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

Recruiting now · Phase 1

Conditions studied: Sanfilippo Syndrome Type A

In brief

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).

Key facts

Study ID

NCT06567769

Run by

GC Biopharma Corp

People needed

9

Starts

2024-11-21

Expected to finish

2027-06-01

Last updated by the study team

2025-08-28

Who can join

Age: 1 and older, up to 18. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify 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

Where it is running

• UCSF Benioff Children's Hospital — Oakland, California, United States (enrolling)
• University of Minnesota — Minneapolis, Minnesota, United States (enrolling)
• National Center for Child Health and Development — Setagaya City, Tokyo, Japan (enrolling)
• Ajou University Medical Center — Suwon, Gyeongi-do, South Korea (enrolling)
• Samsung Medical Center — Seoul, South Korea (enrolling)

Full record on ClinicalTrials.gov

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