drugset / Trial / NCT04453085
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
RandomizedParallel-groupOpen-labelTreatment
Summary
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I
Timeline
- Start
- 2021-10-28
- Primary completion
- 2025-05-02
- Completion
- 2025-05-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | JR-171 | Protein / enzyme biologic | 2 mg/kg | Intravenous |
| Subject | JR-171 | Protein / enzyme biologic | 4 mg/kg | Intravenous |