2026 American Academy of Neurology Abstract Website

Mohamed Khalafalla Darwish¹, Mohamed Nasser², Hazem E. Mohammed³, Mohamed E. Haseeb², Mohamed Salem Abdelqader¹, Mohamed Khamis Abdullah⁴, Zeyad Bady³, Mostafa Meshref⁵
¹Faculty of Medicine, Minia University., ²Faculty of Medicine, Minia University.., ³Faculty of Medicine, Assiut University., ⁴Faculty of Medicine, Suez University., ⁵Neurology department, Faculty Of Medicine, Al-Azhar University.

Objective:

We aimed to evaluate Ecopipam's efficacy and safety in reducing involuntary tics in TS according to different scales, such as the Yale Global Tic Severity Scale (YGTSS), the Clinical Global Impression of Improvement (CGI-Improvement), and the CGI-Severity scale. Furthermore, we gave information about the drug's safety profile and tolerability.

Background:

Tourette syndrome is a neurodevelopmental disorder characterized by the presence of involuntary motor and vocal tics that typically emerge in childhood. Ecopipam, a selective D1 receptor antagonist, has been used to treat Tourette syndrome. (TS).

Design/Methods:

All clinical trials involving Tourette syndrome patients were considered. PubMed, Scopus, Cochrane Central Register of Controlled Trials (CENTRAL), and Web of Science were searched for studies published up until December 2024.

Results:

A total of 76 records were assessed for eligibility after our search strategy. Eventually, two randomized controlled trials and one single-arm trial were included. Ecopipam showed a statistically non-significant reduction in terms of YGTSS total tic score compared to placebo at week 4 (mean difference (MD): -1.73, 95% confidence interval (CI): [-4 to 0.54], P-value = 0.14). It also exhibited statistically significant reductions in terms of CGI-severity and CGI-improvement with P-values of 0.03 and 0.009, respectively. According to safety, ecopipam had a non-significant incidence of nausea, fatigue, somnolence, and headache; only insomnia was statistically significant when compared to placebo.

Conclusions:

Ecopipam demonstrates potential efficacy at reducing tics in TS patients while also having a favorable safety profile. However, because of the small sample size and restricted number of included studies, additional follow-up research is required.