[Translation] An open-label, multinational, multicenter, intravenous infusion study evaluating the efficacy, safety, pharmacokinetics, and pharmacodynamics of avalglucosidase alfa in untreated pediatric subjects with infantile-onset Pompe disease (IOPD)
主要目的:确定avalglucosidase alfa治疗对≤6月龄IOPD受试者治疗52周后的生存期和不使用有创呼吸机生存期的作用。次要目的:(1)确定avalglucosidase alfa治疗对12月龄和18月龄受试者的生存期和不使用有创呼吸机生存期的作用,以及≤6月龄IOPD患者第52周时LVM-Z评分、AIMS评分、体长、体重和头围Z评分以及尿Hex4的变化;(2)确定avalglucosidase alfa的安全性、耐受性和免疫原性;(3)确定第12周和52周时的PK特征。
[Translation] Main objective: To determine the effect of avalglucosidase alfa treatment on survival after 52 weeks of treatment and survival without invasive ventilator in subjects ≤6 months of age with IOPD. Secondary objectives: (1) To determine the effect of avalglucosidase alfa treatment on survival and survival without invasive ventilator use in subjects aged 12 and 18 months, and LVM- Changes in Z score, AIMS score, body length, body weight and head circumference Z score and urinary Hex4; (2) to determine the safety, tolerability and immunogenicity of avalglucosidase alfa; (3) to determine the 12th and 52nd week PK characteristics.