Overview
The purpose of this study is to evaluate whether MSA-01 slows the progression of multiple system atrophy (MSA) and to assess its safety.
The primary question is:
• Does MSA-01 slow the progression of motor impairment as measured by UMSARS Part 2 score?
Participants will be randomly assigned to receive MSA-01 or placebo for 12 months. They will attend regular clinic visits for safety and efficacy assessments and record their medication use and any side effects in a diary.
Description
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.
Eligibility
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.
