MSA-01 in Multiple System Atrophy
MSA-01_P3
A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Study of MSA-01 in Patients With Multiple System Atrophy
2 other identifiers
interventional
140
1 country
12
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Feb 2026
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
February 9, 2026
CompletedFirst Submitted
Initial submission to the registry
February 25, 2026
CompletedFirst Posted
Study publicly available on registry
March 3, 2026
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 31, 2028
March 4, 2026
March 1, 2026
2.1 years
February 25, 2026
March 2, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
Baseline to Week 52
Secondary Outcomes (3)
Change from baseline to Week 52 in the Barthel Index
Baseline to Week 52
Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score
Baseline to Week 52
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score
Baseline to Week 52
Other Outcomes (5)
Change from baseline in individual UMSARS Part 2 item scores
Baseline to Week 52
Change from baseline in individual UMSARS Part 1 item scores
Baseline to Week 52
Change from baseline in the scores of the four ADL-related items of UMSARS Part 1
Baseline to Week 52
- +2 more other outcomes
Study Arms (2)
MSA-01
EXPERIMENTALPlacebo
PLACEBO COMPARATORInterventions
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Tokyo Universitylead
- Nobelpharmacollaborator
Study Sites (12)
Nagoya University Hospital
Nagoya, Aichi-ken, Japan
Chiba University Hospital
Chiba, Chiba, Japan
Kyushu University Hospital
Fukuoka, Fukuoka, Japan
Hokkaido University Hospital
Sapporo, Hokkaido, Japan
Kagoshima University Hospital
Kagoshima, Kagoshima-ken, Japan
Kyoto University Hospital
Kyoto, Kyoto, Japan
Okayama University Hospital
Okayama, Okayama-ken, Japan
NHO Higashisaitama National Hospital
Hasuda, Saitama, Japan
Institute of Science Tokyo Hospital
Bunkyō-Ku, Tokyo, Japan
The University of Tokyo Hospital
Bunkyō-Ku, Tokyo, Japan
National Center Hospital, National Center of Neurology and Psychiatry
Kodaira, Tokyo, Japan
Tottori University Hospital
Yonago, Tottori, Japan
Related Publications (1)
Mitsui J, Matsukawa T, Uemura Y, Kawahara T, Chikada A, Porto KJL, Naruse H, Tanaka M, Ishiura H, Toda T, Kuzuyama H, Hirano M, Wada I, Ga T, Moritoyo T, Takahashi Y, Mizusawa H, Ishikawa K, Yokota T, Kuwabara S, Sawamoto N, Takahashi R, Abe K, Ishihara T, Onodera O, Matsuse D, Yamasaki R, Kira JI, Katsuno M, Hanajima R, Ogata K, Takashima H, Matsushima M, Yabe I, Sasaki H, Tsuji S. High-dose ubiquinol supplementation in multiple-system atrophy: a multicentre, randomised, double-blinded, placebo-controlled phase 2 trial. EClinicalMedicine. 2023 Apr 14;59:101920. doi: 10.1016/j.eclinm.2023.101920. eCollection 2023 May.
PMID: 37256098BACKGROUND
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Project Associate Professor
Study Record Dates
First Submitted
February 25, 2026
First Posted
March 3, 2026
Study Start
February 9, 2026
Primary Completion (Estimated)
March 31, 2028
Study Completion (Estimated)
March 31, 2028
Last Updated
March 4, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share