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

Open to people ages 12 months to 18 years

• Participants with documented MPS IIIA diagnosis
• Participants aged ≥ 12 months and ≤ 18 years

• 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