Objective: Assess the safety and durability of effect of long-term treatment with ecopipam in pediatric patients with Tourette syndrome (TS).

Background: TS is a childhood-onset movement disorder characterized by multiple motor and phonic tics that can disrupt function and can cause pain and self-injury. Currently approved medications are antipsychotics associated with weight gain, drug-induced dyskinesias, and other side effects. Ecopipam, a first-in-class dopamine 1 receptor antagonist, significantly reduced tics in the preceding 12-week Phase 2b (D1AMOND) randomized, double-blind, placebo-controlled clinical trial.

Method: Children and adolescents were eligible for this OLE study if they completed the D1AMOND study and if the family sought continued treatment. After a 4-week up-titration, patients received a 2 mg/kg/day oral dose of ecopipam nightly for up to 12 months. The primary outcome was incidence of treatment related adverse events. The primary efficacy endpoint was the Yale Global Tic Severity Scale-Total Tic Score (YGTSS-TTS). This study is registered with ClinicalTrials.gov (NCT04114539).

Results: Of 153 eligible patients, 124 enrolled, 121 received ecopipam, and 80 completed the study. Withdrawals due to adverse events occurred in 14 (11.3%) patients. The most frequent treatment related adverse events were anxiety (6.6%), insomnia (5.8%), somnolence (5.8%), and depression (4.1%). No clinically significant changes were observed in lab values, vital signs, ECG, physical exams, the Columbia Suicide Severity Rating Scale, Abnormal Involuntary Movement Scale, Barnes Akathisia Rating Scale, Children’s Depression Rating Scale-Revised, or the Pediatric Anxiety Rating Scale. Median YGTSS-TTS was 30.0 at baseline (n=121). YGTSS-TTS scores were meaningfully improved at 3 months (-7.8 ± 8.1; n=106), 6 months (-11.0 ± 8.8; n=87), 9 months (-11.6 ± 8.5; n=85), and 12 months (-11.8 ± 9.6; n=77); all p < 0.0001 compared to baseline. Similarly, the Clinical Global Impression of TS-Severity, the YGTSS-Global Score, and the Child & Adolescent Gilles De La Tourette Syndrome-Quality of Life Total Score each improved at all time points; all p < 0.001.

Conclusion: In this OLE study in children and adolescents with TS, ecopipam demonstrated an acceptable safety profile and continued or improved control of tics.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/long-term-safety-and-durability-of-effect-of-ecopipam-in-pediatric-patients-with-tourette-syndrome-results-of-a-12-month-open-label-extension-ole-study/