Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD) (NCT01418352), a randomized phase 3 study. The primary outcome is Mean Change From Baseline to Week 8 in Yale Global Tic Severity Scale (YGTSS) Total Tic Score.

2. Clinical Question

3. Trial Design

The registry record describes a multicenter, randomized, quadruple-masked, parallel-group, placebo-controlled Phase 3 study. It lists Tourette's Disorder as the condition and reports enrollment of 83. Participants were assigned to one of three fixed-dose aripiprazole groups or matching placebo. Treatment was oral and once weekly during an 8-week double-blind treatment period.

The primary outcome was mean change from baseline to Week 8 in the Yale Global Tic Severity Scale Total Tic Score. The outcome description identifies the intention-to-treat sample as participants randomly assigned to double-blind treatment, regardless of whether they were dosed. It also clarifies that the number analyzed for an outcome reflects participants with data available for that analysis. The primary outcome results list 17.0 analyzed for the 52.5 mg group, 17.0 for the 77.5 mg group, 16.0 for the 110 mg group, and 18.0 for matching placebo.

Two secondary outcomes are listed: change in the Clinical Global Impressions Scale for Tourette's Syndrome score and change in the Gilles de la Tourette Quality of Life Overall Score. Both are described as changes from baseline, with the timeframe stated as baseline and Week 8.

The record marks the study completed and lists Otsuka Pharmaceutical Development & Commercialization, Inc. as sponsor. The registry record does not report the original sample-size justification, detailed allocation procedures, or the statistical analysis plan. It also does not describe the handling of missing outcome data beyond the definition of the analyzed population.

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: This study consisted of 2 distinct phases: a Pre-treatment Phase and a Treatment Phase. The Pre-treatment Phase consisted of a Screening period, a Washout period, and a Baseline visit. This was followed by an 8-week Treatment Phase. There was a follow-up period (30 days) for those participants who did not roll-over into the Open-label Study 31-10-274 (NCT01416441).

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug). Overall number of participants analyzed are participants with data available for analyses.

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: The Intention-to-Treat (ITT) Sample included participants randomly assigned to the double-blind treatment (regardless of whether or not they were dosed with study drug).

6. Primary Endpoint

The registry lists the primary outcome as Mean Change From Baseline to Week 8 in Yale Global Tic Severity Scale (YGTSS) Total Tic Score, measured over Baseline and Week 8.

The primary endpoint is mean change from baseline to Week 8 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score. The YGTSS is described as a semi-structured clinical interview designed to measure tic severity. Its assessment includes a tic inventory, ratings of motor and vocal tics across several dimensions, and an impairment ranking. The Total Tic Score is the sum of the motor and vocal tic severity scores.

The score ranges from 0, indicating none, to 50, indicating severe symptoms. Higher scores represent more severe symptoms. For change from baseline, a more negative value means a greater reduction in tic severity; the registry description treats a greater reduction as greater improvement on this endpoint.

A mean change is useful for comparing the average shift in scores across treatment groups. It gives a compact summary of the direction and size of change, while the standard deviation describes how dispersed individual participants' changes were around their group's mean. These summaries do not show each participant's experience or the full shape of the outcome distribution.

The endpoint is focused on tic severity rather than every aspect of Tourette's Disorder or participants' broader wellbeing. The registry lists quality of life and a clinical global impression measure as secondary outcomes, which address other dimensions. The record does not report additional details about the interview administration, assessor training, or how baseline and Week 8 scores were handled when one was unavailable.

7. Results Posted in the Registry

• Aripiprazole 52.5 mg: mean -8.20 versus -9.60 on control; difference 1.40 (95% CI -2.75 to 5.55), standardized effect d = 0.22; z-test p = 0.508.
• Aripiprazole 77.5 mg: mean -9.90 versus -9.60 on control; difference -0.30 (95% CI -5.01 to 4.41), standardized effect d = -0.04; z-test p = 0.901.
• Aripiprazole 110 mg: mean -14.50 versus -9.60 on control; difference -4.90 (95% CI -10.02 to 0.22), standardized effect d = -0.65; z-test p = 0.061.

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.

Mean Change From Baseline in Clinical Global Impressions Scale for Tourette's Syndrome (CGI-TS) Score

• 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:
  • Aripiprazole 52.5 mg: mean -0.90 versus -1.00 on control; difference 0.10 (95% CI -0.40 to 0.60), standardized effect d = 0.13; z-test p = 0.697.
  • Aripiprazole 77.5 mg: mean -1.10 versus -1.00 on control; difference -0.10 (95% CI -0.70 to 0.50), standardized effect d = -0.11; z-test p = 0.743.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Aripiprazole 52.5 mg and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Aripiprazole 77.5 mg and the control. The interval gives the range of effects that remain compatible with the data.

Mean Change From Baseline in Gilles de la Tourette Quality of Life (GTS-QOL) Overall Score at Week 8

• 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:
  • Aripiprazole 52.5 mg: mean 8.80 versus 13.40 on control; difference -4.60 (95% CI -14.54 to 5.34), standardized effect d = -0.31; z-test p = 0.365.
  • Aripiprazole 77.5 mg: mean 3.80 versus 13.40 on control; difference -9.60 (95% CI -23.33 to 4.13), standardized effect d = -0.47; z-test p = 0.171.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Aripiprazole 52.5 mg and the control. The interval gives the range of effects that remain compatible with the data.
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Aripiprazole 77.5 mg and the control. The interval gives the range of effects that remain compatible with the data.

The registry lists two secondary outcomes: change in the Clinical Global Impressions Scale for Tourette's Syndrome score and change in the Gilles de la Tourette Quality of Life Overall Score. The posted group summaries show that these measures do not form a uniformly clear pattern that mirrors the primary endpoint. The global-impression changes are broadly similar across groups, while the quality-of-life changes vary across groups and do not consistently align with a more favorable primary tic-severity comparison.

These measures address different aspects of the condition, so agreement is not guaranteed. A clinical global impression and a quality-of-life score may capture broader or different experiences than the YGTSS Total Tic Score. Their relationship to the primary result is therefore contextual: they can show whether the observed pattern extends to other measured outcomes, but they do not replace the primary endpoint or resolve its uncertainty.

The recalculated secondary comparisons are unadjusted. The registry record lists no reported analyses for these outcomes and does not report a procedure for controlling multiplicity. General statistical context: when several outcomes and treatment comparisons are examined, the chance of at least one apparently unusual result can increase if all are interpreted as independent confirmatory tests. Without a stated multiplicity plan, secondary p-values should carry less weight than a prespecified, appropriately adjusted analysis.

The secondary results are best viewed as descriptive and exploratory evidence. Their summaries can inform the overall pattern, but the small analyzed groups, uncertainty, lack of reported analysis details, and absence of a reported multiplicity procedure prevent strong conclusions from isolated comparisons. The registry record also does not report how these outcomes were prioritized or whether any secondary outcome was designated as a key confirmatory endpoint.

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

• Mean Change From Baseline in Clinical Global Impressions Scale for Tourette's Syndrome (CGI-TS) Score
• Mean Change From Baseline in Gilles de la Tourette Quality of Life (GTS-QOL) Overall Score at Week 8

9. Statistical Interpretation

10. Statistical Methods Explained

The primary outcome is continuous: it is a score, and the registry reports its mean change and standard deviation in each group. Comparisons are expressed as mean differences in change between each aripiprazole group and matching placebo. These recalculated comparisons use the posted summary statistics and are unadjusted; the registry record lists no sponsor-reported analysis results.

What does a mean difference represent?

A mean difference compares the average change in one group with the average change in another. For this endpoint, more negative change indicates a greater reduction in tic severity. A negative mean difference therefore indicates a more negative average change in the aripiprazole group than in placebo; a positive difference indicates a less negative average change. This is a description of the direction of the contrast, not evidence that the treatment caused the difference.

The recalculated estimates were 1.4 for 52.5 mg, -0.3 for 77.5 mg, and -4.9 for 110 mg, each compared with matching placebo. The point estimate is only one part of the result. Its uncertainty and the observed variation within groups also matter. The registry record does not report an adjusted model or other covariate-adjusted estimate, so the recalculated comparisons should not be described as adjusted analyses.

Why report standard deviations?

A standard deviation describes how spread out participant scores are around the group mean. It is not the same as the uncertainty in the mean or the uncertainty in a difference between group means. General statistical context: when participant-level outcomes vary substantially, a group mean alone gives an incomplete picture, and the comparison can be estimated less precisely. The posted standard deviations allow a simple unadjusted comparison to be recalculated, but they do not reveal the full outcome distribution, outliers, or whether the statistical assumptions fit the data.

How should confidence intervals and p-values be understood?

A confidence interval gives a range of values compatible with the estimated difference under the method used. General statistical context: a wider interval indicates less precision, and an interval that includes zero does not rule out no mean difference. All three primary recalculated intervals include zero. The p-values are also unadjusted and describe how compatible the observed estimates are with a null comparison under the calculation's assumptions. They do not give the probability that a treatment works, nor do they measure clinical importance.

The recalculated intervals and p-values are based on summary data rather than participant-level data. They do not reproduce any analysis that may have been performed by the sponsor. Since the registry record does not report the sponsor's analysis results or statistical methods, there is no basis in this record to assess whether the recalculated approach matches the trial's prespecified analysis.

Why can over-dispersion matter?

General statistical context: over-dispersion means that observed variation is greater than a model expects. When a model underestimates variation, it can make uncertainty appear smaller than it is. The posted standard deviations describe the observed spread, but the registry record does not report a model or assessment of over-dispersion. No conclusion about over-dispersion can be drawn from the registry summaries alone.

What remains unknown about the analysis?

The record defines the primary analysis population as the intention-to-treat sample and explains that the analyzed number is based on availability of outcome data. It does not report how missing values were handled, whether all randomized participants contributed to the primary estimate, or whether sensitivity analyses were conducted. The detailed analysis plan, including any covariate adjustment, assumptions about the outcome distribution, and prespecified handling of multiplicity, is not reported in the registry record. Those omissions limit comparison between the recalculated results and the trial's intended inferential analysis.

11. Confidence Intervals and Precision

The recalculated primary comparisons have confidence intervals that include zero. For 52.5 mg, the estimated mean difference was 1.4, with an interval from -2.749 to 5.549. For 77.5 mg, the estimate was -0.3, with an interval from -5.006 to 4.406. For 110 mg, the estimate was -4.9 and its interval also extends across zero. These intervals indicate that the summaries do not precisely distinguish a mean difference from no difference.

General statistical context: a confidence interval communicates uncertainty around an estimate, rather than the range of outcomes expected for individual participants. A wide interval can be compatible with effects in more than one direction. The span of the intervals is especially important when the point estimate alone might seem to favor one group. Here, the 110 mg point estimate favors a greater reduction than placebo, but the interval includes values on both sides of no difference. The 52.5 mg and 77.5 mg intervals likewise include differences in both directions.

The unadjusted p-values for these comparisons were 0.508, 0.901, and 0.061, respectively. They should be read alongside the estimates and intervals, not as standalone measures of importance. The registry record does not provide sponsor-reported confidence intervals or analyses, so these are recalculated from posted summary statistics. They also do not account for any multiplicity adjustment.

Precision is constrained by the available analyzed groups and the observed variability. The record reports 17.0, 17.0, 16.0, and 18.0 analyzed for the four groups, respectively, and standard deviations between 4.8 and 7.7 for the primary outcome. These summaries do not describe missing-data patterns or allow participant-level checking of assumptions. As a result, the recalculated intervals are useful for understanding uncertainty in the posted comparison, but should not be mistaken for a complete account of the trial's planned statistical inference.

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 illustrations based on the observed effect, not the original trial design. For these continuous outcomes, the figures are participants per arm, not total events. The stated planning assumptions are a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. Each planning scenario uses a specified fraction of the observed effect. The trial's own original design assumptions and sample-size justification are not reported in the registry record.

The 80% power column describes a hypothetical design that, under the stated assumptions and for the effect used in that scenario, would have an 80% chance of detecting that effect using the assumed test. The 90% power column describes the corresponding design with a 90% chance. These are design probabilities under assumptions, not probabilities that the trial result is true. This is not observed power.

Read each row as a planning question: how many participants per arm would be needed if the target effect were the stated fraction of the observed effect? The estimates based on a smaller fraction require more participants because a smaller difference is harder to distinguish from random variation. The calculated values can change considerably when the assumed effect changes, especially when the observed difference is small. Very large planning estimates can therefore reflect a small observed contrast rather than a recommendation for a future study.

The calculations use the observed effect as a starting point, so they should not be treated as reliable forecasts of what a future trial will observe. Observed effects from small groups can be unstable. The illustrations also do not incorporate any design features or adjustments that are not among the stated assumptions. The registry record does not report whether the original study used these assumptions.

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 = 1.400; pooled standard deviation σ = 6.336.
2. n = 2 × (1.96 + 0.842)² × 6.336² / 1.400² = 321.5, rounded up to 322 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 <- 1.4                      # observed difference in means
sigma <- 6.33625929221507                     # 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(1418352)
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 = 322 (80% power), 431 (90% power)
 75% of observed effect: n per arm = 572 (80% power), 766 (90% power)
 50% of observed effect: n per arm = 1,287 (80% power), 1,722 (90% power)
power.t.test: 322.5 
Simulated power at n = 322 per arm: 0.798 (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

The registry record lists one primary outcome and two secondary outcomes, with multiple treatment-versus-placebo comparisons. It does not report a multiplicity adjustment or explain how the family of outcomes and comparisons was handled. The recalculated comparisons are unadjusted. Their p-values should therefore not be interpreted as though a trial-wide error rate had been controlled. General statistical context: multiplicity matters because repeated statistical testing can increase the chance of finding an apparently notable result by chance.

The registry record reports no analyses for the primary or secondary outcomes. It does not state whether the posted summaries come from a prespecified analysis, whether the sponsor used an adjusted model, or which covariates or assumptions were included. The recalculated contrasts are simple unadjusted comparisons from posted group means and standard deviations. They cannot establish what the sponsor's planned analysis would have found.

The primary outcome description defines the intention-to-treat sample as participants randomly assigned to double-blind treatment, whether or not they received study drug. It also says the number analyzed is the number with data available for analysis. This definition does not explain how participants without an observed outcome were treated analytically. The record does not report the amount or reasons for missing data, how missing values were handled, whether any imputation was used, or whether sensitivity analyses assessed the effect of missingness.

These omissions matter because an analysis restricted to participants with available data may differ from an analysis that includes all randomized participants under a prespecified missing-data method. The extent and direction of any resulting bias cannot be determined from the posted summaries. Detailed statistical methods, including the planned analysis population for each secondary endpoint and the handling of protocol deviations, are not reported in the registry record.

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 5% reporting threshold.

Most frequent non-serious adverse event terms

The serious adverse event summaries report zero affected participants in each group. The numbers listed as at risk were 20 for aripiprazole 52.5 mg, 21 for aripiprazole 77.5 mg, 21 for aripiprazole 110 mg, and 21 for matching placebo. The record also reports zero deaths in each group.

These figures describe what was recorded for the listed treatment groups and risk sets. They do not establish that the treatments have no serious risks. With limited group sizes and an 8-week treatment period, an absence of recorded serious events in this record cannot rule out uncommon events or events occurring outside the observed period. The registry record does not provide further detail about event definitions, timing, severity grading, or adjudication.

The serious-event figures are separate from the counts for other adverse events. The latter are not a substitute for the serious-event results and should not be combined with them. Safety interpretation should remain limited to the recorded events, populations at risk, and follow-up described in the registry record.

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

The primary results describe average change in tic severity from baseline to Week 8 in the analyzed groups. Mean scores fell in every group, including matching placebo. The recalculated comparisons show that the estimated difference was small for the 52.5 mg and 77.5 mg groups, while the 110 mg estimate favored a greater mean reduction than placebo. However, all three confidence intervals include zero, and the unadjusted comparisons do not provide precise evidence of a difference.

This is a description of the posted data, not a conclusion that aripiprazole did or did not work. The observed changes cannot, on their own, establish causation. The registry record does not provide sponsor-reported analyses, the original statistical plan, a reported multiplicity method, or details of missing-data handling. The recalculations are simple unadjusted comparisons of group summaries and may not match the planned trial analysis.

The findings also apply to a specific setting: children and adolescents with Tourette's Disorder, fixed-dose once-weekly oral treatment, and the listed 8-week double-blind period. They do not answer questions about other populations, dosing schedules, longer follow-up, or outcomes not reported in this record. The primary endpoint measures tic severity; it does not capture every dimension of health or daily life.

Uncertainty should be considered together with the point estimates. A point estimate describes the observed contrast, while its confidence interval indicates the range of contrasts compatible with the recalculation. Because the intervals span zero, the data do not distinguish confidently between no mean difference and differences in either direction. The secondary outcomes add context but are also unadjusted and do not resolve the primary uncertainty.

In short, the registry record documents group-level changes and an uncertain pattern of differences versus placebo. It does not support a definitive claim about efficacy or safety. The absence of recorded serious adverse events is limited to the reported risk groups and observation period, and should not be interpreted as proof that serious events cannot occur.

17. Why This Trial Matters Statistically

This study illustrates why a group mean and its uncertainty need to be read together. The primary outcome shows reductions in all groups, but the comparison of each dose with placebo is the relevant contrast for interpreting differences between groups. Looking only at the largest observed reduction would omit both the placebo result and the uncertainty around the comparison.

It also illustrates how a small study can yield imprecise estimates even when group summaries appear different. The recalculated intervals include zero for all primary comparisons. That does not prove the groups are equivalent; it means the registry summaries do not precisely separate a difference from no difference. The observed effects also drive the planning illustrations, so a small or unstable observed contrast can produce very large planning requirements when the target is a fraction of that contrast.

The record is useful for teaching the distinction between reported outcomes and reported analyses. It gives means, standard deviations, analyzed counts, and secondary outcome summaries, but lists no analyses. Recalculating contrasts makes the arithmetic interpretation more transparent, while leaving the original analysis plan and sponsor results unknown. Those are different kinds of evidence and should not be conflated.

Finally, multiple outcomes and treatment comparisons raise questions about confirmatory interpretation. Without a reported multiplicity strategy, secondary findings deserve a more cautious role. The trial's short treatment period, limited analyzed groups, and unreported missing-data methods further show why registry results are best interpreted within their design and reporting limits rather than as a complete account of all possible effects.

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 primary estimates are recalculated from posted summary statistics rather than taken from reported sponsor analyses. The registry record lists no reported outcome analyses, and the original statistical plan is not reported. Consequently, the recalculations may not reflect any prespecified adjustment, modeling choice, or analysis population used by the sponsor.

The analyzed groups are limited in size, and their reported counts differ across arms. Although the record defines the analyzed number as participants with data available, it does not report how many randomized participants lacked outcome data, why data were missing, or how missingness was addressed. No sensitivity analyses are listed. The effect of missing data on the comparisons cannot be assessed from the record.

The primary comparison is unadjusted, and no multiplicity procedure is reported despite several treatment comparisons and secondary outcomes. The registry summaries also provide means and standard deviations rather than participant-level data. They cannot show the distribution of individual changes, identify influential observations, or permit independent checking of model assumptions.

Follow-up for the double-blind treatment period was 8 weeks. The record therefore does not characterize longer-term outcomes. The population is described as children and adolescents with Tourette's Disorder, but further eligibility details are not included in the registry record recorded. The posted outcome set focuses on tic severity, a global impression, quality of life, and adverse events; it does not represent every possible outcome of interest.

These limitations do not invalidate the posted summaries, but they constrain what can be inferred from them. In particular, they make it important to distinguish descriptive group changes from treatment comparisons and to avoid treating recalculated unadjusted results as the trial's definitive statistical analysis.

19. Related Tutorials

20. Related Statistical Calculators

21. Sources

• ClinicalTrials.gov: Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder (TD) — NCT01418352.
• Registry record last updated 2021-10-07; status last verified 2021-09.
• Results first posted 2021-10-07.
• 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

The registry record describes a completed, randomized, quadruple-masked, parallel-group Phase 3 study sponsored by Otsuka Pharmaceutical Development & Commercialization, Inc. It enrolled 83 children and adolescents with Tourette's Disorder and compared three once-weekly oral aripiprazole doses with matching placebo during an 8-week double-blind treatment period.

The primary outcome was mean change from baseline to Week 8 in YGTSS Total Tic Score. Mean reductions were recorded in every group. Recalculated unadjusted differences versus placebo had confidence intervals that included zero. Two secondary outcomes were also posted, but no analyses are listed in the registry record.

The record reports zero serious adverse events and zero deaths in each group. The original statistical plan, multiplicity handling, and missing-data methods are not reported, limiting interpretation of the recalculated comparisons.