A recent study evaluating the efficacy and safety of a pharmaceutical treatment, ecopipam, for children with Tourette syndrome (TS), showed promising initial results, according to a study released today at the International Congress of Parkinson's Disease and Movement Disorders® in Madrid, Spain.
MILWAUKEE, Sept. 15, 2022 /PRNewswire-PRWeb/ -- A recent study evaluated the efficacy and safety of a pharmaceutical treatment, ecopipam, for children with Tourette syndrome (TS), according to a study released today at the International Congress of Parkinson's Disease and Movement Disorders® in Madrid, Spain. (Mahableshwarkar et al., 2022)
Ecopipam works by selectively reducing the response to dopamine in neurons. Importantly, ecopipam is known for having a low incidence of adverse effects and was well-tolerated during the study. The double-blind study lasted 12 weeks and examined children with Tourette's ranging from 6 to 17 years old. During the study, reductions in both motor and phonic tics compared to the placebo control group were reported. These promising results will require follow-up, longer-term studies.
Davide Martino, MD, PhD, Associate Professor in the Department of Clinical Neurosciences at the University of Calgary, commented, "Despite several new or re-purposed medications appeared promising for the treatment of tics in recent years, no other drug confirmed effectiveness in randomized controlled trials since the addition of aripiprazole to the therapeutic arsenal of Tourette syndrome. This phase 2b randomized trial breaks the spell by showing significant superiority to placebo of a first-in-class selective dopamine D1 receptor antagonist, ecopipam, in reducing tic severity in children and adolescents with TS. This study is relevant for its adequate sample size and selection of primary and secondary outcomes. Ecopipam showed superiority to placebo in reducing tic severity, based on both clinician's and caregiver's impression, but did not differ from placebo in reducing quality of life. This is not surprising, given that quality of life in TS is often influenced more by comorbid symptoms like those related to attention deficit hyperactivity disorder, obsessive-compulsive disorder, anxiety and depression."
Martino continues, "The treatment duration, albeit standard for clinical trials in tic disorders, was not very long and the observed difference from placebo was not large, hence longer-term monitoring of response and tolerability will be necessary. The side effect profile of ecopipam was also reassuring from this study, with the only caveat that tremor and parkinsonism were not specifically measured. Overall, this phase 2b study generates hope and supports that ecopipam deserves further investigation as a new treatment option for tics in TS."
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Reference #: 917
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The MDS International Congress is the premier annual event to advance the clinical and scientific discipline of Movement Disorders, including Parkinson's disease. Convening thousands of leading clinicians, scientists and other health professionals from around the globe, the International Congress will introduce more than 1,500 original scientific abstracts and provide a forum for education and collaboration on latest research findings and state-of-the-art treatment options.
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REFERENCES
Mahableshwarkar, A. et al. (2022, September 15). Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study of Ecopipam in Children and Adolescents with Tourette Syndrome [abstract]. In: Movement Disorders journal online supplement; International Congress of Parkinson's Disease and Movement Disorders®. Retrieved from https://www.mdsabstracts.org/, Reference #917.
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