Tocilizumab as Add-On Treatment For Residual Positive, Negative, and Cognitive Symptoms of Schizophrenia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Tocilizumab as Add-On Treatment For Residual Positive, Negative, and Cognitive Symptoms of Schizophrenia (NCT02034474), a randomized phase 4 study. The primary outcome is Clinical Response to Tocilizumab.

2. Clinical Question

The clinical question is about an add-on strategy and a measured symptom score. It does not, by itself, address whether tocilizumab changes every aspect of schizophrenia, or whether any measured score change is meaningful to every participant. The primary outcome is a change score over the stated timeframe. The registry record does not report the participants’ prior treatment details, eligibility thresholds for residual symptoms, or the broader care received during the trial; those details are not reported in the registry record.

3. Trial Design

The registry record describes a randomized, parallel-group, placebo-controlled phase 4 trial, with triple masking recorded. Its brief summary calls the study double-blind. The record does not identify which roles were masked, so the precise meaning of the triple-masking designation is not reported in the registry record. Randomization is intended to make treatment groups comparable on average, while a placebo comparator and masking can reduce some sources of expectation and assessment bias. Those design features do not guarantee that groups are balanced in a particular trial or eliminate every possible bias.

The trial evaluated tocilizumab as an addition to treatment for residual symptoms. The tocilizumab schedule is described as intravenous administration at day 0, week 4 and week 8, with a possible dose reduction if intolerable. Placebo was also given intravenously. The primary outcome timeframe runs from baseline through 12 weeks. The registry record lists enrollment as 59; the primary outcome description specifies that 17 placebo patients and 19 tocilizumab patients were included in the ITT analysis. These are different record entries describing enrollment and the reported primary analysis population; the record does not explain the relationship between them.

The New York State Psychiatric Institute is listed as sponsor. The trial is marked completed, with a start date of 2014-02 and primary completion on 2017-02-06. These entries describe the registry’s administrative record, not the timing of any later analysis or publication. Recruitment procedures, site details, allocation concealment, adherence, concomitant treatment rules, and the protocol’s full analysis plan are not reported in the registry record. The stated design provides context for interpreting the posted group summaries, but it cannot answer design questions that the record does not document.

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.

Before assignment: Of the 59 subjects who signed consent, 22 did not receive study drug. In addition, one of the 37 subjects who received study drug did not participate in any follow up assessments so was not included in the ITT analysis

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: 17 placebo patients and 19 tocilizumab patients were included in the ITT analysis

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: One subject was randomized but received no follow up study assessments so was not included in the ITT analysis

6. Primary Endpoint

The registry lists the primary outcome as Clinical Response to Tocilizumab, measured over Baseline (start of tocilizumab) through 12 weeks. We present the change scores.

The primary endpoint is change in total score on the Positive and Negative Syndrome Scale (PANSS) from baseline, described as the start of tocilizumab, through 12 weeks. The record reports this continuous outcome as a mean with standard deviation for each group. PANSS total combines symptom ratings across positive symptoms, negative symptoms, and general psychopathology. The registry description gives a total-score range of 30 to 210 and states that lower scores mean fewer symptoms. It also describes positive and negative subscales and a general psychopathology score.

A change score is used to focus on how the measured score differs from baseline over the specified follow-up period. Comparing mean change between randomized groups provides a summary of the average outcome in each group. It is important to distinguish the within-group changes from the between-group contrast: a mean change in one group alone does not establish how that group compares with the other. The estimated mean difference and its uncertainty address that comparison.

PANSS is used in this trial because the study question concerns residual positive, negative, and cognitive symptoms, and the primary hypothesis specifically names PANSS total score. The score is a measure of symptom severity, not a direct measure of every consequence of illness or treatment. The registry record does not report a threshold for a clinically important change, a responder definition, or a rule for combining this score with other outcomes. The score’s range and direction help interpret the scale, but they do not alone determine the clinical importance of a particular difference.

7. Results Posted in the Registry

• Tocilizumab: mean -0.50 versus -5.33 on control; difference 4.83 (95% CI -5.42 to 15.08), standardized effect d = 0.31; z-test p = 0.356.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Cognitive Symptomatology - Overall MATRICS t Score Change

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Tocilizumab: mean 1.47 versus 2.83 on control; difference -1.36 (95% CI -6.03 to 3.31), standardized effect d = -0.19; z-test p = 0.568.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Tocilizumab and the control. The interval gives the range of effects that remain compatible with the data.

Cognitive Symptomatology - UPSA-B Score Change

• Endpoint type: Continuous: a mean value per participant, compared as a difference in means.
• Test: Unadjusted two-sample z-test for the difference in means, using the posted standard deviations.
• Result:
  • Tocilizumab: mean 1.12 versus 1.12 on control; difference 0.00 (95% CI -0.15 to 0.15), standardized effect d = 0.00; z-test p = 1.000.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Tocilizumab and the control. The interval gives the range of effects that remain compatible with the data.

The secondary outcomes cover cognitive performance using a MATRICS t score and the UPSA-B score. They address domains related to the trial’s broader interest in cognitive symptoms, but they are not substitutes for the primary PANSS total endpoint. Across these measures, the posted results do not provide a clear, consistent pattern that independently strengthens the primary comparison. The registry record lists no reported analyses for either secondary outcome, so the entries are best treated as descriptive group summaries rather than as a fully documented inferential analysis.

Secondary results can be useful for generating questions about whether an intervention may have different associations with symptom ratings and cognitive measures. However, a pattern across endpoints is difficult to assess without knowing which outcomes and analyses were prespecified, how missing observations were handled, and whether statistical testing was adjusted for multiple comparisons. The registry record does not report a multiplicity strategy or a complete analysis plan. Consequently, unadjusted secondary comparisons carry less weight than a primary result defined in advance, and apparent consistency or inconsistency should not be overinterpreted.

The primary result itself is uncertain: its confidence interval includes values on both sides of no mean difference. Secondary measures cannot remove that uncertainty merely by being related to the same clinical question. Nor does a similar-looking average across two groups demonstrate equivalence. The record does not report equivalence margins, a non-inferiority framework, or a prespecified method for combining outcomes. The most defensible reading is that the secondary outcomes provide additional descriptive context, while the available record does not establish a separate or confirmatory cognitive effect.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Cognitive Symptomatology - Overall MATRICS t Score Change
• Cognitive Symptomatology - UPSA-B Score Change

9. Statistical Interpretation

10. Statistical Methods Explained

The main outcome is continuous, so the registry reports means and standard deviations rather than event counts. A mean summarizes the center of a group’s measured change scores. A standard deviation describes how dispersed those scores are around the mean. Neither summary shows the shape of the outcome distribution or whether a small number of unusual observations influenced the average. The registry record does not provide participant-level data or distribution plots from which to examine those features.

What does the mean difference represent?

The reported mean difference compares change in the tocilizumab group with change in the placebo group. Its direction depends on the stated comparison order: a positive estimate means the tocilizumab group’s mean change is higher than the placebo group’s mean change. For PANSS, lower scores indicate fewer symptoms, so the direction of a change-score contrast must be interpreted alongside the scale’s direction. A difference in means is an average contrast; it does not say that every participant had that difference or that every participant changed in the same direction.

The listed comparison is an unadjusted summary-statistic comparison. It is not presented as an adjusted estimate accounting for baseline variables or other covariates. No adjustment factors or model specification are reported in the registry record. The estimated contrast is useful as a compact description of the posted group summaries, but it should not be mistaken for a documented, prespecified sponsor analysis. The registry record lists no reported analyses, and the exact statistical procedure used for any sponsor analysis is not reported in the registry record.

How should the p-value be understood?

The unadjusted p-value is a measure of how inconsistent the observed comparison would be with a specified null model if that model and its assumptions held. It is not the chance that the result occurred by chance, the probability that the treatment has no effect, or a measure of clinical importance. A p-value does not capture the range of effect sizes compatible with the data; the confidence interval helps address that separate question. No adjustment for multiple outcomes is indicated in the posted p-values.

The term unadjusted matters because multiple outcomes or comparisons can raise the chance of finding an apparently notable result even when no corresponding effect exists. A multiplicity procedure can alter how evidence is assessed across a family of tests. The registry record does not report such a procedure, so the posted p-values should be read as nominal rather than as evidence adjusted for the full set of outcomes.

Why does variability affect precision?

Precision depends on both how variable the measurements are and how many observations contribute to the estimate. Greater variability generally makes it harder to estimate a group mean or a between-group contrast precisely. Standard errors describe estimated sampling uncertainty in an estimate; the record lists standard errors for the group means in its comparison data. A confidence interval is built to represent uncertainty around an estimate under a statistical procedure’s assumptions. The registry record does not identify the exact interval construction or test used, so those details should not be inferred.

For a continuous measure, an analysis may rely on assumptions about independence, the distribution of errors, and the behavior of the mean. Randomization supports a treatment-group comparison, but does not itself ensure that every statistical assumption holds. When group sizes are limited or outcomes are highly variable, estimates may be imprecise. The record does not report diagnostics, transformations, robust methods, or sensitivity analyses that would show how alternative assumptions affect the results.

What does the ITT label tell the reader?

ITT conventionally refers to an intention-to-treat analysis, in which participants are analyzed according to their randomized group. The primary outcome description uses the ITT label and specifies the participants included in that analysis. However, the registry record does not describe how missing scores, withdrawals, protocol deviations, or participants without outcome measurements were handled. The label alone cannot show whether every randomized participant contributed an observed outcome or how missingness may have affected the estimate.

The registry record also does not report a per-protocol analysis, a sensitivity analysis for missing data, or the treatment of any outlying observations. Those methods can affect interpretation, especially when outcome data are not available for all participants or when observed outcomes vary widely. Without those details, the posted summary is best read as the record’s reported group-level result, with uncertainty about unreported analysis decisions.

What can secondary outcomes add?

Secondary outcomes can examine related but distinct domains and may help characterize the range of measured responses. They do not automatically corroborate a primary result. Each outcome has its own scale, variability, missing-data pattern, and statistical uncertainty. A result across several endpoints also needs to be interpreted in light of how many comparisons were planned and how they were handled. The registry record lists two secondary cognitive outcomes but does not report their analysis methods or a multiplicity plan. Their summaries should therefore be considered exploratory or descriptive in the absence of further documented analysis details, rather than independent proof of a treatment effect.

More generally, statistical interpretation brings together the estimated contrast, its confidence interval, the measurement scale, the analysis population, and the plan for examining outcomes. A single test statistic cannot answer all of those questions. In this record, the estimated primary contrast and interval are available, while important details of the statistical plan, missing-data methods, and additional analyses are not reported in the registry record.

11. Confidence Intervals and Precision

The primary mean difference is 4.83, with a 95% confidence interval from -5.421 to 15.08. A confidence interval expresses uncertainty around an estimated contrast under the method used to construct it. It is not a range that contains every participant’s outcome, nor does it indicate that all values within it are equally plausible. Its interpretation depends on the statistical assumptions and procedure, which are not described in detail in the registry record.

The interval includes zero, the value corresponding to no difference in mean change between groups. It also extends into both negative and positive values. Thus, the posted estimate is compatible with a difference in either direction as well as no difference. The interval’s width signals limited precision: the data do not pin down the contrast narrowly. That uncertainty is consistent with the unadjusted p-value of 0.356, but the interval is more informative about the range of contrasts compatible with the analysis than the p-value alone.

Precision is not the same as importance. Even a narrow interval would not establish that a difference matters to patients without a meaningful-change threshold or other clinical context. The registry record does not report such a threshold. Nor does it report a confidence interval for a clinically defined responder outcome. The mean and standard deviation summaries give information about average changes and variability, but not the distribution of individual changes or the proportion meeting a meaningful response criterion.

The sample counts specified for the primary ITT analysis and the variability in the outcome provide context for why estimates may remain uncertain, but they do not justify an inference about the trial’s power after completion. The registry record does not report a formal power calculation for the original design. Precision should therefore be judged from the estimate and its interval, not from an assumed design target or a retrospective claim that the study was adequately powered.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are participants per arm, not total participants or total events. They are illustrations based on the observed effect, not the trial’s original design calculation. The record states the assumptions used for these illustrations: a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. For each effect size scenario, the 80% power column describes the planned number per arm for an 80% chance of detecting that stated effect under those assumptions if the assumptions hold. The 90% power column gives the corresponding per-arm planning figure for a 90% chance.

For planning at 100% of the observed primary effect, the figures are 159 participants per arm for 80% power and 213 participants per arm for 90% power. For planning at 75% of the observed effect, they are 283 participants per arm for 80% power and 379 participants per arm for 90% power. For planning at 50% of the observed effect, they are 636 participants per arm for 80% power and 851 participants per arm for 90% power. The progression illustrates that smaller effects are harder to distinguish from random variation, so they require more participants under the stated assumptions.

The curve represents the same planning relationship: the required group size rises as the effect to be detected becomes smaller, with the chosen power target also affecting the planned size. These values are not observed power, and they do not tell whether the completed trial had a particular chance of finding an effect. They are illustrations derived from the observed effect and the stated assumptions, not evidence that the original study was designed around those targets. The original sample-size justification and design assumptions are not reported in the registry record.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, Δ is the difference in means to detect and σ is the common standard deviation.

Worked calculation for this trial

1. Observed difference Δ = 4.830; pooled standard deviation σ = 15.366.
2. n = 2 × (1.96 + 0.842)² × 15.366² / 4.830² = 158.9, rounded up to 159 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
delta <- 4.83                      # observed difference in means
sigma <- 15.3658758675342                     # pooled standard deviation
n_per_arm <- function(power, frac = 1) {
  ceiling(round(2 * (z_a + qnorm(power))^2 * sigma^2 / (delta * frac)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (t-based, so very slightly larger)
cat("power.t.test:", round(power.t.test(delta = delta, sd = sigma, power = 0.80)$n, 1), "\n")

# Simulation: exact two-sample t-test using simulated means and variances
set.seed(2034474)
n    <- n_per_arm(0.80)
reps <- 20000
m_diff <- rnorm(reps, delta, sigma * sqrt(2 / n))             # difference in sample means
s2     <- sigma^2 * rchisq(reps, 2 * (n - 1)) / (2 * (n - 1))   # pooled sample variance
tstat  <- m_diff / sqrt(s2 * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(tstat) > qt(0.975, 2 * (n - 1))), reps))

Output from running the code

100% of observed effect: n per arm = 159 (80% power), 213 (90% power)
 75% of observed effect: n per arm = 283 (80% power), 379 (90% power)
 50% of observed effect: n per arm = 636 (80% power), 851 (90% power)
power.t.test: 159.8 
Simulated power at n = 159 per arm: 0.794 (20000 replicates)

The simulation draws many sets of group means and variances at the planned sample size and counts how often the two-sample t-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Multiplicity, Missing Data and Analysis Populations

Multiplicity arises when a study evaluates multiple outcomes, timepoints, subscales, or treatment contrasts. The more statistical comparisons that are considered, the greater the chance that some may appear notable by chance alone. Researchers can address this through a prespecified testing hierarchy, adjustment of significance thresholds, or other planned methods. The registry record does not report a multiplicity adjustment or a testing hierarchy. The posted p-values are identified as unadjusted, so they should not be interpreted as controlling the chance of false-positive findings across the full outcome set.

The primary endpoint is identified, and the record also lists secondary cognitive outcomes. The primary description includes PANSS total, while its narrative also describes component subscales. The registry record does not state whether subscale analyses were planned, how they would relate to the primary hypothesis, or whether other outcomes and timepoints were examined. The record lists four outcome measures, but does not document a complete analysis plan or explain the relationship between registered outcomes and reported analyses. It is therefore not possible to assess whether all planned tests are represented in the posted results.

Missing-data handling is also important for a change-score analysis. The primary description names an ITT analysis and gives the included group counts, but the registry record does not explain how missing follow-up scores were handled, whether imputation was used, or whether participants with incomplete measurements were excluded from particular analyses. It does not report the reasons for missing outcomes or analyses assessing whether missingness could bias the comparison. The enrolled population and primary analysis counts are recorded separately, without an explanation of the difference.

The record also does not report whether the analysis followed a prespecified protocol, whether interim analyses were conducted, or how deviations from a planned analysis were handled. Those details are not reported in the registry record. Their absence does not establish that these procedures were or were not used; it limits what can be assessed from the public record. Accordingly, the primary summary can be interpreted as the posted result, while confidence in the completeness and confirmatory status of the full analysis is constrained by unreported multiplicity and missing-data methods.

15. 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 1% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: agranulocytosis (5.3%).

The registry record reports serious adverse events for 0 of 17 participants at risk in the placebo group and 1 of 19 participants at risk in the tocilizumab group. It reports other adverse events for 4 of 17 at risk in the placebo group and 5 of 19 at risk in the tocilizumab group. Deaths are reported as 0 in both groups. These are counts of participants with reported events as recorded, not estimates of comparative risk adjusted for exposure or follow-up time.

The record lists agranulocytosis at 5.3% among the top serious adverse-event terms. It does not specify in this entry which group this term belongs to, nor does it provide further detail about timing, severity, relatedness, or outcome. No causal conclusion should be drawn from the listed term alone. Adverse-event counts in a trial of this size are descriptive and can be unstable; absence of a recorded event does not establish that an event cannot occur. The registry record does not report a detailed safety analysis, event rates over time, or a formal comparison between groups.

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

The result describes the mean change in PANSS total score observed in the reported analysis population over baseline through 12 weeks. It provides a between-group estimate, an interval expressing uncertainty, and an unadjusted p-value. The estimate is not precise enough to establish a clear difference in mean change between groups, but it also does not establish that the groups had identical outcomes. The confidence interval includes a range of possible contrasts in either direction.

These findings do not prove that tocilizumab has no effect, and they do not demonstrate that it improves symptoms. They also do not show that placebo and tocilizumab are equivalent; equivalence requires a prespecified margin and suitable analysis, neither of which is reported in the registry record. The posted data concern a particular trial population, outcome measure, treatment schedule, and follow-up timeframe. They cannot establish what would occur in every person with schizophrenia or schizoaffective disorder, over a different duration, or with a different regimen.

The primary endpoint is a symptom rating scale. A group average does not describe each participant’s experience, and the registry record does not report a clinically important difference threshold or individual responder results. The outcome therefore cannot, on its own, establish the practical importance of a change for an individual. The record’s cognitive outcomes provide additional descriptive measures but do not overcome the uncertainty in the primary comparison, particularly because their analysis details and multiplicity handling are not reported.

Safety entries should also be interpreted as recorded counts rather than definitive comparative safety conclusions. The record reports serious and other adverse events and deaths, but does not document detailed event timing, attribution, or a formal group comparison. The findings are a limited statistical account of the registry results, not medical advice or a complete assessment of benefits and risks.

17. Why This Trial Matters Statistically

This trial is statistically useful because it illustrates the difference between an observed group contrast and a precise estimate of that contrast. The posted primary mean difference has a confidence interval that crosses no difference and spans values in both directions. Reading the estimate together with its interval is more informative than treating a single p-value as a verdict. It emphasizes that uncertainty is part of the result, not a reason to convert an inconclusive comparison into a claim of no effect.

The trial also illustrates why outcome definition matters. PANSS total is a continuous symptom score with a stated direction, and the analysis concerns change over a specified timeframe. Interpreting the contrast requires attention to both the direction of the scale and the comparison order. This is distinct from interpreting the mean change within either group, and distinct again from judging clinical importance, for which the registry record gives no threshold.

Finally, the record allows readers to see how limitations in reporting affect statistical interpretation. It identifies an ITT population and gives group summaries, but does not report detailed missing-data methods, a complete analysis plan, or multiplicity handling. Those omissions do not determine the direction of the result; they limit the strength of conclusions that can be drawn. The planning illustrations also show how assumptions about the effect size and desired power influence sample-size requirements, while remaining separate from the trial’s original design. Together, these features make the record a practical example of reading estimates, uncertainty, and reporting completeness as parts of a single evidence assessment.

18. 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 registry record has limitations specific to interpreting this trial’s results. It gives enrollment as 59 but identifies 17 placebo patients and 19 tocilizumab patients in the primary ITT analysis. It does not explain the relationship between those entries, describe exclusions or missing follow-up scores, or document how the analysis population was formed. As a result, the extent to which the posted primary summaries represent all randomized participants cannot be determined from the record alone.

The primary endpoint is a PANSS change score, but the record does not report a clinically important difference threshold or responder definition. The summaries therefore establish neither the proportion of participants experiencing a meaningful improvement nor how individual outcomes were distributed. Standard deviations indicate variability, but participant-level data and distributional diagnostics are not reported. It is not possible to determine whether the group means were strongly affected by unusual observations or whether the distributional assumptions of a particular analysis were checked.

The registry record does not report the full statistical analysis plan, the sponsor’s detailed model, handling of missing data, or any sensitivity analyses. It lists no reported analyses for the secondary outcomes and does not state how multiplicity was addressed. The record also describes masking as triple in the design fields and as double-blind in the summary, without clarifying which participants or staff were masked. Allocation concealment and adherence information are not reported in the registry record.

Finally, the outcome period ends at 12 weeks, and no longer-term outcome information is included in the listed primary result. The record does not describe treatment adherence, concomitant medication changes, or broader care during follow-up. These gaps constrain conclusions about durability, generalizability, and the robustness of the posted comparisons. They should be understood as limits of the registry record, not as evidence that any unreported procedure was absent.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Tocilizumab as Add-On Treatment For Residual Positive, Negative, and Cognitive Symptoms of Schizophrenia — NCT02034474.
• Registry record last updated 2018-12-26; status last verified 2018-12.
• Results first posted 2018-12-26.
• 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.

22. Record Summary

NCT02034474 is a completed, randomized, parallel-group phase 4 trial of tocilizumab versus placebo as add-on treatment for residual symptoms in schizophrenia or schizoaffective disorder. The registry describes triple masking and a double-blind study in its summary. The primary outcome is mean change in PANSS total score from baseline through 12 weeks.

The primary ITT description includes 17 placebo patients and 19 tocilizumab patients. The posted mean difference is 4.83, with a 95% confidence interval from -5.421 to 15.08 and an unadjusted p-value of 0.356. The interval includes no difference and values in either direction, so the estimate is uncertain. Two secondary cognitive outcomes and adverse-event counts are also reported. Detailed analysis methods, missing-data handling, and multiplicity procedures are not reported in the registry record.