Phase 4 Study of Avalglucosidase Alfa Treatment in Chinese Children Under 18 With Infantile-onset Pompe Disease

What this Trial Is About

This research aims to evaluate the safety and effects of avalglucosidase alfa given by intravenous infusion in male and female Chinese children with infantile-onset Pompe disease IOPD. Participants may be new to treatment or have been treated before with enzyme replacement therapy ERT. This is a Phase 4, open-label, single-arm study lasting about 64 weeks, sponsored by Genzyme, a Sanofi Company. Avalglucosidase alfa is given as an IV infusion at a dose of 20 mg per kilogram of body weight every other week. The study includes a screening period of up to 8 weeks, followed by a 52-week treatment period, and then a 4-week follow-up period, which may be shortened to 2 weeks if participants join another study or begin commercial ERT. Participants will have 30 visits in total, including 29 site visits and one follow-up phone call. During the study, participants will be monitored for adverse events, lab test abnormalities, vital signs, and heart function via ECG from baseline to Week 56. Researchers will also assess survival free of invasive ventilation, heart muscle thickness, body growth, walking distance in a six-minute walk test, motor function, and motor milestone achievements at Week 52. Safety and efficacy evaluations will help understand the treatments impact over the course of the study.

China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD