Clinical Trial

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

Recruiting Phase 1
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Summary
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).
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2024-08-20; most recent amendment 2025-08-21.
Status change: Not Yet Recruiting → Recruiting 2024-10-29
Trial Details
NCT Number NCT06567769
Lead Sponsor GC Biopharma Corp
Conditions Sanfilippo Syndrome Type A
Enrollment 9 participants
Start Date 2024-11-21
Primary Completion 2027-06 (estimated)
Study Completion 2027-06 (estimated)
Updated on ClinicalTrials.gov 2025-08-28