Daily pill takes aim at a rare brain disease that mimics Parkinson's

NCT ID NCT07825402

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Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
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Paused for now. It may or may not start again.
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The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
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Cancelled before anyone took part.

Expanded access (not trials)

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Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
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Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

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First seen Sep 17, 2026 · Last updated Sep 18, 2026 · Updated 1 time

Summary

Researchers are testing an experimental oral drug called ONO-2808 in adults with the Parkinsonian subtype of multiple system atrophy (MSA-P), a rare and progressive brain disorder. The phase 3 trial enrolls about 486 people in the early stages of the disease, within five years of their first symptoms. Participants take ONO-2808 or a placebo pill once daily for up to 48 weeks. Doctors track changes in movement, daily function, quality of life, and brain scans to see whether the drug helps.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ONO-2808, an experimental oral drug
What this could lead to
If ONO-2808 works, it could become the first treatment aimed at the underlying disease process of MSA-P, rather than only easing symptoms.
What could go wrong
MSA-P is a rare, fast-moving disease, and many promising drugs fail in phase 3. ONO-2808 may not slow the disease, and participants may experience side effects that are not yet fully known.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 486 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jan 2027

An estimate. Start dates often move.

Expected to finish

Oct 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

30 to 80 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participants with a diagnosis of clinically established or clinically probable MSA-P according to the 2022 Movement Disorder Society (MDS) criteria for MSA diagnosis. * Participants in the early stages of the disease, defined as a maximum of 5 years since the onset of one of the following symptoms associated with MSA-P: * Parkinsonism * Orthostatic hypotension * Urinary dysautonomia * Participants with an anticipated survival of at least 3 years in the opinion of the Investigator. * Participants who are able to ambulate without the assistance of another person, defined as the ability to take at least 10 steps and then to turn around and walk at least another 10 steps. Use of assistive devices (eg, walker or cane) is allowed. * Ability to swallow oral medication and willingness to adhere to the study drug regimen. * Normal range of laboratory values at screening and baseline, especially liver function tests. * Participants receiving treatment (including chronic medications, and herbal or dietary supplements) for symptoms associated with MSA must be on a stable dosage for at least 60 days prior to randomization based on the judgment of the Investigator. Participants must not initiate new treatment within 60 days prior to randomization. Key Exclusion Criteria: * Participants with the cerebellar subtype of MSA according to the 2022 MDS criteria for MSA diagnosis. * Female participants who are pregnant, planning to become pregnant during the study, or breastfeeding. * Participants with a clinically significant or unstable medical or surgical condition other than MSA-P that, in the opinion of the Investigator, might preclude safe completion of the study or might affect the results of the study (eg, pulmonary, cardiovascular \[including bradyarrhythmia\], macular edema, and significant renal or hepatic dysfunction). * Neurological diseases/disorders other than MSA-P, such as Parkinson's disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, normal pressure hydrocephalus, pharmacological, or postencephalitic parkinsonism. * Any abnormalities, other than MSA, found on the centrally read brain MRI that, in the opinion of the Investigator, may constitute a confounder for the study or preclude safe participation. * Participants with documented liver diseases, cirrhosis, prior drug-induced liver injury (DILI), or ascites or symptoms and signs of encephalopathy due to hepatic dysfunction. Other protocol-defined inclusion and exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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