MSA-01 in Multiple System Atrophy

Multiple system atrophy (MSA) is a progressive neurodegenerative disorder with no established disease-modifying treatment. Evidence suggests that coenzyme Q10 (CoQ10) deficiency may contribute to MSA pathophysiology. MSA-01 (ubiquinol), a highly bioavailable form of CoQ10, demonstrated acceptable safety and potential efficacy in a prior phase II trial.

This is a multicenter, randomized, double-blind, placebo-controlled phase III study evaluating the efficacy and safety of MSA-01 in patients with MSA. Approximately 140 participants will be randomized 1:1 to receive oral MSA-01 or placebo for 52 weeks.

The primary endpoint is the change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score. Secondary endpoints include additional clinical scales and safety assessments. Efficacy will be analyzed using a mixed-effects model for repeated measures. The study aims to determine whether MSA-01 slows clinical progression compared with placebo while maintaining an acceptable safety profile.

Inclusion criteria

At the time of informed consent

• Patients diagnosed as 'clinically established' or 'clinically probable' MSA based on the revised MSA diagnostic criteria of the Movement Disorder Society (MDS).
• Patients who are able to walk independently or with the use of assistive devices.
• Patients who are able to attend outpatient visits at the participating study site.

At the start of study drug administration

• Patients who are able to discontinue the use of CoQ10 supplements.

Exclusion criteria

• Patients with severe neurological disorders, other progressive movement disorders, or cognitive impairment.
• Patients with severe liver disease.
• Patients with a known history of hypersensitivity to any component of the investigational drug.
• Pregnant women, breastfeeding women, or women who may be pregnant.
• Patients who have previously participated in a clinical trial of MSA-01.