MSA-01 in Multiple System Atrophy

Last updated: March 2, 2026
Sponsor: Tokyo University
Overall Status: Active - Recruiting

Phase

3

Condition

Multiple System Atrophy

Treatment

MSA-01

Placebo

Clinical Study ID

NCT07446894
2025024-11DX
jRCT2031250696
  • Ages 30-79
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

At the time of informed consent

  1. Patients diagnosed as 'clinically established' or 'clinically probable' MSA based onthe 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

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 theinvestigational 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.

Study Design

Total Participants: 140
Treatment Group(s): 2
Primary Treatment: MSA-01
Phase: 3
Study Start date:
February 09, 2026
Estimated Completion Date:
March 31, 2028

Study 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.

Connect with a study center

  • Nagoya University Hospital

    Nagoya, Aichi-ken
    Japan

    Site Not Available

  • Nagoya University Hospital

    Nagoya 1856057, Aichi-ken 1865694
    Japan

    Site Not Available

  • Chiba University Hospital

    Chiba, Chiba
    Japan

    Site Not Available

  • Chiba University Hospital

    Chiba 2113015, Chiba 2113014
    Japan

    Site Not Available

  • Kyushu University Hospital

    Fukuoka, Fukuoka
    Japan

    Site Not Available

  • Kyushu University Hospital

    Fukuoka 1863967, Fukuoka 1863958
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Sapporo, Hokkaido
    Japan

    Site Not Available

  • Hokkaido University Hospital

    Sapporo 2128295, Hokkaido 2130037
    Japan

    Site Not Available

  • Kagoshima University Hospital

    Kagoshima, Kagoshima-ken
    Japan

    Site Not Available

  • Kagoshima University Hospital

    Kagoshima 1860827, Kagoshima-ken 1860825
    Japan

    Site Not Available

  • Kyoto University Hospital

    Kyoto, Kyoto
    Japan

    Site Not Available

  • Kyoto University Hospital

    Kyoto 1857910, Kyoto 1857907
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama, Okayama-ken
    Japan

    Site Not Available

  • Okayama University Hospital

    Okayama 1854383, Okayama-ken 1854381
    Japan

    Site Not Available

  • NHO Higashisaitama National Hospital

    Hasuda, Saitama
    Japan

    Site Not Available

  • NHO Higashisaitama National Hospital

    Hasuda 1907123, Saitama 1853226
    Japan

    Site Not Available

  • Institute of Science Tokyo Hospital

    Bunkyō-Ku, Tokyo
    Japan

    Site Not Available

  • The University of Tokyo Hospital

    Bunkyō-Ku, Tokyo
    Japan

    Active - Recruiting

  • National Center Hospital, National Center of Neurology and Psychiatry

    Kodaira, Tokyo
    Japan

    Site Not Available

  • National Center Hospital, National Center of Neurology and Psychiatry

    Kodaira 11611930, Tokyo 1850144
    Japan

    Site Not Available

  • Tottori University Hospital

    Yonago, Tottori
    Japan

    Site Not Available

  • Tottori University Hospital

    Yonago 1848277, Tottori 1849890
    Japan

    Site Not Available

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