Multiple System Atrophy (MSA)Phase 3MSA-01_P3NCT07446894
MSA-01 in Multiple System Atrophy
A Phase 3 study testing MSA-01 in people with Multiple System Atrophy (MSA), run by Tokyo University at 12 sites in Japan.
Trial 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… Read more about this study
- Who is it for?
- Ages 30 to 79 years
- Conditions the study is for
- Multiple System Atrophy (MSA)
- What is being tested?
- MSA-01
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 140 participants
- Where
- 12 sitesJapan
- Study dates
- 9 Feb 2026to 31 Mar 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 30 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
12 sites in 1 country. Contact the study team or a site near you to ask about taking part.
12 sites in 1 country
Japan12 sites, 12 recruiting
- Not yet recruiting
Institute of Science Tokyo Hospital
Bunkyō-Ku, Tokyo
Kinya Ishikawa, +81-3-5800-9762, msa-office@umin.ac.jp
- Recruiting
The University of Tokyo Hospital
Bunkyō-Ku, Tokyo
Jun Mitsui, +81-3-5800-9762, msa-office@umin.ac.jp
Ikue Wada, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Chiba University Hospital
Chiba, Chiba
Atsuhiko Sugiyama, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Kyushu University Hospital
Fukuoka, Fukuoka
Dai Matsuse, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
NHO Higashisaitama National Hospital
Hasuda, Saitama
Katsuhisa Ogata, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Kagoshima University Hospital
Kagoshima, Kagoshima-ken
Yujiro Higuchi, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
National Center Hospital, National Center of Neurology and Psychiatry
Kodaira, Tokyo
Yuji Takahashi, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Kyoto University Hospital
Kyoto, Kyoto
Hodaka Yamakado, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Nagoya University Hospital
Nagoya, Aichi-ken
Masahisa Katsuno, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Okayama University Hospital
Okayama, Okayama-ken
Hiroyuki Ishiura, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Hokkaido University Hospital
Sapporo, Hokkaido
Ichiro Yabe, +81-3-5800-9762, msa-office@umin.ac.jp
- Not yet recruiting
Tottori University Hospital
Yonago, Tottori
Ritsuko Hanajima, +81-3-5800-9762, msa-office@umin.ac.jp
About this study
Official title: 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.
Read the 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)
- Being tested
- MSA-01, Placebo
- Medical subject headings
- Multiple System Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSA-01Drug
Ubiquinol
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| MSA-01 | Experimental | Not described Receives: Drug: MSA-01 |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
Measured: Baseline to Week 52
Secondary outcomes (3)
Change from baseline to Week 52 in the Barthel Index
Measured: Baseline to Week 52
Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score
Measured: Baseline to Week 52
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score
Measured: Baseline to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2026
- Study start9 Feb 2026
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Multiple System Atrophy (MSA)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 140 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tokyo University
- Sponsor type
- University, hospital or other
- Collaborators
- Nobelpharma
- Responsible party
- Principal investigator, Jun Mitsui, Tokyo University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07446894
- Sponsor's protocol ID
- 2025024-11DX
- Other IDs
- jRCT2031250696
- Acronym
- MSA-01_P3
- Status last verified
- Mar 2026
- First submitted
- 25 Feb 2026
- Last updated
- 4 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 37256098
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Randomized, Double-blind, Placebo-controlled Phase III Study of MSA-01 in Patients With Multiple System Atrophy
- ClinicalTrials.gov ID
- NCT07446894
- Acronym / sponsor ID
- MSA-01_P3 / 2025024-11DX
- Conditions
- Multiple System Atrophy (MSA)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 140 participants (estimated)
- Lead sponsor
- Tokyo UniversityUniversity, hospital or other
- Collaborators
- Nobelpharma
- Study start
- 9 Feb 2026 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 31 Mar 2028 (estimated)
- First posted
- 3 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| MSA-01 | Experimental | Drug: MSA-01 |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 30 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSA-01Ubiquinol | Drug | MSA-01 |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score
Time frame: Baseline to Week 52
3 secondary outcomes
Change from baseline to Week 52 in the Barthel Index
Time frame: 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
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score
Time frame: Baseline to Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2026.
- Background: PubMed 37256098Mitsui 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing MSA-01 in people with Multiple System Atrophy (MSA), run by Tokyo University at 12 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jun Mitsui (+81-3-5800-9762, msa-office@umin.ac.jp), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score (time frame: Baseline to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07446894, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2026.