Efficacy and Safety of Ecopipam for Tourette Syndrome: A Phase 3 Randomized Clinical Trial - PubMed

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Ecopipam, a selective dopamine D1 receptor antagonist, was evaluated for Tourette Syndrome (TS) maintenance efficacy and safety over 24 weeks.
The study was a phase 3 randomized withdrawal trial with a 12-week open-label period and a 12-week double-blind period.
Pediatric participants showed significantly reduced relapse risk with ecopipam vs placebo (HR 0.47; P = .008).
Adults showed a similar directional effect but it was not statistically significant.
Common adverse events included somnolence, anxiety, headache, insomnia, tic, and fatigue.
No clinically meaningful impacts on weight, metabolic parameters, or psychiatric scales were observed.
Ecopipam was well tolerated, with adverse events primarily affecting the central nervous system.

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