Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen: Retro NusiMFM
试验速览
- 阶段
- 不适用
- 入组人数
- 40
- 试验地点
- 8
- 主要终点
- MFM-20 responsiveness
研究概览
简要总结
The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items validated for patients between 2 and 6 years old (MFM-20).
In order to show the possible use of MFM-20 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-20 and the validity of the scale in this population.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 2 Years 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed
- •Aged 2 to 6 years old
- •Treated by Nusinersen for at least two months
- •With at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months
- •With parental assent
排除标准
- •- Patients with associated cognitive impairment making impossible evaluation of motor function
- •Patients participating to a clinical study with a potential effect on their motor function.
结局指标
主要结局
MFM-20 responsiveness
时间窗: At least 6 Months after the first MFM-20 assessment, and at maximum 24 months
The MFM-20 sensitivity to change indexes considered will be the effect amplitude and the Standardized Response Mean (SRM).
次要结局
未报告次要终点