A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase Alfa in Treatment naïve Patients With Late-onset Pompe Disease
收藏DataCite Commons2024-12-02 更新2026-05-07 收录
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https://search.vivli.org/doiLanding/studies/PR00009818/isLanding
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资源简介:
Primary Objective:
To determine the effect of avalglucosidase alfa treatment on respiratory muscle strength
measured by percent (%) predicted forced vital capacity (FVC) in the upright position, as
compared to alglucosidase alfa.
Secondary Objective:
To determine the safety and effect of avalglucosidase alfa treatment on functional endurance
(6-minute walk test, inspiratory muscle strength (maximum inspiratory pressure), expiratory
muscle strength (maximum expiratory pressure), lower extremity muscle strength (hand-held
dynamometry), motor function (Quick Motor Function Test), and health-related quality of life
(Short Form-12).
提供机构:
Vivli
创建时间:
2024-02-16



