First Patient Enrolled in Pediatric Tourette’s Syndrome Clinical Trial

According to a story from BioPortfolio, the biopharmaceutical company Emalex Biosciences, Inc., has recently announced that the very first patient has been signed up for the company’s phase 2b clinical trial. This clinical trial is testing the company’s experimental drug ecopipam in children and teens with Tourette’s syndrome, a rare neurodevelopmental disorder. Emalex is committed to the development of therapies for psychiatric and neurological diseases.

About Tourette’s Syndrome

Tourette’s syndrome, often known simply as Tourette’s, is an unusual disorder of neurodevelopment that is defined by the appearance of tics, which are brief involuntary sounds or movements. Patients often present with multiple tics that can vary substantially in severity. Most cases are mild and barely noticeable by casual observers. The precise cause of Tourette’s syndrome remains unknown; the condition may be heritable in some cases, and it has been well established that both environmental and genetic factors play a role. Treatment for Tourette’s syndrome may include cognitive behavioral therapy, education, and, in severe cases, certain medications. There is no cure for the condition. Symptoms resolve in many patients as they near adulthood. Severe cases of Tourette’s, such as those in which tics include outbursts of profanity, have occasionally gained attention over social media. Mental health problems may develop in patients with severe Tourette’s. To learn more about Tourette’s syndrome, click here.

About Ecopipam and The Clinical Trial

Ecopipam is designed as an inhibitor of dopamine activity at the D1 receptor. The phase 2b trial is expected to include around 150 patients between the ages of 6 and 18. The treatment period will last 12 weeks and patients will receive either a placebo or a daily dose of ecopipam of 2mg/kg. The drug will be evaluated on its ability to reduce the rate of tic symptoms (both motor and vocal) that the patient experiences.

Prior studies have show ecopipam to have good tolerability in human subjects; adverse effects were generally minor and included vomiting, nausea, sedation, and insomnia. Tourette’s syndrome patients should keep an eye on the progress of this study, as this experimental drug has the potential to be a new effective treatment for the condition.


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