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RecruitingNCT07446894MSA-01_P3Updated Mar 4, 2026

MSA-01 in Multiple System Atrophy

A Phase 3 interventional study of MSA-01 and Placebo in Multiple System Atrophy (MSA), sponsored by Tokyo University. Recruiting at 12 sites in Japan. Open to participants aged 30 Years to 79 Years. Per ClinicalTrials.gov, last updated 2026-03-04.

Sponsored by Tokyo University · Phase 3, Interventional, and Treatment

From the registry’s dates

  • Started Feb 2026; still recruiting 9 months later.
Phase
Phase 3
Study type
Interventional
Enrollment
140
Allocation
Randomized
Ages
30 Years to 79 Years
Sex
All
01

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.

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.

02

Conditions studied

  • Multiple System Atrophy (MSA)
03

In context

Multiple System Atrophy

207 studies on the registry are indexed under Multiple System Atrophy; 73 are open to participants now.

This study's planned enrollment of 140 is above the median of 41 across 134 interventional studies indexed under Multiple System Atrophy.

Browse Multiple System Atrophy studies →

Lead sponsor

Tokyo University is the lead sponsor of 34 studies on the registry; 5 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
30 Years to 79 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

At the time of

  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

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 to any component of the 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.
Terms in these criteria, in plain language(3)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
140 participants (estimated)

Study arms

  • Experimental
    MSA-01

    Drug: MSA-01

  • Placebo comparator
    Placebo

    Drug: Placebo

Interventions

  • DrugMSA-01

    Ubiquinol

  • DrugPlacebo

    Placebo

06

What researchers measure

Primary outcomes

  1. Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 score

    Time frame: Baseline to Week 52

Secondary outcomes

  1. Change from baseline to Week 52 in the Barthel Index

    Time frame: Baseline to Week 52

  2. Change from baseline to Week 52 in the Scale for the Assessment and Rating of Ataxia (SARA) score

    Time frame: Baseline to Week 52

  3. Change from baseline to Week 52 in the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 1 score

    Time frame: Baseline to Week 52

Other outcomes

  1. Change from baseline in individual UMSARS Part 2 item scores

    Time frame: Baseline to Week 52

  2. Change from baseline in individual UMSARS Part 1 item scores

    Time frame: Baseline to Week 52

  3. Change from baseline in the scores of the four ADL-related items of UMSARS Part 1

    Time frame: Baseline to Week 52

  4. Change from baseline in the scores of the eight QOL-related items of UMSARS Part 1

    Time frame: Baseline to Week 52

  5. Change from baseline in the EQ-5D-5L index score

    Time frame: Baseline to Week 52

07

Study locations

1 of 12 sites recruiting
08

References and documents

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 ↗

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Feb 2026 (actual). Primary completion is estimated for Mar 2028, 25 months after the start.

How these figures are worked out, and which studies are left out of them.

Registered
Feb 25, 2026
First posted
Mar 3, 2026
StartActual
Feb 9, 2026
Primary completionEst.
Mar 31, 2028
CompletionEst.
Mar 31, 2028
Last update
Mar 4, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Mar 4, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07446894
Lead sponsor
Tokyo University
Collaborators
Nobelpharma
Responsible party
Jun Mitsui (Project Associate Professor, Tokyo University) — Principal investigator
First posted
Mar 3, 2026
Start date
Feb 9, 2026
Primary completion
Mar 31, 2028 (estimated)
Completion
Mar 31, 2028 (estimated)
Last update
Mar 4, 2026

Study contacts

Jun Mitsui
Contact
msa-office@umin.ac.jp
+81-3-5800-9762
Ikue Wada
Contact
msa-office@umin.ac.jp
+81-3-5800-9762

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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