ClinicalTrials.gov record NCT07446894

MSA-01 in Multiple System Atrophy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

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

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.
Age30 years old to 79 years old
SexAll
LocationJapan
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This randomized, double-blind, placebo-controlled Phase III study compares oral MSA-01 with placebo in people with Multiple System Atrophy (MSA). About 140 participants are randomized 1:1 to receive MSA-01 or placebo for 52 weeks, with regular clinic visits and diaries to record medication use and side effects. The main measure is change in UMSARS Part 2 score from baseline to Week 52.

What it is studying. Whether MSA-01 (ubiquinol) slows motor progression in MSA as measured by the Unified Multiple System Atrophy Rating Scale (UMSARS) Part 2 over 52 weeks, and to assess the safety profile of MSA-01 compared with placebo.

What the researchers hope to learn. Scientific goals: determine whether MSA-01 slows clinical progression of MSA compared with placebo and evaluate its safety. Secondary outcomes include changes in Barthel Index, SARA score, and UMSARS Part 1.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • MSA-01
  • Placebo

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

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.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are randomly assigned to take oral MSA-01 (ubiquinol) or placebo for 52 weeks. They will attend regular clinic visits for safety and efficacy assessments (including clinical rating scales) and will record medication use and any side effects in a diary. Participants must stop any CoQ10 supplements before starting the study drug. The study team will monitor safety throughout the 12‑month study period.

How the study is structured. Participants are randomly assigned to one of two groups and receive either oral MSA-01 or matching placebo for 52 weeks. The trial is double-blind (neither participants, treating clinicians, study staff, nor outcome assessors know who receives the drug), uses a parallel-group design, and plans about 140 people randomized 1:1 with regular clinic visits for safety and efficacy checks and a medication/side-effect diary.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • Some participants receive a placebo. How is that decided, and what happens for placebo-group participants when the study ends?
  • The listed sites are in Japan. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on multiple system atrophy

Clinical trials76 trials listed for multiple system atrophyEvery trial the registry lists for multiple system atrophy, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in multiple system atrophy researchLatest monthly update: September 2026. Includes: Tube feeding. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Feb 9, 2026
Primary completion
Mar 31, 2028 (estimated)
Study completion
Mar 31, 2028 (estimated)
Target enrollment
140 participants (estimated)
Last updated on registry
Mar 4, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Tokyo University
Principal investigator
Jun Mitsui, Project Associate Professor
Tokyo University
Collaborators
  • Nobelpharma

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (12)

Terms used on this page · explanations by MedicalUpdates.ai

randomly assigned
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
placebo
A treatment that looks like the study treatment but contains no active ingredient. Some participants receive it instead of the study treatment, usually without knowing which they got.
informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
study drug
A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
phase II
A study that tests whether a treatment has the intended effect and continues to check safety, usually in a larger group than phase 1.
double-blind
Two types of people involved in the study do not know which intervention a participant is receiving. The ClinicalTrials.gov record specifies who is masked, for example participants, care providers, investigators or outcome assessors.
placebo-controlled
A study design in which one group receives a placebo so that the study treatment's effects can be compared with no active treatment.
1:1
How many participants go to each group. In a 2:1 ratio, two are assigned to the first group for every one assigned to the second.
primary endpoint
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
baseline
Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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