China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
Trial Parameters
Brief Summary
This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include: * The study duration: total study duration is approximately 64 weeks. * Screening period of up to 8 weeks * Treatment period of 52 weeks * Follow-up period of 4 weeks. (if the participant enrolls in another study or receives commercially available ERT, the follow-up period may be reduced from 4 to 2 weeks) * The number of visits will be 30, including 29 site visits and 1 phone call follow-up visit.
Eligibility Criteria
Inclusion Criteria: * Participant must be \<18 years of age, at the time of signing the informed consent. * Participants have documented onset of Pompe disease symptoms before 12 months of age (corrected for gestation if born before 40 weeks); and diagnosis of IOPD is confirmed by GAA enzyme deficiency from any tissue source and GAA gene pathogenic mutations. * Participants must have documented cardiomyopathy at the time of diagnosis. * Contraceptive use should be consistent with local regulations Participant's parent/legally authorized representative (LAR) must be capable of giving signed informed consent. Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply: * Participants with major congenital abnormality that, in the opinion of the Investigator, would preclude participation in the study or potentially decrease survival. * Participants with clinically significant organic disease (with the exception of symptoms relating to Pompe disease)