MONDAY, June 12, 2023 (HealthDay News) -- Valbenazine results in improvement in chorea compared with placebo for patients with Huntington disease, according to a study published in the June issue of The Lancet Neurology.
Erin Furr-Stimming, M.D., from the University of Texas Health Science Center in Houston, and colleagues conducted a phase 3 trial from Nov. 13, 2019, to Oct. 26, 2021, involving adults with genetically confirmed Huntington disease and chorea who were randomly assigned to oral placebo or valbenazine for 12 weeks of treatment. A total of 125 participants were included in the full analysis (64 assigned to valbenazine and 61 to placebo) and 127 were included in the safety analysis (64 and 63, respectively). The primary end point was the least-squares mean change in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) score from the screening and baseline period to the maintenance period (based on the average of weeks 10 and 12 values for each participant).
The researchers found that the least-squares mean changes from the screening and baseline to the maintenance periods for the UHDRS TMC score were −4.6 and −1.4 for valbenazine and placebo, respectively. Somnolence was the most commonly reported treatment-emergent adverse event. There were no clinically important changes in vital signs, electrocardiograms, or laboratory tests during the 12-week period. Participants treated with valbenazine did not have suicidal behavior or worsening of suicidal ideation.
"Positive study results remind us there is a reason for hope," Furr-Stimming said in a statement. "Chorea associated with Huntington's disease can negatively impact quality of life and functional independence; therefore, studying additional medications to address this hallmark motor symptom is imperative."
Several authors disclosed ties to Neurocrine Biosciences, which manufactures valbenazine and funded the study.
Abstract/Full Text (subscription or payment may be required)
Editorial (subscription or payment may be required)
