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

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

Recruiting
12-18 years
All
Phase 1
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Overview

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

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

Study details
    Sanfilippo Syndrome Type A

NCT06567769

GC Biopharma Corp

16 October 2025

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