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VANCOUVER — Treatment with ecopipam reduces tic severity and is generally well tolerated compared with placebo in children and adolescents with Tourette syndrome, according to findings presented at the 2017 International Congress of Parkinson’s Disease and Movement Disorders.
Ecopipam, an investigational selective D1 receptor antagonist, previously demonstrated efficacy for the treatment of tics in adults with Tourette syndrome in a phase 2 clinical trial (ClinicalTrials.gov: NCT01244633). In the current phase 2 study (ClinicalTrials.gov: NCT02102698), a team of investigators led by Donald Gilbert, MD, MS, FAAN, FAAP, of Cincinnati Children’s Hospital in Ohio, sought to investigate the safety and efficacy of ecopipam in patients aged 7 to 17 years.
The double-blind, placebo-controlled, crossover study included 40 patients with a Yale Global Tic Severity Scale (YGTSS) total tic score of 20. Patients enrolled in the study were not allowed to take ADHD-related stimulants or D2 receptor blocking agents, alpha2 noradrenergic agonists, or other medications for tics. Patients were randomly assigned to receive either ecopipam (50 mg/d for weight <34 kg; 100 mg/d for weight >34 kg) or placebo for 4 weeks, followed by a 2-week washout period and 4-week crossover.
At the 2-week and 4-week evaluations, reductions in total YGTSS scores were greater in the treatment group compared with placebo (P ≤.05). Similar reduction was seen for motor and phonic tics (motor, 16 and 30 days: P ≤.05; phonic, 16 days: P ≤.05 and 30 days: P ≤.09). At 4 weeks, 38% of patients in the treatment group showed “much” or “very much” improvement compared with 18% in the placebo group (P =.08). The proportion of patients whose symptoms were in the “marked” or “severe” range dropped from 40% to 15% after treatment, and the proportion of patients with “mild” symptoms increased from 2.5% to 45%.
Overall, adverse events were mainly mild to moderate, including somnolence, gastrointestinal symptoms, nasopharyngitis, and headache. Four incidents were rated as severe, including headache and insomnia, and 1 was unrelated to treatment. Notably, patients in the treatment group experienced neither weight gain, drug-induced movement disorders, nor changes in renal or hepatic values, electrocardiograms, or vital signs.
“Neurologists have long wondered why there were no D1 receptor antagonists available to treat patients with hyperkinetic movement disorders,” Dr Gilbert told Neurology Advisor. “This phase 2, placebo-controlled study of the D1 receptor antagonist ecopipam shows promise for effectively treating tics in adolescents with Tourette syndrome.”
Disclosures: The study was supported by Psyadon Pharmaceuticals.
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Gilbert D, Murphy T, Jankovic J, et al. A randomized, double-blind, placebo-controlled study of the D1 receptor antagonist ecopipam for children and adolescents with Tourette syndrome. Presented at: 2017 International Congress of Parkinson’s Disease and Movement Disorders. June 4-8, 2017; Vancouver, BC, Canada. Abstract 1185.