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.
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
Subject requires invasive ventilation (eg, tracheostomy)
Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
Subject has prior history of illness or condition known to affect motor function
Parallel
Cipaglucosidase alfa
Biological/VaccineSterile lyophilized powder intravenous (IV) infusion
Miglustat
Drug65 mg oral capsules
Proportion of subjects with infusion-associated reactions (IARs)
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