A Phase 2 Study of YA-101 in Patients with Multiple System Atrophy
- Registration Number
- NCT06848231
- Lead Sponsor
- Yoda Therapeutics Inc.
- Brief Summary
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.
- 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.
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 42
- Able to understand the process of the clinical trial and give informed consent for the participation of the study.
- Diagnosis of MSA according to MDS clinical criteria (Wenning et al, 2022), including subjects with MSA of either subtype (MSA-P or MSA-C).
- Males or non-pregnant, non-lactating females with no child-bearing potential, or agree to use 2 forms of contraception.
- Able to take oral medications.
- Able to ambulate without the assistance of another person.
- Positive urine test for drugs of abuse and/or alcohol test both at screening and Day 1.
- Evidence of renal impairment or hepatic impairment.
- Subject with a Mini-Mental State Examination (MMSE) score of 24 or lower.
- 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.
- Positive results for active viral infections, including human immunodeficiency virus (HIV), hepatitis B, and hepatitis C.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description YA-101 YA-101 - Placebo Placebo -
- Primary Outcome Measures
Name Time Method Number of Participants with Treatment-emergent Adverse Events (TEAEs) Baseline to Day 56 Number and percentage of participants with Treatment-emergent Adverse Events (TEAEs).
- Secondary Outcome Measures
Name Time Method Maximum observed concentration (Cmax) Baseline to Day 28 To describe the pharmacokinetics parameter (Cmax) of YA-101 using sparse PK sampling.
Area under the concentration-time curve (AUC) Baseline to Day 28 To describe the pharmacokinetics parameter (AUC) of YA-101 using sparse PK sampling.
Time of maximum observed concentration (Tmax) Baseline to Day 28 To describe the pharmacokinetics parameter (Tmax) of YA-101 using sparse PK sampling.
Change from baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) over time Baseline to Day 56 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.
Change from baseline in the 10-meter walking test Baseline to Day 56
Related Research Topics
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Trial Locations
- Locations (8)
UCLA Health
🇺🇸Los Angeles, California, United States
Brigham and Women's Hospital
🇺🇸Boston, Massachusetts, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Columbia University Irving medical center
🇺🇸New York, New York, United States
Vanderbilt University Medical Center
🇺🇸Nashville, Tennessee, United States
Baylor College of Medicine
🇺🇸Houston, Texas, United States
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
National Taiwan University Hospital Cancer Center
🇨🇳Taipei, Taiwan