NCT07446894

MSA-01 in Multiple System Atrophy

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Study of MSA-01 in Patients With Multiple System Atrophy

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 140 (estimated)
• Sponsor: Tokyo University · Academic / Other
• Sex: All
• Age: 30 Years – 79 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Multiple System Atrophy (MSA)

Interventions

Type	Name	Description
DRUG	MSA-01	Ubiquinol
DRUG	Placebo	Placebo

Timeline

• Start date: 2026-02-09
• Primary completion: 2028-03-31
• Completion: 2028-03-31
• First posted: 2026-03-03
• Last updated: 2026-03-04

Locations

12 sites across 1 country: Japan