Overview

ZIP Study - A Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe Disease

Status:
Recruiting

Trial end date:
2026-06-01

Target enrollment:

Participant gender:

Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of ATB200/AT2221 treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years

Phase:

Phase 3

Details

Lead Sponsor:

Amicus Therapeutics

Treatments:

Miglustat