Actively Recruiting
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Led by GC Biopharma Corp · Updated on 2025-08-28
9
Participants Needed
5
Research Sites
131 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants with documented MPS IIIA diagnosis
- Participants aged 6 12 months and 4 18 years
You will not qualify if you...
- Participants with significant non-MPS IIIA related central nervous system impairment
- Participants with previous complication from intraventricular drug administration
- Participants with contraindications for MRI scans and for neurosurgery
- Participants that received treatment with any investigational drug or a device intended as a treatment for MPS IIIA within 30 days or 5 half-lives prior to the study
- Participants that received a hematopoietic stem cell or bone marrow transplant or received gene therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
Actively Recruiting
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
Actively Recruiting
3
National Center for Child Health and Development
Setagaya City, Tokyo, Japan
Actively Recruiting
4
Ajou University Medical Center
Suwon, Gyeongi-do, South Korea
Actively Recruiting
5
Samsung Medical Center
Seoul, South Korea
Actively Recruiting
Research Team
G
GC Biopharma Corp.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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