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
Sex
ALL
Min age
12 Months
Max age
18 Years
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 stem cell or bone marrow transplant or received gene therapy
Primary outcome measure(s)
Incidences and characteristics of adverse events — up to 108 weeks
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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