Safety Study of Levocetirizine Oral Solution for Japanese Pediatrics: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Safety Study of Levocetirizine Oral Solution for Japanese Pediatrics (NCT01563081), a na phase 3 study. The primary outcome is Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (AEs).

2. Clinical Question

The clinical question concerns safety during treatment with levocetirizine oral solution in children aged 6 months to 2 years who have allergic rhinitis or pruritus associated with skin diseases. The primary outcome counts participants with serious adverse events (SAEs) and non-serious adverse events (AEs) through Week 2 or early withdrawal (EW). The registry record defines an AE as an untoward medical occurrence temporally associated with use of a medicinal product, whether or not considered related to it. It defines an SAE by specified serious consequences, including death, life-threatening events, hospitalization, disability, congenital anomaly, or possible drug-induced liver injury.

The safety outcome is reported for the total population and for age groups described as at least 6 months and under 12 months, and at least 12 months and under 24 months. The record reports 0 participants with SAEs in each age group and in the total population. It reports 7 participants with non-serious AEs in the younger age group, 12 in the older age group, and 19 in the total population. These are counts of participants, not a measure of symptom improvement or treatment benefit.

The study also lists secondary outcomes concerning investigator assessments of changes in allergic rhinitis symptoms and pruritus, pruritus severity, and levocetirizine concentrations in plasma. The primary question remains descriptive: what numbers of participants in this treated group were recorded as having serious or non-serious AEs during the stated follow-up? Because the study is single-group and has no control comparison, it does not estimate how those counts differ from outcomes under another treatment or no treatment.

3. Trial Design

This was a Phase 3, multicenter, open-label study with a single-group model. The registry record lists allocation as not applicable and masking as none. It describes the study as evaluating the safety of levocetirizine hydrochloride oral solution in children aged 6 months to 2 years with allergic rhinitis or pruritus associated with skin diseases. The record lists an enrollment of 60. The listed solution contains 0.50 mg of levocetirizine dihydrochloride per milliliter.

The study status is completed. The recorded start is 2012-04, and primary completion is recorded as 2012-08. The primary safety outcome covers the period up to Week 2 or EW. The safety population is defined as all participants who participated in the study and received at least one dose of medication. The adverse-event counts are presented for two age groups and for the total population; the record lists 30 participants at risk in each age group and 60 in the total population.

There is no like-for-like control group in this design. The listed treatment groups are age-based presentations of the levocetirizine-treated population, not randomized treatment arms. Consequently, comparisons between the age groups are descriptive and cannot isolate an age effect from other differences between participants. Open-label status also means masking was not used, although the registry record does not describe how that might have affected individual assessments.

The record lists symptom assessments and plasma concentration measures among the secondary outcomes. It does not report the detailed statistical plan, the method of treatment assignment, or a sample-size justification. Those features should not be inferred from the study phase or design labels.

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

Analysis population for the primary outcome, as defined in the registry: Safety Population : all participants who participated in this study and who received at least one dose of medication

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 Number of Participants With Serious Adverse Events (SAEs) and Non-serious Adverse Events (AEs), measured over up to Week 2/Early Withdrawal (EW).

The primary endpoint counts participants with SAEs and non-serious AEs through Week 2 or EW. It is a participant-count outcome: the reported values describe how many participants experienced an event in each category during the stated period. They are not event rates per unit of follow-up, and they do not indicate how many separate events occurred in a participant.

Safety outcomes are used to describe the adverse events recorded during exposure to a study treatment. The registry definitions make clear that an AE is temporally associated with medicinal-product use and need not be judged treatment-related. An SAE is distinguished by its seriousness, based on outcomes such as death, life threat, hospitalization, disability, congenital anomaly, or possible drug-induced liver injury. The endpoint therefore provides a structured account of serious and non-serious events, rather than a direct test of whether treatment caused an event.

For this study, the registry reports no participants with SAEs and reports participants with non-serious AEs in the age groups and total population. These counts are useful as a description of the study’s recorded safety experience over its short stated follow-up. They do not, without a comparator and more information about ascertainment and follow-up, establish the excess risk attributable to treatment or the frequency expected in a broader population.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a count of participants with events. The registry labels the outcome unit as participants and the parameter as a number. In practical terms, the outcome asks whether a participant was recorded as having an SAE or a non-serious AE during the specified period. The reported count is not the number of event episodes, because one participant could have more than one event and the primary outcome is phrased in terms of participants.

What does a participant count tell the reader?

General statistical explanation: a count is a direct descriptive summary of observed cases within a defined population and observation period. It is easiest to interpret alongside the population at risk and the outcome definition. The record gives at-risk counts for each age group and the total population, as well as the number with events. Counts alone do not describe event timing, duration, recurrence, severity among non-serious events, or the relationship of an event to treatment.

The serious and non-serious categories are based on the registry’s definitions. An AE is a medical occurrence temporally associated with medicinal-product use, whether or not considered related. An SAE is defined by specified serious consequences. Thus, an AE count is not equivalent to a count of events judged to be caused by the drug. The primary outcome gives a broad safety summary, while the record’s separate AE module is identified as the place for lists of events occurring at a frequency of at least 5%.

Why does the design matter for interpretation?

This is a single-group study with no masking and allocation listed as not applicable. General statistical explanation: a single-group descriptive study can report what was observed among participants who received the study treatment, but it cannot directly estimate a comparative treatment effect. Without a concurrent control group, differences from an external expectation may reflect differences in participants, observation, or clinical context as well as treatment. The age categories are subgroup descriptions within the treated population, not randomized comparisons.

Open-label conduct means that masking was not used. In general, knowledge of treatment may influence reporting or assessment of subjective outcomes. That concern is relevant to symptoms and investigator assessments, but the registry record does not report whether or how assessment procedures addressed it. It would be inappropriate to assume either that assessments were biased or that open-label conduct had no influence.

What do uncertainty measures add?

General statistical explanation: an estimate is more informative when accompanied by a measure of its precision, such as a confidence interval. A confidence interval describes a range of values compatible with the observed data under the interval’s statistical assumptions. For an event count or an event frequency, the interval can help communicate that limited observations leave uncertainty even when the observed count is small or zero. The registry record does not report confidence intervals for the primary event counts, so no numerical precision estimate should be inferred from the counts alone.

General statistical explanation: sparse outcomes require particular care because a small number of observed events can yield imprecise estimates. The absence of an observed SAE in this study is a descriptive result, not evidence that such an event cannot occur. In addition, the follow-up period ends at Week 2 or EW, so the outcome cannot characterize later events. The record does not report a time-to-event analysis or an adjusted model for the primary outcome.

What analysis details are documented?

The registry record lists 5 outcome measures and no analyses. It does not report a statistical analysis plan, a method for handling missing observations, an inferential testing procedure, or a sample-size justification. The study’s Phase 3 label does not supply those missing details. No model, adjustment, or hypothesis test should be attributed to the investigators on the basis of this record alone.

General statistical explanation: multiplicity arises when several outcomes or comparisons are examined, since the chance of at least one apparently unusual result can increase across multiple tests. A prespecified hierarchy or adjustment can address that issue, but the registry record does not report such a plan. The listed secondary outcomes include symptom-change assessments, pruritus severity, and plasma concentration measures. They are distinct from the primary safety endpoint, and their presence does not establish that formal comparisons were made.

For this endpoint, the most defensible reading is descriptive: the record gives participant counts for serious and non-serious AEs, by age group and in the total population, over a specified follow-up. It does not provide the additional statistical detail needed to turn those counts into comparative or causal conclusions.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 5 outcome measures, including the primary safety outcome and secondary outcomes concerning symptom changes, pruritus severity, and plasma concentrations. It reports no analyses. It does not describe a multiplicity strategy, such as a testing hierarchy or adjustment for examining multiple outcomes. General statistical explanation: when several outcomes are tested, multiplicity can increase the chance of a nominally unusual finding arising by chance. Without the analysis plan, it is not possible to determine whether or how that issue was addressed.

Missing-data handling is not reported in the registry record. The primary outcome defines the safety population as participants who participated and received at least one dose, but that definition does not explain how incomplete AE follow-up, early withdrawal, or missing records were handled. The timeframe includes events through Week 2 or EW, yet the record does not describe the completeness of follow-up or the treatment of observations after withdrawal.

The record also does not report an intention-to-treat population or other analysis-population definitions beyond the safety population. The adverse-event counts include the stated at-risk populations, but no further analysis details are given. These omissions limit assessment of whether all eligible observations were included consistently and how missingness could affect the results. They should not be interpreted as evidence that missing data occurred, or that none occurred.

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 primary safety outcome reports 0 participants with SAEs in the total population and 0 in each of the two age groups. For non-serious AEs, it reports 19 participants in the total population, with 7 in the group aged at least 6 months and under 12 months and 12 in the group aged at least 12 months and under 24 months. The record lists 60 participants at risk in the total population and 30 in each age group.

These figures count participants, not separate events, and the event definition does not require a judgment that the medicinal product caused the occurrence. The registry record describes the primary outcome period as up to Week 2 or EW. It does not report deaths in the adverse-event entries; death counts are not reported in the registry record. The listed serious-event terms are empty, while the primary outcome reports 0 participants with SAEs.

General statistical explanation: zero observed events does not prove that the underlying event risk is zero. These counts describe what was recorded in this population and follow-up period; they do not establish comparative risk or causality.

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

The result describes the recorded safety experience in a single-group study of levocetirizine oral solution among children aged 6 months to 2 years with allergic rhinitis or pruritus associated with skin diseases. The registry reports 0 participants with SAEs and 19 participants with non-serious AEs in the total population through Week 2 or EW. Those counts provide a straightforward account of the primary outcome as defined in the record.

They do not establish that levocetirizine caused the reported AEs. The AE definition explicitly includes occurrences whether or not considered related to the medicinal product. Nor do the counts establish that treatment prevents, causes, or changes events compared with another treatment or no treatment: there was no control comparison. The age-group counts are descriptive and should not be treated as evidence of age-related differences in safety.

The reported absence of SAEs in the study does not show that serious events cannot occur. General statistical explanation: an observed count is not the same as certainty about the underlying event frequency; the precision of an estimate depends on the information available and its assumptions. The registry record does not report confidence intervals for these outcomes. The observation period is limited to Week 2 or EW, so the result does not describe later safety outcomes.

The primary endpoint does not measure whether allergic rhinitis symptoms or pruritus improved, and it does not summarize the plasma concentration outcomes. Those are listed as secondary outcomes, but their results are not included in the registry record for this interpretation. The registry record also does not report a detailed statistical analysis plan or missing-data procedures. Accordingly, the result supports a descriptive account of recorded events, not a broader conclusion about treatment effectiveness, comparative safety, or longer-term outcomes.

13. Why This Trial Matters Statistically

This trial is statistically informative as an example of how a safety outcome can be reported using participant counts in a defined population and follow-up period. The primary endpoint distinguishes SAEs from non-serious AEs and presents counts for age groups as well as the total population. That structure helps readers see what was recorded while keeping the unit of analysis clear: participants with events, rather than total event episodes.

The design also illustrates why study structure governs what can be concluded. A single-group, open-label study can describe observed outcomes among treated participants, but it cannot estimate a randomized treatment contrast. The two age-group summaries do not substitute for a control group, because both describe participants receiving the same listed treatment. This distinction is essential when interpreting safety counts that may otherwise appear to imply a treatment-related risk.

The record’s definitions matter as well. An AE is temporally associated with use of a medicinal product, regardless of whether it is considered related. Treating an AE count as proof of causation would therefore misread the endpoint. Similarly, no observed SAE in the study is a statement about the observed follow-up, not a guarantee about risk outside the study.

Finally, the record leaves several statistical details unreported, including analysis methods, missing-data handling, and multiplicity procedures. Recognizing those gaps is part of sound statistical interpretation. The posted safety counts are useful as descriptive evidence within the study’s scope, while their limitations prevent unsupported comparative or causal claims.

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 single-group design is the main limitation for comparative interpretation. There is no randomized control group, so the registry record cannot support a treatment-versus-control estimate of safety. The age groups are not treatment arms and cannot isolate the effect of age. Open-label conduct means masking was not used; the record does not explain whether assessment procedures were standardized or how knowledge of treatment may have affected reporting.

The primary endpoint covers events only through Week 2 or EW. It therefore provides no information about events outside that period. The outcome is reported as counts of participants, without event-level detail in the primary outcome information. The record does not report event timing, duration, severity for non-serious AEs, or participant-level patterns. It identifies a separate AE module for events occurring at a frequency of at least 5%, but those event details are not included in the primary outcome details.

Several analysis features are not reported in the registry record: statistical methods, confidence intervals, missing-data handling, the multiplicity approach, and the sample-size justification. The record also does not report the completeness of follow-up. These omissions limit evaluation of precision and potential bias; they do not establish that missingness or bias occurred. The primary outcome counts should therefore be read as descriptive findings within this study, not as a complete characterization of all possible safety outcomes.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Safety Study of Levocetirizine Oral Solution for Japanese Pediatrics — NCT01563081.
• Registry record last updated 2017-02-28; status last verified 2017-01.
• Results first posted 2013-06-03.
• 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

NCT01563081 is a completed Phase 3, multicenter, open-label, single-group study of levocetirizine oral solution in children aged 6 months to 2 years with allergic rhinitis or pruritus associated with skin diseases. The listed enrollment is 60. The solution contains 0.50 mg of levocetirizine dihydrochloride per milliliter. The primary safety outcome counts participants with SAEs and non-serious AEs through Week 2 or EW.

The registry reports 0 participants with SAEs and 19 with non-serious AEs in the total population. The age-group counts for non-serious AEs are 7 and 12, and the record reports 0 SAEs in each age group. Because the study has no control group, these counts are descriptive rather than comparative. The registry record does not report detailed statistical methods, missing-data procedures, or confidence intervals for the primary safety outcome.