Kinga Tomczak, MD, PhD, Program Director of the Tic Disorders and Tourette Syndrome Program at Boston Children’s Hospital and Assistant Professor of Neurology at Harvard Medical School, discusses phase 3 clinical trial results of ecopipam as a treatment for Tourette syndrome.
Tourette syndrome is a chronic childhood-onset neurodevelopmental disorder characterised by motor and phonic tics that can substantially diminish the quality of life of affected individuals. The underlying pathophysiology of Tourette syndrome is not fully understood, but recent research has brought new insights into the genetic variations and the alterations in neurophysiology and brain networks contributing to its pathogenesis. Tourette syndrome occurs more frequently in males, and males tend to have more severe symptoms than females. Tourette syndrome is often accompanied by comorbid behavioural disorders, including most prominently obsessive-compulsive behaviour and attention deficit disorders.
At the American Academy of Neurology 2026 Annual Meeting (AAN 2026), data was presented from a phase 3 study on the safety and efficacy of ecopipam for the treatment of Tourette syndrome. Ecopipam is a selective dopamine D1 receptor antagonist being investigated as a maintenance therapy in persons with Tourette syndrome.
In the study, patients ages 6 years and older with Tourette syndrome were included in a double-blind, placebo-controlled, randomized withdrawal trial. Ecopipam was titrated over 3 to 4 weeks with a target dose of 1.8 mg/kg/day in the 12 week open-label period. Patients with a clinically meaningful reduction of 25% or greater in Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) at weeks 8 and 12 were randomized to continue ecopipam or taper to placebo in the 12 week double-blind period.
A total of 216 patients were enrolled in the 12 week open-label phase. Overall, 104 patients (90 pediatric) were randomized to the 12 week double-blind period to continue ecopipam or switch to placebo. The risk of relapse was significantly reduced in the ecopipam group compared with placebo in the pediatric and pediatric plus adult populations. The most commonly reported adverse events in the ecopipam group during the 24 week study were somnolence, anxiety, headache, insomnia, tic, and fatigue.
Overall, this study demonstrated ecopipam’s ability to maintain clinically meaningful improvements in Tourette syndrome symptoms. It was generally well-tolerated, with adverse events primarily affecting the central nervous syndrome and did not cause weight gain, dyslipidemia, or drug-induced movement disorders.
To read the abstract presented at AAN 2026, visit https://index.mirasmart.com/AAN2026/PDFfiles/AAN2026-004157.html
To learn more about rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/
Reference:
Johnson, KA. et al. Tourette syndrome: clinical features, pathophysiology, and treatment. Lancet Neurol. 2022;22(2):147-158. https://pmc.ncbi.nlm.nih.gov/articles/PMC10958485/