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)
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).
Eligibility
- Age range
- 1–18 years
- Sex
- All
- Healthy volunteers
- No
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 ste…
Interventions
- DrugGC1130A
ICV injection
Locations (5)
- UCSF Benioff Children's HospitalOakland, California
- University of MinnesotaMinneapolis, Minnesota
- National Center for Child Health and DevelopmentSetagaya City, Tokyo
- Ajou University Medical CenterSuwon, Gyeongi-do
- Samsung Medical CenterSeoul