Objective: Evaluate the efficacy and safety of ecopipam in children and adolescents with Tourette’s Syndrome (TS).

Background: Ecopipam, a first-in-class selective dopamine-1 (D1) receptor antagonist associated with low metabolic and movement-related adverse events (AE), is in clinical development for patients with TS. In previous clinical studies in adult and pediatric patients, ecopipam reduced tics with an acceptable safety profile.

Method: Patients with TS were randomized 1:1 to ecopipam or placebo for a 4-week titration period, an 8-week maintenance period, and a 1-week taper period. The primary endpoint was mean change from Baseline (BL) to Week 12 for the Yale Global Tic Severity Score – Total Tic Score(YGTSS-TTS). Clinical Global Impression of Tourette Syndrome Severity and Improvement (CGI-TS-S and CGI-TS-I), YGTSS-Global Score (GS), Caregiver Global Impression of Change (CaGI-C), and a QoL scale were secondary endpoints. Safety and tolerability were evaluated at each study visit.

Results: 153 patients were randomized, and 149 were included in the modified intent-to-treat population (74 ecopipam, 75 placebo). A significant (p=0.011, LS mean [SE] difference: -3.4 [1.4], 95% CI: -6.1, -0.8 ) improvement in the YGTSS-TTS from BL to Week 12 was observed for ecopipam vs. placebo as well as among those ages 6 to 11 years (nominal p=0.017, LS mean [SE] difference: -4.4 [1.8], 95% CI: -8.0, -0.8) and those ages 12 to 17 years (nominal p=0.004, LS mean [SE] difference: -3.4 [1.2], 95% CI: -5.7, -1.1). Mean change from BL to Week 12 was significant for CGI-TS-S (p=0.001) and YGTSS-GS (p=0.004), trended towards significance for CGI-TS-I (p=0.079), CaGI-C (p<0.05 and was not significant for QoL (p=0978). Treatment-related AEs occurred in 26 (34%) patients with ecopipam and 16 (21%) with placebo; most common 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. From BL to Week 12, mean (SD) AIMS score decreased from 6.9 (9.6) to 4.3 (6.8) and mean (SD) BARS score decreased from 1.3 (2.1) to 1.0 (1.9) with ecopipam.

Conclusion: Ecopipam reduced motor and phonic tics among children and adolescents with TS. In this study ecopipam was well tolerated and demonstrated no effects across movement disorder scales at Week 12.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/randomized-double-blind-placebo-controlled-phase-2b-study-of-ecopipam-in-children-and-adolescents-with-tourette-syndrome/