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Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Sponsor: GC Biopharma Corp
Summary
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).
Official title: A Phase 1, Open-label, Ascending Dose Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of Recombinant Human Heparan N-Sulfatase (rhHNS, GC1130A) Via Intracerebroventricular Access Device in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Key Details
Gender
All
Age Range
12 Months - 18 Years
Study Type
INTERVENTIONAL
Enrollment
9
Start Date
2024-11-21
Completion Date
2027-06
Last Updated
2025-08-28
Healthy Volunteers
No
Conditions
Interventions
GC1130A
ICV injection
Locations (5)
UCSF Benioff Children's Hospital
Oakland, California, United States
University of Minnesota
Minneapolis, Minnesota, United States
National Center for Child Health and Development
Setagaya City, Tokyo, Japan
Ajou University Medical Center
Suwon, Gyeongi-do, South Korea
Samsung Medical Center
Seoul, South Korea