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
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
ClinicalTrials.gov ID: NCT03911505
Sponsor: Amicus Therapeutics
Information provided by: Amicus Therapeutics (Responsible Party)
Last Update Posted: 2024-09-19
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
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
Intervention / Treatment:
- Biological: Cipaglucosidase Alfa
- Drug: Miglustat
| Category | Value |
|---|---|
| Study Start (Actual) |
2020-02-13
|
| Primary Completion (Estimated) |
2026-06-01
|
| Study Completion (Estimated) |
2026-06-01
|
| Enrollment (Estimated) | 22 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
ATB200-04
|