Objective: To describe the Phase 2b study design and optimized dosing strategy to evaluate the efficacy and safety of ecopipam in children and adolescent subjects with Tourette’s syndrome (TS).

Background: Ecopipam, is a selective D1 antagonist, designed to provide D2-like efficacy while minimizing D2-related adverse events. A previous phase 2a, randomized, double-blind, 28-day, placebo-controlled crossover study suggested preliminary efficacy of ecopipam at a fixed daily dose for the treatment of children and adolescents with TS without resulting in extrapyramidal or metabolic adverse events or weight gain.

Method: This is a multicenter, placebo-controlled, double-blind, randomized parallel group, Phase 2b study to evaluate the efficacy and safety of ecopipam tablets in children and adolescents with TS. This study will enroll patients with TS >6 to <18 years of age, >18 kg with both motor and vocal tics that cause impairment in normal routines and >20 on the Yale Global Tic Severity Score Total Motor and Phonic Tic Score (YGTSS-TTS), not currently on any anti-tic medications. The study will exclude patients with significant medical or psychiatric disease. Based on pharmacokinetic modeling and simulations, ecopipam will be dosed on a weight basis for consistency across the pediatric patient population. Following a screening period, subjects will be randomized to ecopipam 2 mg/kg (6 distinct weight bands) or matching placebo and titrated to the appropriate dose over 4 weeks followed by an 8-week treatment period.

Results: 75 subjects per group will be enrolled in the study. The primary endpoint will be change from Baseline to Week 12 in the YGTSS-TTS. The key secondary endpoint will be change from Baseline to Week 12 in the Clinical Global Impression of Tourette Syndrome Severity. Other secondary endpoints include the YGTSS Impairment Score, Total YGTSS, Clinical Global Impression Improvement, potential PK/PD relationships, and other quality of life measures. Safety assessments will include adverse events, vital signs, and measurements of akathisia, abnormal involuntary movements, suicidality, anxiety, depression and obsessive-compulsive disorder.

Conclusion: This study is designed to determine whether ecopipam is efficacious in the treatment of TS and to further characterize the safety profile of this drug in children and adolescent patients.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/ecopipam-a-selective-d1-antagonist-in-development-for-the-treatment-of-tourette-syndrome-in-children-and-adolescents-the-phase-2b-d1amond-study/