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 and Efficacy Study of Paliperidone Extended Release (ER) Among Thai Schizophrenia Participants (NCT01387542), a na phase 4 study. The primary outcome is Change From Baseline in Clinical Global Impression Severity (CGI-S) Scale at Week 10.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01387542 |
| Phase | Phase 4 |
| Condition | Schizophrenia |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 40 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Janssen-Cilag Ltd.,Thailand |
| Results posted | Yes |
2. Clinical Question
Population
Thai participants with schizophrenia were eligible for the study. The registry describes the primary analysis population as the Intent-To-Treat population: participants who received at least 1 dose of study medication and had at least one post-baseline efficacy evaluation. The registry enrollment is 40; the number included in the primary endpoint analysis is not reported in the registry record.
Intervention
Participants received oral paliperidone extended release once daily for 10 weeks. The listed doses were 3 milligram (mg), 6 mg, 9 mg, or 12 mg, with dose selection at the investigator's discretion. This design describes treatment during the study, not a randomized assignment to different doses.
Comparator
There was no control group in the single-group design. Allocation is listed as not applicable, and masking as none. As a result, the registry record does not offer a like-for-like comparison with placebo, usual care, or another treatment. Changes over time cannot be separated from other influences using a concurrent control group.
Primary question
The primary endpoint asks how the Clinical Global Impression Severity (CGI-S) score changed from baseline to Week 10. CGI-S is a clinician-rated assessment of illness severity, with higher scores indicating worsening. The registry record names the endpoint and its timeframe, but the numerical mean change, its variability, and an uncertainty interval are not reported in the registry record. Therefore, the direction and precision of the observed change cannot be assessed from the primary endpoint information recorded.
3. Trial Design
This was a Phase 4, open-label, prospective, single-group study of paliperidone extended release (ER) in Thai participants with schizophrenia. The record lists Janssen-Cilag Ltd.,Thailand as sponsor, a start date of 2009-08, primary completion in 2010-09, and completed status. Enrollment is recorded as 40. These registry entries describe the study record; they do not establish how many participants contributed data to each endpoint.
Participants received paliperidone ER oral tablets once daily for 10 weeks. The listed dose options were 3 milligram (mg), 6 mg, 9 mg, or 12 mg, selected at the investigator's discretion. The record does not describe a randomized dose allocation. With no concurrent control group and no masking, changes in clinical ratings were observed in an open-label treatment setting. This design can describe outcomes recorded during treatment, but it cannot by itself distinguish treatment-related change from natural variation, expectation, concurrent care, or other influences over time.
The primary endpoint was change from baseline in CGI-S at Week 10, reported as a mean with standard deviation. The specified population was the Intent-To-Treat (ITT) population, defined in the record as participants who received at least 1 dose and had at least one post-baseline efficacy evaluation. The number meeting this definition and the number analyzed for CGI-S are not reported in the registry record.
The registry lists four outcome measures and four analyses, but the other outcome details are not reported in the registry record. The protocol, eligibility criteria beyond the described population, dose-adjustment procedures, visit schedule beyond the primary timeframe, and statistical plan are not reported in the registry record. No conclusions about those aspects should be inferred from the design description alone.
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 | Paliperidone Extended Release (ER) |
|---|---|
| STARTED | 40 |
| Treated | 38 |
| COMPLETED | 32 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Paliperidone Extended Release (ER) |
|---|---|
| Adverse Event | 3 |
| Withdrawal by Subject | 1 |
| Changed diagnosis | 1 |
| Insufficient response | 1 |
| Non-compliant | 1 |
| Lost to Follow-up | 1 |
Analysis population for the primary outcome, as defined in the registry: Intent-To-Treat (ITT) population included participants who received at least 1 dose of study medication and had at least one post-baseline efficacy evaluation.
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.
| Characteristic | Unit | Paliperidone Extended Release (ER) |
|---|---|---|
| Participants analysed | 38 | |
| Age Continuous | Years | 41.13 (SD 12.314) |
| Sex: Female, Male: Female | Participants | 23 |
| Sex: Female, Male: Male | Participants | 15 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Clinical Global Impression Severity (CGI-S) Scale at Week 10, measured over Baseline, Week 10.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Units on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is change from baseline in Clinical Global Impression Severity (CGI-S) at Week 10. CGI-S is a clinician's global assessment of the severity of illness. The scale description in the registry ranges from 1, “Normal, not at all ill,” to 7, “Among the most extremely ill participants.” Higher scores indicate worsening. The endpoint therefore represents a change in a clinician-rated severity assessment over the stated study period, rather than a direct measurement of a single symptom or a participant-reported rating.
Change from baseline is commonly used to describe how a measurement differs from its starting value. A mean change summarizes the average change among the participants included in the analysis; the standard deviation describes variability in individual changes or values, depending on how the analysis was defined. The registry identifies the endpoint as a mean with standard deviation and gives the timeframe as baseline and Week 10. It does not report the mean change, standard deviation, confidence interval, or exact analysis method in the record.
Using a clinician-rated severity scale can provide a broad assessment of illness severity. However, interpreting a change depends on the observed estimate, its uncertainty, the analysis population, and how missing assessments were handled. Those numerical results and procedures are not reported in the registry record, so the endpoint definition alone does not establish the size, direction, or precision of any change.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record identifies a clinically interpretable primary measure: change in CGI-S from baseline to Week 10. It also specifies that higher CGI-S scores indicate worsening and that the endpoint is summarized as a mean with standard deviation. These details explain what the outcome was intended to represent, but they are not the outcome result. The mean change, standard deviation, and any confidence interval or test result are not reported in the registry record.
Consequently, the numerical direction of the primary endpoint cannot be established from the registry information. The record does not show whether the mean score increased, decreased, or remained similar, nor does it show how variable participant-level observations were. Without an estimate and an uncertainty measure, it is not possible to judge how precisely the primary change was measured. No statement that the endpoint improved or worsened is warranted from the endpoint description alone.
The open-label, single-group design also limits what a change over time could establish, even if a numerical result were available. There is no concurrent control group against which to compare the change. A before-and-after difference can reflect multiple influences, including changes unrelated to study treatment. Since allocation is listed as not applicable and masking as none, the study is not a randomized, masked comparison. The record explicitly cautions that there is no like-for-like control comparison.
The registry records enrollment as 40, but that does not establish the analysis denominator for CGI-S. The stated ITT definition requires at least 1 dose and at least one post-baseline efficacy evaluation; the number meeting the definition and the number with Week 10 data are not reported in the registry record. Missing assessments could affect the analysis, but the handling of missing data is also not reported.
Safety information is recorded separately. The paliperidone ER group lists 1 participant affected by a serious adverse event among 40 at risk, and 22 affected by other adverse events among 40 at risk. These are counts, not a comparison with another group. The listed serious adverse event terms are Mania and Elevated mood, each recorded as 2.5%. This information describes the registry entries; it does not supply a controlled estimate of treatment-related risk.
Overall, the record identifies the intended endpoint and some safety counts, but it does not provide the primary efficacy estimate or the procedures needed to evaluate its precision. Interpretation should remain limited to those recorded design and safety details.
9. Statistical Methods Explained
The registry describes the primary outcome as a continuous measure summarized by a mean and standard deviation. It does not report the numerical outcome summary, a test statistic, a confidence interval, or the inferential method used for the primary analysis. General explanations below describe how such methods are commonly interpreted; they should not be read as a description of procedures confirmed for this study.
What does a mean change describe?
For a continuous outcome, a mean change is an average of participant-level changes from baseline to the specified follow-up. It gives a concise description of the central direction and magnitude of observed change in the analyzed population. It does not show whether every participant changed in the same direction, and it can conceal variation across participants. Here, the registry names mean change in CGI-S as the primary endpoint, but does not report its numerical value. Thus, the average direction and size cannot be evaluated.
What does the standard deviation add?
In general, a standard deviation describes how spread out observations are around their mean. A larger standard deviation indicates more variability in the observed values, while a smaller one indicates less. It is not the same as a confidence interval and does not, by itself, measure the precision of the estimated mean. The registry says the primary measure is reported with standard deviation, but the value and the precise data to which it applies are not reported in the registry record.
How would a confidence interval be interpreted?
In general, a confidence interval expresses uncertainty around an estimate under the assumptions of the statistical method used. A narrow interval indicates greater precision than a wide interval, while the interval's location conveys which values are compatible with the data and method. A confidence interval is not reported for the primary endpoint in the registry record. Without an estimate and its interval, readers cannot assess the precision of the mean change or distinguish a precisely estimated change from an uncertain one.
What role would a hypothesis test play?
In general, a hypothesis test assesses how compatible observed data are with a specified statistical model or null hypothesis. A p-value is not a measure of effect size, clinical importance, or the probability that a hypothesis is true. The registry does not report a primary endpoint p-value, the null hypothesis, or the testing procedure. The statistical plan is not reported in the registry record, so no particular test should be attributed to the study.
Why does the design matter for inference?
General statistical interpretation depends on how participants enter comparison groups. A single-group study describes outcomes within the treated group over time, but does not estimate a treatment contrast against a concurrent control. Without random allocation to a control group, observed change cannot be isolated from other possible causes. Open-label conduct also means treatment assignment was not masked, as the record explicitly states. These design features are relevant to interpretation independently of the choice of summary statistic.
The record's sample-size planning description is an illustration based on the observed effect, not the trial's own design. It states assumptions of 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. For time-to-event planning, it describes total events; for other outcomes, participants per arm. These are planning conventions, not observed power or results from this single-group trial. The record does not report the values for those planning illustrations, and the two-arm planning assumptions should not be mistaken for the study's actual design.
Over-dispersion is a general issue for count outcomes: it occurs when variability exceeds what a model assumes and can make standard errors too small if ignored. The primary endpoint is identified as continuous, and no count-model method is reported. There is therefore no basis for claiming that over-dispersion was assessed or relevant to the primary analysis.
In short, the registry supplies an endpoint definition and a summary type, but not enough numerical or methodological detail to reproduce or fully assess the primary analysis. The actual statistical model, confidence interval method, hypothesis test, covariate adjustment, and any sensitivity analyses are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists four outcome measures and four analyses, while identifying change from baseline in CGI-S at Week 10 as the primary endpoint. It does not report the other outcome names, the hierarchy among outcomes, or whether formal hypothesis tests were conducted. A multiplicity strategy—such as adjustment across multiple outcomes or tests—is not reported in the registry record. Without these details, the risk of interpreting one result among several as confirmatory cannot be assessed.
The specified ITT population includes participants who received at least 1 dose and had at least one post-baseline efficacy evaluation. This definition does not include participants without any post-baseline efficacy evaluation. The number of participants included in this population, the number analyzed for the primary outcome, and the number with baseline and Week 10 CGI-S measurements are not reported in the registry record.
Missing-data procedures are not reported in the registry record. It is not stated whether missing Week 10 assessments were imputed, whether analyses used available observations, or whether sensitivity analyses were conducted. These choices can affect a mean change estimate when data are incomplete, particularly if the likelihood of missingness is related to outcome values or participant characteristics. This is a general consideration, not evidence about what occurred in this study.
The statistical analysis plan, interim analyses, and sample-size justification are not reported in the registry record. No assumptions about these procedures can be made from the stated endpoint definition or the single-group design.
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 |
|---|---|---|---|
| Paliperidone Extended Release (ER) | 1 | 40 | 2.5% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Paliperidone Extended Release (ER) | 22 of 40 |
Most frequent serious adverse event terms
| Event term | Organ system | Paliperidone Extended Release (ER) |
|---|---|---|
| Mania | Psychiatric disorders | 1 of 40 |
| Elevated mood | Psychiatric disorders | 1 of 40 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Paliperidone Extended Release (ER) |
|---|---|---|
| Drowsiness | Nervous system disorders | 6 of 40 |
| Headache | Nervous system disorders | 6 of 40 |
| Insomnia | Psychiatric disorders | 5 of 40 |
| Extrapyramidal disorder | Nervous system disorders | 3 of 40 |
| Nausea | Gastrointestinal disorders | 3 of 40 |
| Constipation | Gastrointestinal disorders | 2 of 40 |
| Diarrhea | Gastrointestinal disorders | 2 of 40 |
| Hypotension | Vascular disorders | 2 of 40 |
| Pruritis | Skin and subcutaneous tissue disorders | 2 of 40 |
| Palpitation | Cardiac disorders | 2 of 40 |
Serious events with the highest arm-level rate: mania (2.5%), elevated mood (2.5%).
The registry lists 40 participants at risk in the Paliperidone Extended Release (ER) group for serious adverse events, with 1 participant affected. For other adverse events, it lists 40 at risk and 22 affected. These figures describe participants recorded as affected in the respective categories; they are not a controlled comparison and should not be read as evidence of a difference between treatments.
The record lists Mania and Elevated mood as the top serious adverse event terms, each recorded as 2.5%. The serious adverse event group entry does not provide event-level detail, timing, severity descriptions, or an assessment of whether events were considered related to treatment. The death count is not reported in the registry record.
Adverse event counts provide a descriptive account of what was recorded, but they do not by themselves establish causation. In a single-group, open-label study, there is no concurrent control group to contextualize these counts. The registry record also does not report details about how adverse events were collected or how long participants were followed for safety beyond the stated treatment period.
12. What the Result Does, and Does Not, Mean
The registry record establishes that the study examined paliperidone ER in Thai participants with schizophrenia and designated change in CGI-S from baseline to Week 10 as the primary endpoint. It defines the scale, the direction of its scoring, the treatment schedule, and the stated ITT population. These details clarify the question the study intended to address.
The numerical primary endpoint result is not reported in the registry record. Therefore, the record does not establish the size or direction of mean change, its variability, or its precision. It is not possible to conclude from this information whether observed CGI-S scores changed, or how consistent any change was across participants. Nor does the endpoint definition alone establish clinical importance.
Even if a within-group change were available, this design would not show how outcomes compared with a concurrent control condition. The study was open-label and single-group, with no randomized comparison. A change observed between baseline and Week 10 could have several explanations, so it cannot be attributed to treatment on the basis of this design alone.
The safety record gives descriptive counts for serious and other adverse events, plus two listed serious event terms. Those entries do not establish that an event was caused by paliperidone ER, and the lack of a control group prevents a comparative safety estimate. The death count and further event details are not reported in the registry record.
Accordingly, the defensible interpretation is limited: the registry describes the study question, design, endpoint definition, and selected safety counts. It does not provide the primary efficacy result or enough information to assess its statistical uncertainty. It cannot support a treatment-versus-control conclusion or a causal claim.
13. Why This Trial Matters Statistically
This trial matters statistically because its design and endpoint make clear the distinction between describing change and estimating a comparative treatment effect. A single-group study can document how a measured score varies during a study period, but without a concurrent control group it cannot directly estimate what would have happened under another treatment or no study treatment. That distinction is essential when reading before-and-after clinical data.
The selected primary endpoint is a clinician-rated global severity measure, and the record defines its scale direction and timeframe. Such context is necessary for interpreting a mean change: a number alone would not explain what the scale measures or whether higher values indicate worsening. Here, however, the numerical estimate and its variability are not reported, illustrating that an endpoint definition is not a substitute for the result itself.
The registry also makes the analysis population definition explicit. Requiring at least one post-baseline efficacy evaluation means the stated ITT population is not simply every person enrolled. Since the analyzed count and missing-data procedures are not reported, readers cannot determine how complete the primary endpoint data were or how the analysis population differed from enrollment.
Finally, the safety entries show why event counts require context. Counts in a single treatment group can describe recorded events, but cannot establish comparative risk or causation. Clear reporting of denominators, event definitions, follow-up, analysis methods, and uncertainty would help readers interpret such figures. The record provides some of that context, but the efficacy result and several key analytic details are not reported.
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 most direct limitation is that the registry record does not report the numerical result for the primary endpoint. The mean change in CGI-S, standard deviation, and any confidence interval or test result are absent. Without these values, the observed outcome and its precision cannot be evaluated.
The study is open-label and single-group, with no concurrent comparator. This limits causal interpretation of any change over time and prevents a treatment contrast with another condition. The dose was selected at the investigator's discretion from 3 milligram (mg), 6 mg, 9 mg, or 12 mg once-daily options. The record does not report dose-specific outcome results, so responses by dose cannot be assessed.
The enrollment is recorded as 40, but the number included in the primary analysis is not reported. The ITT definition requires at least 1 dose and at least one post-baseline efficacy evaluation; the numbers meeting that definition and contributing Week 10 measurements are not reported. Missing-data methods, sensitivity analyses, and reasons for missing assessments are also not reported in the registry record.
The record identifies four outcome measures and four analyses but does not give details for the other outcomes or describe a multiplicity strategy. The statistical analysis plan, sample-size justification, interim analyses, and model details are not reported. Safety information is limited to the listed counts and serious adverse event terms; event timing, relatedness assessments, and the death count are not reported in the registry record. These gaps limit both efficacy and safety interpretation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety and Efficacy Study of Paliperidone Extended Release (ER) Among Thai Schizophrenia Participants — NCT01387542.
- Registry record last updated 2013-03-05; status last verified 2013-01.
- Results first posted 2013-03-05.
- 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
NCT01387542 is a completed Phase 4, open-label, prospective, single-group study sponsored by Janssen-Cilag Ltd.,Thailand. It enrolled 40 Thai participants with schizophrenia and assessed paliperidone extended release given orally once daily for 10 weeks, at investigator-selected doses of 3 milligram (mg), 6 mg, 9 mg, or 12 mg.
The primary endpoint was mean change from baseline in CGI-S at Week 10, with higher scores indicating worsening. The primary numerical result and its precision are not reported in the registry record. The stated ITT population required at least 1 dose and at least one post-baseline efficacy evaluation; the analysis denominator is not reported.
The record lists 1 participant affected by a serious adverse event and 22 affected by other adverse events, among 40 at risk in each category. Mania and Elevated mood are each listed at 2.5% among the top serious event terms. The record has no concurrent control group, so its outcome and safety entries are descriptive rather than comparative.