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Last Updated: 01/07/2026
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2 clinical trials found
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
Who is this study for: Pediatric patients with infantile-onset Pompe disease aged 0 to < 18 years
Enrollment Status: Recruiting
Publish Date: November 17, 2025
Intervention Type: Drug, Biological
Study Drugs: Cipaglucosidase alfa, Miglustat
Study Phase: Phase 3
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.
A Global Prospective Observational Registry of Patients With Pompe Disease
Enrollment Status: Recruiting
Publish Date: November 17, 2025
Intervention Type: Other, Biological, Drug
Summary: This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe diseas...
Showing 1-2 of 2
Last Updated: 01/07/2026