Long Term Safety Assessment of SER120 in Patients With Nocturia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Long Term Safety Assessment of SER120 in Patients With Nocturia (NCT00981682), a na phase 3 study. The primary outcome is Mean Serum Sodium.

2. Clinical Question

3. Trial Design

This is a Phase III, open-label extension study of SER120 nasal spray formulations in patients with nocturia. Its official title describes enrollment of patients completing one of two earlier SER120 studies. The registry lists the condition as nocturia, the study status as completed, and enrollment as 376. The recorded study period begins in 2009-08, with primary completion in 2011-06.

The design is single-group, with allocation recorded as not applicable and masking recorded as none. There is no listed comparator arm. Consequently, the record describes safety observations during SER120 use, not a randomized estimate of a treatment difference. Changes over time within a single group can be described, but without a concurrent control group they cannot by themselves distinguish treatment-associated changes from other explanations.

The study hypothesis states that SER120 is safe and well tolerated for use in nocturic patients. The primary outcome is mean serum sodium, with change from baseline assessed at specified visits through Week 40. The outcome population is the safety population. The record does not describe additional eligibility criteria, the precise dosing schedule, how baseline was defined, or how participants were handled if they discontinued or missed an assessment.

The registry lists one outcome measure and no analyses. It does not report numerical results for the primary outcome, a statistical analysis plan, or inferential estimates. Thus, the design and intended outcome can be described, but the posted record does not support a numerical assessment of serum-sodium changes or their precision.

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:

Analysis population for the primary outcome, as defined in the registry: Safety population

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: Safety population

6. Primary Endpoint

The registry lists the primary outcome as Mean Serum Sodium, measured over 40 weeks.

The primary endpoint is mean serum sodium, measured in mmol/L, with change from baseline assessed on Day 8, Day 15, Day 22, Week 4, Week 8, Week 12, Week 20, Week 28 and Week 40. The registry identifies the safety population as the population for this outcome and specifies a mean with standard deviation.

Serum sodium is a laboratory measure relevant to the study’s stated safety focus. Looking at change from baseline across scheduled visits can describe whether measured values vary during the observation period. The mean summarizes the group’s average value; the standard deviation describes how spread out individual measurements are around that mean. These summaries answer different questions: a group average does not show the full range of individual values, and a standard deviation is not a confidence interval for the mean.

The registry record does not report the numerical baseline mean, visit-specific means, changes, standard deviations, or confidence intervals. It also does not specify whether the reported change was intended to be calculated for each participant before averaging or by another method. Without the numerical results and analysis details, the direction, magnitude, variability and precision of the endpoint cannot be evaluated from the record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the mean describe?

The registered endpoint is a mean, a measure of the group’s average serum sodium value at a scheduled assessment. The registry specifies change from baseline, so the scientific target is a comparison between measurements at each listed visit and the baseline value. The record does not provide the numerical means or changes. It also does not explain the exact calculation used to obtain each change, so no particular calculation method should be assumed.

General statistical context: an average can make a group’s overall pattern easier to describe, but it can hide differences among individuals. Two groups of measurements can have the same mean while showing different amounts of spread or different patterns among participants. That is why the registered standard deviation is useful alongside a mean. A standard deviation describes dispersion among observations; it does not, by itself, quantify uncertainty in the estimated mean.

What does the standard deviation add?

The registry identifies standard deviation as the dispersion measure for mean serum sodium. In general, a larger standard deviation indicates measurements are more dispersed around the mean, while a smaller one indicates they are more concentrated. This is a description of variability in the observations, not a direct measure of how certain the estimated mean is. A confidence interval, when reported, addresses uncertainty around an estimate under specified assumptions. No confidence interval is reported for this endpoint in the registry record.

The available description does not say whether standard deviations were calculated from observed values at each visit, from participant-level changes, or using another approach. It does not give the number of observations contributing to each visit-specific summary. Such details matter when comparing summaries over time, particularly if the number or characteristics of participants with measurements change across visits.

How should repeated visits be understood?

The endpoint description lists assessments at Day 8, Day 15, Day 22, Week 4, Week 8, Week 12, Week 20, Week 28 and Week 40. These are repeated observations during the study. General statistical context: measurements from the same participant over time are usually related, rather than independent. An analysis of repeated measurements may need to account for that relationship, but the method used in this study is not reported in the registry record.

It is also not reported whether the analysis used every available measurement, a particular repeated-measures model, or a specified approach to missing observations. Without those details and the numerical results, it is not possible to reconstruct the endpoint analysis or judge whether estimates at later visits may reflect a changing set of participants.

Why does the single-group design matter?

The study has a single-group design, no masking, and no listed comparator. General statistical context: a within-group change can describe how measurements differ from baseline among observed participants, but it is not the same as a treatment-versus-control effect. Other factors, including changes over time unrelated to treatment, may also contribute to a within-group pattern. The registry record does not include a concurrent comparison that could help separate these explanations.

Open-label describes the absence of masking in the record. It does not establish whether knowledge of treatment could have affected any particular measurement or event assessment. How events were collected and adjudicated is not reported in the registry record.

What can the planning calculations establish?

The planning assumptions describe illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They describe detecting a stated fraction of the observed effect; the calculations for ordinary outcomes are expressed as participants per arm. Those assumptions are planning conventions, not a description of the actual single-group study design. The registry record does not report the observed endpoint effect needed to interpret such planning illustrations, nor does it report the resulting planning values in the registry record for this analysis.

General statistical context: sample-size planning asks what size a future study might need under selected assumptions. It is not evidence that a completed study had a particular probability of detecting an effect, and it is not observed power. Because this record does not report a primary-outcome estimate or the analysis used to obtain it, the planning assumptions cannot supply the missing endpoint results.

The registry does not report an inferential method, model, confidence-interval procedure, hypothesis test, or multiplicity adjustment for the primary outcome. These omissions limit independent interpretation. General explanations of means, dispersion, repeated observations and planning assumptions do not establish that any particular statistical method was used in the trial.

10. Multiplicity, Missing Data and Analysis Populations

The primary outcome is described at multiple scheduled assessments, from Day 8 through Week 40. The registry record does not state whether one visit was designated as the main inferential comparison, whether each visit was tested separately, or whether an overall repeated-measures analysis was planned. It also does not report any multiplicity procedure.

General statistical context: when multiple outcomes, time points, or comparisons are examined, the chance of at least one apparently notable result can rise if each is treated as a separate test. A prespecified analysis strategy can clarify which comparisons are confirmatory and how uncertainty is handled. The list of visits does not establish which approach was used. No p-values or confidence intervals are reported for the endpoint, so there is no posted inferential result on which to assess multiplicity.

Missing-data handling is also not reported. The endpoint population is identified as the safety population, but the registry does not describe how many participants contributed measurements at each visit, how discontinuations were handled, or whether missing values were imputed. General statistical context: if participants with available measurements differ from those without them, summaries based only on observed measurements may not represent the same group at every visit. The record does not provide information to assess that possibility.

The registry lists one outcome measure and no analyses. It does not report whether an analysis plan was finalized before examining results, whether interim analyses were conducted, or how the analysis population was defined beyond the safety-population label. These items are not reported in the registry record and should not be inferred from the outcome schedule or study status.

11. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 4% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: osteoarthritis (0.8%), basal cell carcinoma (0.5%), atrial fibrillation (0.3%), cardiovascular disorders (0.3%), coronary artery disease (0.3%).

The safety group is labeled SER120 750 mcg. The registry lists 376 at risk for serious adverse events and 30 affected; the other adverse-event fields list 376 at risk and 305 affected. It also records 30 deaths. These are registry counts. The record does not state the timing, causes, treatment relationship, or detailed definitions needed to interpret these entries further. It does not explain how the serious-event, other-event and death counts relate to one another, so they should not be combined or treated as mutually exclusive categories.

The listed serious adverse-event terms and percentages are osteoarthritis at 0.8%, basal cell carcinoma at 0.5%, and Atrial Fibrillation, Cardiovascular disorders, Coronary artery disease and myocardial infarction, each at 0.3%. The record does not provide event counts for these individual terms in the registry record for this analysis. Percentages alone do not show when events occurred, whether they were considered related to treatment, or whether a person experienced more than one event.

General statistical context: adverse-event counts describe events or affected participants according to the registry’s reporting fields; they do not establish causation. Interpretation also depends on consistent definitions, ascertainment, time at risk and follow-up completeness. Those details are not reported in the registry record.

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

The record supports a description of the study’s purpose and structure: a Phase III, open-label, single-group extension study investigating SER120 in patients with nocturia, with mean serum sodium change designated as the primary outcome through Week 40. It also reports selected adverse-event counts and serious-event terms. These are useful details about what was registered and what safety entries were posted.

The record does not report numerical results for the primary endpoint. Without baseline and follow-up means, changes, dispersion measures and uncertainty estimates, the direction and size of serum sodium changes cannot be judged. The scheduled assessments do not establish that values rose, fell, remained stable, or differed meaningfully from baseline.

Nor does the record provide a controlled estimate of a treatment effect. The study has no comparator group, and it is open-label. A within-group pattern, if numerical results were available, would describe observations during the study but would not alone show what would have happened without SER120. The record also does not provide enough event detail to attribute serious adverse events or deaths to treatment.

General statistical context: statistical evidence concerns estimates, uncertainty, design and the assumptions behind an analysis. A study’s stated hypothesis is not a result. Its completion status does not substitute for outcome reporting, and the presence of adverse-event counts does not by itself establish safety or harm. The registry record leaves the primary endpoint’s magnitude and precision unresolved and does not support a causal conclusion about treatment.

13. Why This Trial Matters Statistically

This study is statistically notable for the distinction between a registered safety question and the results available to assess it. The record identifies mean serum sodium change over a series of visits as the primary outcome, but does not report the numerical results or any analyses. That distinction matters: a clearly described endpoint and schedule specify what was intended to be measured, but they do not show the observed pattern or its uncertainty.

The single-group, open-label design also sets important limits on interpretation. It can describe observations among participants receiving SER120, but it does not provide a randomized comparison that isolates a treatment effect. The safety hypothesis, study status and recorded event counts should therefore be read in the context of the design rather than as causal findings.

The record further illustrates why event summaries require context. Counts and listed percentages are informative as registry entries, but details about event timing, treatment relationship, follow-up and the relationship among event categories are not reported. Similarly, the endpoint’s repeated schedule raises questions about analysis across visits, missing measurements and multiplicity that cannot be resolved from the record.

These limitations do not erase the descriptive value of the study record. They identify what it documents—the population and intervention context, the intended laboratory outcome, and selected safety fields—and what remains unavailable for statistical assessment: numerical endpoint estimates, uncertainty, analysis methods and key details needed to interpret safety events.

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 principal limitation for statistical interpretation is that numerical results for the registered primary endpoint are not reported in the registry record. There are no baseline or follow-up means, changes from baseline, standard deviations, confidence intervals or p-values. Although the endpoint is scheduled through Week 40, the record does not show the observed values at any visit.

The single-group, open-label design offers no concurrent control comparison. It therefore cannot separate a within-group change from other changes over time or establish a causal treatment effect. The record does not report details of how SER120 was administered beyond the group label, or how adherence was assessed.

Several analysis details are also absent: the method for calculating change, the number contributing data at each visit, handling of missing measurements, the statistical model, and any approach to multiple time points. The safety population is named, but its operational definition is not reported. The record lists adverse-event counts and terms but does not provide event timing, treatment attribution, definitions, or explanations of how serious events, other adverse events and deaths relate to one another.

Finally, the registry lists one outcome measure and no analyses. It does not report a statistical plan, sample-size justification, interim analyses, or a detailed account of how the posted safety information was collected. These limitations mean the record can describe the planned endpoint and selected safety entries, but cannot support a full evaluation of endpoint precision or a causal interpretation of the safety observations.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Long Term Safety Assessment of SER120 in Patients With Nocturia — NCT00981682.
• Registry record last updated 2021-01-20; status last verified 2014-01.
• Results first posted 2021-01-20.
• 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 record describes a completed Phase III, open-label, single-group extension study of SER120 nasal spray formulations in patients with nocturia who completed one of two named SER120 studies. Enrollment is listed as 376. The study’s primary outcome is mean serum sodium change from baseline at scheduled assessments through Week 40, in the safety population.

The record lists one outcome measure and no analyses. Numerical results for the primary endpoint are not reported in the registry record. Safety entries include 30 affected by serious adverse events among 376 at risk in the SER120 750 mcg group, 305 affected in the other adverse-event fields among 376 at risk, and 30 deaths. The record also lists selected serious-event terms and percentages, without sufficient detail to determine timing, treatment relationship or how the event categories relate to each other.