Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

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)

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).

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