The investigational medication ecopipam can significantly reduce Tourette syndrome symptoms in children—and possibly adults—over 24 weeks, according to a clinical study published today in JAMA Neurology.Why It’s Relevant
Currently approved medications for Tourette-associated tics are the dopamine D2 receptor–targeting antipsychotics haloperidol, pimozide, and aripiprazole—all of which come with metabolic side effects that can be especially difficult for children. The anti-adrenergic medications clonidine and guanfacine are also prescribed off label, but the former can cause excess sedation while the latter isn’t particularly effective.
Ecopipam is a dopamine D1 receptor antagonist that has shown a good combination of efficacy and tolerability to date, including in a recent Phase 2 clinical trial. This new Phase 3 study explored the effectiveness of maintenance ecopipam on tic relapse.
By the Numbers
- To start, 216 participants ages 6 and older received 12 weeks of open-label ecopipam (target dose of 1.8mg/kg per day). Those whose tic symptoms improved by at least 25% were randomized to continue ecopipam or switch to placebo for a further 12 weeks.
- During the randomized period, youth (ages 6 to 18) continuing ecopipam were 53% less likely to experience symptom relapse compared with those on placebo.
- Adults taking ecopipam had a 49% reduced risk of relapse, but the sample size (14 participants) was too small to demonstrate statistical significance.
- The most frequent side effects among participants taking ecopipam were sleepiness (11%), anxiety (10%), and headache (10%). Side effects were generally mild to moderate and mainly occurred during the first 12 weeks.
What’s More
Ecopipam did not worsen any psychiatric symptoms among participants with comorbidities such as attention-deficit/hyperactivity disorder or obsessive-compulsive disorder.
The Other Side
In addition to having a limited number of adult participants, the study cohort had minimal racial and ethnic diversity. Further, some symptom relapses occurred during the 12-week open-label phase, which could affect ecopipam’s true efficacy rate.
Takeaway Message
“The safety profile of ecopipam is notable for the absence of clinically relevant weight gain, adverse metabolic effects, and drug-induced movement disorders,” the researchers wrote. Given the positive efficacy and safety findings from two randomized trials, they think that ecopipam could be a novel pharmacologic option for children, adolescents, and adults with Tourette syndrome.
Related Information
Source
Donald L. Gilbert, et al. Efficacy and safety of ecopipam for Tourette syndrome: a phase 3 randomized clinical trial. JAMA Neurology. Published May 26, 2026. doi: 10.1001/jamaneurol.2026.1431
(Image: Getty Images/iStock/Andrii Dodonov)
