NCT04808505

A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 36 (estimated)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 17 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Glycogen Storage Disease Type II Infantile Onset

Interventions

Type	Name	Description
BIOLOGICAL	Cipaglucosidase alfa	Sterile lyophilized powder intravenous (IV) infusion
DRUG	Miglustat	65 mg oral capsules

Timeline

• Start date: 2023-07-18
• Primary completion: 2027-07-01
• Completion: 2027-07-01
• First posted: 2021-03-22
• Last updated: 2026-01-22

Locations

14 sites across 6 countries: United States, Germany, Italy, Netherlands, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study