Treatment with Ecopipam for Children and Adolescents with Tourette Syndrome Shows No Effect on Psychiatric Comorbidities

For children and adolescents with Tourette syndrome (TS), treatment with the D1 receptor antagonist ecopipam (Emalex Biosciences, Chicago, IL) was not associated with significant effects on common psychiatric comorbidities in the subgroups of participants with attention deficit disorder (ADHD), anxiety disorders, obsessive-compulsive disorder (OCD), and/or depression, according to data presented at the 2024 International Congress of Parkinson’s Disease and Movement Disorders. This finding is significant in the context of phase 2b trial data highlighting that a majority of children and adolescents with TS treated with ecopipam have 1 or more of these comorbidities. The presented data also show that some measures of ADHD trended lower for children treated with ecopipam compared with placebo.

In results of a 12-week, phase 2b, randomized, double-blind, placebo-controlled trial that included children and adolescents aged >6 to <18 years with TS and a Yale Global Tic Severity Scale–Total Tic Score (YGTSS-TTS) of ≥20 at screening, treatment with ecopipam was associated with a 30% improvement (P=.01) in YGTSS-TTS. Findings of this phase 2b trial also revealed that, among participants who received ≥1 dose of ecopipam and ≥1 postbaseline evaluation, 66% had ≥1 comorbidity including ADHD, anxiety disorders, OCD, and/or depression.

In the present study, a mixed model was used compare the effects of ecopipam vs placebo on participants’ serial measures of the Swanson, Nolan and Pelham Teacher and Parent Rating Scale (SNAP-IV) for ADHD, the Pediatric Anxiety Rating Scale (PARS) for anxiety disorders, the Child Yale-Brown Obsessive Scale (CY-BOCS) for OCD, and the Children’s Depression Rating Scale–Revised (CDRS-R) for depression. These scales were administered during the phase 2b trial at baseline, week 4, week 6, week 8, and week 12.

The results of the study demonstrated no statistically significant difference in the change from baseline in any of the serial measures of psychiatric comorbidities between the ecopipam and placebo groups. In addition, for ADHD, which was the most frequent comorbidity, the Conners, Inattention, Opposition, Hyperactive, Aggressive, Academic and Total scores of children and adolescents treated with ecopipam trended lower vs placebo.