ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Brief 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
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date: February 13, 2020
Estimated Primary Completion Date: June 2026
Estimated Study Completion Date: June 2026
Arm:
- Experimental: Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221)
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 22 |
| Actual Study start date | 13 February 2020 |
| Estimated study completion date | 01 June 2026 |