MSA-01 to try to slow progression of Multiple System Atrophy

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

This trial will test whether MSA-01 (a bioavailable form of CoQ10) can slow motor worsening in people with probable or established MSA over 52 weeks.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 140 (estimated)
• Ages: 30 Years to 79 Years
• Sex: All
• Sponsor: Tokyo University (Academic / other)
• Locations: 12 sites (Nagoya, Aichi-ken and 11 other locations)
• Trial ID: NCT07446894 on ClinicalTrials.gov

What this trial studies

This is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial randomizing about 140 participants 1:1 to oral MSA-01 or placebo for 52 weeks. The primary outcome is change from baseline to Week 52 in the UMSARS Part 2 motor score, with secondary clinical scales and safety assessments also collected. Participants attend regular clinic visits, keep a medication/side-effect diary, and efficacy will be analyzed using a mixed-effects model for repeated measures. The trial builds on a Phase 2 signal of acceptable safety and potential benefit for ubiquinol in MSA.

Who should consider this trial

Good fit: Ideal candidates are adults with clinically established or probable MSA who can walk (with or without assistive devices), can attend outpatient visits at a participating site, and can stop taking CoQ10 supplements before starting the study drug.

Not a fit: Patients with severe other neurological disorders or cognitive impairment, severe liver disease, pregnant or breastfeeding women, or those who have previously received MSA-01 are unlikely to qualify or benefit from this trial.

Why it matters

Potential benefit: If successful, MSA-01 could slow motor progression and help preserve daily function in people with MSA.

How similar studies have performed: A prior Phase 2 trial of this ubiquinol formulation showed acceptable safety and signals of potential efficacy, but benefit has not yet been confirmed in a large Phase 3 trial.

Eligibility criteria

Inclusion Criteria:

At the time of informed consent

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

   At the start of study drug administration
4. Patients who are able to discontinue the use of CoQ10 supplements.

Exclusion Criteria:

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

Where this trial is running

Nagoya, Aichi-ken and 11 other locations

• Nagoya University Hospital — Nagoya, Aichi-ken, Japan (Not_yet_recruiting)
• Chiba University Hospital — Chiba, Chiba, Japan (Not_yet_recruiting)
• Kyushu University Hospital — Fukuoka, Fukuoka, Japan (Not_yet_recruiting)
• Hokkaido University Hospital — Sapporo, Hokkaido, Japan (Not_yet_recruiting)
• Kagoshima University Hospital — Kagoshima, Kagoshima-ken, Japan (Not_yet_recruiting)
• Kyoto University Hospital — Kyoto, Kyoto, Japan (Not_yet_recruiting)
• Okayama University Hospital — Okayama, Okayama-ken, Japan (Not_yet_recruiting)
• NHO Higashisaitama National Hospital — Hasuda, Saitama, Japan (Not_yet_recruiting)
• Institute of Science Tokyo Hospital — Bunkyō-Ku, Tokyo, Japan (Not_yet_recruiting)
• The University of Tokyo Hospital — Bunkyō-Ku, Tokyo, Japan (Recruiting)
• National Center Hospital, National Center of Neurology and Psychiatry — Kodaira, Tokyo, Japan (Not_yet_recruiting)
• Tottori University Hospital — Yonago, Tottori, Japan (Not_yet_recruiting)

Study contacts

• Study coordinator: Jun Mitsui

Conditions Multiple System Atrophy