A Pre-Gene Therapy Study of Early Parkinson's or Multiple System Atrophy Progression by Longitudinal Clinical and Biomarker Assessments
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- AskBio Inc
- 入组人数
- 73
- 试验地点
- 22
- 主要终点
- Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
研究概览
简要总结
The objective of this study is to describe disease progression in study participants diagnosed with early Parkinson's Disease or Multiple System Atrophy - Parkinsonian Type up to 18 months as delineated by clinical and biochemical parameters.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 35 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of Idiopathic PD
- •Diagnosis of MSA-P
- •One to six years from clinical diagnosis of PD
- •Less than 4 years from clinical diagnosis of MSA with expected survival >3 years
- •Stable medication regimen
- •Ability to walk with or without an assistive device
排除标准
- •Movement disorder due to known cerebrovascular disease, brain tumor, trauma, exposure to parkinsonian-linked toxicants or other neurological diseases
- •Presence of dementia, psychosis, substance abuse or poorly controlled depression
- •Prior brain surgery (i.e. deep brain stimulation) or other brain imaging abnormalities
- •History of cancer or poorly controlled medical conditions
- •Receiving an investigational drug
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
结局指标
主要结局
Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
时间窗: 18 months
Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores.
MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS)
时间窗: 18 Months
Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) and compared to placebo. UMSARS total scores range from 0-104 points with higher scores indicating greater severity of impairment.
次要结局
- Change in the quality of life as measured by Parkinson's Disease Questionnaire (PDQ-39)(18 months)
- Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL)(18 months)