clinical trial · NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Glycogen Storage Disease Type II Infantile OnsetCipaglucosidase alfaMiglustat
brief summary
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.
started
Jul 18, 2023
primary completion
Jul 1, 2027
completion
Jul 1, 2027
last updated
Jan 22, 2026
official title
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
sourced from ClinicalTrials.gov · pharmadog mirrors structured fields, not the full protocol