Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 300
- 试验地点
- 1
- 主要终点
- Severity of muscle strength decline over time
研究概览
简要总结
The goal of this open prospective multi-disciplinary observational study of patients with OPMD at various stages of clinical manifestations is to explore the natural history of the disease in the Israeli population.
详细描述
The the following procedures will be performed by a multidisciplinary team:
Neurological and neurocognitive examination; Clinical assessment of of swallowing and severity of dysphagia; Clinical assessment of eyelid ptosis severity; Clinical assessment of respiratory muscles function; Clinical assessment of oral health
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD
排除标准
- •Age <18 years and Pregnancy
结局指标
主要结局
Severity of muscle strength decline over time
时间窗: 1 year
10-meter walk test
Penetration/Aspiration Scale
时间窗: 1 year
Penetration/Aspiration Scale (PAS)
Upper Margin Reflex Distance
时间窗: 1 year
Upper Margin Reflex Distance
Lower Margin Reflex Distance
时间窗: 1 year
Lower Margin Reflex Distance
次要结局
- Maximum Expiratory Pressure(2 years)
- Cough Peak Flow(2 years)
- Oral health(2 years)
- Dysphagia severity(1 year)
- Functional Oral Intake Scale(1 year)
- Swallowing Disturbance(1 year)