drugset / Trial / NCT05422482
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug
Timeline
- Start
- 2022-09-20
- Primary completion
- 2027-06
- Completion
- 2027-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Idursulfase-ß | Unknown | 30 mg | Other |