This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
|Full study title
|An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naive Pediatric Subjects With Infantile-onset Pompe Disease Aged 0 to < 18 Years
Male or female subjects who are aged 6 months to < 18 years on Day 1
Subject must have documentation of IOPD genotype
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 whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
Subjects aged ≥ 12 to < 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to < 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to < 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
- Male or female subjects who are aged 0 to
Barry J Byrne, MD, PhDPediatric Cardiologist
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Primary contactBarry Byrne
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