Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3.
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 4
- 试验地点
- 1
- 主要终点
- Change from Baseline in the distance walked at 6-minute walk test at 6 months
研究概览
简要总结
The purpose of this study is to evaluate safety and efficacy of anti-cholinesterase therapy on the motor function in SMA type 3 patients with impaired neuromuscular junction (NMJ).
详细描述
Spinal muscular atrophy (SMA) is the second neuromuscular disease meet in children. SMA is a genetically transmitted disease inducing muscular weakness predominating on shoulders and hips. Currently, there is no effective therapy to slow the progression of the disease. SMA is due to a neuron motor attempt of the spinal cord and recently it has been demonstrated a neuromuscular junction (NMJ) involvement, according to recent studies.
EMOTAS study aim to understand if NMJ abnormalities could have an impact on motor performance and fatigue in SMA type 3 ambulatory patients by electromyogram and to improve by non-invasive therapy quality of life of patients.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Factorial
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Spinal muscular atrophy type 3, genetically confirmed
- •Age higher than 6 years old
- •Ambulatory patient
- •Informed consent signed
- •More than 100 meters of walking at 6-minute walk test at screening
- •Value at screening and baseline in a range of 20% of the highest value at 6-minute walk test
排除标准
- •Patient who had surgical intervention or suffer from a recent traumatism (less than 6 months)
- •Associated pathology such as endocrinopathy, infectious disease, allergy, myopathy, chronic or acute inflammatory pathology, during 3 weeks preceding the inclusion.
- •Other therapeutics than food supplements or those frequently prescribed in spinal muscular atrophy or its complications
- •Non tolerance of electromyography
- •Limited collaboration due to trouble in information comprehension
- •Pathology inducing contra-indication for pyridostigmine treatment (allergy at molecule, asthma, Parkinson disease, mechanic obstruction of urinary or digestive tracts)
研究组 & 干预措施
significant decrement
Patients with significant decrement at electromyogram will be treated by pyridostigmine bromide 60mg 3 times a day for patients older than 18 and 1.5mg/kg 3 times a day for children less than 40kg
干预措施: Pyridostigmine Bromide (Drug)
结局指标
主要结局
Change from Baseline in the distance walked at 6-minute walk test at 6 months
时间窗: 6 months
次要结局
- Change from baseline of decrement at 6 months(6 months)
- Change from baseline of Moviplate values at 6 months(6 months)
- Change from baseline of the ratio at 6 minutes walk test at 6 months(6 months)
- Change from baseline of MFM-D1(6 months)
研究者
Dr. Stéphanie Delstanche
Neurologist
Centre Hospitalier Régional de la Citadelle