MSA-01 in Multiple System Atrophy (MSA-01_P3)

March 2, 2026 updated by: Jun Mitsui, Tokyo University

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

140

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Japan
    • Aichi-ken
      • Nagoya, Aichi-ken, Japan
        • Not yet recruiting
        • Nagoya University Hospital
        • Contact:
    • Chiba
      • Chiba, Chiba, Japan
        • Not yet recruiting
        • Chiba University Hospital
        • Contact:
    • Fukuoka
      • Fukuoka, Fukuoka, Japan
        • Not yet recruiting
        • Kyushu University Hospital
        • Contact:
    • Hokkaido
      • Sapporo, Hokkaido, Japan
        • Not yet recruiting
        • Hokkaido University Hospital
        • Contact:
    • Kagoshima-ken
      • Kagoshima, Kagoshima-ken, Japan
        • Not yet recruiting
        • Kagoshima University Hospital
        • Contact:
    • Kyoto
      • Kyoto, Kyoto, Japan
        • Not yet recruiting
        • Kyoto University Hospital
        • Contact:
    • Okayama-ken
      • Okayama, Okayama-ken, Japan
        • Not yet recruiting
        • Okayama University Hospital
        • Contact:
    • Saitama
      • Hasuda, Saitama, Japan
        • Not yet recruiting
        • NHO Higashisaitama National Hospital
        • Contact:
    • Tokyo
      • Bunkyō-Ku, Tokyo, Japan
      • Bunkyō-Ku, Tokyo, Japan
        • Not yet recruiting
        • Institute of Science Tokyo Hospital
        • Contact:
      • Kodaira, Tokyo, Japan
        • Not yet recruiting
        • National Center Hospital, National Center of Neurology and Psychiatry
        • Contact:
    • Tottori
      • Yonago, Tottori, Japan
        • Not yet recruiting
        • Tottori University Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Placebo
Experimental: MSA-01
Drug: MSA-01
Ubiquinol

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
Time Frame: Baseline to Week 52
Baseline to Week 52

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline to Week 52 in the Barthel Index
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score
Time Frame: Baseline to Week 52
Baseline to Week 52

Other Outcome Measures

Outcome Measure
Time Frame
Change from baseline in individual UMSARS Part 2 item scores
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline in individual UMSARS Part 1 item scores
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline in the scores of the four ADL-related items of UMSARS Part 1
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline in the scores of the eight QOL-related items of UMSARS Part 1
Time Frame: Baseline to Week 52
Baseline to Week 52
Change from baseline in the EQ-5D-5L index score
Time Frame: Baseline to Week 52
Baseline to Week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Tokyo University

Collaborators

Nobelpharma

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 9, 2026

Primary Completion (Estimated)

March 31, 2028

Study Completion (Estimated)

March 31, 2028

Study Registration Dates

First Submitted

February 25, 2026

First Submitted That Met QC Criteria

February 25, 2026

First Posted (Actual)

March 3, 2026

Study Record Updates

Last Update Posted (Actual)

March 4, 2026

Last Update Submitted That Met QC Criteria

March 2, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2025024-11DX
  • jRCT2031250696 (Registry Identifier: Japan Registry of Clinical Trials)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Multiple System Atrophy (MSA)

Clinical Trials on Placebo

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Brazil

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe