Neuroactive Steroid for Essential Tremor Reduced Tremor Amplitude 

In the phase 2 KINETIC study (NCT04305275) individuals treated with neuroactive steroid (SAGE-324; Sage Therapeutics and Biogen, Cambridge, MA) for essential tremor (ET) had reduced tremor amplitude. Those treated with the steroid had a 36% reduction in upper limb tremor compared with a 21% reduction in those treated with placebo (P=.049). Among those with a more severe tremor at baseline (n=47), those treated with the steroid had a 41% reduction from baseline in upper limb tremor amplitude compared with an 18% reduction in those with severe tremor treated with placebo (P=.007). 

“There is an extraordinary unmet need for people with essential tremor, a condition that can cause significant disability in patients,” said Dr. Rodger Elble, MD, a neurologist at the Southern Illinois University School of Medicine. “The only approved medicine was developed more than 50 years ago, most medicines used for ET were developed for other conditions with benefits to people with ET discovered serendipitously. Current investigational drugs like SAGE-324, if successful, may offer potential for new treatment options for tremor management, as more than 50% of people with ET do not respond optimally to the current standard of care.”

Participants were randomly assigned 1:1 to receive the investigational steroid (60 mg/day) or placebo. The trial evaluated treatment at the higher end of the dose range and the daily dose could titrated down to 45 mg or 30 mg if tolerability was poor. Dose reductions occurred in 62% of participants who received SAGE-324 and discontinuations were noted in 38% of participants receiving SAGE-324. The participants were not taking other medication during the 28-day treatment period.

The most common adverse events that occurred in ≥10% of participants were balance disorder (15%), diplopia (12%), dizziness (38%), dysarthria (12%), gait disturbance (12%), and somnolence (68%).