2022-501624-14-00招募中2 期
WALKALS : A pilot phase II study to evaluate the effect of salbutamol on walking capacity in ambulatory ALS patients.
Assistance Publique Hopitaux De Paris, Assistance Publique Hopitaux De Paris1 个研究点 分布在 1 个国家目标入组 36 人开始时间: 2023年5月5日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 36
- 试验地点
- 1
- 主要终点
- Two primary endpoints will be used: change from baseline of the 6 minutes walking test distance (6MWT) at M3 (2mg TID) and at M6 (4mg TID) with type I error controlled through Bonferroni correction.
研究概览
简要总结
To test the efficacy of salbutamol on walking capacity in ALS patients.
入排标准
- 年龄范围
- 18 years 至 65+ years（18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Subjects who meet the revised El Escorial criteria for probable or definite sporadic ALS - Adult patients between 18 and 75 years of age - Patients who are ambulatory and able to perform the 6MWT and quantitative muscle testing at screening (ALSFRS-R-walking = 3) - Patients able and willing to travel to the site, and, in the investigator’s opinion, who are likely to attend visits for at least 6 months - Patients who signed written informed consent - Stable dose of riluzole for a minimum of 14 day4 weeks prior to baseline or has not taken it for 14 days4 weeks prior to baseline - Affiliation to French social security system excluding AME - For child-bearing aged women, efficient contraception - Forced vital capacity (fVC) in a sitting position > 70 %
排除标准
- •Patients with significant spasticity of the lower limbs interfering with walking capacity (Ashworth scale score 2) - Patients with fronto-temporal dementia associated with ALS - Patients presenting respiratory insufficiency causing dyspnea during walking - Patients taking drugs that could interfere with NMJ function (anticholinesterase …) or muscle function (steroids, statins…)- Patients taking any forbidden drugs (see list in annex) - Hypersensitivity to salbutamol or to excipients of the drug and placebo - Known contraindication for the studied drug such as ischemic cardiomyopathy or risk of ischemic cardiomyopathy - Pregnancy or women seeking a pregnancy during the study or breastfeeding - Patients under curatorship or guardianship - Participation in another interventional trial up to 3 months before inclusion - Patients having any relevant concomitant disease considered at risk of interfering with study procedures in the opinion of the investigator - Patients not affiliated to the French social security system
结局指标
主要结局
Two primary endpoints will be used: change from baseline of the 6 minutes walking test distance (6MWT) at M3 (2mg TID) and at M6 (4mg TID) with type I error controlled through Bonferroni correction.
Two primary endpoints will be used: change from baseline of the 6 minutes walking test distance (6MWT) at M3 (2mg TID) and at M6 (4mg TID) with type I error controlled through Bonferroni correction.
次要结局
未报告次要终点
研究者
Dr Giorgia QUERIN
Scientific
Assistance Publique Hopitaux De Paris
研究点 (1)
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