Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
GC Biopharma Corp
Age
1–18
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Participants with documented MPS IIIA diagnosis
Participants aged ≥ 12 months and ≤ 18 years
You may not be if
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
Clareo Health | Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
