NCT06567769PHASE1Recruiting
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
GC Biopharma Corp
Start Date
11/21/2024
Completion Date
6/1/2027
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).
Eligibility Criteria
Age Range: No minimum to 18 years
Inclusion Criteria:
* Participants with documented MPS IIIA diagnosis
* Participants aged ≥ 12 months and ≤ 18 years
Exclusion Criteria:
* 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
Interventions
DRUG
GC1130A
Conditions
Sanfilippo Syndrome Type A
Locations
UCSF Benioff Children's Hospital
Oakland, California 94609
United States
University of Minnesota
Minneapolis, Minnesota 55455
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