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A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
Sponsor: GC Biopharma Corp
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
Official title: An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment with Intravenous Drug
Key Details
Gender
All
Age Range
18 Months - 18 Years
Study Type
INTERVENTIONAL
Enrollment
12
Start Date
2022-09-20
Completion Date
2027-06
Last Updated
2024-10-15
Healthy Volunteers
No
Conditions
Interventions
GC1123
ICV-administered Hunterase, Idursulfase-ß
Locations (3)
Pusan National University Yangsan Hospital
Pusan, South Korea
Seoul National University
Seoul, South Korea
Samsung Medical Center
Seoul, South Korea