A Phase 2 Study of YA-101 in Patients 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 is a Phase 2, 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.

Study Overview

• Status: Recruiting
• Conditions: Multiple System Atrophy
• Intervention / Treatment: Drug: Placebo; Drug: YA-101

Detailed Description

The purpose of the study is to evaluate 2 doses of YA-101 compared to placebo in MSA patients, including: 1) safety and tolerability, 2) pharmacokinetics, and 3) potential efficacy of YA-101.

• Study Type: Interventional
• Enrollment (Estimated): 75
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Email contact via Dasher Neuroscience Inc.; Phone Number: 857-468-9328; Email: info@dasherneuroscience.com

Study Locations

• Japan
  • Tokyo, Japan
    • Recruiting
    • Juntendo University Hospital
• Taiwan
  • Taipei, Taiwan, 100225
    • Recruiting
    • National Taiwan University Hospital
    • Contact: Email: robinwu@ntu.edu.tw
  • Taipei, Taiwan, 106
    • Not yet recruiting
    • National Taiwan University Hospital Cancer Center
• United States
  • California
    • Los Angeles, California, United States, 90095
      • Recruiting
      • UCLA Health
      • Contact: Westley Ulit; Phone Number: 310-206-8153
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Brigham and Women's Hospital
      • Contact: Anya Gemos; Phone Number: 510-755-3948; Email: bwhmovementresearch@bwh.harvard.edu
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
  • New York
    • New York, New York, United States, 10032
      • Recruiting
      • Columbia University Irving Medical Center
      • Contact: Nadia Amokrane; Phone Number: 347-882-4315
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University Medical Center
      • Contact: Maeve Curtin; Email: cognitionandmovement@vumc.org
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Baylor College of Medicine
      • Contact: Rory Mahabir; Phone Number: 713-798-5989; Email: rory.mahabir@bcm.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo taken BID
Experimental: YA-101	Drug: YA-101; Drug: YA-101 • YA-101 taken BID

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and severity of Adverse Events (AEs)	Incidence and severity of Adverse Events (AEs).	Baseline to Day 112

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum observed concentration (Cmax)	To describe the pharmacokinetics parameter (Cmax) of YA-101 using sparse PK sampling.	Baseline to Day 84
Area under the concentration-time curve (AUC)	To describe the pharmacokinetics parameter (AUC) of YA-101 using sparse PK sampling.	Baseline to Day 84
Time of maximum observed concentration (Tmax)	To describe the pharmacokinetics parameter (Tmax) of YA-101 using sparse PK sampling.	Baseline to Day 84
Change from baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) over time	The unified multiple system atrophy rating scale (UMSARS) is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability, UMSARS-II (14 items) assesses motor impairment based on a clinical examination, UMSARS-III records blood pressure and heart rate in the supine and standing positions, and UMSARS-IV (1 item) rates chore-based disability. Higher scores on the UMSARS indicate greater disability.	Baseline to Day 112
Change from baseline in the 10-meter walking test		Baseline to Day 112

Collaborators and Investigators

• Sponsor: Dasher Neuroscience Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 3, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: February 17, 2025
• First Submitted That Met QC Criteria: February 21, 2025
• First Posted (Actual): February 26, 2025

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: June 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Basal Ganglia Diseases; Primary Dysautonomias; Autonomic Nervous System Diseases; Multiple System Atrophy; Substandard Drugs; Pharmaceutical Preparations; Counterfeit Drugs
• Other Study ID Numbers: YA-101-201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No