Clinical trial NCT06848231: A Phase 2 Study of YA-101 in Patients with Multiple System Atrophy
Official title: 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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Multiple System Atrophy
Lead sponsor: Yoda Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-26
Enrollment: 42
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Number of Participants with Treatment-emergent Adverse Events (TEAEs)
timeFrame: Baseline to Day 56
Secondary outcomes:
measure: Maximum observed concentration (Cmax)
timeFrame: Baseline to Day 28
measure: Area under the concentration-time curve (AUC)
timeFrame: Baseline to Day 28
measure: Time of maximum observed concentration (Tmax)
timeFrame: Baseline to Day 28
measure: Change from baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) over time
timeFrame: Baseline to Day 56
measure: Change from baseline in the 10-meter walking test
timeFrame: Baseline to Day 56
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA Health
state: California
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: New York
country: United States
facility: Columbia University Irving medical center
state: New York
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital Cancer Center
Study officials:
References: