NCT02596191进行中（未招募）不适用
Tools for Therapeutic Evaluation in Charcot-Marie-Tooth Disease Type 1A: Outcome Measures and Biomarkers
适应症
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 80
- 试验地点
- 4
- 主要终点
- Change of functional scores at
研究概览
简要总结
This is a 2-year follow-up study of a cohort of 60 CMT1A patients. The objective is to identify markers allowing to better understand the phenotypic variability observed on patients with CMT1A, to identify predictive markers of the disease's progression and to provide validated measurement tools that can be used as outcome measures in future clinical trials.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Patients with CMT 1A disease
- •Diagnosis of CMT 1A confirmed by genotyping (duplication of the 17p11.2 region)
排除标准
- •Patients suffering from co-morbidity at the origin of peripheral neuropathy (diabetes, hypothyroidism, renal insufficiency, drugs...) or muscle, articular, rheumatological disease
- •With HIV or cancer
- •With a significant progressive disease in the previous month
- •With a contra-indication for MRI
- •With a dislocation, fracture, or recent surgery (less than 6 months before inclusion)
- •with alcohol or psychoactive substances abuse
- •Treated by an anti-inflammatory drug over the past four weeks
- •Pregnant or breastfeeding women
- •Homeless patients
结局指标
主要结局
Change of functional scores at
时间窗: 3 months, 12 months and 24 months
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (ONLS)
Change of functional scores
时间窗: 3 months, 12 months and 24 months
several functional scores to evaluate the severity and the progression of the disease will be performed and compared (CMTNDS)
次要结局
- Walkin test(12 months and 24 months)
研究者
研究点 (4)
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