clinical trial · NCT03911505
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Pompe Disease (Late-onset)Cipaglucosidase AlfaMiglustat
brief summary
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
started
Feb 13, 2020
primary completion
Jun 1, 2026
completion
Jun 1, 2026
last updated
Oct 27, 2025
official title
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol