Objective: To evaluate ecopipam efficacy/safety in individuals with Tourette syndrome (TS) with or without common psychiatric comorbidities.

Background: Co-occurring psychiatric conditions are frequently observed with TS and may influence TS-related treatment decisions.

Method: Individuals aged ≥6 years with TS were enrolled in a phase 3, double-blind, placebo (PBO)-controlled, randomized withdrawal trial. Ecopipam 1.8 mg/kg/day was administered in a 12-week, open-label (OL) period. Those with ≥25% Yale Global Tic Severity Scale Total Tic Score (YGTSS-TTS) reduction at Weeks 8 and 12 were randomized to ecopipam or PBO during a 12-week double-blind period. Individuals were subgrouped post hoc by co-occurring anxiety, attention-deficit/hyperactivity disorder (ADHD), depression, or obsessive-compulsive disorder (OCD), determined from medical/psychiatric history at screening. Analyses included mean (SD) YGTSS-TTS change from baseline at Week 12 and time from randomization (Week 12) to relapse (≥50% loss of OL period YGTSS-TTS improvement) for ecopipam vs PBO.

Results: Overall, 216 participants were enrolled, 129 (59.7%) of whom had ≥1 comorbidity (ADHD [40.7%]; OCD [20.4%]; anxiety [18.5%]; depression [7.9%]), and 104 were randomized to ecopipam (n=51 [64.7% with ≥1 comorbidity]) or PBO (n=53 [54.7% with ≥1 comorbidity]). Baseline parameters were similar between enrolled participants with/without co-occurring conditions (mean age, 16.4/16.1 years; 65.1%/71.3% male; mean YGTSS-TTS, 34.9/33.4). Mean (SD) YGTSS-TTS change from baseline at Week 12 was −16.1 (7.6) in participants with ≥1 comorbidity and −15.7 (6.8) in those without. During the randomized withdrawal period, ecopipam showed a reduced risk of relapse vs PBO in participants with ≥1 comorbidity (hazard ratio, 0.6 [95% CI, 0.3-1.1]; P=0.09) and without ≥1 comorbidity (0.3 [0.1-0.9]; P=0.02), with statistical significance in the latter group. The most commonly reported adverse events during ecopipam treatment for those with vs without ≥1 comorbidity were anxiety (11.6% vs 6.9%), tic (10.9% vs 3.4%), insomnia (10.1% vs 6.9%), depressed mood (4.7% vs 2.3%), and irritability (2.3% vs 4.6%).

Conclusion: The presence of commonly co-occurring psychiatric comorbidities (ie, anxiety/ADHD/depression/OCD) with TS did not negatively impact reductions in tic severity or the safety/tolerability of ecopipam in individuals aged ≥6 years.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/presence-of-common-psychiatric-diagnoses-did-not-impact-the-efficacy-or-safety-of-ecopipam-in-individuals-with-tourette-syndrome/