Symptomatic Remission and Social Function in Participants Treated With Paliperidone Extended Release (ER): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Symptomatic Remission and Social Function in Participants Treated With Paliperidone Extended Release (ER) (NCT01577186), a na phase 4 study. The primary outcome is Percentage of Participants Achieving Symptomatic Remission by Means of Positive and Negative Syndrome Scale (PANSS).

2. Clinical Question

The clinical question is therefore descriptive and exploratory: how many participants in the treated group met the stated remission definition, and how did remission status relate to functioning? The registry record identifies a secondary outcome, the Social Functioning Scale (SFS) score, but does not provide its result. Without a comparison group, the record cannot establish how outcomes would differ under another treatment or without treatment. It also does not report results that would quantify whether remission and functioning were associated.

3. Trial Design

This was a phase 4, single-group study of paliperidone ER in participants with schizophrenia. The record lists enrollment of 480 and describes flexible dosing. Allocation is listed as not applicable, masking as none, and the model as single group. There is no listed control arm, so the design supports description of outcomes in the treated group rather than a controlled estimate of treatment effect.

The study start is recorded as 2008-07, and primary completion as 2009-06. The primary endpoint timeframe is end of study, up to Week 12. The record defines the primary analysis population as participants who received at least 1 dose of study drug and had at least one post-baseline assessment. It does not report the number included in that population or the number contributing to the endpoint result.

The stated purpose was to explore the relationship between symptomatic remission, measured using specified PANSS items, and personal and social functioning measured by the PSP scale. The primary outcome is the percentage achieving remission. The record lists SFS score as a secondary outcome. Although it identifies 5 outcome measures, it lists 0 analyses and supplies no numerical outcome estimates in the record summarized by these registry entries. The precise statistical plan, including how functioning and remission were to be related, is not reported in the registry record.

The study is marked completed. Completion status does not itself show that every planned outcome was analyzed or reported. With no concurrent comparator and no masking, interpretation rests on the definition and reporting of outcomes in the treated group. The record does not describe a randomization process, a dose comparison, or a blinded assessment procedure.

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: Intent-to-treat (ITT) population included all participants who received at least 1 dose of study drug and had at least one post-baseline assessment.

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 Percentage of Participants Achieving Symptomatic Remission by Means of Positive and Negative Syndrome Scale (PANSS), measured over End of study (Up to Week 12).

The primary endpoint is the percentage of participants achieving symptomatic remission by the end of the study, up to Week 12. Remission is defined by all 8 specified PANSS items reaching an intensity level of mild or moderate. Those items are delusions, unusual thought content, hallucinatory behavior, conceptual disorganization, mannerisms/posturing, blunted affect, social withdrawal, and lack of spontaneity.

PANSS is a 30-item scale used to assess neuropsychiatric symptoms of schizophrenia. It includes positive, negative, and general psychopathology subscales; each item is scored from 1 (absent) to 7 (extreme). The total score ranges from 30 to 210, with higher scores indicating greater severity. The remission definition focuses on a specified subset of symptoms rather than requiring a particular total score.

A percentage endpoint expresses how common the defined status was among the participants assessed for that outcome. The registry record does not report the observed percentage, its 95% confidence interval, or the endpoint denominator. Consequently, the definition is available, but the numerical frequency and its precision cannot be evaluated from this record.

The study’s stated interest in functioning gives the remission endpoint a broader context: it was intended to be considered alongside personal and social functioning. However, the record does not report a numerical PSP result or an analysis relating remission status to functioning.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a percentage endpoint represent?

A percentage endpoint expresses the fraction of an analysis population meeting a defined condition. Here, the condition is symptomatic remission according to the stated PANSS criteria. For the percentage to be interpretable, the numerator must identify participants who met the definition, and the denominator must identify those assessed for the endpoint. The registry record gives neither the resulting percentage nor the endpoint denominator. The enrollment figure alone does not establish that every enrolled participant contributed to the endpoint.

What does a confidence interval add?

A confidence interval describes the uncertainty associated with an estimate under the statistical method used. It is not a range that guarantees the true value lies within its endpoints, nor does it show the likely result for an individual participant. For a percentage, interval width is affected by the amount of information in the data and by the estimation method. The primary outcome specifies a 95% confidence interval, but the interval itself is not reported in the registry record. Its width and the precision of the remission estimate therefore cannot be assessed.

How should the PANSS definition be read?

PANSS assesses neuropsychiatric symptoms with 30 items, organized into positive, negative, and general psychopathology subscales. Each item ranges from 1 (absent) to 7 (extreme), while the total score ranges from 30 to 210; a higher score indicates greater severity. The primary endpoint does not define remission as reaching a threshold on the total score. Instead, it requires all 8 named items to be at an intensity level of mild or moderate. This distinction matters: an overall total can combine information across many items, whereas the remission definition is a specific status based on a selected set.

What can a single-group design estimate?

A single-group study can describe outcomes observed in its treated participants, provided the outcome data and analysis are reported. It cannot make a controlled comparison when no comparator group is present. In a comparison study, a difference between groups can help distinguish an outcome pattern associated with one assigned condition from the pattern in another group, subject to the design and analysis. That contrast is not available in this record. A before-and-after pattern, if reported, would still not on its own show what would have occurred in the absence of treatment.

Allocation is listed as not applicable, masking as none, and the model as single group. Those design characteristics limit the kinds of statistical claims that can be made. They do not prevent a descriptive estimate, but the record must report that estimate and its uncertainty for readers to judge its precision. Neither the remission estimate nor a numerical association with functioning is reported.

What does the planning language mean?

The registry’s planning assumptions describe illustrations based on an observed effect, not the trial’s own design. They specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. Such calculations ask what sample size might be needed under chosen assumptions for a planned comparison; they are not an estimate of the probability that this completed study detected an effect. They are never observed power. Here, equal allocation between two arms does not match the listed single-group design. The registry record does not report the trial’s sample-size justification or statistical plan.

General statistical context: power calculations depend on assumptions about the effect to detect, variability, significance threshold, allocation, and analysis. If those assumptions differ from the eventual design or data, the resulting planning figure may not describe the study’s actual ability to estimate its endpoint. A planning illustration cannot substitute for a reported estimate and confidence interval. No planning result should be read as evidence about the observed remission percentage.

Why does the analysis population matter?

The stated intent-to-treat population includes participants who received at least 1 dose and had at least one post-baseline assessment. The record does not give the size of this population, explain whether the endpoint analysis used exactly that population, or describe how missing endpoint assessments were handled. Different populations or missing-data approaches can produce estimates with different interpretations. Without these details and the endpoint result, readers cannot determine how much information contributed to the primary estimate.

General statistical context: missing outcome data can reduce precision and can also affect interpretation if the availability of measurements is related to participants’ outcomes. Statistical methods for missingness rely on assumptions; the handling method and relevant assumptions should therefore be described. The registry record does not report such methods for this endpoint.

How should multiple outcomes be considered?

The record lists 5 outcome measures and 0 analyses. Multiple outcomes can create multiple opportunities to observe patterns that may arise by chance, especially when several statistical tests are conducted. A prespecified analysis plan and a stated multiplicity strategy help readers understand which results are primary and how to interpret statistical evidence across outcomes. The record does not report a multiplicity procedure, a list of analysis specifications, or p-values. No adjustment or unadjusted inference can be reconstructed from the outcome definitions alone.

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("Paliperidone Extended Release (ER)")
n      <- c(426)
events <- c(50)
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  Paliperidone Extended Release (ER)  426  50  11.7  8.8  15.2  9.0  15.1  9.0  15.1

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 5 outcome measures and 0 analyses. It identifies symptomatic remission as the primary outcome and SFS score as a secondary outcome, while the study purpose describes exploring the relationship between remission and personal and social functioning. However, the record does not report numerical analyses for these outcomes or specify which comparisons or associations were planned.

When a study evaluates multiple outcomes or conducts multiple tests, the chance of finding at least one apparently notable result can increase. A prespecified statistical plan and a stated multiplicity strategy clarify how the family of analyses is handled. The multiplicity approach is not reported in the registry record. No p-values or analysis details are listed, so the record does not permit assessment of whether any multiplicity adjustment was used.

The primary endpoint population is defined as participants who received at least 1 dose and had at least one post-baseline assessment. The number meeting that definition and the denominator used for the endpoint are not reported. The record also does not state how missing post-baseline measurements, missing PANSS items, or other incomplete endpoint data were handled. It does not describe imputation, exclusions beyond the population definition, or sensitivity analyses.

These omissions affect interpretation of both precision and potential selection. General statistical context: when outcome information is missing, the assumptions and methods used to address it can matter, particularly if missingness is related to participants’ outcomes. The record does not report enough detail to judge those assumptions. The analysis population definition is available, but the actual analyzed population and missing-data procedures are not reported in the registry record.

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 2% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: schizophrenia (5.6%), hallucination, auditory (0.4%), supraventricular tachycardia (0.2%), pulmonary tuberculosis (0.2%), femur fracture (0.2%).

For the paliperidone ER group, the adverse-event record lists 480 participants at risk for serious events and 37 listed under serious adverse events. It separately lists 480 at risk for other adverse events and 169 listed under that category. These are counts recorded in the registry; they do not provide a comparison with another treatment group.

The listed serious-event terms include Schizophrenia (5.6%), Hallucination, Auditory (0.4%), Supraventricular Tachycardia (0.2%), Pulmonary Tuberculosis (0.2%), Femur Fracture (0.2%), and Aggression (0.2%). The record does not provide further detail in the registry record about the denominator or calculation underlying each term percentage, event timing, or participant-level overlap across terms. These percentages should be read as registry-listed values, not as evidence that an event was caused by treatment.

The record does not report deaths in this group. Counts of events or participants listed under adverse-event categories are descriptive safety information. With no control group, they cannot establish whether the frequency was higher or lower than it would have been under another condition. The registry record does not supply a comparative safety estimate or causal attribution for these events.

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

The result that can be interpreted from the registry record is primarily the study’s design and the definition of its remission endpoint. Participants were treated in a single group with flexibly dosed paliperidone ER, and the primary outcome was defined using 8 specified PANSS items at end of study, up to Week 12. The record does not report the numerical remission percentage or its 95% confidence interval. It therefore does not show how frequently the defined remission status was observed or how precisely that frequency was estimated.

The study’s stated aim also concerned the relationship between symptomatic remission and functioning. The PSP measure appears in the purpose, and SFS score is listed as a secondary outcome. Their numerical results and an analysis of their relationship to remission are not reported in the registry record. The stated aim should not be mistaken for a reported association.

The design does not include a comparator group, so it does not provide a controlled estimate of how outcomes differ between paliperidone ER and another treatment or no treatment. The record’s lack of masking is also relevant context when considering how outcomes were assessed, but the registry entries do not describe assessment procedures in further detail. No causal conclusion follows solely from observing outcomes in a treated group.

The adverse-event counts and listed event-term percentages describe entries for the treatment group. They do not establish that treatment caused an event, nor do they provide comparative risk estimates. The record also does not report a numerical endpoint analysis that would allow the clinical question to be judged by its precision or statistical uncertainty.

General statistical context: a study’s endpoint definition, its estimated result, and the uncertainty around that result answer different questions. A well-defined outcome alone does not reveal its observed value. Similarly, a planning calculation is not an observed result or observed power. For this record, the defined remission outcome and design are available, while the main numerical efficacy result is not reported.

14. Why This Trial Matters Statistically

This trial is statistically instructive because its question connects a symptom-based status with social functioning, while its primary endpoint is a defined percentage in a single treated group. The distinction between an outcome definition and an outcome estimate is central: the registry provides a detailed rule for classifying remission but does not report the resulting percentage or confidence interval. Without those values, neither the frequency nor the precision of remission can be assessed from the record.

Its design also illustrates the limits of a single-group study. A descriptive estimate can characterize participants who received treatment, but a treatment comparison requires an appropriate comparator. The record lists no such group. Readers therefore need to distinguish describing what was observed in this group from attributing a difference to treatment. Completion status and enrollment do not supply the missing comparison or endpoint result.

The endpoint definition highlights that clinical scales can be used in different ways. PANSS includes a total score and subscales, but the remission criterion is based on all 8 designated items meeting the stated intensity level. That operational definition determines who counts as having achieved the endpoint; it should not be conflated with a total-score threshold.

Finally, the record illustrates why analysis populations, missing-data methods, and multiplicity plans matter. The intended population is defined, but its analyzed size and the handling of missing measurements are not reported. Multiple outcomes are listed, yet the analysis details and multiplicity approach are absent. These reporting gaps constrain how confidently a reader can interpret an outcome, even when the study question and endpoint are clearly stated.

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 primary limitation for interpreting efficacy is that the registry record does not report a numerical primary endpoint result or its 95% confidence interval. It also does not report the endpoint denominator, so the enrollment figure cannot be assumed to represent the analysis population. The record defines the intent-to-treat population as participants who received at least 1 dose and had at least one post-baseline assessment, but does not report its size or confirm the number included in the primary analysis.

The single-group design has no concurrent comparator, and masking is listed as none. This prevents a like-for-like treatment comparison and limits causal interpretation. The registry record does not report a numerical analysis of the relationship between remission and functioning, despite describing this as a study aim. It lists SFS score as a secondary outcome, but does not report its result.

Further endpoint-specific limitations include the absence of reported missing-data methods, sensitivity analyses, analysis specifications, and a multiplicity strategy. Although 5 outcome measures are listed, the record lists 0 analyses. It is therefore not possible to assess how incomplete assessments or multiple outcomes were addressed.

The adverse-event entries are descriptive and lack a comparison group. The record does not provide further detail about event timing, term-level denominators, or causal attribution. The stated planning assumptions describe a two-arm illustration and do not match the listed single-group design; the trial’s own sample-size justification and statistical plan are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Symptomatic Remission and Social Function in Participants Treated With Paliperidone Extended Release (ER) — NCT01577186.
• Registry record last updated 2014-01-17; status last verified 2013-12.
• Results first posted 2014-01-17.
• 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

This completed phase 4 study enrolled 480 participants with schizophrenia and examined flexible-dose paliperidone ER in a single-group design. Allocation is listed as not applicable and masking as none. The primary endpoint was the percentage achieving symptomatic remission by PANSS criteria at end of study, up to Week 12.

The stated intent-to-treat population included participants who received at least 1 dose and had at least one post-baseline assessment. The record lists SFS score as a secondary outcome and describes exploring the relationship between remission and personal and social functioning. It lists 5 outcome measures and 0 analyses.

The primary remission percentage, its 95% confidence interval, functioning results, and statistical analysis details are not reported in the registry record. Safety entries include 37 listed under serious adverse events and 169 under other adverse events, among 480 at risk in each category; these are descriptive counts without a comparator.