Ecopipam Reduced Tic Severity in Tourette Syndrome 

In a clinical trial (NCT04007991) of ecopipam, children and teens (age 6 to 17 years) with Tourette syndrome who were treated with ecopipam for 3 months had improved scores on tests of tic severity after 3 months. Among those who were treated with ecopipam, motor and vocal tic severity decreased by 30% from baseline, compared with a 19% decrease in those treated with placebo. 

“Previous research suggests that problems with dopamine may be linked to symptoms of Tourette syndrome, and that D1 dopamine receptors play a key role,” said Donald L. Gilbert, MD, professor of pediatrics and neurology, Cincinnati Children’s Hospital Medical Center. “While ecopipam is still in the testing phase, it is the first drug to target the D1 receptor instead of the D2 receptor, which is the one targeted by medications currently on the market. Our results demonstrate that ecopipam deserves more study as a viable treatment option for Tourette syndrome in young people in the future.”

The most common side effects with ecopipam treatment were headaches and fatigue seen in 34% of those who took ecopipam and 21% of those treated with placebo. 
      
This research is being presented at the American Academy of Neurology 74th Annual Meeting in Seattle, WA, April 2 to 7, 2022; and virtually, April 24 to 26, 2022.