drugset / Trial / NCT04808505
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
Non-randomizedParallel-groupOpen-labelTreatment
Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Timeline
- Start
- 2023-07-18
- Primary completion
- 2027-07
- Completion
- 2027-07
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Cipaglucosidase alfa | Protein / enzyme biologic | — | Intravenous |
| Subject | Miglustat | Small molecule | 65 mg | Oral |