drugset / Trial / NCT03708965
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
NaSingle-groupOpen-labelTreatment
Summary
A Phase II open-label, parallel group, 2 sites (Brazil), designed to evaluate the long term safety and efficacy of study drug for the treatment of the MPS II.
Timeline
- Start
- 2019-01-01
- Primary completion
- 2026-12-31
- Completion
- 2027-12-31
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | PABINAFUSP ALFA | Protein / enzyme biologic | 2 mg/kg | Intravenous |