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

Официальное название

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

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат YA-101
  Drug: YA-101 • YA-101 taken BID
• Препарат Placebo
  Placebo taken BID

Первичные конечные точки

• Incidence and severity of Adverse Events (AEs) [Срок оценки: Baseline to Day 112]

Вторичные конечные точки (5)

• Maximum observed concentration (Cmax) [Срок оценки: Baseline to Day 84]
• Area under the concentration-time curve (AUC) [Срок оценки: Baseline to Day 84]
• Time of maximum observed concentration (Tmax) [Срок оценки: Baseline to Day 84]
• Change from baseline in Unified Multiple System Atrophy Rating Scale (UMSARS) over time [Срок оценки: Baseline to Day 112]
• Change from baseline in the 10-meter walking test [Срок оценки: Baseline to Day 112]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Последовательный дизайн

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

США · 6 центров

• UCLA Health — Los Angeles
• Brigham and Women's Hospital — Boston
• Mayo Clinic — Rochester
• Columbia University Irving medical center — New York
• Vanderbilt University Medical Center — Nashville
• Baylor College of Medicine — Houston

Тайвань · 2 центра

• National Taiwan University Hospital — Taipei
• National Taiwan University Hospital Cancer Center — Taipei

Япония · 1 центр

• Juntendo University Hospital — Tokyo

Идентификаторы

NCT: NCT06848231 · YA-101-201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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