power or trial results.

These points make transparent reporting especially important: outcome counts, denominators, analysis populations, uncertainty estimates and missing-data methods would be needed to assess the primary endpoint. Their absence leaves the study’s intended question clearer than its answer.

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.

Several trial-specific limitations follow from the registry information. The study is recorded as single-group, with allocation listed as NA and masking as none. The registry does not describe a randomized comparator, so it cannot support a direct randomized estimate of differences between the study regimen and an alternative. The record also does not describe masking procedures beyond listing masking as none.

The primary endpoint is defined in detail, but its observed category counts, denominator and statistical results are not reported in the registry record. The number of participants assessed, completeness of outcome measurement and handling of missing values are also not reported. Therefore, neither the response distribution nor its precision can be evaluated from the posted information.

The registry describes treatment details for subjects with multiple myeloma, but a corresponding regimen description for patients with lymphoma is not reported in the registry record. The brief summary mentions possible prochlorperazine use, while the listed interventions name palonosetron, aprepitant and dexamethasone. The record does not clarify how this possible additional medication was used or whether treatment differed by group.

The adverse-event entries provide group-level counts but not event terms, severity details, attribution, timing or follow-up duration. The relationship between the enrollment entry and the at-risk counts is not explained. The record also lists a quality-of-life secondary outcome without reported results or measurement details.

A statistical analysis plan, analysis populations, missing-data methods, multiplicity strategy, interim analyses and trial-specific sample-size justification are not reported in the registry record. These omissions prevent a fuller assessment of how the study’s outcomes were analyzed and how robust the intended conclusions would be.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Multi-day Doses in Prevention of Nausea and Emesis — NCT00600353.
• Registry record last updated 2017-03-09; status last verified 2017-01.
• Results first posted 2017-03-09.
• 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.

18. Record Summary

The registry describes NCT00600353, “Multi-day Doses in Prevention of Nausea and Emesis,” as a completed Phase 2 pilot study sponsored by the University of Kansas Medical Center. It records a single-group model, allocation as NA, no masking, and enrollment of 20. The recorded start is 2007-10, with primary completion in 2010-01.

The study concerns patients with multiple myeloma and lymphoma undergoing autologous HSCT. Its primary outcome is acute overall emetic response 24 hours after chemotherapy, classified using episode counts, rescue medication and nausea visual scale criteria. The registry lists five outcome measures and zero analyses. It does not report primary-outcome results, endpoint denominators, or the trial’s statistical analysis plan.

Adverse-event counts are reported separately by condition group. The record also lists a secondary quality-of-life outcome. Interpretation is limited by the single-group design and the absence of primary-outcome analyses and key analysis details.