Huntington’s disease (HD) is a life-threatening genetic disorder that causes a gradual and progressive breakdown of nerve cells in the brain. It affects a person’s mental and physical abilities and is currently incurable. But in recent years, pridopidine has shown to reduce the motor impairment associated with the disease. This study aims to evaluate the efficacy and safety of pridopidine in patients with HD.

This is a randomized, placebo-controlled, dose-ranging, phase-2 trial conducted in a total of 408 adults aged 20 or more and had confirmed Huntington’s disease. Patients were randomly assigned in a 1:1:1:1:1 ratio to 45 mg (n=75), 67.5 mg (n=75), 90 mg (n=81), or 112.5 mg (n=81) pridopidine or placebo (n=81) orally twice a day for 52 weeks. The primary outcome was the change in the Unified Huntington’s Disease Rating Scale total motor score (UHDRS-TMS).

At 26 weeks of follow-up, the use of pridopidine did not result in a change in the UHDRS-TMS score compared with the placebo. Serious adverse events occurred in the pridopidine group only, most common ones being falls, suicide attempts, suicidal ideation, head injury, and aspiration pneumonia.

The research concluded that pridopidine did not improve UHDRS-TMS in patients with Huntington’s disease, as compared with placebo.

Ref: https://www.thelancet.com/journals/laneur/article/PIIS1474-4422(18)30391-0/fulltext