A Study to Evaluate Participants Satisfaction, Quality of Life and Effectiveness of Flexible-Dose of Paliperidone Extended Release (ER) in Participants With Schizophrenia, Previously Treated With Risperidone: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study to Evaluate Participants Satisfaction, Quality of Life and Effectiveness of Flexible-Dose of Paliperidone Extended Release (ER) in Participants With Schizophrenia, Previously Treated With Risperidone (NCT01010776), a na phase 4 study. The primary outcome is Change From Baseline in Positive and Negative Syndrome Scale (PANSS) Total Score at Week 26 - Main Phase.

2. Clinical Question

3. Trial Design

This completed Phase 4 study evaluated a switch from risperidone to flexible-dose paliperidone ER in participants with schizophrenia. The official title describes stable but symptomatic outpatients and emphasizes patient satisfaction and quality of life. The brief description further states that participants had not been satisfied with their prior treatment. Enrollment was 223.

The design is listed as single-group, with allocation recorded as not applicable and masking recorded as none. Participants received paliperidone ER; there was no concurrent control arm described in the registry record. As a result, the study describes outcomes during treatment but does not provide a randomized, like-for-like comparison against continuing risperidone or another treatment. Changes over time cannot be separated from all other possible influences using a control-group contrast.

The intervention was oral paliperidone ER once daily, at a flexible dose ranging from 3 to 12 mg. The main phase lasted 26 weeks. Participants who continued into the extension phase could receive treatment for an additional 26 weeks. The registry record does not report detailed criteria for continuing into the extension or a complete account of dose adjustment procedures.

The study started in 2008-02, reached primary completion in 2009-12, and is listed as completed. The record lists 18 outcome measures, including PANSS response, PANSS subscales, personal and social performance, and sleep quality. Several outcomes are described at multiple time points in the main phase and the main phase plus extension. The registry record does not report the statistical analysis plan, a sample-size justification, or details of interim analyses.

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.

Main Phase

Reasons for not completing this period:

In-Between Main Phase & Extension Phase

Reasons for not completing this period:

Extension Phase

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The intent-to-treat for effectiveness (ITTe) population included all the participants who received at least 1 dose of study medication and provided at least 1 post-baseline effectiveness measurement. Last Observation Carried Forward (LOCF) method was used.

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 Change From Baseline in Positive and Negative Syndrome Scale (PANSS) Total Score at Week 26 - Main Phase, measured over Baseline and Week 26.

The primary endpoint was change from baseline in total score on the Positive and Negative Syndrome Scale (PANSS) at Week 26 of the main phase. PANSS is a clinician-rated measure designed to assess symptoms of schizophrenia. It covers 30 symptoms, including delusions, grandiosity, blunted affect, poor attention, and poor impulse control. Each item is rated on a scale from 1, indicating absent, to 7, indicating extreme psychopathology. The total score ranges from 30 to 210, and higher scores indicate worsening.

A change score compares a participant’s later measurement with the baseline measurement, so it describes movement in the measured symptom scale over the treatment period. A group mean summarizes the average change, while a standard deviation describes how dispersed individual changes are around that average. Neither statistic alone shows how certain the estimated mean change is; that requires an uncertainty measure such as a confidence interval, which is not reported in the registry record.

PANSS is used in this study because the primary question concerns symptom severity in schizophrenia. The registry record specifies the endpoint, scale interpretation, and analysis population, but does not report the mean change or its standard deviation. The direction and magnitude of the primary outcome therefore cannot be interpreted from the available results.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a mean change describe?

The primary outcome is continuous: each participant has a PANSS total score at baseline and a later score, and the analysis concerns change between those measurements. A group mean change summarizes the average movement in scores. It can be useful for describing the group’s overall course, but it does not show whether every participant changed in the same direction. A standard deviation describes the spread of observed values around the mean. It is a measure of variability, not the uncertainty of the mean itself.

The registry record specifies the mean as the outcome summary and standard deviation as the dispersion measure. It does not give the resulting mean change or standard deviation. A confidence interval for the mean change, if reported, would add information about precision. It is not reported in the registry record, and no inferential test result is available in the registry record described for this study.

What does the scale contribute?

PANSS is composed of 30 symptom ratings. Each item has a seven-point response scale, from 1 for absent to 7 for extreme psychopathology; the sum ranges from 30 to 210. Higher totals indicate worsening. This direction is important when interpreting any change estimate: the sign of a change has meaning only in relation to the scale’s direction and the way change was defined. The registry record does not give a numerical estimate to interpret in this way.

A continuous scale retains more gradation than a simple classification into response or no response. The registry record also lists secondary outcomes based on percentage of participants with treatment response in PANSS total score. A response outcome converts individual changes into a category using a response definition. That can be easier to describe, but it depends on the chosen threshold and discards some information about the size of individual changes. The response threshold is not reported in the registry record.

What does the analysis population mean?

For the primary effectiveness outcome, the registry record defines the intent-to-treat for effectiveness population as participants who received at least one dose of study medication and provided at least one post-baseline effectiveness measurement. This definition does not include all enrolled participants automatically: having a post-baseline measurement is part of the stated criterion. The record does not report the number included in this primary analysis or a participant flow explaining how many enrolled participants met the criterion.

Using an analysis population with at least one follow-up measurement means the endpoint estimate depends on participants with observed post-baseline information and on the method used to handle missing follow-up values. The record states that last observation carried forward (LOCF) was used. Under LOCF, a participant’s most recent available observation is carried forward to represent a later measurement when the later value is missing. This approach can keep participants with incomplete follow-up in an analysis, but it treats the last observed value as if it remained unchanged afterward. That assumption may not reflect the participant’s actual later course.

Why does the single-group design matter?

A treatment comparison is strongest when groups provide a credible estimate of what would have happened under different assigned treatments. This study is listed as single-group, with allocation not applicable and no masking. There is no concurrent comparison group described. Consequently, an observed before-and-after difference would combine any treatment-period change with other influences that may also affect measurements over time. The registry record does not provide an adjusted or randomized comparison that could distinguish those influences.

Masking can reduce the influence of expectations on assessments or reporting, depending on the outcome and study procedures. Masking is listed as none. This fact does not establish that any particular assessment was biased, but it is relevant context when considering subjective measurements such as symptom ratings and satisfaction outcomes. The registry record does not describe additional procedures intended to limit assessment influence.

Why can over-dispersion matter?

In general, over-dispersion means that observed variability is greater than a statistical model expects. It matters when a model assumes a particular degree of spread: if the actual data are more variable, standard errors may be too small and uncertainty may be understated unless the analysis accounts for the extra dispersion. This is a general statistical consideration, not a reported finding from this trial. The registry record does not describe a count-data model, a dispersion assessment, or a method for handling over-dispersion.

What is known about statistical planning?

The registry record does not report a detailed statistical analysis plan, the rationale for the planned enrollment, or interim analyses. The sample-size illustrations associated with the results are planning illustrations based on an observed effect, not evidence of the study’s original design or its achieved power. Their assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those assumptions do not describe this single-group study’s allocation, and the illustrations must not be interpreted as observed power.

The main-phase endpoint is specified at Week 26, while several secondary outcomes are described at multiple visits and across the extension. The registry record does not specify how repeated measurements were modeled, whether any covariates were used, how multiplicity was addressed, or what criteria were applied to select final analyses. Without those details and the outcome estimates themselves, the statistical analysis cannot be independently reconstructed from the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Paliperidone Extended Release (ER) - Main Phase")
n   <- c(213)
m   <- c(-22.9)
sp  <- c(20.1)
sd  <- sp      # posted standard deviations
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Paliperidone Extended Release (ER) - Main Phase  213  -22.900  20.100  -25.615  -20.185

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists 18 outcome measures. They span PANSS total-score response, PANSS subscale changes, personal and social performance, and sleep quality, with several outcomes assessed at multiple visits during the main phase and the extension. When many outcomes or time points are examined, the chance of at least one apparently notable result can increase if each is tested separately. Statistical approaches can address this issue by defining a primary comparison in advance, adjusting inference, or interpreting secondary findings as exploratory. The registry record does not report a multiplicity strategy, so it is not possible to determine whether or how the analyses accounted for the range of outcomes.

The primary endpoint is identified as change in PANSS total score at Week 26 of the main phase. However, the registry record does not report the primary mean change, standard deviation, confidence interval, or p-value. It also does not report the results for the listed secondary outcomes in the registry record. Therefore, neither the pattern across endpoints nor the consistency of findings over time can be assessed.

For the primary analysis population, participants needed at least one dose and at least one post-baseline effectiveness measurement. The stated missing-data method was LOCF. Carrying the last observed score forward is a particular assumption about unobserved later values; it does not reveal what those values would have been. The registry record does not describe alternative missing-data analyses, sensitivity analyses, or the amount and pattern of missing outcome data. It is therefore not possible to judge whether conclusions would be robust to different assumptions about missingness.

The registry record also does not report a complete analysis plan or interim-analysis details. These omissions leave uncertainty about prespecification and the handling of multiple measurements. They do not establish that any particular procedure was or was not used outside what is recorded.

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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: psychotic disorder nos (2.3%), schizophrenia nos (0.9%), hospitalization (0.9%), nasal septum deviation (0.6%), cholecystectomy (0.6%).

For the main phase, the registry lists 14 participants affected by serious adverse events among 215 at risk, and 144 affected by other adverse events among 215 at risk. For the extension phase, it lists 3 affected by serious adverse events among 159 at risk, and 70 affected by other adverse events among 159 at risk. These are phase-specific entries; the extension group is not the same-sized at-risk group as the main-phase group. The registry record does not explain the difference in denominators, so it should not be read as a direct comparison of risk between phases.

The most frequently listed serious adverse event terms include Psychotic disorder NOS at 2.3%, Schizophrenia NOS at 0.9%, Hospitalization at 0.9%, Nasal septum deviation at 0.6%, Cholecystectomy at 0.6%, and Pregnancy NOS at 0.6%. The registry record does not report deaths. These entries describe events recorded during the study, not evidence that the study treatment caused them. Because there is no concurrent control group, the counts do not establish how event occurrence compares with another treatment or with no study treatment.

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

This study addresses the experience of participants with schizophrenia who had previously taken risperidone and were described as dissatisfied with treatment. It evaluates flexible-dose paliperidone ER in a single-group design, with a main phase and an extension for participants who continued. The registry record identifies a primary symptom-scale outcome and lists additional measures of symptoms, functioning, sleep, and treatment response.

The registry entries do not include the primary mean change in PANSS total score or its standard deviation. They also do not include a confidence interval or p-value for that endpoint. Consequently, the registry record cannot show how much the average score changed, how variable participant changes were, or how precise an estimated change might be. The endpoint description alone is not evidence of an observed benefit or harm.

The absence of a concurrent comparator matters independently of the missing outcome estimate. In a single-group study, a change from baseline cannot isolate the effect of treatment from other changes over time. Even a reported average change would describe participants’ observed course under the study conditions, not a randomized difference between paliperidone ER and continued risperidone.

The safety counts offer descriptive information about recorded adverse events in each phase. They do not establish comparative safety, and the different at-risk group sizes make direct phase-to-phase comparisons especially difficult without more information about who entered or remained in each phase. The registry record does not report deaths.

Thus, the record documents the study’s design, outcome definitions, analysis population, LOCF method, and selected safety counts, but leaves important efficacy results and analysis details unreported. The study question can be understood, while the treatment effect and its uncertainty cannot be evaluated from the registry information.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the difference between specifying an outcome and reporting its result. The registry record gives a clear primary measurement: PANSS total-score change from baseline to Week 26, along with the scale’s range and direction. Yet it does not report the estimated change or its variability. Without those results, the stated endpoint cannot support an assessment of effect magnitude or precision.

The single-group design also makes the target of inference important. It can describe what happened among participants receiving paliperidone ER, but it does not create a concurrent estimate of what would have happened under continued risperidone or a different treatment. This distinction is central to interpreting change-from-baseline results: a within-group change and a between-treatment effect answer different questions.

The record illustrates how analysis choices shape interpretation. The primary population requires a post-baseline effectiveness measurement, and missing values were handled using LOCF. That method uses the last observed value to stand in for a later missing measurement. The method and its assumptions should be considered alongside the outcome estimate, but the registry record does not report the amount of missingness or sensitivity analyses.

There are also multiple listed outcomes and repeated assessment times, while the registry record does not state how multiplicity was managed. Together, these features show why outcome definitions, analysis populations, missing-data handling, and the statistical plan are needed to assess reported findings. The available record supplies some of that framework, but not the primary result or enough analysis detail for a complete statistical appraisal.

15. Limitations

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

The most important limitation for interpreting effectiveness is that the registry record does not report the primary PANSS change estimate, standard deviation, confidence interval, or p-value. The endpoint and its direction are described, but no numerical outcome result is available in the registry record for this analysis. The listed secondary outcomes also lack reported results here, preventing an assessment of whether findings were consistent across symptom, functioning, sleep, and response measures.

The study is single-group, unmasked, and has no concurrent comparator described. Therefore, any change over time could not be interpreted as a randomized contrast against another treatment. The record does not report procedures beyond the stated design that might address this limitation.

The primary effectiveness population includes participants with at least one dose and at least one post-baseline measurement, and LOCF was used for missing observations. The registry record does not report the number included in the primary analysis, the amount or pattern of missing data, or sensitivity analyses using other assumptions. These gaps limit assessment of how missing measurements may affect the result.

The record lists multiple outcomes and time points but does not report a multiplicity approach or detailed analysis plan. It also does not report a sample-size justification or interim-analysis details. For the extension phase, at-risk denominators differ from those in the main phase, and the record does not explain participant flow into or through the extension. These issues limit interpretation of extension safety counts and any extension outcomes.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study to Evaluate Participants Satisfaction, Quality of Life and Effectiveness of Flexible-Dose of Paliperidone Extended Release (ER) in Participants With Schizophrenia, Previously Treated With Risperidone — NCT01010776.
• Registry record last updated 2013-11-20; status last verified 2013-09.
• Results first posted 2013-11-20.
• 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.

19. Record Summary

This completed Phase 4 study enrolled 223 participants with schizophrenia previously treated with risperidone. It evaluated oral paliperidone ER at a flexible dose ranging from 3 to 12 mg once daily, with a 26-week main phase and an additional 26-week extension for participants who continued.

The study is listed as single-group, with allocation not applicable and no masking. Its primary endpoint was change from baseline in PANSS total score at Week 26. The stated primary analysis population required at least one dose and at least one post-baseline effectiveness measurement; LOCF was used for missing observations.

The registry record reports selected adverse-event counts for the main and extension phases but does not report the primary PANSS result, its precision, a detailed statistical analysis plan, or a multiplicity strategy. The study therefore defines its main question and some methods, but the primary treatment-associated change cannot be evaluated from the reported information.