New drug YA-101 aims to slow rare brain disease
NCT ID NCT06848231
First seen Jun 24, 2026 · Last updated Aug 20, 2026 · Updated 3 times
Summary
This Phase 2 trial tests the safety and effectiveness of YA-101, an oral drug, in 75 people with multiple system atrophy (MSA), a rare and progressive brain disorder. Participants will receive either YA-101 or a placebo twice daily for several weeks. The study will monitor side effects and measure drug levels in the blood to see if YA-101 can help control the disease.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- YA-101
- What this could lead to
- If it works, this could point toward a treatment that slows or controls multiple system atrophy, a rare and devastating brain disease.
- What could go wrong
- This is an early Phase 2 trial with only 75 participants, so results may not apply to everyone. The drug may not work or could cause side effects.
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
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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About 75 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Mar 2025
- Expected to finish
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Dec 2027
An estimate. End dates often move.
- Lead sponsor
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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
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30 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Able to understand the process of the clinical trial and give informed consent for the participation of the study. 2. Diagnosis of MSA according to MDS clinical criteria (Wenning et al, 2022), including subjects with MSA of either subtype (MSA-P or MSA-C). 3. Males or non-pregnant, non-lactating females with no child-bearing potential, or agree to use 2 forms of contraception. 4. Able to take oral medications. 5. Able to ambulate without the assistance of another person. Exclusion Criteria: 1. Positive urine test for drugs of abuse and/or alcohol test both at screening and Day 1. 2. Evidence of renal impairment or hepatic impairment. 3. Subject with a Mini-Mental State Examination (MMSE) score of 24 or lower. 4. Medical history includes severe systemic diseases such as cardiopulmonary failure, severe liver or kidney disease, and uncontrolled diabetes; significant central nervous system disorders like stroke, encephalitis, and epilepsy and severe head trauma; peptic ulcer in one year prior to screening. 5. Positive results for active viral infections, including human immunodeficiency virus (HIV), hepatitis B, and hepatitis C.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Brigham and Women's Hospital
Boston, Massachusetts, 02115, United States
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Columbia University Irving medical center
New York, New York, 10032, United States
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Juntendo University Hospital
Tokyo, Japan
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Mayo Clinic
Rochester, Minnesota, 55905, United States
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National Taiwan University Hospital
Taipei, 100225, Taiwan
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National Taiwan University Hospital Cancer Center
Taipei, 106, Taiwan
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UCLA Health
Los Angeles, California, 90095, United States
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Vanderbilt University Medical Center
Nashville, Tennessee, 37232, United States
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