A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy

Phase 2Active Not RecruitingNCT06848231

Dasher Neuroscience Inc.75 enrolled

Overview

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.

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

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Multiple System Atrophy

Interventions

YA-101DRUG

Drug: YA-101 • YA-101 taken BID

PlaceboDRUG

Placebo taken BID

Eligibility

Sex: ALLMin age: 30 Years

Medical Language ↔ Plain English

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.

Locations (9)

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

Outcomes

Primary Outcomes

Incidence and severity of Adverse Events (AEs)

Incidence and severity of Adverse Events (AEs).

Time frame: Baseline to Day 112

Secondary Outcomes

Maximum observed concentration (Cmax)

To describe the pharmacokinetics parameter (Cmax) of YA-101 using sparse PK sampling.

Time frame: Baseline to Day 84

Area under the concentration-time curve (AUC)

To describe the pharmacokinetics parameter (AUC) of YA-101 using sparse PK sampling.

Time frame: Baseline to Day 84

Time of maximum observed concentration (Tmax)

To describe the pharmacokinetics parameter (Tmax) of YA-101 using sparse PK sampling.

Time frame: 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.

Time frame: Baseline to Day 112

Change from baseline in the 10-meter walking test

Time frame: Baseline to Day 112

Data from ClinicalTrials.gov

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