Status and phase
Conditions
Treatments
About
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
22 participants in 1 patient group
Loading...
Central trial contact
For Site; For Patient
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal