Infectious Disease

New drug improved tic severity in children with Tourette syndrome

April 08, 2022

1 min read

Source/Disclosures

sources:

Gilbert DL, et al. ES1.002: Ecopipam in children and adolescents with Tourette syndrome: Results from a randomized, double-blind, placebo-controlled phase 2b study. Presented at: American Academy of Neurology annual meeting; April 2-7, 2022; Seattle.

Disclosures:
Gilbert reports no relevant financial disclosures.

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SEATTLE — A decrease in the severity of tics in children and adolescents with Tourette syndrome was observed following the use of ecopipam, according to data presented at the 2022 American Academy of Neurology annual meeting.

“Our results are exciting because they suggest ecopipam shows promise as a treatment for reducing the number, frequency and severity of the tics young people experience with Tourette syndrome,” study author Donald L. Gilbert, MD, AAN fellow and professor and neurologist at Cincinnati Children’s Hospital Medical Center, said in a released statement. “That’s especially true because many people with the disease who are taking the medications currently available still have debilitating symptoms or experience weight gain or other side effects.”

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Gilbert and colleagues assessed 149 children and adolescents with Tourette syndrome, aged 6 to 17 years, in a phase 2b trial. Participants were randomized at a 1:1 ratio — 74 were given the experimental drug ecopipam, and 75 were given a placebo.

Researchers assessed tic severity at baseline and at 3 months using two common tic rating scales: The first, which measures motor and vocal tics, has a maximum score of 50, and the second, which evaluates overall symptoms and severity, has a maximum score of 100

On average, participants taking ecopipam improved motor and vocal tic severity scores from 35 to 24, a decrease of 30%, compared with those taking placebos, who had a decrease of 19%, with scores dropping from 35 to 28.

When assessing the drug’s overall effectiveness, researchers reported those taking ecopipam had an average improved score of 68 to 46, a decrease of 32%, compared with those given placebo, who improved from 66 to 54, a decrease of 20%.

According to Gilbert, 34% of participants taking ecopipam experienced side effects, which included headaches and fatigue.

“Previous research suggests problems with dopamine, a neurotransmitter in the brain, may be linked to symptoms of Tourette syndrome, and that D1 dopamine receptors play a key role,” Gilbert said. “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.”

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American Academy of Neurology Annual Meeting

American Academy of Neurology Annual Meeting

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