Phase III Study of MSA-01 for Treating Multiple System Atrophy in Adults Aged 30 to 79

What this Trial Is About

Researchers are evaluating whether MSA-01, a highly bioavailable form of coenzyme Q10 ubiquinol, can slow the progression of multiple system atrophy MSA, a progressive neurodegenerative disorder with no established disease-modifying treatment. This multicenter, randomized, double-blind, placebo-controlled phase III study aims to assess the safety and effectiveness of MSA-01 compared to placebo in patients with MSA. The primary focus is on changes in motor impairment using the Unified Multiple System Atrophy Rating Scale UMSARS Part 2 score over one year. Participants will be randomly assigned to receive either oral MSA-01 or a placebo daily for 52 weeks. The study includes regular clinic visits for safety and efficacy assessments throughout the treatment period. Patients are asked to record their medication use and any side effects in a diary to help monitor their experience with the study drug or placebo. During the study, participants will undergo evaluations using clinical scales including UMSARS Part 1 and Part 2, the Barthel Index, and the Scale for the Assessment and Rating of Ataxia SARA. Safety assessments will be conducted regularly, and researchers will analyze data using statistical models to compare progression between groups. The total participation duration is about one year, with a focus on assessing changes from baseline to Week 52.

MSA-01 in Multiple System Atrophy