A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study of Aripiprazole in Children and Adolescents With Tourette’s Disorder

OBJECTIVE: To examine the short-term efficacy and tolerability of aripiprazole for children and adolescents with Tourette's disorder. METHOD: This 10-week multicenter, double-blind, randomized, placebo-controlled trial was conducted from August 2008 to April 2010. Children and adolescents (aged 6-18 years) with a DSM-IV diagnosis of Tourette's disorder and a Yale Global Tic Severity Scale total tic score of 22 or more were randomly assigned (1:1 ratio) to placebo or aripiprazole. The primary outcome measure was mean change from baseline in the total tic score on the Yale Global Tic Severity Scale (last observation carried forward). Assessments of safety and tolerability included spontaneously reported adverse events, extrapyramidal symptoms, serum prolactin level, metabolic variables, and other laboratory evaluations. RESULTS: Of 61 subjects, 89% completed the study. Patients who received aripiprazole demonstrated a significant reduction from baseline to end of study on the mean (SD) total tic score of the Yale Global Tic Severity Scale compared to those who received placebo (-15.0 [8.4] and -9.6 [8.8], respectively, P=.0196). Response rate on the Tourette's Syndrome Clinical Global Impression-Improvement was 66% and 45% in the aripiprazole and placebo groups, respectively. Mean decrease in the Tourette's Syndrome Clinical Global Impression-Severity of Illness score was significantly different between the groups (P=.0321). In general, aripiprazole was well tolerated and there were no early discontinuations due to adverse events. The incidence of treatment-emergent adverse events between the groups was not significantly different (P=.7550). While aripiprazole decreased serum prolactin concentration (P<.0001), it increased mean body weight, body mass index, and waist circumference significantly (P=.0055, P=.0142, and P=.0270, respectively). CONCLUSIONS: In comparison with placebo, aripiprazole was efficacious, generally tolerated and safe in the short-term treatment of children and adolescents with Tourette's disorder. TRIAL REGISTRATION: ClinicalTrials.gov identifier:NCT00706589.