Parkdale

Researchers are conducting a phase II randomized placebo-controlled trial to study oral N-Acetylcysteine NAC in individuals who carry the Huntington disease gene but do not yet show clear motor symptoms. The study aims to assess clinical and brain imaging outcomes over a three-year period, focusing on slowing disease progression in this premanifest stage. Participants will be randomly assigned to take either 1 gram of NAC capsules or matching placebo capsules twice daily by mouth. The study is quadruple-masked, meaning that participants, caregivers, investigators, and outcome assessors do not know which treatment is assigned. The treatment period lasts up to three years, with ongoing monitoring throughout. During the study, participants will undergo regular brain MRI scans to measure caudate atrophy rate and be evaluated for motor changes indicating disease progression. Various cognitive and behavioral tests, such as the UHDRS motor subscale, Stroop Word test, and Montreal Cognitive Assessment, will be performed at intervals over the three years. Safety will be monitored through laboratory tests, ECGs, and tracking of adverse events. Participants must comply with study procedures including blood draws and MRI scans for the full duration up to the study end in May 2027.