Palonosetron, Ondansetron, and Dexamethasone for Delayed Nausea and Vomiting in Autologous Transplant Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Palonosetron, Ondansetron, and Dexamethasone for Delayed Nausea and Vomiting in Autologous Transplant Patients (NCT01370408), a na phase 2 study. The primary outcome is Complete Response Rate for Delayed Chemotherapy Induced Nausea & Vomiting.

2. Clinical Question

3. Trial Design

The registry classifies NCT01370408 as a phase 2, single-group study. Allocation is recorded as NA, and masking as NONE. The described group received a regimen involving palonosetron, ondansetron and dexamethasone during high-dose chemotherapy for autologous stem cell transplantation. Because no separate control group is described, the design is not a randomized comparison between treatments.

The brief summary describes intravenous ondansetron 8mg and dexamethasone 10mg 30 minutes before myeloablative preparative chemotherapy, continuing through the last chemotherapy day. On that final day, the summary describes palonosetron 0.25mg and dexamethasone 10mg intravenously 30 minutes before chemotherapy. If the chemotherapy regimen consists of only 1 day of chemotherapy, the summary says that only palonosetron and dexamethasone are administered before chemotherapy. Oral dexamethasone 8mg once daily is described for 2 days after chemotherapy. The arm description gives a less complete account of the schedule; the registry record does not explain this difference.

The listed enrollment is 85. The study status is COMPLETED, with a start date of 2012-02 and a primary completion date of 2016-03. These record dates describe the trial timeline, not the timing of individual participants' chemotherapy or follow-up.

The primary outcome is a delayed chemotherapy-induced nausea and vomiting complete response, with a stated 120 hours timeframe. The record also lists outcomes covering acute-phase complete remission, complete remission over the overall chemotherapy period, nausea and vomiting control, emetic episodes, and events or treatment failure during the first 24 hours. The numerical results for these outcomes are not reported in the registry record. The statistical analysis plan, including any prespecified analysis methods, is 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

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.

6. Primary Endpoint

The registry lists the primary outcome as Complete Response Rate for Delayed Chemotherapy Induced Nausea & Vomiting, measured over 120 hours.

The primary endpoint is the complete response rate for delayed chemotherapy-induced nausea and vomiting. A participant meets the endpoint definition by having no emetic episode and using no rescue medication during the 24-120 hour period after chemotherapy. The registry lists the timeframe as 120 hours.

This endpoint combines two clinically distinct observations: whether vomiting or another emetic episode occurred, and whether rescue medication was used. Requiring both conditions to be met makes the endpoint a summary of control over the delayed period rather than a measure of either symptom occurrence or rescue treatment alone. A participant who experiences an emetic episode or uses rescue medication does not meet the stated complete-response definition.

Rates are useful for binary outcomes because they describe the share of participants who meet a clearly defined condition. Interpreting such a rate requires knowing the number meeting the definition and the denominator assessed. The registry record does not report the primary endpoint result, its denominator, or an uncertainty estimate. It therefore does not permit a numerical assessment of how frequently complete response occurred or how precisely that rate was estimated.

The record lists several related outcomes, including complete control and emetic episodes. Those outcomes may capture different aspects of nausea and vomiting, but their definitions and numerical results are not reported in the registry record. They should not be treated as interchangeable with the primary complete-response endpoint.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation begins with the design and the endpoint definition. This study is recorded as single-group, with allocation marked NA and masking marked NONE. The primary endpoint is binary at the participant level: a participant either meets the complete-response definition for the delayed period or does not. The definition requires both no emetic episode and no rescue medication use during 24-120 hours after chemotherapy. The registry record does not report the observed count, proportion, denominator, or an analysis of this endpoint.

What does a binary endpoint measure?

In general, a binary endpoint classifies each participant into one of two outcome categories. A rate or proportion describes the number meeting the endpoint relative to the number assessed. That summary is straightforward to communicate, but it depends on a clear definition and a known denominator. If participants have missing outcome data, decisions about whether they are included, excluded or classified in another way can change the estimate. The registry record does not describe missing-data handling or the endpoint analysis population, so these features cannot be assessed for this study.

What can a single-group design estimate?

In general, a single-group study can describe outcomes among the participants observed under the study regimen. It lacks a concurrent control group, so it cannot separate the regimen's association with an outcome from other explanations, such as participant characteristics, chemotherapy context, changes over time or other care. A randomized comparison is designed to support a treatment contrast by assigning participants to different groups; the registry record does not describe such an allocation. Results from this single group, even if available, would therefore answer a descriptive question rather than provide a randomized estimate of a difference between treatments.

The lack of masking also matters in general, particularly for outcomes with subjective components. The primary endpoint includes an emetic episode and use of rescue medication. Knowledge of treatment assignment can sometimes influence care decisions or outcome reporting, although the registry record does not describe how either outcome was assessed in this study. No claim about the direction or size of any such influence can be made from the record.

How should precision be considered?

In general, an estimate from a study is more informative when its uncertainty is also reported. A confidence interval expresses a range of values compatible with the observed data under specified assumptions; it is not a guarantee that the true value lies within that range. For a binary outcome, uncertainty depends in part on the number of participants with usable endpoint data and on the observed outcome distribution. The registry record does not report a confidence interval, standard error, or equivalent measure for the primary endpoint. Thus, its precision cannot be evaluated from the posted results.

The record's enrollment of 85 does not substitute for an analysis denominator or an uncertainty estimate. Participants enrolled may differ from participants included in a particular outcome analysis if data are unavailable or eligibility for that analysis differs. The registry record does not report whether any participants were excluded from the primary analysis, how many were assessed, or how missing outcome observations were handled.

What do the planning illustrations represent?

The sample-size assumptions describe planning illustrations based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated planning approach considers detecting a fraction of the observed effect. These assumptions describe hypothetical planning calculations, not the design actually recorded for this study. The study is single-group, and the observed primary effect is not reported in the registry record. Consequently, those illustrations should not be read as the number of participants actually needed for this trial, as evidence of its design justification, or as observed power. Sample-size illustrations are planning tools; they do not show how likely the study was to produce a statistically significant result after the data were collected.

In general, a normal approximation replaces a more detailed probability calculation with an approximation based on a normal distribution. Its suitability depends on the outcome and data structure. The registry record does not report the full calculation details or explain how the assumptions were selected. The record also does not report a protocol-based sample-size justification. No further rationale should be inferred from the planning assumptions alone.

What analysis details are unavailable?

The registry record lists no analysis entries. It does not report a statistical test for the primary endpoint, an estimation method, an adjustment for covariates, or a method for accounting for differences in chemotherapy regimens. It does not report an interim analysis plan or a prespecified stopping rule. These details are not necessary to restate the endpoint definition, but they are needed to understand how a numerical result was generated and how robustly it addresses the research question.

The record names several secondary outcomes, but does not report their results or analysis methods. In general, examining multiple outcomes creates multiplicity: the more statistical questions considered, the greater the chance of obtaining an apparently noteworthy result by chance if each is tested separately. Researchers may address this through prespecified priorities or statistical adjustments, but the registry record does not state whether any such approach was planned or used. No conclusion about multiplicity control can be drawn.

For safety outcomes, counts and denominators are descriptive summaries. In general, a count of zero observed serious events does not establish zero risk, particularly when the population observed is limited. Interpretation also depends on the event definitions, observation period and completeness of reporting. Those details are not reported in the registry record. The safety figures therefore should be read as registry summaries rather than as a complete characterization of risk.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Palonosetron")
n      <- c(85)
events <- c(13)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Palonosetron  85  13  15.3  8.4  24.7  9.2  24.4  9.2  24.5

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists a primary endpoint and several secondary outcomes. It does not report an analysis plan specifying which comparisons or outcomes were confirmatory, how secondary outcomes were prioritized, or whether any adjustment for multiple statistical tests was planned. When many outcomes are examined, the chance of at least one apparently unusual result can rise if each is evaluated independently. Methods to manage this issue are general statistical options, not documented features of this study. Since no outcome analyses are listed, the record does not permit an assessment of how multiplicity affected interpretation.

Missing-data methods are not reported in the registry record. The record does not give the number of participants with an observed primary outcome, explain how incomplete diaries or other unavailable measurements were handled, or say whether participants with missing endpoint information were included in an analysis. These details matter because the endpoint requires information about both emetic episodes and rescue medication use during the specified delayed period. Without an analysis denominator and missing-data rules, the completeness of the primary endpoint assessment cannot be evaluated.

The analysis population is also not reported in the registry record. The listed enrollment is 85, but the record does not say whether all enrolled participants contributed to every outcome or whether a defined subset was analyzed. It does not describe an intention-to-treat population or another analysis set. The statistical analysis plan, interim analyses, and any approach to protocol deviations are not reported in the registry record. No specific procedure should be assumed.

12. 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 serious adverse-event summary lists 85 participants at risk and 0 affected. The other-event summary lists 85 participants at risk and 85 affected. The registry record does not identify the specific other events in the information available, so the 85 affected figure cannot be interpreted as a count for any named symptom or diagnosis. The death count is not reported in the registry record, and no top serious-event terms are listed.

These are descriptive registry entries, not a comparison between treatment groups. With no separate comparator group, the figures cannot show whether event occurrence differed from another regimen or from an untreated condition. They also cannot determine whether an event was caused by the study medications. In general, the absence of observed serious events in a study group does not establish that the risk is absent; interpretation depends on the amount and duration of observation and on complete event reporting. Those details are not reported in the registry record.

The other-event count should not be read as a severity measure or as evidence that all participants experienced the same event. The record does not provide event-specific definitions, timing, recurrence, or individual participant-level descriptions. These summaries should therefore be understood narrowly as the counts and denominators recorded, rather than as a full safety profile.

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

The registry record identifies a clinical question and describes a treatment schedule for delayed chemotherapy-induced nausea and vomiting. Its primary endpoint is a complete response during 24-120 hours after chemotherapy, defined as no emetic episode and no rescue medication use. The record also gives the enrollment and design classification. Those details clarify what the study was intended to assess, but they do not show the observed primary outcome.

The numerical primary result, its denominator, and an uncertainty estimate are not reported in the registry record. Therefore, the record does not support a quantitative statement about how often participants achieved complete response or how precisely that frequency was estimated. It also does not support a treatment comparison: the study is described as single-group, with no separate comparator arm.

Even if a within-group outcome estimate were available, a single-group result would describe participants in the study context. It would not by itself establish what would have happened to similar participants under another regimen. Differences between groups in other settings could reflect differences in patients, chemotherapy, supportive care or outcome measurement. A randomized comparator would be needed to estimate a direct contrast under the study design.

The registry names several secondary outcomes and gives brief serious and other adverse-event counts. Their detailed results and definitions are incomplete in the record. As a result, those entries cannot fill the gap left by the missing primary endpoint result or provide a comprehensive assessment of benefits and harms.

Overall, the record documents a phase 2, non-masked, single-group study with enrollment of 85 and a specified delayed complete-response definition. It does not report the outcome analyses needed to judge the observed response rate, its precision, or a comparative treatment effect. Statistical conclusions should remain limited to the design and registry entries that are actually reported.

14. Why This Trial Matters Statistically

This trial illustrates why a precise endpoint definition is essential for interpreting clinical outcomes. Complete response is not simply a report of nausea or vomiting: the registry definition requires both no emetic episode and no rescue medication use during the delayed period. That composite rule makes clear which observations count toward the endpoint, while also making the analysis dependent on recording both components.

The study also demonstrates the distinction between a treatment description and a treatment comparison. A regimen can be described in detail in a single-group study, but without a separate comparator the design does not estimate a randomized difference between strategies. This distinction is central when readers move from describing an outcome within a group to considering whether outcomes differ because of treatment.

Another statistical lesson is that enrollment alone is not enough to understand evidential strength. The registry lists 85 enrolled, but does not give the primary endpoint denominator, observed result, or uncertainty interval. A reader needs those elements to assess both the estimated outcome frequency and its precision. Without them, no reliable quantitative interpretation of the endpoint can be made from the registry record.

Finally, the record illustrates the limits of planning calculations when the study design and outcome result are not available in matching detail. The stated planning assumptions refer to equal allocation between two arms, whereas the trial is classified as single-group. The assumptions are planning illustrations, not evidence that this trial was designed or analyzed as a two-arm comparison. Distinguishing hypothetical sample-size planning from the actual study design prevents an apparent calculation from being mistaken for an observed finding.

15. Limitations

• The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
• Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.

The most important limitation of the registry record for statistical interpretation is that it does not report the primary endpoint result. Although the endpoint definition and timeframe are stated, the number meeting the definition, the denominator assessed, and an uncertainty estimate are not reported in the registry record. The primary outcome therefore cannot be evaluated quantitatively from the posted information.

The study is described as a single-group trial with no masking and no separate comparator. This design can describe outcomes within the treated group but cannot provide a randomized comparison with another regimen. The record does not report the statistical analysis plan, any interim analyses, or the rationale for sample size. The analysis population and handling of missing outcome information are also not reported.

The regimen description has different levels of detail in the brief summary and arm description. The brief summary describes doses, routes and timing, while the arm description is less complete. The registry record does not explain the difference. The record also does not provide participant characteristics, detailed eligibility criteria, chemotherapy-specific outcome summaries, or the duration of safety observation. These omissions limit assessment of how the reported group and outcomes relate to particular clinical circumstances.

Secondary outcomes are named, but their numerical results and analysis methods are not reported in the registry record. Serious adverse events are summarized with zero affected among 85 at risk, but the record does not provide event terms or further safety detail; the death count is not reported. The other-event count is recorded as 85 affected among 85 at risk, without specific event definitions in the available information. These entries cannot be treated as a complete account of safety.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Palonosetron, Ondansetron, and Dexamethasone for Delayed Nausea and Vomiting in Autologous Transplant Patients — NCT01370408.
• Registry record last updated 2017-04-17; status last verified 2017-03.
• Results first posted 2017-02-06.
• 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.

19. Record Summary

NCT01370408 is listed as a completed phase 2, single-group study with 85 enrolled participants. It examined a schedule involving palonosetron, ondansetron and dexamethasone for patients receiving myeloablative chemotherapy for autologous stem cell transplantation. The primary endpoint was delayed complete response, defined as no emetic episode and no rescue medication use during 24-120 hours after chemotherapy.

The registry record does not report the primary endpoint result, its analysis denominator, or an uncertainty estimate. It lists secondary outcomes but does not report their results or analyses. Allocation is marked NA, masking NONE, and no separate comparator group is described. Serious adverse-event entries list 0 affected among 85 at risk; other-event entries list 85 affected among 85 at risk. The event details and death count are not reported in the registry record.