An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
• Cohort 1:
• Male or female subjects who are aged 6 mo to < 18 years on Day 1
• Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject must have received ERT for at least 6 months immediately before enrollment. For subjects on a modified dose and regimen, at least 3 months prior to enrollment
• Subjects must have experienced a clinical decline on their current approved rhGAA dose and frequency
• Cohort 2:
• Male or female subjects who are aged 0 to <12 month at Day 1
• Prior to initiation of study drug subject must have documentation of IOPD genotype, CRIM status, and no or low measurable GAA enzyme activity
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject is ERT-naive