Ecopipam Reduced Tics in Children and Adolescents With Tourette Syndrome

A recent study demonstrated efficacy and safety of ecopipam for treatment of Tourette syndrome (TS) in children and adolescents, ages 6 to 17 years. Both motor and phonic tics were reduced with ecopipam (n=74) compared with placebo (n=75).   

A statistically significant least squares mean difference of -3.4±1.4-point (95% CI: -6.1, -0.8) improvement on the Yale Global Tic Severity Scale (YGTSS-TTS) was observed for ecopipam vs placebo in all participants. Subgroup analysis showed that efficacy was consistent among children, ages 6 to 11 years, and adolescents, ages 12 to 17 years. 

Improvements also occurred with ecopipam vs placebo on the clinical global impression of change (CGI) for TS severity (CGI-TS-S; P=.001) and Caregiver Global Impression of Change (CaGI-C; P<.05). No significant change in quality of life with ecopipam vs placebo was observed, however.

“Despite the fact that several new or repurposed medications appeared promising for the treatment of tics in recent years, no other drug confirmed effectiveness in randomized controlled trials since the addition of aripiprazole to the therapeutic arsenal of TS,” said Davide Martino, MD, PhD, associate professor, Department of Clinical Neurosciences, University of Calgary. “This phase 2b randomized trial breaks the spell by showing significant superiority to placebo of a first-in-class selective dopamine D1 receptor antagonist, ecopipam, in reducing tic severity in children and adolescents with TS. This study is relevant for its adequate sample size and selection of primary and secondary outcomes. Ecopipam showed superiority to placebo in reducing tic severity, based on both clinician’s and caregiver’s impression, but did not differ from placebo in (affecting) quality of life. Overall, this phase 2b study generates hope and supports that ecopipam deserves further investigation as a new treatment option for tics in TS.”

The most common treatment-related adverse events with ecopipam were headache (9.2%), insomnia (9.2%), fatigue (6.6%), somnolence (6.6%), and restlessness (5.3%). No weight gain or metabolic AEs occurred with ecopipam. The overall rate of adverse events was 34% (26/74) with ecopipam and 21% (16/75) with placebo.

These data were presented at the International Congress of Parkinson’s Disease and Movement Disorders in Madrid, Spain.