Clinical Trial

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

Active, Not Recruiting Phase 3

Record status

This record was last updated October 27, 2025 (before its estimated June 2026 completion). Its status may not reflect the trial's current state.

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

Protocol Amendment History 16 amendments

This ClinicalTrials.gov record has been amended 16 times since 2019-04-09; most recent amendment 2025-10-24.

Status change: Recruiting → Active, Not Recruiting 2025-10-24

Status change: Not Yet Recruiting → Recruiting 2019-08-16

Trial Details

NCT Number	NCT03911505
Lead Sponsor	Amicus Therapeutics
Conditions	Pompe Disease (Late-onset)
Enrollment	21 participants
Start Date	2020-02-13
Primary Completion	2026-06 (estimated)
Study Completion	2026-06 (estimated)
Updated on ClinicalTrials.gov	2025-10-27