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 Safety, PK and Effectiveness of IV Peramivir Versus Oseltamivir in Pediatric Subjects With Uncomplicated Influenza (NCT02369159), a randomized phase 3 study. The primary outcome is Safety and Tolerability, as Measured by the Number of Participants Experiencing Adverse Events..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02369159 |
| Phase | Phase 3 |
| Condition | Influenza |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 137 |
| Arms in the primary outcome | n/a |
| Lead sponsor | BioCryst Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Pediatric subjects with acute uncomplicated influenza. The safety population is defined as randomized subjects who received at least one partial dose of peramivir or oseltamivir.
Intervention
Peramivir, given as a single age-appropriate intravenous dose by short infusion. The registry description specifies an infusion lasting at least 15 minutes.
Comparator
Oral oseltamivir at an age-appropriate dose, given twice daily for 5 days.
Primary question
The primary endpoint concerns safety and tolerability, measured by the number of participants experiencing adverse events over 14 days.
The study also planned to evaluate pharmacokinetics and effectiveness. Its listed secondary outcomes include peramivir plasma exposure over 6 hours after dosing, time to resolution of fever and influenza symptoms, time to reduction in viral shedding, changes in influenza virus titer in nasopharyngeal samples, and influenza-related complications. These are stated aims and outcome measures; the registry record does not supply comparative results for them in the information reported.
The question is therefore broader than a single comparison of event counts. The primary endpoint is a safety count, while the study’s other stated aims concern drug exposure, symptoms, viral measures, and complications. The registry record does not report one overall estimate that combines these questions, nor does it establish a treatment effect for effectiveness outcomes.
3. Trial Design
The registry describes a phase 3 randomized, open-label, active-controlled, parallel-group study of intravenous peramivir and oral oseltamivir in pediatric subjects with acute uncomplicated influenza. It lists enrollment as 137 and gives the study status as completed. The study start date is 2015-03-11, and the primary completion date is 2019-10-28. These record details describe the trial’s registration and conduct; they do not by themselves establish how many participants contributed data to each outcome.
Subjects assigned to peramivir were to receive a single age-appropriate intravenous dose, diluted in normal saline and given as a short infusion lasting at least 15 minutes. Subjects assigned to oseltamivir were to receive an age-appropriate oral dose twice daily for 5 days. The registry’s arm descriptions contain additional age-specific dosing text, but portions of those descriptions are incomplete in the record. The omitted details cannot be reconstructed from the information reported.
Random allocation is intended, in general, to reduce systematic differences between treatment groups at assignment. An open-label design means the registry reports no masking. Knowledge of assigned treatment can matter for outcomes involving judgments or reporting, including adverse events and symptom resolution. That design feature is relevant when interpreting results, but it does not establish that any particular assessment was biased.
The reported safety population includes randomized subjects who received at least one partial dose of either study drug. The safety counts use group-specific denominators, and the registry does not provide a complete account of the analysis populations for all outcomes. It also does not report the allocation ratio, details of randomization implementation, or the statistical analysis plan in the information. Those details 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 | Peramivir | Oseltamivir |
|---|---|---|
| STARTED | 114 | 23 |
| ITT Population | 114 | 23 |
| Safety Population | 107 | 23 |
| ITTI Population | 81 | 16 |
| E-R Population | 80 | 0 |
| COMPLETED | 106 | 21 |
| NOT COMPLETED | 8 | 2 |
Reasons for not completing this period:
| Reason | Peramivir | Oseltamivir |
|---|---|---|
| Adverse Event | 0 | 1 |
| Lost to Follow-up | 1 | 1 |
| Withdrawal by Subject | 7 | 0 |
Analysis population for the primary outcome, as defined in the registry: The Safety population included all randomized subjects who received ≥ 1 partial dose of peramivir or oseltamivir.
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 Intent-to-Treat (ITTI) population included all randomized subjects and was the primary population for analyses of demography and subject accountability.
| Characteristic | Unit | Peramivir | Oseltamivir | Total |
|---|---|---|---|---|
| Participants analysed | 114 | 23 | 137 | |
| Age, Continuous: Overall | Age at time of randomisation - years | 8.0 (SD 5.07) | 9.9 (SD 4.99) | 8.4 (SD 5.09) |
| Age, Continuous: ≥ 28 days to < 2-year-old cohort | Age at time of randomisation - years | 1.3 (SD 0.47) | 1.0 (SD NA) | 1.3 (SD 0.47) |
| Age, Continuous: ≥ 2 to < 7-year-old cohort | Age at time of randomisation - years | 4.9 (SD 1.47) | 4.7 (SD 1.60) | 4.8 (SD 1.47) |
| Age, Continuous: ≥ 7 to < 13-year-old cohort | Age at time of randomisation - years | 9.6 (SD 1.75) | 9.7 (SD 1.92) | 9.6 (SD 1.76) |
| Age, Continuous: ≥ 13 to < 18-year-old cohort | Age at time of randomisation - years | 15.7 (SD 1.45) | 15.9 (SD 1.50) | 15.7 (SD 1.44) |
| Sex: Female, Male: Female | Participants | 59 | 14 | 73 |
| Sex: Female, Male: Male | Participants | 55 | 9 | 64 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 8 | 0 | 8 |
| Race (NIH/OMB): White | Participants | 98 | 23 | 121 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 7 | 0 | 7 |
| Baseline Influenza Viral Titer: Positive | Participants | 70 | 14 | 84 |
| Baseline Influenza Viral Titer: Negative | Participants | 37 | 9 | 46 |
| Baseline Influenza Viral Titer: Missing | Participants | 7 | 0 | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Safety and Tolerability, as Measured by the Number of Participants Experiencing Adverse Events., measured over 14 days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is safety and tolerability measured by the number of participants experiencing adverse events during a 14-day timeframe. The registry describes safety evaluation as including assessment of adverse events. The stated safety population consists of randomized subjects who received at least one partial dose of peramivir or oseltamivir.
A participant count is a way to summarize how many people experienced an event, rather than how many separate event episodes occurred. In general, such a count can make a safety outcome relatively direct to interpret, but it does not by itself describe the nature, duration, severity, or timing of each event. Those features require more detailed event-level reporting.
The registry record does not reduce the primary outcome to one comparison, noting that it has several categories or time points. Consequently, the posted information does not give a single primary-endpoint contrast or an associated uncertainty estimate. The group-level adverse-event counts are useful descriptive safety information, but they should not be mistaken for a complete analysis of every category or time point included in the primary outcome.
The endpoint is used to assess safety and tolerability, not to establish effectiveness. The record lists effectiveness-related outcomes separately, including symptom resolution and viral measures. Results for those outcomes are not reported in the information.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry posts safety counts for two groups. For peramivir, the serious adverse-event count is 0 among 107 participants at risk, and the count for other adverse events is 22 among 107. For oseltamivir, the serious adverse-event count is 0 among 23 participants at risk, and the count for other adverse events is 5 among 23. The posted death counts are 0 in each group. These figures are descriptive counts, not a complete comparative analysis.
The group denominators differ, so the event counts alone do not show how the groups compare on a common scale. No percentages or adjusted comparisons are reported in the information available, and none should be inferred from the counts alone. The record does not supply confidence intervals, hypothesis tests, or a single estimate for the primary endpoint. It reports zero analyses in the results information, and the primary outcome is not reduced to one comparison because it includes several categories or time points.
Zero serious adverse events in the posted counts means that no serious events are recorded for these safety groups in this summary. It does not establish that serious events are impossible, nor does it precisely establish that the underlying risk is the same between treatments. General statistical interpretation: when an event is uncommon, a count of zero can still be compatible with a range of possible underlying event rates. The width of that uncertainty depends on the available data and analysis; no interval is reported.
The counts for other adverse events show that participants in both groups were recorded as experiencing such events. The summary does not identify the specific events, their severity, their timing, or whether participants experienced more than one event. It therefore cannot support conclusions about the relative clinical importance of the events or the overall balance of benefits and harms.
The record’s listed secondary outcomes include pharmacokinetic exposure, fever and symptom resolution, viral shedding, viral titer, and influenza-related complications. The outcome names describe what the study set out to assess, not what it found. Comparative results for these measures are not reported in the information summarized. Thus, the posted counts do not answer the broader effectiveness questions identified in the trial description.
Overall, the registry record supports a narrow descriptive reading of the reported safety counts. It does not supply enough inferential detail to judge the precision of treatment-group differences, and it does not support a definitive conclusion about the comparative safety or effectiveness of the two regimens.
9. Statistical Methods Explained
The registry describes randomized allocation in a parallel-group trial. In general, randomization assigns participants to treatment groups using a chance-based process, with the aim of limiting systematic differences at baseline. It does not ensure that every characteristic will be balanced, especially when groups are small, and it does not eliminate uncertainty. The record does not report enough detail to assess the implementation of randomization or the balance of baseline characteristics.
What does a participant count represent?
The primary outcome is framed as the number of participants experiencing adverse events. A participant count differs from a count of event episodes: one participant may have more than one event, but a person-level count records whether participants experienced an event according to the outcome definition. The registry describes the safety measure in terms of participants. Its posted adverse-event summaries list participants affected, rather than giving a full account of event episodes.
Counts are easiest to interpret alongside their denominators and precise outcome definitions. The posted safety summaries include denominators for each group, but the primary endpoint itself covers several categories or time points. The registry notes that it has not been reduced to one comparison. Accordingly, the available count summaries should not be treated as a complete analysis of every component of the primary endpoint.
Why does uncertainty matter?
General statistical explanation: an estimate describes a pattern in the observed data, while an uncertainty interval indicates how precisely the data estimate an underlying quantity under the analysis assumptions. A confidence interval is one common way to express that uncertainty. It is not a guarantee that the true value lies within a particular interval, nor does it correct for limitations in study design or measurement. No confidence intervals are reported for the safety counts in the information summarized.
A significance test, when used, assesses how compatible the observed data are with a specified null hypothesis under a statistical model. A p-value is not the probability that a treatment works, and it does not measure the size or clinical importance of an effect. The registry information does not report a p-value or a test for the displayed safety counts. No significance conclusion should be inferred from the counts alone.
What does the open-label design imply?
General explanation: when participants and study personnel know the assigned treatment, outcome reporting and assessment may be influenced by that knowledge, particularly for outcomes involving symptoms or judgments. This possibility is relevant to an open-label study but does not prove that bias occurred. Randomization and open-label status address different design features: randomization concerns assignment, while masking concerns knowledge of assignment after it occurs. The registry lists no masking.
How should the sample-size illustration be read?
The registry record describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect. For time-to-event outcomes, the illustration uses a total event count; for other outcomes, it uses participants per arm. These assumptions describe a planning calculation, not the trial’s original design or an observed measure of power.
General explanation: sample-size planning asks how much information a future study might need to detect a chosen effect under stated assumptions. A calculation based on an observed effect is sensitive to the estimate used, which may be imprecise. It cannot establish that the completed trial had a particular probability of detecting an effect. The registry does not report the trial’s own sample-size justification, and the planning illustration should not be interpreted as observed power.
What analysis details are available?
The registry information does not report a statistical analysis plan, a model for the primary endpoint, an approach to adjustment, or methods for handling missing observations. It also does not report interim analyses. These omissions prevent assessment of the prespecified analysis choices or whether the posted summaries reflect all planned analyses. General statistical methods cannot fill in those missing trial-specific details.
10. Multiplicity, Missing Data and Analysis Populations
The primary outcome is described as having several categories or time points, and the registry does not reduce it to one comparison. When several outcomes, categories, or time points are assessed, the chance of at least one apparently notable finding can increase if many tests are conducted. General statistical practice may address this through prespecified outcome hierarchies, adjusted testing, or careful interpretation, but the registry information does not say whether any such approach was planned or used.
The record lists multiple secondary outcomes, including pharmacokinetic exposure, fever and symptom resolution, viral shedding, viral titer, and influenza-related complications. Their presence does not show that each was analyzed or that they were all treated as confirmatory. A multiplicity strategy is not reported in the registry record. Without that information and the outcome results, it is not possible to assess how the study handled the possibility of chance findings across outcomes.
The registry information does not describe missing-data amounts, reasons for missingness, imputation, censoring, or sensitivity analyses. General explanation: missing observations can affect an estimate when the probability of being observed is related to the outcome or treatment experience. The consequences depend on why data are missing and how the analysis handles them. Those details are not reported in the registry record, so the impact of missingness cannot be evaluated.
The safety population is defined as randomized subjects who received at least one partial dose of study drug. The record does not provide equivalent analysis-population definitions for the secondary outcomes or explain how participants who did not receive a dose or lacked outcome data were handled. The stated safety population therefore should not be assumed to apply to every analysis.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Peramivir | 0 | 107 | 0.0% |
| Oseltamivir | 0 | 23 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Peramivir | 0 of 107 |
| Oseltamivir | 0 of 23 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 1% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Peramivir | 22 of 107 |
| Oseltamivir | 5 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Peramivir | Oseltamivir |
|---|---|---|---|
| Vomiting | Gastrointestinal disorders | 4 of 107 | 2 of 23 |
| Diarrhoea | Gastrointestinal disorders | 2 of 107 | 0 of 23 |
| Nausea | Gastrointestinal disorders | 0 of 107 | 2 of 23 |
| Pyrexia | General disorders | 2 of 107 | 0 of 23 |
| Tympanic membrane hyperaemia | Ear and labyrinth disorders | 2 of 107 | 0 of 23 |
| Elevated aspartate aminotransferase | Investigations | 2 of 107 | 0 of 23 |
| Elevated blood lactate dehydrogenase | Investigations | 2 of 107 | 0 of 23 |
| Dizziness | Nervous system disorders | 0 of 107 | 1 of 23 |
| Hallucination | Psychiatric disorders | 0 of 107 | 1 of 23 |
| Chest discomfort | General disorders | 1 of 107 | 0 of 23 |
The serious adverse-event summaries report 0 affected participants among 107 at risk in the peramivir group and 0 among 23 at risk in the oseltamivir group. The posted death count is 0 in each group. For other adverse events, the summaries report 22 affected participants among 107 at risk for peramivir and 5 among 23 at risk for oseltamivir.
These are group-specific participant counts. They do not state how many separate events occurred, identify the event terms, or describe severity and timing for the other adverse events. The serious-event term list is empty in the registry information. An empty list does not supply details about events beyond the zero counts reported in the summary.
The group counts should not be compared as if their denominators were the same. No interval estimate or statistical test is reported for these figures. General explanation: an observed zero is not proof that the underlying risk is zero; the amount of uncertainty depends on the amount and structure of the data. The registry record does not provide an uncertainty analysis for these safety counts.
These summaries offer a limited description of recorded events in the safety population. They do not, on their own, establish comparative safety or describe the complete benefit-harm profile.
12. What the Result Does, and Does Not, Mean
The posted results allow readers to identify the safety counts recorded for the two treatment groups and the denominators attached to those counts. They also show that the registry’s primary safety outcome was defined around participants experiencing adverse events over 14 days. Those details are useful for understanding what was measured and the scope of the available summary.
The counts do not establish that one treatment is safer or less safe than the other. The registry does not report a single primary-endpoint comparison, an uncertainty interval, or a statistical test for these figures. The group denominators differ, and the available summary does not provide event-level descriptions or a complete analysis of the primary outcome’s categories and time points.
The results also do not establish comparative effectiveness. Although the registry lists outcomes related to symptoms, viral measures, pharmacokinetics, and complications, the information described does not report comparative results for those outcomes. Stated study aims should not be confused with findings.
General statistical explanation: an observed count is a description of the participants recorded in a study. Drawing conclusions about broader populations requires attention to uncertainty, outcome definitions, study design, missing data, and the analysis plan. In this record, several of those elements are not reported. The absence of those details limits how far the posted counts can be interpreted.
The appropriate reading is therefore descriptive and bounded: the safety summary reports the listed counts for the defined safety population, while leaving the precision and comparative meaning of those counts unresolved. It neither establishes equivalence between treatments nor demonstrates a treatment advantage. The registry record does not support a broader conclusion about effectiveness or overall clinical value.
13. Why This Trial Matters Statistically
This trial is statistically interesting because it places a safety-focused endpoint alongside several distinct pharmacokinetic and effectiveness questions. A count of participants experiencing adverse events is not interchangeable with time to symptom resolution, viral measurements, or drug exposure. Each outcome has its own scale and interpretation, so a single summary cannot answer all the questions raised by the study aims.
The design also makes the distinction between randomization and masking important. Random assignment can help limit systematic differences between groups, while the open-label status means treatment assignment was not masked according to the registry. General statistical reasoning treats these as separate features, and the possible consequences depend on the outcome and its measurement. The record does not provide enough analysis detail to determine how such issues were addressed.
The posted safety summaries illustrate why denominators and uncertainty matter. Counts should be read with their group-specific denominators, and the registry does not provide inferential estimates for the comparison. In particular, zero serious adverse events in the summaries is a description of observed data, not proof of zero underlying risk or equal risk between groups.
The record also highlights the value of complete outcome reporting. It names a range of secondary outcomes but does not include their comparative results in the information described. Without those results, the stated scope of the study is broader than what can be evaluated from the available summaries. The study is therefore useful as an example of how trial design, endpoint definition, and reporting determine which statistical conclusions can be drawn.
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 registry information gives limited detail about the primary endpoint’s categories and time points and does not present one primary comparison. This restricts interpretation of the posted group counts as a complete primary-outcome result. No confidence intervals, hypothesis tests, or adjusted estimates are reported for the safety summaries.
Although the study is described as randomized and open-label, details of randomization implementation and baseline group characteristics are not included in the information summarized. The registry does not report the allocation ratio or a statistical analysis plan. These omissions make it difficult to assess the analysis choices or the comparability of groups beyond the design label.
The safety summaries do not provide event terms, event timing, severity details for other adverse events, or the number of separate event episodes. The serious adverse-event term list is empty. The reported counts therefore cannot characterize the full pattern of safety events.
The trial description lists secondary outcomes, but their results are not included in the information reported. Missing-data methods, reasons for missing observations, interim analyses, and multiplicity handling are also not reported in the registry record. The safety population is defined, but corresponding analysis-population details for other outcomes are not stated.
Finally, portions of the age-specific dose descriptions are incomplete in the registry text. The available information supports describing the broad regimens—single-dose intravenous peramivir and oral oseltamivir twice daily for 5 days—but not reconstructing omitted dosing details. Together, these limitations constrain interpretation to the stated design and posted safety counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety, PK and Effectiveness of IV Peramivir Versus Oseltamivir in Pediatric Subjects With Uncomplicated Influenza — NCT02369159.
- Registry record last updated 2021-03-23; status last verified 2021-02.
- Results first posted 2021-03-23.
- 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
The registry describes a completed phase 3, randomized, open-label, parallel-group study of intravenous peramivir versus oral oseltamivir in pediatric subjects with acute uncomplicated influenza. It lists enrollment as 137 and identifies safety, pharmacokinetics, and effectiveness as study aims. Peramivir was administered as a single age-appropriate intravenous dose; oseltamivir was administered orally twice daily for 5 days.
The primary endpoint concerns the number of participants experiencing adverse events over 14 days. The safety summary reports serious adverse-event counts of 0 among 107 peramivir participants and 0 among 23 oseltamivir participants, with other adverse-event counts of 22 and 5, respectively. The record does not provide a single primary comparison or inferential estimates for these counts. Comparative results for the listed secondary outcomes are not reported in the information summarized.