Results from Phase III Study of Ecopipam for Tourette Syndrome

Key findings, limitations and clinical perspectives on the important phase III study of Ecopipam for TS; taking a closer look with Dr Don Gilbert

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For our November episode, and following his talk at the Athens 2025 Conference, we speak with Dr. Gilbert about Ecopipam, a promising new treatment for Tourette syndrome that has recently completed phase 3 clinical trial.

The discussion explores the need for better therapeutic options in the current treatment landscape of Tourette syndrome and traces Ecopipam's journey from early animal studies through its clinical development programme.
Dr Gilbert explains its mechanism of action and reviews the preclinical evidence that first suggested its potential benefit for Tourette syndrome. The conversation covers the drug's initial exploration in other clinical applications, including stuttering, before pivoting to its development specifically for TS.
The episode provides an in-depth look at three earlier clinical trials that demonstrated Ecopipam's benefit in individuals with TS, examining both their promising results and inherent limitations. Dr Gilbert then walks through the comprehensive methodology of the phase 3 study, explaining the rationale behind the chosen study design and the involvement of 99 sites worldwide that enrolled 216 subjects in the open-label phase.
Key efficacy findings are discussed for both paediatric and adult populations, including specific reductions in YGTSS Total Tic Severity scores. The conversation addresses the critical safety profile, examining psychological, metabolic, and extrapyramidal side effects, with particular attention to concerns regarding suicidality signals observed in the trial data.
He also shares insights on Ecopipam's effects on psychiatric comorbidities commonly associated with Tourette syndrome, acknowledges the study's limitations, and discusses available data from the extension phase.
The episode concludes with perspectives on anticipated regulatory timelines in the United States and Europe, along with Dr Gilbert's thoughts on where Ecopipam might fit in the treatment algorithm—whether as a first-line option, second-line therapy, or add-on treatment—and any additional considerations for clinicians and patients.

Dr Gilbert is Professor of Neurology at Cincinnati Children’s Hospital Medical Center, where he established the Movement Disorders and Tourette Clinics in 1998 and conducts translational research and clinical trials.  

Key references

• Gilbert DL, Budman CL, Singer HS, Kurlan R, Chipkin RE. A D1 Receptor Antagonist, Ecopipam, for treatment of tics in Tourette Syndrome. Clinical Neuropharmacology, 2014. 37:26-30.
• Gilbert DL, Murphy TK, Jankovic J, Budman CL, Black KJ, Kurlan RM, Coffman KA, McCracken JT, Juncos J, Grant JE, Chipkin RE. Ecopipam, a D1 receptor antagonist, for treatment of Tourette Syndrome in Children: A randomized, placebo-controlled crossover study. Movement Disorders 2018. 33: 1272-1280. 
• Chipkin RE, Gilbert DL, Mahableshwarkar A. Ecopipam: A D1 receptor antagonist for the treatment of Tourette syndrome. International Review of Movement Disorders. Elsevier, Inc. 2022. doi  
• Gilbert DL, Dubow JS, Cunniff TM, Wanaski SP, Atkinson SD, Mahableshwarkar AR. Ecopipam for Tourette Syndrome: A Randomized Trial. Pediatrics 2023. doi: 10.1542/2020-000123. PMID: 36628546
• Gilbert DL, Kim DJB, Miller MM, Atkinson SD, Karkanias GB, Munschauer FE, Wanaski SP, Cuniff TM. Safety and Effect of 12-month Ecopipam Treatment in Pediatric Patients with Tourette Syndrome. Movement Disorders Clinical Practice. 2025. doi 

Contact author

Thank you Don for your time. and expertise!