Multiple System Atrophy
Conditions
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.
Interventions
DRUGYA-101
Drug: YA-101 • YA-101 taken BID
DRUGPlacebo
Placebo taken BID
Sponsors
Dasher Neuroscience Inc.
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
30 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of Adverse Events (AEs) | Baseline to Day 112 | Incidence and severity of Adverse Events (AEs). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maximum observed concentration (Cmax) | Baseline to Day 84 | To describe the pharmacokinetics parameter (Cmax) of YA-101 using sparse PK sampling. |
| Area under the concentration-time curve (AUC) | Baseline to Day 84 | To describe the pharmacokinetics parameter (AUC) of YA-101 using sparse PK sampling. |
| Time of maximum observed concentration (Tmax) | Baseline to Day 84 | 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 112 | 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 112 | — |
Countries
Japan, Taiwan, United States
Outcome results
None listed