Status:
RECRUITING
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Lead Sponsor:
GC Biopharma Corp
Conditions:
Sanfilippo Syndrome Type A
Eligibility:
All Genders
12-18 years
Phase:
PHASE1
Brief 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 intracereb...
Eligibility Criteria
Inclusion
- Participants with documented MPS IIIA diagnosis
- Participants aged ≥ 12 months and ≤ 18 years
Exclusion
- 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
Key Trial Info
Start Date :
November 21 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2027
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06567769
Start Date
November 21 2024
End Date
June 1 2027
Last Update
August 28 2025
Active Locations (5)
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1
UCSF Benioff Children's Hospital
Oakland, California, United States, 94609
2
University of Minnesota
Minneapolis, Minnesota, United States, 55455
3
National Center for Child Health and Development
Setagaya City, Tokyo, Japan
4
Ajou University Medical Center
Suwon, Gyeongi-do, South Korea