YA-101 for people with multiple system atrophy

A Double-Blind, Placebo-Controlled, Multi-Center, Phase II, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of YA-101 in Subjects With Multiple System Atrophy

This Phase 2, double-blind trial tests whether two oral doses of YA-101 are safe, reach therapeutic levels, and may improve symptoms in people with multiple system atrophy compared with placebo.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 75 (estimated)
• Ages: 30 Years and up
• Sex: All
• Sponsor: Dasher Neuroscience Inc. (Industry-sponsored)
• Locations: 9 sites (Los Angeles, California and 8 other locations)
• Trial ID: NCT06848231 on ClinicalTrials.gov

What this trial studies

This is a Phase 2, double-blind, placebo-controlled, multi-center dose-escalation trial comparing two oral doses of YA-101 to placebo in people with MSA. Eligible participants meet current MDS clinical criteria for MSA (MSA-P or MSA-C), can take oral medications, ambulate without assistance, and have adequate cognitive function and organ function. The study will collect safety and tolerability data, pharmacokinetic measurements, and exploratory efficacy outcomes over the dosing period. Randomization and blinding are used to reduce bias while dose escalation monitors tolerability as higher-dose cohorts are enrolled.

Who should consider this trial

Good fit: Adults diagnosed with MSA by current MDS clinical criteria (MSA-P or MSA-C) who can take oral medications, ambulate without assistance, are cognitively intact (MMSE > 24), and meet laboratory safety requirements are ideal candidates.

Not a fit: Patients with severe hepatic or renal impairment, significant cognitive impairment (MMSE ≤ 24), inability to ambulate, positive drug or alcohol screens, pregnancy or lactation, or inability to take oral medication are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, YA-101 could offer a well-tolerated oral option that slows disease progression or improves motor and autonomic symptoms in MSA.

How similar studies have performed: Therapies for MSA have had limited clinical success to date, so YA-101 represents a relatively novel approach with limited prior evidence in this disease.

Eligibility criteria

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.

Where this trial is running

Los Angeles, California and 8 other locations

• UCLA Health — Los Angeles, California, United States (Recruiting)
• Brigham and Women's Hospital — Boston, Massachusetts, United States (Recruiting)
• Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
• Columbia University Irving medical center — New York, New York, United States (Recruiting)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
• Baylor College of Medicine — Houston, Texas, United States (Recruiting)
• Juntendo University Hospital — Tokyo, Japan (Recruiting)
• National Taiwan University Hospital — Taipei, Taiwan (Recruiting)
• National Taiwan University Hospital Cancer Center — Taipei, Taiwan (Not_yet_recruiting)

Study contacts

• Study coordinator: Email contact via Dasher Neuroscience Inc.

Conditions Multiple System Atrophy