NCT03911505

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 0 Years – 17 Years
• Healthy volunteers: Not accepted

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

Conditions

• Pompe Disease (Late-onset)

Interventions

Type	Name	Description
BIOLOGICAL	Cipaglucosidase Alfa	Enzyme Replacement Therapy via intravenous infusion
DRUG	Miglustat	Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)

Timeline

• Start date: 2020-02-13
• Primary completion: 2026-06-01
• Completion: 2026-06-01
• First posted: 2019-04-11
• Last updated: 2025-10-27

Locations

17 sites across 6 countries: United States, Australia, Canada, Germany, Italy, Japan

Regulatory

• FDA-regulated drug study